Drug intelligence / Profile preview

elesclomol

Development stage
Phase 2
Lead developer
Engrail Therapeutics
Modality
Small Molecules
Administration
Intravenous
01

Overview

Elesclomol is a novel, investigational small molecule drug that acts as a copper ionophore and induces oxidative stress in cancer cells, leading to apoptosis (programmed cell death). It functions by transporting copper into mitochondria, where it generates high levels of reactive oxygen species (ROS), overwhelming the antioxidant defenses of cancer cells and triggering mitochondrial-mediated apoptosis. Elesclomol has been studied primarily as an anticancer agent and chemotherapy adjuvant, with particular focus on metastatic melanoma. It was developed by Synta Pharmaceuticals and GlaxoSmithKline. The drug has received fast track and orphan drug status from the FDA for metastatic melanoma but clinical development has faced interruptions due to safety concerns. Elesclomol’s mechanism is distinct in its reliance on copper-dependent ROS generation to selectively target cancer cells with elevated basal oxidative stress[1][2][3][4][5][6][8].

Other names
elesclomol sodium
02

Targets

ATP7A (Copper-transporting ATPase 1)DLAT (Dihydrolipoamide S-acetyltransferase)FDX1 (Ferredoxin 1)HSP90 (Heat shock protein 90 chaperone complex)SLC7A11 (Cystine-Glutamate Antiporter)ND1 (Mitochondrial electron transport chain complex I)HSP90AA1 (Heat shock protein HSP90-alpha)

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