This compound is a selective inhibitor of the MDM2 protein (Mouse Double Minute 2), which plays a crucial role in regulating the p53 protein, a key tumor suppressor in the body. Normally, MDM2 binds to p53 and promotes its degradation, thereby reducing p53's effectiveness in preventing cancer cell formation. This substance functions by effectively binding to MDM2, thereby releasing p53 to exert its full activity, leading to cell cycle arrest or apoptosis in cancer cells.

Laboratory and animal studies have demonstrated its anti-tumor activity against various cancer cell types, particularly those retaining wild-type p53 responsive to activation, such as leukemia, lung cancer, and breast cancer. It is regarded as a highly promising lead compound for the development of future cancer therapeutics.

Its high specificity and well-defined mechanism of action may reduce side effects compared to conventional chemotherapy. However, it remains in the research and development phase and has not yet been approved for broad clinical use.