Healthcare Industry News:  Duchenne Muscular Dystrophy 

Biopharmaceuticals FDA

 News Release - October 19, 2006

FDA Awards PTC Therapeutics Orphan Products Development Grant for the Development of PTC124 in Duchenne Muscular Dystrophy

Preliminary Data to be Presented at Parent Project UK Muscular Dystrophy Conference

SOUTH PLAINFIELD, N.J., Oct. 19 (HSMN NewsFeed) -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of small-molecule drugs targeting post-transcriptional control mechanisms, today announced it has been awarded a two-year grant from the Office of Orphan Products Development (OOPD) Grant Program of the U.S. Food and Drug Administration (FDA). The grant will support the patient-related costs of the company's ongoing U.S.-based Phase 2 clinical trial of PTC124 for the treatment of nonsense-mutation-mediated Duchenne Muscular Dystrophy (DMD).

"It is an honor to receive this grant from the FDA in support of our clinical development efforts," stated Stuart Peltz, Ph.D., President and CEO of PTC Therapeutics. "Last year, PTC was awarded a grant from the FDA for the development of PTC124 as a potential treatment for cystic fibrosis (CF), which we have used to advance our Phase 2 CF program. Similarly, this new award will support the development of PTC124 as a potential treatment for DMD."

PTC initiated the Phase 2 DMD clinical trial of PTC124 in January 2006. Patients with DMD lack dystrophin, a protein that is critical to the structural stability of muscle fibers. This Phase 2 multi-site, open-label, dose-ranging clinical trial is evaluating muscle dystrophin expression in patients with nonsense-mutation-mediated DMD. Blood levels of muscle-derived creatine kinase are being measured as assessments of muscle integrity. PTC124 safety, compliance, and pharmacokinetics are also being evaluated.

Preliminary data from this study will be presented at the Parent Project UK Muscular Dystrophy 4th International DMD conference on Saturday, October 21st by Dr. Richard Finkel, Director of the Neuromuscular Program at The Children's Hospital of Pennsylvania and one of the lead investigators of the PTC DMD trial. The conference will be held at the Institute of Education in London, England, October 21st and 22nd, 2006.

About The Office of Orphan Products Development

The Office of Orphan Products Development (OOPD) has been dedicated to promoting the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions since it was created in 1982. OOPD interacts with the medical and research communities, professional organizations, academia, and the pharmaceutical industry, as well as rare disease groups. The OOPD administers the major provisions of the Orphan Drug Act (ODA) which provide incentives for sponsors to develop products for rare diseases. The ODA has been very successful -- more than 200 drugs and biological products for rare diseases have been brought to market since 1983. In contrast, the decade prior to 1983 saw fewer than ten such products come to market. In addition, the OOPD administers the Orphan Products Grants Program which provides funding for clinical research in rare diseases.

About PTC124

PTC124 is an orally delivered investigational product candidate in Phase 2 clinical 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. PTC124 has demonstrated activity in preclinical genetic disease models harboring nonsense mutations allowing the restoration of the production of full-length, functional proteins. 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. PTC is currently conducting Phase 2 clinical trials of PTC124 in nonsense-mutation- mediated cystic fibrosis (CF) and Duchenne Muscular Dystrophy (DMD).

It is estimated that 10% of the cases of CF and 13% 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 designations 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.

About PTC Therapeutics, Inc.

PTC is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. 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.

Source: PTC Therapeutics

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