La Jolla Pharmaceutical : Doses First Patient in Phase 1 Clinical Trial of LJPC-401 in Patients at Risk of Iron Overload

La Jolla Pharmaceutical Company Doses First Patient in Phase 1 Clinical Trial of LJPC-401 in Patients at Risk of Iron Overload

SAN DIEGO-(BUSINESS WIRE)- La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced that the first patient has been dosed in a Phase 1 clinical trial of LJPC-401, La Jolla's novel formulation of hepcidin.

The Phase 1 clinical trial is a multicenter, open-label, dose-escalation trial that is evaluating the safety, tolerability and effect on serum iron parameters, and pharmacokinetics of LJPC-401 in patients at risk of iron overload due to conditions such as hereditary hemochromatosis (HH), beta thalassemia and sickle cell disease (SCD). Preliminary results from this clinical trial are anticipated by the end of 2015.

LJPC-401 is La Jolla's novel formulation of hepcidin, a naturally occurring peptide hormone that is the body's regulator of iron absorption and distribution. Hepcidin prevents excessive iron accumulation in vital organs, such as the liver and heart, where it can cause significant damage and even result in death. La Jolla is developing LJPC-401 for the potential treatment of iron overload, which occurs as a result of diseases such as HH, beta thalassemia and SCD.

HH is a disease caused by a genetic deficiency in hepcidin production, resulting in excessive iron accumulation. HH is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, diabetes, arthritis and joint pain. Beta thalassemia and SCD are genetic diseases of blood cells that can cause life-threatening anemia and often require frequent and life-long blood transfusions. These blood transfusions, while necessary to treat anemia, cause excessive iron accumulation in the body, which is toxic to vital organs. In addition, the underlying anemia may cause excessive iron accumulation independent of the blood transfusions.

'We are excited to be advancing LJPC-401 into its first clinical trial, and we are very grateful to the physicians and patients who have partnered with us on this important program,' said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. 'We believe that LJPC-401 presents a unique opportunity to potentially help patients suffering from the effects of iron overload by restoring normal or near-normal levels of hepcidin, the body's natural regulator of iron absorption and distribution.'

About LJPC-401

LJPC-401 is La Jolla's novel formulation of hepcidin. Hepcidin, an endogenous peptide hormone, is the body's naturally occurring regulator of iron absorption and distribution. Hepcidin prevents excessive iron accumulation in vital organs, such as the liver and heart, where it can cause significant damage and even result in death.

La Jolla is developing LJPC-401 for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH), beta thalassemia and sickle cell disease (SCD). HH is a disease caused by a genetic deficiency in hepcidin that results in excessive iron accumulation. HH is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, diabetes, arthritis and joint pain. Beta thalassemia and SCD are genetic diseases of the blood that can cause life-threatening anemia and usually require frequent and life-long blood transfusions. These blood transfusions cause excessive iron accumulation in the body, which is toxic to vital organs, such as the liver and heart. In addition, the underlying anemia causes excessive iron accumulation independent of blood transfusions.

LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing. La Jolla has initiated a Phase 1 clinical trial of LJPC-401 in patients at risk of iron overload due to conditions such as HH, beta thalassemia and SCD. La Jolla anticipates preliminary results from this Phase 1 clinical trial by the end of 2015.

About Hereditary Hemochromatosis

Hereditary hemochromatosis (HH) is the most common genetic disease in Caucasians. HH is a disease characterized by a genetic mutation that results in a deficiency in the production of hepcidin, which is the body's naturally occurring regulator of iron absorption and distribution. Without normal levels of hepcidin, excessive amounts of iron accumulate in the body and can lead to liver cirrhosis, liver cancer, heart disease and/or failure, diabetes, arthritis and joint pain. With no FDA-approved treatments for HH, patients are treated with phlebotomy, which does not address the underlying disease pathology, carries significant toxicity and adversely impacts quality of life.

About Beta Thalassemia and Sickle Cell Disease

Beta thalassemia is a disease characterized by a genetic mutation that results in the underproduction of hemoglobin, the body's natural oxygen-carrying molecule contained in red blood cells. There are three types of beta thalassemia: beta thalassemia minor, beta thalassemia intermedia and beta thalassemia major. Patients with the more severe forms (intermedia and major) suffer from significant and sometimes life-threatening anemia, bone deformities and enlargement of the spleen.

Sickle cell disease (SCD) is the most common inherited blood disorder in the United States. SCD is characterized by a genetic mutation that results in the production of abnormal hemoglobin, the body's natural oxygen-carrying molecule contained in red blood cells. The abnormal hemoglobin causes the red blood cells to form a 'sickle,' or crescent, shape. Patients with severe forms suffer from significant and sometimes life-threatening anemia, strokes and damage to vital organs such as the lungs, spleen, kidney and liver.

Standard treatment for both beta thalassemia and SCD includes frequent, life-long blood transfusions. While lifesaving, these transfusions cause an excess of iron accumulation, which in turn is toxic to vital organs, such as the liver and heart. In addition, the underlying anemia causes excessive iron accumulation independent of blood transfusions. The only currently approved treatments for iron overload are iron chelators, which may cause kidney failure, liver failure or gastrointestinal hemorrhage.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla's proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla's novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH), beta thalassemia and sickle cell disease (SCD). LJPC-30Sa and LJPC-30Sb are La Jolla's next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit 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 the Company's 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 www.sec.gov. These risks include, but are not limited to, risks relating to: the timing for the filing of an Investigational New Drug Application (IND); commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for the Company's drug candidates; potential indications for which the Company's drug candidates may be developed; and the Company's ability to obtain the potential benefits of orphan drug designation for its drug candidates. 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.

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Contacts

La Jolla Pharmaceutical Company

George F. Tidmarsh, M.D., Ph.D.

President & Chief Executive Officer

(858) 207-4264

gtidmarsh@ljpc.com

or

Dennis M. Mulroy

Chief Financial Officer

(858) 433-6839

dmulroy@ljpc.com

Source: La Jolla Pharmaceutical Company