CYSTIC FIBROSIS FOUNDATION Venture Philanthropy: Tapping Foundations Diana R. Wetmore, Ph.D. Vice Pr - PowerPoint PPT Presentation

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CYSTIC FIBROSIS FOUNDATION Venture Philanthropy: Tapping Foundations Diana R. Wetmore, Ph.D. Vice Pr

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Title: CYSTIC FIBROSIS FOUNDATION Venture Philanthropy: Tapping Foundations Diana R. Wetmore, Ph.D. Vice Pr


1
CYSTIC FIBROSIS FOUNDATIONVenture
PhilanthropyTapping FoundationsDiana R.
Wetmore, Ph.D.Vice President of Alliance
Mgmtdwetmore_at_cff.orgwww.cff.org
adding tomorrows every day.
  • Feb 27, 2007

2
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.

3
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.

4
CFF 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

5
CFF 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

6
Median Survival Age of PatientsWith CF 1940 -
2004
35
32
29
18
16
Median Survival Age
10
1
Year
7
CFFT 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
8
Therapeutics 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
9
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10
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.

11
Therapeutics 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
12
Therapeutics 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

13
General 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.

14
General 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.

15
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

16
JDRFwww.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

17
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

18
adding tomorrows every day.
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