La Jolla Pharmaceutical Company Announces Exclusive Worldwide License Agreements Covering LJPC-30Sa and LJPC-30Sb

SAN DIEGO--()--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 it has signed two exclusive worldwide license agreements for the intellectual property rights covering La Jolla’s next-generation gentamicin derivatives, LJPC-30Sa and LJPC-30Sb. The first license agreement is with the Indiana University Research and Technology Corporation (IURTC), and the second is with the IURTC and the University of Alabama at Birmingham (UAB). The license agreements, which follow from the previously announced option agreements, cover the use of LJPC-30Sa and LJPC-30Sb as antimicrobial agents and for the potential treatment of rare genetic disorders.

Despite kidney toxicity, gentamicin has become one of the most commonly prescribed hospital antibiotics due to its broad spectrum of antimicrobial efficacy. Gentamicin consists primarily of a mixture of four distinct but closely related chemical entities that may contribute differentially to the product’s toxicity profile. LJPC-30Sa and LJPC-30Sb are purified components of the currently marketed gentamicin product that retain the biologic activity of gentamicin, yet appear to lack the traditional kidney toxicity associated with it.

La Jolla plans to pursue a dual development strategy with its next-generation gentamicin derivative program. Specifically, La Jolla plans to develop LJPC-30Sa and LJPC-30Sb not only for the potential treatment of serious bacterial infections but also for the potential treatment of rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. Following a pre-Investigational New Drug Application (IND) meeting with the U.S. Food and Drug Administration (FDA), La Jolla has received guidance that it may proceed with a proposed Phase 1 clinical trial following the submission of an IND.

“We are delighted by the continued progress of our next-generation gentamicin derivative program with our colleagues at IURTC and UAB,” said George Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “Following a positive pre-IND meeting with the FDA, we look forward to further advancing this program with the filing of an IND followed by the commencement of a Phase 1 clinical trial.”

About LJPC-30Sa and LJPC-30Sb

LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives. Despite kidney toxicity, gentamicin has become one of the most commonly prescribed hospital antibiotics due to its broad spectrum of antimicrobial efficacy. Gentamicin consists primarily of a mixture of four distinct but closely related chemical entities that may contribute differentially to the product’s toxicity profile.

LJPC-30Sa and LJPC-30Sb are purified components of the currently marketed gentamicin product that retain the biologic activity of gentamicin, yet appear to lack the traditional kidney toxicity associated with it. La Jolla is developing LJPC-30Sa and LJPC-30Sb not only for the potential treatment of serious bacterial infections but also for the potential treatment of rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy.

La Jolla believes that gentamicin’s ability to induce a lack of fidelity in gene transcription, intrinsic to its antimicrobial mechanism of action, can also be leveraged in the correction of certain human genetic mutations that lead to rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. In spite of favorable short-term clinical proof-of-efficacy data in cystic fibrosis, development of gentamicin as a chronic treatment for these genetic diseases has been limited by its toxicity profile.

Following a pre-Investigational New Drug Application (IND) meeting with the U.S. Food and Drug Administration (FDA), La Jolla has received guidance that it may proceed with a proposed Phase 1 clinical trial following the submission of an IND.

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 conditions characterized by iron overload, 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 http://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 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; the potential market sizes for our product candidates in development; and any future success in out-licensing or partnering GCS-100 or LJPC-1010. 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
and
Dennis M. Mulroy
Chief Financial Officer
858-433-6839
dmulroy@ljpc.com

Release Summary

La Jolla Pharmaceutical Company Announces Exclusive Worldwide License Agreements Covering LJPC-30Sa and LJPC-30Sb.

Contacts

La Jolla Pharmaceutical Company
George F. Tidmarsh, M.D., Ph.D.
President & Chief Executive Officer
858-207-4264
gtidmarsh@ljpc.com
and
Dennis M. Mulroy
Chief Financial Officer
858-433-6839
dmulroy@ljpc.com