La Jolla Pharmaceutical Company Announces Initiation of Pivotal Clinical Study of LJPC‑401 in Patients with Beta Thalassemia

SAN DIEGO, CA, USA I December 04, 2017 I 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.

SOURCE: La Jolla Pharmaceutical Co

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