Experimental Treatments for Dravet Syndrome

EPX-100 is a repurposed antihistamine that is thought to be able to suppress seizures through its action on the serotonin signaling pathways, a mechanism that is different from its anti-histaminic properties. Researchers identified it in a screen of thousands of approved medications that they conducted in a zebrafish model of Dravet syndrome. A Phase 2 trial in Dravet patients is currently underway.

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. It has been tested in five Dravet patients under the FDA’s compassionate use program; all five had fewer seizures while on the experimental medication.

SPN-817 is a synthetic form of a naturally occurring compound, huperzine A, which inhibits an enzyme in the brain that metabolizes neurotransmitters. SPN-817 is able to increase the levels of a neurotransmitter called gamma-aminobutyric acid (GABA). In this way, it is thought to be able to reduce seizure activity in the brain. It is currently being tested for the treatment of focal impaired awareness seizures in a Phase 1/2 trial.