Durability of Hematocrit Control in Polycythemia Vera with the First-in-Class Hepcidin Mimetic Rusfertide: Two-Year Follow up Results from the Revive Study

Background: Polycythemia vera (PV) is a myeloproliferative neoplasm associated with uncontrolled erythrocytosis, systemic symptoms, and an increased risk of thromboembolic and cardiovascular complications largely driven by uncontrolled hematocrit (HCT) levels. Rusfertide is a hepcidin mimetic that controls red blood cell production in PV patients by limiting iron availability. The REVIVE trial (PTG-300-04; NCT04057040) is investigating rusfertide and consists of three stages. Eligibility criteria: diagnosis of PV; ≥3 therapeutic phlebotomies (TP)s in the 28 weeks prior to enrollment with or without concurrent cytoreductive therapies (CYTO). As reported previously, adding rusfertide to standard of care PV treatments optimally controls HCT levels and decreases the need for TP (Kuykendall et al., HemaSphere, 2022;6:(S3):1766; Kremyanskaya et al. EHA2023; Abstract LB2710). Herein, we report results from the ongoing open-label extension (OLE) from REVIVE (Part 3) and explore the long-term durability of response to rusfertide. Methods: During Part 1 (Week 1-29), treatment with rusfertide was initiated at 20 mg and individually titrated to control hematocrit (