Vertex Pharma (VRTX) Reports Statistically Significant Data from VX-809 and KALYDECO Phase 2 in Lung Function
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Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reports an interim analysis of data from an ongoing Phase 2 study of VX-809 and KALYDECO (ivacaftor) that showed significant improvements in lung function (FEV1) among adults with cystic fibrosis (CF) who have two copies (homozygous) of the most common mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, F508del.
A planned interim analysis was conducted after approximately half of the study patients had completed 56 days of treatment. 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. 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. 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 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. Evaluation of patients with one copy (heterozygous, n=21) of the F508del mutation is ongoing, but at the time of this interim analysis not enough patients had completed the study to make any conclusions. These 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. Vertex will host a conference call for investors and media today, May 7, 2012 at 8:30 a.m. ET to discuss this interim data analysis.
Cystic fibrosis is a rare, life threatening genetic disease affecting approximately 30,000 people in the United States and 70,000 people worldwide. Nearly half of those with CF are estimated to have two copies of the F508del mutation.
A planned interim analysis was conducted after approximately half of the study patients had completed 56 days of treatment. 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. 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. 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 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. Evaluation of patients with one copy (heterozygous, n=21) of the F508del mutation is ongoing, but at the time of this interim analysis not enough patients had completed the study to make any conclusions. These 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. Vertex will host a conference call for investors and media today, May 7, 2012 at 8:30 a.m. ET to discuss this interim data analysis.
Cystic fibrosis is a rare, life threatening genetic disease affecting approximately 30,000 people in the United States and 70,000 people worldwide. Nearly half of those with CF are estimated to have two copies of the F508del mutation.
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