SCN8A Developmental and Epileptic Encephalopathy (SCN8A-DEE)

SCN8A-DEE is a rare disorder that causes a range of symptoms including severe epilepsy, developmental delay, and other medical challenges and is highly refractory to currently available antiseizure medication. SCN8A-DEE is linked to gain of function mutations in the SCN8A gene, a gene that affects how brain cells function. Mutations on this gene can cause neurological problems including epilepsy, intellectual disabilities, movement disorders and impaired development.

SCN8A-DEE is rare, with less than 1,000 patients believed to be diagnosed with the disorder worldwide. There are currently no approved therapies for this form of pediatric epilepsy.

NBI-921352: An Investigational Therapy for SCN8A-DEE

NBI-921352 is a potent, highly selective Nav1.6 sodium channel inhibitor being developed to treat pediatric patients with SCN8A developmental and epileptic encephalopathy (SCN8A-DEE).

Neurocrine Biosciences has received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration (FDA) for NBI-921352 in SCN8A-DEE.

Neurocrine Biosciences acquired the exclusive rights to NBI-921352 from Xenon Pharmaceuticals.

Neurocrine Biosciences is also developing NBI-921352 for the potential treatment of focal onset seizures.

Clinical Trials: SCN8A-DEE

Neurocrine Biosciences is conducting the KAYAK™ Phase 2 study to assess the efficacy, safety, tolerability, and pharmacokinetics of NBI-921352 as adjunctive therapy in patients (2-21 years of age) with SCN8A-DEE. For more information about the study, please visit KayakStudy.com or ClinicalTrials.gov.

Following completion of the KAYAK™ study, eligible participants may have the option to enroll in our open-label extension study to evaluate the long-term safety and tolerability of NBI-921352 in patients with SCN8A-DEE. For more information about this open-label study, please visit ClinicalTrials.gov.