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Description

HD is a hereditary, progressive neurodegenerative disorder in which neurons within the brain break down, resulting in motor, cognitive, and psychiatric symptoms. Symptoms generally appear between ages 30 to 50 years and worsen over a 10- to 25-year period. Many patients with HD experience chorea, an involuntary movement disorder, in which patients develop abnormal, abrupt, or irregular movements. Chorea can affect various body regions and interfere with speech, swallowing, posture, and gait, making it difficult or even impossible to independently perform activities of daily living.

Approximately 90% of the roughly 41,000 people in the U.S. living with HD also experience chorea.

Valbenazine* is an investigational, selective, orally active vesicular monoamine transporter 2 (VMAT2) inhibitor being investigated for the potential treatment of chorea in HD.

In December 2021, we announced top-line data from our Phase 3 KINECT-HD™ study evaluating the efficacy, safety, and tolerability of valbenazine* in adults with chorea in HD. We submitted a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) in October 2022.

Description

DCP is a form of cerebral palsy that is associated with a range of developmental delays, difficulties with physical function, and involuntary muscle movements. DCP is caused by damage to the motor circuits in the brain involved in coordination and movement control.

Valbenazine* is an investigational, selective, orally active vesicular monoamine transporter 2 (VMAT2) inhibitor for the potential treatment of DCP.

Description

EE-CSWS is a rare pediatric epilepsy syndrome characterized by onset of seizures with a unique electroencephalogram (EEG) pattern referred to as electrical status epilepticus in sleep (ESES). Cognitive stagnation and regression typically develop one to two years after onset. Following puberty, seizures tend to improve; however developmental delays often remain.

EE-CSWS impacts less than 2% of children living with epilepsy worldwide. There is no approved treatment for the disorder.

NBI-827104^§ is an investigational, selective, orally active, and brain-penetrating T-type calcium channel blocker (Ca_v 3.1, Ca_v 3.2, Ca_v 3.3) currently under development for the potential treatment of EE-CSWS. Neurocrine Biosciences acquired the exclusive rights to NBI-827104^§ from Idorsia.

Description

Focal onset seizures, previously referred to as partial-onset seizures, start in one area of the brain and can involve involuntary movements with alteration or loss of awareness that can last up to several minutes. As the most common type of seizure in adults, focal onset seizures can interfere with activities of daily living and negatively impact quality of life.

NBI-921352^|| is an investigational selective Na_v1.6 sodium channel inhibitor for the potential treatment of focal onset seizures in adults. Neurocrine acquired exclusive rights to NBI-921352^|| from Xenon Pharmaceuticals, Inc.

Description

SCN8A-DEE is a rare pediatric syndrome associated with a genetic mutation of the SCN8A gene. It is characterized by severe epilepsy, early onset developmental delay, cognitive impairment, and other medical challenges. Seizures begin at a median age of four months and are highly refractory to currently available anti-seizure medication. Over 90% of children with SCN8A-DEE are non-verbal, and about half are not ambulatory.

There are currently no approved therapies for this form of pediatric epilepsy.

NBI-921352^|| is an investigational selective Na_v1.6 sodium channel inhibitor for the potential treatment of SCN8A-DEE. Neurocrine acquired exclusive rights to NBI-921352^|| from Xenon Pharmaceuticals, Inc. and received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration (FDA) for NBI-921352^|| in SCN8A-DEE.