Safety and Efficacy of Vatiquinone Treatment in Friedreich Ataxia Patients from MOVE-FA: a Phase 3, Double-blind, Placebo-controlled Trial


Clinical Trials

Poster Number: S79


David Lynch, MD, PhD, Children's Hospital of Philadelphia, Antoine Duquette, MD, MSc, FRCP(C), Centre Hospitalier de l'Universite de Montreal, Marcondes Cavalcante França Jr, MD, PhD, University of Campinas (UNICAMP), Susan Perlman, MD, University of California, Los Angeles, Alexandra Durr, MD, PhD, Sorbonne Université, Paris Brain Institute - ICM, Inserm, CNRS, APHP, Enrico Bertini, MD, Ospedale Pediatrico Bambino Gesu’ IRCCS, Alejandra Darling, MD, PhD, Hospital Sant Joan de Déu, Katherine Mathews, MD, FAAN, University of Iowa, Ludger Schöls, MD, University Hospital of Tübingen, Anne Fournier, MD, FRCPC, FACC, FHRS, FCCS, CHU Sainte-Justine, Martin Delatycki, MD, PhD, Murdoch Children's Research Institute, S H Subramony, M.D., Univsersity of Florida, Fixel Center for Neurological Disorders, Richard Roxburgh, PhD, FRACP, Neurogenetics Clinic Centre for Brain Research, University of Auckland, Auckland, NZ, Olivia Zhang, PhD, PTC Therapeutics, Inc., Christian Rummey, PhD, Clinical Data Science GmbH, Alana Salvucci, DO, PTC Therapeutics, Inc., Bert Yao, MD, PhD, MHS, PTC Therapeutics, Inc., Jonathan Cherry, PhD, PTC Therapeutics, Lee Golden, MD, PTC Therapeutics, Inc., Theresa Zesiewicz, MD, FAAN, University of South Florida

Background and Objectives: Friedreich Ataxia (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 (NCT04577352), a global phase 3 trial, evaluated the safety and efficacy of vatiquinone in patients with FA. Here, we describe the results from the 72-week placebo-controlled phase.

Methods: 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.

Conclusions: 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.