La Jolla Pharmaceutical Company Announces the Start of a Phase 2 Extension Study of GCS-100 in Chronic Kidney Disease
January 15 2014 - 8:00AM
Marketwired
La Jolla Pharmaceutical Company Announces the Start of a Phase 2
Extension Study of GCS-100 in Chronic Kidney Disease
SAN DIEGO, CA--(Marketwired - Jan 15, 2014) - La Jolla
Pharmaceutical Company (OTCBB: LJPC) (the "Company" or "La Jolla"),
a leader in the development of therapeutics targeting significant
unmet life-threatening diseases, today announced that it has
started a Phase 2 Extension study of GCS-100 in chronic kidney
disease ("CKD"). The extension study was initiated at the
request of our lead investigators, to study long-term safety and
efficacy of GCS-100 in patients who completed the initial 12-week
Phase 2 study.
"We are delighted to have this opportunity to evaluate extended
dosing of GCS-100 in our patients who completed the initial study,"
said Pablo Pergola, M.D. PhD, Research Director at Renal
Associates, PA in San Antonio.
"We are excited about the possible benefit GCS-100 may provide
patients with chronic kidney disease," said Geoffrey Block, M.D.,
Director of Clinical Research at Denver Nephrology.
The Phase 2 Extension study (GCS-100-CS-4003) will enroll CKD
patients who completed the 12-week Phase 2 study (GCS-100-CS-4002),
including a maximum of 115 patients at 5 sites. Subjects will
be randomly assigned 1:1 to treatment with 1.5 mg/m2 GCS-100 or 30
mg/m2 GCS-100. All doses of study drug will be administered
intravenously once weekly for up to 8 consecutive weeks with a 4
week follow-up period and then at least once a month for a total of
1 year.
The primary objective of the Phase 2 Extension study is to
determine the safety and tolerability of extended dosing with
GCS-100. The secondary objective will be to determine the
efficacy as measured by eGFR of GCS-100 administered for 8 weeks
relative to the effect of 8 weeks of treatment for the same patient
on the GCS-100-CS-4002 study including those who were on placebo.
This study also provides the opportunity to compare crossover data
for individual patients from the initial Phase 2 study and this
extension study.
The initial 12 week Phase 2 study (GCS-100-CS-4002) enrolled 121
subjects, at least 18 years of age, with Stage 3b or 4 CKD across 6
sites. In this study, subjects were randomly assigned 1:1:1 to
treatment with placebo, 1.5 mg/m2 GCS-100 or 30 mg/m2 GCS-100.
Randomization was stratified into two groups according to baseline
renal function as defined by eGFR values of 15-29 and 30-44
mL/min/1.73m2, respectively. Once randomized, subjects receive
eight consecutive weekly doses of their assigned treatment with a
follow-up four weeks after treatment. Initial data from this study
is expected to be available in March 2014.
About GCS-100
GCS-100 is a complex polysaccharide that has the ability to bind
to and block the effects of galectin-3. Galectin-3 is a
soluble protein, over-expression of which has been implicated in a
number of human diseases including cancer and chronic organ
failure. The unique ability of GCS-100 to bind and sequester
galectin-3 makes it an ideal candidate to prevent and treat
diseases in which galectin-3 plays an important role.
About Chronic Kidney Disease
Chronic kidney disease ("CKD") currently affects 14% of
Americans or approximately 49 million people. The United
States Renal Data System, 2012 Annual Data Report, states that in
2010, costs for CKD reached $41 billion for Medicare
alone. Overall per person per year costs for CKD were
estimated at $22,323 for Medicare patients of age 65 and older and
$13,395 for patients of age 50-64. Patients with CKD may
progress to end-stage renal disease ("ESRD"). According to the
National Institute of Diabetes and Digestive and Kidney Diseases as
of 2008, there were 547,982 individuals in the US under treatment
for ESRD and 88,630 deaths per year from ESRD.
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. 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 and LJPC-501, the
success and timing of future preclinical and clinical studies of
these compounds, and potential indications for which GCS-100 and
LJPC-501 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) 207-4264 Email: GTidmarsh@ljpc.com and
Chester S. Zygmont, III Director of Finance La Jolla
Pharmaceutical Company Phone: (858) 207-4262 Email:
czygmont@ljpc.com
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