Phase 2a clinical trial results of VX-809 in patients with CF announced

Feb 4 2010

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced results from a preliminary analysis of data from a 28-day Phase 2a clinical trial of VX-809 in patients with cystic fibrosis (CF) who are homozygous for the F508del mutation. VX-809, an oral investigational Cystic Fibrosis Transmembrane Conductance Regulator protein (CFTR) corrector, was well-tolerated across all four dose groups studied. In the trial, VX-809 showed a statistically significant decline in sweat chloride at both the 100 mg and 200 mg once-daily doses, suggesting that the activity of the CFTR protein was increased in patients during dosing. Additionally, VX-809 demonstrated a dose response in change in sweat chloride across the four dose groups. On the basis of these results, Vertex plans to initiate a combination trial of VX-809 and VX-770, an investigational CFTR potentiator, in the second half of 2010. VX-809 and VX-770 were developed with support from Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation.

“While the median predicted age of survival for patients with CF has increased to more than 37 years of age, there are no approved therapies that directly target the underlying defect of this disease”

“This Phase 2a trial evaluated the potential effect of an oral compound to improve trafficking of the defective CFTR protein, and its results represent an encouraging step forward in the development of new therapies to treat the underlying cause of CF in patients with the most common CFTR mutation, known as F508del,” said J.P. Clancy, M.D., Director of the Pediatric Pulmonary Center at the University of Alabama at Birmingham and Principal Investigator for the VX-809 Phase 2a trial. “In the trial, VX-809 was well-tolerated across the dose groups, and statistically significant changes in sweat chloride, an important biomarker of CFTR activity, were observed at certain dose levels. There is high interest in the CF community in new approaches to CF therapy, and we look forward to the future exploration of VX-809 and VX-770 as part of a novel combination regimen aimed at treating the majority of CF patients.”

“While the median predicted age of survival for patients with CF has increased to more than 37 years of age, there are no approved therapies that directly target the underlying defect of this disease,” said Robert J. Beall, Ph.D., President and Chief Executive Officer of the Cystic Fibrosis Foundation. “We believe that compounds such as VX-770 and VX-809 represent a promising potential approach to future CF treatment, and the results announced today for VX-809 support future clinical trials of this compound, including a planned clinical trial in combination with VX-770 expected to begin later this year.”