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Sapablursen - Ionis Pharmaceuticals

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Drug Profile

Sapablursen - Ionis Pharmaceuticals

Alternative Names: IONIS TMPRSS6-Lrx; IONIS-TMPRSS6-LRx; ISIS-702843; ONO 0530

Latest Information Update: 14 Aug 2026

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At a glance

  • Originator Ionis Pharmaceuticals
  • Developer Ionis Pharmaceuticals; Ono Pharmaceutical
  • Class Antineoplastics; Antisense oligonucleotides
  • Mechanism of Action Gene silencing; TMPRSS6 protein expression inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Polycythaemia vera
  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Polycythaemia vera
  • Discontinued Beta-thalassaemia; Thalassaemia

Most Recent Events

  • 31 Jul 2026 Phase-III clinical trials in Polycythaemia vera (Adjunctive treatment) in Taiwan, South Korea, European Union, Japan (SC) (jRCT2071260061) (Ono Pharma pipeline, July 2026)
  • 05 Jun 2026 Ono Pharmaceuticals intends to file for approval of Polycythaemia vera in USA, Europe and Japan by 2029 (Ono Pharmaceutical pipeline, May 2026)
  • 02 Jun 2026 Ionis Pharmaceuticals completes a phase II IMPRSSION trial in Polycythaemia vera in Australia, Canada, England, Hungary, Poland, United Kingdom, USA(SC) (NCT05143957) (EudraCT2021-003704-40)

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