Neurocrine Biosciences to Present Wide-Ranging One-Year Data from Phase 3 Cahtalyst?? Pediatric Study At Pediatric Endocrine Society 2025 Annual Meeting

Published: 2025-05-16 09:50:22 ET NBIX

Published on 05/16/2025 at 09:50

Neurocrine Biosciences, Inc. 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?? (crinecerfont) for up to one year.

Results also showed improvements in clinical outcomes, including body mass index and insulin resistance. These data will be presented at the Pediatric Endocrine Society (PES) 2025 Annual Meeting taking place from May 15-18, 2025, in National Harbor, MD. The Phase 3 CAHtalyst Pediatric study was part of the largest-ever interventional clinical trial program in classic congenital adrenal hyper Plasia (CAH) including 103 pediatric patients, four to 17 years of age.

GC Dosing Regimens in the Phase 3 CAHt analyst Adult and CAHtalyst Pediatric Studies. Additional data will be presented regarding changes in GC regimens in both adult and pediatric patients with classic CAH. The CAHtalyst studies were the largest-ever interventionalclinical trial program in classic CAH, including 285 pediatric and adult patients.

Data from the CAHtalyst Phase 3 studies supported approval of CRENESSITY by the U.S. Food and Drug Administration in December 2024. The robust clinical study data demonstrate that lowering adrenal androgen levels with CRENESSITY enables lower, more physiologic dosing of GCs to replace missing cortisol. CRENESSITY comes in capsules and an oral solution.

The capsule formulation is available in 50 mg and 100 mg doses. The oral solution is available as a 50 mg/mL strength formulation.