PTC Therapeutics' vatiquinone misses Phase III endpoint

PTC Therapeutics’ (Nasdaq: PTCT) stock plummeted 19.6%, ending the regular session at $46.95, after it 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.

However, vatiquinone treatment did demonstrate significant benefit on key disease subscales and secondary endpoints. In addition, in the population of subjects that completed the study protocol, significance was reached in the mFARS endpoint and several secondary endpoints.

"While we are disappointed that the study did not achieve its primary endpoint, we are encouraged by the findings of meaningful impact on several different aspects of FA disease progression and morbidity over 72 weeks," said Dr Matthew Klein, chief executive of PTC Therapeutics.