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LA JOLLA PHARMACEUTICAL ANNOUNCES
RESULTS OF PHASE I/II TRIAL IN ANTIBODY-MEDIATED
THROMBOSIS PATIENTS AT ACR
Results presented today at the
American College of Rheumatology Meeting
SAN DIEGO, CA October 27, 2002 - La
Jolla Pharmaceutical Company (Nasdaq:
LJPC) announced preliminary results
from the first clinical trial evaluating
its experimental drug candidate, LJP
1082, for the treatment of antibody-mediated
stroke, heart attack, deep-vein thrombosis
and recurrent miscarriage. The Phase
I/II trial was a randomized, placebo-controlled
study that was designed to evaluate
the safety and activity of a single
dose of LJP 1082. Based on an initial
assessment of the trial data, the drug
was well tolerated at all five dose
levels. LJP 1082 had an elimination
half-life of at least 12 hours following
intravenous administration.
LJP 1082 is designed to reduce antibodies
to the first domain of beta 2 glycoprotein
1 (beta 2 GP1) that are believed to
promote antibody-mediated thrombosis.
LJP 1082 is composed of four copies
of the first domain of beta 2 GP1 coupled
to a synthetic chemical platform. Following
treatment with a single 50 mg or 200
mg dose, antibodies to LJP 1082 were
reduced in some patients. In total,
20 patients with a history of antibody-mediated
thrombosis participated in the trial
period.
Standard safety assessments including
physical exams, lab values and vital
signs, and immunology specific measurements
were taken over 30 days following a
single dose of LJP 1082. All adverse
events observed were categorized as
mild to moderate and deemed to have
no or an unlikely relationship to study
drug. The adverse event profiles appeared
similar between drug-treated and placebo-treated
groups. There were no serious adverse
events. No significant increase in circulating
immune complexes, changes in complement
protein C3 or activation of patient
T cells was observed following drug
treatment.
Even though there were a small number
of patients in the study, there was
an apparent dose-dependent response
following drug treatment. Patients receiving
higher doses of LJP 1082 had larger
reductions in antibodies to LJP 1082.
In the 50 and 200 mg treatment groups,
there was an apparent correlation between
the level of reduction and the affinity
of the patient's antibodies for drug.
Most patients had antibodies that were
specific for the first domain of the
target protein, beta 2 GP1, which is
the epitope presented on LJP 1082.
This study is the first of several
that may be required to establish appropriate
dose regimens and the observed reductions
may not be large enough to affect patient
health or reduce antibodies to beta
2 GP1in a majority of patients. Additional
analyses are ongoing. Potential drug
interference in some of the antibody
assays is also being evaluated. This
study was not designed to evaluate the
ability of LJP 1082 to tolerize B cells
that produce antibodies to beta 2 GP1
and additional studies will be needed.
These findings were presented today
at the American College of Rheumatology
Annual Meeting currently being held
in New Orleans. Matthew Linnik, Ph.D.,
Chief Scientific Officer and Executive
Vice President of Research at La Jolla
Pharmaceutical presented the poster
entitled "A Novel Therapeutic Approach
for Treating Antiphospholipid Syndrome
Based on Tolerizing anti-beta 2 glycoprotein
1 B cells."
Design of Phase I/II trial for LJP
1082
In the Phase I/II trial, five different
groups, each consisting of four or five
patients, were treated with a single
intravenous dose of LJP 1082 of 1, 3,
10, 50 or 200 mg and then monitored
for 30 days. One patient in each group
received placebo. In order to participate
in the trial, patients were required
to have elevated levels of antibodies
to beta 2 GP1, the target of the antibodies
involved in antibody-mediated thrombosis.
"LJP 1082 is designed to specifically
prevent the production of disease-causing
antibodies, while leaving the rest of
the immune system fully functional and
without the increased risk of bleeding
episodes associated with existing anticoagulant
therapy," said Dr. Linnik.
Antibody-mediated Thrombosis
Antibody-mediated thrombosis is an
autoimmune disease characterized by
the formation of blood clots that lead
to stroke, heart attack, deep-vein thrombosis
and recurrent miscarriage. This disease,
also know as Antiphospholipid Syndrome,
affects an estimated one to two million
people in the United States and Europe.
Patients often experience their first
thrombotic event in their 20s or 30s,
and studies indicate they have twice
the probability of a recurrence. Current
treatments include anticoagulants, the
long-term use of which can lead to side
effects including life-threatening bleeding
events. LJP 1082 is the first drug candidate
specifically designed to treat the underlying
cause of antibody-mediated thrombosis.
La Jolla Pharmaceutical Company is
a biotechnology company leading the
development of therapeutics for antibody-mediated
autoimmune diseases afflicting several
million people in the United States
and Europe. The Company is conducting
a Phase III trial of LJP 394 in patients
with lupus kidney disease, a leading
cause of sickness and death in these
patients. The Company recently completed
a Phase I/II trial of LJP 1082 for the
treatment of antibody-mediated thrombosis,
a condition in which patients suffer
from recurrent stroke, deep-vein thrombosis
and other thrombotic events. The Company's
common stock is traded on The Nasdaq
Stock Market under the symbol LJPC.
For more information about the Company,
visit our Web site: http://www.ljpc.com.
Except for historical statements, this
press release contains forward-looking
statements involving significant risks
and uncertainties, and a number of factors,
both foreseen and unforeseen, could
cause actual results to differ materially
from our current expectations. Our analyses
of clinical results of LJP 394, our
drug candidate for the treatment of
systemic lupus erythematosus ("lupus"),
and LJP 1082, our drug candidate for
the treatment of antibody-mediated thrombosis
("thrombosis"), are ongoing
and future analyses 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.
Our blood test to measure the binding
affinity for LJP 394 is experimental,
has not been validated by independent
laboratories, may require regulatory
approval and may be necessary for the
approval and the commercialization of
LJP 394. Our other potential drug candidates
are at earlier stages of development
and involve comparable risks. Analysis
of our clinical trials could have negative
or inconclusive results. Any positive
results observed to date may not be
indicative of future results. Even if
results are promising, the U.S. Food
and Drug Administration ("FDA")
may require additional clinical trials.
The Company's ability to develop and
sell its products in the future may
be affected by the intellectual property
rights of third parties. Additional
risk factors include the uncertainty
of: obtaining required regulatory approvals;
successfully marketing products; receiving
future revenue from product sales or
other sources such as collaborative
relationships; future profitability;
the need for additional financing; our
dependence on patents and other proprietary
rights; FDA approval of our manufacturing
facilities; the increase in capacity
of our manufacturing capabilities for
possible commercialization; and our
lack of marketing experience. 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 other reports and registration
statements filed with the Securities
and Exchange Commission from time to
time, including the report on Form 10-K
for the year ended December 31, 2001.

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