UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
_______________________
FORM 8-K
_______________________
CURRENT REPORT
PURSUANT TO SECTION 13 OR 15(D) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of report (Date of earliest event reported): November 12, 2014
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LA JOLLA PHARMACEUTICAL COMPANY
(Exact name of registrant as specified in its charter)
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California | 1-36282 | 33-0361285 |
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(State or other jurisdiction of incorporation or organization) | (Commission File Number) | (I.R.S. Employer Identification No.) |
4660 La Jolla Village Drive, Suite 1070, San Diego, California 92122
(Address of Principal Executive Offices) (Zip Code)
Registrant’s telephone number, including area code: (858) 207-4264
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Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below): |
o | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
o | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
o | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
o | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
ITEM 2.02 Results of Operations and Financial Condition.
On November 12, 2014, La Jolla Pharmaceutical Company (the "Company") issued a press release announcing its financial results for the three and nine months ended September 30, 2014. A copy of the press release is furnished as Exhibit 99.1. The press release should be read in conjunction with the note regarding forward looking statements, which is included in the text of the press release.
The information in this Item 2.02 and in Exhibit 99.1 will not be treated as "filed" for the purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), or otherwise subject to the liabilities of that section. This information will not be incorporated by reference into any filing under the Securities Act of 1933, as amended, or into another filing under the Exchange Act, unless that filing expressly incorporates this information by reference.
ITEM 8.01 Other Events.
On November 13, 2014, detailed results from the Company's Phase 2 study of GCS-100 in chronic kidney disease were presented at a poster session at the American Society of Nephrology's ("ASN") Kidney Week Annual Meeting. A copy of the poster from the session is attached hereto as Exhibit 99.2 and is also posted on the Company’s website at http://www.ljpc.com. In connection with the poster and data presented, the Company issued a press release on November 12, 2014. A copy of the press release is attached hereto as Exhibit 99.3.
ITEM 9.01. FINANCIAL STATEMENTS AND EXHIBITS.
(d) Exhibits. The following exhibits are filed with this report on Form 8-K:
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Exhibit No. | | Description |
99.1 | | Earnings Press Release dated November 12, 2014. |
99.2 | | Poster Presented at ASN's Kidney Week Annual Meeting on November 13, 2014. |
99.3 | | ASN Press Release dated November 12, 2014. |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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| | | LA JOLLA PHARMACEUTICAL COMPANY |
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Date: | November 18, 2014 | By: | /s/ George F. Tidmarsh |
| | Name: | George F. Tidmarsh, M.D., Ph.D. |
| | Title: | President and Chief Executive Officer |
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
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• | 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. |
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• | 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). |
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• | 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). |
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• | 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. |
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• | 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). |
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• | 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
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• | In the first quarter of 2015, La Jolla plans to initiate a Phase 3 registration program of LJPC-501 in CRH. |
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• | 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. |
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• | 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. |
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• | 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)
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| | | | | | | | | | | | | | | |
| 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 |
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General and administrative | 2,436 |
| | 3,225 |
| | 8,259 |
| | 9,238 |
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Total expenses | 5,061 |
| | 4,173 |
| | 14,477 |
| | 11,541 |
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Loss from operations | (5,061 | ) | | (4,173 | ) | | (14,477 | ) | | (11,541 | ) |
Other income: | |
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Other income, net | 9 |
| | 1 |
| | 13 |
| | 3 |
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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 |
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LA JOLLA PHARMACEUTICAL COMPANY
Condensed Balance Sheets
(in thousands, except share and per-share amounts)
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| September 30,
2014 | | December 31,
2013 |
| (Unaudited) | |
ASSETS: | | | |
Current assets: | | | |
Cash and cash equivalents | $ | 54,131 |
| | $ | 8,629 |
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Restricted cash | 37 |
| | 37 |
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Prepaid expenses and other current assets | 153 |
| | 43 |
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Total current assets | 54,321 |
| | 8,709 |
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Equipment and furnishings, net | 114 |
| | 38 |
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Total assets | $ | 54,435 |
| | $ | 8,747 |
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LIABILITIES AND SHAREHOLDERS' EQUITY: | | | |
Current liabilities: | | | |
Accounts payable | $ | 683 |
| | $ | 834 |
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Accrued expenses | 900 |
| | 187 |
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Accrued payroll and related expenses | 32 |
| | 73 |
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Total current liabilities | 1,615 |
| | 1,094 |
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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 |
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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 |
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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 |
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Additional paid-in capital | 525,866 |
| | 462,684 |
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Accumulated deficit | (479,765 | ) | | (465,301 | ) |
Total shareholders’ equity | 52,820 |
| | 7,653 |
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Total liabilities and shareholders' equity | $ | 54,435 |
| | $ | 8,747 |
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Contacts
George F. Tidmarsh, M.D., Ph.D.
President & Chief Executive Officer
La Jolla Pharmaceutical Company
Phone: (858) 207-4264
Email: GTidmarsh@ljpc.com
or
Chester S. Zygmont, III
Senior Director of Finance
La Jolla Pharmaceutical Company
Phone: (858) 207-4262
Email: czygmont@ljpc.com
Weekly Doses of GCS-100, a Galectin-3 Antagonist, Resulted in Significant Improvement in eGFR in Patients with CKD in a Randomized, Phase 2 Study Pablo E. Pergola, MD, PhD1, Geoffrey A. Block, MD, FASN2, Bhupinder Singh, MD, FASN3, Robert S. Cohen, DO3, George Z. Fadda, MD4, William T. Durham, MD5, James A. Tumlin, MD, FASN6, George F. Tidmarsh, MD, PhD7, James M. Rolke7 1Renal Associates PA, San Antonio, TX, 2Denver Nephrology, Denver, CO, 3Southwest Kidney Institute PLC, Tempe, AZ, 4Balboa Nephrology Medical Group, La Mesa, CA, 5Mountain Kidney and Hypertension Associates, Asheville, NC, 6Southeast Renal Research Institute, Chattanooga, TN, 7La Jolla Pharmaceutical Company, San Diego, CA Study Design and Demographics Dose, dosing schedule and dose rationale: Blinded subjects were randomized 1:1:1 to receive placebo (saline), 1.5 or 30 mg/m2 GCS-100 IV weekly for 8 weeks, followed by a 5-week observational period. The 1.5 mg/m2 dose was chosen based on activity seen at this and similar dose levels in prior studies. The 30 mg/m2 dose was chosen because it was the MTD observed in a Phase 1 dose-escalation study. Endpoints: Primary endpoint: Change in estimated glomerular filtration rate (eGFR) from baseline to the average of study days 50 and 57 (week 8), comparing placebo to each dose group separately. p-value of <0.1 set a priori. Predefined subset analysis, including disease etiology. Composite analysis based on % change in eGFR and % change in albumin/creatinine ratio (ACR) (points given for increase in eGFR or decrease in urine ACR; points subtracted for decrease in eGFR or increase in urine ACR). Efficacy Analysis Extension Study Results Early terminations: Placebo = 1 (withdrew consent prior to dosing); 1.5 mg/m2 = 0; 30 mg/m2 = 3 (2 withdrew consent prior to dosing; 1 discontinued without reason) Serious adverse events (none considered related to study drug): Placebo = 2 (1 worsening urinary tract infection; 1 cerebrovascular accident); 1.5 mg/m2 = 0; 30 mg/m2 = 2 (1 dyspnea secondary to CHF exacerbation; 1 progressive CKD with volume overload) Other Markers of Kidney Function Were Also Improved in 1.5 mg/m2 Group -2.5 -1.5 -0.5 0.5 1.5 2.5 3.5 M ea n C ha ng e in e GF R (m L/ m in /1 .7 3m 2 ), I nc lu di ng S ta nd ar d Er ro r NS Placebo (n=29) 30 mg/m2 (n=26) 1.5 mg/m2 (n=24) p=0.029 -0.6 -0.4 -0.2 0.0 0.2 0.4 0.6 M ea n C om po si te S co re , In cl ud in g S ta nd ar d Er ro r p=0.009 NS Placebo (n=40) 30 mg/m2 (n=36) 1.5 mg/m2 (n=41) -2.5 -1.5 -0.5 0.5 1.5 2.5 3.5 M ea n C ha ng e in e GF R (m L/ m in /1 .7 3m 2 ), I nc lu di ng S ta nd ar d Er ro r NS Placebo (n=40) 30 mg/m2 (n=36) 1.5 mg/m2 (n=41) Primary Endpoint Met in 1.5 mg/m2 Group (From Baseline to Week 8) p=0.045 Safety Analysis Placebo 1.5 mg/m2 Marker Change P-value Change P-value Comparator Creatinine (mg/dL) +0.07 – -0.13 0.02 placebo Potassium (mg/dL) +0.03 – -0.12 0.07 placebo Uric Acidb (mg/dL) -0.55 0.19 -1.64 0.07 baseline BUNb (mg/dL) -0.74 0.61 -3.93 0.08 baseline ACR (log (µg/mg)) +0.04 – +0.05 0.94 placebo Galectin-3 (ng/mL) +1.03 – -0.88 0.07 placebo Composite Analysis Based on eGFR and ACR Statistically Significant in 1.5 mg/m2 Group (From Baseline to Week 8) Conclusions We conclude: 1. GCS-100, at a dose of 1.5 mg/m2, resulted in a significant improvement in eGFR versus placebo; 2. The results of this study are consistent with GCS-100’s proposed anti- fibrotic mechanism; 3. Other markers of kidney function also improved; 4. GCS-100 had a more pronounced effect in diabetic patients; 5. GCS-100 was well-tolerated; and 6. GCS-100, a novel, potential anti-fibrotic treatment for CKD, warrants further study in a large, randomized trial, particularly in patients with diabetic etiology. Study Rationale Approximately 49 million Americans have chronic kidney disease (CKD), which can lead to cardiovascular morbidity and mortality. Renal fibrosis is the principal process underlying the progression of CKD to end-stage renal disease (ESRD). Galectin-3 is a galactose-binding protein that is upregulated in diseases associated with organ failure; increased levels are correlated with disease onset and reduced survival. Galectin-3 upregulates fibroblast proliferation and collagen synthesis, which lead to increased fibrosis. Galectin-3 knockout mice develop significantly less kidney fibrosis and kidney failure compared to wild-type mice. GCS-100 is a well- characterized, complex sugar derived from pectin that binds to and neutralizes the activity of galectin-3. By blocking galectin-3 activity, GCS-100 may attenuate and/or reverse tissue fibrosis in diseases such as CKD. Secondary endpoint: Safety and tolerability, including physical exam and vitals, adverse events, labs (hematology panel, chemistry panel, quantitative urine analysis and urinalysis). Demographics: Subjects: 121 enrolled; 117 completed the study Age: Mean: 64; median: 67; range: 29-85 Gender: 78 males, 43 females Disease etiology: Diabetes (n=79); hypertension (n=29); other (n=9) Baseline eGFR (mL/min/1.73m2): Mean: 29; median: 28; range: 13-51 GCS-100’s effect on eGFR was more pronounced (p=0.029) in the prospectively defined subset of patients with diabetic etiology. Analysis of the diabetic subset was predefined and based on the observation that galectin-3 is elevated in this population and correlates with proteinuria.d 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, vs. 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 was safe and well-tolerated. There were no SAEs and no early terminations in the 1.5 mg/m2 group. GCS-100’s efficacy at 1.5 mg/m2 was reproduced in a randomized extension study in patients who had originally received placebo. Effect on eGFR More Prominent in Diabetic Patients in 1.5 mg/m2 Group (From Baseline to Week 8) An extension study is being conducted in which patients from the Phase 2 study were re-randomized 1:1 to receive either 1.5 or 30 mg/m2 of GCS- 100 weekly for 8 weeks and then dosing was restarted at week 12 (complete data available through week 16). bAnalysis on subset of patients with elevated values at baseline GCS-100, at a dose of 1.5 mg/m2, resulted in a statistically significant (p=0.045) improvement in eGFR versus placebo after 8 weeks of dosing. As such, the primary endpoint in this randomized, Phase 2 study was met. Several aspects of the results of this study support GCS-100’s proposed anti-fibrotic mechanism: 1. The improvement in eGFR was steady over time; 2. This improvement, on a placebo-corrected basis, was maintained at 5 weeks following the completion of dosing (p=0.07); and 3. There were no hemodynamic changes that could account for a change in eGFR. Other markers of kidney function (including creatinine, potassium, uric acid and BUN) also improved in the 1.5 mg/m2 group. eGFR Improves Steadily Over Time in 1.5 mg/m2 Group Adverse events possibly related to study drug (number of patients): Placebo: itching of scalp (1), hypernatremia (1); 1.5 mg/m2: lower extremity muscle cramp (1), foul urine smell (1), dysgeusia (1), lower back pain (1), pruritus (1), facial flushing (1), hives (1); 30 mg/m2: nausea (1), fatigue (1), gastroenteritis (1), hypoalbuminemia (1), hyperkalemia (1), hypermagnesemia (1), weight gain (1), pruritus (1), hyponatremia (1), hot flashes (1), flu-like symptoms (1), worsening eGFR (1) Measurement Placebo 1.5 mg/m2 30 mg/m2 Starting Average BP 132/72 135/72 140/74 Ending Average BP 135/70 135/72 138/72 Physical exam and vitals: No clinically significant negative changes, including no significant weight changes and no effect on blood pressure Labs: No clinically significant negative changes Blood Pressure Discussion -2.0 -1.5 -1.0 -0.5 0.0 0.5 1.0 1.5 0 2 4 6 8 10 12 M ea n C ha ng e in e GF R (m L/ m in /1 .7 3m 2 ) Week p=0.07 1.5 mg/m2 (n=41) 30 mg/m2 (n=36) Placebo (n=40) End of dosing Primary endpoint p=0.045 -2.5 -2.0 -1.5 -1.0 -0.5 0.0 0.5 0 2 4 6 8 10 12 M ea n C ha ng e in e GF R (m L/ m in /1 .7 3m 2 ) Week GCS-100 Phase 2 Placebo Group (n=40) IDNT Placebo Groupa (n=569) Placebo Group Progression Consistent with Natural History of Disease: Both Subject to Hawthorne Effect aEvans M et al. Irbesartan delays progression of nephropathy as measured by estimated glomerular filtration rate: post hoc analysis of the Irbesartan Diabetic Nephropathy Trial. Nephrol Dial Transplant 2012 27: 2255–2263 Statistically Significant Change in eGFR at 16 Weeks vs. 30 mg/m2 Group and Placeboc -3.0 -2.5 -2.0 -1.5 -1.0 -0.5 0.0 0.5 1.0 1.5 2.0 2.5 3.0 M ea n C ha ng e in e GF R (m L/ m in /1 .7 3m 2 ), I nc lu di ng S ta nd ar d Er ro r p=0.04 p=0.02 1.5 mg/m2 (n=20) 30 mg/m2 (n=13) Placebo (n=33) cPrevious response on placebo in Phase 2 study for treatment–naïve patients in extension study (baseline to week 12) dKikuchi Y et al. Galectin-3-positive cell infiltration in human diabetic nephropathy. Nephrol Dial Transplant 2004 19: 602-607 -2.0 -1.5 -1.0 -0.5 0.0 0.5 1.0 1.5 0 4 8 12 16 M ea n C ha ng e in e GF R (m L/ m in /1 .7 3m 2 ) Week Extension Results for Placebo to 1.5 mg/m2 Group End of Initial Dosing Dosing Restarted 93 patients were enrolled in total. Of these, 33 patients had previously received placebo in the Phase 2 study. These previously treatment- naïve patients were analyzed for efficacy. Consistent with the 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 parallel randomized group receiving 30 mg/m2 (p=0.04); and 2. The previous response to placebo in the Phase 2 study for all treatment- naïve patients in the extension study (p=0.02).
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, 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 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.
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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 |
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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.
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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.
Contacts
George F. Tidmarsh, M.D., Ph.D.
President & Chief Executive Officer
La Jolla Pharmaceutical Company
Phone: (858) 207-4264
Email: GTidmarsh@ljpc.com
or
Chester S. Zygmont, III
Senior Director of Finance
La Jolla Pharmaceutical Company
Phone: (858) 207-4262
Email: czygmont@ljpc.com
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