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

ABSTRACT: Patients with myelofibrosis (MF) who are resistant to or relapse after Janus kinase inhibitor (JAKi) therapy have limited treatment options and typically poor prognoses. CD123 is overexpressed in various myeloid malignancies, including MF. Tagraxofusp is a first-in-class CD123-targeted therapy, and the only drug approved globally for the rare myeloid malignancy blastic plasmacytoid dendritic cell neoplasm. We conducted a phase 1/2 trial to determine optimal dosing, and evaluate safety and efficacy of tagraxofusp monotherapy in treatment-naïve (n = 5) MF and patients with MF resistant/refractory to JAKi (n = 25) and not eligible for stem cell transplant. There were no dose-limiting toxicities. The recommended phase 2 dose of tagraxofusp was 12 μg/kg per day for 3 consecutive days per cycle.

SUBMITTER: Yacoub A 

PROVIDER: S-EPMC12554100 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature