SCO-483

SCO-483 is a selective small molecule antagonist of the melanocortin 2 receptor (MC2R), originally discovered by SCOHIA and currently being developed in partnership with Neurocrine Biosciences. By targeting MC2R, the drug aims to block overactive adrenocorticotropic hormone (ACTH) signaling, which is a primary driver of excess cortisol or androgen production in conditions such as classic Congenital Adrenal Hyperplasia (CAH) and ACTH-dependent Cushing's Syndrome. Preclinical studies have demonstrated its efficacy in suppressing ACTH-induced physiological changes. The drug is being explored for both daily oral administration and as a long-acting injection for the treatment of rare metabolic and endocrine disorders.