A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Dose Study to Evaluate the Efficacy and Safety of Oral SKI-O-703, SYK Inhibitor, in Patients With Persistent and Chronic Immune Thrombocytopenia (ITP)
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Sponsor: Oscotec Inc.

CLINICAL STUDY PROTOCOL 
IND 139107 
EUDRA-CT 2018-003329-26 
 
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled, 
Parallel Dose Study to Evaluate the Efficacy and Safety of Oral SKI-O-703, 
SYK Inhibitor, in Patients with Persistent and Chronic Immune 
Thrombocytopenia (ITP) 
 
PROTOCOL NO. OSCO-P2101 
 
Page 1

Sponsor: 
Oscotec Inc. 
Korea Bio-Park, Building A, 9th Floor 
700 Daewangpangyo-ro, Bundang-gu 
Seongnam-si, Gyeonggi-do 13488 
The Republic of Korea 
Sponsor Contact: 
 
Medical Monitor: 
Version of Protocol:  
4.0 (Protocol Amendment 3) 
Date of Protocol:  
22 April 2020 
Previous Date(s) and 
Version(s): 
09 August 2019, Version 3.1 (Country-Specific Protocol 
Amendment 2.1 [Republic of Korea]) 
30 April 2019, Version 3.0 (Protocol Amendment 2) 
03 April 2019, Version 2.0 (Protocol Amendment 1) 
09 January 2019, Final (original) 
 
CONFIDENTIAL 
All financial and nonfinancial support for this study will be provided by Oscotec Inc. The 
concepts and information contained in this document or generated during the study are 
considered proprietary and may not be disclosed in whole or in part without the expressed, 
written consent of Oscotec Inc. 
The study will be conducted according to the International Council for Harmonisation 
harmonised tripartite guideline E6(R2): Good Clinical Practice. 
 
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Oscotec Inc. 
Protocol: OSCO-P2 IO I Version 4.0 (Amendment 3) 
Protocol Approval - Sponsor Signatory 
SKI-O-703 
22 April 
Study Title 
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-
Controlled, Parallel Dose Study to Evaluate the Efficacy and Safety of 
Oral SKI-O-703, SYK Inhibitor, in Patients with Persistent and 
Chronic Immune Thrombocytopenia (ITP) 
Protocol Number OSCO-P2101 
Protocol Date 
22 April 
Protocol accepted and approved by: 
Page 3

Oscotec Inc. 
Protocol: OSCO-P2101 Version 4.0 (Amendment 3) 
Protocol Approval - Principal/Coordinating Investigator 
SKI-O-703 
22 April 
Study Title 
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-
Controlled, Parallel Dose Study to Evaluate the Efficacy and Safety of 
Oral SKI-O-703, SYK Inhibitor, in Patients with Persistent and 
Chronic Immune Thrombocytopenia (ITP) 
Protocol 
Number 
Protocol Date 
OSCO-P2101 
22 April 
Protocol accepted and approved by: 
Page 4

Oscotec Inc. 
Protocol: OSCO-P2101 Version 4.0 (Amendment 3) 
Protocol Approval - Lead Statistician 
SKI-O-703 
22 April 2020 
Study Title 
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-
Controlled, Parallel Dose Study to Evaluate the Efficacy and Safety of 
Oral SKI-O-703, SYK Inhibitor, in Patients with Persistent and 
Chronic Immune Thrombocytopenia (ITP) 
Protocol Number OSCO-P2101 
Protocol Date 
22 April 2020 
Protocol accepted and approved by: 
Page 5

Oscotec Inc. 
Protocol: OSCO-P2101 Version 4.0 (Amendment 3) 
Declaration of Investigator 
SKI-O-703 
22 April 
I have read and understood all sections of the protocol entitled "A Phase 2, Multicenter, 
Randomized, Double-Blind, Placebo-Controlled, Parallel Dose Study to Evaluate the 
Efficacy and Safety of Oral SKI-O-703, SYK Inhibitor, in Patients with Persistent and 
Chronic Immune Thrombocytopenia (ITP)" and the accompanying investigator's brochure. 
I agree to supervise all aspects of the protocol and to conduct the clinical investigation in 
accordance with the Protocol Amendment 3, Version 4.0, dated 22 April , the International 
Council for Harmonisation harmonised tripartite guideline E6(R2): Good Clinical Practice 
and all applicable government regulations. I will not make changes to the protocol before 
consulting with Oscotec Inc. or implement protocol changes without independent ethics 
committee approval except to eliminate an immediate risk to subjects. I agree to administer 
study drug only to subjects under my personal supervision or the supervision of a 
subinvestigator. 
I will not supply the investigational drug to any person not authorized to receive it. 
Confidentiality will be protected. Subject identity will not be disclosed to third parties or 
appear in any study reports or publications. 
I will not disclose information regarding this clinical investigation or publish results of the 
Page 6

Oscotec Inc. 
SKI-O-703 
Protocol: OSCO-P2101 Version 4.0 (Amendment 3) 
22 April 20 
Page 7 
Table of Contents 
Table of Contents .......................................................................................................................7 
List of Tables ............................................................................................................................ 11 
List of Figures .......................................................................................................................... 11 
Protocol Synopsis.....................................................................................................................12 
List of Abbreviations................................................................................................................23 
1 
Introduction ......................................................................................................................25 
1.1 
Clinical Studies .......................................................................................................26 
1.2 
Nonclinical Studies .................................................................................................27 
1.3 
Dose Rationale ........................................................................................................29 
1.4 
Placebo Rationale....................................................................................................31 
1.5 
Benefits and Risks Assessments .............................................................................31 
2 
Study Objectives...............................................................................................................32 
2.1 
Primary Objective ...................................................................................................32 
2.2 
Secondary Objectives..............................................................................................32 
2.3 
Exploratory Objectives ...........................................................................................32 
3 
Investigational Plan ..........................................................................................................33 
3.1 
Study Design ...........................................................................................................33 
3.1.1 
Dose Interruption and Reduction .............................................................36 
3.1.2 
Rationale of Study Design .......................................................................38 
4 
Subject Selection and Withdrawal Criteria ......................................................................40 
4.1 
Selection of Study Population .................................................................................40 
4.1.1 
Inclusion Criteria .....................................................................................40 
4.1.2 
Exclusion Criteria ....................................................................................42 
4.2 
Withdrawal of Subjects From Study Drug and/or the Study ..................................44 
4.2.1 
Reasons for Withdrawal/Discontinuation ................................................44 
4.2.2 
Handling of Withdrawals .........................................................................45 
4.2.3 
Replacements ...........................................................................................46 
5 
Study drugs .......................................................................................................................47 

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5.1 
Method of Assigning Subjects to Treatment Groups ..............................................47 
5.2 
Treatments Administered ........................................................................................47 
5.3 
Identity of Investigational Product..........................................................................48 
5.4 
Management of Clinical Supplies ...........................................................................48 
5.4.1 
Study Drug Packaging and Storage .........................................................48 
5.4.2 
Test Article Accountability ......................................................................49 
5.5 
Overdose Management ...........................................................................................49 
5.6 
Blinding ..................................................................................................................50 
5.6.1 
Breaking the Blind ...................................................................................50 
5.7 
Treatment Compliance ............................................................................................51 
5.8 
Prior and Concomitant Therapy ..............................................................................51 
5.8.1 
Rescue Medications .................................................................................51 
5.8.2 
Management of Nausea and Vomiting .....................................................52 
5.8.3 
Prohibited Concomitant Medications ......................................................52 
6 
Study Assessments and Procedures ..................................................................................54 
6.1 
Study Visits .............................................................................................................54 
6.2 
Demographic and Medical History .........................................................................58 
6.3 
Efficacy Assessments ..............................................................................................58 
6.4 
Safety Assessments .................................................................................................58 
6.4.1 
Physical Examination ..............................................................................58 
6.4.2 
Vital Sign Measurements, Height, and Weight ........................................59 
6.4.3 
Electrocardiograms ..................................................................................59 
6.4.4 
Clinical Laboratory Tests .........................................................................60 
6.4.5 
Adverse Events ........................................................................................61 
6.4.5.1 
Definitions of Adverse Events .................................................................61 
6.4.5.2 
Serious Adverse Events ...........................................................................62 
6.4.5.3 
Eliciting and Documenting Adverse Events ............................................62 
6.4.5.4 
Reporting Adverse Events .......................................................................63 
6.4.5.5 
Reporting Serious Adverse Events ..........................................................63 
6.4.5.6 
Suspected Unexpected Serious Adverse Reactions and Nonserious 
Adverse Events of Special Interest ..........................................................64 
6.4.5.7 
Assessment of Severity ............................................................................65 
6.4.5.8 
Assessment of Causality ..........................................................................65 

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6.4.5.9 
Follow-Up of Subjects Reporting Adverse Events ..................................65 
6.5 
Pharmacokinetic and Pharmacodynamic Assessments ...........................................66 
6.5.1 
Pharmacokinetic Blood Samples .............................................................66 
6.5.2 
Pharmacokinetic Variables .......................................................................67 
6.5.3 
Pharmacodynamic Blood Samples ..........................................................67 
6.5.4 
Pharmacodynamic Variables ....................................................................68 
6.6 
Safety Monitoring Committee ................................................................................68 
6.7 
Pregnancy ................................................................................................................68 
6.8 
Laboratory Analyses ...............................................................................................69 
6.9 
Sample Collections .................................................................................................69 
6.10 Pharmacogenomic Screening ..................................................................................69 
7 
Statistical and Analytical Plan ..........................................................................................70 
7.1 
Primary Endpoint - Platelet Response ....................................................................70 
7.2 
Secondary Endpoints ..............................................................................................70 
7.2.1 
Subjects Achieving Two or More Pre-specified Platelet Counts .............70 
7.2.2 
Change from Baseline in Platelet Counts ................................................70 
7.2.3 
Time to First Platelet Response ...............................................................70 
7.2.4 
Others .......................................................................................................71 
7.3 
Exploratory Endpoints ............................................................................................71 
7.3.1 
Peak Platelet Count ..................................................................................71 
7.3.2 
Bleeding Score .........................................................................................71 
7.3.3 
Quality of Life as Measured by the SF-36 Score ....................................72 
7.4 
Sample Size Calculations ........................................................................................72 
7.5 
Analysis Sets ...........................................................................................................73 
7.6 
Description of Subgroups to be Analyzed ..............................................................74 
7.7 
Statistical Analysis Methodology ...........................................................................74 
7.7.1 
Subject Disposition ..................................................................................74 
7.7.2 
Analyses of Demographic and Baseline Characteristics .........................75 
7.7.3 
Analysis of Platelet Response ..................................................................75 
7.7.4 
Analysis of Secondary Endpoints ............................................................75 
7.7.5 
Analyses of Exploratory Endpoints .........................................................76 
7.7.6 
Pharmacokinetic/Pharmacodynamic Analyses ........................................77 

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7.7.6.1 
Pharmacokinetic Analyses .......................................................................77 
7.7.6.2 
Pharmacodynamic Analyses ....................................................................77 
7.7.6.3 
Exploratory PK/PD Analyses ..................................................................77 
7.7.7 
Safety Analyses ........................................................................................78 
7.7.8 
Interim Analyses ......................................................................................78 
8 
Data Quality Assurance ....................................................................................................79 
8.1 
Data Management ...................................................................................................79 
9 
Ethics ................................................................................................................................80 
9.1 
Independent Ethics Committee or Institutional Review Board ..............................80 
9.2 
Ethical Conduct of the Study ..................................................................................80 
9.3 
Subject Information and Consent ...........................................................................80 
10 Investigator’s Obligations ................................................................................................82 
10.1 Confidentiality ........................................................................................................82 
10.2 Financial Disclosure and Obligations .....................................................................82 
10.3 Investigator Documentation ....................................................................................83 
10.4 Study Conduct .........................................................................................................83 
10.5 Adherence to Protocol ............................................................................................83 
10.6 Adverse Events and Study Report Requirements ...................................................84 
10.7 Investigator’s Final Report .....................................................................................84 
10.8 Records Retention ...................................................................................................84 
10.9 Publications .............................................................................................................84 
11 Study Management ...........................................................................................................85 
11.1 Monitoring ..............................................................................................................85 
11.1.1 
External Data Monitoring Committee .....................................................85 
11.1.2 
Monitoring of the Study ...........................................................................85 
11.1.3 
Inspection of Records ..............................................................................85 
11.2 Management of Protocol Amendments and Deviations..........................................86 
11.2.1 
Modification of the Protocol ....................................................................86 
11.2.2 
Protocol Deviations .................................................................................86 
11.3 Study Termination ...................................................................................................87 
11.4 Final Report ............................................................................................................87 

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Protocol: OSCO-P2101 Version 4.0 (Amendment 3) 
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12 Reference List...................................................................................................................88 
13 Appendices .......................................................................................................................90 
13.1 Appendix: Partial List of Excluded Drugs Known to be Cytochrome P (CYP)1A2 
and UGT1A1 Inhibitors or Inducers ...............................................................90 
13.2 Protocol Amendment ..............................................................................................90 
13.2.1 
Protocol Amendment 1 - Protocol Version 2.0, Dated 03 April 2019 .....90 
13.2.1.1 Overview of Changes ..............................................................................90 
13.2.1.2 Changes to the Protocol Text ...................................................................91 
13.2.2 
Protocol Amendment 2 - Protocol Version 3.0, Dated 30 April 2019 ...109 
13.2.2.1 Overview of Changes ............................................................................109 
13.2.2.2 Changes to the Protocol Text .................................................................109 
13.2.3 
Country-specific Protocol Amendment 2.1 (Republic of Korea), Version 
3.0, Dated 09 August 2019 ....................................................................122 
13.2.3.1 Overview of Changes ............................................................................122 
13.2.3.2 Changes to the Protocol Text .................................................................122 
13.2.4 
Protocol Amendment 3 - Protocol Version 4.0, Dated 22 April 2020 ...125 
13.2.4.1 Overview of Changes ............................................................................125 
13.2.4.2 Changes to the Protocol Text .................................................................126 
 
List of Tables 
Table 1 
Guidelines for Dose Interruption or Reductiona ............................................. 37 
Table 2 
Schedule of Events .......................................................................................... 55 
Table 3 
The ITP Bleeding Score Assessment .............................................................. 71 
 
List of Figures 
Figure 1 
Study Design Schematic (for the First 5 Subjects in the Safety Run-in Period 
only) ................................................................................................................ 34 
Figure 2 
Study Design Schematic (For Subjects Other Than Enrolled in the Safety 
Run-in Period) ................................................................................................. 35 
 

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Protocol: OSCO-P2101 Version 4.0 (Amendment 3) 
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Protocol Synopsis 
Protocol Number: 
OSCO-P2101 
Title: 
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-
Controlled, Parallel Dose Study to Evaluate the Efficacy and Safety 
of Oral SKI-O-703, SYK Inhibitor, in Patients with Persistent and 
Chronic Immune Thrombocytopenia (ITP) 
Sponsor: 
Oscotec Inc. 
Korea Bio-Park, Building A, 9th Floor 
700 Daewangpangyo-ro, Bundang-gu 
Seongnam-si, Gyeonggi-do 13488 
The Republic of Korea 
Study Phase: 
Phase 2 
Study Sites: 
This study will be conducted at multiple sites in Korea, US, and EU. 
Indication: 
Immune Thrombocytopenia (ITP) 
Rationale: 
Primary ITP is an acquired immune mediated disorder characterized 
by isolated thrombocytopenia, defined as a peripheral blood platelet 
count less than 100,000/µL. The ITP is classified by duration into 
newly diagnosed (0-3 months), persistent (3-12 months’ duration) and 
chronic (≥12 months’ duration). 
Spleen tyrosine kinase (SYK) is a non-receptor kinase and an 
inflammatory signal mediator. SYK activation has been known to be 
involved in various autoimmune disorders. Since SYK is a key 
mediator of FcγR-triggered intracellular signal in various 
inflammatory immune cells including macrophages, it was proposed 
as a promising therapeutic target to treat ITP. 
A novel small-molecule SYK inhibitor, SKI-O-703, mesylate salt 
form of SKI-O-592, was designed and synthesized by Oscotec Inc. 
SKI-O-703 displayed high selectivity and potency against SYK in 
both biochemical and cell-based assays. Selective SYK inhibition by 
SKI-O-592 prevents FcγR mediated phagocytosis in vitro and 
downregulates CD63 expression on peripheral blood basophils in 
healthy adults. 
SKI-O-703 was efficacious in preventing thrombocytopenia induction 
in an anti-CD41 antibody induced murine ITP model. Intraperitoneal 
injection of anti-CD41 (integrin αIIb) antibody successfully induced 
immediate decrease of platelets within 24 hours. The reduced platelet 
numbers were normalized in 72 hours after αCD41 antibody 
injection. Both once daily and twice daily (BID) treatment of SKI-O-
703 successfully ameliorated the thrombocytopenia in a dose 
dependent manner compared to vehicle treatment. The high dose 

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Protocol: OSCO-P2101 Version 4.0 (Amendment 3) 
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group of SKI-O-703 (42 mg/kg, equivalent to 30 mg/kg of 
SKI-O-592) showed better protective effect against thrombocytopenia 
induction than R788 (40 mg/kg, equivalent to 30 mg/kg of R406, the 
active compound of prodrug R788). Moreover, the low dose group of 
SKI-O-703 (14 mg/kg, equivalent to 10 mg of SKI-O-592) showed 
comparable efficacy to the high dose group of R788 (40 mg/kg). 
This study will evaluate the efficacy, safety, tolerability, 
pharmacokinetics (PK), and pharmacodynamics (PD) of select 
(200 mg BID and 400 mg BID) doses of SKI-O-703 in persistent and 
chronic ITP patients who have failed to respond or relapsed after 
prior therapy, with a platelet count <30,000/µL. 
Since the 400 mg BID dose level has not been investigated in healthy 
volunteer studies, a safety run-in group will be implemented (ie, first 
5 subjects) and an interim safety and PK data review will be 
performed by the DMC before remaining subjects are enrolled. 
Objectives: 
Primary Objective 
• To evaluate the safety and efficacy on the primary endpoint 
(platelet response) of select (200 mg BID and 400 mg BID) 
doses of SKI-O-703 compared to placebo in patients with 
persistent and chronic ITP, with a platelet count <30,000/µL 
on 2 occasions at least 7 days apart with the confirmatory 
count being taken during Screening. 
Secondary Objectives 
• To evaluate the efficacy on the secondary endpoints of select 
(200 mg BID and 400 mg BID) doses of SKI-O-703 
compared to placebo in patients with persistent and chronic 
ITP 
• To investigate the PK profile of SKI-O-592 (the free base of 
SKI-O-703) and its metabolites (M2 and M4) in patients with 
persistent and chronic ITP 
• To evaluate the effects of SKI-O-703 on PD biomarkers in 
patients with persistent and chronic ITP. 
Exploratory Objectives 
• To evaluate the PK/PD relationship between plasma 
concentrations of SKI-O-592, M2, and M4 and the percent 
change in activated CD63+ basophils in peripheral blood in 
patients with persistent and chronic ITP 
• To evaluate the changes of the exploratory endpoints of select 
(200 mg BID and 400 mg BID) doses of SKI-O-703 
compared to placebo. 

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Protocol: OSCO-P2101 Version 4.0 (Amendment 3) 
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Study Population: 
Inclusion Criteria 
Each subject must meet all the following criteria to be enrolled in this 
study: 
a. 
Able to provide written informed consent and agreeable to the 
schedule of assessments 
b. 
Male or female subjects, aged 18 years or older (or ≥19 years 
of age for Korean subjects per local regulations) 
c. 
Diagnosis of primary ITP (persistent or chronic) made 
according to the American Society of Hematology 2011 
evidence-based guideline 
d. 
Failed to respond or relapsed after at least 1 prior therapy (eg, 
corticosteroids, IVIG, anti-D, rituximab, TPO-receptor 
agonists, splenectomy, and immunosuppressants such as 
vincristine, mycophenolate mofetil, and azathioprine), with a 
platelet count of <30,000/µL on 2 occasions at least 7 days 
apart with the confirmatory count being taken during 
Screening (the most recent result from the central laboratory 
will be considered as the confirmatory platelet count, when an 
unscheduled visit(s) is performed during the screening 
period). Response to such prior therapy is defined as platelet 
count of ≥30,000/µL and >2 times increase from baseline in 
the absence of bleeding. Failure to respond and relapse to 
such prior therapy are defined as failure to achieve and/or 
failure to maintain response on a tolerable regimen of prior 
therapy. 
e. Adequate hematologic, hepatic, and renal function (absolute 
neutrophil count ≥ 1.5 x 109/L, hemoglobin 
[Hgb] ≥ 10.0 g/dL, aspartate aminotransferase [AST] and 
alanine aminotransferase [ALT] ≤ 2.0 x ULN, bilirubin 
≤1.5 x ULN, albumin ≥ 3 g/dL, estimated glomerular filtration 
rate [eGFR] ≥40 mL/min/1.73m2, and creatinine ≤ 1.5 x ULN) 
f. 
ECOG performance status of 0, 1, or 2 
g. 
Both male and female subjects must agree to take the 
following steps to reduce the potential for transmission of 
genetic material containing the investigational product: 
a. Both male and female subjects, the subject and their 
partners of childbearing potential must agree to use 2 of 
the following medically acceptable methods of 
contraception from the time of randomization, during the 

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study, and for 6 months following discontinuation of study 
drug, of which: 
o One must be a highly reliable method of 
contraception, such as: 
 An intrauterine device or intrauterine 
system implanted for at least 30 days prior 
to Day 1  
 Surgical sterilization of one of the partners 
for at least 6 months prior to the date of 
informed consent (assuming this will be the 
only partner for the whole duration of the 
clinical trial) 
 Consistent and correct use of hormonal 
contraceptives (hormonal implants, 
injectables, contraception pills, transdermal 
patches, or contraceptive rings) for at least 
30 days prior to Day 1 
o One supplementary barrier method, such as: 
 Male or female condom always with 
spermicide (a spermicidal 
foam/gel/film/cream)  
 Diaphragm or cervical/vault caps always 
with spermicide (a spermicidal 
foam/gel/film/cream) 
 Double-barrier methods (which means a 
barrier method used by both partners at the 
same time), even when used with 
spermicide, are not considered to be highly 
reliable contraception methods, and as 
such, may not be the only forms of 
contraception used  

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One of the other listed highly reliable methods 
must be used in conjunction with a barrier 
method. 
b. Female subjects must agree not to breastfeed starting from 
the time of Screening, throughout the study, and until after 
6 months following the last dose of study drug. 
c. Male subjects must agree not to donate sperm starting 
from the time of randomization, throughout the study, and 
until after 6 months following the last dose of study drug  
d. For subjects and partners considered not of childbearing 
potential, the following conditions apply: 
a. Menopausal females must have experienced their 
last period more than 12 months prior to the date 
of informed consent to be classified as not of 
childbearing potential. 
b. Male and female subjects are in a situation of 
abstinence from heterosexual intercourse from 
Screening until after 6 months following the last 
dose of study drug when this is in line with the 
preferred lifestyle of the subject (eg, homosexual 
women and men or a member of a religious order 
such as nuns and priests). 
Exclusion Criteria 
Subjects meeting any of the following criteria will be excluded from 
the study: 
1. 
History of any clinically significant disease or disorder that in 
the opinion of the investigator or the sponsor may put the 
subject at risk due to study participation, impact the subject’s 
ability to participate in the study, or influence the study results 
2. 
History of current, active malignancy requiring or likely to 
require chemotherapeutic or surgical treatment during the 
study, with the exception of non-melanoma skin cancer, 

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carcinoma in situ of the cervix, and localized prostate cancer 
managed by active surveillance 
3. 
Transfusion with blood or blood products or plasmapheresis 
within 2 weeks before the first administration of study drug 
(Day 1) 
4. 
PT INR > 1.5 
5. 
History of known inherited coagulopathy (including 
prothrombotic conditions such as Factor V Leiden, APC 
resistance, AT-III deficiency, and lupus anticoagulant), or 
recent arterial or deep venous thrombosis within the preceding 
6 months 
6. 
Change in corticosteroid or immunosuppressant (azathioprine, 
mycophenolate mofetil, cyclosporine) dose within 2 weeks 
prior to Day 1 (more than 10% variation from Day 1 daily 
doses) 
7. 
Treatment with thrombopoietin (TPO) receptor agonists 
within 2 weeks before Day 1 
8. 
Treatment with rituximab or splenectomy within the 8 weeks 
prior to Day 1 
9. 
Treatment with intravenous immunoglobulins (IVIGs) within 
4 weeks prior to Day 1 
10. Infections requiring intravenous antibiotics or hospitalization 
within 3 months prior to Day 1 
11. Subjects had positive test results at Screening for human 
immunodeficiency virus, hepatitis B surface antigen (HBsAg), 
or hepatitis C virus antibody. Subjects with past or resolved 
hepatitis B infection (defined as having a negative HBsAg test 
and a positive IgG antibody to hepatitis B core antigen 
[anti-HBc]) are eligible but hepatitis B virus (HBV) DNA 
must be obtained in these subjects prior to Cycle 1, Day 1, 
and must demonstrate no active infection. Subjects positive 
for hepatitis C virus (HCV) antibody are eligible only if 
polymerase chain reaction (PCR) is negative for HCV RNA.  
12. Received live vaccine within 28 days prior to Day 1 or plan to 
receive one during the study 
13. History or presence of any gastrointestinal, hepatic, or renal 
disease or any other condition known to interfere with the 
absorption, distribution, metabolism, or excretion of drugs 
14. Subject has not recovered from a recent medical/surgical 
procedure or trauma by Day 1 as determined by the 
investigator 

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15. Uncontrolled hypertension (as defined by systolic blood 
pressure ≥160 mmHg or diastolic blood pressure 
≥100 mmHg) 
16. Subject had 12-lead electrocardiogram (ECG) findings of 
corrected QT interval by Fridericia formula (QTcF) > 480 
msec, cardiac arrhythmias, or clinically significant cardiac or 
ECG abnormalities 
17. Subject received any investigational medication within 
30 days or 5 half-lives prior to Day 1, whichever is longer 
18. Concomitant use of any anticoagulants and platelet 
aggregation inhibiting drugs including aspirin (within 14 days 
of planned dosing through end of follow-up) 
19. Female subject who is currently pregnant or breastfeeding 
20. Prior treatment with a SYK inhibitor 
21. Planned surgery in the time frame of the dosing period. 
Study Design: 
This is a randomized, double-blind, multicenter, placebo-controlled, 
parallel dose study to evaluate the efficacy, safety, tolerability, PK, 
and PD of select (200 mg BID and 400 mg BID) doses of SKI-O-703 
in persistent and chronic ITP patients who have failed to respond or 
relapsed after prior therapy, with a platelet count <30,000/µL on 
2 occasions at least 7 days apart with the confirmatory count being 
taken during Screening (the most recent result from the central 
laboratory will be considered as the confirmatory platelet count, when 
an unscheduled visit(s) is performed during the screening period). 
A minimum of 60 subjects are planned to participate in 3 treatment 
groups (24 subjects in each of the active treatment groups and 
12 subjects in the placebo group). Subjects will be randomly assigned 
to 3 groups using a 2:2:1 ratio to receive 1 of the 2 select doses of 
SKI-O-703 or placebo. First 5 subjects randomly assigned in the 
study will have safety run-in period for the first 1 week with interim 
safety and PK assessments on Day 8 (±2 days). Subjects in safety 
run-in period will continue to be dosed whilst the interim analysis is 
being conducted. However, further enrollment in each treatment 
group will only resume after safety and PK data of the first week for 
the first 5 subjects are reviewed and considered satisfactory by the 
Data Monitoring Committee (DMC). A subset of at least 20 subjects, 
who are not included in the safety run-in period, will be randomized 
to undergo PK/PD assessments at a selected number of sites. 
Randomization will be stratified to balance subjects with prior 
TPO-receptor agonist use (yes/no) and degree of baseline 
thrombocytopenia (ie, baseline platelet count < or ≥15,000/µL). 

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The study will include a screening period of up to 4 weeks. After 
completing all screening assessments, subjects who meet all the 
inclusion and none of the exclusion criteria will proceed to the 
treatment period. 
On Day 1, subject’s eligibility will be confirmed and baseline safety 
evaluations will be performed (ECG, physical examination, vital sign 
measurements, and safety laboratory assessment). Then subjects will 
be randomized to receive either one of the doses of SKI-O-703 or 
placebo. The subjects will be administered their morning dose of 
study medication. The evening dose will be administered 12 hours 
after the morning dose. Subjects will be administered study 
medication with enough water to swallow the capsules. The study 
medication will be taken no later than 30 minutes after food. 
After dosing, all subjects will be subjected to safety assessments and 
in addition, subjects in the safety run-in period will be subjected to 
PK assessments and a subset of at least 20 subjects will be subjected 
to PK and PD assessments. The subject will be instructed to 
self-administer the subsequent doses of the study medication in the 
morning and evening (12 hours after the morning dose) for the 
remaining study duration. 
Estimated Study 
Duration: 
The total study duration will be 20 weeks per subject, which consists 
of up to 4 weeks of screening period, 12 weeks of treatment period 
(split into 1-week safety run-in period and 11-week treatment period 
for the first 5 subjects), and 4 weeks of follow-up period. 
Efficacy 
Assessments: 
Primary Endpoint: 
• Patient platelet response is defined as platelet count 
≥ 30,000/µL and doubling the baseline (average of 2 previous 
counts), at any visit during the treatment period and without 
use of rescue medication. 
Secondary Endpoints: 
• Subjects able to achieve 2 or more consecutive platelet counts 
of ≥ 30,000/µL separated by at least 5 days and without use of 
rescue medication 
• Subjects able to achieve 2 or more consecutive platelet counts 
of ≥ 30,000/µL with an increase of platelet count of 
≥20,000/µL from baseline separated by at least 5 days and 
without use of rescue medication  
• Subjects able to achieve 2 or more consecutive platelet counts 
of ≥ 50,000/µL separated by at least 5 days and without use of 
rescue medication 

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• Subjects able to achieve 2 or more consecutive platelet counts 
of ≥ 100,000/µL during the treatment period, separated by at 
least 5 days and without use of rescue medication 
• Subjects able to achieve platelet counts of ≥ 50,000/µL at 3 of 
the last 4 visits 
• Change from baseline in the average of the last 2 platelet 
counts during the treatment period 
• Time to first platelet response (>30,000/µL and 2 x baseline) 
• Number of subjects receiving rescue medication at least once 
during treatment period 
• Subjects with at least one Grade 2 or higher non-skin bleeding 
event during treatment period. 
Exploratory Endpoints: 
• Peak platelet counts during the treatment period without use 
of rescue medication 
• Bleeding score at select time points 
• Quality of life as measured by the SF-36 score. 
Pharmacokinetic 
Assessments: 
Plasma PK parameters will be calculated for SKI-O-592 and its 
metabolites (M2 and M4) in all subjects in the PK/PD subset on 
Day 1 and Week 12 using a noncompartmental approach and will 
include the following: Cmax, Tmax, AUC0-tau, t1/2, Kel, CL/F 
(SKI-O-592 only), Vz/F (SKI-O-592 only), Rmet, and RAUC, as 
applicable. Pharmacokinetic assessments will also be performed on 
Day 8 (±2 days) for subjects in the safety run-in period only. 
Pharmacodynamic 
Assessments: 
Serial blood samples for assessment of the percentage of activated 
CD63+ basophils in peripheral blood will be collected in all subjects 
in the PK/PD subset on Day 1 and Week 12. PD parameters will 
include the following: Emax, TEmax, AUEC0-tau, as applicable. 
Exploratory Endpoint 
An assessment of PK/PD relationship between plasma concentrations 
of SKI-O-592, M2 and M4 and change from baseline in the 
percentage of activated CD63+ basophils in peripheral blood on 
Day 1 and Week 12 
Safety Assessments: Safety variables will include physical examination findings, vital sign 
measurements (systolic and diastolic blood pressure, pulse rate, 
respiratory rate, and temperature), ECG tracings, clinical laboratory 
test results (hematology, coagulation, serum chemistry, and 
urinalysis), bleeding score, quality of life as measured by the SF-36 
score, weight, body mass index, and reporting of adverse events. 

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Safety monitoring will begin when subject signs the informed consent 
form and continue through the end-of-study visit (Week 16). 
Study Drug, 
Dosage, and Route 
of Administration: 
SKI-O-703 and placebo will be administered orally. Two dose levels 
of SKI-O-703 will be evaluated in separate groups: 200 mg BID and 
400 mg BID. Third group will be of matching placebo. 
Sample Size: 
A minimum of 60 subjects will be enrolled in the study. Subjects will 
receive either 200 mg BID or 400 mg BID of SKI-O-703 or placebo 
in a 2:2:1 ratio. 
Based on Podolanczuk et al (2009) at least 50% of subjects in the 
active treatment group will be responders (R). A sample size of 
24 subjects per active treatment group and 12 subjects in the placebo 
group will provide approximately 82.7% power to detect a difference 
between response rates of 50% and 1% in the active treatment and 
placebo groups, respectively, at the 2-sided 5% significance level. 
The test statistic used in the sample size calculation was the 2-sided 
Fisher's Exact test using the normal approximation method. 
Subjects that do not receive the study drug or do not provide at least 1 
post baseline measurement will be excluded from the analysis above 
and replaced. 
The sample size calculation was performed using PASS 15 Power 
Analysis and Sample Size Software (2017). NCSS, LLC. Kaysville, 
Utah, USA, ncss.com/software/pass. 
Statistical Methods: Statistical analysis will be performed using SAS software Version 9.4 
or later. The results for each efficacy endpoint will be summarized by 
visit and treatment for all subjects within the intent-to-treat (ITT) 
analysis set using descriptive statistical methods. Continuous 
variables (quantitative endpoints) will be summarized using the 
mean, the standard deviation, median, range (minimum value and 
maximum value), and interquartile. Categorical variables (qualitative 
endpoints) will be summarized using frequency counts (number) and 
percentages. Data will be listed in data listings. 
The primary efficacy endpoint will be summarized as overall platelet 
response rate (ORR) by treatment group and analyzed using Fisher’s 
exact test to compare the difference in ORR between each active 
treatment group and placebo. A logistic regression model will also be 
performed on platelet response and include treatment, baseline 
platelet count and prior TPO-receptor agonist, provided there are 
enough subjects in each subgroup. The odds ratio for the treatment 
effect compared to placebo (together with 95% confidence interval 
and p-value) will be provided. The analysis will be based on the ITT 
analysis set. 

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All statistical tests will be 2-sided and performed using a 5% 
significance levels, leading to 95% (2-sided) confidence intervals. 
There will be no adjustments for multiplicity. 
Version and Date of 
Protocol: 
4.0 (Protocol Amendment 3); 22 April 2020 

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List of Abbreviations 
Abbreviation 
Definition 
AE 
adverse event 
AESI 
adverse event of special interest 
ALT 
alanine aminotransferase 
Anti-HBc 
hepatitis B core antigen 
AST 
aspartate aminotransferase 
AUC 
area under the concentration-time curve  
BID 
Twice daily 
CD-ROM 
compact disk read-only memory 
CFR 
Code of Federal Regulations 
CI(s) 
confidence interval(s) 
Cmax 
maximum observed concentration 
CV 
coefficient of variation 
ECG 
12-lead electrocardiogram 
eCRF 
electronic case report form 
FcγR 
Fcγ receptor 
FDA 
US Food and Drug Administration 
GCP 
Good Clinical Practice 
HBsAg 
hepatitis B surface antigen 
HCV 
hepatitis C virus 
ICF 
informed consent form 
ICH 
International Council for Harmonisation 
IEC 
Independent Ethics Committee 
IRB 
Institutional Review Board 
ITP 
immune thrombocytopenia 
ITT 
Intent-to-treat 
IVIG 
intravenous immunoglobulin 
IVRS 
interactive voice response system 
MedDRA 
Medical Dictionary for Regulatory Activities 
NOAEL 
no observed adverse effect level 
ORR 
overall platelet response rate 
PCR 
polymerase chain reaction 

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Abbreviation 
Definition 
PD 
pharmacodynamic 
PK 
pharmacokinetic 
QD 
once daily 
QTcF 
corrected QT interval by Fridericia formula 
SAE 
serious adverse event 
SD 
standard deviation 
TEAE 
treatment-emergent adverse event 
TPO 
thrombopoietin 
ULN 
upper limit of normal 
 

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1 
Introduction 
Primary immune thrombocytopenia (ITP) is an acquired immune mediated disorder 
characterized by isolated thrombocytopenia, defined as a peripheral blood platelet count less 
than 100,000/µL. ITP is classified by duration into newly diagnosed (0-3 months), persistent 
(3-12 months’ duration) and chronic (≥12 months’ duration) (Mitchell et al 2013). The 
estimated incidence is 100 cases per 1 million persons per year, and about half of these cases 
occur in children. Patients with ITP have accelerated clearance of circulating 
immunoglobulin G-coated platelets via Fcγ receptor (FcγR)–bearing macrophages in the 
spleen and liver, leading to different levels of thrombocytopenia and variable degrees of 
mucocutaneous bleeding (Kessler et al 2017, Podolanczuk et al 2009). 
Spleen tyrosine kinase (SYK) is a non-receptor kinase and an inflammatory signal mediator. 
A SYK activation has been known to be involved in various autoimmune disorders. Spleen 
tyrosine kinase is broadly involved in regulating leukocyte immune function, principally by 
facilitating cellular activation in response to receptor engagement of antigen or immune 
complex. Receptors that use SYK for signal transduction include the B-cell antigen receptor, 
Fc receptors, integrins, and members of the lectin and selectin families (Turner et al 2000, 
Mócsai et al 2002, Rogers et al 2005, and Zarbock et al 2008). 
A novel small-molecule SYK inhibitor, SKI-
O-703, mesylate salt form of SKI-O-592, was designed and synthesized by Oscotec Inc. SKI-
O-703 displayed high selectivity and potency against SYK in both biochemical and cell-
based assays. Selective SYK inhibition by SKI-O-592 prevents FcγR mediated phagocytosis 
in vitro and downregulates CD63 expression on peripheral blood basophils in healthy adults. 
Many patients exhibit responses to established therapies, including corticosteroids, 
intravenous immunoglobulin (IVIG), anti-D, splenectomy, and rituximab. Nonetheless, there 
are a significant minority of patients who retain persistently low platelet counts despite 
treatment. They consequently remain at risk of intracranial hemorrhage as well as other 
bleeding complications (Podolanczuk et al 2009). 

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Since SYK is a key mediator of FcγR-triggered intracellular signal in various inflammatory 
immune cells including macrophages, it was proposed as a promising therapeutic target to 
treat ITP. 
1.1 
Clinical Studies 
The safety, pharmacokinetics (PK), and pharmacodynamics (PD) of SKI-O-703 have been 
evaluated in 2 clinical studies, a single ascending dose clinical study (OSCO-P1201) and a 
multiple ascending dose clinical study (OSCO-P1202) in healthy subjects. 
In Study OSCO-P1201, single oral doses of SKI-O-703 ranging from 50 to 800 mg were 
generally safe and well tolerated by the healthy male and female subjects. There were no 
deaths, serious adverse events (SAEs), or treatment-emergent adverse events (TEAEs) that 
led to discontinuation of the study. Ten of 48 subjects (20.8%) reported a total of 18 TEAEs; 
of these, 3 TEAEs were reported by 3 subjects (25.0%) after receiving placebo and 
15 TEAEs were reported by 7 subjects (19.4%) in the SKI-O-703 treatment groups. There 
were no dose-dependent trends noted in the TEAEs reported. The most commonly reported 
TEAE was headache, which was reported by 3 subjects (8.3%) who received SKI-O-703 and 
1 subject (8.3%) who received placebo. All TEAEs across the 50 mg to 600 mg dose range 
were considered mild in intensity; 6 treatment-related moderate-intensity TEAEs, reported in 
2 subjects, occurred at the highest dose of 800 mg SKI-O-703. All TEAEs were resolved by 
the end of the study. All individual hematology and coagulation, serum chemistry, and 
urinalysis values outside of the reference ranges were considered not clinically significant by 
the investigator. No TEAEs related to laboratory parameters were reported. 
In Study OSCO-P1202, multiple oral doses of SKI-O-703 200 mg once daily (QD), 400 mg 
QD, and 200 mg twice daily (BID) were safe and well tolerated by the healthy male and 
female subjects. There were no deaths or SAEs; however, 1 subject reported TEAE of back 
pain that led to discontinuation of 200 mg SKI-O-703 BID. Overall, 7 subjects (29.2%) 
reported a total of 17 TEAEs; of these, 2 subjects (33.3%) reported 6 TEAEs after receiving 
placebo and 5 subjects (27.8%) reported 11 TEAEs after receiving SKI-O-703. The most 
commonly reported TEAEs were nausea and headache, each were reported by 2 subjects 
(33.3%) in the 200 mg SKI-O-703 BID treatment group and by 1 subject (16.7%) in the 
placebo group. All TEAEs were considered mild or moderate in intensity, and both moderate 
TEAEs occurred in the placebo group. Except for 1 subject who reported ongoing TEAEs of 
pyuria and chlamydial infection, all TEAEs were resolved by the end of the study. No 

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apparent treatment- or dose-related trends were observed in clinical laboratory values, 
physical examination findings, or 12-lead electrocardiogram (ECG) results. There was a 
slight trend in the mean increase of systolic blood pressure from baseline in the 200 mg BID 
group at Days 16 and 21, and diastolic blood pressure values at Days 16 and 21 also 
demonstrated a slight trend of a mean increase for all doses of SKI-O-703. There were no 
significant changes in systolic and diastolic blood pressure for all doses of SKI-O-703 versus 
placebo at Days 16 and 21. 
1.2 Nonclinical Studies 

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1.3 Dose Rationale 
SKI-O-703 was safe and well-tolerated in healthy volunteers when administered as single 
ascending doses over the dose range of 50 to 800 mg (Study OSCO-P1201), and multiple 
ascending doses at 200 mg QD, 400 mg QD and 200 mg BID for 7 days 
(Study OSCO-P1202). Based on the safety data from the various doses administered in these 
studies, there are no specific considerations that would concern the use of SKI-O-703 at 
doses proposed in this Phase 2 study. 
Following multiple ascending doses of SKI-O-703 at 200 mg QD, 400 mg QD, and 
200 mg BID (Study OSCO-P1202), a reduction in the PD endpoint “percentage of basophils 
expressing CD63+” was observed in healthy volunteers; on average, CD63+ expressing 
basophils were suppressed by 95%, 99% and 86% at steady state (Day 7) following 
administration at 200 mg QD, 400 mg QD, and 200 mg BID, respectively. Thereafter, the 
suppression of basophils expressing CD63+ was maintained at –72% or below for up to 2 
and 8 hours following once-daily dosing at 200 mg and 400 mg, respectively, and up to 
12 hours (ie, over a full 24-hr period) following twice-daily dosing at 200 mg. As the PD 
effect was sustained for longer after twice-daily dosing compared to once-daily dosing, 
twice-daily dosing at 200 mg and 400 mg has been selected. 
As 400 mg BID has not been studied previously in humans, exposure (Cmax and AUC0-tau) to 
SKI-O-592, M2, and M4 following 400 mg BID dosing has been estimated based on data 
observed previously at different dose levels. To ensure that predictions are cautious and 

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robust, steady-state Cmax and AUC0-tau following 400 mg BID have been predicted in 2 ways 
using data from Study OSCO-P1202: 
1. AUC0-12 (400 mg, Day 1) × Accumulation Ratio (200 mg BID) 
2. PK Parameter (200 mg BID, Day 7) × 2 
Systemic exposure (Cmax and AUC0-tau) to SKI-O-592, M2, and M4 at 400 mg BID is 
predicted to be higher using the second approach [PK Parameter (200 mg BID, Day 7) × 2]. 
For SKI-O-592, Cmax and AUC0-tau are predicted to be 6160 ng/mL and 14600 h ng/mL, 
respectively, and for M2 Cmax and AUC0-tau are predicted to be 994 ng/mL and 
10000 h ng/mL, respectively, which is well within exposure levels calculated at the NOAEL 
in rats, and comparable to levels previously observed at 800 mg in Study OSCO-P1201. 
However, due to the very low M4 metabolite ratios observed in rats compared to healthy 
volunteers, predicted exposure to M4 (644 ng/mL and 1970 h ng/mL for Cmax and AUC0-tau, 
respectively) is higher than the NOAEL exposure. Therefore, the dosing regimens proposed 
for this study (200 mg BID and 400 mg BID) have been carefully selected to ensure that 
exposure to M4 does not exceed levels observed previously in healthy volunteers 
(Study OSCO-P1201, 800 mg). 
*Note: Because increases in systemic exposure to M4 were less than proportional to the dose 
of SKI-O-703 in Studies OSCO-P1201 and OSCO-P1202, and a reduction in M4 levels was 
observed over time in Study OSCO-P1202, both means of estimating Cmax and AUC0-tau for 
M4 at 400 mg BID are considered to be cautious approaches, potentially overestimating 
exposure at 400 mg BID. An interim safety and PK data review will be performed by the 
DMC for the first 5 subjects dosed (including 2 subjects dosed with 400 mg BID) before the 
remaining subjects are treated. 
Following single oral administration of 400 mg SKI-O-703 (Study OSCO-P1203), overall 
systemic exposure to SKI-O-592, M2 and M4 (AUC0-t and AUC0-inf) was comparable 
between the fed and fasted state, with 90% CI for the ratio (Fed / Fasted) contained entirely 
within the BE limits (0.80, 1.25) for SKI-O-592 and M4. By contrast, when administered in 
the fed state there was an approximate 56%, 43% and 46% reduction in maximum exposure 
(Cmax) for SKI-O-592, M2 and M4, respectively, with an associated 2.5 to 4 hours delay in 
median Tmax across all analytes. Since the overall extent of absorption is unaffected by the 
intake of food, but peak exposure is reduced, dosing is recommended with food in this 

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Phase 2 study to reduce the potential for AEs which may be associated with spikes (i.e. high 
Cmax) in SKI-O-592, M2 and M4. 
1.4 Placebo Rationale 
The proposed study is a double-blind, placebo-controlled, randomized trial of two doses of 
oral SKI-O-703 for patients with persistent and chronic ITP. Subjects will be allowed to 
receive stable doses of background medications including corticosteroids and 
immunosuppressive drugs, in line with the current standard of care. Any subject who cannot 
adhere to the protocol or who discontinues treatment will have an end-of-treatment visit and 
will be followed for the intended duration of the study. 
1.5 Benefits and Risks Assessments 
In the clinical development program, the safety, tolerability, and PK of SKI-O-703 have been 
evaluated in 2 clinical studies, a single ascending dose clinical study (OSCO-P1201) and a 
multiple ascending dose clinical study (OSCO-P1202) in healthy subjects. No deaths and 
SAEs were reported in these studies; however, 1 subject reported TEAE of back pain that led 
to discontinuation of SKI-O-703. All adverse events (AEs) were mild to moderate in 
intensity. 
As described in Section 1.2, SKI-O-703 was efficacious in preventing thrombocytopenia 
induction in an anti-CD41 antibody induced murine ITP model. 
There are no important identified risks with SKI-O-703. The identified potential risks 
associated with SKI-O-703 included immune system effects, gastrointestinal effects, liver 
enzyme changes, cardiovascular effects, and headache. 
Gastrointestinal effects and headache have been observed clinically, while immune system 
effects, liver enzyme changes, and cardiovascular effects are considered important potential 
risks based on non-clinical data and the SKI-O-703 mechanism of action. Considering the 
measures to minimize the risks to patients participating in the SKI-O-703 clinical 
development program, and the nonclinical and clinical evidence highlighted above, the 
benefits of the conduct of the proposed clinical study outweigh the associated risks. 
 

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2 
Study Objectives 
2.1 
Primary Objective 
To evaluate the safety and efficacy on the primary endpoint (platelet response) of select 
(200 mg BID and 400 mg BID) doses of SKI-O-703 compared to placebo in patients with 
persistent and chronic ITP, with a platelet count <30,000/µL on 2 occasions at least 7 days 
apart with the confirmatory count being taken during Screening. 
2.2 
Secondary Objectives 
The secondary objectives of this study are as below: 
• To evaluate the efficacy on the secondary endpoints of select (200 mg BID and 400 mg 
BID) doses of SKI-O-703 compared to placebo in patients with persistent and chronic 
ITP 
• To investigate the PK profile of SKI-O-592 (the free base of SKI-O-703) and its 
metabolites (M2 and M4) in patients with persistent and chronic ITP 
• To evaluate the effects of SKI-O-703 on PD biomarkers in patients with persistent and 
chronic ITP. 
2.3 
Exploratory Objectives 
The exploratory objectives of this study are as below: 
• To evaluate the PK/PD relationship between plasma concentrations of SKI-O-592, M2, 
and M4 and the percent change in activated CD63+ basophils in peripheral blood in 
patients with persistent and chronic ITP 
• To evaluate the changes of the exploratory endpoints of select (200 mg BID and 400 mg 
BID) doses of SKI-O-703 compared to placebo. 

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3 
Investigational Plan 
3.1 
Study Design 
This is a randomized, double-blind, multicenter, placebo-controlled, parallel dose study to 
evaluate the efficacy, safety, tolerability, PK, and PD of select (200 mg BID and 
400 mg BID) doses of SKI-O-703 in persistent and chronic ITP patients who have failed to 
respond or relapsed after prior therapy, with a platelet count <30,000/µL on 2 occasions at 
least 7 days apart with the confirmatory count being taken during Screening (the most recent 
result from the central laboratory will be considered as the confirmatory platelet count, when 
an unscheduled visit(s) is performed during the screening period). 
A minimum of 60 subjects are planned to enroll in 3 treatment groups (24 subjects in each of 
the active treatment groups and 12 subjects in the placebo group). Subjects will be randomly 
assigned to the 3 groups using a 2:2:1 ratio to receive one of the 2 select doses of SKI-O-703 
or placebo. First 5 subjects randomly assigned in the study will have the safety run-in period 
for the first 1 week with interim safety and PK assessments on Day 8 (±2 days). Subjects in 
the safety run-in period will continue to be dosed whilst the interim analysis is being 
conducted. However, further enrollment in each treatment group will only resume after safety 
and PK data of the first week for the first 5 subjects are reviewed and considered satisfactory 
by the Data Monitoring Committee (DMC). A subset of at least 20 subjects, who are not 
included in the safety run-in period, will be randomized to undergo PK/PD assessments at a 
selected number of sites. Randomization will be stratified to balance subjects with respect to 
prior TPO-receptor agonist use (yes/no) and degree of baseline thrombocytopenia (ie, 
baseline platelet count < or ≥15,000/µL). A schematic diagram of the overall study design is 
presented in Figure 1 and Figure 2. 

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Figure 1 
Study Design Schematic (for the First 5 Subjects in the Safety 
Run-in Period only) 
 
Abbreviation: BID, twice daily 
Note: First 5 subjects randomly assigned in the study will have safety run-in period for the first 1 week with 
interim safety and PK assessments on Day 8 (±2 days). Subjects in the safety run-in period will continue to 
be dosed whilst the interim analysis is being conducted. However, further enrollment in each treatment 
group will only resume after safety and PK data of the first week for the first 5 subjects are reviewed and 
considered satisfactory by the Data Monitoring Committee. 
 

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Figure 2 
Study Design Schematic (For Subjects Other Than Enrolled in the 
Safety Run-in Period) 
 
Abbreviation: BID, twice daily 
 
Screening Period 
The study will include up to 4 weeks of screening period. All potential subjects will sign and 
date the informed consent form (ICF) prior to any study assessments or procedures. These 
subjects will be assessed as per the eligibility criteria at Screening. After completing all 
screening assessments, subjects who meet all the inclusion and none of the exclusion criteria 
will proceed to the treatment period. 

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12-week Treatment Period 
• 1-week Safety Run-in Period (for the first 5 subjects only): First 5 subjects randomly 
assigned in the study will have the safety run-in period for the first 1 week with interim 
safety and PK assessments on Day 8 (±2 days). Subjects in the safety run-in period will 
continue to be dosed whilst the interim analysis is being conducted. However, further 
enrollment in each treatment group will only resume after safety and PK data of the first 
week for the first 5 subjects are reviewed and considered satisfactory by the DMC. 
For all subjects, on Day 1, subject’s eligibility will be confirmed and baseline safety 
evaluations will be performed (ECG, physical examination, vital sign measurements, and 
safety laboratory assessment). Eligible subjects will be randomly assigned to receive either 
one of the doses of SKI-O-703 or placebo. On Day 1, the investigative site staff will 
administer the morning dose. The evening dose of study medication will be administered 
12 hours after the morning dose. Subjects will be administered the study medication with 
enough water to swallow the capsule. The study medication will be taken no later than 
30 minutes after food. 
After dosing, all subjects will be subjected to safety assessments and a subset of at least 
20 subjects, who are not included in the safety run-in period, will be subjected to PK and PD 
assessments according to the Schedule of Events (Table 2). Subjects enrolled in the safety 
run-in period will be admitted for 24 hours on Days 1, 8, and 84 or 85 of the study. Subjects 
selected for the PK/PD portion of the study will be admitted for 24 hours on Days 1 and 84 
or 85 of the study.  
The maximum duration of study participation for a subject will be 20 weeks, which consists 
of up to 4 weeks of screening period, 12 weeks of treatment period, and 4 weeks of follow-up 
period. 
3.1.1 Dose Interruption and Reduction 
It is recognized that some AEs may require dose adjustment or study drug discontinuation. 
Intra-subject dose reduction or escalation is permitted and based upon treatment-related 
adverse events observed (Table 1). 

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After experiencing an AE, subjects whose laboratory results return to baseline values and 
remain stable for 4 consecutive weeks have the option to increase the dose of SKI-O-703 1 
dose level up to the randomized dose level. 
Table 1 
Guidelines for Dose Interruption or Reductiona 
AE 
Occurrence 
Action 
ALT elevations 
(range 3 to 
5 × ULN)  
Alkaline 
Phosphatase 
> 3 × ULN and 
> 2 × baseline 
First 
• 
If asymptomatic (no nausea, anorexia, vomiting), repeat test within 
72 hoursb and, if stable or lower, may continue dosing of study 
drug. 
• 
If on repeat test value has increased further but remains < 5 × ULN 
and asymptomatic, withhold study drug and retest weekly until 
values becomes stable. When stabilized to < 5 × ULN, continue 
dosing with the study drug. 
• 
If symptomatic or > 5 × ULN, withhold study drug. Follow the 
subject weekly until symptoms abate and values return to baseline 
per confirmation from central laboratory; at which time study drug 
may be restarted at a 50% reduced dose by taking only one daily 
dose.  
Note: Discontinue study drug permanently if the ALT elevation is 
accompanied by an elevation in direct bilirubin to > 2 mg/dL 
(34.2 mmol/L). 
 
Second 
Discontinue study drug permanently. 
ANC <1,000/mm3 
or febrile 
neutropenia 
First 
Withhold dose until ANC ≥ 1000/mm3, or has returned to baseline, 
then reduce the dose by skipping one of the daily dose and increase to 
previous dose if ANC remains stable for 2 weeks on reduced dose. 
Second 
Discontinue study drug permanently if the intolerance reoccurs or 
persists. 
Sustained increased 
systolic 
(> 160 mmHg) or 
sustained increased 
diastolic 
(> 100 mmHg) 
blood pressure in a 
previously 
normotensive 
subject 
First 
Study drug may be withheld for 5 to 7 days and then restarted at a 
reduced dose by skipping one of the daily dose. 
Irrespective of study drug dose adjustment, subjects who develop 
a sustained increased systolic (>140 mmHg) or a sustained 
increased diastolic (>90 mmHg) blood pressure should be started 
on antihypertensive medications (e.g. lisinopril 5mg daily) or have 
their antihypertensive regimen adjusted if already on any 
antihypertensive agent. 
Second 
Discontinue study drug permanently if the hypertension reoccurs or 
persists. 
Intolerable nausea 
or vomiting (see 
Section 5.8.2 for 
further details) 
First 
Study drug may be withheld for 5-7 days and then restarted at a 
reduced dose by skipping evening dose. 
Second 
Discontinue study drug permanently if the intolerance reoccurs or 
persist. 

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AE 
Occurrence 
Action 
QTcF >500 ms for 
female subject OR 
>480 ms for male 
subject, at any time 
point 
- 
The ECG should be repeated as triplicate (all three within 2 minutes) 
and a cardiology consult may be obtained as medically necessary. 
• 
If the repeat ECG confirms QTcF >500 ms (females) or >480 ms 
(males), dosing must be interrupted for up to 14 days 
• 
If the QTcF resolves to ≤480 ms (males and females)/they may 
resume dosing at the reduced dose by skipping one of the doses 
(ie, morning or evening) 
• 
If the QTcF remains >480 ms (males and females) after 14 days of 
dose interruption, the study drug must be permanently 
discontinued. 
QTcF change of 
>30 ms from 
baseline 
- 
If the QTcF has increased >30 ms when compared to the baseline with 
no other known etiology, and the principal investigator considers it 
clinically significant then a confirmatory ECG (single) will be 
performed and dose should be reduced by skipping one of the doses 
(ie, morning or evening) for the remainder of the study. 
Abbreviations: ACE, angiotensin-converting-enzyme; AE, adverse event; ALT, alanine aminotransferase; ANC, 
absolute neutrophil count; AST, aspartate aminotransferase; ECG, electrocardiogram; LFTs, liver function 
tests; MOA, mechanism of action; QTcF, corrected QT interval by Fridericia formula; ULN, upper limit of 
normal 
a 
These are examples based on previously observed AEs for agents with similar MOA (ie, fostamatinib). All 
other potential/spontaneous Grade 3/4 AEs that can warrant dose reduction or interruption are not excluded 
and will be assessed on an ongoing basis during the study. Those would be evaluated upon judgment by the 
principal investigator and the safety operations team. 
b 
A central laboratory must be used for repeat ALT, AST, ALP, or ANC. 
 
3.1.2 
Rationale of Study Design 
This double-blind, placebo-controlled study is designed to evaluate the efficacy, safety, 
tolerability, PK, and PD of SKI-O-703 in persistent and chronic ITP patients. The results of 
this study will guide on further clinical evaluation of SKI-O-703 patients with ITP. The 
double-blind placebo-controlled design has been selected to minimize bias in the evaluation 
of the safety, efficacy, PK, and PD. 
Since the 400 mg BID dose level has not been investigated in healthy volunteer studies, a 
safety run-in group will be implemented. To protect the study blind, 5 subjects will be 
included in the safety run-in period to reflect the overall 2:2:1 randomization, resulting in 
2 subjects each being dosed at 400 mg and 200 mg BID and 1 subject being dosed with 
placebo. An interim safety and PK data review will be performed by the DMC once the 
subjects in the safety run-in period complete their Day 8 (±2 days) assessments. Further 

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enrollment in each treatment group will only resume after safety and PK data of the first 
week for the first 5 subjects are reviewed and considered satisfactory by the DMC. 
Serial blood samples (5 mL) for PK assessment of SKI-O-592 and its metabolites (M2 and 
M4) from all subjects in the PK/PD subset will be obtained from this study, which in turn 
would help refine the sparse sampling approaches needed for later Phase 3 studies leading to 
a better population PK analysis in Phase 3. 
The power of a statistical test is typically a function of the magnitude of the treatment effect, 
the designated Type I error rate (α, risk of false-positive result) and the sample size (n). When 
designing a trial, it is important to decide upon the desired study power (typically 80% for a 
Phase 2 study) and calculate the necessary sample size to achieve this goal. Since it is often 
not feasible to conduct large trials during early stage of development, it is necessary to 
identify the strategies to optimize the statistical power of smaller studies. 
In this study, platelet response, defined as platelet count ≥ 30,000/µL and doubling the 
baseline (average of 2 previous counts), at any visit during the treatment period and without 
use of rescue medication, is selected as the primary endpoint. 

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4 
Subject Selection and Withdrawal Criteria 
4.1 
Selection of Study Population 
A minimum of 60 subjects will be enrolled at multiple sites in Korea, US, and EU. Subjects 
will be assigned to study drug only if they meet all of the inclusion criteria and none of the 
exclusion criteria. Deviations from the inclusion and exclusion criteria are not allowed 
because they can potentially jeopardize the scientific integrity of the study, regulatory 
acceptability, or patient safety. Therefore, adherence to the criteria as specified in the 
protocol is essential. 
4.1.1 
Inclusion Criteria 
Each subject must meet all of the following criteria to be enrolled in this study: 
1. Able to provide written informed consent and agreeable to the schedule of assessments 
2. Male or female subjects, aged 18 years or older (or ≥19 years of age for Korean subjects 
per local regulations) 
3. Diagnosis of primary ITP (persistent or chronic) made according to the American Society 
of Hematology 2011 evidence-based guideline 
4. Failed to respond or relapsed after at least 1 prior therapy (eg, corticosteroids, IVIG, 
anti-D, rituximab, TPO-receptor agonists, splenectomy, and immunosuppressants such as 
vincristine, mycophenolate mofetil, and azathioprine), with a platelet count of 
<30,000/µL on 2 occasions at least 7 days apart with the confirmatory count being taken 
during Screening (the most recent result from the central laboratory will be considered as 
the confirmatory platelet count, when an unscheduled visit(s) is performed during the 
screening period). Response to such prior therapy is defined as platelet count of 
≥30,000/µL and >2 times increase from baseline in the absence of bleeding. Failure to 
respond and relapse to such prior therapy are defined as failure to achieve and/or failure 
to maintain response on a tolerable regimen of prior therapy. 
5. Adequate hematologic, hepatic, and renal function (absolute neutrophil 
count ≥ 1.5 x 109/L, hemoglobin [Hgb] ≥ 10.0 g/dL, aspartate aminotransferase [AST] 
and alanine aminotransferase [ALT] ≤ 2.0 x ULN, bilirubin ≤1.5 x ULN, albumin 
≥ 3 g/dL, estimated glomerular filtration rate [eGFR] ≥40 mL/min/1.73m2, and 
creatinine ≤ 1.5 x ULN) 

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6. ECOG performance status of 0, 1, or 2 
7. Both male and female subjects must agree to take the following steps to reduce the 
potential for transmission of genetic material containing the investigational product: 
a. Both male and female subjects, the subject and their partners of childbearing 
potential must agree to use 2 of the following medically acceptable methods of 
contraception from the time of randomization, during the study, and for 6 months 
following discontinuation of study drug, of which: 
o One must be a highly reliable method of contraception, such as: 
 An intrauterine device or intrauterine system implanted for at least 
30 days prior to Day 1  
 Surgical sterilization of one of the partners for at least 6 months 
prior to the date of informed consent (assuming this will be the 
only partner for the whole duration of the clinical trial) 
 Consistent and correct use of hormonal contraceptives (hormonal 
implants, injectables, contraception pills, transdermal patches, or 
contraceptive rings) for at least 30 days prior to Day 1 
o One supplementary barrier method, such as: 
 Male or female condom always with spermicide (a spermicidal 
foam/gel/film/cream)  
 Diaphragm or cervical/vault caps always with spermicide (a 
spermicidal foam/gel/film/cream) 
 Double-barrier methods (which means a barrier method used by 
both partners at the same time), even when used with spermicide, 
are not considered to be highly reliable contraception methods, and 
as such, may not be the only forms of contraception used  
One of the other listed highly reliable methods must be used in 
conjunction with a barrier method. 
b. Female subjects must agree not to breastfeed starting from the time of Screening, 
throughout the study, and until after 6 months following the last dose of study 
drug. 

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c. Male subjects must agree not to donate sperm starting from the time of 
randomization, throughout the study, and until after 6 months following the last 
dose of study drug  
d. For subjects and partners considered not of childbearing potential, the following 
conditions apply: 
a. Menopausal females must have experienced their last period more than 
12 months prior to the date of informed consent to be classified as not of 
childbearing potential. 
b. Male and female subjects are in a situation of abstinence from 
heterosexual intercourse from Screening until after 6 months following the 
last dose of study drug when this is in line with the preferred lifestyle of 
the subject (eg, homosexual women and men or a member of a religious 
order such as nuns and priests). 
4.1.2 
Exclusion Criteria 
Subjects meeting any of the following criteria will be excluded from the study: 
1. 
History of any clinically significant disease or disorder that in the opinion of the 
investigator or the sponsor may put the subject at risk due to study participation, impact 
the subject’s ability to participate in the study, or influence the study results 
2. 
History of current, active malignancy requiring or likely to require chemotherapeutic or 
surgical treatment during the study, with the exception of non-melanoma skin cancer, 
carcinoma in situ of the cervix, and localized prostate cancer managed by active 
surveillance 
3. 
Transfusion with blood or blood products or plasmapheresis within 2 weeks before the 
first administration of study drug (Day 1) 
4. 
PT INR > 1.5 
5. 
History of known inherited coagulopathy (including prothrombotic conditions such as 
Factor V Leiden, APC resistance, AT-III deficiency, and lupus anticoagulant), or recent 
arterial or deep venous thrombosis within the preceding 6 months 

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6. Change in corticosteroid or immunosuppressant (azathioprine, mycophenolate mofetil, 
cyclosporine) dose within 2 weeks prior to Day 1 (more than 10% variation from Day 1 
daily doses) 
7. 
Treatment with thrombopoietin (TPO) receptor agonists within 2 weeks before Day 1 
8. 
Treatment with rituximab or splenectomy within the 8 weeks prior to Day 1 
9. 
Treatment with IVIGs within 4 weeks prior to Day 1 
10. 
Infections requiring intravenous antibiotics or hospitalization within 3 months prior to 
Day 1 
11. 
Subject had positive test results at Screening for human immunodeficiency virus, 
hepatitis B surface antigen (HBsAg), or hepatitis C virus antibody. Subjects with past 
or resolved hepatitis B infection (defined as having a negative HBsAg test and a 
positive IgG antibody to hepatitis B core antigen [anti-HBc]) are eligible but 
hepatitis B virus (HBV) DNA must be obtained in these subject prior to Cycle 1, 
Day 1, and must demonstrate no active infection. Subjects positive for hepatitis C virus 
(HCV) antibody are eligible only if polymerase chain reaction (PCR) is negative for 
HCV RNA. 
12. 
Live vaccine within 28 days prior to Day 1 or plan to receive one during the study 
13. 
History or presence of any gastrointestinal, hepatic, or renal disease or any other 
condition known to interfere with the absorption, distribution, metabolism, or excretion 
of drugs 
14. 
Subject has not recovered from a recent medical/surgical procedure or trauma by Day 1 
as determined by the investigator 
15. 
Uncontrolled hypertension (as defined by systolic blood pressure ≥160 mmHg or 
diastolic blood pressure ≥100 mmHg) 
16. 
Subject had ECG findings of corrected QT interval by Fridericia formula 
(QTcF) > 480 msec, cardiac arrhythmias, or clinically significant cardiac or ECG 
abnormalities 
17. 
Subject have received any investigational medication within 30 days or 5 half-lives 
prior to Day 1, whichever was longer 

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18. 
Concomitant use of any anticoagulants and platelet aggregation inhibiting drugs 
including aspirin (within 14 days of planned dosing through end of follow-up) 
19. 
Female subject who is currently pregnant or breastfeeding 
20. 
Prior treatment with a SYK inhibitor 
21. 
Planned surgery in the time frame of the dosing period. 
4.2 
Withdrawal of Subjects From Study Drug and/or the Study 
The duration of the study is defined for each subject as the date signed informed consent is 
provided through date of the last follow-up visit on Week 16. 
4.2.1 
Reasons for Withdrawal/Discontinuation 
Subjects may withdraw from the study at any time and for any reason without prejudice to 
their future medical care by the investigator or at the study site. Every effort should be made 
to keep subjects in the study. The reasons for subjects not completing the study will be 
recorded. A subject may be withdrawn from the study for any of the following reasons: 
1. Noncompliance with the protocol 
2. A serious or intolerable adverse event(s) (AE[s]) that in the investigator’s opinion 
requires withdrawal from the study 
3. Central nervous system bleeding or life-threatening gastro-intestinal bleeding. Please also 
refer to the additional information in the ‘Note’ at the bottom of this section. 
4. Laboratory safety results that reveal clinically significant hematological or biochemical 
changes from the baseline values 
5. Symptoms of an intercurrent illness not consistent with the protocol requirements or that 
justify withdrawal 
6. Lost to follow-up. Subjects that are considered lost to follow-up, reasonable attempts 
must be made to obtain information on the final status of the subject. At a minimum, 
2 phone calls must be made and 1 certified letter must be sent for documentation 
7. Other reasons (eg, pregnancy, development of contraindications of use of study drug, 
breakthrough bleeding) 

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8. The subject withdraws consent or the investigator or sponsor decides to discontinue the 
subject’s participation in the study 
9. Death of the subject. 
Note: Bleeding events in the ITP studies are common during the first few weeks of the study 
before the drug has a chance to take effect; hence, all the bleeding events in this study should 
be recorded as an AE or SAE (based on seriousness criteria). These events should not result 
in removal of the subjects from the study, at least during the first 4 weeks of treatment. As the 
standard of care among the participating countries may differ, platelet transfusions are 
allowed as a rescue therapy for such events; however, these therapies may be reserved for 
serious or life-threatening bleeding events. 
The investigator will also withdraw a subject if Oscotec Inc. terminates the study. Upon 
occurrence of a serious or intolerable AE, the investigator will confer with the sponsor. If a 
subject is discontinued because of an AE, the event will be followed until it is resolved. Any 
subject may withdraw his or her consent at any time. Although Oscotec Inc. has every 
intention of completing the study, Oscotec Inc. reserves the right to discontinue the study at 
any time for clinical or administrative reasons, please refer to Section 11.3 for further details. 
4.2.2 
Handling of Withdrawals 
Subjects are free to withdraw from the study or study drug at any time upon request. Subject 
participation in the study may be stopped at any time at the discretion of the investigator or at 
the request of the sponsor. 
Subjects who discontinue study drug or active participation in the study will no longer 
receive study drug. When a subject withdraws from the study drug or active participation in 
the study, the reason(s) for withdrawal shall be recorded by the investigator on the relevant 
page of the electronic case report form (eCRF). Whenever possible, all subjects who 
discontinue study drug permanently or withdraw from the study prematurely will undergo all 
end-of-study assessments. Subjects who fail to return for final assessments will be contacted 
by the site in an attempt to have them comply with the protocol. Following a minimum of 
2 documented unsuccessful telephone calls, a registered letter will be sent to the subject in a 
final attempt to ensure protocol compliance. 

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It is vital to obtain follow-up data on any subject withdrawn because of an AE or SAE. In 
every case, efforts must be made to undertake protocol-specified safety follow-up 
procedures. 
4.2.3 
Replacements 
Subjects who are randomly assigned and not dosed will be replaced. Subjects who 
discontinue the study for any reason after dosing will not be replaced. 

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5 
Study drugs 
Two dose levels of SKI-O-703 will be evaluated: 200 mg BID and 400 mg BID. The 
SKI-O-703 capsules will contain 100 mg of drug substance. Placebo capsules are filled with 
microcrystalline cellulose. 
5.1 
Method of Assigning Subjects to Treatment Groups 
Subjects will be randomly assigned in a 2:2:1 ratio to receive SKI-O-703 200 mg BID, 
SKI-O-703 400 mg BID, or matching placebo. A subset of at least 20 subjects will be 
randomized to undergo PK/PD assessments at a selected number of sites. An interactive 
voice response system (IVRS) will be used to administer the randomization schedule. The 
randomization schedule will be generated using SAS software Version 9.4 or later (SAS 
Institute Inc., Cary, North Carolina) for IVRS, which will link sequential subject 
randomization numbers to treatment codes. The randomization will be stratified to balance 
subjects with prior TPO-receptor agonist use (yes/no) and degree of baseline 
thrombocytopenia (ie, baseline platelet count < or ≥15,000/µL). It will also use an 
appropriate block size, which will not be revealed. 
5.2 
Treatments Administered 
Subject specific doses will be prepared and checked by blinded pharmacy staff and 
administered by appropriately trained blinded clinic staff as delegated by the principal 
investigator at the study site. The study drugs will be identical in number and appearance for 
each group. The following dosing scheme will be performed: 
• 200 mg BID group: 2 capsules of 100 mg SKI-O-703 + 2 capsule of placebo 
• 400 mg BID group: 4 capsules of 100 mg SKI-O-703 + 0 capsules of placebo 
• Placebo group: 4 capsules of placebo 
SKI-O-703 200 mg BID, 400 mg BID, and placebo will be administered orally. On Day 1, 
the investigative site staff will administer the morning dose of study medication. The evening 
dose will be administered 12 hours after the morning dose. Subjects will be administered the 
study medication with enough water to swallow the capsules. The investigative site staff will 
check the subject’s mouth to ensure the study drug and entire volume of water was 
swallowed. The study medication will be taken no later than 30 minutes after food. Subjects 
enrolled in the safety run in period will be admitted for 24 hours on Days 1, 8, and 84 or 85 

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of the study. Subjects selected for the PK/PD portion of the study will be admitted for 24 
hours on Days 1 and 84 or 85 of the study. The subject will be instructed to self-administer 
the subsequent doses of the study medication in the morning and evening (12 hours after the 
morning dose) for the remaining study duration. 
Dosing of the study medication that occur on study visit days, subjects will take the morning 
dose of study drug in the office under the guidance of study site personnel after all specified 
study tests and procedures have been completed. However, if the visit cannot be scheduled in 
the morning, the subject will be instructed to take the morning dose at the regular schedule 
on that visit day. 
Each subject’s dosing schedule should be closely monitored by the site at each study visit. 
This will ensure that all subjects enrolled into the study maintain their original dosing 
schedule beginning with the first dose of study drug. 
If the subjects forget to take their study drug dose at their regularly scheduled dosing time, 
they should take the forgotten dose as soon as they remember the dose was missed, within 
4 hours of scheduled timepoint or at least 8 hours before the next scheduled dose. If the 
subjects experience any drug interruption(s), they should notify their study site physician. 
Such protocol violations may result in data that are not deemed evaluable (by the 
investigator) for a protocol analysis and/or may require subject(s) to be discontinued. 
5.3 
Identity of Investigational Product 
SKI-O-703 capsule is a Swedish orange capsule. Oscotec Inc. will provide adequate supplies 
of blinded SKI-O-703 and placebo capsules to the study site. 
SKI-O-703 capsules will contain the active ingredient SKI-O-703 (100 mg). 
Placebo capsules will be identical in appearance to the SKI-O-703 capsule and contain only 
microcrystalline cellulose and magnesium stearate. 
5.4 
Management of Clinical Supplies 
5.4.1 
Study Drug Packaging and Storage 
SKI-O-703 (100 mg) capsules and matching placebo capsules will be manufactured using a 
ProFill® capsule filler, packaged 70 counts each into high density polyethylene bottles, and 

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shipped to the study site. Study drug will be packaged and labelled according to applicable 
local and regulatory requirements. 
Study drug must be stored in accordance with the manufacturer’s instructions. The storage 
conditions and expiry date will be indicated on the label. At the study site, study drug must 
be stored in a securely locked area and kept at a controlled room temperature with excursions 
between 15°C and 30°C. 
5.4.2 
Test Article Accountability 
The sponsor or affiliate will ship a sufficient supply of study drug to the study site. The 
investigator will maintain accurate records of receipt of all test articles, including dates of 
receipt. In addition, accurate records will be kept regarding when and how much test article 
is dispensed and used by each subject in the study. Subjects will be instructed to return all 
drug containers (even if empty) to the study site personnel at each clinic visit. Reasons for 
departure from the expected dispensing regimen must also be recorded. At the completion of 
the study, to satisfy regulatory requirements regarding drug accountability, all study drug will 
be reconciled and retained or destroyed according to applicable regulations. 
Subject dosing will be recorded on eCRF. Study drug will be dispensed bi-weekly (34 doses, 
17 days’ worth of drug at a time) to the subjects. Subjects will be instructed to return all drug 
containers (even if empty) to the study site personnel at each clinic visit. Site personnel will 
document compliance in the study source documents. 
5.5 
Overdose Management 
An overdose is any dose of study drug given to a subject or taken by a subject that exceeds 
the dose described in the protocol. A medication error is a preparation, dispensing, or 
administration error. Any overdose or medication error, with or without associated AEs, must 
be promptly reported to the investigator, medical monitor, and sponsor. Overdoses and 
medication errors without signs or symptoms do not need to be recorded as AEs; in case of 
any AEs associated with the overdose or medication error, these should be reported on 
relevant AE/SAE sections in the eCRF. 
Overdoses and medication errors will be documented as protocol deviations and 
communicated to the clinical study manager and/or safety manager. 

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No specific therapy for an overdose of SKI-O-703 exists. In the event of an overdose or 
medication error, therapy appropriate for the subject’s symptoms and clinical status should be 
provided. 
5.6 
Blinding 
This is a double-blind study. Neither the subjects nor the investigator/site personnel will be 
aware of the treatment assignment for the subjects in each cohort. Additionally, the study 
drugs will be identical in number and appearance for each cohort. Blinding will be 
maintained throughout the study by use of active or placebo dosage forms of similar 
appearance. 
The DMC will receive first 1-week safety and PK unblinded data by treatment group or 
unblinded (if necessary) confidential reports of the first 5 subjects enrolled in the safety 
run-in period from an independent statistician for review, which have to be handled strictly 
confidentially. None of these reports can be delivered to unauthorized persons. 
5.6.1 
Breaking the Blind 
A subject’s treatment assignment will not be broken until the end of the study unless medical 
treatment of the subject depends on knowing the study treatment the subject received, eg, 
bleeding events requiring rescue medication do not require breaking the blind unless 
knowing the treatment allocation may result in different management. 
In the event that the blind needs to be broken because of a medical emergency, the 
investigator may unblind an individual subject’s treatment allocation. As soon as possible, the 
investigator should first contact the medical monitor to discuss the medical emergency and 
the reason for revealing the actual treatment received by that subject. The treatment 
assignment will be unblinded through IVRS. Reasons for treatment unblinding must be 
clearly explained and justified in the eCRF. The date on which the code was broken together 
with the identity of the person responsible must also be documented. 

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5.7 
Treatment Compliance 
Day 1 doses of the study drug will be administered and recorded by study site personnel, and 
any deviations from the dosing schedule will be entered into the subject’s eCRF. From Day 2 
onwards, subjects will be instructed to self-administer their morning dose of study 
medication. The subject will be instructed to self-administer their evening dose 12 hours after 
the morning dose. Treatment compliance will be determined from subject dosing data in 
eCRF. 
5.8 
Prior and Concomitant Therapy 
Use of all concomitant medications that the subject is receiving at the time of Screening or 
receives during the study must be recorded in the subject’s eCRF. The minimum requirement 
is the reason for drug use, drug name, and the dates of administration (including start and end 
dates), dosage information including dose, route, and frequency are to be recorded in the 
eCRF. This will include all prescription drugs, herbal products, vitamins, minerals, vaccines, 
and over-the-counter medications (including folic acid). Any changes in concomitant 
medications also will be recorded in the subject’s eCRF. 
Any concomitant medication, except the prohibited medications listed in Section 5.8.3, 
deemed necessary for the welfare of the subject during the study may be given at the 
discretion of the investigator. However, it is the responsibility of the investigator to ensure 
that details regarding the medication are recorded in full in the eCRF. 
Subjects will be allowed to receive stable doses of background medications in line with the 
current standard of care. The background medications allowed include corticosteroids 
(<20 mg prednisone equivalent per day) and immunosuppressive drugs such as azathioprine 
(≤50 mg twice daily), mycophenolate mofetil (≤500 mg twice daily), and cyclosporine 
(≤100 mg twice daily). The dose of such background medications should be fixed for at least 
2 weeks before Day 1 and remain unchanged until 12 weeks of treatment have been 
completed or unless rescue therapy is required. 
5.8.1 
Rescue Medications 
In case of bleeding events requiring rescue medication, the following rescue medications are 
permitted: IVIG 1 g/kg/day for 2 days (ie, 2 g/kg), romiplostim 2 to 5 µg/kg, dexamethasone 
40 mg/day for 4 days, other high dose parenteral steroids (ie, solumedrol 1 g IV), platelet 

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transfusions (these may be reserved for serious or life-threatening bleeding events), or 
standard of care as per participating countries. 
Subjects should be withdrawn in case of CNS bleeding or life-threatening GI bleeding (see 
Section 4.2.1). 
5.8.2 
Management of Nausea and Vomiting 
Based on Investigator’s discretion, nausea and vomiting can be managed based the National 
Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) grades 
as described below.  
• Grade 1: No routine anti-emetics are necessary. Consider oral domperidone 20 mg 
three times a day as needed or oral metoclopramide 20 mg three times a day as 
needed 
• Grade 2: Oral domperidone 20 mg three times a day as needed or oral 
metoclopramide 20 mg three times a day as needed 
• Grade 3: Oral domperidone 20 mg three times a day as needed or oral 
metoclopramide 20 mg three times a day as needed and consider levomepromazine 
6 mg at night as needed 
• Grade 4: Oral aprepitant 80 mg QD or oral domperidone 20 mg three times a day or 
oral metoclopramide 20 mg three times a day 
5.8.3 Prohibited Concomitant Medications 
The medical monitor must be notified in advance (or as soon as possible thereafter) of any 
instances in which prohibited medications are administered. Prohibited medications during 
the study include: 
• Any biological agents for the treatment of ITP 
• Any rescue medications other than those described in Section 5.8.1 
• Any immunosuppressant drugs other than those described in Section 5.8 
• Change in corticosteroid and immunosuppressants dose as detailed in the exclusion 
criteria 

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• Use of IVIGs within 4 weeks prior to Day 1 
• Treatment with rituximab or splenectomy within the 8 weeks prior to Day 1 
• Any parenteral antibiotics as specified in the exclusion criteria 
• Any live vaccine taken within 28 days prior to Day 1 or plan to receive one during 
the study 
• Concomitant use of any anticoagulants and platelet aggregation inhibiting drugs 
including aspirin (within 14 days of planned dosing through end of follow-up) 
• Known CYP1A2 and UGT1A1 inhibitors and inducers (See Appendix 13.1 for a 
list of suggested named CYP1A2 and UGT1A1 inhibitors and inducers). These 
medications must be discontinued within 5 half-lives or 4 weeks prior to Day 1, 
whichever is longer. 
 

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6 
Study Assessments and Procedures 
Before performing any study procedures, all potential subjects will sign an ICF. Subjects will 
have the opportunity to have any questions answered before signing the ICF. The investigator 
must address all questions raised by the subject. The investigator will also sign the ICF. 
6.1 
Study Visits 
The total study duration will be 20 weeks per subject, which consists of up to 4 weeks of 
screening period. The treatment period will be 12 weeks (split into 1-week safety run-in 
period and 11-week treatment period for the first 5 subjects). The post-treatment follow-up 
period will be of 4 weeks. Each assessment will be carried out at timepoints indicated in the 
Schedule of Events table and clarified in the footnotes presented below the table. The 
schedule of events is summarized in Table 2.

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Table 2 
Schedule of Events 
  
  
On-treatment Visits 
Follow-up Clinic Visits 
Early 
Withdrawal/ 
Unscheduled 
visit 
  
Screening 
(D-28 to 
D-1) 
W1 (D1), 
W5 and 
9 (±3 days) 
W1D4 
(±1 day) 
W2D8 
(±2 days) 
W3, 7, 
11 (±3 days) 
W12 (D84 or 
D85) (EOT) 
(±3 days) 
End of Study 
W16 (±1 week) 
Clinic visits 
X 
Xa 
Xa* 
Xa 
Xa 
Xa 
X 
Informed consent 
X 
 
 
 
 
 
 
 
Inclusion/Exclusion criteria 
X 
Xh 
 
 
 
 
 
 
AEs 
<--------------------------------------------------------------------------------------------------------------> 
X 
Concomitant medications 
X 
X 
 
X* 
X 
X 
X 
X 
Height 
X 
 
 
 
 
 
 
 
Weight 
X 
X 
 
X* 
 
X 
X 
X 
Physical examination/medical 
historyb 
X 
X 
 
X* 
X 
X 
X 
X 
ECGc 
X 
X 
 
X 
 
X 
X 
X 
Vital signs 
X 
X 
 
X* 
X 
X 
X 
X 
Urinalysis 
X 
X 
 
X* 
X 
X 
X 
X 
Hep B & C, HIV, HBV DNA, 
HCV RNA 
X 
 
 
 
 
 
 
 
Pregnancy test/ FSHd 
X 
X 
 
 
 
X 
X 
X 
Contraception status 
X 
X 
 
X* 
 
X 
X 
X 
ABO and Rh blood type 
X 
 
 
 
 
 
 
 
Serum chemistry 
X 
X 
 
X* 
X 
X 
X 
X 
Hematology, differential 
X 
X 
Xa,e 
Xa,e 
X 
X 
X 
X 
PT/INR PTT 
X 
 
 
 
 
X 
 
 
Study drug dispensing 
 
X 
 
 
X 
 
 
Xf 
Study drug administration 
 
X 
X 
X* 
X 
X 
 
Xf 
Blood samples for PK and PD 
assessmentsg 
 
Xh 
 
X* 
 
X 
 
 

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On-treatment Visits 
Follow-up Clinic Visits 
Early 
Withdrawal/ 
Unscheduled 
visit 
  
Screening 
(D-28 to 
D-1) 
W1 (D1), 
W5 and 
9 (±3 days) 
W1D4 
(±1 day) 
W2D8 
(±2 days) 
W3, 7, 
11 (±3 days) 
W12 (D84 or 
D85) (EOT) 
(±3 days) 
End of Study 
W16 (±1 week) 
Bleeding Scorei 
 
X 
 
X* 
 
X 
X 
X 
Quality of life using SF-36 score 
 
X 
 
 
 
X 
X 
X 
Pharmacogenomic screening 
 
Xj 
 
 
Abbreviations: AEs, adverse events; D, Day; ECG, 12-lead electrocardiogram; EOT, end-of-treatment; FSH, follicle stimulating hormone; HBV, hepatitis B virus; HCV, 
hepatitis C virus; hep, hepatitis; HIV, human immunodeficiency virus; PD, pharmacodynamics; PK, pharmacokinetics; PT/INR, prothrombin time/international normalized 
ratio, PTT, partial thromboplastin time; W, Week 
* 
Subjects in the safety run-in period will have clinic visit on Week 2/Day 8 (±2 days) for the mentioned assessments including hematology (differential) tests. For other 
subjects, this visit will be laboratory only visit to perform hematology (differential) tests only. 
a 
Clinic / laboratory visit windows are about ± 3 days for all dosing period and laboratory visits and about ± 1 week for clinic follow-up visits. The W12/D84 or D85 (EOT) 
visit should be performed while on therapy. 
b 
Complete physical examination at Screening and abbreviated physical examination at other visits. 
c 
For all subjects: predose ECG on the first day of dosing and on Day 1 of each subsequent week as specified in the table above. For subjects in the safety run-in subsets and 
PK/PD subset who are in safety run-in subset only: ECG on the first day of dosing at predose, 1, 2, 4, 8, and 12 hours (±10 min) after the study drug administration. 
d 
For women of childbearing potential only: Serum pregnancy tests are performed at Screening. To confirm pregnancy, urine pregnancy test will be performed locally on Day 1 
prior to randomization and prior to study drug administration on clinic visit days at mentioned timepoints. If urine pregnancy test is negative, dosing will be started; if urine 
pregnancy test is positive then with-hold dosing and perform a serum pregnancy test. FSH testing is needed at Screening for post-menopausal female subjects only. 
e 
Laboratory only visits will be performed on Day 4 ±1 day (Week 1) for all subjects and on Week 2/Day 8 (±2 days) for subjects other than those enrolled in the safety run-in 
period.  
f 
Study drug dispensing and administration can occur at unscheduled visit.  
g 
Samples to be collected as follows: 
For the subjects in the safety run-in period only (for PK only; no PD assessments):  
• 
Day 1: morning predose, 0.25 (+5 min), 0.5 (±5min), 1, 2, 4, 8, and 12 hours (±10 min) (evening predose).  
• 
Day 8 (±2 days): morning predose, 0.25 (+5 min), 0.5 (±5min), 1, 2, 4, 8, and 12 hours (±10 min) (evening predose). 
• 
Week 12: morning predose, 0.25 (+5 min), 0.5 (±5min), 1, 2, 4, 8, and 12 hours (±10 min). 
For the subjects in the PK/PD subset only (for PK/PD): 
• 
Day 1: morning predose, 0.25 (+5 min), 0.5 (±5min), 1, 2, 4, 8, and 12 hours (±10 min) (evening predose).  
• 
Week 12: morning predose, 0.25 (+5 min), 0.5 (±5min), 1, 2, 4, 8, and 12 hours (±10 min). 
For other subjects not included in the safety run-in period or PK/PD subset (for PK only): 
• 
Week 12: morning predose. 
h 
Only on Day 1 (Week 1). 

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i 
Bleeding score assessment as described by Page et al 2007 should be performed on Day 1 of each week specified in the above table and at follow-up visit. 
j 
A blood sample for genotyping will be drawn once at any clinic visit from Day 1 (Week 1) to the end-of-treatment visit, only from subjects who have consented to participate 
in the genetic analysis component of the study. 
 

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6.2 Demographic and Medical History 
Demographic data and a complete medical history (including prior and concomitant medical 
conditions and procedures; drug, alcohol, and tobacco use; current use of herbal remedies, 
vitamin supplements, grapefruit juice, and caffeine- and xanthine-containing products) will 
be collected at Screening. 
6.3 
Efficacy Assessments 
Efficacy will be assessed based on the laboratory and clinical assessments/bleeding score. 
See Section 7. 
6.4 
Safety Assessments 
Safety variables will include physical examination findings, vital sign measurements 
(systolic and diastolic blood pressure, pulse rate, respiratory rate, and temperature), ECG 
tracings, clinical laboratory test results (hematology, coagulation, serum chemistry, and 
urinalysis), bleeding score, quality of life as measured by the SF-36 score, weight, body mass 
index, and reporting of AEs. 
Bleeding will be assessed according to the bleeding score as described by Page et al 2007. 
These assessments will be carried out at the time points indicated in the schedule of events. 
Safety assessments will be conducted as shown in the schedule of events (Table 2). 
6.4.1 Physical Examination 
A complete physical examination will be performed at the screening visit and an abbreviated 
physical examination at other time points indicated in the schedule of events (Table 2). An 
abbreviated physical examination will include checks of general appearance, head and neck, 
abdomen, lymph nodes, skin, cardiovascular system, respiratory system, and musculoskeletal 
system. 
A complete physical examination will include assessments of skin, head, ears, eyes, nose, 
throat, neck, thyroid, lungs, cardiovascular system, abdomen, lymph nodes, and 
musculoskeletal system/extremities. Interim physical examinations can be performed at the 
discretion of the investigator, if deemed necessary to evaluate AEs or clinical laboratory 
abnormalities. 

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6.4.2 Vital Sign Measurements, Height, and Weight 
Vital sign measurements will include systolic and diastolic blood pressures, pulse rate, 
respiratory rate, and temperature. The subject will be seated for at least 5 minutes before all 
measurements are taken. Vital signs will be measured at the time points indicated in the 
schedule of events (Table 2). 
Height will be measured at Screening only. Weight will be measured at the time points 
indicated in the schedule of events (Table 2). 
When procedures are overlapping and occurring at the same time point, the order in which 
procedures are performed should be vital sign measurements followed by blood collection. 
The investigator will determine whether any of the vital sign measurements are clinically 
significant or not clinically significant. Clinical significance is defined as any variation in 
results that has medical relevance and may result in an alteration in medical care (eg, active 
observation, diagnostic measures, or therapeutic measures). If a clinically significant change 
from the screening values is noted, the clinically significant value and the reason for clinical 
significance will be documented on the AE page of the subject’s eCRF. The investigator will 
continue to monitor the subject with additional assessments until the value has reached either 
the reference range or the value at Screening or until the investigator determines that 
follow-up is no longer medically necessary. 
6.4.3 Electrocardiograms 
For all subjects, single ECG (predose) will be obtained, after the subject has been in the 
supine position for at least 5 minutes, on the first day of dosing and on Day 1 of each 
subsequent week at the time points indicated in the schedule of events (Table 2). For subjects 
in the safety run-in subsets and PK/PD subset subjects who are in safety run-in subset only, 
ECGs will be obtained on the first day of dosing at predose, 1, 2, 4, 8, and 12 hours (±10 
min) after the study drug administration. Electrocardiogram assessments will include 
comments on the rhythm; presence of arrhythmia or conduction defects; morphology; or 
ST segment, T wave, and U wave abnormalities; as well as whether the tracings are normal 
or abnormal and if there is any evidence of myocardial infarction. In addition, the following 
parameters will be measured and reported: RR interval, PR interval, QRS width, QT interval, 
and QTcF. 

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The investigator will determine whether any of the ECG results are normal or abnormal and 
whether any abnormal results are clinically significant or not clinically significant. Clinical 
significance is defined as any variation in results that has medical relevance and may result in 
an alteration in medical care (eg, active observation, diagnostic measures, or therapeutic 
measures). If a clinically significant change from screening is noted, the clinically significant 
value and the reason for clinical significance will be documented on the AE page of the 
subject’s eCRF. The investigator will continue to monitor the subject with additional 
assessments until the values have reached either the reference range or the values at 
Screening or until the investigator determines that follow-up is no longer medically 
necessary. 
6.4.4 Clinical Laboratory Tests 
Clinical laboratory tests will be performed by PPD’s central laboratory. Blood and urine 
samples will be collected under fasted conditions and at the time points indicated in the 
schedule of events (Table 2). 
For women of childbearing potential only: Serum pregnancy tests will be performed at 
Screening. To confirm pregnancy, urine pregnancy test will be performed locally. For 
post-menopausal female subjects only, FSH testing will be performed at Screening. 
The following hematology, coagulation, serum chemistry, and urinalysis assessments will be 
performed: 
Hematology: Hematocrit, hemoglobin, mean corpuscular hemoglobin, mean corpuscular 
hemoglobin concentration, mean corpuscular volume, platelet count, red blood cell 
count, total and differential (absolute and percent) leukocyte count, and erythrocyte 
sedimentation rate 
Coagulation: International normalized ratio, partial thromboplastin time, and prothrombin time, 
ABO and Rh Blood Type 
Serum 
Chemistry: 
Alanine aminotransferase, albumin, alkaline phosphatase, AST, bilirubin (total and 
direct if clinically indicated), blood urea nitrogen, calcium, creatinine, 
gamma-glutamyltransferase, glucose, lactate dehydrogenase, serum electrolytes 
(sodium, potassium, chlorine, magnesium, phosphate) and total protein 
Urinalysis: 
Appearance, bilirubin, color, glucose, ketones, leukocyte esterase, microscopy 
(performed if dipstick is positive; includes bacteria, casts, crystals, epithelial cells, 
red blood cells, and white blood cells), nitrites, occult blood, pH, protein, specific 
gravity, turbidity, and urobilinogen 
 

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Abnormal clinical laboratory values will be flagged as either high or low (or normal or 
abnormal) based on the reference ranges for each laboratory parameter. The investigator will 
determine whether any abnormally high or low results are clinically significant or not 
clinically significant. Clinical significance is defined as any variation in results that has 
medical relevance and may result in an alteration in medical care (eg, active observation, 
diagnostic measures, or therapeutic measures). If a clinically significant change from the 
screening value is noted, the clinically significant value and the reason for clinical 
significance will be documented on the AE page of the subject’s eCRF. The investigator will 
continue to monitor the subject with additional assessments until the values have reached 
either the reference range or the values at Screening or until the investigator determines that 
follow-up is no longer medically necessary. 
The FDA guidance for industry on Drug-Induced Liver Injury: Premarketing Clinical 
Evaluation (July 2009) will be used as a guideline for the assessment of liver enzymes/liver 
function tests (DHHS 2009). 
Clinically significant laboratory values for individual subjects will be listed. A summary for 
the numbers and percentages of subjects with clinically significant laboratory values at any 
time point will be presented. 
6.4.5 
Adverse Events 
6.4.5.1 
Definitions of Adverse Events 
The investigator is responsible for reporting all TEAEs that are observed or reported during 
the study, regardless of their relationship to study drug or their clinical significance. 
An AE is defined as any untoward medical occurrence in a subject enrolled into this study 
regardless of its causal relationship to study drug. Subjects will be instructed to contact the 
investigator at any time after randomization if any symptoms develop. 
A TEAE is defined as any event not present before exposure to study drug or any event 
already present that worsens in either intensity or frequency after exposure to study drug. 

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6.4.5.2 
Serious Adverse Events 
An SAE is defined as any event that: 
• Results in death 
• Is immediately life threatening 
• Requires inpatient hospitalization or prolongation of existing hospitalization 
• Results in persistent or significant disability/incapacity 
• Is a congenital anomaly/birth defect 
Important medical events that may not result in death, be life threatening, or require 
hospitalization may be considered SAEs when, based upon appropriate medical judgment, 
they may jeopardize the subject or 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 inpatient hospitalization, or the development of 
drug dependency or drug abuse. 
Note: The AE terms neutropenia and hypertension should be used only when reporting 
events that meet the following criteria:  
• The term neutropenia is reserved for reporting an absolute neutrophil 
count <1000/mm3 or <1.0 × 109/L and may or may not be associated with fever or 
concurrent infection. 
• The term hypertension is reserved for reporting an increase in systolic blood pressure 
≥160 mmHg, or diastolic blood pressure ≥100 mmHg, or any clinically significant 
increase in blood pressure that requires modification of the subject’s antihypertensive 
regimen. 
6.4.5.3 
Eliciting and Documenting Adverse Events 
Adverse events will be assessed from the time the subject signs the informed consent form 
until end of study visit (Week 16). 

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If the investigator becomes aware of an SAE that occurs after the end of study visit and is 
assessed by the investigator as at least possibly related to the study drug, they need to report 
the SAE. 
At every study visit, subjects will be asked a standard nonleading question to explore a 
response regarding any medically related changes in their well-being. They will also be asked 
if they have been hospitalized, had any accidents, used any new medications, or changed 
concomitant medication regimens (both prescription and over-the-counter medications). 
In addition to subject observations, AEs identified from any study data (eg, laboratory values, 
physical examination findings, ECG changes) or identified from review of other documents 
that are relevant to subject safety will be documented on the AE page in the eCRF. 
6.4.5.4 
Reporting Adverse Events 
All AEs reported or observed during the study will be recorded on the AE page in the eCRF. 
Information to be collected includes drug treatment, dose, event term, time of onset, 
investigator-specified assessment of severity and relationship to study drug, time of 
resolution of the event, seriousness, any required treatment or evaluations, and outcome. 
Adverse events resulting from concurrent illnesses, reactions to concurrent illnesses, 
reactions to concurrent medications, or progression of disease states must also be reported. 
All AEs will be followed to adequate resolution. The Medical Dictionary for Regulatory 
Activities (MedDRA v20.0) will be used to code all AEs. All AEs, SAEs, drug-related AEs, 
and drug-related SAEs will be summarized using the worst grade per NCI CTCAE v5.0 by 
system organ class and preferred term. 
Any medical condition that is present at the time that the subject is screened but does not 
deteriorate should not be reported as an AE. However, if it deteriorates at any time during the 
study, it should be recorded as an AE. 
6.4.5.5 
Reporting Serious Adverse Events 
Any AE that meets SAE criteria (Section 6.4.5.2) must be reported to PPD immediately (ie, 
within 24 hours) after the time that site personnel first learn about the event. The nonserious 
adverse event of special interest (AESI) defined in Section 6.4.5.6 should be recorded on the 
SAE report form in addition to the eCRF and faxed using the same process as for reporting 
SAEs. 

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Pharmacovigilance 
 
 
6.4.5.6 
Suspected Unexpected Serious Adverse Reactions and 
Nonserious Adverse Events of Special Interest 
The following nonserious AESI should be recorded on the SAE report form in addition to the 
eCRF and faxed using the same process as for reporting SAEs. 
• Neutropenia with fever defined as absolute neutrophil count <1000/mm3 or <1.0 × 109/L 
on the most recent complete blood count with fever equal to or greater than 38°C that 
does not fulfill any of the serious criteria, ie, hospitalization. 
• Hepatotoxicity defined as ALT >3 × ULN plus total bilirubin >2 × ULN that does not 
fulfill any of the serious criteria, ie, hospitalization. This information should be recorded 
on the drug induced liver injury eCRF each time the elevated laboratory parameters are 
met during the study 
Oscotec Inc. will promptly evaluate all suspected unexpected serious adverse reactions 
(SUSARs) and nonserious AESI against cumulative product experience to identify and 
expeditiously communicate possible new safety findings to investigators, Institutional 
Review Board (IRB)/Independent Ethics Committee (IEC), and applicable health authorities 
based on applicable legislation. 
To determine reporting requirements for single AE cases, Oscotec Inc. will assess the 
expectedness of these events using the study drug investigator’s brochure. 
Oscotec Inc. will compare the severity of each SUSAR and the cumulative event frequency 
reported for the study with the severity and frequency reported in the applicable reference 
document. 
Reporting requirements will also be based on the investigator's assessment of causality and 
seriousness, with allowance for upgrading by Oscotec Inc. as needed. 

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6.4.5.7 
Assessment of Severity 
The severity, or intensity, of an AE refers to the extent to which an AE affects the subject’s 
daily activities. The intensity of the AE will be rated using the NCI CTCAE v5.0. 
Changes in the severity of an AE should be documented to allow an assessment of the 
duration of the event at each level of intensity to be performed. Adverse events characterized 
as intermittent do not require documentation of the onset and duration of each episode. 
6.4.5.8 
Assessment of Causality 
The investigator’s assessment of an AE’s relationship to study drug is part of the 
documentation process, but it is not a factor in determining what is or is not reported in the 
study. If there is any doubt as to whether a clinical observation is an AE, the event should be 
reported. The relationship or association of the study drug in causing or contributing to the 
AE will be characterized using the following classification and criteria: 
Unrelated: 
This relationship suggests that there is no association between the study drug 
and the reported event. 
Possible: 
This relationship suggests that treatment with the study drug caused or 
contributed to the AE, ie, the event follows a reasonable temporal sequence 
from the time of drug administration or follows a known response pattern to 
the study drug but could also have been produced by other factors. 
Probable: 
This relationship suggests that a reasonable temporal sequence of the event 
with drug administration exists and, based upon the known pharmacological 
action of the drug, known or previously reported adverse reactions to the drug 
or class of drugs, or judgment based on the investigator’s clinical experience, 
the association of the event with the study drug seems likely. The event 
disappears or decreases on cessation or reduction of the dose of study drug. 
Definite: 
This relationship suggests that a definite causal relationship exists between 
drug administration and the AE, and other conditions (concurrent illness, 
progression/expression of disease state, or reaction to concurrent medication) 
do not appear to explain the event. The event reappears or worsens if the 
study drug is re-administered. 
6.4.5.9 
Follow-Up of Subjects Reporting Adverse Events 
All AEs must be reported in detail on the appropriate page in the eCRF and followed to 
satisfactory resolution, until the investigator deems the event to be chronic or not clinically 
significant, or until the subject is considered to be stable. 

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6.5 
Pharmacokinetic and Pharmacodynamic Assessments 
6.5.1 Pharmacokinetic Blood Samples 
Serial blood samples (5 mL) for PK assessment of SKI-O-592 and its metabolites (M2 and 
M4) will be collected from all subjects in the PK/PD subset at the following time points:  
• Day 1: 0 hour (morning pre-dose), and at 0.25 (+5 min), 0.5 (±5min), 1, 2, 4, 8 and 
12 hours (±10 min) morning post-dose (12-hr sample should be collected prior to 
the evening dose). 
• Week 12 (Day 84 or 85): 0 hour (morning pre-dose), and 0.25 (+5 min), 0.5 
(±5min), 1, 2, 4, 8, and 12 hours (±10 min). 
In addition, PK samples will also be collected at the following time points on Day 8 
(±2 days) from subjects in the safety run-in period only: 
• Day 8 (±2 days): 0 hour (morning pre-dose), and at 0.25 (+5 min), 0.5 (±5min), 1, 
2, 4, 8 and 12 hours (±10 min) morning post-dose (12-hr sample should be 
collected prior to the evening dose). 
For other subjects not included in the safety run-in period or PK/PD subset, morning 
pre-dose PK samples will be collected during Week 12 only. 
When the PK blood sample collection coincides with safety assessments, PK blood samples 
should be collected as close to the scheduled time point as possible, and safety assessments 
should be performed before PK blood samples are taken. 
For each sample collected, separated plasma will be transferred in approximately equal 
portions into 2 tubes that are labeled for sets A and B for SKI-O-592 and metabolites. Labels 
will include the study number, the subject identification number, an indication that they are 
PK samples, the nominal day and collection time, the analyte(s), and the set (A or B). Plasma 
samples will be stored at approximately –70°C in an upright position. 
Set A samples will be transported, frozen with sufficient dry ice for several days, to the 
bioanalytical facility. 
Set B samples will be retained at the study center until the study is completed and the clinical 
study report is issued, unless shipment to another facility is requested by the Oscotec Inc. 

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Instructions regarding the disposition of the B samples will be provided by the Oscotec Inc. 
Specific details and the full protocol for analysis of PK samples can be found in the study 
laboratory manual. 
6.5.2 Pharmacokinetic Variables 
Plasma PK parameters will be calculated for SKI-O-592 and its metabolites M2 and M4 in 
the PK/PD subset using a noncompartmental approach and will include the following, where 
applicable:  
Cmax 
 
maximum observed concentration 
Tmax 
 
time to reach the maximum observed concentration 
AUC0-tau 
area under the concentration versus time curve within a dosing interval 
t1/2 
 
apparent terminal elimination half-life  
Kel 
 
terminal elimination rate constant 
CL/F  
apparent oral clearance (SKI-O-592 only)  
Vz/F 
 
apparent oral volume of distribution (SKI-O-592 only) 
Rmet 
 
metabolite ratio 
RAUC  
accumulation ratio based on AUC0-tau 
Additional PK parameters may be estimated, as deemed appropriate.  
6.5.3 Pharmacodynamic Blood Samples 
Serial blood samples for assessment of the percentage of activated CD63+ basophils in 
peripheral blood will be collected in all subjects in the PK/PD subset at the following times: 
• Day 1: 0 hour (morning pre-dose), and 0.25 (+5 min), 0.5 (±5 min), 1, 2, 4, 8 and 
12 hours (±10 min) morning post-dose (12-hr sample should be collected prior to 
the evening dose).  
• Day 84 or 85 (Week 12): 0 hour (morning pre-dose), and at 0.25 (+5 min), 0.5 
(±5 min), 1, 2, 4, 8, and 12 hours (±10 min). 
Blood samples must be dispatched to the processing laboratory as quickly as possible to 
ensure transport, cataloguing, and processing time is complete within the required timeframe 

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for the selected assay. All blood collection systems must be pyrogen free. Blood samples 
(0.5 mL) will be collected into tubes containing dipotassium ethylene diamine tetra acetic 
acid (K2 EDTA) anticoagulant. Tubes containing ethylene diamine tetra acetic acid or citric 
acid as anticoagulant are prohibited. Samples are to be stored at 2°C to 8°C prior to shipping 
and packed with ice pack for shipping. 
Specific details and the full protocol for analysis of PD samples can be found in the study 
laboratory manual. 
6.5.4 Pharmacodynamic Variables 
The primary PD endpoint will be the change in the percentage of activated CD63+ basophils 
in peripheral blood. The PD parameters will include the following:  
Emax   
 
maximum effect 
TEmax   
 
time to achieve maximum effect 
AUEC0-tau  
 
area under the effect versus time curve within a dosing interval 
Additional PD endpoints/parameters may be evaluated, as deemed appropriate. 
6.6 
Safety Monitoring Committee 
An independent DMC appointed by the Sponsor will review the interim safety and PK data 
of the first 5 subjects enrolled in the safety run-in period. Further details will be provided in 
the DMC charter. 
6.7 
Pregnancy 
Pregnancy is not regarded as an AE unless it is suspected that study drug interfered with the 
effectiveness of a contraceptive medication. Any pregnancy that occurs during study 
participation must be reported using a clinical study pregnancy form. To ensure subject 
safety, each pregnancy must be reported to the sponsor within 2 weeks of learning of its 
occurrence. The pregnancy must be followed up at least 30 days after birth or the first 
well-baby visit to determine the outcome (including spontaneous miscarriage, elective 
termination, normal birth, or congenital abnormality) and the status of mother and child, even 
if the subject was discontinued from the study. Pregnancy complications and elective 

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terminations for medical reasons must be reported as AEs or SAEs. Spontaneous 
miscarriages must be reported as SAEs. 
Any SAE occurring in association with a pregnancy that is brought to the investigator’s 
attention after the subject has completed the study and that is considered by the investigator 
as possibly related to the study drug must be promptly reported to the sponsor. 
Contraception status will be monitored at each visit and documented appropriately. 
6.8 
Laboratory Analyses 
Any abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other 
safety assessments (eg, ECGs, vital sign measurements), including those that worsen from 
baseline, felt to be clinically significant in the medical and scientific judgment of the 
investigator are to be recorded as AEs or SAEs. 
However, any clinically significant safety assessments that are associated with the underlying 
disease, unless judged by the investigator to be more severe than expected for the subject’s 
condition, are not to be reported as AEs or SAEs. 
6.9 
Sample Collections 
The total volume of blood collected for each assessment is discussed in each specific 
laboratory manual. 
6.10 Pharmacogenomic Screening 
A 6-mL whole blood sample for genotyping will be drawn at Baseline (Day 1, before dosing) 
from subjects who have consented to participate in the genetic analysis component of the 
study to potentially explore genetic polymorphisms of the enzymes (eg, UGT1A1) 
responsible for the metabolism of SKI-O-703 and the association of these genetic 
polymorphisms with the PK of SKI-O-703. Further details of blood collection and processing 
will be provided in the laboratory manual. 

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7 
Statistical and Analytical Plan 
7.1 
Primary Endpoint - Platelet Response 
• Patient platelet response is defined as platelet count ≥ 30,000/µL and doubling the 
baseline (average of 2 previous counts), at any visit during the treatment period and 
without the use of rescue medication 
7.2 
Secondary Endpoints 
7.2.1 
Subjects Achieving Two or More Pre-specified Platelet Counts 
• Subjects able to achieve 2 or more consecutive platelet counts of ≥ 30,000/µL separated 
by at least 5 days and without use of rescue medication 
• Subjects able to achieve 2 or more consecutive platelet counts of ≥ 30,000/µL with an 
increase of platelet count of ≥ 20,000/µL from baseline separated by at least 5 days and 
without use of rescue medication 
• Subjects able to achieve 2 or more consecutive platelet counts of ≥ 50,000/µL separated 
by at least 5 days and without use of rescue medication 
• Subjects able to achieve 2 or more consecutive platelet counts of ≥ 100,000/µL during the 
treatment period, separated by at least 5 days and without use of rescue medication 
• Subjects able to achieve platelet counts of ≥ 50,000/µL at 3 of the last 4 visits. 
7.2.2 
Change from Baseline in Platelet Counts 
• Change from baseline in the average of the last 2 platelet counts during the treatment 
period 
For each subject, the change from baseline in the mean of the last 2 platelet counts during the 
treatment period will be calculated. For those subjects with only 1 platelet count during 
treatment period, the change from baseline in the single platelet count will be used. 
7.2.3 
Time to First Platelet Response 
• Time to first platelet response (>30,000/µL and 2 x baseline) 
For each subject, the time of the first platelet response is the first visit during treatment 
period in which platelet count ≥ 30,000/µL without use of rescue medication. 

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7.2.4 
Others 
• Number of subjects receiving rescue medication at least once during treatment period 
• Subjects with at least one Grade 2 or higher non-skin bleeding event during treatment 
period. 
For each endpoint, a subject will be considered to have had an event if the event was 
observed at least once during the treatment period for rescue medication, and during 
treatment or follow-up period for bleeding event. 
7.3 
Exploratory Endpoints 
7.3.1 
Peak Platelet Count 
• Peak platelet counts during the treatment period without use of rescue medication 
For each subject, peak platelet count is subject’s highest platelet count during the treatment 
period, without use of rescue medication. 
7.3.2 
Bleeding Score 
The ITP bleeding assessment score as described by Page et al. 2007 will be used to 
characterize bleeding tendency. 
Table 3 
The ITP Bleeding Score Assessment 
Site 
Bleeding Grade 
0 
1 
2 
Skin [physical 
Examination (PE)] 
None 
1–5 bruises and/or scattered petechiae 
>5 bruises with size >2 cm and/or 
diffuse petechiae 
Oral (PE) 
None 
1 blood blister or >5 petechiae or gum 
bleeding that clears easily with rinsing 
Multiple blood blisters and/or gum 
bleeding 
Skin (Hx) 
None 
1–5 bruises and/or scattered petechiae 
>5 bruises with size >2 cm and/or 
diffuse petechiae 
Oral (Hx) 
None 
1 blood blister or >5 petechiae and/or 
gum bleeding <5 min 
Multiple blood blisters and/or gum 
bleeding >5 min 
Epistaxis 
None 
Blood when blowing nose and/or 
epistaxis <5 min (per episode) 
Bleeding >5 min (per episode) 
Gastrointestinal 
None 
Occult blood 
Gross blood 
Urinary 
None 
Microscopic (+ve dipstick) 
Macroscopic 

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Site 
Bleeding Grade 
0 
1 
2 
Gynecological 
None 
(normal 
period) 
Spotting not at time of normal period 
Bleeding >spotting not at time of 
period or very heavy period 
Pulmonary 
None 
N/A 
Yes 
Intracranial 
haemorrhage 
None 
N/A 
Yes 
Subconjunctival 
haemorrhage 
None 
Yes 
N/A 
Abbreviations: ITP, immune thrombocytopenia; PE, physical examination; N/A, not applicable 
Note: In case of a breakthrough bleeding, the subjects will discontinue the trial. 
7.3.3 
Quality of Life as Measured by the SF-36 Score 
The Short Form (36) Health Survey is a 36-item, patient-reported survey of patient health. 
The SF-36 is a measure of health status and the survey will be conducted according to the 
SF-36 quality of life questionnaire Version 2.0. 
The assessments will be performed at time points indicated in the schedule of events 
(Table 2). 
7.4 
Sample Size Calculations 
A minimum of 60 subjects will be enrolled in the study. Subjects will receive either 200 mg 
BID or 400 mg BID of SKI-O-703 or placebo in a 2:2:1 ratio. 
Based on Podolanczuk et al (2009) at least 50% of subjects in the active treatment group will 
be responders (R). A sample size of 24 subjects per active treatment group and 12 subjects in 
the placebo group will provide approximately 82.7% power to detect a difference between 
response rates of 50% and 1% in the active treatment and placebo groups, respectively, at the 
2-sided 5% significance level. The test statistic used in the sample size calculation was the 
2-sided Fisher's Exact test using the normal approximation method. 
Subjects that do not receive the study drug or do not provide at least 1 post baseline 
measurement will be excluded from the analysis above and replaced. 
There will be no adjustment for multiple comparisons. The sample size calculation was 
performed using PASS 15 Power Analysis and Sample Size Software (2017). NCSS, LLC. 
Kaysville, Utah, USA, ncss.com/software/pass. 

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7.5 
Analysis Sets 
The following analysis sets will be used in the statistical analyses. 
Safety set: The safety set will consist of all subjects who receive at least 1 dose of study drug 
(SKI-O-703 or placebo). Subjects will be analyzed according to the treatment they received. 
This analysis set will be used in the analysis of demographic and safety data. 
Intent-to-Treat (ITT) set: The ITT set will consist of all randomized subjects who receive at 
least 1 dose of study drug (SKI-O-703 or placebo) and have at least 1 post-baseline platelet 
assessments. Subjects will be analyzed according to the treatment arm to which they were 
randomized. This analysis set will be used for efficacy summaries, including the analysis of 
the primary endpoint. 
Per-protocol (PP) set: The PP set will consist of all subjects in ITT set with no major protocol 
violations. Major protocol violations will be identified during the study. Subjects will be 
analyzed according to the treatment arm to which they were randomized. This analysis set 
will be used for efficacy summaries, including the analysis of the primary endpoint. 
Pharmacokinetic (PK) set: The PK set will consist of all subjects who receive SKI-O-703 and 
have at least 1 measurable plasma concentration. Subjects who have partial data and/or major 
protocol deviations that may impact PK, and subjects who experience emesis, will be 
evaluated on a case-by-case basis and may be excluded from the PK analysis set. This 
analysis set will be used for summaries of PK data. 
Pharmacodynamic (PD) set: The PD set will consist of all subjects who receive at least 
1 dose of study drug (SKI-O-703 or placebo) and have at least 1 evaluable postdose PD 
value. Subjects who have partial data and/or major protocol deviations that may impact PD 
will be evaluated on a case-by-case basis and may be excluded from the PD analysis set. This 
analysis set will be used for summaries of PD data. 
Pharmacokinetic (PK)/pharmacodynamic (PD) set: All subjects included in the PK and PD 
analysis sets. This analysis set will be used for the PK/PD analyses. 

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7.6 
Description of Subgroups to be Analyzed 
The primary endpoint will be summarized using the following subgroups of interest: 
• Number of previous lines of therapy (0-2 vs. ≥3) 
• Response to previous treatment (non-responders vs. relapsed) 
• Splenectomized subjects vs. non-splenectomized subjects and  
• Subjects with baseline platelet count < 15,000/µL versus subjects with platelet count 
between 15,000/µL to 30,000/µL. 
• Subjects with prior TPO-receptor agonist use versus subjects without prior TPO-receptor 
agonist use. 
7.7 
Statistical Analysis Methodology 
Statistical analysis will be performed using SAS software Version 9.4 or later. The results for 
each efficacy endpoint will be summarized by visit and treatment for all subjects within the 
ITT analysis set using descriptive statistical methods. Continuous variables (quantitative 
endpoints) will be summarized using the mean, the standard deviation (SD), median, range 
(minimum value and maximum value), and interquartile. Categorical variables (qualitative 
endpoints) will be summarized using frequency counts (number) and percentages. Data will 
be listed in data listings. 
Details of the statistical analyses, methods, and data conventions will be described in the 
statistical analysis plan. 
All statistical tests will be 2-sided using a 5% significance levels, leading to 95% (2-sided) 
confidence intervals (CIs). This will be used for each pairwise comparison with placebo, ie, 
there will be no adjustments for multiplicity since this is a Phase 2 study. 
7.7.1 Subject Disposition 
Subject disposition will be summarized by treatment group and will include subjects that 
enrolled, were randomly assigned, discontinued, and completed the study. For discontinued 
subjects, the reasons for discontinuation will be summarized. 

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7.7.2 Analyses of Demographic and Baseline Characteristics 
Demographic and baseline characteristics such as age, gender, race, body weight, height, 
body mass index, and medical history will be summarized by treatment group using 
descriptive statistics. 
7.7.3 
Analysis of Platelet Response 
The primary efficacy endpoint will be summarized as overall platelet response rate (ORR) by 
treatment group and analyzed using Fisher’s exact test to compare the difference in ORR 
between each active treatment group and placebo. A logistic regression model will also be 
performed on platelet response and include treatment, baseline platelet count and prior 
TPO-receptor agonist use, provided there are enough subjects in each subgroup. The odds 
ratio for the treatment effect compared to placebo (together with 95% CI and p-value) will be 
provided. 
Summary statistics will be provided on the number (%) of subjects achieving platelet 
response by each treatment group and the combined active treatment group. Fisher’s exact 
test on the differences in the ORR will therefore be provided for: 
• Each active treatment group (200 mg BID, 400 mg BID) vs. placebo 
• Combine active treatment groups vs. placebo 
• 200 mg BID vs. 400 mg BID. 
Furthermore, summary statistics will be provided on the number (%) of subjects achieving 
platelet response by each treatment group and the subgroups defined in Section 7.6. 
A subject will be classified as a platelet responder if subject shows at least 1 platelet count 
≥ 30,000/µL and doubling the baseline (average of 2 previous counts), at any visit during 
treatment period, without use of rescue medication. The ITT analysis set will be used in the 
analysis of the primary endpoint. 
7.7.4 
Analysis of Secondary Endpoints 
For each binary endpoint (ie, definition of subject achieving 2 or more consecutive platelet 
counts), a logistic regression model will be performed and include treatment, baseline platelet 
count and prior TPO-receptor agonist use, provided there are enough subjects in each 

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subgroup. The odds ratio for the treatment effect compared to placebo (together with 95% CI 
and p-value) will be provided. 
Time to platelet response will be estimated using the Kaplan-Meier method. Each active 
treatment group curve will be compared against placebo curve using log-rank test. In 
addition, a proportional hazard model will be implemented as well with treatment, baseline 
platelet count, and prior TPO-receptor agonist use. 
Change from baseline in the average of the last 2 platelet counts will be analyzed using an 
analysis of covariance with baseline platelets count as covariate, treatment, and prior 
TPO-receptor agonist use. Each active treatment group will be compared with placebo. 
Summary statistics of the following subjects will be provided by each treatment group: 
• Subjects receiving rescue medication during the treatment period 
• Subjects with Grade 2 or higher non-skin bleeding event during the treatment or 
follow-up period 
A subject will be considered to have an event if the event was observed at least once during 
the treatment period or follow-up period as mentioned above. 
7.7.5 
Analyses of Exploratory Endpoints 
Peak platelet will be summarized by treatment groups. The median peak platelet, and the 
lower and upper quartile will be provided by treatment group. The difference in median peak 
platelet will be calculated for: 
• Each active treatment group (200 mg BID, 400 mg BID) vs. placebo 
• Combined active treatment groups vs. placebo  
• 200 mg BID vs. 400 mg BID 
Bleeding score will be summarized by treatment group and the combined active treatment 
groups. 

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7.7.6 
Pharmacokinetic/Pharmacodynamic Analyses 
7.7.6.1 
Pharmacokinetic Analyses 
Plasma concentrations of SKI-O-592 and its metabolites (M2 and M4) will be listed and 
summarized separately for each treatment and day at each nominal time point using 
descriptive statistics (number of observations [n], mean, SD, coefficient of variation [CV], 
minimum, median, and maximum). Individual and mean plasma concentration versus time 
profiles will be plotted for each treatment and day. 
PK parameters for SKI-O-592 and its metabolites will be calculated using noncompartmental 
methods. Descriptive statistics (n, geometric mean, geometric CV, mean, SD, CV, minimum, 
median, and maximum) for the PK parameters for SKI-O-592 and its metabolites will be 
presented for each treatment and day. Treatment comparisons will be performed as deemed 
appropriate. 
7.7.6.2 
Pharmacodynamic Analyses 
Absolute and percentage change from baseline in the percentage of activated CD63+ 
basophils in peripheral blood will be listed and summarized separately for each treatment and 
day at each nominal time point using descriptive statistics (number of observations [n], mean, 
SD, CV, minimum, median, and maximum). Individual, mean absolute and percentage 
change from baseline in the percentage of activated CD63+ basophils will be plotted versus 
time for each treatment and day. 
PD parameters for the percentage of activated CD63+ basophils will be calculated using 
non-compartmental methods. Descriptive statistics (n, mean, SD, CV, minimum, median, and 
maximum) for the PD parameters will be presented for each treatment and day. Treatment 
comparisons will be performed as deemed appropriate. 
7.7.6.3 
Exploratory PK/PD Analyses 
PK/PD relationship between plasma concentrations of SKI-O-592, M2, and M4 and change 
from baseline in the percentage of activated CD63+ basophils in peripheral blood on Day 1 
and Week 12 will be assessed. Individual and mean plasma concentrations of SKI-O-592, 
M2, and M4 will be plotted versus change from baseline in the percentage of activated 
CD63+ basophils in peripheral blood on Day 1 and Week 12. 

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Exploratory PK/PD modeling may also be performed; full details will be provided in a 
separate Statistical Analysis Plan. 
7.7.7 
Safety Analyses 
Continuous parameters (such as laboratory measurements, ECG results, and vital signs) will 
be summarized separately for each treatment at each nominal time point using descriptive 
statistics (mean, SD, minimum, median, and maximum). 
Discrete parameters (such as deaths, SAEs, AEs, concomitant medications, and physical 
examination results) will be summarized separately for each treatment at each nominal time 
point using frequency counts and percentages. All AEs, SAEs, drug-related AEs, and drug-
related SAEs will be summarized using the worst grade per NCI CTCAE v5.0 by system 
organ class and preferred term. All safety data will be listed. 
Safety results will be summarized using the safety set by treatment groups and the combined 
active treatment group, unless otherwise specified. 
7.7.8 
Interim Analyses 
An unblinded interim safety and PK data review will be performed by the DMC once the 
subjects in the safety run-in period complete their Day 8 (±2 days) assessments. Subjects in 
the safety run-in period will continue to be dosed whilst the interim analysis is being 
conducted. However, further enrollment in each treatment group will only resume after safety 
and PK data of the first week for the first 5 subjects are reviewed and considered satisfactory 
by the DMC. 

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8 
Data Quality Assurance 
This study will be conducted according to the ICH E6(R2) risk and quality processes 
described in the applicable procedural documents. The quality management approach to be 
implemented in this study will be documented and will comply with the current ICH 
guidance on quality and risk management (ICH 2005). 
8.1 
Data Management 
As part of the responsibilities assumed by participating in the study, the investigator agrees to 
maintain adequate case histories for the subjects treated as part of the research under this 
protocol. The investigator agrees to maintain accurate eCRFs and source documentation as 
part of the case histories. These source documents may include laboratory reports and ECG 
strips. 
Investigative site personnel will enter subject data into Oracle™Clinical Remote Data 
Capture. The analysis data sets will be a combination of these data and data from other 
sources (eg, laboratory data). 
Clinical data management will be performed in accordance with applicable sponsor standards 
and data cleaning procedures to ensure the integrity of the data, eg, removing errors and 
inconsistencies in the data. Adverse events and concomitant medication terms will be coded 
using MedDRA Version 20.0 or later, an internal validated medication dictionary. 
After database lock, each study site will receive a CD-ROM containing all of their site 
specific eCRF data as entered into Oracle Clinical Remote Data Capture for the study, 
including full discrepancy and audit history. Additionally, a CD-ROM copy of all of the study 
site’s data from the study will be created and sent to the sponsor for storage. PPD will 
maintain a duplicate CD-ROM copy for their records. In all cases, subject initials will not be 
collected or transmitted to the sponsor. 

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9 
Ethics 
9.1 
Independent Ethics Committee or Institutional Review Board 
Federal regulations and the ICH guidelines require that approval be obtained from an 
IRB/IEC before participation of human subjects in research studies. Before study onset, the 
protocol, informed consent, advertisements to be used for the recruitment of study subjects, 
and any other written information regarding this study to be provided to the subject or the 
subject’s legal guardian must be approved by the IRB/IEC. Documentation of all IRB/IEC 
approvals and of the IRB/IEC compliance with ICH harmonised tripartite guideline E6(R2): 
Good Clinical Practice (GCP) will be maintained by the site and will be available for review 
by the sponsor or its designee. 
All IRB/IEC approvals should be signed by the IRB/IEC chairman or designee and must 
identify the IRB/IEC name and address, the clinical protocol by title or protocol number or 
both, and the date approval or a favorable opinion was granted. 
The investigator is responsible for providing written summaries of the progress and status of 
the study at intervals not exceeding 1 year or otherwise specified by the IRB/IEC. The 
investigator must promptly supply the sponsor or its designee, the IRB/IEC, and, where 
applicable, the institution, with written reports on any changes significantly affecting the 
conduct of the study or increasing the risk to subjects. 
9.2 
Ethical Conduct of the Study 
The study will be performed in accordance with the ethical principles that have their origin in 
the Declaration of Helsinki, ICH GCP, the protocol, and all applicable regulations. 
9.3 
Subject Information and Consent 
A written informed consent in compliance with the respective applicable regulatory authority 
regulations shall be obtained from each subject before entering the study or performing any 
unusual or nonroutine procedure that involves risk to the subject. An informed consent 
template may be provided by the sponsor to investigative sites. If any institution-specific 
modifications to study-related procedures are proposed or made by the site, the consent 
should be reviewed by the sponsor or its designee or both before IRB/IEC submission. Once 
reviewed, the consent will be submitted by the investigator to his or her IRB/IEC for review 

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and approval before the start of the study. If the ICF is revised during the course of the study, 
all active participating subjects must sign the revised form. 
Before recruitment and enrollment, each prospective subject or his or her legal guardian will 
be given a full explanation of the study and be allowed to read the approved ICF. Once the 
investigator is assured that the subject/legal guardian understands the implications of 
participating in the study, the subject/legal guardian will be asked to give consent to 
participate in the study by signing the ICF. 
The investigator shall retain the signed original ICF(s) and give a copy of the signed original 
form to the subject or legal guardian. 

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10 Investigator’s Obligations 
The following administrative items are meant to guide the investigator in the conduct of the 
study but may be changed based on industry and government standard operating procedures, 
working practice documents, or guidelines. Changes will be reported to the IRB/IEC but will 
not result in protocol amendments. 
10.1 Confidentiality 
All laboratory specimens, evaluation forms, reports, and other records will be identified in a 
manner designed to maintain subject confidentiality. All records will be kept in a secure 
storage area with limited access. Clinical information will not be released without the written 
permission of the subject (or the subject’s legal guardian), except as necessary for monitoring 
and auditing by the sponsor, its designee, the US FDA, or the IRB/IEC. 
The investigator and all employees and coworkers involved with this study may not disclose 
or use for any purpose other than performance of the study any data, record, or other 
unpublished, confidential information disclosed to those individuals for the purpose of the 
study. Prior written agreement from the sponsor or its designee must be obtained for the 
disclosure of any said confidential information to other parties. 
10.2 Financial Disclosure and Obligations 
Investigators are required to provide financial disclosure information to allow the sponsor to 
submit the complete and accurate certification or disclosure statements required under 
21 Code of Federal Regulations (CFR) 54. In addition, the investigator must provide to the 
sponsor a commitment to promptly update this information if any relevant changes occur 
during the course of the investigation and for 1 year following the completion of the study. 
Neither the sponsor nor PPD is financially responsible for further testing or treatment of any 
medical condition that may be detected during the screening process. In addition, in the 
absence of specific arrangements, neither the sponsor nor PPD is financially responsible for 
further treatment of the subject’s disease. 

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10.3 Investigator Documentation 
Prior to beginning the study, the investigator will be asked to comply with ICH E6(R2) 8.2 
and Title 21 of the CFR by providing the following essential documents, including but not 
limited to: 
• IRB/IEC approval 
• Original investigator-signed investigator agreement page of the protocol 
• Form FDA 1572, fully executed, and all updates on a new fully executed Form 
FDA 1572 
• Curriculum vitae for the investigator and each subinvestigator listed on Form FDA 1572 
• Financial disclosure information to allow the sponsor to submit complete and accurate 
certification or disclosure statements required under 21 CFR 54. In addition, the 
investigators must provide to the sponsor a commitment to promptly update this 
information if any relevant changes occur during the course of the investigation and for 
1 year after the completion of the study. 
• IRB/IEC-approved informed consent, samples of site advertisements for recruitment for 
this study, and any other written information regarding this study that is to be provided to 
the subject or legal guardian, and 
• Laboratory certifications and normal ranges for any local laboratories used by the site, in 
accordance with 42 CFR 493. 
10.4 Study Conduct 
The investigator agrees that the study will be conducted according to the principles of ICH 
E6(R2). The investigator will conduct all aspects of this study in accordance with all 
national, state, and local laws or regulations. Study information from this protocol will be 
posted on publicly available clinical trial registers before enrollment of subjects begins. 
10.5 Adherence to Protocol 
The investigator agrees to conduct the study as outlined in this protocol in accordance with 
ICH E6(R2) and all applicable guidelines and regulations. 

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10.6 Adverse Events and Study Report Requirements 
By participating in this study, the investigator agrees to submit reports of SAEs according to 
the timeline and method outlined in the protocol. In addition, the investigator agrees to 
submit annual reports to the study site IRB/IEC as appropriate. 
10.7 Investigator’s Final Report 
Upon completion of the study, the investigator, where applicable, should inform the 
institution; the investigator/institution should provide the IRB/IEC with a summary of the 
study’s outcome and the sponsor and regulatory authority(ies) with any reports required. 
10.8 Records Retention 
Essential documents should 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. These documents 
should be retained for a longer period; however, if required by the applicable regulatory 
requirements or by an agreement with the sponsor. It is the responsibility of the sponsor to 
inform the investigator/institution as to when these documents no longer need to be retained. 
10.9 Publications 
After completion of the study, the data may be considered for reporting at a scientific 
meeting or for publication in a scientific journal. In these cases, the sponsor will be 
responsible for these activities and will work with the investigators to determine how the 
manuscript is written and edited, the number and order of authors, the publication to which it 
will be submitted, and other related issues. The sponsor has final approval authority over all 
such issues. 
Data are the property of the sponsor and cannot be published without prior authorization 
from the sponsor, but data and publication thereof will not be unduly withheld. 

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11 Study Management 
11.1 Monitoring 
11.1.1 
External Data Monitoring Committee 
An independent DMC appointed by the Sponsor will review the interim safety and PK data 
of the first 5 subjects enrolled in the safety run-in period (See Section 7.7.8). Further details 
will be provided in the DMC charter. 
11.1.2 
Monitoring of the Study 
The clinical monitor, as a representative of the sponsor, has the obligation to follow the study 
closely. In doing so, the monitor will visit the investigator and study site 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 
personnel. 
All aspects of the study will be carefully monitored, by the sponsor or its designee, for 
compliance with applicable government regulation with respect to current GCP and current 
standard operating procedures. 
11.1.3 
Inspection of Records 
Investigators and institutions involved in the study will permit study-related monitoring, 
audits, IRB/IEC review, and regulatory inspections by providing direct access to all study 
records. In the event of an audit, the investigator agrees to allow the sponsor, representatives 
of the sponsor, or a regulatory agency (eg, FDA) access to all study records. 
The investigator should promptly notify the sponsor and PPD of any audits scheduled by any 
regulatory authorities and promptly forward copies of any audit reports received to the 
sponsor. 

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11.2 Management of Protocol Amendments and Deviations 
11.2.1 
Modification of the Protocol 
Any changes in this research activity, except those necessary to remove an apparent, 
immediate hazard to the subject, must be reviewed and approved by the sponsor or its 
designee. Amendments to the protocol must be submitted in writing to the investigator’s 
IRB/IEC for approval before subjects can be enrolled into an amended protocol. 
11.2.2 
Protocol Deviations 
The investigator or designee must document and explain in the subject’s source 
documentation any deviation from the approved protocol. The investigator may implement a 
deviation from, or a change of, the protocol to eliminate an immediate hazard to study 
subjects without prior IRB/IEC approval. As soon as possible after such an occurrence, the 
implemented deviation or change, the reasons for it, and any proposed protocol amendments 
should be submitted to the IRB/IEC for review and approval, to the sponsor for agreement, 
and to the regulatory authorities, if required. 
A deviation from the protocol is an unintended or unanticipated departure from the 
procedures or processes approved by the sponsor and the IRB/IEC and agreed to by the 
investigator. A significant deviation occurs when there is nonadherence to the protocol by the 
subject or investigator that results in a significant, additional risk to the subject. Significant 
deviations can include nonadherence to inclusion or exclusion criteria, or nonadherence to 
FDA regulations or ICH GCP guidelines, and will lead to the subject being withdrawn from 
the study (Section 4.2). 
Protocol deviations will be documented by the clinical monitor throughout the course of 
monitoring visits. Principal investigators will be notified in writing by the monitor of 
deviations. The IRB/IEC should be notified of all protocol deviations in a timely manner. 

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11.3 Study Termination 
Although Oscotec Inc. has every intention of completing the study, Oscotec Inc. reserves the 
right to discontinue the study at any time for clinical or administrative reasons which may 
include (but not limited to) the followings: 
• Unacceptable risk to a subject participating in the study 
• The decision on the Sponsor to suspend or discontinue evaluation of the study drug 
• Insufficient adherence to protocol  
• Failure to comply with regulation, etc. 
The end of the study is defined as the date on which the last subject completes the last visit 
(includes follow-up visit). 
11.4 Final Report 
Whether the study is completed or prematurely terminated, the sponsor will ensure that the 
clinical study reports are prepared and provided to the regulatory agencies as required by the 
applicable regulatory requirement(s). The sponsor will also ensure that the clinical study 
reports in marketing applications meet the standards of the ICH harmonised tripartite 
guideline E3: Structure and content of clinical study reports. 
Where required by applicable regulatory requirements, an investigator signatory will be 
identified for the approval of the clinical study report. The investigator will be provided 
reasonable access to statistical tables, figures, and relevant reports and will have the 
opportunity to review the complete study results. 
Upon completion of the clinical study report, Oscotec Inc. will provide the investigator with 
the full summary of the study results. The investigator is encouraged to share the summary 
results with the study subjects, as appropriate. The study results will be posted on publicly 
available clinical trial registers. 

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12 Reference List 
Department of Health and Human Services (DHHS), Food and Drug Administration, Center 
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Genosco Inc. (Cambridge, Massachusetts, United States). In vivo efficacy of SKI-O-703 in 
anti-CD41 antibody induced murine ITP (Immune Thrombocytopenic Purpura) model. 
2017 Jul 18. Final study report. Study No.: GNS-SYK-1306. 39 p. 
ICH Steering Committee. Quality risk management Q9: ICH harmonised tripartite guideline. 
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