PTC Therapeutics Announces Initiation of Phase 2b Registration-Directed Clinical Trial of PTC124 in Duchenne/Becker Muscular...( First Registration Study of an Inve...),Health

First Registration Study of an Investigational Drug for Duchenne/Becker

Muscular Dystrophy

SOUTH PLAINFIELD, N.J., April 23 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), today announced the initiation of an international pivotal trial of PTC124 in patients with Duchenne/Becker muscular dystrophy (DMD/BMD) due to a nonsense mutation. The primary objective of this registration-directed Phase 2b trial is to demonstrate the efficacy of PTC124 as measured by improvements in the walking ability of patients with this progressive genetic disease.

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"DMD/BMD is a disorder with a significant need for better treatment options, and we are very encouraged by the promising results we have seen to date with PTC124," said Brenda Wong, M.D., Associate Professor of Pediatrics and Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, who was involved in the Phase 2a study and is one of the trial's lead investigators. "We believe that the safety profile of PTC124 and activity we have seen in the Phase 2a studies clearly support the initiation of this longer-term, registration-directed efficacy and safety study. We are very pleased to be a part of this groundbreaking trial."

Patients with DMD and BMD are boys and young men who lack dystrophin, a protein that is critical to the structural stability of muscle fibers. Patients develop progressive muscle weakness that leads to loss of ambulation, wheelchair dependency, and eventual decline in respiratory and cardiac function. It is estimated that one in 10 DMD patients are likely to have a Becker presentation, a milder form of the disease that is associated with later manifestation of symptoms. In essence, DMD and BMD represent a continuum of the same disease.

PTC124 is a novel, orally delivered drug in development f
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