A Study of Sapablursen Evaluating the Safety and Efficacy in Participants With Polycythemia Vera (PV)
Key claim: This trial evaluates the safety and efficacy of sapablursen added to standard of care in polycythemia vera patients across blinded, open-label, and long-term extension phases over up to 156 weeks.
Abstract
The purpose of this study is to evaluate the efficacy and safety of sapablursen when added on to current standard of care (SOC) for Polycythemia Vera (PV) therapy. The study will be conducted in three sequential parts (Part 1a blinded treatment, Part 1b open-label treatment, & Part 2 long-term extension). Participants may receive treatment for up to 156 weeks.
Why this matters
Sapablursen represents a PV-specific therapeutic strategy that exploits the disease’s hallmark erythrocytosis by pharmacologically inducing iron-restricted erythropoiesis via TMPRSS6/hepcidin modulation, a mechanism with no analog in ET or PMF natural history. If successful, it could reshape PV-specific management by reducing phlebotomy dependence and offering a non-cytoreductive option, distinguishing PV’s treatment trajectory further from other MPN subtypes.