1. Corporate Presentation (TSX: BLU)
Roberto Bellini
President and Chief Executive Officer
Twitter: @rbellini
July 25, 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 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 development of KIACTA™ 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™ development process 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.
4. Significant cash runway with multiple shots on goal
Company Highlights
4
• Pipeline targeting multiple rare diseases
KIACTA, partnered with US private equity fund Auven Therapeutics, has
completed a Phase 3 Study for AA amyloidosis, a rare and deadly kidney disease
KIACTA is also Phase 2 ready for Sarcoidosis, a rare lung disease
Shigamab is a monoclonal antibody preparing for Phase 2 in sHUS, a rare kidney
condition that mostly affects children
• Other projects and assets
Royalty on AMO Pharma’s AMO-01 in preparation for Phase 2 study in Fragile X
Syndrome
Small equity stake in niche Italian specialty pharma
Royalty on Alzheon’s drug ALZ-801 in preparation for Phase 3 study in
Alzheimer’s disease
• Cash of $9.0M as of March 31st with low operational burn rate
5. Late stage pipeline focused mainly on developing innovative drugs for rare
diseases
Pipeline and Asset Overview
Shigamab
sHUS
PRECLINICAL PHASE 1 PHASE 2 PHASE 3
Wholly-owned
KIACTA
AA amyloidosis Partnered (~50% economic interest)
MARKET
Partnered (~50% economic interest)
KIACTA
Sarcoidosis
5
FB Health
Licensed IP (Royalty)
ALZ-801
Alzheimer’s Disease
Licensed IP (Royalty)
AMO-01
Fragile X Syndrome
Equity Stake (5.7%)
6. 6
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. 8
Phase 2/3 Study
New England Journal of
Medicine publication of
Phase 2/3 data showing
KIACTA slows decline of
renal function in AA
amyloidosis patients
Agreement reached with
FDA to conduct
confirmatory Phase 3
Study
Marketing approval
based on achieving
comparable result of
Phase 2/3 Study
9. Experienced and knowledgeable partner working on lead project
Auven is a global biotech private
equity group
Founded by Managing Partners
Stephen Evans-Freke (SUGEN,
Fibrogen, Royalty Pharma) and
Peter Corr (Pfizer)
Partnered on KIACTA project in
2010
All indications including AA amyloidosis
and Sarcoidosis
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
Auven Therapeutics Partnership for KIACTA
PARTNERSHIPBACKGROUND
9
10. Phase 3 Study- Time to First Event
of Renal Function Decline
Phase 2/3 Study – Time to First
Event of Renal Function Decline
Phase 3 Study – Negative Top Line Data
10
Analysis of complete dataset on-going with final assessment and next
steps to be determined in 2H 2016
10
P=0.025
Dember et al. June 7, 2007. New England Journal of Medicine. Vol 356 (23) 2349-2360. Obici L., July 6, 2016. Presented at International Symposium of Amyloidosis.
11. 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
Also partnered with Auven
Licensed IP from Mount Sinai
Hospital New York
11
12. 12
A rare disease
primarily affecting
the kidneys of
children
FOR STEC RELATED
HEMOLYTIC UREMIC
SYNDROME (SHUS),
SHIGAMABSHIGAMAB
13. sHUS Disease Course and Shigamab Mechanism of
Action
E. COLI INGESTION
GUT COLONIZATION AND
SECRETION OF TOXIN
INTO BLOODSTREAM
LONG-TERM SEQUELAE
- CHRONIC KIDNEY DISEASE
- HYPERTENSION
- ENCEPHALOPATHY
- DEATH
10%
1
3
SHIGAMAB BINDING
NEUTRALIZES TOXIN
WHICH IS THEN
ELIMINATED
90%
SPONTANEOUS
RESOLUTION WITH SOME
LONG-TERM SEQUELAE
INCLUDING HYPERTENSION
PROGRESSION TO HUS
-HEMOLYTIC ANEMIA
-THROMBOCYTOPENIA
-ACUTE RENAL FAILURE
Shigamab is a first-in-class, antibody-based therapy
for the treatment of sHUS
14. Shigamab: Mouse Pre-Clinical Proof-of-Concept Studies
In preclinical studies, Shigamab™
demonstrated compelling evidence
as a potential treatment for sHUS:
Lead to increased survival in a lethal
STEC-infected mouse model, even
when treatment was delayed by up to
72 hours post-infection
Reduced kidney injury, as measured
by kidney biomarkers, in Stx2-
intoxication mouse model when
administered up to 4 days post-
intoxication
14
0
2
4
6
8
10
1 2 3 4 5 6 7 8 9 10 11 12
Survival(#animals)
Time (Days Post Infection)
Survival in STEC Mouse Model
Control Treated 24hrs
Treated 48hrs Treated 72hrs
0
100
200
300
400
500
600
700
800
Non-intox'd
(5)
d0 Stx2 +
PBS (7)/Ab(6)
d2 Stx2 +
PBS(7)/Ab(8)
d4 Stx2 +
PBS(5)/Ab(6)
d6 Stx2 +
PBS(5)/Ab(5)
LLipocalin-2,ng/mL
Average Lipocalin-2 (NGAL) Levels
(n)
PBS Shigamab
*
*
15. 15
Objective to evaluate the effect of delayed treatment with Shigamab on
development of Stx2-induced HUS in Baboons
Shigamab administered 24h or
48h post Stx2 injection in
baboons was shown to:
Rescue the animals from a
lethal dose of Stx2
Reduce Stx2-induced
kidney injury and protect
renal function
Reduce extra-renal
symptoms associated with
HUS (reduced
activity/alertness)
Shigamab: Baboon Pre-Clinical Proof-of-Concept
n=1 per group
16. Shigamab: Market Size
Estimated approximately 2,000 –
3,000 cases of sHUS annually in
industrialized nations
sHUS is an ultra-orphan disease
with completely unmet medical
need in a primarily pediatric
population
Premium pricing potential in line
with comparable acute care ultra-
orphan therapies
Potential peak annual sales
estimated at $150M
16
0
10
20
30
40
50
60
70
Cases of HUS: Italian Registry 2000-
2014
MARKET SIZE
17. Shigamab: Clinical
SAFETY FOCUSED PHASE 2 STUDY COMPLETED
NEXT STEP: EFFICACY FOCUSED PHASE 2 STUDY
Shigamab™ was safe and well tolerated at doses 1 and 3 mg/kg in
children
No allergic or hypersensitivity reactions
Plasma half-life = 9 days
Objective to reduce need for dialysis
Study design currently being finalized
Ingestion Colonization Toxin Secretion
-4 0 2 4 6 8-2
Shigamab
intervention*
Diagnosis of
HUS
50%
No need
for dialysis
*within 24 hours of HUS diagnosis
Days
17
Dialysis
50%
18. 18
- AMO-01 for Fragile X
Syndrome (FXS)
- ALZ-801 for
Alzheimer’s Disease
- Equity stake in FB
Health
OTHER PROJECTS AND
ASSETS
SHIGAMABOTHER
19. IP Licensed to AMO Pharma in 2014 in return for mid-single digit
percentage royalty/revenue share
Private company located in London, UK
Clinical stage pipeline focused on the
treatment of CNS and neuromuscular
diseases
Founded and led by seasoned industry
professionals (Novartis, Shire, GSK)
Raised US$25M in Q3 2015 from
Woodford Patient Capital Trust
Small molecule Ras-ERK pathway
inhibitor
Preclinical data shows AMO-01
successfully reversed abnormalities in
neuronal anatomy and cognitive and
behavioral deficits in transgenic mouse
models of FXS
Phase 2 expected to begin in 2016
FXS is deadly disease with no current
approved therapies that affects 180K
patients in the US
Potential peak annual sales of $2B
AMO Pharma’s AMO-01 for Fragile X Syndrome
AMO-01AMO PHARMA
19
20. IP licensed to Alzheon in 2013 in return for mid-single digit
percentage royalty/revenue share
Private company located in
Framingham, MA
Focused on Alzheimer’s disease
and other neurodegenerative
disorders
Founded and led by team with
significant industry and Alzheimer’s
success (Pfizer, Elan, CoMentis)
$10M Series A financing in April
2015
Small molecule oral inhibitor of
amyloid aggregation and
neurotoxicity discovered at Bellus
for the treatment of Alzheimer’s
disease
In preparation for Phase 3
focusing on treatment of
Alzheimer’s patients homozygous
for apolipoprotein E gene
Addresses multibillion dollar
market opportunity
Alzheon’s ALZ-801 for Alzheimer’s Disease
ALZ-801ALZHEON, INC.
20
21. 5.7% ownership stake in FB Health currently valued at $840K
Italy based specialty pharma
focused on neurology and
psychiatry
Distributes over 10 nutraceutical
and pharmaceutical products in
Italy
Growing and profitable business
Equity stake received as partial
consideration for sale of Vivimind to
FB Health in 2013
Equity Stake in FB Health
FB HEALTH
21
-
1,000
2,000
3,000
4,000
5,000
6,000
7,000
2013 2014 2015
FB Health Annual Sales (in €000)
22. Corporate
22
Capital Markets as of July 25th, 2016
Ticker TSX: BLU
Shares (Basic) 61.1M
Shares (Fully Diluted) 65.9M
Daily Volume ~370K
Market Capitalization ~$15M
22
Key Finance Items as of March 31st, 2016
Cash $9.0M
Net Receivables $0.2M
FB Health Investment $0.8M
Tax losses/assets $40M
Burn rate (monthly) ~$225K
23. Shareholder Ownership
Bellini Family ≈ 32%
Power Corporation ≈ 29%
Pharmascience ≈ 10%
Governance and Shareholders
23
Board of Directors Company / Experience
Dr. Francesco Bellini
(Chair)
Franklin Berger
Charles Cavell
Hélène Fortin
Pierre Larochelle
Murielle 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