APTO-253 is primarily investigated for its potential in cancer therapy, particularly due to its ability to inhibit the growth of various tumor cells. It functions by disrupting the function of specific oncogenic proteins and has shown activity in inducing cell cycle arrest and apoptosis in leukemia and other hematologic malignancies. One of its key mechanisms involves the downregulation of MYC, a critical oncogene overexpressed in many cancers, making it a candidate for targeting MYC-driven tumors. APTO-253 has demonstrated favorable pharmacokinetic properties and low toxicity in preclinical studies, supporting its evaluation in clinical trials for advanced or refractory cancers. Its oral bioavailability further enhances its potential as a convenient therapeutic option in oncology.