Neurocrine Biosciences Announces Phase 3 Pediatric Study Results of Crinecerfont in Children and Adolescents for the Treatment of Congenital Adrenal Hyperplasia Met Primary and Key Secondary Endpoints

• CAHtalyst™ Pediatric Study Met Primary Endpoint Demonstrating a Statistically Significant Decrease from Baseline in Serum Androstenedione in Children and Adolescents with Congenital Adrenal Hyperplasia
• Key Secondary Endpoint Demonstrated a Statistically Significant Decrease from Baseline in Daily Glucocorticoid Dose while Maintaining Androgen Control
• Crinecerfont Was Generally Well-Tolerated
• Company to Host Conference Call and Webcast Today at 8:00 a.m. ET with Management and Dr. Richard Auchus, Professor of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes at the University of Michigan 

SAN DIEGO, Oct. 5, 2023 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today 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 (CAH) due to 21-hydroxylase deficiency. Crinecerfont is an oral, selective corticotropin-releasing factor type 1 receptor antagonist being investigated to help reduce and control excess adrenal androgens through a steroid-independent mechanism.

"The outstanding safety and efficacy results reported today for the CAHtalyst Pediatric study and last month for the CAHtalyst Adult study demonstrate the potential benefit of crinecerfont across all groups studied, including children, adolescents and adults," said Kevin Gorman, Ph.D., Chief Executive Officer, Neurocrine Biosciences, Inc.

"As a pediatric endocrinologist, I'm highly encouraged by the results from the CAHtalyst Pediatric study and the potential of crinecerfont to shift the treatment paradigm for a disorder that has seen little innovation in many decades," said Kyriakie Sarafoglou, M.D., Professor, Department of Pediatrics and Department of Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics & Metabolism, University of Minnesota. "The data suggest that crinecerfont might enable us to smooth out the numerous adjustments we have to make in glucocorticoid doses to manage high androgen levels as children grow, potentially improving clinical outcomes related to androgen excess as well as chronic supraphysiologic glucocorticoid dosing."

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 (GC) stable period (p = 0.0002).  

Importantly and consistent with the results from the Phase 3 CAHtalyst Adult study, crinecerfont treatment led to a statistically significant percent reduction from baseline in daily GC dose while maintaining androgen control at Week 28 versus placebo (p < 0.0001). 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 at Week 4 versus placebo (p < 0.0001).