Phase III trial of Translarna for Cystic Fibrosis published in Lancet Resp Med - PTC Therapeutics

PTC Therapeutics has announced that the results of a Phase III study of Translarna (ataluren) in patients with nonsense mutation Cystic Fibrosis (nmCF) were published in Lancet Respiratory Medicine. The results demonstrated positive trends in both the primary endpoint, lung function as measured by relative change in % predicted FEV1 and in the secondary outcome measure, rate of pulmonary exacerbations. There were 23% fewer pulmonary exacerbations in the ataluren group compared to placebo.

Further results from a post hoc analysis of the subgroup of patients not receiving chronic inhaled tobramycin showed a 5.7% difference in relative change from baseline in % predicted FEV1 favoring ataluren, with a mean change from baseline of -0.7% in the ataluren arm, and �6.4% in the placebo arm. In addition, there were 40% fewer exacerbations in ataluren-treated patients in this subgroup.

The collective data from this trial, including retrospective and subgroup analyses support the conclusion that ataluren was active and showed clinically meaningful improvements over placebo in these trials. See: "Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial." Eitan Kerem et al. The Lancet Respiratory Medicine, Early Online Publication, 16 May 2014 doi:10.1016/S2213-2600(14)70100-6