La Jolla Pharma And Vanderbilt University Enter Exclusive Research And License Agreement Covering Novel BMP Type-I Receptor Inhibitors

Novel Compounds Have Therapeutic Potential in Broad Range of Diseases, Including Certain Rare Genetic Disorders

SAN DIEGO & NASHVILLE, Tenn.--(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, and Vanderbilt University (Vanderbilt) today announced an exclusive, worldwide research and license agreement covering Vanderbilt’s research program and intellectual property rights relating to small-molecule kinase inhibitors designed to selectively block specific members of the bone morphogenetic protein (BMP) type-I receptor family.

“Tremendous credit goes to Dr. Hong for his discovery of BMP receptor inhibitors and Dr. Lindsley and Dr. Hopkins for their advancements in medicinal chemistry. Their breakthrough discoveries offer hope to improve the lives of patients suffering from a broad range of debilitating diseases”

The seven members of the BMP type-I receptor family, activin receptor-like kinase (ALK) 1-7, play critical roles in human development and physiology. In turn, the improper activation of these receptor pathways is responsible for a wide range of disease conditions. For example, fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder where the body turns muscle into bone, is caused by a genetic mutation in ALK2 that results in excessive signaling of this pathway.

Members of the BMP type-I receptor family also are involved in other diseases such as acquired heterotopic ossification (formation of bone in soft tissue caused by injury or trauma), muscular dystrophies including Duchenne muscular dystrophy, anemia of chronic disease (decrease of red blood cells or hemoglobin from chronic infection, chronic immune activation and malignancy), cancer, cardiovascular diseases and inflammatory bowel disease.

After the first publication of his discovery in 2008, Charles C. Hong, M.D., Ph.D., in conjunction with Vanderbilt, has led a dedicated research team focused on discovering highly selective, small-molecule kinase inhibitors designed to selectively block specific members of the BMP type-I receptor family. Dr. Hong's team is complemented by the robust medicinal chemistry expertise of Vanderbilt's Craig W. Lindsley, Ph.D., and Corey R. Hopkins, Ph.D. Under the research and license agreement with La Jolla, La Jolla will fund further research under this program at Vanderbilt in return for rights to acquire compounds emerging from this program.

Dr. Hong is an Associate Professor of Cardiovascular Medicine, Pharmacology, and Cell and Developmental Biology at Vanderbilt University School of Medicine and a member of the Veterans Affairs Tennessee Valley Healthcare System. He is also a member of the Vanderbilt Institute of Chemical Biology and the Vanderbilt Center for Stem Cell Biology. Dr. Hong discovered the first pharmacologic inhibitor of the BMP pathway in 2008, and he was instrumental in the discovery of the critical role of the BMP pathway in stem cell maturation, cholesterol homeostasis and cancer.

Dr. Lindsley is a Professor of Pharmacology and Chemistry at Vanderbilt University School of Medicine. He is also the Director of Medicinal Chemistry at the Vanderbilt Center for Neuroscience Drug Discovery and the Director of the Vanderbilt Specialized Chemistry Center for Accelerated Probe Development.

Dr. Hopkins is an Assistant Professor of Pharmacology and Chemistry at Vanderbilt University School of Medicine. He is also the Associate Director of Chemistry at the Vanderbilt Center for Neuroscience Drug Discovery and a co-Director of the Vanderbilt Specialized Chemistry Center for Accelerated Probe Development.

“We are excited to see our work move forward from a basic research program to a therapeutic program in partnership with La Jolla. Together, we see this as an opportunity to position our BMP inhibitors as potential treatments for rare and neglected diseases,” said Drs. Hopkins and Lindsley on behalf of the Vanderbilt team.

“Tremendous credit goes to Dr. Hong for his discovery of BMP receptor inhibitors and Dr. Lindsley and Dr. Hopkins for their advancements in medicinal chemistry. Their breakthrough discoveries offer hope to improve the lives of patients suffering from a broad range of debilitating diseases,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Office of La Jolla. “In collaboration with our innovative partners at Vanderbilt University Medical Center, we will work hard to translate their pioneering discoveries into novel treatments for patients in need.”

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.

About Vanderbilt University Medical Center

Vanderbilt University Medical Center is home to Vanderbilt University Hospital, the Monroe Carell Jr. Children’s Hospital at Vanderbilt, the Vanderbilt Psychiatric Hospital and the Vanderbilt Stallworth Rehabilitation Hospital. These hospitals experienced more than 61,000 inpatient admissions during fiscal year 2015. Vanderbilt’s adult and pediatric clinics treated nearly 2 million patients during this same period.

Both the Vanderbilt University School of Medicine and School of Nursing are recognized by U.S. News & World Report’s annual Best Graduate Schools as among the nation’s best, with the School of Medicine ranked 14th and the School of Nursing 11th. The School of Medicine’s biomedical research program has earned its place among the nation’s top 10 academic medical centers in terms of public and private research funding, receiving more than $500 million in total funding during fiscal year 2015.

Vanderbilt University Hospital and the Monroe Carell Jr. Children’s Hospital at Vanderbilt are recognized again this year by U.S. News & World Report’s Best Hospitals as among the nation’s best, with 18 nationally ranked specialties.

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, 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; and potential indications for which the Company’s drug candidates 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.