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Annual Meeting
2013
Roberto Bellini President and Chief Executive Officer
May 15, 2013
1
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, 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
BELLUS Health Inc. does business, stock market volatility, fluctuations in costs, and changes to
the competitive environment due to consolidation, achievement of forecasted burn rate,
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. The length of
the KIACTA Phase III Confirmatory Study is dependent upon many factors including clinical
sites activation, patient enrolment rate, patient drop out rate and occurrence of clinical endpoint
events.
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 Company and its business. 2
Business Model
3
Focused
Development
in areas of
high unmet
medical need
Strategic
Partnerships
with strong
industry
players
Identification
of New
Product
Opportunities
Focused
Development
4
Dedicated to scientific excellence
Targeted areas of high unmet medical need
Potential for strong shareholder return
Pipeline of Products
Pharmaceutical
Nutraceutical
BLU8499
Alzheimer’s
disease
VIVIMIND™
Memory protection
DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III
COMMERCIA-
LIZATION DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III
NDA/MAA
KIACTA™
AA amyloidosis
5
KIACTA™
Phase II/III clinical trial showing statistically
significant primary efficacy endpoints (p value =
0.025) and clean safety profile
Patient population estimated at 35,000 to 50,000 in
the USA, Europe and Japan1
Orphan Drug Status in the USA and Europe
provides 7 and 10 years market exclusivity upon
commercialization, respectively
KIACTA™ peak annual revenues projected at
$400-600M1 (USA, Europe, Japan)
1 Market assessment by Frankel Group in April 2009.
For AA Amyloidosis, a rare and deadly
disease with no treatment
Market
opportunity
Clinical
evidence
6
Phase III Confirmatory Study
183 patients
Statistically significant composite primary endpoint (p=0.025) principally based on patients reaching kidney function events of worsening:
Doubling serum creatinine
50% decrease in creatinine clearance
Reaching ESRD/dialysis
Death
Fixed treatment duration of 2 years
Completed Phase II/III Study
230 patients
Composite primary endpoint
(target p<0.05) based on patients
reaching kidney function events of
worsening:
Persistent 80% increase serum creatinine
Persistent 40% decrease in creatinine
clearance
Reaching ESRD/dialysis
Event driven trial to conclude on
attainment of 120 events (~90%
power)
KIACTA™ - Phase III Confirmatory Study Design
7
Key improvements made to increase robustness
of confirmatory study
KIACTA™ - Study Progress
Recruitment
>65 sites in >25
countries actively
recruiting
152 of 230 patients
enrolled
Recruitment expected
to be completed in 1H
2014
8
Completion
Event driven trial to
complete on reaching
120 events
Study expected to be
completed in 2017
9
Lower financial risk
Maintain significant upside value
Bring important external expertise
Strategic
Partnerships
Auven Partnership
Global pharmaceutical fund
Strong business expertise
from General Managing
Partner Stephen Evans-
Freke (Sugen, Royalty
Pharma)
Strong drug development
expertise from General
Managing Partner Dr. Peter
Corr (Pfizer)
Financial Implication
US$10M in upfront payments
Auven Therapeutics funding 100% of KIACTA™’s Phase III Confirmatory Study
≥ US$50M in development investments by Auven Therapeutics
Proceeds of any eventual transaction expected to be shared 50%-50% between BELLUS Health and Auven Therapeutics
Auven (previously Celtic) Therapeutics - KIACTA™ Partnership
10
KIACTA™ - Exit Strategy
11
RARE DISEASE DRUGS IN THE NEWS
Alnylam Gets $22.5M From Genzyme for Asia Rights to Amyloidosis Drug (October 2012)
Sarepta shares rocket up on
stellar muscular dystrophy trial
results (October 2012) ALEXION TO AQUIRE ENOBIA
PHARMA FOR UP TO $1.08 BILLION
(December 2011)
BioMarin shares pop on strong pivotal
data for rare disease drug (November
2012)
Sale or commercial partnership at end of study or following close
of recruitment
Novartis signs $665m option deal with
Selexys for sickle cell disease drug
(September 2011)
VIVIMIND™
Partnerships
COUNTRY / REGION PARTNER Status
ITALY
FB Health Commercial
CANADA Advanced
Orthomolecular
Research
Commercial
MIDDLE EAST Agahan Group Commercial
GREECE Integris Commercial
ISRAEL LevPharm Regulatory
TAIWAN Lifenergy Regulatory
SOUTH KOREA IL-SUNG Regulatory
12
Growing cash flow positive business
VIVIMIND Revenues ($K)
0
100
200
300
400
500
2010 2011 2012
BLU8499 – Asclepios Partnership
13
Long term animal toxicity needed
Phase IIa proof of concept study in mild
apoE4+ Alzheimer’s disease patients
Expected to begin in 2014
Development
Plan
Partnership with Asclepios Bioresearch in
September 2012 to finance development of
BLU8499 in Alzheimer’s Disease
Investment of ~$4M in non-dilutive capital
Parties expected to share future proceeds
approximately equally
Partnership
Focused development plan to demonstrate effectiveness in
targeted patient population
Shareholders and Financials
Bellini Family ≈ 30%
Power Corporation ≈ 30%
Pharmascience ≈ 10%
Float ≈ 30%
14
Financial Position
Cash (December 31st,
2012) >$18M
Burn Rate (monthly) <$300K
Strong shareholder base and financial position
with cash until mid-2018
Fully
Diluted Name
15
New Opportunitie
s
Leverage existing intellectual property to start new project(s)
Leverage know how and expertise to add value to acquired project(s)
New
Opportunities
Exploring new projects leveraging existing intellectual
property
16
KIACTA for AA
Amyloidosis
AL Amyloidosis
ATTR Amyloidosis
Sarcoidosis
Bellus Strengths
Expertise of team (drug
development, business
development, capital
markets)
Public vehicle
Strong shareholder base
New Project
High unmet medical need
Close to or in clinical development
Ideally in nephrology or orphan indication
Clear market relevance
New Opportunities – External Sources
17
Leverage BELLUS strengths to develop acquired
or in-licensed new projects
Attractive partnership
with Auven
Therapeutics for
KIACTA
Execution of global
KIACTA Phase III
Confirmatory Study
Cashflow positive
VIVIMIND business
Partnership for
BLU8499
Strong balance sheet
• Completion of recruitment of
KIACTA™ Phase III
Confirmatory Study
• Additional KIACTA™
activities:
- Launch of open label
extension study
- Market and pricing
assessment
- Japan orphan drug
designation
• Expansion of product pipeline
• BLU8499 Pre-Phase II package
completion
• VIVIMIND partnerships
Summary
18
Past Execution
Milestones (12 months)
Long Term Value
• Results of Phase III
Confirmatory Study
and auction of
KIACTA™
• Sale or spin-out of
VIVIMIND business
• BLU8499 Phase IIa
study results
Short-term milestones driving long-term value
Annual Meeting
2013
Roberto Bellini President and Chief Executive Officer
May 15, 2013
19