Harmony Biosciences to Present New Data on EPX-100 at European Epilepsy Congress
News provided byHarmony Biosciences Holdings, Inc · 1 min read
PLYMOUTH MEETING, Pa., Sept. 03, 2026 /CourierPR/ -- Harmony Biosciences, a pharmaceutical company focused on rare neurological diseases, will present new data on its investigational drug EPX-100 (clemizole hydrochloride) at the 16th European Epilepsy Congress in Athens, Greece, from September 5 to 9. The company is conducting clinical trials of EPX-100 for Dravet syndrome and Lennox-Gastaut syndrome, with the ARGUS and LIGHTHOUSE studies, respectively.
EPX-100, in development since 2017, targets central 5-hydroxytryptamine receptors to modulate serotonin signaling. This drug candidate is being tested in an oral, twice-daily liquid formulation. The research is based on a proprietary zebrafish screening platform, which has identified the drug’s potential through high-throughput screening of compounds that modulate Nav1.1 in the central nervous system. Dravet syndrome, a severe form of epilepsy, is caused by a loss-of-function mutation in the SCN1A gene, and zebrafish models with this mutation closely mimic the genetic and phenotypic characteristics observed in Dravet syndrome patients.
The drug's efficacy is also being evaluated in the LIGHTHOUSE trial for Lennox-Gastaut syndrome, a rare and drug-resistant form of epilepsy. Lennox-Gastaut syndrome typically affects children between the ages of 3 and 5, with symptoms including multiple seizure types and developmental issues. The incidence of Lennox-Gastaut syndrome is estimated at 48,000 patients in the United States.
Harmony Biosciences, established by Paragon Biosciences, LLC, in 2017, is headquartered in Plymouth Meeting, Pennsylvania. The company’s mission is to develop and commercialize innovative therapies for patients with unmet medical needs in rare neurological diseases. Harmony Biosciences is dedicated to nurturing a future full of therapeutic possibilities that may enable patients with these diseases to thrive.
The ARGUS and LIGHTHOUSE trials are ongoing, with more information available on the company’s websites: argustrial.com and lighthouselgsstudy.com. The poster presentation details will be shared at the 16th European Epilepsy Congress, providing further insights into the progress of these clinical trials.