NBIX News & Events

Neurocrine Biosciences announced the initiation of its Phase 2 clinical study to assess the safety and tolerability of crinecerfont in children aged 3 months to under 4 years with classic congenital adrenal hyperplasia. Crinecerfont, marketed as CRENESSITY, is approved in the United States as an adjunctive treatment to glucocorticoid replacement to control androgens in adult and pediatric patients 4 years of age and older with classic CAH. Separately, Neurocrine achieved target enrollment for a Phase 2 study in the European Union to evaluate the safety and tolerability of crinecerfont in children from birth to under 2 years of age with classic CAH.

Neurocrine Biosciences announced clinical findings from the first retrospective case series in pediatric and adult patients with classic congenital adrenal hyperplasia due to 11beta-hydroxylase deficiency. This subtype was not previously studied in clinical trials of CRENESSITY and is the second most common form of classic CAH after 21-hydroxylase deficiency, accounting for approximately 5% of cases. The findings were presented at the Endocrine Society's annual meeting, ENDO 2026, in Chicago. Androstenedione and other adrenal hormone levels in 11beta-hydroxylase-deficient patients improved substantially after initiation of CRENESSITY, with greater than90% median reductions in 11-deoxycortisol and 11-deoxycorticosterone. Nearly all patients (14/15) reduced their total glucocorticoid dose with CRENESSITY, and 2 of 5 patients on antihypertensive medications reduced or discontinued these drugs. Findings provide initial clinical insights in patients with classic congenital adrenal hyperplasia due to 11beta-hydroxylase deficiency, a rare subtype not previously studied in clinical trials of CRENESSITY.