Mechanism of action of the anti-cancer drug GMX1778 identified

Metabolon, Inc., the leader in metabolomics-driven biomarker discovery and analysis, today announced a new study has been published identifying the mechanism of action of the anti-cancer drug GMX1778. Co-authored by scientists at Gemin X Pharmaceuticals and Metabolon, the paper “The Small Molecule GMX1778 is a Potent Inhibitor of NAD⁺ Biosynthesis: Strategy for Enhanced Therapy in NAPRT1-Deficient Tumors” appears online ahead of print in Molecular and Cellular Biology.

The compound GMX1778 is a very promising cancer therapeutic drug currently in phase I clinical trials. However, despite years of intensive research, its mode of action remained unclear. In this publication, the authors describe how, by using an untargeted metabolomic approach based on Metabolon’s proprietary platform, the mode of action of GMX1778 was clearly identified to be in the inhibition of nicotinamide adenine dinucleotide (NAD+) biosynthetic pathway. A series of follow up experiments performed by Gemin X scientists convincingly validated this finding and further narrowed down the target to be nicotinamide phosphoribosyltransferase (NAMPT), a key regulatory enzyme in NAD biosynthesis. Because of its unique mode of action, GMX1778 can be used as a novel therapeutic approach in the treatment of human cancers.