BS-003

BS-003 is a preclinical-stage small molecule inhibitor of the MDM2-p53 protein-protein interaction, developed by Biosight for the treatment of hematological malignancies. The drug targets the MDM2 (Mouse double minute 2 homolog) protein, an E3 ubiquitin ligase that negatively regulates the tumor suppressor protein p53. In many cancers, MDM2 is overexpressed, leading to the excessive degradation of p53 and the loss of its protective apoptotic and cell-cycle regulatory functions. By binding to the p53-binding pocket of MDM2, BS-003 prevents this interaction, thereby stabilizing p53 and restoring its ability to induce cell cycle arrest and apoptosis in cancer cells that retain wild-type p53. This targeted approach is intended to provide a novel therapeutic option for patients with leukemias and other blood cancers who express functional p53.