Drug intelligence / Profile preview

azacitidine + venetoclax + filgrastim

Development stage
Phase 2
Lead developer
AbbVie
Modality
Small Molecules, Recombinant Proteins and Enzymes
Administration
Intravenous, Oral, Subcutaneous
01

Overview

Azacitidine + venetoclax + filgrastim is a combination regimen used primarily in the treatment of acute myeloid leukemia (AML), especially in patients who are elderly, unfit for intensive chemotherapy, or have relapsed/refractory disease. - **Azacitidine** is a hypomethylating agent that incorporates into DNA and RNA, inhibiting DNA methyltransferase and leading to hypomethylation of DNA. This reactivates silenced genes involved in cell differentiation and apoptosis. - **Venetoclax** is a selective small molecule inhibitor of B-cell lymphoma 2 (BCL-2), an anti-apoptotic protein overexpressed in AML cells. By inhibiting BCL-2, venetoclax promotes apoptosis of malignant cells. - **Filgrastim** is a recombinant human granulocyte colony-stimulating factor (G-CSF) that stimulates the production and release of neutrophils from the bone marrow, often used to reduce neutropenia associated with chemotherapy. This triplet regimen leverages azacitidine’s epigenetic modulation to sensitize leukemic cells to venetoclax-induced apoptosis while filgrastim supports neutrophil recovery during therapy-induced cytopenias. The combination has been studied mainly for AML but may be considered for other hematologic malignancies as well[1][3][7].

02

Targets

BCL-2 (BCL-2 family)DNMT1 (DNA (cytosine-5)-methyltransferase 1)CSF3R (Granulocyte colony-stimulating factor receptor)

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