PTC Therapeutics has reported that the MOVE-FA trial of vatiquinone failed to meet its primary endpoint of a statistically significant change in the modified Friedreich Ataxia Rating Scale (mFARS) score at 72 weeks.
The mFARS measures disease progression of upper and lower limb coordination, swallowing and speech, and upright stability.
The placebo-controlled, randomised, registration-directed study enrolled 146 pediatric and adult patients aged under 18 years.
The study in Friedreich Ataxia patients, however, did demonstrate significant benefits regarding bulbar and upright stability subscales and secondary endpoints.
A statistically significant difference was also observed in the Modified Fatigue Scale.
The mean placebo corrected change in the mFARS score in the primary analysis population was 1.6 (p=0.14).
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Vatiquinone was also well tolerated throughout the study.
In subjects who completed the study protocol, significance was reached in the mFARS endpoint and several secondary endpoints.
PTC Therapeutics CEO Matthew Klein said: “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.
“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.”
The company intends to enrol patients who completed the placebo portion of the trial in its open label, 24-week extension study.