Post on 20-Mar-2017
Corporate Presentation (TSX: BLU)
Roberto BelliniPresident and Chief Executive OfficerTwitter: @rbellini
March 17, 2017
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Forward Looking StatementsCertain statements contained in this news release, other than statements of fact that are independentlyverifiable at the date hereof, may constitute “forward-looking statements” within the meaning of Canadiansecurities legislation and regulations. Such statements, based as they are on the current expectations ofmanagement, inherently involve numerous important risks, uncertainties and assumptions, known andunknown, many of which are beyond BELLUS Health Inc.'s control. Such risks factors include but are notlimited to: the ability to obtain financing, the impact of general economic conditions, general conditions in thepharmaceutical industry, changes in the regulatory environment in the jurisdictions in which BELLUS HealthInc. does business, stock market volatility, fluctuations in costs, changes to the competitive environment dueto consolidation, achievement of forecasted burn rate, potential payments/outcomes in relation to indemnityagreements and contingent value rights, achievement of forecasted pre-clinical and clinical trial milestonesand that actual results may vary once the final and quality-controlled verification of data and analyses hasbeen completed. In addition, the length of BELLUS Health Inc.’s drug candidates development process, theirmarket size and commercial value, as well as the sharing of proceeds between BELLUS Health Inc. and itspotential partners from potential future revenues, if any, are dependent upon a number of factors.Consequently, actual future results and events may differ materially from the anticipated results and eventsexpressed in the forward-looking statements. The Company believes that expectations represented byforward-looking statements are reasonable, yet there can be no assurance that such expectations will proveto be correct. The reader should not place undue reliance, if any, on any forward-looking statements includedin this news release. These forward-looking statements speak only as of the date made, and BELLUS HealthInc. is under no obligation and disavows any intention to update publicly or revise such statements as a resultof any new information, future event, circumstances or otherwise, unless required by applicable legislation orregulation. Please see BELLUS Health Inc.’s public filings with the Canadian securities regulatoryauthorities, including the Annual Information Form, for further risk factors that might affect BELLUSHealth Inc. and its business.
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INNOVATIVE DRUGSIDENTIFY. DEVELOP. TRANSACT.
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Team with strong track record of execution
Company Highlights
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• Core project targeting chronic cough, potential multi-billion dollar drug class
− BLU-5937: potential best-in-class drug for clinically validated P2X3 target
− First in class program acquired by Merck in 2016 for $US500M after Phase 2
− Clear and efficient development path for value creation
• Partner in three additional pre-Phase 2 programs
• Cash runway to end of 2018
Strong core project, balanced pipeline, multiple prospects
Pipeline Overview
PRECLINICAL PHASE 1 PHASE 2 PHASE 3
BLU-5937Chronic Cough Wholly-ownedWholly-owned
Partnered IP (Revenue Share)Partnered IP (Revenue Share)KIACTASarcoidosis
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Partnered IP (Revenue Share & Royalty)Partnered IP (Revenue Share & Royalty)
ALZ-801Alzheimer’s Disease
Partnered IP (Revenue Share & Royalty)Partnered IP (Revenue Share & Royalty)
AMO-01Fragile X Syndrome
BLU-5937: Investment Thesis
Low risk profile, best-in-class potential for large market with unmet need6
Merck acquired a P2X3 antagonist program in 2016 for US$500M based on positive Phase 2 data in chronic cough
Potential multi-billion dollar drug class in therapeutic indication lacking innovation
Orally bioavailable small molecule P2X3 antagonistHigh potency and P2X3 selectivityPotential for improved efficacy and safety profileClear, efficient development path to demonstrate superiority
Attractive financial termsLeverages core competencies: clinical, BD, financingExperienced, motivated team to drive project to success
Right-sized transaction for BELLUS
BLU-5937: potential to be best-in-class drug
addressing high unmet need
P2X3 receptor: validated target in
emerging drug class for chronic cough
Chronic Cough
Cough lasting > 8 weeks, associated with:
• Pulmonary diseases (asthma, COPD, lung cancer, IPF)
• Extra-pulmonary disorders (post-nasal drip, gastro-oesophageal reflux)
• Use of certain drugs (ACE inhibitors)
• No identifiable cause (unexplained chronic cough)
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ImplicationsCharacteristics
Time and resource intensive for healthcare system
• Responsible for 30M physician visits per year in U.S.
• 38% of pulmonologist outpatient practice
• Unexplained and refractory chronic cough require time and resource intensive differential diagnosis
Chronic cough affects ~10% of the adult population
Major Impact on Patients
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ExhaustionSleep deprivation Retching/vomiting
IncontinenceHeadache
Hoarse voiceChest painRib fracture
Embarrassment of coughing in publicInterference with
lifestyle, work & leisureDifficulty speakingSocial exclusion
DistressAnger
AnxietyDepression
Psychosocial complications
Social complications
Physical complications
Chronic cough has significant impact on patient quality of life8
Few Treatment Options
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Gabapentin/PregabalinOpioids
Centrally acting
Some efficacy demonstrated in small
studiesHigh incidence of adverse effects
Very limited efficacySome efficacy but cause sedation/confusion
Constipation and nausea
Potential for addiction
OTC Products
No novel approach approved to address chronic cough in 40 years9
Pathophysiology: Hypersensitivity of Cough Reflex
Coughing trigger (ex. asthma attack)
Cellular damage causes enhanced ATP release in
respiratory tract
ATP activates P2X3 receptors on airway sensory neurons
Airway hyper-excitability
Chronic Cough
Cell injury
Drug targeting P2X3 has strong rationale for reducing cough frequency 10
ATP andCytokines
Primary afferent(A or C-fiber)
ATP
ATP
Receptors
P2X3
P2X3-containingPrimary afferent
Modified from Purinergic Signaling (2012) 8 (Suppl 1)
P2X3 Family Involved in Taste Perception
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Taste stimuli
ATP release from taste buds
ATP activates P2X3 and P2X2 receptors on taste
sensory neurons
Taste perception
Drug with high selectivity for P2X3 could limit or eliminate taste alteration side effect without compromising effect on cough
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Kinnamon et al., 2013. A Taste for ATP: neurotransmission in taste buds. Frontiers in Cellular Neuroscience. Vol 7, Article 264 pp. 1-7.
mouse tongue
LegendP2X3
P2X2
P2X3 HighlySelective Antagonist
P2X3 & P2X2Double knockout
Taste loss
P2X3Single knockout
P2X2Single knockout
Mild taste alterationMild taste alteration
Expected mild/no taste alteration
P2X3 Antagonist
Knockout Mice
Drug Approach
P2X3 MildlySelective Antagonist
Significant taste alteration
Clinically Validated Molecular Target
Opportunity for highly selective P2X3 antagonist with better efficacy/safety profile to become class leader
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50 to 75%reduction in cough frequency
50 to 100%of patients experience taste disturbance
(likely due to low P2X3 selectivity)
Adbulqawi et al., 2016. P2X3 receptor antagonist (AF-219) in refractory chronic cough: a randomised, double-blind, placebo-controlled phase 2 study. Lancet. Vol 385, No 9974 pp. 1198-1205.Kitt et al., 2016. A Phase 2 Dose-Escalation Study with AF-219, a P2X3 Antagonist for the Treatment of Chronic Cough. American Thoracic Society 2016 International Conference - San Francisco.
Treating chronic cough with low selective P2X3 antagonist results in:
Meaningful Effect Problematic Side Effect
Strong drug candidate profile with potential to be best in P2X3 class
BLU-5937 Profile
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Kgscale CMC
Broad and comprehensive IP to
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HighPotency (low nM) and Selectivity for
P2X3
HighPotency (low nM) and Selectivity for
P2X3
Orally bioavailable
small molecule
Orally bioavailable
small molecule
Zerosafety findings of concern to-date
Preclinical Efficacy: Cough Response
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Treatments (control, BLU-5937) were administered orally (p.o.) two hours prior to tussive agent exposure: citric acid (0.1 M, aerosol) and histamine (0.6 mM, aerosol); n=6 animals (guinea pig) per group *p<0.05
Oral administration of BLU-5937 dose-dependently reduced the frequency of cough in a guinea pig model
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0
5
10
15
20
25
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Control 0.3 mg/kg p.o. 3 mg/kg p.o. 30 mg/kg p.o.
Total coughs (average)
Control BLU-5937
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10-40% patients with refractory / unexplained chronic cough
10-40% patients with refractory / unexplained chronic cough
10% of US adult population has chronic cough
10% of US adult population has chronic cough
Estimated addressable patients in major pharma markets:
6.3M
Large Addressable Market
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275M U.S. adults
27.5M U.S. chronic cough
patients
2.75M Unexplained/
refractory patients
Major pharma markets include the U.S., Europe top five countries and JapanSong et al., 2015. The global epidemiology of chronic cough in adults: a systematic review and meta‐analysis. Eur Respir J. Vol 55 pp. 1479‐1481Zanasi et al., 2014. Chronic and unexplained cough. (Published online) Vol 4, No 3 pp. 159‐164
Key Development Milestones
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Value creating milestones throughout development path
2017 2018 2019/2020
IND-enabling studies Phase I: assess dose and taste effect
Phase II: demonstrate antitussive effect
Complete IND preclinical study
package
Assess safety, tolerability, PK, effect on taste in healthy subjects
Single ascending dose and multiple ascending
dose studies
Assess safety, PK and antitussive effects in patients
suffering from chronic refractory cough
Dose response study with crossover design
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Partnered Programs
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Genetic disorder affecting ~180K in U.S.IP licensed in 2014 for low double-digit royalty/ single digit revenue shareRas-ERK inhibitor that reverses disease abnormalities in preclinical modelStart of Phase 2 expected in 2017
Principally respiratory disease affecting ~100K in U.S.IP licensed in 2009 with double digit revenue shareReduces key inflammatory markers mediated by serum amyloid AAuven to confirm plans to enter Phase 2/3 study in 2017
ALZ-801 for Alzheimer’s
disease (AD)(Alzheon Inc)
KIACTATM for Sarcoidosis
(AuvenTherapeutics)
AMO-01 for Fragile X Syndrome
(AMO Pharma)
IP licensed in 2013 for mid single-digit royalty/revenue shareInhibitor of amyloid aggregationPreparing for Phase 3 for AD patients homozygous for apolipoprotein E gene
Financial Highlights
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Stock Information (March 14, 2017)
Ticker TSX: BLUShares (basic) 66.9MShares (fully diluted) 71.7MDaily Volume ~150KMarket Capitalization ~$20M
Key Financials1
Cash $7.5MCash Runway Q4 2018
Fully Diluted Shareholder OwnershipBellini Family ~25%Power Corporation ~25%1as at December 31, 2016 and pro forma to upfront considerations of Thallion and NEOMED transactions
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Board of DirectorsDr. Francesco Bellini (Chair) Franklin Berger Charles Cavell
Hélène Fortin Pierre Larochelle Muriel Lortie
Joseph Rus Dr. Martin Tolar Roberto Bellini
ManagementRoberto Bellini, President and Chief Executive Officer
Dr. Denis Garceau, Senior Vice President, Drug DevelopmentFrançois Desjardins, Vice President, Finance
Tony Matzouranis, Vice President, Business Development
Governance and Shareholders
LAROSE FORTIN CA Inc.
Execution of BLU-5937 plan in chronic cough:
• IND-enabling studies
• Market assessment
• Present further animal data
Progress in other projects
• KIACTA Phase 2/3 in Sarcoidosis
• AMO-01 Phase 2 in Fragile X
• Alzheon financing
Milestones
Past ExecutionAttracted more than $100M to funding projects
Executed multiple global clinical studies including Phase 3
Completed multiple transactions (in-licensing, partnering, out-licensing)
Milestones (12 months)
Multiple upcoming milestones to drive value20