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La Jolla Pharmaceutical Company and INSERM, Inserm Transfert Announce License Agreement to Develop Hepcidin Agonists

SAN DIEGO, CA and PARIS, FRANCE--(Marketwired - Feb 10, 2014) - La Jolla Pharmaceutical Company ( NASDAQ : LJPC ) (the "Company" or "La Jolla") and The French National Institute of Health and Medical Research ("INSERM"), today announced an exclusive worldwide license agreement to develop hepcidin agonists. 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."

Iron overload is a significant complication in patients with transfusion-dependent anemia; it results from both the underlying medical condition and the need for chronic transfusions. Transfusions that are administered to correct the underlying anemia exacerbate the iron load. This accumulation of iron in tissues can cause functional and structural damage to the liver, heart, pancreas, and other organs.

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. Patients with hemolytic anemia suffer from a lack of sufficient hepcidin likely due to their increased demand for red blood cell production. As a result of this decrease in hepcidin, the delicate balance of dietary iron absorption is upset, circulating iron is increased, and this in turn leads to iron overload.

The iron-regulating effects of hepcidin make it an attractive option for the treatment of iron overload. Animal studies of iron overload 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 itself, as well as that induced by chronic transfusion.

La Jolla 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 Santé et de la Recherche Médicale) 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. INSERM is the only public research body in France entirely dedicated to human health. Its researchers are committed to studying all diseases, whether common or rare, through their research in the fields of biology, medicine and public health.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics for chronic organ failure and cancer. GCS-100, the Company's lead product candidate, is a first-in-class inhibitor of galectin-3, a novel molecular target implicated in chronic organ failure and cancer. LJPC-501, the Company's second product candidate, is a natural peptide for the treatment of hepatorenal syndrome. For more information on the Company please visit http://www.ljpc.com.

Forward Looking Statement Safe Harbor

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or our future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company's filings with the U.S. Securities and Exchange Commission ("SEC"), all of which are available free of charge on the SEC's web site http://www.sec.gov. These risks include, but are not limited to, risks relating to the development of GCS-100, LJPC-501 and LJPC-401, the success and timing of future preclinical and clinical studies of these compounds, and potential indications for which GCS-100, LJPC-501 and LJPC-401 may be developed. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company's reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.