PTC Therapeutics, Inc. Receives Fast Track Designation For PTC124 In The Treatment Of Duchenne Muscular Dystrophy

SOUTH PLAINFIELD, N.J., March 30 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small-molecule drugs that target post-transcriptional control processes, today announced that the company has been granted Fast Track designation from the United States Food and Drug Administration (FDA) for the development of PTC124 for the treatment of Duchenne muscular dystrophy (DMD) due to a nonsense mutation in the dystrophin gene. In December 2004, PTC124 was granted Orphan Drug designation by the FDA for the treatment of DMD. PTC124 is currently in Phase 2 clinical trials in DMD and cystic fibrosis (CF) in cases in which a nonsense mutation is the cause of the disease. PTC expects to complete these Phase 2 clinical trials in the second half of 2006.

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The Fast Track program is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Development programs receiving Fast Track designations typically receive FDA priority review (6-month vs. standard 10-month review).

"Fast Track designation in DMD is an additional important element in the development of PTC124," said Stuart W. Peltz, Ph.D., President and CEO of PTC. "DMD is an unmet medical need where only palliative options are currently available. We hope PTC124 will represent a therapeutic option for patients with DMD due to a nonsense mutation."

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 are of central importance to the regulation of the rate and timing of cellular protein production. PTC has assembled proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities. PTC's current pipeline of clinical and preclinical product candidates addresses multiple indications, including genetic disorders, oncology, and infectious diseases.

About PTC124

PTC124 is an orally delivered investigational product candidate in development for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely halt the translation process, producing a shortened, non-functional protein. In pre-clinical trials, the administration of PTC124 allowed the restoration of the production of full-length, functional proteins. PTC124 has demonstrated activity 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 readthrough of normal stop codons. Pharmacokinetic modeling of the Phase 1 results allowed development of a dosing regimen for the Phase 2 studies in cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD). It is estimated that 10% of the cases of CF and 15% of the cases of DMD 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 Fast-Track designation and 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 DMD and CF by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA). PTC124's development is supported by grants from the Muscular Dystrophy Association (MDA), Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), FDA's Office of Orphan Products Development (OOPD), and by General Clinical Research Center grants from the National Center for Research Resources (NCRR).

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children). More information regarding DMD is available through the Muscular Dystrophy Association ( ) and the Parent Project Muscular Dystrophy ( ).

Photo: NewsCom: Photo Desk, photodesk@prnewswire.comPTC Therapeutics, Inc.

CONTACT: Investors & Media: Jane Baj, +1-908-222-7000, ext. 167,, or Patients, Patients' Families, Investigators and PatientOrganizations: Kerri Donnelly, +1-908-222-7000, ext. 112,, both of PTC Therapeutics, Inc.

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