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news.Vertex Pharmaceuticals (Vertex) has provided 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.
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Reportedly, in the trial, VX-809 showed a decline in sweat chloride at both the 100mg and 200mg once-daily doses, suggesting that the activity of the CFTR protein was increased in patients during dosing.
Additionally, VX-809 has 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, the nonprofit affiliate of the Cystic Fibrosis Foundation.
JP Clancy, principal investigator for the trial, said: “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.
“In the trial, VX-809 was well-tolerated across the dose groups, and changes in sweat chloride 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.”