One month after Akcea’s partner Novartis opted out of its triglyceride-targeting program, the treatment beat placebo handily at lowering blood triglyceride levels in patients who have, or are at risk of, heart disease. The phase 2 data tee it up for trials in specific cardiovascular ailments, including familial chylomicronemia syndrome (FCS).
The study tested various dose levels and frequencies of the antisense drug, known as AKCEA-APOCIII-LRx, against placebo in 114 patients. Patients received injections of the treatment or placebo once a week, every two weeks, or every month, for at least six months.
More than 90% of patients taking the highest dose saw their triglyceride levels drop to healthy levels, compared to just 5% of patients on placebo, the company announced on Wednesday. The treatment also made a “significant” dent in apolipoprotein C-III (ApoC-III), a protein made in the liver that plays a role in triglyceride metabolism, and two kinds of “bad” cholesterol called very low-density lipoprotein cholesterol and remnant cholesterol.
Its safety profile looked good, with side effects similar across treatment and placebo groups. These were mostly injection site reactions, the company said in a statement. Most patients (85%) finished treatment and patients in both groups quit the study at similar rates.
"Based on the positive results from this study, we plan to rapidly pursue development of AKCEA-APOCIII-LRx for familial chylomicronemia syndrome or FCS,” said Akcea Chief Medical Officer Louis O’Dea, M.D., in the statement.
FCS, a rare, genetic disorder that stops the body from breaking down fats, is the target of another Akcea program, volanesorsen, approved in the European Union as Waylivra. Although an FDA advisory committee voted 12-8 in favor of its approval, the agency rejected the drug. The company told BioSpace last summer it was working on a path forward for the treatment in the U.S. and Canada.
Akcea plans to bring AKCEA-APOCIII-LRx beyond FCS, though it hasn’t specified which cardiovascular diseases this might include.
“Because we were able to achieve substantial triglyceride lowering with this investigational medicine, we are also considering developing it for other rare and common diseases associated with elevated triglycerides. We are very grateful to the patients, families and physicians who participated in this study and are excited about the potential to bring this much-needed therapy to patients,” O’Dea added.