Final safety and efficacy results from a phase 1/2 study of tagraxofusp, a CD123-targeted therapy, for myelofibrosis

A phase 1/2 trial evaluated the optimal dosing, safety, and efficacy of tagraxofusp monotherapy in treatment-naive patients with myelofibrosis who were not eligible for stem cell transplant and who had disease that is resistant/refractory to Janus kinase inhibitor therapy. Tagraxofusp monotherapy was well tolerated, did not demonstrate cumulative myelotoxicity, and was associated with symptom score improvements, supporting further investigation into combination therapy.