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Foundation and Vertex Announce Positive Early Results for VX-770

March 27, 2008

The Cystic Fibrosis Foundation and Vertex Pharmaceuticals 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.”

Additional Reading

News releases from the CF Foundation and Vertex Pharmaceuticals

FAQs About VX-770 Phase 2 Study Results

BusinessWeek online article "A Healthy Gamble for Cystic Fibrosis"

 

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