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The CFF Story: Innovating for a Cure
1. The CFF Story:
Innovating for a Cure
Huntington Study Group
November 4, 2016
Robert J. Beall, Ph.D.
Former President and CEO, Cystic Fibrosis Foundation
2. The CFF Story:
Innovating for a Cure
Community
Clinical Care
Research
Discovery & Development
Treatment
3. Median Predicted Survival Age of US Patients
with Cystic Fibrosis
Source: Cystic Fibrosis Foundation, National Patient Registry
41
0
5
10
15
20
25
30
35
40
45
1940 1950 1960 1970 1980 1990 2000 2010 2015
4. As of 2015 There Are More Adults With CF
Than Children With CF in the U.S.
Cystic Fibrosis Foundation. Patient Registry Annual Report. Bethesda, MD: Cystic Fibrosis Foundation.
2015
%ofCFindividualsovertheageof18
1965 1970 1975 1980 1985 1990 1995 2000 2005 2010 2015
0
10%
20%
30%
40%
50%
8. Academics
• Research
• Care
• Training
Biopharmaceutical
• Translation
• Commercialization
CF Foundation
• Care
• Research
• Education
Ecosystem for Cystic Fibrosis Foundation Success
FDA
NIH
23. Therapeutics Development Program (TDP)
Started in 1998
• Created to encourage industry and academia to
focus on CF and CFTR as drug target
• Components of TDP
– Financial assistance
– Research tools and scientific advice
– Well organized clinical trial network
Venture Philanthropy
24. TDP Process
Available
to
CF Patients
DISTRIBUTION
FDA
P
P
R
O
V
A
L
DEVELOPMENT
TDN
Bring Existing Drugs for
CF Indication
NDA
Clinical
Dosage
and
Efficacy
IND
Preclinical
Safety
Testing
Therapeutics
Development
Awards
DISCOVERY
Basic Research
• CFTR Corrector
Consortium
• CFTR Structural
Consortium
• Mucociliary
Clearance
Consortium
High-throughput
Screening
The CF Foundation has “really paved the way for other
small disease nonprofits to take drug discovery into their
own hands.”
-Harvard Business School Professor Robert Higgins
26. Highlights of CF TDN Studies (1998-2016)
• Network supports 25-35 studies annually:
– 75% industry-based therapeutic trials
– 25% PI-initiated
• Initial studies were early Phase (1 and 2)
• Transition to larger Phase 3 trials
– First CFFT Phase 3 study in 2001
– Industry moved to predominantly Phase 3 by
2009
• PI initiated studies have focused on outcome
measure development
• In total, over 150 studies involving thousands of
individuals with CF have been successfully
completed.
32. Venture Philanthropy: It is not just the
money! It also involves access to:
• Expertise
• Patients
• Clinical Trial Network
• Data
• Specialized Resources
• Committed Community
Venture Philanthropy is not for the faint of heart…
The model-adjusted absolute mean difference between ivacaftor and placebo was 10.68 percentage points (95% CI 7.26, 14.10).
Significant ivacaftor treatment effects were also observed at Weeks 2 and 4 (p<0.0001 at both time points) when compared to placebo.
Table 14.2.1.2.1
Table 14.2.1.2.2
Table 14.2.1.2.2
Just a list of some of the partners to take us to the next level—some small molecules approaches--- but generally limited to one day approaches,,, but Shire and ProQR are moving toward newer approaches,,, multiple day novel therapies