Andrew T. Kuykendall, MD, Associate Member in the Department of Hematology at Moffitt Cancer Center, discusses results from the VERIFY clinical trial in polycythemia vera (PV).
PV is a rare condition characterized by an increased number of red blood cells in the bloodstream. Affected people may also have excess white blood cells and platelets. These extra cells cause the blood to be thicker than normal, increasing the risk for blood clots, including deep vein thrombosis (DVT). The condition has been associated with genetic changes in the JAK2 and TET2 genes.
Rusfertide is an investigational, first-in-class hepcidin mimetic peptide therapy with potential to regulate iron homeostasis and red blood cell production, therefore controlling hematocrit levels in patients with PV.
VERIFY Clinical Trial
The VERIFY clinical trial is a phase 3, ongoing, three-part, global, randomized, placebo-controlled study evaluating rusfertide in patients with PV. The study enrolled 293 patients with PV who were dependent on frequent phlebotomy, with or without treatment with cytoreductive therapy. Patients were randomized to receive once-weekly, subcutaneously self-administered rusfertide or placebo, plus standard of care treatment.
Data from the clinical trial was recently presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Il. The primary endpoint, the proportion of patients achieving a clinical response, defined as absence of phlebotomy eligibility during weeks 20-32, was met. Results showed that 76.9% of patients treated with rusfertide plus standard of care achieved a clinical response compared to 32.9% in the placebo plus standard of care group.
All key secondary endpoints also met statistical significance including the mean number of phlebotomies; 0.5 in the rusfertide group, compared to 1.8 in the placebo group during weeks 0-32. Patients requiring a phlebotomy between weeks 0-32 was at 27% in the rusfertide group versus 78% in the placebo group. 62.6% of patients treated with rusfertide maintained hematocrit levels below 45% compared to 14.4% treated with placebo. Rusfertide also illustrated improvements in mean change from baseline to week 32 in PROMIS Fatigue and MFSAF Total Symptom Score.
Additionally, rusfertide had a favorable safety profile and was generally well tolerated. Most adverse events were low grade and non-serious. The most common adverse events were localized injection site reactions, anemia, and fatigue. No serious adverse events were reported. Rusfertide also showed no evidence of increased risk of cancer as compared to placebo.
For more information, click here.
To learn more about PV and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/
