Bellus Corporate Presentation July 2016
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Transcript of Bellus Corporate Presentation July 2016
Corporate Presentation (TSX: BLU)
Roberto BelliniPresident and Chief Executive OfficerTwitter: @rbellini
July 25, 2016
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Forward Looking StatementsCertain 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.
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At BELLUS, we are focused on developing drugs for rare diseases starting with conditions that affect the kidneys.
Significant cash runway with multiple shots on goal
Company Highlights
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• 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
Late stage pipeline focused mainly on developing innovative drugs for rare diseases
Pipeline and Asset Overview
ShigamabsHUS
PRECLINICAL PHASE 1 PHASE 2 PHASE 3
Wholly-owned
KIACTAAA amyloidosis Partnered (~50% economic interest)
MARKET
Partnered (~50% economic interest)KIACTASarcoidosis
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FB Health
Licensed IP (Royalty)
ALZ-801Alzheimer’s Disease
Licensed IP (Royalty)
AMO-01Fragile X Syndrome
Equity Stake (5.7%)
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A rare and deadly kidney disease with no specific treatment
FOR AMYLOID A (AA) AMYLOIDOSIS
Disease and Mechanism of Action
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CHRONIC INFLAMMATION
SERUM AMYLOID APRECURSOR (SAA) PROTEIN
AA PROTEIN + GLYCOSAMINOGLYCANS (GAGs)
ORGAN DAMAGE, IN PARTICULAR TO KIDNEYS LEADING TO DIALYSIS
REDUCTION IN FIBRIL FORMATION & DEPOSITION
Converts toAA Protein
Generatescytokine cascade
(TNFα / IL-1 / IL-6) and increases SAA levels
Rheumatic ConditionsInflammatory Bowel DiseaseChronic InfectionsFamilial Mediterranean Fever
KIACTA™ blocksAA + GAGs interaction
Systemic Amyloid A Fibril Formation & Deposition
KIACTA designed to bind AA amyloid, slow down disease progression and delay dialysis 7
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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
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
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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
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Analysis of complete dataset on-going with final assessment and next steps to be determined in 2H
201610
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.
Second KIACTA Indication – Sarcoidosis
INDICATION
DEVELOPMENT
Chronic sarcoidosis, a rare disease that causes lung scarring and decreased lung functionKIACTA target Serum Amyloid A plays key role in disease
Also partnered with AuvenLicensed IP from Mount Sinai Hospital New York
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A rare disease primarily affecting the kidneys of children
FOR STEC RELATEDHEMOLYTIC UREMIC SYNDROME (SHUS),
SHIGAMABSHIGAMAB
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%
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SHIGAMAB BINDING NEUTRALIZES TOXIN
WHICH IS THEN ELIMINATED
90%
SPONTANEOUS RESOLUTION WITH SOME LONG-TERM SEQUELAE
INCLUDING HYPERTENSION
SHIGAMAB
TREATMENT
PROGRESSION TO HUS-HEMOLYTIC ANEMIA
-THROMBOCYTOPENIA-ACUTE RENAL FAILURE
Shigamab is a first-in-class, antibody-based therapy
for the treatment of sHUS
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
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1 2 3 4 5 6 7 8 9 10 11 1202468
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Survival in STEC Mouse Model
Control Treated 24hrsTreated 48hrs Treated 72hrs
Time (Days Post Infection)Sur
viva
l (#
anim
als)
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)
0
100
200
300
400
500
600
700
800
Average Lipocalin-2 (NGAL) Levels (n)
PBS Shigamab
LLip
ocal
in-2
, ng/
mL
**
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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
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
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Cases of HUS: Italian Registry 2000-2014
MARKET SIZE
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 childrenNo allergic or hypersensitivity reactionsPlasma half-life = 9 days
Objective to reduce need for dialysisStudy design currently being finalized
Ingestion Colonization Toxin Secretion
-4 0 2 4 6 8-2
Shigamab intervention*
Diagnosis ofHUS
50% No need for dialysis
*within 24 hours of HUS diagnosis
Days
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Dialysis 50%
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- AMO-01 for Fragile X Syndrome (FXS)
- ALZ-801 for Alzheimer’s Disease
- Equity stake in FB Health
OTHER PROJECTS AND ASSETS
SHIGAMABOTHER
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
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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.
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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
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2013 2014 2015 -
1,000
2,000
3,000
4,000
5,000
6,000
7,000
FB Health Annual Sales (in €000)
Corporate
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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
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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
Shareholder Ownership
Bellini Family ≈ 32%
Power Corporation ≈ 29%
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
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.
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