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LA JOLLA PHARMACEUTICAL EXPANDS ENROLLMENT IN
PHASE 3 INTERNATIONAL RIQUENT® STUDY
Patients to receive higher doses than in previous studies
SAN
DIEGO ,
CA, August
9, 2006 – La
Jolla Pharmaceutical Company
(Nasdaq: LJPC)
announced today that it has
reactivated enrollment in its
Phase 3 trial of Riquent ® (abetimus
sodium) for
the treatment of lupus renal
disease. To date, the Company
has activated 30 sites, 21 in
the United States and 9 in Asia.
“This
global Phase 3 study will be the
largest clinical trial ever conducted
in lupus patients with a history
of renal disease,” said
Deirdre Y. Gillespie, M.D., President
and CEO of La Jolla
Pharmaceutical Company. “ Of
primary importance is the fact
that w e have worked with the
U.S. Food and Drug Administration
(FDA) and outside experts to optimize
the design of the clinical trial
to increase the potential for
Riquent to address the significant
unmet need in the treatment of
lupus. We are aggressively moving
forward to enroll patients now
and expect to complete recruitment
in the second half of 2007.”
In
addition to sites in the United
States and Asia, the Company is
expanding the study to Europe
and Mexico. A total of approximately
600 patients from about 100 clinical
sites in more than 10 countries
will be enrolled in this international
study.
The
current study follows a review
of the Company’s
New Drug Application for Riquent
by the FDA, which informed the
Company that Riquent was “approvable” subject
to the successful completion of
another Phase 3 clinical benefit
trial. The approvable letter indicated
that the current Phase 3 clinical
trial would appear to satisfy
this requirement.
The
current trial is a double-blind,
placebo-controlled, randomized
trial designed to gain FDA approval
for Riquent as a treatment for
lupus renal disease, a leading
cause of sickness and death in
lupus patients. A differentiating
feature of this trial is that
it specifies two doses of Riquent
that are higher than those used
in previous studies. Previous
research indicates that higher
Riquent doses may further reduce
levels of antibodies to double-stranded
DNA (dsDNA), which are believed
to cause lupus renal disease,
and thus may increase the clinical
benefit of treatment with Riquent.
Phase
3 Trial Overview
The
protocol of the double-blind,
placebo-controlled Phase 3 clinical
trial was finalized under a Special
Protocol Assessment (SPA) with
the FDA and includes several modifications
that are designed to improve the
probability of demonstrating the
clinical benefit of Riquent. The
changes to the protocol incorporated
in the current study are based
on data generated during the Company’s
previous Phase 2/3 and Phase 3
trials of Riquent. These changes
include:
- Twice
as many patients will be treated
with Riquent as will be treated
with placebo (2:1 randomization).
80% of Riquent-treated patients
will be treated with a higher
dose than the previous Phase
3 trial – 40%
of Riquent-treated patients
will receive 300 mg of Riquent
per week, 40% will receive 900
mg per week, and 20% will receive
100 mg per week, the dose used
in previous trials. Furthermore,
the Company believes that higher
doses of Riquent should further
reduce levels of antibodies
to dsDNA, which may increase
the clinical benefit of treatment
with Riquent.
- Approximately
600 patients will be enrolled
in this trial, compared to approximately
300 patients in the previous
Phase 3 study. Studying a larger
number of patients should increase
the likelihood of seeing a statistically
significant difference between
the Riquent-treated group and
the placebo-treated group.
- The
primary endpoint of the study,
time to renal flare, has been
simplified by eliminating hematuria
(blood in the urine) from the
definition of a renal flare.
Hematuria can be less specific
and can occur due to conditions
other than a renal flare.
- The
current trial will evaluate
treatment for 12 months, as
compared to the previous Phase
3 trial, which treated patients
for up to 22 months.
- The
study entry criteria will further
restrict the use of immunosuppressive
agents. In the previous Phase
3 study, the use of these agents
may have reduced the renal flare
rate, especially in placebo-treated
patients.
- The
trial will involve a greater
number of nephrology clinics,
which should allow the study
to enroll more patients with
impaired renal function. It
is that specific group that
showed increased benefit from
Riquent treatment.
About
Riquent
Riquent
is the first drug candidate specifically
developed for the treatment of
lupus renal disease, a leading
cause of sickness and death in
lupus patients. The drug candidate
has already been evaluated in
13 clinical trials over a 10-year
time span that evaluated more
than 800 patients and subjects.
Riquent has been well tolerated
in all of these studies, with
no serious side effects identified
to date. Riquent’s
only known biological activity
is the reduction of antibodies
to dsDNA that are associated with
the progression of lupus renal
disease and renal flares. In all
clinical trials where antibodies
to dsDNA were measured, Riquent
treatment has significantly reduced
these antibody levels. Data generated
from the Company’s
previous Phase 2/3 and Phase 3
trials indicated that patients
with lower antibody levels experienced
significantly fewer renal flares
and improved health-related quality
of life.
About Lupus
Lupus
(systemic lupus erythematosus)
is a chronic, potentially life-threatening
autoimmune disease. About 90%
of lupus patients are female,
and many develop the disease during
their childbearing years. Approximately
50% of lupus patients have renal
disease, which can lead to irreversible
renal damage, renal failure and
the need for dialysis, and is
a leading cause of death in lupus
patients. Latinos, African Americans
and Asians face an increased risk
of serious renal disease associated
with lupus. The current standard
of care for lupus renal disease
often involves treatment with
high doses of corticosteroids
and immunosuppressive drugs that
can cause severe side effects
including diabetes, hypertension
and sterility, and may leave patients
vulnerable to opportunistic infections.
To date, no lupus specific drug
has been approved in the U.S.
La
Jolla Pharmaceutical Company is
a biotechnology company developing
therapeutics for antibody-mediated
autoimmune diseases and inflammation
afflicting several million people
around the world. The Company’s
leading product in development
is Riquent®,
which is designed to treat lupus
renal disease, a leading cause
of sickness and death in patients
with lupus. The Company is also
developing small molecules to
treat various other autoimmune
and inflammatory conditions. The
Company's common stock is traded
on The NASDAQ Global Market under
the symbol LJPC. More information
about the Company is available
on its website: http://www.ljpc.com.
The
forward-looking statements in
this press release involve significant
risks and uncertainties, and
a number of factors, both foreseen
and unforeseen, that could cause
actual results to differ materially
from our current expectations.
Forward-looking statements include
those that express a plan, belief,
expectation, estimation, anticipation,
intent, contingency, future
development or similar expression.
The analyses of clinical results
of Riquent, previously known
as LJP 394, our drug candidate
for the treatment of systemic
lupus erythematosus ("lupus"),
and any other drug candidate
that we may develop, including
the results of any trials or
models that are ongoing or that
we may initiate in the future,
could result in a finding that
these drug candidates are not
effective in large patient populations,
do not provide a meaningful
clinical benefit, or may reveal
a potential safety issue requiring
us to develop new candidates.
The analysis of the data from
our Phase 3 trial of Riquent
showed that the trial did not
reach statistical significance
with respect to its primary
endpoint, time to renal flare,
or with respect to its secondary
endpoint, time to treatment
with high-dose corticosteroids
or cyclophosphamide. The results
from our clinical trials of
Riquent, including the results
of any trials that are ongoing
or that we may initiate in the
future, may not ultimately be
sufficient to obtain regulatory
clearance to market Riquent
either in the United States
or Europe, and we may be required
to conduct additional clinical
studies to demonstrate the safety
and efficacy of Riquent in order
to obtain marketing approval.
There can be no assurance, however,
that we will have the necessary
resources to complete any current
or future trials or that any
such trials will sufficiently
demonstrate the safety and efficacy
of Riquent. Our blood test to
measure the binding affinity
for Riquent is experimental,
has not been validated by independent
laboratories and will likely
be reviewed as part of the Riquent
approval process. Our SSAO inhibitor
program is at a very early stage
of development and involves
comparable risks. Analysis of
our clinical trials could have
negative or inconclusive results.
Any positive results observed
to date in our clinical trials
or animal models may not be
indicative of future results.
In any event, regulatory authorities
may require clinical trials
in addition to our current clinical
trial, or may not approve our
drugs. Our ability to develop
and sell our products in the
future may be adversely affected
by the intellectual property
rights of third parties. Additional
risk factors include the uncertainty
and timing of: obtaining required
regulatory approvals, including
delays associated with any approvals
that we may obtain; our ability
to pass all necessary FDA inspections;
the availability of sufficient
financial resources; the increase
in capacity of our manufacturing
capabilities for possible commercialization;
successfully marketing and selling
our products; our lack of manufacturing,
marketing and sales experience;
our ability to make use of the
orphan drug designation for
Riquent; generating future revenue
from product sales or other
sources such as collaborative
relationships; future profitability;
and our dependence on patents
and other proprietary rights.
Readers are cautioned to not
place undue reliance upon forward-looking
statements, which speak only
as of the date hereof, and we
undertake no obligation to update
forward-looking statements to
reflect events or circumstances
occurring after the date hereof.
Interested parties are urged
to review the risks described
in our Annual Report on Form
10-K for the year ended December
31, 2005, and in other reports
and registration statements
that we file with the Securities
and Exchange Commission from
time to time.
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