The grant will support the patient-related costs of the company’s ongoing phase II clinical trial of PTC124. PTC initiated the phase II clinical trial of PTC124 in January 2006.
Patients with Duchenne muscular dystrophy (DMD) lack dystrophin, a protein that is critical to the structural stability of muscle fibers. This phase II trial is evaluating muscle dystrophin expression in patients with nonsense-mutation-mediated DMD. PTC124 safety, compliance, and pharmacokinetics are also being evaluated.
Last year PTC was awarded a grant from the FDA for the development of cystic fibrosis in support of a phase II study.
PTC’s current pipeline of clinical and preclinical product candidates addresses multiple indications, including genetic disorders, oncology, and infectious diseases.