La Jolla Pharmaceutical Company (NASDAQ:LJPC) (the Company or La
Jolla) today announced the initiation of a pivotal clinical study
of LJPC‑401 (synthetic human hepcidin) in patients with
transfusion-dependent beta thalassemia who, despite chelation
therapy, have cardiac iron levels above normal. A high level of
cardiac iron puts patients at risk of cardiac complications such as
heart failure and sudden death.
LJ401-BT01 is a pivotal, multinational, multicenter, randomized,
controlled study that is designed to enroll approximately 100
patients across 9 countries, including the United States. Patients
will be randomized 1:1 to receive either: (i) weekly subcutaneous
injections of LJPC‑401, while continuing standard-of-care chelation
therapy (LJPC‑401 treatment arm); or (ii) a continuation of
standard-of-care chelation therapy only (observation arm). After 6
months of treatment, patients randomized to the observation arm
will crossover to receive LJPC‑401 (plus standard-of-care chelation
therapy) for 6 months, while patients randomized to the LJPC-401
treatment arm will continue with LJPC-401 (plus standard-of-care
chelation therapy) for an additional 6 months (for a total of one
year).
The primary efficacy endpoint of this study is the change in
iron content in the heart after 6 months, as measured by cardiac
magnetic resonance imaging (MRI). 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.
“It is an important moment for all of those involved in the
research of blood diseases. For the first time, it is possible to
explore in humans the therapeutic potential of a natural master
regulator of body iron,” said Professor Antonio Piga, M.D.,
Department of Clinical and Biological Sciences School of Medicine,
San Luigi Gonzaga University Hospital in Torino, Italy.
“We are pleased to initiate this pivotal study at leading
research centers in the U.S. and worldwide,” said George F.
Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La
Jolla. “We look forward to continuing the research and development
efforts of LJPC‑401, with a goal of helping patients suffering from
iron overload disorders.”
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 Beta Thalassemia
Beta thalassemia is a disease characterized by a genetic
mutation that results in the underproduction of hemoglobin, the
body’s natural oxygen-carrying molecule contained in red blood
cells. There are three types of beta thalassemia: beta thalassemia
minor, beta thalassemia intermedia and beta thalassemia major.
Patients with the more severe forms (intermedia and major) suffer
from significant and sometimes life-threatening anemia, bone
deformities and enlargement of the spleen, 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.
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
Media Contact
Matt Middleman, M.D.LifeSci Public RelationsPhone:
646-627-8384Email: matt.middleman@lifescipublicrelations.com
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