La Jolla Pharmaceutical Company Announces Special Protocol Assessment For Planned Phase III Trial

La Jolla Pharmaceutical Company recently announced it has reached agreement with the US FDA on a Special Protocol Assesment (SPA) for its Phase III clinical trial of LJPC-501 for the treatment of catecholamine-resistant hypotension (CRH). In accordance with the SPA, the primary efficacy endpoint for this Phase III registration trial will be increase in blood pressure. La Jolla plans to initiate this multicenter, randomized, double-blind, placebo-controlled, Phase III clinical trial pursuant to the approved SPA in the first quarter of 2015.

“We are very pleased with the FDA’s agreement on our clinical trial design and planned analysis, which clearly defines what we believe to be a feasible path for the development and registration of LJPC-501.”

“We are very pleased with the FDA’s agreement on our clinical trial design and planned analysis, which clearly defines what we believe to be a feasible path for the development and registration of LJPC-501,” said George F. Tidmarsh, MD, PhD, President and Chief Executive Officer of La Jolla. “The prognosis for patients suffering from CRH is very poor, with less than 50% of these patients surviving 1 month from diagnosis. We believe that LJPC-501 has the potential to reverse hypotension and, therefore, provide a significant benefit to these patients.”

A Special Protocol Assessment is a written agreement between a sponsor and the US FDA on the design, execution, and analysis for a clinical trial that may form the basis of a new drug application, or NDA. Final marketing approval depends upon the efficacy results, the safety profile, and an evaluation of the risk/benefit of treatment demonstrated in the Phase III clinical program.

LJPC-501 is La Jolla’s proprietary formulation of angiotensin II. Angiotensin II, the major bioactive component of the renin-angiotensin system, serves as one of the body’s central regulators of blood pressure. La Jolla is developing LJPC-501 for the treatment of catecholamine-resistant hypotension, or CRH, which is an acute, life-threatening condition in which blood pressure drops to dangerously low levels and is poorly responsive to current treatments. Angiotensin II has been shown to raise blood pressure in a randomized, placebo-controlled clinical trial in CRH, as well as animal models of hypotension. In October 2014, La Jolla presented positive data from a preclinical study of LJPC-501 for the treatment of CRH. In February 2015, La Jolla reached agreement with the FDA on a Special Protocol Assessment, or SPA, for its Phase III clinical trial of LJPC-501 for the treatment of CRH, in which agreement was reached that blood pressure can be the primary endpoint for approval. La Jolla’s Phase III clinical trial of LJPC-501 for the treatment of CRH is expected to begin in the first quarter of 2015. La Jolla has submitted an Orphan Drug Designation application to the FDA for LJPC-501 for the treatment of CRH.

La Jolla is also developing LJPC-501 for hepatorenal syndrome, or HRS. HRS is a life-threatening form of progressive renal failure in patients with liver cirrhosis or fulminant liver failure. In these patients, the diseased liver secretes vasodilator substances (eg, 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. Studies have shown that LJPC-501 may improve renal function in patients with conditions similar to HRS. La Jolla is currently conducting a Phase I/II clinical trial of LJPC-501 in HRS. The Phase I/II clinical trial is currently enrolling patients.

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. For more information, visit www.ljpc.com.