Neurocrine Biosciences Presents New Analysis of CAHtalog® Registry Showing Most Patients with Classic Congenital Adrenal Hyperplasia Experienced High Glucocorticoid Exposure, Suboptimal Disease Control or Both at ENDO 2025
Neurocrine Biosciences (NASDAQ: NBIX) presented new analysis from the CAHtalog® registry revealing significant challenges in managing classic congenital adrenal hyperplasia (CAH). The study found that approximately 95% of patients experienced either high glucocorticoid exposure or elevated androstenedione levels during treatment.
The analysis, presented at ENDO 2025, examined data from 98 patients (37 adults, 42 children/adolescents, 19 cross-age contributors) over a median period of 8.6 years. Among patients eligible for health state transition analysis, 88.9% experienced at least one health state change, with 58.7% undergoing three or more changes, highlighting the unpredictable nature of disease control.
The findings emphasize the limitations of current glucocorticoid therapy and demonstrate the need for alternative treatment approaches for managing CAH effectively.
Neurocrine Biosciences (NASDAQ: NBIX) ha presentato una nuova analisi dal registro CAHtalog® che evidenzia le significative difficoltà nella gestione dell'iperplasia surrenalica congenita classica (CAH). Lo studio ha rilevato che circa il 95% dei pazienti ha sperimentato un'esposizione elevata ai glucocorticoidi o livelli aumentati di androstenedione durante il trattamento.
L'analisi, presentata a ENDO 2025, ha esaminato i dati di 98 pazienti (37 adulti, 42 bambini/adolescenti, 19 contributori cross-age) in un periodo mediano di 8,6 anni. Tra i pazienti idonei all'analisi della transizione dello stato di salute, l'88,9% ha subito almeno un cambiamento di stato di salute, con il 58,7% che ha vissuto tre o più cambiamenti, mettendo in evidenza la natura imprevedibile del controllo della malattia.
I risultati sottolineano i limiti della terapia glucocorticoidea attuale e dimostrano la necessità di approcci terapeutici alternativi per una gestione efficace della CAH.
Neurocrine Biosciences (NASDAQ: NBIX) presentó un nuevo análisis del registro CAHtalog® que revela importantes desafíos en el manejo de la hiperplasia suprarrenal congénita clásica (CAH). El estudio encontró que aproximadamente el 95% de los pacientes experimentaron exposición elevada a glucocorticoides o niveles elevados de androstenediona durante el tratamiento.
El análisis, presentado en ENDO 2025, examinó datos de 98 pacientes (37 adultos, 42 niños/adolescentes, 19 contribuyentes de edades cruzadas) durante un período medio de 8,6 años. Entre los pacientes elegibles para el análisis de transición del estado de salud, el 88,9% experimentó al menos un cambio en el estado de salud, y el 58,7% tuvo tres o más cambios, lo que resalta la naturaleza impredecible del control de la enfermedad.
Los hallazgos enfatizan las limitaciones de la terapia actual con glucocorticoides y demuestran la necesidad de enfoques terapéuticos alternativos para manejar la CAH de manera efectiva.
Neurocrine Biosciences (NASDAQ: NBIX)는 CAHtalog® 등록부에서 새로운 분석 결과를 발표하며 고전적 선천성 부신과형성증(CAH) 관리에 상당한 어려움이 있음을 밝혔습니다. 연구에 따르면 약 95%의 환자가 치료 중에 고용량 글루코코르티코이드 노출 또는 안드로스텐디온 수치 상승을 경험했습니다.
ENDO 2025에서 발표된 이 분석은 98명의 환자(성인 37명, 아동/청소년 42명, 연령대별 기여자 19명)의 데이터를 중앙값 8.6년 동안 조사했습니다. 건강 상태 전환 분석 대상 환자 중 88.9%가 최소 한 번 이상의 건강 상태 변화를 경험했으며, 58.7%는 세 번 이상의 변화를 겪어 질병 조절의 예측 불가능한 특성을 강조했습니다.
이 결과는 현재 글루코코르티코이드 치료의 한계를 강조하며 CAH를 효과적으로 관리하기 위한 대체 치료법의 필요성을 보여줍니다.
Neurocrine Biosciences (NASDAQ : NBIX) a présenté une nouvelle analyse du registre CAHtalog® révélant des défis importants dans la prise en charge de l'hyperplasie congénitale des surrénales classique (CAH). L'étude a montré que près de 95 % des patients ont subi une exposition élevée aux glucocorticoïdes ou des niveaux élevés d'androstènedione pendant le traitement.
L'analyse, présentée lors de ENDO 2025, a examiné les données de 98 patients (37 adultes, 42 enfants/adolescents, 19 contributeurs de différentes tranches d'âge) sur une période médiane de 8,6 ans. Parmi les patients éligibles à l'analyse des transitions d'état de santé, 88,9 % ont connu au moins un changement d'état de santé, avec 58,7 % ayant subi trois changements ou plus, soulignant la nature imprévisible du contrôle de la maladie.
Ces résultats mettent en évidence les limites de la thérapie actuelle par glucocorticoïdes et démontrent la nécessité d'approches thérapeutiques alternatives pour une gestion efficace de la CAH.
Neurocrine Biosciences (NASDAQ: NBIX) präsentierte eine neue Analyse aus dem CAHtalog®-Register, die erhebliche Herausforderungen bei der Behandlung der klassischen kongenitalen adrenalen Hyperplasie (CAH) aufzeigt. Die Studie ergab, dass etwa 95 % der Patienten während der Behandlung entweder einer hohen Glukokortikoidexposition oder erhöhten Androstendionspiegeln ausgesetzt waren.
Die auf der ENDO 2025 vorgestellte Analyse untersuchte Daten von 98 Patienten (37 Erwachsene, 42 Kinder/Jugendliche, 19 altersübergreifende Teilnehmer) über einen medianen Zeitraum von 8,6 Jahren. Unter den für die Analyse des Gesundheitszustandsübergangs geeigneten Patienten erlebten 88,9 % mindestens eine Änderung des Gesundheitszustands, wobei 58,7 % drei oder mehr Veränderungen durchmachten, was die unvorhersehbare Natur der Krankheitskontrolle verdeutlicht.
Die Ergebnisse unterstreichen die Grenzen der aktuellen Glukokortikoidtherapie und zeigen die Notwendigkeit alternativer Behandlungsansätze für eine effektive CAH-Management auf.
- Comprehensive real-world data from CAHtalog registry provides valuable insights into CAH treatment patterns
- Large patient sample size of 98 participants with long-term follow-up (median 8.6 years)
- Study identifies clear unmet medical needs in CAH treatment, supporting potential market opportunity for new therapies
- 95.2% of patients experienced suboptimal health states with high glucocorticoid doses or elevated androstenedione
- Current glucocorticoid therapy shows significant limitations in maintaining long-term disease control
- High variability in treatment outcomes and frequent health state changes indicate challenges in disease management
Insights
Neurocrine's registry data shows current CAH treatments inadequate; supports clinical need for their CRENESSITY therapy.
Neurocrine Biosciences has presented compelling real-world evidence from their CAHtalog registry that highlights significant treatment challenges for patients with classic congenital adrenal hyperplasia (CAH). The data revealed that approximately
The analysis examined 98 patients (37 adults, 42 children/adolescents, and 19 who contributed data in both categories) over a median observation period of 8.6 years. What's particularly noteworthy is that
These findings address a critical clinical dilemma in CAH management: glucocorticoids are essential to replace cortisol deficiency, but supraphysiologic doses required to suppress androgens expose patients to significant side effects. The data demonstrates that current treatment paradigms force clinicians to choose between androgen excess (with its associated complications) and glucocorticoid overexposure (with its own substantial risks including growth suppression, metabolic disorders, and bone density loss).
The company also presented multiple studies on their CRENESSITY (crinecerfont) therapy at ENDO 2025, suggesting their drug may address this precise treatment gap by enabling more physiologic glucocorticoid dosing while maintaining androgen control. This registry data effectively establishes the medical need their product aims to address in the marketplace.
- Approximately
95% of adult and pediatric patients were exposed to supraphysiologic doses of glucocorticoids and/or experienced elevated androstenedione levels at least once during their treatment journey - Real-world findings underscore long-term challenges of managing classic congenital adrenal hyperplasia and highlight clinical need for therapies to reduce androgen excess without high glucocorticoid doses
"The CAHtalog data revealed just how dynamic and unpredictable disease control can be for people with classic congenital adrenal hyperplasia, with most experiencing elevated glucocorticoid exposure and/or loss of androgen control at multiple points throughout their lives," said Sanjay Keswani, M.D., Chief Medical Officer, Neurocrine Biosciences. "This analysis emphasizes that achieving disease control today, often with the use of high-dose glucocorticoids, offers no guarantee of maintaining disease control long term and underscores the ongoing complexity of managing classic congenital adrenal hyperplasia over a lifetime."
While glucocorticoid (GC) therapy is essential to replace cortisol deficiency in patients with classic congenital adrenal hyperplasia (CAH), achieving hormonal control using GC therapy alone requires supraphysiologic doses to reduce elevated adrenocorticotropic hormone (ACTH) and androgen levels. Developed in partnership with the CARES Foundation, the CAHtalog (Congenital Adrenal Hyperplasia: Patient and Clinical Outcomes in Real-World Practice Settings) registry was established to characterize longitudinal treatment patterns, natural history and impact of CAH. This latest analysis specifically examined patterns of GC use and androstenedione (A4) levels over time in pediatric and adult participants.
In this most recent analysis, data were examined from 98 patients, including 37 adults, 42 children and adolescents and 19 individuals who contributed data as both children and adults. Importantly, this analysis was conducted using data collected prior to the commercial availability of CRENESSITY® (crinecerfont) and does not include any CRENESSITY treatment data. GC treatment records were paired with the nearest available A4 laboratory values. GC doses were classified as "higher" (pediatric: >11 mg/m2/d hydrocortisone equivalents [HCe]; adult: >20 mg/d HCe) or "lower" (pediatric: ≤11 mg/m2/d HCe; adult ≤20 mg/d HCe). A4 levels were categorized as "higher" (≥upper limit of normal [ULN]) or "lower" (<ULN). Four health states were defined by combinations of GC dose and A4 level: (1) lower GC/lower A4, (2) lower GC/higher A4, (3) higher GC/lower A4 and (4) higher GC/higher A4. These states were analyzed in patients with at least three matched GC-A4 records to evaluate patterns of treatment intensity and hormonal control over time.
Key findings from the analysis showed considerable variability in GC regimens among patients and frequent transitions across health states, most of which were characterized by higher GC doses and/or elevated A4 levels. Even when patients achieved a health state with low GC doses and A4 levels within the normal range, this state was often short-lived.
Among patients eligible for the health state transition analysis (n=63),
"These real-world findings reinforce what we know about the dynamic nature of CAH management, where patients' treatment needs are constantly evolving," said Dina Matos, Executive Director, CARES Foundation. "The data clearly show that even when individuals achieve disease control with glucocorticoid treatment alone, maintaining that control over time remains a significant challenge. This highlights the importance of continued monitoring and the need for advanced therapeutic options for patients living with CAH."
Additional presentations at ENDO 2025 included:
Title | Oral Presentation |
[NEW] Crinecerfont Allows for More Physiologic Glucocorticoid Treatment with Reduction of Androstenedione to a Normal Range in Adults with Classic Congenital Adrenal Hyperplasia: Post Hoc Analyses of the CAHtalyst Adult Study | OR07-04 |
Crinecerfont Maintains Reductions in Serum Androstenedione Levels and Glucocorticoid Doses in Children and Adolescents with Classic Congenital Adrenal Hyperplasia: 1-Year Results from the CAHtalyst Pediatric Study | OR07-05 |
Title | Poster Presentation |
[NEW] Crinecerfont Maintains Serum Androstenedione Levels with Reduced Glucocorticoid Doses in Adults with Classic Congenital Adrenal Hyperplasia: 1-Year Results from the CAHtalyst Adult Study | SUN-415 |
[NEW] Crinecerfont Maintains Adrenocorticotropic Hormone and 17-Hydroxyprogesterone Levels with Reduced Glucocorticoid Doses in Adults with Classic Congenital Adrenal Hyperplasia: 1-Year Results from the CAHtalyst Adult Study | SUN-417 |
[NEW] Crinecerfont Improves Clinical Outcomes in Adults with Classic Congenital Adrenal Hyperplasia: 1-Year Results from the CAHtalyst Adult Study | SUN-416 |
[NEW] Crinecerfont Shows Favorable Trends in Improving Weight-Related Outcomes in Adults with Classic Congenital Adrenal Hyperplasia: 1-Year Results from the CAHtalyst Adult Study | SUN-405 |
[NEW] Evaluation of Potential Drug-Drug Interactions with Crinecerfont | SUN-440 |
[NEW] Crinecerfont Enables Reduction of Glucocorticoid Doses While Maintaining or Improving Androstenedione in Adults with Classic Congenital Adrenal Hyperplasia: Subgroup Analyses from the Phase 3 CAHtalyst Adult Study | MON-466 |
[NEW] Crinecerfont Shows Favorable Trends in Improving Weight-Related Outcomes in Pediatric Patients with Classic Congenital Adrenal Hyperplasia: 1-Year Results from the CAHtalyst Pediatric Study | MON-459 |
Crinecerfont Allows for More Physiologic Glucocorticoid Treatment with Reduction of Androstenedione to a Normal Range in Pediatric Patients with Classic Congenital Adrenal Hyperplasia: Post Hoc Analysis of the CAHtalyst Pediatric Study | SAT-418 |
Crinecerfont Enables Reduction of Glucocorticoid Doses While Maintaining or Improving Androstenedione in Pediatric Patients with Classic Congenital Adrenal Hyperplasia: Subgroup Analyses from the CAHtalyst Pediatric Phase 3 Study | SAT-442 |
Crinecerfont Improves Reproductive Hormones in Classic Congenital Adrenal Hyperplasia: 1-Year Results from the Phase 3 CAHtalyst Adult Study | SUN-414 |
Crinecerfont Reduces Plasma Adrenocorticotropic Hormone and Serum 17-Hydroxyprogesterone Levels in Children and Adolescents with Classic Congenital Adrenal Hyperplasia: 1-Year Results from the CAHtalyst Pediatric Study | SUN-434 |
Crinecerfont Shows Favorable Trends in Improving Clinical Outcomes in Children and Adolescents with Classic Congenital Adrenal Hyperplasia: 1-Year Results from the CAHtalyst Pediatric Study | SUN-441 |
Crinecerfont Allows for More Physiologic Glucocorticoid Dosing Regimens in Patients with Classic Congenital Adrenal Hyperplasia: Results from the Phase 3 CAHtalyst Adult and CAHtalyst Pediatric Studies | MON-458 |
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. Approximately
Exogeneous 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 high 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 high-dose 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 fertility issues in both sexes. The symptoms of high ACTH may include testicular adrenal rest tumors (TARTs) or ovarian adrenal rest tumors (OARTs).
About The CAHtalog® Registry
In 2021, the CARES Foundation, Neurocrine Biosciences and PicnicHealth partnered to establish the CAHtalog (Congenital Adrenal Hyperplasia: Patient and Clinical Outcomes in Real-World Practice Settings) Registry. The CAHtalog Registry is a
About CRENESSITY® (crinecerfont)
CRENESSITY is a potent and selective oral corticotropin-releasing factor type 1 receptor (CRF1) antagonist developed to reduce and control 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.
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 neuroscience-focused, 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, neuroendocrine and neuropsychiatric 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 and Facebook. (*in collaboration with AbbVie)
The NEUROCRINE BIOSCIENCES Logo, NEUROCRINE, YOU DESERVE BRAVE SCIENCE, CRENESSITY and CAHtalog are registered trademarks of Neurocrine Biosciences, Inc. CAHtalyst is a trademark 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; 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 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, 2025. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof other than required by law.
© 2025 Neurocrine Biosciences, Inc. All Rights Reserved. CAP-CFT-US-0033 07/2025
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FAQ
What are the key findings of Neurocrine's (NBIX) CAHtalog registry analysis for CAH patients?
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