Positive data from Phase III study of PTC 124 (PTC Therapeutics) for patients with nonsense mutation Cystic Fibrosis

Results from a Phase III study of PTC 124 (ataluren), from PTC Therapeutics, in patients with nonsense mutation Cystic Fibrosis (nmCF) demonstrated positive trends in lung function as measured by FEV1 (forced expiratory volume in one second). The secondary endpoint, the number of pulmonary exacerbations in 48 weeks, also showed a positive trend in favor of ataluren, with the rate in the ataluren group being 23% lower than the placebo group. In the patients not receiving chronic inhaled antibiotics, the pulmonary exacerbation rate in the ataluren group was 43% lower than the rate in the placebo group. Safety results indicate that ataluren was generally well tolerated. The 48-week trial enrolled 238 patients, ages six years and older, at multiple sites in North America, Europe, and Israel. These data were presented by Michael Konstan, a principal investigator at University Hospitals Rainbow Babies and Children's Hospital in Cleveland, Ohio, at the European Cystic Fibrosis Society Conference in Dublin, Ireland.