PTC Therapeutics plummets 22% after study in Friedreich Ataxia misses primary endpoint

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According to the report, the study did not meet its primary endpoint of statistically significant change in mFARS score at 72 weeks in the primary analysis population.

While being disappointed that the study did not achieve its primary endpoint, CEO Matthew Klein said they are encouraged by the findings of meaningful impact on several different aspects of FA disease progression and morbidity over 72 weeks.

“Given the signals of clinical benefit, vatiquinone’s well-established safety profile in children, and the unmet medical need for pediatric patients with FA, we look forward to discussing a potential path to registration with regulatory authorities,” added Klein.