La Jolla Pharmaceutical Company Announces Final Dosing in Phase 2 Clinical Trial of GCS-100 in Chronic Kidney Disease
Study Protocol for Phase 2 Clinical Study and Extension Study Released on Website
SAN DIEGO--(BUSINESS WIRE)--La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the “Company” or “La Jolla”), a biotechnology company developing therapeutics targeting significant unmet, life-threatening diseases today announced that it has completed dosing of its Phase 2 clinical trial of GCS-100 for the treatment of chronic kidney disease (“CKD”) and has collected the final data for evaluation of the primary endpoint. The primary efficacy endpoint is the change in estimated glomerular filtration rate (“eGFR”) from baseline to the average at Day 50 and 57 in each GCS-100 dose group compared to placebo. The primary endpoint will be reached if the difference in eGFR between either GCS-100 dose group and placebo has a significance of less than 10% (p-value of <0.1).
The protocol for the Phase 2 study (GCS-100-CS-4002) and the ongoing Phase 2 extension study (GCS-100-CS-4003) have been posted on the company’s website www.ljpc.com.
“We are pleased to have completed the Phase 2 study dosing and want to thank the patients and investigators who participated,” said George Tidmarsh, MD, PhD, President and CEO at La Jolla. “We look forward to analyzing the data which we expect to release in March.”
Of the 121 subjects with Stage 3b or 4 CKD enrolled, 117 subjects completed the study with no discontinuation due to an adverse event. Subjects were randomly assigned 1:1:1 to treatment with placebo, 1.5 or 30 mg/m2 of GCS-100. In addition, randomization was stratified by baseline renal function, defined by eGFR values of 15-29 and 30-44 mL/min/1.73m2. Once randomized, subjects received eight consecutive weekly doses of their assigned treatment with a follow-up four weeks after treatment.
About Pectin and GCS-100
Pectins are high molecular weight, complex polysaccharides that can be obtained from multiple sources (e.g., citrus, apple), all of which share a common basic structure comprising a block co-polymer of 1,4-linked galacturonic acid and 1-2-linked rhamnose with side branches of either 1,4-linked β-D-galactose or 1,5-α-linked L-arabinose. In addition, some portion of the galacturonic acid backbone can be esterified or exist as the uronic acid salt.
GCS-100, the company’s lead product candidate, is a modified pectin and is currently being tested in a Phase 2 clinical trial and extension study in patients with chronic kidney disease.
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. LJPC-401, the Company’s third product candidate, is a natural peptide for the treatment of iron overload. 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, 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. 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.