The document provides an overview of Bellus Health Inc., a biopharmaceutical company focused on developing treatments for rare diseases. It discusses Bellus' lead drug candidate KIACTA, currently in a Phase III confirmatory study for AA amyloidosis, a rare and deadly kidney disease with no approved treatments. The Phase II/III study showed positive efficacy and safety results. The document outlines the Phase III study design and expected milestones, including completion of patient enrollment and top-line data readout. It also summarizes Bellus' pipeline, partnership, financial position, and potential exit strategy following Phase III results for KIACTA.
1. Bloom Burton Healthcare
Investor Conference
Roberto Bellini
President and Chief Executive Officer
Twitter: @rbellini
May 2, 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. 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, 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 in
relation to indemnity agreements, achievement of forecasted clinical trial milestones, 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 III Confirmatory Study is
dependent upon many factors, including clinical sites activation, patient enrollment rate, patient
drop-out rate and occurrence of clinical endpoint events, and the sharing of proceeds between
Auven Therapeutics and BELLUS Health Inc. from potential future revenue of KIACTA™ is
dependent upon a number of factors, including the quantum of proceeds.
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 presentation. 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 BELLUS Health Inc.’s public fillings
including the Annual Information Form for further risk factors that might affect BELLUS Health
Inc. and its business.
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3. 3
At BELLUS, we are focused on developing drugs for rare
diseases starting with conditions that affect the kidneys.
4. Value driving rare disease pipeline fully funded through key
milestones
Company Highlights
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Late-stage pipeline with 4 projects targeting rare diseases
Lead drug candidate, KIACTA, in Phase III Confirmatory Study for
AA amyloidosis
Rare and deadly kidney disease with no treatment
Phase II/III study completed with positive efficacy and clean safety
Similar and confirmatory Phase III study completed (Data expected in Q2
2016)
Potential peak market sales of $600M-$1B
Potential exit to commercial partner following Phase III data
Business plan fully funded through KIACTA Phase III and exit
process
5. Late stage pipeline focused on developing innovative drugs for
rare diseases
Pipeline of Products
Shigamab
sHUS
DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III
KIACTA™
AA amyloidosis
MARKET
AL amyloidosis
KIACTA™
Sarcoidosis
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6. Lead Phase III Product Candidate
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A rare and deadly
kidney disease with
no specific treatment
FOR AMYLOID A (AA)
AMYLOIDOSIS
7. Disease and Mechanism of Action
7
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 7
8. KIACTA™ – Addressable Market
Source: Navigant Consulting 2014 8
Estimated Peak Annual Sales
$600 Million-$1Billion
KIACTA Eligible Patients
10-15 Thousand
Expected Pricing Per Patient Per Year
$200-$275 Thousand
9. 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 III
Confirmatory Study results
Auven Therapeutics Partnership for KIACTA™
BUSINESS PLANAUVEN PARTNERSHIP
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10. 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
Landmark study in AA
Amyloidosis: 183 patients
treated for 2 years
Composite endpoint based on
patients reaching events of
decreasing kidney function or
death
Statistically significant
primary endpoint (Cox
Proportional Hazard Ratio;
p=0.025)
Clinically meaningful
treatment effect with 42%
reduction in risk of reaching
worsening kidney event
Clear regulatory path to
approval based on
confirmatory study
Composite
Endpoint (Time to
First Worse
Event)
Doubling
Serum
Creatinine
50%
Decrease
Creatinine
Clearance
Dialysis/
ESRD
Death
NumberofPatientEvents
10
*
*
**
KIACTA™ - Robust Clinical Results in Phase II/III
*p<0.05
**p<0.01
11. PHASE III 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
Composite primary endpoint
based on patients reaching
kidney function worsening
events
Target statistical significance of
p=0.01
PHASE II/III 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
Lower statistical bar to achieve
success
Primary endpoint with target statistical
significance of p=0.05
KIACTA™ – Phase III Confirmatory Study
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Key improvements made to increase chance of successful study
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12. Study enrolled with 261 patients
Study completed with 120 events
reached (January 2016)
Topline data expected in Q2 2016
Phase III Confirmatory Study
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13. Clean capital structure and cash runway through potential exit
Corporate
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Capital Markets (as of April 28th, 2016)
Ticker TSX: BLU
Shares (Basic) 54.7M
Shares (Fully Diluted) 65.7M
Daily Volume ~100K
Market Capitalization (FD) ~C$110M
13
Finance
Cash (December 31, 2015) C$9.7M
Burn rate (monthly) <C$300K
14. Shareholder Ownership (FD)
Bellini Family ≈ 29%
Power Corporation ≈ 27%
Pharmascience ≈ 10%
Governance and Shareholders
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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.
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15. 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 for
Sarcoidosis (1H 2016)
Shigamab animal data (1H 2016)
Shigamab clinical trial design (1H
2016)
Significant news flow and value inflection point in 2016
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
Milestones
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16. Connect With Us
Follow us on Twitter: @BELLUSHealth
Join our LinkedIn group
Read our blog @ www.bellushealth.com
Join our mailing list