PTC Therapeutics, Inc. (NASDAQ:PTCT) has announced that it would be presenting at the “5th International Congress of Myology”, being held in France, from March 14 to 18, 2016. The company would be using this platform to release data from a phase-3 study of Translarna (ataluren). The drug is being developed for the treatment of nmDMD. PTC has several poster presentations scheduled during the conference, concerning Translarna.
The therapy is aimed at developing a functioning protein, to counter a genetic mutation in the body. Nonsense mutations tend to halt the synthesis of essential proteins in the body, prematurely, by altering the genetic code. Although the therapy is an investigational therapy in the US, it has been licensed in the European Economic Area. Furthermore, Translarna is also classified as an orphan drug by the FDA, the Cystic Fibrosis Foundation and several other prestigious institutions.
Apart from releasing the data during the conference, PTC has also announced that it would be submitting the results for review by Health Canada. The submission would be in accordance with the New Drug Submission (NDS) process. However, in order to submit the ACT DMD results, the company would have to withdraw its current NDS application and file a new one, with the updated data. Consequently, the company would also need to update its guidance of gaining the NDS approval from the regulatory body during the 1H2016.
PTC is also going to face a class action lawsuit, filed by Wolf Popper LLC, in the District Court of New Jersey. The lawsuit concerns all shareholders, who bought a stake in the company, between May 7, 2014 and through February 29, 2016. The lawsuit claims that PTC misguided the public about its NDA application for Translarna being on track. The filing claims that PTC did not indicate that the data was insufficient for FDA to grant an approval, the phase-2b trial failed to show efficacy and the company eliminated some data from phase-3 trials.
PTC Therapeutics, Inc. (NASDAQ:PTCT) closed trading at a share price of $6.32, at the end of the March 14 session. The stock had a trade volume of 1.28 million and gained 2.76% during the session.