Neurocrine announces CAHtalyst Pediatric Phase 3 study met primary endpoint

Neurocrine Biosciences announced that the primary study results from its CAHtalyst Pediatric Phase 3 study investigating crinecerfont for the treatment of congenital adrenal hyperplasia due to 21-hydroxylase deficiency have been published in The New England Journal of Medicine online edition and will appear in a future print issue of the journal. The CAHtalyst Pediatric Phase 3 study met the primary and key secondary endpoints related to androgen reduction and glucocorticoid dose reduction while maintaining androgen control. Favorable trends were observed with endpoints that reflect the consequences of long-term supraphysiologic glucocorticoid therapy and androgen excess. In addition to appearing in The New England Journal of Medicine, CAHtalyst Pediatric Phase 3 data were presented at ENDO 2024. The Phase 3 Pediatric study met the primary endpoint of change from baseline in androstenedione following the initial 4-week GC-stable period. Crinecerfont treatment led to a significantly greater reduction in androstenedione compared to an increase with placebo at Week 4 .