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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.
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