PTC Completes Enrollment of Phase 3 Clinical Trial of Translarna™ for Patients with Cystic Fibrosis

PTC Therapeutics, Inc.

today announced that it has completed enrollment of ACT CF, the company's second Phase 3 clinical trial of Translarna™ (ataluren) for patients with nonsense mutation cystic fibrosis (nmCF). Nonsense mutations in cystic fibrosis are categorized as Class I mutations, which are the most difficult to treat, as they result in little or no production of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Approximately 10 percent of cystic fibrosis patients have their disease as a result of a nonsense mutation. "We are very pleased by the enthusiastic response from our clinical investigators and strong interest from patients to participate in our ACT CF study," said Stuart Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "Based on the results from our previous Phase 3 study, we believe that Translarna has the potential to meaningfully improve lung function and decrease pulmonary exacerbations in nonsense mutation cystic fibrosis patients by targeting the underlying cause of the disease." On September 30, 2015, the European Medicines Agency (EMA) validated the submission of a variation for a new indication for Translarna for the treatment of nmCF. The company's regulatory application for Translarna in nmCF was based on clinical data and analyses generated from the company's previously completed Phase 3 double-blind, placebo-controlled study comparing Translarna to placebo in nmCF patients. Translarna received marketing authorization in Europe in August 2014 for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.