Article published by Dravet Syndrom News
Long-term treatment with soticlestat was found to reduce seizure frequency by more than 50% in most Dravet syndrome patients, according to about two years of data from the ongoing ENDYMION 1 clinical trial.
“Soticlestat was associated with sustained seizure reductions over the interim period presented here,” researchers wrote.
Yuan Yao, MD, from the Takeda Development Center Asia, discussed the findings at last week’s annual meeting of the American Academy of Neurology (AAN), in a talk titled “Long-Term Treatment Effects of Soticlestat in Patients with Dravet Syndrome or Lennox–Gastaut Syndrome: Interim Data from the ENDYMION 1 Trial.”
The work was funded by Takeda Pharmaceuticals, which in 2021 acquired the rights to develop soticlestat for Dravet and Lennox-Gastaut syndrome (LGS), another rare childhood seizure disorder.
The therapy candidate works by blocking CH24H, an enzyme that regulates the activity of a seizure-related brain signaling molecule called glutamate. In Dravet mouse models, the therapy was shown to reduce seizures and prevent the animals’ death.
Soticlestat “is an investigative anti-seizure medication with a mechanism of action different from any approved” anti-seizure therapy, Yao said.