Vertex Pharmaceuticals Inc. (VRTX) announced an interim analysis of data from an ongoing Phase 2 study of VX-809 and Kalydeco (ivacaftor) that showed significant improvements in lung function or FEV1 among adults with cystic fibrosis or CF who have two copies (homozygous) of the most common mutation in the cystic fibrosis transmembrane conductance regulator or CFTR gene, F508del.
A planned interim analysis was conducted after approximately half of the study patients had completed 56 days of treatment.
The company said that today's results are based on data from 37 homozygous F508del patients who completed treatment in the 56-day study and 11 patients with one or two copies of the F508del mutation who received placebo.
According to interim analysis of data, there was a statistically significant improvement in lung function (absolute change in percent predicted FEV1) across the combined treatment groups relative to baseline compared to placebo (p=0.002). Of those who received VX-809 and Kalydeco (250mg, q12h), approximately 46 percent (17/37) experienced an absolute improvement from baseline to Day 56 in lung function of 5 percentage points or more, and approximately 30 percent (11/37) experienced an absolute improvement from baseline to Day 56 of 10 percentage points or more.
The company stated that none of the patients treated with placebo (0/11) achieved a 5-percentage point or more improvement from baseline to Day 56 in lung function. Most adverse events were mild or moderate in severity and comparable between treatment and placebo groups.
The study is ongoing and complete data, including statistical analyses for all patient groups, will be available in mid-2012.
Vertex noted that it plans to start a pivotal study of VX-809 and Kalydeco in people with two copies of the F508del mutation, pending final study results and discussions with regulatory agencies.
by RTT Staff Writer
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