ScienceDaily (Mar. 27, 2008) — The Cystic Fibrosis Foundation announced today that VX-770, an oral drug in development that targets a basic defect in CF, showed promising results in an ongoing Phase 2a clinical trial for patients who carry the G551D mutation of CF. The drug is being developed by Vertex Pharmaceuticals Incorporated.

Patients who took the drug for 14 days showed significant improvements in several key indicators of cystic fibrosis, including lung function, nasal potential difference measurements and sweat chloride levels. The findings suggest that VX-770 improves function of what is known as the faulty CFTR protein. This early data is promising and could have important implications for studies of other drugs in development.

This is the first time that any potential therapy has improved the abnormal sweat chloride (salt) levels in a person with CF. Excessive sweat chloride is a key clinical indicator of cystic fibrosis. The "sweat test" is the traditional diagnostic test for CF.

"These early results are an extraordinary endorsement of our hypothesis--that small molecules can correct the basic defect and affect the clinical indicators of cystic fibrosis," said Robert J. Beall, Ph.D., president and CEO of the Foundation. "The emerging data for VX-770 represents the most exciting results we've seen from a Phase 2 trial and increase our confidence that we're on the right track."

The compound VX-770 resulted from a collaboration between the Foundation and Vertex. In 1998, the Foundation approached Aurora Biosciences (acquired by Vertex in 2001) and made an initial investment to use cutting-edge technology--known as high throughput screening--to find compounds to attack the core defect in CF. Since that time, the Foundation has invested $79 million in the project, and the two 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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The above story is reprinted from materials provided by Cystic Fibrosis Foundation, via EurekAlert!, a service of AAAS.

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