MMV007285

MMV007285 is a small molecule inhibitor of the mitochondrial enzyme phosphatidylserine decarboxylase (PISD). Originally identified from the Medicines for Malaria Venture (MMV) Malaria Box, it has been repurposed for the study and potential treatment of acute myeloid leukemia (AML). The compound works by blocking the conversion of phosphatidylserine (PS) to phosphatidylethanolamine (PE) within the mitochondria, leading to an accumulation of intracellular PS. This metabolic alteration triggers Toll-like receptor (TLR) signaling, specifically upregulating TLR4 and TLR8, which promotes terminal differentiation and reduces the self-renewal capacity (stemness) of AML cells. Preclinical evidence indicates that MMV007285 reduces the growth and viability of AML cell lines and primary patient samples, and impairs leukemic engraftment in xenograft mouse models without significant systemic toxicity.

Other names

7-chloro-N-(4-ethoxyphenyl)-4-quinolinamine