ASCO

ASCO 2025: Investigational drug significantly reduces phlebotomy need in polycythemia vera

June 6, 2025

Rusfertide offers a promising non-cytotoxic alternative for managing polycythemia vera (PV), reducing phlebotomy burden and improving quality of life, according to results shared at the ASCO 2025 annual meeting.

Study details: VERIFY, a global, phase 3, double-blind, placebo-controlled trial, evaluated rusfertide in 293 patients with PV requiring frequent phlebotomy. Participants were randomized 1:1 to weekly rusfertide or placebo for 32 weeks, with or without stable cytoreductive therapy. The primary endpoint was clinical response: no phlebotomy eligibility or procedures from weeks 20–32 and completion of the blinded phase.

Results: Rusfertide significantly outperformed placebo across all key measures. Between weeks 20–32, 76.9% of patients on rusfertide met the clinical response criteria, compared with 32.9% with placebo (p<0.0001). Over the full 32 weeks, rusfertide patients needed fewer phlebotomies (0.5 vs. 1.8; p<0.0001) and were more likely to maintain hematocrit <45% (62.6% vs. 14.4%; p<0.0001). They also reported less fatigue and symptom burden (p<0.03). The most common side effects with rusfertide were injection site reactions, anemia, and fatigue. Serious adverse events were rare and similar between groups, and fewer new malignancies occurred in the rusfertide group (1 vs. 7).

Source:

Kuykendall, A. (2025, June). Results from VERIFY, a phase 3, double-blind, placebo (PBO)-controlled study of rusfertide for treatment of polycythemia vera (PV). American Society of Clinical Oncology (ASCO) 2025 Annual Meeting, Chicago, IL.