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Sapablursen - Ionis Pharmaceuticals

Drug Profile

Sapablursen - Ionis Pharmaceuticals

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

Latest Information Update: 30 Jun 2023

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At a glance

  • Originator Ionis Pharmaceuticals
  • Class Antineoplastics; Antisense oligonucleotides
  • Mechanism of Action TMPRSS6 protein inhibitors
  • Orphan Drug Status

    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

  • 03 May 2023 Discontinued - Phase-II for Beta-thalassaemia in Turkey, Thailand, Lebanon, Australia, Greece, USA (SC)
  • 06 Apr 2023 Discontinued - Phase-I for Thalassaemia (In volunteers) in Australia (SC) (Ionis Pharmaceuticals pipeline, April 2023)
  • 28 Mar 2023 Ionis Pharmaceuticals terminates a phase IIa trial in Beta-thalassaemia in Australia, Lebanon, Thailand, Greece, Turkey (SC), due to favourable safety and tolerability are seen, but efficacy results in the mid-stage study do not meet Ionis' minimum target product profile to justify further development (NCT04059406)
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