Drug intelligence / Profile preview

eltanexor + ASTX727

Development stage
Preclinical
Lead developer
Karyopharm Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

Eltanexor + ASTX727 is an investigational **combination therapy** consisting of **eltanexor** and **ASTX727** (a fixed-dose oral combination of decitabine and cedazuridine). Eltanexor is a selective inhibitor of nuclear export (SINE) that targets exportin 1 (XPO1, also known as CRM1), leading to the retention and reactivation of tumor suppressor proteins in the nucleus, affecting key oncogenic pathways and promoting apoptosis in malignant cells[2][4][6]. ASTX727, a combination of decitabine (a hypomethylating agent) and cedazuridine (a cytidine deaminase inhibitor), allows for the oral administration of decitabine by preventing its degradation, resulting in effective epigenetic modulation and antineoplastic activity[1][5]. The combination is being studied primarily for **myelodysplastic syndromes (MDS)** and **acute myeloid leukemia (AML)**.

02

Targets

DNMT1 (DNA (cytosine-5)-methyltransferase 1)XPO1 (Exportin-1)CDA (Cytidine deaminase)

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