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 The company's stock fell about 16 percent in extended trading on
Thursday, before reversing course to notch marginally gains.
PTC said the drug failed to show a statistically significant benefit
over a placebo in patients with Duchenne muscular dystrophy (DMD),
for which there is no therapy approved by the U.S. Food and Drug
Administration.
However, in a sub-group of patients with less advanced DMD, the
effect of the drug, Translarna, was deemed clinically meaningful.
PTC said the new data, combined with findings from previous trials
on the drug, supported a U.S. marketing application for Translarna,
which won conditional European approval last year.
"Without a doubt these results confirm - from the totality of data -
Translarna's ability to slow disease progression, and we are
actively planning for a U.S. launch in 2016," Chief Financial
Officer Shane Kovacs told Reuters.
DMD is a progressive degenerative disorder that hampers muscle
movement and affects one in 3,600 newborn boys, most of whom die by
the age of 30.
Oppenheimer's Christopher Marai said the findings were positive
enough to support an FDA approval and the continued sale of
Translarna in the European Union.
"It was always difficult to show results on the primary endpoint in
the first place and that's the because of the nature of the endpoint
and disease progression," he said.
PTC conducted the trial on 228 boys, the largest-ever study on DMD
patients, and aimed to show Translarna was more effective than a
placebo in helping patients walk 30 meters more than they could
without medication.
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However, data showed Translarna helped patents walk only 15 meters
more on average, but in less advanced patients it was able to induce
a highly significant benefit of 47 meters.
Translarna met all secondary and tertiary endpoints, with a robust
safety profile.
Translarna targets DMD caused by nonsense mutations. This form of
DMD affects roughly 7,000 boys globally, including about 2,000 in
the United States and Canada.
PTC said its will present data from the study to the European
Medicines Agency in the near term and to the FDA, as part of its
rolling marketing application, by the end of 2015.
Oppenheimer's Marai estimates the drug represents a $1.2 billion
global opportunity.
(Reporting by Natalie Grover in Bengaluru; Editing by Savio D'Souza)
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