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PTC Therapeutics Announces Initiation of Global Phase 3 Clinical Trial to Evaluate Vatiquinone in Friedreich Ataxia
- Third trial initiated in 2020 from compounds identified from PTC's Bio-e platform - SOUTH PLAINFIELD, N.J., Nov. 30, 2020 /PRNewswire/ -- PTC Therapeutics,
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- Third trial initiated in 2020 from compounds identified from PTC's Bio-e platform -\n\n\nSOUTH 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). \n\"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, Inc. \"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.\" \nThe 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.\n\"FARA and the FA community are grateful to PTC Therapeutics for t...