PTC Therapeutics announced the initiation of the registration-directed Phase 3 MOVE-FA study evaluating vatiquinone (PTC743) in children and young adults with Friedreich ataxia (FA). FA is a genetic, progressive, neurodegenerative movement disorder, typically diagnosed in childhood or adolescence. PTC estimates that there are 25,000 patients with FA worldwide and there are currently no approved disease modifying therapies for FA. In a previous Phase 2 trial, vatiquinone demonstrated a statistically significant effect on disease severity at 24 months relative to age and stage-matched natural history controls as assessed by the validated Friedreich ataxia rating scale (FARS) score and a favorable safety profile. Vatiquinone has been granted Orphan Drug Designation and Fast Track Designation for Friedreich ataxia by the U.S. Food and Drug Administration (FDA).

"The initiation of another pivotal trial this year is a major milestone for PTC," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "This is the third clinical trial initiated this year based on compounds using our Bio-e platform. Vatiquinone is an exciting orally bioavailable small molecule that targets 15-lipoxygenase, the key enzyme regulating signaling pathways that control neuroinflammation and oxidative stress. Both pathways are important players in determining the pathology and disease progression seen in Friedreich ataxia patients. Previous clinical trial results give us confidence in vatiquinone's potential in treating patients living with this devastating disease."

The Phase 3 MOVE-FA trial is an 18-month parallel arm, placebo-controlled study evaluating vatiquinone versus placebo in approximately 110 children and young adults with FA. The primary endpoint is the change from baseline in the modified Friedreich ataxia rating scale (mFARS), with key secondary endpoints assessing ambulation and activities of daily living. This endpoint strategy was developed in consultation with both the FDA and European Medicines Agency. The study will include sites in the U.S., E.U., Australia and Latin America.

"FARA and the FA community are grateful to PTC Therapeutics for their commitment to advancing treatments for Friedreich ataxia," said Jennifer Farmer, Chief Executive Officer, FARA. "We are excited for the PTC743 program to reach this milestone of opening enrollment of the MOVE-FA clinical trial and we look forward to assisting PTC with recruitment in the US and internationally."

Vatiquinone is an investigational small molecule that inhibits 15-Lipoxygenase, an enzyme that is a key regulator of the oxidative stress and inflammation response pathways that underpin neurological disease pathology. Vatiquinone has been evaluated in a number of clinical studies in which it has been demonstrated to have an impact on mortality risk and a number of neurological and neuromuscular disease symptoms. In more than 500 patients with duration of exposure up to 10 years, vatiquinone has demonstrated a favorable safety profile.