Neurocrine Biosciences acquired the exclusive rights to NBI-827104 (ACT-709478) from Idorsia Ltd. NBI-827104 is a potent, selective, orally active and brain penetrating T-type calcium channel blocker. Neurocrine Biosciences is initiating a Phase II study of NBI-827104 for the treatment of a form of rare pediatric epilepsy known as epileptic encephalopathy with continuous spikes and waves during sleep (EE-CSWS) in 2020.
EE-CSWS is a rare pediatric epilepsy that impacts more than 6,000 children in the United States. Typical onset occurs between ages 2 and 4 years old with seizures occurring infrequently. Diagnosis of EE-CSWS is based on a unique electroencephalographic (EEG) pattern for electrical status epilepticus in sleep (ESES), together with cognitive stagnation and regression. There is currently no approved treatment for the disorder.