Harmony Biosciences Presents Clinically Meaningful Open-Label Extension Study Effectiveness Data for EPX-100 in Dravet Syndrome

Company Overview - Harmony Biosciences Holdings, Inc. is focused on developing and commercializing innovative therapies for patients with rare neurological diseases, particularly those with unmet medical needs [10] - The company was established in 2017 and is headquartered in Plymouth Meeting, Pennsylvania [10] Product Development - The company is conducting a Phase 3 ARGUS trial for EPX-100 (clemizole hydrochloride) aimed at treating Dravet syndrome, showing clinically meaningful reductions in seizure activity [1][3] - EPX-100 is also being evaluated in the Phase 3 LIGHTHOUSE trial for Lennox-Gastaut syndrome, both trials being global, multicenter, randomized, double-blind, placebo-controlled studies [3] Clinical Trial Results - Initial open-label extension data from the ARGUS trial indicated a median reduction of approximately 50% in countable motor seizure frequency per 28 days, with 50% of participants achieving at least a 50% reduction [8] - The treatment was generally well-tolerated, with the most common adverse events being seizures, pyrexia, and upper respiratory tract infections, while gastrointestinal adverse events were minimal [8] Future Outlook - The company anticipates advancing its epilepsy franchise and is progressing towards topline data readout from the ARGUS trial expected in 2026 [3]