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Open Access CC BY-NC-ND V4.0
Journal article
Sapablursen for Erythrocytosis Control in Patients with Polycythemia Vera
Blood
2026
PMID: 42611791
Abstract
Polycythemia vera is characterized by erythrocytosis and increased risk of thrombosis. Sapablursen is an antisense oligonucleotide that suppresses transmembrane serine protease 6 (TMPRSS6), increases hepcidin production, and reduces iron availability for erythropoiesis. IMPRSSION; an ongoing phase 2a, randomized, open-label study includes patients meeting 2016 WHO criteria for polycythemia vera, requiring ≥3 phlebotomies within 6 months of screening and ≥1 phlebotomy within the last 12 weeks. Cohort A (120 or 80 mg) and Cohort B (40 mg) received sapablursen subcutaneously once every 4 weeks (Q4W). The primary efficacy endpoint was the reduction in phlebotomy frequency from baseline to weeks 17-37 in patients who received ≥1 dose. Forty-nine patients received ≥1 sapablursen dose (median age 61 years; 82% male). The baseline mean±SD weekly phlebotomy rate was 0.15±0.07 in Cohort A; 0.17±0.07 in Cohort B and was reduced by -0.10 (95%CI, -0.13, -0.07; n=32;p<0.0001) in Cohort A; -0.10 (95%CI, -0.14, -0.06; n=17;p=0.0001) in Cohort B. Sapablursen reduced the median (IQR) number of phlebotomies from 5.0 (3.0-6.0,Cohort A; 3.5-6.5,Cohort B) at baseline (26-weeks) to 0 (0-1.0) in the efficacy evaluation window (20-weeks) in Cohort A and 1.5 (0-2.5) in Cohort B. Most adverse events were mild or moderate; anemia and fatigue were most common. The mean change in MPN-SAF-TSS at week 37 was -6.21 points (95%CI: -10.56, -1.85, p=0.0052) in Cohort A; -2.71 points in Cohort B (95% CI: -8.33, 2.91, p=0.34). Sapablursen reduced phlebotomy need, improved symptoms, and was safe and well-tolerated in patients with polycythemia vera. (Funded by Ionis Pharmaceuticals; ClinicalTrials.gov: NCT05143957).
Details
- Title
- Sapablursen for Erythrocytosis Control in Patients with Polycythemia Vera
- Authors/Creators
- Jeanne M Palmer - Mayo ClinicJonathan How - Royal Victoria HospitalPrithviraj Bose - The University of Texas MD Anderson Cancer CenterPankit Vachhani - University of Alabama at BirminghamAdam J Mead - MRC Weatherall Institute of Molecular MedicineJonathan Sillar - Calvary Mater Newcastle HospitalAaron T Gerds - Cleveland ClinicAngela G Fleischman - University of California, IrvineCaitlin M O'Neill - Keck School of Medicine, University of Southern California, Los Angeles, California, United StatesRoss Ian Baker - Perth Blood Institute, PERTH, AustraliaRonald Hoffman - Mount Sinai Medical CenterShiangtung Jung - Ionis Pharmaceuticals (United States)Jon L Ruckle - Pacific Lutheran UniversityKeyvan Yousefi - Ionis Pharmaceuticals (United States)Sanjay Bhanot - Ionis Pharmaceuticals (United States)Terrance D Barrett - Ionis Pharmaceuticals (United States)Ruben A Mesa - Atrium Health Wake Forest Baptist
- Publication Details
- Blood
- Publisher
- American Society of Hematology
- Identifiers
- 991005912898607891
- Copyright
- © 2026 American Society of Hematology
- Murdoch Affiliation
- Centre for Molecular Medicine and Innovative Therapeutics
- Language
- English
- Resource Type
- Journal article
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