BELLUS Health is developing therapies for rare diseases starting with conditions affecting the kidneys. Its lead product KIACTA is in Phase III trials for AA amyloidosis, a rare kidney disease with no approved treatments. It has executed a partnership for KIACTA's development and aims to complete enrollment in 2014. BELLUS' pipeline also includes Shigamab for STEC-HUS and a research program in AL amyloidosis. The company expects key milestones in 2014 to progress these programs and generate long-term value.

1. Corporate Presentation (TSX: BLU)
Winter 2014
Roberto Bellini
President and Chief Executive Officer
Twitter: @rbellini

2. 30 million
people in the United States have a RARE disease.
Source: NIH: National Institutes of Health Office of Rare Diseases
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3. Only about 5%
of these people have a specific therapy
to treat their disease.
3

4. 85-90%
of rare diseases are serious or life threatening.
4

5. Small patient numbers, BIG opportunity
Regulatory advantage
Premium pricing
Market protection
Smaller clinical trials
Efficient commercialization
strategies
5

6. At BELLUS, we are focused on developing drugs for rare
diseases starting with conditions that affect the kidneys.
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7. Executing on Plan
2013
Lead rare disease program,
KIACTA™ for AA amyloidosis in
Phase III Confirmatory Study :
Recruitment on target
Japan orphan drug
designation
Expand rare disease pipeline
Acquisition of Shigamab
for STEC-HUS
Research partnership for
AL amyloidosis
Divestiture of non-core assets
Maintain financial health
2014
Continue executing KIACTA™
plan:
Completion of recruitment
Launch of open label
extension study
Market and pricing
assessment
Progress pipeline projects:
Animal studies to support
Shigamab Phase II
Pre-clinical proof of
concept for AL amyloidosis
Continued financial stewardship
Fruitful 2013 leading into important milestones in 2014
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8. Shareholder Information
OVERVIEW
CAPITAL STRUCTURE
Public company (TSX: BLU)
based in Montreal, QC
Shares outstanding
(Fully Diluted): 65M
Developing drugs for rare
diseases
Cash (09/30/13): ~$16M
Late-stage product pipeline with
fully funded business plan
Burn rate (monthly): <$300K
Shareholder makeup:
70% institutional, 30% retail
Business plan fully funded through KIACTA™ exit
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9. Pipeline of Products
DISCOVERY
PRECLINICAL
PHASE I
PHASE II
PHASE III
MARKET
KIACTA™
AA amyloidosis
Shigamab
sHUS
AL amyloidosis
Late stage pipeline focused on developing innovative drugs for rare
diseases
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10. Lead Phase III Product Candidate
FOR AMYLOID A (AA)
AMYLOIDOSIS
A rare and deadly
kidney disease with
no specific treatment
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11. Disease and Mechanism of Action
SERUM AMYLOID A
PRECURSOR (SAA)
PROTEIN
AA PROTEIN +
GLYCOSAMINOGLYCANS
KIACTA™ blocks
AA + GAGs interaction
(GAGs)
REDUCTION IN
FIBRIL FORMATION
& DEPOSITION
CHRONIC
INFLAMMATION
Generates
cytokine cascade
(TNFα / IL-1 / IL-6)
and increases SAA levels
Rheumatic Conditions
Inflammatory Bowel Disease
Chronic Infections
Familial Mediterranean Fever
Converts to
AA Protein
Systemic Amyloid A Fibril
Formation & Deposition
ORGAN DAMAGE, IN
PARTICULAR TO
KIDNEYS LEADING TO
DIALYSIS
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12. Market
MARKET PROTECTION
Orphan drug designation granted
with market protection in the U.S. (7
years), Europe and Japan (10 years)
Formulation (Dosing Schedule) and
Methods for Treating Amyloidosis
with expiry in 2026
5 year patent extension can be
applied to provide protection until
2031
MARKET SIZE
KIACTA™ peak annual revenues
projected at $500 million
Clear pharmacoeconomic
rationale due to high cost of kidney
disease
Premium pricing for comparative
rare disease drugs
Independent market assessment currently underway and to be
completed by Q1 2014
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13. Strategic Partnership
PARTNERSHIP
FINANCIALS
With global fund Auven
Therapeutics, a private equity
group specialized in drug
development project
financing
US$10M in upfront by Auven
Therapeutics
Auven Therapeutics funding
100% of KIACTA™’s Phase
III Confirmatory Study
Proceeds of exit expected to
be shared 50-50
≥ US$50M in investments by
Auven Therapeutics
Partnership to fund Phase III Confirmatory Study with significant
upside for BELLUS shareholders
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14. Strong Clinical Results in First Phase III Study
50
Placebo
45
KIACTA
Number of Worse Events
40
35
30
Landmark study in AA
amyloidosis: 183 patients
treated for 2 years
Important benefits for
patients on drug:
*
25
Statistically significant
reduction in number and
risk of reaching
worsening kidney event
**
20
15
*
10
Important delay in
reaching dialysis
5
0
Composite
Endpoint (Time to
First Worse
Event)
Doubling
Serum
Creatine
50%
Decrease
Creatine
CIearance
Dialysis/
ESRD
*p<0.05
**p<0.01
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15. Regulatory
New England Journal of
Medicine publication
concludes that KIACTATM
slows decline of renal
function in AA
amyloidosis
Agreement reached in
U.S., Europe, Japan to
conduct Phase III
Confirmatory Study
Approval based on
achieving comparable
result of first Phase III
Study
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16. Phase III Confirmatory Study
Study enrolling 230 patients total
with ~200 patients enrolled
Event driven trial to complete when
120 of 230 patients reach event of
kidney function deterioration
Study completion expected in 2016
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17. Second Rare Disease Product Candidate
SHIGAMAB
FOR STEC RELATED
HEMOLYTIC UREMIC
SYNDROME (SHUS),
A rare disease
primarily affecting
the kidneys of
children
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18. Disease Course and Mechanism of Action
SHIGAMAB TREATMENT
SHIGAMAB BINDING
NEUTRALIZES TOXIN
WHICH IS THEN
ELIMINATED
GUT COLONIZATION AND
SECRETION OF TOXIN
INTO BLOODSTREAM
TOXIN MAY BE CARRIED
BY PMNs IN
BLOODSTREAM
SYMPTOMS: BLOODY
DIARRHEA
Shigamab
Antibody
E. COLI INGESTION
10%
90%
TOXIN BINDS TO GB3
RECEPTORS ON KIDNEY
LEADING TO STEC-HUS.
OUTCOMES:
-CHRONIC KIDNEY DISEASE /
HYPERTENSION: 40%
-ENCEPHALOPATHY / DEATH: 5%
-RESOLUTION: 55%
SPONTANEOUS
RESOLUTION
Day -4
Day 0
Day 4
Day 8
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19. Shigamab Overview
MARKET OPPORTUNITY
2,000-3,000 estimated annual cases of sHUS in developed
countries, principally children
$100-200 million annual sales opportunity
PRE-CLINICAL AND CLINICAL
Treatment with Shigamab led to significantly increased survival in
STEC animal model
Safe and well tolerated in target pediatric population
NEXT STEPS (12 MONTHS)
Proof of concept for treatment of sHUS in animal models
Meetings with regulators to agree on development plan
Potential for partnership in 18-24 months
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20. Research Program
DRUG CANDIDATES
RESEARCH PROJECT FOR
AMYLOID LIGHT-CHAIN
(AL) AMYLOIDOSIS,
A rare and deadly
blood disorder
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21. AL Amyloidosis Project Overview
PARTNERSHIP
Partnership with Amorchem, a Montreal-based venture fund, to
finance research project
Objective: identify and develop drug candidates for AL amyloidosis
to pre-clinical proof-of-concept
DISEASE
AL amyloidosis is a blood disorder that leads to the formation of
toxic amyloid fibrils and plaques
Treatment options are limited leading to death in most cases
2,000-3,000 new cases are reported each year in the United States
Potential for pre-clinical proof-of-concept within 12 months
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22. Governance and Shareholders
Board of Directors
Company / Experience
Management
Title
Roberto Bellini
Hélène Fortin
Pierre Larochelle
Vice President, Finance
Tony Matzouranis
Charles Cavell
Senior Vice President, Drug
Development
François Desjardins
Franklin Berger
President and Chief
Executive Officer
Dr. Denis Garceau
Dr. Francesco Bellini
(Chair)
Vice President, Business
Development
LAROSE FORTIN CA Inc.
Shareholder
Ownership
Bellini Family
Donald Olds
Joseph Rus
≈ 30%
Power Corporation
≈ 30%
Pharmascience
≈ 10%
Dr. Martin Tolar
Roberto Bellini
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23. Milestones
2014 Milestones
Past Execution
Attractive partnership
for KIACTA™
Execution of global
KIACTA™ Phase III
Confirmatory Study
Expansion of rare
disease pipeline
Strong balance sheet
and clean capital
structure
Continue executing KIACTA™
plan:
Completion of recruitment
Launch of open label
extension study
Market and pricing
assessment
Long Term Value
KIACTA™ exit and
results of Phase III
Confirmatory Study
Shigamab partnership
or proof-of -concept
Phase II study results
Progress pipeline projects:
Animal studies to support
Shigamab Phase II
Pre-clinical proof of
concept for AL amyloidosis
Short-term milestones driving long-term value
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24. Forward Looking Statement
Certain statements contained in this presentation, other than statements of fact that are
independently verifiable at the date hereof, may constitute forward-looking statements. Such
statements, based as they are on the current expectations of management, inherently involve
numerous risks and uncertainties, known and unknown, many of which are beyond BELLUS
Health Inc.'s control. Such risks include but are not limited to: the ability to obtain financing
immediately in current markets, the impact of general economic conditions, general conditions
in the pharmaceutical and/or nutraceuticals industry, changes in the regulatory environment in
the jurisdictions in which the BELLUS Health Group does business, stock market volatility,
fluctuations in costs, and changes to the competitive environment due to consolidation,
achievement of forecasted burn rate, and that actual results may vary once the final and
quality-controlled verification of data and analyses has been completed.
Consequently, actual future results may differ materially from the anticipated results expressed
in the forward-looking statements. The reader should not place undue reliance, if any, on any
forward-looking statements included in this news release. These statements speak only as of
the date made and BELLUS Health Inc. is under no obligation and disavows any intention to
update or revise such statements as a result of any event, circumstances or otherwise, unless
required by applicable legislation or regulation. Please see the Company’s public fillings
including the Annual Information Form of BELLUS Health Inc. for further risk factors that might
affect the BELLUS Health Group and its business
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