Neurocrine Biosciences (NASDAQ:NBIX) announced positive interim results from its Phase 2 proof-of-concept study evaluating NBI-74788 in adult patients with classic congenital adrenal hyperplasia (CAH).
Classic CAH is a rare genetic disorder characterized by deficiency of an enzyme responsible for production of cortisol and other adrenal steroids. This causes adrenal insufficiency, overgrowth of the adrenal glands, and excess androgen levels. The standard of care for CAH is ongoing high-dose corticosteroids, which are associated with serious long-term health problems.
NBI-74788, a corticotropin-releasing factor type 1 receptor antagonist, reduced 17-hydroxyprogesterone and adrenocorticotropic hormone levels by at least 50% from baseline in more than 50% of CAH patients. Neurocrine plans to meet with the FDA to discuss the registration program for NBI-74788 in adult and pediatric CAH patients.
