[June 04, 2014]

La Jolla Pharmaceutical Company Announces Completion of Enrollment of Phase 2 Extension Study of GCS-100 in Chronic Kidney Disease

La Jolla Pharmaceutical Company (Nasdaq: LJPC), a leader in the development of therapeutics targeting significant unmet life-threatening diseases, today announced that it has completed enrollment of GCS (News - Alert)-100-CS-4003 ("4003 study"), its Phase 2 Extension study of GCS-100 in chronic kidney disease ("CKD"). The 4003 study was initiated to study the long-term safety and efficacy of GCS-100 in patients who completed the initial 12-week Phase 2 study (GCS-100-CS-4002). A total of 93 patients enrolled across 5 sites. Initial data from the 4003 Extension study is expected late this year.

"We are pleased to have completed enrollment and encouraged by the greater than 80% participation of eligible patients," said George Tidmarsh, MD, PhD, Chief Executive Officer of La Jolla Pharmaceutical Company. "Chronic kidney disease affects nearly 50 million people in the United States. We believe GCS-100 can be an effective treatment option for those suffering from CKD."

"We have been impressed with how many patients were willing to continue participation in the extension study and how well they continue to tolerate GCS-100," said Pablo Pergola, MD, PhD, Renal Associates PA, San Antonio, TX and Clinical Associate Professor of Medicine, University of Texas Health Science Center at San Antonio. "We look forward to seeing if patients on this ongoing trial experience similar or greater improvement in kidney function with longer term treatment."

About the GCS-100 Phase 2 CKD Studies

The 4003 extension study was designed to enroll CKD patients who completed the 4002 study. Subjects were randomized in a 1:1 ratio to either treatment with 1.5 mg/m² GCS-100 or 30 mg/m² GCS-100. All doses of study drug will be administered intravenously once weekly for up to 8 consecutive weeks with a 4-week follow-up period and then once a month for a total of 1 year.

The primary objective of the Phase 2 Extension study is to determine the safety and tolerability of extended dosing with GCS-100. The secondary objective is to determine the efficacy as measured by eGFR of GCS-100 administered for -weeks relative to the effect of 8-weeks of treatment for the same patient on the GCS-100-CS-4002 study including those who were on placebo. This study also provides the opportunity to compare crossover data for individual patients from the initial Phase 2 study and this extension study.

The 4002 study was a blinded, multi-center, randomized clinical trial that enrolled 121 subjects, at least 18 years of age, with Stage 3b or 4 CKD across 6 sites. In this study, subjects were randomly assigned 1:1:1 to treatment with placebo, 1.5 mg/m² GCS-100 or 30 mg/m² GCS-100. Patients received the assigned treatment weekly for eight weeks followed by a four-week follow-up period. According to the pre-defined protocol and statistical analysis plan, the primary endpoint was reached if the change in eGFR from baseline to the end of treatment in either GCS-100 dose group compared to placebo had a significance of less than 10% (p value of <0.10). The t-test was the primary statistical test used for comparison between groups unless a significant covariate was found and then the ANCOVA test was employed.

In March 2014 the Company announced that the trial met its primary efficacy endpoint of a statistically significant improvement in kidney function. Specifically, a dose of 1.5 mg/m² led to a statistically significant (p=0.045) increase in estimated glomerular filtration rate (eGFR) compared to placebo between baseline and end of treatment. Key secondary endpoints were also met, and the effect on circulating galectin-3 levels was consistent with the effect on eGFR. For the 1.5 mg/m² dose, there was a statistically significant (p=0.067) reduction in circulating levels of galectin-3, while there was no significant difference at the 30 mg/m² dose level. GCS-100 was well-tolerated. Out of 121 patients enrolled, 117 completed treatment, including all 41 patients treated at the 1.5 mg/m² dose. There were no serious adverse events (SAEs) in the 1.5 mg/m² dose group compared to two in the placebo group and two in the 30 mg/m² group. All SAEs were deemed by the investigators as not drug-related.

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. LJPC-501, the Company's second product candidate, is a natural peptide for the treatment of hepatorenal syndrome. 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 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 development of GCS-100 and LJPC-501, the success and timing of future preclinical and clinical studies of these compounds, 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.

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