Neurocrine’s Crenessity shows further efficacy in Phase 3 CAH study

Neurocrine (NBIX) announced new data from the Phase 3 CAHtalyst Adult and Pediatric studies of Crenessity – crinecerfont -. The data showed that a substantial proportion of pediatric patients with classic congenital adrenal hyperplasia, or CAH, achieved physiologic-range glucocorticoid doses and normal androstenedione levels. Additionally, adult male patients with classic congenital adrenal hyperplasia observed improvements in select reproductive hormone levels. Both adult and pediatric patients achieved substantial reductions in glucocorticoid doses. Results were presented at the 2025 American Association of Clinical Endocrinology Annual Meeting in Orlando. CAH is characterized by imbalances in hormone production, including cortisol, aldosterone and androgens, which cause a wide range of symptoms and can lead to long-term health problems. 90% of participants on CRENESSITY versus 21% on placebo achieved greater than or equal to1 threshold for A4 reduction or GC reduction. In contrast, no patients who were treated with placebo achieved a physiologic GC dose, and nearly 70% still had elevated A4 levels. Select reproductive hormone changes in adult males with classic CAH who received up to one year of Crenessity were evaluated from the CAHtalyst Adult study. A higher percentage of male patients who had abnormal levels had observed rates of normalization of LH and A4/T with Crenessity vs. placebo, respectively, despite greater decreases in GC dose in the CRENESSITY group.