Initial Phase 1 Study of WT2725 in Patients With Advanced Malignancies
Phase: PHASE1
Conditions: Cancer
Sponsor: Sumitomo Pharma America, Inc.

 
 
Title page
 
 
WT2725 DOSING EMULSION 
PROTOCOL D8350004 
 
INITIAL PHASE 1 STUDY OF WT2725 DOSING 
EMULSION IN PATIENTS WITH ADVANCED 
MALIGNANCIES 
 
IND No. 15,065 
 
 
Version 7.0 
3 Dec 2014 
Incorporates Amendment 6 
 
 
SUNOVION PHARMACEUTICALS INC. 
84 Waterford Drive 
Marlborough, MA 01752 
USA 
(508) 481-6700 
 
 

Protocol D8350004 Version 7.0 
 
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RESTRICTED DISTRIBUTION OF PROTOCOLS 
This protocol and the data gathered during the conduct of this protocol, contains information that 
is confidential and/or of proprietary interest to Sumitomo Dainippon Pharma Co. Ltd. and/or 
Sunovion Pharmaceuticals Inc. (including their predecessors, subsidiaries or affiliates). The 
information cannot be disclosed to any third party or used for any purpose other than the purpose 
for which it is being submitted without the prior written consent of the appropriate Sumitomo 
Dainippon Pharma company.  
This information is being provided to you for the purpose of conducting a clinical study for 
Sunovion Pharmaceuticals Inc. You may disclose the contents of this protocol to the study 
personnel under your supervision and to your Institutional Review Board or Independent Ethics 
Committee for the above purpose. You may not disclose the contents of this protocol to any 
other parties, unless such disclosure is required by government regulations or laws, without the 
prior written permission of Sunovion Pharmaceuticals Inc. 
Any supplemental information (eg, a protocol amendment) that may be added to this document is 
also proprietary to Sunovion Pharmaceuticals Inc. and should be handled consistently with that 
stated above. 

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EMERGENCY CONTACTS 
Table 1: 
Emergency Contact Information 
Role in Study 
Name 
Contact Information 
Responsible Physician 
Vice President, Clinical 
Development and Medical 
Affairs 
Sunovion Pharmaceuticals Inc. 
Medical Monitor 
Medical Director, Oncology 
Medical Affairs 
PRA International 
SAE Reporting  
PPD Pharmacovigilance (PVG) 
Phone hotline: 919-456-6001 
Fax: 919-654-0211 
Email: Sunovionsafety@druginfo.com 

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1. 
SYNOPSIS 
Name of Sponsor/Company: Sunovion Pharmaceuticals Inc. 
Name of Investigational Product: WT2725 Dosing Emulsion 
Name of Active Ingredient: WT2725 
Title of Study: Initial Phase 1 Study of WT2725 Dosing Emulsion in Patients with Advanced 
Malignancies 
Study Center(s): Part 1: 4-8 clinical sites in the United States (US). Part 2: an additional 2 sites in the 
US may be included. 
Phase of Development: 1 
Study Objectives: 
Primary: 
The primary objectives are: 
• 
to evaluate the safety and tolerability of WT2725 Dosing Emulsion 
• 
to determine the maximum tolerated dose (MTD) of WT2725 Dosing Emulsion based on 
the evaluation of dose-limiting toxicity (DLT) 
Secondary: 
The secondary objectives are: 
• 
to describe antitumor responses to WT2725 Dosing Emulsion based on the immune-related 
response criteria (irRC) (Wolchok-2009, Hoos-2010), modified International Working 
Group response criteria in acute myeloid leukemia (IWG) (Cheson-2003), and/or tumor 
markers 
• 
to evaluate the immune responses to WT2725 Dosing Emulsion based on a series of 
circulating biomarkers in peripheral blood, delayed type hypersensitivity (DTH) to the 
WT2725 peptide, and immunohistochemistry in tumor tissue 
Other: 
The other objectives are: 
• 
to evaluate immune response by level of Wilms’ tumor gene product (WT1) protein 
expression in tumor cells 
• 
to obtain blood serum for future retrospective analyses of additional biomarkers 
Study Design: This is a Phase 1, open-label, dose-escalation, 2-part study in adult patients with 
advanced malignancies known to overexpress the WT1 protein. The study will primarily evaluate DLT 
and define the MTD of WT2725 Dosing Emulsion during the DLT Evaluation Period, which extends 
from the day of the first dose of study drug to just prior to the fifth dose (Days 1 - 29). In addition, 
antitumor activity and biomarkers indicative of immune response will be evaluated from the first dose 
until discontinuation of the patient from the study. An end of study assessment will take place within 
28 days after the last dose of study drug and prior to the start of alternate antineoplastic therapy. 
The initial patients in each dose escalation cohort will be enrolled at least one week apart to evaluate 

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Name of Sponsor/Company: Sunovion Pharmaceuticals Inc. 
Name of Investigational Product: WT2725 Dosing Emulsion 
Name of Active Ingredient: WT2725 
initial safety from the first 2 patients, then a “rolling-six” design will be employed that allows accrual 
of 2 to 6 patients concurrently in each dose escalation cohort. 
In Part 1 of the study, the 0.3 mg cohort will be conducted first. After safety and tolerability have been 
established for the first cohort, enrollment of subsequent patients will be conducted sequentially in 
subsequent cohorts and continue until the MTD or all planned cohorts are attained. Enrollment of each 
cohort may include patients with any of the eligible tumor types (see below). Enrollment of the highest 
tolerable dose cohort (MTD or highest planned cohort) will continue until there are at least 6 patients 
total in the study with each tumor type that are evaluable for DLT, biomarkers, and response. This dose 
is considered the recommended phase 2 dose (RP2D). Enrollment of up to 3 RP2D cohorts of 
10 patients each of a specific tumor type may take place at this dose in order to further characterize the 
safety and efficacy of WT2725 Dosing Emulsion at this dose in these subsets of patients. Additional 
dose escalation cohorts after the initial expansion cohorts may be enrolled if a MTD has not been 
determined and significant signs of toxicity have not been observed in the expansion cohorts. 
In Part 2 of the study, the 18.0 mg cohort will be conducted first. After safety and tolerability have 
been established for the 18.0 mg cohort, enrollment of subsequent patients will be conducted 
sequentially in the 27.0 mg cohort. Approximately 10 patients at each dose level (18.0 and 27.0 mg) 
with at least 4 patients with each malignancy type (glioblastoma and acute myeloid leukemia [AML]) 
will be enrolled at each of these dose levels. If the MTD is reached in Part 2 before the total of 
20 patients are enrolled, remaining patients will be enrolled at the prior dose level. In the event that a 
dose level is excluded from further study due to dose limiting toxicity all patients may continue the 
study at the most recently completed dose without DLT, eg, if the 27.0 mg dose is excluded due to 
DLT all patients may continue at 18.0 mg. 
Patients who have completed the consolidation phase of their assigned cohort and have not experienced 
a DLT, ≥ Grade 2 study drug-related injection-site reaction, or required a dose reduction may have 
their dose escalated to that of the highest cohort for which safety and tolerability have been established. 
The expected time to enroll each dose escalation cohort (2 – 6 patients) is between 6 and 12 weeks. 
Each part of the study will include 3 treatment phases based on intended timing of study drug dosing as 
indicated below: 
Part 1: 
• 
Vaccine Induction Phase: once every 7 days for 4 weeks (doses 1 - 5) 
• 
Consolidation Phase: once every 14 days for 6 weeks (doses 6 - 9) 
• 
Maintenance Phase: once every 28 days until discontinuation (dose 10 and thereafter) 
Part 2: 
• 
Vaccine Induction Phase: once every 7 days for 8 weeks (doses 1 - 9) 
• 
Consolidation Phase: once every 14 days for 10 weeks (doses 10 – 15 [maximum of 
6 doses]) 
• 
Maintenance Phase: once every 28 days until discontinuation (dose 16 and thereafter) 
Number of Patients (planned): Approximately 60-80 patients total in Parts 1 and 2 
Diagnosis and Main Criteria for Inclusion: Part 1: Adult patients with one of the following 

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Name of Sponsor/Company: Sunovion Pharmaceuticals Inc. 
Name of Investigational Product: WT2725 Dosing Emulsion 
Name of Active Ingredient: WT2725 
histologically or cytologically documented advanced stage malignancies: non-small cell lung, ovarian, 
glioblastoma, and AML not including acute promyelocytic leukemia, known to overexpress the WT1 
protein who have progressive or recurrent measurable (may be measurable by tumor markers only, 
such as quantitative reverse transcriptase – polymerase chain reaction [RT-PCR] for WT1 transcript for 
AML, or cancer antigen 125 [CA-125] for ovarian carcinoma) neoplastic disease despite administration 
of standard therapy or for whom no standard of therapy exists are eligible for enrollment in this study. 
Patients must also be human leukocyte antigen (HLA)-A*0201+ and/or HLA-A*0206+; have an 
Eastern Cooperative Oncology Group (ECOG) Performance Status score of 0, 1, or 2; have adequate 
bone marrow and immune reserve and renal and hepatic function; and meet criteria for recovery from 
effects of previous antineoplastic therapies.  
Part 2: Adult patients with histologically or cytologically documented advanced stage glioblastoma or 
AML not including acute promyelocytic leukemia, known to overexpress the WT1 protein who have 
progressive or recurrent measurable (may be measurable by tumor markers only, such as quantitative 
RT-PCR for WT1 transcript for AML) neoplastic disease despite administration of standard therapy or 
for whom no standard of therapy exists are eligible for enrollment in this study. Patients must also be 
HLA-A*0201+ and/or HLA-A*0206+; have an ECOG Performance Status score of 0, 1, or 2; have 
adequate bone marrow and immune reserve and renal and hepatic function; and meet criteria for 
recovery from effects of previous antineoplastic therapies. 
Investigational Product, Dosage, and Mode of Administration: 
Part 1: 
The following dose cohorts are planned: 
• 
0.3, 0.9, 3.0 , and 9.0 mg WT2725 Dosing Emulsion 
All study drug will be administered by subcutaneous (sc) injection (at 2 anatomical sites) once every 
7 days for 4 weeks, then once every 14 days for 6 weeks, and then once every 28 days until 
discontinuation. 
 
Part 2: 
The following dose cohorts are planned: 
• 
18.0 and 27.0 mg WT2725 Dosing Emulsion 
All study drug will be administered by sc injection (at 2 anatomical sites) once every 7 days for 
8 weeks, then once every 14 days for 10 weeks, and then once every 28 days until discontinuation. 
Duration of Treatment: Patients may remain on study drug until evidence of confirmed progressive 
disease, intolerance to treatment with study drug, or fulfillment of any of the other criteria for 
discontinuation. 
Reference Therapy, Dosage and Mode of Administration: Not applicable 
Criteria for Evaluation: 
Primary Endpoints:  
The safety and tolerability of WT2725 Dosing Emulsion will be evaluated based on the occurrence of 
DLT and AEs, and the findings from clinical laboratory tests, vital signs measurements, body weight 
measurements, and electrocardiogram (ECG) results. 

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Name of Sponsor/Company: Sunovion Pharmaceuticals Inc. 
Name of Investigational Product: WT2725 Dosing Emulsion 
Name of Active Ingredient: WT2725 
The determination of the MTD of WT2725 Dosing Emulsion will be based on the incidence of DLT at 
each dose level and the overall safety profile of WT2725 Dosing Emulsion. The incidence of DLT will 
be evaluated during the DLT Evaluation Period, which extends from the day of the first dose to just 
prior to the fifth dose of study drug (Days 1 to 29). No more than 4 doses of study drug will be 
administered during the DLT Evaluation Period. 
Dose-limiting toxicities are defined as any Grade 3 or greater AE that occurs after the administration of 
study drug during the DLT Evaluation Period that are not related to underlying disease, intercurrent 
illness, or concomitant medications (Changes in hematology parameters need to have been confirmed 
on repeat assessment and constitute at least a 2 grade shift. Grade 3 AEs of nausea, vomiting, and 
fatigue that are common and manageable in cancer patients will not be considered DLT if they can be 
ameliorated to < Grade 3 with standard supportive care management.). Adverse events will be graded 
according to the Common Terminology Criteria for Adverse Events (CTCAE) V.4.0. 
Secondary Endpoints: 
• 
Proportion of patients in each overall response category for each dose cohort based on 
irRC, modified IWG, and/or tumor markers, as appropriate, at each post-treatment tumor 
assessment and at the last assessment 
• 
Percent change in tumor burden from baseline to each post-treatment tumor assessment 
and the last tumor assessment for each dose cohort 
• 
The amount of immune response evaluated by: 
o measurement in peripheral blood of WT1 peptide-specific cytotoxic T lymphocyte 
(CTL) induction activity at each assessment time 
o measurement in peripheral blood of WT1 serum antibody titer at each assessment 
time 
o delayed-type hypersensitivity (DTH) to the WT2725 peptide at each assessment 
time 
o measurement of level of expression of CD8+, Foxp3, HLA class I, and WT1 
protein in tumor tissue using immunohistochemistry (IHC) methods 
Exploratory Endpoints: 
• 
Correlation between the level of expression of WT1 protein and the degree of the immune 
response at each assessment time as measured by CTL induction 
• 
Evaluation of changes in activity in peripheral blood mononuclear cells (PBMCs) after 
administration of study drug 
Statistical Methods: 
The Safety population will include all enrolled patients who receive at least 1 dose of study drug and 
will be used for the analysis of all safety and efficacy data except as noted. If needed, a DLT 
population will include all patients in the Safety population who are evaluable for DLT evaluation and 
will be used for analysis of DLT. If needed, an Efficacy population will include all patients in the 
Safety population who are evaluable for response and will be used for analysis of response. 
Data will generally be summarized by enrolled or starting dose. Descriptive statistics including number 
of patients, mean, standard deviation, minimum, 25th percentile, median, 75th percentile, and maximum 
will be provided. 
No imputation will be performed for missing data in this Phase 1 study. 

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Name of Sponsor/Company: Sunovion Pharmaceuticals Inc. 
Name of Investigational Product: WT2725 Dosing Emulsion 
Name of Active Ingredient: WT2725 
Safety measures will be summarized using descriptive statistics by enrolled dose cohort and listed for 
each patient. Adverse events also will be summarized by last dose administered before event start. 
Sample Size Calculation: The sample size is based on clinical and practical considerations for a 
Phase 1 dose escalation study using the rolling-six design, and is outside of statistical considerations. 
 

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2. 
TABLE OF CONTENTS, LIST OF TABLES, AND LIST OF 
FIGURES  
TABLE OF CONTENTS 
1. 
SYNOPSIS ...................................................................................................................4 
2. 
TABLE OF CONTENTS, LIST OF TABLES, AND LIST OF FIGURES .................9 
3. 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS.............................16 
4. 
INTRODUCTION ......................................................................................................19 
4.1. 
Study Rationale ...........................................................................................................21 
4.2. 
Dose Justification ........................................................................................................21 
4.2.1. 
Starting Dose Level ....................................................................................................21 
4.2.2. 
Additional Dose Levels for Part 2 ..............................................................................23 
4.2.3. 
Route of Administration .............................................................................................23 
4.2.4. 
Dosing Interval ...........................................................................................................23 
4.3. 
Summary of Known Potential Risks and Benefits .....................................................24 
5. 
STUDY OBJECTIVES ..............................................................................................25 
5.1. 
Primary Objectives .....................................................................................................25 
5.2. 
Secondary Objectives .................................................................................................25 
5.3. 
Other Objectives .........................................................................................................25 
6. 
STUDY ENDPOINTS ................................................................................................26 
6.1. 
Primary Endpoints ......................................................................................................26 
6.2. 
Secondary Endpoints ..................................................................................................26 
6.3. 
Exploratory Endpoints ................................................................................................27 
7. 
INVESTIGATIONAL PLAN .....................................................................................28 
7.1. 
Overall Study Design ..................................................................................................28 
7.2. 
Dose Level Escalation ................................................................................................44 
7.2.1. 
Criteria for Dose-limiting Toxicity .............................................................................46 
7.2.2. 
Reporting of Dose-limiting Toxicity or Completion of DLT Evaluation 
Period ..........................................................................................................................46 
7.3. 
Dose-Level Escalation Stopping Criteria ...................................................................46 
7.4. 
Dose Adjustment Criteria ...........................................................................................47 
7.4.1. 
Dose Reduction Criteria .............................................................................................47 
7.4.2. 
Dose Increases ............................................................................................................47 

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7.5. 
Prevention of Missing Data ........................................................................................47 
7.6. 
Study Termination Criteria .........................................................................................47 
8. 
SELECTION AND WITHDRAWAL OF PATIENTS ..............................................48 
8.1. 
Patient Inclusion Criteria ............................................................................................48 
8.1.1. 
Patient Inclusion Criteria – Part 1 ...............................................................................48 
8.1.2. 
Patient Inclusion Criteria - Part 2 ...............................................................................49 
8.2. 
Patient Exclusion Criteria ...........................................................................................50 
8.2.1. 
Patient Exclusion Criteria – Part 1 ..............................................................................50 
8.2.2. 
Patient Exclusion Criteria – Part 2 ..............................................................................52 
8.3. 
Continuation Criteria ..................................................................................................54 
8.3.1. 
Enrollment – Part 1 and Part 2 ....................................................................................54 
8.3.2. 
Subsequent Dosing .....................................................................................................54 
8.3.2.1. 
Subsequent Dosing – Part 1 ........................................................................................54 
8.3.2.2. 
Subsequent Dosing – Part 2 ........................................................................................54 
9. 
TREATMENT OF PATIENTS ..................................................................................56 
9.1. 
Concomitant Medications ...........................................................................................56 
9.1.1. 
Disallowed Medications During Study .......................................................................56 
9.1.2. 
Allowed Medications During Study ...........................................................................56 
9.1.3. 
Prohibited Therapies ...................................................................................................57 
9.1.4. 
Allowed Therapies ......................................................................................................57 
9.2. 
Treatment Compliance ................................................................................................57 
10. 
STUDY DRUG MATERIALS AND MANAGEMENT ...........................................58 
10.1. 
Description of Study Drug ..........................................................................................58 
10.2. 
Study Drug Packaging and Labeling ..........................................................................58 
10.3. 
Study Drug Storage .....................................................................................................59 
10.4. 
Study Drug Preparation ..............................................................................................59 
10.5. 
Study Drug Administration.........................................................................................60 
10.5.1. 
Assignment of Dose Cohort for Each Patient .............................................................61 
10.5.2. 
Selection of Dose Cohort for Each Patient .................................................................61 
10.6. 
Study Drug Accountability .........................................................................................62 
10.7. 
Study Drug Handling and Disposal ............................................................................62 
11. 
TREATMENT PLAN .................................................................................................63 
11.1. 
Study Assessments......................................................................................................63 

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11.1.1. 
Safety ..........................................................................................................................63 
11.1.2. 
Efficacy .......................................................................................................................63 
11.1.2.1. 
Immune-related Response Criteria (irRC) ..................................................................63 
11.1.2.2. 
Modified International Working Group response criteria in acute myeloid 
leukemia (IWG) ..........................................................................................................65 
11.1.2.3. 
Tumor Markers ...........................................................................................................65 
11.1.3. 
Other ...........................................................................................................................66 
11.2. 
Standardization of Data Capture .................................................................................66 
11.3. 
Electronic Data Capture (EDC) ..................................................................................67 
11.4. 
Study Visits and Assessments ....................................................................................67 
11.4.1. 
Screening – Part 1 and Part 2 ......................................................................................67 
11.4.1.1. 
Screening (within 21 days before the first dose) ........................................................67 
11.4.1.2. 
Screening (within 7 days before the first dose) ..........................................................68 
11.4.2. 
Study Treatment Periods .............................................................................................69 
11.4.2.1. 
Vaccine Induction Phase – Part 1 ...............................................................................69 
11.4.2.2. 
Consolidation Phase – Part 1 ......................................................................................72 
11.4.2.3. 
Maintenance Phase – Part 1 ........................................................................................74 
11.4.2.4. 
Vaccine Induction Phase – Part 2 ...............................................................................75 
11.4.2.5. 
Consolidation Phase Part 2 .........................................................................................79 
11.4.2.6. 
Maintenance Phase – Part 2 ........................................................................................82 
11.4.2.7. 
48 hours Before End of Study Evaluations – Part 1 and Part 2 ..................................83 
11.4.2.8. 
End of Study (EOS) (within 28 days after last dose of study drug and prior to 
start of alternate antineoplastic therapy) – Part 1 and Part 2 ......................................83 
11.4.3. 
Follow-up – Part 1 and Part 2 .....................................................................................84 
12. 
DISCONTINUATION AND REPLACEMENT OF PATIENTS/ CLINICAL 
ASSESSMENTS AFTER STUDY MEDICATION DISCONTINUATION ............85 
12.1. 
Clinical Assessments After Study Medication Discontinuation .................................85 
12.2. 
Study Participation Termination Criteria ....................................................................85 
12.3. 
Replacement of Patients .............................................................................................85 
13. 
ADVERSE EXPERIENCE REPORTING .................................................................86 
13.1. 
Adverse Events ...........................................................................................................86 
13.2. 
Objective Findings ......................................................................................................86 
13.3. 
Immediately Reportable Events ..................................................................................87 

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14. 
STATISTICS ..............................................................................................................89 
14.1. 
Sample Size Determination ........................................................................................89 
14.2. 
Randomization and Blinding ......................................................................................89 
14.3. 
Unblinding Procedures ...............................................................................................89 
14.4. 
Analysis Populations ..................................................................................................89 
14.5. 
Data Analysis ..............................................................................................................89 
14.5.1. 
Patient Disposition ......................................................................................................89 
14.5.2. 
Drug Exposure and Compliance .................................................................................89 
14.5.3. 
Important Protocol Deviations ....................................................................................90 
14.5.4. 
Demographic and Baseline Characteristics ................................................................90 
14.5.5. 
Efficacy Analysis ........................................................................................................90 
14.5.6. 
Adjustment for Multiplicity ........................................................................................90 
14.5.7. 
Safety Analysis ...........................................................................................................91 
14.5.7.1. 
DLT and Adverse Events ............................................................................................91 
14.5.7.2. 
Clinical Laboratory Assessments ...............................................................................91 
14.5.7.3. 
Electrocardiograms .....................................................................................................92 
14.5.7.4. 
Vital Signs ..................................................................................................................92 
14.5.7.5. 
Physical Examination .................................................................................................92 
14.5.7.6. 
Concomitant Medications ...........................................................................................92 
14.5.8. 
Treatment of Missing Data .........................................................................................93 
15. 
COMPUTERIZED SYSTEMS USED FOR SOURCE DATA .................................94 
16. 
ETHICAL AND REGULATORY OBLIGATIONS..................................................95 
16.1. 
Study Conduct ............................................................................................................95 
16.2. 
Institutional Review Board or Independent Ethics Committee ..................................95 
16.3. 
Informed Consent .......................................................................................................96 
16.4. 
Patient Privacy ............................................................................................................96 
16.5. 
Protocol Amendments and Emergency Deviations ....................................................97 
16.6. 
Monitoring and Auditing of the Study........................................................................97 
16.7. 
Study Documentation .................................................................................................97 
16.8. 
Laboratory Certification and Normal Values .............................................................98 
16.9. 
Drug Accountability ...................................................................................................98 
16.10. 
Records Retention .......................................................................................................98 
16.11. 
Inspection of Records .................................................................................................99 

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16.12. 
Financial Disclosure ...................................................................................................99 
17. 
STUDY ACKNOWLEDGMENT ............................................................................100 
18. 
REFERENCES .........................................................................................................101 
19. 
APPENDIX I. CARDIAC SAFETY MONITORING (ECG) ..................................103 
20. 
APPENDIX II. EASTERN COOPERATIVE ONCOLOGY GROUP 
(ECOG) PERFORMANCE STATUS ......................................................................104 
21. 
APPENDIX III. NEW YORK HEART ASSOCIATION [NYHA] 
FUNCTIONAL CLASSIFICATION .......................................................................105 
22. 
APPENDIX IV.  CLINICAL LABORATORY TESTS...........................................106 
23. 
APPENDIX V. BIOMARKER SAMPLING AND PROCESSING ........................109 
24. 
APPENDIX VI. STUDY DRUG PREPARATION .................................................113 
25. 
APPENDIX VII. DTH SKIN TEST PROCEDURES ..............................................116 
26. 
APPENDIX VIII. DEFINITIONS FOR REPORTING ADVERSE EVENTS ........117 
 

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LIST OF TABLES 
Table 1: 
Emergency Contact Information ...................................................................................3 
Table 2: 
Abbreviations and Specialist Terms ...........................................................................16 
Table 3: 
Schedule of Assessments for Screening and Vaccine Induction Phase – 
Part 1 ...........................................................................................................................32 
Table 4: 
Schedule of Assessments for Consolidation Phase, Maintenance Phase, and 
End of Study – Part 1 ..................................................................................................35 
Table 5: 
Schedule of Assessments for Screening and Vaccine Induction Phase – 
Part 2 ...........................................................................................................................38 
Table 6: 
Schedule of Assessments for Consolidation Phase, Maintenance Phase, and 
End of Study – Part 2 ..................................................................................................41 
Table 7: 
Computerized Systems Used for Source Data ............................................................94 
Appendix Table 8: Schedule of Clinical Laboratory Tests – Part 1..........................................107 
Appendix Table 9: Schedule of Clinical Laboratory Tests – Part 2..........................................108 
Appendix Table 10: Schedule of Biomarker Sampling – Part 1 ...............................................110 
Appendix Table 11: Schedule of Biomarker Sampling – Part 2 ...............................................111 
Appendix Table 12: Preparation of WT2725 Dosing Emulsion (Drug Product) – 
                Parts 1 and 2  ......................................................................................... 115 
 

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LIST OF FIGURES 
Figure 1: 
Study Schematic – Part 1 ............................................................................................31 
Figure 2: 
Study Schematic – Part 2 ............................................................................................31 
Figure 3: 
Dose Escalation Scheme – Part 1 ...............................................................................44 
Figure 4: 
Dose Escalation Scheme – Part 2 ...............................................................................44 
Figure 5: 
Description of WT2725 Dosing Emulsion (Drug Product) Preparation ....................59 
Appendix Figure 6: Description of WT2725 Dosing Emulsion (Drug Product) 
Preparation ................................................................................................................113 
 
  

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3. 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS 
 
Table 2: 
Abbreviations and Specialist Terms 
Abbreviation 
Full Form 
AE 
Adverse experience/event 
ALC 
Absolute lymphocyte count 
ALT 
Alanine aminotransferase 
AML 
Acute myeloid leukemia 
ANC 
Absolute neutrophil count 
AST 
Aspartate aminotransferase 
ATC 
Anatomic Therapeutic Class 
CA-125 
Cancer antigen 125 
CFR 
Code of Federal Regulations 
CRF 
Case report form 
CRO 
Contract research organization 
CT 
Computed tomography  
CTCAE 
Common Terminology Criteria for Adverse Events 
CTL 
Cytotoxic T lymphocyte 
CV 
Curricula vitae 
DLT 
Dose-limiting toxicity 
DSWI 
Diluting Solution for WT2725 Injection 
DTH 
Delayed-type hypersensitivity 
ECG 
Electrocardiogram 
ECOG 
Eastern Cooperative Oncology Group  
EDC 
Electronic data capture 
ELISA 
enzyme-linked immunosorbent assay 
EOS 
End of study 
ePRO 
Electronic patient reported outcomes 
FDA 
US Food and Drug Administration 
GCP 
Good Clinical Practice 
GM-CSF 
Granulocyte macrophage-colony stimulating factor 
HIV 
Human immunodeficiency virus  

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Table 2: 
Abbreviations and Specialist Terms (Continued) 
Abbreviation 
Full Form 
HLA 
Human leukocyte antigen 
ICH 
International Conference on Harmonisation 
Id 
intradermal 
IEC 
Independent Ethics Committee  
IFN 
Interferon 
IHC 
Immunohistochemistry 
IND 
Investigational New Drug 
IPD 
Important protocol deviation 
irCR 
Immune-related complete response 
irPD 
Immune-related progressive disease 
irPR 
Immune-related partial response 
irRC 
Immune-related Response Criteria 
IRB 
Institutional Review Board 
irSD 
Immune-related stable disease 
IWG 
International Working Group response criteria in acute myeloid 
leukemia 
LHRH 
Luteinizing hormone-releasing hormone 
MDS 
Myelodysplastic syndrome 
MedDRA 
Medical Dictionary for Regulatory Activities 
MHC 
Major histocompatibility complex 
MRI 
Magnetic resonance imaging 
MTD 
Maximum tolerated dose 
PBMC 
Peripheral blood mononuclear cell 
PET 
Positron-emission tomography 
PPD-PVG 
PPD Pharmacovigilance 
PR 
Time between P wave and QRS in electrocardiography 
PT 
Preferred term 
QRS 
Electrocardiographic wave (complex or interval) 
QT 
Electrocardiographic interval from the beginning of the QRS 
complex to the end of the T wave 
QTc 
QT interval corrected for heart rate 
RP2D 
Recommended phase 2 dose 

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Table 2: 
Abbreviations and Specialist Terms (Continued) 
Abbreviation 
Full Form 
RR 
Respiration rate 
RT-PCR 
Reverse transcriptase-polymerase chain reaction 
SAE 
Serious adverse experience/event 
Sc 
subcutaneous 
SOC 
System organ class 
SPECT 
Single-photon emission computed tomography 
SPD 
Sum of the products of the 2 largest perpendicular diameters 
TAA 
Tumor-associated antigen 
TEAE 
Treatment-emergent adverse event 
ULN 
Upper limit of normal 
US 
United States 
W/O 
Water-in-oil 
WOPE 
W/O pre-Emulsion 
WT1 
Wilms’ tumor gene product 1 
For the purposes of standardization, the following definitions will be used: 
• Case Report Form (CRF): A printed, optical, or electronic document designed to 
record all of the protocol required information to report to the sponsor for each study 
patient. 
• Screened Patient: Any patient who signed the study specific informed consent and 
completed at least one study related procedure. 
• Screen Failures: Any patient who signed the study specific informed consent but 
either failed to meet study requirements during screening or met study requirements 
at screening but was not enrolled. 
• Study Drug (or study medication): Term to cover investigational drug. 
• Enrolled Patient: Any patient who was successfully screened and enrolled into the 
Vaccine Induction Phase of the study. 

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4. 
INTRODUCTION 
Cancer vaccines consisting of synthetic cancer antigen-derived peptides designed to elicit T cell 
immunity have been evaluated as a potential treatment for cancer in clinical trials for more than a 
decade. Although the success rate in inducing specific T-cell responses has been mixed, there 
have been reports of minor tumor regression and occasional objective responses. 
Recent scientific advances have resulted in the identification of a large number of tumor-
associated antigens (TAAs) and their epitopes recognized by human leukocyte antigen (HLA) 
class I-restricted cytotoxic T lymphocytes (CTLs) from various types of malignant neoplasms 
(Buonaguro-2011). Cytotoxic T cells kill cancer cells by recognizing the major 
histocompatibility (MHC) class I-peptide complex on the surface of cancer cells. One such TAA 
is the Wilms’ tumor gene product 1 (WT1), which was isolated as a gene responsible for Wilms’ 
tumor, a pediatric renal cancer, and encodes a zinc finger transcription factor, which is involved 
in cell proliferation, differentiation, and apoptosis, and organ development. Expression of the 
wild-type WT1 gene has been found in various types of malignant tumors (Hutchings-2007) such 
as acute myeloid leukemia (AML), acute lymphoblastic leukemia, and myelodysplastic 
syndrome (MDS) at levels higher than those in normal bone marrow or peripheral blood and can 
confer oncogenic functions (Sugiyama-2005). In addition, various types of solid tumors, 
including breast, lung, thyroid, ovarian, glioblastoma, and colorectal, express the wild-type WT1 
protein at higher levels than surrounding normal tissues (Nakatsuka-2006, Oji-2004). 
Injection of peptides in aqueous solutions alone is generally not effective in stimulating CTL 
response. Therefore, adjuvants, such as water-in-oil (W/O) emulsion, have been used to enhance 
the immunogenicity of TAAs by creating a depot effect that prevents access of the immunogen 
to tissue and blood-borne proteases, while facilitating TAAs translocation into antigen-
presenting cells. 
The WT2725 drug product is referred to as WT2725 Dosing Emulsion and consists of WT2725, 
the acetate salt of a synthetic peptide with the same sequence as the naturally occurring peptide 
WT1126-134, diluted in a peptide diluting solution, and administered with a novel adjuvant (W/O 
pre-Emulsion). WT2725 administered intradermally (id) or subcutaneously (sc), is thought to be 
presented on the surface of antigen-presenting cells, eg, dendritic cells, as an MHC class 
I-peptide complex to be recognized by T cells. As a consequence, vaccination with WT2725 
Dosing Emulsion may stimulate the host immune system to induce a CTL response against 
cancer cells that overexpress the WT1 protein, resulting in cell lysis and inhibition of cancer cell 
proliferation. 
Nonclinical in vitro pharmacology studies confirmed that the WT2725 peptide bound to the 
human antigen-presenting molecule, HLA-A*0201, and induced CD8+ T cells reactive to the 
WT1126-134 peptide in human peripheral blood mononuclear cells (PBMCs) from an 
HLA-A*0201+ healthy donor. Nonclinical in vivo pharmacology studies confirmed that 
WT2725 Injection mixed with the novel W/O pre-Emulsion induced HLA-A*0201-restricted and 
WT2725 peptide-specific CTLs in HLA-A*0201-expressing transgenic mice. 
Additionally, when the WT2725 peptide sequence was analyzed using a web server for the 
prediction of human MHC class I-peptide binding, the binding affinity to HLA-A*0206 was 
strong therefore suggesting that WT2725 is expected to bind to not only HLA-A*0201 but also 

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HLA-A*0206. HLA-A*0206-restricted and WT1126-134-reactive CTLs were induced using 
HLA-A*0206+ PBMCs and the induced CTLs showed killing activity to a target cell line 
transfected with HLA-A*0206 and endogenously expressing WT1 protein (International 
Application Published under the Patent Cooperation Treaty: WO2009/072610). It is reported that 
valine and glutamine at position 2 in peptides are anchor residues of HLA-A*0206 (Sudo-1995) 
and that valine and leucine at position 9 are anchor residues of HLA-A*0206. The WT2725 
peptide has an anchor residue of HLA-A*0206 at position 9. Several HLA-A*0201 binding 
peptides which have an anchor residue of HLA-A*0206 at position 9 are reported to be presented 
by HLA-A*0206 or to elicit a CTL response with restriction of HLA-A*0206 (Eifuku-2001, 
Fleischhauer-1996, Li-2008, Zhang-2003). 
In repeat-dose toxicity studies of up to 13-weeks duration in cynomolgus monkeys, id or sc 
injection of the planned clinical WT2725 Dosing Emulsion was associated with macroscopic and 
microscopic findings along with clinical observations at the injection site. Additionally, non-
adverse macroscopic and microscopic findings were observed at the injection site following id or 
sc administration of the Control dosing emulsion. The skin reactions observed following 
administration of WT2725 Dosing Emulsion were the dose-limiting toxicity (DLT) in these 
studies, were noted to be reversible, and are readily monitorable with regular observation of the 
injection site. 
In a bacterial reverse gene mutation (Ames) assay and an in vitro chromosomal aberration assay, 
WT2725 did not induce mutations or chromosomal aberrations. 
In vitro studies demonstrated that WT2725 was unstable in human and monkey blood, and 
WT2725 was not detected in monkey plasma at any time point after a single id or sc 
administration. 
In previous clinical studies of the WT1126-134 peptide (administered with various adjuvants and 
combination therapy), few significant adverse reactions were noted, however various local skin 
reactions were observed (Kaida-2011, Keilholz-2009, Kuball-2011, Rezvani-2008, 
Gentilini-2006, Soeda-2010). 
Rezvani et al (2008) reported that WT1126-134 peptide in combination with PR1169-177 peptide 
using Montanide ISA-51 VG adjuvant and granulocyte macrophage-colony stimulating factor 
(GM-CSF) resulted in induction of WT1-specific CD8+ T cells. Additionally, Maslak et al 
(2010) using a vaccine of multiple WT1 peptides reported WT1-specific T-cell responses in the 
majority of patients and significant increases in interferon (IFN)-γ-secreting cells and frequency 
of WT1 tetramer+ CD8+ T cells in patients who were HLA-A*0201+. 
This first clinical study of WT2725 Dosing Emulsion is an open-label, dose escalation design to 
evaluate the safety and tolerability of WT2725 Dosing Emulsion delivered via sc injection in 
order to define the maximum tolerated dose (MTD). Additionally, antitumor activity and 
biomarkers indicative of immune response will be evaluated. WT2725 Dosing Emulsion will be 
administered as monotherapy to patients with one of the following advanced malignancies: 
non-small cell lung, ovarian, glioblastoma, and AML (not including acute promyelocytic 
leukemia), known to overexpress the WT1 protein. Tumors that do not overexpress the WT1 
protein will not be sensitive to WT1-specific CTLs therefore a subset of malignancies that do 
overexpress the WT1 protein was selected based on a review of the literature (Franko-2010, 
Nakatsuka-2006, Oji-2003). A rolling-six design (Skolnik-2008) will be used for enrollment into 

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ascending dose cohorts to determine the MTD. Patients may remain on study drug until there is 
evidence of confirmed progressive disease, intolerance to treatment with study drug, or 
fulfillment of any of the other criteria for discontinuation. 
4.1. 
Study Rationale 
Parenteral administration of the WT2725 peptide results in detection and presentation on the 
surface of antigen presenting cells, eg, dendritic cells, as an MHC class I-peptide complex to be 
recognized by T cells. As a result, vaccination with WT2725 Dosing Emulsion may stimulate the 
host immune system to induce a CTL response against WT1-expressing cells, resulting in cell 
lysis and inhibition of cancer cell proliferation. 
Treatment with other WT1 peptide vaccines in clinical trials has shown evidence of 
immunogenicity and clinical response in various malignancies. Based on the nonclinical findings 
for WT2725 and the reported clinical results with other WT1 peptide vaccines in cancer patients 
there is an expectation that some clinical benefit may be derived from treatment with WT2725 
Dosing Emulsion. In these other clinical trials, as well as in the nonclinical studies of WT2725, 
there have been limited significant adverse effects other than local injection site skin reactions. 
This study will assist with determining which doses level(s) to use in future clinical studies. In 
addition, this study will evaluate both clinical and immunological response. 
4.2. 
Dose Justification 
4.2.1. 
Starting Dose Level 
Two repeat-dose toxicology studies of up to 13-weeks duration conducted in cynomolgus 
monkeys using the planned clinical WT2725 Dosing Emulsion administered id (1.0 and 
3.0 mg/body) and sc (3.0 and 9.0 mg/body) once a week were conducted. In both studies, 
WT2725 Dosing Emulsion was associated with macroscopic and microscopic findings along 
with clinical observations at the injection site. Clinical observations at the injection site consisted 
of swelling, erythema, and/or crust which, in some cases, progressed to ulceration and erosion. 
These skin reactions were the DLT as persistence of ulceration/erosion for up to 5 days led to 
early euthanasia in some animals and discontinuation of dosing in the high dose groups after the 
tenth or eleventh administration in females and males, respectively. In surviving animals, the 
skin reactions at the injection site were noted to be reversible with discontinuation of dosing and 
administration of topical or intramuscular antibiotics. Systemic toxicological findings were 
limited to intermittent decreases in body weight and alterations in hematology and blood 
chemistry parameters that were judged to be secondary to stress and inflammatory changes at the 
injection site. 
A clinical study of a HLA-A*02-restricted WT1126-134 peptide vaccine, in which a peptide with 
the same sequence as the WT2725 peptide was administered with the Montanide ISA-51VG 
adjuvant, reported administration of 1.0 and 3.0 mg dose levels without apparent DLT 
(Kaida-2011). Vaccine (0.1 mL) was injected id into 6 sites (bilateral arms, 2 sites on the lower 
abdomen, and femoral areas) biweekly on Days 8 and 22. Gemcitabine was administered 
concurrently on Days 1, 8, and 15. Although the scheduled study period was 2 courses, treatment 
was continued at the patient’s request if there was no disease progression or serious adverse 
events (SAEs). Grade 1 or 2 skin reaction at the site of vaccination, characterized by redness, 

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pruritus, and induration, was observed in all patients (n = 25). WT1-specific T cells in peptide-
stimulated culture were detected by tetramer assay in 46% (5 of 11) of patients. 
The results of this clinical trial provide information about the safety of the WT1126-134 peptide 
vaccine based on pharmacological action of WT1126-134 peptide specific CTL. Although the 
adjuvant used in the clinical trial was different from that of the WT2725 Dosing Emulsion in its 
composition, the potential to induce WT1126-134 peptide specific CTL with the Montanide 
ISA-51VG adjuvant and the novel W/O pre-Emulsion for WT2725 Dosing Emulsion was 
evaluated and found to be similar in HLA-A02 transgenic mice. Furthermore, the W/O 
pre-Emulsion to be used with WT2725 by itself caused no severe skin lesion or systemic toxicity 
in the 13-week cynomolgus monkey studies. In addition, the same W/O pre-Emulsion has 
already been administered to several patients in 2 ongoing clinical trials of another modified 
peptide from the HLA-A*2402-restricted WT1235-243 peptide in Japan. In the Phase 1 solid tumor 
trial, as of 31 Dec 2011, for 3 enrolled patients only Grade 1 adverse events (AEs) had been 
reported including injection site reactions and for 1 additional patient Grade 2 injection site 
reaction and hypertension had been reported; this is preliminary draft data from an active, 
unlocked database and subject to change (data on file Chugai Pharmaceutical Co., Ltd). In the 
Phase 1/2 MDS study, as of the same date, for the one enrolled patient no injection site reactions 
had been observed, although Grade 4 neutropenia and thrombocytopenia, Grade 3 febrile 
neutropenia and leucopenia, and Grade 2 urticaria had been reported in addition to other Grade 1 
AEs; this is preliminary draft data from an active, unlocked database and subject to change (data 
on file Sumitomo Dainippon Pharma Co., Ltd). Because low blood cell counts are common with 
MDS and there have been few other toxicities greater than Grade 1 in these trials, the novel W/O 
pre-Emulsion used with WT2725 is not expected to cause unexpected severe AEs. 
In terms of clinical efficacy, it has been reported that sc injections of 0.2 mg WT1126-134 peptide 
in combination with 0.5 mg PR1169-177 peptide using Montanide ISA-51VG adjuvant and 
GM-CSF resulted in induction of WT1-specific CD8+ T cells (Rezvani-2008). The dose of 
0.2 mg per peptide also has been reported to be within the range found to be safe and active for 
other peptide vaccines (Maslak-2010). These reports suggest that this dose level may have a 
likelihood of immunological activity. 
For this study, a clinical starting dose of 0.3 mg/body (2 injection sites) is planned for Part 1, 
based on the following considerations: 
1. 0.3 mg/body by sc administration is equivalent to 1/10th the lower dose (3.0 mg/body 
WT2725 Dosing Emulsion) in the sc cynomolgus monkey study at which a severe skin 
lesion (ulceration) was noted in one animal. Of note, similar lesions observed at the 
highest dose (9.0 mg/body) showed reversibility with discontinuation of dosing and 
antibiotic treatment. In addition, skin lesions observed in the 1.0 mg/body id cohort 
showed reversibility without discontinuation of dosing or antibiotic treatment. 
2. Similar skin lesions have been observed in clinical studies of WT1126-134 peptide (doses 
ranging from 0.2 mg – 3 mg) (Kaida-2011, Keilholz-2009, Kuball-2011, Rezvani-2008, 
Gentilini-2006, Soeda-2010). Treatment for these skin lesions has included dermal 
steroid treatment and topical nonsteroidal anti-inflammatory drugs. 
3. There were no DLTs reported from the published clinical studies of the naturally 
occurring WT1126-134 peptide. A dose of 0.3 mg/body is approximately 1/10th the dose of 

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WT1126-134 peptide already used clinically, albeit with a different adjuvant, different 
administration route, and different number of injection sites. 
4. As it is ethically important to select a starting dose at which some clinical benefit for 
cancer patients may be expected, based on the results of clinical studies of other WT1 
peptides 0.3 mg/body is anticipated to be within an immunologically active dose range. 
4.2.2. 
Additional Dose Levels for Part 2 
Initial dose escalation in this study was completed, with the highest planned dose of 9.0 mg 
being administered without dose-limiting toxicity. This dose was considered the recommended 
phase 2 dose (RP2D), and additional patients were enrolled and initiated treatment at this dose. 
As of May 2014 the most common drug-related adverse reactions reported with doses up to 
9.0 mg were low grade localized injection site reactions with itching, erythema, pain, bruising, 
and/or swelling. A total of 14 treatment emergent serious adverse events (SAEs) were reported in 
9 patients; there was no pattern in the types of treatment emergent SAEs reported. Two serious 
adverse reactions (SARs; defined as SAEs that were assessed as at least possibly related to study 
drug) were reported in 2 patients; tumor necrosis was reported in 1 patient, and rash maculo-
papular was reported in 1 patient. Further with the expanded cohorts of patients treated at 
9.0 mg, no important unanticipated risks, potential risks, or safety signals have been observed, 
the 9.0 mg dose is not considered the MTD. Preliminary review of efficacy data indicates that 
WT2725 Dosing Emulsion has the potential to be of benefit to patients, which may be further 
enhanced by more regular injections in the early stages of treatment. Based on the results from 
these initial patients in Part 1, additional dose escalation, at no more than double the prior dose 
level, is being explored to evaluate safety, tolerability, and efficacy. In addition, extension of the 
induction and consolidation phases of treatment will be explored. 
4.2.3. 
Route of Administration 
Langerhans cells, which function as antigen-presenting cells, are widely distributed within the 
epidermis, and there are multiple reports of id and sc dosing of cancer vaccines to sequester 
antigen in the intradermal or subcutaneous tissue near the epidermis to ensure efficient 
recognition. A previous clinical study using WT1235-243, 2M→Y peptide (Oka-2008, Oka-2004) 
demonstrated that there was induction of WT1-specific CTL as a result of id dosing. A study by 
Keilholz et al (2009) of WT1126-134 peptide in keyhole-limpet-hemocyanin adjuvant plus 
GM-CSF demonstrated induction of WT1-specific CTL following id and sc injection and 
Rezvani et al (2008) using deep sc injection of WT1126-134 peptide in combination with PR1169-177 
peptide using a different adjuvant observed induction of WT1-specific CD8+ T cells following 
sc injection of the 2 peptides. Subcutaneous dosing may have several advantages over id dosing 
including less injection site pain for patients and easier administration. 
4.2.4. 
Dosing Interval 
Cancer vaccines generally have a slow onset of efficacy, and efficacy requires a significant 
amount of CTL induction in accord with the amount of disease in advanced malignancies. The 
optimal dosing interval for cancer vaccine treatments is unknown. Based on preceding peptide 
vaccine clinical trials, 1 week is thought to be an appropriate initial dosing interval for a Phase 1 
trial. 

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4.3. 
Summary of Known Potential Risks and Benefits 
Treatment with other WT1 peptide vaccines, including some with the same sequence as the 
WT2725 peptide, in clinical trials has shown evidence of immunogenicity and clinical response 
in various malignancies. Based on the nonclinical findings for WT2725 and the reported clinical 
results with other WT1 peptide vaccines in cancer patients there is an expectation that some 
clinical benefit may be derived from treatment with WT2725 Dosing Emulsion. In these clinical 
trials, as well as in the nonclinical studies of WT2725, there have been a limited number of 
significant adverse reactions other than local injection site skin reactions therefore presenting an 
acceptable benefit/risk profile to study participants. 
In this study at doses up to the RP2D of 9.0 mg, as of May 2014 the most common drug-related 
adverse reactions reported were low grade localized injection site reactions with itching, 
erythema, pain, bruising, and/or swelling. A total of 14 treatment-emergent SAEs were reported 
in 9 patients; there was no pattern in the types of treatment emergent SAEs reported. Two SARs 
were reported in 2 patients; tumor necrosis was reported in 1 patient, and rash maculo-papular 
was reported in 1 patient. Further with the expanded RP2D patients, no important unanticipated 
risks, potential risks, or safety signals have been observed, the 9.0 mg dose is not considered the 
MTD. Preliminary review of efficacy data indicates that WT2725 Dosing Emulsion has the 
potential to be of benefit to patients, which may be further enhanced by more regular injections 
in the early stages of treatment. Based on the results from these initial patients in Part 1, 
additional dose escalation, at no more than double the prior dose level, is being explored to 
evaluate safety, tolerability, and efficacy. In addition, extension of the induction and 
consolidation phases of treatment will be explored. 

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5. 
STUDY OBJECTIVES 
5.1. 
Primary Objectives 
The primary objectives are: 
• to evaluate the safety and tolerability of WT2725 Dosing Emulsion 
• to determine the MTD of WT2725 Dosing Emulsion based on the evaluation of DLT. 
5.2. 
Secondary Objectives  
The secondary objectives are: 
• to describe antitumor responses to WT2725 Dosing Emulsion based on the irRC 
(Wolchok-2009, Hoos-2010), modified International Working Group response 
criteria in acute myeloid leukemia (IWG) (Cheson-2003), and/or tumor markers 
• to evaluate the immune response to WT2725 Dosing Emulsion based on a series of 
circulating biomarkers in peripheral blood, delayed-type hypersensitivity (DTH) to 
the WT2725 peptide, and immunohistochemistry in tumor tissue. 
5.3. 
Other Objectives 
The other objectives are: 
• to evaluate immune response by level of WT1 protein expression in tumor cells 
• to obtain blood serum for future retrospective analyses of additional biomarkers. 

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6. 
STUDY ENDPOINTS 
6.1. 
Primary Endpoints 
The safety and tolerability of WT2725 Dosing Emulsion will be evaluated based on the 
occurrence of DLT and AEs, and the findings from clinical laboratory tests, vital signs 
measurements, body weight measurements, and electrocardiogram (ECG) results. 
The determination of the MTD of WT2725 Dosing Emulsion will be based on the incidence of 
DLT at each dose level and the overall safety profile of WT2725 Dosing Emulsion. The 
incidence of DLT will be evaluated during the DLT Evaluation Period, which extends from the 
day of the first dose to just prior to the fifth dose of study drug (Days 1 to 29). No more than 
4 doses of study drug will be administered during the DLT Evaluation Period. 
Dose-limiting toxicities are defined as any Grade 3 or greater AE that occurs after the 
administration of study drug during the DLT Evaluation Period that are not related to underlying 
disease, intercurrent illness, or concomitant medications (Changes in hematology parameters 
need to have been confirmed on repeat assessment and constitute at least a 2 grade shift. Grade 3 
AEs of nausea, vomiting, and fatigue that are common and manageable in cancer patients will 
not be considered DLT if they can be ameliorated to < Grade 3 with standard supportive care 
management.). Adverse events will be graded according to the Common Terminology Criteria 
for Adverse Events (CTCAE) V.4.0 (NCI-2009). 
6.2. 
Secondary Endpoints  
• Proportion of patients in each overall response category for each dose cohort based on 
irRC, modified IWG, and/or tumor markers, as appropriate, at each post-treatment 
tumor assessment and at the last assessment 
• Percent change in tumor burden from baseline to each post-treatment tumor 
assessment and the last tumor assessment for each dose cohort 
• The amount of immune response evaluated by: 
o measurement in peripheral blood of WT1 peptide-specific CTL induction 
activity at each assessment time  
o measurement in peripheral blood of WT1 serum antibody titer at each 
assessment time 
o DTH to the WT2725 peptide at each assessment time 
o measurement of level of expression of CD8+, Foxp3, HLA class I, and WT1 
protein in tumor tissue using immunohistochemistry (IHC) methods 

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6.3. 
Exploratory Endpoints  
• Correlation between the level of expression of WT1 protein and the degree of the 
immune response at each assessment time as measured by CTL induction 
• Evaluation of changes in activity in peripheral blood mononuclear cells (PBMCs) 
after administration of study drug 

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7. 
INVESTIGATIONAL PLAN 
7.1. 
Overall Study Design 
This is a Phase 1, open-label, dose-escalation, 2-part study in adult patients with advanced 
malignancies known to overexpress the WT1 protein and who have progressive or recurrent 
measurable (may be measurable by tumor markers only, such as quantitative reverse 
transcriptase – polymerase chain reaction [RT-PCR] for WT1 transcript for AML, or cancer 
antigen 125 [CA-125] for ovarian carcinoma) neoplastic disease despite administration of 
standard therapy or for whom no standard of therapy exists. In Part 1 of the study patients with 
one of the following malignancies: non-small cell lung, ovarian, glioblastoma, and acute myeloid 
leukemia (AML; not including acute promyelocytic leukemia) meeting the above criteria are 
eligible. In Part 2 of the study patients with glioblastoma and AML (not including acute 
promyelocytic leukemia) meeting the above criteria are eligible. 
Both parts of the study will primarily evaluate DLT and define the MTD of WT2725 Dosing 
Emulsion during the DLT Evaluation Period, which extends from the day of the first dose of 
study drug to just prior to the fifth dose (Days 1 - 29). Dose-limiting toxicities are defined as any 
Grade 3 or greater AE that occurs after the administration of study drug during the DLT 
Evaluation Period that are not related to underlying disease, intercurrent illness, or concomitant 
medications (Changes in hematology parameters need to have been confirmed on repeat 
assessment and constitute at least a 2 grade shift. Grade 3 AEs of nausea, vomiting, and fatigue 
that are common and manageable in cancer patients will not be considered DLT if they can be 
ameliorated to < Grade 3 with standard supportive care management.). In addition, antitumor 
activity and biomarkers indicative of immune response will be evaluated from the first dose until 
discontinuation of the patient from the study. An end of study assessment will take place within 
28 days after the last dose of study drug and prior to the start of alternate antineoplastic therapy. 
The initial patients in each dose escalation cohort will be enrolled at least one week apart to 
evaluate initial safety from the first 2 patients, then a “rolling-six” design will be employed that 
allows accrual of 2 to 6 patients concurrently in each dose escalation cohort. This design was 
chosen in an effort to reduce the delays associated with patient accrual using the classical “3 + 3” 
design as severe immediate reactions are not anticipated following administration of WT2725 
Dosing Emulsion. The decision to enroll an additional patient in the current dose cohort, to dose 
escalate to the next dose cohort, or to dose de-escalate to a lower dose cohort can be made 
without having to suspend enrollment following the initial enrollment of 3 patients, as in the 
“3 + 3” design. The decision regarding dose cohort for an incoming patient is dependent on the 
number of DLTs that have been observed, the number of patients enrolled at any time and the 
duration of their enrollment, and in Part 2 of the study the malignancy type. The goal remains to 
define the MTD as the dose level where no more than 1 of 6 patients experiences a DLT when 
2 of 2 to 6 patients experiences a DLT at the next higher dose level. Meetings to review study 
execution and enrollment/dosing decisions will be held at least monthly between the sponsor, 
clinical sites, and the medical monitor. The expected time to enroll each dose escalation cohort 
(2 – 6 patients) is between 6 and 12 weeks. 
The following dose cohorts of WT2725 Dosing Emulsion are planned for Part 1 of the study: 

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• 0.3, 0.9, 3.0, and 9.0 mg 
An approximate 3-fold increase in dose between cohorts (eg, 0.3, 0.9, 3.0 mg) will allow this 
dose range to be explored with the fewest number of dose levels. 
The following dose cohorts of WT2725 Dosing Emulsion are planned for Part 2 of the study: 
• 18.0 and 27.0 mg 
No more than a 2-fold increase in dose between cohorts (eg, 9.0, 18.0, 27.0 mg) will allow this 
higher dose range to be explored more conservatively. 
In Part 1 of the study, the 0.3 mg cohort will be conducted first. After safety and tolerability have 
been established for the first dose cohort, enrollment of subsequent patients will be conducted 
sequentially in subsequent cohorts and continue until the MTD or all planned cohorts are 
attained. Enrollment of each cohort may include patients with any of the eligible tumor types. 
Enrollment of the highest tolerable dose cohort (MTD or highest planned cohort) will continue 
until there are at least 6 patients total in the study with each tumor type that are evaluable for 
DLT, biomarkers, and response. This dose is considered the recommended phase 2 dose (RP2D). 
Enrollment of up to 3 RP2D cohorts of 10 patients each of specific tumor types may take place at 
this dose in order to further characterize the safety and efficacy of WT2725 Dosing Emulsion at 
this dose in these subsets of patients. 
Additional dose escalation cohorts after the initial expansion cohorts may be enrolled if a MTD 
has not been determined and significant signs of toxicity have not been observed in the 
expansion cohorts. 
In Part 2 of the study, the 18.0 mg cohort will be conducted first. After safety and tolerability 
have been established for the 18.0 mg cohort, enrollment of subsequent patients will be 
conducted sequentially in the 27.0 mg cohort. Approximately 10 patients at each dose level (18.0 
and 27.0 mg) with at least 4 patients with each malignancy type (glioblastoma and AML) will be 
enrolled at each of these dose levels. If the MTD is reached in Part 2 before the total of 
20 patients are enrolled, remaining patients will be enrolled at the prior dose level. Patients who 
experience DLT may remain on the study at a reduced dose if they are deriving some therapeutic 
benefit from study drug and fulfill the criteria for study continuation for subsequent dosing (see 
Section 8.3.2); these decisions will be addressed on a case by case basis. Patients who experience 
a DLT or other significant toxicity at this reduced dose will be discontinued from receiving 
further study drug. In the event that a dose level is excluded from further study due to dose 
limiting toxicity all patients may continue the study at the most recently completed dose without 
DLT, eg, if the 27.0 mg dose is excluded due to DLT all patients may continue at 18.0 mg. 
After the DLT Evaluation Period, if a patient experiences a study drug-related Grade 3 toxicity, 
there will be a review of safety data to determine appropriate dosing for that patient and other 
patients on study, as well as for any subsequently enrolled patients. 
In the event that a Grade 4 or greater toxicity is reported at any time during the study, the 
sponsor and the investigator will conduct a thorough evaluation of the available safety data to 
decide whether to continue enrolling new patients into the study. 
Patients who have completed the consolidation phase of their assigned cohort and have not 
experienced a DLT, ≥ Grade 2 study drug-related injection-site reaction, or required a dose 

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reduction may have their dose level escalated to that of the highest cohort for which safety and 
tolerability have been established. Patients may remain on study drug until evidence of 
confirmed progressive disease, intolerance to study drug, or fulfillment of any of the other 
criteria for discontinuation (see Section 12.2). 
Each part of the study will include 3 treatment phases based on intended timing of study drug 
dosing as indicated below: 
Part 1: 
• Vaccine Induction Phase: once every 7 days for 4 weeks (doses 1 - 5) 
• Consolidation Phase: once every 14 days for 6 weeks (doses 6 - 9) 
• Maintenance Phase: once every 28 days until discontinuation (dose 10 and thereafter) 
Part 2: 
• Vaccine Induction Phase: once every 7 days for 8 weeks (doses 1 - 9) 
• Consolidation Phase: once every 14 days for 10 weeks (doses 10 – 15 [maximum 
6 doses]) 
• Maintenance Phase: once every 28 days until discontinuation (dose 16 and thereafter) 
Approximately 60 to 80 patients total including replacements will be dosed in this clinical trial. 
The total number of patients dosed in this clinical trial will be determined by the number of 
patients treated in each dose cohort and the number of dose cohorts necessary to determine the 
MTD, as well as the number of RP2D cohorts enrolled. If all 4 dose escalation cohorts are 
necessary in Part 1, and if 6 enrolled patients are required in each dose escalation cohort and/or 
tumor type, then 24 patients evaluable for DLT, biomarkers, and response would be necessary in 
the Part 1 dose escalation. In addition, up to 30 patients may be enrolled in the RP2D cohorts in 
Part 1. If both dose escalation cohorts are necessary in Part 2, and if 10 patients are enrolled in 
each of these cohorts, then 20 patients will be enrolled in Part 2. Patients who discontinue from 
the study prior to completion of the DLT, biomarker response evaluation period will be replaced 
likely increasing the total number of patients enrolled.  
Four to 8 investigative sites in the United States (US) will be utilized to enroll patients in Part 1 
of the study. An additional 2 sites in the US may be opened to enroll patients in Part 2. 
A study schematic is presented in Figure 1 for Part 1 and in Figure 2 for Part 2. Details of study 
assessments and other procedures to be performed at each visit in Part 1 are presented in Table 3, 
Schedule of Assessments for Screening and Vaccine Induction Phase – Part 1 and Table 4, 
Schedule of Assessments for Consolidation Phase, Maintenance Phase, and End of Study – 
Part 1; and Section 11, Treatment Plan. Details of study assessments and other procedures to be 
performed at each visit in Part 2 are presented in Table 5, Schedule of Assessments for Screening 
and Vaccine Induction Phase – Part 2 and Table 6, Schedule of Assessments for Consolidation 
Phase, Maintenance Phase, and End of Study – Part 2; and Section 11, Treatment Plan. 

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Figure 1: 
Study Schematic – Part 1 
 
 
Figure 2: 
Study Schematic – Part 2 
 
 
Screening 
 
 
 
within 
21 days 
before first 
dose 
Vaccine 
Induction 
Phase 
 
Dosing 
once every 
7 days  
for 4 weeks 
(doses 1 - 5) 
Consolidation 
Phase 
Dosing once 
every 14 days 
for 6 weeks 
(doses 6 - 9) 
Maintenance 
Phase 
 
Dosing once 
every 28 days 
until 
discontinuation 
(dose 10 and 
thereafter) 
 
 
End of 
Study 
 
within 
28 days 
after last 
dose and 
prior to 
start of 
alternative 
antineoplas
tic therapy 
DLT Evaluation 
Period 
Days 1 to 29 
Follow-up 
Disease Progression/Survival 
Every 3 
months 
Screening 
 
 
 
within 
21 days 
before first 
dose 
Vaccine 
Induction 
Phase 
 
Dosing 
once every 
7 days  
for 8 weeks 
(doses 1 - 9) 
Consolidation 
Phase 
Dosing once 
every 14 days 
for 10 weeks 
(doses 10 – 15 
[maximum 6 
doses]) 
Maintenance 
Phase 
Dosing once 
every 28 days 
until 
discontinuation 
(dose 16 and 
thereafter) 
End of 
Study 
 
within 
28 days 
after last 
dose and 
prior to 
start of 
alternative 
antineoplas
tic therapy 
DLT Evaluation 
Period 
Days 1 to 29 
Follow-up 
Disease Progression/Survival 
Every 3 
months 

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Table 3: 
Schedule of Assessments for Screening and Vaccine Induction Phase – Part 1 
 
Screening 
Vaccine Induction Phasea,b 
 
within 
14/21 days 
before 1st 
dose (-1 – 
 -14/-21) 
within 
7 days 
before 1st 
dose  
(-1 - -7) 
 
1st dose 
 
2nd dose 
3rd dose 
4th dose 
 
5th dose 
 
Day  
-2 
Day 1 (-1) 
Day 
3 
Day 8 (-1) 
Day 15 (-1) 
Day 22 (-1) 
Day 
27 
Day 29 (-1) 
Day 31 
Informed consent 
X 
 
 
 
 
 
 
 
 
 
 
Inclusion/exclusion 
X 
X 
 
 
 
 
 
 
 
 
 
Continuation criteria 
 
 
 
Xc 
 
Xc 
Xc 
Xc 
 
Xc 
 
Study drug 
administration 
 
 
 
X 
 
X 
X 
X 
 
X 
 
Medical history 
X 
 
 
 
 
 
 
 
 
 
 
Physical exam 
 
X 
 
Xc 
 
Xc 
Xc 
Xc 
 
Xc 
 
Height 
 
X 
 
 
 
 
 
 
 
 
 
Weight 
 
X 
 
Xc 
 
Xc 
Xc 
Xc 
 
Xc 
 
Vital Signsd 
 
X 
 
Xc 
 
Xc 
Xc 
Xc 
 
Xc 
 
ECOG performance 
status 
 
X 
 
Xc 
 
Xc 
Xc 
Xc 
 
Xc 
 
HLA typinge 
X 
 
 
 
 
 
 
 
 
 
 
Serum pregnancy testf 
X 
 
 
Xc 
 
 
 
 
 
 
 
Serologyg 
X 
 
 
 
 
 
 
 
 
 
 
Hematology/ serum 
chemistryh 
 
X 
 
Xc,i 
 
Xc 
Xc 
Xc 
 
Xc 
 
Urinalysis 
 
X 
 
Xc,i 
 
 
Xc 
 
 
Xc 
 
12-lead ECG 
 
X 
 
Xc,i 
 
 
 
 
 
Xc 
 
DTH skin test 
injection 
 
 
Xj 
Xj,k 
 
 
 
 
Xj 
Xj,k 
 

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Table 3: 
Schedule of Assessments for Screening and Vaccine Induction Phase – Part 1 (Continued) 
 
Screening 
Vaccine Induction Phasea,b 
 
within 
14/21 days 
before 1st 
dose (-1 – 
 -14/-21) 
within 7 
days 
before 1st 
dose 
(-1 - -7) 
 
1st dose 
 
2nd dose 
3rd dose 
4th dose 
 
5th dose 
 
Day  
-2 
Day 1 (-1) 
Day 
3 
Day 8 (-1) 
Day 15 (-1) 
Day 22 (-1) 
Day 27 
Day 29 (-
1) 
Day 31 
DTH skin test 
evaluationl 
 
 
 
Xm 
Xn 
 
 
 
 
Xm 
Xn 
Blood sample for 
CTL 
 
X 
 
Xc 
 
Xc 
Xc 
Xc 
 
Xc 
 
Blood sample for 
Anti-WT1 
antibody 
 
 
 
Xc 
 
 
Xc 
 
 
Xc 
 
Blood sample for 
PBMC isolation 
 
 
 
Xc 
 
 
 
 
 
Xc 
 
Blood sample for 
retrospective 
biomarker 
analyses 
 
 
 
Xc,o 
Xo 
Xc 
Xc 
 
 
Xc 
 
IHC tumor 
samples 
 
Xp,q 
 
 
 
 
 
 
 
 
 
Tumor 
assessment 
Xr 
 
 
 
 
 
 
 
 
Xs 
 
Adverse events 
 
 
Concomitant 
medications 
 
 
Abbreviations: AML = acute myeloid leukemia, CA-125 = cancer antigen 125, CTL = cytotoxic T lymphocyte induction activity, DTH = delayed-type hypersensitivity reactivity, 
ECG = electrocardiogram, ECOG = Eastern Cooperative Oncology Group, FSH = follicle-stimulating hormone, HLA = human leukocyte antigen, 
IHC = immunohistochemistry, irRC = immune-related response criteria, IWG = International Working Group response criteria in acute myeloid leukemia, MRI = magnetic 
resonance imaging, PBMC = peripheral blood mononuclear cell, PET = positron-emission tomography, RT-PCR = reverse transcriptase-polymerase chain reaction, 
SPECT = single-photon emission computed tomography, WT1 = Wilms’ tumor gene product 1 
a Additional dosing windows are provided for patients who may need to recover from intercurrent medical conditions (see Section 8.3.2.1). 
b All patients should complete an End of Study visit (see Table 4) within 28 days after the last dose of study drug and prior to the start of alternate antineoplastic therapy. 
Footnotes continued on the next page. 

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c Prior to study drug administration. 
d Includes systolic and diastolic blood pressures, pulse, respirations rate, and oral body temperature. 
e If HLA typing has been performed previously, these results may be used, otherwise HLA typing should be the first procedure performed during screening. 
f For females of child-bearing potential. 
g Includes hepatitis B and C, human immunodeficiency virus (HIV)-1, and HIV-2, if there are signs or symptoms suggestive of infection. 
h See Section 22 (Appendix IV) for list of required laboratory tests at each visit. 
i Assessment may be omitted if an assessment was performed within the previous 3 days. If there are multiple assessments the most recent ones should be used. 
j Perform DTH skin test injections approximately 48 hours (but no sooner than 24 hours) prior to the evaluation.  
k Perform DTH skin test injections immediately before administration of study drug. 
l If the DTH reactivity results in erythema with induration greater than 2 cm, or leads to ulceration of the skin test site, further DTH testing should not be conducted for the patient. 
m Evaluation should be made on the planned dosing day, prior to study drug administration. 
n Evaluation should be approximately 48 hours (but no sooner than 24 hours) after the DTH skin test injection. 
o Samples obtained 6 hours (± 30 minutes) and 48 hours (± 6 hours) postdose. 
p In place of tumor tissue samples from a biopsy during screening, archived tumor tissue samples may be provided. Availability of tumor tissue samples should be determined 
during screening through provision of the accession number or other identification number. If necessary, the tumor tissue sample biopsy should be performed after study 
eligibility has been confirmed and before the first dose of study drug. The tumor tissue samples will only be provided to the sponsor for patients who receive study drug. In place 
of archival tumor tissue samples, patients with AML should have available a bone marrow aspirate and/or bone marrow biopsy with PCR for WT1 transcript performed before 
the first dose of study drug. Patients with AML should also have peripheral blood assessed for blasts and WT1 transcript. 
q IHC need not be performed for patients with AML provided RT-PCR for WT1 transcript is assessed. 
r Tumor assessments (per irRC for solid tumors, modified IWG for AML, and/or standard tumor markers as appropriate eg, CA-125 for ovarian carcinoma and quantitative 
RT-PCR for WT1 transcript for AML) are not required to be repeated if they have been performed within the 28 days before first dose (there are no variations allowed beyond 
this window). If multiple assessments are available, the most recent ones should be used as the baseline result. Data from at least one additional historical assessment prior to the 
baseline assessment should also be provided, if available. Progressive disease requires confirmation by a repeat, consecutive assessment at least 4 weeks after the date of first 
documentation, unless rapid clinical deterioration is seen. For patients with glioblastoma, progressive disease should additionally be confirmed by SPECT, perfusion MRI, MRI 
spectroscopy or C-14 methionine PET or pathology from available surgical/biopsy specimens to differentiate from pseudoprogression. For patients with ovarian cancer that 
express CA-125, progressive disease should additionally be confirmed by CA-125 (≥ 2x higher of ULN or on study nadir). For patients with AML, bone marrow 
aspirations/biopsies are to be provided at baseline and subsequently when clinically indicated, however peripheral blood should be assessed for blasts and WT1 transcript at 
baseline and at least every 4 weeks or more frequently as clinically indicated. 
s Tumor markers drawn from peripheral blood samples should be assessed at least every 4 weeks after the 1st dose but may be performed more frequently (For other tumor 
assessments, perform every 8 weeks after 1st dose or more frequently as clinically indicated). 

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Table 4: 
Schedule of Assessments for Consolidation Phase, Maintenance Phase, and End of Study – Part 1 
 
Consolidation Phasea,b 
Maintenance 
Phasea,b 
End of Studyb  
Follow-up 
 
6th dose 
7th dose  
8th dose 
 
9th dose 
 
 
 
 
 
 
Day 43 
(±2) 
Day 57 
(±2) 
Day 71 (±2) 
Day 83  
Day 85 (±2) 
Day 87 
every 28 
(+7) days  
48 hours 
before End 
of Study 
Visit 
within 28 
days after 
last dose 
every 3 
months 
Continuation criteria 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
 
 
 
Study drug 
administration 
X 
X 
X 
 
X 
 
X 
 
 
 
Physical exam 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
 
X 
 
Weight 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
 
X 
 
Vital Signsd 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
 
X 
 
ECOG performance 
status 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
 
X 
 
Serum pregnancy teste 
 
 
 
 
 
 
 
 
X 
 
Hematology/ serum 
chemistryf 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
 
X 
 
Urinalysis 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
 
X 
 
12-lead ECG 
 
Xc 
 
 
Xc 
 
Xc 
 
X 
 
DTH skin test injection 
 
 
 
Xg 
Xg,h 
 
 
Xg 
 
 
DTH skin test evaluation 
 
 
 
 
Xi 
Xj 
 
 
Xj 
 
Blood sample for CTL 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
 
X 
 
Blood sample for Anti-
WT1 antibody 
 
Xc 
 
 
Xc 
 
Xc,k 
 
X 
 
Blood sample for 
retrospective biomarker 
analyses 
 
Xc 
 
 
Xc 
 
Xc,k 
 
X 
 

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Table 4: 
Schedule of Assessments for Consolidation Phase, Maintenance Phase, and End of Study – Part 1 (Continued) 
 
Consolidation Phasea,b 
Maintenance 
Phasea,b 
End of Studyb  
Follow-up 
 
6th dose 
7th dose  
8th dose 
 
9th dose 
 
 
 
 
 
 
Day 43 
(±2) 
Day 57 
(±2) 
Day 71 (±2) 
Day 83  
Day 85 (±2) 
Day 87 
every 28 
(+7) days  
48 hours 
before End 
of Study 
Visit 
within 28 
days after 
last dose 
every 3 
months 
Blood sample for PBMC 
isolation 
 
 
 
 
 
 
 
 
X 
 
IHC tumor samples 
 
 
 
 
 
 
 
 
Xl 
 
Tumor assessment 
 
Xc,m 
 
 
Xc,n 
 
Xc,m 
 
Xm 
 
Adverse events 
 
 
Concomitant medications 
 
 
Disease Progression/ 
Survival 
 
 
 
 
 
 
 
 
Xo 
Xo 
Abbreviations: AML = acute myeloid leukemia, CA-125 = cancer antigen 125, CTL = cytotoxic T lymphocyte induction activity, DTH = delayed-type hypersensitivity reactivity, 
ECG = electrocardiogram, ECOG = Eastern Cooperative Oncology Group, FSH = follicle-stimulating hormone, HLA = human leukocyte antigen, 
IHC = immunohistochemistry, irRC = immune-related response criteria, IWG = International Working Group response criteria in acute myeloid leukemia, MRI = magnetic 
resonance imaging, PBMC = peripheral blood mononuclear cell, PET = positron-emission tomography, RT-PCR = reverse transcriptase-polymerase chain reaction, 
SPECT = single-photon emission computed tomography, WT1 = Wilms’ tumor gene product 1 
a Additional dosing windows are provided for patients who may need to recover from intercurrent medical conditions (see Section 8.3.2.1).  
b All patients should complete an End of Study visit within 28 days after the last dose of study drug and prior to the start of alternate antineoplastic therapy. 
c Prior to study drug administration. 
d Includes systolic and diastolic blood pressures, pulse, respirations rate, and oral body temperature. 
e For females of child-bearing potential. 
f See Section 22 (Appendix IV) for list of required laboratory tests at each visit. 
g Perform DTH skin test injections approximately 48 hours (but no sooner than 24 hours) prior to the evaluation. 
h Perform DTH skin test injections immediately before administration of study drug. 
i Evaluation should be made on the planned dosing day, prior to study drug administration. 
j Evaluation should be approximately 48 hours (but no sooner than 24 hours) after the DTH skin test injection. 
k Perform every 2nd dosing day starting after the 9th dose. 
l Biopsy for tumor tissue samples after the last dose of study drug is not mandatory. Tumor samples will be obtained from all patients who provide consent. IHC need not be 
performed for patients with AML provided RT-PCR for WT1 transcript is assessed. 
 
Footnotes continued on the next page. 

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m Tumor assessments will be by irRC for solid tumors, modified IWG for AML, and/or standard tumor markers as appropriate eg, CA-125 for ovarian carcinoma and quantitative 
RT-PCR for WT1 transcript for AML. Perform every 8 weeks after 1st dose or more frequently as clinically indicated. Tumor markers drawn from peripheral blood samples 
should be assessed every 4 weeks after the 1st dose. This assessment may be performed ± 7 days of the scheduled assessment but must be completed before the next planned 
administration of study drug. For patients with AML if no other measurable disease then bone marrow aspirations/biopsies should follow the general post-transplant biopsy 
schedule or other clinical site guidelines. Progressive disease requires confirmation by a repeat, consecutive assessment at least 4 weeks after the date of first documentation, 
unless rapid clinical deterioration is seen. For patients with glioblastoma, progressive disease should additionally be confirmed by SPECT, perfusion MRI, MRI spectroscopy or 
C-14 methionine PET or pathology from available surgical/biopsy specimens to differentiate from pseudoprogression. For patients with ovarian cancer that express CA-125, 
progressive disease should additionally be confirmed by CA-125 (≥ 2x higher of ULN or on study nadir). For patients with AML, bone marrow aspirations/biopsies are 
scheduled when clinically indicated, however peripheral blood should be assessed for blasts and WT1 transcript at least at every 4 weeks or more frequently as clinically 
indicated. 
n Tumor markers drawn from peripheral blood samples should be assessed at least every 4 weeks after the 1st dose but may be performed more frequently (For other tumor 
assessments, perform every 8 weeks after 1st dose or more frequently as clinically indicated). 
o Following the End of Study visit, patients will be contacted every 3 months to evaluate disease progression, if not already reached, and survival. 

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Table 5: 
Schedule of Assessments for Screening and Vaccine Induction Phase – Part 2 
 
Screening 
Vaccine Induction Phasea,b,t 
within 
21 days 
before 
1st dose 
(-1 - -21) 
within 
7 days 
before 
1st dose  
(-1 - -7) 
 
1st 
dose 
 
2nd dose 
3rd dose 
4th dose 
 
5th dose 
 
6th dose 
7th 
dose 
8th 
dose 
9th 
dose 
Day  
-2 
Day 1 
(-1) 
Day 
3 
Day 8  
(-1) 
Day 15  
(-1) 
Day 22  
(-1) 
Day 
27 
Day 29  
(-1) 
Day 
31 
Day 36 
(-1) 
Day 
43 (-1) 
Day 
50 (-1) 
Day 
57 (-1) 
Informed consent 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Inclusion/exclusion 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
Continuation 
criteria 
 
 
 
Xc 
 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Study drug 
administration 
 
 
 
X 
 
X 
X 
X 
 
X 
 
X 
X 
X 
X 
Medical history 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Physical exam 
 
X 
 
Xc 
 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Height 
 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
Weight 
 
X 
 
Xc 
 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Vital Signsd 
 
X 
 
Xc 
 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
ECOG 
performance status 
 
X 
 
Xc 
 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
HLA typinge 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Serum pregnancy 
testf 
X 
 
 
Xc 
 
 
 
 
 
 
 
 
 
 
 
Serologyg 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Hematology/ 
serum chemistryh 
 
X 
 
Xc,i 
 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Urinalysis 
 
X 
 
Xc,i 
 
 
Xc 
 
 
Xc 
 
 
Xc 
 
Xc 
12-lead ECG 
 
X 
 
Xc,i 
 
 
 
 
 
Xc 
 
 
 
 
Xc 

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Table 5: 
Schedule of Assessments for Screening and Vaccine Induction Phase – Part 2 (Continued) 
 
Screening 
Vaccine Induction Phasea,b,t 
within 
14/21 
days 
before 
1st dose 
(-1 – 
 -14/-21) 
within 
7 days 
before 
1st dose  
(-1 - -7) 
 
1st 
dose 
 
2nd dose 
3rd dose 
4th dose 
 
5th dose 
 
6th dose 
7th 
dose 
8th 
dose 
9th 
dose 
Day  
-2 
Day 1 
(-1) 
Day 
3 
Day 8  
(-1) 
Day 15  
(-1) 
Day 22  
(-1) 
Day 
27 
Day 29  
(-1) 
Day 
31 
Day 36 
(-1) 
Day 
43 (-1) 
Day 
50 (-1) 
Day 
57 (-1) 
DTH skin test 
injection 
 
 
Xj 
Xj,k 
 
 
 
 
Xj 
Xj,k 
 
 
 
 
 
DTH skin test 
evaluationl 
 
 
 
Xm 
Xn 
 
 
 
 
Xm 
Xn 
 
 
 
 
Blood sample for 
CTL 
 
X 
 
Xc 
 
Xc 
Xc 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Blood sample for 
Anti-WT1 
antibody 
 
 
 
Xc 
 
 
Xc 
 
 
Xc 
 
 
 
 
Xc 
Blood sample for 
PBMC isolation 
 
 
 
Xc 
 
 
 
 
 
Xc 
 
 
 
 
 
Blood sample for 
retrospective 
biomarker analyses 
 
 
 
Xc,o 
Xo 
Xc 
Xc 
 
 
Xc 
 
 
 
 
Xc 
IHC tumor samples 
 
Xp,q 
 
 
 
 
 
 
 
 
 
 
 
 
 
Tumor assessment 
Xr 
 
 
 
 
 
 
 
 
Xc,s 
 
 
 
 
Xc,r 
Adverse events 
 
Concomitant 
medications 
 
Abbreviations: AML = acute myeloid leukemia, CTL = cytotoxic T lymphocyte induction activity, DTH = delayed-type hypersensitivity reactivity, ECG = electrocardiogram, 
ECOG = Eastern Cooperative Oncology Group, FSH = follicle-stimulating hormone, HLA = human leukocyte antigen, IHC = immunohistochemistry, irRC = immune-related 
response criteria, IWG = International Working Group response criteria in acute myeloid leukemia, MRI = magnetic resonance imaging, PBMC = peripheral blood mononuclear 
cell, PET = positron-emission tomography, RT-PCR = reverse transcriptase-polymerase chain reaction, SPECT = single-photon emission computed tomography, WT1 = Wilms’ 
tumor gene product 1 
Footnotes provided on the next page. 

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a Additional dosing windows are provided for patients who may need to recover from intercurrent medical conditions (see Section 8.3.2.2). 
b All patients should complete an End of Study visit (see Table 6) within 28 days after the last dose of study drug and prior to the start of alternate antineoplastic therapy. 
c Prior to study drug administration. 
d Includes systolic and diastolic blood pressures, pulse, respirations rate, and oral body temperature. 
e If HLA typing has been performed previously, these results may be used, otherwise HLA typing should be the first procedure performed during screening. 
f For females of child-bearing potential. 
g Includes hepatitis B and C, human immunodeficiency virus (HIV)-1, and HIV-2, if there are signs or symptoms suggestive of infection. 
h See Section 22 (Appendix IV) for list of required laboratory tests at each visit. 
i Assessment may be omitted if an assessment was performed within the previous 3 days. If there are multiple assessments the most recent ones should be used. 
j Perform DTH skin test injections approximately 48 hours (but no sooner than 24 hours) prior to the evaluation. 
k Perform DTH skin test injections immediately before administration of study drug. 
l If the DTH reactivity results in erythema with induration greater than 2 cm, or leads to ulceration of the skin test site, further DTH testing should not be conducted for the patient. 
m Evaluation should be made on the planned dosing day, prior to study drug administration.  
n Evaluation should be approximately 48 hours (but no sooner than 24 hours) after the DTH skin test injection. 
o Samples obtained 6 hours (± 30 minutes) and 48 hours (± 6 hours) postdose. 
p In place of tumor tissue samples from a biopsy during screening, archived tumor tissue samples may be provided. Availability of tumor tissue samples should be determined 
during screening through provision of the accession number or other identification number. If necessary, the tumor tissue sample biopsy should be performed after study 
eligibility has been confirmed and before the first dose of study drug. The tumor tissue samples will only be provided to the sponsor for patients who receive study drug. In place 
of archival tumor tissue samples, patients with AML should have available a bone marrow aspirate and/or bone marrow biopsy with PCR for WT1 transcript performed before 
the first dose of study drug. Patients with AML should also have peripheral blood assessed for blasts and WT1 transcript. 
q IHC need not be performed for patients with AML provided RT-PCR for WT1 transcript is assessed. 
r Tumor assessments (per irRC for solid tumors, modified IWG for AML, and/or standard tumor markers as appropriate, and quantitative RT-PCR for WT1 transcript for AML) 
are not required to be repeated if they have been performed within the 28 days before first dose (there are no variations allowed beyond this window). If multiple assessments are 
available, the most recent ones should be used as the baseline result. Data from at least one additional historical assessment prior to the baseline assessment should also be 
provided, if available. Progressive disease requires confirmation by a repeat, consecutive assessment at least 4 weeks after the date of first documentation, unless rapid clinical 
deterioration is seen. For patients with glioblastoma, progressive disease should additionally be confirmed by SPECT, perfusion MRI, MRI spectroscopy or C-14 methionine 
PET or pathology from available surgical/biopsy specimens to differentiate from pseudoprogression. For patients with AML, bone marrow aspirations/biopsies are to be 
provided at baseline and subsequently when clinically indicated, however peripheral blood should be assessed for blasts and WT1 transcript at baseline and at least every 4 
weeks or more frequently as clinically indicated. 
s Tumor markers drawn from peripheral blood samples should be assessed at least every 4 weeks after the first dose but may be performed more frequently to assess response and 
adjust study drug administration frequency (For other tumor assessments, perform every 8 weeks after first dose or more frequently as clinically indicated). 
t Patients who do not complete the extended dosing in the Induction Phase for reasons other than intercurrent medical conditions may continue in the study and start dosing in the 
Consolidation phase as scheduled on Day 71, at the discretion of the investigator and the sponsor. 

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Table 6: 
Schedule of Assessments for Consolidation Phase, Maintenance Phase, and End of Study – Part 2 
Consolidation Phasea,b,p 
Maintenance 
Phasea,b 
End of Studyb 
Follow-
up 
 
10th dose 
 
11th dose 
 
12th dose 
13th dose 
14th dose 
15th dose 
 
 
 
 
 
Day 71 
(±2) 
Day 83 
Day 85 
(±2) 
Day 
87 
Day 99 
(±2) 
Day113 
(±2) 
Day 127 
(±2) 
Day 141 
(±2) 
every 28 (+7) 
days 
48 
hours 
before 
End of 
Study 
Visit 
within 
28 days 
after 
last dose 
every 3 
months 
Continuation 
criteria 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Xc 
 
 
 
Study drug 
administration 
X 
 
X 
 
X 
X 
X 
X 
X 
 
 
 
Physical exam 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Xc 
 
X 
 
Weight 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Xc 
 
X 
 
Vital Signsd 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Xc 
 
X 
 
ECOG 
performance 
status 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Xc 
 
X 
 
Serum pregnancy 
teste 
 
 
 
 
 
 
 
 
 
 
X 
 
Hematology/ 
serum chemistryf 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Xc 
 
X 
 
Urinalysis 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Xc 
 
X 
 
12-lead ECG 
 
 
Xc 
 
 
Xc 
 
Xc 
Xc 
 
X 
 
DTH skin test 
injection 
 
Xg 
Xg,h 
 
 
 
 
 
 
Xg 
 
 
DTH skin test 
evaluation 
 
 
Xi 
Xj 
 
 
 
 
 
 
Xj 
 
 

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Table 6: 
Schedule of Assessments for Consolidation Phase, Maintenance Phase, and End of Study – Part 2 (Continued) 
Consolidation Phasea,b,p 
Maintenance 
Phasea,b 
End of Studyb 
Follow-
up 
 
10th dose 
 
11th dose 
 
12th dose 
13th dose 
14th dose 
15th dose 
 
 
 
 
 
Day 71 
(±2) 
Day 83 
Day 85 
(±2) 
Day 
87 
Day 99 
(±2) 
Day113 
(±2) 
Day 127 
(±2) 
Day 141 
(±2) 
every 28 (+7) 
days 
48 hours 
before End 
of Study 
Visit 
within 
28 days 
after 
last dose 
every 3 
months 
Blood sample for 
CTL 
Xc 
 
Xc 
 
Xc 
Xc 
Xc 
Xc 
Xc 
 
X 
 
Blood sample for 
Anti-WT1 
antibody 
 
 
Xc 
 
 
Xc 
 
Xc 
Xc,k 
 
X 
 
Blood sample for 
retrospective 
biomarker 
analyses 
 
 
Xc 
 
 
Xc 
 
Xc 
Xc,k 
 
X 
 
Blood sample for 
PBMC isolation 
 
 
 
 
 
 
 
 
 
 
X 
 
IHC tumor 
samples 
 
 
 
 
 
 
 
 
 
 
Xl 
 
Tumor assessment 
 
 
Xc,n 
 
 
Xc,m 
 
Xc,n 
Xc,m 
 
Xm 
 
Adverse events 
 
 
Concomitant 
medications 
 
 
Disease 
Progression/ 
Survival 
 
 
 
 
 
 
 
 
 
 
Xo 
Xo 
Abbreviations: AML = acute myeloid leukemia, CTL = cytotoxic T lymphocyte induction activity, DTH = delayed-type hypersensitivity reactivity, ECG = electrocardiogram, 
ECOG = Eastern Cooperative Oncology Group, FSH = follicle-stimulating hormone, HLA = human leukocyte antigen, IHC = immunohistochemistry, irRC = immune-related 
response criteria, IWG = International Working Group response criteria in acute myeloid leukemia, MRI = magnetic resonance imaging, PBMC = peripheral blood mononuclear 
cell, PET = positron-emission tomography, RT-PCR = reverse transcriptase-polymerase chain reaction, SPECT = single-photon emission computed tomography, WT1 = Wilms’ 
tumor gene product 1 
Footnotes provided on the next page. 

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a Additional dosing windows are provided for patients who may need to recover from intercurrent medical conditions (see Section 8.3.2.2). 
b All patients should complete an End of Study visit within 28 days after the last dose of study drug and prior to the start of alternate antineoplastic therapy. 
c Prior to study drug administration. 
d Includes systolic and diastolic blood pressures, pulse, respirations rate, and oral body temperature. 
e For females of child-bearing potential. 
f See Section 22 (Appendix IV) for list of required laboratory tests at each visit. 
g Perform DTH skin test injections approximately 48 hours (but no sooner than 24 hours) prior to the evaluation. 
h Perform DTH skin test injections immediately before administration of study drug. 
i Evaluation should be made on the planned dosing day, prior to study drug administration. 
j Evaluation should be approximately 48 hours (but no sooner than 24 hours) after the DTH skin test injection. 
k Perform every second dosing day during the Maintenance Phase. 
l Biopsy for tumor tissue samples after the last dose of study drug is not mandatory. Tumor samples will be obtained from all patients who provide consent. IHC need not be 
performed for patients with AML provided RT-PCR for WT1 transcript is assessed. 
m Tumor assessments will be by irRC for solid tumors, modified IWG for AML, and/or standard tumor markers as appropriate, and quantitative RT-PCR for WT1 transcript for 
AML. Perform every 8 weeks after first dose or more frequently as clinically indicated. Tumor markers drawn from peripheral blood samples should be assessed every 4 weeks 
after the first dose. This assessment may be performed ± 7 days of the scheduled assessment but must be completed before the next planned administration of study drug. For 
patients with AML if no other measurable disease then bone marrow aspirations/biopsies should follow the general post-transplant biopsy schedule or other clinical site 
guidelines. Progressive disease requires confirmation by a repeat, consecutive assessment at least 4 weeks after the date of first documentation, unless rapid clinical deterioration 
is seen. For patients with glioblastoma, progressive disease should additionally be confirmed by SPECT, perfusion MRI, MRI spectroscopy or C-14 methionine PET or 
pathology from available surgical/biopsy specimens to differentiate from pseudoprogression. For patients with AML, bone marrow aspirations/biopsies are scheduled when 
clinically indicated, however peripheral blood should be assessed for blasts and WT1 transcript at least every 4 weeks or more frequently as clinically indicated. 
n Tumor markers drawn from peripheral blood samples should be assessed at least every 4 weeks after the first dose but may be performed more frequently (For other tumor 
assessments, perform every 8 weeks after first dose or more frequently as clinically indicated). 
o Following the End of Study visit, patients will be contacted every 3 months to evaluate disease progression, if not already reached, and survival. 
p The Consolidation Phase includes a maximum of 6 doses. Patients who do not complete the extended dosing in the Consolidation Phase for reasons other than intercurrent 
medical conditions may continue in the study and start dosing in the Maintenance phase as scheduled on Day 169, at the discretion of the investigator and the sponsor.

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7.2. 
Dose Level Escalation 
The planned dose level escalation scheme for Part 1 is presented in Figure 3 and for Part 2 is 
presented in Figure 4. 
Figure 3: 
Dose Escalation Scheme – Part 1 
Abbreviations: DLT = dose-limiting toxicity, pts = patients 
a The initial patients in the each dose escalation cohort will be enrolled at least one week apart to evaluate initial safety from the first 2 patients; 
after which, if initial acceptable safety has been observed, there is no requirement to wait between enrolling individual patients within a cohort. 
Figure 4: 
Dose Escalation Scheme – Part 2 
 
 
 
 
 
 
 
 
 
Abbreviations: DLT = dose-limiting toxicity, pts = patients 
a The initial patients in the each dose escalation cohort will be enrolled at least one week apart to evaluate initial safety from the first 2 patients; 
after which, if initial acceptable safety has been observed, there is no requirement to wait between enrolling individual patients within a cohort. 
b If additional patients are ready to enroll in the study prior to completion of the DLT Evaluation Period for the 27.0 mg dose cohort, up to a total 
of 10 patients may be enrolled at the 18.0 mg dose level. At least 4 patients with each malignancy type (glioblastoma and acute myeloid 
leukemia [AML]) will be enrolled at each of these dose levels. If the MTD is reached in Part 2 before the total of 20 patients are enrolled, 
remaining patients will be enrolled at the prior dose level. 
Enroll 
2 to 6 
ptsa 
2 DLT or more 
1DLT  
Enroll to 6 pts 
1 DLT 
out of 6 pts 
 
 
no DLT out of first 3 pts 
∙No-dose escalation 
∙Determine the dose  
 level of ≤1 DLT  
  out of 6 pts 
Next higher dose level
2 DLT or more 
 
 
 
 
 
Enroll 
2 to 6 
ptsa 
 
no DLT out of first 3 pts 
1DLT  
Enroll to 6 pts 
1 DLT 
out of 6 pts 
∙No-dose escalation 
∙Determine the 
dose level of ≤1 
DLT out of 6 pts 
∙Next higher dose 
level  
∙Continue dosing at 
current dose level 
up to 10 ptsb

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The initial patients in each dose escalation cohort will be enrolled at least one week apart to 
evaluate initial safety from the first 2 patients; after which, if initial acceptable safety has been 
observed, there is no requirement to wait between enrolling individual patients within a cohort. 
After completion of DLT evaluation for the first dose cohort, if the study continues, enrollment 
of subsequent patients will be conducted sequentially in subsequent dose escalation cohorts and 
continue until the MTD or all planned cohorts are attained in the dose escalation parts of the 
study. 
The 0.3 mg dose cohort will be conducted first in Part 1. If after 3 patients in this dose cohort 
have completed the DLT Evaluation Period and there are no DLTs identified, enrollment of the 
subsequent cohort will begin. If additional patients are ready to enroll in the study prior to 
completion of the DLT Evaluation Period for the first 3 patients in the 0.3 mg dose cohort, up to 
3 more patients may be enrolled in that cohort. 
Again, in subsequent dose escalation cohorts in Part 1, if 3 patients in a dose cohort complete the 
DLT Evaluation Period and there are no DLTs identified, then the dose level is increased, 
otherwise enrollment of additional patients (up to 3 more) may be permitted in the same cohort. 
If one of 3 patients in a dose escalation cohort experiences a DLT then up to a total of 6 patients 
are enrolled in the same dose cohort and dose escalation may only proceed if no additional 
patients experience DLT. Patients not evaluable for DLT assessment may be replaced. 
In Part 2 of the study, the 18.0 mg cohort will be conducted first. If 3 patients in the dose cohort 
complete the DLT Evaluation Period and there are no DLTs identified, then the 27.0 mg dose 
cohort may begin, otherwise enrollment of additional patients (up to 3 more) may be permitted in 
the same cohort. If one of 3 patients in a dose escalation cohort experiences a DLT then up to a 
total of 6 patients are enrolled in the same dose cohort and dose escalation may only proceed if 
no additional patients experience DLT. After safety and tolerability have been established for the 
18.0 mg cohort, enrollment of subsequent patients will be conducted sequentially in the 27.0 mg 
cohort. If additional patients are ready to enroll in the study prior to completion of the DLT 
Evaluation Period for the 27.0 mg dose cohort, up to a total of 10 patients may be enrolled at the 
18.0 mg dose level. Approximately 10 patients at each dose level (18.0 and 27.0 mg) with at 
least 4 patients with each malignancy type (glioblastoma and AML) will be enrolled. If the MTD 
is reached in Part 2 before the total of 20 patients are enrolled, remaining patients will be 
enrolled at the prior dose level. Patients not evaluable for DLT assessment may be replaced. 
If at any time during dose escalation 2 or more patients experience DLT within a dose cohort that 
suggests that the MTD has been exceeded; no additional patients will be enrolled into that dose 
cohort and dose escalation will end. The MTD will be the dose level below that which resulted in 
2 or more patients experiencing a DLT. If the suspected MTD is based on a cohort in which 
fewer than 6 patients were enrolled then additional patients will be enrolled and treated at that 
dose level until there is a total of 6 patients evaluable for DLT within that dose escalation cohort. 
In the event that a dose level is excluded from further study due to dose limiting toxicity all 
patients may continue the study at the most recently completed dose without DLT, eg, if the 
27.0 mg dose is excluded due to DLT all patients may continue at 18.0 mg. 

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7.2.1. 
Criteria for Dose-limiting Toxicity  
Dose-limiting toxicities are defined as any Grade 3 or greater AE that occurs after the 
administration of study drug during the DLT Evaluation Period that are not related to underlying 
disease, intercurrent illness, or concomitant medications (Changes in hematology parameters 
need to have been confirmed on repeat assessment and constitute at least a 2 grade shift. Grade 3 
AEs of nausea, vomiting, and fatigue that are common and manageable in cancer patients will 
not be considered DLT if they can be ameliorated to < Grade 3 with standard supportive care 
management.). 
The DLT Evaluation Period extends from the day of the first dose of study drug to just prior to 
the fifth dose (Days 1 - 29). No more than 4 doses of study drug will be administered during the 
DLT Evaluation Period. 
In a dose cohort with less than 2 patients with DLT, a minimum of 3 evaluable patients are 
needed to assess dose level escalation. An evaluable patient is defined as a patient who has a 
DLT or a patient who completes the DLT Evaluation Period without the occurrence of a DLT. 
Patients who discontinue from the study for a reason other than DLT prior to completion of the 
DLT Evaluation Period are not evaluable for DLT and will be replaced. 
7.2.2. 
Reporting of Dose-limiting Toxicity or Completion of DLT Evaluation Period 
If a DLT occurs, the investigator or his designee must complete the DLT form with the requested 
information describing the DLT and fax or email the form to Sunovion Pharmaceuticals Inc. or 
its agent within 1 business day of becoming aware of the DLT. 
When a patient completes the DLT Evaluation Period, the investigator or his designee must 
complete the DLT form to confirm no DLT has occurred and fax or email the form to Sunovion 
Pharmaceuticals Inc. or its agent within 1 business day of the patient completing the DLT 
Evaluation Period. 
The DLT form will be provided as a part of the document package necessary to conduct this 
clinical study. 
7.3. 
Dose-Level Escalation Stopping Criteria 
The MTD is defined as the dose at which no more than 1 of 6 patients experiences a DLT when 
2 of 2 to 6 patients experiences a DLT at the next higher dose level. 
Part 1 
Dose level escalation will be completed when the MTD has been determined or the minimum 
number of patients has been enrolled at the highest planned dose level to meet dose escalation or 
MTD criteria (2-6 patients). 
Additional dose escalation cohorts after the initial expansion cohorts may be enrolled if a MTD 
has not been determined and significant signs of toxicity have not been observed in the 
expansion cohorts. 
Part 2 
Dose level escalation will be completed when 20 patients have been enrolled. 

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7.4. 
Dose Adjustment Criteria 
The following sections provide guidelines for intra-patient dose adjustment. Dose level 
escalation among cohorts of patients is described in Section 7.2. 
7.4.1. 
Dose Reduction Criteria 
The following guidelines are provided for dose level reductions: 
• Dose reduction from the lowest dose level (0.3 mg) is not permitted at any time. 
• Patients who experience DLT may remain on the study at a reduced dose, if they are 
deriving some therapeutic benefit from study drug and meet the continuation criteria 
for subsequent dosing (see Section 8.3.2). These decisions will be addressed on a case 
by case basis. The dose will be reduced one level, eg, from 0.9 to 0.3 mg. Patients 
who require reduction from this reduced dose will be discontinued from receiving 
further study drug. 
After the DLT Evaluation Period, if a patient experiences a study drug-related Grade 3 toxicity, 
there will be a review of safety data to determine appropriate dosing for that patient and other 
patients on study, as well as for any subsequently enrolled patients. 
In the event that a Grade 4 or greater toxicity is reported during the study, the sponsor and the 
investigator will conduct a thorough evaluation of the available safety data to decide whether to 
continue enrolling new patients into the study. 
Dose delays are described in Section 8.3.2. 
7.4.2. 
Dose Increases 
Patients who have completed the consolidation phase of their assigned cohort and have not 
experienced a DLT, ≥ Grade 2 study drug-related injection-site reaction, or required a dose 
reduction may have their dose escalated to that of the highest cohort for which safety and 
tolerability have been established. This includes patients from Part 1 of the study having their 
dose escalated to a Part 2 dose. 
7.5. 
Prevention of Missing Data 
In an effort to minimize the number of patients who withdraw consent prior to completion of the 
DLT Evaluation Period, the number of assessments has been limited to those required to collect 
the information needed to address the objectives of the study. 
7.6. 
Study Termination Criteria 
The study may be terminated at any time by the sponsor for any reason. 

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8. 
SELECTION AND WITHDRAWAL OF PATIENTS 
8.1. 
Patient Inclusion Criteria 
8.1.1. 
Patient Inclusion Criteria – Part 1 
To qualify for participation in Part 1 of the study, patients must meet all of the following 
inclusion criteria: 
1. Patient or his or her legal representatives must give written informed consent and privacy 
authorization prior to participation in the study. 
2. Patient must be willing and able to comply with the study procedures and visit schedules 
and must be able to follow verbal and written instructions. 
3. Patient must be ≥ 18 years of age. 
4. Women of childbearing potential and men with female sexual partners of childbearing 
potential must agree to abstain from sexual intercourse or use a double barrier method of 
contraception (eg, condom + diaphragm) for the duration of study participation. Note: 
The definition of menopause from the NCCN Clinical Practice Guidelines for Invasive 
Breast Cancer (Version 1.2012) should be followed to determine if a woman is of 
childbearing potential 
(http://www.nccn.org/professionals/physician_gls/f_guidelines.asp). 
5. Patient must have an Eastern Cooperative Oncology Group (ECOG) Performance Score 
of 0, 1, or 2 (refer to Appendix II). 
6. Patient has a life expectancy of at least 4 months. 
7. Patient must have one of the following histologically or cytologically documented 
measurable (may be measurable by tumor markers only, such as quantitative RT-PCR for 
WT1 transcript for AML, or CA-125 for ovarian carcinoma) advanced stage 
malignancies: non-small cell lung, ovarian, glioblastoma, and AML (not including acute 
promyelocytic leukemia), known to overexpress the WT1 protein. Note: Determination of 
WT1 expression will not be assessed prior to patient enrollment. 
8. Patient must have an advanced stage malignancy defined as meeting at least one of the 
following criteria: 
• progressed or recurred despite standard therapy 
• no standard therapy exists 
• patient is intolerant of standard therapy 
• patient is not a candidate for standard therapy 
9. Patient must be HLA-A*0201+ and/or HLA-A*0206+ 
10. Patient must have adequate bone marrow and immune reserve, as documented by: 
• Absolute neutrophil count (ANC) ≥ 1,000/µl 

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• Platelet count ≥ 10.0 x 104/µl (≥ 5.0 x 104/µl after stem cell transplant) 
• Hemoglobin ≥ 9.0 g/dL 
• Absolute lymphocyte count (ALC) ≥ 1,000/µl (≥ 500/µl after stem cell transplant) 
Note: After completion of dose escalation, patients with AML are not required to meet 
these hematologic criteria. 
11. Patient must have adequate renal function documented by a serum creatinine of 
≤ 1.5 times the upper limit of normal (ULN) for the reference lab. 
12. Patient must have adequate hepatic function documented by a total bilirubin of 
≤ 2.0 mg/dl (≤ 3.0 mg/dl for patients with known Gilbert’s syndrome) and alanine 
aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 times the ULN for the 
reference lab. 
13. Patient must have access to archival tumor tissue sample or agree to undergo biopsy after 
study eligibility has been confirmed to obtain fresh sample for evaluation of WT1 
expression. In place of archival tumor tissue samples, patients with AML should have 
available a bone marrow aspirate and/or bone marrow biopsy with PCR for WT1 
transcript performed before the first dose of study drug. Note: The archived tumor tissue 
sample does not need to be delivered to the clinical site prior to enrollment of the patient, 
however its availability should be confirmed through provision of the accession number 
or other identification number. 
8.1.2. 
Patient Inclusion Criteria - Part 2 
To qualify for participation in Part 2 of the study, patients must meet all of the following 
inclusion criteria: 
1. Patient or his or her legal representatives must give written informed consent and privacy 
authorization prior to participation in the study. 
2. Patient must be willing and able to comply with the study procedures and visit schedules 
and must be able to follow verbal and written instructions. 
3. Patient must be ≥ 18 years of age. 
4. Women of childbearing potential and men with female sexual partners of childbearing 
potential must agree to abstain from sexual intercourse or use a double barrier method of 
contraception (eg, condom + diaphragm) for the duration of study participation. Note: 
The definition of menopause from the NCCN Clinical Practice Guidelines for Invasive 
Breast Cancer (Version 1.2012) should be followed to determine if a woman is of 
childbearing potential 
(http://www.nccn.org/professionals/physician_gls/f_guidelines.asp). 
5. Patient must have an ECOG Performance Score of 0, 1, or 2 (refer to Appendix II). 
6. Patient has a life expectancy of at least 4 months.  
7. Patient must have histologically or cytologically documented measurable (may be 
measurable by tumor markers only, such as quantitative RT-PCR for WT1 transcript for 
AML) advanced stage glioblastoma or AML (not including acute promyelocytic 

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leukemia), known to overexpress the WT1 protein. Note: Determination of WT1 
expression will not be assessed prior to patient enrollment. 
8. Patient must have an advanced stage malignancy defined as meeting at least one of the 
following criteria: 
• progressed or recurred despite standard therapy 
• no standard therapy exists 
• patient is intolerant of standard therapy 
• patient is not a candidate for standard therapy 
9. Patient must be HLA-A*0201+ and/or HLA-A*0206+. 
10. Patient with glioblastoma must have adequate bone marrow and immune reserve, as 
documented by: 
• ANC ≥ 100/µl (≥ 500/µl after stem cell transplant) 
• Platelet count ≥ 10.0 x 104/µl (≥ 5.0 x 104/µl after stem cell transplant) 
• Hemoglobin ≥ 9.0 g/dL 
• ALC ≥ 900/µl 
Note: Patients with AML are not required to meet these hematologic criteria. 
11. Patient must have adequate renal function documented by a serum creatinine of 
≤ 1.5 times the ULN for the reference lab. 
12. Patient must have adequate hepatic function documented by a total bilirubin of 
≤ 2.0 mg/dl (≤ 3.0 mg/dl for patients with known Gilbert’s syndrome) and ALT and 
AST ≤ 3 times the ULN for the reference lab. 
13. Patient must have access to archival tumor tissue sample or agree to undergo biopsy after 
study eligibility has been confirmed to obtain fresh sample for evaluation of WT1 
expression. In place of archival tumor tissue samples, patients with AML should have 
available a bone marrow aspirate and/or bone marrow biopsy with PCR for WT1 
transcript performed before the first dose of study drug. Note: The archived tumor tissue 
sample does not need to be delivered to the clinical site prior to enrollment of the patient, 
however its availability should be confirmed through provision of the accession number 
or other identification number. 
14. Patients with AML must be willing to undergo bone marrow aspiration/biopsy during 
treatment if there are no other indicators of measureable disease. 
8.2. 
Patient Exclusion Criteria 
8.2.1. 
Patient Exclusion Criteria – Part 1 
To qualify for participation in Part 1 of the study, patients must not meet any of the following 
exclusion criteria: 
1. Patient has an extensively disseminated primary glioblastoma. 

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2. Patient has symptomatic brain metastases, ie, presence of neurological symptoms or 
requiring treatment with corticosteroids, or central nervous system (CNS) leukemia. 
3. Patient has an infection requiring treatment with systemic antibiotics or antiviral 
medication or has completed treatment for such an infection within 4 days prior to 
planned first dose of study drug. 
4. Patient requires systemic, pharmacologic doses of corticosteroids (equivalent to > 60 mg 
hydrocortisone/day or 2 mg dexamethasone/day). Replacement doses (equivalent 
to ≤ 5 mg prednisone/day), and topical, ophthalmic, and inhalation steroids are permitted 
as needed. 
5. Patient has a positive test for Hepatitis B surface antigen, Hepatitis C antibody, human 
immunodeficiency virus (HIV)-1, or HIV-2 antibody, or has a history of a positive result. 
6. Patient has received any of the following treatments within the specified timeframe: 
• endocrine therapy, immunotherapy, transfusion, or hematopoietic factors within 
14 days prior to planned first dose of study drug (Note: After completion of dose 
escalation, patients with AML are not required to meet these hematologic criteria, eg. 
transfusions and hematopoietic growth factors.), 
• chemotherapy including molecular-targeting therapy within 21 days prior to planned 
first dose of study drug (for molecular-targeted agents that are not associated with 
myelosuppression or immunosuppression, the minimum interval is 5 half-lives if that 
is less than 21 days), 
• surgery, radiation, or immunosuppressants within 28 days prior to planned first dose 
of study drug, 
• investigational drug within the 28 days prior to planned first dose of study drug, or 
• mitomycin-C or nitrosoureas within 42 days prior to planned first dose of study drug. 
Note: Patient receiving luteinizing hormone-releasing hormone [LHRH] agonists or 
antagonists or antiestrogens or aromatase inhibitors started and at a stable dose for at 
least 90 days prior to planned first dose of study drug is eligible. 
7. Patient has an unresolved ≥ Grade 2 AE from a previous antineoplastic treatment, 
excluding alopecia. 
8. Woman who is pregnant or lactating or has a positive pregnancy test at screening. If a 
woman has a positive pregnancy test, further evaluation may be conducted to rule out 
ongoing pregnancy to allow the patient to be eligible. 
9. Patient has an autoimmune condition, including, but not limited to, multiple sclerosis, 
Grave’s disease, vasculitis, systemic lupus erythematosus, rheumatoid arthritis, systemic 
sclerosis, myasthenia gravis, ankylosing spondylitis, Wegener’s granulomatosis, 
ulcerative colitis, Crohn’s disease, psoriasis requiring systemic therapy, pemphigus, 
temporal arteritis, dermatomyositis, Sjögren’s syndrome, Goodpasture’s syndrome, 
interstitial pneumonitis, interstitial nephritis, or Henoch-Schönlein purpura. 
10. Patient has in the opinion of the investigator any intercurrent conditions that could 
preclude their participation in the study, pose an undue medical hazard, or that could 

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interfere with the interpretation of the study results, including, but not limited to, patients 
with congestive heart failure (New York Heart Association [NYHA] Class III or IV; refer 
to Appendix III), unstable angina, cardiac arrhythmia requiring treatment, recent (within 
the prior 6 months) myocardial infarction, acute coronary syndrome or stroke, severe 
obstructive pulmonary disease, hypertension requiring more than 2 medications for 
adequate control, or diabetes mellitus with more than 2 episodes of ketoacidosis in the 
prior 12 months. 
11. Patient has pleural effusion, ascites, or pericardial fluid requiring drainage. Note: Patient 
who had drain removal ≥ 14 days prior to planned first dose of study drug and has no sign 
of worsening is eligible. 
12. Patient has any other medical, psychiatric, or social condition, including substance abuse 
that in the opinion of the investigator would preclude participation in the study. 
13. Patient has had previous treatment with the study drug or other WT1-related vaccine 
therapy. 
14. Patient has a known hypersensitivity to any of the components of the study drug. 
15. Patient is a staff member of the sponsor or clinical site and is involved in the conduct of 
the study or the relative of such a staff member. 
8.2.2. 
Patient Exclusion Criteria – Part 2 
To qualify for participation in Part 2 of the study, patients must not meet any of the following 
exclusion criteria: 
1. Patient has an extensively disseminated primary glioblastoma. 
2. Patient has symptomatic brain metastases, ie, presence of neurological symptoms or 
requiring treatment with corticosteroids, or CNS leukemia. 
3. Patient has an infection and has had a body temperature of > 38.3˚C within 48 hours prior 
to planned first dose of study drug. 
4. Patient requires systemic, pharmacologic doses of corticosteroids (equivalent to > 60 mg 
hydrocortisone/day or 2 mg dexamethasone/day). Replacement doses (equivalent 
to ≤ 5 mg prednisone/day), and topical, ophthalmic, and inhalation steroids are permitted 
as needed. 
5. Patient has a positive test for Hepatitis B surface antigen, Hepatitis C antibody, HIV-1, or 
HIV-2 antibody, or has a history of a positive result. 
6. Patient has received any of the following treatments within the specified timeframe: 
• endocrine therapy, immunotherapy, transfusion, or hematopoietic factors within 
14 days prior to planned first dose of study drug (Note: Patients with AML are not 
required to meet these hematologic criteria, eg, transfusions and hematopoietic 
growth factors.), 
• chemotherapy including molecular-targeting therapy within 21 days prior to planned 
first dose of study drug (for molecular-targeted agents that are not associated with 

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myelosuppression or immunosuppression, the minimum interval is 5 half-lives if that 
is less than 21 days), 
• surgery, radiation, or immunosuppressants within 28 days prior to planned first dose 
of study drug, 
• investigational drug within the 28 days prior to planned first dose of study drug, or 
• mitomycin-C or nitrosoureas within 42 days prior to planned first dose of study drug. 
Note: Patient receiving LHRH agonists or antagonists or antiestrogens or aromatase 
inhibitors started and at a stable dose for at least 90 days prior to planned first dose of 
study drug is eligible. Patients are permitted one 28 day cycle of concurrent treatment 
with hydroxyurea during the study. 
7. Patient has an unresolved ≥ Grade 2 AE from a previous antineoplastic treatment, 
excluding alopecia. 
8. Woman who is pregnant or lactating or has a positive pregnancy test at screening. If a 
woman has a positive pregnancy test, further evaluation may be conducted to rule out 
ongoing pregnancy to allow the patient to be eligible. 
9. Patient has an autoimmune condition, including, but not limited to, multiple sclerosis, 
Grave’s disease, vasculitis, systemic lupus erythematosus, rheumatoid arthritis, systemic 
sclerosis, myasthenia gravis, ankylosing spondylitis, Wegener’s granulomatosis, 
ulcerative colitis, Crohn’s disease, psoriasis requiring systemic therapy, pemphigus, 
temporal arteritis, dermatomyositis, Sjögren’s syndrome, Goodpasture’s syndrome, 
interstitial pneumonitis, interstitial nephritis, or Henoch-Schönlein purpura. 
10. Patient has in the opinion of the investigator any intercurrent conditions that could 
preclude their participation in the study, pose an undue medical hazard, or that could 
interfere with the interpretation of the study results, including, but not limited to, patients 
with congestive heart failure (NYHA Class III or IV; refer to Appendix III), unstable 
angina, cardiac arrhythmia requiring treatment, recent (within the prior 6 months) 
myocardial infarction, acute coronary syndrome or stroke, severe obstructive pulmonary 
disease, hypertension requiring more than 2 medications for adequate control, or diabetes 
mellitus with more than 2 episodes of ketoacidosis in the prior 12 months. 
11. Patient has pleural effusion, ascites, or pericardial fluid requiring drainage. Note: Patient 
who had drain removal ≥ 14 days prior to planned first dose of study drug and has no sign 
of worsening is eligible. 
12. Patient has any other medical, psychiatric, or social condition, including substance abuse 
that in the opinion of the investigator would preclude participation in the study.  
13. Patient has had previous treatment with the study drug or other WT1-related vaccine 
therapy. 
14. Patient has a known hypersensitivity to any of the components of the study drug. 
15. Patient is a staff member of the sponsor or clinical site and is involved in the conduct of 
the study or the relative of such a staff member. 

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8.3. 
Continuation Criteria 
8.3.1. 
Enrollment – Part 1 and Part 2 
In order to receive the first dose of study drug on Day 1, patients must continue to meet all 
inclusion (see Section 8.1) and no exclusion criteria (see Section 8.2). 
8.3.2. 
Subsequent Dosing 
8.3.2.1. 
Subsequent Dosing – Part 1 
Patients are eligible to continue dosing at each subsequent dosing day if they meet the following 
criteria, have not met any of the discontinuation criteria (see Section 12.2), and can receive the 
next dose of study drug during the prespecified timeframe: 
• ANC ≥ 750/µl 
• Platelet count ≥ 5.0 x 104/µl (≥ 3.5 x 104/µl after stem cell transplant) 
• All non-hematologic toxicity resolved to at least Grade 2 or to pretreatment levels 
Note: After completion of dose escalation, patients with AML are not required to meet these 
hematologic criteria. 
For doses 2 through 5, the subsequent dose may be delayed 1 week (ie, the subsequent dose 
should be administered within 8 days after the intended dosing date inclusive of the day of 
planned dosing) to allow patients to recover from intercurrent medical conditions and meet these 
continuation criteria. 
For doses 6 and beyond, the subsequent dose may be delayed 2 weeks (ie, the subsequent dose 
should be administered within 15 days after the intended dosing date inclusive of the day of 
planned dosing) to allow the patient to recover from intercurrent medical conditions and meet 
these continuation criteria. 
Patients who cannot receive the next dose of study drug within the specified timeframes provided 
above must be discontinued from receiving further study drug. 
8.3.2.2. 
Subsequent Dosing – Part 2 
Patients are eligible to continue dosing at each subsequent dosing day if they meet the following 
criteria, have not met any of the discontinuation criteria (see Section 12.2), and can receive the 
next dose of study drug during the prespecified timeframe: 
• ANC ≥ 750/µl (≥ 500/µl after stem cell transplant) 
• Platelet count ≥ 5.0 x 104/µl (≥ 3.5 x 104/µl after stem cell transplant) 
• All non-hematologic toxicity resolved to at least Grade 2 or to pretreatment levels 
Note: Patients with AML are not required to meet these hematologic criteria. 
During the Induction Phase, the subsequent dose may be delayed 1 week (ie, the subsequent dose 
should be administered within 8 days after the intended dosing date inclusive of the day of 
planned dosing) to allow patients to recover from intercurrent medical conditions and meet these 
continuation criteria. 

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During the Consolidation and Maintenance Phases, the subsequent dose may be delayed 2 weeks 
(ie, the subsequent dose should be administered within 15 days after the intended dosing date 
inclusive of the day of planned dosing) to allow the patient to recover from intercurrent medical 
conditions and meet these continuation criteria. 
Patients who cannot receive the next dose of study drug within the specified timeframes provided 
above may continue in the study at the discretion of the investigator and the sponsor. 
Patients who do not complete the extended dosing in the Induction Phase or the Consolidation 
Phase for reasons other than intercurrent medical conditions may continue in the study and start 
dosing in the next phase as scheduled at the discretion of the investigator and the sponsor. 

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9. 
TREATMENT OF PATIENTS 
9.1. 
Concomitant Medications 
Concomitant medication and therapy guidelines during the study are the same during Part 1 and 
Part 2 of the study except as indicated for hydroxyurea. 
9.1.1. 
Disallowed Medications During Study 
Due to the possibility of affecting the evaluation of safety or response to study drug, use of any 
of the following medications is prohibited during study participation from screening through the 
End of Study visit, unless other timing is noted. 
• All other antineoplastic therapy including systemic radiopharmaceuticals, with the 
exception of LHRH agonists or antagonists or antiestrogens or aromatase inhibitors 
started and at a stable dose for at least 90 days prior to the planned first dose of study 
drug. See Section 9.1.2 regarding hydroxyurea use in Part 2 of the study. 
• Other investigational agents 
• Pharmacologic doses of systemic corticosteroids (equivalent to > 60 mg 
hydrocortisone/day or 2 mg dexamethasone/day). Note: Replacement doses 
(equivalent to ≤ 5 mg prednisone/day), and topical, ophthalmic, and inhalation 
corticosteroids are permitted as needed. 
• Immunomodulatory therapy 
• Systemic immunosuppressive therapy 
• Treatment aimed at preventing the induction of AEs by the study drug. Note: 
Treatment to prevent the recurrence of an AE that has occurred previously on study is 
permitted. 
9.1.2. 
Allowed Medications During Study 
All intercurrent medical conditions should be treated by the investigator according to current 
community standards of care. Treatment to prevent the recurrence of an AE that has occurred 
previously on study is permitted. Patients may also receive medications for symptomatic relief, 
such as analgesics, laxatives, anti-emetics, hypnotics, etc. 
Replacement doses of steroids (equivalent to ≤ 5 mg prednisone/day), and topical, ophthalmic, 
and inhalation corticosteroids are permitted as needed. 
Local injection site reactions may be treated with dermal steroid treatment or topical nonsteroidal 
anti-inflammatory drugs. 
In addition, in Part 2 of the study, one 28-day cycle of concurrent treatment with hydroxyurea is 
permitted. 

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9.1.3. 
Prohibited Therapies 
Due to the possibility of affecting the evaluation of safety or response to study drug, use of any 
of the following therapies is prohibited during study participation from screening through the 
End of Study visit. 
• Surgery for the treatment of neoplastic disease. Note: Palliative surgery for 
symptomatic lesion(s) is permitted as long as the lesion is not the only evaluable one. 
• Radiation therapy for the treatment of neoplastic disease. Note: Palliative radiation 
therapy for symptomatic lesion(s) is permitted as long as the lesion is not the only 
evaluable one. 
• Hyperthermia/thermotherapy for the treatment of neoplastic disease 
9.1.4. 
Allowed Therapies 
Palliative surgery and radiation therapy for symptomatic lesion(s) are permitted during the study 
as long as the lesion is not the only evaluable one. Study drug administration should be 
interrupted during and following these therapies until the patient has adequately recovered from 
the effects of treatment, in the investigator’s judgment. 
Transfusions are permitted. 
9.2. 
Treatment Compliance 
All patients will be administered study drug in-clinic; therefore treatment compliance will be 
documented by site personnel. 

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10. 
STUDY DRUG MATERIALS AND MANAGEMENT 
10.1. 
Description of Study Drug 
The study drug contains 4 components that may be diluted prior to administration: 
1. WT2725 Injection 50 mg/mL 
2. WT2725 Injection 2 mg/mL 
3. Diluting Solution for WT2725 Injection (DSWI) 
4. W/O pre-Emulsion (WOPE) 
WT2725 Injection 50 mg/mL and WT2725 Injection 2 mg/mL are sterile aqueous solutions 
containing WT2725, L-Methionine, Tartaric Acid, Sodium Hydroxide, and Water for Injection, 
and may also contain Hydrochloric Acid. 
Diluting Solution for WT2725 Injection (DSWI) is a sterile aqueous solution containing 
L-Methionine, Tartaric Acid, Sodium Hydroxide, and Water for Injection, and may also contain 
Hydrochloric Acid. 
W/O pre-Emulsion (WOPE) is a sterile water-in-oil emulsion containing Ethyl Oleate, 
2-Octyldodecyl Myristate, Sorbitan Monooleate, Glyceryl Monooleate, Polyoxyl 20 
Hydrogenated Castor Oil, Concentrated Glycerin, Sodium Dihydrogen Phosphate Dihydrate, and 
Water for Injection. 
10.2. 
Study Drug Packaging and Labeling 
WT2725 Injection (2 mg/mL and 50 mg/mL), Diluting Solution for WT2725 Injection (DSWI), 
and W/O pre-Emulsion (WOPE) are packaged in clear type 1 glass vials. The closure is a rubber 
stopper that forms a tight seal when crimp sealed with an aluminum over seal. 
Each vial is labeled with a 1-panel open label. Fifty vials of each component are packaged into 
cartons with protective inserts. The cartons are also labeled with a 1-panel open label. 
The vial labels for each of the 4 components will include the following information at a 
minimum: 
• Protocol number 
• Sponsor’s name and address 
• Content 
• Investigational New Drug statement 
• Instructions for use and storage 
• Lot number 

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The carton labels for each of the 4 components will be labeled with the following information at 
a minimum: 
• Protocol number 
• Sponsor’s name and address 
• Content 
• Investigational New Drug statement 
• Instructions for use and storage 
• Lot number 
• Blank space for site number and date opened 
10.3. 
Study Drug Storage 
All study drug components (WT2725 Injection 50 mg/mL, WT2725 Injection 2 mg/mL, Diluting 
Solution for WT2725 Injection [DSWI], and W/O pre-Emulsion [WOPE]) should be stored 
under refrigeration (cold temperature 2°C to 8°C), and protected from light and freezing. 
10.4. 
Study Drug Preparation 
At the clinical site, WT2725 Injection (2 mg/mL and/or 50 mg/mL) will be diluted to an 
appropriate concentration using DSWI, if needed. Subsequently, WT2725 Injection or diluted 
WT2725 Injection will be mixed with the WOPE at a ratio of 3:7 (by volume) to form the 
WT2725 Dosing Emulsion (Figure 5). WT2725 Dosing Emulsion is a white liquid emulsion and 
will be administered by sc injection. 
 
Figure 5: 
Description of WT2725 Dosing Emulsion (Drug Product) Preparation 
 
 
The following dosages are planned for Part 1 of the study: 
• for DTH skin tests only - 0.01 mg WT2725 in diluted WT2725 Injection (0.05 mL x 1 
site, id) and Control (Diluting Solution for WT2725 Injection [DSWI]) (0.05 mL x 1 
site, id) 
• 0.3 mg WT2725 in Dosing Emulsion (0.3 mL x 2 sites for a total volume of 0.6 mL 
per patient, sc) 
WT2725 Injection 
or 
diluted WT2725 Injection 
W/O pre-Emulsion 
(WOPE) 
Dosing Emulsion 
Mix before use 
Water phase 
(contains WT2725) 
＋
Oil phase 
3 
7 

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• 0.9 mg WT2725 in Dosing Emulsion (0.3 mL x 2 sites for a total volume of 0.6 mL 
per patient, sc) 
• 3.0 mg WT2725 in Dosing Emulsion (0.3 mL x 2 sites for a total volume of 0.6 mL 
per patient, sc) 
• 9.0 mg WT2725 in Dosing Emulsion (0.3 mL x 2 sites for a total volume of 0.6 mL 
per patient, sc) 
The following dosages are planned for Part 2 of the study: 
• 18.0 mg WT2725 in Dosing Emulsion (0.6 mL x 2 sites for a total volume of 1.2 mL 
per patient, sc) 
• 27.0 mg WT2725 in Dosing Emulsion (0.9 mL x 2 sites for a total volume of 1.8 mL 
per patient, sc) 
All drug products must be prepared and stored under ambient conditions on the day of dosing, 
and used or discarded within 6 hours following preparation of the drug product. A brief 
description of study drug preparation is provided in Appendix VI. 
10.5. 
Study Drug Administration 
All open-label study drug will be administered at the study site by trained staff. 
In Part 1 of the study, after safety and tolerability have been established for the 0.3 mg dose 
cohort (following completion of DLT Evaluation Period), enrollment of subsequent patients will 
be conducted sequentially in subsequent dose escalation cohorts and continue until the MTD or 
all planned cohorts are attained. This dose is considered the RP2D, and subsequent patients 
enrolled will initiate treatment at this dose. Additional dose escalation cohorts after the initial 
expansion cohorts may be enrolled if a MTD has not been determined and significant signs of 
toxicity have not been observed in the expansion cohorts. In Part 2 of the study, the 18.0 mg 
cohort will be conducted first. After safety and tolerability have been established for the 18.0 mg 
cohort, enrollment of subsequent patients will be conducted sequentially in the 27.0 mg cohort. If 
additional patients are ready to enroll in the study prior to completion of the DLT Evaluation 
Period for the 27.0 mg dose cohort, up to a total of 10 patients may be enrolled at the 18.0 mg 
dose level. 
Each dose of study drug will be administered using 2 injection sites (eg, 0.3 mL/site x 2 for the 
9.0 mg dose). The only restriction on the dosing site is that the forearm used for the DTH skin 
tests should not be used for administration of study drug. Wherever possible, injections should be 
administered into the area surrounding the regional lymph nodes in the upper arm, the lower 
abdomen, or the femoral area. Rotation of injection sites is permitted. Injection sites should not 
be chosen in the area of any ongoing skin condition, eg, area of psoriasis. 
Each part of the study will include 3 treatment phases based on intended timing of study drug 
dosing as indicated below: 
Part 1: 
• Vaccine Induction Phase: once every 7 days for 4 weeks (doses 1 - 5) 
• Consolidation Phase: once every 14 days for 6 weeks (doses 6 - 9) 

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• Maintenance Phase: once every 28 days until discontinuation (doses 10 and 
thereafter) 
Part 2: 
• Vaccine Induction Phase: once every 7 days for 8 weeks (doses 1 - 9) 
• Consolidation Phase: once every 14 days for 10 weeks (doses 10 – 15 [maximum 
6 doses]) 
• Maintenance Phase: once every 28 days until discontinuation (dose 16 and thereafter) 
10.5.1. 
Assignment of Dose Cohort for Each Patient 
The following steps will be followed to assign the dose cohort for each patient in the study. 
• Once the clinical site has identified a patient for the study, before screening begins, 
the Investigator or his designee must complete the Pre-screening log and fax or email 
the form to Sunovion Pharmaceuticals Inc. or its agent. 
• When a patient completes Screening, the Investigator or his designee must complete 
the Study Registration form and fax or email the form to Sunovion Pharmaceuticals 
Inc. or its agent. 
• Sunovion Pharmaceuticals Inc. or its agent will provide authorization to enroll a 
patient by providing information regarding the appropriate dose cohort and then the 
clinical site will complete the Registration form to confirm assigned dose cohort and 
planned date of first dose and fax or email the form to Sunovion Pharmaceuticals Inc. 
or its agent 
The Registration form will be provided as a part of the document package necessary to conduct 
this clinical study. 
10.5.2. 
Selection of Dose Cohort for Each Patient 
The selection of dose cohort for each patient during the dose escalation of Part 1 of the study is 
made based on the number of DLTs, if any, that have been observed in the current dose cohort, 
the number of patients enrolled in the current dose cohort, and the duration of their enrollment 
(eg, whether the DLT Evaluation Period is complete). After the RP2D is determined in Part 1 of 
the study, subsequent patients enrolled in Part 1 will initiate treatment at this dose. Selection of 
dose cohort for patients in Part 2 of the study is made based on the number of DLTs, if any, that 
have been observed in the current dose cohort, the number of patients enrolled in the current 
dose cohort, and the duration of their enrollment (eg, whether the DLT Evaluation Period is 
complete). 
Upon receipt of the Study Registration form for a patient, a review of available enrollment 
information and occurrence of DLTs, if any, will be conducted to decide the appropriate dose 
cohort for the patient. 
Meetings to review study execution and enrollment/dosing decisions will be held at least 
monthly between the sponsor, clinical sites, and the medical monitor. Instructions regarding 
intra-patient dose changes are described in Section 7.4.2. 

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10.6. 
Study Drug Accountability 
The Investigator is responsible for storing the study drug in a secure location and for maintaining 
adequate records of drug disposition that includes the dates, quantity, and use by patients. If the 
study is terminated, discontinued, suspended, or completed, all unused supplies of drug will be 
returned to Sunovion Pharmaceuticals Inc., unless other instructions are provided in writing by 
Sunovion Pharmaceuticals Inc. or its agent. 
The study drug will not be dispensed to any person who is not a study patient under this protocol. 
10.7. 
Study Drug Handling and Disposal 
A drug inventory record will be supplied. The Investigator on an ongoing basis must maintain a 
drug inventory record, using forms supplied by the Sponsor or another system that meets the 
requirements of Good Clinical Practice (GCP), of supplied, received, dispensed, and returned 
study drug. The Investigator is required to return all unused study drug to the sponsor or 
designee as instructed. The Investigator is required to maintain copies of study drug shipping 
receipts, drug accountability records, and records of return or final disposal of study drug. 

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11. 
TREATMENT PLAN  
11.1. 
Study Assessments 
11.1.1. 
Safety 
Safety assessments will be conducted at each study visit as indicated on the Schedules of 
Assessments (Tables 3 - 6) and will include the spontaneous reporting of AEs, physical 
examinations, determinations of vital signs, body weight measurements, ECGs, and clinical 
laboratory determinations including complete blood count, serum chemistries, and urinalysis. 
Careful monitoring for signs of local injection site reactions will be performed during the study. 
In addition, these assessments will be used to describe the DLT and determine the MTD. The 
definition of DLT is provided in Section 7.2.1 and the determination of MTD is discussed in 
Section 7.3. 
11.1.2. 
Efficacy 
Tumor response will be evaluated according to the irRC (Wolchok-2009, Hoos-2010), modified 
IWG based on Cheson-2003, and/or tumor markers, as appropriate for the diagnosis. An 
evaluable patient for response is defined as a patient who has had at least one tumor assessment 
conducted 8 weeks after the first dose of study drug or a patient who has progressive disease 
prior to this time point. 
Survival also will be assessed. 
11.1.2.1. Immune-related Response Criteria (irRC) 
The irRC was chosen because immunotherapeutic agents produce antitumor effects by inducing 
an immune response specific to cancer cells or by modifying native immune processes that can 
result in clinical response after an initial increase in tumor burden or the appearance of new 
lesions, which is unique from cytotoxic agents. 
The use of radiographic procedures, physical examinations, and measurement of tumor markers 
for evaluation of response will be performed based on the presence of known areas of disease 
and current symptoms. Unless there are compelling reasons otherwise, the tumor assessments 
during the study will be conducted under the same conditions (eg, slice thickness, use of a 
contrast agent, etc) and using the same examination method (eg, CT, MRI) as used for the 
pretreatment assessment. 
Tumor assessments will be conducted during screening, and then every 8 weeks after the first 
dose of study drug, unless clinical indications require more frequent assessment. Tumor 
assessments are first conducted after 8 weeks to allow adequate time for immune activation and 
consequent antitumor responses. All tumor assessments performed on the day of study drug 
administration must be completed before study drug is administered. 
Tumor response is based on total measurable tumor burden. For the irRC, index and measurable 
new lesions are taken into account. A measurable lesion is defined as 5 × 5 mm or more on 
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At the pretreatment tumor assessment, the sum of the products of the 2 largest perpendicular 
diameters (SPD) of all index lesions (5 lesions per organ, up to 10 visceral lesions, and 
5 cutaneous index lesions) is calculated. 
At each subsequent tumor assessment, the SPD of the index lesions and of new, measurable 
lesions (≥ 5 × 5 mm; up to 5 new lesions per organ; 5 new cutaneous lesions; and 10 visceral 
lesions) are added together to provide the total tumor burden: 
Tumor Burden = SPDindex lesions + SPDnew, measurable lesions 
Percentage changes in tumor burden per assessment time point describe the size and growth of 
both baseline and new, measurable lesions as they appear. At each tumor assessment, the 
response in index and new, measurable lesions is defined based on the change in tumor burden 
(after ruling out immune-related progressive disease [irPD]). Decreases in tumor burden must be 
assessed relative to pretreatment measurements (ie, the SPD of all index lesions at screening). 
Overall response will be determined by the investigator according to the irRC response 
assessments based on tumor burden as follows: 
• irCR (immune-related complete response) = complete disappearance of all lesions 
(whether measurable or not, and no new lesions) confirmation by a repeat, 
consecutive assessment 6 weeks after the date of first documentation 
•  irPR (immune-related partial response) = decrease in tumor burden ≥ 50% relative to 
baseline confirmed by a consecutive assessment 6 weeks after the date of first 
documentation 
• irSD (immune-related stable disease) = not meeting criteria for irCR or irPR and the 
absence of irPD 
• irPD (immune-related progressive disease) = increase in tumor burden ≥ 25% relative 
to nadir (prior minimum recorded tumor burden during the study) confirmation by a 
repeat, consecutive assessment at least 4 weeks after the date of first documentation 
Note: For patients with glioblastoma, measurements should include enhancing tumor only, 
nonenhancing lesions (T2/FLAIR) should be recorded as improved, stable, or significant 
increase. 
Note: For patients with glioblastoma, progressive disease should additionally be confirmed by 
single-photon emission computed tomography (SPECT), perfusion MRI, MRI spectroscopy or 
C-14 methionine positron-emission tomography (PET) or pathology from available 
surgical/biopsy specimens to differentiate from pseudoprogression. For patients with ovarian 
cancer that express CA-125, progressive disease should additionally be confirmed by CA-125 
(≥ 2x higher of ULN or on study nadir). 
Note: For irPD confirmation, follow-up with observation alone (during the weeks before 
confirmation) may not be appropriate for patients with a rapid decline in ECOG performance 
status or other signs of rapid clinical deterioration. The investigator should use his best judgment 
in determining if sooner tumor assessment is required in these patients. 
Note: Patients with progressive disease based on tumor assessments who are experiencing 
clinical benefit in the investigator’s opinion may continue on study and undergo repeat tumor 

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assessments. If subsequent stabilization or improvement is observed the patient may continue in 
the study. 
11.1.2.2. Modified International Working Group response criteria in acute myeloid 
leukemia (IWG) 
The modified International Working Group (IWG) response criteria were developed to assess the 
activity of drugs in AML. The criteria are as follows: 
• Complete remission (CR): 
• Morphologic CR 
< 5% Marrow blasts in an aspirate with spicules – patient independent of 
transfusions 
 
Absolute neutrophil count > 1000/μL 
 
Platelets > 100,000/μL 
 
No residual evidence of extramedullary disease 
• Cytogenetic CR- cytogenetics normal (in patients with previously abnormal 
cytogenetics) 
• Molecular CR- molecular studies negative 
• CRi - < 5% marrow blasts but with persistence of cytopenias (ie, absolute neutrophil 
count < 1000 /μL and/or platelets < 100,000/μL) 
• Morphologic leukemia free state (MLFS) - bone marrow blasts < 5% 
• Partial remission: 
Decrease of at least 50% in the percentage of blasts to 5% to 25% in the bone 
marrow aspirate and the normalization of blood counts, as noted above. 
• PRi:  
Decrease of at least 50% in the percentage of blasts to 5% to 25% in the bone 
marrow aspirate but with persistence of cytopenias (ie, absolute neutrophil count 
< 1000/μL and/or platelets < 100,000/μL). 
Measurable disease will be defined as > 5% marrow blasts. 
Relapse following complete remission is defined as reappearance of leukemic blasts in the 
peripheral blood or the finding of more than 5% blasts in the bone marrow, not attributable to 
another cause, or extramedullary relapse. 
Progressive disease will be defined as increase of at least 50% in the percentage of blasts in the 
bone marrow aspirate with an increase of at least 10 percent of blasts in the bone marrow 
aspirate. 
11.1.2.3. Tumor Markers 
Tumor markers may be utilized to assess response as appropriate for the diagnosis. This should 
include at least quantitative RT-PCR for WT1 transcript in blood or bone marrow for patients 

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with AML, and serum CA-125 for patients with ovarian carcinoma. Measurable disease may be 
determined by markers alone as defined by individual laboratory normal ranges. Response will 
be quantified by the fractional decrease on treatment. Tumor markers drawn from peripheral 
blood samples should be assessed at least every 4 weeks after the first dose but may be 
performed more frequently. 
11.1.3. 
Other 
Biomarker analyses will be conducted as indicated on the Schedules of Assessments 
(Tables 3 - 6). An evaluable patient for biomarkers is defined as a patient who has had the 
Day 29 biomarker assessments completed. Directions for the handling and processing of 
biomarker samples are provided in a separate manual. 
Peripheral blood samples will be obtained for evaluation of WT1 peptide-specific CTL induction 
activity by tetramer assay, and WT1 serum antibody titer by enzyme-linked immunosorbent 
assay [ELISA], and for isolation of peripheral blood mononuclear cells (PBMCs) for exploratory 
biomarker analyses. In addition, blood samples will be obtained and blood serum archived for 
possible analyses of other biomarkers, such as protein and microRNA, that may be important to 
the understanding of WT2725 but which have not yet been selected. Providing informed consent 
for use of this sample for pharmacogenetic purposes is optional, and participation in the study is 
not dependent upon providing this sample. 
Delayed-type hypersensitivity to the WT2725 peptide will be measured approximately 48 hours 
(but no sooner than 24 hours) after id inoculation with WT2725 in diluted WT2725 Injection for 
DTH skin tests. The site of diluted WT2725 Injection inoculation will be compared to the site of 
inoculation with the negative control (DSWI). The criteria for evaluation include injection site 
erythema diameter and quality (eg, redness, induration, ulceration, etc). Directions for 
conducting and evaluating DTH skin tests are provided in Appendix VII. 
Immunohistochemistry in tumor tissue will be evaluated by expression of CD8+, Foxp3, HLA 
class I, and WT1 protein using tumor tissue samples obtained before the first dose and after the 
last dose of study drug. In place of tumor tissue samples from biopsy during screening, archived 
tumor tissue samples may be provided. Biopsy for tumor tissue samples after the last dose of 
study drug is not mandatory and refusal to consent to post-treatment biopsy samples will not 
exclude a patient from enrollment in the study. IHC need not be performed for patients with 
AML provided RT-PCR for WT1 transcript is assessed. 
11.2. 
Standardization of Data Capture 
Study Schematic and Schedule of Assessments: For Part 1 of the study a schematic of the 
study design is presented in Figure 1 and a summary of assessments to be conducted at each visit 
are presented in Table 3, Schedule of Assessments for Screening and Vaccine Induction Phase 
and Table 4, Schedule of Assessments for Consolidation Phase, Maintenance Phase, and End of 
Study. For Part 2 of the study a schematic of the study design is presented in Figure 2 and a 
summary of assessments to be conducted at each visit are presented in Table 5, Schedule of 
Assessments for Screening and Vaccine Induction Phase and Table 6, Schedule of Assessments 
for Consolidation Phase, Maintenance Phase, and End of Study. 

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Vital Signs: Vital signs following 5 minutes of seated rest, will consist of supine systolic and 
diastolic blood pressures, respiration rate, pulse, and oral body temperature. If possible, the same 
arm should be used during each assessment of blood pressure and pulse throughout the study. 
Vital signs will be obtained prior to collection of clinical laboratory samples and performance of 
an ECG. 
Weight and height: Weight and height should be measured in street clothing with no shoes. 
Centrally-read ECG: All ECGs will be obtained in the supine position, after the patient has 
been resting supine for at least 10 minutes. ECGs will be 12-lead with a 10-second rhythm strip. 
ECGs should be obtained prior to drawing blood samples. All attempts should be made to use the 
same ECG recorder for all visits within individual patients. ECGs will be centrally read at a core 
lab according to established quality assurance procedures for inter/intra reader variability. Refer 
to Appendix I for additional information. 
Clinical Laboratory: For detailed instructions regarding laboratory procedures, sampling, and 
shipping guidelines refer to the site and local laboratory guidelines. Samples will be processed at 
a local laboratory. All laboratories will be College of American Pathologists (CAP) and Clinical 
Laboratory Improvement Amendments (CLIA) (or equivalent) certified. See Appendix IV for a 
list of required clinical laboratory tests. 
Adverse Events: As a discussion guide, patients should be queried in a non-leading manner, 
without specific prompting (eg, “Has there been any change in your health status since your last 
visit?”). See Appendix VIII for definitions used in reporting AEs. 
Concomitant Medications and Medical History: Patient self-report will be acceptable for 
listing all concomitant medication use, medical history, and evaluation for inclusion/exclusion 
except where specific protocol procedures are mandated to ensure appropriate enrollment (eg, 
certain baseline lab values). 
11.3. 
Electronic Data Capture (EDC) 
The study sites will use a validated EDC system to enter patient data onto case report forms 
(CRFs). The Data Labs system will be used for this clinical study. Password protected access to 
the EDC system will be via a secure website. Data queries and data corrections will be handled 
through the same system. All transactions within the EDC system are fully documented within 
an electronic audit trail. Each set of completed CRFs must be reviewed and electronically signed 
and dated by the Investigator. 
11.4. 
Study Visits and Assessments 
11.4.1. 
Screening – Part 1 and Part 2 
Patients will be evaluated during screening to determine their eligibility to enroll in the study. All 
assessments must be completed within the specified timeframes prior to the planned first dose of 
study drug. 
11.4.1.1. Screening (within 21 days before the first dose) 
The following study-related procedures will be performed 

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• Obtain signed informed consent and privacy authorization from the patient or his or 
her legal representatives before conducting any other visit procedures. 
• Record all concomitant medications, including prior antineoplastic therapy, over-the 
counter (OTC) and health and dietary supplements taken within the previous 30 days. 
• Begin recording AEs 
• Review inclusion and exclusion criteria. 
• Obtain medical history including demographic information. 
• Obtain blood samples for hepatitis B and C, HIV-1 and HIV-2, HLA typing (if 
necessary), and for females of child bearing potential, for pregnancy test. Note: HLA 
typing previously obtained is acceptable, otherwise HLA typing should be the first 
procedure performed. 
• Perform tumor assessments for baseline. Note: Results obtained within the 28 days 
prior to the planned first dose of study drug are acceptable (There are no variations 
allowed beyond this window.). If multiple assessments are available, the most recent 
ones should be used as the baseline result. Data from at least one additional historical 
assessment prior to the baseline assessment should also be provided, if available. 
11.4.1.2. Screening (within 7 days before the first dose) 
The following study-related procedures will be performed 
• Perform physical examination. 
• Obtain vital signs (systolic and diastolic blood pressures, pulse, respiration rate, and 
oral body temperature) including height and weight. 
• Obtain ECOG Performance Status. 
• Perform 12-lead ECG. 
• Obtain urine sample for urinalysis. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood sample for CTL induction activity. 
• Obtain tumor samples for IHC. In place of tumor tissue samples obtained from a 
biopsy during screening, archived tumor tissue samples may be provided. Availability 
of tumor tissue samples should be determined during screening through provision of 
the accession number or other identification number. If necessary, the tumor tissue 
sample biopsy should be performed after study eligibility has been confirmed and 
before the first administration of study drug. The tumor tissues samples will only be 
provided to the sponsor for patients who receive study drug. Instructions for tissue 
sample processing and shipment are provided in a separate manual. In place of 
archival tumor tissue samples, patients with AML should have available a bone 
marrow aspirate and/or bone marrow biopsy with PCR for WT1 transcript performed 
before the first dose of study drug. Patients with AML should also have peripheral 

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blood assessed for blasts and WT1 transcript. IHC need not be performed for patients 
with AML provided RT-PCR for WT1 transcript is assessed. 
• Record AEs and concomitant therapies as necessary. 
• Review inclusion and exclusion criteria. 
11.4.2. 
Study Treatment Periods 
11.4.2.1. Vaccine Induction Phase – Part 1 
During the vaccine induction phase, patients will receive study drug once every 7 days for 
4 weeks, ie, a total of 5 doses. Additional dosing windows are provided for patients who may 
need to recover from intercurrent medical conditions (see Section 8.3.2.1). 
11.4.2.1.1. Day -2 
Approximately 48 hours (but no sooner than 24 hours) before planned first dose: 
• Perform injections for DTH skin test. 
• Record AEs and concomitant therapies as necessary. 
11.4.2.1.2. First Dose (Day 1 [-1]) 
Day 1 
• Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Perform 12-lead ECG. Note: May be omitted if performed within the previous 
3 days. If multiple assessments are available, the most recent ones should be used. 
• Obtain blood samples for hematology and serum chemistry and for females of 
child-bearing potential, for pregnancy test. Note: May be omitted if performed 
within the previous 3 days. If multiple assessments are available, the most recent 
ones should be used. 
• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, PBMC 
isolation, and retrospective biomarker analyses. 
• Obtain urine sample for urinalysis. Note: May be omitted if performed within the 
previous 3 days. If multiple assessments are available, the most recent ones should 
be used. 
• Review continuation criteria. 
• Perform evaluation of DTH skin test. 

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• Perform injections for new DTH skin test immediately before administration of 
study drug. 
Administer study drug. 
6 hours (± 30 minutes) postdose 
• Obtain blood sample for retrospective biomarker analyses. 
11.4.2.1.3. Day 3 
Approximately 48 hours (±6 hours) after the dose on Day 1: 
• Obtain blood sample for retrospective biomarker analyses. 
• Record AEs and concomitant therapies as necessary. 
Perform evaluation of DTH skin test, approximately 48 hours (but no sooner than 24 hours) after 
the DTH skin test injections on Day 1. 
11.4.2.1.4. Second Dose (Day 8 [-1]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity and retrospective biomarker 
analyses. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.1.5. Third Dose (Day 15 [-1]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, and 
retrospective biomarker analyses. 
• Obtain urine sample for urinalysis. 

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• Review continuation criteria. 
Administer study drug. 
11.4.2.1.6. Fourth Dose (Day 22 [-1]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.1.7. Day 27 
Approximately 48 hours (but no sooner than 24 hours) before planned fifth dose: 
• Perform injections for DTH skin test. 
• Record AEs and concomitant therapies as necessary. 
11.4.2.1.8. Fifth Dose (Day 29 [-1]) 
Day 29 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Perform 12-lead ECG. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, 
PBMC isolation, retrospective biomarker analyses, and tumor markers drawn 
from peripheral blood. 
• Obtain urine sample for urinalysis. 
• Review continuation criteria. 
• Perform evaluation of DTH skin test. 

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• Perform injections for new DTH skin test immediately before administration of 
study drug. 
Administer study drug. 
11.4.2.1.9. Day 31 
Approximately 48 hours (but no sooner than 24 hours) after the DTH skin test injections on 
Day 29: 
• Record AEs and concomitant therapies as necessary. 
• Perform evaluation of DTH skin test. 
11.4.2.2. Consolidation Phase – Part 1 
During the consolidation phase, patients will receive study drug once every 14 days for 6 weeks, 
ie, a total of 4 doses. Additional dosing windows are provided for patients who may need to 
recover from intercurrent medical conditions (see Section 8.3.2.1). 
11.4.2.2.1. Sixth Dose (Day 43 [± 2]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood sample for CTL induction activity. 
• Obtain urine sample for urinalysis. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.2.2. Seventh Dose (Day 57 [± 2]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Perform 12-lead ECG. 
• Obtain blood samples for hematology and serum chemistry. 

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• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, and 
retrospective biomarker analyses. 
• Obtain urine sample for urinalysis. 
• Perform tumor assessments for response. For patients with AML, bone marrow 
aspirations/biopsies are scheduled when clinically indicated, however peripheral 
blood should be assessed for blasts and WT1 transcript at least every 4 weeks after 
the first dose. Other tumor markers drawn from peripheral blood samples should be 
assessed every 4 weeks after the first dose. This assessment may be performed 
± 7 days of the scheduled assessment but must be completed before the next planned 
administration of study drug. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.2.3. Eighth Dose (Day 71 [± 2]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood sample for CTL induction activity. 
• Obtain urine sample for urinalysis. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.2.4. Day 83 
Approximately 48 hours (but no sooner than 24 hours) before planned ninth dose: 
• Perform injections for DTH skin test. 
• Record AEs and concomitant therapies as necessary. 
11.4.2.2.5. Ninth Dose (Day 85 [± 2]) 
Day 85 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 

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• Obtain vital signs and weight. 
• Perform 12-lead ECG. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, 
retrospective biomarker analyses, and tumor markers drawn from peripheral blood. 
• Obtain urine sample for urinalysis. 
• Perform evaluation of DTH skin test. Review continuation criteria. 
• Perform injections for new DTH skin test immediately before administration of 
study drug. 
Administer study drug. 
11.4.2.2.6. Day 87 
Approximately 48 hours (but no sooner than 24 hours) after the DTH skin test injections on 
Day 85: 
• Record AEs and concomitant therapies as necessary. 
• Perform evaluation of DTH skin test. 
11.4.2.3. Maintenance Phase – Part 1 
During the maintenance phase, patients will receive study drug once every 28 days until 
discontinuation. Additional dosing windows are provided for patients who may need to recover 
from intercurrent medical conditions (see Section 8.3.2.1). 
During each dosing day the following assessments will be performed as noted. 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Perform 12-lead ECG. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity. 
• Obtain blood samples for WT1 serum antibody titer and retrospective biomarker 
analyses every second dosing day after the ninth dose. 
• Obtain urine sample for urinalysis. 
• Review continuation criteria. 

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• Perform tumor assessments for response every 8 weeks after the first dose or more 
frequently as clinically indicated. For patients with AML, bone marrow 
aspirations/biopsies are scheduled when clinically indicated, however peripheral 
blood should be assessed for blasts and WT1 transcript at least every 4 weeks after 
the first dose. Other tumor markers drawn from peripheral blood samples should be 
assessed every 4 weeks after the first dose. This assessment may be performed 
± 7 days of the scheduled assessment but must be completed before the next planned 
administration of study drug. 
Administer study drug. 
11.4.2.4. Vaccine Induction Phase – Part 2 
During the vaccine induction phase, patients will receive study drug once every 7 days for 
8 weeks, ie, a total of 9 doses. Additional dosing windows are provided in Section 8.3.2.2.  
11.4.2.4.1. Day -2 
Approximately 48 hours (but no sooner than 24 hours) before planned first dose: 
• Perform injections for DTH skin test. 
• Record AEs and concomitant therapies as necessary. 
11.4.2.4.2.  First Dose (Day 1 [-1]) 
Day 1 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Perform 12-lead ECG. Note: May be omitted if performed within the previous 3 days. 
If multiple assessments are available, the most recent ones should be used. 
• Obtain blood samples for hematology and serum chemistry and for females of 
child-bearing potential, for pregnancy test. Note: May be omitted if performed within 
the previous 3 days. If multiple assessments are available, the most recent ones 
should be used. 
• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, PBMC 
isolation, and retrospective biomarker analyses. 
• Obtain urine sample for urinalysis. Note: May be omitted if performed within the 
previous 3 days. If multiple assessments are available, the most recent ones should be 
used. 
• Review continuation criteria. 

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• Perform evaluation of DTH skin test. 
• Perform injections for new DTH skin test immediately before administration of study 
drug. 
Administer study drug. 
6 hours (± 30 minutes) postdose 
• Obtain blood sample for retrospective biomarker analyses. 
11.4.2.4.3. Day 3 
Approximately 48 hours (±6 hours) after the dose on Day 1: 
• Obtain blood sample for retrospective biomarker analyses. 
• Record AEs and concomitant therapies as necessary. 
Perform evaluation of DTH skin test, approximately 48 hours (but no sooner than 24 hours) after 
the DTH skin test injections on Day 1. 
11.4.2.4.4. Second Dose (Day 8 [-1]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity and retrospective biomarker 
analyses. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.4.5. Third Dose (Day 15 [-1]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, and 
retrospective biomarker analyses. 

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• Obtain urine sample for urinalysis. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.4.6. Fourth Dose (Day 22 [-1]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.4.7. Day 27 
Approximately 48 hours (but no sooner than 24 hours) before planned fifth dose: 
• Perform injections for DTH skin test. 
• Record AEs and concomitant therapies as necessary. 
11.4.2.4.8. Fifth Dose (Day 29 [-1]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Perform 12-lead ECG. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, PBMC 
isolation, retrospective biomarker analyses, and tumor markers drawn from peripheral 
blood. 
• Obtain urine sample for urinalysis. 
• Review continuation criteria. 
• Perform evaluation of DTH skin test. 

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• Perform injections for new DTH skin test immediately before administration of study 
drug. 
Administer study drug. 
11.4.2.4.9. Day 31  
Approximately 48 hours (but no sooner than 24 hours) after the DTH skin test injections on 
Day 29: 
• Record AEs and concomitant therapies as necessary. 
• Perform evaluation of DTH skin test. 
11.4.2.4.10. 
Sixth Dose (Day 36 [-1]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.4.11. 
Seventh Dose (Day 43 [-1])  
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood sample for CTL induction activity. 
• Obtain urine sample for urinalysis. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.4.12. 
 Eighth Dose (Day 50 [-1]) 
Record AEs and concomitant therapies as necessary. 

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Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.4.13. 
Ninth Dose (Day 57 [-1)] 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Perform 12-lead ECG. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, and 
retrospective biomarker analyses. 
• Obtain urine sample for urinalysis. 
• Perform tumor assessments for response. For patients with AML, bone marrow 
aspirations/biopsies are scheduled when clinically indicated, however peripheral 
blood should be assessed for blasts and WT1 transcript at least every 4 weeks after 
the first dose but may be performed more frequently. Other tumor markers drawn 
from peripheral blood samples should be assessed every 4 weeks after the first dose 
or more frequently a clinically indicated. This assessment may be performed ± 7 days 
of the scheduled assessment but must be completed before the next planned 
administration of study drug. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.5. Consolidation Phase Part 2 
During the consolidation phase, patients will receive study drug once every 14 days for 10 weeks 
(maximum 6 doses). Additional dosing windows are provided in Section 8.3.2.2.  
11.4.2.5.1. Tenth Dose (Day 71 [± 2]) 
Record AEs and concomitant therapies as necessary. 

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Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood sample for CTL induction activity. 
• Obtain urine sample for urinalysis. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.5.2. Day 83  
Approximately 48 hours (but no sooner than 24 hours) before planned ninth dose: 
• Perform injections for DTH skin test. 
• Record AEs and concomitant therapies as necessary. 
11.4.2.5.3. Eleventh Dose (Day 85 [± 2]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Perform 12-lead ECG. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, 
retrospective biomarker analyses, and tumor markers drawn from peripheral blood. 
• Obtain urine sample for urinalysis. 
• Perform evaluation of DTH skin test. 
• Review continuation criteria. 
• Perform injections for new DTH skin test immediately before administration of study 
drug. 
Administer study drug. 
11.4.2.5.4. Day 87 
Approximately 48 hours (but no sooner than 24 hours) after the DTH skin test injections on 
Day 85: 

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• Record AEs and concomitant therapies as necessary. 
• Perform evaluation of DTH skin test. 
11.4.2.5.5. Twelfth Dose (Day 99 [± 2])  
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity. 
• Obtain urine sample for urinalysis. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.5.6. Thirteenth Dose (Day 113 [± 2])  
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Perform 12-lead ECG. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, and 
retrospective biomarker analyses. 
• Obtain urine sample for urinalysis. 
• Perform tumor assessments for response. For patients with AML, bone marrow 
aspirations/biopsies are scheduled when clinically indicated, however peripheral 
blood should be assessed for blasts and WT1 transcript at least every 4 weeks after 
the first dose but may be performed more frequently. Other tumor markers drawn 
from peripheral blood samples should be assessed every 4 weeks after the first dose 
or more frequently a clinically indicated. This assessment may be performed ± 7 days 
of the scheduled assessment but must be completed before the next planned 
administration of study drug. 
• Review continuation criteria. 

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Administer study drug. 
11.4.2.5.7. Fourteenth Dose (Day 127 [± 2]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity. 
• Obtain urine sample for urinalysis.  
• Review continuation criteria. 
Administer study drug. 
11.4.2.5.8. Fifteenth Dose (Day 141 [± 2]) 
Record AEs and concomitant therapies as necessary. 
Predose 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Perform 12-lead ECG. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, 
retrospective biomarker analyses, and tumor markers drawn from peripheral blood. 
• Obtain urine sample for urinalysis. 
• Review continuation criteria. 
Administer study drug. 
11.4.2.6. Maintenance Phase – Part 2 
During the maintenance phase, patients will receive study drug once every 28 days until 
discontinuation. Additional dosing windows are provided for patients who may need to recover 
from intercurrent medical conditions (see Section 8.3.2.2). 
During each dosing day the following assessments will be performed as noted. 
Record AEs and concomitant therapies as necessary. 
Predose 

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• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 
• Perform 12-lead ECG. 
• Obtain blood samples for hematology and serum chemistry. 
• Obtain blood samples for CTL induction activity. 
• Obtain blood samples for WT1 serum antibody titer and retrospective biomarker 
analyses every second dosing day after the start of the maintenance phase (monthly 
dosing schedule). 
• Obtain urine sample for urinalysis. 
• Review continuation criteria. 
• Perform tumor assessments for response every 8 weeks after the first dose or more 
frequently as clinically indicated. For patients with AML, bone marrow 
aspirations/biopsies are scheduled when clinically indicated or per the post-transplant 
schedule/institutional policy if no other indicator of measurable disease, however 
peripheral blood should be assessed for blasts and WT1 transcript at least every 
4 weeks after the first dose but may be performed more frequently. Other tumor 
markers drawn from peripheral blood samples should be assessed every 4 weeks after 
the first dose or more frequently as clinically indicated. This assessment may be 
performed ± 7 days of the scheduled assessment but must be completed before the 
next planned administration of study drug. 
Administer study drug. 
11.4.2.7. 48 hours Before End of Study Evaluations – Part 1 and Part 2 
Approximately 48 hours (but no sooner than 24 hours) before planned End of Study visit: 
• Perform injections for DTH skin test. 
• Record AEs and concomitant therapies as necessary. 
11.4.2.8. End of Study (EOS) (within 28 days after last dose of study drug and prior to 
start of alternate antineoplastic therapy) – Part 1 and Part 2 
All patients should complete an End of Study visit within 28 days after the last dose of study 
drug and prior to the start of alternate antineoplastic therapy. During the End of Study visit, the 
following assessments will be performed. 
• Record AEs and concomitant therapies as necessary. 
• Obtain ECOG Performance Status. 
• Perform physical examination. 
• Obtain vital signs and weight. 

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• Perform 12-lead ECG. 
• Obtain blood samples for hematology and serum chemistry, and for females of 
child-bearing potential, for pregnancy test. 
• Obtain blood samples for CTL induction activity, WT1 serum antibody titer, PBMC 
isolation, and retrospective biomarker analyses. 
• Obtain urine sample for urinalysis. 
• Perform evaluation of DTH skin test. 
• Obtain tumor samples for IHC from all patients who provided consent. 
• Perform tumor assessments for response. For patients with AML, bone marrow 
aspirations/biopsies are scheduled when clinically indicated, however peripheral 
blood should be assessed for blasts and WT1 transcript at least at every scheduled 
tumor assessment. 
• Assess disease progression and survival. 
11.4.3. 
Follow-up – Part 1 and Part 2 
Following the End of Study visit, patients will be contacted every 3 months until the study is 
closed to evaluate disease progression, if not already reached, and survival. 

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12. 
DISCONTINUATION AND REPLACEMENT OF PATIENTS/ 
CLINICAL ASSESSMENTS AFTER STUDY MEDICATION 
DISCONTINUATION 
12.1. 
Clinical Assessments After Study Medication Discontinuation 
Every effort should be made for all treated patients discontinuing study drug, regardless of cause, 
and, prior to the start of alternate treatment to undergo final evaluation procedures, in accordance 
with the End of Study visit as described in Section 11.4.2.8. 
12.2. 
Study Participation Termination Criteria 
The possible reasons for study drug discontinuation and study participation termination are to be 
assessed for each enrolled patient and are as follows: 
• Adverse event. 
• Progressive disease. 
• Death: 
o Death due to progressive disease. 
o Death due to other causes 
• Study stopped by sponsor. 
• Lost to follow-up. 
• Protocol violation (specify). 
• Pregnancy. 
• Withdrawal by patient (specify). 
• Other (specify). 
12.3. 
Replacement of Patients 
Patients who discontinue from the study for a reason other than DLT prior to completing the 
DLT Evaluation Period will be replaced. 

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13. 
ADVERSE EXPERIENCE REPORTING 
13.1. 
Adverse Events 
An AE is any new, untoward medical occurrence or worsening of a pre-existing medical 
condition that occurs during study participation, whether or not the event is considered drug 
related. 
AEs will be collected from the time the informed consent is signed through the end of the study. 
Serious adverse events will be collected and reported on the SAE form, from the time of 
informed consent through 30 days post last dose and will be followed until resolution or lost to 
follow-up. SAEs occurring from the time of informed consent through the end of study must be 
recorded on the CRF and the data recorded should match that on the SAE form. 
Non-leading questions will be used to ask patients about the possible occurrence of AEs. The 
Investigator should attempt to establish a diagnosis of the event based on signs, symptoms, 
and/or other clinical information. In such cases, the diagnosis should be documented as the AE 
and not the individual signs/symptoms. 
Following questioning and evaluation, all AEs, whether believed by the Investigator to be related 
or unrelated to the study drug, must be documented in the patient’s study records/source 
documents, in accordance with the Investigator’s normal clinical practice, and on the AE page of 
the CRF. Each AE is to be evaluated for duration, severity according to the CTCAE V.4.0, 
seriousness, and causal relationship to the study medication. Appendix VIII (Definitions for 
Reporting Adverse Events) provides definitions for relationship to study medication, and action 
taken, and reference to CTCAE for grade. An AE is deemed associated with the use of the study 
drug “if there is a reasonable possibility that the experience may have been caused by the drug” 
(21 CFR 312.32 [a]). 
The Medical Monitor is the initial contact person for protocol related questions or discussion of 
AEs. The contact information for the Medical Monitor as well as other emergency contact 
information can be found on page 3 of this protocol. 
13.2. 
Objective Findings 
New and worsening signs and symptoms of underlying or emerging disease must be recorded as 
AEs. Clinically significant abnormal objective findings (eg, clinical laboratory value, ECG 
value, and physical examination observation) will also be recorded on the Adverse Event page of 
the CRF from signing of the informed consent onwards. When a clear diagnosis is available that 
explains the objective findings, this diagnosis will be recorded as the AE, and not the abnormal 
objective finding (eg, viral hepatitis will be recorded as the AE, not transaminase elevation). If a 
definite diagnosis is not available, then the sign (eg, clinically significant elevation of 
transaminase levels) or symptom (eg, abdominal pain) will be recorded as the AE. 
Progression of the malignancy being treated in the study is not reported as an AE. 
Hospitalization due solely to progression of the malignancy being treated in the study is not an 
SAE. Clinical symptoms of progression may be reported as AEs if the symptoms cannot be 

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determined as exclusively due to the malignancy being treated in the study, or if they do not fit 
the expected pattern of progression for the malignancy being treated in the study. 
Clinical laboratory test results will be reviewed by an Investigator as they become available. 
Possibly drug-related or clinically relevant abnormal values of uncertain causality must be 
repeated. Any abnormal values that persist should be followed at the discretion of the 
Investigator. Laboratory reports, as appropriate when an electronic medical records system is not 
used, will be initialed and dated on all pages by a Form FDA 1572-listed Investigator. 
All on-site ECG tracings and ECG over-read reports will be reviewed by an Investigator as they 
become available. The Investigator must determine the clinical significance of all abnormal ECG 
interpretations on the machine read tracing. Possibly drug-related or clinically relevant abnormal 
ECGs of uncertain causality must be repeated. Any abnormal ECGs that persist should be 
followed at the discretion of the Investigator. ECG tracings, as necessary when an electronic 
system is not used, will be initialed and dated on all pages by a Form FDA 1572-listed 
Investigator. 
13.3. 
Immediately Reportable Events 
There are 2 categories of medical events that could occur during participation in a clinical study 
that must be immediately reported to Sunovion Pharmaceuticals Inc. or its agent: 
• SAE, including death. 
• The incidence of pregnancy. 
PPD Pharmacovigilance (PVG) must be contacted immediately upon first knowledge of the 
incident. 
PPD PVG: 
Phone hotline: (919) 456-6001 
Fax: (919) 654-0211 
Email: Sunovionsafety@druginfo.com 
Emergency contact information can also be found on page 3 of this protocol. 
An AE or suspected adverse reaction is considered “serious” if, in the view of either the 
Investigator or Sponsor, it results in any of the following outcomes: death, a life-threatening AE, 
in-patient hospitalization or prolongation of existing hospitalization (Hospitalization due solely 
to progression of the malignancy being treated in the study is not an SAE.), a persistent or 
significant incapacity or substantial disruption of the ability to conduct normal life functions, or a 
congenital anomaly/birth defect. Important medical events that may not result in death, be life-
threatening, or require hospitalization may be considered serious when, based upon appropriate 
medical judgment, they may jeopardize the patient and may require medical or surgical 
intervention to prevent one of the outcomes listed in this definition. Examples of such medical 
events include allergic bronchospasm requiring intensive treatment in an emergency room or at 
home, blood dyscrasias or convulsions that do not result in in-patient hospitalization, or the 
development of drug dependency or drug abuse. 

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Life-threatening means that the patient was, in the view of the Investigator, at immediate risk of 
death from the event as it occurred. This definition does not include an event that, had it occurred 
in a more severe form might have caused death. 
If an Investigator or study site staff becomes aware of a SAE that occurs in a study patient from 
the time that informed consent is signed through 30 days following the last dose of study 
medication, this must be reported immediately to PPD PVG. 
In addition to the initial telephone notification, an initial SAE form as applicable must be 
completed and sent via fax to PPD PVG within 1 business day of an Investigator or study site 
staff becoming aware of the event. Sunovion Pharmaceuticals Inc. provides the SAE form used 
to report SAEs as a part of the document package necessary to conduct this clinical study. 
Sunovion Pharmaceuticals Inc. will promptly notify all research sites of an AE that is determined 
to be reportable to the Regulatory Authorities. These AEs must be promptly reported to the 
Institutional Review Board (IRB) by the Principal Investigator. 
If a patient becomes pregnant during the course of the study, she will be instructed to 
immediately stop taking study medication. Further, the patient will be instructed to return within 
48 hours of the first notification of pregnancy to the research site and undergo a serum pregnancy 
test, as confirmation of pregnancy. If positive, the patient will no longer receive any additional 
study medication and will continue to be followed. All pregnancies, whether or not the patient 
received any study medication, will be followed until resolution (ie, termination [voluntary or 
spontaneous] or birth). 
To report a pregnancy, the Pregnancy Event Form must be completed and sent via facsimile to 
PPD PVG within 1 business day of first knowledge by the research personnel of the pregnancy. 
Sunovion Pharmaceuticals Inc. provides the Pregnancy Event Form as a part of the document 
package necessary to conduct this clinical study. Pregnancies occurring from the time the 
informed consent is signed through 30 days following the last dose of study medication will be 
followed quarterly until birth or termination of the pregnancy. 
If a pregnancy is reported for a male study patient’s partner, the Sponsor’s representative will 
provide instructions on how to collect pregnancy information in accordance with local 
requirements. Proper consent to collect the partner’s information will be obtained prior to the 
collection of any information. 

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14. 
STATISTICS 
14.1. 
Sample Size Determination 
The sample size for Part 1 and Part 2 is based on clinical and practical considerations for a 
Phase 1 dose escalation study using the rolling-six design and is outside of statistical 
considerations. 
14.2. 
Randomization and Blinding 
No randomization or blinding will be employed, open-label study drug will be used and there is 
no comparator. 
14.3. 
Unblinding Procedures 
Not applicable for this study. All study drug will be open label. 
14.4. 
Analysis Populations 
There are 3 planned analysis populations for this study. 
The Safety population will include all enrolled patients who receive at least 1 dose of study drug 
and will be used for the analysis of all safety and efficacy data except as noted below. 
If needed, a DLT population will include all patients in the Safety population who are evaluable 
for DLT evaluation and will be used for analysis of DLT. 
If needed, an Efficacy population will include all patients in the Safety population who are 
evaluable for response and will be used for analysis of response assessment. 
14.5. 
Data Analysis 
Data will generally be summarized by enrolled or starting dose, unless otherwise indicated. 
Efficacy data may also be summarized by tumor type and/or outcome measure. Descriptive 
statistics including number of patients, mean, standard deviation, minimum, 25th percentile, 
median, 75th percentile, and maximum will be provided. 
No imputation will be performed for missing data in this Phase 1 study. 
14.5.1. 
Patient Disposition 
The number and percentage of patients will be presented by dose cohort, together, with the 
number and percentage of patients who withdrew from the study for each study discontinuation 
reason. Reason for discontinuation for all patients will be listed by dose cohort. 
14.5.2. 
Drug Exposure and Compliance 
Study drug exposure including cumulative dose and duration of exposure will be summarized by 
dose cohort. Duration of exposure (ie, weeks on study drug) will be calculated as the number of 
weeks from first to last dose date. The number of patients with a dose reduction, dose escalation, 
and no dose modification in each enrolled dose cohort will also be summarized. All patients will 

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be administered study drug in clinic, and all recorded exposure or compliance information will 
listed by patient. 
14.5.3. 
Important Protocol Deviations 
Important Protocol Deviations (IPDs) will be identified and documented based on a review of 
potentially IPDs. The potential IPDs will be identified through programmatic checks of study 
data, as well as through review of selected data listings. The potential IPDs to be reviewed 
include, but are not limited to, patients who: 
• Did not meet inclusion/exclusion or continuation criteria 
• Received any disallowed concomitant medication or therapy 
The number of patients with each type of IPD will be tabulated by category and by deviation 
type for all patients in the Safety population by dose cohort. Individual IPDs will be presented in 
a data listing. 
14.5.4. 
Demographic and Baseline Characteristics 
The number of patients of each sex, in each racial group, in each ethnicity group, and in each 
ECOG performance status score category will be summarized. Age and body mass index (BMI) 
will be summarized using descriptive statistics (number of patients, mean, standard deviation, 
median, minimum, and maximum). 
The medical history of patients will be coded by System Organ Class (SOC) and Preferred Term 
(PT) using the Medical Dictionary for Regulatory Activities (MedDRA), version 14.0 or higher. 
The number of patients with abnormal findings in each SOC and each PT will be summarized. 
Cancer history also will be summarized. 
14.5.5. 
Efficacy Analysis 
Tumor response category evaluated according to the irRC, modified IWG, and/or tumor markers 
will be summarized by dose cohort, and will be listed for each patient. Response assessments for 
the irRC, modified IWG, and/or tumor markers will be summarized separately. The number of 
patients in each overall response category will be provided. Descriptive statistics will similarly 
be provided for the percent change from baseline in tumor burden where applicable. 
The distribution of biomarker values will be summarized at each time point and at the last 
assessment by dose cohort using descriptive statistics, and data will be listed for each patient. 
The relationship between the level of expression of WT1 protein and the degree of immune 
response as measured by CTL induction will be assessed graphically and the Spearman rank 
correlation coefficient will be estimated. 
14.5.6. 
Adjustment for Multiplicity 
No adjustment for multiple comparisons will be used as no statistical testing is planned and all 
analyses are considered exploratory in nature. 

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14.5.7. 
Safety Analysis 
Safety measures will be summarized using descriptive statistics generally by enrolled dose 
cohort and listed for each patient. 
14.5.7.1. DLT and Adverse Events 
All AEs will be coded using MedDRA, version 14.0 or higher. 
The occurrence of DLT events during the DLT evaluation period will be summarized for each 
dose cohort. 
Treatment-emergent adverse events (TEAEs) will be defined as: 
• AEs that occurred on or after the first dose of study drug and on or before the 30th day 
after the last dose of study drug, 
• AEs with a missing start date and a stop date on or after the first dose of study drug, 
or 
• AEs with both a missing start and stop date. 
TEAEs will be summarized by dose cohort and overall and by MedDRA SOC and PT. 
The following AEs will be summarized and presented by enrolled dose cohort and by MedDRA 
SOC and PT for the Safety population: 
• All TEAEs (including number of events and patient incidence) 
• TEAEs by relationship to treatment (unrelated, or related) 
• TEAEs by CTCAE Grade 
Similar summaries will be presented by the last dose received before the start of the event. 
The following conventions will be followed in summarizing AEs: 
• For patient incidence summaries, each patient will be counted only once within each 
SOC and within each PT. 
• If a patient reports more than one AE within a PT and/or a SOC, the AE with the 
highest known grade within each SOC and within each PT will be included in the 
summaries by grade. 
• For summaries by relationship to study medication, AEs whose relationship to 
treatment is assessed as “not related” will be grouped as “unrelated.” AEs assessed as 
“possible,” “probable,” or “definite,” unknown or missing will be grouped as 
“related.” If a patient reports more than one AE within the same treatment regimen, 
SOC and PT, and any are related, it will be summarized as related. 
A listing of all TEAEs, SAEs, and AEs leading to discontinuation or death, will be presented. A 
separate listing for non-treatment-emergent AEs will also be provided. 
14.5.7.2. Clinical Laboratory Assessments 
For laboratory parameters with continuous outcomes, descriptive statistics will be presented for 
each dose cohort and within each tumor group at each time point. Changes from baseline will be 

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summarized in the same manner. For laboratory parameters with categorical outcomes, the 
number of patients with each outcome will be presented. The data listings for laboratory 
parameters will flag values outside of the reference range. 
14.5.7.3. Electrocardiograms 
A standard 12-lead ECG will be used to collect and record electrocardiographic data according to 
the schedule of assessments. Parameters to be collected include ventricular heart rate, QT 
interval, PR interval, QRS duration, and RR interval (ms). The corrected QT interval will be 
derived according to the Fridericia formula (QTc–F = QT/[R-R/1000]1/3) as well as Bazett’s 
formula (QTc–B = QT/[R-R/1000]1/2). 
ECG parameters and changes in these parameters from predose Day 1, as determined by the 
central over-read, will be summarized using descriptive statistics at each time point by dose 
cohort. 
The number of patients with QTc-F values in the following categories will be summarized: 
• QTc-F ≥ 500 ms at any postdose time point and not present at baseline 
• QTc-F ≥ 450 ms at any postdose time point and not present at baseline 
• Change from baseline in QTc-F ≥ 60 ms for at least 1 postdose measurement 
• Change from baseline in QTc-F ≥ 30 ms for at least 1 postdose measurement, but 
< 60 ms for all postdose measurements 
Any unscheduled ECG that occurs after first dose will be included for these post-treatment 
summaries. 
The above categorical analyses will also be performed for QTc-B. 
A listing of patients with over-read ECG abnormalities including overall, rhythm, conduction, 
morphology, myocardial infarction, and the presence of ST, T, and U wave abnormalities will be 
presented. 
14.5.7.4. Vital Signs 
Body temperature, respiration rate, pulse, blood pressure (diastolic and systolic), and weight will 
be summarized using descriptive statistics at each time point by dose cohort. Changes from 
baseline will be summarized in the same manner. 
14.5.7.5. Physical Examination 
Findings from the physical examinations will be presented as follows: pre-existing clinically 
significant conditions recorded as medical history will be summarized as described in 
Section 14.5.4, any new clinically significant conditions recorded as AEs will be summarized as 
described in Section 14.5.7.1. 
14.5.7.6. Concomitant Medications 
Other than the study drug, any medication taken by patients during the course of the study with a 
start date or an end date on or after the date of the first dose of study drug, or marked as ongoing, 
will be considered concomitant. Medications stopped prior to the date of the first dose of study 

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drug will not be considered concomitant. The number of patients using each concomitant 
medication will be summarized for each treatment according to the World Health Organization 
Drug Dictionary (WHODRUG) Anatomic Therapeutic Class (ATC) class and PT. Patients with 
multiple uses of a concomitant medication during a treatment period will be counted once for a 
given ATC and PT for the treatment period. 
14.5.8. 
Treatment of Missing Data 
For analyses of change from baseline, baseline will generally be defined as the predose 
assessment on Day 1 or during screening as scheduled. If this value is unavailable, the last 
non-missing value prior to the first dose of study drug will be used. Otherwise, missing 
observations will be treated as missing at random, and no data imputation will be performed. 

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15. 
COMPUTERIZED SYSTEMS USED FOR SOURCE DATA 
A list of the computerized systems that will be used at each step to create, modify, maintain, 
archive, retrieve, or transmit source data are presented below, per the Guidance for Industry 
Computerized Systems Used in Clinical Investigations, May 2007. 
Table 7: 
Computerized Systems Used for Source Data 
Protocol Step  
Computerized System Type or 
Description 
Informed Consent 
NA 
Inclusion/Exclusion Review 
A 
Continuation Criteria  
A 
Study Drug Administration 
A 
Medical History 
A 
Physical Exam 
A 
Height 
A 
Weight 
A 
Vital Signs 
A 
ECOG performance status 
A 
HLA typing 
B 
FSH testing 
A 
Serum Pregnancy Test 
A 
Serology 
A 
Hematology/Serum Chemistry  
A 
Urinalysis 
A 
12-Lead ECG 
C 
DTH Skin Test 
B 
Blood Sample for CTL 
B 
Blood Sample for Anti-WT1 Antibody 
B 
Blood Sample for PBMC isolation 
B 
Blood Sample for Retrospective Biomarker Analysis 
B 
IHC Tumor Samples 
A 
Tumor Assessment 
A 
Adverse Events 
A 
Concomitant Medications 
A 
Disease Progression/ Survival 
A 
Statistical Analysis 
SAS®, version 9.1.3 or higher 
Abbreviations: CTL = cytotoxic T lymphocyte induction activity, DTH = delayed-type hypersensitivity reactivity, 
ECG = electrocardiogram, ECOG = Eastern Cooperative Oncology Group, EDC = electronic data capture, FSH = follicle-
stimulating hormone, HLA = human leukocyte antigen, IHC = immunohistochemistry, LIMS = laboratory information 
management system, NA = not applicable, PBMC = peripheral blood mononuclear cells, WT1 = Wilms’ tumor gene 
product 1 
Note: A = EDC (PRA International); B = LIMS/ASCII; C = Core Lab Over-read 

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16. 
ETHICAL AND REGULATORY OBLIGATIONS 
16.1. 
Study Conduct 
The Investigator agrees that the study will be conducted according to the protocol, the US Code 
of Federal Regulations (CFR), GCP (E6), and the ethical principles that have their origin in the 
Declaration of Helsinki and the International Conference on Harmonisation (ICH) guidelines. 
The Investigator will conduct all aspects of this study in accordance with all national, state, and 
local laws of the pertinent regulatory authorities. 
The Investigator will assure proper implementation and conduct of the study including those 
study-related duties delegated to other appropriately qualified individuals. The Investigator will 
assure that study staff cooperate with monitoring and current audits, and will demonstrate due 
diligence in recruiting and screening study patients. 
The Investigator must sign and return to Contract Research Organization (CRO)/Sponsor the 
"Study Acknowledgment" page and provide a copy of current curriculum vitae (CV), including a 
copy of a current medical license, and financial disclosure. 
For all studies conducted under an Investigational New Drug (IND), the Investigator must sign 
and return a completed Form FDA 1572 "Statement of Investigator" to CRO/Sponsor. 
16.2. 
Institutional Review Board or Independent Ethics Committee 
Before initiation of the study, the Investigator/CRO must obtain approval or favorable opinion of 
the research protocol, informed consent form, and any advertisement for patient recruitment, 
from an IRB or Independent Ethics Committee (IEC) complying with the provisions specified in 
21 CFR Part 56 or in ICH GCP, as applicable, and applicable pertinent government regulations. 
The Investigator must assure IRB or IEC compliance with the applicable regulations. 
A copy of written IRB or IEC approval or favorable opinion of the protocol, informed consent 
form and advertising (if applicable) must be provided to CRO/Sponsor prior to initiation of the 
study. The approval or favorable opinion letter must be signed by the IRB or IEC chairman or 
designee, identify the IRB/IEC name and address, identify the clinical protocol by title and/or 
protocol number, and include the date that approval or favorable opinion was granted. The letter 
must also contain a statement that the IRB or IEC complies with the requirements in 21 CFR 
Part 56 for a study conducted under an IND or ICH GCP, as applicable. 
The Investigator/CRO is responsible for obtaining continued review of the clinical research or 
submitting periodic progress reports, in accordance with applicable regulations, at intervals not 
exceeding one year or otherwise specified by the IRB or IEC. The Sponsor must be supplied 
with written documentation of continued review of the clinical research. 
The Investigator must promptly inform their IRB/IEC of all SAEs or other safety information 
reported from CRO/Sponsor in accordance with 21 CFR 312.66, or when dictated by applicable 
local regulations (ie, Directive 2001/20/EC), the Sponsor/CRO is responsible for reporting to the 
IEC (eg, reporting of serious AEs). 

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16.3. 
Informed Consent 
The Investigator will prepare the informed consent form and provide the form to CRO/Sponsor 
for approval prior to submission to the IRB or IEC. CRO/Sponsor may provide a template 
informed consent form to be qualified by each research facility to conform to local requirements. 
All informed consent forms must contain the minimum elements as mandated by the FDA or 
governing regulatory authority and ICH guidelines and will be subject to CRO/Sponsor approval 
as well as IRB or IEC approval. CRO/Sponsor may submit informed consent forms to a central 
IRB or IEC for review and approval or favorable opinion contingent upon prior Investigator 
permission and review. 
Before recruitment and enrollment, each prospective candidate will be given a full explanation of 
the study, allowed to read the approved informed consent form and be provided ample time and 
the opportunity to ask any questions that may arise. Once all questions have been answered and 
the Investigator is assured that the individual understands the implications of participating in the 
study, the patient will be asked to give consent to participate in the study by signing the informed 
consent form. As part of the consent process, each patient must consent to direct access to his/her 
medical records for study-related monitoring, auditing, IRB/IEC review, and regulatory 
inspection. The Investigator will provide a copy of the signed informed consent form to each 
patient. It should be clearly explained to each patient that participation in each and every clinical 
visit and assessment is expected. The patient may be discontinued from study drug, but that does 
not necessarily negate the expectation that the patient will continue to participate in the study 
through the final visit/assessment. 
If an amendment to the protocol changes the patient participation schedule in scope or activity, 
or increases the potential risk to the patient, the informed consent form must be revised, 
submitted to the IRB or IEC for review and approval or favorable opinion. The revised informed 
consent form must be used to obtain consent from a patient currently enrolled in the study if he 
or she is affected by the amendment. The revised informed consent form must be used to obtain 
consent from any new patients who are enrolled into the study after the date of the approval or 
favorable opinion of the amendment. 
16.4. 
Patient Privacy 
The Sponsor’s staff or any designees affirm and uphold the patient’s confidentiality. Throughout 
this study, all data forward to the Sponsor will be identified only by an identification number, 
date of birth, gender, and initials. The Investigator agrees that the Sponsor’s representatives, its 
designee, representatives of the relevant IRB/IEC or representatives of the regulatory authorities 
will be allowed to review that portion of the patient’s primary medical records that directly 
concerns this study (including, but not limited to, clinical laboratory test result reports, ECG 
reports, admission/discharge summaries for hospital admissions occurring during a patient’s 
study participation and autopsy reports for deaths occurring during the study). 
For the studies conducted in the US, in accordance with the Healthcare Insurance, Portability, 
and Accountability Act of 1996, the Investigator will prepare a privacy authorization form and 
provide the form to CRO/Sponsor for approval prior to submission to the IRB/IEC or to a 
Privacy Board. CRO/Sponsor may provide a template privacy authorization form to be qualified 
by each research facility to conform to local requirements. The content of the privacy 

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authorization form must comply with the regulations governing the authorization. All 
prospective study candidates will be given full explanation of the privacy authorization form, 
allowed to read the approved form, and be provided the opportunity to ask any questions. Once 
all questions have been answered and the Investigator is assured that the individual understands 
the implications of the privacy authorization form, the patient will be asked to sign the privacy 
authorization. The authorization remains in effect until revoked by the patient. The Investigator 
will provide a copy of the signed privacy authorization form to each patient. Patients who do not 
sign the privacy authorization form will not be permitted to participate in the study. 
16.5. 
Protocol Amendments and Emergency Deviations 
Changes to the research covered by this protocol must be implemented by formal protocol 
amendment. Amendments to the protocol may be initiated by the Sponsor or at the request of the 
Investigator. In either case, a formal amendment cannot be initiated until the Sponsor has 
approved it, the Investigator has signed it off, and it has been reviewed and has received approval 
or favorable opinion by the IRB or IEC. 
Emergency deviations or modifications may be initiated without the Sponsor’s or IRB/IEC 
approval or favorable opinion, only in cases where the change is necessary to eliminate an 
immediate apparent hazard to patients. Emergency deviations or modifications must be reported 
to CRO/Sponsor and the IRB/IEC within five business days of the occurrence, or in accordance 
with applicable regulatory requirements. 
The study will not be un-blinded (where applicable) until all data have been reviewed, data entry 
has been completed, and the database is locked. 
16.6. 
Monitoring and Auditing of the Study 
A clinical monitor, whether an employee of the Sponsor or its designated representative, has the 
obligation to follow this study closely. In doing so, the monitor will visit the clinical study sites 
at periodic intervals, in addition to maintaining necessary telephone and letter contact. The 
monitor will maintain current personal knowledge of the study through observation, review of 
study records and source documentation, and discussion of the conduct of the study with the 
Investigator and staff. Quality assurance auditors, whether an employee of the Sponsor or its 
designated representative, may evaluate the conduct of the study by the clinical study sites. 
These parties must have access to any and all study-related documentation including source 
documentation, regardless of location and format. The Sponsor audit reports will be kept highly 
confidential. 
16.7. 
Study Documentation 
As part of the responsibilities assumed by participating in the study, the Investigator agrees to 
maintain adequate case histories for the patients treated as part of the research under this 
protocol. The Investigator agrees to maintain accurate source documentation and CRFs as part of 
the case histories. 
Study records are comprised of source documents, CRFs, and all other administrative 
documents, eg, IRB/IEC correspondence, clinical study materials and supplies shipment 

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manifests, monitoring logs, Sponsor and CRO correspondence, etc. A study specific binder will 
be provided with instructions for the maintenance of study records. 
Source documentation is defined as any hand written or computer generated document that 
contains medical information or test results that have been collected for or is in support of the 
protocol specifications, eg, clinical lab reports, clinic notes, drug disbursement log, patient sign 
in sheets, patient completed questionnaires, telephone logs, ECGs, etc. All draft, preliminary and 
pre-final iterations of a final report are also considered to be source documents, eg, faxed lab 
reports and hard copy lab reports, faxed initial results and hard copy, final report. 
CRO/Sponsor will supply CRFs. All requested information must be entered on the CRFs. Every 
effort should be made to complete all forms in their entirety. If an item is not available or is not 
applicable, this fact should be indicated; do not leave a space blank. Each set of completed CRFs 
must be reviewed, electronically signed and dated by the Investigator. 
16.8. 
Laboratory Certification and Normal Values 
A local laboratory will be used for analysis for most of the clinical labs for this study. The site 
personnel will provide the CRO and Sponsor with laboratory certification(s), a dated copy of 
normal range values for the local laboratory clinical laboratory selected to analyze clinical 
specimens, and the lab director’s CV. 
16.9. 
Drug Accountability 
The Investigator is responsible for storing the drug in a secure location and for maintaining 
adequate records of drug disposition that includes the dates, quantity, and use by patients. If the 
investigation is terminated, discontinued, suspended, or completed, all unused supplies of drug 
will be returned to the Sponsor, unless other instructions are provided in writing by 
CRO/Sponsor. 
The drug will not be dispensed to any person who is not a study patient under this protocol. 
16.10. 
Records Retention 
The Investigator agrees to retain study records for the time periods stated below. The 
Investigator agrees to contact CRO/Sponsor before destroying any study documentation. Should 
the Investigator leave the site at which the study was conducted, CRO/Sponsor will be contacted 
regarding the disposition of document storage. 
Records will be retained until at least 2 years after the last approval of a marketing application in 
an ICH region and until there are no pending or contemplated marketing applications in an ICH 
region or at least 2 years have elapsed since the formal discontinuation of clinical development 
of the investigational product. 
Dates marking the beginning of the final record retention periods will be sent in writing from 
CRO/Sponsor. If an Investigator withdraws from the study (eg, relocation), the records will be 
transferred to a mutually agreed upon designee (ie, another Investigator). This transfer is subject 
to Sponsor approval and will be documented in writing and a copy sent to the Sponsor. 

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Electronic Data Capture/ Electronic Patient Reported Outcomes (ePRO) Data Archiving: 
In compliance with data retention requirements, and after the capture phase of the study is 
complete, the site will receive a copy of all eSource data and accompanying audit trail from the 
EDC and/or ePRO vendors. The vendors shall certify the integrity of the copy (certified copy) 
and send it on commonly readable storage material (CD or DVD); if necessary, software for 
viewing the data files and instruction on installation and use of the software will also be 
included. This read-only archive will be retained, protected, and made accessible by the site 
throughout the required retention period. 
16.11. 
Inspection of Records 
In the event of an inspection, the Investigator agrees to allow representatives of the Sponsor, its 
representative, the Food and Drug Administration or other regulatory authorities’ access to all 
study records. The Investigator will promptly notify CRO/Sponsor of all requests to inspect by 
government agencies and will promptly forward a copy of all such inspection reports. 
16.12. 
Financial Disclosure 
Prior to the start of the study, Investigators will release sufficient and accurate financial 
information that permits CRO/Sponsor to demonstrate that an Investigator and all sub-
Investigators listed on the Form FDA 1572, if appropriate, have no personal or professional 
financial incentive regarding the future approval or disapproval of the study medication such that 
his or her research might be biased by such incentive. Investigators will provide an update of the 
above financial information at the end of the study and one year following the end of the study. 

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17. 
STUDY ACKNOWLEDGMENT 
I have read the foregoing protocol, D8350004, Version 7.0, “Initial Phase 1 Study of WT2725 
Dosing Emulsion in Patients with Advanced Malignancies”, and agree that it contains all 
necessary details for conducting this study and to conduct the study in strict accordance with the 
specifications outlined herein. 
I agree that no additional procedure(s) will be added during the conduct of the study except 
through protocol amendment by Sunovion Pharmaceuticals Inc. and after documentation of IRB 
approval. 
Investigator Signature: ____________________________________________________ 
Print Investigator Name: __________________________________________________ 
Date: ___________________________________________ 

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18. 
 REFERENCES 
Buonaguro L, Petrizzo A, Tornesello ML, Buonaguro FM. Translating tumor antigens into 
cancer vaccines. Clin Vaccine Immunol 2011;18(1):23-34. 
Cheson BD, Bennett JM, Kopecky KJ, Buchner T, Willman CL, Estey EH, et al. Revised 
recommendation of the international working group for the diagnosis, standardization of 
response criteria, treatment outcomes, and reporting standards for therapeutic trials in acute 
myeloid leukemia. J Clin Oncol 2003;21:4642-9. 
Eifuku R, Takenoyama M, Yoshino I, Imahayashi S, So T, Yasuda M, et al. Analysis of MAGE-
3 derived synthetic peptide as a human lung cancer antigen recognized by cytotoxic T 
lymphocytes. Int J Clin Oncol 2001;6:34-9. 
Fleischhauer K, Tanzarella S, Wallny H, Bordignon C, Traversari C. Multiple HLA-A alleles can 
present an immunodominant peptide of the human melanoma antigen Melan-A/MART-1 to a 
peptide-specific HLA-A*0201+ cytotoxic T cell line. J Immunol 1996;157:787-97. 
Franko A, Magliocco AM, Duan Q, Duggan MA. WT1 Immunoprofiling and Comparison of 
Malignant Mullerian Mixed Tumors of The Female Genital Tract. International Journal of 
Gynecological Pathology 2010;29:452–8. 
Gentilini C, Letsch A, Asemissen A, Muessig A, Rieger K, Scheibenbogen C, et al. Vaccination 
with WT-1 126-134 peptide in patients with acute myeloid leukemia after allogenic stem cell 
transplantation. Blood 2006;108(11):1052a-53a. 
Hoos A, Eggermont AMM, Janetzi S, Hodi FS, Ibrahim R, Anderson A, et al. Improved 
endpoints for cancer immunotherapy trials. JNCI 2010;102(18):1388-97. 
Hutchings Y, Osada T, Woo CY, Clay TM, Lyerly HK, et al. Immunotherapeutic targeting of 
Wilms' tumor protein. Curr Opin Mol Ther 2007;9(1):62-9. 
Kaida M, Morita-Hoshi Y, Soeda A, Wakeda T, Yamaki Y, et al. Phase 1 Trial of Wilms tumor 1 
(WT1) peptide vaccine and gemcitabine combination therapy in patients with advanced 
pancreatic or biliary tract cancer. J Immunother 2011;34(1):92-9. 
Keilholz U, Letsch A, Busse A, Asemissen AM, Bauer S, et al. A clinical and immunologic 
phase 2 trial of Wilms tumor gene product 1 (WT1) peptide vaccination in patients with AML 
and MDS. Blood 2009;113(26):6541-8. 
Kuball J, de Boer K, Wagner E, Wattad M, Antunes E, et al. Pitfalls of vaccinations with WT1-, 
Proteinase3-, and MUC1-derived peptides in combination with MontanideISA51 and CpG7909. 
Cancer Immunol Immunoth 2011;60:161-71. 
Li Z, Oka Y, Tsuboi A, Fujiki F, Harada Y, Nakajima H, et al. Identification of a WT1 protein-
derived peptide, WT1, as a HLA-A 0206-restricted, WT1-specific CTL epitope. Microbiol 
Immunol 2008; 52:551-8, 
Maslak PG, Dao T, Krug LM, Chanel S, Korontsvit T, et al. Vaccination with synthetic analog 
peptides derived from WT1 oncoprotein induces T-cell responses in patients with complete 
remission from acute myeloid leukemia. Blood 2010;116(2):171-9. 

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Nakatsuka S, Oji Y, Horiuchi T, Kanda T, Kitagawa M, et al. Immunohistochemical detection of 
WT1 protein in a variety of cancer cells. Modern Pathology 2006;19:804-14. 
National Cancer Institute (NCI). Common Terminology Criteria for Adverse Events v4.0. 
http://ctep.cancer.gov/protocolDevelopment/electronic_applications/ctc.htm#ctc_40. NIH 
publication # 09-7473. Published 29 May 2009. Accessed 17 Jan 2012. 
Oji Y, Inohara H, Nakazawa M, Nakano Y, Akahani S, Nakatsuka S, et al. Overexpression of the 
Wilms’ tumor gene WT1 in head and neck squamous cell carcinoma. Cancer Sci 2003;94:523-9. 
Oji Y, Suzuki T, Nakano Y, Maruno M, Nakatsuka S, Jomgeow T, et al. Overexpression of the 
Wilms' tumor gene WT1 in primary astrocytic tumors. Cancer Sci 2004;95:822-7. 
Oka Y, Tsuboi A, Taguchi T, Osaki T, Kyo T, Nakajima H, et al. Induction of WT1 (Wilms’ 
tumor gene)-specific cytotoxic T lymphocytes by WT1 peptide vaccine and the resultant cancer 
regression. Proc Natl Acad Sci (USA) 2004;101(38):13885-90. 
Oka Y, Kawase I. Cancer antigen WT1-targeting treatment for the malignancies - Development 
of WT1 peptide vaccine. Jpn J Clin Immunol 2008;31(5):375-82. 
Rezvani K, Yong AS, Mielke S, Savani B, Musse L, et al. Leukemia-associated antigen-specific 
T-cell responses following combined PR1 and WT1 peptide vaccination in patients with myeloid 
malignancies. Blood 2008;111(1):236-42. 
Skolnik JM, Barrett JS, Jayaraman B, Patel D, Adamson PC. Shortening the timeline of pediatric 
phase I trials: the rolling six design. J Clin Oncol 2008;26(2):190-5. 
Soeda A, Morita-Hoshi Y, Kaida M, Wakeda T, Yamaki Y, Kojima Y, et al. Long-term 
administration of Wilms Tumor-1 peptide vaccine in combination with gemcitabine causes 
severe local skin inflammation at injection sites. Jpn J Clin Oncol 2010;40(12):1184–8. 
Sudo T, Kamikawaji N, Kimura A, Date Y, Savoie CJ, Nakashima H, et al. Differences in MHC 
class I self peptide repertoires among HLA-A2 subtypes. J Immunol 1995;155:4749-56. 
Sugiyama H. Cancer immunotherapy targeting Wilms' tumor gene WT1 product. Expert Rev 
Vaccines 2005;4(4):503-12. 
Wolchok JD, Hoos A, O'Day S, Weber JS, Hamid O, Lebbé C, et al. Guidelines for the 
Evaluation of Immune Therapy Activity in Solid Tumors: Immune-Related Response Criteria. 
Clin Cancer Res 2009;15(23):7412-20. 
Zhang HG, Pang XW, Shang XY, Xing Q, Chen WF. Functional supertype of HLA-A2 in the 
presentation of Flu matrix p58-66 to induce CD8+ T-cell response in a Northern Chinese 
population. Tissue Antigens 2003; 62:285-95. 

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19. 
APPENDIX I. CARDIAC SAFETY MONITORING (ECG) 
1. Requirements for Testing 
ECG equipment and supplies will be provided by ECG Vendor and should be used for all in-
clinic protocol ECG assessments. 
• All 12-lead ECGs will be recorded in the same manner. 
• The site personnel must be adequately trained in performing ECGs on the specific 
ECG equipment used in this protocol that is provided by the cardiac safety vendor. 
• To the extent possible, the same ECG machine and personnel should be used to 
acquire a patient’s ECGs throughout the period of their participation in the study. 
• Indelible ink will be used to mark the placement of the leads on the skin to ensure 
consistent placement throughout the study. 
• ECGs will be recorded with at least one 10-second single-lead tracing recorded from 
Lead II. 
2. Patient Restrictions and Instructions 
• Prior to ECG acquisition, the patient will have rested 10 minutes in the supine 
position and will remain so until the ECG is obtained. 
3. Reporting 
• It is the responsibility of the Investigator to perform a safety review of the ECG data 
for changes from previous assessments and/or emergent cardiac dysfunction, and to 
determine patients’ eligibility or continuance in the study. 
• ECGs will be reviewed (signed and dated as necessary when an electronic system is 
not used) by an Investigator listed on the Form FDA 1572 (MD or DO) after each 
ECG collection. The same Investigator should review all ECG reports for a given 
patient whenever possible. 
• For all ECGs, a report will be provided by the cardiac safety vendor to the site for 
review and signature. 
• The ECG tracing will be kept with patient’s source documentation and / or CRF 
unless it is specified otherwise. The original ECG and the cardiologist’s over-read 
will be retained at the site. 
4. Data Standardization 
ECG data will be transmitted to a centralized cardiac safety vendor and centrally over-read and 
interpreted using standardized procedures. 
 

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20. 
APPENDIX II. 
EASTERN COOPERATIVE ONCOLOGY 
GROUP (ECOG) PERFORMANCE STATUS 
 
Grade 
Performance Status 
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, eg, light house work, 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 
Completely disabled. Cannot carry on any self care. Totally confined to bed or chair 
5 
Dead 
As published in Am. J. Clin. Oncol: Oken MM, Creech RH, Tormey DC, Horton J, Davis TE, McFadden ET, Carbone PP: 
Toxicity and Response Criteria of the Eastern Cooperative Oncology Group. Am J Clin Oncol 5:649-55, 1982. 

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21. 
APPENDIX III. NEW YORK HEART ASSOCIATION [NYHA] 
FUNCTIONAL CLASSIFICATION 
 
Class 
Functional Capacity 
I 
Patients with cardiac disease but without resulting limitation of physical activity. 
Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea, or 
anginal pain. 
II 
Patients with cardiac disease resulting in slight limitation of physical activity. They 
are comfortable at rest. Ordinary physical activity results in fatigue, palpitation, 
dyspnea, or anginal pain. 
III 
Patients with cardiac disease resulting in marked limitation of physical activity. They 
are comfortable at rest. Less than ordinary activity causes fatigue, palpitation, 
dyspnea, or anginal pain. 
IV 
Patients with cardiac disease resulting in inability to carry on any physical activity 
without discomfort. Symptoms of heart failure or the anginal syndrome may be 
present even at rest. If any physical activity is undertaken, discomfort is increased. 
The Criteria Committee of the New York Heart Association. Nomenclature and Criteria for Diagnosis of Diseases of the Heart 
and Great Vessels. 9th ed. Boston, Mass: Little, Brown & Co; 1994:253-256. excerpted on American Heart Association 
[homepage on internet]. Dallas, TX, US [updated 18 Mar 2011; cited 14 Dec 2011]. Available from: 
http://my.americanheart.org/professional/StatementsGuidelines/ByPublicationDate/PreviousYears/Classification-of-
Functional-Capacity-and-Objective-Assessment_UCM_423811_Article.jsp 

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22. 
APPENDIX IV.  CLINICAL LABORATORY TESTS 
The following laboratory tests are to be performed: 
Clinical Safety Panel 
HEMATOLOGY: (Differential reported as % and absolute value) 
Hemoglobin, Hematocrit, Platelet Count, Red Blood Cell (RBC) Count, White Blood Cell 
(WBC) Total Count, WBC Differential, (Basophils, Eosinophils, Lymphocytes, Monocytes, 
Neutrophils) 
 
BLOOD CHEMISTRIES: 
Alanine aminotransferase (ALT), Alkaline Phosphatase (ALP), Aspartate aminotransferase 
(AST), Bicarbonate (HCO3), Bilirubin (Total, Direct, Indirect), Blood Urea Nitrogen (BUN), 
Calcium (Ca), Chloride (Cl), Creatinine, Glucose, Magnesium (Mg), Phosphorus (P), Potassium 
(K), Protein (Total), Sodium (Na), Uric Acid, Albumin 
 
URINALYSIS: 
Blood, Glucose, Ketones, Leukocyte esterase, Nitrites, pH, Protein 
Microscopic examination will be conducted if blood or protein is 2+ or higher. 
 
OTHER TESTS: 
Hepatitis B Aga, Hepatitis C Aba, Serum Pregnancy (β-HcG) (in female patients of child-bearing 
potential only), HIV-1 Aba, HIV-2 Aba. 
a For patients with any signs or symptoms suggestive of infection. 
 
Laboratory reports will be initialed and dated on all pages by a 1572-listed investigator (MD or 
DO). Laboratory test results will be reviewed by the Investigator as they become available. 
Possibly study drug-related or clinically relevant abnormal values of uncertain causality must be 
repeated. Any abnormal values that persist should be followed at the discretion of the 
Investigator. 
 
LABORATORY TESTING SCHEDULE 
Laboratory testing to be performed at each visit is indicated in Appendix Table 8 for Part 1 of the 
study and Appendix Table 9 for Part 2 of the study. 
 

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Appendix Table 8: Schedule of Clinical Laboratory Tests – Part 1 
 
Screening 
Vaccine Induction Phase 
Consolidation Phase 
Maintenance 
Phase 
End of Study 
 
 
1st dose 
2nd dose 
3rd dose 
4th dose 
5th dose 
6th dose 
7th dose 
8th dose 
9th dose 
 
 
Test 
 
Day 1     
(-1) 
Day 8    
(-1) 
Day 15 
(-1) 
Day 22 
(-1) 
 
Day 29 
(-1) 
Day 43 
(±2) 
Day 57 
(±2) 
Day 71 
(±2) 
Day 85 
(±2) 
every 28 (+7) 
days  
within 28 
days after 
last dose 
Hematology 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Serum 
chemistry 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Urinalysis 
X 
X 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
Hepatitis B 
Aga 
X 
 
 
 
 
 
 
 
 
 
 
 
Hepatitis C 
Aba 
X 
 
 
 
 
 
 
 
 
 
 
 
HIV-1a 
X 
 
 
 
 
 
 
 
 
 
 
 
HIV-2a 
X 
 
 
 
 
 
 
 
 
 
 
 
Serum 
Pregnancy 
(β-HcG)b 
X 
X 
 
 
 
 
 
 
 
 
 
X 
a For patients with any signs or symptoms suggestive of infection. 
b For female patients of child-bearing potential only. 
 

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Appendix Table 9: Schedule of Clinical Laboratory Tests – Part 2 
 
SCR 
Vaccine Induction Phase 
Consolidation Phase 
Maintenance 
Phase 
End of 
Study 
 
 
1st 
dose 
2nd 
dose 
3rd 
dose 
4th 
dose 
5th 
dose 
6th 
dose 
7th 
dose 
8th 
dose 
9th 
dose 
10th 
dose 
11th 
dose 
12th 
dose 
13th 
dose 
14th 
dose 
15th 
dose 
 
 
 
 
Day 1 
(-1) 
Day 8 
(-1) 
Day 15 
(-1) 
Day 22 
(-1) 
Day 29 
(-1) 
Day 
36       
(-1) 
Day 
43  
(-1) 
Day 
50  
(-1) 
Day 
57  
(-1)  
Day 
71 
(±2) 
Day 
85 
(±2) 
Day 
99 
(±2) 
Day 
113 
(±2) 
Day 
127 
(±2) 
Day 
141 
(±2) 
every 28 (+7) 
days  
within 
28 days 
after 
last 
dose 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Hematology 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Serum 
chemistry 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Urinalysis 
X 
X 
 
X 
 
X 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Hepatitis B 
Aga 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Hepatitis C 
Aba 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
HIV-1a 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
HIV-2a 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Serum 
Pregnancy 
(β-HcG)b 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
Abbreviations: SCR = screening 
a For patients with any signs or symptoms suggestive of infection. 
b For female patients of child-bearing potential only.

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APPENDIX V. BIOMARKER SAMPLING AND PROCESSING 
BIOMARKER SAMPLING SCHEDULE 
Biomarker samples to be collected at each visit are indicated in Appendix Table 10 for Part 1 of 
the study and Appendix Table 11 for Part 2 of the study. Procedures for sample processing and 
handling are detailed in separate manuals. 

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Appendix Table 10: Schedule of Biomarker Sampling – Part 1 
 
Screening 
Vaccine Induction Phase 
Consolidation Phase 
Maintenance 
Phase 
End of 
Study 
 
 
1st 
dose 
 
2nd 
dose 
3rd 
dose 
4th 
dose 
5th 
dose 
 
6th 
dose 
7th 
dose 
8th 
dose 
9th 
dose 
 
 
 
Biomarker 
 
Day 1 
(-1) 
Day 
3 
Day 8 
(-1) 
Day 
15 (-1) 
Day 
22 (-1) 
Day 
29 (-1) 
Day 
31 
Day 
43 
(±2) 
Day 
57 
(±2) 
Day 
71 
(±2) 
Day 
85 
(±2) 
Day 87 
every 28 (+7) 
days  
within 28 
days 
after last 
dose 
DTH Skin Test 
Evaluation 
 
X 
X 
 
 
 
X 
X 
 
 
 
X 
X 
 
X 
Blood for CTL 
X 
X 
 
X 
X 
X 
X 
 
X 
X 
X 
X 
 
X 
X 
Blood for Anti-
WT1 Antibody 
 
X 
 
 
X 
 
X 
 
 
X 
 
X 
 
Xa 
X 
Blood for 
PBMC isolation 
 
X 
 
 
 
 
X 
 
 
 
 
 
 
 
X 
Blood for 
retrospective 
biomarker 
analyses 
 
X 
X 
X 
X 
 
X 
 
 
X 
 
X 
 
Xa 
X 
IHC Tumor 
Tissue 
Samplesb 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
Xc 
Abbreviations: CTL = cytotoxic T lymphocyte induction activity, DTH = delayed-type hypersensitivity reactivity, IHC = immunohistochemistry, PBMC = peripheral blood 
mononuclear cell, WT1 = Wilms’ tumor 
a Perform every 2nd dosing day starting after the 9th dose. 
b In place of tumor tissue samples from a biopsy during screening, archived tumor tissue samples may be provided. Availability of tumor tissue samples should be determined 
during screening through provision of the accession number or other identification number. If necessary, the tumor tissue sample biopsy should be performed after study 
eligibility has been confirmed and before the first dose of study drug. The tumor tissue samples will only be provided to the sponsor for patients who receive study drug. In place 
of archival tumor tissue samples, patients with AML should have available the results from a bone marrow aspirate and/or bone marrow biopsy with PCR for WT1 transcript 
performed before the first dose of study drug. For patients with AML, bone marrow aspirations/biopsies are scheduled when clinically indicated, however peripheral blood 
assessed for blasts and WT1 transcript at least at baseline and every scheduled tumor assessment. 
c Biopsy for tumor tissue samples after the last dose of study drug is not mandatory. Tumor samples will be obtained from all patients who provided consent. IHC need not be 
performed for patients with AML provided RT-PCR for WT1 transcript is assessed. 

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Appendix Table 11: Schedule of Biomarker Sampling – Part 2 
 
SCR 
Vaccine Induction Phase 
 
 
1st dose 
 
2nd dose 
3rd dose 
4th dose 
 
5th 
dose 
 
6th dose 
7th dose 
8th dose 
9th dose 
Biomarker 
 
Day 1 (-1) 
Day 3 
Day 8 
(-1) 
Day 15 
(-1) 
Day 22 
(-1) 
Day 27 
Day 29 
(-1) 
Day 31 
Day 36 
(-1) 
Day 43 
(-1) 
Day 50 
(-1) 
Day 57 
(-1) 
DTH Skin Test 
Evaluation 
 
X 
X 
 
 
 
X 
X 
X 
 
 
 
 
Blood for CTL 
X 
X 
 
X 
X 
X 
 
X 
 
X 
X 
X 
X 
Blood for Anti-WT1 
Antibody 
 
X 
 
 
X 
 
 
X 
 
 
 
 
X 
Blood for PBMC 
isolation 
 
X 
 
 
 
 
 
X 
 
 
 
 
 
Blood for retrospective 
biomarker analyses 
 
X 
X 
X 
X 
 
 
X 
 
 
 
 
X 
IHC Tumor Tissue 
Samplesb 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 

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Appendix Table 11: Schedule of Biomarker Sampling – Part 2 (Continued)  
 
Consolidation Phase 
Maintenance 
Phase  
End of 
Study 
 
10th dose 
 
11th dose 
 
12th dose 
13th dose 
14th dose 
15th dose 
 
 
Biomarker 
Day 71 (±2) 
Day 83 
Day 85 (±2) 
Day 87 
Day 99 (±2) 
Day113 (±2) 
Day 127 
(±2) 
Day141 (±2) 
every 28 (+7) 
days 
within 28 
days after 
last dose 
DTH Skin Test 
Evaluation 
 
 
X 
X 
 
 
 
 
 
X 
Blood for CTL 
X 
 
X 
 
X 
X 
X 
X 
X 
X 
Blood for Anti-WT1 
Antibody 
 
 
X 
 
 
X 
 
X 
Xa 
X 
Blood for PBMC 
isolation 
 
 
 
 
 
 
 
 
 
X 
Blood for retrospective 
biomarker analyses 
 
 
X 
 
 
X 
 
X 
Xa 
X 
IHC Tumor Tissue 
Samplesb 
 
 
 
 
 
 
 
 
 
Xc 
Abbreviations: CTL = cytotoxic T lymphocyte induction activity, DTH = delayed-type hypersensitivity reactivity, IHC = immunohistochemistry, opt = optional, PBMC = peripheral blood mononuclear 
cell, SCR = screening, WT1 = Wilms’ tumor 
a Perform every 2nd dosing day during the Maintenance Phase. 
b In place of tumor tissue samples from a biopsy during screening, archived tumor tissue samples may be provided. Availability of tumor tissue samples should be determined during screening through 
provision of the accession number or other identification number. If necessary, the tumor tissue sample biopsy should be performed after study eligibility has been confirmed and before the first dose 
of study drug. The tumor tissue samples will only be provided to the sponsor for patients who receive study drug. In place of archival tumor tissue samples, patients with AML should have available 
the results from a bone marrow aspirate and/or bone marrow biopsy with PCR for WT1 transcript performed before the first dose of study drug. Patients with AML should also have peripheral blood 
assessed for blasts and WT1 transcript. 
c Biopsy for tumor tissue samples after the last dose of study drug is not mandatory. Tumor samples will be obtained from all patients who provided consent. IHC need not be performed for patients with 
AML provided RT-PCR for WT1 transcript is assessed. 

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APPENDIX VI. STUDY DRUG PREPARATION 
The following is a summary of the study drug preparation procedures. Complete instructions are 
provided in the Pharmacy Manual.  
Warning: When handling WT2725 Injection, Diluting Solution for WT2725 Injection (DSWI), 
and W/O pre-Emulsion (WOPE) wear proper protective equipment including gloves and safety 
glasses or goggles. Use standard procedures for investigational medications. 
The study drug contains 4 components that may be diluted prior to administration.  
1. WT2725 Injection 50 mg/mL 
2. WT2725 Injection 2 mg/mL 
3. Diluting Solution for WT2725 Injection (DSWI) 
4. W/O pre-Emulsion (WOPE) 
At the clinical site, WT2725 Injection (2 mg/mL and/or 50 mg/mL) is diluted to an appropriate 
concentration using DSWI, if needed. Subsequently, WT2725 Injection or diluted WT2725 
Injection is mixed with the WOPE at a ratio of 3:7 (by volume) to form the Dosing Emulsion 
(Appendix Figure 6). The Dosing Emulsion (Drug Product) is a white liquid emulsion and will 
be administered by sc injection. 
 
Appendix Figure 6: Description of WT2725 Dosing Emulsion (Drug Product) Preparation 
The following dose cohorts are planned for Part 1 of the study: 
DTH skin test injections only:  
0.01 mg WT2725 in diluted WT2725 Injection (0.05 mL x 1 
site, id) and Control (Diluting Solution for WT2725 Injection 
[DSWI]) (0.05 mL x 1 site, id) 
Cohort 1:  
0.3 mg WT2725 in Dosing Emulsion (0.3 mL x 2 sites for a total volume of 0.6 mL 
per patient, sc) 
Cohort 2:  
0.9 mg WT2725 in Dosing Emulsion (0.3 mL x 2 sites for a total volume of 0.6 mL 
per patient, sc) 
Cohort 3:  
3.0 mg WT2725 in Dosing Emulsion (0.3 mL x 2 sites for a total volume of 0.6 mL 
per patient, sc) 
WT2725 Injection 
or 
Diluted WT2725 Injection 
W/O pre-Emulsion 
(WOPE) 
Dosing Emulsion 
Mix before use 
Water phase 
(contains WT2725) 
＋
Oil phase 
3 
7 

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Cohort 4:  
9.0 mg WT2725 in Dosing Emulsion (0.3 mL x 2 sites for a total volume of 0.6 mL 
per patient, sc) 
Patients in the RP2D cohorts will initiate treatment at the RP2D (9.0 mg). 
The following dose cohorts are planned for Part 2 of the study: 
Cohort 5:  
18.0 mg WT2725 in Dosing Emulsion (0.6 mL x 2 sites for a total volume of 
1.2 mL per patient, sc) 
Cohort 6:  
27.0 mg WT2725 in Dosing Emulsion (0.9 mL x 2 sites for a total volume of 
1.8 mL per patient, sc) 
All study drug components (WT2725 Injection 50 mg/mL, WT2725 Injection 2 mg/mL, Diluting 
Solution for WT2725 Injection [DSWI], and W/O pre-Emulsion [WOPE]) should be stored 
under refrigeration (cold temperature 2°C to 8°C), and protected from light and freezing. All 
drug products must be prepared and stored under ambient conditions on the day of dosing, and 
used or discarded within 6 hours following preparation of the drug product. 
Following is a descriptive procedure to prepare diluted WT2725 Injection for DTH skin test 
injections (Appendix Table 12): 
Preparation of 0.2 mg/mL WT2725 Injection for DTH skin tests: Add 0.1 mL of WT2725 
Injection 2 mg/mL to DSWI and mix with a vortex mixer to obtain a 0.2 mg/mL WT2725 
Injection for DTH skin test. 
Preparation of Control Solution for DTH skin tests: Withdraw 0.05 mL from a vial of the DSWI 
to obtain the Control Solution for DTH skin test. 
Following is a descriptive procedure to prepare varying concentrations of Dosing Emulsion 
(Drug Product) for clinical administration (Appendix Table 12) in Parts 1 and 2 of the study: 
a. Preparation of 0.5 mg/mL Dosing Emulsion for Dose cohort 1: Add 0.2 mL of DSWI 
to WT2725 Injection 2 mg/mL and mix with a vortex mixer. Withdraw 0.3 mL of 
diluted WT2725 Injection, add to the WOPE and mix with a vortex mixer to obtain a 
0.5 mg/mL Dosing Emulsion. 
b. Preparation of 1.5 mg/mL Dosing Emulsion for Dose cohort 2: Add 0.1 mL of 
WT2725 Injection 50 mg/mL to the DSWI and mix with a vortex mixer. Withdraw 
0.3 mL of diluted WT2725 Injection, add to the WOPE and mix with a vortex mixer 
to obtain 1.5 mg/mL Dosing Emulsion. 
c. Preparation of 5 mg/mL Dosing Emulsion for Dose cohort 3: Add 0.45 mL of 
WT2725 Injection 50 mg/mL to the DSWI and mix with a vortex mixer. Withdraw 
0.3 mL of diluted WT2725 Injection, add to the WOPE and mix with a vortex mixer 
to obtain 5 mg/mL Dosing Emulsion. 
d. Preparation of 15 mg/mL Dosing Emulsion for Dose cohort 4, 5, and 6: Add 0.3 mL 
of WT2725 Injection 50 mg/mL to the WOPE and mix with a vortex mixer to obtain 
15 mg/mL Dosing Emulsion 
The following items will be supplied for preparation of DTH skin test injection and Dose 
cohorts 1 to 6: 
• 1 mL syringes 

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• 24 G needle 
• Vortex mixer  
 
The following items will be supplied for withdrawal and administration of DTH skin test 
injection and Dose cohorts 1 to 6: 
• 1 mL syringes 
• 25 - 27 G needle 
• Vortex mixer 
 
Appendix Table 12: Preparation of WT2725 Dosing Emulsion (Drug Product) – Parts 1 
and 2 
Dose 
cohort # 
Dose 
(mg/body) 
 
WT2725 
concentration 
in Dosing Emulsion 
(mg/mL) 
Dilution of WT2725 Injection 
Volume of WT2725 Injection 
added into a vial of DSWI (mL) 
WT2725 concentration 
prior to mixing with 
WOPE (mg/mL) 
DTH skin 
testa 
0.01 
 
0.1 mL of WT2725 Injection 
2 mg/mL 
NA 
(Not mixed with WOPE) 
1 
0.3b 
0.5 
0.2 mL of DSWI is added to a 
vial of WT2725 Injection 
2 mg/mL (1.0 mL/vial) 
1.667 
(dilution by 1.2) 
2 
0.9b 
1.5 
0.1 mL of WT2725 Injection 
50 mg/mL 
5 
(dilution by 10) 
 
3 
3.0b 
5 
0.45 mL of WT2725 Injection 
50 mg/mL 
16.67 
(dilution by 3) 
 
4 
9.0b 
15 
No dilution of WT2725 Injection 
50 mg/mL (ie, 0.3 mL of 
WT2725 Injection 50 mg/mL is 
mixed directly with WOPE) 
50 
(Not diluted) 
 
5 
18.0c 
15 
No dilution of WT2725 Injection 
50 mg/mL (ie, 0.3 mL of 
WT2725 Injection 50 mg/mL is 
mixed directly with WOPE) 
50 
(Not diluted) 
6 
27.0d 
15 
No dilution of WT2725 Injection 
50 mg/mL (ie, 0.3 mL of 
WT2725 Injection 50 mg/mL is 
mixed directly with WOPE) 
50 
(Not diluted) 
Abbreviations: DSWI = Diluting Solution for WT2725 Injection, DTH = delayed-type hypersensitivity,  
NA = not applicable, sc = subcutaneous, W/O = water-in-oil, WOPE = W/O pre-Emulsion 
a DTH skin test: 0.05 mL × 1 site (DTH testing solution is prepared by adding 0.1 mL of WT2725 Injection 2 mg/mL to a vial of 
DSWI). 
b 0.3 mL × 2 sites for a total volume of 0.6 mL per patient 
c 0.6 mL × 2 sites for a total volume of 1.2 mL per patient 
d 0.9 mL × 2 sites for a total volume of 1.8 mL per patient 

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25. 
APPENDIX VII. DTH SKIN TEST PROCEDURES 
DTH skin tests and evaluations will be performed at the times indicated on the Schedules of 
Assessments (Tables 3 - 6). At each time point indicated, both diluted WT2725 Injection for 
DTH skin test and the control will be administered and evaluated. 
 
Administration of diluted WT2725 Injection for DTH skin test: 
Use the diluted WT2725 Injection for DTH skin test (refer to Appendix VI for preparation). 
Perform intradermal injection of 50 μL (10 μg) into the forearm. 
 
Administration of control for DTH skin test: 
Use the Diluting Solution for WT2725 Injection (refer to Appendix VI for preparation). 
Perform intradermal injection of 50 μL into the same forearm at a site that is a distance of at least 
10 cm or more away from the dosing site of the diluted WT2725 Injection for DTH skin test. 
In the event of an immediate hypersensitivity reaction, future dosing should be evaluated by the 
investigator and medical monitor. 
 
Approximately 48 hours (but no sooner than 24 hours) following the skin test injections, evaluate 
DTH reactions: 
For each site of the DTH skin test injection visually inspect the test site and palpate the area: 
• measure the area of erythema, if any, at the 2 injection sites, across the long and short 
diameters 
• evaluate the quality of the reaction, such as redness, induration, ulceration, etc, if any, 
at the 2 injection sites 
 
If the DTH reactivity results in erythema with induration greater than 2 cm or leads to ulceration 
of the skin test site, further DTH testing should not be conducted for the patient. 

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26. 
APPENDIX VIII. DEFINITIONS FOR REPORTING ADVERSE 
EVENTS 
The Investigator must assess the severity of the AE using the Common Terminology Criteria for 
Adverse Events (CTCAE) V.4.0 (NCI 2009). 
The Investigator must assess the relationship of the AE to the study medication using the 
following: 
• Not related – improbable temporal relationship and is plausibly related to other drugs or 
underlying disease. 
• Possible - occurred in a reasonable time after study drug administration, but could be 
related to concurrent drugs or underlying disease. 
• Probable - occurred in a reasonable time after study drug administration, is unlikely to 
be attributable to concurrent drugs or underlying disease, and there is a plausible 
mechanism to implicate the study drug. 
• Definite - occurred in a reasonable time after study drug administration and cannot be 
explained by concurrent drugs or underlying disease. The adverse event should respond 
to dechallenge/rechallenge, however, this is not mandatory before assigning a definite 
causality. 
The action taken regarding study drug will be defined as follows: 
• Dose Not Changed – no change. 
• Dose Reduced. 
• Dose Increased. 
• Drug Interrupted – study drug stopped temporarily. 
• Drug Withdrawn – study drug stopped permanently. 
• Not Applicable.