Neurocrine Biosciences Presents First Retrospective Case Series of CRENESSITY® (crinecerfont) in Patients with Classic Congenital Adrenal Hyperplasia Due to 11β‑Hydroxylase Deficiency at ENDO 2026

Rhea-AI Summary

Neurocrine Biosciences (Nasdaq: NBIX) reported the first retrospective case series of CRENESSITY (crinecerfont) in 15 pediatric and adult patients with classic congenital adrenal hyperplasia due to 11β‑hydroxylase deficiency, presented at ENDO 2026.

Androstenedione and adrenal precursors fell markedly, with median decreases of 92% in DOC, 95% in 11‑deoxycortisol, and 65% in androstenedione among patients with elevated baseline levels. Fourteen of 15 patients reduced glucocorticoid doses, and 2 of 5 on antihypertensives lowered or stopped these drugs. Hormone normalization was seen as early as one month, and blood pressure improved in adults. The case series is described as preliminary, early evidence supporting CRENESSITY’s efficacy and safety in this rare subtype, where the drug is already approved as an adjunct to glucocorticoids for classic CAH regardless of enzyme deficiency.

Positive

• Median hormone reductions after CRENESSITY: -92% DOC, -95% 11-deoxycortisol, -65% androstenedione
• 14 of 15 patients reduced total glucocorticoid dose after treatment initiation
• 2 of 5 patients on antihypertensives reduced or discontinued these medications
• Hormone precursor levels reached normal ranges and adult blood pressure improved
• CRENESSITY already approved as an adjunctive treatment for classic CAH across enzyme defects
• Results presented at ENDO 2026, increasing clinical visibility of CRENESSITY

Negative

• Evidence is based on a retrospective case series rather than a controlled prospective trial
• Clinical data for 11β-hydroxylase deficiency subtype derived from only 15 patients
• Findings are characterized as preliminary and early, indicating need for further exploration

News Market Reaction – NBIX

-1.94%

-1.94% Session close to close

In the Jun 16 session, NBIX declined 1.94%, reflecting a mild negative market reaction.

Data tracked by StockTitan Argus on the day of publication.

Market Context

This announcement highlights retrospective case‑series data for CRENESSITY in 15 classic CAH patient...

Analysis

This announcement highlights retrospective case‑series data for CRENESSITY in 15 classic CAH patients with 11β‑hydroxylase deficiency, showing large median reductions in key adrenal steroids (up to 95%) and glucocorticoid dose reductions in 14 of 15 patients. It extends evidence into a rarer CAH subtype that comprises about 5% of cases. In context with earlier ENDO 2026 CRENESSITY presentations, this reinforces a broader clinical narrative that investors may track across future, more definitive studies.

Key Figures

11-deoxycortisol reduction: -95% median 11-deoxycorticosterone reduction: -92% median Androstenedione reduction: -65% median +5 more

8 metrics

11-deoxycortisol reduction -95% median Patients with elevated 11‑deoxycortisol (11‑dF, n=7) after CRENESSITY initiation

11-deoxycorticosterone reduction -92% median Patients with elevated DOC (n=5) after CRENESSITY initiation

Androstenedione reduction -65% median Patients with elevated androstenedione (A4, n=3) after CRENESSITY initiation

Case series size 15 patients Pediatric (n=11) and adult (n=4) classic CAH due to 11β‑OHD

Glucocorticoid dose reduction 14 of 15 patients Patients reducing total glucocorticoid dose after starting CRENESSITY

Antihypertensive change 2 of 5 patients Patients able to reduce or discontinue antihypertensives on CRENESSITY

Subtype prevalence 5% of cases 11β‑hydroxylase deficiency share of classic CAH cases

Price change -1.81% NBIX 24h move before/around ENDO 2026 CRENESSITY case-series news

Historical Context

5 past events · Latest: Jun 08 (Positive)

Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
Jun 08	INGREZZA efficacy data	Positive	-0.1%	48-week INGREZZA KINECT 4 data showing 94% meaningful TD response.
Jun 08	INGREZZA functional data	Positive	-0.2%	INGREZZA improvements in TD patients with intellectual and developmental disabilities.
Jun 03	CRENESSITY ENDO preview	Positive	+6.6%	Announcement of multiple new two‑year CRENESSITY analyses for ENDO 2026.
May 26	Investor conferences	Positive	+0.1%	Participation in June investor conferences with executive presentations and webcasts.
May 18	Real-world INGREZZA data	Positive	-2.2%	Real‑world data showing functional improvements in mild tardive dyskinesia with INGREZZA.

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

Pattern Detected

Recent clinically positive updates have often been met with flat to negative next-day moves, with 3 divergences versus 2 alignments.

Recent Company History

Over the past month, NBIX has repeatedly highlighted data across its neurology and endocrine franchises. INGREZZA tardive dyskinesia updates on May 18 and June 8 showed strong functional and symptomatic benefits but saw slightly negative price reactions. In contrast, a June 3 preview of two‑year CRENESSITY data at ENDO 2026 coincided with a 6.63% gain. Today’s detailed CRENESSITY case series in 11β‑hydroxylase deficiency builds on that ENDO narrative of expanding evidence across classic CAH populations.

Key Terms

congenital adrenal hyperplasia, 11β‑hydroxylase deficiency, glucocorticoid, antihypertensive treatment, +4 more

8 terms

congenital adrenal hyperplasia medical

"classic congenital adrenal hyperplasia (CAH) due to 11β‑hydroxylase deficiency"

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.

11β‑hydroxylase deficiency medical

"classic congenital adrenal hyperplasia (CAH) due to 11β‑hydroxylase deficiency"

11β‑hydroxylase deficiency is a hereditary enzyme disorder that disrupts the normal production of adrenal hormones, causing an imbalance of sex hormones and blood pressure regulators. For investors it matters because the condition creates a predictable need for medical care, diagnostic testing, and therapies—similar to a clogged valve that forces a system to run abnormally—so advances in drugs, diagnostics, or screening can affect demand, costs and markets in endocrinology and rare-disease care.

glucocorticoid medical

"prior supraphysiologic glucocorticoid (GC) therapy"

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.

antihypertensive treatment medical

"Among patients receiving antihypertensive treatment, two of five were able to reduce"

Antihypertensive treatment are medicines and therapies used to lower high blood pressure and reduce the risk of heart attack, stroke and other complications; think of it as reducing pressure in an overfilled hose to prevent bursts. Investors care because demand, regulatory approvals, insurance coverage, safety and effectiveness directly affect sales, development costs and long‑term market potential for companies making or selling these treatments.

ACTH medical

"use of CRENESSITY to target ACTH and potentially improve hormonal control"

ACTH is a hormone produced by the pituitary gland that tells the adrenal glands to release cortisol, the body’s main stress-response hormone; think of it as a thermostat setting that controls how much cortisol is pushed into the bloodstream. Investors care because drugs, diagnostics, or devices that alter ACTH signaling or measure its levels can drive clinical trial results, regulatory approvals, and revenue shifts for companies in endocrinology and diagnostics.

17-Hydroxyprogesterone medical

"Long-term Crinecerfont Treatment Reduced ACTH and 17-Hydroxyprogesterone — Clinical Outcomes"

A naturally produced steroid that appears in blood as part of the body’s hormone-making process; think of it like a dashboard warning light that shows when a specific step in hormone production is blocked. Doctors measure 17-hydroxyprogesterone to diagnose and monitor hormone disorders and to judge whether therapies are working, so changes in its levels can influence demand for diagnostics, drug development programs, and regulatory decisions that matter to investors.

Cox Proportional Hazards Analysis technical

"a Cox Proportional Hazards Analysis from the CAHtalog Registry"

A cox proportional hazards analysis is a statistical method used to compare how quickly an outcome (like disease progression or death) occurs between groups while accounting for other influencing factors. Think of it as comparing two runners’ time to a finish line while adjusting for differences in shoe type or training; it tells investors whether a treatment or factor changes the risk or timing of an event and by how much, which can affect future revenue or liability expectations.

Delphi Panel technical

"A Modified Delphi Panel of U.S. Endocrinologists to Align on Minimum Clinically"

A Delphi panel is a structured process that gathers and refines opinions from a group of independent experts through multiple anonymous rounds of surveys and feedback, aiming to reach a clearer collective judgment. For investors it matters because it turns scattered expert views into a more reliable forecast or risk assessment—like repeatedly polling specialists and tuning answers until a clearer signal emerges—helping weigh prospects for new products, regulations, or market trends.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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• Androstenedione and other adrenal hormone levels in 11β‑hydroxylase-deficient patients improved substantially after initiation of CRENESSITY, with >90% median reductions in 11-deoxycortisol and 11-deoxycorticosterone
• Nearly all patients (14/15) reduced their total glucocorticoid dose with CRENESSITY, and 2 of 5 patients on antihypertensive medications reduced or discontinued these drugs
• Findings provide initial clinical insights in patients with classic congenital adrenal hyperplasia due to 11β-hydroxylase deficiency, a rare subtype not previously studied in clinical trials of CRENESSITY

SAN DIEGO, June 15, 2026 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced clinical findings from the first retrospective case series in pediatric and adult patients with classic congenital adrenal hyperplasia (CAH) due to 11β‑hydroxylase deficiency. This subtype was not previously studied in clinical trials of CRENESSITY^® (crinecerfont) and is the second most common form of classic CAH after 21-hydroxylase deficiency, accounting for approximately 5% of cases. The findings were presented at the Endocrine Society's annual meeting, ENDO 2026, in Chicago.

Like other forms of CAH, 11β‑hydroxylase deficiency (11β‑OHD) is characterized by cortisol deficiency and excess adrenal androgens. However, 11β‑OHD is uniquely associated with the accumulation of the adrenal steroid precursors 11-deoxycortisol (11-dF) and 11-deoxycorticosterone (DOC). These hormonal imbalances can contribute to distinct clinical features, including hypertension and other long‑term complications. In this retrospective case series, reductions in androstenedione (A4) were observed across all patients with elevated baseline levels following initiation of CRENESSITY. Steroid precursors also decreased, with values reaching within normal ranges, and blood pressure improved in adult patients.

"Patients with 11β‑hydroxylase deficiency represent a complex subgroup within classic congenital adrenal hyperplasia, with limited data available to guide treatment decisions for these individuals," said Sanjay Keswani, M.D., Chief Medical Officer, Neurocrine Biosciences. "This first case series provides preliminary clinical insights on the use of CRENESSITY to target ACTH and potentially improve hormonal control in patients with 11β‑hydroxylase deficiency, highlighting our ongoing commitment to advancing care for the entire classic CAH community."

This retrospective case series outlines clinical responses to CRENESSITY treatment in 15 pediatric (n=11) and adult (n=4) patients with classic CAH due to 11β‑OHD. Across all patients with elevated A4 and/or adrenal steroid precursors at baseline, initiation of CRENESSITY was associated with reduction of levels.

• A4 and precursor normalization were observed as early as one month following treatment initiation, including in patients whose hormone excess had persisted despite prior supraphysiologic glucocorticoid (GC) therapy.
  • Among patients with elevated hormones at baseline, median decreases of -92% (DOC, n=5), -95% (11‑dF, n=7), and -65% (A4, n=3) were observed after CRENESSITY initiation.

• Among patients receiving antihypertensive treatment, two of five were able to reduce or discontinue these medications.
• Following the initiation of CRENESSITY, 14 of 15 patients were able to reduce their total GC dose.

"Patients with 11β-hydroxylase deficiency often experience both androgen excess and elevated adrenal steroid precursors that can contribute to hypertension and other complications, making disease management particularly challenging," said Kyriakie Sarafoglou, M.D., Professor, Department of Pediatrics and Department of Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics & Metabolism, University of Minnesota. "Given the rarity of this condition, data from 15 patients gives clinicians meaningful insight into the potential role of CRENESSITY in managing this challenging form of classic congenital adrenal hyperplasia."

This case series provides early, foundational evidence supporting the efficacy and safety of CRENESSITY treatment in patients with classic CAH due to 11β-OHD. Although CRENESSITY is approved as an adjunctive treatment to GCs for patients with classic CAH regardless of enzyme deficiency, there is limited evidence on its use in this rare subtype. These findings support further exploration of CRENESSITY in this classic CAH subtype and reinforce Neurocrine's commitment to supporting patients across the full spectrum of classic CAH and other rare endocrine diseases.

Presentations at the ENDO 2026 annual meeting included:

CAHtalyst^® Adult Study Two-Year Results

Title: Weight-Related Outcomes and Insulin Resistance in Adults with Classic Congenital Adrenal Hyperplasia: 2-Year Results from the CAHtalyst Adult Study (Oral Presentation #ORF32-07)
Authors: Oksana Hamidi, D.O., et al

Title: Adults with Classic Congenital Adrenal Hyperplasia Taking Crinecerfont Demonstrated Sustained Decreases in Glucocorticoid Doses: 2-Year Results from the CAHtalyst Adult Study (Poster Presentation #SUN-458)
Authors: Irina Bancos, M.D., et al

Title: A Cross-sectional Survey on Quality of Life of Adults with Classic Congenital Adrenal Hyperplasia in the United States Participating in CAHtalyst Adult Open-Label Extension Study (Poster Presentation #SUN-467)
Authors: Sonal Vaid, M.D., et al

Title: Bone Outcomes in Adults with Classic Congenital Adrenal Hyperplasia Treated with Crinecerfont for Up to 2 Years in CAHtalyst Adult Study (Poster Presentation #SUN-468)
Authors: Maria Vogiatzi, M.D., et al

CAHtalyst Pediatric Study Two-Year Results

Title: Characterization of Children and Adolescents with Classic Congenital Adrenal Hyperplasia Who Had Slowed Bone Age Progression and Improved Height Prediction with Crinecerfont (Oral Presentation #ORF32-05)
Authors: Maria Vogiatzi, M.D., et al

Title: Long-term Crinecerfont Treatment Reduced ACTH and 17-Hydroxyprogesterone — Clinical Outcomes in Children and Adolescents with Classic Congenital Adrenal Hyperplasia: 2-Year Results from CAHtalyst Pediatric (Poster Presentation #SAT-465)
Authors: Natalie Nokoff, M.D., et al

Title: Long-term Crinecerfont Enables Sustained Decreases in Glucocorticoid Doses — Clinical Outcomes in Children and Adolescents with Classic Congenital Adrenal Hyperplasia: 2-Year Results from CAHtalyst Pediatric (Poster Presentation #SUN-465)
Authors: Kyriakie Sarafoglou, M.D., et al

Additional Presentations

Title: Long-Term Risk of Cardiometabolic Comorbidities Associated with Glucocorticoid Exposure and Androgen Control in Classic Congenital Adrenal Hyperplasia: A Cox Proportional Hazards Analysis from the CAHtalog Registry ("New Therapies and Perspectives for Congenital Adrenal Hyperplasia and Adrenal Insufficiency" Rapid Fire Presentation #ORF32-02 and Poster Presentation #MON-495)
Authors: Oksana Lekarev, D.O., et al

Title: Crinecerfont Treatment of Classic Congenital Adrenal Hyperplasia Due to 11β-Hydroxylase Deficiency: A Case Series (Poster Presentation #SAT-466)
Authors: Kyriakie Sarafoglou, M.D., et al 

Title: A Modified Delphi Panel of U.S. Endocrinologists to Align on Minimum Clinically Important Difference in Glucocorticoid Dose and Other Key Considerations in Classic Congenital Adrenal Hyperplasia (Poster Presentation #SAT-459)
Authors: Ahmed Khattab, M.D., et al

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^® (crinecerfont) 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, developing and commercializing life-changing treatments for patients with under-addressed neurological, psychiatric, endocrine 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, hyperphagia in patients with Prader-Willi syndrome, 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 more than 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 target adrenocorticotropic hormone (ACTH) and improve hormonal control in patients with CAH due to 11β-hydroxylase deficiency; 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 quarterly report on Form 10-Q for the quarter ended March 31, 2026. 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-0062 06/2026

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SOURCE Neurocrine Biosciences, Inc.

FAQ

What did Neurocrine Biosciences (NBIX) announce about CRENESSITY at ENDO 2026?

Neurocrine Biosciences announced first retrospective case-series data on CRENESSITY (crinecerfont) in classic congenital adrenal hyperplasia due to 11β‑hydroxylase deficiency. According to Neurocrine, the 15-patient analysis showed marked reductions in adrenal hormones, lower glucocorticoid requirements, and improved blood pressure in adults.

How did CRENESSITY affect hormone levels in classic CAH patients with 11β-hydroxylase deficiency?

CRENESSITY was associated with substantial decreases in androstenedione and adrenal steroid precursors in classic CAH patients. According to Neurocrine, median reductions reached 92% for DOC, 95% for 11-deoxycortisol, and 65% for androstenedione among patients with elevated baseline hormone levels.

Did CRENESSITY (NBIX) reduce glucocorticoid doses in the ENDO 2026 case series?

Yes, most patients reduced glucocorticoid use after starting CRENESSITY in the case series. According to Neurocrine, 14 of 15 pediatric and adult patients were able to lower their total glucocorticoid dose while showing improved hormonal control and precursor normalization.

What blood pressure and antihypertensive outcomes were reported with CRENESSITY in NBIX’s ENDO 2026 data?

Adult patients showed improved blood pressure after CRENESSITY initiation in this analysis. According to Neurocrine, steroid precursors normalized, and 2 of 5 patients receiving antihypertensive treatment were able to reduce or discontinue these medications alongside better hormonal control.

Is CRENESSITY already approved for classic congenital adrenal hyperplasia beyond this 11β-hydroxylase study?

Yes, CRENESSITY is approved as an adjunctive treatment to glucocorticoids for patients with classic CAH regardless of enzyme deficiency. According to Neurocrine, this 11β-hydroxylase case series offers early evidence in a rare subtype not previously studied in trials.

How many patients were included in Neurocrine’s NBIX CRENESSITY 11β-hydroxylase deficiency case series?

The case series evaluated 15 pediatric and adult patients with classic CAH due to 11β‑hydroxylase deficiency. According to Neurocrine, the cohort included 11 children and adolescents and 4 adults, providing preliminary clinical insight for this uncommon CAH subtype.

What other CAH-related CRENESSITY studies did Neurocrine (NBIX) highlight at ENDO 2026?

Neurocrine highlighted multiple CAHtalyst adult and pediatric studies and registry analyses at ENDO 2026. According to Neurocrine, these covered weight and insulin resistance, bone outcomes, glucocorticoid dose reductions, quality of life, and long-term endocrine and cardiometabolic risks in classic CAH.