Vatiquinone Treatment Shows Benefit Across End Points for Friedreich Ataxia in Phase 3 MOVE-FA Trial


A new analysis of phase 3 MOVE-FA trial presented at MDA 2024 revealed promising outcomes for vatiquinone, an oral 15-lipoxygenase inhibitor, in patients with Friedreich ataxia.

David Lynch, MD, PhD, neurologist and director of the Friedreich's Ataxia Program at Children's Hospital of Philadelphia

David Lynch, MD, PhD

Credit: Children's Hospital of Philadelphia

New data from the placebo-controlled phase of the global phase 3 MOVE-FA trial (NCT04577352) assessing vatiquinone (PTC Therapeutics), an oral first-in-class inhibitor of 15-lipoxygenase, in patients with Friedreich ataxia (FA) showed the treatment demonstrated clinically relevant benefits across multiple end points at 72 weeks.1

In the analysis, investigators observed a –1.61 (P=.144) change in modified FA Rating Scale (mFARS)compared with the placebo at 72 weeks in the modified intent-to-treat (mITT) population, which consisted of 123 participants with FA. The researchers reported a sustained vatiquinone treatment benefit observed across all end points including the primary, secondary, and exploratory end points.

Top Clinical Takeaways

  • Vatiquinoneshowcased a promising trend in improving the modified FA Rating Scale over 72 weeks, though this was not statistically significant.
  • Significant benefits were recorded, however, in the Upright Stability subscale and the Modified Fatigue Impact Scale.
  • The MOVE-FA trial, presented at the 2024 MDA Conference, highlighted vatiquinone's safety and tolerability overall.

Presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 3-6, in Orlando, Florida, by lead author David Lynch, MD, PhD, a neurologist and director of the Friedreich's Ataxia Program at Children's Hospital of Philadelphia, and colleagues, the trial included patients 143 aged at least 7 years, with an mFARS score of between 20 and 70 and the ability to ambulate at least 10 feet in 1 minute with or without assistance. The primary end point of the current analysis 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—the mITT population—included participants between 7 years and 21 years of age (mean, 14.6).

READ MORE: Interim Real-World Data From Phase 4 EVOLVE Study Demonstrate Sustained Treatment Effect of Eteplirsen

Investigators observed a nominally significant benefit recorded in the Upright Stability subscale (USS), a relevant metric of disease progression in younger ambulatory patients with FA, of mFARS, with a change of –1.26 (P = .021). In addition, researchers observed nominally significant benefit in the Modified Fatigue Impact Scale, with a change of –5.05 (P = .025). Overall, the authors noted that the treatment was safe and well tolerated among the participants, with no differences in treatment-related adverse events observed between treatment and placebo groups.

In an additional analysis of MOVE-FA presented at MDA 2024 by Lynch and colleagues, findings showed vatiquinone treatment had a clinically meaningful and statistically significant treatment effect on USS progression, which is a key goal for therapy in ambulatory FA patients. Of the 4 subscales of mFARS, USS was the only which displayed progression from baseline to week 72 in the placebo population. This finding is consistent with natural history data that shows that disease progression in ambulatory pediatric and adolescent patients, which is primarily driven by declines in the functions assessed in the USS.

All told, significant benefit was recorded in USS, with a change of –1.26 in the mITT population (P = .021) and vatiquinone treatment delayed the loss of functional milestones represented by individual items in the USS, particularly items E2B (feet apart, eyes closed) and E3A (feet together, eyes open). Notably, findings showed that a comparison of the rate of disease progression on USS predicted a 42% reduction in disease progression per year in those treated with vatiquinone.

Prior to these MOVE-FA data, vatiquinone previously demonstrated a statistically significant effect on disease severity in a phase 2 trial (NCT01962363). The small-scale study, which featured 4 individuals with FA, showed that after 6 months of treatment, the total FARS score improved by an average of 9%. While all subscales improved, bulbar and upper limb coordination subscales were most improved, by 80% and 53%, respectively. Mean improvements persisted at 18 months from baseline, although these findings were attenuated.3

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1. Lynch D, Duquette A, França MC, et al. Safety and Efficacy of Vatiquinone Treatment in Friedreich Ataxia Patients from MOVE-FA: a Phase 3, Double-blind, Placebo-controlled Trial. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6; Poster S79.
2. Lynch D, Duquette A, França MC, et al. Improvement in Upright Stability subscale of mFARS with Vatiquinone Treatment in MOVE-FA: a Phase 3, Double-blind, Placebo-controlled Trial. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6; Poster S80.
3. Sullivan K, Freeman M, Shaw J, et al. EPI-743 for Friedreichs ataxia patients with point mutations. Neurology. 2016;86(16 supplement).
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