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BP1001 + decitabine is an investigational combination therapy for acute myeloid leukemia (AML). BP1001 (prexigebersen) is a liposome-incorporated antisense oligonucleotide targeting growth factor receptor bound protein-2 (Grb2), a key adaptor protein in oncogenic tyrosine kinase signaling pathways that drive AML progression. Decitabine is a hypomethylating agent and pyrimidine nucleoside analogue that inhibits DNA methyltransferase, leading to DNA hypomethylation and reactivation of silenced genes. The combination aims to enhance the sensitivity of AML cells to chemotherapy by disrupting Grb2-mediated signaling while simultaneously altering gene expression through epigenetic modulation. This regimen is being studied primarily in patients with newly diagnosed or relapsed/refractory AML who are unsuitable for intensive chemotherapy[3][7][9].
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