Vertex Begins Clinical Trial to Evaluate Combination of VX-770 and VX-809 Targeting Most Common Mutation in CF
October 18, 2010
Vertex Pharmaceuticals today announced the initiation of a clinical trial that will evaluate –– for the first time — combinations of VX-770 and VX-809, two oral drugs in development that target the underlying cause of CF.
The Phase 2a trial will test the drugs in people who have two copies of the most common mutation of CF, known as Delta F508. About 50 percent of people with CF in the United States have two copies of this mutation; nearly 90 percent have at least one copy.
“The start of this trial has been greatly anticipated by the CF community and marks a milestone in our efforts to discover and develop new treatments to address the underlying cause of this disease,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “This is the first opportunity to evaluate a combination of these promising therapies that together may help address the most prominent and challenging problems associated with the most common defect in CF and provide important insight for future treatment.”
VX-770 and VX-809 were discovered as part of a collaboration with the Cystic Fibrosis Foundation.
CF is caused by defective or missing CFTR proteins. 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 trial is designed to evaluate the safety and tolerability of VX-809 dosed alone for 14 days followed by dosing of VX-770 in combination with VX-809 for 7 days.
The trial will also assess the effect of VX-809 alone and in combination with VX-770 on CFTR function, as measured by sweat chloride.
The three-part trial is designed to enroll up to a total of 160 people and could pave the way for further studies using combinations of compounds to address the underlying defect in cystic fibrosis.
In addition to the combination trial, VX-770 is being tested as a stand-alone drug in a Phase 3 registration program. Results from this program are expected in the first half of 2011, and Vertex anticipates it will submit a New Drug Application for VX-770 to the U.S. Food and Drug Administration in the second half of 2011.
Additional Resources
| 
