La Jolla hits endpoint in phase 3 CRH test, preps for FDA talks

The biotech will discuss its NDA submission to the FDA in the second half of 2017.

La Jolla Pharmaceutical has posted positive top-line data from its late-stage trial of LJPC-501 in patients with a severe condition known as catecholamine-resistant hypotension (CRH) as it looks to discuss an NDA with the U.S. regulator later this year.

The test’s primary endpoint was set as the percentage of patients hitting targeted blood pressure response, and the biotech said its med hit the heights: Only 23% of the 158 placebo-treated patients had this blood response, compared to 70% of the 163 LJPC-501-treated patients (p < 0.00001).

The primary endpoint was the percentage of patients with a mean arterial pressure (or MAP) of ≥ 75 mmHg or a 10-mmHg increase from baseline MAP at three hours following treatment and without an increase in standard-of-care vasopressors.

Under the trial’s guidelines (which came via an FDA Special Protocol Assessment), patients were randomized to receive either LJPC-501 or placebo on a background of standard-of-care vasopressors selected by the investigators.

Although not geared for survival, the company also noted "a trend toward longer survival was observed: 22% reduction in mortality risk through day 28" for LJPC-501-treated patients. But with a hazard ratio of 0.78 (0.57-1.07) and p = 0.12, the data was confirmed by a company spokesman as not being statistically significant.

The company did not however give an update on a secondary efficacy endpoint of the study, i.e., the comparison between its med and a dummy therapy in helping to lower the risk of organ failure.

The biotech said in its PR that these are only top-line data, and more detailed numbers will be published from the ATHOS-3 study “later this year,” according to a statement.

LJPC-501 is La Jolla’s formulation of a synthetic human angiotensin II, the major bioactive component of the renin-angiotensin system, which serves as one of the body’s central regulators of blood pressure.

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 despite treatment.

There are around half a million distributive shock cases in the U.S. every year, with the biotech estimating that around 200,000 of these patients will go on to develop CRH. More than half of these patients die within a month.

“We are grateful to the patients, their families and the dedicated medical teams who contributed to this successful study,” said George Tidmarsh, M.D., PhD., president and CEO of La Jolla.

Tidmarsh added that he and the biotech “look forward to meeting with the FDA to discuss our NDA submission planned for the second half of this year.”

This will be welcome news for the biotech, which back in 2015 pulled the plug on a pair of projects, including one for the common liver disease NASH, after the FDA set out a difficult regulatory road ahead.

The company, which had a market cap of around $400 million as of last Friday, was up as high as 53% premarket on the news this morning, before edging down to around 30%.