La Jolla to Reassess Continued Development of LJPC-401 Based on Recent Clinical Results
The Company plans to discontinue Study LJ401-BT01 due to lack of efficacy. Study LJ401-BT01 is a pivotal, multinational, multicenter, randomized, controlled study with a target enrollment of approximately 100 patients that is designed to evaluate the safety and efficacy of LJPC-401 as a treatment for iron overload in beta thalassemia (BT) patients who, despite chelation therapy, have cardiac iron levels above normal. The primary endpoint of this study is the change in cardiac iron levels, as measured by cardiac T2* magnetic resonance imaging (MRI), from baseline to 6 months following treatment. The Company recently conducted an interim analysis that included approximately one-half of the target-enrolled patients. 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.
Topline results of Study LJ401-HH01 are also now available. Study LJ401-HH01 is a multinational, multicenter, randomized, placebo-controlled, double-blind, Phase 2 study with a target enrollment of approximately 60 patients that is designed to evaluate the safety and efficacy of LJPC-401 as a treatment for patients with hereditary hemochromatosis (HH). Topline results from this study are consistent with the interim results reported in
The Company expects to re-evaluate its current operating plan in light of the mixed results of these studies and to make adjustments as appropriate to manage the Company’s available cash resources. The Company’s near-term focus is to: (1) maximize sales of GIAPREZATM (angiotensin II) as a vasoconstrictor indicated to increase blood pressure in adults with septic or other distributive shock in the U.S., where it was launched by La Jolla in the first quarter of 2018; (2) maximize the value of GIAPREZA for the treatment of refractory hypotension in adults with septic or other distributive shock who remain hypotensive despite adequate volume restitution and application of catecholamines and other available vasopressor therapies in
LJPC‑401 (synthetic human hepcidin) is La Jolla’s investigational product for the potential treatment of conditions characterized by iron overload. 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 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.
This press release contains “forward-looking statements” as defined by the Private Securities Litigation Reform Act of 1995. We caution investors that forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and involve substantial risks and uncertainties that could cause the actual outcomes to differ materially from what we currently expect. These risks and uncertainties include, but are not limited to, those associated with: GIAPREZATM (angiotensin II) sales; cash used in operating activities; regulatory actions relating to La Jolla’s products by the
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Chief Financial Officer
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Source: La Jolla Pharmaceutical Company