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ML309

Development stage: Preclinical
Lead developer: Agios Pharmaceuticals
Modality: Small Molecules

Overview

ML309 is a small molecule inhibitor of mutant isocitrate dehydrogenase 1 (IDH1), specifically targeting the R132H and R132C mutations. It is used to reduce the production of the oncometabolite 2-hydroxyglutarate (2-HG), which is implicated in various cancers such as glioblastoma and acute myeloid leukemia. ML309 demonstrates selective inhibition of mutant IDH1 over wild-type IDH1, with minimal cytotoxicity. Its mechanism involves competitive binding with respect to α-ketoglutarate and uncompetitive binding with respect to NADPH, effectively stopping the catalytic cycle that produces 2-HG.

Targets

IDH1 R132H (Isocitrate Dehydrogenase 1 R132H)

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