Conditions: Pemphigus Vulgaris
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 42
Healthy volunteers: no

Brief summary:
Open-label cohort study in adult patients with newly diagnosed or relapsing pemphigus vulgaris, with intra-patient dose-adjustment based on clinical response and BTK occupancy, and with conventional immunosuppressive "rescue treatment", if indicated. The duration of therapy in Part A will be 12 weeks, followed by 12 weeks of follow up. The extension phase, Part B includes 24 weeks of therapy, followed by 4 weeks of follow-up.

Detailed description:
Primary Objectives:

To evaluate the safety of PRN1008 in patients with pemphigus vulgaris (PV)

To evaluate the clinical activity of PRN1008 in patients with PV, per criteria in the European Academy of Dermatology and Venereology (EADV) 2014 Pemphigus S2 Guideline (Hertl et al. 2015)

Secondary Objectives

To evaluate the pharmacokinetics (PK) and the pharmacodynamics (PD) of multiple doses of PRN1008 in patients with PV

To evaluate the relationship of PK and PD to each other and to efficacy and safety in this patient population

Arms:
arm 1: Part A: Open-label PRN1008, 12 weeks; 12 weeks follow-up; Part B: Open-label PRN1008, 24 weeks; 4 weeks follow-up

Interventions: intervention 1: PRN1008
Intervention descriptions: intervention 1: Part A dosing was initiated administering 400 mg BID. Intrapatient dose adjustments (reductions and increases) were permitted based upon tolerability and clinical response with the maximum dose allowed up to 600 mg BID for 12 weeks.

Part B initial dosing was 400 mg QD for 2 weeks with dose escalation to 400 mg BID at the discretion of the Investigator for the purposes of investigating dose response and identifying the minimal efficacious dose of rilzabrutinib for 24 weeks.

Protocol document:
Prot_000.pdf: 
A Phase 2 Study of PRN1008 in Pemphigus 
Protocol PRN1008-005 Version 7.0 
Principia Biopharma Australia Pty Ltd. 
19 March 2019 
CLINICAL STUDY PROTOCOL 
Protocol Title: 
An Open-Label, Phase 2, Pilot Study Investigating the Safety, 
Clinical Activity, Pharmacokinetics, and Pharmacodynamics of 
Oral Treatment with the BTK Inhibitor PRN1008 in Patients with 
Newly Diagnosed or Relapsing Pemphigus Vulgaris 
Protocol No.: 
PRN1008-005 
Development Phase: 
Phase 2 
Investigational Product: 
PRN1008 
No.: (IND/CTD/CTN) 
EudraCT No. 2015-003564-37 
Protocol Date: 
19 March 2019, Version 7.0 
Sponsor: 
Principia Biopharma Australia Pty Ltd 
Level 29, 525 Collins Street 
Melbourne, VIC 3000 
Australia 
Principia Biopharma Inc. 
220 East Grand Avenue 
South San Francisco 
CA 94080 
USA 
Sponsor's Medical 
 
Monitor:  
CONFIDENTIALITY STATEMENT 
This document contains confidential information. It is provided for the sole use of the principal 
investigator, subinvestigators, staff, institutional review boards, human research ethics 
committees, and regulatory authorities. By accepting this document, you agree to maintain the 
information as confidential and to use it only for the purpose of conducting or reviewing the 
study protocol. 
NCT02704429


A Phase 2 Study of PRN1008 in Pemphigus 
Protocol PRN1008-005, Version 7.0 
Principia Biopharma Australia Pty Ltd.  
19 March 2019 
 
Page 2 of 93 
PROTOCOL SIGNATURE PAGE 
Product Name: 
PRN1008 
Version Number: 
7.0 
Release Date:  
19 March 2019 
 
The undersigned confirms that this Protocol is accurate. 
 
 
Signed: 
 


A Phase 2 Study of PRN1008 in Pemphigus 
Protocol PRN1008-005, Version 7.0 
Principia Biopharma Australia Pty Ltd.  
19 March 2019 
 
Page 3 of 93 
PROTOCOL ACCEPTANCE PAGE 
PROTOCOL NO.:  
PRN1008-005 
 
PROTOCOL TITLE: 
An Open-Label, Phase 2, Pilot Study Investigating the Safety, 
Clinical Activity, Pharmacokinetics, and Pharmacodynamics of 
Oral Treatment with the BTK Inhibitor PRN1008 in Patients with 
Newly Diagnosed or Relapsing Pemphigus Vulgaris 
 
 
PROTOCOL DATE: 
Amendment 7, 19 March 2019 
 
By signing this protocol acceptance page, I confirm that I have read, understood, and agree to 
conduct the study in accordance with the current protocol. 
 
 
 
 
Principal Investigator Name (Printed) 
 
 
 
 
Principal Investigator Signature 
 
Date 
 


A Phase 2 Study of PRN1008 in Pemphigus 
Protocol PRN1008-005, Version 7.0 
Principia Biopharma Australia Pty Ltd.  
19 March 2019 
 
Page 4 of 93 
Please return original Protocol Acceptance to Principia Biopharma Australia Pty Ltd. or 
designee. Please retain a copy for your study files. 
This study is to be conducted in accordance with the ethical principles that have their origin in 
the Declaration of Helsinki, The International Conference on Harmonization (ICH) Tripartite 
Guideline on Good Clinical Practice (GCP), Directive 2001/20/EC and 2005/28/EC, and the 
Title 21 Code of Federal Regulations Parts 50, 54, 56, 312, and 314, where appropriate. 
The study protocol and any amendments are to be reviewed by a Human Research Ethics 
Committee (HREC) before implementation. 
Written informed consent is to be obtained from each study participant prior to the conduct of 
any procedures which exceed or differ from standard practice at the study site. 
Confidential information contained in this document will not be used for any purpose other than 
the evaluation or conduct of the clinical investigation without prior written consent of Principia 
Biopharma Australia Pty Ltd. 


A Phase 2 Study of PRN1008 in Pemphigus 
Protocol PRN1008-005, Version 7.0 
Principia Biopharma Australia Pty Ltd.  
19 March 2019 
 
Page 5 of 93 
TABLE OF CONTENTS 
PROTOCOL SIGNATURE PAGE .................................................................................................2 
PROTOCOL ACCEPTANCE PAGE ..............................................................................................3 
TABLE OF CONTENTS .................................................................................................................5 
LIST OF APPENDICES ..................................................................................................................9 
TABLE OF TABLES ......................................................................................................................9 
TABLE OF FIGURES .....................................................................................................................9 
STUDY SYNOPSIS ......................................................................................................................10 
GLOSSARY OF ABBREVIATIONS AND TERMS ...................................................................20 
1 
BACKGROUND AND SCIENTIFIC RATIONALE FOR THE TREATMENT OF 
PEMPHIGUS WITH PRN1008 .........................................................................................22 
1.1 
Background ............................................................................................................22 
1.2 
Rationale for PRN1008 Treatment in Pemphigus .................................................22 
1.3 
Summary of PRN1008 Animal Toxicity Studies ...................................................23 
1.3.1 
13-week GLP Toxicology .......................................................................23 
1.3.2 
6 and 9-month GLP Toxicology .............................................................24 
1.3.3 
Reproductive and Fertility Studies..........................................................24 
1.4 
Summary of PRN1008 Clinical Experience ..........................................................25 
1.4.1 
Experience in Healthy Volunteer Phase 1 Studies ..................................25 
1.4.2 
Experience in Pemphigus Patients in Study PRN1008-005 ...................27 
1.5 
Clinical Pharmacology of PRN1008 ......................................................................29 
1.5.1 
Pharmacokinetics in Healthy Volunteers ................................................29 
1.5.2 
Pharmacokinetics and BTK Occupancy in Pemphigus Patients .............29 
1.5.3 
BTK Occupancy in Patients with Pemphigus .........................................30 
2 
RATIONALE AND JUSTIFICATION FOR THE STUDY AND THE STUDY 
DESIGN .............................................................................................................................32 
2.1 
Rationale for the Study Design ..............................................................................32 
2.2 
Selection of the Starting Clinical Dose of PRN1008 in Pemphigus ......................32 
2.3 
Overall Risk/Benefit Assessment ..........................................................................33 
2.4 
Drug-Drug Interaction Potential ............................................................................33 
3 
OBJECTIVES OF THE STUDY .......................................................................................35 
3.1 
Primary Objectives .................................................................................................35 
3.2 
Secondary Objective ..............................................................................................35 
3.3 
Exploratory Objective ............................................................................................35 
4 
INVESTIGATIONAL PLAN ............................................................................................36 
4.1 
Overall Study Design and Plan ..............................................................................36 
4.2 
Number of Participants ..........................................................................................36 
4.3 
Study Duration and Duration of Subject Participation ..........................................36 


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4.4 
End of Study Definition .........................................................................................36 
4.5 
PRN1008 Administration.......................................................................................37 
4.5.1 
Rationale for PRN1008 Dosage Selection ..............................................37 
4.5.2 
Concomitant Medications .......................................................................37 
4.6 
PRN1008 Intrapatient Dose Adjustment ...............................................................38 
5 
PROTOCOL DEVIATIONS .............................................................................................40 
6 
STUDY POPULATION ....................................................................................................41 
6.1 
Inclusion Criteria (Part A and Part B unless noted below) ....................................41 
6.2 
Exclusion Criteria ..................................................................................................41 
6.3 
Prior and Concomitant Therapy .............................................................................43 
6.3.1 
Prior Therapy ..........................................................................................43 
6.3.2 
Concomitant Therapy..............................................................................44 
7 
LIST OF SCREENING ASSESSMENTS .........................................................................45 
8 
LIST OF STUDY ASSESSMENTS ..................................................................................46 
8.1 
Clinical Assessments Part A and B ........................................................................46 
8.2 
Laboratory Assessments Part A and B ...................................................................46 
8.3 
Safety and Tolerability Assessments .....................................................................47 
9 
STUDY TREATMENT .....................................................................................................48 
10 
IDENTITY OF INVESTIGATIONAL PRODUCT ..........................................................49 
10.1 
Formulation ............................................................................................................49 
10.2 
Packaging ...............................................................................................................49 
10.3 
Storage and Handling .............................................................................................49 
10.4 
Drug Accountability...............................................................................................49 
10.5 
Destruction of Investigational Product ..................................................................50 
11 
STATISTICAL METHODS AND DETERMINATION OF SAMPLE SIZE ..................51 
11.1 
Primary Outcome Measures ...................................................................................51 
11.1.1 
Safety ......................................................................................................51 
11.1.2 
Efficacy ...................................................................................................51 
11.2 
Secondary Outcome Measures ...............................................................................51 
11.3 
PK/PD Outcome Measures ....................................................................................52 
11.3.1 
PK Outcome Measures ...........................................................................52 
11.3.2 
PD Outcome Measures ...........................................................................52 
11.3.3 
Exploratory PK/PD Analysis ..................................................................52 
11.4 
Determination of Sample Size ...............................................................................52 
11.5 
Analysis Populations ..............................................................................................52 
11.5.1 
Screening Population ..............................................................................52 
11.5.2 
Safety Analysis Population .....................................................................52 
11.5.3 
Efficacy Analysis Population ..................................................................52 


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11.5.4 
Pharmacokinetic Analysis Population ....................................................53 
11.6 
Randomization and Blinding Procedures ...............................................................53 
11.7 
Subject Numbers and Treatment Assignments ......................................................53 
11.8 
Subject Disposition, Subject Replacement, and Demographics and Baseline 
Characteristics ........................................................................................................53 
11.8.1 
Disposition ..............................................................................................53 
11.8.2 
Replacement of Subjects .........................................................................53 
11.8.3 
Demographics and Baseline Characteristics ...........................................53 
11.9 
Efficacy Analysis ...................................................................................................54 
11.9.1 
Subject Response and Disease Progression ............................................54 
11.10 Safety Analysis ......................................................................................................54 
11.10.1 Adverse Events .......................................................................................54 
11.10.2 Clinical Laboratory Tests ........................................................................54 
11.10.3 Vital Signs ...............................................................................................55 
11.10.4 Concomitant Medications .......................................................................55 
11.11 Pharmacokinetics/Pharmacodynamics Analyses ...................................................55 
11.11.1 Missing PK/PD Values ...........................................................................55 
11.12 Statistical and Analytical Methods ........................................................................55 
11.12.1 Descriptive Summaries and Inference ....................................................55 
11.12.2 Exploratory Analyses ..............................................................................56 
11.12.3 Interim Analysis ......................................................................................56 
12 
SAFETY AND TOXICITY MANAGEMENT .................................................................57 
12.1 
Safety Monitoring Committee ...............................................................................57 
12.2 
Adverse Event Collection Period ...........................................................................57 
12.3 
Clinical Adverse Events .........................................................................................57 
12.4 
Adverse Event Intensity Grading ...........................................................................59 
12.5 
Adverse Event Relationship to Study Drug ...........................................................59 
12.6 
Treatment and Follow-Up of Adverse Events .......................................................59 
12.7 
Laboratory and ECG Abnormalities ......................................................................60 
12.7.1 
Follow-Up of Abnormal Laboratory Test Values ...................................60 
12.8 
Serious Adverse Event (SAE) Reporting ...............................................................60 
12.8.1 
SAE Definitions ......................................................................................60 
12.8.2 
SAE Reporting ........................................................................................61 
12.8.3 
Other Safety Findings Requiring Expedited Reporting ..........................62 
12.9 
Pregnancy ...............................................................................................................62 
13 
DATA QUALITY ASSURANCE .....................................................................................63 
13.1 
Assignment of Preferred Terms and Original Terminology ..................................63 
14 
ETHICAL ASPECTS ........................................................................................................64 


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14.1 
Local Regulations/Declaration of Helsinki ............................................................64 
14.2 
Subject Informed Consent......................................................................................64 
14.3 
Institutional Review Board Review .......................................................................64 
14.4 
Conditions for Modifying the Protocol ..................................................................65 
14.5 
Conditions for Terminating the Study ...................................................................65 
14.6 
Financial Disclosure ...............................................................................................65 
15 
STUDY DOCUMENTATION, MONITORING, CASE REPORT FORMS, AND 
RECORD RETENTION REQUIREMENTS ....................................................................66 
15.1 
Investigator's File/Retention of Records ................................................................66 
15.2 
Source Documents and Background Data .............................................................66 
15.3 
Audits and Inspections ...........................................................................................67 
15.4 
Case Report Forms .................................................................................................67 
15.5 
Study Monitoring ...................................................................................................67 
15.6 
Confidentiality of Clinical Trial Documents and Participants' Medical 
Records ..................................................................................................................67 
15.7 
Clinical Study Report .............................................................................................68 
15.8 
Publication of Data and Protection of Trade Secrets .............................................68 
16 
STUDY ADMINISTRATIVE PROCEDURES ................................................................69 
16.1 
Subject Recruitment Procedures ............................................................................69 
16.2 
Subject Enrollment Procedures ..............................................................................69 
16.3 
Patient Premature Withdrawal – Definition ...........................................................69 
16.4 
Procedures for Patients Who Withdraw from the Study ........................................69 
16.5 
Treatment Compliance ...........................................................................................70 
16.6 
Recording of AEs on the CRF ...............................................................................70 
16.7 
Physical Examination Procedures ..........................................................................70 
16.8 
Vital Signs Procedures ...........................................................................................71 
16.9 
Body Weight ..........................................................................................................71 
16.10 ECG Procedures .....................................................................................................71 
16.11 Laboratory Test Procedures ...................................................................................71 
16.11.1 Recording of Laboratory Test Results on the CRF .................................72 
16.12 Recording of Concomitant Medications on the CRF .............................................72 
17 
REFERENCES ..................................................................................................................73 
 


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LIST OF APPENDICES 
APPENDIX 1A 
Corticosteroid Rescue Criteria and Treatment Protocol (Part A) ....................75 
APPENDIX 1B 
Corticosteroid Rescue Criteria and Treatment Protocol (Part B) ....................77 
APPENDIX 2 
Strong And Moderate Cyp3a Inhibitors And Inducers ....................................79 
APPENDIX 3 
Sensitive CYP3A Substrates Including Those with Narrow Therapeutic 
Index ................................................................................................................80 
APPENDIX 4 
Schedule of Assessments .................................................................................81 
APPENDIX 5 
Pemphigus Disease Activity and Quality of Life Evaluation 
Instruments:  ABQOL, Tabqol, PDAI, ABSIS ................................................85 
APPENDIX 6 
Simple Nutritional Appetite Questionnaire (SNAQ) .......................................92 
APPENDIX 7 
Corticosteroid Taper Protocol ..........................................................................93 
 
TABLE OF TABLES 
Table 1 
TEAEs Reported by More than One Patient (N=21) .......................................29 
Table 2 (Part A) General Dose Adjustment Guidelines for Dose Selection in the First 
4 Weeks ............................................................................................................39 
Table 3 (Part B) 
General Dose Escalation Guidelines ................................................................39 
Table 4 
Dose Escalation Rules Part B ..........................................................................77 
 
TABLE OF FIGURES 
Figure 1 
Individual and Mean BTK Occupancy Data from HV Study 
PRN1008-001 with the Liquid Formulation of PRN1008* .............................26 
Figure 2 
PDAI and Clinical Response Patient Profiles of 5 Patients on Zero or 
Doses of CS ≤ 5 mg .........................................................................................27 
Figure 3 
Timing of Individual Clinical Responses (Day 15 to 29 circled) N=21 ..........28 
Figure 4 
Distribution of Plasma Concentrations at BTK Occupancy Timepoints .........30 
Figure 5 
BTK Occupancies (Mean and Standard Deviation) Based on 
Approximate Timepoints Following Dosing ...................................................31 
 


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STUDY SYNOPSIS 
Study Title: 
An Open-Label, Phase 2, Pilot Study Investigating the Safety, Clinical 
Activity, Pharmacokinetics, and Pharmacodynamics of Oral Treatment with 
the BTK Inhibitor PRN1008 in Patients with Newly Diagnosed or Relapsing 
Pemphigus Vulgaris 
Study Number: 
PRN1008-005 
Indication: 
Pemphigus: Pemphigus Vulgaris (PV) 
Background: 
PV, which is characterized by intraepidermal blisters in skin and mucosae, is 
known to be driven by autoantibodies to epidermal proteins and is responsive 
acutely to the anti-inflammatory effects of corticosteroids and chronically to 
B-cell depletion by anti-CD20 therapy. Principia Biopharma has developed a 
novel, highly selective, small molecule inhibitor of non-T cell white blood 
cell signaling (via B- cell receptor, FCɣR, FcεR signaling of the Bruton’s 
tyrosine kinase, i.e., BTK, pathway). In naturally occurring canine 
pemphigus foliaceus (PF), PRN1008, rapidly reversed disease without the 
need for corticosteroid treatment. In Phase 1 studies with 114 healthy 
volunteers, target BTK occupancy levels were safely and consistently 
exceeded, suggesting PRN1008 may be highly effective in human 
pemphigus and other autoimmune diseases. 
 
 
 
 
 
 
 
 
Refer to PRN1008 Investigators’ Brochure, for additional information. 
Study Sites: 
Multiple international sites 
Study Phase: 
Phase 2 


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Study Objectives: 
Primary Objectives: 
• 
To evaluate the clinical safety of PRN1008 in patients with PV over a 
12-week (Part A) or 24-week (Part B) treatment period 
• 
To evaluate the clinical activity of PRN1008 in patients with PV, per 
criteria in the European Academy of Dermatology and Venereology 
(EADV) 2014 Pemphigus S2 Guideline (Hertl et al. 2015)* 
*As modified to define CR without 2-month durability definition 
Secondary Objective: 
• 
To evaluate the pharmacokinetics (PK) and the pharmacodynamics (PD) 
of PRN1008 in patients with PV 
Exploratory Objective: 
• 
To evaluate the relationship of PK and PD to each other and to efficacy 
and safety in this patient population 
Study Design: 
Part A: 
Open-label cohort study, with intrapatient dose-adjustment based on clinical 
response and BTK occupancy, and with conventional immunosuppressive 
“rescue treatment”, if indicated. 
Initial dosing will be 400 mg twice daily (bid). The duration of study therapy 
will be 12 weeks starting on Day 1 and ending on study Day 84 (total 
duration of individual subject participation is 28 weeks). 
Part B: 
Initial dosing will be 400 mg once daily (qd), with intra-patient dose 
escalation to 400 mg bid allowed at or after the Week 3 visit for insufficient 
clinical response (and then again to 600 mg bid if necessary at or after the 
Week 5 visit). The duration of study therapy will be 24 weeks starting on 
Day 1 and ending on study Day 169 (total duration of individual subject 
participation is approximately 32 weeks). 
Both Parts: 
The first dose on Day 1 of Week 1 will be supervised in the clinic and the 
patient observed for approximately 2 hours, at which time a PK/PD sample 
will be drawn. Day 2 study medication will be withheld in the morning in 
order to gain the trough BTK occupancy approximately 12 (when bid 
dosing) or approximately 24 hours (when qd dosing) after the prior dose. 
Where follow-up is not feasible the next day, another day in the first week of 
treatment may be used to get trough occupancy instead, again withholding 
dose on the morning of that day to get an occupancy measurement close to 
12 (when bid dosing) or approximately 24 (when qd dosing) hours post dose. 
At other follow-up visits, BTK occupancy can be measured at any time after 
the usual morning dose of PRN1008. 
The maximum dose of PRN1008 in this study, after dose adjustment, will be 
600 mg bid. 


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Corticosteroid 
Rescue Criteria 
Guidelines: 
Systemic corticosteroids will be avoided during PRN1008 therapy unless 
“rescue criteria” are triggered, or the patient enters the study on low-dose 
corticosteroids (i.e., ≤ 0.5 mg/kg). Corticosteroid rescue criteria are listed in 
Appendix 1. 
Outcome Measures: 
Primary Outcome Measures: 
Safety: 
The incidence of treatment-emergent AEs (TEAEs), including clinically 
significant changes in physical examination, laboratory tests, and vital signs. 
Efficacy: 
The proportion of subjects who are able to achieve control of disease activity 
(CDA) within 4 weeks of starting PRN1008 treatment without the need for 
doses of prednis(ol)one > 0.5 mg/kg. 
Secondary Outcome Measures:* 
• 
Proportion of subjects able to achieve CDA without corticosteroids 
within 4 weeks 
• 
Proportion of subjects able to achieve a complete response (CR) 
without corticosteroids within 12 weeks (and also 24 weeks in Part B) 
• 
Proportion of subjects able to achieve CR without the need for doses 
of prednis(ol)one of greater than 0.5mg/kg within 12 weeks (and also 
24 weeks in Part B) 
• 
Time to CDA 
• 
Time to CR 
• 
Time to end of consolidation phase 
• 
Time to relapse after PRN1008 treatment discontinuation 
• 
Cumulative corticosteroid usage over the first 12 weeks (and also 
24 weeks in Part B) 
• 
Change from baseline in Pemphigus Disease Area Index (PDAI) and 
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) scores at 
each follow-up visit 
• 
Change from baseline in Autoimmune Bullous Diseases Quality of 
Life (ABQOL) and Treatment of Autoimmune Bullous Diseases 
Quality of Life (TABQOL) scores at each follow-up visit 
• 
Change from baseline in appetite (SNAQ score) at each follow-up visit 
*Clinical activity endpoints as defined by the EADV 2014 pemphigus S2 
guideline (Hertl et al. 2015) 
Pharmacokinetics Outcome Measure: 
• 
Plasma concentrations of PRN1008 at approximately the time of 
maximum concentration on Day 1 and at varied subsequent times 
during outpatient dosing 


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Pharmacodynamic Outcome Measures: 
• 
Percentage BTK occupancy in PBMCs at 2 & 24 hours after the first 
PRN1008 dose and at various subsequent times during outpatient 
dosing 
• 
Change from baseline in anti-dsg1-3 autoantibody levels by enzyme-
linked immunosorbent assay (ELISA) at various time points. 
Exploratory Outcome Measures: 
Exploratory PK/PD analysis will examine the effects, if any, of covariates on 
PK and/or PD, and the relationship between PK, PD, and efficacy in this 
population. 
Planned Number of 
Participants and 
Interim Analysis: 
 
 
Participant Selection 
Criteria: 
Inclusion Criteria Part A and Part B (unless noted below): 
1. 
Male or female patients, aged 18 to 80 years old, with biopsy-proven 
(positive direct immunofluorescence and appearance on H&E 
microscopy), mild-moderate PV in Part A (PDAI 8 to 45) and mild-
severe PV in Part B (PDAI 8 to 60) 
2. 
Newly diagnosed or relapsing patients for whom an initial period of 
PRN1008 monotherapy or combination therapy with low-dose 
corticosteroids (≤0.5 mg/kg of prednis[ol]one or equivalent), is judged 
clinically acceptable, provided tapering of the corticosteroid treatment 
regimen is anticipated with good clinical response to PRN1008 
3. 
BMI > 17.5 and < 40 kg/m2 Part A only  
4. 
Adequate hematologic, hepatic, and renal function (absolute neutrophil 
count ≥ 1.5 X 109/L, Hgb > 9 g/dL, platelet count ≥ 100 X 109/L, 
AST/ALT ≤ 1.5 x ULN, albumin ≥ 3 g/dL, creatinine ≤ ULN (Part A) 
and creatinine ≤ 1.5 x ULN (Part B) 
5. 
Female patients who are of reproductive potential must agree for the 
duration of active treatment in the study to use an effective means of 
contraception (hormonal contraception methods that inhibits ovulation, 
intrauterine device, intrauterine hormone-releasing system, bilateral 
tubal ligation, vasectomized partner, condoms or sexual abstinence). 
Unless surgically sterile, postmenopausal females should have 
menopause confirmed by FSH testing 
6. 
Able to provide written informed consent and agreeable to the schedule 
of assessments 
Exclusion Criteria Part A and Part B: 
1. Previous use of a BTK inhibitor.  
Patients enrolled in a previous version of this protocol who are 
still in their 12-week active treatment period with PRN1008 are 
eligible to continue treatment, initially with their current dose 
level, under this amended protocol for an additional 12 weeks, i.e. 


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24 weeks total, following review and signature of the EC 
approved patient’s consent. Patients who completed Part A and 
did not discontinue the study due to a medical condition that 
might compromise safety assessments or for a PRN1008 related 
adverse event may be screened for entry under Part B.  
2. 
Pregnant or lactating women 
3. 
ECG findings of QTc > 450 msec (males) or > 470 msec (females), 
poorly controlled atrial fibrillation (i.e. symptomatic patients or a 
ventricular rate above 100 beats/min on ECG), or other clinically 
significant abnormalities 
4. 
A history of malignancy of any type, other than surgically excised non-
melanoma skin cancers or in situ cervical cancer within 5 years before 
the day of dosing 
5. 
Use of immunologic response modifiers with the following periods 
prior to Day 1: as concomitant therapy, other immunologic response 
modifiers not detailed in this exclusion apart from corticosteroids; 1 
week: cyclophosphamide; 4 weeks: IVIG , Kinaret (anakinra) and 
Enbrel (etanercept); 12 weeks: Remicade (infliximab), Humira 
(adalimumab), Simponi (golimumab), Orencia (abatercept), Actemra 
(tocilizumab), Cimzia (certolizumab), Cosentyx (secukinumab), 
plasmapheresis; 6 months: Rituxan/MabThera (rituximab), 
ofatumumab, any other anti-CD20 antibody, other long-acting biologics 
6. 
More than 0.5 mg/kg of prednis(ol)one per day (“low dose 
corticosteroids”) within the two weeks prior to Day 1 
7. 
Use of proton pump inhibitor drugs such as omeprazole and 
esomeprazole (it is acceptable to change patient to H2 receptor blocking 
drugs prior to the first dose of PRN1008) 
8. 
Concomitant use of known strong-to-moderate inducers or inhibitors of 
CYP3A (Appendix 2) within 3 days or 5 half-lives (whichever is 
longer) of study drug dosing 
9. 
Use of CYP3A-sensitive substrate drugs (Appendix 3) with a narrow 
therapeutic index within 3 days or 5 half-lives (whichever is longer) of 
study drug dosing including, but not limited to, alfentanil, astemizole, 
cisapride, cyclosporine, dihydroergotamine, ergotamine, fentanyl, 
pimozide, quinidine, sirolimus, tacrolimus, or terfenadine 
10. Has received any investigational drug (or is currently using an 
investigational device) within the 30 days before receiving the first dose 
of study medication, or at least 5 times the respective elimination half-
life time (whichever is longer) 
11. History of drug abuse within the previous 12 months 
12. Alcoholism or excessive alcohol use, defined as regular consumption of 
more than approximately 3 standard drinks per day 


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13. Refractory nausea and vomiting, malabsorption, external biliary shunt, 
or significant bowel resection that would preclude adequate study drug 
absorption 
14. History of anorexia nervosa or periods of three months or more of low 
body weight (BMI < 17.5) in the past 5 years 
15. Donation of a unit or more of blood or blood products within 4 weeks 
prior to Day 1 
16. History of solid organ transplant 
17. History of epilepsy or other forms of seizure in the last 5 years 
18. Positive for screening for HIV, hepatitis B (surface and core antibodies 
unrelated to vaccination), or hepatitis C (anti-HCV antibody confirmed 
with Hep C RNA) 
19. Positive interferon-gamma release assay (IGRA) (e.g., T-spot TB Test, 
QuantiFERON®-TB Gold, or QuantiFERON®-TB Gold Plus (QFT 
Plus), at Screening.  Unless, the patient has latent TB and all of the 
following 3 conditions are true: 
a) Chest X-ray does not show evidence suggestive of active 
tuberculosis (TB) disease 
b) There are no clinical signs and symptoms of pulmonary and/or extra-
pulmonary TB disease 
c) Documented receipt of one of the following prophylactic treatment 
regimens: 
i. Oral daily Isoniazid for 6 months 
or 
ii. Oral daily Rifampin (RIF) for 4 months 
or 
iii. Isoniazid and Rifapentine weekly for 3 months 
(3HP) 
On a case by case basis, after discussion and approval by the Sponsor, a 
local TB test that is negative and is considered equivalent to 1 of the 
above tests may be used for eligibility. For example, if a 
QuantiFERON®-TB Gold, or QuantiFERON-TB Gold Plus (QFT Plus) 
is positive and a local blood test or T-Spot TB test is negative, the 
patient may be enrolled using the local result upon approval of the 
Sponsor. 
20. History of serious infections requiring intravenous therapy with the 
potential for recurrence 
21. Live vaccine within 28 days prior to baseline or plan to receive one 
during the study 


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22. Any other clinically significant disease, condition, or medical history 
that, in the opinion of the Investigator, would interfere with subject 
safety, study evaluations, and/or study procedures 
Test Product and 
Method of Dosing 
PRN1008 300 mg and 100 mg tablets. Tablets should be taken with a glass of 
water and may be taken with or without food, i.e., a period of fasting is not 
required. 
Concomitant CYP3A 
Inducers or 
Inhibitors, Clinically 
Relevant CYP3A 
Substrate Drugs, 
Acid-Reducing Drugs 
and Anticoagulant 
Monitoring 
Inducers and inhibitors of Cytochrome P450 3A (CYP3A) should be 
avoided, as they may reduce or increase the exposure of PRN1008 
(Appendix 2) when administered concomitantly. 
PRN1008 is a moderate CYP3A inhibitor and can increase the exposure of 
drugs metabolized by CYP3A when administered concomitantly. Narrow 
therapeutic index CYP3A substrate drugs should be avoided (Appendix 3) 
and care should be taken with other drugs metabolized by the CYP3A. 
Clinically relevant CYP3A substrate drugs, (e.g. “sensitive substrate” listed 
in Appendix 3), should be managed by administering PRN1008 on a time 
schedule such that CYP3A substrate drugs can be given 2 hours or more 
after PRN1008. 
Proton pump inhibitors reduce the exposure of PRN1008 by approximately 
50% due to the effects of a lack of an acidic environment on tablet 
bioavailability. Therefore, subjects who are on proton pump inhibitors 
should be changed to H2 receptor blocking drugs or not enrolled in the study. 
PRN1008 should be given at least 2 hours before any dose of an H2 receptor 
blocking drug or any antacid. 
Anticoagulation with warfarin should be monitored actively in the first 
weeks of PRN1008 therapy, as the combination has not been studied 
previously (interaction is not suggested to be likely based on in vitro 
metabolism studies). 
Screening Procedures 
Part A and Part B: 
Up to 28 days before enrollment into the study, participants will be required 
to sign a consent form, after which screening assessments will be carried out 
as follows: 
• 
Review of medical history including concomitant medications 
• 
PDAI and ABSIS scoring 
• 
Review of inclusion and exclusion criteria 
• 
Measurement of height and weight 
• 
Physical examination 
• 
12-Lead ECG 
• 
Vital signs (blood pressure, heart rate, respiration rate and 
temperature) 
• 
Clinical laboratory testing (hematology, coagulation, serum chemistry 
and urinalysis) 
• 
HIV, hepatitis B (surface antigen and core antigen  and antibodies, 
hepatitis C (anti-HCV antibody confirmed with Hep C RNA) 


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• 
TB screen with T-spot TB Test, or QuantiFERON®-TB Gold test, or 
QuantiFERON®-TB Gold Plus (QFT Plus) 
• 
FSH (in postmenopausal women only who are not surgically sterile) 
• 
Pregnancy test in women of childbearing potential (serum test done at 
screening, urine dip test done at other time points) 
• 
Skin biopsy if not already performed: lesional for H&E staining, 
perilesional for direct immunofluorescence  
Randomization 
Procedure: 
Not applicable; the study is open-label.  
Study Assessments 
(see Schedule in 
Appendix 4): 
Part A: Patients will be screened within 28 days of dosing and will return for 
an end-of-study assessment 84 days after receiving their final dose of study 
drug. During the study, patients will return at specified times on an 
outpatient basis for assessment of vital signs, physical examination, 
assessment of AEs, assessment of concomitant medication use, assessment 
of clinical benefit, and provision of blood samples for PK and PD, and other 
clinical laboratory tests.  
Note: Patients who are currently enrolled in this PRN1008-005 study 
(Part A) and are in the 12-week active treatment period with PRN1008 are 
eligible to continue treatment at their current dose up to a total of 24 weeks 
active treatment, as a Part B patient, upon review and signature of the Part B 
EC-approved Patient Informed Consent Form. Patients that completed 
Part A of the trial are also eligible to be screened as a Part B patient, upon 
review and signature of the Part B EC-approved Patient Informed Consent 
Form. 
Part B: Patients will be screened within 28 days of dosing and will return for 
an end-of-treatment assessment on Day 169 after receiving their final dose of 
study drug on the morning of Day 169. They will return for follow up 28 days 
later for a final, follow up visit. During the study, patients will return at 
specified times on an outpatient basis for assessment of vital signs, physical 
examination, assessment of AEs, assessment of concomitant medication use, 
assessment of clinical benefit, and provision of blood samples for PK and 
PD, and other clinical laboratory tests. 
Clinical Assessments Part A and B: 
• 
Medical history, including evaluation of any on-study AEs and 
periodic vital signs (body temperature, heart rate, respiratory rate, 
blood pressure), concomitant medication use 
• 
At screening only: height and weight, full physical examination, and 
12-lead electrocardiogram (ECG) (an extra ECG may be taken at 
additional visits, if indicated) 
• 
Disease activity scores: PDAI, ABSIS (Appendix 5) 
• 
Standardized photography of the affected area (Optional) 
• 
Quality of Life Scores: ABQOL, TABQOL (Appendix 5) 
• 
SNAQ appetite questionnaire (Appendix 6) 


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Laboratory Assessments Part A and Part B: 
•
Hematology: hemoglobin, hematocrit, erythrocyte count (RBC’s), 
thrombocyte count (platelets), leukocyte count (WBCs) with 
differential in absolute counts (including neutrophils, eosinophils, 
basophils, lymphocytes, and monocytes)
•
Coagulation:  PT/INR, thrombin time, aPTT, fibrinogen level
•
Serum chemistry: Aspartate aminotransferase (AST), Alanine 
aminotransferase (ALT), Total, direct, and indirect bilirubin levels, 
Alkaline phosphatase (ALP), Albumin, Creatinine, Urea, Total 
Protein, Sodium, Chloride, Calcium, Phosphate, Potassium, Glucose 
(random), creatine phosphokinase (CPK) and thyroid stimulating 
hormone (TSH)
•
PK: Plasma PRN1008 concentration
•
PD: BTK occupancy in PBMCs and anti-desmoglein -1
and -3 autoantibody titers by ELISA
•
Urinalysis:  pH, specific gravity, protein, glucose, ketones, bilirubin, 
blood, nitrites, urobilinogen and leukocytes measured by dip stick
•
Serology:  HIV, Hepatitis B (surface antigen and core antigen and 
antibodies) and Hepatitis C and T-spot TB® Test, QuantiFERON®-
TB Gold, or QuantiFERON-TB Gold Plus (QFT Plus) testing at 
screening only
•
FSH for postmenopausal women who are not surgically sterile at 
screening only
•
Pregnancy test for women of childbearing potential 
Safety and 
Tolerability 
Assessments Part A 
and B: 
Specific assessments to evaluate treatment safety include the following: the 
frequency and type of AEs, clinical laboratory testing, SNAQ appetite 
questionnaire (Appendix 6) and vital signs.  
Patients will remain under observation in the clinic for 2 hours after 
administration of the first dose and until the PK/PD sample is drawn. 
A Safety Monitoring Committee (SMC) will regularly review and evaluate 
patient safety data including AEs, laboratory results, concomitant 
medications, dose modifications, and efficacy. 
Statistical Analysis: 
Clinical Activity: 
Given the natural history of PV to not remit spontaneously, a study of 
approximately 25 PV patients completing 12 weeks of treatment and 
approximately 25 PV patients completing 24 weeks of treatment with 
PRN1008 should determine a reasonable approximation of the responder rate 
and average time to response (e.g., estimated response rate of 50% with 80% 
CI of ± 13%). Where appropriate, patients from both parts will be combined 
in analysis. The results from this pilot study will be used to design 
confirmatory, pivotal clinical trials. Time course of changes in clinical and 
laboratory parameters will be described graphically, and at each visit using 
descriptive statistics. 


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Safety and Tolerability: 
Quantitative safety data will be summarized by descriptive statistics 
(arithmetic mean, standard deviation, median, minimum, and maximum) by 
dose level. Summaries will also be presented for the change from baseline, 
when appropriate.  
Pharmacokinetics and Pharmacodynamics: 
Individual and group PK and PD data will be summarized, displayed 
graphically, and by descriptive statistics for each visit, where measured. 
Study Duration: 
Approximately 48 months from the first subject treated to last subject 
completing, and 28 (Part A) – 32 (Part B) weeks per subject. 
 


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GLOSSARY OF ABBREVIATIONS AND TERMS  
ABBREVIATION OR TERM 
DEFINITION 
ABSIS 
Autoimmune Bullous Skin Disorder Intensity Score 
ABQOL 
Autoimmune Bullous Diseases Quality of Life (assessment) 
AE 
Adverse event 
Ae 
Amount excreted unchanged in the urine 
ALP 
Alkaline phosphatase 
ALT 
Alanine aminotransferase 
ANOVA 
Analysis of variance 
AST 
Aspartate aminotransferase 
AUC 
Area under the plasma concentration-time curve 
BCR 
B-cell receptor 
BID 
Twice daily (morning and evening) 
BMI 
Body mass index 
BP 
Blood pressure 
BPM 
Beats per minute 
BTK 
Bruton’s Tyrosine Kinase 
CA 
Competent Authority 
CI 
Confidence Interval 
CLr 
Renal clearance 
Cmax 
Maximum observed plasma concentration 
CNS 
Clinical Network Services 
CPK 
Creatine phosphokinase 
CR 
Clinical Response 
CRF 
Case report form 
CRO 
Contract research organization 
CTCAE 
Common Terminology Criteria for AEs 
CV 
Coefficient of Variation 
CYP 
Cytochrome P450 
DBP 
Diastolic blood pressure 
DSG 
Desmoglein 
EC 
Ethics Committee (see also HREC) 
ECG 
Electrocardiogram 
EDC 
Electronic Data Capture 
ELISA 
Enzyme-Linked Immunosorbent Assay 
FSH 
Follicle Stimulating Hormone 
FDA 
Food and Drug Administration 
GLP 
Good Laboratory Practice 
H2 
Histamine two (receptor) 
HBsAg 
Hepatitis B surface antigen 
HPMC 
Hypromellose 
HCV 
Hepatitis C Virus 
HDL 
High density lipoprotein 
HDPE 
High-density polyethylene 
HIV 
Human Immunodeficiency Virus 
HR 
Heart rate 
HREC 
Human Research Ethics Committee 
IB 
Investigator’s Brochure 
ICF 
Informed Consent Form 
ICH 
International Conference on Harmonization  


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ABBREVIATION OR TERM 
DEFINITION 
IMP 
Investigational medicinal product 
IR 
Immediate Release (tablet formulation) 
IRB 
Institutional Review Board (Human Research Ethics Committee) 
IVIG 
Intravenous immunoglobulin 
LDL 
Low density lipoprotein 
LPLV 
Last participant last visit  
LTFU 
Long-term Follow-up 
MAD 
Multiple ascending dose (trial) 
MedDRA 
Medical Dictionary for Regulatory Activities  
N 
Sample Size 
NOEL 
No observed effect level 
NOAEL 
No observed adverse effect level 
OTC 
Over the counter 
PBMC 
Peripheral Blood Mononuclear Cell 
PD 
Pharmacodynamic 
PDAI 
Pemphigus Disease Area Index 
PF 
Pemphigus foliaceus 
PK 
Pharmacokinetic 
PO 
By Mouth 
PV 
Pemphigus vulgaris 
Q12H 
Every 12 hours 
QD 
Once a day 
QTc 
QT interval corrected for heart rate 
RA 
Rheumatoid arthritis 
RBC 
Red blood cell 
RR 
Resting Rate 
SAD 
Single ascending dose 
SAE 
Serious adverse event 
SBP 
Systolic blood pressure 
SD 
Standard Deviation 
SI 
Système international d’unités (International system of units) 
SMC 
Safety Monitoring Committee 
SoAT 
Schedule of Assessment Table 
SLE 
Systemic Lupus Erythematosus 
SNAQ 
Simplified Nutritional Appetite Questionnaire 
SSR 
Six-Month SUSAR Report 
SUSAR 
Suspected Unexpected Serious Adverse Reaction 
TABQOL 
Treatment of Autoimmune Bullous Diseases Quality of Life (assessment) 
TB 
Tuberculosis 
TEAE 
Treatment-Emergent Adverse Event 
TGA 
Therapeutic Goods Administration 
Tmax 
Time of observed maximum plasma concentration 
TSH 
Thyroid stimulating hormone 
t½ 
Elimination half-life 
WBC 
White blood cell 
WHODD 
World Health Organisation Drug Dictionary 
XLA 
X-linked agammaglobulinemia 


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1 
BACKGROUND AND SCIENTIFIC RATIONALE FOR THE TREATMENT OF 
PEMPHIGUS WITH PRN1008 
1.1 
Background 
Bruton’s agammaglobulinemia tyrosine kinase (BTK) is an essential signaling element 
downstream of the B-cell receptor (BCR), Fc-gamma receptor (FcγR) and Fc-epsilon receptor 
(FcεR). BTK is a non-receptor tyrosine kinase and is a member of the TEC family of kinases. 
Inhibition of BTK activity in cells produces phenotypic changes consistent with blockade of the 
BCR. This activity results in the down-regulation of various B-cell activities including cell 
proliferation, differentiation, maturation, survival, and the up-regulation of apoptosis. BTK also 
regulates the activation of other hematopoietic cells, such as basophils, mast cells, macrophages, 
neutrophils, and platelets. It is not expressed in T cells, natural killer cells, and plasma cells 
(Sideras and Smith 1995; Mohamed, et al., 2009). A selective BTK inhibitor has the potential to 
target multiple pathways involved in inflammation and autoimmunity. These include modulation 
of BCR-mediated B-cell pathways, inhibition of FcγR-induced cytokine release from monocytes 
and macrophages, FcεR-induced mast cell degranulation, granulocyte migration and mediator 
release. These effects lead to the prediction that a BTK inhibitor could block the initiation and 
propagation of various inflammatory diseases and mitigate the resulting tissue damage. 
Important insights into BTK function come from loss of function analyses in humans and mice. 
Individuals with loss of function mutations in the BTK gene develop X-linked 
agammaglobulinemia (XLA) characterized by a complete absence of circulating B cells and 
plasma cells, and very low levels of immunoglobulins of all classes (Tsukada 1993, 
Vetrie 1993). This indicates the potential for BTK inhibition to suppress production of 
autoantibodies thought to be important in development of autoimmune diseases, such as 
pemphigus vulgaris (PV). These individuals have decreased humoral immunity and are 
susceptible to pyogenic bacterial as well as enterovirus infections, requiring treatment with 
intravenous immunoglobulin. However, inhibition of BTK in individuals with an intact immune 
system is not predicted to produce similar susceptibility to infection. 
Several orally administered BTK inhibitors, including ibrutinib (PCI-32765) and CC-292, are 
currently marketed or in clinical development for a range of indications. Ibrutinib has provided 
further clinical validation of the BTK target and was recently approved for human use in mantle 
cell lymphoma, Waldenström’s macroglobulinaemia, and chronic lymphocytic leukemia by the 
U.S. Food and Drug Administration (U.S. FDA), and has also demonstrated activity in other 
hematological malignancies (Wang 2013, Byrd 2013, Imbruvica Package Insert, 2015). CC-292 
has been reported to be well tolerated in a healthy volunteer population, at doses which provide 
100% occupancy of the BTK enzyme (Evans 2013). 
1.2 
Rationale for PRN1008 Treatment in Pemphigus 
PV is known to be driven by autoantibodies to epidermal proteins and is responsive acutely to 
the anti-inflammatory effects of corticosteroids and within 5 to 35 weeks to B- cell depletion by 
anti-CD20 therapy (Horvath et al. 2012). 


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PRN1008 is a novel, highly selective, small molecule inhibitor of non-T cell white blood cell 
signaling (via B- cell receptor, FCɣR, FcεR signaling of the Bruton’s tyrosine kinase, i.e., BTK, 
pathway).  
In Phase 1 studies with 114 healthy volunteers, target BTK occupancy levels were safely and 
consistently exceeded, suggesting PRN1008 at the planned dose levels may be highly effective in 
human pemphigus and other autoimmune diseases. 
As of January 18, 2018, PRN1008 had been administered to 21 patients with pemphigus 
(pemphigus vulgaris [PV] and pemphigus foliaceus [PF]), 18 of whom had 4 or more weeks of 
treatment with PRN1008. Of the 18 patients with efficacy data, 11 (61%) met the primary 
endpoint of “Control of Disease Activity” (CDA) on a CS dose of ≤ 0.5 mg/kg/day (low-dose 
CS) by the Week 5 visit. Three patients achieved CDA on no CS. Two patients required high-
dose CS temporarily during PRN1008 treatment due to worsening of disease activity. Four 
patients achieved complete remission (CR) on 1 to 20 mg/day of CS; 3 achieved CR at Week 13 
(25%) and one achieved CR at Week 21. PRN1008 has been well-tolerated in this study 
(see Section 1.4). 
Refer to PRN1008 Investigators’ Brochure for additional information. 
1.3 
Summary of PRN1008 Animal Toxicity Studies 
1.3.1 
13-week GLP Toxicology 
In the 13-week repeat-dose GLP toxicity studies with PRN1008, doses of up to 500 mg/kg/day 
and 300 mg/kg/day were given to rats and dogs, respectively. The No Observed Adverse Effect 
Level (NOAEL) in rats was considered to be 150 mg/kg/day and the NOAEL for male dogs was 
30 mg/kg/day. The NOAEL in female dogs is less than 30 mg/kg/day, based on body weight loss 
in one female administered 30 mg/kg/day. At doses above 150 mg/kg, the lungs of some rats 
showed increased macrophages and bronchoalveolar papillary hyperplasia. In dogs, PRN1008-
related findings considered potentially PRN1008-related were observed in the liver and 
lymphoid tissues. At doses above 30 mg/kg in dogs, changes in liver function (mainly ALT and 
AST), loss of appetite, and associated weight loss were observed. These observations are 
considered clinically significant and their assessment in human subjects is included in the study 
procedures in this clinical protocol. 
Orally administered PRN1008 did not affect the gross behavioral, physiological, or neurological 
state of rats at dose levels up to 500 mg/kg, and the NOAEL for effects on the cardiovascular and 
respiratory systems in dogs was considered to be 500 mg/kg. 
PRN1008 tested negative in a battery of in vitro and in vivo genotoxicity studies. 


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1.3.2 
6 and 9-month GLP Toxicology 
In the 6-month rat oral GLP toxicology study, the no-observed-adverse-related level (NOAEL) 
was 50 mg/kg/day in females (AUClast 5480 hr.ng/mL) and 150 mg/kg/day in males (AUClast 
5327 hr.ng/mL). The nature and severity of observed findings included lethality, decreased body 
weight gain, macroscopic and microscopic changes in the gastrointestinal tract, and neutrophilic 
inflammation in the brain. Adequate safety factors of ~4X are present to the rat NOAEL, 
assuming a maximum exposure in humans of 1360 ng.h/mL with a 600 mg bid dose, and 6X to 
the target exposure of 890 ng.h/mL with the 400 mg bid dose. 
In the 9-month dog oral GLP toxicology study, the NOAEL for male and female animals was 
30 mg/kg/day (AUClast 963 hr.ng/mL), based on body weight loss that required food 
supplementation and the euthanasia of one male dog dosed at 50mg/kg/day. The observed 
findings in the dog of appetite decrease, weight loss, and reversible changes in liver function are 
readily monitorable in human studies. 
1.3.3 
Reproductive and Fertility Studies 
1.3.3.1 
Embryo-Fetal Development Study in Rat  
There were no PRN1008-related external, visceral, or skeletal fetal malformations or variations, 
and there were no PRN1008-related effects on fetal ossification site averages. 
Pregnant female rats were given daily oral doses of 50, 150, or 300 mg/kg/day of PRN1008 from 
DG 7 through 17. The maternal NOAEL was determined to be 150 mg/kg/day based upon lower 
body weight/gain and food consumption. There were no PRN1008-related fetal development; or 
fetal external, visceral, or skeletal morphology changes. Therefore, the embryo/fetal 
development NOAEL was considered to be 300 mg/kg/day.  
1.3.3.2 
Embryo-Fetal Development Study in Rabbit  
There were no PRN1008-related external, visceral, or skeletal fetal malformations or variations, 
and there were no PRN1008-related effects on fetal ossification site averages. 
Pregnant female rabbits were given daily oral doses of 10, 30, or 100 mg/kg/day of PRN1008 
from DG 7 through 19. The maternal NOAEL was determined to be 30 mg/kg/day based upon 
lower body weight gain and food consumption at 100 mg/kg/day. There were no PRN1008-
related fetal development changes, and no fetal external, visceral, or skeletal morphology 
changes were observed. Therefore, the embryo/fetal development NOAEL was considered to be 
100 mg/kg/day. 
1.3.3.2.1 
Fertility and Early Embryonic Development in Rat  
Administration of PRN1008 in male rats resulted in an increased incidence of rales and reduced 
body weight, body weight gains, and food consumption. The NOAEL in males for general 
toxicity parameters was considered to be 150 mg/kg/day. There were no effects on mating, 


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fertility, reproductive organ weights, or sperm parameters in the treated males. The reproductive 
NOAEL in males was 300 mg/kg/day. 
Administration of PRN1008 in female rats resulted in reductions in body weights, body weight 
gains, and food consumption during the premating period and reductions in food consumption 
during the gestation period. The NOAEL for the females for maternal toxicity was considered to 
be 150 mg/kg/day. There were no effects on estrous cycling, mating, fertility, or ovarian and 
uterine parameters in the females. The reproductive and development NOAEL in females was 
300 mg/kg/day. 
Refer to PRN1008 Investigators’ Brochure, Section 4.4 and 6 5 for a more comprehensive 
preclinical safety summary. 
1.4 
Summary of PRN1008 Clinical Experience 
To date PRN1008 has been administered to 114 healthy adult volunteer subjects in five Phase I 
studies (PRN1008-001, PRN1008-002, PRN1008-004, PRN1008-006 and PRN1008-008) using 
four different formulations. Single doses, repeat single doses and periods of up to 11 days of 
once or twice daily dosing were studied. The maximum single dose administered was 1200 mg 
and the maximum multiday dose was 450 mg twice daily for 10 days (450 mg of the liquid 
formulation is equivalent to 648 mg of the IR tablet formulation). 
As of January 18, 2018, PRN1008 has been administered to 21 patients with pemphigus using 
the IR tablet formulation. Study PRN1008-005, is a Phase 2, open-label cohort study of 12 weeks 
of PRN1008 therapy, followed by 12 weeks of follow up, using a starting dose of 400 mg twice a 
day, with intra-patient dose-escalation based on clinical response and BTK occupancy up to a 
maximum dose of 600 mg twice a day (Part A of this protocol).  
This amendment 5.0, (Part B) allows 24 weeks of treatment followed by 4 weeks of follow-up. 
Initial dosing in Part B will be 400 mg once daily (qd), with intra-patient dose escalation to 
400 mg bid allowed at or after the Week 3 visit for insufficient clinical response (and then again 
to 600 mg bid if necessary at or after the Week 5 visit). 
A Phase 1/2 study of relapsing immune thrombocytopenia is enrolling patients at dose levels 
between 200 mg daily and 400 mg bid. 
1.4.1 
Experience in Healthy Volunteer Phase 1 Studies 
In Study PRN1008-001, volunteers received single oral doses of placebo (n = 6), 50 mg (n = 6), 
150 mg (n = 6), 300 mg (n = 6), 600 mg (n = 6), or 1200 mg (n = 6). The longest duration of 
treatment to date was in this same study, where subjects received 10 or 11 days of dosing, at dose 
regimens of placebo, 300 mg qd, 300 mg q12h, 600 mg qd, and 450 mg q12h (n = 8 per dose 
level). In Study PRN1008-002, a midazolam drug-drug interaction study, 12 subjects received 
single doses of 600 mg PRN1008. In both Study PRN1008-001 and Study PRN1008-002, 
PRN1008 was administered as a liquid formulation, under fasted conditions on all but one day. 
Study PRN1008-004 compared the relative bioavailability of the liquid formulation to a capsule 
formulation, whereby subjects (n = 12) received 300 mg single doses of both PRN1008 


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formulations. Study PRN1008-006 compared the relative bioavailability of the liquid 
formulation to a tablet formulation, whereby subjects (n = 12) received 300 mg single doses of 
both PRN1008 formulations, as well as the tablet after food and after several days of 
esomeprazole treatment. For additional details, refer to the PRN1008 Investigators’ Brochure.  
PRN1008 was safe and well tolerated at the dose levels tested). In these 114 Healthy Volunteer 
subjects, no severe AEs (AEs) or SAEs were reported, and no subjects required discontinuation 
or interruption of dosing. Reported AEs were mild or moderate in severity, with gastrointestinal 
AEs being most common (nausea/vomiting, loose stools) – predominantly with the liquid 
formulation. These AEs were more frequent at the 600 mg qd and 450 mg q12h doses. No 
clinically significant changes in laboratory parameters, vital signs, or ECG parameters were 
observed in 112/114 healthy volunteer subjects tested.  
In Study PRN1008-008 of single doses of 400 mg of tablet formulations, one healthy volunteer 
with a low-normal baseline neutrophil count of 2100/μL experienced a single laboratory reading 
of 800/μL during follow up five days after a series of 4 single doses, having had a single low 
count of 1200/μL four days earlier after the fourth dose. The single low count had returned to 
1900/μL when first remeasured four days later. In Study PRN1008-006, one subject had 
reversible, Grade 2 elevations of ALT during follow-up, associated with Grade 1 elevation of 
AST. 
Following single and multiple oral doses of PRN1008 in Study PRN1008-001, mean BTK 
occupancy in PBMCs of more than 70% was observed 4 hours or later following doses of 
300 mg or more on Day 1. Figure 1 shows clinically effective levels of BTK occupancy with a 
300 mg bid dose on Day 1, with slightly reduced interpatient variability, and slightly higher 
Hour-4 values, observed after multiple days of dosing. Three subjects with BTK occupancies at 
12 hours on Day 1 in the 40% range all had 12-hour occupancies above 60% by Day 10. 
Figure 1 
Individual and Mean BTK Occupancy Data from HV Study PRN1008-001 
with the Liquid Formulation of PRN1008* 
 
 
*Tablet equivalent doses are 428 mg twice daily after correction for relative bioavailability of ~70%.  
A second dose was not administered on Day 10. 
300mg 
bid
74±4
91±2
77±7
60±8


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1.4.2 
Experience in Pemphigus Patients in Study PRN1008-005 
1.4.2.1 
Efficacy 
As of January 18, 2018, PRN1008 had been administered to 21 patients with pemphigus 
(pemphigus vulgaris [PV] and pemphigus foliaceus [PF]), 18 of whom had 4 or more weeks of 
treatment. Of the 18 patients with efficacy data, 11 (61%) met the primary endpoint of “Control 
of Disease Activity” (CDA) on a CS dose of ≤ 0.5 mg/kg/day (low-dose CS) by the Week 5 visit. 
Three patients achieved CDA on no CS (Figure 2). Two patients required high-dose CS 
temporarily during PRN1008 treatment due to worsening of disease activity. Four patients 
achieved complete remission (CR) on 1 to 20 mg/day of CS; 3 achieved CR at Week 13 (25%) 
and one achieved CR at Week 21. Responses were seen in patients with both mild and moderate 
levels of initial disease (PDAI patient profiles, Figure 3). 
Figure 2 
PDAI and Clinical Response Patient Profiles of 5 Patients on Zero or Doses 
of CS ≤ 5 mg 
 
 
0
5
10
15
20
25
30
35
40
45
50
0
50
100
150
200
Days 
PDAI
total
activity
score
PRN1008 treatment
Post treatment follow up
First control of disease activity (CDA) no later CR
First control of disease activity (CDA) with later CR
First complete (clinical) remission (CR)
5mg
2.5mg
0mg
0mg
1mg
1mg
0mg


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Figure 3 
Timing of Individual Clinical Responses (Day 15 to 29 circled) N=21 
 
 
1.4.2.2 
Safety 
The following three serious adverse events (SAEs) have been reported in PV patients: a Grade 3 
event of cellulitis that led to hospitalization (considered by the investigator to be related to 
PRN1008), and two events considered unrelated to PRN1008 of 1) a Grade 3 event of 
“pancreatic pseudocyst – benign” that led to hospitalization and 2) a Grade 5 event of “acute 
respiratory failure - inflammation of an undiagnosed pulmonary sequestration-congenital” that 
led to hospitalization after one week of PRN1008 therapy and led to death following lung 
surgery more than a month after PRN1008 therapy was ceased. 
Table 1 shows treatment-emergent adverse events (TEAEs) reported by more than one patient by 
relatedness and severity. TEAEs reported by more than one patient deemed related to PRN1008 
are Grade 1 headache (10%), Grade 1 upper respiratory infection (10%) and Grade 1/2 dry 
mouth (10%). 
Two patients on chronic anticoagulation (warfarin and rivaroxaban) have been treated without 
bleeding complications. 
0
5
10
15
20
25
30
35
40
45
50
1
29
57
85
113
141
169
Days 
PDAI
total
activity
score
PRN1008 treatment
Post treatment follow up


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Table 1 
TEAEs Reported by More than One Patient (N=21) 
TEAE Term 
Related 
Grade 
Freq. (n, %) 
Pemphigus worsening 
No 
1-2 
4 (19%)* 
Headachea 
Yes/No 
1 
3 (14%)* 
Nausea 
Yes/No 
1 
2 (10%)* 
Upper respiratory tract infection 
Yes 
2 
2 (10%) 
Vomiting 
No 
1 
2 (10%) 
Dry mouth 
Yes 
1-2 
2 (10%)* 
TEAE = treatment-emergent adverse event 
a Two of three headaches were deemed related to PRN1008 therapy. 
*1 patient was at 500 mg bid, all other patients were at 400 mg bid 
 
Refer to PRN1008 Investigators’ Brochure for additional information. 
1.5 
Clinical Pharmacology of PRN1008 
1.5.1 
Pharmacokinetics in Healthy Volunteers 
With multiple dosing, the terminal half-life of PRN1008 is approximately 3 to 4 hours and the 
time of maximum concentration (Tmax) at therapeutic multiple doses of 1.5 to 2.0 hours. With 
300 mg bid of the liquid formulation, the accumulation ratio was 1.8 after 10 days of dosing. 
There is no effect of food on the AUC or Cmax of the tablet formulation although the Tmax is 
slightly delayed from 1.5 hours to approximately 2.5 hours. The volume of distribution is high 
(on the order of 2500 L) and plasma protein binding is 98% (PRN1008 Investigators’ Brochure). 
1.5.2 
Pharmacokinetics and BTK Occupancy in Pemphigus Patients 
In this Study PRN1008-005, PK sampling is being conducted in all patients for population PK 
analysis. Blood samples were drawn pre-dose and at 2 hours following PRN1008 administration 
on the first day of therapy, pre-dose on the second and seventh day of therapy, and then at 
random times following dosing at other study visits. Preliminary data suggest that plasma 
concentrations at 2 hours, steady state 2 hours, and pre-dose were generally consistent to those 
seen in Phase 1 studies, as corrected for formulation (Figure 4). 


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Figure 4 
Distribution of Plasma Concentrations at BTK Occupancy Timepoints 
 
BTK = Bruton’s tyrosine kinase; SS = steady state 
 
1.5.3 
BTK Occupancy in Patients with Pemphigus 
Target occupancies, based on efficacy in animals and the levels reached with a liquid 
formulation dose of 300 mg q12h, were approximately 80% on Day 1, approximately 70% on 
Day 2 pre-dose (indicated as SS 12 hr), and approximately 90% at SS 2 hr (Figure 5). Thus, 
target occupancy levels are being reached in nearly all individuals with a 400 mg twice daily 
dose. 
1 s t D a y 2 h r
S S  2  h r
S S  1 2  h o u r 
1
1 0
1 0 0
1 0 0 0
plasma conc (ng/ml)
15 1.1
19 6.3
9.4 7


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Figure 5 
BTK Occupancies (Mean and Standard Deviation) Based on Approximate 
Timepoints Following Dosing 
 
BTK = Bruton’s tyrosine kinase; SS = steady state 
 
1 s t D a y   2  h r  
S S  2  h r  
S S  1 2  h r  
0
5 0
1 0 0
% Occupancy
8 5 .5 3
86 .5 8
88 .1 3


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2 
RATIONALE AND JUSTIFICATION FOR THE STUDY AND THE STUDY 
DESIGN 
2.1 
Rationale for the Study Design 
This open-label, pilot study is designed to explore the initial efficacy and safety of PRN1008 in 
PV and to enable the accurate design of pivotal randomized clinical trials which are to follow. 
An open-label design was chosen for several reasons: PV does not spontaneously remit so there 
is no placebo effect to account for; clinicians need to know when to use rescue corticosteroids, if 
necessary, in a timely manner; and lastly, the pilot nature of the investigation itself is best 
explored in an open-label fashion. 
Because of the ability to guide individualized dosing with BTK occupancy measurement, the use 
of which has been validated in a naturally occurring canine pemphigus study, this assay was used 
to supplement the clinical response assessments in making dose-adjustments in Part A. More 
than 95% of patients in Part A (20/21) had adequate BTK occupancies (mean 86% pre dose at 
steady-state). In Part B, the clinical efficacy of the 400 mg qd dose is being explored to see if 
lower BTK occupancies will still equate to clinical efficacy. For this reason, clinical response 
alone, and not BTK occupancies, is recommended to guide dose adjustment in Part B. 
Given the efficacy seen in this pemphigus study to date at varied clinical severities, and the 
updated, supporting, chronic, GLP toxicology (Section 1.3), there is clinical equipoise to study 
PRN1008 monotherapy (or with low dose CS) in patients with mild-to-severe (baseline PDAI 
8-60), corticosteroid-naïve or relapsing PV, with close observation and appropriate corticosteroid 
rescue protocols. 
2.2 
Selection of the Starting Clinical Dose of PRN1008 in Pemphigus 
400 mg bid (Part A): The 400 mg bid starting dose is based upon the dose known to produce 
~70% BTK occupancy at trough (~85% average occupancy over the day), as adjusted by results 
of the relative bioavailability study, where the tablet had ~70% of the exposure of the equal dose 
of the liquid formulation. Adequate BTK occupancies with 400 mg bid dosing of the IR tablet 
has been confirmed in nearly all of the 21 patients with pemphigus studied to date. To confirm 
achievement of target, BTK occupancy measurements after the first dose will be expeditiously 
processed and provided to the treating physician in time for a follow-up visit at Day 15 (Part A 
only). This dose level has adequate safety factors to exposures in chronic toxicology studies 
(Section 1.3). 
400 mg qd (Part B): In some but not all animal studies, a dose-response relationship between 
pre-dose BTK occupancy and clinical efficacy was observed. As it is unknown whether a once 
daily PRN1008 dose will provide adequate pharmacodynamics effect, a 400 mg qd dose is being 
tested in Part B, with the option to expeditiously dose-escalate to higher doses at or after the 
Week 3 visit. This dose level has adequate safety factors to exposures in chronic toxicology 
studies (Section 1.3). 
Please refer to Investigator Brochure, Section 5.5.3, for additional information.  


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2.3 
Overall Risk/Benefit Assessment 
Design summary: Study PRN1008-005, is a Phase 2, open-label, cohort study of 12 weeks of 
PRN1008 therapy, followed by 12 weeks of follow up, using a starting dose in Part A of 400 mg 
twice a day (bid), with intra-patient dose-escalation to a maximum dose of 600 mg twice a day. 
In Part B the starting dose is 400 mg once daily (qd) with intra-patient dose-escalation to 
400 mg bid allowed at or after the week 3 visit based upon insuffcient clinical response (and then 
again to 600 mg bid if necessary at or after the Week 5 visit). 
Safety Profile: The one patient with a related SAE of cellulitis and fever observed in a patient 
with a higher risk of infection underscores the likely, mechanism-based risk of infection during 
chronic treatment with a BTK inhibitor. 
Based on data from 21 patients in this Study PRN1008-005, as of January 18, 2018, PRN1008 
has been well tolerated using the IR tablet formulation in doses of up to 400 mg bid (n=19), 
500 mg bid (n=1) and 600 mg bid (n=1). No patients have discontinued PRN1008 therapy or 
dose-reduced due to treatment-related adverse events. 
Efficacy: High clinical response rates have been observed in the first 21 PV patients in this 
study, on a background of zero or low dose CS, using a starting dose of 400 mg bid. It is possible 
that a lower, once daily dose may have a greater benefit-risk relationship, and this is being 
explored in Part B. 
Risk/Benefit: Given the encouraging safety and efficacy profile of PRN1008, further exploration 
of minimally effective dose levels and longer durations of therapy is justified in pemphigus 
patients. The results of this study will be used to guide PRN1008 use in future patient studies. 
No risks other than those previously mentioned in the Investigator’s Brochure or in the summary 
of PRN1008 clinical experience described above are known at this time. Refer to PRN1008 
Investigators’ Brochure, Section 5.6.2, for additional information. 
2.4 
Drug-Drug Interaction Potential 
Based on in vitro data, CYP3A4 appears to be the major CYP isoform responsible for PRN1008 
metabolism, including first-pass metabolism through the intestinal wall and liver. Therefore, 
PRN1008 plasma exposures have the potential to be altered if co-administered with other agents 
which either inhibit or induce CYP3A, however this potential interaction has not been studied 
clinically to date. Concomitant use of strong and moderate inducers of CYP3A should be 
avoided until their effects on PRN1008 exposure have been studied (Appendix 2). 
A clinical drug-drug interaction study (PRN1008-002) was conducted to evaluate the effects of 
the liquid formulation of PRN1008 when administered simultaneously with midazolam, a probe 
CYP3A substrate. When co-administered with midazolam, midazolam plasma exposures 
increased approximately 3-fold. PRN1008 is therefore classified as a moderate inhibitor of 
CYP3A. As a result, sensitive CYP3A substrate drugs, particularly those with a narrow 
therapeutic index, should be avoided during treatment with PRN1008 (Appendix 2). Additional 
details are provided in the Investigator Brochure. PRN1008 does not appear to be an inducer of 
CYP isoenzymes. 


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Acid reducing drugs may reduce the bioavailability of PRN1008 tablets. Therefore, PRN1008 
should be administered 2 hours or more prior to such drugs. Proton pump inhibitors are not 
permitted during the study (they resulted in a 50% decrease in PRN1008 tablet exposure when 
administered concomitantly in Study PRN1008-006). 


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3 
OBJECTIVES OF THE STUDY 
3.1 
Primary Objectives 
• To evaluate the clinical safety of PRN1008 in patients with PV over a 12-week (Part A) or 
24-week (Part B) treatment period 
• To evaluate the clinical activity of PRN1008 in patients with PV, per criteria in the European 
Academy of Dermatology and Venereology (EADV) 2014 Pemphigus S2 Guideline 
(Hertl et al. 2015)* 
*As modified to define CR without 2-month durability definition 
3.2 
Secondary Objective 
• To evaluate the pharmacokinetics (PK) and the pharmacodynamics (PD) of PRN1008 in 
patients with PV 
3.3 
Exploratory Objective 
• To evaluate the relationship of PK and PD to each other and to efficacy and safety in this 
patient population 


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4 
INVESTIGATIONAL PLAN 
4.1 
Overall Study Design and Plan 
This is a Phase 2, open-label, pilot cohort study. 
Part A: Initial PRN1008 dosing will be 400 mg (bid), with intrapatient dose adjustment up to 
600 mg (bid) based on BTK occupancy and clinical response, and corticosteroid rescue 
treatment, if indicated. Patients requiring corticosteroid rescue treatment will be treated 
according to criteria specified in Appendix 7 of the protocol.  
Part B: Initial PRN1008 dosing will be 400 mg (qd), with intra-patient dose escalation to 
400 mg bid allowed at or after Week 3 visit for insufficient clinical response (and then again to 
600 mg bid if necessary at or after Week 5 visit). 
4.2 
Number of Participants 
This study is planned to enroll up to 50 PV patients, with an interim analysis completed when 
6 subjects completed 4 weeks or more of therapy (Study PRN1008-005 Interim Analysis 
Report). 
4.3 
Study Duration and Duration of Subject Participation 
Part A: Subjects will receive twice-daily PRN1008 treatment for 12 weeks, starting on Day 1 
and ending on study Day 85, with a further 12 weeks of follow up (total duration of individual 
subject participation is approximately 28 weeks). 
Part B: Patients will receive once or twice-daily PRN1008 for 24 weeks, starting on Day 1 and 
ending on study Day 169, with a follow up visit 4 weeks later (a total duration of individual 
subject participation is approximately 32 weeks). 
Patients enrolled in a previous version of this protocol who are still in their 12-week 
active treatment period with PRN1008 are eligible to continue treatment, initially with 
their current dose level, under this amended protocol for an additional 12 weeks, i.e. 
24 weeks total, following review and signature of the EC approved patient’s consent see 
Section 6.1 Inclusion Criteria. 
The expected study duration is approximately 48 months from the first subject treated to the last 
subject completing Part B in the study.  
4.4 
End of Study Definition 
The end of the study is defined as the date of the final safety follow-up after the LPLV. 


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4.5 
PRN1008 Administration 
Part A: Initial PRN1008 dosing will be 400 mg bid. The maximum dose in this study, after dose 
adjustment, will be 600 mg bid. Clinical response and tolerability will be assessed at each visit. 
Dosing may be adjusted according to Section 4.6. 
Part B: Initial PRN1008 dosing will be 400 mg qd, unless patients previously initiated the study 
with a 12-week treatment period are on higher doses, in which case they will initially continue 
with their current dose level. The maximum dose in this study, after dose adjustment will be 
600 mg bid. Clinical response and tolerability will be assessed at each visit. Dosing may be 
adjusted according to Section 4.6. 
PRN1008 tablets should be taken with a glass of water and may be taken with or without food, 
i.e., a period of fasting is not required. 
4.5.1 
Rationale for PRN1008 Dosage Selection 
400 mg bid (Part A): The 400 mg bid starting dose is based upon the dose known to produce 
~70% BTK occupancy at trough (~85% average occupancy over the day), as adjusted by results 
of the relative bioavailability study, where the tablet had ~70% of the exposure of the equal dose 
of the liquid formulation. Adequate BTK occupancies with 400 mg bid dosing of the IR tablet 
has been confirmed in nearly all of the 21 patients with pemphigus studied to date. To confirm 
achievement of target, BTK occupancy measurements after the first dose will be expeditiously 
processed and provided to the treating physician in time for a follow-up visit at Day 15 (Part A 
only). This dose level has adequate safety factors to exposures in chronic toxicology studies 
(Section 1.3). 
400 mg qd (Part B): In some but not all animal studies, a dose-response relationship between 
pre-dose BTK occupancy and clinical efficacy was observed. As it is unknown whether a once 
daily PRN1008 dose will provide adequate pharmacodynamic effect, a 400 mg qd dose is being 
tested in Part B, with the option to expeditiously dose-escalate to higher doses at or after the 
Week 3 visit. This dose level has adequate safety factors to exposures in chronic toxicology 
studies (Section 1.3). 
Maximum dose of 600 mg bid: A dose level 50% higher than the target upper dose level of 
400 mg bid was arbitrarily chosen based on previous clinical safety data in healthy volunteers at 
higher exposures and adequate safety factors to exposure in animal toxicology studies. 
4.5.2 
Concomitant Medications 
• Other immunosuppressive medications are not permitted except for low-dose corticosteroids 
and when rescue immunotherapy is triggered. 
• Inducers and inhibitors of Cytochrome P450 3A (CYP3A) should be avoided as they may 
reduce or increase the exposure of PRN1008 (Appendix 2) when administered 
concomitantly. 


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• PRN1008 is a moderate CYP3A inhibitor and can increase the exposure of drugs 
metabolized by CYP3A when administered concomitantly. Narrow therapeutic index CYP3A 
substrate drugs should be avoided (Appendix 3) and care should be taken with other drugs 
metabolized by the CYP3A. 
• Clinically relevant 3A substrate drugs (e.g. “sensitive substrate” listed in Appendix 3) should 
be managed by administering PRN1008 on a time schedule such that CYP3A substrate drugs 
can be given 2 hours or more after PRN1008. 
• Acid reducing drugs may reduce the bioavailability of PRN1008 tablets. Therefore, 
PRN1008 should be administered 2 hours or more prior to such drugs. Proton pump 
inhibitors are not permitted during the study. 
4.6 
PRN1008 Intrapatient Dose Adjustment 
Part A: Initial PRN1008 dosing will be 400 mg bid, with intrapatient dose adjustment based on 
clinical response and tolerability, supplemented by BTK occupancy (Table 2).  
Part B: Initial PRN1008 dosing will be 400 mg qd, with intra-patient dose escalation to 400 mg 
bid allowed at or after Week 3 visit for insufficient clinical response (see Table 3) (and then 
again to 600 mg bid if necessary at or after Week 5 visit. Corticosteroid rescue treatment will be 
initiated, if indicated (Appendix 1). 
“Well tolerated” is defined as the absence of Grade 3 or greater gastrointestinal AEs, or Grade 2 
non-gastrointestinal AEs, including liver function changes, related to PRN1008 therapy. 
Investigators should use their judgment of the risk-benefit of continuing therapy by weighing the 
extent of clinical response, and AEs potentially related to PRN1008 therapy. In general, 
PRN1008 should be stopped and conventional therapy started when tolerability is poor and 
clinical response is also suboptimal. 


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Table 2 (Part A) 
General Dose Adjustment Guidelines for Dose Selection in the First 
4 Weeks  
Clinical 
Response 
Trough BTK 
Occupancy 
Tolerability* 
Action 
Responder 
400 mg bid 
≥ 50% 
Well Tolerated 
Maintain 400 mg bid 
Taper corticosteroids if used in combination 
Poorly Tolerated  
Reduce to 300 mg bid 
Taper corticosteroids if used in combination 
< 50% 
Well Tolerated 
Maintain 400 mg bid 
Taper corticosteroids if used in combination 
Poorly Tolerated  
Reduce to 300 mg bid 
Taper corticosteroids if used in combination 
Suboptimal 
Response  
400 mg bid 
≥ 50% 
Well Tolerated 
Follow rescue criteria if triggered, if not 
maintain dose at 400 mg bid 
Poorly Tolerated  
Follow rescue criteria if triggered, if not 
maintain dose at 400 mg bid if feasible 
< 50% 
Well Tolerated 
Follow rescue criteria if triggered, if not, 
increase dose to 600 mg bid 
Poorly Tolerated  
Follow rescue criteria if triggered, if not, 
increase dose to 600 mg bid if tolerability 
allows.  
* “Well tolerated” is defined as the absence of Grade 3 or greater gastrointestinal AEs, or Grade 2 non-
gastrointestinal AEs, including liver function changes, related to PRN1008 therapy. 
 
Table 3 (Part B) 
General Dose Escalation Guidelines 
Current Dose 
Inadequate Clinical Response* Dose-escalation Rules 
400 mg qd 
Increase to 400 mg bid (allowed at Week 3 visit or later)** 
400 mg bid 
Increase to 600 mg bid (allowed at Week 5 visit or later)** 
600 mg bid 
No dose increase possible. See Appendix 1B for corticosteroid rescue protocol 
* Investigator discretion: Generally, clinical response is shown by some improvement seen in first 2 weeks with 
CDA achieved by the Week 5 visit 
** Unless tolerability issues preclude dose-escalation 


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5 
PROTOCOL DEVIATIONS 
The Principal Investigator is responsible for complying with all protocol requirements, and 
applicable global and local laws regarding protocol deviations. Principia does not allow 
intentional or prospective deviations from the protocol unless necessary to eliminate an 
immediate hazard to study subjects. The Principal Investigator is responsible for immediately 
(within 24 hours) notifying Principia of a major protocol deviation to permit Principia to 
determine the impact of the deviation on the subject and/or the study:  
Primary Principia Contact: 
 
Secondary Principia Contact: 
 
 
The Principal Investigator is responsible for notifying Independent Ethics Committee 
(IEC)/Independent Review Board (IRB) regulatory authorities, and contract research 
organization (CRO) Clinical Monitors of all protocol deviations. 


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6 
STUDY POPULATION 
The study population is male or female patients with newly diagnosed (i.e., naïve to an effective 
induction treatment regimen) or relapsing, biopsy-proven, mild to severe PV (PDAI 8-60), for 
whom an initial period of PRN1008 monotherapy is judged clinically acceptable. 
Because patients without mucosal involvement but with a medical history suggestive of PV are 
allowed into the study, some patients with clinical features suggestive of the pemphigus foliaceus 
(PF) variant of the disease may be enrolled. 
6.1 
Inclusion Criteria (Part A and Part B unless noted below) 
1. 
Male or female patients, aged 18 to 80 years old, with biopsy-proven (positive direct 
immunofluorescence and appearance on H&E microscopy), mild-moderate PV in Part A 
(PDAI 8 to 45) and mild-severe PV in Part B (PDAI 8 to 60) 
2. 
Newly diagnosed or relapsing patients for whom an initial period of PRN1008 monotherapy 
or combination therapy with low-dose corticosteroids (≤0.5 mg/kg of prednis[ol]one or 
equivalent), is judged clinically acceptable, provided tapering of the corticosteroid treatment 
regimen is anticipated with good clinical response to PRN1008 
3. 
BMI > 17.5 and < 40 kg/m2 Part A only  
4. 
Adequate hematologic, hepatic, and renal function (absolute neutrophil count ≥ 1.5 X 109/L, 
Hgb > 9 g/dL, platelet count ≥ 100 X 109/L, AST/ALT ≤ 1.5 x ULN, albumin ≥ 3 g/dL, 
creatinine ≤ ULN (Part A) and creatinine ≤ 1.5 x ULN (Part B) 
5. 
Female patients who are of reproductive potential must agree for the duration of active 
treatment in the study to use an effective means of contraception (hormonal contraception 
methods that inhibits ovulation, intrauterine device, intrauterine hormone-releasing system, 
bilateral tubal ligation, vasectomized partner, condoms or sexual abstinence). Unless 
surgically sterile, postmenopausal females should have menopause confirmed by FSH 
testing. 
6. 
Able to provide written informed consent and agreeable to the schedule of assessments. 
6.2 
Exclusion Criteria 
1. 
Previous use of a BTK inhibitor 
Patients enrolled in a previous version of this protocol who are still in their 12-week 
active treatment period with PRN1008 are eligible to continue treatment, initially with 
their current dose level, under this amended protocol for an additional 12 weeks, i.e. 
24 weeks total, following review and signature of the EC approved patient’s consent. 
Patients who completed Part A and did not discontinue the study due to a medical condition 
that might compromise safety assessments or for a PRN1008 related adverse event may be 
screened for entry under Part B.  
2. 
Pregnant or lactating women 


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3. 
ECG findings of QTc > 450 msec (males) or > 470 msec (females), poorly controlled atrial 
fibrillation (i.e. symptomatic patients or a ventricular rate above 100 beats/min on ECG), or 
other clinically significant abnormalities 
4. 
A history of malignancy of any type, other than surgically excised non-melanoma skin 
cancers or in situ cervical cancer within 5 years before the day of dosing 
5. 
Use of immunologic response modifiers with the following periods prior to Day 1: as 
concomitant therapy, other immunologic response modifiers not detailed in this exclusion 
apart from corticosteroids; 1 week: cyclophosphamide; 4 weeks: IVIG, Kinaret (anakinra) 
and Enbrel (etanercept); 12 weeks: Remicade (infliximab), Humira (adalimumab), Simponi 
(golimumab), Orencia (abatercept), Actemra (tocilizumab), Cimzia (certolizumab), 
Cosentyx (secukinumab), plasmapheresis; 6 months: Rituxan/MabThera (rituximab), 
ofatumumab, any other anti-CD20 antibody, other long-acting biologics 
6. 
More than 0.5 mg/kg of prednis(ol)one per day (“low dose corticosteroids”) within the two 
weeks prior to Day 1 
7. 
Use of proton pump inhibitor drugs such as omeprazole and esomeprazole (it is acceptable 
to change patient to H2 receptor blocking drugs prior to the first dose of PRN1008) 
8. 
Concomitant use of known strong-to-moderate inducers or inhibitors of CYP3A 
(Appendix 2) within 3 days or 5 half-lives (whichever is longer) of study drug dosing 
9. 
Use of CYP3A-sensitive substrate drugs (Appendix 3) with a narrow therapeutic index 
within 3 days or 5 half-lives (whichever is longer) of study drug dosing including, but not 
limited to, alfentanil, astemizole, cisapride, cyclosporine, dihydroergotamine, ergotamine, 
fentanyl, pimozide, quinidine, sirolimus, tacrolimus, or terfenadine 
10. Has received any investigational drug (or is currently using an investigational device) within 
the 30 days before receiving the first dose of study medication, or at least 5 times the 
respective elimination half-life time (whichever is longer) 
11. History of drug abuse within the previous 12 months 
12. Alcoholism or excessive alcohol use, defined as regular consumption of more than 
approximately 3 standard drinks per day 
13. Refractory nausea and vomiting, malabsorption, external biliary shunt, or significant bowel 
resection that would preclude adequate study drug absorption 
14. History of anorexia nervosa or periods of three months or more of low body weight 
(BMI < 17.5) in the past 5 years 
15. Donation of a unit or more of blood or blood products within 4 weeks prior to Day 1 
16. History of solid organ transplant 
17. History of epilepsy or other forms of seizure in the last 5 years 
18. Positive for screening for HIV, hepatitis B (surface and core antibodies unrelated to 
vaccination), or hepatitis C (anti-HCV antibody confirmed with Hep C RNA) 


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19. Positive interferon-gamma release assay (IGRA) (e.g., T-spot TB Test, QuantiFERON®-TB 
Gold, or QuantiFERON®-TB Gold Plus (QFT Plus) at Screening.  Unless, the patient has 
latent TB and all of the following 3 conditions are true: 
a. Chest X-ray does not show evidence suggestive of active tuberculosis (TB) disease 
b. There are no clinical signs and symptoms of pulmonary and/or extra-pulmonary 
TB disease 
c. Documented receipt of one of the following prophylactic treatment regimens: 
i. 
Oral daily Isoniazid for 6 months 
or 
ii. 
Oral daily Rifampin (RIF) for 4 months 
or 
iii. 
Isoniazid and Rifapentine weekly for 3 months (3HP) 
On a case by case basis, after discussion and approval by the Sponsor, a local TB test that is 
negative and is considered equivalent to 1 of the above tests may be used for eligibility. For 
example, if a QuantiFERON®-TB Gold, or QuantiFERON-TB Gold Plus (QFT Plus) is 
positive and a local blood test or T-Spot TB test is negative, the patient may be enrolled 
using the local result upon approval of the Sponsor. 
 
 
20. History of serious infections requiring intravenous therapy with the potential for recurrence 
21. Live vaccine within 28 days prior to baseline or plan to receive one during the study 
22. Any other clinically significant disease, condition, or medical history that, in the opinion of 
the Investigator, would interfere with subject safety, study evaluations, and/or study 
procedures 
6.3 
Prior and Concomitant Therapy 
6.3.1 
Prior Therapy 
Not Permitted: 
Use of immunologic response modifiers within the following periods prior to Day 1:  
• One week for cyclophosphamide 
• Four weeks for Kinaret® (anakinra), intravenous gamma globulin (IVIG), and Enbrel® 
(etanercept) 
• 12 weeks for Remicade® (infliximab), Humira® (adalimumab), Simponi® (golimumab), 
Orencia® (abatercept), Actemra® (tocilizumab), Cimzia® (certolizumab), Cosentyx™ 
(secukinumab), plasmapheresis 


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• 6 months for Rituxan®/MabThera® (rituximab), ofatumumab, any other anti-CD20 
antibody, or any other long-acting biologic 
6.3.2 
Concomitant Therapy 
Not Permitted: 
• Concomitant use of immunosuppressant medication, other than low-dose corticosteroids, 
unless rescue criteria (Appendix 1) are triggered. 
• Concomitant use of known strong to moderate inducers or inhibitors of CYP3A (Appendix 2) 
within 14 days or 5 half-lives (whichever is longer) of dosing with PRN1008. 
• Use of CYP3A-sensitive substrate drugs (Appendix 3) with a narrow therapeutic index 
within 14 days or 5 half-lives (whichever is longer) of PRN1008 dosing including, but not 
limited to, alfentanil, astemizole, cisapride, cyclosporine, dihydroergotamine, ergotamine, 
fentanyl, pimozide, quinidine, sirolimus, tacrolimus, or terfenadine. 
• Proton pump inhibitors are not permitted. Esomeprazole was shown to reduce the exposure 
of PRN1008 by approximately 50%, presumably due to the effects of a lack of an acidic 
environment on tablet dissolution. Therefore, subjects who are on proton pump inhibitors 
should be changed to H2 receptor blocking drugs if possible or not enrolled in the study. 
Permitted: 
• The use of oral prednis(ol)one may be permitted in some circumstances. For admission to the 
study, doses of oral prednis(ol)one in the 2 weeks prior to Day 1 may be no higher than 
0.5 mg/kg per day (inhaled and mucosal [for symptomatic treatment of oral lesions] 
corticosteroids are allowed). Where patients enter the study on low-dose corticosteroids, the 
regimen should be maintained for the initial 2 weeks of PRN1008 therapy. At the Day 15 
review, a good clinical response to PRN1008 should allow the tapering of the corticosteroid 
to commence using the Werth taper (Appendix 7). At all times, the rescue criteria should be 
followed, as indicated (Appendix 1). In some circumstances, corticosteroids should be added 
or the dose increased, with or without cessation of PRN1008. 
• Clinically relevant 3A substrate drugs (e.g., “sensitive substrate” listed in Appendix 3) 
should be managed by administering PRN1008 on a time schedule such that CYP 3A 
substrate drugs can be given 2 hours or more after PRN1008. 
• H2 receptor blocking drugs and antacids are permitted provide PRN1008 is given at least 
2 hours beforehand. 
• Anticoagulation with warfarin should be monitored actively in the first weeks of PRN1008 
therapy, as the combination has not been studied previously. 


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7 
LIST OF SCREENING ASSESSMENTS 
The Schedule of Assessments is presented in Appendix 4. All participants must sign and date the 
most current Institutional Review Board (IRB)-approved written informed consent form before any 
study specific assessments or procedures are performed. An original signed consent form will be 
retained by the Investigator and the participant will be given a copy of the signed consent form. 
Participants must fulfill all entry criteria to be enrolled into the study. Participants who fail to meet 
the entry criteria may be rescreened once at the discretion of the Investigator. A record of 
eligibility screening documenting the Investigator’s assessment of each screened participant with 
regard to the protocol’s inclusion and exclusion criteria, including screen failures, is to be 
completed and signed by the Investigator or designee. Ethnicity of participants will be recorded, 
since this information might be important to evaluate a potential impact of ethnic factors on drug 
properties [see also ICH Guideline E5 (R1)]. 
Up to 28 days before enrollment in the study, participants will be required to sign a consent 
form, after which screening assessments will be carried out. After providing written informed 
consent, subjects will complete the Screening Assessments within 28 days before the first dose of 
PRN1008: 
• Review of medical history and concomitant medication 
• PDAI, ABSIS assessments 
• Review of inclusion and exclusion criteria 
• Measurement of height and weight  
• Physical examination 
• 12-Lead ECG 
• Vital signs (blood pressure, heart rate, respiration rate, and temperature) 
• Clinical laboratory testing (hematology, coagulation, serum chemistry, and urinalysis) 
• HIV, hepatitis B (surface antigen and core antigen and antibodies), hepatitis C (anti-HCV 
antibody confirmed with Hep C RNA)  
• TB screen with T-spot TB Test, QuantiFERON®-TB Gold, or QuantiFERON®-TB Gold 
Plus (QFT Plus) 
• Serum pregnancy test for females of child bearing potential 
• FSH (in postmenopausal women who are not surgically sterile only) 
• Skin biopsy if not already performed: lesional for H&E staining, perilesional for direct 
immunofluorescence 
 


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8 
LIST OF STUDY ASSESSMENTS  
The Schedule of Assessments is presented in Appendix 4. 
8.1 
Clinical Assessments Part A and B 
• Medical history, including evaluation of any on-study AEs and periodic vital signs (body 
temperature, heart rate, respiratory rate, blood pressure), concomitant medication use, full or 
abbreviated physical examination 
• At screening only: height and weight, full physical examination, and 12-lead 
electrocardiogram (ECG) (an extra ECG may be taken at additional visits, if indicated) 
• Disease activity scores (PDAI, Autoimmune Bullous Skin Disorder Intensity Score [ABSIS]) 
(Appendix 5) 
• Standardized photography of the affected area (Optional) 
• Autoimmune Bullous Diseases Quality of Life (ABQOL) and Treatment of Autoimmune 
Bullous Diseases Quality of Life (TABQOL) assessments (Appendix 5) 
• SNAQ appetite questionnaire (Appendix 6) 
8.2 
Laboratory Assessments Part A and B 
Note: Laboratory assessments will be performed at both central and local laboratories, if 
required. 
• Hematology: hemoglobin, hematocrit, erythrocyte count (RBCs), thrombocyte count 
(platelets), leukocyte count (WBCs) with differential in absolute counts (including 
neutrophils, eosinophils, basophils, lymphocytes, and monocytes) 
• Coagulation:  PT/INR, thrombin time, aPTT, fibrinogen level. 
• Serum Chemistry: Aspartate aminotransferase (AST), Alanine aminotransferase (ALT), 
Total, direct, and indirect bilirubin levels, Alkaline phosphatase (ALP), Albumin, Creatinine, 
Urea, Total Protein, Sodium, Chloride, Calcium, Phosphate, Potassium, Glucose (random), 
creatine phosphokinase (CPK) and thyroid stimulating hormone (TSH) 
• PK: Plasma PRN1008 concentration  
• PD: 
- 
BTK occupancy in PBMCs at baseline (pre-dose), and 2 and 24 hours after the first dose, 
then at various times of the day during follow-up visits. For the Day 2 (24 hour) BTK 
occupancy, PRN1008 should be taken as usual in the evening (only if bid dosing) and the 
PRN1008 morning dose withheld in order to take the blood sample approximately 
12 hours after the prior evening dose (for bid dosing) or 24 hours after the last dose 
(for qd dosing). Where follow-up is not feasible on Day 2, another day in the first week 


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of therapy may be used to get trough occupancy instead. For all other visits, occupancy is 
measured at random times after the usual morning dose. 
- 
Anti-desmoglein-1 and -3 autoantibody titers by enzyme-linked immunosorbent assay 
(ELISA) 
• Urinalysis:  pH, specific gravity, protein, glucose, ketones, bilirubin, blood, nitrites, 
urobilinogen and leukocytes measured by dipstick 
• Serology:  HIV, Hepatitis B and Hepatitis C and T-spot TB Test or QuantiFERON® TB 
testing or QuantiFERON®-TB Gold Plus (QFT Plus) at screening only 
• Pregnancy or FSH:  Serum pregnancy test for women of child bearing potential or serum 
FSH for post-menopausal females 
8.3 
Safety and Tolerability Assessments 
Specific assessments to evaluate treatment safety include the following: the frequency and type 
of AEs, clinical laboratory testing, SNAQ appetite questionnaire (Appendix 6) and vital signs. 
Patients will remain under observation in the clinic for 2 hours after administration of the first 
PRN1008 dose and until the PK sample is drawn (see also Section 12.1, Safety Monitoring 
Committee). 


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9 
STUDY TREATMENT 
Part A: Initial PRN1008 dosing will be 400 mg bid. The maximum dose in this study, after dose 
adjustment, will be 600 mg bid. Subjects will be treated with PRN1008 for a maximum of 
12 weeks. 
Patients enrolled in a previous version of this protocol who are still in their 12-week 
active treatment period with PRN1008 are eligible to continue treatment, initially with 
their current dose level, under this amended protocol for an additional 12 weeks, i.e. 
24 weeks total, following review and signature of the EC approved patient’s consent. 
Part B: Initial PRN1008 dosing will be 400 mg qd unless patients are eligible to roll from 
Part A to Part B. The maximum dose in this study, after dose adjustment, will be 600 mg bid. 
Subjects will be treated with PRN1008 for a maximum of 24 weeks. 
PRN1008 tablets should be taken with a glass of water, with or without food, i.e., a period of 
fasting is not required. 


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10 
IDENTITY OF INVESTIGATIONAL PRODUCT 
10.1 
Formulation 
Each PRN1008 film-coated tablet contains either 100 mg or 300 mg of PRN1008 drug 
substance. In addition, the tablet contains Microcrystalline Cellulose (filler), Crospovidone 
(disintegrant), Sodium Stearyl Fumarate (lubricant) and a non-functional film coating. 
The 100 mg tablet is a round shape and orange in color. The 300 mg tablet is an oval shape and 
white in color. 
10.2 
Packaging 
PRN1008 tablets are packaged in white high-density polyethylene (HDPE) bottles with child-
resistant induction-sealed caps. 
10.3 
Storage and Handling 
PRN1008 tablets are supplied in white plastic bottles with child-resistant induction-sealed caps 
or blister packs. The recommended storage condition is 2 to 25°C. Refer to product label for 
specific instructions. 
10.4 
Drug Accountability 
The Investigator or his/her designated representatives will dispense study drug per the Schedule 
of Assessments (Appendix 4). 
The Investigator is responsible for the control of drugs under investigation. Adequate records of 
the receipt (e.g., Drug Receipt Record) and disposition (e.g., Investigational Drug Dispensing 
Log) of the study drug must be maintained. The investigational Drug Dispensing Log must be kept 
current and should contain the following information: 
• The identification of the participant to whom the study drug was dispensed (for example 
subject identification number, participant initials, and date of birth) 
• The date(s), quantity, and lot number(s) of the study drug dispensed to the participant 
• The identification of the person who dispensed the study drug 
All records and drug supplies must be available for inspection by the Study Monitor at every 
monitoring visit. When the study is completed, the Investigator will return any used and unused 
study drug (e.g., empty, partially used, and unused containers), occluded labels (or the equivalent) 
to the Sponsor as requested. The completed Drug Dispensing Log and Drug Return Record(s) will 
be returned to the Local Sponsor. The Investigator's copy of the Drug Return Record(s) must 
accurately document the return of all study drug supplies to the Sponsor. 


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10.5 
Destruction of Investigational Product 
Local or institutional regulations may require immediate destruction of used investigational 
medicinal product for safety reasons. In these cases, it may be acceptable for investigational study 
center staff to destroy dispensed investigational product before a monitoring inspection, provided 
that source document verification is performed on the remaining inventory and reconciled against 
the documentation of quantity shipped, dispensed, returned, and destroyed, and provided that 
adequate storage and integrity of drug has been confirmed. Written authorization must be obtained 
from the Sponsor or Sponsor designee after final accountability prior to destruction. 
Unused study drug from the site that has not been stored properly should not be destroyed until the 
final report has been approved. 
Written documentation of destruction must contain the following: 
• Identity of investigational product(s) destroyed 
• Quantity of investigational product(s) destroyed 
• Date of destruction 
• Method of destruction 
• Name and signature of responsible person who destroyed the investigational products 
Investigational sites that do not have a procedure for destruction and appropriate disposal of 
investigational product, or cannot dispose of investigational product locally may request 
assistance and documentation for return to the Sponsor, or assignment to a local qualified vendor 
for destruction and disposal. 
 


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11 
STATISTICAL METHODS AND DETERMINATION OF SAMPLE SIZE 
11.1 
Primary Outcome Measures 
11.1.1 
Safety  
The incidence of treatment-emergent AEs (TEAEs), including clinically significant changes in 
physical examination, laboratory tests, and vital signs. 
11.1.2 
Efficacy  
The proportion of subjects who are able to achieve control of disease activity (CDA) within 
4 weeks of starting PRN1008 treatment without the need for doses of prednis(ol)one > 0.5 mg/kg.  
11.2 
Secondary Outcome Measures 
The following clinical activity endpoints* will be assessed: 
• Proportion of subjects able to achieve CDA without corticosteroids within 4 weeks 
• Proportion of subjects able to achieve a complete response (CR) without corticosteroids 
within 12 weeks (and 24 weeks in Part B) 
• Proportion of subjects able to achieve CR without the need for doses of prednis(ol)one of 
greater than 0.5mg/kg within 12 weeks (and 24 weeks in Part B) 
• Time to CDA 
• Time to CR 
• Time to end of consolidation phase 
• Time to relapse after PRN1008 treatment discontinuation 
• Cumulative corticosteroid usage over the first 12 weeks (and 24 weeks in Part B) 
• Change from baseline in Pemphigus Disease Area Index (PDAI) and Autoimmune Bullous 
Skin Disorder Intensity Score (ABSIS) scores at each follow-up visit 
• Change from baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL) and 
Treatment of Autoimmune Bullous Diseases Quality of Life (TABQOL) scores at each 
follow-up visit 
• Change from baseline in appetite (SNAQ score) at each follow-up visit 
*Clinical activity endpoints as defined by the EADV 2014 pemphigus S2 guideline (Hertl et al. 2015) with the 
exception that CR is defined as CR at a single point in time rather than present for ≥2 months 
 


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11.3 
PK/PD Outcome Measures 
11.3.1 
PK Outcome Measures 
• Plasma concentrations of PRN1008 at approximately the time of maximum concentration on 
Day 1 and at various subsequent times during outpatient dosing 
11.3.2 
PD Outcome Measures 
• Percentage BTK occupancy for individuals in peripheral blood mononuclear cells (PBMCs) 
at 2 & 24 hours after the first PRN1008 dose and at varied subsequent times during 
outpatient dosing 
• Change from baseline in anti-dsg1-3 autoantibody levels by ELISA at various time points 
11.3.3 
Exploratory PK/PD Analysis 
Exploratory PK/PD analysis will examine the effects, if any, of covariates on PK and/or PD, and 
the relationship between PK, PD, and efficacy in this population. 
11.4 
Determination of Sample Size 
Sample size for this pilot study was determined pragmatically by the number of subjects required 
to determine a preliminary safety profile and a point estimate for the primary endpoint of 
efficacy. If the clinical response rate is 50%, 25 subjects in each Part will result in an 80% CI of 
± 13%. Results from this pilot study will be used to design confirmatory clinical trials. 
11.5 
Analysis Populations 
Four study populations will be defined: Screening Population, Safety Population, Efficacy 
Population, and Pharmacokinetic Population. 
11.5.1 
Screening Population 
All participants who provide informed consent and have screening assessments evaluated for 
study participation are included in the Screening Population. 
11.5.2 
Safety Analysis Population 
All participants who have received at least one dose of PRN1008 will be included in the safety 
analysis. The Safety Analysis Population will be defined for all safety analyses. 
11.5.3 
Efficacy Analysis Population 
All patients who have received at least one dose of PRN1008 will be included in the efficacy 
analysis. Subject response and disease progression will be determined using PDAI, ABSIS, 
ABQOL, and TABQOL scores. Efficacy data will be presented in listings by subject and 
tabulated for each efficacy endpoint. Data may be combined where appropriate for Parts A 


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and B. Additional sub-group analyses (e.g. disease severity, dose group) may be performed as 
appropriate. 
11.5.4 
Pharmacokinetic Analysis Population 
A pharmacokinetic population will be defined for participants who provide adequate plasma 
concentration data to allow for PK analysis. Participants may be excluded from the PK population 
if they significantly violate the inclusion or exclusion criteria, deviate significantly from the 
protocol, or if data are unavailable or incomplete, all of which may influence the analysis. 
Excluded cases will be documented together with the reason for exclusion in the Clinical Study 
Report. 
11.6 
Randomization and Blinding Procedures 
Not applicable; the study is open-label. 
11.7 
Subject Numbers and Treatment Assignments 
As they are enrolled in the study, subjects will be assigned a unique consecutive number. 
The site, in conjunction with the Sponsor, will be responsible for assignment of all unique subject 
numbers and dose assignments. 
11.8 
Subject Disposition, Subject Replacement, and Demographics and Baseline 
Characteristics 
11.8.1 
Disposition 
The numbers of subjects enrolled, completing, and withdrawing, along with reasons for 
withdrawal, will be tabulated. The number of subjects in each analysis population will be 
reported overall. 
11.8.2 
Replacement of Subjects 
Participants prematurely discontinued from the study for non-safety reasons may be replaced at the 
discretion of the Sponsor, to ensure adequate numbers of evaluable participants. A patient is 
considered "evaluable" if they took one or more doses of the study drug. Each patient who 
provides informed consent will be assigned a unique subject identifier (USUBJID). Participants 
recruited to be replacement patients will be assigned the next available subject identifier. 
11.8.3 
Demographics and Baseline Characteristics 
Demographic and baseline characteristics (pemphigus type, age, sex, race, ethnicity, weight, 
height, and body mass index) will be summarized for the Safety Population using descriptive 
statistics. No formal statistical analyses will be performed, and no inferential statistics reported. 
Prior and concomitant medications will be summarized overall, by dose level, and by the number 
and percentage of subjects taking each medication, classified using World Health Organization. 
Drug Dictionary Anatomical Therapeutic Chemical classes and preferred terms (WHODD). 


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Disposition, baseline demographics and subject characteristics will be summarized for the Safety 
Population. 
11.9 
Efficacy Analysis 
11.9.1 
Subject Response and Disease Progression 
Subject response and disease progression will be determined using PDAI, ABSIS, ABQOL, and 
TABQOL scores. Efficacy data will be presented in listings, by subject, and tabulated overall. 
Data may be combined where appropriate for Parts A and B. Additional sub-group analyses 
(e.g. disease severity, dose group) may be performed as appropriate. 
The PDAI, ABSIS, ABQOL, and TABQOL questionnaires are located in Appendix 5. 
11.10 Safety Analysis 
Quantitative safety data will be summarized by descriptive statistics (arithmetic mean, standard 
deviation, median, minimum, and maximum) by dose level. Summaries will also be presented 
for the change from baseline, when appropriate. All safety analyses will be based on the Safety 
Analysis Population. As appropriate, listings, summary tables and graphs (individual plots and/or 
mean plots) by period will be provided to the Investigator and to the SMC for safety and 
tolerability assessment. 
11.10.1 Adverse Events 
The original verbatim AE terms recorded on the participant’s case report form (CRF) by the 
Investigator will be standardized by the Local Sponsor by assigning preferred terms and system 
organ classes from the most recent available version of the Medical Dictionary for Drug 
Regulatory Affairs (MedDRA). 
AEs will be described by individual listings and frequency tables by preferred terms and body 
system. The NCI CTCAE, version 4.0 will be used for grading AEs. 
11.10.2 Clinical Laboratory Tests 
All clinical laboratory data will be stored in the database in the units in which they were reported. 
Normal ranges for the local laboratory parameters must be provided to Principia/designee before 
the study starts. Participant listings and summary statistics at each assessment time, including 
change from baseline, will be presented using the International System of Units (SI units; Système 
International d’Unités). Laboratory data not reported in SI units will be converted to SI units 
before analysis.  
Clinical laboratory results will be summarized descriptively for baseline and each time point. 
The numerical change from baseline to each time point will be computed. In addition, laboratory 
shift tables will be provided for all laboratory parameters where low/normal/high or abnormal/ 
normal status can be ascertained for shift from baseline to each time point. Listings of individual 
laboratory parameters by visit with normal ranges and abnormality assessments will also be 
completed by subject. 


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Scatter plots of the baseline value (x-axis) and end of study (y-axis) will be completed for each 
continuous clinical laboratory parameter. In addition, individual and group mean laboratory 
values over time (i.e., by visit) will also be completed to evaluate changes and trends in 
laboratory safety. Reference ranges will be highlighted on the plots to identify laboratory 
assessment out of range. Other graphical displays may be created as appropriate. 
11.10.3 Vital Signs 
Vital signs data will be presented by individual listings. Systolic blood pressure values < 90 or 
> 140 mmHg and diastolic blood pressure values < 60 or > 80 mmHg will be flagged as outside 
the normal range. Resting heart rate < 40 or > 100 beats per minute will be flagged as abnormal. 
Descriptive statistics of values and changes from baseline (mean, median, standard deviation) will 
be reported for quantitative variables separately for each period. In addition, tabular and graphical 
summaries will be used, as appropriate. 
11.10.4 Concomitant Medications 
The original terms recorded on the participants’ CRF by the Investigator for concomitant 
medications will be standardized by the Local Sponsor by assigning preferred terms from the 
WHODD (World Health Organization Drug Dictionary) drug terms dictionary for treatments or 
coded via generic name of the concomitant medication. 
Concomitant medications will be presented in summary tables and listings. 
11.11 Pharmacokinetics/Pharmacodynamics Analyses 
Individual and group PK and PD data will be summarized, displayed graphically, and by 
descriptive statistics for each visit where measured. Data will be summarized by descriptive 
statistics and related to clinical responses for each patient and as a group. PD and PD data 
will be pooled with data from other studies, and analyzed and reported separately. 
11.11.1 Missing PK/PD Values  
The methodology of handling missing PK/PD values in the analysis will be determined. 
11.12 Statistical and Analytical Methods 
11.12.1 Descriptive Summaries and Inference 
Descriptive summary statistics will be provided for the safety, PK, and PD variables. 
For continuous variables, these statistics will typically include the number of subjects, mean, 
standard deviation, median, minimum, and maximum. For categorical variables, these statistics 
will typically include the number and percentage of subjects in each category. Descriptive 
summaries will be completed by study part, cohort, and dose. 
Due to the small sample sizes, all p-values derived from inferential analyses will be considered 
informative. In general, all significant testing will be two-sided at significance level 0.05. 
All tests will be made without adjustment for multiplicity or multiple comparisons. 


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11.12.2 Exploratory Analyses 
Exploratory analyses not specified in the protocol or statistical analysis plans may be performed 
to further explore study results. 
11.12.3 Interim Analysis 
An interim analysis to evaluate safety, and to reevaluate sample size and other aspects of study 
design, was conducted for this study when 6 patients completed 4 weeks or more of PRN1008 
therapy (Part A only) (Study PRN1008-005 Interim Analysis Report). 


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12 
SAFETY AND TOXICITY MANAGEMENT 
This section provides detailed information on reporting requirements and interpretation of safety 
assessments for AEs and reporting requirements for serious SAEs. Guidance and reporting 
requirements for pregnancy are also provided. 
12.1 
Safety Monitoring Committee  
A Safety Monitoring Committee (SMC) will review the emerging safety data (TEAEs, safety 
labs), and efficacy, PD, and dose modification data approximately every 3 months within the 
first 6 months following the first subject enrolled in the study, and then with a frequency 
determined as appropriate by the SMC. The SMC members will include the Sponsor's Medical 
Monitor, a Principal Investigator, and a pemphigus clinical expert who is not an investigator in 
the study. The trial statistician will also participate in safety reviews. Documentation of the 
patient data reviewed at each meeting, including the individual SMC member’s confirmation of 
data review and the findings and actions of the SMC, will be included in the Trial Master File. 
SMC findings that impact the safety of patients in this study will be immediately reported to the 
local Competent Authority (CA) and Ethics Committee (EC). 
An SMC Charter outlining the SMC composition and responsibilities will be in place prior to the 
first scheduled meeting. 
12.2 
Adverse Event Collection Period 
The AE Collection Period begins at the time of the first screening/eligibility assessment and ends 
at the end of the study for each patient. 
12.3 
Clinical Adverse Events 
An AE is any untoward medical occurrence in a participant or clinical investigation participant 
administered a pharmaceutical product and which does not necessarily have to have a causal 
relationship with the intervention. An AE can therefore be any unfavorable and unintended sign 
(including an abnormal laboratory finding, for example), symptom, or disease temporally 
associated with the use of an investigational product, whether or not considered related to the 
product. All AEs encountered during the clinical study will be reported in detail in the source 
documents and documented in the CRF, from the date of participant consent throughout the 
follow-up visit.  
Pre-existing conditions that worsen during a study are to be reported as AEs, with the exception 
of the disease under study as it is expected that there may be variation in pemphigus disease 
activity, and this is captured in other measurements. 
The below guidelines should be followed when recording AEs: 
Medical terms: Whenever possible, use recognized medical terms when recording AEs on the 
AE CRF. Do not use colloquialisms or abbreviations. 


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Diagnosis: If known or suspected, record the diagnosis rather than component signs and 
symptoms on the AE CRF and SAE form (e.g., if a diagnosis is made of congestive heart failure, 
record congestive heart failure rather than the symptoms of dyspnea, rales, cyanosis). However, 
signs and symptoms that are considered unrelated to an encountered syndrome or disease should 
be recorded as individual AEs on the AE CRF or SAE form. 
Death: Death is an outcome of an event. The event that resulted in the death should be recorded 
and reported on the AE CRF and SAE form (except for sudden, unexplained death.) 
Surgical or diagnostic procedures: For medical or surgical procedures (e.g., colonoscopy, 
biopsy), the medical condition that led to the procedure is an AE. Elective procedures (e.g., 
vasectomy), planned hospitalizations, and procedures for treatment of conditions noted in the 
patient’s medical history that have not worsened (e.g., planned cataract surgery, hernia repair) 
are not considered AEs. 
Chronic disease: In the case of disease (excluding disease under study) that is progressing by 
episodes (chronic disease), if the disease is known when the participant enters the trial, only 
worsening (increased frequency or intensity of the episodes or attacks) will be documented as an 
AE. If the disease is detected during the trial, and if repeated episodes enable diagnosis of a 
chronic disease, the episodes will be entered with the diagnosis and start/stop dates grouped 
together, and clearly described in the source documents. 
Disease under study (Pemphigus): Unexpected progression, signs, or symptoms of the disease 
under study are not AEs and are not to be recorded on the AE page of the CRFs unless the event 
meets the definition of an SAE or is not consistent with the typical clinical course of the patient’s 
disease as established by the patient’s medical history.  Worsening of the disease under study or 
other disease-related symptoms should be recorded as an AE only if the event meets the 
definition of an SAE or is not consistent with the typical clinical course of the disease.  
Laboratory Out of Range Values: An isolated, out-of-range laboratory result in the absence of 
any associated, clinical finding may or may not be considered an AE; the Investigator’s 
evaluation should be based on a consideration of the overall clinical context. An out-of-range 
laboratory result will be considered clinically significant and recorded as an AE when it is part of 
a clinical abnormality requiring specific medical intervention or follow-up. The test will be 
repeated and the patient will be followed-up until the test value has returned to the normal range 
or baseline, or the Investigator has determined that the abnormality is chronic or stable. The 
Investigator will exercise medical judgment in deciding whether out-of-range values are 
clinically significant and document the assessment in the source records. 


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12.4 
Adverse Event Intensity Grading 
All clinical AEs encountered during the clinical study will be reported on the AE page of the 
CRF. Intensity of AEs will be graded based on the NCI CTCAE, Version 4.0 or higher and 
reported in detail as indicated on the CRF. For any AEs not found in the CTCAE, a description 
of intensity grading can be found below: 
• Grade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; 
intervention not indicated. 
• Grade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age-
appropriate instrumental activities of daily living. 
• Grade 3: Severe or medically significant but not immediately life-threatening; 
hospitalization or prolongation of hospitalization indicated; disabling; limiting 
self-care activities of daily living. 
• Grade 4: Life-threatening consequences; urgent intervention indicated. 
• Grade 5: Death related to AE. 
12.5 
Adverse Event Relationship to Study Drug 
Investigators should use their knowledge of the study participant, the circumstances surrounding 
the event, and an evaluation of any potential alternative causes to determine whether or not an 
AE is considered to be related to the study drug, indicating "yes" or "no" accordingly. 
The following guidance should be taken into consideration: 
• Temporal relationship of event onset to the initiation of study drug 
• Course of the event, considering especially the effects of dose reduction, discontinuation of 
study drug, or reintroduction of study drug (if applicable) 
• Known association of the event with the study drug or with similar treatments 
• Known association of the event with the disease under study 
• Presence of risk factors in the study participant or use of concomitant medications known to 
increase the occurrence of the event 
• Presence of non-treatment-related factors that are known to be associated with the occurrence 
of the event 
12.6 
Treatment and Follow-Up of Adverse Events 
SAEs for which the relationship to the study drug is “related”, should be followed up until they 
have returned to baseline status or stabilized. 


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If after follow-up, return to baseline status or stabilization cannot be established an explanation 
should be recorded on the CRF. 
12.7 
Laboratory and ECG Abnormalities 
Laboratory test results will be recorded on the laboratory results pages of the CRF, or appear on 
electronically produced laboratory reports, if applicable. 
Any treatment-emergent abnormal laboratory or ECG result which is clinically significant, i.e., 
meeting one or more of the following conditions, should be recorded as a single diagnosis on the 
AE page in the CRF: 
• Accompanied by clinical symptoms 
• Leading to a change in study drug (e.g. dose modification, interruption or permanent 
discontinuation) 
• Requiring a change in concomitant therapy (e.g., addition of, interruption of, discontinuation 
of, or any other change in a concomitant medication, therapy or treatment) 
Note: Any laboratory or ECG result abnormality fulfilling the criteria for a serious adverse event 
(SAE) should be reported as such, in addition to being recorded as an AE in the CRF. 
12.7.1 
Follow-Up of Abnormal Laboratory Test Values 
In the event of unexplained clinically significant abnormal laboratory test values, the tests should 
be repeated as soon as possible and followed up until they have returned to the normal range 
and/or an adequate explanation of the abnormality is found. If a clear explanation is established, 
it should be recorded on the CRF. 
12.8 
Serious Adverse Event (SAE) Reporting 
12.8.1 
SAE Definitions 
An SAE is any experience (clinical AE or abnormal laboratory test) that suggests a significant 
hazard, contraindication, side effect, or precaution. An SAE must fulfill at least one of the 
following criteria at any dose level: 
• is fatal (results in the outcome death)* 
• is life-threatening 
• requires in-patient hospitalization or prolongation of existing hospitalization 
• results in persistent or significant disability/incapacity 
• is a congenital anomaly/birth defect 
• is medically significant or requires intervention to prevent one or other of the outcomes listed 
above 


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Note that the term "sudden death" should only be used when the cause is of a cardiac origin as 
per standard definition. The terms "death" and "sudden death" are clearly distinct and must not be 
used interchangeably. 
12.8.2 
SAE Reporting 
Any clinical adverse event or abnormal laboratory test value that is serious and which occurs 
during the course of the study (as defined above), occurring from the enrollment visit (start of 
study screening procedures), including long term follow-up must be reported to: 
• The Local Sponsor (or designee) and monitor within 24 hours of the Investigator becoming 
aware of the event (expedited reporting). 
• The investigational site’s IRB by the investigator in accordance with their regulations. 
Initial notification of an SAE must be confirmed in24 hours from the time the investigational 
team first become aware of the event using the PPD RAVE system, if possible. Paper SAE report 
forms should only to be used when the PPD RAVE system is not accessible, and SAEs should be 
transferred into RAVE once the system is available. 
If PPD RAVE is not accessible, a written, narrative description of any SAE must be scanned and 
emailed to 1008005SAE@principiabio.com within 24 hours of awareness of the event. 
If paper SAE forms are used, copies of the initial and follow-up SAE report forms must be made 
and the originals retained of all information faxed to PPD in the Investigator Site File. 
As further information regarding the SAE becomes available, such follow-up information should 
be documented as an update in the RAVE system, or if RAVE is not accessible, on a new SAE 
report form, marked as a follow-up report and emailed to 1008005SAE@principiabio.com. 
After informed consent, but prior to initiation of study medications, only SAEs caused by a 
protocol-mandated intervention must be reported within 24 hours (e.g., SAEs related to invasive 
screening procedures such as biopsies, medication washout, or no treatment run-in). After first 
study medication, all SAEs must be reported within 24 hours. 
SAEs identified after a subject has exited the study, that the Investigator deems related to 
PRN1008 exposure, should be reported to the Sponsor as noted above. 
Unrelated SAEs must be collected and reported during the study and for up to 30 days after the 
last dose of study medication. 
Suspected Unexpected Serious Adverse Reactions (SUSARs) are reported to Investigators at 
each site and associated IRB when the following conditions occur: 
• The event is a SAE 
• There is a reasonable possibility that the event is an adverse reaction caused by the 
administered drug 


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• The adverse reaction is unexpected, that is to say, not foreseen in the Investigator’s Brochure 
• When all participants at a particular site are off treatment, as defined by the protocol, 
individual SUSAR reports will be forwarded to the site and its associated IRB on an 
expedited basis. 
Individual SUSARs considered to be a significant safety issue and/or which result in a change to 
the informed consent form will be reported in an expedited manner to all Investigators and IRBs. 
Reporting of any SAEs to applicable regulatory authorities will be the responsibility of the Local 
Sponsor in compliance with local regulations. 
12.8.3 
Other Safety Findings Requiring Expedited Reporting 
Significant safety findings will be reported to the Investigator by Principia or designee as 
obtained. The Investigator is responsible for reporting to the investigational site’s IRB in 
accordance with their regulations. Reporting to applicable regulatory authorities will be the 
responsibility of Principia (or designee) in compliance with local regulations. 
12.9 
Pregnancy 
Pregnancy in a Female Clinical Trial Participant: If a female clinical trial participant becomes 
pregnant during the study, she must be instructed to stop taking the study drug and immediately 
inform the Investigator. Pregnancies occurring up to 90 days after the completion of the study 
drug must also be reported to the Investigator. The participant should be counseled by a 
specialist, to discuss the risks of continuing with the pregnancy and the possible effects on the 
fetus. Monitoring of the participant should continue until the outcome of the pregnancy is 
known. The Investigator should report all pregnancies in clinical trial participants to the Sponsor 
within 24 hours of becoming aware of them, using the Clinical Trial Pregnancy Reporting Form. 
Pregnancy in the Partner of a Male Clinical Trial Participant: If the Investigator becomes aware 
of a pregnancy occurring in the partner of a participant participating in the study, the pregnancy 
should be reported to the Sponsor within 24 hours working day of obtaining written consent from 
the pregnant partner. The Investigator will make arrangements for the pregnant partner to be 
counseled by a specialist, to discuss the risks of continuing with the pregnancy and the possible 
effects on the fetus. Monitoring of the pregnant partner should continue until the outcome of the 
pregnancy is known. 
Note: The Pregnancy Reporting Form should only be completed by the Investigator if the 
pregnant partner has signed an Informed Consent Form “Information to Pregnant Partner of 
Participant.” 


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13 
DATA QUALITY ASSURANCE 
The overall procedures for quality assurance of clinical study data, including data collection and 
management, will be described in the Data Management Plan. 
Accurate and reliable data collection will be assured by verification and cross–check of the CRF 
against the Investigator’s records by the study monitor (source document verification), and the 
maintenance of a study drug–dispensing log by the Investigator. 
Data for this study will be recorded in the study Electronic Data Capture (EDC) CRFs. The data 
will be entered by the study center from the source documents into the CRF or will be loaded 
from electronic files (e.g., safety lab data). In no case is the CRF to be considered as source data 
for this study. 
A comprehensive validation check program will verify the data and discrepancy reports will be 
generated accordingly for resolution by the Investigator. All discrepant data will be resolved in 
the EDC database and all data entered in the database will be independently compared with the 
original Investigator’s records. 
13.1 
Assignment of Preferred Terms and Original Terminology 
For classification purposes, preferred terms will be assigned to the original terms recorded on the 
CRF, using the most up to date version of the MedDRA for AEs, diseases and surgical and 
medical procedures, and the WHODD for drug and herbal treatments. 


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14 
ETHICAL ASPECTS 
This section provides information for the Investigator on the ethics requirements for the study, 
including subject informed consent, IRB/EC review of the study and study materials, and 
conditions for modifying or terminating the study. Requirements for financial disclosure for the 
Investigator are also described. 
14.1 
Local Regulations/Declaration of Helsinki 
The Investigator will ensure that this study is conducted in full conformance with the principles 
of the “Declaration of Helsinki” or with the laws and regulations of the country in which the 
research is conducted, whichever affords the greater protection to the individual. The study must 
fully adhere to the principles outlined in current “Guideline for Good Clinical Practice” ICH 
Tripartite Guideline or with local law if it affords greater protection to the participant. 
14.2 
Subject Informed Consent 
It is the responsibility of the Investigator, or a person designated by the Investigator 
[if acceptable by local regulations], to obtain signed and dated informed consent from each 
participant prior to participating in this study after adequate explanation of the aims, methods, 
objectives and potential hazards of the study. The Investigator or designee must also explain that 
the participants are completely free to refuse to enter the study or to withdraw from it at any 
time, for any reason. 
The CRFs for this study contain a field for documenting informed participant consent, and this 
must be completed appropriately. If new safety information results in significant changes in the 
risk/benefit assessment, the consent form should be reviewed and updated if necessary. All 
participants should be informed of the new information, given a copy of the revised form and 
give their consent to continue in the study. 
14.3 
Institutional Review Board Review 
This protocol and any accompanying material provided to the participant (such as participant 
information sheets or descriptions of the study used to obtain informed consent), as well as any 
advertising or compensation given to the participant, will be submitted by principal Investigator 
or coordinating Investigator to the relevant institutional IRB responsible for the investigational 
study.  
An approval letter or certificate (specifying the protocol number and title) from the IRB must be 
obtained before starting the study (initiation). The approval letter to the Investigator should 
specify the date on which the committee met and granted the approval. The Local Sponsor must 
also obtain relevant regulatory authority approvals before starting the study. 
Any modifications made to the protocol after receipt of the IRB approval must also be submitted 
by the principal Investigator or coordinating Investigator to the IRB in accordance with local 
procedures and regulatory requirements. The Local Sponsor must also obtain relevant regulatory 


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approval for protocol modifications according to the local regulations. The Local Sponsor will 
assist the Investigator in submitting the protocol to an appropriate IRB. 
14.4 
Conditions for Modifying the Protocol 
Any protocol modifications must be prepared and approved by a representative of The Sponsor. 
All protocol modifications must be submitted to the appropriate IRB for information and/or 
approval in accordance with local requirements, and to Regulatory Agencies if required. 
Approval must be obtained before any changes can be implemented, except for changes 
necessary to eliminate an immediate hazard to study participants, or when the change(s) involves 
only logistical or administrative aspects of the study [e.g., change in monitor(s), change of 
telephone number(s)]. 
14.5 
Conditions for Terminating the Study 
The Sponsor, the Investigator and the IRB responsible for the study reserve the right to terminate 
the study at any time. Should this be necessary, the parties will consult and arrange the 
termination procedures on an individual study basis. In terminating the study, the Sponsor and 
the Investigator will assure that adequate consideration is given to the protection of the 
participant’s interests. The appropriate IRB and Regulatory Agencies should be informed 
accordingly. 
14.6 
Financial Disclosure 
The Investigator(s) will provide the Sponsor with sufficient accurate financial information to 
allow the Sponsor to submit complete and accurate financial certification or disclosure 
statements to the appropriate regulatory authorities. The Investigator is responsible to promptly 
update any information provided to the Sponsor if relevant changes occur in the course of the 
investigation and for 1 year following the completion of the study (last participant, last visit). 


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15 
STUDY DOCUMENTATION, MONITORING, CASE REPORT FORMS, AND 
RECORD RETENTION REQUIREMENTS 
15.1 
Investigator's File/Retention of Records 
The Investigator must maintain adequate and accurate records to enable the conduct of the study 
to be fully documented and the study data to be subsequently verified. These documents should 
be classified into two different separate categories (1) Investigator’s Study File, and 
(2) participant clinical source documents. 
The Investigator’s Study File will contain the protocol/amendments, CRF data and 
Discrepancies, IRB and regulatory authority approval with correspondence, sample informed 
consent, drug records, staff curriculum vitae and authorization forms and other appropriate 
documents/correspondence, etc. 
Participant clinical source documents independent of the CRF would include participant 
hospital/clinic records, physician’s and nurse’s notes, original laboratory reports, ECG, EEG, 
X-ray, pathology and special assessment reports, signed informed consent forms, consultant 
letters, and participant screening and enrollment logs.  
To comply with international regulations, the records should be retained by the investigator for at 
least 2 years after the last approval of a marketing application in an ICH region and until there 
are no pending or contemplated marketing application in an ICH region, or until at least 2 years 
have elapsed since the formal discontinuation of clinical development of the investigational 
medicinal product. However, the investigator may need to retain these documents for a longer 
period if require by the local regulatory requirements or by agreement with the Sponsor. 
Should the Investigator wish to assign the study records to another party or move them to another 
location, the Sponsor must be notified in advance. 
If the Investigator cannot guarantee this archiving requirement at the investigational site for any 
or all of the documents, special arrangements must be made between the Investigator and the 
Sponsor to store these in a sealed container(s) outside of the site so that they can be returned 
sealed to the Investigator in case of a regulatory audit. Where source documents are required for 
the continued care of the participant, appropriate copies should be made for storing outside of the 
site. 
15.2 
Source Documents and Background Data 
The Investigator shall supply the Sponsor on request with any required background data from the 
study documentation or clinic records. This is particularly important when CRFs are illegible or 
when errors in data transcription are suspected. In case of special problems and/or governmental 
queries or requests for audit inspections, it is also necessary to have access to the complete study 
records, provided that participant confidentiality is protected. 


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15.3 
Audits and Inspections 
The Investigator should understand that source documents for this study should be made 
available to appropriately qualified personnel from the Sponsor or its designees, or to health 
authority inspectors after appropriate notification. The verification of the CRF data must be by 
direct inspection of source documents. 
15.4 
Case Report Forms 
The data collected in the source documents for this study will be entered into the study EDC 
CRF. An audit trail will maintain a record of initial entries and changes made; time and date of 
entry; and name of person making entry or change. For each participant enrolled, a CRF must be 
completed and signed by the Principal Investigator or authorized delegate from the study staff. 
If a participant withdraws from the study, the reason must be noted in the CRF. If a participant is 
withdrawn from the study because of a treatment-limiting adverse event, thorough efforts should 
be made to clearly document the outcome. 
The Investigator should ensure the accuracy, completeness and timeliness of the data reported to 
the Sponsor in the CRFs and in all required reports. 
15.5 
Study Monitoring 
It is understood that the responsible Sponsor study monitor (or designee) will contact and visit 
the Investigator regularly and will be allowed, on request, to inspect the various records of the 
study (CRFs and other pertinent data) provided that participant confidentiality is maintained in 
accordance with local requirements. 
It will be the monitor's responsibility to inspect the EDC CRFs at regular intervals throughout the 
study, to verify the adherence to the protocol and the completeness, consistency and accuracy of 
the data being entered on them. The monitor must verify that the participant received the 
assigned dose. The monitor should have access to laboratory test reports and other participant 
records needed to verify the entries in the EDC CRF. The Investigator (or deputy) agrees to 
cooperate with the monitor to ensure that any problems detected in the course of these 
monitoring visits are resolved. 
15.6 
Confidentiality of Clinical Trial Documents and Participants' Medical Records 
The Investigator must assure that participants’ anonymity will be maintained and that their 
identities are protected from unauthorized parties. In the EDC CRF or other documents 
submitted to the Sponsor, participants should not be identified by their names, but by an 
identification code. 
The Sponsor already maintains rigorous confidentiality standards for clinical studies by “coding” 
(i.e. assigning a unique participant identification (ID) number at the Investigator site) all 
participants enrolled in Sponsor clinical studies. This means that participant names are not 
included in data sets that are transmitted to any Sponsor location.  


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15.7 
Clinical Study Report 
A Clinical Study Report will be written and submitted to relevant IRB/HREC/EC and regulatory 
authorities in accordance with local requirements. 
15.8 
Publication of Data and Protection of Trade Secrets 
It is anticipated that the results of this study may be published or presented at scientific meetings. 
Investigators cannot publish without prior written permission of the Sponsor. Authors must allow 
a reasonable period for manuscript review of at least 30 days. The Sponsor may delay 
publication for commercial reasons for up to 6 months. 


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16 
STUDY ADMINISTRATIVE PROCEDURES 
This section provides additional information on study-related administrative procedures, 
definitions, requirements, and record-keeping activities related to the study assessments 
described in Section 8. 
16.1 
Subject Recruitment Procedures 
Participants will be identified for potential recruitment by the Investigator using recruitment plan 
agreed upon with Principia, possibly including, but not limited to, a listing from study center, 
volunteer database, newspaper/radio/internet advertisement, or mailing list. 
16.2 
Subject Enrollment Procedures 
Participants cannot commence enrollment procedures until all entry criteria have been fulfilled. 
Where the clinical significance of an abnormal screening test result (lab or any other tests) is 
considered uncertain, the test may be repeated. 
The Investigator or designee will enter data for each enrolled participant in the study CRF and 
enter the corresponding number for allocation to the treatment groups in the appropriate place on 
each participant’s CRF. A participant enrollment and Identification Code List must be 
maintained by the Investigator or Pharmacist, or designee. 
Under no circumstances will participants who enroll in this study and complete treatment as 
specified be permitted to re-enroll in the study. 
16.3 
Patient Premature Withdrawal – Definition  
A patient who withdraws from the study before taking one or more doses of study drug is 
considered to have withdrawn from the study early. 
16.4 
Procedures for Patients Who Withdraw from the Study 
Participants have the right to withdraw from the study at any time for any reason. In the event 
that a participant decides to discontinue from the study, he/she should be asked if he/she can still 
be contacted for further information. The outcome of that discussion should be documented in 
both the medical records and in the CRF. 
When applicable, participants should be informed of circumstances under which their 
participation may be terminated by the Investigator without the participant's consent. The 
Investigator may withdraw participants from the study in the event of intercurrent illness, AEs, 
treatment failure after a prescribed procedure, lack of compliance with the study and/or study 
procedures or any other reasons where the Investigator feels it is in the best interest of the 
participant to be terminated from the study. 


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Reasons for withdrawal must be documented and explained to the participant. It is understood by 
all concerned that an excessive rate of withdrawals can render the study uninterpretable; 
therefore, unnecessary withdrawal of participants should be avoided. Should a participant decide 
to withdraw, all efforts should be made to complete and report the observations as thoroughly as 
possible, particularly the follow-up examinations. 
The Investigator should contact the participant or a responsible relative either by telephone 
(followed by registered mail) or through a personal visit to determine as completely as possible 
the reason for the withdrawal. A complete final evaluation at the time of the participant’s 
withdrawal should be made, with an explanation of why the participant is withdrawing from the 
study. If the reason for removal of a participant from the study is an AE, the principal specific 
event must be recorded on the CRF. The participant should be followed until the AE is resolved, 
if possible. 
16.5 
Treatment Compliance 
Accountability and participant compliance will be assessed by maintaining adequate study drug 
dispensing records and medication counts. The Investigator is responsible for ensuring that 
dosing is administered in compliance with the protocol. See Section 10.4 for instructions on 
Drug Accountability procedures. 
16.6 
Recording of AEs on the CRF 
All AEs encountered during the clinical study will be reported in detail in the source documents 
and documented in the CRF, from the date of participant consent throughout the follow-up visit. 
SAEs for which the relationship to test drug is considered “related”, should be followed up until 
they have returned to the baseline status or stabilized. If the cause of the AE is established, it 
should be described in detail, including supporting information, in the source documents and on 
the CRF. 
16.7 
Physical Examination Procedures 
At screening and follow-up visits, a complete physical examination will consist of checking the 
normality or abnormality of the following body systems: general appearance, skin, eyes, ears, 
nose, throat, heart, chest/breast, abdomen, neurological system, lymph nodes, spine and 
extremities (skeletal). 
An abbreviated physical examination will consist of checking the normality or abnormality of 
the following body systems: general appearance, abdomen, and cardiorespiratory examination. 
The results of the physical examination will be summarized as normal/abnormal. In case of 
abnormality, details will be recorded in the participant notes and in the CRF. 
Height will be recorded at screening only. 


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16.8 
Vital Signs Procedures 
Blood pressure (BP), pulse rate, body temperature and respiratory rate will be recorded at the 
time points specified in the Schedule of Assessments (Appendix 4). 
16.9 
Body Weight 
Body weight should be measured on the same digital clinic scale, after checking for accurate 
zero calibration each time. Weight is recorded in kg to one decimal place. 
16.10 ECG Procedures 
Single 12-lead ECG assessments will be obtained as specified in the Schedule of Assessments 
(Appendix 4) to confirm eligibility and to ensure real time safety evaluation of the participants in 
the study, as specified in the Schedule of Assessments. Unscheduled ECGs may be performed as 
clinically indicated and recorded in the eCRF. 
Participants should be in a resting position for at least 10 minutes prior to any measurement. 
Body position should also be consistently maintained for each ECG evaluation. In particular, 
changes in heart rate should be avoided. There should be no environmental distractions 
(TV, radio, conversation) during the pre-ECG rest and the ECG recording time. 
Heart rate (HR), QRS duration and respiratory rate (RR), and QT intervals will be recorded. 
Changes of the T-wave and U-wave morphology and overall ECG interpretation will be 
documented. 
All ECG recordings must be performed using a standard high-quality, high-fidelity digital 
electrocardiograph machine equipped with computer-based interval measurements. 
For safety monitoring purposes, the Investigator must review, sign and date all ECG tracings. 
Paper copies will be kept at the study center with the participant's clinical file as part of the 
permanent record. The ECG intervals and interpretation will be recorded on the CRF. 
16.11 Laboratory Test Procedures 
The laboratory assessments will be performed at a central laboratory, with the provision for 
occasional local laboratory testing, if required. Laboratory safety tests shall be collected at time 
points specified in the Schedule of Assessments (Appendix 4). 
Additional blood or urine samples may be taken at the discretion of the Investigator if the results 
of any test fall outside the reference ranges, or clinical symptoms necessitate additional testing to 
monitor participant safety. Where the clinical significance of abnormal lab results is considered 
uncertain, screening lab tests may be repeated before randomization to confirm eligibility. In the 
event of unexplained abnormal clinically significant laboratory test values, the tests should be 
repeated immediately and followed up until they have returned to the normal range, are 
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16.11.1 Recording of Laboratory Test Results on the CRF  
Results of clinical laboratory testing will be recorded on the CRF or be received as electronically 
produced laboratory reports submitted directly from the local or central laboratory, if applicable. 
The procedures for the collection, handling and shipping of laboratory samples will be specified 
in the Sample Collection, Handling and Logistics Manual. 
16.12 Recording of Concomitant Medications on the CRF 
All medications (prescription and over-the-counter [OTC]) taken within 28 days prior to study 
screening (reference Exclusion Criteria, Item #5) will be recorded in the CRF. 


A Phase 2 Study of PRN1008 in Pemphigus 
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Page 73 of 93 
17 
REFERENCES 
Byrd JC, Furman RR, Coutre SE, Flinn IW, Burger JA, Blum KA, Grant B, Sharman JP, 
Coleman M, Wierda WG, Jones JA, Zhao W, Heerema NA, Johnson AJ, Sukbuntherng J, Chang 
BY, Clow F, Hedrick E, Buggy JJ, James DF, O'Brien S. Targeting BTK with Ibrutinib in 
Relapsed Chronic Lymphocytic Leukemia. N Engl J Med., 369(1):32-42, 2013.  
Bizikova P, Olivry T, Mamo LB, Dunston SM. Serum autoantibody profiles of IgA, IgE and IgM 
in canine pemphigus foliaceus. Vet Dermatol 2014;25:471-e75. 
Bizikova P, Dean GA, Hashimoto T, Olivry T. Cloning and establishment of canine 
desmocollin-1 as a major autoantigen in canine pemphisgus foliaceus. Vet Immunol 
Immunopathol 2012;149:197-207. 
Development Report #DVR0210, A Pilot Study of the Efficacy of a Bruton’s Tyrosine Kinase 
Inhibitor (BTKi) in the Treatment of Dogs with Pemphigus Foliaceus (PF). Principia Biopharma, 
Inc., South San Francisco, CA, 94080, U.S.A. 
Evans EK, Tester R, Aslanian S, Karp R, Sheets M, Labenski MT, Witowski SR, Lounsbury H, 
Chaturvedi P, Mazdiyasni H, Zhu Z, Nacht M, Freed MI, Petter RC, Dubrovskiy A, Singh J, 
Westlin WF. Inhibition of Btk with CC-292 Provides Early Pharmacodynamic Assessment of 
Activity in Mice and Humans. J Pharmacol Exp Ther, 346(2):219-28, 2013. 
Hertl, M., Jedlickova, H., Karpati, S., et al. (2015), Pemphigus. S2 Guideline for diagnosis and 
treatment – guided by the European Dermatology Forum (EDF) in cooperation with the 
European Academy of Dermatology and Venereology (EADV). Journal of the European 
Academy of Dermatology and Venereology, 29: 405–414. doi: 10.1111/jdv.12772. 
Horváth, B., Huizinga, J., Pas, H.H., Mulder, A.B., Jonkman, M.F., Low Dose rituximab is 
effective in pemphigus. Br J Dermatol. 166(2): 405-12, 2012.  
Imbruvica [package insert]. Pharmacyclics, Inc., Sunnyvale, CA; 2015. 
Murrell DF, Dick S, Ahmed AR, Amagai M, Barnadas MA, Borradori L, et al. Consensus 
statement on definitions of disease, end points, and therapeutic response for pemphigus. J Am 
Acad Dermatol. 2008;58:1043–6. 
Mohamed AJ, Yu L, Backesjo CM, Vargas L, Faryal R, et al. Bruton's tyrosine kinase (Btk): 
function, regulation, and transformation with special emphasis on the PH domain. Immunol 
Rev 228, 58-73, 2009. 
PRN1008-005 Interim Analysis Report February 2017 
PRN1008 Investigator Brochure, Principia Biopharma. 
Principia Study PRN1008-006, data on file, 2016. 


A Phase 2 Study of PRN1008 in Pemphigus 
Protocol PRN1008-005, Version 7.0 
Principia Biopharma Australia Pty Ltd.  
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Page 74 of 93 
Rosenbach M, Murrell D, Bystryn JC, et al. Reliability and convergent validity of two outcome 
instruments for pemphigus. J Invest Dermatol 2009;129(10):2404–10.  
Sideras P and Smith CI. Molecular and cellular aspects of X-linked agammaglobulinemia. 
Adv Immunol, 59: 135-223, 1995. 
Tjokrowidjaja A, Daniel BS, Frew JW, Sebaratnam DF, Hanna AM, Chee S, Dermawan A, 
Wang CQ, Lim C, Venugopal SS, Rhodes LM, Welsh B, Nijsten T, Murrell DF. Br J Dermatol. 
2013 Nov;169(5):1000-6. 
Tsukada, S., Saffran, D. C., Rawlings, D. J., Parolini, O., Allen, R. C., Klisak, I., Sparkes, R. S., 
Kubagawa, H., Mohandas, T., Quan, S., and et al. Deficient expression of a B cell cytoplasmic 
tyrosine kinase in human X-linked agammaglobulinemia. Cell, 72: 279-290, 1993. 
Drug Development and Drug Interactions: Table of Substrates, Inhibitors and Inducers. U.S. 
Food and Drug Administration. 
http://www.fda.gov/drugs/developmentapprovalprocess/developmentresources/druginteractionsla
beling/ucm093664.htm# Accessed 07 April 2016. 
Vetrie, D., Vorechovsky, I., Sideras, P., Holland, J., Davies, A., Flinter, F., Hammarstrom, L., 
Kinnon, C., Levinsky, R., Bobrow, M., and et al. The gene involved in X-linked 
agammaglobulinaemia is a member of the src family of protein-tyrosine kinases. Nature, 361: 
226-233, 1993. 
Wang ML, Rule S, Martin P, Goy A, Auer R, Kahl BS, Jurczak W, Advani RH, Romaguera JE, 
Williams ME, Barrientos JC, Chmielowska E, Radford J, Stilgenbauer S, Dreyling M, 
Jedrzejczak WW, Johnson P, Spurgeon SE, Li L, Zhang L, Newberry K, Ou Z, Cheng N, Fang 
B, McGreivy J, Clow F, Buggy JJ, Chang BY, Beaupre DM, Kunkel LA, Blum KA. Targeting 
BTK with Ibrutinib in Relapsed or Refractory Mantle-Cell Lymphoma. N Engl J Med. 2013 
Jun 19. [Epub ahead of print]. 
Werth VP, Fivenson D, Pandya AG, et al. Multicenter randomized, double-blind, placebo-
controlled, clinical trial of dapsone as a glucocorticoid-sparing agent in maintenance-phase 
pemphigus vulgaris. Arch Dematol. 2008 Jan;144(1):25-32.  
Wilson MM, Thomas DR, Rubenstein LZ, Chibnall JT, Anderson S, Baxi A, Diebold MR, 
Morley JE. Appetite assessment: simple appetite questionnaire predicts weight loss in 
community-dwelling adults and nursing home residents. Am J Clin Nutr. 2005 
Nov;82(5):1074-81. 


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APPENDIX 1A 
CORTICOSTEROID RESCUE CRITERIA AND TREATMENT 
PROTOCOL (PART A) 
Systemic corticosteroids will be avoided during PRN1008 monotherapy unless these rescue 
criteria are triggered, as described below, or if patients enter the study on low doses (≤ 0.5 mg/kg 
mg per day). In each rescue scenario, the recommended method of corticosteroid taper is shown 
in Appendix 7. 
Rescue Criteria #1: Flare which, in the mind of investigator, is potentially life threatening  
• Commence conventional immunosuppressive therapy and discontinue PRN1008 
Rescue Criteria #2: Development of severe but not life-threatening PV (defined as PDAI 
score ≥ 45) at any time during PRN1008 therapy: 
• If most recent BTK trough occupancy was 50% or more, commence conventional 
immunosuppressive therapy and discontinue PRN1008 
• If most recent BTK trough occupancy was < 50% and PRN1008 dose has not been increased 
since last BTK assay, commence, or increase dose to, full-dose prednis(ol)one (1.0 mg/kg) 
orally and increase dose of PRN1008 by an amount agreed to with the Sponsor’s Medical 
Monitor. At next review: 
- 
If not responding, commence conventional immunosuppressive therapy and discontinue 
PRN1008 
- 
If responding, maintain or taper corticosteroids as clinically appropriate while 
maintaining PRN1008 treatment 
• If no BTK trough occupancy is available to guide therapy, maintain PRN1008, and 
commence, or increase to, full-dose prednis(ol)one (1.0 mg/kg) orally. Seek to expedite latest 
BTK trough occupancy test result or arrange for a new blood draw to measure BTK trough 
occupancy. At next review: 
- 
If not responding, commence conventional immunosuppressive therapy and discontinue 
PRN1008 
- 
If responding, maintain or taper corticosteroids as clinically appropriate while 
maintaining PRN1008 treatment (including adjust dose upwards if BTK trough 
occupancy < 50% in consultation with the Sponsor Medical Monitor) 


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Rescue Criteria #3: Patients with non-severe PV but who fail to achieve “Control of Disease 
Activity (CDA*)” by 4 weeks 
• If most recent BTK trough occupancy was 50% or more, commence, or increase dose to, low 
dose prednis(ol)one (0.5 mg/kg) orally. Review after another ~2 weeks: 
- 
If not responding, commence conventional immunosuppressive therapy and discontinue 
PRN1008 
- 
If responding, maintain or taper corticosteroids as clinically appropriate while 
maintaining PRN1008 treatment 
• If most recent BTK trough occupancy was < 50% and PRN1008 dose has not been increased 
since last BTK assay, increase dose of PRN1008 by an amount agreed to with the Sponsor’s 
Medical Monitor. At review after another ~2 weeks: 
- 
If not responding, commence, or increase dose to, low-dose prednis(ol)one (0.5 mg/kg) 
orally and maintain PRN1008 treatment. If a dose of ≥ 0.5 mg/kg is already in use, or if 
after further review the patient is still not responding, commence conventional 
immunosuppressive therapy and discontinue PRN1008. 
- 
If responding, maintain PRN1008 treatment and consider tapering of prednis(ol)one 
In each rescue scenario, the recommended method of corticosteroid taper is shown in 
Appendix 7. 
When patients enter the study on corticosteroids, the dose should be maintained for the initial 
2 weeks of PRN1008 therapy if possible in order to evaluate the additive effects of PRN1008. 
During this time and subsequently, the rescue criteria should be followed if indicated. In other 
words, in some circumstances the corticosteroid dose should be increased, with or without 
cessation of PRN1008. If patients respond well to PRN1008 without increasing the corticosteroid 
dose, the Werth taper (Appendix 7) should be commenced after four weeks of PRN1008 therapy. 
*As defined by the EADV 2014 Pemphigus S2 Guideline (Hertl et al. 2015) 
 
 


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APPENDIX 1B 
CORTICOSTEROID RESCUE CRITERIA AND TREATMENT 
PROTOCOL (PART B) 
Systemic corticosteroids will be avoided during PRN1008 monotherapy unless these rescue 
criteria are triggered, as described below, or if patients enter the study on low doses (≤ 0.5 mg/kg 
mg per day). In each rescue scenario, the recommended method of corticosteroid taper is shown 
in Appendix 7. 
Rescue Criteria #1: Flare which, in the mind of investigator, is potentially life threatening  
• Commence conventional immunosuppressive therapy and discontinue PRN1008 
Rescue Criteria #2: Development of severe (defined as PDAI score ≥ 45) but not life-
threatening PV or clinically significant worsening of already severe PV during PRN1008 
therapy: 
• Commence, or increase dose to, full-dose prednis(ol)one (1.0 mg/kg) orally and increase 
dose of PRN1008 by an amount agreed to with the Sponsor’s Medical Monitor to a 
maximum of 600 mg bid. At next review: 
- 
If not responding, commence conventional immunosuppressive therapy and discontinue 
PRN1008 
- 
If responding, maintain or taper corticosteroids as clinically appropriate while 
maintaining PRN1008 treatment. 
Rescue Criteria #3: Patients with non-severe PV but who fail to achieve “Control of Disease 
Activity (CDA*)” by 4 weeks 
• If partially responding, increase dose of PRN1008 if not already maximal (Table 4), review 
in 1-2 weeks, and then consider additional CS up to a dose of 0.5 mg/kg. If subsequently 
responding, maintain PRN1008 treatment and taper prednis(ol)one. 
• If not responding, increase dose of PRN1008 if not already maximal (Table 4), review in 
1-2 weeks, and then consider additional CS up to a dose of 1.0 mg/kg. If subsequently 
responding, maintain PRN1008 treatment and taper prednis(ol)one. 
Table 4 
Dose Escalation Rules Part B 
Current Dose 
Inadequate Clinical Response1 Dose-Escalation Rules 
400 mg qd 
Increase to 400 mg bid (allowed at Week 3 visit or later)2 
400 mg bid 
Increase to 600 mg bid (allowed at Week 5 visit or later)2 
600 mg bid 
No dose increase possible. 
1. Investigator discretion: Generally, clinical response is shown by some improvement seen in first 2 weeks with 
CDA achieved by the Week 5 visit 
2. Unless tolerability issues preclude dose-escalation 
 


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In each rescue scenario, the recommended method of corticosteroid taper is shown in 
Appendix 7. 
When patients enter the study on corticosteroids, the dose should be maintained for the initial 
2 weeks of PRN1008 therapy if possible in order to evaluate the additive effects of PRN1008. 
During this time and subsequently, the rescue criteria should be followed if indicated. In other 
words, in some circumstances the corticosteroid dose should be increased, with or without 
cessation of PRN1008. If patients respond well to PRN1008 without increasing the corticosteroid 
dose, the Werth taper (Appendix 7) should be commenced after four weeks of PRN1008 therapy. 
* As defined by the EADV 2014 Pemphigus S2 Guideline (Hertl et al. 2015) 
 


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APPENDIX 2 
STRONG AND MODERATE CYP3A INHIBITORS AND 
INDUCERS 
 
Strong 
Moderate 
3A Inducers 
Avasimibe, carbamazepine, phenytoin, 
rifampin, St. John’s wort 
Bosentan, efavirenz, etravirine, 
modafinil, nafcillin 
3A Inhibitors 
Boceprevir, 
clarithromycin, conivaptan, 
grapefruit juice, indinavir, itraconazole, 
ketoconazole,  
lopinavir/ritonavir, 
mibefradil,  
nefazodone, nelfinavir, 
posaconazole, ritonavir,  
saquinavir,  
telaprevir, 
telithromycin, 
voriconazole 
Amprenavir, aprepitant, 
atazanavir, ciprofloxacin, 
darunavir/ritonavir, diltiazem, 
erythromycin, fluconazole, 
fosamprenavir, Seville orange juice, 
grapefruit juice, 
imatinib, verapamil 
From:  Drug Development and Drug Interactions: Table of Substrates, Inhibitors and Inducers. U.S. Food and 
Drug Administration.  


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APPENDIX 3 
SENSITIVE CYP3A SUBSTRATES INCLUDING THOSE WITH 
NARROW THERAPEUTIC INDEX 
3A Sensitive Substrate 
Subset with Narrow Therapeutic Index 
Alfentanil, aprepitant, budesonide, buspirone, 
conivaptan, darifenacin, darunavir, dasatinib, 
dronedarone, eletriptan, eplerenone, everolimus, 
felodipine, indinavir, fluticasone, lopinavir, 
lovastatin, lurasidone, maraviroc, midazolam, 
nisoldipine, quetiapine, saquinavir, sildenafil, 
simvastatin, sirolimus, tolvaptan, tipranavir, 
triazolam, vardenafil 
Alfentanil, astemizole, cisapride, cyclosporine, 
dihydroergotamine, ergotamine, fentanyl, 
pimozide, quinidine, sirolimus, tacrolimus,  
terfenadine 
From: Drug Development and Drug Interactions: Table of Substrates, Inhibitors and Inducers. U.S. Food and 
Drug Administration.  
 
 


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Page 81 of 93 
APPENDIX 4 
SCHEDULE OF ASSESSMENTS  
PART A: 12-WEEK TREATMENT/ 12-WEEK FOLLOW-UP: 
 
Screen 
Day 1, 
Week 1 
Pre-dose 
Day 1 
Week 1 
Post-doseg 
Day 2, 
Week 1a 
Day 15, 
Week 3 
+/- 3 days 
Day 29, 
Week 5 
+/- 3 days 
Day 57, 
Week 9 
+/- 7 days 
Day 85, 
Week 13 
+/- 7 days 
Day 113, 
Week 17 
+/- 7 days 
Day 141, 
Week 21 
+/- 7 days 
Day 169, 
Week 25 
+/- 7 days 
Unscheduled 
Visit 
Informed Consent 
X 
 
 
 
 
 
 
 
 
 
 
 
Inclusion/Exclusion Criteria 
X 
X 
 
 
 
 
 
 
 
 
 
 
Height 
X 
 
 
 
 
 
 
 
 
 
 
 
Weight 
X 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
Physical exam./med. 
History, PDAI and ABSIS 
X 
 
 
 
 
 
 
 
 
 
 
 
Abbreviated physical exam, 
PDAI, ABSIS 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
ECG (12-lead) 
X 
 
 
 
 
 
 
 
 
 
 
(X)b 
Vital Signs 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Urinalysis 
X 
 
 
 
 
 
 
 
 
 
 
 
Hep B & C, HIV,  
T-spot TB Test, 
QuantiFERON®-TB Gold, 
QuantiFERON®-TB Gold 
Plus (QFT Plus) 
X 
 
 
 
 
 
 
 
 
 
 
 
Pregnancy testc 
X 
X 
 
 
 
X 
X 
X 
 
 
X 
 
Skin biopsyd 
X 
 
 
 
 
 
 
 
 
 
 
 
Hem, Coag, Chem 
Xe 
X 
 
 
X 
X 
X 
X 
 
 
 
X 
FSHf 
X 
 
 
 
 
 
 
 
 
 
 
 
BTK occupancy sample 
 
Xg 
Xh 
X 
X 
X 
X 
X 
 
 
 
(X)b 
PK sample 
 
X 
Xh 
X 
X 
X 
X 
X 
 
 
 
(X)b 
Anti-DSG antibodies. 
 
X 
 
 
 
X 
X 
X 
X 
X 
X 
 
Photography (Optional)i 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
ABQOL & TABQOL 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
SNAQ questionnaire 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
AEs 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Concomitant Medications 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Drug dispensed 
 
X 
 
 
 
X 
X 
 
 
 
 
 
Drug reconciliation 
 
 
 
 
X 
X 
X 
X 
 
 
 
 


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Page 82 of 93 
Schedule of Assessments Footnotes: 
a. 
Withhold PRN1008 on morning of Day 2 until PK/PD measurement has been taken as close to 12 hours after second dose as possible. Where follow-up on Day 2 is not 
possible, PK/PD samples may be taken on another day in the first week of treatment. On all other days, instruct patient to take PRN1008 in the morning as 
usual prior to clinic and take note of the time taken. Extra PK/PD sample intended for 1 to 5 days after dose adjustment or to replace missing samples–not 
required for other extra visits.  
b. Only if clinically indicated. 
c. 
For women of childbearing potential only. Serum pregnancy test done at screening, urine dip test done at other time points. 
d. Performed only if no suitable prior biopsy. 
e. 
TSH and CPK taken as part of chemistry panel. 
f. 
To confirm postmenopausal status for women who are not surgically sterile only. 
g. Two-8mL pre-dose blood tubes to be collected at baseline to ensure sufficient samples for later time point assay calculations. 
h. 2 hours post-dose (+/- 15 mins) 
i. 
Photography is used to document skin disease changes; ideally in most patients. Strict masking of patient identity is required. 


A Phase 2 Study of PRN1008 in Pemphigus 
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Page 83 of 93 
PART B:  SCHEDULE OF ASSESSMENTS (24-weeks Treatment/ 4-weeks Follow Up) 
 
Screen 
Day 1, 
Week 1 
Pre-dose 
Day 1 
Week 1 
Post-dosea 
Day 2, 
Week 1g 
Day 15, 
Week 3 
+/-3 days 
Day 29, 
Week 5 
+/-3 days 
Day 57, 
Week 9 
+/-7 days 
Day 85, 
Week 13 
+/-7 days 
Day 113, 
Week 17 
+/-7 days 
Day 141, 
Week 21 
+/-7 days 
Day 169, 
Week 25 
+/-7 days 
Day 197, 
Week 29 
+/-7 days 
Unscheduled 
Visit 
Informed Consentk 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Inclusion/Exclusion 
Criteria 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
Height 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Weight 
X 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Physical 
exam/med.History PDAI, 
ABSIS 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Abbreviated physical 
exam., PDAI, ABSIS 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
ECG (12-lead) 
X 
 
 
 
 
 
 
 
 
 
 
 
(X)b 
Vital Signsj 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Urinalysis 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Hep B &C, HIV,  
T-spot TB Test, 
QuantiFERON®-TB 
Gold, QuantiFERON®-
TB Gold Plus (QFT Plus) 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Pregnancy testc 
X 
X 
 
 
 
X 
X 
X 
X 
X 
X 
X 
 
Skin biopsyd 
X 
 
 
 
 
 
 
 
 
 
 
 
Hem, Coag, Chem 
Xe 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
FSHf 
X 
 
 
 
 
 
 
 
 
 
 
 
 
BTK occupancy sample 
 
Xg 
Xh 
X 
X 
X 
X 
X 
X 
X 
X 
 
(X) b 
PK sample  
 
X 
Xh 
Xa 
X 
X 
X 
X 
X 
X 
X 
 
(X) b 
Anti-DSG antibodies. 
 
X 
 
 
 
X 
X 
X 
X 
X 
X 
X 
 
Photography (Optional)i 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
ABQOL & TABQOL 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
SNAQ questionnaire 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
AEs 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Concomitant 
Medications 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Drug dispensed 
 
X 
 
 
 
X 
X 
X 
X 
X 
 
 
 
Drug reconciliation 
 
 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
 


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Schedule of Assessments Footnotes: 
a. 
Withhold PRN1008 on morning of Day 2 until PK/PD measurement has been taken as close to 12 hours (when bid dosing) or 24 hours (when qd dosing) after the prior dose 
as possible. Where follow-up on Day 2 is not possible, PK/PD samples may be taken on another day in the first week of treatment. On all other days, instruct 
patient to take PRN1008 in the morning as usual prior to clinic and take note of the time taken. Extra PK/PD sample intended for 1 to 5 days after dose 
adjustment or to replace missing samples–not required for other extra visits. 
b. Only if clinically indicated. 
c. 
For women of childbearing potential only. Serum pregnancy test done at screening, urine dip test done at other time points. 
d. Performed only if no suitable prior biopsy. 
e. 
TSH and CPK taken as part of chemistry panel. 
f. 
To confirm postmenopausal status for women who are not surgically sterile only. 
g. Two-8mL pre-dose blood tubes to be collected at baseline to ensure sufficient samples for later time point assay calculations. 
h. 2 hours post-dose (+/- 15 mins) 
i. 
Photography is used to document skin disease changes; ideally in most patients. Strict masking of patient identity is required. 
j. 
Vital sign include blood pressure (BP), pulse rate, body temperature and respiratory rate and are recorded at the time points specified 
k.  The Schedule of Assessments has been revised to be consistent with the 24 week treatment duration and 4 week safety follow up assessments described in 
this protocol Version 5, Part B. Patients enrolled in an earlier protocol version (Part A) who are in the active12-week treatment period are eligible to 
continue treatment, initially at their current dose, for up to a total of 24 weeks active treatment upon review and signature of the Part B EC approved Patient 
Informed Consent Form, with procedures and tests per this Schedule. Patients that have completed Part A of the trial are also eligible to be screened as a 
Part B patient, upon review and signature of the Part B EC-approved Patient Informed Consent Form. 
 
 
 


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Page 85 of 93 
APPENDIX 5 
PEMPHIGUS DISEASE ACTIVITY AND QUALITY OF LIFE 
EVALUATION INSTRUMENTS:  ABQOL, TABQOL, PDAI, ABSIS 


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Treatment of Autoimmune Bullous Diseases Quality of Life Assessment (TABQOL) 


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Principia Biopharma Australia Pty Ltd.  
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Page 89 of 93 
From: Tjokrowidjaja et al. 2013 


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Principia Biopharma Australia Pty Ltd.  
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Page 90 of 93 
Pemphigus Disease Activity Index (PDAI) (Murrell et al. 2008)  


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Page 91 of 93 
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) (Rosenbach et al. 2009) 


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Principia Biopharma Australia Pty Ltd.  
19 March 2019 
 
Page 92 of 93 
APPENDIX 6 
SIMPLE NUTRITIONAL APPETITE QUESTIONNAIRE (SNAQ) 
From Wilson et al., 2005 
 


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Principia Biopharma Australia Pty Ltd.  
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Page 93 of 93 
APPENDIX 7 
CORTICOSTEROID TAPER PROTOCOL 
Maintain corticosteroid dose for 2 weeks after disease control has been achieved. 
Subsequently, reduce the corticosteroid dose by 15% every three weeks. 
-OR- 
 
 
From: Werth et al. 2008 
 
 

SAP_001.pdf: 
Confidential 
STATISTICAL ANALYSIS PLAN 
Protocol Title: 
An Open-Label, Phase 2, Pilot Study Investigating the Safety, 
Clinical Activity, Pharmacokinetics, and Pharmacodynamics of 
Oral Treatment with the BTK Inhibitor PRN1008 in Patients with 
Newly Diagnosed or Relapsing Pemphigus Vulgaris (Part A) 
Protocol Number: 
PRN1008-005 
Sponsor: 
Principia Biopharma Inc. 
400 East Jamie Court, Suite 302 
South San Francisco, CA 94080 
Phone: (650) 416-7700 
Author: 
Summit Analytical, LLC 
8354 Northfield Blvd. 
Bldg. G Suite 3700 
Denver, CO 80205 
SAP Date: 
2019-1-16 
Status: 
Final V2.0 
NCT02704429


Principia Biopharma                             .                                                                                                               Statistical Analysis Plan, Final V2.0 
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Table of Contents 
LIST OF ABBREVIATIONS ...................................................................................................... V 
1. 
INTRODUCTION ............................................................................................................ 1 
2. 
OBJECTIVES ................................................................................................................... 3 
2.1. PRIMARY OBJECTIVES ........................................................................................................ 3 
2.2. SECONDARY OBJECTIVE ..................................................................................................... 3 
2.3. EXPLORATORY OBJECTIVE ................................................................................................. 3 
3. 
INVESTIGATIONAL PLAN .......................................................................................... 4 
3.1. OVERALL STUDY DESIGN AND PLAN .................................................................................. 4 
3.2. STUDY ENDPOINTS ............................................................................................................. 5 
3.2.1. 
Safety Endpoints.......................................................................................................... 5 
3.2.2. 
Primary Efficacy Endpoint ......................................................................................... 5 
3.2.3. 
Secondary Efficacy Endpoints .................................................................................... 5 
3.2.4. 
PK/PD Endpoints ........................................................................................................ 6 
3.3. TREATMENTS ...................................................................................................................... 7 
3.4. DOSE ADJUSTMENT/MODIFICATIONS ................................................................................. 7 
4. 
GENERAL STATISTICAL CONSIDERATIONS ....................................................... 8 
4.1. SAMPLE SIZE ...................................................................................................................... 8 
4.2. RANDOMIZATION, STRATIFICATION, AND BLINDING .......................................................... 8 
4.3. ANALYSIS POPULATIONS .................................................................................................... 9 
4.3.1. 
Screening Population .................................................................................................. 9 
4.3.2. 
Safety Analysis Population ......................................................................................... 9 
4.3.3. 
Efficacy Analysis Population (ITT Population) .......................................................... 9 
4.3.4. 
mITT Population ......................................................................................................... 9 
4.3.5. 
Per Protocol (PP) Population .................................................................................... 9 
4.3.6. 
Pharmacokinetic Analysis Population ........................................................................ 9 
5. 
PATIENT DISPOSITION ............................................................................................. 10 
5.1. DISPOSITION ..................................................................................................................... 10 
5.2. PROTOCOL DEVIATIONS ................................................................................................... 10 
6. 
DEMOGRAPHICS AND BASELINE CHARACTERISTICS .................................. 11 
6.1. DEMOGRAPHICS ................................................................................................................ 11 
6.2. MEDICAL HISTORY ........................................................................................................... 11 
6.3. INCLUSION AND EXCLUSION CRITERIA ............................................................................. 12 
7. 
TREATMENTS AND MEDICATIONS ....................................................................... 13 
7.1. PRIOR AND CONCOMITANT MEDICATIONS ........................................................................ 13 
7.2. STUDY TREATMENTS ........................................................................................................ 13 
7.2.1. 
Extent of Exposure .................................................................................................... 13 
7.2.2. 
Treatment Compliance and Modifications................................................................ 14 
8. 
EFFICACY ANALYSIS................................................................................................. 15 


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 III 
8.1. PRIMARY EFFICACY ENDPOINT ......................................................................................... 15 
8.2. SECONDARY EFFICACY ENDPOINT .................................................................................... 15 
8.2.1. 
Achieved CDA without CS within 4 Weeks ............................................................... 15 
8.2.2. 
Achieved CR without CS within 12 Weeks ................................................................ 16 
8.2.3. 
Achieved CR without Prednis(ol)one >0.5 mg/kg within 12 Weeks ......................... 16 
8.2.4. 
Proportion of CDA and CR at each Visit.................................................................. 16 
8.2.5. 
Cumulative Incidents of CDA and CR at each Visit ................................................. 17 
8.2.6. 
Time to first CDA, CR, End of Consolidation Phase, and Relapse .......................... 17 
8.2.7. 
Cumulative CS Usage ............................................................................................... 18 
8.2.8. 
Mean CS Dose on the Day Before Each Visit ........................................................... 19 
8.2.9. 
PDAI, ABSIS, ABQOL and TABQOL scores ............................................................ 19 
8.2.10. 
Simple Nutritional Appetite Questionnaire (SNAQ) score .................................... 20 
8.2.11. 
Subgroup Analysis ................................................................................................. 20 
9. 
SAFETY ANALYSIS ..................................................................................................... 22 
9.1. ADVERSE EVENTS ............................................................................................................. 22 
9.1.1. 
Incidence of Treatment Emergent Adverse Events ................................................... 22 
9.1.2. 
Relationship of Adverse Events to Study Drug ......................................................... 22 
9.1.3. 
Intensity Grade of Adverse Event ............................................................................. 23 
9.1.4. 
Serious Adverse Events ............................................................................................. 23 
9.1.5. 
Adverse Events Leading to Treatment Discontinuation............................................ 23 
9.1.6. 
Death ......................................................................................................................... 23 
9.2. CLINICAL LABORATORY EVALUATIONS ........................................................................... 23 
9.3. VITAL SIGN MEASUREMENTS ........................................................................................... 24 
9.4. PHYSICAL EXAMINATION ................................................................................................. 25 
9.5. ELECTROCARDIOGRAM ..................................................................................................... 25 
9.6. FEMALE REPRODUCTIVE STATUS AND PREGNANCY TEST RESULTS ................................. 25 
9.7. FOOD INTAKE STATUS ...................................................................................................... 25 
9.8. BRAIN MRI ...................................................................................................................... 25 
10. 
PHARMACOKINETICS/ PHARMACODYNAMICS ............................................... 26 
11. 
INTERIM ANALYSIS ................................................................................................... 27 
12. 
SAFETY MONITORING COMMITTEE ................................................................... 27 
13. 
CHANGES IN THE PLANNED ANALYSIS .............................................................. 27 
14. 
REFERENCES ................................................................................................................ 28 
15. 
APPENDICES ................................................................................................................. 29 
15.1. 
SCHEDULE OF ASSESSMENTS ......................................................................................... 29 
15.2. 
GENERAL DOSE ADJUSTMENT GUIDELINES FOR DOSE SELECTION IN THE FIRST 4 WEEKS
 
31 
15.3. 
CS RESCUE CRITERIA AND TREATMENT PROTOCOL ....................................................... 32 
15.4. 
MISSING DATE IMPUTATION .......................................................................................... 34 
15.4.1. 
Missing medication and AE stop dates imputation ............................................... 34 
15.4.2. 
Missing medication start dates and AE onset dates imputation ............................ 34 


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 IV 
15.5. 
PEMPHIGUS DISEASE ACTIVITY AND QUALITY OF LIFE EVALUATION INSTRUMENTS: 
ABQOL, TABQOL, PDAI, ABSIS ........................................................................................... 36 
15.6. 
SIMPLE NUTRITIONAL APPETITE QUESTIONNAIRE (SNAQ) .......................................... 43 
15.7. 
CS TAPER PROTOCOL ..................................................................................................... 44 


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List of Abbreviations 
 
ABSIS 
Autoimmune Bullous Skin Disorder Intensity Score 
ABQOL 
Autoimmune Bullous Diseases Quality of Life (assessment) 
AE 
Adverse event 
ALT 
Alanine Aminotransferase or SGPT 
ALP 
Alkaline Phosphatase 
AST 
Aspartate Transaminase or SGOT 
AUC 
Area under the plasma concentration-time curve 
BCR 
B-cell receptor 
BID 
Twice daily (morning and evening) 
BMI 
Body mass index 
BPM 
Beats per minute for Heart Rate; Breaths per minute for Respiratory Rate 
BTK 
Bruton’s Tyrosine Kinase 
CDA 
Control of Disease Activity 
CI 
Confidence Interval 
Cmax 
Maximum observed plasma concentration 
CPK 
Creatine phosphokinase 
CR 
Clinical Response 
CRF 
Case report form 
CS 
Corticosteroid(s) 
CTCAE 
Common Terminology Criteria for Adverse Events 
DILI 
Drug Induced Liver Injury  
DSG 
Desmoglein 
EADV 
European Academy of Dermatology and Venereology 
ECG 
Electrocardiogram 
ELISA 
enzyme-linked immunosorbent assay 
FcJR 
Fc-gamma receptor 
FcHR 
Fc-epsilon receptor 
FSH 
Follicle Stimulating Hormone 
HIV 
Human Immunodeficiency Virus 
LPLV 
Last participant last visit  
MedDRA 
Medical Dictionary for Regulatory Activities  
N 
Sample Size 
PBMC 
Peripheral Blood Mononuclear Cell 
PD 
Pharmacodynamic 
PDAI 
Pemphigus Disease Area Index 
PK 
Pharmacokinetic 
PV 
Pemphigus vulgaris 
Q12H 
Every 12 hours 
QD 
Once a day 
QTc 
QT interval corrected for heart rate 
SAE 
Serious adverse event 
SI 
Système international d’unités (International system of units) 
SMC 
Safety Monitoring Committee 
SNAQ 
Simplified Nutritional Appetite Questionnaire 


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 VI 
TABQOL 
Treatment of Autoimmune Bullous Diseases Quality of Life (assessment) 
TBL 
Total Bilirubin 
TEAE 
Treatment-emergent AEs 
Tmax 
Time of observed maximum plasma concentration 
TSH 
Thyroid stimulating hormone 
ULRR 
Upper limit of the Reference Range  
WHODD 
World Health Organization Drug Dictionary 


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1. Introduction 
Pemphigus vulgaris (PV), which is characterized by intraepidermal blisters in skin and mucosae, 
is known to be driven by autoantibodies to epidermal proteins and is responsive acutely to the 
anti-inflammatory effects of CSs and within five to 35 weeks to B cell depletion by anti-CD20 
therapy (Horváth et al. 2012). 
Bruton’s agammaglobulinemia tyrosine kinase (BTK) is an essential signaling element 
downstream of the B-cell receptor (BCR), Fc-gamma receptor (FcJR) and Fc-epsilon receptor 
(FcHR). A selective BTK inhibitor has the potential to target multiple pathways involved in 
inflammation and autoimmunity. These include modulation of BCR-mediated B-cell pathways, 
inhibition of FcJR-induced cytokine release from monocytes and macrophages, FcHR-induced 
mast cell degranulation, granulocyte migration and mediator release. These effects lead to the 
prediction that a BTK inhibitor could block the initiation and propagation of various 
inflammatory diseases and mitigate the resulting tissue damage. 
PRN1008 is a novel, highly selective, small molecule inhibitor of non-T cell white blood cell 
signaling (via B cell receptor, FcJR, FcHR signaling of the Bruton’s Tyrosine Kinase, i.e. BTK, 
pathway). In rodent models of autoimmune arthritis, PRN1008 rapidly reverses disease with 
maximal efficacy similar to dexamethasone. In naturally occurring canine pemphigus foliaceus, 
an oral BTK inhibitor sharing an identical kinase inhibition profile to PRN1008 (PRN473) can 
rapidly reverse disease without the need for CS treatment. 
To date, PRN1008 has been administered orally to 98 healthy volunteers in four Phase 1 studies 
(PRN1008-001, PRN1008-002, PRN1008-004, and PRN1008-006). No clinically significant 
changes in laboratory parameters or in vital signs or ECG parameters were observed in 97/98 
patients tested. In Study PRN1008-001, healthy volunteers received 10 or 11 days of dosing, at 
dose regimens of placebo, 300 mg qd, 300 mg q12h, 600 mg qd, and 450 mg q12h (n=8 per dose 
level), with a liquid formulation, under fasted conditions on all but one day. PRN1008 was safe 
and well tolerated at the dose level tested. No severe or serious adverse events (SAEs) were 
reported, and no patients required discontinuation or interruption of dosing. Reported adverse 
events were mild or moderate in severity, with gastrointestinal adverse events being most 
common (nausea/vomiting, loose stools). Study PRN1008-006 compared the relative 
bioavailability of the liquid formulation to a tablet formulation, whereby patients (n=12) received 
300 mg single doses of both PRN1008 formulations, as well as the tablet after food and after 
several days of esomeprozole treatment. The tolerability was improved with the tablet compared 
with the liquid formulation, primarily by the elimination of acute throat irritation reported with 
the liquid formulation, and elimination the most common of gastrointestinal adverse events (AEs) 
reported in PRN1008-001 study when the tablet was administered following food.  
Clinical pharmacokinetics showed that with therapeutic multiple dosing, the terminal half-life of 
PRN1008 is approximately 3 to 4 hours and the time of maximum concentration (Tmax) is 1.5 to 
2.0 hours. There is no effect of food on the AUC or Cmax of the tablet formulation although the 
Tmax is slightly delayed from 1.5 hours to approximately 2.5 hours. The volume of distribution is 
high (on the order of 2500 L) and plasma protein binding is 98%. 


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Following single and multiple oral doses of PRN1008 in Study PRN1008-001, mean BTK 
occupancy in peripheral blood mononuclear cells (PBMCs) of more than 70% was observed 4 
hours or later following doses of 300 mg or more on Day 1. The target trough BTK occupancy 
levels were safely and consistently exceeded, suggesting PRN1008 may be a safe and highly 
effective treatment in human PV and other autoimmune diseases. 


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2. Objectives 
2.1. Primary Objectives 
x To evaluate the safety of PRN1008 in patients with PV 
x To evaluate the clinical activity of PRN1008 in patients with PV, per criteria in the European 
Academy of Dermatology and Venereology (EADV) 2014 Pemphigus S2 Guideline (Hertl et 
al. 2015) 
2.2. Secondary Objective 
x To evaluate the pharmacokinetics (PK) and the pharmacodynamics (PD) of multiple doses of 
PRN1008 in patients with PV 
2.3. Exploratory Objective 
x To evaluate the relationship of PK and PD to each other and to efficacy and safety in this 
patient population 


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3. Investigational Plan 
3.1. Overall Study Design and Plan 
This is a Phase 2, open-label, pilot cohort study to evaluate the safety, clinical activity, 
pharmacodynamics, and pharmacodynamics of PRN1008 in patients with newly diagnosed or 
relapsing PV. An open-label design was chosen for several reasons: PV does not spontaneously 
remit so there is no placebo effect to account for; clinicians need to know when to use rescue 
CSs, if necessary, in a timely manner; and lastly, the pilot nature of the investigation itself is best 
explored in an open-label fashion. 
Up to 28 days before enrollment in the study, patients will be required to sign an informed 
consent form, after which screening assessments will be carried out. Patients must fulfill all entry 
criteria to be enrolled into the study. Patients who fail to meet the entry criteria may be 
rescreened at the discretion of the Investigator. Up to 25 male or female patients with newly 
diagnosed (i.e., naïve to an effective induction treatment regimen) or relapsing, biopsy-proven, 
mild or moderate PV patients (Pemphigus Disease Activity Index [PDAI] 8 to 45) will be 
enrolled in the study. Enrolled patients will receive twice daily (bid) PRN1008 treatment for 12 
weeks, starting on Day 1 and ending on study Day 84, followed by 12 weeks of follow up (total 
duration of individual patient participation is 28 weeks). The expected study duration is 
approximately 18 months from the first patient treated to the last patient completing the study. 
The end of the study is defined as the date of the final safety follow-up after the last participant 
last visit (LPLV). 
Based on available data from the Phase 1 clinical studies, a 400 mg bid oral dose of PRN1008 is 
expected to be safe and well tolerated. Initial PRN1008 dosing in this study will be 400 mg bid, 
with intra-patient dose-adjustment based on clinical response and tolerability, supplemented by 
BTK occupancy. CS rescue treatment will be initiated, if indicated. The maximum dose in this 
study, after dose adjustment, will be 600 mg bid. Clinical response and tolerability will be 
assessed at each visit.  
Patients will complete the visit procedures per the Schedule of Assessment (Appendix 15.1). 
After providing written informed consent, patients will complete the screening assessments 
within 28 days before the first dose of PRN1008. Patients will return at specified times on an 
outpatient basis for assessment of vital signs, physical examination, assessment of adverse events, 
assessment of concomitant medication use, assessment of clinical benefit, and provision of blood 
samples for PK and PD, and other clinical laboratory tests. Height and weight, full physical 
examination, 12-lead electrocardiogram (ECG) (an extra ECG may be taken at additional visits, 
if indicated), FSH for postmenopausal women who are not surgically sterile, and some clinical 
laboratory testing (HIV, hepatitis B, hepatitis C, TB) will be taken at screening only. Skin biopsy 
will be also collected at screening if not already performed. During study drug treatment and 
follow up visit, Disease activity scores (PDAI and Autoimmune Bullous Skin Disorder Intensity 
Score [ABSIS]), Autoimmune Bullous Diseases Quality of Life (ABQOL) assessment, 
Treatment of Autoimmune Bullous Diseases Quality of Life (TABQOL) assessment, and Simple 
Nutritional Appetite Questionnaire (SNAQ) will be performed. Patient PDAI and ABSIS scores 
at screening will be collected as part of the medical history review. Brain MRI with macrocyclic 
contrast will be conducted at baseline and the Day 15 visit for patients from French sites. If 


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headache, nausea, vomiting or visual disturbances have occurred, a full neurological examination, 
including cranial nerves, will be performed for patients from French sites. 
The first PRN1008 dose on Day 1 of Week 1 will be supervised in the clinic and the patients will 
under observation for approximately two hours after administration of the first dose and until 
PK/PD sample is drawn. BTK occupancy in PBMCs will be measured at baseline (pre-dose), and 
2 and 24 hours after the first dose, then at various times of the day during follow up visits. For 
the Day 2 (24 hour) BTK occupancy, PRN1008 should be taken as usual in the evening and the 
PRN1008 morning dose withheld in order to take the blood sample approximately 12 hours after 
the prior evening dose. Where follow-up is not feasible on Day 2, another day in the first week of 
therapy may be used to get trough occupancy instead. For all other visits, occupancy is measured 
at random times after the usual morning dose. Anti-desmoglein-1 and -3 autoantibody titers will 
be measured by enzyme-linked immunosorbent assay (ELISA). 
3.2. Study Endpoints 
3.2.1. Safety Endpoints 
The incidence of treatment-emergent AEs (TEAEs), including clinically significant changes in 
physical examination, laboratory tests, and vital signs. 
3.2.2. Primary Efficacy Endpoint 
The primary efficacy endpoint is the proportion of patients who are able to achieve control of 
disease activity (CDA) within 4 weeks of starting PRN1008 treatment without the need for doses 
of prednis(ol)one >0.5 mg/kg at any time on or before the Week 5 visit.  
CDA is defined as the time at which new lesions cease to form and established lesions begin to 
heal per European Academy of Dermatology and Venereology (EADV) 2015 Pemphigus S2 
Guideline. 
3.2.3. Secondary Efficacy Endpoints  
x Proportion of patients able to achieve CDA without CSs within 4 weeks 
x Proportion of patients able to achieve a complete response (CR) without CSs within 12 
weeks 
x Proportion of patients able to achieve CR without the need for doses of prednis(ol)one of 
greater than 0.5 mg/kg at any point within 12 weeks 
x Proportion of patients able to achieve CDA at each visit 
x Proportion of patients able to achieve CDA at each visit with or without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
x Cumulative incidence of patients ever achieved CDA by each visit 
x Cumulative incidence of patients ever achieved CDA by each visit with or without the need 
for doses of prednis(ol)one of greater than 0.5 mg/kg 


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x Proportion of patients able to achieve CR at each visit 
x Proportion of patients able to achieve CR at each visit with or without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
x Cumulative incidence of patients ever achieved CR by each visit 
x Cumulative incidence of patients ever achieved CR by each visit with or without the need for 
doses of prednis(ol)one of greater than 0.5 mg/kg 
x Time to first CDA 
x Time to first CR 
x Time to first end of consolidation phase 
x Time to first relapse after PRN1008 treatment completion or discontinuation 
x Cumulative CS usage over 12 weeks 
x Change from baseline in Pemphigus Disease Area Index (PDAI) and Autoimmune 
Bullous Skin Disorder Intensity Score (ABSIS) scores at each follow-up visit 
x Change from baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL) and 
Treatment of Autoimmune Bullous Diseases Quality of Life (TABQOL) scores at each 
follow-up visit 
x Change from baseline in appetite (SNAQ score) at each follow-up visit 
CR, End of consolidation phase, and Relapse as defined in European Academy of Dermatology 
and Venereology (EADV) 2015 Pemphigus S2 Guideline are listed below: 
Complete response (CR) is defined as the absence of new or established lesions while the patient 
is receiving minimal or no non-PRN1008 immunotherapy. In this study, “minimal therapy” is 
defined as daily doses of ≤ 10 mg of prednisolone or equivalent doses of other CS for any period 
of time. 
End of consolidation phase: The time at which no new lesions have developed for minimum of 2 
weeks, approximately 80% of lesions have healed, and when most clinicians start to taper 
steroids. 
Relapse/flare: Appearance of ≥3 new lesions/month that do not heal spontaneously within 1 
week, or by extension of established lesions, in a patient who has achieved disease control. 
3.2.4. PK/PD Endpoints 
3.2.4.1. 
PK Endpoints 
x Plasma concentrations of PRN1008 at approximately the time of maximum concentration on 
Day 1 and at various subsequent times during outpatient dosing 


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3.2.4.2. 
PD Endpoints 
x Percentage BTK occupancy for individuals in PBMCs at 2 & 24 hours after the first 
PRN1008 dose and at varied subsequent times during outpatient dosing 
x Change from baseline in anti-dsg1-3 autoantibody levels by ELISA at various time points 
3.2.4.3. 
Exploratory PK/PD Analysis 
x Exploratory PK/PD analysis will examine the effects, if any, of covariates on PK and/or PD, 
and the relationship between PK, PD and efficacy in this population. 
3.3. Treatments  
PRN1008 Dosage Initial PRN1008 dosing will be 400 mg bid. The maximum dose in this study, 
after dose adjustment, will be 600 mg bid. Patients will be treated with PRN1008 for a maximum 
of 12 weeks. PRN1008 tablets should be taken with a glass of water and may be taken with or 
without food, i.e., a period of fasting is not required. 
Based on available data from clinical studies PRN1008-001 and PRN1008-002, a 400 mg bid 
oral dose of PRN1008 to be used in this study is expected to be safe and well tolerated. The 400 
mg bid starting dose is also based upon the dose known to produce ~70% BTK occupancy at 
trough (~85% average occupancy over the day), as adjusted by results of the relative 
bioavailability study, where the tablet had ~70% of the exposure of the equal dose of the liquid 
formulation. 
Each PRN1008 film-coated tablet contains either 100 mg or 300 mg of PRN1008 drug 
substance. In addition, the tablet contains Microcrystalline Cellulose (filler), Crospovidone 
(disintegrant), Sodium Stearyl Fumarate (lubricant) and a non-functional film coating. The 100 
mg tablet is a round shape and orange in color. The 300 mg tablet is an oval shape and white in 
color. 
3.4. Dose Adjustment/Modifications 
Initial PRN1008 dosing will be 400 mg bid. Patients will return to the study center and complete 
the visit procedures per the Schedule of Assessment (Appendix 15.1). The investigator will 
review the patient’s clinical response, tolerability, and BTK occupancy to determine if a dose-
adjustment is needed (Appendix 15.2). The maximum dose in this study, after dose-adjustment, 
will be 600 mg bid. Doses may range from 100 mg bid to 600 mg bid in 100 mg bid increments 
(100 mg is the smallest tablet size). CS rescue treatment will be initiated, if indicated (Appendix 
15.3). 
“Well tolerated” is defined as the absence of Grade 3 or greater gastrointestinal AEs, or Grade 2 
non-gastrointestinal AEs, including liver function changes, related to PRN1008 therapy. 
Investigators should use their judgment of the risk-benefit of continuing therapy by weighing the 
extent of clinical response, and AEs potentially related to PRN1008 therapy. In general, 
PRN1008 should be stopped and conventional therapy started when tolerability is poor and 
clinical response is also suboptimal. 


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4. General Statistical Considerations 
Continuous data will be described using descriptive statistics (i.e. n, mean, standard deviation, 
median, minimum, and maximum). Categorical data will be described using the frequency and 
percentage in each category. For the summary statistics of all numerical variables unless 
otherwise specified, minimum and maximum will be displayed to the same level of precision as 
reported. Mean and median will be displayed to one level of precision greater than the data 
collected. Standard deviation / standard error will be displayed to two levels of precision greater 
than the data collected. P-values will be rounded to three decimal places. If a p-value is less than 
0.001 it will be reported as “<0.001”. If a p-value is greater than 0.999 it will be reported as 
“>0.999”.  
Due to the small sample sizes, all p-values derived from inferential analyses will be considered 
informative. In general, all significant testing will be two-sided at significance level 0.05. All 
tests will be made without adjustment for multiplicity or multiple comparisons. Two-sided 80% 
and 95% Confidence Intervals of the response rate for efficacy endpoints will be provided. 
When frequency are presented, the percentage will be suppressed when the count is zero in order 
to draw attention to the non-zero counts. A row denoted “Missing” will be included in count 
tabulations where specified on the shells to account for dropouts and missing values. The 
denominator for all percentages will be the number of patients within the analysis population of 
interest, unless otherwise specified. 
Unless otherwise specified, baseline will be defined as the last non-missing assessment prior to 
the first dosing of study drug.  
Patients will be identified in the listings by patient number. Patients will be grouped into dose 
levels according to the maximum dose they received during the 12 week treatment period and all 
data will be summarized based on dose levels as well as overall unless otherwise specified. If 
data are to be summarized by visit, the visit name collected on the CRF page will be used. All 
analyses will be conducted using SAS Version 9.4 or above. 
4.1. Sample Size 
Sample size for this pilot study was determined pragmatically by the number of patients required 
to determine a preliminary safety profile and a point estimate for the primary endpoint of 
efficacy. If the clinical response rate is 50%, 25 patients will result in an 80% CI of ±13%. 
Results from this pilot study will be used to design confirmatory clinical trials. Sample size will 
be reevaluated based on an interim analysis when approximately 6 patients have completed 4 
weeks of PRN1008 therapy. 
4.2. Randomization, Stratification, and Blinding 
Not applicable; the study is open-label. 


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4.3. Analysis Populations 
Four study populations will be defined: Screening Population, Safety Population, Efficacy 
Population, and Pharmacokinetic Population. 
4.3.1. Screening Population 
All patients who provide informed consent and have screening assessments evaluated for study 
participation are included in the Screening Population. 
4.3.2. Safety Analysis Population 
All patients who have received at least one dose of PRN1008 will be included in the safety 
analysis. The Safety Analysis Population will be defined for all safety analyses. 
4.3.3. Efficacy Analysis Population (ITT Population) 
All patients who have received at least one dose of PRN1008 will be included in the efficacy 
analysis. Patient response and disease progression will be determined using PDAI, ABSIS, 
ABQOL, and TABQOL scores. Efficacy data will be presented in listings, by patient and 
tabulated for each efficacy endpoint. 
4.3.4. mITT Population 
Patients with at least Week 5 visit follow up will be included in the mITT population. Patients 
who terminated therapy prior to the Week 5 visit on the first day of treatment without any further 
follow up are not included in this population. 
4.3.5. Per Protocol (PP) Population 
Patients who have  
1) No major protocol deviations relevant to data integrity per Medical Monitor review 
2) At least 80% compliance based on drug accountability 
3) Completed Week 13/Day 85 visit. 
 
4.3.6. Pharmacokinetic Analysis Population 
The definition of Pharmacokinetic Analysis Population will be described outside of this 
Statistical Analysis Plan. 


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5. Patient Disposition 
5.1. Disposition 
The number and percentage of patients in each analysis population defined in Section 4.3, the 
number and percentage of patients enrolled and patients screen failure will be reported overall 
and by maximum dose level.  
The number and percentage of patients completing, withdrawing, along with reasons for 
withdrawal, will be tabulated overall and by maximum dose level for the Safety Analysis 
Population. The percentage of reasons for withdrawal will be based on the number of patients 
who withdraw from the study. 
A listing of patient disposition data will be presented for the Safety Population, include informed 
consent date, study completion status, study completion/termination date, and reasons for 
withdrawal. 
Reasons for withdrawal are specified in the CRF as: Adverse Event, Death, Lack of Efficacy, 
Lost of Follow-up, Non-Compliance with Study Drug, Physician Decision, Pregnancy, 
Progressive Disease, Protocol Violation, Recovery, Study Terminated by Sponsor, Technical 
Problem, Withdrawal by Subject, and Other. 
 
5.2. Protocol Deviations 
Protocol deviations are any unanticipated or unintentional divergence or departure from the 
expected conduct of an approved clinical study that is not consistent with the protocol, ancillary 
study documents, study plans/guides, or Informed Consent Form. Protocol deviations will be 
categorized to major and non-major deviations. Major deviations are departures from the 
protocol that impact patient safety or data integrity. 
Major protocol deviation information will be documented and reviewed by the PPD project team 
and approved by the sponsor.  
The number and percentage of patients with major deviations will be summarized overall in a 
table for the Safety Analysis Population.  
All subject level protocol deviations will be listed for the Screening Population. Site level 
protocol deviations will be presented in the CSR separately. 


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6. Demographics and Baseline Characteristics 
6.1. Demographics 
A summary of demographics and baseline characteristics will be presented overall and by 
maximum dose level for the Safety Analysis Population/ITT Population, mITT Population and 
PP Analysis Population. The demographic characteristics consist of age (years), sex, race, and 
ethnicity. The baseline characteristics consist of baseline pemphigus (clinical diagnosis) type, 
time from pemphigus vulgaris confirmed date to screen date (Months), baseline Anti-DSG  1 and 
Anti-DSG  3 antibody status/profile, baseline height (cm), baseline weight (kg), baseline body 
mass index (BMI) (kg/m^2), PDAI total activity score, PDAI mucous membrane activity (% >0), 
PDAI scalp activity (% >0), PDAI skin activity (% >0), ABSIS oral activity (% >0), and CS dose 
on screening date and Week 1 Day 1 date, which is summarized in the CS summary table 
separate from the demographics summary.  
A patient’s age in years is calculated using the date of the informed consent and date of birth. 
Age, height, weight, BMI, PDAI total activity score, and CS Dose on the day of screening and 
Week 1 Day 1 will be summarized using descriptive statistics. The number and percentage of 
patients by sex (Male, Female), race (White, Black or African American, Asian, American 
Indian or Alaska Native, Native Hawaiian or Other Pacific Islander, and Other), ethnicity 
(Hispanic or Latino, Not Hispanic or Latino), baseline pemphigus type (Pemphigus Vulgaris, 
Bullous Pemphigoid, Pemphigus Foliaceus, Epidermolysis Bullosa Aquisita, Linear IgA Bullous 
Dermatoses, Mucous Membrane Pemphigoid, and Other). Time from pemphigus vulgaris 
confirmed date to screen date in months is defined as (Date of Screening – Date of pemphigus 
vulgaris confirmed +1) / 30.4375.  
Baseline Anti-DSG status will be presented in the following categories, using the limits of 
detection as the basis for positive/negative determination: 
x Anti-DSG 1 positive, Anti-DSG 3 negative; 
x Anti-DSG 1 negative, Anti-DSG 3 positive; 
x Anti-DSG 1 positive, Anti-DSG 3 positive; 
x Anti-DSG 1 negative, Anti-DSG 3 negative; 
No formal statistical analyses will be performed, and no inferential statistics reported. 
Subgroup analyses variables detailed in Section 8.2.11 will also be summarized by categories. 
Patient demographic and baseline characteristics will be presented in a listing for the Safety 
Analysis Population. 
6.2. Medical History 
Patient medical history data including specific details will be presented in a listing for the Safety 
Analysis Population. 


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6.3. Inclusion and Exclusion Criteria 
Patients who meet all of the protocol defined inclusion criteria and none of the exclusion criteria 
will be enrolled into the study. Inclusion/exclusion criteria data will be summarized and listed for 
the Screening Population. 


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7. Treatments and Medications 
7.1. Prior and Concomitant Medications 
All medications taken within 28 days prior to the date of study screening will be recorded in the 
CRF. Concomitant medication will be coded according to the World Health Organization Drug 
Dictionary (WHODD, version 01SEP2015). A prior medication is defined as any medication that 
has a stop date before the date of first dose of study drug. A concomitant medication is defined 
as any medication that has a stop date on or after the date of first dose of study drug. 
The number and percentage of patients who used prior or concomitant medications will be 
summarized in two separate tables by Anatomical Therapeutic Chemical (ATC) level 4 and 
preferred term, maximum dose levels and overall using the Safety Analysis Population.  
All prior and concomitant medication will be presented in a listing for the Screening Population. 
Imputation for missing medication start dates and stop dates can be found in Appendix 15.4. 
Medications with missing stop dates after imputation will be classified as both prior and 
concomitant. 
7.2. Study Treatments 
A 400 mg bid initial oral dose of PRN1008 will be used in this study, and dosage will be 
adjusted during the treatment of study drug. Patients will be treated with PRN1008 for a 
maximum of 12 weeks. Tablets may be taken with or without food. 
7.2.1. Extent of Exposure 
Overall duration of exposure is defined as the total number of days a patient is exposed to the 
study drug and will be presented as the total number of days from the first dose date (Day 1) to 
the last dose date: 
Overall Duration of Exposure = Date of last dose - Date of first dose + 1. 
If date of last dose date is missing, date of last visit during the treatment will be used. 
The average daily dose will also be introduced to characterize features of the dose adjustment 
design adopted in this study, which is calculated as the following: 
Average Daily Dose = Cumulative dose / Overall duration of exposure 
The cumulative dose will be defined as the sum of doses taken by patients during the treatment. 
The overall duration of exposure and average daily dose will be summarized using descriptive 
statistics overall and by maximum dose level in a table for all patients in the Safety Analysis 
Population.  
The number of adjustments in dose level and the minimum dose level will also be summarized.  


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Patient’s overall duration of exposure, average daily dose, minimum dose level, maximum dose 
level, and number of adjustments in dose will be presented in a listing for the Safety Population.  
7.2.2. Treatment Compliance and Modifications 
Study drug accountability will be performed during the treatment. Patients will be asked to bring 
back all remaining study drug and all study drug packaging at each study visit for drug 
accountability.  
The overall study drug compliance (%) will be calculated as following: 
Overall Compliance (%) = Total number of tablets taken during the treatment / Total number of 
tablets planned during the treatment * 100 
The total number of tablets taken will be calculated by subtracting the number of tablets returned 
from the number of tablets dispensed. The total number of tablets planned will be calculated by 
multiplying the total number of days of planned exposure to study drug by the number of tablets 
planned per day. 
The overall study drug compliance will be summarized by descriptive statistics overall and by 
maximum dose level. The number and percentage of patients in compliance category of <80%, 
>=80 and <=120%, and >120% will also be presented.  
Study drug dispensed and returned and study drug compliance will be presented in two listings 
by patient for the Safety Analysis Population.  


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8. Efficacy Analysis 
All efficacy analyses will be conducted using the ITT, mITT and PP Analysis Population.  
All efficacy analyses consider CS used in endpoints to be systemic CS only, i.e. oral (not 
mouthwash), intramuscular or intravenously administered. Topical CS are not considered 
systemic CS. 
For cumulative by visit summaries, unscheduled visits are not included in the summary.  For 
other by visit summaries, unscheduled visits, if exists in the data, will be presented separately 
after other visits. All visits will be presented in listings. 
 
8.1. Primary Efficacy Endpoint 
The primary efficacy endpoint is “The proportion of patients who are able to achieve CDA 
within 4 weeks of starting PRN1008 treatment without the need for doses of prednis(ol)one >0.5 
mg/kg at any time”. 
Other types of CS are converted to dose-equivalent prednis(ol)one (see Section 8.2.6). 
This is operationalized as meaning the first CDA is achieved on or before the Week 5 visit but 
after first dose of PRN1008, and will be summarized, by maximum dose level to the Week 5 
visit and overall in a table, using frequencies and percentages. In addition, two-sided 80% and 
95% CIs (Confidence Intervals) of the response rate will be provided for each maximum dose 
level and overall using the Exact (Clopper-Pearson) method.  
Patients who are able to achieve CDA between first dose of study drug and the Week 5 visit and 
have no occurrence of daily prednis(ol)one >0.5 mg/kg before the date of the first CDA will be 
considered as responders, even if the CDA status was not maintained or higher doses of 
prednis(ol)one were used after the first CDA. Patients who are not able to achieve CDA prior to 
or at the Week 5 visit or have any occurrence of daily prednis(ol)one >0.5 mg/kg before a first 
CDA during the study treatment will be considered as non-responders. Patients whose CDA 
status is unknown at the Week 5 visit for whatever reason (include early termination before 
Week 5 visit) will be also considered as non-responders. Only systemic CSs (as defined by 
medication taken orally or intravenously)are to be used to evaluate the efficacy endpoints, topical 
CSs will not be counted. 
8.2. Secondary Efficacy Endpoint 
8.2.1. Achieved CDA without CS within 4 Weeks 
x The proportion of patients who are able to achieve CDA without CSs within 4 weeks will 
be summarized in the same way as that described in Section 8.1. Patients who are able to 
achieve CDA by the Week 5 visit and have no occurrence of any CS on or before the first 
CDA visit during the study treatment will be considered as responders. Patients who are 
not able to achieve CDA by the Week 5 visit or have any occurrence of any CS on or 


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before a CDA visit during the study treatment will be considered as non-responders. 
Patients whose CDA status is unknown at the Week 5 visit for whatever reason (include 
early termination before Week 5 visit) will be also considered as non-responders. Only 
systemic CSs (as defined by medication taken orally or intravenously) are to be used to 
evaluate the efficacy endpoints, topical CSs will not be counted.   
8.2.2. Achieved CR without CS within 12 Weeks 
The proportion of patients who are able to achieve CR without CSs within 12 weeks will be 
summarized in the same way as that described in Section 8.1. Patients who are able to achieve 
CR between treatment start date and the Week 13 visit and have no occurrence of any CS after 
the treatment start date and before the first CR visit during the study treatment will be considered 
as responders. Patients who are not able to achieve CR between start of treatment and the Week 
13 visit or have any occurrence of any CS between start of treatment and a first CR visit during 
the study treatment will be considered as non-responders. Patients whose CR status is unknown 
at the Week 13 visit for whatever reason (include early termination before Week 13 visit) will be 
also considered as non-responders. Only systemic CSs (as defined by medication taken orally or 
intravenously) are to be used to evaluate the efficacy endpoints, topical CSs will not be counted.   
8.2.3. Achieved CR without Prednis(ol)one >0.5 mg/kg within 12 Weeks 
The proportion of patients who are able to achieve CR without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg within 12 weeks will be summarized in the same way as 
that described in Section 8.1. Patients who are able to achieve CR at any visit on or before the 
Week 13 visit during the study treatment and have no occurrence of daily prednis(ol)one >0.5 
mg/kg between the first dose date of PRN1008 treatment and the day before the date of the first 
CR will be considered as responders. Patients who are not able to achieve CR at all visits on or 
before the Week 13 visit during the study treatment or have any occurrence of daily 
prednis(ol)one >0.5 mg/ between the first dose date of PRN1008 treatment and the day before 
the date of the first CR will be considered as non-responders. Patients whose CR status is 
unknown at all visits on or before Week 13 visit during the study treatment for whatever reason 
(include early termination before Week 13 visit) will be also considered as non-responders. Only 
systemic CSs (as defined by medication taken orally or intravenously) are to be used to evaluate 
the efficacy endpoints, topical CSs will not be counted.   
8.2.4. Proportion of CDA and CR at each Visit  
The proportion of patients who are able to achieve CDA and the proportion of patients who are 
able to achieve CR will be summarized by maximum dose level and overall by CDA and CR 
status for each visit by using frequencies and percentages, respectively. This is a cross sectional 
analysis and differs from the cumulative assessment of efficacy described above. In addition, 
two-sided 80% and 95% CIs of the response rate will be provided for each maximum dose level 
and overall using the Exact (Clopper-Pearson) method. 
The proportion of patients who are able to achieve CDA and/or CR without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg (CS doses from Day 1 to the day before the date of the 
visit) will also be summarized by maximum dose level and overall for each visit. Two-sided 80% 
and 95% CIs will be provided by using the Exact (Clopper-Pearson) method. 


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The disease status at each visit, and the CS dose on the day before the achievement date will be 
listed. 
8.2.5. Cumulative Incidents of CDA and CR at each Visit  
The cumulative incidents of patients who are able to achieve CDA and CR by each visit will be 
summarized by maximum dose level and overall. This is a cumulative analysis compared to 
section 8.2.4. In addition, two-sided 80% and 95% CIs of the response rate will be provided for 
each maximum dose level and overall using the Exact (Clopper-Pearson) method. 
The proportion of patients who are able to achieve CDA and/or CR without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg (CS doses from Day 1 to the day before the date of the 
visit) will also be summarized by maximum dose level and overall for each visit. Two-sided 80% 
and 95% CIs will be provided by using the Exact (Clopper-Pearson) method. 
If a subject drops out, they will still accommodate to future unattended visits cumulatively, i.e. 
the denominator for this analysis would not diminish over time. 
8.2.6. Time to first CDA, CR, End of Consolidation Phase, and Relapse 
Time to first CDA is defined as the following: 
Time to first CDA = Date of the first CDA confirmed - Date of first dose + 1. 
Time to first CR and End of Consolidation Phase are defined in the same way as Time to first 
CDA. 
Time to first relapse after PRN1008 treatment completion or discontinuation is defined as the 
following: 
Time to first Relapse = Date of the first relapse after Week 13 visit confirmed – Date of last 
PRN1008 dose + 1. 
If the date of last PRN1008 dose is missing, the date of the Week 13 visit during treatment will 
be used. If patients did not complete the Week 13 visit they will be excluded from the relapse 
analysis. 
The time to relapse analysis will be conducted in two subpopulations for each analysis 
population: 1) Patients with CDA at the Week 13 visit, 2) Patients with CR at the Week 13 visit. 
Patients who have not achieved the above events by the date of study completion or 
discontinuation will be censored at the date of completion or discontinuation respectively. 
Kaplan-Meier survival distribution plots for time to each event will be provided. Kaplan-Meier 
estimates of quartiles in days and its associated 80% and 95% CI will be calculated by using the 
LOGLOG transformation. Min and Max survival times in days as well as number of the patients 
and the probability of experiencing the event on or before specific date will be provided in a 
table. 


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The CDA status, CR status, End of Consolidation Phase status, and Relapse status by visit, by 
patient will be presented in a listing.  
Duration of CR will be summarized and listed, and is defined by the last known CR date – first 
known CR date + 1. Each subject will only have one CR date as a result. 
8.2.7. Cumulative CS Usage 
Cumulative corticosteroid (CS) usage per patient (mg) over 12 weeks will be presented overall 
and by maximum dose level in a table.  Only subjects who complete at least 12 weeks of 
treatment (terminated at or after the Week 13 (Day 85) Visit) will be included. To calculate this 
endpoint, the following steps will be followed: 
x Step 1: All other CSs will be converted into prednisolone/prednisone dosage equivalent. 
Each 10 mg of prednisolone is equivalent to 10 mg of prednisone, 8 mg of 
methylprednisolone, 1.5 mg dexamethasone, 40 mg hydrocortisone or 1.5 mg 
betamethasone. 
x Step 2: The CSs usage will be converted to standard frequency using the following 
conversion chart, the standard frequency is in the first column (i.e., mg/day).  
Standard frequency 
Unit 
Frequency 
Conversion factor 
mg/day 
mg 
QD 
1 
mg/day 
mg 
BID 
2 
mg/day 
mg 
QOD 
0.5 
mg/day 
mg/day 
Any value 
1 
mg/day 
mg 
ONCE 
1 
mg/day 
mg 
Q24H 
1 
mg/day 
mg 
TID 
3 
mg/day 
mg 
QS 
0.1428571 
mg/day 
mg 
QID 
4 
Once the CSs type and frequency are converted as above, each record of CSs taken will 
be converted to prednisolone with standard frequency (mg/day). 
x Step 3: The duration for each record of CSs (CS) taken will be calculated using the 
formula: Duration = CS end date – CS start date + 1, in days. Any missing start or end 
date of CSs taken will be imputed using the algorithm specified in Section 15.4. 
o If the CS end date is after the date of Week 13 (Day 85) - 1, then the CS end date 
will be replaced by the date of Week 13 (Day 85) - 1; if the CS end date is before 
the study treatment start date, this record will not be used to summarize 
cumulative dose for CSs.  
o If the CS start date is before the study treatment start date, then the CS start date 
will be replaced by the study treatment start date (i.e. Day 1); if the CS start date 
is after the date of Week 13 (Day 85) - 1, this record will not be used to 
summarize cumulative dose for CSs. 


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o Note that above date adjustments only apply to cumulative CS dose calculation. 
Other analyses, where CS dose is needed, will use CS start and end dates as 
reported.  
x Step 4: The “record” level cumulative CSs usage will be calculated as: record level 
cumulative dose = standard frequency * duration (days). 
x Step 5: The patient level cumulative CSs usage will be calculated as the summation of all 
the non-missing record level cumulative doses. 
All CS usage will be presented in a listing showing CS type, dose, frequency, route, start and 
stop dates.   
 
8.2.8. Mean CS Dose on the Day Before Each Visit 
For each subject on the day before each scheduled visit, dosage level of Predniso(lo)ne in mg 
will be summarized using summary statistics by maximum dose level and overall.  
 
For Week 1/Day 1 visit, however, the CS dose is set as the day of, rather than the day before. 
 
If a subject is still enrolled in the study but does not report any CS, then the dosage level is set as 
0. If a subject is terminated prior to a visit, then this subject is not included in the calculation for 
visit. 
 
8.2.9. PDAI, ABSIS, ABQOL and TABQOL scores 
Quantitative assessment of patient response and disease progression will be determined using 
PDAI, ABSIS, ABQOL, and TABQOL scores. The PDAI, ABSIS, ABQOL and TABQOL 
questionnaires are located in Appendix 15.5. 
For each questionnaire, we assume they are taken on the day of visit dates, and use the visit date 
indicated on the Visit Date CRF as questionnaire date, as the latter are not rigorously QC’ed.  
The PDAI questionnaire has two components including activity and damage. The activity 
component consists of skin, scalp and mucosa parts and the damage component consists of skin 
and scalp parts. Actual values and change from baseline in PDAI total activity, total damage 
scores, and total PDAI score will be summarized by maximum dose level and overall, 
respectively. 
The PDAI activity or damage total scores per body part will be calculated as the sum of the 
scores on that body part as follows. PDAI total activity and total damage scores are calculated 
and reported by site investigator into EDC. 
x PDAI total activity score = Total skin activity + Total scalp activity + Total mucosa activity 
x PDAI total damage score = Total skin damage + Total scalp damage 


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The ABSIS questionnaire has three components, including skin involvement, oral involvement 
and (oral) severity. The ABSIS scores will be calculated separately for each component. The 
ABSIS total activity score and total ABSIS score will also be calculated as follows.  
x Skin involvement total score = sum of (%BSA * Weighting factor)  
x Oral involvement total score = sum score from each oral part 
x (Oral) Severity total score = sum of (Level * Factor of discomfort ) 
x ABSIS total activity score = Skin involvement total score + Oral involvement total score 
x Total ABSIS score = Skin involvement total score + Oral involvement total score + Severity 
total score 
Actual values and change from baseline in ABSIS skin involvement total score, ABSIS oral 
involvement total score and ABSIS total activity score will be summarized for all patients, and 
for patient subgroups categorized into two groups by a baseline oral involvement total score 
equal to 0 and >0, and for patient subgroups categorized into two groups by a baseline skin 
involvement score equal to 0 or >0.  
When calculating skin involvement total score, if sum of %BSA = 0, then skin involvement 
score component is 0. If %BSA > 0, then weighting factor needs to be considered, and if 
weighting factor is missing in this circumstance, then the skin involvement total score and 
ABSIS total score are considered missing.  
The ABQOL and TABQOL questionnaires both contains 17 questions with numerical scores. 
The ABQOL and TABQOL scores will be calculated as the sum of scores from each question. 
Actual values and change from baseline in ABQOL and TABQOL scores will also be 
summarized overall and by maximum dose level in a table.  If individual scores are missing, then 
total scores as set as missing. 
PDAI, ABSIS, ABQOL and TABQOL scores and subscores (for PDAI and ABSIS) by patient 
and visit will be presented in separate listings. 
8.2.10. Simple Nutritional Appetite Questionnaire (SNAQ) score 
Simple Nutritional Appetite Questionnaire (SNAQ) contains 4 questions with numerical scores. 
The SNAQ score will be calculated as the sum of scores from each question. The appetite 
questionnaire is located in Appendix 15.6. If individual scores are missing, then total scores as 
set as missing. 
Actual values and change from baseline in SNAQ score will be summarized by visit overall and 
by maximum dose level in a table. 
The SNAQ score will be presented in a listing by patient and visit. 
8.2.11. Subgroup Analysis 
The rates of the primary endpoint will be calculated in selected subgroups: 


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1. Age (≥ 50, < 50) 
2. Gender 
3. Total anti-desmoglein (dsg) antibody (≥ 100, < 100 units) at baseline. 
4. Pemphigus History Type: Newly diagnosed (< 6 months from screening) vs. relapsed 
(> 6 months) 
5. Pemphigus Anti-DSG Profile at baseline: PV vs. PF vs. negative anti-dsg profile, 
where PV is defined as anti-dsg3 positive with anti-dsg1 negative or anti-dsg 3 
positive with anti-dsg1 positive. PF=anti-dsg1 positive only vs. neither detectable).  
6. Pemphigus Severity: Mild vs. moderate disease severity at baseline (PDAI total 
activity < 15 vs. ≥ 15). If screening PDAI is not available use Day 1. 
 


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9. Safety Analysis 
Specific assessments to evaluate treatment safety include the following: the frequency and type 
of adverse events, clinical laboratory testing, brain MRI (baseline and Day 15 in a subset of 
patients), vital signs, and symptoms and/or signs of headache, nausea, vomiting or visual 
disturbances. All analyses of safety will be conducted using the Safety Analysis Population. 
For by visit summaries, unscheduled visits, if they exist in the data, will be presented separately 
after other visits. 
9.1. Adverse Events 
An AE can be any unfavorable and unintended sign (including an abnormal laboratory finding, 
for example), symptom, or disease temporally associated with the use of an investigational 
product, whether or not considered related to the product. All AEs encountered during the study 
will be reported in detail in the CRF, begins at the time of the first screening/eligibility 
assessment and ends at the end of the study for each patient. Pre-existing conditions that worsen 
during a study are to be reported as AEs. Clinically significant changes in physical examination, 
laboratory safety tests, and vital signs will also be defined as AEs and recorded appropriately. 
All AEs will be coded using the MedDRA Dictionary (MedDRA, Version 18.0). 
Imputation for missing AE onset dates and end dates can be found in Appendix 15.4. 
A treatment-emergent AE (TEAE) is defined as an AE that begins on or after the first dose of 
study drug.  
If the AE onset date is missing after imputation and the imputed end date is on or after the first 
dose of study drug, or if onset date and end date are both missing, then the AE will be classified 
as a TEAE. If the imputed AE onset date is the same as the first dose of study drug, and AE 
onset time on or after the first dose of study drug or AE onset time is missing, then the AE will 
also be classified as a TEAE. 
An overall summary of the number and percentage of patients with any TEAE, serious TEAE, 
study drug-related TEAE, study drug-related serious TEAE, TEAE leading to treatment 
discontinuation, and AEs leading to death will be provided overall and by maximum dose level.  
All AEs will be presented in a listing for the Safety Population. 
9.1.1. Incidence of Treatment Emergent Adverse Events 
The number and percentage of patients with at least one TEAE and the number of TEAEs will be 
summarized by SOC and PT, maximum dose level and overall. At each level of SOC or PT, a 
patient will be counted only once if the patient reported multiple events.  
9.1.2. Relationship of Adverse Events to Study Drug 
The investigator will provide an assessment of the relationship of the event to the study drug. 
The possible relationships defined in the protocol are “Not Related” and “Related”.  


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The TEAEs categorized by SOC and PT will be summarized by maximum dose level and 
relationship to study drug in a manner similar to that described in Section 9.1.1. 
TEAEs that are missing a relationship will be presented in the summary table as “Related” but 
will be presented in the data listing with a missing relationship. 
Related TEAEs will also be presented by SOC and PT.  
9.1.3. Intensity Grade of Adverse Event 
The NCI CTCAE (version 4.0) will be used for grading AEs.  
The TEAEs categorized by SOC and PT will be summarized by maximum dose level and 
intensity grade in a manner similar to that described in Section 9.1.1. Per each SOC or PT, a 
patient will be counted only once at the highest grade of intensity. 
TEAEs with missing intensity grade will be presented in tables as “Grade 3” but will be 
presented in the data listing with a missing severity. 
9.1.4. Serious Adverse Events 
The seriousness of an AE should be assessed by the Investigator independently from the severity 
of the AE.  
The treatment-emergent SAEs categorized by SOC and PT will be summarized by maximum 
dose level in a manner similar to that described in Section 9.1.1. Treatment-emergent SAEs by 
relationship to study drug will also be presented in a table. SAE information by patient will be 
presented in a listing. 
9.1.5. Adverse Events Leading to Treatment Discontinuation 
The TEAE leading to treatment discontinuation will be summarized by SOC and PT, and 
maximum dose level in the same way as that described in Section 9.1.1. 
All patients who have an AE leading to treatment discontinuation will be presented in a listing. 
9.1.6. Death 
The TEAE leading to death will be summarized by SOC and PT by SOC and PT, maximum dose 
level in the same way as that described in Section 9.1.1. 
All patients who have an AE with an outcome of death will be presented in a listing.  
9.2. Clinical Laboratory Evaluations 
Laboratory assessments will be performed at a central laboratory, with the provision for 
occasional local laboratory testing, if required. The International System of Units (SI units; 
Système international d’unités) will be used and those not reported in SI units will be converted 
to SI units before analysis. 


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Summary tables presenting actual values and changes from baseline will be presented for 
chemistry tests, hematology tests, and coagulation tests overall and by maximum dose level. All 
relevant clinical laboratory tests will be flagged as Low, Normal, and High according to the 
normal ranges if applicable. Shift tables will be provided to summarize changes in normality 
status from baseline to each scheduled post-baseline visit for those tests with normality flags. 
The number and percentage of patients for urinalysis tests - will be presented in separate tables. 
This categorical data will be summarized in shift tables comparing the results at each scheduled 
post-baseline visit with those at baseline visit.  
All lab tests results with normality status will be presented by patient and visit in a listing for the 
Screening Population. 
A special group of clinical laboratory tests which are believed to be the indicators for drug 
induced liver injury (DILI) will be assessed separately with graphical displays and tabulations. 
They include: 
Ь 
ALT (alanine aminotransferase or SGPT) 
Ь 
AST (aspartate transaminase or SGOT) 
Ь 
TBL (total bilirubin) 
Ь 
ALP (alkaline phosphatase) 
The test results of the above clinical laboratory tests will be reported in a separate listing and the 
values outside a number of designated reference ranges will be marked. 
Furthermore, scatter plots of the peak TBL divided by the upper limit of the reference range 
(ULRR) (Y-axis) against peak ALT divided by the ULRR (X-axis), on a log scale, will be 
created. Lines for Upper limit of the Reference Range (ULRR) for both TBL and ALT, 2xULRR 
for TBL and 3xULRR for ALT will be presented in the same scatter plot graph. Any patients in 
the upper right quadrant, which is defined by 2xULRR for TBL and 3xULRR for ALT, would 
represent cases that were further investigated for potential DILI. 
9.3. Vital Sign Measurements 
Summary tables presenting observed values and changes from baseline will be provided overall 
for vital sign measurements, which including systolic blood pressure (mmHg), diastolic blood 
pressure (mmHg), heart rate (bpm), respiratory rate (bpm), and temperature (qC). All vital sign 
measurements will be presented in a listing by patient and visit for Safety Population. 
Systolic blood pressure values <90 or >140 mmHg or diastolic blood pressure values <60 or >80 
mmHg will be flagged as outside the normal range. Resting heart rate <40 or >100 beats per 
minute will be flagged as abnormal. The number and percentage of patients for each normality 
category will be summarized overall at each visit. 


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9.4. Physical Examination 
The number and percentage of patients for each body system will be summarized by normality 
status and overall at each visit. 
For the screening visit, the physical examination includes the following body systems: general 
appearance; skin; eyes, ears, nose, throat; heart; chest/breast; abdomen; neurological; lymph 
nodes; skeletal; and other. Physical examinations at all other visits will only include: general 
appearance; abdomen; cardiorespiratory; and other. 
Physical examination results for the safety analysis population will be presented in a listing.  
For patients from French sites only: if clinically significant headache, nausea, vomiting or visual 
disturbances have occurred, a full neurological examination, including cranial nerves, will be 
performed. The neurological symptoms will be documented on AE page. The status of 
neurological symptoms with relevant AE number, and results of neurological examination will 
be presented in separate listings. 
9.5. Electrocardiogram 
Summary tables will be presented for ECG data including ventricular rate (bpm), PR Interval 
(msec), QRS Duration (msec), QT Interval (msec), QTcF Interval (msec) and QTcB Interval 
(msec), overall and by maximum dose level for the screening visit. 
ECG interpretation at the screening visit will also be summarized overall and by maximum dose 
level.  
If ECG results are collected or interpreted multiple times, the latest one prior to dosing will be 
used for this summary. 
ECG results for the safety analysis population will be presented in a listing. 
9.6. Female Reproductive Status and Pregnancy Test Results 
The fertility status and method of birth control used for women will be recorded in the CRF at 
the screening visit. The pregnancy test will be performed for women of childbearing potential 
only. Pregnancy serum test will be done at the screening visit, urine dip tests will be done at 
other time points. The reproductive status as well as serum and urine pregnancy test results will 
be presented in a listing for the safety analysis population. 
9.7. Food Intake Status 
The food intake status will be recorded in the CRF at each visit. A listing of patient food intake 
status will be presented for the safety analysis population. 
9.8. Brain MRI 
Brain MRI with macrocyclic contrast will be conducted on baseline and Day 15 visit for patients 
from French sites. Date of scan and results will be presented in a listing for the safety analysis 
population. 


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10. Pharmacokinetics/ Pharmacodynamics 
The analyses of pharmacokinetics and pharmacodynamics (BTK occupancy) will be described 
and reported outside of the PRN1008-005 study report and attached as appendices. 
ITT Analysis Population will be used for TFLs below. 
The limit of quantifications for Anti-DSG 1, Anti-DSG 3 and Total Anti-DSG are 14, 9 and one 
or both of 14 or 9, respectively. Below the threshold is defined as negative, at or above these 
thresholds is defined as positive.  
For Anti-DSG 1 and Anti-DSG 3 data, and a calculated variable of “total DSG” (Anti-DSG 3 
and Anti-DSG 1 summed) summary tables presenting actual values and changes from baseline 
will be presented by visit overall and by maximum dose level. Missing values will not be 
imputed.  This analysis will be performed only using Anti-DSG 1 and Anti-DSG 3 data above 
the limit of quantification, and for Total Anti-DSG, only use samples where at least one of Anti-
DSG 1 or 3 is above the limit of quantification. Two exploratory DSG analyses will be 
performed: 
1) CR status (without more than 0.5 mg/kg of CS prior to Week 13) yes/no at the Week 13 
visit will be used to compare the change from baseline to Week 5 & 13 in Anti-DSG 1, 
Anti-DSG 3 data, and total DSG levels.  
2) CDA status (without more than 0.5 mg/kg of CS and as defined for the primary endpoint) 
yes/no before Week 5 visit will be used to compare the change from baseline to Week 5 
& 13 in Anti-DSG 1, Anti-DSG 3 data, and total DSG levels 
All pharmacokinetics and pharmacodynamics data, including plasma concentrations of PRN1008, 
percentage BTK occupancy in PBMCs, and Anti-DSG 1-3 autoantibody levels (including 
quantified values below limit of quantification), will be presented in listings. 
BTK occupancy data will also be described in a separate report. 


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11. Interim Analysis 
An interim analysis to evaluate safety, and to reevaluate sample size and other aspects of study 
design, was conducted for this study when 6 patients completed 4 weeks or more of PRN1008 
therapy (PRN1008-005 Interim Analysis Report March 20 2017), and again when 26 patients 
had completed 4 weeks or more of PRN1008 therapy (PRN1008-005 Interim Analysis Report 
August 15 2018).  A further interim analysis will be performed when Part A enrollment and 
follow up is complete. 
12. Safety Monitoring Committee 
A Safety Monitoring Committee (SMC) will review the emerging safety data (TEAEs, safety 
labs), and efficacy, PD, and dose modification data approximately every 3 months within the 
first 6 months following the first patient enrolled in the study, and then with a frequency 
determined as appropriate by the SMC. The SMC members will include the Sponsor's Medical 
Monitor, a Principal Investigator, and a pemphigus clinical expert who is not an investigator in 
the study. The trial statistician will also participate in safety reviews. Documentation of the 
patient data reviewed at each meeting, including the individual SMC member’s confirmation of 
data review and the findings and actions of the SMC, will be included in the Trial Master File. 
SMC findings that impact the safety of patients in this study will be immediately reported to the 
local Competent Authority (CA) and Ethics Committee (EC). 
An SMC Charter outlining the SMC composition and responsibilities will be in place prior to the 
first scheduled meeting. 
13. Changes in the Planned Analysis  
In addition to the secondary efficacy endpoints listed in protocol section 11.2, the proportion of 
patients who are able to achieve CDA/CR at each visit, with or without the need for doses of CS 
of greater than 0.5 mg/kg will be summarized. Cumulative incidence of patients who ever 
achieved CDA/CR by each visit will be summarized as secondary efficacy endpoints also. 


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 28 
14. References 
 
Hertl, M., Jedlickova, H., Karpati, S., et al. (2015), Pemphigus. S2 Guideline for diagnosis and 
treatment – guided by the European Dermatology Forum (EDF) in cooperation with the 
European Academy of Dermatology and Venereology (EADV). Journal of the European 
Academy of Dermatology and Venereology, 29: 405–414. doi: 10.1111/jdv.12772. 
 
Horváth, B., Huizinga, J., Pas, H.H., Mulder, A.B., Jonkman, M.F., Low Dose rituximab is 
effective in pemphigus. Br J Dermatol., 166(2): 405-12, 2012. 
 
PRN1008-005 Interim Analysis Report March 20 2017. 
 
Tjokrowidjaja A, Daniel BS, Frew JW, Sebaratnam DF, Hanna AM, Chee S, Dermawan A, 
Wang CQ, Lim C, Venugopal SS, Rhodes LM, Welsh B, Nijsten T, Murrell DF. 
Br J Dermatol. 2013 Nov;169(5):1000-6. 
 
Werth VP, Fivenson D, Pandya AG, et al. Multicenter randomized, double-blind, placebo-
controlled, clinical trial of dapsone as a glucocorticoid-sparing agent in maintenance-phase 
pemphigus vulgaris. Arch Dematol. 2008 Jan;144(1):25-32.  
 
Wilson MM, Thomas DR, Rubenstein LZ, Chibnall JT, Anderson S, Baxi A, Diebold MR, 
Morley JE. Appetite assessment: simple appetite questionnaire predicts weight loss in 
community-dwelling adults and nursing home residents. Am J Clin Nutr. 2005 
Nov;82(5):1074-81. 
 
 


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29 
15. Appendices 
15.1. 
Schedule of Assessments 
 
 
Screen 
Day 1, 
Week 1 
Pre-dose 
Day 1 
Week 1  
Post-dose 
Day 2, 
Week 1a 
Day 15, 
Week 3 
+/- 3 days 
Day 29, 
Week 5 
+/- 3 days 
Day 57, 
Week 9 
+/- 7 days 
Day 85, 
Week 13  
+/- 7 days 
Day 113, 
Week 17 
+/- 7 days  
Day 141, 
Week 21 
+/- 7 days 
Day 169, 
Week 25 
+/- 7 days 
Unscheduled 
Visit 
Informed Consent 
X 
 
 
 
 
 
 
 
 
 
 
 
Inclusion/Exclusion Criteria 
X 
X 
 
 
 
 
 
 
 
 
 
 
Height 
X 
 
 
 
 
 
 
 
 
 
 
 
Weight 
X 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
Physical exam./med. 
History PDAI/ABSIS 
X 
 
 
 
 
 
 
 
 
 
 
 
Abbreviated physical 
exam., PDAI, ABSIS 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
ECG (12-lead) 
X 
 
 
 
 
 
 
 
 
 
 
(X)b 
Vital Signs 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
MRIc 
 
X 
 
 
X 
 
 
 
 
 
 
 
Urinalysis 
X 
 
 
 
 
 
 
 
 
 
 
 
Hep B &C, HIV, 
QuantiFERON® TB 
X 
 
 
 
 
 
 
 
 
 
 
 
Pregnancy testd 
X 
X 
 
 
 
X 
X 
X 
 
 
X 
 
Skin biopsye 
X 
 
 
 
 
 
 
 
 
 
 
 
Hem, Coag, Chemf 
X 
X 
 
 
X 
X 
X 
X 
 
 
 
X 
FSHg 
X 
 
 
 
 
 
 
 
 
 
 
 
BTK occupancy sampleh 
 
X 
Xh 
X 
X 
X 
X 
X 
 
 
 
(X)b 
PK sample 
 
X 
Xh 
X 
X 
X 
X 
X 
 
 
 
(X)b 
Anti-DSG antibodies. 
 
X 
 
 
 
X 
X 
X 
X 
X 
X 
 
Photography (Optional)i 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
ABQOL & TABQOL 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
SNAQ questionnaire 
 
X 
 
 
 
X 
X 
X 
X 
X 
X 
X 
AEsj 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Concomitant Medications 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Drug dispensed 
 
X 
 
 
 
X 
X 
 
 
 
 
 
Drug reconciliation 
 
 
 
 
X 
X 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 


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30 
Schedule of Assessments Footnotes: 
 
a. Withhold PRN1008 on morning of Day 2 until PK/PD measurement has been taken as close to 12 hours after second dose as possible. Where follow-up on Day 2 is not possible, 
PK/PD samples may be taken on another day in the first week of treatment. On all other days, instruct patient to take PRN1008 in the morning as usual prior to clinic and take note 
of the time taken. Extra PK/PD sample intended for 1 to 5 days after dose adjustment or to replace missing samples–not required for other extra visits. 
b. Only if clinically indicated. 
c. Patients from French sites only: Brain MRI with macrocyclic contrast. Baseline examination to be conducted during the screening period. Follow up examination at Day 15 ± 3 
days. 
d. For women of childbearing potential only. Serum pregnancy test done at screening, urine dip test done at other time points 
e. Performed only if no suitable prior biopsy. 
f. TSH and CPK are only taken at Screening as part of chemistry panel.  
g. To confirm postmenopausal status for women who are not surgically sterile only. 
h. Two-8mL pre-dose blood tubes to be collected at baseline to ensure sufficient samples for later time point assay calculations. On Day 1 blood draw also taken at 2 hours post-
dose (+/- 15 mins) 
i. Photography is used to document skin disease changes; ideally in most patients. Strict masking of patient identity is required. 
j. Including particular questioning on symptoms of headache, nausea, vomiting and visual disturbances. If any of these symptoms are reported, a full neurological examination, 
including the cranial nerves, should be performed. 
 


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15.2. 
General Dose Adjustment Guidelines for Dose Selection in the First 4 
Weeks 
Clinical 
Response 
Trough BTK 
occupancy 
Tolerability* 
Action 
Responder 
≥ 50% 
Well Tolerated Maintain 400 mg bid 
Taper CSs if used in combination 
Poorly 
Tolerated  
Reduce to 300 mg bid 
Taper CSs if used in combination 
< 50% 
Well Tolerated Maintain 400 mg bid 
Taper CSs if used in combination 
Poorly 
Tolerated  
Reduce to 300 mg bid 
Taper CSs if used in combination 
Suboptimal 
Response  
≥ 50% 
Well Tolerated Follow rescue criteria if triggered, if 
not maintain dose at 400 mg bid 
Poorly 
Tolerated  
Follow rescue criteria if triggered, if 
not maintain dose at 400 mg bid if 
feasible 
< 50% 
Well Tolerated Follow rescue criteria if triggered, if 
not, increase dose to 600 mg bid, 
Poorly 
Tolerated  
Follow rescue criteria if triggered, if 
not, increase dose to 600 mg bid if 
tolerability allows. 
* “Well tolerated” is defined as the absence of Grade 3 or greater gastrointestinal AEs, or Grade 2 non-gastrointestinal 
AEs, including liver function changes, related to PRN1008 therapy. 


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 32 
15.3. 
CS rescue criteria and treatment protocol 
Systemic CSs will be avoided during PRN1008 monotherapy unless these “rescue criteria” 
are triggered, as described below, or patients enter the study on low doses (≤ 0.5 mg/kg per 
day). In each rescue scenario, the recommended method of CS taper is shown in Appendix 
15.7. 
Rescue criteria #1: Flare which, in the mind of investigator, is potentially life 
threatening  
x Commence conventional immunosuppressive therapy and discontinue PRN1008 
Rescue criteria #2: Development of severe but not life threatening PV (defined as 
PDAI score ≥ 45) at any time during PRN1008 therapy: 
x If most recent BTK trough occupancy was 70% or more, commence conventional 
immunosuppressive therapy and discontinue PRN1008 
x If most recent BTK trough occupancy was <50% and PRN1008 dose has not been 
increased since last BTK assay, commence, or increase dose to, full dose CS (1.0 
mg/kg) orally and increase dose of PRN1008 by an amount agreed to with the 
Sponsor’s Medical Monitor. At next review: 
o If not responding, commence conventional immunosuppressive therapy and 
discontinue PRN1008 
o If responding, maintain or taper CSs as clinically appropriate while 
maintaining PRN1008 treatment 
x If no BTK trough occupancy is available to guide therapy, maintain PRN1008, and 
commence, or increase to, full dose CS (1.0 mg/kg) orally. Seek to expedite latest 
BTK trough occupancy test result or arrange for a new blood draw to measure BTK 
trough occupancy. At next review: 
o If not responding, commence conventional immunosuppressive therapy and 
discontinue PRN1008 
o If responding, maintain or taper CSs as clinically appropriate while 
maintaining PRN1008 treatment (including adjust dose upwards if BTK 
trough occupancy <50% in consultation with the Sponsor Medical Monitor) 
Rescue criteria #3: Patients with non-severe PV but who fail to achieve “Control of 
Disease Activity (CDA*)” as monotherapy by 4 weeks 


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 33 
x If most recent BTK trough occupancy was 50% or more, commence, or increase 
dose to, low dose CS (0.5 mg/kg) orally. Review after another ~2 weeks: 
o If not responding, commence conventional immunosuppressive therapy and 
discontinue PRN1008 
o If responding, maintain or taper CSs as clinically appropriate while 
maintaining PRN1008 treatment 
x If most recent BTK trough occupancy was <70% and PRN1008 dose has not been 
increased since last BTK assay, increase dose of PRN1008 by an amount agreed 
with the Sponsor’s Medical Monitor. At review after another ~2 weeks: 
o If not responding, commence, or increase dose to, low dose CS (0.5 mg/kg) 
orally and maintain PRN1008 treatment. If a dose of ≥ 0.5 mg/kg is already 
in use, or if after further review the patient is still not responding, 
commence conventional immunosuppressive therapy and discontinue 
PRN1008 
o If responding, maintain PRN1008 treatment and consider tapering of CS 
In each rescue scenario the recommended method of CS taper is shown in Appendix 15.7. 
When patients enter the study on CSs, the dose should be maintained for the initial 2 weeks 
of PRN1008 therapy if possible in order to evaluate the additive effects of PRN1008. 
During this time and subsequently, the rescue criteria should be followed if indicated. In 
other words, in some circumstances the CS dose should be increased, with or without 
cessation of PRN1008. If patients respond well to PRN1008 without increasing the CS 
dose, the Werth taper (Appendix 15.7) should be commenced after four weeks of 
PRN1008 therapy. 
* As defined by the EADV 2014 Pemphigus S2 Guideline (Hertl et al. 2015) 


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 34 
15.4. 
Missing date imputation 
15.4.1. Missing medication and AE stop dates imputation 
For the purpose of inclusion in prior and/or concomitant medication tables, incomplete 
medication stop dates will be imputed as follows: 
x If only day is missing, then set to the last day of the month. 
x If only month is missing, then set to December.  
x If both month and day are missing, then set to December 31.  
x Completely or Year missing date will not be imputed. 
15.4.2. Missing medication start dates and AE onset dates imputation 
For the purpose of inclusion in TEAE tables, incomplete AE onset dates will be imputed as 
follows: 
Missing day only:  
x 
If the year is the same as the year of the first dose of study drug 
x 
If the month is before the month of the first dose of study drug, then the last 
day of the month will be assigned to the missing day. 
x 
If the month is the same as the month of the first dose of study drug, then 
the day of the first dose of study drug will be assigned to the missing day. 
x 
If the month is after the month of the first dose of study drug, then “01” will 
be assigned to the missing day. 
x 
If the year is before the year of the first dose of study drug, then the last day of the 
month will be assigned to the missing day. 
x 
If the year is after the year of the first dose of study drug, then “01” will be 
assigned to the missing day. 
Missing month only: 
x 
If the year is the same as the year of the first dose of study drug, then the month of 
the first dose of study drug will be assigned to the missing month. 
x 
If the year is before the year of the first dose of study drug, then December will be 
assigned to the missing month. 


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 35 
x 
If the year is after the year of the first dose of study drug, then January will be 
assigned to the missing month. 
Missing day and month: 
x 
If the year is the same as the first dose of study drug, then the date of the first dose 
of study drug will be assigned to the missing fields. 
x 
If the year is before the year of first dose of study drug, then December 31 will be 
assigned to the missing fields. 
x 
If the year is after the year of first dose of study drug, then January 1 will be 
assigned to the missing fields. 
If year is missing, onset date will not be imputed. If the imputed end date is non-missing 
and the imputed onset date is after the imputed end date, the onset date will be imputed by 
the imputed end date. 
 


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 36 
15.5. 
Pemphigus Disease 
Activity and 
Quality of Life Evaluation 
Instruments: ABQOL, TABQOL, PDAI, ABSIS 


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Treatment of Autoimmune Bullous Diseases Quality of Life Assessment (TABQOL) 


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 40 
From: Tjokrowidjaja et al. 2013 


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PRN1008-005                                                                                                                                                          Date Issued: 16JAN2019 
 
  
 
 41 
Pemphigus Disease Activity Index (PDAI) (Murell et al. 2008)  


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 42 
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) (Rosenbach et al. 2009) 
 


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 43 
15.6. 
Simple Nutritional Appetite Questionnaire (SNAQ) 
 From Wilson et al., 2005 
 


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 44 
15.7. 
CS taper protocol 
Maintain CS dose for 2 weeks after disease control has been achieved. 
Subsequently, reduce the CS dose by 15% every three weeks. 
 
 
 
 
 
 
-OR-  
 
 
From: Werth et al. 2008 
 
 
 




STATISTICAL ANALYSIS PLAN 
TABLES AND LISTINGS 
MOCK SHELLS AND 
PROGRAMMING NOTES 
 
 
Protocol Title: 
An Open-Label, Phase 2, Pilot Study Investigating the Safety, 
Clinical Activity, Pharmacokinetics, and Pharmacodynamics of 
Oral Treatment with the BTK Inhibitor PRN1008 in Patients with 
Newly Diagnosed or Relapsing Pemphigus Vulgaris (Part A) 
Protocol Number: 
PRN1008-005 
Sponsor: 
Principia Biopharma Australia Pty Ltd. 
400 East Jamie Court, Suite 302 
South San Francisco, CA 94080 
Phone: (650) 416-7700 
Author: 
Summit Analytical, LLC 
8354 Northfield Blvd. 
Bldg. G Suite 3700 
Denver, CO 80205 
SAP Date: 
2019-01-16 
Status: 
Final 2.0 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Statistical Analysis Plan Shells Version 2.0 
   Page 2 of 195 
Document History – Changes compared to previous version of SAP Shells: 
Version 
Date 
Changes 
V1.1 
05JAN2018 
Protocol amendment for French site: add listings for Brain MRI, 
Neurological symptoms, Neurological examination 
V1.1 
05JAN2018 
Protocol addendum: update exist listings for PDAI and ABSIS to add 
screening visit results 
V1.1 
05JAN2018 
ABSIS score: include Total activity score for all patients. Add subset 
analyses for patients with baseline oral involvement total score >0 
and =0 
V1.1 
05JAN2018 
Add new tables for cumulative incidence of CDA and CR by visit 
V1.1 
22JAN2018 
Add baseline anti-DSG 1 and anti-DSG 3 antibody status in 
T14.1.2.1 
V1.2 
28JUL2018 
Incorporated sponsor comments. 
V1.3 
02NOV2018 
Modify Demographics table for DSG, add Treatment related TEAE 
table, add CDA/CR by DSG tables. Remove Urinanalysis shift table. 
Other minor updates. 
V1.4 
12DEC2018 
Updated with new Analysis population 
V2.0 
20DEC2018 
Updated demographics table and corticosteroid by visit table, and 
upversion to final 
 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 3 of 195 
Table of Contents 
 
Table 14.1.1.1 Summary of Analysis Populations Screening Population ............................................................................................ 13 
Table 14.1.1.2 Summary of Patient Disposition Safety (ITT) [1] Analysis Population ......................................................... 14 
Table 14.1.1.3 Summary of Protocol Deviations Safety Analysis Population................................................................................. 15 
Table 14.1.1.4 Summary of Screen Failures  Screening Population ...................................................................................................... 16 
Table 14.1.2.1 Summary of Demographics and Baseline Characteristics Safety Analysis Population ............................ 17 
Table 14.1.2.2 Summary of Demographics and Characteristics at Screening ITT Population ............................................... 23 
Table 14.1.2.3 Summary of Demographics and Characteristics at Screening mITT Population ............................................. 23 
Table 14.1.2.4 Summary of Demographics and Characteristics at Screening Per Protocol Population .......................... 23 
Table 14.1.3.1 Summary of Prior Corticosteroid Medications Safety Analysis Population .................................................. 24 
Table 14.1.3.2 Summary of Concomitant Corticosteroid Medications Safety Analysis Population ................................... 25 
Table 14.1.3.3 Summary of Prior Non-Corticosteroid Medications Safety Analysis Population ........................................ 26 
Table 14.1.3.4 Summary of Concomitant Non-Corticosteroid Medications Safety Analysis Population .......................... 27 
Table 14.1.3.5 Summary of Treatment Exposure Safety Analysis Population ................................................................................... 28 
Table 14.1.3.6 Summary of Treatment Compliance Safety Analysis Population .............................................................................. 29 
Table 14.2.1.1 Proportion of Patients who Achieved Control of Disease Activity (CDA) at or Prior to Week 5 
Visit without Prednis(ol)one >0.5 mg/kg  ITT Population ......................................................................................................................... 30 
Table 14.2.1.2 Proportion of Patients who Achieved Control of Disease Activity (CDA) at or Prior to Week 5 
Visit without Prednis(ol)one >0.5 mg/kg  mITT Population ....................................................................................................................... 30 
Table 14.2.1.3 Proportion of Patients who Achieved Control of Disease Activity (CDA) at or Prior to Week 5 
Visit without Prednis(ol)one >0.5 mg/kg  Per Protocol Population .................................................................................................... 30 
Table 14.2.2.1.1 Proportion of Patients who Achieved Control of Disease Activity (CDA) at or Prior to Week 
5 Visit without Corticosteroids  ITT Population ............................................................................................................................................ 32 
Table 14.2.2.1.2 Proportion of Patients who Achieved Control of Disease Activity (CDA) at or Prior to Week 
5 Visit without Corticosteroids  mITT Population .......................................................................................................................................... 32 
Table 14.2.2.1.3 Proportion of Patients who Achieved Control of Disease Activity (CDA) at or Prior to Week 
5 Visit without Corticosteroids  Per Protocol Population ....................................................................................................................... 32 
Table 14.2.2.2.1 Proportion of Patients who Achieved Complete Response (CR) at or Prior to Week 13 Visit 
without Corticosteroids  ITT Population ............................................................................................................................................................... 33 
Table 14.2.2.2.2 Proportion of Patients who Achieved Complete Response (CR) at or Prior to Week 13 Visit 
without Corticosteroids  mITT Population ............................................................................................................................................................. 33 
Table 14.2.2.2.3 Proportion of Patients who Achieved Complete Response (CR) at or Prior to Week 13 Visit 
without Corticosteroids  Per Protocol Population .......................................................................................................................................... 33 
Table 14.2.2.3.1 Proportion of Patients who Achieved Complete Response (CR) at or Prior to Week 13 Visit 
without Prednis(ol)one >0.5 mg/kg  ITT Population ....................................................................................................................................... 34 
Table 14.2.2.3.2 Proportion of Patients who Achieved Complete Response (CR) at or Prior to Week 13 Visit 
without Prednis(ol)one >0.5 mg/kg  mITT Population ..................................................................................................................................... 34 


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Table 14.2.2.3.3 Proportion of Patients who Achieved Complete Response (CR) at or Prior to Week 13 Visit 
without Prednis(ol)one >0.5 mg/kg  Per Protocol Population .................................................................................................................. 34 
Table 14.2.2.4.1.1 Proportion of Patients who Achieved Control of Disease Activity (CDA) at Visit ITT 
Population .................................................................................................................................................................................................................................... 35 
Table 14.2.2.4.1.2 Proportion of Patients who Achieved Control of Disease Activity (CDA) at Visit mITT 
Population .................................................................................................................................................................................................................................... 36 
Table 14.2.2.4.1.3 Proportion of Patients who Achieved Control of Disease Activity (CDA) at Visit Per 
Protocol Population ............................................................................................................................................................................................................... 36 
Table 14.2.2.4.2.1 Proportion of Patients who Achieved Control of Disease Activity (CDA) without 
Prednis(ol)one >0.5 mg/kg at Visit ITT Population ....................................................................................................................................... 37 
Table 14.2.2.4.2.2 Proportion of Patients who Achieved Control of Disease Activity (CDA) without 
Prednis(ol)one >0.5 mg/kg at Visit mITT Population ..................................................................................................................................... 38 
Table 14.2.2.4.2.3 Proportion of Patients who Achieved Control of Disease Activity (CDA) without 
Prednis(ol)one >0.5 mg/kg at Visit6uiol Per Protocol Population ...................................................................................................... 38 
Table 14.2.2.4.3.1 Proportion of Patients who Achieved Control of Disease Activity (CDA) with 
Prednis(ol)one >0.5 mg/kg at Visit ITT Population ....................................................................................................................................... 39 
Table 14.2.2.4.3.2 Proportion of Patients who Achieved Control of Disease Activity (CDA) with 
Prednis(ol)one >0.5 mg/kg at Visit mITT Population ..................................................................................................................................... 40 
Table 14.2.2.4.3.3 Proportion of Patients who Achieved Control of Disease Activity (CDA) with 
Prednis(ol)one >0.5 mg/kg at Visit Per Protocol Population .................................................................................................................. 40 
Table 14.2.2.4.4.1 Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) by Visit 
ITT Population ........................................................................................................................................................................................................................... 41 
Table 14.2.2.4.4.2 Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) by Visit 
mITT Population ........................................................................................................................................................................................................................ 42 
Table 14.2.2.4.4.3 Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) by Visit 
Per Protocol Population ..................................................................................................................................................................................................... 42 
Table 14.2.2.4.5.1 Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) without 
Prednis(ol)one >0.5 mg/kg by Visit ITT Population ....................................................................................................................................... 43 
Table 14.2.2.4.5.2 Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) without 
Prednis(ol)one >0.5 mg/kg by Visit mITT Population ..................................................................................................................................... 44 
Table 14.2.2.4.5.3 Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) without 
Prednis(ol)one >0.5 mg/kg by Visit Per Protocol Population .................................................................................................................. 44 
Table 14.2.2.4.6.1 Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) with 
Prednis(ol)one >0.5 mg/kg by Visit ITT Population ....................................................................................................................................... 45 
Table 14.2.2.4.6.2 Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) with 
Prednis(ol)one >0.5 mg/kg by Visit mITT Population ..................................................................................................................................... 46 
Table 14.2.2.4.6.3 Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) with 
Prednis(ol)one >0.5 mg/kg by Visit Per Protocol Population .................................................................................................................. 46 
Table 14.2.2.5.1.1 Proportion of Patients who Achieved Complete Response (CR) at Visit ITT Population ........... 47 


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Table 14.2.2.5.1.2 Proportion of Patients who Achieved Complete Response (CR) at Visit mITT Population ......... 48 
Table 14.2.2.5.1.3 Proportion of Patients who Achieved Complete Response (CR) at Visit Per Protocol 
Population .................................................................................................................................................................................................................................... 48 
Table 14.2.2.5.2.1 Proportion of Patients who Achieved Complete Response (CR) without Prednis(ol)one >0.5 
mg/kg at Visit ITT Population ....................................................................................................................................................................................... 49 
Table 14.2.2.5.2.2 Proportion of Patients who Achieved Complete Response (CR) without Prednis(ol)one >0.5 
mg/kg at Visit mITT Population..................................................................................................................................................................................... 50 
Table 14.2.2.5.2.3 Proportion of Patients who Achieved Complete Response (CR) without Prednis(ol)one >0.5 
mg/kg at Visit Per Protocol Population ................................................................................................................................................................. 50 
Table 14.2.2.5.3.1 Proportion of Patients who Achieved Complete Response (CR) with Prednis(ol)one >0.5 
mg/kg at Visit ITT Population ....................................................................................................................................................................................... 51 
Table 14.2.2.5.3.2 Proportion of Patients who Achieved Complete Response (CR) with Prednis(ol)one >0.5 
mg/kg at Visit mITT Population..................................................................................................................................................................................... 52 
Table 14.2.2.5.3.3 Proportion of Patients who Achieved Complete Response (CR) with Prednis(ol)one >0.5 
mg/kg at Visit PP Population ......................................................................................................................................................................................... 52 
Table 14.2.2.5.4.1 Cumulative Incidence of Patients who Achieved Complete Response (CR) by Visit ITT 
Population .................................................................................................................................................................................................................................... 53 
Table 14.2.2.5.4.2 Cumulative Incidence of Patients who Achieved Complete Response (CR) by Visit mITT 
Population .................................................................................................................................................................................................................................... 54 
Table 14.2.2.5.4.3 Cumulative Incidence of Patients who Achieved Complete Response (CR) by Visit Per 
Protocol Population ............................................................................................................................................................................................................... 54 
Table 14.2.2.5.5.1 Cumulative Incidence of Patients who Achieved Complete Response (CR) without 
Prednis(ol)one >0.5 mg/kg by Visit ITT Analysis Population .................................................................................................................. 55 
Table 14.2.2.5.5.2 Cumulative Incidence of Patients who Achieved Complete Response (CR) without 
Prednis(ol)one >0.5 mg/kg by Visit mITT Population ..................................................................................................................................... 56 
Table 14.2.2.5.5.3 Cumulative Incidence of Patients who Achieved Complete Response (CR) without 
Prednis(ol)one >0.5 mg/kg by Visit ........................................................................................................................................................................... 56 
Per Protocol Population ..................................................................................................................................................................................................... 56 
Table 14.2.2.5.6.1 Cumulative Incidence of Patients who Achieved Complete Response (CR) with 
Prednis(ol)one >0.5 mg/kg by Visit ITT Population ....................................................................................................................................... 57 
Table 14.2.2.5.6.2 Cumulative Incidence of Patients who Achieved Complete Response (CR) with 
Prednis(ol)one >0.5 mg/kg by Visit mITT Population ..................................................................................................................................... 58 
Table 14.2.2.5.6.3 Cumulative Incidence of Patients who Achieved Complete Response (CR) with 
Prednis(ol)one >0.5 mg/kg by Visit Per Protocol Population .................................................................................................................. 58 
Table 14.2.2.5.7.1 Duration of Complete Response (CR) Status Among CR Subjects ITT Population............................... 59 
Table 14.2.2.5.7.2 Duration of Complete Response (CR) Status Among CR Subjects mITT Population ............................ 59 
Table 14.2.2.5.7.3 Duration of Complete Response (CR) Status Among CR Subjects Per Protocol Population ......... 59 
Table 14.2.3.1.1 Time to First Control of Disease Activity (CDA): Kaplan Meier Estimates ITT Population ....... 60 
Table 14.2.3.1.2 Time to First Control of Disease Activity (CDA): Kaplan Meier Estimates mITT Population .... 61 


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Table 14.2.3.1.3 Time to First Control of Disease Activity (CDA): Kaplan Meier Estimates Per Protocol 
Population .................................................................................................................................................................................................................................... 61 
Table 14.2.3.2.1 Time to First Complete Response (CR): Kaplan Meier Estimates ITT Population ................................. 62 
Table 14.2.3.2.2 Time to First Complete Response (CR): Kaplan Meier Estimates mITT Population............................... 63 
Table 14.2.3.2.3 Time to First Complete Response (CR): Kaplan Meier Estimates Per Protocol Population ........... 63 
Table 14.2.3.3.1 Time to First End of Consolidation Phase: Kaplan Meier Estimates ITT Population ....................... 64 
Table 14.2.3.2.2 Time to First Complete Response (CR): Kaplan Meier Estimates mITT Population............................... 65 
Table 14.2.3.2.3 Time to First Complete Response (CR): Kaplan Meier Estimates Per Protocol Population ........... 65 
Table 14.2.3.4.1 Time to First Relapse after PRN1008 Treatment Completion or Discontinuation: Kaplan Meier 
Estimates ITT Population ................................................................................................................................................................................................... 66 
Table 14.2.3.4.2 Time to First Relapse after PRN1008 Treatment Completion or Discontinuation Among Subjects 
who Achieved CDA at Week 13 Visit: Kaplan Meier Estimates mITT Population .............................................................................. 67 
Table 14.2.3.4.3 Time to First Relapse after PRN1008 Treatment Completion or Discontinuation Among Subjects 
who Achieved CDA at Week 13 Visit: Kaplan Meier Estimates Per Protocol Population ........................................................... 67 
Table 14.2.4.1.1 Cumulative Corticosteroid Usage over 12 Weeks ITT Population ..................................................................... 68 
Table 14.2.4.1.2 Cumulative Corticosteroid Usage over 12 Weeks mITT Population .................................................................. 68 
Table 14.2.4.1.3 Cumulative Corticosteroid Usage over 12 Weeks Per Protocol Population ............................................... 68 
Table 14.2.4.2.1 Corticosteroid Usage by Visit ITT Population ........................................................................................................... 69 
Table 14.2.4.2.2 Corticosteroid Usage by Visit mITT Population ........................................................................................................ 69 
Table 14.2.4.2.3 Corticosteroid Usage by Visit Per Protocol Population ..................................................................................... 70 
Table 14.2.5.1.1 Summary of Actual Value and Change from Baseline in Pemphigus Disease Area Index (PDAI) 
Total Activity Score by Visit ITT Population ................................................................................................................................................... 71 
Table 14.2.5.1.2 Summary of Actual Value and Change from Baseline in Pemphigus Disease Area Index (PDAI) 
Total Activity Score by Visit mITT Population ................................................................................................................................................. 72 
Table 14.2.5.1.3 Summary of Actual Value and Change from Baseline in Pemphigus Disease Area Index (PDAI) 
Total Activity Score by Visit Per Protocol Population .............................................................................................................................. 72 
Table 14.2.5.2.1 Summary of Actual Value and Change from Baseline in Pemphigus Disease Area Index (PDAI) 
Total Damage Score by Visit ITT Population ........................................................................................................................................................ 73 
Table 14.2.5.2.2 Summary of Actual Value and Change from Baseline in Pemphigus Disease Area Index (PDAI) 
Total Damage Score by Visit mITT Population ...................................................................................................................................................... 74 
Table 14.2.5.2.3 Summary of Actual Value and Change from Baseline in Pemphigus Disease Area Index (PDAI) 
Total Damage Score by Visit Per Protocol Population................................................................................................................................... 74 
Table 14.2.6.1.1 Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder 
Intensity Score (ABSIS) by Visit ITT Population ............................................................................................................................................ 75 
Table 14.2.6.1.2 Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder 
Intensity Score (ABSIS) by Visit mITT Population .......................................................................................................................................... 76 
Table 14.2.6.1.3 Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder 
Intensity Score (ABSIS) by Visit Per Protocol Population ....................................................................................................................... 76 


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Table 14.2.6.2.1 Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder 
Intensity Score (ABSIS) by Visit for Patients with Baseline Oral Involvement Total Score Greater than Zero 
ITT Population ........................................................................................................................................................................................................................... 77 
Table 14.2.6.2.2 Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder 
Intensity Score (ABSIS) by Visit for Patients with Baseline Oral Involvement Total Score Greater than Zero 
mITT Population ........................................................................................................................................................................................................................ 78 
Table 14.2.6.2.3 Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder 
Intensity Score (ABSIS) by Visit for Patients with Baseline Oral Involvement Total Score Greater than Zero 
Per Protocol Population ..................................................................................................................................................................................................... 78 
Table 14.2.6.3.1 Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder 
Intensity Score (ABSIS) by Visit for Patients with Baseline Oral Involvement Total Score Equal to Zero ITT 
Population .................................................................................................................................................................................................................................... 79 
Table 14.2.6.3.2 Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder 
Intensity Score (ABSIS) by Visit for Patients with Baseline Oral Involvement Total Score Equal to Zero mITT 
Population .................................................................................................................................................................................................................................... 80 
Table 14.2.6.3.3 Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder 
Intensity Score (ABSIS) by Visit for Patients with Baseline Oral Involvement Total Score Equal to Zero Per 
Protocol Population ............................................................................................................................................................................................................... 80 
Table 14.2.7.1 Summary of Actual Value and Change from Baseline in Autoimmune Bullous Diseases Quality of 
Life (ABQOL) Score by Visit ITT Population ........................................................................................................................................................ 81 
Table 14.2.7.2 Summary of Actual Value and Change from Baseline in Autoimmune Bullous Diseases Quality of 
Life (ABQOL) Score by Visit mITT Population ...................................................................................................................................................... 82 
Table 14.2.7.3 Summary of Actual Value and Change from Baseline in Autoimmune Bullous Diseases Quality of 
Life (ABQOL) Score by Visit Per Protocol Population................................................................................................................................... 82 
Table 14.2.8.1 Summary of Actual Value and Change from Baseline in Treatment of Autoimmune Bullous Disease 
Quality of Life (TABQOL) Total Score by Visit ITT Population ............................................................................................................. 83 
Table 14.2.8.2 Summary of Actual Value and Change from Baseline in Treatment of Autoimmune Bullous Disease 
Quality of Life (TABQOL) Total Score by Visit mITT Population ........................................................................................................... 84 
Table 14.2.8.3 Summary of Actual Value and Change from Baseline in Treatment of Autoimmune Bullous Disease 
Quality of Life (TABQOL) Total Score by Visit Per Protocol Population ........................................................................................ 84 
Table 14.2.9.1 Summary of Actual Value and Change from Baseline in Simplified Nutritional Appetite 
Questionnaire (SNAQ) Score by Visit ITT Population ..................................................................................................................................... 85 
Table 14.2.9.2 Summary of Actual Value and Change from Baseline in Simplified Nutritional Appetite 
Questionnaire (SNAQ) Score by Visit mITT Population................................................................................................................................... 86 
Table 14.2.9.3 Summary of Actual Value and Change from Baseline in Simplified Nutritional Appetite 
Questionnaire (SNAQ) Score by Visit Per Protocol Population ............................................................................................................... 86 
Table 14.2.10.1.1 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Age Group ITT Population ................................................................................................................................................. 87 


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Table 14.2.10.1.2 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Age Group mITT Population .............................................................................................................................................. 87 
Table 14.2.10.1.3 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Age Group Per Protocol Population ........................................................................................................................... 87 
Table 14.2.10.2.1 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Gender ITT Population ........................................................................................................................................................ 87 
Table 14.2.10.2.2 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Gender mITT Population ...................................................................................................................................................... 87 
Table 14.2.10.2.3 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Gender Per Protocol Population................................................................................................................................... 87 
Table 14.2.10.3.1 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Baseline Total Anti-DSG Category ITT Population .......................................................................................... 88 
Table 14.2.10.3.2 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Baseline Total Anti-DSG Category mITT Population ........................................................................................ 88 
Table 14.2.10.3.3 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Baseline Total Anti-DSG Category Per Protocol Population ..................................................................... 88 
Table 14.2.10.4.1 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Pemphigus History Type ITT Population .................................................................................................................. 88 
Table 14.2.10.4.2 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Pemphigus History Type mITT Population ............................................................................................................... 88 
Table 14.2.10.4.3 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Pemphigus History Type Per Protocol Population ............................................................................................ 88 
Table 14.2.10.5.1 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Pemphigus Anti-DSG Profile ITT Population ........................................................................................................ 89 
Table 14.2.10.5.2 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Pemphigus Anti-DSG Profile mITT Population ...................................................................................................... 89 
Table 14.2.10.5.3 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Pemphigus Anti-DSG Profile Per Protocol Population ................................................................................... 89 
Table 14.2.10.6.1 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Pemphigus Anti-DSG Profile ITT Population ........................................................................................................ 89 
Table 14.2.10.6.2 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Pemphigus Anti-DSG Profile mITT Population ...................................................................................................... 89 
Table 14.2.10.6.3 Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 
0.5mg/kg by Visit by Pemphigus Anti-DSG Profile Per Protocol Population ................................................................................... 89 
Table 14.3.1.1 Overall Summary of Treatment-Emergent Adverse Events Safety Analysis Population ............................ 90 
Table 14.3.1.2 Treatment-Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis 
Population .................................................................................................................................................................................................................................... 91 
Table 14.3.1.3 Treatment-Emergent Adverse Events by Relationship to Study Drug Safety Analysis Population .. 92 
Table 14.3.1.4 Treatment-Emergent Adverse Events by Severity Safety Analysis Population ............................................. 93 


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Table 14.3.1.5 Treatment Related Treatment-Emergent Adverse Events by Severity Safety Analysis Population .. 94 
Table 14.3.2.1 Serious Treatment-Emergent Adverse Events by System Organ Class and Preferred Term Safety 
Analysis Population ............................................................................................................................................................................................................... 95 
Table 14.3.2.2 Serious Treatment-Emergent Adverse Events by Relationship to Study Drug Safety Analysis 
Population .................................................................................................................................................................................................................................... 96 
Table 14.3.2.3 Treatment-Emergent Adverse Events Leading to Treatment Discontinuation Safety Analysis 
Population .................................................................................................................................................................................................................................... 97 
Table 14.3.2.4 Treatment-Emergent Adverse Events Leading to Death Safety Analysis Population ................................. 98 
Table 14.3.3.1 Summary of Actual Value and Change from Baseline in Chemistry Tests by Visit Safety Analysis 
Population .................................................................................................................................................................................................................................... 99 
Table 14.3.3.2 Summary of Actual Value and Change from Baseline in Clinical Laboratory Tests Monitored for 
Drug Induced Liver Injury (DILI) by Visit Safety Analysis Population ........................................................................................ 101 
Table 14.3.3.3 Summary of Actual Value and Change from Baseline in Hematology Tests by Visit Safety 
Analysis Population ............................................................................................................................................................................................................. 103 
Table 14.3.3.4 Summary of Actual Value and Change from Baseline in Coagulation Tests by Visit Safety 
Analysis Population ............................................................................................................................................................................................................. 105 
Table 14.3.3.5 Summary of Urinalysis Tests at Screening Safety Analysis Population ....................................................... 107 
Table 14.3.3.6 Shift from Baseline to Post-baseline Visits in Normality Status of Chemistry Tests Safety 
Analysis Population ............................................................................................................................................................................................................. 108 
Table 14.3.3.7 Shift from Baseline to Post-baseline Visits in Normality Status of Clinical Laboratory Tests 
Monitored for Drug Induced Liver Injury (DILI) Safety Analysis Population ............................................................................ 109 
Table 14.3.3.8 Shift from Baseline to Post-baseline Visits in Normality Status of Hematology Tests Safety 
Analysis Population ............................................................................................................................................................................................................. 111 
Table 14.3.3.9 Shift from Baseline to Post-baseline Visits in Normality Status of Coagulation Tests Safety 
Analysis Population ............................................................................................................................................................................................................. 113 
Table 14.3.4.1 Summary of Actual Value and Change from Baseline in Vital Signs by Visit Safety Analysis 
Population .................................................................................................................................................................................................................................. 115 
Table 14.3.4.2 Summary of Blood Pressure and Heart Rate Outside Normal Range Safety Analysis Population ..... 117 
Table 14.3.5.1 Summary of Physical Examination at Screening Visit Safety Analysis Population ............................... 118 
Table 14.3.5.2 Summary of Physical Examination in Post Screening Visit Safety Analysis Population ................... 119 
Table 14.3.6.1 Summary of Electrocardiogram at Screening Visit Safety Analysis Population ...................................... 120 
Table 14.4.1.1 Summary of Actual Value and Change from Baseline in Anti-DSG by Visit Safety Analysis 
Population .................................................................................................................................................................................................................................. 122 
Table 14.4.2.1 Change from Baseline in Anti-DSG Status by CDA status at Week 5 without Prednis(ol)one >0.5 
mg/kg  ITT Population ........................................................................................................................................................................................................ 123 
Table 14.4.2.2 Change from Baseline in Anti-DSG Status by CR status at Week 13 without Prednis(ol)one >0.5 
mg/kg  ITT Population ........................................................................................................................................................................................................ 125 
Figure 14.2.3.1.1 Kaplan Meier Plots for Time to First Control of Disease Activity (CDA) ITT Population ..... 126 
Figure 14.2.3.1.2 Kaplan Meier Plots for Time to First Control of Disease Activity (CDA) mITT Population .. 127 


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Figure 14.2.3.1.3 Kaplan Meier Plots for Time to First Control of Disease Activity (CDA) PP Population ....... 127 
Figure 14.2.3.2.1 Kaplan Meier Plots for Time to First Complete Response (CR)  ITT Population .............................. 128 
Figure 14.2.3.2.2 Kaplan Meier Plots for Time to First Complete Response (CR)  mITT Population............................ 129 
Figure 14.2.3.2.3 Kaplan Meier Plots for Time to First Complete Response (CR)  PP Population ................................ 129 
Figure 14.2.3.3.1 Kaplan Meier Plots for Time to First End of Consolidation Phase ITT Population ..................... 130 
Figure 14.2.3.3.2 Kaplan Meier Plots for Time to First End of Consolidation Phase mITT Population ................... 131 
Figure 14.2.3.3.3 Kaplan Meier Plots for Time to First End of Consolidation Phase PP Population ........................ 131 
Figure 14.2.3.4.1 Kaplan Meier Plots for Time to First Relapse after PRN1008 Treatment Completion or 
Discontinuation ITT Population................................................................................................................................................................................... 132 
Figure 14.2.3.4.2 Kaplan Meier Plots for Time to First Relapse after PRN1008 Treatment Completion or 
Discontinuation mITT Population ................................................................................................................................................................................ 133 
Figure 14.2.3.4.3 Kaplan Meier Plots for Time to First Relapse after PRN1008 Treatment Completion or 
Discontinuation PP Population ..................................................................................................................................................................................... 133 
Figure 14.3.3.2 Scatter Plots of Peak TBL vs Peak ALT for Identification of Drug Induced Liver Injury (DILI) 
Safety Analysis Population ............................................................................................................................................................................................ 134 
Listing 16.2.1.1  Patient Disposition Screening Population ................................................................................................................ 135 
Listing 16.2.1.2  Protocol Deviations Screening Population ................................................................................................................ 136 
Listing 16.2.2.1  Demographics and Baseline Characteristics Safety Analysis Population ............................................. 137 
Listing 16.2.2.2 Medical History Safety Analysis Population ............................................................................................................. 138 
Listing 16.2.2.3 Inclusion/Exclusion Criteria Screening Population ............................................................................................. 139 
Listing 16.2.3.1 Prior and Concomitant Corticosteroid Medications Safety Analysis Population ............................... 140 
Listing 16.2.3.2 Prior and Concomitant Non-Corticosteroid Medications Safety Analysis Population ..................... 141 
Listing 16.2.3.3 Study Drug Accountability Safety Analysis Population ...................................................................................... 142 
Listing 16.2.3.4 Study Drug Administration Safety Analysis Population ...................................................................................... 143 
Listing 16.2.3.5 Study Drug Exposure Safety Analysis Population .................................................................................................... 144 
Listing 16.2.3.6 Study Drug Compliance Safety Analysis Population ............................................................................................... 145 
Listing 16.2.4.1.1 Control of Disease Activity (CDA) Status and Predni(ol)one Use Condition Within 4 Weeks 
IIT Population ......................................................................................................................................................................................................................... 146 
Listing 16.2.4.1.2 Control of Disease Activity (CDA) Status and Corticosteroids Use Condition Within 4 
Weeks IIT Population .......................................................................................................................................................................................................... 147 
Listing 16.2.4.1.3 Complete Response (CR) Status and Corticosteroids Use Condition Within 12 Weeks ITT 
Population .................................................................................................................................................................................................................................. 148 
Listing 16.2.4.1.4 Complete Response (CR) Status and Predni(ol)one Use Condition Within 12 Weeks ITT 
Population .................................................................................................................................................................................................................................. 149 
Listing 16.2.4.1.5 Disease Outcome Status ITT Population ..................................................................................................................... 150 
Listing 16.2.4.1.6 Time to First Control of Disease Activity (CDA) ITT Population ......................................................... 151 
Listing 16.2.4.1.7 Time to First Complete Response (CR) IIT Population ................................................................................... 152 
Listing 16.2.4.1.8 Time to First End of Consolidation Phase IIT Population .......................................................................... 153 


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Listing 16.2.4.1.9 Time to First Relapse after Week 13 Visit ITT Population ....................................................................... 154 
Listing 16.2.4.1.10 Duration of Complete Response (CR) Status Among CR Subjects .............................................................. 155 
ITT Population ......................................................................................................................................................................................................................... 155 
Listing 16.2.4.2.1 Pemphigus Disease Area Index (PDAI) Score IIT Population ....................................................................... 156 
Listing 16.2.4.2.2 Pemphigus Disease Area Index (PDAI) Total Scores ITT Population ....................................................... 158 
Listing 16.2.4.3.1 Autoimmune Bullous Skin Disorder Intensity Score (ABSIS)—Skin Involvement ITT Population
 .......................................................................................................................................................................................................................................................... 159 
Listing 16.2.4.3.2 Autoimmune Bullous Skin Disorder Intensity Score (ABSIS)—Oral Involvement ITT Population
 .......................................................................................................................................................................................................................................................... 160 
Listing 16.2.4.3.3 Autoimmune Bullous Skin Disorder Intensity Score (ABSIS)—Severity ITT Population .............. 161 
Listing 16.2.4.3.4 Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) ITT Population .................................... 162 
Listing 16.2.4.4.1 Autoimmune Bullous Diseases Quality of Life (ABQOL) Questionnaire ITT Population .............. 163 
Listing 16.2.4.4.2 Autoimmune Bullous Diseases Quality of Life (ABQOL) Score ITT Population ................................. 165 
Listing 16.2.4.5.1 Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL) Questionnaire ITT 
Population .................................................................................................................................................................................................................................. 166 
Listing 16.2.4.5.2 Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL) Score ITT Population .. 168 
Listing 16.2.4.6.1 Simplified Nutritional Appetite Questionnaire (SNAQ) Questionnaire ITT Population ............ 169 
Listing 16.2.4.6.2 Simplified Nutritional Appetite Questionnaire (SNAQ) Score ITT Population ............................... 170 
Listing 16.2.5.1 Adverse Events Safety Analysis Population ................................................................................................................ 171 
Listing 16.2.5.2 Serious Adverse Events Safety Analysis Population ............................................................................................. 173 
Listing 16.2.5.3 Adverse Events Leading to Treatment Discontinuation Safety Analysis Population ........................ 175 
Listing 16.2.5.4 Adverse Events Leading to Death Safety Analysis Population ....................................................................... 177 
Listing 16.2.6.1 Clinical Laboratory Results - Chemistry Tests Safety Analysis Population ...................................... 179 
Listing 16.2.6.2 Clinical Laboratory Results – Drug Induced Liver Injury Safety Analysis Population .............. 180 
Listing 16.2.6.3 Clinical Laboratory Results – Hematology Safety Analysis Population .................................................. 181 
Listing 16.2.6.4 Clinical Laboratory Results – Coagulation Safety Analysis Population ................................................ 182 
Listing 16.2.6.5 Clinical Laboratory Results – Urinalysis Safety Analysis Population .................................................. 183 
Listing 16.2.6.6 Clinical Laboratory Results – Serology Safety Analysis Population ....................................................... 184 
Listing 16.2.6.7 Vital Sign Results Safety Analysis Population ...................................................................................................... 185 
Listing 16.2.6.8 Physical Examination Safety Analysis Population .................................................................................................. 186 
Listing 16.2.6.9 Electrocardiogram Results at Screening Visit Safety Analysis Population ........................................ 187 
Listing 16.2.6.10 Female Reproductive Status and Pregnancy Test Results Safety Analysis Population ................. 188 
Listing 16.2.6.11 Food Intake Status Safety Analysis Population .................................................................................................... 189 
Listing 16.2.6.12 Brain MRI Results Safety Analysis Population ...................................................................................................... 190 
Listing 16.2.6.13 Neurological Symptoms Safety Analysis Population ............................................................................................. 191 
Listing 16.2.6.14 Neurological Examination Safety Analysis Population ...................................................................................... 192 
Listing 16.2.7.1 Plasma Concentrations of PRN1008 Safety Analysis Population ..................................................................... 193 
Listing 16.2.7.2 Percentage BTK Occupancy ITT Population ..................................................................................................................... 194 


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Listing 16.2.7.3 Anti-DSG 1-3 Autoantibody Levels ITT Population .................................................................................................. 195 
  


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Table 14.1.1.1 
Summary of Analysis Populations 
Screening Population 
 
 
400 mg 
500 mg 
600 mg 
Total [1] 
 
 
 
 
 
SCREENING POPULATION 
 
 
 
xx 
  ENROLLED 
 
 
 
xx (xx.x)  
  SCREEN FAILURE 
 
 
 
xx (xx.x) 
SAFETY ANALYSIS POPULATION [2] 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
ITT POPULATION [2] 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
mITT POPULATION [2] 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
PP POPULATION [2] 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
[1] Percentages are based on the number of patients in Screening Population. 
[2] Percentages in respective dose levels are based on the number of patients in Total column. 
Source Data: Listing 16.2.1.1 and Listing 16.2.2.3 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 


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Table 14.1.1.2 
Summary of Patient Disposition 
Safety (ITT) [1] Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
n (%)  
n (%) 
n (%) 
n (%) 
 
 
 
 
 
COMPLETION STATUS 
 
 
 
 
  COMPLETED 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  EARLY WITHDRAWAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
PRIMARY REASONS FOR EARLY 
WITHDRAWAL [2] 
 
 
 
 
  ADVERSE EVENT 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  DEATH 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  LACK OF EFFICACY 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  LOST TO FOLLOW-UP 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NON-COMPLIANCE WITH STUDY DRUG 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PHYSICIAN DECISION 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREGNANCY 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PROGRESSIVE DISEASE 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PROTOCOL VIOLATION 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  RECOVERY 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  STUDY TERMINATED BY SPONSOR 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  TECHNICAL PROBLEM 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  WITHDRAWAL BY SUBJECT 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  OTHER 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period.  
[1] Safety and ITT Populations include the same patients. 
[2] Percentages are based on the number of patients who withdrew early from the study in the respective 
dose levels.  
Source Data: Listing 16.2.1.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
 


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Table 14.1.1.3 
Summary of Protocol Deviations 
Safety Analysis Population 
 
 
Total 
 
(N=xx) 
 
n (%) 
 
 
PATIENTS WITH AT LEAST ONE MAJOR PROTOCOL DEVIATION 
xx (xx.x) 
 
 
CATEGORY 1 
xx (xx.x) 
CATEGORY 2 
xx (xx.x) 
CATEGORY 3 
xx (xx.x) 
CATEGORY 4 
xx (xx.x) 
… 
 
Note: Patients could have more than one major protocol deviation. Site-level Protocol Deviations are not 
included in this table. 
Source Data: Listing 16.2.1.2 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 


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Table 14.1.1.4 
Summary of Screen Failures  
Screening Population 
 
 
Total 
 
(N=xx) 
 
n (%) 
 
 
SCREEN FAILURES 
xx (xx.x) 
 
 
SCREAN FAILURE REASONS 
 
  <Inclusion Cetieria 1 verbatim> 
xx (xx.x) 
  … 
xx (xx.x) 
  … 
xx (xx.x) 
 
xx (xx.x) 
… 
 
Note: Only Inclusion/Exclusion Critiera with at least one subject are presented.  
      Each subject could be excluded due to multiple reasons. 
Source Data: Listing 16.2.1.3 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
PROGRAMMING NOTE: All subjects in Part A is included in this table, regardless of whether they have signed 
ICF.  
Percentage for total screen failures uses the Total number of subjects as denominator. 
Percentages for each screen fail reasons use total screen failures as denominator. 
Use IETEST, IE criteria verbatim as the screen fail reasons, instead of IN01, IN02 etc.  
Only include criteria where at least one subject is present.  
Total number of screen fail reasons should be at least as many as total screen failed, but may be more if 
some subjects have more than one screen fail reason. 


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Table 14.1.2.1 
Summary of Demographics and Baseline Characteristics 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
AGE (years) 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
  MEDIAN 
xx.x 
 
 xx.x 
xx.x 
xx.x 
  MIN, MAX 
xx, xx 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
 
SEX n (%) 
 
 
 
 
  MALE 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  FEMALE 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
RACE n (%) 
 
 
 
 
  WHITE 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  BLACK OR AFRICAN AMERICAN 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  ASIAN 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  AMERICAN INDIAN OR ALASKA NATIVE 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NATIVE HAWAIIAN OR OTHER PACIFIC 
ISLANDER 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  OTHER 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  FRENCH SITES ONLY – NOT COLLECTED 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
ETHNICITY n (%) 
 
 
 
 
  HISPANIC OR LATINO 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NOT HISPANIC OR LATINO 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
FRENCH SITES ONLY – NOT COLLECTED 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
BMI=(Baseline body weight in kilograms)/(Baseline height in meters)^2 
[1]   Time from pemphigus vulgaris confirmed date to screen date in months is defined as (Screening Date – 
Date of pemphigus vulgaris confirmed +1) / 30.4375 
[2]   Newly diagnosed is defined as subjects who were diagnosed at most 6 months prior to date of 
screening, otherwise the subject is relapsed. 
Source Data: Listing 16.2.2.1 


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<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 


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Table 14.1.2.1 
Summary of Demographics and Baseline Characteristics 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
PEMPHIGUS TYPE n (%) 
 
 
 
 
  PEMPHIGUS VULGARIS 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  BULLOUS PEMPHIGOID 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PEMPHIGUS FOLLACEUS 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  EPIDERMOLYSIS BULLOSA AQUISITA 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  LINEAR IGA BULLOUS DERMATOSES 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  MUCOUS MEMBRANE PEMPHIGOID 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  OTHER 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
TIME FROM PEMPHIGUS VULGARIS 
CONFIRMED DATE TO SCREEN DATE 
(Months) [1] 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.xxx (xx.xxxx) xx.xxx (xx.xxxx) xx.xxx (xx.xxxx) xx.xxx (xx.xxxx) 
  MEDIAN 
xx.xxx 
xx.xxx 
xx.xxx 
xx.xxx 
  MIN, MAX 
xx.xx, xx.xx 
xx.xx, xx.xx 
xx.xx, xx.xx 
xx.xx, xx.xx 
 
 
 
 
 
PEMPHIGUS HISTORY TYPE n (%) [2] 
 
 
 
 
  NEWLY DIAGNOSED 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  RELAPSED 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
PEMPHIGUS ANTI-DSG PROFILE 
 
 
 
 
  ANTI-DSG 3 POSITIVE (PV) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  ANTI-DSG 1 POSITIVE AND ANTI-DSG 
3 NEGATIVE (PF) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  ANTI-DSG 1 NEGATIVE AND ANTI-DSG 
3 NEGATIVE  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
BMI=(Baseline body weight in kilograms)/(Baseline height in meters)^2 
[1]   Time from pemphigus vulgaris confirmed date to screen date in months is defined as (Screening Date – 
Date of pemphigus vulgaris confirmed +1) / 30.4375 
[2]   Newly diagnosed is defined as subjects who were diagnosed at most 6 months prior to date of 
screening, otherwise the subject is relapsed. 


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Source Data: Listing 16.2.2.1 
<DIRECTORY PATH>PROGRAM NAME Executed: DDMMMYYYY hh:mm 
 


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Table 14.1.2.1 
Summary of Demographics and Baseline Characteristics 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
PEMPHIGUS SEVERITY AT BASELINE 
 
 
 
 
  MILD (PDAI ACTIVITY < 15) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  MODERATE (PDAI ACTIVITY > 15) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
ANTI-DSG STATUS AT BASELINE n (%) 
 
 
 
 
  ANTI-DSG 1 + and ANTI-DSG 3 - 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  ANTI-DSG 1 - and ANTI-DSG 3 + 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  ANTI-DSG 1 + and ANTI-DSG 3 + 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  ANTI-DSG 1 - and ANTI-DSG 3 - 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
TOTAL ANTI-DSG n (%) 
 
 
 
 
  ≥ 100 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  < 100 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
PDAI SKIN ACTIVITY > 0 n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
PDAI SCALP ACTIVITY > 0 n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
PDAI MUCOSAL ACTIVITY > 0 n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
ABSIS ORAL ACTIVITY > 0 n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
BMI=(Baseline body weight in kilograms)/(Baseline height in meters)^2 
[1]   Time from pemphigus vulgaris confirmed date to screen date in months is defined as (Screening Date – 
Date of pemphigus vulgaris confirmed +1) / 30.4375 
[2]   Newly diagnosed is defined as subjects who were diagnosed at most 6 months prior to date of 
screening, otherwise the subject is relapsed. 
Source Data: Listing 16.2.2.1 
 
<DIRECTORY PATH>PROGRAM NAME Executed: DDMMMYYYY hh:mm 
 


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Table 14.1.2.1 
Summary of Demographics and Baseline Characteristics 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
HEIGHT (cm) 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.xx (xxx.xxx) xxx.xx (xxx.xxx) xxx.xx (xxx.xxx) xxx.xx (xxx.xxx) 
  MEDIAN 
xxx.xx 
xxx.xx 
xxx.xx 
xxx.xx 
  MIN, MAX 
xxx.x, xxx.x 
xxx.x, xxx.x 
xxx.x, xxx.x 
xxx.x, xxx.x 
 
 
 
 
 
WEIGHT (kg) 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.xx (xx.xxx) 
xx.xx (xx.xxx) 
xx.xx (xx.xxx) 
xx.xx (xx.xxx) 
  MEDIAN 
xx.xx 
xx.xx 
xx.xx 
xx.xx 
  MIN, MAX 
xx.x, xxx.x 
xx.x, xxx.x 
xx.x, xxx.x 
xx.x, xxx.x 
 
 
 
 
 
BODY MASS INDEX (BMI) (kg/m^2) 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.xxx (xx.xxxx) xx.xxx (xx.xxxx) xx.xxx (xx.xxxx) xx.xxx (xx.xxxx) 
  MEDIAN 
xx.xxx 
xx.xxx 
xx.xxx 
xx.xxx 
  MIN, MAX 
xx.xx, xx.xx 
xx.xx, xx.xx 
xx.xx, xx.xx 
xx.xx, xx.xx 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
BMI=(Baseline body weight in kilograms)/(Baseline height in meters)^2 
[1]   Time from pemphigus vulgaris confirmed date to screen date in months is defined as (Screening Date – 
Date of pemphigus vulgaris confirmed +1) / 30.4375 
[2]   Newly diagnosed is defined as subjects who were diagnosed at most 6 months prior to date of 
screening, otherwise the subject is relapsed. 
Source Data: Listing 16.2.2.1 
 
<DIRECTORY PATH>PROGRAM NAME Executed: DDMMMYYYY hh:mm 
Prog Notes: for pemphigus type, do not need to display a category if count of this category equal to 0. The 
pemphigus type should be all PV since this is a PV trial. 
Repeat the above table for the other analysis populations titled below. 


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Table 14.1.2.2 
Summary of Demographics and Characteristics at Screening 
ITT Population 
Table 14.1.2.3 
Summary of Demographics and Characteristics at Screening 
mITT Population 
Table 14.1.2.4 
Summary of Demographics and Characteristics at Screening 
Per Protocol Population 
 
 


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Table 14.1.3.1 
Summary of Prior Corticosteroid Medications 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
ATC Level 4 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
  Preferred Term 
n (%)  
n (%) 
n (%) 
n (%) 
 
 
 
 
 
TOTAL NUMBER OF PRIOR CORTICOSTEROID 
 MEDICATIONS 
xx 
xx 
xx 
xx 
 
 
 
 
 
NUMBER OF PATIENTS WITH AT LEAST ONE 
 PRIOR CORTICOSTEROID MEDICATION 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
ATC LEVEL 4 #1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #3 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #4 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
A prior medication is defined as any medication that has a stop date before the date of first dose of 
study drug. A patient is counted only once if the patient reported multiple medications per each ATC 
level 4 or preferred term. 
Medications with start date missing or before first dose and missing stop dates are classified as 
both prior and concomitant. Medications with start date on or after first dose and missing stop dates 
are classified as concomitant. 
      Prior medications were coded with the WHO Drug dictionary (WHO Drug DDE June 2015). 
Source Data: Listing 16.2.3.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Medications will be sorted in descending order by ATC level 4 category based on the total 
column. Within each ATC level 4 category, preferred terms will be sorted in descending order based on the 
total column. 
 
  


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Table 14.1.3.2 
Summary of Concomitant Corticosteroid Medications 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
ATC Level 4 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
  Preferred Term 
n (%)  
n (%) 
n (%) 
n (%) 
 
 
 
 
 
TOTAL NUMBER OF CONCOMITANT CORTICOSTEROID 
 MEDICATIONS 
xx 
xx 
xx 
xx 
 
 
 
 
 
NUMBER OF PATIENTS WITH AT LEAST ONE 
 CONCOMITANT CORTICOSTEROID MEDICATION 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
ATC LEVEL 4 #1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #3 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #4 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
A Concomitant medication is defined as any medication that has a stop date on or after the date of 
first dose of study drug. A patient is counted only once if the patient reported multiple medications 
per each ATC level 4 or perferred term. 
Medications with start date missing or before first dose and missing stop dates are classified as 
both prior and concomitant. Medications with start date on or after first dose and missing stop dates 
are classified as concomitant. 
      Concomitant medications were coded with the WHO Drug dictionary (WHO Drug DDE June 2015). 
Source Data: Listing 16.2.3.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Medications will be sorted in descending order by ATC level 2 category based on the total 
column. Within each ATC level 2 category, preferred terms will be sorted in descending order based on the 
total column. 
 
 
  


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Table 14.1.3.3 
Summary of Prior Non-Corticosteroid Medications 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
ATC Level 4 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
  Preferred Term 
n (%)  
n (%) 
n (%) 
n (%) 
 
 
 
 
 
TOTAL NUMBER OF PRIOR NON-CORTICOSTEROID 
MEDICATIONS 
xx 
xx 
xx 
xx 
 
 
 
 
 
NUMBER OF PATIENTS WITH AT LEAST ONE 
 PRIOR NON-CORTICOSTEROID MEDICATION 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
ATC LEVEL 4 #1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #3 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #4 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
A prior medication is defined as any medication that has a stop date before the date of first dose of 
study drug. A patient is counted only once if the patient reported multiple medications per each ATC 
level 4 or preferred term. 
Medications with start date missing or before first dose and missing stop dates are classified as 
both prior and concomitant. Medications with start date on or after first dose and missing stop dates 
are classified as concomitant. 
Prior medications were coded with the WHO Drug dictionary (WHO Drug DDE June 2015). 
Source Data: Listing 16.2.3.2 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Medications will be sorted in descending order by ATC level 2 category based on the total 
column. Within each ATC level 2 category, preferred terms will be sorted in descending order based on the 
total column. 
 
  


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Table 14.1.3.4 
Summary of Concomitant Non-Corticosteroid Medications 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
ATC Level 4 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
  Preferred Term 
n (%)  
n (%) 
n (%) 
n (%) 
 
 
 
 
 
TOTAL NUMBER OF CONCOMITANT NON-CORTICOSTEROID 
MEDICATIONS 
xx 
xx 
xx 
xx 
 
 
 
 
 
 
 
 
 
 
NUMBER OF PATIENTS WITH AT LEAST ONE 
 CONCOMITANT NON-CORTICOSTEROID MEDICATION 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
ATC LEVEL 4 #1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #3 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #4 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
A Concomitant medication is defined as any medication that has a stop date on or after the date of 
first dose of study drug. A patient is counted only once if the patient reported multiple medications 
per each ATC level 4 or perferred term. 
Medications with start date missing or before first dose and missing stop dates are classified as 
both prior and concomitant. Medications with start date on or after first dose and missing stop dates 
are classified as concomitant. 
Concomitant medications were coded with the WHO Drug dictionary (WHO Drug DDE June 2015). 
Source Data: Listing 16.2.3.2 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Medications will be sorted in descending order by ATC level 2 category based on the total 
column. Within each ATC level 2 category, preferred terms will be sorted in descending order based on the 
total column. 
 
 
  


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Table 14.1.3.5 
Summary of Treatment Exposure 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
OVERALL DURATION OF EXPOSURE (Days) [1] 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
AVERAGE DAILY DOSE (mg/day) [2] 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
   
 
 
 
 
NUMBER OF DOSE ADJUSTMENTS n (%) 
 
 
 
 
  1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  3 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  … 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
MINIMUM DOSE LEVEL (mg) n (%) 
 
 
 
 
  100  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  200  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  300  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  400  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
[1] Overall duration of exposure = Date of last dose – Date of first dose + 1 
[2] Average daily dose = Sum of doses taken during the treatment / Overall duration of exposure 
Source Data: Listing 16.2.3.5 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 


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Table 14.1.3.6 
Summary of Treatment Compliance 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
OVERALL COMPLIANCE (%) [1]  
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
OVERALL COMPLIANCE 
CATEGORY n (%) 
 
 
 
 
  < 80% 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  >= 80% and <=120% 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  > 120% 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
[1] Overall compliance (%) = (Total number of tablets taken during the treatment / Total number of tablets 
planned during the treatment) * 100 
Source Data: Listing 16.2.3.6 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
 


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Table 14.2.1.1 
Proportion of Patients who Achieved Control of Disease Activity (CDA) at or Prior to Week 5 Visit without 
Prednis(ol)one >0.5 mg/kg  
ITT Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Control of disease activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. 
Patients who achieved CDA at or prior to the Week 5 visit and had no occurrence of daily 
prednis(ol)one >0.5 mg/kg before the first CDA date during the study treatment will be counted as 
responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
Source Data: Listing 16.2.4.1.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog note: Early treatment terminators or subjects whose CDA status are completely missing are considered 
non-responders. 
 
Repeat this table for the other populations: 
Table 14.2.1.2 
Proportion of Patients who Achieved Control of Disease Activity (CDA) at or Prior to Week 5 Visit without 
Prednis(ol)one >0.5 mg/kg  
mITT Population 
 
Table 14.2.1.3 
Proportion of Patients who Achieved Control of Disease Activity (CDA) at or Prior to Week 5 Visit without 
Prednis(ol)one >0.5 mg/kg  
Per Protocol Population 
 
 


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Table 14.2.2.1.1 
Proportion of Patients who Achieved Control of Disease Activity (CDA) at or Prior to Week 5 Visit without 
Corticosteroids  
ITT Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL  
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Control of disease activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. 
Patients who achieved CDA at or before the Week 5 visit and had no occurrence of any 
corticosteroidbefore the first CDA visit during the study treatment will be counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
Source Data: Listing 16.2.4.1.2 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog note: Early treatment terminators or subjects whose CDA status are completely missing are considered 
non-responders. 
Repeate this table for the other populations 
Table 14.2.2.1.2 
Proportion of Patients who Achieved Control of Disease Activity (CDA) at or Prior to Week 5 Visit without 
Corticosteroids  
mITT Population 
 
Table 14.2.2.1.3 
Proportion of Patients who Achieved Control of Disease Activity (CDA) at or Prior to Week 5 Visit without 
Corticosteroids  
Per Protocol Population 
 


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Table 14.2.2.2.1 
Proportion of Patients who Achieved Complete Response (CR) at or Prior to Week 13 Visit without 
Corticosteroids  
ITT Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL  
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
receiving minimal or no non-PRN 1008 immunotherapy. 
Patients who achieved CR at or prior to the Week 13 visit and had no occurrence of any corticosteroid 
before the first CR date during the study treatment will be counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
Source Data: Listing 16.2.4.1.3 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog note: Early treatment terminators or subjects whose CR status are completely missing are considered 
non-responders. 
Repeat for the other populations. 
Table 14.2.2.2.2 
Proportion of Patients who Achieved Complete Response (CR) at or Prior to Week 13 Visit without 
Corticosteroids  
mITT Population 
 
Table 14.2.2.2.3 
Proportion of Patients who Achieved Complete Response (CR) at or Prior to Week 13 Visit without 
Corticosteroids  
Per Protocol Population 
 


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Table 14.2.2.3.1 
Proportion of Patients who Achieved Complete Response (CR) at or Prior to Week 13 Visit without 
Prednis(ol)one >0.5 mg/kg  
ITT Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL  
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
receiving minimal or no non-PRN 1008 immunotherapy. 
Patients achieved CR at any visit on or before the Week 13 visit during the study treatment and had no 
occurrence of daily prednis(ol)one >0.5 mg/kg from the first dose taken date up to the day before 
first CR date will be counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
Source Data: Listing 16.2.4.1.4 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog note: Early treatment terminators or subjects whose CR status are completely missing are considered 
non-responders. 
Repeat above table for the other populations. 
Table 14.2.2.3.2 
Proportion of Patients who Achieved Complete Response (CR) at or Prior to Week 13 Visit without 
Prednis(ol)one >0.5 mg/kg  
mITT Population 
 
Table 14.2.2.3.3 
Proportion of Patients who Achieved Complete Response (CR) at or Prior to Week 13 Visit without 
Prednis(ol)one >0.5 mg/kg  
Per Protocol Population 
 


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Table 14.2.2.4.1.1 
Proportion of Patients who Achieved Control of Disease Activity (CDA) at Visit 
ITT Population 
 
 
Visit 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
WEEK25 D169 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Control of disease activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
Source Data: Listing 16.2.4.1.5 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
Repeat the above table for the other populations 


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Table 14.2.2.4.1.2 
Proportion of Patients who Achieved Control of Disease Activity (CDA) at Visit 
mITT Population 
 
Table 14.2.2.4.1.3 
Proportion of Patients who Achieved Control of Disease Activity (CDA) at Visit 
Per Protocol Population 
 


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Table 14.2.2.4.2.1 
Proportion of Patients who Achieved Control of Disease Activity (CDA) without Prednis(ol)one >0.5 mg/kg at 
Visit 
ITT Population 
 
 
Visit 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
WEEK25 D169 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Control of disease activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. 
Patients who achieved CDA at a specific visit and had no occurrence of daily prednis(ol)one >0.5 mg/kg 
before each visit date during the study treatment will be counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
Source Data: Listing 16.2.4.1.5 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
Repeat above table for other populations. 


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Table 14.2.2.4.2.2 
Proportion of Patients who Achieved Control of Disease Activity (CDA) without Prednis(ol)one >0.5 mg/kg at 
Visit 
mITT Population 
 
Table 14.2.2.4.2.3 
Proportion of Patients who Achieved Control of Disease Activity (CDA) without Prednis(ol)one >0.5 mg/kg at 
Visit6uiol 
Per Protocol Population 
 


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Table 14.2.2.4.3.1 
Proportion of Patients who Achieved Control of Disease Activity (CDA) with Prednis(ol)one >0.5 mg/kg at 
Visit 
ITT Population 
 
 
Visit 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
WEEK25 D169 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Control of disease activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. 
Patients who achieved CDA at a specific visit and had no occurrence of daily prednis(ol)one >0.5 mg/kg 
before each visit date during the study treatment will be counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
Source Data: Listing 16.2.4.1.5 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
Repeat above table with the other populations. 


Principia Biopharma Australia Pty Ltd. 
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Page 40 of 195 
Table 14.2.2.4.3.2 
Proportion of Patients who Achieved Control of Disease Activity (CDA) with Prednis(ol)one >0.5 mg/kg at 
Visit 
mITT Population 
 
Table 14.2.2.4.3.3 
Proportion of Patients who Achieved Control of Disease Activity (CDA) with Prednis(ol)one >0.5 mg/kg at 
Visit 
Per Protocol Population 
 
 


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Page 41 of 195 
 
 
Table 14.2.2.4.4.1 
Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) by Visit 
ITT Population 
 
 
Visit 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK3 D15 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK5 D29 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK25 D169 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Control of disease activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. Cumulative responder rate is used, i.e., any patient achieves the 
response at any study visit is counted into all the later study visits. 
      Unscheduled visits are not included in this summary. 
Patients who ever achieved CDA on or before a specific visit during the study treatment will be 
counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
Source Data: Listing 16.2.4.1.5 


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PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 42 of 195 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Repeat above table with other populations: 
Table 14.2.2.4.4.2 
Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) by Visit 
mITT Population 
 
 
Table 14.2.2.4.4.3 
Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) by Visit 
Per Protocol Population 
 
 


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Page 43 of 195 
 
Table 14.2.2.4.5.1 
Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) without Prednis(ol)one >0.5 
mg/kg by Visit 
ITT Population 
 
 
Visit 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK3 D15 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK5 D29 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK25 D169 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Control of disease activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. Cumulative responder rate is used, i.e., any patient achieves the 
response at any study visit is counted into all the later study visits. 
Unscheduled visits are not included in this summary. 
      Patients who ever achieved CDA and had no occurrence of daily prednis(ol)one >0.5 mg/kg before the 
first CDA date during the study treatment will be counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
Source Data: Listing 16.2.4.1.5 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 44 of 195 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Repeat above table for the following: 
Table 14.2.2.4.5.2 
Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) without Prednis(ol)one >0.5 
mg/kg by Visit 
mITT Population 
 
Table 14.2.2.4.5.3 
Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) without Prednis(ol)one >0.5 
mg/kg by Visit 
Per Protocol Population 
 


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Page 45 of 195 
 
Table 14.2.2.4.6.1 
Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) with Prednis(ol)one >0.5 
mg/kg by Visit 
ITT Population 
 
 
Visit 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK3 D15 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK5 D29 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK25 D169 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Control of disease activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. Cumulative responder rate is used, i.e., any patient achieves the 
response at any study visit is counted into all the later study visits. 
Unscheduled visits are not included in this summary. 
      Patients who ever achieved CDA and had daily prednis(ol)one >0.5 mg/kg before a speicfic visit during 
the study treatment will be counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
Source Data: Listing 16.2.4.1.5 


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PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 46 of 195 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Repeat table for following populations 
Table 14.2.2.4.6.2 
Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) with Prednis(ol)one >0.5 
mg/kg by Visit 
mITT Population 
 
Table 14.2.2.4.6.3 
Cumulative Incidence of Patients who Achieved Control of Disease Activity (CDA) with Prednis(ol)one >0.5 
mg/kg by Visit 
Per Protocol Population 
 


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Page 47 of 195 
 
 
Table 14.2.2.5.1.1 
Proportion of Patients who Achieved Complete Response (CR) at Visit 
ITT Population 
 
 
Visit 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
WEEK25 D169 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
receiving minimal or no non-PRN 1008 immunotherapy. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
Source Data: Listing 16.2.4.1.5 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
Repeat for the following populations. 


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Page 48 of 195 
Table 14.2.2.5.1.2 
Proportion of Patients who Achieved Complete Response (CR) at Visit 
mITT Population 
 
 
Table 14.2.2.5.1.3 
Proportion of Patients who Achieved Complete Response (CR) at Visit 
Per Protocol Population 
 


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Page 49 of 195 
 
Table 14.2.2.5.2.1 
Proportion of Patients who Achieved Complete Response (CR) without Prednis(ol)one >0.5 mg/kg at Visit 
ITT Population 
 
 
Visit 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
WEEK25 D169 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
receiving minimal or no non-PRN 1008 immunotherapy. 
Patients who achieved CR at a specific visit and had no occurrence of daily prednis(ol)one >0.5 mg/kg 
on or before the first CR date during the study treatment will be counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
      Only Systemetic Corticosteroids are considered for this table. 
Source Data: Listing 16.2.4.1.5 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
Repeat for the following populations 


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Page 50 of 195 
Table 14.2.2.5.2.2 
Proportion of Patients who Achieved Complete Response (CR) without Prednis(ol)one >0.5 mg/kg at Visit 
mITT Population 
Table 14.2.2.5.2.3 
Proportion of Patients who Achieved Complete Response (CR) without Prednis(ol)one >0.5 mg/kg at Visit 
Per Protocol Population 
 


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Page 51 of 195 
 
Table 14.2.2.5.3.1 
Proportion of Patients who Achieved Complete Response (CR) with Prednis(ol)one >0.5 mg/kg at Visit 
ITT Population 
 
 
Visit 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
WEEK25 D169 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
receiving minimal or no non-PRN 1008 immunotherapy. 
Patients who achieved CR at a specific visit and had daily prednis(ol)one >0.5 mg/kg on or before the 
first CR date during the study treatment will be counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
      Only Systemetic Corticosteroids are considered for this table. 
Source Data: Listing 16.2.4.1.5 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
Repeat for the following populations 


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Page 52 of 195 
Table 14.2.2.5.3.2 
Proportion of Patients who Achieved Complete Response (CR) with Prednis(ol)one >0.5 mg/kg at Visit 
mITT Population 
 
Table 14.2.2.5.3.3 
Proportion of Patients who Achieved Complete Response (CR) with Prednis(ol)one >0.5 mg/kg at Visit 
PP Population 
 


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Page 53 of 195 
 
Table 14.2.2.5.4.1 
Cumulative Incidence of Patients who Achieved Complete Response (CR) by Visit 
ITT Population 
 
 
Visit 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK3 D15 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK5 D29 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK25 D169 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
receiving minimal or no non-PRN 1008 immunotherapy. Cumulative responder rate is used, i.e., any 
patient achieves the response at any study visit is counted into all the later study visits. 
Unscheduled visit are not included in this summary. 
      Patients who ever achieved CR on or before a specific visit during the study treatment will be counted 
as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
Source Data: Listing 16.2.4.1.5 
 


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Page 54 of 195 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog note: Subject who terminated on the first day of treatment without any further follow up is not 
included in this analysis. Further, if a subject drop out, they will still accommodate to future unattended 
visits cumulatively. 
Repeat for following populations: 
Table 14.2.2.5.4.2 
Cumulative Incidence of Patients who Achieved Complete Response (CR) by Visit 
mITT Population 
 
Table 14.2.2.5.4.3 
Cumulative Incidence of Patients who Achieved Complete Response (CR) by Visit 
Per Protocol Population 
 


Principia Biopharma Australia Pty Ltd. 
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Page 55 of 195 
 
Table 14.2.2.5.5.1 
Cumulative Incidence of Patients who Achieved Complete Response (CR) without Prednis(ol)one >0.5 mg/kg by 
Visit 
ITT Analysis Population 
 
 
Visit 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK3 D15 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK5 D29 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK25 D169 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
receiving minimal or no non-PRN 1008 immunotherapy. Cumulative responder rate is used, i.e., any 
patient achieves the response at any study visit is counted into all the later study visits. 
Unscheduled visits are not included in this summary. 
      Patients who ever achieved CR and had no occurrence of daily prednis(ol)one >0.5 mg/kg before the 
first CR date during the study treatment will be counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
      Only Systemetic Corticosteroids are considered for this table. 


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Source Data: Listing 16.2.4.1.5 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Repeat above table for the following 
 
Table 14.2.2.5.5.2 
Cumulative Incidence of Patients who Achieved Complete Response (CR) without Prednis(ol)one >0.5 mg/kg by 
Visit 
mITT Population 
 
Table 14.2.2.5.5.3 
Cumulative Incidence of Patients who Achieved Complete Response (CR) without Prednis(ol)one >0.5 mg/kg by 
Visit 
 Per Protocol Population 
 


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Table 14.2.2.5.6.1 
Cumulative Incidence of Patients who Achieved Complete Response (CR) with Prednis(ol)one >0.5 mg/kg by 
Visit 
ITT Population 
 
 
Visit 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK3 D15 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Cumulative Responder Rate by 
WEEK5 D29 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
… 
 
 
 
 
Cumulative Responder Rate by 
WEEK25 D169 
 
 
 
 
  n (%) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
receiving minimal or no non-PRN 1008 immunotherapy. Cumulative responder rate is used, i.e., any 
patient achieves the response at any study visit is counted into all the later study visits. 
Unscheduled visits are not included in this summary. 
      Patients who ever achieved CR and had daily prednis(ol)one >0.5 mg/kg before a specific visit during 
the study treatment will be counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
      Only Systemetic Corticosteroids are considered for this table. 
Source Data: Listing 16.2.4.1.5 
 


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<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Repeat table above for following populations 
Table 14.2.2.5.6.2 
Cumulative Incidence of Patients who Achieved Complete Response (CR) with Prednis(ol)one >0.5 mg/kg by Visit 
mITT Population 
 
Table 14.2.2.5.6.3 
Cumulative Incidence of Patients who Achieved Complete Response (CR) with Prednis(ol)one >0.5 mg/kg by Visit 
Per Protocol Population 
 
 


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Table 14.2.2.5.7.1 
Duration of Complete Response (CR) Status Among CR Subjects 
ITT Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
receiving minimal or no non-PRN 1008 immunotherapy. 
Patients achieved CR at any visit on or before the Week 13 visit during the study treatment and had no 
occurrence of daily prednis(ol)one >0.5 mg/kg from the first dose taken date up to the day before 
first CR date will be counted as responders. 
Two-sided 80% and 95% confidence intervals are calculated using the Exact (Clopper-Pearson) method. 
Percentages are based on the number of patients in the respective dose levels in the Analysis 
Population. 
      Duration of CR is defined as the longest time subject is known to have had complete response, i.e. the 
earliest of either date of last known CR date or last follow up date – start date of CR + 1. 
Source Data: Listing 16.2.4.1.10 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog note: Early treatment terminators or subjects whose CR status are completely missing are considered 
non-responders. 
Repeat above table for the other populations. 
Table 14.2.2.5.7.2 
Duration of Complete Response (CR) Status Among CR Subjects 
mITT Population 
 
Table 14.2.2.5.7.3 
Duration of Complete Response (CR) Status Among CR Subjects 
Per Protocol Population 
 


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Table 14.2.3.1.1 
Time to First Control of Disease Activity (CDA): Kaplan Meier Estimates 
ITT Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
TIME TO FIRST CDA (days) [1] 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  25% QUARTILE  
xx.x 
xx.x 
xx.x 
xx.x 
    80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
    95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
    80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
    95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  75% QUARTILE 
xx.x 
xx.x 
xx.x 
xx.x 
    80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
    95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  MIN, MAX [2] 
xx, xx 
xx, xx* 
xx, xx 
xx*, xx 
 
 
 
 
 
KAPLAN-MEIER ESTIMATE % (n’) [3] 
 
 
 
 
  15 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  29 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  57 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  85 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  113 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  141 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  169 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. Control of disease activity (CDA) is defined as the time at which new 
lesions cease to form and established lesions begin to heal. 
[1] Time to first CDA = Date of first CDA confirmed – Date of first dose + 1. Patients who did not achieve 
CDA by the date of study completion or discontinuation were censored at the date of completion or 
discontinuation respectively. 80% and 95% confidence intervals are calculated using the LOGLOG 
transformation. 
[2] * indicate that this value is from a censored data. 
[3] Kaplan-Meier estimate of the probability of experiencing the event on or before the specified timepoint 
(n’ = number of patients remaining at risk beyond the specified timepoint). 
Source Data: Listing 16.2.4.1.6 


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<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Repeat the table above with the following population. 
Table 14.2.3.1.2 
Time to First Control of Disease Activity (CDA): Kaplan Meier Estimates 
mITT Population 
 
Table 14.2.3.1.3 
Time to First Control of Disease Activity (CDA): Kaplan Meier Estimates 
Per Protocol Population 
 


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Table 14.2.3.2.1 
Time to First Complete Response (CR): Kaplan Meier Estimates 
ITT Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
TIME TO FIRST CR (days) [1] 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  25% QUARTILE  
xx.x 
xx.x 
xx.x 
xx.x 
    80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
    95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
    80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
    95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  75% QUARTILE 
xx.x 
xx.x 
xx.x 
xx.x 
    80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
    95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  MIN, MAX [2] 
xx, xx 
xx, xx* 
xx, xx 
xx*, xx 
 
 
 
 
 
KAPLAN-MEIER ESTIMATE % (n’) [3] 
 
 
 
 
  15 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  29 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  57 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  85 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  113 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  141 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  169 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. Complete response (CR) is defined as the absence of new or established 
lesions while the patient is receiving minimal or no non-PRN 1008 immunotherapy. 
[1] Time to first CR = Date of first CR confirmed – Date of first dose + 1. Patients who did not achieve CR 
by the date of study completion or discontinuation were censored at the date of completion or 
discontinuation respectively.80% and 95% confidence intervals are calculated using the LOGLOG 
transformation. 
[2] * indicate that this value is from a censored data. 
[3] Kaplan-Meier estimate of the probability of experiencing the event on or before the specified timepoint 
(n’ = number of patients remaining at risk beyond the specified timepoint). 
Source Data: Listing 16.2.4.1.7 


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<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Repeat for the following populations 
Table 14.2.3.2.2 
Time to First Complete Response (CR): Kaplan Meier Estimates 
mITT Population 
 
Table 14.2.3.2.3 
Time to First Complete Response (CR): Kaplan Meier Estimates 
Per Protocol Population 
 


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Table 14.2.3.3.1 
Time to First End of Consolidation Phase: Kaplan Meier Estimates 
ITT Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
TIME TO END OF CONSOLIDATION PHASE 
(days) [1] 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  25% QUARTILE  
xx.x 
xx.x 
xx.x 
xx.x 
    80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
    95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
    80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
    95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  75% QUARTILE 
xx.x 
xx.x 
xx.x 
xx.x 
    80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
    95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  MIN, MAX [2] 
xx, xx 
xx, xx* 
xx, xx 
xx*, xx 
 
 
 
 
 
KAPLAN-MEIER ESTIMATE % (n’) [3] 
 
 
 
 
  15 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  29 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  57 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  85 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  113 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  141 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  169 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. End of consolidation phase: The time at which no new lesions have 
developed for minimum of 2 weeks, approximately 80% of lesions have healed, and when most clinicians 
start to taper steroids. 
[1] Time to end of consolidation phase = Date of end of consolidation phase confirmed – Date of first dose + 
1. Patients who did not achieve end of consolidation phase by the date of study completion or 
discontinuation were censored at the date of completion or discontinuation respectively. 80% and 95% 
confidence intervals are calculated using the LOGLOG transformation. 
[2] * indicate that this value is from a censored data. 
[3] Kaplan-Meier estimate of the probability of experiencing the event on or before the specified timepoint 


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(n’ = number of patients remaining at risk beyond the specified timepoint). 
Source Data: Listing 16.2.4.1.8 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Repeat for the following population 
Table 14.2.3.2.2 
Time to First Complete Response (CR): Kaplan Meier Estimates 
mITT Population 
 
Table 14.2.3.2.3 
Time to First Complete Response (CR): Kaplan Meier Estimates 
Per Protocol Population 


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Table 14.2.3.4.1 
Time to First Relapse after PRN1008 Treatment Completion or Discontinuation: Kaplan Meier Estimates 
ITT Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
PATIENTS WITH CDA AT WEEK 13 
XX 
XX 
XX 
XX 
 
 
 
 
 
TIME TO FIRST RELAPSE (days) [1] 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  25% QUARTILE  
xx.x 
xx.x 
xx.x 
xx.x 
    80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
    95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
    80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
    95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  75% QUARTILE 
xx.x 
xx.x 
xx.x 
xx.x 
    80% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
    95% CONFIDENCE INTERVAL 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
(xx.x, xx.x) 
  MIN, MAX [2] 
xx, xx 
xx, xx* 
xx, xx 
xx*, xx 
 
 
 
 
 
KAPLAN-MEIER ESTIMATE % (n’) [3] 
 
 
 
 
  29 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  57 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
  85 DAYS 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
xx.x (xx) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Relapse/flare: Appearance of >=3 new lesions/month that do not heal spontaneously within 1 week, or by 
extension of established lesions, in a patient who has achieved disease control. 
Only patients with confirmed CDA before treatment completion or discontinuation will be included into 
this analysis. 
[1]   Time to first relapse = Date of first relapse confirmed after Week 13 – Date of last dose + 1. 
Patients who did not achieve relapse between Week 13 visit and the date of study completion or 
discontinuation were censored at the date of completion or discontinuation respectively.80% and 95% 
confidence intervals are calculated using the LOGLOG transformation. 
[2]   * indicate that this value is from a censored data. 
[3]   Kaplan-Meier estimate of the probability of experiencing the event on or before the specified 
timepoint (n’ = number of patients remaining at risk beyond the specified timepoint). 


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Source Data: Listing 16.2.4.1.9 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog note: Repeat for 2) Patients with CR at the Week 13 visit in the same table, and change the first row 
accordingly. 
Repeat the above table with all the subpopulations using the following analysis populations. 
Table 14.2.3.4.2 
Time to First Relapse after PRN1008 Treatment Completion or Discontinuation Among Subjects who Achieved CDA 
at Week 13 Visit: Kaplan Meier Estimates 
mITT Population 
 
 
Table 14.2.3.4.3 
Time to First Relapse after PRN1008 Treatment Completion or Discontinuation Among Subjects who Achieved CDA 
at Week 13 Visit: Kaplan Meier Estimates 
Per Protocol Population 
 
 


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Table 14.2.4.1.1 
Cumulative Corticosteroid Usage over 12 Weeks 
ITT Population 
 
Cumulative Corticosteroid 
Usage (mg) 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
      Patients who have completed at least Week 13 Visit are included. 
All corticosteriods were converted into prednisolone. Each 10 mg of prednisolone is equivalent to 10 
mg of prednisone, 8 mg of methylprednisolone, 1.5 mg dexamethasone, 40 mg hydrocortisone or 1.5 mg 
betamethasone. 
Source Data: Listing 16.2.3.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Repeat the above table using the other populations 
Table 14.2.4.1.2 
Cumulative Corticosteroid Usage over 12 Weeks 
mITT Population 
 
Table 14.2.4.1.3 
Cumulative Corticosteroid Usage over 12 Weeks 
Per Protocol Population 
 


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Table 14.2.4.2.1 
Corticosteroid Usage by Visit 
ITT Population 
 
CUMULATIVE CORTICOSTEROID 
USAGE (mg) 
400 mg 
500 mg 
600 mg 
Total 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
SCREENING 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK1 D1 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
      All corticosteriods were converted into prednisolone. Each 10 mg of prednisolone is equivalent to 10 
mg of prednisone, 8 mg of methylprednisolone, 1.5 mg dexamethasone, 40 mg hydrocortisone or 1.5 mg 
betamethasone. 
      The dosage level at screening and Week 1 day 1 visit is defined as the dose level on the visit day, 
and that on all post baseline visits is defined as the dose on the day before the visit date. 
Source Data: Listing 16.2.3.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
PROG NOTE: If a subject is still enrolled in the study but does not report any CS, then the dosage level is 
set as 0. If a subject is terminated prior to a visit, then this subject is not included. 
 
Repeat the above table with the following populations. 
 
Table 14.2.4.2.2 
Corticosteroid Usage by Visit 
mITT Population 
 


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Table 14.2.4.2.3 
Corticosteroid Usage by Visit 
Per Protocol Population 
 
 


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Page 71 of 195 
 
Table 14.2.5.1.1 
Summary of Actual Value and Change from Baseline in Pemphigus Disease Area Index (PDAI) Total Activity 
Score by Visit 
ITT Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Total activity score = Total skin activity + Total scalp activity + Total mucosa activity. 
Source Data: Listing 16.2.4.2.2 
 


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Page 72 of 195 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
Repeat for the following populations 
Table 14.2.5.1.2 
Summary of Actual Value and Change from Baseline in Pemphigus Disease Area Index (PDAI) Total Activity 
Score by Visit 
mITT Population 
 
Table 14.2.5.1.3 
Summary of Actual Value and Change from Baseline in Pemphigus Disease Area Index (PDAI) Total Activity 
Score by Visit 
Per Protocol Population 
 
 


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Page 73 of 195 
 
Table 14.2.5.2.1 
Summary of Actual Value and Change from Baseline in Pemphigus Disease Area Index (PDAI) Total Damage Score 
by Visit 
ITT Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
  MIN, MAX 
xx, xx 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
  MIN, MAX 
xx, xx 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
  MIN, MAX 
xx, xx 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Total damage score = Total skin damage + Total scalp damage 
Source Data: Listing 16.2.4.2.2 
 


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Page 74 of 195 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
Repeat for the following population. 
Table 14.2.5.2.2 
Summary of Actual Value and Change from Baseline in Pemphigus Disease Area Index (PDAI) Total Damage Score 
by Visit 
mITT Population 
 
Table 14.2.5.2.3 
Summary of Actual Value and Change from Baseline in Pemphigus Disease Area Index (PDAI) Total Damage Score 
by Visit 
Per Protocol Population 
 


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Page 75 of 195 
 
 
Table 14.2.6.1.1 
Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) 
by Visit 
ITT Population 
Score: Skin Involvement Total Score 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Source Data: Listing 16.2.4.3.1 – Listing 16.2.4.3.4 
 


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<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Programmer note: please repeat “Oral Involvement Total Score” and “Total Activity Score” on the new pages. 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
 
Repeat for the following Populations 
Table 14.2.6.1.2 
Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) 
by Visit 
mITT Population 
 
Table 14.2.6.1.3 
Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) 
by Visit 
Per Protocol Population 
 


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Page 77 of 195 
 
Table 14.2.6.2.1 
Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) 
by Visit for Patients with Baseline Oral Involvement Total Score Greater than Zero 
ITT Population 
Score: Skin Involvement Total Score 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
 
Source Data: Listing 16.2.4.3.1 – Listing 16.2.4.3.4 
 


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Page 78 of 195 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Programmer note: please repeat “Oral Involvement Total Score”, “Severity Total Score”, “Total Activity Score”, 
and “Total ABSIS Score” on the new pages. 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
Repeat for the following populations: 
Table 14.2.6.2.2 
Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) 
by Visit for Patients with Baseline Oral Involvement Total Score Greater than Zero 
mITT Population 
 
 
Table 14.2.6.2.3 
Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) 
by Visit for Patients with Baseline Oral Involvement Total Score Greater than Zero 
Per Protocol Population 
 


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Page 79 of 195 
 
Table 14.2.6.3.1 
Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) 
by Visit for Patients with Baseline Oral Involvement Total Score Equal to Zero 
ITT Population 
Score: Skin Involvement Total Score 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
 
Source Data: Listing 16.2.4.3.1 – Listing 16.2.4.3.4 
 


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Page 80 of 195 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Programmer note: please repeat “Oral Involvement Total Score” and “Total Activity Score” on the new pages. 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
Repeat for the following populations: 
Table 14.2.6.3.2 
Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) 
by Visit for Patients with Baseline Oral Involvement Total Score Equal to Zero 
mITT Population 
 
Table 14.2.6.3.3 
Summary of Actual Value and Change from Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) 
by Visit for Patients with Baseline Oral Involvement Total Score Equal to Zero 
Per Protocol Population 
 


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Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 81 of 195 
 
 
Table 14.2.7.1 
Summary of Actual Value and Change from Baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL) 
Score by Visit 
ITT Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Source Data: Listing 16.2.4.4.1 and Listing 16.2.4.4.2 


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<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
 
Repeat for the following populations 
Table 14.2.7.2 
Summary of Actual Value and Change from Baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL) 
Score by Visit 
mITT Population 
 
Table 14.2.7.3 
Summary of Actual Value and Change from Baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL) 
Score by Visit 
Per Protocol Population 
 


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Page 83 of 195 
 
Table 14.2.8.1 
Summary of Actual Value and Change from Baseline in Treatment of Autoimmune Bullous Disease Quality of Life 
(TABQOL) Total Score by Visit 
ITT Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Source Data: Listing 16.2.4.5.1 and Listing 16.2.4.5.2 
 


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Page 84 of 195 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
Repeat for the following populations 
Table 14.2.8.2 
Summary of Actual Value and Change from Baseline in Treatment of Autoimmune Bullous Disease Quality of Life 
(TABQOL) Total Score by Visit 
mITT Population 
 
Table 14.2.8.3 
Summary of Actual Value and Change from Baseline in Treatment of Autoimmune Bullous Disease Quality of Life 
(TABQOL) Total Score by Visit 
Per Protocol Population 
 


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PRN1008-005 
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Page 85 of 195 
 
Table 14.2.9.1 
Summary of Actual Value and Change from Baseline in Simplified Nutritional Appetite Questionnaire (SNAQ) 
Score by Visit 
ITT Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
  MIN, MAX 
xx, xx 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
  MIN, MAX 
xx, xx 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
xx.x (xx.xx) 
  MEDIAN 
xx.x 
xx.x 
xx.x 
xx.x 
  MIN, MAX 
xx, xx 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Source Data: Listing 16.2.4.6.1 and Listing 16.2.4.6.2 
 


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PRN1008-005 
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Page 86 of 195 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 
Repeat for the following populations: 
Table 14.2.9.2 
Summary of Actual Value and Change from Baseline in Simplified Nutritional Appetite Questionnaire (SNAQ) 
Score by Visit 
mITT Population 
Table 14.2.9.3 
Summary of Actual Value and Change from Baseline in Simplified Nutritional Appetite Questionnaire (SNAQ) 
Score by Visit 
Per Protocol Population 
 


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Page 87 of 195 
Table 14.2.10.1.1 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by Age 
Group 
ITT Population 
Prog Note: Repeat Table 14.2.2.4.5 with the addition of page variable Age (ı 50, < 50).  
 
 
Repeat for the following populations. 
Table 14.2.10.1.2 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by Age 
Group 
mITT Population 
 
 
Table 14.2.10.1.3 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by Age 
Group 
Per Protocol Population 
 
 
Table 14.2.10.2.1 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Gender 
ITT Population 
Prog Note: Repeat Table 14.2.2.4.5 with the addition of page variable female male.  
 
 
Repeat for the following populations. 
Table 14.2.10.2.2 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Gender 
mITT Population 
 
 
Table 14.2.10.2.3 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Gender 
Per Protocol Population 
 
 


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Page 88 of 195 
Table 14.2.10.3.1 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Baseline Total Anti-DSG Category 
ITT Population 
Prog Note: Repeat Table 14.2.2.4.5 with the addition of page variable baseline total anti-DSG (ı 100, < 100).  
 
 
Repeat for the following populations. 
Table 14.2.10.3.2 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Baseline Total Anti-DSG Category 
mITT Population 
 
 
Table 14.2.10.3.3 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Baseline Total Anti-DSG Category 
Per Protocol Population 
 
Table 14.2.10.4.1 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Pemphigus History Type 
ITT Population 
Prog Note: Repeat Table 14.2.2.4.5 with the addition of page variable Pemphigus History Type (Newly Diagnosed 
vs Relapsed).  
Add footnote: Newly diagnosed subjects are defined as diagnostic date being less than 6 months to Screening 
date, otherwise a subject is defined as relapsed. 
 
 
Repeat for the following populations. 
Table 14.2.10.4.2 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Pemphigus History Type 
mITT Population 
 
 
Table 14.2.10.4.3 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Pemphigus History Type 
Per Protocol Population 
 


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Page 89 of 195 
 
Table 14.2.10.5.1 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Pemphigus Anti-DSG Profile 
ITT Population 
Prog Note: Repeat Table 14.2.2.4.5 with the addition of page variable anti-dsg1 positive vs. anti-dsg1 positive 
anti-dag3 negative vs. neither positive.  
 
Repeat for the following populations. 
Table 14.2.10.5.2 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Pemphigus Anti-DSG Profile 
mITT Population 
 
 
Table 14.2.10.5.3 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Pemphigus Anti-DSG Profile 
Per Protocol Population 
 
 
Table 14.2.10.6.1 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Pemphigus Anti-DSG Profile 
ITT Population 
Prog Note: Repeat Table 14.2.2.4.5 with the addition of page variable Pemphigus Severity: mild vs moderate.  
Add footnote: Mild Pemphigus subject is defined as screening PDAI < 15, otherwise moderate. 
 
 
Repeat for the following populations. 
Table 14.2.10.6.2 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Pemphigus Anti-DSG Profile 
mITT Population 
 
 
Table 14.2.10.6.3 
Cumulative Incidents of Control of Disease Activity (CDA) without Predniso(lo)ne > 0.5mg/kg by Visit by 
Pemphigus Anti-DSG Profile 
Per Protocol Population 


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Table 14.3.1.1 
Overall Summary of Treatment-Emergent Adverse Events 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
n (%) [E] 
n (%)[E] 
n (%)[E] 
n (%)[E] 
 
 
 
 
 
ANY TREATMENT-EMERGENT ADVERSE EVENT 
xx (xx.x) xx 
xx (xx.x) xx 
xx (xx.x) xx 
xx (xx.x) xx 
 
 
 
 
 
ANY SERIOUS TREATMENT-EMERGENT ADVERSE 
  EVENT 
xx (xx.x) xx 
xx (xx.x) xx 
xx (xx.x) xx 
xx (xx.x) xx 
 
 
 
 
 
ANY STUDY DRUG-RELATED TREATMENT-EMERGENT 
  ADVERSE EVENT 
xx (xx.x) xx 
xx (xx.x) xx 
xx (xx.x) xx 
xx (xx.x) xx 
 
 
 
 
 
ANY STUDY DRUG-RELATED TREATMENT-EMERGENT 
  SERIOUS ADVERSE EVENT 
xx (xx.x) xxx 
xx (xx.x) xxx 
xx (xx.x) xxx 
xx (xx.x) xxx 
 
 
 
 
 
ANY TREATMENT-EMERGENT ADVERSE EVENT 
  LEADING TO TREATMENT DISCONTINUATION 
xx (xx.x) xx 
xx (xx.x) xx 
xx (xx.x) xx 
xx (xx.x) xx 
 
 
 
 
 
ANY TREATMENT-EMERGENT ADVERSE EVENT  
  LEADING TO DEATH 
xx (xx.x) xx 
xx (xx.x) xx 
xx (xx.x) xx 
xx (xx.x) xx 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
A treatment-emergent AE (TEAE) is defined as an AE that begins on or after the first study drug 
taken. 
n: number of patients at each level of summarization. 
[E]: number of events at each level of summarization. 
Source Data: Listing 16.2.5.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
  


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Table 14.3.1.2 
Treatment-Emergent Adverse Events by System Organ Class and Preferred Term 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
System Organ Class 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
  Preferred Term 
n (%) [E]  
n (%) [E]  
n (%) [E]  
n (%) [E]  
 
 
 
 
 
ALL TREATMENT-EMERGENT ADVERSE 
  EVENTS 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
 
 
 
 
 
SYSTEM ORGAN CLASS #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  … 
 
 
 
 
 
 
 
 
 
SYSTEM ORGAN CLASS #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  … 
 
 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
At each level of patient summarization, a patient is counted once if the patient reported one or more 
events. 
n: number of patients at each level of summarization. 
[E]: number of events at each level of summarization. 
Adverse Events were coded using MedDRA, Version 18.0. 
Source Data: Listing 16.2.5.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: AEs will be sorted in descending order of frequency of SOC based on the total of all treatment 
groups. Within each SOC, Preferred terms will also be sorted in descending order. 
  


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Table 14.3.1.3 
Treatment-Emergent Adverse Events by Relationship to Study Drug 
Safety Analysis Population 
 
 
400 mg (N=xx) 
System Organ Class 
Not Related 
Related 
Total 
  Preferred Term 
n (%) [E] 
n (%) [E] 
n (%) [E] 
 
 
 
 
ALL TREATMENT-EMERGENT ADVERSE 
  EVENTS 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
 
 
 
 
SYSTEM ORGAN CLASS #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #3 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  … 
 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
At each level of patient summarization, a patient is counted once if the patient reported one or more 
events. If the relationship of an AE is missing, the AE is imputed as “Related”. 
n: number of patients at each level of summarization. 
[E]: number of events at each level of summarization. 
Adverse Events were coded using MedDRA, Version 18.0. 
Source Data: Listing 16.2.5.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: Include 500 mg, 600 mg, Total on the subsequent pages. AEs will be sorted in descending order of 
frequency of SOC based on the total of all treatment groups. Within each SOC, Preferred terms will also be 
sorted in descending order. 


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Table 14.3.1.4 
Treatment-Emergent Adverse Events by Severity 
Safety Analysis Population 
 
 
400 mg (N=xx) 
System Organ Class 
Grade 1  
Grade 2  
Grade 3  
Grade 4  
Grade 5  
Total 
  Preferred Term 
n (%) 
n (%) 
n (%) 
n (%) 
n (%) 
n (%) 
 
 
 
 
 
 
 
TOTAL NUMBER OF TREATMENT- 
  EMERGENT ADVERSE EVENTS 
xx 
xx 
xx 
xx 
xx 
xx 
 
 
 
 
 
 
 
NUMBER OF PATIENTS 
  WITH AT LEAST ONE TREATMENT- 
  EMERGENT ADVERSE EVENT 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
 
SYSTEM ORGAN CLASS #1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #3 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  … 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
At each level of patient summarization, a patient is counted once for the most severe event if the 
patient reported one or more events. If the severity of an AE is missing, the AE is imputed as “Grade 
3”. 
Adverse Events were coded using MedDRA, Version 18.0. 
Source Data: Listing 16.2.5.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Include 500 mg, 600 mg and Total on the subsequent pages AEs will be sorted in descending order 
of frequency of SOC based on the total of all treatment groups. Within each SOC, Preferred terms will also 
be sorted in descending order. 
  


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Table 14.3.1.5 
Treatment Related Treatment-Emergent Adverse Events by Severity 
Safety Analysis Population 
 
 
400 mg (N=xx) 
System Organ Class 
Grade 1  
Grade 2  
Grade 3  
Grade 4  
Grade 5  
Total 
  Preferred Term 
n (%) 
n (%) 
n (%) 
n (%) 
n (%) 
n (%) 
 
 
 
 
 
 
 
TOTAL NUMBER OF TREATMENT 
RELATED TREATMENT- 
  EMERGENT ADVERSE EVENTS 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
 
NUMBER OF PATIENTS 
  WITH AT LEAST ONE TREATMENT- 
  EMERGENT ADVERSE EVENT 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
 
SYSTEM ORGAN CLASS #1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  PREFERRED TERM #3 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  … 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
At each level of patient summarization, a patient is counted once for the most severe event if the 
patient reported one or more events. If the severity of an AE is missing, the AE is imputed as “Grade 
3”. 
Adverse Events were coded using MedDRA, Version 18.0. 
Source Data: Listing 16.2.5.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Include 500 mg, 600 mg and Total on the subsequent pages AEs will be sorted in descending order 
of frequency of SOC based on the total of all treatment groups. Within each SOC, Preferred terms will also 
be sorted in descending order. 
 


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Table 14.3.2.1 
Serious Treatment-Emergent Adverse Events by System Organ Class and Preferred Term 
Safety Analysis Population 
 
 
400 mg 
500 mg 
600 mg 
Total 
System Organ Class 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
  Preferred Term 
n (%) [E] 
n (%) [E] 
n (%)[E] 
n (%)[E] 
 
 
 
 
 
ALL SERIOUS TREATMENT-EMERGENT 
  ADVERSE EVENT 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
 
 
 
 
 
SYSTEM ORGAN CLASS #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  … 
 
 
 
 
 
 
 
 
 
SYSTEM ORGAN CLASS #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  … 
 
 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
At each level of patient summarization, a patient is counted once if the patient reported one or more 
events. 
n: number of patients at each level of summarization. 
[E]: number of events at each level of summarization. 
Adverse Events were coded using MedDRA, Version 18.0. 
Source Data: Listing 16.2.5.2 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: AEs will be sorted in descending order of frequency of SOC based on the total of all treatment 
groups. Within each SOC, Preferred terms will also be sorted in descending order. 
  


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Table 14.3.2.2 
Serious Treatment-Emergent Adverse Events by Relationship to Study Drug 
Safety Analysis Population 
 
 
400 mg (N=xx) 
System Organ Class 
Not Related 
Related 
Total 
  Preferred Term 
n (%) [E] 
n (%) [E] 
n (%) [E] 
 
 
 
 
ALL SERIOUS TREATMENT-EMERGENT 
 ADVERSE EVENTS 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
 
 
 
 
SYSTEM ORGAN CLASS #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #3 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  … 
 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
At each level of patient summarization, a patient is counted once if the patient reported one or more 
events. If the relationship of an AE is missing, the AE is imputed as “Related”. 
n: number of patients at each level of summarization. 
[E]: number of events at each level of summarization. 
Adverse Events were coded using MedDRA, Version 18.0. 
Source Data: Listing 16.2.5.2 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: Include 600 mg, Total on the subsequent pages. AEs will be sorted in descending order of 
frequency of SOC based on the total of all treatment groups. Within each SOC, Preferred terms will also be 
sorted in descending order. 


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Table 14.3.2.3 
Treatment-Emergent Adverse Events Leading to Treatment Discontinuation 
Safety Analysis Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
System Organ Class 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
  Preferred Term 
n (%) [E]  
n (%) [E]  
n (%) [E]  
n (%) [E]  
 
 
 
 
 
ALL TREATMENT-EMERGENT ADVERSE EVENTS  
  LEADING TO TREATMENT DISCONTINUATION 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
 
 
 
 
 
SYSTEM ORGAN CLASS #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  … 
 
 
 
 
 
 
 
 
 
SYSTEM ORGAN CLASS #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  … 
 
 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
At each level of patient summarization, a patient is counted once if the patient reported one or more 
events. 
n: number of patients at each level of summarization. 
[E]: number of events at each level of summarization. 
Adverse Events were coded using MedDRA, Version 18.0. 
Source Data: Listing 16.2.5.3 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: AEs will be sorted in descending order of frequency of SOC based on the total of all treatment 
groups. Within each SOC, Preferred terms will also be sorted in descending order. 
 


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Table 14.3.2.4 
Treatment-Emergent Adverse Events Leading to Death 
Safety Analysis Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
System Organ Class 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
  Preferred Term 
n (%) [E]  
n (%) [E]  
n (%) [E]  
n (%) [E]  
 
 
 
 
 
ALL TREATMENT-EMERGENT ADVERSE EVENTS  
  LEADING TO DEATH 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
 
 
 
 
 
SYSTEM ORGAN CLASS #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  … 
 
 
 
 
 
 
 
 
 
SYSTEM ORGAN CLASS #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #1 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  PREFERRED TERM #2 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
xx (xx.x) [xx] 
  … 
 
 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
At each level of patient summarization, a patient is counted once if the patient reported one or more 
events. 
n: number of patients at each level of summarization. 
[E]: number of events at each level of summarization. 
Adverse Events were coded using MedDRA, Version 18.0. 
Source Data: Listing 16.2.5.4 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: AEs will be sorted in descending order of frequency of SOC based on the total of all treatment 
groups. Within each SOC, Preferred terms will also be sorted in descending order. 
 


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Table 14.3.3.1 
Summary of Actual Value and Change from Baseline in Chemistry Tests by Visit 
Safety Analysis Population 
 
Parameter: LAB TEST PARAMETER #1 (UNIT) 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Source Data: Listing 16.2.6.1 
 


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<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Programming Note: Please include “Unscheduled” as the last visit if there are records with unscheduled visit as 
a visit label.


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Table 14.3.3.2 
Summary of Actual Value and Change from Baseline in Clinical Laboratory Tests Monitored for Drug Induced 
Liver Injury (DILI) by Visit 
Safety Analysis Population 
 
 
Parameter: LAB TEST PARAMETER #1 (UNIT) 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 


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Clinical laboratory tests monitored for drug induced liver injury (DILI) include ALT (alanine 
aminotransferase or SGPT), AST (aspartate transaminase or SGOT), TBL (total bilirubin), and ALP 
(alkaline phosphatase). 
Source Data: Listing 16.2.6.2 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Programming Note: Please include “Unscheduled” as the last visit if there are records with unscheduled 
visit as a visit label. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 103 of 195 
 
Table 14.3.3.3 
Summary of Actual Value and Change from Baseline in Hematology Tests by Visit 
Safety Analysis Population 
 
Parameter: LAB TEST PARAMETER #1 (UNIT) 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Source Data: Listing 16.2.6.3 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 104 of 195 
Programming Note: Please include “Unscheduled” as the last visit if there are records with unscheduled visit as 
a visit label.


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 105 of 195 
 
Table 14.3.3.4 
Summary of Actual Value and Change from Baseline in Coagulation Tests by Visit 
Safety Analysis Population 
 
Parameter: LAB TEST PARAMETER #1 (UNIT) 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK5 D29 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Source Data: Listing 16.2.6.4 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 106 of 195 
Programming Note: Please include “Unscheduled” as the last visit if there are records with unscheduled visit as 
a visit label.


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 107 of 195 
 
Table 14.3.3.5 
Summary of Urinalysis Tests at Screening 
Safety Analysis Population 
 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
 
Laboratory Test 
(N=xx) 
n (%) 
(N=xx) 
n (%) 
(N=xx) 
n (%) 
(N=xx) 
n (%) 
 
 
 
 
 
LAB TEST PARAMETER #1 
 
 
 
 
  CATEGORY 1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  CATEGORY 2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  … 
 
 
 
 
 
 
 
 
 
LAB TEST PARAMETER #2 
(UNIT) 
 
 
 
 
  CATEGORY 1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  CATEGORY 2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  … 
 
 
 
 
 
 
 
 
 
LAB TEST PARAMETER #3 
(UNIT)  
 
 
 
 
  n 
xx 
xx 
xx 
xx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Percentages are based on the number of patients with data at each visit of the respective dose 
levels.  
Source Data: Listing 16.2.6.5 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Programming Note: Please include “Unscheduled” as the last visit if there are records with unscheduled 
visit as a visit label. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 108 of 195 
 
Table 14.3.3.6 
Shift from Baseline to Post-baseline Visits in Normality Status of Chemistry Tests 
Safety Analysis Population 
 
Parameter: LAB TEST #1 (UNIT) 
 
400 mg (N=xx) 
 
500 mg (N=xx) 
 
Baseline 
 
Baseline 
Visit 
Low 
n (%) 
Normal 
n (%) 
High 
n (%) 
Total 
n (%) 
 
Low 
n (%) 
Normal 
n (%) 
High 
n (%) 
Total 
n (%) 
 
 
 
 
 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
 
 
 
 
 
  LOW 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  HIGH 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  TOTAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
 
 
 
 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
 
 
 
 
 
  LOW 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  HIGH 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  TOTAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Only patients with non-missing values at both baseline and respective post-baseline visit will be 
counted in the summary table. 
Percentages are based on the number of patients with data at each post-baseline visit of the 
respective dose levels. 
Source Data: Listing 16.2.6.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMONYYYY hh:mm 
 
Prog Note: Include 600 mg, Total on the subsequent pages. Lab test parameters will be displayed in the same 
order as the lab test listing.  
Programming Note: Please include “Unscheduled” as the last visit if there are records with unscheduled 
visit as a visit label. 
In the case where Lower or higher upper ranges are not available for a specific parameter, the lower and 
higher categories will be populated with N/A. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 109 of 195 
 
Table 14.3.3.7 
Shift from Baseline to Post-baseline Visits in Normality Status of Clinical Laboratory Tests Monitored for 
Drug Induced Liver Injury (DILI) 
Safety Analysis Population 
 
Parameter: LAB TEST #1 (UNIT) 
 
400 mg (N=xx) 
 
500 mg (N=xx) 
 
Baseline 
 
Baseline 
Visit 
Low 
n (%) 
Normal 
n (%) 
High 
n (%) 
Total 
n (%) 
 
Low 
n (%) 
Normal 
n (%) 
High 
n (%) 
Total 
n (%) 
 
 
 
 
 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
 
 
 
 
 
  LOW 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  HIGH 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  TOTAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
 
 
 
 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
 
 
 
 
 
  LOW 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  HIGH 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  TOTAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Only patients with non-missing values at both baseline and respective post-baseline visit will be 
counted in the summary table. 
Clinical laboratory tests monitored for drug induced liver injury (DILI) include ALT (alanine 
aminotransferase or SGPT), AST (aspartate transaminase or SGOT), TBL (total bilirubin), and ALP 
(alkaline phosphatase). 
Percentages are based on the number of patients with data at each post-baseline visit of the 
respective dose levels. 
Source Data: Listing 16.2.6.2 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMONYYYY hh:mm 
Prog Note: Include 600 mg, Total on the subsequent pages. Lab test parameters will be displayed in the same 
order as the lab test listing.  
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 110 of 195 
In the case where Lower or higher upper ranges are not available for a specific parameter, the lower and 
higher categories will be populated with N/A. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 111 of 195 
 
Table 14.3.3.8 
Shift from Baseline to Post-baseline Visits in Normality Status of Hematology Tests 
Safety Analysis Population 
 
Parameter: LAB TEST #1 (UNIT) 
 
400 mg (N=xx) 
 
500 mg (N=xx) 
 
Baseline 
 
Baseline 
Visit 
Low 
n (%) 
Normal 
n (%) 
High 
n (%) 
Total 
n (%) 
 
Low 
n (%) 
Normal 
n (%) 
High 
n (%) 
Total 
n (%) 
 
 
 
 
 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
 
 
 
 
 
  LOW 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  HIGH 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  TOTAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
 
 
 
 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
 
 
 
 
 
  LOW 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  HIGH 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  TOTAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Only patients with non-missing values at both baseline and respective post-baseline visit will be 
counted in the summary table. 
Percentages are based on the number of patients with data at each post-baseline visit of the 
respective dose levels. 
Source Data: Listing 16.2.6.3 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMONYYYY hh:mm 
 
Prog Note: Include 600 mg, Total on the subsequent pages. Lab test parameters will be displayed in the same 
order as the lab test listing.  
Please include “Unscheduled” as the last visit if there are records with unscheduled visit as a visit 
label. 
In the case where Lower or higher upper ranges are not available for a specific parameter, the lower and 
higher categories will be populated with N/A. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 112 of 195 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 113 of 195 
 
Table 14.3.3.9 
Shift from Baseline to Post-baseline Visits in Normality Status of Coagulation Tests 
Safety Analysis Population 
 
Parameter: LAB TEST #1 (UNIT) 
 
400 mg (N=xx) 
 
500 mg (N=xx) 
 
Baseline 
 
Baseline 
Visit 
Low 
n (%) 
Normal 
n (%) 
High 
n (%) 
Total 
n (%) 
 
Low 
n (%) 
Normal 
n (%) 
High 
n (%) 
Total 
n (%) 
 
 
 
 
 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
 
 
 
 
 
  LOW 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  HIGH 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  TOTAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
 
 
 
 
 
 
 
 
 
WEEK5 D29 
 
 
 
 
 
 
 
 
 
  LOW 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  HIGH 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  TOTAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx 
 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Only patients with non-missing values at both baseline and respective post-baseline visit will be 
counted in the summary table. 
Percentages are based on the number of patients with data at each post-baseline visit of the 
respective dose levels. 
Source Data: Listing 16.2.6.4 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMONYYYY hh:mm 
 
Prog Note: Include 600 mg, Total on the subsequent pages. Lab test parameters will be displayed in the same 
order as the lab test listing.  
In the case where Lower or higher upper ranges are not available for a specific parameter, the lower and 
higher categories will be populated with N/A. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 114 of 195 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 115 of 195 
 
 
Table 14.3.4.1 
Summary of Actual Value and Change from Baseline in Vital Signs by Visit 
Safety Analysis Population 
 
Parameter: VITAL SIGN PARAMETER #1 (UNIT) 
 
400 mg 
500 mg 
600 mg 
Total 
Visit 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
BASELINE 
 
 
 
 
  n 
xxx 
xxx 
xxx 
xxx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK1 D2 
 
 
 
 
  n 
xxx 
xxx 
xxx 
xxx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK1 D2 
 
 
 
 
  n 
xxx 
xxx 
xxx 
xxx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
WEEK 3 D15 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
CHANGE FROM BASELINE  
  AT WEEK3 D15 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Source Data: Listing 16.2.6.7 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 116 of 195 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Programming Note: Please include “Unscheduled” as the last visit if there are records with unscheduled 
visit as a visit label. 
 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 117 of 195 
 
Table 14.3.4.2 
Summary of Blood Pressure and Heart Rate Outside Normal Range 
Safety Analysis Population 
 
Parameter: VITAL SIGN PARAMETER #1 (UNIT) 
 
400 mg 
 
500 mg 
 
(N=xx) 
 
(N=xx) 
Visit 
n 
Low 
Normal 
High 
 
n 
Low 
Normal 
High 
 
 
 
 
 
  
 
 
 
BASELINE 
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
WEEK1 D2 
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
WEEK3 D15 
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
WEEK5 D29 
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
WEEK9 D57 
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
WEEK13 D85 
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
WEEK17 D113 
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
WEEK21 D141 
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
WEEK25 D169 
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x)  
xx 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Systolic blood pressure values <90 or >140 mmHg, diastolic blood pressure values <60 or >80 mmHg, 
heart rate <40 or >100 beats per minute will be counted as Low or High, respectively. 
Percentages are based on number of patients with non-missing values in each visit. 
Source Data: Listing 16.2.6.7 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMONYYYY hh:mm 
 
 Prog Note: Include 600 mg and Total on the subseqent pages. 
 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 118 of 195 
 
 
Table 14.3.5.1 
Summary of Physical Examination at Screening Visit 
Safety Analysis Population 
 
 
 
Body System 
400 mg 
(N=xx) 
n (%) 
500 mg 
(N=xx) 
n (%) 
600 mg 
(N=xx) 
n (%) 
Total 
(N=xx) 
n (%) 
 
 
 
 
 
CRITERIA #1 
 
 
 
 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  ABNORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NOT DONE 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
CRITERIA #2 
 
 
 
 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  ABNORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NOT DONE 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
CRITERIA #3 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Source Data: Listing 16.2.6.8 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: physical examination at screening visit include: general appearance; skin; eyes, ears, nose, 
throat; heart; chest/breast; abdomen; neurologycal; lymphnodes; skeletal; other. 


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Table 14.3.5.2 
Summary of Physical Examination in Post Screening Visit 
Safety Analysis Population 
 
Body System: CRITERIA #1 
 
 
 
Visit 
400 mg 
(N=xx) 
n (%) 
500 mg 
(N=xx) 
n (%) 
600 mg 
(N=xx) 
n (%) 
Total 
(N=xx) 
n (%) 
 
 
 
 
 
BASELINE 
 
 
 
 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  ABNORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NOT DONE 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
WEEK3 D15 
 
 
 
 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  ABNORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NOT DONE 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Source Data: Listing 16.2.6.8 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: physical examination after screening visit include: general appearance; abdomen; 
cardiorespiratory; other. 
Prog Note: For all efficacy and safety by visit summaries, add “unscheduled” visit after other visits if such 
data exist in the database. 


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Table 14.3.6.1 
Summary of Electrocardiogram at Screening Visit 
Safety Analysis Population 
 
 
 
400 mg 
500 mg 
600 mg 
Total 
Parameter 
(N=xx) 
(N=xx) 
(N=xx) 
(N=xx) 
 
 
 
 
 
Ventricular Rate (BEATS/MIN) 
 
 
 
 
  n 
xxx 
xxx 
xxx 
xxx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
PR Interval (msec) 
 
 
 
 
  n 
xxx 
xxx 
xxx 
xxx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
QRS Duration (msec) 
 
 
 
 
  n 
xxx 
xxx 
xxx 
xxx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
QT Interval (msec) 
 
 
 
 
  n 
xxx 
xxx 
xxx 
xxx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
QTcF Interval (msec) 
 
 
 
 
  n 
xxx 
xxx 
xxx 
xxx 
  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
QTcB Interval (msec) 
 
 
 
 
  n 
xxx 
xxx 
xxx 
xxx 


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  MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
  MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
  MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
Normal Sinus Rhythm, n (%) 
 
 
 
 
  YES 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  NO 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
Interpretation, n (%) 
 
 
 
 
  NORMAL 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  ABNORMAL NOT CLINICALLY  
    SIGNIFICANT 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
  ABNORMAL CLINICALLY  
    SIGNIFICANT 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
Source Data: Listing 16.2.6.9 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 


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Table 14.4.1.1 
Summary of Actual Value and Change from Baseline in Anti-DSG by Visit 
Safety Analysis Population 
 
Repeat table 14.3.3.1, for Anti-DSG 1, Anti-DSG 3 and Total Anti-DSG, which is defined as Anti-DSG 1+Anti-DSG 
3. The unit is to be shown as “unit(s)”.  
If either Anti-DSG 1 or 3 is missing, then total Anti-DSG is set as missing. 
Missing values are not imputed and not included.   
Only include visits where Anti-DSG are supposed to be measured per protocol, i.e. day 1, week 3, 5, 9, 13, 17, 
21 and 25.  
Percentage is based on the number of subject with non-missing data for both baseline and post-baseline visit in 
the parameter of interest. 
This analysis will be performed only using Anti-DSG 1 and Anti-DSG 3 data above the limit of quantification, 
and for Total Anti-DSG, only use samples where at least one of Anti-DSG 1 or 3 is above the limit of 
quantification at baseline Anti-DSG value is available for each given visit. 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
      Total Anti-DSG is defined as the sum of Anti-DSG 1 and 3, and is set to missing if either is missing. 
Source Data: Listing 16.2.7.3 
 


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Table 14.4.2.1 
Change from Baseline in Anti-DSG Status by CDA status at Week 5 without Prednis(ol)one >0.5 mg/kg  
ITT Population 
CDA BY WEEK 5: YES 
ANTI-DSG PARAMETER 
  TIME POINT 
    Statistics 
400mg 
500mg 
600mg 
Total 
 
(N=xx) 
(N=xx) 
 
(N=xx) 
(N=xx) 
Anti-DSG 1 (unit) 
 
 
 
 
  BASELINE   
 
 
 
 
    n 
xx 
xx 
xx 
xx 
    MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
    MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
    MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
  WEEK5 D29  
 
 
 
 
    n 
xx 
xx 
xx 
xx 
    MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
    MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
    MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
  CHANGE FROM BASELINE AT WEEK5 D29  
 
 
 
 
    n 
xx 
xx 
xx 
xx 
    MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
    MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
    MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Control of disease activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. 
Patients who achieved CDA at the Week 5 visit and had no occurrence of daily prednis(ol)one >0.5 mg/kg 
before the the first CDA date during the study treatment will be counted as responders. 
Total Anti-DSG is defined as the sum of Anti-DSG 1 and 3, and is set to missing if either is missing. 
Source Data: Listing 16.2.4.1.1, 16.2.7.3 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: repeat for Anti-DSG 3 and Total Anti-DSG. 
PROG NOTE: Repeat for CDA BY WEEK 5 VISIT: No AND OVERALL. 


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          This analysis will be performed only using Anti-DSG 1 and Anti-DSG 3 data above the limit of 
quantification, and for Total Anti-DSG, only use samples where at least one of Anti-DSG 1 or 3 is above the 
limit of quantification at baseline and the Week 5 visit Anti-DSG value is available. 
 


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Table 14.4.2.2 
Change from Baseline in Anti-DSG Status by CR status at Week 13 without Prednis(ol)one >0.5 mg/kg  
ITT Population 
CR STATUS BY WEEK 13: YES 
ANTI-DSG PARAMETER 
  TIME POINT 
    Statistics 
400mg 
500mg 
600mg 
Total 
 
(N=xx) 
(N=xx) 
 
(N=xx) 
(N=xx) 
Anti-DSG 1 (unit) 
 
 
 
 
  BASELINE   
 
 
 
 
    n 
xx 
xx 
xx 
xx 
    MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
    MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
    MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
  WEEK13 D85  
 
 
 
 
    N 
xx 
xx 
xx 
xx 
    MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
    MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
    MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
  CHANGE FROM BASELINE AT WEEK13 D85  
 
 
 
 
    N 
xx 
xx 
xx 
xx 
    MEAN (SD) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
xxx.x (xxx.xx) 
    MEDIAN 
xxx.x 
xxx.x 
xxx.x 
xxx.x 
    MIN, MAX 
xxx, xxx 
xxx, xxx 
xxx, xxx 
xxx, xxx 
 
 
 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
receiving minimal or no non-PRN 1008 immunotherapy. 
Patients who achieved CR at the Week 13 visit and had no occurrence of daily prednis(ol)one >0.5 mg/kg 
on or before the CR date during the study treatment will be counted as responders. 
Total Anti-DSG is defined as the sum of Anti-DSG 1 and 3, and is set to missing if either is missing. 
Source Data: Listing 16.2.4.1.1, 16.2.7.3 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
Prog Note: repeat for Anti-DSG 3 and Total Anti-DSG. This analysis will be performed only using Anti-DSG 1 
and Anti-DSG 3 data above the limit of quantification, and for Total Anti-DSG, only use samples where at 
least one of Anti-DSG 1 or 3 is above the limit of quantification at baseline and the Week 5 visit Anti-DSG 
value is available. 


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Figure 14.2.3.1.1 
Kaplan Meier Plots for Time to First Control of Disease Activity (CDA) 
ITT Population 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. Control of disease activity (CDA) is defined as the time at 
which new lesions cease to form and established lesions begin to heal. 
Time to first CDA = Date of first CDA confirmed – Date of first dose + 1.  
Patients who have not achieved CDA by the date of study completion or discontinuation will be 
censored at the date of completion or discontinuation. 
Source Data: Table 14.2.3.1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog NOTE: Please also add “all dose levels” in this plot. 
Repeat for the following populations 


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Figure 14.2.3.1.2 
Kaplan Meier Plots for Time to First Control of Disease Activity (CDA) 
mITT Population 
 
Figure 14.2.3.1.3 
Kaplan Meier Plots for Time to First Control of Disease Activity (CDA) 
PP Population 
 


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Figure 14.2.3.2.1 
Kaplan Meier Plots for Time to First Complete Response (CR)  
ITT Population 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. Complete response (CR) is defined as the absence of new or 
established lesions while the patient is Receiving minimal or no non-PRN 1008 immunotherapy. 
Time to first CR = Date of first CR confirmed – Date of first dose + 1. 
Patients who have not achieved CR by the date of study completion or discontinuation will be 
censored at the date of completion or discontinuation. 
Source Data: Table 14.2.3.2 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog NOTE: Please also add “all dose levels” in this plot. 


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Repeat for the following populations 
Figure 14.2.3.2.2 
Kaplan Meier Plots for Time to First Complete Response (CR)  
mITT Population 
 
Figure 14.2.3.2.3 
Kaplan Meier Plots for Time to First Complete Response (CR)  
PP Population 
 
 


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Figure 14.2.3.3.1 
Kaplan Meier Plots for Time to First End of Consolidation Phase 
ITT Population 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. End of consolidation phase: The time at which no new lesions 
have developed for minimum of 2 weeks, approximately 80% of lesions have healed, and when most 
clinicians start to taper steroids. 
Time to first end of consolidation phase = Date of end of consolidation phase confirmed – Date of 
first dose + 1. 
Patients who have not achieved End of Consolidation Phase by the date of study completion or 
discontinuation will be censored at the date of completion or discontinuation. 
Source Data: Table 14.2.3.3 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 


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Prog NOTE: Please also add “all dose levels” in this plot. 
 
Repeat for the following population 
 
Figure 14.2.3.3.2 
Kaplan Meier Plots for Time to First End of Consolidation Phase 
mITT Population 
 
 
Figure 14.2.3.3.3 
Kaplan Meier Plots for Time to First End of Consolidation Phase 
PP Population 
 


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Figure 14.2.3.4.1 
Kaplan Meier Plots for Time to First Relapse after PRN1008 Treatment Completion or Discontinuation 
ITT Population 
 
 
 
Note: Patients are classified into different dose levels according to the maximum dose they received during 
the 12 week treatment period. 
Relapse/flare: Appearance of >=3 new lesions/month that do not heal spontaneously within 1 week, or by 
extension of established lesions, in a patient who has achieved disease control. 
Only patients with confirmed CDA before treatment completion or discontinuation will be included into 
this analysis. 
Time to first relapse = Date of first relapse confirmed – Date of last dose + 1. 
Patients who have not achieved Relapse by the date of study completion or discontinuation will be 
censored at the date of completion or discontinuation. 
Source Data: Table 14.2.3.4 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 


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Prog NOTE: Please also add “all dose levels” in this plot. 
Repeat for the following populations. 
 
Figure 14.2.3.4.2 
Kaplan Meier Plots for Time to First Relapse after PRN1008 Treatment Completion or Discontinuation 
mITT Population 
 
 
Figure 14.2.3.4.3 
Kaplan Meier Plots for Time to First Relapse after PRN1008 Treatment Completion or Discontinuation 
PP Population 


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Figure 14.3.3.2 
Scatter Plots of Peak TBL vs Peak ALT for Identification of Drug Induced Liver Injury (DILI) 
Safety Analysis Population 
 
Note: Patients are classified into different dose levels according to the maximum dose they received 
during the 12 week treatment period. 
TBL = total bilirubin, ALT = alanine aminotransferase or SGPT 
The maximum measurement value observed during the 12 week treatment will be defined as the Peak 
value. 
Source Data: Listing 16.2.6.2 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 


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Listing 16.2.1.1  
Patient Disposition 
Screening Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Site 
Informed 
Consent  
Date Time 
Analysis  
Set 
First Dose 
Date/ 
Last Dose 
Date(Day) 
Study 
Completed 
Completion or 
Early 
Withdrawl 
Date(Day) 
If Early 
Withdrawl, 
Reason 
 
 
 
 
 
 
 
 
 
XXXXXX 
65/F/W 
XXXXXX DDMMMYYYY 
XX:XX 
Safety/ 
ITT/mITT/
PP 
DDMMMYYYY/ 
DDMMMYYYY (XX) 
Yes 
DDMMMYYYY(XX) 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
XXXXXX DDMMMYYYY 
XX:XX 
Safety/ 
ITT/mITT/
PP 
DDMMMYYYY/ 
DDMMMYYYY(XX) 
No 
 
DDMMMYYYY(XX) 
Lost of 
Follow-up 
 
 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
XXXXXX DDMMMYYYY 
XX:XX 
Safety/ 
ITT/mITT/
PP 
DDMMMYYYY/ 
DDMMMYYYY(XX) 
No 
DDMMMYYYY(XX) 
Physician 
Decision: 
XXXXXXXXXXXX
XXXX 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID.  
 


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Listing 16.2.1.2  
Protocol Deviations 
Screening Population 
 
 
 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Date of 
Deviation 
 
Significant 
Deviations 
 
 
Category  
 
 
Protocol Deviation 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
Safety/ 
Efficacy 
DDMMMYYYY 
Yes 
XXXXXXXXXXXXXX 
 
XXXXXXXXXXXXXX 
 
 
 
 
 
 
 
 
XXXXXX 
65/F/BL 
Safety/ 
Efficacy 
DDMMMYYYY 
No 
XXXXXXXXXXXXXX 
 
XXXXXXXXXXXXXX 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Patient ID, Date of Deviation. 
 


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Listing 16.2.2.1  
Demographics and Baseline Characteristics 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Ethnicity 
Skin Biopsy 
Confirmed PV/ 
Date of Biopsy 
Pemphigus 
Type 
Height 
(cm) 
Weight 
(kg) 
BMI 
(kg/m2) 
 
 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
Safety/ 
Efficacy 
NOT HISPANIC OR 
LATINO 
Yes/ 
DDMMMYYYY 
Pemphigus 
Follaceus 
XXX.X 
XXX.X 
XX.XX 
 
 
 
 
 
 
 
 
 
XXXXXX 
65/F/BL 
Safety/ 
Efficacy 
HISPANIC OR 
LATINO 
No/ 
DDMMMYYYY 
Pemphigus 
Vulgaris 
XXX.X 
XXX.X 
XX.XX 
 
 
 
 
 
 
 
 
 
XXXXXX 
60/F/W 
Safety/ 
Efficacy 
HISPANIC OR 
LATINO 
Not Done 
Linear IgA 
Bullous 
Dermatoses 
XXX.X 
XXX.X 
XX.XX 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected; PV= Pemhigus Vulgaris 
BMI=(Baseline body weight in kilograms)/(Baseline height in meters)^2 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID.  
 


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Listing 16.2.2.2 
Medical History 
Safety Analysis Population 
 
 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Body System/ 
  Medical Condition(s) 
   Reported 
 
 
Start Date 
 
 
End Date 
 
 
Ongoing 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXXXXX/ 
  XXXXXXXXXXXXXXXXXXXXXX 
DDMMMYYYY 
DDMMMYYYY 
No 
 
 
 
 
 
 
 
XXXXXX 
65/F/BL 
Safety/ 
Efficacy 
XXXXXXXXXXXXXXXXX/ 
  XXXXXXXXXXXXXXXXXXXXXX 
DDMMMYYYY 
DDMMMYYYY 
No 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Patient ID, Body System (in order presented on CRF), and alphabetically by Medical 
Condition Reported, then start date. Body System will be taken directly from the CRF page. Only those body 
systems with non-missing results will be displayed. 
 


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Listing 16.2.2.3 
Inclusion/Exclusion Criteria 
Screening Population 
 
 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Protocol  
Version 
Met all 
Eligibility  
Criteria? 
Inclusion Criteria  
Not Met 
Exclusion Criteria  
Met 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
Safety/ 
Efficacy 
1.1 
No 
Ix, Ix, Ix 
Ex, Ex, Ex 
 
 
 
 
 
 
 
XXXXXX 
65/F/BL 
Safety/ 
Efficacy 
1.0 
Yes 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Patient ID.  
 


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Listing 16.2.3.1 
Prior and Concomitant Corticosteroid Medications 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
 
Patient 
ID 
 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
ATC Level 2/ 
  Preferred Term/ 
    Medication Reported/ 
      Indication 
 
 
 
Flag 
Start Date 
(Day)/ 
Stop Date 
(Day) 
 
 
On- 
going 
 
 
 
Dose 
 
 
 
Unit 
 
 
 
Route 
 
 
 
Freq 
 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXX/ 
 XXXXXXXXXXXXXXXXXXXX/ 
    XXXXXXXXXXXXXXXXX/ 
      XXXXXXXXXXXXXXXX 
P 
DDMMMYYYY 
(XX)/ 
DDMMMYYYY 
(XX) 
Yes 
XXX 
XXXX XXXXX XXXX 
 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
32/F/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXX/ 
 XXXXXXXXXXXXXXXXXXXX/ 
    XXXXXXXXXXXXXXXXX/ 
      XXXXXXXXXXXXXXXX 
P/C 
DDMMMYYYY 
(XX)/ 
DDMMMYYYY 
(XX) 
No 
XXX 
XXXX XXXXX XXXX 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Flag: P=Prior medication, C=Concomitant medication; Freq=Frequency 
A prior medication is defined as any medication that has a stop date before the date of first dose of 
study drug. A Concomitant medication is defined as any medication that has a stop date on or after the 
date of first dose of study drug. 
Medications with missing stop dates will be classified as both prior and concomitant. 
Prior and Concomitant medications were coded with the WHO Drug dictionary (WHO Drug DDE June 2015). 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Start Date, and 
alphabetically by ATC Level 2, Preferred Term, and Medication Reported. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 141 of 195 
 
Listing 16.2.3.2 
Prior and Concomitant Non-Corticosteroid Medications 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
 
Patient 
ID 
 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
ATC Level 2/ 
  Preferred Term/ 
    Medication Reported/ 
      Indication 
 
 
 
Flag 
Start Date 
(Day)/ 
Stop Date 
(Day) 
 
 
On- 
going 
 
 
 
Dose 
 
 
 
Unit 
 
 
 
Route 
 
 
 
Freq 
 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXX/ 
 XXXXXXXXXXXXXXXXXXXX/ 
    XXXXXXXXXXXXXXXXX/ 
      XXXXXXXXXXXXXXXX 
P 
DDMMMYYYY 
(XX)/ 
DDMMMYYYY 
(XX) 
Yes 
XXX 
XXXX XXXXX XXXX 
 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
32/F/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXX/ 
 XXXXXXXXXXXXXXXXXXXX/ 
    XXXXXXXXXXXXXXXXX/ 
      XXXXXXXXXXXXXXXX 
P/C 
DDMMMYYYY 
(XX)/ 
DDMMMYYYY 
(XX) 
No 
XXX 
XXXX XXXXX XXXX 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Flag: P=Prior medication, C=Concomitant medication; Freq=Frequency 
A prior medication is defined as any medication that has a stop date before the date of first dose of 
study drug. A Concomitant medication is defined as any medication that has a stop date on or after the 
date of first dose of study drug. 
Medications with missing stop dates will be classified as both prior and concomitant. 
Prior and Concomitant medications were coded with the WHO Drug dictionary (WHO Drug DDE June 2015). 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Start Date, and 
alphabetically by ATC Level 2, Preferred Term, and Medication Reported. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 142 of 195 
 
Listing 16.2.3.3 
Study Drug Accountability 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
 
Patient 
ID 
 
Age(yrs)/ 
Sex/ 
Race 
 
 
First Dose 
Date 
 
 
Visit Name 
Formulation 
(mg) 
 
Dispensed 
 
 
Returned 
 
Date(Day) 
 
No. 
  
Date(Day) 
 
No. 
 
 
 
 
 
 
 
  
 
XXXXXX 
50/M/W 
DDMMMYYYY 
WEEK1 D1 
100 
DDMMMYYYY 
(XXX) 
XXX 
 DDMMMYYYY 
(XXX) 
XXX 
 
 
 
WEEK1 D1 
300 
DDMMMYYYY 
(XXX) 
XXX 
 DDMMMYYYY 
(XXX) 
XXX 
 
 
 
XXXXXXXXXXX 
XXX 
DDMMMYYYY 
(XXX) 
XXX 
 DDMMMYYYY 
(XXX) 
XXX 
 
 
 
 
 
 
 
  
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg), Patient ID, Visit.  
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 143 of 195 
 
Listing 16.2.3.4 
Study Drug Administration 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
First Dose 
Date 
Visit 
Name 
Dose 
(mg) 
Start Date 
(Day) 
Stop Date 
(Day) 
 
Duration 
(Days) 
[1] 
 
Dose 
Adjusted/ 
Reason 
 
 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
DDMMMYYYY 
WEEK1 D1 
400 
DDMMMYYYY(XX) DDMMMYYYY(XX) 
XXX 
No 
 
 
 
 
WEEK5 D29 
XXX 
DDMMMYYYY(XX) DDMMMYYYY(XX) 
XXX 
Yes/ 
XXXXXXXXX 
XXXXX 
 
 
 
 
WEEK9 D57 
XXX 
DDMMMYYYY(XX) DDMMMYYYY(XX) 
XXX 
No 
 
 
 
 
 
 
 
 
 
XXXXXX 
32/F/W 
DDMMMYYYY 
WEEK1 D1 
400 
DDMMMYYYY(XX) DDMMMYYYY(XX) 
XXX 
No 
 
 
 
 
XXXXXXXXX 
XXX 
DDMMMYYYY(XX) DDMMMYYYY(XX) 
XXX 
No 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
[1] Duration (Days) = Stop Date – Start Date +1 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level(400 mg, 500 mg, 600 mg), Patient ID, Visit, Start Date. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 144 of 195 
 
Listing 16.2.3.5 
Study Drug Exposure 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
 
First Dose 
Date 
Last Dose 
Date 
Overall 
Duration 
(Days) [1] 
 
Cumulative 
Dose (mg) 
[2] 
Average 
Daily Dose 
(mg/day) [3] 
 
Minimum 
Dose 
Level 
(mg) 
 
No. of Dose 
Adjustments 
 
 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
DDMMMYYYY 
DDMMMYYYY 
XXX 
XXX 
XXX 
XXX  
XXX 
 
 
 
 
 
 
 
 
 
XXXXXX 
32/F/W 
DDMMMYYYY 
DDMMMYYYY 
XXX 
XXX 
XXX 
XXX  
XXX 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
[1] Overall Duration (Days) = Last Dose Date – First Dose Date +1   
[2] Cumulative Dose is defined as the sum of the doses taken by patients during the treatment 
[3] Average Daily Dose = Cumulative Dose / Overall Duration 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level(400 mg, 500 mg, 600 mg), Patient ID. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 145 of 195 
 
Listing 16.2.3.6 
Study Drug Compliance 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
 
 
First Dose Date Last Dose Date 
Number of 
Tablets  
Taken [1] 
Number of 
Tablets  
Planned [2] 
Overall 
Compliance (%) 
[3] 
 
 
 
 
 
 
 
XXXXXX  
50/M/W 
DDMMMYYYY 
DDMMMYYYY 
XXX 
XXX 
XX.X 
 
 
 
 
 
 
 
XXXXXX 
32/F/W 
DDMMMYYYY 
DDMMMYYYY 
XXX 
XXX 
XX.X 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
[1] Number of tablets taken is calculated by subtracting the number of tablets returned from the number of 
tablets dispensed 
[2] Number of tablets planned is calculated by multiplying the number of planned exposure days by number of 
tablets planned per day. 
[3] Overall Compliance (%) = Total number of tablets taken / Total number of tablets planned * 100 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level(400 mg, 500 mg, 600 mg), Patient ID. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 146 of 195 
 
Listing 16.2.4.1.1 
Control of Disease Activity (CDA) Status and Predni(ol)one Use Condition Within 4 Weeks 
IIT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Achieved CDA  
at the Week 5 Visit 
Predni(ol)one >0.5 mg/kg  
on or before the Week 5 Visit Responder [1] 
 
 
 
 
 
 
XXXXXX  
50/M/W 
Safety/ 
Efficacy 
Yes 
No 
Yes 
 
 
 
 
 
 
XXXXXX 
32/F/W 
Safety/ 
Efficacy 
Yes 
Yes 
No 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Control of disease activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. 
[1] Patients who achieved CDA at the Week 5 visit and had no occurrence of daily prednis(ol)one >0.5 mg/kg 
on or before the Week 5 visit during the study treatment will be considered as responders. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Visit. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 147 of 195 
 
Listing 16.2.4.1.2 
Control of Disease Activity (CDA) Status and Corticosteroids Use Condition Within 4 Weeks 
IIT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Achieved CDA  
at the Week 5 Visit 
Any Corticosteroid Used  
on or before the Week 5 Visit Responder [1] 
 
 
 
 
 
 
XXXXXX  
50/M/W 
Safety/ 
Efficacy 
Yes 
No 
Yes 
 
 
 
 
 
 
XXXXXX 
32/F/W 
Safety/ 
Efficacy 
Yes 
Yes 
No 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Control of disease activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. 
[1] Patients who achieved CDA at the Week 5 visit and had no occurrence of any corticosteroid on or before 
the Week 5 visit during the study treatment will be considered as responders. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Visit. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 148 of 195 
 
Listing 16.2.4.1.3 
Complete Response (CR) Status and Corticosteroids Use Condition Within 12 Weeks 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Achieved CR  
at the Week 13 Visit 
Any Corticosteroid Used  
on or before the Week 13 
Visit 
Responder [1] 
 
 
 
 
 
 
XXXXXX  
50/M/W 
Safety/ 
Efficacy 
Yes 
No 
Yes 
 
 
 
 
 
 
XXXXXX 
32/F/W 
Safety/ 
Efficacy 
Yes 
Yes 
No 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
Receiving minimal or no non-PRN 1008 immunotherapy. 
[1] Patients who achieved CR at the Week 13 visit and had no occurrence of any corticosteroid on or before 
the Week 13 visit during the study treatment will be considered as responders. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Visit. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 149 of 195 
 
Listing 16.2.4.1.4 
Complete Response (CR) Status and Predni(ol)one Use Condition Within 12 Weeks 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Achieved CR  
on or before the 
Week 13 Visit 
Predni(ol)one >0.5 mg/kg on 
or before the Fist CR  
Responder [1] 
 
 
 
 
 
 
XXXXXX  
50/M/W 
Safety/ 
Efficacy 
Yes 
No 
Yes 
 
 
 
 
 
 
XXXXXX 
32/F/W 
Safety/ 
Efficacy 
Yes 
Yes 
No 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
Receiving minimal or no non-PRN 1008 immunotherapy. 
[1] Patients who achieved CR at any visit on or before the Week 13 visit during the study treatment and had 
no occurrence of daily prednis(ol)one >0.5 mg/kg between the first dose date of PRN1008 treatment and 
the date of the first CR will be considered as responders. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Visit. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 150 of 195 
 
Listing 16.2.4.1.5 
Disease Outcome Status 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
Visit 
Name 
Prednis(ol)one >0.5 
mg/kg [1] 
Disease Status 
Achieved? 
Achieved 
Date 
CS Dose at 
Time of 
First CR 
(mg) [2] 
 
 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
Safety/ 
Efficacy 
Screen 
No 
Control of 
Disease Activity 
No 
 
 
 
 
 
 
 
Complete 
Response 
No 
 
 
 
 
 
 
 
End of 
Consolidated 
Phase 
No 
 
 
 
 
 
 
 
Relapse 
Yes 
DDMMMYYYY XX.X 
 
 
 
WEEK1 
D1 
No 
… 
… 
 
 
 
 
 
… 
 
… 
… 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
[1] Daily prednis(ol)one >0.5 mg/kg on or before the specific visit during the study treatment. 
[2] Dose of CS combined from all recorded CS on the day before date of the first CR for this subject. 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Visit. 
           For CS dose at time of achievement, please note that if a record of CS is ongoing and does not 
have an end date, the end date is assumed to be the date of last follow up. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 151 of 195 
 
Listing 16.2.4.1.6 
Time to First Control of Disease Activity (CDA) 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
First Dose Date/ 
Last Dose Date 
Date of First CDA 
or Censored Date 
Time to First CDA 
(Days) 
Censored 
 
 
 
 
 
 
 
XXXXXX  
50/M/W 
Safety/ 
Efficacy 
DDMMMYYYY/ 
DDMMMYYYY 
DDMMMYYYY 
xx 
Yes 
 
 
 
 
DDMMMYYYY 
xx 
Yes 
 
 
 
 
DDMMMYYYY 
xx 
Yes 
 
 
 
 
DDMMMYYYY 
xx 
No 
 
 
 
 
 
… 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Control of disease activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. 
Time to first CDA = Date of first CDA confirmed – Date of first dose + 1. Patients who did not 
achieve CDA by the date of study completion or discontinuation were censored at the date of 
completion or discontinuation respectively. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Visit. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 152 of 195 
 
Listing 16.2.4.1.7 
Time to First Complete Response (CR) 
IIT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
First Dose Date/ 
Last Dose Date 
Date of First CR 
or Censored Date 
Time to First CR 
(Days) 
Censored 
 
 
 
 
 
 
 
XXXXXX  
50/M/W 
Safety/ 
Efficacy 
DDMMMYYYY/ 
DDMMMYYYY 
DDMMMYYYY 
xx 
Yes 
 
 
 
 
DDMMMYYYY 
xx 
Yes 
 
 
 
 
DDMMMYYYY 
xx 
Yes 
 
 
 
 
DDMMMYYYY 
xx 
No 
 
 
 
 
 
… 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
receiving minimal or no non-PRN 1008 immunotherapy. 
Time to first CR = Date of first CR confirmed – Date of first dose + 1. Patients who did not achieve 
CR by the date of study completion or discontinuation were censored at the date of completion or 
discontinuation respectively. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Visit. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 153 of 195 
 
Listing 16.2.4.1.8 
Time to First End of Consolidation Phase 
IIT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
First Dose Date/ 
Last Dose Date 
Date of First End 
of Consolidation 
Phase or Censored 
Date 
Time to First End 
of Consolidation 
Phase (Days) 
Censored 
 
 
 
 
 
 
 
XXXXXX  
50/M/W 
Safety/ 
Efficacy 
DDMMMYYYY/ 
DDMMMYYYY 
DDMMMYYYY 
xx 
Yes 
 
 
 
 
DDMMMYYYY 
xx 
Yes 
 
 
 
 
DDMMMYYYY 
xx 
Yes 
 
 
 
 
DDMMMYYYY 
xx 
No 
 
 
 
 
 
… 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
End of consolidation phase: The time at which no new lesions have developed for minimum of 2 weeks, 
approximately 80% of lesions have healed, and when most clinicians start to taper steroids. 
Time to first end of consolidation phase = Date of first end of consolidation phase confirmed – Date 
of first dose + 1. Patients who did not achieve end of consolidation phase by the date of study 
completion or discontinuation were censored at the date of completion or discontinuation 
respectively. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Visit. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 154 of 195 
 
Listing 16.2.4.1.9 
Time to First Relapse after Week 13 Visit 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
First Dose 
Date/ 
Last Dose 
Date 
Date of First 
Relapse After Week 
13 or Censored Date 
CDA at 
Week 
13 
CR at 
Week 
13 
Time to First 
Relapse From 
Week 13 
(Days) 
Censored 
 
 
 
 
 
Yes/No Yes/No  
 
XXXXXX  50/M/W 
Safety/ 
Efficacy 
DDMMMYYYY/ 
DDMMMYYYY 
DDMMMYYYY 
Yes/No Yes/No 
xx 
Yes 
 
 
 
 
DDMMMYYYY 
Yes/No Yes/No xx 
Yes 
 
 
 
 
DDMMMYYYY 
Yes/No Yes/No xx 
Yes 
 
 
 
 
DDMMMYYYY 
Yes/No Yes/No xx 
No 
 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Relapse/flare: Appearance of >=3 new lesions/month that do not heal spontaneously within 1 week, or 
by extension of established lesions, in a patient who has achieved disease control. Only patients 
with confirmed CDA before treatment completion or discontinuation will be included into this listing. 
Time to first relapse = Date of first relapse confirmed after week 13 – Date of week 13 visit + 1. 
Patients who did not achieve relapse by the date of study completion or discontinuation were censored 
at the date of completion or discontinuation respectively. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Visit. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 155 of 195 
Listing 16.2.4.1.10 
Duration of Complete Response (CR) Status Among CR Subjects 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
CR Start Date 
Last Known CR Date 
Last Follow up 
Date  
Duration [1] 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
Safety/ 
Efficacy 
DDMMMYYYY 
DDMMMYYYY 
DDMMMYYYY 
Xx 
 
 
 
 
 
 
 
XXXXXX 
32/F/W 
Safety/ 
Efficacy 
DDMMMYYYY 
DDMMMYYYY 
DDMMMYYYY 
xx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Complete response (CR) is defined as the absence of new or established lesions while the patient is 
Receiving minimal or no non-PRN 1008 immunotherapy. 
[1]   Duration is defined as the earlier of 1) Last known CR date and 2) Last follow up date –CR start date 
+ 1. Only CR after start of treatment is counted in this listing. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Only include subjects with CR, Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), 
Patient ID, Visit. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 156 of 195 
 
Listing 16.2.4.2.1 
Pemphigus Disease Area Index (PDAI) Score 
IIT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Visit Name 
Assessment 
Date 
Body Part 
Anatomical Location 
Parameter 
Result 
 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
SCREEN 
DDMMMYYY 
Skin 
EARS 
xxxxxxxxxxx 
x 
 
 
 
 
 
NOSE 
xxxxxxxxxxx 
x 
 
 
 
 
 
REST OF THE FACE  
xxxxxxxxxxx 
x 
 
 
 
 
 
NECK 
xxxxxxxxxxx 
x 
 
 
 
 
 
CHEST 
xxxxxxxxxxx 
x 
 
 
 
 
 
ABDOMEN 
xxxxxxxxxxx 
x 
 
 
 
 
 
BACK, BUTTOCKS 
xxxxxxxxxxx 
x 
 
 
 
 
 
ARMS 
xxxxxxxxxxx 
x 
 
 
 
 
 
HANDS 
xxxxxxxxxxx 
x 
 
 
 
 
 
LEGS 
xxxxxxxxxxx 
x 
 
 
 
 
 
FEET 
xxxxxxxxxxx 
x 
 
 
 
 
 
GENITALS 
xxxxxxxxxxx 
x 
 
 
 
 
Scalp 
SCALP 
xxxxxxxxxxx 
x 
 
 
 
 
Mucousa 
EYES 
xxxxxxxxxxx 
x 
 
 
 
 
 
NOSE 
xxxxxxxxxxx 
x 
 
 
 
 
 
BUCCAL MUCOSA 
xxxxxxxxxxx 
x 
 
 
 
 
 
HARD PALATE 
xxxxxxxxxxx 
x 
 
 
 
 
 
SOFT PALATE 
xxxxxxxxxxx 
x 
 
 
 
 
 
UPPER GINGIVA 
xxxxxxxxxxx 
x 
 
 
 
 
 
LOWER GINGIVA 
xxxxxxxxxxx 
x 
 
 
 
 
 
TOGUE 
xxxxxxxxxxx 
x 
 
 
 
 
 
FLOOR OF MOUTH 
xxxxxxxxxxx 
x 
 
 
 
 
 
LABIAL MUCOSA 
xxxxxxxxxxx 
x 
 
 
 
 
 
POSTERIOR PHARYNX 
xxxxxxxxxxx 
x 
 
 
 
 
 
ANOGENITAL 
xxxxxxxxxxx 
x 
 
 
WEEK1 D1 
DDMMMYYYY 
… 
… 
… 
… 
 
 
… 
… 
… 
… 
… 
… 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 


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[1] Activity Score for Skin: 0=absent; 1=1-3 lesions up to one >2 cm in any diameter, none >6 cm; 2=2-3 
lesions, at least two >2 cm diameter, none >6 cm; 3=>3 lesions, none >6 cm diameter; 5=>3 lesions, 
and/or at least one >6 cm; 10=>3 lesions, and/or at least one lesion > 16 cm diameter or entire area. 
Activity Score for Scalp: 0=absent; 1=in one quadrant; 2=two quadrants; 3=three quadrants; 4 =affects 
whole skull; 10=at least one lesion > 6cm. 
Activity Score for Mucosa: 0= absent; 1=1 lesion; 2=2-3 lesions; 5=>3 lesions or 2 lesions >2 cm; 
10=entire area. 
[2] Damage Score for Skin and Scalp: 0=absent; 1=present. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Visit, Assessment Date, 
Body Part, Anatomical Location. 
 


Principia Biopharma Australia Pty Ltd. 
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Listing 16.2.4.2.2 
Pemphigus Disease Area Index (PDAI) Total Scores 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Visit Name 
Assessment Date 
PDAI Scores 
Score 
 
 
 
 
 
 
XXXXXX  
50/M/W 
SCREEN 
DDMMMYYY 
Total Skin Activity Score 
XXX 
 
 
 
 
Total Scalp Activity Score 
 XX 
 
 
 
 
Total Mucosa Activity Score 
XXX 
 
 
 
 
Total Skin Damage Score 
 XX 
 
 
 
 
Total Scalp Damage Score 
  X 
 
 
 
 
Total Activity Score 
XXX 
 
 
 
 
Total Damage Score 
 XX 
 
 
 
 
 
 
 
 
WEEK1 D1 
DDMMMYYY 
… 
… 
 
 
… 
… 
… 
… 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Total Activity Score = Total Skin Activity Score + Total Scalp Activity Score + Total Mucosa Activity 
Score 
Total Damage Score = Total Skin Damage Score + Total Scalp Damage Score 
Total PDAI Score = Total Activity Score + Total Damage Score 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Visit, Assessment Date, 
PDAI Score. 
 


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Listing 16.2.4.3.1 
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS)—Skin Involvement 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
 
 
Visit Name 
Assessment 
Date 
Skin Involvement (Max BSA) 
Patient’s 
BSA (%) 
Weighting 
Factor 
[1] 
Skin 
Involvement 
Total Score 
[2] 
 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
SCREEN 
DDMMMYYY 
HEAD & NECK (9%) 
XX 
X.X 
XX.X 
 
 
 
 
L ARM INCLUDING HAND (9%) XX 
X.X 
XX.X 
 
 
 
 
R ARM INCLUDING HAND (9%) XX 
X.X 
XX.X 
 
 
 
 
TRUNK (FRONT & BACK)(36%) XX 
X.X 
XX.X 
 
 
 
 
L LEG (18%) 
XX 
X.X 
XX.X 
 
 
 
 
R LEG (18%) 
XX 
X.X 
XX.X 
 
 
 
 
GENITALS (1%) 
XX 
X.X 
XX.X 
 
 
WEEK1 D1 
DDMMMYYYY 
… 
… 
… 
… 
 
 
… 
… 
… 
… 
… 
… 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected; BSA=Body Surface Area 
[1] 1.5 = Erosive, exudative lesions; 1 = Erosive, dry lesions; 0.5 = reepithelialized lesions 
[2] • Skin involvement total score = sum of (%BSA * Weighting factor) 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Visit, Assessment 
Date. 
 


Principia Biopharma Australia Pty Ltd. 
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Listing 16.2.4.3.2 
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS)—Oral Involvement 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Visit Name 
Assessment Date Oral Involvement 
Extent [1] 
 
 
 
 
 
 
XXXXXX  
50/M/W 
SCREEN 
DDMMMYYY 
UPPER GINGIVAL MUCOSA 
X 
 
 
 
 
LOWER GINGIVAL MUCOSA 
X 
 
 
 
 
UPPER LIP MUCOSA 
X 
 
 
 
 
LOWER LIP MUCOSA 
X 
 
 
 
 
LEFT BUCCAL MUCOSA 
X 
 
 
 
 
RIGHT BUCCAL MUCOSA 
X 
 
 
 
 
TONGUE 
X 
 
 
 
 
FLOOR OF THE MOUTH 
X 
 
 
 
 
HARD PALATE 
X 
 
 
 
 
SOFT PALATE 
X 
 
 
 
 
PHARYNX 
X 
 
 
WEEK1 D1 
DDMMMYYYY 
… 
… 
 
 
… 
… 
… 
… 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
[1] 1=presence of lesions; 0=absence of lesion 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level(400 mg, 500 mg, 600 mg, missing), Patient ID, Visit, Assessment Date. 
 


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Listing 16.2.4.3.3 
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS)—Severity 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
 
Visit Name 
Assessment 
Date 
Food 
Level 
Factor of 
Discomfort 
[1] 
Severity 
Score [2] 
 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
SCREEN 
DDMMMYYY 
WATER 
1 
X.X 
X.X 
 
 
 
 
SOUP 
2 
X.X 
X.X 
 
 
 
 
YOGURT 
3 
X.X 
X.X 
 
 
 
 
CUSTARD 
4 
X.X 
X.X 
 
 
 
 
MASHED POTATOES / SCRAMBLED 
EGG 
5 
X.X 
X.X 
 
 
 
 
BAKED FISH 
6 
X.X 
X.X 
 
 
 
 
WHITE BREAD 
7 
X.X 
X.X 
 
 
 
 
APPLE / RAW CARROT 
8 
X.X 
X.X 
 
 
 
 
FRIED STEAK / WHOLE-GRAIN 
BREAD 
9 
X.X 
X.X 
 
 
WEEK1 D1 
DDMMMYYYY 
… 
… 
… 
… 
 
 
… 
… 
… 
… 
… 
… 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
[1] 1=Pain/bleeding occurred always; 0.5=Pain/bleeding occurred sometimes; 0=Never experienced problems 
[2] Severity Score = Level * Factor of Discomfort 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level(400 mg, 500 mg, 600 mg, missing), Patient ID, Visit, Assessment Date. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 162 of 195 
 
Listing 16.2.4.3.4 
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Visit Name 
Assessment Date 
Sub-Category 
ABSIS Score 
 
 
 
 
 
 
XXXXXX  
50/M/W 
SCREEN 
DDMMMYYY 
Skin Involvement Total Score 
XXX 
 
 
 
 
Oral Involvement Total Score 
 XX 
 
 
 
 
Severity Total Score 
 XX 
 
 
 
 
Total Activity Score 
 
 
 
 
 
Total ABSIS Score 
 
 
 
WEEK1 D1 
DDMMMYYY 
… 
… 
 
 
… 
… 
… 
… 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Skin involvement total score = sum of %BSA * Weighting factor 
Oral involvement total score = sum score from each oral part 
Severity total score = sum of Level * Factor of discomfort 
      ABSIS total activity score = Skin involvement total score + Oral involvement total score 
      Total ABSIS score = Skin involvement total score + Oral involvement total score + Severity total 
score 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level(400 mg, 500 mg, 600 mg, missing), Patient ID, Visit, Assessment Date. 
 


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PRN1008-005 
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Listing 16.2.4.4.1 
Autoimmune Bullous Diseases Quality of Life (ABQOL) Questionnaire 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Visit Name 
Assessment 
Date 
ABQOL Question 
ABQOL Answer 
ABQOL 
Score 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
WEEK1 D1 
DDMMMYYY 
In regards to your blistering 
 disease, does your skin burn, 
 sting or hurt in any way? 
All the time 
  1 
 
 
 
 
In regards to your blistering 
 disease, does you skin itch? 
Sometimes 
  2 
 
 
 
 
Have you had to change your  
 clothing because of your 
 blistering disease? 
I have had to 
 change most of 
 the things I 
 wear 
  2 
 
 
 
 
Do you notice your skin heals 
 slowly? 
I notice this 
 occasionally 
  3 
 
 
 
 
Do you have difficulty bathing or 
 showering because of your 
 blistering disease? 
Occasionally 
  3 
 
 
 
 
In regards to your blistering 
 disease, does your mouth have 
 erosions which are painful? 
Sometimes 
  2 
 
 
 
 
In regards to your blistering 
 disease, do your gums bleed 
 easily? 
All the time 
  1 
 
 
 
 
Does your blistering disease 
 results in you having to avoid 
 food or drinks that you enjoy? 
I can eat some 
 of the foods I 
 enjoy 
  2 
 
 
 
 
As a result of your blistering 
 disease, are you embarrassed 
 about your appearance? 
Sometimes 
  2 
 
 
 
 
Do you feel depressed or angry 
 because of your blistering 
 disease? 
Occasionally 
  3 
 
 
 
 
Do you feel anxious or cannot relax 
 as a result of your blistering 
Not at all 
  4 


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Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
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 disease? 
 
 
 
 
Do you worry that friends and 
 family find your blistering 
 skin condition tiresome? 
Not at all 
  4 
 
 
 
 
Is your blistering disease causing 
 sexual difficulties? 
Sometimes 
  2 
 
 
 
 
Do you worry that friends and 
 family find your blistering 
 skin condition tiresome? 
Relationships 
 are very 
 difficult 
  2 
 
 
 
 
Does your blistering disease affect 
 your social life? 
I can go to 
 most social 
 events 
  3 
 
 
 
 
Does your blistering disease affect 
 your work or study? 
Yes, I find it 
 difficult to 
 work or study 
  2 
 
 
 
 
Do employers discriminate against 
 you because of your blistering 
 disease? 
My employers 
 are completely 
 understanding 
 OR not 
 Applicable 
 (N/A) 
  4 
 
 
WEEK3 D15 
DDMMMYYY 
… 
… 
… 
 
 
… 
… 
… 
… 
… 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level(400 mg, 500 mg, 600 mg, missing), Patient ID, Visit, Assessment Date. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 165 of 195 
 
Listing 16.2.4.4.2 
Autoimmune Bullous Diseases Quality of Life (ABQOL) Score 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Visit Name 
Assessment Date 
ABQOL Score 
 
 
 
 
 
XXXXXX  
50/M/W 
WEEK1 D1 
DDMMMYYY 
 XX 
 
 
WEEK3 D15 
DDMMMYYY 
 XX 
 
 
XXXX 
DDMMMYYY 
 XX 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level(400 mg, 500 mg, 600 mg, missing), Patient ID, Visit, Assessment Date. 
 


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Page 166 of 195 
 
Listing 16.2.4.5.1 
Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL) Questionnaire 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Visit Name 
Assessment 
Date 
TABQOL Question 
TABQOL Answer 
TABQOL 
Score 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
WEEK1 D1 
DDMMMYYY 
As a result of your blistering 
 disease treatment, do you notice 
 you bruise or bleed easily? 
I notice this 
 all the time 
  1 
 
 
 
 
As a result of your blistering 
 disease treatment, can you still 
 tolerate hot or cold temperatures? 
I am sometimes 
 sensitive to 
 changes in 
 temperature 
  2 
 
 
 
 
Do you have to take your 
 medications for your blistering 
 disease at a specific time? 
Yes: it is a 
 little annoying 
  2 
 
 
 
 
Do you take many medications for 
 your blistering disease? 
Yes but I do not 
 mind 
  3 
 
 
 
 
Does the treatment for your 
 blistering disease result in you 
 feeling bloated? 
Some of the time 
  3 
 
 
 
 
Does the treatment for your 
 blistering disease make it 
 difficult to walk? 
A lot of the time 
  2 
 
 
 
 
As a result of your blistering 
 disease treatment, can you think 
 as quickly or as clearly as you 
 used to? 
No it is very 
 frustrating 
  1 
 
 
 
 
Do you find your blistering disease 
 treatment very time consuming? 
Yes it is quite 
 annoying 
  2 
 
 
 
 
Do you mind the needles or blood 
 tests involved in the treatment of 
 your blistering disease? 
Yes I dislike 
 needles 
  2 
 
 
 
 
Do you worry your blistering 
 disease will get worse when you 
 drop the dosage of your 
 medications? 
Yes I sometimes 
 worry about it 
  3 


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Page 167 of 195 
 
 
 
 
 
 
 
 
 
 
 
Do you worry about your blistering 
 disease being dangerous? 
I never worry 
 about it 
  4 
 
 
 
 
Do you feel tired and lethargic as 
 a result of the treatment for your 
 blistering disease? 
Not at all 
  4 
 
 
 
 
Do you worry about getting sick 
 (with the flu, etc.) due to your 
 depressed immunity because of the 
 treatment for your blistering 
 disease? 
Yes I worry about 
 it a lot 
  2 
 
 
 
 
As a result of your blistering 
 disease treatment, have you 
 stopped doing many activities to 
 avoid getting sick? 
Yes I cannot do 
 many of the 
 activities I 
 enjoy 
  2 
 
 
 
 
Do you have nightmares or bad 
 memories as a result of your 
 blistering disease treatments? 
Some of the time 
  3 
 
 
 
 
Does your blistering disease 
 treatment affect your holidays? 
Does your 
 Blistering 
 disease treatment 
 affect your 
 holidays? 
  2 
 
 
 
 
Is your blistering disease 
 treatment giving you financial 
 difficulties? 
No 
  4 
 
 
WEEK3 D15 
DDMMMYYY 
… 
… 
… 
 
 
… 
… 
… 
… 
… 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level(400 mg, 500 mg, 600 mg, missing), Patient ID, Visit, Assessment Date. 


Principia Biopharma Australia Pty Ltd. 
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Page 168 of 195 
 
Listing 16.2.4.5.2 
Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL) Score 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Visit Name 
Assessment Date 
TABQOL Score 
 
 
 
 
 
XXXXXX  
50/M/W 
WEEK1 D1 
DDMMMYYY 
 XX 
 
 
WEEK3 D15 
DDMMMYYY 
 XX 
 
 
XXXX 
DDMMMYYY 
 XX 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level(400 mg, 500 mg, 600 mg, missing), Patient ID, Visit, Assessment Date. 
 


Principia Biopharma Australia Pty Ltd. 
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Page 169 of 195 
 
Listing 16.2.4.6.1 
Simplified Nutritional Appetite Questionnaire (SNAQ) Questionnaire 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Visit Name 
Assessment 
Date 
SNAQ Question 
SNAQ Answer 
SNAQ 
Score 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
WEEK1 D1 
DDMMMYYY 
My appetite is 
average 
  3 
 
 
 
 
When I eat 
I feel full after eating about a 
 third of a meal 
  2 
 
 
 
 
Food tastes 
bad 
  2 
 
 
 
 
Normally I eat 
more than three meals a day 
  5 
 
 
WEEK3 D15 
DDMMMYYY 
… 
… 
… 
 
 
… 
… 
… 
… 
… 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level(400 mg, 500 mg, 600 mg, missing), Patient ID, Visit, Assessment Date. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 170 of 195 
 
 
 
Listing 16.2.4.6.2 
Simplified Nutritional Appetite Questionnaire (SNAQ) Score 
ITT Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Visit Name 
Assessment Date 
SNAQ Score 
 
 
 
 
 
XXXXXX  
50/M/W 
WEEK1 D1 
DDMMMYYY 
 XX 
 
 
WEEK3 D15 
DDMMMYYY 
 XX 
 
 
XXXX 
DDMMMYYY 
 XX 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level(400 mg, 500 mg, 600 mg, missing), Patient ID, Visit, Assessment Date. 
 


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Page 171 of 195 
 
Listing 16.2.5.1 
Adverse Events 
Safety Analysis Population 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
*=Treatment-emergent AE 
Dur=Duration (Days = Stop date – Start date + 1) 
Pat of AE=Pattern of AE: Int=intermittent, Cont=continuous 
Outcome: 1=Recovered/Resolved, 2=Recovering/Resolving, 3=Recovered/Resolved with sequelae, 4=Not 
recovered/not resolved, 5=Fatal, 6=Unknown 
C/A=Concomitant or Additional Treatment Given? 
Sev=Severity: 1=Grade 1, 2=Grade 2, 3=Grade 3, 4=Grade 4, 5=Grade 5 
Act=Action taken with study treatment: 1=Dose increased, 2=Dose not changed, 3=Dose reduced, 4=Drug 
interrupted, 5=Drug Withdrawn, 6=Not Applicable, 7=Unknown 
Rel=Relationship to study treatment: 1=Not related, 2=Related 
Study Disc=Caused study discontinuation? 
Ser=Serious? 
Adverse Events were coded using MedDRA, Version 18.0. 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 


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Listing 16.2.5.1 
Adverse Events 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
System Organ Class/  
  Preferred Term/  
    Adverse Event 
     Reported 
Start Date Time 
(Day)/ 
Stop Date Time 
(Day) 
Dur 
(Days) 
Pat 
of 
AE 
Out-
come 
C/
A 
Sev 
Act/ 
Rel 
Study 
Disc/ 
Ser 
 
 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
65/F/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXXXXXXX/ 
  XXXXXXXXXXXXXXXXX/ 
    XXXXXXXXXXXXXXXXXXX 
DDMMMYYYY XX:XX 
(XX)/ 
DDMMMYYYY XX:XX 
(XX) 
126 
Cont 
4 
Y 
2 
5/1 
Y/Y 
 
 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXXXXXXX/ 
  XXXXXXXXXXXXXXXXX/ 
    XXXXXXXXXXXXXXXXXXX * 
DDMMMYYYY XX:XX 
(XX)/ 
DDMMMYYYY XX:XX 
(XX) 
45 
Int 
2 
Y 
1 
2/2 
Y/Y 
 
 
 
 
 
 
 
 
 
 
 
 
Notes are listed on page 1. 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, AE Start Date/Time, and 
alphabetically by System Organ Class, Preferred Term, and Adverse Event Reported. 
 


Principia Biopharma Australia Pty Ltd. 
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Page 173 of 195 
 
Listing 16.2.5.2 
Serious Adverse Events 
Safety Analysis Population 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
*=Treatment-emergent AE 
Dur=Duration (Days = Stop date – Start date + 1) 
Pat of AE=Pattern of AE: Int=intermittent, Cont=continuous 
Outcome: 1=Recovered/Resolved, 2=Recovering/Resolving, 3=Recovered/Resolved with sequelae, 4=Not 
recovered/not resolved, 5=Fatal, 6=Unknown 
C/A=Concomitant or Additional Treatment Given? 
Sev=Severity: 1=Grade 1, 2=Grade 2, 3=Grade 3, 4=Grade 4, 5=Grade 5 
Act=Action taken with study treatment: 1=Dose increased, 2=Dose not changed, 3=Dose reduced, 4=Drug 
interrupted, 5=Drug Withdrawn, 6=Not Applicable, 7=Unknown 
Rel=Relationship to study treatment: 1=Not related, 2=Related 
Study Disc=Caused study discontinuation? 
Adverse Events were coded using MedDRA, Version 18.0. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 


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Page 174 of 195 
Listing 16.2.5.2 
Serious Adverse Events 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
System Organ Class/  
  Preferred Term/  
    Adverse Event  
      Reported 
Start Date Time 
(Day)/ 
Stop Date Time 
(Day) 
Dur 
(Days) 
Pat 
of 
AE 
Out-
come 
C/
A 
Sev 
Act/ 
Rel 
Study 
Disc/ 
Ser 
 
 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
65/F/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXXXXXXX/ 
  XXXXXXXXXXXXXXXXX/ 
    XXXXXXXXXXXXXXXXXXX 
DDMMMYYYY XX:XX 
(XX)/ 
DDMMMYYYY XX:XX 
(XX) 
126 
Cont 
4 
Y 
2 
5/1 
Y/Y 
 
 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXXXXXXX/ 
  XXXXXXXXXXXXXXXXX/ 
    XXXXXXXXXXXXXXXXXXX * 
DDMMMYYYY XX:XX 
(XX)/ 
DDMMMYYYY XX:XX 
(XX) 
45 
Int 
2 
Y 
1 
2/2 
Y/Y 
 
 
 
 
 
 
 
 
 
 
 
 
Notes are listed on page 1. 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, AE Start Date/Time, and 
alphabetically by System Organ Class, Preferred Term, and Adverse Event Reported. 
 
 
 


Principia Biopharma Australia Pty Ltd. 
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Page 175 of 195 
 
Listing 16.2.5.3 
Adverse Events Leading to Treatment Discontinuation 
Safety Analysis Population 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
*=Treatment-emergent AE 
Dur=Duration (Days = Stop date – Start date + 1) 
Pat of AE=Pattern of AE: Int=intermittent, Cont=continuous 
Outcome: 1=Recovered/Resolved, 2=Recovering/Resolving, 3=Recovered/Resolved with sequelae, 4=Not 
recovered/not resolved, 5=Fatal, 6=Unknown 
C/A=Concomitant or Additional Treatment Given? 
Sev=Severity: 1=Grade 1, 2=Grade 2, 3=Grade 3, 4=Grade 4, 5=Grade 5 
Rel=Relationship to study treatment: 1=Not related, 2=Related 
Study Disc=Caused study discontinuation? 
Ser=Serious? 
Adverse Events were coded using MedDRA, Version 18.0. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 


Principia Biopharma Australia Pty Ltd. 
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Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 176 of 195 
Listing 16.2.5.3 
Adverse Events Leading to Treatment Discontinuation 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
System Organ Class/  
  Preferred Term/  
    Adverse Event  
      Reported 
Start Date Time 
(Day)/ 
Stop Date Time 
(Day) 
Dur 
(Days) 
Pat 
of 
AE 
Out-
come 
C/
A 
Sev 
Rel 
Study 
Disc/ 
Ser 
 
 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
65/F/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXXXXXXX/ 
  XXXXXXXXXXXXXXXXX/ 
    
XXXXXXXXXXXXXXXXXXX 
DDMMMYYYY XX:XX 
(XX)/ 
DDMMMYYYY XX:XX 
(XX) 
126 
Cont 
4 
Y 
2 
1 
Y/Y 
 
 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXXXXXXX/ 
  XXXXXXXXXXXXXXXXX/ 
    
XXXXXXXXXXXXXXXXXXX * 
DDMMMYYYY XX:XX 
(XX)/ 
DDMMMYYYY XX:XX 
(XX) 
45 
Int 
2 
Y 
1 
2 
Y/Y 
 
 
 
 
 
 
 
 
 
 
 
 
Notes are listed on page 1. 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg), Patient ID, AE Start Date/Time, and 
alphabetically by System Organ Class, Preferred Term, and Adverse Event Reported. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 177 of 195 
 
Listing 16.2.5.4 
Adverse Events Leading to Death 
Safety Analysis Population 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
*=Treatment-emergent AE 
Dur=Duration (Days = Stop date – Start date + 1) 
Pat of AE=Pattern of AE: Int=intermittent, Cont=continuous 
Outcome: 1=Recovered/Resolved, 2=Recovering/Resolving, 3=Recovered/Resolved with sequelae, 4=Not 
recovered/not resolved, 5=Fatal, 6=Unknown 
C/A=Concomitant or Additional Treatment Given? 
Sev=Severity: 1=Grade 1, 2=Grade 2, 3=Grade 3, 4=Grade 4, 5=Grade 5 
Act=Action taken with study treatment: 1=Dose increased, 2=Dose not changed, 3=Dose reduced, 4=Drug 
interrupted, 5=Drug Withdrawn, 6=Not Applicable, 7=Unknown 
Rel=Relationship to study treatment: 1=Not related, 2=Related 
Study Disc=Caused study discontinuation? 
Ser=Serious? 
Adverse Events were coded using MedDRA, Version 18.0. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 178 of 195 
Listing 16.2.5.4 
Adverse Events Leading to Death 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
System Organ Class/  
  Preferred Term/  
    Adverse Event  
      Reported 
Start Date Time 
(Day)/ 
Stop Date Time 
(Day) 
Dur 
(Days) 
Pat 
of 
AE 
Out-
come 
C/
A 
Sev 
Act/ 
Rel 
Study 
Disc/ 
Ser 
 
 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
65/F/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXXXXXXX/ 
  XXXXXXXXXXXXXXXXX/ 
    
XXXXXXXXXXXXXXXXXXX 
DDMMMYYYY XX:XX 
(XX)/ 
DDMMMYYYY XX:XX 
(XX) 
126 
Cont 
5 
Y 
2 
5/1 
Y/Y 
 
 
 
 
 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
Safety/ 
Efficacy 
XXXXXXXXXXXXXXXXXXX/ 
  XXXXXXXXXXXXXXXXX/ 
    
XXXXXXXXXXXXXXXXXXX * 
DDMMMYYYY XX:XX 
(XX)/ 
DDMMMYYYY XX:XX 
(XX) 
45 
Int 
5 
Y 
1 
2/2 
Y/Y 
 
 
 
 
 
 
 
 
 
 
 
 
Notes are listed on page 1. 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, AE Start Date/Time, and 
alphabetically by System Organ Class, Preferred Term, and Adverse Event Reported. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 179 of 195 
 
 
Listing 16.2.6.1 
Clinical Laboratory Results - Chemistry Tests 
Safety Analysis Population 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
Visit 
Collection Date 
Time(Day) 
Laboratory Test 
(Unit) 
Result 
Flag[1] 
Normal 
Range 
 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
Safety/ 
Efficacy 
XXXXXXXX DDMMMYYYY XX:XX 
(XX) 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
  
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
L 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
 
 
 
 
 
 
 
 
DMMMYYYY XX:XX 
(XX) 
XXXXXXXXXXXXXXX 
XXX.X 
H 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 H* 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
[1] L=Low, H=High, *= Newly Notable if the patient did not meet the abnormality criteria at baseline 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Lab Category, Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, 
Laboratory Test Collection Date, Lab test. For Result and Normal Range, display the number of decimal 
places where Applicable exactly as they are in the data. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 180 of 195 
 
Listing 16.2.6.2 
Clinical Laboratory Results – Drug Induced Liver Injury 
Safety Analysis Population 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
Visit 
Collection Date 
Time(Day) 
Laboratory Test 
(Unit) 
Result 
Flag[1] 
Normal 
Range 
 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
Safety/ 
Efficacy 
XXXXXXXX DDMMMYYYY XX:XX 
(XX) 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
  
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
L 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
 
 
 
 
 
 
 
 
DMMMYYYY XX:XX 
(XX) 
XXXXXXXXXXXXXXX 
XXX.X 
H 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 H* 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Clinical laboratory tests related to drug induced liver injury (DILI) include ALT (alanine 
aminotransferase or SGPT), AST (aspartate transaminase or SGOT), TBL (total bilirubin), and ALP 
(alkaline phosphatase). 
[1] L=Low, H=High, *= Newly Notable abnormality if the patient did not meet the abnormality criteria at 
baseline 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Lab Category, Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, 
Laboratory Test Collection Date/Time, Lab test. For Result and Normal Range, display the number of decimal 
places where Applicable exactly as they are in the data. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 181 of 195 
 
Listing 16.2.6.3 
Clinical Laboratory Results – Hematology 
Safety Analysis Population 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
Visit 
Collection Date 
Time(Day) 
Laboratory Test 
(Unit) 
Result 
Flag[1] 
Normal 
Range 
 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
Safety/ 
Efficacy 
XXXXXXXX DDMMMYYYY XX:XX 
(XX) 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
  
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
L 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
 
 
 
 
 
 
 
 
DMMMYYYY XX:XX 
(XX) 
XXXXXXXXXXXXXXX 
XXX.X 
H 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 H* 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
[1] L=Low, H=High, *= Newly Notable abnormality if the patient did not meet the abnormality criteria at 
baseline 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Lab Category, Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, 
Laboratory Test Collection Date/Time, Lab test. For Result and Normal Range, display the number of decimal 
places where Applicable exactly as they are in the data. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 182 of 195 
 
Listing 16.2.6.4 
Clinical Laboratory Results – Coagulation 
Safety Analysis Population 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
Visit 
Collection Date 
Time(Day) 
Laboratory Test 
(Unit) 
Result 
Flag[1] 
Normal 
Range 
 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
Safety/ 
Efficacy 
XXXXXXXX DDMMMYYYY XX:XX 
(XX) 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
  
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
L 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
 
 
 
 
 
 
 
 
DMMMYYYY XX:XX 
(XX) 
XXXXXXXXXXXXXXX 
XXX.X 
H 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 H* 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXX.X 
 
XXX.X-XXX.X 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
[1] L=Low, H=High, *= Newly Notable abnormality if the patient did not meet the abnormality criteria at 
baseline 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Lab Category, Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, 
Laboratory Test Collection Date/Time, Lab test. For Result and Normal Range, display the number of decimal 
places where Applicable exactly as they are in the data. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 183 of 195 
 
Listing 16.2.6.5 
Clinical Laboratory Results – Urinalysis 
Safety Analysis Population 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
Visit 
Collection Date 
Time(Day) 
Laboratory Test 
(Unit) 
Result 
Normal 
Range 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
Safety/ 
Efficacy 
XXXXXXXX DDMMMYYYY XX:XX 
(XX) 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
  
 
 
 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX-XXXX 
 
 
 
 
 
 
 
 
 
 
 
 
DMMMYYYY XX:XX 
(XX) 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX-XXXX 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Lab Category, Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, 
Laboratory Test Collection Date/Time, Lab test. For Result and Normal Range, display the number of decimal 
places where Applicable exactly as they are in the data. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 184 of 195 
 
Listing 16.2.6.6 
Clinical Laboratory Results – Serology 
Safety Analysis Population 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis 
Set 
Visit 
Collection Date 
Time(Day) 
Laboratory Test 
(Unit) 
Result 
Normal 
Range 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
Safety/ 
Efficacy 
XXXXXXXX DDMMMYYYY XX:XX 
(XX) 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
  
 
 
 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
 
 
 
 
 
 
 
 
 
 
 
 
DMMMYYYY XX:XX 
(XX) 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
 
 
 
 
 
XXXXXXXXXXXXXXX 
XXXXXX 
XXXX 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Lab Category, Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, 
Laboratory Test Collection Date/Time, Lab test. For Result and Normal Range, display the number of decimal 
places where Applicable exactly as they are in the data. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 185 of 195 
Listing 16.2.6.7 
Vital Sign Results 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Visit/ 
Date(Day) 
Vital Sign Parameter 
Result [1] 
 
 
 
 
 
 
XXX  
50/M/W  
 
 
Safety/ 
Efficacy 
XXXX/ 
DDMMMYYYY(XXX) 
Systolic Blood Pressure (mmHg) 
       XXX (L) 
  
 
 
 
Diastolic Blood Pressure (mmHg) 
        XX (H*)  
 
 
 
 
Heart Rate (BEATS/MIN) 
       XXX 
 
 
 
 
Respiratory Rate (BREATHS/MIN) 
        XX 
 
 
 
 
Temperature (C) 
        XX.X 
 
 
 
 
Height (cm) 
       XXX.X 
 
 
 
 
Weight (kg) 
       XXX.X 
 
 
 
 
 
         
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
[1] L= Low (systolic blood pressure value <90 mmHg, diastolic blood pressure value <60 mmHg, heart rate 
<40 BEATS/MIN); H=High (systolic blood pressure value >140 mmHg, diastolic blood pressure value >80 
mmHg, heart rate >100 BEATS/MIN); *=Newly notable abnormality if the patient did not meet the 
abnormality criteria at baseline 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Date.  
 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 186 of 195 
 
Listing 16.2.6.8 
Physical Examination 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Visit/ 
Exam Date 
Time(Day) 
Assessment 
Status 
Abnormality Comment(s) 
 
 
 
 
 
 
 
XXXXXX 
50/M/W 
Safety/ 
Efficacy 
XXXX/ 
DDMMMYYYY 
XX:XX(XX) 
SKIN 
Abnormal XXXXXXXXXXXXXXXXXXXXX 
 
 
 
 
HEAD, EYES, EARS, NOSE, 
THROAT 
Normal 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note:  Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, Exam Date, 
Assessment. All assessments will be displayed for each patient. 
 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 187 of 195 
 
Listing 16.2.6.9 
Electrocardiogram Results at Screening Visit 
Safety Analysis Population 
 
Maximum Dose Level: XXX mg 
Patient 
ID 
Age(yrs)
/ 
Sex/ 
Race 
Analysis
Set 
Visit/ 
ECG Date 
Time(Day
) 
Normal 
Sinus 
Rhythm ECG Parameter 
Result 
Status 
Interpretati
on [1] 
Abnormalit
y 
Detail 
 
 
 
 
 
 
 
 
 
 
XXXXXX  
50/M/W 
Safety/ 
Efficacy
XXXX/ 
DDMMMYYY
Y 
XX:XX(XX
) 
Yes 
Ventricular Rate 
BEATS/MINUTE) 
     
Not Done 
 NCS 
XXXXXXXXXX
XXXXXXX 
  
 
 
 
 
PR Interval 
(msec) 
    XX 
 
 CS 
XXXXXXXXXX
XXXXXXX 
 
 
 
 
 
QRS Duration 
(msec) 
   XXX 
 
 N 
 
 
 
 
 
 
QT Interval 
(msec) 
      
Not Done 
 NCS 
XXXXXXXXXX
XXXXXXX 
 
 
 
 
 
QTcF Interval 
(msec) 
   XXX 
 
 N 
 
 
 
 
 
 
QTcB Interval 
(msec) 
   XXX 
 
 N 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
[1] NCS=Not Clinically Significant, CS=Clinically Significant, N=Normal 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Maximum Dose Level (400 mg, 500 mg, 600 mg, missing), Patient ID, ECG Date, Time. 
 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 188 of 195 
 
Listing 16.2.6.10 
Female Reproductive Status and Pregnancy Test Results 
Safety Analysis Population 
 
 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Fertility 
Status 
Method of 
Birth 
Control 
Pregnancy Test 
Visit 
Test  
Performed 
Collection 
Date 
Results 
 
 
 
 
 
 
 
 
 
 
XXXXXXX
X 
33/  
F/ 
W 
Safety/ 
Efficacy 
XXXXXX 
XXXXXXXX 
XXXXXX 
Urine Pregnancy 
Yes 
DDDMMYYY  
XXXXXXX 
 
 
 
 
 
 
Serum Pregnancy 
Yes 
DDDMMYYY  
XXXXXXX 
 
 
 
 
 
 
FSH 
Yes 
DDDMMYYY  
XXX.X 
 
 
 
 
 
XXXXXX 
Urine Pregnancy 
No 
DDDMMYYY  
 
 
 
 
 
 
 
Serum Pregnancy 
Yes 
DDDMMYYY  
XXXXXXX 
 
 
 
 
 
 
FSH 
Yes 
DDDMMYYY  
XXX.X 
 
 
 
 
 
 
 
 
 
 
XXXXXXX
X 
56/  
F/ 
W 
Safety/ 
Efficacy 
 
XXXXXX 
XXXXXXXX 
XXXXXX 
Urine Pregnancy 
Yes 
DDDMMYYY  
XXXXXXX 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or Alaska 
Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Patient ID, Visit, Test. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 189 of 195 
 
Listing 16.2.6.11 
Food Intake Status 
Safety Analysis Population 
 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Visit 
Food Intake Date 
Time 
Food Intake 
 
 
 
 
 
 
XXXXXX  
50/M/W 
Safety/ 
Efficacy 
WEEK1 D1 
DDMMMYYYY XX:XX Liquid 
  
 
 
WEEK1 D2 
DDMMMYYYY XX:XX Liquid 
 
 
 
XXXXX 
DDMMMYYYY XX:XX Liquid 
 
 
 
XXXXX 
DDMMMYYYY XX:XX Solid such as toast 
 
 
 
XXXXX 
DDMMMYYYY XX:XX Not Applicable 
 
 
 
XXXXX 
DDMMMYYYY XX:XX Solid such as toast 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Patient ID, Date. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 190 of 195 
 
Listing 16.2.6.12 
Brain MRI Results 
Safety Analysis Population 
 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Visit 
Allergy to MRI 
Macrocyclic 
Contrast 
Date of Scan 
Result 
 
 
 
 
 
 
 
XXXXXX  
50/M/W 
Safety/ 
Efficacy 
SCREEN 
No 
DDMMMYYYY 
Normal 
  
 
 
WEEK1 D15 
No 
DDMMMYYYY 
Abnormal clinically 
significant 
XXXXXX 
56/F/W 
Safety/ 
Efficacy 
XXXXXX 
No 
DDMMMYYYY 
XXXXX 
 
 
 
XXXXXX 
No 
DDMMMYYYY 
XXXXX 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Only patients from French sites had this measurement. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Patient ID, Date. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 191 of 195 
 
Listing 16.2.6.13 
Neurological Symptoms 
Safety Analysis Population 
 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Visit 
Headache 
Nausea 
Vomiting 
Visual 
Disturbance AE Number 
 
 
 
 
 
 
 
 
 
XXXXXX  
50/M/W 
Safety/ 
Efficacy 
XXXXXX 
No 
Yes 
No 
Yes 
XXXX 
  
 
 
XXXXXX 
No 
No 
No 
No 
 
XXXXXX 
56/F/W 
Safety/ 
Efficacy 
XXXXXX 
Yes 
Yes 
Yes 
No 
XXXX 
 
 
 
XXXXXX 
No 
Yes 
No 
Yes 
XXXX 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Only patients from French sites had this measurement. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Patient ID, Visit. 


Principia Biopharma Australia Pty Ltd. 
PRN1008-005 
Final Statistical Analysis Plan Shells Version 2.0   
 
 
 
Page 192 of 195 
 
Listing 16.2.6.14 
Neurological Examination 
Safety Analysis Population 
 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis  
Set 
Visit 
Date of 
Assessment 
Neurological 
Assessment 
Result 
 
 
 
 
 
 
 
XXXXXX  
50/M/W 
Safety/ 
Efficacy 
XXXXXX 
DDMMMYYYY 
Cranial Nerve 1 Normal 
  
 
 
 
 
XXXXXX 
Abnormal, XXXXX 
 
 
 
XXXXXX 
DDMMMYYYY 
XXXXXX 
XXXXX 
 
 
 
 
 
XXXXXX 
XXXXX 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
Only patients from French sites had this measurement. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Patient ID, Date. 
 


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Page 193 of 195 
Listing 16.2.7.1 
Plasma Concentrations of PRN1008 
Safety Analysis Population 
 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Visit 
Sample 
Collected 
 
Collection Date 
Time 
Collection 
Timepoint 
Sample 
Performed 
Concentration 
(ng/mL) 
 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
WEEK1 D1 
Yes 
DDMMMYYYY XX:XX 
Pre-Dose 
Yes 
  LLOQ (<0.100) 
  
 
 
 
 
2 Hours Post Dose 
Yes 
  XX.X 
  
 
WEEK1 D2 
Yes 
DDMMMYYYY XX:XX 
 
Yes 
  XX.X 
 
 
WEEK3 D15 
No 
 
 
No 
 
 
 
… 
 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
LLOQ = Lower Limit of quantitation 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Patient ID, Visit. 


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Listing 16.2.7.2 
Percentage BTK Occupancy 
ITT Population 
  
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Visit 
Sample 
Collected 
 
Collection 
Date Time 
Collection 
Timepoint 
Date Time 
of Last 
Dose 
Time Since 
Last Dose 
(Hr) 
BTK Occupancy 
(%) 
 
 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
WEEK1 D1 
Yes 
DDMMMYYYY 
XX:XX 
Pre-Dose 
DDMMMYYYY 
XX:XX 
X.X 
  XX.X 
  
 
 
 
 
2 Hours Post 
Dose 
 
 
  XX.X 
  
 
WEEK1 D2 
Yes 
DDMMMYYYY 
XX:XX 
 
DDMMMYYYY 
XX:XX 
X.X 
  XX.X 
 
 
WEEK3 D15 
No 
 
 
 
 
 
 
 
… 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Patient ID, Visit. 
 


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Page 195 of 195 
 
Listing 16.2.7.3 
Anti-DSG 1-3 Autoantibody Levels 
ITT Population 
 
Patient 
ID 
Age(yrs)/ 
Sex/ 
Race 
Analysis Set 
Visit 
 
Collection Date Time 
Anti-DSG 1 
Anti-DSG 3 
 
 
 
 
 
 
 
XXXXXX  50/M/W 
Safety/ITT/mITT/PP WEEK1 D1 
DDMMMYYYY XX:XX 
XX 
XX 
  
 
 
WEEK5 D29 
DDMMMYYYY XX:XX 
XX 
XX 
 
 
 
WEEK9 D57 
 
 
 
 
 
… 
… 
 
 
 
 
 
 
 
 
 
 
Note: Sex: M=Male, F=Female; Race: W=White, BL=Black or African American, AS=Asian, AI=American Indian or 
Alaska Native, PI=Native Hawaiian or Other Pacific Islander, O=Other, FSNC=French Sites Only – Not 
Collected 
      The limit of quantification for Anti-DSG 1 and 3 are 14 and 9 respectively. 
      Collection time is not available beyond Week 13/Day 85. 
 
<DIRECTORY PATH>PROGRAM NAME  Executed: DDMMMYYYY hh:mm 
 
Prog Note: Sort by Patient ID, Visit. 
           Values are presented as collected, disregarding limit of quantification. 


CONFIDENTIAL 
No part of this document may be passed on, reproduced, or published without written 
permission of Principia Biopharma. 
STATISTICAL ANALYSIS PLAN 
Protocol PRN1008-005 (Part B) 
An Open-Label, Phase 2, Pilot Study Investigating the Safety, Clinical Activity, Pharmacokinetics, and 
Pharmacodynamics of Oral Treatment with the BTK Inhibitor PRN1008 in Patients with Newly Diagnosed 
or Relapsing Pemphigus Vulgaris 
Development Phase:  Phase 2 
Analysis Stage: Final 
Sponsor 
Principia Biopharma 
Document Version 
Final, 24-Feb-2020 
Prepared by 
[ Filename: PRN1008-005B - SAP final 24Feb2020.docx ] 
NCT02704429




Statistical Analysis Plan− Final  24-Feb-2020 
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VERSION HISTORY 
Version 
No. 
Version Date 
(dd-MMM-yyyy) 
Description of Changes 
1 
24-Feb-2020 
Original version 
 
 
 
 
 
 
 
 
 
 
 
 
 
 


Statistical Analysis Plan− Final  24-Feb-2020 
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ABBREVIATIONS 
Abbreviation 
Definition 
ABSIS 
Autoimmune Bullous Skin Disorder Intensity Score 
ABQOL 
Autoimmune Bullous Diseases Quality of Life (assessment) 
ADaM 
Analysis Data Model 
AE 
Adverse Event 
ALP 
Alkaline Phosphatase 
ALT 
Alanine Aminotransferase 
Anti-DSG 
Anti-Desmoglein 
AST 
Aspartate Aminotransferase 
BID 
bis in die (twice a day) 
BMI 
Body Mass Index 
BP 
Blood Pressures 
bpm 
Beats per Minute 
BTK 
Bruton’s Tyrosine Kinase 
CDISC 
Clinical Data Interchange Standards consortium 
CDA 
Control of Disease Activity 
CI 
Confidence Interval 
CPK 
Creatinine phosphokinase 
CR 
Complete Response 
CRF 
Case Report Form 
CS 
Clinically Significant 
CTCAE 
Common Terminology Criteria for Adverse Events 
DBP 
Diastolic Blood Pressure 
DILI 
Drug Induced Liver Injury 
DSG 
Desmoglein 
ECG 
Electrocardiogram 
EOT 
End of Treatment 
ELISA 
enzyme-linked immunosorbent assay 
EQ-5D VAS 
Euro-QoL 5-Dimension Visual Analog Scale  
EOT 
End of Treatment 
FSH 
Follicle Stimulating Hormone 
GLP 
Good Laboratory Practice 


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Abbreviation 
Definition 
Hep 
Hepatitis 
HIV 
Human Immunodeficiency Viruses 
IDSM 
Independent Data Safety Monitor 
IP 
Investigational Product 
ITT 
Intent-to-Treat 
MedDRA 
Medical Dictionary for Regulatory Activities 
NCS 
Not Clinically Significant 
PD 
Pharmacodynamic 
PDAI 
Pemphigus Disease Area Index 
PF 
Pemphigus foliaceus 
PK 
Pharmacokinetic 
PT 
Preferred Term 
PV 
Pemphigus vulgaris 
QD 
quaque die (once a day) 
QTc 
QT interval corrected for heart rate 
QOL 
Quality of Life 
SAE 
Serious adverse event 
SAP 
Statistical Analysis Plan 
SBP 
Systolic Blood Pressure 
SD 
Standard Deviation 
SDTM 
Study Data Tabulation Model 
SI 
Système international d’unités (International system of units) 
SMC 
Safety Monitoring Committee 
SNAQ 
Simplified Nutritional Appetite Questionnaire 
SOC 
System Organ Class 
TABQOL 
Treatment of Autoimmune Bullous Diseases Quality of Life (assessment) 
TBL 
Total Bilirubin 
TEAE 
Treatment Emergent Adverse Event 
TLFs 
Tables, Listings and Figures 
TPO 
Thrombopoietin  
TSH 
Thyroid stimulating hormone 
ULN 
Upper Limit of Normal  
WHODRUG 
World Health Organization Drug  


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Abbreviation 
Definition 
WHO DDE 
World Health Organization Drug Dictionary  
 
 
 
 
 


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TABLE OF CONTENTS 
1 
INTRODUCTION .................................................................................................................... 10 
2 
SYNOPSIS OF STUDY DESIGN & PROCEDURES ..................................................................... 10 
2.1 
Study Objectives ...................................................................................................... 10 
2.1.1 
Primary Objectives ................................................................................................................10 
2.1.2 
Secondary Objective .............................................................................................................10 
2.1.3 
Exploratory Objectives ..........................................................................................................10 
2.2 
Study Design ............................................................................................................ 10 
2.3 
Expected Sample Size .............................................................................................. 11 
2.4 
Randomization and Blinding ................................................................................... 11 
2.5 
Protocol Versions .................................................................................................... 12 
2.6 
Protocol Amendments ............................................................................................ 12 
2.7 
Schedule of Assessments ........................................................................................ 13 
3 
ANALYSIS CONSIDERATIONS ................................................................................................ 15 
3.1 
Type of Analyses...................................................................................................... 15 
3.2 
Analysis Populations ............................................................................................... 15 
3.2.1 
Screening Population ............................................................................................................15 
3.2.2 
Safety Population .................................................................................................................15 
3.2.3 
Efficacy Analysis/Intent-to-Treat (ITT) Population ...............................................................15 
3.2.4 
Modified Intent-to-Treat (mITT) Population ........................................................................15 
3.2.5 
Completer Population ..........................................................................................................15 
3.2.6 
Per-Protocol (PP) Population ................................................................................................15 
3.2.7 
Pharmacokinetic Analysis Population ...................................................................................15 
3.2.8 
Patients from Part A .............................................................................................................16 
3.3 
Treatment Groups ................................................................................................... 16 
3.3.1 
Treatment Group Labels .......................................................................................................16 
3.4 
Analysis Timepoints and Definitions ....................................................................... 16 
3.4.1 
Baseline for PDAI, ABSIS, ABQOL, TABQOL and SNAQ scores ..............................................17 
3.4.2 
Baseline for Lab and Vital Signs ............................................................................................17 
3.4.3 
Pre-treatment vs. treatment emergent vs. post last dose adverse events ..........................17 
3.4.4 
Prior medications vs. concomitant medications vs. post last dose medications..................17 


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3.4.5 
Study Day ..............................................................................................................................17 
3.4.6 
Time from Pemphigus Diagnosis (years) ..............................................................................18 
3.4.7 
Control of Disease Activity (CDA), Complete Remission (CR), End of Consolidation 
Phase (ECP) and Relapse ......................................................................................................18 
3.5 
Data Handling .......................................................................................................... 18 
3.5.1 
Partial dates ..........................................................................................................................18 
3.5.2 
Missing data imputation .......................................................................................................19 
3.5.3 
Lab values below or above a threshold ................................................................................20 
3.6 
Interim Analyses ...................................................................................................... 20 
3.7 
Handling of Study Center Effects ............................................................................ 20 
3.8 
Documentation & Other Considerations ................................................................ 20 
4 
STATISTICAL METHODS ........................................................................................................ 21 
4.1 
Safety Endpoints ..................................................................................................... 21 
4.2 
Primary Efficacy Endpoint ....................................................................................... 21 
4.3 
Secondary Efficacy Endpoints ................................................................................. 21 
4.4 
Pharmacokinetic Outcome Measure ...................................................................... 22 
4.5 
Pharmacodynamic Outcome Measures .................................................................. 22 
4.6 
Exploratory Measures ............................................................................................. 23 
4.7 
Changes to Planned Analyses from Protocol .......................................................... 23 
5 
ANALYSIS DETAILS ................................................................................................................ 24 
5.1 
Patient Disposition, Demographics and Baseline Characteristics ........................... 24 
5.1.1 
Patient Disposition................................................................................................................24 
5.1.2 
Demographics and Baseline Characteristics .........................................................................25 
5.1.3 
Disease History .....................................................................................................................25 
5.1.4 
Medical History .....................................................................................................................25 
5.1.5 
Prior and Concomitant Medications .....................................................................................26 
5.2 
Protocol Deviations ................................................................................................. 26 
5.3 
Study Drug Treatment Exposure and Compliance .................................................. 26 
5.3.1 
Study Drug Exposure ............................................................................................................26 
5.3.2 
Study Drug Compliance ........................................................................................................27 


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5.4 
Efficacy Analyses ..................................................................................................... 27 
5.4.1 
Primary Efficacy Endpoint .....................................................................................................27 
5.4.1.1 Subset Analyses ....................................................................................................................28 
5.4.2 
Secondary Efficacy Endpoints ...............................................................................................29 
5.4.2.1 CDA Responses .....................................................................................................................29 
5.4.2.2 Time-to-Event .......................................................................................................................30 
5.4.2.3 Cumulative Corticosteroid (CS) Usage ..................................................................................30 
5.4.2.4 CS Dose on the Day Before Each Visit ...................................................................................32 
5.4.2.5 Change from Baseline in Simple Nutritional Appetite Questionnaire (SNAQ) score ............33 
5.4.2.1 Change from Baseline and Percent Change from Baseline in Total Anti-DSG, Anti-DSG 
1 and Anti-DSG .....................................................................................................................34 
5.5 
Safety Analyses ....................................................................................................... 34 
5.5.1 
Adverse Events .....................................................................................................................34 
5.5.2 
Laboratory Data ....................................................................................................................35 
5.5.3 
Vital Signs and Body Weight .................................................................................................36 
5.5.4 
12-Lead ECG ..........................................................................................................................37 
5.5.5 
Physical Examinations ...........................................................................................................37 
5.5.6 
Female Reproductive Status and Pregnancy Test Results ....................................................37 
5.5.7 
Food Intake Status ................................................................................................................37 
6 
MOCK SHELLS FOR TABLES, LISTINGS AND FIGURES (TLFS) ................................................. 38 
 
 
 


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1 INTRODUCTION 
The purpose of this document is to describe the final statistical analysis for Part B of the PRN1008-005 
study. This statistical analysis plan (SAP) is based on protocol version 7.0 dated 19-Mar-2019 and case 
report form dated 08-Aug-2018.  
The last Part A patient completed the study on 20-Jun-2019. The final Part A study SAP is version 2.0 
dated 06-Jan-2019. 
2 SYNOPSIS OF STUDY DESIGN & PROCEDURES 
2.1 Study Objectives 
2.1.1 
Primary Objectives 
• 
To evaluate the clinical safety of PRN1008 in patients with PV over a 24-week (Part B) treatment 
period 
• 
To evaluate the clinical activity of PRN1008 in patients with PV  
2.1.2 
Secondary Objective 
• 
To evaluate the pharmacokinetics (PK) and the pharmacodynamics (PD) of PRN1008 in patients 
with PV 
2.1.3 
Exploratory Objectives 
• 
To evaluate the relationship of PK and PD to each other and to efficacy and safety in this patient 
populationTo explore effect of PRN1008 on markers of hemolysis 
2.2 Study Design 
This is a Phase 2, open-label, pilot cohort study to evaluate the safety, clinical activity, 
pharmacodynamics, and pharmacodynamics of PRN1008 in patients with newly diagnosed or relapsing 
PV. An open-label design was chosen for several reasons: PV does not spontaneously remit so there is 
no placebo effect to account for; clinicians need to know when to use rescue CSs, if necessary, in a 
timely manner; and lastly, the pilot nature of the investigation itself is best explored in an open-label 
fashion. 
Up to 28 days before enrollment in the study, patients will be required to sign an informed consent 
form, after which screening assessments will be carried out. Patients must fulfill all entry criteria to be 
enrolled into the study. Patients who fail to meet the entry criteria may be rescreened at the discretion 
of the Investigator. Up to 25 male or female patients with newly diagnosed (i.e., naïve to an effective 
induction treatment regimen) or relapsing, biopsy-proven, mild or moderate PV patients (Pemphigus 
Disease Activity Index [PDAI] 8 to 45) to enrolled into each parts of the study.  


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In Part A, the initial dosing was 400 mg twice daily (bid). The duration of study therapy was 12 weeks 
starting on Day 1 and ending on study Day 84 (total duration of individual subject participation is 28 
weeks), with follow-up visits (off-treatment) until Week 29. 
 
In Part B, the initial dosing is 400 mg once daily (qd), with intra-patient dose escalation to 400 mg bid 
allowed at or after the Week 3 visit for insufficient clinical response (and then again to 600 mg bid if 
necessary at or after the Week 5 visit). The duration of study therapy is 24 weeks starting on Day 1 and 
ending on study Day 169, with a follow-up visit (off-treatment) on Day 197 (Week 29).  The total 
duration of individual subject participation is approximately 32 weeks. 
 
Dose Escalation Rules Part B 
Current Dose 
Inadequate Clinical Response* Dose-Escalation Rules 
400 mg qd 
Increase to 400 mg bid (allowed at Week 3 visit or later)** 
400 mg bid 
Increase to 600 mg bid (allowed at Week 5 visit or later)** 
600 mg bid 
No dose increase possible. 
* Investigator discretion: Generally, clinical response is shown by some improvement seen in first 2 weeks with 
CDA achieved by the Week 5 visit 
** Unless tolerability issues preclude dose-escalation 
 
The first dose on Day 1 of Week 1 is supervised in the clinic and the patient observed for approximately 
2 hours, at which time a PK/PD sample will be drawn. Day 2 study medication is withheld in the morning 
in order to gain the trough BTK occupancy approximately 12 (when bid dosing) or approximately 24 
hours (when qd dosing) after the prior dose. 
 
Where follow-up is not feasible the next day, another day in the first week of treatment may be used to 
get through occupancy instead, again withholding dose on the morning of that day to get an occupancy 
measurement close to 12 (when bid dosing) or approximately 24 (when qd dosing) hours post dose. 
At other follow-up visits, BTK occupancy can be measured at any time after the usual morning dose of 
PRN1008. 
Part B patients will complete the visit procedures per the Schedule of Assessment (Table 1 ). 
 
The maximum dose of PRN1008 in both parts of this study, after dose adjustment, will be 600 mg bid. 
2.3 Expected Sample Size 
Sample size for this pilot study was determined pragmatically by the number of subjects required to 
determine a preliminary safety profile and a point estimate for the primary endpoint of efficacy. If the 
clinical response rate is 50%, 25 subjects in each Part will result in an 80% CI of ± 13%. Results from this 
pilot study will be used to design confirmatory clinical trials. 
2.4 Randomization and Blinding 
This is an open-label study. 


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2.5 Protocol Versions 
Study enrollment started on protocol version v3.0 04NOV2015.  The current protocol version is version 
6.0 dated 30 Jul 2018. 
2.6 Protocol Amendments 
There are 7 versions of the protocol: 
Protocol Version 
Version Date 
Version 1 – Original protocol 
25SEP2015 
Version 2 
05OCT2015 
Version 3 
13OCT2015 
Version 3.1 
04APR2016 
Version 4 
20APR2016 
Version 4.1 – French Protocol Amendment 
12MAY2017 
Version 5 
06APR2018 
Version 6  
30JUL2018 
Version 7 
19MAR2019 


Statistical Analysis Plan− Final  24-Feb-2020 
 
 
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2.7 Schedule of Assessments 
Table 1  
Schedule of Assessments – Part B (24-weeks Treatment/ 4-weeks Follow Up) 
 
Screen 
Day 1, 
Week 1 
Pre-dose 
Day 1 
Week 1 
Post-dose a 
Day 2, 
Week 1g 
Day 15, 
Week 3 
+/- 3 days 
Day 29, 
Week 5 
+/- 3 days 
Day 57, 
Week 9 
+/- 7 days 
Day 85, 
Week 13 
+/- 7 days 
Day 113, 
Week 17 
+/- 7 days  
Day 141, 
Week 21 
+/- 7 days 
Day 169, 
Week 25 
+/- 7 days 
Day 197, 
Week 29 
+/- 7 days 
Unscheduled 
Visit 
Informed Consentk 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Inclusion/Exclusion 
Criteria 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
Height 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Weight 
X 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Physical exam/med. 
History PDAI/ABSIS 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Abbreviated 
physical exam, 
PDAI, ABSIS 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
ECG (12-lead) 
X 
 
 
 
 
 
 
 
 
 
 
 
(X)b 
Vital Signsj 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Urinalysis 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Hep B &C, HIV, T-
spot TB Test, 
QuantiFERON®-TB 
Gold, 
QuantiFERON®-TB 
Gold 
Plus (QFT Plus) 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Pregnancy testc 
X 
X 
 
 
 
X 
X 
X 
X 
X 
X 
X 
 
Skin biopsyd 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Hem, Coag, Chem 
Xe 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
FSHf 
X 
 
 
 
 
 
 
 
 
 
 
 
 
BTK occupancy 
sample 
 
Xg 
Xh 
X 
X 
X 
X 
X 
X 
X 
X 
 
(X)b 
PK sample 
 
X 
Xh 
Xa 
X 
X 
X 
X 
X 
X 
X 
 
(X)b 
Anti-DSG 
antibodies. 
 
X 
 
 
 
X 
X 
X 
X 
X 
X 
X 
 


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Screen 
Day 1, 
Week 1 
Pre-dose 
Day 1 
Week 1 
Post-dose a 
Day 2, 
Week 1g 
Day 15, 
Week 3 
+/- 3 days 
Day 29, 
Week 5 
+/- 3 days 
Day 57, 
Week 9 
+/- 7 days 
Day 85, 
Week 13 
+/- 7 days 
Day 113, 
Week 17 
+/- 7 days  
Day 141, 
Week 21 
+/- 7 days 
Day 169, 
Week 25 
+/- 7 days 
Day 197, 
Week 29 
+/- 7 days 
Unscheduled 
Visit 
Photography 
(Optional)i 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
ABQOL & TABQOL 
 
X 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
SNAQ questionnaire 
 
X 
 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
AEs 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Concomitant 
Medications 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Drug dispensed 
 
X 
 
 
 
X 
X 
X 
X 
X 
 
 
 
Drug reconciliation 
 
 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
 
a. Withhold PRN1008 on morning of Day 2 until PK/PD measurement has been taken as close to 12 hours (when bid dosing) or 24 hours (when qd 
dosing) after the prior dose as possible. Where follow-up on Day 2 is not possible, PK/PD samples may be taken on another day in the first week of 
treatment. On all other days, instruct patient to take PRN1008 in the morning as usual prior to clinic and take note of the time taken. Extra PK/PD 
sample intended for 1 to 5 days after dose adjustment or to replace missing samples–not required for other extra visits. 
b. Only if clinically indicated. 
c. For women of childbearing potential only. Serum pregnancy test done at screening, urine dip test done at other time points. 
d. Performed only if no suitable prior biopsy. 
e. TSH and CPK taken as part of chemistry panel. 
f. To confirm postmenopausal status for women who are not surgically sterile only. 
g. Two-8mL pre-dose blood tubes to be collected at baseline to ensure sufficient samples for later time point assay calculations. 
h. 2 hours post-dose (+/- 15 mins) 
i. Photography is used to document skin disease changes; ideally in most patients. Strict masking of patient identity is required. 
j. Vital sign include blood pressure (BP), pulse rate, body temperature and respiratory rate and are recorded at the time points specified 
k. The Schedule of Assessments has been revised to be consistent with the 24 week treatment duration and 4 week safety follow up assessments 
described in this protocol Version 5, Part B. Patients enrolled in an earlier protocol version (Part A) who are in the active12-week treatment period 
are eligible to continue treatment, initially at their current dose, for up to a total of 24 weeks active treatment upon review and signature of the Part 
B EC approved Patient Informed Consent Form, with procedures and tests per this Schedule. Patients that have completed Part A of the trial are also 
eligible to be screened as a Part B patient, upon review and signature of the Part B EC-approved Patient Informed Consent Form. 
 
 


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3 ANALYSIS CONSIDERATIONS 
3.1 Type of Analyses 
The analyses and documentation described in this SAP are for the final analysis of Part B of the study. 
3.2 Analysis Populations 
3.2.1 
Screening Population 
All patients who provide informed consent and have screening assessments in Part B evaluated for study 
participation will be included in the Screening Population. 
3.2.2 
Safety Population 
All patients who received at least one dose of PRN1008 in Part B will be included in the safety 
population. The Safety  population will be used for all safety analyses. 
3.2.3 
Efficacy Analysis/Intent-to-Treat (ITT) Population 
All patients who received at least one dose of PRN1008 in Part B will be included in the Efficacy/ITT 
analysis population. 
3.2.4 
Modified Intent-to-Treat (mITT) Population  
Patients treated up to Week 5 and had a Week 5 disease assessment will be included in the mITT 
population.  
3.2.5 
Completer Population 
Patients  treated up to Week 25 and had a Week 29 disease assessment will be included in the 
Completer population.  
3.2.6 
Per-Protocol (PP) Population 
The PP population will include patients who had:  
1) No major protocol deviations relevant to data integrity per Medical Monitor review 
2) At least 80% compliance based on drug accountability 
3) Completed Week 29 visit. 
3.2.7 
Pharmacokinetic Analysis Population 
The Pharmacokinetic Analysis Population will be described in the PK SAP, a separate document. 


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3.2.8 
Patients from Part A 
Two patients who were enrolled in Part A of the study also participated in Part B of the study. 
Patient 
 relapsed in Part A, was then requalified and entered into Part B.  In Part B, the patient’s 
number was 
. The date of last dose in Part A is 13MAR2018 and the date of first dose in Part B is 
06DEC2018.   In the Part B statistical documentation, the baseline values for the patient will be the 
measurements made prior to date of first study drug dosing in Part B.  
Patient 
 with Part A starting dose of 400 mg BID rolled over into the Part B study due to timing 
of protocol amendment version 5. This patient is included in the Part B analyses with starting dose of 
400 mg BID. 
3.3 Treatment Groups 
3.3.1 
Treatment Group Labels 
Patient disposition, demographics and other baseline data will be summarized by starting dose.  Efficacy 
and safety data will be summarized by the dose the patient received at or prior to the assessment or at 
or prior to the time of event (i.e. AE start date).  As a result, a given patient can be accounted for in 
multiple dose level groups. 
The following treatment group labels will be used in the tables, listings and figures: 
• 
Starting Dose 400 mg QD 
• 
Starting Dose 400 mg BID 
• 
400 mg QD 
• 
400 mg BID 
• 
600 mg BID  
• 
Overall 
• 
Post Last Dose 
One dosing error has been reported in the study.  Between the Week 13/Day 85 and Week 17/Day 113 
visits, patient 
 received 600 mg QD instead of 400 mg BID for 30 days; from 28MAY2019 to 
29JUN2019. The assessments or events reported under the 600 mg QD dose will be summarized in the 
Overall group only. 
3.4 Analysis Timepoints and Definitions 
The study visits in Part B are: 
• 
SCREENED  
• 
WEEK 1 DAY 1 
• 
WEEK 1 DAY 2 
• 
WEEK 3 DAY 15 
• 
WEEK 5 DAY 29 


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• 
WEEK 9 DAY 57 
• 
WEEK 13 DAY 85 
• 
WEEK 17 DAY 113 
• 
WEEK 21 DAY 141 
• 
WEEK 25 DAY 141 
• 
WEEK 25 DAY 169 
• 
UNSCHEDULED 
All scheduled visits will be summarized. ‘Last Visit’ will be summarized for selected parameters and is 
defined as the last non-missing post-baseline visit, including unscheduled visits. 
3.4.1 
Baseline for PDAI, ABSIS, ABQOL, TABQOL and SNAQ scores 
The value measured at Week 1 Day 1 pre-dose will be used as the Baseline value. If the Week 1  
Day 1 Pre-dose result is missing and the screening result is available, then the screening result will be 
used as Baseline.  
3.4.2 
Baseline for Lab and Vital Signs 
The value measured at Week 1 Day 1 pre-dose will be used as the Baseline value. If the Week 1 Day 1 
pre-dose result is missing and the screening result is available, then the screening result will be used as 
Baseline. 
3.4.3 
Pre-treatment vs. treatment emergent vs. post last dose adverse events 
Pre-treatment events are the events that started prior to the first study drug dosing.  
Treatment emergent adverse events (TEAEs) are the events started on or after the first study drug.  
Post last dose adverse events are the events that started after the last date of study drug dosing. 
3.4.4 
Prior medications vs. concomitant medications vs. post last dose medications 
Prior medications are the medications stopped before the date of first study drug dosing. 
Concomitant medications are the medications that are ongoing or stopped after the date of first study 
drug dosing and started on or prior to the last date of study drug dosing. 
Post last dose medications are medications that started after the last date of study drug dosing. 
3.4.5 
Study Day 
The date of first study drug dosing is considered as Study Day 1.  
• 
Study Day = (Date of assessment – date of first dose in Part B + 1), if the assessment date is on 
or after the date of first dose in Part B.  
• 
Study Day = (Date of assessment – date of first dose in Part B), if the assessment date is prior to 
the date of first dose in Part B. 


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3.4.6 
Time from Pemphigus Diagnosis (years) 
Time from event (years) = (Date of screening – First date of Pemphigus reported in Medical Hisotry + 
1)/365.25 
 
3.4.7 
Control of Disease Activity (CDA), Complete Remission (CR), End of Consolidation Phase (ECP) 
and Relapse 
Control of Disease Activity (CDA) is defined as the time at which new lesions cease to form and 
established lesions begin to heal. This is also considered the beginning of the consolidation phase. Date 
of CDA should be the first date CDA occurred i.e. a visit date or earlier date reported by the patient. If 
there is a relapse and a subsequent second CDA, visits from that second CDA visit forward would record 
the date of the second CDA. If CDA is yes, relapse must be no. 
 
Complete remission (CR) in this study is defined as the complete healing of all lesions and absence of 
new lesions on any dose of corticosteroid or no steroids. Date of CR should be the first date CR occurred 
i.e. a visit date or earlier date reported by the patient. The duration of time in CR e.g.  8 weeks is not 
included in the definition.  If CR is yes, CDA, ECP must be yes also, and relapse must be no. 
 
End of Consolidation Phase (ECP) is defined as the time at which no new lesions have developed for a 
minimum of 2 weeks and the majority (approximately 80%) of established lesions have healed. It is at 
this point that most clinicians begin to taper corticosteroid doses. Date of ECP should be the first date 
ECP occurred i.e. a visit date or earlier date reported by the patient. If ECP is yes, CDA must be yes also, 
and relapse must be no. 
 
Relapse (i.e. flare) is defined by the appearance of 3 or more new lesions in a month that do not heal 
spontaneously within 1 week, or by the extension of established lesions, in a patient who has achieved 
control of disease (i.e. CDA). Date of relapse should be the first date relapse occurred i.e. a visit date or 
earlier date reported by the patient. If relapse is yes, CDA, ECP and CR must be no. 
3.5 Data Handling 
3.5.1 
Partial dates 
Imputation of day, month and/or year for partial or missing AE onset dates will be performed as 
described below. 
• 
Day, month and year missing 
- 
Leave as missing, no imputation.  In this case, the adverse event will be considered as 
treatment emergent. 
• 
Day and month missing 
- 
If year of start date is the same as the year of the 1st dose of PRN1008, then impute day and 
month as the day and month of 1st dose of PRN1008, ELSE   
- 
Impute day and month as 31-DEC, if the year is before the year of the 1st dose of PRN1008, 
ELSE 


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- 
Impute day and month as 01-JAN, if the year is after the year of the 1st dose of PRN1008 
• 
Only day missing 
o If the year is the same as the year of the 1st dose of PRN1008 
▪ 
If the month is before the month of the 1st dose of PRN1008, then the last day 
of the month will be assigned to the missing day. 
▪ 
If the month is the same as the month of the 1st dose of PRN1008, then the day 
of the 1st dose of PRN1008 will be assigned to the missing day. 
▪ 
If the month is after the month of the 1st dose of PRN1008, then “01” will be 
assigned to the missing day. 
o If the year is before the year of the first dose of study drug, then the last day of the 
month will be assigned to the missing day. 
o If the year is after the year of the first dose of study drug, then “01” will be assigned to 
the missing day. 
 
Imputation of day, month and/or year for partial or missing medication stop dates will be performed as 
described below if the medication is not flagged as “Ongoing”. 
• 
Day, month and year missing  
- 
Leave as missing, no imputation.  In this case, the medication will be considered as 
concomitant. 
• 
Day and month missing 
- 
If year of stop date is the same as the year of the 1st dose of PRN1008, then impute day and 
month as the day and month of 1st dose of PRN1008, ELSE   
- 
Impute day and month as 31-DEC. 
• 
Only day missing 
- 
If month and year of start date are prior to the month and year of the 1st dose of PRN1008, 
then impute day as 01, ELSE 
- 
Impute day as day of 1st dose of PRN1008. 
Imputation of day and month for partial Date of PV Diagnosis (years) will be performed as described 
below. 
- 
If only day is missing, impute as 01 
- 
If day and month are missing, impute as 01-JAN 
3.5.2 
Missing data imputation 
For AE data, missing causality will be imputed to “Related” and missing intensity grade will be imputed 
to “Grade 3 – Severe”.  
Other missing data will not be imputed unless other specified. 


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3.5.3 
Lab values below or above a threshold 
Lab results below or above a threshold, e.g. “>= 50 mg/dL, will be excluded from descriptive statistics. 
The number of patients with results below or above the threshold will be presented for each lab 
parameter.  
3.6 Interim Analyses 
No interim analysis was planned for Part B of the study. 
3.7 Handling of Study Center Effects 
Study center effects will not be assessed.  
3.8 Documentation & Other Considerations 
Data are entered electronically into a clinical database built by Medidata RAVE and exported as SAS® 
datasets. SDTM and ADaM datasets will be generated following the Clinical Data Interchange Standards 
consortium (CDISC) conventions (SDTMIG v3.2 and ADaMIG v1.1). Summary tables and figures will be 
produced based on the ADaM datasets and patient data listings will be produced based on the SDTM 
datasets or raw clinical database. All tables, figures and listings will be produced using SAS® version 9.4.  
AEs are coded using the Medical Dictionary for Regulatory Activities (MedDRA) Version 18.0, to assign a 
system organ class (SOC) and preferred term (PT) to each AE. Prior and concomitant medications are 
coded to preferred drug names using the World Health Organization Drug Dictionary (WHODRUG) 
dictionary WHO-DDE-B2-June 2015. 
Continuous data will be summarized with the following descriptive statistics unless otherwise noted: 
number of observations, mean, standard deviation (SD), median, minimum, and maximum. Categorical 
data will be summarized with frequencies and percentages. Patient data listings will be listed patient ID 
and visit, unscheduled visits will be placed in chronological order. 
No hypothesis testing will be performed. 
For presentation of data, the mean and median will be presented to 1 decimal place greater than the 
original data, SD will be to 2 decimal places greater than the original data, and the minimum and 
maximum will have the same number of decimal places as the original data. Change from screening and 
change from baseline will be 1 decimal place greater than the observed statistics. The format for 
minimum and maximum will be “Min, Max”. Standard deviation will be abbreviated as “SD”. Percentage 
will be reported to 1 decimal place. 
For the shift tables, the Normal to Abnormal or Normal to Out-of-Range shifts will be highlighted in 
yellow. Zero (0) count and percentage will not be displayed. 
For the abnormal results listings, the abnormal or out-of-range results will be highlighted in yellow and 
clinically significant values will be in highlighted in a different color with bold font. 


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4 STATISTICAL METHODS 
4.1 Safety Endpoints 
The incidence of treatment-emergent AEs (TEAEs), including clinically significant changes in physical 
examination, laboratory tests, and vital signs. 
4.2 Primary Efficacy Endpoint 
The primary efficacy endpoint is the proportion of patients who are able to achieve control of disease 
activity (CDA) within 4 weeks of starting PRN1008 treatment without the need for doses of 
prednis(ol)one >0.5 mg/kg on or before the Week 5 visit.  
CDA is defined as the time at which new lesions cease to form and established lesions begin to heal. 
4.3 Secondary Efficacy Endpoints 
The secondary efficacy endpoints are:  
1) Cumulative incidence of patients able to achieve CDA by each visit 
2) Cumulative incidence of patients able to achieve CDA by each visit on zero steroids. 
3) Cumulative incidence of patients able to achieve CDA by each visit with the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
4) Cumulative incidence of patients able to achieve CDA by each visit without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
5) Proportion of patients able to achieve CDA at each visit 
6) Proportion of patients able to achieve CDA at each visit with the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
7) Proportion of patients able to achieve CDA at each visit without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
8) Cumulative incidence of patients able to achieve CR by each visit 
9) Cumulative incidence of patients able to achieve CR by each visit with the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
10) Cumulative incidence of patients able to achieve CR by each visit without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
11) Proportion of patients able to achieve CR at each visit 
12) Proportion of patients able to achieve CR at each visit with the need for doses of prednis(ol)one 
of greater than 0.5 mg/kg 
13) Proportion of patients able to achieve CR at each visit without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
14) Proportion of patients able to achieve a complete CR with the need for dose of prednis(ol)one 
of greater than 0.5 mg/kg within 24 weeks, i.e. on or prior to Week 25 visit 
15) Proportion of patients able to achieve a complete response (CR) without prednis(ol)one of 
greater than 0.5 mg/kg within 24 weeks, i.e. on or prior to Week 25 visit 


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16) Time to first CDA 
17) Time to first CR 
18) Time to first end of consolidation phase 
19) Time to first relapse (after CDA has been achieved, as per relapse definition)  
20) Time to first increase in PDAI 
21) Cumulative CS usage over 24 weeks 
22) Change from baseline in Pemphigus Disease Area Index (PDAI) and Autoimmune Bullous Skin 
Disorder Intensity Score (ABSIS) scores at each visit 
23) Change from baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL) and Treatment of 
Autoimmune Bullous Diseases Quality of Life (TABQOL) scores at each visit 
24) Change from baseline in appetite (SNAQ score) at each visit 
CR, End of consolidation phase, and Relapse definitions are listed below and noted where they differ 
listed below: 
▪ 
Complete response (CR): the absence of new or established lesions while the patient is receiving 
low dose CS, defined in this study as < or = 0.5mg/kg or 0, as stated in the specific endpoint. The 
duration of CR is not part of the definition for this study. 
▪ 
End of consolidation phase: The time at which no new lesions have developed for minimum of 2 
weeks, approximately 80% of lesions have healed, and when most clinicians start to taper 
steroids. 
▪ 
Relapse/flare: Appearance of ≥3 new lesions/month that do not heal spontaneously within 1 
week, or by extension of established lesions, in a patient who has achieved disease control 
4.4 Pharmacokinetic Outcome Measure 
Plasma concentrations of PRN1008 will be measured at approximately the time of maximum 
concentration on Day 1 and at various subsequent times during outpatient dosing.  
A separate SAP will describe the PK outcome measures and analyses.  PK results will be summarized in 
an independent report. 
4.5 Pharmacodynamic Outcome Measures 
• 
Percentage BTK occupancy for individuals in PBMCs will be determined at 2 & 24 hours after the first 
PRN1008 dose and at varied subsequent times during outpatient dosing 
• 
Actual result, change and percent change from screening and change and percent change from 
baseline in Anti-DSG-1-3 autoantibody levels by ELISA at various time points for all patients and by 
baseline Anti-DSG-1-3 values of ≥ 100 unit vs. < 100 unit.  
 
A separate SAP will be created for the BTK occupancy analysis and the results will be summarized in a 
separate report. 


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4.6 Exploratory Measures 
• 
Exploratory PK/PD analysis will examine the effects, if any, of covariates on PK and/or PD, and 
the relationship between PK, PD and efficacy in this population  
A separate SAP will be created for the PK/PD analysis and the results will be summarized in a separate 
report. 
4.7 Changes to Planned Analyses from Protocol 
Change 
Description  
See SAP 
section 
Analysis 
populations 
The following analysis populations are added: 
▪ 
Modified Intent-to-Treat (mITT) Population 
▪ 
Completer Population 
▪ 
Per-Protocol (PP) Population 
3.2.4, 
3.2.5, 
3.2.6 
Secondary 
endpoints 
The secondary endpoints are re-ordered to group all the CDA 
endpoints together and then followed by the CR endpoints. Some of 
the endpoints are re-worded for clarity and consistency within the 
SAP but the meaning and interpretation remained the same as 
defined in the protocol. 
4.3, 
5.4.2.1 
Additional 
secondary 
endpoints 
The following  secondary efficacy endpoints were not listed in the 
protocol but included in the Part A SAP.  They will be also analyzed for 
Part B of the study. 
• 
Proportion of patients able to achieve CDA at each visit 
• 
Proportion of patients able to achieve CDA at each visit with or 
without the need for doses of prednis(ol)one of greater than 0.5 
mg/kg 
• 
Cumulative incidence of patients ever achieved CDA by each visit 
• 
Cumulative incidence of patients able to achieve CDA by each 
visit on zero steroids. 
• 
Cumulative incidence of patients ever achieved CDA by each visit 
with or without the need for doses of prednis(ol)one of greater 
than 0.5 mg/kg 
• 
Proportion of patients able to achieve CR at each visit 
• 
Proportion of patients able to achieve CR at each visit with or 
without the need for doses of prednis(ol)one of greater than 0.5 
mg/kg 
• 
Cumulative incidence of patients ever achieved CR by each visit 
4.3 


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• 
Cumulative incidence of patients ever achieved CR by each visit 
with or without the need for doses of prednis(ol)one of greater 
than 0.5 mg/kg 
• 
Time to first increase in PDAI 
 
5 ANALYSIS DETAILS 
5.1 Patient Disposition, Demographics and Baseline Characteristics 
The Screening population will be used for patient disposition and reason for screen fails. The ITT 
population will be used for demographics and baseline characteristics. Descriptive statistics will be done 
by starting dose and overall, unless specified otherwise. Patient data listings will be listed by starting 
dose and patient and start date of event, if applicable. 
5.1.1 
Patient Disposition 
The number and percentage of patients will be tabulated for patients who: 
• 
Enrolled into the study (Screening population) 
• 
Had at least one dose of PRN1008 (Safety and ITT populations) 
• 
Completed treatment up to Week 5 and had a Week 5 disease assessment (mITT population) 
• 
Had no major protocol deviations, completed Week 29/Day 197 visit and had at least 80% study 
drug compliance (PP population) 
• 
Completed the study 
• 
Early discontinued 
• 
Reason for early discontinuation for patients who early discontinued 
- 
Adverse Event 
- 
Death 
- 
Lack of Efficacy 
- 
Lost to Follow-up 
- 
Non-Compliance with Study Drug 
- 
Physician Decision 
- 
Pregnancy 
- 
Progressive Disease 
- 
Recovery 
- 
Trial Screen Failure 
- 
Study Terminated by Sponsor 
- 
Technical Problem 
- 
Withdrawal by Subject 
- 
Other 


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The number and percentage of patients who attended each visit and the number of percentages of 
patients enrolled by country and study site will also be tabulated.  
Detailed disposition, study enrollment and analysis population information will be listed. 
Screen failures will be listed with date and reason for screen failure. 
5.1.2 
Demographics and Baseline Characteristics 
Demographics variables including age, gender, fertility status and method of birth control for female 
patients, race, ethnicity, height at screening (cm), weight at screening (kg) and body mass index (BMI) 
(kg/m2) at screening will be summarized by descriptive statistics and listed in a patient data listing. 
5.1.3 
Disease History 
Disease history data including pemphigus subtype at screening, time from pemphigus diagnosis (by the 
earliest medical history record) date to screen date (years), Pemphigus history type (Newly diagnosed 
vs. Relapsed), baseline Anti-DSG 1 and Anti-DSG 3 antibody status/profile, Total Anti-DSG at baseline (< 
100 vs ≥100) PDAI total activity score, Pemphigus severity at baseline (PDAI < 15 (mild-moderate) or 
PDAI ≥ 15 (moderate-severe)), PDAI mucous membrane activity (% >0), PDAI scalp activity (% >0), PDAI 
skin activity (% >0) and ABSIS oral activity (% >0) will be summarized in a frequency table and listed.  
Newly diagnosed pemphigus is defined as subjects who were diagnosed at most 180 days prior to date 
of screening, otherwise the patient is defined as relapsed. 
Baseline Anti-DSG status will be determined by the central lab results and presented in the following 
categories: 
• 
Anti-DSG 1 positive, Anti-DSG 3 negative; 
• 
Anti-DSG 1 negative, Anti-DSG 3 positive; 
• 
Anti-DSG 1 positive, Anti-DSG 3 positive; 
• 
Anti-DSG 1 negative, Anti-DSG 3 negative 
Ranges for Anti-DSG status: 
 
Status 
Range 
Anti-DSG 1 
positive 
≥ 14 
 
negative 
< 14 
Anti-DSG 3 
positive 
≥ 9 
 
negative 
< 9 
 
5.1.4 
Medical History 
Medical history events are reported for the following Body Systems: Skin, Head-Eyes-Ears-Nose-Throat, 
Respiratory, Cardiovascular, Gastrointestinal, Endocrine/Metabolic, Genitourinary, Neurological, 
Blood/Lymphatic, Musculoskeletal, Hepatic, Allergies, Psychological/Psychiatric and Other. The medical 
history events will be summarized by Body System, Starting dose and overall. 


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Medical history data will be listed in a patient data listing. 
5.1.5 
Prior and Concomitant Medications 
All medications will be coded according to the WHODRUG dictionary WHO-DDE-B2-June 2015. 
The following prior medications will be summarized by Level 2 Classification and Preferred Name for 
each starting dose and overall: 
• 
Previous corticosteroid medications taken and stopped prior to date of first study drug dosing 
• 
Other medications taken and stopped prior to date of first study drug dosing 
The following concomitant medications will be summarized by Level 2 Classification and Preferred Name 
for each dose level and overall: 
• 
Corticosteroid medications taken during treatment 
• 
Corticosteroid medications started after date of last study drug dosing 
• 
Other medications taken on or after date of first study drug dosing 
Medications will be attributed to the dose level where the medication is taken during the dose level. The 
same medication may be attributed to more than one dose level. Corticosteroid medication started 
after the date of last study drug dosing will be summarized separately. 
Separate listings will be provided for prior corticosteroid treatment, concomitant corticosteroid 
medications, post last dose corticosteroid medications, other prior medications and other concomitant 
medications. 
5.2 Protocol Deviations 
Protocol deviations will be identified and classified into major vs. non-major by the Sponsor prior to 
database lock.   
The major protocol deviations will be tabulated by the deviation categories, starting dose and overall for 
patients who are in the ITT population.  
A patient data listing with deviation category and deviation details for both will be provided for the 
major protocol deviations and the non-major protocol deviations. 
5.3 Study Drug Treatment Exposure and Compliance 
Study drug treatment information will be summarized and documented in the ITT and mITT Population.  
Number of dose adjustments and maximum dose will be tabulated by starting dose. Descriptive 
statistics for study drug exposure and compliance will be done by dose level and overall. Patient data 
listings will be listed by starting dose and patient and study visit.  
5.3.1 
Study Drug Exposure 
Study drug exposure will be summarized by dose level. 


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• 
Duration of exposure for Dose level i (days) = 
(date of last dose for dose level i – date of first dose for dose level i) + 1. 
 
• 
Dose Taken for Dose level i (mg) = (Number of tablets dispensed – Number of tablets returned)* 
Current Dose Level (mg) 
• 
Calculated Dose for Dose level i (mg/day) = 
(Dose taken for Dose level i )/Duration of exposure for Dose level i. 
5.3.2 
Study Drug Compliance 
Compliance with study drug at each dose level and overall will be calculated as the actual number of 
doses taken divided by the expected number of doses to be taken (taking into account the # tablets for 
each dose), expressed as a percentage.  Expected number of doses will be determined by the number of 
days in between visits and the frequency of dosing (QD or BID). 
Compliance will be summarized by descriptive statistics for each dose level and overall. The number and 
percentage of patients with 100% compliance, greater than 80% but less than 100% compliance and less 
than 80% compliance will be tabulated for each dose level. 
5.4 Efficacy Analyses 
The mITT population will be used for all efficacy analyses. For analyses at a particular study drug dose, 
all patients who received that dose at least once will be included. In addition, for selected analyses, 
where specified, the Completer and PP population will be used.  
The descriptive statistics will be done by dose level and overall for all study visits, unless specified 
otherwise.  All binomial confidence intervals for efficacy analyses will use the Exact (Clopper-Pearson) 
method.  Patient data listings will be listed by starting dose, patient, dose level and study visit. 
5.4.1 
Primary Efficacy Endpoint 
The primary efficacy endpoint is: 
▪ 
The proportion of patients who are able to achieve CDA within 4 weeks (on or prior to Week 5 
visit) of starting PRN1008 treatment without the need for doses of prednis(ol)one > 0.5 mg/kg 
The Control of Disease Activity (CDA) responses are assessed at visits specified in the Schedule of 
assessments (Table 1 ). Types of CS other than prednis(ol)one are converted to dose-equivalent 
prednis(ol)one (see Section 5.4.2.3). 
This is operationalized as meaning the first CDA is achieved on or before the Week 5 visit but after first 
dose of PRN1008, and will be summarized, by dose level to the Week 5 visit and overall in a table, using 
frequencies and percentages. In addition, two-sided 80% and 95% CIs (Confidence Intervals) of the 
response rate will be provided for each dose level and overall.  
 


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Patients who are able to achieve CDA between first dose of study drug and the Week 5 visit without 
prednis(ol)one >0.5 mg/kg on the date prior to the CDA will be considered as responders, even if the 
CDA status was not maintained or higher doses of prednis(ol)one were used after the first CDA. Patients 
who are not able to achieve CDA prior to or at the Week 5 visit or  had prednis(ol)one >0.5 mg/kg on the 
date prior to CDA will be considered as non-responders. Patients whose CDA status is unknown at the 
Week 5 visit for whatever reason (including early termination before Week 5 visit) will be also 
considered as non-responders. Only systemic CSs (as defined by medication taken orally or 
intravenously) are to be used to evaluate the efficacy endpoints, topical CSs (including buccal CS) will 
not be counted.  
The number and percentage of patients who meet the primary efficacy endpoint criteria will be 
tabulated by dose level and overall (all patients).   
A sensitivity analysis using the Completer and PP population will be provided.    
Panel plots will be provided for PDAI score over the study period for each patient. Dose level (mg/day) 
will also be plotted on the same figure. Lines for the steroid equivalent dose in mg/kg will be added to 
the plots. A total of 9 plots (3 x 3) will be fitted on one page. 
5.4.1.1 
Subset Analyses 
The robustness of the primary endpoint results will be explored by calculating the number and percent 
of patients meeting the primary efficacy endpoint overall (i.e. across all doses) within the following 
patient subsets: 
1) Age (< 50 years, ≥ 50 years) 
2) Gender 
3) Total anti-desmoglein (DSG) antibody (≥ 100, < 100 units) at baseline. 
4) Pemphigus History Type: Newly diagnosed (< 6 months from screening) vs. relapsed (> 6 
months from screening) 
5) Pemphigus anti-DSG Profile at baseline: Pemphigus Vulgaris (PV) vs. Pemphigus foliaceus (PF) 
vs. negative anti-DSG profile, defined as follows: 
o PV: anti-DSG3 positive with anti-DSG1 negative or anti-DSG3 positive with anti-DSG1 
positive.  
o PF: anti-DSG1 positive only  
o Negative anti-DSG profile: neither detectable.  
6) Pemphigus Severity: PDAI < 15 (mild-moderate) vs PDAI ≥ 15 (moderate-severe) at baseline If 
screening PDAI is not available use Week 1 Day 1 Pre-dose.  
A sensitivity analysis using the Completer and PP populations will be provided for each subset analysis. 


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5.4.2 
Secondary Efficacy Endpoints 
5.4.2.1 
CDA Responses 
The number and percentage of patients who meet the criteria below will be tabulated by dose level and 
overall where the response first occurred and overall. 80% and 95% CIs will be calculated for the 
proportions using the Exact (Clopper-Pearson) method . 
1) Cumulative incidence of patients able to achieve CDA by each visit 
2) Cumulative incidence of patients able to achieve CDA by each visit on zero steroids. 
3) Cumulative incidence of patients able to achieve CDA by each visit with the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
4) Cumulative incidence of patients able to achieve CDA by each visit without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
5) Proportion of patients able to achieve CDA at each visit 
6) Proportion of patients able to achieve CDA at each visit with the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
7) Proportion of patients able to achieve CDA at each visit without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
8) Cumulative incidence of patients able to achieve CR by each visit 
9) Cumulative incidence of patients able to achieve CR by each visit with the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
10) Cumulative incidence of patients able to achieve CR by each visit without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
11) Proportion of patients able to achieve CR at each visit 
12) Proportion of patients able to achieve CR at each visit with the need for doses of prednis(ol)one 
of greater than 0.5 mg/kg 
13) Proportion of patients able to achieve CR at each visit without the need for doses of 
prednis(ol)one of greater than 0.5 mg/kg 
14) Proportion of patients able to achieve a complete CR with the need for dose of prednis(ol)one 
of greater than 0.5 mg/kg within 24 weeks, i.e. on or prior to Week 25 visit 
15) Proportion of patients able to achieve a complete response (CR) without prednis(ol)one of 
greater than 0.5 mg/kg within 24 weeks, i.e. on or prior to Week 25 visit 
 
The CDA status, CR status, ECP status, and Relapse status by visit, by patient will be presented in a 
listing.  
Duration of CR will be summarized and listed, and is defined by the longest period of consecutive visits 
in CR (last known CR date – first known CR date + 1). Each subject will only have one CR duration as a 
result. 
 
A sensitivity analysis using the Completer and PP populations will be provided for all analyses of CDA 
response. 


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5.4.2.2 
Time-to-Event 
The following time-to-event analyses will be performed by dose level and overall for events occurred 
after date of first study drug dosing: 
 
a) Time to first CDA 
b) Time to first CR 
c) Time to first ECP 
d) Time to first relapse (after CDA has been achieved, as per the definition of relapse) after first 
dose of study drug 
e) Time to first increase in PDAI   
 
For each event listed above, the number of days to the event will be calculated as: 
 
(Date of first occurrence of event – Date of first study drug dosing of the dose level where the event 
is observed) + 1 
 
The number of days to first occurrence of each event (a to d) will be summarized by descriptive statistics 
for each dose level and overall.  
 
Patients who did not achieve the specific event are censored at the last day in a dose level or date of 
study completion/discontinuation. 
 
Kaplan-Meier estimates of quartiles in days and their associated 80% and 95% CI will be calculated by 
using the LOGLOG transformation. Minimum and maximum survival times in days as well as number of 
the patients and the probability of experiencing the event on or before specific date will be provided in a 
table. 
 
A sensitivity analysis using the Completer and PP populations will be provided.    
5.4.2.3 
Cumulative Corticosteroid (CS) Usage 
Cumulative corticosteroid (CS) usage per patient (mg) will be presented by treatment period (up to first 
12 weeks, after first 12 weeks, entire treatment period and follow-up period), by dose level and overall 
in a table.  To calculate this endpoint, the following steps will be followed: 
Step 1: Select CSs with route = “ORAL” or “INTRAVENOUS”. The CSs will be converted into 
prednisolone/prednisone dosage equivalent using the below conversion chart.  
 
CS medication 
Conversion factor 
Prednisone, Prednisolone    
1 
Hydrocortisone  
0.25 
Methylprednisolone, Methylprednisolone Sodium 
Succinate 
1.25 


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Dexamethasone, Betamethasone      
6.67 
Step 2: The CSs usage will be converted to standard frequency using the following conversion chart.  
 
Frequency 
Unit 
Conversion 
factor 
QD, Q24H, ONCE, CONTINUOUS, BD PRN, PRN 
mg 
1 
5 TIME A DAY 
mg 
5 
QID 
mg 
4 
TID 
mg 
3 
2-3 DAILY 
mg 
2.5 
BID 
mg 
2 
QOD, EVERY 2 DAYS 
mg 
1/2 
BIS 
mg 
2/7 
QS, EVERY WEEK 
mg 
1/7 
Any value 
mg/day 
1 
 
Once the prednisolone/prednisone dosage equivalent and frequency are converted as above, each 
record of CSs taken will be converted to prednisolone with standard frequency (mg/day). 
Step 3: The duration for each record of CS taken will be calculated using the formula: Duration = CS end 
date – CS start date + 1, in days. Any partial start or end date of CSs taken will be imputed using the 
algorithm specified in Section 3.5.1. If the CS start date is completely missing, then the date will be 
imputed as Week 1 Day 1, i.e. it is assumed that the medication is taken at the study entry. If the CS stop 
date is completely missing, then the date will be imputed as Date of End of Study, i.e. it is assumed that 
the medication is taken up to the end of study. 
If the CS start date is before the Week 1 Day, then the CS start date will be replaced by the Week 1 Day 
1; if the CS start date is after the date of Week 29 (Day 197) - 1, this record will not be used to 
summarize cumulative dose for CSs. 
If the CS end date is after the date of Week 29 (Day 197) - 1, then the CS end date will be replaced by 
the date of Week 29 (Day 197) - 1; if the CS end date is before the study treatment start date, this 
record will not be used to summarize cumulative dose for CSs.  
Note that above date adjustments only apply to cumulative CS dose calculation. Other analyses, where 
CS dose is needed, will use CS start and end dates as reported.  
Step 4: The “record” level cumulative CSs usage will be calculated as: record level cumulative dose = 
prednisolone/prednisone dosage equivalent * conversion factor for frequency * duration (days). 
Step 5: The patient level cumulative CSs usage will be calculated as the summation of all the non-missing 
record level cumulative doses. If a patient had no CS records that meet the above criteria, the CS 


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cumulative dose is set as 0. The average daily CS usage in mg/day will be calculated as the cumulative 
doses dividual the number of days within a study period. 
All CS usage will be presented in a listing showing record level prednisone dosage equivalent (mg), dose, 
frequency, route, start and stop dates. The total prednisone dosage equivalent in mg/kg will also be 
calculated by using the last measured weight prior to the current visit. 
5.4.2.4 
CS Dose on the Day Before Each Visit 
For each patient on the day before each scheduled visit, dosage level of CS dose in mg/day and 
mg/kg/day will be summarized using summary statistics by dose level and overall. Types of CS other 
than prednis(ol)one are converted to dose-equivalent prednis(ol)one (see Section 5.4.2.3). 
For Screening and Week 1/Day 1 visits, however, the CS dose is set as the day of, rather than the day 
before. 
If a patient did not report any CS, then the dosage level is set as 0. If a subject is terminated prior to a 
visit, then this subject is not included in the calculation for visit. 
The same table will be repeated for: 1) patients achieved at least one CDA and 2) to included patients 
achieved at least one. 
A listing of the CS dose in my/day and mg/kg/day will be listed with the CDA and CR response at each 
visit.Change from Baseline in PDAI ABSIS, ABQOL and TABQOL Scores 
Quantitative assessment of patient response and disease progression will be determined using the 
following scores at visits specified in the Schedule of assessments (Table 1 ): 
1) Pemphigus Disease Area Index (PDAI) score – Total Activity Score 
2) Pemphigus Disease Area Index (PDAI) score – Total Damage Score 
3) Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) score 
4) Autoimmune Bullous Disease Quality of Life (ABQOL) score 
5) Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL) score 
The PDAI questionnaire has two components including activity and damage. The activity component 
consists of skin, scalp and mucosa parts and the damage component consists of skin and scalp parts. 
Actual values, change from screening, percent change from screening, change from baseline and 
percent change from baseline in PDAI total activity, total damage scores will be summarized by dose 
level and overall, respectively. 
The PDAI activity or damage total scores per body part will be calculated as the sum of the scores on 
that body part as follows. PDAI total activity and total damage scores will be calculated as follows: 
▪ 
PDAI total activity score = Total skin activity + Total scalp activity + Total mucosa activity 
▪ 
PDAI total damage score = Total skin damage + Total scalp damage 


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The ABSIS questionnaire has three components, including skin involvement, oral involvement and (oral) 
severity. The ABSIS scores will be calculated separately for each component. The ABSIS total activity 
score and total ABSIS score will also be calculated as follows: 
▪ 
Skin involvement total score = sum of (%BSA * Weighting factor for each skin involvement area)  
▪ 
Oral involvement total score = sum of (score from each oral part) 
▪ 
(Oral) Severity total score = sum of (Level * Factor of discomfort for each food item) 
▪ 
ABSIS total activity score = Skin involvement total score + Oral involvement total score 
▪ 
Total ABSIS score = Skin involvement total score + Oral involvement total score + Severity total 
score 
Actual values and change from baseline in ABSIS skin involvement total score, ABSIS oral involvement 
total score and ABSIS total activity score will be summarized for all patients, and for the two sub-groups: 
▪ 
baseline oral involvement total score = 0 vs. >0 
▪ 
baseline skin involvement score = 0 vs >0.  
When calculating skin involvement total score, if %BSA = 0, then skin involvement score component is 0. 
If %BSA > 0, then weighting factor needs to be considered, and if weighting factor is missing in this 
circumstance, then the skin involvement total score and ABSIS total score are considered missing.  
The ABQOL and TABQOL questionnaires both contains 17 questions with numerical scores. The ABQOL 
and TABQOL scores will be calculated as the sum of scores from each question. Actual values and 
change from baseline in ABQOL and TABQOL scores will also be summarized by dose level and overall in 
a table.  If individual scores are missing, then the total scores are set as missing. 
PDAI, ABSIS, ABQOL and TABQOL scores and sub-scores (for PDAI and ABSIS) by patient and visit will be 
presented in separate listings. 
5.4.2.5 
Change from Baseline in Simple Nutritional Appetite Questionnaire (SNAQ) score 
Simple Nutritional Appetite Questionnaire (SNAQ) contains 4 questions with numerical scores and is 
assessed at visits specified in the Schedule of assessments (Table 1 ). The SNAQ score will be calculated 
as the sum of scores from each question. The SNAQ score ranges from 4 to 20.  If individual scores are 
missing, then the total score is set as missing. 
Actual values and change from baseline in SNAQ score at each visit will be summarized by dose level and 
overall. 
The SNAQ score will be presented in a listing by patient and visit. 


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5.4.2.1 
Change from Baseline and Percent Change from Baseline in Total Anti-DSG, Anti-DSG 1 
and Anti-DSG  
The Anti-DSG 1 and Anti-DSG 3 results are measured at visits specified in the Schedule of assessments 
(Table 1 ).  Total Anti-DSG is the sum  of Anti-DSG 1 and Anti-DSG3, where at least one of Anti-DSG 1 or 3 
is positive per Section 5.1.3, i.e. Anti-DSG 1 ≥ 14 and/or Anti-DSG 3 ≥ 9.  
Actual result, change from baseline and percent change from baseline in Total Anti-DSG, positive Anti-
DSG1, i.e. ≥ 14, and positive Anti-DSG 3, i.e. ≥ 9, at each visit will be summarized by dose level and 
overall. The same descriptive statistics will be produced by baseline Anti-DSG value (>= 100 units vs. < 
100 unit).  
The Total Anti-DSG, Anti-DSG 1 and Anti-DSG 3 values will be presented in a listing by patient and visit. 
5.5 Safety Analyses 
The Safety Population will be used for the safety analyses. The summary statistics will be done by dose 
level and overall for all study visits, unless specified otherwise. Patient data listings will be listed by 
starting dose, patient, dose level and study visit or start date of event if applicable. 
5.5.1 
Adverse Events 
TEAEs will be attributed to the dose level where the adverse event start date is on or after the start date 
of that dose level and before the next dose level if applicable.  
Adverse events with preferred term (PT) = “Pemphigus” will be excluded from the TEAE tables. All 
pemphigus PT are Disease Under Study and will be documented in a separate summary table and a 
separate patient data listing labeled as Disease Under Study Events. 
A summary of adverse events by dose level and overall will include: 
• 
The number of pre-treatment AEs and the number/percentage of patients reporting at least one 
pre-treatment AE 
• 
The number of pre-treatment SAEs and the number/percentage of patients reporting at least 
one pre-treatment SAE 
• 
The number of TEAEs and the number/percentage of patients reporting at least one TEAE 
• 
The number of post last dose adverse events and the number/percentage of patients reporting 
at least one post last dose adverse event 
• 
The number of related TEAEs and the number/percentage of patients reporting at least one 
related TEAE 
• 
The number of serious TEAEs and the number/percentage of patients reporting at least one 
serious TEAE 
• 
The number of related serious TEAEs and the number/percentage of patients reporting at least 
one related serious TEAE 


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• 
The number of TEAEs leading to study discontinuation and the number/percentage of patients 
reporting at least one TEAE leading to study discontinuation 
• 
The number of deaths and the number/percentage of patients reporting at least one TEAE death 
• 
The number/percentage of patients reporting TEAEs by Action Taken with Study Drug 
• 
The number/percentage of patients reporting TEAEs by the maximum grade  
TEAEs will be summarized by SOC and PT by dose level and overall. Patients with multiple events within 
a particular SOC or PT within each dose level will be counted once within that dose level. The number of 
episodes of events for each SOC and PT will also be displayed. The same tabulation will be performed for 
related TEAEs, SAEs and AE leading to study drug discontinuation. 
TEAEs will also be summarized by the maximum intensity grade (Grade 1 to 5), SOC and PT. Patients 
with multiple events within a particular SOC or PT will be counted once under the maximum intensity 
grade. The same tabulation will be performed for related TEAEs.   
Patient data listings will be generated for: 
• 
Prior adverse events 
• 
Prior SAE 
• 
TEAEs 
• 
Related TEAEs 
• 
SAEs 
• 
TEAE leading to study drug discontinuation 
• 
TEAE leading to death (AE with intensity = Grade 5 – Death related AE) 
• 
Post last dose TEAEs 
• 
Disease Under Study (Pemphigus PT events) 
5.5.2 
Laboratory Data 
The following laboratory parameters are measured at visits specified in the Schedule of Assessments 
(Table 1 ). 
Laboratory Panel 
Test Parameters 
Hematology 
hemoglobin, hematocrit, erythrocyte count (RBCs), thrombocyte count 
(platelets), leukocyte count (WBCs) with differential in absolute counts 
(including neutrophils, eosinophils, basophils, lymphocytes, and monocytes) 
Coagulation 
PT/INR, thrombin time, aPTT, fibrinogen level. 
Serum Chemistry  
aspartate aminotransferase (AST), slanine aminotransferase (ALT), total, 
direct, and indirect bilirubin levels, alkaline phosphatase (ALP), albumin, 
creatinine, urea, total protein, sodium, chloride, calcium, phosphate, 
potassium, glucose (random), creatine phosphokinase (CPK) and thyroid 
stimulating hormone (TSH), chloride, calcium, phosphate, potassium, 
glucose (random), creatinine phosphokinase (CPK) and serum pregnancy 
Urinalysis 
pH, specific gravity, protein, glucose, ketones, bilirubin, blood, nitrites, 
urobilinogen and leukocytes measured by dipstick 


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Laboratory Panel 
Test Parameters 
Serology 
HIV, hepatitis B and hepatitis C and QuantiFERON® TB testing at screening 
only 
 
Serology tests are only measured at screening. These results will be presented in a patient data listing. 
For other laboratory panels, the actual values and change from baseline values in numerical results will 
be summarized by dose level and overall. For categorical results, the frequency counts and percentages 
will be tabulated by each dose level and overall. 
Shifts from baseline tables will be provided for all laboratory parameters where low/normal/high can be 
determined by the normal ranges or can be classified as normal/abnormal. 
A special group of clinical laboratory tests, including ALT, AST, TBL and ALP, which are believed to be the 
indicators for drug induced liver injury (DILI) will be assessed separately. Frequency tables will be used 
to summarize elevations in these parameters.  The following criteria will be documented: 
• 
AST > 3x, 5x, 10x and 20x Upper Limit of Normal (ULN) 
• 
ALT > 3x, 5x, 10x, and 20x ULN 
• 
TBL > 1.5x and 2x ULN 
• 
ALP > 1.5x ULN 
• 
ALT or AST > 3x ULN and Total Bilirubin > 1.5x ULN 
• 
ALT or AST > 3x ULN and Total Bilirubin > 2x ULN 
Scatter plots of the peak TBL divided by the ULN will be plotted on a log scale against the peak ALT 
divided by the ULN. Reference lines will be plotted for the ULN for TBL and ALT, 2x ULN for TBL and 3x 
ULN for ALT. Any patients in the upper right quadrant, which is defined by 2x ULN for TBL and 3x ULN for 
ALT, would represent cases to be investigate for potential DILI. 
Patient data listings will be provided for each laboratory panel and the DILI parameters. Separate listings 
will be provided for patients with out of range results or abnormal results at any visits. 
5.5.3 
Vital Signs and Body Weight 
Vital signs include blood pressures (BP), pulse, temperature and respiratory rate and body weight are 
measured at visits specified in the Schedule of assessments (Table 1 ).  
The actual values and change from baseline values will be summarized by dose level and overall for each 
visit. A separate table will be generated to include only the baseline, Day 85, Day 169 and Day 197 visits. 
The following ranges will be used to classified the BP and pulse measurements as Low/Normal/High: 
• 
Systolic blood pressure (SBP): 90 to 140 mmHg, inclusive 
• 
Diastolic blood pressure (DBP): 60 to 80 mmHg, inclusive 
• 
Pulse: 40 to 100 bpm, inclusive 
Shift from baseline tables will be produced by using the above criteria. 


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Patient data listings will be provided for the vital sign parameters and body weight. Separate listings will 
be provided for patients with out of range SBP, DBP or pulse results at any visits. 
5.5.4 
12-Lead ECG 
ECG assessment is performed at the screening visit and unscheduled visit, if clinically indicated.  
The ECG parameters include: normal sinus rhythm? (yes or no), ventricular rate, P-R interval, QRS 
interval, QT duration, QTcF, QTcB interval and overall ECG interpretation (normal, abnormal – not 
clinically significant (NCS) or abnormal – clinically significant (CS)). 
For the numerical values, actual values will be summarized by descriptive statistics for each dose level 
and overall. For normal sinus rhythm and ECG interpretation, the results will be tabulated by each dose 
level and overall in a shift from baseline table.  
A patient data listing will be provided for the ECG results. 
5.5.5 
Physical Examinations 
Physical examinations are performed as specified in the Schedule of Assessments (Table 1 ). 
At the screening visit, a complete physical examination consists of checking the normality or abnormality 
of the following body systems: general appearance, skin, eyes, ears, nose, throat, heart, chest/breast, 
abdomen, neurological system, lymph nodes, skeletal and other.  
At other visits, an abbreviated physical examination consists of checking the normality or abnormality of 
the following body systems: general appearance, abdomen, cardiorespiratory and other. 
The number and percentage of patients with assessments of normal, abnormal NCS, abnormal CS or Not 
Done at all scheduled visits, will be summarized in a frequency table for each body system by dose level 
and overall.  
Patient data listings will be provided for all physical examinations results. Separate listings will be 
provided for patients with abnormal NCS or abnormal CS findings at any visits. 
5.5.6 
Female Reproductive Status and Pregnancy Test Results 
The fertility status and method of birth control used for women is reported at the screening visit. The 
pregnancy test is performed for women of childbearing potential only. A pregnancy serum test is done 
at the screening visit and urine dipstick tests are done at other time points. The reproductive status as 
well as serum and urine pregnancy test results will be presented. 
5.5.7 
Food Intake Status 
Date and time of food intake and type of food intake (liquid, solid such as toast or not applicable) are 
reported at each visit. A listing of patient food intake status will be presented. 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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6 MOCK SHELLS FOR TABLES, LISTINGS AND FIGURES (TLFS) 
The mock shells define the layout of the TFLs and may be adjusted to incorporate the actual data collected. The decimals shown on the 
shells are examples, the actual decimals to be presented will be based on the precision of the data collected. 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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TABLE OF CONTENTS 
Table B.14.1.1.1 
Patient Disposition – Completion Status (Screening Population) ........................................................................................ 52 
Table B.14.1.1.2 
Patient Disposition by Study Visit (ITT Population) .............................................................................................................. 54 
Table B.14.1.1.3 
Patient Disposition by Country and Study Site (ITT Population) .......................................................................................... 55 
Table B.14.1.1.4 
Analysis Populations (ITT Population) .................................................................................................................................. 56 
Table B.14.1.2.1 
Demographics and Baseline Characteristics (ITT Population) .............................................................................................. 58 
Table B.14.1.3.1 
Disease History (ITT Population) .......................................................................................................................................... 61 
Table B.14.1.4.1 
Medical History (ITT Population) ......................................................................................................................................... 64 
Table B.14.1.5.1 
Major Protocol Deviations (ITT Population) ......................................................................................................................... 65 
Table B.14.1.6.1 
Prior Corticosteroid Medications (ITT Population) .............................................................................................................. 66 
Table B.14.1.6.2 
Concomitant Corticosteroid Medications (ITT Population) ................................................................................................. 67 
Table B.14.1.6.3 
Concomitant Corticosteroid Medications Started Post Last Dose (ITT Population) ............................................................ 68 
Table B.14.1.7.1 
Prior Medications other than Corticosteroid Medications (ITT Population) ....................................................................... 69 
Table B.14.1.7.2 
Concomitant Medications other than Corticosteroid Medications (ITT Population) .......................................................... 70 
Table B.14.1.7.3 
Concomitant Medications other than Corticosteroid Medications Started Post Last Dose (ITT Population)...................... 71 
Table B.14.1.8.1 
Number of Dose Adjustments and Maximum Dose (ITT Population) .................................................................................. 72 
Table B.14.1.8.2 
Study Drug Exposure and Compliance (ITT Population) ...................................................................................................... 73 
Table B.14.2.1.1.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg (mITT Population) ......... 75 
Table B.14.2.1.1.2 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg (Completer Population) 75 
Table B.14.2.1.1.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg (PP Population) ............. 75 
Table B.14.2.1.2.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Age (mITT 
Population) ................................................................................................................................................................................. 76 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Table B.14.2.1.2.2 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Age 
(Completer Population) .............................................................................................................................................................. 76 
Table B.14.2.1.2.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Age (PP 
Population) ................................................................................................................................................................................. 76 
Table B.14.2.1.3.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Gender 
(mITT Population) ....................................................................................................................................................................... 76 
Table B.14.2.1.3.2 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Gender 
(Completer Population) .............................................................................................................................................................. 76 
Table B.14.2.1.3.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Gender 
(PP Population) ........................................................................................................................................................................... 76 
Table B.14.2.1.4.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Baseline 
Total AntiDSG (mITT Population) ............................................................................................................................................... 77 
Table B.14.2.1.4. Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Baseline 
Total Anti-DSG (Completer Population) ..................................................................................................................................... 77 
Table B.14.2.1.4.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Baseline 
Total Anti-DSG (PP Population) .................................................................................................................................................. 77 
Table B.14.2.1.5.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – 
Pemphigus History (mITT Population) ........................................................................................................................................ 77 
Table B.14.2.1.5.2 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – 
Pemphigus History (Completer Population) ............................................................................................................................... 77 
Table B.14.2.1.5.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – 
Pemphigus History (PP Population) ............................................................................................................................................ 77 
Table B.14.2.1.6.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Baseline 
Pemphigus Anti-DSG Profile (mITT Population) ......................................................................................................................... 77 
Table B.14.2.1.6.2 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Baseline 
Pemphigus Anti-DSG Profile (Completer Population) ................................................................................................................ 77 
Table B.14.2.1.6.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Baseline 
Pemphigus Anti-DSG Profile (PP Population) ............................................................................................................................. 77 


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Table B.14.2.1.7.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup Analysis – 
Baseline Pemphigus Severity (mITT Population) ........................................................................................................................ 77 
Table B.14.2.1.7.2 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup Analysis – 
Baseline Pemphigus Severity (Completer Population) ............................................................................................................... 77 
Table B.14.2.1.7.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup Analysis – 
Baseline Pemphigus Severity (PP Population) ............................................................................................................................ 77 
Table B.14.2.2.1.1 Cumulative Incidence of Patients Achieved CDA by Visit – Cumulative Incidence (mITT Population) ................................. 78 
Table B.14.2.2.1.2 Cumulative Incidence of Patients Achieved CDA by Visit (Completer Population) .............................................................. 79 
Table B.14.2.2.1.3 Cumulative Incidence of Patients Achieved CDA by Visit (PP Population) ........................................................................... 79 
Table B.14.2.2.2.1 Cumulative Incidence of Patients Achieved CDA by Visit without Corticosteroids (mITT Population) ................................ 79 
Table B.14.2.2.2.2 Cumulative Incidence of Patients Achieved CDA by Visit without Corticosteroids (Completer Population) ....................... 79 
Table B.14.2.2.2.3 Cumulative Incidence of Patients Achieved CDA by Visit without Corticosteroids (PP Population) .................................... 79 
Table B.14.2.2.3.1 Cumulative Incidence of Patients Achieved CDA by Visit with prednis(ol)one >0.5 mg/kg (mITT Population) .................... 79 
Table B.14.2.2.3.2 Cumulative Incidence of Patients Achieved CDA by Visit with prednis(ol)one >0.5 mg/kg (Completer Population) ........... 79 
Table B.14.2.2.3.3 Cumulative Incidence of Patients Achieved CDA by Visit with prednis(ol)one >0.5 mg/kg (PP Population) ........................ 79 
Table B.14.2.2.4.1 Cumulative Incidence of Patients Achieved CDA by Visit without prednis(ol)one >0.5 mg/kg – Cumulative Incidence 
(mITT Population) ....................................................................................................................................................................... 79 
Table B.14.2.2.4.2 Cumulative Incidence of Patients Achieved CDA by Visit without prednis(ol)one >0.5 mg/kg – Cumulative Incidence 
(Completer 4opulation) .............................................................................................................................................................. 79 
Table B.14.2.2.4.3 Cumulative Incidence of Patients Achieved CDA by Visit without prednis(ol)one >0.5 mg/kg (PP Population) .................. 79 
Table B.14.2.2.5.1 Proportion of patients Achieved CDA by Visit (mITT Population)......................................................................................... 80 
Table B.14.2.2.5.2 Proportion of patients Achieved CDA by Visit (Completer Population)................................................................................ 80 
Table B.14.2.2.5.3 Proportion of patients Achieved CDA by Visit (PP Population) ............................................................................................ 80 
Table B.14.2.2.6.1 Proportion of patients Achieved CDA with prednis(ol)one >0.5 mg/kg by Visit (mITT Population) ..................................... 81 
Table B.14.2.2.6.2 Proportion of patients Achieved CDA with prednis(ol)one >0.5 mg/kg by Visit (Completer Population) ............................ 81 
Table B.14.2.2.6.3 Proportion of patients Achieved CDA with prednis(ol)one >0.5 mg/kg by Visit (PP Population) ......................................... 81 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
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Table B.14.2.2.7.1 Proportion of patients Achieved CDA without prednis(ol)one >0.5 mg/kg by Visit (mITT Population) ............................... 81 
Table B.14.2.2.7.2 Proportion of patients Achieved CDA without prednis(ol)one >0.5 mg/kg by Visit (Completer Population) ...................... 81 
Table B.14.2.2.7.3 Proportion of patients Achieved CDA without prednis(ol)one >0.5 mg/kg by Visit (PP Population) ................................... 81 
Table B.14.2.2.8.1 Cumulative Incidence of Patients Achieved CR by Visit – Cumulative Incidence (mITT Population) .................................... 82 
Table B.14.2.2.8.2 Cumulative Incidence of Patients Achieved CR by Visit – Cumulative Incidence (Completer Population) ........................... 83 
Table B.14.2.2.8.3 Cumulative Incidence of Patients Achieved CR by Visit – Cumulative Incidence (PP Population)........................................ 83 
Table B.14.2.2.9.1 Cumulative Incidence of Patients Achieved CR with prednis(ol)one >0.5 mg/kg by Visit – Cumulative Incidence (mITT 
Population) ................................................................................................................................................................................. 83 
Table B.14.2.2.9.2 Cumulative Incidence of Patients Achieved CR with prednis(ol)one >0.5 mg/kg by Visit – Cumulative Incidence 
(Completer Population) .............................................................................................................................................................. 83 
Table B.14.2.2.9.3 Cumulative Incidence of Patients Achieved CR with prednis(ol)one >0.5 mg/kg by Visit – Cumulative Incidence (PP 
Population) ................................................................................................................................................................................. 83 
Table B.14.2.2.10.1 Cumulative Incidence of Patients Achieved CR without prednis(ol)one >0.5 mg/kg by Visit – Cumulative Incidence 
(mITT Population) ....................................................................................................................................................................... 83 
Table B.14.2.2.10.2 Cumulative Incidence of Patients Achieved CR without prednis(ol)one >0.5 mg/kg by Visit – Cumulative Incidence 
(Completer Population) .............................................................................................................................................................. 83 
Table B.14.2.2.10.3 Cumulative Incidence of Patients Achieved CR without prednis(ol)one >0.5 mg/kg by Visit – Cumulative Incidence (PP 
Population) ................................................................................................................................................................................. 83 
Table B.14.2.2.11.1 Proportion of patients Achieved CR by Visit (mITT Population) ......................................................................................... 84 
Table B.14.2.2.11.2 Proportion of patients Achieved CR by Visit (Completer Population) ................................................................................ 84 
Table B.14.2.2.11.3 Proportion of patients Achieved CR by Visit (PP Population) ............................................................................................. 84 
Table B.14.2.2.12.1 Proportion of patients Achieved CR with prednis(ol)one >0.5 mg/kg by Visit (mITT Population) ..................................... 85 
Table B.14.2.2.12.2 Proportion of patients Achieved CR with prednis(ol)one >0.5 mg/kg by Visit (Completer Population) ............................ 85 
Table B.14.2.2.12.3 Proportion of patients Achieved CR with prednis(ol)one >0.5 mg/kg by Visit (PP Population) ......................................... 85 
Table B.14.2.2.13.1 Proportion of patients Achieved CR without prednis(ol)one >0.5 mg/kg by Visit (mITT Population) ................................ 85 
Table B.14.2.2.13.2 Proportion of patients Achieved CR without prednis(ol)one >0.5 mg/kg by Visit (Completer Population) ....................... 85 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
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Table B.14.2.2.13.3 Proportion of patients Achieved CR without prednis(ol)one >0.5 mg/kg by Visit (PP Population) .................................... 85 
Table B.14.2.2.14.1 Proportion of patients Achieved CR at or Prior to Week 25 with prednis(ol)one >0.5 mg/kg (mITT Population) ............. 85 
Table B.14.2.2.14.2 Proportion of patients Achieved CR at or Prior to Week 25 with prednis(ol)one >0.5 mg/kg (Completer Population) .... 85 
Table B.14.2.2.14.3 Proportion of patients Achieved CR at or Prior to Week 25 with prednis(ol)one >0.5 mg/kg (PP Population) ................. 85 
Table B.14.2.2.15.1 Proportion of patients Achieved CR at or Prior to Week 25 without prednis(ol)one >0.5 mg/kg (mITT Population) ........ 85 
Table B.14.2.2.15.2 Proportion of patients Achieved CR at or Prior to Week 25 without prednis(ol)one >0.5 mg/kg (Completer Population)85 
Table B.14.2.2.15.3 Proportion of patients Achieved CR at or Prior to Week 25 without prednis(ol)one >0.5 mg/kg (PP Population) ............ 85 
Table B.14.2.2.16.1 Duration of Complete Remission (CR) (mITT Population)................................................................................................... 86 
Table B.14.2.2.16.2 Duration of Complete Remission (CR) (Completer Population).......................................................................................... 86 
Table B.14.2.2.16.3 Duration of Complete Remission (CR) (PP Population) ...................................................................................................... 86 
Table B.14.2.2.17.1 Time to First CDA: Kaplan Meier Estimates (mITT Population) .......................................................................................... 87 
Table B.14.2.2.17.2 Time to First CDA: Kaplan Meier Estimates (Completer Population) ................................................................................. 88 
Table B.14.2.2.17.3 Time to First CDA: Kaplan Meier Estimates (PP Population) .............................................................................................. 88 
Table B.14.2.2.18.1 Time to First CR: Kaplan Meier Estimates (mITT Population) ............................................................................................. 88 
Table B.14.2.2.18.2 Time to First CR: Kaplan Meier Estimates (Completer Population) .................................................................................... 88 
Table B.14.2.2.18.3 Time to First CR: Kaplan Meier Estimates (PP Population) ................................................................................................. 88 
Table B.14.2.2.19.1 Time to First ECP: Kaplan Meier Estimates (mITT Population) ........................................................................................... 88 
Table B.14.2.2.19.2 Time to First ECP: Kaplan Meier Estimates (Completer Population) .................................................................................. 88 
Table B.14.2.2.19.3 Time to First ECP: Kaplan Meier Estimates (PP Population) ............................................................................................... 88 
Table B.14.2.2.20.1 Time to First Relapse: Kaplan Meier Estimates (mITT Population) ..................................................................................... 88 
Table B.14.2.2.20.2 Time to First Relapse: Kaplan Meier Estimates (Completer Population) ............................................................................ 88 
Table B.14.2.2.20.3 Time to First Relapse: Kaplan Meier Estimates (PP Population) ......................................................................................... 88 
Table B.14.2.2.21.1 Time to First Increase in PDAI: Kaplan Meier Estimates (mITT Population) ....................................................................... 88 
Table B.14.2.2.21.2 Time to First Increase in PDAI: Kaplan Meier Estimates (Completer Population) .............................................................. 88 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
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Table B.14.2.2.21.3 Time to First Increase in PDAI: Kaplan Meier Estimates (PP Population) ........................................................................... 88 
Table B.14.2.2.22.1 Cumulative and Daily Average Corticosteroid Usage by Treatment Period (mITT Population) ......................................... 89 
Table B.14.2.2.22.2 Cumulative and Daily Average Corticosteroid Usage by Treatment Period – (Completer Population).............................. 89 
Table B.14.2.2.22.3 Cumulative and Daily Average Corticosteroid Usage by Treatment Period – (PP Population) .......................................... 89 
Table B.14.2.2.23.1 Corticosteroid Dose (mg/day) on the Day or at the Day Prior to Visit by Study Visit – (mITT Population) ........................ 90 
Table B.14.2.2.22.2 Corticosteroid Dose (mg/day) on the Day or at the Day Prior by Study Visit – (Completer Population) ........................... 91 
Table B.14.2.2.22.3 Corticosteroid Dose (mg/day) on the Day or at the Day Prior by Study Visit – (PP Population) ........................................ 91 
Table B.14.2.2.24.1 Corticosteroid Dose (mg/kg/day) on the Day or at the Day Prior by Study Visit – (mITT Population) ............................... 91 
Table B.14.2.2.24.2 Corticosteroid Dose (mg/kg/day) on the Day or at the Day Prior by Study Visit – (Completer Population) ...................... 91 
Table B.14.2.2.24.3 Corticosteroid Dose (mg/kg/day) on the Day or at the Day Prior by Study Visit – (PP Population) ................................... 91 
Table B.14.2.2.25.1 Corticosteroid Dose (mg/day) by Study Visit in Patients who achieved at least one CDA – (mITT Population) ................. 92 
Table B.14.2.2.25.2 Corticosteroid Dose (mg/day) by Study Visit in Patients who achieved at least one CDA – (Completer Population) ........ 92 
Table B.14.2.2.26.1 Corticosteroid Dose (mg/kg/day) by Study Visit in Patients who achieved at least one CDA – (mITT Population) ........... 93 
Table B.14.2.2.26.2 Corticosteroid Dose (mg/kg/day) by Study Visit in Patients who achieved at least one CDA – (Completer Population) .. 93 
Table B.14.2.2.27.1 Corticosteroid Dose (mg/day) by Study Visit in Patients who achieved at least one CR – (mITT Population) ................... 93 
Table B.14.2.2.27.2 Corticosteroid Dose (mg/day) by Study Visit in Patients who achieved at least one CR – (Completer Population) .......... 93 
Table B.14.2.2.27.3 Corticosteroid Dose (mg/day) by Study Visit in Patients who achieved at least one CR – (PP Population) ....................... 93 
Table B.14.2.2.28.1 Corticosteroid Dose (mg/kg/day) by Study Visit in Patients who achieved at least one CR – (mITT Population) .............. 93 
Table B.14.2.2.28.2 Corticosteroid Dose (mg/kg/day) by Study Visit in Patients who achieved at least one CR – (Completer Population) ..... 93 
Table B.14.2.2.29.1 PDAI – Total Activity Score – Actual Values, Change and Percent Change from Screening and from Baseline (mITT 
Population) ................................................................................................................................................................................. 94 
Table B.14.2.2.29.2 PDAI – Total Activity Score – Actual Values, Change and Percent Change from Screening and from Baseline 
(Completer Population) .............................................................................................................................................................. 95 
Table B.14.2.2.29.3 PDAI – Total Activity Score – Actual Values, Change and Percent Change from Screening and from Baseline (PP 
Population) ................................................................................................................................................................................. 95 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
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Table B.14.2.2.30.1 PDAI – Total Damage Score – Actual Values and  Change from Baseline (mITT Population) ............................................. 95 
Table B.14.2.2.30.2 PDAI – Total Damage Score – Actual Values and  Change from Baseline (Completer Population) .................................... 95 
Table B.14.2.2.30.3 PDAI – Total Damage Score – Actual Values and  Change from Baseline (PP Population) ................................................. 95 
Table B.14.2.2.31.1 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline (mITT Population) .............................. 96 
Table B.14.2.2.31.2 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline (Completer Population) ..................... 97 
Table B.14.2.2.29.3 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline (PP Population) .................................. 97 
Table B.14.2.2.32.1 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline (mITT Population) ............................... 97 
Table B.14.2.2.32.2 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline (Completer Population) ...................... 97 
Table B.14.2.2.32.3 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline (PP Population) ................................... 97 
Table B.14.2.2.33.1 ABSIS – Severity Total Score –Actual Values and Change from Baseline (mITT Population) .............................................. 97 
Table B.14.2.2.33.2 ABSIS – Severity Total Score –Actual Values and Change from Baseline (Completer Population) ..................................... 97 
Table B.14.2.2.33.3 ABSIS – Severity Total Score –Actual Values and Change from Baseline (PP Population) .................................................. 97 
Table B.14.2.2.34.1 ABSIS – Total Activity Score –Actual Values and Change from Baseline (mITT Population) ............................................... 97 
Table B.14.2.2.34.2 ABSIS – Total Activity Score –Actual Values and Change from Baseline (Completer Population) ...................................... 97 
Table B.14.2.2.34.3 ABSIS – Total Activity Score –Actual Values and Change from Baseline (PP Population) ................................................... 97 
Table B.14.2.2.35.1 ABSIS – Total Score –Actual Values and Change from Baseline (mITT Population) ............................................................ 97 
Table B.14.2.2.35.2 ABSIS – Total Score –Actual Values and Change from Baseline (Completer Population) ................................................... 97 
Table B.14.2.2.35.3 ABSIS – Total Score –Actual Values and Change from Baseline (PP Population) ................................................................ 97 
Table B.14.2.2.36.1 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline – by Baseline Oral Involvement (mITT 
Population) ................................................................................................................................................................................. 98 
Table B.14.2.2.36.2 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline – by Baseline Oral Involvement 
(Completer Population) .............................................................................................................................................................. 98 
Table B.14.2.2.36.3 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline – by Baseline Oral Involvement (PP 
Population) ................................................................................................................................................................................. 98 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
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Table B.14.2.2.37.1 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (mITT 
Population) ................................................................................................................................................................................. 98 
Table B.14.2.2.37.2 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline – by Baseline Oral Involvement 
(Completer Population) .............................................................................................................................................................. 98 
Table B.14.2.2.37.3 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (PP 
Population) ................................................................................................................................................................................. 98 
Table B.14.2.2.38.1 ABSIS – Severity Total Score –Actual Values and Change from Baseline– by Baseline Oral Involvement (mITT 
Population) ................................................................................................................................................................................. 98 
Table B.14.2.2.38.2 ABSIS – Severity Total Score –Actual Values and Change from Baseline– by Baseline Oral Involvement (Completer 
Population) ................................................................................................................................................................................. 98 
Table B.14.2.2.38.3 ABSIS – Severity Total Score –Actual Values and Change from Baseline– by Baseline Oral Involvement (PP Population) 98 
Table B.14.2.2.39.1 ABSIS – Total Activity Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (mITT 
Population) ................................................................................................................................................................................. 98 
Table B.14.2.2.39.2 ABSIS – Total Activity Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (Completer 
Population) ................................................................................................................................................................................. 98 
Table B.14.2.2.39.3 ABSIS – Total Activity Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (PP Population) 98 
Table B.14.2.2.40.1 ABSIS – Total Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (mITT Population) ......... 98 
Table B.14.2.2.40.2 ABSIS – Total Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (Completer Population) 98 
Table B.14.2.2.40.3 ABSIS – Total Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (PP Population) ............. 98 
Table B.14.2.2.41.1 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline – by Baseline Skin Involvement (mITT 
Population) ................................................................................................................................................................................. 98 
Table B.14.2.2.41.2 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline – by Baseline Skin Involvement 
(Completer Population) .............................................................................................................................................................. 98 
Table B.14.2.2.41.3 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline – by Baseline Skin Involvement (PP 
Population) ................................................................................................................................................................................. 98 
Table B.14.2.2.42.1 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (mITT 
Population) ................................................................................................................................................................................. 99 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
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Table B.14.2.2.42.2 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline – by Baseline Skin Involvement 
(Completer Population) .............................................................................................................................................................. 99 
Table B.14.2.2.42.3 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (PP 
Population) ................................................................................................................................................................................. 99 
Table B.14.2.2.43.1 ABSIS – Severity Total Score –Actual Values and Change from Baseline– by Baseline Skin Involvement (mITT 
Population) ................................................................................................................................................................................. 99 
Table B.14.2.2.43.2 ABSIS – Severity Total Score –Actual Values and Change from Baseline– by Baseline Skin Involvement (Completer 
Population) ................................................................................................................................................................................. 99 
Table B.14.2.2.43.3 ABSIS – Severity Total Score –Actual Values and Change from Baseline– by Baseline Skin Involvement (PP Population) 99 
Table B.14.2.2.44.1 ABSIS – Total Activity Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (mITT 
Population) ................................................................................................................................................................................. 99 
Table B.14.2.2.44.2 ABSIS – Total Activity Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (Completer 
Population) ................................................................................................................................................................................. 99 
Table B.14.2.2.44.3 ABSIS – Total Activity Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (PP Population) 99 
Table B.14.2.2.45.1 ABSIS – Total Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (mITT Population) ......... 99 
Table B.14.2.2.45.2 ABSIS – Total Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (Completer Population) 99 
Table B.14.2.2.45.3 ABSIS – Total Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (PP Population) ............. 99 
Table B.14.2.2.46.1 ABQOL – Actual Values and Change from Baseline (mITT Population) .............................................................................. 99 
Table B.14.2.2.46.2 ABQOL – Actual Values and Change from Baseline (Completer Population) ..................................................................... 99 
Table B.14.2.2.46.3 ABQOL – Actual Values and Change from Baseline (PP Population) .................................................................................. 99 
Table B.14.2.2.47.1 TABQOL – Actual Values and Change from Baseline (mITT Population) ............................................................................ 99 
Table B.14.2.2.47.2 TABQOL – Actual Values and Change from Baseline (Completer Population) ................................................................... 99 
Table B.14.2.2.47.3 TABQOL – Actual Values and Change from Baseline (PP Population) ................................................................................ 99 
Table B.14.2.2.48.1 SNAQ – Actual Values and Change from Baseline (mITT Population) ................................................................................ 99 
Table B.14.2.2.48.2 SNAQ – Actual Values and Change from Baseline (Completer Population) ....................................................................... 99 
Table B.14.2.2.48.3 SNAQ – Actual Values and Change from Baseline (PP Population) .................................................................................... 99 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
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Table B.14.2.2.49.1 Total Anti-DSG – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (mITT 
Population) ............................................................................................................................................................................... 100 
Table B.14.2.2.49.2 Total Anti-DSG – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value 
(Completer Population) ............................................................................................................................................................ 101 
Table B.14.2.2.49.3 Total Anti-DSG – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (PP 
Population) ............................................................................................................................................................................... 101 
Table B.14.2.2.50.1 Anti-DSG 1 – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (mITT 
Population) ............................................................................................................................................................................... 101 
Table B.14.2.2.50.2 Anti-DSG 1  – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (Completer 
Population) ............................................................................................................................................................................... 101 
Table B.14.2.2.50.3 Anti-DSG 1 – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (PP 
Population) ............................................................................................................................................................................... 101 
Table B.14.2.2.51.1 Anti-DSG 3 – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (mITT 
Population) ............................................................................................................................................................................... 101 
Table B.14.2.2.51.2 Anti-DSG 3  – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (Completer 
Population) ............................................................................................................................................................................... 101 
Table B.14.2.2.51.3 Anti-DSG 3 – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (PP 
Population) ............................................................................................................................................................................... 101 
Table B.14.3.1.1 
Summary of Adverse Events (Safety Population) ............................................................................................................... 103 
Table B.14.3.1.2 
Treatment Emergent Adverse Events by SOC and PT (Safety Population) ........................................................................ 105 
Table B.14.3.1.3 
Related Treatment Emergent Adverse Events by SOC and PT (Safety Population) ........................................................... 106 
Table B.14.3.1.4 
Treatment Emergent Adverse Events by Maximum Intensity, SOC and PT (Safety Population) ....................................... 107 
Table B.14.3.1.5 
Related Treatment Emergent Adverse Events by Maximum Intensity, SOC and PT (Safety Population) .......................... 108 
Table B.14.3.1.6 
Serious Treatment Emergent Adverse Events by SOC and PT (Safety Population) ............................................................ 109 
Table B.14.3.1.7 
Treatment Emergent Adverse Events Leading to Study Drug Discontinuation by SOC and PT (Safety Population) ......... 110 
Table B.14.3.2.1 
Hematology – Actual Values and Change from Baseline by Study Visit (Safety Population) ............................................. 111 
Table B.14.3.2.2 
Hematology – Shift from Baseline by Study Visit (Safety Population) ............................................................................... 113 


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Table B.14.3.3.1 
Coagulation – Actual Values and Change from Baseline by Study Visit (Safety Population) ............................................. 114 
Table B.14.3.3.2 
Coagulation – Shift from Baseline by Study Visit (Safety Population) ............................................................................... 114 
Table B.14.3.4.1 
Serum Chemistry – Actual Values and Change from Baseline by Study Visit (Safety Population) ..................................... 114 
Table B.14.3.4.2 
Serum Chemistry – Shift from Baseline by Study Visit (Safety Population) ....................................................................... 114 
Table B.14.3.4.3 
Serum Chemistry – Drug Induced Liver Injury (DILI) Criteria (Safety Population) ............................................................. 115 
Table B.14.3.5.1 
Urinalysis – Actual Values and Change from Baseline by Study Visit (Safety Population) ................................................. 116 
Table B.14.3.5.2 
Urinalysis – Shift from Baseline by Study Visit (Safety Population) ................................................................................... 116 
Table B.14.3.6.1 
Vital Signs and Body Weight – Actual Values and Change from Baseline by Study Visit (Safety Population) ................... 117 
Table B.14.3.6.2 
Vital Signs – BP and Pulse – Shift from Baseline by Study Visit (Safety Population) .......................................................... 118 
Table B.14.3.7.1 
12-Lead ECG Results – Actual Values and Change from Baseline (Safety Population) ...................................................... 120 
Table B.14.3.8.3 
12-Lead ECG – Overall Assessment – Shift from Baseline to Last Post-Baseline Visit (Safety Population) ........................ 121 
Table B.14.3.9.1 
Abbreviated Physical Examination by Study Visit (Safety Population) .............................................................................. 122 
Listing B.16.2.1.1  
Patient Disposition (Screening Population) ................................................................................................................... 123 
Listing B.16.2.1.2  
Patient Enrollment (Screening Population) ................................................................................................................... 124 
Listing B.16.2.1.3  
Screen Failure (All Screen Failures) ................................................................................................................................ 125 
Listing B.16.2.1.4  
Analysis Population (Screening Population) .................................................................................................................. 126 
Listing B.16.2.1.5  
Demographics and Baseline Characteristics (ITT Population) ....................................................................................... 127 
Listing B.16.2.1.6  
Disease History – Pemphigus history and Anti-DSG at Screening (ITT Population) ....................................................... 128 
Listing B.16.2.1.7  
Disease History – Baseline PDAI and ABSIS Score (ITT Population) ............................................................................... 129 
Listing B.16.2.1.8  
Medical History (ITT Population) ................................................................................................................................... 130 
Listing B.16.2.1.9  
Prior Corticosteroid Medications (ITT Population) ........................................................................................................ 131 
Listing B.16.2.1.10  
Concomitant Corticosteroid Medications (ITT Population) ........................................................................................... 132 
Listing B.16.2.1.11  
Concomitant Corticosteroid Medications Started Post Last Dose (ITT Population) ...................................................... 133 
Listing B.16.2.1.11  
Rescue Medications Taken During Study (ITT Population) ............................................................................................ 134 


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Listing B.16.2.1.12  
Prior Medications other than Corticosteroid Medications (ITT Population) ................................................................. 135 
Listing B.16.2.1.13  
Concomitant Medications other than Corticosteroid Medications (ITT Population) .................................................... 136 
Listing B.16.2.1.14  
Protocol Deviations (ITT Population) ............................................................................................................................. 137 
Listing B.16.2.5.1  
Study Drug Administration (ITT Population) .................................................................................................................. 138 
Listing B.16.2.5.2  
Study Drug Exposure and Compliance (ITT Population) ................................................................................................ 139 
Listing B.16.2.6.1  
Disease Assessment (ITT Population) ............................................................................................................................ 140 
Listing B.16.2.6.2  
Pemphigus Disease Area Index (PDAI) - Skin (ITT Population)....................................................................................... 141 
Listing B.16.2.6.3  
Pemphigus Disease Area Index (PDAI) - Scalp (ITT Population) ..................................................................................... 142 
Listing B.16.2.6.4  
Pemphigus Disease Area Index (PDAI) – Mucous Membrane (ITT Population) ............................................................. 143 
Listing B.16.2.6.5  
Pemphigus Disease Area Index (PDAI) – Total Scores (ITT Population) ......................................................................... 144 
Listing B.16.2.6.6  
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) - Skin and Oral Involvement (ITT Population) .................. 145 
Listing B.16.2.6.7  
Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL) (ITT Population) ............................................... 146 
Listing B.16.2.6.8  
Simple Nutritional Appetite Questionnaire (SNAQ) (ITT Population) ............................................................................ 147 
Listing B.16.2.6.9  
Corticosteroid (CS) Usage (ITT Population) ................................................................................................................... 148 
Listing B.16.2.6.10  
Corticosteroid (CS) Usage on the Day prior to each Visit and CDA/CR Response (ITT Population) ............................... 149 
Listing B.16.2.7.1  
Pre-treatment Adverse Events (ITT Population) ............................................................................................................ 150 
Listing B.16.2.7.2  
Pre-treatment Serious Adverse Events (ITT Population) ............................................................................................... 151 
Listing B.16.2.7.3  
Treatment Emergent Adverse Events (Safety Population) ............................................................................................ 152 
Listing B.16.2.7.4  
Related Treatment Emergent Adverse Events (Safety Population) ............................................................................... 153 
Listing B.16.2.7.5  
Serious Adverse Events (Safety Population) .................................................................................................................. 153 
Listing B.16.2.7.6  
Treatment Emergent Adverse Events Leading to Study Drug Discontinuation (Safety Population) ............................. 153 
Listing B.16.2.7.7  
Serious Treatment Emergent Adverse Events Leading to Study Drug Discontinuation (Safety Population)................. 154 
Listing B.16.2.7.8  
Treatment Emergent Adverse Events Leading to Death (Safety Population) ................................................................ 156 
Listing B.16.2.7.9 
    Treatment Emergent Adverse Events Started Post Last Dose (Safety Population).................................................... 156 


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Listing B.16.2.7.10 
Disease under Study Events (Safety Population) ........................................................................................................... 156 
Listing B.16.2.8.1  
Hematology Results (Safety Population) ....................................................................................................................... 157 
Listing B.16.2.8.2  
Hematology Results – Patients with Abnormal Results (Safety Population) ................................................................. 158 
Listing B.16.2.8.3  
Coagulation Results (Safety Population)........................................................................................................................ 159 
Listing B.16.2.8.4  
Coagulation Results – Patients with Abnormal Results (Safety Population) ................................................................. 159 
Listing B.16.2.8.5  
Serum Chemistry Results (Safety Population) ............................................................................................................... 159 
Listing B.16.2.8.6  
Serum Chemistry Results – Patients with Abnormal Results (Safety Population) ......................................................... 159 
Listing B.16.2.8.7  
Urinalysis Results (Safety Population) ........................................................................................................................... 159 
Listing B.16.2.8.8  
Urinalysis Results – Patients with Abnormal Results (Safety Population) ..................................................................... 159 
Listing B.16.2.8.9  
Drug Induced Liver Injury (DILI) Test Results (Safety Population) ................................................................................. 159 
Listing B.16.2.8.10  
Drug Induced Liver Injury (DILI) Test Results – Patients with Abnormal Results (Safety Population) ........................... 159 
Listing B.16.2.8.11 
Anti-DSG Antibodies Results (ITT Population) ............................................................................................................... 160 
Listing B.16.2.8.12  
PK and BTK Sample Collection (ITT Population)............................................................................................................. 161 
Listing B.16.2.8.13  
Vital Signs and Body Weight (Safety Population) .......................................................................................................... 162 
Listing B.16.2.8.14  
Vital Signs – Blood Pressure and Pulse – Patient with Abnormal Results (Safety Population) ...................................... 163 
Listing B.16.2.8.15  
12-Lead ECG Results (Safety Population) ...................................................................................................................... 164 
Listing B.16.2.8.16  
12-Lead ECG Results – Patient with Abnormal Results (Safety Population) .................................................................. 165 
Listing B.16.2.8.17  
Physical Examination (Safety Population) ..................................................................................................................... 166 
Listing B.16.2.8.18 
Physical Examination – Patients with Abnormal Results (Safety Population) ............................................................... 167 
Listing B.16.2.8.19  
Pregnancy Test Results (Safety Population) .................................................................................................................. 168 
Listing B.16.2.8.20  
Food Intake (Safety Population) .................................................................................................................................... 169 
Figure B.14.1.1.1 PDAI Score and PRN Dose Level By Study Day – Panel Plots (mITT Population) .................................................................. 170 
Figure B.14.3.2.1 Scatter Plots of Peak TBL vs Peak ALT for Identification of Drug Induced Liver Injury (DILI) (Safety Population) ................ 171 
 
 


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Table B.14.1.1.1 Patient Disposition – Completion Status (Screening Population) 
 
Number of patients (%) 
 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
Screening population – Enrolled  
n 
n 
n 
ITT population – Had at least one dose of PRN1008 [1] 
x (x.x) 
x (x.x) 
x (x.x) 
Safety population – Had at least one dose of PRN1008 [1] 
x (x.x) 
x (x.x) 
x (x.x) 
mITT population – Treated up to Week 5 and had a Week 5 CDA assessment [1] 
x (x.x) 
x (x.x) 
x (x.x) 
Completer population – Treated up to Week 25 andhad a Week 29 CDA 
assessment [1] 
x (x.x) 
x (x.x) 
x (x.x) 
PP population – Had no major protocol deviations, completed Week 13/Day 85 
visit and had at least 80% study drug compliance [1] 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Completion status [2] 
 
 
 
Completed 
x (x.x) 
x (x.x) 
x (x.x) 
Early discontinuation 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Primary reason(s) for early discontinuation [3] 
 
 
 
Adverse Event 
x (x.x) 
x (x.x) 
x (x.x) 
Death 
x (x.x) 
x (x.x) 
x (x.x) 
Lack of Efficacy 
x (x.x) 
x (x.x) 
x (x.x) 
Lost to Follow-up 
x (x.x) 
x (x.x) 
x (x.x) 
Non-Compliance with Study Drug 
x (x.x) 
x (x.x) 
x (x.x) 
Physician Decision 
x (x.x) 
x (x.x) 
x (x.x) 
Pregnancy 
x (x.x) 
x (x.x) 
x (x.x) 
Progressive Disease 
x (x.x) 
x (x.x) 
x (x.x) 


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Table B.14.1.1.1 Patient Disposition – Completion Status (Screening Population) 
 
Number of patients (%) 
 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
Recovery 
x (x.x) 
x (x.x) 
x (x.x) 
Trial Screen Failure 
x (x.x) 
x (x.x) 
x (x.x) 
Study Terminated by Sponsor 
x (x.x) 
x (x.x) 
x (x.x) 
Technical Problem 
x (x.x) 
x (x.x) 
x (x.x) 
Withdrawal by Subject 
x (x.x) 
x (x.x) 
x (x.x) 
Other 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
[1] Percentages are based on the number of patients enrolled into the study. 
[2] Percentages are based on the number of patients who received at least one dose of PRN1008. 
[3] Percentages are based on the number of patients who withdrew early. 
Program: <program name (date, time)>, Path: <path> 
 
 


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Table B.14.1.1.2 Patient Disposition by Study Visit (ITT Population) 
 
Number of patients (%) 
Attended 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
SCREENED 
x (x.x) 
x (x.x) 
x (x.x) 
WEEK 1 DAY 1 
x (x.x) 
x (x.x) 
x (x.x) 
WEEK 1 DAY 2 
x (x.x) 
x (x.x) 
x (x.x) 
WEEK 3 DAY 15 
x (x.x) 
x (x.x) 
x (x.x) 
WEEK 5 DAY 29 
x (x.x) 
x (x.x) 
x (x.x) 
WEEK 9 DAY 25 
x (x.x) 
x (x.x) 
x (x.x) 
WEEK 13 DAY 85 
x (x.x) 
x (x.x) 
x (x.x) 
WEEK 17 DAY 113 
x (x.x) 
x (x.x) 
x (x.x) 
WEEK 21 DAY 141 
x (x.x) 
x (x.x) 
x (x.x) 
WEEK 25 DAY 169 
x (x.x) 
x (x.x) 
x (x.x) 
WEEK 29 DAY 197 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Percentages are based on the number of patients in each starting dose and overall.  
Program: <program name (date, time)>, Path: <path> 
 


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Table B.14.1.1.3 Patient Disposition by Country and Study Site (ITT Population) 
 
Number of patients (%) 
 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
Country xx 
x (x.x) 
x (x.x) 
x (x.x) 
Site xx 
x (x.x) 
x (x.x) 
x (x.x) 
Site xx 
x (x.x) 
x (x.x) 
x (x.x) 
…etc… 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Country xx 
x (x.x) 
x (x.x) 
x (x.x) 
Site xx 
x (x.x) 
x (x.x) 
x (x.x) 
Site xx 
x (x.x) 
x (x.x) 
x (x.x) 
…etc… 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Country xx 
x (x.x) 
x (x.x) 
x (x.x) 
Site xx 
x (x.x) 
x (x.x) 
x (x.x) 
Site xx 
x (x.x) 
x (x.x) 
x (x.x) 
…etc… 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Country xx 
x (x.x) 
x (x.x) 
x (x.x) 
Site xx 
x (x.x) 
x (x.x) 
x (x.x) 
Site xx 
x (x.x) 
x (x.x) 
x (x.x) 
…etc… 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Percentages are based on the number of patients in each starting dose and overall.  
Program: <program name (date, time)>, Path: <path> 
 
 


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Table B.14.1.1.4 Analysis Populations (ITT Population) 
 
Number of patients (%) 
 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
ITT/Safety Population – PRN Dose Level 
x  
x 
x 
 
400 mg QD 
x (x.x) 
x (x.x) 
x (x.x) 
 
400 mg QD 
x (x.x) 
x (x.x) 
x (x.x) 
 
600 mg BID 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
mITT Population – PRN Dose Level 
x (x.x) 
x (x.x) 
x (x.x) 
 
400 mg QD 
x (x.x) 
x (x.x) 
x (x.x) 
 
400 mg QD 
x (x.x) 
x (x.x) 
x (x.x) 
 
600 mg BID 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Completer Population – PRN Dose Level 
x (x.x) 
x (x.x) 
x (x.x) 
 
400 mg QD 
x (x.x) 
x (x.x) 
x (x.x) 
 
400 mg BID 
x (x.x) 
x (x.x) 
x (x.x) 
 
600 mg BID 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
PP Population – PRN Dose Level 
x (x.x) 
x (x.x) 
x (x.x) 
 
400 mg QD 
x (x.x) 
x (x.x) 
x (x.x) 
 
400 mg BID 
x (x.x) 
x (x.x) 
x (x.x) 
 
600 mg BID 
x (x.x) 
x (x.x) 
x (x.x) 
 ITT/Safety population – Had at least one dose of PRN1008  
mITT population – Treated up to Week 5 and had a Week 5 CDA assessment 
Completer population – Treated up to Week 25 andhad a Week 29 CDA assessment  
PP population – Had no major protocol deviations, completed Week 13/Day 85 visit and had at least 80% study drug compliance 
Percentages are based on the number of patients in each starting dose and overall. 


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Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Table B.14.1.2.1 Demographics and Baseline Characteristics (ITT Population) 
 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
Age (years) 
 
 
 
     n 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
Gender n (%) 
 
 
 
     Male 
x (x.x) 
x (x.x) 
x (x.x) 
     Female 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
If Female – Fertility Status n (%) 
 
 
 
     Sterile 
x (x.x) 
x (x.x) 
x (x.x) 
     Post-Menopausal 
x (x.x) 
x (x.x) 
x (x.x) 
     Potentially Able to Bear Children 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
If Female – Method of birth control n (%) 
 
 
 
     Effective Methods of Contraception 
x (x.x) 
x (x.x) 
x (x.x) 
     Post-menopausal 
x (x.x) 
x (x.x) 
x (x.x) 
     Surgically sterilized 
x (x.x) 
x (x.x) 
x (x.x) 
     Complete abstinence 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Race n (%) 
 
 
 
     American Indian or Alaska Native  
x (x.x) 
x (x.x) 
x (x.x) 


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Table B.14.1.2.1 Demographics and Baseline Characteristics (ITT Population) 
 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
     Asian 
x (x.x) 
x (x.x) 
x (x.x) 
     Black or African American 
x (x.x) 
x (x.x) 
x (x.x) 
     Native Hawaiian or Other Pacific Islander 
x (x.x) 
x (x.x) 
x (x.x) 
     White 
x (x.x) 
x (x.x) 
x (x.x) 
     Other 
x (x.x) 
x (x.x) 
x (x.x) 
     Not Reported 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Ethnicity n (%) 
 
 
 
     Hispanic or Latino  
x (x.x) 
x (x.x) 
x (x.x) 
     Not Hispanic or Latino 
x (x.x) 
x (x.x) 
x (x.x) 
     Not Reported 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Height (cm) at Screening 
 
 
 
     n 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
Weight (kg) at Screening 
 
 
 
     n 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Table B.14.1.2.1 Demographics and Baseline Characteristics (ITT Population) 
 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
Body Mass Index (BMI) (kg/m2) 
 
 
 
     n 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
Percentages are based on the number of patients in each starting dose and overall. 
Calculated Body Mass Index (BMI) = (Weight (kg) at Screening)/(Height (m) at Screening)2 
Program: <program name (date, time)>, Path: <path> 
 
 


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Table B.14.1.3.1 Disease History (ITT Population) 
 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
Pemphigus subtype at Week 1 Day 1 n (%) 
 
 
 
     Pemphigus Vulgaris 
x (x.x) 
x (x.x) 
x (x.x) 
     Bullous Pemphigoid 
x (x.x) 
x (x.x) 
x (x.x) 
     Pemphigus Follaceus 
x (x.x) 
x (x.x) 
x (x.x) 
     Epidermolysis Bullosa Aquisita 
x (x.x) 
x (x.x) 
x (x.x) 
     Linear IgA Bullous Dermatoses 
x (x.x) 
x (x.x) 
x (x.x) 
     Mucous Membrane Pemphigoid 
x (x.x) 
x (x.x) 
x (x.x) 
     Other 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Time from Pemphigus Diagnosis [1] (years) 
 
 
 
     n 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
Pemphigus history type [2] n (%) 
 
 
 
     Newly Diagnosed 
x (x.x) 
x (x.x) 
x (x.x) 
     Relapsed 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Anti-DSG status [3] n (%) 
 
 
 
     Anti-DSG 1 positive, Anti-DSG 3 negative 
x (x.x) 
x (x.x) 
x (x.x) 
     Anti-DSG 1 negative, Anti-DSG 3 positive 
x (x.x) 
x (x.x) 
x (x.x) 
     Anti-DSG 1 positive, Anti-DSG 3 positive 
x (x.x) 
x (x.x) 
x (x.x) 


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Table B.14.1.3.1 Disease History (ITT Population) 
 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
     Anti-DSG 1 negative, Anti-DSG 3 negative 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Total Anti-DSG [3][4] 
 
 
 
     < 100 unit 
x (x.x) 
x (x.x) 
x (x.x) 
     ≥ 100 unit 
 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
PDAI total activity score [3] 
 
 
 
     n 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
Pemphigus Severity [3]  
 
 
 
     PDAI < 15 (mild-moderate)  
x (x.x) 
x (x.x) 
x (x.x) 
     PDAI ≥ 15 (moderate-severe)  
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
PDAI Skin Activity > 0 n (%) [3] 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
PDAI Scalp Activity > 0 n (%)[3] 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
PDAI Mucous Membrane Activity > 0 n (%)[3] 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
ABSIS Oral Involvement > 0 n (%) [3] 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 


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[1] Based on date of first pemphigus reported in Medical History and date of screening 
[2] Newly diagnosed is defined as subjects who were diagnosed at most 180 days prior to date of screening, otherwise the subject is relapsed. 
[3] Result at Week 1 Day 1. If Week 1 Day 1 is missing, then the Screening result is used. 
[4] Total Anti-DSG is the sum  of Anti-DSG 1 and Anti-DSG3, where at least one of Anti-DSG 1 or 3 is positive, i.e. Anti-DSG 1 ≥ 14 and/or Anti-DSG 3 ≥ 9. 
  
Percentages are based on the number of patients in each starting dose and overall. 
Program: <program name (date, time)>, Path: <path> 
 
 
 


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Table B.14.1.4.1 Medical History (ITT Population) 
 
Number of patients (%) 
Body System Code 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
Number of patients reporting any medical history 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Skin 
x (x.x) 
x (x.x) 
x (x.x) 
Head, Eyes, Ears, Nose, Throat 
x (x.x) 
x (x.x) 
x (x.x) 
Respiratory 
x (x.x) 
x (x.x) 
x (x.x) 
Cardiovascular 
x (x.x) 
x (x.x) 
x (x.x) 
Gastrointestinal 
x (x.x) 
x (x.x) 
x (x.x) 
Endocrine/Metabolic 
x (x.x) 
x (x.x) 
x (x.x) 
Genitourinary 
x (x.x) 
x (x.x) 
x (x.x) 
Neurological 
x (x.x) 
x (x.x) 
x (x.x) 
Blood/Lymphatic 
x (x.x) 
x (x.x) 
x (x.x) 
Musculoskeletal 
x (x.x) 
x (x.x) 
x (x.x) 
Hepatic 
x (x.x) 
x (x.x) 
x (x.x) 
Allergies 
x (x.x) 
x (x.x) 
x (x.x) 
Psychological/Psychiatric 
x (x.x) 
x (x.x) 
x (x.x) 
Other 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Percentages are based on the number of patients in each starting dose and overall. 
If a medical history event of the same Body System Code is reported multiple times for a patient, it is counted only once. 
Program: <program name (date, time)>, Path: <path> 
 
 


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Table B.14.1.5.1 Major Protocol Deviations (ITT Population) 
 
Number of patients (%) 
Protocol Deviation Categories 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
Number of patients reporting major protocol deviations 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Category 1 
x (x.x) 
x (x.x) 
x (x.x) 
Category 2 
x (x.x) 
x (x.x) 
x (x.x) 
Category 3 
x (x.x) 
x (x.x) 
x (x.x) 
Category 4 
x (x.x) 
x (x.x) 
x (x.x) 
Category 5 
x (x.x) 
x (x.x) 
x (x.x) 
Category 6 
x (x.x) 
x (x.x) 
x (x.x) 
…etc… 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Percentages are based on the number of patients in each starting dose and overall. 
If the same protocol deviation category is reported multiple times for a patient, it is counted only once.  
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Table B.14.1.6.1 Prior Corticosteroid Medications (ITT Population) 
 
Number of patients (%) 
ATC Level 2 Class 
    Preferred Name 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
Number of patients reporting prior corticosteroid medications 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
ATC Level 2 Class 1 
x (x.x) 
x (x.x) 
x (x.x) 
Preferred Name 1.1 
x (x.x) 
x (x.x) 
x (x.x) 
         Preferred Name 1.2 
x (x.x) 
x (x.x) 
x (x.x) 
        Preferred Name 1.3 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
ATC Level 2 Class 2 
x (x.x) 
x (x.x) 
x (x.x) 
Preferred Name 2.1 
x (x.x) 
x (x.x) 
x (x.x) 
Preferred Name 2.2 
x (x.x) 
x (x.x) 
x (x.x) 
         Preferred Name 2.3 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
…etc… 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
 
 
Percentages are based on the number of patients in each starting dose and overall. 
Medications are coded using WHODD, version 01SEP2015. 
ATC Level 2 Class is sorted in descending order in the Overall group. Within each ATC Level 2 Class, Preferred Name is sorted in descending order in the Overall group. 
If a medication of the same ATC Level 2 Class and Preferred Name is reported multiple times for a patient, it is counted only once.  
Prior corticosteroid medications are those corticosteroid mediations that have stopped prior to the first dose of study drug.  
Program: <program name (date, time)>, Path: <path> 
 
 


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Table B.14.1.6.2 Concomitant Corticosteroid Medications (ITT Population) 
 
Number of patients (%) 
ATC Level 2 Class 
    Preferred Name 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
Number of patients reporting concomitant corticosteroid 
medications  
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
ATC Level 2 Class 1 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
Preferred Name 1.1 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
         Preferred Name 1.2 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
        Preferred Name 1.3 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
ATC Level 2 Class 2 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
Preferred Name 2.1 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
Preferred Name 2.2 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
         Preferred Name 2.3 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
…etc… 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received during the time the medication was taken. 
Percentages are based on the number of patients in each dose group and overall. 
Medications are coded using WHODD, version 01SEP2015. 
ATC Level 2 Class is sorted in descending order in the Overall group. Within each ATC Level 2 Class, Preferred Name is sorted in descending order in the Overall group. 
If a medication of the same ATC Level 2 Class and Preferred Name is reported multiple times for a patient, it is counted only once.  
This table includes concomitant corticosteroid medications that are ongoing or stopped on or after the date of first dose of study drug and started prior to date of last 
dose of study drug. 
Program: <program name (date, time)>, Path: <path> 
 
 


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Table B.14.1.6.3 Concomitant Corticosteroid Medications Started Post Last Dose (ITT Population) 
 
Number of patients (%) 
ATC Level 2 Class 
    Preferred Name 
Overall 
(N = n) 
Number of patients reported post last dose corticosteroid 
medications  
x (x.x) 
 
 
ATC Level 2 Class 1 
x (x.x) 
Preferred Name 1.1 
x (x.x) 
         Preferred Name 1.2 
x (x.x) 
        Preferred Name 1.3 
x (x.x) 
 
 
ATC Level 2 Class 2 
x (x.x) 
Preferred Name 2.1 
x (x.x) 
Preferred Name 2.2 
x (x.x) 
         Preferred Name 2.3 
x (x.x) 
 
 
…etc… 
x (x.x) 
 
 
 
 
Percentages are based on the number of patients in the ITT population. 
Medications are coded using WHODD, version 01SEP2015. 
ATC Level 2 Class is sorted in descending order in the Overall group. Within each ATC Level 2 Class, Preferred Name is sorted in descending order in the Overall group. 
If a medication of the same ATC Level 2 Class and Preferred Name is reported multiple times for a patient, it is counted only once.  
This table includes corticosteroid medications started after the date of last dose of study drug. 
Program: <program name (date, time)>, Path: <path> 
Programming note for “Post Last Dose” column: N for Post Last dose column is the number of patients reported a Date of Last Dose. 
 
 


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Table B.14.1.7.1 Prior Medications other than Corticosteroid Medications (ITT Population) 
 
Number of patients (%) 
ATC Level 2 Class 
    Preferred Name 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
Number of patients reported prior medications other than 
corticosteroid medications 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
ATC Level 2 Class 1 
x (x.x) 
x (x.x) 
x (x.x) 
Preferred Name 1.1 
x (x.x) 
x (x.x) 
x (x.x) 
         Preferred Name 1.2 
x (x.x) 
x (x.x) 
x (x.x) 
         Preferred Name 1.3 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
ATC Level 2 Class 2 
x (x.x) 
x (x.x) 
x (x.x) 
Preferred Name 2.1 
x (x.x) 
x (x.x) 
x (x.x) 
Preferred Name 2.2 
x (x.x) 
x (x.x) 
x (x.x) 
         Preferred Name 2.3 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
…etc… 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
 
 
Percentages are based on the number of patients in each starting dose and overall. 
Medications are coded using WHODD, version 01SEP2015. 
ATC Level 2 Class is sorted in descending order in the Overall group. Within each ATC Level 2 Class, Preferred Name is sorted in descending order in the Overall group. 
If a medication of the same ATC Level 2 Class and Preferred Name is reported multiple times for a patient, it is counted only once.  
Prior medications are those non-corticosteroid mediations that have stopped prior to the first dose of study drug.  
Program: <program name (date, time)>, Path: <path> 
 
 
 


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Table B.14.1.7.2 Concomitant Medications other than Corticosteroid Medications (ITT Population) 
 
Number of patients (%) 
ATC Level 2 Class 
    Preferred Name 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
Number of patients reported concomitant medications other than 
corticosteroid medications 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
ATC Level 2 Class 1 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
Preferred Name 1.1 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
         Preferred Name 1.2 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
         Preferred Name 1.3 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
ATC Level 2 Class 2 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
Preferred Name 2.1 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
Preferred Name 2.2 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
          Preferred Name 2.3 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
…etc… 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received during the time the medication was taken. 
Percentages are based on the number of patients in each dose group and overall. 
Medications are coded using WHODD, version 01SEP2015. 
ATC Level 2 Class is sorted in descending order in the Overall group. Within each ATC Level 2 Class, Preferred Name is sorted in descending order in the Overall group. 
If a medication of the same ATC Level 2 Class and Preferred Name is reported multiple times for a patient, it is counted only once.  
Concomitant medications are those non- corticosteroid mediations that are ongoing or stopped on or after the date of first dose of study drug. 
Program: <program name (date, time)>, Path: <path> 


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Table B.14.1.7.3 Concomitant Medications other than Corticosteroid Medications Started Post Last Dose (ITT Population) 
 
Number of patients (%) 
ATC Level 2 Class 
    Preferred Name 
Overall 
(N = n) 
Number of patients reported post last dose non-CS medications  
x (x.x) 
 
 
ATC Level 2 Class 1 
x (x.x) 
Preferred Name 1.1 
x (x.x) 
         Preferred Name 1.2 
x (x.x) 
        Preferred Name 1.3 
x (x.x) 
 
 
ATC Level 2 Class 2 
x (x.x) 
Preferred Name 2.1 
x (x.x) 
Preferred Name 2.2 
x (x.x) 
         Preferred Name 2.3 
x (x.x) 
 
 
…etc… 
x (x.x) 
 
 
 
 
Percentages are based on the number of patients in the ITT population. 
Medications are coded using WHODD, version 01SEP2015. 
ATC Level 2 Class is sorted in descending order in the Overall group. Within each ATC Level 2 Class, Preferred Name is sorted in descending order in the Overall group. 
If a medication of the same ATC Level 2 Class and Preferred Name is reported multiple times for a patient, it is counted only once.  
This table includes corticosteroid medications started after the date of last dose of study drug. 
Program: <program name (date, time)>, Path: <path> 
 


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Table B.14.1.8.1 Number of Dose Adjustments and Maximum Dose (ITT Population) 
 
Starting Dose  
400 mg QD 
(N = n) 
Starting Dose  
400 mg BID 
(N = n) 
Overall 
(N = n) 
Number of Dose Adjustments 
 
 
 
     n 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
Maximum Dose 
 
 
 
     400 mg QD 
x (x.x) 
x (x.x) 
x (x.x) 
     400 mg BID 
x (x.x) 
x (x.x) 
x (x.x) 
     600 mg BID 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Program: <program name (date, time)>, Path: <path> 
 


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Table B.14.1.8.2 Study Drug Exposure and Compliance (ITT Population) 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
Duration of Exposure (day) 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
Calculated Dose (mg/day) 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
Compliance (%) 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
Compliance (%) n (%) 
 
 
 
 
    100 % 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
    >=80 % and < 100 % 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
    < 80% 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
The duration of exposure at each dose level is the date of last dose in a given dose level – date of first dose in the same dose level + 1. 
The duration of exposure for “Overall” is date of last dose – date of first dose  + 1. 


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Calculated dose in mg/day at each dose level = [Dose level (mg/day) x (Duration of exposure (x2 if BID) – doses missed)]/Duration of Exposure. 
Compliance with study drug = the actual number of doses taken divided by the expected number of doses take, expressed as a percentage. 
Program: <program name (date, time)>, Path: <path> 


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Table B.14.2.1.1.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg (mITT Population) 
Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall  
(N = n) 
N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
 
 
 
 
Percentages are based on the number of patients in each dose group and overall.   
Patients are classified into different dose levels according to the dose they received prior to achieving CDA. 
Confidence Intervals are based on the Exact (Clopper-Pearson) method. 
Primary efficacy endpoint: Proportion of patients who achieved CDA within 4 weeks of staring PRN1008 treatment, i.e. At or prior to Week 5, without prednis(ol)one equivalent 
> 0.5 mg/kg on the day pror to the assessment. 
Program: <program name (date, time)>, Path: <path> 
 
Table B.14.2.1.1.2 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg (Completer Population) 
Table B.14.2.1.1.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg (PP Population) 
 
 


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Table B.14.2.1.2.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Age (mITT Population) 
Subgroup 
Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 
mg/kg 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall  
(N = n) 
Age < 50 
Number of patients 
n 
n 
n 
n 
 
N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
Age ≥ 50 
Number of patients 
n 
n 
n 
n 
 
N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
Percentages are based on the number of patients in each subgroup, dose group and overall.   
Patients are classified into different dose levels according to the dose they received prior to achieving CDA. 
Confidence Intervals are based on the Exact (Clopper-Pearson) method. 
Primary efficacy endpoint: Proportion of patients who achieved CDA within 4 weeks of staring PRN1008 treatment, i.e. At or prior to Week 5, without prednis(ol)one equivalent 
> 0.5 mg/kg on the day pror to the assessment.. 
Program: <program name (date, time)>, Path: <path> 
 
Table B.14.2.1.2.2 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Age (Completer Population) 
Table B.14.2.1.2.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Age (PP Population) 
Note: Subgroups are: ‘Age < 50 years’ and ‘Age  ≥ 50 years’ 
 
Table B.14.2.1.3.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Gender (mITT Population) 
Table B.14.2.1.3.2 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Gender (Completer 
Population) 
Table B.14.2.1.3.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Gender (PP Population) 
Note: Subgroups are: ‘Male’ and ‘Female’ 
 


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Table B.14.2.1.4.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Baseline Total AntiDSG 
(mITT Population) 
Table B.14.2.1.4. Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Baseline Total Anti-DSG 
(Completer Population) 
Table B.14.2.1.4.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Baseline Total Anti-DSG (PP 
Population) 
Note: Subgroups are: ‘Baseline Total DSG antibody ≥ 100 units’ and ‘Baseline Total DSG antibody < 100 units' 
 
Table B.14.2.1.5.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Pemphigus History (mITT 
Population) 
Table B.14.2.1.5.2 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Pemphigus History 
(Completer Population) 
Table B.14.2.1.5.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Pemphigus History (PP 
Population) 
Note: Subgroups are: ‘Newly diagnosed (< 6 months from screening)’, ‘Relapsed (>6 months from screening) 
Additional footnote: Newly diagnosed is defined as subjects who were diagnosed at most 6 months prior to date of screening, otherwise the subject 
is relapsed. 
 
Table B.14.2.1.6.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Baseline Pemphigus Anti-
DSG Profile (mITT Population) 
Table B.14.2.1.6.2 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Baseline Pemphigus Anti-
DSG Profile (Completer Population) 
Table B.14.2.1.6.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup – Baseline Pemphigus Anti-
DSG Profile (PP Population) 
Note: Subgroups are: ‘Phemphigus Vularis (PV)’, ‘Pemphigus foliaceus (PF)’ and ‘Negative anti-DSG profile’ 
 
Table B.14.2.1.7.1 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup Analysis – Baseline 
Pemphigus Severity (mITT Population) 
Table B.14.2.1.7.2 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup Analysis – Baseline 
Pemphigus Severity (Completer Population) 
Table B.14.2.1.7.3 Proportion of patients Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg – Subgroup Analysis – Baseline 
Pemphigus Severity (PP Population) 
Note: Subgroups are: ‘Mild at Baseline’, ‘Moderate at Baseline’ 
Additional footnote: Based on PDAI total activity score. For newly diagnosed patients: Mild is 0 to 14 and Moderate is 15 to 44. For relapsed patients: 
Mild is 0 to 8 and Moderate is 9 to 24. 
 
 


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Table B.14.2.2.1.1 Cumulative Incidence of Patients Achieved CDA by Visit – Cumulative Incidence (mITT Population) 
Visit 
Achieved CDA 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall  
(N = n) 
SCREENED 
N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
WEEK 1 DAY 1 
Cumulative N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
WEEK 1 DAY 2 
Cumulative N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
WEEK 3 DAY 15 
…etc… 
 
 
 
 
…etc.. 
 
 
 
 
 
WEEK 29 DAY 197  
Cumulative N achieved (%) 
 
 
 
x (x.x) 
 
     - 80% CI 
 
 
 
x.xx, x.xx 
 
     - 95% CI 
 
 
 
x.xx, x.xx 
 
 
 
 
 
 
Percentages are based on the number of patients in each dose group and overall.   
Patients are classified into different dose levels according to the dose they received prior to achieving CDA.  
Confidence Intervals are based on the Exact (Clopper-Pearson) method. 
Secondary efficacy endpoint: Proportion of patients who achieved CDA at each visit.  
Program: <program name (date, time)>, Path: <path> 
 
 


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Table B.14.2.2.1.2 Cumulative Incidence of Patients Achieved CDA by Visit (Completer Population) 
Table B.14.2.2.1.3 Cumulative Incidence of Patients Achieved CDA by Visit (PP Population) 
 
Table B.14.2.2.2.1 Cumulative Incidence of Patients Achieved CDA by Visit without Corticosteroids (mITT Population) 
Table B.14.2.2.2.2 Cumulative Incidence of Patients Achieved CDA by Visit without Corticosteroids (Completer Population) 
Table B.14.2.2.2.3 Cumulative Incidence of Patients Achieved CDA by Visit without Corticosteroids (PP Population) 
 
Table B.14.2.2.3.1 Cumulative Incidence of Patients Achieved CDA by Visit with prednis(ol)one >0.5 mg/kg (mITT Population) 
Table B.14.2.2.3.2 Cumulative Incidence of Patients Achieved CDA by Visit with prednis(ol)one >0.5 mg/kg (Completer Population) 
Table B.14.2.2.3.3 Cumulative Incidence of Patients Achieved CDA by Visit with prednis(ol)one >0.5 mg/kg (PP Population) 
 
Table B.14.2.2.4.1 Cumulative Incidence of Patients Achieved CDA by Visit without prednis(ol)one >0.5 mg/kg – Cumulative Incidence (mITT Population) 
Table B.14.2.2.4.2 Cumulative Incidence of Patients Achieved CDA by Visit without prednis(ol)one >0.5 mg/kg – Cumulative Incidence (Completer 
4opulation) 
Table B.14.2.2.4.3 Cumulative Incidence of Patients Achieved CDA by Visit without prednis(ol)one >0.5 mg/kg (PP Population) 
 
 
 


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Table B.14.2.2.5.1 Proportion of patients Achieved CDA by Visit (mITT Population) 
Visit 
Achieved CDA 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall  
(N = n) 
SCREENED 
N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
WEEK 1 DAY 1 
N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
WEEK 1 DAY 5 
 
 
 
 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
WEEK 29 DAY 197   
 
 
 
 
 
 
 
 
 
 
 
Percentages are based on the number of patients in each dose group and overall.   
Patients are classified into different dose levels according to the dose they received prior to achieving CDA. 
Confidence Intervals are based on the Exact (Clopper-Pearson) method. 
Secondary efficacy endpoint: Proportion of patients who achieved CDA at each visit.  
Program: <program name (date, time)>, Path: <path> 
 
Table B.14.2.2.5.2 Proportion of patients Achieved CDA by Visit (Completer Population) 
Table B.14.2.2.5.3 Proportion of patients Achieved CDA by Visit (PP Population) 
 


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Table B.14.2.2.6.1 Proportion of patients Achieved CDA with prednis(ol)one >0.5 mg/kg by Visit (mITT Population) 
Table B.14.2.2.6.2 Proportion of patients Achieved CDA with prednis(ol)one >0.5 mg/kg by Visit (Completer Population) 
Table B.14.2.2.6.3 Proportion of patients Achieved CDA with prednis(ol)one >0.5 mg/kg by Visit (PP Population) 
 
Table B.14.2.2.7.1 Proportion of patients Achieved CDA without prednis(ol)one >0.5 mg/kg by Visit (mITT Population) 
Table B.14.2.2.7.2 Proportion of patients Achieved CDA without prednis(ol)one >0.5 mg/kg by Visit (Completer Population) 
Table B.14.2.2.7.3 Proportion of patients Achieved CDA without prednis(ol)one >0.5 mg/kg by Visit (PP Population) 
 
 


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Table B.14.2.2.8.1 Cumulative Incidence of Patients Achieved CR by Visit – Cumulative Incidence (mITT Population) 
Visit 
Achieved CR 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall  
(N = n) 
SCREENED 
N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
WEEK 1 DAY 1 
Cumulative N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
WEEK 1 DAY 2 
Cumulative N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
WEEK 3 DAY 15 
…etc… 
 
 
 
 
…etc.. 
 
 
 
 
 
WEEK 29 DAY 197  
Cumulative N achieved (%) 
 
 
 
x (x.x) 
 
     - 80% CI 
 
 
 
x.xx, x.xx 
 
     - 95% CI 
 
 
 
x.xx, x.xx 
 
 
 
 
 
 
Percentages are based on the number of patients in each dose group and overall.   
Patients are classified into different dose levels according to the dose they received prior to achieving CR.  
Confidence Intervals are based on the Exact (Clopper-Pearson) method. 
Secondary efficacy endpoint: Proportion of patients who achieved CDA at each visit.  
Program: <program name (date, time)>, Path: <path> 


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Table B.14.2.2.8.2 Cumulative Incidence of Patients Achieved CR by Visit – Cumulative Incidence (Completer Population) 
Table B.14.2.2.8.3 Cumulative Incidence of Patients Achieved CR by Visit – Cumulative Incidence (PP Population) 
Table B.14.2.2.9.1 Cumulative Incidence of Patients Achieved CR with prednis(ol)one >0.5 mg/kg by Visit – Cumulative Incidence (mITT Population) 
Table B.14.2.2.9.2 Cumulative Incidence of Patients Achieved CR with prednis(ol)one >0.5 mg/kg by Visit – Cumulative Incidence (Completer Population) 
Table B.14.2.2.9.3 Cumulative Incidence of Patients Achieved CR with prednis(ol)one >0.5 mg/kg by Visit – Cumulative Incidence (PP Population) 
Table B.14.2.2.10.1 Cumulative Incidence of Patients Achieved CR without prednis(ol)one >0.5 mg/kg by Visit – Cumulative Incidence (mITT Population) 
Table B.14.2.2.10.2 Cumulative Incidence of Patients Achieved CR without prednis(ol)one >0.5 mg/kg by Visit – Cumulative Incidence (Completer 
Population) 
Table B.14.2.2.10.3 Cumulative Incidence of Patients Achieved CR without prednis(ol)one >0.5 mg/kg by Visit – Cumulative Incidence (PP Population) 
 
 


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Table B.14.2.2.11.1 Proportion of patients Achieved CR by Visit (mITT Population) 
Visit 
Achieved CR 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall  
(N = n) 
SCREENED 
N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
WEEK 1 DAY 1 
N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
 
WEEK 1 DAY 2 
 
 
 
 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
WEEK 29 DAY 197   
 
 
 
 
 
 
 
 
 
 
 
Percentages are based on the number of patients in each dose group and overall.   
Patients are classified into different dose levels according to the dose they received prior to achieving CDA. 
Confidence Intervals are based on the Exact (Clopper-Pearson) method. 
Secondary efficacy endpoint: Proportion of patients who achieved CDA at each visit.  
Program: <program name (date, time)>, Path: <path> 
 
Table B.14.2.2.11.2 Proportion of patients Achieved CR by Visit (Completer Population) 
Table B.14.2.2.11.3 Proportion of patients Achieved CR by Visit (PP Population) 
 


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Table B.14.2.2.12.1 Proportion of patients Achieved CR with prednis(ol)one >0.5 mg/kg by Visit (mITT Population) 
Table B.14.2.2.12.2 Proportion of patients Achieved CR with prednis(ol)one >0.5 mg/kg by Visit (Completer Population) 
Table B.14.2.2.12.3 Proportion of patients Achieved CR with prednis(ol)one >0.5 mg/kg by Visit (PP Population) 
 
Table B.14.2.2.13.1 Proportion of patients Achieved CR without prednis(ol)one >0.5 mg/kg by Visit (mITT Population) 
Table B.14.2.2.13.2 Proportion of patients Achieved CR without prednis(ol)one >0.5 mg/kg by Visit (Completer Population) 
Table B.14.2.2.13.3 Proportion of patients Achieved CR without prednis(ol)one >0.5 mg/kg by Visit (PP Population) 
 
Table B.14.2.2.14.1 Proportion of patients Achieved CR at or Prior to Week 25 with prednis(ol)one >0.5 mg/kg (mITT Population) 
Achieved CDA at or Prior to Week 5 without prednis(ol)one >0.5 mg/kg 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall  
(N = n) 
N achieved (%) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     - 80% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
     - 95% CI 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
x.xx, x.xx 
 
 
 
 
 
Percentages are based on the number of patients in each dose group and overall.   
Patients are classified into different dose levels according to the dose they received prior to achieving CR. 
Confidence Intervals are based on the Exact (Clopper-Pearson) method. 
Primary efficacy endpoint: Proportion of patients who achieved CR within 24 weeks of staring PRN1008 treatment, i.e. At or prior to Week 5, without prednis(ol)one equivalent 
> 0.5 mg/kg on the day pror to the assessment. 
Program: <program name (date, time)>, Path: <path> 
 
Table B.14.2.2.14.2 Proportion of patients Achieved CR at or Prior to Week 25 with prednis(ol)one >0.5 mg/kg (Completer Population) 
Table B.14.2.2.14.3 Proportion of patients Achieved CR at or Prior to Week 25 with prednis(ol)one >0.5 mg/kg (PP Population) 
 
Table B.14.2.2.15.1 Proportion of patients Achieved CR at or Prior to Week 25 without prednis(ol)one >0.5 mg/kg (mITT Population) 
Table B.14.2.2.15.2 Proportion of patients Achieved CR at or Prior to Week 25 without prednis(ol)one >0.5 mg/kg (Completer Population)  
Table B.14.2.2.15.3 Proportion of patients Achieved CR at or Prior to Week 25 without prednis(ol)one >0.5 mg/kg (PP Population) 
 


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Table B.14.2.2.16.1 Duration of Complete Remission (CR) (mITT Population) 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall  
(N = n) 
Duration of CR 
x 
x 
x 
x 
     n 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Mean (SD) 
x.xx 
x.xx 
x.xx 
x.xx 
     Median 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
     Min, Max 
 
 
 
 
 
 
 
 
 
Duration of CR = Last Known CR Date – First Known CR Date + 1. 
Patients are classified into different dose levels according to the dose they received prior to achieving CR.  
 
Table B.14.2.2.16.2 Duration of Complete Remission (CR) (Completer Population) 
Table B.14.2.2.16.3 Duration of Complete Remission (CR) (PP Population) 
 
 


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Table B.14.2.2.17.1 Time to First CDA: Kaplan Meier Estimates (mITT Population) 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
Time to first CDA (days) [1] 
 
 
 
 
     n 
x 
x 
x 
x 
     25% Quartile 
x.x 
x.x 
x.x 
x.x 
          - 80% CI 
(x.xx, x.xx) 
(x.xx, x.xx) 
(x.xx, x.xx) 
(x.xx, x.xx) 
          - 95% CI 
(x.xx, x.xx) 
(x.xx, x.xx) 
(x.xx, x.xx) 
(x.xx, x.xx) 
     Median 
x.x 
x.x 
x.x 
x.x 
          - 80% CI 
(x.xx, x.xx) 
(x.xx, x.xx) 
(x.xx, x.xx) 
(x.xx, x.xx) 
          - 95% CI 
(x.xx, x.xx) 
(x.xx, x.xx) 
(x.xx, x.xx) 
(x.xx, x.xx) 
     75% Quartile 
x.x 
x.x 
x.x 
x.x 
          - 80% CI 
(x.xx, x.xx) 
(x.xx, x.xx) 
(x.xx, x.xx) 
(x.xx, x.xx) 
          - 95% CI 
(x.xx, x.xx) 
(x.xx, x.xx) 
(x.xx, x.xx) 
(x.xx, x.xx) 
     Min, Max [2] 
x , x 
x , x 
x , x* 
x , x* 
 
 
 
 
 
Kaplan-Meier estimates % (n) [3] 
 
 
 
 
    15 Days 
x.x (xx) 
x.x (xx) 
x.x (xx) 
x.x (xx) 
    29 Days 
x.x (xx) 
x.x (xx) 
x.x (xx) 
x.x (xx) 
    57 Days 
x.x (xx) 
x.x (xx) 
x.x (xx) 
x.x (xx) 
    85 Days 
x.x (xx) 
x.x (xx) 
x.x (xx) 
x.x (xx) 
    113 Days 
x.x (xx) 
x.x (xx) 
x.x (xx) 
x.x (xx) 
    141 Days 
x.x (xx) 
x.x (xx) 
x.x (xx) 
x.x (xx) 
    169 Days 
x.x (xx) 
x.x (xx) 
x.x (xx) 
x.x (xx) 
Patients are classified into different dose levels according to the dose they received at a given visit. 
[1] Time to first CDA in days will be calculated as: Date of first occurrence of CDA – Date of first study drug dosing of the dose level where the CDA is observed + 1. 
Patients who did not achieve CDA are censored at the last day in a dose level or date of study completion/discontinuation. 80% and 95% confidence intervals are calculated 
using the LOGLOG transformation. 
[2] * indicates that this value is from a censored data. 


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[3] Kaplan-Meier estimate of the probability of experiencing the event on or before the specified timepoint (n = number of patients remaining at risk beyond the specified 
timepoint.) 
Program: <program name (date, time)>, Path: <path> 
 
Table B.14.2.2.17.2 Time to First CDA: Kaplan Meier Estimates (Completer Population) 
Table B.14.2.2.17.3 Time to First CDA: Kaplan Meier Estimates (PP Population) 
 
Table B.14.2.2.18.1 Time to First CR: Kaplan Meier Estimates (mITT Population) 
Table B.14.2.2.18.2 Time to First CR: Kaplan Meier Estimates (Completer Population) 
Table B.14.2.2.18.3 Time to First CR: Kaplan Meier Estimates (PP Population) 
 
Table B.14.2.2.19.1 Time to First ECP: Kaplan Meier Estimates (mITT Population) 
Table B.14.2.2.19.2 Time to First ECP: Kaplan Meier Estimates (Completer Population) 
Table B.14.2.2.19.3 Time to First ECP: Kaplan Meier Estimates (PP Population) 
 
Table B.14.2.2.20.1 Time to First Relapse: Kaplan Meier Estimates (mITT Population) 
Table B.14.2.2.20.2 Time to First Relapse: Kaplan Meier Estimates (Completer Population)  
Table B.14.2.2.20.3 Time to First Relapse: Kaplan Meier Estimates (PP Population) 
 
Table B.14.2.2.21.1 Time to First Increase in PDAI: Kaplan Meier Estimates (mITT Population) 
Table B.14.2.2.21.2 Time to First Increase in PDAI: Kaplan Meier Estimates (Completer Population)  
Table B.14.2.2.21.3 Time to First Increase in PDAI: Kaplan Meier Estimates (PP Population) 
 


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Table B.14.2.2.22.1 Cumulative and Daily Average Corticosteroid Usage by Treatment Period (mITT Population) 
Period 
Overall 
(N = n) 
Cumulative CS Usage (mg) 
Daily Average CS Usage (mg/day) 
Treatment Period 1 (Up to Week 12) [1] 
 
 
     n 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
 
 
 
Treatment Period 2 (from Week 13 to Week 24) [2] 
 
 
 …etc.. 
 
 
 
 
 
Entire Treatment Period (from Week 1 to Week 24) [3] 
 
 
…etc.. 
 
 
 
 
 
Follow Up Period [2] 
 
 
…etc.. 
 
 
All corticosteroids were converted into prednisolone. Each 10 mg of prednisolone is equivalent to 10 mg of prednisone, 8 mg of methylprednisolone, 1.5 mg dexamethasone, 
40 mg hydrocortisone or 1.5 mg betamethasone. 
 [1] Period Start Date = Week 1 Day 1. Period End Date = Date of Week 13 Visit -1 or Date of Last Study Drug if patient discontinued prior to Week 13. 
[2] Period Start Date = Date of Week 13 Visit; Period End Date = Date of Week 24 Visit – 1 or Date of Last Study Drug if patient discontinued prior to Week 24. 
[3] Period Start Date = Week 1 Day 1; Period End Date = Date of Week 24 Visit – 1 or Date of Last Study Drug if patient discontinued prior to Week 24.. 
[4] Period Start Date = Date of Last Study Drug + 1. Period End Date = Date of Week 29/End of Study.  
Program: <program name (date, time)>, Path: <path> 
 
Table B.14.2.2.22.2 Cumulative and Daily Average Corticosteroid Usage by Treatment Period – (Completer Population) 
Table B.14.2.2.22.3 Cumulative and Daily Average Corticosteroid Usage by Treatment Period – (PP Population) 
 
 


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Table B.14.2.2.23.1 Corticosteroid Dose (mg/day) on the Day or at the Day Prior to Visit by Study Visit – (mITT Population) 
Study Visit 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
SCREENED (on the Day of) 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 1 DAY 1 (On the Day of) - BASELINE 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 1 DAY 1 (On the Day of) - CHANGE FROM SCREENED  
 
 
 
 
 
 
 
 
 
WEEK 3 DAY 15 (the day prior )  
 
 
 
 
WEEK 3 DAY 15 (the day prior)  - CHANGE FROM SCREENED 
 
 
 
 
WEEK 3 DAY 15 (the day prior)  - CHANGE FROM BASELINE 
 
 
 
 
 
 
 
 
 
..etc.. 
 
 
 
 
All corticosteroids were converted into prednisolone. Each 10 mg of prednisolone is equivalent to 10 mg of prednisone, 8 mg of methylprednisolone, 1.5 mg dexamethasone, 
40 mg hydrocortisone or 1.5 mg betamethasone. 
For SCREENED and WEEK 1 DAY1 (BASELIEN): The dosage level on the day of the visit date.  
For the post-baseline visit: The dosage level at each visit is defined as the dose level the patient is on the day before the visit date. 
Week 1 Day 2 and unscheduled assessments are not included. 
Program: <program name (date, time)>, Path: <path> 


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Table B.14.2.2.22.2 Corticosteroid Dose (mg/day) on the Day or at the Day Prior by Study Visit – (Completer Population) 
Table B.14.2.2.22.3 Corticosteroid Dose (mg/day) on the Day or at the Day Prior by Study Visit – (PP Population) 
 
Table B.14.2.2.24.1 Corticosteroid Dose (mg/kg/day) on the Day or at the Day Prior by Study Visit – (mITT Population) 
Table B.14.2.2.24.2 Corticosteroid Dose (mg/kg/day) on the Day or at the Day Prior by Study Visit – (Completer Population)  
Table B.14.2.2.24.3 Corticosteroid Dose (mg/kg/day) on the Day or at the Day Prior by Study Visit – (PP Population) 
 
 


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Table B.14.2.2.25.1 Corticosteroid Dose (mg/day) by Study Visit in Patients who achieved at least one CDA – (mITT Population) 
Study Visit 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
WEEK 3 DAY 15 (the day prior )  
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 3 DAY 15 (the day prior ) - Change from Baseline  
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
 WEEK 5 DAY 29 
 
 
 
 
 
 
 
 
 
 WEEK 5 DAY 29 – Cshange from Baseline 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
All corticosteroids were converted into prednisolone. Each 10 mg of prednisolone is equivalent to 10 mg of prednisone, 8 mg of methylprednisolone, 1.5 mg dexamethasone, 
40 mg hydrocortisone or 1.5 mg betamethasone. 
The dosage level at each visit is defined as the dose level the patient is on the day before the CDA reponse visit date. 
Week 1 Day 2 and unscheduled assessments are not included. 
Program: <program name (date, time)>, Path: <path> 
 
Table B.14.2.2.25.2 Corticosteroid Dose (mg/day) by Study Visit in Patients who achieved at least one CDA – (Completer Population) 
Table B.14.2.2.25.3 Corticosteroid Dose (mg/day) by Study Visit in Patients who achieved at least one CDA – (PP Population) 


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Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
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Table B.14.2.2.26.1 Corticosteroid Dose (mg/kg/day) by Study Visit in Patients who achieved at least one CDA – (mITT Population) 
Table B.14.2.2.26.2 Corticosteroid Dose (mg/kg/day) by Study Visit in Patients who achieved at least one CDA – (Completer Population) 
Table B.14.2.2.26.3 Corticosteroid Dose (mg/kg/day) by Study Visit in Patients who achieved at least one CDA – (PP Population 
Table B.14.2.2.27.1 Corticosteroid Dose (mg/day) by Study Visit in Patients who achieved at least one CR – (mITT Population) 
Table B.14.2.2.27.2 Corticosteroid Dose (mg/day) by Study Visit in Patients who achieved at least one CR – (Completer Population) 
Table B.14.2.2.27.3 Corticosteroid Dose (mg/day) by Study Visit in Patients who achieved at least one CR – (PP Population) 
 
Table B.14.2.2.28.1 Corticosteroid Dose (mg/kg/day) by Study Visit in Patients who achieved at least one CR – (mITT Population) 
Table B.14.2.2.28.2 Corticosteroid Dose (mg/kg/day) by Study Visit in Patients who achieved at least one CR – (Completer Population) 
Table B.14.2.2.28.3 Corticosteroid Dose (mg/kg/day) by Study Visit in Patients who achieved at least one CR – (PP Population) 
 
 


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Table B.14.2.2.29.1 PDAI – Total Activity Score – Actual Values, Change and Percent Change from Screening and from Baseline (mITT Population) 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
SCREENED 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 1 DAY 1 (BASELINE) 
 
 
 
 
    n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 3 DAY 15  
 
 
 
 
    n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 3 DAY 15 – CHANGE FROM SCREENED 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 


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Table B.14.2.2.29.1 PDAI – Total Activity Score – Actual Values, Change and Percent Change from Screening and from Baseline (mITT Population) 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
WEEK 3 DAY 15 – PERCENT CHANGE FROM SCREENED 
 
 
 
 
 
 
 
 
 
WEEK 3 DAY 15 – CHANGE FROM BASELINE 
 
 
 
 
 
 
 
 
 
WEEK 3 DAY 15 – PERCENT CHANGE FROM BASELINE 
 
 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at each visit. 
Program: <program name (date, time)>, Path: <path> 
 
Table B.14.2.2.29.2 PDAI – Total Activity Score – Actual Values, Change and Percent Change from Screening and from Baseline (Completer Population) 
Table B.14.2.2.29.3 PDAI – Total Activity Score – Actual Values, Change and Percent Change from Screening and from Baseline (PP Population) 
 
Table B.14.2.2.30.1 PDAI – Total Damage Score – Actual Values and  Change from Baseline (mITT Population) 
Table B.14.2.2.30.2 PDAI – Total Damage Score – Actual Values and  Change from Baseline (Completer Population) 
Table B.14.2.2.30.3 PDAI – Total Damage Score – Actual Values and  Change from Baseline (PP Population) 
 
 


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Table B.14.2.2.31.1 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline (mITT Population) 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
WEEK 1 DAY 1 (BASELINE) 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 3 DAY 15 
 
 
 
 
    n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 3 DAY 15 – CHANGE FROM BASELINE 
 
 
 
 
    n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 5 DAY 29  
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Table B.14.2.2.31.1 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline (mITT Population) 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
WEEK 5 DAY 29 – CHANGE FROM BASELINE 
 
 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at each visit. 
Program: <program name (date, time)>, Path: <path> 
 
Table B.14.2.2.31.2 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline (Completer Population) 
Table B.14.2.2.29.3 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline (PP Population) 
 
Table B.14.2.2.32.1 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline (mITT Population) 
Table B.14.2.2.32.2 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline (Completer Population) 
Table B.14.2.2.32.3 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline (PP Population) 
 
Table B.14.2.2.33.1 ABSIS – Severity Total Score –Actual Values and Change from Baseline (mITT Population) 
Table B.14.2.2.33.2 ABSIS – Severity Total Score –Actual Values and Change from Baseline (Completer Population) 
Table B.14.2.2.33.3 ABSIS – Severity Total Score –Actual Values and Change from Baseline (PP Population) 
 
Table B.14.2.2.34.1 ABSIS – Total Activity Score –Actual Values and Change from Baseline (mITT Population) 
Table B.14.2.2.34.2 ABSIS – Total Activity Score –Actual Values and Change from Baseline (Completer Population) 
Table B.14.2.2.34.3 ABSIS – Total Activity Score –Actual Values and Change from Baseline (PP Population) 
 
Table B.14.2.2.35.1 ABSIS – Total Score –Actual Values and Change from Baseline (mITT Population) 
Table B.14.2.2.35.2 ABSIS – Total Score –Actual Values and Change from Baseline (Completer Population) 
Table B.14.2.2.35.3 ABSIS – Total Score –Actual Values and Change from Baseline (PP Population) 
 
 
 
 


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Note: For Tables B.14.2.2.36.1 to B.14.2.2.45.3, Baseline Oral Involvement = ‘Baseline Oral Involvement = 0’ vs ‘Baseline Oral Involvement > 0’, start each 
subgroup on a new page. 
Table B.14.2.2.36.1 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline – by Baseline Oral Involvement (mITT Population) 
Table B.14.2.2.36.2 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline – by Baseline Oral Involvement (Completer Population) 
Table B.14.2.2.36.3 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline – by Baseline Oral Involvement (PP Population) 
 
Table B.14.2.2.37.1 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (mITT Population) 
Table B.14.2.2.37.2 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (Completer Population) 
Table B.14.2.2.37.3 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (PP Population) 
 
 
Table B.14.2.2.38.1 ABSIS – Severity Total Score –Actual Values and Change from Baseline– by Baseline Oral Involvement (mITT Population) 
Table B.14.2.2.38.2 ABSIS – Severity Total Score –Actual Values and Change from Baseline– by Baseline Oral Involvement (Completer Population)  
Table B.14.2.2.38.3 ABSIS – Severity Total Score –Actual Values and Change from Baseline– by Baseline Oral Involvement (PP Population) 
 
Table B.14.2.2.39.1 ABSIS – Total Activity Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (mITT Population) 
Table B.14.2.2.39.2 ABSIS – Total Activity Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (Completer Population) 
Table B.14.2.2.39.3 ABSIS – Total Activity Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (PP Population) 
 
Table B.14.2.2.40.1 ABSIS – Total Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (mITT Population) 
Table B.14.2.2.40.2 ABSIS – Total Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (Completer Population) 
Table B.14.2.2.40.3 ABSIS – Total Score –Actual Values and Change from Baseline – by Baseline Oral Involvement (PP Population) 
 
Table B.14.2.2.41.1 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline – by Baseline Skin Involvement (mITT Population) 
Table B.14.2.2.41.2 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline – by Baseline Skin Involvement (Completer Population) 
Table B.14.2.2.41.3 ABSIS – Skin Involvement Total Score – Actual Values and Change from Baseline – by Baseline Skin Involvement (PP Population) 
 


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Table B.14.2.2.42.1 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (mITT Population) 
Table B.14.2.2.42.2 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (Completer Population) 
Table B.14.2.2.42.3 ABSIS – Oral Involvement Total Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (PP Population) 
 
Table B.14.2.2.43.1 ABSIS – Severity Total Score –Actual Values and Change from Baseline– by Baseline Skin Involvement (mITT Population) 
Table B.14.2.2.43.2 ABSIS – Severity Total Score –Actual Values and Change from Baseline– by Baseline Skin Involvement (Completer Population) 
Table B.14.2.2.43.3 ABSIS – Severity Total Score –Actual Values and Change from Baseline– by Baseline Skin Involvement (PP Population) 
 
Table B.14.2.2.44.1 ABSIS – Total Activity Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (mITT Population) 
Table B.14.2.2.44.2 ABSIS – Total Activity Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (Completer Population) 
Table B.14.2.2.44.3 ABSIS – Total Activity Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (PP Population) 
 
Table B.14.2.2.45.1 ABSIS – Total Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (mITT Population) 
Table B.14.2.2.45.2 ABSIS – Total Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (Completer Population) 
Table B.14.2.2.45.3 ABSIS – Total Score –Actual Values and Change from Baseline – by Baseline Skin Involvement (PP Population) 
 
Table B.14.2.2.46.1 ABQOL – Actual Values and Change from Baseline (mITT Population) 
Table B.14.2.2.46.2 ABQOL – Actual Values and Change from Baseline (Completer Population) 
Table B.14.2.2.46.3 ABQOL – Actual Values and Change from Baseline (PP Population) 
 
Table B.14.2.2.47.1 TABQOL – Actual Values and Change from Baseline (mITT Population) 
Table B.14.2.2.47.2 TABQOL – Actual Values and Change from Baseline (Completer Population) 
Table B.14.2.2.47.3 TABQOL – Actual Values and Change from Baseline (PP Population) 
 
Table B.14.2.2.48.1 SNAQ – Actual Values and Change from Baseline (mITT Population) 
Table B.14.2.2.48.2 SNAQ – Actual Values and Change from Baseline (Completer Population) 
Table B.14.2.2.48.3 SNAQ – Actual Values and Change from Baseline (PP Population) 
 
 


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Table B.14.2.2.49.1 Total Anti-DSG – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (mITT Population) 
 
Group: All patients 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
WEEK 1 DAY 1 (BASELINE) 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 3 DAY 15 
 
 
 
 
    n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 3 DAY 15 – CHANGE FROM BASELINE 
 
 
 
 
    n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 3 DAY 15 – PERCENT CHANGE FROM BASELINE 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  


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Table B.14.2.2.49.1 Total Anti-DSG – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (mITT Population) 
 
Group: All patients 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
 
 
 
 
 
WEEK 5 DAY 29  
 
 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at each visit. 
Total Anti-DSG is the sum of Anti-DSG 1 and Anti-DSG 3 if at least one of them is positive (Anti-DSG 1 ≥ 14 and/or Anti-DSG 3 ≥ 9). 
Program: <program name (date, time)>, Path: <path> 
Repeat for “Group: Baseline < 100” units and “Group: Baseline ≥ 100 units” 
 
Table B.14.2.2.49.2 Total Anti-DSG – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (Completer Population) 
Table B.14.2.2.49.3 Total Anti-DSG – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (PP Population) 
 
Table B.14.2.2.50.1 Anti-DSG 1 – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (mITT Population) 
Footnote: Anti-DSG 1 data above the limit of quantification, i.e. ≥ 14, are included. 
Table B.14.2.2.50.2 Anti-DSG 1  – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (Completer Population) 
Table B.14.2.2.50.3 Anti-DSG 1 – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (PP Population) 
 
Table B.14.2.2.51.1 Anti-DSG 3 – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (mITT Population) 
Footnote: Anti-DSG 3 data above the limit of quantification, i.e. ≥ 9, are included. 
Table B.14.2.2.51.2 Anti-DSG 3  – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (Completer Population) 
Table B.14.2.2.51.3 Anti-DSG 3 – Actual Values, Change and Percent Change from Baseline – Overall and by Baseline Value (PP Population) 
 
 
 


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Table B.14.3.1.1 Summary of Adverse Events (Safety Population) 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Post Last 
Dose 
(N =n) 
Overall 
(N = n) 
Number of Pre-treatment AEs 
 
 
 
 
x 
Number (%) of patients with at least one Pre-treatment AE 
 
 
 
 
x (x.x) 
 
 
 
 
 
 
Number of Pre-treatment SAEs 
 
 
 
 
x  
Number (%) of patients with at least one Pre-treatment SAE 
 
 
 
 
x (x.x) 
 
 
 
 
 
 
Number of Treatment Emergent Adverse Events (TEAE) 
x  
x  
x 
x 
x 
Number (%) of patients with at least one TEAE 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
Number of Related TEAEs 
x  
x  
x 
x 
x 
Number (%) of patients with at least one Related TEAE 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
Number of Treatment Emergent SAEs 
x  
x  
x 
x 
x 
Number (%) of patients with at least one Treatment Emergent SAE 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
Number of Related Treatment Emergent SAEs 
x  
x  
x 
x 
x 
Number (%) of patients with at least one Related Treatment Emergent SAE 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
TEAE - Action taken with study drug – Number (%) of patients* 
 
 
 
 
 
     Dose not changed 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Dose reduced 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Study drug interrupted 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Study drug discontinued 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 


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Table B.14.3.1.1 Summary of Adverse Events (Safety Population) 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Post Last 
Dose 
(N =n) 
Overall 
(N = n) 
     Not applicable 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Unknown 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
TEAE - Maximum Intensity of TEAE – Number (%) of patients 
 
 
 
 
 
     Grade 1 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Grade 2 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Grade 3 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Grade 4 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Grade 5 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at the start of AE. 
Percentages are based on the number of patients in each dose group and overall. 
Adverse events where PT='Pemphigus' are excluded from this table. 
Adverse events are attributed to the dose level where the AE start date is on or after the start date of the dose level, but before the next dose level, if applicable. 
Pre-treatment adverse events are the events that started prior to the first dose. 
Treatment emergent adverse events are the events started on or after the first dose of study drug and prior to the date of last dose of study drug.  
Post last dose adverse events are the events started after the last dose of study drug. 
* Patient is counted for each reported Action taken, therefore patient may be counted more than once. 
Program: <program name (date, time)>, Path: <path> 
Programming note for all AE tables with “Post Last Dose” column: N for Post Last dose column is the number of patients reported a Date of Last Dose. 
 
 


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Table B.14.3.1.2 Treatment Emergent Adverse Events by SOC and PT (Safety Population) 
System Organ Class (SOC) 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Post Last Dose 
(N = n) 
Overall 
(N = n) 
     Preferred Term (PT) 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
Patients with at least one TEAE   
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
SOC1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.3 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
SOC2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.3 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
…etc… 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at the start of AE. 
Percentages are based on the number of patients in each dose group and overall. 
Adverse Events were coded using MedDRA, Version 18.0. 
Adverse events where PT='Pemphigus' are excluded from this table. 
Adverse events are attributed to the dose level where the AE start date is on or after the start date of the dose level, but before the next dose level, if applicable. 
Treatment emergent adverse events are the events started on or after the first dose of study drug and prior to the date of last dose of study drug.  
Adverse events are sorted by overall descending frequency of SOC and within each SOC, by overall descending frequency of PT. 
Program: <program name (date, time)>, Path: <path> 
 
 


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Table B.14.3.1.3 Related Treatment Emergent Adverse Events by SOC and PT (Safety Population) 
System Organ Class (SOC) 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Post Last Dose 
(N = n) 
Overall 
(N = n) 
     Preferred Term (PT) 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
Patients with at least one related TEAE   
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
SOC1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.1 
 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.3 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
SOC2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.3 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
…etc… 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at the start of AE. 
Percentages are based on the number of patients in each dose group and overall. 
Adverse Events were coded using MedDRA, Version 18.0. 
Adverse events where PT='Pemphigus' are excluded from this table. 
Adverse events are attributed to the dose level where the AE start date is on or after the start date of the dose level, but before the next dose level, if applicable. 
Treatment emergent adverse events are the events started on or after the first dose of study drug and prior to the date of last dose of study drug.  
Adverse events are sorted by overall descending frequency of SOC and within each SOC, by overall descending frequency of PT. 
Program: <program name (date, time)>, Path: <path> 
 
 


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Table B.14.3.1.4 Treatment Emergent Adverse Events by Maximum Intensity, SOC and PT (Safety Population) 
 
Overall (N = n) 
System Organ Class (SOC) 
Grade 1 
Grade 2 
Grade 3 
Grade 4 
Grade 5 
     Preferred Term (PT) 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
Patients with at least one TEAE   
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
SOC1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.3 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
SOC2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.3 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
…etc… 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at the start of AE. 
Percentages are based on the number of patients in each dose group and overall. 
Adverse Events were coded using MedDRA, Version 18.0. 
Adverse events where PT='Pemphigus' are excluded from this table. 
Adverse events are attributed to the dose level where the AE start date is on or after the start date of the dose level, but before the next dose level, if applicable. 
Treatment emergent adverse events are the events started on or after the first dose of study drug and prior to the date of last dose of study drug.  
Adverse events are sorted by overall descending frequency of SOC and within each SOC, by overall descending frequency of PT. 
Patients with multiple events within a particular SOC or PT will be counted once under the maximum intensity grade. 
Program: <program name (date, time)>, Path: <path> 
Programming note:  Repeat table for the 400 mg QD, 400 mg BID and 600 mg BID, Post Last Dose groups. 
 


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Table B.14.3.1.5 Related Treatment Emergent Adverse Events by Maximum Intensity, SOC and PT (Safety Population) 
 
Overall (N = n) 
System Organ Class (SOC) 
Grade 1 
Grade 2 
Grade 3 
Grade 4 
Grade 5 
     Preferred Term (PT) 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
Patients with at least one related TEAE   
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
SOC1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.3 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
SOC2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.3 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
…etc… 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at the start of AE. 
Percentages are based on the number of patients in each dose group and overall. 
Adverse Events were coded using MedDRA, Version 18.0. 
Adverse events where PT='Pemphigus' are excluded from this table. 
Adverse events are attributed to the dose level where the AE start date is on or after the start date of the dose level, but before the next dose level, if applicable. 
Treatment emergent adverse events are the events started on or after the first dose of study drug and prior to the date of last dose of study drug.  
Adverse events are sorted by overall descending frequency of SOC and within each SOC, by overall descending frequency of PT. 
Patients with multiple events within a particular SOC or PT will be counted once under the maximum intensity grade. 
Program: <program name (date, time)>, Path: <path> 
Programming note:  Repeat table for the 400 mg QD, 400 mg BID and 600 mg BID, Post Last Dose groups. 
 


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Table B.14.3.1.6 Serious Treatment Emergent Adverse Events by SOC and PT (Safety Population) 
System Organ Class (SOC) 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
400 mg BID 
(N = n) 
Post Last Doe 
(N = n) 
Overall 
(N = n) 
     Preferred Term (PT) 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
Patients with at least one SAE   
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
SOC1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.3 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
SOC2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.3 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
…etc… 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at the start of AE. 
Percentages are based on the number of patients in each dose group and overall. 
Adverse Events were coded using MedDRA, Version 18.0. 
Serious adverse events where PT='Pemphigus' are included in this table. 
Adverse events are attributed to the dose level where the AE start date is on or after the start date of the dose level, but before the next dose level, if applicable. 
Treatment emergent adverse events are the events started on or after the first dose of study drug and prior to the date of last dose of study drug.  
Adverse events are sorted by overall descending frequency of SOC and within each SOC, by overall descending frequency of PT. 
Program: <program name (date, time)>, Path: <path> 
 
 


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Table B.14.3.1.7 Treatment Emergent Adverse Events Leading to Study Drug Discontinuation by SOC and PT (Safety Population) 
System Organ Class (SOC) 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Post Last Dose 
(N = n) 
Overall 
(N = n) 
     Preferred Term (PT) 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
n (%) 
[E] 
Patients with at least one TEAE leading to study drug 
discontinuation 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
SOC1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT1.3 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
SOC2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.1 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.2 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
     PT2.3 
x (x.x)  
x 
x (x.x)  
x 
x (x.x) 
x 
x (x.x) 
x 
x (x.x) 
x 
 
 
 
 
 
 
 
 
 
 
 
…etc… 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at the start of AE. 
Percentages are based on the number of patients in each dose group and overall. 
Adverse Events were coded using MedDRA, Version 18.0. 
Adverse events where PT='Pemphigus' are excluded from this table. 
Adverse events are attributed to the dose level where the AE start date is on or after the start date of the dose level, but before the next dose level, if applicable. 
Treatment emergent adverse events are the events started on or after the first dose of study drug and prior to the date of last dose of study drug.  
Adverse events are sorted by overall descending frequency of SOC and within each SOC, by overall descending frequency of PT. 
Program: <program name (date, time)>, Path: <path> 
 
 


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Table B.14.3.2.1 Hematology – Actual Values and Change from Baseline by Study Visit (Safety Population) 
Parameter = Parameter x [unit] 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
SCREENED 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK1 DAY 1 (BASELINE)  
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 3 DAY 15 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 3 DAY 15 – CHANGE FROM BASELINE 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 


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     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
… etc… 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at a given visit. 
BASELINE: If Week 1 Day 1 is missing, then screening value is used. 
Program: <program name (date, time)>, Path: <path> 
 
 
 


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Table B.14.3.2.2 Hematology – Shift from Baseline by Study Visit (Safety Population) 
Parameter = Parameter x [unit] 
 
Overall (N = n) 
WEEK DAY 1 (BASELINE) 
Post-baseline  
N assessed 
Low 
Normal 
High 
WEEK 3 DAY 15 
n 
x (x.x) 
x (x.x) 
x (x.x) 
     Low 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Normal 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     High 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
WEEK 5 DAY 29 
n 
x (x.x) 
x (x.x) 
x (x.x) 
     Low 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Normal 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     High 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
… etc…  
 
 
 
 
 
 
 
 
 
BASELINE: If Week 1 Day 1 is missing, then screening value is used. 
Program: <program name (date, time)>, Path: <path> 
 
Programming notes: 
1) 
Repeat table for the 400 mg QD, 400 mg BID and 600 mg BID groups. 
2) 
Highlight Normal to Low/High shifts in yellow. Do not display 0 ( 0.0)% for zero count for shift. 
 
 


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Mock shells for Table B.14.3.2.1 to Table B.14.3.2.2 will be used for the following tables:  
Table B.14.3.3.1 Coagulation – Actual Values and Change from Baseline by Study Visit (Safety Population) 
Table B.14.3.3.2 Coagulation – Shift from Baseline by Study Visit (Safety Population) 
 
Table B.14.3.4.1 Serum Chemistry – Actual Values and Change from Baseline by Study Visit (Safety Population) 
Table B.14.3.4.2 Serum Chemistry – Shift from Baseline by Study Visit (Safety Population) 
 
 


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Table B.14.3.4.3 Serum Chemistry – Drug Induced Liver Injury (DILI) Criteria (Safety Population) 
Patient who met the DILI criteria during treatment 
period 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
AST  
 
 
 
 
     > 3x ULN 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     > 5x ULN 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     > 10x ULN 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     > 20x ULN 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
ALT  
 
 
 
 
     > 3x ULN 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     > 5x ULN 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     > 10x ULN 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     > 20x ULN 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
TBL 
 
 
 
 
     > 1.5x ULN 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     > 2x ULN 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
ALP > 1.5x ULN 
…etc.. 
 
 
 
 
 
 
 
 
ALT or AST > 3x ULN and Total Bilirubin > 1.5x ULN 
…etc.. 
 
 
 
 
 
 
 
 
ALT or AST > 3x ULN and Total Bilirubin > 2x ULN 
…etc.. 
 
 
 
ULN = Upper Limit of Normal 
Patients are classified into different dose levels according to the dose they received at the time a given DILI criteria was met. 
Program: <program name (date, time)>, Path: <path> 
 


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Mock shells for Table B.14.3.2.1 to Table B.14.3.2.2 will be used for the following tables: 
 
Table B.14.3.5.1 Urinalysis – Actual Values and Change from Baseline by Study Visit (Safety Population) 
Table B.14.3.5.2 Urinalysis – Shift from Baseline by Study Visit (Safety Population) 
 
 
 
 


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Table B.14.3.6.1 Vital Signs and Body Weight – Actual Values and Change from Baseline by Study Visit (Safety Population) 
Parameter = Parameter x [unit] 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
SCREENED 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 1 DAY 1 (BASELINE) 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 1 DAY 2 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
WEEK 1 DAY 2 – CHANGE FROM BASELINE 
 
 
 
 
    n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  


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… etc…  
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at a given visit. 
BASELINE: If Week 1 Day 1 is missing, then screening value is used. 
Program: <program name (date, time)>, Path: <path> 
 
 
 
Table B.14.3.6.2 Vital Signs – BP and Pulse – Shift from Baseline by Study Visit (Safety Population) 
Parameter = Parameter x [unit] 
 
Overall (N = n) 
WEEK 1 DAY 1 (BASELINE)  
Post-baseline  
N assessed 
Low 
Normal 
High 
WEEK 1 DAY 2  
n 
x (x.x) 
x (x.x) 
x (x.x) 
     Low 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Normal 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     High 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
WEEK 3 DAY 15  
n 
x (x.x) 
x (x.x) 
x (x.x) 
     Low 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Normal 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     High 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
… etc…  
 
 
 
 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at a given visit. 
BASELINE: If Week 1 Day 1 is missing, then screening value is used. 
Program: <program name (date, time)>, Path: <path> 
 


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Programming notes: 
• 
Repeat table for the 400 mg QD,400 mg BID and 600 mg BID groups.  
• 
Highlight Normal to Low/High shifts in yellow. Do not display 0 ( 0.0)% for zero count for shifts. 
 
 


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Table B.14.3.7.1 12-Lead ECG Results – Actual Values and Change from Baseline (Safety Population) 
Parameter = Parameter x [unit] 
 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
SCREENED (BASELINE) 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
LAST VISIT* 
 
 
 
 
     n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
LAST VISIT* – CHANGE FROM BASELINE  
 
 
 
 
    n 
x 
x 
x 
x 
     Mean (SD) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
x.xx (x.xxx) 
     Median 
x.xx 
x.xx 
x.xx 
x.xx 
     Min, Max 
x.x, x.x  
x.x, x.x  
x.x, x.x  
x.x, x.x  
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at a given visit. 
*LAST VISIT is the last post-baseline visit. 
Program: <program name (date, time)>, Path: <path> 
 
 
 


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Table B.14.3.8.3 12-Lead ECG – Overall Assessment – Shift from Baseline to Last Post-Baseline Visit (Safety Population) 
Parameter = Parameter x [unit] 
 
Overall (N = n) 
SCREENED (BASELINE) 
Post-baseline  
N assessed 
Low 
Normal 
High 
LAST VISIT   
n 
x (x.x) 
x (x.x) 
x (x.x) 
     Low 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     Normal 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
     High 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
Patients are classified into different dose levels according to the dose they received at a given visit. 
Program: <program name (date, time)>, Path: <path> 
 
Programming notes: 
• 
Repeat table for the 400 mg QD, 400 mg BID and 600 mg BID. 
• 
Highlight Normal to Low/High shifts in yellow. Do not display 0 ( 0.0)% for zero count for shifts. 
 
 


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Table B.14.3.9.1 Abbreviated Physical Examination by Study Visit (Safety Population) 
Body System 
Study Visit 
N attended visit/ 
   Result 
400 mg QD 
(N = n) 
400 mg BID 
(N = n) 
600 mg BID 
(N = n) 
Overall 
(N = n) 
General Appearance 
SCREENED 
N attended visit 
x 
x 
x 
x 
 
 
   Normal 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
   Abnormal NCS 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
   Abnormal CS 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
   Not None 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
 
 
WEEK 1 DAY 1 
N attended visit 
x 
x 
x 
x 
 
 
   Normal 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
   Abnormal NCS 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
   Abnormal CS 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
   Not None 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
 
 
WEEK 3 DAY 15 
N attended visit 
x 
x 
x 
x 
 
 
   Normal 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
   Abnormal NCS 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
   Abnormal CS 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
   Not None 
x (x.x) 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
 
 
 
…etc… 
 
 
 
 
 
 
 
 
 
 
 
 
 
The full physical examination was done at Screening visit. Abbreviated Physical Examination was consists of checking the normality or abnormality of the following body 
systems: general appearance, abdomen, cardiorespiratory and other was done at Day 1, Week 1 and at each visit from Day 15, Week 3 onward. 
Patients are classified into different dose levels according to the dose they received at a given visit. 
Percentages are based on the number of patients in each dose group and overall. 
Program: <program name (date, time)>, Path: <path> 
 


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Listing B.16.2.1.1  
Patient Disposition (Screening Population) 
Starting 
Dose 
Country 
Patient 
ID 
Age/ 
Gender 
Date of First 
Dose 
(ddMMMyyyy) 
Initial Dose 
Maximum 
Dose 
Completed 
Study? 
Date of 
Completion/ 
Withdrawal 
(ddMMMyyyy) 
Last 
Visit 
Reason for 
Study 
Discontinuation 
400 mg 
QD 
xxx 
xxxxxxx 
xx/F 
ddMMMyyyy 
400 mg QD  
400 mg BID 
No 
ddMMMyyyy 
DXWX 
xxxx 
 
 
 
 
 
 
 
 
 
 
 
400 mg 
BID 
xxx 
xxxxxxx 
xx/M 
ddMMMyyyy 
400 mg BID  
600 mg BID 
Yes 
ddMMMyyyy 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


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Listing B.16.2.1.2  
Patient Enrollment (Screening Population) 
Startin
g Dose 
Countr
y 
Patient 
ID 
Age/Gende
r 
Date and 
time of 
Informed 
Consent 
(ddMMMyyy
y hh:mm 
24hr) 
Protoco
l 
Version 
Does the 
subject 
response 
to the 
question 
for 
opting in 
or out 
allow for 
the use 
of their 
clinical 
data in 
future 
research
? 
Does the 
subject 
response 
to the 
question 
for 
opting in 
or out of 
having 
pictures 
taken for 
the 
purpose 
of the 
study 
and use 
by the 
sponsor 
in future 
research
? 
Is the 
patient 
consenting 
to enroll into 
A New 
Protocol 
Amendment
? 
French sites only 
Is the 
patient 
enrolling 
into a 
French site 
under 
French 
Protocol 
amendmen
t V4.1-12 
May 2017? 
Is the 
patient 
consenting 
to enroll into 
a French site 
under A New 
Protocol 
Amendment
? 
If the 
patient is 
enrolling 
into a 
French site 
are 
additional 
MRI and 
Neurologica
l forms 
required? 
400 mg 
QD 
xxx 
xxxxxx
x 
xx/F 
ddMMMyyyy 
hh:mm 
Version 
x.x 
Y/N 
Y/N 
Y/N 
Y/N 
Y/N 
Y/N 
 
 
 
 
 
 
 
 
 
 
 
 
400 mg 
BID 
xxx 
xxxxxx
x 
xx/M 
ddMMMyyyy 
hh:mm 
Version 
x.x 
Y/N 
Y/N 
Y/N 
Y/N 
Y/N 
Y/N 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


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Listing B.16.2.1.3  
Screen Failure (All Screen Failures) 
Country 
Patient ID 
Age/Gender 
Did the subject meet all 
eligibility criteria? 
Category of failed criteria 
Inclusion/Exclusion Criterion Short Name 
xxx 
xxxxxxx 
xx/F 
No 
Inclusion Criteria 
INCxxx 
 
 
 
 
 
 
xxx 
xxxxxxx 
xx/M 
No 
Exclusion Criteria 
EXCxxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


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Listing B.16.2.1.4  
Analysis Population (Screening Population) 
Starting Dose 
Country 
Patient 
ID 
Age/Gender 
Analysis Populations 
Screening  Safety 
Efficacy Analysis  
(Intent-to-Treat) (ITT) 
Modified Intent-to-
Treat (mITT) 
Completer  
Per-Protocol 
(PP) 
400 mg QD 
xxx 
xxxxxxx 
xx/F 
Yes 
Yes 
Yes 
Yes 
Yes 
Yes 
 
 
 
 
 
 
 
 
 
 
400 mg BID 
xxx 
xxxxxxx 
xx/M 
Yes 
No 
Yes 
Yes 
No 
No 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
The Screening population consists of all patients who provided informed consent and have screening assessments in Part B evaluated for study participation. 
The Safety population consists of all patients who have received at least one dose of PRN1008 in Part B. 
The ITT population consists of all patients who have received at least one dose of PRN1008 in Part B. 
The mITT population consists of all patients treated up to Week 5 and had a Week 5 disease assessment.  
The Completer population consists of all patients treated up to Week 25and had a Week 29 disease assessment. 
The PP population consists of all patients who have: 1) No major protocol deviations relevant to data integrity per Medical Monitor review; 2) At least 80% compliance based 
on drug accountability; and 3) Completed Week 29visit. 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Listing B.16.2.1.5  
Demographics and Baseline Characteristics (ITT Population) 
Starting 
Dose 
Country 
Patient 
ID 
Birth Date 
(ddMMMyyyy) 
Age 
(years) 
Sex 
If female 
Ethnicity 
Race 
At Screening 
Fertility 
status 
Method of 
birth 
control 
Height 
(cm) 
Weight 
(kg) 
BMI 
(kg/m2) 
400 mg 
QD 
xxx 
xxxxxxx 
xxxx 
xx 
Female 
xxxx 
Other - xxx 
xxx 
xxx 
xxx 
xx.x 
xx.x 
 
 
 
 
 
 
 
 
 
 
 
 
 
400 mg 
BID 
xxx 
xxxxxxx 
xxxx 
xx 
Male 
 
 
xxx 
xxx 
xxx 
xx.x 
xx. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Body Mass Index (BMI) = (Weight (kg) at Screening)/(Height (m) at Screening)2 
Program: <program name (date, time)>, Path: <path> 
 
 


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Listing B.16.2.1.6  
Disease History – Pemphigus history and Anti-DSG at Screening (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/Gender 
Pemphigus 
subtype  
Pemphigus Vulgaris 
Pemphigus history 
type [1] 
Anti-DSG status 
Date Confirmed 
Time to Screening 
(months) 
400 mg 
QD 
xxxxxxx 
xx/F 
xxx 
ddmmmyyyy 
x.x months 
Newly diagnosed / 
Relapsed 
Anti-DSG 1 xxx, Anti-DSG 3 xxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
[1] Newly diagnosed is defined as subjects who were diagnosed at most 6 months prior to date of screening, otherwise the subject is relapsed. 
Program: <program name (date, time)>, Path: <path> 
 
 


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Listing B.16.2.1.7  
Disease History – Baseline PDAI and ABSIS Score (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/Gender 
Total 
Activity 
Score 
Total 
Damage 
Score 
Pemphigus 
Severity* 
PDAI Skin 
Activity 
PDAI 
Scalp 
Activity 
PDAI 
Mucous 
Membrane 
Activity 
ABSIS Oral 
Activity 
ABSIS Skin 
Involvement 
ABSIS Oral 
Involvement 
400 mg 
QD 
xxxxxxx 
xx/F 
xx 
xx 
xx 
xx 
xx 
xx 
xx 
xx 
xx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
*Mild = PDAI Activity < 15; Moderate = PDAI Activity ≥ 15 
F=Female; M=Male 
 
 


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Listing B.16.2.1.8  
Medical History (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/Gender 
Any medical 
History to 
report? 
Body System 
Verbatim Term 
Start Date (ddMMMyyyy) 
End Date (ddMMMyyyy) 
/Ongoing 
400 mg 
QD 
xxxxxxx 
xx/F 
Yes 
xxx 
xxx 
ddMMMyyyy 
ddMMMyyyy 
 
 
 
 
 
 
 
 
400 mg 
BID 
xxxxxxx 
xx/M 
No 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Protocol: PRN1008-005 (Part B)  
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Listing B.16.2.1.9  
Prior Corticosteroid Medications (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Any Prior 
corticosteroid 
medication? 
Reported Medication (R) 
ATC Level 2 Class (Level 2) 
Preferred Name/Term (PT) 
Start Date (ddMMMyyyy) (Study 
Day) 
Stop Date (ddMMMyyyy) (Study 
Day) 
/Ongoing 
Indication:  
AE or MH No.: 
Dose: 
Dose unit: 
Frequency 
Route: 
Rescue  
criteria 
400 mg 
QD 
xxxxxxx 
xx/F 
Yes 
R: xxx 
Level 2: xxx 
PT: xxxx 
Start Date: ddMMMyyyy (Day x)  
Stop Date ddMMMyyyy(Day x) 
Indication: xxx 
AE or MH No: AE #1 
Dose: xx  
Dose unit: mg 
Frequency: xxx 
Route: xxx 
Not applicable 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Medications are coded using WHODD, version 01SEP2015. 
Prior corticosteroid medications are those corticosteroid mediations that have stopped prior to the first dose of study drug.  
If Date >= first dose date, then Study Day =  (Date - Date of first dose +1). If Date < first dose date, then Study Day = (Date - Date of first dose). 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
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Listing B.16.2.1.10  
Concomitant Corticosteroid Medications (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Any 
corticosteroid 
medication? 
PRN Dose 
Level at 
Medication 
Start 
Reported Medication (R) 
ATC Level 2 Class (Level 
2) 
Preferred Name/Term 
(PT) 
Start Date 
(ddMMMyyyy) (Study 
Day) 
Stop Date (ddMMMyyyy) 
(Study Day) 
/Ongoing 
Indication:  
AE or MH No.: 
Dose: 
Dose unit: 
Frequency 
Route: 
Rescue  
criteria 
400 mg 
QD 
xxxxxxx 
xx/F 
Yes 
400 mg QD 
R: xxx 
Level 2: xxx 
PT: xxxx 
Start Date: ddMMMyyyy 
(Day x) 
Stop Date: ddMMMyyyy 
(Day x) 
Indication: xxx 
AE or MH No: AE #1 
Dose: xx  
Dose unit: mg 
Frequency: xxx 
Route: xxx 
Not 
applicable 
 
 
 
 
600 mg BID 
R: xxx 
Level 2: xxx 
PT: xxxx 
Start Date: ddMMMyyyy 
(Day x) 
Stop Date: Ongoing 
Indication: xxx 
Treatment for an 
AE?: No 
Dose: xx mg 
Frequency: xxx 
Route: xxx 
xxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Medications are coded using WHODD, version 01SEP2015. 
This table includes concomitant corticosteroid medications that are ongoing or stopped on or after the date of first dose of study drug and started prior to date of last dose of 
study drug. 
If Date >= first dose date, then Study Day = (Date - Date of first dose +1). If Date < first dose date, then Study Day = (Date - Date of first dose). 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
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Listing B.16.2.1.11  
Concomitant Corticosteroid Medications Started Post Last Dose (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Any Post Last 
Dose 
Corticosteroid 
med? 
PRN 
Dose 
Level 
Prior to 
Last 
Dose of 
study 
drug 
Reported Medication (R) 
ATC Level 2 Class (Level 2) 
Preferred Name/Term 
(PT) 
Start Date (ddMMMyyyy) 
(Study Day) 
Stop Date (ddMMMyyyy) 
(Study Day) 
/Ongoing 
Indication:  
AE or MH No.: 
Dose: 
Dose unit: 
Frequency 
Route: 
Rescue  
criteria 
400 mg 
QD 
xxxxxxx 
xx/F 
 
600 mg 
BID 
R: xxx 
Level 2: xxx 
PT: xxxx 
Start Date: ddMMMyyyy 
(Day x) 
Stop Date: ddMMMyyyy 
(Day x) 
Indication: xxx 
AE or MH No: AE #1 
Dose: xx  
Dose unit: mg 
Frequency: xxx 
Route: xxx 
Not 
applicable 
 
 
 
 
600 mg 
BID 
R: xxx 
Level 2: xxx 
PT: xxxx 
Start Date: ddMMMyyyy 
(Day x) 
Stop Date: Ongoing 
Indication: xxx 
AE or MH No: MH #3 
Dose: xx  
Dose unit: mg 
Frequency: xxx 
Route: xxx 
No 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Medications are coded using WHODD, version 01SEP2015. 
This table includes patients entered into follow up period and corticosteroid medications started after the date of last dose of study drug. 
If Date >= first dose date, then Study Day = (Date - Date of first dose +1). If Date < first dose date, then Study Day = (Date - Date of first dose). 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
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Listing B.16.2.1.11  
Rescue Medications Taken During Study (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Any 
corticosteroid 
medication? 
PRN Dose 
Level at 
Medication 
Start 
Reported Medication 
(R) 
ATC Level 2 Class 
(Level 2) 
Preferred Name/Term 
(PT) 
Start Date 
(ddMMMyyyy) (Study 
Day) 
Stop Date 
(ddMMMyyyy) (Study 
Day) 
/Ongoing 
Indication:  
AE or MH No.: 
Dose: 
Dose unit: 
Frequency 
Route: 
Rescue  
criteria 
400 mg 
QD 
xxxxxxx 
xx/F 
Yes 
400 mg QD 
R: xxx 
Level 2: xxx 
PT: xxxx 
Start Date: 
ddMMMyyyy (Day) 
Stop Date: 
ddMMMyyyy (Day x) 
Indication: xxx 
AE or MH No: AE #1 
Dose: xx  
Dose unit: mg 
Frequency: xxx 
Route: xxx 
Rescue Criteria 
#1 
400 mg 
BID 
xxxxxxx 
xx/M 
Yes 
600 mg 
BID 
R: xxx 
Level 2: xxx 
PT: xxxx 
Start Date: 
ddMMMyyyy (Day x) 
Stop Date: Ongoing 
Indication: xxx 
AE or MH No: MH #3 
Dose: xx  
Dose unit: mg 
Frequency: xxx 
Route: xxx 
Rescue Criteria 
#2 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Medications are coded using WHODD, version 01SEP2015. 
This listing includes rescued corticosteroid medications reported as “Ongoing” or stopped on or after the date of first study drug dosing. 
If Date >= first dose date, then Study Day = (Date - Date of first dose +1). If Date < first dose date, then Study Day = (Date - Date of first dose). 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
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Listing B.16.2.1.12  
Prior Medications other than Corticosteroid Medications (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Any 
medication 
taken? 
Reported Medication (R) 
ATC Level 2 Class (Level 2) 
Preferred Name/Term (PT) 
Start Date (ddMMMyyyy) (Study Day) 
Stop Date (ddMMMyyyy) (Study Day) 
/Ongoing 
Indication:  
AE or MH No.: 
Dose: 
Dose unit: 
Frequency 
Route: 
400 mg 
QD 
xxxxxxx 
xx/F 
Yes 
R: xxx 
Level 2: xxx 
PT: xxxx 
Start Date: ddMMMyyyy (Day x) 
Stop Date: ddMMMyyyy (Day x) 
Indication: xxx 
AE or MH No: MH #3 
Dose: xx  
Dose unit: mg 
Frequency: xxx 
Route: xxx 
 
 
 
 
R: xxx 
Level 2: xxx 
PT: xxxx 
Start Date: ddMMMyyyy (Day x) 
Stop Date: Ongoing 
Indication: xxx 
AE or MH No: AE #3 
Dose: xx  
Dose unit: mg 
Frequency: xxx 
Route: xxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Medications are coded using WHODD, version 01SEP2015. 
Prior medications are those non-corticosteroid mediations that have stopped prior to the first dose of study drug.  
If Date >= first dose date, then Study Day = (Date - Date of first dose +1). If Date < first dose date, then Study Day = (Date - Date of first dose). 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 136 of 171 
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A7.10-F1_v3_SAP Template_2016-08-29 
Listing B.16.2.1.13  
Concomitant Medications other than Corticosteroid Medications (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Any 
medication 
taken? 
PRN Dose 
Level at 
Medication 
Start 
Reported Medication (R) 
ATC Level 2 Class (Level 
2) 
Preferred Name/Term 
(PT) 
Start Date (ddMMMyyyy) (Study 
Day) 
Stop Date (ddMMMyyyy) (Study 
Day) 
/Ongoing 
Indication:  
AE or MH No.: 
Dose: 
Dose unit: 
Frequency 
Route: 
400 mg 
QD 
xxxxxxx 
xx/F 
Yes 
400 mg QD 
R: xxx 
Level 2: xxx 
PT: xxxx 
Start Date: ddMMMyyyy (Day x) 
Stop Date: ddMMMyyyy (Day x) 
Indication: xxx 
AE or MH No: MH #3 
Dose: xx  
Dose unit: mg 
Frequency: xxx 
Route: xxx 
 
 
 
 
600 mg BID 
R: xxx 
Level 2: xxx 
PT: xxxx 
Start Date: ddMMMyyyy (Day x) 
Stop Date: Ongoing 
Indication: xxx 
AE or MH No: AE #3 
Dose: xx  
Dose unit: mg 
Frequency: xxx 
Route: xxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Medications are coded using WHODD, version 01SEP2015. 
Concomitant medications are those non-corticosteroid mediations that are ongoing or stopped on or after the date of first dose of study drug. 
If Date >= first dose date, then Study Day = (Date - Date of first dose +1). If Date < first dose date, then Study Day = (Date - Date of first dose). 
Program: <program name (date, time)>, Path: <path> 
 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 137 of 171 
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Listing B.16.2.1.14  
Protocol Deviations (ITT Population) 
Starting 
Dose 
Patient ID 
Age/Gender 
Major or Minor 
Protocol Deviation 
Category 
Details 
400 mg QD 
xxxxxxx 
xx/F 
Minor 
Visit Schedule 
xxx 
 
 
 
 
 
 
400 mg QD 
xxxxxxx 
xx/M 
Major 
Concomitant Medication 
xxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
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Listing B.16.2.5.1  
Study Drug Administration (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Dose 
Start 
Date 
and 
Time 
(ddMM
Myyyy 
hh:mm 
24hr) 
Was dose 
admin on 
PK sample 
day? 
Date and 
Time of 
current 
dose 
received 
(ddMM
Myyyy 
hh:mm 
24hr) 
Dose: 
Route: 
Freque
ncy 
Dose 
Missed
? 
Date 
and 
Time 
Dose 
Missed 
(ddM
MMyy
yy 
hh:mm 
24hr) 
Reason 
Dose 
Missed 
Dose 
Restart
ed? 
Date 
Dose 
Restarted 
(ddMMM
yyyy) 
Reason if 
dose 
adjusted 
Adjusted 
dose 
Dose 
Stop 
date 
(ddM
MMyy
yy) 
400 mg 
QD 
xxxxxxx 
xx/F 
Dx, Wx 
ddMM
Myyyy 
hh:mm 
Yes or No 
ddMMM
yyyy 
hh:mm 
 
 
 
 
 
 
 
 
 
 
 
Dx, Wx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Dx, Wx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Dx, Wx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Protocol: PRN1008-005 (Part B)  
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Listing B.16.2.5.2  
Study Drug Exposure and Compliance (ITT Population) 
Starting 
Dose 
Patient ID 
Age/Gender 
PRN Dose 
Level 
Date of First Dose 
(ddMMMyyyy) 
Date of Last 
Dose 
(ddMMMyyyy) 
Duration 
of 
Exposure 
(day) 
Number 
of 
Missed 
doses 
Calculated dose 
(mg/day) 
Compliance 
(%) 
400 mg QD 
xxxxxxx 
xx/F 
400 mg QD 
ddMMMyyyy 
 ddMMMyyyy 
x days 
x 
xxx mg/day 
xxx % 
 
 
 
400 mg QD 
ddMMMyyyy 
 ddMMMyyyy 
x days 
x 
xxx mg/day 
xxx % 
 
 
 
Overall 
ddMMMyyyy  
ddMMMyyyy 
x days 
x 
xxx mg/day 
xxx % 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Calculated dose in mg/day at each dose level = [Dose level (mg/day) x (Duration of exposure (x2 if BID) – doses missed)]/Duration of Exposure. 
Compliance with study drug = the actual number of doses taken divided by the expected number of doses take, expressed as a percentage. 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 140 of 171 
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Listing B.16.2.6.1  
Disease Assessment (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Current PRN 
Dose 
Control of Disease 
Activity (CDA) Status 
Achieved at this visit 
If Yes, Date 
(ddMMMyyyy) 
Complete Remission 
(CR) Status Achieved 
at this visit 
If Yes, Date 
(ddMMMyyyy) 
End of Consolidated 
Phase Status 
Achieved at this visit 
If Yes, Date 
(ddMMMyyyy) 
Relapse Status 
Achieved at this visit 
If Yes, Date 
(ddMMMyyyy) 
400 mg 
QD 
xxxxxxx 
xx/F 
xxx 
400 mg QD 
No 
No 
No 
No 
 
 
 
xxx 
400 mg QD 
Yes – ddMMMyyyy 
No 
No 
No 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 141 of 171 
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A7.10-F1_v3_SAP Template_2016-08-29 
Listing B.16.2.6.2  
Pemphigus Disease Area Index (PDAI) - Skin (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Current 
Dose 
Date if PDAI 
(ddMMMMyyyy) 
Total 
Skin 
(/120) – 
Activity 
Total 
Skin 
(/12) – 
Damage 
Anatomical 
Location 
Erosion /blister 
or new 
erythema 
Number 
Lesion 
if <=3 
Post-
inflammato
ry 
hyperpigm
entation or 
erythema 
from 
resolving 
lesions 
400 mg 
QD 
xxxxxxx 
xx/F 
xxx 
400 mg 
QD 
xxxx 
xx 
xx 
EARS 
xx 
xx 
Absent 
 
 
 
 
 
 
 
 
NOSE 
xx 
xx 
Absent 
 
 
 
 
 
 
 
 
REST OF 
THE FACE 
xx 
xx 
Present 
 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
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Listing B.16.2.6.3  
Pemphigus Disease Area Index (PDAI) - Scalp (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Current 
PRN Dose 
Date if PDAI 
(ddMMMMyyyy) 
Total 
Scalp 
(/10) – 
Activity 
Total 
Scalp 
(/1) – 
Damage 
Anatomical 
Location 
Erosion /blister 
or new 
erythema 
Number 
Lesion 
if <=3 
Post-
inflammato
ry 
hyperpigm
entation or 
erythema 
from 
resolving 
lesions 
400 mg 
QD 
xxxxxxx 
xx/F 
xxx 
400 mg 
QD 
xxxx 
xx 
xx 
SCALP 
xx 
xx 
Absent 
 
 
 
xxx 
400 mg 
QD 
xxxx 
xx 
xx 
SCALP 
xx 
xx 
Absent 
 
 
 
xxx 
400 mg 
QD 
xxxx 
xx 
xx 
SCALP 
xx 
xx 
Present 
 
 
 
 
 
 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 143 of 171 
SCiAN Services Inc. 
A7.10-F1_v3_SAP Template_2016-08-29 
 
 
Listing B.16.2.6.4  
Pemphigus Disease Area Index (PDAI) – Mucous Membrane (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Current 
PRN Dose 
Date if PDAI 
(ddMMMMyyyy) 
Total 
Mucosa 
(/120) – 
Activity 
Anatomical 
Location 
Erosion/blister  Number Lesion if <=3 
400 mg 
QD 
xxxxxxx 
xx/F 
xxx 
400 mg 
QD 
xxxx 
xx 
EYES 
xx 
xx 
 
 
 
 
 
 
 
NOSE 
xx 
xx 
 
 
 
 
 
 
 
BUCCAL MUCOSA 
xx 
xx 
 
 
 
 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


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Listing B.16.2.6.5  
Pemphigus Disease Area Index (PDAI) – Total Scores (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Current PRN Dose 
PDAI Total 
Activity 
Score 
PDAI Total 
Damage 
Score 
Total Skin 
(/120) – 
Activity 
Total Skin 
(/12) – 
Damage 
Total 
Scalp (/10) 
– Activity 
Total 
Scalp (/1) 
– Damage 
Total 
Mucosa 
(/120) – 
Activity 
400 mg 
QD 
xxxxxxx 
xx/F 
xxx 
400 mg QD 
xx 
xx 
xx 
xx 
xx 
xx 
xx 
 
 
 
xxx 
400 mg QD 
xx 
xx 
xx 
xx 
xx 
xx 
xx 
 
 
 
xxx 
400 mg QD 
xx 
xx 
xx 
xx 
xx 
xx 
xx 
 
 
 
 
 
 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


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Listing B.16.2.6.6  
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) - Skin and Oral Involvement (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Current PRN Dose 
Date 
(ddMMMy
yyy) 
Skin  
Oral  
Patient’s 
weight 
(unit) 
Skin 
Involvement 
(Max BSA) 
Patient’s 
BSA 
Weighting 
factor 
 
Oral 
Involvement 
Lesion 
Presences 
or 
Absence 
400 mg 
QD 
xxxxxxx 
xx/F 
xxx 
400 mg QD 
ddMMMyy
yy 
xx 
xx 
xx 
xx 
xx 
xx 
 
 
 
xxx 
400 mg QD 
xx 
xx 
xx 
xx 
xx 
xx 
xx 
 
 
 
xxx 
400 mg QD 
xx 
xx 
xx 
xx 
xx 
xx 
xx 
 
 
 
 
 
 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


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Listing B.16.2.6.7  
Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL) (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Current PRN Dose 
Date 
completed 
(ddMMMy
yyy) 
Time take 
to finish 
the survey 
Total 
Score 
Question 
Answer 
400 mg 
QD 
xxxxxxx 
xx/F 
xxx 
400 mg QD 
ddMMMy
yyy 
x min x 
secs 
xxx 
1. As a result of your blistering disease 
treatment, do you notice your bruise or 
bleed easily 
I notice this all 
the time 
 
 
 
 
 
 
 
 
2. As a result of your blistering disease 
treatment, can you still tolerate hot or 
cold temperatures 
I have not had 
this problem 
 
 
 
 
 
 
 
 
3. xxx 
xxxx 
 
 
 
 
 
 
 
 
…etc.. 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


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A7.10-F1_v3_SAP Template_2016-08-29 
 
 
Listing B.16.2.6.8  
Simple Nutritional Appetite Questionnaire (SNAQ) (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Current 
PRN Dose 
Date completed 
(ddMMMyyyy) 
Total Score 
Question 
Answer 
400 mg 
QD 
xxxxxxx 
xx/F 
xxx 
400 mg QD 
ddMMMyyyy 
xxx 
My appetite is 
Good 
 
 
 
 
 
 
 
When I eat 
I hardly ever feel full 
 
 
 
 
 
 
 
xxx 
xxxx 
 
 
 
 
 
 
 
…etc.. 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


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A7.10-F1_v3_SAP Template_2016-08-29 
 
 
Listing B.16.2.6.9  
Corticosteroid (CS) Usage (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Current 
PRN 
Dose 
Total 
Prednisone 
dosage 
equivalent 
(mg/kg) 
 
Weight 
(kg) 
Total 
Prednisone 
dosage 
equivalent 
(mg) 
Reported 
Medication (R) 
ATC Level 2 Class  
Preferred 
Name/Term (PT) 
Start Date 
(ddMMMyyyy) 
(Study Day) 
Stop Date 
(ddMMMyyyy) 
(Study Day) 
/Ongoing 
Indication:  
AE or MH 
No.: 
Dose: (Pred. 
equiv. dose) 
Dose unit: 
Frequency 
Route: 
Rescue  
criteria 
400 mg 
QD 
xxxxxxx 
xx/F 
xxx 
400 mg 
QD 
xx.xx 
xx.x 
xx 
xxx 
xxx 
xxx 
ddMMMyyyy 
ddMMMyyyy 
 
Indication: 
xxx 
AE or MH No: 
AE #1 
Dose: 10 
(12.5) 
Dose unit: mg 
Frequency: 
xxx 
Route: xxx 
Not 
applicable 
 
 
 
 
 
 
 
xx 
xxx 
xxx 
xxx 
ddMMMyyyy 
Ongoing 
Indication: 
xxx 
AE or MH No: 
AE #1 
Dose: xx  
Dose unit: mg 
Frequency: 
xxx 
Route: xxx 
Not 
applicable 
 
 
 
 
 
 
 
xx 
xxx 
xxx 
xxx 
ddMMMyyyy 
Ongoing 
…etc… 
 


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etc. 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 
Listing B.16.2.6.10  
Corticosteroid (CS) Usage on the Day prior to each Visit and CDA/CR Response (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Current PRN 
Dose 
Previous PRN 
Dose 
Prednisone dosage 
equivalent 
(mg/day)*  
Prednisone dosage 
equivalent 
(mg/kg/day)* 
CDA? 
CR? 
400 mg 
QD 
xxxxxxx 
xx/F 
SCREENED 
--- 
--- 
Xx 
xx 
No 
No 
 
 
 
Week 1 DAY 1 
400 mg QD 
--- 
Xx 
xx 
No 
No 
 
 
 
 
 
 
 
 
 
 
 
 
 
WEEK 3 DAY 
15 
400 mg BID 
400 mg QD 
Xx 
xx 
Yes 
No 
 
 
 
etc. 
 
 
 
 
 
 
F=Female; M=Male 
*For SCREENED and WEEK 1 DAY1: The dosage level on the day of the visit date.  For the post-baseline visits: The dosage level at each visit is defined as the dose level the 
patient is on the day before the visit date. 
Program: <program name (date, time)>, Path: <path> 
 
 


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Listing B.16.2.7.1  
Pre-treatment Adverse Events (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
SOC: System 
Organ Class 
PT: Preferred 
Term 
V: Verbatim 
Start Date 
(ddMMMyyyy) (Study 
Day) 
Stop Date 
(ddMMMyyyy) (Study 
Day) or Ongoing 
Duration (days) (if not 
ongoing)  
Pattern of AE 
(Continuous or 
Intermittent) 
SAE? (Y/N) 
 
CTCAE 
Grade 
 
Relationship 
to Study 
Drug 
Details 
400 mg 
QD 
xxxxxxx 
xx/F 
SOC: xxx 
PT: xxx 
V: xxx 
Start: ddMMMyyyy 
(Day X) 
Stop: ddMMMyyyy 
(Day X) 
Duration: x days 
Pattern of AE: xxx 
Y 
  
Grade x 
xxxx 
Action Taken to study 
drug: xxx 
Outcome: xxx 
Has the Cause of the AE 
been established: Y - xxx 
Caused study 
discontinuation?: N or Y 
Treated for this AE?: Y or 
N (AE description) 
 
 
 
SOC: xxx 
PT: xxx 
V: xxx 
Start: ddMMMyyyy 
(Day X) 
Stop: ddMMMyyyy 
(Day X) 
Duration: x days 
Pattern of AE: xxx 
N 
 
  
Grade x 
xxxx 
Action Taken to study 
drug: xxx 
Outcome: xxx 
Has the Cause of the AE 
been established: Y - xxx 
Caused study 
discontinuation?: N or Y 
Treated for this AE?: Y or 
N (AE description) 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Adverse Events were coded using MedDRA, Version 18.0. 


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Adverse events where PT='Pemphigus' are excluded from this listing. 
Pre-treatment adverse events are the events that started prior to the first dose. 
Study Day: If Date >= first dose date, then Study Day = (Date - Date of first dose +1). If Date < first dose date, then Study Day = (Date - Date of first dose). 
Program: <program name (date, time)>, Path: <path> 
 
Programming note: For all AE listings, sort by cohort, patient, start date, stop date, SOC and PT. 
 
Same listing shell will be used for the following listing:  
Listing B.16.2.7.2  
Pre-treatment Serious Adverse Events (ITT Population) 
Programming note: The following variables will be added to the “SAE” column for the SAE listing: Start Date (ddMMMyyyy) (Study Day), Stop Date (ddMMMyyyy) (Study 
Day) or Ongoing, Duration (if not ongoing), SAE Criteria 
 
 


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Listing B.16.2.7.3  
Treatment Emergent Adverse Events (Safety Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
PRN 
Dose 
Level 
at AE 
start  
SOC: System 
Organ Class 
PT: Preferred 
Term 
V: Verbatim 
Start Date (ddMMMyyyy) (Study 
Day) 
Stop Date (ddMMMyyyy) (Study 
Day) or Ongoing 
Duration (if not ongoing) 
Pattern of AE (Continuous or 
Intermittent) 
SAE? 
(Y/N) 
CTCAE 
Grade 
 
Relationship 
to Study 
Drug 
Details 
400 mg 
QD 
xxxxxxx 
xx/F 
400 
mg 
QD 
SOC: xxx 
PT: xxx 
V: xxx 
Start: ddMMMyyyy (Day X) 
Stop: ddMMMyyyy (Day X) 
Duration: x days 
Pattern of AE: xxx 
Y 
 
Grade x 
xxxx 
Action Taken to study 
drug: xxx 
Outcome: xxx 
Has the Cause of the AE 
been established: Y - xxx 
Caused study 
discontinuation?: N or Y 
Treated for this AE?: Y or 
N (AE description) 
 
 
 
 
SOC: xxx 
PT: xxx 
V: xxx 
Start: ddMMMyyyy (Day X) 
Stop: ddMMMyyyy (Day X) 
Duration: x days 
Pattern of AE: xxx 
N 
 
  
Grade x 
xxxx 
Action Taken to study 
drug: xxx 
Outcome: xxx 
Has the Cause of the AE 
been established: Y - xxx 
Caused study 
discontinuation?: N or Y 
Treated for this AE?: Y or 
N (AE description) 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Adverse Events were coded using MedDRA, Version 18.0. 
Adverse events where PT='Pemphigus' are excluded from this listing. 
Treatment emergent adverse events are the events started on or after the first dose and prior to the date of last dose of study drug.  
If Date >= first dose date, then Study Day = (Date - Date of first dose +1). If Date < first dose date, then Study Day = (Date - Date of first dose). 
Program: <program name (date, time)>, Path: <path> 
 
Programming note for all TEAE listings: Display “Post last dose” if AE started after date of last study drug dosing 


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Same listing shell will be used for the following listing:  
Listing B.16.2.7.4  
Related Treatment Emergent Adverse Events (Safety Population) 
Listing B.16.2.7.5  
Serious Adverse Events (Safety Population) 
Programming note: The following variables will be added to the “SAE” column for the SAE listing: Start Date (ddMMMyyyy) (Study Day), Stop Date (ddMMMyyyy) (Study 
Day) or Ongoing, Duration (if not ongoing), SAE Criteria 
 
Listing B.16.2.7.6  
Treatment Emergent Adverse Events Leading to Study Drug Discontinuation (Safety Population)


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Listing B.16.2.7.7  
Serious Treatment Emergent Adverse Events Leading to Study Drug Discontinuation (Safety Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
PRN 
Dose 
Level at 
AE start  
SOC: System 
Organ Class 
PT: Preferred 
Term 
V: Verbatim 
Start Date 
(ddMMMyyyy) 
(Study Day) 
Stop Date 
(ddMMMyyyy) 
(Study Day) or 
Ongoing 
Duration (if not 
ongoing) 
Pattern of AE 
(Continuous or 
Intermittent) 
SAE? (Y/N) 
If yes: 
Start Date (ddMMMyyyy) 
(Study Day) 
Stop Date (ddMMMyyyy) 
(Study Day) or Ongoing 
Duration (if not ongoing) 
SAE Criteria 
Details 
CTCAE 
Grade 
 
Relationship 
to Study 
Drug 
Details 
400 mg 
QD 
xxxxxxx 
xx/F 
400 mg 
QD 
SOC: xxx 
PT: xxx 
V: xxx 
Start: 
ddMMMyyyy 
(Day X) 
Stop: 
ddMMMyyyy 
(Day X) 
Duration: x 
days 
Pattern: xxx 
Y 
Start: ddMMMyyyy (Day 
X) 
Stop: ddMMMyyyy (Day 
X) 
Duration: x days  
 
SAE Criteria: xxx 
 
Hospitalization: Initial or 
Prolongation 
Admission Date: 
Discharge Date: 
 
Date of Death: 
Cause of Death: 
Autopsy performed?: 
Death certificate 
completed?: 
Grade x 
xxxx 
Action Taken to study 
drug: xxx 
Outcome: xxx 
Has the Cause of the 
AE been established: Y 
- xxx 
Caused study 
discontinuation?: N or 
Y 
Treated for this AE?: Y 
or N (AE description) 
 
 


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Description of SAE: 
Did the SAE abate after 
use of study treatment 
stopped? 
Did the SAE reoccur after 
reintroduction of study 
treatment? 
Interruption of study 
treatment: 
Date stopped: 
Date restarted: 
Type of Sequelae: 
PI rationale for “Not 
Related”: 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Adverse Events were coded using MedDRA, Version 18.0. 
Serious Adverse events where PT='Pemphigus' are included in this listing. 
Treatment emergent adverse events are the events started on or after the first dose and prior to the date of last dose of study drug.  
If Date >= first dose date, then Study Day = (Date - Date of first dose +1). If Date < first dose date, then Study Day = (Date - Date of first dose). 
Program: <program name (date, time)>, Path: <path> 
 
Programming note for all SAE listing: Display ‘Hospitalization’ rows only if patient was hospitalized and display Death rows  only If death is reported. 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Protocol: PRN1008-005 (Part B)  
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A7.10-F1_v3_SAP Template_2016-08-29 
 
 
Shell for Listing B.16.2.7.3 will be used for the following listing:  
Listing B.16.2.7.8  
Treatment Emergent Adverse Events Leading to Death (Safety Population) 
Listing B.16.2.7.9     
Treatment Emergent Adverse Events Started Post Last Dose (Safety Population) 
Listing B.16.2.7.10 
Disease under Study Events (Safety Population) 
 
Programming note: For Listings B.16.2.7.10 replace the footnote regarding ‘Pemphigus’ with the following footnote: Only include adverse events where PT='Pemphigus'. 
 
 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Listing B.16.2.8.1  
Hematology Results (Safety Population) 
Startin
g Dose 
Patient 
ID 
Age/ 
Gender 
Paramete
r 
Current 
PRN Dose 
Visit 
Date / Time of Sample 
(ddMMMyyyy / hh:mm 
24 hr) 
Lab 
Result 
Unit 
Normal Range 
Low or High 
400 
mg QD 
xxxxxxx 
xx/F 
xxxxx 
--- 
xxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
400 mg 
QD 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
L 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
400 mg 
QD 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
H 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Abnormal results are highlighted in yellow. 
Program: <program name (date, time)>, Path: <path> 
 
Programming note for all safety listings: Display “Post last dose” if sample date is after date of last study drug dosing. 
 
 


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Listing B.16.2.8.2  
Hematology Results – Patients with Abnormal Results (Safety Population) 
Startin
g Dose 
Patient 
ID 
Age/ 
Gender 
Parameter 
Current 
PRN Dose 
Visit 
Date / Time of Sample 
(ddMMMyyyy / hh:mm 
24 hr) 
Lab 
Result 
Unit 
Normal Range 
Low or High 
400 mg 
QD 
xxxxxxx 
xx/F 
xxxxx 
--- 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
400 mg 
QD 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
L 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
400 mg 
QD 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
H 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
xx 
x.x to x.x 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Abnormal results are highlighted in yellow 
Program: <program name (date, time)>, Path: <path> 
 
Programming note: This listing excludes patients/parameters with normal results for all visits.  Abnormal results and the Low/High flag will be highlighted in yellow. 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Protocol: PRN1008-005 (Part B)  
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A7.10-F1_v3_SAP Template_2016-08-29 
 
Mock shells for Listing B.16.2.8.1 and Listing B.16.2.8.2 will be used for the following listings:  
 
Listing B.16.2.8.3  
Coagulation Results (Safety Population) 
Listing B.16.2.8.4  
Coagulation Results – Patients with Abnormal Results (Safety Population) 
Listing B.16.2.8.5  
Serum Chemistry Results (Safety Population) 
Listing B.16.2.8.6  
Serum Chemistry Results – Patients with Abnormal Results (Safety Population) 
Listing B.16.2.8.7  
Urinalysis Results (Safety Population) 
Listing B.16.2.8.8  
Urinalysis Results – Patients with Abnormal Results (Safety Population) 
Listing B.16.2.8.9  
Drug Induced Liver Injury (DILI) Test Results (Safety Population) 
Listing B.16.2.8.10  
Drug Induced Liver Injury (DILI) Test Results – Patients with Abnormal Results (Safety Population) 
 
 
 
 


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Listing B.16.2.8.11 
Anti-DSG Antibodies Results (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Current PRN 
Dose 
Was the 
sample 
collected
? 
Collection Date 
(ddMMMyyyy) 
Collection Time 
(hh:mm 24h) 
Lab Test 
Result (unit) 
400 mg 
QD 
xxxxxxx 
xx/F 
xxx 
400 mg QD 
Yes 
ddMMMyyyy 
Hh:mm  
Anti-DSG 1 
xxx unit 
 
 
 
 
 
 
 
 
Anti-DSG 3 
Xxx unit 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Listing B.16.2.8.12  
PK and BTK Sample Collection (ITT Population) 
Starting 
Dose 
Patient 
ID 
Age/ 
Gender 
Visit 
Current PRN 
Dose 
Was the 
sample 
collected? 
Collection Date 
(ddMMMyyyy) 
Collection 
Timepoint  
Collection 
Time 
(hh:mm 
24h) 
Was PK 
sample 
performed 
for this 
visit? 
Date of most 
recent prior 
dose received 
(ddMMMyyyy
) 
Time of 
most recent 
prior dose 
received 
(Hh:mm 
24h) 
400 mg 
QD 
xxxxxxx 
xx/F 
xxx 
400 mg QD 
Yes 
ddMMMyyyy 
xxx  
Hh:mm  
Yes 
ddMMMyyyy 
Hh:mm 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
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Protocol: PRN1008-005 (Part B)  
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A7.10-F1_v3_SAP Template_2016-08-29 
 
 
Listing B.16.2.8.13  
Vital Signs and Body Weight (Safety Population) 
Startin
g Dose 
Patient 
ID 
Age/ 
Gender 
Parameter 
Current 
PRN Dose 
Visit 
Date / Time  
(ddMMMyyyy / hh:mm 24 hr) 
Result 
Normal Range 
Low or 
High 
400 mg 
QD 
xxxxxxx 
xx/F 
SBP (mmHg) 
--- 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140  
 
 
 
 
 
400 mg QD 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
L 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
 
 
 
 
 
400 mg QD 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
H 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Low or High results are highlighted in yellow. 
Program: <program name (date, time)>, Path: <path> 
 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 163 of 171 
SCiAN Services Inc. 
A7.10-F1_v3_SAP Template_2016-08-29 
Listing B.16.2.8.14  
Vital Signs – Blood Pressure and Pulse – Patient with Abnormal Results (Safety Population) 
Startin
g Dose 
Patient 
ID 
Age/ 
Gender 
Parameter 
Current 
PRN Dose 
Visit 
Date / Time  
(ddMMMyyyy / hh:mm 24 hr) 
Result 
Normal Range 
Low or 
High 
400 mg 
QD 
xxxxxxx 
xx/F 
SBP (mmHg) 
--- 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140  
 
 
 
 
 
400 mg QD 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
L 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
 
 
 
 
 
400 mg QD 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
H 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
 
 
 
 
 
 
xxxx 
ddMMMyyyy / hh:mm 
x.x 
90 to 140 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Low or High results are highlighted in yellow. 
Program: <program name (date, time)>, Path: <path> 
 
Programming note: This listing excludes patients/parameters with normal results for all visits.  Abnormal results and the Low/High flag will be highlighted in yellow. 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 164 of 171 
SCiAN Services Inc. 
A7.10-F1_v3_SAP Template_2016-08-29 
 
Listing B.16.2.8.15  
12-Lead ECG Results (Safety Population) 
Starting Dose 
Patient ID 
Age/Gender 
Current PRN 
Dose 
Visit 
Assessment # 
Date / Time 
(ddMMMyyyy / 
hh:mm 24 hr) 
Heart Rate 
(bpm) 
QT Interval 
(msec) 
PR Interval 
(mesc) 
RR Interval 
(msec) 
QRS Duration 
(mesc) 
QTcF Interval 
(msec) 
ECG Assessment 
If Abnormal, 
details 
AE #, if 
abnormal CS 
400 mg QD 
xxxxx
xx 
xx/F 
--- 
Screening 
 
ddMMMyyyy 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Normal 
 
 
 
 
 
400 mg QD 
xxxx 
#1 
ddMMMyyyy / 
hh:mm 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Normal 
 
 
 
 
 
 
 
#2 
ddMMMyyyy / 
hh:mm 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Normal 
 
 
 
 
 
 
 
#3 
ddMMMyyyy / 
hh:mm 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Abn, 
NCS 
 
 
 
 
 
 
 
Average/W
orst finding 
 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Normal 
 
 
 
 
 
 
 
Additional 
ddMMMyyyy / 
hh:mm 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Normal 
 
 
 
 
 
 
 
…etc… 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Average/W
orst finding 
 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Normal 
 
 
 
 
 
 
xxxx 
…etc.. 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Abnormal NCS assessments are highlighted in yellow. Abnormal CS assessments are highlighted in pink. 
Program: <program name (date, time)>, Path: <path> 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 165 of 171 
SCiAN Services Inc. 
A7.10-F1_v3_SAP Template_2016-08-29 
Listing B.16.2.8.16  
12-Lead ECG Results – Patient with Abnormal Results (Safety Population) 
Cohort 
Patient ID 
Age/Gender 
Current PRN 
Dose 
Visit 
Assessment # 
Date / Time 
(ddMMMyyyy / 
hh:mm 24 hr) 
Heart Rate 
(bpm) 
QT Interval 
(msec) 
PR Interval 
(mesc) 
RR Interval 
(msec) 
QRS Duration 
(mesc) 
QTcF Interval 
(msec) 
ECG Assessment 
If Abnormal, 
details 
AE #, if 
abnormal CS 
400 mg QD 
xxxxx
xx 
xx/F 
--- 
xxxx 
 
ddMMMyyyy 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Normal 
 
 
 
 
 
400 mg QD 
xxxx 
#1 
ddMMMyyyy / 
hh:mm 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Normal 
 
 
 
 
 
 
 
#2 
ddMMMyyyy / 
hh:mm 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Abn, 
NCS 
xxx 
x 
 
 
 
 
 
#3 
ddMMMyyyy / 
hh:mm 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Normal 
 
 
 
 
 
 
 
Average/W
orst finding 
 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Abn, CS 
xxx 
x 
 
 
 
 
 
Additional 
ddMMMyyyy / 
hh:mm 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Normal 
 
 
 
 
 
 
 
Additional 
ddMMMyyyy / 
hh:mm 
x.x 
x.x 
x.x 
x.x 
x.x 
x.x 
Normal 
 
 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Abnormal NCS assessments are highlighted in yellow. Abnormal CS assessments are highlighted in pink. 
Program: <program name (date, time)>, Path: <path> 
Programming note: This listing excludes patients with normal assessments for all visits.  Results for abnormal assessments will be highlighted in yellow. 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 166 of 171 
SCiAN Services Inc. 
A7.10-F1_v3_SAP Template_2016-08-29 
 
Listing B.16.2.8.17  
Physical Examination (Safety Population) 
Startin
g Dose 
Patient 
ID 
Age/ 
Gender 
Body System 
Current 
PRN Dose 
Visit 
Result 
If abnormal, briefly describe 
400 mg 
QD 
xxxxxxx 
xx/F 
General 
Appearance 
--- 
xxxx 
Normal 
 
 
 
 
 
400 mg QD 
xxxx 
Abnormal  
xxxx 
 
 
 
 
 
xxxx 
Not Done 
 
 
 
 
 
 
xxxx 
xxx 
 
 
 
 
 
 
xxxx 
Abnormal  
xxx 
 
 
 
 
400 mg QD 
xxxx 
xxx 
 
 
 
 
 
 
xxxx 
xxx 
 
 
 
 
 
 
xxxx 
xxx 
 
 
 
 
 
 
xxxx 
xxx 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Abnormal assessments are highlighted in yellow 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 167 of 171 
SCiAN Services Inc. 
A7.10-F1_v3_SAP Template_2016-08-29 
Listing B.16.2.8.18 
Physical Examination – Patients with Abnormal Results (Safety Population) 
Startin
g Dose 
Patient 
ID 
Age/ 
Gender 
Body System 
Current 
PRN Dose  
Visit 
Exam Date and 
Time 
(ddMMMMyyyy 
hh:mm 24h) 
Result 
If abnormal, briefly 
describe 
400 mg 
QD 
xxxxxxx 
xx/F 
General 
Appearance 
--- 
xxxx 
dddMMMMyyyy 
hh:mm 
Normal 
 
 
 
 
 
400 mg QD 
xxxx 
xxxx 
Abnormal  
xxxx 
 
 
 
 
 
xxxx 
xxxx 
Not Done 
 
 
 
 
 
 
xxxx 
xxxx 
xxx 
 
 
 
 
 
 
xxxx 
xxxx 
Abnormal  
xxx 
 
 
 
 
400 mg QD 
xxxx 
xxxx 
xxx 
 
 
 
 
 
 
xxxx 
xxxx 
xxx 
 
 
 
 
 
 
xxxx 
xxxx 
xxx 
 
 
 
 
 
 
xxxx 
xxxx 
xxx 
 
 
 
 
 
…etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Abnormal assessments are highlighted in yellow.  
Program: <program name (date, time)>, Path: <path> 
 
Programming note: This listing excludes patients with normal results for all body systems and all visits.  Abnormal results will be highlighted in yellow. 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 168 of 171 
SCiAN Services Inc. 
A7.10-F1_v3_SAP Template_2016-08-29 
 
 
Listing B.16.2.8.19  
Pregnancy Test Results (Safety Population) 
Starting 
Dose 
Patient ID 
Age/Gender 
Current PRN 
Dose 
Visit 
Pregnancy 
sample 
collected? 
Collection Date 
(ddMMMyyyy) 
Collection Time 
(hh:mm 24h) 
Pregnancy Lab 
Test 
Result  
400 mg 
QD 
xxxxxxx 
xx/F 
400 mg QD 
xxx 
Yes 
ddMMMyyyy 
hh:mm 
FSH 
Negative  
 
 
 
400 mg QD 
xxx 
Yes 
ddMMMyyyy 
hh:mm 
Serum 
Pregnancy 
Negative  
 
..etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 169 of 171 
SCiAN Services Inc. 
A7.10-F1_v3_SAP Template_2016-08-29 
 
 
Listing B.16.2.8.20  
Food Intake (Safety Population) 
Starting 
Dose 
Patient ID 
Age/Gender 
Current PRN Dose 
Visit 
Date of Food Intake 
(ddMMMyyyy) 
Time of Food 
Intake (hh:mm 
24h) 
Food Intake 
400 mg 
QD 
xxxxxxx 
xx/F 
400 mg QD 
xxx 
ddMMMyyyyy 
Hh:mm  
xxxx 
 
 
 
 
 
 
 
 
 
..etc.. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
F=Female; M=Male 
Program: <program name (date, time)>, Path: <path> 
 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 170 of 171 
SCiAN Services Inc. 
A7.10-F1_v3_SAP Template_2016-08-29 
 
Figure B.14.1.1.1 PDAI Score and PRN Dose Level By Study Day – Panel Plots (mITT Population) 
 


Statistical Analysis Plan – Final 24-Feb-2020 
 
 
CONFIDENTIAL 
Protocol: PRN1008-005 (Part B)  
Principia Biopharma 
 
 
Page 171 of 171 
SCiAN Services Inc. 
A7.10-F1_v3_SAP Template_2016-08-29 
 
Figure B.14.3.2.1 Scatter Plots of Peak TBL vs Peak ALT for Identification of Drug Induced Liver Injury (DILI) (Safety Population) 
 
 
Patients are classified into different dose levels according to the dose they received at a given visit. 
TBL = total bilirubin, ALT = alanine aminotransferase or SGPT, ULN = Upper Limit of Normal. 
The maximum measurement value observed while on a given dose is defined as the peak value. 
Program: <program name (date, time)>, Path: <path>