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Discovery of Alternative Binding Poses through Fragment-Based Identification of DHODH Inhibitors.

Lindsey G DeRattE Christine PietschJustin S CisarEdgar JacobyFaraz KazmiRosalie MaticoPaul ShafferAlexandra TannerWeixue WangRicardo AttarJames P EdwardsScott D Kuduk
Published in: ACS medicinal chemistry letters (2024)
Dihydroorotate dehydrogenase (DHODH) is a mitochondrial enzyme that affects many aspects essential to cell proliferation and survival. Recently, DHODH has been identified as a potential target for acute myeloid leukemia therapy. Herein, we describe the identification of potent DHODH inhibitors through a scaffold hopping approach emanating from a fragment screen followed by structure-based drug design to further improve the overall profile and reveal an unexpected novel binding mode. Additionally, these compounds had low P-gp efflux ratios, allowing for applications where exposure to the brain would be required.
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