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PTC Therapeutics Announces Initiation of Phase 2b Registration-Directed Clinical Trial of PTC124 in Duchenne/Becker Muscular Dystrophy
Date:4/23/2008

, producing a shortened, non- functional protein. PTC124 has restored production of full-length, functional proteins in preclinical genetic disease models harboring nonsense mutations. In Phase 1 clinical trials, PTC124 was generally well tolerated, achieved target plasma concentrations that have been associated with activity in preclinical models and did not induce ribosomal read through of normal stop codons. PTC124 has demonstrated pharmacodynamic proof of concept in Phase 2a clinical trials in nonsense-mutation-mediated Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF).

It is estimated that 13% of the cases of DMD and 10% of the cases of CF are due to nonsense mutations. PTC believes that PTC124 is potentially applicable to a broad range of other genetic disorders in which a nonsense mutation is the cause of the disease. The FDA has granted PTC124 Subpart E designation for expedited development, evaluation, and marketing and has granted Orphan Drug designations for the treatment of CF and DMD due to nonsense mutations. PTC124 has also been granted orphan drug status for the treatment of CF and DMD by the European Commission. PTC124's development has been supported by grants from the Muscular Dystrophy Association (MDA), Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), Parent Project Muscular Dystrophy (PPMD), FDA's Office of Orphan Products Development (OOPD) and by General Clinical Research Center grants from the National Center for Research Resources (NCRR). For additional information on the PTC124 clinical trial, please visit http://www.clinicaltrials.gov and search using the keyword: PTC124.

About PTC Therapeutics, Inc.

PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary, small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulat
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SOURCE PTC Therapeutics, Inc.
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