Neurocrine Biosciences Presents New Two-Year CRENESSITY® (crinecerfont) Data Showing Sustained Glucocorticoid Dose Reductions While Maintaining Androgen Control in Adults with Classic Congenital Adrenal Hyperplasia

Rhea-AI Summary

Neurocrine Biosciences (Nasdaq: NBIX) presented two-year Phase 3 CAHtalyst Adult data showing sustained glucocorticoid (GC) dose reductions with CRENESSITY (crinecerfont) in adults with classic congenital adrenal hyperplasia.

At Month 24, 69% (103/149) reached physiologic GC dosing (≤11 mg/m2/day HCe); mean daily dose fell from 17.6 to 10.6 mg/m2/day HCe (−38%). Study retention exceeded 80% at two years with no new safety signals reported.

Positive

• 69% of participants (103/149) achieved physiologic GC dose (≤11 mg/m2/day HCe) at Month 24
• Mean daily GC dose fell from 17.6 to 10.6 mg/m2/day HCe, a −38% change from baseline
• Study retention >80% at two years with no new safety signals observed

Negative

• 31% of participants (46/149) did not reach a physiologic GC dose by Month 24
• Mean GC dose at Month 24 (10.6 mg/m2/day HCe) remains close to the physiologic threshold

News Market Reaction – NBIX

-0.75%

-0.75% Session close to close

In the Apr 23 session, NBIX declined 0.75%, reflecting a mild negative market reaction.

Data tracked by StockTitan Argus on the day of publication.

Market Context

This announcement highlights two-year CRENESSITY data in classic congenital adrenal hyperplasia, wit...

Analysis

This announcement highlights two-year CRENESSITY data in classic congenital adrenal hyperplasia, with around 70% of adults achieving physiologic glucocorticoid dosing, mean doses reduced to 10.6 mg/m²/day, and no new safety signals. It extends earlier one-year results and supports a durable efficacy and safety profile. In context of recent clinical and strategic milestones, investors may watch future updates on broader clinical endpoints, regulatory developments, and real-world adoption trends for CRENESSITY within Neurocrine’s endocrinology portfolio.

Key Figures

Patients at physiologic GC dose: ≈70% of adult patients Dexamethasone discontinuation: 75% of patients Physiologic GC dose threshold: ≤11 mg/m²/day HCe +5 more

8 metrics

Patients at physiologic GC dose ≈70% of adult patients CRENESSITY-treated adults at two years

Dexamethasone discontinuation 75% of patients Adults originally taking dexamethasone transitioned off this treatment

Physiologic GC dose threshold ≤11 mg/m²/day HCe Definition of physiologic glucocorticoid dosing in study

Month 24 physiologic dosing 69% (103/149) participants Participants at physiologic GC dose at Month 24

Hydrocortisone dose reduction 62% (37/60) patients Patients on >2 hydrocortisone doses/day able to eliminate a dose

Mean GC dose baseline 17.6 mg/m²/day HCe Observed mean daily GC dose at baseline

Mean GC dose Month 24 10.6 mg/m²/day HCe Observed mean daily GC dose at Month 24

Mean GC dose change -38% Mean % change from baseline in GC dose at Months 18 and 24

Historical Context

5 past events · Latest: Apr 14 (Neutral)

Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
Apr 14	Earnings call scheduling	Neutral	+0.5%	Set date and time for Q1 2026 earnings release and call.
Apr 14	Clinical real-world data	Positive	+3.5%	Claims analysis showed greater treatment persistence with INGREZZA vs AUSTEDO XR.
Apr 06	M&A acquisition deal	Positive	+0.7%	Announced Soleno acquisition, adding VYKAT XR and expanding rare-disease portfolio.
Mar 26	Clinical guidance update	Positive	+1.0%	Presented expert consensus recommendations for tardive dyskinesia in long-term care.
Mar 17	Executive leadership change	Neutral	+0.0%	Promoted Andrew Ratz to Chief Technical Operations Officer to support expansion.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Pattern Detected

Recent NBIX news over the past month has generally coincided with small, positive price reactions across clinical, strategic, and corporate updates.

Recent Company History

Over the last month, Neurocrine has announced an acquisition of Soleno, multiple INGREZZA data presentations, and corporate updates, with each item followed by modestly positive or flat price reactions (e.g., moves of 0.48%, 3.55%, 0.67%, and 0.98%). The current CRENESSITY two‑year CAH data extend this pattern of clinically oriented news supporting the company’s endocrinology and rare‑disease franchise, adding long‑term efficacy and safety evidence alongside prior real‑world and strategic developments.

Key Terms

glucocorticoid, androgen, adrenocorticotropic hormone, congenital adrenal hyperplasia, +3 more

7 terms

glucocorticoid medical

"demonstrating sustained, substantial reductions in glucocorticoid (GC) doses in adults"

A glucocorticoid is a type of steroid hormone—produced naturally by the body and also made as a medicine—that quiets inflammation and helps control how the body uses energy and responds to stress. Investors watch glucocorticoids because they are widely used drugs whose effectiveness, side effects and regulatory approval or supply issues can drive sales, affect healthcare costs and change demand for related treatments, much like a widely used tool that can both fix a problem and create new ones.

androgen medical

"to control adrenal androgen and adrenocorticotropic hormone excess, exposing patients"

Androgens are a group of natural hormones, like testosterone, that act as chemical messengers to promote male physical traits, reproductive function, and tissue growth; they work like keys that unlock specific responses inside cells. Investors care because drugs or therapies that boost, block, or mimic androgens can drive sales, regulatory reviews, and patent value, and they often carry distinct safety and market risks that affect company prospects.

adrenocorticotropic hormone medical

"to control adrenal androgen and adrenocorticotropic hormone excess, exposing"

Adrenocorticotropic hormone (ACTH) is a signaling protein made by the pituitary gland that tells the adrenal glands to release cortisol and other stress-related hormones; think of it as a thermostat that triggers the body’s emergency response. For investors, ACTH matters because it is a target for diagnostic tests, drug development, and therapeutic treatments for adrenal and stress-related disorders, influencing regulatory approvals, clinical trial outcomes, and market demand for related medicines and assays.

congenital adrenal hyperplasia medical

"management of classic congenital adrenal hyperplasia has relied exclusively"

Congenital adrenal hyperplasia is a group of inherited disorders in which the adrenal glands lack an enzyme needed to make certain hormones, causing a chronic imbalance of cortisol, aldosterone and/or sex hormones. Think of it as a factory assembly line missing a key part, so the body overproduces some products and underproduces others, requiring lifelong monitoring or hormone treatment. For investors, it matters because diagnosis, ongoing therapy, newborn screening and potential new drugs or gene therapies can drive medical spending, regulatory approvals and market opportunity in endocrinology and rare disease care.

hydrocortisone equivalents medical

"physiologic GC dose (≤11 mg/m2/day hydrocortisone equivalents), with many"

Hydrocortisone equivalents express the strength of any corticosteroid drug as an equal dose of hydrocortisone, the standard reference. Think of it like converting different-sized batteries to a common AA-equivalent so you can compare power; investors use the conversion to judge dosing, safety margins, market demand for stronger or weaker formulations, and to compare clinical trial results or regulatory labeling across drugs.

supraphysiologic medical

"Chronic exposure to supraphysiologic GC doses is associated with cardiometabolic"

Supraphysiologic describes a dose, concentration or level of a drug, hormone or other substance that is higher than what the human body normally produces or maintains. For investors, this signals a potential for stronger effect but also greater safety concerns, side effects and regulatory scrutiny—think of turning a thermostat well above the usual setting to boost performance but risk overheating and damage.

phase 3 medical

"new two-year data from the Phase 3 CAHtalyst® Adult study demonstrating"

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

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• Approximately 70% of adult patients treated with CRENESSITY achieved and sustained physiologic‑range glucocorticoid dosing at two years
• 75% of patients originally taking dexamethasone transitioned off this treatment, enabling a more physiologic glucocorticoid regimen without compromising androgen control
• Findings reinforce durable efficacy and a favorable long‑term safety profile in largest interventional trial to date conducted in classic congenital adrenal hyperplasia

SAN DIEGO, April 22, 2026 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the first presentation of new two-year data from the Phase 3 CAHtalyst^® Adult study demonstrating sustained, substantial reductions in glucocorticoid (GC) doses in adults with classic congenital adrenal hyperplasia treated with CRENESSITY^® (crinecerfont), with approximately 70% of patients achieving GC doses within the physiologic range. These data build upon previously reported one-year results and were presented at the American Association of Clinical Endocrinology 2026 Annual Meeting in Las Vegas.

Chronic exposure to supraphysiologic GC doses is associated with cardiometabolic comorbidities, bone density reductions, mental health issues and other long-term health risks that contribute to the cumulative treatment burden faced by patients over their lifetimes. Reducing high-dose, long-term GC exposure represents one of the most important goals in the management of classic congenital adrenal hyperplasia (CAH).

"For decades, the management of classic congenital adrenal hyperplasia has relied exclusively on supraphysiologic glucocorticoid dosing to control adrenal androgen and adrenocorticotropic hormone excess, exposing patients to significant cumulative long‑term risks," said Sanjay Keswani, M.D., Chief Medical Officer, Neurocrine Biosciences. "These two‑year findings demonstrated that CRENESSITY provided durable androgen control while enabling meaningful reductions in glucocorticoid exposure. Importantly, these reductions were sustained over time without new safety or tolerability concerns, supporting CRENESSITY as a long‑term treatment option that advances the standard of care for people living with this complex condition."

At Month 24 of the study, 69% (103/149) of participants achieved a physiologic GC dose (≤11 mg/m²/day hydrocortisone equivalents), with many participants eliminating nonphysiologic GC types. Of participants originally taking dexamethasone (n=20), 75% switched to a dexamethasone-free regimen, while 62% (37/60) of patients taking more than two doses of hydrocortisone per day were able to eliminate a dose outright. GC dose reductions and regimen changes were achieved without worsening androstenedione levels relative to baseline, indicating that lowering the GC dose was not achieved at the expense of androgen control.

Measure	Baseline	Month 18	Month 24
Mean daily GC dose (mg/m²/day HCe*), observed	17.6	10.6	10.6
Mean % change from baseline in GC dose, observed	—	-38 %	-38 %
Participants achieving physiologic GC dose (≤11mg/m²/day HCe*), n/N (%)	0/182  (0%)	114/161 (71%)	103/149 (69%)

*HCe denotes hydrocortisone equivalents.

Long‑term treatment with CRENESSITY was generally well tolerated, with more than 80% study retention at two years and no new safety signals observed.

"Many years of supraphysiologic glucocorticoid exposure increase the risk for long-term health consequences, which include obesity, diabetes, reduced bone density and psychosocial struggles," said Richard J. Auchus, M.D., Ph.D., Professor of Internal Medicine and Pharmacology, University of Michigan Medical School and Principal Investigator for the CAHtalyst Adult study. "These sequelae significantly impact quality of life and commonly develop with traditional CAH treatment regimens. The two-year findings provide important information on the durable benefit of treatment with CRENESSITY. Providers can confidently incorporate this additional knowledge to guide their management of adult patients with CAH into the future."

Neurocrine will be sharing additional two-year data across clinical endpoints and outcomes at upcoming medical meetings.

Additional presentation at the American Association of Clinical Endocrinology 2026 Annual Meeting includes:

• Cardiometabolic Outcomes Associated with Chronic Supraphysiologic Glucocorticoid Exposure and Crinecerfont Treatment: 1-Year Results from CAHtalyst Adult

About Congenital Adrenal Hyperplasia 
Congenital adrenal hyperplasia (CAH) is a rare genetic condition that results in an enzyme deficiency that alters the production of adrenal steroid hormones, such as cortisol, aldosterone and adrenal androgens. Severe enzyme deficiency leads to an inability of the adrenal glands to produce enough cortisol and, in approximately 75% of cases, aldosterone. Because individuals with CAH are typically still able to produce androgens, the unused precursors that would normally be used to make cortisol instead result in the production of excess amounts of androgens. If left untreated, CAH can result in adrenal crisis and even death. 

Exogenous glucocorticoids (GCs) are necessary to correct the endogenous cortisol deficiency, but historically, doses higher than those needed for cortisol replacement (supraphysiologic) have been used to lower the elevated levels of adrenocorticotropic hormone (ACTH) and adrenal 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. Additionally, long-term treatment with supraphysiologic GCs may have psychological and cognitive impacts, such as changes in mood and memory. Adrenal androgen excess has been associated with abnormal bone growth and development in pediatric patients, female health problems such as excess facial hair growth and menstrual irregularities, in addition to cardiometabolic and fertility issues in both sexes. The symptoms of high ACTH may include testicular adrenal rest tumors (TARTs). 

About CRENESSITY^® (crinecerfont)
CRENESSITY is a potent and selective oral corticotropin-releasing factor type 1 receptor (CRF₁) antagonist that reduces and controls excess adrenocorticotropic hormone (ACTH) and adrenal androgens through a non-glucocorticoid (GC) mechanism for the treatment of classic congenital adrenal hyperplasia (CAH). Antagonism of CRF₁ receptors in the pituitary has been shown to decrease ACTH levels, which in turn decreases the production of adrenal androgens and potentially the symptoms associated with CAH. The robust clinical study data demonstrate that lowering adrenal androgen levels with CRENESSITY enables lower, more physiologic dosing of GCs to replace missing cortisol. 

CRENESSITY comes in capsules and an oral solution. For adults 18 years of age and older, the recommended dosage is 100 mg twice daily taken orally with a meal. For pediatric patients four to 17 years of age weighing less than 55 kg (121 lbs), the recommended dosage is based on body weight and is administered twice daily, taken orally with a meal. For pediatric patients weighing more than 55 kg (121 lbs), the recommended dosage is 100 mg twice daily taken orally with a meal. Healthcare providers can work with patients to determine the appropriate formulation for use depending on patient needs. Patients receiving CRENESSITY should continue GC therapy for cortisol replacement.  

About The CAHtalyst^® Studies
The Phase 3 CAHtalyst global registrational studies were designed to evaluate the safety, efficacy and tolerability of CRENESSITY^® in children and adults with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The CAHtalyst studies were the largest-ever clinical trial program in classic CAH, including 285 pediatric and adult patients.   

The CAHtalyst Pediatric study included 103 pediatric patients four to 17 years of age. The study tested two questions. The first question evaluated whether four weeks of CRENESSITY treatment could improve androgen control. The second question evaluated whether an additional 24 weeks of CRENESSITY treatment enabled customized glucocorticoid (GC) down-titration while androstenedione levels were maintained or improved.    

The CAHtalyst Adult study included 182 adult patients 18 to 58 years of age. Similarly, the first question of the study evaluated whether four weeks of CRENESSITY treatment could improve androgen control, and the second question evaluated whether an additional 20 weeks of CRENESSITY treatment enabled GC reduction to physiologic range while androstenedione levels were maintained or improved.   

Data from the CAHtalyst Phase 3 studies supported approval of CRENESSITY by the U.S. Food and Drug Administration in December 2024. The open-label extension treatment portions of both studies are ongoing.   

Important Information  

Approved Uses
CRENESSITY^® (crinecerfont) is a prescription medicine used together with glucocorticoids (steroids) to control androgen (testosterone-like hormone) levels in adults and children 4 years of age and older with classic congenital adrenal hyperplasia (CAH).  

IMPORTANT SAFETY INFORMATION  

Do not take CRENESSITY if you:  

Are allergic to crinecerfont, or any of the ingredients in CRENESSITY.  

CRENESSITY may cause serious side effects, including:   

Allergic reactions. Symptoms of an allergic reaction include tightness of the throat, trouble breathing or swallowing, swelling of the lips, tongue, or face, and rash. If you have an allergic reaction to CRENESSITY, get emergency medical help right away and stop taking CRENESSITY.  

Risk of Sudden Adrenal Insufficiency or Adrenal Crisis with Too Little Glucocorticoid (Steroid) Medicine. Sudden adrenal insufficiency or adrenal crisis can happen in people with congenital adrenal hyperplasia who are not taking enough glucocorticoid (steroid) medicine. You should continue taking your glucocorticoid (steroid) medicine during treatment with CRENESSITY. Certain conditions such as infection, severe injury, or shock may increase your risk for sudden adrenal insufficiency or adrenal crisis. Tell your healthcare provider if you get a severe injury, infection, illness, or have planned surgery during treatment. Your healthcare provider may need to change your dose of glucocorticoid (steroid) medicine.  

Before taking CRENESSITY, tell your healthcare provider about all of your medical conditions, including if you: are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.  

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins and herbal supplements.  

The most common side effects of CRENESSITY in adults include tiredness, headache, dizziness, joint pain, back pain, decreased appetite, and muscle pain.  

The most common side effects of CRENESSITY in children include headache, stomach pain, tiredness, nasal congestion, and nosebleeds.  

These are not all the possible side effects of CRENESSITY. Call your healthcare provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch at www.fda.gov/medwatch or call 1-800-FDA-1088.  

Dosage Forms and Strengths: CRENESSITY is available in 50 mg and 100 mg capsules, and as an oral solution of 50 mg/mL.  

Please see full Prescribing Information.  

About Neurocrine Biosciences, Inc.   
Neurocrine Biosciences is a leading biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, endocrine, psychiatric and immunological disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, classic congenital adrenal hyperplasia, endometriosis* and uterine fibroids,* as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders because you deserve brave science. For more information, visit neurocrine.com, and follow the company on LinkedIn, X, Facebook and YouTube. (*in collaboration with AbbVie)  

The NEUROCRINE BIOSCIENCES Logo, NEUROCRINE, YOU DESERVE BRAVE SCIENCE, CRENESSITY and CAHtalyst are registered trademarks of Neurocrine Biosciences, Inc.

Forward-Looking Statements 
In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements regarding the potential benefits to be derived from CRENESSITY for the treatment of classic congenital adrenal hyperplasia (CAH); the value and benefits CRENESSITY brings to patients with CAH, including its potential to enable patients to transition toward more physiologic glucocorticoid dosing; the ability of Neurocrine Biosciences to ensure patients have access to CRENESSITY; and whether the results from our clinical trials of CRENESSITY are indicative of real-world results. Factors that could cause actual results to differ materially from those stated or implied in the forward-looking statements include, but are not limited to, the following: risks and uncertainties as to whether the data described in this press release will be replicated in additional studies or will be predictive of efficacy or other clinical outcomes in subsequent clinical studies or real-world use of CRENESSITY; risks and uncertainties associated with Neurocrine Biosciences' business and finances in general, as well as risks and uncertainties associated with the commercialization of CRENESSITY, including the extent to which patients and physicians accept and adopt CRENESSITY; whether CRENESSITY receives adequate reimbursement from third-party payors; risks and uncertainties relating to competitive products and technological changes that may limit demand for CRENESSITY; risks associated with the Company's dependence on third parties for development and manufacturing activities related to CRENESSITY, and the ability of the Company to manage these third parties; risks that additional regulatory submissions for CRENESSITY may not occur or be submitted in a timely manner; risks that the FDA or other regulatory authorities may make adverse decisions regarding CRENESSITY; risks that post-approval CRENESSITY commitments or requirements may be delayed; risks that CRENESSITY may be precluded from commercialization by the proprietary or regulatory rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; risks and uncertainties relating to competitive products and technological changes that may limit demand for CRENESSITY; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's annual report on Form 10-K for the year ended December 31, 2025. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof other than required by law.  

© 2026 Neurocrine Biosciences, Inc. All Rights Reserved. CAP-CFT-US-0054   04/2026  

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SOURCE Neurocrine Biosciences, Inc.

FAQ

What did Neurocrine Biosciences (NBIX) announce about CRENESSITY two-year results on April 22, 2026?

They announced two-year Phase 3 CAHtalyst Adult data showing sustained GC dose reductions with CRENESSITY, with 69% reaching physiologic dosing. According to Neurocrine, mean daily GC dose decreased from 17.6 to 10.6 mg/m2/day HCe (−38%) and no new safety signals emerged.

How many patients on NBIX CRENESSITY achieved physiologic glucocorticoid dosing at Month 24?

At Month 24, 69% (103/149) of participants achieved ≤11 mg/m2/day HCe physiologic dosing. According to Neurocrine, this result occurred alongside a mean GC dose reduction from 17.6 to 10.6 mg/m2/day HCe and sustained retention over two years.

Did CRENESSITY maintain androgen control while reducing glucocorticoid doses in the NBIX study?

Yes, GC dose reductions were achieved without worsening androstenedione levels relative to baseline. According to Neurocrine, lowering GC doses did not compromise androgen control in the Phase 3 CAHtalyst Adult study at two years.

What safety and tolerability findings did Neurocrine report for CRENESSITY at two years (NBIX)?

The two-year data showed more than 80% study retention and no new safety signals. According to Neurocrine, long-term treatment with CRENESSITY was generally well tolerated across the Phase 3 CAHtalyst Adult population.

What changes occurred for patients originally taking dexamethasone in the NBIX CRENESSITY study?

Of participants originally on dexamethasone (n=20), 75% transitioned to a dexamethasone-free regimen by Month 24. According to Neurocrine, many participants also eliminated nonphysiologic GC types and reduced overall dosing while maintaining androgen control.