After stumbling on high investor expectations for its experimental hepatitis C therapy, telaprevir, Vertex garnered a dose of good news with the release of positive Phase IIa data on VX-770 for cystic fibrosis. Researchers reported that the small, 20-patient study revealed improved lung function in patients taking the oral drug. There was no discerned improvement in patients taking a placebo. Vertex shares jumped 10 percent on the news.
"While these are early data, it is unprecedented for an investigational oral compound for the treatment of CF to have such a marked effect on multiple measures of CF disease activity. We saw an average improvement in lung function of 10 percent in patients receiving the highest dose, compared to no observed improvement in patients who received placebo," said Frank Accurso, M.D., Director of the Cystic Fibrosis Center and Professor of Pediatrics at the University of Colorado School of Medicine in Denver.
"More than 10 years ago, the Cystic Fibrosis Foundation believed that Vertex's approach to treating CF by directly targeting CFTR function--the underlying mechanism of the disease--was an important opportunity to meaningfully change the course of cystic fibrosis," said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation.
- see the release
- read the AP report on the data