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PTC Therapeutics

Latest Headlines

Latest Headlines

PTC wins EU approval for its DMD drug as Phase III data pend

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.

PTC soars as EU changes its tune on the DMD-treating ataluren

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.

Sarepta rockets up after mapping a shortcut to the FDA with DMD hopeful eteplirsen

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.

Scientists dispute PTC's claim on muscular dystrophy drug mechanism

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.

PTC overcomes repeated setbacks to raise $125M in IPO

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.

UPDATED: PTC, Esperion, bluebird join the IPO frenzy in biotech with genetic therapies

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.

UPDATED: Biotech PTC reloads coffers with $60M for late-stage DMD drug

PTC Therapeutics has scored new financing to bankroll development of a compound for certain types of Duchenne muscular dystrophy and cystic fibrosis.

PTC investors back lead orphan drug with $30M round

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 tanks after positive PhIIb fails to improve outcomes for DMD

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,

Biogen Idec strikes $299M deal for Isis antisense drug

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