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La Jolla Pharmaceutical Company Acquires Rights to Next-Generation Gentamicin Derivatives
[May 07, 2015]

La Jolla Pharmaceutical Company Acquires Rights to Next-Generation Gentamicin Derivatives


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 entered into an exclusive option agreement to acquire the Indiana University (News - Alert) Research and Technology Center's (IURTC) intellectual property rights covering next-generation gentamicin derivatives. Gentamicin has become one of the most commonly prescribed hospital antibiotics, despite causing kidney toxicity. Gentamicin consists of a mixture of distinct but closely related chemical entities that may contribute differentially to the product's toxicity profile. IURTC's technology covers the use of next-generation, parenteral gentamicin derivatives as antimicrobial agents with the potential for reduced toxicity.

La Jolla also entered into a second option agreement with IURTC and the University of Alabama at Birmingham (UAB) for the use of these next-generation compounds for the treatment of certain rare genetic diseases, such as cystic fibrosis and Duchenne muscular dystrophy. Gentamicin's ability to induce a lack of fidelity in gene transcription, intrinsic to its antimicrobial mechanism of action, can be leveraged in the correction of certain human genetic mutations that lead to rare genetic disorders. 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.

La Jolla has initially selected two lead development candidates from the technology, LJPC-30Sa and LJPC-30Sb, which are purified components of the currently marketed gentamicin product. LJPC-30Sa and LJPC-30Sb 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 in serious bacterial infections and rare genetic disorders characterized by stop codon mutations, 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, La Jolla has received guidance that it may proceed with its proposed Phase 1 clinical trial following the submission of an IND.

"We are very pleased to gain access to this intellectual property covering next-generation gentamicin derivatives," said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. "The use of aminoglycoside antibiotics has been limited primarily by treatment-related toxicity. We believe that our next-generation gentamicin derivatives may retain the activity of gentamicin, but improve the therapeutic window, thereby improving the outcome for patients requiring atimicrobial agents and potentially creating new opportunities for the treatment of rare genetic disorders."



Conference Call and Webcast

The Company will hold an investor conference call and webcast at 8:00 AM Eastern Time/5:00 AM Pacific Time on Friday, May 8, 2015. You can participate on the call by either dialing (877) 359-9508 pin: 44727023 or click here for the webcast.


About Gentamicin and Aminoglycoside Antibiotics

Gentamicin is an FDA-approved aminoglycoside antibiotic that is commonly used in infections in pregnancy and end-stage renal disease, urinary tract infections, endocarditis, serious Staphylococcus infections, and a wide range of other infections. Aminoglycosides are a broad-spectrum class of Gram-negative antibiotics that are a mainstay in the hospital setting. They are also used to potentiate the activity of certain classes of Gram-positive antibiotics. The use of the aminoglycoside class has increased over the last 10 years due to its broad spectrum activity against resistant bacteria, concentration-dependent killing, and prolonged duration of action, but its usage has been limited by dose-dependent and cumulative kidney toxicity and ototoxicity, which is toxicity to the ear. Current prescription data for aminoglycosides as antimicrobial agents represent over a $500 million market opportunity in the U.S., adjusted for branded pricing of comparable hospital antimicrobials.

A product manufactured through fermentation, gentamicin consists of a mixture of distinct but closely related chemical entities. While these distinct chemical entities appear to all retain antimicrobial activity, they may contribute differentially to toxicity. In particular, gentamicin's kidney toxicity appears to be associated with some but not all of its constituent chemical entities. A gentamicin derivative that retains antimicrobial activity but had reduced kidney toxicity would represent a major advance for patients.

Gentamicin's ability to induce a lack of fidelity in gene transcription, intrinsic to its antimicrobial mechanism of action, can 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 currently marketed gentamicin as a chronic treatment for these genetic diseases has been limited by its toxicity profile.

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 and hepatorenal syndrome. LJPC-401 is La Jolla's novel formulation of hepcidin for the potential treatment of conditions characterized by iron overload, such as 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 (News - Alert)), 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 (News - Alert)-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.


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