bellus health corporate presentation may 2016
TRANSCRIPT
Corporate Presentation (TSX: BLU)
Roberto BelliniPresident and Chief Executive OfficerTwitter: @rbellini
May 2016
r
Forward Looking StatementsCertain statements contained in this presentation, other than statements of fact that are independently verifiable at the date hereof, may constitute “forward-looking statements” within the meaning of Canadian securities legislation and regulations. Such statements, based as they are on the current expectations of management, inherently involve numerous important risks, uncertainties and assumptions, known and unknown, many of which are beyond BELLUS Health Inc.'s control. Such risks factors include but are not limited to: the ability to obtain financing, the impact of general economic conditions, general conditions in the pharmaceutical industry, changes in the regulatory environment in the jurisdictions in which the BELLUS Health Inc. does business, stock market volatility, fluctuations in costs, changes to the competitive environment due to consolidation, achievement of forecasted burn rate, potential payments/outcomes in relation to indemnity agreements and contingent value rights, achievement of forecasted pre-clinical and clinical trial milestones, dependence on Auven Therapeutics for the completion of the KIACTA™ Phase 3 Confirmatory Study and that actual results may vary once the final and quality-controlled verification of data and analyses has been completed. In addition, the length of the KIACTA™ Phase 3 Confirmatory Study and the sharing of proceeds between Auven Therapeutics and BELLUS Health Inc. from potential future revenue of KIACTA™ are dependent upon a number of factors, including the quantum of proceeds. Consequently, actual future results and events may differ materially from the anticipated results and events expressed in the forward-looking statements. The Company believes that expectations represented by forward-looking statements are reasonable, yet there can be no assurance that such expectations will prove to be correct. The reader should not place undue reliance, if any, on any forward-looking statements included in this presentation. These forward-looking statements speak only as of the date made, and BELLUS Health Inc. is under no obligation and disavows any intention to update publicly or revise such statements as a result of any new information, future event, circumstances or otherwise, unless required by applicable legislation or regulation. Please see BELLUS Health Inc.’s public filings with the Canadian securities regulatory authorities, including the Annual Information Form, for further risk factors that might affect BELLUS Health Inc. and its business.
2
3
At BELLUS, we are focused on developing drugs for rare diseases starting with conditions that affect the kidneys.
Regulatory advantage
Premium pricing
Market protection
Smaller clinical trials
Efficient commercialization strategies
4
Small patient numbers, BIG opportunity
Value driving rare disease pipeline fully funded through key milestones
Company Highlights
5
• Late-stage pipeline with 4 projects targeting rare diseases
• Lead drug candidate, KIACTA, in Phase 3 Confirmatory Study for AA amyloidosis Rare and deadly kidney disease with no treatment
Phase 2/3 study completed with positive efficacy and clean safety
Similar and confirmatory Phase 3 study completed (Data expected in Q2 2016)
Validating development partner investing $70M in project
Potential exit to commercial partner following Phase 3 data
• Business plan fully funded through KIACTA Phase 3 and exit process
Late stage pipeline focused on developing innovative drugs for rare diseases
Pipeline of Products
ShigamabsHUS
DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3
KIACTAAA amyloidosis Partnered
MARKET
AL amyloidosis
PartneredKIACTASarcoidosis
6
Lead Phase 3 Product Candidate
7
A rare and deadly kidney disease with no specific treatment
FOR AMYLOID A (AA) AMYLOIDOSIS
Disease and Mechanism of Action
8
CHRONIC INFLAMMATION
SERUM AMYLOID APRECURSOR (SAA) PROTEIN
AA PROTEIN + GLYCOSAMINOGLYCANS (GAGs)
ORGAN DAMAGE, IN PARTICULAR TO KIDNEYS LEADING TO DIALYSIS
REDUCTION IN FIBRIL FORMATION & DEPOSITION
Converts toAA Protein
Generatescytokine cascade
(TNFα / IL-1 / IL-6) and increases SAA levels
Rheumatic ConditionsInflammatory Bowel DiseaseChronic InfectionsFamilial Mediterranean Fever
KIACTA™ blocksAA + GAGs interaction
Systemic Amyloid A Fibril Formation & Deposition
KIACTA designed to bind AA amyloid, slow down disease progression and delay dialysis 8
HR 0.58 0.41 0.48 0.54 0.95
95% C.I 0.37, 0.93 0.19,0.86 0.28,0.82 0.22,1.37 0.27,3.29
P value 0.025 0.019 0.008 0.20 0.94
Graphical representation of the information in this table
Composite Endpoint (Time to
First Worse Event)
Doubling Serum
Creatinine
50%DecreaseCreatinine Clearance
Dialysis/ESRD Death
Num
ber o
f Pat
ient
Eve
nts
9
*
*
**
KIACTA - Robust Clinical Results in Phase 2/3
*p<0.05 **p<0.01Dember et al. June 7, 2007. New England Journal of Medicine. Vol 356 (23) 2349-2360.
Landmark study in AA amyloidosis: 183 patients treated for 2 years
Important benefits for patients on drug:
Statistically significant (p-value=0.025) reduction in number and risk of reaching worsening kidney event
Important delay in reaching dialysis
Clean safety profile without any important differences
between groups in Phase 2/3 study 10
KIACTA – Clean Safety Profile
Adverse Events Serious Adverse Events Discontinuations due to Adverse Events
0%
20%
40%
60%
80%
100% 98%
36%
23%
93%
42%
25%
KIACTA
Placebo
% o
f Pat
ient
s
11
Regulatory
New England Journal of Medicine publication concludes that KIACTA slows decline of renal function in AA amyloidosis
Agreement reached in U.S., Europe, Japan to conduct Phase 3 Confirmatory Study
Marketing approval based on achieving comparable result with lower statistical bar than first Phase 3 Study
Experienced and knowledgeable partner working on lead project
Auven is a global biotech private equity group
Partnered on KIACTA project in 2010
Funding 100% of KIACTA project including studies in AA Amyloidosis and Sarcoidosis
≥ US$70M in investments
Overall proceeds of exit expected to be shared 50-50
KIACTA to be sold/partnered to commercial entity after Phase 3 Confirmatory Study results
Auven Therapeutics Partnership for KIACTA
BUSINESS PLANAUVEN PARTNERSHIP
12
PHASE 3 CONFIRMATORY STUDYKey entry criteria based on kidney function: High proteinuria (>1 g/d) or low
creatinine clearance (< 60 ml/min/1.73m2)
183 patients in 13 countries
Fixed treatment duration of 2 years 74 kidney function worsening
events
PHASE 2/3 STUDY
Enriched patient population High proteinuria (>1 g/d)
More patients 261 patients in >25 countries
Increased power Event driven trial to conclude on
reaching 120 events
KIACTA – Phase 3 Confirmatory Study
13
Key improvements made to increase chance of successful study
13
Study enrolled with 261 patients
Study completed with 120 events reached (January 2016)
Last Patient Last Visit in March 2016
Topline data expected in Q2 2016
Phase 3 Confirmatory Study
14
Patient Population
Source: Navigant Consulting 2014
10,000-15,000 potential KIACTA
patients in the United States and Europe
MARKET RESEARCHNavigant Consulting conducted extensive primary and secondary research including over 60 interviews with treating physicians and key opinion leaders in the United States and Europe
15
PRICING
Orphan drug designation granted with
market protection in the U.S. (7 years),
Europe and Japan (10 years)
Intellectual property protection to 2031
PROTECTION
Disease with large unmet medical need and no specific treatment
Clear pharmaco-economic rationale due to high cost of kidney disease
Premium pricing for comparative rare disease drugs
Market Considerations
KIACTA is well positioned to achieve premium pricing in line with comparable rare disease drugs
16
Drug U.S. Patients Disease Price
Vyndaqel 1,500 Transthyretin amyloid polyneuropathy $200K
Gattex 9,500 Short Bowel Syndrome $295K
Kalydeco 1,350 Cystic Fibrosis (G551D mutation) $335K
Procysbi 500 Nephropathic cystinosis $250K
Juxtapid 3,000 Familial hypercholesterolemia $250K
Jakafi 1,500 Splenomegaly $87K
COMPARABLES
POTENTIAL ACQUIRERSPharma/Big biotech with inflammation and/or nephrology franchise
Orphan disease focused biotech
Exit Strategy
Strong M&A environment for rare disease products17
Company Main Drug / Disease Stage Transaction
Synageva Kanuma/ LAL-D Registration (no sales) Acquired by Alexion in June 2015 for $8.4B
NPSGattex / Short Bowel Syndrome Market ($350M in sales) Acquired by Shire in January 2015 for
$6.2B
Scioderm Zorblisa / E. Bullosa Phase 3 (no sales)Acquired by Amicus in August 2015 for $230M upfront plus $600M in milestones
RECENT RARE DISEASE M&A
Second KIACTA Indication – Sarcoidosis
INDICATION
DEVELOPMENT
Chronic sarcoidosis, a rare disease that causes lung scarring and decreased lung functionKIACTA target Serum Amyloid A plays key role in disease
Partnered with AuvenAgreement with Mount Sinai Hospital New York to start Phase 2/3 studyIND filing expected in 2016
18
Second Rare Disease Product Candidate
19
A rare disease primarily affecting the kidneys of children
FOR STEC RELATEDHEMOLYTIC UREMIC SYNDROME (SHUS),
SHIGAMABSHIGAMAB
Disease Course and Mechanism of Action
E. COLI INGESTION
GUT COLONIZATION AND SECRETION OF TOXIN INTO BLOODSTREAM
TOXIN MAY BE CARRIED BY PMNs IN BLOODSTREAMSYMPTOMS: BLOODY DIARRHEA
SHIGAMAB BINDING NEUTRALIZES TOXIN WHICH IS THEN ELIMINATED
Shigamab Antibody
Day -4 Day 0 Day 4 Day 8
TOXIN BINDS TO GB3 RECEPTORS ON KIDNEY LEADING TO STEC-HUS. OUTCOMES: -CHRONIC KIDNEY DISEASE / HYPERTENSION: 40%-ENCEPHALOPATHY / DEATH: 5%-RESOLUTION: 55%
20
90%
SPONTANEOUSRESOLUTION
10%
SHIGAMAB TREATMENT
Mice rescued from shigatoxin induced weight loss and kidney injury up to 4 days post intoxicationData presented at VTEC conference in September 2015
Shigamab Overview
NEXT STEPS (12 MONTHS)
MARKET OPPORTUNITY
CLINICAL
Further animal model data in treatment of sHUSMeetings with regulators to agree on clinical development plan
2,000-3,000 estimated annual cases of sHUS in developed countries, principally children$100-200 million annual sales opportunity
Safe and well tolerated in target pediatric population
21
PRE- CLINICAL
Clean capital structure and cash runway through potential exit
Corporate
22
Capital Markets (as of May 9th, 2016)
Ticker TSX: BLU
Shares (Basic) 54.7M
Shares (Fully Diluted) 65.9M
Daily Volume ~100K
Market Capitalization (FD) ~C$150M
22
Finance
Cash (March 31, 2016) C$9.0M
Burn rate (monthly) <C$300K
Shareholder Ownership (FD)
Bellini Family ≈ 33%
Power Corporation ≈ 27%
Pharmascience ≈ 10%
Governance and Shareholders
23
Board of Directors Company / Experience
Dr. Francesco Bellini (Chair)
Franklin Berger
Charles Cavell
Hélène Fortin
Pierre Larochelle
Muriel Lortie
Joseph Rus
Dr. Martin Tolar
Roberto Bellini
Management Title
Roberto Bellini President and Chief Executive Officer
Dr. Denis Garceau Senior Vice President, Drug Development
François Desjardins Vice President, Finance
Tony Matzouranis Vice President, Business Development
LAROSE FORTIN CA Inc.
23
Potential KIACTA exit
Continue executing KIACTA for AA Amyloidosis plan:
Reach 120 event target (Q1 2016)
Top Line Data (Q2 2016)
Progress rare disease pipeline projects:
IND filing for KIACTA Phase 2/3 for Sarcoidosis (mid 2016)
Shigamab animal data (mid 2016)
Shigamab clinical trial design (mid 2016)
Significant news flow and value inflection point in 2016
Milestones
Past Execution
Attractive partnership for KIACTA
Execution of global KIACTA Phase 3 Confirmatory Study
Expansion of rare disease pipeline
Strong balance sheet and clean capital structure
Milestones
24
Connect With Us
Follow us on Twitter: @BELLUSHealth
Join our LinkedIn group
Read our blog @ www.bellushealth.com
Join our mailing list