Vertex reports VX-809 data

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) said two doses of oral VX-809 once daily met the secondary efficacy endpoint of significantly reducing mean sweat chloride levels vs. placebo in a Phase IIa trial to treat cystic fibrosis. Specifically, the 100 and 200 mg doses had reductions of 6.13 and 8.21 mmol/L, respectively, vs. placebo (p=0.05 and p=0.01). However, the 25 and 50 mg doses of VX-809 both missed the efficacy endpoint vs. placebo. The primary endpoint of the trial was safety. Vertex said VX-809 was well-tolerated at all doses and a detailed safety analysis is ongoing. The double-blind trial enrolled 89 patients who are homozygous for the delta F508 cystic fibrosis transmembrane conductance regulator (CFTR) mutation. VX-809 is a small molecule CFTR corrector. ...