Has ataluren been tested in clinical trials?
Yes. We have completed a small Phase 2a trial in nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) as well as a Phase 2b trial. The latter was a randomized, double-blind, placebo-controlled trial designed to evaluate the safety and efficacy of 48 weeks of ataluren therapy. The study enrolled 174 participants at 37 sites in North America, Europe, Australia, and Israel. PTC has also conducted four Phase 2 trials in patients with nmCF in the U.S., Israel, France, and Belgium. In late 2010 we completed enrollment of a large Phase 3 clinical trial in nonsense mutation cystic fibrosis, which will be on-going through 2011 at sites in 11 countries.
