AMX6001

AMX6001 is a potent and highly selective small molecule inhibitor of Ubiquitin-Specific Peptidase 1 (USP1), developed by Anrun Pharmaceutical Technology (Arromax). USP1 is a deubiquitinating enzyme that plays a critical role in the DNA damage response (DDR) pathway by regulating the monoubiquitination of PCNA and FANCD2, which are essential for translesion synthesis and Fanconi anemia pathway-mediated DNA repair. By inhibiting USP1, AMX6001 promotes the accumulation of ubiquitinated substrates, leading to genomic instability and cell death. This mechanism is particularly effective in cancers with existing DDR deficiencies, such as those with homologous recombination deficiency (HRD) or BRCA mutations, through the principle of synthetic lethality. AMX6001 is being investigated for the treatment of advanced solid tumors, including ovarian and triple-negative breast cancers, and has shown potential in overcoming resistance to PARP inhibitors.