A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Phase: PHASE2
Conditions: Duchenne Muscular Dystrophy
Sponsor: NS Pharma, Inc.

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 1 of 112 
Clinical Study Protocol: NS-065/NCNP-01-202 
Study Title: 
A Phase II, Open-Label, Extension Study to Assess the Safety 
and Efficacy of NS-065/NCNP-01 in Boys with Duchenne 
Muscular Dystrophy (DMD)  
Protocol Number: 
NS-065/NCNP-01-202 
Study Phase: 
Phase II 
Product Name: 
NS-065/NCNP-01 Injection 
IND Number: 
127,474 
Investigators: 
Up to 6 clinical sites located in North America 
Sponsor: 
NS Pharma, Inc. 
140 E Ridgewood Avenue, Suite 280S 
Paramus, NJ 07652 
Study Chair: 
Paula R. Clemens, MD 
Professor of Neurology 
University of Pittsburgh 
Sponsor Medical 
Monitor: 
Helmut Albrecht, MD, MS, FFPM 
PharmaLex Development Services, LLC 
Original Protocol Date: 
17Feb2017 
Amendment 1 Date: 
21Apr2017 
Amendment 2 Date: 
08Sep2017 
Amendment 3 Date: 
13Nov2017 
Amendment 4 Date:  
08Dec2017 
Amendment 5 Date: 
02Feb2018 
Amendment 5.1 Date:  
01Mar2018 
Amendment 6.0 Date: 
10Apr2018 
Amendment 7.0 Date 
Amendment 8.0 Date: 
04Jun2018 
19Nov2019 
Amendment 9.0 Date: 
26Feb2020 
Amendment 10.0 Date: 
21Jul2020 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 2 of 112 
 
Confidentiality Statement 
This protocol contains confidential, proprietary information which is the property of NS Pharma, Inc. 
No information contained herein may be published or disclosed without written approval from NS 
Pharma, Inc. 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 3 of 112 
STUDY SYNOPSIS 
Study Title 
A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy 
of NS-065/NCNP-01 in Boys with Duchenne Muscular Dystrophy (DMD) 
Protocol Number NS-065/NCNP-01-202 
Name of Sponsor NS Pharma, Inc. 
Investigative 
Product 
NS-065/NCNP-01 (viltolarsen) Injection 
Study Phase 
Phase II 
Indication 
Treatment of Duchenne muscular dystrophy (DMD) 
Objectives 
Primary:  
1. To evaluate the safety and tolerability of low (40 mg/kg/week) and high 
(80 mg/kg/week) intravenous (IV) doses of NS-065/NCNP-01 Injection 
up to 192-week treatment period (up to a total of 216 weeks of treatment 
in Study NS-065/NCNP-01-201 and 202), in boys with DMD that 
completed Study NS-065/NCNP-01-201.  
2. To investigate the effect of low and high IV doses of NS-065/NCNP-01 
Injection after up to 192-week Treatment Period (up to a total of 216 
weeks of treatment in Study NS-065/NCNP-01-201 and 202) on muscle 
strength, mobility, and functional exercise capacity, as measured by 
Time to Stand (TTSTAND) in boys with DMD that completed Study 
NS-065/NCNP-01-201 vs. matched DMD historical controls in boys 
with DMD.  
Secondary:  
1. To investigate the effect of low and high IV doses of NS-065/NCNP-01 
Injection after up to 192-weeks treatment (up to a total of 216 weeks of 
treatment in Study NS-065/NCNP-01-201 and 202) on muscle strength, 
mobility, and functional exercise capacity, as measured by Time to 
Run/Walk 10 meters (TTRW), Time to Climb 4 stairs (TTCLIMB), 
North Star Ambulatory Assessment (NSAA), Six-minute Walk Test 
(6MWT), and Quantitative Muscle Testing (QMT) in boys with DMD 
that completed Study NS-065/NCNP-01-201 vs. matched historical 
controls in boys with DMD.  
Exploratory:  
1. To investigate the effects of low and high IV doses of NS-065/NCNP-01 
Injection on serum pharmacodynamic (PD) biomarkers.  
Study Design 
This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-
01 Injection 250 mg administered intravenously once weekly for up to 192-
weeks (up to a total of 216 weeks of treatment in Study NS-065/NCNP-01-
201 and 202) to boys with DMD who complete Study NS-065/NCNP-01-201. 
This study will evaluate the safety, tolerability, and clinical efficacy of NS-

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 4 of 112 
065/NCNP-01 Injection 250 mg at dose levels of up to 80 mg/kg/week 
administered by weekly IV infusion over an additional treatment period of up 
to 192-weeks (up to a total of 216 weeks of treatment in Study NS-
065/NCNP-01-201 and 202) or until enrollment in a separate long-term 
follow up program of NS-065/NCNP-01, whichever is earlier. Week 193 is a 
comprehensive assessment at the beginning of the extension visits 
incorporated in protocol amendment 10, and Week 217 is the final study 
assessment for the treatment period ending with Week 216.  
Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-
201 are eligible to enroll.  Patients interested in enrolling in this extension 
study will be consented prior to or during the Week 24 study visit of the NS-
065/NCNP-01-201 study. In order to accommodate patient recovery 
following the scheduled biopsy during the Week 25 of Study NS-065/NCNP-
01-201, patients will be allowed a visit window of 7 days (+/- 3 days) from 
the Week 24 infusion to complete both the end of study procedures for study 
201 and the first infusion in this extension study. All Week 25 study 
assessments, including muscle biopsy, of the NS-065/NCNP-01-201 study 
must be completed prior to first infusion in this extension study.  Patients can 
receive the first infusion of this extension study on the same day as the Week 
25 study (final) visit of the NS-065/NCNP-01-201 study, as long as all study 
assessments (including muscle biopsy) of the NS-065/NCNP-01-201 study 
are completed prior to infusion.   
Study Population Inclusion Criteria:  
1. Patient’s parent or legal guardian has provided written informed 
consent/HIPAA authorization prior to any extension study-specific 
procedures and patient has provided assent appropriate for his age and 
developmental status.  
2. Patient completed Study NS-065/NCNP-01-201 through Week 25.  
3. Patient and parent/guardian are willing and able to comply with 
scheduled visits, investigational product administration plan, and study 
procedures.  
4. Patient must be on a stable dose of glucocorticoid (GC), and is expected 
to remain on the stable dose of GC treatment for the duration of the 
study. 
Exclusion Criteria:  
1. Patient had a serious or severe adverse event in Study NS-065/NCNP-
01-201 that, in the opinion of the Investigator and/or Sponsor, was 
probably or definitely related to NS-065/NCNP-01 Injection 250 mg use 
and precludes safe use of NS-065/NCNP-01 Injection 250 mg for the 
patient in this study. 
2. Patient had a treatment which was made for the purpose of dystrophin or 
dystrophin-related protein induction after completion of Study NS-
065/NCNP-01-201. 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 5 of 112 
3. Patient took any other investigational drugs after completion of Study 
NS-065/NCNP-01-201.  
4. Patient was judged by the investigator and/or the Sponsor that it was not 
appropriate to participate in the extension study for other reasons. 
Clinical Sites 
Up to 6 clinical sites located in North America 
Test Product, 
Dose, and Mode 
of 
Administration 
NS-065/NCNP-01 Injection 250 mg aqueous infusions will be supplied as a 
10-mL or 5-mL glass vial containing 25 mg/mL or 50 mg/mL of drug 
product in saline, respectively. 
Patients will begin dosing in this study at the same dose level they received 
in Study NS-065/NCNP-01-201. Once sites receive IRB approval of 
Protocol Amendment 10, all subjects who were previously in the low dose 
cohort of 40mg/kg/week will receive 80mg/kg/week after obtaining patient 
consent/assent. Patients will continue at dose level of up to 80 mg/kg/week 
for the remaining duration of the trial, up to 192-week (up to a total of 216 
weeks of treatment in Study NS-065/NCNP-01-201 and 202) extension 
study or enrollment in a separate long-term follow up program of NS-
065/NCNP-01, whichever is earlier.  
Investigational product will be prepared in accordance with an 
Investigational Product Instruction Manual (IPIM) and administered by 
intravenous infusion over a 1-hour period.  
Safety 
Assessments 
1. Overall incidence of adverse events (AEs) and serious adverse events 
(SAEs) 
2. Changes from baseline compared to Screening Visit and Week 25 of 
Study NS-065/NCNP-01-201 in laboratory parameters (blood, urine). 
3. Changes from baseline compared to Day 1 and Week 25 of Study NS-
065/NCNP-01-201 in electrocardiograms (ECGs), anthropometrics, 
vital signs, and diagnostic parameters (physical exam). 
4. Anti-NS-065/NCNP-01 antibodies at Weeks 37, 49, 73, 97, 121, 145, 
169,193 and 217 compared to Day 1 and Week 24 of Study NS-
065/NCNP-01-201. 
5. Anti-dystrophin antibodies at Weeks 37, 49, 73, 97, 121, 145, 169, 193 
and 217 compared to Day 1 and Week 24 of Study NS-065/NCNP-01-
201. 
6. Cytokine levels at Weeks 37, 49, 73, and 97 compared to Day 1 and 
Week 24 of Study NS-065/NCNP-01-201. 
Efficacy 
Assessments 
Primary: 
Time to Stand (TTSTAND) at 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 
157,169, 181, 193, 205 and 217 Weeks compared to Pre-Infusion Visit and 
Week 25 of Study NS-065/NCNP-01-201. 
Secondary: 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 6 of 112 
1. Time to Run/Walk 10 meters (TTRW) at 37, 49, 61, 73, 85, 97, 109, 
121, 133, 145, 157, 169, 181, 193, 205 and 217 Weeks compared to 
Pre-Infusion Visit and Week 25 of Study NS-065/NCNP-01-201 
2. Time to Climb 4 stairs (TTCLIMB) at 37, 49, 61, 73, 85, 97, 109, 121, 
133, 145, 157, 169, 181, 193, 205 and 217 Weeks compared to Pre-
Infusion Visit and Week 25 of Study NS-065/NCNP-01-201 
3. North Star Ambulatory Assessment (NSAA) at 37, 49, 61, 73, 85, 97, 
109, 121, 133, 145, 157, 169, 181, 193, 205 and 217 Weeks compared 
to Pre-Infusion Visit and Week 25 of Study NS-065/NCNP-01-201 
4. Six-minute Walk Test (6MWT) at 37, 49, 61, 73, 85, 97, 109, 121, 133, 
145, 157, 169, 181, 193, 205 and 217 Weeks compared to Pre-Infusion 
Visit and Week 25 of Study NS-065/NCNP-01-201 
5. Quantitative Muscle Testing (QMT) at 37, 49, 61, 73, 85, 97, 109, 121, 
133, 145, 157, 169, 181, 193, 205 and 217 Weeks compared to Pre-
Infusion Visit and Week 25 of Study NS-065/NCNP-01-201  
Pharmacokinetic 
Assessments  
Blood samples will be collected during the same visits when blood samples 
are collected for anti-NS-065/NCNP-01 and anti-dystrophin antibody 
analyses. PK samples will only be analyzed in the event that anti-NS-
065/NCNP-01 antibodies are detected in order to understand the impact of 
immunogenicity on plasma levels of the drug. 
Exploratory 
Assessments 
Blood will be drawn for a serum PD biomarker panel to explore effect of 
NS-065/NCNP-01 on biomarkers of muscle cellular pathology.  
Statistical 
Methods 
Sample size:  
Approximately 16 patients from Study NS-065/NCNP-01-201 will enter this 
study.  Because this is an open-label extension study conducted in patients 
who completed Study NS-065/NCNP-01-201, the sample size is not based 
on any statistical considerations.    
 
Analysis Populations:  
All analyses will be based on the actual treatment each patient received. 
Two populations will be defined for data analysis: the Safety Population and 
the Full Analysis Set (FAS).  
 
Safety Population  
All patients who receive at least one dose of NS-065/NCNP-01 Injection 
250 mg in both the dose-finding (NS-065/NCNP-01-201) and extension 
study (NS-065/NCNP-01-202) will be included in the Safety Population. 
The Safety Population is the primary analysis population for safety 
assessments.  
 
Full Analysis Set (FAS)  
All patients who receive at least one dose of NS-065/NCNP-01 Injection 
250 mg in both the dose-finding (NS-065/NCNP-01-201) and extension 
study (NS-065/NCNP-01-202) will be included in the FAS. The FAS is the 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 7 of 112 
primary analysis population for clinical efficacy and exploratory PD 
assessments.  
General Statistical Considerations:  
All measurements will be analyzed based upon the type of distribution and 
descriptive statistics presented by dose level and time point, as appropriate.  
Analyses utilizing pooled dose levels may also be performed. Interim 
analyses for safety are planned and interim analyses for efficacy may be 
conducted. 
   
Efficacy Evaluation:  
The primary efficacy outcome measure (TTSTAND), as well as secondary 
and exploratory outcome measures, will be summarized by dose level over 
time using descriptive statistics.  Actual values and change from Pre-
Infusion Visit (from Study NS-065/NCNP-01-201) and change from Week 
25 (baseline of the NS-065/NCNP-01-202 study) will be presented. 
Outcomes will also be compared between NS-065/NCNP-01 Injection 250 
mg and matched historical untreated controls possibly using a mixed-effects 
linear model.  
Safety and Tolerability Evaluation:  
Safety analyses will be performed using the Safety Population. Vital signs, 
12-lead ECG, and clinical laboratory test results will be summarized by 
dose level over time using descriptive statistics. Actual values and change 
from Screening Visit (baseline of Study NS-065/NCNP-01-201) and change 
from Week 25 (baseline of Study NS-065/NCNP-01-202) will be presented.  
Treatment-emergent AEs (TEAEs) will be summarized by dose level, 
system organ class and preferred term (using the Medical Dictionary for 
Regulatory Activities (MedDRA) version 16.1), by dose level and 
relationship to study medication, and by dose level and intensity (CTCAE 
grade). All safety data will be listed.  

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 8 of 112 
TABLE OF CONTENTS 
STUDY SYNOPSIS ..................................................................................................................3 
LIST OF IN-TEXT TABLES ..................................................................................................12 
LIST OF IN-TEXT FIGURES.................................................................................................12 
LIST OF APPENDICES ..........................................................................................................12 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS ..........................................12 
1. 
INTRODUCTION ...........................................................................................................15 
1.1. 
Duchenne Muscular Dystrophy– Epidemiology and Genetic/Biochemical Basis 15 
1.2. 
Current Natural History, Disease Management and Treatment Recommendations15 
1.2.1. 
Glucocorticoid Treatment ............................................................................16 
1.2.2. 
Dystrophin restoring interventions ..............................................................16 
1.3. 
Background on NS-065/NCNP-01 ........................................................................17 
1.3.1. 
Mechanism of Action ...................................................................................17 
1.3.2. 
Summary of Non-Clinical Findings .............................................................18 
1.3.3. 
Pharmacokinetics .........................................................................................18 
1.3.4. 
Toxicology ...................................................................................................19 
1.3.5. 
Summary of Clinical Findings .....................................................................20 
1.3.5.1. Phase I ..........................................................................................................20 
1.3.5.2. Phase II Trials ..............................................................................................22 
1.4. 
Rationale for Study Design and Dose Selection ....................................................22 
2. 
STUDY OBJECTIVES ....................................................................................................27 
2.1. 
Primary Objectives.................................................................................................27 
2.2. 
Secondary Objectives.............................................................................................27 
2.3. 
Exploratory Objectives ..........................................................................................27 
3. 
INVESTIGATIONAL PLAN ..........................................................................................28 
3.1. 
Overall Study Design and Plan ..............................................................................28 
3.2. 
Design Implementation ..........................................................................................28 
3.2.1. 
Randomization .............................................................................................28 
3.2.2. 
Investigational Product Dosing ....................................................................28 
3.2.2.1. Low Dose Cohort .........................................................................................29 
3.2.2.2. High Dose Cohort ........................................................................................29 
3.2.3. 
Potential Design Modifications Due to Toxicities .......................................30 
3.3. 
Study Duration .......................................................................................................30 
3.4 
SARS-CoV-2 (COVID-19) Pandemic Study Impact ............................................31 
4. 
STUDY POPULATION SELECTION ...........................................................................32 
4.1. 
Study Population ....................................................................................................32 
4.2. 
Inclusion Criteria ...................................................................................................32 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
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4.3. 
Exclusion Criteria ..................................................................................................32 
5. 
INVESTIGATIONAL PRODUCT ..................................................................................33 
5.1. 
Description of NS-065/NCNP-01 ..........................................................................33 
5.2. 
Dispensing Investigational Product .......................................................................33 
5.3. 
Instructions for Administration of Investigational Product ...................................33 
5.4. 
Blinding..................................................................................................................34 
5.5. 
Treatment Compliance ...........................................................................................34 
5.6. 
Packaging and Labeling .........................................................................................34 
5.7. 
Storage and Accountability ....................................................................................34 
6. 
CONCOMITANT MEDICATIONS AND TREATMENTS ..........................................35 
6.1. 
Prohibited Medications ..........................................................................................35 
6.2. 
Allowable Medications ..........................................................................................35 
7. 
STUDY PROCEDURES .................................................................................................36 
7.1. 
Time and Events Schedule .....................................................................................36 
7.2. 
Informed Consent...................................................................................................47 
7.3. 
Assignment of Patient Identification Number .......................................................47 
7.4. 
Demographics ........................................................................................................48 
7.5. 
Medical History .....................................................................................................48 
7.6. 
Weight and Height .................................................................................................48 
7.7. 
Vital Signs ..............................................................................................................48 
7.8. 
Physical and Neurological Examination ................................................................49 
7.9. 
Renal and Urinary Bladder Ultrasound..................................................................50 
7.10. Adverse Events and Serious Adverse Events ........................................................50 
7.11. 12-Lead Electrocardiograms ..................................................................................50 
7.12. Clinical Laboratory Tests .......................................................................................50 
7.12.1. 
Sample Collection, Storage, and Shipping ..................................................51 
7.12.2. 
Cytokine Testing ..........................................................................................52 
7.12.3. 
Anti-Dystrophin Antibody ...........................................................................53 
7.12.4. 
Anti-NS-065/NCNP-01 Antibody ...............................................................53 
7.13. Pharmacodynamics and Efficacy Assessments .....................................................53 
7.13.1. 
Serum Pharmacodynamic Biomarkers .........................................................53 
7.14. Function and Strength ............................................................................................53 
7.14.1. 
Time to Stand (TTSTAND) - Primary Efficacy Endpoint...........................53 
7.14.2. 
Time to Run/Walk 10 Meters (TTRW) .......................................................53 
7.14.3. 
Time to climb 4 stairs (TTCLIMB) .............................................................54 
7.14.4. 
North Star Ambulatory Assessment (NSAA) ..............................................54 
7.14.5. 
Six-minute Walk Test (6MWT) ...................................................................54 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
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7.14.6. 
Quantitative Muscle Testing (QMT) ...........................................................55 
7.14.7. 
Ulnar Length ................................................................................................55 
7.15. Pharmacokinetic Assessments ...............................................................................55 
7.15.1. 
Collection and Assessment of Pharmacokinetic Samples ...........................55 
7.15.2. 
Shipment of Pharmacokinetic Samples .......................................................56 
7.16. Total Blood Volume of Clinical Laboratory and Pharmacokinetic Samples ........56 
8. 
STUDY ACTIVITIES .....................................................................................................58 
8.1. 
Treatment Phase .....................................................................................................58 
8.1.1. 
Day of First Infusion (Week 25) ..................................................................59 
8.1.2. 
Routine Infusion Visits ................................................................................59 
8.1.3. 
Weeks 29, 33, 41, and 45 .............................................................................60 
8.1.4. 
Week 37, 49, 73, and 97 ..............................................................................60 
8.1.5. 
Routine Infusion Visits with Weight Collection ..........................................61 
8.1.6. 
Weeks 61, 85, 109, 133, 157, 181, and 205 .................................................61 
8.1.7. 
Week 121, 145, 169, and 193 ......................................................................62 
8.2. 
Post-Treatment Phase.............................................................................................63 
8.2.1. 
Week 217 or Early Termination Visit ..........................................................63 
8.2.2. 
Follow-Up Phone Call .................................................................................64 
8.2.4. 
Early Termination or Withdrawal from the Study .......................................64 
8.2.5. 
Procedures for Early Termination ................................................................65 
8.3. 
Unscheduled Visit ..................................................................................................66 
8.4. 
Patient Replacement...............................................................................................66 
8.5. 
Suspension or Termination of Study......................................................................66 
9. 
SAFETY PROCEDURES AND PROCESSES ...............................................................67 
9.1. 
Definition of an Adverse Event .............................................................................67 
9.2. 
Definition of a Serious Adverse Event ..................................................................68 
9.3. 
Severity ..................................................................................................................69 
9.4. 
Relationship ...........................................................................................................70 
9.5. 
Reporting................................................................................................................70 
9.5.1. 
Adverse Event Reporting .............................................................................70 
9.5.2. 
Serious Adverse Event Reporting ................................................................71 
9.6. 
Serious Adverse Event Follow-up .........................................................................72 
9.7. 
Reporting of Serious Adverse Events to Regulatory Authorities ..........................72 
9.8. 
Reporting of Patient Death.....................................................................................72 
9.9. 
Monitoring and Follow-up of Adverse Events ......................................................72 
9.10. General Monitoring and Management of Abnormal Clinical Labs .......................73 
9.12. Monitoring and Management of Abnormal Electrocardiograms ...........................74 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
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9.13 
Intravenous (IV) Access Considerations ...............................................................74 
9.14. Medical Monitor ....................................................................................................75 
9.15. Data and Safety Monitoring Board ........................................................................75 
10. PLANNED STATISTICAL METHODS ..........................................................................77 
10.1 
General Considerations ..........................................................................................77 
10.2 
Determination of Sample Size ...............................................................................77 
10.3 
Analysis Populations ..............................................................................................78 
10.4 
Demographics and Baseline Characteristics ..........................................................78 
10.5 
Safety Assessments ................................................................................................78 
10.5.1 
Anthropometrics, Vital Signs, Laboratory Assessments, and ECG .............79 
10.5.2 
Physical Exam and Adverse Events .............................................................79 
10.5.3 
Concomitant Medications and/or Other Treatments ....................................80 
10.5.4 
Cytokines, Antibodies and Pharmacokinetics..............................................80 
10.6 
Efficacy Endpoints .................................................................................................80 
10.6.1 
Primary Efficacy Objective ..........................................................................80 
10.6.2 
Secondary Efficacy Objectives ....................................................................81 
10.6.2.1 
Analyses of NS-065/NCNP-01 patients only ....................................81 
10.6.2.2 
Analyses comparing NS-065/NCNP-01 patients to historical controls81 
10.7 
Interim Analyses ....................................................................................................82 
10.8 
Handling of Missing Data ......................................................................................82 
11 ADMINISTRATIVE CONSIDERATIONS ....................................................................83 
11.1 
Investigators ...........................................................................................................83 
11.2 
Informed Consent, Protected Health Information (PHI) and Confidentiality ........84 
11.2.1 
Informed Consent.........................................................................................84 
11.2.2 
Confidentiality .............................................................................................84 
11.2.3 
Protected Health Information (PHI) .............................................................85 
11.3 
Study Administrative Structure..............................................................................85 
11.4 
Institutional Review Board/Research Ethics Board Approval...............................86 
11.5 
Ethical Conduct of the Study .................................................................................87 
11.6 
Study Monitoring ...................................................................................................87 
11.7 
On-Site Audits .......................................................................................................88 
11.8 
Case Report Forms and Study Records .................................................................89 
11.9 
Amendments ..........................................................................................................90 
11.10 Access to Source Documentation ..........................................................................91 
11.11 Record Retention ...................................................................................................91 
11.12 Financial Disclosure...............................................................................................92 
11.13 Publication and Disclosure Policy .........................................................................92 
12 REFERENCE LIST .........................................................................................................94 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
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NS Pharma, Inc. – CONFIDENTIAL  
 
Page 12 of 112 
 
LIST OF IN-TEXT TABLES 
Table 1. 
Schedule of Study Assessments .................................................................38 
Table 2.  
Clinical Laboratory Tests ...........................................................................52 
Table 3. 
Blood Sample Number and Volume ..........................................................57 
 
LIST OF IN-TEXT FIGURES 
Figure 1. 
 Dose-Dependency of AUC0-t or AUC0-24 for Humans and Monkeys .......25 
Figure 2. 
 Correlation of AUC0-t or AUC0-24 with Dose from 0 to 200 mg/kg......25 
Figure 3. 
 Expansion of Correlation from 0 to 80 mg/kg ..........................................26 
 
LIST OF APPENDICES 
Appendix 1 
Sponsor Signatures.....................................................................................97 
Appendix 2 
Investigator’s Signature .............................................................................98 
Appendix 3 
Summary of Changes .................................................................................99 
 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS 
2’O-Me 
6MWT  
2’O-methyl phosphorothioate 
Six minute walk test  
aPTT 
ADL 
AE 
Activated partial thromboplastin time 
Activities of Daily Living 
Adverse Event 
ATC 
Anatomical Therapeutic Chemical classification 
AUC  
Area under the curve  
BMD 
BNP 
Cmax 
Becker muscular dystrophy 
Brain Natriuretic Peptide 
Maximum Drug Concentration 
Ca 
CGH 
Cl 
CE 
Calcium 
Comparative genomic hybridization 
Chloride 
Clinical evaluator 
CINRG 
Cr 
Cooperative International Neuromuscular Research Group  
Creatinine  
CS 
CK  
Clinically significant 
Creatine kinase  
Cm 
Centimeter 
CTCAE 
Common Terminology Criteria for Adverse Events v4.03 
CVA 
CVC 
Central Venous Access 
Central Venous Catheter 
CYP 
Cytochrome P450 
DMD  
Duchenne muscular dystrophy  

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DNHS  
Duchenne Natural History Study  
DSMB 
ECG 
eCRF 
FDA 
GC 
GCP 
HEENT 
HIPAA 
ICF  
Data and Safety Monitoring Board 
Electrocardiogram 
Electronic case report form 
Food and Drug Administration 
Glucocorticoid 
Good Clinical Practice 
Head, ears, eyes, nose, and throat 
Health Insurance Portability and Accountability Act 
Informed consent form 
ICH 
International Council of Harmonization 
IND  
Investigational New Drug  
IP 
IPIM 
Inorganic phosphorous 
Investigational Product Instruction Manual 
IRB 
IV 
K 
Kg 
Institutional Review Board 
Intravenous 
Potassium 
Kilogram 
MCP-1 
Monocyte chemoattractant protein-1 
MedDRA 
Medical Dictionary for Regulatory Activities 
mITT 
MLPA 
Modified intention to treat 
Multiplex Ligation-dependent Probe Amplification 
MM 
mRNA 
Medical Monitor 
Messenger ribonucleic acid 
MS  
Mass spectrometry  
MSO 
MTD 
Medical Safety Officer 
Maximum tolerated dose  
Na 
Sodium 
NAG 
N-acetyl-beta-D-glucosaminidase 
NCNP  
National Center of Neurology and Psychiatry, Japan 
NOAEL  
No observed adverse effect level  
NSAA 
NSP  
North Star Ambulatory Assessment 
NS Pharma, Inc. 
NS-065/NCNP-01  
NS-065/NCNP-01 drug substance  
NS-065/NCNP-01 
Injection 
NS-065/NCNP-01 drug product  
NS-065/NCNP-01 
Injection 250 mg 
NS-065/NCNP-01 investigational drug product (vial strength) 
PHI 
Protected Health Information 
PI 
PD 
PK  
Principal Investigator 
Pharmacodynamic 
Pharmacokinetic 
PMO  
Phosphorodiamidate morpholino oligomer  
PT-INR 
QMT  
Prothrombin – international normalization ratio 
Quantitative Muscle Testing  

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QWBA 
RT-PCR 
RBC 
REB 
RIPA 
RNA 
SAE  
SAP 
SD 
SDS-PAGE 
SILAC 
t1/2 
Tmax 
TEAEs 
TK 
Quantitative Whole-Body Autoradiography 
Reverse transcriptase polymerase chain reaction 
Red blood cell count 
Research Ethics Board 
Radioimmunoprecipitation assay 
Ribonucleic acid 
Serious Adverse Event 
Statistical analysis plan 
Standard deviation 
sodium dodecyl sulfate polyacrylamide gel electrophoresis 
Stable isotope labeling using amino acids (in cell culture) 
Terminal Elimination Half-Life 
Time of Maximum Drug Concentration 
Treatment-emergent AEs 
Toxicokinetic 
TNF-α 
Tumor necrosis factor-alpha 
TTCLIMB  
Time to Climb 4 stairs  
TTRW  
Time to Run/Walk 10 Meters  
TTSTAND  
Time to Stand 
WBC 
White blood cell count 

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1. INTRODUCTION 
1.1. Duchenne Muscular Dystrophy– Epidemiology and Genetic/Biochemical Basis 
Duchenne muscular dystrophy (DMD) is a disorder of progressive weakness leading to severe 
disability and ultimately death caused by a deficiency of the dystrophin protein. DMD is the most 
common form of muscular dystrophy, affecting 1 in every 3,500-6,000 live male births (1). The 
symptoms of DMD are often first noted at about 3-5 years of age, although clinical manifestations 
may be present as early as the first year of life. Proximal leg weakness impairs mobility and 
precludes the ability to run or to rise from a squatting position. Complete loss of ambulation 
follows, with a progressive decline of upper extremity strength and function. Declines in 
respiratory and cardiac function contribute to morbidity later in the disease, ultimately culminating 
in early lethality (1, 2).  The impact of this debilitating condition on those affected by it and their 
families is significant.  
The biochemical basis of DMD is the absence of a functional dystrophin protein that is essential 
for healthy muscle function and muscle fiber integrity.  In normal striated muscle, the cytoplasmic 
dystrophin protein links intracellular actin with the extracellular matrix to provide structural 
stability of the muscle cell membrane.  In the majority of patients with DMD, dystrophin protein 
is not produced because of out-of-frame mutations characterized by a deletion of one or more 
exons from the dystrophin gene, which is located on the short arm of the X chromosome.  
Dystrophin mutations in which some dystrophin protein function remains are associated with a 
similar, but often milder phenotype, classified as Becker muscular dystrophy (BMD). DMD and 
BMD exhibit X-linked recessive inheritance. 
1.2. Current Natural History, Disease Management and Treatment Recommendations 
The Cooperative International Neuromuscular Research Group (CINRG) (3) is conducting the 
largest prospective multicenter natural history study to date in DMD, the CINRG Duchenne 
Natural History Study (DNHS) (4, 5).  The study includes >400 boys and men with DMD, followed 
for up to a decade at present.  The study has annual follow-up visits.  These visits include timed 
function tests, muscle strength, questionnaire functional assessments, pulmonary function tests and 
quality of life assessments.  The DNHS database may be used as a reference dataset for the analysis 
of the cases in this study and Study NS-065/NCNP-01-201. 

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Since there is currently no cure for DMD, the goal of care is to provide the best quality of life 
through all stages of the disease.  To date, treatments focus on optimizing strength and function 
through the use of pharmacological interventions, physical therapy and assistive and adaptive 
devices. 
1.2.1. Glucocorticoid Treatment 
At present, treatment with glucocorticoid (GC) medication is the only pharmacological 
intervention that has been shown to slow the decline of strength and function in DMD patients. 
The two main GCs used in DMD are prednisone and deflazacort (EMFLAZA™). Daily oral 
administration of prednisone or deflazacort stabilizes or improves strength and prolongs 
ambulation (6-11). The mechanism by which GCs are beneficial in dystrophin deficiency is likely 
multifactorial, including anti-inflammatory actions.  The immunosuppressive effects of GCs may 
not be beneficial, and other immunosuppressants have not shown benefit (8). 
In 2005, the American Academy of Neurology issued a practice parameter regarding GC treatment 
in DMD and recommended that GC should be offered as treatment, despite known side effects 
(12).  The significant side effects of GCs include cushingoid features, adverse behavioral changes, 
obesity, growth retardation, increased risk for bone fractures, gastritis, delayed puberty, cataracts, 
hypertension, glucose intolerance, susceptibility to infection, and masking of response to stress 
(13). On February 9, 2017, the United States Food and Drug Administration (FDA) approved 
EMFLAZA™ to treat patients 5 years of age and older with DMD.   
1.2.2. Dystrophin restoring interventions 
New therapies based on specific genotypes are in development.  Small molecules that can read 
through nonsense mutations could potentially treat approximately 10% of DMD patients (14).  
Exon skipping, which uses antisense oligonucleotides to alter the splicing pattern of the genes is 
designed to bring out-of-frame deletions into frame.  The technology of exon skipping utilizes 
antisense oligonucleotides that bind to a specific sequence in the messenger ribonucleic acid 
(mRNA) to alter splicing of exons.  By this means, specific exons can be excluded from the final 
transcript that is exported to the cytoplasm from the nucleus; hence the term ‘exon skipping’.  By 
the design of the oligonucleotide, the out-of-frame deletion can be enlarged to include the adjacent 
exon such that the resulting deletion is in-frame (15-17). 

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This new type of treatment could potentially treat more than 85% of DMD patients who have large-
scale deletion or duplication mutations in the dystrophin gene (15-17).  The full characterization 
of DMD patient mutations and further development of the technology will be crucial to fully 
realize these novel therapies as they are developed. 
To date, 2 oligonucleotide chemistries have been brought to the stage of human clinical trial: 2’O-
methyl phosphorothioate (2’O-Me) antisense oligonucleotides (18, 19) and phosphorodiamidate 
morpholino oligomers (PMO) for skipping of exon 51 in the dystrophin gene (20-22). 
A 48-week study of the 2’O-Me compound drisapersen did not reach significance in the six-minute 
walk test (6MWT), which was its primary outcome measure for a Phase II study (18). The PMO 
compound eteplirsen was tested in a 48-week study with the number of muscle fibers with restored 
dystrophin as its primary outcome measure (20). Eteplirsen (Exondys 51®) was approved by the 
FDA under the accelerated approval pathway on September 19, 2016. 
1.3. Background on NS-065/NCNP-01 
NS-065/NCNP-01 is a novel antisense oligonucleotide for the treatment of DMD, which has been 
discovered jointly by National Center of Neurology and Psychiatry (NCNP), which is a National 
Research and Development Agency in Japan, and Nippon Shinyaku Co., Ltd.  Details of data 
summarized in the following sections can be found in the Investigator’s Brochure. 
1.3.1. Mechanism of Action 
NS-065/NCNP-01 is designed to interact with the dystrophin gene ribonucleic acid (RNA), and 
alter the exon/intron splicing patterns. The mechanism of action is for NS-065/NCNP-01 to bind 
to a specific sequence in or near exon 53 of the dystrophin pre-RNA transcript, and block the 
exon/intron splicing of exon 53, leading to mature mRNA transcripts that lack exon 53. NS-
065/NCNP-01 is thought to be effective on DMD patients with exon deletions amenable to 
skipping of exon 53 such as 43-52, 45-52, 47-52, 48-52, 49-52, 50-52, or 52.  The loss of exon 53 
restores the mRNA reading frame, thus converting a DMD (out-of-frame) deletion mutation to a 
Becker-like (in-frame) deletion mutation.  In-frame deletion mutations are typically compatible 
with production of a shortened dystrophin protein, although the resulting Becker-like dystrophin 
protein will be smaller in molecular weight compared to the normal dystrophin protein, and likely 

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lower in abundance (quantity) compared to normal muscle, and thus may have lower function than 
normal amounts of wild-type dystrophin protein. 
1.3.2. Summary of Non-Clinical Findings 
In silico studies to identify potential off-target sequences suggest a very low probability that   NS-
065/NCNP-01, n-1, n-2 and shorter oligonucleotides would have an off-target effect, whereas n+1 
oligonucleotides may have off-target effects on four genes (DHX16, DMRTA2 MALAT1, and 
CX3CL1) in humans.  
NS-065/NCNP-01 did not display any adverse effects in in vitro and in vivo cardiovascular, in vivo 
CNS, or in vivo respiratory safety pharmacology studies.  
1.3.3. Pharmacokinetics 
Pharmacokinetic (PK) and toxicokinetic (TK) analyses revealed no apparent species differences 
for NS-065/NCNP-01.  None of the in vitro or in vivo metabolism studies showed any distinct 
evidence of metabolism of NS-065/NCNP-01.  After IV administration, Tmax occurred at the first 
sampling time after the injection or at the end of the infusion for mice, rats, and monkeys. Cmax 
and AUC increased with dose, and most increases were approximately proportional to dose, with 
some increases being greater than dose proportional.  For rats, the mean values for t1/2 were 1.19, 
1.19, and 10.5 hours for 6, 20, and 60 mg/kg.  For monkeys, the mean values for t1/2 ranged from 
1.7 to 3.5 hours.  For mice and monkeys, exposure did not change with 12 or 13 weeks of repeat 
dosing. 
Results for IM administration of NS-065/NCNP-01 to monkeys compared to IV administration 
indicate complete bioavailability, a longer time to Tmax, a similar half-life for elimination, and no 
increase in exposure with repeat dosing.  
The fraction of NS-065/NCNP-01 bound to rat, monkey, and human serum proteins was low, 
≤40%, for all species and was independent of concentration.  The distribution of NS-065/NCNP-
01 into red blood cells was ≤2.5%, ≤6.7%, and ≤3.5% for rat, monkey, and human, respectively, 
indicating low distribution of NS-065/NCNP-01 to red blood cells.  Quantitative whole-body 
autoradiography (QWBA) studies showed wide tissue distribution of [14C]NS-065/NCNP-01 in 
both mice and monkeys, with the highest concentrations observed in the kidney, and general 
distribution to muscle tissues.  For both rats and monkeys, renal excretion was the major route of 

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elimination, with less than 10% in the feces.  No radioactivity was in the expired air from the rats.  
Most of the radioactivity was excreted within the first 24 hours.  However, small measurable 
amounts continued to be excreted in the urine and feces throughout the seven-day collection 
periods.  
The degree of inhibition of CYP1A2 by NS-065/NCNP-01 was low and the type of inhibition 
could not be determined.  The inhibition was less than 30% for the other tested CYP isozymes: 
2A6, 2B6, 2C8, 2C9, 2C19, 2D6, 2E1, and 3A4/5. NS-065/NCNP-01 was not an inducer of 
CYP1A2, CYP2B6, or CYP3A4/5. 
1.3.4. Toxicology 
Repeated administration of NS-065/NCNP-01 via the clinically relevant route of administration 
(weekly IV injections) in both mice and monkeys resulted in decreases in red blood cell 
parameters, increased values for cytokines and histopathological effects in the kidney, testes and/or 
urinary bladder.  The kidney is the primary target organ in both mice and monkeys, as shown by 
increased values in clinical chemistry parameters indicative of renal effects (1000 mg/kg/dose in 
13- and 26-week mouse studies or 600 mg/kg/dose in a 12-week monkey study) and by 
histopathological findings of effects in renal tubules (≥240 mg/kg/dose in 13- and 26-week mouse 
studies, ≥200 mg/kg/dose in a 12-week monkey study or 360 mg/kg/dose in a 39-week monkey 
study) occasionally accompanied by increased kidney weight at necropsy.  This observation is 
consistent with the PK data that show high tissue distribution to the kidney and excretion via the 
urine.  An additional histopathological finding in a 26-week mouse study was the presence of 
cytoplasmic eosinophilic material in the transitional epithelium of the urinary bladder at 60 
mg/kg/dose and above.  At high dose (1000 mg/kg/dose in 13- and 26-week mouse studies or 600 
mg/kg/dose in a 12-week monkey study), reductions in red blood cell parameters were also seen.  
Higher doses in mice (≥240 mg/kg/dose in a 13-week mouse study or 1000 mg/kg/dose in a 26-
week mouse study) showed increased values for interleukin-6 (IL-6), tumor necrosis factor-alpha 
(TNF-α) and/or monocyte chemoattractant protein-1 (MCP-1) and alterations in complement; 
these findings were considered to be due to irritation of the injection site.  Data from 4- and 8-
week recovery periods indicated a trend toward reversal of these effects in both species. 

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 Moreover, no anti-NS-065/NCNP-01 antibodies were detected in the 13-week or 26-week mouse 
studies or in the 39-week monkey study.  Anti-NS-065/NCNP-01 antibodies were detected in one 
monkey at 200mg/kg in the 12-week monkey study.  However, antibody detection was not 
considered to affect the toxicological evaluation in this study, since skipping efficiency was 
confirmed in the muscle of this animal at the end of the treatment period and no remarkable 
decrease in exposure to NS-065/NCNP-01 was observed after repeated dosing.  Based on these 
data, the NOAELs were concluded to be 60 and 15 mg/kg/dose in 13-week and 26-week mouse 
studies respectively and 60 mg/kg/dose in 12-week and 39-week monkey studies respectively.  
Both the in vitro and in vivo genotoxicity studies were negative. 
NS-065/NCNP-01 was administered once weekly for 26 weeks at a dose level of 0 (vehicle: 
physiological saline), 50, 150 and 500 mg/kg (51 mice per group) male CByB6F1-Tg(HRAS)2Jic 
mice via intravenous route with a bolus injection. After the terminal necropsy, macroscopic 
examinations showed a mass and/or thickening in one side of the ureter in one mouse at 50 mg/kg, 
in two mice at 150 mg/kg, and no findings at 500 mg/kg dose group. In subsequent 
histopathological examinations on these three mice, transitional cell carcinoma was noted. 
Histopathological examination of the ureters for the other mice was conducted and no further 
tumorigenic changes were identified. Additionally, no treatment-related tumors were noted in any 
other organs. The blood concentration of NS-065/NCNP-01 in mice who received 50 mg/kg/wk 
was lower than the blood concentration of NS-065/NCNP-01 expected in human patients who will 
receive 80mg/kg/wk. 
 
1.3.5. Summary of Clinical Findings 
1.3.5.1. 
Phase I 
A Phase I investigator-initiated study of NS-065/NCNP-01 Injection was conducted in DMD 
patients (aged 5-18 years) to investigate the overall usefulness of NS-065/NCNP-01 Injection in 
the treatment of DMD, based on evaluations of safety, exploration of predictive markers of 
treatment response, and assessment of pharmacokinetics.  A total of 10 DMD patients were 
enrolled and randomized. IV administration of NS-065/NCNP-01 Injection in doses of 1.25 mg/kg, 
5 mg/kg and 20 mg/kg to DMD patients once weekly for 12 weeks was well tolerated, and no 

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dose-limiting toxicity was observed, although all patients had at least one AE. Moreover, neither 
serious adverse events (SAE) nor incidences of Common Terminology Criteria for Adverse Events 
(CTCAE) v4.03 Grade 3 (severe) or worse were reported.  
Among the mild and moderate AEs, an increase in urine beta-N-acetyl-D-glucosaminidase (Grade 
1) was found in all patients in both cohorts 1 and 2 [Cohort 1 (n=3) Cohort 2 (n=3)] and in all but 
one patient in Cohort 3 [Cohort 3 (n=4)].  
Initially, testing appeared to reveal proteinuria in 8 of 10 patients.  However, it was subsequently 
determined that there was a cross reaction between NS-065/NCNP-01 and the pyrogallol red dye-
binding method which was used for urinary protein measurement resulting in a false positive result 
for protein in the 24-hour pooled urine samples.  To evaluate the 24-hour pooled urine samples for 
protein, Coomassie brilliant blue method was used to re-measure urinary protein in the frozen urine 
samples and none of the retested samples showed urinary protein levels exceeding the normal 
range of the institution (i.e., 31.2 to 120 mg/day).  Current studies utilize a urine protein 
Benzethonium Chloride method using reagents that do not cross react with NS-065/NCNP-01.  
NS-065/NCNP-01 exhibited no cross reactivity with the Benzethonium Chloride Method up to 
500 mg/dL.  
The 24-hour pooled urine samples did not show increased levels of albumin.  Spot measurements 
of urinary albumin were positive in 7 patients (Grade 1).  
 Interleukin levels were increased (Grade 1) in the serum in 6 out of 10 patients (high IL-6 level in 
4 subjects, high IL-1β level in 1 subject, and high IL-2 level in 1 subject), and some level of anemia 
(Grade 1) was observed in 7 out of 10 patients.   
Increased levels of brain natriuretic peptide (BNP) in serum (Grade 1) were present in four patients. 
White blood cell count was increased in 3 patients (Grade 1).  All other mild and moderate AEs 
occurred in two or fewer patients. 
The maximal plasma concentration and area under the curve (AUC)0-t values increased in a dose-
dependent manner, and the t1/2 value was between 1.52 and 1.84 hours. 
Distinct exon 53 skipping efficiency by reverse transcriptase polymerase chain reaction (RT-PCR), 
positive dystrophin fibers by immunofluorescent staining, and dystrophin protein expression by 

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western blotting were detected in one patient in cohort 3, who was the largest patient enrolled in 
the cohort and hence received the largest absolute dose of NS-065/NCNP-01 Injection that was 
administered in the study.  
1.3.5.2. 
Phase II Trials 
NS065/NCNP01-P1/2 is a Phase I/II study of NS-065/NCNP-01 Injection being conducted in 
Japan.  This is a multicenter, parallel-group, open-label, 24-week study.  Participants receive 
weekly IV administration of NS-065/NCNP-01 Injection 250 mg (40 mg/kg and 80 mg/kg) over 
24 weeks.  The primary efficacy endpoint of this study is dystrophin protein expression as 
measured by Western blot, immunofluorescence staining, and RT-PCR.  16 DMD patients 
amenable to Exon 53 skipping, aged 5-<18 years are enrolled. 
NS-065/NCNP-01-201 is a Phase II study conducted in the United States and Canada.   
This is a multicenter, 24-week dose finding study to assess the safety, tolerability, 
pharmacokinetics and pharmacodynamics of NS-065/NCNP-01 in boys with DMD.  Participants 
receive weekly IV administration of NS-065/NCNP-01 Injection 250mg (40 mg/kg and 80 mg/kg) 
or Placebo for the first 4 weeks, followed by weekly IV administration of NS-065/NCNP-01 
Injection 250mg (40 mg/kg and 80 mg/kg) over the remaining 20 weeks.  The primary efficacy 
endpoint of this study is dystrophin protein expression as measured by Western blot.  16 DMD 
patients amenable to Exon 53 skipping, aged 4-<10 years were enrolled. 
1.4. Rationale for Study Design and Dose Selection 
This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-01 Injection 250 mg 
administered intravenously once weekly for up to 192 weeks (up to a total of 216 weeks of 
treatment in Study NS-065/NCNP-01-201 and 202) or until enrollment in a separate long-term 
follow up program of NS-065/NCNP-01, whichever is earlier to boys with DMD who complete 
Study NS-065/NCNP-01-201.  This study will evaluate the safety, tolerability, and clinical efficacy 
of NS-065/NCNP-01 Injection 250 mg at dose levels of up to 80 mg/kg/week, administered 
intravenously by infusion, over an additional treatment period of up to 192 weeks (up to a total of 
216 weeks of treatment in Study NS-065/NCNP-01-201 and 202) or enrollment in a separate long-
term follow up program of NS-065/NCNP-01, whichever is earlier.  Patients who complete the 
Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll at the same dose and will 

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undergo baseline assessments at Week 25 of Study NS-065/NCNP-01-201.  In order to 
accommodate patient recovery following the scheduled biopsy during Week 25 of Study NS-
065/NCNP-01-201, patients will be allowed a visit window of 7 days (±3 days) from the Week 24 
dose to complete both the end of study procedures and the first infusion in the extension study.  
All Week 25 study assessments, including muscle biopsy, of the NS-065/NCNP-01-201 study 
must be completed prior to first infusion in this extension study.  Patients can receive the first 
infusion of this extension study on the same day as the Week 25 study visit of the NS-065/NCNP-
01-201 study, provided all study assessments (including muscle biopsy) of the NS-065/NCNP-01-
201 study are completed prior to infusion.  
Dose selection for Study NS-065/NCNP-01-201 was based on exposure data in humans compared 
to mice and monkeys over a comparable 12-week treatment period.  Based on the t1/2 values, the 
kinetics for monkeys are closer to human kinetics than the kinetics for mice.  The AUC values for 
monkeys are higher than those for mice at similar doses.  In the 12-week mouse study, the AUC0-
24 values were 83.5, 421, and 12,300 µg•hr/mL for 60, 240, and 1,000 mg/kg, respectively.  In the 
12-week monkey study, the mean AUC0-24 values were 270, 963, and 5,581 µg•hr/mL for 60, 200, 
and 600 mg/kg, respectively.  Hepatic and nuclease metabolism of NS-065/NCNP-01 was similar 
for monkey and human sources.  Overall, there was no apparent degradation or metabolism of NS-
065/NCNP-01 in the presence of hepatic microsomes, deoxyribonuclease I or phosphodiesterase 
I.  Because monkeys are larger animals than mice, and because the half-life and metabolism is 
similar for humans and monkeys, the results of the monkey studies were chosen for the estimation 
of AUC values for human subjects. 
Figure 1 shows the mean AUC0-t values from the human clinical study and the mean AUC0-24 
values from the single dose and 12-week monkey studies.  The mean AUC values from both the 
monkey and human were approximately linear up to 200 mg/kg.  At 200 mg/kg/day in Study 
TX1076, there were very slight effects on the kidneys of one of three male monkeys.  The effects 
were more pronounced at 600 mg/kg/day, affecting three of three animals.  There was little or no 
difference between Day 1 and Week 12 for either the humans or monkeys.  The single-dose 
monkey study used doses that overlapped the doses used in the 12-week studies of humans and 
monkeys. 

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The individual AUC values for doses up to 200 mg/kg were subjected to regression analysis fitting 
the equation y = mx.  The intercept was set to 0 since vehicle-treated animals had no measurable 
concentrations of NS-065/NCNP-01.  The results for both Day 1 or single doses and after 12 weeks 
of dosing were included since there was no apparent difference between the results for the first 
and 12th dose.  Using the results for monkeys only, the coefficient of determination, r2, was 0.9622, 
indicating a good fit to a line defined by AUC = 4,756 x Dose.  The regression analysis was 
repeated using both the human and monkey results.  The resulting equation was AUC = 4,760 x 
Dose with r2 = 0.9608.  The addition of the human results changed the slope by only 0.1%, and the 
fit remained good.  The results are shown in Figure 2, and Figure 3 is an expansion showing the 
results between 0 and 80 mg/kg.  At a dose of 60 mg/kg given to human subjects, the expected 
average AUC0-t after either the first or multiple doses would be expected to be approximately 
286,000 ng•hr/mL. 
In the 12-week toxicity study using monkeys, the NOAEL dose was 60 mg/kg/dose, and very slight 
effects were seen at the 3.3-fold higher dose, 200 mg/kg/dose.  A dose of 80 mg/kg is only 1.3-
fold higher than a 60 mg/kg and less than ½ the 200 mg/kg dose where slight adverse effects were 
observed.  It is possible that a human dose of 80 mg/kg producing an average AUC of 381,000 
ng•hr/mL would have an acceptable safety profile. 

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Clinical Study Protocol  NS-065/NCNP-01-202   
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NS Pharma, Inc. – CONFIDENTIAL  
 
Page 25 of 112 
Figure 1.  
Dose-Dependency of AUC0-t or AUC0-24 for Humans and Monkeys 
 
Figure 2.  
Correlation of AUC0-t or AUC0-24 with Dose from 0 to 200 mg/kg 
 
 
0
1,000,000
2,000,000
3,000,000
4,000,000
5,000,000
6,000,000
7,000,000
0
100
200
300
400
500
600
700
AUC0-24 or AUC0-t (ng*hr/mL)
Dose (mg/kg)
Human, Day 1
Humans, 12 Weeks
Monkeys, BP-065-008, Single Dose
Monkeys, TX10746, Day 1
Monkeys, TX10746, 12 Weeks
0
100,000
200,000
300,000
400,000
500,000
600,000
700,000
800,000
900,000
1,000,000
0
20
40
60
80
100
120
140
160
180
200
AUC0-24 or AUC0-t (ng*hr/mL)
Dose (mg/kg)
Monkeys, BP-065-008, Single Dose
Human, Day 1
Humans, 12 Weeks
Monkeys, TX10746, Day 1
Monkeys, TX10746, 12 Weeks
Regression line

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
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Page 26 of 112 
Figure 3.  
Expansion of Correlation from 0 to 80 mg/kg 
 
AUC (ng•hr/mL) = 4,760 ((ng•hr/mL)/(mg/kg)) x Dose (mg/kg) 
r2 = 0.9608. 
 
 
By the time of enrollment into the extension study NS-065/NCNP-01-202 the eligible patients are 
expected to have already successfully completed a 20-24-week treatment period at the 40 or 80 
mg/kg/wk dosing level.  Thus, the tolerability of the 2 dosing levels tested in study NS-065/NCNP-
201 are no longer expected to be in question.  
0
100,000
200,000
300,000
400,000
500,000
600,000
0
10
20
30
40
50
60
70
80
AUC0-24 or AUC0-t (ng*hr/mL)
Dose (mg/kg)
Monkeys, BP-065-008, Single Dose
Human, Day 1
Humans, 12 Weeks
Monkeys, TX10746, Day 1
Monkeys, TX10746, 12 Weeks
Regression line

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 27 of 112 
2. STUDY OBJECTIVES 
2.1. Primary Objectives 
1. To evaluate the safety and tolerability of low (40 mg/kg/week) and high (80 
mg/kg/week) IV doses of NS-065/NCNP-01 Injection up to 192-week Treatment Period 
(up to a total of 216 weeks of treatment in Study NS-065/NCNP-01-201 and 202), in 
boys with DMD completing Study NS-065/NCNP-01-201. 
2. To investigate the effect of low and high IV doses of NS-065/NCNP-01 Injection after 
an additional up to 192-week Treatment Period (up to a total of 216 weeks of treatment 
in Study NS-065/NCNP-01-201 and 202) on muscle strength, mobility, and functional 
exercise capacity, as measured by Time to Stand (TTSTAND), in boys with DMD who 
complete Study NS-065/NCNP-01-201 vs. matched historical controls in boys with 
DMD. 
2.2. Secondary Objectives 
1. To investigate the effect of low and high IV doses of NS-065/NCNP-01 Injection, after 
an additional up to 192-weeks treatment (up to a total of 216 weeks of treatment in 
Study NS-065/NCNP-01-201 and 202) on muscle strength, mobility, and functional 
exercise capacity, as measured by Time to Run/Walk 10 meters (TTRW), Time to Climb 
4 stairs (TTCLIMB), North Star Ambulatory Assessment (NSAA), Six-minute Walk 
Test (6MWT), and Quantitative Muscle Testing (QMT) in boys with DMD that 
complete the 24-week dose-finding Study NS-065/NCNP-01-201 vs. matched historical 
controls in boys with DMD.  
2.3. Exploratory Objectives 
1. To investigate the effects of low and high IV doses of NS-065/NCNP-01 Injection on 
serum pharmacodynamic (PD) biomarkers. 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
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Page 28 of 112 
3. INVESTIGATIONAL PLAN 
3.1. Overall Study Design and Plan 
This is a Phase II, open-label, extension study to assess the safety and efficacy of NS-065/NCNP-
01 Injection in boys with DMD after completing the 24-week dose-finding study NS-065/NCNP-
01-201.  
Patients must complete all study assessments of the Week 25 of Study NS-065/NCNP-01-201 
before performing any study assessments in this extension study.  In this extension study (NS-
065/NCNP-01-202) patients will continue to receive weekly study drug infusions at the dose level 
up to 80 mg/kg/week for up to 192 weeks (up to a total of 216 weeks of treatment in Study NS-
065/NCNP-01-201 and 202) or enrollment in a separate long-term follow up program of NS-
065/NCNP-01, whichever is earlier . Once sites receive IRB approval of Protocol Amendment 10, 
all subjects who were previously in the low dose cohort of 40 mg/kg/week will receive 80 
mg/kg/week after obtaining patient consent/assent. Week 193 is a comprehensive assessment at 
the beginning of the extension visits incorporated in protocol amendment 10, and Week 217 is the 
final study assessment for the treatment period ending with Week 216.  If the site is selected to 
participate in the separate long-term follow-up program and the patient agrees to participate by 
signing the consent form before the final study visit for the NS-065/NCNP-01-202 study, then the 
patient should complete the Early Termination visit and transition to the new study. The decision 
to transition to the follow up program will be made jointly by the patient/caregiver and investigator.  
3.2. Design Implementation 
3.2.1. Randomization 
Study NS-065/NCNP-01-202 is an open-label extension study and not a randomized trial.  
3.2.2. Investigational Product Dosing 
The dose per patient (in mg) will be calculated based on the most recent body weight in kg 
collected per the protocol and not including the current visit.  Details of dose preparation can be 
found in the Investigational Product Instruction Manual (IPIM).  Doses will be administered by an 
IV infusion over approximately 1-hour.  All missed or incomplete doses will be documented.  The 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 29 of 112 
dispensed study medication vials will be stored at the research site until drug accountability is 
verified by the pharmacy monitor. 
Peripheral venous access is the preferred route of IV administration and should be used for all IP 
infusions throughout the study unless otherwise approved by the sponsor. 
Implantable central venous access (CVA) ports will be considered on a case-by-case basis for 
participants who experience extreme difficulty with peripheral venous access. Discussions 
regarding implantable CVA ports for participants will include the site Principal Investigator (PI), 
Study Chair, Medical Monitor and Sponsor. Before final decision, NS Pharma will obtain 
documentation from the site investigator that the consulting surgeon who will place the port holds 
hospital privileges as a board eligible/board certified surgeon.  Central venous access should not 
proceed without Sponsor approval. 
An alternative method of central venous access may only be considered in the case of a 
contraindication, in the opinion of the consulting surgeon for the placement of a totally implantable 
central venous access device (port). 
3.2.2.1. 
Low Dose Cohort 
All patients in the low dose cohort of Study NS-065/NCNP-01-201 will continue to receive NS-
065/NCNP-01 Injection at 40 mg/kg/week for up to 192-weeks (up to a total of 216 weeks of 
treatment in Study NS-065/NCNP-01-201 and 202) or enrollment in a separate long-term follow 
up program of viltolarsen, whichever is earlier . The PI can increase the dose from 40 mg/kg/week 
to 80 mg/kg/week once the site’s IRB of record approves Protocol Amendment 10 and the patient 
signs the corresponding ICF.  The first infusion of 80 mg/kg/week should be administered at the 
site. 
3.2.2.2. 
High Dose Cohort 
All patients in the high-dose cohort will continue to receive NS-065/NCNP-01 Injection at 80 
mg/kg/week for up to 192-weeks (up to a total of 216 weeks of treatment in Study NS-065/NCNP-
01-201 and 202) or enrollment in a separate long-term follow up program of NS-065/NCNP-01, 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 30 of 112 
whichever is earlier..  If cumulative safety results do not warrant continuation at this dose level, 
the dose may be modified as described in Section 3.2.3. 
3.2.3. Potential Design Modifications Due to Toxicities 
Patients will receive the same dose in the extension study as they received in Study NS-065/NCNP-
01-201 to ensure continuity of care and ongoing safety considerations for NS-065/NCNP-01 
clinical data.  Dose reductions may be necessary for individual patients.  The dose level will be 
determined jointly by the Investigator, Study Chair and Medical Monitor in consultation with the 
Sponsor.  Any modifications in the study design will occur in consultation with the Study Chair, 
Medical Monitor, Sponsor and the data and safety monitoring board (DSMB), depending on the 
timing and nature of the toxicities.  
 
 
3.2.4 Dose Interruptions 
Infusion interruptions may be necessary for individual participants.  Infusion interruptions should 
be handled in accordance with standard procedures should an acute reaction occur during an 
infusion.  Sites should notify the medical monitor in the event of an infusion interruption due to 
an acute reaction. 
3.3. Study Duration 
The expected study duration for each patient is approximately  192 weeks of treatment (up to a 
total of 216 weeks of treatment in Study NS-065/NCNP-01-201 and 202), plus a 30-day post-
treatment phase, or until enrollment in a separate long-term follow up program of NS-065/NCNP-
01, whichever is earlier. If the site is selected to participate in the separate long-term follow-up 
program, and the patient agrees to participate by signing the consent form before the final study 
visit for the NS-065/NCNP-01-202 study, then the patient should complete the Early Termination 
visit and transition to the new study. Week 193 is a comprehensive assessment at the beginning of 
the extension visits incorporated in protocol amendment 10, and Week 217 is the final study 
assessment for the treatment period ending with Week 216.  The decision to transition to the follow 
up program will be made jointly by the patient/caregiver and investigator. 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 31 of 112 
3.4 SARS-CoV-2 (COVID-19) Pandemic Study Impact  
In early 2020, a global novel coronavirus outbreak of the SARS-CoV-2 (COVID-19) became a 
public health emergency. The full impact to this clinical trial is yet to be determined, however, 
provisions were put in place to ensure the safety of study participants as well as site staff.  
Memoranda were developed by the medical monitor which informed sites how to comply with 
protocol-specified procedures and mandated visits.  While the COVID-19 pandemic is ongoing  
the 3rd and most recent of these memoranda may be updated on an ongoing basis to stay in 
alignment with FDA guidance related to the conduct of clinical trials during the COVID-19 
pandemic. These documents are acknowledged by the IRBs of record as required. The full impact 
of COVID-19’s impact on the clinical trial will be outlined in the final Clinical Study Report as 
well as the Statistical Analysis Plan. 
 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 32 of 112 
4. STUDY POPULATION SELECTION 
4.1. Study Population 
Approximately 16 patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 
are eligible to enroll.  
4.2. Inclusion Criteria 
1. Patient’s parent or legal guardian has provided written informed consent/HIPAA 
authorization prior to any extension study-specific procedures and patient has provided 
assent appropriate for his age and developmental status. 
2. 
Patient completed Study NS-065/NCNP-01-201 through Week 25.  
3. 
Patient and parent/guardian are willing and able to comply with scheduled visits, 
investigational product administration plan, and study procedures. 
4. 
Patient must be on a stable dose of GC and is expected to remain on the stable dose of 
GC treatment for the duration of the study. 
4.3. Exclusion Criteria 
1. Patient had a serious or severe adverse event in Study NS-065/NCNP-01-201 that, in 
the opinion of the Investigator and/or the Sponsor, was probably or definitely related 
to NS-065/NCNP-01 Injection 250 mg use and precludes safe use of NS-065/NCNP-
01 Injection 250 mg for the patient in this study. 
2. Patient had a treatment which was made for the purpose of dystrophin, or dystrophin-
related protein induction after completion of Study NS-065/NCNP-01-201. 
3. Patient took any other investigational drugs after completion of Study NS-065/NCNP-
01-201.  
4. Patient is judged by the investigator and/or the Sponsor to not be appropriate to 
participate in the study for other reasons. 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 33 of 112 
5. INVESTIGATIONAL PRODUCT 
5.1. Description of NS-065/NCNP-01 
Investigational product is provided in 10 mL or 5mL glass vials for dilution and IV administration.  
• NS-065/NCNP-01: 10 mL or 5 mL glass vial containing 25 mg/mL or 50 mg/mL of drug 
substance solution in saline, respectively. 
Description:  
• NS-065/NCNP-01: Clear, colorless to pale yellow solution  
Stability: NS-065/NCNP-01 Injection 250 mg is stable at 5 ± 3°C. Additional stability details can 
be found in the IPIM. 
Storage conditions: Store refrigerated at 2° to 8°C. 
Investigational product will be packaged, labeled and distributed to clinical sites and home-
infusion central pharmacy by Xerimis in Moorestown, NJ. Additional details for ordering the 
investigational product can be found in the IPIM and study Manual of Operations.  
5.2. Dispensing Investigational Product 
Patients will begin dosing in this study at the same dose level they received in the Phase II NS-
065/NCNP-01-201 dose-finding study. Patients will continue the dose level up to 80 mg/kg/week 
for the duration of up to 192-week (up to a total of 216 weeks of treatment in Study NS-065/NCNP-
01-201 and 202)_study. Once sites receive IRB approval of Protocol Amendment 10, all subjects 
who were previously in the low dose cohort of 40 mg/kg/week will receive 80 mg/kg/week after 
obtaining patient consent/assent. Investigational product will be prepared in accordance with the 
IPIM and administered by IV infusion over approximately 1-hour.  
5.3. Instructions for Administration of Investigational Product 
Prepared investigational product (diluted solution) is administered intravenously within 6 hours 
of preparation and may be stored at room temperature during this time. Additional stability details 
can be found in the IPIM.  A minimum of 3 days (72 hours) should elapse between treatments. 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 34 of 112 
5.4. Blinding 
Study NS-065/NCNP-01-202 is an open-label extension study and is not blinded.  
5.5. Treatment Compliance 
The patient’s compliance with the treatment regimen will be monitored in terms of the patient 
receiving the investigational product infusion every week within a ±3-day window.  Weekly study 
drug treatments for this study should be calculated from the 1st infusion of protocol NS-
065/NCNP-01-201, not from the previous week’s infusion.  If an infusion day is rescheduled, the 
original scheme should be reinstated as soon as possible.  Missed, delayed or incomplete infusions 
will be clearly documented and considered in the analysis.  The amount of infusion received should 
be documented for all infusions.  
5.6. Packaging and Labeling 
Investigational product will be packaged and shipped from Xerimis directly to the investigative 
site and home infusion central pharmacy as a patient kit. Each patient kit consists of a single carton 
of 10 vials.  Ancillary supplies will be provided by Xerimis with each patient kit. Investigational 
product will be labeled in compliance with 21CFR312.6, Labeling of and Investigational New 
Drug.  
5.7. Storage and Accountability 
Investigational Product Storage: Refrigerated (2-8°C), store in light resistant, airtight containers. 
An identified, appropriate and secure storage location will be defined at each site’s pharmacy and 
central home-infusion pharmacy for the investigational product. Additional details regarding 
proper handling of the investigative product can be found in the IPIM. 
The investigator’s or site’s designated investigational product manager is required to maintain 
accurate investigational product accountability records.  In the case of home-infusions, the central 
pharmacy staff is required to maintain accurate investigational product accountability records. All 
unused investigational product will be returned or disposed of as defined in the IPIM. This 
information will be included as part of the investigational product accountability record. 
 
 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 35 of 112 
6. CONCOMITANT MEDICATIONS AND TREATMENTS 
Lifetime use of GC and other pharmacological medications, including over the counter 
medications, herbal remedies, supplements and vitamins used after completion of the Week 25 of 
Study NS-065/NCNP-01-201 and throughout the study will be recorded in source documents and 
in the electronic case report form (eCRF).  All medications taken throughout the study will be 
recorded in the eCRF.  The following information will be collected: the medication name, dose, 
unit, frequency, route, indication, start and stop dates.  
Any non-pharmacological treatment the patient has received after completion of the Week 25 of 
Study NS-065/NCNP-01-201 will be collected.  The following information will be collected: name 
of treatment, indication, and start and stop date. Prior non-pharmacologic treatment will be 
recorded in source documents and captured in the relevant eCRF.  Physical therapy schedule 
should not change and no new physical therapy should be started during the study.  The need for 
changes to physical therapy should be discussed with the study chair prior to implementation. 
6.1. Prohibited Medications 
Concomitant medication use or changes to GC use are discouraged unless necessary for medical 
management.  Any other experimental/investigational products are prohibited after completion of 
Study NS-065/NCNP-01-201 and throughout participation in this extension study. Patients who 
begin another investigational product will be withdrawn from the study. 
6.2. Allowable Medications 
Investigators may prescribe concomitant medications or treatments deemed necessary to provide 
adequate therapeutic and supportive care.  Specifically, patients should receive full medical care 
during the study, including transfusions of blood and blood products, treatments with antibiotics, 
anti-emetics, anti-diarrheals, analgesics, topical or inhaled steroids, and other care as deemed 
appropriate, and in accordance with their institutional guidelines.  All concomitant blood products, 
medications and supplements will be recorded in source documents and in the relevant eCRF. 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 36 of 112 
7. STUDY PROCEDURES 
7.1. Time and Events Schedule 
The schedule of study assessments is described in Table 1; however, a patient can be seen at any 
time for reasons of safety. Study events are divided into the following phases: 
• Informed Consent:  Execution of the Informed Consent/HIPAA authorization/Assent will 
take place prior to or during the Week 24 study visit of Study NS-065/NCNP-01-201. 
• Treatment Phase: The up to 192-week (up to a total of 216 weeks of treatment in Study 
NS-065/NCNP-01-201 and 202) interval of Study NS-065/NCNP-01-202, starting with 
administration of the first dose of study medication at Week 25 and continuing through 
the time of administration of the final dose of study medication.  If the site is selected to 
participate in the separate long-term follow-up program and the patient agrees to 
participate by signing the consent form before the final study visit for the NS-065/NCNP-
01-202 study, then the patient should complete the final study visit and transition to the 
new study. The decision to transition to the long-term follow up program will be made 
jointly by the patient/caregiver and investigator. 
• Post-treatment Follow-up Phase: The up to 192-week Treatment Phase in study 202 (i.e. 
up to 216 weeks for the combined studies NS-065/NCNP-01-201 and 202) is followed by 
a final study visit with comprehensive safety and clinical efficacy assessments at the Week 
217 visit.  A 30-day follow-up interval begins after completion of the week 217 visit and 
ends after a final phone call for collection of any information about adverse events and 
concomitant medications for all participants. This 30-day follow-up interval and final 
phone call will only occur for patients who do not continue into a separate long-term 
follow up program of NS-065/NCNP-01. For subjects who do continue into the long-term 
follow up program, the Week 217 visit or the Early Termination (ET) visit will also serve 
as the baseline visit for the follow-up study.   
Subjects who terminate the 202 study before completing the 216 week treatment period 
will undergo an Early Termination (ET) visit which should include all safety and efficacy 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 37 of 112 
assessments planned for the week 217 visit and they will complete the subsequent 30-day 
follow-up interval.    

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 38 of 112 
Table 1. Schedule of Study Assessments 
Phase 2 Extension Study 
Protocol # NS-065/NCNP-01-202 
NS-065/NCNP- 
201 Study 1 
Treatment Phase 
Week 
24 
25 
25 26 
27 28 29 30 
31 32 33 34 
35 36 
37 38 
39 40 41 42 43 44 45 46 
47 48 49 50 
51 52 53 
Window 
±3 days window 
Consent/Assent 
X 
Inclusion/Exclusion 
X 
X 
X 
Investigative Drug 
Investigative Drug 
Investigational Product Administration 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Safety Assessments 
Safety Assessments 
Weight 
X 
X 
X 
X 
X 
X 
X 
X 
Height 
X 
X 
X 
Vitals 2 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Physical and Neurological Exam 
X 
X 
X 
X 
X 
X 
X 
Medical, Surgical, Medication, and 
Treatment History Review 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Hematology 3 
X 
X 
X 
X 
X 
X 
X 
Chemistry 4 
X 
X 
X 
X 
X 
X 
X 
BNP 
X 
X 
Urinalysis (Random Urine) 5 
X 
X 
X 
X 
X 
X 
X 
Urinalysis (Pre-dose & Post-dose) 
Urine (Cytology)6 
Urinalysis (24hr pooled Urine)7 
X 
12-lead ECG 
X 
X 
X 
Renal & Urinary Bladder Ultrasound8 
Adverse Event review 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Cytokine 9 
X 
X 
X 
anti-dystrophin antibody 
X 
X 
X 
anti-NS-065/NCNP-01 antibody 
X 
X 
X 
Efficacy Assessments 
Efficacy Assessments 
Muscle biopsy (for dystrophin) 
X 
Function and Strength 10 
X 
X 
X 
Pharmacokinetics and Pharmacodynamics 
Pharmacokinetics and Pharmacodynamics 
PK (Blood)11 
X 
X 
X 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 39 of 112 
Serum PD Biomarker 
X 
X 
X 
Phase 2 Extension Study 
Protocol # NS-065/NCNP-01-202 
 
Treatment Phase 
Week 
54 
55 56 57 58 
59 60 61 62 
63 64 
65 66 
67 68 69 70 
71 72 
73 74 
75 76 77 78 
79 80 81 82 
83 84 85 
86 
Window 
±3 days window 
Consent/Assent 
Inclusion/Exclusion 
Investigative Drug 
Investigative Drug 
Investigational Product Administration 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Safety Assessments 
Safety Assessments 
Weight 
X 
X 
X 
X 
X 
X 
X 
X 
Height 
X 
X 
X 
Vitals 2 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Physical and Neurological Exam 
X 
X 
X 
Medical, Surgical, Medication, and 
Treatment History Review 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Hematology 3 
X 
X 
X 
Chemistry 4 
X 
X 
X 
BNP 
X 
Urinalysis (Random Urine) 5 
X 
X 
X 
Urinalysis (Pre-dose & Post-dose) 
Urine (Cytology)6 
Urinalysis (24hr pooled Urine)7 
12-lead ECG 
X 
Renal and Urinary Bladder Ultrasound8 
Adverse Event review 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Cytokine 9 
X 
anti-dystrophin antibody 
X 
anti-NS-065/NCNP-01 antibody 
X 
Efficacy Assessments 
Efficacy Assessments 
Muscle biopsy (for dystrophin) 
Function and Strength 10 
X 
X 
X 
PK and PD 
Pharmacokinetics and Pharmacodynamics 
PK (Blood)11 
X 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 40 of 112 
Serum PD Biomarker 
X 
Phase 2 Extension Study 
Protocol # NS-065/NCNP-01-202 
 
Treatment Phase 
Week 
87 
88 89 90 91 
92 93 94 95 96 97 98 99 100 101 102 103 104 
105 106 107 
108 109 110 111 112 113 114 115 
116 
Window 
±3 days window 
Consent/Assent 
Inclusion/Exclusion 
Investigative Drug 
Investigative Drug 
Investigational Product Administration 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Safety Assessments 
Safety Assessments 
Weight 
X 
X 
X 
X 
X 
X 
X 
Height 
X 
X 
Vitals 2 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Physical and Neurological Exam 
X 
X 
Medical, Surgical, Medication, and 
Treatment History Review 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Hematology 3 
X 
X 
Chemistry 4 
X 
X 
BNP 
X 
Urinalysis (Random Urine) 5 
X 
X 
Urinalysis (Pre-dose & Post-dose) 
Urine (Cytology)6 
Urinalysis (24hr pooled Urine)7 
12-lead ECG 
X 
Renal and Urinary Bladder Ultrasound8 
Adverse Event review 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Cytokine 9 
X 
anti-dystrophin antibody 
X 
anti-NS-065/NCNP-01 antibody 
X 
Efficacy Assessments 
Efficacy Assessments 
Muscle biopsy (for dystrophin) 
Function and Strength 10 
X 
X 
PK and PD 
Pharmacokinetics and Pharmacodynamics 
PK (Blood)11 
X 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 41 of 112 
Serum PD Biomarker 
X 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 42 of 112 
 
Phase 2 Extension Study
Protocol # NS-065/NCNP-01-202
Week 
117 118 119 120 121 122
123
124
125
126
127
128
129
130 131 132
133
134
135
136
137
138
139
140 141 142
143
144
145
146
147
Window
Consent/Assent
Inclusion/Exclusion
Investigative Drug
Investigational Product Administration
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
Safety Assessments
Weight
X
X
X
X
X
X
X
X
Height
X
X
X
Vitals 2
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
Physical and Neurological Exam
X
X
X
Medical, Surgical, Medication, and 
Treatment History Review
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
Hematology 3
X
X
X
Chemistry 4
X
X
X
BNP
X
X
Urinalysis (Random Urine) 5
X
X
X
Urine (Cytology)6
X
X
Urinalysis (24hr pooled Urine)7
12-lead ECG
X
X
Renal Ultrasound8
X
X
Adverse Event review
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
Cytokine 9
anti-dystrophin antibody
X
X
anti-NS-065/NCNP-01 antibody
X
X
Efficacy Asssessments
Muscle biopsy (for dystrophin)
Function and Strength 10
X
X
X
Pharmacokinetics and Pharmacodyn
PK (Blood)11
Serum PD Biomarker
X
X
Treatment Phase
±3 days window
Investigative Drug
Safety Assessments
Efficacy Asssessments
Pharmacokinetics and Pharmacodynamics

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 43 of 112 
 
Phase 2 Extension Study
Protocol # NS-065/NCNP-01-202
Week 
148
149
150
151
152
153
154
155
156
157
158
159
160
161
162
163
164
165
166
167
168
169
170
171
172
173
174
175
176
Window
Consent/Assent
Inclusion/Exclusion
Investigative Drug
Investigational Product Administration
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
Safety Assessments
Weight
X
X
X
X
X
X
X
Height
X
X
Vitals 2
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
Physical and Neurological Exam
X
X
Medical, Surgical, Medication, and 
Treatment History Review
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
Hematology 3
X
X
Chemistry 4
X
X
BNP
X
Urinalysis (Random Urine) 5
X
X
Urine (Cytology)6
X
X
Urinalysis (24hr pooled Urine)7
12-lead ECG
X
Renal Ultrasound8
X
X
Adverse Event review
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
Cytokine 9
anti-dystrophin antibody
X
anti-NS-065/NCNP-01 antibody
X
Efficacy Asssessments
Muscle biopsy (for dystrophin)
Function and Strength 10
X
X
Pharmacokinetics and Pharmacodyn
PK (Blood)11
Serum PD Biomarker
X
Treatment Phase
±3 days window
Investigative Drug
Safety Assessments
Efficacy Asssessments
Pharmacokinetics and Pharmacodynamics

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 44 of 112 
 
Phase 2 Extension Study
Protocol # NS-065/NCNP-01-202
Week 
177
178
179
180
181
182
183
184
185
186
187
188
189
190
191
192
193
194
195
196
197
198
199
200
201
202
203
204
205
Window
Consent/Assent
Inclusion/Exclusion
Investigative Drug
Investigational Product 
Administration
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
Safety Assessments
Weight
X
X
X
X
X
X
X
X
Height
X
X
X
Vitals 2
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
Physical and Neurological Exam
X
X
X
Medical, Surgical, Medication, and 
Treatment History Review
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
Hematology 3
X
X
X
Chemistry 4
X
X
X
BNP
X
Urinalysis (Random Urine) 5
X
X
X
Urine (Cytology)6
X
X
X
Urinalysis (24hr pooled Urine)7
12-lead ECG
X
Renal Ultrasound8
X
X
X
Adverse Event review
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
Cytokine 9
anti-dystrophin antibody
X
anti-NS-065/NCNP-01 antibody
X
Efficacy Asssessments
Muscle biopsy (for dystrophin)
Function and Strength 10
X
X
X
Pharmacokinetics and Pharmacody
PK (Blood)11
Serum PD Biomarker
X
Treatment Phase
±3 days window
Investigative Drug
Safety Assessments
Efficacy Asssessments
Pharmacokinetics and Pharmacodynamics

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 45 of 112 
 
Phase 2 Extension Study
Protocol # NS-065/NCNP-01-202
Week 
206
207
208
209
210
211
212
213
214
215
216
217 or ET
30-day Follow-
up13
Window
Consent/Assent
Inclusion/Exclusion
Investigative Drug
Investigational Product 
Administration
X
X
X
X
X
X
X
X
X
X
X
X12
Safety Assessments
Weight
X
X
X
Height
X
Vitals 2
X
X
X
X
X
X
X
X
X
X
X
X
Physical and Neurological Exam
X
Medical, Surgical, Medication, and 
Treatment History Review
X
X
X
X
X
X
X
X
X
X
X
X
X
Hematology 3
X
Chemistry 4
X
BNP
X
Urinalysis (Random Urine) 5
X
Urine (Cytology)6
X
Urinalysis (24hr pooled Urine)7
12-lead ECG
X
Renal Ultrasound8
X
Adverse Event review
X
X
X
X
X
X
X
X
X
X
X
X
X
Cytokine 9
X
anti-dystrophin antibody
X
anti-NS-065/NCNP-01 antibody
X
Efficacy Asssessments
Muscle biopsy (for dystrophin)
Function and Strength 10
X
Pharmacokinetics and Pharmacody
PK (Blood)11
Serum PD Biomarker
X
Post-Treatment Phase
Treatment Phase
±3 days window
Investigative Drug
Safety Assessments
Efficacy Asssessments
Pharmacokinetics and Pharmacodynamics

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 46 of 112 
 
 
 
 
 
 
 
 
 
1. Week 25 of Study NS-065/NCNP-01-201 will also serve as Week 25 for first dose of Study NS-065/NCNP-01-202.  End of study procedures of Study NS-065/NCNP-01-
201 must be completed prior to dosing in Extension Study NS-065/NCNP-01-202
2. Perform vitals at pre-dose, at the end of infusion (+10 mins) and 1 hr (+/- 5 mins)
3. Hematology: RBC, Hb, Ht, Ret, MCV, MCH, MCHC, WBC, PLT, WBC Differential Fibrinogen, aPTT, PT-INR
4. Chemistry: Na, K, Cl, Ca, IP, BUN, Serum Creatinine, Cystatin C, AST, ALT, GGT, ALP, Haptoglobin, LDH/LDH isozyme, CK, Total bilirubin (Direct/Indirect), Total 
protein, Albumin, Albumin to globulin ratio, Total cholesterol, Triglyceride, Blood glucose, C-reactive protein
5. Urinalysis (Random Urine): Urine glucose, Urine blood, Urine urobilinogen, Urine specific gravity, Urine osmolarity, Urinary sediment (RBC, WBC, Cast), Urine protein 
(benzothonium chloride method), Albumin in urine, NAG in urine, α1-microgloblin in urine, Urine Creatnine
6. Urine Cytology is collected along with pre-dose Urinalysis. Following Protocol Amendment 9.0 approval, the assessment should be performed at the next clinic visit, 
then every 6 months thereafter (i.e. Visits 133, 157, 181 and 193 or Visits 145, 169,  193, 205 and 217). Urine Cytology is collected at Visit 217 for all subjects.                 
7. 24hr pooled Urine in Study NS-065/NCNP-01-201 only: Urine protein (benzothonium chloride method) , Albumin in urine, NAG in urine, α1-microgloblin in urine, Urine Cr, 
Urine Na, Urine K, Urine Cl, Urine IP, Uric acid in urine, β2-microgloblin in urine and PK  
8. Renal Ultrasound includes imaging of kidneys,  bladder and ureters. Following Protocol Amendment 9.0 approval, the assessment should be performed at the next clinic 
visit, then every 6 months thereafter (i.e. Visits 133, 157, 181 and 193 or Visits 145, 169, 193, 205 and 217). Renal Ultrasound is collected at Visit 217 for all subjects.       
9. Cytokine: IL-6, TNF-α, MCP-1 at pre-dose and 8hr (±30min) after end of infusion
10. Function and Strength: Time to Stand (TTSTAND), Time to Climb (TTCLIMB), Time to run/walk 10 meters (TTRW), Six-minute walk Test (6MWT), North Start 
Ambulatory Assessment (NSAA), Quantitative Muscle Testing (QMT) and Ulnar Length
11. PK sampling timepoints: pre-dose, 1hr (+/- 5 mins) and 8hr (+/- 5 mins) after the end of the infusion 
12. Only for subjects continuing in separate long-term follow-up study                                                                                                                                                      
13. Ony subjects who ET or do not continue in to separate long-term follow-up study
Notes: 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 47 of 112 
   
7.2. Informed Consent 
Each patient’s parent or legal guardian will receive an explanation of the nature and purposes of 
the study from the Investigator or designee.  The Investigator or designee will ensure the study is 
appropriate for the patient.  Consent must be obtained in accordance with the principles outlined 
in the current version of the Declaration of Helsinki.  The patient’s parent or guardian will confirm 
that s/he understands that the study is for research purposes only and that it may not provide any 
therapeutic benefit to the individual.  Each patient’s parent or guardian will confirm that s/he 
understands that the patient is free to withdraw from the study at any time without prejudice.  The 
Investigator or designee will review the elements of the HIPAA and Protected Health Information 
(PHI) with each patient’s parent or guardian and each patient’s parent or guardian will confirm 
that s/he understands HIPAA authorization and PHI.  The Investigator (or designated staff) will 
obtain the written informed consent and HIPAA authorization on the approved ICF by the 
appropriate IRB/REB at each site, from the patient’s parent or guardian prior to any study-related 
procedures, including agreement to discontinuation of any prohibited medications, prior to the start 
of the study. The written assent of children will be obtained per individual site guidelines.  
The ICF must be dated and signed by the Investigator or designee and the patient’s legal 
representative and the original signed consent form must be kept by the Investigator in the study 
patient’s file.  “Legal representative” means an individual or judicial or other body authorized 
under applicable law to consent on behalf of a prospective study patient to the patient’s 
participation in the procedure(s) involved in the research.  The study patient’s legal representative 
will receive a copy of the signed consent form. 
If the ICF is amended during the study, the Investigator must follow all applicable regulatory 
requirements pertaining to all new patients and repeat the consent process with the amended ICF 
for any ongoing patients. 
7.3. Assignment of Patient Identification Number 
Study NS-065/NCNP-01-202 participation begins once written informed consent/Assent is 
obtained from the parent/legal guardian for a patient before any study-specific procedures are 
performed.  

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 48 of 112 
Following the signing of the written ICF/Assent Form, patients maintain their Study NS-
065/NCNP-01-201 unique, site-specific, 6-digit patient identification number.  All data will be 
identified using the unique patient identification number.   The assigned patient identification 
number will be retained through enrollment and throughout participation in the study.   
7.4. Demographics 
Demographics will be collected during the Dose-finding Study NS-065/NCNP-01-201 and will 
remain in the EDC database for Extension Study NS-065/NCNP-01-202.   
7.5. Medical History 
Patient medical, surgical, medication and treatment history will be reviewed from Study NS-
065/NCNP-01-201 and reviewed throughout the study. The dates and descriptions of past events 
will be documented in source documents and captured in the relevant eCRF. 
7.6. Weight and Height 
Height and weight will be collected at the visits specified in Table 1. Standing height will be 
collected with the patient barefoot (without shoes).  The participant’s legs should be kept as close 
together as possible and the heels should be placed back as close to the wall as possible.  Participant 
may hold on to an object to facilitate balance.  If a participant is non-ambulant, the ulnar length 
will be used to calculate height. Ulnar length will be measured by the CE during functional 
assessments (see Section 7.14.7). Weight will be collected with the patient barefoot and wearing 
light-weight clothes.  Height and weight should take approximately 2 minutes and are not 
associated with any risks. These measurements are routinely performed during standard clinical 
examinations of patients with DMD.  Weight in kilograms (kg) and height in centimeters (cm) will 
be documented in source documents and captured in the relevant eCRF. 
7.7. Vital Signs 
Vital signs will be performed at each study visit as specified in Table 1. For each visit that includes 
a study drug administration, vital signs will be performed at pre-dose, at the end of infusion (+10 
mins) (approximately 1 hour after initiation of infusion), and 2 hours (+/- 5 mins) after initiation 
of infusion.  If a clinically significant change from pre-dose is observed at 2 hours after initiation 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 49 of 112 
of infusion, the parameter is followed every 30 mins (+/- 5 mins) until return to no clinical 
significant change from pre-dose.  
Vital signs will include the following: 
• Systolic blood pressure 
• Diastolic blood pressure  
• Heart rate 
• Temperature 
Vital signs will be documented in source documents and captured in the relevant eCRF. Any 
clinically significant changes noted by the investigator should be reported as an adverse event and 
should be addressed as clinically appropriate. 
The site PI and the home infusion nurse will discuss vital sign parameters for each participant 
individually who is eligible and has elected to receive home infusions.  To develop these 
guidelines, the site PI will take into consideration the range of clinically safe vital signs that have 
been observed from the participant during past study site infusions.  The home infusion nurse will 
consult with the site investigator for any confirmed vital signs outside of these parameters (low or 
high). 
7.8. Physical and Neurological Examination 
The physical examination will be performed at the visits specified in Table 1 to assess any changes 
in physical presentation and symptoms. Physical examinations will include an assessment of the 
following: 
• General appearance 
• HEENT (head, ears, eyes, nose, and throat) 
• Skin 
• Lymph nodes 
• Heart, including rhythm, heart sounds and presence of cardiac abnormalities 
• Lungs 
• Abdomen 
• Extremities/joints 
• Nervous system 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 50 of 112 
• Any additional assessments necessary to establish baseline status or evaluate symptoms or 
adverse experiences 
Documentation of the physical examination findings will be included in the source documentation 
at the clinical site.  Only changes from baseline physical examination findings that meet the 
definition of an adverse event will be recorded on the adverse event page of the eCRF. 
7.9. Renal and Urinary Bladder Ultrasound 
Renal ultrasound will include imaging of the kidneys, ureters, and bladder and be performed at 
visits specified in the schedule of study assessments. Ultrasound of the kidneys, ureters and 
bladder will be assessed by local urologists or other trained medical professionals. 
7.10. 
Adverse Events and Serious Adverse Events 
Investigators will assess the occurrence of AEs and SAEs each study visit or patient contact during 
the study.  AEs and SAEs may be reported by the patient/parent, discovered upon questioning, 
detected during examinations or review of test and lab results. AEs and SAEs should be 
documented in the source documents and the relevant eCRF with a full description including the 
nature, date and time of onset and resolution, determination of seriousness, severity, causality, 
corrective treatment, and outcome. Refer to Section 9 for safety procedures and reporting. 
7.11. 
12-Lead Electrocardiograms 
A standard 12 lead ECG will be performed at the visits specified in the Schedule of Study 
Assessments (Table 1).  ECG collection will be preceded by a 10-minute rest time during which 
the patient will remain in the supine position.  At all time-points, ECGs will be collected prior to 
blood collection.  ECG results will be based on machine readings and interpretation by local 
cardiologist or other trained medical professionals. ECG results will be recorded in the eCRF. 
7.12. 
Clinical Laboratory Tests 
Clinical laboratory assessments will be performed at visits specified in the Schedule of 
Assessments table (Table 1).  Any blood sampling that occurs during the IP infusion should be 
collected from a location away from the IP infusion placement (i.e. opposite arm). 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 51 of 112 
7.12.1. Sample Collection, Storage, and Shipping 
Each patient will have blood drawn and urine collected for the blood and urine laboratory safety 
assessments as listed/described in the sections below, including hematology, chemistry, BNP, 
urinalysis, urine cytology, cytokines, anti-dystrophin antibody, and anti-NS-065/NCNP-01 
antibody. 
 
Laboratory safety assessments will be performed at the visits specified in Table 1.  Samples will 
be collected by a trained member of the study team.  This assessment is associated with the usual 
risks of a blood draw which include pain, bruise at the point where the blood is taken, redness and 
swelling of the vein, infection, and a rare risk of fainting. In order to decrease any of these possible 
risks the sites will employ pediatric trained staff and will use a numbing cream, if desired by the 
patient, to reduce the risk of pain.  
 
All blood and urine samples will be sent to the designated central laboratory for testing unless 
otherwise noted.  The procedures for the collection, handling, and shipping of laboratory samples 
will be specified in the Laboratory manual.  Clinical laboratory tests are listed in Error! Reference 
source not found.. 
 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
_____________________________________________________________________________________________ 
NS Pharma, Inc. – CONFIDENTIAL  
 
Page 52 of 112 
Table 2. Clinical Laboratory Tests 
Hematology, Chemistry, Urinalysis and Urine Cytology – Safety Labs 
• 
Hematology 
o 
Red blood cell count 
o 
Hemoglobin  
o 
Hematocrit 
o 
Reticulocyte count 
o 
Mean corpuscular volume 
o 
Mean corpuscular hemoglobin 
o 
Mean corpuscular hemoglobin concentration 
 
o 
White blood cell count 
o 
White blood cell differential 
o 
Platelet count 
o 
Fibrinogen  
o 
Activated partial thromboplastin time 
o 
Prothrombin international normalization 
ratio 
• 
Blood Chemistry 
 
o 
Sodium 
o 
Potassium 
o 
Chloride 
o 
Calcium 
o 
Inorganic Phosphorus 
o 
Blood Urea Nitrogen 
o 
Creatinine 
o 
Cystatin C  
o 
Aspartate aminotransferase  
o 
Alanine aminotransferase   
o 
Gamma-glutamyl transferase 
o 
Alkaline phosphatase 
o 
Haptoglobin  
o 
Lactate dehydrogenase 
o 
Creatine kinase 
o 
Total bilirubin (Direct/Indirect)  
o 
Total protein 
o 
Albumin 
o 
Albumin to globulin ratio 
o 
Total cholesterol  
o 
Triglyceride  
o 
Blood glucose   
o 
C-reactive protein 
• 
Brain Natriuretic Protein (BNP) 
• 
Urinalysis 
Urinalysis includes:  
o 
Urine glucose 
o 
Urine blood 
o 
Urine urobilinogen 
o 
Urine specific gravity 
o 
Urine osmolality 
o 
Urinary sediment (erythrocytes, WBC, Casts, 
epithelium, crystals) 
o 
Urine protein (benzethonium chloride 
method) 
o 
Urine protein (benzethonium chloride 
method)  
o 
Urine microalbumin 
o 
Urine N-acetyl-beta-D-glucosaminidase 
o 
Urine α1-microglobulin 
o 
Urine Creatinine 
o 
Urine Cytology 
7.12.2. Cytokine Testing 
Cytokine testing will be performed at visits as specified in the Schedule of Assessments (Table 1).  
The following tests will be performed: IL-6, TNF-α, MCP-1.  Blood will be drawn from patients 
at pre-dose and 9 hours (+/- 30min) after initiation of infusion at Weeks 37, 49, 73, 97, and the 
early termination. 

NCT03167255 
NS-065/NCNP-01 Injection 
 
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Clinical Study Protocol  NS-065/NCNP-01-202   
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7.12.3. Anti-Dystrophin Antibody 
Anti-dystrophin antibody testing will be performed on serum samples collected predose during the 
visits specified in the Schedule of Assessments (Table 1).  Samples will be analyzed by Shin 
Nippon Biomedical Laboratories, Ltd. (Japan).  
7.12.4. Anti-NS-065/NCNP-01 Antibody 
Anti-NS-065/NCNP-01 antibody testing will be performed on serum samples collected pre-dose 
during the visits specified in the Schedule of Assessments (Table 1).  Samples will be analyzed by 
Shin Nippon Biomedical Laboratories, Ltd. (Japan).  
7.13. 
Pharmacodynamics and Efficacy Assessments 
7.13.1. Serum Pharmacodynamic Biomarkers 
Serum samples will be collected and stored for future pharmacodynamic biomarker studies.  
7.14. 
Function and Strength 
All function and strength testing will be performed by a trained clinical evaluator (CE). Assistive 
devices may be utilized during the testing as specified in the study Manual of Operations.  The 
same CE should perform testing on the same patient throughout the study when possible. 
7.14.1. Time to Stand (TTSTAND) - Primary Efficacy Endpoint 
TTSTAND will be performed by a CE at visits specified in the schedule of study assessments 
(Table 1).  This test will assess the time it takes the patient to go from lying flat on the floor to 
standing and is administered as part of the NSAA (see Section 7.14.4).  This test should take 
approximately 1 minute and is not associated with any risks.  This test is routinely performed 
during standard clinical examinations of patients with DMD.  The number of seconds required to 
perform the test and the 6-point rating scale of how the patient attains the standing position will be 
documented in source documents and captured in the relevant eCRF. 
7.14.2. Time to Run/Walk 10 Meters (TTRW) 
TTRW will be performed by a CE at visits specified in the schedule of study assessments (Table 
1).  This test will assess the time it takes the patient to walk/run 10 meters including a 6-point 
rating scale for quality of the run/walk administered as part of the NSAA (see Section 7.14.4). This 
assessment should take 2 minutes and can be associated with falls; however, these are infrequently 

NCT03167255 
NS-065/NCNP-01 Injection 
 
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Clinical Study Protocol  NS-065/NCNP-01-202   
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Page 54 of 112 
reported.  The number of seconds required to perform the test will be documented in source 
documents and captured in the relevant eCRF. 
7.14.3. Time to climb 4 stairs (TTCLIMB) 
TTCLIMB will be performed by a CE at visits specified in the schedule of study assessments 
(Table 1).  This test will assess the time it takes the patient to climb 4 stairs (23) including a 6-
point rating scale to assess how the patient negotiates the stairs administered as part of the NSAA 
(see Section 7.14.4).  This test should take approximately 1 minute and is not associated with any 
risks.  The number of seconds required to perform the test will be documented in source documents 
and captured in the relevant eCRF. 
7.14.4. North Star Ambulatory Assessment (NSAA) 
NSAA will be performed by a CE at visits specified in the schedule of study assessments (Table 
1).  The NSAA is a clinician rated, 17-item, functional scale originally designed for ambulant boys 
with DMD who are able to ambulate at least 10 meters (24). This evaluation tool assesses 
functional activities including standing, getting up from the floor, negotiating steps, hopping, and 
running.  The assessment is based on a 3-point rating scale of 2 = ability to perform the test 
normally, 1 = modified method or assistance to perform test, 0 = unable to perform the test. Thus, 
total score can range from 0 (completely non-ambulant) to 34 no impairment on these assessments. 
Individual test item scores and total score will be recorded in source documents and in the relevant 
eCRF.  This test should take approximately 10 minutes and is not associated with any risks.  The 
NSAA should be administered before the 6MWT at each occurrence.  
7.14.5. Six-minute Walk Test (6MWT) 
6MWT will be performed by a CE at visits specified in the schedule of study assessments (Table 
1).  The 6MWT is a widely used and accepted test in numerous diseases.  The version of the 6MWT 
adapted for use in DMD will be used (25).  To perform the test, two points (cones) are set 25 
meters apart and patients are asked to walk back and forth, between the cones quickly and safely 
for 6 minutes.  The total distance in meters that the patient walks in 6 minutes is recorded. This 
test is considered a simple, standardized, low-technology and cost-effective means of clinically 
assessing 1) functional motor status and 2) integrated and global responses to exercise. The CE 
will measure the number of steps taken by the patient for the first 50 meters and total meters walked 
in 6 minutes.  This test should take approximately 10 minutes.  The 6MWT may cause feelings of 

NCT03167255 
NS-065/NCNP-01 Injection 
 
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Clinical Study Protocol  NS-065/NCNP-01-202   
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Page 55 of 112 
pressure or pain in the patient’s chest, difficulty breathing, and shortness of breath.  The 6MWT 
may also cause an increased risk of falling and muscle cramping. 
7.14.6. Quantitative Muscle Testing (QMT) 
QMT will be performed by a CE at visits specified in the schedule of study assessments (Table 1).  
QMT assessments are designed to measure muscle force production during an isometric 
contraction.  QMT is a well-established method for measuring muscle weakness in neuromuscular 
disease (26).  Patients will be placed on an examination table with a back-support system to 
eliminate the need for manual back stabilization.  Following a single practice administration, each 
patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values 
used for data analysis).  QMT will be performed by recording force in pounds through a direct 
computer interface with a strain gauge.  Testing positions and test order will be standardized. 
Bilateral testing of the muscle groups listed below will be performed: 
• Handgrip 
• Elbow flexors (biceps) 
• Elbow extensors (triceps) 
• Knee flexors (hamstrings) 
• Knee extensors (quadriceps) 
7.14.7. Ulnar Length 
Ulnar length will be measured by a CE at visits specified in the schedule of study assessments 
(Table 1).  In the event that a patient becomes non-ambulant the ulnar length will be utilized to 
impute height.   
7.15. 
Pharmacokinetic Assessments 
7.15.1. Collection and Assessment of Pharmacokinetic Samples 
Blood samples for pharmacokinetic (PK) assessments will be taken at visits specified in the study 
assessment table (Table 1).  PK sampling times are measured from the start of infusion. Infusion 
is expected to take approximately 1 hour to complete. PK samples will be analyzed in the event 
that anti-NS-065/NCNP-01 antibodies are detected in order to understand the impact of 

NCT03167255 
NS-065/NCNP-01 Injection 
 
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Clinical Study Protocol  NS-065/NCNP-01-202   
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Page 56 of 112 
immunogenicity on plasma levels of the drug.  Blood will be drawn from patients at pre-dose, 2 
hours (+/- 5 min) and 9 hours (+/- 5 min) after initiation of infusion at Weeks 37, 49, 73, and 97.  
7.15.2. Shipment of Pharmacokinetic Samples 
Plasma PK samples will be shipped frozen on dry ice according to instructions provided in the 
Laboratory Manual. 
7.16. 
Total Blood Volume of Clinical Laboratory and Pharmacokinetic Samples 
The total number of venipunctures and total volume of whole blood collected during the study will 
be limited to that needed for safety monitoring and efficacy assessments.  Total scheduled whole 
blood volume collected over the study duration is not to exceed 321.1 mL for each patient.  In the 
event of an SAE or an AE, additional blood may be taken for safety assessments based on timing 
and clinical judgement from PI, study chair and medical monitor. A breakdown of total volume of 
blood collected is summarized in Error! Reference source not found.. 
 
 
 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
v11.0 21Jul2020 
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Page 57 of 112 
 
Table 3. Blood Sample Number and Volume 
Tests
WK 
25c
WK 
29
WK 
33
WK 
37
WK 
41
WK 
45
WK 
49
WK 
61
WK 
73
WK 
85
WK 
97
WK 
109
WK 
121
WK 
133
WK 
145
WK 
157
WK 
169
WK 
181
WK 
193
WK 
205
WK 217 
or ET
Total mL 
of Blood
Safety
10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6 10.6
10.6
222.6
BNP
3.0
3.0
3.0
3.0
3.0
3.0
3.0
3.0
3.0
27.0
PD
1.0
1.0
1.0
1.0
1.0
1.0
1.0
1.0
1.0
9.0
PKa,b
6.0
6.0
6.0
6.0
24.0
Cytokine
4.0
4.0
4.0
4.0
16.0
Anti-dystrophin 
& Anti-NS-
065/NCNP-01
2.5
2.5
2.5
2.5
2.5
2.5
2.5
2.5
2.5
22.5
321.1
a. Blood samples for PK analysis are collected according to the schedule in Section 7.15
b. For SAEs, additional blood draws for PK and immunogenicity will be based on timing and clinical judgement from PI, study chair and medical 
monitor.
c. Blood samples for safety analysis at Week 25 will be collected at the Week 25 of Study NS-065/NCNP-01-201.
d. Early termination (ET) blood draw volumes based on clinical judgement from PI, study chair and medical monitor.
e. Total volume of blood per patient does not include any additional testing that may be required for follow-up of AEs or re-testing and follow-up of 
abnormal laboratory results.  
Total volume based on maximum expected blood draw from Week 25 through Week 217.
Approximately total volume of blood per patiente

NCT03167255 
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8. STUDY ACTIVITIES 
8.1.  Treatment Phase 
In anticipation of the Week 50 study visit, which is the first study visit where optional home 
infusion could take place, the option of home infusion can be introduced to the patient by the study 
physician. Home infusion, which is limited to selected visits described below, is not required for 
participation in this study. The option of home infusion should be considered if the study physician 
believes that it will help the patient to derive safety, benefit and convenience from participation in 
the trial.  The routine study visits that are eligible to be conducted in the home are limited to those 
whose study procedures do not extend beyond the following list:  investigational product 
administration, vital signs collection, review of medical, surgical, medication and treatment 
history, and AE review. Specifically, visits 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 105, 
109, 113, 117, 121, 125, 129, 133, 137, 141, 145, 149, 153, 157, 161, 165, 169, 173, 177, 181, 
185, 189, 193, 197, 201, 205, 209, 213 and 217 (or early termination) must be conducted at the 
study site and are not eligible for home infusion. Participation in optional home infusion requires 
written approval from the study sponsor, study chair, medical monitor and site PI. As advised by 
the study chair, medical monitor, and site PI, infusions taking place outside of the primary study 
site (e.g. home infusion) would only be considered starting at the Week 50 study visit or after, if 
the participant had not experienced any significant adverse events related to infusion of the 
investigational product.  Patients must also reside in a municipality or region with access to 
emergency medical services (such as 911) to participate in home infusions. The study sponsor 
approval will only occur following endorsement by the Nippon Shinyaku Chief Medical Officer. 
The safety monitoring by the site PI, sponsor, study chair and medical monitor will continue 
throughout the treatment phase for all study visits, without regard for whether the infusion occurs 
at the study site or the home.  Furthermore, the site PI is responsible for immediate contact with 
the study chair and medical monitor for any safety concern, without regard for whether the infusion 
occurs at the study site or the home. 
All visit activities are to occur in the order in which they are listed below. 

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8.1.1. Day of First Infusion (Week 25) 
The Week 25 study visit of Study NS-065/NCNP-01-201 shall also serve as the Week 25 dose 
visit if the patient wishes to continue into Extension Study NS-065/NCNP-01-202.  In order to 
accommodate patient recovery following the scheduled biopsy during the Week 25 of Study NS-
065/NCNP-01-201, patients will be allowed a visit window of 7 days (+/- 3 days) from the Week 
24 infusion to complete both the end of study procedures and the first infusion in the extension 
study.  All Week 25 study assessments of the NS-065/NCNP-01-201 study, including muscle 
biopsy, must be completed prior to first infusion in this extension study.  Patients can receive the 
first infusion of this extension study on the same day as the Week 25 study visit of NS-065/NCNP-
01-201, as long as all study assessments (including muscle biopsy) of NS-065/NCNP-01-201 are 
completed prior to infusion.  Initial and Week 25 study assessments of NS-065/NCNP-01-201 will 
be used as baseline values for this extension study. 
At Week 25 of Extension Study NS-065/NCNP-01-202, the following activities will occur: 
• Inclusion/Exclusion Confirmation and Review 
• Review of Medical, Surgical, Medication, and Treatment History (confirmation of 
concomitant medications and other treatments; any changes will be noted)* 
• AE Review* 
• Vital signs 
• Blood draw for BNP (note this assessment may be collected with the other laboratory 
assessment draws for week 25 as part of Study NS-065/NCNP-01-201) 
• Investigational product administration 
*If Week 25 of the Extension Study occurs on the same day as Week 25 of NS-065/NCNP-
01-201, these assessments do not need to be repeated. 
8.1.2. Routine Infusion Visits  
These visits will occur at: Weeks 26-28, 30-32, 34-36, 38-40, 42-44, 46-48, 50-52, 54-56, 58-60, 
62-64, 66-68, 70-72, 74-76, 78-80, 82-84, 86-88, 90-92, 94-96, 98-100, 102-104, 106-108, 110-
112, 114-116, 118-120, 122-124, 126-128, 130-132, 134-136, 138-140, 142-144, 146-148, 150-
152, 154-156, 158-160, 162-164, 166-168, 170-172, 174-176, 178-180, 182-184, 186-188, 190-
192, 194-196, 198-200, 202-204, 206-208, 210-212 and 214-216. 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
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At these visits the following activities will occur: 
• Review of Medical, Surgical, Medication and Treatment History 
• AE Review 
• Vital Signs 
• Investigational product administration 
8.1.3. Weeks 29, 33, 41, and 45 
At these visits the following activities will occur: 
• Review of Medical, Surgical, Medication, and Treatment History 
• AE Review 
• Weight   
• Vital Signs 
• Physical and Neurological Exam 
• Hematology  
• Chemistry  
• Urinalysis (Random Urine) 
• Investigational product administration 
8.1.4. Week 37, 49, 73, and 97 
At the visit the following activities will occur: 
• Review of Medical, Surgical, Medication, and Treatment History 
• AE Review 
• Height & Weight  
• Vital Signs 
• Physical and Neurological Exam 
• 12-Lead ECG 
• Function and Strength: 
o TTSTAND 
o TTRW 
o TTCLIMB 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
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o NSAA 
o 6MWT 
o QMT 
o Ulnar Length 
• Hematology  
• Chemistry  
• Blood draw for BNP (at Week 49, 73, and 97) 
• Urinalysis (Random Urine) 
• Cytokine  
• Anti-dystrophin antibody (pre-dose) 
• Anti-NS-065/NCNP-01 antibody (pre-dose) 
• Serum PD biomarker 
• Investigational product administration 
• PK Blood 
8.1.5. Routine Infusion Visits with Weight Collection  
These visits occur at: Weeks 53, 57, 65, 69, 77, 81, 89, 93, 101, 105, 113, 117, 125, 129, 137, 
141, 149, 153, 161, 165, 173, 177, 185, 189, 197, 201, 209, and 213. At these visits the following 
activities will occur: 
• Review of Medical, Surgical, Medication, and Treatment History 
• AE Review  
• Weight  
• Vital Signs 
• Investigational product administration 
8.1.6. Weeks 61, 85, 109, 133, 157, 181, and 205 
At these visits the following activities will occur: 
• Review of Medical, Surgical, Medication, and Treatment History  
• AE Review 
• Height & Weight,  

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Clinical Study Protocol  NS-065/NCNP-01-202   
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• Vital Signs 
• Physical and Neurological Exam 
• Function and Strength: 
o TTSTAND 
o TTRW 
o TTCLIMB 
o NSAA 
o 6MWT 
o QMT 
o Ulnar Length 
• Hematology  
• Chemistry 
• Urinalysis (predose and first void taken within 5 hours postdose) 
• Urine Cytology (pre-dose) (133, 157 and 181 only) 
• Renal and Urinary Bladder Ultrasound (Visits 133, 157 and 181 only) 
• Investigational product administration 
8.1.7. Week 121, 145, 169, and 193 
At the visit the following activities will occur: 
• Review of Medical, Surgical, Medication, and Treatment History 
• AE Review 
• Height & Weight  
• Vital Signs 
• Physical and Neurological Exam 
• 12-Lead ECG 
• Function and Strength: 
o TTSTAND 
o TTRW 
o TTCLIMB 
o NSAA 

NCT03167255 
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Clinical Study Protocol  NS-065/NCNP-01-202   
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o 6MWT 
o QMT 
o Ulnar Length 
• Hematology  
• Chemistry  
• Blood draw for BNP  
• Urinalysis (predose and first void taken within 5 hours postdose) 
• Urine Cytology (pre-dose) (Visits 145 and 169 only) 
• Renal and Urinary Bladder Ultrasound (Visits 145 and 169 only) 
• Anti-dystrophin antibody (pre-dose) 
• Anti-NS-065/NCNP-01 antibody (pre-dose) 
• Serum PD biomarker 
• Investigational product administration 
8.2. Post-Treatment Phase 
8.2.1. Week 217 or Early Termination Visit 
The following activities will occur at the end of the treatment (Week 217) for all patients.  If a 
patient withdraws from the study or is withdrawn by the site PI (Early Termination Visit) the 
activities listed below should be completed.  All visit activities are to occur in the order in which 
they are listed below. 
• Review of Medical, Surgical, Medication, and Treatment History 
• AE Review 
• Height & Weight  
• Vital Signs 
• Physical and Neurological Exam 
• 12-Lead ECG 
• Function and Strength: 
o TTSTAND 
o TTRW 

NCT03167255 
NS-065/NCNP-01 Injection 
 
NS Pharma, Inc. 
Clinical Study Protocol  NS-065/NCNP-01-202   
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o TTCLIMB 
o NSAA 
o 6MWT 
o QMT 
o Ulnar Length 
• Hematology  
• Chemistry  
• Blood draw for BNP  
• Urinalysis (Random Urine) 
• Urine Cytology  
• Renal and Urinary Bladder Ultrasound 
• Serum PD biomarker 
8.2.2. Follow-Up Phone Call 
Participants who do not enroll in a separate long-term follow up program of NS-065/NCNP-01 
will have a phone call with a member of the site study staff, 30 days (+3 days) following Week 
217/ET visit, to assess adverse events and document changes in concomitant medications. Any 
AEs that are unresolved at the patient’s last AE assessment in the study are followed up by the 
site PI or designee for as long as medically indicated, but without further recording in the eCRF.  
The CRO retains the right to request additional information for any patient with ongoing AEs at 
the end of the study, if judged necessary. 
8.2.4. Early Termination or Withdrawal from the Study 
A patient (or the legal guardian acting on behalf of the patient) is free to withdraw consent and 
discontinue participation in the study at any time, without prejudice to further treatment according 
to standard clinical practice. Study participation may be discontinued at any time at the discretion 
of the site PI or Sponsor. The following may be justifiable reasons for removing a patient:  
• Withdrawal of consent by the patient/legal guardian; 
• Failure to comply with the protocol; 
• Lost-to-follow-up; 

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• Illness, condition, or procedural complication (including adverse events) affecting the 
patient’s ability to participate or requiring prohibited medication; 
• In the Investigator’s judgment, it is deemed in the best interest of the patient to 
discontinue his/her participation in the study; 
• The study drug becomes commercially available 
• The Investigator, Sponsor, DSMB and/or regulatory authority terminates the study; or 
• Any other reason. 
A Patient Completion/Discontinuation eCRF describing the reason for discontinuation must be 
completed, for any discontinued or withdrawn patient regardless of reason. If a patient withdraws 
from the study or if the study is prematurely terminated, the site PI or designee will contact the 
patient or the patient’s legal guardian within 30 days after withdrawal or termination to assess any 
AEs. The site PI will be asked to follow all SAEs until the event returns to baseline or until the 
site PI determines that follow-up is no longer medically necessary.  Patients who are withdrawn 
from the study may not re-enter. 
If a patient is lost to follow-up, every reasonable effort must be made by the clinical site personnel 
to contact the patient and determine the reason for discontinuation/withdrawal (including 
assessment of any AEs reported by the patient/caregiver). The measures taken to follow-up must 
be documented in source documents. 
8.2.5. Procedures for Early Termination 
If a patient withdraws or is removed from the study for any reason, all early termination procedures 
should be completed.  Reason for withdrawal, date of the discontinuation, and date of the last dose 
of investigational product should be recorded in source documents and in the appropriate section 
of the eCRF.  Investigational product assigned to the withdrawn patient may not be assigned to 
another patient. 
The Medical Monitor and Study Chair should be consulted prior to the withdrawal of the study 
patient, except in the case of a medical emergency.  Written notice (regardless of cause) is to be 
provided to the Medical Monitor within 48 hours of the withdrawal. At the time of discontinuation, 
every effort should be made to ensure all relevant procedures and evaluations scheduled for the 

NCT03167255 
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Clinical Study Protocol  NS-065/NCNP-01-202   
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final study visit are performed.  Except in the case of a medical emergency, assessments described 
in Section 8.2.1, Week 217 or Early Termination Visit, will be performed. 
8.3. Unscheduled Visit 
If a patient returns to the clinic for a visit outside of the protocol evaluation time points, the visit 
and any assessments and/or tests performed will be recorded into the source documents and the 
eCRF as an Unscheduled Visit.   
8.4. Patient Replacement 
If a patient withdraws during the extension study, he will not be replaced.  
8.5. Suspension or Termination of Study 
If, in the opinion of the Study Chair, and the Medical Monitor, clinical observations in the study 
suggest that it may be unwise to continue, the study may be suspended. The Study Chair will 
request a DSMB meeting and consult with the Sponsor. If the Study Chair, Medical Monitor, 
DSMB and Sponsor agree that safety concerns warrant termination of the study, the Sponsor will 
terminate the study.  A written statement fully documenting the reasons for such a termination will 
be provided to investigators, IRBs/REBs and regulatory authorities, if required.  
NS Pharma has the right to terminate an Investigator’s participation in the study and remove all 
study materials from a clinical site.  A written statement will be provided to the Investigator, the 
IRB/REB, and regulatory authorities, if required.   
Possible reasons for termination of the study at a clinical site include, but are not limited to: 
• Unsatisfactory enrollment with respect to quantity or quality, 
• Inaccurate or incomplete data collection on an ongoing basis, 
• Falsification of records, or 
• Failure to adhere to the protocol. 
If any serious or non-serious adverse events have occurred at such a clinical site, all 
documentation relating to the event(s) must be obtained. 
 
 

NCT03167255 
NS-065/NCNP-01 Injection 
 
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9. SAFETY PROCEDURES AND PROCESSES 
9.1. Definition of an Adverse Event 
An AE is any untoward medical occurrence in a patient or clinical investigation patient 
administered a pharmaceutical product, including control, and which does not necessarily have a 
causal relationship with treatment.  This includes any untoward signs or symptoms experienced by 
the patient from the time of consent until completion of the study. 
AEs may include, but are not limited to: 
• Any unfavorable and unintended sign (including an abnormal laboratory finding), 
symptom, or disease temporally associated with the use of a medicinal product, whether 
or not considered related to the medicinal product. 
• Any new disease or exacerbation of an existing disease. 
• Any deterioration in non-protocol-required measurements of laboratory value or other 
clinical test (e.g., ECG) that results in symptoms, a change in treatment, or 
discontinuation from investigational product. 
Disease signs, symptoms, and/or laboratory abnormalities already existing prior to the use of the 
product are not considered AEs after treatment, unless they reoccur after the patient has recovered 
from the preexisting condition or in the opinion of the Investigator they represent a clinically 
significant exacerbation in intensity or frequency. If clinically significant worsening from baseline 
is noted, the changes will be documented in the AE source document and the eCRF.   
Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an 
existing condition after initiation of the investigational product and through 30 days following the 
last dose of investigational product.   
Suspected adverse reaction means any AE for which there is a reasonable possibility that the drug 
caused the AE. For the purposes of IND safety reporting, ‘reasonable possibility’ means there is 
evidence to suggest a causal relationship between the drug and the AE. A suspected adverse 
reaction implies a lesser degree of certainty about causality than adverse reaction, which means 
any adverse event caused by a drug.  

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An AE or suspected adverse reaction is considered “unexpected” if it is not listed in the 
Investigator’s Brochure or is not listed at the specificity or severity that has been previously 
observed.  During the course of the study, the Investigator’s Brochure should be updated on an 
ongoing basis with new important safety information.  
9.2. Definition of a Serious Adverse Event 
An adverse event is serious when the patient outcome is: 
• Death 
• Life-threatening (see below for expanded definition) 
• Hospitalization (initial or prolonged) 
• Disability or permanent damage (see below for expanded definition) 
• Congenital anomaly/Birth defect 
• Important medical events that, based upon appropriate medical judgment, may jeopardize 
the patient and may require medical or surgical intervention to prevent one of the 
outcomes listed above. 
Life-threatening Experience: Any AE that places the patient, in the view of the site PI, at immediate 
risk of death from the AE as it occurred, i.e. does not include an AE that, had it occurred in a more 
severe form, might have caused death. 
Any hospital admission with at least one overnight stay will be considered an inpatient 
hospitalization.  However, emergency room visits that do not result in admission to the hospital 
should be evaluated for one of the other serious outcomes (e.g., life-threatening; required 
intervention to prevent permanent impairment or damage; other serious medically important 
event). 
Hospitalization for an elective or outpatient procedure scheduled or planned before signing of 
informed consent will not be considered to be an SAE.  However, unexpected complications and/or 
prolongation of hospitalization that occur during elective or outpatient surgery should be recorded 
as adverse events and assessed for seriousness.  Admission to the hospital for social or situational 
reasons (e.g., no place to stay, live too far away to come for hospital visits) will not be considered 
inpatient hospitalizations.  

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Disability or permanent damage: Any AE that results in a substantial disruption of a patient’s 
ability to conduct normal life functions, i.e., the AE resulted in a significant, persistent or 
permanent change, impairment, damage or disruption in the patient's body function/structure, 
physical activities and/or quality of life. 
Important medical events that may jeopardize the patient and may require medical or surgical 
intervention to prevent one of the outcomes listed above: an AE that may not result in death, be 
life-threatening, or require hospitalization may be considered an SAE when, based upon 
appropriate medical judgment, it may jeopardize the patient and may require medical or surgical 
intervention to prevent one of the outcomes listed above. 
9.3. Severity 
It is the Investigator’s responsibility to assess the intensity (severity) of an adverse event.   
The severity of the adverse event will be characterized and recorded as “mild, moderate, severe, 
life-threatening or death” according to the following definitions:  The CTCAE v4.03 guidelines 
for severity assessments will be used to grade adverse events for this trial (available at 
evs.nci.nih.gov/ftp1/CTCAE/About.html). The CTCAE v4.03 listed guidelines for severity 
assessment are: 
• Mild: Asymptomatic or mild symptoms; clinical or diagnostic observations only or 
intervention not indicated 
• Moderate: minimal, local or noninvasive intervention indicated or limited age-appropriate 
instrumental activities of daily living (ADL)  
• Severe: Severe or medically significant but not immediately life-threatening; or 
hospitalization or prolongation of hospitalization indicated; or disabling; or limiting self-
care ADL  
• Life-threatening: life-threatening consequences or urgent intervention indicated 
• Death: Death related to AE 
Note:  A severe adverse event need not be serious and a serious adverse event (SAE) need not, 
by definition, be severe. 

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9.4. Relationship 
It is the Investigator’s responsibility to assess the relationship between the investigational product 
and the adverse event.  The degree of “relatedness” of the adverse event to the investigational 
product may be described using the following scale: 
Not Related 
• Not Related: No temporal association and other etiologies are likely the cause.  
• Unlikely: Event or laboratory test abnormality, with a time to drug that makes a 
relationship improbable (but not impossible). Diseases or other drugs provide plausible 
explanations. 
Related 
• Possible: Temporal association, but other etiologies are likely the cause.  However, 
involvement of the investigational product cannot be excluded. 
• Probable: Temporal association, other etiologies are possible but unlikely.  The event may 
respond if the investigational product is discontinued. 
• Definite: Established temporal association with administration of the investigational 
product with no other more probable cause.  The event should resolve when the 
investigational product is discontinued and recur on re-challenge. 
9.5. Reporting 
9.5.1. Adverse Event Reporting 
All adverse events occurring during the course of the study (starting from signing informed consent 
to study completion) will be collected on the adverse event eCRF.  Each adverse event is to be 
evaluated for duration, severity, seriousness, and causal relationship to the investigational product.  
For each adverse event, the following information will be recorded: 
• Description of the event (e.g., headache), 
• Date of onset, 
• Date of resolution (or that the event is continuing), 
• Action taken as a result of the event, 
• Seriousness of the event, 

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• Severity of the event, 
• Outcome of the event, and 
• Investigator’s assessment of relationship to investigational product. 
A cluster of signs and symptoms that results from a single cause should be reported as a single 
adverse event (e.g., fever, elevated WBC, cough, abnormal chest x-ray, etc., can all be reported as 
“pneumonia”). 
The Investigator will carefully evaluate the comments of the patient and the response to treatment 
in order that he/she may judge the true nature and severity of the adverse event.  The question of 
the relationship of adverse events to investigational product administration should be determined 
by the Investigator or study physician after thorough consideration of all facts that are available. 
Clinically significant (CS) changes from time of informed consent will be documented as AEs on 
the AE eCRF. CS changes are physical findings that have medical relevance and may result in an 
alteration in medical care. 
9.5.2. Serious Adverse Event Reporting 
All SAEs, including death due to any cause and whether or not deemed drug-related or expected, 
must be reported within the study EDC system (eCRF) within 24 hours (1 working day) of the 
Principal Investigator or the clinical site becoming aware of the occurrence.  
The Investigators responsible for ongoing clinical studies with the investigational product will be 
notified by the CRO of all SAEs. 
▪ 9.5.3 Adverse Events of Special Interest 
The following events are considered AEs of special interest based on the route of administration 
and toxicology profile for the study drug: 
• 
 
Access device complication (for participants with indwelling access devices); 
• 
 
Cystatin C >1.5 × ULN or >1.5 × baseline if baseline >ULN; 
• 
 
Urine protein-osmolality > 0.3 mg/L/mOsm/kg; 
• 
Any confirmed instances of hematuria or other potentially clinically significant 
abnormalities on urinalysis.  

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9.6.Serious Adverse Event Follow-up 
SAEs will be followed by the site investigator until resolution or until the site PI determines that 
follow-up is no longer medically necessary. 
Follow-up information, or new information regarding an ongoing SAE, must be provided promptly 
to the CRO within 24 hours of knowledge of the new or follow-up information.  The Coordinating 
Center will forward the information to the Study Chair, Sponsor, the Medical Monitor and DSMB. 
9.7. Reporting of Serious Adverse Events to Regulatory Authorities 
PharmaLex is responsible for submitting Serious and Unexpected Suspected Adverse Reaction 
(SUSAR) reports of SAEs to the appropriate regulatory authorities.  All Investigators responsible 
for ongoing clinical studies with the investigational product will be notified by the CRO of all 
SAEs. The investigator shall report all SAEs occurring within 24 hours after becoming aware via 
the study EDC system. In the event the EDC system is down, the SAE report form should be 
completed and emailed or faxed to IQVIA. Reports of all SAEs must be communicated as soon 
as possible to the appropriate IRB/REB and/or reported in accordance with local laws and 
regulations.  Investigators should provide written documentation of IRB/REB notification for 
each report to CRO. 
The sponsor must report any suspected adverse reaction to the study medication, that is both 
serious and unexpected, or any serious adverse events suspected to be related to the CVA port, to 
the FDA and Health Canada (21 CFR 312.32(c)(1)(i) and C.05.014, respectively).  
9.8. Reporting of Patient Death 
The death of any patient during the study or within 30 days of study completion, regardless of the 
cause, must be reported as detailed in Section 9.5. 
9.9. Monitoring and Follow-up of Adverse Events 
Patients who experience adverse events will be monitored with relevant clinical assessments and 
laboratory tests, as determined by the Investigator.  All follow-up results are to be reported to the 
Medical Monitor.  Any actions taken and follow-up results must be recorded either on the 
appropriate page of the eCRF- or in appropriate follow-up written correspondence, as well as in 

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the patient’s source documentation.  Follow-up laboratory results should be filed with the patient’s 
source documentation. 
For all adverse events that require the patient to be discontinued from the study, relevant clinical 
assessments and laboratory tests must be repeated at appropriate intervals until final resolution or 
stabilization of the event(s). 
Any AEs that are unresolved at the patient’s last AE assessment in the study are followed up by 
the site PI or designee for as long as medically indicated, but without further recording in the 
eCRF. The sponsor and designated CRO retain the right to request additional information for any 
patient with ongoing AEs/SAEs at the end of the study, if judged necessary.  
9.10. General Monitoring and Management of Abnormal Clinical Labs 
It is the Investigator’s responsibility to review the results of all laboratory tests as they become 
available and to sign and date the results indicating review.  For each laboratory test outside of the 
laboratory normal range, the Investigator must ascertain if this represents a clinically significant 
change from baseline for the individual patient.  The Investigator may repeat a laboratory test or 
request additional tests to verify results of the original laboratory test. 
If a laboratory value is determined to be an abnormal and clinically significant change from 
baseline for the patient, the Investigator should determine if it qualifies as an adverse event, and if 
so, an appropriate eCRF will be completed.  All clinically significant laboratory abnormalities 
occurring during the study and that were not present at baseline should be followed and evaluated 
with additional tests if necessary until diagnosis of the underlying cause, or resolution.  
9.11 
Monitoring of Urine Analyses 
Any instances of hematuria or other potentially clinically significant abnormalities on urinalysis 
will be confirmed at the following week’s visit, or sooner at discretion of the investigator. 
Confirmed clinically significant treatment-emergent abnormalities will be recorded as AEs and 
discussed with the study chair and medical monitor. Abnormalities will be monitored and 
evaluated with additional tests or consultations, if necessary, until the underlying cause is 
determined, or the event is brought to an acceptable resolution. Additional clinical and laboratory 

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information will be collected and documented in order to better characterize abnormalities and 
identify etiology and appropriate management. 
9.12. Monitoring and Management of Abnormal Electrocardiograms 
If a clinically significant ECG abnormality occurs that was not present at baseline (Screening Visit 
of Study NS-065/NCNP-01-201) and the Investigator determines that the abnormality is related to 
investigational product, the abnormality will be discussed with the Medical Monitor. The ECG 
abnormality will be monitored and evaluated with additional tests (if necessary) until the 
underlying cause is determined or the event is brought to an acceptable resolution.  Additional 
clinical and laboratory information will be collected and carefully documented in order to better 
characterize the ECG abnormality and rule out alternative causes.  ECG findings determined to be 
a clinically significant change from baseline should be reported as an adverse event regardless of 
causality. 
Unscheduled ECG assessments will be completed at the discretion of the Investigator. 
9.13 
Intravenous (IV) Access Considerations 
Investigational product dosing will be administered through IV infusion.  Peripheral venous access 
(IV catheter that empties into a peripheral vein in the arms, hands, legs or feet) is the preferred 
route of IP administration for this study.   
Central venous access (IV catheter that empties into a large central vein) will be considered on a 
case-by-case basis for participants who have difficulty with peripheral venous access.  An 
implantable central venous access (CVA) port is the preferred option of central venous access, if 
necessary, for this study.  The Sponsor will decide whether or not to approve this option after 
discussions with the site PI, Study Chair and Medical Monitor have ensured mutual agreement that 
central venous access will still maintain a positive benefit/risk ratio for the participant in this study.  
Before final decision, NS Pharma will obtain documentation from the site investigator that the 
consulting surgeon who will place the port holds hospital privileges as a board eligible/board 
certified surgeon.  The decision, rationale and conclusion regarding the maintained positive 
benefit/risk ratio will be detailed in writing and sent to the requesting site.  Central venous access 
should not be implemented without Sponsor approval. 

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An alternative method of central venous access may only be considered in the case of a 
contraindication, in the opinion of the consulting surgeon for the placement of a totally implantable 
central venous access device (port). 
9.14. Medical Monitor 
The Medical Monitor (MM, in the SMP also referred to as MSO) in this study will assist the 
Sponsor and clinical sites with the review, assessment and reporting of adverse event cases, and 
the discussion of safety reporting issues and application of stopping rules as needed.  The MM 
will interact with the Sponsor’s pharmacovigilance team and the study sites to gain a full 
understanding of the reported cases to ensure the assessment and narrative of AE cases is accurate 
and captures the appropriate medical detail of the event for recording and reporting purposes.  The 
MM will work closely with the Study Chair and the DSMB as appropriate. 
9.15. Data and Safety Monitoring Board 
CINRG has a standing DSMB that is made up of at least: 
• 
Two medical doctors with experience in DMD;  
• 
One medical doctor who is familiar with the interventions and the anticipated adverse 
events;  
• 
A statistician; and  
• 
A patient advocate. 
It is the responsibility of the CINRG DSMB to review data quality, relevant safety data and AEs 
for all patients enrolled in the study and to make recommendations to the Study Chair, Medical 
Monitor and Sponsor regarding the ongoing conduct and monitoring of the study.  
The DSMB will first meet to review and approve the study protocol.  The DSMB will then meet 
every 6 months or as needed. Additional DSMB reviews may be requested by the Study Chair, 
Medical Monitor or Sponsor, as needed.  DSMB meetings may be held via teleconference as 
necessary. 
A summary safety report will be produced every 6 months in collaboration between the clinical 
CRO, data management group and statistician and provided to the DSMB for review.  The report 
will include safety data and study progress data (enrollment, data completion and data quality).  
Upon completion of safety data reviews, the DSMB may recommend revision or modification to 

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the study (e.g., change in eligibility criteria, revision of informed consent). The DSMB may also 
assist the Sponsor to evaluate regulatory reporting requirements of an event or group of events. 
 
 

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10. PLANNED STATISTICAL METHODS 
10.1  General Considerations 
The statistical analyses described in this section will be performed as further outlined in the 
Statistical Analysis Plan (SAP), which will be finalized prior to database lock.  The SAP will 
supersede the protocol if there are any differences between the two documents in the plans for data 
analysis and the differences will be noted in the SAP.  The SAP will be included as an appendix 
in the clinical study report for this protocol. Statistical analyses will be performed using SAS 9.4 
or higher. 
Patients will be enrolled in this study after completing Study NS-065/NCNP-01-201 and will 
continue with the study dose (40 mg/kg/week or 80 mg/kg/week) they received in Study NS-
065/NCNP-01-201.  Results for this study will be summarized by study dose.   
 
Statistical analyses will occur at the following times: 
• Every 6 months. A report for the DSMB will be produced including all safety data available 
at the temporary data lock. A template report will be provided to the DSMB prior to the 
first report, and the report formats will be updated at the DSMB's requests as the data 
develop. The DSMB will review completeness of efficacy data, but will not review the 
efficacy data itself. 
• At the conclusion of the study. A summary report will be generated on all baseline 
characteristics, safety assessments and efficacy assessments, addressing each of the 
primary, secondary and exploratory objectives of the study.  
All statistical tests will be performed at a two-sided significance level of 0.05 with no corrections 
for multiple comparisons or multiple outcomes. Note however, that since this is an early phase 
extension study, with a focus on safety, the emphasis will not be on statistical tests (i.e.,  
p-values).  
10.2  Determination of Sample Size 
Approximately 16 patients from Study NS-065/NCNP-01-201 will enter this study.  Because this 
is an open-label extension study conducted in patients who complete Study NS-065/NCNP-01-
201, the sample size is not based on any statistical considerations.    

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10.3  Analysis Populations 
All analyses will be based on the actual treatment each patient received. Two populations will be 
defined for data analysis: the Safety Population and the Full Analysis Set.  
Safety Population  
All patients who receive at least one dose of NS-065/NCNP-01 Injection 250 mg in both the dose-
finding (NS-065/NCNP-01-201) and extension study (NS-065/NCNP-01-202) will be included in 
the Safety Population. The Safety Population is the primary analysis population for safety 
assessments.  
Full Analysis Set (FAS)  
All patients who receive at least one dose of NS-065/NCNP-01 Injection 250 mg in both the dose-
finding (NS-065/NCNP-01-201) and extension study (NS-065/NCNP-01-202) will be included in 
the FAS. The FAS is the primary analysis population for clinical efficacy and exploratory PD 
assessments.  
10.4  Demographics and Baseline Characteristics 
Summaries of patient demographics (age, race, ethnicity and dominant hand), baseline safety 
characteristics (anthropometrics, vital signs, physical examination, hematology, chemistry, BNP, 
urinalysis, ECG and antibodies), and baseline efficacy parameters will be done by dose level.  Note 
that two sets of baseline values will be summarized, Screening Visit (from Dose-finding Study 
NS-065/NCNP-01-201) and Week 25 (from Extension Study NS-065/NCNP-01-202).  
Any differences in distribution of baseline characteristics will be noted, although with such a small 
study, it is to be expected that some differences will emerge. 
10.5  Safety Assessments 
Safety analyses will be performed using the safety population and will address one of two co-
primary objectives of the study.  All safety assessments will be based on actual treatments received 
by participants. 

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10.5.1 Anthropometrics, Vital Signs, Laboratory Assessments, and ECG 
Anthropometrics, vital signs, hematology, chemistry, BNP, urinalysis, and ECG results will be 
summarized by dose level over time using descriptive statistics for continuous outcomes.   Actual 
values and change from Screening Visit (hematology, chemistry and urinalysis) or Day 1 
(anthropometrics, vital signs and ECG) from Study NS-065/NCNP-01-201 and change from 
Week 25 (baseline of the NS-065/NCNP-01-202 study) will be presented.  Further, all lab 
abnormalities will be listed. 
10.5.2 Physical Exam and Adverse Events 
Physical exam results will be summarized by frequency of presence of abnormalities in body 
system (beyond the DMD diagnosis) and in particular any changes in the physical exam over time. 
Any new adverse events (AEs) occurring after the time of enrollment into NS-065/NCNP-01-202 
(defined as the time of IP infusion at the Week 25 visit) will be considered for analysis for this 
study.  For AEs starting in study NS-065/NCNP-01-201 which are not resolved at the time of 
enrollment into NS-065/NCNP-01-202, any change in outcome or relatedness will be reported in 
study NS-065/NCNP-01-201.  For AEs starting in study NS-065/NCNP-01-201 which increase in 
severity or becomes serious after enrollment in study NS-065/NCNP-01-202, a new AE will be 
reported in NS-065/NCNP-01-202. 
Treatment-emergent AEs (TEAEs) will be summarized by dose level.  Coding will be done by 
system organ class and preferred term (using the Medical Dictionary for Regulatory Activities 
(MedDRA)). Level of severity will be assessed using the CTCAE grading system. Summaries will 
include: 
A. Summaries at the patient level  
How many patients had any TEAE, any SAE, highest severity of TEAE within a patient across all 
infusions, highest relationship level of TEAE within a patient across all infusions, highest 
intervention level regarding investigational product (e.g., discontinued, vs. reduced dose vs. 
temporarily stopped vs. no interruption in infusions), and worst outcome within a patient (e.g., AE 
did not resolve and has a permanent effect). 
B. Summary at the event level  

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Summaries will be done using the MedDRA coding by events and overall, summarizing by system 
organ class and preferred term, by relationship to investigational product, severity, intervention, 
and outcome. 
Listings tables will be provided for all AEs. 
10.5.3 Concomitant Medications and/or Other Treatments 
GCs, which are required as part of the inclusion criteria, will be summarized by type of GC 
(prednisone vs. deflazacort), by schedule (daily vs. any other), and by dose level. Patients are 
required not to change the GC dose while on study. Any changes in doses or schedule will be 
listed. 
Other concomitant medications will be summarized by ATC class and preferred term. Each 
medication will be counted once within a patient using it, regardless of the number of times it was 
reported on the eCRFs. The summaries will note new medications or supplements vs. those already 
given at baseline and study entry. Any other treatment, surgeries, will be listed and described; 
however, those are expected to be few without a need to be summarized using tables. 
10.5.4 Cytokines, Antibodies and Pharmacokinetics 
Cytokines and antibodies and pharmacokinetic concentrations will be summarized by dose level 
over time.  Pharmacokinetic concentrations may be summarized at a later date.  PK samples will 
only be analyzed in the event that anti-NS-065/NCNP-01 antibodies are detected in order to 
understand the impact of immunogenicity on plasma levels of the drug. 
10.6  Efficacy Endpoints 
All efficacy analyses will be performed using the FAS population. In addition, additional analyses 
will utilize historical control group data from the CINRG DNHS.  
10.6.1 Primary Efficacy Objective 
The primary efficacy outcome measure (TTSTAND) will be summarized by dose level over time 
using descriptive statistics.  Actual values and change from the Pre-Infusion Visit (baseline from 
Study NS-065/NCNP-01-201) and change from Week 25 (baseline of the NS-065/NCNP-01-202 
study) will be presented. 

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The TTSTAND times to perform the test will also be converted to velocities. (Note that a test 
result that is to be converted to velocity that the patient could not perform due to disease 
progression will have velocity set to zero only at the first visit where this occurs.  After that visit, 
missing observations due to disease progression will be left as missing.).  If needed, a data 
transformation or nonparametric approach will be performed for these assessments. Within patient 
changes and velocities (possibly transformed) will be tested using a one-sided paired t-test within 
dose level and within both dose groups combined.  
10.6.2 Secondary Efficacy Objectives 
10.6.2.1 
Analyses of NS-065/NCNP-01 patients only 
TTRW, TTCLIMB, NSAA, 6MWT and QMT will be summarized by dose level over time using 
descriptive statistics.  Actual values and change from the Pre-Infusion Visit (baseline from Study 
NS-065/NCNP-01-201) and change from Week 25 (baseline of the NS-065/NCNP-01-202 study) 
will be presented. 
The TTRW and TTCLIMB times to perform the test will also be converted to velocities. For a test 
that the patient could not perform, the velocity will be set to zero.  If needed, a data transformation 
or nonparametric approach will be performed for these assessments. Within patient changes and 
velocities (possibly transformed) will be tested using a one-sided paired t-test within dose group 
and within both dose groups combined. 
10.6.2.2 
Analyses comparing NS-065/NCNP-01 patients to historical controls 
A matched data set from the CINRG DNHS data will be created. The purpose of the matching is 
to create a group data set that corresponds in characteristics to the patients in this study. The 
CINRG DNHS data set includes patients from age 2 years to over 30 years old, and some have 
been followed for close to a decade. Therefore, it is important to create a comparator group which 
will allow valid group comparisons. No patient to patient matched analysis is proposed. The sole 
purpose of the matching is to create a historical control group which is comparable in its basic 
characteristics to the study patient group.  The final CINRG DNHS data set is expected to have 
between 16 and 32 patients included with time intervals of evaluations between 6 and 15 months.   
TTSTAND, TTRW, TTCLIMB, NSAA, 6MWT, and QMT results will be compared between the 
NS-065/NCNP-01 patients and the CINRG DNHS patients possibly using mixed-effects linear 

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models.  The TTSTAND, TTRW, and TTCLIMB times to perform the test will be converted to 
velocities. For a test that the patient could not perform, the velocity will be set to zero.  
10.7  Interim Analyses 
Safety analyses are planned every 6 months as described above. Moreover, semi-annual reports to 
the DSMB will be descriptive and focus on safety.  
In addition, interim analyses for safety are planned and interim analyses for efficacy may be 
conducted and further described in the Statistical Analysis Plan. 
10.8  Handling of Missing Data 
Every effort will be made to collect all data. However, despite best efforts, missing or incomplete 
data may be reported. All missing or partial data will be presented in the patient data listing, as 
they are recorded on the eCRF. 
Patients lost to follow-up or withdrawn will be included in statistical analyses up to the point of 
their last evaluation. Unless otherwise specified, no imputation of values for missing data will be 
performed. Of note, since patients with DMD are expected to decline over time, imputing efficacy 
parameters by last value carried forward mostly biases towards patients appearing stronger or 
faster than they are, since it carries forward potentially a better value than the value at the time of 
the missed observation. Therefore, for this study, we will summarize how much data are missing, 
but do not expect to need to impute any data to accomplish the analyses as described.  Details of 
handling missing data will be described in the Statistical Analysis Plan. 
Missed assessments which occurred as a result of the COVID-19 pandemic will be entered on the 
COVID-19 Protocol Deviation Log by site personnel. It is anticipated that some or all assessments 
and laboratory tests required by the protocol schedule of events may be missed during the 
pandemic. Some of the missed assessments may be able to be collected at the next home or onsite 
visit. The missing data will be summarized in the Statistical Analysis Plan.  

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11 ADMINISTRATIVE CONSIDERATIONS 
11.1  Investigators 
The Investigator must agree to the responsibilities and obligations listed below, as specified by the 
appropriate FDA/Health Canada regulatory requirements or ICH/GCP guidelines: 
• Agree to conduct the study in accordance with the relevant current protocol; 
• Agree to personally conduct or supervise the described investigation(s); 
• Agree to inform any patients, or persons used as controls, that the investigational products 
are being used for investigational purposes and ensure that the requirements relating to 
obtaining informed consent and IRB/REB review and approval are met; 
• Agree to report adverse experiences that occur during the course of the investigation(s); 
• Read and understand the information in the Investigator’s Brochure, including the potential 
risks and side effects of the investigational product; 
• Ensure that all associates, colleagues, and employees assisting in the conduct of the study are 
informed about their obligations in meeting the above commitments; 
• Maintain adequate and accurate records and make those records available for inspection; 
• Ensure that an IRB/REB will be responsible for the initial and continuing review and 
approval of the clinical investigation; 
• Agree to promptly report to the IRB/REB all changes in the research activity and all 
unanticipated problems involving risks to patients or others; 
• Agree to not make changes in the research without IRB/REB approval, except where 
necessary to eliminate apparent hazards to patients; and 
• Comply with all other requirements regarding the obligations of clinical investigators and all 
other pertinent requirements. 
Refer also to: 
• FDA Regulations Related to Good Clinical Practice and Clinical Trials: 
http://www.fda.gov/oc/gcp/regulations.html 
• Guidance and Information Sheets on GCP in FDA-Regulated Clinical Trials: 
http://www.fda.gov/oc/gcp/guidance.html 
• Guidance for IRBs and Clinical Investigators: http://www.fda.gov/oc/ohrt/irbs/default.htm 

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• Guidance for Industry - E6 Good Clinical Practice:  Consolidated Guidance 
http://www.fda.gov/cder/guidance/959fnl.pdf 
11.2  Informed Consent, Protected Health Information (PHI) and Confidentiality 
11.2.1 Informed Consent 
The ICF, assent form and consent process must comply with US 21CFR Part 50 and local laws. 
The ICF/Assent Form will document the study-specific information provided to the patient by the 
investigator or designee and the patient’s/legal guardian’s agreement to participate in the study.  
The investigator, or designee (as described on Delegation of Authority log), must explain in terms 
understandable to the patient, the purpose and nature of the study, the study procedures, anticipated 
benefits, potential risks, the possible adverse effects and any discomfort participation in the study 
may involve. Each patient must provide a signed and dated ICF before any study related procedures 
are performed. In the case of a subject who is incapable of providing informed consent, the 
investigator or designee must obtain a signed and dated ICF from the patient’s legal guardian.  
Minors, who are not legally capable of giving informed consent, may possess the ability to assent 
or dissent to participation in the study. The investigator, or designee, should explain the study and 
study procedures to the minor in as much detail as the minor is able to comprehend.  IRB/REB-
approved, age appropriate Assent Forms must be obtained from minor patients as required by local 
laws and governing IRB/REBs. 
11.2.2 Confidentiality 
Authority regulations (FDA/Health Canada) require the Sponsor or the Sponsor’s authorized 
representative to inspect all study documents and records maintained by the Investigator, including 
but not limited to medical records (office, clinic, or hospital) for the patients in this study.  These 
regulations also allow the Sponsor’s records to be inspected by authorized representatives of the 
regulatory authorities.  The names and identities of all research patients will be kept in strict 
confidence and will not appear on eCRFs or other records provided to or retained by the Sponsor 
or the Sponsor’s authorized representative.  Patient confidentiality will be respected during review 
of source documents by monitors, auditors and other Sponsor representatives. Review procedures 
will adhere to regulatory requirements and professional standards for confidentiality. Names and 
identities of patients can be protected by de-identifying (i.e., “blacking-out”) patient’s name and 

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replacing the name with the patient’s study identification number.  The ICF must include 
appropriate statements explaining these requirements. 
11.2.3 Protected Health Information (PHI) 
Information on maintaining patient confidentiality in accordance with US and local patient privacy 
regulations must be provided to each patient/legal guardian as part of the informed consent process, 
either as part of the ICF or as a separate signed HIPAA consent.  The investigator or designee must 
explain to each patient that for the evaluation of study results, the patient’s PHI obtained during 
the study may be shared with NS Pharma and its designees, regulatory agencies and IRBs/REBs. 
As the Study Sponsor, NS Pharma will not use the patient’s PHI or disclose it to a third party 
without applicable patient authorization. It is the investigator’s responsibility to obtain written 
permission to use PHI from each patient/legal guardian. If a patient or patient’s legal guardian 
withdraws permission to use PHI, it is the investigator’s responsibility to obtain the request in 
writing and ensure that no further data is collected on the patient. Any data collected up to the 
point of HIPAA consent withdrawal may be used in analysis of the study results. 
11.3  Study Administrative Structure 
Study Chair 
Paula R. Clemens, MD 
University of Pittsburgh 
A506 Scaife Hall 
3550 Terrace Street 
Pittsburgh, Pennsylvania 15261 
Phone: (412) 648-9762 
Email: pclemens@pitt.edu  
 
Medical Monitor 
Helmut H. Albrecht, MD 
PharmaLex Development Services, LLC 
3350 SW 27th Ave, Apt. 2203 
Miami, FL 33133 
Phone: (973) 454-9859 
Email:  helmut@H2A-Associates.com  
 

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11.4 
 Institutional Review Board/Research Ethics Board Approval 
Before initiation of the study, the Investigator must obtain approval or favorable opinion of the 
research protocol, informed consent form, and any material related to patient recruitment from an 
IRB or REB complying with the provisions specified in 21 CFR Part 56 and applicable pertinent 
state and federal requirements of each participating location, including International Conference 
on Harmonization (ICH) and Good Clinical Practice (GCP) guidelines. 
Institutional Review Boards and Research Ethics Boards must be constituted according to the 
applicable laws.  It is the responsibility of each clinical site to submit the protocol, Investigator’s 
Brochure, patient informed consent, patient recruitment materials (if applicable), and other 
documentation as required by the IRB/REB for review and approval.  A copy of the written 
approval must be provided to NS Pharma.  
The documentation should clearly mention the approval/favorable opinion of the protocol, the 
patient informed consent form, and patient recruitment materials (if applicable), including 
respective version dates.  The written approval and a list of the voting members, their titles or 
occupations, and their institutional affiliations must be obtained from the IRBs/REBs and provided 
to NS Pharma (or its authorized CRO) prior to the release of clinical study supplies to the clinical 
site and commencement of the study.  If any member of the IRB/REB has direct participation in 
this study, written notification regarding his or her abstinence from voting must also be obtained. 
Clinical sites must adhere to all requirements stipulated by their respective IRB/REB.  This 
includes notification to the IRB/REB regarding: protocol amendments, updates to the patient 
informed consent, recruitment materials intended for viewing by patients, IND Safety Reports, 
serious and unexpected adverse events, reports and updates regarding the ongoing review of the 
study at intervals specified by the respective IRB/REB, and submission of final study reports and 
summaries to the IRB/REB. 
It is the responsibility of each clinical site to submit information to the appropriate IRB/REB for 
annual review and annual re-approval. 
The Investigator must promptly inform their IRB/REB of all SAEs or other safety information 
reported from the patient or NS Pharma (or the Sponsor’s authorized CRO). 

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11.5  Ethical Conduct of the Study 
The Investigator agrees, when signing the protocol, to adhere to the instructions and procedures 
described in the protocol and conduct the study in accordance with the CFRs (21 CFR Parts 11, 
50, 54, 56, 312, 314, and 320) and local regulations, which originate from the ethical principles 
laid down in the current revision of the Declaration of Helsinki, GCPs, and policies and procedures 
as outlined by the ethical requirements for IRB/REB review and informed consent forms.  
The Investigator agrees to allow monitoring and auditing of all essential clinical study documents 
by NS Pharma or its authorized representatives and inspection by the FDA or other appropriate 
regulatory authorities.  Monitoring and auditing visits by NS Pharma or authorized designee will 
be scheduled with the appropriate staff at mutually agreeable times periodically throughout the 
study. 
The Investigator will assure proper implementation and conduct of the study, including those 
study-related duties delegated to other appropriately qualified individuals.  The Investigator will 
assure that study staff cooperates with monitoring and audits and will demonstrate due diligence 
in recruiting and screening study patients. The Investigator must sign and return to NS Pharma (or 
its authorized CRO) the “Study Acknowledgment” page and provide a copy of current curriculum 
vitae.  For this study and all studies conducted under an IND in the United States, the Investigator 
must sign and return a complete Form FDA 1572 “Statement of Investigator” to NS Pharma (or 
the Sponsor’s authorized CRO). Similarly, Investigators in Canada must sign and return the 
“Qualified Investigator Undertaking” form to be retained by the Sponsor. 
11.6  Study Monitoring 
NS Pharma (or its authorized CRO) has the obligation to follow this study closely to ensure that 
the study is conducted in accordance with the protocol, ICH and GCP regulatory requirements, the 
CFRs, FDA, and the current Declaration of Helsinki throughout its duration by means of personal 
visits to the Investigator’s facilities and other communications.  
These visits will be conducted to evaluate the progress of the study, verify the rights and well-being 
of the patients are protected, and verify the reported clinical study data are accurate, complete, and 
verifiable from source documents.  This includes review of informed consent forms, results of tests 
performed as a requirement for participation in this study, and any other medical records (e.g., 

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laboratory reports, clinic notes, investigational product disbursement log, pharmacy records, 
patient sign-in sheets, patient-completed questionnaires, telephone logs, ECGs) required to 
confirm information contained in the eCRFs. 
A monitoring visit should include a review of the essential clinical study documents (regulatory 
documents, case report forms, medical records and source documents, investigational product 
disposition records, patient informed consent forms, etc.) as well as discussion on the conduct of 
the study with the Investigator and staff. 
The monitor should conduct these visits as frequently as appropriate for the clinical study.  The 
Investigator and staff should be available during these visits for discussion of the conduct of the 
study as well as to facilitate the review of the clinical study records and resolve/document any 
discrepancies found during the visit.  
All monitoring activities will be reported and archived.  In addition, monitoring visits will be 
documented at the clinical site by signature and date on the study-specific monitoring log. 
Details of monitoring procedures will be described in the study monitoring plan and the COVID-
19 study monitoring plan addendum. 
11.7  On-Site Audits 
Representatives of NS Pharma or its authorized clinical quality assurance group may visit a clinical 
site at any time during the study to conduct an audit of the study in compliance with regulatory 
guidelines and company policy. These audits will require access to all study records, including 
source documents, for inspection and comparison with the eCRFs.  Patient privacy must be 
respected.  The Investigator and clinical site personnel are responsible for being present and 
available for consultation during routinely scheduled site audit visits conducted by NS Pharma or 
its authorized representative.   
The clinical study may also be inspected by the FDA (or other regulatory authorities) to verify that 
the study was conducted in accordance with protocol requirements, as well as the applicable 
regulations and guidelines. 

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In the event the Investigator is contacted by regulatory authorities who wish to conduct an 
inspection of the clinical site, the Investigator will promptly notify NS Pharma (or its authorized 
CRO) of all such requests and will promptly forward a copy of all such inspection reports. 
Due to the global COVID-19 pandemic of 2020, on-site monitoring visits were temporarily 
converted to remote monitoring visits as a result of travel, site and safety restrictions. During this 
time, the site provided redacted source documents for monitoring verification. The monitor 
followed the procedures outlined in the COVID-19 study monitoring plan addendum. Once the 
COVID-19 travel and monitoring bans are lifted, the on-site monitoring will return to its normal 
schedule and procedures.  
11.8  Case Report Forms and Study Records 
Access to eCRFs will be provided to the clinical site.  As part of the responsibilities assumed by 
participating in the study, the Investigator agrees to maintain adequate case histories for the 
patients treated as part of the research under this protocol.  The Investigator agrees to maintain 
accurate source documentation and eCRFs as part of the case histories.  
Study records are comprised of source documents, eCRFs, and all other administrative documents 
(e.g., IRB/REB correspondence, clinical study materials and supplies shipment manifests, 
monitoring logs and correspondence).  A study-specific binder will be provided with instructions 
for the maintenance of study records. 
Source documentation is defined as any hand written or computer-generated document that 
contains medical information or test results that have been collected for or in support of the 
protocol specifications (e.g., laboratory reports, clinic notes, investigational product disbursement 
log, pharmacy records, patient sign-in sheets, patient completed questionnaires, telephone logs, x-
rays, and ECGs).  All draft, preliminary, and pre/final iterations of a final report are also considered 
to be source documents (e.g., faxed and hard copy of laboratory reports, faxed and hard copy of 
initial results, and final report).   
The Investigator agrees to allow direct access to all essential clinical study documents for the 
purpose of monitoring and/or auditing by NS Pharma or its authorized representatives and 
inspection by the appropriate regulatory authorities. 

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Data reflecting the patient’s participation with the investigational product under investigation are 
to be reported to NS Pharma.  The data are to be recorded on the eCRFs and/or other media 
provided or approved by NS Pharma.  
A complete eCRF must be submitted for each patient who receives investigational product, 
regardless of duration.  All supportive documentation submitted with the eCRF, such as laboratory 
or hospital records, should be clearly identified with the study and patient number.  Any personal 
information, including patient name, should be removed or rendered illegible to preserve 
individual confidentiality.  The eCRF should not be used as a source document unless otherwise 
specified by NS Pharma.  
Neither NS Pharma nor a service provider contracted to analyze data and complete the study report 
is permitted to interpret a blank answer; therefore, all fields should be complete.  All requested 
information must be entered on the eCRFs.  If an item is not available or is not applicable, this fact 
should be indicated as (N/A) not available or (N/D) not done; do not leave a space blank.  
It is essential that all dates appearing on NS Pharma patient data collection forms for laboratory 
tests, cultures, etc., be the dates on which the specimens were obtained, or the procedures 
performed.  The eCRFs will be electronically signed by the Investigator and dated as verification 
of the accuracy of the recorded data.  All data collection forms should be complete within 48 hours 
following the evaluation. 
11.9  Amendments 
Changes to the research covered by this protocol must be implemented by formal protocol 
amendment.  All amendments to the protocol must be initiated by NS Pharma and signed and dated 
by the Investigator. Protocol amendments must not be implemented without prior IRB/REB 
approval.  Documentation of amendment approval by the Investigator and IRB/REB must be 
provided to NS Pharma or its authorized CRO.  When the change(s) involve only logistic or 
administrative aspects of the study, the IRB/REB only needs to be notified.   
During the course of the study, in situations where a departure from the protocol is unavoidable, 
the Investigator will contact the NS Pharma Medical Monitor and the Study Chair. Except in 
emergency situations, this contact should be made before implementing any departure from the 
protocol.  In all cases, contact with the Medical Monitor and Study Chair must be made as soon as 

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possible to discuss the situation and agree on an appropriate course of action. The data recorded 
on the eCRF and source documents will reflect any departure from the protocol and the source 
documents will describe the departure and the circumstances requiring it. 
11.10 Access to Source Documentation 
Authority regulations require the Sponsor (or the Sponsor’s authorized representative) to inspect 
all documents and records to be maintained by the Investigator, including but not limited to, 
medical records (office, clinic, or hospital) for the patients in this study.  These regulations also 
allow the Sponsor’s records to be inspected by authorized representatives of regulatory authorities.  
The Investigator will permit study-related monitoring, audits, IRB/REB review, and regulatory 
inspections by providing direct access to source data/documents.  Direct access includes 
permission to examine, analyze, verify, and reproduce any records and reports that are important 
to the evaluation of a clinical study. 
11.11 Record Retention 
In compliance with the ICH/GCP guidelines, the Investigator/Institution agrees to retain and 
maintain all study records that support the data collected from each patient, as well as all study 
documents as specified in ICH/GCP, Section 8 Essential Documents for the Conduct of a Clinical 
Trial.  The Investigator agrees to contact NS Pharma before destroying or relocating any study 
documentation and is expected to take measures to prevent accidental or premature destruction of 
these documents.   
If the Investigator retires, relocates, or for other reasons withdraws from the responsibility of 
keeping the study records, custody must be transferred to a person who will accept responsibility.  
NS Pharma must be contacted in writing regarding the name and address of the new person 
responsible as well as the disposition of document storage. Under no circumstances shall the 
Investigator relocate or dispose of any study documents before having obtained written approval 
from NS Pharma. 
Essential records (including eCRFs, source documents, clinical drug disposition records, signed 
patient informed consent forms, adverse event reports, and other regulatory documents) as required 
by the applicable regulations, must be maintained for 2 years after a marketing application is 
approved for the drug for the indication for which it is being investigated; or, if no application is 

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to be filed or if the application is not approved for such indication, until 2 years after the 
investigation is discontinued and the FDA is notified. 
It is the responsibility of NS Pharma or its authorized CRO to inform the Investigator/Institution 
as to when these documents no longer need to be retained. 
11.12 Financial Disclosure 
Prior to the start of the study, Investigators will release sufficient and accurate financial 
information that permits NS Pharma to demonstrate that an Investigator and all study personnel 
listed on the FDA Form 1572 or the Health Canada Qualified Investigator Undertaking form have 
no personal or professional financial incentive regarding the future approval or disapproval of the 
investigational product such that his or her research might be biased by such incentive. 
11.13 Publication and Disclosure Policy 
It is understood by the Investigator that the information and data included in this protocol may be 
disclosed to and used by the Investigator’s staff and associates as may be necessary to conduct this 
clinical study. 
All information derived from this clinical study will be used by NS Pharma (or designee) and 
therefore, may be disclosed by NS Pharma (or designee) as required to other clinical investigators, 
to the FDA, and to other government agencies, or in connection with intellectual property filings 
or publications.  In order to allow for the use of the information derived from this clinical study, it 
is understood by the Investigator that there is an obligation to provide NS Pharma with complete 
test results and all data from this clinical study. The Investigator agrees to maintain this information 
in confidence, to use the information only to conduct the study and to use the information for no 
other purpose without NS Pharma’s prior written consent (or as otherwise may be permitted 
pursuant to a written agreement with NS Pharma or its designee). 
The results of the study will be reported in a clinical study report (CSR) prepared by NS Pharma 
(or designee), which will contain eCRF data from all clinical sites that conducted the study. 
Patient identifiers will not be used in publication.  
NS Pharma shall have the right to publish data from the study without approval from the 
Investigator. Manuscript(s) and abstract(s) may only be prepared through cooperation between NS 

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Pharma (or designee) and the study investigator(s).  If an investigator wishes to publish 
information from the study, a copy of the manuscript must be provided to NS Pharma for review 
in accordance with the provisions of such investigator’s written agreement with NS Pharma (or 
designee) before submission for publication or presentation.  If requested by NS Pharma in writing, 
the investigator will withhold such publication in accordance with the provisions of such 
agreement. 
 
 

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ARCHIVES OF NEUROLOGY. 1991;48(4):383-8. EPUB 1991/04/01. 

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11. FENICHEL GM, FLORENCE JM, PESTRONK A, MENDELL JR, MOXLEY RT, 3RD, 
GRIGGS RC, ET AL. LONG-TERM BENEFIT FROM PREDNISONE THERAPY IN 
DUCHENNE MUSCULAR DYSTROPHY. NEUROLOGY. 1991;41(12):1874-7. EPUB 
1991/12/01. 
12. MOXLEY RT, 3RD, ASHWAL S, PANDYA S, CONNOLLY A, FLORENCE J, 
MATHEWS K, ET AL. PRACTICE PARAMETER: CORTICOSTEROID TREATMENT 
OF DUCHENNE DYSTROPHY: REPORT OF THE QUALITY STANDARDS 
SUBCOMMITTEE OF THE AMERICAN ACADEMY OF NEUROLOGY AND THE 
PRACTICE COMMITTEE OF THE CHILD NEUROLOGY SOCIETY. NEUROLOGY. 
2005;64(1):13-20. EPUB 2005/01/12. 
13. MANZUR AY, KUNTZER T, PIKE M, SWAN A. GLUCOCORTICOID 
CORTICOSTEROIDS FOR DUCHENNE MUSCULAR DYSTROPHY. THE COCHRANE 
DATABASE OF SYSTEMATIC REVIEWS. 2008(1):CD003725. EPUB 2008/02/07. 
14. MAZZONE E, VASCO G, SORMANI MP, TORRENTE Y, BERARDINELLI A, 
MESSINA S, ET AL. FUNCTIONAL CHANGES IN DUCHENNE MUSCULAR 
DYSTROPHY: A 12-MONTH LONGITUDINAL COHORT STUDY. NEUROLOGY. 
2011;77(3):250-6. EPUB 2011/07/08. 
15. HOFFMAN EP. SKIPPING TOWARD PERSONALIZED MOLECULAR MEDICINE. 
THE NEW ENGLAND JOURNAL OF MEDICINE. 2007;357(26):2719-22. EPUB 
2007/12/28. 
16. VAN DEUTEKOM JC, JANSON AA, GINJAAR IB, FRANKHUIZEN WS, AARTSMA-
RUS A, BREMMER-BOUT M, ET AL. LOCAL DYSTROPHIN RESTORATION WITH 
ANTISENSE OLIGONUCLEOTIDE PRO051. THE NEW ENGLAND JOURNAL OF 
MEDICINE. 2007;357(26):2677-86. EPUB 2007/12/28. 
17. PENNISI E. GENETICS. HOPPING TO A BETTER PROTEIN. SCIENCE. 
2008;322(5907):1454-5. EPUB 2008/12/06. 
18. VOIT T, TOPALOGLU H, STRAUB V, MUNTONI F, DECONINCK N, CAMPION G, ET 
AL. SAFETY AND EFFICACY OF DRISAPERSEN FOR THE TREATMENT OF 
DUCHENNE MUSCULAR DYSTROPHY (DEMAND II): AN EXPLORATORY, 
RANDOMISED, PLACEBO-CONTROLLED PHASE 2 STUDY. LANCEL NEROL. 
2014;13(10):987-96. 
19. HAMMOND SM, WOOD MJ. PRO-051, AN ANTISENSE OLIGONUCLEOTIDE FOR 
THE POTENTIAL TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY. CURR 
OPIN MOL THER. 2010;12(4):478-86. 
20. MENDELL JR, RODINO-KLAPAC LR, SAHENK Z, ROUSH K, BIRD L, LOWES LP, ET 
AL. ETEPLIRSEN FOR THE TREATMENT OF DUCHENNE MUSCULAR 
DYSTROPHY. ANN NEUROL. 2013;74(5):637-47. 
21. CIRAK S, FENG L, ANTHONY K, ARECHAVALA-GOMEZA V, TORELLI S, SEWRY 
C, ET AL. RESTORATION OF THE DYSTROPHIN-ASSOCIATED GLYCOPROTEIN 
COMPLEX AFTER EXON SKIPPING THERAPY IN DUCHENNE MUSCULAR 
DYSTROPHY MOL THER. 2012;20(2):462-7. 
22. CIRAK S, ARECHAVALA-GOMEZA V, GUGLIERI M, FENG L, TORELLI S, 
ANTHONY K, ET AL. EXON SKIPPING AND DYSTROPHIN RESTORATION IN 
PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY AFTER SYSTEMIC 
PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER TREATMENT: AN OPEN-

NCT03167255 
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LABEL, PHASE 2, DOSE-ESCALATION STUDY. LANCET. 2011;378(9791):595-605. 
EPUB 2011/07/26. 
23. BROOKE MH, GRIGGS RC, MENDELL JR, FENICHEL GM, SHUMATE JB, 
PELLEGRINO RJ. CLINICAL TRIAL IN DUCHENNE DYSTROPHY. I. THE DESIGN 
OF THE PROTOCOL. MUSCLE & NERVE. 1981;4(3):186-97. EPUB 1981/05/01. 
24. MAZZONE ES, MESSINA S, VASCO G, MAIN M, EAGLE M, D'AMICO A, ET AL. 
RELIABILITY OF THE NORTH STAR AMBULATORY ASSESSMENT IN A 
MULTICENTRIC SETTING. NEUROMUSCULAR DISORDERS : NMD. 2009;19(7):458-
61. EPUB 2009/06/26. 
25. MCDONALD CM, HENRICSON EK, HAN JJ, ABRESCH RT, NICORICI A, ELFRING 
GL, ET AL. THE 6-MINUTE WALK TEST AS A NEW OUTCOME MEASURE IN 
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26. MAYHEW JE, FLORENCE JM, MAYHEW TP, HENRICSON EK, LESHNER RT, 
MCCARTER RJ, ET AL. RELIABLE SURROGATE OUTCOME MEASURES IN 
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MUSCLE & NERVE. 2007;35(1):36-42. EPUB 2006/09/14. 
27. ROSENBERG JJ, PRASAD V, GAEBLER J, DREISINGER N, MANZURI S. INDEX OF 
SUSPICION. PEDIATRICS IN REVIEW / AMERICAN ACADEMY OF PEDIATRICS. 
2008;29(4):131-6. EPUB 2008/04/03. 
 
 
 

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Appendix 1 
Sponsor Signatures 
 
Study Title: 
A Phase II, Open-Label, Extension Study to Assess the Safety and 
Efficacy of NS-065/NCNP-01 in Boys with Duchenne Muscular 
Dystrophy (DMD) 
Study Number: 
NS-065/NCNP-01-202 
Final Date: 
 21Jul2020 
 
This clinical study protocol was subject to critical review and has been approved by the Sponsor. 
The following personnel contributed to writing and/or approving this protocol: 
 
 
Signed: ___________________________________  
Date: __________________  
 
Tatsuya Horiguchi 
Senior Director, Clinical Research 
NS Pharma, Inc. 
 
 
 
Signed: ___________________________________  
Date: __________________  
 
Paula R. Clemens, MD 
Study Chair 
Professor of Neurology 
University of Pittsburgh  
 
 
 
Signed: ___________________________________  
Date: __________________  
 
Helmut H. Albrecht, MD, MS, FFPM 
Medical Monitor 
PharmaLex Development Services, LLC 
 
 
 
Signed: ___________________________________  
Date: __________________  
 
Mark J. Jaros, PhD 
Statistician 
Summit Analytical, LLC  
 
 

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Appendix 2 
Investigator’s Signature 
Study Title: 
A Phase II, Open-Label, Extension Study to Assess the Safety and 
Efficacy of NS-065/NCNP-01 in Boys with Duchenne Muscular 
Dystrophy (DMD) 
Study Number: 
NS-065/NCNP-01-202 
 
Final Date: 
 21Jul2020 
 
I have read the protocol described above. I agree to comply with all applicable regulations and to 
conduct the study as described in the protocol.  
 
 
 
Signed: 
 
 
Date:  
 
<enter name and credentials> 
<enter title> 
<enter affiliation> 
<enter address> 
<enter phone number> 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

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Appendix 3 
Summary of Changes 
 
Amendment 1 Changes to the Protocol: 
Updates to the protocol include the following changes:  
1. Change study site locations to North America 
2. Include Research Ethics Boards (REB) in addition to IRBs 
3. Include references to Health Canada where applicable 
4. Allow additional patients to enroll beyond 8 in each cohort 
 
Change 
Rationale  
Affected Area 
Added Amendment 1 and 
date. 
Identify amendment 1. 
Title page 
 Header throughout. 
Update study site locations to 
North America. 
Include participation of 
Canadian sites. 
Title Page 
Synopsis Clinical Sites. 
Addition of Research Ethic 
Boards. 
Update terminology to apply 
to Canadian sites. 
Document throughout. 
Addition of Health Canada 
and applicable regulations. 
Include participation of 
Canadian sites. 
Document throughout. 
 
 
Addition of brain natriuretic 
protein (BNP) lab collection. 
Allows collection of cardiac 
function data in this long-
term exposure to the 
investigational drug. 
Section 7.11.1. Sample 
Collection, Storage, and 
Shipping. 
Table 2 Clinical Laboratory 
Tests. 
Section 8.1.1. Day of First 
Infusion (Week 25). 
Section 8.2.1 Week 49 or 
Early Termination. 
Table 3 Blood Sample 
Volume and Number. 
 
 
 
 

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Amendment 2 Changes to the Protocol: 
Updates to the protocol include the following changes:  
1. Added information regarding EMFLAZA™ approval by the FDA 
2. Increase weekly IP infusion timeline from 48 weeks to 96 weeks 
3. Add additional site locations for infusion visits  
4. Combine week 48 blood draw assessments to week 49 
5. Provided clarification of the minimum time that must elapse between infusions 
6. Clarified details of height measurements 
7. Clarified analysis of adverse events that occur during study NS-065/NCNP-01-201 and 
are not resolved prior to enrollment into this study 
8. Clarified language regarding collection times for PK samples relative to infusion start 
time 
9. Clarified language regarding collection times for vital signs relative to infusion start time 
 
 
Change 
Rationale  
Affected Area 
Added Amendment 2 and 
date. 
Identify amendment 2 
Title page 
 Header throughout. 
Added information regarding 
EMFLAZA™ approval. 
Provide more complete 
information on DMD 
management. 
Section 1.2.1. Glucocorticoid 
Treatment. 
Increase infusion timeline 
from 48 weeks to 96 weeks. 
Streamline extension 
protocols by combining this 
24-week extension protocol 
with a proposed 48-week 
long-term extension protocol.  
Document throughout. 
Update number of 
participating sites. 
Include participation of 
regional sites for infusion 
visits to reduce participant 
travel burden. 
Title Page 
Synopsis, Clinical Sites 
Section 8.1. Treatment Phase 
Move week 48 blood draws 
to week 49. 
Reduce participant travel 
burden.  Reduce the number 
of venipunctures. 
Document throughout. 
Change Instructions from a 
minimum of 2 days to a 
minimum of 3 days (72 
hours) between infusions 
Provide clarification of 
intention. 
Section 5.3.  Instructions for 
Administration of 
Investigational Product 
Added details of height 
measurements. 
Clarification of procedure. 
Section 7.6. Weight and 
Height. 
Added language for analysis 
of AEs that occur in study 
NS-065/NCNP-01-201 and 
are not resolved upon 
enrollment into this study. 
Provide clarification for AE 
analysis. 
Section 10.5.2. Physical 
Exam and Adverse Events 

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Updated PK collection time 
language. 
Provide clarification of 
procedure times. 
7.14.1 Collection and 
Assessment of 
Pharmacokinetic Samples, 
Table 1 Schedule of Study 
Assessments 
Updated Vital signs 
collection time language. 
Provide clarification of 
procedure times. 
7.7 Vital Signs, Table 1 
Schedule of Study 
Assessments 
 
 
 
 

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Amendment 3 Changes to the Protocol: 
Updates to the protocol include the following changes:  
1. Clarified procedure for determining visit windows 
2. Added language to clarify that blood sample collection should not occur at the same 
venous site as IP infusion 
3. Corrected blood total volume by removing Early Termination samples from total 
4. Corrected order of assessments at each study visit 
5. Clarified differences between procedures at Week 97 visit and Early Termination Visit 
6. Included option for central venous access for participants who experience difficulty with 
peripheral intravenous access 
 
 
Change 
Rationale  
Affected Area 
Added Amendment 3 and 
date. 
Identify amendment 3 
Title page 
 Header throughout. 
Included sentence regarding 
weekly visit windows. 
To clarify that all visits 
should be calculated from 1st 
infusion of NS-065/NCNP-
01-201. 
Section 5.5. Treatment 
Compliance 
Added language regarding 
blood sampling during IP 
infusion. 
To clarify any blood 
sampling occurring during IP 
infusion must be collected 
from a site away from IP 
infusion. 
Section 7.11. Clinical 
Laboratory Tests 
Corrected blood total volume 
collected 
Early Termination volumes 
should not be included in 
total 
Section 7.15. Total Blood 
Volume of Clinical 
Laboratory and 
Pharmacokinetic Samples 
Table 3.  Blood Sample 
Number and Volume 
Corrected Order of 
Assessments at each study 
visit. 
To accurately reflect the 
order in which study 
procedures should be 
performed. 
Section 8.1. Treatment Phase 
Section 8.2. Post-Treatment 
Phase 
Created separate sections for 
Week 97 and Early 
Termination Visit 
To clarify differences 
between procedures at these 
visits.  Early Termination 
visit includes PK, Cytokines 
and Antibodies.  These 
procedures are not done at 
Week 97 
Section 8.2.1. Week 97 
Section 8.2.2. Early 
Termination Visit 
Added option for use of 
central line for weekly study 
infusions 
The placement of peripheral 
intravenous lines has been 
very difficult for some 
participants.  These 
Section 3.2.2. Investigational 
Product Dosing 
Section 9.12. Intravenous 
(IV) Access Considerations 

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difficulties add additional 
stress to the participant and 
families.  Site PIs have 
expressed concern about 
continual weekly access and 
the potential for participants 
to miss study infusions or 
discontinue participation.   
 
 
 

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Amendment 4 Changes to the Protocol: 
 
Updates to the protocol include the following changes:  
1. Revised to specify “implantable ports” may be used for this trial.  An alternative method 
of central venous access may only be considered in the case of a contraindication. 
2. NS Pharma must obtain surgeon credentials prior to port placement. 
3. Included reporting of serious adverse events suspected to be related to the port to the 
FDA and Health Canada 
 
Change 
Rationale 
Affected Area 
Added Amendment 4 and 
updated version date. 
Identify Amendment 4 
Title page 
Appendix 1 – Sponsor 
Signatures 
Appendix 2 – Investigator 
Signatures 
Header throughout document 
Added CVA to list of 
abbreviations 
Identify the abbreviation and 
provide definition 
List of Abbreviations 
Specified “implantable 
central venous access ports” 
An implantable port is the 
preferred option of the 
sponsor for central venous 
access given the reported 
lower infection rates and 
lower replacement rates. 
Section 3.2.2. Investigational 
Product Dosing 
Section 9.12. Intravenous 
(IV) Access Consideration 
Sites that request use of 
implantable port must provide 
documentation of consulting 
surgeon’s hospital privileges 
as a board eligible/board 
certified surgeon. 
To be consistent with the 
sponsor’s rationale for 
continued positive 
benefit/risk ratio with 
implantable ports. 
Section 3.2.2. Investigational 
Product Dosing 
Section 9.12. Intravenous 
(IV) Access Consideration 
Indicated that alternative 
methods of CVA will only be 
considered in cases of 
contraindication. 
Although, an implantable port 
is the preferred option, the 
sponsor may allow alternative 
methods based on the best 
judgement of site PI and 
consulting surgeon. 
Section 3.2.2. Investigational 
Product Dosing 
Section 9.12. Intravenous 
(IV) Access Consideration 
Indicated the sponsor will 
report any serious adverse 
events suspected to be related 
to the CVA port to the FDA 
and HC 
Risks associated with 
implantable CVA ports can 
become a serious 
complication.  
Documentation and 
notification will ensure 
continued positive 
benefit/risk ratio of the study. 
Section 9.7 Reporting of 
Serious Adverse Events to 
Regulatory Authorities 
 

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Amendment 5 Changes to the Protocol: 
 
Updates to the protocol include the following changes:  
1. Revised to include new concentration of investigational product (IP). 
2. Revised the study visit window to +/- 3 days. 
3. Revised to not allow IP infusions at regional centers when participant weight needs to be 
collected. 
 
Change 
Rationale 
Affected Area 
Added Amendment 5 and 
updated version date. 
Identify Amendment 5 
Title page 
Appendix 1 – Sponsor 
Signatures 
Appendix 2 – Investigator 
Signatures 
Header throughout document 
Added new concentration of 
IP (50 mg/mL) 
New strength of IP is 250 mg 
vial in 5mL saline.  Both 
strengths need to be retained 
since the original strength and 
new strength will both be 
used during the course of this 
study. 
Study Synopsis 
Section 5.1 Description of 
NS-065/NCNP-01 
Updated study visit windows 
to +/- 3 days 
To accommodate participant 
and site schedules as this 
study requires weekly site 
visits over 72 weeks.  72-
hours between infusions shall 
be maintained for safety 
reasons, as stated in the 
protocol. 
Section 5.5 Treatment 
Compliance 
Table 1 Schedule of Study 
Assessments 
Removed “weight” as a study 
procedure allowed to be done 
at regional infusion centers 
To maintain consistency in 
proper weight collection and 
to have more consistent PI 
oversight by requiring 
participants to return to 
central study site more often. 
 
Section 8.1 Treatment Phase 
 
 
 
 

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Amendment 5.1 Changes to the Protocol: 
 
Updates to the protocol include the following changes:  
• Corrected “Amendment 5.0 Changes to the Protocol” 
 
Change 
Rationale 
Affected Area 
Added Amendment 5.1 and 
updated version date. 
Identify Amendment 5.1 
Title page 
Appendix 1 – Sponsor 
Signatures 
Appendix 2 – Investigator 
Signatures 
Header throughout document 
Removed item #4 from list of 
Amendment 5.0 Changes 
Language indicating BNP lab 
sample could be collected 
with lab samples collected for 
the NS-065/NCNP-01-201 
Week 25 study visit was not 
removed from the protocol. 
Appendix 3, Summary of 
Changes; “Amendment 5.0 
Changes to the Protocol” 
 
 
 

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Amendment 6 Changes to the Protocol: 
 
Updates to the protocol include the following changes:  
1. Revised to replace possibility of regional infusions with home infusions. 
  
Change 
Rationale 
Affected Area 
Added Amendment 6 and 
updated version date. 
Identify Amendment 6 
Title page 
Appendix 1 – Sponsor 
Signatures 
Appendix 2 – Investigator 
Signatures 
Header throughout document 
Changed number of study 
sites from 25 to 6 
The accurate number of study 
sites participating in this trial 
is 6.  No additional sites will 
participate. 
Title page 
Study Synopsis 
Replaced possibility of 
regional infusions with home 
infusions 
To offer a more convenient 
option for IP infusions 
Section 8.1 Treatment Phase 
Removed paragraph detailing 
the W25 visit window as +/- 
3 days 
Paragraph was now 
unnecessary as all study visit 
windows were updated to +/- 
3 days. 
Study Synopsis 
Section 8.1.1. Day of First 
Infusion (Week 25) 
Updated wording about 
length of infusion to describe 
“approximately 1-hour”.  
To account for variability in 
IV pump rate. 
 
Section 7.14.1 Collection and 
Assessment of 
Pharmacokinetic Samples 
 
Updated to include central 
pharmacy of home infusion 
company as a recipient of IP 
and IP accountability 
Home infusion company will 
store drug at their central 
pharmacy and ship to 
participants’ homes as needed 
for weekly infusions 
Section 5.0 Investigational 
Product 
Identified which visits may 
be home infusion visits on 
Schedule of Procedures 
To identify which visits may 
be done in a home setting. 
Table 1 
Corrected Time to Stand 
point scale  
Time to Stand point scale is a 
6-point scale 
Section 7.13.1 Time to Stand 
(TTS) – Primary Efficacy 
Endpoint 
 
 
 

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Amendment 7 Changes to the Protocol: 
 
Updates to the protocol include the following changes:  
1. Extended study to 168 weeks or until IP is commercially available 
  
Change 
Rationale 
Affected Area 
Added Amendment 7 and 
updated version date. 
Identify Amendment 7 
Title page 
Appendix 1 – Sponsor 
Signatures 
Appendix 2 – Investigator 
Signatures 
Header throughout document 
Increase infusion timeline 
from 96 weeks to up to 168 
weeks or until study drug is 
commercially available. 
To make study IP available to 
study participants until 
commercially available. 
Document throughout and 
schedule of assessment 
Interim analyses may be 
conducted 
Interim review of safety and 
efficacy data 
Synopsis 
Section 10.7: Interim 
Analyses  
Updated wording regarding 
post-dose vitals 
Clarification needed for sites 
and for re-collection of visits 
in the event of any changes 
from pre-dose vitals  
Section 7.7:  Vital Signs 
Clinical laboratory tests 
Format updates 
Table 2 
Updated wording for PK 
samples collection 
Clarification needed for sites 
Section 7.14.:  
Pharmacokinetic Assessments 
Blood volume increase 
Increase volume due to 
extension until up to 168 
weeks 
Section 7.15: Total Blood 
Volume and Table 3 
Updated description of study 
visits 
Study timeline increased to 
up to 168 weeks and 
combined visits that are alike 
Section 8.1.:  Treatment 
Phase 
Added criteria for home 
infusion 
Increase safety 
Section 8.1.:  Treatment 
Phase 
Removed language about 
further long-term study 
There will be no further long-
term study as this study will 
make IP available until 
commercially available. 
Section 8.2.3.:  Follow-up 
Phone Call 
Added reason for participant 
withdrawal, “The study drug 
becomes commercially 
available” 
The sponsor wants to make IP 
available to participants until 
commercially available  
Section 8.2.4.:   Early 
Termination or Withdrawal 
from Study 
 
 
 

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Amendment 8 Changes to the Protocol: 
 
Updates to the protocol include the following changes:  
1. Extended study to 193 weeks or until IP is commercially available 
2. Updated SAE reporting to a new central pharmacovigilance team at IQVIA 
  
Change 
Rationale 
Affected Area 
Added Amendment 8 and 
updated version date. 
Identify Amendment 8 
Title page 
Appendix 1 – Sponsor 
Signatures 
Appendix 2 – Investigator 
Signatures 
Header throughout document 
Extended study from 168 
weeks to up to 192 weeks or 
until study drug is 
commercially available. 
To make study IP available to 
study participants until 
commercially available. 
Synopsis, document throughout 
and Table 1 
Removal of statement that 
certain P1/2 and P2 studies 
are still ongoing 
P1/2 in Japan and P2 (Study 
201) are both completed  
Section 1.3.5.2 Phase II Trials 
Blood volume increase 
Increase volume due to 
extension until up to 193 
weeks 
Section 7.15: Total Blood 
Volume and Table 3 
Updated SAE Event 
Reporting  
Investigators will be notified 
by the CRO of all SAEs 
Section 9.5 
Updated SAE reporting 
vendor 
SAE reporting changed from 
PharmaLex to IQVIA 
Section 9.7 
 
 
 
 
 
 

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Amendment 9 Changes to the Protocol: 
 
Updates to the protocol include the following changes:  
1. Added pre-clinical toxicology findings 
2. Added Urine Cytology assessments 
3. Added Renal and Urinary Bladder Ultrasound assessments 
4. Revised language throughout to correspond with language in NS-065/NCNP-01-301 
protocol 
 
Change 
Rationale 
Affected Area 
Added Amendment 9 and 
updated version date 
Identify Amendment 9 
Title page 
Appendix 1 – Sponsor 
Signatures 
Appendix 2 – Investigator 
Signatures 
Header throughout document 
Added additional pre-clinical 
toxicology findings 
New pre-clinical toxicology 
findings 
Section 1.3.4 
Added language regarding 
dose interruptions 
Language aligns with NS-
065/NCNP-01-301 protocol 
Section 3.2.4 
Updated time IP preparation 
instructions 
Change based on updated 
stability data 
Section 5.3 
Added Renal and Urinary 
Bladder Ultrasound 
Safety assessment added 
based on new pre-clinical 
toxicology findings  
Table 1 
Section 7.9  
Section 8.1.6  
Section 8.1.7  
Section 8.2.1 
Section 8.2.2 
Updated 12-lead ECG 
Updated who may interpret 
ECG read out 
Section 7.11 
Updated Urinalysis from 
random collection to pre- and 
post-dose collections at 
specified visits 
Collection of pre- and post-
dose urinalysis specimens 
align with NS-065/NCNP-01-
301 protocol 
Table 1 
Table 2 
Section 8.1.6 
Section 8.1.7 
Added urine cytology 
Safety assessment added 
based on new pre-clinical 
toxicology findings 
Table 1 
Section 7.12.1  
Table 2  
Section 8.1.6  
Section 8.1.7  
Section 8.2.1 
Section 8.2.2 
Added Adverse Events of 
Special Interest  
Added adverse events which 
are considered special 
interest, which aligns with 
Section 9.5.3 

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NS-065/NCNP-01-301 
protocol 
Added Urine Analysis 
Monitoring section 
Added Monitoring of Urine 
Analysis verbiage to align 
with NS-065/NCNP-01-301 
protocol 
Section 9.11 
Removed PK assessment 
PK blood collection 
unnecessary at week 192  
Section 8.1.7 
 
 
 

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Amendment 10 Changes to the Protocol: 
 
Updates to the protocol include the following changes:  
1. Extended study to 217 weeks or until patients transition to a long-term follow-up 
program of NS-065/NCNP-01. 
2. Increase patients in the low dose cohort group from 40mg/kg/week to 80mg/kg/week. 
3. Added details regarding the COVID-19 pandemic and how it affected the conduct of the 
study. 
 
Change 
Rationale 
Affected Area 
Added Amendment 10 and 
updated version date 
Identify Amendment 10 
Title page 
Appendix 1 – Sponsor 
Signatures 
Appendix 2 – Investigator 
Signatures 
Header throughout document 
Extended study from 192 
weeks to up to 217 weeks  
To make study IP available to 
study participants until a 
long-term follow-up program 
is available. 
Synopsis, document 
throughout, Tables 1 and 3 
Increase 40mg/kg/week low 
cohort dose patients to 
80mg/kg/week 
All patients to be transitioned 
to 80mg/kg/week per 
advisement of FDA and to 
align with Japan package 
insert label approval. 
Synopsis 
Section 3.1 
Section 3.2.2.1 
Section 5.2 
Blood volume increase 
Increase volume due to 
extension until up to 217 
weeks 
Section 7.16: Total Blood 
Volume and Table 3 
COVID-19 Pandemic impact 
to study 
Added details surrounding 
actions taken in response to 
the global COVID-19 
pandemic.  
Section 3.4 
Section 10.8 
Section 11.6 
Section 11.7 
Added viltolarsen to 
description of study drug NS-
065/NCNP-01  
Updated investigational study 
drug product with generic 
drug name   
Synopsis 
Deleted Early Termination 
section 
Visit Week 217 and Early 
Termination (ET) are 
identical thus eliminated 
redundancy.  
Section 8.2.1 
Section 8.2.2 
Updated Study Signatories 
Changed signatory for NS 
Pharma due to personnel 
change 
Appendix 1