La Jolla Pharmaceutical Company Announces Financial Results for the Three Months Ended March 31, 2018 and Recent Corporate Progress

La Jolla Pharmaceutical Company Announces Financial Results for the Three Months Ended March 31, 2018 and Recent Corporate Progress
SAN DIEGO, May 10, 2018 (GLOBE NEWSWIRE) — La Jolla Pharmaceutical Company (Nasdaq:LJPC), a leader in the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced financial results for the three months ended March 31, 2018 and highlighted recent corporate progress.

Recent Corporate Progress

  • In May 2018, La Jolla closed a $125 million royalty financing agreement with HealthCare Royalty Partners (HCR). Under the terms of the agreement, La Jolla will receive $125 million in exchange for tiered royalty payments on worldwide net sales of GIAPREZA. Payments under the agreement start annually at a maximum royalty rate, with step-downs based on the achievement of annual net sales thresholds. Through December 31, 2021, the royalty rate will be a maximum of 10%. Starting January 1, 2022, the maximum royalty rate may increase by 4% if an agreed-upon, cumulative sales threshold has not been met, and, starting January 1, 2024, the maximum royalty rate may increase by an additional 4% if a different agreed-upon, cumulative sales threshold has not been met. The agreement is subject to maximum aggregate royalty payments to HCR of 180% of the $125 million to be received by La Jolla, at which time the payment obligations under the agreement would expire. The agreement was entered into by La Jolla’s wholly owned subsidiary, La Jolla Pharma, LLC, and HCR has no recourse under the agreement against La Jolla or any assets other than GIAPREZA.
  • In March 2018, La Jolla announced the commercial availability of GIAPREZA™ (angiotensin II) injection for intravenous infusion (formerly known as LJPC-501). In December 2017, GIAPREZA was approved by the U.S. Food and Drug Administration (FDA) to increase blood pressure in adults with septic or other distributive shock. GIAPREZA mimics the body’s endogenous regulatory peptide that is central to the renin-angiotensin-aldosterone system to increase blood pressure. Prescribing information for GIAPREZA is available at www.giapreza.com.
  • In March 2018, an analysis, entitled “Outcomes in Patients with Acute Kidney Injury Receiving Angiotensin II for Vasodilatory Shock,” was presented at the 23rd International Conference on Advances in Critical Care Nephrology AKI & CRRT 2018. The manuscript of this analysis, entitled “Outcomes in patients with vasodilatory shock and renal replacement therapy treated with intravenous angiotensin II,” was published online in Critical Care Medicine. The presentation and manuscript detail the outcomes of patients with acute kidney injury (AKI) and vasodilatory shock enrolled in the ATHOS-3 (Angiotensin II for the Treatment of High-Output Shock) Phase 3 study of GIAPREZA. In this post-hoc analysis, the data from 105 AKI patients (GIAPREZA n=45; placebo n=60) requiring renal replacement therapy (RRT) at study drug initiation were analyzed. Survival through day 28 was 53% (95% CI: 38%-67%) for the GIAPREZA group compared to 30% (95% CI: 19%-41%) for the placebo group (p = 0.012). By day 7, 38% (95% CI: 25%-54%) of patients treated with GIAPREZA discontinued RRT compared to 15% (95% CI: 8%-27%) of patients treated with placebo (p = 0.007). Mean arterial pressure (MAP) response at hour 3 was achieved in 53% (95% CI: 38%-68%) of patients treated with GIAPREZA compared to 22% (95% CI: 12%-34%) of patients treated with placebo (p = 0.001).
  • In February 2018, an abstract, entitled “Effect of Disease Severity on Survival in Patients Receiving Angiotensin II for Vasodilatory Shock,” was presented at the Society of Critical Care Medicine’s (SCCM) 47th Critical Care Congress. The abstract, which was published in the January Supplement of Critical Care Medicine, includes results from a pre-specified analysis from the ATHOS-3 Phase 3 study of GIAPREZA in patients with high severity of illness, defined as an APACHE II (Acute Physiology and Chronic Health Evaluation II) score > 30 or baseline MAP < 65 mmHg, despite treatment with high-dose vasopressors. The authors presented data showing a lower 28-day mortality rate in patients with baseline APACHE II scores > 30 in the GIAPREZA group versus the placebo group: 28-day mortality was 51.8% (n = 58) for the GIAPREZA group compared to 70.8% (n = 65) for the placebo group (hazard ratio=0.62 [95% CI: 0.39, 0.98; p=0.037]). In patients with a baseline MAP < 65 mmHg, a trend towards improved 28-day mortality was seen in the GIAPREZA group compared to the placebo group: 28-day mortality was 54.2% (n = 52) for the GIAPREZA group compared to 70.4% (n = 50) for the placebo group (hazard ratio=0.66 [95% CI: 0.40, 1.09; p=0.10]).

“This first quarter was the start of an exciting and transformational year for La Jolla, highlighted by the commercial launch of GIAPREZA,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “We are excited to bring this new treatment option to the many critically ill patients suffering from septic or other distributive shock.”

Results of Operations

As of March 31, 2018, La Jolla had $154.4 million in cash and cash equivalents, compared to $90.9 million as of December 31, 2017. On a pro-forma basis, adjusting for the net proceeds from the May 2018 royalty financing, La Jolla’s cash and cash equivalents as of March 31, 2018 were $279 million. Cash used for operating activities for the three months ended March 31, 2018 was $45.9 million, compared to $22.0 million for the same period in 2017. La Jolla recognized GIAPREZA net product sales of $0.8 million for the three months ended March 31, 2018. La Jolla launched GIAPREZA in March 2018. La Jolla’s net loss for the three months ended March 31, 2018 was $50.5 million, or $2.22 per share, compared to $23.2 million, or $1.26 per share, for the same period in 2017.

About Septic or Other Distributive Shock

Over one million Americans are affected by shock on an annual basis, with one in three patients being treated for shock in the intensive care unit. Distributive shock is the most common type of shock in the inpatient setting with approximately 800,000 distributive shock cases in the U.S. per year. Of these cases, an estimated 90% are septic shock patients. Approximately 300,000 do not achieve adequate blood pressure response with standard of care vasopressor therapy (catecholamines and vasopressin). The inability to achieve or maintain adequate blood pressure results in inadequate blood flow to the body’s organs and tissue and is associated with a mortality rate exceeding most acute conditions requiring hospitalization.

About GIAPREZA

In December 2017, GIAPREZA™ (angiotensin II) was approved by the U.S. Food and Drug Administration (FDA) to increase blood pressure in adults with septic or other distributive shock. GIAPREZA mimics the body’s endogenous regulatory peptide that is central to the renin-angiotensin-aldosterone system to increase blood pressure. Prescribing information for GIAPREZA is available at www.giapreza.com.

IMPORTANT SAFETY INFORMATION

Contraindications

None

Warnings and Precautions

There is a potential for venous and arterial thrombotic and thromboembolic events in patients who receive GIAPREZA. Use concurrent venous thromboembolism (VTE) prophylaxis.

Adverse Reactions

The most common adverse reactions that were reported in greater than 10% of GIAPREZA-treated patients were thromboembolic events.

Drug Interactions

Angiotensin converting enzyme (ACE) inhibitors may increase response to GIAPREZA. Angiotensin II receptor blockers (ARB) may reduce response to GIAPREZA.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

For additional information, please see Full Prescribing Information.

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. GIAPREZA™ (angiotensin II), formerly known as LJPC-501, was approved by the U.S. Food and Drug Administration (FDA) on December 21, 2017 as a vasoconstrictor to increase blood pressure in adults with septic or other distributive shock. LJPC‑401 (synthetic human hepcidin), a clinical-stage investigational product, is being developed for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. For more information, please visit www.ljpc.com.

Forward-looking Statements

This press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to expectations regarding future events or La Jolla’s future results of operations. These statements are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from those anticipated by the forward-looking statements. La Jolla 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 La Jolla’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s website www.sec.gov. These risks include, but are not limited to, risks relating to: our ability to successfully commercialize, market and achieve market acceptance of GIAPREZA and other product candidates; potential market sizes, including for septic or other distributive shock; the success of development activities for LJPC-401 and other product candidates, including post-approval studies for GIAPREZA; the successful and timely completion of clinical trials; unforeseen safety issues from the administration of product and product candidates in patients; and other risks and uncertainties identified in our filings with the SEC. Forward-looking statements are presented as of the date of this press release, and La Jolla expressly disclaims any intent to update any forward‑looking statements to reflect the outcome of subsequent events.

LA JOLLA PHARMACEUTICAL COMPANY

Unaudited Condensed Consolidated Statements of Operations
(in thousands, except per share amounts)

Three Months Ended
March 31,
2018 2017
Revenue
Net product sales $ 809 $
Total revenue 809
Operating expenses
Cost of product sales 58
Research and development 28,429 17,765
Selling, general and administrative 23,016 5,503
Total operating expenses 51,503 23,268
Loss from operations (50,694 ) (23,268 )
Other income, net 166 28
Net loss $ (50,528 ) $ (23,240 )
Net loss per share, basic and diluted $ (2.22 ) $ (1.26 )
Weighted-average common shares outstanding, basic and diluted 22,742 18,410

LA JOLLA PHARMACEUTICAL COMPANY

Condensed Consolidated Balance Sheets
(in thousands, except share and par value amounts)

March 31,
2018
December 31,
2017
(Unaudited)
ASSETS
Current assets:
Cash and cash equivalents $ 154,408 $ 90,915
Inventory 820
Prepaid expenses and other current assets 6,326 3,147
Total current assets 161,554 94,062
Property and equipment, net 24,438 24,568
Restricted cash 909 909
Total assets $ 186,901 $ 119,539
LIABILITIES AND SHAREHOLDERS’ EQUITY
Current liabilities:
Accounts payable $ 7,321 $ 11,484
Accrued clinical and other expenses 4,280 703
Accrued payroll and related expenses 3,044 4,995
Deferred rent, current portion 1,370 1,370
Total current liabilities 16,015 18,552
Deferred rent, less current portion 13,473 12,785
Total liabilities 29,488     31,337
Shareholders’ equity:
Common Stock, $0.0001 par value; 100,000,000 shares authorized,
26,154,439 and 22,167,529 shares issued and outstanding at March 31, 2018 and December 31, 2017, respectively
3 2
Series C-12 Convertible Preferred Stock, $0.0001 par value; 11,000 shares authorized, 3,906 shares issued and outstanding at March 31, 2018 and December 31, 2017, and liquidation preference of $3,906 at March 31, 2018 and December 31, 2017 3,906 3,906
Series F Convertible Preferred Stock, $0.0001 par value; 10,000 shares authorized,
2,737 shares issued and outstanding at March 31, 2018 and December 31, 2017, and liquidation preference of $2,737 at March 31, 2018 and December 31, 2017
2,737 2,737
Additional paid-in capital 922,809 803,071
Accumulated deficit (772,042 ) (721,514 )
Total shareholders’ equity 157,413 88,202
Total liabilities and shareholders’ equity $ 186,901 $ 119,539

Company Contacts

Sandra Vedrick
Director, Investor Relations & Human Resources
La Jolla Pharmaceutical Company
Phone: (858) 207-4264 Ext: 1135
Email: svedrick@ljpc.com

and

Dennis M. Mulroy
Chief Financial Officer
La Jolla Pharmaceutical Company
Phone: (858) 207-4264 Ext: 1040
Email: dmulroy@ljpc.com

GIAPREZA™ (angiotensin II) Significantly Improved Survival and Reduced Time on Renal Replacement Therapy in Patients with Acute Kidney Injury

Analyses to Be Presented in Oral and Poster Sessions at The 23rd International Conference on Advances in Critical Care Nephrology AKI & CRRT 2018

Manuscript Published Online in Critical Care Medicine

SAN DIEGO, March 06, 2018 (GLOBE NEWSWIRE) — La Jolla Pharmaceutical Company (Nasdaq:LJPC) today announced the release of data from analyses of the impact of GIAPREZA™ (angiotensin II) on outcomes of a subset of patients with acute kidney injury requiring renal replacement therapy (AKI-RRT) enrolled in the ATHOS-3 (Angiotensin II for the Treatment of High Output Shock) study.

The presentation, entitled “Outcomes in Patients with Acute Kidney Injury Receiving Angiotensin II for Vasodilatory Shock,” will take place during The 23rd International Conference on Advances in Critical Care Nephrology – AKI & CRRT 2018, being held March 6-9, 2018 in San Diego, California. The manuscript, entitled “Outcomes in patients with vasodilatory shock and renal replacement therapy treated with intravenous angiotensin II” was published online in Critical Care Medicine.

“Acute kidney injury requiring dialysis associated with distributive shock, also referred to as vasodilatory shock, represents a significant medical risk for patients and a significant financial burden to the health care system,” said James Tumlin M.D., Professor of Medicine and Director of NephroNet Clinical Trials Consortium. “These analyses of the effect of angiotensin II on AKI patients requiring dialysis in the ATHOS-3 study demonstrated angiotensin II is a promising therapy to address this unmet need.”

The presentations and manuscript detail the outcomes of patients with acute kidney injury (AKI) and vasodilatory shock enrolled in the ATHOS-3 study of GIAPREZA. In this post-hoc analysis, the data from 105 AKI patients (GIAPREZA n=45; placebo n=60) requiring renal replacement therapy (RRT) at study drug initiation were analyzed. Survival through day 28 was 53% (95% CI: 38%-67%) for the GIAPREZA group compared to 30% (95% CI: 19%-41%) for the placebo group (p = 0.012). By day 7, 38% (95% CI: 25%-54%) of patients treated with GIAPREZA discontinued RRT compared to 15% (95% CI: 8%-27%) of patients treated with placebo (p = 0.007). Mean arterial pressure (MAP) response at hour 3 was achieved in 53% (95% CI: 38%-68%) of patients treated with GIAPREZA compared to 22% (95% CI: 12%-34%) of patients treated with placebo (p = 0.001).

23rd International Conference on Advances in Critical Care Nephrology – AKI & CRRT 2018 Presentation Details
Presentation Title: Outcomes in Patients with Acute Kidney Injury Receiving Angiotensin II for Vasodilatory Shock
Presenter: James Tumlin, M.D., Professor of Medicine and Director of NephroNet Clinical Trials Consortium
Poster Presentation Date: Tuesday, March 6, 2018 5:30 pm – 7:00 pm and Wednesday, March 7, 2018 6:00 pm – 8:00 pm Pacific Time
Oral Presentation Date: Friday, March 9, 2018 11:00 am – 12:25 pm Pacific Time
Location: Manchester Grand Hyatt, San Diego, CA

About GIAPREZA

In December 2017, GIAPREZA™ (angiotensin II) was approved by the U.S. Food and Drug Administration (FDA) to increase blood pressure in adults with septic or other distributive shock. GIAPREZA mimics the body’s endogenous regulatory peptide that is central to the renin-angiotensin-aldosterone system to increase blood pressure. Prescribing information for GIAPREZA is available at www.giapreza.com.

IMPORTANT SAFETY INFORMATION

Contraindications

None

Warnings and Precautions

There is a potential for venous and arterial thrombotic and thromboembolic events in patients who receive GIAPREZA. Use concurrent venous thromboembolism (VTE) prophylaxis.

Adverse Reactions

The most common adverse reactions reported in greater than 10% in GIAPREZA treated patients were thromboembolic events.

Drug Interactions

Angiotensin converting enzyme (ACE) inhibitors may increase response to GIAPREZA.
Angiotensin II receptor blockers (ARB) may reduce response to GIAPREZA.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

For additional information, please see Full Prescribing Information.

About Shock and Septic or Other Distributive Shock

Over 1 million Americans are affected by shock on an annual basis, with 1 in 3 patients being treated for shock in the intensive care unit. Distributive shock is the most common type of shock in the inpatient setting with approximately 800,000 distributive shock cases in the United States per year. Of these cases, an estimated 90% are septic shock patients. Approximately 300,000 do not achieve adequate blood pressure response with standard of care vasopressor therapy (catecholamines and vasopressin). The inability to achieve or maintain adequate blood pressure results in inadequate blood flow to the body’s organs and tissue and is associated with a mortality rate exceeding most acute conditions requiring hospitalization.

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. GIAPREZA™ (angiotensin II), formerly known as LJPC-501, was approved by the U.S. Food and Drug Administration (FDA) on December 21, 2017 as a vasoconstrictor to increase blood pressure in adults with septic or other distributive shock. LJPC‑401 (synthetic human hepcidin), a clinical-stage investigational product, is being developed for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. For more information, please visit www.ljpc.com.

Forward-looking Statements

This press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to expectations regarding future events or La Jolla’s future results of operations. These statements are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from those anticipated by the forward-looking statements. La Jolla 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 La Jolla’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s website www.sec.gov. These risks include, but are not limited to, risks relating to: unforeseen safety issues from the administration of GIAPREZA in patients; the anticipated treatment of future clinical data by the FDA, the EMA or other regulatory authorities; potential market sizes, including for septic or other distributive shock; our ability to successfully commercialize, market and achieve market acceptance of GIAPREZA; and other risks and uncertainties identified in our filings with the SEC. La Jolla expressly disclaims any intent to update any forward‑looking statements to reflect the outcome of subsequent events.

Company Contacts

Sandra Vedrick
Director, Investor Relations & Human Resources
La Jolla Pharmaceutical Company
Phone: (858) 207-4264 Ext: 1135
Email: svedrick@ljpc.com

and

Dennis M. Mulroy
Chief Financial Officer
La Jolla Pharmaceutical Company
Phone: (858) 207-4264 Ext: 1040
Email: dmulroy@ljpc.com

La Jolla Pharmaceutical Company Announces Financial Results for the Three and Twelve Months Ended December 31, 2017 and Recent Corporate Progress

SAN DIEGO, Calif., Feb. 22, 2018 (GLOBE NEWSWIRE) — 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 financial results for the three and twelve months ended December 31, 2017 and highlighted recent corporate progress.

Recent Corporate Progress

  • In December 2017, GIAPREZA™ (angiotensin II), injection for intravenous infusion, formerly known as LJPC-501, was approved by the U.S. Food and Drug Administration (FDA) as a vasoconstrictor indicated to increase blood pressure in adults with septic or other distributive shock.
  • In December 2017, La Jolla announced the initiation of LJ401-HH01, a multinational, multicenter, randomized, placebo-controlled, double-blind, Phase 2 study that is designed to evaluate the safety and efficacy of LJPC-401 (synthetic human hepcidin) as a treatment for hereditary hemochromatosis (HH). Approximately 60 patients in 5 countries will be randomized to receive weekly subcutaneous injections of either LJPC-401 or placebo for 12 weeks. The primary efficacy endpoint of the study is the change in transferrin saturation, a standard measurement of iron levels in the body and one of the two key measurements used to detect iron overload, from baseline to end of treatment. Secondary efficacy endpoints include: (i) the change in serum ferritin, the other key measurement used to detect iron overload, from baseline to end of treatment; and (ii) the requirement for and frequency of phlebotomy procedures used during the study.
  • In December 2017, La Jolla announced the initiation of LJ401-BT01, a pivotal, multinational, multicenter, randomized, controlled study of LJPC-401 in patients with transfusion-dependent beta thalassemia who, despite chelation therapy, have cardiac iron levels above normal. Approximately 100 patients in 9 countries, including the U.S., will be randomized 1:1 to receive either: (i) weekly subcutaneous injections of LJPC-401, while continuing standard-of-care chelation therapy (LJPC-401 treatment arm); or (ii) a continuation of standard-of-care chelation therapy only (observation arm). After 6 months of treatment, patients randomized to the observation arm will cross over to receive LJPC-401 (plus standard-of-care chelation therapy) for 6 months, while patients randomized to the LJPC-401 treatment arm will continue with LJPC-401 (plus standard-of-care chelation therapy) for an additional 6 months (for a total of one year). The primary efficacy endpoint of this study is the change in iron content in the heart after 6 months, as measured by cardiac magnetic resonance imaging (MRI). If this study is successful, we would anticipate filing a market authorization application (MAA) for LJPC-401 in Europe.

“2017 was an exciting year for La Jolla, highlighted by the FDA’s approval of GIAPREZA,” said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. “We are excited to bring this new treatment option to the many critically ill patients suffering from septic or other distributive shock.”

Results of Operations

As of December 31, 2017, La Jolla had $90.9 million in cash and cash equivalents, compared to $65.7 as of December 31, 2016. La Jolla’s net cash used for operating activities for the twelve months ended December 31, 2017 was $84.9 million compared to net cash used for operating activities of $58.7 million for the same period in 2016. La Jolla’s net loss for the three and twelve months ended December 31, 2017 was $38.5 million and $114.8 million, or $1.74 per share and $5.41 per share, respectively, compared to a net loss of $24.9 million and $78.2 million, or $1.44 per share and $4.54 per share, respectively, for the same periods in 2016.

About GIAPREZA

In December 2017, GIAPREZA™ (angiotensin II) was approved by the U.S. Food and Drug Administration (FDA) to increase blood pressure in adults with septic or other distributive shock. GIAPREZA mimics the body’s endogenous regulatory peptide that is central to the renin-angiotensin-aldosterone system to increase blood pressure. In the ATHOS-3 Phase 3 study, the primary endpoint of: (i) a mean arterial pressure (MAP) increase of ≥ 10 mmHg; or (ii) a MAP of ≥ 75 mmHg, was achieved by 70% of patients randomized to GIAPREZA, compared to 23% of patients randomized to placebo (p < 0.0001); both arms were treated with standard-of-care vasopressors. The recommended starting dosage of GIAPREZA is 20 nanograms (ng)/kg/min via continuous intravenous infusion. Close monitoring during the first 5 minutes of GIAPREZA initiation is recommended. GIAPREZA is available in 1 mL single dose vials, each containing 2.5 mg of angiotensin II (as a sterile liquid). Prescribing information for GIAPREZA is available at www.giapreza.com.

IMPORTANT SAFETY INFORMATION

Warnings and Precautions

The safety of GIAPREZA was evaluated in 321 adults with septic or other distributive shock in the randomized, double-blind, placebo-controlled Phase 3 study, ATHOS-3. There was a higher incidence of venous and arterial thrombotic and thromboembolic events in patients who received GIAPREZA compared to placebo-treated patients in the ATHOS-3 study [13% (21/163 patients) vs. 5% (8/158 patients)]. The major imbalance was in deep venous thromboses. Use concurrent venous thromboembolism prophylaxis.

Adverse Reactions

Adverse reactions that occurred in ≥4% of patients treated with GIAPREZA and ≥1.5% more often than placebo-treated patients in the ATHOS-3 study were thromboembolic events (including deep vein thrombosis), thrombocytopenia, tachycardia, fungal infection, delirium, acidosis, hyperglycemia and peripheral ischemia.

Drug Interactions

Angiotensin converting enzyme (ACE) inhibitors may increase response to GIAPREZA. Angiotensin II receptor blockers (ARB) may reduce response to GIAPREZA.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

For additional information, please see Full Prescribing Information.

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. GIAPREZA™ (angiotensin II), formerly known as LJPC-501, was approved by the U.S. Food and Drug Administration (FDA) on December 21, 2017 as a vasoconstrictor to increase blood pressure in adults with septic or other distributive shock. LJPC‑401 (synthetic human hepcidin), a clinical-stage investigational product, is being developed for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. For more information, please visit www.ljpc.com.

Forward-looking Statements

This press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to expectations regarding future events or La Jolla’s future results of operations. These statements are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from those anticipated by the forward-looking statements. La Jolla 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 La Jolla’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s website www.sec.gov. These risks include, but are not limited to, risks relating to: our ability to successfully commercialize, market and achieve market acceptance of GIAPREZA and other product candidates; potential market sizes, including for septic or other distributive shock; the success of development activities for LJPC-401 and other product candidates; and other risks and uncertainties identified in our filings with the SEC. Forward-looking statements are presented as of the date of this press release, and La Jolla expressly disclaims any intent to update any forward‑looking statements to reflect the outcome of subsequent events.

Company Contacts

Sandra Vedrick

Director, Investor Relations & Human Resources

La Jolla Pharmaceutical Company

Phone: 858 207 4264 Ext: 1135

Email: svedrick@ljpc.com

and

Dennis M. Mulroy

Chief Financial Officer

La Jolla Pharmaceutical Company

Phone: 858 207 4264 Ext: 1040

Email: dmulroy@ljpc.com

La Jolla Pharmaceutical Company Announces FDA Approval of Giapreza™ (angiotensin II)

Giapreza increases blood pressure in adults with septic or other distributive shock

La Jolla to host conference call and webcast at 8:30 a.m. EST on December 22, 2017

SAN DIEGO, Dec. 21, 2017 (GLOBE NEWSWIRE) — La Jolla Pharmaceutical Company (Nasdaq:LJPC) (the Company or La Jolla) today announced that the U.S. Food and Drug Administration (FDA) has approved GiaprezaTM (angiotensin II) to increase blood pressure in adults with septic or other distributive shock.

“We appreciate FDA’s rapid review and approval of Giapreza and are especially grateful to the patients, families and dedicated critical care teams who made the development of Giapreza possible,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We look forward to bringing this new treatment option to the many critically ill patients suffering from septic or other distributive shock.”

“Vasopressors are critical to treat patients with shock. The critical care community now has another tool to use,” said John A. Kellum, M.D., Director of Center for Critical Care Nephrology, Vice Chair for Research, and Professor of Critical Care Medicine, University of Pittsburgh. “The approval of angiotensin II represents a major advance in the treatment of patients with septic or distributive shock.”

La Jolla plans to make Giapreza available for patients in the U.S. in March 2018. Prescribing information for Giapreza is available at www.giapreza.com.

About Septic or Other Distributive Shock

Distributive shock is the most common type of shock in the inpatient setting, affecting approximately one-third of intensive care unit patients. There are approximately 800,000 distributive shock cases in the United States per year. Of these cases, an estimated 90% are septic shock patients. Approximately 300,000 do not achieve adequate blood pressure response with current standard therapy. The inability to achieve or maintain adequate blood pressure results in inadequate blood flow to the body’s organs and tissue and is associated with a mortality rate exceeding most acute conditions requiring hospitalization.

Conference Call at 8:30 a.m. Eastern Time on December 22, 2017

The Company will host a conference call and webcast at 8:30 a.m. Eastern Time (5:30 a.m. Pacific Time) on December 22, 2017. The conference call can be accessed by dialing 877‑359‑9508 for domestic callers and 224‑357‑2393 for international callers. Please provide the operator with the passcode 7979625 to join the conference call or click here for the webcast. A slide presentation accompanying today’s press release and the conference call may also be found on La Jolla’s website at www.ljpc.com thirty minutes prior to the call under the investor relations section. An archive of the conference call and webcast will be available on La Jolla’s website for 30 days following the call.

About 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. Giapreza (angiotensin II), formerly known as LJPC-501, was approved by the U.S. Food and Drug Administration in December 2017 as a vasoconstrictor to increase blood pressure in adults with septic or other distributive shock. La Jolla plans to make Giapreza available for patients in the U.S. starting in March 2018. LJPC‑401 (synthetic human hepcidin) is being developed for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. For more information on La Jolla, please visit www.ljpc.com.

Forward Looking Statement Safe Harbor

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to expectations regarding future events or La Jolla’s future results of operations. These statements are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from those anticipated by the forward-looking statements. La Jolla 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 La Jolla’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s website www.sec.gov. These risks include, but are not limited to, risks relating to: the timing for commercial launch of Giapreza (angiotensin II); the degree of physician or pharmacy and therapeutics committee adoption of Giapreza and La Jolla’s success in commercializing Giapreza; the timing and availability of Giapreza in the market; the anticipated treatment of future clinical data by the FDA, the EMA or other regulatory authorities, including whether such data will be sufficient for approval of Giapreza in the EMA and for approval of other product candidates by either the FDA or EMA; risks relating to the scope of the Giapreza product label; potential market sizes, including for septic or other distributive shock; potential indications for which La Jolla’s products and product candidates may be developed; the anticipated timing for regulatory actions; the timing, costs, conduct and outcome of clinical studies; the impact of pharmaceutical industry regulation and healthcare legislation in the United States; and the success of future development activities. La Jolla expressly disclaims any intent to update any forward‑looking statements to reflect the outcome of subsequent events.

Company Contacts

Sandra Vedrick

Director, Investor Relations & Human Resources

La Jolla Pharmaceutical Company

Phone: 858 207 4264 Ext: 1135

Email: svedrick@ljpc.com

and

Dennis M. Mulroy

Chief Financial Officer

La Jolla Pharmaceutical Company

Phone: 858 207 4264 Ext: 1040

Email: dmulroy@ljpc.com

La Jolla Pharmaceutical Company Announces Initiation of Multicenter, Randomized, Phase 2 Clinical Study of LJPC-401 in Patients with Hereditary Hemochromatosis

SAN DIEGO, CA – December 18, 2017 – La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla) today announced the initiation of LJ401-HH01, a Phase 2 clinical study of LJPC-401 (synthetic human hepcidin) in patients with hereditary hemochromatosis (HH). The most common genetic disease in Caucasians, HH is a disease characterized by a genetic mutation that results in a deficiency in the production of hepcidin, which is the body’s naturally occurring regulator of iron absorption and distribution. Without normal levels of hepcidin, excessive amounts of iron accumulate in the body. Symptoms of the disease include joint pain, abdominal pain, fatigue and weakness. If left untreated, HH can lead to liver cirrhosis, liver cancer, heart disease and/or failure and diabetes.

LJ401-HH01 is a multinational, multicenter, randomized, placebo-controlled, double-blind, Phase 2 study that is designed to evaluate the safety and efficacy of LJPC-401 as a treatment for HH. Approximately 60 patients will be randomized to receive weekly subcutaneous injections of either LJPC-401 or placebo for 12 weeks. The primary efficacy endpoint of the study is the change in transferrin saturation, a standard measurement of iron levels in the body and one of the two key measurements used to detect iron overload, from baseline to end of treatment. Secondary efficacy endpoints include: (i) the change in serum ferritin, the other key measurement used to detect iron overload, from baseline to end of treatment; and (ii) the requirement for and frequency of phlebotomy procedures during the study.

“There is a major need for new treatment modalities that improve the quality of life of the many patients suffering from hereditary hemochromatosis,” stated Jeff Vacirca, M.D., Chief of Clinical Research at New York Cancer & Blood Specialists and investigator in the study. “I am excited to be included in the clinical evaluation of a treatment that harnesses the body’s natural mechanism for iron regulation in a patient-friendly treatment regimen that could potentially reduce or eliminate the need for phlebotomy procedures in these patients.”

“Following the results of Phase 1 clinical studies that demonstrated that the administration of LJPC-401 resulted in dose-dependent reductions in iron levels, we are pleased to initiate LJ401- HH01 to evaluate the therapeutic potential of LJPC-401 in the important patient population of hereditary hemochromatosis,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “With the recent initiation of LJ401-BT01, our pivotal study of LJPC-401 in beta thalassemia patients, LJPC-401 now is being evaluated in two, multicenter, randomized, controlled clinical studies. These studies are important components of our overall development program for LJPC-401, which is aimed at helping the many patients suffering from the effects of iron overload due to a variety of underlying causes.”

The current standard treatment for HH is a blood removal procedure known as phlebotomy. Each phlebotomy procedure, which is usually conducted at a hospital, medical office or blood center, typically involves the removal of approximately a pint of blood. The required frequency of procedures varies by patient, but often ranges from one to two times per week for an initial period after diagnosis and once every one to three months chronically. Since most of the body’s iron is stored in red blood cells, chronic removal of blood can effectively lower iron levels if a phlebotomy regimen is adhered to. However, phlebotomy procedures may cause and may be associated with pain, bruising and scarring at the venous puncture site, fatigue and dizziness during and following the procedure and disruption of daily activities. Furthermore, phlebotomy is not appropriate in patients with poor venous access, anemia or heart disease.

About LJPC-401

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

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

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

About Hereditary Hemochromatosis

Hereditary hemochromatosis (HH) is the most common genetic disease in Caucasians. HH is a disease characterized by a genetic mutation that results in a deficiency in the production of hepcidin, which is the body’s naturally occurring regulator of iron absorption and distribution. Without normal levels of hepcidin, excessive amounts of iron accumulate in the body. Symptoms of the disease include joint pain, abdominal pain, fatigue and weakness. If left untreated, HH can lead to liver cirrhosis, liver cancer, heart disease and/or failure and diabetes.

The current standard treatment for HH is a blood removal procedure known as phlebotomy. Each phlebotomy procedure, which is usually conducted at a hospital, medical office or blood center, typically involves the removal of approximately a pint of blood. The required frequency of procedures varies by patient, but often ranges from one to two times per week for an initial period after diagnosis and once every one to three months chronically. Since most of the body’s iron is stored in red blood cells, chronic removal of blood can effectively lower iron levels if a phlebotomy regimen is adhered to. However, phlebotomy procedures may cause and may be associated with pain, bruising and scarring at the venous puncture site, fatigue and dizziness during and following the procedure and disruption of daily activities. Furthermore, phlebotomy is not appropriate in patients with poor venous access, anemia or heart disease.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 (synthetic human angiotensin II) is being developed for the potential treatment of hypotension in adult patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. LJPC-401 (synthetic human hepcidin) is being developed for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. For more information on La Jolla, please visit www.ljpc.com.

Forward Looking Statement Safe Harbor

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors, that may cause actual results to be materially different from those anticipated by the forward-looking statements. The Company cautions readers not to place undue reliance on any such forward- looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s website www.sec.gov. These risks include, but are not limited to, risks relating to: the timing, costs, conduct and outcome of clinical studies; the anticipated treatment of future clinical data by the FDA, the EMA or other regulatory authorities, including whether such data will be sufficient for approval; the timing and prospects for approval of LJPC-501 or LJPC-401 by the FDA, the EMA or other regulatory authorities; risks relating to the scope of product labels (if approved); potential market sizes; the success of future development activities; potential indications for which the Company’s product candidates may be developed; the anticipated timing for regulatory actions; the impact of pharmaceutical industry regulation and healthcare legislation in the United States; and the success of future development activities. The Company expressly disclaims any intent to update any forward-looking statements to reflect the outcome of subsequent events.

Company Contacts

Sandra Vedrick

Associate Director, Investor Relations & Human Resources

La Jolla Pharmaceutical Company Phone: 858 207 4264 Ext: 1135 Email: svedrick@ljpc.com

and

Dennis M. Mulroy

Chief Financial Officer

La Jolla Pharmaceutical Company Phone: 858 207 4264 Ext: 1040 Email: dmulroy@ljpc.com

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

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

 

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

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of synthetic human angiotensin II. Angiotensin II is a major bioactive component of the renin-angiotensin-aldosterone system (RAAS). The

RAAS system is one of three central regulators of blood pressure. LJPC-501 is a vasopressor that leverages the RAAS system. LJPC-501 is being developed for the treatment of patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy (catecholamines and/or vasopressin).

In August 2017, the Company announced that its New Drug Application (NDA) for LJPC-501 had been accepted for review by the U.S. Food and Drug Administration (FDA). The review classification for the application is Priority, and the user fee goal date under the Prescription Drug User Fee Act (PDUFA) is February 28, 2018. The NDA is based on results of the ATHOS- 3 (Angiotensin II for the Treatment of High Output Shock) multicenter, randomized, double- blind, placebo-controlled, Phase 3 clinical study of LJPC-501 in patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. The study was conducted under a Special Protocol Assessment (SPA) agreed to with the FDA in 2015. A total of 344 patients were randomized across nine countries, 321 of whom received study treatment and were included in the primary analysis. In May 2017 the results of the ATHOS-3 study were published by The New England Journal of Medicine in an article entitled “Angiotensin II for the Treatment of Vasodilatory Shock”.

About Patients with Distributive or Vasodilatory Shock Failing Standard Therapy

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

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of synthetic human angiotensin II for the treatment of hypotension in adult patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. LJPC-401 is La Jolla’s proprietary formulation of synthetic human hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. LJPC-30S is La Jolla’s next-generation gentamicin derivative program that is focused on the potential treatment of serious bacterial infections as well as rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

Forward Looking Statement Safe Harbor

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors, that may cause actual results to be materially different from those anticipated by the forward-looking statements. The Company cautions readers not to place undue reliance on any such forward- looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the

U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing and prospects for approval of LJPC-501 by the FDA, EMA and other regulatory authorities; risks relating to the scope of product labels (if approved); potential market sizes; the anticipated timing for regulatory actions; the impact of pharmaceutical industry regulation and health care legislation in the Europe and the United States; and the success of future development activities. The Company expressly disclaims any intent to update any

forward-looking statements to reflect the outcome of subsequent events.

Company Contacts

Sandra Vedrick

Associate Director, Investor Relations & Human Resources

La Jolla Pharmaceutical Company Phone: 858-256-7910

Email: svedrick@ljpc.com

and

Dennis M. Mulroy

Chief Financial Officer

La Jolla Pharmaceutical Company Phone: 858-433-6839

Email: dmulroy@ljpc.com

Media Contact

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

Email: matt.middleman@lifescipublicrelations.com

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

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

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

 

The abstract published online today includes results from a pre-specified analysis of the ATHOS-3 (Angiotensin II for the Treatment of High Output Shock) multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical study of LJPC-501 (synthetic human angiotensin II) in patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. The authors present data showing that a relatively low angiotensin II state (as measured by the ratio of angiotensin I to angiotensin II) predicted increased mortality in patients with vasodilatory shock, suggesting that the activity of angiotensin converting enzyme (ACE), which converts angiotensin I to angiotensin II, may play a critical physiologic role in patients with vasodilatory shock. Furthermore, there was a statistically significant treatment effect of LJPC-501 compared to placebo on mortality in these patients with a relatively low angiotensin II state (relative risk reduction of 36%; HR=0.64; 95% CI: 0.41-1.00; p=0.047).

 

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

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

Poster Number:         0703

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

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

Session Date:             Tuesday, September 26, 2017

Session Time:            16:00 – 17:50

Session Room:           Area Graz

About LJPC-501

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

In August 2017, the Company announced that its New Drug Application (NDA) for LJPC-501 had been accepted for review by the U.S. Food and Drug Administration (FDA). The review classification for the application is Priority, and the user fee goal date under the Prescription Drug User Fee Act (PDUFA) is February 28, 2018. The NDA is based on results of the ATHOS- 3 (Angiotensin II for the Treatment of High Output Shock) multicenter, randomized, double- blind, placebo-controlled, Phase 3 clinical study of LJPC-501 in patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. The study was conducted under a Special Protocol Assessment (SPA) agreed to with the FDA in 2015. A total of 344 patients were randomized across nine countries, 321 of whom received study treatment and were included in the primary analysis. In May 2017 the results of the ATHOS-3 study were published by The New England Journal of Medicine in an article entitled “Angiotensin II for the Treatment of Vasodilatory Shock”.

About Patients with Distributive or Vasodilatory Shock Failing Standard Therapy

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

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of synthetic human angiotensin II for the treatment of hypotension in adult patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. LJPC-401 is La Jolla’s proprietary formulation of synthetic human hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. LJPC-30S is La Jolla’s next-generation gentamicin derivative program that is focused on the potential treatment of serious bacterial infections as

well as rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

Forward Looking Statement Safe Harbor

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors, that may cause actual results to be materially different from those anticipated by the forward-looking statements. The Company cautions readers not to place undue reliance on any such forward- looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the

U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing and prospects for approval of LJPC-501 by the FDA and other regulatory authorities; risks relating to the scope of product labels (if approved); potential market sizes; the anticipated timing for regulatory actions; the impact of pharmaceutical industry regulation and health care legislation in the United States; and the success of future development activities. The Company expressly disclaims any intent to update any forward-looking statements to reflect the outcome of subsequent events.

Company Contacts

Sandra Vedrick

Associate Director, Investor Relations & Human Resources

La Jolla Pharmaceutical Company Phone: 858-256-7910

Email: svedrick@ljpc.com

and

Dennis M. Mulroy

Chief Financial Officer

La Jolla Pharmaceutical Company Phone: 858-433-6839

Email: dmulroy@ljpc.com

Media Contact

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

Email: matt.middleman@lifescipublicrelations.com

La Jolla Pharmaceutical Company Announces Launch of Expanded Access Program for LJPC-501

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

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

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

About LJPC-501 Expanded Access Program

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

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

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

FDA guidance on expanded access can be found here.

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

About LJPC-501

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

About Catecholamine Resistant Hypotension

Catecholamine resistant hypotension or clinically refractory hypotension (CRH) is a life-threatening syndrome in patients with vasodilatory (also known as distributive) shock (dangerously low blood pressure with adequate cardiac function) who cannot achieve target mean arterial pressure (MAP) despite adequate fluid resuscitation and treatment with currently available vasopressors (catecholamines and/or vasopressin). There are approximately 500,000 distributive shock cases in the United States per year, an estimated 200,000 of which develop CRH. On current therapies, more than 50% of CRH patients die within 30 days.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of synthetic human angiotensin II for the potential treatment of catecholamine resistant hypotension. LJPC-401 is La Jolla’s proprietary formulation of synthetic human hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. LJPC-30S is La Jolla’s next-generation gentamicin derivative program that is focused on the potential treatment of serious bacterial infections as well as rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

Forward Looking Statement Safe Harbor

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the Company’s future results of operations. These statements are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors, that may cause actual results to be materially different from those anticipated by the forward-looking statements. The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the Company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing and availability of LJPC-501 under the expanded access program; the timing and prospects for approval of LJPC-501 by the FDA and the other regulatory authorities; risks relating to the scope of product labels (if approved) and potential market sizes, as well as the broader commercial opportunity; the anticipated timing for regulatory actions; and the success of future development activities; potential indications for which the Company’s product candidates may be developed. The Company expressly disclaims any intent to update any forward-looking statements to reflect the outcome of subsequent events.

Source: La Jolla Pharmaceutical Company

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

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

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

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

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

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

About the ATHOS-3 Study

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

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

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of synthetic human angiotensin II. Angiotensin II, the major bioactive component of the renin-angiotensin system, serves as one of the body’s central regulators of blood pressure. LJPC-501 is being developed for the treatment of patients with CRH. LJPC-501 is the first synthetic human angiotensin II product candidate to be tested in a Phase 3 study.

About Catecholamine Resistant Hypotension

Catecholamine resistant hypotension (CRH) is a life-threatening syndrome in patients with vasodilatory (also known as distributive) shock (dangerously low blood pressure with adequate cardiac function) who cannot achieve target mean arterial pressure (MAP) despite adequate fluid resuscitation and treatment with currently available vasopressors (catecholamines and/or vasopressin). There are approximately 500,000 distributive shock cases in the United States per year, an estimated 200,000 of which develop CRH. More than 50% of CRH patients die within 30 days.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of synthetic human angiotensin II for the potential treatment of catecholamine resistant hypotension. LJPC-401 is La Jolla’s proprietary formulation of synthetic human hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. LJPC-30S is La Jolla’s next-generation gentamicin derivative program that is focused on the potential treatment of serious bacterial infections as well as rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

Forward Looking Statement Safe Harbor

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the company’s future results of operations. These statements are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors, that may cause actual results to be materially different from those anticipated by the forward-looking statements. The company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing of the planned NDA submission for LJPC-501 and prospects for approval of LJPC-501 by the FDA and the other regulatory authorities; risks relating to the scope of product labels (if approved) and potential market sizes, as well as the broader commercial opportunity; the anticipated timing for regulatory actions; and the success of future development activities; potential indications for which the company’s product candidates may be developed. The company expressly disclaims any intent to update any forward-looking statements to reflect the outcome of the consequent events.

Source: La Jolla Pharmaceutical Company

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

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

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

— Trend toward longer survival observed

— New Drug Application planned for second half of 2017

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

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

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

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

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

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

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

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

La Jolla will host a conference call and webcast at 8:30 a.m. EST (5:30 a.m. PST) on Monday, February 27, 2017. The conference call can be accessed by dialing 877-359-9508 for domestic callers and 224-357-2393 for international callers. Please provide the operator with the passcode 78311826 to join the conference call or click here for the webcast. A slide presentation accompanying today’s press release and the conference call may also be found on La Jolla’s website at www.ljpc.com under the investor relations section. An archive of the conference call and webcast will be available on La Jolla’s website for 30 days following the call.

About the ATHOS-3 Study

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

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

About LJPC-501

LJPC-501 is La Jolla’s proprietary formulation of synthetic human angiotensin II. Angiotensin II, the major bioactive component of the renin-angiotensin system, serves as one of the body’s central regulators of blood pressure. LJPC-501 is being developed for the treatment of patients with catecholamine resistant hypotension (CRH). LJPC-501 is the first synthetic human angiotensin II product candidate to be tested in a Phase 3 study.

About Catecholamine Resistant Hypotension

Catecholamine resistant hypotension (CRH) is a life-threatening syndrome in patients with distributive shock (dangerously low blood pressure with adequate cardiac function) who cannot achieve target mean arterial pressure (MAP) despite adequate fluid resuscitation and treatment with currently available vasopressors (catecholamines and/or vasopressin). There are approximately 500,000 distributive shock cases in the United States per year, an estimated 200,000 of which develop CRH. More than 50% of CRH patients die within 30 days.

About La Jolla Pharmaceutical Company

La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of synthetic human angiotensin II for the potential treatment of catecholamine resistant hypotension. LJPC-401 is La Jolla’s proprietary formulation of synthetic human hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. LJPC-30S is La Jolla’s next-generation gentamicin derivative program that is focused on the potential treatment of serious bacterial infections as well as rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy. For more information on La Jolla, please visit www.ljpc.com.

Forward Looking Statement Safe Harbor

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or the company’s future results of operations. These statements are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors, that may cause actual results to be materially different from those anticipated by the forward-looking statements. The company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties, and other factors are described in greater detail in the company’s filings with the U.S. Securities and Exchange Commission (SEC), all of which are available free of charge on the SEC’s web site www.sec.gov. These risks include, but are not limited to, risks relating to: the timing of the NDA submission for LJPC-501 and prospects for approval of the NDA; risks that the full data set from the ATHOS-3 study will not be consistent with the top-line results of the study; risks relating to the scope of product labels (if approved) and potential market sizes, as well as the broader commercial opportunity; the anticipated timing for regulatory actions; the success of future development activities; potential indications for which the company’s product candidates may be developed; and the expected duration over which the company’s cash balances will fund its operations. Subsequent written and oral forward-looking statements attributable to the company or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in the company’s reports filed with the SEC. The company expressly disclaims any intent to update any forward-looking statements.

Source: La Jolla Pharmaceutical Company

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