La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced that it has initiated its Phase 2b clinical trial of GCS-100 for the treatment of advanced chronic kidney disease (CKD) caused by diabetes. CKD is a condition characterized by a gradual loss of kidney function over time that may eventually lead to kidney failure, requiring dialysis or a kidney transplant to maintain life.

The Phase 2b clinical trial is a double-blind, multi-center, placebo-controlled, randomized trial of GCS-100 in diabetic patients with Stage 3b or 4 CKD. The clinical trial is designed to enroll approximately 375 patients. Patients will be randomized 1:1:1:1 to receive fixed doses of GCS-100 (1, 3 or 9 mg) or placebo. Randomized patients will receive their assigned treatment via IV injection once a week for 8 weeks and then once every other week for an additional 16 weeks.

The primary endpoint of this Phase 2b clinical trial is to compare the change in kidney function, as measured by estimated glomerular filtration rate (eGFR), from baseline to week 26, which is 2 weeks after the last injection, between patients receiving GCS-100 or placebo. Secondary efficacy endpoints include a responder analysis based on pre-specified percentage changes in eGFR and an analysis on progression to renal replacement therapy. Other secondary endpoints are focused on the long-term safety and tolerability of GCS-100, including an evaluation of the incidence of major cardiac events.

“Chronic kidney disease is an enormous and growing medical problem worldwide. A disease-modifying agent that could slow and potentially reverse the tissue fibrosis that is a hallmark of this disease would be a welcome advance in the field and could have a major impact on patients’ lives,” stated the trial’s principal investigator, Pablo E. Pergola, M.D., Ph.D., Director of the Clinical Advancement Center subsidiary of Renal Associates P.A. and Clinical Associate Professor of Medicine at the University of Texas Health Science Center at San Antonio. “Our experience with GCS-100 in previous trials was very encouraging, and my patients tolerated the therapy well.”

“We are excited to be advancing GCS-100 into the next phase of development,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “This Phase 2b trial will build on the knowledge we gained from our recently completed Phase 2 trial, in which treatment with GCS-100 resulted in a statistically significant improvement in kidney function.”

About GCS-100

GCS-100, La Jolla’s first-in-class galectin-3 inhibitor, is a complex polysaccharide derived from pectin that binds to, and blocks the activity of, the pro-fibrotic mediator galectin-3. Over-expression of galectin-3 has been implicated in a number of human diseases characterized by progressive tissue fibrosis, such as chronic kidney disease (CKD). La Jolla recently completed a multicenter, randomized, placebo-controlled, Phase 2 clinical trial in advanced CKD patients, in which treatment with GCS-100 resulted in a statistically significant improvement in kidney function compared to placebo.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has four product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension and hepatorenal syndrome. GCS-100 is La Jolla’s first-in-class galectin-3 inhibitor for the potential treatment of chronic kidney disease. LJPC-1010, La Jolla’s second-generation galectin-3 inhibitor, is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis and other diseases characterized by tissue fibrosis. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of conditions characterized by iron overload, such as hemochromatosis and beta thalassemia. For more information on La Jolla, please visit 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 the Company’s expectations regarding future events or its future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, any one of 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 future development of GCS-100 for the treatment of advanced CKD and the success of future development activities for this and other drug development programs sponsored by the Company and potential indications for which these drug candidates 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.

La Jolla Pharmaceutical CompanyGeorge F. Tidmarsh, M.D., Ph.D.President & Chief Executive Officer(858) 207-4264gtidmarsh@ljpc.comorChester S. Zygmont, IIISenior Director of Finance(858) 207-4262czygmont@ljpc.com

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