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Foundation Announces Positive Results for VX-770 – An Investigational Drug that Could Treat Basic Defect

October 20, 2008

The CF Foundation announced today that VX-770, an investigational oral drug aimed at treating the basic defect in cystic fibrosis, showed positive results in a 28-day, Phase 2 clinical trial. These data bolster the successful interim results reported from the first part of the trial in March of this year. Both studies were done with patients who carry the G551D mutation of CF.

“Data from the Phase 2 trial of VX-770 provide evidence that a small molecule can address the basic defect in cystic fibrosis, and suggest that modulation of CFTR  may play an important role in CF therapy,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation.

VX-770 is being developed and tested by Vertex Pharmaceuticals Incorporated, with support from the CF Foundation. The potential drug aims to open the chloride channels that do not function correctly in people with CF.

Going Forward

Based on the positive results from this trial, Vertex plans to work with global regulatory authorities, including the U.S. Food and Drug Administration, to move VX-770 forward in the approval process. The company is targeting the first half of 2009 to initiate a pivotal trial that could eventually lead to drug approval.

The Foundation will work with Vertex to identify which CF patients this drug may work for going forward. Read more about about participating in CF clinical trials.

Detailed data from the VX-770 clinical trial will be presented this week at the 2008 North American Cystic Fibrosis Conference. Read the full Vertex press release here.



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