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Synonyms: Ionis-tmprss6-lrx | ISIS 702843 | ISIS-702843 | ISIS702843
Compound class:
Nucleic acid
Comment: Sapablursen (ISIS 702843) is a N-acetyl galactosamine (GalNAc3) modified antisense oligonucleotide that is designed to reduce production of transmembrane protease serine 6 messenger (TMPRSS6) protein [1], as a mechanism to elevate expression of hepcidin and stabilise iron homeostasis in hematologic diseases.
Primary nucleotide sequence is provided without annotation of base and bond modifications, or glycan conjugation. |
| No information available. |
Summary of Clinical Use  |
| Sapablursen (ISIS 702843) is a clinical candidate for the management of polycythemia vera (PV). It was was granted FDA Fast Track designation and orphan drug designation to treat PV in 2024. A phase 2 study in β-thalassemia intermedia was terminated due to lack of efficacy determined mid-study. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT05143957 | A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera | Phase 2 Interventional | Ionis Pharmaceuticals, Inc. | ||
| NCT04059406 | Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) | Phase 2 Interventional | Ionis Pharmaceuticals, Inc. | ||
