RARE Daily

La Jolla Pharmaceuticals Halts Development of Beta Thalassemia Drug as CEO Departs

November 27, 2019

La Jolla Pharmaceuticals said it was halting development of its experimental therapy to treat iron overload in beta thalassemia patients because of a lack of clinical efficacy as its president and CEO George Tidmarsh stepped down. A small committee of the company’s board will provide oversight of the management team.

The company said Tidmarsh’s left the company “to pursue other interests.”

LJPC‑401 is La Jolla’s experimental product for the potential treatment of conditions characterized by iron overload. It is a synthetic human hepcidin, an endogenous peptide hormone that 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 has been 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.

La Jolla said it is reassessing the development of LJPC-401 to treat iron overload in beta thalassemia patients who, despite chelation therapy, have cardiac iron levels above normal. It stopped the study after an interim analysis found that there were no significant differences in the primary endpoint or key secondary endpoints between patients on the treatment arm and patients on the control arm.

The company said it will now focus on maximizing sales of Giapreza, its vasoconstrictor used to increase blood pressure in adults with septic or other distributive shock and pursue approval in the United States of LJPC-0118 for the treatment of severe malaria. La Jolla has already submitted an application for approval with the U.S. Food and Drug Administration.

Photograph: George Tidmarsh, who just stepped down as president and CEO of La Jolla Pharmaceuticals

Author: Rare Daily Staff

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