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ACC010 is a novel small molecule inhibitor targeting bromodomain-containing protein 4 (BRD4). It has been developed primarily for the treatment of acute myeloid leukemia (AML), particularly in cases with FMS-like tyrosine kinase 3-internal tandem duplication (FLT3-ITD) mutations. Preclinical and early clinical studies indicate that ACC010 inhibits cell proliferation, induces apoptosis, and arrests the cell cycle in AML cells. Mechanistically, it downregulates MYC expression and inhibits FLT3 activation. Notably, combination therapy with homoharringtonine (HHT) has shown synergistic effects in overcoming drug resistance associated with WNT/β-catenin pathway activation. The drug's development status is currently suspended at Phase 1[1][2][3][4].
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