La Jolla Pharmaceutical Announces Positive Pre-Clinical Data for Oral Galectin-3 Inhibitor in Nonalcoholic Steatohepatitis (NASH)

SAN DIEGO--La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the "Company" or "La Jolla"), a leader in the development of therapeutics targeting significant unmet life-threatening diseases, today announced positive pre-clinical data for LJPC-1010 in non-alcoholic steatohepatitis (NASH). LJPC-1010 is a derivative of GCS-100, a carbohydrate inhibitor of galectin-3, which has shown activity in chronic kidney disease in a Phase 2 trial.

In a pre-clinical study of liver fibrosis in the STAM model of NASH, LJPC-1010 showed a significant reduction (p<0.001) vs control vehicle in nonalcoholic fatty liver disease (NAFLD) activity score, a system of scoring the features of NAFLD. Importantly, LJPC-1010 had a significant impact on all three measures used to derive the NAFLD score, namely steatosis, lobular inflammation and hepatocyte ballooning indicating broad effects. In addition to NAFLD, animals in the LJPC-1010 group had decreased yellowing of the liver, decreased liver weight and decreased fibrosis compared to the control group. Like GCS-100, LJPC-1010 is a modified pectin, however, LJPC-1010, at 50 mg/kg showed significant activity in NASH compared to GCS-100 at 300 mg/kg (both given orally) which showed a trend toward improvement but did reach statistical significance. LJPC-1010 given orally was comparable to an equivalent dose of GCS-100 given intravenously.

"NASH is a common and often referred to as a 'silent' liver disease, as it resembles alcoholic liver disease, but occurs in people who drink little or no alcohol," said James Rolke, Vice President of Research and Development of La Jolla Pharmaceutical Company. "LJPC-1010 is a modified and purified form of GCS-100. We believe oral LJPC-1010 could be an effective treatment option for NASH, which currently has very few treatment options."

La Jolla Pharmaceutical Company intends to file an Investigational New Drug application and initiate a Phase 1/2 clinical trial in NASH in the first half of 2015.

About LJPC-1010

LJPC-1010 is a more potent and purified form of GCS-100, our lead compound with activity in chronic kidney disease. Like GCS-100 it is also a modified pectin that has the ability to bind to and block the effects of galectin-3. Galectin-3 is a soluble protein, over-expression of which has been implicated in a number of human diseases including chronic organ failure and cancer. The unique ability of LJPC-1010 to bind and sequester galectin-3 makes it an ideal candidate to prevent and treat diseases in which galectin-3 plays an important role.

About NASH

NAFLD is a term used to describe the accumulation of fat in the liver of people who drink little or no alcohol. NAFLD is common and, for most people, causes no signs and symptoms and no complications. But in some people with NAFLD, the fat that accumulates can cause inflammation and scarring in the liver. This more serious form of NAFLD is sometimes called NASH. At its most severe, NAFLD can progress to liver failure. NASH affects 2 to 5 percent of Americans. An additional 10 to 20 percent of Americans have fat in their liver, but no inflammation or liver damage, a condition called "fatty liver."

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics for chronic organ failure and cancer. GCS-100, the Company's lead product candidate, is a first-in-class inhibitor of galectin-3, a novel molecular target implicated in chronic organ failure and cancer. GCS-100 currently is in Phase 2 clinical development for chronic kidney disease. LJPC-501, the Company's second product candidate, is a natural peptide for the potential treatment of hepatorenal syndrome. LJPC-401, the Company's third product candidate, is a natural peptide for the potential treatment of iron overload. For more information on the Company, please visit

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 our 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 These risks include, but are not limited to: risks relating to the development of GCS-100, LJPC-501 and LJPC-401; the success and timing of future preclinical and clinical studies of these compounds, and potential indications for which GCS-100, LJPC-501 and LJPC-401 may be developed; the ability of the Company to secure the necessary intellectual property rights to commercialize its product candidates, if successfully developed; and the ability to successfully obtain Orphan Drug status for LJPC-501. 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.



La Jolla Pharmaceutical Company
George F. Tidmarsh, M.D., Ph.D.
President & Chief Executive Officer
[email protected]
Chester S. Zygmont, III
Director of Finance
[email protected]