AI-10-49

Biological Activity

AI-10-49 is a protein-protein interaction inhibitor that selectively binds to CBFβ-SMMHC and disrupts its binding to RUNX1 with a FRET IC50 of 0.26 uM.AI-10-49 restores RUNX1 transcriptional activity, displays favorable pharmacokinetics,and delays leukemia progression in mice. Treatment of primary inv(16) AML patient blasts with AI-10-49 triggers selective cell death. These data suggest that direct inhibition of the oncogenic CBFβ-SMMHC fusion protein may be an effective therapeutic approach for inv(16) AML, and they provide support for transcription factor targeted therapy in other cancers.

Published experimental findings depend on the model and assay conditions. They do not guarantee equivalent performance in every application. For laboratory research use only.