SOURCE: La Jolla Pharmaceutical Company

April 16, 2012 08:30 ET

La Jolla Pharmaceutical Company Announces Development Programs for Lead Product, GCS-100

GCS-100 Represents a First-in-Class Inhibitor of Galectin-3

Initial Programs to Focus on Chronic Kidney Disease and Cancer

SAN DIEGO, CA--(Marketwire - Apr 16, 2012) - La Jolla Pharmaceutical Company (OTCQB: LJPC) (PINKSHEETS: LJPC) today announced initial product development plans for advancing GCS-100, its first-in-class inhibitor of galectin-3.

On January 20, 2012, we announced the acquisition of GCS-100 from Solana Therapeutics, Inc. This acquisition makes us a leader in the development of therapeutics that target galectin-3, a protein that has been shown to play an important role in chronic organ failure and cancer. The Company believes that GCS-100 is the most advanced galectin-3 inhibitor in development and plans to initially focus its development activities in two areas of great unmet medical need: chronic kidney disease and cancer.

Chronic Kidney Disease: A Significant Unmet Medical Need

The developed world is suffering an epidemic of diabetes and hypertension, both of which cause kidney damage, chronic kidney disease and end-stage renal disease (ESRD). The National Institute of Diabetes and Digestive and Kidney Diseases estimates that 20 million United States adults suffered chronic kidney disease in 2008 [REF 1]. Of these 20 million people, 547,982 received treatment for ESRD and an estimated 88,630 died of ESRD. In total, a staggering $40 billion was spent in the United States on ESRD in 2008. ESRD leads to the need for dialysis and kidney transplantation. In 2008, 16,557 patients in the United States received a kidney transplant. Of these, 10,551 received a kidney from a non-living donor (cadaveric transplant). These transplants are at much higher risk of being rejected due to an attack by the patient's immune system. Despite improvements in therapies designed to suppress rejection by the recipient's immune system, 10% of patients receiving cadaveric transplants suffer acute rejection episodes which increases the risk of ultimate graft failure. In addition, the immunosuppressive therapies given to these patients increase the risk of infection and cancer due to sustained immune suppression.

The Role of Galectin-3 in Chronic Kidney Disease

Chronic organ failure involving the kidney, heart, liver or other organs is caused by fibrosis, or scar formation. Galectin-3 is normally found in most tissues at low concentration, but is up-regulated (increased) in response to injury. Several recent studies conducted by leading investigators have shown that increased circulating levels of galectin-3 are associated with poorer outcomes in patients with heart and kidney failure [REF 2, 3]. Furthermore, a number of preclinical studies have demonstrated a direct, causal role of galectin-3 in tissue fibrosis leading to kidney failure. Specifically, animals that have been genetically engineered to lack galectin-3 do not produce harmful scar formation after kidney injury or transplantation and have better kidney function. [REF 4, 5, 6].

GCS-100 for Chronic Kidney Disease

By inhibiting galectin-3, GCS-100 has the potential to delay or even reverse organ failure by preventing tissue fibrosis. GCS-100 has already been studied in more than 100 patients and has demonstrated an excellent side effect profile with mild-to-moderate, self-limiting rash as the principal side effect observed in clinical trials to date. We plan to initiate a clinical trial this year to study GCS-100 in cancer patients with renal (kidney) insufficiency. We will be evaluating several end points related to renal function, as well as looking at the effect of GCS-100 on plasma levels of galectin-3 and their relation to renal function.


The American Cancer Society estimates that over 1.6 million new cases of cancer will be diagnosed in the United States in 2012 [REF 7]. The lifetime chance of developing cancer is 1:2 for men and 1:3 for women. Cancer strikes all ages, races and segments of our society. Despite significant advances in recent years, cancer is the second leading cause of death in the United States, responsible for almost 600,000 deaths annually. Additionally, traditional therapies for cancer do not specifically target the cancer cells and therefore cause significant debilitating side effects. In an effort to specifically target the cancer cells, biologists and drug developers have long sought to harness the patient's own immune system for the treatment of cancer. In recent years, progress has been made on this goal with the successful development and approval of Yervoy™ (ipilimumab) for advanced-stage melanoma and Provenge™ (sipuleucel-T) for the treatment of advanced-stage prostate cancer. These therapies work by stimulating or assisting the body's active immune response to fight the tumor. Active immune therapies cause the patient's immune system to adapt to the ever-present changes in the tumor. In contrast, passive monoclonal antibody therapies do not stimulate the patient's own immune defenses to combat the cancer and therefore mutating cancer cells can often evade these therapies. Immunotherapy approaches hold particular promise to treat the cancer while sparing normal tissues.

The Role of Galectin-3 in the Body's Immune Response to Cancer

A number of studies have suggested that galectin-3 impairs the active immune system's attack on cancer [REF 8, 9]. Specifically, these publications have shown that cancer cells secrete high levels of galectin-3, which serves to deactivate the body's immune response to the cancer cell.

GCS-100 for Cancer

By antagonizing galectin-3, GCS-100 disrupts this mechanism of immune evasion and has the potential to activate the patient's immune system to fight cancer. Preclinical studies of GCS-100 have shown that GCS-100 can reactivate killer T-cells directed at the cancer that have been silenced by galectin-3 [REF 9]. Because GCS-100's mechanism of immune activation is complementary to those of approved cancer immunotherapies Yervoy and Provenge, there is the potential that GCS-100 will work in synergy with them and similar agents. GCS-100 has been studied in over 100 cancer patient and has demonstrated activity in chronic lymphocytic leukemia (CLL), multiple myeloma (MM) and renal cell cancer (RCC). We believe that the safety profile of GCS-100 to date has been excellent, with mild-to-moderate, self-limiting rash as the principal side effect.

We plan on evaluating GCS-100 in combination with Yervoy in preclinical models. This work builds on previous studies in which GCS-100 was shown to be additive or synergistic with several chemotherapeutic and targeted anti-cancer agents. Following successful completion of this study, we plan on initiating a clinical trial in melanoma.


We are dedicated to developing safe and effective therapies for significant life-threatening diseases including organ failure and cancer. We are attacking these fatal disorders by targeting galectin-3, a member of the galectin family of proteins that is implicated in their pathology. Our lead product candidate against galectin-3, GCS-100, has been tested in more than 100 patients and is generally well tolerated. We have an experienced management team dedicated to accomplishing our corporate milestones, as well as sufficient cash to execute on these near-term activities. Please visit us at


1. Centers for Disease Control and Prevention (CDC). National Chronic Kidney Disease Fact Sheet: General Information and National Estimates on Chronic Kidney Disease in the United States, 2010. Atlanta, GA: U.S. Department of Health and Human Services (HHS), CDC, 2010.

2. Predictive value of plasma galectin-3 levels in heart failure with reduced and preserved ejection fraction. Annals of Medicine, 2011; 43: 60-68.

3. Galectin-3 and Outcomes in Patients with End-Stage Renal Disease: Data from the German Diabetes and Dialysis Study, presented by Rudolf de Boer, MD, PhD, Associate Professor of Cardiology at the University of Groningen, the Netherlands; American Heart Association Scientific Presentation, November 2011.

4. Galectin-3 Expression and Secretion Links Macrophages to the Promotion of Renal Fibrosis. The American Journal of Pathology, 2008; Vol. 172, No. 2: 288-298.

5. Tubular Atrophy and Interstitial Fibrosis After Renal Transplantation Is Dependent on Galectin-3. Transplantation, 2012; Vol. 93, No. 5:477-484.

6. A Role for galectin-3 in renal tissue damage triggered by ischemia and reperfusion injury. Transplantation International, 2008; Vol. 21, No. 10: 999-1007.

7. American Cancer Society. Cancer Facts & Figures 2012. Atlanta: American Cancer Society; 2012.

8. Restoring the association of the T cell receptor with CD8 reverses anergy in human tumor-infiltrating lymphocytes. Immunity, 2008; 28:414-424.

9. A Galectin-3 Ligand Corrects the Impaired Function of Human CD4 and CD8 Tumor-Infiltrating Lymphocytes and Favors Tumor Rejection in Mice. Cancer Res 2010; 70:7476-7488.

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.

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 at These risks include, but are not limited to, risks relating to the development of GCS-100, the success and timing of future preclinical and clinical studies of this compound, and potential indications for which GCS-100 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.

Contact Information

  • Company Contact
    George F. Tidmarsh, M.D., Ph.D.
    President & Chief Executive Officer
    La Jolla Pharmaceutical Company
    Phone: (858) 646-6682

    Media Contact:
    Michael Rice
    LifeSci Advisors, LLC
    Phone: (646) 597-6979