Title: CYSTIC FIBROSIS FOUNDATION Venture Philanthropy: Tapping Foundations Diana R. Wetmore, Ph.D. Vice Pr
1CYSTIC FIBROSIS FOUNDATIONVenture
PhilanthropyTapping FoundationsDiana R.
Wetmore, Ph.D.Vice President of Alliance
Mgmtdwetmore_at_cff.orgwww.cff.org
adding tomorrows every day.
2Drug 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.
3CF 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.
4CFF Strategy Progress Realized
- 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
5CFF Strategy Progress Realized
- 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
6Median Survival Age of PatientsWith CF 1940 -
2004
35
32
29
18
16
Median Survival Age
10
1
Year
7CFFT Alliances for Drug Discovery Drug
Development
- CFFT helps reduce the risk for our partners in CF
indications
Risk Uncertainty x Cost x Timing
Understanding Path to Proof-of-Concept
Validation of Underlying Science
Funding Vehicles
8Therapeutics Development Program
Therapeutics Development Awards
- Basic Research
- Corrector Consortium
- High-throughput
- Screening
- Vertex
- Structural Genomics
- SGX
- Predix
- Functional Genomics
- Proteomics
- siRNA Technology
- Alnylam
- Galapagos
Development
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10What 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.
11Therapeutics Development Network
Harvard University - Boston Childrens
Case Western Reserve Univ.
University of North Carolina
WashingtonUniversity(St. Louis)
University of Alabama
Ohio State University
Cytology Interpretation
NPD IPFT Interpretation
Harvard University - Mass. General
University of Utah
Cystic Fibrosis Foundation
University of Washington
Coordinating Center Seattle, WA
Baylor University
Microbiology
External Advisory Committee
University of Pittsburgh
Stanford University
University of California,San Diego
University of Colorado
University of Cincinnati
University of Minnesota
Johns Hopkins University
University of Iowa
Inflammatory Mediators
Imaging (HRCT) Interpretation
12Therapeutics Development Awards
A peer-reviewed, milestone-driven mechanism to
enable the evaluation of promising therapies for
CF patients
- 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
13General Elements ofAlliance 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.
14General Elements ofAlliance 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 CFFs
investment is returned.
15Other 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
16JDRFwww.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_at_jdrf.org
17Other 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
18adding tomorrows every day.