Vertex Pharmaceuticals Inc, Cambridge, Mass, released yesterday an interim analysis of data from its ongoing phase 2 study of [removed]VX-809[/removed] and [removed]KALYDECO™[/removed] (ivacaftor) that showed improvements in lung function (FEV1) among adults with cystic fibrosis who have two copies (homozygous) of the most common mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, F508del.
The 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.
A statistically significant improvement in FEV1 was found 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% (17/37) experienced an absolute improvement from baseline to day 56 in lung function of 5 percentage points or more, and approximately 30% (11/37) experienced an absolute improvement from baseline to day 56 of 10 percentage points or more. 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 plans to start a study of VX-809 and KALYDECO in people with two copies of the F508del mutation, pending final study results and discussions with regulatory agencies. Evaluation of patients with one copy (heterozygous, n=21) of the F508del mutation in ongoing, but at the time of this interim analysis, not enough patients had completed the study to make any conclusions. Complete data will be included in the final study analysis and will be used to determine next steps for the development of VX-809 and KALYDECO in heterozygous F508del patients.
Source: Vertex Pharmaceuticals Inc