Drug intelligence / Profile preview

LMP744

Development stage
Phase 2
Lead developer
Gibson Oncology
Modality
DNA Intercalators/Alkylators → Nucleic Acid-Directed Small Molecules → Small Molecules
Administration
Intravenous
01

Overview

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].

Other names
Topoisomerase-1 Inhibitor LMP744Topoisomerase1 Inhibitor LMP744Topoisomerase 1 Inhibitor LMP744indenoisoquinoline LMP744
02

Targets

telomeric G4 (Telomeric DNA G-quadruplex)TOP1 (DNA Topoisomerase I)

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