La Jolla Pharmaceutical Company Announces Initiation of Pivotal Clinical Study of LJPC-401 in Patients with Beta Thalassemia

SAN DIEGO, CA – December 4, 2017 – La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla) today announced the initiation of a pivotal clinical study of LJPC-401 (synthetic human hepcidin) in patients with transfusion-dependent beta thalassemia who, despite chelation therapy, have cardiac iron levels above normal. A high level of cardiac iron puts patients at risk of cardiac complications such as heart failure and sudden death.

LJ401-BT01 is a pivotal, multinational, multicenter, randomized, controlled study that is designed to enroll approximately 100 patients across 9 countries, including the United States. Patients will be randomized 1:1 to receive either: (i) weekly subcutaneous injections of LJPC-401, while continuing standard-of-care chelation therapy (LJPC-401 treatment arm); or (ii) a continuation of standard-of-care chelation therapy only (observation arm). After 6 months of treatment, patients randomized to the observation arm will crossover to receive LJPC-401 (plus standard-of-care chelation therapy) for 6 months, while patients randomized to the LJPC-401 treatment arm will continue with LJPC-401 (plus standard-of-care chelation therapy) for an additional 6 months (for a total of one year).

The primary efficacy endpoint of this study is the change in iron content in the heart after 6 months, as measured by cardiac magnetic resonance imaging (MRI). La Jolla had previously announced that it had reached agreement with the European Medicines Agency (EMA) on the design of this registration study of LJPC-401.

“It is an important moment for all of those involved in the research of blood diseases. For the first time, it is possible to explore in humans the therapeutic potential of a natural master regulator of body iron,” said Professor Antonio Piga, M.D., Department of Clinical and Biological Sciences School of Medicine, San Luigi Gonzaga University Hospital in Torino, Italy.

“We are pleased to initiate this pivotal study at leading research centers in the U.S. and worldwide,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We look forward to continuing the research and development efforts of LJPC-401, with a goal of helping patients suffering from iron overload disorders.”

About LJPC-401

La Jolla is developing LJPC-401 (synthetic human hepcidin) for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH), beta thalassemia, sickle cell disease (SCD) and myelodysplastic syndrome (MDS). Hepcidin, an endogenous peptide hormone, is the body’s naturally occurring regulator of iron absorption and distribution. In healthy individuals, hepcidin prevents excessive iron accumulation in vital organs, such as the liver and heart, where it can cause significant damage and even result in death. The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has designated LJPC-401 as an orphan medicinal product for the treatment of beta thalassemia intermedia and major and SCD.

In September 2016, La Jolla reported positive results from a Phase 1 study of LJPC-401 in patients at risk of iron overload suffering from hereditary hemochromatosis, thalassemia and SCD. Single, escalating doses of LJPC-401 were associated with a dose-dependent, statistically significant reduction in serum iron. LJPC-401 was well-tolerated with no dose-limiting toxicities. Injection-site reactions were the most commonly reported adverse event and were all mild or moderate in severity, self-limiting and fully resolved.

In December 2017, La Jolla announced the initiation of a pivotal, multinational, multicenter, randomized, controlled study of LJPC-401 in patients with transfusion-dependent beta thalassemia who, despite chelation therapy, have cardiac iron levels above normal. La Jolla had previously announced that it had reached agreement with the European Medicines Agency (EMA) on the design of this registration study of LJPC-401.

About Beta Thalassemia

Beta thalassemia is a disease characterized by a genetic mutation that results in the underproduction of hemoglobin, the body’s natural oxygen-carrying molecule contained in red blood cells. There are three types of beta thalassemia: beta thalassemia minor, beta thalassemia intermedia and beta thalassemia major. Patients with the more severe forms (intermedia and major) suffer from significant and sometimes life-threatening anemia, bone deformities and enlargement of the spleen, and usually require frequent and life-long blood transfusions. These blood transfusions cause excessive iron accumulation in the body, which is toxic to vital organs, such as the liver and heart. In addition, the underlying anemia causes excessive iron accumulation independent of blood transfusions.

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 (synthetic human angiotensin II) is being developed for the potential treatment of hypotension in adult patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. LJPC-401 (synthetic human hepcidin) is being developed for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. For more information on La Jolla, please visit www.ljpc.com.

Forward Looking Statement Safe Harbor

This press release 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 or statements of current expectations and involve known and unknown risks, uncertainties and other factors, that may cause actual results to be materially different from those anticipated by the 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 website www.sec.gov. These risks include, but are not limited to, risks relating to: the timing, costs, conduct and outcome of clinical studies; the anticipated treatment of future clinical data by the FDA, the EMA or other regulatory authorities, including whether such data will be sufficient for approval; the timing and prospects for approval of LJPC-501 or LJPC-401 by the FDA, the EMA or other regulatory authorities; risks relating to the scope of product labels (if approved); potential market sizes; the success of future development activities; potential indications for which the Company’s product candidates may be developed; the anticipated timing for regulatory actions; the impact of pharmaceutical industry regulation and healthcare legislation in the United States; and the success of future development activities. The Company expressly disclaims any intent to update any forward-looking statements to reflect the outcome of subsequent events.

Company Contacts

Sandra Vedrick

Associate Director, Investor Relations & Human Resources

La Jolla Pharmaceutical Company Phone: 858 207 4264 Ext: 1135

Email: svedrick@ljpc.com

and

Dennis M. Mulroy

Chief Financial Officer

La Jolla Pharmaceutical Company Phone: 858 207 4264 Ext: 1040

Email: dmulroy@ljpc.com

Media Contact

Matt Middleman, M.D. LifeSci Public Relations Phone: 646-627-8384

Email: matt.middleman@lifescipublicrelations.com

La Jolla Pharmaceutical Company Announces U.S. FDA Acceptance of New Drug Application for LJPC-501

La Jolla Pharmaceutical Company Announces U.S. FDA Acceptance of New Drug Application for LJPC-501

– Priority Review Granted 

SAN DIEGO–(BUSINESS WIRE)–Aug. 28, 2017– La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Company’s New Drug Application (NDA) for the investigational drug LJPC-501 (angiotensin II) for the treatment of hypotension in adults with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. The review classification for the application is Priority, and the user fee goal date under the Prescription Drug User Fee Act (PDUFA) is February 28, 2018. In its letter to the Company, the FDA stated that it does not currently plan to hold an advisory committee meeting to discuss this application.

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of synthetic human angiotensin II. Angiotensin II is a major bioactive component of the renin-angiotensin-aldosterone system (RAAS). RAAS is one of three central regulators of blood pressure. LJPC-501 is being developed for the treatment of patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy (catecholamines and/or vasopressin).

The New Drug Application for LJPC-501 is based on data from the ATHOS-3 (Angiotensin II for the Treatment of High Output Shock) multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical study of LJPC-501 in patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. The study was conducted under a Special Protocol Assessment (SPA) agreed to with the FDA in 2015. A total of 344 patients were randomized across nine countries, 321 of whom received study treatment and were included in the primary analysis. In May 2017, the results of the ATHOS-3 studywere published by The New England Journal of Medicine in an article entitled “Angiotensin II for the Treatment of Vasodilatory Shock.”

About Patients with Distributive or Vasodilatory Shock Failing Standard Therapy

Distributive or vasodilatory shock (dangerously low blood pressure with adequate cardiac function) can become life-threatening when a patient is unable to achieve or maintain target mean arterial pressure (MAP) despite treatment with the currently available standard of care (fluids and vasopressors). This life-threatening syndrome has been described as clinically refractory hypotension, catecholamine-resistant hypotension, high-dose vasopressor-dependent shock, catecholamine or vasopressor refractory shock, or catecholamine-resistant vasodilatory shock. There are approximately 500,000 distributive or vasodilatory shock patients in the United States per year with an estimated 200,000 patients failing standard therapy. Approximately 50% of these patients die within 30 days.

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 synthetic human angiotensin II for the treatment of hypotension in adult patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. LJPC-401 is La Jolla’s proprietary formulation of synthetic human hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. LJPC-30S is La Jolla’s next-generation gentamicin derivative program that is focused on the potential treatment of serious bacterial infections as well as 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 press release 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 or statements of current expectations and involve known and unknown risks, uncertainties and other factors, that may cause actual results to be materially different from those anticipated by the 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 and prospects for approval of LJPC-501 by the FDA and other regulatory authorities; risks relating to the scope of product labels (if approved); potential market sizes; the anticipated timing for regulatory actions; the impact of pharmaceutical industry regulation and health care legislation in the United States; and the success of future development activities. The Company expressly disclaims any intent to update any forward-looking statements to reflect the outcome of subsequent events.

Source: La Jolla Pharmaceutical Company

Company Contacts
La Jolla Pharmaceutical Company
Sandra Vedrick
Associate Director, Investor Relations & Human Resources
Phone: 858-256-7910
Email: svedrick@ljpc.com
and
Dennis M. Mulroy
Chief Financial Officer
Phone: 858-433-6839
Email: dmulroy@ljpc.com
or
Media Contact
LifeSci Public Relations
Matt Middleman, M.D.
Phone: 646-627-8384
Email: matt.middleman@lifescipublicrelations.com

 

La Jolla Pharmaceutical Company Reports Positive Results from Phase 1 Study of LJPC-401

La Jolla Pharmaceutical Company Reports Positive Results from Phase 1 Study of LJPC-401

-Dose-dependent, Statistically Significant Reduction in Serum Iron Observed (p=0.008)

-LJPC-401 Well Tolerated; No Dose-Limiting Toxicities at Any Dose Level

-Conference Call at 9:00 AM Eastern Time on Thursday, September 8, 2016

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 positive results from its Phase 1 study of LJPC-401 in patients at risk of iron overload.

The Phase 1 study is a multi-center, open-label, dose-escalation study evaluating the safety, tolerability, and effect on serum iron of a single dose of LJPC-401 in patients at risk of iron overload due to conditions such as hereditary hemochromatosis (HH), thalassemia, and sickle cell disease (SCD). Fifteen patients were dosed at escalating dose levels ranging from 1 mg to 20 mg.

LJPC-401 was well tolerated, and there were no dose-limiting toxicities observed. Injection-site reactions were the most commonly reported adverse event. These were all mild or moderate in severity, self-limiting, and fully resolved. There were no significant changes in serum chemistries or hematology other than serum iron parameters.

A dose-dependent, statistically significant reduction in serum iron was observed (p=0.008 for dose response; not adjusted for multiple comparisons). At the 20 mg dose level, LJPC-401 reduced serum iron by an average of 58.1% from baseline to hour 8 (p=0.001; not adjusted for potential regression to the mean effect), and serum iron had not returned to baseline through day 7 (21.2% reduction from baseline to the end of day 7).

 

Dose Group

Mean Percent Change in Serum Iron

from Baseline to Hour 8

1 mg (n=3)

-14.2% (p=0.149)

5 mg (n=3)

-26.7% (p=0.304)

10 mg (n=3)

-45.5% (p=0.054)

20 mg (n=6)

-58.1% (p=0.001)

P-values not adjusted for potential regression to the mean effect

 

“The results from this study demonstrate a clear, dose-dependent effect of LJPC-401 on serum iron, a clinically important measure,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We look forward to continuing the development of LJPC-401, which we believe can potentially help patients suffering from the effects of iron overload by restoring physiologically relevant levels of hepcidin, the body’s natural regulator of iron absorption and distribution.”

Conference Call at 9:00 AM Eastern Time on Thursday, September 8, 2016

La Jolla will host a conference call and webcast on Thursday, September 8, 2016 at 9:00 AM Eastern Time (6:00 AM Pacific Time). The conference call can be accessed by dialing 877-359-9508 for domestic callers and 224-357-2393 for international callers. Please provide the operator with the passcode 77161203 to join the conference call or click here for the webcast. A slide presentation accompanying today’s press release and the conference call may also be found on La Jolla’s website at www.ljpc.com under the investor relations section. An archive of the conference call and webcast will be available on La Jolla’s website for 30 days following the call.

About LJPC-401

LJPC-401 is La Jolla’s novel formulation of synthetic hepcidin. Hepcidin, an endogenous peptide hormone, is the body’s naturally occurring regulator of iron absorption and distribution. In healthy individuals, hepcidin prevents excessive iron accumulation in vital organs, 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 potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH), beta thalassemia, sickle cell disease (SCD) and myelodysplastic syndrome (MDS). HH is a disease characterized by a genetic deficiency in hepcidin. HH is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, diabetes, arthritis and joint pain. Beta thalassemia, SCD and MDS are genetic diseases of the blood that can cause life-threatening anemia and usually require frequent and life-long blood transfusions. These blood transfusions cause excessive iron accumulation in the body, which is toxic to vital organs, such as the liver and heart. In addition, the underlying anemia causes excessive iron accumulation independent of blood transfusions.

In September 2016, La Jolla reported positive results from a Phase 1 study of LJPC-401 in patients at risk of iron overload suffering from HH, thalassemia and SCD. Single, escalating doses of LJPC-401 were associated with a dose-dependent, statistically significant reduction in serum iron. LJPC-401 was well tolerated with no dose-limiting toxicities. Injection-site reactions were the most commonly reported adverse event. These were all mild or moderate in severity, self-limiting, and fully resolved.

Also in September 2016, La Jolla announced that it has reached agreement with the European Medicines Agency (EMA) on the design of a pivotal study of LJPC-401. The pivotal study will be a randomized, controlled, multi-center study in beta thalassemia patients suffering from iron overload, a major unmet need in an orphan patient population. The primary endpoint will be a clinically relevant measurement directly related to iron overload. La Jolla plans to initiate this study in mid-2017.

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 synthetic hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. LJPC-30S is our next-generation gentamicin derivative program that is focused on therapeutics for the potential treatment of serious bacterial infections as well as 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 commencement of clinical studies, the anticipated timing for completion of such studies, and the anticipated timing for regulatory actions; the success of future development activities; potential indications for which the Company’s product candidates 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.

View source version on businesswire.com: http://www.businesswire.com/news/home/20160907006780/en/

Contacts

La Jolla Pharmaceutical Company

Sandra Vedrick

Senior Manager, Investor Relations & Human Resources

Phone: (858) 256-7910

Email: svedrick@ljpc.com

and

Dennis M. Mulroy

Chief Financial Officer

Phone: (858) 433-6839

Email: dmulroy@ljpc.com

Source: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company Announces Agreement with European Medicines Agency on Pivotal Study of LJPC-401

La Jolla Pharmaceutical Company Announces Agreement with European Medicines Agency on Pivotal Study of LJPC-401

-Conference Call at 9:00 AM Eastern Time on Thursday, September 8, 2016

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 European Medicines Agency (EMA) on the design of a pivotal study of LJPC-401, La Jolla’s novel formulation of synthetic hepcidin. The pivotal study will be a randomized, controlled, multi-center study in beta thalassemia patients suffering from iron overload, a major unmet need in an orphan patient population. The primary endpoint will be a clinically relevant measurement directly related to iron overload. La Jolla plans to initiate this pivotal study in mid-2017.

“We very much appreciate the EMA’s support of advancing LJPC-401’s development for this major unmet need,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “The EMA’s insights have been invaluable as we sought to design a pivotal study that would best evaluate LJPC-401’s potential to help beta thalassemia patients suffering from iron overload. Based on our recently announced Phase 1 results, and the fact that hepcidin is the body’s natural regulator of iron absorption and distribution, we remain confident that LJPC-401 can help restore normal or near-normal levels of iron in patients suffering from iron overload and its devastating consequences.”

Conference Call at 9:00 AM Eastern Time on Thursday, September 8, 2016

La Jolla will host a conference call and webcast on Thursday, September 8, 2016 at 9:00 AM Eastern Time (6:00 AM Pacific Time). The conference call can be accessed by dialing 877-359-9508 for domestic callers and 224-357-2393 for international callers. Please provide the operator with the passcode 77161203 to join the conference call or click here for the webcast. A slide presentation accompanying today’s press release and the conference call may also be found on La Jolla’s website at www.ljpc.com under the investor relations section. An archive of the conference call and webcast will be available on La Jolla’s website for 30 days following the call.

About LJPC-401

LJPC-401 is La Jolla’s novel formulation of synthetic hepcidin. Hepcidin, an endogenous peptide hormone, is the body’s naturally occurring regulator of iron absorption and distribution. In healthy individuals, hepcidin prevents excessive iron accumulation in vital organs, 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 potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH), beta thalassemia, sickle cell disease (SCD) and myelodysplastic syndrome (MDS). HH is a disease characterized by a genetic deficiency in hepcidin. HH is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, diabetes, arthritis and joint pain. Beta thalassemia, SCD and MDS are genetic diseases of the blood that can cause life-threatening anemia and usually require frequent and life-long blood transfusions. These blood transfusions cause excessive iron accumulation in the body, which is toxic to vital organs, such as the liver and heart. In addition, the underlying anemia causes excessive iron accumulation independent of blood transfusions.

In September 2016, La Jolla reported positive results from a Phase 1 study of LJPC-401 in patients at risk of iron overload suffering from HH, thalassemia and SCD. Single, escalating doses of LJPC-401 were associated with a dose-dependent, statistically significant reduction in serum iron. LJPC-401 was well tolerated with no dose-limiting toxicities. Injection-site reactions were the most commonly reported adverse event. These were all mild or moderate in severity, self-limiting, and fully resolved.

Also in September 2016, La Jolla announced that it has reached agreement with the European Medicines Agency (EMA) on the design of a pivotal study of LJPC-401. The pivotal study will be a randomized, controlled, multi-center study in beta thalassemia patients suffering from iron overload, a major unmet need in an orphan patient population. The primary endpoint will be a clinically relevant measurement directly related to iron overload. La Jolla plans to initiate this study in mid-2017.

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 synthetic hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. LJPC-30S is our next-generation gentamicin derivative program that is focused on therapeutics for the potential treatment of serious bacterial infections as well as 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 commencement of clinical studies, the anticipated timing for completion of such studies, and the anticipated timing for regulatory actions; the success of future development activities; potential indications for which the Company’s product candidates 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.

View source version on businesswire.com: http://www.businesswire.com/news/home/20160907006755/en/

Contacts

La Jolla Pharmaceutical Company

Sandra Vedrick

Senior Manager, Investor Relations & Human Resources

858-256-7910

svedrick@ljpc.com

and

La Jolla Pharmaceutical Company

Dennis M. Mulroy

Chief Financial Officer

858-433-6839

dmulroy@ljpc.com

Source: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company Announces Financial Results for the Three and Six Months Ended June 30, 2016

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 financial results for the three and six months ended June 30, 2016.

Results of Operations

As of June 30, 2016, La Jolla had $100.6 million in cash and cash equivalents, compared to $126.5 million as of December 31, 2015. The decrease in cash and cash equivalents was primarily due to net cash used for operating activities. Based on current operating plans and projections, La Jolla believes that its current cash and cash equivalents are sufficient to fund operations into 2018.

La Jolla’s net cash used for operating activities for the six months ended June 30, 2016 was $25.1 million, compared to net cash used for operating activities of $11.2 million for the same period in 2015. La Jolla’s net loss for the three and six months ended June 30, 2016 was $15.6 million and $32.0 million, or $0.90 per share and $1.86 per share, respectively, compared to a net loss of $10.7 million and $19.6 million, or $0.70 per share and $1.29 per share, respectively, for the same periods in 2015. During the three and six months ended June 30, 2016, La Jolla recognized contract revenue of approximately $0.3 million and $0.5 million, respectively. The net loss includes non-cash, share-based compensation expense of $3.3 million and $7.0 for the three and six months ended June 30, 2016, respectively, compared to $3.9 million and $7.3 million, respectively, for the same periods in 2015.

The increases in net cash used for operating activities and net loss in the 2016 periods as compared to the 2015 periods were primarily due to increased development costs associated with our ATHOS 3 Phase 3 trial of LJPC-501 in patients with catecholamine-resistant hypotension and our Phase 1 trial of LJPC-401 in patients with iron overload. There also were increases in personnel and facility costs associated with the support of these increased development activities.

“The first half of 2016 was a productive period for La Jolla, highlighted by the continued enrollment of our ATHOS 3 Phase 3 trial of LJPC-501 and encouraging interim data from our Phase 1 trial of LJPC-401,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “Catecholamine-resistant hypotension and iron overload remain significant unmet medical needs, and we remain dedicated to bringing our potentially important therapies to patients as expeditiously as possible. We plan to report results from our Phase 1 trial of LJPC-401 in September 2016 and from our ATHOS 3 Phase 3 trial of LJPC-501 in the first quarter of 2017.”

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, beta thalassemia, sickle cell disease and myelodysplastic syndrome. LJPC-30S is our next-generation gentamicin derivative program that is focused on therapeutics for the potential treatment of serious bacterial infections as well as 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 commencement of clinical studies, the anticipated timing for completion of such studies, and the anticipated timing for regulatory actions; the success of future development activities; potential indications for which the Company’s product candidates 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 Financials

 

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

La Jolla Pharmaceutical Company Announces First Quarter 2016 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 first quarter 2016 financial results and highlighted recent corporate progress.

Recent Corporate Progress

La Jolla’s ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial continued to enroll as planned through the first quarter of 2016. The ATHOS 3 trial is La Jolla’s multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial of LJPC-501, the Company’s proprietary formulation of angiotensin II, in catecholamine-resistant hypotension (CRH), which was initiated in 2015. The Company initiated the ATHOS 3 trial after reaching an agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA), in which the agreed-upon primary efficacy endpoint in ATHOS 3 is an increase in blood pressure. Results of ATHOS 3 are expected by the end of 2016.
La Jolla reported interim results in January 2016 of its multicenter, open-label, dose-escalation Phase 1 clinical trial of LJPC-401, the Company’s novel formulation of hepcidin, in patients at risk for iron overload due to conditions such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. These results suggested a dose-dependent reduction in serum iron following a single dose of LJPC-401. Complete results from this Phase 1 clinical trial, which was initiated in October 2015, are expected in the second half of 2016.
“The first quarter was a productive start to 2016 for La Jolla, highlighted by the continued enrollment of our ATHOS 3 trial and encouraging interim data from our Phase 1 clinical trial of LJPC-401,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “We look forward to continuing the advancement of each of our exciting programs during the rest of 2016, culminating with the results from the ATHOS 3 trial that are expected by the end of the year.”

Results of Operations

As of March 31, 2016, La Jolla had $113.1 million in cash and cash equivalents, compared to $126.5 million as of December 31, 2015. The decrease in cash and cash equivalents was primarily due to net cash used for operating activities. Based on current operating plans and projections, La Jolla believes that its current cash and cash equivalents are sufficient to fund operations into 2018.

La Jolla’s net cash used for operating activities for the three months ended March 31, 2016 was $13.0 million, compared to net cash used for operating activities of $5.5 million for the same period in 2015. La Jolla’s net loss for the three months ended March 31, 2016 was $16.5 million, or $0.96 per share, compared to a net loss of $9.0 million, or $0.59 per share, for the same period in 2015. During the three months ended March 31, 2016, La Jolla recognized contract revenue of approximately $0.2 million, which was pursuant to a services agreement initiated in 2015 under which La Jolla provides research and development services to a related party. The net loss includes non-cash, share-based compensation expense of $3.7 million for the three months ended March 31, 2016, compared to $3.4 million for the same period in 2015.

The increases in net cash used for operating activities and net loss in 2016 as compared to 2015 were primarily due to increased development costs associated with the ATHOS 3 trial and the costs associated with the initiation of the Phase 1 clinical trial of LJPC-401 in iron overload. In addition, there were increases in personnel and related costs, which were mainly due to the hiring of additional personnel and increased facility costs to support the 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 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, beta thalassemia, sickle cell disease and myelodysplastic syndrome. LJPC-30S is our next-generation gentamicin derivative program that is focused on therapeutics for the potential treatment of serious bacterial infections as well as 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 commencement of clinical studies, the anticipated timing for completion of such studies, and the anticipated timing for regulatory actions; the success of future development activities; potential indications for which the Company’s product candidates 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.

 

First Quarter 2016 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 Fourth Quarter and Full Year 2015 Financial Results and Highlights 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 fourth quarter and full year 2015 financial results and highlighted 2015 corporate progress.

La Jolla Pharmaceutical Company Announces Fourth Quarter and Full Year 2015 Financial Results and Highlights 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 fourth quarter and full year 2015 financial results and highlighted 2015 corporate progress.

2015 Corporate Progress

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. The initiation of the ATHOS 3 trial followed the reaching of an agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA), in which the agreed-upon primary efficacy endpoint in ATHOS 3 is increase in blood pressure. ATHOS 3 is enrolling as planned, and results are expected by the end of 2016.
A multicenter, open-label, dose-escalation Phase 1 clinical trial of LJPC-401, the Company’s novel formulation of hepcidin, in patients at risk for iron overload due to conditions such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome was initiated in October 2015. Interim results, reported in January 2016, suggested a dose-dependent reduction in serum iron following a single dose of LJPC-401. Additionally, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) designated LJPC-401 an orphan medicinal product for the treatment of beta thalassemia intermedia and major.
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 in May 2015. 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.
La Jolla completed a public offering of common stock in September 2015, whereby La Jolla received approximately $104.6 million, net of issuance costs. La Jolla finished 2015 with $126.5 million in cash and cash equivalents and believes this is sufficient to fund operations into 2018.
“2015 was a very exciting year for La Jolla, highlighted by the initiation and continued progress of our Phase 3 clinical trial of LJPC-501 and encouraging interim data from our recently initiated Phase 1 clinical trial of LJPC-401,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “We look forward to a productive 2016, with the continued advancement of our exciting product candidates and results from our LJPC-501 Phase 3 clinical trial expected by the end of the year.”

Results of Operations

As of December 31, 2015, La Jolla had $126.5 million in cash and cash equivalents, compared to $48.6 million as of December 31, 2014. The increase in cash and cash equivalents was primarily due to cash provided by our common stock offering that was completed in September 2015, which was partially offset by net cash used for operating activities. Based on current operating plans and projections, La Jolla believes that its current cash and cash equivalents are sufficient to fund operations into 2018.

La Jolla’s net cash used for operating activities for the three and twelve months ended December 31, 2015 was $8.5 million and $25.2 million, respectively, compared to net cash used for operating activities of $5.4 million and $12.9 million, respectively, for the same periods in 2014. La Jolla’s net loss for the three and twelve months ended December 31, 2015 was $11.8 million and $41.9 million, or $0.69 per share and $2.68 per share, respectively, compared to a net loss of $6.8 million and $21.3 million, or $0.45 per share and $2.00 per share, respectively, for the same periods in 2014. During the three and twelve months ended December 31, 2015, La Jolla recognized contract revenue of approximately $0.4 million and $1.1 million, respectively, which was pursuant to a services agreement initiated in 2015 under which La Jolla provides research and development services to a related party. The net loss includes non-cash, share-based compensation expense of $2.8 million and $13.1 million for the three and twelve months ended December 31, 2015, respectively, compared to $2.5 million and $9.1 million of noncash, share-based compensation expense, 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 development costs associated with the Phase 3 clinical trial of LJPC-501 in catecholamine-resistant hypotension and the costs associated with the initiation of the Phase 1 clinical trial of LJPC-401 in iron overload. In addition, there were increases in personnel and related costs, which were mainly due to the hiring of additional personnel and increased facility costs to support the 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 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, beta thalassemia, sickle cell disease and myelodysplastic syndrome. 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 commencement of clinical studies, the anticipated timing for completion of such studies, and the anticipated timing for regulatory actions; the success of future development activities; potential indications for which the Company’s product candidates 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.

4th Quarter Earnings

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