Objective: Describe the results of vatiquinone on the Upright Stability Subscale (USS) from MOVE-FA (NCT04577352), the 72-week placebo-controlled phase 3 study of patients with Friedreich Ataxia (FA).

Background: FA is characterized by progressive neurological damage and loss of ambulation. Vatiquinone is an oral, first-in-class inhibitor of 15-lipoxygenase. MOVE-FA, a global phase 3 trial, evaluated vatiquinone safety and efficacy in patients with FA. The USS, or FARS E, is the axial component of the modified FA Rating Scale (mFARS) that assesses functions related to balance, stance, and mobility. Analysis of natural history data shows that disease progression in ambulatory pediatric and adolescent FA patients is primarily driven by declines in the functions assessed in the USS.

Method: The study enrolled 143 subjects with FA aged ≥7 years, mFARS score of 20–70, and the ability to ambulate ≥10 feet in 1 minute. The primary endpoint was placebo corrected change from baseline in mFARS at 72-weeks. The Intent-to-Treat (ITT) population had a mean age of 18.7 and the primary analysis population modified ITT (mITT) included 123 subjects 7-21 years (mean 14.6). USS was collected as part of mFARS, the primary endpoint.

Results: In the placebo population, of the four subscales of mFARS, USS is the only one to demonstrate progression from baseline to week 72, consistent with natural history data. Significant benefit was recorded in USS (-1.26 [p=0.021]) in the mITT population. Vatiquinone treatment also delayed the loss of functional milestones represented by individual items within the USS, specifically items E2B (feet apart eyes closed) and E3A (feet together eyes open). Comparison of rate of disease progression in USS predicted a 42% reduction in disease progression per year in the vatiquinone-treated group.

Conclusion: Vatiquinone treatment resulted in clinically meaningful and statistically significant treatment effects on the USS, a sensitive and predictive endpoint for risk of loss of ambulation, prevention of which is a key goal for therapy in ambulatory FA patients.

Previously presented at Muscular Dystrophy Association (MDA) on March 3, 2024.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/improvement-in-upright-stability-subscale-of-mfars-with-vatiquinone-treatment-in-move-fa-a-phase-3-double-blind-placebo-controlled-trial/