Ontario’s Perspective on Personalized Medicine: Innovative Research, Innovative Translation

Dr. Mark Poznansky Ontario Genomics Institute

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

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

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

4

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

5

Unique Initiatives in Personalized Medicine

6

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

7

RA = rheumatoid arthritis PS = psoriasis IBD = inflammatory bowel disease SLE = systemic lupus erythematosus

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

8

Control

s

Cases

100,000 - 1 million

markers

Discovery of risk variants

• 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

9

IMPACT Study Individualized Medicine: Pharmacogenetic

Assessment and Clinical Treatment

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

10

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

11

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

12

Facing the Facts

13

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 “The State of Science and Technology 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”

Personalized Medicine Unfolds L

evel

of

exci

tem

ent

Initial idea

Enthusiasm

Hype

Reality check

Disillusionment

True performance

Time

Hypothesis testing Descriptive phase Setting up

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

15

• 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

16

MaRS Discovery District

•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

17

MaRS Innovation

MaRS EXCITE

• MaRS Excellence in Clinical Innovation and Technology 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

• 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

19

Institute for Clinical Evaluative Services (ICES)

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; Ontario Tumour Bank; Informatics and Biocomputing; High Impact Clinical Trials; Clinical Genomics Assessment Trial (CGAT)

20

Ontario Brain Institute

21

Neu

roim

ag

ing

Clin

ical

Gen

om

ics

Pro

teo

mic

s

Oth

er

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

• 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

22

Clinical Trials Ontario

What Can We Offer?

A wealth of patients, investigators and platforms geared towards the objectives

of personalized medicine

23

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

24

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

25

Top Three Recommendations for Achieving Tangible Results

1. Integrate data

2. Collect data

3. Share data

26

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

27

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

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