Vertex Pharmaceuticals Inc.
) share price jumped 55.36% following the release of positive
interim data from a mid-stage study of its combination t herapy -
VX-809 and Kalydeco (ivacaftor). The study is evaluating adults
with cystic fibrosis (CF) who have two copies (homozygous) of the
most common mutation in the CF transmembrane conductance regulator
(CFTR) gene, F508del.
The 56-day study data compared 37 homozygous F508del patients
who were treated with the combination drug with 11 patients with
one or two copies of the F508del mutation treated with placebo.
Interim data from the study revealed that while 46% patients in
the VX-809 plus Kalydeco arm experienced at least 5% absolute
improvement in lung function from baseline, 30% patients in the
cohort experienced at least 10% absolute improvement. However, none
of the patients in the placebo arm achieved 5% improvement in lung
function from baseline.
Vertex Pharma plans to report the complete data from the study
in mid-2012. Following these results and discussions with
regulatory agencies, the company plans to initiate a pivotal study
of VX-809 and Kalydeco in patients with two copies of the F508del
We believe that if the combination therapy manages to clear the
regulatory hurdles and hit the markets, then the top-line at Vertex
Pharma will be boosted.
We remind investors that Kalydeco was approved in the U.S. in
February 2012, for treating CF patients above 6 years of age.
Vertex Pharma is also seeking approval of the drug for the same
indication in the European Union with a response expected in
We currently have a Neutral recommendation on Vertex Pharma. The
stock carries a Zacks #3 Rank (Hold rating) in the short-run.
VERTEX PHARM (VRTX): Free Stock Analysis Report
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