Phase 2a randomized study to evaluate sapablursen in patients with non-transfusion dependent β-thalassemia intermedia - PubMed
4 hours ago
- #sapablursen
- #hepcidin
- #thalassemia
- Sapablursen, an antisense oligonucleotide targeting TMPRSS6 to increase hepcidin, was evaluated in a Phase 2a study involving 29 patients with non-transfusion-dependent β-thalassemia intermedia (NTDT) over up to 2 years.
- The study found sapablursen to be generally safe and well-tolerated but did not achieve its primary or secondary endpoints of increasing hemoglobin or reducing liver iron content (LIC) in NTDT patients.
- Unlike earlier trials in healthy volunteers and polycythemia vera patients, sapablursen did not produce consistent, dose-dependent increases in serum hepcidin in NTDT patients, likely due to elevated baseline LIC and serum erythroferrone interfering with the TMPRSS6 pathway.