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.
Cancer
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.
Summary
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 http://www.ljpc.com.
REFERENCES
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 http://www.sec.gov. 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.
Company Contact George F. Tidmarsh, M.D., Ph.D. President
& Chief Executive Officer La Jolla Pharmaceutical Company
Phone: (858) 646-6682 Email: GTidmarsh@ljpc.com Media
Contact: Michael Rice LifeSci Advisors, LLC Phone: (646)
597-6979 Email: mrice@lifesciadvisors.com
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