EPX-105287

EPX-105287 is a potent and selective small molecule inhibitor of dihydroorotate dehydrogenase (DHODH), a key enzyme in the de novo pyrimidine biosynthesis pathway located in the inner mitochondrial membrane. Developed by Epics Therapeutics, the compound is primarily being investigated for the treatment of acute myeloid leukemia (AML). By blocking DHODH activity, EPX-105287 depletes intracellular uridine monophosphate (UMP) levels, which triggers metabolic stress and induces the differentiation of myeloid blasts into mature cells, ultimately leading to apoptosis. The drug, which is also known by the clinical code EPX-115, has shown promising preclinical activity in various hematological malignancy models and is currently undergoing Phase 1 clinical evaluation to determine its safety, tolerability, and preliminary efficacy in patients with relapsed or refractory AML.