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Bellus Health Corporate Presentation June 2016
1. Corporate Presentation (TSX: BLU)
Roberto Bellini
President and Chief Executive Officer
Twitter: @rbellini
June 2016
r
2. Forward Looking Statements
Certain 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. 3
At BELLUS, we are focused on developing drugs for rare
diseases starting with conditions that affect the kidneys.
5. 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
6. Late stage pipeline focused on developing innovative drugs for
rare diseases
Pipeline of Products
Shigamab
sHUS
DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3
KIACTA
AA amyloidosis Partnered
MARKET
AL amyloidosis
Partnered
KIACTA
Sarcoidosis
6
7. Lead Phase 3 Product Candidate
7
A rare and deadly
kidney disease with
no specific treatment
FOR AMYLOID A (AA)
AMYLOIDOSIS
8. Disease and Mechanism of Action
8
CHRONIC
INFLAMMATION
SERUM AMYLOID A
PRECURSOR (SAA)
PROTEIN
AA PROTEIN +
GLYCOSAMINOGLYCANS
(GAGs)
ORGAN DAMAGE, IN
PARTICULAR TO
KIDNEYS LEADING TO
DIALYSIS
REDUCTION IN
FIBRIL FORMATION
& DEPOSITION
Converts to
AA Protein
Generates
cytokine cascade
(TNFα / IL-1 / IL-6)
and increases SAA levels
Rheumatic Conditions
Inflammatory Bowel Disease
Chronic Infections
Familial Mediterranean Fever
KIACTA™ blocks
AA + GAGs interaction
Systemic Amyloid A Fibril
Formation & Deposition
KIACTA designed to bind AA amyloid, slow down disease
progression and delay dialysis 8
9. 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%
Decrease
Creatinine
Clearance
Dialysis/
ESRD
Death
NumberofPatientEvents
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
10. Clean safety profile without any important differences
between groups in Phase 2/3 study 10
KIACTA – Clean Safety Profile
98%
36%
23%
93%
42%
25%
0%
20%
40%
60%
80%
100%
Adverse Events Serious Adverse Events Discontinuations due to
Adverse Events
%ofPatients
KIACTA
Placebo
11. 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
12. 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
13. PHASE 3 CONFIRMATORY STUDY
Key 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
14. 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
15. Patient Population
Source: Navigant Consulting 2014
10,000-
15,000
potential KIACTA
patients in the United
States and Europe
MARKET RESEARCH
Navigant 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
16. 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
17. POTENTIAL ACQUIRERS
Pharma/Big biotech with inflammation and/or nephrology franchise
Orphan disease focused biotech
Exit Strategy
Strong M&A environment for rare disease products
17
Company Main Drug / Disease Stage Transaction
Synageva Kanuma/ LAL-D Registration (no sales)
Acquired by Alexion in June 2015 for
$8.4B
NPS
Gattex / 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
18. Second KIACTA Indication – Sarcoidosis
INDICATION
DEVELOPMENT
Chronic sarcoidosis, a rare
disease that causes lung scarring
and decreased lung function
KIACTA target Serum Amyloid A
plays key role in disease
Partnered with Auven
Agreement with Mount Sinai Hospital
New York to start Phase 2/3 study
IND filing expected in 2016
18
19. Second Rare Disease Product Candidate
19
A rare disease
primarily affecting
the kidneys of
children
FOR STEC RELATED
HEMOLYTIC UREMIC
SYNDROME (SHUS),
SHIGAMABSHIGAMAB
20. Disease Course and Mechanism of Action
E. COLI INGESTION
GUT COLONIZATION AND
SECRETION OF TOXIN
INTO BLOODSTREAM
TOXIN MAY BE CARRIED
BY PMNs IN
BLOODSTREAM
SYMPTOMS: 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%
SPONTANEOUS
RESOLUTION
10%
SHIGAMAB TREATMENT
21. Mice rescued from shigatoxin induced weight loss and kidney injury up
to 4 days post intoxication
Data presented at VTEC conference in September 2015
Shigamab Overview
NEXT STEPS (12 MONTHS)
MARKET OPPORTUNITY
CLINICAL
Further animal model data in treatment of sHUS
Meetings 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
22. Clean capital structure and cash runway through potential exit
Corporate
22
Capital Markets (as of June 3rd, 2016)
Ticker TSX: BLU
Shares (Basic) 61.1M
Shares (Fully Diluted) 65.9M
Daily Volume ~150K
Market Capitalization ~C$170M
22
Finance
Cash (March 31, 2016) C$9.0M
Burn rate (monthly) <C$300K
23. 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
24. 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
25. Connect With Us
Follow us on Twitter: @BELLUSHealth
Join our LinkedIn group
Read our blog @ www.bellushealth.com
Join our mailing list