PTC Therapeutics CF Data Published in PNAS
February 4, 2008
PTC Therapeutics, Inc. today announced the publication of new preclinical data in the February 12, 2008 edition of the Proceedings of the National Academy of Sciences (PNAS) which show that PTC124, a novel drug designed to bypass nonsense mutations, was active in a preclinical model of cystic fibrosis (CF). These results support and add to research published last year in the journal Nature, which demonstrated the activity of PTC124 in a preclinical model of Duchenne muscular dystrophy (DMD). PTC124 has demonstrated pharmacodynamic proof of concept in Phase 2a clinical trials in nonsense-mutation-mediated CF and DMD.
“The preclinical and clinical data on PTC124 support our hope that this drug will be an important disease-modifying therapy for cystic fibrosis,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “This is an exciting potential new treatment for patients afflicted with nonsense-mutation-mediated CF. We look forward to the next stage of clinical development to demonstrate the benefits of this promising new investigational drug.”
Read the full press release.