Neurocrine Reports Positive Two-Year Phase 3 Data For Crenessity In Pediatric CAH

(RTTNews) - Neurocrine Biosciences Inc. (NBIX) on Monday reported positive two-year data from its Phase 3 CAHtalyst Pediatric study of Crenessity in children and adolescents with classic congenital adrenal hyperplasia (CAH).

The data showed that pediatric patients with advanced bone age at baseline experienced slowed bone age progression and improved predicted adult height after up to two years of treatment.

In a subset of 41 growing patients, bone age and predicted adult height were assessed at 24 months. Among 24 patients with advanced bone age at baseline, bone age scores were stable or improved in most cases, with a mean decrease of 1.12 standard deviation score. A subset of nine patients showed reductions greater than two standard deviations, alongside a mean increase of 4.7 cm in predicted adult height.

The company said the findings suggest that sustained hormonal control and reduced glucocorticoid use enabled by Crenessity may support improved growth outcomes in childhood and adolescence.

These data were presented at the Endocrine Society's annual meeting, ENDO 2026, in Chicago.

Crenessity, which was approved by the U.S. Food and Drug Administration in 2024 for patients aged four years and older with classic CAH, is used alongside glucocorticoid replacement therapy to control excess androgen levels.

NBIX shares rose more than 1% in pre-market trading after closing at $159.76 on Friday.