A Robust
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Areas of investigation include potential new treatments in neurology, neuroendocrinology, and neuropsychiatry.

 
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Our Pipeline

We have a robust pipeline of investigational therapies with the potential to address unmet clinical needs of patients.

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  • Our pipeline programs in neurology consist of investigational therapies targeting movement disorders, pediatric and adult epilepsies, and other neurological conditions with high unmet need that may benefit from precision treatment.

    Huntington Disease (HD)
    valbenazine*

    Chorea in Huntington Disease

    Phase 1
    Phase 2
    Phase 3

    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.

    • Status

    • Submitted sNDA to U.S. FDA; ongoing Phase 3 open-label study

    We are conducting KINECT‑HD2, a Phase 3, open-label study to evaluate the long-term safety and tolerability of valbenazine* for the treatment of chorea in HD.

    For more information about the KINECT‑HD2 study, please visit HuntingtonStudyGroup.com and ClinicalTrials.gov.

    Learn More About How valbenazine is Thought to Work

    Dyskinetic Cerebral Palsy (DCP)
    valbenazine*

    Dyskinetic Cerebral Palsy

    Phase 1
    Phase 2
    Phase 3

    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.

    • Status

    • Ongoing Phase 3 study

    We are currently conducting a Phase 3 study to assess valbenazine* in patients with DCP.

    For more information about this Phase 3 study, please visit KINECT-DCP Study or ClinicalTrials.gov.

    Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep (EE-CSWS)
    NBI-827104§

    Rare Pediatric Epilepsy: EE-CSWS

    Phase 1
    Phase 2
    Phase 3

    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 (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.

    • Status

    • Ongoing Phase 2 STEAMBOAT™ study

    We are conducting the STEAMBOAT Phase 2 study to evaluate NBI-827104§ in pediatric patients with EE-CSWS.

    For more information about this Phase 2 study, please visit SteamboatStudy.com and ClinicalTrials.gov.

    Learn More About EE-CSWS and How NBI-827104 is Thought to Work

    Focal Onset Seizures in Adults
    NBI-921352||

    Focal Onset Seizures in Adults

    Phase 1
    Phase 2
    Phase 3

    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 Nav1.6 sodium channel inhibitor for the potential treatment of focal onset seizures in adults. Neurocrine acquired exclusive rights to NBI-921352|| from Xenon Pharmaceuticals, Inc.

    • Status

    • Ongoing Phase 2 study

    We are currently conducting a Phase 2 study of NBI-921352|| in adult patients with focal onset seizures.

    For more information about this Phase 2 study, please visit ClinicalTrials.gov.

    Learn More About Focal Onset Seizures and How NBI-921352 is Thought to Work

    SCN8A Developmental and Epileptic Encephalopathy (SCN8A-DEE)
    NBI-921352||

    Rare Pediatric Epilepsy: SCN8A-DEE

    Phase 1
    Phase 2
    Phase 3

    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 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.

    • Status

    • Ongoing Phase 2 KAYAK™ study

    We are currently conducting the KAYAK Phase 2 study of NBI-921352|| as an adjunctive therapy in children and young adults living with SCN8A-DEE.

    For more information about this Phase 2 study, please visit KayakStudy.com and ClinicalTrials.gov.

    Learn More About SCN8A-DEE and How NBI-921352 is Thought to Work

    Hear from Dr. Schreiber on SCN8A History, Early Diagnosis and Hope for Future Medication

  • 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.

    Congenital Adrenal Hyperplasia (CAH)
    crinecerfont

    Congenital Adrenal Hyperplasia in Adults

    Phase 1
    Phase 2
    Phase 3
    crinecerfont

    Congenital Adrenal Hyperplasia in Children & Adolescents

    Phase 1
    Phase 2
    Phase 3

    Description

    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.

    • Status

    • 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.

    For more information about the CAHtalyst™ Adult Phase 3 study, please visit CAHtalyst.CAHStudies.com or ClinicalTrials.gov.

    For more information about the CAHtalyst™ Pediatric Phase 3 study, please visit CAHtalystPeds.CAHStudies.com or ClinicalTrials.gov.

    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, major depressive disorder, and anhedonia.

    valbenazine*

    Adjunctive Treatment of Schizophrenia

    Phase 1
    Phase 2
    Phase 3

    Description

    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. About 30% of the 3.5 million people living with schizophrenia in the U.S. 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.

    • Status

    • Ongoing Phase 3 registrational Journey™ Study

    We are currently conducting the Journey™ Study, a Phase 3 study of valbenazine* as an adjunctive treatment in patients with schizophrenia.

    For more information on this study, please visit JourneyResearchStudies.com or ClinicalTrials.gov.

    NBI-1065846

    Anhedonia in Major Depressive Disorder

    Phase 1
    Phase 2
    Phase 3

    Description

    Anhedonia results in an inability to experience pleasure from normally enjoyable experiences. It is a core symptom of major depressive disorder (MDD), and frequently occurs in people with schizophrenia, bipolar disorder, substance abuse, Parkinson’s disease, diabetes, and coronary artery disease.

    NBI-1065846 is a potential first-in-class, investigational G protein-coupled receptor (GPR)-139 agonist being developed for the treatment of anhedonia in MDD.

    • Status

    • Ongoing Phase 2 TERPSIS™ study

    We are currently conducting the Phase 2 TERPSIS™ study of NBI-1065846 in patients with anhedonia.

    For more information about this Phase 2 study, please visit ClinicalTrials.gov.

    NBI-1065845

    Inadequate Response to Treatment in Major Depressive Disorder

    Phase 1
    Phase 2
    Phase 3

    Description

    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.

    • Status

    • Ongoing Phase 2 SAVITRI™ study

    We are currently conducting the Phase 2 SAVITRI™ study of NBI-1065845 in patients with inadequate response to treatment in MDD.

    For more information about this Phase 2 study, please visit TERPSIS Study or StudiesonMDD.com or ClinicalTrials.gov.

    luvadaxistat

    Cognitive Impairment Associated With Schizophrenia

    Phase 1
    Phase 2
    Phase 3

    Description

    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 3.5 million people living with schizophrenia in the U.S. 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).

    • Status

    • 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#

    Schizophrenia

    Phase 1
    Phase 2
    Phase 3

    Description

    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.

    • Status

    • 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 Disorder

    Phase 1
    Phase 2
    Phase 3

    Description

    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.

    • Status

    • Ongoing Phase 1 study
    Neurocrine Biosciences has global rights unless otherwise noted. Neurocrine Biosciences has commercial rights to Ongentys in the U.S. and Canada and shares profits and losses on NBI-1065845 and NBI-1065846§ with Takeda Pharmaceutical Company Limited.

    *Mitsubishi Tanabe Pharma Corporation (MTPC) has commercialization rights in Japan and other select Asian markets.
    Under license from Bial.
    AbbVie has global commercialization rights.
    §Licensed from Idorsia Ltd.
    Licensed from Xenon Pharmaceuticals, Inc.
    Licensed from Takeda Pharmaceutical Company Limited.
    #Licensed from Sosei Heptares.
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