All content on this site is intended for healthcare professionals only. By acknowledging this message and accessing the information on this website you are confirming that you are a Healthcare Professional. If you are a patient or carer, please visit the MPN Advocates Network.
Bookmark this article
High iron levels are a known complicator of survival and overall quality of life for patients with PV. On September 20, 2023, the United States Food and Drug Administration granted fast-track designation to MWTX-003, an investigational anti-transmembrane serine protease 6 (TMPRSS6) monoclonal antibody designed to increase hepcidin levels and, thereby, reduce serum iron levels in patients with polycythemia vera (PV).
Preclinical data have highlighted strong pharmacodynamic effects, with a single dose resulting in a 70% reduction in serum iron levels for 21 days. Moreover, a related β-thalassemia clinical model demonstrated a 4-fold increase in hepcidin levels, a 60–65% reduction in serum and liver iron, increased red blood cell production, and a decrease in spleen weight. A phase I proof-of-mechanism trial is to begin in the second half of 2023. Further plans for a phase 1b/2a proof-of-concept study evaluating the safety and pharmacokinetic profile of MWTX-003 in patients with PV are also in place.
Polycythemia vera and essential thrombocythemia: 2021 updates
In a recent publication in the American Journal of Hematology, Tefferi and Barbui provided a review of the 2021 updates on the diagnosis, risk‐stratification, and management of...
Coupling germline genetics to interferon-alpha responses in polycythemia vera treatment
Pegylated interferon alfa-2 (PEG-IFN) induces high response rates in many patients with polycythemia vera (PV), while some patients do not respond well. In order...
Subscribe to get the best content related to MPN delivered to your inbox