SAN DIEGO, CA -- (Marketwired) -- 05/29/13 -- La Jolla Pharmaceutical Company (OTCBB: LJPC) ("La Jolla" and "Company"), a leader in the development of therapeutics targeting galectin proteins, released data today from the Phase 1 (Part A) portion of the Phase 1/2 clinical trial of GCS-100 in patients with chronic kidney disease ("CKD"). Based on the positive results of this study, the Company expects to start a randomized, Phase 2a clinical trial next month.
The completed Phase 1 study was an open-label, multi-center dose escalation study of GCS-100 in patients with Stage 3b and 4 CKD. No serious adverse events were observed, and there were no adverse changes in laboratory measures. A small number of patients, 4 of the 29, experienced at least one adverse event, including mild hives and itching or muscle cramping. Although the maximum tolerated dose according to the protocol was not reached, moderate to severe cramping in one patient lead to the termination of the dose escalation.
Baseline serum galectin-3 levels inversely correlated with renal function as measured by estimated glomerular filtration rate ("eGFR"), with higher levels of galectin-3 found in those patients with worse renal function (lower eGFR). 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.
"The reduction in serum galectin-3 concentration after a single dose of GCS-100 is a good indication that we are successfully having an impact on the target biomarker, a molecule we know correlates with organ fibrosis," said George Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. "We are very pleased with the quick progress made enrolling the Phase 1 portion of the trial and believe this underscores the significant unmet medical need of CKD patients."
Based upon the favorable safety profile and encouraging galectin-3 results, the company plans to close this study and initiate a randomized Phase 2a study in early June. Details of this study will be presented shortly.
About The Phase 1/2 Clinical Study
Enrollment was open to patients at least 18 years of age with moderately severe to severe renal impairment and treatment comprised a single dose of GCS-100 from 0.5 to 30 mg/m2 with a 2 week follow-up in cohorts of 3-6 patients. The primary objective of the study was to evaluate the safety of GCS-100. Secondary study objectives included evaluating serum levels of galectin-3, renal function, and other markers of disease activity in CKD. The study consisted of 2 Parts; Phase 1 (Part A) and Phase 2 (Part B). The company plans to close this study before enrolling Part B and instead, start a Phase 2a randomized clinical trial in the same patient population.
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.
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.
George F. Tidmarsh, M.D., Ph.D.
President & Chief Executive Officer
La Jolla Pharmaceutical Company
Phone: (858) 207-4264
Chester S. Zygmont, III
Director of Finance
La Jolla Pharmaceutical Company
Phone: (858) 207-4262
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