La Jolla Pharma (LJPC) is rallying this morning (+63%) on some positive clinical data news. Before getting into the details, let's provide some quick background on La Jolla Pharma. It's a San Diego-based biopharmaceutical company focused on developing therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The company has several product candidates in development. Its three primary product candidates are as follows:
- LJPC-501 is La Jolla's proprietary formulation of synthetic human angiotensin II for the potential treatment of catecholamine resistant hypotension (CRH).
- LJPC-401 is La Jolla's proprietary formulation of synthetic human hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome.
- LJPC-30S is La Jolla's next-generation gentamicin derivative program that is focused on the potential treatment of serious bacterial infections as well as rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy.
Turning to today's news, La Jolla announced positive top-line results from its ATHOS-3 Phase 3 study of LJPC-501 (angiotensin II) in patients with catecholamine resistant hypotension (CRH). 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. There are approximately 500,000 distributive shock cases in the US per year, an estimated 200,000 of which develop CRH. More than 50% of CRH patients die within 30 days.
La Jolla describes the analysis of the primary efficacy endpoint as being 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. In addition, a trend toward longer survival was observed: 22% reduction in mortality risk through day 28 for LJPC-501-treated patients.
ATHOS-3 was conducted under a Special Protocol Assessment with the FDA, in which the company and FDA agreed on the study design, study endpoints and study analyses. Daniel Sessler, M.D. of the Cleveland Clinic says that these study results support that angiotensin II, a molecule first synthesized by Dr. Irvine Page at the Cleveland Clinic, improves outcomes in distributive shock patients requiring high-dose catecholamines. Given the high mortality from this condition, it is important to offer physicians another potential treatment option.
In collaboration with the investigators, La Jolla plans to present and publish detailed results from the ATHOS-3 study later this year. La Jolla also plans to meet with the FDA to discuss its NDA submission planned for the second half of 2017.
In sum, investors are clearly pleased with this clinical data as the stock is jumping more than 60% to a new 52-week high. This stock has had its ups and downs over the past year, it rallied in September 2016 from $16 to $28 then fell back to $16 by November. But today's news is definitely a positive. It follows some encouraging results from La Jolla's Phase 1 trial of LJPC-401 in 4Q16. Looking ahead, La Jolla expects to initiate its pivotal trial for LJPC-401 in mid-2017.