La Jolla Pharmaceutical Company Announces Start of Phase 2 Clinical Trial of GCS-100 for Chronic Kidney Disease

SAN DIEGO, CA--(Marketwired - July 08, 2013) - La Jolla Pharmaceutical Company (OTCBB: LJPC) ("La Jolla" and "Company"), a leader in the development of therapeutics targeting galectin proteins, announced today that the first patient was dosed in a randomized, single blind, Phase 2 clinical trial of GCS-100 for chronic kidney disease (CKD).

"Based on the favorable safety profile and encouraging Phase 1 results, we are pleased to continue the development of GCS-100 with the initiation of this Phase 2 trial in patients with CKD," said George Tidmarsh, MD, PhD, President and Chief Executive Officer of La Jolla. "The reduction in serum galectin-3 concentration we observed after a single dose of GCS-100 in the Phase 1 trial suggests an effect on a significant biomarker of disease and indicates that GCS-100 may have clinical activity. Millions of people in the world suffer from CKD without curative therapy and we hope to one day provide help to those in need."

This Phase 2 study is designed to enroll approximately 117 subjects, at least 18 years of age, with Stage 3b or 4 CKD. Subjects will be randomly assigned 1:1:1 to treatment with placebo, 1.5 mg/m2 GCS-100 or 30 mg/m2 GCS-100. In addition, randomization will be stratified into two groups according to baseline renal function as defined by eGFR values of 15-29 and 30-44 mL/min/1.73m2, respectively. Once randomized, subjects will receive eight consecutive weekly doses of their assigned treatment with a follow-up four weeks after treatment.

The primary objective of this study is to compare the change in eGFR from baseline to Week 8 between placebo and GCS-100 treatment. The secondary objective is to determine the safety and tolerability of GCS-100 administered for 8 weeks relative to placebo. In addition, the study will measure the effect of GCS-100 on circulating galectin-3 and other markers of disease activity.

La Jolla Pharmaceutical Company recently completed and announced the results of a Phase 1 study of GCS-100 in patients with Stage 3b and 4 CKD. The product candidate was found to be safe with no serious adverse events observed and no adverse changes in laboratory measures. Baseline serum galectin-3 levels inversely correlated with renal function as measured by eGFR, with higher levels of galectin-3 found in those patients with worse renal function (lower eGFR). This correlation strengthens the hypothesis that high levels of galectin-3 cause organ fibrosis resulting in reduced kidney function. A statistically significant post-dose reduction of serum galectin-3 was also observed at the higher doses of GCS-100. This reduction in serum galectin-3 was sustained until Day 7 when a rebound to levels above baseline was observed. This rebound may be due to release of galectin-3 from cellular receptors, including those found in kidney tissue.

About Chronic Kidney Disease

Chronic kidney disease ("CKD") currently affects 14% of Americans or approximately 49 million people. The United States Renal Data System, 2012 Annual Data Report, states that in 2010, costs for CKD reached $41 billion for Medicare alone. Overall per person per year costs for CKD were estimated at $22,323 for Medicare patients of age 65 and older and $13,395 for patients of age 50-64. Patients with CKD may progress to end-stage renal disease ("ESRD"). According to the National Institute of Diabetes and Digestive and Kidney Diseases as of 2008, there were 547,982 individuals in the US under treatment for ESRD and 88,630 deaths per year from ESRD.

About GCS-100

GCS-100 is a complex polysaccharide 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 cancer and chronic organ failure. The unique ability of GCS-100 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 La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company dedicated to the development of medical treatments that significantly improve outcomes in patients with life-threatening diseases. 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. For more information on the Company please visit http://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 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 from time to time 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 development of GCS-100 and LJPC-501, the success and timing of future preclinical and clinical studies of this compound, and potential indications for which GCS-100 and LJPC-501 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.