* Statistically significant lung function improvement seen
* Combination designed to address larger CF population
* Final Phase II data expected mid year
* Shares jump 45 percent
(Adds company comments, further analyst comment)
By Bill Berkrot and Lewis Krauskopf
May 7 (Reuters) – Vertex Pharmaceuticals Inc’s new
cystic fibrosis drug Kalydeco, when combined with its
experimental treatment for the disease, led to significant
improvement in lung function in a mid-stage study, sending
Vertex shares soaring 45 percent.
The data suggested Vertex could have a multibillion-dollar
franchise in cystic fibrosis, a life threatening genetic
disorder that affects about 70,000 people worldwide.
Kalydeco, which in January became the first approved drug to
treat the underlying cause rather than symptoms of the
life-shortening lung disease, helps only about 4 percent of
cystic fibrosis patients with a specific gene mutation.
Vertex is testing combinations it hopes will eventually be
able to address the larger CF population. The combination
involved in Monday’s data studied patients with genetic
mutations that make up about half the patients with the disease.
Cystic fibrosis causes the thin layer of mucus that helps
keep the lungs free of germs to become thick, clogging airways
and leading to infections that damage the lungs.
The average life expectancy for the disease is 37 years, as
damage to the lungs progresses, severely limiting the ability to
breathe.
The interim analysis of the study looked at 37 patients who
completed 56 days of treatment with the Kalydeco/VX-809
combination, and 11 patients who received a placebo. It found a
statistically significant lung function improvement for those on
the study drugs.
“These improvements were clinically very, very meaningful
for these patients,” said Brian Skorney, an analyst with Brean
Murray, Carret & Co. “This is really a game-changing combination
therapy.”
The data could be the clearest sign yet that Vertex will
soon be as well known for its cystic fibrosis medicines as its
hepatitis C treatments. Vertex’s Incivek has transformed care
for hepatitis C patients in the past year, but investors are
concerned a new generation of drugs will unseat the Vertex drug
for addressing the infectious disease in the next few years.
ISI Group analyst Mark Schoenebaum said Vertex could have a
$4 billion franchise in cystic fibrosis.
Vertex shares soared 45.1 percent to $54.30 in morning
trading on Nasdaq, elevating the biotechnology company’s market
value to more than $11 billion.
Vertex officials said they would seek to start clinical
trials to support approval for the combination at the end of the
year or early next year.
“These data did exceed our expectations,” Vertex Chief
Executive Officer Jeff Leiden told analysts on a conference
call. “They are leading us to accelerate 809 and Kalydeco into
pivotal trials.”
In the data released on Monday, about 46 percent of patients
who received the drugs experienced lung function improvement of
5 percentage points or more based on FEV1 — a measure of the
maximum amount of air that can be exhaled in one second, Vertex
said.
About 30 percent who received Kalydeco and VX-809 had a lung
function improvement of at least 10 percentage points. None of
the placebo patients achieved a 5 percent improvement from
baseline to day 56, the company said.
“The fact that it showed improvements in lung function in
this small-sized trial I think is very impressive,” Skorney
said.
The drugs were generally well tolerated with serious adverse
events similar between the treatment and placebo groups, the
company said. Complete data from the Phase II study are expected
to be available in the middle of this year.
(Reporting By Bill Berkrot and Lewis Krauskopf; Editing by
Gerald E. McCormick, Maureen Bavdek, Dave Zimmerman)
