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Cystic Fibrosis Foundation Announces Positive Early Results for Phase 2 Clinical Trial of VX-770 - an Oral Compound to Treat CF
Date:3/27/2008

wo organizations have closely collaborated to advance VX-770 and a second CF compound, known as VX-809, through research and into development.

"At a time when the model of venture philanthropy was wholly untested, the Cystic Fibrosis Foundation was willing to take a chance that Vertex's CF program would yield meaningful results for people with cystic fibrosis," said Joshua Boger, Ph.D., president and CEO of Vertex Pharmaceuticals. "The project carried significant risk that would have dissuaded many companies and investors from moving forward. Today, however, with the Foundation's financial, scientific and clinical network support, we have been able to bring two novel CF compounds into development and are encouraged by these early results for VX-770."

Overall, the Foundation has invested nearly $300 million in CF drug research with dozens of biotech collaborators nationwide. These collaborations with for-profit companies are part of the Foundation's unique venture philanthropy business model. The model, which is being emulated by other orphan disease organizations, has spawned a drug pipeline of more than 30 therapies in development.

Part One of the Phase 2a trial of VX-770 studied 20 cystic fibrosis patients over a 14-day period. Part Two of the study is expected to begin in the second quarter of 2008. Additional studies will evaluate the longer-term safety and efficacy of the compound.

Cystic fibrosis is a life-threatening genetic disease that affects about 30,000 people in the United States and 70,000 worldwide. CF is caused by a genetic mutation that results in a malfunctioning (or missing) protein that results in an imbalance of salt and water. This imbalance causes a cascade of mucus plugging, infection and inflammation in the lungs and other organs. The primary cause of death in CF is lung disease.

The predicted median age of survival for people with CF is age 37 - more than double what it was 25 years ago.

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SOURCE Cystic Fibrosis Foundation
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