Drug intelligence / Profile preview

BP1001 + decitabine

Development stage
Unknown
Lead developer
Bio-Path Holdings
Modality
Small Molecules, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

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].

Brand names
Dacogen
Other names
prexigebersen + decitabineBP1001 + decitabine
02

Targets

DNMT1 (DNA (cytosine-5)-methyltransferase 1)Growth factor receptor-bound protein 2 mRNA (GRB2 mRNA)

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