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]

"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 pa ...