BioCentury
ARTICLE | Top Story

Vertex jumps on CF data

June 25, 2014 12:24 AM UTC

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) jumped $26.92 (40%) to $93.53 on Tuesday on positive data for two of the company's CF agents in a pair of Phase III trials -- a data readout that investors told BioCentury would be among the biggest make-or-break events of the quarter. In the identical TRANSPORT and TRAFFIC trials, Vertex's Kalydeco ivacaftor plus lumacaftor ( VX-809) met the primary endpoint of improving the absolute change from baseline in percent predicted forced expiratory volume in one second (FEV1) at week 24 vs. placebo. The double-blind trials enrolled a combined 1,108 CF patients with two copies of the delta F508 CF transmembrane conductance regulator ( CFTR) mutation.

In 4Q14, Vertex plans to submit an NDA to FDA and an MAA to EMA for the Kalydeco/lumacaftor combination for CF patients ages 12 and older with the homozygous delta F508 mutation. The combination has breakthrough therapy designation for the indication in the U.S. According to the company, there are more than 22,000 CF patients ages 12 and older in North America, Europe and Australia with homozygous delta F508 mutation, which Vertex said is the most prevalent genetic mutation that causes the disease. ...