La Jolla Pharmaceutical Company (Nasdaq: LJPC), a leader in the
discovery, development and commercialization of innovative
therapies intended to significantly improve outcomes in patients
suffering from life-threatening diseases, today announced positive
results from the pre-specified interim analysis of its Phase 2
study of LJPC-401 (synthetic human hepcidin) in patients with
hereditary hemochromatosis (HH). The interim analysis of efficacy
included 26 patients who have reached the end of the
16-week treatment period (the efficacy population:
12 LJPC-401-treated patients; 14 placebo-treated
patients), and the interim analysis of safety included 60
randomized patients (the safety population: 29 LJPC-401-treated
patients; 31 placebo-treated patients).
The change in TSAT from baseline to the end of treatment (16
weeks), the primary efficacy endpoint of the study, was
statistically significant: LJPC‑401‑treated patients had a mean
reduction in TSAT of 42% compared to placebo-treated patients who
had a mean reduction of 6% (p<0.0001).
The requirement for and frequency of phlebotomy procedures, a
key secondary endpoint of the study, was statistically significant:
LJPC-401-treated patients had 0.06 phlebotomies per month compared
to placebo-treated patients who had 0.41 phlebotomies per month
(p=0.003). There were 3 phlebotomies in 2 LJPC-401-treated patients
and 24 phlebotomies in 9 placebo-treated patients.
LJPC-401 was well tolerated. The most frequent
treatment-emergent adverse events (TEAEs) were injection site
reactions (ISRs), which occurred in 79% of LJPC‑401-treated
patients compared to 6% of placebo-treated patients. The ISRs were
all mild or moderate in severity, and no ISRs resulted in treatment
discontinuation. As of the interim analysis, there were no serious
TEAEs reported.
“The robustness of this early readout supporting further
development of LJPC-401 is very encouraging,” said Jeff Vacirca,
M.D., Chief of Clinical Research at New York Cancer & Blood
Specialists and an Investigator in the Study. “There have been no
new treatment modalities introduced for patients with hereditary
hemochromatosis in more than a decade. In light of the negative
impact that repeated phlebotomy procedures have on patient
quality-of-life, patients would welcome a pharmacologic treatment
that they can self-administer and that addresses the underlying
pathophysiology of the disease.”
About the LJ401-HH01 Study LJ401-HH01 is a
multinational, multicenter, randomized, placebo-controlled,
double-blind, Phase 2 study designed to evaluate the safety and
efficacy of LJPC-401 as a treatment for hereditary hemochromatosis
(HH). Approximately 60 patients have been randomized to receive
weekly subcutaneous injections of either LJPC‑401 or placebo for 16
weeks. Targeted enrollment consisted primarily of patients in the
maintenance phase of their HH treatment, where serum ferritin had
already been lowered while transferrin saturation (TSAT) remained
elevated. There was a subset of patients in the study in the
induction phase of their HH treatment where both serum ferritin and
TSAT were elevated.
The primary efficacy endpoint of the study is the change in
TSAT, 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 at week 16. Secondary efficacy
endpoints include the requirement for and frequency of phlebotomy
procedures during the study.
We expect to announce topline results of LJ401-HH01 in the
second half of 2019.
About LJPC-401 LJPC-401, a clinical-stage
investigational product, is La Jolla’s proprietary 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 primary iron overload
diseases such as hereditary hemochromatosis (HH), or secondary iron
overload diseases such as beta thalassemia (BT), sickle cell
disease (SCD), myelodysplastic syndrome (MDS) and polycythemia
vera. The European Medicines Agency (EMA) Committee for Orphan
Medicinal Products has designated LJPC‑401 as an orphan medicinal
product for the treatment of BT intermedia and major and SCD.
About Hereditary Hemochromatosis
Hereditary hemochromatosis (HH) is the most common genetic
disease in Caucasians. HH is a disease characterized by a genetic
mutation that causes excessive iron absorption and accumulation due
to hepcidin deficiency or insensitivity. Hepcidin 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.
There are no FDA approved therapies for HH and 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 for life. 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, joint
pain, 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. GIAPREZA™
(angiotensin II), formerly known as LJPC-501, was approved by
the U.S. Food and Drug Administration on December
21, 2017 as a vasoconstrictor indicated to increase blood pressure
in adults with septic or other distributive shock. LJPC-0118 is La
Jolla’s investigational product for the treatment of severe
malaria. LJPC‑401 (synthetic human hepcidin), a clinical-stage
investigational product, is being developed for the potential
treatment of conditions characterized by iron overload, such as
hereditary hemochromatosis, beta thalassemia, sickle cell disease,
myelodysplastic syndrome and polycythemia vera. For more
information on La Jolla, please visit www.ljpc.com.
Forward-Looking Statements
This press release contains forward-looking statements as
defined by the Private Securities Litigation Reform Act of 1995.
These statements relate to expectations regarding future events or
La Jolla’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. La Jolla 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
La Jolla’s filings with the U.S. Securities and Exchange
Commission (SEC), all of which are available free of charge on
the SEC’s website at www.sec.gov. These risks include, but are not
limited to, risks relating to: the success of development
activities for LJPC-401 and other product candidates; the
consistency between the full data set, topline data and interim
results from the LJ401-HH01 study; scope of product labels (if
approved) and potential market sizes, as well as the broader
commercial opportunity; potential indications for which La Jolla’s
product candidates may be developed; the timing, costs, conduct and
outcome of clinical studies; risks relating to the development of
drug candidates; the anticipated treatment of future clinical data
by the regulatory authorities, including whether such data will be
sufficient for approval; and other risks and uncertainties
identified in our filings with the SEC. Forward-looking
statements are presented as of the date of this press release, and
La Jolla expressly disclaims any intent to update any
forward‑looking statements to reflect the outcome of subsequent
events.
Company Contacts
Sandra VedrickSenior 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
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