Neurocrine reports data from Phase 3 CAHtalyst Pediatric study

Neurocrine (NBIX) Biosciences announced it will present new data from the Phase 3 CAHtalyst Pediatric study showing lasting reductions in glucocorticoid doses in pediatric patients with classic congenital adrenal hyperplasia who received Crenessity, or crinecerfont, for up to one year. In addition, the study showed that for patients on Crenessity, adrenocorticotropic hormone, 17-hydroxyprogesterone and androstenedione remained below baseline levels, despite substantially decreased glucocorticoid doses. Results also showed improvements in clinical outcomes, including body mass index and insulin resistance. These data will be presented at the Pediatric Endocrine Society 2025 Annual Meeting taking place from May 15-18 in National Harbor, MD. The Phase 3 CAHtalyst Pediatric study was part of the largest-ever interventional clinical trial program in classic congenital adrenal hyperplasia including 103 pediatric patients, four to 17 years of age. The trial consisted of a 28-week, double-blind, placebo-controlled period, during which patients with classic CAH on supraphysiologic glucocorticoid doses were randomized 2:1 to receive CRENESSITY or placebo, and a 24-week, open-label period, during which all patients received CRENESSITY. During both the DBPC and OL periods, GC doses were kept stable for the first four weeks and then decreased as tolerated toward more physiologic levels while maintaining or improving androstenedione relative to Day 1 baseline. Three separate analyses examined the effect of CRENESSITY on Changes in serum A4 levels and GC doses; changes in adrenocorticotropic hormone and 17-hydroxyprogesterone levels. Long-term, sustained reductions in high GC doses, in addition to improvements in key clinical outcomes, were observed in pediatric patients with up to one year of treatment with CRENESSITY: Reductions in supraphysiologic GC doses observed with 28 weeks of treatment with Crenessity were maintained during long-term treatment of up to one year.Crenessity was generally well tolerated, with no adrenal crises reported in the DBPC period. The most common adverse reactions with Crenessitywere headache abdominal pain, fatigue, nasal congestion and epistaxis. Additional data will be presented regarding changes in GC regimens in both adult and pediatric patients with classic CAH.