-Dose-dependent, Statistically Significant
Reduction in Serum Iron Observed (p=0.008)
-LJPC-401 Well Tolerated; No Dose-Limiting
Toxicities at Any Dose Level
-Conference Call at 9:00 AM Eastern Time on
Thursday, September 8, 2016
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 reported positive results
from its Phase 1 study of LJPC-401 in patients at risk of iron
overload.
The Phase 1 study is a multi-center, open-label, dose-escalation
study evaluating the safety, tolerability, and effect on serum iron
of a single dose of LJPC-401 in patients at risk of iron overload
due to conditions such as hereditary hemochromatosis (HH),
thalassemia, and sickle cell disease (SCD). Fifteen patients were
dosed at escalating dose levels ranging from 1 mg to 20 mg.
LJPC-401 was well tolerated, and there were no dose-limiting
toxicities observed. Injection-site reactions were the most
commonly reported adverse event. These were all mild or moderate in
severity, self-limiting, and fully resolved. There were no
significant changes in serum chemistries or hematology other than
serum iron parameters.
A dose-dependent, statistically significant reduction in serum
iron was observed (p=0.008 for dose response; not adjusted for
multiple comparisons). At the 20 mg dose level, LJPC-401 reduced
serum iron by an average of 58.1% from baseline to hour 8 (p=0.001;
not adjusted for potential regression to the mean effect), and
serum iron had not returned to baseline through day 7 (21.2%
reduction from baseline to the end of day 7).
Dose Group
Mean Percent Change in Serum Iron
from Baseline to Hour 8
1 mg (n=3) -14.2% (p=0.149) 5 mg (n=3) -26.7% (p=0.304) 10 mg (n=3)
-45.5% (p=0.054) 20 mg (n=6) -58.1% (p=0.001) P-values not adjusted
for potential regression to the mean effect
“The results from this study demonstrate a clear, dose-dependent
effect of LJPC-401 on serum iron, a clinically important measure,”
said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive
Officer of La Jolla. “We look forward to continuing the development
of LJPC-401, which we believe can potentially help patients
suffering from the effects of iron overload by restoring
physiologically relevant levels of hepcidin, the body’s natural
regulator of iron absorption and distribution.”
Conference Call at 9:00 AM Eastern Time on Thursday,
September 8, 2016
La Jolla will host a conference call and webcast on Thursday,
September 8, 2016 at 9:00 AM Eastern Time (6:00 AM Pacific Time).
The conference call can be accessed by dialing 877-359-9508 for
domestic callers and 224-357-2393 for international callers. Please
provide the operator with the passcode 77161203 to join the
conference call or click here for the webcast. A slide presentation
accompanying today’s press release and the conference call may also
be found on La Jolla’s website at www.ljpc.com under the investor
relations section. An archive of the conference call and webcast
will be available on La Jolla’s website for 30 days following the
call.
About LJPC-401
LJPC-401 is La Jolla’s novel formulation of synthetic hepcidin.
Hepcidin, an endogenous peptide hormone, is the body’s naturally
occurring regulator of iron absorption and distribution. In healthy
individuals, hepcidin prevents excessive iron accumulation in vital
organs, such as the liver and heart, where it can cause significant
damage and even result in death.
La Jolla is developing LJPC-401 for the potential treatment of
iron overload, which occurs as a result of diseases such as
hereditary hemochromatosis (HH), beta thalassemia, sickle cell
disease (SCD) and myelodysplastic syndrome (MDS). HH is a disease
characterized by a genetic deficiency in hepcidin. HH is the most
common genetic disease in Caucasians and causes liver cirrhosis,
liver cancer, heart disease and/or failure, diabetes, arthritis and
joint pain. Beta thalassemia, SCD and MDS are genetic diseases of
the blood that can cause life-threatening anemia and usually
require frequent and life-long blood transfusions. These blood
transfusions cause excessive iron accumulation in the body, which
is toxic to vital organs, such as the liver and heart. In addition,
the underlying anemia causes excessive iron accumulation
independent of blood transfusions.
In September 2016, La Jolla reported positive results from a
Phase 1 study of LJPC-401 in patients at risk of iron overload
suffering from HH, thalassemia and SCD. Single, escalating doses of
LJPC-401 were associated with a dose-dependent, statistically
significant reduction in serum iron. LJPC-401 was well tolerated
with no dose-limiting toxicities. Injection-site reactions were the
most commonly reported adverse event. These were all mild or
moderate in severity, self-limiting, and fully resolved.
Also in September 2016, La Jolla announced that it has reached
agreement with the European Medicines Agency (EMA) on the design of
a pivotal study of LJPC-401. The pivotal study will be a
randomized, controlled, multi-center study in beta thalassemia
patients suffering from iron overload, a major unmet need in an
orphan patient population. The primary endpoint will be a
clinically relevant measurement directly related to iron overload.
La Jolla plans to initiate this study in mid-2017.
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
several product candidates in development. LJPC-501 is La Jolla’s
proprietary formulation of angiotensin II for the potential
treatment of catecholamine-resistant hypotension. LJPC-401 is La
Jolla’s novel formulation of synthetic hepcidin for the potential
treatment of conditions characterized by iron overload, such as
hereditary hemochromatosis, beta thalassemia, sickle cell disease
and myelodysplastic syndrome. LJPC-30S is our next-generation
gentamicin derivative program that is focused on therapeutics for
the potential treatment of serious bacterial infections as well as
rare genetic disorders, such as cystic fibrosis and Duchenne
muscular dystrophy. For more information on La Jolla, please visit
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 the Company’s 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 www.sec.gov. These risks include, but
are not limited to, risks relating to: the timing for commencement
of clinical studies, the anticipated timing for completion of such
studies, and the anticipated timing for regulatory actions; the
success of future development activities; potential indications for
which the Company’s product candidates may be developed; and the
expected duration over which the Company’s cash balances will fund
its operations. 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.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20160907006780/en/
La Jolla Pharmaceutical CompanySandra VedrickSenior Manager,
Investor Relations & Human ResourcesPhone: (858) 256-7910Email:
svedrick@ljpc.comandDennis M. MulroyChief Financial OfficerPhone:
(858) 433-6839Email: dmulroy@ljpc.com
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