La Jolla Pharmaceutical Company Announces Notice of Allowance for U.S. Patent Covering LJPC-501

Nov. 30, 2015 13:00 UTC

La Jolla Pharmaceutical Company Announces Notice of Allowance for U.S. Patent Covering LJPC-501

Patent Term Extends to 2034

SAN DIEGO–(BUSINESS WIRE)– La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced the issuance of a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for U.S. patent application number 14/575,127, entitled “Angiotensin II Alone or in Combination for the Treatment of Hypotension,” which covers LJPC-501, La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension (CRH). LJPC-501 is currently the subject of a Phase 3 clinical trial for the treatment of CRH, and results from this trial are expected by the end of 2016.

La Jolla has an exclusive license to this patent application from the George Washington University, and the resulting patent will have a term extending to 2034. This Notice of Allowance concludes substantive examination of the patent application and will result in the issuance of a U.S. patent after administrative processes are completed. Additional claims are being pursued in a continuation application.

“This newly allowed patent covering LJPC-501 is a valuable addition to La Jolla’s intellectual property portfolio,” said George F. Tidmarsh, M.D., Ph.D., President and CEO of La Jolla. “We believe that LJPC-501 has the potential to reverse acute hypotension in critically ill patients and, therefore, provide a significant benefit to patients suffering from CRH.”

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of angiotensin II. Angiotensin II, the major bioactive component of the renin-angiotensin system, serves as one of the body’s central regulators of blood pressure. La Jolla is developing LJPC-501 for the treatment of catecholamine-resistant hypotension (CRH), which is an acute, life-threatening condition in which blood pressure drops to dangerously low levels in patients who respond poorly to current treatments. Angiotensin II has been shown to raise blood pressure in a randomized, placebo-controlled clinical trial in CRH, which was recently published in the journal Critical Care, as well as in animal models of hypotension. Preclinical pharmacology studies that La Jolla has conducted have demonstrated that catecholamine resistance may be in part a result of reduced endogenous production of angiotensin II. In October 2014, La Jolla presented positive data from a preclinical study of LJPC-501 for the treatment of CRH.

La Jolla initiated a Phase 3 clinical trial with LJPC-501 for the treatment of CRH, called the ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial, in March 2015. In February 2015, La Jolla reached agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for this multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial. In accordance with the SPA, the primary efficacy endpoint for the ATHOS 3 registration trial is increase in blood pressure at three hours. The ATHOS 3 trial is designed to enroll approximately 315 patients. Patients are to be randomized in a 1:1 fashion to receive either: (i) LJPC-501 plus standard-of-care vasopressors; or (ii) placebo plus standard-of-care vasopressors. Randomized patients are to receive their assigned treatment via continuous IV infusion for up to seven days. The primary efficacy endpoint in the study is to compare the change in mean arterial pressure in patients with CRH who receive an IV infusion of LJPC-501 plus standard-of-care vasopressors to those that receive placebo plus standard-of-care vasopressors. Secondary endpoints include comparison of changes in Cardiovascular Sequential Organ Failure Assessment, or SOFA scores, and the safety and tolerability of LJPC-501 in patients with CRH. Results from ATHOS 3 are expected by the end of 2016.

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 and sickle cell disease. LJPC-30Sa and LJPC-30Sb are Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

Forward-Looking Statement Safe Harbor

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing for 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 for LJPC-501, LJPC-401, LJPC-30Sa and LJPC-30Sb; potential indications for which LJPC-501, LJPC-401, LJPC-30Sa and LJPC-30Sb may be developed; events that could interfere with the issuance of a patent, or once issued, the continued validity or enforceability of a patent; the Company’s ability generally to maintain adequate patent protection and successfully enforce patent claims against third parties; 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/20151130005355/en/

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 Third Quarter 2015 Financial Results and Recent Corporate Progress

SAN DIEGO–(BUSINESS WIRE)– La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today reported third quarter 2015 financial results and highlighted recent corporate progress.

Recent Corporate Progress

La Jolla initiated a Phase 1 clinical trial of LJPC-401, the Company’s novel formulation of hepcidin, in patients at risk of iron overload due to conditions such as hereditary hemochromatosis, beta thalassemia and sickle cell disease. Preliminary results from this clinical trial are anticipated by the end of 2015.
La Jolla received a positive opinion from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP), which the European Commission subsequently adopted, for designation of LJPC-401 as an orphan medicinal product for the treatment of beta thalassemia intermedia and major.
La Jolla and Vanderbilt University entered into an exclusive, worldwide research and license agreement covering Vanderbilt’s research program and intellectual property rights relating to small-molecule kinase inhibitors designed to selectively block specific members of the bone morphogenetic protein (BMP) type-I receptor family, for the potential treatment of fibrodysplasia ossificans progressiva (FOP), acquired heterotopic ossification, muscular dystrophies including Duchenne muscular dystrophy, anemia of chronic disease, cancer, cardiovascular diseases and inflammatory bowel disease.
La Jolla received orphan drug designation from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development on two novel compounds, which were licensed from Vanderbilt University, for FOP.
La Jolla completed an underwritten public offering of approximately 2.9 million shares of common stock, which includes the full exercise of the underwriters’ overallotment option, at a public offering price of $38.00 per share. The Company received total net proceeds of approximately $104.6 million from this offering.
“The second half of 2015 is off to an exciting and productive start for La Jolla, highlighted by the initiation of our Phase 1 clinical trial for LJPC-401, continued progress with our Phase 3 clinical trial of LJPC-501, the receipt of a positive opinion for designation of LJPC-401 as an orphan medicinal product in Europe and the receipt of orphan drug designation for two of the recently licensed compounds from Vanderbilt in the U.S.,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “We look forward to building on this momentum, with the anticipation of preliminary results from the LJPC-401 Phase 1 clinical trial by the end of this year and results from the LJPC-501 Phase 3 clinical trial by the end of 2016.”

Results of Operations

As of September 30, 2015, La Jolla had $135.1 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 nine months ended September 30, 2015 was $5.5 million and $16.7 million, respectively, compared to net cash used for operating activities of $2.8 million and $7.5 million, respectively, for the same periods in 2014. La Jolla’s net loss for the three and nine months ended September 30, 2015 was $10.5 million and $30.1 million, or $0.70 per share and $1.99 per share, respectively, compared to a net loss of $5.1 million and $14.5 million, or $0.37 per share and $1.58 per share, respectively, for the same periods in 2014. During the three months ended September 2015, La Jolla recognized approximately $0.6 million of contract revenue, which was pursuant to a services agreement under which La Jolla provides research and development services to a related party. The net loss includes noncash, share-based compensation expense of $3.1 million and $10.3 million for the three and nine months ended September 30, 2015, respectively, compared to $1.7 million and $6.6 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 clinical development costs associated with the continuation of 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 and clinical 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 and sickle cell disease. LJPC-30Sa and LJPC-30Sb are Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

Forward Looking Statement Safe Harbor

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing for 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 for LJPC-501, LJPC-401, LJPC-30Sa and LJPC-30Sb; potential indications for which LJPC-501, LJPC-401, LJPC-30Sa and LJPC-30Sb may be developed; and the expected duration over which the Company’s cash balances will fund its operations. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

3rd Quarter Financial Results

View source version on businesswire.com: http://www.businesswire.com/news/home/20151106005122/en/

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 Doses First Patient in Phase 1 Clinical Trial of LJPC-401 in Patients at Risk of Iron Overload

La Jolla Pharmaceutical Company Doses First Patient in Phase 1 Clinical Trial of LJPC-401 in Patients at Risk of Iron Overload

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 first patient has been dosed in a Phase 1 clinical trial of LJPC-401, La Jolla’s novel formulation of hepcidin.

The Phase 1 clinical trial is a multicenter, open-label, dose-escalation trial that is evaluating the safety, tolerability and effect on serum iron parameters, and pharmacokinetics of LJPC-401 in patients at risk of iron overload due to conditions such as hereditary hemochromatosis (HH), beta thalassemia and sickle cell disease (SCD). Preliminary results from this clinical trial are anticipated by the end of 2015.

LJPC-401 is La Jolla’s novel formulation of hepcidin, a naturally occurring peptide hormone that is the body’s regulator of iron absorption and distribution. 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 HH, beta thalassemia and SCD.

HH is a disease caused by a genetic deficiency in hepcidin production, resulting in excessive iron accumulation. 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 and SCD are genetic diseases of blood cells that can cause life-threatening anemia and often require frequent and life-long blood transfusions. These blood transfusions, while necessary to treat anemia, cause excessive iron accumulation in the body, which is toxic to vital organs. In addition, the underlying anemia may cause excessive iron accumulation independent of the blood transfusions.

“We are excited to be advancing LJPC-401 into its first clinical trial, and we are very grateful to the physicians and patients who have partnered with us on this important program,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We believe that LJPC-401 presents a unique opportunity to potentially help patients suffering from the effects of iron overload by restoring normal or near-normal levels of hepcidin, the body’s natural regulator of iron absorption and distribution.”

About LJPC-401

LJPC-401 is La Jolla’s novel formulation of hepcidin. Hepcidin, an endogenous peptide hormone, is the body’s naturally occurring regulator of iron absorption and distribution. 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 and sickle cell disease (SCD). HH is a disease caused by a genetic deficiency in hepcidin that results in excessive iron accumulation. HH is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, diabetes, arthritis and joint pain. Beta thalassemia and SCD 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.

LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing. La Jolla has initiated a Phase 1 clinical trial of LJPC-401 in patients at risk of iron overload due to conditions such as HH, beta thalassemia and SCD. La Jolla anticipates preliminary results from this Phase 1 clinical trial by the end of 2015.

About Hereditary Hemochromatosis

Hereditary hemochromatosis (HH) is the most common genetic disease in Caucasians. HH is a disease characterized by a genetic mutation that results in a deficiency in the production of hepcidin, which is the body’s naturally occurring regulator of iron absorption and distribution. Without normal levels of hepcidin, excessive amounts of iron accumulate in the body and can lead to liver cirrhosis, liver cancer, heart disease and/or failure, diabetes, arthritis and joint pain. With no FDA-approved treatments for HH, patients are treated with phlebotomy, which does not address the underlying disease pathology, carries significant toxicity and adversely impacts quality of life.

About Beta Thalassemia and Sickle Cell Disease

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.

Sickle cell disease (SCD) is the most common inherited blood disorder in the United States. SCD is characterized 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. Patients with severe forms suffer from significant and sometimes life-threatening anemia, strokes and damage to vital organs such as the lungs, spleen, kidney and liver.

Standard treatment for both beta thalassemia and SCD includes frequent, life-long blood transfusions. While lifesaving, these transfusions cause an excess of iron accumulation, which in turn is toxic to vital organs, such as the liver and heart. In addition, the underlying anemia causes excessive iron accumulation independent of blood transfusions. The only currently approved treatments for iron overload are iron chelators, which may cause kidney failure, liver failure or gastrointestinal hemorrhage.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH), beta thalassemia and sickle cell disease (SCD). LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

Forward-Looking Statement Safe Harbor

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing for the filing of an Investigational New Drug Application (IND); commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for the Company’s drug candidates; potential indications for which the Company’s drug candidates may be developed; and the Company’s ability to obtain the potential benefits of orphan drug designation for its drug candidates. 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/20151019005433/en/

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 Closes Offering of Common Stock

La Jolla Pharmaceutical Company Closes Offering of Common Stock

SAN DIEGO–(BUSINESS WIRE)– La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the “Company” or “La Jolla”), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced the closing of its underwritten public offering of 2,932,500 shares of common stock at a public offering price of $38.00 per share, which includes the exercise of the underwriters’ option to purchase up to an additional 382,500 shares of common stock. Gross offering proceeds are approximately $111.4 million, including proceeds from the exercise of the underwriters’ option to purchase additional shares, before deducting customary underwriting discounts and commissions and offering expenses.

La Jolla intends to use the net proceeds from the offering for general corporate purposes, including funding its ongoing and future clinical trials, and for general and administrative expenses.

Jefferies LLC and Cowen and Company, LLC acted as joint book-running managers for the offering. Chardan Capital Markets, LLC, LifeSci Capital LLC and Noble Life Science Partners acted as co-managers for the offering.

The securities described above were offered pursuant to a shelf registration statement (File No. 333-197092), including a base prospectus, which was declared effective by the United States Securities and Exchange Commission (“SEC”) on July 11, 2014, and a related automatically effective registration statement filed pursuant to Rule 462(b) of the Securities Act of 1933 (File No. 333-206855). The specific terms of the offering are described in a prospectus supplement filed with the SEC in connection with the offering. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. The offering was made only by means of the prospectus supplement and accompanying prospectus, copies of which may be obtained at the SEC’s website at www.sec.gov, or by request at Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, telephone: (877) 547-6340, e-mail: Prospectus_Department@Jefferies.com, or at Cowen and Company, LLC, c/o Broadridge Financial Services, Attention: Prospectus Department, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: (631) 274-2806, fax: (631) 254-7140.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH), beta thalassemia and sickle cell disease. 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.

View source version on businesswire.com: http://www.businesswire.com/news/home/20150915006924/en/

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

Source: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company Receives Positive Opinion from European Orphan Committee for LJPC-401

La Jolla Pharmaceutical Company Receives Positive Opinion from European Orphan Committee for LJPC-401

EMA Innovation Task Force Meeting Granted

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, La Jolla’s novel formulation of hepcidin, for designation as an orphan medicinal product for the treatment of chronic iron overload requiring chelation therapy. Chronic iron overload occurs in patients suffering from beta thalassemia, sickle cell disease and hereditary hemochromatosis (HH). The final opinion, which is subject to review and approval by the European Commission (EC), may include all or a subset of these conditions.

Beta thalassemia and sickle cell disease are genetic diseases of blood cells that can cause life-threatening anemia and often require frequent and life-long blood transfusions. These blood transfusions, while necessary to treat anemia, cause excessive iron accumulation in the body, which is toxic to vital organs. HH is a disease caused by a genetic deficiency in hepcidin production, resulting in excessive iron accumulation. HH is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, dementia and diabetes.

Iron chelators are drugs that bind to and help clear excessive iron from the body. However, chelators cause significant toxicity, including kidney failure, liver failure or gastrointestinal hemorrhage.

LJPC-401 is La Jolla’s novel formulation of hepcidin, a naturally occurring peptide hormone that is the body’s regulator of iron absorption and distribution. Hepcidin prevents excessive 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 treatment of iron overload, which occurs as a result of diseases such as HH, beta thalassemia and sickle cell disease.

In a separate decision, the EMA’s Innovation Task Force (ITF) has granted a meeting with La Jolla to review future development plans for LJPC-401. The ITF provides an interactive platform for applicants with novel and innovative therapies to proactively discuss the scientific, legal and regulatory aspects of development for such therapies with the EMA. This early dialogue is intended to contribute to the preparedness of an applicant and to increase EMA awareness and education of emerging therapies and technologies.

“We believe that LJPC-401 can have a major impact on the lives of patients suffering from chronic iron overload,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We are encouraged by the positive feedback and support of the EU regulatory authorities and are excited to further our clinical development of LJPC-401.”

Emas Pharma Limited served as La Jolla’s European regulatory representative for these regulatory interactions.

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 the European Medicines Agency’s Innovation Task Force

The European Medicines Agency (EMA) established its Innovation Task Force (ITF) in order to provide support for medical innovation in the European Union (EU), with a particular focus on emerging therapies and technologies. The ITF provides an interactive platform for applicants with novel and innovative therapies to proactively discuss the scientific, legal and regulatory aspects of development for such therapies with the EMA. This early dialogue is intended to contribute to the preparedness of an applicant and to increase EMA awareness and education of emerging therapies and technologies.

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 severe and sometimes life-threatening anemia, bone deformities and enlargement of the spleen. Standard treatment includes frequent, life-long blood transfusions. While lifesaving, these transfusions cause an excess of 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.

About Sickle Cell Disease

Sickle cell disease is the most common inherited blood disorder in the United States. Sickle cell disease is characterized 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. Patients with severe forms suffer from severe and sometimes life-threatening anemia, strokes, and damage to vital organs such as the lungs, spleen, kidney and liver. Standard treatment includes frequent, life-long blood transfusions. While lifesaving, these transfusions cause an excess of 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.

About Hereditary Hemochromatosis

Hereditary hemochromatosis (HH) is the most common genetic disease in Caucasians. HH is a disease characterized by a genetic mutation that results in a deficiency in the production of hepcidin, which is the body’s naturally occurring regulator of iron absorption and distribution. Without proper levels of hepcidin, excessive amounts of iron accumulate in the body and can lead to liver cirrhosis, liver cancer, heart disease and/or failure, dementia and diabetes. With no FDA-approved treatments for HH, patients are treated with iron chelators and phlebotomy, which do not address the underlying disease pathology, carry significant toxicity and/or adversely impact quality of life.

About LJPC-401

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

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

LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing. La Jolla’s Investigational New Drug Application (IND) has been accepted by the FDA, and La Jolla expects to release preliminary results from a Phase 1 study by the end of 2015.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH), beta thalassemia and sickle cell disease. LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

Forward-Looking Statement Safe Harbor

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing for the filing of an Investigational New Drug Application (IND), commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for the Company’s drug candidates; potential indications for which the Company’s drug candidates may be developed; and the Company’s ability to obtain the potential benefits of orphan drug designation for its drug candidates. 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/20150911005176/en/

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

Source: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company Announces Pricing of Underwritten Offering of Common Stock

La Jolla Pharmaceutical Company Announces Pricing of Underwritten Offering of Common Stock

SAN DIEGO–(BUSINESS WIRE)– La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the “Company” or “La Jolla”), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced the pricing of an underwritten offering of 2,550,000 shares of its common stock, offered at a price of $38.00 per share. La Jolla has granted the underwriters a 30-day option to purchase up to an additional 382,500 shares of common stock. The offering is expected to close on or about September 15, 2015, subject to customary closing conditions. Jefferies LLC and Cowen and Company, LLC are acting as joint book-running managers for the offering. Chardan Capital Markets, LLC, LifeSci Capital LLC and Noble Life Science Partners are acting as co-managers for the offering.

Gross offering proceeds will be $96,900,000 before deducting underwriting discounts and commissions and estimated offering expenses payable by La Jolla. La Jolla intends to use the net proceeds from the underwritten offering for general corporate purposes, including funding its ongoing and future clinical trials, and for general and administrative expenses.

The securities described above are being offered pursuant to a shelf registration statement (File No. 333-197092), including a base prospectus, which was declared effective by the United States Securities and Exchange Commission (the “SEC”) on July 11, 2014, and a related automatically effective registration statement filed pursuant to Rule 462(b) of the Securities Act of 1933 (File No. 333-206855). The specific terms of the offering are described in a prospectus supplement filed with the SEC in connection with the offering. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. The offering will be made only by means of the prospectus supplement and accompanying prospectus, copies of which may be obtained at the SEC’s website at www.sec.gov, or by request at Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, telephone: (877) 547-6340, e-mail: Prospectus_Department@Jefferies.com, or at Cowen and Company, LLC, c/o Broadridge Financial Services, Attention: Prospectus Department, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: (631) 274-2806, fax: (631) 254-7140.

This press release contains statements relating to the proposed offering that are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. Such statements involve risks and uncertainties, such as the risk that the conditions to the closing of the offering will not be satisfied. All forward-looking statements are based upon information available to La Jolla on the date the statements are first published. La Jolla undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH), beta thalassemia and sickle cell disease. 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.

View source version on businesswire.com: http://www.businesswire.com/news/home/20150910005574/en/

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 Proposed Underwritten Offering of Common Stock

La Jolla Pharmaceutical Company Announces Proposed Underwritten Offering of Common Stock

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 its intention to offer and sell shares of its common stock in an underwritten offering pursuant to its existing shelf registration statement. All of the shares in the proposed offering are to be sold by La Jolla.

Jefferies LLC is acting as the book-running manager for the offering. La Jolla intends to grant the underwriters a 30-day option to purchase additional shares of its common stock. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or the actual size or terms of the offering.

La Jolla intends to use the net proceeds from the underwritten offering for general corporate purposes, funding its ongoing and future clinical trials, general and administrative expenses and potential future acquisitions and other strategic purposes.

The securities described above are being offered pursuant to a shelf registration statement (File No. 333-197092), including a base prospectus, which was declared effective by the United States Securities and Exchange Commission (“SEC”) on July 11, 2014. The specific terms of the offering are described in a prospectus supplement to be filed with the SEC in connection with the offering. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. The offering will be made only by means of the prospectus supplement and accompanying prospectus, copies of which may be obtained at the SEC’s website at www.sec.gov, or by request at Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, telephone: (877) 547-6340, e-mail: Prospectus_Department@Jefferies.com.

This press release contains statements relating to the proposed offering that are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. Such statements involve risks and uncertainties, such as the risk that the conditions to the closing of the offering will not be satisfied. All forward-looking statements are based upon information available to La Jolla on the date the statements are first published. La Jolla undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH), beta thalassemia and sickle cell disease. 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.

View source version on businesswire.com: http://www.businesswire.com/news/home/20150909006680/en/

Contacts

La Jolla Pharmaceutical Company

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

President & Chief Executive Officer

Phone: (858) 207-4264

Email: gtidmarsh@ljpc.com

and

Dennis M. Mulroy

Chief Financial Officer

Phone: (858) 433-6839

Email: dmulroy@ljpc.com

Source: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company Receives Orphan Drug Designation for Two Novel Compounds for Fibrodysplasia Ossificans Progressiva

SAN DIEGO–(BUSINESS WIRE)– La Jolla Pharmaceutical Company(Nasdaq: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted La Jolla orphan drug designation for two novel compounds for fibrodysplasia ossificans progressiva (FOP).

The compounds that received orphan drug designation are small-molecule kinase inhibitors designed to selectively block a specific member of the bone morphogenetic protein (BMP) type-I receptor family, ALK2. La Jolla recently entered a worldwide, exclusive license agreement with Vanderbilt University covering this technology. The seven members of the BMP type-I receptor family, activin receptor-like kinase (ALK) 1-7, play critical roles in human development and physiology. In turn, the improper activation of these receptor pathways is responsible for a wide range of disease conditions.

FOP is a rare genetic disorder where the body turns muscle into bone. FOP is caused by a genetic mutation in ALK2 that results in excessive signaling of this pathway. In early childhood, afflicted individuals develop soft tissue swellings that transform into bone. Development of such lesions is exacerbated by trauma, and surgical intervention leads to dramatic and explosive new bone growth. The median survival of FOP patients is approximately 40 years, and death often results from complications of thoracic insufficiency syndrome, which is the inability of the thorax to support normal respiration.

“There are no known treatment options available for patients suffering from FOP, and the orphan drug designations recognize the significant unmet need that exists within this disease,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We look forward to further collaborating with our colleagues at Vanderbilt University to find a potential treatment for these patients in need.”

About Orphan Drug Designation

Orphan drug designation is granted by the FDA Office of Orphan Products Development to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity if the drug is the first of its type approved for the specified indication or if it demonstrates superior safety, efficacy, or a major contribution to patient care versus another drug of its type previously granted the designation for the same indication. The designation also provides the drug developer with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and a waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis and beta thalassemia. LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

Forward-Looking Statement Safe Harbor

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing for the filing of an Investigational New Drug Application (IND), commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for the Company’s drug candidates; potential indications for which the Company’s drug candidates may be developed; and the Company’s ability to obtain the potential benefits of orphan drug designation for its drug candidates. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

Contacts

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

 

Source: La Jolla Pharmaceutical Company

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

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

SAN DIEGO & NASHVILLE, Tenn.–(BUSINESS WIRE)– La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, and Vanderbilt University (Vanderbilt) today announced an exclusive, worldwide research and license agreement covering Vanderbilt’s research program and intellectual property rights relating to small-molecule kinase inhibitors designed to selectively block specific members of the bone morphogenetic protein (BMP) type-I receptor family.

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

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

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

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

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

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

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

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

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis and beta thalassemia. LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

About Vanderbilt University Medical Center

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

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

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

Forward-Looking Statement Safe Harbor

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing for the filing of an Investigational New Drug Application, commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for the Company’s drug candidates; and potential indications for which the Company’s drug candidates may be developed. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

Contacts

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

 

Source: La Jolla Pharmaceutical Company

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

SAN DIEGO–(BUSINESS WIRE)– La Jolla Pharmaceutical Company(Nasdaq: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company’s Investigational New Drug Application (IND) for LJPC-401, La Jolla’s novel formulation of hepcidin. La Jolla expects to release preliminary results from a Phase 1 clinical trial of LJPC-401 by the end of 2015.

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

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

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

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

About LJPC-401

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

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

LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing. La Jolla’s Investigational New Drug Application (IND) has been accepted by the FDA, and La Jolla expects to release preliminary results from a Phase 1 study by the end of 2015.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis and beta thalassemia. LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

Forward-Looking Statement Safe Harbor

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing for the filing of an Investigational New Drug Application (IND), commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for LJPC-501, LJPC-401, LJPC-30Sa and LJPC-30Sb; potential indications for which LJPC-501, LJPC-401, LJPC-30Sa and LJPC-30Sb may be developed; and the expected duration over which the Company’s cash balances will fund its operations. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

Contacts

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

 

Source: La Jolla Pharmaceutical Company