BELLUS Health — Investor Presentation (September 2022)
39 slides from BELLUS Health's investor presentation, Sep 16, 2022
Slide 1: Investor Presentation
Investor Presentation NASDAQ/TSX - BLU September 16 th , 2022
Slide 2: Forward Looking Statements
Forward Looking Statements 2 Certain statements contained in this presentation, other than statements of fact that are independently verifiable at the dat e h ereof, may constitute "forward - looking statements" within the meaning of Canadian securities legislation and regulations, the U.S. Private
Slide 3: Bellus Company Overview
(e) Bellus _ Company Overview
Slide 4: BELLUS Health - A Leader in the Development of the Novel P2X3 Antagonist Class
BELLUS Health - A Leader in the Development of The Novel P2X3 Antagonist Class Targeting Cough Hypersensitivity Financials Key Upcoming Events Lead Indication - Refractory Chronic Cough (RCC) • Positive FDA End - of - Phase 2 meeting with clear path forward for BLU - 5937’s Phase 3 CALM program in R
Slide 5: Strong Leadership and Advisory Group
Strong Leadership and Advisory Group Management Board of Directors Clinical Advisory Board Dr. Surinder Birring, MB ChB (Hons), MD King’s College London Dr. Michael S. Blaiss, MD Medical College of Georgia Dr. Peter Dicpinigaitis, MD Albert Einstein Medical College Dr. Jacky Smith (Chair) , MB, ChB,
Slide 6: Refractory Chronic Cough
Refractory Chronic Cough Cough lasting ≥ 8 weeks that does not respond to treatment for underlying cause or is unexplained 1 No approved treatment , current options are inadequate and non - specific 3 Significant impact on patients ’ quality of life, including impact on social, physical and psychoso
Slide 7: P2X3 Receptor - Linked to Cough & Other Hypersensitization Disorders
P2X3 Receptor - Linked to Cough & Other Hypersensitization Disorders • Afferent neurons in peripheral nervous system express P2X3 receptor 2 - 7 • Activation of P2X3 triggers neuronal hypersensitization 8,9 , proposed to play a role in 1. Ford et al. (2013) Front Cell Neurosci. 7:267 . 2. Shiers et
Slide 8: Best-In-Class Potential
BLU - 5937 - Best - In - Class Potential 8 Compelling efficacy from SOOTHE Phase 2b trial completed in December 2021 Well - tolerated with a low rate of class - related taste side effects Twice daily dosing with once - daily dosing in development BLU - 5937 Highly potent P2X3 antagonist with best -
Slide 9: Pipeline
BLU - 5937 - Pipeline 9 PROGRAM DEVELOPMENT STATUS Indication / Project Preclinical Phase 1 Phase 2 Phase 3 Worldwide Rights Next Anticipated Step BLU - 5937 Refractory Chronic Cough (BID Formulation) Q4 2022: Start of CALM Phase 3 Program Refractory Chronic Cough (QD Formulation) Q4 2022: Phase 1 T
Slide 10: Bellus SOOTHE Phase 2b Results
Bellus SOOTHE Phase 2b Results
Slide 11: SOOTHE Trial Design
SOOTHE Trial Design 11 Randomized, double - blind, 4 - week placebo - controlled parallel arm study with 3 active doses PRIMARY ENDPOINT Placebo - adjusted change from baseline in 24H cough frequency (Day 28) POPULATION Refractory chronic cough for ≥1 year Awake cough frequency: ≥25 coughs/h 249 par
Slide 12: SOOTHE: Primary Efficacy Endpoint
SOOTHE: Primary Efficacy Endpoint Placebo - Adjusted Change in 24H Cough Frequency 12 34% placebo - adjusted reduction in 24 - hour cough frequency at 50 mg and 200 mg BID doses (p ≤ 0.005) Dose response observed between 12.5 mg and 50 mg BID doses -60 -40 -20 0 20 40 60 Dose Δ p - value 12.5 mg - 2
Slide 13: SOOTHE: Change from Baseline in 24H Cough Frequency
-60.0% -50.0% -40.0% -30.0% -20.0% -10.0% 0.0% Placebo 12.5 mg BID 50 mg BID 200 mg BID Relative change from baseline in 24H cough frequency (ITT) Baseline Day 15 Day 28 SOOTHE: Change from Baseline in 24H Cough Frequency 13 Percentage change in 24H cough frequency 53% reduction from baseline in 24
Slide 14: SOOTHE: Responder Rates in 24H Cough Frequency
36% 14% 7% 52% 35% 19% 61% 44% 25% 62% 48% 19% 0% 10% 20% 30% 40% 50% 60% 70% 80% ≥ 30% ≥ 50% ≥ 70% Placebo BLU-5937 12.5 mg BID BLU-5937 50 mg BID BLU-5937 200 mg BID Day 28 12.5 mg BID 50 mg BID 200 mg BID -1 1 3 5 7 9 11 13 15 p=0.0865 p=0.0077 p=0.0080 ≥ 30% -1 1 3 5 7 9 11 13 15 p=0.0147 p=0.00
Slide 15: SOOTHE: Secondary Endpoints
SOOTHE: Secondary Endpoints Patient Reported Outcomes (PRO): Cough Severity Visual Analog Scale (CS - VAS) and Leicester Cough Questionnaire (LCQ) Clinically meaningful and statistically significant benefit of BLU - 5937 at multiple time points in patient reported outcomes LCQ: scale of 3 - 21; High
Slide 16: SOOTHE: Safety and Tolerability
SOOTHE: Safety and Tolerability 16 Generally well - tolerated S imilar rate of treatment emergent adverse events (TEAEs) reported for placebo and BLU - 5937 n (%) Placebo (n= 63) BLU - 5937 12.5 mg BID (n= 62) BLU - 5937 50 mg BID (n= 62) BLU - 5937 200 mg BID (n= 62) Subjects with ≥1 TEAE 22 (34.9%
Slide 17: SOOTHE: Low Taste-Related Adverse Events
SOOTHE: Low Taste - Related Adverse Events Associated to P2X3 Class 17 Low rate of taste disturbance adverse events at all doses (≤ 6.5%) with: • No loss of taste • No discontinuations due to taste disturbance INCIDENCE OF TASTE DISTURBANCE ADVERSE EVENTS Placebo (n= 63 ) BLU - 5937 12.5 mg BID (n=
Slide 18: Bellus CALM Phase 3 Program Plans
Bellus CALM Phase 3 Program Plans
Slide 19: CALM Program: Study Design
CALM Program: Study Design Two randomized, double - blind, placebo - controlled parallel arm trials with 2 active doses 12 weeks CALM - 1 Run In Screening Randomization Primary Randomized Treatment Period 50 mg BID –– n≈225 25 mg BID –– n≈225 Placebo BID –– n≈225 40 weeks Randomized Blinded Extensio
Slide 20: CALM Program: Enrichment Strategy
CALM Program: Enrichment Strategy 20 CALM - 1 and CALM - 2 populations to be enriched for baseline cough frequency PRIMARY EFFICACY ENDPOINT: COUGH FREQUENCY IN PRIMARY POPULATION (90% POWER) • 24H cough frequency vs placebo in Primary Population SECONDARY EFFICACY ENDPOINTS (80% POWER ) • Leicester
Slide 21: CALM Program: Dose Selection
CALM Program 3: Dose Selection Well supported clinical rationale for 25 and 50mg BID dose selection: • No dose response observed between 25 and 200mg BID in RELIEF Ph2a study • 50mg BID lowest optimal dose in SOOTHE Phase 2b study • 50 and 200mg BID achieved 34% placebo - adjusted reduction in cough
Slide 22: CALM Program: Clinical Development Milestones and Planned Next Steps
CALM Program: Clinical Development Milestones and Planned Next Steps 22 Topline results from CALM - 1 Phase 3 trial expected in 2H 2024 December 13 , 2021 SOOTHE Phase 2b Topline Results 2H 2021 2022 PH 2B June 2022 End - of - Phase 2 Meeting with FDA 4Q 2022 Expected First Patients Enrolled in Phas
Slide 23: Market and Competitive Landscape
Market and Competitive Landscape
Slide 24: The Market for Refractory Chronic Cough in Key Regions
The Market for Refractory Chronic Cough in Key Regions 24 CHRONIC COUGH patients in key geographies • ~ 10% prevalence in the U.S. & Europe - 5 1 • ~ 4% prevalence in China • Refractory chronic cough patients represent an important segment of the chronic cough population 1 : REFRACTORY CHRONIC COUGH
Slide 25: P2X3 Competitive Landscape
25 P2X3 Competitive Landscape 1 Company Candidate Gefapixant Sivopixant BLU - 5937 Stage of Development Approved in Japan, Switzerland Phase 2b Phase 2b Expected Next Steps Resubmission in U.S. * ; EU under review Planning next clinical trial Phase 3 in Q4 2022 Dosing BID QD BID / QD in development
Slide 26: First-in-Class P2X3 Antagonist, Merck's Gefapixant
First - in - Class P2X3 Antagonist, Merck’s MK - 7264 ( gefapixant ) 26 18% & 15 % Cough 1 MK - 7264 58% & 69% Taste AEs 1 Placebo - adjusted reduction in 24H cough frequency (primary endpoint) of patients have taste alteration and/or taste loss Two Phase 3 Trials of gefapixant: COUGH - 1 (12 week d
Slide 27: Shionogi's Sivopixant
Shionogi’s S - 600918 (sivopixant) 27 12 % Cough 1 Sivopixant 33% Taste AEs 1 Placebo - adjusted reduction in 24H cough frequency (primary endpoint) of patients with taste alteration and/or taste loss Phase 2b Trial (4 week duration) 300mg QD Selective P2X3 antagonist Three doses tested with none ac
Slide 28: Well-Positioned for Potential Class Differentiation
BLU - 5937 Well - Positioned for Potential Class Differentiation Source: Bellus Health, BLU - 5937 Commercial Opportunity Assessment Criteria • Treatment effect vs. placebo • Taste effects • Positive Phase 2b results with potential best in class efficacy and tolerability • Best - in - class selectiv
Slide 29: U.S. Commercialization Strategy
U.S. Commercialization Strategy
Slide 30: Established Unmet Need and Willingness to Rx in RCC with Specialists
91 % 30 25 M 8.6 M Chronic Cough Refractory Chronic Cough Established Unmet Need and Willingness to Rx in RCC with Specialists 2 Source: ZS Associates/Bellus Health Market Research (2022) 1. All - comer survey (n=1483) of US Pulmonologists (n= 289) , Allergists (n= 207) , Otolaryngologists (n= 217)
Slide 31: Solid Foundation for U.S. P2X3 Market Success
31 EXPECTED BY LAUNCH OF BLU - 5937 EXISTS NOW High Treatment Rate Medical Education Awareness & Guidelines Payer Track Record of Reimbursing Poor Quality - of - Life Conditions Prescription Patterns and Physician Experience with P2X3 Intent to Use P2X3 P2X3 Pricing and Access Established with Payer
Slide 32: Early U.S. Commercialization Strategy
32 • Leverage potential best in class profile to target P2X3 naïve and first - generation switchers / discontinuations • 150 - 175 sales representatives targeting top 30% respiratory specialists • Multichannel consumer outreach, referral, and telemedicine platforms • Achieve optimal payer access • C
Slide 33: Potential Additional Indications
BLU - 5937 Potential Additional Indications
Slide 34: Cough Hypersensitivity in Additional Cough Indications
Cough Hypersensitivity In Additional Cough Indications 34 Success of SOOTHE supports potential evaluation of BLU - 5937 in other cough populations Cough is an important health burden • Across the U.S. in 2018, cough was the reason for 1 : - 18.5M in - office physician consultations - 5M emergency vi
Slide 35: Bellus IP and Corporate Summary
Baus IP and Corporate Summary
Slide 36: 100% Owned Intellectual Property Portfolio
100% Owned Intellectual Property Portfolio 36 N N X N R 1 R 3 O N O R 9 R 2 R 4 R 6 R 5 R 8 R 7 • All intellectual property 100% owned by BELLUS with no future obligations owed • U.S. and international patent estate covering BLU - 5937 and related compounds • Composition of matter patent for BLU - 5
Slide 37: Stock and Financial Information
Stock and Financial Information 37 125.8M basic shares 136.8M fully diluted shares Capital Structure Cash, cash equivalent and short - term investments position of US$384.6M* Pro Forma Cash Position * Cash position as of June 30, 2022 plus approx. net proceeds from July 2022 raise
Slide 38: Expected Milestones
Expected Milestones Potential Catalysts & Upcoming Events 38 BLU - 5937 in Refractory C hronic C ough BLU - 5937 Platform FDA End - of - Phase 2 Meeting (June 2022) EMA Feedback (Q3 2022) Final Protocols (Q3 2022) CALM - 1 & CALM - 2 trial initiations (Q4 2022) Once - daily extended release formulat
Slide 39: Investor Contact
Investor Contact: Ramzi Benamar CFO [email protected]
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