La Jolla Pharmaceutical Company Announces Positive Results from Pre-Specified Interim Analysis of Phase 2 Study of LJPC-401 in Patients with Hereditary Hemochromatosis
- Treatment with LJPC-401 Resulted in Statistically Significant Reduction in Change in Transferrin Saturation (TSAT), the Primary Efficacy Endpoint of the Study, and Frequency of Phlebotomies, a Key Secondary Efficacy Endpoint of the Study -
The change in TSAT from baseline to the end of treatment (16 weeks), the primary efficacy endpoint of the study, was statistically significant: LJPC‑401‑treated patients had a mean reduction in TSAT of 42% compared to placebo-treated patients who had a mean reduction of 6% (p<0.0001).
The requirement for and frequency of phlebotomy procedures, a key secondary endpoint of the study, was statistically significant: LJPC-401-treated patients had 0.06 phlebotomies per month compared to placebo-treated patients who had 0.41 phlebotomies per month (p=0.003). There were 3 phlebotomies in 2 LJPC-401-treated patients and 24 phlebotomies in 9 placebo-treated patients.
LJPC-401 was well tolerated. The most frequent treatment-emergent adverse events (TEAEs) were injection site reactions (ISRs), which occurred in 79% of LJPC‑401-treated patients compared to 6% of placebo-treated patients. The ISRs were all mild or moderate in severity, and no ISRs resulted in treatment discontinuation. As of the interim analysis, there were no serious TEAEs reported.
“The robustness of this early readout supporting further development of LJPC-401 is very encouraging,” said
About the LJ401-HH01 Study
LJ401-HH01 is a multinational, multicenter, randomized, placebo-controlled, double-blind, Phase 2 study designed to evaluate the safety and efficacy of LJPC-401 as a treatment for hereditary hemochromatosis (HH). Approximately 60 patients have been randomized to receive weekly subcutaneous injections of either LJPC‑401 or placebo for 16 weeks. Targeted enrollment consisted primarily of patients in the maintenance phase of their HH treatment, where serum ferritin had already been lowered while transferrin saturation (TSAT) remained elevated. There was a subset of patients in the study in the induction phase of their HH treatment where both serum ferritin and TSAT were elevated.
The primary efficacy endpoint of the study is the change in TSAT, a standard measurement of iron levels in the body and one of the two key measurements used to detect iron overload, from baseline to end of treatment at week 16. Secondary efficacy endpoints include the requirement for and frequency of phlebotomy procedures during the study.
We expect to announce topline results of LJ401-HH01 in the second half of 2019.
LJPC-401, a clinical-stage investigational product, is La Jolla’s proprietary formulation of synthetic human 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 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.
About Hereditary Hemochromatosis
Hereditary hemochromatosis (HH) is the most common genetic disease in Caucasians. HH is a disease characterized by a genetic mutation that causes excessive iron absorption and accumulation due to hepcidin deficiency or insensitivity. Hepcidin is the body’s naturally occurring regulator of iron absorption and distribution. Without normal levels of hepcidin, excessive amounts of iron accumulate in the body. Symptoms of the disease include joint pain, abdominal pain, fatigue and weakness. If left untreated, HH can lead to liver cirrhosis, liver cancer, heart disease and/or failure and diabetes.
There are no
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Source: La Jolla Pharmaceutical Company