Pipeline
Changing the Treatment Landscape for Rare and Intractable Genetic Epilepsies
Our six proprietary compounds were selected as part of a rigorous screening program in genetically edited, disease-specific zebrafish from Dr. Scott Baraban’s laboratory at UCSF. Additional programs in Ohtahara Syndrome and other developmental and epileptic encephalopathies are on the horizon.
CLINICAL
CANDIDATE
INDICATION
PRECLINICAL
PHASE 1
PHASE 2
PHASE 3
PATHWAY: 505 (b)(1)
Dravet Syndrome (DS)
POTENTIAL SINGLE PIVOTAL STUDY
Lennox-Gastaut Syndrome (LGS)
POTENTIAL SINGLE PIVOTAL STUDY
Ohtahara Syndrome (OS)
Developmental Epileptic Encephalopathies (DEEs)
DEEs
PATHWAY: 505 (b)(2)
DEEs
DEEs
Current Candidates
Current epilepsy drugs aren’t working for those who need them most. We’re committed to changing the treatment landscape for rare and intractable genetic epilepsies with our multi-asset pipeline of preclinically validated drug candidates. Powered by a first-of-its-kind, highly predictive, genetic screening platform that validates treatment responsiveness, our pipeline includes compounds and corresponding analogs that have demonstrated promising antiseizure activity in preclinical studies.
EPX-100
Our lead drug candidate is currently in a pivotal Phase 2 clinical trial for Dravet Syndrome (DS) and a Phase 2 trial for Lennox-Gastaut Syndrome (LGS) is planned for 2023.
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Safety profile confirmed through a full battery of preclinical IND-enabling toxicology, chronic toxicology, and pharmacology studies
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Received both Orphan Drug and Rare Pediatric Disease Designations from the FDA
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Previously approved by the FDA as a first-generation antihistamine, has an excellent safety profile.
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A dose range finding study for DS is complete
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Has three promising analogs (EPX-101, 102, and 103) that suppressed spontaneous convulsive behavior and electrographic seizures in the zebrafish disease model for DS
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EPX-100 is a potent serotonergic agonist with binding to 5HT2B, 5HT2C and 5HT2A receptors
EPX-200
This FDA-approved weight management drug modulates serotonin (5HT) signaling pathways. When tested in zebrafish disease models, it powerfully eliminated spontaneous convulsive behavior and electrographic seizures for DS. Clinical results from a small-scale trial showed that EPX-200 reduced convulsive seizure frequency and/or severity in people with DS.
EPX-300
This FDA-approved antidepressant, anti-anxiety and sleep-inducing insomnia drug acts by modulating serotonin (5HT) signaling pathways. When tested in zebrafish models for DS, it was found to eliminate spontaneous convulsive behavior and electrographic seizures. An early Proof of Concept trial in a small number of patients found similar results.