Neurocrine reports two-year Phase 3 data showing crinecerfont reduces glucocorticoid doses in adults with congenital adrenal hyperplasia while maintaining disease control.
Written By: Dr. Anuja Badgujar, BDS
Reviewed By: Pharmacally Editorial Team
Neurocrine has reported new two-year Phase 3 data from the CAHtalyst Adult study (NCT04490915) showing that treatment with CRENESSITY (crinecerfont) enables sustained reductions in glucocorticoid (GC) dosing in adults with congenital adrenal hyperplasia (CAH) while maintaining disease control.
Presented at the American Association of Clinical Endocrinology 2026 Annual Meeting, the data show that 69% of patients achieved physiologic glucocorticoid dosing levels (≤11 mg/m²/day hydrocortisone equivalents) at 24 months, compared to none at baseline.
The mean daily glucocorticoid dose was reduced by 38%, declining from 17.6 to 10.6 mg/m²/day, with the reduction sustained over two years.
These reductions were achieved without compromising androgen control, as indicated by stable androstenedione levels, while treatment optimization included 75% of patients transitioning off dexamethasone and 62% of those on multiple daily hydrocortisone doses eliminating at least one dose.
Long-term treatment with CRENESSITY (crinecerfont) was generally well tolerated, with more than 80% study retention and no new safety signals observed.
Chronic exposure to supraphysiologic glucocorticoid doses is associated with significant long-term risks, including cardiometabolic complications, bone density loss, and psychological effects, underscoring the importance of reducing glucocorticoid burden in the management of congenital adrenal hyperplasia.
Congenital adrenal hyperplasia is a rare inherited endocrine disorder characterized by enzyme deficiencies that impair cortisol production in the adrenal glands. This leads to elevated adrenocorticotropic hormone levels and excess androgen production, which can result in hormonal imbalance, metabolic complications, and, if untreated, life-threatening adrenal crises.
Sanjay Keswani M.D., Chief Medical Officer, Neurocrine Biosciences said the findings demonstrate durable androgen control alongside sustained GC reduction without new safety concerns, supporting the therapy’s potential as a long-term treatment option.
Richard J. Auchus, M.D., Ph.D, Principal Investigator for the CAHtalyst Adult study noted that long-term high-dose glucocorticoid exposure contributes to significant health complications and that the results provide clinically relevant evidence to inform future management of adult CAH.
Crinecerfont, an oral corticotropin-releasing factor type 1 receptor antagonist, reduces excess adrenocorticotropic hormone and adrenal androgen production through a non-glucocorticoid mechanism, enabling lower, more physiologic GC dosing.
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About the Writer
Dr. Anuja Badgujar, BDS is a dentist with expertise in US healthcare data and medical data annotation. With four years of experience handling US healthcare datasets, she brings strong domain knowledge and precision to her work. She is also deeply passionate about medical writing, with a focus on translating complex medical information into clear and structured content.