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|La Jolla Pharmaceutical Company Announces Initiation of Pivotal Clinical Study of LJPC‑401 in Patients with Beta Thalassemia|
LJ401-BT01 is a pivotal, multinational, multicenter, randomized, controlled study that is designed to enroll approximately 100 patients across 9 countries, including
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
“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.”
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.
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.
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
Matt Middleman, M.D.
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