Clinical trial NCT04128579
Conditions: Lupus Erythematosus Lupus Nephritis
Keywords: Systemic Lupus Erythematosus Active Proliferative Lupus Nephritis
Sponsor: Equillium
Start date: 2019-10-01; Completion date: 2024-01-18
Enrollment: 52 participants; Allocation: NON_RANDOMIZED; Intervention model: SEQUENTIAL

Brief summary:
This is a multi-center study to evaluate the safety, tolerability, PK, PD, and clinical activity of itolizumab (EQ001) in subjects with Systemic Lupus Erythematosus with or without Active Proliferative Lupus Nephritis

Detailed description:
The study will enroll approximately 55 subjects, with up to 5 dose escalating cohorts of 6 open-label subjects enrolled for Type A-SLE and a single dose cohort of approximately 20 open-label subjects enrolled for Type B-Lupus Nephritis.

Subjects will receive itolizumab administered subcutaneously every two weeks for a total of either 2 (Type A) or 13 (Type B) doses with 4 or 12 weeks of follow-up after the last dose of investigational product.

Arms: arm 1: EQ001 administered in an unblinded dose escalating cohort fashion by subcutaneous injection every two weeks for a total of 2 doses (up to 5 cohorts with dosing to be determined in the range of 0.4 -- 3.2 mg/kg). arm 2: EQ001 administered in an unblinded single dose cohort by subcutaneous injection every two weeks for a total of 13 doses (1.6 mg/kg).
Interventions: intervention 1: Itolizumab [Bmab 600]
Intervention descriptions: intervention 1: EQ001

Protocol documents:
Prot_000.pdf: 
 
 
 
Study Title: 
A Phase 1b, Multiple Ascending-dose Study of EQ001 in 
Subjects with Systemic Lupus Erythematosus with or 
without Active Proliferative Lupus Nephritis 
Protocol Number: 
EQ001-19-002 
 
Investigational Product(s): 
EQ001 
 
Sponsor: 
Equillium, Inc. 
 
Development Phase: 
Phase 1b 
 
NCT Number: 
NCT04128579 
 
Document Version Date: 
 
03 Mar 2022 
 
 
 
 
CONFIDENTIAL 
 
Information contained in this protocol is proprietary and confidential in nature and may not be used, divulged, published, or 
otherwise disclosed to others except to the extent necessary to obtain approval of the Institutional Review Board/Independent 
Ethics Committee, or as required by law. Persons to whom this information is disclosed should be informed that this information 
is confidential and may not be further disclosed without the express permission of Equillium AUS Pty Ltd. 


 
 
Clinical Study Protocol 
Study Title: 
A Phase 1b, Multiple Ascending-dose Study of EQ001 in 
Subjects with Systemic Lupus Erythematosus with or 
without Active Proliferative Lupus Nephritis 
Protocol Number: 
EQ001-19-002 
IND Number: 
143830 
Investigational Product(s): 
EQ001 
Sponsor: 
Equillium, Inc. 
 
 
 
Development Phase: 
Phase 1b 
Sponsor's Responsible 
Medical Officer: 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
CONFIDENTIAL 
 
Information contained in this protocol is proprietary and confidential in nature and may not be 
used, divulged, published, or otherwise disclosed to others except to the extent necessary to obtain 
approval of the Institutional Review Board/Independent Ethics Committee, or as required by law. 
Persons to whom this information is disclosed should be informed that this information is 
confidential and may not be further disclosed without the express permission of Equillium, Inc. 


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4 
TABLE OF CONTENTS 
1 
Cover Page ..........................................................................................................................1 
 
 
 
 
4 
Table of Contents ...............................................................................................................4 
5 
Synopsis and Schedule of Events ....................................................................................10 
5.1 
Synopsis ..............................................................................................................10 
 
 
 
 
 
 
 
 
6 
List of Abbreviations and Definitions of Terms ............................................................29 
7 
Introduction ......................................................................................................................32 
7.1 
Background .........................................................................................................32 
7.2 
Itolizumab ...........................................................................................................32 
 
 
 
 
 
 
 
 
 
 
7.5 
Rationale for Evaluating EQ001 for the Treatment of LN .................................36 
8 
Investigational Plan .........................................................................................................38 
8.1 
Overall Study Design and Plan ...........................................................................38 
8.1.1 
Type A ..............................................................................................38 
8.1.2 
Type B...............................................................................................39 
8.2 
Study Periods ......................................................................................................39 
8.3 
Safety Monitoring and Considerations ...............................................................40 
 
 
 
 
 
 
8.4 
Rationale for Study Design .................................................................................43 


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8.4.1 
Rationale for Evaluating EQ001 for the Treatment of Patients 
with Systemic Lupus Erythematosus with or without Active 
Proliferative Lupus Nephritis............................................................43 
8.4.2 
Rationale for Selection of Starting Dose of EQ001 ..........................44 
8.5 
Study Objectives .................................................................................................45 
8.5.1 
Primary Objectives ...........................................................................45 
8.5.2 
Secondary Objectives .......................................................................45 
 
 
8.6 
Endpoints ............................................................................................................45 
8.6.1 
Primary Endpoints ............................................................................45 
8.6.2 
Secondary Endpoints ........................................................................45 
 
 
8.7 
Cohort Dose Escalation (Type A Cohorts) .........................................................46 
8.8 
Data Review Committee, Maximum Tolerated Dose Determination, 
Dose-Limiting Toxicity, and Stopping Criteria ..................................................47 
8.8.1 
Data Review Committee ...................................................................47 
 
 
 
 
 
 
 
 
9 
Study Population ..............................................................................................................51 
9.1 
Inclusion Criteria ................................................................................................51 
9.1.1 
Type A Cohort (Subjects Without Active proliferative LN) 
Inclusion Criteria. .............................................................................51 
9.1.2 
Type B Cohort (Subjects with Active proliferative LN) 
Inclusion Criteria. .............................................................................52 
9.2 
Exclusion Criteria ...............................................................................................55 
9.2.1 
Type A Cohort (Subjects Without Active proliferative LN) 
Exclusion Criteria. ............................................................................55 
9.2.2 
Type B Cohort (Subjects with Active proliferative LN) 
Exclusion Criteria .............................................................................56 
10 
Study Treatments .............................................................................................................58 
 
 
10.2 
Study Drug: EQ001 ............................................................................................58 


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10.5 
Study Drug Administration .................................................................................58 
 
 
 
 
10.8 
Prior and Concomitant Medications ...................................................................61 
10.8.1 
Permitted Therapies ..........................................................................61 
10.8.2 
Restricted Medications .....................................................................61 
10.9 
Enrollment ..........................................................................................................65 
 
 
11 
Study Procedures .............................................................................................................67 
11.1 
Screening and Informed Consent ........................................................................67 
11.2 
Demographics and Medical/Surgical History .....................................................67 
11.2.1 
Demographics ...................................................................................67 
11.2.2 
Medical and Surgical Histories .........................................................67 
11.3 
Physical Examination .........................................................................................68 
11.4 
Vital Signs...........................................................................................................68 
11.5 
SLEDAI-2K Assessment ....................................................................................68 
11.6 
Urine Protein Creatinine Ratio ...........................................................................68 
11.7 
12-Lead Electrocardiography .............................................................................69 
11.8 
Patient-Reported Outcomes Measures ................................................................69 
11.9 
Physician Global Assessment .............................................................................69 
 
 
11.11 
Clinical Laboratory Tests....................................................................................70 
11.11.1 
Safety Laboratory Tests ....................................................................70 
 
 
 
 
12 
Adverse Events .................................................................................................................74 
12.1 
Severity ...............................................................................................................74 
12.2 
Causality Assessment .........................................................................................75 
12.3 
Clinical Laboratory Adverse Events ...................................................................75 
12.4 
Serious Adverse Events ......................................................................................76 


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12.5 
Reporting Adverse Events ..................................................................................76 
12.5.1 
Reporting Procedures for Nonserious Adverse Events.....................76 
12.5.2 
Reporting Procedures for Serious Adverse Events ...........................77 
 
 
13 
Planned Statistical Methods ............................................................................................79 
13.1 
General Considerations .......................................................................................79 
 
 
13.3 
Analysis Populations...........................................................................................79 
13.3.1 
Safety Population ..............................................................................79 
 
 
 
 
13.3.4 
Efficacy Population...........................................................................79 
13.4 
Subject Disposition .............................................................................................79 
13.5 
Demographics and Baseline Characteristics .......................................................80 
 
 
13.7 
Safety Analyses...................................................................................................80 
 
 
13.9 
Efficacy Analyses ...............................................................................................81 
13.9.1 
Lupus Nephritis Subjects (Type B Cohort) ......................................81 
13.9.2 
All Subjects .......................................................................................82 
14 
Administrative Considerations .......................................................................................83 
14.1 
Investigator Responsibilities ...............................................................................83 
14.2 
Independent Ethics Committee Approval ...........................................................84 
14.3 
Ethical Conduct of Study ....................................................................................84 
14.4 
Subject Information and Consent .......................................................................85 
14.5 
Confidentiality ....................................................................................................85 
14.6 
Study Initiation ...................................................................................................86 
14.7 
Case Report Forms and Other Study Records ....................................................86 
14.8 
Study Monitoring ................................................................................................87 
14.9 
Access to Source Documentation .......................................................................87 
14.10 
Study or Study Site Termination ........................................................................87 
 
 




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5 
SYNOPSIS AND SCHEDULE OF EVENTS 
5.1 
Synopsis 
Title of Study Protocol: A Phase 1b, Multiple Ascending-dose Study of EQ001 in Subjects with Systemic Lupus 
Erythematosus with or without Active Proliferative Lupus Nephritis 
Protocol Number: EQ001-19-002 
Name of Sponsor Company: 
Equillium, Inc. 
 
 
Name of Finished Product: EQ001 (itolizumab) 
Phase of Development: Phase 1b 
Objectives: 
Primary Objective 
Characterize the safety and tolerability of EQ001 (itolizumab) in subjects with Systemic Lupus Erythematosus 
(SLE) with or without active proliferative Lupus Nephritis (apLN) 
Secondary Objectives 
1. Characterize the pharmacokinetic (PK) profile of EQ001  
 
  
 
  
4. Characterize the clinical activity of EQ001 in Type B Cohort subjects 
Exploratory Objectives 
 
 
2. Characterize the clinical activity of EQ001 in Type A Cohort subjects  
Endpoints: 
Primary Endpoints 
Safety and tolerability of EQ001, as assessed by treatment-emergent adverse events (TEAEs), treatment-emergent 
serious adverse events (TESAEs), clinical laboratory values, vital signs, 12-lead electrocardiogram (ECG), and 
physical examinations. 
Secondary Endpoints 
1. Pharmacokinetics of EQ001, as assessed by EQ001 serum concentrations at specified timepoints. 
 
 
  
 
 
 
4. Clinical activity of EQ001 in subjects with apLN, as assessed by the following variables):  
 
 
 
 
 
 
 
 
 
 




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The MTD determination will be made based only on Type A Cohorts. Dose escalation is allowed to proceed if no 
predefined stopping criteria are met and the DRC recommends that it is safe to proceed. 
Type B  
Up to approximately 20 subjects will be enrolled and dosed in the Type B Cohort. Subjects will receive a total of 
13 doses of EQ001 1.6 mg/kg administered SC every 2 weeks over 24 weeks. All subjects must be on a stable 
dose of mycophenolate mofetil (MMF) or mycophenolic acid (MPA) for the duration of the study. Subjects that 
require induction treatment with pulse steroid treatment of methylprednisolone ≥500 mg or equivalent will 
undergo a rapid steroid taper with a 20% reduction per week. The targeted goal is to reach a prednisone dose of 
10 mg or less per day by Week 10.
  
The study will be conducted in 3 defined periods, described below: 
1. Screening Period 
a. 
Type A Cohorts: The Screening Period for Cohort A subjects will be up to 4 weeks in duration, 
starting with the first screening procedure in the Schedule of Events (SOE; excluding the informed 
consent), and completed prior to Study Day 1 (Section 5.2) 
b. Type B Cohort: The Screening Period for Cohort B subjects will be up to 6 weeks in duration 
(excluding the informed consent) and completed prior to Study Day 1 and may be completed over the 
course of multiple clinic visits. (Section 5.3). 
2. Treatment Period 
a. 
Type A Cohorts: The Treatment Period for Cohort A subjects will be 4 weeks in duration, beginning 
on Day 1 with the first study drug dose. During the Treatment Period, 2 doses of study drug will be 


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administered subcutaneously (SC), 1 dose each on Days 1 and 15. Subjects will return to the study 
site for follow-up evaluations according to the SOE (Section 5.2). 
b. Type B Cohort: The Treatment Period for Cohort B subjects will be 24 weeks in duration, beginning 
on Day 1 with the first study drug dose. During the Treatment Period, 13 doses of study drug will be 
administered SC, 1 dose each on Days 1, 15, 29, 43, 57, 71, 85, 99, 113, 127, 141, 155, and 169. 
Subjects will return to the study site for follow-up evaluations according to the SOE (Section 5.3 and 
Section 5.4).  
3. Follow-up Period 
a. 
Type A Cohorts: The Follow-up Period for Cohort A subjects will end approximately 6 weeks after 
the last dose of study drug (Section 5.2) 
b. Type B Cohort: The Follow-up Period for Cohort B subjects will end approximately 12 weeks after 
the last dose of study drug (Section 5.4) 
Note: Contraception requirements must be continued for 130 days after the last dose of study drug for 
Type A and B Cohorts. 
Written informed consent for study participation may be obtained up to 4 weeks prior to the Screening Visit for 
Type A Cohort subjects and 8 weeks prior to the Screening Visit for Type B Cohort subjects. Evaluation of 
eligibility will be based on laboratory results, medical history, concomitant and prior medications, vital signs, 
12-lead ECG, physical examination, pregnancy testing (if applicable), and, in Type B Cohort, renal biopsy results. 
Subjects will be evaluated for eligibility to receive the next dose based on laboratory and clinical criteria prior to 
each dose. The pre-dose laboratory evaluations may be performed at a local or central laboratory up to 14- days 
prior to the next target dosing day or on the target dosing day. If a subject discontinues study drug treatment prior 
to the last dose, they will be asked to return to the clinical study site to complete all follow up visits, as described 
in the SOE. 
If a subject withdraws from the study, they should complete an Early Termination Visit.  
 
 
 
 
 
 
 
 
 
 
 
  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
  


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Number of Subjects Planned: 
Approximately 50 subjects with SLE are planned to be enrolled in the study: approximately 30 subjects in Type A 
(subjects without apLN) and up to approximately 20 subjects in Type B (with apLN). Additional subjects may be 
added based on emerging data to meet the objectives of the study. 
Inclusion Criteria: 
Type A Cohort Inclusion Criteria. Subjects will be required to meet all of the following inclusion criteria in 
order to be eligible for study enrollment: 
1. Is male or female, age ≥ 18 and ≤ 75 years 
 
 
 
3. Has previously been documented to have met or currently meets Systemic Lupus International 
Collaborating Clinics (SLICC) and/or American College of Rheumatology (ACR) criteria for SLE 
 
4. Received at least 1 immunosuppressive or immunomodulatory treatment (including antimalarials) for SLE 
at any time in the past or currently 
 
 
 
6. Has documented elevation of antinuclear antibodies (ANA) in the past or during Screening 
7. Restricted SLE treatments (e.g., other immunosuppressive medications, glucocorticoids, and antimalarials) 
are stable and/or washed out at Screening 
 
8. Changes in SLE drug treatment(s), including increased doses of glucocorticoids or other SLE treatments, 
are not anticipated during the study 
9. During Screening, has adequate hematologic function 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 


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Type B Cohort Inclusion Criteria. Subjects will be required to meet all of the following inclusion criteria in 
order to be eligible for study enrollment: 
1. Is male or female, age ≥ 18 and ≤ 75 years 
 
 
 
3. Has a diagnosis of SLE  
4. Kidney biopsy 
 with a histologic diagnosis of LN 
(ISN/RPS 2003 classification of LN) Classes III, IV-S or IV-G, (A) or (A/C); subjects with Class III or IV 
and concomitant Class V will be permitted. 
5. Has a urine protein to creatinine ratio of > 1000 mg/g 
 
 
 
  
6. 
 
 
  
 Requires induction treatment due to newly diagnosed LN or relapsing/flaring disease. 
 
 
 
 
 
 
 
 
 
8. 
 has adequate hematologic function 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
11. Has documented elevation of ANAs in the past or currently 




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Exclusion Criteria: 
 
 
 
 
 
 
 
 
 
 
 
 
 
3. Acute or chronic infections requiring systemic antibacterial, antifungal, or antiviral therapy within 21 days 
of Screening or active infection during Screening 
4. History of or Screening lab test positive for hepatitis B virus (HBV), hepatitis C virus (HCV), or human 
immunodeficiency virus (HIV) 
 
 
 
 
6. Active tuberculosis (TB) or a positive interferon-gamma release assay (IGRA) TB test 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 


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3. Acute or chronic infections requiring systemic antibacterial, antifungal, or antiviral therapy within 21 days 
of the Screening Visit or active infection during Screening 
4. History of or Screening lab test positive for HBV, HCV, or HIV 
 
 
 
6. Active TB or untreated latent TB (as determined by the treating physician and local practice and 
requirements) 
 
 
 
 
 
 
 
 
 
  
 
 
 
 
 
 
 
 
  
 
 
 
 
 


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Investigational Products, Dosage, and Mode of Administration: 
The investigational product is EQ001 (itolizumab), a humanized recombinant IgG1 mAb that selectively targets 
the extracellular scavenger receptor cysteine-rich- (Sc) membrane-distal domain 1 of human CD6. 
 
 
 
 
 
The planned doses and dosing regimen at each dose level 
. The planned doses may be 
substituted for lower doses (including previously studied doses) based on accruing data. The highest dose level 
that will be studied is 3.2 mg/kg. 
Measurements: 
• 
Evaluation of safety will be performed by assessing adverse events, vital signs, 12-lead ECGs, physical 
examinations, and laboratory tests. 
• 
Pharmacokinetics 
. 
• 
Anti-drug antibodies (ADA) will be assessed, as well as neutralizing ADA in subjects that test positive 
for ADAs. 
 
 
 
 
• 
Evaluation of clinical activity will be performed in all subjects using the SLEDAI-2K, patient-reported 
outcomes, serological markers, spot urine PCR, urinalysis, and serum complement. Clinical activity in 
apLN subjects will be evaluated with urine PCR (including 24-hour urine collections), eGFR, and doses 
of prednisone (or equivalent). 
Statistical Methods: 
All safety, PK, 
 and efficacy endpoints will be tabulated with descriptive statistics by cohort for Type A and 
for Type B portions of the study. Data may be pooled across cohorts. 
Analysis Populations: 
The study consists of the following analysis populations: 
Safety Population: All subjects who receive any study drug. 
PK Population: Subjects in the safety population who have at least one measurable post-EQ001 exposure serum 
concentration. 


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Efficacy Population: Subjects in the safety population who have at least 1 post-treatment assessment for a marker 
of clinical activity. 
 
 
 
 
 
 
Safety Analyses: 
Extent of exposure to the study treatment as measured by number of doses administered and duration of exposure 
to the study drug will be summarized using descriptive statistics. Adverse events (including DLTs) will be coded 
using the Medical Dictionary for Regulatory Activities (MedDRA) v 20.1, or higher version and graded using the 
National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE v 5.0) for the 
purposes of data summarization. Incidence of TEAEs, TESAEs, TEAEs leading to study drug discontinuation, 
 TEAEs with an outcome of death will be summarized by MedDRA system 
organ class (SOC) and preferred term. Adverse events will also be summarized by worst CTCAE severity grade 
and causality relationship to study drug. 
 
 
Clinical laboratory data will be descriptively summarized, including observed values at collection timepoints and 
change from baseline. All laboratory parameters that can be graded using CTCAE v 5.0 will be graded. For 
selected parameters, the following summaries may be produced: 
• 
Worst post-baseline severity grade 
• 
Shift summary of baseline grade to worst post-baseline severity grade 
Safety evaluations may also include changes in the subject’s vital signs, and ECG results. The incidence of 
treatment-emergent anti-EQ001 binding and neutralization antibodies will be reported. 
PK Analyses: 
Serum concentrations will be listed and summarized for each EQ001 dose using descriptive statistics for each 
cohort. 
Efficacy Analyses: 
apLN Subjects:  
The geometric mean fold change from baseline in UPCR from a urine collection to FU1 and FU2 will be 
summarized descriptively, along with their 2-sided 95% CI for the fold change. The geometric mean fold change 
from baseline over time by visit will also be described.  
The proportion of subjects with a > 30% and > 50% decline in UPCR and those with a UPCR < 500 mg/g at FU1 
and FU2 will be described, along with their 2-sided 95% CI.  
Time to UPCR decrease of > 50%, > 30%, and to a level of < 500 mg/g from baseline will be summarized using 
the Kaplan-Meier method. Baseline proteinuria is defined as the mean of the 2 UPCR measurements from 24-hour 
urine collections prior to Day 1 dosing. Descriptive analyses of serologic markers, eGFR, and prednisone use will 
be performed. 
Subjects may be assessed based on the time since they had been on apLN treatment, such as MMF/MPA or time 
since pulse corticosteroids (i.e., >12 weeks vs < 12 weeks, or by quartiles). Additional subgroup analyses may be 
performed based on the data collection such as severity of lupus nephritis, baseline proteinuria or previous 
treatments. 


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All Subjects: The changes in SLEDAI-2K scores from baseline will be summarized in both continuous and binary 
scale (e.g., decrease from baseline of ≥ 4 or < 4) with their 95% exact confidence intervals. Other measures of 
clinical activity of EQ001, such as change from baseline in autoantibody titers, serologic markers, and serum 
complement, will be summarized descriptively.  
A detailed description of data analyses and statistical methods will be outlined separately in the Statistical 
Analysis Plan. 


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6 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS 
ACE 
angiotensin converting enzyme 
ACR 
American College of Rheumatology 
ADA 
anti-drug antibody 
AE 
adverse event 
AESI 
adverse event of special interest 
aGVHD 
acute graft versus host disease 
ALC 
absolute lymphocyte count 
ALCAM 
activated leukocyte cell adhesion molecule  
ALT 
alanine aminotransferase 
ANA 
antinuclear antibodies 
ANC 
absolute neutrophil count 
APAC 
Asia-Pacific 
apLN 
active proliferative lupus nephritis 
ARB 
angiotensin-receptor blockers 
AST 
aspartate aminotransferase 
AUC0-inf 
area under the curve from time 0 to infinity 
AUC0-t 
area under the curve at time 0 to t 
Bmab 600 
itolizumab; EQ001 
BUN 
blood urea nitrogen 
CCL 
chemokine (C-C motif) ligand 
CD6 
cluster of differentiation 6 molecule 
CFR 
Code of Federal Regulations 
CH50 
Total hemolytic complement 
Cl 
clearance 
CKD-EPI 
Chronic Kidney Disease Epidemiology Collaboration equation 
Cmax 
maximum serum drug concentration 
Cmin 
minimum serum drug concentration 
CNS 
central nervous system 
CTCAE 
Common Terminology Criteria for Adverse Events 
CV 
cardiovascular  
CXCL 
C-X-C motif chemokine 
DLT 
dose-limiting toxicity 
DRC 
Data Review Committee 
ECG 
electrocardiogram 
eCRF 
electronic case report form 


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eGFR 
estimated glomerular filtration rate 
ET 
early termination 
FACIT-F 
Functional Assessment of Chronic Illness Therapy – Fatigue 
FDA 
Food and Drug Administration 
FU 
Follow up 
GCP 
Good Clinical Practice 
GGT 
gamma-glutamyl transferase 
GI 
gastrointestinal(ly) 
HbA1c 
glycated hemoglobin 
HBV 
hepatitis B virus 
Hct 
hematocrit 
HCV 
hepatitis C virus 
Hgb 
hemoglobin 
HIV 
human immunodeficiency virus 
HbsAg 
hepatitis B virus surface antigen 
ICF 
informed consent form 
ICH 
International Council for Harmonisation of Technical Requirements for 
Pharmaceuticals for Human Use 
IEC 
Independent Ethics Committee 
IFNg 
Interferon gamma 
IgG 
immunoglobulin G 
IgG1 
immunoglobulin G1  
IGRA 
interferon-gamma release assay 
IL- 
interleukin- 
IRB 
Institutional Review Board 
ISN 
International Society of Nephrology 
IUD 
intrauterine device 
IV 
intravenous(ly) 
IWRS 
Interactive Web Response System 
LDH 
lactate dehydrogenase 
LN 
lupus nephritis 
mAb 
monoclonal antibody 
MCP 
monocyte chemoattractant protein 
MedDRA 
Medical Dictionary for Regulatory Activities 
MMF 
mycophenolate mofetil 
MPA 
mycophenolic acid 
MTD 
maximum tolerated dose 


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NCI 
National Cancer Institute 
OVA 
ovalbumin 
PBMC 
peripheral blood mononuclear cell 
PD 
pharmacodynamic(s) 
PGA 
Physician Global Assessment 
Ph.Eur. 
European Pharmacopoeia 
PK 
pharmacokinetic(s) 
QoL 
quality of life 
RANTES 
regulated on activation, normal T cell expressed and secreted 
RBC 
red blood cell 
RNA 
ribonucleic acid 
RO 
receptor occupancy 
RPS 
Renal Pathology Society 
SAE 
serious adverse event 
SC 
subcutaneous(ly) 
Sc 
scavenger receptor cysteine-rich 
SF-36 
The Short Form 36 Health Survey 
SLE 
Systemic Lupus Erythematosus 
SLEDAI-2K 
Systemic Lupus Erythematosus Disease Activity Index 2000 
SLICC 
Systemic Lupus International Collaborating Clinics 
SOC 
system organ class 
SOE 
Schedule of Events 
t½ 
half-life 
TB 
tuberculosis 
TEAE 
treatment-emergent adverse event 
Teff 
effector T cell 
TESAE 
treatment-emergent serious adverse event 
Th 
T helper  
Tmax 
time to maximum concentration 
TNF-α 
tumor necrosis factor alpha 
TNF(R) 
tumor necrosis factor (receptor) 
Treg 
regulatory T cells 
UPCR 
urine protein creatinine ratio 
USP 
United States Pharmacopeia 
Vd 
volume of distribution 
WBC 
white blood cell 
 


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7 
INTRODUCTION 
7.1 
Background 
Systemic Lupus Erythematosus (SLE) is an autoimmune chronic inflammatory disease 
predominantly affecting women in the reproductive age range. The prevalence of SLE varies 
considerably by race, ethnicity, and geographical location 
 Prevalence of SLE in 
the United States has been reported to be between 20 to 150 cases per 100,000 (but as high as 
406 per 100,000 among African American women) 
 
Lupus nephritis (LN) is a serious complication of SLE that results in significant morbidity and 
mortality. Despite the use of currently available anti-inflammatory and immunosuppressive 
treatments, progression of LN to chronic kidney disease is common and 10% of patients develop 
end-stage kidney disease 
 A number of risk factors affect the likelihood of 
progression of LN to end-stage kidney disease, including histologic class 
. The 
presence of Class III (focal segmental) or Class IV (diffuse proliferative) LN increases mortality 
by 6 fold compared to the general population 
 Patients who achieve disease 
remission have improved overall survival and renal survival; however, in a study of patients with 
Class IV LN (diffuse glomerulonephritis), only 43% achieved a complete remission, with 24% 
achieving a partial remission, and 32% achieving no remission 
). Ten-year patient 
survival for patients with complete, partial, and no remission in this study was 95%, 76% and 
46%, respectively. The corresponding 10-year renal survival was 94%, 45%, and 19%, 
respectively. Patient survival with freedom from end-stage kidney disease at 10 years was 92%, 
43%, and 13% for patients with complete, partial, and no remission, respectively. 
T cells are a major factor in driving the immune pathology associated with LN 
 
 One important regulator of T cell activity is CD6, a tightly-regulated, 
co-stimulatory receptor that serves as a key checkpoint in regulating Teff cells that are central to 
autoimmune responses 
 CD6 is highly expressed on CD4+ Teff 
cells but not on regulatory T cells [Treg] 
 Via interactions with its ligand, 
activated leukocyte cell adhesion molecule (ALCAM), CD6 plays an integral role in modulating 
T cell activation and trafficking. Preclinical data and clinical studies in other autoimmune 
diseases (psoriasis and rheumatoid arthritis) have demonstrated that blockade of CD6 
co-stimulation leads to selective inhibition of pathogenic Teff cell activity and trafficking, while 
preserving the essential regulatory function of Treg cells 
 
 
 
7.2 
Itolizumab 
Itolizumab (Bmab 600; EQ001) is a humanized recombinant immunoglobulin G1 (IgG1) 
monoclonal antibody (mAb) that selectively targets the extracellular scavenger receptor cysteine-
rich (Sc) membrane-distal domain 1 of human CD6 
 
 a co-stimulatory membrane glycoprotein associated with T cell modulation and 


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implicated in several autoimmune and inflammatory diseases, including psoriasis, multiple 
sclerosis, rheumatoid arthritis, and Sjögren’s disease 
 
. 
EQ001 binds to domain-1 of human CD6 and is believed to sterically hinder the optimal 
engagement of ALCAM with domain-3, modulating both co-stimulation and trafficking without 
causing T cell depletion. 
 
 
 
 
 
 
 
The binding of ALCAM to CD6 also facilitates lymphocyte trafficking, and disruption of this 
interaction, as demonstrated through the use of CD6KO mice and/or anti-CD6 mAbs, results in 
reduced T cell infiltration into inflamed tissues 
 Additionally, in 
clinical trials with itolizumab, skin biopsies exhibited significantly reduced levels of infiltrating 
CD3 positive T cells 
 
 
 
 
 
 
 
  
 
 
 
 
 
 
 
 
 
 
 
 


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. 
 
 
 
 
 
 
 


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7.4 
Clinical Findings 
 
 
 
 
 
 
. 
Three clinical studies of itolizumab have been performed in India in subjects with rheumatoid 
arthritis and chronic plaque psoriasis. A total of 368 subjects were exposed to T1h at doses of 
0.1 to 1.6 mg/kg. T1h demonstrated preliminary evidence of efficacy in rheumatoid arthritis and 
definitive evidence of efficacy in plaque psoriasis 
 leading to approval of 
itolizumab in India for the treatment of plaque psoriasis. A conditional approval for itolizumab 
for the treatment of plaque psoriasis was also granted by Centro de Immunologia Molecular, 
Cuba on 16 May 2014. 
 
 
 
 
 
 
 
 
 
 




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The unique mechanism of action of EQ001 can selectively target elements of the underlying 
pathogenesis of LN by: (a) inhibiting multiple pathogenic Teff cells and cytokine secretion; 
(b) inhibiting trafficking of Teff cells into kidney tissues; and (c) reducing the Th17:Treg ratio 
associated with LN. Given the central role that Teff cells play in the immunopathogenesis of SLE 
and LN, EQ001, which blocks the CD6-ALCAM pathway and inhibits both the activity as well 
as the trafficking of Teff cells, represents a promising therapeutic approach in this disease. 
 
 
 
 
 
 
 
 
 


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8 
INVESTIGATIONAL PLAN 
8.1 
Overall Study Design and Plan 
This is a Phase 1b, multiple ascending-dose study to evaluate the safety, tolerability, PK, 
 and 
clinical activity of EQ001, a humanized IgG1 mAb that selectively targets CD6 on Teff cells, in 
subjects with SLE.  
Approximately 30 subjects with active or inactive SLE who do not have apLN are anticipated to 
be studied in dose escalation cohorts of 6 subjects each in Type A Cohorts. Up to approximately 
20 subjects with apLN are anticipated to be studied in a Type B Cohort. Additional subjects may 
be added at the maximum-tolerated dose (MTD) cohort and/or to an additional cohort at an 
intermediate or lower dose in Type A. The exact numbers of each type of cohort will be 
determined by emerging data during the study.  
To qualify for enrollment in the study, all subjects must have a diagnosis of SLE. Additionally, 
to qualify for enrollment in a Type B Cohort, subjects must have apLN, defined as proliferative 
LN (International Society of Nephrology [ISN]/Renal Pathology Society [RPS] Class III or IV 
with or without Class V) diagnosed by renal biopsy within 12 months prior to Day 1 or during 
Screening, and previous evidence of serologic and clinical disease activity. Type B Cohort 
subjects may require induction treatment for apLN for newly diagnosed disease or 
relapsing/flaring disease or need additional treatment for incomplete responders. Restricted SLE 
treatments (e.g., immunosuppressive medications and antimalarials) are required to be stable 
and/or washed out 
 Subjects in the Type B Cohort may have 
glucocorticoids administered and tapered 
 
). Investigators will adhere to the protocol steroid taper schedule unless a safety 
concern (including concern related to insufficient response) necessitates departures from the 
schedule. Such planned departures must be discussed with the Medical Monitor, preferably in 
advance of the change in medication. 
8.1.1 
Type A 
Type A will utilize a cohort dose escalation design. If < 2 of the 6 evaluable subjects in a Type A 
dose-escalation cohort experience a dose-limiting toxicity (DLT) during the DLT evaluation 
period (Days 1-29), then escalation may occur to the next higher dose level. If 2 or more of the 6 
initial subjects in a Type A dose-escalation cohort experience a DLT, then no further dose 
escalation will occur. That dose will then be considered to have exceeded the MTD and the next-
lower dose may be declared the MTD. Once a dose has been determined to have exceeded the 
MTD, the Data Review Committee (DRC) may recommend continuation of the study (with or 
without modification) and may expand enrollment to evaluate additional subjects at the MTD 
and/or an additional cohort at an intermediate or lower dose to better define the MTD. 
Type A Cohort enrollment will proceed in a sequential manner to evaluate escalating dose levels 
of EQ001. The first dose level to be studied, Dose Level 1, will be 0.4 mg/kg and the maximum 




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complement. Clinical activity in apLN subjects will be evaluated with urine PCR (including 
24-hour urine collections), eGFR, and doses of prednisone (or equivalent). 
8.3 
Safety Monitoring and Considerations 
Study subjects will be under close medical supervision by the investigator throughout the study. 
Study drug administration will be in the clinic. Scheduled visits for safety assessments will occur 
every 2 weeks during the treatment period. Blood counts will be measured and reviewed prior to 
each dose. Type A Cohorts will be the first cohorts to be studied at any new higher dose and will 
receive only 2 doses (over 4 weeks). Only once a dose is determined to not have exceeded the 
MTD will that dose be allowed to be studied in an expansion cohort of Type B subjects over a 
longer treatment duration (24 weeks). 
Ongoing review of safety data for adverse trends will occur throughout the study. A DRC with 
expertise in nephrology and rheumatology will review data prior to dose escalations and will 
provide recommendations to the Sponsor as described in the DRC Charter and in Section 8.8.1. 
In addition, the Sponsor and/or Medical Monitor will perform regular ongoing review of safety 
data during the study. 
Clinical experience with EQ001 has identified lymphopenia and injection-site (hypersensitivity) 
reactions such as fever as potential adverse drug reactions related to study drug administration.  
8.3.1 
Lymphopenia 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 




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8.3.3 
Systemic Reactions 
8.3.3.1 
Systemic Hypersensitivity Reactions 
Therapeutic protein products may elicit a range of acute effects, from symptomatic discomfort to 
sudden, fatal reactions that have often been described as “infusion reactions” in the past. Infusion 
reactions may encompass a wide range of clinical events, including anaphylaxis and other events 
that may not be directly related to antibody responses, such as cytokine release syndrome. All 
monoclonal antibodies have the potential for infusion/injection site and hypersensitivity 
reactions; therefore, these reactions are regarded as class-specific toxicities for monoclonal 
antibody drugs.  
8.3.3.2 
Fever and other Injection-Related Reactions 
Potential systemic manifestations of infusion/injection-related/hypersensitivity reactions 
including urticaria, pruritus, and fever have been observed following treatment with EQ001. 
For this study, in which the study drug will be administered SC, injection-related reactions are 
defined as acute onset of an illness (other than those limited to the injection-site, which are 
described above) occurring within 48 hours of study drug administration that is otherwise 
unexplained (e.g., by an intercurrent event or the subject’s medical history) but does not meet the 
definition of anaphylaxis 
. Signs of possible injection-related reactions include: 
fever, chills, pruritus, urticaria, chest pain, dyspnea, hypotension, and hypertension. 
In this study, dosing will be done at the study site, and subjects will be monitored for evidence of 
such reactions. Because individual signs and symptoms of hypersensitivity such as fever are non-
specific, investigators are required to clinically evaluate symptoms for etiology to guide 
management. Guidance for symptomatic and supportive treatment of presumed signs and 
symptoms of hypersensitivity are included in the protocol (Section 10.7). Subjects experiencing 
Grade 3 or higher injection-related reactions will be discontinued from study drug treatment. 
Specific information about such reactions will be collected in the eCRF to fully characterize 
them. 
8.3.3.3 
Anaphylaxis 
Anaphylaxis is a severe, potentially fatal, systemic allergic reaction that occurs suddenly after 
contact with an allergy-causing substance, such as an investigational product. Anaphylaxis will 
be evaluated using the diagnostic criteria for anaphylaxis as outlined by the 2006 Joint National 
Institute of Allergy and Infectious Disease/Food Allergy and Anaphylaxis Network Second 
Symposium on Anaphylaxis (see Appendix D). Subjects with a history of anaphylaxis to foods 
or drugs will be excluded from the study. Any subject experiencing anaphylaxis considered 


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related to the study drug by the investigator will be discontinued permanently from study drug 
treatment.  
Doses will be escalated by cohort only after review of safety data by the DRC, with special 
attention to hypersensitivity dose-limiting toxicities. Systemic hypersensitivity reactions 
(Injection-related reactions and anaphylaxis) have also been defined as AESIs, and thus are to be 
reported with clinical details of the event within 24 hours (Section 12.6) for review by the 
Sponsor or designee. 
8.4 
Rationale for Study Design 
The open-label study design will ensure subject safety by allowing frequent ongoing assessment 
of safety by the Sponsor, investigators, and the DRC. All subjects will receive EQ001. The dose 
levels selected (from 0.4 to 3.2 mg/kg) will allow an assessment of the full dose-response curve 
of EQ001. Dose escalation will occur by cohort following review of data by the DRC to allow 
emerging safety, PK, and other data to inform dose-escalation decisions based on pre-defined 
DLT criteria and stopping rules.  
The Type A Cohort subjects will receive 2 doses of study drug, which are expected to have 
pharmacodynamic effects for approximately 4 weeks. The multiple ascending dose data from 
Type A Cohorts is expected to provide sufficient safety and pharmacokinetic data to allow the 
study of a dose (1.6 mg/kg) determined not to exceed the MTD in the apLN population (Type B 
Cohort subjects) over a longer duration. Type B Cohort subjects will receive 13 doses over 
24 weeks as prolonged treatment may be needed for subjects with chronic disease. This is 
reflective of lupus nephritis induction treatments, which generally require at least 6 months of 
treatment as noted in the Kidney Disease Improving Global Outcomes (KDIGO) 
glomerulonephritis guidelines (https://kdigo.org/guidelines/gn/). Prolonged treatment will also 
enable a better understanding of the exposure-response relationship, long-term safety, dose 
selection, and the effect size. 
This design feature is intended to minimize risk to subjects and to allow exploration of a safe 
range of doses over relatively short treatment duration as well as to explore doses associated with 
PD and clinical activity. 
8.4.1 
Rationale for Evaluating EQ001 for the Treatment of Patients with Systemic 
Lupus Erythematosus with or without Active Proliferative Lupus Nephritis 
Current treatment for Class III and IV LN are non-targeted immunosuppressive medications: 
cyclophosphamide and/or mycophenolate with glucocorticoids 
 For 
patients who do not achieve remission with these treatments or who are unable to tolerate them, 
there is no standardized approach to management because there is a general lack of evidence-
basis 
 This study will enroll subjects with biopsy-proven active proliferative Class 
III or IV (with or without Class V) LN who have proteinuria (> 1000 mg/g) and evidence of 
serologic activity who require induction treatment for apLN for newly diagnosed disease or 


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relapsing/flaring disease. In this study, we will enroll patients with apLN who are, by definition, 
at high risk for adverse renal outcomes (Section 7). Further, the study population will consist of 
subjects who continue to have LN activity clinically and serologically despite induction 
treatment. This is a patient population for whom there is a clear unmet medical need and for 
whom the risk-benefit for the addition of an investigational agent to standard treatments is 
justifiable, particularly with close and careful monitoring that is required as part of this protocol. 
In order to select EQ001 doses to examine in the apLN population for a 24-week treatment period, 
the safety, pharmacokinetics, and pharmacodynamics effects of EQ001 of each dose will first be 
studied in patients with SLE with or without active disease (and who do not have apLN) over a 
4-week treatment period. This sequential approach of first evaluating each dose in a shorter 
treatment period in a less ill population allows for the initial evaluation of safety with multiple 
doses, optimization of dose selection for study in the apLN population, and also is intended to 
mitigate risk by initially exposing subjects treated at a given dose to a shorter course of treatment. 
8.4.2 
Rationale for Selection of Starting Dose of EQ001 
A robust body of clinical data exists to inform the selection of a starting dose and dose regimen 
for EQ001 in this study. 
A starting dose of 0.4 mg/kg administered SC was chosen for evaluation in the initial group of 
6 subjects, with additional cohorts receiving the planned doses of 0.8, 1.6, 2.4, and 3.2 mg/kg 
EQ001, based on safety, tolerability, PK, 
 and clinical activity. 
 
 
 
 
 
 
The present study is designed to assure adequate trough levels while assessing the potential for 
accumulation in this short-term study. Because the study population is at increased risk for 
lymphopenia due to the underlying disease condition (SLE) and concomitant background 
treatment(s) (e.g. mycophenolate mofetil), and because of the potential risk of EQ001 causing a 
decrease in lymphocyte counts, the starting dose of EQ001 in this study will be lower 
(0.4 mg/kg) than the lowest dose studied in the Phase 1 healthy volunteer study (0.8 mg/kg). 
Based on the safety, PK, 
 results from Type A cohorts in conjunction with data from 
other EQ001 studies such as in aGVHD, the 1.6 mg/kg was selected as the starting dose for the 
Type B cohorts. This dose was well tolerated in Type A subjects, and there was evidence of 
reduced cell surface level of CD6 on T cells, a PD marker for cellular activity. 
 
 
 
 
 


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8.5 
Study Objectives 
8.5.1 
Primary Objectives 
Characterize the safety and tolerability of EQ001 in subjects with SLE with or without apLN 
8.5.2 
Secondary Objectives 
In subjects with SLE with or without apLN, 
1. Characterize the PK profile of EQ001  
 
  
 
  
4. Characterize the clinical activity of EQ001 in Type B Cohort subjects 
8.5.3 
Exploratory Objectives 
 
  
2. Characterize the clinical activity of EQ001 in Type A Cohort subjects  
8.6 
Endpoints 
8.6.1 
Primary Endpoints 
Safety and tolerability of EQ001, as assessed by TEAEs, treatment emergent-serious adverse 
events (TESAEs), clinical laboratory values, vital signs, 12-lead ECG, and physical examinations. 
8.6.2 
Secondary Endpoints 
1. Pharmacokinetics of EQ001, as assessed by EQ001 serum concentrations at specified 
timepoints.  
 
  
 
 
 
4. Clinical activity of EQ001 in subjects with apLN, as assessed by the following variables:  
 
 
 
 
 
 
 
 
 
 
 
 
 


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8.6.3 
Exploratory Endpoints 
 
 
 
 
 
 
2. For Type B cohort subjects: Change from baseline in the Short-Form 36 Health Survey 
(SF-36) and FACIT-F (Additional Concerns domain) total score to FU1 and FU2 and 
change from baseline in the Lupus Quality of Life (QoL), patient global assessment, and 
physician global assessment (PGA) to FU2 
8.7 
Cohort Dose Escalation (Type A Cohorts) 
The study will utilize a dose escalation design for Type A Cohorts. If < 2 of the 6 evaluable 
EQ001-treated subjects in a Type A dose-escalation cohort experience a DLT during the DLT 
evaluation period (Days 1-29), then escalation may occur to the next higher dose level. If 2 or 
more of the 6 initial subjects in a Type A dose-escalation cohort experience a DLT, then no 
further dose escalation will occur. That dose will then be considered to have exceeded the MTD 
and the next-lower dose may be declared the MTD. Once a dose has been determined to have 
exceeded the MTD, the DRC may recommend continuation of the study (with or without 
modification) at doses at or below the MTD. Alternatively, an additional cohort at an 
intermediate lower dose may be added to better define the MTD. 
Type A Cohort enrollment will proceed in a sequential manner to evaluate escalating dose levels 
of EQ001. The first dose level to be studied, Dose Level 1, will be 0.4 mg/kg and the maximum 
dose level to be studied will be 3.2 mg/kg. Intermediate dose levels of 0.8, 1.6, 2.4 mg/kg are 
planned, but other intermediate doses or doses lower than 0.4 mg/kg may be chosen based on 
emerging PK, PD, and safety data. Subjects in Type A Cohorts will receive a total of 2 doses, 
2 weeks apart. 
 
The MTD determination will be made based only on Type A Cohorts. Dose escalation is allowed 
to proceed if no predefined stopping criteria are met and the DRC recommends that it is safe to 
proceed. 
 Based on emerging data, the DRC may recommend expanding a dose cohort that has not 
exceeded the MTD (up to 6 additional subjects) and/or adding a dose cohort at an intermediate 
lower dose level (with up to 6 subjects).  


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8.8 
Data Review Committee,
 
 
8.8.1 
Data Review Committee 
The DRC membership will consist of 3 independent medical experts with expertise in 
rheumatology and/or nephrology (voting members), as well as an independent statistician, and 
2 Sponsor representatives, which may include one clinical and one safety representative 
(non-voting members). The full description of the DRC structure and responsibilities are 
described in the Data Review Committee Charter. 
The DRC will be responsible for reviewing data from Type A Cohorts throughout the study.  
The MTD determination will be made based only on Type A Cohorts.  
For Type A Cohorts, dose escalation is allowed to proceed if no predefined stopping criteria are 
met (Section 8.8.2) and the DRC recommends that it is safe to proceed. Enrollment at a higher 
dose level will not commence until all safety data from all Type A Cohort subjects from the 




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9 
STUDY POPULATION 
Each cohort will enroll unique subjects who may not be treated in more than 1 cohort. Subjects 
will have SLE without active proliferative LN in Type A cohorts. The subjects in Type B cohorts 
will have SLE with active proliferative LN. 
9.1 
Inclusion Criteria 
9.1.1 
Type A Cohort (Subjects Without Active proliferative LN) Inclusion Criteria.  
Subjects will be required to meet all of the following inclusion criteria in order to be eligible for 
study enrollment: 
1. Is male or female, age ≥ 18 and ≤ 75 years 
2. Has provided written informed consent prior to participation in the study in accordance 
with federal, local, and institutional guidelines 
3. Has previously been documented to have met or currently meets Systemic Lupus 
International Collaborating Clinics (SLICC) and/or American College of Rheumatology 
(ACR) criteria for SLE 
 
4. Received at least 1 immunosuppressive or immunomodulatory treatment (including 
antimalarials) for SLE at any time in the past or currently 
5. If the subject is taking prednisone (or equivalent glucocorticoid), the dose should be 
≤ 10 mg per day and stable for at least 2 weeks prior to Screening. 
6. Has documented elevation of antinuclear antibodies (ANA) in the past or during Screening 
7. Restricted SLE treatments (e.g., other immunosuppressive medications, glucocorticoids, 
and antimalarials) are stable and/or washed out at Screening 
  
8. Changes in SLE drug treatment(s), including increased doses of glucocorticoids or other 
SLE treatments, are not anticipated during the study 
9. During Screening, has adequate hematologic function as defined by 
a. Absolute neutrophil count (ANC) ≥ 1.5 × 109/L 
b. ALC ≥ 1.0 x 109/L 
c. Platelet count > 75 x 109/L 
d. Hemoglobin > 8.5 g/dL 
Note: If a more recent central laboratory value is available at the time of enrollment, the 
most recent value should be used 
10. eGFR ≥ 40 mL/min/1.73m2 during Screening 
Note: If a more recent central laboratory value is available at the time of enrollment, the 
most recent value should be used  
11. If the subject is a female of childbearing potential, she must be willing to practice 
complete abstinence, or she and any male partner are required to simultaneously use 


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2 effective contraceptive methods, from the following list of 5 methods below throughout 
the study and for 130 days after the last dose of study drug: 
• A barrier (condoms, diaphragm or cervical cap) with or without spermicide; 
• A second, different barrier method (condoms, diaphragm or cervical cap); 
• Oral or similar contraceptive, which includes, but is not limited to: injectable, implanted, 
or patch hormone therapy, and hormone-containing intrauterine device (IUD); 
• Non-hormonal IUD (must be used with another non-IUD method); 
• Partner vasectomy at least 6 months prior to Screening. 
For purposes of this study, females will be considered of childbearing potential unless 
they are naturally postmenopausal (i.e., no menses for 2 years) or permanently sterilized 
(i.e., bilateral tubal ligation, bilateral salpingectomy, bilateral oophorectomy, 
hysterectomy, or bilateral tubal occlusion with confirmation of occlusion by 
hysterosalpingogram). 
Male subjects must have had a vasectomy at least 6 months prior to the first dose of the 
study drug or agree with their partner to use one of the acceptable birth control methods 
as listed above throughout the study and for 130 days after the last dose of study drug. 
12. Is willing and able to comply with all protocol procedures and assessments required for 
the study 
13. Is deemed by the investigator to be likely to comply with the protocol for the duration of 
the subject’s participation in the study 
9.1.2 
Type B Cohort (Subjects with Active proliferative LN) Inclusion Criteria.  
Subjects will be required to meet all of the following inclusion criteria in order to be eligible for 
study enrollment: 
1. Is male or female, age ≥ 18 and ≤ 75 years 
2. Has provided written informed consent prior to participation in the study in accordance 
with federal, local, and institutional guidelines 
3. Has diagnosis of SLE  
4. Kidney biopsy within 12 months prior to baseline or during Screening with a histologic 
diagnosis of LN (ISN/RPS 2003 classification of LN) Classes III, IV-S or IV-G, (A) or 
(A/C); Subjects with Class III or IV and concomitant Class V will be permitted. 
5. Has a urine protein to creatinine ratio of > 1000 mg/g based on the mean of two 24-hour 
urine collections or a single urine protein to creatinine ratio of > 2000 mg/g based on a 
single 24-hour urine collection. The 2 collections must be within 12 weeks from Day 1 
with 1 collected and resulted within 6 weeks of Day 1. 


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6. (All Regions except Asia-Pacific [APAC]) All subjects must be on MMF/MPA 2 to 
3 g/day. For subjects that require induction treatment due to newly diagnosed LN or 
relapsing/flaring disease, subjects can have pulse systemic corticosteroids administered as 
part of their induction treatment. 
(APAC) Requires induction treatment due to newly diagnosed LN or relapsing/flaring 
disease. Subjects need to be on MMF/MPA (2 to 3 g/day) and pulse systemic 
corticosteroids as part of their induction treatment. The MMF/MPA and pulse systemic 
corticosteroids can be started within 12 weeks prior to Day 1 or on Day 1. 
7. Subjects that require induction treatment must receive at least 2 consecutive days of pulse 
systemic corticosteroids of methylprednisolone (≥500 mg) or equivalent and be able to 
receive it 12 weeks prior to Day 1 or start on Days 1 and 2; all induction subjects must be 
able to undergo a taper of systemic corticosteroids during the first 10 weeks of treatment. 
8. Within 6 weeks of Day 1 has adequate hematologic function as defined by 
a. ANC ≥ 1.5 × 109/L 
b. ALC ≥ 1.0 x 109/L 
c. Platelet count > 75 x 109/L 
d. Hemoglobin > 8.5 g/dL 
9. Blood pressure ≤ 170/95 mm Hg without recent clinically significant changes per the 
investigator 
10. The investigator considers the protocol-recommended steroid tapering schedule to be 
clinically appropriate for the subject based on the subject’s current status and history 
11. Has documented elevation of ANAs in the past or currently 
12. The dose of MMF/MPA and other SLE drug treatments (other than glucocorticoids) are 
expected to remain stable during the study 
13. Restricted SLE treatments (e.g., other immunosuppressive medications, antimalarials, 
ACE inhibitors, and angiotensin receptor blockers) are stable and/or washed out at 
Screening 
  
14. Most recent eGFR ≥ 40 mL/min/1.73m2 during Screening  
15. No recent clinically significant changes in eGFR (increases or decreases) per the 
investigator 
16. If the subject is a female of childbearing potential, she must be willing to practice 
complete abstinence, or she and any male partner are required to simultaneously use 
2 effective contraceptive methods, from the following list of 5 methods below throughout 
the study and for 130 days after the last dose of study drug: 
• A barrier (condoms, diaphragm or cervical cap) with or without spermicide; 
• A second, different barrier method (condoms, diaphragm or cervical cap); 
• Oral or similar contraceptive, which includes, but is not limited to: injectable, 
implanted, or patch hormone therapy, and hormone-containing IUD; 


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• Non-hormonal IUD (must be used with another non-IUD method); 
• Partner vasectomy at least 6 months prior to Screening. 
For purposes of this study, females will be considered of childbearing potential unless 
they are naturally postmenopausal (i.e., no menses for 2 years) or permanently sterilized 
(i.e., bilateral tubal ligation, bilateral salpingectomy, bilateral oophorectomy, 
hysterectomy, or bilateral tubal occlusion with confirmation of occlusion by 
hysterosalpingogram).  
Male subjects must have had a vasectomy at least 6 months prior to the first dose of the 
study drug or agree with their partner to use one of the acceptable birth control methods 
as listed above throughout the study and for 130 days after the last dose of study. 
17. Is willing and able to comply with all protocol procedures and assessments required for 
the study 
18. Is deemed by the investigator to be likely to comply with the protocol for the duration of 
the subject’s participation in the study 
19. Has documented within 12 weeks of Day 1 one or more of the following, only if subject 
has a kidney biopsy > 6 months from Day 1: 
a. Anti-dsDNA immunoglobulin (IgG) above the reference range 
b. Complement C3 below the reference range 
c. Complement C4 below the reference range 


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9.2 
Exclusion Criteria 
9.2.1 
Type A Cohort (Subjects Without Active proliferative LN) Exclusion Criteria. 
Subjects will be ineligible for enrollment in the study if they meet any of the following criteria: 
1. Active central nervous system (CNS) lupus (including seizures, psychosis, organic brain 
syndrome, cerebrovascular accident, cerebritis or CNS vasculitis) requiring therapeutic 
intervention within 60 days of Screening 
2. Clinical evidence of significant unstable or uncontrolled acute or chronic diseases that are 
not considered related to SLE (e.g., pulmonary [including congestive heart failure NYHA 
III/IV, myocardial infarction or stroke within 6 months, chronic obstructive pulmonary 
disease, pulmonary hypertension, and pulmonary fibrosis], hematologic, gastrointestinal, 
hepatic, renal, neurological, malignancy or infectious diseases) which, in the opinion of 
the investigator, could confound the results of the study, put the subject at undue risk, or 
interfere with protocol adherence or a subject’s ability to give informed consent  
3. Acute or chronic infections requiring systemic antibacterial, antifungal, or antiviral 
therapy within 21 days of Screening or active infection during Screening 
4. History of or Screening lab test positive for hepatitis B virus (HBV), hepatitis C virus 
(HCV), or HIV 
Note: Subjects with a history of HCV treatment who do not have detectable HCV 
ribonucleic acid (RNA) may be enrolled 
5. Primary immunodeficiency other than complement deficiencies 
6. Active TB or a positive interferon-gamma release assay (IGRA) TB test 
Note: Based on local practice and requirements, additional testing can be conducted with 
Medical Monitor approval to rule out active or untreated latent TB for eligibility 
7. Receipt of a live-attenuated vaccine within 2 months prior to Screening or anticipated to 
be required during the study 
8. Female subjects who are pregnant or breastfeeding 
9. History of clinical manifestations of antiphospholipid syndrome (e.g., arterial, venous 
thrombosis, miscarriage, or pre-eclampsia) within 12 months of screening  
10. History of apLN 
11. History of cancer with signs of disease within the 5 years prior to Screening  
Note: Does not apply to subjects with in situ or non-melanoma skin cancer or in situ 
cervical carcinoma that has been completely excised or has been curatively treated. 
12. Has a history of anaphylaxis to foods, drugs, or therapeutic biologic products 
13. Has a history of substance abuse (including alcohol) that may, in the investigator’s 
judgment, increase the risk to the subject of participation in the study 


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14. The subject has received an investigational drug within 30 days or within 5 half-lives 
prior to Screening, whichever is longer or is expected to receive any investigational 
product other than the study drug during the study. 
15. Body mass index ≥ 40 kg/m2 
9.2.2 
Type B Cohort (Subjects with Active proliferative LN) Exclusion Criteria 
Subjects will be ineligible for enrollment in the study if they meet any of the following criteria: 
1. Active CNS lupus (including seizures, psychosis, organic brain syndrome, 
cerebrovascular accident, cerebritis or CNS vasculitis) requiring therapeutic intervention 
within 60 days of Screening 
2. Clinical evidence of significant unstable or uncontrolled acute or chronic diseases that are 
not considered related to SLE (e.g., pulmonary [including congestive heart failure NYHA 
III/IV, myocardial infarction or stroke within 6 months, chronic obstructive pulmonary 
disease, pulmonary hypertension, and pulmonary fibrosis], hematologic, gastrointestinal, 
hepatic, renal, neurological, malignancy or infectious diseases) which, in the opinion of 
the investigator, could confound the results of the study, put the subject at undue risk, or 
interfere with protocol adherence or a subject’s ability to give informed consent  
3. Acute or chronic infections requiring systemic antibacterial, antifungal, or antiviral 
therapy within 21 days or active infection during Screening 
4. History of or Screening lab test positive for HBV, HCV, or HIV 
Note: Subjects with a history of HCV treatment who do not have detectable HCV RNA 
may be enrolled 
5. Primary immunodeficiency other than complement deficiencies 
6. Active TB or untreated latent TB (as determined by the treating physician and local 
practice and requirements) 
Note: Based on local practice and requirements, specific testing or additional testing can 
be conducted with Medical Monitor approval to rule out active or untreated latent TB for 
eligibility as required 
7. Receipt of a live-attenuated vaccine within 2 months prior to Screening or anticipated to 
be required during the study 
8. Female subjects who are pregnant or breastfeeding 
9. History of clinical manifestations of antiphospholipid syndrome (e.g., arterial, venous 
thrombosis, miscarriage, or pre-eclampsia) within 12 months of screening  
10. History of cancer with signs of disease within the 5 years prior to Screening.  
Note: Does not apply to subjects with in situ or non-melanoma skin cancer or in situ 
cervical carcinoma that has been completely excised or has been curatively treated. 
11. Has a history of anaphylaxis to foods, drugs, or therapeutic biologic products 


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12. Has a history of substance abuse (including alcohol) that may, in the investigator’s 
judgment, increase the risk to the subject of participation in the study 
13. History of any concurrent illness (e.g., other than SLE or LN), that has required treatment 
with oral or parenteral glucocorticoids for more than a total of 2 weeks within the last 
12 weeks prior to the Screening Visit 
14. History or rapidly progressive glomerulonephritis and/or other renal disease not directly 
due to LN 
15. The subject has received an investigational drug within 30 days or within 5 half-lives 
prior to Screening, whichever is longer or is expected to receive any investigational 
product other than the study drug during the study. 
16. Body mass index ≥ 40 kg/m 2 


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10 
STUDY TREATMENTS 
 
 
 
 
10.2 
Study Drug: EQ001 
All subjects will receive EQ001 (study drug). EQ001, containing the active ingredient 
itolizumab, is a humanized recombinant IgG1 mAb that selectively targets the extracellular Sc 
membrane-distal domain 1 of human CD6. 
 
 
 
 
 
 
 
 
 
  
 
 
 
 
 
 
 
10.5 
Study Drug Administration 
EQ001 doses will be administered at the study site by a qualified staff member and the time of 
injection will be recorded; subjects will be observed for a minimum of 30 minutes after 
administration of the study drug. Study drug will be administered via SC injection into the 
abdomen. 
In Type A Cohorts, study drug will be administered SC every 2 weeks for 4 weeks (a total of 
2 doses). The first dose level to be studied, Dose Level 1, will be 0.4 mg/kg and the maximum 
dose level to be studied will be 3.2 mg/kg. Intermediate dose levels are currently anticipated to 
be 0.8, 1.6, and 2.4 mg/kg, but other intermediate doses or doses lower than 0.4 mg/kg may be 
chosen 
. 


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The dose level planned for the Type B Cohort is 1.6 mg/kg. Study drug will be administered SC 
every 2 weeks for 24 weeks (a total of 13 doses). This dose has been determined to not have 
exceeded the MTD in a Type A Cohort. 
 
 
 
 
 
 
 
 
 
 
  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 


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10.7 
Management of injection-site and hypersensitivity reactions  
 
 
 
 
 
 
Injection-Related (Hypersensitivity) Reactions  
For this study, in which the study drug will be administered SC, injection-related reactions are 
defined as acute onset of an illness (other than those limited to the injection-site, which are 
described in Section 8.3.3.2 occurring within 48 hours of study drug administration that is 
otherwise unexplained (e.g., by an intercurrent event or the subject’s medical history) but does not 


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meet the definition of anaphylaxis 
 
 Signs of a possible injection-related reaction 
include fever, chills, pruritus, urticaria, chest pain, dyspnea, hypotension, and hypertension. 
As clinically indicated, subjects may be treated for the reaction with antihistamines (H1 ± H2 
blocker), glucocorticoids, supportive care, and any additional interventions. Subjects with Grade 
1 or 2 reactions may continue treatment with study drug with prophylactic measures such as 
acetaminophen, NSAIDs, H1 or H2 blockers prior to each injection. The investigator and Medical 
Monitor should discuss and agree on a plan for prophylaxis for future doses. Grade 3 or higher-
grade reactions require permanent discontinuation of study drug treatment. 
Anaphylaxis 
Anaphylaxis 
 determined to be related to the study drug by the investigator 
requires permanent discontinuation of study drug treatment. Immediate treatment and support 
should be given with oxygen, bronchodilators, steroids, epinephrine, etc. in accordance with 
investigator/local standard of care. 
10.8 
Prior and Concomitant Medications 
Prior and concomitant medications and treatments will be collected at the time of enrollment and 
throughout the study. All prior treatments and treatment regimens for SLE and LN will be 
collected. 
10.8.1 
Permitted Therapies 
Other than restricted medications described in Section 10.8.2, subjects may continue to take their 
chronic medications. 
10.8.2 
Restricted Medications 
Treatments for SLE and its complications may need to be kept stable, washed out, or may be 
disallowed. 
 
 
10.8.2.1 
Glucocorticoids 
In subjects in the Type A cohorts, the dose of glucocorticoids must be kept stable during the 
study
 
Subjects in the Type B Cohort should be on a stable dose of MMF or MPA for the duration of 
the study. For subjects that are undergoing induction treatment, they may require additional pulse 
steroid treatment and receive methylprednisolone ≥500 mg or equivalent within 12 weeks prior 
to starting study treatment or receive a dose start on Day 1 and Day 2 and then undergo a rapid 
oral corticosteroid taper, which will be reduced by 20% per week targeting to reach prednisone 
of approximately <10 mg per day by Week 10. Prednisone or equivalent can be maintained at 
7.5 mg per day for the duration of the study 
  
Investigators will adhere to the protocol steroid taper schedule unless a safety concern (including 


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concern related to insufficient response) necessitates departures from the schedule. Such planned 
departures must be discussed with the Medical Monitor, preferably in advance of the change in 
medication.  


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10.9 
Enrollment  
It is the investigator’s responsibility to ensure that subjects are eligible to participate in the study 
prior to enrollment and continue to remain eligible throughout the study. An IWRS will be used 
to ensure study drug inventory, accountability, and appropriate cohort allocation.  
The study will be open label. After informed consent has been obtained, all screening procedures 
have been assessed, and study eligibility has been confirmed, subjects will receive EQ001 on 
Study Day 1. Subjects will be formally enrolled into the study on Day 1, as defined by 
completing the enrollment transaction within the IWRS.  
The study pharmacist (or the designated site staff) will access the IWRS to receive the dose of 
study drug assigned, record drug accountability, and request resupply.  
 
 
 
 


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11 
STUDY PROCEDURES 
The following assessments will be conducted during screening and at the time points specified in 
the Schedule of Events (SOE) (Section 5.2, Section 5.3, and Section 5.4) and protocol 
Section 10. All missed visits must be documented in the subject’s medical record and the 
appropriate eCRF. 
11.1 
Screening and Informed Consent 
Each subject must sign and date the informed consent form (ICF) before participating in any 
study-specific activities. The ICF may be signed up to 4 weeks prior to the Screening Visit for 
Type A Cohort subjects and 8 weeks prior to the Screening Visit for Type B Cohort Subjects. 
After the ICF is signed, the subject will be assigned a unique subject identification number by IWRS. 
The subject number, assigned at the time of screening, will be used to identify the subject during 
the study and must be used on all study documentation related to that subject. The subject 
identification number will remain constant throughout the entire study and must not be changed 
after initial assignment. Each study site will maintain a list identifying all subjects by subject 
identification number and subject initials. 
After completing the Screening Period, the subject will be evaluated by the investigator to confirm 
eligibility. A subject is considered to have started screening on the date that the Screening Visit 
is recorded in the IWRS. Screen failure subjects will be entered into the IWRS. Investigators will 
maintain a screening log of all potential study subjects that includes limited information about 
each candidate, including dates of screening and procedures, and the outcome of the screening 
process (e.g., enrolled into the study, reason for ineligibility, or withdrawal of consent). 
At Baseline and before any study drug is administered, potential subjects will be reviewed by the 
site to reconfirm their eligibility. 
11.2 
Demographics and Medical/Surgical History 
11.2.1 
Demographics 
Demographic data will be collected, including each subject’s sex, age, race, and ethnicity. Where 
local regulations do not permit certain demographic data to be collected, collection of those data 
will not be required. 
11.2.2 
Medical and Surgical Histories 
The investigator or designee will collect the subject’s medical and surgical histories, including 
information on the subject’s concurrent medical conditions. All findings will be recorded on the 
medical history and surgical history eCRFs. A complete medical history at the Screening Visit 
 


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11.3 
Physical Examination 
The investigator or designee will conduct a complete physical examination as outlined in the 
SOE. Physical examination clinically significant findings prior to the first dose of study drug 
administration will be recorded on the medical history eCRF; clinically significant findings after 
the first study drug dose will be recorded as AEs. 
 
 
 
 
11.4 
Vital Signs 
The following vital sign measurements will be performed: systolic and diastolic blood pressure, 
pulse, respiration rate, and temperature. Vital signs will be measured according to the SOE. On 
study drug dosing days, vital signs will be measured prior to the injection and approximately 15 
and 30 minutes following the end of the injection. The subject must be seated or in a semi-
recumbent position in a rested, calm state for at least 3 minutes before vital signs are collected. 
The position selected for a subject should be the same throughout the study and documented on 
the vital signs eCRF whenever possible. Height will be measured without shoes at Screening. 
Weight without shoes will be obtained at Screening. On dosing days, weight will be obtained 
before dosing. The weight at Screening will be used to calculate all study drug doses, unless 
there has been a weight change of ≥ 20% from Baseline. 
11.5 
SLEDAI-2K Assessment 
The investigator and/or appropriately qualified designee will complete the SLEDAI-2K data 
collection sheet 
 at specific time points, as specified in the SOE. The same 
individual should perform all SLEDAI-2K assessments for a given subject whenever possible. 
11.6 
Urine Protein Creatinine Ratio 
The UPCR from a urine collection will be assessed at the timepoints specified in the SOE.  


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11.7 
12-Lead Electrocardiography 
12-Lead ECGs will be centrally read and obtained on standardized ECG equipment supplied for 
the study. The subject must be in a semi-recumbent or supine position in a rested, calm state for 
at least 10 minutes before ECG assessment is performed. Each 12-lead ECG should be 
performed prior to blood draws, dosing, or other invasive procedures whenever possible. 
The investigator or designated study site physician will review, sign, and date all ECGs.  
Any clinically significant abnormal ECG findings will be recorded as an AE. 
11.8 
Patient-Reported Outcomes Measures 
The Medical Outcomes SF-36 
 FACIT-F 
 
 Lupus QoL 
 and Patient Global Assessment
 
questionnaires will be completed by the subjects while they are at the study site. Subjects should 
be seated in a location away from others during the questionnaire completion. Subjects should 
not confer with anyone on what responses to give nor should anyone else suggest or imply the 
responses to give. Subjects should be told that there is no time limit for completion of the 
questionnaires. After the questionnaires are completed, the site staff should collect them and 
review them to ensure that all questions have been completed before the subject leaves the clinic. 
11.9 
Physician Global Assessment 
At the visits during which the PGA 
 is assessed, investigators will score their global 
assessment of disease activity after reviewing the most recent laboratory results and completing 
the physical examination but before completing the SLEDAI-2K. The same investigator or sub-
investigator should complete all the physician global assessment for a given subject whenever 
possible. 
11.10 
Kidney Biopsy (if needed and approved) 
Kidney biopsies are required in Type B Cohort subjects to determine study eligibility. 
 
 
 If the most recent biopsy was performed more than 12 months prior to 
baseline, a new biopsy is required to confirm the diagnosis of apLN. This study biopsy may be 
performed only following approval of the Sponsor or designee based on key eligibility criteria 
including SLE markers (e.g. C3, anti-dsDNA antibodies), eGFR, UPCR, hematology, 
concomitant medications, and history of apLN. The biopsy will be read locally in accordance 
with the RPS/ISN classification system with an indicator of the activity and chronicity index, 
whenever possible. Slides should be submitted for future central reading. 


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11.11 
Clinical Laboratory Tests 
11.11.1 
Safety Laboratory Tests 
Blood and urine samples will be collected according to the SOE (Section 5.2, Section 5.3, and 
Section 5.4) and below. All scheduled laboratory tests, other than urine pregnancy testing and 
laboratory testing to evaluate hematology parameters prior to dosing, will be performed at the 
central or local laboratory. The pre-dose laboratory evaluations (ie, hematology for ALC) may be 
performed at a local or central laboratory up to 14 days prior to the next target dosing day or on 
the target dosing day. Blood testing for TB may be performed locally, at the investigator’s 
discretion. Repeat laboratory testing will not be required if a test was already performed within 
the specified time window for collection. Samples may be analyzed for the tests outlined in this 
protocol and for any additional tests (with the Sponsor’s approval) necessary to further evaluate 
subject safety. These may include, but are not limited to, investigation of unexpected results. 
Subjects will be in a seated, semi-recumbent, or supine position during blood collection. 
Collection procedures are described in the study Laboratory Manual. 
 
 
  
 


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11.11.1.1 
Complement and Anti-dsDNA 
Blood samples will be assayed for complement C3 and C4 and anti-dsDNA at selected time 
points (see SOE for details). Total hemolytic complement (CH50) may also be measured in the 
Type B Cohort. 
11.11.1.2 
Autoantibodies 
Serum will be assayed for autoantibodies, including anti-nuclear antibodies (ANA) at selected 
time points (see SOE for details). Anti-C1q antibodies may also be analyzed. 
11.11.1.3 
Pharmacokinetics 
Serum will be assayed for EQ001 concentrations using a validated assay. The PK samples will 
be collected at time points specified in the SOE. 
11.11.1.4 
Anti-Drug Antibody and Neutralizing ADA 
Serum samples will be obtained for detection of ADA against EQ001 at the time points specified 
in the SOE, and, if required, a neutralizing ADA assay will be performed on the same samples. 
ADA sampling may be requested by the Sponsor at an unscheduled visit to further evaluate 
safety in individual patients. 
 
 
 
 
 


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12 
ADVERSE EVENTS 
An AE is defined as any unfavorable and unintended sign (including an abnormal laboratory 
finding), symptom, or disease or any worsening of a pre-existing condition temporally associated 
with the use of a study drug, whether or not related to study drug. The AEs that occur after the 
first dose of EQ001 or during the study treatment and safety follow-up periods will be 
documented on the AE eCRF. The investigator will assess AE severity and the causality 
relationship of the AE to study drug. The investigator will treat the subject as medically required 
to ensure the subject’s safety.  
Laboratory values that are outside the laboratory reference range should be reported as AEs only 
if the investigator considered those AEs to be clinically significant. 
From the time of signing the ICF through the first study drug administration, all SAEs and 
nonserious AEs related to protocol-mandated procedures (including medication washout 
performed specifically for the study) will be recorded on the SAE/AE eCRF. All other untoward 
medical occurrences observed during screening, including exacerbation or changes in the 
medical and surgical history, will be captured on the medical and surgical history eCRF. Details 
on recording and reporting AEs are provided below. All AEs will be captured through the last 
required Follow-up visit or the ET Visit, whichever is later. 
Investigators should use their clinical judgment to determine whether a subject is to be 
withdrawn due to an AE. In the event the subject discontinues study treatment or is withdrawn 
from the study due to an AE, the subject should be asked to complete all remaining FU study 
visits. In the event a subject is withdrawn from the study, an ET Visit should be completed. 
All subjects experiencing AEs, including clinically significant abnormal laboratory values, 
whether or not associated with the study drug, must be monitored until the condition (1) returns 
to normal, (2) returns to the subject’s baseline, (3) the investigator determines the AE has 
reached a stable outcome and is no longer clinically significant, or (4) the subject is considered 
lost to follow-up. 
12.1 
Severity 
All AEs, both serious and nonserious, will be assessed for severity using the NCI CTCAE v 5.0. 
The CTCAE scale includes unique clinical descriptions of AEs categorized by anatomy and/or 
pathophysiology. Reference the following website: 
https://ctep.cancer.gov/protocolDevelopment/electronic_applications/docs/CTCAE_v5_Quick_R
eference_8.5x11.pdf 
The CTCAE scale displays Grades 1 through 5 with unique clinical descriptions of severity for 
each AE (including abnormal laboratory values), based on this general guideline provided in the 
scale. For AEs not covered by CTCAE, the conventional definition of severity will be used, as 
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• Grade 1 (mild) AE: Minor; no specific medical intervention; marginal clinical relevance. 
• Grade 2 (moderate) AE: Minimal intervention; local intervention; noninvasive 
intervention. 
• Grade 3 (severe) AE: Significant symptoms requiring hospitalization or invasive 
intervention. 
• Grade 4 (life-threatening or disabling) AE: Complicated by acute, life-threatening 
complications; need for intensive care or emergent invasive procedure. 
• Grade 5: Fatal AE 
12.2 
Causality Assessment 
The investigator or qualified subinvestigator is responsible for assessing and assigning the 
causality relationship of the event to study drug or study-related procedures (e.g., invasive 
procedures, such as venipuncture) using clinical judgment and the following categories of 
relatedness: 
• Related: There is at least some possibility that the AE could be related to study drug or a 
study-related procedure. 
• Not related: There is a high degree of certainty that the AE is NOT related to study drug 
or a study-related procedure.  
In making a causality assessment of an AE, it should be considered as to whether or not the AE 
is expected to occur due to the underlying disease, based on the investigator’s clinical experience 
in managing SLE (Section 12.5.1). 
12.3 
Clinical Laboratory Adverse Events 
The investigator is responsible for reviewing the results of all laboratory tests as they become 
available and determining whether an abnormal value in an individual subject is a clinically 
significant change from the subject’s baseline value(s). The investigator may repeat the 
laboratory test or request additional tests to verify the results of the original laboratory tests. In 
general, abnormal laboratory findings without clinical significance (based on the investigator's 
judgment) are not to be recorded as AEs. However, laboratory value changes that require 
treatment or adjustment in current therapy are to be considered AEs. When applicable, clinical 
sequelae (not the laboratory abnormality) are to be recorded as the AE. 


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12.4 
Serious Adverse Events 
A serious adverse event (SAE) is defined as any AE occurring at any dose that results in any of 
the following outcomes: 
• Death 
• Life-threatening AE (Note: A life-threatening AE is one that, in the view of the 
investigator places the subject at immediate risk of death from the reaction as it occurred) 
• Hospitalization or prolongation of existing hospitalization 
• A persistent or significant disability/incapacity 
• A congenital anomaly/birth defect. 
Important medical events that may not result in death, be life threatening, or require 
hospitalization may be considered an SAE when, based upon appropriate medical judgment, they 
may jeopardize the subject and may require medical or surgical intervention to prevent one of the 
outcomes listed in this definition. Examples of such medical events include blood dyscrasias or 
convulsions that do not result in subject hospitalization, or the development of drug dependency 
or drug abuse. 
 
 
All SAEs, regardless of cause(s) or causal relationship to study drug, must be reported within 
24 hours to the study Sponsor and/or designee. If an SAE occurs outside the reporting window of 
the subject’s follow-up or ET visits and is considered related to study treatment, it must be 
reported to the Sponsor within 24 hours of the site’s awareness. 
12.5 
Reporting Adverse Events 
12.5.1 
Reporting Procedures for Nonserious Adverse Events 
The investigator is responsible for ensuring that all AEs observed by the investigator or designee 
or reported by the subject are reported using the AE eCRF. 
The investigator will assign the following AE attributes: 
• AE diagnosis or syndrome(s), if known (if not known, signs or symptoms) 
• Dates of onset and resolution (if resolved) 
• Severity 
• Causality relationship to study drug or study-related procedures  
• Action taken 
• Outcome 


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Follow-up of nonserious AEs will continue through the last day on the study and/or until a 
definitive outcome (e.g., resolved, resolved with sequelae, lost to follow-up) is achieved. 
When a subject is withdrawn from the study because of a nonserious AE, the Sponsor and/or 
designee must be notified by email or phone within 48 hours. 
12.5.2 
Reporting Procedures for Serious Adverse Events 
The investigator is responsible for ensuring that all SAEs observed by the investigator or 
reported by the subject are promptly assessed and reported to the Sponsor and/or designee. The 
investigator must assess the causality relationship of the SAE to study drug or any study-related 
procedure. 
The procedures for reporting SAEs are as follows: 
• Within 24 hours of the investigator’s knowledge of the event, enter data into the AE/SAE 
eCRF. If the EDC is not available, the paper SAE Report Form may be used, but all data 
must be entered into the EDC once the EDC is available. 
• Additional contact numbers and AE/SAE reporting instructions will be provided in the 
eCRF Completion Guidelines. 
• For fatal or life-threatening events and all SAEs, also email and/or fax redacted copies of 
hospital records, autopsy reports, and other documents, when requested and applicable. 
Transmission of such documents should occur with personal subject details de-identified 
(redacted), without losing the traceability of a document to the subject identifiers. Entry 
of the initial report of the event into EDC should not be delayed in order to include these 
additional documents. 
• The Sponsor and/or designee may request additional information from the investigator to 
ensure the timely completion of accurate safety reports. 
The investigator must take all necessary therapeutic measures for resolution of the SAE. Any 
medications or therapies necessary for treatment of the SAE must be recorded in the event 
description section of the SAE form and the concomitant medication eCRF. 
Follow-up of SAEs will continue through the last day on the study and/or until a definitive 
outcome (e.g., resolved, resolved with sequelae, lost to follow-up, fatal) is achieved. 
While pregnancy is not considered an AE, all cases of fetal drug exposure via a maternal parent 
as study subject or pregnancy of a partner of a male study subject that occurs during the study or 
within 130 days after the last dose of study drug will be reported immediately to the Sponsor or 
its designee. Information related to the pregnancy must be documented on a Pregnancy 
Confirmation and Outcome Form, provided by the Sponsor or its designee, and the pregnancy 
should be followed until a definitive outcome has been determined. 




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13.1 
General Considerations 
Data will be summarized using descriptive statistics (n, mean, median, standard deviation, 
minimum, maximum) for continuous variables and frequencies and percentages for discrete 
variables. All data summaries will be displayed by cohort. Data may also be pooled across 
cohorts. Baseline values of variables are considered the last available non-missing value prior to 
receiving any study drug unless otherwise specified. By-subject listings of the data will also be 
provided. All data summaries and listings will be produced using the SAS® software, version 9.3 
or higher. 
 
 
 
 
. 
13.3 
Analysis Populations 
13.3.1 
Safety Population 
The safety population consists of all subjects who receive any study drug.  
13.3.2 
Pharmacokinetic Analysis Population 
The pharmacokinetics population includes those subjects in the safety population who have at 
least one measurable post-EQ001 exposure serum concentration. 
 
 
 
 
13.3.4 
Efficacy Population 
Subjects in the safety population who have at least 1 post-treatment assessment for a marker of 
clinical activity will be included in the efficacy population. 
13.4 
Subject Disposition 
Subject disposition data will be summarized and will include the number and percent of enrolled 
subjects; number and percent of subjects initiating and completing treatment; and number and 
percent of subjects discontinuing treatment and discontinuing the study, further broken down by 


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the reasons for discontinuation. Subject enrollment will also be summarized by study site. A 
summary of major protocol deviations will be tabulated for each cohort. 
13.5 
Demographics and Baseline Characteristics 
Demographics and other baseline characteristics will be summarized for the subjects in the safety 
population. Demographic data will include age, gender, race, and ethnicity. Baseline 
characteristics data will include (but not be limited to) renal function, proteinuria (UPCR), 
complement, and autoantibody titers. Medical history data will include (but not be limited to) 
time from diagnosis of SLE to Screening, prior and current medications used for SLE, including 
use of biologic therapy for SLE. 
13.6 
Statistical Analysis of Pharmacokinetic Variables 
Serum concentrations will be listed and summarized for each EQ001 dose using descriptive 
statistics for each cohort. 
 
 
13.7 
Safety Analyses 
All safety data summaries will use the safety population. 
The number and percent of Type A subjects experiencing any DLTs will be presented by cohort. 
A listing of DLTs will also be provided. 
Adverse events (including DLTs) will be coded using the Medical Dictionary for Regulatory 
Activities (v 20.1, or higher version) and will be graded by the investigator using the NCI 
CTCAE v 5.0. Subject incidence of TEAEs, TESAEs, TEAEs leading to treatment 
discontinuation, AESIs, and TEAEs with an outcome of death will be summarized by SOC and 
preferred term. Adverse events will also be further summarized by worst CTCAE severity grade 
and relationship to study drug. In addition, AESIs as described in Section 12.6 will also be 
summarized by SOC, preferred term, and worst severity grade.  
Clinical laboratory data will be summarized descriptively, these summaries will include observed 
values at collection timepoints and their changes from baseline. All laboratory parameters that 
can be graded using the CTCAE v 5.0 will be graded. For selected parameters, the following 
summaries may be produced: 
• Worst post-baseline severity grade 
• Shift summary of baseline grade to worst post-baseline severity grade. 
Safety evaluations may also include changes in the subject’s vital signs and ECG findings. 


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13.9 
Efficacy Analyses 
All efficacy analyses will use the Efficacy population. Handling of efficacy data obtained after a 
subject receives new immunosuppressive or other treatments for LN or has an increase in 
glucocorticoids will be described in the Statistical Analysis Plan. 
 
 
 
 
Analyses will be performed by dose and with all doses combined. 
13.9.1 
Lupus Nephritis Subjects (Type B Cohort) 
13.9.1.1 
Proteinuria 
The geometric mean fold change from baseline in UPCR from a 24-hour urine collection and 
spot collections to FU1 and to FU2 will be summarized descriptively, along with their 2-sided 
95% CI for the fold change. The geometric mean fold change from baseline over time by visit 
will also be described. The proportion of subjects with a > 30% and > 50% decline in UPCR and 
those with a UPCR < 500 mg/g at FU1 and FU2 will be described, along with their 2-sided 95% 
CI. 
Time to earliest response of UPCR with response defined as a decrease of > 50% from baseline, 
a decrease of > 30% from baseline, and a decrease to a level < 500 mg/g will be summarized 
using the Kaplan-Meier method. 
 
 
 
 
 
 
13.9.1.2 
Serologic Markers Associated with Active LN (C3, C4, anti-dsDNA) 
The mean change from baseline in C3, C4, and anti-dsDNA to FU1 and FU2 will be 
summarized, along with their 2-sided 95% CI for the change. The mean change from baseline 
over time by visit will also be described. 


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13.9.1.3 
eGFR 
The change from baseline in eGFR (mean of all Screening and Day 1 values) will be summarized 
by time point. The proportion of subjects with a change from baseline to FU1 and FU2 of < 20%, 
< 30%, and < 40% will be summarized along with their 2-sided 95% CI. 
13.9.1.4 
Glucocorticoid Dose 
The mean change from baseline in the daily prednisone equivalent dose during Screening to the 
mean daily prednisone dose during the period from FU1 to FU2 will be summarized along with 
the 2-sided 95% CI. The proportion of subjects (with the 2-sided 95% CI) who had a prednisone 
dose equivalent < 10.0 mg, < 7.5 mg and < 5.0 mg at FU1 and FU2 will be summarized. This 
analysis will also be performed for FU3 and FU4. 
13.9.1.5 
Responders 
The proportion of subjects achieving a complete and partial renal response 
 
will be summarized along with the 2-sided 95% CI at FU1 and FU2. Time to earliest response 
will be summarized using the Kaplan-Meier method.  
13.9.1.6 
PRO Endpoints 
Analyses of patient-reported outcomes measures (SF-36, Lupus QoL, FACIT-F 
 
 and Patient Global Assessment) and the Physician Global Assessment will be 
described in the Statistical Analysis Plan. 
13.9.2 
All Subjects  
The changes in SLEDAI-2K scores from baseline will be summarized in both continuous and 
binary scale (e.g., decrease from baseline of > 4 or ≤ 4) with their 95% exact confidence 
intervals. Other measures of clinical activity of EQ001, such as change from baseline in 
autoantibody titers, serologic markers, and serum complement, will be summarized descriptively.  


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14 
ADMINISTRATIVE CONSIDERATIONS 
14.1 
Investigator Responsibilities 
The investigator is responsible for complying with all regulatory requirements relating to 
performing clinical research with an investigational product. The investigator is responsible for 
ensuring that the investigation is conducted according to the signed Investigator Agreement 
(FDA Form 1572, or equivalent), the approved protocol, and applicable regulations for 
protecting the rights, safety, and welfare of study subject under the investigator's care. The 
investigator is additionally responsible for the control of investigational product and for 
providing accurate and verifiable data to the Sponsor. 
The investigator must obtain the written informed consent of each subject before participation in 
the study. The investigator must assure initial and continuing review of the study by an IRB/IEC 
that complies with applicable national and local regulations. 
The investigator will ensure adequate documentation of the training of research study personnel 
for conduct of the study, including qualifications, experience, and study role. 
The investigator will be given a copy of the most current version of the EQ001 Investigator’s Brochure 
and appropriate study process manuals and plans. The investigator is obligated to become 
familiar with these documents prior to initiation of the study. 
Other investigator responsibilities relative to the IRB/IEC include, but are not limited to, the 
following: 
• Submit to the IRB/IEC for review any advertisements that will be used to recruit subjects, 
as applicable 
• Submit all protocol amendments, revisions of the Investigator’s Brochure, or revisions of 
the Informed Consent to the IRB/IEC for review 
• If Sponsor notifies the investigator about SAEs reported in other studies associated with 
this investigational product, report that information to the IRB/IEC if required per local 
regulations 
• Provide the IRB/IEC with any other information it requests before or during the conduct 
of the study 
• Report to the IRB/IEC all adverse drug reactions that are serious, unexpected, and related 
to investigational product as per local regulations 
• Maintain a file of study-related information 
• Update the IRB/IEC on a minimum of a yearly basis as per local regulations 


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14.2 
Independent Ethics Committee Approval 
Before initiation of the study at a study site, the protocol, including the final version of the ICF, 
the subject information sheet (if applicable), and any other applicable/relevant study 
documentation will be submitted to the appropriate IRB/IEC. In addition, the IRB/IEC must 
approve all advertising used to recruit subjects for the study prior to use. Written approval of the 
study documentation must be obtained and sent to the Sponsor or its designee before the study 
drug can be released to the investigator.  
The investigator is responsible for informing the IRB/IEC of any amendments to the protocol, 
ICF, written information provided to subjects, and/or other procedures in accordance with local 
requirements. The protocol must be re-approved by the IRB/IEC upon receipt of amendments, in 
accordance with applicable law. The investigator must send a copy of the approval letter from 
the IRB/IEC to the Sponsor or its designee. 
The investigator will report promptly to the IRB/IEC and the Sponsor any new information that 
may adversely affect the health or safety of past or current subjects or the conduct of the study, 
including deviations from the protocol or reports of any reportable SAEs, during and for 
one (1) year after study completion.  
The investigator should submit written reports of clinical study status to their IRB/IEC annually 
or more frequently if required. A final study notification will also be forwarded to the IRB/IEC 
after the study is completed or in the event of premature termination of the study in accordance 
with the applicable regulations. After completion of the study, the investigator will provide the 
IRB/IEC with a report of the outcome of the study. Copies of all contacts with the IRB/IEC 
should be maintained in the study file. Copies of clinical study status reports (including 
termination) should be provided to the Sponsor. 
14.3 
Ethical Conduct of Study 
The investigator(s) and all parties involved in this study should conduct the study in adherence to the 
ethical principles based on the Declaration of Helsinki, GCP, ICH guidelines, and the applicable 
national and local laws and regulatory requirements (referred to herein as “applicable law”). 
Investigators and all sub-investigators will comply with 21 Code of Federal Regulations (CFR), 
Part 54, 1998 and similar conflicts of interest laws requiring documentation of financial interests 
or arrangements with the Sponsor, or proprietary interests in the drug under study and any other 
local regulatory requirements as applicable. Any required documentation must be provided prior 
to the investigator’s (and any sub-investigator’s) participation in the study. The investigator and 
sub-investigator(s) will notify the Sponsor or its designee of any change in reportable interests 
during the study and for 1 year following completion of the study. Study completion is defined 
as the date when the last subject completes all protocol-defined activities. 


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14.4 
Subject Information and Consent 
Prior to conducting any study-related procedures, the investigator must obtain written informed 
consent from each subject, in accordance with applicable law. Consent will be documented on a 
written ICF. The ICF must be approved both by the Sponsor and by the reviewing IRB/IEC prior 
to presenting it to a subject. Each ICF must comply with the ICH GCP Guidelines and applicable 
regulatory requirements.  
Investigators may discuss study availability and the possibility for entry with a potential subject 
without first obtaining consent. However, informed consent must be obtained and documented 
prior to initiation of any procedures that are performed solely for the purpose of determining 
eligibility for research, including withdrawal from current medication(s). The informed consent 
process should take place under conditions where the subject has adequate time to consider the 
risks and benefits associated with his/her participation in the study. The investigator or qualified 
designee must explain to each subject the aims, methods, reasonably anticipated benefits, and 
potential hazards of the study. 
Once appropriate essential information has been provided and fully explained in layman’s 
language to the subject by the investigator (or a qualified designee), the approved ICF will be 
signed and dated by both the subject and the person obtaining consent (investigator or designee), 
as well as by any other parties required by the IRB/IEC. The subject will receive a copy of the 
signed ICF; the original will be retained in the study files. The investigator must document the 
consent interview and place the record in the study files. The investigator shall also maintain a 
log of all subjects who sign the ICF and indicate if the subject was enrolled into the study or 
reason for non-enrollment. 
14.5 
Confidentiality 
The investigator must assure that subjects’ anonymity will be strictly maintained and that their 
identities are protected from unauthorized parties. Only subject initials, age, another unique 
identifier (as allowed by local law) and an identification code will be recorded on any form or 
biological sample submitted to the Sponsor, IRB/IEC, or laboratory. Laboratory specimens must 
be labeled in such a way as to protect subject identity while allowing the results to be recorded to 
the proper subject. Refer to specific laboratory instructions (or in accordance with local 
regulations). NOTE: The investigator must keep a screening log showing codes, names, and 
addresses for all subjects screened and for all subjects enrolled in the study. Subject data will be 
processed in accordance with all applicable regulations. (Some studies may require double-
coding of samples). 
The investigator agrees that all information received from the Sponsor, including but not limited 
to the EQ001 Investigator’s Brochure, this protocol, CRF/eCRF, the study drug, and any other 
study information, remain the sole and exclusive property of the Sponsor during the conduct of 
the study and thereafter. This information is not to be disclosed to any third party (except 
employees or agents directly involved in the conduct of the study or as required by law) without 


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prior written consent from the Sponsor. The investigator further agrees to take all reasonable 
precautions to prevent the disclosure by any employee or agent of the study site to any third 
party or otherwise into the public domain. In compliance with applicable law and/or ICH GCP 
Guidelines, the investigator will permit the Sponsor’s representatives and, when necessary, 
representatives of the regulatory authorities, direct access to any medical records relevant to the 
study for verification of study-related procedures and data. Direct access includes examining, 
analyzing, verifying, and reproducing any records and reports that are important to the evaluation 
of the study. 
Investigators will obtain authorization from the subject to permit access to study-related records, 
including personal information.  
Authorization is required from each subject (e.g., specific permission granted by such individual 
to a covered entity for the use or disclosure of an individual's protected health information). The 
investigator and institution must obtain such waiver/authorization in writing from the subject. 
Valid authorization must meet the implementation specifications under the applicable privacy 
laws. Authorization may be combined in the ICF (approved by the IRB/IEC), or it may be a 
separate document (approved by the IRB/IEC) or provided by the investigator or Sponsor 
(without IRB/IEC approval). 
14.6 
Study Initiation 
Before the study drug can be shipped to the study site and before enrollment can begin, all 
applicable documentation and approvals as per local regulations must be in place. 
The Sponsor or designee will notify the site when they are activated.  
14.7 
Case Report Forms and Other Study Records 
The investigator will comply with the requirements for all assessments and data collection for 
each subject, as specified in the protocol. 
During each subject’s visit to the study site, the investigator or qualified designee will record 
progress notes in the subject’s medical record to document all significant observations. At a 
minimum, these notes will contain the following: 
• Documentation of the informed consent process, including any revised consents. 
• The date of the visit and the corresponding visit or day in the study schedule. 
• General subject status remarks, including any significant medical findings. The severity, 
frequency, and duration of any AEs and the investigator's assessment of relationship to 
study drug must also be recorded. 
• Any changes in concomitant medications. 
• A general reference to the procedures completed. 


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In addition, any contact with the subject via telephone or other means that provides significant 
clinical information will also be documented in the progress notes, as described above. 
Data for this study will be captured in electronic eCRFs. Study auditing, data entry, verification 
and validation, and subsequent analysis will be performed by the Sponsor, or its designees, in 
accordance with GCPs and established Standard Operating Procedures. 
Clinical data (including AEs, concomitant medications, and applicable clinical laboratory data) 
will be entered into an electronic database. The creation and validation of the database, data 
entry, validation, and verification will be performed according to 21 CFR part 11 and other 
applicable local regulations. The data system includes password protection and internal quality 
checks, such as automatic range checks, to identify data that appear inconsistent, incomplete, or 
inaccurate. 
14.8 
Study Monitoring 
The Sponsor or designee will assign monitors who will perform on site monitoring as frequently 
as necessary and in accordance with ICH GCP. 
Source documents and eCRFs will be reviewed at monitoring visits and any findings will be 
discussed with the investigational staff. The Sponsor expects that at monitoring visits study 
documents and staff will be available and a suitable space will be provided for review of the 
study documents. The monitor will meet with the investigator on a regular basis to provide 
feedback on the conduct of the study. 
14.9 
Access to Source Documentation 
The study may be subject to audit by the Sponsor, its designee, or by regulatory authorities. If 
such an audit occurs, the investigator must agree to allow access to required subject records. The 
investigator should notify the Sponsor promptly of regulatory authority audits that are scheduled 
and must forward copies of any findings or audit reports to the Sponsor promptly. 
By signing this protocol, the investigator grants permission to personnel from the Sponsor, its 
representatives, and appropriate regulatory authorities for on-site monitoring and review of all 
appropriate study documentation, as well as on-site review of the procedures employed in data 
collection, where clinically appropriate. 
14.10 
Study or Study Site Termination 
The Sponsor may suspend or stop the study at all centers or at specific study centers due to (but 
not limited to) the discovery of an unexpected, serious, or unacceptable risk to the subjects 
enrolled in the study, a decision on the part of the Sponsor to suspend or discontinue 
development of the product, failure of the investigator to enroll subjects into the study at an 
acceptable rate, failure of the investigator to comply with regulatory authority or ICH 
Guidelines, or submission of knowingly false information. 


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14.13 
Quality Assurance 
Authorized representatives of the Sponsor, a regulatory authority, or IRB/IEC may visit the study 
site to perform audits or inspections, including source data verification. The purpose of any such 
audit or inspection is to systematically and independently examine all study-related activities and 
documents to determine whether these activities were conducted, and data were recorded, 
analyzed, and accurately reported according to the protocol, ICH GCP guidelines, and any other 
applicable regulatory requirements. 
The investigator should contact the Sponsor immediately if contacted by a regulatory agency 
about an inspection. 
It is important that the investigator and relevant personnel are available during the possible audits 
or inspections and that sufficient time is devoted to the process. 


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14.14 
Retention of Data 
When the study is completed, the investigator must retain the essential documents for as long as 
needed (up to 25 years) to comply with regulatory guidelines and Sponsor requirements. The 
investigator will notify the Sponsor prior to moving or destroying any of the study documents. It 
is the responsibility of the Sponsor to inform the investigator/institution as to when these 
documents no longer need to be retained. The investigator should take measures to prevent any 
accidental or premature destruction of these documents. 
14.15 
Study Report and Publications 
A clinical study report will be prepared and submitted to the appropriate regulatory agency or 
agencies. The Sponsor will ensure that the report meets the standards set out in the ICH 
Guideline for Structure and Content of Clinical Study Reports (ICH E3). Note that an 
abbreviated report may be prepared in certain cases. 
Investigators in this study may communicate, orally present, or publish in scientific journals or 
other scholarly media only after written consent has been obtained from the Sponsor. 
The investigator will submit to the Sponsor any proposed publication or presentation along with 
the respective scientific journal or presentation forum at least 60 days before submission of the 
publication or presentation.  
No such communication, presentation, or publication will include the Sponsor’s confidential 
information (see Section 14.5). 
The investigator will comply with the Sponsor’s request to delete references to its confidential 
information (other than the study results) in any paper or presentation and agrees to withhold 
publication or presentation for an additional 60 days in order to obtain patent protection if 
deemed necessary. 


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SAP_001.pdf: 
 
 
 
Study Title: 
A Phase 1b, Multiple Ascending-dose Study of EQ001 in 
Subjects with Systemic Lupus Erythematosus with or 
without Active Proliferative Lupus Nephritis 
Protocol Number: 
EQ001-19-002 
 
Investigational Product(s): 
EQ001 
 
Sponsor: 
Equillium, Inc. 
 
Development Phase: 
Phase 1b 
 
NCT Number: 
NCT04128579 
 
Document Version Date: 
 
17 January 2024 
 
 
 
 
CONFIDENTIAL 
 
Information contained in this protocol is proprietary and confidential in nature and may not be used, divulged, published, or 
otherwise disclosed to others except to the extent necessary to obtain approval of the Institutional Review Board/Independent 
Ethics Committee, or as required by law. Persons to whom this information is disclosed should be informed that this information 
is confidential and may not be further disclosed without the express permission of Equillium AUS Pty Ltd. 


nfidential. 
Page 1 of 55 
Statistical Analysis Plan 
Sponsor Name: Equillium Inc. 
Protocol Number: EQ001-19-002 
 
Protocol Title: A Phase 1b, Multiple Ascending-dose Study of EQ001 in Subjects with 
Systemic Lupus Erythematosus with or without Active 
Proliferative Lupus Nephritis 
 
 
 
 
 
Notice of Confidential and Proprietary Information: 
The information contained in this document is confidential belonging to Equillium Inc. Acceptance of this 
document constitutes agreement by the recipient that no information contained herein will be published or 
disclosed without prior written authorization from an official of Equillium Inc. However, this document may 
be disclosed to appropriate Institutional Review Board and Ethics Committees or duly authorized 
representatives of a national regulatory authority under the condition that they are requested to keep it 
confidential. In the event of an actual or suspected breach of this obligation, Syneos Health should be 
notified promptly.   
 
 
 
 
 
 


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The final analyses of safety and efficacy data are planned after all subjects complete the study (or 
terminate early from the study). Final analyses were planned to be done in two stages; when Type A 
subjects complete the study and when Type B subjects complete the study. After Type A subjects 
completed the study, a database freeze was performed and an analysis was conducted on data collected 
for all Type A subjects. Once Type B subjects complete the study and database is locked, a final analysis 
will be conducted using all data collected in the database through the time of the database lock.  








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4
Endpoints
4.1 
Primary Endpoint 
The primary endpoint of the study is the safety and tolerability of EQ001, as assessed by treatment-
emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), clinical 
laboratory values, vital signs, 12-lead electrocardiogram (ECG), and physical examinations. 
4.2 
Secondary Endpoints 
The secondary endpoints of the study are: 
 
Pharmacokinetics of EQ001, as assessed by EQ001 serum concentrations at specified 
timepoints 
4.3 
Exploratory Endpoints 
 
For Type B cohort subjects: Change from baseline in the Short-Form 36 Health Survey (SF-36) 
and FACIT-F (Additional Concerns domain) total score to FU1 and FU2 and change from 


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baseline in the Lupus Quality of Life (QoL), patient global assessment, and physician global 
assessment (PGA) to FU2 
 


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5
Analysis Populations
5.1 
Safety Population  
The Safety Population consists of all subjects who receive any study drug. All safety data summaries will 
use this population. 
5.2 
PK Population 
The PK Population consists of subjects in the Safety Population who have at least one measurable post-
EQ001 exposure serum concentration. 
5.4 
Efficacy Population 
The Efficacy Population consists of subjects in the Safety Population who have at least one post-
treatment assessment for a marker of clinical activity. Note that for Type B subjects, only post-treatment 
within 14 weeks of last treatment will be considered for the purpose of defining the Efficacy Population. 
 


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6
General Considerations
6.1 
General Methods 
Statistical analysis of data from this study will be performed using SAS version 9.4 or higher. 
Summaries of baseline characteristics (including demographics), safety, PD parameter receptor 
occupancy, and efficacy will be presented for each EQ001 dose level within each Type A cohorts and the 
Type B cohort as applicable. Additionally, data will also be summarized for all Type A subjects combined. 
A given dose may be studied in more than one cohort of Type A subjects and/or in Type B subjects. 
Selected safety summaries will also be produced that combine like doses across Type A and B cohorts. 
Groups Presented for Baseline Characteristics, Safety, PD, and Efficacy Data Summaries 
Type A Cohorts 
 
Dose 1 
 
Dose 2 
 
Dose 3 
 
Dose 4 
 
Dose 5 
 
All doses combined 
Type B Cohort 
 
Dose 1 
Type A + Type B Cohort 
Selected summaries that combine doses across Type A and B cohorts will be produced for the following: 
Adverse events by systemic organ class (SOC) and preferred terms, adverse events of special interest, 
serious adverse events, and adverse events resulting in study drug discontinuation. Like doses from Type 
A and Type B will be pooled, eg., Type A Dose 1.6 mg/kg combined with Type B Dose 1.6 mg/kg. 
As the primary objective of this Phase 1b study is to characterize the safety and tolerability of EQ001, no 
formal statistical hypothesis testing will be performed. Confidence intervals (CIs) for selected clinical 
activity parameters may be presented for exploratory purposes only.  
Most statistical summaries will be descriptive in nature. Unless otherwise indicated, continuous variables 
will be summarized using the number of observations (n), mean, standard deviation (SD), median, 
minimum, and maximum and categorical variables will be summarized using number of observations (n), 
frequency and percentages of subjects. 
All dosed subjects will be included in subject data listings unless otherwise noted. If applicable, the 
relative day (i.e., relative to date of first dose of study treatment) of all complete dates will be presented in 
subject listings.  
Data collected at unscheduled visits will also be included in the subject listings. 
Adverse events, medical and surgical histories will be coded for summarization using the Medical 
Dictionary for Regulatory Activities (MedDRA® Version 23.1). Concomitant medications will be coded 
using the World Health Organization (WHO) Drug Dictionary Global B3 (March 2019 Version). 




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7.6.1 
Prior Medications 
Prior medications are defined as those medications with a start date prior to the first dose date of any 
study drug. Prior medications will be summarized in a table and presented in a by-subject listing.  


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7.6.2 
Concomitant Medications 
Concomitant medications are defined as any medications taken on or after the date of first dose of any 
study drug, including any medications that were started before the first dose date of any study drug and 
are ongoing. Concomitant medications will be summarized descriptively and presented in a by-subject 
listing. 
Medications can be both prior and concomitant depending on the start and stop date as related to the first 
dose date of any study drug. 
7.6.3 
Induction Treatments 
Summary of induction treatments is provided for Type B subjects only using the Safety Population. Time 
from last induction regimen to first dose (months) and time from last best response to first dose (months) 
will be summarized as continuous variables.  Time from last induction treatment start date to first dose is 
calculated as (first dose date – start date of last induction treatment) / 30.4375, rounded to one decimal 
place. Time from last best response to first dose is calculated as (first dose date – end date of the last 
best response) / 30.4375, rounded to one decimal place. Best response and the last induction regimen 
will be summarized as categorical variables.   
Complete response or partial response are considered as best response for the calculation of time from 
last best response to first dose. Complete Response > Partial Response > No Response > Ongoing > 
Unknown where ‘>’ symbol indicates ‘better than’ and ‘Ongoing’ indicates that the treatment is ongoing. 
7.6.4 
Systemic Steroid Use 
All systemic steroid use will be summarized by ATC2 and ATC4 using the Safety Population. Subjects will 
be counted only once for each ATC2 term in the event that they have multiple records of the same ATC 
level in the database. The tables will be sorted in the descending order of frequency ATC2 terms and 
within each ATC2, descending order of frequency of ATC4 in the Total column (all subjects pooled). By-
subject listings will also be created.    
7.6.5 
 Systemic Lupus Erythematosus Treatments  
The most recent use of prior systemic lupus erythematosus treatments will be summarized by drug/drug 
class as collected in the CRF and by use (never used, discontinued >6 months ago, discontinued <= 6 
months ago, ongoing, unknown) using the Safety Population. By-subject listings will also be created. 
 




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8.1 
Secondary Endpoints Analyses 
8.1.1 
Fold Change from Baseline in UPCR to Follow-Up Visit 2 (FU2)  
This secondary endpoint of fold change in 24-hour UPCR from baseline pertains to Type B subjects only. 
Fold change from baseline is defined as UPCR value at visit/Baseline UPCR value. UPCR data are 
collected from central and local laboratories for Type B subjects. They are generally left-skewed. UPCR 
will be analyzed using central and local data combined (as defined in Section 6.2). 
The fold change from baseline in UPCR from a 24-hour urine collection to FU2 will be descriptively 
summarized using geometric statistics (geometric mean, geometric CV, 95% confidence interval of 
geometric mean etc.). The fold change from baseline over time by visit will also be summarized 
descriptively and geometric mean and 95% confidence limits will be graphically displayed by visit. 
As a sensitivity analysis, the 24-hour UPCR values collected more than 56 days after the last treatment 
date, or after a subject received new immunosuppressive or other treatments for LN, or after subject had 
an increase in glucocorticoids will not be included and fold changes from baseline will summarized 
descriptively as mentioned above.  
The proportion of subjects with a > 30% and > 50% decline in UPCR from a 24-hour urine collection and 
those with a UPCR < 500 mg/g at FU2 will be described, along with their 2-sided exact Clopper-Pearson 
95% CIs.  


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Time to earliest response of 24-hour UPCR with response defined as a decrease of > 50% from baseline, 
a decrease of > 30% from baseline, and a decrease to a level < 500 mg/g for each of these cases will be 
summarized using the Kaplan-Meier method. The baseline will be the derived 24-hour UPCR value (as 
defined in Section 6.2). Post-treatment UPCR values from both scheduled and unscheduled visits will be 
considered. Subjects who have not had a response at the end of participation will be censored on the 
date of end of participation.  
For both Type A and Type B Cohort subjects, the fold change from baseline in spot UPCR will be 
descriptively summarized using geometric statistics (geometric mean, geometric CV etc.).  The proportion 
of subjects with a > 30% and > 50% decline in spot UPCR and those with a spot UPCR < 500 mg/g by 
visit will be described, along with their 2-sided exact Clopper-Pearson 95% CIs specifically provided for 
FU1 for Type A subjects and FU2 for Type B subjects. 
In addition, an exploratory analysis of spot UPCR will be performed for Type A cohorts. In this analysis, 
two-sided p-value to compare values from FU1 and baseline will be obtained by testing LS mean 
associated with each dose resulting by fitting an ANCOVA model equals to 0. ANCOVA model will be 
fitted to the log-transformed fold change data with EQ001 dose level as a factor and log-transformed 
baseline spot UPCR as a covariate for Type A cohorts.   
8.1.2 
Change from Baseline to FU1 and FU2 in Serologic Markers (C3, C4, and anti-dsDNA) 
This endpoint pertains to both Type A and Type B Cohort subjects.  
The mean change from baseline in C3, C4, and anti-dsDNA to FU1 (Type A Cohorts [exploratory 
endpoint]) and FU2 (Type B [secondary endpoint]) will be summarized, along with their 2-sided 95% CI 
for the change. For data summarization purposes, if a value is recorded as <x or <=x, the value is 
considered as x/2. If a value is recorded as >x or >=x, the value is considered as x. For Type A, the 2-
sided 95% CI will be presented by fitting an ANCOVA model, that is, an ANCOVA model on the changes 
in C3 and C4, from baseline to FU1 with EQ001 dose as a factor and baseline value as a covariate will be 
fitted. Baseline adjusted LS mean changes from each group will also be reported.  
Serologic markers are collected from central and local laboratories for Type B cohort. Same tests with 
both central and local version will be combined and analyzed, using descriptive summary statistics by 
assessment visit. For site 8xx subjects, only local laboratory data (may come from different local 
laboratories) will be used for analysis; for non-site 8xx subjects, only central laboratory data will be used 
for analysis. 
8.1.3 
Change from Baseline in eGFR to FU1 and FU2 
This secondary endpoint pertains to Type B cohort subjects only. 
The change from baseline in eGFR (baseline value is defined as the mean of Screening and Day 1 
values) will be descriptively summarized by visit with central and local versions combined.   
The proportion of subjects with a percent decline in eGFR from baseline to FU2 of >=20%, >=30% and 
>=40% will be summarized along with their 2-sided Clopper–Pearson 95% CIs.  In addition, these 
proportions will also be summarized for all visits. 
8.1.4 
Change from Baseline in Daily Prednisone Dose and Cumulative Prednisone Dose Through FU1 
and Through FU2 
This secondary endpoint pertains to Type B subjects only. 








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Sponsor at an unscheduled visit to further evaluate safety in individual subjects.  ADA and neutralizing ADA 
results will be presented in a by-subject listing for the Safety Population with Type A and Type B combined. 


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10 Safety
All safety analyses will be performed using the Safety Population.
10.1 Extent of Exposure 
All subjects start with dose level of 1.6 mg/kg but allowed to change the dose level to 0.8 mg/kg. Dose 
level administrated at each visit will be summarized. Treatment exposure will be determined by overall 
duration of treatment and duration at each dose level. In addition, measures of extent of exposure at each 
dose level include the screening weight normalized average dose of study drug (mg/kg) administered, 
total dose of study drug (mg/kg) administered, and the number of doses administered per subject. 
The duration of exposure in weeks is defined as the (last dose date - first dose date + 1)/7. 
eCRFs captures the volume of EQ001 prepared and volume administered at each dosing visit. As 
described in the Pharmacy Manual, volume prepared at a dosing visit is based on the subject’s weight 
(kg) and the randomized dose level (mg/kg); i.e., volume prepared (mL) = weight x dose level /100. The 
weight at screening is used to calculate the study drug dose, unless there has been a weight change of 
20% or more from the screening weight, then the weight at visit is used. 
The screening weight normalized average dose of study drug (mg/kg) administered at a dosing visit = 
total volume of study drug administered at all dosing visits (mL) x (100 mg/1 mL)/ (the screening weight x 
number of doses administered).   
Duration of exposure and screening weight normalized average dose of study drug administered per dose 
will be summarized by continuous summary statistics (n, mean, standard deviation (SD), median, 
minimum, and maximum). The number and percentage of doses administered will be summarized by 
treatment group in each Type A and Type B cohort separately. 
Cumulative volume administered will also be summarized by continuous summary statistics by treatment 
group in each Type A and Type B cohort separately. 
10.2 Treatment Compliance  
Overall treatment compliance will be calculated based on the cumulative volume of study drug 
administered (ml) and the cumulative volume of study drug prepared (ml).  
Compliance (%) = 100 x (cumulative volume of drug administered / cumulative volume of drug prepared).  
Treatment compliance will be summarized by continuous summary statistics (n, mean, standard deviation 
(SD), median, minimum, and maximum) by treatment group in each Type A and Type B cohort 
separately. 
A by-subject listing of exposure and compliance data will also be created.  
10.3 Adverse Events 
An adverse event is defined as any untoward medical occurrence in a subject. Treatment-emergent AEs 
(TEAE) are defined as any AE that started after dosing or prior to dosing and that worsens following 
exposure to the study drug. If the start date of an AE is partially or completely missing, the date will be 
compared as far as possible with the date of the start of administration of study drug. The AE will be 
assumed to be treatment-emergent if it cannot be definitively shown that the AE did not occur during the 
treatment-emergent period (worst case approach; see Section 6.3 for imputation algorithm).




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TEAEs of Special Interest by SOC  PT and Maximum Severity Grade
A by-subject listing of AE data will also be created. Adverse events occurring within 30 days after last 
dose of study drug and AEs occurring >30 days after the last dose of study drug through the end of the 
study will be separately summarized. All AEs will be included in the listings. AEs occurring before start of 
study treatment will be included in the data listings but will not be included in the summary tables of AEs. 
10.4 Laboratory Evaluations 


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The actual and change from Baseline values of clinical laboratory data (hematology, chemistry, and 
urinalysis) will be summarized by visit presented in a table, using continuous descriptive summary 
statistics at Baseline and each post-Baseline visit. For Type B, laboratory tests with both local and central 
versions will be combined and analyzed.  
Laboratory parameters that can be graded using CTCAE will be graded. The number and percentage of 
subjects will be summarized for the following:   
 
Grade 2 and Grade 3 or higher laboratory abnormalities 
 
Worst post-Baseline severity grade 
 
Shift summary of Baseline grade to worst post-Baseline severity grade 
 
ALT or AST >=3xULN  
 
ALT or AST >=3x ULN and total bilirubin > 2x ULN  
Box plots of selected hematology (hemoglobin, absolute lymphocyte count, absolute neutrophil count, 
platelets, white blood cell count) and chemistry parameter values (sodium, potassium, eGFR, ALT, AST) 
will be produced by assessment visit.  
Mean +/- standard error (SE) figures will be provided for serum albumin, spot UPCR, 24-hour UPCR, spot 
UACR, 24-hour UACR, eGFR and Spot UPCR, spot UPCR and 24-hour UPCR, and spot UACR and 24-
hour UACR overtime, for Type-B subjects only.  
A by-subject listing will also be created for laboratory results in conventional units.   
A by-subject laboratory findings prior to induction listing will also be created for prior to induction 
laboratory results in conventional units.   
10.5 Pregnancy 
Pregnancy testing in women of childbearing potential will be obtained with a urine and serum beta human 
1, Day 15, 
Day 29, Day 43 and at Day 57 visit in Type A subjects and during Screening, Day 1, Day 15, Day 29, Day 
43, Day 57, Day 71, Day 85, Day 99, Day 113, Day 127, Day 141, Day 155, Day 169, FU2, FU3, and FU4 
for Type B subjects. 
Pregnancy test results, including the type of pregnancy test performed at visit will be presented in a by-
subject listing.  
10.6 Vital Signs  
Vital signs will be collected pre-dose and 30 minutes after the injection at each dosing visit.  For Type B 
subjects, vital signs will be measured on 2 different days at least 3 days apart at Screening.  Additionally 
for Type B subjects, blood pressure will be measured twice at least 3 days apart at Screening.  Vital signs 
will include systolic and diastolic blood pressures (mmHg), pulse rate (beats/min), respiratory rate 
(breaths/min), temperature (°C or °F) and weight (kg). 
Temperature collected in °F will be converted into °C using the following formula: 
Temperature (°C) = [Temperature (°F) – 32] x (5/9)  


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The actual and change from Baseline values of vital signs will be summarized by visit presented in a 
table, using continuous descriptive summary statistics at Baseline and each post-Baseline visit. In 
addition, number and percent of subjects with out of range vital signs at any post-baseline visit will also 
be summarized for the following categories:  
Blood Pressure:   
 
SBP > 150 mmHg or DBP > 100 mmHg  
 
SBP <90 mmHg or DBP <60 mmHg  
Heart Rate:   
 
< 50 beats per min  
 
>120 beats per minute  
 
 30 beats per minute increase from baseline  
 
 30 beats per minute decrease from baseline  
 
A by-subject listing will also be created.  
10.7 ECG 
A standard 12-lead ECG will be performed at Screening, Day 1, Day 29, FU2/Day 57, and Early 
Termination for Type A Cohort Subjects and at Screening, Day 1, Day 43, Day 85, Day 141, Day 169, 
FU4/Day 253, and Early Termination for Type B Subjects. The subjects will be in a seated, semi-
recumbent, or supine position in a rested, calm state for at least 10 minutes before ECG assessment is 
performed. Any clinically significant abnormal ECG findings will be recorded as an AE. 
The actual and change from Baseline values of ECG parameter HR and intervals (PR, RR, QRS, QTcF)  
will be summarized by visit presented in a table, using continuous descriptive summary statistics at 
Baseline and each post-Baseline visit. ECG overall interpretation will be summarized by visit as a 
categorical variable (normal, abnormal – not clinically significant, abnormal – clinically significant).  
QT interval corrected for heart rate will be calculated using the Fridericia’s formula: QTcF=10xQT/RR1/3, 
where RR represents RR interval measured in milliseconds. 
Worst post-baseline increase in QTcF by visit will be summarized for the following: 
 
<= 0 msec increase from baseline  
 
>0 - <= 30 msec increase from baseline 
 
>30 - <= 60 msec increase from baseline 
 
>60 msec increase from baseline 
Outlier ECG intervals will be summarized for the following: 
 
PR > 200 msec 
 
HR > 120 or < 40 bpm 
 
QTcF increased by >30 msec from baseline 
 
QTcF increased by >60 msec from baseline 
 
QTcF > 480 msec 
 
QTcF > 500 msec 
A by-subject listing will also be created.