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 –

SAN DIEGO, CA, USA I June 06, 2019 I La Jolla Pharmaceutical Company (Nasdaq: LJPC), a leader in the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced positive results from the pre-specified interim analysis of its Phase 2 study of LJPC-401 (synthetic human hepcidin) in patients with hereditary hemochromatosis (HH). The interim analysis of efficacy included 26 patients who have reached the end of the 16-week treatment period (the efficacy population: 12 LJPC-401-treated patients; 14 placebo-treated patients), and the interim analysis of safety included 60 randomized patients (the safety population: 29 LJPC-401-treated patients; 31 placebo-treated patients).

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 Jeff Vacirca, M.D., Chief of Clinical Research at New York Cancer & Blood Specialists and an Investigator in the Study. “There have been no new treatment modalities introduced for patients with hereditary hemochromatosis in more than a decade. 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.”

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.

About LJPC-401

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. The European Medicines Agency (EMA) Committee for Orphan Medicinal Products has designated LJPC‑401 as an orphan medicinal product for the treatment of BT intermedia and major and SCD.

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 FDA approved therapies for HH and the current standard treatment for HH is a blood removal procedure known as phlebotomy. Each phlebotomy procedure, which is usually conducted at a hospital, medical office or blood center, typically involves the removal of approximately a pint of blood. The required frequency of procedures varies by patient but often ranges from one to two times per week for an initial period after diagnosis and once every one to three months for life. Since most of the body’s iron is stored in red blood cells, chronic removal of blood can effectively lower iron levels if a phlebotomy regimen is adhered to. However, phlebotomy procedures may cause and may be associated with pain, bruising and scarring at the venous puncture site, joint pain, fatigue and dizziness during and following the procedure and disruption of daily activities. Furthermore, phlebotomy is not appropriate in patients with poor venous access, anemia or heart disease.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. GIAPREZA™ (angiotensin II), formerly known as LJPC-501, was approved by the U.S. Food and Drug Administration on December 21, 2017 as a vasoconstrictor indicated to increase blood pressure in adults with septic or other distributive shock. LJPC-0118 is La Jolla’s investigational product for the treatment of severe malaria. LJPC‑401 (synthetic human hepcidin), a clinical-stage investigational product, is being developed for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease, myelodysplastic syndrome and polycythemia vera. For more information on La Jolla, please visit www.ljpc.com.

SOURCE: La Jolla Pharmaceuticals

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