Application for Orphan Drug Designation Filed
July 8, 2014
La Jolla Pharmaceutical Company (the “Company” or “La Jolla”), a biotechnology company developing therapeutics targeting significant unmet, life-threatening diseases, today announced that the Company plans to begin a phase 3 registration program for LJPC-501 for the treatment of catecholamine-resistant hypotension (CRH), a new indication. Initiation of this registration program is the result of a recent meeting between the Company and the U.S. Food and Drug Administration at which agreement was reached that blood pressure is an appropriate primary endpoint for approval.
“We are excited to begin a registration program in CRH,” said George F. Tidmarsh, M.D., Ph.D., President and CEO of La Jolla. “There are approximately 75,000-100,000 patients per year who suffer from CRH, and the prognosis is very poor. We are excited to advance LJPC-501 into a registration trial for this significant unmet medical need.”
Hypotension, if uncorrected, is life-threatening and occurs as the result of various underlying conditions such as blood loss due to trauma, septic shock, poor heart function or drug reactions. The first line of treatment is catecholamine infusion. Catecholamines are derived from the amino acid tyrosine and include epinephrine (adrenaline), norepinephrine (noradrenaline), and dopamine, which act as neurotransmitters that increase blood pressure. While largely effective, some patients fail to respond to adequate doses and are defined as catecholamine-resistant. Furthermore, high doses of catecholamines can lead to cardiac and other toxicities, and can contribute to the high rate of mortality in these patients. Therefore, there is a major unmet need for a new pharmacological agent that can restore blood pressure while maintaining catecholamine doses at acceptable levels.
LJPC-501 (angiotensin II) is a peptide agonist of the renin-angiotensin system that acts to stabilize blood pressure. One of the most widely prescribed classes of blood pressure medication, angiotensin converting enzyme inhibitors, inhibits the production of angiotensin II, thereby reducing blood pressure. A randomized, placebo-controlled pilot study of angiotensin II in 20 patients with catecholamine-resistant hypotension was presented at the 34th International Symposium on Intensive Care and Emergency Medicine by Dr. Lakhmir S. Chawla from George Washington University. This study demonstrated that the mean norepinephrine dose for the placebo cohort was 20.1 ± 16.8 μg/minute vs. 7.3 ± 11.9 μg/minute for the angiotensin II cohort (P = 0.022). This was the pre-defined primary endpoint and a surrogate measure of blood pressure effect. Approximately 80% of patients treated with angiotensin II experienced a rise in blood pressure compared to 10-20% with placebo. The most common adverse event was hypertension, which occurred in 20% of patients receiving angiotensin II. Due to the estimated size of the patient population in the United States for this indication, the Company has filed for Orphan Drug status for angiotensin II.
About La Jolla Pharmaceutical Company
La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics for chronic organ failure and cancer. GCS-100, the Company’s lead product candidate, is a first-in-class inhibitor of galectin-3, a novel molecular target implicated in chronic organ failure and cancer. LJPC-501, the Company’s second product candidate, is a natural peptide being developed for the treatment of hepatorenal syndrome and CRH. LJPC-401, the Company’s third product candidate, is a natural peptide being developed for the treatment of iron overload. For more information on the Company please visit http://www.ljpc.com.