EPX-200, a compound similar to an approved weight management medicine, is in early testing by Epygenix Therapeutics as a potential treatment for Dravet syndrome, a severe form of epilepsy. This compound works on serotonin signaling pathways linked to epilepsy.
How EPX-200 works
Using a zebrafish model of Dravet syndrome, researchers screened more than 3,000 compounds to identify ones that suppressed neurological symptoms associated with the disease. EPX-200 was one of the compounds identified and found to be effective in reducing seizures and convulsive behaviors in the animals.
EPX-200 is a serotonin-receptor agonist that works by modifying serotonin signaling pathways. Preclinical studies suggest that the activation of serotonin receptors called HTR2A and HTR2C have anti-epileptic effects. EPX-200 specifically activates HTR2C, suggesting this may be its mechanism of action.
Studies of EPX-200
EPX-200 has been tested off-label in five children with severe Dravet syndrome under the FDA’s compassionate use program. These children were resistant to at least five different anti-epileptic medicines and had mutations in the SCN1A gene.
All five had fewer seizures while taking the medication, according to a study published in the journal Brain in 2017. One child with previous seizures daily went seizure-free for two weeks while taking EPX-200. After a few months, the child’s seizure activity increased but remained less frequent than before starting EPX-200 treatment. No severe side effects were reported in any of the children taking EPX-200 and the medication — which can decrease appetite — was well-tolerated.
Epygenix is still evaluating EPX-200 in preclinical studies as a possible Dravet therapy.
EPX-200 was granted orphan drug status by the U.S. Food and Drug Administration as a potential treatment for Dravet syndrome in 2017. The orphan drug program promotes the development of new treatments for rare diseases.
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