La Jolla Pharmaceutical Company Announces LJPC-401 Presentations at 23rd Congress of the European Hematology Association
May 17 2018 - 12:00PM
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 that
there will be two presentations on LJPC-401 (synthetic human
hepcidin) at the 23rd Congress of the European Hematology
Association (EHA) to be held June 14 - 17, 2018 in Stockholm,
Sweden. Abstracts for these presentations were made publicly
available today and can be found on the EHA website and by clicking
the titles below.
Presentation Details:
Oral Presentation Title: |
A
Phase 1, Open-Label Study to Determine the Safety, Tolerability,
and Pharmacokinetics of Escalating Doses of LJPC-401 (Synthetic
Human Hepcidin) in Patients with Iron Overload |
Presenter: |
Vip
Viprakasit, M.D., Professor of Pediatrics, Division of
Hematology-Oncology, Department of Pediatrics & Thalassemia
Center, Faculty of Medicine, Siriraj Hospital, Mahidol University,
Bangkok, Thailand |
Session Title: |
Iron:
From basic science to clinical application |
Oral Presentation Date/Time: |
Saturday,
June 16, 2018 / 4:45 pm - 5:00 pm CEST |
Session Room: |
A13 |
|
|
Poster Presentation Title: |
A
Phase 1, Placebo-Controlled Study to Determine the Safety,
Tolerability, and Pharmacokinetics of Escalating Subcutaneous Doses
of LJPC-401 (Synthetic Human Hepcidin) in Healthy
Adults |
Presenter: |
Antonis
Kattamis M.D., Professor, Head, Division of Pediatric
Hematology-Oncology, First Department of Pediatrics, National and
Kapodistrian University of Athens, Aghia Sophia Children's
Hospital, Athens, Greece |
Session Title: |
Iron
metabolism, deficiency and overload |
Poster Presentation Date/Time: |
Friday,
June 15, 2018 / 5:30 pm - 7:00 pm CEST |
Session Room: |
Poster
Area |
|
|
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, sickle cell disease (SCD) and myelodysplastic
syndrome (MDS). 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 HH, thalassemia and SCD. In this study, 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 September 2016, La Jolla reached agreement with the EMA on
the design of a pivotal study of LJPC-401 for the treatment of beta
thalassemia patients suffering from iron overload, a major unmet
need in an orphan patient population. This study, also known as
LJ401-BT01, was initiated in December 2017. If this study is
successful, La Jolla anticipates filing an MAA for LJPC-401 in
Europe. Also in December 2017, La Jolla announced the initiation of
LJ401-HH01, a Phase 2 clinical study of LJPC‑401 in patients with
HH. 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.
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
(FDA) on December 21, 2017 as a vasoconstrictor to
increase blood pressure in adults with septic or other distributive
shock. 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
and myelodysplastic syndrome. For more information, please
visit www.ljpc.com.
Forward-looking Statements
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 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 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-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; and other risks and
uncertainties identified in our filings with the SEC. La Jolla
expressly disclaims any intent to update any forward‑looking
statements to reflect the outcome of subsequent events.
Company Contacts
Sandra VedrickDirector, 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
Sylvia WheelerWheelhouse Life Science AdvisorsEmail:
swheeler@wheelhouselsa.com
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