PTC Therapeutics provides update on vatiquinone Friedreich ataxia program

PTC Therapeutics provided today several positive updates on the vatiquinone Friedreich ataxia – FA – program. The pre-specified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression. In addition, PTC recently aligned with FDA on key aspects of the planned NDA submission for vatiquinone. Analysis of the MOVE-FA long-term extension study demonstrated that 144 weeks of vatiquinone treatment resulted in a 3.7-point benefit on the modified Friedreich Ataxia Rating Scale relative to a matched natural history cohort. This treatment difference on the primary endpoint represents a clinically meaningful 50% slowing in disease progression over 3 years. These results confirm that the slowing of disease progression recorded in the 72-week placebo-controlled MOVE-FA trial are maintained over 144 weeks of treatment. In addition, vatiquinone continued to be safe and well tolerated without any treatment-related serious adverse events reported. Following 24-months of treatment with vatiquinone, subjects had a 4.8-point benefit on the mFARS relative to a matched natural history population. PTC plans to submit the vatiquinone NDA in December.