Vertex Pharmaceuticals late-stage program for the closely-watched cystic fibrosis drug VX-770 wrapped another successful Phase III clinical trial. News of the data helped buoy one of the hottest stocks in biotechnology as Vertex remains right on track for a New Drug Application later this year.
VX-770 is an example of the kind of personalized medicines that promise to gradually revolutionize patient therapy. By targeting a genetic trigger linked to four percent of all CF cases, the developer is attempting to carefully target the cause rather than just the symptoms of the disease. And in this new Phase III-which included 52 children--the experimental drug produced a 12.5 percent improvement in lung function over the baseline among young patients aged 6 to 11 after 24 weeks of treatment. Investigators will continue to track patients and provide a 48-week update on results.
"In this study, children with CF treated with VX-770 showed the same profound improvements in lung function seen in the recently-announced STRIVE study among an older group of people with the G551D mutation," said Robert Kauffman, M.D., Ph.D., the CMO at Vertex. "We are committed to making VX-770 available as soon as possible and are moving ahead quickly with our U.S. and European applications for regulatory approval that we plan to submit in the second half of this year."
News of the fresh clinical advance lifted Vertex shares by 2.2 percent. Vertex has already filed for an approval of its hepatitis C drug telaprevir, which is widely seen as an odds-on favorite at the FDA.
- here's the Vertex release
- check out the Forbes report