La Jolla Pharmaceutical is turning its focus to its lone FDA-approved product and another one in the pipeline after an experimental drug for a rare blood disease failed a late-stage study.
On Monday, La Jolla Pharma (NASDAQ: LJPC) said it was halting a clinical trial testing LJPC-401, its drug candidate for iron overload in patients with beta thalassemia. The company said an interim analysis showed no benefit compared to the placebo. On the same day the company said CEO George Tidmarsh had left “to pursue other interests,” and that a search for a new chief executive was underway.
The La Jolla Pharma drug is a synthetic version of human hepcidin, a hormone that regulates iron metabolism. In the wake of the failure it plans to “reassess” the continued development of the investigational drug, including tests in patients with another iron overload disorder.
Beta thalassemia is caused by a mutation in the gene that codes for hemoglobin, which carries oxygen throughout the body. Some patients require regular blood transfusions to handle the symptoms, and those transfusions can cause dangerous levels of iron to accumulate in their organs. Patients need another treatment, chelation therapy, to remove the buildup.
La Jolla Pharma was testing its drug in 100 patients with the disorder who—despite chelation therapy—have abnormally high levels of iron in the heart. The latest analysis, which looked at data from about half of the enrolled patients, showed no significant difference between those receiving standard chelation therapy and those receiving the therapy and the La Jolla Pharma drug. The data also showed no significant difference on secondary goals, including blood iron levels.
Following the news, the company’s stock price fell more than 50 percent, from $5.62 apiece as of market close Friday to $2.59 per share on Monday.
Other companies are developing treatments for beta thalassemia. Cambridge, MA-based Bluebird Bio (NASDAQ: BLUE) earlier this year received approval from European regulators for Zynteglo, a gene therapy it developed to treat the disorder. Vertex Pharmaceuticals (NASDAQ: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) are testing a gene-edited therapy for beta thalassemia.
The La Jolla Pharma drug is also in mid-stage trials as a potential treatment for hereditary hemochromatosis, a condition that causes patients to absorb too much iron. Results from a Phase 2 study showed an average 33 percent reduction in iron levels in those given the drug compared to 3 percent in those receiving a placebo, the company said Monday. But the failure in beta thalassemia now has it assessing whether to continue developing the drug for hereditary hemochromatosis.
Nearly two years have passed since La Jolla Pharma, founded in 1989, brought its first drug to market. The FDA OK’d angiotensin II (Giaprezatm), a drug to increase blood pressure in patients with septic or other types of shock; it was also approved this year by European regulators to treat a subset of patients with those conditions whose blood pressure hasn’t responded to other therapies. That treatment, which was launched in the US in March 2018, brought in $15.8 million in the first nine months of 2019, according to company documents. In that time, La Jolla Pharma reported a net loss of $29 million.
In the wake of LJPC-401’s failure, La Jolla Pharma says it will focus on maximizing sales of angiotensin II in the US and Europe. It also plans to devote resources to artesunate, an experimental treatment for severe malaria that has been recently submitted to the FDA for review.