La Jolla Pharmaceutical (NASDAQ:LJPC) reported results from a pre-specified interim analysis of its Phase 2 study of LJPC-401 in patients with hereditary hemochromatosis (HH).
HH is caused by a genetic mutation that results in deficiency or insensitivity of hepcidin, a hormone that regulates iron absorption and accumulation. There are currently no FDA-approved therapies for HH; the current standard treatment is blood removal via phlebotomy.
The interim analysis included 26 patients who have reached the end of the 16-week treatment period with LJPC-401, a formulation of synthetic human hepcidin. Patients reported a mean reduction in transferrin saturation of 42% compared to placebo-treated patients, who had a mean reduction of 6%.
“There have been no new treatment modalities introduced for patients with hereditary hemochromatosis in more than a decade,” said Dr. Jeff Vacirca, an investigator in the study.
“In light of the negative impact that repeated phlebotomy procedures have on patient quality-of-life, patients would welcome a pharmacologic treatment that they can self-administer and that addresses the underlying pathophysiology of the disease,” he added.
La Jolla expects to announce topline results of this study in the second half of 2019.