Protagonist And Takeda Report Positive Phase 3 Results For Rusfertide In Polycythemia Vera

Protagonist Therapeutics and Takeda have announced that the Phase 3 VERIFY trial of rusfertide in polycythemia vera (PV) patients met its primary and all key secondary endpoints, highlighting the therapy’s potential in controlling hematocrit and reducing the need for phlebotomy. PV is a blood disorder marked by the overproduction of red blood cells, which increases blood viscosity and the risk of serious thrombotic events. Current treatments often fail to consistently maintain hematocrit levels below the target threshold of 45%.

“PV poses significant challenges for patients, including debilitating symptoms and the risk of serious thrombotic events, and hematocrit control is crucial to improving patient outcomes. The VERIFY study demonstrated that treatment with rusfertide controls hematocrit levels in phlebotomy-dependent patients, including patients receiving cytoreductive therapies. These results suggest rusfertide has the potential to become part of the standard of care treatment for patients with PVs,” said Dr. Andrew T. Kuykendall, M.D., VERIFY Lead Investigator and Associate Member in the Department of Hematology at Moffitt Cancer Center. 

Rusfertide, a first-in-class hepcidin mimetic peptide, works by regulating iron metabolism and red blood cell production. In the VERIFY trial, 76.9% of patients on rusfertide plus standard of care achieved a clinical response (no need for phlebotomy between Weeks 20–32), compared to only 32.9% in the placebo group—a highly significant difference (p<0.0001). These results were consistent across different patient subgroups, regardless of risk status or use of other therapies.

“These findings underscore Rusfertide’s potential as a first-in-class erythrocytosis-specific treatment for PV and validate more than a decade of scientific innovation originating from Protagonist’s peptide technology platform. We would like to thank all the patients, study staff and investigators for participating in the VERIFY study. We are pleased to partner with Takeda as we continue to advance rusfertide to potentially transform the standard of care in PV patients around the world,” said Dinesh V. Patel, Ph.D., President and Chief Executive Officer at Protagonist. 

The treatment was well tolerated, with most adverse events being mild and no serious events attributed to rusfertide. Notably, cancer incidence was lower in the rusfertide group than in the placebo group at the time of analysis. Rusfertide has received Orphan Drug and Fast Track designations from the FDA, further reinforcing its promise as a new option for PV patients.

“These promising pivotal data strongly support rusfertide's potential benefit for a broad spectrum of patients with PV who may be receiving current standard of care therapies but not achieving adequate hematocrit control. We look forward to receiving additional data from the VERIFY trial later this year, advancing rusfertide towards regulatory approval and continuing our collaboration with Protagonist to bring this innovative therapy to patients,” said Phuong Khanh (P.K.) Morrow, M.D., Head of the Oncology Therapeutic Area Unit (OTAU) at Takeda.