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 The data announced by the company on Tuesday are seen as
critically important to Vertex, which is virtually assured of a
return to profitability following approval of the therapy.
"The data are good and will clearly support worldwide regulatory
approval for this drug regimen," ISI Group analyst Mark Schoenebaum
said in a research note. "Worldwide peak sales could exceed $3
billion."
Vertex shares were up 42.3 percent at $94.80 on Nasdaq after the
company reported its CF drug Kalydeco in combination with its
experimental lumacaftor achieved the main goals of the studies
called Traffic and Transport in patients with the most common
genetic mutation associated with the disease.
Based on the results, the company said it would file applications
for U.S. and European approval for the combination in the fourth
quarter.
In the two, 24-week studies of patients 12 years and older with two
copies of F508del genetic mutation, the combination therapy led to
mean absolute improvements in lung function of between 2.6 and 4
percentage points, which was deemed to be statistically significant.
 "(The data) opens up 28,000 F508Delta patients, giving Vertex a
multi-billion dollar franchise and essentially a monopoly for
years," Maxim Group analyst Jason Kolbert said.
Kalydeco is approved to treat cystic fibrosis (CF) patients with a
different genetic mutation that accounts for about 2,000 patients
worldwide. It had sales of $371 million in 2013.
About 22,000 CF patients aged 12 and over have two copies of the
F508del mutation. Once pediatric trials are completed another 6,000
patients under 12 could become eligible for the treatment.
Lung function improvement was measured by FEV1, or the amount of air
a patient can exhale in one second.
The mean relative improvements in lung function seen for patients
from baseline measurements was 4.3 percent to 6.7 percent, the
company reported.
"On average, people with CF who have two copies of the F508del
mutation lose nearly two percent of their lung function each year,
underscoring the urgent need for new medicines," Dr. Bonnie Ramsey,
lead investigator of one of the studies, said in a statement.
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A pooled analysis of data from the two trials also showed
significant reductions of between 30 and 39 percent in the rate of
pulmonary exacerbations, or acute worsening of symptoms, for those
who got the two drugs compared with the placebo groups.
The combination also led to small but statistically significant
increases in body mass index. That is viewed as a beneficial as CF
patients have trouble gaining weight.
Kalydeco (ivacaftor) and lumacaftor address the underlying cause of
CF rather than just symptoms of the disease, in which a missing or
defective protein called CFTR results in poor flow of salt and water
into and out of cells in the lungs. That causes a buildup of thick,
sticky mucus that can lead to chronic lung infections, progressive
lung damage and death at an early age.
The two studies involved a total of about 1,100 patients with each
trial having two active treatment dosing regimens and a placebo
group.
Among those who received the combination, 4.2 percent dropped out
due to adverse side effects compared with a 1.6 percent dropout rate
in the placebo group. The most common adverse side effects were
infective pulmonary exacerbation, cough, headache and increased
sputum.
(Reporting by Bill Berkrot; additional reporting by Esha Dey in
Bangalore; Editing by Bernard Orr and Sofina Mirza-Reid)
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