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PTC Therapeutics Reports Second Quarter Financial Results and Provides Corporate Update
Date:8/7/2014

SOUTH PLAINFIELD, N.J., Aug. 7, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported financial results for the second quarter ended June 30, 2014.

"This has been an exciting quarter for PTC. In May we received a positive opinion from the CHMP for marketing approval for Translarna™ (ataluren) an investigational new drug in the US, for nonsense mutation Duchenne muscular dystrophy and we recently received the approval from the European Commission granting the conditional marketing authorization. It is an honor to bring the world's first therapy for Duchenne muscular dystrophy to patients who have been waiting too long for a treatment. We are actively focused on providing Translarna to patients as quickly as possible and are working aggressively to prepare for launch across the EU," stated Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "In addition to our efforts in nmDMD, we recently initiated our confirmatory Phase 3 clinical trial in nonsense mutation cystic fibrosis and it is our goal to initiate a Phase 2 proof-of-concept study for Translarna in a new indication, MPS I caused by a nonsense mutation, later this year. We expect the second half of the year will be a transformative time at PTC, as we expand our global commercial organization focused on our mission of bringing new therapies to patients with rare and neglected disorders."

Corporate Highlights:

  • ACT DMD: The confirmatory Phase 3 ACT DMD (Ataluren Confirmatory Trial in DMD) trial of Translarna™ (ataluren) in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) is well underway. Enrollment is expected to be completed near term with initial, top-line data available in the second half of 2015.
  • ACT CF: In June, the confirmatory Phase 3 ACT CF clinical trial was initiated. This tr
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  • SOURCE PTC Therapeutics, Inc.
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