A Single/Multiple Ascending Dose Phase 1 Study of the Safety, Tolerability and Pharmacologic Activity of BT200 in Normal Human Volunteers
Phase: PHASE1
Conditions: Cerebrovascular Stroke, Large-Artery Atherosclerosis (Embolus/Thrombosis), Intracranial Arteriosclerosis
Sponsor: Band Therapeutics

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 
1.5.1 
22-APR--2020 
Proprietary & Confidential Information 
pg. 1 
 
 
BT200-01 
A SINGLE/MULTIPLE ASCENDING DOSE PHASE 1 STUDY OF THE SAFETY, TOLERABILITY, 
AND PHARMACOLOGIC ACTIVITY OF BT200 IN NORMAL HUMAN VOLUNTEERS 
Protocol identification number:   
BT200-01 
EudraCT No.:   
2019-001818-42 
Protocol version:   
FINAL VERSION 1.5.1, DATE 22-APR-2020 
Test product:   
BT200 
 
Principal Investigator: 
Ao. Univ.-Prof. Dr. Ulla Derhaschnig 
 
Sponsor: 
Band Therapeutics, LLC 
 
 
 
 
 
Confidentiality 
The contents of the protocol are confidential and may not be communicated verbally or in writing without 
permission from the study sponsor. 

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 
1.5.1 
22-APR--2020 
Proprietary & Confidential Information 
pg. 2 
CONTACT ADDRESSES 
 
Sponsor 
Band Therapeutics, LLC 
101 Hartwell Avenue, Suite 2 
Lexington, MA 02421 USA 
Phone: + 1.781.918.6580 
Email: jim.gilbert@bandtherapeutics.com 
Sponsor’s Medical 
Monitor 
James C. Gilbert, MD 
Chief Executive Officer 
Band Therapeutics, LLC 
Phone (mobile): +1.617.281.3550 
Email: jim.gilbert@bandtherapeutics.com 
Principal Investigator: 
Ao. Univ.-Prof. Dr. Ulla Derhaschnig 
Medical University of Vienna 
Department of Clinical Pharmacology 
Waehringer Guertel 18-20 
1090 Vienna, Austria 
Phone: +43 1 40400 29810 
Fax: +43 140400 29980 
Email: ulla.derhaschnig@meduniwien.ac.at 
Data Monitoring 
Committee 
Univ.-Prof. Dr.med. univ. Dr. Heinz Burgmann, Chairman 
Department of Internal Medicine I, Div. of Infectious Diseases 
Währinger Gürtel 18-20 
1090 Vienna, Austria 
Phone: +43 1 40400 44400 
Project Management 
Data Monitoring 
Celerion 
Hainburger Strasse 33 
1030 Vienna| Austria 
Phone: +43 1 403 38 05 – 54 
Fax: +43 1 403 38 05 -66  
www.celerion.com 
Data Management 
Statistics 
Assign Data Management and Biostatistics GmbH 
Stadlweg 23 
6020 Innsbruck 
Austria 
Ph: +43 512 890 064 121 
Fax: +43 512 281 514 
BT200 Bioanalysis 
QPS 
3 Innovation Way #240 
Newark, DE 19711 USA  
Phone:  +1. 302 369-5601 
Pharmacokinetic and 
Pharmacodynamic 
Analysis 
Certara 
2000 Peel Street, Suite 570 
Montréal, Québec 
H3A 2W5 Canada 

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 
1.5.1 
22-APR--2020 
Proprietary & Confidential Information 
pg. 3 
Tel: +1 514 789 2180 
Immunogenicity (Anti-
Drug Antibody) 
Analysis 
STC Biologics 
330 Nevada Street, Newton, MA 02460, USA 
Phone: +1 617 916-9659 
Drug Supply 
ABF Pharmaceutical Services GmbH 
Brunner Strasse 63/18-19 
1230 Vienna Austria 

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 
1.5.1 
22-APR--2020 
Proprietary & Confidential Information 
pg. 4 
Summary of Changes in Version 1.1 Relative to Version 1.0 
• 
Reference to contraceptives in the description of permitted concomitant medications was 
deleted as Not Applicable given that the inclusion of women volunteers has been restricted to 
those who are post-menopausal or status post hysterectomy. 
• 
Cohorts 8-10 of Part A will not be initiated before a Substantial Amendment summarizing the 
results of Part A Cohorts 1-7 has been submitted to EC and AGES and authorized. 
• 
Information supporting the IV route of administration was added to Section 3.1.1, Justification 
for Dose Regimen. 
 
Summary of Changes in Version 1.2 Relative to Version 1.1 
• 
Contact Information was updated with the correct new phone number for Band Therapeutics’ 
company offices. 
• 
The address for ABF Pharmaceutical Services GmbH was updated to correspond to the 
location of the company’s new facility where BT200 packaging and labeling is now being 
performed. 
• 
ABO blood group typing was added to the Screening Visit tests as an extra precaution so that 
in case a serious bleeding event should occur requiring urgent blood transfusion this 
information would already be available. 
• 
ISTH Bleeding Score was added as a specific and standardized safety assessment in case of 
the occurrence of "clinically evident bleeding". 
• 
A discrepancy between the Schedule of Events and the Lab Sampling table in Appendix 4 with 
respect to the timing of samples for anti-drug antibody testing in Part D was corrected. 
• 
An error in the Study Diagram for Part A was corrected, now placing the DSMB review after 
Cohort 7 and before Cohort 8, as per agreement with AGES. 
 
Summary of Changes in Version 1.3 Relative to Version 1.2 
Emerging PK and PD data from Part A suggest that the SC bioavailability of BT200 in humans may be 
lower than predicted.  To address this concern the following modifications of the Phase 1 plan have 
been introduced; 
• 
In Part A (the Single Ascending Dose part of the study) the mode of study drug administration 
was changed from rapid SC injection to gradual SC infusion. 
• 
In Part A two additional dose cohorts were added in order that: 
o 
the 24 mg dose could be repeated (in a new cohort of subjects) using the technique 
of gradual SC infusion; 
o 
the highest dose to be increased to 48 mg dose cohort (i.e., exceeding the previous 
top dose of 36 mg, which will be retained in the new version of the protocol). 
• 
Part D (the IV crossover part of the study) was moved ahead of Part B (the Multiple 
Ascending Dose part of the study) in the order of clinical operations so that a definitive read-
out of relative bioavailability of BT200 (SC versus IV) will be obtained prior to conduct of Part 
B in case modification of the dosing scheme for Part B should be required. 
• 
Part C (the desmopressin challenge part of the study) was reduced from two to one cohort of 
subjects. 
• 
A second DSMB review and Substantial Amendment based upon the results of the expanded 
Part A together with those of Part C and Part D will be performed prior to initiation of Part B. 
In addition, clarification of the timing of post-dose immunogenicity samples in Parts A, B, C, and D 
was added to the corresponding tables of Study Procedures. 
 
 Summary of Changes in Version 1.4 Relative to Version 1.3 
 
Emerging PK and PD data from the additional Part A cohorts introduced in the preceding amendment 
show a favorable trend toward increased SC bioavailability of BT200 after SC infusion relative to SC 
small volume bolus injection, but suggest that the dose needs to be increased further to achieve the 
therapeutic target concentration of BT200.  In order to accomplish this two new Part A cohorts have 
been added, both with BT200 administration by the SC infusion technique. 
• 
BT200 72 mg SC injection or infusion; N=8; 6:2 active:placebo randomization 
• 
BT200 96 mg SC injection or infusion; N=8; 6:2 active:placebo randomization 

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 
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22-APR--2020 
Proprietary & Confidential Information 
pg. 5 
The visit schedule and planned study assessments will remain the same as for all previous Part A 
cohorts DSMB review of safety after Part A cohorts 8-10 and Part D (the IV crossover cohort) will be 
completed prior to initiation of these two new Part A cohorts. 
 
In order to optimize the route, dose, and timing of BT200 administration for Part C according to the 
emerging PK data from Part A, study drug administration in Part C, is being changed as follows: 
• 
BT200 administration will now be done only by SC infusion; 
• 
The dose of BT200 will now be 48 mg, not 24 mg; 
• 
BT200 will now be administered 96 hours prior to desmopressin, not 24 hours.  
 
Summary of Changes in Version 1.5.1 Relative to Version 1.4 
Substantial Changes: 
After reviewing the safety and pharmacodynamic results of Part A Cohorts 8-10 and Part D Cohort 11 
the DSMB recommended not to escalate the dose of BT200 any further in healthy volunteers than the 
48 mg dose that had been administered in Part A Cohort 10.  Therefore, the following modifications to 
the study will be made via this amendment. 
 
• 
Part A Cohorts 16 (72 mg) and 17 (96 mg) will not be implemented as planned in Version 1.4 
of this study protocol.  Part A will now be closed. 
• 
With no further dose escalation there is no longer the need for another interim DSMB safety 
review before the initiation of Part B, the Multiple Ascending Dose component of this study. 
• 
The dose regimens for Part B can now be finalized based upon the safety, pharmacokinetics, 
and pharmacodynamics observed in the completed Part A. 
• 
Part B Cohort 13:  BT200 12 mg IV plus 12 mg SC on Day 0, then 12 mg SC on Days 7, 14, 
21, and 28 
• 
Part B Cohort 14:  BT200 24 mg IV plus 24 mg SC on Day 0, then 24 mg SC on Days 7, 14, 
21, and 28 
• 
Part B Cohort 15, the high dose cohort previously planned, will be eliminated. 
 
Part C, the desmopressin challenge will still be performed as planned in Version 1.4 of this study 
protocol (with a single dose of 48 mg of BT200). 
 
Miscellaneous Changes: 
• 
One additional VWF-related biomarker, VWF propeptide, will be added to the laboratory 
analysis program for Part A, in order to clarify the mechanism underlying the observed, 
unexpected rise in VWF antigen after BT200 treatment.  No additional blood sampling will be 
required as this assay can be performed with the unused PK back-up samples. 
• 
A typographical error in the heading of Table 3 Study Procedures Part B was corrected. 
• 
Screening for coronavirus by nasal swab testing was added to the Screening Visit procedures 
for Part B and Part C. 
 

Band Therapeutics, LLC 
1.5.1 
Approval of the Protocol 
Clinical Study Protocol BT200-01 FINAL VERSION 
':4 SINGLE/MULTIPLE ASCENDING DOSE PHASE 1 STUDY OF THE SAFETY, TOLERABILITY, AND 
PHARMACOLOGIC ACTIVITY OF BT2OO IN NORMAL HUMAN VOLUNTEERS* 
FINAL VERSION 1.5.1, DATE 22-APR-2O2O 
Representative of the Sponsor: Dr. James Gilbert, Chief Executive Officer, Band Therapeutics, LLC 
Date (DD Month YYYY) 
Principal Investigator: Ao. Univ.-Prof. Dr. Ulla Derhaschnig 
Signature 
Date (DD Month YYYY) 

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 
1.5.1 
22-APR--2020 
Proprietary & Confidential Information 
pg. 8 
PROTOCOL SYNOPSIS 
Title 
A SINGLE/MULTIPLE ASCENDING DOSE PHASE 1 STUDY OF THE 
SAFETY, TOLERABILITY, AND PHARMACOLOGIC ACTIVITY OF 
BT200 IN NORMAL HUMAN VOLUNTEERS  
Protocol Number 
BT200-01 
Version 
DRAFT VERSION 1.5.1, Date 22-APR-2020 
EUDRACT Number 
2019-001818-42 
Test Product 
BT200 
Phase 
Phase 1 
Indication 
Stroke prevention in high-risk patients 
Sponsor 
Band Therapeutics, LLC 
101 Hartwell Avenue, Suite 2 
Lexington, MA 02421 USA 
Phone: + 1.781.918.6580 
Email: jim.gilbert@bandtherapeutics.com 
Sponsor’s Medical 
Monitor 
James C. Gilbert, MD 
Chief Executive Officer 
Band Therapeutics, LLC 
Phone: + 1.781.918.6580 
Email: jim.gilbert@bandtherapeutics.com 
Principal Investigator 
Ao. Univ.-Prof. Dr. Ulla Derhaschnig 
Medical University of Vienna 
Department of Clinical Pharmacology 
Waehringer Guertel 18-20 
1090 Vienna, Austria 
Phone: +43 1 40400 29810 
Fax: +43 140400 29980 
Email: ulla.derhaschnig@meduniwien.ac.at 
Data Monitoring 
Committee 
Univ.-Prof. Dr. Heinz Burgmann, Chairman 
Department of Internal Medicine I, Div. of Infectious Diseases 
Währinger Gürtel 18-20 
1090 Vienna, Austria 
Phone: +43 1 40400 44400 
Project Management 
Data Monitoring 
Celerion 
Hainburger Strasse 33 
1030 Vienna| Austria 
Phone: +43 1 403 38 05 – 54 
Fax: +43 1 403 38 05 -66  
www.celerion.com 
Data Management 
Statistics 
Assign Data Management and Biostatistics GmbH 
Stadlweg 23 
6020 Innsbruck 
Austria 
Ph: +43 512 890 064 121 

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 
1.5.1 
22-APR--2020 
Proprietary & Confidential Information 
pg. 9 
Fax: +43 512 281 514 
BT200 Bioanalysis 
QPS 
3 Innovation Way #240 
Newark, DE 19711 USA  
Phone:  +1. 302 369-5601 
Pharmacokinetic and 
Pharmacodynamic 
Analysis 
Certara 
2000 Peel Street, Suite 570 
Montréal, Québec 
H3A 2W5 Canada 
Phone: +1 514 789 2180 
Immunogenicity (Anti-
Drug Antibody) 
Analysis 
STC Biologics 
330 Nevada Street, Newton, MA 02460, USA 
Phone: +1 617 916-9659 
Drug Supply 
ABF Pharmaceutical Services GmbH 
Brunner Strasse 63/18-19 
1230 Vienna Austria 
Phone: +43 1 8901200 
Study Design 
Prospective, double-blind, randomized, placebo-controlled First-In-
Human (FIH) study in male and female normal human volunteers (NHVs) 
with 4 sub-parts: 
• 
Part A, a single ascending dose (SAD) study; 
• 
Part B, a multiple ascending dose (MAD) study; 
• 
Part C, a desmopressin challenge study; 
• 
Part D, a relative bioavailability study. 
Rationale 
BT200 is a PEGylated aptamer that binds to the A1 domain of human von 
Willebrand Factor (VWF) and thereby inhibits VWF binding to platelet 
glycoprotein Ib (GP1b), the first step in the cascade of platelet-mediated 
thrombogenesis. Study BT200-01 is a Phase 1 FIH study designed to 
assess 
the 
human 
safety, 
tolerability, 
pharmacokinetics, 
and 
pharmacologic activity of BT200 in NHVs. In order to ensure the well-being 
of study subjects, some key safety measures have been incorporated into 
the design of this study, including use of sentinel pairs of subjects and an 
independent Data and Safety Monitoring Board (DSMB). In order to 
assess the effect of elevated VWF levels (as might be encountered in 
patients in the future) on the BT200 concentration-effect relationship,1 
single-dose cohort of BT200 administered together with a secretagogue 
for VWF (desmopressin) will be included as Part C. Finally, in order to 
assess the relative bioavailability of BT200 via subcutaneous (SC) vs. 
intravenous (IV) administration, a single dose IV cohort will be included as 
Part D. Based upon the PK and PD data observed in Parts A, C, and D 
the nominal dose levels specified in Part B may be adjusted by means of 
a Substantial Amendment to this protocol prior to initiation of dosing 
therein.   
Anticipated Start Date 
Q3/2019 
Duration of Study 
1.5 years 
Proposed Country 
Austria 
Study Site 
Medical University of Vienna 
Department of Clinical Pharmacology 
Waehringer Guertel 18-20 

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 
1.5.1 
22-APR--2020 
Proprietary & Confidential Information 
pg. 10 
1090 Vienna, Austria 
Phone: +43 1 40400 29810 
Fax: +43 140400 29980 
Email: klin-pharmakologie@meduniwien.ac.at 
Planned Sample Size 
Part A:80 NHVs 
Part B: 16 NHVs 
Part C: 8 NHVs 
Part D: 8 NHVs (including NHVs who may have previously participated 
in Part A) 
Primary Objective 
The primary objective of this study is to assess the clinical safety and 
tolerability of BT200 in NHVs. 
Secondary Objectives 
The secondary objective of this study is to assess the human PK of 
BT200. 
Exploratory Objectives 
The exploratory objectives of this study are to evaluate the human 
immunogenicity and PD of BT200, as follows: 
• 
To evaluate the presence of anti-drug antibodies (ADAs) against 
BT200 
• 
To evaluate the human pharmacodynamics (PD) of BT200 with 
respect to VWF-mediated platelet function  
• 
To evaluate the human PK/PD relationship of BT200 
• 
To evaluate potentially useful biomarkers for future clinical trials 
• 
To investigate the mechanism of action of BT200 on the human 
coagulation system 
Target Population 
NHVs 
Inclusion Criteria 
To be eligible for this study, subjects must meet all of the following 
inclusion criteria: 
1. Healthy male or female volunteers, age ≥ 18 years old at screening 
2. If female, must be post-menopausal or status post hysterectomy 
3. Able to comprehend and to give informed consent 
4. Able to cooperate with the Investigator, to comply with the 
requirements of the study, and to complete the full sequence of 
protocol-related procedures 
Exclusion Criteria 
Subjects meeting any of the following criteria will be excluded from the 
study: 
1. Clinically significant medical history (including von Willebrand 
Disease, thrombocytopathy, or any type of bleeding diathesis) or 
ongoing chronic illness that would jeopardize the safety of the subject 
or compromise the quality of the data derived from his/her 
participation in this study 
2. Clinically relevant abnormal findings on physical examination or 
clinically relevant laboratory abnormalities 
3. History of infusion hypersensitivity reactions, significant drug allergy, 
or anaphylactic reactions 
4. Substance abuse, mental illness, or any reason that makes it unlikely 
in the judgment of the Investigator for the subject to be able to comply 
fully with study procedures 

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 
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Proprietary & Confidential Information 
pg. 11 
5. Use of medication during 2 weeks before the start of the study, which 
in the judgment of the Investigator may adversely affect the subject’s 
welfare or the integrity of the study’s results  
6. Concurrent treatment with other experimental drugs or participation in 
another clinical trial with any investigational drug within 30 days or 
5 elimination half-lives (whichever is longer) prior to treatment start 

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 
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pg. 12 
Treatments 
Administered 
Part A: 
• 
Randomization ratio for BT200 to placebo = 6:2 (except for the 
sentinel pair, which will be at a ratio of 1:1) 
• 
Single SC doses of placebo or BT200 at the following BT200 dose 
levels per cohort: 
o 
Cohort 1: 0.18 mg (N=8) 
o 
Cohort 2: 0.6 mg (N=8) 
o 
Cohort 3: 1.8 mg (N=8) 
o 
Cohort 4: 6.0 mg (N=8) 
o 
Cohort 5: 12.0 mg (N=8) 
o 
Cohort 6: 18.0 mg (N=8) 
o 
Cohort 7: 24.0 mg (N=8) 
o 
Cohort 8: 24.0 mg by SC infusion (N=8) 
o 
Cohort 9: 36.0 mg by SC infusion (N=8) 
o 
Cohort 10: 48.0 mg by SC infusion (N=8) 
 
Cohorts 8-10 will be initiated only after submission and authorization of a 
Substantial Amendment to EC and AGES summarizing the results of 
Cohorts 1-7. 
. 
 
Part B: 
• 
Randomization ratio for BT200 to placebo = 6:2 
• 
Loading and maintenance doses of placebo or BT200 via a 2-hour 
IV infusion and a SC injection at the following BT200 dose levels 
per cohort: 
o 
Cohort 13: Loading dose of 12.0 mg IV + 12.0 mg SC, 
followed by 4 weekly maintenance doses of 12.0 mg SC 
(N=8) 
o 
Cohort 14: Loading dose of 24.0 mg IV + 24.0 mg SC, 
followed by4 weekly  maintenance doses of 24.0 mg SC 
(N=8) 
. 
 
Part C: 
• 
Randomization ratio for BT200 to placebo = 6:2, plus 
desmopressin challenge 
• 
Single doses of placebo or BT200 via SC injection at the following 
BT200 dose levels per cohort: 
o 
Cohort 12:  BT200 + desmopressin challenge cohort: 
48.0 mg (N=8) Single IV dose of desmopressin at the 
following dose level for all NHVs: 
o 
0.3 µg/kg 
Part D: 
• 
Randomization ratio for BT200 to placebo = 6:2 
• 
Single IV dose of placebo or BT200 at the following BT200 dose 
level for all NHVs: 
o 
Cohort 11: 24.0 mg (N=8) 
. 

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 
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pg. 13 
Treatment Schedule & 
Route of Administration 
Part A: 
• 
All dose cohorts will receive a single SC dose of BT200 or placebo 
on Day 0 at Time 0 
Cohorts 8-10 will be initiated only after submission and authorization of a 
Substantial Amendment to EC and AGES summarizing the results of 
Cohorts 1-7. 
Cohorts 16-17 will be initiated only after submission and authorization of 
a Substantial Amendment to EC and AGES summarizing the results of 
Cohorts 8-10 of Part A and Cohort 11 of Part D. 
 
Part B: 
• 
All dose cohorts will receive a total of 6 SC or IV doses of BT200 
or placebo: 
o 
Loading dose regimen consisting of equal IV and SC doses 
on Day 0 at Time 0 
o 
IV dose will be infused over 2 hours 
o 
Maintenance doses: 4 SC doses thereafter every 7 days 
 
Part C: 
• 
All subjects will receive only a single SC dose of BT200 or placebo 
on Day -4 
• 
All subjects will receive desmopressin by IV infusion over 
30 minutes on Day 0 at approximately 96 hours after the single 
SC dose of BT200 or placebo 
 
Part D: 
• 
One cohort will receive BT200 or placebo administered as an 
IV infusion over 24 hours 
Investigational 
Medicinal Product 
Test Product:  
• 
BT200 as a sterile solution for injection 
Comparator (Placebo): 
• 
Sterile saline for injection 
Non-investigational Medicinal Product (NIMP; Challenge Agent): 
• 
Desmopressin (MINIRIN® Injection) as a sterile solution for 
injection 
Primary Endpoint 
Overall safety and tolerability of BT200, assessed in terms of: 
• 
Serious, drug-related adverse events (AEs) 
• 
Premature terminations due to drug-related AEs 
• 
Patterns of serious or non-serious, drug-related AEs and/or 
clinically relevant laboratory abnormalities, vital signs, ECGs, or 
physical findings suggestive of one or more specific target organs 
for toxicity of BT200 
• 
Clinically evident bleeding assessed using the ISTH Bleeding 
Score (see Appendix 6) 
Secondary Endpoints 
Pharmacokinetic Endpoints: 
• 
Measured concentration of BT200 (and derived PK parameters) 
Exploratory Endpoints 
Immunogenicity Endpoint: 
• 
ADAs against BT200 
Pharmacodynamic Endpoints: 

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 
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Proprietary & Confidential Information 
pg. 14 
• 
Platelet Function Analyzer (PFA-100®) 
• 
VWF:ristocetin co-factor assay (VWF:RiCo) 
• 
Multiplate electrode platelet aggregometer (ristocetin) 
• 
Enzyme-linked immunosorbent assay (ELISA) for unbound 
VWF-A1 domain (REAADS®) 
Biomarker Endpoints: 
• 
VWF antigen and propeptide 
• 
Factor VIIIc activity 
• 
aPTT FS (elagic acid) 
• 
Thrombin generation Calibrated Automated Thrombogram (CAT) 
assay (Thrombinoscope®) 
Statistical 
Considerations 
Sample Size Calculation: 
As this study is exploratory and descriptive in nature, no inferential 
statistical testing is planned and therefore an exact sample size 
requirement cannot be specified. The number of NHVs planned to be 
included is expected to be sufficient to fulfill the study’s objectives, but 
not excessive. 
Analysis Plan: 
For the analysis of AEs, summary tables will be generated for the 
incidence of AEs overall and by severity. The same will be calculated for 
serious adverse events (SAEs), related AEs, and related SAEs. The AE 
and SAE summary tables will provide the number and percentage of 
subjects with AEs and the 95% confidence intervals for the event rates. 
All safety analyses will be performed within the Safety Population. 
Pharmacokinetic sampling results (serum concentration of BT200) will be 
tabulated descriptively by sampling time point. The area under the curve 
from 0 to 24 hours (AUC[0-24h]) and area under the curve from time 0 to 
time t (AUC[0-t]) will be calculated via trapezoidal rule and will be tabulated 
together with the maximum plasma concentration (Cmax) and time to 
maximum plasma concentration (Tmax), descriptively. 
Details regarding further safety endpoints and the analysis of secondary 
or exploratory endpoints will be described in the Statistical Analysis Plan. 
Interim Analysis 
A complete interim analysis is not planned; however, the DSMB will 
review relevant safety data after Cohorts 1-7 of Part A prior to initiation 
of Cohorts 8-10 of Part A, and Part D; after Cohorts 8-10 of Part A and 
Cohort 11 of Part D and prior to initiation of Cohorts 16-17 of Part A and 
Cohort C;. The data prepared for DSMB review together with the 
resulting DSMB opinion will be provided to EC and AGES as part of a 
Substantial Amendment after Cohorts 1-7 of Part A prior to initiation of 
Cohorts 8-10 of Part A and Part D;; after Cohorts 8-10 of Part A and Part 
D prior to initiation of Part B. 
Study Plan 
First Subject In (FSI) to Part A: 
Q3/2019 
Last Subject In (LSI) to Part B: 
Q3/2020 
Last Subject Out (LSO): 
Q4/2020 
Database Lock: 
Q1/2021 
Clinical Study Report (CSR):  
Q2/2021 
 

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 1.5.1 
22-APR--2020 
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pg. 15 
 
STUDY DIAGRAM 
Figure 1 
Study Diagram 
The following is an idealized diagram of the study intended to show the sequence and relative timing of dosing in Parts A, B, C, and D. Note that for the sake of 
visual simplicity, this idealized diagram gives the appearance of simultaneous dosing within each cohort; it does NOT reflect the sentinel pair dosing scheme for 
Part A, nor the possibility that the remainder of each cohort will be broken into small groups, each of which might be dosed on consecutive days.  The first DSMB 
and Regulatory interim review will occur after Cohorts 1-7 of Part A have been treated (shown in the diagram for Part A).  A second DSMB and Regulatory interim 
review will occur after Cohorts 8-10 of Part A and Cohort 11 of Part D and a third DSMB and Regulatory interim review will occur prior to initiation of Part B (not 
shown).  
 
 
 
STUDY PART A
W1
W2
W3
W4
W5
W6
W7
W8
W9
W10
W11
W12
End of 
Part A
1
↓
2
↓
3
↓
4
↓
5
 ↓
6
↓
7
↓
8
↓
9
↓
10
↓
DSMB #2 and Regulatory review
Part A Study Duration (Relative Weeks)
48.0 mg SC infusion
24.0 mg SC infusion
36.0 mg SC infusion
0.6 mg SC injection
24.0 mg SC injection
18.0 mg SC injection
12.0 mg SC injection
6.0 mg SC injection
1.8 mg SC injection
DSMB #1 and Regulatory review
Cohort 1
Single Placebo or BT200 Dose SC 2
N=8 per Cohort
0.18 mg SC injection
STUDY PART B
W1
W2
W3
W 4
W5
W 6
W7
W 8
W9
W10
W11
W12
W13
W14
End of 
Part B
Loading Placebo or BT200 Dose2
Maintenance 
Placebo or 
BT200 Dose 
SC injection 
or IV 2
13
12.0 mg IV + 12.0 mg SC
12.0 mg
↓
↓
↓
↓
↓
14
24.0 mg IV + 24.0 mg SC
24.0 mg
↓
↓
↓
↓
↓
Cohort 3
Part B Study Duration (Relative Weeks)
N=8 per Cohort

Band Therapeutics, LLC                                                 Clinical Study Protocol BT200-01 FINAL VERSION 1.5.1 
22-APR--2020 
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pg. 16 
 
  
  
 
 
 
  
 
Abbreviations: ↓=dosing; DSMB=Data and Safety Monitoring Board; IV=intravenous(ly); N/A=not applicable; NHV=normal human volunteer; SC=subcutaneous(ly); W=week 
1. 
Part A Cohorts will also include NHVs treated with placebo (N=2 for all Cohorts). Each cohort will include a sentinel pair, with a randomization ratio of 1:1 for BT200 to placebo. All 
dose cohorts will receive a single SC dose of BT200 or placebo on Day 0 of Week 1 (Cohort 1), Week 2 (Cohort 2), Week 3 (Cohort 3), Week 4 (Cohort 4), Week 5 (Cohort 5), 
Week 6 (Cohort 6), Week 7 (Cohort 7),  Week 9 (Cohort 8), Week 10 (Cohort 9), Week 11 (Cohort 10),). 
2. 
In this figure, BT200 doses are shown for each cohort. 
3. 
Part B Cohorts will also include NHVs treated with placebo (N=2 for Cohorts 13 and 14). Both dose cohorts will receive total of 6 doses of BT200 or placebo, comprising the split IV 
+ SC loading dose  Day 0 and maintenance SC doses as follows: 4 doses every 7 days of Weeks 1 to 5 (Cohort 13)and Weeks 3 to 7 (Cohort 14). 
4. 
Part C Cohorts will also include NHVs treated with placebo (N=2 for Cohort 12 [BT200 + desmopressin challenge] All subjects will receive only a single SC injection  of BT200 or 
placebo on Day -4 of Week 1. All subjects will receive desmopressin by IV infusion over 30 minutes on Day 0 of Week 1 (Cohort 12) at approximately 96 hours after the single 
dose of BT200 or placebo. 
5. 
Part D Cohort 11 will also include NHVs treated with placebo (N=2). This cohort will receive BT200 or placebo administered as an IV infusion over 24 hours on Day 0 of Week 1. 
STUDY PART D
W1
W2
W3
W 4
W5
W 6
W7
W 8
End of 
Part D
11
↓
Part D Study Duration (Relative Weeks)
Cohort 5
Single Placebo or BT200 Dose IV 2
N=8
24.0 mg
STUDY PART C
W1
W2
W3
W 4
W5
W 6
W7
W 8
End of 
Part C
Cohort 4
Single Dose SC injection2
N=8 per Cohort
Part C Study Duration (Relative Weeks)
12
Placebo or BT200: 48.0 mg SC injection
↓
Desmopressin:
0.3 µg/kg IV