Drug intelligence / Profile preview

CM03

Development stage
Preclinical
Lead developer
UCL School of Pharmacy
Modality
Small Molecules
Administration
Intravenous
01

Overview

CM03 is a naphthalene diimide (ND) derivative small molecule that functions as a potent DNA G-quadruplex (G4) ligand. Developed by researchers at the UCL School of Pharmacy, it is primarily being investigated for the treatment of pancreatic ductal adenocarcinoma (PDAC). The compound works by binding to and stabilizing G-quadruplex structures within DNA, which leads to the downregulation of G4-containing oncogenes and induces torsional strain that results in double-strand DNA breaks. Preclinical studies have demonstrated that CM03 possesses significant anticancer activity in both gemcitabine-sensitive and gemcitabine-resistant PDAC models, showing a superior efficacy profile compared to standard chemotherapy.

Other names
2101208-44-8
02

Targets

telomeric G4 (Telomeric DNA G-quadruplex)

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