Vatiquinone (EPI-743) is an investigational, oral, first-in-class 15-lipoxygenase inhibitor being developed for the treatment of patients with Friedreich’s ataxia (FA). Here, we report long-term results of vatiquinone treatment in patients with FA from the MOVE-FA extension study (36 months) and Study EPI-2010-006 (24 months), compared with matched natural history cohorts from FACOMS (Friedreich Ataxia Clinical Outcome Measures). MOVE-FA (NCT04577352) was a global phase 3 study of vatiquinone in patients with FA aged ≥ 7 years (N = 143; mean age: 18.7 years); participants who completed MOVE-FA were eligible to rollover into the long-term extension (NCT05515536). EPI-2010-006 (NCT01728064) was a phase 2 study of vatiquinone in adult patients with FA ≥ 18 years (N = 63; mean age: 28.9 years). The pre-specified primary endpoint for these analyses was the modified Friedreich’s Ataxia Rating Scale (mFARS). After 36 months in the MOVE-FA long-term extension study, participants in the vatiquinone treatment group demonstrated a 3.75-point increase in mFARS. The matched FACOMS cohort progressed by 7.48 points over the same period. Vatiquinone treatment resulted in a 3.7-point benefit (p < 0.0001, n = 70) in mFARS relative to FACOMS, representing a clinically meaningful 50% slowing of disease progression over 3 years. Following 24-months of treatment with vatiquinone in EPI-2010-006, participants demonstrated a 0.92-point decrease in mFARS while participants in the matched FACOMS cohort progressed by 3.89 points. This resulted in a 4.8-point treatment benefit (p < 0.0001, n = 41), consistent with a 2-year delay in progression. The results of the extension studies provide further evidence of the potential benefit of vatiquinone for the treatment of FA. The pre-specified endpoints for two different long-term extension studies were met, with highly statistically significant evidence of durable treatment benefit in slowing disease progression in paediatric and adult patients.
Monday, October 6, 2025
357P Long-term vatiquinone treatment slows Friedreich’s ataxia disease progression relative to FACOMS natural history
A. Vagabov, J. Cherry, A. Duqette, M. França, S. Perlman, A. Durr, E. Bertini, K. Mathews, L. Schöls, A. Fournier, M. Delatycki, S. Subramony, R. Roxburgh, M. Rance, O. Zhang, L. Golden, J. Gruenert, C. Werner, D. Lynch, T. Zesiewicz, 357PLong-term vatiquinone treatment slows Friedreich’s ataxia disease progression relative to FACOMS natural history, Neuromuscular Disorders, Volume 53, Supplement, 2025, 106087, ISSN 0960-8966,
doi:10.1016/j.nmd.2025.106087.
