Drug intelligence / Profile preview

ziftomenib + cytarabine + daunorubicin

Development stage
Phase 3
Lead developer
Kura Oncology
Modality
Small Molecules
Administration
Oral, Intravenous
01

Overview

This drug is a combination therapy consisting of **ziftomenib**, **cytarabine**, and **daunorubicin**. Ziftomenib is a selective, oral small molecule inhibitor of the interaction between menin and MLL (KMT2A), developed to target menin-dependent acute myeloid leukemia, particularly in patients with NPM1 mutations or KMT2A rearrangements. Cytarabine is an antimetabolite chemotherapeutic agent that inhibits DNA synthesis by acting as a cytosine nucleoside analog, and daunorubicin is an anthracycline antibiotic that intercalates into DNA and inhibits topoisomerase II, causing DNA damage and cell death. The combination is being evaluated in clinical trials as a potential treatment for relapsed or refractory acute myeloid leukemia (AML) with NPM1 mutations, with studies assessing safety, dose, and preliminary efficacy in this patient population[2][4][6][8]. Ziftomenib alone is pending regulatory review for NPM1-mutant AML, but combination strategies are under investigation.

02

Targets

DNA polymerase familyKMT2ATOP2A (DNA topoisomerase II)MEN1 (Menin)

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