CYSTIC FIBROSIS FOUNDATION

CYSTIC FIBROSIS FOUNDATION Venture Philanthropy: Tapping Foundations Diana R. Wetmore, Ph.D. Vice President of Alliance Mgmt dwetmore@cff.org www.cff.org …adding tomorrows every day. Feb 27, 2007 Drug Development for CF Statement of Problem: How do you convince the biopharmaceutical industry to develop drugs for a disease with a population of less than 30,000 patients in the United States, and 70,000 worldwide? Solution: Therapeutics Development Program, initiated in 1997 to provide financial and resource support to pharmaceutical partners to encourage development of new drugs for CF. CF Drug Development ASSUMPTIONS:     Must have knowledge about the basic defect and underlying pathophysiology of disease (CF). Must work to minimize the risk for partner to enter the field of CF. Must establish a business relationship with partner (not necessarily a charitable one). Must have ready access to clinical population and informational systems to support clinical development. CFF Strategy: Progress Realized 1. Solve the Science    1962 – First research grant ($12,000) 1980 – RDP established 1980s – Scientific breakthroughs begin   1985 – Chloride channel defect discovered 1989 – CF gene discovered   1990s – Improved understanding of how CFTR leads to disease 2000s – Biotechnological advances accelerate science CFF Strategy: Progress Realized 2. Translate Science into New Therapies  Mid 1990s - Therapeutics era begins 1994 – Pulmozyme  1997 – Tobi  2002 – Azithromycin  2005 – Hypertonic saline    1998 – Therapeutics Development Program established 2007 – Rapidly increasing opportunities exist that now address both the complications and the basic defect Median Survival Age of Patients With CF: 1940 - 2004 35 35 30 25 20 15 10 5 0 1940 1950 1960 1970 1980 1990 2000 2004 32 29 16 10 1 18 <1 Year CFFT Alliances for Drug Discovery & Drug Development • CFFT helps reduce the risk for our partners in CF indications Risk = Uncertainty x Cost x Timing Validation of Underlying Science Funding Vehicles Understanding Path to Proof-of-Concept Therapeutics Development Program Therapeutics Development Awards DISCOVERY Basic Research • Corrector Consortium High-throughput Screening • Vertex Structural Genomics • SGX • Predix Functional Genomics Proteomics siRNA Technology • Alnylam • Galapagos Our Biotech Partners Altus Corus Pharma Hollis-Eden Lantibio Parion Transave Chiron Genaera Inologic Mpex PTC Yahoo Copernicus Genzyme Inspire Nitrox SEER Development IND Preclinical Safety Testing TDN FDA NDA P P Clinical R Dosage O and V Efficacy A L Distribution Available to CF Patients Bring Existing Drugs for CF Indication What is the CF Therapeutics Development Network (TDN)? A non-profit clinical trials network established in 1998 Purpose: To facilitate development and conduct of early phase clinical trials involving new therapeutic agents for cystic fibrosis and identify appropriate outcomes for future studies. Funding: Primarily grant support from the CF Foundation, with additional support from the National Center for Research Resources and private industry sponsors. Therapeutics Development Network Harvard University Boston Children’s University of Alabama University of North Carolina NPD & IPFT Interpretation Case Western Reserve Univ. Cytology Interpretation Washington University (St. Louis) Ohio State University Harvard University Mass. General University of Washington Microbiology Cystic Fibrosis Foundation University of Utah Coordinating Center Seattle, WA Baylor University CF Data Safety Monitoring Board Stanford University External Advisory Committee University of Pittsburgh University of Iowa University of Colorado Inflammatory Mediators University of Cincinnati Imaging (HRCT) Interpretation University of Minnesota University of California, San Diego Johns Hopkins University Therapeutics Development Awards A peer-reviewed, milestone-driven mechanism to enable the evaluation of promising therapies for CF patients Component I $100,000/yr x 2 Pre-clinical    Component II $750,000/yr x 2 Initial Clinical Trials Oversight by advisory group (progress reports) CF Foundation investment repaid after FDA approval CF Foundation receives percentage of royalty from net sales or multiple of principal investment General Elements of Alliance Agreements    The CFF agrees to fund, on a matching basis, the development of products/compounds for the purpose of identifying new drugs for CF. Negotiated portions of the awards from the CFF are dependent on the accomplishment of predetermined, success-driven milestones. The CFF may withdraw from the project in the event of failure to achieve milestones. The CFF may also withdraw at various intervals at its discretion notwithstanding results of milestones. The CFF requires the establishment of a Scientific Advisory Council made up of two CFF representatives, two sponsor representatives and one jointly appointed to monitor progress of development and to resolve scientific disputes. General Elements of Alliance Agreements   Once a drug is approved, the CFF receives a multiple of its investment (or a royalty agreement based on net sales). The CFF also requires additional compensation for extraordinary sales results. If there is a suspension in development activities, the CFF obtains worldwide rights to develop the product with agreement to negotiate royalties to original partner after the CFF’s investment is returned. Other Foundations • CFFT has shared its TDP model with many other disease-based organizations: – MMRC – JDRF – BIO Ventures for Global Health – Michael J. Fox Foundation – Myelin Repair Foundation – National MS Society – others JDRF www.jdrf.org/industry_partnerships • Industry Discovery & Development Partnerships • 14 partnerships with Biotech & Pharma • >$20M committed • Up to $5M per program • Milestone based • Matching funds • pburn@jdrf.org Other Foundations with Industry Partnership Programs • Michael J. Fox Foundation – Sangamo • Families of Spinal Muscular Atrophy – Paratek Pharmaceuticals, Inc. • Myelin Repair Foundation • MMRF – www.multiplemyeloma.org – www.themmrc.org …adding tomorrows every day.