Neurocrine Biosciences Presents One-Year Data Showing Sustained Efficacy of CRENESSITY® (crinecerfont) in Adult Patients, at ENDO 2025
Neurocrine Biosciences (NASDAQ: NBIX) presented one-year data from the CAHtalyst™ Adult study of CRENESSITY® (crinecerfont) at ENDO 2025. The Phase 3 trial, involving 182 adult patients aged 18-58, demonstrated sustained efficacy in treating classic congenital adrenal hyperplasia (CAH).
Key findings include 25-30% reduction in glucocorticoid doses while maintaining or improving androstenedione levels. The study showed improvements in insulin resistance and hirsutism in female participants. CRENESSITY demonstrated a favorable safety profile, with headache and fatigue as the most common side effects, mostly mild to moderate and temporary.
The data complements previously reported pediatric results and reinforces CRENESSITY's role in CAH management by effectively controlling ACTH and adrenal steroid imbalances while enabling lower, more physiologic steroid dosing.
Neurocrine Biosciences (NASDAQ: NBIX) ha presentato i dati a un anno dello studio CAHtalyst™ Adult su CRENESSITY® (crinecerfont) durante ENDO 2025. Lo studio di Fase 3, che ha coinvolto 182 pazienti adulti di età compresa tra 18 e 58 anni, ha dimostrato un'efficacia duratura nel trattamento dell'iperplasia surrenalica congenita (CAH) classica.
I risultati principali evidenziano una riduzione del 25-30% delle dosi di glucocorticoidi mantenendo o migliorando i livelli di androstenedione. Lo studio ha mostrato miglioramenti nella resistenza all'insulina e nell'irsutismo nelle partecipanti di sesso femminile. CRENESSITY ha mostrato un profilo di sicurezza favorevole, con mal di testa e affaticamento come effetti collaterali più comuni, per lo più lievi o moderati e temporanei.
I dati integrano i risultati pediatrici precedentemente riportati e rafforzano il ruolo di CRENESSITY nella gestione della CAH, controllando efficacemente ACTH e gli squilibri degli steroidi surrenalici, consentendo al contempo dosaggi steroidei più bassi e fisiologici.
Neurocrine Biosciences (NASDAQ: NBIX) presentó datos a un año del estudio CAHtalyst™ Adult de CRENESSITY® (crinecerfont) en ENDO 2025. El ensayo de Fase 3, que involucró a 182 pacientes adultos de entre 18 y 58 años, demostró una eficacia sostenida en el tratamiento de la hiperplasia suprarrenal congénita (CAH) clásica.
Los hallazgos clave incluyen una reducción del 25-30% en las dosis de glucocorticoides mientras se mantienen o mejoran los niveles de androstenediona. El estudio mostró mejoras en la resistencia a la insulina y el hirsutismo en las participantes femeninas. CRENESSITY demostró un perfil de seguridad favorable, con cefalea y fatiga como los efectos secundarios más comunes, en su mayoría leves a moderados y temporales.
Los datos complementan los resultados pediátricos previamente reportados y refuerzan el papel de CRENESSITY en el manejo de la CAH al controlar eficazmente el ACTH y los desequilibrios de esteroides suprarrenales, permitiendo al mismo tiempo dosis más bajas y fisiológicas de esteroides.
Neurocrine Biosciences (NASDAQ: NBIX)는 ENDO 2025에서 CRENESSITY®(크리네서폰트)의 CAHtalyst™ 성인 연구 1년 데이터를 발표했습니다. 18세에서 58세 사이의 182명의 성인 환자를 대상으로 한 3상 임상시험에서 고전적 선천성 부신과형성증(CAH) 치료에 지속적인 효능을 입증했습니다.
주요 결과로는 안드로스텐디온 수치를 유지하거나 개선하면서 글루코코르티코이드 용량을 25-30% 감소시킨 점이 포함됩니다. 연구에서는 여성 참가자들의 인슐린 저항성과 다모증이 개선되었습니다. CRENESSITY는 두통과 피로가 가장 흔한 부작용으로 보고되었으며, 대부분 경증에서 중등도의 일시적인 증상이었습니다.
이 데이터는 이전에 보고된 소아 결과를 보완하며, ACTH 및 부신 스테로이드 불균형을 효과적으로 조절하면서 더 낮고 생리학적인 스테로이드 용량을 가능하게 하는 CRENESSITY의 CAH 관리 역할을 강화합니다.
Neurocrine Biosciences (NASDAQ : NBIX) a présenté lors de l'ENDO 2025 les données à un an de l'étude CAHtalyst™ Adult sur CRENESSITY® (crinecerfont). L'essai de phase 3, impliquant 182 patients adultes âgés de 18 à 58 ans, a démontré une efficacité soutenue dans le traitement de l'hyperplasie congénitale des surrénales (CAH) classique.
Les résultats clés incluent une réduction de 25 à 30 % des doses de glucocorticoïdes tout en maintenant ou en améliorant les niveaux d'androstènedione. L'étude a montré des améliorations de la résistance à l'insuline et de l'hirsutisme chez les participantes féminines. CRENESSITY a présenté un profil de sécurité favorable, les maux de tête et la fatigue étant les effets secondaires les plus courants, principalement légers à modérés et temporaires.
Ces données complètent les résultats pédiatriques précédemment rapportés et renforcent le rôle de CRENESSITY dans la prise en charge de la CAH en contrôlant efficacement l'ACTH et les déséquilibres des stéroïdes surrénaliens tout en permettant des doses stéroïdiennes plus faibles et plus physiologiques.
Neurocrine Biosciences (NASDAQ: NBIX) präsentierte auf der ENDO 2025 einjährige Daten der CAHtalyst™-Erwachsenenstudie zu CRENESSITY® (Crinecerfont). Die Phase-3-Studie mit 182 erwachsenen Patienten im Alter von 18 bis 58 Jahren zeigte eine anhaltende Wirksamkeit bei der Behandlung der klassischen kongenitalen adrenalen Hyperplasie (CAH).
Wesentliche Ergebnisse umfassen eine 25–30%ige Reduktion der Glukokortikoiddosen, während die Androstendionwerte erhalten blieben oder sich verbesserten. Die Studie zeigte Verbesserungen bei Insulinresistenz und Hirsutismus bei weiblichen Teilnehmern. CRENESSITY zeigte ein günstiges Sicherheitsprofil, wobei Kopfschmerzen und Müdigkeit die häufigsten Nebenwirkungen waren, meist mild bis moderat und vorübergehend.
Die Daten ergänzen zuvor berichtete pädiatrische Ergebnisse und stärken die Rolle von CRENESSITY im Management der CAH, indem ACTH und adrenale Steroidungleichgewichte effektiv kontrolliert werden und gleichzeitig niedrigere, physiologischere Steroiddosierungen ermöglicht werden.
- Sustained 25-30% reduction in glucocorticoid doses while maintaining effectiveness
- Improvements observed in insulin resistance and hirsutism in female patients
- Favorable safety profile with mostly mild to moderate temporary side effects
- Successful maintenance or reduction of ACTH and 17-OHP levels below baseline
- Headache and fatigue reported as common side effects during treatment
- More frequent side effects during the double-blind period with bi-weekly glucocorticoid reductions
Insights
CRENESSITY shows sustained efficacy at one year, allowing substantial glucocorticoid dose reductions while maintaining hormonal control in CAH patients.
Neurocrine's Phase 3 CAHtalyst Adult study delivers compelling one-year data for CRENESSITY® (crinecerfont) in classic congenital adrenal hyperplasia (CAH) management. The results demonstrate that patients achieved 25-30% reductions in glucocorticoid doses while maintaining or improving control of key hormonal markers (ACTH, 17-hydroxyprogesterone, and androstenedione).
This data addresses a critical clinical challenge in CAH treatment: the need for supraphysiologic glucocorticoid doses that cause significant side effects. CRENESSITY appears to enable more physiologic steroid dosing – a substantial benefit for patients who typically require lifelong therapy.
The study's robust design included 182 adult patients in a 24-week placebo-controlled phase followed by 24 weeks of open-label treatment. Measurable clinical improvements were observed in insulin resistance (-0.5 to -0.9 from baseline) and hirsutism in female participants (approximately 12-point reduction on a 100-point visual analog scale).
The safety profile appears favorable, with headache and fatigue reported as the most common side effects. These were predominantly mild to moderate and temporary, with limited discontinuations – particularly important for a chronic medication.
These adult results complement previously announced positive one-year pediatric data, suggesting CRENESSITY may provide consistent benefits across age groups. The presentation of additional analyses at ENDO 2025, including weight-related outcomes, further expands understanding of this medication's clinical profile in this rare genetic disorder.
- One-year data from CAHtalyst™ Adult study demonstrated lasting reductions in glucocorticoid dose and improvement in clinical outcomes in adults with classic congenital adrenal hyperplasia
- Results build upon previously reported one-year data from CAHtalyst™ Pediatric study
"Results from the pivotal CAHtalyst clinical trial program continue to reinforce the critical role of CRENESSITY in the management of classic congenital adrenal hyperplasia," said Sanjay Keswani, M.D., Chief Medical Officer, Neurocrine Biosciences. "These one-year data show the lasting ability of CRENESSITY to effectively manage the ACTH and adrenal steroid imbalances in adults while allowing for lower, more physiologic steroid dosing and improved clinical outcomes."
The Phase 3 CAHtalyst Adult study was part of the largest-ever interventional clinical trial program in classic congenital adrenal hyperplasia (CAH) and included 182 adult patients, 18 to 58 years of age. The study consisted of a 24-week, double-blind, placebo-controlled (DBPC) period, during which patients with classic CAH on supraphysiologic glucocorticoid (GC) doses were randomized 2:1 to receive CRENESSITY or placebo, and a 24-week, open-label (OL) period, during which all patients received CRENESSITY. During both the DBPC and OL periods, GC doses were kept stable for the first four weeks and then decreased as tolerated toward more physiologic levels while maintaining or improving androstenedione (A4) relative to Day 1 baseline.
These analyses examined the effect of up to one year of CRENESSITY in adults on changes in serum A4 levels and GC doses, changes in adrenocorticotropic hormone (ACTH) and 17-hydroxyprogesterone (17-OHP) levels and clinical outcomes.
- Lasting and substantial reductions in high GC doses were observed in adult patients with up to one year of treatment with CRENESSITY, while A4 was maintained or improved even in the context of lower, more physiologic GC dosing.
- The proportion of participants who received continuous CRENESSITY and achieved a GC dose within the physiologic range (defined in the study as ≤11 mg/m2/d hydrocortisone equivalents) while maintaining or improving pre-GC A4 levels remained generally stable from six to 12 months.
- ACTH and 17-OHP levels were maintained or reduced to below Day 1 baseline levels in adults taking CRENESSITY for up to one year, despite substantial reductions in GC doses.
- Improvements were observed in insulin resistance and in hirsutism (females) in adults taking CRENESSITY for up to one year.
- New data on weight-related outcomes will also be shared at ENDO 2025 and in a subsequent press release.
Measure | Baseline | Month 12 change from baseline (continuous CRENESSITY group) | Month 12 change from baseline (placebo to CRENESSITY group) |
Mean GC dose* | 32.4 mg/day CRENESSITY; 32.1 mg/day placebo | -25 % | -30 % |
Mean homeostatic model assessment of insulin resistance | 3.2 CRENESSITY; 3.1 placebo | -0.5 | -0.9 |
Mean hirsutism VAS scores (out of 100) in female participants | 40.6 mm CRENESSITY; 37.4 mm placebo | -11.5 | -12.9 |
VAS: visual analog scale. *In hydrocortisone equivalents, with A4 maintained or reduced below Day 1 baseline; conversion factors: prednisone/prednisolone/methylprednisolone, 4; dexamethasone, 60. | |||
CRENESSITY has a demonstrated safety profile. Headache and fatigue were the most common side effects in both study periods in adults taking CRENESSITY. Both occurred more frequently during the DBPC period (when GCs were reduced every two weeks) than during the open-label period (when GCs were reduced once a month). Most side effects were temporary and mild to moderate in severity and generally did not lead to discontinuation of the study drug.
Additional presentations, highlighting both new and encore data, at ENDO 2025 include:
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 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 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 |
[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] Treatment Patterns and Changes in Health States in Patients with Classic Congenital Adrenal Hyperplasia: An Analysis of Data from the CAHtalog Registry | MON-467 |
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 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 Improves Reproductive Hormones in Classic Congenital Adrenal Hyperplasia: 1-Year Results from the Phase 3 CAHtalyst Adult Study | SUN-414 |
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 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 interventional 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 could enable 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 could enable 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.
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-0031 07/2025
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FAQ
What are the key findings from Neurocrine's (NBIX) one-year CRENESSITY trial in adult CAH patients?
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How does CRENESSITY affect glucocorticoid dosing in adult CAH patients?
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