La Jolla Pharmaceutical Company Announces Special Protocol Assessment for Planned Phase 3 Trial of LJPC-501 in Catecholamine-Resistant Hypotension

SAN DIEGO–(BUSINESS WIRE)– La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced that it has reached agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assesment (SPA) for its Phase 3 clinical trial of LJPC-501 for the treatment of catecholamine-resistant hypotension (CRH). In accordance with the SPA, the primary efficacy endpoint for this Phase 3 registration trial will be increase in blood pressure. La Jolla plans to initiate this multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial pursuant to the approved SPA in the first quarter of 2015.

“We are very pleased with the FDA’s agreement on our clinical trial design and planned analysis, which clearly defines what we believe to be a feasible path for the development and registration of LJPC-501,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “The prognosis for patients suffering from CRH is very poor, with less than 50% of these patients surviving one month from diagnosis. We believe that LJPC-501 has the potential to reverse hypotension and, therefore, provide a significant benefit to these patients.”

About Special Protocol Assessments

A Special Protocol Assessment is a written agreement between a sponsor and the U.S. Food and Drug Administration on the design, execution and analysis for a clinical trial that may form the basis of a new drug application, or NDA. Final marketing approval depends upon the efficacy results, the safety profile and an evaluation of the risk/benefit of treatment demonstrated in the Phase 3 clinical program.

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of angiotensin II. Angiotensin II, the major bioactive component of the renin-angiotensin system, serves as one of the body’s central regulators of blood pressure. La Jolla is developing LJPC-501 for the treatment of catecholamine-resistant hypotension, or CRH, which is an acute, life-threatening condition in which blood pressure drops to dangerously low levels and is poorly responsive to current treatments. Angiotensin II has been shown to raise blood pressure in a randomized, placebo-controlled clinical trial in CRH, as well as animal models of hypotension. In October 2014, La Jolla presented positive data from a preclinical study of LJPC-501 for the treatment of CRH. In February 2015, La Jolla reached agreement with the FDA on a Special Protocol Assessment, or SPA, for its Phase 3 clinical trial of LJPC-501 for the treatment of CRH, in which agreement was reached that blood pressure can be the primary endpoint for approval. La Jolla’s Phase 3 clinical trial of LJPC-501 for the treatment of CRH is expected to begin in the first quarter of 2015. La Jolla has submitted an Orphan Drug Designation application to the FDA for LJPC-501 for the treatment of CRH.

La Jolla is also developing LJPC-501 for hepatorenal syndrome, or HRS. HRS is a life-threatening form of progressive renal failure in patients with liver cirrhosis or fulminant liver failure. In these patients, the diseased liver secretes vasodilator substances (e.g., nitric oxide and prostaglandins) into the bloodstream that cause under-filling of blood vessels. This low blood pressure state causes a reduction in blood flow to the kidneys. As a means to restore systemic blood pressure, the kidneys induce both sodium and water retention, which contribute to ascites, a major complication associated with HRS. Studies have shown that LJPC-501 may improve renal function in patients with conditions similar to HRS. La Jolla is currently conducting a Phase 1/2 clinical trial of LJPC-501 in HRS. The Phase 1/2 clinical trial is currently enrolling patients.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has four product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension and hepatorenal syndrome. GCS-100 is La Jolla’s first-in-class galectin-3 inhibitor for the potential treatment of chronic kidney disease. LJPC-1010, La Jolla’s second-generation galectin-3 inhibitor, is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis and other diseases characterized by tissue fibrosis. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload. For more information on La Jolla, please visit www.ljpc.com.

Forward-Looking Statement Safe Harbor

This document contains “forward-looking statements,” as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to the Company’s expectations regarding future events or its future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, any one of which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site http://www.sec.gov. These risks include, but are not limited to, risks relating to the future development of LJPC-501 for the treatment of CRH and the success of future development activities for this and other drug development programs sponsored by the Company; the ability of the Company to obtain orphan drug status for LJPC-501; the ability of the Company to use the planned Phase 3 study of LJPC-501 in CRH as a registration study; and the success of other potential indications for which this and other drug candidates may be developed. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

Contacts

La Jolla Pharmaceutical Company

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

President & Chief Executive Officer

(858) 207-4264

gtidmarsh@ljpc.com

or

Chester S. Zygmont, III

Senior Director of Finance

(858) 207-4262

czygmont@ljpc.com

Source: La Jolla Pharmaceutical Company

View this news release online at:

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

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

SAN DIEGO, CA – February 4, 2015La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced that George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer, will provide a corporate overview at the 17th Annual BIO CEO & Investor Conference taking place February 9-10 in New York City.

 

17th Annual BIO CEO & Investor Conference Presentation Details

 

Date:               Monday, February 9, 2015

Time:               5:30 pm Eastern Time

Location:         Louis XVI Room @ The Waldorf Astoria New York

Webcast:         LJPC Webcast Link

 

About La Jolla Pharmaceutical Company

 

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has four product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension and hepatorenal syndrome. GCS-100 is La Jolla’s first-in-class galectin-3 inhibitor for the potential treatment of chronic kidney disease. LJPC-1010, La Jolla’s second-generation galectin-3 inhibitor, is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis and other diseases characterized by tissue fibrosis. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload. For more information on La Jolla, please visit www.ljpc.com.

 

Company Contact

 

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

President & Chief Executive Officer

La Jolla Pharmaceutical Company

Phone: (858) 207-4264

Email: gtidmarsh@ljpc.com

 

and

 

Chester S. Zygmont, III

Senior Director of Finance

La Jolla Pharmaceutical Company

Phone: (858) 207-4262

Email: czygmont@ljpc.com

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

Dec. 9, 2014 21:01 UTC

License Covers Intellectual Property Relating to LJPC-501

SAN DIEGO & WASHINGTON–(BUSINESS WIRE)– La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, and the George Washington University (GW), today announced that La Jolla and GW have entered into an exclusive worldwide license agreement for GW intellectual property rights covering the use of angiotensin II for the therapeutic treatment of patients with hypotension and shock. La Jolla plans to initiate a phase 3 registration program of LJPC-501, La Jolla’s proprietary formulation of angiotensin II, in catecholamine-resistant hypotension (CRH) in the first quarter of 2015.

“We are very pleased to gain access to this additional intellectual property covering LJPC-501,” said George F. Tidmarsh, M.D., Ph.D., President and CEO of La Jolla. “The prognosis for patients suffering from CRH is very poor, with less than 50% of these patients surviving one month from diagnosis. We believe that LJPC-501 has the potential to reverse hypotension and, therefore, provide a significant benefit to these patients.”

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

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

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of angiotensin II. Angiotensin II, the major bioactive product of the renin-angiotensin system, serves as one of the body’s central regulators of blood pressure. La Jolla is developing LJPC-501 for the treatment of catecholamine-resistant hypotension, or CRH, which is an acute, life-threatening condition in which blood pressure drops to dangerously low levels and is poorly responsive to current treatments. Angiotensin II has been shown to raise blood pressure in a randomized, placebo-controlled clinical trial in CRH, as well as animal models of hypotension. In October 2014, La Jolla presented positive data from a preclinical study of LJPC-501 in the treatment of CRH. In June 2014, La Jolla had a meeting with the U.S. Food and Drug Administration, or FDA, in which agreement was reached that blood pressure can be the primary endpoint for approval in CRH. As a result of this meeting, La Jolla plans to initiate a Phase 3 registration clinical trial of LJPC-501 in CRH in the first quarter of 2015. La Jolla believes CRH to be an orphan indication, and, therefore, in July 2014, La Jolla submitted an Orphan Drug Designation application to the FDA for LJPC-501 in the treatment of CRH.

La Jolla is also developing LJPC-501 for hepatorenal syndrome, or HRS. HRS is a life-threatening form of progressive renal failure in patients with liver cirrhosis or fulminant liver failure. In these patients, the diseased liver secretes vasodilator substances (e.g., nitric oxide and prostaglandins) into the bloodstream that cause under-filling of blood vessels. This low blood pressure state causes a reduction in blood flow to the kidneys. As a means to restore systemic blood pressure, the kidneys induce both sodium and water retention, which contribute to ascites, a major complication associated with HRS. Studies have shown that LJPC-501 may improve renal function in patients with conditions similar to HRS. La Jolla is currently conducting a Phase 1/2 clinical trial of LJPC-501 in HRS, and, in August 2014, La Jolla enrolled its first patient in this trial. The Phase 1/2 clinical trial is currently enrolling patients.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has four product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension (CRH) and hepatorenal syndrome (HRS). GCS-100 is La Jolla’s first-in-class galectin-3 inhibitor for the potential treatment of chronic kidney disease (CKD). LJPC-1010, La Jolla’s second-generation galectin-3 inhibitor, is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis (NASH) and other diseases characterized by tissue fibrosis. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload. For more information on La Jolla, please visit www.ljpc.com.

About the George Washington University

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

Forward-Looking Statement Safe Harbor

This document contains “forward-looking statements,” as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to the Company’s expectations regarding future events or its future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, any one of which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site http://www.sec.gov. These risks include, but are not limited to, risks and uncertainties relating to: the timing for the commencement of clinical studies and the anticipated timing for completion of such studies; whether patent applications licensed from the George Washington University will result in issued patents and whether such patents, if issued, would cover LJPC-501; the success of future development activities for LJPC-501, GCS-100, LJPC-1010 and LJPC-401; estimated market sizes and the ability to successfully receive Orphan Drug designation for LJPC-501; and potential indications for which LJPC-501, GCS-100, LJPC-1010 and LJPC-401 may be developed. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

Contacts

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

Source: La Jolla Pharmaceutical Company

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

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

 

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

 

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

 

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

 

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

 

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

 

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

 

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

 

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

 

 

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

 

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

 

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

 

About GCS-100

 

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

 

About La Jolla Pharmaceutical Company

 

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has four product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension (CRH) and hepatorenal syndrome (HRS). GCS-100 is La Jolla’s first-in-class galectin-3 inhibitor for the potential treatment of chronic kidney disease (CKD). LJPC-1010, La Jolla’s second-generation galectin-3 inhibitor, is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis (NASH) and other diseases characterized by tissue fibrosis. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload. For more information on La Jolla, please visit http://www.ljpc.com.

 

About the American Society of Nephrology and Kidney Week

 

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

 

Forward-Looking Statement Safe Harbor

 

This document and the poster presentation referred to herein contain “forward-looking statements,” as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to the Company’s expectations regarding future events or the Company’s future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, any one of which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site http://www.sec.gov. These risks include, but are not limited to, risks relating to the future development of GCS-100 for the treatment of CKD and the success of future development activities for this and other drug development programs sponsored by the Company. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

 


 

Company Contact

 

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

President & Chief Executive Officer

La Jolla Pharmaceutical Company

Phone: (858) 207-4264

Email: GTidmarsh@ljpc.com

 

and

 

Chester S. Zygmont, III

Senior Director of Finance

La Jolla Pharmaceutical Company

Phone: (858) 207-4262

Email: czygmont@ljpc.com

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

SAN DIEGO, CA – November 12, 2014 – La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today reported third quarter and year-to-date 2014 financial results and highlighted recent corporate progress and near-term milestones.

 

Recent Corporate Highlights

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

 

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

 

 

Near-Term Corporate Milestones

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

 

Results of Operations

 

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

 

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

 

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

 

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

 

About La Jolla Pharmaceutical Company

 

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has four product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension (CRH) and hepatorenal syndrome (HRS). GCS-100 is La Jolla’s first-in-class galectin-3 inhibitor for the potential treatment of chronic kidney disease (CKD). LJPC-1010, La Jolla’s second-generation galectin-3 inhibitor, is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis (NASH) and other diseases characterized by tissue fibrosis. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of iron overload. For more information on La Jolla, please visit

http://www.ljpc.com.

 

 

Forward Looking Statement Safe Harbor

 

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (“SEC”), all of which are available free of charge on the SEC’s web site http://www.sec.gov. These risks include, but are not limited to, risks relating to the timing for the commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for LJPC-501, GCS-100, LJPC-1010 and LJPC-401; estimated market sizes and the ability to successfully receive Orphan Drug designation for LJPC-501; and potential indications for which LJPC-501, GCS-100, LJPC-1010 and LJPC-401 may be developed. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

 

 

(financial tables follow)

 

LA JOLLA PHARMACEUTICAL COMPANY

Unaudited Condensed Statements of Operations and Comprehensive Loss

(in thousands, except per-share amounts)

 

 

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

 

 

LA JOLLA PHARMACEUTICAL COMPANY

Condensed Balance Sheets

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

 

 

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

 

 

Company Contact

 

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

President & Chief Executive Officer

La Jolla Pharmaceutical Company

Phone: (858) 207-4264

Email: GTidmarsh@ljpc.com

 

and

 

Chester S. Zygmont, III

Senior Director of Finance

La Jolla Pharmaceutical Company

Phone: (858) 207-4262

Email: czygmont@ljpc.com

La Jolla Pharmaceutical Company Announces Data Presentation at the American Society of Nephrology Kidney Week

Oct. 23, 2014 20:01 UTC

La Jolla Pharmaceutical Company Announces Data Presentation at the American Society of Nephrology Kidney Week

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 an abstract of the results for the Company’s Phase 2 clinical trial of GCS-100 in chronic kidney disease (CKD) has been selected by the American Society of Nephrology’s (ASN) Program Committee for poster presentation at the Kidney Week Annual Meeting. Kidney Week will be held November 11-16, 2014 in Philadelphia, Pennsylvania.

“We are delighted that results from our Phase 2 study of GCS-100 in CKD have been selected by the ASN for presentation,” said George Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “More than 13,000 kidney professionals are expected to attend Kidney Week this year, and it is well established as the world’s premier nephrology meeting.”

La Jolla’s abstract, #3724 “Galectin-3 Inhibition with GCS-100 Improves eGFR in Patients with Chronic Kidney Disease, will be presented in a Poster Session titled Glomerular and Tubulointerstitial Disease: Clinical Trials and Outcomes, on Thursday, November 13, 2014 from 10:00am to 12:00pm Eastern Standard Time. The abstract is reprinted below:

Pablo E. Pergola, MD, PhD1, Geoffrey Block, MD2, Bhupinder Singh, MD3, George Fadda, MD4, Robert Cohen, DO3, William Durham, MD5, James Tumlin, MD6, George Tidmarsh, MD, PhD7, James Rolke7

1Clinical Advancement Center, San Antonio, TX

2Denver Nephrology, Denver, CO

3Southwest Kidney Institute, Tempe, AZ

4Balboa Nephrology Medical Group, La Mesa, CA

5Mountain Kidney and Hypertension Associates, Ashville, NC

6SouthEast Renal Research Institute, Chattanooga, TN

7La Jolla Pharmaceutical Company, San Diego, CA

Galectin-3 inhibition with GCS-100 Improves eGFR in Patients with Chronic Kidney Disease

INTRODUCTION:

Galectin-3 has been implicated in interstitial and glomerular fibrosis resulting in progressive loss of kidney function.

METHODS:

GCS-100, a polysaccharide inhibitor of galectin-3, was evaluated in CKD stages 3b/4 in a multicenter, randomized, placebo-controlled Phase 2 study. 121 consenting adults received placebo or GCS-100 at doses of 1.5 or 30 mg/m2 IV weekly for 8 weeks followed by a 4 week observation period. The primary endpoint was the change in eGFR from baseline to end of treatment versus placebo. Both T-test and ANCOVA were used for analyses.

RESULTS:

Demographics: 117 patients (65 Stage 4) completed treatment (mean age 65; 43 female). Baseline eGFR was 29.2 ml/min/1.73m2 ± 9.00 (SD). Diabetes and HTN were the most common etiologies of CKD.

Efficacy: In the 1.5 mg/m2 group, a change in eGFR of +1.26 ±0.77 versus placebo (-0.58±0.46) was observed (p=0.045). When restricted to diabetic etiology, for 1.5 mg/m2 the change in eGFR was 2.33 ± 1.13 versus placebo (-0.53±0.56; p=0.028). Galectin-3 and K+ were significantly reduced (p=0.07 each). Uric acid and BUN were significantly reduced vs. baseline (p=0.07, 0.08 respectively). No significant changes in eGFR, galectin-3, K+, uric acid or BUN were observed among those receiving 30 mg/m2.

Safety: Four SAEs occurred, 2 in the placebo, 2 in the 30 mg/m2 group and none at 1.5 mg/m2. None of the SAEs were drug-related.

DISCUSSION:

Short term therapy with the galectin-3 inhibitor, GCS-100 at 1.5 mg/m2, resulted in small but significant improvement in eGFR. If future, long-term studies confirm these findings, GCS-100 could be used as a disease-modifying agent to slow and potentially reverse the renal fibrosis common in CKD.

About the American Society of Nephrology and Kidney Week

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

About GCS-100

GCS-100 is a complex polysaccharide derived from pectin that binds to, and blocks the activity of, galectin-3. Over-expression of galectin-3 has been implicated in a number of human diseases, including chronic organ failure and cancer. La Jolla is developing GCS-100 for the treatment of chronic kidney disease (CKD). The Company recently completed a multicenter, randomized, placebo-controlled, Phase 2 study in CKD patients in which treatment with GCS-100 resulted in a statistically significant improvement in estimated glomerular filtration rate (eGFR) compared to placebo.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has four product candidates in development. LJPC-501 is a proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension (CRH) and hepatorenal syndrome (HRS). GCS-100 is a first-in-class inhibitor of galectin-3 for the potential treatment of chronic kidney disease (CKD). LJPC-1010 is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis (NASH). LJPC-401 is a novel formulation of the natural peptide hepcidin for the potential treatment of iron overload. For more information on La Jolla, please visit http://www.ljpc.com.

Forward-Looking Statement Safe Harbor

This document and the abstract referred to herein contain “forward-looking statements,” as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to our expectations regarding future events or our future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, any one of which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site http://www.sec.gov. These risks include, but are not limited to, risks relating to the future development of LJPC-501 for the treatment of CRH and HRS, GCS-100 for the treatment of CKD, LJPC-1010 for the treatment of NASH, and LJPC-401 for the treatment of iron overload and the success of future development activities for these and other drug development programs sponsored by the Company; and potential indications for which these drug candidates may be developed. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

Contacts

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

View this news release online at:

http://www.businesswire.com/news/home/20141023006448/en

La Jolla Pharmaceutical Company to Provide Corporate Overview at the Rodman & Renshaw 16th Annual Global Investment Conference

September 08, 2014 08:00 AM Eastern Daylight Time

Rodman & Renshaw Global Investment Conference 2014

SAN DIEGO–(BUSINESS WIRE)–La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the “Company” or “La Jolla”), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced that George Tidmarsh, M.D., Ph.D., President and Chief Executive Officer, will provide a corporate overview at the Rodman & Renshaw 16th Annual Global Investment Conference taking place September 8-10 in New York City.

Rodman & Renshaw 16th Annual Global Investment Conference Presentation Details

Date: Wednesday, September 10, 2014

Time: 11:40 am Eastern Time

Location: Holmes I Salon @ New York Palace Hotel

Webcast: LJPC Webcast Link

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. LJPC-501, La Jolla’s lead product candidate, is a proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension and hepatorenal syndrome. GCS-100, La Jolla’s second product candidate, is a first-in-class inhibitor of galectin-3 for the potential treatment of chronic kidney disease. LJPC-1010, La Jolla’s third product candidate, is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis. LJPC-401, La Jolla’s fourth product candidate, is a natural peptide for the potential treatment of iron overload. For more information on La Jolla, please visit http://www.ljpc.com.

Contacts

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

La Jolla Pharmaceutical Company Announces First Patient Enrolled in Clinical Trial of LJPC-501 in Hepatorenal Syndrome

SAN DIEGO–(BUSINESS WIRE)–La Jolla Pharmaceutical Company (NASDAQ: LJPC), 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 enrolled in the Phase 1/2 clinical trial of LJPC-501 for the treatment of type 1 and type 2 hepatorenal syndrome (HRS). HRS is a life-threatening form of progressive renal failure in patients with liver cirrhosis or fulminant liver failure.

“We are very grateful to the investigator, the patient and the patient’s family,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “We believe that LJPC-501, which helps the kidneys balance body fluids and electrolytes, may improve kidney function in patients with HRS. For many patients with HRS, reduced kidney function is the major debilitating aspect of their illness.”

The clinical trial is an open-label, multi-center study of LJPC-501 in patients with type 1 and/or type 2 HRS.

The trial is designed to enroll up to 15 patients with type 1 or type 2 HRS. The primary endpoint is safety and tolerability. Secondary endpoints include determining the maximum tolerated dose and the effects of LJPC-501 on serum creatinine through 5 days of treatment. Patients will receive LJPC-501 at titrated doses, with a starting range from 1 to 100 ng/kg/min, by continuous infusion on days 1 through 5. Dose titrations will occur every 4 hours until a mean arterial pressure of 110 mmHg is reached, maximum urine output is achieved or a dose of 100 ng/kg/min is achieved. Dosing will then continue at such maximum dose through day 5.

About Hepatorenal Syndrome

Hepatorenal syndrome (HRS) is a life-threatening form of progressive renal failure in patients with liver cirrhosis. In these patients, the diseased liver secretes vasodilator substances (e.g., nitric oxide and prostaglandins) into the bloodstream that cause under-filling of blood vessels. This low blood pressure state causes a reduction in blood flow to the kidneys. As a means to restore systemic blood pressure, the kidneys induce both sodium and water retention, which contribute to ascites, a major complication associated with HRS.

About LJPC-501

LJPC-501, a proprietary formulation of angiotensin II, is a peptide agonist of the renin-angiotensin system that acts to stabilize 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 and is poorly responsive to current treatments. LJPC-501 has been shown to raise blood pressure in a randomized, placebo-controlled clinical trial in CRH, as well as animal models of hypotension. La Jolla plans to initiate a Phase 3 registration program with LJPC-501 in the treatment of CRH as the result of a recent meeting with the U.S. Food and Drug Administration (FDA) at which agreement was reached that blood pressure could serve as an appropriate primary endpoint for approval. Due to the estimated size of the patient population in the United States for this indication, La Jolla has filed an application for Orphan Drug status for LJPC-501. Studies have shown that LJPC-501 may also improve renal function in patients with hepatorenal syndrome (HRS).

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. LJPC-501, La Jolla’s lead product candidate, is a proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension and hepatorenal syndrome. GCS-100, La Jolla’s second product candidate, is a first-in-class inhibitor of galectin-3 for the potential treatment of chronic kidney disease. LJPC-1010, La Jolla’s third product candidate, is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis. LJPC-401, La Jolla’s fourth product candidate, is a natural peptide for the potential treatment of iron overload. For more information on La Jolla, please visit http://www.ljpc.com.

Forward-Looking Statement Safe Harbor

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or our future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (“SEC”), all of which are available free of charge on the SEC’s web site http://www.sec.gov. These risks include, but are not limited to, risks relating to the timing for the commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for LJPC-501, GCS-100, LJPC-1010 and LJPC-401; estimated market sizes and the ability to successfully receive Orphan Drug designation for LJPC-501; and potential indications for which LJPC-501, GCS-100, LJPC-1010 and LJPC-401 may be developed. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

Contacts

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

La Jolla Pharmaceutical Company Announces Second Quarter and Year-to-Date 2014 Financial Results

SAN DIEGO–(BUSINESS WIRE)– La Jolla Pharmaceutical Company (NASDAQ: LJPC), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today reported second quarter and year-to-date 2014 financial results and highlighted recent corporate progress.

Recent Corporate Highlights

  • On July 28, 2014, La Jolla closed an underwritten public offering of approximately 5.4 million shares of common stock, which includes a portion of the overallotment option, at a public offering price of $10.50 per share. The Company received total net proceeds of approximately $53.1 million.
  • On July 15, 2014, La Jolla announced positive data from a preclinical study of LJPC-1010, its oral galectin-3 inhibitor, in the treatment of nonalcoholic steatohepatitis (NASH).
  • On July 8, 2014, La Jolla announced that it plans to initiate a Phase 3 registration program with LJPC-501 in the treatment of catecholamine-resistant hypotension (CRH).
  • On June 27, 2014, La Jolla joined the Russell Microcap Index, as a result of the annual Russell Index Reconstitution.
  • On June 4, 2014, La Jolla announced the completion of enrollment of its Phase 2 extension trial of GCS-100 in Chronic Kidney Disease (CKD).

“We have had a very exciting year so far, highlighted by the announcement of our plans for our LJPC-501 Phase 3 registration program, the positive top-line results from our Phase 2 clinical trial of GCS-100 in severe CKD and the close of our recent financing,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “During the second half of 2014, we plan to move our new LJPC-501 CRH Phase 3 program forward, continue the progress of our GCS-100 CKD program and advance our other new programs.”

Results of Operations

At June 30, 2014, La Jolla had $3.9 million in cash, as compared to $8.6 million in cash at December 31, 2013. As of August 1, 2014, La Jolla had approximately $56.6 million in cash, which includes the net proceeds of the public offering of common stock, which closed on July 28, 2014.

La Jolla’s comprehensive net loss attributable to common shareholders for the three and six months ended June 30, 2014 was $4.3 million and $9.4 million, or $0.63 per share and $1.16 per share, respectively, compared to a comprehensive net loss attributable to common shareholders of $3.7 million and $7.9 million, or $6.77 per share and $17.57 per share, respectively, for the same periods in 2013.

The increase in comprehensive net loss attributable to common shareholders was primarily due to increases in research and development expenses related to the GCS-100 Phase 2 program in the treatment of CKD, as well as preclinical work on LJPC-1010 and LJPC-401.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. LJPC-501, La Jolla’s lead product candidate, is a proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension and hepatorenal syndrome. GCS-100, La Jolla’s second product candidate, is a first-in-class inhibitor of galectin-3 for the potential treatment of chronic kidney disease. LJPC-1010, La Jolla’s third product candidate, is a more potent and purified derivative of GCS-100 that can be delivered orally for the potential treatment of nonalcoholic steatohepatitis. LJPC-401, La Jolla’s fourth product candidate, is a natural peptide for the potential treatment of iron overload. For more information on La Jolla, please visit http://www.ljpc.com.

About LJPC-501

LJPC-501, a proprietary formulation of angiotensin II, is a peptide agonist of the renin-angiotensin system that acts to stabilize 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 and is poorly responsive to current treatments. LJPC-501 has been shown to raise blood pressure in a randomized, placebo-controlled clinical trial in CRH, as well as animal models of hypotension. La Jolla plans to initiate a Phase 3 registration program with LJPC-501 in the treatment of CRH as the result of a recent meeting with the U.S. Food and Drug Administration (FDA) at which agreement was reached that blood pressure could serve as an appropriate primary endpoint for approval. Due to the estimated size of the patient population in the United States for this indication, La Jolla has filed an application for Orphan Drug status for LJPC-501. Studies have shown that LJPC-501 may also improve renal function in patients with hepatorenal syndrome (HRS).

About GCS-100

GCS-100 is a complex polysaccharide derived from pectin that binds to and blocks the activity of galectin-3, a type of galectin. Over-expression of galectin-3 has been implicated in a number of human diseases, including chronic organ failure and cancer. La Jolla is developing GCS-100 for the treatment of chronic kidney disease (CKD). In March of 2014, La Jolla announced positive top-line results from its randomized, placebo-controlled Phase 2 clinical trial of GCS-100 in severe CKD, in which the predefined primary efficacy endpoint was met. La Jolla completed enrollment for a Phase 2 extension clinical trial of GCS-100 in patients with severe CKD in June of 2014 and plans to initiate a Phase 2b clinical trial in late 2014.

About LJPC-1010

LJPC-1010 is a more potent and purified derivative of GCS-100 that can be delivered orally. The Company is developing LJPC-1010 for the treatment of nonalcoholic steatohepatitis (NASH). NASH is the more serious form of nonalcoholic fatty liver disease, or NAFLD, which can lead to liver failure. In July of 2014, La Jolla announced positive preclinical data of LJPC-1010 in NASH. La Jolla plans to file an Investigational New Drug Application (IND) with the U.S. Food and Drug Administration (FDA) and initiate a Phase 1 clinical trial of LJPC-1010 in NASH in early 2015.

About LJPC-401

LJPC-401 is La Jolla’s formulation of hepcidin, which is an endogenous peptide hormone that controls and regulates iron metabolism. La Jolla licensed intellectual property covering the composition of hepcidin from INSERM in February of 2014. The active form of hepcidin is a 25 amino acid protein that serves as a master regulator of iron metabolism. Hepcidin synthesis in the liver is regulated by multiple signals, including iron stores, erythropoietic activity (the production of red blood cells) and inflammatory cytokines. La Jolla is currently in the preclinical development stage with LJPC-401 and expects to file an IND and commence a Phase 1 clinical trial of LJPC-401 in iron overload in 2015.

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 our future results of operations. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause actual results to be materially different from these forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (“SEC”), all of which are available free of charge on the SEC’s web site http://www.sec.gov. These risks include, but are not limited to, risks relating to the timing for the commencement of clinical studies and the anticipated timing for completion of such studies; the success of future development activities for LJPC-501, GCS-100, LJPC-1010 and LJPC-401; estimated market sizes and the ability to successfully receive Orphan Drug designation for LJPC-501; and potential indications for which LJPC-501, GCS-100, LJPC-1010 and LJPC-401 may be developed. Subsequent written and oral forward-looking statements attributable to the Company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the Company’s reports filed with the SEC. The Company expressly disclaims any intent to update any forward-looking statements.

LA JOLLA PHARMACEUTICAL COMPANY

Unaudited Condensed Statements of Operations and Comprehensive Loss

(in thousands, except per-share amounts)

Three Months Ended

June 30,

Six Months Ended

June 30,

2014

2013

2014

2013

Expenses:

Research and development

$

1,597

$

700

$

3,593

$

1,355

General and administrative

2,689

2,463

5,823

6,011

Total expenses

4,286

3,163

9,416

7,366

Loss from operations

(4,286

)

(3,163

)

(9,416

)

(7,366

)

Other income:

Other income, net

2

1

4

2

Net loss and comprehensive loss

(4,284

)

(3,162

)

(9,412

)

(7,364

)

Preferred stock dividends earned

(562

)

(562

)

Net loss attributable to common shareholders

$

(4,284

)

$

(3,724

)

$

(9,412

)

$

(7,926

)

Basic and diluted net loss per share

$

(0.63

)

$

(6.77

)

$

(1.16

)

$

(17.57

)

Shares used in computing basic and diluted net loss per share

6,836

550

8,122

451

LA JOLLA PHARMACEUTICAL COMPANY

Condensed Balance Sheets

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

June 30,
2014

December 31,

2013

(Unaudited)

ASSETS:

Current assets:

Cash and cash equivalents

$

3,888

$

8,629

Restricted cash

37

37

Prepaid expenses and other current assets

148

43

Total current assets

4,073

8,709

Equipment and furnishings, net

89

38

Total assets

$

4,162

$

8,747

LIABILITIES AND SHAREHOLDERS EQUITY:

Current liabilities:

Accounts payable

$

917

$

834

Accrued expenses

58

187

Accrued payroll and related expenses

94

73

Total current liabilities

1,069

1,094

Shareholders’ equity:

Common stock, $0.0001 par value; 12,000,000,000 shares authorized, 9,838,298 and 4,404,407 shares issued and outstanding at June 30, 2014 and December 31, 2013, respectively

4

4

Series C-12 Convertible Preferred Stock, $0.0001 par value; 11,000 shares authorized, 3,917 and 7,016 shares issued and outstanding at June 30, 2014 and December 31, 2013, respectively

3,917

7,016

Series F Convertible Preferred Stock, $0.0001 par value; 10,000 shares authorized, 2,798 and 3,250 shares issued and outstanding at June 30, 2014 and December 31, 2013, respectively

2,798

3,250

Additional paid-in capital

471,087

462,684

Accumulated deficit

(474,713

)

(465,301

)

Total shareholders’ equity

3,093

7,653

Total liabilities and shareholders’ equity

$

4,162

$

8,747

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
Chester S. Zygmont, III
Senior Director of Finance
(858) 207-4262
czygmont@ljpc.com

Source: La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company Closes Offering of Common Stock

July 28, 2014 06:29 PM Eastern Daylight Time

SAN DIEGO–(BUSINESS WIRE)–La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the “Company” or “La Jolla”), a leader in the development of therapeutics targeting significant unmet life-threatening diseases, today announced the closing of its underwritten public offering of 5,395,000 shares of common stock at a public offering price of $10.50 per share, which includes the partial exercise of the underwriters’ option to purchase up to an additional 720,000 shares of common stock. Gross offering proceeds are approximately $56.6 million, including proceeds from the partial 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 acted as sole book-runner for the offering. Chardan Capital Markets, LLC, H.C. Wainwright & Co., LLC, LifeSci Capital, LLC and Noble Financial Group, Inc. 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. 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.

Contacts

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