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January 2013

Expanded Collaboration with Pfizer Targets Most Common CF Mutation

Expanded Research Collaboration with Pfizer Will Target Most Common CF Mutation

The Cystic Fibrosis Foundation recently announced a major expansion of its collaboration with Pfizer Inc., aimed at discovering new drugs to treat people with the most common mutation of cystic fibrosis, Delta F508.

In the new six-year program with Pfizer, Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), the CF Foundation’s nonprofit drug discovery and development affiliate, will invest up to $58 million to speed the discovery of therapies that target the underlying cause of CF.

The collaboration will focus on identifying compounds that help restore normal function of the CFTR protein, which does not fold correctly in people with the Delta F508 mutation. The program’s goal is to move one or more potential CF drugs into clinical trials by the end of the multiyear collaboration.

Nearly 90 percent of people with CF have at least one copy of the Delta F508 mutation.

“The Foundation’s expanded collaboration with Pfizer is an exciting step forward in the fight against CF,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “Pfizer brings impressive scientific expertise and technical capacity to our ongoing efforts to accelerate the development of more therapies that treat the root cause of CF in all people with the disease.”

One of the largest pharmaceutical companies in the world, Pfizer has a massive chemical library of about 5 million chemical compounds that it will draw on to screen for potential CF therapies.

Pfizer researchers also will draw on their expertise in developing therapies that help mutated proteins fold and route correctly within the cell. The Foundation will provide scientific as well as financial support to help advance the discovery program.

Through its innovative venture philanthropy business model, the Foundation invests in CF research and drug development with leading biotech and pharmaceutical companies around the world.

The first drug to treat the underlying cause of CF, Kalydeco™, was developed by Vertex Pharmaceuticals Inc. with significant support from the Foundation. In January 2012, the U.S. Food and Drug Administration approved Kalydeco for people with the G551D mutation of CF ages 6 and older.

In addition to its expanded research program with Pfizer, the Foundation continues its collaboration with Vertex to discover and develop potential new therapies to treat people with the Delta F508 mutation.

Vertex plans to begin a Phase 3 clinical trial in early 2013 of Kalydeco in combination with another potential drug, VX-809, in people with two copies of the Delta F508 mutation. 

The Foundation has also entered into new collaborations with other companies, including Genzyme, a Sanofi company, and Proteostasis Therapeutics Inc., to discover more compounds that could potentially target the underlying cause of the disease in people with the most common mutation of CF.

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The Cystic Fibrosis Foundation is an accredited charity of the Better Business Bureau.