Drug intelligence / Profile preview

cenersen + idarubicin

Development stage
Phase 2
Lead developer
Genta
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules
Administration
Intravenous
01

Overview

cenersen + idarubicin is an investigational combination therapy evaluated primarily for the treatment of refractory or relapsed acute myeloid leukemia (AML). Cenersen is a phosphorothioate antisense oligonucleotide targeting the tumor suppressor gene TP53 (p53). Its mechanism sensitizes AML stem cells to DNA-damaging agents by inhibiting p53 expression, thus potentially enhancing the cytotoxicity of chemotherapy. Idarubicin is an anthracycline chemotherapeutic agent that intercalates DNA and inhibits topoisomerase II, leading to DNA breaks and cell death. Together, the combination is designed to increase the susceptibility of leukemic cells to chemotherapy-induced genomic damage. This regimen was studied with or without cytarabine in salvage AML, primarily via intravenous administration. Clinical trials to date have focused on safety and efficacy in populations with poor prognosis. No unique toxicities attributable to cenersen were observed in combination with idarubicin, and the safety profile aligned with expectations for these agents[1][2][3].

02

Targets

TP53 (Cellular Tumor Antigen p53 R175H)TOP2A (DNA topoisomerase II)

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