Neurocrine Biosciences Announces Publication of Primary CAHtalyst™ Pediatric Phase 3 Study Results of Crinecerfont for the Treatment of CAH in The New England Journal of Medicine

Core Insights - The CAHtalyst Pediatric Phase 3 study demonstrated that crinecerfont effectively reduced androstenedione levels and allowed for glucocorticoid dose reduction while maintaining androgen control in pediatric patients with congenital adrenal hyperplasia (CAH) [1][3][4] Group 1: Study Results - The study met its primary endpoint, showing a significant reduction in androstenedione levels with crinecerfont compared to placebo, with a least squares mean change of -196.8 ng/dL versus +71.0 ng/dL at Week 4 [4][5] - At Week 28, crinecerfont treatment resulted in a significant glucocorticoid dose reduction of -18% compared to a +5.6% increase in the placebo group, with 30% of crinecerfont participants achieving a physiologic glucocorticoid dose [5][6] - Favorable trends were observed in secondary endpoints, including reductions in body weight, improved insulin resistance, and decreased hirsutism in female participants [2][5] Group 2: Treatment Mechanism - Crinecerfont is an investigational oral selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist designed to reduce excess adrenocorticotropic hormone (ACTH) and adrenal androgens through a glucocorticoid-independent mechanism [2][10] - The mechanism of action allows for lower glucocorticoid dosing, potentially reducing complications associated with long-term supraphysiologic glucocorticoid use [11] Group 3: Safety and Tolerability - Crinecerfont was generally well tolerated, with common adverse events including headache, fever, and vomiting, and no adrenal crises reported during the study [6][9] - The incidence of adverse events leading to glucocorticoid stress dosing was similar between crinecerfont and placebo groups, indicating a favorable safety profile [6][9]