August 25th, 2014
La Jolla Pharmaceutical Company, a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced that the first patient has been enrolled in the Phase 1/2 clinical trial of LJPC-501 for the treatment of type 1 and type 2 hepatorenal syndrome (HRS). HRS is a life-threatening form of progressive renal failure in patients with liver cirrhosis or fulminant liver failure.
“We are very grateful to the investigator, the patient and the patient’s family,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “We believe that LJPC-501, which helps the kidneys balance body fluids and electrolytes, may improve kidney function in patients with HRS. For many patients with HRS, reduced kidney function is the major debilitating aspect of their illness.”
The clinical trial is an open-label, multi-center study of LJPC-501 in patients with type 1 and/or type 2 HRS.
The trial is designed to enroll up to 15 patients with type 1 or type 2 HRS. The primary endpoint is safety and tolerability. Secondary endpoints include determining the maximum tolerated dose and the effects of LJPC-501 on serum creatinine through 5 days of treatment. Patients will receive LJPC-501 at titrated doses, with a starting range from 1 to 100 ng/kg/min, by continuous infusion on days 1 through 5. Dose titrations will occur every 4 hours until a mean arterial pressure of 110 mmHg is reached, maximum urine output is achieved or a dose of 100 ng/kg/min is achieved. Dosing will then continue at such maximum dose through day 5.
About Hepatorenal Syndrome
Hepatorenal syndrome (HRS) is a life-threatening form of progressive renal failure in patients with liver cirrhosis. In these patients, the diseased liver secretes vasodilator substances (e.g., nitric oxide and prostaglandins) into the bloodstream that cause under-filling of blood vessels. This low blood pressure state causes a reduction in blood flow to the kidneys. As a means to restore systemic blood pressure, the kidneys induce both sodium and water retention, which contribute to ascites, a major complication associated with HRS.
LJPC-501, a proprietary formulation of angiotensin II, is a peptide agonist of the renin-angiotensin system that acts to stabilize blood pressure. La Jolla is developing LJPC-501 for the treatment of catecholamine-resistant hypotension (CRH), which is an acute, life-threatening condition in which blood pressure drops to dangerously low levels and is poorly responsive to current treatments. LJPC-501 has been shown to raise blood pressure in a randomized, placebo-controlled clinical trial in CRH, as well as animal models of hypotension. La Jolla plans to initiate a Phase 3 registration program with LJPC-501 in the treatment of CRH as the result of a recent meeting with the U.S. Food and Drug Administration (FDA) at which agreement was reached that blood pressure could serve as an appropriate primary endpoint for approval. Due to the estimated size of the patient population in the United States for this indication, La Jolla has filed an application for Orphan Drug status for LJPC-501. Studies have shown that LJPC-501 may also improve renal function in patients with hepatorenal syndrome (HRS).
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. LJPC-501, La Jolla’s lead product candidate, is a proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension and hepatorenal syndrome. GCS-100, La Jolla’s second product candidate, is a first-in-class inhibitor of galectin-3 for the potential treatment of chronic kidney disease. LJPC-1010, La Jolla’s third product candidate, is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis. LJPC-401, La Jolla’s fourth product candidate, is a natural peptide for the potential treatment of iron overload. For more information on La Jolla, please visit http://www.ljpc.com.