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Item 1. Business

2026-02-11 compared with 2025-02-10 · 205 added, 161 removed, 249 unchanged (60% of the section changed)

Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with a simple purpose:

to relieve suffering for people with great needs, but few options.

We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neuropsychiatric, neurological, and neuroendocrine disorders.

to relieve suffering for people with great needs.

We are dedicated to discovering, developing, and commercializing life-changing treatments for patients with under-addressed neurological, psychiatric, endocrine, and immunological disorders.

Our portfolio of products includes U.S.

Food and Drug Administration (FDA) approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, classic congenital adrenal hyperplasia (CAH), and endometriosis and uterine fibroids in collaboration with AbbVie Inc.

In addition, we have a diversified portfolio of multiple compounds in mid- to late-phase development across our core therapeutic areas and an expanding early-phase pipeline that includes a range of modalities including small molecules, peptides, proteins, antibodies, and gene therapy.

Food and Drug Administration (FDA) approved treatments for tardive dyskinesia (TD), chorea associated with Huntington's disease, classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (CAH), and endometriosis and uterine fibroids in collaboration with AbbVie Inc.

We launched INGREZZA ® (valbenazine) in the U.S.

as the first FDA-approved drug for the treatment of tardive dyskinesia in May 2017 and for the treatment of chorea associated with Huntington's disease in August 2023 and launched CRENESSITY TM (crinecerfont) in the U.S.

as the first FDA-approved drug for the treatment of TD in May 2017 and for the treatment of chorea associated with Huntington's disease in August 2023 and launched CRENESSITY ® (crinecerfont) in the U.S.

as a first-in-class FDA-approved treatment of CAH in December 2024.

We estimate that tardive dyskinesia affects approximately 800,000 people in the U.S., that approximately 90% of the 40,000 people in the U.S.

affected by Huntington’s disease will develop chorea, and that CAH affects approximately 30,000 people in the U.S.

Key elements of our commercial strategy include maximizing the opportunities in INGREZZA and CRENESSITY through consistent and effective commercial execution, continued development of valbenazine as the best-in-class treatment for new patient populations, and to lead the evolving understanding of VMAT2 biology and its role in disease.

INGREZZA net product sales totaled $2.3 billion for 2024, $1.8 billion for 2023, and $1.4 billion for 2022 and accounted for substantially all of our total net product sales during each of these years.

Our partner Mitsubishi Tanabe Pharma Corporation (MTPC) launched DYSVAL ® (valbenazine) in Japan for the treatment of tardive dyskinesia in June 2022 and subsequently in other select Asian markets, where it is marketed as REMLEAS ® (valbenazine).

We receive royalties at tiered percentage rates on MTPC net sales of valbenazine.

Our partner AbbVie launched ORILISSA ® (elagolix tablets) in the U.S.

for the treatment of endometriosis in August 2018 and ORIAHNN ® (elagolix, estradiol and norethindrone acetate capsules and elagolix capsules) in the U.S.

for the treatment of heavy menstrual bleeding due to uterine fibroids in June 2020.

We receive royalties at tiered percentage rates on AbbVie net sales of elagolix.

We estimate that TD affects approximately 800,000 people in the U.S., that approximately 90% of the 40,000 people in the U.S.

affected by Huntington’s disease will develop chorea, and that CAH affects at least 20,000 people in the U.S.

INGREZZA net product sales were $2.51 billion for 2025, $2.31 billion for 2024, and $1.84 billion for 2023 and accounted for a significant portion of our total net product sales during each of these years.

CRENESSITY net product sales were $301.2 million for 2025 during its first full-year of launch.

Commercial Products

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Uterine Fibroids

Adrenal Insufficiency

U.S., United Kingdom, EU4 (2) (3)

Classic Congenital Adrenal Hyperplasia

United Kingdom, EU4 (3)

(1) INGREZZA is marketed as DYSVAL ® (valbenazine) in Japan and REMLEAS ® (valbenazine) in other select Asian markets, where MTPC retains commercialization rights.

(2) ALKINDI is marketed as ALKINDI SPRINKLE ® (hydrocortisone) in the U.S., where Eton Pharmaceuticals, Inc.

retains commercialization rights.

(3) The EU4 market is made up of the following countries:

Germany, France, Italy, and Spain.

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(1) INGREZZA is marketed as DYSVAL ® (valbenazine) in Japan and REMLEAS ® (valbenazine) in other select Asian markets, where Tanabe Pharma Corporation (formerly Mitsubishi Tanabe Pharma Corporation) retains commercialization rights.

(2) AbbVie retains global commercialization rights to elagolix.

Commercial Operations

Our specialty sales force consists of approximately 600 experienced sales professionals located in the U.S.

and is divided into four dedicated sales teams focused on psychiatry, neurology, long-term care, and rare diseases.

We sell INGREZZA in the U.S.

principally to a limited network of specialty pharmacy providers, wholesale distributors, and specialty distributors.

We sell INGREZZA exclusively in the U.S.

through a limited specialty network.

Our customers include select specialty pharmacy providers, wholesale distributors, and specialty distributors.

This focused distribution model allows us to closely manage product supply and support services.

In addition, we sell CRENESSITY in the U.S.

to a specialty pharmacy provider.

through a single specialty pharmacy provider, reflecting the product’s rare disease focus and the need for high-touch distribution.

We rely on third-party logistics providers for packaging, warehousing, and shipment of our products, leveraging their expertise to ensure timely delivery and broad patient access.

Our commercial reach is powered by a specialized sales force of approximately 600 professionals across the U.S.

focused on neurology, psychiatry, long-term care, and rare diseases.

In October 2025, we announced the planned expansion of the INGREZZA and CRENESSITY sales teams to maximize our commercial momentum.

We expect this expansion, which we anticipate completing by the end of the first quarter of 2026, will boost our reach and frequency with prescribers, accelerating INGREZZA’s penetration in both community and institutional settings and supporting the growth trajectory of CRENESSITY in the endocrinology and rare disease community.

Importantly, a larger sales force and enhanced infrastructure also position us for potential upcoming product launches from our diversified pipeline, which includes late-stage candidates in major depressive disorder (osavampator) and schizophrenia (direclidine).

We believe these investments in commercial capabilities will translate into sustained revenue growth and shareholder value, as we drive current product performance and prepare to bring new therapies to market.

Manufacturing and Supply

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Raw materials, active pharmaceutical ingredients (API), and other supplies required for the production of INGREZZA, CRENESSITY, and our product candidates are sourced from various third-party manufacturers and suppliers in quantities adequate to meet our needs.

Continuing adequate supply of such raw materials and API is assured through long-term commercial supply and manufacturing agreements with multiple manufacturers and a continued focus on the expansion and diversification of our third-party manufacturing relationships.

We believe our outsourced manufacturing strategy enables us to direct our financial resources to the maximization of our opportunity with INGREZZA and CRENESSITY, investment in our internal research and development programs, and expansion of our clinical pipeline through business development opportunities.

We believe that continuing adequate supply of such raw materials and API is assured through long-term commercial supply and manufacturing agreements with multiple manufacturers and a continued focus on the expansion and diversification of our third-party manufacturing relationships.

We believe our outsourced manufacturing strategy enables us to direct our financial resources to maximize our commercial opportunities with INGREZZA and CRENESSITY, invest in our internal research and development programs, and expand our clinical pipeline through business development opportunities.

Our third-party manufacturers, suppliers, and service providers may be subject to routine current Good Manufacturing Practice (cGMP) inspections by the FDA or comparable agencies in other jurisdictions.

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Clinical Development Programs

The following chart summarizes our clinical development programs

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Small Molecule

* Initiating Phase 1 clinical study in the first quarter of 2025

Neuropsychiatry

Valbenazine is a highly selective VMAT2 inhibitor.

VMAT2 is a protein concentrated in the human brain that is essential for the transmission of nerve impulses between neurons.

VMAT2 is primarily responsible for packaging and transporting monoamines (dopamine, norepinephrine, serotonin, and histamine) in neurons.

Specifically, dopamine enables neurotransmission among nerve cells that are involved in voluntary and involuntary motor control.

We have ongoing the Journey TM study, a Phase 3 randomized, double-blind, placebo-controlled clinical study to evaluate the efficacy, safety, and tolerability of valbenazine when administered orally once daily as adjunctive treatment in adolescents and adults with schizophrenia who have had an inadequate response to antipsychotics.

Schizophrenia is a serious and complex syndrome with heterogeneous symptoms.

The World Health Organization estimates that the disorder impacts more than 20 million people worldwide.

Annual associated costs for schizophrenia are estimated to be more than $150 billion in the U.S.

As one of the leading causes of disability worldwide, it often results in significant emotional and functional burden for those who experience symptoms, as well as their family and friends.

This chronic and disabling mental health condition is thought to result from a complex interplay of genetic and environmental risk factors.

Traditional treatment approaches for schizophrenia rely on the use of antipsychotic medications that can lead to considerable short- and long-term health impacts.

Osavampator (formerly NBI-1065845)

Osavampator is a potential first-in-class alpha-amino-3-hydroxy-5-methyl-4-isoxazole propionic acid (AMPA) positive allosteric modulator (PAM) in development for patients with inadequate response to treatment of major depressive disorder.

We originally acquired the global rights to osavampator in June 2020 as a 50:50 profit-share product from Takeda.

In January 2025, we agreed with Takeda to convert from sharing operating profits and losses with respect to the development and commercialization of osavampator to a royalty-bearing license.

Pursuant to the license, we have exclusive rights to develop and commercialize osavampator for all indications in all territories worldwide, excluding Japan.

In April 2024, we announced positive top-line data from the Phase 2 SAVITRI™ study of osavampator in adults with major depressive disorder.

The Phase 2 dose-finding study met its primary and key secondary endpoints, demonstrating that once-daily, oral administration of NBI-1065845 produced a statistically significant change from baseline in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score at both Day 28 (primary, p=0.0159) and Day 56 (secondary, p=0.0016).

We have an ongoing Phase 3, randomized, double-blind, placebo-controlled clinical study to evaluate the efficacy and safety of osavampator in adults with major depressive disorder.

Major depressive disorder is a mental health disorder characterized by a persistently depressed mood, loss of interest, lack of enjoyment in daily activities, poor concentration, and decreased energy.

Major depressive disorder is one of the leading causes of disability.

Approximately 21 million people in the U.S.

live with major depressive disorder.

It is estimated that roughly one-third of people living with major depressive disorder do not respond to available antidepressants.

NBI-1117568 is a first-in-class, orally active, highly selective investigational M4 agonist in development as a potential treatment for schizophrenia.

As a selective M4 orthosteric agonist, NBI-1117568 offers the potential for a novel mechanism with an improved safety profile without the need of combination therapy to minimize off-target pharmacology-related side effects, while also not being dependent on the presence of acetylcholine for efficacy.

Muscarinic receptors are central to brain function and validated as drug targets in psychosis and cognitive disorders.

We acquired the global rights to NBI-1117568, excluding in Japan, from Nxera in 2021.

In August 2024, we announced positive top-line data from the Phase 2 clinical study of NBI-1117568 in adults with schizophrenia.

The Phase 2 dose-finding study met its primary endpoint for the once-daily 20 mg dose, demonstrating a clinically meaningful and statistically significant reduction from baseline in the Positive and Negative Syndrome Scale (PANSS) total score at Week 6 (p=0.011 and effect size of 0.61).

The once-daily 20 mg dose also demonstrated statistically significant improvement for additional endpoints, including improvement in the Clinical Global Impression of Severity (CGI-S) scale, Marder Factor Score – Positive Symptom Change, and Marder Factor Score - Negative Symptom Change.

We expect to advance NBI-1117568 into Phase 3 development in the first half of 2025.

NBI-1070770 is a novel, selective, and orally active, negative allosteric modulator (NAM) of the NR2B subunit-containing N-methyl-D-aspartate (NMDA NR2B) receptor in development as a potential treatment for major depressive disorder.

We acquired the global rights to NBI-1070770 from Takeda in 2020.

We have an ongoing Phase 2, multi-center, randomized, double-blind, placebo-controlled clinical study to evaluate the efficacy, safety, and tolerability of NBI-1070770 in adults with major depressive disorder.

Other Early-Stage Neuropsychiatry Programs

We have ongoing Phase 1 first-in-human clinical studies to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of investigational compounds NBI-1117570, NBI-1117567, NBI-1117569, and NBI-1065890 in healthy adult participants.

NBI-1117570, NBI-1117567 (M1 preferring), and NBI-1117569 (M4 preferring) are investigational, oral, muscarinic M1/M4 agonists being developed for the potential treatment of certain neuropsychiatric and neurological conditions.

We acquired the global rights NBI-1117570, NBI-1117567, and NBI-1117569 from Nxera in 2021.

NBI-1065890 is an investigational, oral, selective VMAT2 inhibitor for the potential treatment of certain neuropsychiatric and neurological conditions.

NBI-1065890 was discovered and is being developed internally at Neurocrine Biosciences.

We have an ongoing Phase 3 randomized, double-blind, placebo-controlled clinical study to evaluate the efficacy, safety, and tolerability of valbenazine for the treatment of dyskinetic cerebral palsy in pediatrics and adults.

Dyskinetic cerebral palsy is a non-progressive, permanent disorder marked by involuntary movement and is a result of damage to the fetal or infant brain’s basal ganglia.

The basal ganglia are responsible for submitting messages to the body to help coordinate and control movements.

When damaged, voluntary movements are compromised, resulting in involuntary and abnormal movements.

It affects development and movement and has long term effects on patients’ quality of life.

The long-term outlook for patients with dyskinetic cerebral palsy will depend upon the severity of the brain damage and how well the treatment works.

Dyskinetic cerebral palsy affects up to 15% of the estimated 500,000 to 1 million people affected by cerebral palsy in the U.S.

NBI-1076986 is an investigational, oral, muscarinic M4 selective acetylcholine antagonist for the potential treatment of certain movement disorders.

NBI-1076986 was discovered and is being developed internally at Neurocrine Biosciences.

We have an ongoing Phase 1 first-in-human clinical study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of investigational compound NBI-1076986 in healthy adult participants.

Our research and development pipeline is robust and diversified across neurology, psychiatry, endocrinology, and immunology.

This pipeline is fueled by core expertise in key areas of receptor biology developed over three decades.

We are leveraging validated biological pathways and novel modalities to advance what we believe are potential first-in-class or best-in-class treatment candidates for patients with serious and under-addressed conditions.

Our strategy is to maintain a balanced portfolio by stage of development and across our therapeutic areas of interest – targeting prevalent central nervous system (CNS) disorders such as depression, bipolar, and schizophrenia on one hand, and rare/orphan diseases such as CAH on the other – thereby positioning Neurocrine Biosciences to deliver a steady cadence of innovative medicines for years to come.

By harnessing both novel validated mechanisms and our proprietary platforms, we aim to launch, on average, approximately one new medicine every two years, driving long-term value for patients and shareholders.

The following chart summarizes select clinical development programs.

Psychiatry Portfolio Highlights

Our psychiatry pipeline is built on mechanisms with strong biological rationale (glutamate, muscarinic, monoamine modulation) and is aligned with areas of high unmet need in mental illness.

We believe the breadth of this portfolio, ranging from late-stage to first-in-human studies, positions us to deliver multiple potential first-in-class or best-in-class treatments in psychiatry over the coming years.

Osavampator (NBI-1065845)

Osavampator is a potential first-in-class alpha-amino-3-hydroxy-5-methyl-4-isoxazole propionic acid (AMPA) receptor potentiator in development for adults with major depressive disorder (MDD) who have inadequate response to oral antidepressant treatment.

Approximately one-third of over 16 million people in the U.S.

who live with MDD do not achieve adequate relief with existing antidepressants, and osavampator’s novel mechanism is being studied for antidepressant effects in these insufficient responders.

We have initiated a comprehensive Phase 3 clinical program for osavampator in MDD (with initial topline data expected in 2027), including three acute randomized, double‑blind, placebo‑controlled studies, a randomized‑withdrawal maintenance‑of‑effect study, and a long‑term open‑label safety extension, reflecting our confidence in osavampator’s potential to elevate the standard of care in MDD.

Osavampator was in-licensed from Takeda Pharmaceutical Company Limited (Takeda) in 2020, and we have exclusive worldwide rights to develop and commercialize osavampator, except in Japan, where Takeda has exclusive development and commercialization rights.

Direclidine (NBI-1117568)

Direclidine is an investigational oral agonist targeting the muscarinic M4 receptor, representing a novel mechanism for the treatment of schizophrenia and bipolar mania.

Schizophrenia is a severe, disabling disorder affecting approximately 24 million people globally and more than 3 million people in the U.S., and roughly one-third of patients do not respond adequately to current antipsychotics.

Current antipsychotics largely modulate dopamine receptors and can cause significant side effects;

by contrast, direclidine’s selective stimulation of M4 (a receptor involved in cholinergic neurotransmission) offers a new approach to treating psychosis and has the potential to help these patients and improve outcomes, and may avoid the safety and tolerability issues of commercially available antipsychotics and non-selective muscarinic agonists by not requiring coadministration with a peripheral muscarinic blocker.

We have initiated a comprehensive Phase 3 clinical program for direclidine in schizophrenia (with initial topline data expected in 2027), including studies evaluating efficacy in patients experiencing an acute relapse of schizophrenia and long-term safety.

In addition, we initiated a Phase 2 study evaluating direclidine in bipolar mania in the fourth quarter of 2025.

Direclidine was in-licensed from Nxera Pharma UK Limited (Nxera) in 2021, and we retain global rights.

Early-Stage Psychiatry Programs

Our early-stage psychiatry pipeline is focused on muscarinic receptor modulators and next-generation VMAT2 inhibitors – both areas where we have deep expertise.

We have initiated a Phase 2 study of NBI-1117570, a dual M1/M4 muscarinic agonist intended for schizophrenia and other psychiatric indications.

Based on its pharmacologic profile, we are also evaluating long‑acting injectable formulations of NBI‑1117570 that, if successful, could support development as a potential first‑in‑class long‑acting antipsychotic designed to improve medication adherence (an important factor, as non‑adherence is common in schizophrenia).

Another muscarinic agonist, NBI-1117569, is in Phase 1 development.

NBI-1117569 targets M1/M4 receptors and is initially aimed at Alzheimer’s disease-related psychosis – an area of immense unmet need, as no therapies are specifically approved for this condition.

It is estimated that over 6 million Americans live with Alzheimer’s, and neuropsychiatric symptoms (such as psychosis) pose significant challenges;

our muscarinic agonist programs could represent a new therapeutic class addressing these symptoms without the downsides of antipsychotic off-label use.

Additionally, we have advanced next-generation VMAT2 inhibitors NBI-1065890 into Phase 2 development and NBI-1140675 into Phase 1 development.

These compounds are engineered to have increased half-life, potency, and enhanced physiochemical properties relative to INGREZZA that may enable long-acting injectable administration.

The aim is to develop long-acting injectable treatments for disorders characterized by abnormal involuntary movements (VMAT2 is the target of INGREZZA, the first FDA-approved drug for the treatment of TD).

The new VMAT2 candidates could potentially treat a broader range of CNS conditions – such as additional mood or movement disorders – with less frequent dosing (e.g., monthly injections).

These programs underscore our strategy of continual innovation even on proven targets:

by delivering superior molecules, we can address patient needs such as convenience and adherence in chronic therapies.

Neurology Portfolio Highlights

Across our neurology pipeline, we focus on precision, mechanism-based approaches to epilepsy and movement disorders.

Our programs are anchored in clinically and genetically validated targets, such as sodium channels and muscarinic receptors, where we believe selective modulation of disease-relevant pathways can meaningfully improve outcomes for patients whose conditions are not adequately controlled with existing therapies.

Early-Stage Neurology Programs

Our early-stage neurology programs aim to broaden our impact on neurological diseases.

NBI-1076986 is a selective M4 receptor antagonist in Phase 1 development, being evaluated for potential use in movement disorders such as dystonia.

By selectively blocking the M4 muscarinic receptor, NBI-1076986 may normalize motor circuit activity in conditions of excessive cholinergic tone – an approach backed by preclinical models of dystonia and other hyperkinetic states.

NBI-1117567 is an investigational oral M1-preferring muscarinic agonist in Phase 1 development, being evaluated for the potential treatment of Alzheimer’s disease, with the goal of improving cognition.

NBI‑921355 is an investigational, selective inhibitor of voltage-gated sodium channels Na v 1.2 and Na v 1.6 in Phase 1 development for the potential treatment of certain types of epilepsy.

Na v 1.2 and Na v 1.6 are predominantly expressed in excitatory neurons, and selective inhibition of these channels is intended to reduce pathological neuronal hyperexcitability while sparing Na v 1.1, which is mainly expressed in inhibitory interneurons.

This mechanistic profile is designed to offer a more targeted approach than broad-spectrum sodium channel blockers and may translate into improved efficacy and tolerability for people living with epilepsy.

If successful, NBI‑921355 could provide a differentiated, mechanism-based treatment option for certain epilepsy syndromes that are not adequately controlled with existing antiseizure medications.

NBI‑921355 was in-licensed from Xenon Pharmaceuticals Inc.

in 2019, and we retain global rights.

Endocrinology Portfolio Highlights

Our endocrinology pipeline underscores our commitment to innovative science with real-world impact.

We take on conditions where patients have limited or suboptimal therapeutic options and aim to develop therapies that significantly advance care.

We have a legacy of leadership in corticotropin-releasing factor (CRF) biology, which has already yielded CRENESSITY (a CRF type 1 receptor (CRF-1) antagonist) as a first-in-class FDA-approved treatment of CAH – and the first new treatment for CAH in over 70 years.

Building on this foundation, we are advancing next-generation compounds targeting the CRF hormone pathways to address endocrine and metabolic diseases.

Early-Stage Endocrinology and Metabolic Programs

NBIP-01435 is an investigational, long-acting CRF-1 receptor antagonist peptide, administered as a subcutaneous injection, and is currently in Phase 1 development for the potential treatment of CAH.

CAH is a rare genetic disorder in which cortisol synthesis is impaired, leading to life-threatening adrenal crises and excessive androgen levels from birth.

Standard of care relies on supraphysiologic doses of glucocorticoids, which cause multiple well-established complications over time.

CRF-1 receptor antagonism offers a novel therapeutic approach:

by antagonizing CRF-1, NBIP-01435 aims to reduce the drive to produce excess androgens, thereby allowing more physiologic glucocorticoid dosing and better disease control.

In December 2024, the FDA approved our oral CRF-1 receptor antagonist (CRENESSITY) as a first-in-class treatment of CAH, validating this mechanism.

NBIP-01435, as a long-acting injectable, could further improve convenience and outcomes for CAH patients by providing sustained CRF-1 antagonism with less frequent dosing.

This program represents the first biologic (peptide) to emerge from our internal discovery collaboration (with Sentia Medical Sciences, Inc.) on novel peptide CRF receptor antagonists, highlighting our expansion into biologics to complement our small-molecule portfolio.

NBIP-2118 is a highly innovative program targeting the CRF type 2 receptor (CRF-2) for the treatment of obesity and related metabolic diseases.

NBIP-2118 is a CRF-2 selective agonist designed to engage a natural satiety pathway that may induce potent weight loss while preserving lean muscle mass – an issue with some current weight-loss drugs that mainly reduce fat but also muscle.

Preclinical studies have shown that activating CRF-2 may enhance metabolism and energy expenditure in a favorable manner.

We view NBIP-2118 as a potential first-in-class metabolic therapy, and plan to file an investigational new drug application (IND) with the FDA and advance NBIP-2118 into Phase 1 development in the first half of 2026.

Obesity remains a global epidemic with few options that achieve substantial, sustainable, and quality weight loss without surgery.

By leveraging the CRF-2 pathway (distinct from glucagon-like peptide-1 and other current targets), NBIP-2118 could potentially differentiate itself in this burgeoning field, including as a monotherapy, as an add-on or in combination with existing therapies, or as a follow-on or maintenance option for patients who do not respond to or do not achieve desired outcomes with current treatments.

This CRF-2 agonist initiative also exemplifies our strategy to apply our neuroscience expertise to systemic disorders (like metabolic disease) where neuroendocrine pathways play a key role.

Our confidence in this approach is bolstered by our internal know-how of over 30 years studying CRF biology and the success of our first CRF program in CAH.

Research and Discovery Engine and Ongoing Innovation

Underpinning all our programs is a reinvigorated research and discovery engine that we have built to be both innovative and efficient.

We have refined our discovery efforts to focus on genetically or clinically validated mechanisms of action, which improves our probability of success in the clinic.

At the same time, to maintain our leadership edge, we encourage the pursuit of first-in-class targets.

Our goal is to advance at least four new programs into Phase 1 and two programs into Phase 2 each year going forward.

This rapid cadence is enabled by internal investments in discovery (including medicinal chemistry, biologics, and translational sciences) and strategic business development when complementary opportunities arise.

Our scientists are also leveraging state-of-the-art discovery platforms – from structure-based drug design to human translational models – to generate the next wave of development candidates, particularly in areas such as neurology and immunology.

Our clinical development pipeline is both deep and differentiated, comprising multiple late-stage opportunities with near-term registration potential and a wide base of early-stage programs designed to drive growth into the next decade.

We believe our pipeline’s breadth, scientific sophistication, and strategic alignment position us to deliver sustainable innovation and therapeutic breakthroughs to relieve suffering for people with great needs.

Intellectual Property

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• INGREZZA, our highly selective VMAT2 inhibitor approved in the U.S.

for the treatment of tardive dyskinesia and of chorea associated with Huntington’s disease, is covered by 22 issued, FDA Orange Book-listed U.S.

for the treatment of TD and of chorea associated with Huntington’s disease, is covered by 22 issued, FDA Orange Book-listed U.S.

patents which are set to expire between 2027 and 2040.

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• CRENESSITY, a CRF1 receptor antagonist approved in the U.S.

for the treatment of CAH in adults and children, is covered by U.S.

patents among other patents set to expire between 2035 and 2041 (not including any potential patent term extensions), and pending patent applications, which, if issued, could expire at least as late as 2045.

for the treatment of CAH in adults and children, is covered by 4 issued, FDA Orange Book-listed U.S.

patents which are set to expire between 2035 and 2041 (not including any potential patent term extensions), and pending patent applications, which, if issued, could expire at least as late as 2046.

CRENESSITY has been granted seven years of orphan drug exclusivity by the FDA.

We also own, or have licensed rights to, patents covering our other products and earlier stage product candidates.

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If this protection is available, no competitor may use the original applicant’s data as the basis of a generic marketing application during the period of data and marketing exclusivity, which is measured from the date of marketing approval by the FDA or corresponding foreign regulatory authority.

This period of exclusivity is generally five years in the U.S., six years in Japan and eight years in the EU, with marketing exclusivity lasting an additional two years in the EU, except that for biologics, the period of exclusivity in the U.S.

This period of exclusivity is generally five years in the U.S., six years in Japan and eight years in the EU, with marketing exclusivity lasting an additional two years, plus another optional year, in the EU, except that for biologics, the period of exclusivity in the U.S.

is 12 years under the Biologics Price Competition and Innovation Act.

In addition, if granted orphan drug designation, certain of our product candidates, including, for example, crinecerfont, may also be eligible for marketing exclusivity in the U.S.

In addition, if granted orphan drug designation, certain of our product candidates may also be eligible for marketing exclusivity in the U.S.

for seven years and EU for 10 years.

Refer to Part I, Item 1A.

See Part I, Item 1A.

Risk Factors for a discussion of the challenges we may face in obtaining or maintaining patent and/or trade secret protection and Note 15 to the consolidated financial statements f or a description of our legal proceedings related to intellectual property matters.

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Such developments by others (including the development of generic equivalents) may render our product candidates or technologies obsolete or noncompetitive.

• INGREZZA competes with AUSTEDO ® (deutetrabenazine), marketed by Teva Pharmaceuticals Industries, for the treatment of tardive dyskinesia in adults and chorea associated with Huntington's disease.

• INGREZZA competes with AUSTEDO (deutetrabenazine), marketed by Teva Pharmaceuticals Industries, for the treatment of TD in adults and chorea associated with Huntington's disease.

A once-daily dosing of AUSTEDO (AUSTEDO XR) was introduced in February 2023.

Additionally, there are a number of commercially available medicines used to treat tardive dyskinesia off-label, such as XENAZINE ® (tetrabenazine) and generic equivalents, and various antipsychotic medications (e.g., clozapine), anticholinergics, benzodiazepines (off-label), and botulinum toxin.

Additionally, there are a number of commercially available medicines used to treat TD off-label, such as XENAZINE (tetrabenazine) and generic equivalents, and various antipsychotic medications (e.g., clozapine), anticholinergics, benzodiazepines (off-label), and botulinum toxin.

In addition, there are several programs in clinical development by other companies targeting Huntington's disease.

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• Our investigational treatments for potential use in schizophrenia and depression may in the future compete with several development-stage programs being pursued by other companies.

In addition, there are a number of different anti-psychotic and anti-depressant medications currently used in these patient populations.

• Our investigational treatments for potential use in neurology, neuroendocrinology and neuropsychiatry may in the future compete with numerous approved products and development-stage programs being pursued by several other companies.

In addition, there are a number of different anti-psychotic, including the muscarinic agonist COBENFY, and anti-depressant medications currently used in these patient populations.

• Our investigational treatments for potential use in neurology, psychiatry, endocrinology, obesity and related metabolic diseases, and immunology may in the future compete with numerous approved products and development-stage programs being pursued by several other companies.

Collaboration and License Agreements

Refer to Note 2 to the consolidated financial statements for more information on our significant collaboration and license agreements.

See Note 11 to the consolidated financial statements for more information on our significant collaboration and license agreements.

Government Regulation

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corporations and their foreign affiliates, which are intended to prevent the diversion of corporate funds to the payment of bribes and other improper payments.

Similar laws exist in other countries, such as the United Kingdom (UK) or in EU member states, that restrict improper payments to public and private parties.

Similar laws exist in other countries, such as the United Kingdom (UK) or in EU member states, which restrict improper payments to public and private parties.

Many countries have laws prohibiting these types of payments within the respective country.

In addition to these anti-corruption laws, we are subject to import and export control laws, tariffs, trade barriers, economic sanctions, and regulatory limitations on our ability to operate in certain foreign markets.

In August 2025, we received a civil investigative demand from the U.S.

Department of Justice (DOJ) requesting certain documents and information related to our sales and marketing of INGREZZA.

We are cooperating with the DOJ's request.

No assurance can be given as to the timing or outcome of the DOJ's investigation.

Failure to comply with these laws, where applicable, can result in significant penalties, including the imposition of significant civil, criminal and administrative penalties, damages, monetary fines, disgorgement, imprisonment, possible exclusion from participation in Medicare, Medicaid and other federal and equivalent foreign healthcare programs, and additional reporting requirements and regulatory oversight, any of which could adversely affect our ability to operate our business and our results of operations.

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Typically, clinical evaluation involves a time consuming and costly multi-phase process.

Phase 1 Clinical trials are conducted with a small number of subjects to determine the early safety profile, maximum tolerated dose and pharmacokinetic properties of the product in human volunteers or in patients with the target disease.

Phase 2 Clinical trials are conducted with groups of patients afflicted with a specific disease in order to determine preliminary efficacy, optimal dosages and expanded evidence of safety.

Phase 3 Larger, multi-center, comparative clinical trials are conducted with patients afflicted with a specific disease in order to determine safety and efficacy as primary support for regulatory approval by the FDA, the European Commission, or equivalent foreign authorities, to market a product candidate for a specific disease.

Clinical trials are conducted with a small number of subjects to determine the early safety profile, maximum tolerated dose and pharmacokinetic properties of the product in human volunteers or in patients with the target disease.

Clinical trials are conducted with groups of patients afflicted with a specific disease in order to determine preliminary efficacy, optimal dosages and expanded evidence of safety.

Larger, multi-center, comparative clinical trials are conducted with patients afflicted with a specific disease in order to determine safety and efficacy as primary support for regulatory approval by the FDA, the European Medicines Agency (EMA) and European Commission, or equivalent foreign authorities, to market a product candidate for a specific disease.

The FDA closely monitors the progress of each of the three phases of clinical trials that are conducted in the U.S.

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Before approving an NDA, the FDA typically will inspect the facility or facilities where the product is manufactured.

The FDA will not approve an application unless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to assure consistent production of the product within required specifications.

The FDA will not approve an application unless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to ensure consistent production of the product within required specifications.

Additionally, before approving an NDA, the FDA may inspect one or more clinical trial sites to assure compliance with Good Clinical Practice (GCP) requirements.

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Third-party payors are increasingly challenging the price, examining the medical necessity and reviewing the cost-effectiveness of drug products and medical services, in addition to questioning their safety, efficacy and clinical appropriateness.

Such payors may limit coverage to specific drug products on an approved list, also known as a formulary, which might not include all of the FDA-approved drugs for a particular indication.

Such payors may limit coverage to specific drug products on an approved list, also known as a

formulary, which might not include all of the FDA-approved drugs for a particular indication.

We may need to conduct expensive pharmaco-economic studies in order to demonstrate the medical necessity and cost-effectiveness of our products, in addition to the costs required to obtain the FDA approvals.

Nonetheless, our products or product candidates, including INGREZZA, may not be considered medically necessary or cost-effective.

Nonetheless, our products or product candidates, including INGREZZA and CRENESSITY, may not be considered medically necessary or cost-effective.

Moreover, the process for determining whether a third-party payor will provide coverage for a drug product may be separate from the process for setting the price of a drug product or for establishing the reimbursement rate that such a payor will pay for the drug product.

A payor’s decision to provide coverage for a drug product does not imply that an adequate reimbursement rate will be approved.

Further, one payor’s determination to provide coverage for a drug product does not assure that other payors will also provide coverage for the drug product.

Further, one payor’s determination to provide coverage for a drug product does not ensure that other payors will also provide coverage for the drug product.

Adequate third-party payor reimbursement may not be available to enable us to maintain price levels sufficient to realize an appropriate return on our investment in product development.

The marketability of any product or product candidates for which we or our collaborators receive regulatory approval for commercial sale may suffer if third-party payors fail to provide coverage and adequate reimbursement.

In addition, emphasis on managed care in the U.S.

has increased and we expect will continue to increase the pressure on pharmaceutical pricing.

Coverage policies and third-party payor reimbursement rates may change at any time.

Even if favorable coverage and reimbursement status is attained for one or more products for which we receive regulatory approval, less favorable coverage policies and reimbursement rates may be implemented in the future.

Healthcare Reform Measures

and some foreign jurisdictions have enacted a number of legislative and regulatory proposals to change the healthcare system in ways that could affect our ability to sell our products profitably.

In the U.S., the pharmaceutical industry and the cost of prescription drugs has been a continuous focus of these efforts and has been significantly affected by major legislative initiatives.

Most recently, in August 2022, the Inflation Reduction Act of 2022 (IRA) was signed into law, which, among other things, (1) directs the Secretary of the U.S.

Department of Health and Human Services (HHS) to negotiate the price of certain high-expenditure, single-source drugs and biologics covered under Medicare, (2) redesigns the Medicare Part D prescription drug benefit to lower patient out-of-pocket costs and increase manufacturer liability and (3) requires drug manufacturers to pay rebates on drugs whose prices increase greater than the rate of inflation.

The IRA also extends enhanced subsidies for individuals purchasing health insurance coverage in the ACA marketplaces through plan year 2025 and eliminated the “donut hole” under the Medicare Part D program in 2025 by significantly lowering the beneficiary maximum out-of-pocket cost to $2,000 through a newly established manufacturer discount program.

These provisions took effect progressively starting in 2023.

On August 15, 2024, HHS announced the negotiated prices of the first 10 drugs that were subject to price negotiations, although the Medicare drug price negotiation program is currently subject to legal challenges.

On January 17, 2025, HHS announced its selection of fifteen additional drugs covered under Part D for negotiation in 2025 (for initial price applicability year 2027).

Certain high-expenditure Part B and Part D drugs/biologics will be selected for negotiation in 2026 (for initial price applicability year 2028) and annually thereafter.

AUSTEDO and AUSTEDO XR, marketed by Teva Pharmaceuticals Industries, have been selected for the Medicare drug negotiation program in 2025 (for initial price applicability year 2027).

If the negotiation program results in a decrease in the price of AUSTEDO or AUSTEDO XR, it may result in increased competitive pressure on INGREZZA.

The overall impact of any potential negotiated priced reduction of AUSTEDO or AUSTEDO XR on INGREZZA revenues is inherently uncertain and difficult to predict.

Each year up to twenty (20) products will be selected by HHS for the Medicare Drug Price Negotiation Program.

Products subject to the Medicare Drug Price Negotiation Program are expected to experience a significant reduction in reimbursement from the Medicare program on a per unit basis.

AUSTEDO and AUSTEDO XR, marketed by Teva Pharmaceuticals Industries, were selected for the Medicare drug negotiation program in 2025 (for initial price applicability year 2027) and CMS has announced a negotiated maximum fair price (MFP) for these products that is lower than their pre-negotiation prices.

Lower negotiated prices for AUSTEDO and AUSTEDO XR may increase competitive pressures on INGREZZA, including heightened pricing pressure and potential adverse effects on formulary coverage.

While the Medicare drug negotiation program targets high-expenditure drugs/biologics that have been on the market for several years without generic or biosimilar competition, we were notified in January 2025 that INGREZZA qualifies for the small biotech exception, which provides an exemption from selection for price negotiation until 2027 (for initial price applicability year 2029, pursuant to which negotiated pricing would go into effect, if selected).

Additionally, on January 1, 2025, the Centers for Medicare & Medicaid Services (CMS) implemented those provisions of the IRA establishing a new Medicare Part D manufacturer discount program.

Additionally, on January 1, 2025, CMS implemented those provisions of the IRA establishing a new Medicare Part D manufacturer discount program.

Under this discount program and subject to certain exceptions, manufacturers must give a 10 percent discount on Part D program drugs in the initial coverage phase, and a 20 percent discount on Part D drugs when the beneficiary enters the catastrophic coverage phase (the phase after the patient incurs costs above the initial phase out-of-pocket threshold, which is $2,000).

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During this phase-in period, such manufacturers would pay a lower percentage discount on Medicare Part D program drugs.

In April 2024, the Company was notified by CMS that it qualified as a “specified small manufacturer” and will receive the discount phase-in discussed above for INGREZZA.

In April 2024, we were notified by CMS that it qualified as a “specified small manufacturer” and will receive the discount phase-in discussed above for INGREZZA.

INGREZZA is reimbursed under Medicare Part D, and increased discounts could impact INGREZZA revenues, while also having an industry-wide impact on the cost of other Part D program drugs such as AUSTEDO and AUSTEDO XR.

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The loss or potential loss of these exemptions, including as a result of a third party acquiring us, could have an adverse impact on our business.

In addition, emphasis on managed care in the U.S.

has increased and we expect will continue to increase the pressure on pharmaceutical pricing.

Coverage policies and third-party payor reimbursement rates may change at any time.

Healthcare Reform Measures

and some foreign jurisdictions have enacted a number of legislative and regulatory proposals to change the healthcare system in ways that could affect our ability to sell our products profitably.

In the U.S., the pharmaceutical industry and the cost of prescription drugs has been a continuous focus of these efforts and has been significantly affected by major legislative initiatives.

The most significant prior revisions to federal law governing the pharmaceutical industry and prescription drug pricing were enacted through the March 2010 Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act (collectively, the ACA).

This law was intended to broaden access to health insurance by reducing the number of uninsured persons, reducing or constraining the growth of healthcare spending, enhancing remedies against fraud and abuse, adding transparency requirements for the healthcare and health insurance industries, imposing taxes and fees on the health industry and imposing additional health policy reforms.

Congress is considering proposed legislation intended to further reduce healthcare costs with alternatives to replace the expiring ACA subsidies.

The IRA also extends enhanced subsidies for individuals purchasing health insurance coverage in the ACA marketplaces through plan year 2025 and eliminated the "donut hole" under the Medicare Part D program in 2025 by significantly lowering the beneficiary maximum out-of-pocket cost to $2,000 through a newly established manufacturer discount program.

Additionally, on July 4, 2025, the One Big Beautiful Bill Act (OBBBA) was signed into law, which is expected to reduce Medicaid spending and enrollment by implementing work requirements for some beneficiaries, capping state-directed payments, reducing federal funding, and limiting provider taxes used to fund the program.

The OBBBA also narrows access to ACA marketplace exchange enrollment and declines to extend the ACA enhanced advanced premium tax credits, set to expire at the end of 2025, which, among other provisions in the law, are anticipated to reduce the number of Americans with health insurance.

We expect that these health reform measures may result in more rigorous coverage criteria and lower reimbursement for prescription drugs, as well as result in additional downward pressure on any price that we receive for any approved product.

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These changes include, among others, aggregate reductions to Medicare payments to providers of up to 2% per fiscal year pursuant to the Budget Control Act of 2011, which began in 2013 and, due to subsequent legislative amendments, including the Investment and Jobs Act, will remain in effect through 2032.

The current administration is pursuing policies to reduce regulations and expenditures across government including at HHS, the FDA, CMS and related agencies.

These actions, presently directed by executive orders or memoranda from the Office of Management and Budget, may propose policy changes that create additional uncertainty for our business.

For example, the current administration has announced agreements with several pharmaceutical companies that require the drug manufacturers to offer, through a direct-to-consumer platform, U.S.

patients and Medicaid programs prescription drug Most-Favored Nation pricing equal to or lower than those paid in other developed nations, with additional mandates for direct-to-patient discounts and repatriation of foreign revenues.

Other recent actions, for example, include (1) directives to reduce agency workforces and cut programs;

(2) directing HHS and other agencies to lower prescription drug costs through a variety of initiatives, including by improving upon the Medicare Drug Price Negotiation Program and establishing Most-Favored-Nation pricing for pharmaceutical products;

(3) imposing tariffs on imported pharmaceutical products;

and (4) as part of the Make America Healthy Again (MAHA) Commission’s recent Strategy Report, working across government agencies to increase enforcement on direct-to-consumer pharmaceutical advertising.

These actions and policies may significantly reduce U.S.

drug prices, potentially impacting manufacturers’ global pricing strategies and profitability, while increasing their operational costs and compliance risks.

In June 2024, the U.S.

Supreme Court’s Loper Bright decision greatly reduced judicial deference to regulatory agencies, which could increase successful legal challenges to federal regulations affecting our operations.

Congress may introduce and ultimately pass health care related legislation that could impact the drug approval process and make changes to the Medicare Drug Price Negotiation Program created under the IRA.

At the state level, legislatures have increasingly passed legislation and implemented regulations designed to examine and/or control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.

For example, on January 5, 2024, the FDA approved Florida’s Section 804 Importation Program (SIP) proposal to import certain drugs from Canada for specific state healthcare programs.

It is unclear how this program will be implemented, including which drugs will be chosen, and whether it will be subject to legal challenges in the U.S.

Other states have also submitted SIP proposals that are pending review by the FDA.

Any such approved importation plans, when implemented, may result in lower drug prices for products covered by those programs.

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Clinical Trials in the EU

In the EU, the Clinical Trials Regulation (EU) No 536/2014 (CTR) entered into application on January 31, 2022 and became effective for all clinical trials on January 31, 2025, repealing and replacing the former Clinical Trials Directive 2001/20 (CTD).

The regulation introduces a streamlined application procedure via a single entry point, the “EU portal”, the Clinical Trials Information System (CTIS);

In the EU, clinical trials are governed by the Clinical Trials Regulation (EU) No 536/2014 (CTR) which entered into application on January 31, 2022, repealing and replacing the former Clinical Trials Directive 2001/20 (CTD).

The CTR became effective for all clinical trials on January 31, 2025.

The regulation introduces a streamlined application procedure via a single entry point, the Clinical Trials Information System (CTIS);

a single set of documents to be prepared and submitted for the application as well as simplified reporting procedures for clinical trial sponsors.

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The European Commission or the competent authorities of the EU Member States may decide on justified grounds relating to pharmacovigilance, to proceed with one further five-year renewal period for the MA.

Once subsequently definitively renewed, the MA shall be valid for an unlimited period.

Once renewed, the MA shall be valid for an unlimited period.

Any authorization which is not followed by the actual placing of the medicinal product on the EU market (for a centralized MA) or on the market of the authorizing EU Member State within three years after authorization ceases to be valid (the so-called sunset clause).

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Upon grant of a marketing authorization, orphan medicinal products are entitled to a ten-year period of market exclusivity for the approved therapeutic indication, which means that the EMA cannot accept another marketing authorization application or accept an application to extend for a similar product and the European Commission cannot grant a marketing authorization for the same indication for a period of ten years.

The period of market exclusivity is extended by two years for orphan medicinal products that have also complied with an agreed PIP.

The period of market exclusivity is extended by two years for orphan medicinal products that have also complied with an agreed Pediatric Investigation Plan (PIP).

The period of market exclusivity may, however, be reduced to six years if, at the end of the fifth year, it is established that the product no longer meets the criteria on the basis of which it received orphan medicinal product destination.

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However, the details are governed by regulations in individual EU Member States and can differ from one country to another.

Brexit and the Regulatory Framework in the UK

The UK’s withdrawal from the EU on January 31, 2020, commonly referred to as Brexit, has changed the regulatory relationship between the UK and the EU.

The Medicines and Healthcare products Regulatory Agency (MHRA) is now the UK’s standalone regulator for medicinal products and medical devices.

Great Britain (England, Scotland and Wales) is now a third country to the EU.

Northern Ireland continues to follow the EU regulatory rules.

Regulatory Framework in the UK

The Medicines and Healthcare products Regulatory Agency (MHRA) is the UK’s standalone regulator for medicinal products and medical devices.

The UK regulatory framework in relation to clinical trials is governed by the Medicines for Human Use (Clinical Trials) Regulations 2004, as amended, which is derived from the CTD, as implemented into UK national law through secondary legislation.

In October 2023, the MHRA announced a new Notification Scheme for clinical trials which enables a more streamlined and risk-proportionate approach to initial clinical trial applications for Phase 4 and low-risk Phase 3 clinical trial applications.

On April 28, 2026, the Medicines for Human Use (Clinical Trials) (Amendment) Regulations 2024 will enter into application to modernize the UK clinical trials framework and introduce significant changes.

Marketing authorizations in the UK are governed by the Human Medicines Regulations (SI 2012/1916), as amended.

This legislation includes procedures to prioritize access to new medicines that will benefit patients, including a 150-day assessment route, a rolling review procedure and the International Recognition Procedures (IRP) which entered into application on January 1, 2024.

Since January 1, 2024, the MHRA may rely on the IRP when reviewing certain types of marketing authorization applications.

There is no pre-marketing authorization orphan designation for medicinal products in the UK.

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The criteria are essentially the same as those in the EU but have been tailored for the market.

Human Capital

Our Employees

We have grown to a team of approximately 1,800 employees as of December 31, 2024, primarily employed in the U.S.

Our highly qualified and experienced team, which includes scientists, physicians, and professionals across sales, marketing, manufacturing, regulatory, finance, and other essential functions are critical to our success.

We also leverage temporary workers to provide flexibility for our business needs.

During 2024, we added more than 400 new employees to our team.

We expect to add additional employees in 2025 with a focus on expanding our research and development organization.

We continually evaluate our business needs and opportunities and balance in-house with external expertise and capacity.

Currently, we rely on third-party contract manufacturers.

The success of our human capital management investments is evidenced by our low employee turnover, a number which is regularly reviewed by our Board of Directors as part of their oversight of our human capital strategy.

In recognition of our efforts, in 2024, we were ranked #7 in Fortune Best Workplaces in Biopharma TM and named Company of the Year:

Specialty Pharma/Biotech in the PM360 Trailblazer Awards.

Employee Engagement, Talent Development & Benefits

We believe that our future success largely depends upon our continued ability to attract and retain highly skilled employees.

We provide our employees with competitive salaries and bonuses, opportunities for equity ownership, development programs that enable continued learning and growth and a robust employment package that promotes well-being across all aspects of their lives, including healthcare, retirement planning and paid time off.

As part of our promotion and retention efforts, we also invest in ongoing leadership development programs as well as offer tuition reimbursement.

In addition, we regularly conduct employee surveys to gauge employee engagement and identify areas of focus.

Diversity & Inclusion

Much of our success is rooted in the diversity of our teams and our commitment to inclusion.

We value diversity at all levels and continue to focus on extending our diversity and inclusion initiatives across our entire workforce.

We believe that our business benefits from the different perspectives a diverse workforce brings, and we pride ourselves on having a strong, inclusive and positive culture based on our shared mission and values.

Human Capital Management

Employee Profile

Since 2017, the number of our full-time employees (which are primarily based in the U.S.) has grown from approximately 200 to approximately 2,000 as of December 31, 2025.

We also rely on a number of highly skilled consultants and contingent workers who support our commercialization and research and development efforts.

In October 2025, we announced the planned expansion of the INGREZZA and CRENESSITY sales teams to maximize our commercial momentum.

The expansion is expected to be completed by the end of the first quarter of 2026.

In addition, we expect to continue making substantial investments in our research and development personnel to support our expansion into the development of biologics, including peptides, proteins, antibodies, conjugates, and gene therapies.

Culture and Engagement

Our core values of Passion, Integrity, Collaboration, Innovation, and Tenacity are the foundation on which we have deliberately cultivated a mission-driven culture centered on a simple purpose:

to relieve suffering for people with great needs.

To ensure we foster a culture where every individual feels respected, valued, and able to contribute as their authentic self, we listen to our employees and act on their feedback.

We conduct a confidential employee engagement survey annually to assess employee engagement and identify the key drivers of attrition and retention, thereby enabling us to develop targeted, data-driven action plans that protect our talent assets and support a high-performance culture.

Senior management and our Board of Directors review the results of our engagement initiatives at least annually, underscoring that human capital is a strategic priority at Neurocrine Biosciences.

We are proud that our efforts have earned Neurocrine Biosciences recognition as an employer of choice – we ranked among the Fortune “Best Workplaces in Biopharma” Top 10 for the third consecutive year (2023-2025) and continue to strive for workplace excellence.

Talent Development and Growth

Developing the skills and careers of our employees is critical to our long-term success.

We provide a range of opportunities and resources to help employees learn, grow, and lead.

All new hires undergo a structured onboarding program that immerses them in our culture and core values.

In addition, we offer continuous learning through professional development courses ranging from technical training, competency-based workshops, and leadership development programs facilitated by external partners who are experts in their respective fields.

By investing in our people’s growth, we not only increase engagement but also ensure that Neurocrine Biosciences has the skills and leadership needed to sustain our innovation and growth.

Compensation and Benefits

Our compensation philosophy is to attract, motivate, and retain top talent in a competitive industry by offering market-driven, performance-oriented pay and comprehensive benefits.

We provide competitive base salaries and annual performance bonuses tied to both company and individual performance.

All regular employees are eligible for our annual cash bonus program, which is funded based on corporate goal attainment and allocated in line with individual contributions.

We also believe in employees sharing in our long-term success:

equity grants (such as stock options or restricted stock units) are a key component of compensation for the majority of our employees, not just executives.

We make refresh equity grants on a periodic basis and new-hire grants for eligible employees, aligning their interests with shareholders.

Beyond pay, our benefits package is designed to support the well-being of our employees and their families:

we offer comprehensive health insurance (medical, dental, and vision plans), company-paid life and disability insurance, a 401(k) retirement plan with company matching contributions (dollar-for-dollar up to 6% of salary), and a generous paid time off policy (including vacation, sick leave, holidays, and floating days for personal or cultural observances).

Additional benefits include an enhanced family leave policy providing 12 weeks of paid parental leave for all new parents (maternal, paternal, and adoptive), flexible spending accounts, an Employee Stock Purchase Plan (ESPP) enabling employees to buy shares of our common stock at a discount, and free employee assistance programs offering confidential counseling and work-life support services.

Overall, our goal is to reward employees fairly for their contributions and provide benefits that promote their health, safety, and work-life balance.

Corporate Information

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Our telephone number is (858) 617-7600.

Our Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and amendments to reports filed pursuant to Sections 13(a) and 15(d) of the Securities Exchange Act of 1934, as amended, are available free of charge on our website at www.neurocrine.com , as soon as reasonably practicable after such reports are available on the Securities and Exchange Commission (SEC) website at www.sec.gov .

Additionally, copies of our Annual Report will be made available, free of charge, upon written request.

Our Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and amendments to those reports filed or furnished pursuant to Sections 13(a) and 15(d) of the Securities Exchange Act of 1934, as amended, are available free of charge on our website at www.neurocrine.com , as soon as reasonably practicable after such reports are available on the Securities and Exchange Commission (SEC) website at www.sec.gov .

Additionally, copies of our Annual Report to Security Holders will be made available, free of charge, upon written request.

Information found on, or accessible through, our website is not a part of, and is not incorporated into, this Annual Report on Form 10-K.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 1. Business