amxi-5001

AMXI-5001 is a novel, orally bioavailable small molecule that acts as a dual inhibitor of poly(ADP-ribose) polymerase 1 and 2 (PARP1/2) and microtubule polymerization. Its mechanism involves binding to the catalytic domain of human PARP1, thereby inhibiting DNA repair processes critical for cancer cell survival, while simultaneously targeting the colchicine binding site on tubulin to disrupt microtubule formation. This dual action results in potent antitumor activity across a wide range of human cancer cells—including both BRCA-mutated and wild-type cancers—with much lower IC50s than existing clinical PARP inhibitors. Preclinical studies have shown complete regression of established tumors in models such as BRCA-mutated triple-negative breast cancer (TNBC). AMXI-5001 demonstrates superior efficacy compared to single-agent or combination therapies with current PARP or microtubule inhibitors. The drug is being developed by AtlasMedx for use in cancers including breast and ovarian cancer, with ongoing Phase I/II clinical trials[1][4][5][6][7].

Other names

PARP/microtubule polymerization inhibitor AMXI-5001bifunctional PARP and microtubule polymerization inhibitor - AtlasMedx