PTC Therapeutics

Latest Headlines

Latest Headlines

PTC's muscular dystrophy drug fails another key test with approval on the line

PTC Therapeutics' in-development treatment for Duchenne muscular dystrophy failed on its main goal in a Phase III trial. But, poring over subpopulation data, the company still believes it can make its case to global regulators.

Roche presses pause on PTC's SMA drug over a safety scare

Roche has suspended dosing in a Phase II trial of PTC Therapeutics' in-development spinal muscular atrophy trial, cautious of a potential safety problem stemming from a preclinical study.

PTC is trawling for a buyer with its risky DMD drug, Reuters says

PTC Therapeutics is looking to get bought, Reuters ' sources say, betting that a larger company will covet its treatment for Duchenne muscular dystrophy enough to overlook a checkered history.

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.