bellus corporate presentation - february 2016
TRANSCRIPT
Corporate Presentation (TSX: BLU)
Roberto BelliniPresident and Chief Executive Officer
Twitter: @rbellini
February, 2016
r
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.2
3
At BELLUS, we are focused on developing drugs for rare
diseases starting with conditions that affect the kidneys.
Regulatory advantage
Premium pricing
Market protection
Smaller clinical trials
Efficient commercialization
strategies
4
Small patient numbers, BIG opportunity
Value driving rare disease pipeline fully funded through key
milestones
Investment Highlights
5
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 full funded through KIACTA Phase III and exit
process
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
6
Lead Phase III Product Candidate
7
A rare and deadly
kidney disease with
no specific treatment
FOR AMYLOID A (AA)
AMYLOIDOSIS
Disease and Mechanism of Action
8
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
Patient Population
Source: Navigant Consulting 2014
10,000-
15,000potential KIACTATM
patients in the United
States and Europe
MARKET RESEARCHNavigant Consulting conducted
extensive primary and secondary
research including over 60
interviews with treating physicians
and key opinion leaders in the
United States and Europe
9
PRICING
Orphan drug designation granted with
market protection in the U.S. (7 years),
Europe and Japan (10 years)
Intellectual property protection to 2031
PROTECTION
Disease with large unmet medical need
and no specific treatment
Clear pharmaco-economic
rationale due to high cost of kidney
disease
Premium pricing for comparative rare
disease drugs
Market Considerations
KIACTA is well positioned to achieve premium pricing in line with
comparable rare disease drugs10
Drug U.S. Patients Disease Price
Vyndaqel 1,500Transthyretin amyloid
polyneuropathy$200K
Gattex 9,500 Short Bowel Syndrome $295K
Kalydeco1,350
Cystic Fibrosis (G551D
mutation)$335K
Procysbi500 Nephropathic cystinosis $250K
Juxtapid 3,000Familial
hypercholesterolemia$250K
Jakafi1,500 Splenomegaly $87K
COMPARABLES
KIACTA™ – Addressable Market
Source: Navigant Consulting 2014 11
Estimated Peak Annual Sales
$600 Million-$1Billion
KIACTA Eligible Patients
10-15 Thousand
Expected Pricing Per Patient Per Year
$200-$275 Thousand
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
12
POTENTIAL ACQUIRERSPharma/Big biotech with inflammation and/or nephrology franchise
Orphan disease focused biotech
Exit Strategy
Strong M&A environment for rare disease products13
Company Main Drug / Disease Stage Transaction
Synageva Kanuma/ LAL-D Registration (no sales)Acquired by Alexion in June 2015 for
$8.4B
NPS
Gattex / Short Bowel
Syndrome Market ($350M in sales)Acquired by Shire in January 2015 for
$6.2B
Scioderm Zorblisa / E. Bullosa Phase 3 (no sales)
Acquired by Amicus in August 2015 for
$230M upfront plus $600M in
milestones
Tripex Quincair / Cystic Fibrosis Registration (no sales)
Acquired by Raptor in August 2015 for
$68M upfront plus $350M in
milestones and royalties
RECENT RARE DISEASE M&A
0
5
10
15
20
25
30
35
40
45
50
Placebo
KIACTA
Composite
Endpoint (Time to
First Worse
Event)
Doubling
Serum
Creatinine
50%
Decrease
Creatinine
CIearance
Dialysis/
ESRD
Num
ber
of W
ors
e E
vents
14
*
*
**
Strong Clinical Results in Phase II/III Study
Landmark study in AA
amyloidosis: 183 patients
treated for 2 years
Important benefits for
patients on drug:
Statistically significant (p-
value=0.025) reduction in
number and risk of
reaching worsening
kidney event
Important delay in
reaching dialysis
*p<0.05
**p<0.01
Clean safety profile without any important differences
between groups in Phase II/III study15
KIACTA™ – Clean Safety Profile
98%
36%
23%
93%
42%
25%
0%
20%
40%
60%
80%
100%
Adverse Events Serious Adverse Events Discontinuations due toAdverse Events
% o
f P
ati
en
ts
KIACTA
Placebo
16
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
Marketing approval
based on achieving
comparable result with
lower statistical bar than
first Phase III Study
PHASE III CONFIRMATORY STUDY
183 patients in 13 countries
Composite primary endpoint based on patients reaching kidney function worsening events
Target statistical significant of p=0.01
Key entry criteria based on kidney function:
High proteinuria (>1 g/d) or low creatinine clearance (< 60 ml/min/1.73m2)
Fixed treatment duration of 2 years
74 kidney function worsening events
PHASE II/III STUDY
More patients
261 patients in >25 countries
Lower statistical bar to achieve
success
Primary endpoint with target statistical significant of p=0.05
Enriched patient population
High proteinuria (>1 g/d)
Increased power
Event driven trial to conclude on reaching
120 events
KIACTA™ – Phase III Confirmatory Study
17
Key improvements made to increase chance of successful study17
Study enrolled with 261 patients
Study completed with 120 events
reached (January 2016)
Topline data expected in Q2 2016
Phase III Confirmatory Study
18
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 triggering
disease
Agreement with Mount Sinai Hospital
New York to start Phase 2 proof-of-
concept study
IND filing expected in 1H 2016
19
Second Rare Disease Product Candidate
20
A rare disease
primarily affecting
the kidneys of
children
FOR STEC RELATED
HEMOLYTIC UREMIC
SYNDROME (SHUS),SHIGAMABSHIGAMAB
Disease Course and Mechanism of Action
E. COLI INGESTION
GUT COLONIZATION AND
SECRETION OF TOXIN
INTO BLOODSTREAM
TOXIN MAY BE CARRIED
BY PMNs IN
BLOODSTREAM
SYMPTOMS: BLOODY
DIARRHEA
SHIGAMAB BINDING
NEUTRALIZES TOXIN
WHICH IS THEN
ELIMINATED
Shigamab
Antibody
Day -4 Day 0 Day 4 Day 8
TOXIN BINDS TO GB3
RECEPTORS ON KIDNEY
LEADING TO STEC-HUS.
OUTCOMES:
-CHRONIC KIDNEY DISEASE /
HYPERTENSION: 40%
-ENCEPHALOPATHY / DEATH: 5%
-RESOLUTION: 55%
21
90%
SPONTANEOUS
RESOLUTION
10%
SHIGAMAB TREATMENT
Data presented at VTEC conference September 14-16
Mice rescued from shigatoxin induced weight loss and kidney injury up
to 4 days post intoxication
Shigamab Overview
NEXT STEPS (12 MONTHS)
MARKET OPPORTUNITY
CLINICAL
Further animal model data in treatment of sHUS
Meetings with regulators to agree on clinical development plan
2,000-3,000 estimated annual cases of sHUS in developed countries,
principally children
$100-200 million annual sales opportunity
Safe and well tolerated in target pediatric population
25
PRE- CLINICAL
Clean capital structure and cash runway through potential exit
Corporate
23
Capital Markets (as of February 23th, 2015)
Ticker TSX: BLU
Shares (Basic) 54.7M
Shares (Fully Diluted) 65.7M
Market Capitalization (FD) C$76M
23
Finance
Cash (December 31, 2015) C$9.7M
Burn rate (monthly) <C$300K
Shareholder Ownership (FD)
Bellini Family ≈ 29%
Power Corporation ≈ 27%
Pharmascience ≈ 10%
Governance and Shareholders
24
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 BelliniPresident and Chief Executive
Officer
Dr. Denis GarceauSenior Vice President, Drug
Development
François Desjardins Vice President, Finance
Tony MatzouranisVice President, Business
DevelopmentLAROSE FORTIN CA Inc.
24
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
25