2. 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
2
3. Background and Business Model
Public company (TSX: BLU) based in Montreal, QC
Focused namely on the development of products in amyloid-
related fields, principally AA Amyloidosis, an orphan indication
affecting the kidneys
Pipeline also includes product candidate for the treatment of
Alzheimer’s disease
BUSINESS MODEL
Focused on building value for clinical
stage health products in critical unmet
medical needs
3
4. Pipeline of Products
PHARMACEUTICALS DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III NDA/MAA
KIACTA™
AA amyloidosis
BLU8499
Alzheimer’s
disease
COMMERCIA
NUTRACEUTICAL DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III -LIZATION
VIVIMIND™
Memory protection
4
5. KIACTA™
For AA Amyloidosis, an orphan indication and a
deadly disease with no treatment
Orphan population of ≈50,000 in the USA, Europe
Market and Japan with peak annual revenues projected at
opportunity $400-600M1
1 Independent market assessment by Frankel Group in April 2009.
Phase II/III clinical trial showing statistically
Clinical
significant primary efficacy endpoints
evidence
(p value = 0.025) and clean safety profile
Partnership with Auven Therapeutics (previously
Partnership known as Celtic Therapeutics) to conduct and
finance (>$50M) Phase III Confirmatory Study
Marketing approval based on confirming safety
Phase III
Confirmatory and efficacy of phase II/III study
Study >50% patients recruited
5
6. AA Amyloidosis – A Rare and Lethal Disease
AA PROTEIN + REDUCTION IN
SERUM AMYLOID A GLYCOSAMINOGLYCANS KIACTA blocks FIBRIL FORMATION
PRECURSOR (SAA) (GAGs) AA + GAGs interaction & DEPOSITION
PROTEIN
CHRONIC
INFLAMMATION
Generates Converts to
cytokine cascade AA Protein
(TNFα / IL-1 / IL-6) Systemic Amyloid A Fibril
and increases SAA levels Formation & Deposition
ORGAN DAMAGE, IN
Rheumatic Conditions PARTICULAR TO
Inflammatory Bowel Disease KIDNEYS
Chronic Infections -Rapid deterioration of
Familial Mediterranean Fever kidney function leading to
dialysis
6
7. KIACTA™ - Targeted Opportunity
Patient population estimated at
34-50,000 in the U.S., EU5 and
Japan1
Diagnosed AA Patients (000s)
Clear pharmacoeconomic
rationale for premium pricing
KIACTA™ peak annual revenues
projected at $400-600M1
(U.S., EU5, Japan)
Orphan Drug Status in the U.S.
and EU provides 7 and 10 years
market exclusivity upon
commercialization, respectively
1Independent market assessment by Frankel Group in April 2009 7
8. KIACTA™ - Strategic Partnership
PARTNERSHIP FINANCIAL IMPLICATION
Partnered with private equity US$10M upfront
firm Auven Therapeutics
≥ US$50M in investments by
Auven Therapeutics funding Auven Therapeutics
100% of KIACTA™’s Phase
III Confirmatory Study Proceeds of any eventual
transaction expected to be
Auction process for the shared 50%-50% between
commercialization rights of BELLUS Health and Auven
KIACTA™ on completion of Therapeutics
Phase III Confirmatory Study
8
9. KIACTA™ - Robust Clinical Results in Phase II/III
• Landmark study in AA
Number of Patient Events
Amyloidosis: 183 patients
treated for 2 years
• Composite endpoint based
on patients reaching events
* of decreasing kidney
Graphical
function or death
representation of the
* information in this
• Statistically significant
primary endpoint (Cox
table
Proportional Hazard Ratio;
* p=0.025)
• Cox test takes into account
event and time to event
Composite 50% • Most sensitive component
Doubling Dialysis/
Endpoint (Time Decrease Death of composite endpoint highly
Serum ESRD
to First Worse Creatine
Creatine significant (CrCl, p < 0.01)
Event) CIearance
HR 0.58 0.41 0.48 0.54 0.95 • Clinically meaningful treatment
95% C.I 0.37, 0.93 0.19,0.86 0.28,0.82 0.22,1.37 0.27,3.29 effect with 42% reduction in
P value 0.025 0.019 0.008 0.20 0.94 risk of reaching event
9
10. KIACTA™ - Phase II/III Key Secondary Endpoints
Kaplan Meier: Time to First Worst Event Slope of Creatinine Clearance Decline
Months of Treatment
Mean ∆ CrCl (mL/min/1.73m2)
KIACTATM
KIACTATM
Placebo -10.9 ± 5.1 mL/min/
1.73 m2/year
Placebo
-15.6 ± 4.0 mL/min/ ∆= 9.4mL/min/
1.73 m2/year 1.73 m2
HR=0.58
after 24
Wald Chi Square test: p=0.025
months
p=0.025
Key secondary endpoints show robust effect of KIACTA™
Using slope of creatinine clearance decline, calculated delay
to time to dialysis is ~2 years on KIACTA™ versus placebo
10
11. KIACTA™ - Phase II/III Feedback
2007 NEJM article published by lead investigators concludes that
KIACTA™ slows decline of renal function in AA Amyloidosis
Agreement reached under Special Protocol Assessment with FDA and
Scientific Advice with EMEA to conduct Phase III Confirmatory Trial
Marketing approval based on positive result (p value <0.05) from confirmatory study with
same scope of first phase II/III clinical trial
11
12. KIACTA™ - Phase III Confirmatory Study
PHASE III
COMPLETED PHASE II/III STUDY CONFIRMATORY STUDY
183 patients in 13 countries 230 patients in >25 countries
Statistically significant composite Composite primary endpoint
primary endpoint (p=0.025) (target p<0.05) based on patients
principally based on patients reaching kidney function events:
reaching kidney function events:
80% increase serum creatinine
Doubling serum creatinine
40% decrease in creatinine
50% decrease in creatinine clearance
clearance
Reaching ESRD/dialysis
Reaching ESRD/dialysis
Death Event driven trial to conclude on
attainment of 120 events (~90%
Fixed treatment duration of two power)
years
Key improvements made to
increase robustness of
confirmatory study
12
13. KIACTA™ - Study Progress
Recruitment Completion
>70 sites in >25 Event driven trial to
countries actively complete on reaching
recruiting 120 events
~120 patients Study expected to be
enrolled1 completed in 2017
Recruitment expected
to be completed in 1H
2014
Patient baseline characteristics and demographics to date are similar
to those in the first Phase III study
1 Data as of November 2012 13
14. VIVIMIND™
Nutraceutical for memory protection
Health claims include: VIVIMIND Revenues ($K)
500
Regulatory ‘Protects the
400
hippocampus’ and
300
‘Enhances cognitive
200
function and memory’
100
0
2010 2011 2012
Partnerships for Italy (launched), Canada
(launched), South Korea (regulatory), Greece (pre-
Partnerships launch), Middle East (pre-launch), Taiwan
(regulatory) and Israel (regulatory)
Pursuing creation of worldwide distributor network
Growing cash flow positive business
14
15. BLU8499
Next generation of tramiprosate intended
for the treatment of Alzheimer's disease
Large and growing epidemic currently affecting
Market over 30M patients worldwide
opportunity Represents > $180B in annual costs in the United
States alone
Evidence of effectiveness of parent compound
Clinical tramiprosate in ApoE4+ Alzheimer’s patients
Evidence
Safe and well tolerated in Phase I
15
16. BLU8499 – Asclepios Partnership
Partnership with Asclepios Bioresearch in
September 2012 to finance development of
BLU8499
Partnership
Investment of ~$4M in non-dilutive capital
Parties expected to share any future proceeds
approximately equally
Long term animal toxicity to be completed in
2013
Development
Phase IIa proof of concept study in mild apoE4+
Plan
Alzheimer’s disease patients
Expected to begin in 2014
Focused development plan to demonstrate
effectiveness in targeted patient population
16
17. Financial Position and Capital Structure
Capital Structure
Basic Shares Outstanding 47M
Fully Diluted Shares Outstanding1 61M
Financial Position
Cash (December 31st, 2012) >$18M
Burn Rate (monthly) <$300K
Strategic financing completed with Pharmascience in May 2012
$17.25M total investment: $8.15M in non-dilutive capital and $9.1M
for 10.4% stake
Operations funded into 2018
1Does not include stock option grants 17
18. Governance and Shareholders
Board of Directors Company / Experience Management Title
Dr. Francesco Bellini President and Chief
Roberto Bellini
(Chair) Executive Officer
Senior Vice President, Drug
Dr. Denis Garceau
Franklin Berger Development
François Desjardins Vice President, Finance
Charles Cavell
Vice President, Business
Tony Matzouranis
Development
Hélène Fortin LAROSE FORTIN CA Inc.
Pierre Larochelle
Shareholder Ownership
Donald Olds Bellini Family ≈ 30%
Joseph Rus Power Corporation ≈ 30%
Dr. Martin Tolar Pharmascience ≈ 10%
Roberto Bellini Visium Asset Management ≈ 10%
18
19. Milestones
Milestones (12 months)
Past Execution Completion of recruitment of Long Term Value
Attractive KIACTA™ Phase III
partnership with for Confirmatory Study Results of Phase III
Kiacta Confirmatory Study
Additional KIACTA™ and auction of
Execution of global activities: KIACTA™
Kiacta Phase III
Launch of open label Sale or spin-out of
Confirmatory Study
extension study VIVIMIND business
Cashflow positive
Market and pricing BLU8499 Phase IIa
VIVIMIND business
assessment study results
Partnership for
Japan orphan drug
BLU8499
designation
Strong balance
BLU 8499 Pre-Phase II
sheet
package completion
VIVIMIND partnerships
Short-term milestones driving long-term value
19
