Pivotal Trial in Muscular Dystrophy

Posted - May 2009 in Research Highlights

Verdana-Bold;mso-ansi-language:EN-US”>PTC Therapeutics Completes Enrollment of Pivotal Trial in Muscular Dystrophy

ed note: PTC124 works on nonsense mutations in MD.  There is promise that it may also work on nonsense mutations in HSP, and these account for about 10% of all HSP.  So this trial holds significant interest for HSPers.

mso-ansi-language:EN-US”>SOUTH PLAINFIELD, NJ – February 3, 2009 – PTC Therapeutics, Inc. (PTC) today announced that it has successfully completed patient enrollment of its pivotal clinical trial of PTC124 in patients with nonsense mutation Duchenne and Becker muscular dystrophy (nmDMD/BMD). Accrual was completed two months ahead of schedule. The company also announced today that PTC124 has been issued the generic name, ataluren.

EN-US”>The one-year, multi-center, randomized, double-blind, placebo-controlled study is designed to determine whether ataluren can improve walking, muscle function and strength in patients with nmDMD/BMD. It will also evaluate ataluren’s long-term safety profile over a longer term than in previous studies. The pivotal trial is ongoing at 37 sites on four continents and in 11 countries. The study has enrolled 174 patients across the United States, Europe, Canada, Australia, and Israel.

EN-US”>"This trial represents a critical milestone for the entire DMD/BMD community – 20 years after sequencing the dystrophin gene we have the first pivotal study for a potential disease-modifying therapy," said Brenda Wong, MD, associate professor of pediatrics and neurology, Cincinnati Children’s Hospital Medical Center. "The rapid accrual is a testament to the commitment of boys with DMD/BMD and their families and the strong sense of urgency on the part of so many investigational teams around the world."

EN-US”>"Duchenne/Becker muscular dystrophy is a rare disorder and patients whose disease is due to a nonsense mutation represent only 13 percent of these cases, which created an enrollment challenge. However, the collaborative approach taken by clinical trial sites, advocacy groups, and PTC Therapeutics encouraged rapid identification of eligible candidates for this important study," said Richard Finkel, MD, attending physician, director of the neuromuscular program, Children’s Hospital of Philadelphia. "The completion of this trial enrollment is a striking example of the successes we can achieve when patients, families and the medical community come together to address an area of such high unmet medical need."

EN-US”>"Currently available therapies for nmDMD/BMD may alleviate the symptoms of this progressive debilitating disease, but none address the underlying cause of the disease." said Langdon Miller, MD, chief medical officer of PTC. "Ataluren represents a potential breakthrough as the first treatment designed to enable the formation of functioning dystrophin, the missing protein in patients with nmDMD/BMD."

EN-US”>This is the first pivotal trial for ataluren. In addition, PTC is conducting an open-label, long-term safety study in boys with nmDMD who participated in an early Phase 2a trial. PTC also plans to initiate a second pivotal clinical trial of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) in 2009.

EN-US”>The United States Adopted Names Council (USAN) in conjunction with the World Health Organization, International Nonproprietary Names Committee deemed ataluren’s mechanism of action to be significantly novel, warranting creation of a new stem, -luren, for pharmaceuticals that are inducers of ribosomal readthrough of nonsense mutation mRNA stop codons.

"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 Stuart W. Peltz, PhD, president and CEO, PTC Therapeutics. "We look forward to completing this study over the next year as we continue to work to develop additional innovative therapies for the muscular dystrophy community."