La Jolla Pharmaceutical has started dosing in its Phase 1 clinical trial of LJPC-401, its new formulation of hepcidin in patients at risk of iron overload due to conditions, such as hereditary hemochromatosis (HH), beta thalassemia and sickle cell disease (SCD).
Hepcidin is a naturally occurring peptide hormone that is the body's regulator of iron absorption and distribution and it 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 trial will evaluate the safety, tolerability and effect on serum iron parameters, and pharmacokinetics of LJPC-401 in these patients.
Preliminary results from the multicenter, open-label, dose-escalation clinical trial are expected to be reported by this year-end.
La Jolla president and chief executive officer George Tidmarsh said: "We are excited to be advancing LJPC-401 into its first clinical trial, and we are very grateful to the physicians and patients who have partnered with us on this important program.
"We believe that LJPC-401 presents a unique opportunity to potentially help patients suffering from the effects of iron overload by restoring normal or near-normal levels of hepcidin, the body's natural regulator of iron absorption and distribution."
In preclinical testing, LJPC-401 has been shown to be effective in reducing serum iron.
HH is a disease caused by a genetic deficiency in hepcidin production, resulting in excessive iron accumulation, while beta thalassemia and SCD are genetic diseases of blood cells that can cause life-threatening anemia and often require frequent and life-long blood transfusions.
The company develops new therapies intended to improve outcomes in patients suffering from life-threatening diseases.
LJPC-30Sa and LJPC-30Sb are the company's next-generation gentamicin derivatives designed to treat serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy.