(Clarifies in fourth paragraph that Kalydeco is not used for
patients with the most common genetic mutation associated with
By Deena Beasley
May 1 Vertex Pharmaceuticals Inc on
Thursday said a combination of its cystic fibrosis drug Kalydeco
and an experimental compound was shown to improve lung function
in a mid-stage trial, sending its shares up nearly 8 percent.
The study found that treatment with Kalydeco and the
experimental drug VX-661 for 28 days resulted in a 4.6
percentage point improvement in mean lung function for patients
with two specific genetic mutations.
If eventually approved by regulators, VX-661 would be the
second drug from Vertex that works by treating the underlying
cause of cystic fibrosis, a rare genetic disease that impairs
the lungs and digestive system.
The disease is caused by defective or missing CF
transmembrane conductane regulator (CFTR) proteins. Kalydeco is
designed for patients with certain CFTR mutation. VX-661 is
being tested in combination with Kalydeco, as in this Phase 2
trial, and in patients with different genetic mutations.
"It is good data and some believe it does have an
incremental positive read-through to the all-important Phase 3
data later this summer," said RBC Capital Markets analyst
That trial will answer the question of whether a combination
of Vertex drugs is effective in patients with two copies of the
gene mutation not currently addressed by Kalydeco - a market
representing about half of the 30,000 cystic fibrosis patients
in the United States.
A positive result "would be transformative to the cystic
fibrosis community," Yee said.
The most common side effects seen during the Phase 2 trial
included cough, headache and upper respiratory tract infection.
But ISI Group analyst Mark Schoenebaum cautioned that
Vertex has not disclosed results from the mid-stage trial for
patients treated with a placebo.
Vertex also announced that first quarter sales of Kalydeco
totaled $100 million, which fell short of the average analyst
estimate of $107 million, according to Wells Fargo Securities.
Separately, The company reported an adjusted first quarter
loss of $1.00 per share, also below the average analyst
per-share loss estimate of 67 cents, according to Thomson
Vertex said it would discontinue research into treatments
for hepatitis C, a market now dominated by sales of Gilead
Sciences Inc's Sovaldi. Vertex late last year sold off
its non-North America royalty rights to hepatitis C drug
Shares of Vertex, which closed at $68.95 in regular Nasdaq
trading, were up $5.30 at $74.25 after hours.
(Reporting By Deena Beasley; Editing by David Gregorio and