Per Perseus Proteomics Inc. announced that the first patient has been dosed with PPMX-T003 in the Phase I/II investigator-led clinical trial for aggressive NK-cell leukemia (ANKL). This clinical trial has been prepared and performed under the support by AMED since April 2022 after adopted as Project Promoting Support for Drug Discovery Support Program for Orphan drug prior to the Designation. Its purpose is to assess the safety and tolerability in administering PPMX-T003 to ANKL patients, as well as efficacy and pharmacokinetics.

ANKL is a rare refractory hematological malignancy, with a need to establish the effective standard treatment as soon as possible. PPMX-T003 is an antibody targeting transferrin receptor 1 (TfR1) that is related to iron intake into cells. TfR1 is highly expressed in hematopoietic cells that require much more iron than usual cells, as well as cancer cells.

When PPMX-T003 binds to TfR1, it is expected to inhibit cancer cell proliferation by inhibiting iron uptake into cancer cells. The Company has been also conducting the phase I clinical trial of PPMX-T003 for polycythemia vera, where RBCs increase at an abnormal level.