Drugs / sapablursen
last change Jun 2026 re-read 3 minutes ago

sapablursen

also known as IONIS TMPRSS6-Lrx · ISIS 702843

Antisense oligonucleotide targets TMPRSS6 via inhibition

Developed for
acquired polycythemia vera · beta-thalassemia intermedia
Investigated by
Ionis Pharmaceuticals, Inc. · Deciphera Pharmaceuticals, LLC · Ono Pharmaceutical Co., Ltd.

Trials 4

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT03165864 May 2017 → Sep 2018 thalassemia Ionis Pharmaceuticals, Inc. Completed No outcome recorded
Phase 22 trials · 1 met primary · 1 missed
Phase 2 NCT05143957 Dec 2021 → Jun 2025 acquired polycythemia vera Ionis Pharmaceuticals, Inc. Completed Met primary
Phase 2 NCT04059406 Sep 2020 → Mar 2023 beta-thalassemia intermedia Ionis Pharmaceuticals, Inc. Terminated Missed primary
Phase 31 trial
Phase 3 NCT07429266 May 2026 → Sep 2028 expected acquired polycythemia vera Ono Pharmaceutical Co., Ltd. Recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 2

DateIssuerRelease
2025-12-08 Ono Pharmaceutical Co., Ltd. Results Ono Announces Oral Presentation of Positive Topline Results from Phase 2a Study of Sapablursen in Polycythemia Vera at the 67th American Society of Hematology (ASH) Annual Meeting ono-pharma.com ↗
Results from Phase 2a IMPRSSION study demonstrate sapablursen significantly reduced phlebotomy rate, controlled hematocrit and increased serum hepcidin
2025-12-06 Deciphera Pharmaceuticals, LLC Results Deciphera Announces Oral Presentation of Positive Topline Results from Phase 2a Study of Sapablursen in Polycythemia Vera at the 67th American Society of Hematology (ASH) Annual Meeting deciphera.com ↗
The results were presented by Ionis Pharmaceuticals, who discovered and developed sapablursen and conducted the IMPRSSION study.

Evidence & citations 7 cited values

Every value below carries the sentence it was read from. 6 sources stand behind the page.

FieldValueCited text
Known as Sapablursen ClinicalTrials.gov intervention name — accepted as the source's own label NCT07429266 ↗
2

NCT05143957 ↗

NCT04059406 ↗

Known as IONIS TMPRSS6-Lrx ClinicalTrials.gov intervention name — accepted as the source's own label NCT03165864 ↗
Known as ISIS 702843 ChEMBL registry synonym — accepted as the source's own label CHEMBL4650478 ↗
Action Inhibit “reduces the production of transmembrane protease serine 6 (TMPRSS6)” PMID 42241700 ↗ Jun 2026
Modality Antisense oligonucleotide “Sapablursen is an antisense oligonucleotide (ASO)” PMID 42241700 ↗ Jun 2026
Route Subcutaneous “IONIS TMPRSS6-Lrx administered subcutaneously” NCT03165864 ↗
Target TMPRSS6 “reduces the production of transmembrane protease serine 6 (TMPRSS6)” PMID 42241700 ↗ Jun 2026