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 the European
Medicines Agency (EMA) Committee for Orphan Medicinal Products
(COMP) issued a positive opinion recommending LJPC-401 (synthetic
human hepcidin) for designation as an orphan medicinal product for
the treatment of Sickle Cell Disease (SCD).
SCD is the most common inherited blood disorder in the United
States and is caused by a genetic mutation that results in the
production of abnormal hemoglobin, the body’s natural
oxygen-carrying molecule contained in red blood cells. The abnormal
hemoglobin causes the red blood cells to form a “sickle,” or
crescent, shape, which may cause occlusion of blood vessels.
Patients with severe forms suffer from sometimes life-threatening
chronic hemolytic anemia, strokes, and damage to vital organs such
as the lungs, spleen, kidney and liver. In patients with chronic
hemolytic anemia, hepcidin levels may also be suppressed, which may
lead to iron overload. Standard treatment of SCD includes frequent,
life-long blood transfusions. While lifesaving, these transfusions
cause excess iron accumulation, which in turn is toxic to vital
organs, such as the liver and heart. The only currently approved
treatments for iron overload are iron chelators, which may cause
kidney failure, liver failure or gastrointestinal hemorrhage. In
addition to potentially improving iron overload in these patients,
LJPC-401 holds the potential to improve the consequences of the
disease by reducing the red cell hemoglobin concentration leading
to less sickle cell formation.
LJPC-401 is La Jolla’s novel formulation of synthetic human
hepcidin, a naturally occurring peptide hormone that is the body’s
regulator of iron absorption and distribution. Hepcidin prevents
abnormal iron accumulation in 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, beta thalassemia, SCD and myelodysplastic
syndrome. In September 2015, the COMP designated LJPC-401 as an
orphan medicinal product for the treatment of beta thalassemia
intermedia and major.
“We are encouraged by the positive feedback and continued
support of the European regulatory authorities,” said George F.
Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La
Jolla. “Following our recently reported, positive results from our
Phase 1 study of LJPC-401, which demonstrated a clear,
dose-dependent effect of LJPC-401 on serum iron, and our reaching
of an agreement with the EMA on study design, we look forward to
initiating our pivotal study of LJPC-401 in mid-2017.”
About European Orphan Drug Designation
Orphan drug designation is a status assigned to a medicine
intended for use in rare diseases. To be granted orphan status in
the European Union (EU), the medicine must be for the treatment of
a life-threatening or chronically debilitating condition that
affects no more than five in 10,000 people in the EU and for which
no satisfactory treatments exist or, where they do exist, the
medicine will be of significant benefit to those affected by that
condition.
Applications for orphan designation are evaluated by the
European Medicines Agency’s (EMA) Committee for Orphan Medicinal
Products (COMP), which provides its opinion on whether or not the
medicine qualifies as an orphan medicine for the treatment,
prevention or diagnosis of a rare disease. If the COMP issues a
positive opinion, the European Commission (EC) may then grant the
medicine orphan status.
An orphan designation allows a pharmaceutical company to benefit
from incentives from the EU to develop a medicine for a rare
disease, such as reduced fees, regulatory support during the
product development phase, access to the centralized authorization
procedure (a single application for all EU countries), and 10 years
of market exclusivity once the medicine is approved.
About LJPC-401
LJPC-401 is La Jolla’s novel formulation of synthetic human
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 human 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20161024005420/en/
La Jolla Pharmaceutical CompanySandra VedrickSenior Manager,
Investor Relations & Human
Resources858-256-7910svedrick@ljpc.comorLa Jolla Pharmaceutical
CompanyDennis M. MulroyChief Financial
Officer858-433-6839dmulroy@ljpc.com
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