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, which has had a long and often difficult passage in the clinic.

Just a few weeks ago PTC put out the news that a Phase III study of ataluren in CF patients registered "positive trends" among patients taking the drug. At week 48 in the study patients taking ataluren (formerly PTC124) saw a measure of lung function decline 2.5% compared to 5.5% among the placebo arm. Ataluren--which aims at creating a functional protein to can correct these rare genetic disorders caused by nonsense mutations--has been awarded orphan drug status.

After reviewing its pipeline deals, Genzyme announced last fall that it was restructuring its partnership on ataluren, walking away from its North American rights on DMD while gaining an option on commercializing the treatment for other conditions. About two years ago the drug failed a Phase IIb, which measured improvements in 6-minute walking distances. Encouraged by the positive trends seen in that study, PTC has maintained its unwavering conviction that it has a viable treatment on its hands. And it has maintained longstanding partnerships with other companies.

Evidently long-term investors such as Credit Suisse First Boston Equity Partners, HBM BioVentures, Vulcan Ventures, Celgene, Delphi Ventures, The Column Group, and Novo A/S all agreed on ataluren's potential. They put up the new round in PTC, which has also benefited from millions of dollars in government grants through the years.

- here's the press release