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LMP744 is a small molecule topoisomerase I inhibitor of the indenoisoquinoline class developed for the treatment of cancer. It binds to and stabilizes cleaved DNA-topoisomerase I complexes, preventing DNA re-ligation and repair, which leads to stable, irreversible DNA strand breaks, cell cycle arrest, and apoptosis[1][2]. LMP744 has demonstrated potent activity against both topoisomerase I (TOPO 1) and the cMyc oncogene[5]. The drug is notable for its ability to cross the blood-brain barrier at concentrations significantly higher than those required to kill cancer cells and sustain these levels for over 24 hours per dose[5][8]. It has received orphan drug designation from the FDA for glioma/glioblastoma treatment. Clinical studies have evaluated its safety and efficacy in patients with advanced solid tumors or lymphoma who have progressed after other treatments[4][7].
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