- Data Consistent Across All Patient Subgroups, Including Demographic Subgroups, by Baseline Androstenedione Levels and by Baseline Glucocorticoid Dose
- Findings to be Presented at the 2025 Joint Congress of the European Society for Paediatric Endocrinology and the European Society of Endocrinology
SAN DIEGO, May 8, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced new results from subgroup analyses of the Phase 3 CAHtalyst™ Pediatric study. The analyses showed that, consistently across all of the different subgroups analyzed, pediatric patients with classic congenital adrenal hyperplasia maintained or improved their androstenedione levels with CRENESSITY™ (crinecerfont) while reducing glucocorticoid dosing. These data will be presented at the 2025 Joint Congress of European Society for Paediatric Endocrinology and the European Society of Endocrinology in Copenhagen, Denmark.
Overproduction of androstenedione, a key adrenal androgen, in pediatric patients with congenital adrenal hyperplasia (CAH) can lead to abnormal growth and development, premature puberty and various developmental challenges. For decades, high levels of androstenedione (A4) were treated with glucocorticoids (GCs) only.
"High-dose steroids are often accompanied by side effects and complications," said Eiry W. Roberts, M.D., Chief Medical Officer, Neurocrine Biosciences. "By enabling patients to maintain or improve their androgen levels while reducing their reliance on high-dose glucocorticoids, CRENESSITY has the potential to meaningfully enhance long-term outcomes, helping patients with both the hormonal imbalances that characterize CAH, as well as the challenges associated with chronic high-dose glucocorticoid treatment."
The CAHtalyst Pediatric study included 103 patients who were randomly assigned to receive either CRENESSITY (N=69) or a placebo (N=34) for 28 weeks. The primary endpoint was the least-squares (LS) mean change from baseline in A4 levels (before the morning GC dose) at Week 4. A key secondary endpoint was GC dose reduction at Week 28. Prespecified subgroup analyses of the primary endpoint and post-hoc subgroup analyses of GC dose reduction at Week 28 were conducted for region, sex, race, age, body mass index, pubertal stage and baseline A4 levels; and weight and baseline GC dose subgroups were also analyzed for GC dose reduction at Week 28. This comprehensive approach allowed for a thorough evaluation of treatment effects across diverse patient characteristics.
Across all subgroups, CRENESSITY enabled reduction of GC doses while maintaining or improving A4 levels:
- Substantial Reduction in Adrenal Androgens at Week 4: CRENESSITY significantly reduced A4 levels from baseline compared with placebo (overall, -6.9 versus +2.5 nmol/L; LS mean difference [LSMD]: -9.3 nmol/L; p=0.0002); subgroup analyses of A4 reduction at Week 4 were consistent with the results in the overall population.
- Substantial Reduction in GC Doses at Week 28: GC doses were significantly reduced from baseline with CRENESSITY (while maintaining or improving A4 levels) compared with placebo (overall, -18.0% versus +5.6%; LSMD: -23.5%; p