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La Jolla Pharmaceutical Company and INSERM Ink License Deal to Develop Hepcidin Agonists

February 13, 2014

La Jolla Pharmaceutical Company and The French National Institute of Health and Medical Research (INSERM), announced an exclusive worldwide license agreement to develop hepcidin agonists.

According to a release, Hepcidin is the body's master regulator of iron, and patients with insufficient hepcidin levels suffer from chronic iron overload, which can lead to organ and tissue damage. The Company plans to develop hepcidin agonists for the treatment of chronic iron overload in patients who are refractory to or intolerant to chelators.

"Iron overload is a significant complication in patients with transfusion-dependent anemia," said George F. Tidmarsh, MD, PhD, 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. We are excited to license the hepcidin technology, and currently anticipate starting a Phase 1 study in 2014."

"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. "Current treatment options for iron overload are not covering all patient needs, and hepcidin agonists offer the promise of more successful treatment intervention in some patient populations. La Jolla has proven expertise in kidney disease and chronic organ failure, and we believe they are an ideal partner to move hepcidin forward in the clinic with the ultimate goal of developing a commercial product with a different approach. It also shows that our strategy and belief in the potential of hepcidin over the years has proven to be a right strategy."

La Jolla said it plans to start a single dose, Phase 1 exploratory study in patients with transfusion-dependent anemia and severe chronic iron overload who are intolerant to or have failed chelation therapy. This proposed clinical study will be the first to test administration of the synthetically-derived endogenous hormone hepcidin to treat chronic iron overload.

INSERM (Institut National de la Sante et de la Recherche Medicale) is a public institution with a scientific and technical vocation, under the dual auspices of the French Ministry of Health and the Ministry of Higher Education and Research.

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of therapeutics for chronic organ failure and cancer.

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