La Jolla Pharmaceutical Company Appoints Lakhmir S. Chawla, M.D., as Chief Medical Officer

La Jolla Pharmaceutical Company Appoints Lakhmir S. Chawla, M.D., as Chief Medical Officer

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 Lakhmir S. Chawla, M.D., will be joining the Company as Chief Medical Officer, effective July 1, 2015.

“Dr. Chawla brings to La Jolla a wealth of knowledge and experience, including very relevant expertise in the areas of critical care medicine and nephrology,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We are excited to welcome him to La Jolla’s executive team at this important stage of our growth and development.”

“I am delighted to be joining this highly innovative company,” added Dr. Chawla. “La Jolla has assembled an exceptional team of professionals, and I look forward to working with them in developing novel therapeutics for life-threatening diseases.”

Dr. Chawla is currently an Associate Professor of Medicine at the George Washington University, where he has dual appointments in the Department of Anesthesiology and Critical Care Medicine and in the Department of Medicine, Division of Renal Diseases and Hypertension. Dr. Chawla is also currently the Chief of the Division of Intensive Care Medicine at the Washington D.C. Veterans Affairs Medical Center. During his tenure at George Washington, Dr. Chawla was the designer and lead investigator of a pilot study called the ATHOS (Angiotensin II for the Treatment of High Output Shock) trial. Data from the ATHOS trial was published in the medical journal Critical Care during 2014 and demonstrated the utility of angiotensin II in patients with severe shock. These data were also used in support of the initiation of La Jolla’s ATHOS 3 trial, a Phase 3 clinical trial of LJPC-501, La Jolla’s proprietary formulation of angiotensin II, for the treatment of catecholamine-resistant hypotension, which was initiated in March 2015.

Dr. Chawla is an internationally renowned expert in the field of acute kidney injury (AKI) and is an active investigator in the fields of inflammation and AKI, AKI biomarkers, AKI risk prediction, chronic kidney disease caused by AKI and AKI therapeutics. In addition, Dr. Chawla is an active investigator in shock, inflammation and extracorporeal therapies, including: continuous renal replacement therapy, dialysis and albumin dialysis. Dr. Chawla is also the author of over 100 peer-reviewed publications and an Associate Editor for the Clinical Journal of the American Society of Nephrology.

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 conditions characterized by iron overload, such as hemochromatosis and beta thalassemia. 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, GCS-100, LJPC-1010 and LJPC-401; and potential indications for which LJPC-501, GCS-100, LJPC-1010 and LJPC-401 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.

Contacts

La Jolla Pharmaceutical Company

George F. Tidmarsh, M.D., Ph.D.

President & Chief Executive Officer

Phone: (858) 207-4264

Email: gtidmarsh@ljpc.com

and

Dennis M. Mulroy

Chief Financial Officer

Phone: (858) 433-6839

Email: dmulroy@ljpc.com

Source: La Jolla Pharmaceutical Company

View this news release online at:

http://www.businesswire.com/news/home/20150427005218/en

La Jolla Pharmaceutical Company Announces Additions to LJPC-401 Advisory Board

La Jolla Pharmaceutical Company Announces Additions to LJPC-401 Advisory Board

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 the addition of four new members to its advisory board for the development of LJPC-401, La Jolla’s novel formulation of hepcidin. The advisory board is composed of internationally renowned clinicians, highly respected academics and key opinion leaders in the fields of gastroenterology, hematology and oncology. The new members of the advisory board are Paul Adams, M.D., Victor Gordeuk, M.D., Ashutosh Lal, M.D., and Gordon McLaren, M.D.

La Jolla seeks the advisory board’s guidance on all aspects of LJPC-401’s therapeutic development, including unmet medical needs, new targets and paths, target validation, optimal indications, patient populations and trial designs. The advisory board further connects La Jolla to specific and relevant expertise that enhances La Jolla’s efforts to base its therapeutic programs on the best science and critical thinking.

“We are very pleased to bring these key thought leaders together to strengthen La Jolla’s advisory board for the development of LJPC-401,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “This advisory board provides strategic guidance to the LJPC-401 development program, in which we expect to start a Phase 1 clinical trial later this year.”

Paul Adams, M.D., is a Professor of Medicine and Chief of Gastroenterology at the University of Western Ontario, in London, Ontario. Dr. Adams has been working in the field of hemochromatosis and iron overload since 1977. He graduated from medical school at Queens University (Kingston, Ontario) in 1980 and completed internal medicine training in Toronto and New York (Mount Sinai). He completed a gastroenterology fellowship at the University of California at San Francisco (UCSF) and studied hemochromatosis at the Liver Research Unit of the Royal Brisbane Hospital in Australia and the Liver Research Unit in Rennes, France.

Victor Gordeuk, M.D., is a Professor of Medicine in the Division of Hematology and Oncology at the University of Illinois. Dr. Gordeuk specializes in treatment of patients with sickle cell disease and in conducting research to understand new ways to prevent complications of this condition. He has expertise in treating disorders of iron metabolism, including iron overload and iron deficiency. He also has a research and treatment interest in patients with congenital or unexplained polycythemia. He graduated from the University of Pittsburgh School of Medicine, completed an internship and residency at York Hospital and a fellowship at Case Western Reserve School of Medicine.

Ashutosh Lal, M.D., is a Pediatric Hematologist/Oncologist and the Director of the thalassemia program at the University of California, San Francisco, Benioff Children’s Hospital in Oakland, CA. Dr. Lal’s research areas of interest are in the development of intensive chelation regimens for transfusional iron overload, iron-induced cellular injury, the natural history of hemoglobin H disease and micronutrient inadequacy in hemoglobinopathies. He graduated from Government Medical College in Punjab, India, completed training at the Postgraduate Institute of Medical Education and Research in Chandigarh, India, a residency in pediatrics at the University of Illinois, Chicago, as well as a fellowship in pediatric hematology/oncology at University of California, San Francisco, Benioff Children’s Hospital in Oakland, CA.

Gordon McLaren, M.D., is a Professor of Medicine in the Division of Hematology/Oncology at the University of California, Irvine. Dr. McLaren’s research interests include disorders of iron metabolism, focusing on the areas of hemochromatosis and other forms of iron overload, control of intestinal iron absorption and regulation of cellular iron-binding proteins. He graduated from medical school at Stanford University in 1970, completed an internship in internal medicine at Mary Imogene Bassett Hospital in Cooperstown, NY, a residency at the University Hospitals of Cleveland in internal medicine and hematology, and a fellowship in hematology/oncology.

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, 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 increasing hepcidin, either via synthetic hepcidin injection or genetic induction, results in reduced iron overload in organs. La Jolla expects to file an IND and commence a Phase 1 clinical trial of LJPC-401 in the second half of 2015.

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 conditions characterized by iron overload, such as hemochromatosis and beta thalassemia. 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 successful future development of LJPC-401 and anticipated contributions of the LJPC-401 advisory board. 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.

Contacts

La Jolla Pharmaceutical Company

George F. Tidmarsh, M.D., Ph.D.

President & Chief Executive Officer

(858) 207-4264

gtidmarsh@ljpc.com

and

Dennis M. Mulroy

Chief Financial Officer

(858) 433-6839

dmulroy@ljpc.com

Source: La Jolla Pharmaceutical Company

View this news release online at:

http://www.businesswire.com/news/home/20150422005223/en

La Jolla Pharmaceutical Company Announces Initiation of Phase 3 Clinical Trial of LJPC-501 in Catecholamine-Resistant Hypotension

La Jolla Pharmaceutical Company Announces Initiation of Phase 3 Clinical 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 initiated its ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial, a Phase 3 clinical trial of LJPC-501 for the treatment of catecholamine-resistant hypotension (CRH). In February 2015, La Jolla reached agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA), in which it was agreed that the primary efficacy endpoint for the trial would be increase in blood pressure. CRH is an acute, life-threatening condition in which blood pressure drops to dangerously low levels in patients who respond poorly to current treatments.

In accordance with the SPA, the ATHOS 3 trial is a multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial of LJPC-501 in patients with CRH. The ATHOS 3 trial is designed to enroll approximately 315 patients. Patients will be randomized 1:1 to receive either LJPC-501 plus standard-of-care vasopressors or placebo plus standard-of-care vasopressors. Randomized patients will receive their assigned treatment via continuous IV infusion for up to 7 days.

The primary efficacy endpoint of the ATHOS 3 trial is to compare the change in mean arterial pressure between the two groups in the trial. Secondary endpoints include comparison of changes in Sequential Organ Failure Assessment scores and the safety and tolerability of LJPC-501 in patients with CRH. La Jolla has reached agreement with the FDA that an adequately sized Phase 3 trial comprised of 200-300 patients would provide a sufficient safety database to support FDA review and consideration for marketing approval.

“The prognosis for patients suffering from CRH is very poor, and current treatment options are limited. We believe that LJPC-501 has the potential to reverse hypotension and, therefore, provide a significant benefit to these difficult-to-treat patients,” said Lakhmir Chawla, M.D., Associate Professor of Medicine, Department of Anesthesiology and Critical Care Medicine, George Washington University.

“We are pleased to have advanced LJPC-501 into a Phase 3 registration clinical trial,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We would like to thank the FDA and our clinical collaborators for helping us to advance this medically important program so expeditiously.”

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, 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. 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.

La Jolla is also developing LJPC-501 for hepatorenal syndrome (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 enrolling patients into a Phase 1/2 clinical trial of LJPC-501 in HRS.

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 (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.

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 conditions characterized by iron overload, such as hemochromatosis and beta thalassemia. 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 and potential indications for which these 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.

Contacts
La Jolla Pharmaceutical Company
George F. Tidmarsh, M.D., Ph.D.
President & Chief Executive Officer
(858) 207-4264
gtidmarsh@ljpc.com
or
Chester S. Zygmont, III
Senior Director of Finance
(858) 207-4262
czygmont@ljpc.com

Source: La Jolla Pharmaceutical Company

Powered by Business Wire
View this news release online at:
http://www.businesswire.com/news/home/20150324005346/en

La Jolla Pharmaceutical Company Announces Initiation of Phase 2b Clinical Trial of GCS-100 in Advanced Chronic Kidney Disease

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 initiated its Phase 2b clinical trial of GCS-100 for the treatment of advanced chronic kidney disease (CKD) caused by diabetes. CKD is a condition characterized by a gradual loss of kidney function over time that may eventually lead to kidney failure, requiring dialysis or a kidney transplant to maintain life.

The Phase 2b clinical trial is a double-blind, multi-center, placebo-controlled, randomized trial of GCS-100 in diabetic patients with Stage 3b or 4 CKD. The clinical trial is designed to enroll approximately 375 patients. Patients will be randomized 1:1:1:1 to receive fixed doses of GCS-100 (1, 3 or 9 mg) or placebo. Randomized patients will receive their assigned treatment via IV injection once a week for 8 weeks and then once every other week for an additional 16 weeks.

The primary endpoint of this Phase 2b clinical trial is to compare the change in kidney function, as measured by estimated glomerular filtration rate (eGFR), from baseline to week 26, which is 2 weeks after the last injection, between patients receiving GCS-100 or placebo. Secondary efficacy endpoints include a responder analysis based on pre-specified percentage changes in eGFR and an analysis on progression to renal replacement therapy. Other secondary endpoints are focused on the long-term safety and tolerability of GCS-100, including an evaluation of the incidence of major cardiac events.

“Chronic kidney disease is an enormous and growing medical problem worldwide. A disease-modifying agent that could slow and potentially reverse the tissue fibrosis that is a hallmark of this disease would be a welcome advance in the field and could have a major impact on patients’ lives,” stated the trial’s principal investigator, Pablo E. Pergola, M.D., Ph.D., Director of the Clinical Advancement Center subsidiary of Renal Associates P.A. and Clinical Associate Professor of Medicine at the University of Texas Health Science Center at San Antonio. “Our experience with GCS-100 in previous trials was very encouraging, and my patients tolerated the therapy well.”

“We are excited to be advancing GCS-100 into the next phase of development,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “This Phase 2b trial will build on the knowledge we gained from our recently completed Phase 2 trial, in which treatment with GCS-100 resulted in a statistically significant improvement in kidney function.”

About GCS-100

GCS-100, La Jolla’s first-in-class galectin-3 inhibitor, is a complex polysaccharide derived from pectin that binds to, and blocks the activity of, the pro-fibrotic mediator galectin-3. Over-expression of galectin-3 has been implicated in a number of human diseases characterized by progressive tissue fibrosis, such as chronic kidney disease (CKD). La Jolla recently completed a multicenter, randomized, placebo-controlled, Phase 2 clinical trial in advanced CKD patients, in which treatment with GCS-100 resulted in a statistically significant improvement in kidney function compared to placebo.

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 conditions characterized by iron overload, such as hemochromatosis and beta thalassemia. 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 GCS-100 for the treatment of advanced CKD and the success of future development activities for this and other drug development programs sponsored by the Company and potential indications for which these 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.

Contacts

La Jolla Pharmaceutical Company

George F. Tidmarsh, M.D., Ph.D.

President & Chief Executive Officer

(858) 207-4264

gtidmarsh@ljpc.com

or

Chester S. Zygmont, III

Senior Director of Finance

(858) 207-4262

czygmont@ljpc.com

La Jolla Pharmaceutical Company Announces Fourth Quarter and Full Year 2014 Financial Results and Corporate Highlights

SAN DIEGO, CA – March 16, 2015 – 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 reported fourth quarter and full year 2014 financial results and highlighted recent corporate progress and near-term milestones.

Recent Corporate Highlights

  • In February 2015, La Jolla reached agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for its planned Phase 3 clinical trial of LJPC-501, La Jolla’s propriety formulation of angiotensin II, for the treatment of catecholamine-resistant hypotension (CRH), in which the agreed-upon primary efficacy endpoint will be increase in blood pressure.
  • In December 2014, La Jolla entered into an exclusive worldwide license agreement with the George Washington University for intellectual property rights covering the use of angiotensin II (LJPC-501) for the therapeutic treatment of patients with hypotension and shock.
  • In November 2014, La Jolla presented positive results from its randomized, placebo-controlled, Phase 2 clinical trial of GCS-100, La Jolla’s first-in-class galectin-3 inhibitor, in advanced chronic kidney disease (CKD) patients, in which GCS-100 met its primary efficacy endpoint of a statistically significant improvement in kidney function, at the American Society of Nephrology’s Kidney Week Annual Meeting.
  • In October 2014, La Jolla presented positive data from a preclinical study of LJPC-501 for the treatment of CRH.

 

“2014 was a very exciting year for La Jolla, highlighted by the announcement of our LJPC-501 Phase 3 registration program, the positive results from our Phase 2 clinical trial of GCS-100 in advanced CKD patients and the close of our financing in July,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “2015 is off to a great start as well, highlighted by the FDA’s agreement on our Phase 3 clinical trial design and planned analysis for LJPC-501. We look forward to the start of our Phase 3 trial of LJPC-501 in CRH and our Phase 2b trial of GCS-100 in advanced CKD, as well as the advancement of our other programs.”

 

Near-Term Corporate Milestones

  • In the first quarter of 2015, La Jolla plans to initiate a multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial of LJPC-501 in CRH, in accordance with its recently obtained SPA.
  • In the first quarter of 2015, La Jolla plans to initiate a multicenter, randomized, double-blind, placebo-controlled, Phase 2b clinical trial of GCS-100 in diabetic patients with Stage 3b or 4 CKD.
  • In the second quarter of 2015, La Jolla plans to file an Investigational New Drug Application (IND) with the FDA and initiate a Phase 1 clinical trial of LJPC-1010, La Jolla’s second-generation galectin-3 inhibitor.
  • In the second half of 2015, La Jolla plans to file an IND with the FDA and initiate a Phase 1 clinical trial of LJPC-401, La Jolla’s novel formulation of hepcidin.

 

Results of Operations

At December 31, 2014, La Jolla had $48.6 million in cash, compared to $8.6 million at December 31, 2013. The significant increase in cash is due to the completion of an underwritten public offering of common stock in July 2014, with net proceeds to La Jolla of approximately $53.1 million.

La Jolla’s cash used in operating activities for the three months ended December 31, 2014 was $5.4 million, compared to $2.0 million for the same period in 2013. Cash used in operating activities for the year ended December 31, 2014 was $12.9 million, compared to $4.7 million for the same period in 2013.

La Jolla’s comprehensive net loss attributable to common shareholders for the three months ended December 31, 2014 was $6.8 million, or $0.45 per share, compared to a comprehensive net loss attributable to common shareholders of $6.4 million, or $1.45 per share, for the same period in 2013. Comprehensive net loss attributable to common shareholders for the year ended December 31, 2014 was $21.3 million, or $2.00 per share, compared to a comprehensive net loss attributable to common shareholders of $18.7 million, or $12.16 per share, for the same period in 2013.

The increase in cash used in operating activities and comprehensive net loss attributable to common shareholders was primarily due to increases in research and development expenses related to the Phase 2 clinical trial of GCS-100 in advanced CKD, the Phase 1/2 clinical trial of LJPC-501 in hepatorenal syndrome (HRS), preclinical work on LJPC-1010 and LJPC-401 and intellectual property in-licensing costs related to LJPC-501 and LJPC-401.

 

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. We have four product candidates in development. LJPC-501 is our proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension and hepatorenal syndrome. GCS-100 is our first-in-class galectin-3 inhibitor for the potential treatment of chronic kidney disease. LJPC-1010, our 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 our novel formulation of hepcidin for the potential treatment conditions characterized by iron overload, such as hemochromatosis and beta thalassemia. For more information on La Jolla, please visit http://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 IND, commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for LJPC-501, GCS-100, LJPC-1010 and LJPC-401; and potential indications for which LJPC-501, GCS-100, LJPC-1010 and LJPC-401 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.

 

LJPC Statement of Operations and Balance Sheet

 

Contacts 

George F. Tidmarsh, M.D., Ph.D.

President & Chief Executive Officer

La Jolla Pharmaceutical Company

Phone: (858) 207-4264

Email: GTidmarsh@ljpc.com

 

or

 

Chester S. Zygmont, III

Senior Director of Finance

La Jolla Pharmaceutical Company

Phone: (858) 207-4262

Email: czygmont@ljpc.com

 

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.

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, 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.

Contacts

La Jolla Pharmaceutical Company

George F. Tidmarsh, M.D., Ph.D.

President & Chief Executive Officer

(858) 207-4264

gtidmarsh@ljpc.com

or

Chester S. Zygmont, III

Senior Director of Finance

(858) 207-4262

czygmont@ljpc.com

Source: La Jolla Pharmaceutical Company

View this news release online at:

http://www.businesswire.com/news/home/20150209005213/en

La Jolla Pharmaceutical Company to Provide Corporate Overview at 17th Annual BIO CEO & Investor Conference

SAN DIEGO, CA – February 4, 2015La 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 George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer, will provide a corporate overview at the 17th Annual BIO CEO & Investor Conference taking place February 9-10 in New York City.

 

17th Annual BIO CEO & Investor Conference Presentation Details

 

Date:               Monday, February 9, 2015

Time:               5:30 pm Eastern Time

Location:         Louis XVI Room @ The Waldorf Astoria New York

Webcast:         LJPC Webcast Link

 

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.

 

Company Contact

 

George F. Tidmarsh, M.D., Ph.D.

President & Chief Executive Officer

La Jolla Pharmaceutical Company

Phone: (858) 207-4264

Email: gtidmarsh@ljpc.com

 

and

 

Chester S. Zygmont, III

Senior Director of Finance

La Jolla Pharmaceutical Company

Phone: (858) 207-4262

Email: czygmont@ljpc.com

La Jolla Pharmaceutical Company and the George Washington University Announce Exclusive Worldwide License Agreement

Dec. 9, 2014 21:01 UTC

License Covers Intellectual Property Relating to LJPC-501

SAN DIEGO & WASHINGTON–(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, and the George Washington University (GW), today announced that La Jolla and GW have entered into an exclusive worldwide license agreement for GW intellectual property rights covering the use of angiotensin II for the therapeutic treatment of patients with hypotension and shock. La Jolla plans to initiate a phase 3 registration program of LJPC-501, La Jolla’s proprietary formulation of angiotensin II, in catecholamine-resistant hypotension (CRH) in the first quarter of 2015.

“We are very pleased to gain access to this additional intellectual property covering LJPC-501,” said George F. Tidmarsh, M.D., Ph.D., President and CEO 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.”

“Moving this important innovation from GW’s School of Medicine directly to a Phase 3 clinical trial means that doctors treating hypotension and shock could have better options relatively soon,” said Steven Kubisen, Director of the GW Office of Technology Transfer. “La Jolla’s expertise with LJPC-501 makes this partnership well-positioned for success.”

“The discovery of novel therapies to help patients in need is at the core of the mission of the GW School of Medicine and Health Sciences. With exceptional research, coupled with La Jolla’s development expertise, we aim to bring better options to patients who face life-threatening risks associated with hypotension,” said Robert Miller, Ph.D., GW’s Senior Associate Dean for Research, Vivian Gill Distinguished Research Professor and Professor of Anatomy and Regenerative Biology.

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of angiotensin II. Angiotensin II, the major bioactive product 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 in the treatment of CRH. In June 2014, La Jolla had a meeting with the U.S. Food and Drug Administration, or FDA, in which agreement was reached that blood pressure can be the primary endpoint for approval in CRH. As a result of this meeting, La Jolla plans to initiate a Phase 3 registration clinical trial of LJPC-501 in CRH in the first quarter of 2015. La Jolla believes CRH to be an orphan indication, and, therefore, in July 2014, La Jolla submitted an Orphan Drug Designation application to the FDA for LJPC-501 in 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, and, in August 2014, La Jolla enrolled its first patient in this trial. 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 (CRH) and hepatorenal syndrome (HRS). GCS-100 is La Jolla’s first-in-class galectin-3 inhibitor for the potential treatment of chronic kidney disease (CKD). 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 (NASH) 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.

About the George Washington University

In the heart of the nation’s capital, with additional programs in Virginia, the George Washington University, or GW, was created by an Act of Congress in 1821. Today, GW is the largest institution of higher education in the District of Columbia. The university offers comprehensive programs of undergraduate and graduate liberal arts study, as well as degree programs in medicine, public health, law, engineering, education, business and international affairs. Each year, GW enrolls a diverse population of undergraduate, graduate and professional students from all 50 states, the District of Columbia and more than 130 countries.

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 and uncertainties relating to: the timing for the commencement of clinical studies and the anticipated timing for completion of such studies; whether patent applications licensed from the George Washington University will result in issued patents and whether such patents, if issued, would cover LJPC-501; the success of future development activities for LJPC-501, GCS-100, LJPC-1010 and LJPC-401; estimated market sizes and the ability to successfully receive Orphan Drug designation for LJPC-501; and potential indications for which LJPC-501, GCS-100, LJPC-1010 and LJPC-401 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.

Contacts

La Jolla Pharmaceutical Company
George F. Tidmarsh, M.D., Ph.D.
President & Chief Executive Officer
(858) 207-4264
gtidmarsh@ljpc.com
or
Chester S. Zygmont, III
Senior Director of Finance
(858) 207-4262
czygmont@ljpc.com
or
The George Washington University
Emily Grebenstein
Media Relations Specialist
(202) 994-3087
emgreb@gwu.edu

Source: La Jolla Pharmaceutical Company

Results from Phase 2 Study of GCS-100 in Chronic Kidney Disease Being Presented at American Society of Nephrology Kidney Week

Primary Endpoint Met; Statistically Significant Improvement in Kidney Function Observed
Improvement More Pronounced in Diabetic Patients
Results Reproduced in Extension Study

 

PHILADELPHIA, PA – November 12, 2014La 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 detailed results from its Phase 2 study of GCS-100 in chronic kidney disease (CKD) patients are being presented at a poster session (Glomerular and Tubulointerstitial Disease: Clinical Trials and Outcomes, November 13, 10:00 am – 12:00 pm Eastern Standard Time, Poster TH-PO460) at the American Society of Nephrology’s (ASN) Kidney Week Annual Meeting. GCS-100, which blocks the activity of the pro-fibrotic mediator galectin-3, has the potential to be a first-in-class disease-modifying agent for the treatment of diseases characterized by progressive tissue fibrosis, such as CKD.

 

This multicenter, randomized, blinded, placebo-controlled, Phase 2 study in 121 advanced CKD patients met its primary efficacy endpoint of a statistically significant improvement in kidney function. Specifically, GCS-100, at a dose of 1.5 mg/m2, led to a statistically significant (p=0.045) improvement in estimated glomerular filtration rate (eGFR) versus placebo after 8 weeks of dosing. This improvement, compared to placebo, was maintained 5 weeks following the completion of dosing (p=0.07). No statistically significant improvement in eGFR was observed in the 30 mg/m2 dose group. The lack of consistent response in the 30 mg/m2 group may be due to off-target drug effects, as this dose is 1,400-fold in excess, on a molar basis, versus known circulating galectin-3 levels. Off-target effects may include antagonizing other galectins like galectin-9, which has opposing biological effects to galectin-3.

 

GCS-100 CKD Phase 2 Results – All Patients
Dose Group Placebo(n=40) 1.5 mg/m2(n=41) 30 mg/m2(n=36)
Change in eGFR at Week 8 (mL/min/1.73m2) -0.6 1.3 0.1
P Value (vs. Placebo)   0.045 NS
Change in eGFR at Week 12 (mL/min/1.73m2) -1.5 0.2 0.0
P Value (vs. Placebo)   0.07 NS

 

GCS-100’s effect on eGFR was more pronounced (p=0.029) in the prospectively defined subset of patients with diabetic etiology. Analysis of this subset was predefined based on the observation that galectin-3 is elevated in the kidneys of diabetic CKD patients, and the level of galectin-3 correlates with proteinuria (a marker of kidney health) in these patients.

 

GCS-100 CKD Phase 2 Results – Patients with Diabetes
Dose Group Placebo(n=29) 1.5 mg/m2(n=24) 30 mg/m2(n=26)
Change in eGFR at Week 8 (mL/min/1.73m2) -0.5 2.3 -0.3
P Value (vs. Placebo)   0.029 NS

 

GCS-100 was well-tolerated. There were no serious adverse events, no Grade 3/4 adverse events and no early study discontinuations in the 1.5 mg/m2 group. There was no observed effect on blood pressure in any dose group.

 

“We are highly encouraged by these results,” stated Pablo E. Pergola, M.D., Ph.D., Director of the Clinical Advancement Center subsidiary of Renal Associates P.A. and Clinical Associate Professor of Medicine at the University of Texas Health Science Center at San Antonio. “The steady improvement in eGFR over time that was maintained five weeks following the last dose and the lack of a hemodynamic effect both lend support to GCS-100’s novel, anti-fibrotic mechanism. Chronic kidney disease remains a major medical problem, with tissue fibrosis at its core.”

 

An extension study is currently being conducted in which patients from the Phase 2 study were re-randomized to receive either 1.5 or 30 mg/m2 of GCS-100 (complete data available through week 16). Of the 93 patients enrolled in total, 33 patients had previously received placebo in the Phase 2 study before being treated with GCS-100 in the extension study. This group, which represents a set of patients receiving GCS-100 for the first time, was analyzed for efficacy. Consistent with the blinded Phase 2 results, the 1.5 mg/m2 group experienced a significant improvement in eGFR. This is the case when comparing these patients’ response to both: (1) the response in the parallel randomized group receiving 30 mg/m2 (p=0.04); and (2) the previous response to placebo in the blinded Phase 2 study for placebo-treated patients enrolled in the extension study (p=0.02).

 

 

GCS-100 Extension Study Results – Phase 2 Placebo Patients
Dose Group Placebo*(n=33) 1.5 mg/m2(n=20) 30 mg/m2(n=13)
Change in eGFR at Week 16 (mL/min/1.73m2) -2.0 1.3 -1.8
P Value (vs. Placebo)   0.02 NS
P Value (vs. 30 mg/m2 Dose Group)   0.04

 

*Previous response to placebo in the blinded Phase 2 study for placebo-treated patients enrolled in the extension study (baseline to week 12)

 

“We want to thank all of the patients and investigators who contributed to making this study a success,” said George Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We look forward to advancing GCS-100 to the next phase of development in chronic kidney disease, and to bringing this potentially important therapy one step closer to the many patients suffering from this terrible disease.”

 

About GCS-100

 

GCS-100, La Jolla’s first-in-class galectin-3 inhibitor, is a complex polysaccharide derived from pectin that binds to, and blocks the activity of, the pro-fibrotic mediator galectin-3, a type of galectin. Over-expression of galectin-3 has been implicated in a number of human diseases characterized by progressive tissue fibrosis, such as chronic kidney disease (CKD). La Jolla is developing GCS-100 for the treatment of CKD. The Company recently completed a multicenter, randomized, placebo-controlled, Phase 2 study in CKD patients in which treatment with GCS-100 resulted in a statistically significant improvement in kidney function compared to placebo.

 

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 (CRH) and hepatorenal syndrome (HRS). GCS-100 is La Jolla’s first-in-class galectin-3 inhibitor for the potential treatment of chronic kidney disease (CKD). 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 (NASH) 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 http://www.ljpc.com.

 

About the American Society of Nephrology and Kidney Week

 

The American Society of Nephrology (ASN) leads the fight against kidney disease by educating health professionals, sharing new knowledge, advancing research and advocating the highest quality care for patients. Kidney Week is the world’s premier nephrology meeting, providing participants exciting and challenging opportunities to exchange knowledge, learn the latest scientific and medical advances, and listen to engaging and provocative discussions with leading experts in the field.

 

Forward-Looking Statement Safe Harbor

 

This document and the poster presentation referred to herein contain “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 the Company’s 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 GCS-100 for the treatment of CKD and the success of future development activities for this and other drug development programs sponsored by the Company. 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.

 


 

Company Contact

 

George F. Tidmarsh, M.D., Ph.D.

President & Chief Executive Officer

La Jolla Pharmaceutical Company

Phone: (858) 207-4264

Email: GTidmarsh@ljpc.com

 

and

 

Chester S. Zygmont, III

Senior Director of Finance

La Jolla Pharmaceutical Company

Phone: (858) 207-4262

Email: czygmont@ljpc.com

La Jolla Pharmaceutical Company Announces Third Quarter and Year-to-Date 2014 Financial Results and Corporate Highlights

SAN DIEGO, CA – November 12, 2014 – 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 reported third quarter and year-to-date 2014 financial results and highlighted recent corporate progress and near-term milestones.

 

Recent Corporate Highlights

  • On November 12, 2014, La Jolla announced that it is presenting detailed results from its multicenter, randomized, placebo-controlled, Phase 2 study of GCS-100, La Jolla’s first-in-class galectin-3 inhibitor, in advanced chronic kidney disease (CKD) patients, in which GCS-100 met its primary efficacy endpoint of a statistically significant improvement in kidney function, at a poster session at the American Society of Nephrology’s (ASN) Kidney Week Annual Meeting.
  • On October 14, 2014, La Jolla presented positive data from a preclinical study of LJPC-501, La Jolla’s proprietary formulation of angiotensin II, in the treatment of catecholamine-resistant hypotension (CRH).
  • On August 25, 2014, La Jolla announced that the first patient had been enrolled in the Phase 1/2 clinical trial of LJPC-501 for the treatment of type 1 and type 2 hepatorenal syndrome (HRS).
  • On July 28, 2014, La Jolla closed an underwritten public offering of approximately 5.4 million shares of common stock at a public offering price of $10.50 per share. The Company received total net proceeds of approximately $53.1 million, providing sufficient capital to fund operations through 2016.
  • On July 15, 2014, La Jolla announced positive data from a preclinical study of LJPC-1010, La Jolla’s second-generation galectin-3 inhibitor, in the treatment of nonalcoholic steatohepatitis (NASH).
  • On July 8, 2014, La Jolla announced that it plans to initiate a Phase 3 registration program with LJPC-501 in the treatment of CRH, as a result of a meeting between La Jolla and the U.S. Food and Drug Administration (FDA) at which agreement was reached that blood pressure is an appropriate primary endpoint for approval.

 

“We have continued to have a very exciting year so far, highlighted by the announcement of our plans for our LJPC-501 Phase 3 registration program, the positive detailed results from our Phase 2 clinical trial of GCS-100 in advanced CKD and the close of our recent financing,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “During the remainder of 2014, we look forward to moving our new LJPC-501 CRH Phase 3 program forward, continuing the progress of our GCS-100 CKD program and advancing our other new and exciting programs.”

 

 

Near-Term Corporate Milestones

  • In the first quarter of 2015, La Jolla plans to initiate a Phase 3 registration program of LJPC-501 in CRH.
  • In the first quarter of 2015, La Jolla plans to initiate a large, multicenter, randomized, placebo-controlled, Phase 2b clinical trial of GCS-100 in CKD.
  • In the first quarter of 2015, La Jolla plans to file an Investigational New Drug Application (IND) with the FDA and initiate a Phase 1 clinical trial of LJPC-1010.
  • In 2015, La Jolla plans to file an IND and commence a Phase 1 clinical trial of LJPC-401, La Jolla’s novel formulation of hepcidin, in iron overload.

 

Results of Operations

 

At September 30, 2014, La Jolla had $54.1 million in cash, as compared to $8.6 million of cash at December 31, 2013. The significant increase in cash is due to the close of an underwritten public offering of common stock in July of 2014, with net proceeds to La Jolla of approximately $53.1 million.

 

La Jolla’s cash used in operating activities for the nine months ended September 30, 2014 was $7.5 million, compared to $2.7 million for the same period in 2013.

 

La Jolla’s comprehensive net loss attributable to common shareholders for the three and nine months ended September 30, 2014 was $5.1 million and $14.5 million, or $0.37 per share and $1.58 per share, respectively, compared to a comprehensive net loss attributable to common shareholders of $4.5 million and $12.3 million, or $5.45 per share and $21.35 per share, respectively, for the same periods in 2013.

 

The increase in cash used in operating activities and comprehensive net loss attributable to common shareholders was primarily due to increases in research and development expenses related to the Phase 2 clinical trial of GCS-100 in advanced CKD, the Phase 1/2 clinical trial of LJPC-501 in HRS and preclinical work on LJPC-1010 and LJPC-401.

 

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 (CRH) and hepatorenal syndrome (HRS). GCS-100 is La Jolla’s first-in-class galectin-3 inhibitor for the potential treatment of chronic kidney disease (CKD). 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 (NASH) 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

http://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 commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for LJPC-501, GCS-100, LJPC-1010 and LJPC-401; estimated market sizes and the ability to successfully receive Orphan Drug designation for LJPC-501; and potential indications for which LJPC-501, GCS-100, LJPC-1010 and LJPC-401 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.

 

 

(financial tables follow)

 

LA JOLLA PHARMACEUTICAL COMPANY

Unaudited Condensed Statements of Operations and Comprehensive Loss

(in thousands, except per-share amounts)

 

 

Three Months Ended
September 30,
Nine Months Ended
September 30,
2014 2013 2014 2013
Expenses:
Research and development $ 2,625 $ 948 $ 6,218 $ 2,303
General and administrative 2,436 3,225 8,259 9,238
Total expenses 5,061 4,173 14,477 11,541
Loss from operations (5,061) (4,173) (14,477) (11,541)
Other income:
Other income, net 9 1 13 3
Net loss and comprehensive loss (5,052) (4,172) (14,464) (11,538)
Convertible preferred stock dividends earned (337) (801)
Net loss attributable to common shareholders $ (5,052) $ (4,509) $ (14,464) $ (12,339)
Basic and diluted net loss per share $ (0.37) $ (5.45) $ (1.58) $ (21.35)
Shares used in computing basic and diluted net loss per share 13,646 827 9,131 578

 

 

LA JOLLA PHARMACEUTICAL COMPANY

Condensed Balance Sheets

(in thousands, except share and per-share amounts)

 

 

September 30,
2014
December 31,
2013
(Unaudited)
ASSETS:
Current assets:
Cash and cash equivalents $ 54,131 $ 8,629
Restricted cash 37 37
Prepaid expenses and other current assets 153 43
Total current assets 54,321 8,709
Equipment and furnishings, net 114 38
Total assets $ 54,435 $ 8,747
LIABILITIES AND SHAREHOLDERS’ EQUITY:
Current liabilities:
Accounts payable $ 683 $ 834
Accrued expenses 900 187
Accrued payroll and related expenses 32 73
Total current liabilities 1,615 1,094
Shareholders’ equity:
Common Stock, $0.0001 par value; 100,000,000 and 12,000,000,000 shares authorized, and 15,225,980 and 4,404,407 shares issued and outstanding at September 30, 2014 and December 31, 2013, respectively 4 4
Series C-12 Convertible Preferred Stock, $0.0001 par value; 11,000 shares authorized, 3,917 and 7,016 shares issued and outstanding at September 30, 2014 and December 31, 2013, respectively 3,917 7,016
Series F Convertible Preferred Stock, $0.0001 par value; 10,000 shares authorized, 2,798 and 3,250 shares issued and outstanding at September 30, 2014 and December 31, 2013, respectively 2,798 3,250
Additional paid-in capital 525,866 462,684
Accumulated deficit (479,765) (465,301)
Total shareholders’ equity 52,820 7,653
Total liabilities and shareholders’ equity $ 54,435 $ 8,747

 

 

Company Contact

 

George F. Tidmarsh, M.D., Ph.D.

President & Chief Executive Officer

La Jolla Pharmaceutical Company

Phone: (858) 207-4264

Email: GTidmarsh@ljpc.com

 

and

 

Chester S. Zygmont, III

Senior Director of Finance

La Jolla Pharmaceutical Company

Phone: (858) 207-4262

Email: czygmont@ljpc.com