GNE617

GNE617 is a potent and selective small-molecule inhibitor of nicotinamide phosphoribosyltransferase (NAMPT), the rate-limiting enzyme in the nicotinamide adenine dinucleotide (NAD+) salvage pathway. Developed by Genentech, GNE617 works by depleting intracellular NAD+ levels, which leads to metabolic stress, ATP depletion, and subsequent cell death in cancer cells that are highly dependent on this pathway. It has been extensively studied in preclinical models for various malignancies, including small cell lung cancer (SCLC) and ovarian cancer. In research settings, GNE617 is often used as a reference compound to validate NAMPT as a therapeutic target and has shown synergistic anti-tumor effects when combined with other agents, such as the aldehyde dehydrogenase (ALDH) inhibitor disulfiram.