Drug intelligence / Profile preview

eicosenol

Development stage
Preclinical
Lead developer
Berg
Modality
Small Molecules
01

Overview

Eicosenol is a 20-carbon fatty alcohol that acts as a first-in-class inhibitor of peroxisomal fatty acid oxidation (pFAO) by selectively binding to and reducing the expression of the ATP-binding cassette subfamily D member 1 (ABCD1) transporter. Developed through a collaboration involving BPGbio and the University of Guelph, eicosenol targets the metabolic dependency of Acute Myeloid Leukemia (AML) cells on pFAO. By blocking the import of very-long-chain fatty acids (VLCFAs) into peroxisomes, the drug induces lipotoxicity through the accumulation of toxic VLCFAs and the depletion of essential long-chain fatty acids like palmitate. In preclinical models, eicosenol has demonstrated selective toxicity against AML blasts while sparing normal hematopoietic cells, which can compensate via glycolysis.

Other names
20-carbon fatty alcohol
02

Targets

ABCD1

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