SAN DIEGO, CA--(Marketwired - Jan 15, 2014) - La Jolla Pharmaceutical Company (OTCBB: LJPC) (the "Company" or "La Jolla"), a leader in the development of therapeutics targeting significant unmet life-threatening diseases, today announced that it has started a Phase 2 Extension study of GCS-100 in chronic kidney disease ("CKD"). The extension study was initiated at the request of our lead investigators, to study long-term safety and efficacy of GCS-100 in patients who completed the initial 12-week Phase 2 study.
"We are delighted to have this opportunity to evaluate extended dosing of GCS-100 in our patients who completed the initial study," said Pablo Pergola, M.D. PhD, Research Director at Renal Associates, PA in San Antonio.
"We are excited about the possible benefit GCS-100 may provide patients with chronic kidney disease," said Geoffrey Block, M.D., Director of Clinical Research at Denver Nephrology.
The Phase 2 Extension study (GCS-100-CS-4003) will enroll CKD patients who completed the 12-week Phase 2 study (GCS-100-CS-4002), including a maximum of 115 patients at 5 sites. Subjects will be randomly assigned 1:1 to 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 at least 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 will be to determine the efficacy as measured by eGFR of GCS-100 administered for 8 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 initial 12 week Phase 2 study (GCS-100-CS-4002) 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. Randomization was 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 receive eight consecutive weekly doses of their assigned treatment with a follow-up four weeks after treatment. Initial data from this study is expected to be available in March 2014.
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 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 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"), 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.