La Jolla Pharmaceutical Company and Vanderbilt University Enter Exclusive Research and License Agreement Covering Novel BMP Type-I Receptor Inhibitors

Novel Compounds Have Therapeutic Potential in Broad Range of Diseases, Including Certain Rare Genetic Disorders

SAN DIEGO & NASHVILLE, Tenn.–(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 Vanderbilt University (Vanderbilt) today announced an exclusive, worldwide research and license agreement covering Vanderbilt’s research program and intellectual property rights relating to small-molecule kinase inhibitors designed to selectively block specific members of the bone morphogenetic protein (BMP) type-I receptor family.

The seven members of the BMP type-I receptor family, activin receptor-like kinase (ALK) 1-7, play critical roles in human development and physiology. In turn, the improper activation of these receptor pathways is responsible for a wide range of disease conditions. For example, fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder where the body turns muscle into bone, is caused by a genetic mutation in ALK2 that results in excessive signaling of this pathway.

Members of the BMP type-I receptor family also are involved in other diseases such as acquired heterotopic ossification (formation of bone in soft tissue caused by injury or trauma), muscular dystrophies including Duchenne muscular dystrophy, anemia of chronic disease (decrease of red blood cells or hemoglobin from chronic infection, chronic immune activation and malignancy), cancer, cardiovascular diseases and inflammatory bowel disease.

After the first publication of his discovery in 2008, Charles C. Hong, M.D., Ph.D., in conjunction with Vanderbilt, has led a dedicated research team focused on discovering highly selective, small-molecule kinase inhibitors designed to selectively block specific members of the BMP type-I receptor family. Dr. Hong’s team is complemented by the robust medicinal chemistry expertise of Vanderbilt’s Craig W. Lindsley, Ph.D., and Corey R. Hopkins, Ph.D. Under the research and license agreement with La Jolla, La Jolla will fund further research under this program at Vanderbilt in return for rights to acquire compounds emerging from this program.

Dr. Hong is an Associate Professor of Cardiovascular Medicine, Pharmacology, and Cell and Developmental Biology at Vanderbilt University School of Medicine and a member of the Veterans Affairs Tennessee Valley Healthcare System. He is also a member of the Vanderbilt Institute of Chemical Biology and the Vanderbilt Center for Stem Cell Biology. Dr. Hong discovered the first pharmacologic inhibitor of the BMP pathway in 2008, and he was instrumental in the discovery of the critical role of the BMP pathway in stem cell maturation, cholesterol homeostasis and cancer.

Dr. Lindsley is a Professor of Pharmacology and Chemistry at Vanderbilt University School of Medicine. He is also the Director of Medicinal Chemistry at the Vanderbilt Center for Neuroscience Drug Discovery and the Director of the Vanderbilt Specialized Chemistry Center for Accelerated Probe Development.

Dr. Hopkins is an Assistant Professor of Pharmacology and Chemistry at Vanderbilt University School of Medicine. He is also the Associate Director of Chemistry at the Vanderbilt Center for Neuroscience Drug Discovery and a co-Director of the Vanderbilt Specialized Chemistry Center for Accelerated Probe Development.

“We are excited to see our work move forward from a basic research program to a therapeutic program in partnership with La Jolla. Together, we see this as an opportunity to position our BMP inhibitors as potential treatments for rare and neglected diseases,” said Drs. Hopkins and Lindsley on behalf of the Vanderbilt team.

“Tremendous credit goes to Dr. Hong for his discovery of BMP receptor inhibitors and Dr. Lindsley and Dr. Hopkins for their advancements in medicinal chemistry. Their breakthrough discoveries offer hope to improve the lives of patients suffering from a broad range of debilitating diseases,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Office of La Jolla. “In collaboration with our innovative partners at Vanderbilt University Medical Center, we will work hard to translate their pioneering discoveries into novel treatments for patients in need.”

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. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary 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.

About Vanderbilt University Medical Center

Vanderbilt University Medical Center is home to Vanderbilt University Hospital, the Monroe Carell Jr. Children’s Hospital at Vanderbilt, the Vanderbilt Psychiatric Hospital and the Vanderbilt Stallworth Rehabilitation Hospital. These hospitals experienced more than 61,000 inpatient admissions during fiscal year 2015. Vanderbilt’s adult and pediatric clinics treated nearly 2 million patients during this same period.

Both the Vanderbilt University School of Medicine and School of Nursing are recognized by U.S. News & World Report’s annual Best Graduate Schools as among the nation’s best, with the School of Medicine ranked 14th and the School of Nursing 11th. The School of Medicine’s biomedical research program has earned its place among the nation’s top 10 academic medical centers in terms of public and private research funding, receiving more than $500 million in total funding during fiscal year 2015.

Vanderbilt University Hospital and the Monroe Carell Jr. Children’s Hospital at Vanderbilt are recognized again this year by U.S. News & World Report’sBest Hospitals as among the nation’s best, with 18 nationally ranked specialties.

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 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 the Company’s drug candidates; and potential indications for which the Company’s 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
Phone: (858) 207-4264
Email: gtidmarsh@ljpc.com
or
Dennis M. Mulroy
Chief Financial Officer
Phone: (858) 433-6839
Email: dmulroy@ljpc.com

 

Source: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company Announces FDA Acceptance of IND for LJPC-401

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 the U.S. Food and Drug Administration (FDA) has accepted the Company’s Investigational New Drug Application (IND) for LJPC-401, La Jolla’s novel formulation of hepcidin. La Jolla expects to release preliminary results from a Phase 1 clinical trial of LJPC-401 by the end of 2015.

Hepcidin is a naturally occurring regulator of iron absorption and distribution. By regulating the absorption and distribution of iron, hepcidin prevents excessive iron accumulation in tissues, such as the liver and heart, where it can cause significant damage and even result in death.

La Jolla is developing LJPC-401 for the treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH) and beta thalassemia. HH is a disease caused by a genetic deficiency in hepcidin that results in excessive iron accumulation. HH is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, dementia and diabetes.

LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing. Specifically, La Jolla has completed animal toxicology studies that demonstrated a dose-dependent reduction in serum iron levels in all species tested.

“We are pleased to have received clearance from the FDA to begin a Phase 1 study of LJPC-401,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “LJPC-401 presents a unique opportunity to potentially help patients suffering from the effects of iron overload by restoring normal or near-normal levels of hepcidin, the body’s natural regulator of iron absorption and distribution.”

About LJPC-401

LJPC-401 is La Jolla’s novel formulation of hepcidin. Hepcidin is a naturally occurring regulator of iron absorption and distribution. By regulating the absorption and distribution of iron, hepcidin prevents excessive iron accumulation in tissues, such as the liver and heart, where it can cause significant damage and even result in death.

La Jolla is developing LJPC-401 for the treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH) and beta thalassemia. HH is a disease caused by a genetic deficiency in hepcidin that results in excessive iron accumulation. HH is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, dementia and diabetes.

LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing. La Jolla’s Investigational New Drug Application (IND) has been accepted by the FDA, and La Jolla expects to release preliminary results from a Phase 1 study by the end 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 several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary 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 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 (IND), 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 the expected duration over which the Company’s cash balances will fund its operations. 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
Dennis M. Mulroy
Chief Financial Officer
858-433-6839
dmulroy@ljpc.com

 

Source: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company Announces Second Quarter 2015 Financial Results and Recent Corporate Progress

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 reported second quarter 2015 financial results and highlighted recent corporate progress.

Recent Corporate Progress

La Jolla initiated its ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial, a multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial of LJPC-501, La Jolla’s proprietary formulation of angiotensin II, for catecholamine-resistant hypotension (CRH).
La Jolla filed an Investigational New Drug Application (IND) for LJPC-401, La Jolla’s novel formulation of hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis and beta thalassemia, and expects to release preliminary results from a Phase 1 study by the end of 2015. In preparation for this Phase 1 study, La Jolla completed animal toxicology studies that have established the study’s proposed doses and demonstrated a dose-dependent reduction in serum iron levels in all species tested.
La Jolla entered into exclusive worldwide license agreements with the Indiana University Research and Technology Corporation and the University of Alabama at Birmingham to acquire intellectual property rights covering LJPC-30Sa and LJPC-30Sb, 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.
La Jolla announced a reprioritization of its product development programs that resulted in the discontinuation of the development of its polysaccharide-based galectin-3 inhibitors, GCS-100 and LJPC-1010. This reprioritization has allowed La Jolla to reallocate resources to its other development candidates that are more in line with its strategic focus.
“The first half of 2015 was exciting and productive for La Jolla, highlighted by the initiation of the ATHOS 3 trial, the preparation of LJPC-401 for a Phase 1 study and the addition of our next-generation gentamicin derivative program,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “We look forward to building on this momentum and continuing the advancement of each of our exciting programs during the second half of the year.”

Results of Operations

As of June 30, 2015, La Jolla had $36.0 million in cash, compared to $48.6 million as of December 31, 2014. The decrease in cash was primarily due to net cash used for operating activities.

La Jolla’s net cash used for operating activities for the three and six months ended June 30, 2015 was $5.7 million and $11.2 million, respectively, compared to net cash used for operating activities of $2.0 million and $4.7 million, respectively, for the same periods in 2014.

La Jolla’s net loss for the three and six months ended June 30, 2015 was $10.7 million and $19.6 million, or $0.70 per share and $1.29 per share, respectively, compared to a net loss of $4.3 million and $9.4 million, or $0.53 per share and $1.38 per share, respectively, for the same periods in 2014.

The increases in net cash used for operating activities and net loss in 2015 as compared to 2014 were primarily due to increased clinical development costs associated with the initiation of the ATHOS 3 trial of LJPC-501 in CRH, the Phase 2b clinical trial of GCS-100 in advanced chronic kidney disease, the continuing Phase 1/2 clinical trial of LJPC-501 in hepatorenal syndrome and preclinical costs associated with LJPC-401. In addition, there were increases in personnel and related costs, which were mainly due to the hiring of additional personnel to support increased development activities, and costs associated with relocating La Jolla’s corporate headquarters in the second quarter 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 several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis and beta thalassemia. LJPC-30Sa and LJPC-30Sb are 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 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 (IND), 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 the expected duration over which the Company’s cash balances will fund its operations. 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.

2nd Quarter Financial Results

 

Contacts

La Jolla Pharmaceutical Company
George F. Tidmarsh, M.D., Ph.D.
President & Chief Executive Officer
858-207-4264
GTidmarsh@ljpc.com
or
Dennis M. Mulroy
Chief Financial Officer
858-433-6839
dmulroy@ljpc.com

 

Source: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company Announces Exclusive Worldwide License Agreements Covering LJPC-30Sa and LJPC-30Sb

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 signed two exclusive worldwide license agreements for the intellectual property rights covering La Jolla’s next-generation gentamicin derivatives, LJPC-30Sa and LJPC-30Sb. The first license agreement is with the Indiana University Research and Technology Corporation (IURTC), and the second is with the IURTC and the University of Alabama at Birmingham (UAB). The license agreements, which follow from the previously announced option agreements, cover the use of LJPC-30Sa and LJPC-30Sb as antimicrobial agents and for the potential treatment of rare genetic disorders.

Despite kidney toxicity, gentamicin has become one of the most commonly prescribed hospital antibiotics due to its broad spectrum of antimicrobial efficacy. Gentamicin consists primarily of a mixture of four distinct but closely related chemical entities that may contribute differentially to the product’s 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 plans to pursue a dual development strategy with its next-generation gentamicin derivative program. Specifically, La Jolla plans to develop LJPC-30Sa and LJPC-30Sb not only for the potential treatment of serious bacterial infections but also for the potential treatment of 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 (FDA), La Jolla has received guidance that it may proceed with a proposed Phase 1 clinical trial following the submission of an IND.

“We are delighted by the continued progress of our next-generation gentamicin derivative program with our colleagues at IURTC and UAB,” said George Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “Following a positive pre-IND meeting with the FDA, we look forward to further advancing this program with the filing of an IND followed by the commencement of a Phase 1 clinical trial.”

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 due to its broad spectrum of antimicrobial efficacy. Gentamicin consists primarily of a mixture of four distinct but closely related chemical entities that may contribute differentially to the product’s 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 not only for the potential treatment of serious bacterial infections but also for the potential treatment of rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy.

La Jolla believes that 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.

Following a pre-Investigational New Drug Application (IND) meeting with the U.S. Food and Drug Administration (FDA), La Jolla has received guidance that it may proceed with a 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. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary 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; the potential market sizes for our product candidates in development; 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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20150805005559/en/

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

Lakhmir S. Chawla, M.D. Receives the International Vicenza Award

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 “Mink” S. Chawla, M.D., the Company’s Chief Medical Officer, received the International Vicenza Award for Critical Care Nephrology at the 33rd Course on Critical Care Nephrology in June 2015. This award recognizes individuals who have made seminal clinical research advancements that have significantly improved the care of critically ill patients with acute kidney injury (AKI) and have been adopted worldwide.

Dr. Chawla was selected as the recipient of the Vicenza Award for his contributions to the development of the renal angina model, the development and standardization of the furosemide stress test, the link between AKI and chronic kidney disease (CKD), and the use of angiotensin II in the treatment of high-output shock. Dr. Chawla is the tenth recipient to receive the Vicenza Award.

Prior to his appointment at La Jolla, Dr. Chawla was an Associate Professor of Medicine at the George Washington University, where he had 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 was also the Chief of the Division of Intensive Care Medicine at the Washington D.C. Veterans Affairs Medical Center.

Dr. Chawla has been an active investigator in the field of critical care nephrology since 2002. His research has focused on shock, inflammation, AKI epidemiology and outcomes, the link between AKI and CKD, and AKI risk assessment. He is also a leading researcher in the field of AKI biomarkers and functional kidney testing.

“On behalf of the entire La Jolla team, I would like to congratulate Mink on receiving this prestigious award,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “Mink has made many valuable contributions to the field of medicine, and we are honored to have him on our team.”

Professors Claudio Ronco, M.D. and Rinaldo Bellomo, MBBS, M.D., FRACP, FCICM, PGDipEcho presented the award. Professor Ronco founded the International Renal Research Institute of Vicenza that has hosted more than 100 fellows from all over the world and is renowned for the interdisciplinary nature of the research carried out in its many laboratories. Professor Bellomo is the Director of Intensive Care Research at the Austin Hospital in Melbourne, the Founding Chair of the Australian and New Zealand Intensive Care (ANZIC) Society Clinical Trials Group and the current Co-Director of the ANZIC Research Centre.

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 hereditary 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.

Contacts

La Jolla Pharmaceutical Company
George F. Tidmarsh, M.D., Ph.D.
President & Chief Executive Officer
858-207-4264
gtidmarsh@ljpc.com
or
Dennis M. Mulroy
Chief Financial Officer
858-433-6839
dmulroy@ljpc.com

 

Source: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company to Provide Corporate Overview at the JMP Securities Life Science Conference 2015

La Jolla Pharmaceutical Company to Provide Corporate Overview at the JMP Securities Life Science Conference 2015

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 the Company will provide a corporate overview at the JMP Securities Life Science Conference 2015 taking place June 23–24 in New York City.

JMP Securities Life Science Conference 2015 Presentation Details

Date:

 

 

Wednesday, June 24, 2015

Time:

9:30 a.m. Eastern Time

Location:

Fontainebleau Room @ The St. Regis New York

Webcast:

LJPC Webcast Link

A live webcast of the presentation will also be available in the Investor Relations section of La Jolla’s website at www.ljpc.com. A replay of the presentation will be available on La Jolla’s website for 30 days following the event.

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.

View source version on businesswire.com: http://www.businesswire.com/news/home/20150617005422/en/

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

La Jolla Pharmaceutical Company to Provide Corporate Overview at the Jefferies 2015 Global Healthcare Conference

Logo
May 27, 2015 01:01 PM Pacific Daylight Time

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 George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla, will provide a corporate overview at the Jefferies 2015 Global Healthcare Conference taking place June 1–4 in New York City.

Jefferies 2015 Global Healthcare Conference Presentation Details

Date:  Monday, June 1, 2015

Time:  8:30 a.m. Eastern Time

Location:  Imperial Room @ Grand Hyatt Hotel New York

Webcast:  LJPC Webcast Link

A live webcast of the presentation will also be available in the Investor Relations section of La Jolla’s website at www.ljpc.com. A replay of the presentation will be available on La Jolla’s website for 30 days following the event.

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.

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

La Jolla Pharmaceutical Company Acquires Rights to Next-Generation Gentamicin Derivatives

Logo
May 7, 2015 20:17 UTC

Conference Call and Webcast at 8:00 AM Eastern Time on Friday, May 8

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 entered into an exclusive option agreement to acquire the Indiana University Research and Technology Center’s (IURTC) intellectual property rights covering next-generation gentamicin derivatives. Gentamicin has become one of the most commonly prescribed hospital antibiotics, despite causing kidney toxicity. Gentamicin consists of a mixture of distinct but closely related chemical entities that may contribute differentially to the product’s toxicity profile. IURTC’s technology covers the use of next-generation, parenteral gentamicin derivatives as antimicrobial agents with the potential for reduced toxicity.

La Jolla also entered into a second option agreement with IURTC and the University of Alabama at Birmingham (UAB) for the use of these next-generation compounds for the treatment of certain rare genetic diseases, such as cystic fibrosis and Duchenne muscular dystrophy. Gentamicin’s ability to induce a lack of fidelity in gene transcription, intrinsic to its antimicrobial mechanism of action, can be leveraged in the correction of certain human genetic mutations that lead to rare genetic disorders. 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.

La Jolla has initially selected two lead development candidates from the technology, LJPC-30Sa and LJPC-30Sb, which are purified components of the currently marketed gentamicin product. LJPC-30Sa and LJPC-30Sb retain the biologic activity of gentamicin, yet appear to lack the traditional kidney toxicity associated with it. La Jolla plans to pursue a dual development strategy in serious bacterial infections and rare genetic disorders characterized by stop codon mutations, 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.

“We are very pleased to gain access to this intellectual property covering next-generation gentamicin derivatives,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “The use of aminoglycoside antibiotics has been limited primarily by treatment-related toxicity. We believe that our next-generation gentamicin derivatives may retain the activity of gentamicin, but improve the therapeutic window, thereby improving the outcome for patients requiring antimicrobial agents and potentially creating new opportunities for the treatment of rare genetic disorders.”

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 Gentamicin and Aminoglycoside Antibiotics

Gentamicin is an FDA-approved aminoglycoside antibiotic that is commonly used in infections in pregnancy and end-stage renal disease, urinary tract infections, endocarditis, serious Staphylococcus infections, and a wide range of other infections. Aminoglycosides are a broad-spectrum class of Gram-negative antibiotics that are a mainstay in the hospital setting. They are also used to potentiate the activity of certain classes of Gram-positive antibiotics. The use of the aminoglycoside class has increased over the last 10 years due to its broad spectrum activity against resistant bacteria, concentration-dependent killing, and prolonged duration of action, but its usage has been limited by dose-dependent and cumulative kidney toxicity and ototoxicity, which is toxicity to the ear. Current prescription data for aminoglycosides as antimicrobial agents represent over a $500 million market opportunity in the U.S., adjusted for branded pricing of comparable hospital antimicrobials.

A product manufactured through fermentation, gentamicin consists of a mixture of distinct but closely related chemical entities. While these distinct chemical entities appear to all retain antimicrobial activity, they may contribute differentially to toxicity. In particular, gentamicin’s kidney toxicity appears to be associated with some but not all of its constituent chemical entities. A gentamicin derivative that retains antimicrobial activity but had reduced kidney toxicity would represent a major advance for patients.

Gentamicin’s ability to induce a lack of fidelity in gene transcription, intrinsic to its antimicrobial mechanism of action, can 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 currently marketed gentamicin as a chronic treatment for these genetic diseases has been limited by its toxicity profile.

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 visitwww.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; the potential market sizes for our product candidates in development; 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.

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

La Jolla Pharmaceutical Company Announces Portfolio Reprioritization

Logo
May 7, 2015 20:11 UTC

Conference Call and Webcast at 8:00 AM Eastern Time on Friday, May 8

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 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.

Contacts

La Jolla Pharmaceutical Company
George F. Tidmarsh, M.D., Ph.D., 858-207-4264
President & Chief Executive Officer
gtidmarsh@ljpc.com
or
Dennis M. Mulroy, 858-433-6839
Chief Financial Officer
dmulroy@ljpc.com

 

Source: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company Announces First Quarter 2015 Financial Results and Corporate Highlights

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 reported first quarter 2015 financial results and highlighted recent corporate progress and near-term milestones.

Recent Corporate Highlights

  • La Jolla reached agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for its ATHOS 3 trial, in which the agreed-upon primary efficacy endpoint is increase in blood pressure.
  • The ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial, La Jolla’s multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial of LJPC-501, La Jolla’s proprietary formulation of angiotensin II, in catecholamine-resistant hypotension (CRH) was initiated in March 2015.
  • La Jolla’s multicenter, randomized, double-blind, placebo-controlled, Phase 2b clinical trial of GCS-100, La Jolla’s first-in-class galectin-3 inhibitor, in diabetic patients with Stage 3b or 4 Chronic Kidney Disease (CKD) was initiated in March 2015.
  • The executive team of La Jolla was strengthened with the appointment of Lakhmir Chawla, M.D. as its Chief Medical Officer and Dennis Mulroy as its Chief Financial Officer.
  • La Jolla added four new members, Paul Adams, M.D., Victor Gordeuk, M.D., Ashutosh Lal, M.D. and Gordon McLaren, M.D., to its advisory board for the development of LJPC-401, La Jolla’s novel formulation of hepcidin.

“The first quarter was an exciting start to 2015 for La Jolla, highlighted by the initiation of both our LJPC-501 Phase 3 registration trial and our Phase 2b clinical trial of GCS-100 in advanced CKD patients,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “With the recent strengthening of our executive team and the LJPC-401 advisory board, we look forward to building upon our recent progress and continuing the advancement of all of our programs, including the initiation of Phase 1 clinical trials for both LJPC-1010 and LJPC-401 later this year.”

Results of Operations

At March 31, 2015, La Jolla had $42.7 million in cash, compared to $48.6 million at December 31, 2014. The decrease in cash was primarily due to cash used for operating activities for the three months ended March 31, 2015. La Jolla’s net loss for the three months ended March 31, 2015 was $9.0 million, or $0.59 per share, compared to a net loss of $5.1 million, or $0.93 per share, for the same period in 2014. The increase in net loss was primarily due to increased clinical development costs associated with the initiation of our ATHOS 3 trial, the initiation of our Phase 2b clinical trial of GCS-100 in advanced CKD, the continuing Phase 1/2 clinical trial of LJPC-501 in HRS and preclinical costs associated with LJPC-1010 and LJPC-401, as well as increases in personnel and related costs, which were mainly due to additional headcount to support such increased development activities.

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 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.

1st Quarter 2015 Financial Results

Contacts

La Jolla Pharmaceutical Company

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

President & Chief Executive Officer

858-207-4264

GTidmarsh@ljpc.com

or

Dennis M. Mulroy

Chief Financial Officer

858-433-6839

dmulroy@ljpc.com

Source: La Jolla Pharmaceutical Company

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