Rusfertide (PTG-300) treatment in phlebotomy-dependent polycythemia vera patients

Rusfertide (PTG-300) is a hepcidin mimetic used in two Phase 2 clinical trials as a non-cytoreductive option to consistently control HCT < 45% in patients with PV. Results from these trials show consistent control of hematocrit <45% with near elimination of therapeutic phlebotomy needs in patients with previously sub-optimally controlled erythrocytosis. Additionally, these patients had normalization of serum ferritin, MCV values and iron deficiency. Patients treated with rusfertide had statistically significant improvement in symptom burden at week 28. The results of these studies report good tolerance of rusfertide with mostly grade 1-2 adverse events. Phase 3 clinical trial currently ongoing.