AMX5035

AMX5035 is a potent and selective small molecule inhibitor of Ubiquitin-Specific Peptidase 1 (USP1), developed by Anrun Pharmaceutical Technology (also known as Arromax Pharma). USP1 is a deubiquitinating enzyme that plays a critical role in the DNA damage response (DDR) pathway, specifically in the deubiquitination of PCNA and FANCD2. By inhibiting USP1, AMX5035 disrupts DNA translesion synthesis and the Fanconi anemia pathway, leading to the accumulation of DNA damage and subsequent apoptosis in cancer cells. This mechanism is particularly effective in tumors with homologous recombination deficiency (HRD), such as those with BRCA1/2 mutations, where it can act as a synthetic lethal agent. AMX5035 has received Investigational New Drug (IND) clearance from both the US FDA and China's NMPA and is currently being evaluated in Phase 1 clinical trials for the treatment of advanced solid tumors, both as a monotherapy and in potential combination with other DNA-damaging agents or PARP inhibitors.