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Epygenix Therapeutics

Paramus or Englewood Cliffs*, New Jersey, United Statesorpha.net ↗

Ownership: Private
Development stage: Phase 3

Overview

Epygenix advanced several small molecule candidates for rare genetic epilepsies into late-stage clinical development. Its lead compound, EPX‑100 (clemizole hydrochloride), demonstrated safety in Phase I and was positioned as a potential best-in-class therapy for Dravet syndrome after successful preclinical and early clinical studies. The company’s approach leverages unique animal models to identify both repurposed drugs and novel compounds targeting serotonin pathways—differentiating from standard GABAergic or ion channel therapies.

Therapeutic areas: Neurology
Modalities: Small moleculesRepurposed drugsNovel chemical entities
Industry: Biotech

Drug pipeline

2 assets

clemizole hydrochloride

Phase 3

Indications: Dravet syndrome, Lennox-Gastaut syndrome

1 more asset in the full pipeline.

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Partnerships

University of California San Francisco/UCSF lab of Prof. Scott C. Baraban, PhD [collaborative technology originator].

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