The Future of Rusfertide Research in Polycthemia Vera
In an interview with Targeted OncologyTM, Andrew Kuykendall, MD, assistant member at Moffitt Cancer Center in the Department of Malignant Hematology, discusses the future research of the hepcidin mimetic agent rusfertide for the treatment of patients with polycythemia vera.
Beyond the initial 32-week findings from part 1A, the VERIFY study (NCT05210790) is designed for extensive long-term follow-up. In part 1b (weeks 32-52), all patients, including those previously on placebo, will receive rusfertide. This will be followed by part 2, a long-term safety assessment where patients will continue on the drug. During these later phases, researchers will have increased flexibility to adjust concomitant cytoreductive therapies, aiming to optimize dosing and minimize side effects.
Crucial long-term objectives include assessing thrombotic risk, disease progression, and the overall safety profile of rusfertide. While the initial 32-week data on blood count control and symptom reduction are clinically significant, future research will concentrate on dissecting patient-reported outcomes. A key question is whether all patients experience similar symptomatic benefits, or if specific subgroups derive greater relief, especially since some participants entered the study with few symptoms.
Further research will explore rusfertide's potential for earlier intervention in the disease course, particularly for newly diagnosed patients with highly uncontrolled red blood cell counts who require frequent phlebotomies. The current trial primarily targeted patients with established, consistent phlebotomy needs. Additionally, there's significant interest in evaluating rusfertide as a frontline combination therapy alongside cytoreductive agents, which could offer enhanced and rapid disease control, potentially delaying disease progression.
