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Vertex reports potential CF drug combination significantly improves lung function; shares rise

Monday, May 07 2012 | Comments
Evidence Grade 0 What's This?
Vertex Pharmaceuticals Inc.'s VX-809, an investigational treatment for cystic fibrosis (CF), provided significant improvements in lung function in a recent midstage study when administered in combination with Vertex's Kalydeco (ivacaftor), which just gained Food and Drug Administration approval in January for certain patients with CF.

Shares of Vertex soared 55.4 percent on news of the positive interim data.

The recently released findings are from the second part of an ongoing, double-blind, randomized, Phase II study. A total of 108 adults with CF with one or two copies of the F508del mutation are participating in this portion of the trial. The participants have been divided into five treatment arms. Three groups of homozygous patients are receiving VX-809 alone, at a dose level of 200 mg, 400 mg or 600 mg, for 28 days and then in combination with Kalydeco 250 mg twice daily for an additional 28 days. A group of heterozygous patients is receiving VX-809 alone, at a dose level of 600 mg, for 28 days and then in combination with Kalydeco 250 mg twice daily for an additional 28 days. A placebo group includes both homozygous and heterozygous patients.

An interim analysis was conducted after approximately half of the participants had completed 56 days of treatment. The latest results were based on data from 37 homozygous patients and 11 patients from the placebo group.

As compared with the placebo-treated patients, the combined treatment groups experienced a statistically significant improvement in lung function, as determined by an absolute change in percent predicted forced expiratory volume in one second, relative to baseline.

Seventeen of the 37 patients who received VX-809 plus Kalydeco experienced an absolute improvement from baseline to day 56 in lung function of 5 percentage points or more, and 11 experienced an absolute improvement from baseline to day 56 of 10 percentage points or more.

None of the placebo-treated patients achieved an improvement in lung function from baseline to day 56 of at least 5 percentage points.

Most adverse events were comparable between the treatment and placebo groups and were mild or moderate in severity.

Complete data from the trial will be available midyear, Vertex noted. Pending final study data and discussions with regulatory authorities, the firm is planning to initiate a pivotal study of VX-809 and Kalydeco at that time. The intended patient population for that study is individuals with CF who have two copies of the F508del mutation.

Vertex shares closed at $58.12, up $20.71, in heavy trading on the Nasdaq.

Kalydeco is indicated for patients aged 6 years or older who have CF and at least one copy of the G551D mutation in the CF transmembrane conductance regulator gene.

This information concerns a use that has not been approved by the FDA.

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