La Jolla Pharmaceutical Company Announces Special Protocol Assessment for Planned Phase 3 Trial of LJPC-501 in Catecholamine-Resistant Hypotension
SAN DIEGO--(BUSINESS WIRE)--La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced that it has reached agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assesment (SPA) for its Phase 3 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 3 registration trial will be increase in blood pressure. La Jolla plans to initiate this multicenter, randomized, double-blind, placebo-controlled, Phase 3 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,” said George F. Tidmarsh, M.D., Ph.D., 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 one month from diagnosis. We believe that LJPC-501 has the potential to reverse hypotension and, therefore, provide a significant benefit to these patients.”
About Special Protocol Assessments
A Special Protocol Assessment is a written agreement between a sponsor and the U.S. Food and Drug Administration 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 3 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 3 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 3 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 (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. Studies have shown that LJPC-501 may improve renal function in patients with conditions similar to HRS. La Jolla is currently conducting a Phase 1/2 clinical trial of LJPC-501 in HRS. The Phase 1/2 clinical trial is currently enrolling patients.
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. The Company has four product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension and hepatorenal syndrome. GCS-100 is La Jolla’s first-in-class galectin-3 inhibitor for the potential treatment of chronic kidney disease. LJPC-1010, La Jolla’s second-generation galectin-3 inhibitor, is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis and other diseases characterized by tissue fibrosis. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload. For more information on La Jolla, please visit www.ljpc.com.
Forward-Looking Statement Safe Harbor
This document contains “forward-looking statements,” as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to the Company’s expectations regarding future events or its future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, any one of which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site http://www.sec.gov. These risks include, but are not limited to, risks relating to the future development of LJPC-501 for the treatment of CRH and the success of future development activities for this and other drug development programs sponsored by the Company; the ability of the Company to obtain orphan drug status for LJPC-501; the ability of the Company to use the planned Phase 3 study of LJPC-501 in CRH as a registration study; and the success of other potential indications for which this and other drug candidates may be developed. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.