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EHA 2022 | Phase IIa study of vamifeport in patients with non-transfusion-dependent beta-thalassemia

Ali Taher, MD, PhD, American University of Beirut Medical Center, Beirut, Lebanon, outlines the mechanism of action of vamifeport and comments on the results of a Phase IIa study evaluating the safety, tolerability, and preliminary efficacy of vamifeport in patients with non-transfusion-dependent beta-thalassemia (NTDT; NCT04364269). The study achieved its primary and secondary endpoints, reporting a favorable tolerability profile and promising target engagement on serum iron and transferrin saturation levels versus placebo in this patient population. This interview took place at the European Hematology Association (EHA) Congress 2022 held in Vienna, Austria.

Disclosures

Novartis Pharmaceuticals: Consultancy, Research funding; Bristol-Myers Squibb (Celgene): Consultancy, Research funding; Ionis Pharmaceuticals: Consultancy, Research Funding; Vifor: Consultancy, Research Funding; Imara: Consultancy, Research Funding; Agios: Consultancy