SOUTH PLAINFIELD, N.J., Jan. 19 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124(R)) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently. This trial is evaluating the best methods for measuring functional abilities in patients who have lost independent mobility. Patients with nmDBMD develop progressive muscle weakness that leads to deterioration of ambulation, wheelchair dependency, progressive loss of upper limb strength, and eventual respiratory and cardiac failure. The study, which is expected to complete enrollment rapidly, is being funded in part by a $1 million grant from the Muscular Dystrophy Association, and will involve MDA’s five-center DMD Clinical Research Network and a site in the United Kingdom.
“Given the relentlessly progressive course of DBMD and its impact on multiple organ systems, we believe patients could benefit from therapeutic interventions at all stages of the disease. This trial is an important step in understanding the best methods for evaluating new clinical treatments for these boys and young men,” stated Langdon Miller, M.D., Chief Medical Officer at PTC.
This one-year, Phase 2a study is evaluating the safety, pharmacodynamic activity, and pharmacokinetics of ataluren, an investigational new drug, while assessing the use of several outcome measures of physical, pulmonary, and cardiac function in patients with advanced disease. Approximately 30 patients are being enrolled in the trial at five sites in the United States, which are part of the MDA’s DMD Research Network, and at one site in the United Kingdom. Additional information on this study can be found on http://clinicaltrials.gov.
PTC announced in February 2009 that it has completed enrollment of a registration-directed clinical trial in patients with nonsense mutation Duchenne and Becker muscular dystrophy. The trial is a multi-center, randomized, double-blind, placebo-controlled study to determine whether ataluren can improve walking, activity, muscle function, and strength and whether the drug can safely be given for a long period of time. Results from this trial are expected to be available in the first half of 2010.
ABOUT ATALUREN (PTC124(R))
Ataluren is the first investigational new drug designed to restore the production of a functioning protein in patients with genetic disorders due to a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein, such as dystrophin in the case of nmDBMD. Ataluren is currently being investigated for use in patients with nonsense mutation Duchenne and Becker muscular dystrophy (nmDBMD), nonsense mutation cystic fibrosis (nmCF) and nonsense mutation hemophilia A and B (nmHA/nmHB). Ataluren has been granted orphan drug status for the treatment of nmDBMD and nmCF by the U.S. Food and Drug Administration (FDA) and the European Commission. The FDA has also granted ataluren Subpart E and Fast Track designation for expedited development, evaluation, and marketing. The development of ataluren has been supported by the FDA Office of Orphan Products Development, the Muscular Dystrophy Association, Parent Project Muscular Dystrophy, the Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation), and the National Center for Research Resources.
ABOUT THE MUSCULAR DYSTROPHY ASSOCIATION
Founded in 1950, the Muscular Dystrophy Association is the nation’s largest non-governmental funder of research seeking treatments and cures for more than 40 neuromuscular diseases, including muscular dystrophy, amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), Charcot-Marie-Tooth disease (CMT), and Friedreich’s ataxia (FA).
Thanks to decades of generous contributions from caring individuals, plus outstanding support received from local, regional and national sponsors, MDA is credited for its role in building the entire field of neuromuscular disease research, while simultaneously nurturing clinical care to significantly improve both quality and length of lives for those affected by neuromuscular diseases.
Photo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGOPTC Therapeutics, Inc.
