Neurocrine Biosciences has reported positive top-line results from the Phase III CAHtalyst Pediatric study of crinecerfont for the treatment of congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency.

The global, registrational study assessed the tolerability, efficacy and safety of the corticotropin-releasing factor type 1 receptor antagonist crinecerfont in children and adolescents.

It comprised a 28-week placebo-controlled, double-blind, randomised period followed by 24 weeks of open-label crinecerfont treatment and an optional open-label extension.

The open-label treatment portion of the study began in July 2021 and is still ongoing. 

A total of 103 children and adolescents aged two to 17 years were enrolled to receive the treatment orally.

The study met its primary endpoint, demonstrating a decrease from baseline in serum androstenedione at week four against placebo following a glucocorticoid (GC) stable period (p = 0.0002). 

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A decrease in serum 17-hydroxyprogesterone from baseline at week four versus placebo (p < 0.0001) was also observed in patients receiving crinecerfont treatment, a key secondary endpoint.

Neurocrine Biosciences CEO Kevin Gorman said: “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.”

In the Phase III CAHtalyst Adult study, treatment with crinecerfont resulted in a statistically significant percentage reduction from baseline in daily GC dose while maintaining androgen control at week 28 versus placebo.

Crinecerfont is being developed for reducing and controlling excess adrenal androgens through a steroid-independent mechanism.

The company is also engaged in developing treatments for neuropsychiatric, neuroendocrine and neurological disorders.