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 high
SOUTH 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. “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.” The 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 phase. Secondary endpoints include the occurrence of status epilepticus, number of hospitalizations, rescue medication use, and caregiver burden. About Vatiquinone (PTC743) About the Bio-e platform About PTC Therapeutics, Inc. For More Information: Investors: Forward-Looking Statements: PTC’s actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the eligible patient base and commercial potential of PTC’s products and product candidates; the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC’s products or product candidates that PTC commercializes or may commercialize in the future; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; PTC’s scientific approach and general development progress; and the factors discussed in the “Risk Factors” section of PTC’s most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC’s other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory or prove to be commercially successful. The forward-looking statements contained herein represent PTC’s views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. 1Gorman et al. Annals of Neurology.2015; Chevallier et al. Epilepsia. 2014; View original content:http://www.prnewswire.com/news-releases/ptc-therapeutics-announces-initiation-of-a-registration-directed-clinical-trial-to-evaluate-vatiquinone-in-mitochondrial-epilepsy-301159255.html SOURCE PTC Therapeutics | ||
Company Codes: NASDAQ-NMS:PTCT |