Vertex Pharmaceuticals Inc. (VRTX) announced a correction to the previously reported responder analysis, as well as additional data from, the recent interim analysis of an ongoing Phase 2 study of VX-809 and KALYDECO (ivacaftor) that showed significant improvements in lung function among adults with cystic fibrosis who have two copies of the most common mutation in the cystic fibrosis transmembrane conductance regulator gene, F508del.
As previously announced, there was a statistically significant improvement in lung function across the combined treatment groups relative to baseline compared to placebo.
Vertex Pharma said today's announcement provides a correction to the responder analysis for the absolute improvement in lung function compared to baseline and, for the first time, provides the mean absolute improvement in lung function compared to placebo observed among patients who received VX-809 and KALYDECO. The data reported today and earlier this month are based on 37 patients who completed 56 days of treatment with VX-809 and KALYDECO and 11 patients with one or two copies of the F508del mutation who received placebo.
The company also provided additional data from the interim analysis for people with two copies of the F508del mutation. A mean absolute improvement in lung function of 8.5 percentage points was observed among those who were treated with VX-809 and KALYDECO compared to placebo. In addition, the within-group mean absolute improvement from baseline to Day 56 was 4.0 percentage points for patients treated with the combination.
Analyses are ongoing and complete data, including statistical analyses for all patient groups, will be available mid-year. Vertex 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.
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