Sapablursen (TMPRSS6 antisense oligonucleotide)
Current understanding
To be populated as evidence accumulates.
Evidence log
- 2026-09-01 — FDA Approves Mimrylo (rusfertide) for Polycythemia Vera: cross-connection with rusfertide: Both agents target the hepcidin axis for erythrocytosis control in PV; rusfertide’s approval validates the hepcidin-mimetic/hepcidin-elevating class. (novelty: 4)
- 2026-08-31 — FDA Approves First-of-its-Kind Drug for Polycythemia Vera (Rusfertide/Mimrylo): cross-connection with rusfertide: Both target the hepcidin axis in PV — rusfertide as a direct hepcidin mimetic (now FDA-approved), sapablursen by suppressing TMPRSS6 to raise endogenous hepcidin. (novelty: 4)
- 2026-06-01 — A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera: Early-phase clinical trial evaluating sapablursen (ISIS 702843 / IONIS-TMPRSS6-LRx) in polycythemia vera, testing whether TMPRSS6 antisense knockdown upregulates hepcidin to restrict iron availability for erythropoiesis, with co-primary endpoints of reduced phlebotomy frequency and improved quality of life — first-in-class hepcidin-axis approach distinct from JAK inhibitors and cytoreductive agents. (novelty: 4)
Open questions
Related
Contributing findings
A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera
01-jun-2026
novelty 4
per-area 4
introduces