PTC Therapeutics announced another flop for its drug vatiquinone a little over a month after it failed to hit a primary endpoint in a separate Phase III trial.
SOUTH PLAINFIELD, N.J., June 29, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the MIT-E trial of vatiquinone for the treatment of mitochondrial disease associated seizures (MDAS) failed to achieve its primary endpoint of reduction in observable motor seizures. The study showed evidence of treatment effect in reducing seizure frequency in the overall study population and in the largest subgroup of children with Leigh syndrome, in whom benefit was also observed in the key secondary endpoints of occurrence of status epilepticus and disease-related hospitalizations.
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).
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).