April 18 Vertex Pharmaceuticals Inc
said its experimental drug improved lung function in adults with
cystic fibrosis in a mid-stage trial, sending its shares 54
percent higher in after-hours trading.
The company said on Thursday the favorable results were seen
in patients who took the drug, known as VX-661, in combination
with its already approved treatment for cystic fibrosis,
Kalydeco (ivacaftor) in a short 28-day study.
The Phase II study involved 128 people with cystic fibrosis
who had two copies of the most common gene mutation responsible
for the debilitating condition in which the lungs are unable to
flush out salt and therefore become flooded with mucous and can
become easily infected.
Patients treated in the two highest dose groups had a mean
relative increase in lung function of between 7.5 percent and 9
percent, compared with patients given a placebo.
The results were "impressive," Wells Fargo analyst Brian
Abrahams said in a research note. He added that they
"meaningfully increase the likelihood of success" for the
company's program to develop combination treatments for cystic
He has estimated the market for a dual cystic fibrosis
treatment at over $3 billion.
Vertex said the experimental drug was generally well
tolerated when used alone and in combination with Kalydeco.
Vertex shares were up 54 percent at $81.55 in after-hours
trading from their closing of $52.87 Thursday on the Nasdaq.