MC4R pharmacochaperone

The MC4R pharmacochaperone (also known as UM0130866) is a small molecule drug candidate being developed by Iricor Therapeutics (IRICoR) for the treatment of metabolic disorders, specifically melanocortin-4 receptor (MC4R)-linked obesity. Its mechanism of action is pharmacochaperoning: the small molecule penetrates the cell membrane, binds to misfolded or intracellularly retained mutant MC4R, stabilizes a near-native receptor conformation, and facilitates trafficking of the receptor to the plasma membrane, thereby restoring its functional signaling. MC4R is a G protein-coupled receptor (GPCR) that plays a key role in regulating energy homeostasis, and loss-of-function mutations in MC4R are the most common cause of severe early-onset monogenic obesity. The program was identified through a collaboration with Pfizer and the medicinal chemistry platform of the Institute for Research in Immunology and Cancer (IRIC).

Other names

MC4R pharmacological chaperoneMC-4R pharmacological chaperoneMC 4R pharmacological chaperoneMelanocortin-4 receptor pharmacological chaperoneMelanocortin4 receptor pharmacological chaperoneMelanocortin 4 receptor pharmacological chaperoneMelanocortin-4 receptor pharmacochaperoneMelanocortin4 receptor pharmacochaperoneMelanocortin 4 receptor pharmacochaperone