Neurocrine Biosciences Inc NBIX announced positive topline data from the Phase 3 CAHtalyst Adult Study evaluating crinecerfont in adults with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21-OHD).

CAH is a group of genetic conditions limiting hormone production in the adrenal glands. These glands produce hormones your body needs to function properly.

Also Read: FDA Greenlights Expanded Use Of Neurocrine Biosciences' Ingrezza: Analysts Weigh In Depression Warning.

Approximately 95% of CAH cases are caused by a mutation that leads to a deficiency of the enzyme 21-hydroxylase (21-OHD). 

The Phase 3 study met its primary endpoint at Week 24, demonstrating that treatment with crinecerfont resulted in a statistically significant percent reduction in daily glucocorticoid (GC) dose versus placebo while maintaining androgen control (p-value <0.0001).

The study also met important key secondary endpoints, with a statistically significant decrease in androstenedione at Week 4 versus placebo (p-value <0.0001). 

At Week 24, approximately 63% of patients on crinecerfont achieved a reduction to a physiologic GC dose versus approximately 18% on placebo (p-value <0.0001).

Crinecerfont was generally well tolerated. As planned, data from the Phase 3 CAHtalyst Pediatric Study will be available in early Q4 2023.

Price Action: NBIX shares are up 7.82% at $118.21 on the last check Tuesday.