- Third trial initiated in 2020 from compounds identified from PTC's Bio-e platform -
SOUTH PLAINFIELD, N.J., Nov. 30, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), today 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.1 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).
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