La Jolla Pharmaceutical Company Announces Data Presentation at the American Society of Nephrology Kidney Week

SAN DIEGO--()--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 an abstract of the results for the Company’s Phase 2 clinical trial of GCS-100 in chronic kidney disease (CKD) has been selected by the American Society of Nephrology’s (ASN) Program Committee for poster presentation at the Kidney Week Annual Meeting. Kidney Week will be held November 11-16, 2014 in Philadelphia, Pennsylvania.

“We are delighted that results from our Phase 2 study of GCS-100 in CKD have been selected by the ASN for presentation,” said George Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “More than 13,000 kidney professionals are expected to attend Kidney Week this year, and it is well established as the world’s premier nephrology meeting.”

La Jolla’s abstract, #3724 “Galectin-3 Inhibition with GCS-100 Improves eGFR in Patients with Chronic Kidney Disease, will be presented in a Poster Session titled Glomerular and Tubulointerstitial Disease: Clinical Trials and Outcomes, on Thursday, November 13, 2014 from 10:00am to 12:00pm Eastern Standard Time. The abstract is reprinted below:

Pablo E. Pergola, MD, PhD1, Geoffrey Block, MD2,Bhupinder Singh, MD3, George Fadda, MD4, Robert Cohen, DO3, William Durham, MD5, James Tumlin, MD6, George Tidmarsh, MD, PhD7, James Rolke7

1Clinical Advancement Center, San Antonio, TX
2Denver Nephrology, Denver, CO
3Southwest Kidney Institute, Tempe, AZ
4Balboa Nephrology Medical Group, La Mesa, CA
5Mountain Kidney and Hypertension Associates, Ashville, NC
6SouthEast Renal Research Institute, Chattanooga, TN
7La Jolla Pharmaceutical Company, San Diego, CA

Galectin-3 inhibition with GCS-100 Improves eGFR in Patients with Chronic Kidney Disease


Galectin-3 has been implicated in interstitial and glomerular fibrosis resulting in progressive loss of kidney function.


GCS-100, a polysaccharide inhibitor of galectin-3, was evaluated in CKD stages 3b/4 in a multicenter, randomized, placebo-controlled Phase 2 study. 121 consenting adults received placebo or GCS-100 at doses of 1.5 or 30 mg/m2 IV weekly for 8 weeks followed by a 4 week observation period. The primary endpoint was the change in eGFR from baseline to end of treatment versus placebo. Both T-test and ANCOVA were used for analyses.


Demographics: 117 patients (65 Stage 4) completed treatment (mean age 65; 43 female). Baseline eGFR was 29.2 ml/min/1.73m2 ± 9.00 (SD). Diabetes and HTN were the most common etiologies of CKD.

Efficacy: In the 1.5 mg/m2 group, a change in eGFR of +1.26 ±0.77 versus placebo (-0.58±0.46) was observed (p=0.045). When restricted to diabetic etiology, for 1.5 mg/m2 the change in eGFR was 2.33 ± 1.13 versus placebo (-0.53±0.56; p=0.028). Galectin-3 and K+ were significantly reduced (p=0.07 each). Uric acid and BUN were significantly reduced vs. baseline (p=0.07, 0.08 respectively). No significant changes in eGFR, galectin-3, K+, uric acid or BUN were observed among those receiving 30 mg/m2.

Safety: Four SAEs occurred, 2 in the placebo, 2 in the 30 mg/m2 group and none at 1.5 mg/m2. None of the SAEs were drug-related.


Short term therapy with the galectin-3 inhibitor, GCS-100 at 1.5 mg/m2, resulted in small but significant improvement in eGFR. If future, long-term studies confirm these findings, GCS-100 could be used as a disease-modifying agent to slow and potentially reverse the renal fibrosis common in CKD.

About the American Society of Nephrology and Kidney Week

The American Society of Nephrology (ASN) leads the fight against kidney disease by educating health professionals, sharing new knowledge, advancing research and advocating the highest quality care for patients. Kidney Week is the world's premier nephrology meeting, providing participants exciting and challenging opportunities to exchange knowledge, learn the latest scientific and medical advances, and listen to engaging and provocative discussions with leading experts in the field.

About GCS-100

GCS-100 is a complex polysaccharide derived from pectin that binds to, and blocks the activity of, galectin-3. Over-expression of galectin-3 has been implicated in a number of human diseases, including chronic organ failure and cancer. La Jolla is developing GCS-100 for the treatment of chronic kidney disease (CKD). The Company recently completed a multicenter, randomized, placebo-controlled, Phase 2 study in CKD patients in which treatment with GCS-100 resulted in a statistically significant improvement in estimated glomerular filtration rate (eGFR) 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 a proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension (CRH) and hepatorenal syndrome (HRS). GCS-100 is a first-in-class inhibitor of galectin-3 for the potential treatment of chronic kidney disease (CKD). LJPC-1010 is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis (NASH). LJPC-401 is a novel formulation of the natural peptide hepcidin for the potential treatment of iron overload. For more information on La Jolla, please visit

Forward-Looking Statement Safe Harbor

This document and the abstract referred to herein contain “forward-looking statements,” as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to our expectations regarding future events or our 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 These risks include, but are not limited to, risks relating to the future development of LJPC-501 for the treatment of CRH and HRS, GCS-100 for the treatment of CKD, LJPC-1010 for the treatment of NASH, and LJPC-401 for the treatment of iron overload and the success of future development activities for these 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 Company
George F. Tidmarsh, M.D., Ph.D.
President & Chief Executive Officer
Phone: (858) 207-4264
Chester S. Zygmont, III
Senior Director of Finance
Phone: (858) 207-4262