This corporate presentation by BELLUS Health provides an overview of the company and its lead drug candidate KIACTA for the rare kidney disease AA amyloidosis. Key points include:
- KIACTA showed positive efficacy and safety in a Phase II/III trial, reducing worsening kidney events. A confirmatory Phase III trial has completed enrollment.
- AA amyloidosis has no approved treatments and represents a potential $600M-$1B market opportunity for KIACTA.
- The company has a late-stage pipeline of drugs for rare diseases and is fully funded through milestones. Positive Phase III data for KIACTA could lead to a commercial partnership.
Safety Data Exchange Agreements (Brief)
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Strategic management framework
Governance model
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Roles and responsibilities
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http://semoegy.com
Existing pharmacovigilance post-market surveillance system is based on reporting of adverse drug reactions to national databases in real-time and post-marketing safety studies.
Quantitative evaluation of incidence, prevalence trends, and patterns of use from reporting data is problematic due to under-reporting and missing data on exposure. This is especially true if drugs are used off-label, in populations they were not intended for, or in combination with other medications.
While the case study below emphasizes post-market surveillance of marketed drugs, the same principles apply to other areas within clinical research and drug development, outcomes research, monitoring, and evaluation of the quality of provided care and pharmaco-economic applications.
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Point of Care Testing Market and Forecast to 2016: Global Analysis - Market Overview
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Blood glucose test controls a lion’s market share of around 70% for the year 2011 and it is expected to continue its dominance till 2016. Rest of all the others point of care tests market share covered in this report are in single digit. Infectious disease testing market is expected to double by 2016 from its market of 2011. As countries are making the healthcare more and more accessible to people, the demand for various point of care testing is keep on rising. The point of care testing market has become an established sector worldwide and will continue to provide vital contribution in in-vitro diagnostics industry.
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10 Point of Care Test Segments Covered in this Report
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The paper makes a case for change in the way data on the safety of medicines is collected, structured, analyzed, visualized, and shared. Post-market surveillance shall move away from active reporting of individual case reports into national and international databases toward the collection and analysis of anonymous structured summary data from health care providers. The objective is to enable an analysis of total numbers of treated patients and treatment outcomes, including adverse drug reactions and off-label drug use, to provide meaningful, population-based, statistically valid, bias-free, real-time information on safety and efficacy of products on the market without endangering patients' privacy. Such approach would significantly reduce privacy concerns and add value for stakeholders who are interested in timely and accurate information on benefit:risk profile of medicinal products.
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For details visit: www.PharmXL.com
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Safety Data Exchange Agreements (Brief)
An integral part of enterprise risk management systems
Strategic management framework
Governance model
Standards, mechanisms, and metrics for management oversight
Roles and responsibilities
Stakeholder collaboration
Procedural documents and SDEA templates
CAPA management processes
Supporting systems
Document repositories
This 10-minute lecture gives a step-by-step instruction on medical device definition. It covers the basic definition of a medical device, its key attributes, and intended purpose. A brief insight is provided into in vitro diagnostic medical devices. At the end of this lecture, the student will be able to differentiate a medical device from pharmaceutical and biological healthcare products. The student will also be able to decide whether a particular healthcare product comes under the definition of a medical device.
http://semoegy.com
Existing pharmacovigilance post-market surveillance system is based on reporting of adverse drug reactions to national databases in real-time and post-marketing safety studies.
Quantitative evaluation of incidence, prevalence trends, and patterns of use from reporting data is problematic due to under-reporting and missing data on exposure. This is especially true if drugs are used off-label, in populations they were not intended for, or in combination with other medications.
While the case study below emphasizes post-market surveillance of marketed drugs, the same principles apply to other areas within clinical research and drug development, outcomes research, monitoring, and evaluation of the quality of provided care and pharmaco-economic applications.
A presentation at "Linked Data in Sweden - Linköping University" showing the benefit of using WHODrug as linked data. Should this work be continued or is there no business value in it?
Investigated the market for cardiovascular medical devices and clinical research organizations (CROs) to understand what roles play in the development and FDA approval process for therapeutics.
Point of care testing market and forecast to 2016 global analysisRenub Research
Renub Research (http://www.renub.com/report/point-of-care-testing-market-and-forecast-to-2016-global-analysis-88) has announced the addition of the "Point of Care Testing Market and Forecast to 2016: Global Analysis" report to its offering
Point of Care Testing Market and Forecast to 2016: Global Analysis - Market Overview
The concept of Point of Care Testing (POCT), also known as bedside, near-patient testing and decentralized testing, relates to tests that are conducted by clinical operators at the site of patient care where immediate medical action is taken on the results. The fact that point of care (POCT) represents a departure from conventional laboratory medicine has created new opportunities in the field of diagnostics industry. Technical advancements over recent years have helped point of care testing (POCT) to grow with double digit CAGR from 2009 to 2011 and evolve into a vital diagnostic tool. It is predicted that point of care testing market will be approximately US$ 25 Billion by 2016.
Blood glucose test controls a lion’s market share of around 70% for the year 2011 and it is expected to continue its dominance till 2016. Rest of all the others point of care tests market share covered in this report are in single digit. Infectious disease testing market is expected to double by 2016 from its market of 2011. As countries are making the healthcare more and more accessible to people, the demand for various point of care testing is keep on rising. The point of care testing market has become an established sector worldwide and will continue to provide vital contribution in in-vitro diagnostics industry.
This 83 page report contains 26 Figures and 12 Tables provides a comprehensive analysis of the emerging point of care tests market segments, including their dynamics, size, growth, regulatory requirements, technological trends, competitive landscape, and emerging opportunities for instrument and consumable suppliers. Renub Research report entitled “Point of Care Testing Market and Forecast to 2016: Global Analysis” report also provides market landscape and market share information in the point of care testing market. The report brings together major merger & acquisition, distribution agreement, licensing deals information in point of care testing market. The report also entails major drivers and challenges of point of care testing market.
10 Point of Care Test Segments Covered in this Report
Blood Glucose Testing, Cardiac Marker Testing, Lipid Panel/Cholesterol Testing, Blood Coagulation Testing, Infectious Disease Testing, Urinalysis Testing, Drug of Abuse Testing, Fecal Occult Blood Testing, Pregnancy & Fertility Testing and Tumor Marker Testing
Big Data in Drug Safety: Making post-marketing surveillance in pharmacovigila...Arete-Zoe, LLC
The paper makes a case for change in the way data on the safety of medicines is collected, structured, analyzed, visualized, and shared. Post-market surveillance shall move away from active reporting of individual case reports into national and international databases toward the collection and analysis of anonymous structured summary data from health care providers. The objective is to enable an analysis of total numbers of treated patients and treatment outcomes, including adverse drug reactions and off-label drug use, to provide meaningful, population-based, statistically valid, bias-free, real-time information on safety and efficacy of products on the market without endangering patients' privacy. Such approach would significantly reduce privacy concerns and add value for stakeholders who are interested in timely and accurate information on benefit:risk profile of medicinal products.
A presentation on Pharmacovigilance System in United States.
We at PharmXL International Pvt. Ltd., offer wide range of services for pharma industry like Pharmacovigilance services, Clinical Trials services, Regulatory Affairs services, Medical writing services etc to comply with required regulatory obligations across major regions.
For details visit: www.PharmXL.com
Email us: contact@pharmxl.com
Medical device industry 2014 - A Healthcare Sector AnalysisVikas Soni
Medical Device Industry and its impact on Healthcare Sector of India for FY2014 along with Current Scenario of Global, US, EU and Indian Medical device market.
Leveraging Imaging and Wearable Technology For Agile Clinical TrialsPAREXEL International
Learn how to manage and overcome key challenges to deploying imaging and wearable technology effectively in clinical trials from PAREXEL Consulting experts.
Taking The Pulse of Medtech innovation_Pitchbook, Dec, 2021Levi Shapiro
Report by Pitchbook (a Morningstar company), December, 2021- Taking the Pulse of Medtech Innovation. Medtech versus biotech: A capital investment comparison of the life sciences subsectors.
The red-hot life sciences industry has seen incredible growth in the last decade as the biomedical revolution of the early 21st century has gained traction. Key discoveries, ranging from the development of recombinant protein production to bioprosthetic implants to genome engineering, have propelled capital investment from VC investors to the tune of$20 billion annually for the past three years, with over $44 billion already deployed to life sciences companies in 2021 alone. Given large-cap multinational companies’ aversion to investing financial and human capital into high-risk early-stage research & development (R&D) projects, innovation within life sciences has fallen upon startups and venture-backed companies.
Introduction to MedDRA Coding in Drug Safety & Pharmacovigilance Process for Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
Contact:
"Katalyst Healthcares & Life Sciences"
South Plainfield, NJ, USA
info@KatalystHLS.com
The first series of TechRasa’s market overview white paper reports starting with Iran AdTech Overview with sponsorship and data partnership with Adro AdExchange, Iran’s first AdExchange opening Iran’s online advertising landscape to the world.
In this report you’ll find out about:
* The evolution of advertising
* Iran AdTech ecosystem
* Adtech’s local and international trends
and Adtech’s challenges and opportunities
Burson-Marsteller Global Corporate Twitter Influence StudyBurson-Marsteller
The Global Corporate Twitter Influence Study analyzes the influence and demographics of people following large global companies on Twitter. The research, produced by Burson-Marsteller, a leading global public relations firm, and StatSocial, a leading social media analytics platform, found that individuals who follow Fortune Global 100 companies on Twitter are more connected and more influential than the average Twitter user.
Henkel - History, Evolution, Present and the FutureGreg Thain
A comprehensive background of Henkel containing its History and Origins, Early Evolution, Modern Business, Global Expansion, Company Structure, Recent Efforts and Company DNA. As one of the chapters of the book FMCG: The Power of Fast-Moving Consumer Goods by authors Greg Thain and John Bradley. For more details on their success story and that of other leading FMCG companies, check www.fmcgbook.com or Amazon http://amzn.to/1jRyd20.
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Merck & co., inc. barclays final 3-10-2015Marcel Brussee
What does Merck's $10B buyback plan say about pharma M&A?
Adding $10 billion to its stock buyback program might mean Merck & Co. ($MRK) wants to keep investors happy as its sales continue to shrink. That would be the typical explanation. But The Wall Street Journal sees a different possibility--one the rest of the industry might want to pay attention to.
Source: http://www.fiercepharma.com/story/what-does-mercks-10b-buyback-plan-say-about-pharma-ma/2015-03-26?utm_campaign=SocialMedia
Can-Fite BioPharma Ltd. (NYSE American: CANF) is an advanced clinical stage drug development company with a platform technology that addresses multi-billion-dollar markets in the treatment of autoimmune inflammatory diseases including Psoriasis, and liver diseases including advanced liver cancer and NASH. Can-Fite’s drugs have an excellent safety profile with experience in over 1,000 patients. Can-Fite’s intellectual property portfolio consists of 13 patent families issued and pending. Piclidenoson and Namodenoson have been out-licensed in select territories with approximately $18 million received to date. Piclidenoson received approval for COVID-19 clinical trial in Israel in April 2020 and is expected to file its IND in the US in the near-term.
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Bellus corporate presentation April 5 2016
1. Corporate Presentation (TSX: BLU)
Roberto Bellini
President and Chief Executive Officer
Twitter: @rbellini
April 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.
2
3. 3
At BELLUS, we are focused on developing drugs for rare
diseases starting with conditions that affect the kidneys.
5. Value driving rare disease pipeline fully funded through key
milestones
Company 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 fully funded through KIACTA Phase III and exit
process
6. 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
7. Lead Phase III Product Candidate
7
A rare and deadly
kidney disease with
no specific treatment
FOR AMYLOID A (AA)
AMYLOIDOSIS
8. 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 8
9. Patient Population
Source: Navigant Consulting 2014
10,000-
15,000
potential KIACTATM
patients in the United
States and Europe
MARKET RESEARCH
Navigant 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
10. 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 drugs 10
Drug U.S. Patients Disease Price
Vyndaqel 1,500
Transthyretin amyloid
polyneuropathy
$200K
Gattex 9,500 Short Bowel Syndrome $295K
Kalydeco
1,350
Cystic Fibrosis (G551D
mutation)
$335K
Procysbi
500 Nephropathic cystinosis $250K
Juxtapid 3,000
Familial
hypercholesterolemia
$250K
Jakafi
1,500 Splenomegaly $87K
COMPARABLES
11. 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
12. 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
13. POTENTIAL ACQUIRERS
Pharma/Big biotech with inflammation and/or nephrology franchise
Orphan disease focused biotech
Exit Strategy
Strong M&A environment for rare disease products
13
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
RECENT RARE DISEASE M&A
14. 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
NumberofWorseEvents
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
15. Clean safety profile without any important differences
between groups in Phase II/III study 15
KIACTA™ – Clean Safety Profile
98%
36%
23%
93%
42%
25%
0%
20%
40%
60%
80%
100%
Adverse Events Serious Adverse Events Discontinuations due to
Adverse Events
%ofPatients
KIACTA
Placebo
16. 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
17. PHASE III CONFIRMATORY STUDY
183 patients in 13 countries
Composite primary endpoint
based on patients reaching
kidney function worsening
events
Target statistical significance 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
significance 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 study
17
18. Study enrolled with 261 patients
Study completed with 120 events
reached (January 2016)
Topline data expected in Q2 2016
Phase III Confirmatory Study
18
19. 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
20. Second Rare Disease Product Candidate
20
A rare disease
primarily affecting
the kidneys of
children
FOR STEC RELATED
HEMOLYTIC UREMIC
SYNDROME (SHUS),
SHIGAMABSHIGAMAB
21. 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
22. 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
22
PRE- CLINICAL
23. Clean capital structure and cash runway through potential exit
Corporate
23
Capital Markets (as of April 1st, 2016)
Ticker TSX: BLU
Shares (Basic) 54.7M
Shares (Fully Diluted) 65.7M
Volume (3 month) ~100K
Market Capitalization (FD) ~C$100M
23
Finance
Cash (December 31, 2015) C$9.7M
Burn rate (monthly) <C$300K
24. 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 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.
24
25. 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
26. Connect With Us
Follow us on Twitter: @BELLUSHealth
Join our LinkedIn group
Read our blog @ www.bellushealth.com
Join our mailing list