EuroBioForum 2013 2nd Annual Conference 27-28 May 2013 - Hilton Munich City, Munich, Germany http://www.eurobioforum.eu/2013 ======================================= # REGIONAL PERSPECTIVES # Ontario Genomics Institute, Canada: Innovative Research, Innovative Translation Dr Mark Poznansky President and CEO Ontario Genomics Institute ======================================= http://www.eurobioforum.eu

1. Ontario’s Perspective on
Personalized Medicine:
Innovative Research, Innovative Translation
Dr. Mark Poznansky
Ontario Genomics Institute

2. 2
Ontario Genomics Institute
• Driver and catalyst for the life sciences industry in
Ontario
• Integrated approach, science and business expertise,
experience brokering relationships and supporting
research through to commercialization
• Business development – 18 companies; attracted
$42 million in venture capital
• Research - 65 projects; total funding of $800 million; including economic,
ethical, legal, environmental, social aspects
• Not-for-profit corporation supported by the federal
and provincial governments

3. Ontario, Canada
3
13.5M population
$71.8B in health expenditures
158 hospitals
25 research/academic hospitals,
employing 10,000 researchers
44 universities/colleges
Among the largest
single-payer HMOs
$1.9B/yr on health-
related R&D
Toronto has
Canada’s largest concentration
of scientific research and is in the
top three bioscience clusters in
North America

4. Life and Health R&D
Ontario
Health
Research
1.9B/yr
Health Charities
• Heart and Stroke
Foundation
• Canadian Cancer
Society
Federal
• Canadian Institutes of Health Research
• Canadian Foundation for Innovation
• National Research Council
• Natural Sciences and Engineering
Research Council
• Genome Canada
• Tax credits
Provincial
• Ontario Research Fund
• Ontario Centres of Excellence
• MaRS
• Ontario Inst. for Cancer Research
• Ontario Brain Institute
• Provincial match of federal grants
• Tax credits
Private Funding
• Industrial
• Venture financing
• Institutional
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5. Ontario Funding:
Human Genomics Research
~$190M/year in federal operating funding:
• Genome Canada – $915M over lifetime of GC plus over $900M in
co-funding from other organizations
• Genomics and personalized health competition - 4 projects
• CIHR, others
~$130+M/year in provincial operating funding:
• Ontario Research Fund – Research Excellence: ~$165M to genomics
since 2007
• Ontario Institute for Cancer Research: $82M/yr
• Ontario Brain Institute: $20M/yr
• Excludes matching funding
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6. Unique Initiatives in
Personalized Medicine
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7. Rheumatoid Arthritis Pilot Study
Canada (2010): $10.2B in direct health care costs
$17.3B in indirect (wage-based
productivity) costs
Total economic burden $27.5B
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RA = rheumatoid arthritis
PS = psoriasis
IBD = inflammatory bowel disease
SLE = systemic lupus erythematosus

8. Rheumatoid Arthritis Pilot Study
• Dr. Katherine Siminovitch – Mt.
Sinai,Toronto
1.Discover genes via targeted and
whole exome sequencing that:
• Confer risk for rheumatoid arthritis
• Modulate outcome/drug response
2.Develop informatics solution to:
• Collect, store & mine clinical data
• Integrate clinical & genomics data
• Apply genomics data in the clinic to improve
healthcare delivery
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Control
s
Cases
100,000 - 1 million
markers
Discovery of risk variants

9. • Dr. James Kennedy – CAMH,Toronto
• Assessment of rapid PGx in primary
care setting (depression, schizophrenia)
• 48 hour turnaround
• Red/Yellow/Green advisory based on
panel of SNP markers (liver enzymes,
drug target)
• Expand to >250 primary care physicians;
20,000 patients
• New biomarker discovery
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IMPACT Study
Individualized Medicine: Pharmacogenetic
Assessment and Clinical Treatment

10. Enhanced CARE for RARE
• Building on success of Finding of Rare Disease Genes in Canada
(FORGE Canada) project
• Identified over 100 genetic mutations underlying rare disorders in children
• Reduce the “diagnostic odyssey”
• International consortium
• Drs. Boycott and MacKenzie – CHEO, Ottawa
• Disorders are individually rare but affect ~3% of population
• Enhanced CARE for RARE Genetic Diseases in Canada:
• Expanded search for causative mutations in rare disorders
• Identify therapeutics from pool of marketed drugs
• Testing ground for evaluating genome-wide analysis
as a diagnostic tool, and of the identification of
therapeutics, in small patient groups
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11. Autism Spectrum Disorders:
Genomes to Outcomes
• Drs. Stephen Scherer, Peter Szatmari – SickKids,Toronto
• ASD affects one in 88 children, costs healthcare system $1B/yr
• Early identification and intervention improves outcomes
• Diagnosis difficult and slow
• Objective:
• WGS to identify remaining diagnostic genetic risk factors
• Clinical guidelines
• Early, objective diagnosis
• Component of international Autism Sequencing
Consortium
• 10,000 genomes of ASD patients and their families
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12. Top Tier Health
Bioinformatics Research
•Ontario is home to:
•MedSavant – search for genetic variants
•GeneMania – functional prediction, association
data based on sequence
•Cytoscape – visualization of complex networks
•Infrastructure support
•Canadahealth Infoway
•eHealth
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13. Facing the Facts
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1. Canada is a research powerhouse
“ Canada‘s science system is a success story”
Richard Hawkins (University of Calgary) Lab Business Sept/Oct 2012
“With less than 0.5 per cent of the world’s population, Canada
produces 4.1 per cent of the world’s scientific papers and nearly
5 per cent of the world’s most frequently cited papers.”
Council of Canadian Academies “TheState of Science andTechnology in Canada, 2012”
2. Canada receives a failing grade when it comes to
innovation
“Despite a decade or so of innovation agendas and prosperity
reports, Canada remains near the bottom of its peer group on
innovation, ranking 14th among the 17 peer countries.”
Conference Board of Canada “How Canada Performs: Innovation”

14. Personalized Medicine UnfoldsLevelofexcitement
Initial idea
Enthusiasm
Hype
Reality check
Disillusionment
True
performance
Time
Hypothesis testingDescriptive phaseSetting up

15. Challenges of Implementing
Personalized Medicine
• Integrated infrastructure to support the type of data
that will be generated and need to be shared
• Resources to provide clinical validation
• Privacy policy to address public concerns over data
storage and release
• Education about this emerging field (both for the
public and health professionals)
• Regulatory and reimbursement policies that can
accommodate these new technologies
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16. • Large-scale innovation centre focused
on building Canada’s next generation of
growth companies
• Provides office/lab space and resources
including education, market intelligence
and business mentoring
• Industries: health care, IT, cleantech,
social innovation
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MaRS Discovery District

17. •Commercialization agent for IP created by
16 members: institutions, research
hospitals, universities
•Tapping in to $1B annual R&D
•20+ start-up companies; 20+ licensable
technologies; over 100 projects in
developmental pipeline
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MaRS Innovation

18. MaRS EXCITE
• MaRS Excellence in Clinical Innovation andTechnology
Evaluation (EXCITE) helps companies accelerate adoption and
reimbursement of disruptive health technologies
• Single, harmonized, pre-market, evidence-based process to
expedite market penetration and mitigate the risk of rejection
18

19. • 20 year repository of anonymous linked
population-based health information on an
individual patient basis
• Unique ICES identifiers that ensure the privacy
and confidentiality of health information
• Unique linkage between research data and
clinical data
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Institute for Clinical Evaluative
Services (ICES)

20. Ontario Institute for Cancer
Research
• Independent not-for-profit translational cancer research
institute
• $150M /yr funding including $82M/yr from the Government of
Ontario
• Supports each step in the development of important new cancer
products, from basic research through clinical studies and
regulatory approvals
• Research priorities include facilitating the adoption of
personalized medicine for cancer
• ICGC Member; OntarioTumour Bank; Informatics
and Biocomputing; High Impact ClinicalTrials;
Clinical Genomics AssessmentTrial (CGAT)
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21. Ontario Brain Institute
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Neuroimaging
Clinical
Genomics
Proteomics
Other
Cerebral Palsy
Neurodevelopmental Disorders
Neurodegeneration
Depression
Other Brain Disorders
Data Modalities
Epilepsy
• New institute founded 2010
• Two imperatives:
• Integrated Discovery: science +
commercialization + real-world
outcomes
• Integrated Data via Brain-CODE:
open-access informatics platform
• Security
• Standardization
• Federation
• Analytics

22. • Streamline multicentre trials
• Single ethical review process
• Harmonized administration and platforms
• Attract clinical trial investment
• Demonstrate our strengths/advantages
• Increase awareness at a global level
• Engage public; improve patient recruitment
• Increase pubic awareness of clinical trials
• Establish strategies to support patient
recruitment and retention
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Clinical Trials Ontario

23. What Can We Offer?
A wealth of patients, investigators and
platforms geared towards the objectives
of personalized medicine
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24. What Are We Looking For?
1. To understand where we sit on the
“path” and push those boundaries
2. Outstanding further “fundamental/
discovery” science
3. To develop close partnerships to help
further the agenda
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25. Challenges
• Timing: are we ready; do we have the data?
• Will personalized genome sequencing become
a driver of health care?
• It is a disruptive technology – we have no
models for implementation
• Genomic data is (mostly) static but our
understanding of its meaning is changing rapidly
• Reimbursement
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26. Top Three Recommendations
for Achieving Tangible Results
1. Integrate data
2.Collect data
3. Share data
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27. In Summary
1. Ontario has a huge, well-funded health research
infrastructure
2. Personalized medicine is a key part of the health
sciences agenda and Ontario's infrastructure –
Ontario’s health care system is well suited for PM
uptake.The potential for partnering is tremendous
3. Ontario faces considerable challenges bringing
discoveries to market, especially at the level of
commercialization.The investment opportunity is
huge
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28. 28
Ontario Successes
• Analytical equipment and reagents development company –
developed mass cytometer for single cell analysis
• Raised $14.6 million in follow on financing in 2011
• Operates facility in Markham, ON and has 45 employees
• Molecular diagnostic company: colorectal cancer and age-
related macular degeneration
• Revenues of $5.8 million in 2011 and $8 million in the first
quarter of 2012.
• Secured $2.3 million in funding in the past year

29. 29
Ontario Successes
• Early-stage molecular diagnostics company - developing
assays to help manage cancer chemotherapy
• Received OGI investment to further develop and validate lead
product, the RNA Disruption Assay™ (RDA™), which enables a
personalized approach to chemotherapy management
• Raised additional $1.6 million in financing to further develop
their assay