Vertex Pharmaceuticals Incorporated ( VRTX ) received a huge
boost with the company reporting impressive phase II data on its
cystic fibrosis (CF) candidate, VX-661.
The randomized, double-blind, placebo-controlled phase II study
was conducted in cystic fibrosis patients (adults) who have two
copies (homozygous) of F508del, the most common mutation in the
cystic fibrosis transmembrane conductance regulator (CFTR)
Four once-daily doses of VX-661 (10, 30, 100 and 150 mg)
administered with ivacaftor (150 mg twice-daily) were evaluated in
the study for 28 days. While VX-661 is a CFTR corrector, ivacaftor
is a CFTR potentiator. VX-661 was also evaluated as a monotherapy
in a separate group of patients. Primary endpoints were safety,
tolerability and change in sweat chloride with change in lung
function being a secondary endpoint.
Vertex Pharma said that dose-dependent, mean relative
improvements in lung function were observed both within the group
and compared to placebo. Statistically significant mean relative
improvements in lung function were observed in the 100 and 150 mg
combination groups compared to placebo. The results provide further
support to Vertex Pharma's strategy of combining a corrector with
VX-661 was found to be well-tolerated as monotherapy as well as
combination therapy. Going forward, Vertex Pharma intends to
evaluate VX-661 in additional studies following discussions with
Vertex Pharma's shares were up significantly in pre-market
trading. We are pleased with the company's progress with its cystic
Lead cystic fibrosis candidate, VX-809, is currently in phase
III development. Positive 24-week safety and efficacy data on
VX-809 would allow Vertex Pharma to seek US Food and Drug
Administration (FDA) approval in 2014.
The company has another cystic fibrosis candidate, VX-983, which
is currently in a phase I multiple-ascending-dose study in healthy
volunteers. VX-983 is scheduled to move into a 28-day study in
combination with ivacaftor in the second half of this year.
We note that ivacaftor is currently approved in the US, Canada
and the EU under the trade name Kalydeco for the treatment of
cystic fibrosis patients (six years and older) who have at least
one copy of the G551D mutation in the CFTR gene. Kalydeco, which
delivered revenues of $171.6 million in 2012, is off to a strong
start and should continue performing well in 2013.
We are optimistic on the growth prospects of Vertex Pharma's
cystic fibrosis franchise. Vertex Pharma currently carries a Zacks
Rank #1 (Strong Buy).
Companies that look equally attractive include Actelion
Ltd. ( ALIOF ),
Athersys, Inc. ( ATHX ) and
Osiris Therapeutics, Inc. ( OSIR ). All three are
Zacks Rank #1 stocks.ACTELION LTD (ALIOF): Get Free ReportATHERSYS INC (ATHX): Free Stock Analysis ReportOSIRIS THERAPTC (OSIR): Free Stock Analysis
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