Drug intelligence / Profile preview

ziftomenib + venetoclax + azacitidine

Development stage
Unknown
Lead developer
Kura Oncology
Modality
Small Molecules
Administration
Oral, Intravenous, Subcutaneous
01

Overview

This combination refers to the simultaneous use of ziftomenib, venetoclax, and azacitidine for the treatment of acute myeloid leukemia (AML), especially in patients harboring nucleophosmin 1 mutations (NPM1-m). - **Ziftomenib** is a selective, oral small molecule inhibitor targeting the menin-MLL (KMT2A) interaction, thereby disrupting leukemogenic gene expression programs critical in AML subtypes with NPM1 mutations or KMT2A rearrangements. - **Venetoclax** is a small molecule inhibitor of the anti-apoptotic protein BCL-2, restoring apoptotic processes in malignant cells. - **Azacitidine** is a nucleoside metabolic inhibitor (hypomethylating agent) that incorporates into RNA and DNA, resulting in reduced DNA methylation and direct cytotoxicity to abnormal hematopoietic cells. This triple combination is under evaluation in randomized, double-blind, placebo-controlled phase 3 clinical trials for newly diagnosed, untreated NPM1-mutant AML in adults, specifically as a non-intensive regimen[5].

02

Targets

BCL-2 (BCL-2 family)MEN1 (Menin)DNMT (DNA methyltransferase)

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