Conditions: Overactive Bladder
Keywords: Overactive Bladder Benign Prostatic Hyperplasia Vibegron Beta-3 adrenergic receptor (β3-AR) β3-AR agonist
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 276
Healthy volunteers: no

Brief summary:
This study will assess the long-term safety of vibegron when dosed up to 52 weeks in men with overactive bladder (OAB) symptoms on pharmacological therapy for Benign Prostatic Hyperplasia (BPH) who previously completed treatment in Study URO-901-3005 (NCT03902080).

Detailed description:

Arms:
arm 1: Participants will receive 75 milligrams (mg) vibegron orally once daily (QD).

Interventions: intervention 1: Vibegron
Intervention descriptions: intervention 1: oral administration

Protocol document:
Prot_000.pdf: 
Urovant Sciences, GmbH 
 Confidential 
Protocol URO-901-3006 
Version 1.0 
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22 May 2019 
Protocol Title: A Phase 3 Open-Label Extension Study to Evaluate the Long-Term Safety and 
Efficacy of Vibegron in Men with Overactive Bladder (OAB) Symptoms on Pharmacological 
Therapy for Benign Prostatic Hyperplasia (BPH) 
Protocol Number: 
URO-901-3006 
Version Number: 
1.0 (Original) 
Product: 
Vibegron (URO-901, RVT-901, MK-4618, 
KRP-114V) 
Development Phase: 
3 
Sponsor:  
Urovant Sciences, GmbH 
Viaduktstrasse 8 
4051 Basel 
Switzerland 
Telephone +41 (42) 2155999 
Sponsor Contact: 
, MD, PhD 
Senior Medical Director  
5151 California Avenue, Suite 250 
Irvine, CA  92617, USA 
Telephone:
Email:
IND Number: 
106410 
EudraCT Number: 
2018-003136-72 
Emergency Contact Telephone Number: 
Refer to separate Study Contacts Page 
Contact for Serious Adverse Events 
(SAEs): 
Email: PHV_Urovant@iqvia.com 
Phone: +1- 866-599-1341 
Phone: +1-984-219-4638 (International) 
Fax: +1-866-599-1342 
Fax: +1-984-219-5032 (International) 
Approval Date: 
22 May 2019 
Sponsor Signatory: 
, MD, PhD 
Chief Medical Officer 
CONFIDENTIAL 
This document contains proprietary and confidential information of Urovant Sciences, GmbH Acceptance of this 
document constitutes agreement by the recipient that no previously unpublished information contained herein will 
be published or disclosed without the prior written approval of Urovant Sciences, GmbH with the exception that this 
document may be disclosed to study personnel under your supervision who need to know the contents for 
conducting the study and appropriate Institutional Review Boards (IRBs)/Ethics Committees (EC) under the 
condition that the personnel have agreed to keep this information confidential. The foregoing shall not apply to 
disclosure required by governmental regulations or laws, however, Urovant Sciences, GmbH shall be promptly 
notified of any such disclosure. 
                                                            NCT Number: NCT04103450   
This NCT number has been applied to the document for purposes of posting on Clinicaltrials.gov




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INVESTIGATOR STATEMENT 
Study URO-901-3006: A Phase 3 Open-Label Extension Study to Evaluate the Long-Term 
Safety and Efficacy of Vibegron in Men with Overactive Bladder (OAB) Symptoms on 
Pharmacological Therapy for Benign Prostatic Hyperplasia (BPH) (Version 1.0) 
 
• I confirm agreement to conduct the study in compliance with the protocol. 
• I acknowledge that I am responsible for overall study conduct. I agree to personally 
conduct or supervise the described study. 
• I agree to ensure that all associates, colleagues, and employees assisting in the 
conduct of the study understand their obligations and will comply with the study 
protocol. Mechanisms are in place to ensure that site staff receives the appropriate 
training and information throughout the study. 
 
 
 
 
Principal Investigator Name (Printed) 
 
Signature 
 
 
 
Date (DD/MMM/YYYY) 
 
Site Number 


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Table of Contents 
Sponsor Signature Page .................................................................................................................2 
Investigator Statement...................................................................................................................3 
Table of Contents ...........................................................................................................................4 
1. 
Protocol Summary ..........................................................................................................7 
1.1. 
Synopsis ............................................................................................................................7 
1.2. 
Schema ............................................................................................................................10 
1.3. 
Schedule of Assessments ................................................................................................11 
2. 
Introduction ...................................................................................................................12 
2.1. 
Study Rationale ...............................................................................................................12 
2.2. 
Background .....................................................................................................................12 
2.3. 
Overactive Bladder in Men with Benign Prostatic Hyperplasia .....................................12 
2.3.1. 
Vibegron .........................................................................................................................13 
2.4. 
Benefit/Risk Assessment ................................................................................................14 
2.4.1. 
Potential Benefits ............................................................................................................14 
2.4.2. 
Potential Risks ................................................................................................................14 
3. 
Objectives and Endpoints ............................................................................................16 
4. 
Study Design ..................................................................................................................18 
4.1. 
Overall Design ................................................................................................................18 
4.1.1. 
Clinical Hypotheses ........................................................................................................18 
4.2. 
Scientific Rationale for Study Design ............................................................................18 
4.3. 
Justification for Dose ......................................................................................................19 
4.4. 
End of Study Definition ..................................................................................................19 
5. 
Study Population ...........................................................................................................20 
5.1. 
Inclusion Criteria ............................................................................................................20 
5.2. 
Exclusion Criteria ...........................................................................................................20 
5.3. 
Lifestyle Considerations .................................................................................................21 
5.4. 
Screen Failures ................................................................................................................21 
6. 
Study Drug.....................................................................................................................22 
6.1. 
Study Drugs Administered..............................................................................................22 
6.2. 
Preparation/Handling/Storage/Accountability ................................................................22 
6.3. 
Study Drug Compliance..................................................................................................23 
6.4. 
Concomitant Therapy .....................................................................................................23 
6.4.1. 
Prohibited Drugs .............................................................................................................23 
6.4.2. 
Permitted Drugs ..............................................................................................................23 
6.5. 
Dose Modification ..........................................................................................................24 
6.6. 
Drug After the End of the Study .....................................................................................24 
7. 
Discontinuation of Study Drug and Subject Discontinuation 
or Withdrawal ...............................................................................................................25 
7.1. 
Discontinuation of Study Drug .......................................................................................25 
7.2. 
Subject Discontinuation/Withdrawal from the Study .....................................................25 
7.3. 
Lost to Follow Up ...........................................................................................................26 


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7.4. 
Early Study Termination .................................................................................................26 
8. 
Study Assessments and Procedures.............................................................................27 
8.1. 
Study Entry Procedures ..................................................................................................27 
8.1.1. 
Informed Consent............................................................................................................27 
8.1.2. 
Assignment of Subject Number ......................................................................................28 
8.1.3. 
Concomitant Medications ...............................................................................................28 
8.2. 
Efficacy Assessments .....................................................................................................28 
8.2.1. 
Bladder Diaries ...............................................................................................................28 
8.2.2. 
Patient-Reported Outcomes ............................................................................................29 
8.3. 
Safety Assessments .........................................................................................................30 
8.3.1. 
Vital Signs .......................................................................................................................30 
8.3.2. 
Physical Examinations ....................................................................................................30 
8.3.3. 
Post-Void Residual Volume ...........................................................................................30 
8.3.4. 
Clinical Safety Laboratory Assessments ........................................................................31 
8.4. 
Adverse Events and Serious Adverse Events .................................................................31 
8.4.1. 
Time Period and Frequency for Collecting Adverse Event and 
Serious Adverse Event Information ................................................................................32 
8.4.2. 
Method of Detecting Adverse Events and Serious Adverse 
Events ..............................................................................................................................32 
8.4.3. 
Follow-up of Adverse Events and Serious Adverse Events ...........................................32 
8.4.4. 
Regulatory Reporting Requirements for Serious Adverse Events ..................................33 
8.4.5. 
Disease-Related Events and/or Disease-Related Outcomes Not 
Qualifying as Adverse Events or Serious Adverse Events .............................................33 
8.4.6. 
Adverse Events of Special Interest .................................................................................33 
8.4.7. 
Pregnancy Management and Reporting to the Sponsor ..................................................36 
8.4.8. 
Medication Errors ...........................................................................................................37 
8.5. 
Treatment of Overdose ...................................................................................................37 
9. 
Statistical Considerations .............................................................................................38 
9.1. 
Hypotheses ......................................................................................................................38 
9.1.1  
Primary Objective and Hypothesis .................................................................................38 
9.1.2  
Secondary Objectives and Hypotheses ...........................................................................38 
9.2. 
Analysis Endpoints .........................................................................................................38 
9.2.1. 
Efficacy and Health Outcome Endpoints ........................................................................38 
9.2.2. 
Safety Endpoints .............................................................................................................40 
9.3. 
Analysis Populations.......................................................................................................40 
9.3.1. 
Efficacy Analysis Populations ........................................................................................40 
9.3.2. 
Safety Analysis Populations ...........................................................................................41 
9.4. 
Statistical Analyses .........................................................................................................41 
9.4.1. 
Statistical Methods for Efficacy Analysis.......................................................................41 
9.4.2. 
Safety Analyses ...............................................................................................................42 
9.5. 
Multiplicity .....................................................................................................................42 
9.6. 
Subgroup Analyses .........................................................................................................43 
9.7. 
Sample Size Determination ............................................................................................43 
9.8. 
Interim Analyses .............................................................................................................43 
9.9. 
Data Safety Monitoring Board ........................................................................................43 


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10. 
Supporting Documentation and Operational Considerations ..................................44 
10.1. 
Appendix 1: Regulatory, Ethical, and Study Oversight 
Considerations ................................................................................................................45 
10.1.1. Regulatory and Ethical Considerations ...........................................................................45 
10.1.2. Financial Disclosure........................................................................................................45 
10.1.3. Informed Consent Process ..............................................................................................46 
10.1.4. Data Protection................................................................................................................46 
10.1.5. Committees Structure......................................................................................................46 
10.1.6. Posting Clinical Study Data ............................................................................................46 
10.1.7. Data Quality Assurance ..................................................................................................46 
10.1.8. Source Documents ..........................................................................................................48 
10.1.9. Study and Site Closure ....................................................................................................49 
10.1.10. Publication Policy ...........................................................................................................49 
10.1.11. Compliance with Protocol...............................................................................................49 
10.2. 
Appendix 2: Clinical Laboratory Tests ...........................................................................50 
10.3. 
Appendix 3: Adverse Events: Definitions and Procedures for 
Recording, Evaluating, Follow-up, and Reporting .........................................................51 
10.4. 
Appendix 4: Abbreviations .............................................................................................57 
10.5. 
Appendix 6: Study Tabular Summary ............................................................................60 
10.6. 
Appendix 7: Prohibited Medications and Non-Drug Therapies .....................................62 
10.7. 
Appendix 12: Study Schedule Supplement ....................................................................63 
10.7.1. Study Entry, Visit 11 (Week 24 [Day 169 ± 4 days]) .....................................................63 
10.7.2. Open-label Treatment Period, Visit 12 (Week 28 [Day 197 ± 4 
days])...............................................................................................................................63 
10.7.3. Open-label Treatment Period, Visit 13 (Week 36 [Day 253 ± 
4 days])............................................................................................................................64 
10.7.4. Open-label Treatment Period, Telephone Contact (Week 44 [Day 
308 ± 4 days]) .................................................................................................................64 
10.7.5. Open-label Treatment Period, Visit 14 (Week 52 [Day 365 ± 4 
days]) or Early Withdrawal .............................................................................................64 
10.8. 
Appendix 14: Patient-Reported Outcomes Questionnaires, 
Descriptions, and Instructions.........................................................................................65 
10.8.1. International Prostate Symptom Score (IPSS) ................................................................65 
10.8.4. Patient Global Impression Scale .....................................................................................75 
11. 
References ......................................................................................................................82 
 
List of Tables 
Table 1 
Summary of Study Drugs .................................................................................. 22 
Table 10-1  
Clinical Laboratory Tests .................................................................................. 50 
 


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1. 
Protocol Summary 
1.1. 
Synopsis 
Protocol Title: A Phase 3 Open-Label Extension Study to Evaluate the Long-Term Safety and 
Efficacy of Vibegron in Men with Overactive Bladder (OAB) Symptoms on Pharmacological 
Therapy for Benign Prostatic Hyperplasia (BPH) 
Protocol Number: URO-901-3006 
Brief Title: Extension Study of Vibegron in Men with OAB Symptoms With BPH 
Study Rationale: Beta-3 adrenergic receptor (β3-AR) agonists have shown efficacy (with 
improved safety relative to anticholinergics) in treating persistent OAB symptoms in men on 
BPH therapies. This subset of the broader OAB population setting has unique safety needs (eg, 
possibility of urinary retention) that have not been fully evaluated in previous clinical programs 
of OAB treatments. Study URO-901-3006 is designed to evaluate long-term safety and efficacy 
of vibegron (75 mg once daily [QD] administered for 28 weeks) in men with symptoms of OAB 
while receiving pharmacological therapy for BPH who have received 24 weeks of study drug in 
double-blind, placebo-controlled, Phase 3 Study URO-901-3005.  
Objectives: The primary study objective is to assess the long-term safety of vibegron when 
dosed for up to 52 weeks in men with OAB symptoms on pharmacological therapy for BPH who 
previously completed treatment in Study URO-901-3005. The primary clinical hypothesis is that 
vibegron + BPH pharmacological therapy for BPH treatment is safe for long-term use in men 
with OAB symptoms and BPH. 
The secondary study objective is to assess the long-term efficacy of vibegron when dosed for up 
to 52 weeks in men with OAB symptoms on pharmacological therapy for BPH who previously 
completed treatment in Study URO-901-3005. 
Endpoints:  
Note: With respect to study endpoints, “per day” refers to a “Diary Day”, which is defined as the 
time between when the subject gets up for the day each morning and the time the subject gets up 
for the day the next morning as recorded in the subject bladder diary (approximately a 24-hour 
period). 
Safety assessments will include treatment emergent AEs, clinical laboratory tests, physical 
examinations, vital signs, PVR volume, and Total IPSS. 


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Efficacy endpoints include the following: 
• Change from baseline (CFB) at Week 52 in the average number of micturition episodes 
per day 
• CFB at Week 52 in the average number of urgency episodes (urgency: need to urinate 
immediately) per day 
• CFB at Week 52 in the average the number of nocturia episodes per night  
• CFB at Week 52 in the average number of urge urinary incontinence episodes per day in 
subjects with incontinence at baseline in Study URO-901-3005  
• CFB at Week 52 in the average of International Prostate Symptom Score (IPSS) 1-week 
recall) Storage score (1-week recall) 
• CFB at Week 52 in the average volume voided per micturition 
See Section 3 (Objective and Endpoints) for the other/exploratory endpoints, including quality-
of-life endpoints. 
Statistical Methods: 
Safety analyses will be based on all subjects who receive vibegron (through Week 52 of this 
extension study). Only one treatment group (vibegron) will be reported for safety data. Baseline 
will be the baseline for Study URO-901-3005. Descriptive statistics will be used to summarize 
safety endpoints.  
The efficacy analyses will be for descriptive purposes only and will be conducted using the FAS-
Extension population, which is a subset of the Study URO-901-3005 treated population which 
have completed the 24-week treatment period in Study URO-901-3005 and enrolled into this 
extension study.   
The efficacy endpoints of change from baseline in average number of micturitions, average 
number of urgency episodes, average number of nocturia episodes, average number of urge 
urinary incontinence (UUI) episodes, average of IPSS storage score, and average volume voided 
per micturition will be analyzed separately using a mixed model for repeated measure (MMRM) 
with restricted maximum likelihood estimation. The analysis model for each efficacy endpoint 
will include terms or visit, baseline, baseline stratification factors (those found to be significant 
in Study URO-901-3005). An unstructured covariance matrix will be used to model the 
correlation among repeated measurements. The Kenward-Roger adjustment will be used with 
restricted (or residual) maximum likelihood (REML) to make statistical inference. The adjusted 
means for vibegron and visit will be estimated along with the 95% CI. Only the week-52 subjects 
(those on active treatment in Study URO-901-3005 and in Study URO-901-3006) will be 
included in the model. 
Descriptive statistics will be used to summarize all efficacy endpoints. Summaries of efficacy 
endpoint will be presented by two treatment groups: vibegron for 52 weeks and vibegron for 
28 weeks as determined by the randomized treatment in Study URO-901-3005. 


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Overall Study Design: This is an international Phase 3, open-label 28-week extension study to 
evaluate the safety and efficacy of vibegron 75 mg in men with symptoms of OAB on stable 
doses of pharmacological therapy for BPH. This study is an extension for subjects who have 
completed the Phase 3, double-blind, randomized, 24-week Study URO-901-3005.  
Approximately 300 men with symptoms of OAB on stable doses of pharmacological therapy for 
BPH who completed 24 weeks in Study URO-901-3005 and continue to qualify for this study 
may be permitted to enroll in this extension study, from approximately 60 study sites. 
During this extension study, subjects who had been randomized in Study URO-901-3005 to 
receive vibegron 75 mg will continue their same treatment once daily for an additional 28 weeks, 
and subjects who had been randomized in Study URO-901-3005 to the placebo group will 
receive study treatment of vibegron 75 mg once daily for 28 weeks during the extension. Thus, 
through participation in both the URO-901-3005 and URO-901-3006 (extension) studies, 
subjects originally randomized to vibegron will receive 52 weeks total of vibegron treatment, and 
subjects originally randomized to placebo will receive 28 weeks total of vibegron treatment. 
Study visits will be named to reflect continuation from Study URO-901-3005, with the first 
study visit of this extension study occurring at Week 24. Following enrollment in this extension 
study, subjects will return to the clinic for visits at Week 28, Week 36, and Week 52 (all relative 
to Day 1 of Study URO-901-3005). A telephone contact will occur at Week 44 to review 
AEs/serious AEs (SAEs) and concomitant medications. 
This study consists of a 28-week open-label Treatment Period. Unscheduled Visit(s) may be 
arranged as needed. 
Number of Subjects: Up to 300 subjects who complete Study URO-901-3005 may be offered 
the opportunity to roll over into this study.  
Number of Sites: Approximately 60 sites in North America and Europe. 
Study Drug Groups and Study Duration: 75 mg vibegron, QD, administered orally. Study 
drug will be administered in an open-label (not blinded) manner for 28 weeks on Study URO-
901-3006. Note: upon entering this extension study, all subjects will have already received 
2 weeks of treatment with placebo plus an additional 24 weeks of blinded treatment with either 
vibegron or placebo in Study URO-901-3005. No dosage adjustments will be allowed. 
Subjects will continue on the same alpha-blockers (eg, tamsulosin, doxazosin, and alfuzosin) 
with or without 5α-reductase inhibitors (5-ARIs) use (eg, finasteride, dutasteride, and alfatradiol) 
in Study URO-901-3006 as administered in Study URO-901-3005. 
Data Safety Monitoring Board (DSMB): A DSMB will be retained to assess, on an ongoing 
basis, all safety aspects of this study. This will be an external independent DSMB that monitors 
the safety for both Study URO-901-3005 and Study URO-901-3006. The detailed activities 
including meeting plans will be described and documented in the DSMB Charter. A separate 
statistical analysis plan will be prepared for the DSMB. 


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1.2. 
Schema 
 
 


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1.3. 
Schedule of Assessments  
 
Open-Label Treatment 
 
Visit 11 
Visit 12 
Visit 13 
Telephone Call  
Visit 14 
or Early WD 
Day/Week 
Week 24  
(Day 169) a 
Week 28  
(Day 197) 
Week 36  
(Day 253) 
Week 44  
(Day 308) 
Week 52 or  
Early WD (Day 365) 
Visit Window 
±4  
days 
± 4  
days 
± 4  
days 
± 4  
days  
± 4  
days 
Informed Consent 
X 
 
 
 
 
Subject Entry into IWRS 
X 
 
 
 
 
Inclusion and Exclusion Criteria Reviewa 
X 
 
 
 
 
AE/SAE Review 
† 
X 
X 
X 
X 
Concomitant Medication Review  
† 
X 
X 
X 
X 
IPSSb  
† 
 
X 
 
X 
PGIb  
† 
 
 
 
X 
Vital Signsc 
† 
X 
X 
 
X 
Brief Physical Examination 
† 
 
 
 
X 
PVR volume 
† 
X 
X 
 
X 
Laboratory Assessmentsd  
† 
 
X 
 
X 
Review and Data Enter Completed 
Bladder Diarye 
† 
X 
X 
 
X 
Dispense Bladder Diary  
X 
X 
X 
 
 
Dispense Open-label Study Drug  
X 
X 
X 
 
 
AE = adverse event;
IPSS = International Prostate Symptom Score; IWRS = interactive web 
response system; OAB = overactive bladder; 
 PGI = Patient Global Impression; PRO = patient-reported outcomes; 
PVR = Post-void residual; SAE = serious adverse event; WD = withdrawal  
† 
For noted procedures, the Week 24 (Visit 11) assessments for Study URO-901-3005 serve as initial on-study visit for Study URO-901-3006; these procedures will not be duplicated.  
a 
Recheck clinical status before enrollment and first dose of open-label study drug. Initiation of Study URO-901-3006 should occur within 7 days of Study URO-901-3005 
Week 24/End-of-Study visit.  
b 
PRO questionnaires should be completed prior to other procedures, including vital signs, blood draws, and study drug dosing. 
c 
Vital signs (including blood pressure [in triplicate], pulse, body temperature, respiration rate, and weight) should be taken prior to blood draws and study drug dosing. 
d 
Refer to Section 10.2 for a list of laboratory assessments performed. 
e 
Subject completes the Bladder Diary PRIOR to each visit.  


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2. 
Introduction 
2.1. 
Study Rationale 
Men with overactive bladder (OAB) symptoms associated with benign prostatic hypertrophy 
(BPH) represent an OAB subpopulation that has not been extensively studied. Often in these 
patients, OAB symptoms remain inadequately addressed despite treatment for their BPH 
(ie, alpha-blocker with or without 5-ARI). For reasons described below, this population warrants 
specific clinical studies to adequately evaluate and address their unique medical needs. Study 
URO-901-3006 is an extension study to Phase 3 Study URO-901-3005 designed to evaluate 
safety, tolerability, and efficacy of vibegron (75 mg once daily [QD]) for up to 52 weeks in men 
with OAB symptoms on pharmacological therapy for BPH. 
2.2. 
Background 
2.3. 
Overactive Bladder in Men with Benign Prostatic Hyperplasia 
OAB is highly prevalent and affects approximately 16% of the population in the United States 
and Europe. Prevalence increases with age, affecting approximately one third of people 75 years 
and older [Stewart, 2003; Milsom, 2001]. Despite similar prevalence between the sexes and 
growing clinical trial evidence that men benefit from pharmacotherapy, men are less likely to use 
treatment for OAB [Goldman, 2016].  
The International Continence Society (ICS) defines OAB as urgency, with or without urge 
incontinence, usually associated with frequency and nocturia [Abrams, 2002]. Urgency is 
defined as a sudden compelling desire to void which is difficult to defer and, from the medical 
definition perspective, it is a necessary symptom for OAB. Urgency incontinence is the 
involuntary loss of urine accompanied by urgency (referred to as OAB Wet) and is present in 
approximately one-third of patients with OAB [Stewart, 2003; Milsom, 2001]. In the absence of 
incontinence, OAB is referred to as OAB Dry. Urgency incontinence is distinguished from stress 
urinary incontinence, which is the involuntary loss of urine on effort or physical exertion (eg, 
sporting activities), or upon sneezing or coughing. When both components are present, the 
classification is mixed urinary incontinence, with either urgency or stress specified as the 
predominant component.  
Although the overall prevalence of OAB in men and women is similar, there are major 
differences in predominating symptoms. Men are more likely to experience urgency, frequency, 
and nocturia accompanied by lower urinary tract symptoms (LUTS) associated with voiding 
dysfunction, whereas women are twice as likely to experience incontinence [Tubaro, 2017]. 
Since most men with OAB symptoms do not have urinary incontinence, the micturition 
frequency and the urgency become the predominant symptoms that characterize the disease for 


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the men with OAB on pharmacological therapy for BPH [Helfand, 2012]. Among men, urinary 
incontinence significantly increases after the age of 65 years.  
Historically in men with BPH, LUTS have been presumed to result from bladder outlet 
obstruction secondary to prostate enlargement. However, men may have OAB symptoms in the 
absence of, or in conjunction with, voiding symptoms associated with BPH or urodynamic 
evidence of bladder outlet obstruction [Nakagawa, 2008; Hyman, 2001]. BPH can be associated 
with debilitating LUTS, categorized by storage symptoms (eg, urinary frequency, urgency, and 
nocturia) and voiding dysfunction (eg, decreased and intermittent force of stream and the 
sensation of incomplete bladder emptying).  
2.3.1. 
Vibegron 
Initial pharmacologic therapy for OAB symptoms in men with BPH traditionally has been 
directed at minimizing the obstruction and has included α1-adrenergic receptor antagonists to 
relax the muscles of the prostate and bladder neck or 5-ARIs to reduce prostate growth 
[American Urological Association, 2010]. While patients may see some improvement in OAB 
symptoms with these BPH therapies (eg, improvement in urinary flow), symptom control for 
OAB symptoms is incomplete, for many, and urinary urgency, incontinence, frequency, or 
nocturia persists [Abrams, 2002]. To address persistent OAB symptoms in men with BPH, 
combinations of BPH therapies with OAB medications, such as anticholinergics, have shown 
promise [Chapple, 2009; Oelke, 2013]. However, the clinical use of anticholinergics is limited 
by mechanism-based side effects including dry mouth and constipation [MacDiarmid, 2008] and 
the potential for CNS adverse effects [Gray, 2015; Risacher, 2016]. Further, 
contraindications/precautions exist due to the possibility of urinary retention and bladder outflow 
obstruction [Detrol prescribing information, 2016].  
ß3-adrenoceptor (β3-AR) agonists for OAB (eg, mirabegron) have demonstrated efficacy in 
treating persistent OAB symptoms in men on BPH therapies [Ichihara, 2014] with a more 
favorable safety profile relative to the anticholinergics. In fact, experts have recommended that 
these agents be specifically studied in this patient population [Maman, 2014; Chapple, 2009; 
Van Kerrebroeck, 2001; Chapple, 2017].  
Vibegron is a potent and highly selective β3-AR agonist demonstrating > 9000-fold selectivity 
for activation of β3-AR over β2-AR and β1-AR in cell based in vitro assays. β-adrenergic 
receptors are prototypic G-protein coupled receptors expressed on the surface of cells, and 
mediate intracellular signaling via coupling to Gs and increasing levels of intracellular cyclic 
adenosine monophosphate (cAMP). β3-ARs are widely distributed in humans and are the most 
prevalent β-AR subtype expressed on human detrusor smooth muscle [Takeda, 2000]. In isolated 
human bladder smooth muscle, activation of β3-AR using subtype-selective agonists results in 
smooth muscle relaxation suggesting a role for β3-AR agonists during the filling phase of the 
micturition cycle [Yamaguchi, 2002; Biers, 2006]. In rodent models of bladder overactivity, β3-
AR agonists relax bladder smooth muscle and suppress detrusor smooth muscle instability and 
hyperreflexia [Takeda, 2000; Woods, 2001; Takeda, 2002; Kaidoh, 2002]. In rhesus monkeys, 


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dose dependent increases in bladder capacity and decreases in micturition pressure were 
observed with vibegron. Bladder capacity was further increased by vibegron in combination with 
tolterodine or darifenacin [Di Salvo, 2017]. These results have supported a large and 
comprehensive clinical development program for vibegron in the general OAB population, 
including both male and female subjects. 
More than 2300 subjects (including 1840 with OAB and 460 healthy volunteers) have received 
vibegron in Phase 1, 2, and 3 clinical studies. Assessment of safety laboratory parameters and 
mean vital sign values over time, including heart rate and blood pressure, showed no clinically 
meaningful differences for any active treatment group relative to placebo or anti-muscarinic 
comparators. A large, multicenter Phase 3 study of vibegron for the treatment of OAB in women 
and men is ongoing. Discontinuation rates due to adverse events (AEs) were low (< 5%) in all 
clinical studies conducted to date with vibegron. In a completed Phase 3 study in Japan in more 
than 1200 subjects with symptoms of OAB, there were no notable mean changes in post voided 
residuals observed [Yoshida, 2018]. A more detailed description of vibegron, including 
pharmacology, efficacy, and safety data in overactive bladder, is provided in the Investigator’s 
Brochure.  
2.4. 
Benefit/Risk Assessment 
2.4.1. 
Potential Benefits 
Vibegron’s mechanism of action has the potential to demonstrate significant therapeutic benefit 
in the treatment of OAB symptoms in men with BPH on pharmacological therapy for their BPH 
symptoms. 
2.4.2. 
Potential Risks 
The initial clinical program for vibegron in the treatment of OAB includes subjects of both sexes. 
It is estimated that the ongoing Phase 3 program in OAB (RVT-901-3003 and RVT-901-3004) 
will include approximately 15% males, which will contribute to safety data comprising more 
than 200 men in addition to those enrolled in the URO-901-3005 and URO-901-3006 studies. 
Based on aggregate preclinical, clinical pharmacology, and Phase 2 and 3 studies, AEs of special 
interest (AESIs) predefined for specific evaluations in vibegron clinical studies are as follows:  
• Potential major cardiovascular events 
• Hypertension 
• AEs suggestive of orthostatic hypotension as confirmed by orthostatic vital signs 
• AEs suggestive of cystitis or urinary tract infection 
• Elevated aspartate transaminase (AST) or alanine transaminase (ALT) laboratory value 
requiring that the study drug be temporarily withheld or permanently discontinued 
• Neoplasm 


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In addition, the risk for increases in post void residual urine volume and urinary volume will be 
closely monitored for post-void retention during the study. Study URO-901-3005 enrollment 
criteria will exclude men with PVR volume ≥ 100 mL at screening and at baseline and those with 
maximum urinary flow (Qmax) less than 5 mL/second. Post-void residual urine volume will be 
assessed throughout the study, and subjects with a single PVR volume of ≥ 300 mL or an AE of 
urinary retention will be withdrawn.  
Prespecified definitions of urinary retention and other AESIs are provided in the Adverse Event 
of Special Interest section (Section 8.4.6). More detailed information about the known and 
expected benefits and risks from the study and reasonably expected AEs of vibegron may be 
found in the Investigator’s Brochure.  


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3. 
Objectives and Endpoints 
Assessments in Study URO-901-3006 are referenced to the initial Baseline (Visit 3) from 
Study URO-901-3005. Therefore, CFB study endpoints for Study URO-901-3006 are relative to 
Study URO-901-3005 baseline. At the Week 52 visit, CFB comprises 52 weeks of vibegron 
exposure for subjects randomized to vibegron in Study URO-901-3005 and 28 weeks of vibegron 
exposure for subjects randomized to placebo.  
With respect to study endpoints, “per day” refers to a “Diary Day”, which is defined as the time 
between when the subject gets up for the day each morning and the time the subject gets up for the 
day the next morning as recorded in the subject bladder diary (approximately a 24-hour period) 
Objective 
Endpoints 
The primary study objective is to 
demonstrate the long-term safety of 
vibegron 75 mg in men with BPH with 
symptoms of OAB.  
 
The secondary study objective is to 
demonstrate the long-term efficacy of 
vibegron 75 mg in men with BPH with 
symptoms of OAB.   
Safety 
• 
Including AEs, clinical laboratory, vital sign assessments, PVR 
volume, and Total IPSS 
Efficacy 
• 
CFB at Week 52 in the average number of micturition episodes per 
day 
• 
CFB at Week 52 in the average number of urgency episodes 
(urgency: need to urinate immediately) per day 
• 
CFB at Week 52 in the average number of nocturia episodes per 
night  
• 
CFB at Week 52 in the average number of urge urinary 
incontinence episodes per day in subjects with incontinence at 
Study URO-901-3005 baseline  
• 
CFB at Week 52 in the average of IPSS Storage score (1-week 
recall) 
• 
CFB at Week 52 in the average volume voided per micturition 
 
 
 
Other/Exploratory – Efficacy 


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Other/Exploratory – Quality of Life
Notes: “change from baseline” (CFB) refers to the change from the baseline in the parent study, URO-901-3005; “per day” refers 
to a continuous 24-hour period as defined above; week timepoints (eg, Week 52) refer to the weeks since the baseline assessment 
in the parent study, URO-901-3005. 
Abbreviations: AE = adverse events; BPH = benign prostatic hypertrophy; CFB = change from baseline; 
 
IPSS = International Prostate Symptom Score; OAB = overactive 
bladder;
PGI = Patient Global Impression; QoL = quality of life 


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4. 
Study Design 
4.1. 
Overall Design 
This study is a Phase 3, open-label, 28-week extension study to evaluate the safety and efficacy 
of vibegron 75 mg in men with symptoms of overactive bladder (OAB) on stable doses of 
pharmacological therapy for BPH. This study is an extension for subjects who have completed 
the Phase 3, double-blind, randomized, 24-week Study URO-901-3005.  
Subjects who complete Study URO-901-3005 may be given the opportunity to enroll in this 
extension study. It is anticipated that approximately 300 men who completed Study URO-
9013005 and continue to qualify for this study may be permitted to enroll in this extension study. 
During this extension study, subjects who had been randomized to receive vibegron 75 mg in 
Study URO-901-3005 will continue their same treatment once daily for an additional 28 weeks; 
subjects who had been randomized to receive placebo in Study URO-901-3005 will receive 
study treatment of vibegron 75 mg once daily for 28 weeks during this extension study. Thus, 
through participation in both the URO-901-3005 and URO-901-3006 (extension) studies, 
subjects originally randomized to vibegron will receive 52 weeks total of vibegron treatment, and 
subjects originally randomized to placebo will receive 28 weeks total of vibegron treatment. 
Study visits will be named to reflect continuation from the URO-901-3005 study, with the first 
study visit of this extension study occurring at Week 24 (Visit 11). Following enrollment in this 
extension study, subjects will return to the clinic for visits at Week 28, Week 36, and Week 52 
(all relative to Day 1 of URO-901-3005). A telephone contact will occur at Week 44 to review 
AEs/SAEs and concomitant medications. 
This study consists of a 28-week open-label Treatment Period. Unscheduled Visit(s) may be 
arranged as needed.  
4.1.1. 
Clinical Hypotheses 
The primary clinical hypothesis is that is that the vibegron + pharmacological therapy for BPH 
treatment is safe in men with OAB symptoms and BPH. 
4.2. 
Scientific Rationale for Study Design 
The clinical use of anticholinergics is limited by adverse effects and, notably, contraindications 
and precautions unique to men with OAB symptoms receiving treatment for BPH due to the 
possibility of urinary retention and bladder outflow obstruction. ß3-adrenoceptor agonists have 
shown efficacy (with improved safety relative to anticholinergics) in treating persistent OAB 
symptoms in men on BPH therapies [Ichihara, 2014]; however, no therapeutic agents are labeled 
specifically for this population, and clinical study data have been limited. In addition, this 
population has a distinct symptom profile and relevant efficacy needs (eg, nocturia) that warrant 
further study.   


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4.3. 
Justification for Dose 
This study will use a vibegron dose of 75 mg QD, which is the same dose used for the pivotal 
Phase 3 study of OAB in men and women (RVT-901-3003) and the associated long-term Phase 3 
extension study (RVT-901-3004) as well as in the parent OAB/BPH study, URO-901-3005.  
4.4. 
End of Study Definition 
The end of the study is defined as the date that the last subject has completed the study (ie, 
through the Week 52 Visit), discontinued from the study, or is lost to follow-up.  


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5. 
Study Population 
The study is being conducted in men with OAB symptoms while receiving pharmacological 
therapy for BPH who have completed vibegron Study URO-901-3005. Specific inclusion and 
exclusion criteria are specified below. Prospective approval of protocol deviations to recruitment 
and enrollment criteria, also known as protocol waivers or exemptions, is not permitted.  
Eligibility for Study URO-901-3006, including blood pressure thresholds, should be confirmed 
at the Week 24 visit (Visit 11) of Study URO-901-3005. For laboratory assessments, the most 
recent results available from Study URO-901-3005, including those from Week 24, may be used 
to confirm eligibility.  The subject should be enrolled into Study URO-901-3006 within 7 days of 
completing the Week 24 visit from Study URO-901-3005. 
5.1. 
Inclusion Criteria 
Subjects will be eligible for inclusion in this study only if all of the following criteria apply: 
1. Has completed participation of the 24-week double-blind treatment period in 
Study URO-901-3005 and demonstrated compliance with the study procedures and study 
medication schedule in the opinion of the investigator. 
2. Is capable of giving written informed consent, which includes compliance with the 
requirements and restrictions listed in the consent form. 
3. Has ability to continue to receive a stable dose of BPH treatment with either a) alpha blocker 
monotherapy or b) alpha blocker + 5-ARI.  
4. In the opinion of the investigator, is able and willing to comply with the requirements of the 
protocol, including completing study questionnaires and the Bladder Diary. 
5.2. 
Exclusion Criteria 
Subjects will not be eligible for inclusion in this study if any of the following criteria apply: 
1. Experienced any SAE in Study URO-901-3005 that was reported as “possibly or probably 
related” to study treatment by the investigator. 
2. Is a night-shift worker or plans to become a night-shift worker during the study. 
3. Is using any prohibited medications as detailed in Section 10.6 (Appendix 7). 
4. Is taking or using any medications to treat erectile dysfunction (ED) but is not using them on 
a regular schedule. If taking less than 2 times per week, ED medications with a short half-
life such as sildenafil and vardenafil are allowed. 
5. Has any planned procedures to treat ED (eg, implantation of a penile device) during the 
treatment period or has any planned prostate procedure.  
6. Has a planned procedure to implant a sacral neurostimulation (SNS) or use of any posterior 
tibial nerve stimulation (PTNS) device. 
7. Has coronary or neurovascular interventions planned during the duration of the study. 


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8. Has uncontrolled hyperglycemia (defined as fasting blood glucose > 150 mg/dL or 
8.33 mmol/L and/or non-fasting blood glucose > 200 mg/dL or 11.1 mmol/L) based on most 
recent available lab results in Study URO-901-3005 or uncontrolled in the opinion of the 
investigator. 
9. Has uncontrolled hypertension (systolic blood pressure of ≥ 180 mmHg and/or diastolic 
blood pressure of ≥ 100 mmHg) or has a resting heart rate (by pulse) > 100 beats per minute. 
10. Has systolic blood pressures ≥ 160 mmHg but < 180 mmHg, unless deemed by the 
investigator as safe to proceed in this study and able to complete the study per protocol. 
11. Subject has current evidence of any clinically significant condition, therapy, lab 
abnormality, or other circumstances that might, in the opinion of the investigator, confound 
the results of the study, interfere with the subject’s ability to comply with study procedures, 
or make participation in the study not in the subject’s best interest. 
5.3. 
Lifestyle Considerations 
Not applicable for this study.  
5.4. 
Screen Failures 
Not applicable for this study. 


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6. 
Study Drug 
Subjects will retain the same subject identification number as was assigned in Study URO-901-
3005. 
6.1. 
Study Drugs Administered 
Table 1 
Summary of Study Drugs 
Study Drug Name 
Vibegron 
Dosage Formulation 
tablet 
Identity of Formulation 
75 mg 
Route of Administration  
oral 
Dosing Instructions 
once daily 
Packaging and Labeling 
Study drug will be provided in HDPE bottles with 
child-resistant caps. Each bottle will contain 32 tablets and 
will be labeled as required per country requirement. 
Manufacturer 
Patheon, Cincinnati, Ohio, US 
Number and Timing of Drugs 
Treatment Period: 1 tablet daily  
The study drug will be supplied in bottles each containing 32 tablets (4-week treatment and 
4-day extra supply) and labeled with the protocol number, lot number, expiration date, study 
drug name (vibegron 75 mg tablets) and number of tablets, directions for use, storage 
information, warning language (Keep Out of Reach of Children. For Clinical Trial Use Only. To 
be used by qualified investigators only. Caution: New Drug—Limited by United States Law to 
Investigational Use.), and the US Sponsor name and address. Immediately before dispensing the 
study drug, the investigator (or appropriately trained designee) will write the subject number, 
visit no. and the dispense date on the detachable panel of the label, which also includes the 
protocol number, bottle number and lot number. 
Subjects will take 1 tablet of study drug (vibegron) once daily for up to 28 weeks.   
6.2. 
Preparation/Handling/Storage/Accountability 
Clinical supplies must be stored in a secure, limited-access location under the storage conditions 
specified on the label. 
Receipt and dispensing of Study Treatment must be recorded by an authorized person at the 
study site. 
Clinical supplies may not be used for any purpose other than that stated in the protocol. 


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The investigator is responsible for keeping accurate records of the clinical supplies received from 
the Sponsor or designee, the amount dispensed to and returned by the subjects, and the amount 
remaining at the conclusion of the study. These records will be monitored throughout the study. 
For all sites, the local country Sponsor personnel or designee will provide appropriate 
documentation that must be completed for drug accountability and return.  
6.3. 
Study Drug Compliance 
Study drug compliance will be closely monitored by counting the number of tablets dispensed 
and returned. Before dispensing new study drug at applicable visits, study site personnel will 
make every effort to collect all unused study drug and empty bottles. 
The study site will keep an accurate drug disposition record that specifies the amount of study 
drug administered to each subject and the date of administration. 
6.4. 
Concomitant Therapy 
The use of any concomitant medication, prescription or over-the-counter, is to be recorded on the 
subject’s electronic case report form (eCRF) at each visit along with the reason the medication is 
taken, the dates of administration, and the dose. 
6.4.1. 
Prohibited Drugs  
Section 10.6 provides a listing of specific restrictions for concomitant therapy use during the 
study. If there is a clinical indication for any therapy that is specifically prohibited during the 
study, discontinuation from Study Treatment may be required. The investigator should discuss 
any questions regarding this with the Sponsor’s representing Medical Monitor. The final decision 
on any supportive therapy rests with the investigator and/or the subject's primary physician. 
However, the decision to continue the subject on Study Treatment requires the mutual agreement 
of the investigator and the Sponsor’s designated Medical Monitor. 
Consult the Sponsor’s designated Medical Monitor if there is any uncertainty regarding subject 
use of a particular drug or drug class.  
6.4.2. 
Permitted Drugs 
With the exception of the agents described in Section 10.6, any other concomitant medication 
deemed necessary for the welfare of the subject during the study may be given at the discretion 
of the investigator. If the permissibility of a specific medication/drug is in question, please 
contact the Sponsor’s designated Medical Monitor. Any medication or vaccine (including over-
the-counter or prescription medicines, vitamins, and/or herbal supplements) that the subject is 
receiving at the time of enrollment or receives during the study must be recorded in the eCRF. 


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6.5. 
Dose Modification 
No dose modification of study drug is permitted. Study drug should be withheld for liver test 
abnormalities as described in Section 8.4.6.2. 
6.6. 
Drug After the End of the Study 
Study drug (vibegron) will not be provided after the Week 52 visit. 


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7. 
Discontinuation of Study Drug and Subject Discontinuation or 
Withdrawal 
A premature discontinuation will occur if a subject who signs the informed consent form (ICF) 
and is dosed ceases participation in the study, regardless of circumstances, before the completion 
of the protocol-defined study procedures. 
7.1. 
Discontinuation of Study Drug 
Subjects who discontinue study drug will complete the Week 52/Early Withdrawal assessments 
specified in the Schedule of Assessments (Section 1.3).  
Reasons for discontinuation from the study drug include the following: 
• Any AE of urinary retention  
• Any occurrence of PVR volume ≥ 300 mL 
Discontinuation of study drug for abnormal liver function should be considered by the 
investigator when a subject meets all of the conditions outlined in Section 8.4.6.2 for Hy’s law or 
if the investigator believes that it is in best interest of the subject (reason for discontinuation will 
be AE).  
7.2. 
Subject Discontinuation/Withdrawal from the Study 
• A subject who discontinues study drug will also be withdrawn from the study. 
• A subject may choose to withdraw from the study at any time at his own request, or may be 
withdrawn at any time at the discretion of the investigator for safety, behavioral, compliance, 
or administrative reasons. 
• If a subject withdraws consent for disclosure of future information, the Sponsor or designee 
may retain and continue to use any data collected before such a withdrawal of consent. 
• If a subject withdraws from the study, he may request destruction of any blood or urine 
samples taken and not tested, and the investigator must document this in the site study 
records. 
• Subjects who withdraw from the study will not be replaced.  
The Week 52/Early Withdrawal assessments will be completed when a subject withdraws or is 
withdrawn from the study, if possible (see Schedule of Assessments in Section 1.3). 


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Reasons for discontinuation from the study include the following: 
• AE 
• Lack of efficacy 
• Noncompliance 
• Withdrawal of consent 
• Lost to follow-up 
• Physician decision 
• Protocol deviation 
• Death 
• Other 
7.3. 
Lost to Follow Up 
Should a subject fail to attend a required study visit, the site should attempt to contact the subject 
and re-schedule the missed visit as soon as possible. The site should also counsel the subject on 
the importance of maintaining the assigned visit schedule and ascertain whether or not the 
subject wishes to and/or should continue in the study based on previous non-compliance. In 
cases where the subject does not return for the rescheduled visit or cannot be reached to 
reschedule the missed visit, the site should make every effort to regain contact with the subject 
so that they can appropriately be withdrawn from the study with a primary reason of “Lost to 
Follow-up”. Including at least three documented attempts to contact the subject (ie, phone, email, 
or certified letter). Efforts to establish the possible reason for discontinuation should be 
documented. 
7.4. 
Early Study Termination 
The Sponsor reserves the right to terminate the study for any reason. The study may be 
terminated early if the extent (incidence and/or severity) of emerging effects/clinical endpoints is 
such that the risk/benefit ratio to the study population as a whole is unacceptable. In addition, 
further recruitment in the study overall or at a particular study site may be stopped due to 
insufficient compliance with the protocol, Good Clinical Practice and/or other applicable 
regulatory requirements, procedure-related problems or the number of discontinuations for 
administrative reasons is too high.  


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8. 
Study Assessments and Procedures 
Study procedures and their scheduled timepoints are summarized in the Schedule of Assessments 
(Section 1.3). A detailed listing of study assessments by visit is provided in Section 10.7. Site 
personnel should note the following: 
• Protocol waivers or exemptions are not allowed. Any notable protocol deviations should 
be noted and raised to the Sponsor’s or designee’s attention. 
• Immediate safety concerns should be discussed with the Sponsor’s designated Medical 
Monitor immediately upon occurrence or awareness to determine if the subject should 
continue or discontinue study drug. 
• Adherence to the study design requirements, including those specified in the Schedule of 
Assessments, is essential and required for study conduct. 
• Note that patient-reported outcome (PRO) assessments will be completed prior to vital 
signs, and vital signs will be taken prior to blood draws.  
• Procedures conducted as part of the subject’s routine clinical management (eg, blood 
count) and obtained before signing of the ICF may be utilized for the purposes of 
confirming eligibility (in addition to the most recent available assessments from Study 
URO-901-3005).  
8.1. 
Study Entry Procedures 
As this is an extension study that includes subjects from the ongoing parent study, URO-
9013005, assessments performed at the end of the parent study will not be repeated on study 
entry into Study URO-901-3006. Specifically, vital sign, physical examination, laboratory, 
bladder diary, PVR urine, and PRO assessments performed at the Week 24 Visit of Study URO-
901-3005 will be considered the initial study assessments at entry into extension Study URO-
901-3006. 
In addition, medical history and demographic information from entry into Study URO-901-3005 
will be used as relevant to provide information for Study URO-901-3006.  
8.1.1. 
Informed Consent 
Documented consent (for Study URO-901-3006) must be obtained from each potential subject 
prior to the first dose of open-label study drug (vibegron) on Study URO-901-3006 according to 
the process described in Section 10.1.3.  


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8.1.2. 
Assignment of Subject Number 
All subjects who enroll in Study URO-901-3006 will retain the same study number that they 
were assigned in Study URO-901-3005.  
The investigator will maintain a log to confirm eligibility or record reasons for ineligibility, as 
applicable. Inclusion and exclusion criteria (Section 5.1 and Section 5.2) will be reviewed by the 
investigator or qualified designee at Week 24 of Study URO-901-3005). If needed, the Week 24 
laboratory results from Study URO-901-3005 may be used to confirm eligibility. 
8.1.3. 
Concomitant Medications 
Ongoing medications will be recorded beginning at the signing of the ICF and continuing until 
the last dose of study drug. 
8.2. 
Efficacy Assessments 
Efficacy assessments will be collected as outlined in the Schedule of Assessments (Section 1.3) 
in the form of 3-day Bladder Diary. Information collected in the diaries will be used for 
assessment of the efficacy endpoints related to the number of micturition, urgency, nocturia, and 
incontinence episodes per day as well as the volume voided per micturition. In addition, subjects 
will complete IPSS assessments and
Patient Global Impression (PGI), and
scores to assess quality of life parameters. 
Subjects will complete questionnaires at the site at the start of each required study visit (before 
vital signs and blood draws). The PRO questionnaires are provided in Section 10.8. Additional 
information on the Bladder Diary and questionnaires is provided below. 
8.2.1. 
Bladder Diaries 
Bladder Diary is used by subjects to record the frequency of daily OAB symptoms including all 
micturitions, urgency, incontinence, nocturia, one 24-hr volume voided of urine (used to derive 
the volume per micturition), and main reason for incontinence, and volume voided per 
micturition (over one 24-hour period) by selecting the respective box for each symptom 
occurring during the course of a given day and night. 
The Bladder Diary should be completed by the subject on 3 consecutive days within the 7 days 
prior to Visit 12 (Week 28), Visit 13 (Week 36) and Visit 14 (Week 52). A “Diary Day” is 
defined as the time between when the subject gets up for the day each morning (ie, the time the 
subject got up for the day yesterday to the time the subject got up for the day today; 
approximately a 24-hour period). Urine volume may be collected during any one (1) of the 3 
Diary Days prior to the visit. 


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At all study visits, the site staff should inquire whether the subject had any difficulties with the 
diary and address any questions subjects may have. Instructions for proper completion of the 
diary should be re-reviewed. Subjects will be trained to enter data immediately following each 
event (in real time) and to input data from any “missed” events as soon as they are able. They 
will review and confirm that data from all events occurring within the preceding Diary Day 
(approximately 24 hours) have been entered at a consistent time each morning (eg, upon getting 
up for the day). 
8.2.2. 
Patient-Reported Outcomes 
Subjects will complete questionnaires at the site at the start of each required study visit (before 
vital signs and blood draws) to assess subject-perceived symptom relief, symptom bother, and 
health-related quality of life. These include the following questionnaires: 
• The IPSS includes 8 questions (7 concerning urinary symptoms and 1 concerning quality 
of life), each with answers based on a 6-point scale indicating increasing severity. The 
urinary symptom responses are assigned points from 0 to 5.  The Total IPSS Score can 
therefore range from 0 to 35 (asymptomatic to very symptomatic). The quality of life 
responses are assigned points from 0 to 6. A sample of the IPSS is provided in 
Section 10.8.1. 
• 
 (1-week recall) is a multi-item questionnaire that was developed to assess 
symptom bother and the impact of 
 
The instrument was developed and validated in both continent and incontinent OAB 
patients, including both men and women. A sample of the
 scales is provided 
in Section 10.8.2. 
• The EQ-5D health questionnaire is a standardized instrument for use as a measure of 
health outcome [Rabin 2014]. It is applicable to a wide range of health conditions and 
treatments; it provides a simple descriptive profile and a single index value for health 
status. A sample of the EQ-5D health questionnaire is provided in Section 10.8.3. 
• Global Impression Items include 
 A sample of the PGI scale is provided in Section 10.8.4. 
• 
The investigator should not provide any additional information to subjects prior to completing 
the questionnaires that might influence responses. 


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8.3. 
Safety Assessments 
Planned timepoints for all safety assessments, including assessments for PVR, are provided in 
the Schedule of Assessments (Section 1.3). Immediate safety concerns should be discussed with 
the Sponsor’s designated Medical Monitor immediately upon occurrence or awareness to 
determine if the subject should continue or discontinue study drug. 
8.3.1. 
Vital Signs 
Vital signs, including blood pressure, heart rate (by pulse), body temperature, and weight, will be 
assessed at the timepoints specified in the Schedule of Activities and Section 10.7 as follows: 
• Blood pressure and pulse will be measured after the subject has been resting in a seated 
position for 5 minutes, after PRO assessments and before blood draws.  
• Blood pressure measurements will be taken on the same arm and by the same site staff 
throughout the study, if possible.  
• Sitting systolic and diastolic blood pressures will be determined by averaging 3 replicate 
measurements obtained approximately 1 to 2 minutes apart. The average of the 3 
measurements will be used for eligibility and safety assessments.  
• The same method for assessing temperature should be used at all visits for a particular 
subject. 
• Body weight will be measured with subjects in street clothing with jacket/coat and shoes 
removed, using the same scale throughout the study, if possible. 
8.3.2. 
Physical Examinations 
Brief physical examinations will include examination of the heart, lungs, abdomen, and visual 
pelvic examination. In addition, any organ system in which a previous abnormality was noted at 
Baseline or a subject has a complaint or AE will be examined.  
8.3.3. 
Post-Void Residual Volume 
The volume of urine that remains in the bladder after voiding (PVR) is an objective measurement 
that may serve as a proxy for impaired ability to void. The physician should assess subjects with 
an increase in PVR volume to determine whether an AE should be reported. (Refer to 
Section 8.4.6 for additional information on reporting AEs of urinary retention associated with 
increased PVR.) 
The PVR assessment will be performed via ultrasound at the visits indicated in the Schedule of 
Assessments (Section 1.3). All efforts will be made to ensure the same device and operator are 
used for all PVR volume measurements for individual subjects. 


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8.3.4. 
Clinical Safety Laboratory Assessments 
All protocol-required laboratory assessments must be conducted in accordance with the 
laboratory manual and the Schedule of Assessments. See Section 10.2 for the list of clinical 
laboratory tests to be performed and the Schedule of Assessments for the timing and frequency. 
Repeat or unscheduled samples may be taken for safety reasons or for technical issues with the 
samples. 
The clinical significance of test results will be evaluated as follows: 
• At Week 24 of Study URO-901-3005, the investigator or physician subinvestigator will 
assess the clinical significance of any values outside the reference ranges provided by the 
laboratory, and subjects with abnormalities judged to be clinically significant will be 
excluded from the study. 
• The investigator or physician subinvestigator must review the laboratory report, document 
this review, and record any clinically relevant changes occurring during the study in the AE 
section of the eCRF. The laboratory reports must be filed with the source documents. 
Laboratory abnormalities associated with the underlying disease are not considered clinically 
significant unless judged by the investigator to have worsened or be more severe than 
expected for the subject’s condition. 
• All laboratory tests with values considered clinically significant during participation in the 
study or within 21 days after the last dose of study drug should be repeated until the values 
return to normal or baseline or are no longer considered clinically significant by the 
investigator or the Sponsor’s designated Medical Monitor.  
- If such values do not return to normal/baseline within a period of time judged reasonable 
by the investigator, the etiology should be identified and the Sponsor’s designated 
Medical Monitor notified. 
- If laboratory values from nonprotocol specified laboratory assessments performed at the 
institution’s local laboratory require a change in subject management or are considered 
clinically significant by the investigator (eg, SAE or AE or dose modification), then the 
results must be recorded in the eCRF. 
8.4. 
Adverse Events and Serious Adverse Events 
The definitions of an AE or SAE can be found in Section 10.3. 
AEs will be reported by the subject (or, when appropriate, by a caregiver, surrogate, or the 
subject’s legally authorized representative). 
The investigator and any qualified designees are responsible for detecting, documenting, and 
recording events that meet the definition of an AE or SAE and remain responsible for following 
up AEs that are serious, considered related to the study drug or study procedures, or that caused 
the subject to discontinue the study drug or the study (see Section 7). 


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8.4.1. 
Time Period and Frequency for Collecting Adverse Event and Serious Adverse 
Event Information 
All AEs and SAEs will be collected from the signing of the informed consent form through the 
end of study.  In addition, any AEs and SAEs reported within 21 days after the last dose of study 
drug (5 half-lives of vibegron [half-life approximately 80 hours]) will also be collected. AEs will 
be collected at the timepoints specified in the Schedule of Assessments (Section 1.3), and as 
observed or reported spontaneously by study subjects. 
Medical occurrences that begin before the start of study drug, but after obtaining informed 
consent will be recorded in the AE section of the eCRF. 
All SAEs (including serious AESIs) will be recorded and reported on the eCRF within 24 hours 
of the study site personnel’s knowledge of the event, as indicated in Section 8.4.4. Marking the 
event as “serious” will automatically send required notifications for Sponsor or designee review. 
The investigator will also submit any updated SAE data within 24 hours of receipt of the 
information. Nonserious AESIs will be reported on the eCRF within 72 hours of the site’s 
knowledge of the information 
Investigators are not obligated to actively seek AE or SAE information after conclusion of the 
study participation. However, if the investigator learns of any SAE, including a death, at any 
time after a subject has been discharged from the study, and he/she considers the event to be 
reasonably related to the study drug or study participation, the investigator must promptly notify 
the Sponsor or designee. 
The method of recording, evaluating, and assessing causality of AEs and SAEs and the 
procedures for completing and transmitting SAE reports are provided in Section 10.3. 
8.4.2. 
Method of Detecting Adverse Events and Serious Adverse Events 
Care will be taken not to introduce bias when detecting AEs and SAEs. Open-ended and 
non-leading verbal questioning of the subject is the preferred method to inquire about AE 
occurrences. 
8.4.3. 
Follow-up of Adverse Events and Serious Adverse Events 
After the initial AE or SAE report, the investigator is required to proactively follow each subject 
at subsequent visits/contacts. All AEs will be followed until approximately 21 days after the last 
dose of study drug. All SAEs will be followed until resolution, stabilization, the event is 
otherwise explained, or the subject is lost to follow-up (as defined in Section 7.3). If a subject 
dies during participation in the study or within 21 days of the last dose of study drug, the 
investigator will provide the Sponsor or designee with a copy of any postmortem findings 
including histopathology. 


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The investigator is obligated to perform or arrange for the conduct of supplemental 
measurements and/or evaluations as medically indicated or as requested by the Sponsor or 
designee to elucidate the nature and/or causality of the AE or SAE as fully as possible. This may 
include additional laboratory tests or investigations, histopathological examinations, or 
consultation with other health care professionals. 
New or updated information will be recorded in the originally completed eCRF. As noted above, 
the investigator will submit any updated SAE data to the Sponsor or designee within 24 hours of 
receipt of the information. 
8.4.4. 
Regulatory Reporting Requirements for Serious Adverse Events 
• Prompt notification of an SAE by the investigator to the Sponsor or designee is essential so 
that legal obligations and ethical responsibilities towards the safety of subjects and the safety 
of a study drug under clinical investigation are met.  
• The Sponsor has a legal responsibility to notify both the local regulatory authority and other 
regulatory agencies about the safety of a study drug under clinical investigation. The Sponsor 
will comply with country-specific regulatory requirements relating to safety reporting to the 
regulatory authority, IRBs/IECs, and investigators.  
• Investigator safety reports must be prepared for suspected unexpected serious adverse 
reactions (SUSARs) according to local regulatory requirements and Sponsor policy and 
forwarded to investigators as necessary. 
• An investigator who receives an investigator safety report describing an SAE or other 
specific safety information (eg, summary or listing of SAEs) from the Sponsor or designee 
will review and then file it and will notify the IRB/IEC/REB, if appropriate according to 
local requirements. 
8.4.5. 
Disease-Related Events and/or Disease-Related Outcomes Not Qualifying as 
Adverse Events or Serious Adverse Events 
No disease-related events or outcomes are excluded from AE reporting. Any worsening of OAB 
or BPH symptoms or any events of bladder obstruction should be collected as AEs.  
8.4.6. 
Adverse Events of Special Interest 
Selected nonserious and SAEs will be reported as AESIs. AESIs that also meet the definition of 
an SAE must be reported as described in Section 10.3.  
Adverse events of special interest for this study include:  
• Adverse events consistent with urinary retention 
- An AE of urinary retention should only be recorded when a subject has a raised PVR 
that requires intervention with clean intermittent catherization (CIC) or temporary 
placement of a urinary catheter according to the following criteria: 
a) subject has a PVR of ≥ 300 mL (regardless of symptoms), OR 


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b) subject has a PVR ≥ 200 mL and < 300 mL and the subject reports associated 
symptoms, ie, sensation of bladder fullness or inability to void despite persistent 
effort, that in the investigator’s opinion require CIC. 
Note: An AE of residual urine volume should be recorded if, in the investigator’s 
opinion, the raised PVR is clinically significant but does not fulfill the above 
definition for urinary retention. As stated above in Section 7, subjects with a 
single PVR volume of ≥ 300 mL or an AE of urinary retention will be withdrawn 
from the study. 
• Adverse events suggestive of cystitis or urinary tract infection (UTI) 
- An AE of UTI will be recorded if both the following criteria are fulfilled, regardless 
of subject symptoms: 
 a) a positive urine culture result with a bacteriuria count of ≥ 105 CFU/mL 
 b) leukocyturia of ≥ 5/hpf 
If a subject meets the criteria for the definition of a UTI, the investigator will 
record whether the UTI was “symptomatic” or “asymptomatic” on the AE 
eCRF. 
Note: If urinalysis/culture results are reported which, in the opinion of the 
investigator, are considered clinically significant but do not fulfill the above 
definition of a UTI, the findings should be recorded as AEs (eg, bacteriuria, 
leukocyturia). 
• Potential major cardiac and cerebrovascular events, including death (or any event with fatal 
outcome), myocardial infarction, cerebrovascular accident, hospitalization for unstable 
angina or chest pain, hospitalization for heart failure requiring hospitalization, and coronary 
revascularization/angioplasty/stent 
• Hypertension, defined as follows: 
- For subjects with systolic blood pressure < 140 mmHg and diastolic blood pressure 
< 90 mmHg at the Study URO-901-3005 baseline assessment, the average of 3 
systolic blood pressures ≥ 140 mmHg or diastolic blood pressures ≥ 90 mmHg (or 
both) at any 2 consecutive visits after baseline 
- For subjects with systolic blood pressure ≥ 140 mmHg or diastolic blood pressure 
≥ 90 mmHg at Study URO-901-3005 baseline, an increase compared to baseline at 
2 consecutive visits in the average of 3 systolic blood pressures by ≥ 20 mmHg or 3 
diastolic blood pressures by ≥ 10 mmHg 
- Initiation or increase in dose of medication for treatment of hypertension in any 
subject 
• Adverse events consistent with orthostatic hypotension as confirmed by orthostatic vital 
signs 


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• Elevated serum aspartate aminotransferase (AST) or alanine aminotransferase (ALT) lab 
value requiring that study drug be temporarily withheld or permanently discontinued (see 
Section 8.4.6.1 and 8.4.6.2). To date, no concern regarding drug-induced liver toxicity has 
been identified; however, the Sponsor is monitoring laboratory data for a potential safety 
signal, consistent with Food and Drug Administration guidance [FDA Guidance, 2009]. 
• Neoplasms 
Serious AESIs and elevated liver enzymes or bilirubin requiring withholding of study drug (see 
Section 8.4.6.1) must be reported within 24 hours of the study site personnel’s knowledge of the 
event by marking the appropriate box on the AE eCRF and assigning the most appropriate 
category. Additional information should be provided as directed in the eCRF Completion 
Guidelines. AESIs that also meet the definition of an SAE must be reported as an SAE, as 
described in Section 8.4.4. Nonserious AESIs should be reported within 72 hours of the site 
personnel’s knowledge, using the AE eCRF. 
8.4.6.1. 
Criteria for Temporary Withholding of Study Treatment in Association with 
Liver Test Abnormalities 
Elevated liver enzymes or bilirubin sufficient to require withholding study medication must be 
reported within 24 hours of the study site personnel’s knowledge of the event using AESI 
specific eCRFs/forms/worksheets provided for the study.  
Hepatic enzymes will be monitored in accordance with FDA drug-induced liver injury guidelines 
[FDA, 2009].  
If any of the following liver test abnormalities develop, Study Treatment should be withheld 
immediately with appropriate clinical follow-up (including repeat laboratory tests, until a 
subject’s laboratory profile has returned to normal/baseline status), and the event reported as an 
SAE:  
• ALT or AST > 8 x upper limit of normal (ULN) 
• ALT or AST > 5 x ULN and persists for more than 2 weeks  
• ALT or AST > 3 x ULN and total bilirubin > 2 x ULN or international normalized ratio 
(INR) > 1.5 
• ALT or AST > 3 x ULN with appearance of fatigue, nausea, vomiting, right upper 
quadrant pain or tenderness, fever, rash and/or eosinophilia (> 5%)  
Rechallenge may be considered if an alternative cause for the abnormal liver tests (ALT, AST, 
total bilirubin) is discovered and the laboratory abnormalities resolve to normal or baseline 
values. The investigator and Sponsor’s designated Medical Monitor must discuss and agree with 
any decision to rechallenge.  
Rechallenge should not occur when the etiology of the liver test abnormalities is considered 
possibly drug induced. 


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8.4.6.2. 
Criteria for Permanent Discontinuation of Study Treatment in Association with 
Liver Test Abnormalities 
Study treatment should be discontinued permanently if all of the following 4 criteria are met (ie, 
potential severe drug-induced liver injury/Hy’s law case):  
• Total bilirubin increases to > 2 x ULN or INR > 1.5 and  
• AST or ALT increases to ≥ 3 x ULN and  
• Alkaline phosphatase value does not reach 2 x ULN and  
• No alternative cause explains the combination of the above laboratory abnormalities; 
important alternative causes include, but are not limited to the following:  
- Hepatobiliary tract disease  
- Viral hepatitis (eg, hepatitis A/B/C/D/E, Epstein-Barr virus)  
- Exposure to hepatotoxic agents/drugs or hepatotoxins, including herbal and dietary 
supplements, plants, and mushrooms  
- Alcoholic hepatitis  
- Non-alcoholic steatohepatitis  
- Autoimmune hepatitis  
If an alternative cause for hepatotoxicity is identified, then it should be determined (based on the 
severity of the hepatotoxicity or event) whether study drug should be withheld or permanently 
discontinued as appropriate for the safety of the subject, following consultation with the 
Sponsor’s designated Medical Monitor. 
8.4.7. 
Pregnancy Management and Reporting to the Sponsor 
In the event that a partner of a male study subject becomes pregnant during the study, if the 
subject agrees, the subject’s pregnant partner should be notified of the subject’s study 
participation and be requested to sign a Release of Information form, permitting transfer of 
information regarding the pregnancy and outcome to the Sponsor. If the subject and the subject’s 
partner agree, the investigator should notify the partner’s primary care physician and provide 
details of the subject’s participation in the study. 
Partner pregnancies are to be reported to the Sponsor within 24 hours of awareness by the study 
site personnel, using the pregnancy reporting forms and information for safety event reporting in 
Section 10.3. The expected date of delivery or expected date of the end of the pregnancy, last 
menstruation, estimated conception date, pregnancy result, neonatal data, etc. should be included 
in this information, as available. 
If the subject and partner agree, the investigator will follow the medical status of the mother, the 
pregnancy, as well as the outcome of the infant at birth, and will report the outcome to the 
Sponsor. 


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8.4.8. 
Medication Errors 
Medication error refers to any unintended error in the dosing and/or administration of the study 
drug as per instructions in the protocol. Medication errors generally fall into 4 categories as 
follows: 
• Wrong study drug 
• Wrong dose (including dosing regimen, strength, form, concentration, amount) 
• Wrong route of administration 
• Wrong subject (ie, not administered to the intended subject) 
Medication errors include occurrences of overdose and underdose of the study drug. 
Overdose: Unintentional administration of a quantity of the study drug given per administration 
or per day that is above the maximum recommended dose according to the protocol. This also 
takes into account cumulative effects due to overdose (see Section 8.5 for treatment and 
reporting of overdose). For this study, any dose of vibegron of 3 or more tablets within a 24-hour 
window is an overdose. There is no known antidote for an overdose.  
Underdose: No underdose is defined for this study. 
8.5. 
Treatment of Overdose 
In the event of an overdose (3 or more tablets of study drug within 24 hours), the investigator or 
treating physician should:  
• Contact the Sponsor’s designated Medical Monitor immediately  
• Closely monitor the subject for any AEs, SAEs, and laboratory abnormalities  
• Report all overdose events on the eCRF, whether or not the overdose is associated with 
an AE. If the overdose resulted in an AE or SAE, refer to Section 10.3 for reporting 
requirements.  
• Document the quantity of the excess dose as well as the duration of the overdosing in the 
eCRF.  
Decisions regarding dose interruptions or modifications will be made by the investigator in 
consultation with the Sponsor’s designated Medical Monitor based on the clinical evaluation of 
the subject. 


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9. 
Statistical Considerations 
This section contains a brief summary of the statistical analyses for this study; full details will be 
provided in the Statistical Analysis Plan (SAP).  
9.1. 
Hypotheses 
9.1.1  
Primary Objective and Hypothesis 
The primary study objective is to demonstrate long-term safety of vibegron 75 mg in men with 
BPH with symptoms of OAB. There is no formal statistical primary endpoint hypothesis. 
Descriptive statistics will be used to evaluate safety endpoints, including the incidence of 
treatment-emergent AEs (SOC and PT) by originally randomized treatment group and for 
vibegron treatment overall, including events during all vibegron exposure from both Study URO-
901-3005 and URO-301-3006. 
9.1.2  
Secondary Objectives and Hypotheses 
There are no formal statistical hypotheses for this trial. The secondary study objective is to 
demonstrate long-term efficacy of vibegron 75 mg in men with BPH with symptoms of OAB.  
9.2. 
Analysis Endpoints 
The description of the endpoints and study time points at which each is measured are described 
in Section 3 and Section 1.3, respectively. 
9.2.1. 
Efficacy and Health Outcome Endpoints 
For purposes of this study, the number of micturitions will be defined as the number of times a 
subject has voided in the toilet as indicated on the Bladder Diary. Average daily micturitions are 
calculated using the daily entries in the Bladder Diary, which is completed prior to each study 
visit. Average daily number of micturitions will be calculated as the total number of micturitions 
that occur on a Diary Day divided by the number of Diary Days in the Bladder Diary. Baseline 
will be the same baseline determined from the URO-901-3005 trial. 
An urgency episode is defined as the “Need to Urinate Immediately” as indicated on the Bladder 
Diary. Average daily urgency episodes at each study visit will be calculated in the same manner 
as described above for the micturitions.  
A UUI episode is defined as having "urge" as the main reason for the leakage as indicated on the 
Bladder Diary, regardless of whether more than one reason for leakage in addition to “urge” is 
checked.  
A nocturia episode is defined as waking to pass urine during the main sleep period as indicated 
on the Bladder diary.  


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Efficacy Endpoints: 
• CFB at Week 52 in the average number of micturition episodes per day 
• CFB at Week 52 in the average number of urgency episodes (urgency: need to urinate 
immediately) per day 
• CFB at Week 52 in the average number of nocturia episodes per night  
• CFB at Week 52 in the average number of UUI episodes per day in subjects with 
incontinence at Study URO-901-3005 baseline  
• CFB at Week 52 in the average of IPSS Storage score (1-week recall) 
• CFB at the Week 52 in the average volume voided per micturition 
Other/Exploratory – Efficacy 
Other/Exploratory – Quality of Life 


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9.2.2. 
Safety Endpoints 
Safety will be assessed via clinical review of all relevant safety parameters including AEs, 
clinical laboratory, vital sign assessments, PVR volume, and Total IPSS. 
9.3. 
Analysis Populations 
9.3.1. 
Efficacy Analysis Populations 
The Full Analysis Set Extension (FAS-Ext) population will serve as the population for the 
analysis of efficacy data in this study. Since the endpoints related to incontinence would only 
apply to subjects who meet the definition of incontinence at study entry, it is necessary to have a 
separate FAS definition with the additional criterion to define the analysis population for 
incontinence endpoints. 
The following FAS populations are defined in this study: 
• Full Analysis Set Extension (FAS-Ext): All OAB subjects who took at least one dose of 
vibegron in the extension study and have at least one evaluable change from baseline 
micturition measurement in this study. 
• Full Analysis Set Extension for incontinence (FAS-Ext-I): All OAB subjects who took at 
least one dose of vibegron in the extension study, were included in the FAS-I population 
in the URO-901-3005 study, and have at least one evaluable change from baseline (ie, 
Study URO-901-3005 baseline) urge urinary incontinence measurement. 
Per-Protocol Extension population (PP-Ext) and Per-Protocol extension population for 
incontinence (PP-Ext-I) exclude subjects due to important deviations from the protocol that may 
substantially affect the results of the efficacy endpoints. A supportive analysis using the Per 
Protocol populations will be performed for 6 efficacy endpoints as defined in Section 3 (ie, CFB 
in micturition, urgency, nocturia, and urinary incontinence episodes; IPSS Storage score; and 


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volume voided per micturition). The final determination of protocol violations, and thereby the 
composition of the Per-Protocol populations will be made prior to database lock and will be 
documented. 
Using the Full Analysis Set and Per-Protocol populations, subjects will be included in one of two 
vibegron treatment groups based on the original randomization from the parent study (URO-901-
3005): vibegron for 52 weeks or vibegron for 28 weeks. Efficacy endpoints will be descriptively 
summarized by treatment group. 
9.3.2. 
Safety Analysis Populations 
The Safety Analysis Set - Extension (SAF-Ext) population will be used to for the analysis of 
safety data in this study.   
The SAF-Ext consists of all randomized subjects in Study URO-901-3005 who  
a) have completed the 24-week treatment period in the Study URO-901-3005, and  
b) enrolled into the extension Study URO-901-3006 and received at least one dose of 
vibegron in this study.   
The SAF-Ext will be used for all the safety analyses. Safety endpoints will be descriptively 
summarized based on the treatment in study URO-901-3006. This means there will be one 
vibegron treatment group (combining 52 weeks of vibegron dosing for those continuing vibegron 
from Study URO-901-3005 and 28 weeks of vibegron dosing for those who received placebo in 
Study URO-901-3005).  
No imputation will be performed for missing safety data. Baseline will be the same as the 
baseline defined in the parent study. Further inclusion of data from URO-901-3005 in the 
reporting of this study will be described in the SAP.  
9.4. 
Statistical Analyses 
9.4.1. 
Statistical Methods for Efficacy Analysis 
Statistical analysis of efficacy endpoints will be for descriptive purposes only. Baseline will be 
defined as the baseline from the Study URO-901-3005. Further inclusion of data from URO-901-
3005 in the reporting of this study will be described in the SAP. 
For the analysis of continuous change from baseline endpoints (eg, change from baseline in 
average number of micturitions, change from baseline in the average number of urgency 
episodes, change from baseline in average number of nocturia episodes, change from baseline in 
daily urge urinary incontinence episodes, change from baseline in average IPSS Storage score, 
and change from baseline in average volume voided per micturitions), a mixed model for 
repeated measure (MMRM) with restricted maximum likelihood estimation will be used. This 
model corrects for dropout and accounts for the fact that measurements taken on the same 
subject over time tend to be correlated, by using all available information on subjects within the 
same covariate set to derive an estimate of the treatment effect for a dropout-free population.  


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The analysis model for each efficacy endpoint will include terms for visit, baseline, stratification 
factors (only those that were statistically significant in URO-901-3005 will be included in the 
models), and baseline score. Only subjects on active treatment in both URO-901-3005 and URO-
901-3006 will be included in the model. Adjusted means and 95% confidence intervals will be 
presented for each visit.   
An unstructured covariance matrix will be used to model the correlation among repeated 
measurements. The Kenward-Roger adjustment will be used with restricted (or residual 
maximum likelihood (REML) to make proper statistical inference. If the unstructured covariance 
model fails to converge with the default Newton-Raphson algorithm, the Fisher scoring 
algorithm or other appropriate methods can be used to provide initial values of the covariance 
parameters. In the rare event that none of the above methods yield convergence, a structured 
covariance will be used to model the correlation among repeated measurements. 
In general, continuous variables will be summarized by treatment to indicate the population 
sample size (N), number of subjects with available data (n), arithmetic mean, SD, median, Q1, 
Q3, minimum, and maximum values. Categorical variables will be summarized by N, n, number 
of subjects in each category, and the percentage of subjects in each category. Unless otherwise 
noted, the denominator to determine the percentage of subjects in each category will be based on 
the number of subjects with available data. 
9.4.2. 
Safety Analyses 
Safety analysis will be conducted using the SAF-Ext population and summarized by the 
Vibegron treatment group.  The treatment-emergent period will be defined as the period of time 
from the first dose date of vibegron in this study through 21 days after the last dose of treatment, 
or the date of initiation of another investigational agent or surgical intervention, whichever 
occurs first.  Safety will be assessed through summaries of adverse events, the frequency of 
treatment discontinuation due to adverse events, and clinical laboratory evaluations. 
AEs will be coded using the latest version of MedDRA (20.0 or higher). The severity of all 
adverse events will be evaluated by the Investigator. The incidence of adverse events will be 
presented by system organ class and preferred term, relationship to study treatment and severity.  
Laboratory data will consist of chemistry, hematology and urinalysis data. Only data collected by 
the central laboratory will be included in the analysis.  
Descriptive statistics of observed values and change from baseline for vital signs and post void 
residual urine volume will be listed and summarized. 
9.5. 
Multiplicity 
All efficacy endpoints will be considered descriptive and no multiplicity adjustments will be 
performed for these endpoints. 


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9.6. 
Subgroup Analyses 
Subgroup analyses of endpoints may be conducted. Additional details of any planned subgroup 
analyses will be provided in the SAP. 
9.7. 
Sample Size Determination 
Approximately 300 subjects rolling over from Study URO-901-3005, in addition to other long-
term safety data with vibegron, is sufficient to characterize the long-term safety profile of 
vibegron 75 mg once daily men with symptoms of OAB on stable doses of pharmacological 
therapy for BPH. With the assumption of approximately 33% dropout, 100 subjects will be 
expected to complete the study for the duration of 1-year on vibegron.  
9.8. 
Interim Analyses 
There are no interim analyses planned for this study.  
9.9. 
Data Safety Monitoring Board 
One external independent DSMB will be formed for Studies URO-901-3005 and URO-901-
3006. The detailed activities including meeting and analysis plan will be described and 
documented in the DSMB charter and the DSMB SAP, respectively. 


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10. 
Supporting Documentation and Operational Considerations 


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10.1. 
Appendix 1: Regulatory, Ethical, and Study Oversight 
Considerations 
10.1.1. 
Regulatory and Ethical Considerations 
• This study will be conducted in accordance with the protocol and with the following: 
- Consensus ethical principles derived from international guidelines including the 
Declaration of Helsinki and Council for International Organizations of Medical Sciences 
(CIOMS) International Ethical Guidelines 
- Applicable ICH/ISO Good Clinical Practice (GCP) guidelines 
- Applicable laws and regulations 
• The protocol, protocol amendments, ICF, Investigator’s Brochure, and other relevant 
documents (eg, advertisements) must be submitted to an IRB/IEC/REB by the investigator 
and reviewed and approved by the IRB/IEC/REB before the study is initiated. 
• Any amendments to the protocol will require IRB/IEC/REB approval before implementation 
of changes made to the study design, except for changes necessary to eliminate an immediate 
hazard to study subjects.  
• The investigator will be responsible for the following: 
- Providing written summaries of the status of the study to the IRB/IEC/REB annually or 
more frequently in accordance with the requirements, policies, and procedures 
established by the IRB/IEC/REB 
- Notifying the IRB/IEC/REB of SAEs or other significant safety findings as required by 
IRB/IEC/REB procedures 
- Providing oversight of the overall conduct of the study at the site and adherence to 
requirements of applicable local regulations, for example 21 Code of Federal Regulations 
(CFR), ICH guidelines, the IRB/IEC/REB, and European regulation 536/2014 for clinical 
studies (if applicable) 
10.1.2. 
Financial Disclosure 
Investigators and subinvestigators will provide the Sponsor or designee with sufficient, accurate 
financial information as requested to allow the Sponsor to submit complete and accurate 
financial certification or disclosure statements to the appropriate regulatory authorities. 
Investigators are responsible for providing information on financial interests during the course of 
the study and for 1 year after completion of the study. 


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10.1.3. 
Informed Consent Process 
• The investigator or his/her representative will explain the nature of the study to the subject or 
his legally authorized representative and answer all questions regarding the study.  
• Subjects must be informed that their participation is voluntary. Subjects or their legally 
authorized representatives will be required to sign a statement of informed consent that meets 
the requirements of 21 CFR 50, local regulations, ICH guidelines, HIPAA requirements, 
where applicable, and the IRB/IEC/REB or study center.  
• The medical record must include a statement that written informed consent was obtained 
before the subject was enrolled in the study and the date the written consent was obtained. 
The authorized person obtaining the informed consent must also sign the ICF. 
• Subjects must be re-consented to the most current version of the ICF(s) during their 
participation in the study.  
• A copy of the ICF(s) must be provided to the subject or the subject’s legally authorized 
representative.  
10.1.4. 
Data Protection 
• Subjects will be assigned a unique identifier. Any subject records or datasets that are 
transferred to the Sponsor or designee will contain the identifier only; subject names or any 
information which would make the subject identifiable will not be transferred.  
• The subject must be informed that his personal study-related data will be used by the Sponsor 
or designee in accordance with local data protection law. The level of disclosure must also be 
explained to the subject.  
• The subject must be informed that his medical records may be examined by Clinical Quality 
Assurance auditors or other authorized personnel appointed by the Sponsor or designee, by 
appropriate IRB/IEC/REB members, and by inspectors from regulatory authorities. 
10.1.5. 
Committees Structure 
Not applicable. 
10.1.6. 
Posting Clinical Study Data 
Clinical study information will be posted on external registries and websites (eg, US National 
Institutes of Health’s website www.ClinicalTrials.gov and European Clinical Trial Register) as 
per applicable regulatory requirements. 
10.1.7. 
Data Quality Assurance 
• All subject data relating to the study will be recorded on case report forms (CRFs) unless 
transmitted to the Sponsor or designee electronically (eg, laboratory data). The investigator is 
responsible for verifying that data entries are accurate and correct by physically or 
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• The investigator must maintain accurate documentation (source data) that supports the 
information entered in the CRF.  
• The investigator must permit study-related monitoring, audits, IRB/IEC/REB review, and 
regulatory agency inspections and provide direct access to source data documents.  
• The Sponsor is responsible for the data management of this study including quality checking 
of the data. Management of clinical data will be performed in accordance with applicable 
Sponsor-approved standards and data cleaning procedures to ensure the integrity of the data 
• Study monitors will perform ongoing source data verification to confirm that data entered 
into the eCRF by authorized site personnel are accurate, complete, and verifiable from source 
documents; that the safety and rights of subjects are being protected; and that the study is 
being conducted in accordance with the currently approved protocol and any other study 
agreements, ICH GCP, and all applicable regulatory requirements.  
Study Site 
All clinical study documentation must be retained by the investigator until at least 2 years after 
the last approval of a marketing application in an ICH region (ie, United States, Europe, or 
Japan) and until there are no pending or contemplated marketing applications in an ICH region; 
or, if no application is filed or if the application is not approved for such indication, until 2 years 
after the investigation is discontinued and regulatory authorities have been notified. Investigators 
may be required to retain documents longer if required by applicable regulatory requirements, by 
local regulations, or by an agreement with the Sponsor. The investigator must notify the Sponsor 
before destroying any clinical study records. 
The study site and the record retainer should take measures in such a way that these records are 
not lost or abandoned during the designated period of preservation and that they are presented 
upon request. 
Institutional Review Board, Independent Ethics Committee, and Research Ethics Board 
(IRB/IEC/REB) 
The protocol, protocol amendments, informed consent form, Investigator’s Brochure, and any 
other relevant materials, including accompanying material to be provided to the subject (eg, 
advertisements, patient information sheets, or descriptions of the study used to obtain informed 
consent) will be submitted by the investigator to an IRB/REB/IEC. Approval from the 
IRB/REB/IEC must be obtained before starting the study and should be documented in a letter to 
the investigator specifying the following: 
• Protocol number 
• Protocol version 
• Protocol date 
• Documents reviewed  
• Date on which the committee met and granted the approval 


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Any amendments to the protocol will require IRB/REB/IEC approval before implementation of 
changes made to the study design, except for changes necessary to eliminate an immediate 
hazard to study participants. 
The investigator will be responsible for the following: 
• Providing written summaries of the status of the study to the IRB/REB/IEC’s annually or 
more frequently in accordance with the requirements, policies, and procedures 
established by the IRB/REB/IEC 
• Notifying the IRB/REB/IEC of SAEs or other significant safety findings as required by 
procedures established by the IRB/REB/IEC 
10.1.8. 
Source Documents 
• Source documents provide evidence for the existence of the subject and substantiate the 
integrity of the data collected. Source documents are filed at the investigator’s site. 
• Data entered in the eCRF that are transcribed from source documents must be consistent with 
the source documents or the discrepancies must be explained. The investigator may need to 
request previous medical records or transfer records, depending on the study. Current 
medical records must be available during the site monitor’s visit. 
• The required source documents are: 
− Subject identification (name, date of birth, sex) 
− Documentation that the subject meets eligibility criteria, (eg, history, physical 
examination, and confirmation of diagnosis to support inclusion and exclusion 
criteria) 
− Participation in the study (including study number) 
− Study discussed and date of informed consent 
− Dates of all visits 
− Documentation that protocol-specific procedures were performed 
− Results of efficacy parameters, as required by the protocol 
− Start and end date (including dose regimen) of Study Treatment (drug dispensing 
and return should be documented as well) 
− Record of all AEs and other safety parameters (start and end date, and causality 
and intensity as assigned by the investigator) 
− Concomitant medication (including start and end date) 
− Date of study completion and reason for early discontinuation, if applicable 


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10.1.9. 
Study and Site Closure 
The Sponsor designee reserves the right to close the study site or terminate the study at any time 
for any reason at the sole discretion of the Sponsor. Study sites will be closed upon study 
completion. A study site is considered closed when all required documents and study supplies 
have been collected and a study-site closure visit has been performed. 
The investigator may initiate study-site closure at any time, provided there is reasonable cause 
and sufficient notice is given in advance of the intended termination. 
Reasons for the early closure of a study site by the Sponsor or investigator may include but are 
not limited to: 
• Failure of the investigator to comply with the protocol, the requirements of the IRB/IEC/REB 
or local health authorities, the Sponsor’s procedures, or GCP guidelines 
• Inadequate recruitment of subjects by the investigator 
• Discontinuation of further study drug development 
10.1.10. Publication Policy 
• The Sponsor has proprietary interest in this study. Authorship and manuscript composition 
will reflect joint cooperation between multiple investigators and sites and Sponsor or 
designee personnel. Authorship will be established prior to the writing of the manuscript. As 
this study involves multiple centers, no individual publications will be allowed prior to 
completion of the final report of the multicenter study except as agreed with the Sponsor. 
• The Sponsor will comply with the requirements for publication of study results. In 
accordance with standard editorial and ethical practice, the Sponsor will generally support 
publication of multicenter studies only in their entirety and not as individual site data. 
10.1.11. Compliance with Protocol 
The investigator is responsible for compliance with the protocol at the investigational site. A 
representative of the Sponsor will make frequent contact with the investigator and his/her 
research staff and will conduct regular monitoring visits at the site to review subject and study 
drug accountability records for compliance with the protocol. Protocol deviations will be 
discussed with the investigator upon identification. The use of the data collected for the subject 
will be discussed to determine if the data are to be included in the analysis. The investigator will 
enter data that may be excluded from analysis as defined by the protocol deviation specifications. 
Significant protocol deviations will be reported to the IRB/IEC/REB according to the 
IRB/IEC/REB’s reporting requirements. 


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10.2. 
Appendix 2: Clinical Laboratory Tests 
The tests detailed in Table 10-1 will be performed by the central laboratory chosen by the 
Sponsor. Subjects do not need to fast prior to laboratory testing. A sample for urinalysis 
(including microscopy for RBCs, WBCs, epithelial cells, and bacteria) and urine culture will be 
sent to the central laboratory only if the urine dipstick tests positive for the presence of 
leukocytes, nitrites, or blood cells. If a subject reports symptoms suggestive of a urinary tract 
infection at any visit, a urine dipstick should be performed as needed, and a sample will be sent 
out for urinalysis and culture and sensitivity testing.  
Table 10-1  
Clinical Laboratory Tests 
Hematology 
Chemistry 
Urinalysisa 
Other 
Hematocrit 
Albumin 
Blood 
Coagulation 
INR/PT/APTTd  
Hemoglobin 
Alkaline phosphatase 
Glucose 
 
Platelet count 
ALT 
Protein 
 
WBC (total and 
differential) 
AST 
Specific gravity 
 
RBC 
Bicarbonate 
Microscopic exam 
(RBCs, WBCs, 
epithelial cells and 
bacteria) 
 
 
Calcium 
pH 
 
 
Chloride 
Color 
 
 
Creatineb 
 
 
 
Glucose (fasting or 
non-fasting) 
 
 
 
Potassium 
 
 
 
Sodium 
 
 
 
Total bilirubin 
 
 
 
Direct bilirubinc 
 
 
 
Blood urea nitrogen 
 
 
 
Total cholesterol 
 
 
ALT = alanine aminotransferase; APTT = activated partial thromboplastin time; AST = aspartate aminotransferase; 
eGRF = estimated glomerular filtration rate; INR = international normalized ratio; PSA = prostate-specific antigen; PT = 
prothrombin time; RBC = red blood cell count; WBC = white blood cell count  
a 
A sample for urinalysis and urine culture/sensitivity will be sent to the central laboratory only if the urine dipstick 
performed at the site is positive for the presence of leukocytes, nitrites, or blood cells.  
b 
eGFR will be calculated and reported by the central lab.  
c 
If total bilirubin is elevated above the upper limit of normal. 
d 
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10.4. 
Appendix 4: Abbreviations 
Term 
Description 
β3-AR 
beta-3 adrenergic receptor 
5-ARI 
5α-reductase inhibitors 
AE 
adverse event 
AESI 
Adverse Events of Special Interest 
ALT 
alanine aminotransferase 
APR 
abdominoperineal resection 
AR 
adrenergic receptor 
AST 
aspartate aminotransferase 
BPH 
benign prostatic hypertrophy 
cAMP 
cyclic adenosine monophosphate 
CFB 
change from baseline 
CFR 
Code of Federal Regulations 
CFU 
colony-forming unit 
CIC 
clean intermittent catheterization 
CIOMS 
Council for International Organizations of Medical Sciences 
CONSORT 
Consolidated Standards of Reporting Trials 
CRF 
case report form 
CT 
computerized tomography 
DSMB 
Data Safety Monitoring Board 
ECG 
electrocardiogram 
eCRF 
electronic case report form 
ED 
erectile dysfunction 
eGFR 
estimated glomerular filtration rate 
 
 
FAS 
full analysis set 
FAS-I 
full analysis set for incontinence 
FDA 
(United States) Food and Drug Administration 
GCP 
good clinical practice 
 
 
ICH 
International C on Harmonisation of Technical Requirements for 
Registration of Pharmaceuticals for Human Use 
ICF 
informed consent form 
ICS 
International Continence Society 


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Term 
Description 
IDC 
involuntary detrusor contraction 
IEC 
independent ethics committee 
 
 
INR 
international normalized ratio 
IPSS 
International Prostate Symptom Score 
IRB 
institutional review board 
IWRS 
interactive web response system 
LAR 
low anterior resection 
LF 
long form 
LUTS 
lower urinary tract symptoms 
MedDRA 
Medical Dictionary for Regulatory Activities 
MMRM 
mixed model for repeated measure 
MRI 
magnetic resonance imaging 
OAB 
overactive bladder 
 
 
PGI 
Patient Global Impression 
PRO 
patient-reported outcome 
PSA 
prostate-specific antigen 
PT 
preferred term 
PTNS 
percutaneous tibial nerve stimulation 
PVR 
post-void residual 
QD 
once daily 
Qmax 
maximum urinary flow; peak flow rate during voiding 
QTc 
corrected QT 
RBC 
red blood cell 
REB 
research ethics board  
REML 
restricted (or residual) maximum likelihood 
SAE 
serious adverse event 
SAP 
statistical analysis plan 
SBP 
systolic blood pressure 
SNS 
sacral neurostimulation 
SOC 
System Organ Class  
SOP 
standard operating procedure 
SUSAR 
suspected unexpected serious adverse reaction 


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Term 
Description 
TEAE 
treatment-emergent adverse event 
TESAE 
treatment-emergent serious adverse event 
ULN 
upper limit of normal 
Urovant 
Urovant Sciences GmbH 
US 
United States 
UTI 
urinary tract infection 
UUI 
urge urinary incontinence 
WBC 
white blood cell 
WHO-DDE 
World Health Organization Drug Dictionary Enhanced 
 


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10.5. 
Appendix 6: Study Tabular Summary 
 
Parameter 
Group 
Parameter 
Value 
Trial information 
Trial Title 
A Phase 3 Open-Label Extension 
Study to Evaluate the Long-Term 
Safety and Efficacy of Vibegron in 
Men with Overactive Bladder (OAB) 
Symptoms on Pharmacological 
Therapy for Benign Prostatic 
Hyperplasia (BPH) 
 
Clinical Study Sponsor 
Urovant Sciences, GmbH 
 
Trial Phase Classification 
Phase 3 
 
Trial Indication 
OAB Symptoms in Men with BPH 
 
Trial Indication Type 
Treatment 
 
Trial Type 
Long-term extension 
 
Trial Length 
Up to 28 weeks 
 
Planned Country of Investigational Sites 
North America and Europe 
 
Planned Number of Subjects 
Approximately 300 
 
FDA-Regulated Device Study 
No 
 
FDA-Regulated Drug Study 
Yes 
 
Pediatric Study 
No 
Subject 
information 
Diagnosis Group 
OAB Symptoms with BPH 
Healthy Subject Indicator 
No 
 
Planned Minimum Age of Subjects 
45 
 
Planned Maximum Age of Subjects 
None 
 
Sex of Subjects 
Male 
 
Stable Disease Minimum Duration 
3 months 


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Parameter 
Group 
Parameter 
Value 
Treatments 
Investigational Therapy or Treatment 
vibegron 
 
Drug Type 
drug 
 
Pharmacological Class of Invest. Therapy 
beta-3 adrenergic receptor agonist 
 
Dose per Administration 
75 
 
Dose Units 
mg 
 
Dosing Frequency 
Twice daily 
 
Route of Administration 
Oral 
 
Current Therapy or Treatment 
Pharmacotherapy for BPH: alpha 
blocker monotherapy or alpha blocker 
+ 5-ARI 
 
Added on to Existing Treatments 
Subjects not receiving current 
treatments for OAB symptoms 
 
Control Type 
Not applicable 
 
Comparative Treatment Name 
Not applicable 
Trial design 
Study Type 
Long-term extension 
 
Drug Model 
Single-arm, open-label 
 
Planned Number of Arms 
1 
 
Trial is Randomized 
No 
 
Randomization Quotient 
NA 
 
Trial Blinding Schema 
Open-label 
 
Stratification Factor 
From Parent Study URO-901-3005: 
Baseline average micturition episodes 
per day (≤ 12 vs > 12), alpha blocker 
use with or without 5-ARI (yes or no), 
and urinary incontinence (yes or no) 
 
Adaptive Design 
No 
BPH = benign prostatic hypertrophy; OAB = overactive bladder 
 


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10.6. 
Appendix 7: Prohibited Medications and Non-Drug Therapies 
The following medications are prohibited, as outlined in Section 6.4.1. 
Prohibited Medications Class 
Examples  
Comments  
Anticholinergics  
darifenacin, fesoterodine, 
hyoscyamine, oxybutynin, 
propantheline, solifenacin, 
tolterodine, and trospium  
Subject must remain off this therapy 
during the study. If an 
anticholinergic/ antimuscarinic is 
used in an inhaler on a PRN/ as 
needed basis for the treatment of 
chronic obstructive pulmonary 
disease, it will be permitted.  
Smooth muscle relaxants  
flavoxate, dicyclomine, 
propiverine  
Subject must remain off this therapy 
during the study  
Beta-2 adrenergic agonists used 
for the treatment of stress urinary 
incontinence  
clenbuterol  
Subject must remain off this therapy 
during the study  
Systemic beta-2 adrenergic 
agonist  
terbutaline  
No washout period; subject must 
remain off this therapy during the 
study  
Antidiuretic hormones  
desmopressin  
Subject must remain off this therapy 
during the study  
Beta-3 adrenergic agonists  
mirabegron  
Subject must remain off this therapy 
during the study  
New start of BPH medications  
Alpha-Blockers, eg, tamsulosin, 
doxazosin, and alfuzosin 
5-ARI, eg, Finasteride, and 
dutasteride 
PDE5 inhibitors, eg, tadalafil  
Subject must not start new use of 
these therapies during the study.  
Intradetrusor or intraprostatic 
botulinum toxins  
Intradetrusor injection of 
botulinum toxin or intraprostatic 
injections   
Subject must not receive this therapy 
during the study  
Any herbal medications to treat 
OAB symptoms, lower urinary 
tract symptoms of BPH or erectile 
dysfunction 
 
Subject must not receive them during 
the study. 
Diuretics 
 
Subject must not start new diuretics 
during the study. 
Percutaneous tibial nerve 
stimulation (PTNS) 
 
Subject must not start new PTNS or 
related therapies during the study. 
 
 


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10.7. 
Appendix 12: Study Schedule Supplement 
10.7.1. 
Study Entry, Visit 11 (Week 24 [Day 169 ± 4 days])  
• Obtain Informed Consent 
• Assess inclusion/exclusion criteria 
• Enter subject in IWRS 
• Dispense open-label study drug 
Note: The below (*) assessments performed at Week 24 of Study URO-901-3005 will be used 
for the initial procedures for Study URO-901-3006; they do not need to be repeated. 
* Review AEs/SAEs 
* Review concomitant medications 
* Administer IPSS,
 PGI,
scales 
* Record blood pressure (3 results taken 1-2 minutes apart after sitting for 5 minutes), 
pulse, respiration, temperature, and weight 
* Perform brief physical examination 
* Measure post-void residual volume 
* Perform laboratory assessments 
* Review and data enter completed Bladder Diary 
10.7.2. 
Open-label Treatment Period, Visit 12 (Week 28 [Day 197 ± 4 days])  
• Review AEs/SAEs 
• Review concomitant medications 
• Record blood pressure (3 results taken 1-2 minutes apart after sitting for 5 minutes), 
pulse, respiration, temperature, and weight 
• Measure post-void residual volume 
• Review and data enter completed Bladder Diary 
• Dispense Bladder Diary 
• Dispense open-label study drug  


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10.7.3. 
Open-label Treatment Period, Visit 13 (Week 36 [Day 253 ± 4 days]) 
• Review AEs/SAEs 
• Review concomitant medications 
• Administer IPSS and 
 assessments  
• Record blood pressure (3 results taken 1-2 minutes apart after sitting for 5 minutes), 
pulse, respiration, temperature, and weight 
• Measure post-void residual volume 
• Perform laboratory assessments 
• Review and data enter completed Bladder Diary 
• Dispense Bladder Diary 
• Dispense open-label study drug  
10.7.4. 
Open-label Treatment Period, Telephone Contact (Week 44 [Day 308 ± 4 days]) 
• Review AEs/SAEs 
• Review concomitant medications 
10.7.5. 
Open-label Treatment Period, Visit 14 (Week 52 [Day 365 ± 4 days]) or Early 
Withdrawal 
• Review AEs/SAEs 
• Review concomitant medications 
• Administer IPSS, 
, PGI, 
 scales 
• Record blood pressure (3 results taken 1-2 minutes apart after sitting for 5 minutes), 
pulse, respiration, temperature, and weight 
• Perform brief physical examination 
• Measure post-void residual volume 
• Perform laboratory assessments 
• Review and data enter completed Bladder Diary 
 


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10.8. 
Appendix 14: Patient-Reported Outcomes Questionnaires, 
Descriptions, and Instructions 
Information on each of the scales and questionnaires for patient-reported outcomes are provided 
below. A sample for each assessment is also provided. 
10.8.1. 
International Prostate Symptom Score (IPSS) 
The International Prostate Symptom Score (IPSS) is based on the answers to 7 questions 
concerning urinary symptoms and 1 question concerning quality of life. Each question 
concerning urinary symptoms allows the subject to choose 1 out of 6 answers indicating 
increasing severity of the particular symptom. The answers are assigned points from 0 to 5. The 
total score can therefore range from 0 to 35 (asymptomatic to very symptomatic). 
The questions refer to the following urinary symptoms: 
Questions 
Symptom 
1 
Incomplete emptying 
2 
Frequency 
3 
Intermittency 
4 
Urgency 
5 
Weak Stream 
6 
Straining 
7 
Nocturia 
Question 8 refers to the subject’s perceived quality of life. 
The first 7 questions of the I-PSS are identical to the questions appearing on the American 
Urological Association (AUA) Symptom Index which currently categorizes symptoms as 
follows: 
Mild (symptom score ≤ 7) Moderate (symptom score range 8 to 19) Severe (symptom score 
range 20 to 35) 
The answers to the single question to assess the quality of life this question range from 
“delighted” to “terrible” or 0 to 6.  Although this single question may or may not capture the 
global impact of BPH Symptoms or quality of life, it may serve as a valuable starting point for a 
doctor-patient conversation. 








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Takeda H, Yamazaki Y, Akahane M, Igawa Y, Ajisawa Y, Nishizawa O. Role of the β 3-
adrenoceptor in urine storage in the rat: comparison between the selective β 3-
adrenoceptor agonist, CL316, 243, and various smooth muscle relaxants. J Pharm Exp 
Ther 2000;293: 939-45. 


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Version 1.0 
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Takeda H, Yamazaki Y, Igawa Y, Kaidoh K, Akahane S, Miyata H, Nishizawa O, Akahane M, 
Andersson KE. Effects of β3-adrenoceptor stimulation on prostaglandin E2-induced 
bladder hyperactivity and on the cardiovascular system in conscious rats. Neurology and 
Urodynamics 2002;21: 558-65. 
Tubaro, A, Batista JE, Nitti, VW, et al. Efficacy and safety of daily mirabegron 50 mg in male 
patients with overactive bladder: a critical analysis of five phase III studies. The Adv 
Urol, 2017, Vol, 9(6): 137-154. 
Van Kerrebroeck P, Kreder K, Jonas U, Zinner N, and Wein A. Tolterodine once daily: superior 
efficacy and tolerability in the treatment of the overactive bladder. Urol 2001;57: 414–21. 
Woods M, Carson N, Norton N, Wesley S, Jeffery H, Argentieri TM. Efficacy of the [Beta]3-
adrenergic receptor agonist CL-316243 on experimental bladder hyperreflexia and 
detrusor instability in the rat. J Urol 2001;166: 1142-7. 
Yamaguchi O. β3-adrenoceptors in human detrusor muscle. Urology 2002;59 (Supplement 5A): 
25-29. 
Yoshida M, Takeda M, Gotoh M, Nagai S, Kurose T. Vibegron, a novel potent and selective B3-
Adrenoreceptor Agonist, for the treatment of patients with overactive bladder: a 
randomized, double-blind, placebo-controlled Phase 3 study. Euro Urol. 2018;73:783-90. 
 

SAP_001.pdf: 
STATISTICAL ANALYSIS PLAN 
Protocol Number: 
URO-901-3006 
Protocol Title: 
A Phase 3 Open-Label Extension Study to Evaluate the Long-Term 
Safety and Efficacy of Vibegron in Men with Overactive Bladder 
(OAB) Symptoms on Pharmacological Therapy for Benign Prostatic 
Hyperplasia (BPH) 
Protocol Version and 
Date: 
Version 1.0; 22-MAY-2019 
Investigational 
Product: 
Vibegron (URO-901, RVT-901, MK-4618, KRP-114V) 
Indication 
OAB symptoms for men with BPH 
Development Phase: 
Phase 3 
US IND Number: 
106410 
EudraCT Number: 
2018-003136-72 
Sponsor: 
Urovant Sciences, GmbH  
Gotthardstrasse 3 
CH-6300 Zug  
Switzerland  
Telephone +41 (42) 2155999  
SAP Version 
Effective Date 
v1.0 
31-Jul-2023
SAP Author 
, MS 
Sr. Principal Biostatistician/Director, Urovant Sciences, Inc. 
                                                           NCT Number: NCT04103450   
     This NCT number has been applied to the document for purposes of posting on Clinicaltrials.gov




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TABLE OF CONTENTS 
TABLE OF CONTENTS .................................................................................................................3 
SAP VERSION HISTORY..............................................................................................................8 
1. 
INTRODUCTION ........................................................................................................9 
1.1. 
Study Objectives and Endpoints ...................................................................................9 
1.1.1. 
Primary Objective .........................................................................................................9 
1.1.2. 
Secondary Efficacy Objectives ...................................................................................10 
1.1.3. 
Exploratory Efficacy Objectives .................................................................................10 
1.1.4. 
Exploratory Quality of Life (QoL) Objectives ...........................................................11 
1.2. 
Study Design ...............................................................................................................13 
1.2.1. 
Overall Study Design ..................................................................................................13 
1.2.2. 
Randomization and Blinding ......................................................................................14 
1.2.3. 
Statistical Hypotheses .................................................................................................14 
1.2.4. 
Sample Size Justification ............................................................................................14 
2. 
PLANNED ANALYSES ............................................................................................14 
2.1. 
Interim Analysis ..........................................................................................................14 
2.2. 
Final Analysis .............................................................................................................15 
3. 
ANALYSIS POPULATION ......................................................................................15 
3.1. 
Analysis Sets ...............................................................................................................15 
3.1.1. 
All Subjects Extension ................................................................................................15 
3.1.2. 
Safety Analysis Set Extension ....................................................................................15 
3.1.3. 
Full Analysis Set Extension ........................................................................................16 
3.1.4. 
Per-Protocol Populations ............................................................................................16 
3.2. 
Violations and Deviation ............................................................................................16 
4. 
GENERAL STATISTICAL CONSIDERATIONS ....................................................17 
4.1. 
General Principles for Data Analysis .........................................................................17 
4.1.1. 
Multicenter Study .......................................................................................................17 
4.2. 
General Data Handling Conventions ..........................................................................17 
4.2.1. 
Study Treatment Description ......................................................................................17 
4.2.2. 
Reporting Conventions ...............................................................................................18 
4.2.3. 
Premature Withdrawal and Missing Data ...................................................................19 
4.2.4. 
Assessment Windows .................................................................................................20 


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4.2.4.1. 
Study Reporting Periods .............................................................................................20 
4.2.4.2. 
Analysis Visit Windows .............................................................................................21 
4.3. 
Data Definitions and Deviations .................................................................................24 
4.3.1. 
Bladder Diary Endpoints ............................................................................................24 
4.3.1.1. 
Secondary Efficacy Endpoints (Bladder Diary) .........................................................24 
4.3.1.2. 
Exploratory Efficacy Endpoints (Bladder Diary) .......................................................25 
4.3.2. 
Patient Reported Questionnaire Endpoints .................................................................25 
4.3.2.1. 
International Prostate Symptom Score .......................................................................25 
4.3.3. 
Study Day and Duration .............................................................................................29 
4.3.4. 
Baseline and Change from Baseline ...........................................................................29 
5. 
DEMOGRAPHICS, OTHER BASELINE CHARACTERISTICS, 
PROCEDURES AND MEDICATION ......................................................................30 
5.1. 
Subject Disposition and Withdrawals .........................................................................30 
5.2. 
Demographic and Baseline Characteristics ................................................................30 
5.3. 
Other Baseline Characteristics ....................................................................................31 
5.4. 
Medical History and Concomitant Disease ................................................................31 
5.5. 
Prior and Concomitant Procedures .............................................................................32 
5.6. 
Prior and Concomitant Medication .............................................................................32 
5.6.1. 
Prior Medication .........................................................................................................32 
5.6.2. 
Concomitant Medication ............................................................................................33 
6. 
EFFICACY ANALYSIS ............................................................................................33 
6.1. 
Secondary Efficacy Analysis ......................................................................................33 
6.1.1. 
Bladder Diary Endpoints ............................................................................................35 
6.1.2. 
IPSS Endpoint .............................................................................................................35 
6.2. 
Exploratory Endpoints ................................................................................................35 
6.2.1. 
Efficacy and IPSS Endpoints ......................................................................................35 
6.2.2. 
Other Quality of Life Endpoints .................................................................................36 
6.2.2.3. 
Patient Global Impression Scores ...............................................................................36 


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7. 
SAFETY ANALYSIS ................................................................................................38 
7.1. 
Extent of Exposure .....................................................................................................38 
7.2. 
Treatment Compliance ................................................................................................39 
7.3. 
Adverse Events ...........................................................................................................39 
7.4. 
Laboratory Evaluations ...............................................................................................42 
7.5. 
Vital Sign Assessments...............................................................................................43 
7.6. 
Physical Examination .................................................................................................43 
7.7. 
Post-Void Residual (PVR) Urine Volume ..................................................................43 
7.8. 
IPSS Total Score .........................................................................................................43 
8. 
COVID-19 CONSIDERATIONS ...............................................................................44 
9. 
CHANGE FROM ANALYSIS PLANNED IN PROTOCOL....................................44 
9.1. 
Secondary Efficacy .....................................................................................................44 
9.2. 
Exploratory Endpoints ................................................................................................44 
9.3. 
COVID-19 Considerations .........................................................................................44 
9.4. 
Physical Examination .................................................................................................44 
10. 
REFERENCES ...........................................................................................................44 
11. 
APPENDIX.................................................................................................................45 
11.1. 
Table of Contents for Data Display Specifications ....................................................45 
11.1.1. 
Output Tables ..............................................................................................................45 
11.1.2. 
Output Figures ............................................................................................................52 
11.1.3. 
Output Listings ...........................................................................................................54 
  
 
 


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 LIST OF ABBREVIATIONS  
Abbreviation 
Term 
5-ARI 
5α-reductase inhibitor 
ADaM 
analysis data model 
AE 
adverse event 
AESI 
adverse events of special interest 
BMI 
body mass index 
BP 
blood pressure 
BPH 
benign prostatic hyperplasia 
CFB 
change from baseline 
CI 
confidence interval 
CRF 
case report form 
DBP 
diastolic blood pressure 
DSMB 
data safety monitoring board 
eCRF 
electronic case report form 
FAS 
full analysis set 
FAS-EXT 
full analysis set extension 
FAS-EXT-I 
full analysis set extension for incontinence 
IPSS 
International Prostate Symptom Score 
IWRS 
interactive web response system 
LS 
least squares 
MAR 
missing at random 
MedDRA 
Medical Dictionary for Regulatory Activities 
MMRM 
mixed model for repeated measures 
OAB 
overactive bladder 
 
 
PGI 
patient global impression 
PPS 
per-protocol set 
PT 
preferred term 
PRO 
patient reported outcome 
PVR 
post-void residual 
QoL 
quality of life 
REML 
restricted (or residual) maximum likelihood 
SAE 
serious adverse event 
SAF-EXT 
safety set extension 
SAP 
statistical analysis plan 
SBP 
systolic blood pressure 
SD 
standard deviation 
SDTM 
study data tabulation model 


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Abbreviation 
Term 
SE 
standard error 
SOC 
system organ class 
TEAE 
treatment emergent adverse event 
TLF 
table, listing, figure 
UUI 
urge urinary incontinence 
VAS 
visual analog scale 
VV 
volume voided 
WHO 
World Health Organization  
β3-AR 
beta-3 adrenergic receptor 


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SAP VERSION HISTORY 
Version 
Date 
Description of Changes 
1.0 
<< Date >> 
Original Document 


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1. 
INTRODUCTION 
This Statistical Analysis Plan (SAP) provides a description of the statistical methodology to be 
implemented for the analyses of data from Study URO-901-3006 Protocol Version 1.0. Any 
deviations from this analysis plan will be substantiated by statistical rationale and will be 
documented in the final clinical study report.  
1.1. 
Study Objectives and Endpoints 
Assessments in Study URO-901-3006 are referenced to the initial Baseline (Visit 3) from Study 
URO-901-3005. Change from baseline (CFB) study endpoints for safety parameters for study 
URO-901-3006 are relative to the most recent assessment prior to starting vibegron. CFB study 
endpoints for efficacy in Study URO-901-3006 are relative to Study URO-901-3005 baseline. At 
the Week 52 visit, CFB comprises 52 weeks of vibegron exposure for subjects randomized to 
vibegron in Study URO-901-3005 and 28 weeks of vibegron exposure for subjects randomized to 
placebo. 
With respect to study endpoints, “per day” refers to a “Diary Day”, which is defined as the time 
between when the subject gets up for the day each morning and the time the subject gets up for the 
day the next morning as recorded in the subject bladder diary (approximately a 24-hour period). 
1.1.1. 
Primary Objective  
Primary Objective 
 Endpoints 
To demonstrate the long-term 
safety of vibegron 75 mg in men 
with BPH with symptoms of OAB 
x Adverse Events (AEs) 
x Clinical laboratory assessments 
x Vital sign assessments 
x Post-void residual (PVR) urine volume 
x International Prostate Symptom Score (IPSS) Total 
Score 


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1.1.2. 
Secondary Efficacy Objectives  
Secondary Efficacy Objectives 
 Endpoints 
To demonstrate the long-term 
efficacy of vibegron 75 mg in men 
with BPH with symptoms of OAB
x CFB to Week 52 in the average number of 
micturition episodes per day 
x CFB to Week 52 in the average number of urgency 
episodes (urgency: need to urinate immediately) 
per day 
x CFB to Week 52 in the average number of nocturia 
episodes per night 
x CFB to Week 52 in the average number of urge 
urinary incontinence episodes per day in subjects 
with incontinence at Study URO-901-3005 
baseline 
x CFB to Week 52 in the IPSS Storage score (1-
week recall) 
x CFB to Week 52 in the average volume voided per 
micturition 
1.1.3. 
Exploratory Efficacy Objectives  
Exploratory Efficacy Objectives 
 Endpoints 


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1.1.4. 
Exploratory Quality of Life (QoL) Objectives  


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Exploratory QoL Objectives 
 Endpoints 


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1.2. 
Study Design 
1.2.1. 
Overall Study Design 
This is an international Phase 3, open-label 28-week extension study to evaluate the safety and 
efficacy of vibegron 75 mg in men with symptoms of OAB on stable doses of pharmacological 
therapy for BPH. This study is an extension for subjects who have completed the Phase 3, 
double-blind, randomized, 24-week Study URO-901-3005. 
Approximately 300 men with symptoms of OAB on stable doses of pharmacological therapy for 
BPH who completed 24 weeks in Study URO-901-3005 and continue to qualify for this study 
may be permitted to enroll in this extension study, from approximately 60 study sites. 
During this extension study, subjects who had been randomized in Study URO-901-3005 to 
receive vibegron 75 mg will continue their same treatment once daily for an additional 28 weeks, 
and subjects who had been randomized in Study URO-901-3005 to the placebo group will 
receive study treatment of vibegron 75 mg once daily for 28 weeks during the extension. Thus, 
through participation in both the URO-901-3005 and URO-901-3006 (extension) studies, 
subjects originally randomized to vibegron will receive 52 weeks total of vibegron treatment, and 
subjects originally randomized to placebo will receive 28 weeks total of vibegron treatment. 
Study visits will be named to reflect continuation from Study URO-901-3005, with the first 
study visit of this extension study occurring at Week 24. Following enrollment in this extension 
study, subjects will return to the clinic for visits at Week 28, Week 36, and Week 52 (all relative 
to Day 1 of Study URO-901-3005). A telephone contact will occur at Week 44 to review 
AEs/serious AEs (SAEs) and concomitant medications. 
This study consists of a 28-week open-label Treatment Period. Unscheduled Visit(s) may be 
arranged as needed. 
A Data Safety Monitoring Board (DSMB) will be retained to assess, on an ongoing basis, all 
safety aspects of this study. This will be an external independent DSMB that monitors the safety 
for both Study URO-901-3005 and Study URO-901-3006. The detailed activities including 
meeting plans will be described and documented in the DSMB Charter. A separate statistical 
analysis plan will be prepared for the DSMB. 
The study schema is shown in Figure 1.  


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Figure 1: 
Study Schema 
 
1.2.2. 
Randomization and Blinding 
No additional randomization will be applied for this study, and all subjects will receive open-
label treatment with vibegron 75 mg QD during the study treatment period. 
1.2.3. 
Statistical Hypotheses 
There are no formal statistical hypotheses for this trial. 
1.2.4. 
Sample Size Justification 
Approximately 300 subjects rolling over from Study URO-901-3005, in addition to other long-
term safety data with vibegron, is sufficient to characterize the long-term safety profile of 
vibegron 75 mg once daily in men with symptoms of OAB on stable doses of pharmacological 
therapy for BPH. With the assumption of approximately 33% dropout, 100 subjects will be 
expected to complete the study for the duration of 1-year on vibegron. 
2. 
PLANNED ANALYSES  
2.1. 
Interim Analysis  
No interim analysis is planned for this study. 
 
An Independent DSMB will be utilized in the study to ensure objective medical and/or statistical 
review of safety issues in order to protect the ethical and safety interests of subjects and to 
protect the scientific validity of the study. An external statistician will provide the required 
summaries to the DSMB. Further details of the DSMB review are described in the charter and 
separate DSMB SAP.  


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2.2. 
Final Analysis  
Analysis of data in study URO-901-3006 includes baseline assessments and post-vibegron-
exposure safety and efficacy data collected in study URO-901-3005 and depends on double-blind 
randomized treatment assignments from URO-901-3005. No treatment assignments from URO-
901-3005 will be unblinded within study URO-901-3006 until after study URO-901-3005 has 
been formally unblinded.    
eClinical Solutions, LLC will perform the production and quality control of all tables, figures 
and listings on behalf of Urovant Sciences, Inc. 
Statistical programming will start after data have been collected and are available in the 
database. Blinded dry-runs using dummy treatment codes for study URO-901-3005 will be 
performed prior to database lock to ensure programming displays and algorithms are developed 
as planned.   
The planned final analysis will be performed once the clinical database lock has taken place on 
both URO-901-3005 and URO-901-3006 and treatment codes for URO-901-3005 have been 
unblinded. 
3. 
ANALYSIS POPULATION  
3.1. 
Analysis Sets 
3.1.1. 
All Subjects Extension 
The All Subjects Extension consists of all subjects who signed the informed consent form (ICF) 
for URO-901-3006 and have screening data entered into the database. This population is used 
primarily for subject accounting purposes and will generally not be used for summary or 
analysis. 
3.1.2. 
Safety Analysis Set Extension 
The Safety Analysis Set Extension (SAF-Ext) will serve as the primary population for the 
analysis of safety data in this trial. 
The SAF-Ext consists of all randomized subjects in Study URO-901-3005 who 
a) have completed the 24-week treatment period in the Study URO-901-3005, and   
b) enrolled into the extension Study URO-901-3006 and received at least one dose of 
vibegron in this study. 
Safety endpoints will be descriptively summarized for the entire Safety Analysis Set Extension.  
No imputation will be performed for missing safety data. Baseline will be established as the last 
observation prior to the first dose of vibegron. All safety data collected on or after the first 
exposure to vibegron within the combined experience of the patient on URO-901-3005 and 


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URO-901-3006 will be included in the safety analysis for URO-901-3006, and all analyses by 
visit will index visits from the first dose of vibegron on either study; subjects who received 
placebo in study URO-901-3005 will only contribute safety data up to Week 28.  
3.1.3. 
Full Analysis Set Extension  
The Full Analysis Set Extension (FAS-Ext) population will serve as the primary population for 
the analysis of efficacy data in this trial. Since the endpoints related to incontinence only apply to 
subjects who meet the definition of incontinence at trial entry, it is necessary to have a separate 
FAS definition with an additional criterion to define the primary analysis population for 
incontinence endpoints. Model-based estimates will be additionally restricted to only include 
subjects who were randomized to vibegron in the parent study. 
The following FAS populations are defined in the study: 
x Full Analysis Set Extension (FAS-Ext): All OAB subjects who took at least one dose of 
vibegron in the extension study and have at least one evaluable change from URO-901-
3005 baseline micturition measurement in this study. 
x Full Analysis Set Extension for Incontinence (FAS-Ext-I): All OAB subjects who took 
at least one dose of vibegron in the extension study, were included in the FAS-I 
population in the URO-901-3005 study, and have at least one evaluable change from 
baseline (i.e., Study URO-901-3005 baseline) urge urinary incontinence measurement in 
this study. 
 
Subjects will be included in one of two vibegron treatment groups based on the original 
randomization from the parent study (URO-901-3005): 28 weeks Vibegron 75 mg, 52 weeks 
Vibegron 75 mg. Efficacy endpoints will be descriptively summarized by treatment group. 
3.1.4. 
Per-Protocol Populations  
The Per-Protocol Extension (PP-Ext) population and Per-Protocol Extension population for 
Incontinence (PPS-Ext-I) exclude patients from the FAS-Ext and FAS-Ext-I respectively due to 
important deviations from either protocol that may substantially affect the results of the efficacy 
endpoints (i.e., Major PDs associated with efficacy). Subjects assigned placebo in URO-901-
3005 will only consider protocol deviations from URO-901-3006 and inclusion/exclusion 
protocol deviations from study URO-901-3005. The final determination of protocol violations, 
and thereby the composition of the Per-Protocol populations will be made prior to database lock 
and will be documented. A supportive analysis using the Per Protocol populations will be 
performed for the 6 secondary efficacy endpoints as defined in Section 1.1.2 (i.e., CFB in 
micturition, urgency, nocturia, and urge urinary incontinence episodes; IPSS Storage score; and 
volume voided per micturition). 
Efficacy endpoints will be descriptively summarized by treatment group (i.e. 28 weeks Vibegron 
75 mg, 52 weeks Vibegron 75 mg). 
3.2. 
Violations and Deviation  
 


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Major protocol deviations are considered to have major impact on subject safety, efficacy or the 
validity of the study data.  
 
Exclusion from per-protocol analysis is noted for each protocol deviation in the Protocol 
Deviation specifications. Patients with major protocol deviations that may impact efficacy results 
will be excluded from the PPS-Ext and PPS-Ext-I. Efficacy protocol deviations may include, but 
are not limited to the following:  
 
x Subjects who do not meet the inclusion criteria 
x Subjects who meet any of the exclusion criteria 
x Concomitant use of prohibited medications  
x Other (e.g., IP compliance<70%) 
 
Subjects excluded from the per protocol analysis in URO-901-3005 will also be excluded from 
per protocol analysis in URO-901-3006, with the exception of on-study protocol deviations in 
the placebo arm as noted in section 3.1.4. 
 
The final list of major protocol deviations will be finalized and documented before database 
lock. All major protocol deviations related to efficacy will be discussed and finalized prior to 
database lock. Only major protocol deviations will be summarized and listed in the Clinical 
Study Report (CSR).  
4. 
GENERAL STATISTICAL CONSIDERATIONS 
4.1. 
General Principles for Data Analysis  
4.1.1. 
Multicenter Study  
In the parent study the stratified permuted block randomization is not done within centers. The 
analyses will be conducted by pooling data from all study centers and will not include study 
center as a covariate in the statistical modeling. 
4.2. 
General Data Handling Conventions 
4.2.1. 
Study Treatment Description 
Treatment groups based on the randomized treatment in the parent study will be displayed as 
shown in the following table: 
Table 1:  
 Study Treatment Description 
Data Displays for Reporting 
Description 
Sections Included 
Order in TLF 
52 weeks Vibegron 75mg  
Demographic and baseline data, 
Efficacy, Safety 
1 


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28 weeks Vibegron 75mg 
Demographic and baseline data, 
Efficacy (descriptive tables only), 
Safety 
2 
Overall Vibegron 75 mg  
Demographic and baseline data, 
Safety 
3 
4.2.2. 
Reporting Conventions 
General rules 
In general, all collected safety data and any derived efficacy and PRO data will be presented in 
subject data listings for all enrolled subjects. This includes all data collected on the parent study, 
URO-901-3005, relevant to the definition of baseline and all data collected on URO-901-3005 
after the first dose of vibegron, excluding assessments not repeated during 3006 (orthostatic vital 
signs, ECGs, urodynamics, and prostate volume measurement). Listings will be ordered by 
treatment group, subject number, and assessment week or event date. The treatment group 
presented in listings will be based on the actual treatment assignment in study URO-901-3005 
(placebo as 28 Weeks Vibegron 75mg, vibegron 75mg as 52 weeks Vibegron 75mg), unless 
otherwise noted. 
Summary tables will be provided for all subjects who sign informed consent for study URO-901-
3006. Unless otherwise specified; all demographic and baseline data and safety data will be 
presented by treatment group and overall. Unless otherwise specified; all efficacy data will be 
presented by treatment group. In general, continuous variables will be summarized to indicate 
the population sample size (N), number of subjects with available data (n), arithmetic mean, SD, 
median, minimum, Q1, Q3 and maximum values. Categorical variables will be summarized by 
the population size (N), number of subjects with available data (n), number of subjects in each 
category, and the percentage of subjects in each category. Unless otherwise noted, the 
denominator to determine the percentage of subjects in each category will be based on the 
number of subjects with available data (n) in the analysis set of interest. Select ordinal data may 
be summarized using both descriptive statistics and counts and percentages of subjects in each 
category, as appropriate.  
The data analyses will be conducted using the SAS® System (SAS Institute Inc., Cary, NC) 
version 9.4 or above. The eClinical Solutions standard operating procedures will be followed for 
the validation of all SAS programs and outputs. 
Formats 
Numeric data will be reported at the precision collected on the eCRF.  The reported precision 
from non eCRF sources may be adjusted to a clinically interpretable number of decimal places. 
Unscheduled Visits 
Unscheduled visits will be assigned to a study visit using the all-inclusive windows defined in 
Section 4.2.4.  However, data summaries for non-diary data will only report visits that are 
planned assessment time points for each parameter, according to the Time and Events table. All 


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visits for bladder diary data will be fully derived as described in section 4.2.4.2.  Assessments at 
unscheduled visits will be included for “any time On-treatment” endpoints and in data listings. 
4.2.3. 
Premature Withdrawal and Missing Data  
All data collected in the study database after the subject’s cessation of study treatment should be 
included in summary and analysis as defined by analysis rules. Data of subjects who are not treated 
with vibegron on either Study URO-901-3005 or URO-901-3006 will only be listed.  
If any missing data are present in diary data for any reasons, no explicit missing data imputation 
will be performed for the analysis of change from baseline since a mixed model for repeated 
measures (MMRM) will be applied to change from baseline analysis.   
 
Missing items from the subject reported outcomes (PROs) will not be imputed. Some scores may 
still be calculated from the non-missing questions; handling rules are detailed for each individual 
PRO within the appropriate subsection of 4.3.2. 
 
In general, missing safety data will not be imputed and only observed values will be analyzed.  
 
If the relationship of an AE record (“Relationship to investigational product” on AE CRF) is 
missing this AE will be considered “Probably Related” to the study treatment. If the AE intensity 
is missing every effort should be made to acquire the information from the investigator. “Severe” 
will be assigned to a missing intensity for reporting purposes. 
 
Dates of AEs and medications reported under the URO-901-3005 protocol (including all start 
dates of events and medications initially reported in URO-901-3005 and end dates of events and 
medications that were not ongoing at the time of URO-901-3006 entry) will follow the date 
imputation rules from the URO-901-3005 SAP; dates will not be re-imputed. 
 
The general imputation rules of partial missing date and time for both AE and medication is 
detailed below: 
 
Dates missing the day of the year will adhere to the following conventions:  
x The missing day of onset date will be set to: 
 The day of first active treatment with vibegron in URO-901-3006, if the onset YYYY-
MM is the same as YYYY-MM of the first active treatment 
 First day of the month that the event occurred otherwise. 
 
x The missing day of end date will be set to: 
 The death date, if the end date YYYY-MM is the same as the YYYY-MM of the death 
date  
 The end of study participation, if the end date YYYY-MM is the same as the YYYY-
MM of the end of study participation date 
 The last day of the month of the occurrence otherwise. 
 
Dates missing both the day and month of the year will adhere to the following conventions: 


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x Missing onset date will be set to: 
 January 1 of the year of the onset, if the onset YYYY is after the YYYY of the first 
active treatment 
 The date of the first active treatment in URO-901-3006, if the onset YYYY is the same 
as the YYYY of the first active treatment 
 
x The missing end date will be set to: 
 The death date, if the end date YYYY is the same as the YYYY of the death date 
 The end of study participation, if the end date YYYY is the same as the YYYY of the 
end of study participation date 
 December 31 of the year of occurrence otherwise. 
4.2.4. 
Assessment Windows 
4.2.4.1. 
Study Reporting Periods 
Based on study design and variables under consideration, study time periods are defined as 
below. 
 
   Table 2:  
 Definition of Study Reporting Periods 
Analysis 
description 
Analyzing 
Study Period 
Start Date  
End Date 
General 
Pre Treatment 
(Day -49 – 
Day 1/Week 24) 
Date of informed 
consent 
Prior to the date of first dose 
of vibegron on either study 
URO-901-3005 or URO-
901-3006 
Treatment (Day 
1/Week 24 – 
Week 52)  
Date of first dose of 
vibegron on either study 
URO-901-3005 or 
URO-901-3006  
Date of last dose of 
vibegron on URO-901-3006 
+ 5 or EOS, whichever is 
earlier 
Adverse 
events  
Prior 
(Summarized as 
Medical History) 
Start date ≥ date of 
informed consent on 
URO-901-3005 and < 
date of first dose of 
vibegron in either study. 
No restriction 
 
Treatment 
Emergent 
(TEAE) 
Start date (AE begins or 
worsens in severity) ≥ 
date of first dose of 
vibegron* and ≤ date of 
last dose of vibegron + 5 
days or EOS visit, 
whichever is earlier. 
No restriction 


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Analysis 
description 
Analyzing 
Study Period 
Start Date  
End Date 
Prior or 
Concomitant 
Medication/ 
Procedure  
Prior medication/ 
procedure 
Start date < date of first 
vibegron dose  
End date < date of first 
vibegron dose OR 
medication not marked as 
‘ongoing’ on study URO-
901-3005 and subject 
received placebo on study 
URO-901-3005 
 
Concomitant 
medication/ 
procedure 
Start date ≥ date of first 
dose of vibegron and ≤ 
date of last vibegron 
dose + 5 days or EOS 
visit, whichever is 
earlier. 
 
 
Start date < date of first  
vibegron dose, but 
ongoing during 
vibegron treatment 
period 
End date ≥ date of first 
vibegron dose OR 
medication marked as 
‘ongoing’ on study URO-
901-3005. 
First and last dose of vibegron refer to the first and last dose across both study URO-901-
3005 and URO-901-3006, for subjects who were treated with at least one dose of vibegron 
in study URO-901-3006. 
EOS: End of study is defined as the date when the subject has completed one of the 
following: completes Week 52 Safety Follow-up, discontinued from the study, or is lost to 
follow-up. 
*AEs with a start date equal to the date of first dose of vibegron will be considered treatment-
emergent unless the relatedness to study drug is marked ‘Not applicable’. 
Overlapping period: Medication start date <= period start date < medication end date, 
medication start date < period end date <=medication end date, or period start date <= 
medication start or end date < period end date, then the medication is considered overlapping 
with the period.      
4.2.4.2. 
Analysis Visit Windows  
 
Analysis visit windows for both diary and non-diary efficacy data are shown in Table 3. All 
observations will be given an analysis visit, assigned based on relative study day from the first 
blinded treatment in study URO-901-3005. If multiple repeats of the same assessment are 
observed within an analysis window, unless the measure was intended to be averaged over 
replicates, the record closest to the nominal time (earlier in case of a tie) will be used for by-visit 
summaries. If fewer than three of the diary days are within the analysis window, only the data 
within the window will be used to calculate visit-level averages. 
 
 


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             Table 3: Efficacy Analysis Visit Window Slotting 
Analysis 
window label 
Non-diary data 
Diary data 
Nominal 
visit 
Nominal 
day 
Visit Window Nominal 
visita 
Nominal 
day 
Window 
           
Baseline       
Visit 3 
1 
NAb 
Run-in 
-1 
[-7c, -1] 
Week 1 
Visit 4 
8 
[2, 11] 
 
 
 
Week 2 
Visit 5 
15 
[12, 22] 
Visit 4 
15 
[7,18] 
Week 4 
Visit 6 
29 
[23, 42] 
Visit 5 
29 
[19, 39] 
Week 8  
Visit 7 
57 
[43, 70] 
Visit 6 
57 
[47, 67] 
Week 12  
Visit 8  
85 
[71, 98] 
Visit 7 
85 
[68, 102] 
Week 16 
Visit 9 
113 
[99, 126] 
Visit 8 
113 
[103, 123] 
Week 20 
Visit 10 
141 
[127, 154] 
Visit 9 
141 
[131, 151] 
Week 24 
Visit 11 
169 
[155, 182b] 
Visit 10 
169 
[159, 179] 
Week 28 
Visit 12 
197 
[183d, 224] 
Visit 11 
197 
[187, 207] 
Week 36 
Visit 13 
253 
[225, 308] 
Visit 12 
253 
[243, 263] 
Week 52 
Visit 14 
365 
> Day 309 
Visit 13 
365 
[345, 385] 
a Nominal visit for diary data is the visit where the diary was dispensed. 
b Must have occurred prior to the first dose administered in study URO-901-3006. 
c If randomization was delayed for any reason, diaries completed within 7 days prior to the baseline visit will still 
be considered within the analysis window. 
d Must have occurred on or after the first dose administered in study URO-901-3006. 
 
For all non-diary safety data including safety laboratory, vital signs, and PVR volume to be 
summarized by visit, data records will be slotted to one of the protocol-specified visits using the 
following algorithm: 
 
1. Determine relative study day and last dose day: 
      Relative study day will be derived as described in Section 4.3.3. 
      Note that the date of first double-blind dose on URO-901-3005 is Day 1 and the day 
before the date of double-blind first dose is Day -1. There is no Study Day 0. 
 
2. For non-early withdrawal records, if a nominal visit number for a scheduled visit was 
assigned, then it will be used to summarize the record after checking to make sure that 
visit numbers match with therapy phases: 
x Check that all records entered as a pre-treatment visit for study URO-901-3005 occur 
on or before the day of the first dose of double-blind treatment (i.e. day d1).  Any 
record with day > 1 should be re-slotted to an on-treatment visit using the relative day 
window below. 
x Check that all records slotted to an on-treatment visit for study URO-901-3005 and 
all records for study URO-901-3006 are on or after the day of the first dose of double-
blind treatment.  If they are not, the visit should be moved to pre-treatment.  


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3. Any early withdrawal record (regardless of the visit number assigned), any unscheduled 
visit record at any time, any repeat visit record, or any record for which the visit number 
was missing or the visit number was not for a scheduled visit, should be slotted or re-
slotted to a visit using the window below.  
 
             Table 4: Analysis Visit Window Slotting 
Analysis 
window label 
URO-901-3005 Randomized Arm 
Vibegron 
Placebo 
Nominal 
visit 
Nominal 
day 
Visit Window Nominal 
visit 
Nominal 
day 
Visit 
Window 
           
Baseline             
Visit 3 
1 
NAa 
Visit 3 
1 
[1,182b] 
Week 1 
Visit 4 
8 
[2, 11] 
NA 
NA 
NA 
Week 2 
Visit 5 
15 
[12, 22] 
NA 
NA 
NA 
Week 4 
Visit 6 
29 
[23, 42] 
Visit 12 
197 
[183c,224] 
Week 8  
Visit 7 
57 
[43, 70] 
NA 
NA 
NA 
Week 12  
Visit 8  
85 
[71, 98] 
Visit 13 
253 
[225,308] 
Week 16 
Visit 9 
113 
[99, 126] 
NA 
NA 
NA 
Week 20 
Visit 10 
141 
[127, 154] 
NA 
NA 
NA 
Week 24 
Visit 11 
169 
[155, 182a] 
NA 
NA 
NA 
Week 28 
Visit 12 
197 
[183b, 224] 
Visit 14 
365 
> Day 309 
Week 36 
Visit 13 
253 
[225, 308] 
NA 
NA 
NA 
Week 52 
Visit 14 
365 
> Day 309 
NA 
NA 
NA 
a Must have occurred prior to the first dose administered in study URO-901-3005. 
b Must have occurred prior to the date of first dose administered in study URO-901-3006. 
c Must have occurred on or after the date of first dose administered in study URO-901-3006. 
Note: The cut-off days to define the window for slotting through Week 20 are aligned 
with study URO-901-3005.    
For parameters which were not scheduled to be collected at all visits, the all-inclusive 
visit intervals defined for all visits (i.e., the window defined above) will still be used to 
slot records. If a value is slotted to a visit unscheduled for a parameter, it will not be 
summarized (and not be included in the efficacy datasets) but will be included in data 
listings.  
4. If after all records have been slotted or re-slotted, there are multiple valid records within a 
visit, use the record following the rules below: 
x If there are multiple values for an endpoint in one window, the visit closest to the 
nominal day will be selected for by-visit displays. If the visits are equidistant from the 
nominal day, then the earlier visit will be selected. All values will be stored in 
analysis datasets.  


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4.3. 
Data Definitions and Deviations 
4.3.1. 
Bladder Diary Endpoints 
The bladder diary records what time subjects woke up for the day, what time they went to bed, 
and asks the subjects to record every time they had a urination event. For each event, subjects 
recorded the time, if they had a need to urinate immediately, if they urinated in the toilet, if they 
had accidental urine leakage, and if they had leakage what the main reason was for the leakage 
(urge or other) for 3 days and volume voided per micturition (over one 24-hour period) prior to 
each study visit. If fewer than three days were collected within the diary window, the average of 
the days within window will be used. If more than three days were collected within the diary 
window, the average of the three closest days to the nominal timepoint will be used. 
The following efficacy parameters will be derived based on the Diary data for baseline, Weeks 2, 
4, 8, 12, 16, 20, 24, 28, 36, and 52.  
4.3.1.1. 
Secondary Efficacy Endpoints (Bladder Diary) 
 
Although these endpoints are automatically derived in eCRF Day 3 Bladder Diary page, the final 
statistical analysis will use the value derived by the biometrics team based on the algorithm 
described below. 
CFB in average daily number of micturition episodes to Week 52 
The number of micturition episodes will be defined as the number of times a subject has voided 
in the toilet as indicated on the Bladder Diary.  Average number of micturition episodes per day 
at each study visit will be calculated as the total number of micturition records within the diary 
analysis visit windows described in Table 3 divided by non-missing diary days (diary days with 
at least one void reported).   
CFB in average daily number of urgency episodes to Week 52 
The number of urgency episodes will be defined as the number of times a patient has checked 
that he had “need to urinate immediately” micturition in the toilet or “urge” accident leakage as 
indicated on the Bladder Diary.  Average daily number of urgency episodes at each study visit 
will be calculated as the total number of urgency episodes within the diary analysis visit 
windows divided by non-missing diary days.  
CFB in average nightly number of nocturia episodes to Week 52 
Average nightly number of nocturia episodes will be calculated in the same manner as average 
daily number of micturition episodes but only include episodes occurring during sleep (all 
episodes occurring after the time reported that a subject went to bed on the same diary day and 
prior to the time a subject reported getting up for the day on the subsequent diary day). If no 
subsequent time of awakening was filled out, all records after the time a subject went to bed on 
the day will be used. 
CFB in average daily number of urge urinary incontinence (UUI) episodes to Week 52 for 
subjects with UI at study URO-901-3005 baseline 


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The number of UUI episodes will be defined as the number of times a subject has checked 
that they had "urge" as the main reason for accident leakage regardless of whether “other” is 
checked in addition to “urge”. Average UUI episodes per day at each study visit will be 
calculated in the same manner as described above for the micturition endpoint. The UUI 
endpoint will be analyzed in the FAS-Ext-I and PPS-Ext-I population. 
CFB in average volume voided per micturition to Week 52 
The total volume voided will be the sum of all urinary volumes collected regardless of whether 
patients checked “Urinated in Toilet” or not. Average volume voided per micturition at each 
study visit will be calculated as the total volume voided divided by the total number of 
micturition episodes during non-missing diary days. 
4.3.1.2. 
Exploratory Efficacy Endpoints (Bladder Diary) 
4.3.2. 
Patient Reported Questionnaire Endpoints 
4.3.2.1. 
International Prostate Symptom Score 
The International Prostate Symptom Score (IPSS) is based on the responses to 7 questions 
concerning urinary symptoms and 1 question concerning quality of life. Each question concerning 
urinary symptoms allows the subject to choose 1 out of 6 answers indicating increasing severity of 
the particular symptom. The responses are assigned points from 0 to 5. The total score can therefore 
range from 0 to 35 (asymptomatic to very symptomatic).  
The questions refer to the following urinary symptoms: 


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Questions  Symptom  
1  
Incomplete emptying  
2  
Frequency  
3  
Intermittency  
4  
Urgency  
5  
Weak Stream  
6  
Straining  
7  
Nocturia  
Question 8 refers to the subject’s perceived quality of life (QoL). The answers to the single question 
to assess the quality of life this question range from 0: “delighted” to 6: “terrible”. 
The IPSS questions reference storage symptoms (frequency, urgency and nocturia scores) and 
voiding symptoms (incomplete empty, intermittency, weak stream and straining) [1]. The 
following parameters will be derived:  
CFB in IPSS storage score to Week 52 (secondary efficacy) 
The IPSS storage score will be derived as the sum of the IPSS storage questions (sum of 
frequency, urgency and nocturia scores) for each visit. If any of these questions are not 
answered, the IPSS storage score will not be calculated. 




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Function Domain 
Questions 
Direction of Domain Scores 
4.3.3. 
Study Day and Duration  
Study day is relative to the start date of the double-blind treatment on the parent study URO-901-
3005. This is used to describe the relative time of an event or assessment that happened during 
the study. The first day of the study is defined as the day a patient first receives either vibegron 
or placebo in the double-blind treatment period. This is expected to be the same day as the 
randomization as Day 1.  There is no study Day 0 defined in the study.  
x For event or assessment that occurred on or after the first dose of double-blind treatment 
date:   
           Study day = Date of event or examination – date of first study treatment + 1 
x For event or assessment that occurred prior to the first dose of double-blind treatment 
date:   
           Study day = Date of event or examination – date of first study treatment 
Treatment day will be defined in the same manner relative to the date of first dose of vibegron. 
Similarly, a duration between any two dates (such as AE duration) expressed in days will be 
calculated using the following conventions: 
x Duration = Later date – earlier date + 1 
4.3.4. 
Baseline and Change from Baseline 
In general, the last recorded value on or prior to the date of randomization in the parent study 
URO-901-3005 will serve as the baseline measurement for efficacy endpoints while the last 
recorded value prior to first double-blind dose of study treatment will serve as the baseline 
measurement for safety endpoints. The mean of multiple values will be used as baseline for the 
following situations: 
x If multiple measurements are scheduled on the same baseline day (i.e., blood pressure) 
x If multiple measurements are collected on the same baseline day without the time or 
“repeat” status to differentiate the records 
For efficacy endpoints derived from the Bladder Diary, the baseline will be the average value 
over the diary days in the baseline analysis visit window on study URO-901-3005.   
Change from baseline will be calculated as the post-baseline value minus the baseline value. 
Percentage change from baseline will be calculated as the change from baseline divided by the   
baseline value, multiplied by 100. If either the baseline or post-baseline value is missing, then 
change from baseline and percentage change from baseline will be set to missing. 


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5. 
Demographics, Other Baseline Characteristics, Procedures And 
Medication 
5.1. 
Subject Disposition and Withdrawals 
Subject disposition will be summarized by treatment group and overall for the consented and 
treated subjects. The summary table will present the frequency and percentage of subjects in 
each of the analysis sets, treatment on URO-901-3006, and those who discontinued the study 
prematurely along with the primary reasons for discontinuation.  
For the summary of the treated subjects, the following additional categories will be presented as 
well; discontinued treatment with study drug (and reason), completed study, and withdrawn from 
study (and reason). 
The frequency and percentage of subjects with at least one major Protocol Deviation (PD), major 
PD by classification and reasons/category for PD will be summarized by planned overall 
treatment duration and overall for the FAS-Ext. Inclusion in each of the analysis sets (SAF-Ext, 
FAS-Ext, FAS-Ext-I, PPS-Ext, PPS-Ext-I), and any reasons for exclusion will be summarized by 
planned overall treatment duration for the Treated Set.  
Eligibility criteria, screening failures (including date and primary reason for failure), and 
informed consent (protocol version, informed consent version date and date signed) will be listed 
for all patients screened. 
A summary of treated subjects by country and investigator will be provided. Randomization 
details will also be listed, including the date of randomization, randomization number and 
randomization strata. 
5.2. 
Demographic and Baseline Characteristics 
All demographic and baseline characteristic data will be summarized by planned overall 
treatment duration and overall using descriptive statistics for all subjects for each of the 
following analysis sets: SAF-Ext, FAS-Ext, FAS-Ext-I, PPS-Ext and PPS-Ext-I.  
The summary table will include Ethnicity and Race, Region (US and non-US), Prior 
Anticholinergic Use (Yes/No), Prior Beta-3 agonist Use (Yes/No), Diabetes Mellitus (Yes/No), 
Baseline Hypertension (Yes/No), Pre-existing Hypertension (Yes/No), Age category 1 (<55, ≥55 
to <65, ≥65 to <75, ≥75 years), Age category 2 (<65, ≥65 years), Age category 3 (<65, ≥65 to 
<85 years, ≥85 years), baseline average micturition episodes per day (≤ 12 vs > 12), Alpha 
blocker use with or without 5-ARI (with vs without), Urinary Incontinence (yes or no), total 
IPSS score, alpha blocker (tamsulosin, doxazosin, alfuzosin, other), and 5-ARI (finasteride, 
dutasteride, other).  
Prior Anticholinergic Use and Prior Beta-3 agonist Use will be calculated from all prior 
medications documented on the Prior and Concomitant Medications electronic case report form 
(eCRF).  


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Baseline Hypertension will be defined as baseline systolic blood pressure (SBP) ≥140 mmHg or 
baseline diastolic blood pressure (DBP) ≥90 mmHg, regardless of medical history. 
Pre-existing hypertension will be defined as having a medical history of hypertension or Baseline 
hypertension (baseline SBP ≥140 mmHg or baseline DBP ≥90 mmHg). The following list of 
preferred terms will be used to search for medical history of hypertension:  
x 
Accelerated hypertension 
x 
Diastolic hypertension 
x 
Essential hypertension 
x 
Hypertension 
x 
Hypertensive crisis 
x 
Hypertensive emergency 
x 
Hypertensive heart disease 
x 
Malignant hypertension 
x 
Malignant hypertensive heart disease 
x 
Secondary hypertension 
x 
Supine hypertension 
x 
Systolic hypertension 
Age (years), height (cm), weight (kg), BMI captured at Screening on study URO-901-3005, and 
baseline IPSS total score will be summarized as a continuous variable. 
Unless otherwise stated, percentages will be calculated out of the number of patients in the given 
analysis set. 
All demographic data will be listed. 
5.3. 
Other Baseline Characteristics 
The data from the 3-day bladder diary during run-in period on study URO-901-3005 (reviewed at 
Visit 3 Baseline visit) prior to first dose of double-blind medication will be used as baseline for 
each subject. This includes average daily number of micturition episodes, average daily number 
of urgency episodes, average number of nocturia episodes per night, average daily number of 
urge urinary incontinence episodes, and average volume voided per micturition. In addition, the 
baseline IPSS storage score will be presented. These will be summarized by treatment group and 
overall using descriptive statistics for continuous data for all subjects in FAS-Ext, FAS-Ext-I, 
PPS-Ext and PPS-Ext-I analysis sets. 
5.4. 
Medical History and Concomitant Disease 
Descriptions of medical history findings will be coded using Medical Dictionary for Regulatory 
Activities (MedDRA) version 23.0 or higher. 
Medical history will include all events entered on the medical history form in URO-901-3005 as 
well as any AEs with the end date prior to the first date of active treatment on either URO-901-
3005 or URO-901-3006.  


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A disease or illness reported as medical history without a start date will be included in medical 
history without a date assigned. Medical history will be sorted by descending overall frequency, 
by system organ class (SOC) and preferred term (PT) in the summary table. Medical history data 
listings will be sorted by treatment, subject number, start date, SOC and PT. The SAF-Ext will 
be the analysis set for medical history data.  
5.5. 
Prior and Concomitant Procedures 
Prior and concomitant procedures will be coded using Medical Dictionary for Regulatory 
Activities (MedDRA) version 23.0 or higher. 
Prior procedures are defined as procedures with a start date prior to the first dose of vibegron on 
study URO-901-3005 or URO-901-3006. Concomitant procedures will be defined as procedures 
started on or after the first dose of vibegron but prior to the last dose + 5 days, or started prior to 
the first dose of vibegron and were ongoing during the study. Post procedures are defined as any 
procedure initiated after last dose of vibegron + 5 days (See Table 2). Partial procedure start 
dates will be imputed as detailed in 4.2.3. 
All procedures will be listed for the SAF-Ext population, sorted by treatment, subject number, 
start date, SOC and PT, with indicators of prior and concomitant status. 
5.6. 
Prior and Concomitant Medication  
Prior and concomitant medications will be coded using the World Health Organization (WHO) 
Drug B3-Sept Format, 2020 version or later. Except for Prior OAB medication, the number and 
percentage of subjects taking prior medications and concomitant medications will be 
summarized overall by ATC (Anatomical Therapeutic Chemical) Levels 2 and 4 for all subjects 
in the SAF-Ext. Prior OAB medications will be summarized by ATC Levels 2, 4 and Preferred 
Term in the SAF-Ext. Prior medications, OAB medication and concomitant medications will be 
listed for all subjects in the SAF-Ext. 
5.6.1. 
Prior Medication 
Prior medications are defined as those medications taken prior to the first dose of vibegron on 
study URO-901-3005 or URO-901-3006. The prior non-OAB and prior OAB medications will 
be summarized separately.  
The following criterion will be used for selecting prior OAB medication: 
 
 
 
 
 


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Table 7. 
OAB Medication Selection 
Class 
Variable 
Selected 
Selection 
Anticholinergi
cs  
Preferred 
Term 
darifenacin,  
fesoterodine,  
festoterodine fumarate hyoscyamine, oxybutynin, 
oxybutynin hydrochloride,  
propantheline, 
solifenacin,  
solifenacin succinate,  
tolterodine, 
tolterodine l-tartrate,  
trospium,  
trospium chloride 
Beta-3 
adrenergic 
agonists 
Preferred 
Term 
mirabegron,  
vibegron 
Combination medications will be identified if the preferred term contains a term from the 
selection. 
5.6.2. 
Concomitant Medication 
Concomitant medications are defined as medications that were started on or after the first dose of 
vibegron on study URO-901-3005 or URO-901-3006 but prior to the last dose of vibegron + 5 
days or started prior to the first dose of vibegron and were ongoing or ended after the first dose 
of vibegron. Partial medication start dates will be imputed as detailed in 4.2.3.  
Non-OAB and OAB concomitant medications will be summarized separately. 
6. 
EFFICACY ANALYSIS 
Subjects were asked to fill out a Bladder Diary for 3 consecutive days prior to the Run-in visit, 
Baseline visit, Weeks 2, 4, 8, 12, 16, 20, 24, 28, 36, and 52.  
In general, the FAS-Ext will be used for all analyses of non-incontinence efficacy endpoints. The 
FAS-Ext-I will be used for all incontinence efficacy endpoints; these are the endpoints related to 
UUI episodes and total incontinence episodes.  
6.1. 
Secondary Efficacy Analysis 
The secondary efficacy analysis will be done on the FAS-Ext or FAS-Ext-I population, repeated 
in a supportive analysis on the PP-Ext or PP-Ext-I population, respectively. Descriptive 


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summaries for observed and CFB for each efficacy endpoint will be provided by visit and 
treatment group. 
For the assessment of change from baseline to Week 52 for endpoints with repeated-measures 
over time, a mixed model for repeated measures (MMRM) analysis incorporating on-treatment 
values at all time points for the subjects in the 52-week treatment group will be used. The model 
takes into account correlation within the same subject over time by using all available data on the 
subjects within the same covariate set. With the assumption of missing at random (MAR), this 
approach will provide unbiased estimates of treatment effects.   
The MMRM model will include terms for visit, baseline value, alpha blocker use with or without 
5-ARI (with vs. without), baseline urinary incontinence (yes vs. no) [for the FAS-Ext and PP-Ext 
analyses only], region (US vs. non-US), and any other parameter-specific covariates noted in 
sections 6.1.1 and 6.1.2.  The Kenward-Roger adjustment will be used with restricted (or 
residual) maximum likelihood (REML) to make statistical inference.  An unstructured 
covariance matrix will be used to model the correlation among repeated measurements. If the 
unstructured covariance model fails to converge with the default Newton-Raphson algorithm, the 
Fisher scoring algorithm will be used to provide initial values of the covariance parameters.  In 
the rare event that none of the above methods yield convergence, the following structures will be 
investigated:  heterogeneous Toeplitz, Toeplitz, heterogeneous First-Order Autoregressive [AR 
(1)], heterogeneous compound symmetry (HCS), and compound symmetry (CS).  The 
covariance structure converging to the best fit, as determined by Akaike’s information criterion 
(AIC), will be used. 
Estimates of least square means (LSMEANS), standard errors, and 95% confidence intervals will 
be presented at each time point. The estimated LSMEANS (Standard Error (SE)) of CFB over 
time from the model will be plotted. 
An example of the SAS code for the base procedure is given below: 
    proc mixed data = datain method = reml; 
  class AVISITN USUBJID ARI UI REGION; 
  model CHG = AVISITN ARI UI REGION BASE 
        / ddfm=KR solution chisq; 
  repeated AVISITN / subject=USUBJID type=UN r rcorr; 
  lsmeans AVISITN / cl alpha=0.05; 
    run; 
 
Where AVISITN is the visit number, USUBJID is the unique subject identifier, ARI indicates if 
the subject used alpha blocker with or without 5-ARI at baseline (with or without), UI indicates 
baseline urinary incontinence (yes or no), REGION indicates if the subject is in the United States 
or in the non-US countries and BASE indicates baseline value. The default Newton-Raphson 
algorithm is employed for type = UN. 
The normality assumptions for the primary analysis model will be assessed by inspection of the 
residuals from the model and normal probability plots. If assumptions of normality are not met, 
additional supportive analysis of the data will be performed in order to assess the robustness of 
the conclusions drawn from the model. 


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6.1.1. 
Bladder Diary Endpoints  
Refer to parameter derivations from Bladder Diary data in Section 4.3.1. Scheduled post-
treatment visits for bladder diary endpoints include Weeks 2, 4, 8, 12, 16, 24, 28, 36, and 52.  
CFB to Week 52 in average daily number of micturition episodes 
This analysis will be done on the FAS-Ext set and repeated on the PP-Ext set, with no additional 
covariates. 
CFB to Week 52 in average daily number of urgency episodes 
This analysis will be done on the FAS-Ext set and repeated on the PP-Ext set, with an additional 
categorical covariate of average number of micturition episodes per day (≤ 12 vs. > 12).  
CFB to Week 52 in the average nightly number of nocturia episodes 
This analysis will be done on the FAS-Ext set and repeated on the PP-Ext set, with no additional 
covariates. 
CFB to Week 52 in the average daily number of UUI episodes for subjects with baseline 
incontinence in study URO-901-3005  
This analysis will be done on the FAS-Ext-I set and repeated on the PP-Ext-I set, with no 
additional covariates. 
CFB to Week 52 in the average volume voided per micturition 
This analysis will be done on the FAS-Ext and PP-Ext sets, with no additional covariates. 
6.1.2. 
IPSS Endpoint  
Refer to endpoint definitions in section 4.3.2.1. Scheduled visits for the IPSS include Weeks 4, 8, 
12, 24, 36, and 52. 
CFB to Week 52 in the IPSS storage score (1-week recall) 
This analysis will be done on the FAS-Ext set and repeated on the PP-Ext set, with no additional 
covariates. 
6.2. 
Exploratory Endpoints 


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6.2.2. 
Other Quality of Life Endpoints 
The FAS-Ext will be the analysis population for the PRO data unless stated otherwise. 
6.2.2.1. 
CFB in 
 Scores 
6.2.2.3. 
Patient Global Impression Scores 
The questionnaire is assessed at baseline, Weeks 4, 12, 24, and 52 for the 52-week treatment 
group, and baseline and Week 28 (Week 52 relative to the start of URO-901-3005) for the 28-
week treatment group. The Patient Global Impression (PGI) questions are designed to assess a 


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subject’s overall impression of OAB in 5 categories: 
 
  
Each of the responses to the PGI questions will be assigned a numerical value starting from 1. A 
higher score represents a worse outcome in each category. 
Table 8: 
PGI Scoring 
PGI Category 
Score 
 
1 = None 
2 = Mild 
3 = Moderate 
4 = Severe 
 
1 = Complete control 
2 = A lot of control 
3 = Some control 
4 = Only a little control 
5 = No control 
1 = Never 
2 = Rarely 
3 = Sometimes 
4 = Often 
5 = Very often 
 
1 = Much better 
2 = Moderately better 
3 = A little better 
4 = No change 
5 = A little worse 
6 = Moderately worse 
7 = Much worse 
 
The questions 
PGI-Control, PGI-Frequency and PGI-Leakage are ordinal 
assessments going from lower impact of disease to highest impact of disease with score with 
scores ranging from 1 to 4 for 
 and 1 of 5 for 
 
 These questions will be summarized descriptively as numerical variables of score 
and change from baseline by treatment group and visit. Additionally, these questions will be 
analyzed as categorical variables with count and percent for each category by treatment group 
and visit. 
 
The 
 question is asked relative to baseline. The score will only be summarized 
as a categorical variable with count and percent for each category by treatment group and visit. 
 
The FAS-Ext will be the analysis set for all categories except for 
The FAS-Ext-I 
will be the analysis set for the
 analysis. 


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7. 
SAFETY ANALYSIS 
The SAF-Ext will be used for all safety analyses. Safety will be assessed based on extent of 
exposure and compliance, AE reports, clinical laboratory data, physical examinations, vital signs, 
post-void residual volume, and total IPSS score. 
No inferential statistical testing is planned on the safety data, all data will be summarized and 
listed only. 
7.1. 
Extent of Exposure 
The duration of exposure with vibegron over studies URO-901-3005 and URO-901-3006 will be 
expressed as the time in days from the first dose of vibegron as recorded on the Study Drug 
Administration CRF page through to last treatment day (inclusive) as recorded on the End of 
Treatment CRF page, excluding any days where it is recorded that an interruption has occurred 
on the AE CRF page. If no last treatment date is recorded, it will be imputed as the minimum of 
the date of the last visit on study 3006 and the date when last dispensed study treatment was 
scheduled to be exhausted (last dispensation date + number of doses dispensed). 
Interruptions to dosing can be due to delays in resupply (measured by gaps in the study drug log) 
or interruptions due to AE. Interruptions due to AE that overlap partially or completely will be 
listed separately but not double-counted for summing interruption days. 
Gaps in the study drug log will be determined by comparing the dispensation date of a record to 
the previous dispensation date (where up to four bottles may have been dispensed). The gap is 
measured from the study day of previous dispensation + number of pills dispensed + number of 
days of AE interruption within the period. 
Interruption in days = date interruption started – date interruption stopped + 1  
This is given by the following formula: 
Duration (days) = date of last vibegron dose – date of first vibegron dose – 
                              interruption days + 1 
Duration of exposure will be summarized by treatment group and overall for the SAF-Ext set 
using summary statistics for continuous variables. A listing will present the treatment start and 
end date together with date(s) of interruption and the overall duration of exposure. 


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7.2. 
Treatment Compliance 
Study treatment compliance (%) will be calculated as the actual number of vibegron doses taken 
divided by the expected number of vibegron doses, multiplied by 100 and summarized by 
treatment duration. 
These numbers will be calculated as the sum of the number of tablets indicated as taken by the 
subject on each dispensation record. Where no treatment bottle is returned, and thus the actual 
number of doses taken is unknown, it will be assumed that the subject took medication as directed 
until another bottle was dispensed, their supply was exhausted, or end of treatment was noted. This 
is calculated as (minimum of [next dispensation date – dispensation date + 1], [doses dispensed] 
and [EOT date – dispensation date + 1]).. Imputed values will be clearly noted in the listing.  
The expected number of vibegron doses is the same as the calculated duration of exposure detailed 
in section 7.1. 
Treatment Compliance will be summarized for the SAF-Ext and FAS-Ext population. 
Additionally, the number and percentage of subjects within each planned treatment duration with 
compliance in the following categories will be provided: <80%, 80 – 120%, and >120%. All data 
will be listed. 
7.3. 
Adverse Events 
AEs will be coded using MedDRA version 23.0 or higher. 
All reported AEs (whether treatment emergent or not) on both study URO-901-3005 and URO-
901-3006 will be included in by-subject AE listings on the Safety Set Extension. Sorting will be 
by country, site, subject, date of event, SOC, PT and then verbatim description. An additional 
overall AE listing will be presented for subjects that were enrolled but not dosed with vibegron 
on URO-901-3006. 
An AE will be considered treatment emergent (TEAE) if it begins or worsens in severity after the 
first dose of vibegron on study URO-901-3005 or URO-901-3006 through 5 days after the last 
dose of vibegron. Partial AE start dates will be imputed as detailed in Section 4.2.3. 
Summary tables will be based on TEAEs. The incidence of TEAEs will be presented using 
counts and percentages of subjects with TEAEs and tabulated by SOC and PT. SOC will be 
sorted in descending frequency and PT within SOC will be sorted by descending frequency 
based on the incidence across subjects overall. If a subject has multiple occurrences (start and 
stop) of an event associated with a specific SOC or PT within a SOC, a subject will only be 
counted once in the incidence count for the SOC or PT within SOC respectively. 
An overall summary table of AEs will be presented detailing the number and percentage of 
subjects, and number of events for the following categories: 
x 
At least one TEAE; 


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x 
At least one Treatment-Related TEAE; 
x 
At least one Grade ≥ 3 TEAE (Mild = Grade 1, Moderate = Grade 2, Severe of 
Medically Significant = Grade 3, Life-Threatening = Grade 4, Death = Grade 5) 
x 
At least one Grade ≥ 3 Treatment-Related TEAE; 
x 
At least one Serious TEAE; 
x 
At least one Serious Treatment-Related TEAE; 
x 
At least one TEAE leading to Discontinuation from Study Medication; 
x 
At least one TEAE of Special Interest; 
x 
At least one Treatment-Related TEAE of Special Interest 
Where not otherwise noted, all the following summaries are presented by PT:  
x 
All TEAEs (by SOC and PT); 
x 
Treatment-Related TEAEs (i.e., possibly or probably related); 
x 
TEAEs occurring in 2% or more of the population; 
x 
All TEAEs by SOC, PT, and maximum severity (where the maximum intensity per 
patient will be counted at each level of summarization);  
x 
TEAES with Grade ≥ 3; 
x 
Treatment-Related TEAES with Grade ≥ 3; 
x 
Serious TEAEs; 
x 
Treatment-Related Serious TEAEs; 
x 
Fatal TEAEs; 
x 
TEAEs leading to Discontinuation from Study Treatment; 
x 
TEAE of Special Interest (by protocol-defined AESI category); 
x 
Treatment-Related TEAE of Special Interest (by protocol-defined AESI category); 
x 
Non-fatal TEAEs; 
x 
Hypertension TEAEs by Pre-existing Hypertension (Yes vs No) and Baseline 
Hypertension (Yes vs. No). Hypertension TEAEs will be selected as any TEAE with a 
preferred term of Hypertension.  


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In addition, a summary of all TEAEs by PT occurring in at least 2% of subjects will be created 
and ordered by descending overall frequency. 
Adverse events of special interest for this study include:  
x 
Adverse events consistent with urinary retention 
x 
Adverse events suggestive of cystitis or urinary tract infection (UTI)  
x 
Potential major cardiac and cerebrovascular events, including death (or any event with 
fatal outcome), myocardial infarction, cerebrovascular accident, hospitalization for 
unstable angina or chest pain, hospitalization for heart failure requiring hospitalization, 
and coronary revascularization/angioplasty/stent  
x 
Adverse events consistent with hypertension (see protocol section 8.4.6) 
x 
Adverse events consistent with orthostatic hypotension as confirmed by orthostatic vital 
signs  
x 
Elevated serum aspartate aminotransferase (AST) or alanine aminotransferase (ALT) lab 
value requiring that study drug be temporarily withheld or permanently discontinued  
x 
Neoplasms 
Treatment listings will include the planned treatment duration, start and stop dates/times of the 
AE, study day (relative to start of double-blind treatment on URO-901-3005) and treatment day 
(relative to the day of first dose of vibegron). A treatment related AE is defined as an AE for 
which the investigator classifies the AE as being “Probably Related” or “Possibly Related” to 
study treatment on the Adverse Event CRF. Missing relationship and severity (intensity) will be 
imputed per Section 4.2.3. 
 
The following additional listings will be provided: 
x 
Listing of Deaths 
x 
Listing of Serious TEAEs 
x 
Listing of Treatment-Emergent AESIs 
x 
Listing of TEAEs Leading to Withdrawal or Interruption of Study Treatment 
x 
Listing of all AEs with a flag for TEAEs and onset (Prior = prior to first dose of vibegron 
or Treatment = on or after first dose of vibegron.) 
x 
Listing of all medical history including pre-treatment adverse events with coded preferred 
terms in the hypertension AE search list: 
 Accelerated hypertension 
 Diastolic hypertension 
 Essential hypertension 
 Hypertension 


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 Hypertensive crisis 
 Hypertensive emergency 
 Hypertensive heart disease 
 Malignant hypertension 
 Malignant hypertensive heart disease 
 Secondary hypertension 
 Supine hypertension 
 Systolic hypertension. 
7.4. 
Laboratory Evaluations 
All continuous laboratory parameters will be summarized descriptively by absolute value at each 
visit together with the corresponding changes from baseline. All parameters will be summarized 
in SI units. 
The number and percentage of subjects with laboratory measurements outside of the central 
laboratory normal range will also be summarized by visit. Shift tables from baseline to maximum 
post-baseline value, to minimum post-baseline value, last post-baseline value, and at each post-
baseline visit will be provided for hematology and chemistry parameters to display low, normal, 
high, and missing values by treatment group in a 3-by-3 contingency table. Denominators for 
percentages will be the number of subjects with non-missing data at the specific assessment and 
baseline. 
ALT/AST and Total Bilirubin will be displayed as eDISH plots. Maximum post-baseline total 
bilirubin will be presented (<2 and ≥2 x ULN) and plotted against maximum post-baseline ALT 
(<3, ≥3 - <5, ≥5-<10, and ≥10 x ULN), expressed as multiples of ULN. This will be repeated to 
show maximum post-baseline total bilirubin against maximum post-baseline AST. 
Data for subjects with ALT or AST ≥3 x ULN, and bilirubin ≥2 x ULN will be presented, which 
will include all visits for this subset of subjects. A line plot of liver biochemistry test results 
(including ALP, ALT, AST, total bilirubin, and GGT) over time will also be presented for this 
subset of patients. 
A sample for the urinalysis and urine culture will be sent to the central laboratory only if the 
urine dipstick performed at the site tests positive for the presence of leukocytes, nitrites, or blood 
cells. Central urinalysis data will be provided only in the listing. 
Any data outside the central laboratory normal reference ranges will be explicitly noted on the 
listings that are produced.  


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7.5. 
Vital Sign Assessments 
Vital sign data including blood pressure, pulse rate, respiration rate, body temperature and 
weight will be collected at all study visits except for the Week 1, Week 16, and 44 telephone 
visits. Height will be measured at Screening only. Blood pressure will be measured in triplicate 
at each visit. The average of triplicates will be used for summary.  For subjects in URO-901-
3005 Study Part 1 at any visits with orthostatic blood pressure measurements: blood pressure (in 
triplicate) and pulse rate taken pre-dose in sitting position are equivalent to all other pre-dose 
vital sign values and will be used. Orthostatic and standing blood pressure measurements 
collected during Part 1 of URO-901-3005 will not be included in any summaries on URO-901-
3006. 
For all parameters, absolute values and change from baseline will be presented for scheduled 
visits using descriptive statistics for continuous variables. In addition, the maximum post-
baseline change from baseline value and change from baseline to the end of treatment will be 
summarized for each of the vital signs. 
To further investigate changes in SBP/DBP/pulse rate from baseline, the following tables will be 
prepared:  
x Counts and percent of subjects in each group with at least a 5/10/15 CFB at 2 
consecutive post-baseline visits and at Week 52 will be produced for all subjects, by pre-
existing hypertension category (Yes/No) and by pre-existing hypertension category 
(Yes/No).  
x Maximum post-baseline CFB (including 95% CI of Mean) 
The line plot of mean (SE) of change from baseline in blood pressure and pulse rate over time 
will be prepared. A by-subject listing, sorted by subject identifier, will be presented including all 
vital sign results (scheduled or unscheduled).  
7.6. 
Physical Examination 
Brief physical examination data will be collected at Screening, Baseline, Week 12, 24, and 52. A 
listing of all physical exam results for subjects with at least one abnormal result will be 
produced.  
7.7. 
Post-Void Residual (PVR) Urine Volume 
PVR urine volume data will be summarized at Baseline and every visit. The summary will 
comprise a continuous summary at each visit, including change from baseline, and a categorical 
summary of PVR at the following categories: <100 mL, ≥ 100 and < 200 mL, ≥ 200 and < 350 
mL, and ≥ 350 mL. All PVR data will be listed. 
7.8. 
IPSS Total Score 
IPSS total score at Screening, Day 1, Weeks 4, 8, 12, 24, 36, and 52 for the 52-week treatment 
group and baseline, Week 12 and Week 28 for the 28-week treatment group will be summarized 
by treatment group and visit, including change from baseline.  


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8. 
COVID-19 CONSIDERATIONS 
This study was conducted during the COVID-19 global pandemic. During the time period of the 
pandemic, it is anticipated that changes in study visit schedules, missed visits, or patient 
discontinuations may lead to missing information (e.g., for protocol-specified procedures). In 
accordance with FDA guidance on statistical considerations for clinical trials during the COVID-
19 public health emergency [3], it is important to capture specific information in the case report 
form that explains the basis of the missing data, including the relationship to COVID-19 for 
missing protocol-specified information (e.g., from missed study visits or study discontinuations 
due to COVID-19).  
The proportion of subjects with COVID-19 impact on visits will be summarized by treatment 
group overall and by visit. The reasons (e.g. subject acquired COVID-19, subject unable to travel 
due to COVID-19, investigative site closure due to COVID-19, etc.) will also be summarized. In 
addition, subjects who discontinued treatment due to COVID-19 will be summarized with the 
COVID-19 reason for discontinuation. A listing of subjects who had COVID-19 information 
collected will be generated which will document the date of contact, the reason for the telephone 
follow-up, the visit that was missed, and if any doses were missed due to IP availability. 
9. 
CHANGE FROM ANALYSIS PLANNED IN PROTOCOL 
9.1. 
Secondary Efficacy 
CFB in average IPSS Storage score has been changed to CFB in IPSS Storage score. 
9.2. 
Exploratory Endpoints 
CFB at all URO-901-3005 or URO-901-3006 protocol-specified timepoints apart from 
 
have been added for all secondary efficacy endpoints. 
9.3. 
COVID-19 Considerations 
The COVID-19 global pandemic began during the conduct of the study. The recommended 
impact analysis was therefore added to the SAP, as the operational change of increased visit 
windows had been communicated via study memo with no protocol amendment. 
9.4. 
Physical Examination 
Physical examination was removed from primary objectives; any clinically significant finding is 
reported by the investigator as an adverse event and is summarized with adverse events. Physical 
examination results will be only listed. 
10. 
REFERENCES 
 [1] Jiang YH, Lin VC, Liao CH, Kuo HC. International Prostatic Symptom Score-
voiding/storage subscore ratio in association with total prostatic volume and maximum flow rate 
is diagnostic of bladder outlet-related lower urinary tract dysfunction in men with lower urinary 
tract symptoms. PLoS One. 2013;8(3):e59176.  


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[2] 
 crosswalk value set https://euroqol.org/eq-5d-instruments/
about/valuation-standard-value-sets/crosswalk-index-value-calculator/ 
[3] Statistical Considerations for Clinical Trials During the COVID-19 Public Health Emergency 
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/statistical-
considerations-clinical-trials-during-covid-19-public-health-emergency-guidance-industry 
11. 
APPENDIX 
11.1. 
Table of Contents for Data Display Specifications 
11.1.1. 
Output Tables 
Table 9: 
List of Output Tables  
Title 
Population 
 
Programming notes 
Study Population  
14.1.1.1 
Subject Disposition 
Screened 
Include two sections: 
screened, treated  
14.1.1.2 
Enrollment by Country and Investigator 
SAF-Ext 
 
14.1.2.1 
Major Protocol Deviations 
FAS-Ext 
 
14.1.2.2 
Reasons for Exclusion from Analysis Sets 
SAF-Ext 
 
14.1.3.1 
Subject Demographic and Baseline Characteristics 
SAF-Ext 
 
14.1.3.2 
Subject Demographic and Baseline Characteristics 
FAS-Ext 
 
14.1.3.3 
Subject Demographic and Baseline Characteristics 
FAS-Ext-I 
 
14.1.3.4 
Subject Demographic and Baseline Characteristics 
PPS-Ext 
 
14.1.3.5 
Subject Demographic and Baseline Characteristics 
PPS-Ext-I 
 
14.1.3.8 
Other Baseline Characteristics 
FAS-Ext 
 
14.1.3.9 
Other Baseline Characteristics 
FAS-Ext-I 
 
14.1.3.10 
Other Baseline Characteristics 
PPS-Ext 
 


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Title 
Population 
 
Programming notes 
14.1.3.11 
Other Baseline Characteristics 
PPS-Ext-I 
 
14.1.4 
Medical History 
SAF-Ext 
 
14.1.5.1 
Prior Non-Overactive-Bladder (OAB) Medication 
SAF-Ext 
 
14.1.5.2 
Prior Overactive Bladder (OAB) Medication 
SAF-Ext 
 
14.1.6.1 
Non-OAB Concomitant Medication  
SAF-Ext 
 
14.1.6.2 
OAB Concomitant Medication  
SAF-Ext 
 
14.1.7.1 
COVID-19 Impact 
SAF-Ext 
 
Efficacy Endpoints 
Secondary Efficacy Endpoints 
 
 
14.2.1.1 
Average Daily Number of Micturition Episodes  
FAS-Ext 
Descriptive statistics, 
include CFB 
14.2.1.2 
Change from Baseline in Daily Average Number 
of Micturition Episodes (MMRM) 
FAS-Ext 
Include all intervals, 
but present Week 52 
first 
14.2.1.3 
Change from Baseline in Daily Average Number 
of Micturition Episodes (MMRM) 
PPS-Ext 
Include all intervals, 
but present Week 52 
first 
14.2.2.1 
Average Daily Number of Urgency Episodes  
FAS-Ext 
Descriptive statistics, 
include CFB 
14.2.2.2 
Change from Baseline in Average Daily Number 
of Urgency Episodes (MMRM) 
FAS-Ext 
Include all intervals, 
but present Week 52 
first 
14.2.2.3 
Change from Baseline in Average Daily Number 
of Urgency Episodes (MMRM) 
PPS-Ext 
Include all intervals, 
but present Week 52 
first 
14.2.3.1 
Average Number of Nocturia Episodes per Day 
FAS-Ext 
Descriptive statistics, 
include CFB 


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Title 
Population 
 
Programming notes 
14.2.3.2 
Change from Baseline in Average Number of 
Nocturia Episodes per Day (MMRM) 
FAS-Ext 
Include all intervals, 
but present Week 52 
first 
14.2.3.3 
Change from Baseline in Average Number of 
Nocturia Episodes per Day (MMRM) 
PPS-Ext 
Include all intervals, 
but present Week 52 
first 
14.2.4.1 
Average Daily Number of Urge Urinary 
incontinence Episodes  
FAS-Ext-I 
Descriptive statistics, 
include CFB 
14.2.4.2 
Change from Baseline in Average Daily Number 
of Urge Urinary incontinence Episodes (MMRM) 
FAS-Ext-I 
Include all intervals, 
but present Week 52 
first 
14.2.4.3 
Change from Baseline in Average Daily Number 
of Urge Urinary incontinence Episodes (MMRM) 
PPS-Ext-I 
Include all intervals, 
but present Week 52 
first 
14.2.5.1 
International Prostate Symptom Score (IPSS) 
Storage Score 
FAS-Ext 
Descriptive statistics, 
include CFB 
14.2.5.2 
Change from Baseline in International Prostate 
Symptom Score (IPSS) Storage Score (MMRM) 
FAS-Ext 
Include all intervals, 
but present Week 52 
first 
14.2.5.3 
Change from Baseline in International Prostate 
Symptom Score (IPSS) Storage Score (MMRM) 
PPS-Ext 
Include all intervals, 
but present Week 52 
first 
14.2.6.1 
Average Volume Voided per Micturition 
FAS-Ext 
Descriptive statistics, 
include CFB 
14.2.6.2 
Change from Baseline in Average Volume Voided 
per Micturition (MMRM) 
FAS-Ext 
Include all intervals, 
but present Week 52 
first 
14.2.6.3 
Change from Baseline in Average Volume Voided 
per Micturition (MMRM) 
PPS-Ext 
Include all intervals, 
but present Week 52 
first 
Other Efficacy and PROs Endpoints 
 
 






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Title 
Population 
 
Programming notes 
14.3.2.4 
Treatment-Emergent Adverse Events by Preferred 
Term and Maximum Intensity 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.5 
Treatment Emergent Adverse Events with Grade ≥ 
3 by Preferred Term 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.6 
Treatment-Related Emergent Adverse Events with 
Grade ≥ 3 by Preferred Term 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.7 
Treatment-Emergent Adverse Events occurring in 
≥ 2% subjects by Preferred Term 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.8 
Serious Treatment Emergent Adverse Events by 
Preferred Term 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.9 
Treatment-Related Serious Treatment Emergent 
Adverse Events by Preferred Term 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.10 
Treatment Emergent Adverse Events Leading to 
Discontinuation from Study Treatment by 
Preferred Term 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.11 
 Fatal TEAEs by Preferred Term 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.12 
Treatment Emergent Adverse Events of Special 
Interest by Preferred Term 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.13 
Treatment-Related Treatment Emergent Adverse 
Events of Special Interest by Preferred Term 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.14 
Non-Fatal Treatment-Emergent Adverse Events by 
Preferred Term 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.15 
Hypertension Treatment Emergent Adverse Events 
by Preferred Term, and Pre-Existing Hypertension 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.16 
Hypertension Treatment Emergent Adverse Events 
by Preferred Term, and Baseline Hypertension 
SAF-Ext 
Descending frequency 
of preferred term 
14.3.2.17 
Listing of Deaths 
SAF-Ext 
 


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Title 
Population 
 
Programming notes 
14.3.2.18 
Listing of Treatment Emergent Serious Adverse 
Events 
SAF-Ext 
 
14.3.2.19 
Listing of Treatment Emergent Adverse Events 
Leading to Withdrawal or Interruption of Study 
Treatment 
SAF-Ext 
 
14.3.2.20 
Listing of TEAEs of Special Interest 
SAF-Ext 
 
Safety Labs 
 
 
14.3.3.1 
Hematology Laboratory Parameters 
SAF-Ext 
Include observed and 
CFB 
14.3.3.2 
Clinical Chemistry Laboratory Parameters 
SAF-Ext 
Include observed and 
CFB 
14.3.3.3 
Urinalysis Laboratory Parameters 
SAF-Ext 
Include observed and 
CFB 
14.3.3.4 
Summary of Other Laboratory Parameters 
SAF-Ext 
 
14.3.3.5 
Abnormal Classification of Hematology 
Laboratory Parameters 
SAF-Ext 
 
14.3.3.6 
Abnormal Classification of Clinical Chemistry 
Laboratory Parameters 
SAF-Ext 
 
14.3.3.7 
Abnormal Classification of Urinalysis Laboratory 
Parameters 
SAF-Ext 
 
14.3.3.8 
Abnormal Classification Summary of Other 
Laboratory Parameters 
SAF-Ext 
 
14.3.3.9 
Shift Table of L/N/H Classification for 
Hematology Laboratory Parameters 
SAF-Ext 
 
14.3.3.10 
Shift Table of L/N/H Classification for Chemistry 
Laboratory Parameters 
SAF-Ext 
 
14.3.3.11 
Maximum Post-baseline ALT and AST vs. 
Maximum Post-baseline Bilirubin 
SAF-Ext 
 


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Title 
Population 
 
Programming notes 
14.3.3.12 
Listing of Subjects who Potentially Met Hy’s Law 
SAF-Ext 
 
14.3.3.13 
Summary of Liver Function Laboratory Findings 
SAF-Ext 
 
Vital Signs 
 
 
14.3.4.1 
Vital Sign Parameters 
SAF-Ext 
Include all observed 
and CFB 
14.3.4.2 
Vital Sign Parameter Change from Baseline Shifts 
at 3 Consecutive Visits 
SAF-Ext 
SBP, DBP and PR 
14.3.4.3 
Vital Sign Parameter Change from Baseline Shifts 
at Week 52 
SAF-Ext 
SBP, DBP and PR 
14.3.4.4 
Vital Sign Parameter Maximum Post-Baseline 
Change from Baseline  
SAF-Ext 
SBP, DBP and PR 
Other 
 
 
14.3.5.1 
Post-Void Residual Volume 
SAF-Ext 
 
14.3.6.1 
Total IPSS score 
SAF-Ext 
 
14.3.7.1 
Physical Examination Shift from Baseline 
SAF-Ext 
 
 
11.1.2. 
Output Figures 
Table 10: 
List of Output Figures 
Title 
Population    Programming notes 
Efficacy Endpoints 
 
 
14.2.1.1 
Plot of LS Means (SE) of Change from Baseline in 
Average Daily Number of Micturition Episodes 
(MMRM) 
FAS-Ext 
Including all visits 
14.2.2.1 
Plot of LS Means (SE) of Change from Baseline in 
Average Daily Number of Urgency Episodes 
(MMRM) 
FAS-Ext 
Including all visits 


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Title 
Population    Programming notes 
14.2.3.1 
Plot of LS Means (SE) of Change from Baseline in 
Average Number of Nocturia Episodes per Day 
(MMRM) 
FAS-Ext 
Including all visits 
14.2.4.1 
Plot of LS Means (SE) of Change from Baseline in 
Average Number of Urge Urinary incontinence 
Episodes per Day (MMRM) 
FAS-Ext-I 
Including all visits 
14.2.5.1 
Plot of LS Means (SE) of Change from Baseline in 
International Prostate Symptom Score (IPSS) Storage 
Score (MMRM) 
FAS-Ext 
Including all visits 
14.2.6.1 
Plot of LS Means (SE) of Change from Baseline in 
Volume Voided per Micturition (MMRM) 
FAS-Ext 
Including all visits 
Safety Endpoints 
 
 
14.3.3.1 
Scatter Plot of Maximum ALT Post-Baseline versus 
Maximum Total Bilirubin Expressed as Multiples of 
ULN 
SAF-Ext 
 
14.3.3.2 
Scatter Plot of Maximum AST Post-Baseline versus 
Maximum Total Bilirubin Expressed as Multiples of 
ULN 
SAF-Ext 
 
14.3.3.3 
Line Plot of Liver Chemistry Test Results over Time 
for Subjects with Elevated ALT or AST, and Elevated 
Total Bilirubin at Any Time 
SAF-Ext 
Only produce for 
subjects who 
potentially met Hy’s 
law 
14.3.4.1 
Line Plot of Mean (SE) in CFB of Systolic Blood 
Pressure  
SAF-Ext 
X-axis starts from 
baseline visit  
14.3.4.2 
Line Plot of Mean (SE) in CFB of Diastolic Blood 
Pressure 
SAF-Ext 
X-axis starts from 
baseline visit  
14.3.4.3 
Line Plot of Mean (SE) in CFB of Pulse Rate 
SAF-Ext 
X-axis starts from 
baseline visit  
14.3.5.1 
Line Plot of Mean (SE) Post-Void Residual Urine 
Volume 
SAF-Ext 
X-axis starts from 
baseline visit, separate 
lines for treatment 
groups 


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Title 
Population    Programming notes 
14.3.6.1 
Line Plot of Mean (SE) IPSS Total Scores 
SAF-Ext 
X-axis starts from 
baseline visit, separate 
lines for treatment 
groups 
 
11.1.3. 
Output Listings 
Table 11: 
List of Output Listings 
Title 
Population Programming notes 
Disposition and Demographics 
 
 
16.2.1.1 
Subject Disposition – Screen Failures 
Screened 
 
16.2.1.2 
Eligibility Criteria-Inclusion/Exclusion Criteria – 
Screen Failures 
Screened 
 
16.2.1.3 
Subject Disposition 
SAF-Ext 
 
16.2.1.4 
Protocol Deviation 
FAS-Ext 
 
16.2.1.5 
Exclusion from Analysis Sets 
SAF-Ext 
 
16.2.1.6 
Demographic and Baseline Characteristics 
SAF-Ext 
 
16.2.1.7 
Medical History  
SAF-Ext 
 
16.2.1.8 
Procedures 
SAF-Ext 
 
16.2.1.9 
Prior Non-OAB Medications 
SAF-Ext 
Separate from prior 
OAB medication 
listing. 
16.2.1.10 
Concomitant Medications 
SAF-Ext 
 
16.2.1.11 
Prior OAB Medication  
SAF-Ext 
 
16.2.1.12 
Treatment Administration 
SAF-Ext 
 
16.2.1.13 
Study Drug Accountability 
SAF-Ext 
 
16.2.1.14 
Study Drug Compliance 
SAF-Ext 
 
16.2.1.15 
COVID-19 Impact 
SAF-Ext 
 
Efficacy  
 
 
16.2.2.1 
Derived Average Bladder Diary Parameters  
FAS-Ext 
 
16.2.2.2 
Derived Responder Parameters from Bladder Diary 
FAS-Ext 
 


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Title 
Population Programming notes 
16.2.2.3 
Derived Parameters from International Prostate 
Symptom Score (IPSS) Data 
FAS-Ext 
 
16.2.2.4 
FAS-Ext 
 
16.2.2.5 
FAS-Ext 
 
16.2.2.6 
Global Impression Data 
FAS-Ext 
 
16.2.2.7 
FAS-Ext 
 
 
 


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Safety 
 
 
16.2.3.1 
 All Adverse Events 
SAF-Ext 
 
16.2.3.2 
All Adverse Events Prior to Treatment 
Screened 
 
16.2.3.3 
All Adverse Events During Treatment 
SAF-Ext 
 
16.2.3.4 
Hematology Laboratory Parameters 
SAF-Ext 
 
16.2.3.5 
Clinical Chemistry Laboratory Parameters 
SAF-Ext 
 
16.2.3.6 
Urinalysis Laboratory Parameters 
SAF-Ext 
 
16.2.3.7 
Other Laboratory Parameters 
SAF-Ext 
 
16.2.3.8 
Vital Signs 
SAF-Ext 
 
16.2.3.8 
 Physical Examination 
SAF-Ext 
 
16.2.3.9 
Post-Void Residual (PVR) Volume  
SAF-Ext