Vertex announced its latest phase III trial results with a cystic fibrosis (CF) drug, VX-770, that improved patients lung performance by 17 percent versus a placebo - a remarkable result. The effect lasted over the entire 48 week course of the study.
The drug is targeted at a subpopulation of CF suffers (approx 4-5%) who have a particular mutation known as G551D. In these patients, the drug is the first to actually treat the abnormal CFTR protein underlying the disease. Vertex will apply for approval with the FDA and EMEA before the end of 2011. The company also plans to test whether the drug will have any benefits for patients with the F508 mutation - the most common among CF patients.
Vertex is also working on a complementary drug, VX-809, and data on the performance of the two drugs in combination is expected later this year. The positive news increased Vertex's stock price by more than 15% to 44.03 per share earlier today. This is more welcome news for Vertex which also has the promising hepatitis C drug, telaprevir, wending its way through clinical trials to approval.
Posted by Bruce Lehr Feb 25th 2011.