After missing a key trial goal and getting a rare second chance from EU regulators, PTC Therapeutics' ataluren is now the world's first approved therapy for Duchenne muscular dystrophy, picking up a conditional European OK as the company awaits results from a late-stage study.
Reversing an earlier rejection, European regulators are recommending early approval for PTC Therapeutics' ataluren, a treatment for Duchenne muscular dystrophy, news that more than doubled the biotech's share value in premarket trading.
After keeping investors on a roller coaster ride of speculation for months, Sarepta says it now plans to shoot for an accelerated approval of its Duchenne muscular dystrophy drug eteplirsen. The biotech says it will file for an early approval by the end of this year after launching a slate of new clinical trials to get the data that the FDA is looking for.
The Scientist is reporting that a group of investigators have completed an in vitro study of their lead treatment and came to a startling conclusion: It doesn't work the way PTC has said it does.
The market for biotech IPOs has proven strong enough to welcome PTC Therapeutics, a player in the Duchenne muscular dystrophy field with a consistent record of late-stage failure. The biotech announced today that it has priced its IPO at $15, right at the middle of its range, while boosting the number of shares on offer to 8.4 million in order to reap a larger harvest of cash.
Two more biotech outfits emerged this week as contenders to complete initial public offerings, highlighting the open season for the deals this year. Bluebird bio and PTC Therapeutics has mounted campaigns to go public with pipelines that target the molecular causes of rare genetic diseases.
PTC Therapeutics has scored new financing to bankroll development of a compound for certain types of Duchenne muscular dystrophy and cystic fibrosis.
Two years after putting together a $50 million round, PTC Therapeutics has gone back to the venture well and drawn up a bucket of bucks worth $30 million. The money will be used to back the late-stage development of ataluren, PTC's lead program for Duchenne/Becker muscular dystrophy as well as cystic fibrosis.
AVI Biopharma heralded the news that its lead therapy hit the primary endpoint in a Phase IIb study, increasing levels of dystrophin in a tiny trial for Duchenne muscular dystrophy, but the boys did not experience a significant improvement in walking ability,
Joining a race of pharma giants to treat rare cases of spinal muscular atrophy, Biogen Idec ($BIIB) has committed up to $299 million to grab rights to an antisense drug program in the pipeline at