Takeda and Protagonist Therapeutics have delivered promising news for patients with polycythemia vera — a rare blood disorder where the body produces too many red blood cells, thickening the blood and increasing the risk of heart problems and blood clots.
In the pivotal Phase III VERIFY study, researchers evaluated rusfertide, a first‑in‑class investigational hepcidin mimetic peptide therapeutic designed to mimic the natural hormone that regulates iron, to see if it could reduce the need for regular blood removals (phlebotomies).
Polycythemia vera is often tough to manage because its symptoms can be vague, which sometimes delays diagnosis. Patients usually need regular phlebotomies and cytoreductive treatments that may not fully control the condition, leaving them at risk for complications like blood clots.
In the Phase III VERIFY trial, patients were divided into two groups: one received rusfertide alongside their standard treatment, while the other received a placebo.
The primary goal was to determine if more patients on rusfertide could avoid phlebotomies.
Seventy-seven percent of patients treated with rusfertide managed to avoid phlebotomies between Weeks 20 and 32, compared to only 33% on the placebo.
Moreover, the rusfertide group averaged 0.5 phlebotomies per person versus 1.8 in the placebo group, with notable improvements in hematocrit control and fatigue reduction.
Rusfertide was generally well tolerated, with adverse events often characterized as mild injection site reactions and no new safety concerns.
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These findings align with a broader strategic vision at Takeda.
At its December 2024 R&D Day, Takeda highlighted its high‑value, late‑stage pipeline by announcing three anticipated Phase III data readouts in 2025 — oveporexton for narcolepsy type 1, zasocitinib for psoriasis and, of course, rusfertide for polycythemia vera.
Looking ahead, Takeda plans to file for five additional indications between fiscal years 2027 and 2029, including further applications for zasocitinib (psoriatic arthritis), mezagitamab (immune thrombocytopenia and immunoglobulin A nephropathy), fazirsiran (liver disease related to alpha‑1 antitrypsin deficiency) and elritercept (anemia associated with myelodysplastic syndrome).
The development of rusfertide is governed by a worldwide license agreement in which Takeda secured exclusive ex‑US global commercialization rights with an upfront payment of $300 million to Protagonist.
In addition, Protagonist will receive a $25 million milestone payment upon completion of the Phase III VERIFY study report — a payment that is dependent on future milestones and the subsequent commercial success of rusfertide.
The competitive landscape in polycythemia vera is intensifying.
Silence Therapeutics is advancing divesiran — a siRNA therapy targeting disease-causing genes in polycythemia vera — into Phase II, while Merck showcased bomedemstat, an investigational small molecule lysine‑specific demethylase 1 (LSD1) inhibitor as part of its hematology pipeline, at the American Society of Hematology (ASH) 2024 Annual Meeting.
Moreover, a recent market analysis noted that alongside mainstays like hydroxyurea, interferon and ruxolitinib, new contenders from companies such as Incyte and Novartis are further intensifying competition in this space.
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