Neurocrine Biosciences Presents Findings Contributing to the Growing Body of Evidence on the Impact of High-Dose Glucocorticoids on Clinical Outcomes in Congenital Adrenal Hyperplasia
Neurocrine Biosciences (NBIX) presented a systematic literature review at the 2025 ISPOR Annual Meeting, examining the relationship between glucocorticoid (GC) dosage and adverse clinical outcomes in congenital adrenal hyperplasia (CAH) patients. The study analyzed 105 publications, with 65% focusing on CAH patients.
Key findings revealed that 62% of publications found statistically significant associations between GC dose and clinical outcomes, with 98% of these indicating higher GC doses led to adverse effects, including decreased bone mineral density, increased insulin resistance, and higher body mass index. The most commonly reported outcomes were bone health (43%), cardiometabolic (41%), and height and growth (24%).
Neurocrine Biosciences (NBIX) ha presentato una revisione sistematica della letteratura al Congresso Annuale ISPOR 2025, analizzando la relazione tra la dose di glucocorticoidi (GC) e gli esiti clinici avversi nei pazienti con iperplasia surrenalica congenita (CAH). Lo studio ha esaminato 105 pubblicazioni, di cui il 65% focalizzate su pazienti con CAH.
I risultati principali hanno evidenziato che il 62% delle pubblicazioni ha riscontrato associazioni statisticamente significative tra la dose di GC e gli esiti clinici, con il 98% di queste che indicano come dosi più elevate di GC provochino effetti avversi, tra cui riduzione della densità minerale ossea, aumento della resistenza all’insulina e incremento dell’indice di massa corporea. Gli esiti più frequentemente riportati sono stati la salute delle ossa (43%), gli aspetti cardiometabolici (41%) e l’altezza e la crescita (24%).
Neurocrine Biosciences (NBIX) presentó una revisión sistemática de la literatura en la Reunión Anual ISPOR 2025, examinando la relación entre la dosis de glucocorticoides (GC) y los resultados clínicos adversos en pacientes con hiperplasia suprarrenal congénita (CAH). El estudio analizó 105 publicaciones, de las cuales el 65% se centraron en pacientes con CAH.
Los hallazgos clave revelaron que el 62% de las publicaciones encontraron asociaciones estadísticamente significativas entre la dosis de GC y los resultados clínicos, y el 98% de estas indicaron que dosis más altas de GC conducen a efectos adversos, incluyendo disminución de la densidad mineral ósea, aumento de la resistencia a la insulina y mayor índice de masa corporal. Los resultados más reportados fueron la salud ósea (43%), cardiometabólicos (41%) y altura y crecimiento (24%).
Neurocrine Biosciences (NBIX)는 2025년 ISPOR 연례 회의에서 문헌 체계적 검토를 발표하며, 선천성 부신과형성증(CAH) 환자에서 글루코코르티코이드(GC) 용량과 임상 부작용 간의 관계를 조사했습니다. 이 연구는 105건의 출판물을 분석했으며, 그중 65%가 CAH 환자에 초점을 맞추었습니다.
주요 결과는 62%의 출판물이 GC 용량과 임상 결과 간에 통계적으로 유의한 연관성을 발견했다는 점이며, 이 중 98%는 높은 GC 용량이 골밀도 감소, 인슐린 저항성 증가, 체질량 지수 상승 등 부작용을 초래한다고 보고했습니다. 가장 흔히 보고된 결과는 골 건강(43%), 심장대사(41%), 키 및 성장(24%)이었습니다.
Neurocrine Biosciences (NBIX) a présenté une revue systématique de la littérature lors de la réunion annuelle ISPOR 2025, examinant la relation entre la dose de glucocorticoïdes (GC) et les effets cliniques indésirables chez les patients atteints d'hyperplasie congénitale des surrénales (CAH). L’étude a analysé 105 publications, dont 65 % portaient sur des patients atteints de CAH.
Les résultats clés ont révélé que 62 % des publications ont trouvé des associations statistiquement significatives entre la dose de GC et les résultats cliniques, 98 % d’entre elles indiquant que des doses plus élevées de GC entraînaient des effets indésirables, notamment une diminution de la densité minérale osseuse, une augmentation de la résistance à l’insuline et un indice de masse corporelle plus élevé. Les résultats les plus fréquemment rapportés concernaient la santé osseuse (43 %), les aspects cardiométaboliques (41 %) et la taille et la croissance (24 %).
Neurocrine Biosciences (NBIX) präsentierte auf dem ISPOR-Jahrestreffen 2025 eine systematische Literaturübersicht, die den Zusammenhang zwischen Glukokortikoid(GC)-Dosierung und unerwünschten klinischen Ergebnissen bei Patienten mit kongenitaler adrenaler Hyperplasie (CAH) untersuchte. Die Studie analysierte 105 Publikationen, von denen 65 % sich auf CAH-Patienten konzentrierten.
Wesentliche Erkenntnisse zeigten, dass 62 % der Publikationen statistisch signifikante Zusammenhänge zwischen GC-Dosis und klinischen Ergebnissen fanden, wobei 98 % davon angaben, dass höhere GC-Dosen zu unerwünschten Effekten führten, darunter verringerte Knochendichte, erhöhte Insulinresistenz und höhere Körpermasseindizes. Die am häufigsten berichteten Ergebnisse betrafen die Knochengesundheit (43 %), kardiometabolische Aspekte (41 %) sowie Größe und Wachstum (24 %).
- First comprehensive systematic review analyzing the relationship between GC dose and all relevant adverse clinical outcomes in CAH patients
- Research supports the company's efforts in developing alternative therapeutic options for CAH treatment
- Study findings could help drive adoption of new treatment options that reduce reliance on high-dose glucocorticoids
- Study confirms significant clinical burden and complications from current standard CAH treatment
- Research highlights limitations and risks of existing glucocorticoid therapy
- 98% of significant findings showed adverse effects from higher GC doses, indicating serious treatment challenges
Insights
Neurocrine's research demonstrates high-dose glucocorticoids used to treat CAH significantly worsen patient outcomes, supporting development of alternative therapies.
The systematic literature review presented by Neurocrine Biosciences provides compelling evidence that higher glucocorticoid (GC) doses used to treat congenital adrenal hyperplasia (CAH) are associated with significant adverse clinical outcomes. This is a pivotal finding for the 25,000 CAH patients in the U.S. who currently rely on lifelong GC therapy to manage their condition.
The data is particularly striking: 98% of publications finding statistical significance concluded that higher GC doses negatively impact health outcomes. Specifically, these higher doses led to decreased bone mineral density, increased insulin resistance, and higher BMI - representing a classic treatment dilemma where the standard therapy causes substantial iatrogenic complications.
This research strategically positions Neurocrine in the CAH treatment landscape. The company is developing crinecerfont, a corticotropin-releasing factor type 1 (CRF₁) receptor antagonist designed to reduce adrenocorticotropic hormone (ACTH) levels in CAH patients. By potentially reducing ACTH-driven androgen production without glucocorticoids, crinecerfont could address a significant unmet medical need highlighted by this research.
The timing of this presentation suggests Neurocrine is building a comprehensive case for its alternative treatment approach. By establishing the serious drawbacks of current standard therapy through rigorous literature review, they're creating context for clinicians to understand the potential value of CRF₁ antagonism as a GC-sparing strategy in CAH management.
- Most Studies Meeting the Literature Review Criteria Found That a Higher Glucocorticoid Dose was Statistically Significantly Associated with Adverse Clinical Outcomes
- Findings Presented at the 2025 International Society for Pharmacoeconomics and Outcomes Research Annual Meeting
Congenital adrenal hyperplasia (CAH) is a rare, lifelong genetic condition linked to overproduction of adrenal androgens and cortisol deficiency. CAH is traditionally treated with lifelong high-dose glucocorticoids (GCs), which increase the risk of complications across multiple aspects of health. However, the relationship between GC dose and adverse clinical outcomes in CAH has not been fully explored. This systematic literature review was conducted to analyze the relationship between GC dose and clinical outcomes, and is the first to capture the impact of higher GC dose on the incidence and severity of all relevant adverse clinical outcomes in patients with CAH.
"CAH requires lifelong glucocorticoid therapy to manage adrenal androgen excess and cortisol deficiency, but high doses often lead to significant complications, including cardiometabolic, bone and growth issues," said Eiry W. Roberts, M.D., Chief Medical Officer, Neurocrine Biosciences. "This review highlights the significant clinical burden of long-term, high-dose glucocorticoid use in CAH patients and the challenges clinicians face in optimizing treatment. The emergence of new therapeutic options offers hope for potentially reducing reliance on high-dose glucocorticoids, representing an important advancement in the management of CAH."
The systematic literature review, conducted using PubMed and Embase, identified 105 publications, of which
More than half (
- Of these,
98% (n=64/65) concluded that higher GC doses were significantly linked to adverse clinical outcomes, including decreased bone mineral density, increased insulin resistance and higher body mass index. - The remaining
38% (n=40/105) of the publications did not reach statistically significant relationships between GC dose and clinical outcomes: nine (9% ) reported trends in adverse clinical outcomes, 29 (28% ) reported no trend and two (2% ) reported trends in improved clinical outcomes.
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, which are essential for life. Approximately
Historically, exogenous glucocorticoids (GCs) have been used to correct the endogenous cortisol deficiency, but doses higher than those for cortisol replacement (supraphysiologic) are needed 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 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 and YOU DESERVE BRAVE SCIENCE 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 new therapeutic options which may reduce reliance on high-dose glucocorticoids for people affected by CAH, and the benefit that such treatments may bring to these individuals. 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: data that we report may change following a more comprehensive review of the data related to the study; risks and uncertainties associated with Neurocrine Biosciences' business and finances in general, as well as risks and uncertainties associated with the commercialization of the Company's products; risks that the Company's products may be precluded from development by the proprietary rights of third parties, or have unintended side effects or adverse reactions; risks and uncertainties relating to competitive products and technological changes that may limit demand for the Company's products; risks associated with our dependence on third parties for development and manufacturing activities related to the Company's products, and our ability to manage these third parties; risks that the FDA or other regulatory authorities may make adverse decisions regarding our products; risks associated with potential generic entrants for our products; 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-0023 05/2025
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