AVN-944 is primarily investigated for its potential in cancer therapy. It functions as an inhibitor of inosine monophosphate dehydrogenase (IMPDH), an enzyme crucial for the synthesis of guanine nucleotides. By inhibiting IMPDH, AVN-944 disrupts the production of DNA and RNA in rapidly dividing cells, such as cancer cells, thereby inhibiting their proliferation. This mechanism makes it a promising candidate for treating various cancers, particularly those with high rates of cell division. Research has explored its efficacy in hematologic malignancies and solid tumors, often in combination with other chemotherapeutic agents to enhance therapeutic outcomes. Additionally, its role in inducing apoptosis in cancer cells while sparing normal cells has been a focus of preclinical studies.