
Epygenix
Epygenix Therapeutics - Precision Biopharmaceutical Company.
Date | Investors | Amount | Round |
---|---|---|---|
- | investor | €0.0 | round |
* | N/A | Acquisition | |
Total Funding | 000k |
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Epygenix Therapeutics, a clinical-stage biopharmaceutical company, focuses on developing treatments for rare and severe forms of genetic epilepsy. Founded in 2016 by Hahn-Jun Lee, M.Sc., Ph.D., and Scott C. Baraban, Ph.D., the company originated from technology developed in Dr. Baraban's lab at the University of California, San Francisco (UCSF). Dr. Lee brought industry experience from his time at Columbia University and a focus on small molecule solutions for niche markets, while Dr. Baraban, a professor and neuroscientist, contributed his deep expertise in epilepsy research and the development of a novel zebrafish model for drug screening.
The company's core business revolves around identifying and advancing drug candidates to treat catastrophic childhood epilepsies like Dravet syndrome and Lennox-Gastaut syndrome (LGS), which are characterized by frequent, debilitating seizures and developmental delays. Epygenix's primary drug discovery tool is an innovative, high-throughput screening system using a zebrafish model of Dravet syndrome that replicates the human genetic mutation and pathology. This 'aquarium-to-bedside' approach enables the rapid identification of promising compounds. The business model is centered on advancing these candidates through clinical trials to gain regulatory approval and commercialization, addressing a significant unmet medical need in the orphan disease market. In April 2024, Epygenix was acquired by Harmony Biosciences for $35 million upfront, with potential milestone payments, a move designed to accelerate the development and commercialization of its pipeline.
Epygenix's lead product candidate is EPX-100 (clemizole hydrochloride), a repurposed first-generation antihistamine identified through the zebrafish screening. Its anti-seizure properties are not derived from its antihistamine function but from its ability to modulate serotonin (5HT) signaling pathways, a different mechanism from many existing antiepileptic drugs. EPX-100 is administered as an oral liquid and has been granted Orphan Drug Designation by the FDA for both Dravet syndrome and LGS. The company has advanced EPX-100 into a pivotal Phase 2/3 trial for Dravet syndrome and is preparing for a Phase 3 trial for LGS. The pipeline also includes EPX-200 (lorcaserin) and EPX-300 (trazodone), other repurposed FDA-approved drugs, as well as novel chemical entities, all targeting serotonin pathways to treat various genetic epilepsies.
Keywords: Epygenix Therapeutics, rare epilepsy treatment, Dravet syndrome, Lennox-Gastaut syndrome, genetic epilepsy, biopharmaceutical, clinical-stage, Harmony Biosciences acquisition, Hahn-Jun Lee, Scott C. Baraban, EPX-100, clemizole, serotonin agonist, zebrafish drug screening, orphan drugs, pediatric epilepsy, neurotherapeutics, antiepileptic drugs, seizure disorders, precision medicine