elesclomol + copper

Elesclomol + copper is a combination therapeutic consisting of elesclomol (a bis(thiohydrazide) amide compound) complexed with copper in a 1:1 ratio. Elesclomol functions as a copper ionophore that transports copper across biological membranes. The mechanism involves: (1) elesclomol binding copper(II) in the extracellular environment, (2) forming a membrane-permeable complex that enters mitochondria, (3) release of copper(I) upon reduction by mitochondrial ferredoxin 1 (FDX1), and (4) making copper bioavailable for metalation of cuproenzymes, particularly cytochrome c oxidase. The drug also delivers copper to non-mitochondrial compartments through FDX1-independent mechanisms. Originally developed by Synta Pharmaceuticals, the therapeutic potential for copper deficiency disorders was discovered by researchers at Texas A&M University led by Vishal Gohil. The combination is now licensed to **Engrail Therapeutics** for development as a treatment for Menkes disease and other copper deficiency disorders.

Brand names

ENX-203ENX203ENX 203

Other names

ES-Cuelesclomol-copper complexES-copper