A Robust
Pipeline

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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 epilepsies, and other neurological conditions with high unmet need that may benefit from precision treatment.

    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

    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

  • Our neuroendocrinology portfolio includes investigational therapies for a rare endocrine disorder known as classic congenital adrenal hyperplasia (CAH), and also a chronic adrenal disorder known as adrenal insufficiency. These disorders impact thousands of children and adults worldwide.

    Congenital Adrenal Hyperplasia (CAH)
    crinecerfont

    Congenital Adrenal Hyperplasia in Children & Adolescents

    Phase 1
    Phase 2
    Phase 3
    crinecerfont

    Congenital Adrenal Hyperplasia in Adults

    Phase 1
    Phase 2
    Phase 3

    Description

    CAH is a group of genetic conditions that result in an enzyme deficiency that alters the production of adrenal hormones that are essential for life. Approximately 95% of CAH cases are caused by a mutation that leads to deficiency of 21-hydroxylase (21-OHD). 21-OHD is an enzyme that is essential for making cortisol and aldosterone, two hormones that are critical to numerous physiologic functions. Cortisol allows the body to respond to injury, stress, or illness; and aldosterone maintains proper blood pressure and sodium levels. Severe deficiency of 21-OHD is called classic CAH, and it leads to an inability of the adrenal glands to produce cortisol and, in approximately 75% of cases, aldosterone. If left untreated, classic CAH can result in salt wasting, dehydration, and even death. This condition also results in overproduction of androgens, which can affect growth and sexual development and reduce fertility.

    There are currently no non-glucocorticoid (GC) treatments approved by the US Food and Drug Administration for classic CAH. GCs are the current standard of care, and they are used to correct the cortisol deficiency. They are also typically used at greater-than-physiologic doses to try to suppress the high levels of corticotropin-releasing factor (CRF) and adrenocorticotropic hormone (ACTH) that result in androgen excess. However, long-term exposure to high doses of GCs can cause metabolic issues, such as weight gain and diabetes, cardiovascular disease, and osteoporosis, and may also have psychological and cognitive impact, such as changes in mood and memory. On the other hand, androgen excess as a result of insufficient GC doses has been associated with abnormal bone growth and development in pediatric patients, acne, excess hair growth, menstrual irregularities in women, and adrenal rest tumors and fertility issues in both sexes.

    Crinecerfont is an investigational, oral, selective corticotropin-releasing factor type 1 (CRF1) receptor antagonist being studied to reduce and control excess adrenal androgens in people with CAH. Blocking CRF1 receptors in the pituitary gland has been shown to decrease ACTH levels, which in turn decreases the production of adrenal androgens and potentially the symptoms associated with classic CAH. Our data also suggests that lowering androgen levels enables lower, more physiologic dosing of GCs and thus could potentially reduce the complications associated with exposure to greater-than-normal GC doses in patients with classic CAH. Crinecerfont has been granted orphan drug designation in the US and European Union.

    • Status

    • Active; not recruiting CAHtalyst™ Pediatric and CAHtalyst™ Adult studies

    In September and October 2023, we announced top-line data from our CAHtalyst™ Pediatric and CAHtalyst™ Adult studies evaluating the efficacy, safety, and tolerability of crinecerfont in children, adolescents, and adults with CAH due to 21-OHD. The data from the CAHtalyst Pediatric and Adult studies, including data from the open-label treatment periods, will support regulatory submissions to the FDA in 2024 and later to the European Medicines Agency.

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

    For more information about the CAHtalyst™ Adult Phase 3 study, please visit 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 and major depressive disorder.

    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. 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.

    • 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-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 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).

    • 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
    NBI-1117570#

    Undisclosed

    Phase 1
    Phase 2
    Phase 3

    Description

    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.

    • Status

    • 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.
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