La Jolla discontinued LJPC-401 (iron overload drug) in β-thalassaemia patients

La Jolla Pharmaceutical Company has announced plans to discontinue Study LJ401-BT01 due to mixed clinical results. LJPC-401 is a synthetic human hepcidin for the treatment of conditions characterized by iron overload. Study LJ401-BT01 (NCT03381833) is designed to evaluate the safety and efficacy of LJPC-401 as a treatment for iron overload in beta thalassemia patients who, despite chelation therapy, have cardiac iron levels above normal. A recent interim analysis involving half of the enrolled population suggested lack of efficacy, with patients on the treatment and control arms exhibiting similar changes in cardiac iron levels, the primary endpoint, as well as key secondary endpoints. For more information, read La Jolla News here.