La Jolla Pharmaceutical Company (NASDAQ:LJPC) (the Company or La
Jolla) today announced the initiation of LJ401-HH01, a Phase 2
clinical study of LJPC‑401 (synthetic human hepcidin) in patients
with hereditary hemochromatosis (HH). The most common genetic
disease in Caucasians, HH is a disease characterized by a genetic
mutation that results in a deficiency in the production of
hepcidin, which is the body’s naturally occurring regulator of iron
absorption and distribution. Without normal levels of hepcidin,
excessive amounts of iron accumulate in the body. Symptoms of the
disease include joint pain, abdominal pain, fatigue and weakness.
If left untreated, HH can lead to liver cirrhosis, liver cancer,
heart disease and/or failure and diabetes.
LJ401-HH01 is a multinational, multicenter, randomized,
placebo-controlled, double-blind, Phase 2 study that is designed to
evaluate the safety and efficacy of LJPC-401 as a treatment for HH.
Approximately 60 patients will be randomized to receive weekly
subcutaneous injections of either LJPC‑401 or placebo for 12 weeks.
The primary efficacy endpoint of the study is the change in
transferrin saturation, a standard measurement of iron levels in
the body and one of the two key measurements used to detect iron
overload, from baseline to end of treatment. Secondary efficacy
endpoints include: (i) the change in serum ferritin, the other key
measurement used to detect iron overload, from baseline to end of
treatment; and (ii) the requirement for and frequency of phlebotomy
procedures during the study.
“There is a major need for new treatment modalities that improve
the quality of life of the many patients suffering from hereditary
hemochromatosis,” stated Jeff Vacirca, M.D., Chief of Clinical
Research at New York Cancer & Blood Specialists and
investigator in the study. “I am excited to be included in the
clinical evaluation of a treatment that harnesses the body’s
natural mechanism for iron regulation in a patient-friendly
treatment regimen that could potentially reduce or eliminate the
need for phlebotomy procedures in these patients.”
“Following the results of Phase 1 clinical studies that
demonstrated that the administration of LJPC-401 resulted in
dose-dependent reductions in iron levels, we are pleased to
initiate LJ401-HH01 to evaluate the therapeutic potential of
LJPC-401 in the important patient population of hereditary
hemochromatosis,” said George F. Tidmarsh, M.D., Ph.D.,
President and Chief Executive Officer of La Jolla. “With the recent
initiation of LJ401-BT01, our pivotal study of LJPC-401 in beta
thalassemia patients, LJPC-401 now is being evaluated in two,
multicenter, randomized, controlled clinical studies. These studies
are important components of our overall development program for
LJPC-401, which is aimed at helping the many patients suffering
from the effects of iron overload due to a variety of underlying
causes.”
The current standard treatment for HH is a blood removal
procedure known as phlebotomy. Each phlebotomy procedure, which is
usually conducted at a hospital, medical office or blood center,
typically involves the removal of approximately a pint of blood.
The required frequency of procedures varies by patient, but often
ranges from one to two times per week for an initial period after
diagnosis and once every one to three months chronically. Since
most of the body’s iron is stored in red blood cells, chronic
removal of blood can effectively lower iron levels if a phlebotomy
regimen is adhered to. However, phlebotomy procedures may cause and
may be associated with pain, bruising and scarring at the venous
puncture site, fatigue and dizziness during and following the
procedure and disruption of daily activities. Furthermore,
phlebotomy is not appropriate in patients with poor venous access,
anemia or heart disease.
About LJPC‑401
La Jolla is developing LJPC-401 (synthetic human hepcidin) 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). 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. The European
Medicines Agency (EMA) Committee for Orphan Medicinal Products
(COMP) has designated LJPC‑401 as an orphan medicinal product for
the treatment of beta thalassemia intermedia and major and SCD.
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 hereditary hemochromatosis, 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 and were all mild or moderate in severity, self-limiting and
fully resolved.
In December 2017, La Jolla announced the initiation of a
pivotal, multinational, multicenter, randomized, controlled study
of LJPC-401 in patients with transfusion-dependent beta thalassemia
who, despite chelation therapy, have cardiac iron levels above
normal. La Jolla had previously announced that it had reached
agreement with the European Medicines Agency (EMA) on the design of
this registration study of LJPC-401.
About Hereditary Hemochromatosis
Hereditary hemochromatosis (HH) is the most common genetic
disease in Caucasians. HH is a disease characterized by a genetic
mutation that results in a deficiency in the production of
hepcidin, which is the body’s naturally occurring regulator of iron
absorption and distribution. Without normal levels of hepcidin,
excessive amounts of iron accumulate in the body. Symptoms of the
disease include joint pain, abdominal pain, fatigue and weakness.
If left untreated, HH can lead to liver cirrhosis, liver cancer,
heart disease and/or failure and diabetes.
The current standard treatment for HH is a blood removal
procedure known as phlebotomy. Each phlebotomy procedure, which is
usually conducted at a hospital, medical office or blood center,
typically involves the removal of approximately a pint of blood.
The required frequency of procedures varies by patient, but often
ranges from one to two times per week for an initial period after
diagnosis and once every one to three months chronically. Since
most of the body’s iron is stored in red blood cells, chronic
removal of blood can effectively lower iron levels if a phlebotomy
regimen is adhered to. However, phlebotomy procedures may cause and
may be associated with pain, bruising and scarring at the venous
puncture site, fatigue and dizziness during and following the
procedure and disruption of daily activities. Furthermore,
phlebotomy is not appropriate in patients with poor venous access,
anemia or heart disease.
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 (synthetic
human angiotensin II) is being developed for the potential
treatment of hypotension in adult patients with distributive or
vasodilatory shock who remain hypotensive despite fluid and
vasopressor therapy. LJPC‑401 (synthetic human hepcidin) is being
developed for the potential treatment of conditions characterized
by iron overload, such as hereditary hemochromatosis, beta
thalassemia, sickle cell disease and myelodysplastic syndrome. For
more information on La Jolla, please visit www.ljpc.com.
Forward Looking Statement Safe Harbor
This press release 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
or statements of current expectations and involve known and unknown
risks, uncertainties and other factors, that may cause actual
results to be materially different from those anticipated by the
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 website www.sec.gov. These risks include, but are not
limited to, risks relating to: the timing, costs, conduct and
outcome of clinical studies; the anticipated treatment of future
clinical data by the FDA, the EMA or other regulatory authorities,
including whether such data will be sufficient for approval; the
timing and prospects for approval of LJPC-501 or LJPC-401 by the
FDA, the EMA or other regulatory authorities; risks relating to the
scope of product labels (if approved); potential market sizes; the
success of future development activities; potential indications for
which the Company’s product candidates may be developed; the
anticipated timing for regulatory actions; the impact of
pharmaceutical industry regulation and healthcare legislation
in the United States; and the success of future development
activities. The Company expressly disclaims any intent to update
any forward‑looking statements to reflect the outcome of subsequent
events.
Company Contacts
Sandra VedrickAssociate Director, Investor Relations & Human
ResourcesLa Jolla Pharmaceutical CompanyPhone: 858 207 4264 Ext:
1135Email: svedrick@ljpc.com
and
Dennis M. MulroyChief Financial OfficerLa Jolla Pharmaceutical
CompanyPhone: 858 207 4264 Ext: 1040Email: dmulroy@ljpc.com
La Jolla Pharmaceutical (NASDAQ:LJPC)
Historical Stock Chart
From Mar 2024 to Apr 2024
La Jolla Pharmaceutical (NASDAQ:LJPC)
Historical Stock Chart
From Apr 2023 to Apr 2024