Conditions: Follicular Lymphoma (FL) Non Hodgkin Lymphoma Marginal Zone Lymphoma
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 169
Healthy volunteers: no

Brief summary:
This is the study of the PI3Kδ inhibitor Zandelisib (ME-401) in subjects with relapsed/refractory follicular lymphoma or marginal zone lymphoma after failure of at least 2 prior lines of systemic therapy

Detailed description:
This is a global, multicenter open-label, single-arm, Phase 2 study of the PI3Kδ inhibitor Zandelisib (ME-401) in subjects with relapsed/refractory follicular lymphoma or marginal zone lymphoma after failure of at least 2 prior lines of systemic therapy which must have included an anti-CD20 antibody and chemotherapy with an alkylating agent or a purine analogue.

Approximately 180 subjects will be enrolled and treated with Zandelisib (ME-401) on the intermittent schedule.

Arms:
arm 1: Subjects with relapsed/refractory FL or MZL will be administered 60 mg of ME-401 orally, once a day on an intermittent schedule (IS).

Interventions: intervention 1: Zandelisib (ME-401)
Intervention descriptions: intervention 1: Zandelisib (ME-401) 60 mg

Protocol document:
Prot_000.pdf: 
CONFIDENTIAL 
This document is confidential and is the property of MEI Pharma, Inc.  No part of this document may be transmitted, 
reproduced, published, or used by other persons without written authorization from MEI Pharma, Inc. 
TITLE PA GE 
A Multicenter, Open-Label, Single-Arm, Phase 2 Study of Zandelisib 
(ME-401) in Subjects with Follicular Lymphoma or Marginal Zone 
Lymphoma After Failure of Two or More Prior Systemic Therapies – 
The TIDAL Study 
Protocol Number: ME-401-003 
Sponsor: MEI Pharma, Inc. 
11455 El Camino Real, Suite 250 
San Diego, CA 92130 USA 
Sponsor Contact: Richard Ghalie, MD 
Chief Medical Officer  
MEI Pharma, Inc. 
Phone: +1 858 369 7116 
rghalie@meipharma.com 
Medical Monitor: Richard Ghalie, MD 
Chief Medical Officer 
MEI Pharma, Inc. 
Mobile: +1 619 990 1153 
rghalie@meipharma.com 
Clinical Research 
Organization: 
Syneos Health 
1030 Sync Street 
Morrisville, NC 27560 USA 
Investigational Drug: ME-401 
EudraCT Number: 2018-002896-17 
IND Number: 129,845 
Original Protocol: 25 July 2018 
Amendment 1: 09 October 2018 
Amendment 2: 05 December 2019 
Amendment 3: 24 August 2020 
Amendment 4: 08 June 2021 
Amendment 5: 18 October 2022 
NCT 03768505


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SPONSOR’S PROTOCOL SIGNATURE PAGE 
By signing below, the Sponsor declares that this study will be conducted in accordance with 
current United States (US) Food and Drug Administration Code of Federal Regulations, Good 
Clinical Practice (GCP) standards, the Declaration of Helsinki (Brazil 2013), and local ethical 
and legal requirements. 
Richard Ghalie, MD  
Date 
Chief Medical Officer 
31-Oct-2022 | 10:50 PDT


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INVESTIGATOR’S SIGNATURE PAGE 
By signing below, the Investigator agrees to adhere to the protocol as written and agrees that any 
changes to the protocol must be approved by MEI Pharma, Inc. before seeking approval from the 
Institutional Review Board (IRB)/Ethics Committee (EC). 
The study will be conducted in accordance with the current International Council for 
Harmonisation (ICH) Guidelines, the Guidelines for Good Clinical Practice (GCP), and local 
ethical and regulatory requirements. 
The information contained in this protocol is proprietary and provided to me in confidence, 
and may not be disclosed to any other party, in any form, without prior authorization from 
MEI Pharma, Inc., except to the extent necessary for the conduct of the study at this study site. 
Principal Investigator: 
 
 
 
 
 
Signature 
Date 
 
 
 
 
 
Printed name 
 
 
 
 
 
 
Institution 
 
 


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PROTOCOL SYNOPSIS 
TITLE 
A Multicenter, Open-Label, Single-Arm, Phase 2 Study of Zandelisib (ME-401) in Subjects 
with Follicular Lymphoma or Marginal Zone Lymphoma After Failure of Two or More Prior 
Systemic Therapies – The TIDAL Study 
PROTOCOL NUMBER 
ME-401-003 
STUDY DURATION 
Individual subject participation in the study will include 28 days for screening, approximately 
18 months of dosing with study drug, and follow-up for survival after discontinuation of study 
drug. Actual subject participation in the study may be shorter or longer depending on disease 
response, tolerability to therapy, and duration of long-term follow-up. 
For the follicular lymphoma (FL) group, the study will continue until all enrolled subjects in 
the Primary Efficacy Population (PEP) (except those who discontinue early) have been 
followed for approximately 14 months from the first dose of study drug. For the marginal zone 
lymphoma (MZL) group, the study will continue until all enrolled subjects (except those who 
discontinue early) have been followed for approximately 14 months from the first dose of 
study drug. 
SUBJECT POPULATION 
Subjects with relapsed/refractory FL or MZL after failure of at least two prior systemic 
therapies. 
INVESTIGATIONAL PRODUCTS 
Zandelisib, (code ME-401); a phosphatidylinositol-4,5-biphosphate 3-kinase delta (PI3Kδ) 
inhibitor 
PRIMARY OBJECTIVE 
• To evaluate the objective response rate (ORR) of ME-401 in relapsed or refractory FL 
or MZL, based on the Modified Lugano Response Criteria (Appendix 5), and 
determined by an Independent Response Review Committee (IRRC) 
SECONDARY OBJECTIVES 
• To evaluate the efficacy of ME-401 as assessed by an IRRC: 
o Duration of response (DOR) 
o Complete response (CR) rate 
o Progression-free survival (PFS) 
o Time to treatment failure (TTF)  


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• To evaluate the efficacy of ME-401 as assessed by the Investigator: 
o Objective response rate (ORR) 
o Duration of response (DOR)  
o Complete response (CR) rate 
o Progression-free survival (PFS) 
o Time to treatment failure (TTF)  
o Recapture of response 
o Duration of recaptured response (DORR)  
• To evaluate the overall survival (OS) 
• To evaluate the safety profile of ME-401 
o Overall incidence of treatment-emergent adverse events (TEAEs) 
o Incidence of adverse events of special interest (AESIs) 
o Time to occurrence of AESIs 
• To evaluate the pharmacokinetics (PK) of ME-401 
SUBJECT ELIGIBILITY 
Inclusion Criteria 
1. Signed informed consent. 
2. Age ≥18 years (or age of majority). 
3. Histologically confirmed diagnosis as defined in the World Health Organization 
(WHO) classification (Swerdlow 2016) of: 
a. FL limited to Grade 1, 2, or 3a; or  
b. MZL, including nodal, extranodal, and splenic MZL  
(histopathological report confirming diagnosis must be available during screening 
procedures). 
4. Subjects with relapsed or refractory FL or MZL who received ≥2 prior therapy 
regimens. A previous regimen is defined as one of the following: at least two months 
of single-agent therapy or at least two consecutive cycles of polychemotherapy, 
autologous transplant, or radioimmunotherapy. Prior therapy must include an 
anti-CD20 monoclonal antibody (mAb) and an alkylating agent(s). Relapsed or 
refractory disease is defined as:  
a. Relapsed disease: disease progression after a response (CR or PR) lasting 
≥6 months  
b. Refractory disease: no response to therapy (no CR or PR) or response lasting 
<6 months 


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5. At least one bi-dimensionally measurable nodal lesion >1.5 cm or extranodal lesions 
>1 cm in its longest diameter by computed tomography (CT) scan as defined by the 
Modified Lugano Classification (Appendix 5).  
a. Previously irradiated lesions can be selected as target lesions only in cases of 
unequivocal evidence of progression 
b. For subjects with splenic MZL only: diffuse spleen involvement with 
splenomegaly, which is defined as the splenic vertical length greater than 13 cm. 
6. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1 
(Oken 1982; Appendix 6). 
7. Adequate hematologic parameters at screening unless abnormal values are due to 
lymphoma per Investigator assessment: 
a. Absolute neutrophil count (ANC) ≥1.0 × 109/L (≥1,000/mm3) 
b. Platelet count ≥75.0 × 109/L (≥75,000/mm3) 
8. Adequate renal and hepatic function per local laboratory reference range at screening 
as follows: 
a. Aspartate aminotransferase (AST)/serum glutamic-oxaloacetic transaminase 
(SGOT), alanine aminotransferase (ALT)/serum glutamic-pyruvate transaminase 
(SGPT) ≤3.0 × upper limit of normal (ULN) 
b. Total bilirubin ≤2.0 × ULN or ≤3 × ULN for subjects with Gilbert’s syndrome  
c. Serum creatinine ≤1.5 × ULN or estimated glomerular filtration rate (eGFR) 
>50 mL/min using the Cockcroft-Gault equation (Appendix 2) 
9. QT-interval corrected according to Fridericia’s formula (QTcF) ≤450 milliseconds 
(msec); subjects with QTc >450 msec but <480 msec may be enrolled provided the 
QTc prolongation is due to a right bundle branch block (RBBB), left bundle branch 
block (LBBB), or pacemaker and is confirmed stable by a cardiologist. 
10. Left ventricular ejection fraction (LVEF) ≥45% as measured by echocardiogram or 
multigated acquisition scan (MUGA). If LVEF <45% by ECHO, a repeat measurement 
can be conducted within the screening period. 
11. Subjects must have completed any prior systemic anti-cancer treatment within 
≥4 weeks of Cycle 1 Day 1 (or ≥5 times the half-life [t½], whichever is longer); 
≥8 weeks for antibody agents; ≥2 weeks for radiation therapy; and ≥3 months for high 
dose therapy with stem cell transplantation or CAR T-cell therapy or 
radioimmunotherapy. 
12. All adverse events (AEs) and laboratory toxicities related to prior therapy must resolve 
to Grade ≤1 prior to the start of the study therapy (unless otherwise specified in 
eligibility criteria).  
13. For females of childbearing potential, a negative serum human chorionic gonadotropin 
(hCG) pregnancy test within 28 days of study Day 1 and negative hCG result on study 
Day 1. 


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14. Subjects must agree to use appropriate contraception methods during the clinical study 
(Appendix 4).  
15. Subject is willing and able to comply with all scheduled visits, treatment plans, 
laboratory tests, and other study procedures. 
Exclusion Criteria 
1. Histologically confirmed FL Grade 3b, or transformed disease (assessed by the 
Investigator): 
a. For patients with clinical (e.g., marked B-symptoms), laboratory (e.g., high lactate 
dehydrogenase [LDH]) or radiographic (e.g., high standardized uptake value by 
positron emission tomography [PET]) signs of rapid disease progression, a fresh 
tumor biopsy prior to enrollment is required to rule out transformed disease. 
2. Known lymphomatous involvement of the central nervous system. 
3. Major surgical procedure within 4 weeks prior to study Day 1 (minor surgical 
procedures, [e.g., lymph node biopsy] performed within 1 day or with an overnight 
stay are allowed). 
4. Prior therapy with PI3K inhibitors. 
5. Any uncontrolled clinically significant illness including, but not limited to, active 
infections requiring systemic antimicrobial therapy, hypertension, angina, arrhythmias, 
pulmonary disease, or autoimmune dysfunction. 
6. Subjects who have tested positive for hepatitis B surface antigen and/or hepatitis B 
core antibody plus have a positive hepatitis B polymerase chain reaction (PCR) assay; 
subjects who have previously tested positive with a negative PCR assay are permitted 
with appropriate anti-viral prophylaxis. 
7. Positive hepatitis C virus antibody (HCV Ab); subjects with positive HCV Ab are 
eligible if they are negative for HCV by PCR. 
8. Known history of, or active human immunodeficiency virus (HIV) infection. 
9. Ongoing or history of drug-induced pneumonitis. 
10. Previous or concurrent cancer that is distinct in primary site or histology from indolent 
B cell non-Hodgkin’s lymphoma (iNHL) within 3 years before start of study treatment 
except for curatively treated cervical cancer in situ, non-melanoma skin cancer, 
superficial bladder tumors (Ta [non invasive tumor], Tis [carcinoma in situ], and T1 
[tumor invades lamina propria]), and asymptomatic localized prostate cancer with no 
requirement for systemic therapy (or requiring only hormonal therapy) and with 
normal prostate-specific antigen values within ≥12 months prior to enrollment. 
11. History of clinically significant cardiovascular abnormalities such as congestive heart 
failure (New York Heart Association classification ≥II [NYHA 1994]), myocardial 
infarction within 6 months of study entry. 


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12. History of clinically significant gastrointestinal (GI) conditions, particularly: 
a. Known GI condition that would interfere with swallowing or the oral absorption or 
tolerance of study drug 
b. Pre-existing malabsorption syndrome or other clinical situation that would affect 
oral absorption 
13. Females who are pregnant; females who plan to breastfeed during study treatment 
through 90 days after ending treatment. 
14. Psychiatric illness/social situations that would interfere with study compliance. 
15. Hypersensitivity or other clinically significant reaction to the study drug or its inactive 
ingredients. 
16. Any other condition for which, in the opinion of the Investigator, participation would 
not be in the best interest of the subject. 
STUDY DESIGN 
This is a global, multicenter, open-label, single-arm, Phase 2 study of the PI3Kδ inhibitor 
ME-401 in subjects with relapsed/refractory FL or MZL. ME-401 will be administered orally 
at a dose of 60 mg given once a day. A cycle of treatment is 28 days in duration. Treatment 
with ME-401 is administered based on an intermittent schedule (IS).  
The IS includes: 
• continuous daily therapy for the initial 2 cycles of treatment 
• the intermittent treatment begins at Cycle 3, with ME-401 administered daily for the 
first 7 days of every 28-day cycle (7 days on treatment and 21 days off treatment)  
The first day of treatment is designated as Day 1. ME-401 will be administered until 
progressive disease (PD), development of unacceptable toxicity (despite ME-401 modified 
dosing schedule), or subject withdrawal.  
Review of the ongoing Phase 1b study evaluating different dosing schedules for ME-401 in 
subjects with relapsed/refractory FL showed improved risk-benefit profile of the IS over a 
continuous schedule (CS), i.e., ME-401 given daily continuously). With Amendment 2, the 
initial CS treatment arm was terminated, and subjects were enrolled to receive therapy on the 
IS only. If subjects already completed 2 initial cycles of therapy, they were switched to 
IS dosing once they completed therapy in the ongoing cycle of treatment, and if they had not 
yet completed the first 2 cycles of therapy they were switched to IS dosing after completion of 
the first two cycles of therapy. Subjects who progress on IS dosing may be switched to CS 
dosing. 
Upon confirmation of meeting eligibility criteria, subjects should be treated with ME-401 
within a reasonable time frame (within 5 days). Once subjects are treated with ME-401, they 
are defined as being enrolled in the study.  
The study schema is presented in Figure 1. 


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Figure 1: 
Study Schema 
 
For subjects who experience hematologic or non-hematologic adverse reactions, please refer to 
ME-401 toxicity management (Table 4) and modified dosing schedules in Section 5.3.1. 
ME-401 is provided as 60 mg capsules. ME-401 is to be taken orally once a day at 
approximately the same time each day in accordance with the IS regimen (or CS dosing 
regimen if switched from IS after PD). 
Subjects must receive prophylaxis treatment for Pneumocystis jirovecii pneumonia (PJP) as 
prescribed within this protocol (see Section 5.4.3). 
See the Schedule of Assessments (Appendix 1) for a detailed list of study procedures. 
The Modified Lugano 2014 criteria will be used for response assessment (Appendix 5). The 
Investigator’s assessment of disease response will be used for subject management. For the 
primary analysis, disease/response assessment will be performed by an IRRC blinded to 
Investigator assessment and treatment assignment. Methods for disease/response assessment 
by the IRRC are described in the IRRC Charter. Disease/response assessment will occur every 
2 months for the first 6 months, every 3 months during the next 12 months, and every 6 
months thereafter. 
Safety will be assessed by laboratory tests including hematology (complete blood count 
[CBC]), serum chemistry, and cytomegalovirus (CMV) serology and/or quantitative PCR; and 
clinical assessments including physical examination, vital signs, ECOG performance status, 


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and 12 lead electrocardiogram (ECG). Adverse events will be graded according to National 
Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0. 
Before treatment discontinuation due to any reason, including disease progression, the 
Investigator must review the reasons with the Sponsor’s Medical Monitor or designee.  
The End of Treatment (EOT)/30-Day Safety Follow-up visit will occur 30 (±3) days from the 
last dose of study drug. Subjects who discontinue study drug will remain in the study with 
follow-up conducted every 3 months from the last dose of study drug to obtain post-treatment 
long-term follow-up information on disease status (i.e., change in disease response, 
progression), start of subsequent therapy, and survival (death). Survival follow-up will 
continue for up to 3 years after the last subject is enrolled in the study.  
Plasma samples will be obtained from all subjects to evaluate ME-401 PK and for ME-401 
exposure-response and exposure-toxicity modeling. For subjects enrolled at United States 
(US) sites only, blood samples will be obtained for a correlative immune study. See Schedule 
of Assessments (Appendix 1) for blood sample collection. 
The primary analysis of ORR for FL will occur after the initial 91 consecutive FL subjects 
administered ME-401 by IS, which defines the PEP (except those who discontinue early), have 
been followed for at least 6 months from the start of study drug. 
The final analysis of DOR for FL will be triggered after most responders in the PEP have a 
minimum of 12 months of follow-up from first response. 
The final analysis of ORR and DOR for MZL will be triggered after most responders in the 
ITT MZL population have a minimum of 12 months of follow-up from first response. This 
usually takes place within the first 2 cycles; therefore, 14 months from start of treatment. 
Analysis projected to occur approximately 14 months after the first dose of study drug in the 
last subject enrolled in the MZL cohort. 
NUMBER OF SUBJECTS 
Approximately 120 subjects with FL and 32 subjects with MZL will be enrolled and treated 
with ME-401 on the IS. 
ESTIMATED NUMBER OF SITES 
Approximately 125 sites will be opened globally. 
STATISTICAL METHODOLOGY 
This is an open-label, single-arm study. The objectives of this study are to estimate the 
efficacy and safety of ME-401 administered using an IS dosing regimen in patients with 
relapsed or refractory FL or MZL. 
Sample Size for FL  
The study will test the null hypothesis that the IRRC-reviewed ORR is ≤48% against the 
alternative hypothesis that it is ≥65.5%. The sample size for the PEP for FL is estimated to be 
91 subjects, with at least 90% power and 1-sided alpha = 0.025. 


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With a total sample size of 120 FL subjects treated with ME-401 IS dosing regimen, if 
≤15 subjects experience Grade ≥3 AESIs (i.e., subject incidence rate of ≤12.5% with 95% CI 
of 7.2–19.8%), the study can rule out the possibility of having a incidence rate ≥20% for 
Grade ≥3 AESIs with the IS dosing regimen. In addition, with a total sample size of 120 FL 
subjects, if ≤5 subjects experience a particular type of AE (i.e., subject incidence rate of ≤4.2% 
with 95% CI 1.4-9.5%), the study can rule out the possibility of having an incidence rate 
≥10% for the AE. 
Sample Size for MZL 
Initially, the study was designed to test the null hypothesis that the IRRC-reviewed ORR is 
≤46% against the alternative hypothesis that it is ≥66.5%. The sample size for the MZL 
population was estimated to be 64 subjects, with at least 90% power and 1-sided 
alpha = 0.025. Enrollment in the MZL arm was closed by the Sponsor on 31 August 2022, 
with 32 of the 64 planned subjects enrolled. A sample of 32 subjects will provide preliminary 
data on the efficacy and safety of single agent zandelisib in relapsed/refractory MZL. 
Statistical Analysis 
The ITT FL Population includes all FL subjects randomized per Protocol Amendment 1 (two 
arm design with randomization), and all enrolled FL subjects who received at least one dose of 
ME-401 per Protocol Amendment 2 or later amendment (single-arm, open-label design).  
The ITT IS FL Population is defined as all FL subjects who are intended to receive ME-401 IS 
regimen, including subjects who:  
• Were randomized to the ME-401 IS dosing regimen group (Group B) per 
Protocol Amendment 1  
• Were randomized to the ME-401 CS dosing regimen group (Group A) per 
Protocol Amendment 1, but re-consented to Protocol Amendment 2 (for IS dosing 
regimen) prior to or on their Cycle 3 visit date 
• Were enrolled per Protocol Amendment 2 or later amendment who received ME-401 
IS dosing regimen.  
The ITT CS Group is defined as all FL subjects who are randomized to the ME-401 CS 
regimen group (Group A) per Protocol Amendment 1 and did not switch/plan to switch to 
IS dosing regimen at Cycle 3 per Protocol Amendment 2. 
The PEP in FL is defined as the initial 91 consecutive subjects enrolled in the ITT IS 
FL Population.  
The primary analysis of ORR for FL will be triggered after all 91 subjects in the PEP in 
FL enrolled in the study (except those who discontinue early) have been followed for at least 
6 months from the start of study drug.  
Primary efficacy analyses for FL will be based on the PEP in FL.  
The final analysis of DOR for FL will be triggered after most responders in the PEP have a 
minimum of 12 months of follow-up from first response.  


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The ITT MZL population is defined as all enrolled subjects with MZL who received at least 
one dose of ME-401. 
The final analysis for MZL will be triggered after most responders in the MZL ITT population 
have been followed for at least 12 months from first response. 
Efficacy analysis for MZL will be based on the ITT MZL population. 
For both FL and MZL, methods for disease/response assessment by the IRRC will be 
described in the IRRC Charter. 
Safety analyses will be performed on the Safety Population including all subjects who 
received at least one dose of ME-401.  


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TABLE OF CONTENTS 
SPONSOR’S PROTOCOL SIGNATURE PAGE ...........................................................................2 
INVESTIGATOR’S SIGNATURE PAGE......................................................................................3 
PROTOCOL SYNOPSIS ................................................................................................................4 
ABBREVIATIONS .......................................................................................................................18 
1. 
BACKGROUND ........................................................................................................22 
1.1. 
Follicular Lymphoma .................................................................................................22 
1.2. 
Marginal Zone Lymphoma .........................................................................................23 
1.3. 
Therapeutic Options for Third-Line Treatment ..........................................................24 
1.4. 
Phosphatidylinositol-4,5-bisphosphate 3-kinase δ (PI3Kδ) .......................................24 
1.5. 
ME-401 .......................................................................................................................24 
1.6. 
Experience with ME-401 ............................................................................................25 
1.6.1. 
Single Ascending Dose Healthy Volunteer Study ......................................................25 
1.6.2. 
Phase 1b Study in Relapsed FL and CLL/SLL (ME-401-002) ..................................26 
1.6.2.1. 
Dose-Escalation and Expansion (Monotherapy) ........................................................26 
1.6.2.2. 
ME-401 + Rituximab (Combination Therapy) ...........................................................27 
1.6.2.3. 
Continuous and Intermittent Dosing Schedules .........................................................27 
1.6.2.4. 
Efficacy .......................................................................................................................28 
2. 
RATIONALE FOR CURRENT STUDY ...................................................................28 
2.1. 
Patient Population .......................................................................................................28 
2.2. 
Rationale for a 60 mg Dose ........................................................................................29 
2.3. 
Rationale for ME-401 Intermittent Schedule Dosing Regimen .................................29 
2.4. 
Risk-Benefit of Study Participation ............................................................................33 
3. 
STUDY OBJECTIVES ..............................................................................................34 
3.1. 
Primary Objective .......................................................................................................34 
3.2. 
Secondary Objectives .................................................................................................34 
4. 
SUBJECT ELIGIBILITY ...........................................................................................35 
4.1. 
Inclusion Criteria ........................................................................................................35 
4.2. 
Exclusion Criteria .......................................................................................................36 
5. 
STUDY DESIGN .......................................................................................................37 
5.1. 
Overall Study Design ..................................................................................................37 


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5.2. 
Study Product: ME-401 ..............................................................................................40 
5.2.1. 
Preparation and Administration of ME-401 ...............................................................40 
5.2.2. 
Storage of ME-401 .....................................................................................................40 
5.2.3. 
ME-401 Accountability and Compliance ...................................................................41 
5.3. 
ANTICIPATED TOXICITIES ...................................................................................41 
5.3.1. 
Modified Dosing Schedules ........................................................................................41 
5.3.2. 
Adverse Events of Special Interest (AESIs) ...............................................................45 
5.3.3. 
Other Potential Toxicities ...........................................................................................46 
5.3.3.1. 
Phototoxicity ...............................................................................................................46 
5.4. 
Monitoring and Prophylaxis of Opportunistic Infection ............................................46 
5.4.1. 
Monitoring for Opportunistic Infection ......................................................................46 
5.4.2. 
Prophylaxis of OI Other Than PJP/PCP .....................................................................47 
5.4.3. 
Prophylaxis of PJP/PCP ..............................................................................................47 
5.5. 
Prohibited Treatments and Cautions for Concomitant Medications...........................48 
5.6. 
Advisory on COVID-19 Vaccination, Management, and Risk Assessment ..............48 
5.7. 
Discontinuation of ME-401 Treatment .......................................................................49 
5.8. 
Discontinuation of Subjects from Study .....................................................................49 
5.9. 
Site Discontinuation ....................................................................................................49 
5.10. 
Study Termination ......................................................................................................50 
6. 
SAFETY PARAMETERS, DEFINITIONS, AND REPORTING .............................50 
6.1. 
Adverse Event (AE) ....................................................................................................50 
6.2. 
Assessment of Severity ...............................................................................................51 
6.3. 
Action Taken with Study Drug in Response to the AE ..............................................52 
6.4. 
Outcome of AE ...........................................................................................................52 
6.5. 
Serious Adverse Event (SAE) Definition ...................................................................53 
6.6. 
Serious Adverse Event Reporting ...............................................................................53 
6.7. 
Relationship of Adverse Events to Study Drug ..........................................................54 
6.8. 
Pregnancies .................................................................................................................55 
6.9. 
Data Safety Monitoring Board ....................................................................................55 
7. 
RESPONSE ASSESSMENT AND STUDY ENDPOINTS.......................................55 
7.1. 
Overview .....................................................................................................................55 
7.2. 
Efficacy Endpoints ......................................................................................................56 
7.2.1. 
Objective Response Rate (ORR) ................................................................................57 


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7.2.2. 
Duration of Response (DOR) and Duration of Recaptured Response 
(DORR) .......................................................................................................................57 
7.2.3. 
Progression-Free Survival (PFS) ................................................................................57 
7.2.4. 
Time to Treatment Failure (TTF) ...............................................................................57 
7.2.5. 
Recapture of Response ...............................................................................................57 
7.2.6. 
Overall Survival (OS) .................................................................................................57 
7.3. 
Safety Endpoints .........................................................................................................58 
7.3.1. 
Overall Incidence Rate of Treatment-Emergent Adverse Events (TEAEs) ...............58 
7.3.2. 
Incidence Rate of AESIs .............................................................................................58 
7.3.3. 
Time to Occurrence of AESIs .....................................................................................58 
7.3.4. 
Other Safety Endpoints ...............................................................................................58 
7.4. 
Pharmacokinetics ........................................................................................................58 
8. 
STUDY PROCEDURES ............................................................................................59 
8.1. 
Informed Consent .......................................................................................................59 
8.2. 
Screening Period .........................................................................................................59 
8.3. 
Subject Enrollment .....................................................................................................59 
8.4. 
Cycle 1 Day 1 Tests and Administration of ME-401 .................................................59 
8.5. 
Treatment Phase ..........................................................................................................60 
8.6. 
End of Treatment (EOT) Visit (30-Day Safety Follow-up Visit) ...............................60 
8.7. 
Post-Treatment Long-Term Follow-up .......................................................................60 
9. 
STUDY ASSESSMENTS ..........................................................................................60 
9.1. 
Medical History ..........................................................................................................61 
9.2. 
Demographics .............................................................................................................61 
9.3. 
Concomitant Medication ............................................................................................61 
9.4. 
Physical Examination and Vital Signs ........................................................................61 
9.5. 
Adverse Events ...........................................................................................................61 
9.6. 
Cardiac Evaluation ......................................................................................................61 
9.7. 
Laboratory Tests .........................................................................................................62 
9.8. 
Disease/Response Assessment ....................................................................................63 
9.9. 
Compliance Diary .......................................................................................................63 
9.10. 
Dispensing of ME-401 ................................................................................................63 
9.11. 
Managing the Impact of COVID-19 on Study Assessments ......................................64 
10. 
CORRELATIVE IMMUNE STUDY (US SITES ONLY) ........................................65 


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11. 
STATISTICS ..............................................................................................................65 
11.1. 
Sample Size ................................................................................................................65 
11.2. 
Statistical Analysis ......................................................................................................66 
11.2.1. 
Analysis Populations and Analysis Sets Definitions ..................................................66 
11.2.2. 
Statistical Analyses .....................................................................................................67 
12. 
REGULATORY AND REPORTING REQUIREMENTS ........................................69 
12.1. 
Compliance with Laws and Regulations ....................................................................69 
12.2. 
Informed Consent .......................................................................................................69 
12.3. 
Institutional Review Board/Ethics Committee ...........................................................69 
12.4. 
Public Clinical Trial Registry .....................................................................................69 
12.5. 
Confidentiality and Data Protection ...........................................................................69 
12.6. 
Financial Disclosure ...................................................................................................70 
12.7. 
Data Quality Assurance ..............................................................................................70 
12.8. 
Insurance .....................................................................................................................70 
13. 
DATA MANAGEMENT ...........................................................................................70 
13.1. 
Use of Electronic Case Report Forms ........................................................................70 
13.2. 
Study Site Monitoring.................................................................................................70 
13.3. 
Record Retention ........................................................................................................71 
14. 
AMENDMENTS TO THE PROTOCOL ...................................................................71 
15. 
PUBLICATION POLICY ..........................................................................................72 
16. 
REFERENCES ...........................................................................................................73 
APPENDIX 1. SCHEDULE OF ASSESSMENTS .....................................................................78 
APPENDIX 2. COCKCROFT-GAULT EQUATION FOR ESTIMATED 
CREATININE CLEARANCE............................................................................82 
APPENDIX 3. SKIN ASSESSMENT .........................................................................................83 
APPENDIX 4. GUIDELINES FOR WOMEN OF CHILDBEARING POTENTIAL 
AND FERTILE MALE SUBJECTS ..................................................................85 
APPENDIX 5. LYMPHOMA RESPONSE CRITERIA: MODIFIED LUGANO 
CLASSIFICATION ............................................................................................86 
APPENDIX 6. ECOG PERFORMANCE STATUS CRITERIA................................................92 
APPENDIX 7. LIST OF CYP2C8 INHIBITORS AND INDUCERS ........................................93 
APPENDIX 8. LIST OF CYP3A INHIBITORS AND INDUCERS ..........................................94 
APPENDIX 9. LIST OF DRUGS KNOWN TO PROLONG QT/QTC INTERVAL ................95 


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LIST OF TABLES 
Table 1: 
Objective Response Rate in CS and IS Groups in FL – Study ME-401-002 .............30 
Table 2: 
Most Common (≥20%) Adverse Events in the CS and IS Groups – 
Study ME-401-002 (N = 96).......................................................................................31 
Table 3: 
Grade 3* Drug-Related Adverse Events of Special Interest (AESI) in the CS 
and IS Groups – Study ME-401-002 (N = 96) ...........................................................31 
Table 4: 
ME-401 Toxicity Management ...................................................................................42 
Table 5: 
NCI CTCAE v5.0 Adverse Event Grading System ....................................................52 
Table 6: 
Pharmacokinetics Sampling Schedule ........................................................................58 
Table 7: 
Revised Criteria for Response Assessment from Cheson et al. 2014 .........................88 
Table 8: 
Modified Lugano Response Criteria ...........................................................................91 
 
LIST OF FIGURES 
Figure 1: 
Study Schema ...............................................................................................................9 
Figure 2: 
BAT Response Curve for ME-401 in Normal Volunteers .........................................25 
Figure 3: 
Duration of Response in CS and IS Arms in FL – Study ME-401-002 .....................30 
Figure 4: 
Number of Subjects and Time to Grade 3 Drug-Related AESIs in CS and IS 
Groups – Study ME-401-002 (N = 96) .......................................................................32 
 


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ABBREVIATIONS 
Abbreviation 
Definition 
AE 
adverse event 
AESI 
adverse events of special interest 
ALP 
alkaline phosphatase 
ALT 
alanine aminotransferase 
ANC 
absolute neutrophil count 
aPTT 
activated partial thromboplastin time 
AST 
aspartate aminotransferase 
AUC 
area under the concentration versus time curve 
AUC(0–last) 
area under the concentration versus time curve from time zero to the last 
timepoint 
B 
bendamustine 
BAT 
basophil activation test 
BCR 
B-cell antigen receptor 
BCRP 
breast cancer resistance protein transporter 
BMB 
bone marrow biopsy 
BP 
blood pressure 
BTK 
Bruton tyrosine kinase 
BUN 
blood urea nitrogen 
CAR T 
chimeric antigen receptor T-cell 
CBC 
complete blood count  
CI 
confidence interval 
CLL 
chronic lymphocytic leukemia 
Cmax 
maximum plasma concentration 
Cmin 
minimum plasma concentration 
CMR 
complete metabolic response 
CMV 
cytomegalovirus 
CR 
complete response/remission 
CRO 
Contract Research Organization 
CS 
continuous schedule 
CT 
computed tomography 
CVP 
cyclophosphamide, vincristine, prednisone 
CYP3A4 
cytochrome P450 3A4 
CYP2C8 
cytochrome P450 2C8 


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Abbreviation 
Definition 
CyTOF 
mass cytometry 
DLBCL 
diffuse large B-cell lymphoma 
DLT 
dose-limiting toxicity  
DOR 
duration of response  
DORR 
duration of recaptured response 
DSMB 
Data Safety Monitoring Board 
EC 
Ethics Committee 
EC50 
half maximal effective concentration 
EC90 
90% maximal effective concentration 
ECG 
electrocardiogram 
ECOG 
Eastern Cooperative Oncology Group 
eCRF 
electronic case report form 
EE 
Efficacy Evaluable 
eGFR 
estimated glomerular filtration rate 
Emax 
maximum possible effect 
EU 
European Union 
FDA 
Food and Drug Administration 
FDG 
18F-fluorodeoxyglucose 
FL 
follicular lymphoma 
FLIPI 
Follicular Lymphoma International Prognostic Index 
GCP 
Good Clinical Practice 
GELF 
Groupe d’Etudes des Lymphomes Folliculaires 
GI 
gastrointestinal 
HcT 
hematocrit 
HGB 
hemoglobin 
HR 
heart rate 
IC50 
half-maximal inhibitory concentration  
ICF 
informed consent form 
ICH 
International Council for Harmonisation 
IND 
Investigational New Drug 
iNHL 
indolent non-Hodgkin's lymphoma 
INR 
international normalized ratio 
IP 
investigational product 
IQR 
interquartile range 


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Abbreviation 
Definition 
IRB 
Institutional Review Board 
IRRC 
Independent Response Review Committee 
IS 
intermittent schedule 
ITT 
Intent-to-Treat 
IV 
intravenous 
kg 
kilogram 
KM 
Kaplan-Meier 
L 
liter 
LDH 
lactate dehydrogenase 
mAb 
monoclonal Antibody 
MCH 
mean corpuscular hemoglobin 
mm3 
cubic millimeter 
mg 
milligram 
mL 
milliliter 
MTD 
maximum tolerated dose 
MUGA 
multigated acquisition scan 
MZL 
marginal zone lymphoma 
NCI CTCAE 
National Cancer Institute Common Terminology Criteria for Adverse Events 
NDA 
New Drug Application 
NE 
not evaluated 
NHL 
non-Hodgkin’s lymphoma 
NIP 
non-infectious pneumonitis  
NMR 
no metabolic response 
NOS 
not otherwise specified 
OI 
opportunistic infection 
ORR 
objective response rate 
OS 
overall survival 
PCR 
polymerase chain reaction 
PCP 
Pneumocystis pneumonia 
PD 
pharmacodynamic (in the context of the effect of a drug on biomarkers) 
PD 
progressive disease (in the context of tumor response) 
PEP 
primary efficacy population 
PET/CT 
positron emission tomography/computed tomography 
PFS 
progression-free survival 


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Abbreviation 
Definition 
P-gp 
P-glycoprotein 
PI 
prescribing information 
PI3K 
phosphatidylinositol 3-kinase 
PI3Kδ 
phosphatidylinositol 3-kinase delta 
PJP 
Pneumocystis jirovecii pneumonia 
PK 
pharmacokinetic(s) 
PMD 
progressive metabolic disease 
PMR 
partial metabolic response 
PR 
partial response 
PT 
prothrombin time 
QTc 
QT corrected (corrected QT-interval) 
QTcF 
QT-interval corrected according to Fridericia’s formula 
R 
rituximab 
R-B 
rituximab, bendamustine 
RBC 
red blood cell 
R-CVP 
rituximab, cyclophosphamide, vincristine, prednisone 
R-CHOP 
rituximab, cyclophosphamide, doxorubicin, vincristine, prednisone 
R-L 
rituximab-lenalidomide 
RP2D 
Recommended Phase 2 Dose 
SAE 
serious adverse event 
SAP 
Statistical Analysis Plan 
SGOT 
serum glutamic-oxaloacetic transaminase 
SGPT 
serum glutamic-pyruvate transaminase 
SLL 
small lymphocytic lymphoma 
SOC 
standard of care 
SUSAR 
suspected unexpected serious adverse reaction 
TEAE 
treatment-emergent adverse event 
TMP-SMX 
trimethoprim-sulfamethoxazole  
TREG 
T regulatory cell 
TTF 
time to treatment failure 
t½ 
half-life 
ULN 
upper limit of normal 
US(A) 
United States (of America) 
WBC 
white blood cell 
WHO 
World Health Organization 


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1. 
BACKGROUND 
Non-Hodgkin’s lymphoma (NHL) is one of the most common cancers in the world ranking as 
the 5th to 9th among cancer in most countries. In 2020, the reported incidence of new cases of 
NHL was approximately 545,000 globally and resulted in approximately 260,000 deaths 
(Sung 2021). It is estimated that in 2022 approximately 80,500 people will be diagnosed with 
NHL and approximately 20,250 will die from this cancer in the United States (US) (NCI 2022).  
NHL comprises more than 30 types; classification is based on the type of lymphocyte involved, 
B-cells or T-cells. NHL is further classified by other factors, including whether it is aggressive 
(fast-growing) or indolent (slow-growing) (Swerdlow 2016). 
1.1. 
Follicular Lymphoma 
Follicular lymphoma (FL) is typically a slow-growing or indolent form of B-cell NHL 
(i.e., iNHL). This lymphoma subtype accounts for 20–30% of all NHL cases (Provencio 2017). 
Of the approximately 80,500 cases of NHL diagnosed in 2022 in the US, approximately 20% 
were likely to be FL (NCI 2022). About 18,000 and 25,000 new cases of FL were expected in the 
US and the European Union (EU), respectively, in 2015 (Dreyling 2014, Howlader 2020). No 
sex preponderance is seen for FL, but the incidence increases with age, and varies across racial 
groups and geographic regions. The incidence of FL is low in China and Japan, but people of 
Ashkenazi Jewish ancestry have a higher incidence of lymphoma. In the US, the incidence 
is 2-3 times higher in White individuals than in those of African descent (Nabhan 2012).  
Transformation to diffuse large-cell lymphoma is associated with rapid progression of the 
disease including increasing adenopathy, development of systemic symptoms, and infiltration 
of extranodal sites. Recent estimates suggest that the ratio of histologic transformation may be 
2-3% per year (Tan 2013, Al-Tourah 2008).  
The overall survival (OS) rate of patients with FL is 77–86% at 5 years with a median survival of 
approximately 20 years for all patients, and is better for younger patients and those with better 
performance status, and significantly lower for patients with transformed disease 
(Provencio 2017, Mozas 2020). The majority of patients are diagnosed with advanced (i.e., Stage 
III and IV) disease. Both the Groupe d’Etudes des Lymphomes Folliculaires (GELF) and the 
Follicular Lymphoma International Prognostic Index (FLIPI) can provide prognostic information 
for specific patients (Solal-Céligny 1998, Swenson 2005). 
Several systemic therapeutic options are available for patients with FL, ranging from single agent 
anti-CD20 immunotherapy (most commonly with rituximab (Rituxan®; [R]) (McLaughlin 1998, 
Hainsworth 2005); to anti-CD20-based immunochemotherapy (most commonly the combination 
of R with cyclophosphamide, hydroxydoxorubicin, vincristine, and prednisone [CHOP] 
designated as R-CHOP [Czuczman 1999] or the alkylating agent bendamustine [B] designated as 
R-B [Rummel 2005, Robinson 2008]). Other chemotherapy regimens are also acceptable in 
combination with an anti-CD20 antibody. Immunochemotherapy is typically preferred in 
younger and fit patients, and achieves an objective response rate (ORR) >90%, with a substantial 
proportion of complete responses (CRs), and a progression-free survival (PFS) of 65.5% at 
5 years of follow-up (Flinn 2019).  
Rituximab-lenalidomide (R-L) combination has been evaluated in randomized studies in patients 
with relapsed/refractory iNHL (Leonard 2019) and for treatment of previously untreated patients 


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with FL (Morschhauser 2018). It has been reported that efficacy in patients treated with R-L as 
front-line therapy are comparable with results in patients treated with chemoimmunotherapy, 
where the overall response rate was 61% in the R-L arm and 65% in the R-chemotherapy arm, 
with PFS at three years of follow-up being 77% and 78% for R-L and R-chemotherapy, 
respectively (Leonard 2019).  
For subjects with relapsed disease, a similar immunochemotherapy approach, utilizing 
a chemotherapy regimen not previously administered, can be used. The combinations of 
R-CHOP or R-B showed satisfactory rates of objective response, ranging from 50–90%, 
and PFS, with median PFS ranging from 18–36 months (Czuczman 1999, Rummel 2005, 
Robinson 2008). Although the current treatments for iNHL are initially effective in inducing 
responses in most patients, they are not curative and show decreasing efficacy and development 
of resistance to therapy with repeated administrations.  
However, the disease will inevitably relapse; therefore, active agents with different mechanisms 
of action than cytotoxic chemotherapy are needed for this patient population, with the goal of 
extending the duration of disease control at relapse from initial therapy. Furthermore, since the 
median age of patients with FL and marginal zone lymphoma (MZL) at relapse is >60 years, new 
treatment options must be well tolerated and avoid the toxicities typically reported with 
chemotherapy.  
1.2. 
Marginal Zone Lymphoma 
Marginal zone lymphoma represents approximately 5–15% of all NHLs (Zucca 2020) and is the 
third most common type among iNHLs (Al-Hamadani 2015); however, it remains largely 
understudied. Frontline therapy for MZL differs greatly based upon the subtype (extranodal, 
nodal, mucosal) (Swerdlow 2016), and the underlying etiology; hence, defining a standard 
treatment regimen for MZL has been difficult (Noy 2017). Treatments include anti-CD20 
antibody (rituximab) based regimens either as monotherapy or in combination with systemic 
chemotherapy. Targeted therapy with ibrutinib (a Bruton tyrosine kinase [BTK] inhibitor) is also 
now used in relapsed/refractory cases across MZL subtypes (Denlinger 2018) for patients who 
have received at least one prior anti-CD20 based therapy (Imbruvica® US Prescribing 
Information); ORR with ibrutinib was 48% and median PFS was 14.2 months (95% confidence 
interval [CI]: 8.3 to not estimable). Outcomes were also analyzed by MZL subtypes, the median 
PFS was 13.8 months (95% CI: 8.3 to not estimable) for extranodal MZL, 19.4 months (95% 
CI: 8.2 to not estimable) for splenic MZL, and 8.3 months (95% CI: 2.8 to not estimable) for 
nodal MZL. The estimated 18-month OS rate was 81% (95% CI: 68 to 89; Noy 2017). 
No standard of care is available for patients who relapse after initial treatment and depending on 
disease characteristics and previously used therapies various regimens combining rituximab 
and chemotherapy-containing regimens have classically been the treatment of choice in the 
second line of treatment, resulting in ORR of 85–93% and CR rate of 54–78% (Brown 2009, 
Cervetti 2010, Orciuolo 2010, Laribi 2016, Zucca 2017). Treatment-related toxicities with these 
therapies such as Grade 3 and 4 myelosuppression frequently limit treatment participation in 
many patients (Noy 2017). Autologous and allogeneic hematopoietic progenitor cell 
transplantation has been performed in selected patients with relapsed/refractory MZL, achieving 
durable remissions and frequently cures, albeit at cost of significant morbidity (Shimoni 2017).  


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In the current landscape of MZL, standardized treatment algorithms still lack sufficient 
evidence-based guidelines required to guide optimal therapy. Combination therapies that include 
BTK inhibition as well as other agents are ongoing. PI3K inhibitors have shown strong activity 
in iNHL.  
1.3. 
Therapeutic Options for Third-Line Treatment 
For patients with advanced-stage FL and MZL requiring systemic lymphoma-directed 
treatment, there are currently no approved drugs for third-line therapy, justifying the use 
of an investigational agent such as ME-401. 
1.4. 
Phosphatidylinositol-4,5-bisphosphate 3-kinase δ (PI3Kδ) 
Signaling through the BCR provides a strong proliferative and survival stimulus to the cell; 
interfering with such signaling is therefore a rational approach to the treatment of B-cell 
malignancies. In leukocytes, BCR signaling is mediated partly by the activation of PI3Kδ. 
The activation of PI3Kδ ultimately leads to cell survival, proliferation, and immune regulation 
(Herman 2012).  
PI3Kδ is frequently active in B-cell malignancies and is central to multiple signaling pathways 
that drive proliferation, survival, homing, and retention of malignant B-cells in lymphoid tissue 
and bone marrow. Because PI3Kδ is the main PI3K isoform expressed in lymphoid cells, but is 
expressed at low or undetectable levels in most other tissues, inhibitors against PI3Kδ should be 
selective for the immune system and relatively non-toxic in other organs (Brown 2014). 
Four drugs with PI3Kδ inhibitory activity, Zydelig® (idelalisib), Aliqopa™ (copanlisib), 
Copiktra® (duvelisib), and Ukoniq™ (umbralisib) have been approved in the US for clinical use 
in patients with FL, and MZL (for Ukoniq), based on single arm studies. Approvals in FL and 
MZL were withdrawn in the US for Zydelig, Copiktra, and Ukoniq due to inability to initiate or 
complete the required confirmatory Phase 3 study.  
With the withdrawal in the US of all 3 oral PI3Kδ inhibitors, the treatment choices for relapsed 
FL and MZL patients is more limited. Therefore, there is a need for new treatment options for 
patients with relapsed indolent B-cell NHL, including efficacious and safer PI3Kδ inhibitors. 
1.5. 
ME-401 
Zandelisib (ME-401) is an orally bioavailable PI3Kδ inhibitor with optimal pharmacologic 
properties, including high potency with half-maximal inhibitory concentration (IC50) for PI3Kδ 
of 0.6 nM in a cellular assay, plasma half-life (t½) of approximately 28 hours supporting 
once-daily dosing, high volume of distribution indicating wide access to tissues, high specificity 
to the δ isoform at clinically relevant concentrations, and prolonged residence time on the target.  
ME-401 has demonstrated substantial single-agent anti-tumor activity in a Phase 1b 
dose-escalation study in patients with relapsed FL, small lymphocytic lymphoma (SLL), 
and chronic lymphocytic leukemia (CLL), with an ORR of 90% (Soumerai 2018) 
and manageable toxicity; 4 subjects with relapsed/refractory MZL treated on the intermittent 
schedule (IS) showed a 100% ORR (25% CR). These encouraging preliminary results justify 
the evaluation of ME-401 in a larger cohort of patients with FL and MZL. 


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1.6. 
Experience with ME-401 
1.6.1. 
Single Ascending Dose Healthy Volunteer Study 
A single ascending dose study of zandelisib across a range of dose levels from 10 to 150 mg was 
conducted in normal healthy volunteers. Both maximum plasma concentration (Cmax) and area 
under the concentration versus time curve from time zero to the last timepoint (AUC[0 last]) 
demonstrated linear increases over the dose range of 10 to 150 mg, with a time to maximum 
plasma concentration (tmax) of 5–6 hours.  
To assess the on-target pharmacodynamic (PD) effect of ME-401, inhibition of PI3K signaling in 
peripheral blood basophils was measured using a PD assay (basophil activation test [BAT]), 
which measured basophil activation by CD63 upregulation following ex-vivo stimulation with an 
anti-FCεεR1 monoclonal antibody. From the fitted maximum possible effect (Emax) model, the 
concentrations of ME-401 estimated to give 50% and 90% BAT inhibition (i.e., EC50 and EC90, 
respectively) of the maximum effect (Emax 91.6%) were 0.6 ng/ml and 5.2 ng/mL, respectively 
(Figure 2). Further pharmacokinetic (PK) modeling projected that a daily ME-401 dose of 60 mg 
will result in steady-state minimum plasma concentration (Cmin) in excess of 5 ng/mL, the EC90 
in the BAT assay. This led to the selection of 60 mg as the initial dose level in the Phase 1b dose 
escalation study in subjects with indolent B-cell malignancies, a dose lower than the starting 
dose for oncology trials calculated based on animal toxicokinetic studies. 
Figure 2: 
BAT Response Curve for ME-401 in Normal Volunteers 
 


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1.6.2. 
Phase 1b Study in Relapsed FL and CLL/SLL (ME-401-002) 
A Phase 1b study is currently ongoing evaluating zandelisib as a single agent and in combination 
with rituximab or zanubrutinib. As of 30 November 2019, a total of 103 subjects were enrolled: 
31 in the single-agent dose-escalation group, 41 in the rituximab combination safety group, 24 in 
a single-agent expansion cohort, and 7 in the zanubrutinib combination safety group. Enrollment 
in the single agent dose-escalation group was completed, and the Sponsor concluded that 60 mg 
is the Recommended Phase 2 Dose (RP2D) for future development. Enrollment is ongoing in an 
expansion cohort of ME-401 monotherapy at 60 mg in subjects with relapsed FL and CLL/SLL. 
Enrollment in the rituximab combination safety group is now completed. The Sponsor has 
concluded that ME-401 at 60 mg plus rituximab at 375 mg/m2 is well tolerated and is currently 
being evaluated in subjects with relapsed FL, CLL/SLL, MZL, diffuse large B-cell lymphoma 
not otherwise specified (DLBCL NOS) (germinal center B-cell type or activate B-cell type), 
and high-grade B--cell lymphoma (NOS or with MYC and BCL2 and/or BCL6 rearrangements). 
Enrollment in the arm assessing safety and efficacy of combination with BTK inhibitor 
zanubrutinib is ongoing.  
As of 09 June 2022, 164 subjects have been treated in the study, 38 dosed on continuous daily 
dosing (CS) and 126 dosed on the IS (see Section 1.6.2.3). Sixty subjects have received 
zandelisib monotherapy, 41 subjects have received zandelisib at 60 mg in combination with 
rituximab, and 63 subjects have received zandelisib 60 mg in combination with zanubrutinib. 
For a more detailed description of safety and efficacy findings in ME-401-002 study please refer 
to the most recent edition of the IB, currently Edition 9 as of the date of this amendment, and the 
published manuscript (Pagel 2022).  
1.6.2.1. 
Dose-Escalation and Expansion (Monotherapy) 
A dose-escalation with adaptive design using a modified continuous reassessment model was 
utilized in the single-agent dose-escalation part of the study. Six subjects per dose level (60, 120, 
and 180 mg) were enrolled to assess dose-limiting toxicity (DLT) with an option to enroll 
6 additional subjects at any dose level with a response rate ≥30% in the first 6 subjects. A total of 
31 subjects were enrolled at 3 dose levels and no DLT was observed in the 56-day DLT window 
period. Dose-escalation was stopped at 180 mg because there was a comparable and high ORR at 
the initial 3 dose levels with similar safety. The maximum tolerated dose (MTD) was not 
identified and the RP2D was declared as 60 mg because of the high response rate observed at 
this dose, manageable toxicities, and trough plasma concentrations exceeding the EC90 from the 
BAT assay in all subjects administered ME-401 at 60 mg. In addition, 21 subjects were enrolled 
in the expansion cohort evaluating ME-401 at 60 mg in relapsed FL and CLL/SLL. 
In subjects treated in the ME-401 dose-escalation cohorts (N = 31) and monotherapy expansion 
cohort (N = 21), there were no treatment-emergent adverse events (TEAEs) leading to death 
and 5 TEAEs leading to treatment withdrawal. Overall, 51 of 52 subjects (98.1%) had at least 
one TEAE; 25 (48.1%) experienced a Grade 3 TEAE, and 4 (7.7%) experienced a Grade 4 
TEAE. The most common TEAEs in ≥25% of subjects were diarrhea (28 [53.8%] subjects), 
cough (17 [32.7%] subjects), fatigue and nausea (16 [30.8%] subjects each), and rash 
maculo-papular (13 [25.0%] subjects). All other TEAEs in the ME-401 monotherapy arm 
occurred in <25% of subjects.  


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As of 09 June 2019, 22 of 52 subjects (42.3%) in the monotherapy cohorts have discontinued 
therapy, with a median follow up of 236.5 days (range: 28–869, interquartile range [IQR]: 
131.5–605.5): 5 due to an adverse event (AE), 9 due to disease progression, 4 due to the subject 
withdrawing consent, and 4 for stem cell transplant. 
1.6.2.2. 
ME-401 + Rituximab (Combination Therapy) 
Overall, 34 of 38 subjects (89.5%) in the ME-401 + rituximab combination arm had at least 
one TEAE; 8 (21.1%) experienced a Grade 3 TEAE; and 1 (2.6%) experienced a Grade 4 TEAE. 
The most common TEAEs were aspartate aminotransferase (AST) increased, diarrhea, fatigue 
(9 [23.7%] subjects each); alanine aminotransferase (ALT) increased and nausea (7 [18.4%] 
subjects each).  
In the combination arm (ME-401 + rituximab), median follow up was 89 days (range: 16–571, 
IQR: 56–268). Of the 38 subjects enrolled, 12 have discontinued therapy: 1 due to an AE, 9 due 
to disease progression, 1 due to the Investigator’s decision, and 1 due to the subject withdrawing 
consent.  
1.6.2.3. 
Continuous and Intermittent Dosing Schedules 
Initially, the Phase 1b study explored the CS of ME-401 dosing when the product was given 
daily continuously. To address the potential for delayed AEs, the protocol was amended to 
include a modified dosing schedule strategy with an intermittent schedule (IS) of ME-401 
administered once daily for 7 days followed by 21 days without therapy in each 28-day cycle. 
Intermittent therapy was introduced after an initial 2 cycles of daily dosing. The rationale for 
these treatment-free intervals was to potentially enable reconstitution of T regulatory cells 
(TREGs). The kinetics of TREGs repopulation following the administration of TREG-specific 
cytotoxic therapy has been studied by others and, in general, numeric recovery of the 
CD4+/CD25+/FoxP3+ cell population requires approximately 2 weeks (Mahnke 2007). This 
suggested that a 3-week treatment break might be reasonable, allowing approximately 1 week for 
ME-401 wash-out (approximately 5 half-lives) and 2 weeks for functional TREGs recovery.  
Overall, 46 of 47 subjects (97.9%) on CS dosing (including subjects with no switch to IS dosing 
or switched later than Cycle 3) had at least one TEAE; 22 (46.8%) experienced a Grade 3 TEAE 
and 3 (6.4%) experienced a Grade 4 TEAE. The most common TEAEs in ≥25% of subjects were 
diarrhea (19 [40.4%] subjects), cough (15 [31.9%] subjects), and fatigue (13 [27.7%] subjects). 
All other TEAEs on CS dosing occurred in <25% of subjects.  
On CS dosing, median follow up was 210 days (range: 28–869, IQR: 99–627). Of the 
47 subjects, 21 (44.7%) have discontinued therapy: 4 due to an AE, 9 due to disease progression, 
1 due to the Investigator’s decision, 4 due to the subject withdrawing consent, and 3 for stem cell 
transplantation. 
All subjects who had completed at least 2 cycles of treatment at the time Amendment 6 was 
implemented were planned to switch to IS dosing. Overall, 39 of 43 subjects (90.7%) in the 
IS dosing group had at least one TEAE; 11 (25.6%) experienced a Grade 3 TEAE and 2 (4.7%) 
experienced a Grade 4 TEAE. The most common TEAEs were diarrhea (18 [41.9%] subjects), 
fatigue and nausea (12 [27.9%] subjects, each), and AST increased (9 [20.9%] subjects).  


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On the IS, median follow up was 155 days (range: 16–552, IQR: 57–308). Of the 43 subjects, 
13 (30.2%) have discontinued therapy: 2 due to AE, 9 due to disease progression, 1 due to the 
subject withdrawing consent, and 1 for stem cell transplantation. 
For more details, please refer to most current Investigator’s Brochure. 
1.6.2.4. 
Efficacy 
The efficacy analysis is based on 28 April 2019 data-cut off and is focused on subjects with 
relapsed or refractory FL and CLL/SLL. The Efficacy analysis set included 64 subjects who had 
at least one response assessment after baseline and included 50 subjects with FL and 14 subjects 
with CLL/SLL. The ORR was 80% for subjects with FL and 100% for subjects with CLL/SLL. 
There was no significant difference in response between subjects who received ME-401 as 
a single agent or in combination with rituximab, and no significant difference between treatment 
schedules (CS vs IS). Among all the subjects achieving a partial response (PR) or CR, 
the responses occurred rapidly for the majority of subjects (48/54, 89%), with response being 
assessed after the first 2 cycles of therapy. Median duration of response (DOR) for the IS group 
is 11.2 months, with a median (IQR) -follow up time on study of 8.8 (4.9–12.0) months. The 
median DOR for the CS group has not been reached, with a median (IQR) follow-up time on 
study of 8.3 (4.6–17.5) months. Updated data on efficacy and safety of ME-401 from the 
ongoing Phase 1b study is presented in Section 2.3. 
2. 
RATIONALE FOR CURRENT STUDY 
While first line CD20-based immunotherapy, chemotherapy, or immunochemotherapy achieves 
responses in most patients with FL or MZL, relapses are inevitable and subsequent remissions 
after a relapse are of progressively shorter duration. In addition, patients develop primary 
and secondary resistance to treatment with anti-CD20 mAb (Davis 2000, Czuczman 2012). 
The choice of therapy at relapse requires consideration of many factors including age, 
comorbidities, duration of prior response, and future treatment possibilities; hence, it is highly 
individualized. This study will evaluate ME-401 as a treatment option in patients with FL 
or MZL after failure of at least two prior therapies. 
2.1. 
Patient Population 
Treatment with an anti-CD20 antibody is considered standard of care (SOC) as front-line therapy 
in FL and MZL as it was shown to improve disease outcome in several randomized trials. 
Chemotherapy for FL and MZL consists of an alkylating agent either given as a single agent 
(i.e., bendamustine) or in combination regimens (e.g., CHOP; cyclophosphamide, vincristine, 
prednisone [CVP]; or similar). Purine analogues such as fludarabine, typically administered 
in combination with other chemotherapy agents, are also accepted treatment options for FL 
and MZL. Anti-CD20-based radioimmunotherapy is also an accepted treatment option for FL 
and MZL. For eligible patients, high dose chemotherapy followed by allo- or autologous stem 
cell transplantation is considered an acceptable option as a salvage therapy. However, all patients 
will eventually relapse and duration of remission with every line of treatment will get shorter. 
Unlike DLBCL, FL and MZL are still generally considered incurable diseases.  


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The high frequency of PI3Kδ pathway alterations in NHL led to the development of PI3Kδ 
inhibitors in this disease. The approval and later withdrawal (see Section 1.4) of several of the 
PI3K inhibitors for relapsed or refractory FL such as idelalisib, umbralisib, and duvelisib in the 
US, has created a treatment gap and limited the treatment options targeted to this pathway for 
subjects with FL after failure of at least 2 prior lines of therapy. Despite ongoing efforts in recent 
years to provide new therapies for patients with relapsed FL and MZL, the high unmet clinical 
need remains, as patients need active and well-tolerated therapy to delay time to the next disease 
progression. As the majority of patients are >65 years old, finding effective and well tolerated 
therapeutic options for patients who failed initial treatment remains a high clinical unmet need in 
this patient population. 
2.2. 
Rationale for a 60 mg Dose 
• A dose of 60 mg has been identified for evaluation in this study on the basis of a high 
response rate, acceptable toxicity, and plasma concentrations at or above the EC90 of the 
BAT assay.  
• No MTD had been reached in the dose escalation part of the ongoing Phase 1b study. 
• The ORR that was observed in 31 evaluable subjects with FL (n = 22) and CLL/SLL 
(n = 9) was 90%, and 86% in subjects with FL was comparable at all 3 doses evaluated 
(i.e., 60, 120, 180 mg).  
• PK/PD analysis confirmed target saturation at 60 mg. The steady state mean Cmin 
concentrations at all 3 dose levels studied were at or above the BAT EC90. This supports 
the Sponsor’s hypothesis that maximal anti-tumor activity is dependent on complete 
inhibition of PI3Kδ signaling and that higher doses do not result in greater inhibition nor 
greater anti-tumor activity. Therefore, 60 mg daily was selected as a recommended dose 
for expansion cohorts in the ME-401-002 study and for this Phase 2 study.  
2.3. 
Rationale for ME-401 Intermittent Schedule Dosing Regimen  
Recently presented data about treatment patterns of patients with relapsed or refractory FL 
treated in community practice with the first-approved PI3Kδ inhibitor idelalisib indicates that 
many patients cannot tolerate long term therapy, with 46.9–91.7% discontinuing therapy due 
to toxicities, and median duration of therapy of 5.5 months (Andorsky 2019). Establishing 
an effective, but at the same time well-tolerated inhibitors of the BCR-signaling pathway is very 
important to find optimal treatment for patients with B-cell malignancies. 
Additional data from the ongoing Phase 1b study (ME-401-002) allowed the Sponsor to 
evaluate the efficacy and safety of two different treatment schedules – CS and IS. As of 
16 September 2019, 96 subjects received ME-401 as a single agent or in combination with 
rituximab. Subjects were treated with ME-401 either on CS or IS. Evaluation of preliminary 
efficacy in subjects with FL indicated there was comparable efficacy between treatment arms 
(CS or IS), with an ORR of 78% in all subjects, and slightly better ORR in the IS group versus 
the CS group (79% and 77%, respectively) (Table 1). With a median follow-up time of 
9.2 months in the IS group and 11.5 months in the CS group, the median DOR has not been 
reached yet for subjects in the IS group, indicating sustained efficacy of ME-401 with the 
intermittent dosing schedule (Figure 3). 


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Table 1: 
Objective Response Rate in CS and IS Groups in FL – Study ME-401-002 
Evaluable Subjects 
N 
Objective Response Rate 
n (%) 
All subjects 
55 
43 (78) 
IS group 
29 
23 (79) 
CS group 
26 
20 (77) 
Abbreviations: CS = continuous schedule; IS = intermittent schedule; N = number of subjects; n = number of 
subjects in the category. 
Figure 3: 
Duration of Response in CS and IS Arms in FL – Study ME-401-002 
 
Follow-up median (range ). 
IS (N = 23): 9.2 months (3.4–20.7); CS (N = 20): 11.5 months (3.0–30.4). 
Abbreviations: CS = continuous schedule; IS = intermittent schedule; N = number of subjects. 
Source: Zelenetz 2019. 
Review of safety data indicated that subjects in the IS group tolerated ME-401 better than those 
in the CS group, with differences observed in all categories of reported AEs (Table 2). There is 
a lower incidence of Grade 3 AEs in subjects in the IS group: diarrhea/colitis were reported in 
only 7% of subjects in IS groups (5% were related) compared to 25.6% (23% related) in the CS 
group; there was only one case of Grade 3 rash (0% related) observed in the IS group while 
10.3% (8% related) observed in the CS group; no Grade 3 increase in transaminases were 
observed in subjects treated in the IS group, and overall incidence of Grade 3 AESIs was very 
low (Table 2, Table 3). No Grade 4 toxicities have occurred in subjects treated with either the IS 
or CS regimen in this study to date. 


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Table 2: 
Most Common (≥20%) Adverse Events in the CS and IS Groups – 
Study ME-401-002 (N = 96) 
Preferred Term, n (%) 
All Grades 
Grade 3* 
CS 
(N = 39) 
IS 
(N = 57) 
CS 
(N = 39) 
IS 
(N = 57) 
Diarrhea/colitis 
19 (48.7) 
21 (36.8) 
10 (25.6) 
4 (7.0) 
Rash (all types) 
15 (38.5) 
8 (14.0) 
4 (10.3) 
1 (1.8) 
Cough 
14 (35.9) 
9 (15.8) 
0 
0 
Fatigue 
12 (30.8) 
14 (24.6) 
1 (2.6) 
0 
Nausea 
11 (28.2) 
14 (24.6) 
1 (2.6) 
0 
Decreased appetite 
10 (25.6) 
3 (5.3) 
0 
0 
Pyrexia 
9 (23.1) 
5 (8.8) 
1 (2.6) 
0 
Nasal congestion 
9 (23.1) 
5 (8.8) 
0 
0 
Aspartate aminotransferase increased 
8 (20.5) 
11 (19.3) 
2 (5.1) 
0 
Abdominal pain 
8 (20.5) 
8 (14.0) 
0 
0 
Gastroesophageal reflux 
8 (20.5) 
4 (7.0) 
0 
0 
Abbreviations: Aes = adverse events; CS = continuous schedule; IS = intermittent schedule; N = number of subjects; 
n = number of subjects in the category. 
* There were no Grade 4 or 5 AEs reported.
Table 3: 
Grade 3* Drug-Related Adverse Events of Special Interest (AESI) in the CS 
and IS Groups – Study ME-401-002 (N = 96) 
AESI, n (%) 
CS Group 
(N = 39) 
IS Group 
(N = 57) 
Diarrhea/colitis 
9 (23) 
3 (5) 
Rash, all types 
3 (8) 
0 
AST/ALT increased 
3 (8) 
1 (2) 
Mucositis 
1 (3) 
0 
Pneumonia/pneumonitis 
4 (10) 
1 (2) 
Abbreviations: AESI = adverse events of special interest; ALT = alanine aminotransferase; AST = aspartate 
aminotransferase; CS = continuous schedule; IS = intermittent schedule; N = number of subjects; n = number of 
subjects in the category. 
* There were no Grade 4 or 5 AEs reported.
It is hypothesized that delayed toxicities, such as diarrhea/colitis, rash, increases in 
transaminases, and pulmonary toxicities associated with PI3Kδ inhibitors therapy might be due 
to an effect on regulatory T cells. To avoid such on-target side effects that are associated with 
continuous dosing schedules with other products in this class, the alternative option is to mitigate 
those toxicities by utilizing an intermittent dosing schedule. Preliminary data with ME-401 
indicates that overall incidence of such events, especially clinically significant Grade 3 events, 
is lower in subjects treated with the IS. Figure 4 represents time to adverse events of special 
interest (AESIs) in subjects treated in the CS and IS groups, with only 5 (9%) subjects who 


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experienced a related Grade 3 AESI compared to those in the CS group (16 [41%] subjects). This 
data indicates that the IS may significantly reduce a potential risk of developing immune-
mediated adverse reactions. 
Figure 4: 
Number of Subjects and Time to Grade 3 Drug-Related AESIs in CS and IS 
Groups – Study ME-401-002 (N = 96) 
 
Dosing Group 
Subjects with Grade 3 AESI, n (%) 
CS (N = 39) 
16 (41) 
IS (N = 57) 
5 (9) 
Abbreviations: AESI = adverse event of special interest; CS = continuous schedule; IS = intermittent schedule; N = 
number of subjects; n = number of subjects in the category. 
Source: Zelenetz 2019. 
Observations in the ongoing clinical Phase 1b study indicated that treatment with the IS dosing 
regimen is as active as daily dosing, and at the same time associated with improved tolerability. 
These findings warranted a protocol amendment for the Phase 2 study (i.e., Amendment 2) to 
discontinue enrollment of subjects in the CS arm, and continuation of the study with the IS arm 
only. While subjects in the CS arm in the Phase 1b study did not experience significant or 
life-threatening toxicities, overall assessment of the risk-benefit profile indicates that IS dosing 
is favorable and may potentially provide subjects with potentially highly effective and 
well-tolerated therapy over the long term. Once Amendment 2 of the study protocol had been 
approved by local regulatory authorities and Institutional Review Boards (IRBs)/Ethics 
Committees (ECs), subjects who were randomized to the CS arm under Amendment 1 continued 
therapy with a switch to the IS. If subjects already completed 2 initial cycles of therapy, they 
were switched to IS dosing once they completed therapy in the ongoing cycle of treatment, 
and if they had not yet completed the first 2 cycles of therapy they switched to IS dosing after 
completion of the first two cycles of therapy. Subjects who experienced disease progression 


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during treatment on the IS could continue therapy with ME-401 on the CS after discussion of 
available options with the Investigator. Continuation of treatment was allowed after consultation 
with the study Medical Monitor or designee after radiographic confirmation of disease 
progression. 
While it was established that PI3K inhibitors provide strong efficacy in patients with relapsed 
or refractory iNHL, duration of therapies might be limited by intolerable side effects leading to 
treatment discontinuation. Finding effective and well-tolerated therapies remains a high clinical 
unmet need for patients with relapsed or refractory FL or MZL. An intermittent dosing schedule 
of ME-401 may provide substantial benefit for this population of patients. 
2.4. 
Risk-Benefit of Study Participation 
As of the date of zandelisib IB Edition 9, more than 422 subjects with B-cell malignancies have 
been dosed with ME-401 from the ongoing Phase 1b, and Phase 2 (ME-401-003) and Phase 3 
(ME-401-004) studies. Treatment with ME-401 as single agent or in combination with rituximab, 
given on IS dosing, has been associated with high ORR and is well tolerated with few 
discontinuations due to toxicity. For details of the risk-benefit of zandelisib refer to the current 
edition of the IB, Edition 9 as of the date of this amendment. 
One Phase 1 study (PWT-001) has been completed in healthy subjects to evaluate the 
pharmacokinetics (PK) and pharmacodynamics (PD) of ME-401. In addition to this Phase 2 
study, there is an ongoing Phase 1b study (ME-401-002) evaluating the safety, efficacy, and PK 
of ME-401 in subjects with relapsed B-cell malignancies an ongoing randomized Phase 3 study 
(ME-401-004) evaluating zandelisib + rituximab versus standard immunochemotherapy in 
subjects with relapsed or refractory FL or MZL who received ≥1 line of prior systemic therapy, 
and an ongoing Phase 1 study (ME-401-K01) sponsored by Kyowa Kirin Co., Ltd, evaluating the 
safety and tolerability of ME-401 monotherapy in Japanese subjects with relapsed or refractory 
B-cell malignancies. With more than 422 subjects treated with zandelisib (monotherapy or in 
combination with another drug) the available safety/efficacy data indicate a favorable risk-
benefit profile.. Additional information is available in the Investigator’s Brochure. 
Diarrhea/colitis, rash, transaminitis, and stomatitis are AEs of interest that may be attributed to 
immune-related dysfunction due to TREGs suppression, which is indirectly affected by PI3Kδ 
inhibition. Other possible events such as pneumonitis, pneumonia, and opportunistic infections 
(OIs) have been observed in similar-class drugs and have been observed in the Phase 1b study 
evaluating ME-401. All subjects should be closely monitored for signs of acute toxicities, such 
as anaphylactic and other hypersensitivity reactions after receiving ME-401. 
To be eligible for the study, potential subjects must have experienced disease progression after 
receiving at least 2 prior lines of standard systemic anti-lymphoma therapy. This is a patient 
population with a poor prognosis and participation in a clinical trial is generally considered 
a reasonable option for such patients according to clinical practice guidelines 
(NCCN Guidelines V4.2019). Preliminary data suggests strong efficacy and manageable 
toxicities in subjects with B-cell malignancies treated with ME-401 as a single agent or in 
combination with rituximab. As of Protocol Amendment 2, the study is enrolling subjects into an 
open-label, single-arm study to evaluate the efficacy and safety of IS dosing for ME-401. As 
indicated above (Section 2.3), preliminary results from the ongoing clinical Phase 1b study 
indicate that the IS dosing regimen provides high ORR in patients with multiple relapsed disease 


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and is well-tolerated. An independent Data Safety Monitoring Board (DSMB) will be evaluating 
safety and efficacy in this clinical study on an ongoing basis to assure that subjects are not 
exposed to an undue risk (Section 6.9). There are no standards of care available for patients with 
relapsed or refractory FL or MZL who have received ≥2 lines of prior therapy. Therefore, the 
potential benefit for subjects in the ME-401-003 clinical study may outweigh the potential risks. 
The Investigator should discuss the risks and benefits of participating in the study with potential 
subjects to ensure that they are aware of any alternative treatment options.  
3. 
STUDY OBJECTIVES 
This is a single-arm study. The objectives of this study are to evaluate the efficacy and safety of 
the IS dosing regimen of ME-401. 
3.1. 
Primary Objective 
• To evaluate the objective response rate (ORR) of ME-401 in relapsed or refractory FL 
or MZL, based on the Modified Lugano Response Criteria (Appendix 5), and determined 
by an Independent Response Review Committee (IRRC) 
3.2. 
Secondary Objectives 
• To evaluate the efficacy of ME-401 as assessed by an IRRC: 
o Duration of response (DOR)  
o Complete response (CR) rate  
o Progression-free survival (PFS) 
• To evaluate the efficacy of ME-401 as assessed by the Investigator: 
o Objective response rate (ORR) 
o Duration of response (DOR) 
o Complete response (CR) rate  
o Progression-free survival (PFS) 
o Time to treatment failure (TTF)  
o Recapture of response 
o Duration of recaptured response (DORR) 
• To evaluate overall survival (OS) 
• To evaluate the safety profile of ME-401 
o Overall incidence of TEAEs 
o Incidence of AESIs  
o Time to occurrence of AESIs  
• To evaluate the pharmacokinetics (PK) of ME-401 


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4. 
SUBJECT ELIGIBILITY 
This study can fulfill its objectives only if appropriate subjects are enrolled. The following 
eligibility criteria are designed to select subjects for whom participation in the study is 
considered appropriate. All relevant medical conditions and alternative treatment options should 
be taken into consideration when deciding whether a particular subject is suitable for this 
protocol. 
4.1. 
Inclusion Criteria 
1. Signed informed consent. 
2. Age ≥18 years (or age of majority). 
3.  Histologically confirmed diagnosis as defined in the World Health Organization (WHO) 
classification (Swerdlow 2016) of:  
a. FL limited to Grade 1, 2, or 3a; or  
b. MZL, including nodal, extranodal, and splenic MZL (histopathological report 
confirming diagnosis must be available during screening procedures). 
4. Subjects with relapsed or refractory FL or MZL who received ≥2 prior therapy regimens. 
A previous regimen is defined as one of the following: at least two months of single-
agent therapy or at least two consecutive cycles of polychemotherapy, autologous 
transplant, or radioimmunotherapy. Prior therapy must include an anti-CD20 monoclonal 
antibody (mAb) and an alkylating agent(s). Relapsed or refractory disease is defined as:  
a. Relapsed disease: disease progression after a response (CR or PR) lasting ≥6 months  
b. Refractory disease: no response to therapy (no CR or PR), or response lasting 
<6 months  
5. At least one bi-dimensionally measurable nodal lesion >1.5 cm or extranodal lesions 
>1 cm in its longest diameter by computed tomography (CT) scan as defined by the 
Modified Lugano Classification (Appendix 5).  
a. Previously irradiated lesions can be selected as target lesions only in cases of 
unequivocal evidence of progression.  
b. For subjects with splenic MZL only: diffuse spleen involvement with splenomegaly, 
which is defined as the splenic vertical length greater than 13 cm. 
6. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1 (Oken 1982; 
Appendix 6). 
7. Adequate hematologic parameters at screening unless abnormal values are due to 
lymphoma per Investigator assessment: 
a. Absolute neutrophil count (ANC) ≥1.0 × 109/L (≥1,000/mm3) 
b. Platelet count ≥75.0 × 109/L (≥75,000/mm3) 
8. Adequate renal and hepatic function per local laboratory reference range at screening as 
follows: 


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a. Aspartate aminotransferase (AST)/serum glutamic-oxaloacetic transaminase (SGOT), 
alanine aminotransferase (ALT)/serum glutamic-pyruvate transaminase (SGPT) 
≤3.0 × upper limit of normal (ULN) 
b. Total bilirubin ≤2.0 × ULN or ≤3 × ULN for subjects with Gilbert’s syndrome  
c. Serum creatinine ≤1.5 × ULN or estimated glomerular filtration rate (eGFR) 
>50 mL/min using the Cockcroft-Gault equation (Appendix 2) 
9. QT-interval corrected according to Fridericia’s formula (QTcF) ≤450 milliseconds 
(msec); subjects with QTc >450 msec but <480 msec may be enrolled provided the QTc 
prolongation is due to a right bundle branch block (RBBB), left bundle branch block 
(LBBB), or pacemaker and is confirmed stable by a cardiologist. 
10. Left ventricular ejection fraction (LVEF) ≥45% as measured by echocardiogram 
or multigated acquisition scan (MUGA). If LVEF <45% by ECHO, a repeat measurement 
can be conducted within the screening period.  
11. Subjects must have completed any prior systemic anti-cancer treatment within ≥4 weeks 
of Cycle 1 Day 1 (or ≥5 times the half-life [t½], whichever is longer); ≥8 weeks for 
antibody agents; ≥2 weeks for radiation therapy; and ≥3 months for high dose therapy 
with stem cell transplantation or CAR T-cell therapy or radioimmunotherapy.  
12. All AEs and laboratory toxicities related to prior therapy must resolve to Grade ≤1 prior 
to the start of the study therapy (unless otherwise specified in eligibility criteria). 
13. For females of childbearing potential, a negative serum human chorionic gonadotropin 
(hCG) pregnancy test within 28 days of study Day 1 and negative hCG result on study 
Day 1. 
14. Subjects must agree to use appropriate contraception methods during the clinical study 
(Appendix 4). 
15. Subject is willing and able to comply with all scheduled visits, treatment plans, 
laboratory tests, and other study procedures. 
4.2. 
Exclusion Criteria 
1. Histologically confirmed FL Grade 3b, or transformed disease (assessed by the 
Investigator): 
a. For patients with clinical (e.g., marked B-symptoms), laboratory (e.g., high lactate 
dehydrogenase [LDH]), or radiographic (e.g., high standardized uptake value by 
positron emission tomography [PET]) signs of rapid disease progression, a fresh 
tumor biopsy prior to enrollment is required to rule out transformed disease. 
2. Known lymphomatous involvement of the central nervous system. 
3. Major surgical procedure within 4 weeks prior to study Day 1 (minor surgical procedures, 
[e.g., lymph node biopsy] performed within 1 day or with an overnight stay are allowed). 
4. Prior therapy with PI3K inhibitors. 


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5. Any uncontrolled clinically significant illness including, but not limited to, active 
infections requiring systemic antimicrobial therapy, hypertension, angina, arrhythmias, 
pulmonary disease, or autoimmune dysfunction. 
6. Subjects who have tested positive for hepatitis B surface antigen and/or hepatitis B core 
antibody plus have a positive hepatitis B polymerase chain reaction (PCR) assay; subjects 
who have previously tested positive with a negative PCR assay are permitted with 
appropriate anti-viral prophylaxis. 
7. Positive hepatitis C virus antibody (HCV Ab); subjects with positive HCV Ab are 
eligible if they are negative for HCV by PCR. 
8. Known history of, or active human immunodeficiency virus (HIV) infection. 
9. Ongoing or history of drug-induced pneumonitis. 
10. Previous or concurrent cancer that is distinct in the primary site or histology from 
indolent B-cell NHL within 3 years before start of study treatment except for curatively 
treated cervical cancer in situ, non-melanoma skin cancer, superficial bladder tumors 
(Ta [non-invasive tumor], Tis [carcinoma in situ], and T1 [tumor invades lamina 
propria]), and asymptomatic localized prostate cancer with no requirement for systemic 
therapy (or requiring only hormonal therapy) and with normal prostate-specific antigen 
values within ≥12 months prior to enrollment. 
11. History of clinically significant cardiovascular abnormalities such as congestive heart 
failure (New York Heart Association classification ≥II [NYHA 1994]), myocardial 
infarction within 6 months of study entry. 
12. History of clinically significant gastrointestinal (GI) conditions, particularly: 
a. Known GI condition that would interfere with swallowing or the oral absorption 
or tolerance of study drug 
b. Pre-existing malabsorption syndrome or other clinical situation that would affect oral 
absorption 
13. Females who are pregnant; females who plan to breastfeed during study treatment 
through 90 days after ending treatment. 
14. Psychiatric illness/social situations that would interfere with study compliance. 
15. Hypersensitivity or other clinically significant reaction to the study drug or its inactive 
ingredients. 
16. Any other condition for which, in the opinion of the Investigator, participation would not 
be in the best interest of the subject. 
5. 
STUDY DESIGN 
5.1. 
Overall Study Design 
This is a global, multicenter, open-label, single-arm, Phase 2 study of the PI3Kδ inhibitor 
ME-401 in subjects with relapsed/refractory FL or MZL. ME-401 will be administered orally 


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at a dose of 60 mg given once a day. A cycle of treatment is 28 days in duration. Treatment with 
ME-401 is administered based on an intermittent schedule (IS).  
The IS includes: 
• continuous daily therapy for the initial 2 cycles of treatment 
• the intermittent treatment begins at Cycle 3, with ME-401 administered daily for the 
first 7 days of every 28-day cycle (7 days on treatment and 21 days off treatment)  
The first day of treatment is designated as Day 1. ME-401 will be administered until progressive 
disease (PD), development of unacceptable toxicity (despite ME-401 modified dosing schedule), 
or subject withdrawal.  
Review of the ongoing Phase 1b study evaluating different dosing schedules for ME-401 
in subjects with relapsed/refractory FL showed improved risk-benefit profile of the IS over 
a continuous schedule (CS, i.e., ME-401 given daily continuously). With Amendment 2, 
the initial CS treatment arm was terminated, and subjects were enrolled to receive therapy on the 
IS only. If subjects already completed 2 initial cycles of therapy, they were switched to IS dosing 
once they completed therapy in the ongoing cycle of treatment, and if they had not yet completed 
the first 2 cycles of therapy they were switched to IS dosing after completion of the first two 
cycles of therapy. Subjects who progress on IS dosing may be switched to CS dosing. 
Upon confirmation of meeting eligibility criteria, subjects are enrolled and should be treated with 
ME-401 within a reasonable time frame (within 5 days). It is important to ensure that subjects 
continue meeting protocol specified eligibility criteria to enroll in the study, and if there are 
>7 days between screening laboratory assessments and expected Cycle 1 Day 1 dosing, then the 
laboratory assessments should be repeated and reviewed prior to the first dose of ME-401.  
The study schema is presented in Figure 1. 
For subjects who experience hematologic toxicity; diarrhea/colitis, cutaneous reactions 
(including rash and mucositis), AST/ALT elevation, or non-infectious pneumonitis (NIP); 
or other toxicities, refer to ME-401 toxicity management (Table 4) and modified dosing 
schedules in Section 5.3.1. Study treatment interruption is acceptable due to observed adverse 
reactions, and is allowed for up to 6 continuous weeks from drug interruption. Once a subject has 
recovered from a toxicity, treatment may be resumed beginning at Day 1 of the subsequent cycle 
of therapy according to the toxicity management indicated in Table 4. 
Study drug administration, study visits, and protocol-mandated assessments are conducted in 
28-day cycles. Subject participation in the study is expected to be approximately 3-4 years, 
including 28 days for screening, 18 months of dosing with study drug, and up to 3 years of 
follow-up for survival after discontinuation of study drug. This is an estimated average duration 
of subject participation; actual duration may be shorter or longer based on disease 
response, tolerability to therapy, and duration of the long-term follow-up period. 
Disease/response assessments utilizing diagnosis quality scans (CT scan with contrast, 
diagnostic quality PET/CT scan) will be initially performed at screening, after 4 months 
and 12 months on therapy, and at any time to confirm CR. Once a CR has been documented, 
18F-fluorodeoxyglucose (FDG)-PET scan is no longer required to monitor disease response 
and a CT scan is an appropriate imaging test. CT scans will be performed at all other protocol 


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prespecified timepoints for disease/response assessment. The disease/response assessment will 
be performed every 2 months (±7 days) for the first 6 months on study, every 3 months (±7 days) 
during the following 12 months, and every 6 months (±14 days) thereafter. 
Disease/response assessment will be based on radiological tumor evaluation of neck, chest, 
abdomen, and pelvis by using IV (and oral, if indicated, per Imaging Manual) contrast enhanced 
CT/magnetic resonance imaging (MRI) and/or PET-CT imaging modalities at screening and at 
follow-up visits at the frequency described in the Schedule of Assessments (Appendix 1), 
and disease response will be based on the Modified Lugano Classification response criteria 
(Appendix 5). Please see Table 7 for overall guidance on response assessment criteria based on 
Lugano Classification response criteria. In this clinical study, additional modifications to the 
Lugano Classification response criteria are incorporated per regulatory request to clarify rules 
for partial response (PR) assessment. These modifications highlight that morphologic (CT scan 
based) response will be the basis for PR assessment and not metabolic (FDG-PET scan) based 
assessment of response. Please see Table 8 for details regarding combined response assessment 
criteria based on beforementioned modified Lugano Classification. For subjects followed by 
CT scans only, a bone marrow biopsy will be required to confirm a CR in a subject who achieve 
a morphologic CR by CT scan. To rule out a potential of false positive diagnosis of disease 
progression in ambiguous cases, it is highly recommended to repeat imaging assessments within 
4-8 weeks after initial suspicion of PD. The Investigator’s assessment of disease response will be 
used for subject management. For the final analysis of study efficacy endpoints, disease/response 
assessment will be performed by an IRRC blinded to Investigator assessment. For subjects with 
gastric MZL, endoscopy with biopsy must be performed to confirm CR.  
Safety will be assessed by laboratory safety tests including hematology (complete blood count 
[CBC]), serum chemistry, and cytomegalovirus (CMV) using quantitative PCR; and clinical 
assessments including physical examination, vital signs, ECOG performance status, and 12-lead 
electrocardiogram (ECG). Adverse events will be graded according to National Cancer Institute 
Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0. 
Subjects will continue to receive study drug in accordance with the IS dosing regimen (or 
CS dosing regimen if switched from IS after PD) until Investigator-assessed disease progression 
or unmanageable toxicity requiring treatment discontinuation, subject’s decision to withdraw 
their consent to participate on study, or study closure by the Sponsor. A notification to the 
Medical Monitor will be sent prior to subject discontinuation. The End of Treatment 
(EOT)/30-Day Safety Follow-up visit will occur 30 (±3) days from the last dose of study drug.  
Subjects who discontinue study drug will remain in the study with follow-up conducted every 
3 months from the last dose of study drug to obtain post-treatment long-term follow-up 
information on disease status (i.e., change in disease response, progression), start of subsequent 
therapy, and survival (death). During this post-treatment follow-up phase, all subjects 
discontinued for reasons other than PD must continue imaging tests to assess disease status, 
performed per the original protocol-defined schedule for disease/response assessment, or earlier 
if clinically indicated. Imaging test results will no longer be collected when a subject starts a new 
therapy or disease progression occurs. The progression will be documented in the post-treatment 
long-term follow-up phase. Survival follow-up will continue for up to 3 years after the last 
subject is enrolled in the study.  


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Plasma samples will be obtained from all subjects to evaluate ME-401 PK and to serve for 
ME-401 exposure-response and exposure-toxicity modeling. For subjects enrolled at US sites 
only, blood samples will be obtained for a correlative immune study. See Schedule of 
Assessments (Appendix 1) for blood sample collection. 
The primary analysis of ORR for FL will occur after the initial 91 consecutive subjects 
randomized to or assigned to the IS arm, which represent the Primary Efficacy Population (PEP) 
(except those who discontinue early), have been followed for at least 6 months from the start of 
study drug. The final analysis of DOR for FL will occur after most responders in the PEP have at 
least 12 months of follow-up from first response.  
Enrollment in the MZL arm was closed by the Sponsor on 31 August 2022, with 32 of the 
64 planned subjects enrolled. The reason for early closure of enrollment was to focus the 
enrollment of subjects with MZL in the global Phase 3 study ME-401-004. Early enrollment 
closure was communicated to all investigators in August 2022 and is being formalized in 
Amendment 5.  
The final analysis of ORR and DOR for MZL will be triggered after most responders in the ITT 
MZL population (except those who discontinue early) have been followed for at least 12 months 
from first response. This is projected to occur approximately 14 months after first dose of the last 
subject with MZL enrolled in the study.  
5.2. 
Study Product: ME-401 
5.2.1. 
Preparation and Administration of ME-401 
Zandelisib (code ME-401) is provided as 60 mg capsules, which require no preparation prior to 
administration. Subjects should be instructed to swallow the capsules whole and to not chew 
or crush them.  
ME-401 is dosed on a milligram (mg) basis with no adjustment in dosing based on subject 
weight. ME-401 is to be taken orally once a day at approximately the same time each day in 
accordance with the IS dosing regimen (or CS dosing regimen if switched from IS after PD).  
ME-401 should be taken with a glass of water on an empty stomach (i.e., at least 1 hour prior to 
food intake or 2 hours after food intake). If a subject routinely experiences discomfort with 
dosing, he or she is permitted to take ME-401 with a light, non-fatty snack. If a dose is missed it 
may be taken up to 12 hours after the specified time. After 12 hours the missed dose should be 
omitted. If possible, the subject should be advised to separate administration of ME-401 capsules 
from that of concomitant medications that have a known effect on P-glycoprotein (P-gp) and 
breast cancer resistance protein transporter (BCRP) (see Section 5.5 for prohibited concomitant 
medications). 
5.2.2. 
Storage of ME-401 
ME-401 should be protected from direct sunlight and stored in a cool place (at the subject’s 
home) or at room temperature, 15–25°C (59–77°F). 


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5.2.3. 
ME-401 Accountability and Compliance 
The Principal Investigator or their representative will account for all ME-401 provided by the 
Sponsor. All ME-401 will be stored at the site in a secure and locked location. The Principal 
Investigator shall maintain adequate records of the disposition of ME-401, including dates, 
quantity, and use by subjects. Upon completion of the study, all remaining ME-401 will be 
accounted for and unused material will either be returned to the Sponsor (or designee) by 
a traceable method (UPS, FedEx, etc.) or destroyed (with documentation of drug destruction 
provided to the Sponsor), as instructed by the Sponsor. 
5.3. 
ANTICIPATED TOXICITIES 
As of the 09 June 2019 data cut-off date, the most common Grade ≥3 AEs noted in the Phase 1b 
study for the 52 subjects administered ME-401 alone consisted of diarrhea/colitis in 14 (26.9%) 
subjects; neutrophil count decreased in 4 (7.7%) subjects; and ALT increased, AST increased, 
and rash in 2 (3.8%) subjects each. There were no DLTs reported across all dose levels in the 
56-day DLT window period. 
Refer to Section 5.3.1 for study drug dose interruption or treatment discontinuation in a subject 
if such toxicity occurs and is not considered related to the underlying disease. Subject 
management will be determined by the treating physician and may include administration of 
a hematopoietic growth factor for neutropenia, administration of corticosteroids for the treatment 
of colitis, pneumonitis, and other AEs considered immune mediated (Table 4), and antimicrobial 
or anti-viral treatment for the prevention and/or management of OIs (Section 5.4). 
Refer to the current Investigator’s Brochure for more details. 
5.3.1. 
Modified Dosing Schedules 
Modification of the dosing schedule and dosing interruptions are utilized for toxicity 
management in this study. For subjects who experience toxicity during the first 2 cycles while 
receiving daily therapy with ME-401, dose interruption followed by a switch to intermittent 
schedule daily dosing for the first 7 days of every 28-day cycle is allowed. Subjects who 
experience toxicities on the IS will be managed by the instructions for management of potential 
drug-related toxicity in Table 4, which may include dose interruption or permanent 
discontinuation of ME-401. All treatment interruptions (including any missed doses, treatment 
delays) and discontinuations and their reasons are to be recorded in the electronic case report 
form (eCRF). Interrupted treatment is resumed on the IS after subjects recover to the grade 
specified in the table (subjects who permanently discontinue ME-401 dosing will proceed to the 
EOT/30-Day Safety Follow-up visit). Subjects on the IS who recover within the same treatment 
cycle, will complete the planned days off, and resume treatment on Day 1 of the next planned 
cycle.  
Subjects who interrupt study drug dosing for >6 continuous weeks will have ME-401 
permanently discontinued unless restarting ME-401 is authorized by the study Medical Monitor. 
Consultation with the Sponsor’s Medical Monitor or designee is recommended before treatment 
interruption or discontinuation due to any reason. 


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Table 4: 
ME-401 Toxicity Management 
Toxicity 
Grade* 
Management of ME-401 
Management of Toxicity 
Diarrhea/colitis 
2 
• 
Hold ME-401 unless diarrhea 
is confirmed to be due to 
an infectious agent. 
• 
If ME-401 is held, once diarrhea 
resolves to Grade ≤1, resume 
ME-401 at 60 mg administered on 
IS (the first 7 days of each 28-day 
cycle). 
• 
If infectious cause of the diarrhea, 
including clostridium difficile, 
was ruled out then proceed with 
following management steps:  
• 
Hold ME-401 and start 
loperamide or similar 
anti-diarrheal agent.  
• 
If no improvement occurs within 
48 hours, start prednisolone  
0.5–1 mg/kg IV or oral 
budesonide 9 mg daily.  
• 
Based on data from the Phase 1b 
study, subjects with diarrhea that 
was preceded by rash in the prior  
1–2 weeks often experienced 
more prolonged and severe 
diarrhea. Thus, if diarrhea is 
preceded by rash in the prior 
weeks, start prednisolone  
0.5–1 mg/kg IV or oral 
budesonide 9 mg daily at the time 
when a subject experiences rash.  
3 
• 
Hold ME-401 until AE resolves to 
Grade ≤1, then, if clinically 
indicated, resume ME-401 at 
60 mg administered on IS. 
• 
For recurrence of Grade 3 
diarrhea/colitis, discontinue study 
drug permanently. 
• 
Subjects should be hydrated 
as clinically indicated 
and administered (methyl) 
prednisolone 1–2 mg/kg/day IV 
or equivalent oral systemic 
steroids. 
o 
If no improvement occurs 
within 2–3 days, the 
corticosteroid dose should be 
increased to 2 mg/kg/day IV 
or equivalent oral systemic 
steroids.  
• 
Once improved to Grade ≤1, start 
tapering the corticosteroid as 
clinically indicated.  
4 
• 
Permanently discontinue ME-401. 
• 
Treat per institutional standard 
of care. 


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Table 4: 
ME-401 Toxicity Management (Continued) 
Toxicity 
Grade* 
Management of ME-401 
Management of Toxicity 
Cutaneous 
Reactions and 
Mucositis 
2 
• 
Hold ME-401 unless rash 
ascertained to be unrelated to study 
drug.  
• 
If ME-401 is held, once AE 
resolves to Grade ≤1, resume 
ME-401 at 60 mg administered on 
the IS.  
• 
Monitor subjects and determine 
cause of rash.  
• 
Per Investigator’s discretion, 
antihistamines, topical steroids, 
and systemic steroids may be 
given.  
≥3 
• 
Hold ME-401 until AE resolves to 
Grade ≤1, then, if clinically 
indicated, resume ME-401 at 
60 mg administered on the IS. 
• 
For recurrence of Grade 3, 
permanently discontinue ME-401. 
• 
Monitor subjects as clinically 
indicated until resolution. 
• 
Oral antihistamines and systemic 
corticosteroids such as 
prednisone 0.5–1 mg/kg/day 
(or equivalent dose of 
methylprednisolone) should be 
given until rash resolves to 
Grade ≤1. 
Any 
• 
Permanently discontinue ME-401 
for life-threatening toxicity, 
Stevens-Johnson syndrome of any 
grade, and TENS of any grade. 
• 
Treat per institutional standard 
of care. 
Hepatotoxicity 
2 
• 
For ALT/AST >3–5 × ULN but 
<5 × ULN, maintain ME-401 
treatment dose and schedule. 
• 
Monitor ALT/AST weekly until 
resolved to Grade ≤1 or baseline 
level. 
3 
• 
For ALT/AST >5–20 × ULN, hold 
ME-401.  
• 
Resume ME-401 at 60 mg 
administered on the IS. 
• 
For recurrence of Grade 3, 
permanently discontinue ME-401. 
• 
Monitor ALT/AST once a week 
until resolved to Grade ≤1 
or baseline level. 
• 
Treat per institutional standard 
of care, which may include 
a course of corticosteroids if 
clinically indicated. 
• 
Assess if Hy’s law applies. 
4 
• 
For ALT/AST >20 × ULN, 
permanently discontinue ME-401. 
• 
Assess if Hy’s law applies. 
• 
Treat per institutional standard 
of care, which may include 
a course of corticosteroids if 
clinically indicated. 
Non-infectious 
pneumonitis 
(NIP) 
2 
• 
Hold ME-401 until AE resolves to 
Grade ≤1. 
• 
Resume ME-401 at 60 mg 
administered on the IS. 
• 
Rule out infectious etiology of 
pneumonitis based on institutional 
guidance, utilizing following 
methods: chest X-ray, CT scan, 
pulsometry, nasal swab, sputum 
culture and sensitivity, blood 
culture and sensitivity, urine 
culture and sensitivity. 
• 
Treat with corticosteroids and per 
institutional standard of care. 


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Table 4: 
ME-401 Toxicity Management (Continued) 
Toxicity 
Grade* 
Management of ME-401 
Management of Toxicity 
 
≥3 
• 
Permanently discontinue ME-401. 
• 
Treat per institutional standard 
of care, including systemic 
corticosteroids. 
Lung Infection/ 
Pneumonia 
2 
• 
Holding ME-401 is not required. 
Continue ME-401. 
• 
Dosing may continue at 60 mg 
administered on the IS. 
• 
Treat per institutional standard 
of care. 
3 
• 
Hold ME-401 until AE resolved to 
Grade ≤1 or baseline. 
• 
Resume ME-401 at 60 mg 
administered on the IS. 
• 
Discontinue if same event occurs 
a third time. 
• 
Treat per institutional standard 
of care. 
4 
• 
Permanently discontinue ME-401. 
• 
Treat per institutional standard 
of care. 
Cytomegalovirus 
(CMV) 
Any 
• 
If test is positive (PCR or antigen 
assessment) based on local 
laboratory assessments 
and requires treatment, then hold 
ME-401 until recovery.  
• 
Resume ME-401 on the IS after 
resolution of asymptomatic CMV 
reactivation if all the following 
conditions are met: 
o 
CMV reactivation is 
resolved per the Investigator 
assessment 
• 
CMV reactivation was not 
associated with end organ disease 
(e.g., pneumonia, hepatitis, 
gastroenteritis, retinitis, 
encephalitis). 
• 
Treat per institutional standard 
of care. 
• 
If all conditions for restarting 
study drug are met, then 
secondary CMV prophylaxis must 
be maintained as per institutional 
guidelines.  
Hematological 
Toxicity 
3 
• 
Grade 3 neutropenia, febrile 
neutropenia or thrombocytopenia: 
ME-401 dosing may continue. 
• 
Perform CBC weekly until 
resolves to Grade <2 
(unless myelosuppression is due 
to follicular lymphoma). 
4 
• 
Grade 4 thrombocytopenia or 
Grade 4 neutropenia or febrile 
neutropenia: Hold ME-401 until 
AE resolves to Grade ≤3. Resume 
ME-401 at 60 mg administered on 
the IS. 
• 
Perform CBC weekly until 
resolves to Grade <3. 


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Table 4: 
ME-401 Toxicity Management (Continued) 
Toxicity 
Grade* 
Management of ME-401 
Management of Toxicity 
 
 
• 
Grade 4 neutropenia without 
associated symptoms: ME-401 
therapy may continue. If symptoms 
persist despite adequate supportive 
care, ME-401 should be held until 
toxicity resolves to Grade ≤1 
or baseline values and then 
ME-401 may be resumed at 60 mg 
administered on the IS. 
• 
Initiate growth factor support if 
clinically indicated. If subject is 
receiving filgrastim support or 
equivalent, CBC should be 
repeated after 3 days. Weekly 
CBCs should continue until 
resolves to Grade <3. 
 
 
• 
Grade 4 thrombocytopenia without 
associated symptoms, ME-401 
therapy may continue if 
Investigator deems safe. 
• 
Check platelets every 3 days until 
resolves to Grade <3. 
Other, not listed 
above, that are 
considered 
related to 
ME-401 
2 
• 
Holding ME-401 is not required. 
Continue ME-401. 
• 
Dosing may continue at the current 
dose or at 60 mg administered on 
the IS.  
• 
Treat per institutional standard 
of care. 
3 a 
• 
Hold ME-401 until AE resolves to 
Grade ≤1 or baseline.  
• 
Resume ME-401 at 60 mg 
administered on the IS. 
• 
For recurrence of the same 
Grade 3, permanently discontinue 
ME-401. 
• 
Treat per institutional standard 
of care. 
4 
• 
Permanently discontinue ME-401. 
• 
Treat per institutional standard 
of care. 
Abbreviations: AE = adverse event; ALT = alanine aminotransferase; AST = aspartate aminotransferase; 
CBC = complete blood count; CMV = cytomegalovirus; CT = computed tomography; CTCAE = Common 
Terminology Criteria for Adverse Events; IS = intermittent schedule; IV = intravenous; NIP = non-infectious 
pneumonitis; PCR = polymerase chain reaction; TENS = toxic epidermal necrolysis syndrome; ULN = upper limit 
of normal. 
* Toxicity graded per CTCAE v5.0. 
Hy’s law is defined as: cases of drug-related hepatocellular injury are defined as elevated ALT or AST ≥3 × ULN, 
plus elevated total bilirubin >2 × ULN without findings of cholestasis (defined as serum alkaline phosphatase [ALP] 
activity <2 × ULN), and no other reason can be found to explain. 
a. For Grade 3 AEs not related to study drug, it is upon the discretion of the Investigator if holding ME-401 is 
warranted. Treatment interruption must be discussed with the Sponsor’s Medical Monitor or designee in such 
cases. 
5.3.2. 
Adverse Events of Special Interest (AESIs) 
In this study, the following are considered AESIs: 
• Cutaneous reactions, including skin rash Grade ≥2 (see Appendix 3 for guidance for 
toxicity assessment)  
• Oral mucositis or stomatitis Grade ≥2  
• Diarrhea/colitis Grade ≥2  


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• It is important to differentiate infectious diarrhea from non-infectious diarrhea, as 
treatment and management of subjects will depend on the identified cause (see Table 4 
for management of non-infectious diarrhea). To confirm suspected colitis, utilize 
colonoscopy or diagnostic methods as per institutional standards.  
• AST/ALT Grade ≥2  
• Non-infectious pneumonitis (NIP) of any grade 
• Lung infection/pneumonia Grade ≥2 
• Cardiomyopathy of any grade 
Refer to Table 4 for ME-401 Toxicity Management. 
5.3.3. 
Other Potential Toxicities 
5.3.3.1. 
Phototoxicity 
Based on neutral red uptake phototoxicity assay of ME-401 in BALB/c 3T3 mouse fibroblasts, 
ME-401 did not demonstrate phototoxic potential. 
5.4. 
Monitoring and Prophylaxis of Opportunistic Infection  
5.4.1. 
Monitoring for Opportunistic Infection 
In addition to the clinical review and laboratory tests outlined in the Schedule of Assessments 
(Appendix 1), the following should be performed in all subjects prior to start of every cycle of 
treatment with ME-401: 
• Evaluation of any new onset or worsening of pulmonary symptoms (i.e., cough, dyspnea, 
or fever) that includes a lung examination at each visit prior to treatment to rule out 
opportunistic infection (OI), including PJP Pneumocystis jirovecii pneumonia/PCP 
(pneumocystis pneumonia).  
• Laboratory tests:  
o CD4 (for subjects with signs of infection) 
o Blood cultures if febrile neutropenia occurs, or when ANC by NCI CTCAE is 
Grade 4 
o PCR for CMV (every cycle for first 6 months of treatment and every 3 months 
thereafter). If PCR test is positive for CMV and treatment is initiated, then treatment 
with ME-401 should be delayed until recovery. Treatment of CMV should be 
initiated based on local SOC. Retreatment with ME-401 will be allowed on the IS 
once PCR test for CMV is negative. Please note: quantitative results from PCR 
measurements must be reported for all CMV assessments. 
Implement enhanced monitoring prior to start of every cycle of treatment with ME-401 when 
prior medical history or laboratory parameters could be associated with one of the following risk 
factors: 
• Intensive chemotherapy (≥2 lines of myelosuppressive cytotoxic therapy) 


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• History of symptomatic CMV infection, systemic herpes 
• History of lower respiratory tract infection 
• History of immunodeficiency in the last 12 months (excluding lymphoma) 
• Lymphocyte count <500/mm3 while on treatment in clinical study 
For subjects with identified risk factors and those who developed an OI while on study treatment, 
additional assessments may include: 
• CD4 and CD8 count and ratio, c-reactive protein, blood cultures 
• Any additional laboratory and diagnostic methods according to local SOC reported as 
unscheduled laboratory and diagnostic methods of assessment  
• Radiological imaging (i.e., chest X-ray or CT scans) 
o Note: Treatment of the OI that started while on study should be based on local SOC. 
5.4.2. 
Prophylaxis of OI Other Than PJP/PCP 
Mandatory prophylactic therapy is not mandated in all subjects for the following reasons: 
• Mandatory prophylaxis may cause a higher risk of side effects associated with supportive 
treatment where no risk factors are present 
• The current schedule of assessments and additional steps noted above provide frequent 
monitoring and flexibility for prophylaxis based on local SOC 
Although not mandated in all subjects, OI prophylaxis may be initiated at the discretion of the 
treating Investigator’s judgment of the risk-benefit ratio in any subject, irrespective of whether 
a high-risk feature is present. Treatment, dosage, and route of administration must be reported on 
the concomitant medication page of the eCRF.  
Prophylactic treatment of OI should be initiated based on SOC in subjects when high risk factors 
are identified (Section 5.4.1). 
5.4.3. 
Prophylaxis of PJP/PCP 
Prophylaxis against PJP/PCP is mandatory; the preferred treatment is trimethoprim-
sulfamethoxazole (TMP-SMX) option because of ease of administration and is typically 
achieved with one tablet (TMP 80 mg/SMX 400 mg) administered once or twice daily, 
3 times/week on consecutive or alternate days. However, TMP/SMX is (1) a selective inhibitor 
of CYP2C8, a cytochrome P450 isoform that affects zandelisib metabolism, and (2) can be 
associated with myelosuppression, requiring a switch to an alternate option. If TMP-SMX is not 
tolerated, PJP prophylaxis with either atovaquone or aerosolized pentamidine is recommended. 
Because dapsone has been associated with methemoglobinemia in the zandelisib Phase 1b study, 
it should not be used for PJP prophylaxis.  
The mandatory PJP prophylaxis should start within one week of starting zandelisib until the 
completion of 30-day safety follow up from the last dose of zandelisib treatment.  
When Prophylaxis is used for OI and PJP, treatment, dosage, and route of administration must be 
reported on the concomitant medication page of the eCRF.  


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5.5. 
Prohibited Treatments and Cautions for Concomitant Medications 
The following medications/therapies are prohibited during the study: 
• Other investigational agents 
• Cancer therapies not mentioned in this protocol 
• Dapsone (for PJP prophylaxis) 
• Drugs that are strong inhibitors/inducers of CYP3A4 (Appendix 8)  
• Systemic corticosteroids in doses >10 mg in prednisone equivalency are prohibited for 
one week prior to Cycle 1 Day 1 and during the study, except when used:  
1) for the treatment of an adverse reaction or other acute clinical situations 
2) for the treatment of an exacerbation of known chronic conditions 
The following medications/therapies should be used with caution  
• Drugs that are strong inhibitors/inducers of CYP2C8 (Appendix 7).  
• Drugs that are known to prolong the QT/QTc interval should be used with caution 
(Appendix 9) 
• Orally administered drugs known to interact with drugs that inhibit the intestinal 
transporters BCRP and P-gp should be used with caution 
Note: Palliative radiation therapy of non-curative intent is permitted on study except to target 
and non-target lesions.  
5.6. 
Advisory on COVID-19 Vaccination, Management, and Risk 
Assessment 
COVID-19 vaccination, where authorized or approved by national regulatory authorities, is 
recommended for patients who are currently enrolled in this study, in accordance with 
COVID-19 vaccination guidance from scientific and medical associations (e.g., American 
Society of Hematology, American Society of Clinical Oncology, NCCN, and European 
Hematology Association).  
The Sponsor completed a risk assessment for COVID-19 vaccination of patients while on study 
drug and concluded the benefit of receiving anti-lymphoma therapy for patients with R/R FL and 
MZL outweigh the potential risk of delaying treatment due to scheduled COVID-19 vaccination. 
Potential risks associated with COVID-19 vaccination while on study drug have not been fully 
established. 
Vaccination is recommended, whether administered prior to enrollment or during study 
participation. Investigators are advised to adhere to local regulatory and institutional guidance 
for vaccination.  
Information about vaccination should be recorded as a concomitant medication with the dates of 
each dose of vaccination.  


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If COVID-19 infection is confirmed, COVID-19 infection should be managed per local guidance 
until the subject recovers. In the event of a positive COVID-19 case, the investigator must 
determine the risk and benefit of interruption versus continuation of zandelisib. Zandelisib 
should be temporally interrupted. After the subject recovers from the COVID-19 event, 
zandelisib treatment should be resumed. Permanent discontinuation of study drug (if COVID-19 
test is positive or if symptomatic COVID is diagnosed) must be discussed with the study medical 
monitor. If a subject is found to have a positive COVID-19 test, enter the COVID-19 results in 
the AE CRF page. Also, enter any AEs associated with COVID-19 infection in the AE CRF 
page.  
5.7. 
Discontinuation of ME-401 Treatment 
Administration of ME-401 will continue unless one of the following occurs: 
• Progressive disease, unless the Investigator deems a switch to continuous dosing is in the 
best interest of the subject and the subject agrees to continue treatment with CS dosing  
• Unacceptable AE(s) considered related/possibly related to ME-401 despite appropriate 
therapy and modified dosing schedule 
• Withdrawal of consent by the subject 
• Changes in the subject’s medical condition that render further administration of ME-401 
unacceptable in the judgement of the Principal Investigator or Sponsor 
• Non-compliance 
• Termination of study by Sponsor 
Subjects who discontinue from therapy should be followed for efficacy and survival unless they 
withdraw consent to participate. 
5.8. 
Discontinuation of Subjects from Study 
Subjects may be discontinued from the study for any of the following reasons: 
• Withdrawal of consent for further participation or follow-up 
• Subject is lost to follow-up 
• Termination of study by Sponsor 
5.9. 
Site Discontinuation 
The Sponsor has the right to close a site at any time. Reasons for closing a site may include, but 
are not limited to, the following: 
• Non-compliance with International Council for Harmonisation Good Clinical Practice 
(ICH-GCP) guidelines 
• Inadequate rate of subject recruitment 
• Poor protocol adherence 
• Inaccurate or incomplete data recording 


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• No study activity (i.e., all subjects have completed, and all obligations have been 
fulfilled)  
5.10. 
Study Termination 
Study termination is defined as the time when all study treatments, study-specific assessments, 
and study data collection are completed. Upon termination of the study, the Sponsor or designee 
will conduct site closure activities with the Investigator or site staff (as appropriate), in 
accordance with applicable regulations. 
The Sponsor reserves the right to temporarily suspend or terminate the study at any time for 
reasons including, but not limited to, safety issues or ethical reasons. The Sponsor or designee 
will also promptly inform the relevant regulatory authorities of the suspension/termination along 
with the reasons for such action, when applicable. Where required by applicable regulations, the 
Investigator or head of the medical institution must inform the IRB/EC. 
6. 
SAFETY PARAMETERS, DEFINITIONS, AND REPORTING 
Safety assessments will consist of monitoring and recording AEs, serious AEs (SAEs), 
non-serious AESIs listed in Section 5.3.2; performing protocol-specified laboratory assessments; 
measuring -protocol-specified vital signs; and conducting other protocol-specified tests that are 
deemed critical to the evaluation of ME-401.  
The Investigator will assess AEs for severity and relationship to study drug and determine if 
an AE meets the criteria for an SAE. Each AE will be graded in the source documents using 
NCI CTCAE v5.0. 
All AEs observed within 30 days after study drug last dose should be reported recorded. All 
SAEs will be reported for up to 90 days after the last dose of study drug. All SAEs that are 
deemed related to the study drug must be reported even if beyond 90 days after last dose of study 
drug, if they become known to the investigator. All deaths should be reported and communicated 
to the sponsor or designee. Death not due to disease progression should be reported as an SAE. 
Death associated with disease progression is not an SAE.  
6.1. 
Adverse Event (AE) 
An AE is any untoward medical event that occurs in a subject following the start of study drug 
administration, whether or not the event is considered drug-related (ICH Guideline E6 §1.2). 
An AE can therefore be any of the following: 
• A pre-existing medical condition can be recorded as an AE if the condition worsens by 
at least one grade following the start of study drug administration and if the frequency, 
severity, or character of the condition worsens during the study. When recording on the 
eCRF it is important to capture applicable descriptors (e.g., more frequent arthritic pain).  
• Disease-related out-of-range laboratory values will not be considered AEs/SAEs if there 
is no change from the screening laboratory values. Any deterioration in a laboratory value 
or other clinical test that is associated with symptoms, or leads to a change in study 


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treatment or concomitant treatment, or discontinuation from study product, or deemed 
clinically significant by the Investigator will be considered an AE. 
Adverse events will be recorded using the terminology defined in the NCI CTCAE v5.0.  
Adverse events will be captured from the time of the first dose of study drug on Cycle 1 Day 1 
and continue until 30 days after the last dose of study drug.  
Any TEAE of Grade 2 or higher should be followed for resolution until either: 
• The start of subsequent anti-cancer therapy 
• Death 
A TEAE is defined as an AE starting after the first dose until 30 days after the last dose of study 
drug. 
Death related to disease progression is not considered an AE. Signs and symptoms related to 
disease progression are not considered AEs. Anticipated fluctuations of pre-existing conditions, 
including the disease under study that do not represent a clinically significant exacerbation 
or worsening, are not considered AEs. 
Only one AE term should be recorded in the event field on the AE eCRF if a specific AE is 
attributable to a primary diagnosis. Adverse events that are secondary to other events should be 
identified by their primary cause, with the exception of severe or serious secondary events. 
For example: 
• If a subject initially had diarrhea and is subsequently diagnosed with colitis, this event 
should be consolidated to one event, colitis  
• If a subject had diarrhea that resulted in mild dehydration, only diarrhea should be 
reported in the eCRF 
• If a subject who had diarrhea developed acute renal failure, both acute renal failure 
and diarrhea should be reported in the eCRF  
For AEs, a diagnosis should be recorded on the AE eCRF rather than individual signs 
and symptoms (e.g., record only colitis rather than diarrhea, abdominal pain, decreased appetite). 
However, if a constellation of signs and symptoms cannot be medically characterized as a single 
diagnosis or syndrome, each individual event should be reported on the AE eCRF. If a diagnosis 
is subsequently established, all previously reported AEs based on signs and symptoms should be 
nullified and replaced on the AE eCRF based on a single diagnosis, with a starting date that 
corresponds to the starting date of the first symptom of the eventual diagnosis. 
6.2. 
Assessment of Severity 
The term “severity” is used to describe the intensity of an AE. Severity will be graded according 
to NCI CTCAE v5.0. For AEs not covered by CTCAE, each AE will be assigned a category by 
the Investigator as described in Table 5. 


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Table 5: 
NCI CTCAE v5.0 Adverse Event Grading System 
Grade 
Comments 
1 
Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not 
indicated 
2 
Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate 
instrumental ADL a 
3 
Severe or medically significant but not immediately life-threatening; hospitalization 
or prolongation of hospitalization indicated; disabling; limiting self-care ADL b 
4 
Life-threatening; urgent intervention indicated 
5 
Death related to AE 
Abbreviations: ADL = activities of daily living; AE = adverse event 
a. Instrumental ADL refer to preparing meals, shopping for groceries or clothes, using the telephone, managing 
money, etc. 
b. Self-care ADL refer to bathing, dressing and undressing, feeding self, using the toilet, taking medications, and not 
bedridden. 
6.3. 
Action Taken with Study Drug in Response to the AE  
The Investigator will record the action taken regarding the study treatment in response to an AE: 
• Dose not changed 
• Dosing schedule changed from CS to IS 
• Drug interrupted 
• Drug withdrawn 
• Not Applicable 
• Unknown 
6.4. 
Outcome of AE 
The Investigator will record the outcome of the AE as follows: 
• Recovered/resolved 
• Recovered/resolved with sequelae  
• Not recovered/not resolved 
• Death 
• Unknown 
All AEs should be followed until the event has resolved or the condition is stabilized as assessed 
by the Investigator. 


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6.5. 
Serious Adverse Event (SAE) Definition 
Per ICH Guideline E2A §II.B, an SAE is any untoward medical occurrence (i.e., AE) that at any 
dose results in any of the following outcomes: 
• Death 
• A life-threatening condition  
• An inpatient hospitalization or prolongation of an existing hospitalization 
• A persistent or significant disability/incapacity or substantial disruption of the ability to 
conduct normal life functions 
• A congenital anomaly/birth defect 
• Any other medically important event: defined as an event that may jeopardize the subject 
or may require intervention to prevent one of the outcomes listed above. Medical 
and scientific judgement must be exercised in deciding whether an event is serious. 
Any new primary cancer must be reported as an SAE. 
The term “life-threatening” in the definition refers to an event in which the subject was at risk 
of death at the time of the event; it does not include any AE that had it occurred in a more severe 
form or was allowed to continue might have caused death. 
Elective hospitalizations not in response to an AE are not considered SAEs. Hospitalizations 
related to disease progression are not considered SAEs. A procedure (e.g., surgery) is not an AE 
in itself, but the reason for the procedure may be an AE. Admission for social reasons, without 
an underlying medical condition, including hospice, will not be reported as an SAE.  
• After informed consent has been obtained and prior to start of study drug, only SAEs 
caused by a protocol-mandated intervention (e.g., invasive procedures such as biopsies) 
should be reported (see Section 6.6 for SAE reporting instructions). 
• All SAEs will be reported for up to 90 days after last dose of study drug. After 90 days 
following last dose of study drug, SAEs deemed related to study treatment must be 
reported to the Sponsor or Sponsor’s designee if they become known to the Investigator.  
Severity and seriousness are independent of each other and need to be independently assessed for 
each AE recorded on the eCRF.  
6.6. 
Serious Adverse Event Reporting 
All SAEs must be reported to the Sponsor or Sponsor’s designee (the Contract Research 
Organization [CRO]), namely Parexel, within 24 hours of the Investigator becoming aware of the 
SAE.  
To report an SAE, please refer to the applicable study manual or SAE Form instructions. 
An SAE shall be reported to MEI254808@Parexel.com.  
Sites must follow-up with Parexel to confirm receipt of the SAE Report.  
The 4 minimum criteria for a valid SAE report include: 
• Study identifier 


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• Subject identifier 
• Event term 
• Study drug 
The Investigator must report new significant follow-up information for these events to the CRO 
immediately (i.e., no more than 24 hours after becoming aware of the information). New 
significant information includes the following: 
• New signs or symptoms or a change in the diagnosis 
• Significant new diagnostic test results 
• Change in causality based on new information 
• Change in the event’s outcome, including recovery 
• Additional narrative information on the clinical course of the event 
All SAEs also must be reported by each site to the appropriate IRB/EC in accordance with local 
requirements for reporting SAEs to their IRB/EC. 
Reporting requirements will also be based on the Investigator's assessment of causality 
and seriousness, with allowance for upgrading by the Sponsor. 
All deaths should be recorded and reported to the Sponsor or Sponsor’s designee. Disease 
progression, hospitalization to assess disease progression, and death due to disease progression 
as determined by the Investigator, are not considered SAEs. All other causes of death must be 
reported as SAEs. The Investigator should make every effort to obtain and send death certificates 
and autopsy reports for all deaths to the Sponsor. 
In the event of a medical emergency (requiring immediate attention regarding operation of the 
clinical study and/or the use of study product), study site staff will apply appropriate medical 
intervention according to current standards of care and will contact the Medical Monitor or 
designee (e.g., CRO representative) for further consultation and guidance. The Sponsor will be 
reporting all AEs, SAEs, and expedited safety reports (suspected unexpected serious adverse 
reactions [SUSARs]) according to local regulatory requirements.  
6.7. 
Relationship of Adverse Events to Study Drug 
The Investigator is obligated to assess the relationship between study drug and the occurrence 
of each AE. The Investigator is to use his/her best medical judgement in determining the likely 
relationship of the AE to study drug. The relationship of an AE or SAE to study drug is to be 
classified as: 
• Related 
• Possibly Related 
• Not Related 


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6.8. 
Pregnancies 
To ensure subject safety, each pregnancy in a subject on study treatment must be reported within 
24 hours of learning of its occurrence on a Pregnancy Report Form using email and fax number 
listed in Section 6.6. Pregnancy occurring up to 3 months after receiving the last dose of study 
drug must be reported. 
Study drug must be discontinued in a subject who becomes pregnant. The pregnancy must be 
followed to determine outcome, including spontaneous or voluntary termination, details of the 
birth, and the presence or absence of any birth defects, congenital abnormalities, or maternal 
and/or newborn complications. Subjects must be followed at least 8 weeks after giving birth 
to a child. 
Pregnancy is not an AE but should be recorded on a Pregnancy Form and reported by the 
Investigator to the Sponsor or Designee. Pregnancy follow-up should be recorded and should 
include an assessment of the possible relationship to the study product of any pregnancy 
outcome. Any SAE experienced during pregnancy must be reported on the SAE Report Form 
as described in in Section 6.6. Examples of pregnancy outcomes that are SAEs include reports 
of: 
• Congenital anomalies or developmental delay in the fetus or the child 
• Fetal death or spontaneous abortion 
• Suspected adverse reactions in the neonate that are classified as serious 
Pregnancy outcomes should be collected for the female partners of any males who took study 
treatment in this study. Consent to report information regarding these pregnancy outcomes 
should be obtained from the mother. 
6.9. 
Data Safety Monitoring Board 
A Data Safety Monitoring Board (DSMB) will be established to review safety and efficacy data 
collected in the study on a regular basis with the primary purpose of protecting the safety of trial 
participants, the credibility of the trial, and the validity of trial results. It is expected the DSMB 
will meet at least once every 6 months but may meet more frequently if deemed necessary. 
Details regarding the DSMB are contained in a separate DSMB Charter document. 
7. 
RESPONSE ASSESSMENT AND STUDY ENDPOINTS 
7.1. 
Overview 
Efficacy assessments will be based on the Modified Lugano Classification response criteria 
(Appendix 5).  
Response to treatment based on the Investigator evaluation of imaging tests will be the basis for 
subject management.  
An IRRC composed of radiologists and oncologists will review all imaging tests, and clinical 
data if necessary, to determine disease response and progression. All radiographic images/scans 
will be sent by the study sites to the central imaging vendor and archived for potential future 


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evaluation (e.g., evaluation using Cheson criteria). The process for image collection 
and transmission to the central imaging vendor can be found in the Study Manual. 
Tumor imaging (neck, chest, abdomen, and pelvis) should be performed by contrast-enhanced 
CT and/or FDG-PET scan. Because FL and MZL is typically (FDG)-avid, as per Modified 
Lugano Classification (Appendix 5) FDG-PET scan is the preferred method for disease/response 
assessment to confirm CR. If a combined/dual PET/CT scanner is the available modality, then 
the CT portion of a PET/CT may be used instead of a dedicated CT provided that the CT scan is 
performed with a high-resolution imaging quality to ensure optimal measurement for 
disease/response assessment. CT scans must be performed using intravenous (IV) contrast. For 
subjects who are intolerant to contrast agents, the CT scan will be performed with oral contrast 
or MRI scans will be used. If a PET/CT is not available or is not evaluable, bone marrow biopsy 
will be utilized to confirm a morphologic CR documented by CT scan. The imaging schedule is 
described in Appendix 1- Response Assessment. In summary, imaging scans (CT scan 
or PET/CT scan) will be obtained with the following frequency: 
• Every 2 months (±7 days) for the first 6 months on study  
• Every 3 months (±7 days) for the next 12 months on study  
• Every 6 months (±14 days) thereafter, starting with month 18 
For subjects with gastric MZL, endoscopy with biopsy must be performed to confirm CR.  
For those subjects who switch to CS after disease progression on the IS, the date of PD will be 
designated as a new baseline, and subsequent response assessments will be scheduled per 
the protocol-specified frequency (Appendix 1- Response Assessment): every 2 months for the 
first 6 months, then every 3 months during the next 12 months up to month 18, and every 
6 months thereafter. The date of PD and CT scan at PD will be the new baseline date and CT 
scan for reference for assessment of recapture of response. The determination that a subject has 
experienced disease progression should be based on the results of a diagnostic quality CT or 
FDG-PET scan performed with contrast. Please refer to the Schedule of Assessments for details 
(Appendix 1).  
To rule out a potential of false positive diagnosis of disease progression in ambiguous cases, it is 
highly recommended to repeat imaging assessments within 4–8 weeks after initial suspicion of 
PD. In case of unequivocal disease progression, repeat of scans is not necessary.  
7.2. 
Efficacy Endpoints 
The primary analysis of ORR for FL will be triggered after all 91 subjects in the FL PEP enrolled 
in the study (except those who discontinue early) have been followed for at least 6 months from 
the start of study drug. 
The final analysis of ORR and DOR for MZL will be triggered after all 32 MZL subjects 
enrolled in the study (except those who discontinue early) have been followed for at least 
14 months from the start of study drug.  


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7.2.1. 
Objective Response Rate (ORR) 
The primary efficacy endpoint is ORR as determined by the IRRC, defined as the proportion 
of subjects achieving the best response rating of CR or PR based on the Modified Lugano 
Classification. ORR will also be assessed by the Investigator as a secondary endpoint. 
7.2.2. 
Duration of Response (DOR) and Duration of Recaptured Response (DORR) 
Duration of response (DOR) will be evaluated as a secondary endpoint and is defined as the time 
from documentation of the first CR or PR to the time of first disease progression based on the 
Modified Lugano Response Criteria. The endpoint DOR will be assessed by the IRRC and 
Investigator independently. To evaluate the duration of response for subjects who had recapture 
of response (Section 7.2.5) on the CS dosing, duration of recaptured response (DORR) is defined 
as the time from recapture of response to the time of second disease progression. The endpoint 
DORR will be assessed by the Investigator only.  
7.2.3. 
Progression-Free Survival (PFS) 
Progression-free survival (PFS) will be assessed as a secondary endpoint and is defined as the 
time from initiation of treatment (Day 1) until first disease progression or death from any cause. 
Disease progression criteria are defined per Modified Lugano Response Criteria and will be 
assessed by the IRRC and Investigator independently.  
7.2.4. 
Time to Treatment Failure (TTF)  
Time to treatment failure will be assessed as a secondary endpoint and is defined as the time 
from first dose of study drug to treatment failure. 
Treatment failure is defined as any treatment discontinuation due to disease progression, toxicity, 
or death. 
7.2.5. 
Recapture of Response  
Recapture of response is defined as achieving a second documented CR or PR after experiencing 
the first PD.  
Recapture of response rate will be assessed by the Investigator as a secondary endpoint, and is 
defined as the proportion of subjects who achieved a CR or PR followed by a PD during IS 
dosing and subsequently switched to CS dosing and achieved a second documented CR or PR. 
Time to recapture of response will be assessed as a secondary endpoint, and is defined as the 
time from the first PD until the second documented CR or PR.  
7.2.6. 
Overall Survival (OS) 
Overall survival (OS) will be assessed as a secondary endpoint and is defined as the time from 
initiation of treatment (Day 1) until death from any cause independently from the study period 
or dosing schedule. Overall survival time will be censored at the last date the subject is known to 
be alive when the confirmation of death is absent or unknown.  


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7.3. 
Safety Endpoints 
7.3.1. 
Overall Incidence Rate of Treatment-Emergent Adverse Events (TEAEs) 
A TEAE is defined as any new or worsening AE occurred after first dose of study drug until 
30 days after last dose of ME-401. 
The overall incidence rate of TEAEs is a secondary safety endpoint. It is calculated as the 
proportion of subjects with at least one TEAE.  
The overall incidence rate of SAEs will also be calculated. 
7.3.2. 
Incidence Rate of AESIs 
The rate of AESIs is a secondary safety endpoint. It is calculated as the proportion of subjects 
with at least one AESI. 
7.3.3. 
Time to Occurrence of AESIs 
Time to occurrence of AESIs will be assessed as a secondary endpoint and is defined as the time 
from first dose of study drug to first occurrence of an AESI. 
7.3.4. 
Other Safety Endpoints 
Other safety endpoints are the additional safety measures described in Section 11.2.2. 
7.4. 
Pharmacokinetics 
To enable PK/PD modeling, sparse PK sampling will be obtained on all subjects as shown in 
Table 6.  
Table 6: 
Pharmacokinetics Sampling Schedule 
Cycle 
Sampling Time Within a Cycle a 
Cycle 1 
• 
Day 1: pre-dose 
• 
Day 15:  
o pre-dose  
o 3 hours post intake of study drug 
Cycle 2 
• 
Day 1: pre-dose 
Cycle 4 and Cycle 6 
• 
Day 7: pre-dose 
a. Pre-dose PK samples are to be obtained within 1 hour before study drug administration on Cycle 1 Day 1, Cycle 2 
Day 1, Cycle 4 Day 7, and Cycle 6 Day 7. Pre-dose PK samples collected after Cycle 1 Day 1 are to be obtained 
within ±60 minutes of the scheduled time, which is based on the Cycle 1 Day 1 dosing time. For the 3 hours 
post-dose sample on Cycle 1 Day 15, the allowable window is ±30 minutes from the indicated time. 
For better modeling of efficacy/exposure and safety/exposure, an accurate estimate of the time 
from drug intake to PK sample collection is needed. Collect accurate study drug dosing history 
(i.e., time of intake) on days when PK samples are collected. The subject will be asked to record 
information on food intake on the day preceding and on the day of the visit on which blood 
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Detailed instructions on collection of plasma for determination of ME-401 PK will be provided 
in a study manual. 
8. 
STUDY PROCEDURES 
See the Schedule of Assessments (Appendix 1) for a detailed list of study procedures 
and assessments. Instructional details (where applicable) for procedures are contained in 
Section 9. 
8.1. 
Informed Consent 
Each subject’s signature must be obtained on a written Inform Consent Form (ICF) that has been 
approved by an IRB, EC, or Ethical Review Board (ERB) prior to any study-specific procedures 
being performed. The ICF must incorporate a Release of Medical Information that authorizes 
release of medical records to the trial Investigators, monitors, Sponsor and its designees, 
the Food and Drug Administration (FDA), or other regulatory authority. The ICF must be in 
a language fully comprehensible to the prospective subject. The consenting process must be 
documented in the medical chart and a signed copy of the ICF provided to the subject. 
8.2. 
Screening Period 
Subjects will be screened during the 28-day period prior to Cycle 1 Day 1 to ensure they meet 
the entry criteria for the study. During the screening period, the Investigator should carefully 
review each subject’s medical history to ensure eligibility. All inclusion and exclusion criteria 
should be supported by corresponding documentation in the medical records, including 
a pathology report confirming the diagnosis of FL or MZL. 
The Investigator or an appropriate designee will obtain informed consent from each subject 
prior to conducting any study-specific procedures. Bone marrow biopsies performed as part 
of the subject’s medical care within 12 weeks of Cycle 1 Day 1 may be used for screening 
and eligibility determination. PET/CT or CT scans performed within 6 weeks of Cycle 1 Day 1 
may be used for screening and eligibility determination as long as they meet the minimum 
requirements per Lugano criteria (i.e., includes all appropriate anatomical areas of the body, 
a slice thickness ≤5 mm). This avoids unnecessary repeat tests and undue burden on subjects. 
8.3. 
Subject Enrollment 
As of this amendment, 121 subjects with FL and 32 subjects with MZL have been enrolled and 
treated with ME-401 on the IS. Enrollment in this study is now closed.  
Subjects will be enrolled using an Interactive Web Response System (IWRS) process.  
8.4. 
Cycle 1 Day 1 Tests and Administration of ME-401 
All Cycle 1 Day 1 tests and procedures outlined in the Schedule of Assessments (Appendix 1) 
must be performed prior to study drug administration. Laboratory assessments need not be 
repeated on Cycle 1 Day 1 if they were performed during screening period within 7 days prior to 
the start of treatment. Physical examination need not be performed on Cycle 1 Day 1 if it was 


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performed during screening within 7 days prior to the start of treatment. The Investigator should 
assess any changes in the subject’s medical condition or history since the Screening visit. 
A record of the administration of the first dose of study drug will be documented in the source 
documents. 
8.5. 
Treatment Phase 
The treatment phase begins from the time study drug is first administered (Day 1 of Cycle 1), 
and continues until the subject discontinues study treatment. Each cycle during the treatment 
period will consist of 28 days and will be denoted numerically (Cycle 1, Cycle 2, etc.). 
After the initial 2 cycles of therapy, study visits will occur on Day 1 of each cycle for Cycles 3 
to 7, then every 3 cycles thereafter (i.e., Day 1 of Cycle 10, 13, etc.) as long as the subject 
continues to receive study drug. The modified dosing schedule for AEs is described in Table 4.  
Response assessments, as defined in Section 7, will be performed according to the Schedule of 
Assessments (Appendix 1) and detailed in Appendix 5. The disease/response assessment will be 
performed every 2 months (±7 days) for the first 6 months on study, every 3 months (±7 days) 
during the following 12 months, and every 6 months (±14 days) thereafter.  
For those subjects who switch to CS after disease progression on IS, the date of PD will be 
designated as a new baseline, and subsequent response assessments will be scheduled per 
the protocol-specified frequency using the new baseline date and CT scan for reference.  
8.6. 
End of Treatment (EOT) Visit (30-Day Safety Follow-up Visit) 
Upon discontinuation of ME-401, an End of Treatment (EOT)/30-Day Safety Follow-up visit 
will occur 30 (±3) days from the last dose of study drug. 
All ongoing and new AEs should be reviewed and entered on the eCRF. All assessments as 
indicated per the Schedule of Assessments should be completed (Appendix 1). 
8.7. 
Post-Treatment Long-Term Follow-up 
All subjects who stop study drug treatment will be followed long-term by the site every 3 months 
from the last dose of study drug to obtain data regarding changes in efficacy assessments, death, 
or subsequent therapy based on SOC assessments, which includes tumor imaging tests based on 
the original protocol-defined schedule for response assessment. This will be captured on the 
post-treatment eCRF. All subjects will be followed for survival. Survival follow-up will continue 
for up to 3 years after the last subject is enrolled in the study. Follow-up will be discontinued if 
the subject withdraws consent to participate in the study. 
9. 
STUDY ASSESSMENTS 
The following sections describe the methods for assessments of clinical and functional outcomes 
included in the trial. Refer to the Schedule of Assessments (Appendix 1) for frequency of 
assessments. 


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9.1. 
Medical History 
A complete medical history will be collected during screening. Data associated with any 
interventional procedure, surgical operation (for any reason), and all prior cancer therapies will 
be collected. If known, record in the medical history information on COVID-19 vaccination 
administered at any time prior to subject consent to participate in the study. 
9.2. 
Demographics 
Subject demographic data including date of birth (year of birth required at a minimum), sex, 
and ethnicity and/or race will be recorded in the eCRFs. 
9.3. 
Concomitant Medication  
Data on concomitant prescription and over-the-counter medications will be collected. This data 
will be recorded from 28 days prior to Cycle 1 Day 1 and continues until 30 days after the last 
dose of study drug or start of new anti-cancer therapy. Information on COVID-19 vaccination 
administered while in the study, i.e., from when the subject signs the ICF until the subject 
discontinues from the study should be recorded as concomitant medication.  
9.4. 
Physical Examination and Vital Signs 
The physical examination will include the measurements of height (Screening visit only) 
and weight, and examination of all organ systems (at the Investigator’s discretion, genitourinary 
system may be excluded). Vital sign measurements will include systolic and diastolic blood 
pressure, heart rate, and body temperature. 
A symptom-directed exam will be performed on Cycle 1 Day 8, Cycle 1 Day 15, and Cycle 2 
Day 15, only if needed/indicated, based on interim history. 
Cutaneous assessments: For subjects who develop a rash, a detailed skin assessment is required 
(see Appendix 3). If a dermatology referral is warranted, biopsy results (if any) will be collected, 
including assessments from dermatology and pictures of rashes.  
Gastrointestinal assessments: For subjects who develop diarrhea and/or colitis and undergo 
colonoscopy and biopsy, the results will be collected in the eCRF including pathology results.  
9.5. 
Adverse Events 
Adverse events will be collected at each visit as described in Section 6, and will be recorded 
from the time of dosing on Cycle 1 Day 1 until 30 days after last dose of study drug or start 
of new anti-cancer treatment. 
Any SAE due to protocol-mandated procedures will be collected from the time of informed 
consent. Other SAEs will be collected from the start of study drug.  
9.6. 
Cardiac Evaluation 
12-lead ECGs will be performed at the following visits: 
• Screening 


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• Pre-dose on Cycle 1 Day 1 (unless the Screening visit is within 3 days prior to Day 1) 
• Pre-dose on Day 1 of Cycles 3, 6, 13, 19, and 25 
• On Day 1 of Cycles 1 and 3 at the expected time of Cmax (3–5 hours after dosing) 
• After Cycle 25, every 6 months, pre-dose 
• EOT/30-Day Safety Follow-up visit 
• As clinically indicated 
Left ventricular ejection fraction measurement by echocardiogram or MUGA will be performed 
at screening, EOT/30-Day Safety Follow-up visit, and as clinically indicated. 
9.7. 
Laboratory Tests 
Laboratory tests listed on the Schedule of Assessments (Appendix 1) will be analyzed at the 
investigative site’s local laboratory and used in all safety analyses and response assessments 
(see Appendix 5); PK samples discussed in Section 7.4 will be shipped to a central laboratory 
for analysis. 
The following tests are to be performed: 
• CBC with differential: white blood cell (WBC), ANC, red blood cell (RBC), hemoglobin 
(HGB), hematocrit (HcT), lymphocytes, eosinophils, monocytes, basophils, platelets, 
mean corpuscular hemoglobin (MCH) 
• Serum chemistry: glucose, blood urea nitrogen (BUN) or urea, creatinine, sodium, 
potassium, chloride, calcium, alkaline phosphatase (ALP), AST, ALT, total bilirubin, 
total protein, albumin, LDH, phosphorus 
• Coagulation: activated partial thromboplastin time (aPTT), prothrombin time (PT)/ 
international normalized ratio (INR) 
• Urinalysis: Dipstick or urine test (pH, specific gravity, glucose, ketones, blood, protein) 
• Pharmacokinetic sampling (see Section 7.4) 
• Pregnancy test 
• HIV testing 
• Hepatitis B and Hepatitis C testing 
• PCR test for CMV 
• OI monitoring:  
o CD4 (for subjects with signs of infection) 
o Blood cultures if febrile neutropenia occurs, or when ANC is NCI CTCAE Grade 4 
o PCR for CMV (every cycle for first 6 months of treatment and every 3 months 
thereafter). If PCR test is positive for CMV, treatment should be delayed until 
recovery. Treatment of CMV should be initiated based on local SOC. Retreatment 


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with ME-401 will be allowed without dose reduction once PCR test for CMV is 
negative. 
Subjects with identified risk factors will have the following tests performed: 
o CD4 and CD8 count and ratio, c-reactive protein, blood cultures 
o Any additional laboratory and diagnostic methods according to local SOC reported as 
unscheduled laboratory and diagnostic methods of assessment  
o Radiological imaging (i.e., chest X-ray or CT scans) 
Note: Treatment of the OI that started while on study should be based on local SOC. 
• Bone marrow aspiration/biopsy: performed at screening at the discretion of the 
Investigator and repeated as indicated for subjects with known or suspected marrow 
involvement. Bone marrow aspiration/biopsy is to include surgical pathology, 
immunohistochemistry, and flow cytometry as per SOC. Bone marrow aspiration/biopsy 
is required to confirm a CR when a nodal CR is documented by a CT scan.  
• There is no requirement for routine COVID-19 testing. However, if a patient is found to 
have a positive COVID-19 test, enter the COVID-19 results in the AE CRF page. Also, 
enter any AEs associated with COVID-19 infection in the AE CRF page.  
9.8. 
Disease/Response Assessment 
All CT scans, PET/CT scans, bone marrow biopsy, and laboratory evaluation will be completed 
as appropriate for individual subjects (scan should be conducted using IV, or oral if indicated, 
contrast enhanced CT/MRI and/or PET-CT imaging modalities). Efficacy assessments will be 
based on the Modified Lugano Classification response criteria in Appendix 5. 
All radiographic images/scans will be sent by the study sites to the central imaging vendor 
and archived for potential future evaluation. The process for image collection and transmission to 
the central imaging vendor can be found in the Study Manual. 
For subjects with gastric MZL, endoscopy with biopsy will be performed to confirm CR.  
9.9. 
Compliance Diary 
A compliance diary will be provided to the subject at Cycle 1 Day 1 to record daily study drug 
administration. A new compliance diary will be provided at each study drug dispensing, 
according to the Schedule of Assessments (Appendix 1). 
9.10. 
Dispensing of ME-401 
Zandelisib (ME-401) is to be taken orally once a day at approximately the same time each day in 
accordance with the IS regimen (or CS dosing regimen if switched from IS after PD). ME-401 
should be taken with a glass of water on an empty stomach (i.e., at least 1 hour prior to food 
intake or 2 hours after food intake). If a subject routinely experiences discomfort with dosing, he 
or she is permitted to take ME-401 with a light, non-fatty snack. On days with PK sampling, 
subjects should be instructed not to take study drug until after the PK sample is collected. 
A compliance diary assessment and returned study drug accountability will be performed at each 
visit which includes study drug dispensing. 


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9.11. 
Managing the Impact of COVID-19 on Study Assessments 
In light of the emerging COVID-19 pandemic, MEI acknowledges that at times it might not be 
feasible for ongoing study patients to visit a site due to local requirements to remain at home 
or existing health problems that increase their risk of infection with the novel coronavirus. 
To allow subjects who are enrolled in the study to continue on treatment if it is deemed in their 
best interest, the following mitigation activities are allowed: 
• If a subject cannot visit a site for investigational product (IP) dispensing, IP may be 
shipped directly to the subject or be dispensed to a family member if this is acceptable 
per institutional procedures and local regulations. If IP is shipped to a subject’s home, 
this should be recorded in the source documents. If the subject will require additional 
compliance diaries, these should be included in the IP shipment. The site should 
follow up with the subject to ensure receipt of IP, enter the date of receipt in the source 
documents, and remind the subject to complete the compliance diary. Subjects should be 
instructed to return all used IP containers and compliance diaries to the site at their next 
study visit. 
• Safety laboratory assessments may be conducted at any accredited laboratory. 
The Investigator should review laboratory results from tests conducted at another lab to 
determine if the subject should continue to receive ME-401. If safety laboratory 
assessments are conducted at a facility that is not listed on the site’s form FDA-1572 
or equivalent document, this should be recorded in the source documents. All laboratory 
results should be entered into the eCRF as soon as possible. Laboratory accreditation 
certificates and normal ranges should be collected whenever feasible for any alternative 
laboratories that are used. 
• If an onsite study visit cannot be conducted as scheduled, site personnel should contact 
the subject by telephone or e-mail to assess his or her current status. Telemedicine visits 
are allowed if acceptable per institutional procedures or local regulations. Any new AEs 
or changes in AEs should be recorded in the source documents and entered into the eCRF 
as soon as possible. Any new concomitant medications or changes to existing 
concomitant medications should be recorded in the source documents and entered into the 
eCRF as soon as feasible. Any procedures that cannot be conducted remotely or at 
another facility, such as vital signs or physical examinations, should be documented in 
the source and will be recorded as protocol deviations. 
• If the subject cannot have imaging for disease assessment conducted at the facility that is 
approved for the study, the imaging may be delayed or conducted at an alternate facility. 
If imaging is conducted at another facility, please obtain the imaging report and inform 
your CRA. If possible, images acquired at an alternate facility should be submitted to the 
central imaging vendor to determine whether the imaging results are of acceptable quality 
and may be used for disease assessment. 
• If it is not feasible to obtain PK samples, please record this in the source documents. 
Any missing PK samples will be considered as minor protocol deviations as PK data are 
not critical for assessing safety or efficacy for this study. 


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If a study visit or procedure is delayed due to COVID-19, this should be recorded in the source 
documents. If an entire study visit is missed due to COVID-19, this should be recorded in the 
source documents. All missed or delayed study visits or procedures will be reported as protocol 
deviations. 
10. 
CORRELATIVE IMMUNE STUDY (US SITES ONLY) 
A correlative immune study will be performed to characterize the effects of ME-401 over time 
on T-cell numbers and function, the pattern of T-cell recovery during the 3-week treatment 
holiday in the IS, and potential correlation between T-cells number and function and the 
development of delayed AEs. Because the assays for these immune studies require fresh blood 
samples, only US sites will be participating in this sub-study. This study is optional for subjects 
enrolled at US sites. Fresh blood samples will be collected and shipped overnight to a central 
laboratory for analysis. Samples may be shipped only Monday to Thursday. Samples may not be 
shipped for delivery on weekends or holidays. 
Samples will be obtained pre-dose on Day 1 of Cycle 1, and then pre-dose on the following 
visits: Day 15 of Cycle 1; Day 1 of Cycles 2, 3, 5, 7, 10; then every 3 cycles thereafter; and at the 
EOT/30-Day Safety Follow-up visit. If possible, a sample will also be obtained when a subject 
develops a late onset Grade 2 AE of diarrhea/colitis, rash/mucositis, transaminase elevation, 
or NIP, or when a subject discontinues study drug permanently for another reason. Samples will 
be collected at the first visit of the AE, at the time when treatment is held, and at the time when 
treatment is resumed.  
Two sets of assays will be performed on these samples: 
• Mass cytometry (CyTOF) assay evaluating approximately 37 separate cell markers to 
measure T-cell count and function  
• Luminex assay to assess changes in the chemokines CCL3, CCL4, IL-6, and TNF 
Blood sample collection and processing will be described in the Study Manual. 
11. 
STATISTICS 
11.1. 
Sample Size 
This is an open-label, single-arm study. The objectives of this study are to evaluate the efficacy 
and safety of ME-401 administered using an IS dosing regimen in patients with relapsed 
or refractory FL or MZL.  
Sample Size for FL  
The study will test the null hypothesis that the IRRC-reviewed ORR is ≤48% against the 
alternative hypothesis that it is ≥65.5%. The sample size for the PEP for FL is estimated to be 
91 subjects, with at least 90% power and 1-sided alpha = 0.025.The selection of a null hypothesis 
of 48% was based on a review of the ORR reported with therapies that have full approval in the 
US for the treatment of relapsed FL (single agent rituximab, single agent bendamustine, 
lenalidomide plus rituximab, and obinutuzumab plus bendamustine) and therapies that have 


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accelerated approval in the US for the treatment of relapsed FL (tazemetostat [Tazverik® US PI], 
duvelisib, umbralisib (Ukoniq™ US PI), idelalisib, copanlisib), focusing on ORR reported in 
patients who have received ≥2 lines of therapy prior to enrollment. The selection of the 
alternative hypothesis of 65.5% was based on the results achieved with single agent zandelisib in 
Study ME-401-002, with an ORR of 70% observed in patients enrolled in the study after failure 
of ≥2 prior therapies (data on record).  
Safety analysis will be performed based on the Safety Population, with analysis focused on the 
IS FL subjects at the primary analysis database lock for FL. With a total sample size of 120 FL 
subjects treated with ME-401 IS dosing regimen, if ≤15 subjects experience Grade ≥3 AESIs 
(i.e., subject incidence rate of ≤12.5% with 95% CI of 7.2–19.8%), the study can rule out the 
possibility of having a incidence rate ≥20% for Grade ≥3 AESIs with the IS dosing regimen. In 
addition, with a total sample size of 120 FL subjects, if ≤5 subjects experience a particular type 
of AE (i.e., subject incidence rate of ≤4.2% with 95% CI 1.4-9.5%), the study can rule out the 
possibility of having an incidence rate ≥10% for the AE. 
Sample Size for MZL 
Initially, the study was designed to test the null hypothesis that the IRRC-reviewed ORR is 
≤46% against the alternative hypothesis that it is ≥66.5%. The sample size for the MZL 
population was estimated to be 64 subjects, with at least 90% power and 1-sided alpha = 0.025.  
The selection of a null hypothesis of 46% was based on a review of the ORR reported with 
therapies that have full or accelerated approval in the US for the treatment of relapsed MZL 
(single agent lenalidomide plus rituximab, ibrutinib, umbralisib), adjusting for the requirement 
that subjects with MZL enrolled in the TIDAL study must have received ≥2 prior lines of therapy 
including an anti-CD20 antibody and chemotherapy. The selection of the alternative hypothesis 
was based on a clinically meaningful increase in the ORR by 20.5%. 
Enrollment in the MZL arm was closed by the Sponsor on 31 August 2022, with 32 of the 
64 planned subjects enrolled. The reason for early closure of enrollment is to focus enrollment of 
subjects with MZL in the global Phase 3 study ME-401-004. Early enrollment closure was 
communicated to all investigators in August 2022 and is being formalized in Amendment 5. 
A sample of 32 subjects will provide preliminary data on the efficacy and safety of single agent 
zandelisib in relapsed/refractory MZL.  
11.2. 
Statistical Analysis 
11.2.1. 
Analysis Populations and Analysis Sets Definitions 
Detail on populations definition will be provided in the Statistical Analysis Plan (SAP). In brief: 
• Safety Population includes all subjects who receive at least one dose of study drug 
• Intent-to-Treat (ITT) FL Population includes all FL subjects randomized per 
Protocol Amendment 1 (two-arm design with randomization), and all enrolled 
FL subjects who received at least one dose of ME-401 per Protocol Amendment 2 or later 
amendment (single-arm, open-label design).  


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• The ITT IS FL Population is defined as all FL subjects who are intended to receive 
ME-401 IS regimen, including subjects who:  
o Were randomized to the ME-401 IS dosing regimen group (Group B) per 
Protocol Amendment 1  
o Were randomized to the ME-401 CS dosing regimen group (Group A) per 
Protocol Amendment 1, but re-consented under Protocol Amendment 2 (for IS dosing 
regimen) prior to or on their Cycle 3 visit date 
o Were enrolled per Protocol Amendment 2 or later amendment who received ME-401 
IS dosing regimen.  
• The ITT CS Group is defined as all FL subjects who are randomized to the ME-401 CS 
regimen group (Group A) per Protocol Amendment 1 and did not switch/plan to switch to 
the IS dosing regimen at Cycle 3 visit per Protocol Amendment 2. More specifically, this 
population includes subjects who were originally randomized to the ME-401 CS dosing 
regimen group (Group A) per Protocol Amendment 1, had their Cycle 3 visits prior to 
when the subjects re-consented to Protocol Amendment 2, or discontinued treatment 
without being re-consented under Protocol Amendment 2.  
• The ITT MZL population is defined as all enrolled subjects with MZL who received at 
least one dose of ME-401 per Protocol Amendment 3. 
• The PEP in FL is defined as the initial 91 consecutive subjects enrolled in the ITT IS 
FL Population.  
• The DOR Primary Analysis Set for FL will include subjects in the FL PEP who achieved 
PR or CR prior to first PD, that is reported before the data cutoff date for the FL primary 
analysis. 
• The DOR Supportive Analysis Set for FL will include subjects in the ITT IS FL who 
achieved PR or CR prior to first PD, that is reported before the data cutoff date for the 
primary analysis of DOR for FL.  
• The DOR Analysis Set for MZL will include subjects in the ITT MZL who achieved 
PR or CR prior to first PD, that is reported before the data cutoff date for the MZL 
primary analysis. 
• Efficacy Evaluable (EE) Population includes all subjects who receive at least one dose 
of study drug and have at least one response assessment post baseline. 
• PK Population includes all subjects who receive at least one dose of study drug and who 
have evaluable PK data. 
11.2.2. 
Statistical Analyses 
The primary analysis of ORR for FL will be triggered after all 91 subjects in the PEP enrolled in 
the study (except those who discontinue early) have been followed for at least 6 months from the 
start of study drug. 
The final analysis of DOR for FL will be triggered after most responders in the Primary Efficacy 
Population have a minimum of 12 months of follow-up from first response. 


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The final analysis of ORR and DOR for MZL will be triggered after most of the responders in 
the MZL ITT population (except those who discontinue early) have been followed for at least 
12 months from first response.  
Summary statistics for continuous variables will include the mean, standard deviation, median, 
and range (minimum/maximum). Categorical variables will be presented as frequency counts and 
percentages. Time-to-event variables will be summarized using Kaplan-Meier (KM) methods 
(median, 95% CI, number of events, number censored, KM figures). Data listings will be created 
to support each table and to present all data.  
The efficacy analysis of ORR for MZL as determined by the IRRC will be based on the 
ITT MZL Population, utilizing the best overall ORR prior to first PD. The CR rate as determined 
by the IRRC will be reported similarly. The ORR as determined by the IRRC in the EE 
Population will be reported as a sensitivity analysis. 
Secondary efficacy endpoints including DOR, DORR, PFS, TTF, time to recapture of response 
(as assessed by the IRRC and/or Investigator); and overall survival (OS) will be summarized 
using KM summary statistics for the ITT IS FL Population and ITT MZL Population 
respectively. 
Similarly, the efficacy endpoints based on Investigator assessments will be summarized as 
supportive analyses. 
Safety analyses will be performed on the Safety Population, as defined in Section 11.2.1.  
Overall summary of safety will include but not limited to the following analyses: 
• TEAEs, including severity and possible relationship to study drug and/or study treatment 
• AESIs 
• treatment-emergent SAEs 
• discontinuations from study treatment due to AEs 
• treatment-emergent abnormal changes in laboratory values 
• treatment-emergent abnormal changes in vital signs. 
The rate of AESIs and the respective 95% CI will be calculated by dosing group (CS and IS) 
and overall.  
Time to AESIs will be calculated from the first day of study treatment to first occurrence of 
AESI, utilizing KM methodology.  
Safety variables including TEAEs, AESIs, laboratory tests, vital signs, and ECGs will be 
summarized descriptively. 
Further safety and efficacy analyses, handling of missing information, and subset analyses will 
be detailed in the study SAP.  
Population PK analyses will be described in the Population PK Analysis Plan. 


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12. 
REGULATORY AND REPORTING REQUIREMENTS 
12.1. 
Compliance with Laws and Regulations 
The study will be conducted according to the principles of the Declaration of Helsinki 
(World Medical Association 2013), the International Council for Harmonisation Guideline E6 
(R2) Good Clinical Practice (ICH 2016), the applicable laws and regulations of the country in 
which the research is conducted, and the requirements of all local regulatory authorities 
regarding the conduct of clinical trials and the protection of human subjects. The study will be 
conducted by scientifically and medically qualified persons and the rights and welfare of the 
subjects will be respected. 
12.2. 
Informed Consent 
The Investigator or an appropriate designee is responsible for obtaining written informed 
consent(s) from each study subject prior to conducting any study-specific procedures. 
The Investigator must use the most current IRB/EC-approved consent form when obtaining 
consent.  
At any time, all signed and dated consent forms must be available for Sponsor/designee 
verification. 
12.3. 
Institutional Review Board/Ethics Committee 
Approval of this study will be obtained from an IRB/EC prior to enrolling subjects and will be 
reviewed and approved on an annual basis by the IRBs/ECs representing the participating 
institutions. Such IRBs/ECs must be appropriately constituted and meet all requirements 
as described in Title 21, Part 56 of the Code of Federal Regulations. The review must include 
the protocol, subject recruitment materials, the Investigator’s Brochure, the ICF, and any other 
study-specific material that will be provided to subjects (including study product compliance 
diaries). A copy of the letter or notice of approval from the IRB/EC must be received by the 
Sponsor prior to shipment of drug supplies to the Investigator. The IRB/EC membership list 
or Federal Wide Assurance (FWA) number must be submitted to the Sponsor with the written 
IRB approval, and lists must be updated, if applicable. 
12.4. 
Public Clinical Trial Registry 
This study will be listed on a public clinical trial registry such as www.clinicaltrials.gov as per 
applicable regulations. 
12.5. 
Confidentiality and Data Protection 
The Principal Investigator and designees, employees, and agents involved with this study will 
comply with relevant local, state, federal and regional laws, as applicable, relating to the 
confidentiality, privacy, and security of subjects’ health information. Data generated during this 
study or disclosed by the Sponsor to the Investigator will only be used as appropriate for the 
execution, analysis, review, and reporting of this study. Such information shall not be used for 
any other purposes and will remain confidential. 


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In order to verify subject eligibility criteria and ensure ongoing subject safety during the study 
and preserve the integrity of study data, notwithstanding source data verification at the study site, 
the Sponsor/CRO may request to review subject source medical data. Should the Sponsor request 
copies of subject medical data, the rationale for such request will be ethically and scientifically 
justified. Such records will promptly be destroyed by the Sponsor/CRO when the purpose of the 
review has been met.  
Though the results of the study may be presented in reports, published in scientific journals, 
or presented at medical meetings, subject names will never be used. 
12.6. 
Financial Disclosure 
Investigators must maintain compliance with the current country-specific guidelines 
and regulations concerning financial disclosure. 
12.7. 
Data Quality Assurance 
Good Clinical Practice (GCP) guidelines regarding clinical data management practices 
and procedures are to be utilized to ensure accurate, consistent, and reliable data. The Sponsor 
or designee will establish a Data Management Plan for this study. 
12.8. 
Insurance 
The Sponsor will obtain liability insurance, which covers health impairments resulting from 
drugs and/or substances/IPs administered in the course of this study for which the subject has 
given his/her written Informed Consent. 
13. 
DATA MANAGEMENT 
13.1. 
Use of Electronic Case Report Forms 
Study data will be stored and transmitted using eCRFs, using a system determined by the 
Sponsor. The Sponsor will provide secure access and eCRF completion guidelines, as well as 
study-specific training, as needed, to each site. 
Data recorded in the eCRFs must be supported by information captured in source documents, 
which must be available at all times for inspection by authorized representatives of the Sponsor, 
the FDA, or other regulatory agencies. The eCRFs must be completed as soon as possible after 
each study visit or contact. 
13.2. 
Study Site Monitoring 
A Sponsor-designated study monitor will conduct a site initiation visit prior to the first subject 
signing the ICF. The study monitor will conduct routine monitoring visits at periodic intervals 
during the course of the study, including a visit shortly after enrollment of the first subject, as per 
the study-specific monitoring plan. Monitoring visits may be conducted onsite or remotely. 
Source data verification may be conducted remotely if allowable per institutional guidelines 
and local regulations. 


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The primary purposes of monitoring visits are to: 
• Ensure the safety and welfare of study subjects 
• Ensure the accuracy and completeness of eCRF entries, as verified against the source 
documentation 
• Verify that the conduct of the study adheres to the written protocol approved by the 
IRB/EC, as well as regulatory requirements 
• Verify that regulatory and other study-specific documentation is maintained and current 
• Perform study drug product accountability (reconcile study product receipt, storage, 
dispensing, and return records) 
13.3. 
Record Retention 
The Investigator will retain the records of the study for two (2) years following the last date 
that a marketing application for ME-401 is approved in any ICH region, or if marketing approval 
is not obtained, for two (2) years after the IND for ME-401 has been closed in the US or until 
there are no pending contemplated marketing applications in an ICH region. These documents 
should be retained for a longer period, however, if required by the applicable regulatory 
requirements or by an agreement with the Sponsor. The Sponsor will notify Investigators when 
study records retention is no longer required.  
For studies conducted outside the US, the Principal Investigator must comply with the 
record -retention requirements set forth in the US FDA IND regulations and the relevant national 
or local health authorities, whichever is longer. 
The site will retain copies of all versions of the protocol, Investigator’s Brochure, 
correspondence with the IRB/EC (including submission and approval letters, approved ICFs), 
curricula vitae and medical licenses of the Investigator and sub-Investigator(s), forms FDA-1572 
or similar forms, correspondence, laboratory documentation (including accreditation documents, 
reference ranges, and manuals), Delegation of Authority Log (documenting procedures delegated 
by the Investigator to be performed by study staff), ME-401 study product records (including 
receipt, storage, dispensing, and return records), source documents (including clinic charts, 
medical records, laboratory results, radiographic reports), training records, screening/enrollment 
logs, monitoring visit logs, and study procedure manuals. 
Should the Investigator leave the institution or otherwise withdraw from the investigation, 
or should there be any changes in the archival arrangements for the study records, the Sponsor 
will be notified. The Sponsor will be notified of the identity of any individual assuming 
responsibility for maintaining the study records and the location of their storage. If no other 
individual at the investigational site is willing to assume this responsibility, the Sponsor will 
assume responsibility for maintaining the study records. 
14. 
AMENDMENTS TO THE PROTOCOL 
Amendments will be originated and documented by the Sponsor. Individual study sites should 
communicate requests for protocol amendments directly to the Sponsor or its designee. The 


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Sponsor may be required to discuss potential protocol amendments with the appropriate 
regulatory agencies. Amendments shall only be implemented following the required regulatory 
and ethical review and approval. Protocol amendments also may require changes to the ICF. 
15. 
PUBLICATION POLICY 
The Sponsor intends to publish the results of this trial as soon as possible following completion 
of data analysis. Data derived from the trial are the exclusive property of the Sponsor. 
Authorship (both inclusion and sequence) will be determined by mutual agreement. In the event 
of a disagreement on authorship, the Sponsor will serve as adjudicator. 


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maintenance therapy versus re-treatment at progression in patients with indolent non-Hodgkin’s 
lymphoma–A randomized phase II trial of the Minnie Pearl Cancer Research Network. 
J Clin Oncol. 2005; 23: 1088–1095. 
Herman SEM, Johnson AJ. Molecular pathways: targeting phosphoinositide 3-kinase p110-delta 
in chronic lymphocytic leukemia. Clin Cancer Res. 2012; 18(15): 4013–4018. 
Howlader N, Noone AM, Krapcho M, et al (eds). SEER Cancer Statistics Review, 1975-2017, 
National Cancer Institute. Bethesda, MD, https://seer.cancer.gov/csr/1975_2017/, based on 
November 2019 SEER data submission, posted to the SEER web site, April 2020. 
ICH Harmonised Guideline, Integrated Addendum to ICH E6(R1): Guideline for Good Clinical 
Practice, E6(R2) 2016. 
Imbruvica® (ibrutinib) tablet, capsule [Prescribing Information], Sunnyvale, CA. 
Pharmacyclics LLC, 2022. 
Laribi K, Tempescul A, Ghnaya H, et al. The bendamustine plus rituximab regimen is active 
against primary nodal marginal zone B-cell lymphoma. Hematol Oncol. 2016; 35(4): 536–541. 
Leonard JP, Trneny M, Izutsu K, et al. AUGMENT: A phase III study of lenalidomide plus 
rituximab versus placebo plus rituximab in relapsed or refractory indolent lymphoma. J Clin 
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in vivo: Kinetics of Treg depletion and alterations in immune functions in vivo and in vitro. 
Int J Cancer. 2007; 120: 2723–2733. 
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antibody therapy for relapsed indolent lymphoma: half of patients respond to a four-dose 
treatment program. J Clin Oncol. 1998; 16(8): 2825–2833. 


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Morschhauser F, Fowler NH, Feugier P, et al. Rituximab plus lenalidomide in advanced 
untreated follicular lymphoma. N Engl J Med. 2018; 379: 934–947. 
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experience. Blood Cancer J. 2020; 10(3): 31. 
Nabhan C, Byrtek M, Taylor MD, et al. Racial differences in presentation and management of 
follicular non-Hodgkin lymphoma in the United States: report from the National LymphoCare 
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National Cancer Institute. Adult non-Hodgkin lymphoma treatment (PDQ®)–Health Professional 
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pdq#_2_toc. Accessed 26 Sept, 2022. National Comprehensive Cancer Network (NCCN) 
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2020. 
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Zydelig® (idelalisib) tablet [Prescribing Information], Foster City, CA: Gilead Sciences, Inc.; 
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Accessed 16 August 2019. 


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APPENDIX 1. SCHEDULE OF ASSESSMENTS 
Assessment/Procedure 
Screenin
g b 
Cycle 
1 
Cycle 
2 
Cycle 
3 
Cycle 
4 
Cycle 
5 
Cycle 
6 
Cycle 
7 
Cycles 10, 
13, 16, 19, 
22, 25 d… 
EOT/30-Day 
Safety 
Follow-up e 
Post-
Treatment 
Long-Term 
Follow-up 
Day(s) of Cycle 
-28 to -1 
1 c 
8 
15 
1 
15 
1 
1 
7 
1 
1 
7 
1 
1 
30 (± 3) days  
post last dose 
or treatment 
discontinuation 
Every  
3 Months 
Study Day(s) 
-28 to -1 
1 
8 
15 
29 
43 
57 
85 
91 
113 
141 
147 
169 
253, 337, 
421, 505, 
589… 
 
 
Visit Window (days) a 
 
 
±3 
±3 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±7 a 
 
 
Informed consent form 
(ICF) 
X 
 
 
 
  
 
 
 
 
 
 
 
 
 
 
 
Medical historyf 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Physical examination 
X 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Symptom-directed exam g 
 
 
X 
X 
 
X 
 
 
 
 
 
 
 
 
 
 
Vital signs h 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Height 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Weight 
X 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
X 
 
ECOG performance status 
X 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Pregnancy test i 
X 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
X 
 
HIV testing j 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Hepatitis B and C testing k 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Adverse events l 
 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Concomitant medication m 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
 
X 
X 
X 
 
ECG n  
X 
X 
 
 
 
 
X 
 
 
 
X 
 
 
X 
X 
 
CBC with differential o 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Serum chemistry p 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Coagulation q 
X 
X 
 
 
X 
 
X 
 
 
X 
 
 
X 
X 
X 
 
PCR test for CMV 
X 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
 
 
Urinalysis (Dipstick) r 
X 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
X 
 
COVID-19 tests 
X 
 


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Assessment/Procedure 
Screenin
g b 
Cycle 
1 
Cycle 
2 
Cycle 
3 
Cycle 
4 
Cycle 
5 
Cycle 
6 
Cycle 
7 
Cycles 10, 
13, 16, 19, 
22, 25 d… 
EOT/30-Day 
Safety 
Follow-up e 
Post-
Treatment 
Long-Term 
Follow-up 
Day(s) of Cycle 
-28 to -1 
1 c 
8 
15 
1 
15 
1 
1 
7 
1 
1 
7 
1 
1 
30 (± 3) days  
post last dose 
or treatment 
discontinuation 
Every  
3 Months 
Study Day(s) 
-28 to -1 
1 
8 
15 
29 
43 
57 
85 
91 
113 
141 
147 
169 
253, 337, 
421, 505, 
589… 
 
 
Visit Window (days) a 
 
 
±3 
±3 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±7 a 
 
 
Issue and assess compliance 
diary t 
 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
 
 
Dispense study drug u 
 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
 
 
PK Sampling v 
 
X 
 
Xw 
X 
 
 
 
X 
 
 
X 
 
 
 
 
Echocardiogram or MUGA x 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
Correlative immune study 
(pre-dose) y 
 
X 
 
X 
X 
 
X 
 
 
X 
 
 
X 
X 
X 
 
Opportunistic infection 
monitoring z 
 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Post-Treatment Follow-up 
procedures aa 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
Response Assessment ,bb,cc 
Screening 
Every 2 Months for the 
First 6 Months 
Every 3 Months for the Next 12 Months 
(through 18 Months) 
Every 6 Months 
Thereafter 
Study Day 
 
61 
121 
182 
274 
365 
456 
547 
730, 912, 1095… 
Visit Window (days) 
 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±14 
CT neck/chest/abdomen/pelvis dd 
X 
X 
X 
X 
X 
X 
X 
X 
X 
FDG-PET scan ee 
X 
 
X 
 
 
X 
 
 
 
Bone marrow biopsy/aspirate ff 
X 
Endoscopy with biopsy gg 
X 


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Abbreviations: ALP = alkaline phosphatase; ALT = alanine aminotransferase; ANC = absolute neutrophil count; aPTT = activated partial thromboplastin time; 
AST = aspartate aminotransferase; BP = blood pressure; BUN = blood urea nitrogen; CBC = complete blood count; CMV = cytomegalovirus; CMR = complete 
metabolic response; CO2 = carbon dioxide; CR = complete response; CT = computed tomography; ECG = electrocardiogram; ECOG = Eastern Cooperative 
Oncology Group; EOT = end of treatment; FDG-PET = 18F-fluorodeoxyglucose positron emission tomography; HcT = hematocrit; HGB = hemoglobin; 
HIV = human immunodeficiency virus; HR = heart rate; ICF = informed consent form; INR = international normalized ratio; LDH = lactate dehydrogenase; 
MCH = mean corpuscular hemoglobin; MUGA = multigated acquisition scan; PCR = polymerase chain reaction; PK = pharmacokinetic; PT = prothrombin time; 
RBC = red blood cell; SOC = standard of care; WBC = white blood cell 
a. Study Assessment Day(s) – There is ±3-day window allowable for each clinic visit for Cycle 1 Day 8 and Cycle 1 Day 15. For Cycles 2 through 25, 
the allowable window is ±7 days. After Cycle 25, the allowable window is ±1 month for each clinic visit and its associated assessments. 
b. Screening – Screening assessments are to be performed within 28 days prior to enrollment. Upon confirmation of meeting eligibility criteria, subjects should 
be treated within a reasonable time frame (within 5 days). If there are >7 days between screening laboratory assessments and expected Cycle 1 Day 1 dosing, 
then the laboratory assessments should be repeated and reviewed prior to the first dose of ME-401. Some tests performed prior to obtaining ICF (e.g., CT scan, 
bone marrow biopsy) may not need to be repeated for the study as long as they were performed within the allowed screening window: refer to Appendix 5. 
c. Cycle 1 Day 1 – Physical examination need not be repeated on Cycle 1 Day 1 if performed within 7 days prior to start of treatment. Laboratory assessments 
(CBC with differential, serum chemistry, coagulation, urinalysis) need not be repeated on Cycle 1 Day 1 if performed within 7 days prior to start of treatment.  
d. Cycle 25+ – Subjects who remain on study after 25 cycles (approximately 2 years) will visit the clinic every 3 months (±1 month), with a response assessment 
performed every 6 months as per standard of care. 
e. EOT/30-Day Safety Follow-up – Visit will be completed 30 (±3) days post last dose of study drug administration (or prior to starting a new treatment if 
urgent treatment is required) or from study drug discontinuation.  
f. If known, record in the medical history information on COVID-19 vaccination administered at any time prior to subject consent to participate in the study. 
g. Symptom-directed exam – Performed on Cycle 1 Day 8, Cycle 1 Day 15, and Cycle 2 Day 15, only as needed/indicated, based on interim history.  
h. Vital signs – BP, HR, temperature. 
i. Pregnancy test – The screening serum pregnancy test in females of childbearing potential must be completed within 28 days prior to Cycle 1 Day 1. Urine or 
serum pregnancy test must be performed on study Day 1. For women of childbearing potential, a monthly serum or urine pregnancy test will be conducted 
every dosing cycle and 30 days after discontinuation of dosing.  
j. HIV testing – HIV antibody is required. 
k. Hepatitis B and C testing – Hepatitis B core antibody, Hepatitis B surface antigen, and hepatitis C antibody are required. Hepatitis B PCR is required if 
hepatitis B core antibody and/or surface antigen is positive. Hepatitis C PCR is required if hepatitis C antibody is positive. 
l. Adverse events – Recorded from the time of the first dose of study drug on Cycle 1 Day 1 and continues until 30 days after the last dose of study drug or start 
of new anti-cancer treatment.  
m. Concomitant medications – Recorded from 28 days prior to Cycle 1 Day 1 until the EOT/30-Day Safety Follow-up visit, or start of new anti-cancer 
treatment. Please, record information about COVID-19 vaccination, if performed. 
n. ECG – 12-lead ECGs performed at screening; Cycle 1 Day 1 pre-dose (unless the Screening visit is within 3 days prior to Day 1); pre-dose on Day 1 of Cycles 
3, 6, 13, 19, and 25, and every 6 months thereafter; at the EOT/30-Day Safety Follow-up visit, and as clinically indicated. ECGs should be performed pre-dose. 
For Cycle 1 Day 1 and Cycle 3 Day 1, ECGs should be taken pre-dose and at expected Cmax (3–5 hours after dosing). 
o. CBC with differential – WBC, ANC, RBC, HGB, HcT, lymphocytes, eosinophils, monocytes, basophils, platelets, and MCH. Screening labs must be 
repeated if performed >7 days prior to Cycle 1 Day 1.  
p. Serum chemistry – Glucose, BUN or urea, creatinine, sodium, potassium, chloride, calcium, ALP, AST, ALT, total bilirubin, total protein, albumin, LDH, 
and phosphorous. Screening labs must be repeated if performed >7 days prior to Cycle 1 Day 1.  
q. Coagulation – aPTT, PT/INR. Screening labs must be repeated if performed >7 days prior to Cycle 1 Day 1.  


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r. Urinalysis – Dipstick or urine test (pH, specific gravity, glucose, ketones, blood, and protein). Screening labs must be repeated if performed >7 days prior to 
Cycle 1 Day 1.  
s. COVID-19 test results – Please record COVID-19 test results in the AE CRF page, if positive.  
t. Issue and Assess Compliance Diary – Compliance diary to record daily study drug administration. Returned diaries are to be assessed for study product 
compliance. New compliance diary is to be issued with each dispensing of ME-401.  
u. Dispense Study Drug – Study drug is to be taken orally once a day on an empty stomach at least 1 hour prior to food intake or 2 hours after food intake at the 
same time each day. On days of PK sampling, instruct subjects to not take study drug until after the pre-dose PK sample is drawn. Compliance diary and count 
of returned capsules are performed on each day of dispensing study drug. 
v. PK sampling – Pre-dose PK samples are to be obtained within 1 hour before study drug administration on Cycle 1 Day 1, Cycle 2 Day 1, Cycle 4 Day 7, and 
Cycle 6 Day 7. Pre-dose PK samples collected after Cycle 1 Day 1 are to be obtained within ±60 minutes of the scheduled time, which is based on the Cycle 1 
Day 1 dosing time.  
w. PK Sampling – Cycle 1 Day 15: pre-dose and 3 hours (±30 min) post intake of study drug. Collect data regarding food and drink intake 4 hours prior to study 
drug intake. 
x. Echocardiogram or MUGA – Performed at screening, EOT/30-Day Safety Follow-up visit, and as clinically indicated. The same methodology 
(echocardiogram or MUGA) should be used throughout the study. 
y. Correlative Sample Study – US Sites only. Samples will be obtained pre-dose on Day 1 of Cycle 1, and then pre-dose on the following visits: Day 15 of 
Cycle 1; Day 1 of Cycles 2, 3, 5, 7, 10; then every 3 cycles thereafter; and at the EOT/30-Day Safety Follow-up visit. Fresh blood samples will be collected 
and shipped overnight to a central laboratory for analysis. Samples may be shipped only Monday to Thursday. Samples may not be shipped for delivery on 
weekends or holidays. If possible, a sample will also be obtained when a subject develops a late onset Grade 2 AE of diarrhea/colitis, rash/mucositis, 
transaminase elevation, or non-infectious pneumonitis, or when a subject discontinues study drug permanently for another reason. Samples will be collected at 
the first visit of the adverse event, at the time when treatment is held, and at the time when treatment is resumed.  
z. Opportunistic Infection Monitoring – Prior to start of every cycle of treatment with ME-401, monitoring will be conducted, and for subjects with identified 
risk factors and those who developed an OI while on study treatment, additional assessments should include: CD4 and CD8 count and ratio, c-reactive protein, 
blood cultures, any additional laboratory and diagnostic methods according to local SOC reported as unscheduled laboratory and diagnostic methods of 
assessment, radiological imaging (i.e., chest X-ray or CT scans). Note: Treatment of the OI that started while on study should be based on local SOC. 
aa. Post-treatment Follow-up Procedures –Every 3 months from the last dose of study drug to obtain data regarding changes in efficacy assessments, death or 
subsequent therapy based on standard of care assessments. Imaging tests to assess disease status will be performed at least once every 6 months or earlier as 
indicated per standard of care. Any AE that has not returned to baseline or Grade ≤2 should be followed until it has returned to baseline or better, the event is 
assessed as stable by the Investigator, the subject is lost to follow up, or the subject withdraws consent. Survival follow-up will continue for up to 3 years after 
the last subject is enrolled in the study.  
bb. Response assessment – Imaging scans, physical examination, bone marrow aspirate/biopsy (if applicable), and laboratory evaluation as appropriate for 
individual subjects.  
cc. Response assessment at the EOT/30-Day Safety Follow-up visit – Perform imaging test (CT scan or PET/CT scan) only if last imaging tests outside the 
window noted for CT scan or PET-CT. 
dd. CT Scan – CT of the neck, chest, abdomen, and pelvis must be obtained at screening (within 6 weeks prior to Cycle 1 Day 1). CT scans must be performed 
using IV contrast. For subjects who are intolerant to contrast agents, the CT scan will be performed with oral contrast or MRI scans will be used. 
ee. PET/CT – FDG-PET scan must be obtained at screening (within 6 weeks prior to Cycle 1 Day 1), as indicated, and to confirm CR/CMR. PET/CT imaging is 
no longer needed once a CMR has been achieved and a CT scan is an appropriate imaging test. 
ff. Bone marrow biopsy/aspirate – Bone marrow biopsy/aspirate is obtained at screening and to confirm CR if PET scans were not performed or were not 
evaluable to assess bone marrow involvement. In addition, it can be performed as clinically indicated at the discretion of the Investigator.  
gg. Endoscopy with biopsy – For subjects with gastric MZL, endoscopy with biopsy to be performed to confirm CR. 


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APPENDIX 2. COCKCROFT-GAULT EQUATION FOR ESTIMATED 
CREATININE CLEARANCE 
Estimate the subject’s creatinine clearance using the serum creatinine value provided by 
the local laboratory, actual body weight, and the appropriate Cockcroft-Gault formula 
(if necessary, convert serum creatinine values from µmol/L to mg/dL by dividing by 88.4; 
for example, 100 µmol/L divided by 88.4 equals 1.131 mg/dL): 
Estimated Creatinine Clearance (CCr) by the Cockcroft-Gault Equation: 
 
𝐶𝐶𝐶𝐶𝐶𝐶= 
(140 −𝑎𝑎𝑎𝑎𝑎𝑎 𝑖𝑖𝑖𝑖 𝑦𝑦𝑦𝑦𝑦𝑦𝑦𝑦𝑦𝑦) × (𝑤𝑤𝑤𝑤𝑤𝑤𝑤𝑤ℎ𝑡𝑡 𝑖𝑖𝑖𝑖 𝑘𝑘𝑘𝑘)
72 × (𝑠𝑠𝑠𝑠𝑠𝑠𝑠𝑠𝑠𝑠 𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐𝑐 𝑖𝑖𝑖𝑖 𝑚𝑚𝑚𝑚/𝑑𝑑𝑑𝑑)
 × (0.85 𝑖𝑖𝑖𝑖 𝑓𝑓𝑓𝑓𝑓𝑓𝑓𝑓𝑓𝑓𝑓𝑓) 
 
Abbreviations: CCr = creatinine clearance (in mL/minute) 
 
 


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APPENDIX 3. SKIN ASSESSMENT 
In the event of cutaneous or mucosal disease, the following assessments are required: 
Date Rash or Mucosal Disease Began 
________________________ 
Date Resolved 
________________________ 
Date of Consultation 
________________________ 
Event date as related to the study Cycle 1 Day 1 
________________________ 
 
RASH SYMPTOMS 
Skin Pain/Tenderness 
Y 
N 
Pruritus 
Y 
N 
Blistering/sloughing/epidermal detachment 
Y 
N 
If yes, please estimate total body surface area (TBSA) of blistered/detached skin   ________%  
 
 
Pustules 
Y 
N 
Facial/ear edema 
 
 
Y 
N 
Other, Please specify _____________________________________________________________ 
Please estimate TBSA of all skin involvement _____________% 
 
MUCOSAL INVOLVEMENT 
Ocular symptoms  
Y 
N 
Oral Symptoms 
Y 
N 
 
 
 
SYSTEMIC INVOLVEMENT 
Elevated AST, ALT, Alk Phos, and/or Tbili 
Y 
N 
Elevated total number of eosinophils: 
≥500/μL     
Y 
N 
 
 
 
 
 
≥1500/μL  
Y 
N 
 
HISTORY 
Please circle Y for yes, N for no, or UNK for Unknown 
 
 
Allergies to medicine 
Y 
N 
UNK 
Specify: _______________________________________________________________ 
 
 
Skin Problems 
Y 
N 
UNK 
Specify: _______________________________________________________________ 
 
 
 
TREATMENT FOR SKIN/MUCOSAL DISEASE 
High dose steroids ≥1 mg/kg prednisone (or equivalent) 
Y 
N 
If yes, dated began: _____________________________ 
 
 
Low dose steroids <1 mg/kg prednisone (or equivalent) 
Y 
N 
If yes, dated began: _____________________________ 
 
 
Topical steroids of any potency 
Y 
N 
Other, please specify _____________________________________________________________ 
 
 
Other, please specify _____________________________________________________________ 
 
 
 
PROCEDURE 
Did the subject undergo skin biopsy 
Y 
N 
Results: _____________________________________________________________________ 
 
 
____________________________________________________________________________ 
 
 
____________________________________________________________________________ 
 
 
Did disease resolve in ≥15 days?   
Y 
N 
Do you think this was a drug reaction?  
Y 
N 
If yes, what do you think is/are the likely culprit drug(s)? ______________________________ 
 
 
____________________________________________________________________________ 
 
 


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APPENDIX 4. GUIDELINES FOR WOMEN OF CHILDBEARING 
POTENTIAL AND FERTILE MALE SUBJECTS 
For females of childbearing potential, defined as all premenopausal women capable of becoming 
pregnant, must agree to use a medically effective contraceptive method (has a failure rate of <1% 
per year) starting with the first dose of study drug through 6 months and 1 week (i.e., 25 weeks) 
after study drug discontinuation.  
Postmenopausal women are defined as no menstrual periods for at least 12 consecutive months 
OR follicle-stimulating hormone (FSH) greater than 40 IU/L on at least two occasions.   
Fertile male subjects, defined as all males physiologically capable of conceiving offspring, must 
agree to use a medically effective contraceptive method starting with the first dose of study drug 
through 3 months and a week (i.e., 13 weeks) after study drug discontinuation.  This requirement 
applies to fertile male subjects who are sexually active with a female partner of child-bearing 
potential.  Male subjects must also refrain from donating sperm during their participation in the 
study until 90 days after completing the study. 
Acceptable Contraception Methods:  
Highly effective contraception is defined as: 
 
True Abstinence 
When this is in line with the preferred and usual lifestyle of the 
subject.  Periodic abstinence (e.g., calendar, ovulation, symptothermal, 
post-ovulation methods) and withdrawal are not acceptable 
methods of contraception. 
Sterilization 
When a woman of childbearing potential has had surgical bilateral 
oophorectomy (with or without hysterectomy) or tubal ligation at 
least six weeks prior to study entry.  In case of oophorectomy alone, 
only when the reproductive status of the woman has been confirmed 
by follow-up hormone level assessment. 
Male Partner Sterilization 
When the appropriate post-vasectomy documentation of the 
absence of sperm in the ejaculate. 
Use of a combination 
of any two of the following 
(one  from a and one from b): 
a) Placement of an intrauterine device (IUD) or intrauterine system 
(IUS) or established use of oral, injected, or implanted hormonal 
methods of contraception 
b) Barrier methods of contraception: Condom or 
Occlusive cap (diaphragm or cervical/vault caps) with 
spermicidal foam/gel/film/cream/vaginal suppository. 
Unacceptable Contraception Methods:  
Unacceptable contraception methods for women of childbearing potential include:  
 
IUD progesterone T  
 
Female condom  
 
Natural family planning (rhythm method) or breastfeeding  
 
Fertility awareness  
 
Withdrawal  
 
Cervical shield 


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APPENDIX 5. LYMPHOMA RESPONSE CRITERIA: MODIFIED 
LUGANO CLASSIFICATION 
SELECTION OF TARGET LESIONS 
Up to six of the largest dominant nodes or tumor masses selected according to all of the 
following: 
1. Clearly measurable in two diameters (longest diameter [LDi] and shortest diameter) 
at baseline 
a. All nodal lesions must measure >1.5 cm in longest diameter regardless of short axis 
measurement 
b. All measurable extranodal lesions should have a longest tumor diameter >1.0 cm 
2. All other lesions (including nodal, extranodal, and assessable disease) should be followed 
as nontarget lesions 
3. If possible, the lesions should be from disparate regions of the body 
4. Should include mediastinal and retroperitoneal areas of disease whenever these sites are 
involved 
SELECTION OF NON-TARGET LESIONS 
Non-target lesions will be qualitatively assessed at each subsequent timepoint.  All of the sites 
of disease present at baseline and not classified as target lesions will be classified as non-target 
lesions, including any measurable lesions that were not chosen as target lesions. 
Examples of non-target lesions include: 
1. All bone lesions, irrespective of the modality used to assess them 
2. Lymphangitis of the skin or lung 
3. Cystic lesions 
4. Irradiated lesions 
5. Measurable lesions beyond the maximum number of 6 
6. Groups of lesions that are small and numerous 
7. Pleural/pericardial effusions and/or ascites 
a. Effusions, ascites, or other fluid collections will be followed as non-target lesions 
b. At each assessment point, radiologists will check for the presence or absence of 
effusions/ascites.  If there is a significant volume increase in the absence of a benign 
etiology, progression can be assessed 
c. Significant new effusions, ascites, or other fluid collections, which are 
radiographically suggestive of malignancy should be recorded as new lesions and 
should be assessed 


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Response should be determined on the basis of radiographic and clinical evidence of disease.  
For subjects who achieve a CR by CT criteria, an FDG-PET will be performed. Assessment 
by PET should follow the criteria described by Cheson 2014 which is summarized in Table 7. 
• Overall response should be determined based upon the Modified Lugano response criteria 
(Table 8). 
Spleen Involvement 
• Splenomegaly defined as enlargement of spleen measured by vertical (cranial or caudal) 
length of >13 cm.  Nodal measurable lesions can be followed as target, non-target, or new 
extra-nodal lesions.  
Liver Involvement 
• CT measurements used for qualitative assessment of liver involvement, and FDG-PET 
used for assessment of response (e.g., new uptake indicative of disease progression).  
Nodal measurable lesions followed as target, non-target, or new extra-nodal lesions.  


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Table 7: 
Revised Criteria for Response Assessment from Cheson et al. 2014 
Response Site 
PET/CT−−Based Response 
CT-Based Response 
Complete Response 
Complete metabolic response 
Complete radiologic response (all of the 
following) 
Lymph nodes and extralymphatic sites 
• 
Score 1, 2, or 3 a with or without a residual 
mass on 5PS b 
• 
It is recognized that in Waldeyer’s ring 
or extranodal sites with high physiologic 
uptake or with activation within spleen or 
marrow (e.g., with chemotherapy or myeloid 
colony stimulating factors), uptake may be 
greater than normal mediastinum and/or liver.  
In this circumstance, complete metabolic 
response may be inferred if uptake at sites of 
initial involvement is no greater than 
surrounding normal tissue even if the tissue 
has high physiologic uptake. 
• 
Target nodes/nodal masses must regress to 
≤1.5 cm in LDi 
• 
No extralymphatic sites of disease 
Nonmeasured lesions 
• 
Not applicable 
• 
Absent 
Organ enlargement 
• 
Not applicable 
• 
Regress to normal 
New lesions 
• 
None 
• 
None 
Bone marrow 
• 
No evidence of FDG-avid disease in marrow 
• 
Normal by morphology; if indeterminate, 
IHC-negative 
Partial Response 
Partial metabolic response 
Partial remission (all of the following):  
Lymph nodes and extralymphatic sites 
• 
Score 4 or 5b with reduced uptake compared 
with baseline and residual mass(es) of any size. 
• 
At interim, these findings suggest responding 
disease. 
• 
At end of treatment, these findings indicate 
residual disease. 
• 
≥50% decrease in SPD of up to 6 target 
measurable nodes and extranodal sites 
• 
When a lesion is too small to measure on CT, 
assign 5 mm × 5 mm as the default value 
• 
When no longer visible, 0 × 0 mm 
• 
For a node >5 mm × 5 mm, but smaller than 
normal; use actual measurement for calculation 
Nonmeasured lesions 
• 
Not applicable 
• 
Absent/normal, regressed, but no increase 
Organ enlargement 
• 
Not applicable 
• 
Spleen must have regressed by >50% in length 
beyond normal 
New lesions 
• 
None 
• 
None 


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Table 7: 
Revised Criteria for Response Assessment from Cheson et al. 2014 (Continued) 
Response Site 
PET/CT−−Based Response 
CT-Based Response 
Bone marrow 
• 
Residual uptake higher than uptake in normal 
marrow but reduced compared with baseline 
(diffuse uptake compatible with reactive changes 
from chemotherapy allowed).  If there are 
persistent focal changes in the marrow in the 
context of a nodal response, consideration should 
be given to further evaluation with MRI or biopsy 
or an interval scan. 
• 
Not applicable 
No Response or Stable Disease 
No metabolic response 
Stable disease 
Target nodes/nodal masses, extranodal lesions 
• 
Score 4 or 5 with no significant change in FDG 
uptake from baseline at interim or end of 
treatment 
• 
<50% decrease from baseline in SPD of up to 
6 dominant, measurable nodes and extranodal 
sites; no criteria for progressive disease are 
met 
Nonmeasured lesions 
• 
Not applicable 
• 
No increase consistent with progression 
Organ enlargement 
• 
Not applicable 
• 
No increase consistent with progression 
New lesions 
• 
None 
• 
None 
Bone marrow 
• 
No change from baseline 
• 
Not applicable 
Progressive Disease 
Progressive metabolic disease 
Progressive disease requires at least one of the 
following  
 Individual target nodes/nodal masses 
• 
Score 4 or 5 with an increase in intensity of 
uptake from baseline and/or 
• 
PPD progression: 
 Extranodal lesions 
• 
New FDG-avid foci consistent with lymphoma at 
interim or end-of-treatment assessment 
• 
An individual node/lesion must be abnormal 
with 
• 
LDi >1.5 cm and 
• 
Increase by ≥50% from PPD nadir and 
• 
An increase in LDi or SDi from nadir 
o 
0.5 cm for lesions ≤2 cm 
o 
1.0 cm for lesions >2 cm 
• 
In the setting of splenomegaly, the splenic 
length must increase by >50% of the extent of 
its prior increase beyond baseline 
(e.g., a 15 cm spleen must increase to >16 cm).  
If no prior splenomegaly, must increase by at 
least 2 cm from baseline.  New or recurrent 
splenomegaly 


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Table 7: 
Revised Criteria for Response Assessment from Cheson et al. 2014 (Continued) 
Response Site 
PET/CT−−Based Response 
CT-Based Response 
 Nonmeasured lesions 
• 
None 
• 
New or clear progression of pre-existing 
nonmeasured lesions 
 New Lesions 
• 
New FDG-avid foci consistent with lymphoma 
rather than another etiology (e.g., infection, 
inflammation).  If uncertain regarding etiology of 
new lesions, biopsy or interval scan may be 
considered. 
• 
Regrowth of previously resolved lesions 
• 
A new node >1.5 cm in any axis 
• 
A new extranodal site >1.0 cm in any axis; if 
<1.0 cm in any axis, its presence must be 
unequivocal and must be attributable to 
lymphoma 
• 
Assessable disease of any size unequivocally 
attributable to lymphoma 
 Bone Marrow 
• 
New or recurrent FDG-avid foci 
• 
New or recurrent involvement 
Abbreviations: 5PS = 5-point scale; CT = computed tomography; FDG = 18F-fluorodeoxyglucose; GI = gastrointestinal; IHC = immunohistochemistry; 
LDi = longest transverse diameter of a lesion; MRI = magnetic resonance imaging; PET = positron emission tomography; PPD = cross product of the LDi and 
perpendicular diameter; SDi = shortest axis perpendicular to the LDi; SPD = sum of the product of the perpendicular diameters for multiple lesions. 
a. Measured dominant lesions: up to six of the largest dominant nodes, nodal masses, and extranodal lesions selected to be clearly measurable in two diameters.  
Nodes should preferably be from disparate regions of the body and should include, where applicable, mediastinal and retroperitoneal areas. Non-nodal lesions 
include those in solid organs (e.g., liver, spleen, kidneys, lungs), GI involvement, cutaneous lesions, or those noted on palpation. Nonmeasured lesions: any 
disease not selected as measured, dominant disease and truly assessable disease should be considered not measured. These sites include any nodes, nodal 
masses, and extranodal sites not selected as dominant or measurable or that do not meet the requirements for measurability but are still considered abnormal, as 
well as truly assessable disease, which is any site of suspected disease that would be difficult to follow quantitatively with measurement, including pleural 
effusions, ascites, bone lesions, leptomeningeal disease, abdominal masses, and other lesions that cannot be confirmed and followed by imaging. In Waldeyer’s 
ring or in extranodal sites (e.g., GI tract, liver, bone marrow), FDG uptake may be greater than in the mediastinum with complete metabolic response, but 
should be no higher than surrounding normal physiologic uptake (e.g., with marrow activation as a result of chemotherapy or myeloid growth factors). 
b. PET 5PS: 1, no uptake above background; 2, uptake ≤ mediastinum; 3, uptake > mediastinum but ≤ liver; 4, uptake moderately > liver; 5, uptake markedly 
higher than liver and/or new lesions; X, new areas of uptake unlikely to be related to lymphoma. 
 


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Table 8: 
Modified Lugano Response Criteria 
Morphologic 
Response 
Metabolic 
Response 
Bone 
Marrow 
Biopsy 
Prior Combined Response 
Lugano Combined Response 
NE/No CT 
CMR 
 
 
CR 
PMR 
 
 
SD unless the previous CT scan 
indicated CR or PR, then 
combined response is PR* 
NMR 
 
 
SD 
PMD 
 
 
PD 
NE/No PET 
 
 
NE 
PD 
CMR 
 
 
PD 
PMR 
 
 
PD 
NMR 
 
 
PD 
PMD 
 
 
PD 
NE/No PET 
 
 
PD 
SD 
CMR 
 
 
CR 
PMR 
 
 
SD* 
NMR 
 
 
SD 
PMD 
 
 
PD 
NE/No PET*** 
 
 
SD 
PR 
CMR 
 
 
CR 
PMR 
 
 
PR 
NMR 
 
 
PR* 
PMD 
 
 
PD 
NE/No PET*** 
 
 
PR 
CR 
CMR 
 
 
CR 
PMR 
 
 
PR 
NMR 
 
 
PR 
PMD 
 
 
PD 
NE/No PET 
 
No prior PET response 
PR 
NE/No PET 
 
No prior response and prior 
PET was negative 
CR 
NE/No PET 
 
SD 
PR 
NE/No PET 
 
PR 
PR 
NE/No PET 
 
CR 
CR 
NE/No PET 
 
PD and prior evaluable PET 
was not PMD 
PR 
NE/No PET*** 
 
PD and prior PET was PMD 
NE 
NE/No PET 
CR** 
 
CR 
Abbreviations:  BMB = bone marrow biopsy; CMR = complete metabolic response; CR = complete response; 
CT = computed tomography; NE = not evaluated; NMR = no metabolic response; PD = progressive disease; 
PET = positron emission tomography; PMD = progressive metabolic disease; PMR = partial metabolic response; 
PR = partial response; SD = stable disease 
    * Lugano combine response criteria for this response combination has been modified per FDA request. 
  ** When a PET scan is not acquired or not evaluable, a bone marrow biopsy (BMB) is required to confirm a CR 
by CT. If no BMB is taken (and no PET can be acquired for this time point), the best possible overall response 
is a PR unless the prior overall response meets either criteria: prior overall response was CR or there was no 
prior overall response and the baseline/prior PET was negative. 
*** When a timepoint does not have an evaluable PET scan, the morphologic response is not PD, and the most 
recent evaluable PET scan indicated a metabolic response of PMD, then the combined response will be NE. 
An improved combined response without evidence that the findings in the prior PET scan have resolved isn’t 
justified. However, there is not PET-based evidence that the subject has continuing PD, so the response is 
designated as NE. 


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APPENDIX 6. ECOG PERFORMANCE STATUS CRITERIA 
Score 
Description 
0 
Fully active, able to carry on all pre-disease performance without restriction. 
1 
Restricted in physically strenuous activity but ambulatory and able to carry out work of a light 
or sedentary nature (e.g., light housework, office work). 
2 
Ambulatory and capable of all self-care but unable to carry out any work activities.  Up and about more 
than 50% of waking hours. 
3 
Capable of only limited self- care, confined to bed or chair more than 50% of waking hours. 
4 
100% bedridden.  Completely disabled.  Cannot carry on any self-care.  Totally confined to bed 
or chair. 
5 
Dead. 
References:  Oken 1982. 
 


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APPENDIX 7. LIST OF CYP2C8 INHIBITORS AND INDUCERS 
Following is a table of known inducers and inhibitors of CYP2C8.  
Inhibitors of CYP2C8 can be classified by their potency, such as: 
• Strong inhibitor being one that causes at least a 5-fold increase in the plasma AUC 
values, or more than 80% decrease in clearance. 
• Moderate inhibitor being one that causes at least a 2-fold increase in the plasma AUC 
values, or 50–80% decrease in clearance.  
• Weak inhibitor being one that causes at least a 1.25-fold but less than 2-fold increase 
in the plasma AUC values, or 20–50% decrease in clearance.  
Inhibitors 
Inducers 
Strong inhibitor 
gemfibrozil 
 
Moderate inhibitor 
trimethoprim 
glitazones 
montelukast 
quercetin 
Unspecified potency 
rifampin 
Note:  Medicines on this list must be reviewed by Principal Investigators on an ongoing basis to 
assure updates.  
Please note the following:  This is not an exhaustive list.  For an updated list, see the following 
links: 
• http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/DrugIn
teractionsLabeling/ucm093664.htm#4 
• http://medicine.iupui.edu/clinpharm/ddis/main-table/ 
 


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APPENDIX 8. LIST OF CYP3A INHIBITORS AND INDUCERS 
Strong CYP3A Inhibitors 
Antibiotics: clarithromycin, telithromycin, troleandomycin 
Antifungals: itraconazole, ketoconazole, posaconazole, voriconazole 
Antivirals: boceprevir, telaprevir 
Other: cobicistat, conivaptan, elvitegravir, mibefradil, nefazodone 
Protease inhibitors: indinavir, lopinavir, nelfinavir, ritonavir, saquinavir, tipranavir 
Moderate CYP3A Inhibitors 
CYP3A4, CYP3A5, CYP3A7 
Antibiotics: ciprofloxacin, erythromycin 
Antifungals: fluconazole, clotrimazole 
Protease inhibitors: amprenavir, atazanavir, darunavir/ritonavir, fosamprenavir 
Calcium channel blockers: diltiazem, verapamil 
Tyrosine kinase inhibitors (anticancer): 
imatinib, crizotinib 
Food products: grapefruit juice (citrus paradisi juice) 
Herbal medications: Schisandra sphenanthera 
Others: amiodarone, aprepitant, casopitant, cimetidine, cyclosporine, dronedarone, tofisopam 
Strong/Moderate CYP3A 
Inducers* 
Avasimibe, bosentan, carbamazepine, efavirenz, enzalutamide, etravirine, phenytoin, rifampin 
(rifampicin), St. John’s wort (hypericum perforatum), mitotane modafinil, phenobarbital, 
rifabutin 
Abbreviation: CYP = cytochrome P450. 
Note: The list of drugs in this table is not exhaustive. Please refer to the prescribing information of concomitant 
medication to check for CYP3A inhibition or induction risks or contact the medical monitor of the protocol. 
Source: Food and Drug Administration Drug Development and Drug Interactions: Table of Substrates, Drug 
Development and Drug Interactions and Inducers. Flockhart DA. Drug Interactions: Cytochrome P450 Drug 
Interaction Table. Indiana University School of Medicine. 
http://medicine.iupui.edu/flockhart/table.htm. 
* Strong inducers of CYP3A are shown in bold. 
 
 


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APPENDIX 9. LIST OF DRUGS KNOWN TO PROLONG QT/QTC 
INTERVAL 
The table below includes a list of drugs known to prolong QT/QTc interval from 
https://www.cpsbc.ca/files/pdf/Methadone-Drugs-QT-Interval-Prolongation.pdf. 
Note:  Medicines on this list must be reviewed by Principal Investigators on an ongoing basis to 
assure updates.  
Generic Name 
Brand Names (Partial List) 
Alfuzosin 
Uroxatral® 
Amantadine 
Symmetrel®, Symadine® 
Amiodarone 
Cordarone®, Pacerone®, Nexterone® 
Amisulpride (Only on Non US Market) 
Solian®, Supitac®, Soltus®, Amitrex®, Amazeo® 
Amitriptyline 
Elavil® (Discontinued 6/13), Tryptomer®, Tryptizol®, 
Laroxyl®, Saroten®, Sarotex® Lentizol®, Endep® 
Anagrelide 
Agrylin®, Xagrid® 
Apomorphine 
Apokyn®, Ixense®, Spontane®, Uprima® 
Aripiprazole 
Abilify®, Aripiprex® 
Arsenic trioxide 
Trisenox® 
Artenimol+piperaquine 
Eurartesim® 
Asenapine 
Saphris®, Sycrest® 
Astemizole (Removed from Market) 
Hismanal® 
Atazanavir 
Reyataz® 
Atomoxetine 
Strattera® 
Azithromycin 
Zithromax®, Zmax® 
Bedaquiline 
Sirturo® 
Bepridil (Removed from Market) 
Vascor® 
Bortezomib 
Velcade®, Bortecad® 
Bosutinib 
Bosulif® 
Ceritinib 
Zykadia® 
Chloral hydrate 
Aquachloral®, Novo-Chlorhydrate®, Somnos®, Noctec®, 
Somnote® 
Chloroquine 
Aralen® 
Chlorpromazine 
Thorazine®, Largactil®, Megaphen® 
Cilostazol 
Pletal® 
Ciprofloxacin 
Cipro®, Cipro-XR®, Neofloxin® 
Cisapride (Removed from Market) 
Propulsid® 
Citalopram 
Celexa®, Cipramil® 
Clarithromycin 
Biaxin®, Prevpac® 
Clomipramine 
Anafranil® 
Clozapine 
Clozaril®, Fazaclo®, Versacloz® 
Cocaine 
Cocaine 


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Generic Name 
Brand Names (Partial List) 
Crizotinib 
Xalkori® 
Cyamemazine (cyamepromazine) (Only on 
Non-US Market) 
Tercian® 
Dabrafenib 
Tafinlar® 
Dasatinib 
Sprycel® 
Degarelix 
Firmagon® 
Delamanid (Only on Non-US Market) 
Deltyba® 
Desipramine 
Pertofrane®, Norpramine® 
Dexmedetomidine 
Precedex®, Dexdor®, Dexdomitor® 
Diphenhydramine 
Benadryl®, Nytol®, Unisom®, Sominex®, Dimedrol®, 
Daedalon® 
Disopyramide 
Norpace® 
Dofetilide 
Tikosyn® 
Dolasetron 
Anzemet® 
Domperidone (Only on Non-US Market) 
Motilium®, Motillium®, Motinorm Costi®, Nomit® 
Donepezil 
Aricept® 
Doxepin 
Sinequan®, Silenor®, Aponal®, Adapine®, Doxal®, Deptran®, 
Sinquan® 
Dronedarone 
Multaq® 
Droperidol 
Inapsine®, Droleptan®, Dridol®, Xomolix® 
Eribulin mesylate 
Halaven® 
Erythromycin 
E.E.S.®, Robimycin®, EMycin®, Erymax®, Ery-Tab®, Eryc 
Ranbaxy®, Erypar®, Eryped®, Erythrocin Stearate Filmtab®, 
Erythrocot®, E-Base®, Erythroped®, Ilosone®, MY-E®, 
Pediamycin®, Zineryt®, Abboticin®, Abboticin-ES®, Erycin®, 
PCE Dispertab®, Stiemycine®, Acnasol®, Tiloryth® 
Escitalopram 
Cipralex®, Lexapro®, Nexito®, Anxiset-E® (India), Exodus® 
(Brazil), Esto® (Israel), Seroplex®, Elicea®, Lexamil®, Lexam®, 
Entact® (Greece), Losita® (Bangladesh), Reposil® (Chile), 
Animaxen® (Colombia), Esitalo® (Australia), Lexamil® (South 
Africa) 
Famotidine 
Pepcid®, Fluxid®, Quamatel® 
Felbamate 
Felbatol® 
Fingolimod 
Gilenya® 
Flecainide 
Tambocor®, Almarytm®, Apocard®, Ecrinal®, Flécaine® 
Fluconazole 
Diflucan®, Trican® 
Fluoxetine 
Prozac®, Sarafem®, Fontex® 
Foscarnet 
Foscavir® 
Furosemide (frusemide) 
Lasix®, Fusid®, Frumex® 
Galantamine 
Reminyl®, Nivalin®, Razadyne-ER®, 
Gatifloxacin (Removed from Market) 
Tequin® 
Gemifloxacin 
Factive® 


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Generic Name 
Brand Names (Partial List) 
Granisetron 
Kytril®, Sancuso®, Granisol® 
Grepafloxacin 
Raxar® 
Halofantrine 
Halfan® 
Haloperidol 
Haldol® (US & UK), Aloperidin®, Bioperidolo®, Brotopon®, 
Dozic®, Duraperidol® (Germany), Einalon S®, Eukystol®, 
Halosten®, Keselan®, Linton®, Peluces®, Serenace®, Serenase®, 
Sigaperidol® 
Hydrochlorothiazide 
Apo-Hydro®, Aquazide H®, BP Zide®, Dichlotride®, 
Hydrodiuril®, HydroSaluric®, Hydrochlorot®, Microzide®, 
Esidrex®, Oretic® 
Hydrocodone - ER 
Hysingla™ ER, Zohydro ER 
Hydroxychloroquine 
Plaquenil®, Quineprox® 
Hydroxyzine 
Atarax®, Vistaril®, Aterax®, Alamon®, Durrax®, Equipose®, 
Masmoran®, Orgatrax®, Paxistil® Quiess®, Tran-Q®, 
Tranquizine® 
Ibutilide 
Corvert® 
Iloperidone 
Fanapt®, Fanapta®, Zomaril® 
Imipramine (melipramine) 
Tofranil® 
Indapamide 
Lozol®, Natrilix®, Insig® 
Isradipine 
Dynacirc® 
Itraconazole 
Sporanox®, Onmel® 
Ivabradine 
Procoralan®, Coralan®, Corlentor®, Coraxan®, Ivabid®, Bradia® 
Ketoconazole 
Nizoral®, Sebizole®, Ketomed®, Keton® 
Lapatinib 
Tykerb®, Tyverb® 
Lenvatinib 
Lenvima® 
Leuprolide 
Lupron®, Eligard®, Viadur®, Carcinil®, Enanton®, Leuplin®, 
Lucrin®, Procren®, Prostap® and others 
Levofloxacin 
Levaquin®, Tavanic® 
Levomepromazine (Only on Non-US Market) 
Nosinan®, Nozinan®, Levoprome® 
Levomethadyl (Removed from Market) 
Orlaam® 
Lithium 
Eskalith®, Lithobid® 
Mesoridazine (Removed from Market) 
Serentil® 
Methadone 
Dolophine®, Symoron®, Amidone®, Methadose®, Physeptone®, 
Heptadon® 
Metoclopramide 
Reglan®, Afipran®, Maxolon®, Cerucal®, Clopamon®, Clopra®, 
Maxeran®, Maxolon®, Metozolv®, Plasil®, Pramin®, 
Primperan®, Perinorm® 
Metronidazole 
Flagyl® and many others 
Mifepristone 
Korlym®, Mifeprex® 
Mirabegron 
Myrbetriq® 
Mirtazapine 
Remeron 
Moexipril/HCTZ 
Uniretic®, Univasc® 


MEI Pharma, Inc. 
Protocol: ME-401-003
Version: Amendment 5
 
Version date:18 October2022 
Confidential 
Page 98 of 99
 
Generic Name 
Brand Names (Partial List) 
Moxifloxacin 
Avelox®, Avalox®, Avelon® 
Nelfinavir 
Viracept® 
Nicardipine 
Cardene® 
Nilotinib 
Tasigna® 
Norfloxacin 
Noroxin®, Ambigram® 
Nortriptyline 
Pamelor®, Sensoval®, Aventyl®, Norpress®, Allegron®, 
Noritren®, Nortrilen® 
Ofloxacin 
Floxin® 
Olanzapine 
Zyprexa®, Zydis®, Relprevv® 
Ondansetron 
Zofran®, Anset®, Ondemet®, Zuplenz®, Emetron®, Ondavell®, 
Emeset®, Ondisolv®, Setronax® 
Osimertinib 
Tagrisso® 
Oxaliplatin 
Eloxatin® 
Oxytocin 
Pitocin®, Syntocinon® 
Paliperidone 
Invega®, Xepilon® 
Panobinostat 
Farydak® 
Pantoprazole 
Protonix® and others 
Papaverine HCl 
none 
Paroxetine 
Paxil®, Aropax®, Pexeva®, Seroxat®, Sereupin® 
Pasireotide 
Signifor® 
Pazopanib 
Votrient® 
Pentamidine 
Pentam® 
Perflutren lipid microspheres 
Definity® 
Pimozide 
Orap® 
Pipamperone (Only on Non-US Market) 
Dipiperon (E.U), Propitan (Japan) 
Posaconazole 
Noxafil®, Posamol® 
Probucol (Removed from Market) 
Lorelco® 
Procainamide 
Pronestyl®, Procan® 
Promethazine 
Phenergan® 
Propofol 
Diprivan®, Propoven® 
Quetiapine 
Seroquel® 
Quinidine 
Quinaglute®, Duraquin®, Quinact®, Quinidex®, Cin-Quin®, 
Quinora® 
Quinine sulfate 
Qualaquin® 
Ranolazine 
Ranexa®, Ranozex® 
Rilpivirine 
Edurant®, Complera®, Eviplera® 
Risperidone 
Risperdal® 
Ritonavir 
Norvir® 


MEI Pharma, Inc. 
Protocol: ME-401-003
Version: Amendment 5
 
Version date:18 October2022 
Confidential 
Page 99 of 99
 
Generic Name 
Brand Names (Partial List) 
Roxithromycin (Only on Non-US Market) 
Rulide®, Xthrocin®, Roxl-150®, Roxo®, Surlid®, Rulide®, 
Biaxsig®, Roxar®, Roximycinv®, Roxomycin®, Rulid®, 
Tirabicin®, Coroxin® 
Saquinavir 
Invirase®(combo) 
Sertindole (Only on Non-US Market) 
Serdolect®, Serlect® 
Sertraline 
Zoloft®, Lustral®, Daxid®, Altruline®, Besitran®, Deprax®, 
Elrval®, Emergen®, Gladem®, Implicane®, Sedoran®, Sealdin®, 
SerivoLowfin®, Stimuloton®, Tresleen®, Sertralin Bluefish® 
Sevoflurane 
Ulane®, Sojourn® 
Solifenacin 
VESIcare® 
Sorafenib 
Nexavar® 
Sotalol 
Betapace®, Sotalex®, Sotacor® 
Sparfloxacin (Removed from Market) 
Zagam® 
Sulpiride (Only on Non-US Market) 
Dogmatil®, Dolmatil®, Eglonyl®, Espiride®, Modal®, Sulpor® 
Sunitinib 
Sutent® 
Tacrolimus 
Prograf®, Prograf®, Advagraf®, Protopic® 
Tamoxifen 
Nolvadex®(discontinued 6/13), Istubal®, Valodex® 
Telaprevir 
Incivo® 
Telavancin 
Vibativ® 
Telithromycin 
Ketek® 
Terfenadine (Removed from Market) 
Seldane® 
Tetrabenazine 
Nitoman®, Xenazine® 
Thioridazine 
Mellaril®, Novoridazine®, Thioril® 
Tizanidine 
Zanaflex®, Sirdalud® 
Tolterodine 
Detrol®, Detrusitol® 
Toremifene 
Fareston® 
Torsemide 
Demadex®, Diuver®, Examide® 
Trazodone 
Desyrel® (discontinued 6/13), Oleptro®, Beneficat®, Deprax®, 
Desirel®, Molipaxin®, Thombran®, Trazorel®, Trialodine®, 
Trittico®, Mesyrel® 
Trimipramine 
Surmontil®, Rhotrimine®, Stangyl® 
Tropisetron (Only on Non-US Market) 
Navoban®, Setrovel® 
Vandetanib 
Caprelsa® 
Vardenafil 
Levitra® 
Vemurafenib 
Zelboraf® 
Venlafaxine 
Effexor®, Efexor® 
Voriconazole 
VFend® 
Vorinostat 
Zolinza® 
Ziprasidone 
Geodon®, Zeldox® 
 

SAP_001.pdf: 
 
 
STATISTICAL ANALYSIS PLAN FOR INTERVENTIONAL STUDIES 
 
A Multicenter, Open-Label, Single-Arm, Phase 2 Study of 
Zandelisib (ME-401) in Subjects with Follicular Lymphoma or 
Marginal Zone Lymphoma After Failure of Two or More Prior 
Systemic Therapies – The TIDAL Study 
 
SAP Version Date: 21 June 2021 
Protocol: ME-401-003 
Protocol Version and Date: Amendment 4, 08 Jun 2021 
 
Syneos Health Project Code: 1011094 
 
Author: Fenfen Wang, Senior Biostatistician 
 
NCT03768505 
 
 
Notice of Confidential and Proprietary Information: 
The information contained in this document is confidential belonging to MEI Pharma, 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 MEI Pharma, 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. 


MEI Pharma, Inc. 
Statistical Analysis Plan
Protocol ME-401-003 
Page 2 of 49
 
 
CONFIDENTIAL 
 
 
REVISION HISTORY 
Version # 
Date 
(DD-MMM-YYYY) 
Document 
Owner 
Revision Summary 
0.1 
11-MAR-2019 
Gargi Patel 
Initial Release Version 
0.2 
05-APR-2019 
Gargi Patel 
Added revisions based on sponsor comments on 
v0.1 / 29MAR 
1.0 
18-APR-2019 
Gargi Patel 
Added revisions based on sponsor comments on 
v0.2 / 05APR 
1.1 
12-Jun-2020 
Fenfen Wang 
Full version of SAP, update per protocol 
amendment 2 
1.2 
15-Jan-2021 
Fenfen Wang 
Updated full version of SAP, based on protocol 
amendment 3 and sponsor’s comments 
2.0 
12-Feb-2021 
Fenfen Wang 
Final full version of SAP, based on sponsor’s 
comments on v1.2 
3.0 
21-Jun-2021 
Joanne Li 
Updated per Protocol Amendment 4 
 


MEI Pharma, Inc. 
Statistical Analysis Plan
Protocol ME-401-003 
Page 3 of 49
 
 
CONFIDENTIAL 
 
 
I confirm that I have reviewed this document and agree with the content. 
 
APPROVALS 
Syneos Health Approval 
Nimra Tariq, Senior Biostatistician 
 
 
 
 
Name, Title 
Lead Biostatistician 
 
Signature 
 
Date 
Mike Ou, Senior Director, Biostatistics 
 
 
 
 
Name, Title 
 
Signature 
 
Date 
MEI Pharma, Inc. Approval 
Joanne Li, Ph.D., Ex. Director, Biometrics 
 
 
 
 
Name, Title 
Sponsor Contact 
 
Signature 
 
Date 
Richard Ghalie, M.D., Chief Medical Officer  
 
 
 
Name, Title 
Sponsor Contact 
 
Signature 
 
Date 
 


MEI Pharma, Inc. 
Statistical Analysis Plan
Protocol ME-401-003 
Page 4 of 49
 
 
CONFIDENTIAL 
 
 
TABLE OF CONTENTS 
REVISION HISTORY.....................................................................................................................2 
APPROVALS ..................................................................................................................................3 
TABLE OF CONTENTS .................................................................................................................4 
LIST OF ABBREVIATIONS ..........................................................................................................8 
1. 
PURPOSE ...................................................................................................................11 
1.1. 
Responsibilities ...........................................................................................................11 
1.2. 
Timings of Analyses ...................................................................................................11 
2. 
STUDY OBJECTIVES ..............................................................................................11 
2.1. 
Primary Objective .......................................................................................................11 
2.2. 
Secondary Objectives .................................................................................................12 
2.3. 
Brief Description ........................................................................................................12 
2.4. 
Determination of Sample Size ....................................................................................15 
2.5. 
Treatment Assignment ................................................................................................15 
2.6. 
Study Procedures and Flowchart ................................................................................16 
3. 
ENDPOINTS ..............................................................................................................22 
3.1. 
Primary Efficacy Endpoint .........................................................................................22 
3.2. 
Secondary Efficacy Endpoints ....................................................................................22 
3.2.1. 
Objective Response Rate (ORR) by Investigator .......................................................22 
3.2.2. 
Duration of Response (DOR) and Duration of Recaptured Response 
(DORR) .......................................................................................................................22 
3.2.3. 
Complete Response (CR) Rate ...................................................................................25 
3.2.4. 
Progression-Free Survival (PFS) ................................................................................25 
3.2.5. 
Time to Treatment Failure (TTF) ...............................................................................25 
3.2.6. 
Recapture of Response ...............................................................................................25 
3.2.7. 
Overall Survival (OS) .................................................................................................26 
3.3. 
Safety Endpoints .........................................................................................................26 
3.3.1. 
Overall Incidence Rate of Treatment-Emergent Adverse Events (TEAEs) ...............26 
3.3.2. 
Incidence Rate of Adverse Event of Special Interests (AESIs) ..................................26 
3.3.3. 
Time to Occurrence of Adverse Event of Special Interests (AESIs) ..........................26 
3.3.4. 
Other Safety Endpoints ...............................................................................................26 
4. 
ANALYSIS POPULATIONS AND ANALYSIS SETS ...........................................26 
4.1. 
Safety Population ........................................................................................................26 


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Statistical Analysis Plan
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4.2. 
Intent-to-Treat (ITT) Population .................................................................................27 
4.3. 
Efficacy Analysis Populations and Analysis Sets ......................................................28 
4.4. 
Pharmacokinetic (PK) Population ..............................................................................28 
4.5. 
Per Protocol (PP) Population ......................................................................................28 
4.6. 
Protocol Deviations ....................................................................................................28 
5. 
GENERAL ASPECTS FOR STATISTICAL ANALYSIS ........................................29 
5.1. 
General Methods .........................................................................................................29 
5.2. 
Key Definitions ...........................................................................................................31 
5.2.1. 
Baseline .......................................................................................................................31 
5.2.2. 
Change from Baseline .................................................................................................31 
5.2.3. 
Study Day ...................................................................................................................31 
5.3. 
Missing Data ...............................................................................................................31 
5.3.1. 
Handling of Missing Dates/Months/Years for All Adverse Events ...........................31 
5.3.2. 
Handling of Missing Dates/Months/Years for Concomitant Medications .................32 
5.4. 
Visit Windows ............................................................................................................32 
5.5. 
Subgroups ...................................................................................................................33 
6. 
DEMOGRAPHIC, OTHER BASELINE CHARACTERISTICS AND 
MEDICATION ...........................................................................................................33 
6.1. 
Subject Eligibility, Disposition and Protocol Deviations ...........................................33 
6.2. 
Demographic and Other Baseline Characteristics ......................................................34 
6.3. 
Baseline Disease Characteristics ................................................................................34 
6.4. 
Refractory Disease: No Response to Therapy (no CR or PR) or Response 
Lasting <6 months Medical History and Concomitant Diseases ................................35 
6.5. 
Prior and Concomitant Medications ...........................................................................35 
6.6. 
Prior Therapies ............................................................................................................35 
6.7. 
Concomitant Procedures .............................................................................................36 
7. 
EFFICACY ANALYSIS ............................................................................................36 
7.1. 
Primary Efficacy Endpoint and Analysis ...................................................................36 
7.2. 
Secondary Efficacy Endpoints and Analyses .............................................................37 
7.2.1. 
Objective Response Rate (ORR) by Investigator .......................................................37 
7.2.2. 
Duration of Response (DOR) and Duration of Recaptured Response 
(DORR) .......................................................................................................................37 
7.2.3. 
Complete Response (CR) Rate ...................................................................................38 


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Statistical Analysis Plan
Protocol ME-401-003 
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CONFIDENTIAL 
 
 
7.2.4. 
Progression Free Survival (PFS) ................................................................................38 
7.2.5. 
Time to Treatment Failure (TTF) ...............................................................................38 
7.2.6. 
Recapture of Response Rate (RRR) ...........................................................................38 
7.2.7. 
Time to Recapture of Response ..................................................................................39 
7.2.8. 
Overall Survival (OS) .................................................................................................39 
8. 
SAFETY ANALYSES ...............................................................................................39 
8.1. 
Extent of Exposure .....................................................................................................39 
8.2. 
Treatment Compliance ................................................................................................40 
8.3. 
Adverse Events and Adverse Events of Special Interest ............................................41 
8.3.1. 
Treatment-Emergent Adverse Event (TEAE) ............................................................41 
8.3.2. 
Adverse Events of Special Interest (AESI) ................................................................41 
8.3.3. 
Summaries of Incidence of Adverse Events ...............................................................42 
8.3.4. 
Analysis of Time to Occurrence of AESIs .................................................................42 
8.4. 
Laboratory Evaluations ...............................................................................................43 
8.5. 
Vital Signs ..................................................................................................................43 
8.6. 
Cardiac Evaluations ....................................................................................................43 
8.7. 
Physical Examination .................................................................................................44 
8.8. 
Eastern Co-operative Oncology Group (ECOG) ........................................................44 
9. 
INTERIM ANALYSES ..............................................................................................44 
10. 
CHANGES FROM ANALYSIS PLANNED IN PROTOCOL .................................44 
11. 
REFERENCE LIST ....................................................................................................45 
12. 
PROGRAMMING CONSIDERATIONS ..................................................................46 
12.1. 
General Considerations ...............................................................................................46 
12.2. 
Table, Listing, and Figure Format ..............................................................................46 
12.2.1. 
General ........................................................................................................................46 
12.2.2. 
Headers .......................................................................................................................46 
12.2.3. 
Display Titles ..............................................................................................................47 
12.2.4. 
Column Headers .........................................................................................................47 
12.2.5. 
Body of the Data Display ...........................................................................................47 
12.2.5.1. 
General Conventions ..................................................................................................47 
12.2.5.2. 
Table Conventions ......................................................................................................47 
12.2.5.3. 
Listing Conventions ....................................................................................................48 
12.2.6. 
Footnotes.....................................................................................................................48 


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Statistical Analysis Plan
Protocol ME-401-003 
Page 7 of 49
 
 
CONFIDENTIAL 
 
 
13. 
QUALITY CONTROL ...............................................................................................49 
 
 
LIST OF TABLES 
Table 1: 
Schedule of Assessments (Amendment 4) .................................................................17 
Table 2: 
Censoring Rules for the Primary Analysis of DOR and DORR .................................24 
Table 3: 
Censoring Rules for the Supportive Analysis of DOR ...............................................24 
Table 4: 
Types of Analyses and Corresponding Populations for Planned Primary 
Analysis and NDA Analysis for FL ............................................................................30 
 
 
LIST OF FIGURES 
Figure 1a: Study Schema (Protocol Amendment 1) ....................................................................13 
Figure 1b: Study Schema (Protocol Amendments 2 and 3) .........................................................14 
 
 
 


MEI Pharma, Inc. 
Statistical Analysis Plan
Protocol ME-401-003 
Page 8 of 49
 
 
CONFIDENTIAL 
 
 
LIST OF ABBREVIATIONS 
Abbreviation 
Description 
AE  
Adverse Event  
AESI 
Adverse Events of Special Interest 
ALP 
Alkaline Phosphatase 
ALT 
Alanine Aminotransferase 
ANC 
Absolute Neutrophil Count 
aPTT 
Activated Partial Thromboplastin Time 
AST 
Aspartate Aminotransferase 
ATC 
Anatomical Therapeutic Chemical  
BAT 
Basophil activation test 
BCRP 
Breast cancer resistance protein transporter 
BP 
Blood Pressure 
BTK 
Bruton tyrosine kinase 
BUN 
Blood Urea Nitrogen 
CAR T 
Chimeric Antigen Receptor T-cell 
CBC 
Complete Blood Count 
CI  
Confidence Interval  
CLL 
Chronic lymphocytic leukemia 
CMR 
Complete metabolic response 
CMV 
Cytomegalovirus 
CR  
Complete Response/remission  
CRi 
Complete remission with incomplete marrow recovery 
CS  
Continuous Schedule  
CT 
Computed Tomography 
CYP2C8 
Cytochrome P450 2C8 
CyTOF 
Mass cytometry 
DLBCL 
Diffuse large B-cell lymphoma 
DLT  
Dose Limiting Toxicity  
DNA 
Dexyribonucleic acid 
DOR  
Duration of Response  
DORR 
Duration of recaptured response 
DSMB  
Data Safety and Monitoring Board 
EC 
Ethics Committee 
ECG 
Electrocardiogram 
ECOG 
Eastern Cooperative Oncology Group 
EDC   
Electronic Data Capture  
eCRF  
Electronic Case Report Form  
eGFR 
Estimated glomerular filtration rate 
Emax 
Maximum possible effect 
FDA  
Food and Drug Administration  
FDG 
18F-fluorodeoxyglucose 


MEI Pharma, Inc. 
Statistical Analysis Plan
Protocol ME-401-003 
Page 9 of 49
 
 
CONFIDENTIAL 
 
 
Abbreviation 
Description 
Abbreviation 
Description 
FL  
Follicular Lymphoma  
FLIPI 
Follicular Lymphoma International Prognositc Index 
GCP  
Good Clinical Practice  
GELF 
Groupe d’Etudes des Lymphomes Folliculaires  
GI 
Gastrointestinal  
HcT 
Hematocrit  
HR 
Heart Rate 
ICF  
Informed Consent Form  
ICH  
International Council for Harmonisation  
IF  
immunofixation  
IND  
Investigational New Drug  
IRB  
Institutional Review Board  
IRRC  
Independent Response Review Committee  
IS  
Intermittent Schedule  
IV  
intravenous  
iwCLL  
International Workshop on Chronic Lymphocytic Leukemia  
kg  
kilogram  
L  
liter  
mAb  
monoclonal Antibody  
MCL  
mantle cell lymphoma  
MCH 
Mean Corpuscular Hemoglobin 
MCR 
Metabolic Complete Response 
Min  
Minimum   
mm3  
cubic millimeter  
mg  
milligram  
mL  
milliliter  
MRD  
minimal residual disease  
MTD  
Maximum tolerated dose  
MUGA 
Multigated Acquisition Scan 
MZL  
marginal zone lymphoma  
NCI CTCAE  
National Cancer Institute Common Terminology Criteria for Adverse Events  
ND  
Not Detected  
NDA 
New Drug Application 
NE  
not evaluable 
NHL  
non-Hodgkin lymphoma  
NIP  
non-infectious pneumonitis  
NMR  
no metabolic response  
NOAEL  
no-observed-adverse-effect level  
NOS  
not otherwise specified  
OI 
Opportunistic Infection  


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Statistical Analysis Plan
Protocol ME-401-003 
Page 10 of 49
 
 
CONFIDENTIAL 
 
 
 
Abbreviation 
Description 
OR  
rates of overall response rate  
Abbreviation 
Description 
ORR  
Objective Response Rate  
OS  
Overall Survival  
PCR 
Polymerase Chain Reaction 
PD  
Progressive disease  
PET/CT  
Positron Emission Tomography – Computed Tomography 
PFS  
Progression-free survival  
PI3K  
phosphatidylinositol 3-kinase  
PI3Kδ  
Phosphatidylinositol 3-kinase Delta  
PJP  
Pneumocystis jiroveci pneumonia  
PK  
Pharmacokinetics  
PMD  
progressive metabolic disease  
PMR  
partial metabolic response  
POD24  
progression of disease within 24 months of diagnosis after frontline treatment 
with chemoimmunotherapy  
PR  
Partial Response  
QTc  
QT corrected (corrected QT-interval)  
QTcF  
QT-interval corrected according to Fridericia’s formula  
R-bendamustine  
rituximab, bendamustine  
R-CVP  
rituximab, cyclophosphamide, vincristine, prednisone  
R-CHOP  
rituximab, cyclophosphamide, doxorubicin, vincristine, prednisone  
RP2D  
Recommended Phase 2 Dose  
SAE  
Serious Adverse Event  
SAP  
Statistical Analysis Plan  
SGOT  
serum glutamic-oxaloacetic transaminase  
SGPT  
serum glutamic-pyruvate transaminase  
SLL  
small lymphocytic lymphoma  
SUSAR  
suspected unexpected serious adverse reaction  
TEAE  
Treatment-emergent Adverse Event  
TMP-SMX  
trimethoprim-sulfamethoxazole  
TREG  
T regulatory cells  
TTF 
Time to Treatment Failure 
ULN  
upper limit of normal  
US(A)  
United States (of America)  
WHO  
World Health Organization 


MEI Pharma, Inc. 
Statistical Analysis Plan
Protocol ME-401-003 
Page 11 of 49
 
 
CONFIDENTIAL 
 
 
1. 
PURPOSE 
The purpose of this statistical analysis plan (SAP) is to ensure that the data listings, summary 
tables and figures which will be produced, and the statistical methodologies that will be used, are 
complete and appropriate to allow valid conclusions regarding the study objectives. 
1.1. 
Responsibilities 
Syneos Health will perform the statistical analyses and are responsible for the production and 
quality control of all tables, figures and listings, except for the Population Pharmacokinetics 
(PK) and Exposure-Response analyses. A separate Population PK and Exposure-Response 
analysis plan will be developed by Nuventra for these analyses. MEI Pharma will review the 
statistical analyses and are responsible for final approval of all tables, figures and listings.  
1.2. 
Timings of Analyses 
The primary analysis for the primary efficacy endpoint of overall response rate (ORR) for the 
follicular lymphoma (FL) cohort (i.e., Primary Analysis for FL) will be triggered after all 
91 subjects in the Primary Efficacy Population in FL (PEP FL) enrolled in the study (except 
those who discontinue early) have been followed for at least 6 months from the start of study 
drug.  
The final analysis for DOR for follicular lymphoma (FL) to support the New Drug Application 
(NDA) initial filing for FL (i.e., NDA Analysis for FL) will be triggered after most responders in 
the Primary Efficacy Population have a minimum of 12 months of follow-up from first response. 
The primary analysis for the primary efficacy endpoint of ORR for marginal zone lymphoma 
(MZL) (i.e., Primary Analysis for MZL) will be triggered after all subjects in the ITT MZL 
population enrolled in the study (except those who discontinue early) have been followed for 
at least 6 months from the start of study drug.  
The final analysis for DOR for MZL to support the NDA filing for MZL (i.e., NDA Analysis 
for MZL) will be triggered after most responders in the ITT MZL population have a minimum 
of 12 months of DOR follow-up. 
The final analysis includes all data collected through the time of the database lock. PK and 
Exposure-Response analyses will be performed by Nuventra, who will produce the analysis 
report. 
An independent DSMB (Data Safety and Monitoring Board) will review descriptive summaries 
of accumulating safety, subject disposition, and limited efficacy data every 6 months. 
Further description of the DSMB analyses can be found in the DSMB charter.  
2. 
STUDY OBJECTIVES 
2.1. 
Primary Objective 
• To evaluate the objective response rate (ORR) of ME-401 in relapsed or refractory FL or 
MZL, defined as the best response rating of complete response (CR) or partial 
response (PR) based on the Lugano Response Criteria (Cheson 2014) as modified in 


MEI Pharma, Inc. 
Statistical Analysis Plan
Protocol ME-401-003 
Page 12 of 49
 
 
CONFIDENTIAL 
 
 
the study protocol, and as determined by an Independent Response Review Committee 
(IRRC).  
2.2. 
Secondary Objectives 
• To evaluate the efficacy of ME-401 as assessed by an IRRC: 
o Duration of response (DOR)  
o Complete response (CR) rate 
o Progression-free survival (PFS) 
o Time to treatment failure (TTF) 
o Recapture of response 
o Duration of recaptured response (DORR) 
• To evaluate the efficacy of ME-401 as assessed by the Investigator: 
o Objective response rate (ORR) 
o Duration of response (DOR)  
o Complete response (CR) rate 
o Progression-free survival (PFS) 
o Time to treatment failure (TTF) 
o Recapture of response 
o Duration of recaptured response (DORR) 
• To evaluate overall survival (OS) 
• To evaluate the safety profile of ME-401 
o Overall incidence of treatment-emergent adverse events (TEAEs) 
o Incidence of adverse events of special interest (AESIs) 
o Time to occurrence of AESIs 
• To evaluate the pharmacokinetics (PK) of ME-401 
2.3. 
Brief Description 
Under Protocol Amendment 2, this is a global, multicenter, open label, single-arm, phase 2 study 
of the PI3Kδ inhibitor ME-401 (zandelisib) in subjects with relapsed/refractory FL or MZL. 
ME-401 will be administered orally at a dose of 60 mg given once a day in 28-day cycles, with 
the first day of treatment designated as Day 1. Treatment with ME-401 can be administered 
based on a continuous schedule (CS) or an intermittent schedule (IS). 
The CS (or Group A in Protocol Amendment 1) includes daily dosing for the whole duration of a 
treatment cycle until protocol-defined criteria is met for treatment interruption, modification to 
intermittent dosing to manage toxicity, or discontinuation.  


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Protocol ME-401-003 
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The IS (or Group B in Protocol Amendment 1) includes: 
• continuous daily therapy for the initial 2 cycles (~56 days) of treatment 
• the intermittent treatment begins at Cycle 3, with ME-401 administered daily for the first 
7 days of every 28-day cycle (7 days on treatment and 21 days off treatment) 
Under Protocol Amendment 1 (Figure 1a), subjects were randomized 1:1 to receive CS or IS. 
Since Protocol Amendment 2 (Figure 1b), the initial CS treatment arm was terminated, and all 
new subjects were enrolled to receive therapy on the IS only. If CS subjects already completed 
2 initial cycles of therapy, they were switched to IS dosing after they complete therapy in the 
ongoing cycle of treatment, and if they had not completed the first 2 cycles of therapy they were 
switched to IS dosing after completion of the first two cycles of therapy on CS. Subjects who 
progress on IS dosing may be switched to CS dosing. Subjects will continue to receive study 
drug in accordance with the IS dosing regimen (or CS dosing regimen if switched from IS after 
disease progression [PD]) until investigator-assessed disease progression or unmanageable 
toxicity requiring treatment discontinuation, subject’s decision to withdraw their consent to 
participate on study, or study closure by the Sponsor.  
Figure 1a: 
Study Schema (Protocol Amendment 1) 
  
 
Abbreviations: AERDM = AEs requiring dose modification or drug discontinuation; Rand = randomization. 
a For subjects who require study drug interruption to manage ME-401 related toxicity or have disease progression. 
b Safety follow up will occur 30 days (±3 days) from the last day of study drug treatment. Post-treatment follow-up 
will be conducted every 3 months to obtain data regarding disease progression, improvement in disease response, 
start of subsequent lymphoma therapy and death.  


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Protocol ME-401-003 
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CONFIDENTIAL 
 
 
Figure 1b: 
Study Schema (Protocol Amendments 2 and 3) 
 
Abbreviations: EOT = End of Treatment; FU = follow up; PD = progressive disease. 
a Screening from Day -28 to Day -1. 
b IS dosing schedule: 60 mg daily for the first 2 cycles (56 days) followed by 60 mg daily for 7 days of every 28-day 
cycle (7 days on / 21 days off treatment). 
c Follow-up Period will include: 1) EOT/30-Day Safety Follow-up visit will occur 30 (±3) days from the last day of 
study drug treatment. 2) Efficacy Follow-up - for subjects who discontinue study treatment due to reasons other 
than disease progression or death, response follow up will occur until PD of the start of a new anti-cancer therapy. 
3) Survival Follow-up visits will occur every 3 months until death, and will continue for up to 3 years after the last 
subject was enrolled in the study. 
d Response assessments will be performed every 2 months (±7 days) for the first 6 months of therapy, every 
3 months (±7 days) during the following 12 months, and every 6 months (±14 days) thereafter (starting with 
month 18).  
Subjects who discontinue study drug will remain in the study with follow-up conducted every 
3 months from the last dose of study drug to obtain post-treatment long-term follow-up 
information on disease status (i.e., change in disease response, progression), start of subsequent 
therapy, and survival (death). During this post-treatment follow-up phase, all subjects who 
discontinued treatment due to reasons other than PD must continue imaging tests to assess 
disease status, performed per the original protocol-defined schedule for disease/response 
assessment, or earlier if clinically indicated. Imaging test results will no longer be collected when 
a subject starts a new therapy or disease progression occurs and is documented in the 
post-treatment long-term follow-up phase. Survival follow-up will continue for up to 3 years 
after the last subject is enrolled in the study.  
Individual subject participation in the study will include 28 days for screening, approximately 
18 months of dosing with study drug, and follow-up for survival after discontinuation of study 
drug. Actual subject participation in the study may be shorter or longer depending on disease 
response, tolerability to therapy, and duration of long-term follow-up. The study will continue 
until all enrolled subjects (except those who discontinue early) have been followed for at least 
6 months from the start of study drug. 


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2.4. 
Determination of Sample Size 
Sample Size for FL   
The study will test the null hypothesis that the IRRC-reviewed ORR is ≤48% against the 
alternative hypothesis that it is ≥65.5%. The sample size for the primary efficacy population for 
FL is estimated to be 91 subjects, with at least 90% power and 1-sided alpha = 0.025. 
Safety analysis will be performed based on the Safety Population, with analysis focused on the 
IS FL subjects at the primary analysis database lock for FL submissions. With a total sample size 
of 120 FL subjects treated with ME-401 IS dosing regimen, if ≤15 subjects experience Grade 
≥3 AESIs (i.e., subject incidence rate of ≤12.5% with 95% CI of 7.2–19.8%), the study can rule 
out the possibility of having an incidence rate ≥20% for Grade ≥3 AESIs with the IS dosing 
regimen. In addition, with a total sample size of 120 FL subjects, if ≤5 subjects experience a 
particular type of events (i.e., subject incidence rate of ≤4.2% with 95% CI 1.4-9.5%), the study 
can rule out the possibility of having an incidence rate of ≥10% for this AE. 
Sample Size for MZL 
The study will test the null hypothesis that the IRRC-reviewed ORR is ≤46% against the 
alternative hypothesis that it is ≥66.5%. The sample size for the MZL population is estimated to 
be 64 subjects, with at least 90% power and 1-sided alpha = 0.025. 
For the supplement submission for MZL indication, safety analysis will be focused on all 
subjects treated with ME-401 IS dosing regimen. With a total sample size of 184 subjects treated 
with the ME-401 IS dosing regimen in both the FL and MZL subjects, if ≤25 subjects experience 
Grade ≥3 AESIs (i.e., subject incidence rate of ≤13.6% with 95% CI of 9.0 – 19.4%), the study 
can rule out the possibility of having an incidence rate ≥20% for Grade ≥3 AESIs with the IS 
dosing regimen. In addition, with a total sample size of 184 subjects treated with the ME-401 
dosing regimen in both the FL and MZL populations, if ≤9 subjects experience this type of 
adverse events (i.e., subject incidence rate of ≤4.9% with 95% CI 2.3-9.1%), the study can rule 
out the possibility of having an incidence rate of ≥10% for this AE. 
2.5. 
Treatment Assignment  
Under Protocol Amendment 1 (Figure 1a), subjects are randomized 1:1 to Group A (CS) or 
Group B (IS), stratified accordingly to tumor bulk (largest lymphoid mass <5 cm vs. ≥5cm) and 
disease response to last therapy (relapse vs. refractory). 
Since Protocol Amendment 2 (Figure 1b), this is an open-label and single-arm phase 2 study that 
all new subjects will be enrolled and treated by IS dosing only. During the first 2 cycles, subjects 
will receive open-label ME-401 administered at 60 mg once daily. After completing 2 cycles of 
daily therapy, subjects will continue ME-401 administered at 60 mg daily for the first 7 days of 
every 28-day cycle (7 days on treatment and 21 days off treatment). Subjects who progress 
disease on IS dosing have the option to have their ME-401dosing switched to CS dosing.  
Since Protocol Amendment 2, the initial CS treatment arm was terminated, and switched to IS 
dosing. For CS Subjects from Protocol Amendment 1, those who already completed 2 initial 
cycles of therapy were switched to IS dosing once they complete the ongoing cycle; those who 
had not completed the first 2 cycles were switched to IS dosing after the first 2 cycles.   


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Protocol ME-401-003 
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For subjects who experience toxicity during the first 2 cycles while receiving daily therapy with 
ME-401, dose interruption followed by a switch to the intermittent schedule daily dosing for the 
first 7 days of every 28-day cycle is allowed. Subjects who experience toxicities on the IS will be 
managed by the instructions for management of potential drug-related toxicity 
(Protocol Amendment 3, Table 4), which may include dose interruption or permanent 
discontinuation of ME-401.  
2.6. 
Study Procedures and Flowchart 
All study assessments for procedures will be performed at the visits and time points outlined in 
the Schedule of Assessments (Table 1). 
 


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Protocol ME-401-003 
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CONFIDENTIAL 
 
 
Table 1: 
Schedule of Assessments (Amendment 4) 
Assessment/Procedure 
Screening b 
Cycle 
1 
Cycle 
2 
Cycle 
3 
Cycle 
4 
Cycle 
5 
Cycle 
6 
Cycle 
7 
Cycles 10, 
13, 16, 19, 
22, 25 d… 
EOT/30-Day 
Safety 
Follow-up e 
Post-
Treatment 
Long-Term 
Follow-up 
Day(s) of Cycle 
-28 to -1 
1 c 
8 
15 
1 
15 
1 
1 
7 
1 
1 
7 
1 
1 
30 (± 3) days  
post last dose 
or treatment 
discontinuation 
Every  
3 Months 
Study Day(s) 
-28 to -1 
1 
8 
15 
29 
43 
57 
85 
91 
113 
141 
147 
169 
253, 337, 
421, 505, 
589… 
 
 
Visit Window (days) a 
 
 
±3 
±3 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±7 a 
 
 
Informed consent form 
(ICF) 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Medical history f 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Physical examination 
X 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Symptom-directed exam g 
 
 
X 
X 
 
X 
 
 
 
 
 
 
 
 
 
 
Vital signs h 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Height 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Weight 
X 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
X 
 
ECOG performance status 
X 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Pregnancy test i 
X 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
X 
 
HIV testing j 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Hepatitis B and C testing k 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Adverse events l 
 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Concomitant medication m 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
 
X 
X 
X 
 
ECG n 
X 
X 
 
 
 
 
X 
 
 
 
X 
 
 
X 
X 
 
CBC with differential o 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Serum chemistry p 
X 
X 
X 
X 
X 
X 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Coagulation q 
X 
X 
 
 
X 
 
X 
 
 
X 
 
 
X 
X 
X 
 
PCR test for CMV 
X 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
 
 
Urinalysis (Dipstick) r 
X 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
X 
 


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Protocol ME-401-003 
Page 18 of 49
 
 
CONFIDENTIAL 
 
 
Assessment/Procedure 
Screening b 
Cycle 
1 
Cycle 
2 
Cycle 
3 
Cycle 
4 
Cycle 
5 
Cycle 
6 
Cycle 
7 
Cycles 10, 
13, 16, 19, 
22, 25 d… 
EOT/30-Day 
Safety 
Follow-up e 
Post-
Treatment 
Long-Term 
Follow-up 
Day(s) of Cycle 
-28 to -1 
1 c 
8 
15 
1 
15 
1 
1 
7 
1 
1 
7 
1 
1 
30 (± 3) days  
post last dose 
or treatment 
discontinuation 
Every  
3 Months 
Study Day(s) 
-28 to -1 
1 
8 
15 
29 
43 
57 
85 
91 
113 
141 
147 
169 
253, 337, 
421, 505, 
589… 
 
 
Visit Window (days) a 
 
 
±3 
±3 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±7 a 
 
 
COVID-19 test s 
X 
 
Issue and assess 
compliance diary t 
 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
 
 
Dispense study drug u 
 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
 
 
PK Sampling v 
 
X 
 
Xw 
X 
 
 
 
X 
 
 
X 
 
 
 
 
Echocardiogram 
or MUGA x 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 
Correlative immune study 
(pre-dose) y 
 
X 
 
X 
X 
 
X 
 
 
X 
 
 
X 
X 
X 
 
Opportunistic infection 
monitoring z 
 
X 
 
 
X 
 
X 
X 
 
X 
X 
 
X 
X 
X 
 
Post-Treatment Follow-up 
procedures aa 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
 


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Protocol ME-401-003 
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Response Assessment bb,cc 
Screening 
Every 2 Months for the 
First 6 Months 
Every 3 Months for the Next 12 Months 
(through 18 Months) 
Every 6 Months Thereafter 
Study Day 
 
61 
121 
182 
274 
365 
456 
547 
730, 912, 1095… 
Visit Window (days) 
 
±7 
±7 
±7 
±7 
±7 
±7 
±7 
±14 
CT neck/chest/abdomen/pelvis dd 
X 
X 
X 
X 
X 
X 
X 
X 
X 
FDG-PET scan ee 
X 
 
X 
 
 
X 
 
 
 
Bone marrow biopsy/aspirate ff 
X 
Endoscopy with biopsy gg 
X 
Abbreviations: ALP = alkaline phosphatase; ALT = alanine aminotransferase; ANC = absolute neutrophil count; aPTT = activated partial thromboplastin time; AST = aspartate 
aminotransferase; BP = blood pressure; BUN = blood urea nitrogen; CBC = complete blood count; CMV = cytomegalovirus; CMR = complete metabolic response; CO2 = carbon 
dioxide; CR = complete response; CT = computed tomography; ECG = electrocardiogram; ECOG = Eastern Cooperative Oncology Group; EOT = end of treatment; 
FDG-PET = 18F-fluorodeoxyglucose positron emission tomography; HcT = hematocrit; HGB = hemoglobin; HIV = human immunodeficiency virus; HR = heart rate; 
ICF = informed consent form; INR = international normalized ratio; LDH = lactate dehydrogenase; MCH = mean corpuscular hemoglobin; MUGA = multigated acquisition scan; 
PCR = polymerase chain reaction; PK = pharmacokinetic; PT = prothrombin time; RBC = red blood cell; SOC = standard of care; WBC = white blood cell. 
a. Study Assessment Day(s) – There is ±3-day window allowable for each clinic visit for Cycle 1 Day 8 and Cycle 1 Day 15.  For Cycles 2 through 25, the allowable window is 
±7 days. After Cycle 25, the allowable window is ±1 month for each clinic visit and its associated assessments. 
b. Screening – Screening assessments are to be performed within 28 days prior to enrollment. Upon confirmation of meeting eligibility criteria, subjects should be treated within a 
reasonable time frame (within 5 days). If there are >7 days between screening laboratory assessments and expected Cycle 1 Day 1 dosing, then the laboratory assessments 
should be repeated and reviewed prior to the first dose of ME-401. Some tests performed prior to obtaining ICF (e.g., CT scan, bone marrow biopsy) may not need to be 
repeated for the study as long as they were performed within the allowed screening window. 
c. Cycle 1 Day 1 – Physical examination need not be repeated on Cycle 1 Day 1 if performed within 7 days prior to start of treatment. Laboratory assessments (CBC with 
differential, serum chemistry, coagulation, urinalysis) need not be repeated on Cycle 1 Day 1 if performed within 7 days prior to start of treatment. 
d. Cycle 25+ – Subjects who remain on study after 25 cycles (approximately 2 years) will visit the clinic every 3 months (±1 month), with a response assessment performed every 
6 months as per standard of care. 
e. EOT/30-Day Safety Follow-up – Visit will be completed 30 (±3) days post last dose of study drug administration (or prior to starting a new treatment if urgent treatment 
is required) or from study drug discontinuation. 
f. If known, record in the medical history information on COVID-19 vaccination administered at any time prior to subject consent to participate in the study. 
g. Symptom-directed exam – Performed on Cycle 1 Day 8, Cycle 1 Day 15, and Cycle 2 Day 15, only as needed/indicated, based on interim history. 
h. Vital signs – BP, HR, temperature. 
i. Pregnancy test – The screening serum pregnancy test in females of childbearing potential must be completed within 28 days prior to Cycle 1 Day 1. Urine or serum pregnancy 
test must be performed on study Day 1. For women of childbearing potential, a monthly serum or urine pregnancy test will be conducted every dosing cycle and 30 days after 
discontinuation of dosing. 
j. HIV testing – HIV antibody is required. 
k. Hepatitis B and C testing – Hepatitis B core antibody, Hepatitis B surface antigen, and hepatitis C antibody are required. Hepatitis B PCR is required if hepatitis B core 
antibody and/or surface antigen is positive. Hepatitis C PCR is required if hepatitis C antibody is positive. 
l. Adverse events – Recorded from the time of the first dose of study drug on Cycle 1 Day 1 and continues until 30 days after the last dose of study drug or start of new 
anti-cancer treatment. 
m. Concomitant medications – Recorded from 28 days prior to Cycle 1 Day 1 until the EOT/30-Day Safety Follow-up visit, or start of new anti-cancer treatment. Please, record 
information about COVID-19 vaccination, if performed. 


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Statistical Analysis Plan
Protocol ME-401-003 
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CONFIDENTIAL 
 
 
n. ECG – 12-lead ECGs performed at screening; Cycle 1 Day 1 pre-dose (unless the Screening visit is within 3 days prior to Day 1); pre-dose on Day 1 of Cycles 3, 6, 13, 19, 
and 25, and every 6 months thereafter; at the EOT/30-Day Safety Follow-up visit, and as clinically indicated. ECGs should be performed pre-dose. For Cycle 1 Day 1 and 
Cycle 3 Day 1, ECGs should be taken pre-dose and at expected Cmax (3–5 hours after dosing). 
o. CBC with differential – WBC, ANC, RBC, HGB, HcT, lymphocytes, eosinophils, monocytes, basophils, platelets, and MCH. Screening labs must be repeated if performed 
>7 days prior to Cycle 1 Day 1. 
p. Serum chemistry – Glucose, BUN or urea, creatinine, sodium, potassium, chloride, calcium, ALP, AST, ALT, total bilirubin, total protein, albumin, LDH, and phosphorous.  
Screening labs must be repeated if performed >7 days prior to Cycle 1 Day 1.   
q. Coagulation – aPTT, PT/INR. Screening labs must be repeated if performed >7 days prior to Cycle 1 Day 1.  
r. Urinalysis – Dipstick or urine test (pH, specific gravity, glucose, ketones, blood, and protein). Screening labs must be repeated if performed >7 days prior to Cycle 1 Day 1. 
s. COVID-19 test results – Please record COVID-19 test results in the AE CRF page, if positive. 
t. Issue and Assess Compliance Diary – Compliance diary to record daily study drug administration. Returned diaries are to be assessed for study product compliance. 
New compliance diary is to be issued with each dispensing of ME-401. 
u. Dispense Study Drug – Study drug is to be taken orally once a day on an empty stomach at least 1 hour prior to food intake or 2 hours after food intake at the same time each 
day. On days of PK sampling, instruct subjects to not take study drug until after the pre-dose PK sample is drawn. Compliance diary and count of returned capsules are 
performed on each day of dispensing study drug. 
v. PK sampling – Pre-dose PK samples are to be obtained within 1 hour before study drug administration on Cycle 1 Day 1, Cycle 2 Day 1, Cycle 4 Day 7, and Cycle 6 Day 7. 
Pre-dose PK samples collected after Cycle 1 Day 1 are to be obtained within ±60 minutes of the scheduled time, which is based on the Cycle 1 Day 1 dosing time.  
w. PK Sampling – Cycle 1 Day 15 – pre-dose and 3 hours (±30 min) post intake of study drug. Collect data regarding food and drink intake 4 hours prior to study drug intake. 
x. Echocardiogram or MUGA – Performed at screening, EOT/30-Day Safety Follow-up visit, and as clinically indicated. The same methodology (echocardiogram or MUGA) 
should be used throughout the study. 
y. Correlative Sample Study – US Sites only. Samples will be obtained pre-dose on Day 1 of Cycle 1, and then pre-dose on the following visits: Day 15 of Cycle 1; Day 1 of 
Cycles 2, 3, 5, 7, 10; then every 3 cycles thereafter; and at the EOT/30-Day Safety Follow-up visit.  Fresh blood samples will be collected and shipped overnight to a central 
laboratory for analysis.  Samples may be shipped only Monday to Thursday. Samples may not be shipped for delivery on weekends or holidays. If possible, a sample will also 
be obtained when a subject develops a late onset Grade 2 AE of diarrhea/colitis, rash/mucositis, transaminase elevation, or non-infectious pneumonitis, or when a subject 
discontinues study drug permanently for another reason. Samples will be collected at the first visit of the adverse event, at the time when treatment is held, and at the time when 
treatment is resumed.  
z. Opportunistic Infection Monitoring – Prior to start of every cycle of treatment with ME-401, monitoring will be conducted, and for subjects with identified risk factors and 
those who developed an OI while on study treatment, additional assessments should include: CD4 and CD8 count and ratio, c-reactive protein, blood cultures, any additional 
laboratory and diagnostic methods according to local SOC reported as unscheduled laboratory and diagnostic methods of assessment, radiological imaging (i.e., chest X-ray or 
CT scans). Note: Treatment of the OI that started while on study should be based on local SOC. 
aa. Post-treatment Follow-up Procedures – Every 3 months from the last dose of study drug to obtain data regarding changes in efficacy assessments, death or subsequent 
therapy based on standard of care assessments. Imaging tests to assess disease status will be performed at least once every 6 months or earlier as indicated per standard of care. 
Any AE that has not returned to baseline or Grade ≤2 should be followed until it has returned to baseline or better, the event is assessed as stable by the Investigator, the subject 
is lost to follow up, or the subject withdraws consent. Survival follow-up will continue for up to 3 years after the last subject is enrolled in the study.  
bb. Response assessment – Imaging scans, physical examination, bone marrow aspirate/biopsy (if applicable), and laboratory evaluation as appropriate for individual subjects.   
cc. Response assessment at the EOT/30-Day Safety Follow-up visit – Perform imaging test (CT scan or PET/CT scan) only if last imaging tests outside the window noted for 
CT scan or PET-CT. 
dd. CT Scan – CT of the neck, chest, abdomen, and pelvis must be obtained at screening (within 6 weeks prior to Cycle 1 Day 1). CT scans must be performed using IV contrast. 
For subjects who are intolerant to contrast agents, the CT scan will be performed with oral contrast or MRI scans will be used. 
ee. PET/CT – FDG-PET scan must be obtained at screening (within 6 weeks prior to Cycle 1 Day 1), as indicated, and to confirm CR/CMR. PET/CT imaging is no longer needed 
once a CMR has been achieved and a CT scan is an appropriate imaging test. 
ff. Bone marrow biopsy/aspirate – Bone marrow biopsy/aspirate is obtained at screening and to confirm CR if PET scans were not performed or were not evaluable to assess 
bone marrow involvement. In addition, it can be performed as clinically indicated at the discretion of the Investigator.   


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Statistical Analysis Plan
Protocol ME-401-003 
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CONFIDENTIAL 
 
 
gg. Endoscopy with biopsy – For subjects with gastric MZL, endoscopy with biopsy to be performed to confirm CR. 
 


MEI Pharma, Inc. 
Statistical Analysis Plan
Protocol ME-401-003 
Page 22 of 49
 
 
CONFIDENTIAL 
 
 
3. 
ENDPOINTS 
3.1. 
Primary Efficacy Endpoint 
The primary efficacy endpoint is ORR, defined as the proportion of subjects achieving the best 
response rating of CR or PR based on the Modified Lugano Classification (response criteria 
summarized in Appendix 5 of Protocol Amendment 2), as determined by IRRC. 
For both Primary Analysis and NDA Analysis for FL, ORR will include best response of PR 
and/or CR prior to first PD that is reported before the data cutoff date for FL Primary Analysis, 
among all patients in the Primary Efficacy Population in FL (PEP FL). 
As supportive analyses for NDA Analysis for FL, ORR will be summarized by 1) including PR 
or CR achieved that is reported before the data cutoff date for NDA Analysis for FL, among all 
patients in the Primary Efficacy Population in FL; 2) including PR or CR achieved that is 
reported before the data cutoff date for NDA Analysis for FL, among all patients in the ITT FL 
Population. 
Time to response will be assessed to provide supportive information for the primary endpoint of 
ORR. It is defined as the time from the first dose of study drug to the first objective response for 
the subjects who achieved best response of CR or PR. 
3.2. 
Secondary Efficacy Endpoints 
The analysis of secondary efficacy endpoints, DOR, CR rate, PFS, TTF, recapture of response, 
DORR will be performed accordingly to the disease assessment reported by the IRRC. Analysis 
of ORR, DOR, CR rate, PFS, TTF, recapture of response, DORR will also be performed using 
Investigator assessment of disease response. In addition, the overall survival will be analyzed. 
Recapture of analysis and DORR will only be summarized for the IS patients who switched back 
to CS after PD. 
3.2.1. 
Objective Response Rate (ORR) by Investigator 
ORR defined as the proportion of subjects achieving the best response rating of CR or PR based 
on the Lugano Classification, as determined by the Investigator. 
3.2.2. 
Duration of Response (DOR) and Duration of Recaptured Response (DORR) 
DOR is defined as time from first (CR or PR) response to the time of first PD (based on the 
Modified Lugano Response Criteria) or the date of death due to any cause (whichever occurs 
earlier).  
For Primary Analysis and NDA Analysis for FL, DOR will be analyzed based on the patients 
who achieved PR or CR prior to first PD, that is reported before the data cutoff date for the 
FL Primary Analysis.  
As a supportive analysis for NDA Analysis for FL, DOR will be analyzed based on all patients 
who achieved PR or CR prior to first PD, that is reported before the data cutoff date for the 
NDA Analysis for FL submission. 


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Protocol ME-401-003 
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To evaluate the duration of response for subjects who had recapture of response on the CS 
dosing (Section 3.2.6), DORR is defined as the time from recapture of response (CR or PR) to 
the time of second PD or the date of death due to any cause (whichever occurs earlier).  
Detailed censoring rules are specified in Table 2 (FDA guidance [FDA 2015] and [FDA 2018]). 
In general, in primary analysis for DOR and analysis for DORR, subjects will be censored at the 
date of last adequate assessment with evidence of no progression by the IRRC, in the following 
situations: 
• No progression nor death 
• Discontinuation for any reason other than IRRC documented PD or death, including loss 
to follow-up, toxicity, withdrawal of consent, etc. 
• Start of new anti-lymphoma treatment without evidence of IRRC documented PD 
• Death or progression after two or more consecutive missed scheduled visits. 
Supportive analysis for DOR will be conducted to evaluate the robustness of the primary analysis 
result for DOR, by considering all progressions and deaths as events per European Medicines 
Agency (EMA) guidance (EMA 2013), regardless of whether they occurred after initiating next 
anti-lymphoma treatment, after 2 or more consecutive missed scheduled assessments, or being 
reported as clinical (non-IRRC reviewed radiographic) progression (Table 3). 
For subjects in IS dosing switching to the CS at PD, both DOR and DORR will be estimated. 


MEI Pharma, Inc. 
Statistical Analysis Plan
Protocol ME-401-003 
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CONFIDENTIAL 
 
 
Table 2: 
Censoring Rules for the Primary Analysis of DOR and DORR 
Situation 
Date of Event or Censoring 
Outcome 
Progression documented  
Date of earliest assessment which revealed progression 
determined by the IRRC 
Event 
Death before first PD assessment 
Date of death  
Event  
Death between adequate assessment visits  Date of death  
Event 
No progression, nor death 
 
Date of last adequate assessment with evidence of no 
progression by the IRRC  
Censored  
Discontinuation for any reason other than 
documented PD or death 
Date of last adequate assessment with evidence of no 
progression by the IRRC 
Censored  
Loss to follow-up 
Date of last adequate assessment with evidence of no 
progression by the IRRC 
Censored  
Switching back to CS dosing schedule 
without evidence of PD by IRRC prior to 
switching 
Date of last adequate assessment with evidence of no 
progression by the IRRC before switching back to the 
CS dosing schedule  
Censored  
 
New anti-lymphoma treatment started 
without evidence of PD by IRRC 
Date of last adequate assessment with evidence of no 
progression by the IRRC before the start of new anti-
lymphoma treatment  
Censored  
 
Death or progression after two or more 
consecutive missed scheduled visits  
Date of last adequate assessment with evidence of no 
progression by the IRRC  
Censored  
Table 3: 
Censoring Rules for the Supportive Analysis of DOR 
Situation 
Date of Event or Censoring 
Outcome 
Progression documented  
Date of earliest assessment which revealed progression 
determined by the IRRC 
Event 
Death before first PD assessment 
Date of death  
Event  
Death between adequate assessment visits  Date of death  
Event 
No progression, nor death 
 
Date of last adequate assessment with evidence of no 
progression by the IRRC  
Censored  
 
Treatment discontinuation for any reason 
other than documented PD or death 
Date of last adequate assessment with evidence of no 
progression by the IRRC 
Censored  
Loss to follow-up 
Date of last adequate assessment with evidence of no 
progression by the IRRC 
Censored  
Switching back to CS dosing schedule 
without evidence of PD by IRRC prior to 
switching * 
Date of earliest assessment which revealed progression 
determined by the IRRC or death after switching to CS 
Event 
 
New anti-lymphoma treatment started * 
Date of earliest assessment which revealed progression 
determined by the IRRC or death 
Event 
Death or progression after two or more 
consecutive missed scheduled visits * 
Date of earliest assessment which revealed progression 
determined by the IRRC or death 
Event  
Investigator claim of clinical progression 
leading to treatment discontinuation * 
Date of clinical progression 
Event 


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* Outcome definitions are different from primary analysis of DOR 
3.2.3. 
Complete Response (CR) Rate 
CR rate is defined as the proportion of subjects achieving the best response rating of CR based 
on the Lugano Classification, as determined by IRRC or Investigator. 
The primary analysis for CR will include CR prior to first PD. 
As a supportive analysis, CR will be summarized by including CR achieved overall. 
i.e, throughout the duration of the study for subjects in IS group switching to CS group.  
3.2.4. 
Progression-Free Survival (PFS) 
PFS is defined as time from first dose of study drug until first disease progression or death from 
any cause for all groups. Disease progression criteria are defined per Modified Lugano Response 
Criteria and will be assessed by IRRC and Investigator, independently. 
As supportive analysis, PFS2 will be defined as time from first dose of CS after switching until 
disease progression or death from any cause, and PFS3 will be defined as time from first dose of 
study drug until disease progression or death from any cause after switching back to the CS. 
PFS2 and PFS3 are only defined for patients in the ITT IS Population (Section 4.2) who 
switched back to the CS. 
The censoring rule for PFS will be the same as the ones for primary analysis for DOR (Table 3), 
with the exception that subjects with Incomplete or no baseline tumor assessments or no 
additional follow-up data obtained will be censored at first dose date. 
3.2.5. 
Time to Treatment Failure (TTF) 
Time to treatment failure is defined as time from the first dose of study drug to treatment failure. 
Treatment failure is defined as any treatment discontinuation due to PD, toxicity, or death. 
Censoring rule for TTF: 
• Subjects who are still alive and on treatment will be censored at the last disease 
assessment date indicating the absence of progression; 
• Subjects who have treatment discontinued for reasons other than PD, AE, or death, will 
be censored at the treatment discontinuation date. 
3.2.6. 
Recapture of Response 
Recapture of response is defined as achieving a second documented CR or PR after experiencing 
the first PD.  The CT scan at PD will be used as new baseline for assessing disease response to 
ME-401. 
Recapture of response rate (RRR) is defined as the proportion of subjects who achieved a CR or 
PR followed by a PD during IS dosing and subsequently switched to CS dosing and achieved a 
second documented CR or PR. 
Time to recapture of response is defined as the time from the first PD until the second 
documented CR or PR. Subjects who do not have the second documented CR or PR will be 
censored at last time of response assessment. 


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3.2.7. 
Overall Survival (OS) 
OS is defined as time from date of first dose of study drug until death from any cause. OS will be 
censored at the last date the subject is known to be alive when the confirmation of death is absent 
or unknown.  
3.3. 
Safety Endpoints 
3.3.1. 
Overall Incidence Rate of Treatment-Emergent Adverse Events (TEAEs) 
A TEAE is defined as any new or worsening adverse event occurred after first dose of study drug 
until 30 days after last dose of ME-401. 
The overall incidence rate of TEAEs is calculated as the proportion of subjects with at least one 
TEAE. 
The overall incidence rate of serious adverse events (SAEs) will also be calculated. 
3.3.2. 
Incidence Rate of Adverse Event of Special Interests (AESIs) 
The rate of AESIs is calculated as the proportion of subjects with at least one AESI. 
3.3.3. 
Time to Occurrence of Adverse Event of Special Interests (AESIs) 
Time to occurrence of AESIs is defined as the time from first dose of study drug to first 
occurrence of an AESI.  
Censoring rules for time to occurrence of AESIs: 
• Subjects without AESI who are still on treatment will be censored at the data cutoff date;  
• Subjects without AESI who discontinue from treatment will be censored at the treatment 
discontinuation date. 
3.3.4. 
Other Safety Endpoints   
Other safety endpoints include: 
• Laboratory test 
• Vital signs 
• Electrocardiogram (ECG)  
• Physical examination 
4. 
 ANALYSIS POPULATIONS AND ANALYSIS SETS 
4.1. 
Safety Population  
The Safety Population will include all FL and MZL subjects who received at least one dose of 
study drug. The safety population will be used for analyses of safety endpoints for the 
Final Analysis for the NDA submission.  


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4.2. 
Intent-to-Treat (ITT) Population  
The ITT FL population will include all FL subjects randomized per Protocol Amendment 1 
(two-arm design with randomization), and all enrolled FL subjects who received at least one 
dose of study drug per Protocol Amendment 2 or later amendment (single-arm, open-label 
design). 
The ITT IS FL Population is defined as all FL subjects who are intended to receive ME-401 IS 
regimen, either per Protocol Amendment 1 or per Protocol Amendment 2 or later amendments, 
including subjects who: 
• Were randomized to the ME-401 IS dosing regimen group (Group B) per 
Protocol Amendment 1 
• Were randomized to the ME-401 CS dosing regimen group (Group A) per Protocol 
Amendment 1, but re-consented to Protocol Amendment 2 (for IS dosing regimen) prior 
to or on their Cycle 3 visit date 
• Were enrolled per Protocol Amendment 2 or later amendment who received ME-401 IS 
dosing regimen. 
The ITT CS Group in the ITT FL Population is defined as all FL subjects who are randomized to 
the ME-401 CS regimen group (Group A) per Protocol Amendment 1, and did not switch to IS 
dosing regimen at Cycle 3 visit per Protocol Amendment 2. More specifically, this population 
includes subjects who were originally randomized to the ME-401 CS dosing regimen group 
(Group A) per Protocol Amendment 1, had their Cycle 3 visits prior to when the subjects 
re-consented to Protocol Amendment 2, or discontinued treatment without being re-consented 
under Protocol Amendment 2. 
The ITT MZL population is defined as all enrolled MZL subjects who received at least one dose 
of ME-401. 
Subjects’ original treatment assignments are documented in IVRS vendor’s randomization 
listings.  
• If the FL patients’ ORIGINAL_TREATMENT=”ME-401 60 mg 1 wk on/3 wks off” or 
“Open Label Treatment”, they will be included in ITT IS FL Population;    
• If the FL patient’s ORIGINAL_TREATMENT=”ME-401 60 mg daily”, and 
o If VISIT_TYPE=”Cycle 3 Day 1” and VISIT DATE >= the date when subjects 
re-consented to Protocol Amendment 2 from Inform Consent CRF data from 
EDC, they will be included in ITT IS FL Population; 
o If VISIT_TYPE=”Cycle 3 Day 1” and VISIT DATE < the date when subjects 
re-consented to Protocol Amendment 2 from Inform Consent CRF data from 
EDC, the subject will be included in ITT CS Population; 
o If patients were never consented to Protocol Amendment 2, they will be included 
in ITT CS Population. 


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The ITT FL population and ITT MZL population will be used for the analyses of disposition, 
baseline characteristics, and efficacy endpoints. The overall ITT population (including both ITT 
FL and ITT MZL populations) will be used for the presentation of subjects in all subject listings. 
To evaluate the impact of the covid-19 pandemic on ORR, a Modified ITT (mITT) Population is 
defined as all ITT FL or ITT MZL patients excluding who discontinued from treatment early due 
to covid-19 deaths with no post-baseline efficacy assessment. 
4.3. 
Efficacy Analysis Populations and Analysis Sets 
The primary efficacy population in FL (PEP FL) is defined as the initial 91 consecutive subjects 
enrolled in the ITT IS FL Population.  
The DOR Primary Analysis Set for FL will include subjects in the PEP FL who achieved PR or 
CR prior to first PD, that is reported before the data cutoff date for the FL Primary Analysis. 
The DOR Supportive Analysis Set for FL will include subjects in the ITT IS FL who achieved 
PR or CR prior to first PD, that is reported before the data cutoff date for the NDA Analysis for 
FL. 
The DOR Analysis Set for MZL will include subjects in the ITT MZL FL who achieved PR or 
CR prior to first PD, that is reported before the data cutoff date for the MZL Primary Analysis. 
The efficacy evaluable (EE) population will include all subjects who received at least one dose 
of study drug and had at least one response assessment post baseline.  
Subjects will be analyzed by randomized group per Protocol Amendment 1 and by enrolled 
group (IS dosing) per Protocol Amendments 2 and 3. EE population will be used for selected 
efficacy analyses. 
4.4. 
Pharmacokinetic (PK) Population   
The PK population will include all subjects who received at least one dose of ME-401 and who 
had evaluable PK data, i.e. quantifiable ME-401 concentrations. The PK population will be used 
for the analyses of PK endpoints. PK Analysis will be performed separately by central Lab 
(LGC). 
4.5. 
Per Protocol (PP) Population 
The per protocol population will include all subjects who received at least one dose of 
study drug, had at least one response assessment after Day 1, and did not have any major 
protocol deviation during the study. Subjects will be analyzed by randomized group per 
Protocol Amendment 1 and by enrolled group (IS dosing) per Protocol Amendments 2 and 3. 
PP population will be used for primary efficacy analyses. 
4.6. 
Protocol Deviations 
Study protocol deviations will be identified by the clinical team and assigned within the 
appropriate deviation category. All protocol deviations will be collected separately from the 
database, and graded as minor or major before the database lock. A summary table for number 


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and percentage of subjects with major protocol will be summarized by type of deviation and 
treatment group. A listing will be generated for protocol deviations. 
5. 
GENERAL ASPECTS FOR STATISTICAL ANALYSIS 
5.1. 
General Methods 
• All statistical analyses and summaries will be performed using SAS® 9.4 or higher. 
• Unless otherwise specified, safety summarized will be presented for all dosed patients by 
actual dose schedule (CS and IS). For IS group, the safety tables further have 3 
sub-columns: FL, MZL, and all IS. 
• Detailed types of analyses and corresponding populations for planned Primary Analysis 
and NDA Analysis for FL is shown in Table 4. 
• Unless otherwise specified, descriptive statistics for continuous data will include the 
number of subjects (n), mean, standard deviation (SD), minimum and maximum. For 
discrete variables, frequency count and percentages will be computed. Percentages based 
on frequency counts will be based on available data, and denominators will generally 
exclude missing values, unless otherwise stated. All percentages will be presented with 
one decimal, unless otherwise specified. Percentages equal to 100 will be presented as 
100, and percentages will not be presented for zero frequencies. 
• The same number of decimal places as in the raw data will be presented when reporting 
min and max, 1 more decimal place than in the raw data will be presented when reporting 
mean and median, and 2 more decimal places than in the raw data will be presented when 
reporting SD. If the raw data has 3 decimal places or more, 3 decimal places will be 
presented for mean, median, Q1/Q3, and SD. For continuous variables assessed at 
baseline and post-baseline, the change from baseline summary statistics will also be 
presented. 
• Subject listings of all data in the database will be provided, including data collected in the 
electronic case report form (eCRF) as well as data transferred electronically. 
Measurements from subjects excluded from the pre-defined analysis populations or extra 
measurements (such as unscheduled or repeat assessments) will not be included in 
summary tables unless specified otherwise, but will be included in the subject listings. 
Listings will be sorted by treatment groups, subject number, and visit/collection date/time 
if applicable, unless otherwise stated.  
• All fractional numeric values will be presented with a zero to the left of the decimal point 
(for example, 0.12 - 0.30, not .12 - .30). 
 


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Table 4: 
Types of Analyses and Corresponding Populations for Planned Primary Analysis and NDA Analysis for FL 
Planned Analysis 
Type of Analysis 
Population 
Analysis Set / Group 
Primary Analysis for FL  
Baseline disposition and demographics 
ITT IS FL 
ITT IS FL Total 
Primary endpoint ORR  
PEP FL1 
PEP FL 
Secondary endpoint DOR 
PEP FL 
DOR Primary Analysis Set for FL 
Selected Critical Secondary endpoints (PFS, OS) 
PEP FL 
PEP FL 
Selected Safety endpoints 
Safety IS FL population 
Safety IS FL Total 
NDA Analysis for FL 
Baseline disposition and demographics 
ITT FL 
PEP FL, ITT IS FL, ITT CS, ITT FL Total 
Primary endpoint ORR (Primary Analysis) and all 
secondary endpoints 
PEP FL1 
PEP FL 
Primary endpoint ORR (Supportive Analysis) 
PEP FL2  
PEP FL 
Primary endpoint ORR (Supportive Analysis) 
ITT FL3 
PEP FL, ITT IS FL, ITT CS, ITT FL Total 
Secondary endpoint DOR (Primary Analysis) 
PEP FL 
DOR Primary Analysis Set for FL 
Secondary endpoint DOR (Supportive Analysis) 
ITT IS FL 
DOR Supportive Analysis Set for FL 
Selected critical efficacy endpoints (PFS, OS, etc.) 
ITT FL  
PEP FL, ITT IS FL, ITT CS, ITT FL Total 
Safety endpoints 
Safety population 
IS FL, IS MZL4, IS Total, CS 
Primary Analysis and NDA 
Analysis for MZL 
Baseline disposition and demographics 
ITT MZL 
ITT MZL 
Primary endpoint ORR and all secondary endpoints 
(except DOR) 
ITT MZL 
ITT MZL 
Secondary endpoint DOR 
ITT MZL 
DOR Analysis Set for MZL 
Safety endpoints 
Safety population 
IS FL, IS MZL, IS Total, CS 
1 Primary analysis for ORR will include best response of PR and/or CR prior to first PD that is reported before the data cutoff date for FL Primary Analysis, 
among all patients in the Primary Efficacy Population in FL (PEP FL).  
2 Supportive analysis for ORR will include PR or CR achieved that is reported before the data cutoff date for NDA Analysis for FL, among all patients in the 
Primary Efficacy Population in FL. 
3 Supportive analysis for ORR will include PR or CR achieved that is reported before the data cutoff date for NDA Analysis for FL, among all patients in the 
ITT FL Population. 
4 Including MZL patients in the Safety Population who were dosed prior to the data cutoff date for the NDA Analysis for FL.


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5.2. 
Key Definitions 
5.2.1. 
Baseline 
Unless otherwise specified, baseline is defined as the last non-missing assessment prior to the 
first dose date of study drug and within 28 days of first dose date. In cases where measurements 
are taken on the same day as the first dose of study drug and no times are reported, it will be 
assumed that these measurements are taken prior to study drug being administered therefore can 
be defined as baseline measurements.   
5.2.2. 
Change from Baseline  
Change from baseline will be calculated as post-baseline value – baseline value. If either the 
baseline or post-baseline value is missing, the change from baseline is set to “missing”. 
Percentage change from baseline will be calculated as [(Post baseline value – Baseline 
value)/Baseline value] ×100. 
5.2.3. 
Study Day  
Study day will be calculated from first dose as:  
Study day = date of assessment – date of first dose +1 if the date of assessment is later than the 
date of first dose; Otherwise Study day = date of assessment – date of first dose. 
One cycle corresponds to 28-days, with the first day of treatment designated as Day 1. For 
subjects who are randomized/enrolled but not dosed, Day 1 will be the randomization/enrollment 
day. 
5.3. 
Missing Data  
Missing efficacy data will not be imputed. 
5.3.1. 
Handling of Missing Dates/Months/Years for All Adverse Events  
If the start/end date of an AE is partially missing, the date will be compared as far as possible 
with the first dose date of study drug. The AE will be assumed as treatment emergent if it cannot 
be definitively shown that the AE did not occur or worsen during the treatment-emergent period, 
from first dose of study drug to 30 days after last dose of study drug (worst case approach).  
• Start dates: 
o For missing start day only: Day will be imputed as the first day of the month (i.e., 01) 
with the following exception: if the partial date falls in the same month and year as 
first dose date, then the partial date will be imputed to equal first dose date. 
o For missing start day and month: Day and month will be imputed as the first day of 
the year (i.e., 01 January) with the following exception: if the partial date falls in the 
same year as first dose date, then the partial date will be imputed to equal first dose 
date. 
o For missing start day, month, and year: Date will be imputed to the first dose date.  


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o Imputed start dates must be prior to the discontinuation date or completion date.  
• Stop dates: 
o For missing stop day only: Day will be imputed as the last day of the month 
(i.e., 28, 29, 30, or 31). 
o For missing stop day and month: Day and month will be imputed as the last day of 
the year (i.e., 31 December). 
o For missing stop day, year, and month: Date will be imputed as date of 
discontinuation or completion.  
o Imputed dates should not extend beyond the date of completion or discontinuation. 
o Imputed stop dates must be on or after the start date. 
5.3.2. 
Handling of Missing Dates/Months/Years for Concomitant Medications   
Prior or concomitant medications with incomplete dates will be handled as follows for the sole 
purpose of determining whether a non-study medication is a concomitant medication. If the 
start/stop date of a medication is partially missing, the date will be compared as far as possible 
with the first dose date of study drug and the medication will be assumed as concomitant if it 
cannot be definitively shown that the stop date is before the start of administration of study drug, 
or the start date is after the last visit of the study. 
• If the start and stop dates are both completely missing, a medication will be considered 
concomitant. 
• Start date: 
o For missing start day only: Day will be imputed as the first day of the month 
(i.e., 01). 
o For missing start day and month: Day and month will be imputed as the first day of 
the year (i.e., 01 January). 
o Missing start dates will not be imputed.  
• Stop date: 
o For missing stop day only: Day will be imputed as the last day of the month 
(i.e., 28, 29, 30, or 31). 
o For missing stop day and month: Day and month will be imputed as the last day of 
the year (i.e., 31DEC). 
o Imputed stop dates must be on or after the start date. 
The original partial or missing date will be shown in listings of all non-study medications. 
5.4. 
Visit Windows 
For study assessment days, there is ± 3-day window allowable for each clinic visit during 
Cycle 1. For Cycles 2 through 25, the allowable window is ± 7 days. After Cycle 25, the 
allowable window is ± 1 month for each clinic visit and its associated assessments. 


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There is no plan to re-assign visits based on actual visit dates. The analysis will be based nominal 
visit/time points collected.  
5.5. 
Subgroups 
• Tumor bulk (largest lymphoid mass <5 cm vs. ≥5 cm)  
• Tumor bulk (largest lymphoid mass <7 cm vs. ≥7 cm)  
• Prior use of lenalidomide in combination of rituximab (yes vs. no) 
• Disease response to last therapy (relapse vs. refractory) 
• Disease response to prior anti-CD monoclonal antibodies (relapse vs. refractory) 
• Disease response to prior alkylating agents (relapse vs. refractory) 
• Disease response to prior bendamustine (relapse vs. refractory) 
• Disease response to prior immunochemotherapy (relapse vs. refractory) 
• Number of prior lines of systemic cancer treatments (2 vs. >2) 
• Number of prior lines of chemo regimens (1, 2-3, 4+) 
• PD within 24 months of first line immunochemotherapy (POD24) (yes vs. no) 
• Age (<65 vs. ≥65) 
• Sex (Male vs. Female) 
• Region (North America, Europe, Asia-Pacific) 
• Race (Caucasian, Black, Asian, Other) 
• Baseline Eastern Cooperative Oncology Group (ECOG) (0 vs. ≥1) 
• Disease stage at diagnosis (1-2 vs 3-4) 
• Disease stage at study enrollment (1-2 vs 3-4) 
• Duration of treatment-free interval from the last lymphoma-directed therapy: ≤6 months, 
>6 months 
• Baseline lactate dehydrogenase (LDH) (normal vs abnormal) 
• History of bone marrow involvement (yes vs. no) 
• Extra-nodal disease (yes vs. no) 
6. 
DEMOGRAPHIC, OTHER BASELINE CHARACTERISTICS 
AND MEDICATION 
6.1. 
Subject Eligibility, Disposition and Protocol Deviations 
The subject disposition table will summarize the number of subjects screened, the number and 
percentage of subjects in each analysis set, the number and percentage of subjects who 


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discontinued treatment, along with reasons of treatment discontinuation, and the number and 
percentage of subjects who discontinued study, along with reasons of study discontinuation. 
The summary tables will be generated separately for all subjects, ITT FL Population, and 
ITT MZL Population.  
Separate by-subject listings will be provided for subject disposition, eligibility, analysis 
populations, and protocol deviations. 
Subject follow-up time (months) on study will be provided in disposition summaries. It is 
defined as (date of data cutoff – date of first dose + 1) / 30.4375 for patients remaining on study, 
and (End of Study date – date of first dose + 1) / 30.4375 for patients who discontinued from 
study. 
6.2. 
Demographic and Other Baseline Characteristics 
Descriptive statistics will be presented for continuous variables including age, height, weight, 
body mass index (BMI) at baseline. The frequency and percentage of subjects will be tabulated 
for categorical variables including age group (<65 vs. ≥65), sex, child-bearing potential, race, 
ethnicity, and region of enrollment at baseline. The summary tables will be produced separately 
for Safety Population, ITT FL Population, and ITT MZL Population. 
All demographic data at baseline will be listed. 
6.3. 
Baseline Disease Characteristics  
The following baseline disease characteristics will be summarized by treatment group and 
overall for Safety Population, ITT FL Population, and ITT MZL Population. 
• Tumor bulk (largest lymphoid mass <5 cm vs. ≥5 cm, and 7 cm vs. ≥7 cm)  
• Disease response to last therapy (relapse vs. refractory) 
• Disease response to specific prior cancer therapies (relapse vs. refractory) 
o Anti-CD20 monoclonal antibodies  
o Alkylating agents 
o Bendamustine  
o Immunochemotherapies 
• Stage at diagnosis (I, II, III, IV), stage at study entry (I, II, III, IV), and grade at study 
entry (1, 2, 3a) 
• POD24 (yes vs. no) 
• Baseline ECOG (0 vs. ≥1) 
• Duration of treatment-free interval from the last lymphoma-directed therapy: ≤6 months, 
>6 months 
• Baseline LDH (normal vs abnormal) 


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Disease response to prior therapy is defined as follows:  
• Relapsed disease: disease progression after a response (CR or PR) lasting ≥6 months 
6.4. 
Refractory Disease: No Response to Therapy (no CR or PR) or 
Response Lasting <6 months Medical History and Concomitant 
Diseases 
Medical History will be coded into system organ class (SOC) and preferred term (PT) by 
Medical Dictionary for Regulatory Activities (MedDRA) Version 21.1 or later. Medical history 
will be listed but not summarized.  
Medical history data listings will be produced. 
6.5. 
Prior and Concomitant Medications  
All prior and concomitant medications and therapies will be recorded from 28 days prior to 
Cycle 1 Day 1 through 30 days after last dose of ME-401, treatment discontinuation, or until a 
subsequent anti-cancer therapy is initiated. Medications will be coded by World Health 
Organization Drug Dictionary (WHO DD) Version Global B3 Sep2018 or later. 
Prior medications are medications which stop before the start of study drug. Concomitant 
medications are medications which start or stop on or after the start of study drug.  
For the classification of prior medications vs. concomitant medications, incomplete start or stop 
dates of medications will be imputed using the algorithm in Section 5.3.2. The medication will 
be assumed as concomitant if it cannot be definitively shown that the stop date is before the first 
dose date of study drug.  
Prior and concomitant medications will be coded into Anatomical Therapeutic Chemical (ATC) 
classification levels and preferred term.  All prior and concomitant medications will be listed but 
not summarized. 
6.6. 
Prior Therapies 
The following prior therapies will be summarized using the number and percentage of subjects 
with each therapy type using Safety Population. 
• Prior cancer surgery for follicular lymphoma 
• Prior radiotherapy 
• Prior transplant 
• Prior Cancer therapy 
o Anti-CD20 monoclonal antibodies  
o Alkylating agents 
o Bendamustine  
o Immunochemotherapies 
o Lenalidomide in combination of rituximab 


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• Number of prior lines of cancer therapies (2 vs. >2) 
• Number of prior lines of chemo regimens (1, 2-3, 4+) 
The data of prior therapies will also be listed. 
6.7. 
Concomitant Procedures 
The concomitant medical procedures will be listed but not summarized. 
7. 
EFFICACY ANALYSIS 
7.1. 
Primary Efficacy Endpoint and Analysis 
The primary efficacy endpoint is ORR, defined as the proportion of subjects achieving the best 
response rating of CR or PR based on the Modified Lugano Classification, assessed by IRRC. 
For Primary Analysis and NDA Analysis for FL, ORR will be evaluated by summarizing the 
number and percentage of subjects with the best overall response (CR/PR) prior to first PD as 
assessed by IRRC that is reported before the data cutoff date for the Primary Analysis, along 
with 2-sided 95% exact Clopper-Pearson confidence interval (CI). The primary efficacy analysis 
will be based on the Primary Efficacy Population for FL (PEP FL).  
As a supportive analysis for NDA Analysis for FL, ORR will be analyzed 
• using the ITT FL Population by including the PEP FL, ITT IS FL, ITT CS, ITT FL Total 
patients 
• by summarizing the number (percentage) of patients who achieved PR or CR before the 
data cutoff date for the NDA Analysis for FL submission. 
As sensitivity analyses, the above analyses in Section 7.1 will be repeated for EE population and 
PP population. 
All subjects who did not meet the criteria for an objective response by the analysis cut-off date 
will be considered non-responders.  
To evaluate the impact of the covid-19 pandemic on efficacy of ME-401, the above analyses in 
Section 7.1 will also be conducted using the Modified ITT Population in IS FL patients, 
i.e., patients who discontinued from treatment early due to covid-19 deaths with no post-baseline 
efficacy assessment will be excluded from the analysis. 
Subgroup analysis of ORR (the proportion of subjects with the best overall response [CR/PR] 
prior to first PD as assessed by IRRC), that is reported before the data cutoff date for the Primary 
Analysis, will be performed by the subgroups defined in Section 5.5, based on PEP FL and 
ITT MZL population. Forest plots will be provided for ORR and 95% CI in selected subgroups. 
Waterfall plots of maximum percentage decrease in sum of the products of longest diameters 
(SPD) measured by the IRRC will be provided for the patients in the PEP FL and ITT MZL 
population. 


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7.2. 
Secondary Efficacy Endpoints and Analyses 
7.2.1. 
Objective Response Rate (ORR) by Investigator  
Secondary analysis of the ORR, as determined by the investigator, will be repeated as described 
above for the analyses of primary efficacy endpoint in Section 7.1.    
The concordance in best overall response between IRRC and Investigator assessments will be 
summarized. 
Waterfall plots of maximum percentage decrease in sum of the products of longest diameters 
(SPD) measured by the investigator will be provided for the patients in the PEP FL and 
ITT MZL population. 
7.2.2. 
Duration of Response (DOR) and Duration of Recaptured Response (DORR) 
DOR is the time from first response (CR or PR) to the time of first PD or the date of death due to 
any cause (whichever occurs earlier).  
For Primary Analysis and NDA Analysis for FL, DOR will be analyzed using the DOR Primary 
Analysis Set for FL.  
As a supportive analysis for NDA Analysis for FL, DOR will be analyzed using the DOR 
Supportive Analysis Set. 
For the Primary and NDA Analysis for MZL, DOR will be analyzed using the DOR Analysis Set 
for MZL. 
Subgroup analysis of DOR using the DOR Primary Analysis Set for FL and the DOR Analysis 
Set for MZL will be performed by the subgroups defined in Section 5.5. 
DORR is the time from recapture of response (CR or PR) to the time of second PD or the date of 
death due to any cause (whichever occurs earlier).  
The assessment of DOR and DORR assessed by IRRC and by Investigator will be summarized 
separately.  
DOR and DORR will be summarized using Kaplan-Meier (KM) summary statistics. The min, 
max, median duration of response with 95% CI (based on the LOGLOG transformation), number 
of events, number censored with types of censoring and, KM plot for cumulative probability of 
DOR and DORR over time will be presented. DOR and DORR will be reported in months. Only 
subjects who had a response will be included in this summary table, and the denominator will be 
all subjects in each population who had a documented CR or PR response. Estimates and 95% 
CIs for DOR rates at 3 months, 6 months, 9 months and 12 months, and, median with 95% CI of 
the follow-up time (months) based on reverse KM estimate of the DOR time (by reversing the 
censoring/event status) will be summarized.    
KM estimates of DOR will be provided by best response type (CR vs PR). 
Forest plots will be provided for median DOR and 95% CI in different subgroups. 


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7.2.3. 
Complete Response (CR) Rate 
The CR rate is proportion of subjects achieving the best response rating of CR, and will be 
assessed separately by IRRC and investigator.  
For FL Primary Analysis and Final Analysis, the CR rate will include CR prior to first PD that is 
reported before the data cutoff date for Primary Analysis.  
As a supportive analysis in Final Analysis, CR rate will be summarized by including CR 
achieved that is reported before the data cutoff date for NDA Analysis for FL. 
For CR rate, the number and percent (%) of subjects with 2-sided 95% exact Clopper-Pearson 
CIs will be summarized for subjects in PEP FL, ITT MZL population, ITT FL population 
(for NDA Analysis for FL only), EE population, and PP Population.  
7.2.4. 
Progression Free Survival (PFS) 
PFS is the time from first dose of study drug until first disease progression or death from any 
cause. The analysis of PFS will be performed based on the assessment by IRRC and investigator, 
separately.  
PFS will be summarized using Kaplan-Meier summary statistics for PEP FL, ITT MZL 
population, and ITT FL population (for NDA Analysis for FL only). The min, max, and median 
PFS with 95% CI (based on the LOGLOG transformation), number of events, number censored 
with types of censoring, and KM plot for cumulative probability of PFS over time will be 
presented. PFS will be reported in months. Estimate and 95% CI for PFS rates at 6 months, 
12 months, 18 months and 24 months, and median with 95% CI of the follow-up time (months) 
based reverse KM estimate of the PFS time (by reversing the censoring/event status) will be 
summarized.    
KM estimates of PFS will be provided by best response type (CR, PR and non-responders). 
7.2.5. 
Time to Treatment Failure (TTF)  
TTF is the time from the first dose of study drug to treatment failure (any treatment 
discontinuation due to PD, toxicity, or death). The analysis of TTF will be performed based on 
the assessment by IRRC and investigator, separately. 
TTF will be summarized using Kaplan-Meier summary statistics for PEP FL and ITT MZL 
population. The median TTF with 95% CIs (Brookmeyer and Crowley method), number of 
events, number censored, and KM plot of proportion of subjects without treatment failure over 
time will be presented. TTF will be reported in months. Estimate and 95% CI for 1-year TTF 
rate, and reverse KM estimate median with 95% CI for follow-up time (months) will be 
summarized.    
7.2.6. 
Recapture of Response Rate (RRR) 
RRR is the proportion of subjects who achieved a CR or PR followed by a PD during IS dosing 
and subsequently switched to CS dosing and achieved a second documented CR or PR. The 
analysis of RRR will be performed based on the assessment by IRRC and investigator, 
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For RRR, the number and percent (%) of subjects with 2-sided 95% exact Clopper-Pearson CIs 
will be summarized for subjects who achieved a CR or PR followed by a PD during IS dosing 
and subsequently switched to CS dosing, in PEP FL and ITT MZL population.  
7.2.7. 
Time to Recapture of Response  
Time to recapture of response is the time from the first PD until the second documented CR or 
PR. The analysis of time to recapture of response will be performed based on the assessment by 
IRRC and investigator, separately. 
Time to recapture of response will be summarized using Kaplan-Meier summary statistics only 
for subjects who achieved a CR or PR followed by a PD during IS dosing and subsequently 
switched to the CS dosing, in PEP FL and ITT MZL population. The median time to recapture of 
response with 95% CI (Brookmeyer and Crowley method), number of events, number censored 
and, and KM plot of proportion of subjects without recapture of response over time will be 
presented. Time to recapture of response will be reported in months. Estimate and 95% CI for 
1-year rate of time to recapture of response, and reverse KM estimate median with 95% CI for 
follow-up time (months) will be summarized.    
7.2.8. 
Overall Survival (OS)  
OS is the time from date of first dose of study drug until death from any cause, and will be 
summarized by Kaplan-Meier summary statistics for PEP-FL, ITT MZL population, ITT FL 
population (for NDA Analysis for FL only).  
Kaplan-Meier plots of OS will be presented. Summaries of the number and percentage of 
subjects who had died, were still in survival follow-up, were lost to follow-up and had 
withdrawn consent will be provided along with the median OS times and 95% CIs (Brookmeyer 
and Crowley method). Overall survival will be reported in months. Estimate and 95% CI for 
1-year OS rate, and reverse KM estimate median with 95% CI for follow-up time (months) will 
be summarized.    
8. 
SAFETY ANALYSES 
Safety analyses will be presented and summarized in the safety population for the Primary 
Analysis for FL and NDA Analysis for FL, and include overall IS group, IS FL, MZL, and 
CS group.  
Safety IS FL population will be used to analyze safety endpoints for the FL Primary Analysis. 
8.1. 
Extent of Exposure 
Exposure to ME-401 will be evaluated by duration of study drug exposure (months), actual dose 
intensity, and relative dose intensity for the safety population. 
 Descriptive statistics will be presented for continuous exposure variables: duration of exposure, 
actual dose intensity, and relative dose intensity. The number and percentage of subjects with 
dose schedule modified or dose interrupted due to AE or progressive disease will be 
summarized. The number and percentage of subjects with dose missed will be summarized by 


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reason. For subjects on study drug at the time of the ORR analysis, the data cut-off (DCO) date 
will be used to calculate exposure. 
• Duration of exposure (months) = (Date of last non-zero dose – Date of first non-zero dose 
+1) /30.4375.  
• Duration of exposure on the CS (months) 
o = (Date of last non-zero CS dose – Date of first non-zero CS dose +1) /30.4375 for 
subjects who never switched dosing regimen; or 
o = sum of (Date of last non-zero CS dose – Date of first non-zero CS dose +1) 
/30.4375 from each CS dosing interval for subjects who switched dosing regimens; 
date of first or last CS dose can be derived based on the switching date. 
• Duration of exposure on the IS (months) 
o = (Date of last zero/non-zero IS dose – Date of first non-zero IS dose +1) /30.4375 
• Actual total dose exposed (mg) is defined as the sum of ‘total mg’ of study drug received.   
• Total dose intended (mg) is defined as the sum of ‘total mg’ of study drug planned.  
o Total dose intended for the CS is estimated to be 60 mg x Duration of exposure on the 
CS (months) x 30.4375 
o Total dose intended for the IS is estimated to be (60 mg /4) x Duration of exposure on 
the IS (months) x 30.4375 
• Actual dose intensity (mg/month) = 30.4375 x Actual total dose exposed (mg) ÷ [(Date of 
last dose, zero or non-zero dose of study drug) – (Date of first non-zero dose of study 
drug) + 1] 
• Intended dose intensity (mg/month) = 30.4375 x Total dose intended (mg) ÷ [(Date of 
last dose, zero or non-zero dose of study drug) – (Date of first non-zero dose of study 
drug) + 1] 
• Relative dose intensity (RDI) (%) is the percentage of the actual dose intensity delivered 
relative to the intended dose intensity through to study drug discontinuation. RDI (%) = 
(actual dose intensity) ÷ (intended dose intensity) x 100 
8.2. 
Treatment Compliance  
The study drug compliance (%) will be calculated as: 
• Overall Compliance (%) = [total dose exposed (mg) / total dose intended (mg)] x 100 
• Compliance (%) on the CS = [Total dose exposed (mg) on the CS / total dose intended 
(mg) on the CS] x 100 
• Compliance (%) on the IS = [Total dose exposed (mg) on the IS / total dose intended 
(mg) on the IS] x 100 
For subjects on study drug at the time of the ORR analysis, the data cut-off (DCO) date will be 
used to calculate compliance.  


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8.3. 
Adverse Events and Adverse Events of Special Interest  
Adverse events (AEs) will be captured from the first dose of study drug and continue until 
30 days after the last dose of study drug. All AEs will be classified to SOC and PT by 
MedDRA Version 21.1 or later.  
The Investigator will assess AEs for severity and relationship to study drug and determine if an 
AE meets the criteria for a SAE. The relationship of an AE to study drug is to be classified as 
‘Related’, ‘Possibly Related’, or ‘Not Related’ by investigators. The study drug related AEs 
include both ‘Related’ and ‘Possibly Related’ AEs. Each AE will be graded using National 
Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0. For the 
purpose of the summaries, AEs with missing relatedness will be considered to be related 
ME-401. AEs with missing severity (CTCAE grade) will be considered grade 3 (severe). 
All AE listings will include the date of onset, date of resolution (if AE is resolved) and 
investigator’s assessment of severity and relationship to study drug. Missing (partial) start/stop 
dates will appear as missing (partial) in the subject data listings, but will be imputed to permit 
proper tabulation of AE data (See Section 5.3.1). 
AEs will be summarized descriptively by number and percentage (%) of subjects with an event 
(n (%)), and number of events [e], by MedDRA SOC/PT and CTCAE grade.  
A subject will be counted once at the SOC level and once at PT level. For summaries by SOC, 
PT, and intensity, a subject will be counted only at the maximum intensity level. Summaries by 
relatedness would be handled similarly to the summaries by intensity. Summaries by SOC and 
PT will be ordered by overall descending frequency of SOC and then, within a SOC, by overall 
descending frequency of PT. Summaries of PT will be ordered in descending frequency of PT.  
8.3.1. 
Treatment-Emergent Adverse Event (TEAE) 
TEAE is any new or worsening AE that started after the first dose of study drug until 30 days 
after the last dose of study drug. Treatment emergence will be determined by comparing the AE 
onset date with first dose date. If AE onset date is incomplete, treatment emergence will be 
imputed according to the algorithm in Section 5.3.1. In general, only TEAEs will be included in 
the AE summaries but all AEs will be included in the listings. 
8.3.2. 
Adverse Events of Special Interest (AESI) 
AESIs are events of scientific and medical interest specific to the further understanding of the 
ME-401 safety profile. For ME-401, AESIs will comprise of (see study protocol Section 5.3.2): 
• Cutaneous reactions, including skin rash Grade ≥2  
• Oral mucositis or stomatitis Grade ≥2  
• Diarrhea/colitis Grade ≥2  
• AST/ALT Grade ≥2  
• Non-infectious pneumonitis (NIP) of any grade 
• Lung infection/pneumonia Grade ≥2 
• Cardiomyopathy of any grade 


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AESI will be summarized by selected subgroups. 
8.3.3. 
Summaries of Incidence of Adverse Events  
The number and percentage of subjects reporting TEAEs and AESIs will be summarized as 
below: 
• Overall summary of TEAEs and AESIs 
• TEAEs by SOC and PT 
• TEAEs by PT  
• TEAEs of Grade 3 and above by PT  
• TEAEs by maximum grade and PT  
• TEAEs leading to treatment discontinuation by PT  
• TEAEs related to ME-401 leading to treatment discontinuation by PT  
• TEAEs leading to treatment interruption by PT 
• TEAEs related to study drug by PT 
• AEs leading to death by PT  
• Treatment-related AEs leading to death by PT  
• Treatment-Emergent SAEs by SOC and PT  
• Treatment-Emergent SAEs by PT  
• Treatment-Emergent SAEs by maximum grade and PT 
• Treatment-emergent AESIs by AESI Type and PT  
• Treatment-emergent Grade 3 and Above AESIs by AESI type and PT 
• Covid-19 events by grade 
• Other important safety events including opportunistic infection, CMV infection, 
PJP infection, and fungal infection, cytopenia event, pulmonary event by maximum grade 
and PT 
Selected analysis on TEAEs will be summarized by subgroups. 
Listings will be produced for all AEs, TEAEs, SAEs, AEs leading to treatment discontinuation, 
AEs leading to death, AESI, and for subjects with grade 3 and above AEs. 
8.3.4. 
Analysis of Time to Occurrence of AESIs 
As primary analysis, time to occurrence of AESIs is the time from first dose of study drug to first 
occurrence of AESI. 
As a supportive analysis, time to first occurrence of Grade 3 and above AESIs will be analyzed 
similarly.   


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Kaplan-Meier summary statistics median time to occurrence of AESI and 95% CI (Brookmeyer 
and Crowley method), number of events, number censored and, KM figures will be presented by 
dosing group. Time to occurrence of AESI will be reported in weeks. A separate listing of time 
to occurrence of AESI will be created.  Reverse KM estimate median with 95% CI for follow-up 
time (months) will be summarized.    
8.4. 
 Laboratory Evaluations 
Lab evaluations will include hematology (including coagulation), serum chemistry, urinalysis, 
pregnancy test, HIV testing, Hepatitis B and Hepatitis C testing, bone marrow aspiration/biopsy, 
PCR tests for CMV and opportunistic infection (OI) monitoring.  
For statistical and graphical summaries of the laboratory tests, values below or above the limit of 
detection (e.g., ‘< 3’ or ‘>500’) are substituted with the lower limit of detection minus 1% for 
values below the lower limit and are substituted with the upper limit of detection plus 1% for 
values above the upper limit (e.g., ‘< 3’ is substituted by ‘2.97’, ‘> 500’ is substituted by ‘505’). 
In data listings, the values are shown including the < or > sign.  
Results from the hematology, chemistry, and urinalysis will be summarized by visit using 
descriptive statistics, including the observed values and change from baseline values for numeric 
parameters and distribution of categories for categorical parameters. Boxplots by visit will be 
generated for selected hematology/chemistry parameters. 
In addition, the incidence rate of grade 3 and above toxicity for selected hematology and 
chemistry parameters will be summarized by visit. Separate listings will be produced for subjects 
with grade 3 and above toxicity for selected hematology and chemistry parameters. 
Treatment-emergent toxicities are defined as the worsening of lab toxicity grade comparing to 
the baseline. The incidence rate of treatment-emergent grade 3 and above toxicity for 
hematology and chemistry parameters will be summarized by visit.  
By-subject listings of hematology, serum chemistry, and urinalysis tests, including lab normal 
ranges, will be provided. Derived CTCAE grades will be included in listings, as applicable.  
The following lab data will be listed only: pregnancy test, HIV, Hepatitis B and Hepatitis C tests, 
bone marrow aspiration/biopsy, CMV titers, and OI monitoring. 
8.5. 
Vital Signs 
Vital sign measurements will include the measurement of height, weight, and systolic and 
diastolic blood pressure, heart rate, and body temperature.  
Results from each parameter will be summarized by visit using descriptive statistics, including 
the observed values and changes from baseline.  
All vital signs data will be listed. 
8.6. 
Cardiac Evaluations  
12-lead ECGs parameters including hear rate, RR interval, PR interval, QRS interval and QTcF 
will be assessed. For each parameter, the observed values and change from baseline will be 
summarized by visit. 


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The overall ECGs interpretations will be collected as ‘Normal’, ‘Abnormal, not clinically 
significant’ and ‘Abnormal, clinically significant. Shift tables from baseline will be produced 
based on these categories for each post-baseline. In addition, the incidence rate of grade 3 and 
above QT prolongation will be summarized by visit. 
All 12-lead ECG data will be listed. In addition, the results of echocardiogram will be listed.  
8.7. 
Physical Examination 
The physical examination will include examination of all organ systems. A symptom-directed 
exam will be performed only if needed/indicated, based on interim history. Skin assessments will 
be required for subjects who developed a rash. 
Listings will be produced for physical examination and skin assessments. 
8.8. 
Eastern Co-operative Oncology Group (ECOG) 
ECOG performance status data will be summarized by visit, and listed. 
9. 
INTERIM ANALYSES 
No formal interim analysis is planned for this study. 
An independent DSMB (Data Safety and Monitoring Board) will review descriptive summaries 
of accumulating safety, subject disposition and limited efficacy data every 6 months. Further 
description of the DSMB analyses are included in the DSMB charter. 
10. 
CHANGES FROM ANALYSIS PLANNED IN PROTOCOL 
No changes from planned analysis in protocol. 


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11. 
REFERENCE LIST 
Cheson BD, Fisher RI, Barrington SF, Cavalli F, Schwartz LH, Zucca E, et al. 
Recommendations for Initial Evaluation, Staging, and Response Assessment of Hodgkin and 
Non-Hodgkin Lymphoma: The Lugano Classification. J Clin Oncol. 2014;32(27):3059–67. 
EMA (2013) Appendix 1 to the guideline on the evaluation of anticancer medicinal products in 
man (CHMP/EWP/205/95 Rev.4): methodological considerations for using progression-free 
survival (PFS) or disease-free survival (DFS) in confirmatory trials. 
EMA/CHMP/27994/2008/Rev.1. 
FDA 2015 Guidance for Industry: Clinical Trial Endpoints for the Approval of Non-Small Cell 
Lung Cancer Drugs and Biologics. 
FDA 2018 Guidance for Industry: Clinical Trial Endpoints for the Approval of Cancer Drugs and 
Biologics. 


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12. 
PROGRAMMING CONSIDERATIONS 
12.1. 
General Considerations 
• One SAS program can create several outputs. 
• One output file can contain several outputs. 
• Output files will be delivered in Word format (.rtf) which enable pasting to Word 
document with minimal formatting required / portable document format (.pdf). 
• Numbering of TLFs will follow International Conference on Harmonization (ICH) 
E3 guidance. 
12.2. 
Table, Listing, and Figure Format 
12.2.1. 
General 
• All TLFs will be produced in landscape format, unless otherwise specified.  
• All TLFs will be produced using the Courier New font or Times New Roman, size 9. 
• The data displays for all TLFs will have a minimum blank 1-inch margin on all 4 sides. 
• Headers and footers for figures will be in Courier New font or Times New Roman, size 9. 
• Legends will be used for all figures with more than 1 variable, group, or item displayed. 
• TLFs will be in black and white (no color), unless otherwise specified. 
• Specialized text styles, such as bolding, italics, borders, shading, and superscripted and 
subscripted text, will not be used in the TLFs, unless otherwise specified.  
• Only standard keyboard characters will be used in the TLFs. Special characters, such as 
non-printable control characters, printer-specific, or font-specific characters, will not be 
used. Hexadecimal-derived characters will be used, where possible, if they are 
appropriate to help display math symbols (e.g., μ). Certain subscripts and superscripts 
(e.g., cm2, Cmax) will be employed on a case-by-case basis. 
• Mixed case will be used for all titles, footnotes, column headers, and programmer-
supplied formats, as appropriate. 
• Tables and listing should not have vertical grid lines or borders, but only horizontal lines 
on the header rows and bottom row. 
12.2.2. 
Headers 
• All output should have the following header at the top left of each page: 
MEI Pharma; Protocol No.: ME-401-003  
Draft/Final Run 
• All output should have Page n of N at the top or bottom right corner of each page. TLFs 
are internally paginated in relation to the total length (i.e., the page number should appear 
sequentially as page n of N, where N is the total number of pages in the table). 


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• The date output was generated should appear along with the program name as a footer on 
each page. 
12.2.3. 
Display Titles 
• Each TLF is identified by the designation and a numeral. (i.e., Table 14.1.1). The title is 
centered. The analysis set are identified on the line immediately following the title. The 
title and table designation are single spaced. A solid line spanning the margins will 
separate the display titles from the column headers. There will be 1 blank line between 
the last title and the solid line. 
Table x.y.z 
First Line of Title 
Second Line of Title if Needed 
(ITT Analysis Set) 
12.2.4. 
Column Headers 
• Column headings are displayed immediately below the solid line described above in 
initial upper-case characters.  
• Analysis set sizes will be presented for each treatment group in the column heading as 
(N=xx) (or in the row headings, if applicable). This is distinct from the ‘n’ used for the 
descriptive statistics representing the number of subjects in the analysis set. 
• The order of treatments in the tables and listings will be Placebo first in the case of 
placebo controlled studies and Active comparators first in the case of active comparator 
trials, followed by a total column (if applicable). 
12.2.5. 
Body of the Data Display 
12.2.5.1. General Conventions 
Data in columns of a table or listing are formatted as follows: 
• Alphanumeric values are left-justified; 
• Whole numbers (e.g., counts) are right-justified; and 
• Numbers containing fractional portions are decimal aligned. 
12.2.5.2. Table Conventions 
• Units will be included where available 
• If the categories of a parameter are ordered, then all categories between the maximum 
and minimum category are presented in the table, even if n=0 for all treatment groups in a 
given category that is between the minimum and maximum level for that parameter. 
If the categories are not ordered (e.g., Medical History, Reasons for Discontinuation from 
the Study, etc.), then only those categories for which there is at least 1 subject represented 
in 1 or more groups are included. 


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• P-values are output in the format: “0.xxx”, where xxx is the value rounded to 3 decimal 
places. Every p-value less than 0.001 will be presented as <0.001. If the p-value is 
returned as >0.999, then present as >0.999 
• Unless otherwise noted, for all percentages, the number of subjects in the analysis set for 
the treatment group will be the denominator.  
• Where a category with a subheading (such as system organ class) has to be split over 
more than one page, output the subheading followed by “(count)” at the top of each 
subsequent page.  The overall summary statistics for the subheading should only be 
output on the first relevant page. 
12.2.5.3. Listing Conventions 
• Missing data are represented on subject listings as either a hyphen (“-”) with a 
corresponding footnote (“- = unknown or not evaluated”), or as “N/A”, with the footnote 
“N/A = not applicable”, whichever is appropriate. 
• Dates are printed in SAS DATE9.format (“ddMMMyyyy”: 01JUL2000). Missing 
portions of dates are represented on subject listings as dashes (--JUL2000). Dates that are 
missing because they are not applicable for the subject are output as “N/A”, unless 
otherwise specified. 
• All observed time values are to be presented using a 24-hour clock HH:MM or 
HH:MM:SS format (e.g., 11:26:45, or 11:26). Time will only be reported if it was 
measured as part of the study. 
• Units will be included where available. 
12.2.6. 
Footnotes 
• A solid line spanning the margins will separate the body of the data display from the 
footnotes.  
• All footnotes will be left justified with single-line spacing immediately below the solid 
line underneath the data display. 
• Footnotes should always begin with “Note:” if an informational footnote, or 1, 2, 3, 
etc. if a reference footnote. Each new footnote should start on a new line, where possible. 
• Subject specific footnotes are avoided, where possible. 
• Footnotes will be used sparingly and add value to the table, figure, or listing. If more than 
six lines of footnotes are planned, then a cover page is strongly recommended to be used 
to display footnotes, and only those essential to comprehension of the data will be 
repeated on each page.  
• The last line of the footnote section will be a standard source line that indicates the name 
of the program used to produce the data display and date the program was run 
(i.e., ‘Program : xxx.sas   Table Generation: DDMMMYYYY’). 


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13. 
QUALITY CONTROL 
SAS programs are developed to produce output such as analysis data sets, summary tables, data 
listings, figures or statistical analyses. An overview of the development of programs is detailed 
in Syneos Health SOP Developing Statistical Programs (3907). 
Syneos Health SOPs Developing Statistical Programs (3907) and Conducting the Transfer of 
Biostatistical Deliverables (3908) describes the quality control procedures that are performed for 
all SAS programs and output. Quality control is defined here as the operational techniques and 
activities undertaken to verify that the SAS programs produce the output by checking for their 
logic, efficiency and commenting and by review of the produced output.