Phase III VERIFY trial of rusfertide in patients with PV: Positive topline results announced

Positive topline results have been announced from the ongoing, global, randomized, placebo-controlled phase III VERIFY trial (NCT05210790).¹ This trial assessed the efficacy and safety of once-weekly, subcutaneously self-administered rusfertide, an investigational hepcidin mimetic peptide therapy, vs placebo in 293 phlebotomy-dependent patients with polycythemia vera (PV).¹ The MPN Hub previously reported results from the phase II REVIVE trial of rusfertide in patients with PV.

The VERIFY trial primary endpoint was met, with a higher proportion of patients treated with rusfertide achieving a clinical response (defined as the absence of phlebotomy eligibility, during Weeks 20–32) vs placebo (77% vs 33%; p < 0.0001).¹

All four key secondary endpoints were also met, including mean number of phlebotomies during Weeks 0–32 (0.5 vs 1.8; p < 0.0001), hematocrit control, and patient-reported outcomes using Patient Reported Outcome Measurement Information System (PROMIS) Fatigue SF-8a and Myelofibrosis Symptom Assessment Form Total Symptom Score (MFSAF-TSS)-7.¹ The safety profile of rusfertide was consistent with previous reports.¹

These results highlight the potential for rusfertide as a first-in-class erythrocytosis-specific agent to address unmet treatment needs in patients with PV who are unable to achieve adequate hematocrit control with currently approved treatments.