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La Jolla Pharmaceutical Company Announces Portfolio Reprioritization

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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 a reprioritization of its product development programs. As a result of this reprioritization, La Jolla will discontinue development of its polysaccharide-based galectin-3 inhibitors, GCS-100 and LJPC-1010. The reprioritization will allow La Jolla to reallocate resources to its other development candidates, including its lead product candidate LJPC-501, La Jolla’s proprietary formulation of angiotensin II that is currently the subject of a Phase 3 registration clinical trial in patients suffering from catecholamine-resistant hypotension, LJPC-401, La Jolla’s novel formulation of hepcidin for the potential treatment of conditions characterized by iron overload, and LJPC-30Sa and LJPC-30Sb, La Jolla’s recently announced next-generation gentamicin derivative product candidates for the potential treatment of serious bacterial infections and rare genetic disorders.

The portfolio reprioritization is the result of both the addition of the next-generation gentamicin derivative program and a recent interaction with the U.S. Food and Drug Administration (FDA) regarding La Jolla’s galectin-3 inhibitor program. In this interaction, the FDA indicated that the Company would be required to conduct additional chemical characterization of GCS-100 prior to further clinical development. GCS-100 and LJPC-1010 are complex polysaccharide mixtures that cannot be readily chemically characterized using conventional analytical methods, and the Company believes that the timeframe and ultimate success of developing analytical methods that would satisfy the FDA’s requirements are highly uncertain. There were no issues raised by the FDA related to patient safety or preclinical toxicology.

The Company will continue to treat and follow patients already enrolled in its Phase 2b study in diabetic patients with advanced chronic kidney disease, but will stop enrolling new patients. The Company will not proceed with a Phase 1 study of LJPC-1010 as previously planned. La Jolla will instead explore out-licensing opportunities for these product candidates.

“We are very enthusiastic about LJPC-501 and LJPC-401 and the unmet medical needs that they may fulfill, and we are excited to add our new next-generation gentamicin derivative program to our portfolio. These product candidates all benefit from strong scientific and clinical rationale, and today’s reprioritization will allow us to sharpen our focus on bringing them closer to patients,” stated George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “While it was a difficult decision to discontinue our galectin-3 inhibitor programs, we believe that this is the right decision for the Company and its stakeholders as we evaluate our portfolio opportunities and how best to allocate our resources. This reprioritization will free up significant financial resources, and we continue to project that our current cash resources will be sufficient to fund our operations, including the advancement of all three of these programs, through the end of 2016.”

Conference Call and Webcast

The Company will hold an investor conference call and webcast at 8:00 AM Eastern Time/5:00 AM Pacific Time on Friday, May 8, 2015. You can participate on the call by either dialing 877-359-9508 pin: 44727023 or click here for the webcast.

About LJPC-501

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 (CRH), which is an acute, life-threatening condition in which blood pressure drops to dangerously low levels in patients who respond poorly to current treatments. Angiotensin II has been shown to raise blood pressure in a randomized, placebo-controlled clinical trial in CRH, which was recently published in the journal Critical Care, as well as in animal models of hypotension. Preclinical pharmacology studies conducted by La Jolla have demonstrated that catecholamine resistance may be in part a result of reduced endogenous production of angiotensin II. La Jolla is currently enrolling patients into its ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial, which is a multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial of LJPC-501 in patients with CRH. The ATHOS 3 trial is being conducted under a Special Protocol Assessment with the U.S. Food and Drug Administration in which it was agreed that the primary efficacy endpoint is increase in blood pressure. Results from ATHOS 3 are expected by the end of 2016.

About LJPC-401

LJPC-401 is La Jolla’s novel formulation of hepcidin. Hepcidin is a naturally occurring peptide hormone that controls and regulates iron metabolism. By suppressing iron release and absorption, hepcidin prevents iron accumulation in tissues, such as the liver, heart and pancreas, where it can cause significant damage and even result in death. La Jolla is developing LJPC-401 for the treatment of conditions characterized by iron overload, such as hemochromatosis and beta thalassemia. Preclinical studies have shown that the administration of LJPC-401 results in reduced serum iron in all animal species tested. Animal toxicology studies have established the proposed doses for LJPC-401’s Phase 1 clinical trial, and these doses have translated into animal exposures that have shown to lower serum iron levels in all animal species tested. La Jolla expects to file an Investigational New Drug application (IND) with the U.S. Food and Drug Administration in mid-2015. LJPC-401 has been successfully manufactured under Good Manufacturing Practice (GMP) for use in La Jolla’s single, ascending-dose Phase 1 clinical trial, which is expected to commence in the second half of 2015. This proposed Phase 1 clinical trial will evaluate the effects of LJPC-401 on serum iron parameters, as well as safety and pharmacodynamics, in healthy volunteers, with results expected by the end of 2015.

About LJPC-30Sa and LJPC-30Sb

LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives. Despite kidney toxicity, gentamicin has become one of the most commonly prescribed hospital antibiotics. Gentamicin consists primarily of a mixture of four distinct but closely related chemical entities that may contribute differentially to the product’s toxicity profile. Gentamicin’s ability to induce a lack of fidelity in gene transcription, intrinsic to its antimicrobial mechanism of action, can also be leveraged in the correction of certain human genetic mutations that lead to rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. In spite of favorable short-term clinical proof-of-efficacy data in cystic fibrosis, development of gentamicin as a chronic treatment for these genetic diseases has been limited by its toxicity profile. LJPC-30Sa and LJPC-30Sb are purified components of the currently marketed gentamicin product that retain the biologic activity of gentamicin, yet appear to lack the traditional kidney toxicity associated with it. La Jolla is developing LJPC-30Sa and LJPC-30Sb for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. Following a pre-Investigational New Drug application (IND) meeting with the U.S. Food and Drug Administration, La Jolla has received guidance that it may proceed with its proposed Phase 1 clinical trial following the submission of an IND.

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 several 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. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of conditions characterized by iron overload, such as hemochromatosis and beta thalassemia. LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. 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 future events or the Company’s future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, 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 timing for the filing of an Investigational New Drug Application, commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for LJPC-501, LJPC-401, LJPC-30Sa and LJPC-30Sb; potential indications for which LJPC-501, LJPC-401, LJPC-30Sa and LJPC-30Sb may be developed; and any future success in out-licensing or partnering GCS-100 or LJPC-1010. 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.

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