Investigational
Therapies
Our team strives to advance medicines for neurological,
neuroendocrine, and neuropsychiatric disorders.

We relentlessly pursue scientific discovery and development that can lead to important therapies for patients who desperately need new, better options.
Movement Disorders
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valbenazine* for chorea in Huntington disease (HD)
Valbenazine* is an investigational, selective, orally active vesicular monoamine transporter 2 (VMAT2) inhibitor for the potential treatment of chorea in HD.
Clinical Studies
valbenazine* for dyskinetic cerebral palsy (DCP)
Valbenazine* is an investigational, selective vesicular monoamine transporter 2 (VMAT2) inhibitor for the potential treatment of DCP.
Clinical Studies
Epilepsy
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NBI-827104† for epileptic encephalopathy with continuous spike-and-wave during sleep (EE-CSWS)
NBI-827104† is a selective, and brain penetrating T-type calcium channel blocker (Cav 3.1, Cav 3.2, Cav 3.3) for the potential treatment of EE-CSWS.
Clinical Studies
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NBI-921352‡ for SCN8A developmental and epileptic encephalopathy (SCN8A-DEE)
NBI-921352‡ is an investigational, selective sodium channel inhibitor (Nav1.6) for the potential treatment of SCN8A-DEE.
Clinical Studies
NBI-921352‡ for focal onset seizures in adults (FOS)
NBI-921352‡ is an investigational, selective sodium channel inhibitor (Nav1.6) for the potential treatment of focal onset seizures in adults.
Clinical Studies
Congenital Adrenal Hyperplasia
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crinecerfont for congenital adrenal hyperplasia (CAH)
Crinecerfont is an investigational, oral, non-steroidal, selective corticotropin-releasing factor type 1 (CRF1) receptor antagonist for the potential treatment of CAH due to 21-hydroxylase deficiency (21-OHD) in children and adults.
Clinical Studies
Neuropsychiatric Disorders
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valbenazine* as an add-on treatment for schizophrenia
Valbenazine is an investigational, selective vesicular monoamine transporter 2 (VMAT2) inhibitor being developed as a potential add-on treatment for patients with schizophrenia.
Clinical Studies
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NBI-1065846§ for anhedonia in major depressive disorder (MDD)
NBI-1065846§ is a potential first-in-class, investigational G protein-coupled receptor 139 (GPR139) agonist for the potential treatment of anhedonia in major depressive disorder (MDD).
Clinical Studies
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NBI-1065845§ for inadequate response to treatment in major depressive disorder (MDD)
NBI-1065845§ is a potential first-in-class, investigational alpha-amino-3-hydroxy-5-methyl-4-isoxazole propionic acid (AMPA) potentiator for the potential treatment of patients with inadequate response to treatment in MDD.
Clinical Studies
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luvadaxistat§ for cognitive impairment associated with schizophrenia (CIAS)
Luvadaxistat§ is a potential first-in-class, investigational, selective d-amino acid oxidase (DAAO) inhibitor for the potential treatment of cognitive impairment associated with schizophrenia (CIAS).
Clinical Studies
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NBI-1117568¶ for schizophrenia
NBI-1117568¶ is an oral, investigational muscarinic M4 agonist for the potential treatment of schizophrenia.
Clinical Studies
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NBI-1070770§ for major depressive disorder (MDD)
Neurocrine Biosciences has initiated a Phase 1 clinical study with NBI-1070770§, a novel, orally active, small molecule as a potential treatment for major depressive disorder.
*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.