MPCI

MPCI is a small molecule research compound that acts as a selective ligand and pharmacological chaperone for the melanocortin 4 receptor (MC4R). Chemically identified as 2-(5-bromo-2-methoxyphenethyl)-N-(N-((1-ethylpiperidin-4-yl)methyl)carbamimidoyl)-3-fluorobenzamide, it was investigated for its potential to treat severe early-onset obesity caused by misfolding mutations in the MC4R gene. MPCI is a cell-permeant antagonist that binds to mutant MC4R proteins within the cell, stabilizing their near-native conformation and facilitating their proper trafficking from the endoplasmic reticulum to the cell surface. By increasing the density of functional receptors at the plasma membrane, MPCI aims to restore melanocortin signaling. Although it demonstrated efficacy in academic research settings supported by Amicus Therapeutics, it has not progressed into clinical development.

Other names

2-(5-bromo-2-methoxyphenethyl)-N-(N-((1-ethylpiperidin-4-yl)methyl)carbamimidoyl)-3-fluorobenzamideCAS 884538-31-2CAS884538-31-2CAS-884538-31-2