# Presentation: 849254

## CYSTIC FIBROSIS FOUNDATIONVenture Philanthropy:Tapping FoundationsDiana R. Wetmore, Ph.D.Vice President of Alliance Mgmtdwetmore@cff.orgwww.cff.org

***Feb 27, 2007***

***...******adding tomorrows every day.***

## Drug Development for CF

- Solution:** ****Therapeutics Development Program, initiated in 1997 to provide financial and resource support to pharmaceutical partners to encourage development of new drugs 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?**

## CF Drug Development

**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.**

- ASSUMPTIONS:

## 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

## 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

- 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

**CFF Strategy: Progress Realized **

## Median Survival Age of PatientsWith CF: 1940 - 2004

**Median Survival Age**

**Year**

**<1**

**1**

**10**

**16**

**18**

**29**

**32**

**35**

## CFFT Alliances for Drug Discovery & Drug Development

**CFFT Alliances for Drug Discovery & Drug Development**

- CFFT helps reduce the risk for our partners in CF indications

- Validation of
- Underlying
- Science

- Funding
- Vehicles

- Understanding
- Path to
- Proof-of-Concept

**Risk = Uncertainty x Cost x Timing**

## Therapeutics Development Program

**Development**

**Therapeutics**
**Development**
**Awards**

- Basic Research
- Corrector Consortium
- High-throughput
-     Screening
- Vertex
- Structural Genomics
- SGX
- Predix
- Functional Genomics
- Proteomics
- siRNA Technology
- Alnylam
- Galapagos

## Slide 9

## What is the CF Therapeutics Development Network (TDN)? 

- 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.

**A non-profit clinical trials network established in 1998**

## Therapeutics Development Network

**University of Colorado**

**Inflammatory Mediators **

**University of**
**North Carolina **

**NPD & IPFT**
**Interpretation**

**University**
**of Washington**

**Microbiology**

**Imaging (HRCT)**
**Interpretation **

**University of **
**Cincinnati**

**Stanford**
**University**

**Case Western**
**Reserve Univ.**

**Cytology Interpretation **

**Coordinating Center**
**Seattle, WA **

**University of**
**Minnesota **

**University of **
**Alabama**

**University of**
**Iowa**

**Johns Hopkins**
**University**

**University of**
**Pittsburgh**

**Baylor**
**University**

**Ohio State**
**University**

**Washington****University****(St. Louis)**

**University of California,****San Diego**

**University of Utah**

**Harvard **
**University -**
**Mass. General**

**Cystic Fibrosis Foundation**

**External Advisory Committee**

**Harvard **
**University -**
**Boston**
**Children’s**

## 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**

## 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.

- 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.

- 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.

**General Elements of****Alliance Agreements**

## 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](http://www.multiplemyeloma.org/)_
- www.themmrc.org

## ...adding tomorrows every day.

***...******adding tomorrows every day.***