La Jolla Pharmaceutical and The French National Institute of Health and Medical Research (INSERM) entered into an exclusive worldwide license agreement to develop hepcidin agonists for the treatment of chronic iron overload in patients who are refractory to or intolerant to chelators.

“We are very pleased to license our hepcidin technology to La Jolla, since we have been working on the portfolio around hepcidin and its different assets for a long period of time with different approaches,” said Augustin Godard, EVP Open Innovation Inserm Transfert.

“Iron overload is a significant complication in patients with transfusion-dependent anemia,” said George F. Tidmarsh, M.D., Ph.D., president and CEO of La Jolla. “The role of hepcidin in modulating iron levels presents a unique opportunity to help patients suffering from the damaging effects of iron overload.”

This year, La Jolla plans to start a single dose, Phase I exploratory study in patients with transfusion-dependent anemia and severe chronic iron overload who are intolerant to or have failed chelation therapy. According to the company, this proposed clinical study will be the first to test administration of the synthetically derived endogenous hormone hepcidin to treat chronic iron overload.

La Jolla said hepcidin regulates dietary iron absorption and tissue distribution. By suppressing iron release into plasma, hepcidin prevents iron accumulation in tissues, thereby reducing damage to organs, according to the company.

Animal studies have shown that increasing hepcidin via injection of synthetic hepcidin or by genetic induction results in reduced organ iron overload. These studies suggest that administration of hepcidin may be beneficial in reducing iron overload that results from hemolytic anemia, as well as that induced by chronic transfusion.

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