On August 1, 2019 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, reported financial results for the three and six months ended June 30, 2019 and highlighted recent corporate progress (Press release, La Jolla Pharmaceutical, AUG 1, 2019, View Source [SID1234538315]).

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Recent Corporate Progress

GIAPREZATM (angiotensin II)

Net Sales: For the three months ended June 30, 2019, GIAPREZA net sales were $5.7 million, up 258% from the same period in 2018, and up 30% from the three months ended March 31, 2019. For the six months ended June 30, 2019, GIAPREZA net sales were $10.1 million, up 320% from the same period in 2018.

Positive CHMP Opinion: In June 2019, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for La Jolla’s Marketing Authorisation Application (MAA) for GIAPREZA for the treatment of refractory hypotension in adults with septic or other distributive shock. The CHMP’s positive opinion was sent to the European Commission (EC), which has the authority to approve medicines for the 28 European Union member countries. Approval would also be recognized in Iceland, Norway and Liechtenstein. We expect a final approval decision on the GIAPREZA MAA by the EC in the third quarter of 2019.

Investigational Products

Breakthrough Therapy Designation and Orphan Drug Designation Received from the FDA for LJPC-0118 (artesunate): The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation and Orphan Drug designation for LJPC-0118 for the treatment of malaria in April 2019 and July 2019, respectively. The active pharmaceutical ingredient in LJPC-0118, artesunate, was demonstrated to be superior to quinine in reducing mortality in patients with severe falciparum malaria infection in two randomized, controlled, clinical studies. We plan to file a New Drug Application (NDA) for LJPC-0118 with the FDA in the fourth quarter of 2019.

Positive Results Announced from Pre-Specified Interim Analysis of Phase 2 Study of LJPC-401 in Patients with Hereditary Hemochromatosis: In June 2019, we announced positive results from the pre-specified interim analysis of our Phase 2 study of LJPC-401 (synthetic human hepcidin) in patients with hereditary hemochromatosis (HH). The interim analysis of efficacy included 26 patients who had reached the end of the 16-week treatment period, and the interim analysis of safety included 60 randomized patients. Treatment with LJPC-401 resulted in a statistically significant reduction in transferrin saturation (TSAT) from baseline to the end of treatment (16 weeks), the primary efficacy endpoint of the study: LJPC‑401‑treated patients had a mean reduction in TSAT of 42% compared to placebo-treated patients who had a mean reduction of 6% (p<0.0001). The requirement for and frequency of phlebotomy procedures, a key secondary endpoint of the study, also was statistically significant: LJPC-401-treated patients had 0.06 phlebotomies per month compared to placebo-treated patients who had 0.41 phlebotomies per month (p=0.003). There were 3 phlebotomies in 2 LJPC-401-treated patients and 24 phlebotomies in 9 placebo-treated patients. LJPC-401 was well tolerated. The most frequent treatment-emergent adverse events (TEAEs) were injection site reactions (ISRs). The ISRs were all mild or moderate in severity, and no ISRs resulted in treatment discontinuation. As of the interim analysis, there were no serious TEAEs reported. We expect to announce top-line results of LJ401-HH01 in the fourth quarter of 2019.

"We are pleased with the progress made in the first half of 2019, which included the achievement of significant milestones for each of GIAPREZA, LJPC-0118 and LJPC-401," said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. "In the second half of 2019, we look forward to continued growth in GIAPREZA net sales, the final approval decision

on the GIAPREZA MAA by the EC, our filing of an NDA for LJPC-0118 with the FDA and top-line results of our Phase 2 study of LJPC-401 in patients with HH."

Financial Results

For the three and six months ended June 30, 2019, GIAPREZA net sales were $5.7 million and $10.1 million, respectively, compared to $1.6 million and $2.4 million, respectively, for the same periods in 2018. La Jolla’s net loss for the three and six months ended June 30, 2019 was $30.4 million and $62.1 million, or $1.12 per share and $2.29 per share, respectively, compared to $52.8 million and $103.3 million, or $2.02 per share and $4.22 per share, respectively, for the same periods in 2018. La Jolla continues to expect full-year 2019 GIAPREZA net sales of $24 million to $28 million.

As of June 30, 2019, La Jolla had $123.4 million in cash, compared to $172.6 million as of December 31, 2018. Net cash used in operating activities for the three and six months ended June 30, 2019 was $16.5 million and $49.2 million, respectively, compared to $37.5 million and $83.4 million, respectively, for the same periods in 2018. La Jolla has no debt. La Jolla continues to expect that its net cash used in operating activities in 2019 will be $89 million to $94 million.

About GIAPREZA

In December 2017, GIAPREZA (angiotensin II) 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. 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. GIAPREZA is marketed by La Jolla Pharmaceutical Company on behalf of La Jolla Pharma, LLC, its wholly owned subsidiary.

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 for the United States.

About LJPC-0118

LJPC-0118 is La Jolla’s investigational product for the treatment of severe malaria. The active pharmaceutical ingredient in LJPC-0118, artesunate, was demonstrated to be superior to quinine in reducing mortality in patients with severe falciparum malaria infection in two randomized, controlled, clinical studies. The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation and Orphan Drug designation for LJPC-0118 for the treatment of malaria in April 2019 and July 2019, respectively. La Jolla plans to file a New Drug Application (NDA) for LJPC-0118 with the FDA in the fourth quarter of 2019 for the treatment of severe malaria. Severe malaria is a serious and sometimes fatal disease caused by a parasite that

commonly infects a certain type of mosquito, which feeds on humans. Symptoms include but are not limited to: fever, chills, sweating, hypoglycemia and shock. Severe malaria is often complicated by central nervous system infections that may lead to delirium, which may progress to coma. Infections usually occur a few weeks after being bitten. In 2017, an estimated 219 million cases of malaria occurred worldwide, with an estimated 200 million of these cases occurring in the World Health Organization (WHO) African Region, and, in 2013, the global annual incidence of severe malaria was estimated to be 2 million cases. In 2017, an estimated 435,000 people died from malaria worldwide.

About LJPC-401

LJPC-401, a clinical-stage investigational product, is La Jolla’s proprietary formulation of synthetic human hepcidin. Hepcidin, an endogenous peptide hormone, is the body’s naturally occurring regulator of iron absorption and distribution. In healthy individuals, hepcidin prevents excessive iron accumulation in vital organs, such as the liver and heart, where it can cause significant damage and even result in death. La Jolla is developing LJPC-401 for the potential treatment of iron overload, which occurs as a result of primary iron overload diseases such as hereditary hemochromatosis (HH), or secondary iron overload diseases such as beta thalassemia (BT), sickle cell disease (SCD), myelodysplastic syndrome (MDS) and polycythemia vera. 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.