Full Press Release Details
PLYMOUTH MEETING, Pa., Sept. 03, 2026 (GLOBE NEWSWIRE) -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) today announced that it will present encore data from the company’s investigation of EPX-100 (clemizole hydrochloride) at the 16 th European Epilepsy Congress being held from September 5-9, in Athens, Greece.
The company is investigating EPX-100 for the treatment of Dravet syndrome and Lennox-Gastaut syndrome in the ongoing ARGUS and LIGHTHOUSE trials, respectively. These trials are currently enrolling, and more information can be found at argustrial.com and lighthouselgsstudy.com.
Poster presentation details are listed below:
| Abstract Title: | EPX-100 as Adjunctive Therapy in Patients with Dravet Syndrome: Preliminary Results From the Open-Label Extension Phase of the ARGUS Study | |
| Poster #: | P0342 | |
| Date/Time: | September 6, from 2:00-3:00 pm EEST |
| Abstract Title: | A Drug-Drug Interaction Study of EPX-100 (Clemizole Hydrochloride) With Clinical Probe Substrates for Selected CYP Enzymes | |
| Poster #: | P0331 | |
| Date/Time: | September 6, from 2:00-3:00 pm EEST | |
About Clemizole Hydrochloride (EPX-100) EPX-100, clemizole hydrochloride, is under development for the treatment of Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). EPX-100 acts by targeting central 5-hydroxytryptamine receptors to modulate serotonin signaling. The drug candidate is administered orally twice a day in a liquid formulation and has been developed based on a proprietary phenotype-based zebrafish drug screening platform. DS is caused by a loss of function mutation in the SCN1A gene, and scn1 mutant zebrafish replicate the genetic etiology and phenotype observed in the majority of individuals with DS. The scn1Lab mutant zebrafish model that expresses voltage gated sodium channels has been used for high-throughput screening of compounds that modulate Nav1.1 in the central nervous system.
About Dravet Syndrome Dravet syndrome (DS) is a severe and progressive epileptic encephalopathy that begins in infancy and causes significant impact on patient functioning. DS begins in the first year of life and is characterized by high seizure frequency and severity, intellectual disability, and a risk of sudden unexpected death in epilepsy. Approximately 85% of Dravet syndrome cases are caused by de novo loss-of-function (LOF) mutations in SCN1A gene, which codes for a voltage-gated sodium channel. DS has an estimated incidence rate of 1:15,700.
About Lennox-Gastaut Syndrome Lennox-Gastaut syndrome (LGS) is a rare and drug-resistant epileptic encephalopathy characterized by onset in children between 3-5 years of age. The underlying cause of LGS is unknown and can be related to a wide range of factors including genetic differences and structural differences in the brain. As a result, patients experience multiple seizure types, including atonic seizures, and developmental, cognitive, and behavioral issues. LGS affects approximately 48,000 patients in the U.S.
About Harmony Biosciences Harmony Biosciences is a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases who have unmet medical needs. Driven by novel science, visionary thinking, and a commitment to those who feel overlooked, Harmony Biosciences is nurturing a future full of therapeutic possibilities that may enable patients with rare neurological diseases to truly thrive. Established by Paragon Biosciences, LLC, in 2017 and headquartered in Plymouth Meeting, Pa., we believe that when empathy and innovation meet, a better future can begin; a vision evident in the therapeutic innovations we advance, the culture we cultivate, and the community programs we foster. For more information, please visit www.harmonybiosciences.com.