Successful Results From The Phase 3 VERIFY Study: Rusfertide Treats PV

Living with polycythemia vera (PV) often means regular blood draws, fatigue, and worrying about serious complications like blood clots. Managing the increased percentage of red blood cells can be challenging, especially when current treatments are not enough. The Phase 3 VERIFY study is investigating a new treatment option, rusfertide, that may reduce the need for frequent phlebotomies (blood draws) and improve quality of life for people with PV. In this article, you’ll learn what PV is, how rusfertide works, and what the VERIFY study results could mean for patients.

About the VERIFY Study

Results from the Phase 3 VERIFY study (NCT05210790) show strong evidence that rusfertide may help manage erythrocytosis, or high red blood cell counts, in people with polycythemia vera. For many patients, current treatments are not always enough to keep blood counts under control, creating an urgent need for new options.

The trial focused on individuals with PV who rely on frequent phlebotomies (regular blood draws) to reduce their blood thickness and prevent complications. Rusfertide significantly outperformed placebo in helping these patients maintain healthy blood levels, meeting all of the study’s main goals. Importantly, the treatment was generally well tolerated, with the most common side effect being mild reactions at the injection site.

Patients with PV face a higher risk of life-threatening cardiovascular or blood clotting events when red blood cell counts remain too high. Frequent phlebotomies can help but also come with challenges, including fatigue, iron deficiency, and vision problems that can greatly affect daily life. By reducing the need for ongoing blood draws, rusfertide may lessen this burden and improve quality of life.

How Rusfertide Works

Rusfertide mimics the action of hepcidin, a protein that controls iron absorption and release into the body. Rusfertide restricts the availability of iron for erythropoiesis (red blood cell production), which is essential for managing PV. By mimicking hepcidin, rusfertide reduces iron availability, leading to a decrease in red blood cell production and a reduction in circulating red blood cells. 

Rusfertide has shown promise in clinical studies for treating PV, including reducing the need for phlebotomies (blood draws) to control blood cell levels. It’s being investigated as a potential treatment for conditions characterized by excessive erythropoiesis (a process of producing red blood cells in the bone marrow), such as polycythemia vera, and also iron deficiency anemia and iron overload secondary to hematologic disorders. 

Rusfertide has received Orphan Drug designation and Fast-Track designation from the U.S. Food & Drug Administration (FDA).

Conclusion

The VERIFY study results show that rusfertide may become an important new treatment option for people living with PV, particularly those who depend on frequent phlebotomies to control their blood counts. By mimicking the natural protein hepcidin, rusfertide offers a different way to manage hematocrit levels and reduce treatment burden. While more research is still needed, the study findings are encouraging, and FDA Fast Track designation highlights its potential path toward approval. For patients and caregivers, these results represent hope for a future with more effective and less burdensome treatment choices.

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Keep reading more details about rusfertide here. 

Source: 

• Protagonist and Takeda Announce Positive Topline Results from Phase 3 VERIFY Study of Rusfertide in Patients with Polycythemia Vera