Dive Brief:
- La Jolla Pharmaceutical announced positive top-line results from the ATHOS-3 Phase 3 study of LJPC-501 (angiotensin II) in certain shock patients on Monday morning, sending shares up more than 75% to trade near $35 per share.
- In the clinical trial, 70% of patients with catecholamine resistant hypotension who were treated with LJPC-501 achieved the target blood pressure response at three hours. That compares with 23% of patients treated with placebo. There was also a trend toward longer survival in patients treated with LJPC-501: a 22% reduction in mortality risk through day 28.
- The San Diego-based pharma plans to meet with the Food and Drug Administration to discuss its New Drug Application submission planned for the second half of 2017, La Jolla President and CEO George Tidmarsh said during a Feb. 27 conference call. He added that La Jolla will request priority review.
Dive Insight:
Dive Insight:
"This is a very costly, prevalent and deadly disease," Tidmarsh said, noting that shock affects one-third of ICU patients across the U.S. and can kill within hours. The 30-day mortality rate tops 50% for shock patients (adults and children alike), well above the 16% rate for pneumonia and 14% rate for acute myocardial infarction. Moreover, the average treatment cost for a shock patient approaches $90,000 versus $42,000 for AMI and nearly $31,000 for pneumonia.
Currently, norepinephrine, a drug in the market since the early 1950s, is the first-line standard of care that treats about 703,000 shock patients annually in the U.S., costing about $153 per patient. If that treatment fails, about 244,000 patients get vasopressin at a cost of about $1,385 per patient. LJPC-501 would come into play for the estimated 196,000 shock patients who don’t adequately respond to the first- or second-line treatments, Tidmarsh explained. "This is our target population for a new trial," he added.
He noted that La Jolla has formulated the first synthetic human angiotensin II, a naturally occurring regulator of blood pressure, to be tested in a randomized Phase 3 study. ATHOS-3, which treated 321 adult patients at 74 centers in nine countries, completed enrollment in December 2016. The trial was conducted under a Special Protocol Assessment (SPA) with the FDA.
La Jolla is on track with manufacturing of LJPC-501 for the NDA, having completed several batches of the product, and has exclusivity on the product until at least 2034.
Tidmarsh wouldn't comment on the product’s market potential and number of sales representatives that would be required. He said La Jolla is focused in the near term on getting the product through the regulatory process. Upon approval, he said, the company "will do everything we can to get [LJPC-501] to as many patients as possible." He added that the company plans to take the data and discuss a regulatory pathway in Europe, though it has yet to meet with authorities there. La Jolla plans to build its own infrastructure to provide the drug to patients in the U.S.
The company would discuss only top-line trial results, noting that the company plans to discuss details and results at a major scientific meeting and to publish them in a "top-tier" peer-reviewed journal later this year.