Interim Phase I Safety Study of Anti-Transferrin Receptor Antibody(PPMX-T003) in Patients with Polycythemia Vera (ASH 2023)
Except for an antibody drug conjugate (CX-2029), this is the first-in-class trial of an unmodified human antibody other than the murine P1a trial conducted in the 1990s. AEs are mild as observed within the level of previous healthy volunteer's trial. Efficacy observed in all erythroid parameters including hematocrit, hemoglobin and other parameter shown symptoms seen in iron deficiency anemia. This antibody can specifically suppress erythrocyte differentiation by inhibiting iron influx in the target erythroid lineage, which cells are highly expressed TfR1, without any significant AE on other normal organs with low expression level of TfR.