Drug intelligence / Profile preview

tagraxofusp + azacitidine

Development stage
Unknown
Lead developer
Stemline Therapeutics
Modality
Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes, Bacterial Toxin Conjugates → Immunotoxins → Antibody Conjugates → Antibody-Based Therapeutics, Covalent Small Molecules → Small Molecules, Classical Binding Small Molecules → Small Molecules
Administration
Intravenous
01

Overview

Tagraxofusp + azacitidine is a combination therapy being evaluated as a maintenance treatment for patients with CD123-positive acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) following allogeneic hematopoietic cell transplantation (HCT). Tagraxofusp is a first-in-class recombinant fusion protein consisting of human interleukin-3 (IL-3) fused to a truncated diphtheria toxin payload. It specifically targets the CD123 receptor (IL-3Rα), which is highly expressed on the surface of AML and MDS malignant cells, delivering the toxin to inhibit protein synthesis and induce cell death. Azacitidine is a hypomethylating agent that acts as a DNA methyltransferase inhibitor, promoting cell differentiation and exerting direct cytotoxic effects. This combination regimen is being investigated by the City of Hope Medical Center to improve outcomes and prevent disease relapse in high-risk post-transplant settings.

Brand names
ElzonrisVidaza
Other names
tagraxofusp-erzsCD123 Antibody Toxin CongregateCD-123 Antibody Toxin CongregateCD 123 Antibody Toxin Congregate
02

Targets

EEF2 (Eukaryotic elongation factor 2)IL3RA (Interleukin 3 Receptor)DNMT1 (DNA (cytosine-5)-methyltransferase 1)

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