CF Investigational Drug VX-809 Shows Encouraging Results
in Phase 2a Trial
February 3, 2010
Vertex Pharmaceuticals Incorporated announced today results from a Phase 2a trial of VX-809, an oral investigational drug that aims to correct the basic defect in cystic fibrosis. VX-809 was found to be well-tolerated and to reduce sweat chloride levels — a key indicator of CF.
The 28-day, Phase 2a trial of VX-809 examined the drug in cystic fibrosis patients who have the Delta F508 gene mutation, the most common mutation in CF. The study focused primarily on the safety and tolerability of the drug and changes in sweat chloride.
A reduction in sweat chloride levels in the Phase 2a data suggests that VX-809 may improve the function of CFTR, the faulty protein in CF.
“These are very exciting and important results in our effort to find ways to treat the basic defect in cystic fibrosis,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “The VX-809 data further supports our hypothesis that small molecules can be used to treat the underlying genetic cause of cystic fibrosis.”
The data from this trial pave the way for future studies of VX-809, including testing the therapy in combination with VX-770. Also developed by Vertex, VX-770 is an oral investigational drug that showed encouraging Phase 2 results in restoring the function of CFTR in patients with the G551D mutation of CF.
Research in the laboratory suggests that using two therapies in combination may increase CFTR function in cells with the Delta F508 mutation when compared to using a single therapy alone.
The first trial examining VX-809 and VX-770 in combination in cystic fibrosis patients is currently in the planning phases, and is expected to begin in the United States in the second half of 2010.
In addition, Vertex is further reviewing the Phase 2a data and may explore the option of studying VX-809 as a single drug in higher doses in a separate clinical trial.
Through its Therapeutics Development Program, the CF Foundation collaborated with Vertex to discover and develop VX-809 and VX-770, investing approximately $76 million in the effort. This represents the largest single investment in CF drug discovery by the Foundation.
Additional Resources
| 
