Core Insights - Neurocrine Biosciences, Inc. announced significant variability in glucocorticoid treatment regimens for patients with classic congenital adrenal hyperplasia (CAH), highlighting the complexity of managing the condition over a lifetime [1][2][4] Treatment Patterns and Findings - The CAHtalog registry analysis included data from 98 patients, revealing that 88.9% experienced at least one health state change, with 58.7% undergoing three or more changes during a median observation period of 8.6 years [3][5] - Most patients were exposed to supraphysiologic doses of glucocorticoids and/or elevated androstenedione levels at least once, indicating ongoing challenges in achieving long-term disease control [8][10] Implications for Future Treatment - The analysis emphasizes the need for advanced therapeutic options to manage CAH effectively, as current glucocorticoid treatments often require high doses that can lead to significant complications [6][12] - CRENESSITY (crinecerfont), a new treatment option, aims to reduce ACTH and adrenal androgens through a non-glucocorticoid mechanism, potentially allowing for more physiologic glucocorticoid dosing [14][15]

Core Insights - Neurocrine Biosciences presented new data from the Phase 3 CAHtalyst studies showing that CRENESSITY® (crinecerfont) leads to significant improvements in weight-related outcomes for patients with classic congenital adrenal hyperplasia (CAH) [1][2][5] Group 1: Study Overview - The Phase 3 CAHtalyst program is the largest interventional clinical trial for classic CAH, involving 285 patients (103 pediatric and 182 adult) [2][12] - The studies included a double-blind, placebo-controlled period followed by an open-label period where all patients received CRENESSITY [2][12] Group 2: Weight-Related Outcomes - Adult patients treated with CRENESSITY showed a mean BMI reduction of 0.8 kg/m² compared to a 0.4 kg/m² reduction in the placebo group at Month 12 [4][6] - 39% of adult patients on CRENESSITY achieved over a 5% weight reduction, compared to 14% in the placebo group [4][6] - Pediatric patients on CRENESSITY maintained reductions in BMI standard deviation scores (SDS) through Week 52, while those on placebo saw an increase [6][4] Group 3: Insulin Resistance Improvements - Significant reductions in insulin resistance were observed in both adult and pediatric patients treated with CRENESSITY compared to placebo [5][6] - The mean HOMA-IR score decreased more significantly in the CRENESSITY group than in the placebo group [6][7] Group 4: Safety Profile - CRENESSITY demonstrated a favorable safety profile, with common side effects being mild to moderate and not leading to discontinuation of the drug [8][20] - Common side effects in children included headache and stomach pain, while adults reported fatigue and headache [8][22] Group 5: Regulatory and Market Context - Data from the CAHtalyst studies supported the FDA approval of CRENESSITY in December 2024 [15] - CRENESSITY is positioned to evolve the standard of care for classic CAH by allowing for reduced glucocorticoid doses while maintaining or improving androgen control [2][16]

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]