VERIFY Trial: Rusfertide for Polycythemia Vera Patients

By Srdan Verstovsek, MD, PhD

The VERIFY trial polycythemia vera study examines Rusfertide (PTG-300), a hepcidin mimetic, for patients with polycythemia vera (PV). Presented at ASH 2022 (Poster #1709), this Phase 3 study builds on Phase 2 results. Specifically, it assesses Rusfertide’s ability to reduce phlebotomy needs. So, how does it function, and what could it mean for PV treatment?

How Rusfertide Works

Rusfertide mimics hepcidin, a key regulator of iron metabolism. Consequently, it restricts iron in the spleen, liver, and GI tract, thus reducing red blood cell production. For PV patients, this decreases phlebotomy, the standard treatment for high red blood cell counts. In Phase 2, weekly self-injected Rusfertide reduced phlebotomy needs within 2–3 weeks for most patients. Moreover, it alleviated symptoms like fatigue, shortness of breath, and concentration issues.

Unlike hydroxyurea, interferon, or ruxolitinib, which target cell growth or inflammation, Rusfertide focuses on red blood cell production, a primary PV issue.

Overview of the VERIFY Trial

The VERIFY trial polycythemia vera study is a randomized, placebo-controlled Phase 3 trial with three phases:

1. Part 1a: A 32-week double-blind phase comparing Rusfertide to placebo.

2. Part 1b: A 20-week open-label phase where all patients receive Rusfertide.

3. Part 2: A 104-week extension for continued Rusfertide use.

However, patients with recent thrombosis, significant bleeding, or invasive cancer within five years are excluded. The trial evaluates whether Rusfertide maintains hematocrit below 45%, potentially reducing clotting risks and improving quality of life.

Rusfertide vs. Other PV Treatments

Rusfertide’s hepcidin-mimicking action differs from hydroxyurea (chemotherapy), interferon (biological agent), or ruxolitinib (JAK inhibitor). For instance, it can be used alone for PV patients needing frequent phlebotomies (3–5 per year). Alternatively, it can complement hydroxyurea to further reduce phlebotomy while controlling hematocrit. Therefore, the VERIFY trial polycythemia vera study supports tailored PV management. Learn more about PV treatment options.

Implications for Clinicians and Patients

Phase 2 data indicates Rusfertide may reduce phlebotomy and clotting risks, a leading cause of death in PV. Although Phase 3 results are pending, the VERIFY trial polycythemia vera study emphasizes hematocrit control and quality of life. As a result, clinicians are refining PV treatment strategies based on these insights.

Next Steps for the VERIFY Trial

As of June 2025, the VERIFY trial polycythemia vera study continues enrolling patients to assess Rusfertide’s efficacy and safety. In the meantime, results will determine if Rusfertide could be approved for PV treatment. For updates, visit ClinicalTrials.gov.

Key Takeaways

The VERIFY trial polycythemia vera study suggests Rusfertide may:

• Reduce phlebotomy needs.

• Improve symptoms and quality of life.

• Lower clotting risks.

• Provide a new PV treatment option.

Conclusion

The VERIFY trial polycythemia vera study may expand PV treatment options. Accordingly, share your thoughts on Rusfertide’s potential in the comments. Additionally, explore MPN research for more insights.

References: ASH Publications, ClinicalTrials.gov

Image Alt Text: “Diagram of Rusfertide’s mechanism in the VERIFY trial for polycythemia vera treatment”

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