Drug intelligence / Profile preview

bleximenib + cytarabine + daunorubicin or idarubicin

Development stage
Unknown
Lead developer
Janssen Research & Development
Modality
Small Molecules
Administration
Oral, Intravenous
01

Overview

This drug regimen is a **combination therapy** for the treatment of newly diagnosed acute myeloid leukemia (AML) with NPM1 mutation or KMT2A rearrangement, composed of bleximenib, cytarabine, and either daunorubicin or idarubicin. - **Bleximenib** is a potent, selective menin-KMT2A complex inhibitor, disrupting the interaction between menin and the KMT2A fusion protein, leading to the suppression of leukemogenic gene expression driven by the mutated KMT2A or NPM1. - **Cytarabine** is a nucleoside analog and antimetabolite incorporated into DNA, inhibiting DNA synthesis and inducing apoptosis in rapidly dividing cells, particularly myeloid leukemic blasts. - **Daunorubicin and idarubicin** are anthracycline antibiotics that intercalate into DNA and inhibit topoisomerase II, causing double-strand breaks and apoptosis in cancer cells. The combination is used in an intensive induction chemotherapy regimen (“7+3”: cytarabine for 7 days, daunorubicin or idarubicin for 3 days) along with oral bleximenib, followed by consolidation with intermediate-dose cytarabine plus bleximenib for patients achieving complete remission. This regimen is investigational and currently being evaluated in phase 1b clinical trials for safety and preliminary efficacy in AML patients eligible for intensive chemotherapy[1][3][7].

02

Targets

KMT2ADNATOP2A (DNA topoisomerase II)

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