Crohn s and Colitis Foundation of Canada / Vertex Pharmaceuticals. Sponsored Research Program in Inflammatory Bowel Disease

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1 Crohn s and Colitis Foundation of Canada / Vertex Pharmaceuticals Sponsored Research Program in Inflammatory Bowel Disease

2 Presentation Overview Background on inflammatory bowel disease Partner Overview CCFC/Vertex Sponsored Research Program Overview Challenges in IBD research Objectives Strategy Accomplishments Benefits to partners Future milestones Budget/financing Summary/key points

3 Inflammatory Bowel Disease (IBD) Group of disorders that causes the bowel to become severely inflamed and ulcerated Two main forms of IBD are Crohn s disease and ulcerative colitis Symptoms include diarrhea, rectal bleeding, abdominal pain, weight loss, anemia, sores in mouth & anus

4 Inflammatory Bowel Disease (IBD)

5 Inflammatory Bowel Disease (IBD)

6 Inflammatory Bowel Disease (IBD) IBD is a chronic, life-long disease Currently, there is no cure Therapy is aimed at achieving and maintaining remission from symptoms Most people require ongoing medication and when this fails, surgery is often required Associated with increased risk of colon cancer

7 Inflammatory Bowel Disease (IBD)

8 Partnership Overview Crohn s & Colitis Foundation of Canada Non-for-profit health research organization Mission is to find cures and improve the lives of children and adults living with IBD One of world s leading funders of IBD research Invested more than $76 million in research since it inception Supports over 70 research projects & training awards annually

9 Partnership Overview Vertex Phamaceuticals (Canada) Inc. Working on discovering, developing and commercializing innovative therapies to treat people with serious diseases (infection, oncology, immune & neurological diseases) Research site established in Laval, Quebec focusing on IBD genetics, immune therapy and human microbiome

10 Initiative Overview Collaboration between CCFC and Vertex Pharmaceuticals (Canada) Inc. Unique business model between non-profit organization and biotech Capitalized on each partners assets and strengths Request for Application (RFA) developed that ensured alignment of research priorities for both organizations

11 Challenges in IBD research Causes and underlying mechanisms of disease remain unknown Current treatment options are limited and ineffective in a significant proportion of IBD patients

12 Objectives Increase our understanding of the mechanisms associated with IBD Build IBD research capacity in Canada Accelerate the discovery, development and commercialization of new IBD treatments

13 Strategy Fund early-stage basic research projects that would meet program objectives Funding decisions based on scientific merit and translatability of applications Leverage dollars for research from industry and donors

14 Accomplishments Through CCFC s IBD research network promoted RFA (16 applications in Year 1, 17 in year 2) Expert review panel evaluated scientific merit and translatability First round of projects funded (examining the role of bacteria in IBD and body s response to microbiome imbalance) Inter-institutional research agreements executed between CCFC, Vertex & university Joint Research Committees (CCFC, Vertex and Principal Investigator) established to track progress to date, advise on research plan and report outcomes

15 Benefits to Partners CCFC/CCFC-affiliated researchers Increased experience in outcome-focused research Access to Vertex s emerging technologies Leverage investment in IBD research Vertex Extended reach to IBD scientific community Access to scientific and translational expertise Execution of grant competition Leverage investment in IBD research

16 Future Milestones Second round of applications in progress Translatability criteria to be incorporated into review of Grants-in-Aid of research grants Joint Research Committee model expanded to other CCFC research programs to ensure outcomes-focused progress and assess impact

17 Budget/Financing Research program involves a 3-yr co-funding partnership for up 6 grants (3 grants per year for two years) Seed investment of $200,000 per project for up to six projects co-funded by CCFC and Vertex

18 Summary/Key Points Positive (win-win) collaborations exist between charitable and industry sectors, even in early-stage basic research Leveraging funding is critical to support research investments Best practices can be shared between sectors to improve outcomes and identify promising projects