Areas of investigation include potential new treatments in neurology, neuroendocrinology, and neuropsychiatry.
Our pipeline programs in neurology consist of investigational therapies targeting movement disorders, pediatric epilepsies, and other neurological conditions with high unmet need that may benefit from precision treatment.Phase 1Phase 2Phase 3Dyskinetic Cerebral Palsy (DCP)valbenazine*
Dyskinetic Cerebral PalsyPhase 1Phase 2Phase 3
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.Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep (EE-CSWS)NBI-827104§
Rare Pediatric Epilepsy: EE-CSWSPhase 1Phase 2Phase 3
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 (Cav 3.1, Cav 3.2, Cav 3.3) currently under development for the potential treatment of EE-CSWS. Neurocrine Biosciences acquired the exclusive rights to NBI-827104§ from Idorsia.
Learn More About EE-CSWS and How NBI-827104 is Thought to WorkSCN8A Developmental and Epileptic Encephalopathy (SCN8A-DEE)NBI-921352||
Rare Pediatric Epilepsy: SCN8A-DEEPhase 1Phase 2Phase 3
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 Nav1.6 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.
Learn More About SCN8A-DEE and How NBI-921352 is Thought to Work
Our neuroendocrinology portfolio includes an investigational therapy for classic congenital adrenal hyperplasia (CAH), a rare endocrine disorder that impacts children and adults, and presents a high unmet medical need despite current treatments.Phase 1Phase 2Phase 3Congenital Adrenal Hyperplasia (CAH)crinecerfont
Congenital Adrenal Hyperplasia in AdultsPhase 1Phase 2Phase 3crinecerfont
Congenital Adrenal Hyperplasia in Children & AdolescentsPhase 1Phase 2Phase 3
Classic CAH is a genetic disorder that causes little to no cortisol production and increased secretion of adrenocorticotropic hormone (ACTH) and androgens. In approximately 75% of cases, the adrenal glands cannot produce aldosterone, which can result in salt wasting adrenal crisis, causing extreme weakness, low blood pressure, shock, and even death. There are currently no non-steroidal U.S. Food and Drug Administration (FDA)-approved treatments for classic CAH. Glucocorticoids at replacement, or physiologic, doses to treat cortisol deficiency alone are typically not enough to address high ACTH and androgen levels. Long-term, chronic exposure to greater than physiologic dosing of glucocorticoids can cause metabolic issues, bone loss, growth impairment, and infection risk typical of iatrogenic Cushing’s syndrome. The result is an undesirable trade-off of trying to balance the negative effects of too much glucocorticoids with the negative symptoms of too much androgen.
Crinecerfont is an investigational, oral, non-steroidal, selective corticotropin-releasing factor type 1 (CRF1) receptor antagonist. Adding crinecerfont to glucocorticoid therapy may help allow normal dosing of glucocorticoids to replace cortisol, while reducing ACTH and androgen levels. Crinecerfont has been granted orphan drug designation in the U.S. and the European Union.
- Ongoing Phase 3 global registrational CAHtalyst™ Adult and CAHtalyst™ Pediatric studies
We are currently conducting two global, registrational Phase 3 studies of crinecerfont in adults (ages 18 years and older) and children and adolescents (ages 2 to 17 years old) with classic CAH at study sites in the U.S., Canada, and Europe.
Learn More About the Cause of Classic CAH
Mental health disorders occur in more than 792 million people globally. New treatment options are critical to help lessen the personal, social, and economic toll on people who suffer from them.
Our pipeline includes early-to-mid-stage programs evaluating potential novel therapies for schizophrenia and major depressive disorder.Phase 1Phase 2Phase 3valbenazine*
Adjunctive Treatment of SchizophreniaPhase 1Phase 2Phase 3
Schizophrenia is a serious and complex mental disorder that affects how a person thinks, feels, and behaves. As one of the leading causes of disability worldwide, it often results in significant emotional burden for those who experience symptoms, as well as their family and friends. Approximately 33% of the people living with schizophrenia fail to respond to current antipsychotic therapy.
Scientists believe that elevated dopamine in an area of the brain called the striatum is associated with symptoms of schizophrenia. Valbenazine* is an investigational, selective, orally active vesicular monoamine transporter 2 (VMAT2) inhibitor that is being investigated as a potential adjunctive treatment for patients with schizophrenia.
Inadequate Response to Treatment in Major Depressive DisorderPhase 1Phase 2Phase 3
Major depressive disorder (MDD) is a mental health disorder characterized by a persistently depressed mood, loss of interest, lack of enjoyment in daily activities, and decreased energy. MDD is one of the leading causes of disability. Approximately one-third of the more than 16 million people in the U.S. who live with major depressive disorder do not respond to available antidepressants.
NBI-1065845¶ is a potential first-in-class, investigational alpha-amino-3-hydroxy-5-methyl-4-isoxazole propionic acid (AMPA) potentiator being developed for the potential treatment of patients with inadequate response to treatment in MDD.
Cognitive Impairment Associated With SchizophreniaPhase 1Phase 2Phase 3
Schizophrenia is a serious and complex mental disorder that affects how a person thinks, feels, and behaves. As one of the leading causes of disability worldwide, it often results in significant emotional burden for those who experience symptoms, as well as their family and friends. Approximately 80% of the approximately 24 million people living with schizophrenia worldwide experience clinically relevant cognitive symptoms. Cognitive symptoms are characterized by poor mental function and include difficulty paying attention, processing information, and making decisions.
Luvadaxistat¶ is a potential first-in-class, investigational selective d-amino acid oxidase (DAAO) inhibitor being developed for the potential treatment of cognitive impairment associated with schizophrenia (CIAS).
- Ongoing Phase 2 ERUDITE™ study
We are currently conducting the ERUDITE™ study, a Phase 2 study of luvadaxistat¶ in adults with CIAS.
For more information about this Phase 2 study, please visit ClinicalTrials.gov.NBI-1117568#
SchizophreniaPhase 1Phase 2Phase 3
Schizophrenia is a serious and complex mental disorder that affects how a person thinks, feels, and behaves. As one of the leading causes of disability worldwide, it often results in significant emotional burden for those who experience symptoms, as well as their family and friends. All currently approved antipsychotic medications are believed to work through direct action on monoaminergic receptors. However, approximately 30% of patients do not benefit adequately from these medications, and at least 40% report bothersome side effects, including symptoms of metabolic syndrome and neurologic symptoms.
NBI-1117568# is an investigational, muscarinic M4 selective acetylcholine receptor agonist for the potential treatment of adults with schizophrenia. Muscarinic receptors are central to brain function and have been validated as drug targets in psychosis and cognitive disorders. Highly selective muscarinic agonists represent a novel mechanism of action to target symptoms of schizophrenia.
- Ongoing Phase 2 study
We are conducting a Phase 2 study of NBI-1117568# in adults with schizophrenia.
We are developing NBI-1117568# as part of a strategic collaboration and licensing agreement with Sosei Heptares.
For more information about this Phase 2 study, please visit ClinicalTrials.gov.NBI-1070770¶
Major Depressive DisorderPhase 1Phase 2Phase 3
MDD is a mental health disorder characterized by a persistently depressed mood, loss of interest, lack of enjoyment in daily activities, and decreased energy. MDD is one of the leading causes of disability. Approximately one-third of the more than 16 million people in the U.S. who live with major depressive disorder do not respond to available antidepressants.
NBI-1070770¶ is an investigational, novel, orally active small molecule that is being developed for the treatment of MDD.
- Ongoing Phase 1 study
UndisclosedPhase 1Phase 2Phase 3
NBI-1117570 is an investigational, oral, muscarinic M1/M4 selective dual agonist that is being studied for the treatment of symptoms of psychosis and cognition in neurological and neuropsychiatric conditions.
- Ongoing Phase 1 study
Neurocrine Biosciences has global rights unless otherwise noted. Neurocrine Biosciences shares profits and losses on NBI-1065845¶ with Takeda Pharmaceutical Company Limited.*Mitsubishi Tanabe Pharma Corporation (MTPC) has commercialization rights in Japan and other select Asian markets.
§Licensed from Idorsia Ltd.
║Licensed from Xenon Pharmaceuticals, Inc.
¶Licensed from Takeda Pharmaceutical Company Limited.
#Licensed from Sosei Heptares.