Business
Harmony Biosciences Presents Clinically Meaningful Open-Label Extension Study Effectiveness Data for EPX-100 in Dravet Syndrome
Data presented at the 2025 American Epilepsy Society Annual Meeting PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)-- Harmony Biosciences Holdings, Inc. (Nasdaq:
About this update from Harmony Biosciences Holdings, Inc.
[{"type":"text","content":"\nData presented at the 2025 American Epilepsy Society Annual Meeting\n\n PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)--\nHarmony Biosciences Holdings, Inc. (Nasdaq: HRMY), today announced the presentation of initial open-label extension (OLE) data from the company’s ongoing Phase 3 ARGUS trial investigating EPX-100 (clemizole hydrochloride) for the treatment of Dravet syndrome (DS), which showed clinically meaningful reductions in seizure activity in participants with DS along with a favorable benefit-risk profile.\n\nThe data from poster #3.353 will be presented at the American Epilepsy Society (AES) Annual Meeting in Atlanta, Ga., on Monday, December 8, from 12:00 pm – 1:45 pm ET.\n\nData from the eighteen participants in the OLE trial with at least six months’ exposure to EPX-100 demonstrated the following:\n\n\nA median reduction of approximately 50% in countable motor seizure frequency per 28 days (CMS-28)\n\n\n50% of these participants achieved at least a 50% reduction in CMS-28\n\n\nEPX-100 was generally well-tolerated in participants receiving treatment for more than two years and approaching three years in the OLE phase\n\n\nThe most common treatment emergent adverse events (> 5%) were seizures, pyrexia and upper respiratory tract infection\n\n\nThere were no significant gastrointestinal adverse events (2%) and no additional laboratory testing or special monitoring is being performed in the trial\n\n\nThese data suggest a positive emerging benefit-risk profile for EPX-100, supported by clinically meaningful reduction in seizure frequency and a favorable safety/tolerability profile.\n\n“The ARGUS trial is one of the most advanced development programs in the 5-HT2 (serotonin) agonist class and the effectiveness, safety and tolerability data of EPX-100 dosed BID from the open-label extension study are very encouraging,” said Kumar Budur, MD, MS, Chief Medical and Scientific Officer at Harmony Biosciences. “These initial results support the advancement of our epilepsy franchise as we progress toward the topline data readout from the ARGUS trial in 2026.”\n\nEPX-100 is being evaluated as an investigational treatment for DS in the Phase 3 ARGUS trial, and for Lennox-Gastaut syndrome in the Phase 3 LIGHTHOUSE trial in global, multicenter, randomized, double-blind, placebo-controlled clinical trials to assess its safety and efficacy...