12:00 AM
Jun 04, 2012
 |  BioCentury  |  Product Development

Combo confidence

Vertex, trial investigators look past data hiccup in cystic fibrosis study

Vertex Pharmaceuticals Inc. and two academic investigators contend that a missed primary endpoint, a data snafu and atypical results in placebo patients all should take a back seat to FEV1 improvements produced by the company's VX-809 plus Kalydeco ivacaftor in a Phase II cystic fibrosis trial.

A clearer picture of the study should emerge shortly, as final data from all patients are expected by mid-year.

The study enrolled 108 CF patients with either one or two copies of the delta F508 CFTR mutation. Patients received placebo or once-daily VX-809 alone for 28 days and then in combination with twice-daily Kalydeco for an additional 28 days.

The saga began on May 7, when Vertex announced data from an interim analysis of 37 homozygous patients and 11 placebo patients who had one or two copies of the mutation.

Although the 37 patients missed the primary endpoint of a reduction in sweat chloride from day 28 to day 56 vs. placebo, investors focused on secondary endpoint data on the more clinically relevant measure of FEV1.

Indeed, Vertex shares surged 66% to $62.02 on the news that 46% (17/37) of the patients receiving VX-809 plus Kalydeco had a >=5% absolute improvement from baseline in FEV1, while 30% (11/37) of patients in the pooled treatment group had a >=10% absolute improvement in...

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