Primary Endpoint Met; Statistically Significant
Improvement in Kidney Function Observed
Improvement More Pronounced in Diabetic
Patients
Results Reproduced in Extension Study
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 detailed
results from its Phase 2 study of GCS-100 in chronic kidney disease
(CKD) patients are being presented at a poster session (Glomerular
and Tubulointerstitial Disease: Clinical Trials and Outcomes,
November 13, 10:00 a.m. - 12:00 p.m. Eastern Standard Time, Poster
TH-PO460) at the American Society of Nephrology’s (ASN) Kidney Week
Annual Meeting. GCS-100, which blocks the activity of the
pro-fibrotic mediator galectin-3, has the potential to be a
first-in-class disease-modifying agent for the treatment of
diseases characterized by progressive tissue fibrosis, such as
CKD.
This multicenter, randomized, blinded, placebo-controlled, Phase
2 study in 121 advanced CKD patients met its primary efficacy
endpoint of a statistically significant improvement in kidney
function. Specifically, GCS-100, at a dose of 1.5 mg/m2, led to a
statistically significant (p=0.045) improvement in estimated
glomerular filtration rate (eGFR) versus placebo after 8 weeks of
dosing. This improvement, compared to placebo, was maintained 5
weeks following the completion of dosing (p=0.07). No statistically
significant improvement in eGFR was observed in the 30 mg/m2 dose
group. The lack of consistent response in the 30 mg/m2 group may be
due to off-target drug effects, as this dose is 1,400-fold in
excess, on a molar basis, versus known circulating galectin-3
levels. Off-target effects may include antagonizing other galectins
like galectin-9, which has opposing biological effects to
galectin-3.
GCS-100 CKD Phase 2 Results – All Patients
Placebo
1.5 mg/m2
30 mg/m2
Dose Group (n=40)
(n=41) (n=36)
Change in eGFR at Week 8
(mL/min/1.73m2)
-0.6 1.3
0.1
P Value (vs. Placebo)
0.045 NS
Change in eGFR at Week 12 (mL/min/1.73m2)
-1.5 0.2
0.0
P Value (vs. Placebo)
0.07 NS
GCS-100’s effect on eGFR was more pronounced (p=0.029) in the
prospectively defined subset of patients with diabetic etiology.
Analysis of this subset was predefined based on the observation
that galectin-3 is elevated in the kidneys of diabetic CKD
patients, and the level of galectin-3 correlates with proteinuria
(a marker of kidney health) in these patients.
GCS-100 CKD Phase 2 Results – Patients with Diabetes
Placebo
1.5 mg/m2
30 mg/m2
Dose Group (n=29)
(n=24) (n=26) Change
in eGFR at Week 8 (mL/min/1.73m2) -0.5
2.3 -0.3
P Value
(vs. Placebo)
0.029 NS
GCS-100 was well-tolerated. There were no serious adverse
events, no Grade 3/4 adverse events and no early study
discontinuations in the 1.5 mg/m2 group. There was no observed
effect on blood pressure in any dose group.
“We are highly encouraged by these results,” stated 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. “The steady improvement in eGFR over time
that was maintained five weeks following the last dose and the lack
of a hemodynamic effect both lend support to GCS-100’s novel,
anti-fibrotic mechanism. Chronic kidney disease remains a major
medical problem, with tissue fibrosis at its core.”
An extension study is currently being conducted in which
patients from the Phase 2 study were re-randomized to receive
either 1.5 or 30 mg/m2 of GCS-100 (complete data available through
week 16). Of the 93 patients enrolled in total, 33 patients had
previously received placebo in the Phase 2 study before being
treated with GCS-100 in the extension study. This group, which
represents a set of patients receiving GCS-100 for the first time,
was analyzed for efficacy. Consistent with the blinded Phase 2
results, the 1.5 mg/m2 group experienced a significant improvement
in eGFR. This is the case when comparing these patients’ response
to both: (1) the response in the parallel randomized group
receiving 30 mg/m2 (p=0.04); and (2) the previous response to
placebo in the blinded Phase 2 study for placebo-treated patients
enrolled in the extension study (p=0.02).
GCS-100 Extension Study Results – Phase 2 Placebo
Patients Placebo(*)
1.5 mg/m2
30 mg/m2
Dose Group (n=33)
(n=20) (n=13) Change
in eGFR at Week 16 (mL/min/1.73m2) -2.0
1.3 -1.8
P Value
(vs. Placebo) 0.02
NS
P Value (vs. 30 mg/m2 Dose Group)
0.04
*Previous response to placebo in the
blinded Phase 2 study for placebo-treated patientsenrolled in the
extension study (baseline to week 12)
“We want to thank all of the patients and investigators who
contributed to making this study a success,” said George Tidmarsh,
M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We
look forward to advancing GCS-100 to the next phase of development
in chronic kidney disease, and to bringing this potentially
important therapy one step closer to the many patients suffering
from this terrible disease.”
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, a
type of galectin. 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 is
developing GCS-100 for the treatment of 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 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 (CRH) and
hepatorenal syndrome (HRS). GCS-100 is La Jolla’s first-in-class
galectin-3 inhibitor for the potential treatment of chronic kidney
disease (CKD). 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 (NASH) and other diseases characterized by tissue
fibrosis. LJPC-401 is La Jolla’s novel formulation of hepcidin for
the potential treatment of iron overload. For more information on
La Jolla, please visit http://www.ljpc.com.
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.
Forward-Looking Statement Safe Harbor
This document and the poster presentation 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 the Company’s expectations regarding future
events or the Company’s 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 CKD and the success of future
development activities for this and other drug development programs
sponsored by the Company. 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
Officer858-207-4264GTidmarsh@ljpc.comorChester S. Zygmont,
IIISenior Director of Finance858-207-4262czygmont@ljpc.com
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