Ataluren: Final Phase IIb data

Final data from a double-blind, international, pivotal Phase IIb trial in 174 patients showed that low-dose (10 mg/kg ataluren in the morning and at midday and 20 mg/kg in the evening) ataluren led to a 29.7 meter improvement over placebo in the mean change from baseline in 6MWD at week 48 (p=0.058). PTC said the result is consistent with the study hypothesis of a 30 meter difference as well as the average change in 6MWD observed in registration-directed trials of approved drugs for other diseases. Furthermore, PTC said that although patient variability in the study was greater than expected, it believes a 29.7 meter change in 6MWD at a p-value of 0.058 is "clinically meaningful" and plans to use the data as a basis for discussions with FDA and regulatory authorities in Europe this quarter. The partners previously reported that ataluren missed the primary endpoint of significantly improving 6MWD from baseline to week 48 vs. placebo, but did not provide a p-value (see BioCentury, March 8). ...