▲ReadoutGlobal3 Mar 20251 min read
Rusfertide meets its phase 3 goal in polycythemia vera
A hepcidin mimetic keeps blood counts in range without regular blood draws for most patients in the VERIFY trial.
Holds at Phase 3 The molecule is closer to patients than before.
The readPhlebotomy is cheap, old and unpleasant. A weekly injection has to beat it on quality of life, and VERIFY gives the first large-trial evidence that it can.
The result
Polycythemia vera is a blood cancer in which the bone marrow makes too many red cells. Thick blood raises the risk of clots, so the standard approach is to remove blood regularly. VERIFY added rusfertide or placebo to standard care.
The primary endpoint was the share of patients who did not become eligible for a phlebotomy between weeks 20 and 32. About three quarters of patients on rusfertide responded, against about a third on placebo, according to the companies. Patient-reported fatigue and other symptoms also improved.
How it works
Hepcidin is the body’s iron gatekeeper. Rusfertide copies it, keeping iron away from the marrow, which then makes red cells more slowly. It is a peptide mimetic, not a copy of the natural hormone, built to last long enough for weekly self-injection.
Who owns it
Protagonist Therapeutics discovered rusfertide. Takeda licensed worldwide rights in 2024; Protagonist runs development through the US filing. Protagonist’s platform also produced icotrokinra, discovered jointly with Johnson & Johnson scientists, which J&J later filed in the US.
- Stage stays at phase 3.
- Signal up: primary and key secondary endpoints met.
Updated 3 November 2026: source links added. An earlier version said Takeda leads the filings; under the deal Protagonist runs development through the filing, and the two companies filed together in December 2025.
The new drug application the partners planned, and longer follow-up on safety.
Primary sourceProtagonist and Takeda press releases, VERIFY phase 3 topline, March 2025
Sources
- Protagonist and Takeda Announce Positive Topline Results from Phase 3 VERIFY Study of Rusfertide in Patients with Polycythemia Vera (joint press release, 3 March 2025)
- VERIFY: rusfertide in patients with polycythemia vera (ClinicalTrials.gov NCT05210790)
- Takeda, Protagonist Submit NDA for Rusfertide in Polycythemia Vera (Takeda press release, December 2025)
- FDA Approval of MIMRYLO (rusfertide) (Takeda press release, 2026)
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