Researchers are working on a number of experimental treatments for Dravet syndrome and to more effectively control seizures.
Ataluren is an experimental therapy that PTC Therapeutics is developing to treat Dravet syndrome. This medication is already marketed under the brand name Translarna to treat Duchenne muscular dystrophy within the European Union, Iceland, Liechtenstein, Norway, Israel, and South Korea.
Ataluren works by causing the machinery that makes protein to “read-through” gene mutations that would lead to the production of a faulty protein. By helping overcome these errors, the medications may be able to reduce the symptoms of Dravet syndrome.
An ongoing Phase 2 clinical trial (NCT02758626) is testing the safety and effectiveness of Ataluren in 16 patients with drug-resistant Dravet syndrome. Researchers expect to conclude the study in 2022.
EPX-100 was used as an antihistamine in the 1950s and 1960s. Researchers identified it in a screen of thousands of approved medications that they conducted in a zebrafish model of Dravet syndrome. They don’t fully understand the exact mechanism of how the compound works in treating Dravet syndrome. Epygenix Therapeutics is developing EPX-100.
A placebo-controlled, double-blind, two-part Phase 1 clinical trial (NCT04069689) tested the safety and pharmacokinetics (a drug’s movement in the body) of oral EPX-100 in 24 healthy volunteers. Epygenix announced in January 2020 that the study results showed EPX-100 was safe and that both male and female volunteers tolerated it well. The company is preparing Phase 2 efficacy studies to test EPX-100 in patients with Dravet syndrome in the U.S. and Australia.
EPX-200 is a weight-loss compound that researchers identified in the same screen as EPX-100. Epygenix is testing the medication in preclinical studies.
Researchers also tested the experimental treatment off-label in five children with severe Dravet syndrome under the U.S. Food and Drug Administration (FDA) compassionate use program. The study showed that all five patients had fewer seizures while taking the compound. Researchers reported no severe side effects.
EPX-300 is another compound that researchers identified in the same screen as EPX-100 and EPX-200. The medication is approved to treat insomnia, depression, and anxiety. Epygenix is developing the EPX-300 to treat Dravet syndrome, which the FDA granted orphan drug designation.
Researchers do not yet know the mechanism of how EPX-300 may benefit Dravet syndrome.
Soticlestat (previously TAK-935 or OV935) is an experimental therapy that Takeda Pharmaceuticals is developing in collaboration with Ovid Therapeutics to treat rare developmental and epileptic encephalopathies (DEE), including Dravet syndrome. The FDA granted soticlestat orphan drug status in 2017.
The medication works by inhibiting the activity of the cholesterol 24-hydroxylase (CH24H) enzyme, which plays a role in regulating the neurotransmitter glutamate, which can increase the initiation and spread of seizure activity.
A multicenter Phase 1b/2a clinical trial (NCT03166215) studied the safety and tolerability of soticlestat in 18 patients with rare epileptic disorders, including Dravet syndrome, Lennox-Gastaut syndrome, and tuberous sclerosis complex. This study also demonstrated that soticlestat was safe and well-tolerated.
Ovid and Takeda are conducting a Phase 2 extension study called ENDYMION (NCT03635073) to assess the long-term safety and tolerability of soticlestat in patients with Dravet syndrome, Lennox-Gastaut syndrome, Dup15q syndrome, and CDKL5 deficiency disorder. The trial is still recruiting participants in the U.S., Australia, China, Israel, Poland, and Spain. Researchers expect to complete it in April 2023.
SPN-817 (previously BIS-001) a treatment that Supernus Pharmaceuticals is developing. It 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). Researchers think this can reduce seizure activity in the brain.
An open-label Phase 1b trial (NCT03156439) tested the safety, tolerability, and pharmacokinetics of SPN-817.
Last updated: July 24, 2020
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