MIMRYLO (Rusfertide) - (VERIFY)

August 28, 2026: Protagonist Therapeutics (NASDAQ: PTGX) said Takeda received FDA approval for MIMRYLO (rusfertide), a subcutaneous hepcidin mimetic peptide, for treatment of erythrocytosis in adults with polycythemia vera (PV). The company says this is the first approved hepcidin mimetic for PV.

Approval was based on the Phase 3 VERIFY randomized, double-blind, placebo-controlled trial. In the study, 76.9% of patients receiving MIMRYLO plus standard of care achieved a clinical response during weeks 20-32 versus 32.9% on placebo plus standard of care. The press release says MIMRYLO showed statistically significant benefit over placebo on responder rate, mean number of phlebotomies, proportion maintaining hematocrit below 45%, and mean change from baseline in PROMIS Fatigue Short Form 8a at week 32, meeting all four key secondary endpoints. The label also reflects improvement in fatigue. The most common adverse reactions over 15% were injection-site reactions (56%) and anemia (16%).

Under the 2024 Takeda collaboration, the approval triggers $275 million to Protagonist ($200 million opt-out fee plus $75 million approval milestone), with up to $875 million in additional milestones and tiered royalties of 14%-29%. Protagonist scheduled an investor call for August 31, 2026.