La Jolla Pharmaceutical Company Announces Intent to Submit Marketing Authorization Application for LJPC-501 in the Third Quarter of 2018

— Decision follows successful Scientific Advice meeting with European Medicines Agency —

 

SAN DIEGO, CA – September 25, 2017 – La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued favorable Scientific Advice regarding the EU regulatory pathway for LJPC-501 (angiotensin II) for the treatment of hypotension in adults with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. Based on this Advice, La Jolla intends to submit a Marketing Authorization Application (MAA) for LJPC-501 in the third quarter of 2018.

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). The

RAAS system is one of three central regulators of blood pressure. LJPC-501 is a vasopressor that leverages the RAAS system. 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).

In August 2017, the Company announced that its New Drug Application (NDA) for LJPC-501 had been accepted for review by the U.S. Food and Drug Administration (FDA). 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. The NDA is based on results of 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 study were 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, EMA 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 Europe and 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: 858-256-7910

Email: svedrick@ljpc.com

and

Dennis M. Mulroy

Chief Financial Officer

La Jolla Pharmaceutical Company Phone: 858-433-6839

Email: dmulroy@ljpc.com

Media Contact

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

Email: matt.middleman@lifescipublicrelations.com

Prespecified Analysis of ATHOS-3 Indicates Survival Benefit in LJPC-501-Treated Patients with Relatively Low Angiotensin II State Analysis

Analysis to Be Presented in Oral Session at the 30th Annual Congress of The European Society of Intensive Care Medicine

SAN DIEGO, CA – September 20, 2017 – La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla) today announced an abstract entitled “Baseline angiotensin levels and ACE effects in patients with vasodilatory shock treated with angiotensin II” has been selected for presentation at the upcoming 30th European Society of Intensive Care Medicine (ESICM) LIVES Annual Congress being held September 23 to 27, 2017 in Vienna, Austria.

 

The abstract published online today includes results from a pre-specified analysis of 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 (synthetic human angiotensin II) in patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. The authors present data showing that a relatively low angiotensin II state (as measured by the ratio of angiotensin I to angiotensin II) predicted increased mortality in patients with vasodilatory shock, suggesting that the activity of angiotensin converting enzyme (ACE), which converts angiotensin I to angiotensin II, may play a critical physiologic role in patients with vasodilatory shock. Furthermore, there was a statistically significant treatment effect of LJPC-501 compared to placebo on mortality in these patients with a relatively low angiotensin II state (relative risk reduction of 36%; HR=0.64; 95% CI: 0.41-1.00; p=0.047).

 

These results will be reviewed by Marlies Ostermann, M.D.,Ph.D., of Guy’s and St. Thomas’ Hospital, London, on Tuesday, September 26, 2017.

30th European Society of Intensive Care Medicine Annual Congress Presentation Details 

Poster Number:         0703

Presentation Title:    Baseline angiotensin levels and ACE effects in patients with vasodilatory shock treated with angiotensin II

Presenter:                  Marlies Ostermann, M.D.,Ph.D., of Guy’s and St. Thomas’ Hospital, London

Session Date:             Tuesday, September 26, 2017

Session Time:            16:00 – 17:50

Session Room:           Area Graz

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of synthetic human angiotensin II. Angiotensin II is the major bioactive component of the renin-angiotensin-aldosterone system (RAAS). The RAAS is one of three central regulators of blood pressure. LJPC-501 is a first in class vasopressor that leverages RAAS. 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).

In August 2017, the Company announced that its New Drug Application (NDA) for LJPC-501 had been accepted for review by the U.S. Food and Drug Administration (FDA). 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. The NDA is based on results of 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 study were 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.

Company Contacts

Sandra Vedrick

Associate Director, Investor Relations & Human Resources

La Jolla Pharmaceutical Company Phone: 858-256-7910

Email: svedrick@ljpc.com

and

Dennis M. Mulroy

Chief Financial Officer

La Jolla Pharmaceutical Company Phone: 858-433-6839

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 Announces Launch of Expanded Access Program for LJPC-501

SAN DIEGO–(BUSINESS WIRE)–Aug. 8, 2017– La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla), today announced that the Company has initiated an expanded access program (EAP) in the United States to provide its investigational drug, LJPC-501 (angiotensin II), to patients with vasodilatory or distributive shock who cannot achieve target mean arterial pressure (MAP) despite adequate fluid resuscitation and treatment with currently available vasopressors (a syndrome alternatively known as “catecholamine resistant hypotension” or “clinically refractory hypotension” (CRH)).

Expanded access, sometimes known as compassionate use, is an option facilitated by the U.S. Food and Drug Administration (FDA) to make available prior to regulatory approval investigational medicine(s) for the treatment of serious or life-threatening diseases or conditions where there are no ongoing clinical trials and there is a lack of satisfactory therapeutic alternatives.

“We are pleased to commence this expanded access program for eligible patients in the U.S.,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “This EAP provides a mechanism for eligible patients to access LJPC-501 while the Company is diligently working to pursue FDA approval.”

About LJPC-501 Expanded Access Program

The LJPC-501 EAP is a program for U.S. patients 18 years of age or older with vasodilatory or distributive shock who are unable to achieve target MAP despite adequate fluid resuscitation and treatment with currently available vasopressors. The enrollment criteria are available at www.clinicaltrials.gov.

Expanded access, sometimes called “compassionate use,” is the use of an investigational medical product to treat a patient’s serious or life-threatening disease or condition outside of a clinical trial. The main distinction between expanded access and the use of an investigational medicine in clinical studies is that expanded access programs are not intended to obtain information about the safety or effectiveness of the drug. Expanded access to an investigational medicinal product can only be provided if the sponsor is actively pursuing marketing approval of the drug. The investigational medicine(s) made available through expanded access have not yet received regulatory approval; therefore, their potential safety and efficacy have not yet been established by the FDA. Doctors and patients should consider all possible benefits and risks when seeking access to investigational medicine(s) prior to regulatory approval.

Under 21 CFR 312.305(a)(3), to authorize any category of expanded access, FDA must determine that expanded access to the drug for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the expanded access use or otherwise compromise the potential development of the drug for the expanded access use. In addition, FDA must determine that there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance.

FDA guidance on expanded access can be found here.

Once a regulatory agency approves the investigational medicine for commercial use and the medicine is commercially available, existing expanded access programs for that medicine will be phased out as soon as practicable while not interfering with patient safety.

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of synthetic human 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. LJPC-501 is being developed for the treatment of patients with catecholamine resistant hypotension (CRH). LJPC-501 is the first synthetic human angiotensin II product candidate to be tested in a Phase 3 study. In February 2017, La Jolla reported positive top-line results from the ATHOS-3 (Angiotensin II for the Treatment of High-Output Shock) Phase 3 study of LJPC-501 in patients with CRH. In May 2017, the results of ATHOS-3 were published by The New England Journal of Medicine in an article entitled “Angiotensin II for the Treatment of Vasodilatory Shock.” The NEJM article and its Supplementary Appendix can be found here.

About Catecholamine Resistant Hypotension

Catecholamine resistant hypotension or clinically refractory hypotension (CRH) is a life-threatening syndrome in patients with vasodilatory (also known as distributive) shock (dangerously low blood pressure with adequate cardiac function) who cannot achieve target mean arterial pressure (MAP) despite adequate fluid resuscitation and treatment with currently available vasopressors (catecholamines and/or vasopressin). There are approximately 500,000 distributive shock cases in the United States per year, an estimated 200,000 of which develop CRH. On current therapies, more than 50% of CRH 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 potential treatment of catecholamine resistant hypotension. 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 availability of LJPC-501 under the expanded access program; the timing and prospects for approval of LJPC-501 by the FDA and the other regulatory authorities; risks relating to the scope of product labels (if approved) and potential market sizes, as well as the broader commercial opportunity; the anticipated timing for regulatory actions; and the success of future development activities; potential indications for which the Company’s product candidates may be developed. 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, 858-256-7910
Associate Director, Investor Relations & Human Resources
svedrick@ljpc.com
and
Dennis M. Mulroy, 858-433-6839
Chief Financial Officer
dmulroy@ljpc.com
or
Media Contact
LifeSci Public Relations
Matt Middleman, M.D., 646-627-8384
matt.middleman@lifescipublicrelations.com

Results of ATHOS‑3 Phase 3 Study of LJPC-501 Published in The New England Journal of Medicine

SAN DIEGO–(BUSINESS WIRE)–May 21, 2017– La Jolla Pharmaceutical Company (Nasdaq: LJPC) (La Jolla) today announced that results of the ATHOS-3 (Angiotensin II for the Treatment of High-OutputShock) Phase 3 study of LJPC-501 (angiotensin II) have been published online by The New England Journal of Medicine (NEJM). The article, entitled “Angiotensin II for the Treatment of Vasodilatory Shock,” also will be published in the May 25, 2017 print issue of NEJM.

“There is a major need for new treatment options for critically ill patients who do not adequately respond to available vasopressors,” stated Rinaldo Bellomo, M.D., Professor of Intensive Care Medicine atUniversity of Melbourne and Director of Intensive Care Research at Austin Health. “In ATHOS-3, angiotensin II was shown to raise blood pressure with no increase in overall adverse events as compared to placebo in this highly treatment-resistant patient population. The effect of angiotensin II resulted in reduced use of other vasopressors. If approved, angiotensin II, in combination with other vasopressors, may allow clinicians to leverage all three major physiologic systems that regulate blood pressure.”

The analysis of the primary efficacy endpoint of ATHOS-3, defined as the percentage of patients achieving a mean arterial pressure (MAP) ≥ 75 mmHg or a 10 mmHg increase from baseline MAP at 3 hours following the initiation of study treatment without an increase in standard-of-care vasopressors, was statistically significant: 23.4% of the 158 placebo-treated patients achieved the pre-specified blood pressure response, compared to 69.9% of the 163 angiotensin II-treated patients (p<0.001). In addition, a trend toward longer survival was observed for angiotensin II-treated patients (22% reduction in mortality risk through day 28; hazard ratio=0.78 [CI: 0.57, 1.07; p=0.12]). In this critically ill patient population, 91.8% of placebo-treated patients experienced at least one adverse event, compared to 87.1% of angiotensin II-treated patients, and 21.5% of placebo-treated patients discontinued treatment due to an adverse event, compared to 14.1% of angiotensin II-treated patients.

The NEJM article can be found here and its Supplementary Appendix can be found here.

About the ATHOS-3 Study

The ATHOS-3 study was a multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical study of LJPC-501 in patients with catecholamine resistant hypotension (CRH), which is a severe form of vasodilatory shock. A total of 344 patients were randomized across nine countries, 321 of whom received study treatment and are included in the primary analysis. Patients were randomized 1:1 to receive either LJPC-501 or placebo on a background of standard-of-care vasopressors selected by the investigators. Randomized patients received their assigned treatment via continuous intravenous infusion.

The primary efficacy endpoint was the percentage of patients achieving a mean arterial pressure (MAP) ≥ 75 mmHg or a 10 mmHg increase from baseline MAP at 3 hours following the initiation of study treatment without an increase in standard-of-care vasopressors. The study was conducted under a Special Protocol Assessment (SPA) agreed to with the U.S. Food and Drug Administration (FDA) in 2015. The SPA stipulates that a study of this size and design could provide sufficient safety and efficacy data and an adequate evaluation of the risk/benefit to the patients to support FDA review and consideration for marketing approval. La Jolla reported positive top-line results in February 2017.

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of synthetic human 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. LJPC-501 is being developed for the treatment of patients with CRH. LJPC-501 is the first synthetic human angiotensin II product candidate to be tested in a Phase 3 study.

About Catecholamine Resistant Hypotension

Catecholamine resistant hypotension (CRH) is a life-threatening syndrome in patients with vasodilatory (also known as distributive) shock (dangerously low blood pressure with adequate cardiac function) who cannot achieve target mean arterial pressure (MAP) despite adequate fluid resuscitation and treatment with currently available vasopressors (catecholamines and/or vasopressin). There are approximately 500,000 distributive shock cases in the United States per year, an estimated 200,000 of which develop CRH. More than 50% of CRH 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 potential treatment of catecholamine resistant hypotension. 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 of the planned NDA submission for LJPC-501 and prospects for approval of LJPC-501 by the FDA and the other regulatory authorities; risks relating to the scope of product labels (if approved) and potential market sizes, as well as the broader commercial opportunity; the anticipated timing for regulatory actions; and the success of future development activities; potential indications for which the company’s product candidates may be developed. The company expressly disclaims any intent to update any forward-looking statements to reflect the outcome of the consequent events.

Source: La Jolla Pharmaceutical Company

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

La Jolla Pharmaceutical Company Announces Positive Top-Line Results from ATHOS-3 Phase 3 Study of LJPC-501

— Primary efficacy endpoint analysis highly statistically significant (p<0.00001)

— Trend toward longer survival observed

— New Drug Application planned for second half of 2017

— Company to host conference call and webcast at 8:30 a.m. EST on Monday, Feb. 27, 2017

SAN DIEGO–(BUSINESS WIRE)–Feb. 27, 2017– La Jolla Pharmaceutical Company (Nasdaq: LJPC) (La Jolla), today announced positive top-line results from the ATHOS-3 (Angiotensin II for the Treatment of High-Output Shock) Phase 3 study of LJPC-501 (angiotensin II) in patients with catecholamine resistant hypotension (CRH).

The analysis of the primary efficacy endpoint, defined as the percentage of patients achieving a pre-specified target blood pressure response, was highly statistically significant: 23% of the 158 placebo-treated patients had a blood pressure response compared to 70% of the 163 LJPC-501-treated patients (p<0.00001). In addition, a trend toward longer survival was observed: 22% reduction in mortality risk through day 28 [hazard ratio=0.78 (0.57-1.07), p=0.12] for LJPC-501-treated patients.

Throughout the study, safety outcomes were followed by an independent Data Safety Monitoring Board (DSMB). The DSMB recommended that the study continue as originally planned. In this critically ill patient population: 92% of placebo-treated patients compared to 87% of LJPC-501-treated patients experienced at least one adverse event, and 22% of placebo-treated patients compared to 14% of LJPC-501-treated patients discontinued treatment due to an adverse event. In collaboration with the investigators, La Jolla plans to present and publish detailed results from the ATHOS-3 study later this year.

ATHOS-3 was conducted under a Special Protocol Assessment with the U.S. Food and Drug Administration (FDA), in which the company and FDA agreed on the study design, study endpoints and study analyses.

“These study results support that angiotensin II, a molecule first synthesized by Dr. Irvine Page at the Cleveland Clinic, improves outcomes in distributive shock patients requiring high-dose catecholamines. Given the high mortality from this condition, it is important to offer physicians another potential treatment option,” said Daniel Sessler, M.D., the Michael Cudahy Professor and Chair of the Department of Outcomes Research at Cleveland Clinic.

“We are grateful to the patients, their families and the dedicated medical teams who contributed to this successful study,” said George F. Tidmarsh, M.D., Ph.D., president and chief executive officer of La Jolla. “We also are very appreciative of the FDA’s advice and contributions in the development of LJPC-501 and look forward to meeting with the FDA to discuss our NDA submission planned for the second half of this year.”

Conference Call at 8:30 a.m. EST on Monday, February 27, 2017

La Jolla will host a conference call and webcast at 8:30 a.m. EST (5:30 a.m. PST) on Monday, February 27, 2017. 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 78311826 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 the ATHOS-3 Study

The ATHOS-3 study (https://www.ncbi.nlm.nih.gov/pubmed/28215131) was a multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical study of LJPC-501 in patients with catecholamine resistant hypotension. A total of 344 patients were randomized across nine countries, 321 of whom received study treatment and are included in the primary analysis. Patients were randomized 1:1 to receive either LJPC-501 or placebo on a background of standard-of-care vasopressors selected by the investigators. Randomized patients received their assigned treatment via continuous intravenous infusion.

The primary efficacy endpoint was the percentage of patients with a mean arterial pressure (MAP) ≥ 75 mmHg or a 10 mmHg increase from baseline MAP at 3 hours following the initiation of study treatment without an increase in standard-of-care vasopressors. The study was conducted under a Special Protocol Assessment (SPA) agreed to with the U.S. Food and Drug Administration (FDA) in 2015. The SPA stipulates that a study of this size and design could provide sufficient safety and efficacy signals and an adequate evaluation of the risk/benefit to the patients to support FDA review and consideration for marketing approval.

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of synthetic human 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. LJPC-501 is being developed for the treatment of patients with catecholamine resistant hypotension (CRH). LJPC-501 is the first synthetic human angiotensin II product candidate to be tested in a Phase 3 study.

About Catecholamine Resistant Hypotension

Catecholamine resistant hypotension (CRH) is a life-threatening syndrome in patients with distributive shock (dangerously low blood pressure with adequate cardiac function) who cannot achieve target mean arterial pressure (MAP) despite adequate fluid resuscitation and treatment with currently available vasopressors (catecholamines and/or vasopressin). There are approximately 500,000 distributive shock cases in the United States per year, an estimated 200,000 of which develop CRH. More than 50% of CRH 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 potential treatment of catecholamine resistant hypotension. 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 of the NDA submission for LJPC-501 and prospects for approval of the NDA; risks that the full data set from the ATHOS-3 study will not be consistent with the top-line results of the study; risks relating to the scope of product labels (if approved) and potential market sizes, as well as the broader commercial opportunity; 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.

Source: La Jolla Pharmaceutical Company

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

La Jolla Pharmaceutical Company Announces Fourth Quarter and Full Year 2016 Financial Results and Corporate Progress

Feb. 23, 2017– 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 2016 financial results and highlighted 2016 corporate progress.

2016 Corporate Progress

  • In the fourth quarter of 2016, La Jolla’s ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 Phase 3 trial completed patient enrollment. ATHOS 3 is La Jolla’s 344-patient, 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). Top-line results are expected in the first quarter of 2017.
  • In the fourth quarter of 2016, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) designated LJPC-401, the Company’s novel formulation of synthetic human hepcidin, as an orphan medicinal product for the treatment of sickle cell disease (SCD).
  • In the third quarter of 2016, La Jolla reported positive results from a multicenter, open-label, dose-escalation Phase 1 trial of LJPC-401 in patients at risk for iron overload due to conditions such as hereditary hemochromatosis (HH), thalassemia, and SCD. LJPC-401 was well tolerated, and there were no dose-limiting toxicities observed. Furthermore, a dose-dependent, statistically significant reduction in serum iron was observed.
  • In the third quarter of 2016, La Jolla reached agreement with the EMA on the design of a pivotal trial of LJPC-401. The pivotal trial will be a randomized, controlled, multicenter trial 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 trial in mid-2017.

“2016 was a productive year for La Jolla, highlighted by the completion of enrollment of our ATHOS 3 Phase 3 trial of LJPC-501 and encouraging results from our Phase 1 trial of LJPC-401,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “We look forward to an exciting 2017, with the expected reporting of top-line results from our ATHOS 3 Phase 3 trial of LJPC-501 in the first quarter of 2017 and the initiation of our pivotal trial for LJPC-401 in mid-2017.”

Results of Operations

As of December 31, 2016, La Jolla had $65.7 million in cash and cash equivalents, compared to $126.5 million as of December 31, 2015. 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, 2016 was $18.6 million and $58.7 million, respectively, compared to net cash used for operating activities of $8.5 million and $25.2 million, respectively, for the same periods in 2015. La Jolla’s net loss for the three and twelve months ended December 31, 2016 was $24.9 million and $78.2 million, or $1.44 per share and $4.54 per share, respectively, compared to a net loss of $11.8 million and $41.9 million, or $0.69 per share and $2.68 per share, respectively, for the same periods in 2015. During the three and twelve months ended December 31, 2016, La Jolla recognized contract revenue of approximately $0.1 million and $0.6 million, respectively, compared to contract revenue of $0.4 million and $1.1 million, respectively, for the same periods in 2015. The net loss for the three and twelve months ended December 31, 2016 includes non-cash, share-based compensation expense of $3.6 million and $14.5 million, respectively, compared to non-cash, share-based compensation expense of $2.7 million and $13.1 million, respectively, for the same periods 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 LJPC-501 and LJPC-401.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. 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 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 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 website 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, the anticipated timing for the Company’s filing of new drug applications or similar filings for regulatory approval, and the anticipated timing for regulatory actions; the success of current and 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 Financials

Source: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company
Sandra Vedrick, 858-256-7910
Associate Director, Investor Relations & Human Resources
svedrick@ljpc.com
or
Dennis M. Mulroy, 858-433-6839
Chief Financial Officer
dmulroy@ljpc.com

La Jolla Pharmaceutical Company Announces Financial Results for the Three and Nine Months Ended September 30, 2016 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 financial results for the three and nine months ended September 30, 2016 and recent corporate progress.

Recent Corporate Progress

In September 2016, La Jolla reported positive results from a multicenter, open-label, dose-escalation Phase 1 trial of LJPC-401, the Company’s novel formulation of synthetic human hepcidin, in patients at risk for iron overload due to conditions such as hereditary hemochromatosis (HH), beta thalassemia, sickle cell disease (SCD) and myelodysplastic syndrome (MDS). Fifteen patients were dosed at escalating dose levels ranging from 1 to 20 mg. LJPC-401 was well tolerated, and there were no dose-limiting toxicities observed. 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).

In September 2016, La Jolla reached agreement with the European Medicines Agency (EMA) on the design of a pivotal trial of LJPC-401, the Company’s novel formulation of synthetic human hepcidin. The pivotal trial will be a randomized, controlled, multicenter trial 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 trial in mid-2017.

In October 2016, the EMA Committee for Orphan Medicinal Products (COMP) issued a positive opinion recommending LJPC-401, synthetic human hepcidin, for designation as an orphan medicinal product for the treatment of SCD.

“The first nine months of 2016 have been productive for La Jolla, highlighted by continued enrollment of our ATHOS 3 Phase 3 trial of LJPC-501 and encouraging results from our Phase 1 trial of LJPC-401, which demonstrated a clear dose-dependent effect of LJPC-401 on serum iron, a clinically important measure,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “We look forward to building on this positive momentum, with the anticipation of reporting results from our ATHOS 3 Phase 3 trial of LJPC-501 in the first quarter of 2017 and initiation of our pivotal trial for LJPC-401 in mid-2017.”

Results of Operations

As of September 30, 2016, La Jolla had $85.0 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 nine months ended September 30, 2016 was $40.1 million, compared to net cash used for operating activities of $16.7 million for the same period in 2015. La Jolla’s net loss for the three and nine months ended September 30, 2016 was $21.3 million and $53.3 million, or $1.23 per share and $3.10 per share, respectively, compared to a net loss of $10.5 million and $30.1 million, or $0.70 per share and $1.99 per share, respectively, for the same periods in 2015. During the three and nine months ended September 30, 2016, La Jolla recognized contract revenue of approximately $44,000 and $531,000, respectively. The net loss includes non-cash, share-based compensation expense of $3.9 million and $11.0 million for the three and nine months ended September 30, 2016, respectively, compared to $3.1 million and $10.3 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.

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 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 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.

3rd Quarter Financials

 

Contacts

La Jolla Pharmaceutical Company

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

President & Chief Executive Officer

(858) 207-4264

gtidmarsh@ljpc.com

and

La Jolla Pharmaceutical Company

Dennis M. Mulroy

Chief Financial Officer

(858) 433-6839

dmulroy@ljpc.com

La Jolla Pharmaceutical Company Receives Positive Opinion from European Orphan Committee 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 European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion recommending LJPC-401 (synthetic human hepcidin) for designation as an orphan medicinal product for the treatment of Sickle Cell Disease (SCD).

SCD is the most common inherited blood disorder in the United States and is caused by a genetic mutation that results in the production of abnormal hemoglobin, the body’s natural oxygen-carrying molecule contained in red blood cells. The abnormal hemoglobin causes the red blood cells to form a “sickle,” or crescent, shape, which may cause occlusion of blood vessels. Patients with severe forms suffer from sometimes life-threatening chronic hemolytic anemia, strokes, and damage to vital organs such as the lungs, spleen, kidney and liver. In patients with chronic hemolytic anemia, hepcidin levels may also be suppressed, which may lead to iron overload. Standard treatment of SCD includes frequent, life-long blood transfusions. While lifesaving, these transfusions cause excess iron accumulation, which in turn is toxic to vital organs, such as the liver and heart. The only currently approved treatments for iron overload are iron chelators, which may cause kidney failure, liver failure or gastrointestinal hemorrhage. In addition to potentially improving iron overload in these patients, LJPC-401 holds the potential to improve the consequences of the disease by reducing the red cell hemoglobin concentration leading to less sickle cell formation.

LJPC-401 is La Jolla’s novel formulation of synthetic human hepcidin, a naturally occurring peptide hormone that is the body’s regulator of iron absorption and distribution. Hepcidin prevents abnormal iron accumulation in 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, beta thalassemia, SCD and myelodysplastic syndrome. In September 2015, the COMP designated LJPC-401 as an orphan medicinal product for the treatment of beta thalassemia intermedia and major.

“We are encouraged by the positive feedback and continued support of the European regulatory authorities,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “Following our recently reported, positive results from our Phase 1 study of LJPC-401, which demonstrated a clear, dose-dependent effect of LJPC-401 on serum iron, and our reaching of an agreement with the EMA on study design, we look forward to initiating our pivotal study of LJPC-401 in mid-2017.”

About European Orphan Drug Designation

Orphan drug designation is a status assigned to a medicine intended for use in rare diseases. To be granted orphan status in the European Union (EU), the medicine must be for the treatment of a life-threatening or chronically debilitating condition that affects no more than five in 10,000 people in the EU and for which no satisfactory treatments exist or, where they do exist, the medicine will be of significant benefit to those affected by that condition.

Applications for orphan designation are evaluated by the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP), which provides its opinion on whether or not the medicine qualifies as an orphan medicine for the treatment, prevention or diagnosis of a rare disease. If the COMP issues a positive opinion, the European Commission (EC) may then grant the medicine orphan status.

An orphan designation allows a pharmaceutical company to benefit from incentives from the EU to develop a medicine for a rare disease, such as reduced fees, regulatory support during the product development phase, access to the centralized authorization procedure (a single application for all EU countries), and 10 years of market exclusivity once the medicine is approved.

About LJPC-401

LJPC-401 is La Jolla’s novel formulation of synthetic human 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 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 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/20161024005420/en/

Contacts

La Jolla Pharmaceutical Company

Sandra Vedrick

Senior Manager, Investor Relations & Human Resources

858-256-7910

svedrick@ljpc.com

or

La Jolla Pharmaceutical Company

Dennis M. Mulroy

Chief Financial Officer

858-433-6839

dmulroy@ljpc.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.

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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