Ataluren Slows the Loss of Walking Ability in Patients with Nonsense Mutation Duchenne/Becker Muscular Dystrophy

Pivotal Data Presented at the World Muscle Society Congress Suggest

Ataluren Slows the Loss of Walking Ability in Patients with Nonsense Mutation Duchenne/Becker Muscular Dystrophy

- 29.7 meter average change in 6-minute walk distance compared to placebo at 48 weeks -
- Safety results show ataluren was generally well tolerated -
- Data to be the basis of interactions with regulatory authorities -

SOUTH PLAINFIELD, NJ – October 15, 2010 – PTC Therapeutics, Inc. today announced that final analyses of Phase 2b efficacy data suggest the investigational new drug ataluren slowed the loss of walking ability in patients with nonsense mutation dystrophinopathy, a disease continuum comprising Duchenne and Becker muscular dystrophy (nmDBMD). These data were presented at the International Congress of the World Muscle Society in Kumamoto, Japan and will be the basis of interactions with the U.S. Food and Drug Administration (FDA) and national regulatory authorities in Europe in the fourth quarter of 2010.