Sapablursen (TMPRSS6 inhibitor / hepcidin-axis RNA interference)

Current understanding

To be populated as evidence accumulates.

Evidence log

• 2026-06-15 — A Study of Rusfertide in Japanese Adults With Polycythemia Vera: cross-connection with rusfertide-pv: Both are hepcidin-axis therapies (mimetic vs. TMPRSS6 ASO) being evaluated for phlebotomy reduction in PV, representing converging strategies to restrict erythropoiesis via iron-limitation. (novelty: 3)
• 2026-06-01 — A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera: cross-connection with sapablursen-tmprss6-antisense: Likely duplicate pages for the same agent (sapablursen / ISIS 702843 / IONIS-TMPRSS6-LRx) — consider merging. (novelty: 4)
• 2026-02-04 — Treating Anemia in Myelofibrosis With Repurposed Drugs (Nelfinavir) That Restore Iron Delivery to the Bone Marrow: cross-connection with nelfinavir-myelofibrosis: Both target the iron/hepcidin axis to address MF-associated anemia, though via distinct mechanisms (nelfinavir restoring iron delivery to marrow vs. sapablursen modulating hepcidin via TMPRSS6 RNAi). (novelty: 4)
• 2026-02-24 — A Study of Sapablursen Evaluating the Safety and Efficacy in Participants With Polycythemia Vera (PV): Phase 2 trial (early/ongoing) evaluating sapablursen added to standard of care in PV across three sequential parts — Part 1a (blinded), Part 1b (open-label), and Part 2 (long-term extension) — with treatment duration up to 156 weeks; first-in-class RNAi targeting TMPRSS6 to suppress hepatic TMPRSS6 expression, raising hepcidin and inducing iron-restricted erythropoiesis as a phlebotomy-sparing mechanism. (novelty: 4)

Open questions

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