AMXI-5001 (AMXI 5001) is a novel, highly potent, orally active dual PARP1/2 (IC50 5/0.05 nM) and microtubule polymerization inhibitor, inhibits intracellular PAR formation with IC50 of 7 nM.
AMXI-5001 binds to the catalytic domain of human PARP1 and is a weak tankyrase inhibitor (800-fold lower than IC50 towards either PARP1 or PARP2 enzymes).
AMXI-5001 inhibited tubulin polymerization in a dose-dependent manner.
AMXI-5001 exhibited selective antitumor cytotoxicity across a wide variety of human cancer cells with much lower IC50s than existing clinical PARP1/2 inhibitors.
AMXI-5001 is highly active in both BRCA mutated and wild type cancers.
AMXI-5001 elicited a remarkable In vivo preclinical anti-tumor activity in a BRCA mutated TNBC model induced complete regression of established tumors, including exceedingly large tumors, demonstrated superior anti-tumor effects compared to either single agent (PARP or microtubule) inhibitor or combination with both agents.