Sapablursen - Ionis Pharmaceuticals
Alternative Names: IONIS TMPRSS6-Lrx; IONIS-TMPRSS6-LRx; ISIS-702843; ONO 0530Latest Information Update: 02 Mar 2026
At a glance
- Originator Ionis Pharmaceuticals
- Class Antineoplastics; Antisense oligonucleotides
- Mechanism of Action Gene silencing; TMPRSS6 protein expression inhibitors
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Orphan Drug Status
Yes - Polycythaemia vera
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Phase II Polycythaemia vera
- Discontinued Beta-thalassaemia; Thalassaemia
Most Recent Events
- 18 Feb 2026 Ono Pharmaceutical plans a phase III trial for Polycythemia Vera (SC) in June 2026 (NCT07429266)
- 08 Dec 2025 Adverse events and efficacy data from a phase IIa IMPRSSION trial in Polycythemia vera released by Ono Pharmaceutical
- 06 Dec 2025 Pharmacodynamics, efficacy and adverse events data from phase II IMPRSSION clinical trials in Polycythaemia vera presented at the 67th American Society of Hematology Annual Meeting and Exposition (ASH-2025)