Monday, June 30, 2014
anticipated Phase III data for the combination of lumacaftor and Kalydeco
ivacaftor from Vertex Pharmaceuticals Inc.
didn't disappoint. Investors boosted the biotech's market cap by $6.4 billion,
or 40%, on data that could substantially increase Vertex's penetration of the
CF market. The improvements in FEV1 were relatively small but could add up over
time and yield long-term benefit for patients.
Vertex said that in two identical Phase III studies, TRAFFIC and TRANSPORT,
Kalydeco and lumacaftor met the primary endpoint of statistically significant
improvements in percent predicted forced expiratory volume in one second
(ppFEV1) at 24 weeks. The trials enrolled patients ages 12 and older who were
homozygous for the delta F508 mutation in the CF transmembrane conductance
regulator (CFTR) gene.