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PTC Therapeutics Announces Initiation of a Registration-Directed Clinical Trial to Evaluate Vatiquinone in Mitochondrial Epilepsy
- Global Phase 2/3 trial now enrolling patients with highly morbid unmet medical condition - SOUTH PLAINFIELD, N.J., Oct. 26, 2020 /PRNewswire/ -- PTC
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- Global Phase 2/3 trial now enrolling patients with highly morbid unmet medical condition -\n\n\nSOUTH PLAINFIELD, N.J., Oct. 26, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), today announced the initiation of a registration-directed Phase 2/3 clinical trial to evaluate vatiquinone (PTC743) in patients with mitochondrial epilepsy, the highly morbid condition of refractory seizures in children with inherited mitochondrial disease. It is estimated that there are approximately 11,000-13,0001 patients with mitochondrial epilepsy in the United States, European Union, Japan and Latin America. Vatiquinone, developed from PTC's Bio-e platform, is an investigational oral small molecule that inhibits 15-Lipoxygenase, a key enzyme that regulates oxidative stress and inflammation response pathways underpinning many neurological disease pathologies, including epilepsy. The U.S. Food and Drug Administration has granted PTC743 orphan drug designation and pediatric rare disease designation for mitochondrial epilepsy. \n\"We are excited to initiate this registrational clinical trial to evaluate the first indication for vatiquinone that was identified from our Bio-e platform,\" said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. \"As a devastating and highly fatal pediatric disorder with no approved treatment options, mitochondrial epilepsy represents an area of significant unmet need. Vatiquinone has been evaluated in over 500 patients to date, and in clinical studies it has demonstrated effects in resolving refractory status epilepticus, decreasing seizure frequency and seizure-related morbidity in certain patients. We look forward to advancing this compound for patients who are clearly in need of treatment.\"\nThe Phase 2/3 trial in mitochondrial epilepsy, named MIT-E, is a randomized, placebo-controlled study evaluating vatiquinone in approximately 60 patients with genetically confirmed mitochondrial disease and associated refractory epilepsy. Enrolled patients will participate in a one-month run-in phase to ensure a minimum number of observable motor seizures. Patients who meet that threshold will then be enrolled in a six-month parallel arm placebo-controlled phase. The study's primary endpoint is the reduction of observed motor seizure frequency in the placebo-controlled phase relative to the run-in ...