• Adult and pediatric patients with classic congenital adrenal hyperplasia treated with CRENESSITY® (crinecerfont) achieved clinically meaningful weight reductions
• Substantial improvements in insulin resistance were also observed in both adult and pediatric patients treated with CRENESSITY compared with placebo through one year of treatment

SAN DIEGO, July 14, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced the presentation of new data from the Phase 3 CAHtalyst™ Adult and Pediatric studies showing favorable trends in improvement of weight-related outcomes in patients with classic congenital adrenal hyperplasia who were treated with CRENESSITY® (crinecerfont) for up to one year. These data were presented at the Endocrine Society's Annual Meeting, ENDO 2025, that is taking place July 12-15 in San Francisco.  

The Phase 3 CAHtalyst program was the largest-ever interventional clinical trial program in classic congenital adrenal hyperplasia (CAH) and included 103 pediatric (four to 17 years of age) patients and 182 adult (18 to 58 years of age) patients. Both the CAHtalyst Pediatric study and the CAHtalyst Adult study consisted of an initial six-month, double-blind, placebo-controlled (DBPC) period (28 weeks for the pediatric study and 24 weeks for the adult study) followed by a 24-week open-label (OL) period, during which all patients received CRENESSITY. During both the DBPC and OL periods, glucocorticoid (GC) doses were kept stable for the first four weeks and then decreased as tolerated toward more physiologic levels while maintaining or improving androstenedione (A4) relative to Day 1 baseline.

Adult and pediatric patients receiving CRENESSITY for up to one year saw improvements in key weight-related outcomes:  

• Adults taking CRENESSITY experienced greater reductions in body mass index (BMI) at Week 24 versus placebo, with further reductions from baseline observed through Month 12.
• Pediatric patients taking CRENESSITY saw reductions in BMI standard deviation scores (SDS) at Week 28, while those taking placebo saw an increase. Reductions in the CRENESSITY group were maintained through Week 52.
• Among adult and pediatric patients who were overweight or obese at baseline (~70% and ~60%, respectively):
  • A higher percentage of adults achieved a greater than 5% reduction in weight at Week 24 with CRENESSITY versus placebo.
  • A higher percentage of pediatric patients achieved at least a 0.2 reduction from baseline in BMI SDS at Week 28 with CRENESSITY versus placebo.
  • Importantly, the percentage of adult and pediatric patients achieving these thresholds increased through Week 52 for both patients on continuous CRENESSITY and those who switched to CRENESSITY from placebo.
  • In those with insulin resistance, mean homeostatic model assessment for insulin resistance (HOMA-IR) was elevated at baseline; greater reductions in HOMA-IR were observed with CRENESSITY than placebo in both adult and pediatric patients at the end of the DBPC periods, and substantial reductions from baseline were observed through one year of treatment.