Harmony Biosciences to Present Open-Label Extension Data From Phase 3 ARGUS Trial at the 2026 American Academy of Neurology Annual Meeting
Poster presentation details are listed below:
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Abstract Title: |
ARGUS: A Study of EPX-100 (Clemizole Hydrochloride) as Adjunctive Therapy in Participants with Dravet Syndrome (DS): Preliminary Results from the Open-label Extension Phase |
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Poster #: |
P10.010 |
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Date/Time: |
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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
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Harmony Biosciences Investor Contact:
484-566-3685
bdoyle@harmonybiosciences.com
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202-641-6086
cmccanless@harmonybiosciences.com
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