Protagonist and Takeda Report Positive Phase 3 VERIFY Results for Rusfertide in Polycythemia Vera

Protagonist Therapeutics and Takeda have announced the promising results from their Phase 3 VERIFY study, which evaluated the effects of rusfertide versus a placebo in phlebotomy-dependent individuals suffering from polycythemia vera (PV), a rare blood disorder. The study achieved its primary goal, with a significantly greater proportion of participants responding positively to rusfertide compared to the placebo group. Additionally, all four pivotal secondary endpoints were met, underscoring rusfertide’s potential as an innovative treatment option for PV patients.

Rusfertide, an investigational hepcidin mimetic peptide therapeutic, has been recognized with both Orphan Drug and Fast Track designations by the U.S. Food & Drug Administration (FDA). The VERIFY study’s outcomes indicate that rusfertide could serve as a groundbreaking erythrocytosis-specific agent, particularly aiding PV patients who struggle to control hematocrit levels through standard treatments. Arturo Molina, Chief Medical Officer at Protagonist, expressed optimism about presenting further study details at upcoming medical conferences in 2025. He highlighted the significant role of patients and research teams in achieving these results.

Polycythemia vera is a condition that increases the risk of cardiovascular and thrombotic events due to an overproduction of red blood cells, leading to elevated hematocrit levels. Patients often undergo regular phlebotomy to manage these levels, a process that can be taxing and exacerbate symptoms like fatigue, visual issues, and iron deficiency, ultimately affecting quality of life. Current guidelines recommend maintaining hematocrit levels below 45% as a primary treatment goal for PV management.

Andy Plump, President of R&D at Takeda, expressed enthusiasm about rusfertide’s potential to significantly alleviate the treatment burden and severe symptoms that PV patients endure. He emphasized Takeda’s commitment to developing new therapeutic options for those battling blood cancers, including PV.

Dinesh V. Patel, President and CEO of Protagonist, noted that the extensive clinical data collected so far suggests rusfertide could have a profound positive impact on PV patients’ lives. He looked forward to collaborating with Takeda to submit the study findings to regulatory authorities. Patel also acknowledged the milestone that these results represent in Protagonist’s ten-year journey within the hepcidin program, validating their expertise in creating unique peptide-based medicines to address unmet medical needs.

As part of a licensing and collaboration agreement between Protagonist and Takeda, Protagonist is entitled to a $25 million milestone payment following these positive study results, which will be finalized after the completion of the VERIFY clinical study report. Takeda has stated that the financial impact of these results on its fiscal year ending March 31, 2025, will be minor.

The Phase 3 VERIFY trial is a comprehensive global study involving 293 PV patients over a 156-week period. It assesses the efficacy and safety of weekly self-administered rusfertide injections in patients who remain dependent on phlebotomy despite standard care, which may include treatments like hydroxyurea, interferon, or ruxolitinib. The primary endpoint was achieved by observing the absence of phlebotomy eligibility, defined by specific hematocrit criteria, in the rusfertide group.

Protagonist Therapeutics is a biopharmaceutical firm focusing on developing novel peptides through an advanced proprietary platform, with rusfertide being one of the key drugs currently in Phase 3 clinical trials. Collaborating with Takeda, they aim to advance rusfertide towards regulatory approval, potentially offering a new ray of hope for PV patients worldwide.