Neurocrine’s crinecerfont meets primary and secondary endpoints in Phase 3 trial

Neurocrine announced positive top-line data from the Phase 3 CAHtalyst Pediatric Study evaluating the efficacy, safety, and tolerability of crinecerfont in children and adolescents with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency. Crinecerfont is being investigated to help reduce and control excess adrenal androgens through a steroid-independent mechanism. The Phase 3 Pediatric study met its primary endpoint, demonstrating that treatment with crinecerfont resulted in a statistically significant decrease in serum androstenedione from baseline at Week 4 versus placebo following a glucocorticoid stable period. Crinecerfont treatment led to a statistically significant percent reduction from baseline in daily GC dose while maintaining androgen control. Approximately 30% of participants receiving crinecerfont achieved a reduction to a physiologic GC dose while maintaining androgen control compared to 0% of participants receiving placebo. The study also met the other key secondary endpoint demonstrating a statistically significant decrease in serum 17-hydroxyprogesterone from baseline. Crinecerfont was generally well tolerated. There were few serious adverse events, with none assessed as related to crinecerfont. The data from the CAHtalyst Pediatric and Adult studies will support regulatory submissions to the FDA in 2024 and later to the European Medicines Agency.