BioCentury
ARTICLE | Clinical News

Vertex reports Kalydeco data for another CF population

June 5, 2014 12:51 AM UTC

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) said Kalydeco ivacaftor met the primary endpoint vs. placebo in the first part of a crossover Phase II trial to treat cystic fibrosis (CF) in patients with a residual function mutation in the CF transmembrane conductance regulator ( CFTR) gene. Kalydeco improved mean absolute change in percent predicted forced expiratory volume in one second (FEV1) from baseline to the end of the two-week treatment period vs. placebo (2.8 vs. 0.6 percentage points, p=0.004). The data came from 24 CF patients with clinical evidence of residual CFTR function in part 1 of the trial. In 21 patients in the second, eight-week, open-label portion of the trial, Kalydeco led to a mean absolute change in percent predicted FEV1 of 4.7 percentage points (p<0.0001) compared to baseline of part 2.

Vertex plans to start a Phase III trial of Kalydeco in CF patients with residual CFTR function mutations, but declined to disclose a time frame. According to Vertex, there are more than 3,000 CF patients six years of age and older in North America, Europe and Australia who have a non-R117H residual function mutation. Last year, Vertex reported that Kalydeco missed the primary endpoint in CF patients with at least one copy of the R117H mutation in the Phase III KONDUCT trial (see BioCentury Extra, Dec. 19, 2013). ...