Neurocrine Biosciences Presents One-Year Data Showing Sustained Efficacy of CRENESSITY® (crinecerfont) in Adult Patients, at ENDO 2025

Core Insights - Neurocrine Biosciences presented new one-year data from the CAHtalyst™ Adult study of CRENESSITY® (crinecerfont), demonstrating effective management of glucocorticoid doses and hormonal levels in patients with classic congenital adrenal hyperplasia (CAH) [1][5][6] Study Overview - The Phase 3 CAHtalyst Adult study involved 182 adult patients aged 18 to 58, focusing on the ability of CRENESSITY to manage ACTH and adrenal steroid imbalances while allowing for lower glucocorticoid dosing [2][15] - The study included a 24-week double-blind placebo-controlled period followed by a 24-week open-label period, where patients transitioned to CRENESSITY [2][15] Key Findings - Patients receiving CRENESSITY achieved a mean glucocorticoid dose reduction of 25% from baseline, while the placebo group saw a 30% reduction [4] - Serum androstenedione levels were maintained or improved, and ACTH and 17-hydroxyprogesterone levels were kept at or below baseline levels [6][4] - Improvements in insulin resistance and hirsutism were noted among female participants, with hirsutism VAS scores decreasing by 11.5 mm in the CRENESSITY group [4][6] Safety Profile - CRENESSITY exhibited a favorable safety profile, with headache and fatigue being the most common side effects, primarily occurring during the double-blind period [8][6] Future Presentations - Additional data on weight-related outcomes and other analyses will be presented at the Endocrine Society's Annual Meeting, ENDO 2025 [6][9] Background on CAH - Congenital adrenal hyperplasia (CAH) is a rare genetic condition caused by enzyme deficiencies affecting adrenal steroid hormone production, leading to excess androgen production if untreated [10][11] - CRENESSITY is designed to reduce ACTH and adrenal androgens through a non-glucocorticoid mechanism, allowing for more physiologic glucocorticoid dosing [17][19] Company Overview - Neurocrine Biosciences is focused on developing treatments for neurological and neuroendocrine disorders, with a portfolio that includes FDA-approved therapies for various conditions, including CAH [27]