SMS1-IN-1 is a selective inhibitor of sphingomyelin synthase 1 (SMS1), an enzyme involved in sphingolipid metabolism. It is primarily used in research settings to study the role of sphingomyelin and related lipid mediators in cellular processes. By inhibiting SMS1, this compound helps modulate the balance of bioactive lipids such as ceramide and sphingomyelin, which are critical in regulating cell membrane integrity, apoptosis, and stress signaling.

It has been applied in studies exploring cancer cell survival, as altered sphingolipid metabolism is linked to tumor progression and drug resistance. Researchers use SMS1-IN-1 to investigate how disrupting sphingomyelin synthesis affects cancer cell proliferation and response to therapies. It is also used in metabolic and neurodegenerative disease research, where lipid homeostasis plays a key role. Due to its specificity, it serves as a valuable tool for probing SMS1-dependent pathways without significantly affecting other sphingolipid enzymes.