BRIEF—Another flop for PTC Therapeutics' vatiquinone

In a second flop within a month, PTC Therapeutics announced today that the MIT-E trial of vatiquinone for the treatment of mitochondrial disease associated seizures (MDAS) failed to achieve its primary endpoint of reduction in observable motor seizures.

In May, PTC revealed top-line results from the MOVE-FA Phase III trial of vatiquinone in patients with Friedreich ataxia (FA) that did not meet its primary endpoint of statistically-significant change in mFARS score at 72 weeks in the primary analysis population.

The latest study showed evidence of treatment effect in reducing seizure frequency in the overall study population and in the largest subgroup of children with Leigh syndrome, in whom benefit was also observed in the key secondary endpoints of occurrence of status epilepticus and disease-related hospitalizations.

While the MIT-E study results do not support the advancement of vatiquinone for the treatment of MDAS, PTC continues to plan to discuss the results of the MOVE-FA trial of vatiquinone for the treatment of Friedreich ataxia with regulatory authorities based on the important signals of clinical benefit on key subscales of the mFARS, such as upright stability.