In patients with polycythemia vera (PV) receiving standard of care (SOC) therapy, treatment with rusfertide resulted in a statistically significant reduction in the mean number of phlebotomies and improved hematocrit control, as well as better patient-reported outcomes, according to results from VERIFY, a phase 3, double-blind, placebo-controlled study.
The results were presented at a plenary session at the 2025 ASCO(R) Annual Meeting in Chicago.
Rusfertide is a subcutaneous, self-injected, first-in-class peptide hepcidin mimetic that decreases erythrocytosis. The ongoing VERIFY trial is designed to assess rusfertide vs placebo in phlebotomy-dependent patients with PV receiving SOC.
The study, led by Andrew Kuykendall, MD, of Moffitt Cancer Center, Tampa, Florida, recruited patients requiring frequent phlebotomies with or without stable cytoreductive therapy to control hematocrit.
A total of 293 patients (male, 73.0%; median age, 57 [27-86] years) were randomized to receive rusfertide (n=147) or placebo (n=146). In the rusfertide and placebo groups, 56.5% (n=83) and 55.5% (n=81) of patients, respectively, received concurrent cytoreductive therapy.
The results for weeks 20-32 showed that significantly more patients in the rusfertide group (76.9%) achieved a clinical response vs placebo (32.9%) (P<0.0001), where clinical response was defined as the absence of phlebotomy eligibility and no phlebotomies during weeks 20-32. The mean (SE) number of phlebotomies (weeks 0-32) was 0.5 (0.2) with rusfertide vs 1.8 (0.2) with placebo (P<0.0001). More patients treated with rusfertide maintained hemocrit <45% from weeks 0-32 vs placebo (rusfertide, 62.6%; placebo, 14.4%; P<0.0001).
In terms of patient-reported outcomes, patients treated with rusfertide demonstrated a statistically significant improvement in the PROMIS Fatigue Short Form 8a (SF-8a) total T-score and MFSAF v4.0 Total Symptom Score.
“Rusfertide is the first investigational agent to target the hepcidin pathway to control hematocrit and the first agent to prospectively demonstrate a statistically significant improvement in the PROMIS Fatigue SF-8a and MFSAF patient-reported outcomes in patients with PV,” Dr. Kuykendall and colleagues wrote.
Reference
Kuykendall A, Pemmaraju N, Pettit K, et al. Results from VERIFY, a phase 3, double-blind, placebo (PBO)-controlled study of rusfertide for treatment of polycythemia vera (PV). Presented at the 2025 ASCO® Annual Meeting; May 30-June 3, 2025; Chicago.
