APTO-253 is a small molecule inhibitor primarily investigated for its potential in cancer therapy, specifically targeting the MYC oncoprotein, which plays a critical role in cancer cell proliferation and survival. Its application is focused on hematologic malignancies, such as acute myeloid leukemia (AML) and lymphoma, where it has shown promise in preclinical and early clinical studies. The compound works by inhibiting MYC function, leading to cell cycle arrest and promoting apoptosis in cancer cells, making it a promising candidate for oncology treatments. Researchers are exploring its use in monotherapy and combination therapies to enhance efficacy and overcome drug resistance in various cancer types.