EHA 2025 | Anti-tumor activity and hematopoietic restoration achieved with tagraxofusp in treatment-naïve BPDCN

Yeah, so tagraxofusp is the first targeted therapy that is approved for BPDCN. It targets the antigen called CD123 that is highly expressed on BPDCN cells and it’s actually the ligand for that conjugated to the toxin. So the drug gets internalized and then it kills the BPDCN cells in fairly selective fashion. With that, there’s always a concern that it will cause more myelosuppression because the CD123 is expressed on some normal hematopoietic stem cells, although it’s not as much as some other antigens. And also the time of recovery CD123 can be upregulated. So the question we asked by reanalyzing the data from the pivotal trial is whether this particular therapy is associated with myelosuppression, which has been seen with some other CD123 targeted drugs unfortunately. Analysis of all the data show that there’s really no, like very quick count recovery, both as far as neutrophils and platelet count recovery. There’s no further delays of the cycles because of myelosuppression. And even patients who presented with neutropenia or thrombocytopenia after they achieved remission, their counts remain stable. And so the drug is really kind of safe as far as normal hematopoiesis and that of course it’s good to know in the first place but it also allows for potential future combinations with chemotherapy or HMA based regimens that are currently being sought to be explored

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