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|La Jolla Pharmaceutical Company Reports Positive Results from Phase 1 Study of LJPC-401|
-Dose-dependent, Statistically Significant Reduction in Serum Iron Observed (p=0.008)
-LJPC-401 Well Tolerated; No Dose-Limiting Toxicities at Any Dose Level
-Conference Call at
The Phase 1 study is a multi-center, open-label, dose-escalation study evaluating the safety, tolerability, and effect on serum iron of a single dose of LJPC-401 in patients at risk of iron overload due to conditions such as hereditary hemochromatosis (HH), thalassemia, and sickle cell disease (SCD). Fifteen patients were dosed at escalating dose levels ranging from 1 mg to 20 mg.
LJPC-401 was well tolerated, and there were no dose-limiting toxicities observed. Injection-site reactions were the most commonly reported adverse event. These were all mild or moderate in severity, self-limiting, and fully resolved. There were no significant changes in serum chemistries or hematology other than serum iron parameters.
A dose-dependent, statistically significant reduction in serum iron was observed (p=0.008 for dose response; not adjusted for multiple comparisons). At the 20 mg dose level, LJPC-401 reduced serum iron by an average of 58.1% from baseline to hour 8 (p=0.001; not adjusted for potential regression to the mean effect), and serum iron had not returned to baseline through day 7 (21.2% reduction from baseline to the end of day 7).
“The results from this study demonstrate a clear, dose-dependent effect
of LJPC-401 on serum iron, a clinically important measure,” said
Conference Call at
La Jolla will host a conference call and webcast on
LJPC-401 is La Jolla’s novel formulation of synthetic 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 diseases such as hereditary hemochromatosis (HH), beta thalassemia, sickle cell disease (SCD) and myelodysplastic syndrome (MDS). HH is a disease characterized by a genetic deficiency in hepcidin. HH is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, diabetes, arthritis and joint pain. Beta thalassemia, SCD and MDS are genetic diseases of the blood that can cause life-threatening anemia 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 document 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 and involve known and
unknown risks, uncertainties and other factors, which may cause actual
results to be materially different from these 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
La Jolla Pharmaceutical Company
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