Investigational Friedreich Ataxia Treatment Improved Neurologic Function at 72 Weeks

People with Friedreich ataxia (FA) treated with vatiquinone (PTC743; PTC Therapeutics, South Plainfield, NJ) showed clinically meaningful and statistically significant improvements in metrics of neurologic function, relative to placebo. According to results of the phase 3 MOVE-FA clinical trial (NCT04577352) presented at the 2024 International Congress of Parkinson’s Disease and Movement Disorders, treatment with vatiquinone also was associated with clinical benefit in participants’ Upright Stability subscale (USS) score. The USS is a component of the FA Rating Scale (FARS) that is predictive of risk of loss of ambulation in younger people with FA.

MOVE-FA was a randomized, parallel-arm, double-blind, placebo-controlled study that included 143 participants with FA aged ≥7 years. Only individuals with a modified FARS (mFARS) score of 20 to 70 and the ability to ambulate ≥10 feet in 1 minute with or without assistance were included in the study population. The modified intention-to-treat (mITT) population included 123 participants (mean age, 14.6 years) who were assessed according to placebo-corrected change from baseline in mFARS at 72 weeks as a primary endpoint.

At 72 weeks, participants in the mITT population demonstrated:

• A -1.61 change in mFARS (P=.144) relative to placebo, suggesting a benefit for neurologic function
• A change in USS score of -1.26 (P=.021)
• A change in Modified Fatigue Impact Scale (MFIS) score of -5.05 (P=.025), an endpoint assessing the impact of fatigue on a person’s daily life

Additionally, treatment with vatiquinone was shown to be safe and well tolerated with no differences identified in terms of treatment-related adverse events (TAEs) between the mITT and placebo group.