Neurocrine Biosciences, Inc. (NBIX) announced on Monday that their CAHtalyst Pediatric and Adult Phase 3 studies of Crinecerfont for congenital adrenal hyperplasia (CAH) have achieved their primary objectives.
CAH is a genetic disorder affecting the adrenal glands, which are crucial for hormone production.
The CAHtalyst Adult Phase 3 study successfully met its primary endpoint along with key secondary endpoints, including androgen reduction and glucocorticoid (GC) dose reduction while maintaining androgen control. In the study, 62.7% of participants receiving Crinecerfont attained a physiological glucocorticoid dose with androgen control, compared to only 17.5% of those on a placebo.
Similarly, the Phase 3 Pediatric study achieved its primary endpoint, showing a change from baseline in androstenedione after an initial 4-week GC-stable period. By week 28, 30% of participants treated with Crinecerfont reached a physiological glucocorticoid dose while maintaining androgen control, a result not mirrored in placebo-treated participants. The study also achieved its key secondary endpoint of percent change from baseline in GC dose.
These findings have been published online by The New England Journal of Medicine.
Neurocrine Biosciences stated that the data from both the CAHtalyst Pediatric and Adult Phase 3 studies will support two New Drug Application submissions to the U.S. Food and Drug Administration, scheduled for April 2024.
