Core Insights - Neurocrine Biosciences announced positive Phase 3 data for CRENESSITY (crinecerfont) in treating congenital adrenal hyperplasia (CAH), showing significant improvements in hormone levels and glucocorticoid dose reductions in both pediatric and adult patients [1][3][16] Group 1: Study Results - In the pediatric study, 90% of participants on CRENESSITY achieved at least one threshold for androstenedione reduction or glucocorticoid reduction, compared to 21% on placebo [7][8] - At Week 28, 30% of pediatric participants on CRENESSITY reached a physiologic glucocorticoid dose while maintaining or improving androstenedione levels, whereas no placebo participants achieved this [8] - In the adult study, at Week 24, 47% of male patients on CRENESSITY normalized luteinizing hormone (LH) levels compared to 22% on placebo [10] Group 2: Treatment Mechanism - CRENESSITY is a novel oral corticotropin-releasing factor type 1 receptor antagonist that aims to reduce excess adrenal androgens and allow for lower glucocorticoid doses, addressing complications from high-dose steroid use [3][17] - The drug works by decreasing adrenocorticotropic hormone (ACTH) levels, which in turn reduces the production of adrenal androgens, potentially alleviating symptoms associated with CAH [17] Group 3: Safety and Tolerability - CRENESSITY was generally well tolerated in pediatric patients, with no adrenal crises reported during the double-blind treatment period [5] - The most common adverse reactions in pediatric patients included headache (25% vs. 6% for placebo) and abdominal pain (13% vs. 0%) [5] - In adult patients, 1.6% experienced adrenal crisis while on CRENESSITY, with common side effects including fatigue (25% vs. 15% for placebo) and headache (16% vs. 15%) [10]