PTC Therapeutics has announced that the MOVE-FA trial of vatiquinone in patients with Friedreich ataxia (FA) has failed to reach its primary endpoint of significant changes in the modified Friedreich Ataxia Rating Scale (mFARS) score.

However, the results, reported via news release, did demonstrate significant benefits on several disease subscales and secondary outcome measures.

“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 Matthew B. Klein, MD, chief executive officer, PTC Therapeutics. “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.”

One hundred forty-six pediatric and adult patients with FA, mostly under 18 years of age, were enrolled in the phase 3 randomized, placebo-controlled 72-week trial of vatiquinone. The primary outcome measure was change in mFARS score, and the main secondary outcome was change from baseline in activities of daily living.

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Vatiquinone is a selective inhibitor of 15-lipoxygenase, an enzyme that regulates the oxidative stress and energy pathways affected in FA. 15-Lipoxygenase inhibition prevents ferroptosis and improves neuron survival by alleviating the consequences of mitochondrial dysfunction and oxidative stress. Previous studies have shown vatiquinone to positively impact mortality risk and various neurological disease symptoms.

Although the MOVE-FA trial did not achieve its primary endpoint, the results did find significant benefit in the bulbar and upright stability subscales of the mFARS, as well as a significant change in the Modified Fatigue Scale. Compared with placebo, vatiquinone demonstrated a 75% slowing of disease progression over the 72-week study period.

Vatiquinone was also shown to be well-tolerated by patients, similar to findings of other pediatric studies on the therapy.  


PTC Therapeutics’ MOVE-FA trial fails to meet primary endpoint. News release. PTC Therapeutics; May 23, 2023.