PTC Therapeutics initiates additional clinical trial of ataluren in boys and young men with nmDBMD

PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124®) 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.  

"The Muscular Dystrophy Association is proud to provide $1 million in funding towards this ground-breaking study in DBMD patients who have lost the ability to walk," said Valerie Cwik, M.D., Executive Vice President -- Research and Medical Director, Muscular Dystrophy Association.  "This trial underscores the importance of MDA's translational research effort.  In April 2005, MDA awarded an initial $1.5 million grant to PTC in support of early clinical development of ataluren in nmDBMD.  Now we have a trial that will provide valuable information on older DBMD boys and young men that will assist in the design of future clinical trials."

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

"We are eager to participate in this innovative study of non-ambulatory DMD patients," stated John W. Day, M.D., Ph.D., principal investigator at the University of Minnesota's Paul and Sheila Wellstone Muscular Dystrophy Center.  "This precedent-setting trial is an important extension of current studies of younger, ambulatory boys with DMD, and will help define methods for assessing heart, lung and muscle function in patients with more advanced disease as we all continue to work to halt its progressive and debilitating effects on all affected individuals."

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.

SOURCE PTC Therapeutics, Inc.


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