GMX1778 is primarily investigated for its potential in cancer therapy. It functions as a potent inhibitor of nicotinamide phosphoribosyltransferase (NAMPT), an enzyme crucial for the production of NAD+ in cancer cells. By targeting NAMPT, GMX1778 disrupts the energy metabolism of cancer cells, leading to their death. This mechanism is particularly effective in tumors that rely heavily on NAD+ for their survival and growth. Research has shown promising results in preclinical studies, where GMX1778 has demonstrated significant anti-tumor activity in various cancer models, including hematologic malignancies and solid tumors. Its application is being explored both as a standalone treatment and in combination with other chemotherapeutic agents to enhance efficacy and overcome drug resistance. Clinical trials are ongoing to evaluate its safety, tolerability, and therapeutic potential in human patients.