A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Design, Prospective, Phase IIa Exploratory Clinical Trial to Evaluate the Efficacy and Safety of SC Administration of GV1001 0.56 or 1.12 mg/Day in Patients With PSP
Phase: PHASE2
Conditions: Progressive Supranuclear Palsy
Sponsor: GemVax & Kael



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[Table of Contents] 
[TABLE OF CONTENTS]........................................................................................................................................................... 2
[PROTOCOL ESTABLISHMENT/REVISION HISTORY] ............................................................................................... 6
[PROTOCOL SYNOPSIS]........................................................................................................................................................... 8
[CLINICAL STUDY SCHEDULE] ........................................................................................................................................ 13
[ABBREVIATIONS AND TERMS] ....................................................................................................................................... 16
1.
CLINICAL STUDY TITLE AND PHASE.............................................................................................................. 17
1.1. 
Clinical Study Title ........................................................................................................................................................ 17
1.2.
Phase.................................................................................................................................................................................. 17
2.
STUDY SITES, PRINCIPAL INVESTIGATORS, AND SPONSOR............................................................... 17
3. 
INTRODUCTION ......................................................................................................................................................... 18
3.1.
Background and Rationale........................................................................................................................................... 18
3.1.1.
Overview of Progressive Supranuclear Palsy ............................................................................................ 18
3.1.2.
Information on Existing Therapies ............................................................................................................... 21
3.1.3.
Background of Clinical Study (Rationale)................................................................................................. 22
3.2. 
Mechanism of Action from Nonclinical Study Data of GV1001 ....................................................................... 22
3.3. 
Results of Past Clinical Studies .................................................................................................................................. 30
3.4. 
Rationale for Dose Setting ........................................................................................................................................... 31
4. 
STUDY OBJECTIVES ................................................................................................................................................. 33
4.1. 
Primary Objective .......................................................................................................................................................... 33
4.2. 
Secondary Objectives .................................................................................................................................................... 33
5. 
CLINICAL STUDY POPULATION ......................................................................................................................... 34
5.1. 
Target Number of Subjects .......................................................................................................................................... 34
5.2. 
Target Patients ................................................................................................................................................................. 34
5.3. 
Inclusion Criteria ............................................................................................................................................................ 34
5.4. 
Exclusion Criteria .......................................................................................................................................................... 34
5.5. 
Dropping Out .................................................................................................................................................................. 35
5.5.1.
Definition ........................................................................................................................................................... 35
5.5.2.
Dropout Criteria ................................................................................................................................................ 35
5.5.3.
Protocol Violations........................................................................................................................................... 36
5.5.4.
Handling of Dropouts and Protocol Violations......................................................................................... 36
6. 
DETAILS OF CLINICAL STUDY DESIGN ......................................................................................................... 38
6.1. 
Clinical Study Duration ................................................................................................................................................ 38
6.2. 
Design of Clinical Study .............................................................................................................................................. 38
6.3. 
Randomization ................................................................................................................................................................ 40
6.4. 
Blinding and Unblinding .............................................................................................................................................. 40
7. 
CRITERIA FOR STUDY TERMINATION AND EARLY DISCONTINUATION ...................................... 42
7.1. 
Termination Criteria ...................................................................................................................................................... 42
7.2. 
Discontinuation or Early Termination of Clinical Study ...................................................................................... 42
7.2.1.
Sponsor ............................................................................................................................................................... 42
7.2.2.
Investigator ........................................................................................................................................................ 42
8. 
INFORMATION ON AND MANAGEMENT OF INVESTIGATIONAL PRODUCT ............................... 43
8.1. 
Ingredients, Content, Dosage Form, etc., of Investigational Product ................................................................ 43

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8.1.1.
Study Drug 1: GV1001 0.84 mg/vial .......................................................................................................... 43
8.1.2.
Study Drug 2: GV1001 1.68 mg/vial .......................................................................................................... 43
8.1.3.
Comparator: Placebo of GV1001................................................................................................................. 43
8.2. 
Packaging and Labeling of Investigational Product .............................................................................................. 44
8.3.
Dosage, Administration Method, and Administration Period.............................................................................. 44
8.4. 
Acquisition and Dispensing Management, Collection, and Disposal of Investigational Product .............. 45
9. 
ADMINISTRATION PLAN ....................................................................................................................................... 45
9.1.
Administration and Treatment Schedule .................................................................................................................. 45
9.2. 
Permitted Concomitant Medications ......................................................................................................................... 45
9.3.
Contraindicated Concomitant Medications.............................................................................................................. 46
9.4. 
Drugs Requiring Precautions....................................................................................................................................... 46
9.5. 
Drug Compliance ........................................................................................................................................................... 46
10.
CLINICAL STUDY PROCEDURES AND EVALUATIONS............................................................................ 48
10.1. 
Clinical Study Schedule ........................................................................................................................................... 48
10.2. 
Evaluation Items by Visit ......................................................................................................................................... 51
10.2.1.
Visit 1 (Screening, From -4 Weeks)............................................................................................................. 51
10.2.2. 
Visit 2 (Randomization and Baseline, Week 1) ......................................................................................... 51
10.2.3. 
Visits 3 to 5 (Weeks 2, 3, and 4 ± 3 days) .................................................................................................. 52
10.2.4. 
Visits 6 (Weeks 6 ± 5 days) ............................................................................................................................ 52
10.2.5. 
Visits 7 to 9 (Weeks 8, 10, 12 ± 5 days)...................................................................................................... 53
10.2.6. 
Visit 10 (Week 12 [3 months] Efficacy Evaluation, Week 14 ± 5 days) ............................................. 53
10.2.7. 
Visits 11 to 14 (Weeks 16, 18, 20, and 22 ± 5 days) ................................................................................ 53
10.2.8. 
Visit 15 (End-of-Treatment Visit; Week 24 ± 5 days) ............................................................................. 54
10.2.9. 
Visit 16 (Week 24 [6 months] Efficacy Evaluation, Week 26 ± 5 days) ............................................. 54
10.2.10. 
Visit 17 (End-of-Study Visit, Week 28 ± 5 days) ..................................................................................... 55
10.2.11. 
Unscheduled Visits ........................................................................................................................................... 55
10.3.
Evaluation Methods .................................................................................................................................................. 55
10.3.1.
Subject Consent and Assignment of Screening Number ........................................................................ 55
10.3.2. 
Demographic Information .............................................................................................................................. 55
10.3.3. 
Checking Medical/Surgical History ............................................................................................................. 55
10.3.4. 
Checking Prior/Concomitant Medications ................................................................................................. 55
10.3.5.
Physical Examination...................................................................................................................................... 55
10.3.6. 
Vital Signs .......................................................................................................................................................... 56
10.3.7. 
Weight and Height ............................................................................................................................................ 56
10.3.8. 
Electrocardiography ......................................................................................................................................... 56
10.3.9. 
Laboratory Tests ............................................................................................................................................... 56
10.3.10. 
Urine hCG .......................................................................................................................................................... 57
10.3.11. 
K-MMSE ............................................................................................................................................................ 57
10.3.12. 
PSP-rating scale ................................................................................................................................................ 57
10.3.13. 
MoCA-K ............................................................................................................................................................. 57
10.3.14. 
K-FAB ................................................................................................................................................................. 57
10.3.15. 
ES ADL ............................................................................................................................................................... 57
10.3.16. 
Brain CT ............................................................................................................................................................. 58

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10.3.17. 
Collecting Human-Derived Samples ........................................................................................................... 58
10.3.18.
Investigational Product Administration ...................................................................................................... 58
10.3.19.
Adverse Event Assessment ............................................................................................................................ 58
10.4. 
Endpoints ..................................................................................................................................................................... 59
10.4.1.
Primary Efficacy Endpoint............................................................................................................................. 59
10.4.2. 
Secondary Efficacy Endpoints ...................................................................................................................... 59
10.4.3. 
Exploratory Endpoints .................................................................................................................................... 59
10.4.4.
Safety Endpoints............................................................................................................................................... 59
10.5. 
Definition and Reporting of Adverse Events ...................................................................................................... 59
10.5.1.
Definition of Adverse Events ........................................................................................................................ 59
10.5.2. 
Reporting of Adverse Events ......................................................................................................................... 60
10.5.3. 
Reporting of Serious Adverse Events .......................................................................................................... 61
10.5.4.
Reporting Procedures for Suspected Unexpected Serious Adverse Reactions (SUSARs)............. 62
10.6. 
Pregnancy .................................................................................................................................................................... 62
10.7. 
Overdosing .................................................................................................................................................................. 62
11.
DATA ANALYSIS AND STATISTICAL CONSIDERATIONS........................................................................ 64
11.1. 
Analysis Set ................................................................................................................................................................ 64
11.1.1. 
Efficacy Evaluation Set................................................................................................................................... 64
11.1.2. 
Safety Evaluation Set ...................................................................................................................................... 64
11.2. 
Statistical Analysis Method ..................................................................................................................................... 64
11.2.1. 
General Principles of Analysis ...................................................................................................................... 64
11.2.2. 
Method of Handling Missing Data ............................................................................................................... 64
11.2.3. 
Covariate Adjustment ...................................................................................................................................... 64
11.2.4. 
Interim Analysis ................................................................................................................................................ 65
11.2.5. 
Multiple Comparisons and Multiplicity ...................................................................................................... 65
11.2.6. 
Subgroup Analysis ........................................................................................................................................... 65
11.2.7.
Demographic Information and Other Characteristics Prior to Treatment........................................... 65
11.2.8.
Efficacy Analysis.............................................................................................................................................. 66
11.2.9. 
Exploratory Evaluation Analysis .................................................................................................................. 67
11.2.10. 
Safety Analysis ................................................................................................................................................. 68
11.2.11. 
Handling Data of Dropout Subjects ............................................................................................................. 69
11.3.
Analysis Period .......................................................................................................................................................... 69
11.4. 
Rationale for Setting Number of Subjects ........................................................................................................... 69
12.
DATA MANAGEMENT .............................................................................................................................................. 70
12.1. 
Data Collection/Access ............................................................................................................................................ 70
12.2. 
Data Protection/Storage ........................................................................................................................................... 70
13.
ETHICAL CONSIDERATIONS AND ADMINISTRATIVE PROCEDURES .............................................. 71
13.1. 
Relevant Laws and Regulations ............................................................................................................................. 71
13.2. 
Subject’s Consent ...................................................................................................................................................... 71
13.3. 
Indemnification Policy for Injured Subjects ....................................................................................................... 71
13.4. 
Criteria for Medical Care and Treatment of Subjects after Completion of Study Participation or 
Dropping Out ............................................................................................................................................................................... 71
13.5. 
Ethics Compliance and Protection Measures for Subject Safety ................................................................... 71
13.5.1. 
Approval and Amendment of Clinical Study Plan ................................................................................... 72

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13.5.2. 
Protocol Violations ........................................................................................................................................... 73
13.6.
Disclosure of Results................................................................................................................................................ 73
13.7.
Confidentiality of Patient Records........................................................................................................................ 73
13.8. 
Quality Control and Reliability Assurance .......................................................................................................... 74
13.8.1.
Monitoring of Clinical Study......................................................................................................................... 74
13.8.2. 
Audit and Inspection ........................................................................................................................................ 74
13.9. 
Other Matters Necessary to Conduct Clinical Study Safely and Scientifically .......................................... 74
13.9.1.
Matters Necessary to Initiate Clinical Study ............................................................................................. 74
13.9.2. 
Investigator’s Obligations .............................................................................................................................. 74
14.
REFERENCES ............................................................................................................................................................... 75
[List of Appendices]
Appendix 1. List of clinical trial participants and clinical trial pharmacists 
Appendix 2. Subject information sheet and informed consent form 
Appendix 3. Indemnification policy for injured subjects 
Appendix 4. K-MMSE 
Appendix 5. PSP-rating scale 
Appendix 6. MoCA-K 
Appendix 7. K-FAB 
Appendix 8. ES ADL 
 
 
 
 
 

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5.1
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6.4. Blinding and Unblinding: Clarification of wording 
for person in charge of dilution and administration  
 Addition of phrases regarding secondary use of human-
derived samples, etc.

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[Exploratory Endpoints]
1)
Change from the baseline in total tau (t-tau), phosphorylated tau (p-tau), and 
neurofilament light chain (NfL) markers in blood after 12 weeks (3 months), and 
in blood and cerebrospinal fluid after 24 weeks (6 months) of the investigational 
product administration  
2) Change from the baseline in inflammatory cytokine markers (IL-6, IL-8, IL-2, 
IFN- 
TNF-
etc.) in blood after 12 weeks (3 months), and in blood and 
cerebrospinal fluid after 24 weeks (6 months) of the investigational product 
administration  
3) Change from the baseline in blood after 12 weeks (3 months), and in blood and 
cerebrospinal fluid markers after 24 weeks (6 months) of investigational product 
administration, and correlation between clinical markers and treatment responses 
 
[Safety Endpoints] 
1) Adverse events 
2) Laboratory tests (hematology test, blood chemistry test, urinalysis) 
3) Vital signs 
4) Electrocardiogram 

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[6] Collecting human-derived samples: Human-derived samples (DNA, plasma, serum, and cerebrospinal fluid) will be collected for exploratory evaluations, and they will be transferred to the central lab for analysis 
and storage. However, in the case of cerebrospinal fluid, it will be collected only from subjects who have separately consented to collection of samples. For Visit 1 (screening), separate visits for collecting human-
derived samples can be set up and implemented within the screening period. 
[7] Investigational product administration: Subjects will be observed for approximately 20 minutes after the administration of the investigational product from Visit 2 to Visit 15. 
[8] Adverse events assessment: Adverse events that occurred after the first administration of the investigational product will be identified, and prior to the first administration, it will be collected as medical history. 

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1. 
Clinical Study Title and Phase 
1.1. Clinical Study Title 
GV1001 0.56 
1.12 mg/day 
 
 
 
 
 
, 
 
, 
 
, 
 
, 
 
, 
, 
2a
 
 
 
A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel design, Prospective, Phase IIa 
Exploratory Clinical Trial to evaluate the efficacy and safety of Subcutaneous Administration of GV1001 0.56 or 
1.12 mg/day in Patients with Progressive Supranuclear Palsy 
 
1.2.
Phase
Phase 2a clinical study
 
2. 
Study Sites, Principal Investigators, and Sponsor 
See [Appendix 1]. List of clinical trial participants and clinical trial pharmacists 
 

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3. 
Introduction 
3.1. Background and Rationale 
3.1.1. Overview of Progressive Supranuclear Palsy 
repeat (4R) tau protein aggregation and deposition, followed by progressive death of brain neurons, resulting in 
motor and cognitive impairment as well as emotional and behavioral regression. It shows rapid progression after 
the onset of symptoms and the patient dies within 5 to 8 years on average. As with most neurodegenerative diseases, 
it is difficult to confirm the exact prevalence or incidence of PSP because adequate population-based surveys have 
not been conducted. However, the prevalence is known to be 5 to 7 patients per 100,000 of the population1)–3)
because of the rare occurrence of the disease worldwide. It has been reported that the proportion of patients 
diagnosed with PSP among patients who visit hospitals with an onset of Parkinsonism is approximately 5 to 6%.4), 
5)
The pathologic mechanism of PSP is yet to be fully identified, but various mechanisms in the function and 
metabolism of tau protein in the brain nervous system are known to eventually result in aggregation of tau protein 
in cells and subsequent death of brain neurons that rapidly spreads into the brain nervous system, resulting in 
atrophy and degeneration of the brain.  
The tau protein is a microtubule-associated protein (MAP) that stabilizes intracellular microtubules in the axon, a 
branch extending from the cell body of a neuron, and it mainly plays the role of regulating the assembly and 
migration of microtubules, spatial arrangement and composition, and transport through the axons of small 
organelles. The tau protein is finally synthesized as a protein after transcription and translation from a gene, and 
in this process, it undergoes modification processes such as phosphorylation, acetylation, and breaks to finally 
synthesize into a protein. In addition to PSP, neurodegenerative diseases characterized by the accumulation of tau 
protein such as Alzheimer’s disease, corticobasal degeneration (CBD), Pick’s disease, and frontotemporal 
dementia and parkinsonism linked to chromosome 17 (FTDP-17) are called tauopathies. The aforementioned 
protein synthesis process plays an important role in the pathophysiology that induces functional abnormalities of 
tau protein in tauopathies including PSP.  
 
 
 
 
 
 

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Figure 1 The MAPT locus, its pathological mutations, and tau protein isoforms
The tau protein is generated from the microtubule-associated protein tau (MAPT) gene (i.e., the tau gene) located 
at the q21 locus (17q21) of chromosome 17. The fact that mutations in the MAPT gene can cause FTDP-17 clearly 
supports the causal role of tauopathy in neurodegeneration.6) The tau gene consists of a total of 16 exons numbered 
from 0 to 14.7) Among them, according to the number of C-terminal (31 or 32 amino acids) amino acid repeats 
generated in exon 10 depending on the alternative splicing of exons 2, 3, and 10, it is classified as 3-repeat (3R) 
or 4-repeat (4R), respectively, if there are 3 or 4 repeated sequences. In addition, according to the number of 
insertion sequences of N-terminal (29 amino acids) present in exons 2 and 3, respectively, it is classified as 0N, 
1N, or 2N if the number of insertion sequences is 0, 1, or 2. Summarizing the above, 6 tau isoforms are generated, 
ranging from 352 amino acids (shortest with 3R/0N) to 441 amino acids (longest with 4R/2N) in total. PSP is 
classified as a representative neurodegenerative disease of 4R tauopathy, and it can be seen that there are some 
common aspects with Alzheimer’s disease in terms of pathophysiology[Figure 1].8)

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Figure 2 A schematic illustrating the central role of tau oligomers in neurodegeneration.
The tau monomers that are formed incorrectly during the modification process of protein synthesis are known to 
form tau oligomers, in which tau proteins are tangled and clumped together, and induce pathologies in brain 
neurons with the occurrence of hyperphosphorylation as well as cause apoptosis through various mechanisms, 
which ultimately results in neurodegenerative diseases.[Figure 2].9)
The latest strategy for the development of therapeutic agents based on the pathophysiology of tauopathies is as 
shown below[Table 1, Figure 3].8) The post-translational modification strategy is a strategy applied in common 
with therapeutic agents being developed to target Alzheimer’s disease, and the drugs are being developed as oral 
or parenteral administration drugs, rather than antibodies (passive immunity) or active immunity or gene 
therapeutic agents. 

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that are undergoing preclinical and clinical study Phases 1 and 2. 
3.1.3.
Background of Clinical Study (Rationale)
The clinical study results of the above drugs under development are still not promising, and there are no drugs 
that were able to successfully move on to a Phase 3 clinical study. In addition to PSP, new-concept clinical studies 
on new drugs for refractory neurodegenerative diseases have recently been conducted in North America and 
Europe, and drugs that have been approved by the U.S. Food and Drug Administration (US FDA) for some rare 
and refractory diseases and have begun sales are not realistically accessible for domestic patients because of their 
astronomical costs. Furthermore, since the pharmaceutical companies that developed the drugs are often overseas 
R&D companies, they do not have global sales lines, and thus are not applicable to domestic patients. Even if a 
drug is not a treatment for rare refractory diseases, various investigational drugs for relatively common 
neurodegenerative diseases such as Alzheimer’s disease or Parkinson’s disease take at least 5 to 10 years from the 
time of overseas sales in the US or Europe to their domestic introduction. Thus, waiting without appropriate 
treatment until the drugs developed overseas reach domestic sales is too dangerous for patients with 
neurodegenerative diseases whose first 3 years after diagnosis are crucial due the rapid progression of disease. 
Therefore, even in the case of PSP, which is a representative refractory neurodegenerative disease, the 
development of treatments that meet the treatment standards and circumstances of domestic patients is absolutely 
necessary in terms of the cost and efficiency of Korean healthcare.  
 
Summarizing the above, PSP is a neurodegenerative disease caused by tau protein abnormalities, and the 
pathophysiology is similar to Alzheimer’s disease in that it is a tauopathy. Therefore, based on experimental 
evidence showing that GV1001 will improve neuronal viability and reduce the amount of aggregated tau, and the 
results of a preceding Phase 2 clinical study (KG6/2016) on Alzheimer’s disease that was conducted domestically, 
there is a need to develop it as a treatment for PSP as well. PSP, which is 3 times faster than relatively common 
neurodegenerative diseases such as Alzheimer’s disease or Parkinson’s disease and is severely disabling, has no 
realistic treatment, and thus, the development of a treatment is desperately needed. 
 
This clinical study has been planned in the form of a Phase 2 clinical study to scientifically verify the possibility 
of developing GV1001 as a treatment for PSP. Since GV1001 has already been studied in large-scale clinical 
studies on cancer diseases and Alzheimer’s disease, and safety data have also been obtained from elderly patients 
with dementia, it is considered that the additional risks that occur in the process of developing it as a treatment 
for refractory neurodegenerative diseases such as PSP will be minimal. In addition, it is anticipated that it can 
become a breakthrough treatment if it is able to improve the symptoms that substantially lower the quality of life 
or partially reduce the speed of rapid progression that threatens life in PSP patients who have no existing 
treatments. Since the initial development process is conducted domestically, it has the conditions to be a very 
realistically useful drug that can be immediately applied to domestic patients if the clinical study is successful and 
product approval is obtained in the future. Therefore, there is an even more desperate need to conduct the clinical 
study domestically in Republic of Korea. 
 
3.2. Mechanism of Action from Nonclinical Study Data of GV1001 
- 
 
 
 
 
 
 
  
- 
No serious adverse events were reported for GV1001 in previously conducted nonclinical and clinical studies. 
In particular, safety in humans has been confirmed in various clinical studies on several target diseases in 

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Summarizing the above results, the safe starting dose for the PSP clinical study was 0.56 mg/day (
 
), and the second dose was determined as 1.12 mg/day (
 
).
 
  
In addition, at the previously implemented doses of GV1001 0.56 to 1.12 mg/day used in patients approved for 
therapeutic use of the drug, no significant adverse events were reported, similar to other clinical studies, and 
significant clinical improvements were shown in the change of the PSP-RS score. Moreover, since results of 
improvement could be observed in other clinical symptoms, it was determined that the dose of 0.56 to 1.12 mg/day 
is appropriate in this clinical study. 
 
Based on the above considerations, this clinical study intends to conduct a final validation on the efficacy and 
safety of two doses of GV1001 0.56 mg/day and 1.12 mg/day. 
 
 

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4. 
Study Objectives 
4.1. Primary Objective 
To explore the efficacy and evaluate the safety of GV1001 0.56 mg/day or 1.12 mg/day administered for 24 weeks 
on the severity of disease in patients with progressive supranuclear palsy. 
4.2. Secondary Objectives 
To collect source data to determine the possibility of progression to the next stage of development and to 
subsequent clinical studies by analyzing the clinical and biological parameters associated with the treatment 
response and safety information of 2 dose levels of GV1001, 0.56 mg/day or 1.12 mg/day, in patients with 
progressive supranuclear palsy.  
 

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4)
Patients with acute or unstable cardiovascular disease, uncontrolled hypertension (exceeding 160/100 
mmHg), uncontrolled diabetes (insulin-dependent or HbA1c >8%), or any other medical condition that can 
interfere with completing the clinical study 
5) 
Patients with hypersensitivity reactions to the ingredients of the investigational product 
6) 
Patients with a history of cancer within 5 years prior to screening (however, nonmetastatic basal cell 
carcinoma of the skin, squamous cell carcinoma of the skin, carcinoma in situ of the cervix, or 
nonprogressive prostate cancer are allowed) 
7) 
Patients with abnormal renal function (creatinine clearance [CLcr] < 30 mL/min on screening test) 
8) 
 2.5 times the upper limit of normal on 
screening test) 
9) 
 35 kg 
10) The woman of childbearing potential and male subjects who do not agree to contraception using medically 
acceptable methods (surgical sterilization, intrauterine contraceptive device, tubal ligation, double-barrier 
methods [combined use of barrier methods such as male condoms, female condoms, cervical caps, 
contraceptive diaphragms, and contraceptive sponges]), a single-barrier method with spermicide, and 
complete abstinence) during the clinical study and up to 90 days after the end (discontinuation) of their 
participation in the clinical study. 
However, women who have gone through menopause* or women or men who have undergone surgical 
sterilization (vasectomy, bilateral tubal ligation, etc.) before participating in the clinical study can participate 
even if they do not agree to contraception. 
* In this clinical study, menopause is defined as one or more of the following 
 
 
(3) In case of artificial menopause caused by bilateral oophorectomy 
11) Pregnant or breastfeeding women 
12) Patients who participated in another clinical study within 4 weeks prior to screening and were administered 
investigational products or were applied investigational medical devices 
13) Patients who were administered the study drug (GV1001) of this clinical study within 12 months prior to 
screening 
14) Patients who participated in a clinical study for progressive supranuclear palsy within 6 months prior to 
screening  
15) Other patients judged by the investigator as ineligible to participate in this clinical study  
 
 
5.5.
Dropping Out
5.5.1. Definition 
Subjects who have been randomized but are unable to participate for the entire duration of the clinical study for 
any reason will be classified as dropouts. Subjects may discontinue the study at any time at their own request, or 
may be dropped out at any time at the discretion of the investigator or sponsor for safety, behavioral, or 
administrative reasons.  
The investigator shall ask the subject regarding the reason for dropping out, and request the subject to visit the 
study site for the last time to undergo the tests performed at Visit 16. If applicable, every effort shall be made to 
follow up on the subject with regard to unresolved adverse events.  
 
5.5.2. Dropout Criteria 
The following are the cases in which the subjects may request to discontinue the clinical study, or in which they 
may be discontinued or dropped out according to a discussion between the investigator and the sponsor.  
 The subject or the subject’s representative has withdrawn consent to participate in the clinical study. 

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 Violation of the inclusion/exclusion criteria is found during the clinical study. 
 Continuation of the clinical study is difficult because of adverse events or serious adverse events. 
 There are issues in administering the investigational product to the subject. 
 The subject is lost to follow up. 
 The subject has taken a prohibited concomitant medication, or it is determined necessary to administer a 
prohibited concomitant medication. 
 The subject has become pregnant during the clinical study. 
 Other cases in which the sponsor or the investigator determines that conducting the clinical study is not 
appropriate  
 
For other matters related to the administration schedule of the investigational product, whether to drop out a 
subject will be determined based on the criteria described below. 
A violation of the drug administration schedule will be treated as a minor protocol violation up to 5 times, and it 
will not be a condition for dropping out a subject within this limit. However, if it exceeds 5 times, the subject will 
be dropped out. The administration window of the drug for the first 4 weeks will be ±3 days (however, excluding 
Visit 2), and thereafter, the window will be ±5 days for the biweekly administration days. If the window of the 
administration day is exceeded, it will be treated and recorded as a minor violation. If the window has passed, the 
corresponding drug administration will be skipped and an injection will be given at the next administration day 
that was originally scheduled. If an injection is given before reaching the window of the administration day (e.g., 
at -6 days), the schedule will be adjusted by moving all subsequent administration days forward by one week. If 
the subject arrives at the visit day for the PSP-rating scale evaluation without 12 hours of off-medication for oral 
drugs that are being taken, the evaluation will be performed in that state, but it will be recorded as a minor protocol 
violation. However, the off-medication evaluation rules shall be followed for the remaining visit days. 
 
5.5.3. Protocol Violations 
The principal investigator and staff members of this clinical study shall fully understand and thoroughly 
implement the protocol to prevent violations of the protocol. To comply with the administration and test schedule 
of the investigational product in this clinical study, the site staff shall provide written notifications or make 
telephone calls regarding the study site visit schedule to allow the subjects to make visits on the applicable days 
without omission. However, protocol violations that occur unavoidably will be handled as shown below. 
In the case of major protocol violations, the corresponding subject will be dropped out from the analysis (excluded 
from the per-protocol set) as a rule, and the applicable situations are as follows: 
 Informed consent form was not obtained. 
 The inclusion/exclusion criteria were violated. 
 Prohibited concomitant medications were administered during the clinical study. 
 The PSP-rating scale test of Visit 1 (screening), which is the first visit of this study, and Visit 16 (Week 
26) were missed. 
 
Other minor protocol violations that are judged as having no impact on the interpretation of the clinical study 
results will be included in the per-protocol set analysis by accurately describing the extent of violation or delay 
and the reason, and comprehensively considering whether the clinical study has been affected through a blind 
meeting prior to database lock.  
 
5.5.4. Handling of Dropouts and Protocol Violations 
Any of these situations shall be documented in the subjects’ case report forms and source documents. For subjects 
who have been dropped out, the relevant reason will be recorded in the Clinical Trial Completion section of the 
case report form. Even if a subject has been dropped out, the results of previous tests shall be included in the 
analysis. 
In the case of a subject who has been dropped out because of an adverse event, the subject will be followed up 

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until the subject recovers to the state prior to investigational product administration or to the baseline, or until the 
principal investigator can determine that the adverse event has normalized. 
After randomization, subjects who discontinue participating in the clinical study or drop out, regardless of the 
reason, shall not be replaced. 
 

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6. 
Details of Clinical Study Design 
6.1. Clinical Study Duration
Approximately 24 months from the approval date of the Ministry of Food and Drug Safety and the Institutional 
Review Board (IRB) of the study site 
Subject enrollment duration: Approximately 10 months 
- 
Duration of participation for each subject: Approximately 8 months (32 weeks in total—4 weeks for 
screening, 24 weeks for treatment, 4 weeks for safety follow-up) 
- 
Data management, statistical analysis, and completion of clinical study report after the end of study: 
Approximately 6 months 
 
However, the planned duration of study may be extended or shortened depending on the speed of subject 
enrollment, changes in the procedures after the end of study, etc. 
 
6.2.
Design of Clinical Study
This is a 24-week, multicenter, randomized, double-blind, placebo-controlled, parallel design, prospective, Phase 
2a exploratory clinical study.  
 
If the subject and/or the subject’s representative provide a written consent to participate in this clinical study, the 
required examinations and tests will be performed at the screening visit, and the screening period will run for 4 
weeks or shorter. 
Subjects who are ultimately determined as eligible by the inclusion/exclusion criteria after screening will be 
randomized at a 1:1:1 ratio to Study Group 1 (GV1001 0.56 mg/day), Study Group 2 (GV1001 1.12 mg/day), or 
the placebo group depending on the study site in which they are enrolled. Depending on the randomization results, 
subjects will be administered the investigational product (study drug or placebo) once weekly for the first 4 weeks 
(1 month), and then administered 10 times at 2-week intervals for 20 weeks (5 months) for a total of 14 doses over 
24 weeks (6 months).  
All subjects will visit the study site according to the planned clinical study schedule to receive the investigational 
product and to be evaluated for efficacy and safety. To ensure the objectivity and accuracy of the study results, the 
efficacy evaluators evaluating the PSP-rating scale will be limited to neurologists who have been sufficiently 
educated and trained, and the collection of efficacy and safety evaluation data and biomarkers will be performed 
in a consistent order at each visit. 
’s participation in this 
clinical study (prior to screening) will be continued without changing the dose during the clinical study 
participation period. 
 

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[Clinical Study Schematic]

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6.3. Randomization 
Subjects who provide a written consent to participate in this clinical study will be assigned a screening number 
consisting of [study site number: 2 digits]-S-[serial number: 3 digits] in the order in which the written consent 
was obtained. Subjects who meet the inclusion and exclusion criteria will then be randomized according to the 
randomization plan, and will be assigned a unique randomization number consisting of [study site number: 2 
digits]-R-[serial number: 2 digits] in the order in which the investigator decided to enroll the subjects. Subjects 
who meet the inclusion and exclusion criteria will be assigned sequentially according to the order of randomization 
at each study site.  
 
A randomization table will be generated by an independent statistician not related to this clinical study prior to 
the start of the clinical study by using SAS® (Version 9.4 or higher; SAS institute, Cary, NC, USA), and the table 
will be generated to allow assignment of subjects to Study Group 1, Study Group 2, or the control group at a 1:1:1 
ratio by applying a stratified block randomization method. Subjects who are enrolled in this clinical study and 
ultimately determined to be eligible to participate in the clinical study will be assigned a randomization number 
sequentially through the Interactive Web Response System (IWRS), and afterward, the subjects will be assigned 
to Study Group 1, Study Group 2, or the control group and receive a unique IP number.  
 
6.4. Blinding and Unblinding 
To maintain double-blinding, a placebo that is indistinguishable from the investigational product in appearance 
will be used in this clinical study. In addition, the exterior of the investigational product container will be masked 
using an opaque plastic cover and sticker, and a label will be affixed on the surface to ensure that double-blinding 
is strictly maintained. However, to minimize the possibility of unblinding the investigator and the subjects in the 
management, transfer, preparation (diluting lyophilized powder in normal saline), and administration processes 
of the investigational product at the study site, the management of the investigational product shall be conducted 
as described below. 
 
 
For each study site, a clinical trial pharmacist who manages the investigational product, a person in 
charge of diluting the investigational product, and a person in charge of administration will be 
designated in advance.  
 
Dilution and administration of the investigational product may only be performed by a designated 
individual, and the person in charge of dilution and administration should be distinguished. 
 
The person in charge of dilution and administration shall be trained in advance on the dilution and 
management of the investigational product and shall have a training log. 
 
Furthermore, to maintain double-blinding, the details of assigning unique codes to each group will not be disclosed 
until the end of the clinical study.  
The details of assigning unique codes to each group will be managed by the Interactive Web Response System 
(IWRS) of the central registration center. In principle, the randomization codes shall maintain a blinded state for 
the treatment group assignment of each subject until the database lock is completed after the end of the clinical 
study (completion of the blinded study period for all subjects or dropping out of all subjects). 
 
Double-blinding may be unblinded in the case that viewing of the relevant codes is inevitably necessary due to 
the occurrence of emergency situations such as serious adverse drug reactions, or if the sponsor determines that it 
is absolutely necessary for the rights, interests, and safety of the subjects. When double-blinding is unblinded, 
management will be conducted to allow only the viewing of the unique codes of relevant subjects.  
 
Unblinding shall be considered on a case-by-case basis and only in the event of a serious medical emergency. In 
general, blinding shall be unblinded only if the information on the treatment group affects the subjects’ treatment. 
If the principal Investigator or the sponsor deems it is necessary to unblind, the sponsor or the principal 
investigator will be contacted to obtain consent for unblinding, and then the principal investigator or the sponsor 

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will unblind. In this case, the unblinding will be recorded and kept as a document. Subjects whose randomization 
codes have been unblinded may not continue in this clinical study and will be handled as dropouts.  

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7. 
Criteria for Study Termination and Early Discontinuation 
7.1. Termination Criteria 
When the procedures of Visit 16 (Week 26), which is the subjects’ Week 24 (6 months) efficacy evaluation visit, 
are completed after starting investigational product administration, this clinical study will be deemed completed.  
However, if there are adverse events that have not disappeared at Week 24 weeks (6 months), safety follow-up 
will be performed until Week 28 (7 months) thereafter, or if possible, until the corresponding adverse events 
disappear. This will be conducted either by the subject visiting the study site in person or by the investigator or 
the investigator’s delegatee calling the subject over the telephone to collect relevant information such as whether 
the adverse event has disappeared and the date of resolution.  
The termination visit of the last enrolled subject is defined as the end of the entire clinical study. 
7.2. Discontinuation or Early Termination of Clinical Study 
7.2.1. Sponsor 
If the study site, investigator, or the sponsor’s delegatee fails to comply with basic obligations, including the 
Korean Good Clinical Practice (KGCP), the protocol, the contract terms, and relevant laws and regulations, the 
sponsor shall immediately make corrections and takes actions. In the case that the following should occur, the 
sponsor may decide to early discontinue the entire clinical study or the clinical study at specific study sites. 
1) 
There is a failure to enroll the target number of subjects at all or specific study sites. 
2) 
Efficacy/safety data have emerged that can significantly affect the continuation of the clinical study. 
3) 
The extent of violation committed by the study site or the investigator with regard to the KGCP, protocol, 
contract terms, and relevant laws and regulations has caused issues with the continuation of the clinical study. 
4) 
Other administrative reasons that can have a significant impact on the continuation of the clinical study 
 
In the case that the clinical study is terminated early or discontinued, the sponsor shall promptly report the relevant 
facts and reasons in writing to the principal investigator and the Minister of Food and Drug Safety, and in the case 
of a multicenter clinical study, the relevant facts and reasons shall also be notified in writing to the principal 
investigators of other study sites.  
7.2.2. Investigator 
If the sponsor terminates or discontinues the clinical study early, the principal investigator shall immediately notify 
this fact to the Institutional Review Board, submit a detailed statement of reasons for early termination and 
discontinuation, and also immediately notify the subjects and ensure that appropriate actions are taken and follow-
ups are conducted. 
 
If the principal investigator intends to terminate or discontinue the clinical study early, the principal investigator 
shall inform the sponsor in advance, immediately notify the Institutional Review Board of the decisions and the 
reasons, and submit a detailed statement of reasons for early termination and discontinuation.  
 
 

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be administered as soon as possible within a maximum of 6 hours after dilution, and it should be refrigerated at 2 
to 8°C until administration.  
It is recommended to use a 
mL unibody syringe for the administration of GV1001. If the injection is too 
shallow, loss of drug will occur at the injection site during the injection or after removing the needle. 
Administration period of the study drug and the placebo 
The study drug or the placebo will be administered once weekly for the first 4 weeks (1 month), and then 
administered 10 times at 2-week intervals for 20 weeks (5 months) for a total of 14 subcutaneous doses over 24 
weeks (6 months). 
8.4. Acquisition and Dispensing Management, Collection, and Disposal of Investigational 
Product 
1)
The investigational product (study drug and placebo) shall be stored in a frozen (-25 to -15°C) and sealed 
container, and it shall not be used without the instruction (prescription) of the principal investigator and the 
person in charge.  
2) 
The sponsor shall directly deliver the investigational product, etc., to the clinical trial pharmacist after 
consulting the principal investigator and shall obtain and keep a certificate of receipt.  
3) 
The clinical trial pharmacist shall store and manage the investigational product, etc., to ensure that they are 
not used for purposes other than the clinical study. The clinical trial pharmacist shall perform tasks such as 
receiving the investigational product, inventory management, administration by subject, and return, as well 
as record relevant information and regularly notify the principal investigator of the relevant information. 
4) 
The sponsor shall check the quantity and storage conditions of the investigational product, etc., during the 
clinical study and take actions to ensure that the clinical study is conducted appropriately. 
5) 
The sponsor shall collect and dispose of unused investigational products, etc., in the case that the clinical 
study is discontinued or terminated, or issues occur in the investigational product, or the shelf life or 
expiration date is exceeded. In such cases, the clinical trial pharmacist shall return the unused investigational 
products to the sponsor and a certificate of their return shall be kept. 
6) 
The container of the investigational product dispensed to and used by the subjects during the clinical study 
shall be disposed of immediately after administration of the investigational product at each study site in 
accordance with the regulations of the respective study sites. In case of disposal, the relevant records shall 
be kept. 
9.
Administration Plan
9.1. Administration and Treatment Schedule 
For 24 weeks (6 months) according to the randomization results, this clinical study will administer GV1001 0.56 
mg/day for Study Group 1, GV1001 1.12 mg/day for Study Group 2, and a placebo of GV1001 for the control 
group once weekly for the first 4 weeks (1 month), and thereafter 10 times at 2-week intervals for 20 weeks (5 
months) for a total of 14 subcutaneous doses over 24 weeks (6 months). 
See section 8.3 Dosage, Administration Method, and Administration Period for details. 
9.2. Permitted Concomitant Medications 
Since there is no treatment that has been clearly proven to improve the symptoms of progressive supranuclear 
palsy, which is the target disease of this clinical study, it is considered that there will be few concomitant 
medications that have a significant effect on the determination of efficacy of the study drug. However, as a rule, 
anti-parkinsonian drugs that were being administered orally and cognitive function-improvement drugs that were 
being administered orally or transdermally, shall be maintained at the same dose during the clinical study. All 
other drugs prescribed symptomatically, such as for gastrointestinal disorders, pain, and colds, will be permitted 

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in the case that a change in the dose or regimen is required, discontinuation of administration is necessary, or new 
administration is needed. The rules for permitted concomitant medications are further summarized below.  
 Concomitant medications that the subject has been taking prior to participating in this clinical study that are 
considered to not affect the interpretation of the results of this clinical study will be permitted based on 
judgment of the clinical study doctor.  
 Drugs used temporarily for the treatment of other diseases or adverse events shall be concomitantly 
administered after a discussion with the investigator. 
 Dopaminergic drugs that were being taken at a stable dose for at least 1 month prior to the screening visit 
shall be taken without changing the dose during the clinical study.  
 When administering any concomitant medication (including drugs for treatment of other diseases or adverse 
events), the information on the corresponding drug (product name, purpose of administration, dose 
administered, administration period, etc.) will be recorded in detail in the case report form. 
 
9.3.
Contraindicated Concomitant Medications
The medications listed below are prohibited from concomitant administration during the clinical study.
 
All investigational drugs or medical devices not specified in this clinical study 
 
Abuse of any drugs (including, but not limited to, illegal amphetamines, cannabis, cocaine, illegal opiates, 
propoxyphene, methadone, methaqualone, phencyclidine, or illegal barbiturates) 
 
Regular use of narcotics (3 days a week or more) 
 
Administration of other drugs that affect the immune system (immunosuppressants, oral or parenteral 
steroids, etc.) 
 
Anticancer drugs 
 
9.4. Drugs Requiring Precautions
The following drugs may be taken during the clinical study, but they shall be administered with caution, and the 
information on the corresponding drug (product name, purpose of administration, dose administered, 
administration period, etc.) shall be recorded in detail in the case report form. 
 
 Anticholinergic drugs, amantadine, levodopa, dopamine agonists: These are allowed if the same stable dose 
was maintained for at least 1 month prior to screening, but if a dose adjustment is required during the clinical 
study, the reason as well as the detailed dose and regimen will be recorded. However, at the visits requiring 
an evaluation of the PSP-rating scale, the subject shall make the visit in a 12-hour off-medication state. Other 
cognitive evaluations or efficacy evaluations will be performed at least 1 hour after taking the drug. 
 
 
Drugs known to affect the central nervous system such as hypnotics (short-acting hypnotics: zolpidem, 
brotizolam lormetazepam, rilmazafone, zopiclone, ramelteon), anticonvulsants, atypical antipsychotics 
(seroquel, cacepin, etc.), anti-anxiety drugs (benzodiazepines [chlordiazepoxide, diazepam, alprazolam, 
lorazepam], buspirone, hydroxyzine), antidepressants (tricyclic antidepressants, antidepressants other than 
MAO inhibitors, selective serotonin reuptake inhibitors [SSRIs], etc.) (However, benzodiazepines for tests 
such as CTs or MRIs at visits are acceptable.) 
 
 Vitamin E, Ginko biloba extracts, estrogen, and brain function-improvement drugs (citicoline, oxiracetam, 
piracetam, acetyl-L-carnitine, nimodipine, etc.) may be used during the clinical study intermittently at low a 
low dose for a short term (use for less than 2 consecutive months) without changing the dose and regimen if 
they were being administered at a stable dose since 4 week prior to study drug administration.  
 
9.5. Drug Compliance 
The drug compliance of the investigational product is based on the number of times the study drug and the placebo 

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of the study drug should be administered during the clinical study. 
Drug compliance (%) = 
Actual number of doses 
administered 
Number of doses that 
should be administered 

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[4] K-MMSE: The results of a test performed at the study site within 2 weeks prior to the screening visit may be used if available.
[5] Brain CT: This will be performed only on subjects without brain CT imaging within 12 months prior to the screening visit. 
[6] Collecting human-derived samples: Human-derived samples (DNA, plasma, serum, and cerebrospinal fluid) will be collected for exploratory evaluations, and they will be transferred to the central lab for analysis 
and storage. However, in the case of cerebrospinal fluid, it will be collected only from subjects who have separately consented to collection of samples. For Visit 1 (screening), separate visits for collecting human-
derived samples can be set up and implemented within the screening period. 
[7] Investigational product administration: Subjects will be observed for approximately 20 minutes after the administration of the investigational product from Visit 2 to Visit 15. 
[8] Adverse event assessment: Adverse events that occurred after the first administration of the investigational product will be identified, and prior to the first administration, it will be collected as medical history. 
 

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10.2. Evaluation Items by Visit 
10.2.1. 
Visit 1 (Screening, From -4 Weeks) 
After the consent procedures of the subject and/or the subject’s representative are completed and the written 
informed consent form is obtained, the following evaluations will be performed after the screening number is 
assigned. However, a written consent may be obtained prior to Visit 1 (screening), and the tests that could not be 
performed at Visit 1 (screening), they may be performed at a revisit within the screening period.  
- 
The demographic information of the subject, medical/surgical history, and prior/concomitant drug 
administration history will be investigated. 
- 
A physical examination will be performed. 
- 
Vital signs will be measured. 
- 
Weight and height will be measured. 
- 
Electrocardiography will be performed.  
(However, the results of a test performed within 3 months may be used if available, and retesting may be 
performed only once at the discretion of the investigator.) 
- 
Laboratory tests will be performed.  
(However, the results of tests performed within 4 weeks may be used if available, and retesting may be 
performed only once at the discretion of the investigator. The serum FSH tests shall be performed to 
than 1 year.) 
- 
Urine hCG pregnancy tests will be performed only for women of childbearing potential, excluding 
subjects confirmed to have undergone surgical sterilization and as being menopausal.  
(However, the subjects who performed the serum FSH test to confirm menopause when participating in 
the clinical study will also perform urine hCG pregnancy test.) 
- 
The inclusion/exclusion criteria will be checked. 
- 
The K-MMSE test will be performed.  
(However, the results of a test performed at the study site within 2 weeks prior to the screening visit may 
be used if available.) 
- 
The PSP-rating scale test will be performed.  
- 
A brain CT will be performed only on subjects without brain CT imaging within 12 months prior to the 
screening visit. 
- 
Human-derived samples (DNA, plasma, serum, cerebrospinal fluid) will be collected for exploratory 
evaluations. However, in the case of cerebrospinal fluid, it will be collected only from subjects who have 
separately consented to collection of samples. Separate visits for collecting human-derived samples can 
be set up and implemented within the screening period. 
- 
The date of the next visit will be designated. 
 
10.2.2. 
Visit 2 (Randomization and Baseline, Week 1) 
This visit will occur within 4 weeks after Visit 1 (screening, initial visit date), and the evaluations that should be 
performed at this visit are as follows:  
- 
Changes in the medical/surgical history and prior/concomitant drug administration history will be 
checked compared with the previous visit. 
- 
A physical examination will be performed. 
- 
Vital signs will be measured. 

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- 
Weight will be measured. 
- 
Laboratory tests will be performed. 
- 
Urine hCG pregnancy tests will be performed only for women of childbearing potential, excluding 
subjects confirmed to have undergone surgical sterilization and as being menopausal. 
- 
The results of all tests and evaluations including the screening visit thus far will be combined to finally 
evaluate whether the subjects who meet the inclusion/exclusion criteria have been included. 
- 
Randomization numbers will be assigned by performing randomization. 
- 
The MoCA-K test will be performed.  
- 
The K-FAB test will be performed.  
- 
The ES ADL test will be performed.  
- 
The investigational product will be administered.  
- 
The subjects will be observed for adverse events for approximately 20 minutes after investigational 
product administration. 
- 
The date of the next visit will be designated. 
 
10.2.3. 
Visits 3 to 5 (Weeks 2, 3, and 4 ± 3 days) 
- 
Changes in the prior/concomitant drug administration history will be checked compared with the 
previous visit. 
- 
A physical examination will be performed. 
- 
Vital signs will be measured. 
- 
The occurrence of adverse events after the administration of the investigational product at the previous 
visit and their details will be investigated. 
- 
The investigational product will be administered after confirming that the clinical study can be continued.  
- 
The subjects will be observed for adverse events for approximately 20 minutes after investigational 
product administration. 
- 
The date of the next visit will be designated. 
 
10.2.4.
Visits 6 (Weeks 6 ± 5 days) 
- 
Changes in the prior/concomitant drug administration history will be checked compared with the 
previous visit. 
- 
A physical examination will be performed. 
- 
Vital signs will be measured. 
- 
Weight will be measured. 
- 
Laboratory tests will be performed. 
- 
Urine hCG pregnancy tests will be performed only for women of childbearing potential, excluding 
subjects confirmed to have undergone surgical sterilization and as being menopausal. 
- 
The occurrence of adverse events after the administration of the investigational product at the previous 
visit and their details will be investigated. 
- 
The investigational product will be administered after confirming that the clinical study can be continued.  
- 
The subjects will be observed for adverse events for approximately 20 minutes after investigational 
product administration. 
- 
The date of the next visit will be designated. 
 

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10.2.5. 
Visits 7 to 9 (Weeks 8, 10, 12 ± 5 days) 
-
Changes in the prior/concomitant drug administration history will be checked compared with the 
previous visit. 
- 
A physical examination will be performed. 
- 
Vital signs will be measured. 
- 
For Visit 8 only, urine hCG pregnancy tests will be performed only for women of childbearing potential, 
excluding subjects confirmed to have undergone surgical sterilization and as being menopausal.
- 
The occurrence of adverse events after the administration of the investigational product at the previous 
visit and their details will be investigated. 
- 
The investigational product will be administered after confirming that the clinical study can be continued.  
- 
The subjects will be observed for adverse events for approximately 20 minutes after investigational 
product administration. 
- 
The date of the next visit will be designated. 
 
10.2.6. 
Visit 10 (Week 12 [3 months] Efficacy Evaluation, Week 14 ± 5 days) 
- 
Changes in the prior/concomitant drug administration history will be checked compared with the 
previous visit. 
- 
A physical examination will be performed. 
- 
Vital signs will be measured. 
- 
Weight will be measured. 
- 
Electrocardiography will be performed.  
- 
Laboratory tests will be performed.  
- 
Urine hCG pregnancy tests will be performed only for women of childbearing potential, excluding 
subjects confirmed to have undergone surgical sterilization and as being menopausal. 
- 
The PSP-rating scale test will be performed.  
- 
The MoCA-K test will be performed.  
- 
The K-FAB test will be performed.  
- 
The ES ADL test will be performed.  
- 
Human-derived samples (DNA, plasma, serum) will be collected for exploratory evaluations.  
- 
The occurrence of adverse events after the administration of the investigational product at the previous 
visit and their details will be investigated. 
- 
The investigational product will be administered after confirming that the clinical study can be continued.  
- 
The subjects will be observed for adverse events for approximately 20 minutes after investigational 
product administration. 
- 
The date of the next visit will be designated. 
 
10.2.7. 
Visits 11 to 14 (Weeks 16, 18, 20, and 22 ± 5 days) 
- 
Changes in the prior/concomitant drug administration history will be checked compared with the 
previous visit. 
- 
A physical examination will be performed. 
- 
Vital signs will be measured. 
- 
For Visit 12 and Visit 14 only, urine hCG pregnancy tests will be performed only for women of 

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childbearing potential, excluding subjects confirmed to have undergone surgical sterilization and as being 
menopausal. 
- 
The occurrence of adverse events after the administration of the investigational product at the previous 
visit and their details will be investigated. 
- 
The investigational product will be administered after confirming that the clinical study can be continued.  
- 
The subjects will be observed for adverse events for approximately 20 minutes after investigational 
product administration. 
- 
The date of the next visit will be designated. 
 
10.2.8. 
Visit 15 (End-of-Treatment Visit; Week 24 ± 5 days) 
-
Changes in the prior/concomitant drug administration history will be checked compared with the 
previous visit. 
- 
A physical examination will be performed. 
- 
Vital signs will be measured. 
- 
The occurrence of adverse events after the administration of the investigational product at the previous 
visit and their details will be investigated. 
- 
The investigational product will be administered after confirming that the clinical study can be continued.  
- 
The subjects will be observed for adverse events for approximately 20 minutes after investigational 
product administration. 
- 
The date of the next visit will be designated. 
 
10.2.9. 
Visit 16 (Week 24 [6 months] Efficacy Evaluation, Week 26 ± 5 days) 
At this visit, the Week 24 (6 months) efficacy evaluation will be performed.  
- 
Changes in the prior/concomitant drug administration history will be checked compared with the 
previous visit. 
- 
A physical examination will be performed. 
- 
Vital signs will be measured. 
- 
Weight will be measured. 
- 
Electrocardiography will be performed.  
- 
Laboratory tests will be performed.  
- 
Urine hCG pregnancy tests will be performed only for women of childbearing potential, excluding 
subjects confirmed to have undergone surgical sterilization and as being menopausal. 
- 
The PSP-rating scale test will be performed.  
- 
The MoCA-K test will be performed.  
- 
The K-FAB test will be performed.  
- 
The ES ADL test will be performed.  
- 
Human-derived samples (DNA, plasma, serum, cerebrospinal fluid) will be collected for exploratory 
evaluations. However, in the case of cerebrospinal fluid, it will be collected only from subjects who have 
separately consented to collection of samples. 
- 
The occurrence of adverse events after the administration of the investigational product at the previous 
visit and their details will be investigated. 
- 
The date of the next telephone visit will be scheduled. 
 

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10.2.10. Visit 17 (End-of-Study Visit, Week 28 ± 5 days) 
This is the study termination visit, which will be conducted over the telephone.  
- 
Changes in the prior/concomitant drug administration history will be checked compared with the 
previous visit. 
- 
The occurrence of adverse events since the previous visit and their details will be investigated. 
 
10.2.11.
Unscheduled Visits
Additional visits may be made from time to time, outside of the scheduled visits, at the request of the subject or 
the subject’s representative or as deemed necessary based on the judgment of the investigator. To this end, the 
investigator shall inform the subjects of this during their regular visits to the study site, and ensure that the subjects 
immediately contact the investigator if any adverse events should occur. The visit schedule shall not be changed 
because of the unscheduled visits. At unscheduled visits, examinations and tests may be performed at the 
discretion of the investigator.  
 
10.3. Evaluation Methods 
10.3.1. 
Subject Consent and Assignment of Screening Number 
The investigator will explain this clinical study to the subject in detail, answer questions asked by the subject, and 
request the subject to participate in the clinical study. If the subject consents to participate in the clinical study, a 
written consent will be obtained from the subject (or the subject’s representative). A screening number will be 
assigned in the order in which the written consent is obtained. 
A written consent will be obtained from the subject’s guardian as well when consent is obtained from the subject. 
The guardian and the subject’s representative may be the same or different (even if the guardian and the subject’s 
representative are the same, the guardian’s written consent shall be obtained separately).  
 
10.3.2. 
Demographic Information 
The subject’s name, initials, sex, month and year of birth, age, etc., will be investigated. 
10.3.3.
Checking Medical/Surgical History
The subject’s medical history will be investigated in detail and recorded through an interview and a review of past 
medical records. History of operations, surgical history, allergy history, etc., within the 6 months prior to screening 
(however, for malignant tumors [cancers], the history within 5 years prior to screening will be checked), present 
illness, presence or absence of hypersensitivity reactions, time of occurrence (year or month of occurrence), and 
persistence at screening will be recorded.  
10.3.4. 
Checking Prior/Concomitant Medications 
History of drug administration within 4 weeks from Visit 1 (screening) and administration status (product name, 
purpose of administration, dose administered, administration period, etc.) will be investigated. However, in the 
case of the administration history of this investigational product to check the exclusion criteria, the administration 
history within 12 months prior to screening will be investigated.  
By comparing the details confirmed in the investigation of the drug administration history at screening, the 
changes at subsequent visits will be investigated, and if there are changes in the concomitant medications, the 
relevant information shall be recorded in detail in the source documents.  
 
10.3.5. 
Physical Examination 
At all visits, a physical examination through inspection, palpation, percussion, and auscultation will be performed 

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for each subject to check the subject’s health status and to check for any adverse events. The physical examination 
will include appearance, skin, head/neck, chest/lungs, heart, abdomen, urinary/genital system, extremities, 
musculoskeletal system, nervous system, and lymph nodes.  
 
10.3.6.
Vital Signs
Blood pressure, pulse, respiratory rate, and body temperature will be measured as vital signs. This shall be 
performed prior to the planned tests as much as possible according to the clinical study schedule, and 
measurements shall be taken in a sitting position after 5 minutes of rest.  
 
10.3.7.
Weight and Height
Height will be measured only at Visit 1 (screening), and weight will be measured at Visit 1 (screening) and at each 
time point thereafter according to the set schedule. Weight will be measured after taking off outerwear and shoes 
and emptying the pockets, and the same weight scale will be used whenever possible when measuring weight.  
 
10.3.8.
Electrocardiography
For the ECG (12-lead electrocardiogram) of Visit 1 (screening), the results of a test performed within 3 months 
may be used if available, and retesting may be performed only once at the discretion of the investigator.  
 
10.3.9. 
Laboratory Tests 
For the laboratory tests of Visit 1 (screening), the results of tests performed within 4 weeks may be used if available, 
and retesting may be performed only once at the discretion of the investigator. If retesting is performed, the final 
checking of the inclusion/exclusion criteria will be based on the results of retesting. 
Among the blood chemistry test items, HbA1c and coagulation tests, thyroid test, syphilis, acquired immune 
deficiency syndrome (AIDS) test, and creatinine clearance test will be performed only at Visit 1 (screening). At 
<55 years who have experienced amenorrhea for more than 1 year. 
Hematology test
White blood cell (WBC), Red blood cell (RBC), Hemoglobin, Hematocrit, 
Platelets count, WBC differential count (Neutrophils, Lymphocytes, 
Monocytes, Eosinophils, Basophils)
Blood chemistry test
Blood urea nitrogen (BUN), Creatinine, Uric acid, Total bilirubin, Albumin, 
Total Protein, Alanine aminotransferase (ALT), Aspartate aminotransferase 
(AST), -Glutamyl transpeptidase (GTP), Alkaline phosphatase, Glucose, 
Total Cholesterol, HbA1c (performed only at screening visit)  
Serum 
Follicle 
Stimulating 
Hormone test 
FSH (Performed only at screening visit for those who require confirmation 
of menopause)
Urinalysis
Protein (Albumin), Glucose, Ketones, WBC, Blood (RBC) 
Blood coagulation test
International normalized ratio (INR) (performed only at screening visit)
Thyroid test
Thyroid stimulating hormone (TSH), free T4 (performed only at screening 
visit) 
Syphilis, AIDS tests
Venereal disease research laboratory (VDRL), or Rapid plasma reagin 
(RPR), anti-HIV (performed only at screening visit) 
Creatinine clearance 
This will be confirmed using the Cockcroft-Gault formula. (performed only 
at screening visit) 
 

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10.3.10. 
Urine hCG 
Pregnancy will be checked with a urine human chorionic gonadotropin (hCG) response test only for women of 
childbearing potential, excluding subjects confirmed to have undergone surgical sterilization and as being 
menopausal. However, for Visit 1 (screening), the subjects who performed the serum FSH test to confirm 
menopause when participating in the clinical study will also perform the urine hCG pregnancy test. 
Women of childbearing potential are defined as individuals who have experienced menarche and who have not 
undergone successful surgical sterilization (hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or 
who are not menopausal.  
In this clinical study, menopause is defined as one or more of the following. 
(1) 
 
<55 years who have had amenorrhea for more than 1 year and have a 
 
(3) In case of artificial menopause caused by bilateral oophorectomy 
 
10.3.11. 
K-MMSE 
The Korean Mini-Mental State Examination (K-MMSE) will be used to screen subjects for cognitive function at 
Visit 1 (screening). However, the results of a test performed at the study site within 2 weeks may be used if 
available. 
The K-MMSE consists of “orientation,” “memory registration,” “attention and calculation,” “memory recall,” and 
“verbal ability and visuospatial organizing ability.” The score range is from 0 to 30, with greater cognitive 
impairments resulting in lower scores.17)  
See [Appendix 4]. K-MMSE for details. 
 
10.3.12. 
PSP-rating scale
The PSP-rating scale evaluation will be performed at Visit 1 (screening), Visit 10, and Visit 16, and the baseline 
of the PSP-rating scale shall be Visit 1 (screening). At the visits requiring an evaluation of the PSP-rating scale, 
the subject shall make the visit in a 12-hour off-medication state for the oral drugs specified in advance. This is 
corresponds to a neurological examination that is directly evaluated by a neurologist who has received or is 
receiving a subspecialty training on dyskinesia, and the scores are summed according to a set form.14, 33)
See [Appendix 5]. PSP-rating scale for details. 
10.3.13. 
MoCA-K 
The Montreal Cognitive Assessment-Korean version (MoCA-K) will be used as a measure of overall cognitive 
function in patients with progressive supranuclear palsy. The MoCA-K test will be performed a total of 3 times at 
Visit 2, Visit 10, and Visit 16, and the scores will be summed according to a set form.18)
See [Appendix 6]. MoCA-K for details. 
 
10.3.14. 
K-FAB 
The Korean Frontal Assessment Battery (K-FAB) test will be used as a measure to show the extent of frontal lobe 
dysfunction. The K-FAB test will be performed a total of 3 times at Visit 2, Visit 10, and Visit 16, and the scores 
will be summed according to a set form.19)
See [Appendix 7]. K-FAB for details. 
10.3.15. 
ES ADL 
England & Schwab Activity of Daily Living (ES ADL) is a scale that distinguishes the overall performance of 
daily living in patients with Parkinson’s disease, etc., from 0 (complete dependence) to 100% (complete 
independence) based on the level of independence. The ES ADL test will be performed a total of 3 times at Visit 

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2, Visit 10, and Visit 16, and the scores will be evaluated according to a set form.20)
See [Appendix 8]. ES ADL for details. 
 
10.3.16.Brain CT
This will be performed only on subjects without brain CT imaging within 12 months prior to Visit 1 (screening).
10.3.17. Collecting Human-Derived Samples 
Human-derived samples (DNA, plasma, serum, and cerebrospinal fluid) will be collected for exploratory 
evaluations, and they will be transferred to the central lab for analysis and storage.  
Blood collection for DNA, plasma, and serum is performed at Visit 1, Visit 10, and Visit 16, in the case of 
cerebrospinal fluid, it will be collected only from subjects who have separately consented to collection of samples 
at Visit 1 and Visit 16. For Visit 1 (screening), separate visits for collecting human-derived samples can be set up 
and implemented within the screening period. 
Samples collected for exploratory evaluations will be stored for 10 years from the termination date of the clinical 
study and then disposed of according to the regulations of the relevant study site. 
The collected samples may be used for similar or comprehensive studies only if the subjects agree to provide them 
for secondary use in the Human-Derived Samples Consent form, and the samples collected will also be stored for 
10 years from the termination date of the clinical study and then disposed of according to the regulations of the 
relevant study site. 
Plasma protein and inflammatory response markers in blood and cerebrospinal fluid will be tested at the central 
lab, and the test items are as shown below. The baseline of the test results using human-derived samples shall be 
Visit 1 (screening). 
 
 
Total tau (t-Tau), phosphorylated tau (p-Tau), and neurofilament-light chain (NfL) in blood and cerebrospinal 
fluid 
 
Inflammatory cytokine markers (IL-6, IL-8, IL-2, IFN- 
TNF-
etc.) in blood and cerebrospinal fluid 
 
10.3.18. Investigational Product Administration 
This clinical study will administer GV1001 0.56 mg/day for Study Group 1, GV1001 1.12 mg/day for Study 
Group 2, and a placebo of GV1001 for the control group once weekly for the first 4 weeks (1 month), and thereafter 
10 times at 2-week intervals for 20 weeks (5 months) for a total of 14 subcutaneous doses over 24 weeks (6 
months). Observation for adverse events will be conducted for approximately 20 minutes after the administration 
of the investigational product from Visit 2 to Visit 15.  
10.3.19. Adverse Event Assessment 
Adverse events that occurred after the first administration of the investigational product will be identified, and 
prior to the first administration, it will be collected as medical history.  
 

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10.4. Endpoints 
10.4.1. 
Primary Efficacy Endpoint 
Change from the baseline in the total score of PSP-rating scale after 24 weeks (6 months) of investigational 
product administration 
 
10.4.2. 
Secondary Efficacy Endpoints 
1) Change from the baseline in the total score of PSP-rating scale after 12 weeks (3 months) of investigational 
product administration 
2) Change from the baseline in the Montreal Cognitive Assessment - Korea (MoCA-K) after 12 weeks (3 months) 
and 24 weeks (6 months) of investigational product administration 
3) Change from the baseline in the Korean Frontal Assessment Battery (K-FAB) after 12 weeks (3 months) and 
24 weeks (6 months) of investigational product administration 
4) Change from the baseline in the England & Schwab Activity of Daily Living (ES ADL) scale after 12 weeks 
(3 months) and 24 weeks (6 months) of investigational product administration 
5) Change from the baseline in the score of each domain of the PSP-rating scale after 12 weeks (3 months) and 
24 weeks (6 months) of investigational product administration 
6) Change from the baseline in the score of each item of the PSP-rating scale after 12 weeks (3 months) and 24 
weeks (6 months) of investigational product administration 
 
10.4.3. 
Exploratory Endpoints 
1) Change from the baseline in total tau (t-tau), phosphorylated tau (p-tau), and neurofilament light chain (NfL) 
markers in blood after 12 weeks (3 months), and in blood and cerebrospinal fluid after 24 weeks (6 months) 
of investigational product administration  
2) Change from the baseline in inflammatory cytokine markers (IL-6, IL-8, IL-2, IFN- 
TNF-
etc.) in blood 
after 12 weeks (3 months), and in blood and cerebrospinal fluid after 24 weeks (6 months) of investigational 
product administration  
3) Change from the baseline in blood after 12 weeks (3 months), and in blood and cerebrospinal fluid markers 
after 24 weeks (6 months) of investigational product administration, and correlation between clinical markers 
and treatment responses 
 
10.4.4.
Safety Endpoints
1)
Adverse events
2) Laboratory tests (hematology test, blood chemistry test, urinalysis) 
3) Vital signs 
4) Electrocardiogram 
 
10.5. Definition and Reporting of Adverse Events 
10.5.1. 
Definition of Adverse Events 
(1) Adverse event (AE) 
This refers to any harmful and unintended signs (including abnormal laboratory test results, etc.), symptoms, or 
diseases that occur in a subject who has been administered an investigational product, and it does not necessarily 
must have a causal relationship to the investigational product.  
 
(2) Adverse drug reaction (ADR) 
This refers to any harmful and unintended reactions that occur at any dose of an investigational product and of 
which the causal relationship to the investigational product cannot be denied. 
 
(3) Unexpected adverse drug reaction 

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This refers to a difference in the pattern of an adverse drug reaction or the extent of harm in light of the available 
information on the drug such as the investigator’s brochure or the product insert of the drug.  
(4) Serious adverse event/adverse drug reaction (serious AE/ADR) 
This refers to an adverse event or adverse drug reaction that occurs at any dose of an investigational product that 
corresponds to any one of the following:  
 
Occurrence of death or life-threatening risks 
 
Requires hospitalization or prolongation of hospitalization 
 
Results in permanent or significant disability or functional decline  
 
Occurrence of malformations or abnormalities in a fetus  
 
Other than cases 
 through 
, occurrence of drug dependence or abuse, or cases in which medically 
significant situations occur, such as hematopathies 
 
However, in this clinical study, a visit made for the following reasons will not be considered as a serious adverse 
event. 
 
Hospitalization planned prior to the clinical study 
 
Hospitalization for medical examination, cosmetic reasons, and recuperation 
 
Cases in which the time of visiting the emergency room has not exceeded 24 hours. However, it may be 
considered a serious adverse event based on the investigator’s judgment even if it does not exceed 24 hours. 
 
If a situation occurs that is considered to have a significant impact on the patient’s well-being and health status, 
even if not listed above, it shall be determined whether to consider it as a serious adverse event according to the 
medical judgment of the doctor in charge and relevant specialists, and appropriate measures shall be taken 
accordingly.  
 
10.5.2. 
Reporting of Adverse Events 
 
Adverse events will be collected during the clinical study from the time point after investigational product 
administration and up to the final visit. All medical events that occur prior to investigational product 
administration will be recorded as medical history. 
 
Adverse events shall be reported by including the name of the adverse event, duration (start date and 
disappearance date), severity, causal relationship to the investigational product, actions taken for the 
investigational product, outcome, corrective treatment for the adverse event, and whether the event is a 
serious adverse event. 
 
When recording an adverse event, the investigator shall use a comprehensive diagnosis or symptom using 
standard medical terminology rather than recording each symptom or sign.  
 
Adverse events that occur during the clinical study shall be followed up until they disappear, show stable 
outcomes, or the subject is lost to follow-up. 
 
Adverse events that occur after the end of the clinical study shall be reported only if they are serious and 
related to the investigational product. 
 
1) Severity Assessment 
The investigator will assess the severity of adverse events and serious adverse events by referring to the criteria 
described below.  
Mild 
The event causes minimal discomfort without interfering with the subject’s normal daily 
life (function), and it can be easily tolerated by the subject. 
Moderate 
The event causes discomfort that significantly interferes with the subject’s normal daily 
life (function). 

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Severe 
The event makes the subject’s normal daily life (function) impossible.
Activities of daily living are defined below. 
: All activities that occur in daily life to take care of one’s own body (e.g., bathing, dressing and undressing, eating, 
taking medications, hygiene, and personal care) 
An adverse event or serious adverse event can both be evaluated as severe according to the definition above, and 
one should not be confused for the other since a severe adverse event does not necessarily correspond to a serious 
adverse event.  
2)
Causality Assessment
The investigator will assess the causal relationship between an adverse event and the investigational product by 
referring to the criteria described below. If it is assessed as unlikely, it can be determined as “no causal relationship” 
(non-ADR). If it is assessed as other items, it will be determined that there is a causal relationship (ADR). 
 Certain 
The relationship between the administration and use of the drug, etc., is reasonable, and it is not explained 
by other drugs, chemicals, or accompanying diseases, and a clinically reasonable response is shown when 
administration of the drug, etc., is discontinued, and it is pharmacologically or phenomenologically 
conclusive when the drug, etc., is rechallenged as needed. 
 Probable, likely 
The chronological relationship between the administration and use of the drug, etc., is reasonable, and it does 
not appear to be due to other drugs, chemicals, or accompanying diseases, and a clinically reasonable 
response is shown when administration of the drug, etc., is discontinued (no information on rechallenge) 
 Possible 
The chronological relationship between the administration and use of the drug, etc., is reasonable, but it is 
also explained by other drugs, chemicals, or accompanying diseases, and there is insufficient or unclear 
information on discontinuing administration of the drug, etc. 
 Unlikely 
It is a transient case that does not seem to have a causal relationship to the administration or use of the drug, 
etc., and it can be reasonably explained as being caused by other drugs, chemicals, or potential diseases. 
 Conditional, unclassified 
More data are needed for an appropriate evaluation or additional data are being reviewed. 
 Unassessable, unclassifiable 
A determination cannot be made because the information is insufficient or contradictory, and it cannot be 
supplemented or confirmed. 
 
10.5.3.
Reporting of Serious Adverse Events
All serious adverse events that occur from the time point after investigational product administration and up to 
the final visit shall be reported to the sponsor via telephone, fax, e-mail, etc., within 24 hours of the investigator’s 
recognition, regardless of their relationship to the investigational product, and they must be reported to the IRB 
of the affiliated study site within the timeline specified by the IRB.  
 
Adverse events that occur after the end of the clinical study shall be reported only if they are serious and related 
to the investigational product. 
 
When reporting serious adverse events to the sponsor, the investigator shall use the subject identification code 
instead of the subject’s personal information such as the name, resident registration number, and address in order 
to protect the confidentiality of the subject’s identity. Even if copies of some medical records are requested as 
needed, the personal information, except the identification code, will be redacted. 

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If the investigator becomes aware of any additional information on reported serious adverse events, a follow-up 
report shall be made. The investigator shall conduct follow-ups and submit reports to the sponsor and the IRB 
until the corresponding serious adverse event disappears, shows a stable outcome, or the subject is lost to follow-
up.
10.5.4. 
Reporting Procedures for Suspected Unexpected Serious Adverse Reactions (SUSARs) 
In the case that suspected unexpected serious adverse reactions (SUSARs) occur during the study period, the 
sponsor shall report them to the investigator, the Minister of Food and Drug Safety, and the IRB within the period 
specified in each of the following paragraphs. For details, see “Regulations on the Safety of Drugs, etc.” Appendix 
4 Korean Good Clinical Practice for Drugs and the guide for civil petitioners, “Considerations for Safety 
Evaluation and Reporting of the Sponsor of Clinical Trial for Drugs.” 
 For unexpected adverse drug reactions that result in death or are life-threatening, they shall be reported as 
soon as possible via telephone, fax, and documents within 7 days from the date the sponsor was initially 
reported of the relevant fact or became aware of it. If not all of the information was reported according to the 
adverse drug reaction report, a detailed report shall be additionally submitted within 15 days from the date 
the sponsor was initially reported of the relevant adverse drug reaction or became aware of it.  
 All other SUSARs shall be reported within 15 days from the date the sponsor was reported of the relevant 
fact or became aware of it. 
 
In the case that suspected unexpected serious adverse reactions (SUSARs) occur during the clinical study, the 
principal investigator and study staff members shall make every effort for the safety of the subjects, and take 
prompt and appropriate actions to minimize the adverse events. If additional information on the reported adverse 
drug reaction is available, the sponsor shall be report it until the corresponding adverse drug reaction is ended 
(refers to the disappearance of the corresponding adverse drug reaction or becoming impossible to follow up). 
 
If the clinical study is conducted at multiple study sites, the sponsor shall promptly notify the study sites through 
the principal investigators. 
 
10.6. Pregnancy 
Pregnancy of a female subject during the clinical study is not considered an adverse event, and an elective abortion 
without complications (not applicable for therapeutic abortion) or hospitalization for live birth of a healthy 
newborn is also not considered an adverse event.  
 
However, if a female subject becomes pregnant during the clinical study, the subject shall be dropped out from 
the clinical study, and the investigator shall complete a pregnancy report form and submit it to the clinical study 
sponsor within 24 hours of becoming aware of the pregnancy. In addition, even for cases in which a pregnancy is 
confirmed within 90 days after the end of the clinical study (discontinuation), the investigator shall complete a 
pregnancy report form and submit it to the clinical study sponsor within 24 hours of becoming aware of the 
pregnancy. The investigator shall submit follow-up reports on the subsequent progress of the pregnant woman and 
the fetus until birth even if the subject discontinues participation in the clinical study or completes the clinical 
study.  
 
Serious maternal complications, spontaneous abortions, ectopic pregnancy, stillbirths, neonatal deaths, congenital 
malformations, etc., are considered serious adverse events, and the investigator shall report them according to the 
procedures in section 10.5.3Reporting of Serious Adverse Events. 
10.7. Overdosing 
Overdosing is defined as an accidental or intentional administration of a drug at a dose considered by the 

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investigator to be excessive and medically significant. The investigator will consider overdosing as a significant 
medical event and report it to the sponsor, regardless of the adverse event outcome. 
 

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11. Data Analysis and Statistical Considerations 
11.1. Analysis Set 
The main analysis of efficacy evaluation of this clinical study will be performed on the full analysis set (FAS), 
and the analysis on the per-protocol set (PPS) will be performed as a supplement. The safety evaluation analysis 
will be performed as actually administered in the safety set. The exploratory evaluation analysis will be performed 
on the FAS and PPS, and the demographic and the characteristics prior to administration will be analyzed in all 
randomized subjects. 
 
11.1.1.
Efficacy Evaluation Set 
Full Analysis Set (FAS)
This includes subjects who received at least 1 dose of the investigational product after randomization and had at 
least 1 evaluation with the PSP-rating scale after the administration of the investigational product and up to the 
end of the clinical study. 
Per-Protocol Set (PPS) 
Among the subjects included in FAS, the subjects who completed the clinical study without major protocol 
violations are included. Subjects with major protocol violations (see 5.5.3 Protocol Violations) may be excluded 
from PPS. 
 
Exclusion from PPS will be decided through a blind meeting prior to database lock, after comprehensively 
discussing whether the clinical study was affected. 
 
11.1.2. 
Safety Evaluation Set 
Safety set 
Among the subjects who were administered at least one dose of the investigational product after randomization, 
subjects whose safety-related data were evaluated at least once after the administration will be included. 
11.2. Statistical Analysis Method 
11.2.1. 
General Principles of Analysis 
All statistical analyses will use SAS® (version 9.4 or higher; SAS Institute, Cary, NC, USA). In principle, all tests 
are two-sided tests under a significance level of 5% unless otherwise specified. The efficacy evaluation of this 
clinical study will perform analysis according to the “intention-to-treat principle,” and the safety evaluation will 
perform analysis as actually administered. Continuous data will present the number of subjects observed, mean, 
standard deviation, median, minimum, and maximum, while categorical data will present frequencies and 
percentages by category.  
 
11.2.2. 
Method of Handling Missing Data 
When analyzing the efficacy endpoints and exploratory endpoints, in the case of utilizing the mixed-effect model 
repeated measure (MMRM) method, the available data will be analyzed as they are since the method does not 
require the replacement of missing values. If missing values occur in the safety endpoints and exploratory 
endpoints analyzed by methods other than MMRM, analysis will be performed only on the collected data without 
imputation.  
11.2.3. 
Covariate Adjustment 
Efficacy endpoints will be analyzed with adjustments for the baseline values as well as for the age and sex. 

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11.2.4. 
Interim Analysis 
No interim analysis will be performed before the end of the clinical study. However, an interim analysis related 
to the safety of this clinical study may be conducted when a serious issue related to safety is discovered and the 
need is recognized through a meeting or a deliberation of the investigator or principal investigator. 
11.2.4.1. 
Independent Data Monitoring Committee (IDMC) 
A discussion may be held according to the charter of an independent data monitoring committee (IDMC) 
composed of clinical experts not including the principal investigator, sub-investigators, and sponsor to make 
objective decisions on details related to safety by clarifying the judgment on safety findings obtained during the 
course of this clinical study.  
11.2.4.2. 
Safety Monitoring 
The principal investigator will act as the protocol safety manager based on the risk level of this clinical study, and 
the principal investigator at each study site will monitor the safety of the subjects participating in the relevant 
study site in accordance with the protocol during the clinical study.  
Whenever serious adverse events and adverse drug reactions occur, the principal investigator will collect and 
review the relevant subject’s data and safety information. If adverse events, unexpected problems, and protocol 
noncompliance cases should occur, the principal investigator shall submit reports to the sponsor (including the 
CRO) and the IRB in accordance with the protocol, IRB regulations of each study site, KGCP, and relevant 
regulations, and also submit reports to the Ministry of Food and Drug Safety if necessary. In addition, in 
accordance with the criteria for early termination or discontinuation of the clinical study in section 7.2, reports 
will be submitted to the sponsor in a timely manner if any efficacy and safety information that can significantly 
impact the continuation of the clinical study should emerge, and the sponsor will discuss and decide on the 
continuation of the clinical study according to the sponsor’s standard operating procedure (SOP). 
 
11.2.5. 
Multiple Comparisons and Multiplicity 
Given that this is a Phase 2 clinical study for therapeutic exploratory purposes, no correction of type I error due 
to comparisons with the control group were considered for each of the study groups (GV1001 0.56 mg/day, 1.12 
mg/day).  
11.2.6.
Subgroup Analysis
Subgroup analysis will not be considered in this clinical study.
 
11.2.7. 
Demographic Information and Other Characteristics Prior to Treatment 
The demographic information and other characteristics prior to treatment of the subjects included in this clinical 
study will be summarized by treatment group. Continuous variables will present the number of subjects observed, 
mean, standard deviation, median, minimum, and maximum. In addition, depending on whether the assumption 
of normality is satisfied, the two-sample t-test (if normality assumption is satisfied) or Wilcoxon’s rank-sum test 
(if normality assumption is not satisfied) will be used to compare each study group with the control group 
(GV1001 0.56 mg/day vs. control group, GV1001 1.12 mg/day vs. control group). Categorical variables will 
present frequencies and percentages, and depending on whether cells with an expected frequency less than 5 
exceed 20% of the total cells, Pearson’s chi-square test (if the cells with an expected frequency less than 5 does 
not exceed 20% of the total cells) or Fisher’s exact test (if the cells with an expected frequency less than 5 exceed 
20% of the total cells) will be used to compare each study group with the control group (GV1001 0.56 mg/day vs. 
control group, GV1001 1.12 mg/day vs. control group). 
Medical history will be standardized using the Medical Dictionary for Regulatory Activities (MedDRA), and the 
frequency, percentage, and number of cases will be presented by group for each standardized medical history. 
Prior/concomitant medications will be standardized by the ATC code of the WHO Drug Dictionary, and the 

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frequency and percentage of subjects as well as the number of cases will be presented by group for each 
standardized concomitant medication. 
11.2.8.
Efficacy Analysis
11.2.8.1. 
Primary Efficacy Analysis 
Descriptive statistics (number of subjects observed, mean, standard deviation, median, minimum, and maximum) 
will be presented by treatment group for the change from the baseline in the total score of the PSP-rating scale 
after 24 weeks (6 months) of investigational product administration. To test the difference between each study 
group and the control group (GV1001 0.56 mg/day vs. control group, GV1001 1.12 mg/day vs. control group), an 
MMRM analysis will be conducted with the treatment groups, visits, and interactions between the visits and the 
treatment groups set as fixed effects, and the subjects set as the random effect, as well as considering the baseline 
values, age, and sex as covariates. The results of the MMRM analysis will be summarized by LSM and standard 
error (SE) for each treatment group, the LSM difference between the study groups and the control group (GV1001 
0.56 mg/day vs. control group, GV1001 1.12 mg/day vs. control group) and its 95% confidence interval as well 
as the two-side p-value. 
 
11.2.8.2. 
Secondary Efficacy Analysis 
1) Change from the baseline in the total score of PSP-rating scale after 12 weeks (3 months) of investigational 
product administration 
Descriptive statistics (number of subjects observed, mean, standard deviation, median, minimum, and 
maximum) will be presented by treatment group for the change from the baseline in the total score of the 
PSP-rating scale after 12 weeks (3 months) of investigational product administration. To test the difference 
between each study group and the control group (GV1001 0.56 mg/day vs. control group, GV1001 1.12 
mg/day vs. control group), an MMRM analysis will be conducted with the treatment groups, visits, and 
interactions between the visits and the treatment groups set as fixed effects, and the subjects set as the random 
effect, as well as considering the baseline values, age, and sex as covariates. The results of the MMRM 
analysis will be summarized by LSM and standard error (SE) for each treatment group, the LSM difference 
between treatment groups and its 95% confidence interval, and the two-sided p-value. 
 
2) Change from the baseline in the Montreal Cognitive Assessment - Korea (MoCA-K) after 12 weeks (3 months) 
and 24 weeks (6 months) of investigational product administration 
Descriptive statistics (number of subjects observed, mean, standard deviation, median, minimum, and 
maximum) will be presented by treatment group for the change from the baseline in MoCA-K after 12 weeks 
(3 months) and 24 weeks (6 months) of investigational product administration. To test the difference between 
each study group and the control group (GV1001 0.56 mg/day vs. control group, GV1001 1.12 mg/day vs. 
control group), an MMRM analysis will be conducted with the treatment groups, visits, and interactions 
between the visits and the treatment groups set as fixed effects, and the subjects set as the random effect, as 
well as considering the baseline values, age, and sex as covariates. The results of the MMRM analysis will 
be summarized by LSM and standard error (SE) for each treatment group, the LSM difference between 
treatment groups and its 95% confidence interval, and the two-sided p-value. 
 
3) Change from the baseline in the Korean Frontal Assessment Battery (K-FAB) after 12 weeks (3 months) and 
24 weeks (6 months) of investigational product administration 
Descriptive statistics (number of subjects observed, mean, standard deviation, median, minimum, and 
maximum) will be presented by treatment group for the change from the baseline in K-FAB after 12 weeks 
(3 months) and 24 weeks (6 months) of investigational product administration. To test the difference between 
each study group and the control group (GV1001 0.56 mg/day vs. control group, GV1001 1.12 mg/day vs. 
control group), an MMRM analysis will be conducted with the treatment groups, visits, and interactions 
between the visits and the treatment groups set as fixed effects, and the subjects set as the random effect, as 
well as considering the baseline values, age, and sex as covariates. The results of the MMRM analysis will 

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be summarized by LSM and standard error (SE) for each treatment group, the LSM difference between 
treatment groups and its 95% confidence interval, and the two-sided p-value. 
 
4) Change from the baseline in the England & Schwab Activity of Daily Living (ES ADL) scale after 12 weeks 
(3 months) and 24 weeks (6 months) of investigational product administration 
Descriptive statistics (number of subjects observed, mean, standard deviation, median, minimum, and 
maximum) will be presented by treatment group for the change from the baseline in ES ADL after 12 weeks 
(3 months) and 24 weeks (6 months) of investigational product administration. To test the difference between 
each study group and the control group (GV1001 0.56 mg/day vs. control group, GV1001 1.12 mg/day vs. 
control group), an MMRM analysis will be conducted with the treatment groups, visits, and interactions 
between the visits and the treatment groups set as fixed effects, and the subjects set as the random effect, as 
well as considering the baseline values, age, and sex as covariates. The results of the MMRM analysis will 
be summarized by LSM and standard error (SE) for each treatment group, the LSM difference between 
treatment groups and its 95% confidence interval, and the two-sided p-value. 
 
5) Change from the baseline in the score of each domain of the PSP-rating scale after 12 weeks (3 months) and 
24 weeks (6 months) of investigational product administration 
Descriptive statistics (number of subjects observed, mean, standard deviation, median, minimum, and 
maximum) will be presented by treatment group for the change from the baseline in the score of each domain 
of the PSP-rating scale after 12 weeks (3 months) and 24 weeks (6 months) of investigational product 
administration. To test the difference between each study group and the control group (GV1001 0.56 mg/day 
vs. control group, GV1001 1.12 mg/day vs. control group), an MMRM analysis will be conducted with the 
treatment groups, visits, and interactions between the visits and the treatment groups set as fixed effects, and 
the subjects set as the random effect, as well as considering the baseline values, age, and sex as covariates. 
The results of the MMRM analysis will be summarized by LSM and standard error (SE) for each treatment 
group, the LSM difference between treatment groups and its 95% confidence interval, and the two-sided p-
value. 
 
6) Change from the baseline in the score of each item of the PSP-rating scale after 12 weeks (3 months) and 24 
weeks (6 months) of investigational product administration 
Descriptive statistics (number of subjects observed, mean, standard deviation, median, minimum, and 
maximum) will be presented by treatment group for the change from the baseline in the score of each item of 
the PSP-rating scale after 12 weeks (3 months) and 24 weeks (6 months) of investigational product 
administration. To test the difference between each study group and the control group (GV1001 0.56 mg/day 
vs. control group, GV1001 1.12 mg/day vs. control group), an MMRM analysis will be conducted with the 
treatment groups, visits, and interactions between the visits and the treatment groups set as fixed effects, and 
the subjects set as the random effect, as well as considering the baseline values, age, and sex as covariates. 
The results of the MMRM analysis will be summarized by LSM and standard error (SE) for each treatment 
group, the LSM difference between treatment groups and its 95% confidence interval, and the two-sided p-
value. 
 
11.2.9. 
Exploratory Evaluation Analysis 
1) Change from the baseline in total tau (t-tau), phosphorylated tau (p-tau), and neurofilament light chain (NfL) 
markers in blood after 12 weeks (3 months), and in blood and cerebrospinal fluid after 24 weeks (6 months) 
of investigational product administration  
2) Change from the baseline in inflammatory cytokine markers (IL-6, IL-8, IL-2, IFN- 
TNF-
etc.) in blood 
after 12 weeks (3 months), and in blood and cerebrospinal fluid after 24 weeks (6 months) of investigational 
product administration  
3) Change from the baseline in blood after 12 weeks (3 months), and in blood and cerebrospinal fluid markers 
after 24 weeks (6 months) of investigational product administration, and correlation between clinical markers 
and treatment responses 

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Appropriate statistical analyses, such as MMRM and correlation analysis, will be performed for exploratory 
endpoints after investigational product administration, compared with the baseline according to data 
characteristics such as continuous data and categorical data. 
 
11.2.10. Safety Analysis 
11.2.10.1. 
Adverse Events 
The analysis of adverse events will be based on treatment-emergent adverse events (TEAEs). The number of 
subjects with onset by treatment group, the incidence, and the 95% confidence interval of the incidence will be 
presented for adverse events, adverse drug reactions, serious adverse events, and serious adverse drug reactions. 
In addition, to test the difference in the incidence of adverse events by treatment group, comparisons between 
groups (GV1001 0.56 mg/day vs. control group, GV1001 1.12 mg/day vs. control group) will be performed with 
Pearson’s chi-square test (if the cells with an expected frequency less than 5 does not exceed 20% of the total cells) 
or Fisher’s exact test (if the cells with an expected frequency less than 5 exceed 20% of the total cells), depending 
on whether cells with an expected frequency less than 5 exceed 20% of the total cells Adverse events, adverse 
drug reactions, serious adverse events, and serious adverse drug reactions will be coded according to the system 
organ class (SOC) and preferred term (PT) by using MedDRA, and the number of subjects with onset, the 
incidence, and the number of occurrences of the coded adverse events will be presented by treatment group. In 
addition, the status of adverse event occurrences according to their severity and causal relationship to the 
investigational product will be presented. 
 
11.2.10.2. 
Laboratory Tests 
For the laboratory test results, continuous variables will present the test values at each visit as well as descriptive 
statistics (number of subjects observed, mean, standard deviation, median, minimum, and maximum) for changes 
from the baseline by treatment group. In addition, depending on whether the assumption of normality is satisfied, 
the intragroup comparison will use the paired t-test or Wilcoxon’s signed-rank test, while the intergroup 
comparison (GV1001 0.56 mg/day vs. control group, GV1001 1.12 mg/day vs. control group) will use the two-
sample t-test or Wilcoxon’s rank-sum test to perform analysis. For categorical variables, shift tables will be 
presented by treatment group, and the intragroup comparison will use McNemar’s test, while the intergroup 
comparison (GV1001 0.56 mg/day vs. control group, GV1001 1.12 mg/day vs. control group) will use Pearson’s 
chi-square test or Fisher’s exact test to perform analysis. 
 
11.2.10.3. 
Vital Signs 
For vital signs, the measurement values at each visit as well as descriptive statistics (number of subjects observed, 
mean, standard deviation, median, minimum, and maximum) for the changes from the baseline will be presented 
by treatment group. In addition, to test the intragroup difference before and after investigational product 
administration, the paired t-test (if normality assumption is satisfied) or Wilcoxon’s signed-rank test (if normality 
assumption is not satisfied) will be performed depending on whether the assumption of normality is satisfied. To 
test the difference between the study groups and the control group (GV1001 0.56 mg/day vs. control group, 
GV1001 1.12 mg/day vs. control group) for the changes from the baseline, the two-sample t-test (if normality 
assumption is satisfied) or Wilcoxon’s rank-sum test (if normality assumption is not satisfied) will be performed 
depending on whether the assumption of normality is satisfied. 
 
11.2.10.4. 
Electrocardiogram 
For electrocardiography, the frequency and percentage by treatment group will be presented using the shift table 
for the changes in “normal or clinically insignificant abnormal” / “clinically significant abnormal” before and 
after administration. In addition, the intragroup comparison will use McNemar’s test, while the intergroup 
comparison (GV1001 0.56 mg/day vs. control group, GV1001 1.12 mg/day vs. control group) will use Pearson’s 
chi-square test or Fisher’s test to perform analysis. 

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11.2.11. Handling Data of Dropout Subjects 
The handling of data of dropout subjects out will be performed according to 11.2.2 Method of Handling Missing 
Data. 
 
11.3. Analysis Period 
No interim analysis will be performed in this clinical study, and the final analysis will be performed to prepare 
the clinical study report after the final visits of all subjects are completed. 
11.4. Rationale for Setting Number of Subjects 
This is a Phase 2a exploratory clinical study conducted for the purposes of collecting initial safety and efficacy 
information on the investigational product GV1001 in patients with progressive supranuclear palsy as well as 
providing the basis for design, evaluation items, and evaluation methods for subsequent clinical studies. Since this 
is a study to estimate the clinically significant effect size to demonstrate the efficacy of this investigational product, 
the number of subjects was not calculated based on statistical considerations. The minimum number of subjects 
required in a pilot study is 12 subjects21) and approximately 30 subjects can be considered according to the rule of 
thumb22), 23). Therefore, by combining these, it is intended to set 20 subjects per group, and a total of 75 subjects 
with 25 subjects per group will be enrolled considering a dropout rate of 20%. 
 
 

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12. Data Management 
12.1. Data Collection/Access 
Source documents refer to the patient records of the physician in charge, which are kept at the study site. Most 
source documents are charts of the hospital or physician in charge, and in these cases, all information collected 
and recorded in the patient’s case report form must be consistent with the relevant charts. This clinical study will 
collect data by using electronic case report forms, and only authorized individuals will be allowed to access and 
correct the data. The investigator shall ensure that the sponsor, the clinical research associate (hereinafter referred 
to as the “monitor”), the auditor, or the inspector has direct access to relevant data such as basic clinical study 
documents including electronic documents. 
 
12.2. Data Protection/Storage 
The investigator shall store various data and records related to the conduct of the clinical study in a secure location 
and maintain their security, and shall retain them for 3 years from the product approval date of the investigational 
product. After completion of the clinical study report, the documents related to the clinical study shall be 
transferred to the person in charge of storage, and the sponsor shall be informed in advance if the investigator 
intends to destroy the records related to the clinical study or move them to another location. However, the storage 
period may be extended if instructed by the Minister of Food and Drug Safety or if deemed necessary by the 
sponsor. The sponsor shall inform the investigator and the director of the study site regarding the need for data 
storage as well as the data storage duration in writing, and if the sponsor determines that the data no longer need 
to be stored, the sponsor shall inform the principal investigator and the director of the study site of this fact in 
writing. 
 

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13. Ethical Considerations and Administrative Procedures 
13.1. Relevant Laws and Regulations 
This clinical study will be conducted in accordance with all international and domestic laws and regulations, 
including the Declaration of Helsinki (1964) and the Korean Good Clinical Practice (KGCP), approval of the 
clinical study plan by the Ministry of Food and Drug Safety, approval from the IRB of the principal investigator’s 
affiliated study site, and the standard operating procedures (SOPs) of the contract research organization, CMIC 
Korea Co., Ltd. 
13.2. Subject’s Consent
A written informed consent form shall be obtained from each subject prior to participation in the clinical study or 
prior to performing certain unconventional procedures that pose risks to the subject. The subject’s informed 
consent form will be submitted to the relevant IRB by the principal investigator for review and approval prior to 
the start of the study. 
Prior to the recruitment and enrollment of subjects, the clinical study details as well as the effects and adverse 
events of the investigational product and all information on safety will be described to the subject, and then the 
clinical study will be initiated after obtaining the subject’s informed consent form which confirms that the subject 
will voluntarily participate in this clinical study. 
If the subject or the legally authorized representative is unable to read the informed consent form, subject 
information sheet, and other documented information, an impartial witness will attend the entire process of 
obtaining consent. In this case, after reading and explaining all documented information to the subject or legally 
authorized representative, the subject or legally authorized representative shall be allowed to provide verbal 
consent for the subject’s participation in the clinical study, and if possible, the subject shall be allowed to sign and 
date the informed consent form in his/her own handwriting. In addition, prior to the impartial witness signing the 
informed consent form, he/she shall also confirm that the documented information was accurately explained to 
the subject or legally authorized representative, and that the subject or legally authorized representative 
understood the relevant facts and that process of obtaining consent was conducted according to their own free will. 
The original informed consent form will be signed by the patient and the principal investigator (or his/her 
delegatee), and the principal investigator or the investigator’s delegatee will provide a copy of the original signed 
informed consent form to the subject. The original informed consent form will be retained by the investigator at 
the study site. 
 
13.3. Indemnification Policy for Injured Subjects 
In the case that an adverse event is caused by the investigational product or an injury occurs in the course of a 
corrective treatment of an adverse event, the principal investigator will indemnify the subject in accordance with 
the subject indemnification policy for the injury directly caused by the investigational product. 
 
13.4. Criteria for Medical Care and Treatment of Subjects after Completion of Study 
Participation or Dropping Out 
The investigator shall ensure that other appropriate treatments can be received by the subjects who have been 
dropped out from the clinical study or who do not show response, and shall ensure that subjects who have 
completed the clinical study also receive appropriate alternative treatments if it is determined that continuous 
treatment is required. 
 
13.5. Ethics Compliance and Protection Measures for Subject Safety 
This clinical study will be conducted scientifically and ethically in accordance with the Korean Good Clinical 
Practice and the laws and regulations relevant to clinical studies. In addition, this clinical study will be conducted 
in accordance with the Declaration of Helsinki to respect the dignity, rights, and interests of human beings, and to 
avoid placing disadvantages on the subjects. 

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• 
Study site 
The director of the study site shall ensure that clinical laboratories, facilities, and specialized personnel necessary 
to conduct the clinical study are in place, and shall ensure that the clinical study can be properly conducted by 
allowing necessary actions to be taken in the case of an emergency.  
 
• 
Institutional Review Board (IRB) 
The IRB shall be organized according to domestic regulations/practices, and shall protect the rights, safety, and 
well-being of the subjects, and carefully review the validity of the reason in the case that vulnerable subjects 
participate in the clinical study. In performing its duties, the IRB shall take necessary actions such as issuing an 
order of discontinuation to the principal investigator for a part or for the entire clinical study if the subjects’ 
consent for participation is not appropriately obtained, or the clinical study is not conducted in accordance with 
the protocol, or if serious adverse events and adverse drug reactions are shown. The IRB shall evaluate/approve 
this protocol in accordance with the Korean Good Clinical Practice, and shall regularly evaluate whether the 
clinical study is conducted in accordance with the protocol. 
 
• 
Investigator 
The word “investigator” refers to the principal investigator, sub-investigator, and coordinating investigator. Prior 
to enrolling subjects in the clinical study, the investigator shall check the health status of each subject to confirm 
whether he/she can participate in the clinical study. In addition, the investigator shall be sufficiently familiar with 
the study drug and make every effort to ensure the safety of the subjects. If medical treatment is needed for the 
subject’s intercurrent disease which the investigator has become aware of, this shall be informed to the subject. 
The investigator shall accurately analyze and comprehend the clinical study plan, and actively respond to the 
issues of the subject. 
 
• 
Sponsor 
This refers to the individual, company, institution, and organization with responsibilities related to the planning, 
management, finances, etc., of the clinical study. It shall ensure that the subject of the clinical study, test methods, 
and case report forms and their details are managed in accordance with the procedures in the clinical study protocol. 
The sponsor’s audit plan and procedures shall be determined based on the importance of the clinical study, the 
number of subjects, the type and complexity of the clinical study, the level of potential risks to the subjects, and 
the issues in conducting the clinical study that have already been identified.  
 
The subjects’ information and medical records obtained in this clinical study will be shared only with the principal 
investigator, the investigator’s delegatee, and CRO, and may only be provided upon request by the jurisdictional 
departments such as the IRB and the Ministry of Food and Drug Safety. 
In the case that an adverse event due to the clinical study has occurred, appropriate medical measures will be taken 
until the subject recovers, and the subject will be indemnified for the injury caused by the investigational product 
in accordance with the indemnification policy for injured subjects. 
 
13.5.1. Approval and Amendment of Clinical Study Plan 
The clinical study plan shall be submitted to the Ministry of Food and Drug Safety in accordance with Article 24 
Paragraph 1 of the “Regulations on the Safety of Drugs, etc.,” and the decision of the Ministry of Food and Drug 
Safety and the IRB regarding the conduct of the clinical study will be delivered to the investigator and the sponsor 
in writing before the start of the clinical study. The protocol, informed consent form, advertisements used for 
subject recruitment, and other written information provided to the subject (or the subject’s legally authorized 
representative) regarding this study shall be approved by the Ministry of Food and Drug Safety and/or the IRB, 
and the documents shall be retained by the investigator and the sponsor. 
The principal investigator or the investigator’s delegate is responsible for undergoing a continuation review of the 
clinical study at intervals within 1 year or as otherwise specified by the IRB. 

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The principal investigator and the sponsor shall submit reports to the Ministry of Food and Drug Safety and/or 
the IRB on the progress of the clinical study, serious adverse events, life-threatening issues or deaths, and inform 
the Ministry of Food and Drug Safety and the IRB of this at the end of the clinical study. 
Any amendments to the protocol shall be made through a consultation between the investigator and the sponsor, 
and any changes to the protocol shall be approved by the Ministry of Food and Drug Safety and the IRB prior to 
implementation. However, if the details of the protocol that do not significantly affect the safety of the subjects or 
the reliability of the study results are to be amended in accordance with Article 10 Paragraph 2 of the “Regulations 
on Approval for Investigational New Drug Application,” it may be done after obtaining an approval from the IRB 
without a separate approval from the Ministry of Food and Drug Safety. Administrative changes do not generally 
require an IRB approval, but the IRB shall be informed of the relevant information. 
Changes related to the safety of the subjects may be implemented without the approval of the Ministry of Food 
and Drug Safety and the IRB if a subject’s sudden injury needs to be eliminated, and in such cases, the investigator 
shall submit reports to the Ministry of Food and Drug Safety and the IRB within a specified period after treatment 
and obtain approval. 
 
13.5.2. Protocol Violations 
The principal investigator or sub-investigator may violate the protocol or implement changes without a prior IRB 
approval in order to eliminate immediate risks posed to the subjects. After such occurrences, the committed 
violations or implemented changes, their reasons, and all proposed amendments to the protocol shall be submitted 
to the IRB for review and approval as soon as possible, and if necessary, they shall be also submitted to the 
regulatory authorities. 
The principal investigator will report to the IRB any major protocol violations and violations deemed to be 
significant that affect the safety and integrity of this study. 
13.6. Disclosure of Results 
The sponsor shall prepare a report and inform the investigator of the results of the clinical study once the data 
from all study sites have been fully analyzed.  
All data and results obtained from this clinical study are the property of the sponsor, and the sponsor has the right 
to publish the results of this clinical study at any time. The investigator shall not publish, present, or disclose any 
information related to the results of this study without a prior written consent of the sponsor, and shall also ensure 
that the sub-investigator complies with these conditions. To ensure that only accurate and validated data are used, 
the investigator shall provide the sponsor all draft publications or presentation manuscripts that were made prior 
to publication or presentation to discuss the materials with the sponsor, and the presentation shall be put on hold 
until a written approval is obtained.  
 
For multicenter clinical studies, the investigator shall agree to not present the results of his/her affiliated study site 
or of some study sites prior to the presentation of the results collected from all of the study sites. However, 
exceptions are made when it is officially recognized by principal investigators of all study sites and the sponsor. 
 
13.7. Confidentiality of Patient Records 
All clinical study results and documents will be considered confidential. The investigator, the contract research 
organization, and the clinical research associate of the clinical study sponsor shall not disclose any information 
related to the clinical study without a signed approval of the clinical study sponsor. Records that can identify the 
subjects will be kept confidential, and all documents related to the clinical study, such as case report forms, will 
be recorded and classified with identification codes rather than the names of the subjects. Even when the results 
of the clinical study are published, the identities of the subjects shall be kept confidential. 

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13.8. Quality Control and Reliability Assurance 
13.8.1. Monitoring of Clinical Study 
Monitoring will be performed to protect the rights and well-being of the subjects, to confirm the accuracy, integrity, 
and verifiability of data through comparisons of reported clinical study data with source documents, and to confirm 
that the clinical study is being conducted in accordance with the approved protocol and Article 30 of the 
“Regulations on the Safety of Drugs, etc.” and Appendix 4. Korean Good Clinical Practice. 
A monitor designated by the sponsor will conduct monitoring of the clinical study, evaluate the progress of the 
clinical study, and check whether the investigator is fulfilling his/her obligations according to the protocol and 
regulations through regular visits to the study site and telephone calls. At visits to the study site, the monitor will 
check the original subject records, case report forms, drug management records, and storage of clinical study-
related data. If any discrepancies or issues are found in the clinical study records, they will be discussed with the 
investigator. 
 
13.8.2. Audit and Inspection
To ensure the safety of the subjects in this clinical study and to obtain accurate, complete, and reliable data, the 
investigator shall keep the clinical test results, clinical records, and the subjects’ medical records as source 
documents. The principal investigators or sub-investigators and study sites participating in this study shall permit 
study-related monitoring, audits, IRB reviews, and inspections by regulatory authorities. The investigator shall 
allow direct access to the clinical study-related data upon request of the Ministry of Food and Drug Safety and the 
Institutional Review Board. 
 
13.9. Other Matters Necessary to Conduct Clinical Study Safely and Scientifically 
Through a meeting of investigators prior to the start of the clinical study, violations in the clinical study will be 
minimized by reviewing relevant documents such as the protocol and gathering the opinions of the investigators. 
In addition, an interim investigator meeting may be held if the sponsor or investigators determine that it is 
necessary to control the study sites and resolve violations, etc.  
 
13.9.1. Matters Necessary to Initiate Clinical Study 
The following are required prior to delivering the investigational product to the study site and prior to initiating 
the clinical study. 
 
1) 
A copy of the approved protocol, and (if necessary) a copy of the approved protocol amendment 
2) 
Career history of the principal investigator and sub-investigator 
3) 
A written notification of approval of the clinical study plan by the Ministry of Food and Drug Safety and 
IRB 
4) 
Copies of the subject information sheet and informed consent form 
5) 
Normal ranges for clinical laboratory results 
6) 
Other documents required by the government 
Subjects shall not be enrolled prior to a formal meeting between the investigator and the sponsor for initiating the 
clinical study, and the meeting shall include conducting reviews on the protocol and case report forms. 
13.9.2. Investigator’s Obligations 
The investigator is obligated to conduct the clinical study in accordance with the protocol, and has the 
responsibility of obtaining written informed consent from the subjects, or if necessary, the subjects’ legally 
authorized representatives and guardians prior to performing any processes in accordance with the protocol. 
 
 

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14. References 
1)
Bluett B, Litvan I. Pathophysiology, genetics, clinical features, diagnosis and therapeutic trials in progressive 
supranuclear palsy. Exp Opin Orphan Drugs. 2015;3(3):253–265. 
2) Coyle-Gilchrist IT, Dick KM, Patterson K, et al. Prevalence, characteristics, and survival of frontotemporal 
lobar degeneration syndromes. Neurology. 2016;86(18):1736-1743.
3)
Schrag A, Ben-Shlomo Y, Quinn NP. Prevalence of progressive supranuclear palsy and multiple system 
atrophy: a cross-sectional study. Lancet. 1999;354(9192):1771-1775.
4) Höglinger GU, Respondek G, Stamelou M, et al. Clinical diagnosis of progressive supranuclear palsy: The 
movement disorder society criteria. Mov Disord. 2017;32(6):853-864.
5) McFarland NR. Diagnostic Approach to Atypical Parkinsonian Syndromes. Continuum (Minneap Minn). 
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GemVax & KAEL Co., Ltd.
 
Page 77 of 78 
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[Protocol Approval Letter] 
Title: 
 
GV1001 0.56 
 1.12 mg/day 
 
 
 
 
 
 
, 
, 
 
, 
 
, 
 
, 
, 
2a
 
 
A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel design, Prospective, Phase 
IIa Exploratory Clinical Trial to evaluate the efficacy and safety of Subcutaneous Administration of 
GV1001 0.56 or 1.12 mg/day in Patients with Progressive Supranuclear Palsy 
 
Protocol No.:
GV1001-PSP-CL2-011
Version (Date):
5.1 (15 Mar 2024) 
I have read and reviewed this protocol, and I understand and agree that this protocol contains all the information 
necessary to conduct the clinical study. I will make reasonable efforts to complete this clinical study within the 
planned schedule. I will conduct this clinical study in accordance with the Declaration of Helsinki, ICH Good 
Clinical Practice (GCP), and all applicable regulations of relevant countries. 
Sponsor 
Printed Name 
 
Signature 
 
(DD/MMM/YYYY)
Date
 
 

Protocol No. GV1001-PSP-CL2-011
Protocol Version 5.1 (15 Mar 2024)
GemVax & KAEL Co., Ltd.
 
Page 78 of 78 
- Confidential - 
[Protocol Approval Letter] 
Title: 
 
GV1001 0.56 
 1.12 mg/day 
 
 
 
 
 
 
, 
, 
 
, 
 
, 
 
, 
, 
2a
 
 
A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel design, Prospective, Phase 
IIa Exploratory Clinical Trial to evaluate the efficacy and safety of Subcutaneous Administration of 
GV1001 0.56 or 1.12 mg/day in Patients with Progressive Supranuclear Palsy 
 
Protocol No.:
GV1001-PSP-CL2-011
Version (Date):
5.1 (15 Mar 2024)
I have read and reviewed this protocol, and I understand and agree that this protocol contains all the information 
necessary to conduct the clinical study. I will make reasonable efforts to complete this clinical study within the 
planned schedule. I will conduct this clinical study in accordance with the Declaration of Helsinki, ICH Good 
Clinical Practice (GCP), and all applicable regulations of relevant countries. 
Principal Investigator 
Printed Name 
 
Signature 
 
(DD/MMM/YYYY)
Date