Objective: Describe the results of vatiquinone from MOVE-FA (NCT04577352), the 72-week placebo-controlled phase 3 study of patients with Friedreich Ataxia (FA).
Background: FA, the most common inherited ataxia, 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 the safety and efficacy of vatiquinone in patients with FA.
Method: The study enrolled 143 subjects with FA aged ≥7 years, modified FA Rating Scale (mFARS) score of 20–70, and the ability to ambulate ≥10 feet in 1 minute +/- assistance. 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 years and the primary analysis population (modified ITT; mITT) included 123 subjects 7-21 years (mean 14.6).
Results: In the mITT population, there was a -1.61 (p=0.144) change in mFARS at 72-weeks relative to placebo. A consistent vatiquinone treatment benefit was observed across the primary, secondary, and exploratory endpoints. Notably, there were nominally significant benefits recorded in the Upright Stability subscale (USS) of mFARS (-1.26 [p=0.021]), a relevant metric of disease progression in younger, ambulatory FA patients, and the Modified Fatigue Impact Scale (MFIS), -5.05 (p=0.025).
Vatiquinone was safe and well tolerated and there was no difference in treatment related AEs between treatment and placebo groups.
Conclusion: MOVE-FA demonstrated clinically relevant benefits across the primary, secondary, and exploratory endpoints. Vatiquinone treatment resulted in a clinically meaningful and statistically significant treatment effect 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 6, 2024.
To cite this abstract in AMA style:
D. Lynch, A. Duquette, M. França Jr, S. Perlman, A. Durr, E. Bertini, A. Darling, K. Mathews, L. Schöls, A. Fournier, M. Delatycki, S. Subramony, R. Roxburgh, O. Zhang, C. Rummey, A. Salvucci, B. Yao, J. Cherry, L. Golden, T. Zesiewicz. Safety and Efficacy of Vatiquinone Treatment in Friedreich Ataxia Patients from MOVE-FA: a Phase 3, Double-blind, Placebo-controlled Trial [abstract]. Mov Disord. 2024; 39 (suppl 1). https://www.mdsabstracts.org/abstract/safety-and-efficacy-of-vatiquinone-treatment-in-friedreich-ataxia-patients-from-move-fa-a-phase-3-double-blind-placebo-controlled-trial/. Accessed June 14, 2025.« Back to 2024 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/safety-and-efficacy-of-vatiquinone-treatment-in-friedreich-ataxia-patients-from-move-fa-a-phase-3-double-blind-placebo-controlled-trial/