A Robust Pipeline
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Areas of investigation include potential new treatments in neurology, neuroendocrinology, and neuropsychiatry.
We have a robust pipeline of investigational therapies with the potential to address unmet clinical needs of patients.
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.
valbenazine*
Dyskinetic Cerebral Palsy
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.
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.
NBI-921352||
Rare Pediatric Epilepsy: SCN8A-DEE
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.
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
NBI-1076986
NBI-1076986 Movement Disorder
NBI-1076986 is an investigational, oral, muscarinic M4 selective acetylcholine antagonist for the potential treatment of certain movement disorders.
Our clinical neuroendocrinology portfolio includes investigational therapies for rare and chronic endocrine disorder such as congenital adrenal hyperplasia (CAH) and adrenal insufficiency. These disorders impact thousands of children and adults worldwide. In addition, compounds in our pre-clinical pipeline are focused on next-generation therapies with potential deep and durable efficacy for neuroendocrine and related metabolic disorders.
crinecerfont
Congenital Adrenal Hyperplasia in Children & Adolescents
crinecerfont
Congenital Adrenal Hyperplasia in Adults
CAH is a rare genetic condition that results 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-OH). 21-OH 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-OH can lead to an inability of the adrenal glands to produce cortisol and, in approximately 75% of cases, aldosterone. If left untreated, CAH can result in salt wasting, dehydration, and even death. 21-OH deficiency (21-OHD) associated with CAH results in overproduction of adrenocorticotropic hormone (ACTH) and androgens, which can affect growth and sexual development and reduce fertility.
Glucocorticoids (GCs), the current standard of care, are used to treat the endogenous cortisol deficiency but doses in excess of the amount needed for cortisol replacement (supraphysiologic) are needed to lower levels of ACTH and androgens. However, GC treatment at supraphysiologic doses has been associated with serious and significant complications of steroid excess, including 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, memory, and sleep. On the other hand, treatment with lower GC doses may result in androgen excess which has been associated with abnormal bone growth and development in pediatric patients, female health problems such as excess hair growth and menstrual irregularities, testicular rest tumors in males and acne and fertility issues in both sexes.
Crinecerfont is an investigational, oral, selective corticotropin-releasing factor type 1 (CRF1) receptor antagonist being developed to reduce and control excess ACTH and adrenal androgens through a GC-independent mechanism for the treatment of 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. Data from the studies demonstrated that crinecerfont may lower elevated adrenal androgen levels, which could enable lower, more physiologic dosing of glucocorticoids and thus potentially reduce the complications associated with exposure to high dose glucocorticoids in patients with CAH. Crinecerfont has been granted orphan drug designation in the US and European Union.
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, supported regulatory submissions to the FDA in April 2024. The FDA granted Priority Review with Prescription Drug User Fee Act target action dates in late December 2024.
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 CAH
Adrenal Insufficiency
Modified-release hydrocortisone
Adrenal insufficiency is a chronic endocrine condition that occurs when the body does not make enough of certain adrenal hormones, including cortisol and often aldosterone. Steroids such as hydrocortisone and glucocorticoids are used to replace the missing cortisol, but they cannot match the natural diurnal rhythm of the body’s cortisol production.
Modified-release hydrocortisone is an investigational therapy being developed to mimic the circadian rhythm of the body’s natural cortisol production as a potential treatment for adrenal insufficiency.
Congenital Adrenal Hyperplasia
Modified-release hydrocortisone
CAH is a rare genetic condition that results 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-OH). 21-OH 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-OH can lead to an inability of the adrenal glands to produce cortisol and, in approximately 75% of cases, aldosterone. If left untreated, CAH can result in salt wasting, dehydration, and even death. 21-OH deficiency (21-OHD) associated with CAH results in overproduction of adrenocorticotropic hormone (ACTH) and androgens, which can affect growth and sexual development and reduce fertility. Steroids such as hydrocortisone and glucocorticoids are used to replace the missing cortisol, but they cannot match the natural diurnal rhythm of the body’s cortisol production.
Modified-release hydrocortisone is an investigational therapy being developed to mimic the circadian rhythm of the body’s natural cortisol production as a potential treatment for congenital adrenal hyperplasia.
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
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.
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
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.
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 ClinicalTrials.gov.
NBI-1117568#
Schizophrenia
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.
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 Nxera Pharma (formerly Sosei Heptares).
For more information about this Phase 2 study, please visit ClinicalTrials.gov.
NBI-1070770#
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-1070770 is an investigational, oral, negative allosteric modulator (NAM) of the NR2B subunit-containing N-methyl-D-aspartate receptor (NR2B-NMDAR) being developed for the potential treatment of patients with inadequate response to treatment in MDD.
NBI-1117570#
Undisclosed
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.
NBI-1117569#
CNS Indications
NBI-1117569 is an investigational, oral, muscarinic M1/M4 (M4 preferring) acetylcholine agonist for the potential treatment of certain neurological and neuropsychiatric conditions.
NBI-1117567#
CNS Indications
NBI-1117567 is an investigational, oral, muscarinic M1/M4 (M1 preferring) acetylcholine agonist for the potential treatment of certain neurological and neuropsychiatric conditions.
NBI-1065890
CNS indications
NBI-1065890 is an investigational, oral, selective inhibitor of vesicular monoamine transporter-2 (VMAT2). NBI-1065890 is being developed as a potential treatment for certain neurological and neuropsychiatric conditions.
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 Xenon Pharmaceuticals, Inc.
¶Licensed from Takeda Pharmaceutical Company Limited.
#Licensed from Nxera Pharma (formerly Sosei Heptares).
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