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PTC Therapeutics has completed patient enrollment of a pivotal clinical trial of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy (nmDMD/BMD) two months ahead of schedule. The investigational new drug, which was formerly known by its scientific name PTC124, has been given the generic name ataluren.

The large, controlled clinical trial is designed to generate the key long-term efficacy and safety information needed to support regulatory approval by the U.S. Food and Drug Administration, the European Medicines Evaluation Agency, and other health authorities. With three sites in the United Kingdom, the trial enrolled a total of 174 patients at 37 sites in 11 countries.

The main objectives of the trial are to determine whether ataluren can improve walking, muscle function, and strength in patients with nmDMD/BMD, and whether the drug can be used safely over a long period of time. PTC expects that results from the clinical trial will be available in 2010.

“We are pleased to have completed enrollment of our first pivotal study of ataluren in boys and young men with nmDMD/BMD. To have achieved this milestone so far ahead of schedule is a tremendous achievement and we are grateful to the investigators, study coordinators, and the patients and their families for their efforts,” said Dr. Stuart Peltz, president and Chief Executive Officer of PTC Therapeutics.

“We also want to thank our advocacy partners, including Action Duchenne, for all they did to help spread the word about the trial,” said Dr. Leone Atkinson, Senior Director of Clinical Development at PTC Therapeutics. “Action Duchenne’s patient registry was invaluable in identifying potential candidates for the trial who had expressed interest in participating in clinical research.”

Although the trial will still be identified as PTC124, the nonproprietary generic name ataluren was recently assigned by the United States Names Council and the World Health Organization’s International Nonproprietary Names Committee. Both groups deemed ataluren’s mechanism of action to be significantly novel to warrant the creation of a new stem –luren. This means that future drugs that work similarly to ataluren will share the –luren stem. Like all pharmaceutical products, ataluren will also be given a brand name if the drug is approved by regulatory authorities.

Ataluren is the first investigational new drug designed to enable the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation, such as nmDMD/BMD. A nonsense mutation is a premature stop signal in the genetic code that results in an interruption in the production of an essential protein.

All patients with Duchenne and Becker muscular dystrophy lack adequate levels of the dystrophin protein, which is needed to maintain muscles throughout the body including the heart. For patients with nmDMD/BMD, ataluren has the potential to address the underlying cause of the disease by enabling the cellular machinery to override the premature stop signal in the genetic code and produce a fully functioning dystrophin protein.

Patients and families interested in receiving periodic e-mail updates from PTC Therapeutics about ataluren can sign up via the “contact us” section of the PTC Web site at linktext. Additional information about ataluren is also available at linktext