La Jolla Pharmaceutical Company Announces FDA Acceptance of IND for LJPC-401

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 U.S. Food and Drug Administration (FDA) has accepted the Company’s Investigational New Drug Application (IND) for LJPC-401, La Jolla’s novel formulation of hepcidin. La Jolla expects to release preliminary results from a Phase 1 clinical trial of LJPC-401 by the end of 2015.

Hepcidin is a naturally occurring regulator of iron absorption and distribution. By regulating the absorption and distribution of iron, hepcidin prevents excessive iron accumulation in tissues, 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 treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH) and beta thalassemia. 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, dementia and diabetes.

LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing. Specifically, La Jolla has completed animal toxicology studies that demonstrated a dose-dependent reduction in serum iron levels in all species tested.

“We are pleased to have received clearance from the FDA to begin a Phase 1 study of LJPC-401,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “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 is a naturally occurring regulator of iron absorption and distribution. By regulating the absorption and distribution of iron, hepcidin prevents excessive iron accumulation in tissues, 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 treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH) and beta thalassemia. 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, dementia and diabetes.

LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing. La Jolla’s Investigational New Drug Application (IND) has been accepted by the FDA, and La Jolla expects to release preliminary results from a Phase 1 study by the end of 2015.

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 and beta thalassemia. 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 LJPC-501, LJPC-401, LJPC-30Sa and LJPC-30Sb; potential indications for which LJPC-501, LJPC-401, LJPC-30Sa and LJPC-30Sb may be developed; and the expected duration over which the Company’s cash balances will fund its operations. 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.

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