A Study to Investigate AG-236 in Subjects ≥18 Years of Age With Polycythemia Vera
Key claim: AG-236 is being evaluated in a Phase 2/3 placebo-controlled trial in adults with polycythemia vera to increase the proportion of patients free of phlebotomy and phlebotomy eligibility over 13 weeks, with dose-finding and open-label extension.
Abstract
This clinical trial is a Phase 2/3 study that will determine the recommended dose and dose regimen of AG-236 and evaluate the efficacy, safety, and pharmacokinetics of AG-236 in polycythemia vera (PV) by testing how well AG-236 works compared to placebo to increase the number of subjects with no phlebotomy and absence of phlebotomy eligibility over a 13-week period. In addition, the long-term effect of AG-236 on efficacy and safety will be explored in an open-label extension portion.
Why this matters
Phlebotomy-sparing control of hematocrit is a growing therapeutic axis in PV, with hepcidin mimetics (rusfertide) and TMPRSS6-targeted agents (sapablursen, DISC-3405) already in advanced trials. AG-236 adds another candidate to this landscape, and its Phase 2/3 placebo-controlled design with a 13-week phlebotomy-free endpoint will provide directly comparable efficacy data against competing agents. No outcomes are yet reportable.