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
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
“These study results support that angiotensin II, a molecule first
synthesized by Dr.
“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
Conference Call at
La Jolla will host a conference call and webcast at
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
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
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forward-looking statements, which speak only as of the date they were
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La Jolla Pharmaceutical Company
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Dennis M. Mulroy
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