This corporate presentation by Roberto Bellini, President and CEO of BELLUS Health Inc., provides an overview of the company. BELLUS is focused on developing products for amyloid-related diseases, including their late-stage product KIACTA for AA amyloidosis, a rare kidney disease. The presentation summarizes the positive Phase II/III results for KIACTA, the confirmatory Phase III trial funded through a partnership with Celtic Therapeutics, and the potential market opportunity. It also provides an overview of their nutraceutical product VIVIMIND and early-stage asset NRM8499 for Alzheimer's disease, as well as the company's financial position and key milestones for 2012.
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
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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
Late-stage product pipeline
BUSINESS MODEL
Focused on building value for clinical
stage health products in critical unmet
medical needs
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4. Pipeline of Products
PHARMACEUTICALS DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III NDA/MAA
Eprodisate
(KIACTA™)
AA amyloidosis
NRM8499
Alzheimer’s
disease
COMMERCIA
NUTRACEUTICAL DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III -LIZATION
Homotaurine VIVIMIND™
Memory protection
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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 Celtic Therapeutics to conduct
Partnership and finance confirmatory phase III clinical trial
Marketing approval based on confirming safety
Confirmatory and efficacy of phase II/III study
phase III
clinical trial Actively recruiting patients
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6. AA Amyloidosis
Rare and life-threatening disease
Associated with chronic inflammatory diseases
65% of patients are rheumatoid arthritis patients
AA protein accumulates in major organs, mainly kidneys, leading to:
Rapid and significant deterioration of kidney function, and
Eventual progress to dialysis
Kiacta slows the deterioration of kidney function by preventing amyloid A
protein fibril formation, accumulation and deposition
Orally bioavailable small molecule
Na+-03S S03-Na+
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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
With global fund Celtic US$10M in upfront payments
Therapeutics
≥ US$35M in investments by
Celtic Therapeutics funding Celtic Therapeutics
100% of KIACTA™’s
confirmatory phase III clinical Proceeds of any eventual
trial transaction expected to be
shared 50%-50% between
Auction process for the BELLUS Health and Celtic
commercialization rights of Therapeutics
KIACTA™ on completion of
Phase III clinical trial
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9. KIACTA™ - Robust Clinical Results in Phase II/III
Statistical significant on
primary endpoint
(p value <0.05) and clinically
meaningful treatment effect
(42% reduction in risk)
Calculated 2-year delay to
dialysis for patients on
KIACTATM
Number of
Relative Risk
Events Clean safety profile
NC-503 Placebo HR 95% C.I. P value
Primary
Agreement with FDA/EMEA
composite for confirmatory phase III
29 45 0.58 0.37, 0.93 0.025
clinical trial
endpoint
(First “worse”
event)
Marketing approval based on
Doubling SCr 9 17 0.41 0.19, 0.86 0.019
positive result (p value <0.05)
50% decrease CrCl 19 31 0.48 0.28, 0.82 0.008
from confirmatory study with
Dialysis/ESRD 7 13 0.54 0.22, 1.37 0.20 same scope of first phase III
Death 5 5 0.95 0.27, 3.29 0.94 clinical trial
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10. KIACTA™ - Confirmatory Phase III Study
CONFIRMATORY PHASE III
COMPLETED PHASE II/III STUDY STUDY
183 patients in 13 countries 230 patients in 28 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 2 power)
years
Key improvements made to
increase robustness of
confirmatory study
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11. KIACTA™ - Study Progress
Recruitment1 Completion
>70 sites in 26 Event driven trial to
countries actively complete on reaching
recruiting 120 events
90 patients enrolled Study expected to be
completed in 2H 2015
Recruitment expected
to be completed in 2H
2013
Patient baseline characteristics and demographics to date are similar
to those in the first Phase III study
1 Data as of May 10, 2012 11
12. KIACTA™ - Providing Base Value
AUCTION
DEVELOPMENT
LOW RISK PROCESS WITH
COST FULLY
CONFIRMATORY EQUALLY
FUNDED BY
PHASE III STUDY SHARED
CELTIC
PROCEEDS
+
SIGNIFICANT SHAREHOLDER
VALUE BASE
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13. VIVIMIND™
Nutraceutical for memory protection
Canada: Protects the hippocampus
Health Claims
Italy: Enhances cognitive function and memory
Regulatory approvals in Italy and Spain obtained
Regulatory in 2009
Approval
NPN number issued by Health Canada in 2010
Partnerships for Italy, Canada, Greece, Middle East
and Israel
Partnerships Pursuing efforts to conclude additional partnerships
in other territories: creating a distributor network
worldwide
Growing cash flow positive business
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14. NRM8499
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 USA
alone
Completed in 67 young and elderly healthy
subjects
Phase I Safe and well tolerated at the intended
clinical trial therapeutic dose
Better gastrointestinal tolerability and
pharmacokinetic profile than tramiprosate
Company seeking a potential partnership to
pursue development process
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15. Strategic Partnership and Financing
Pharmascience Strategic Investment
Leading Canadian $17.25M total investment:
manufacturer of generic drugs
$8.15M in non-dilutive capital
Private company $9.1M for 10.4% stake
Annual sales >$700M 1 board member
R&D budget >$30M
More than 1,300 employees
Pharmascience transaction closed on May 25th
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16. Financial Position and Capital Structure
Capital Structure
Basic Shares Outstanding 47M
Fully Diluted Shares Outstanding 61M
Financial Position
Cash (as of May 31st, 2012) >$20M
Burn Rate (monthly) <$300K
Operations funded beyond 2016
Kiacta Phase 3 data expected in 2015
Additional funds can be used for new potential projects
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17. Governance and Shareholders
Board of Directors Company / Experience Management Title
Dr. Francesco Bellini President and Chief
Roberto Bellini
(Chair) Executive Officer
Senior Vice President,
Dr. Denis Garceau
Franklin Berger Drug Development
François Desjardins Vice President, Finance
Charles Cavell
Vice President, Business
Tony Matzouranis
LAROSE FORTIN CA Inc.
Development
Hélène Fortin
Pierre Larochelle
Shareholder Ownership
Donald Olds Bellini Family ≈ 30%
Joseph Rus Power Corporation ≈ 30%
Dr. Martin Tolar Pharmascience ≈ 10%
Roberto Bellini
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18. Milestones
2012 Milestones
Past Execution Long Term Value
Continued execution of
Attractive KIACTA Confirmatory phase Results of confirmatory
partnership with III clinical trial: phase III clinical trial
Celtic for Kiacta and auction of
Launch of Japan sites KIACTA™
Execution of global and receipt of Japanese
Kiacta Phase III orphan drug designation Sale or spin-out of
confirmatory study nutraceutical business
Partnership for NRM8499 for
Building cashflow Phase 2 study NRM8499 Phase 2
positive VIVIMIND study results
business Financial partner with no
shareholder dilution
Fully financed
business plan Large regional partnership
for VIVIMIND™
Short-term milestones driving long-term value
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