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

Protagonist Therapeutics, Inc. (NASDAQ: PTGX) and Takeda (NYSE: TAK) have announced positive topline results from the Phase 3 VERIFY study evaluating rusfertide in phlebotomy-dependent polycythemia vera (PV) patients. The study met its primary endpoint, with 77% of rusfertide-treated patients achieving hematocrit control compared to 33% in the placebo group (p<0.0001). Additionally, all four key secondary endpoints were met, including reduced phlebotomy needs and improved patient-reported outcomes. Rusfertide, a first-in-class investigational hepcidin mimetic peptide, has already received Orphan Drug and Fast Track designations from the U.S. FDA.

The safety profile of rusfertide remained consistent with previous trials, with no new safety concerns or increased cancer risk observed. The majority of side effects were mild injection site reactions, and serious adverse events were deemed unrelated to the drug. Given the promising results, Protagonist and Takeda plan to submit regulatory filings and present further findings at medical conferences in 2025. If approved, rusfertide could significantly reduce the treatment burden for PV patients, who often rely on frequent phlebotomies and cytoreductive therapies to manage their condition.