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COMPASS: Agent reduces triglycerides in patients with severe hypertriglyceridemia
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Akcea Therapeutics and Ionis Pharmaceuticals announced that a novel antisense inhibitor of apolipoprotein C-III for treatment of patients with severe hypertriglyceridemia was associated with significant reductions in triglycerides in a phase 3 study.
Researchers conducted COMPASS, a randomized, double blind, placebo-controlled study of volanesorsen in participants with an average triglyceride level of 1,262 mg/dL.
Participants treated with volanesorsen (n = 75) achieved a mean reduction in triglycerides of 71.2% from baseline after 13 weeks vs. 0.9% in patients assigned placebo (n = 38); the treatment effect was maintained through the end of the 26-week treatment, according to a press release.
In a subset of patients with familial chylomicronemia syndrome (n = 7), participants who were treated (n = 5) had a mean reduction in triglycerides of 73% from baseline to 13 weeks, compared to a mean increase of 70% in those assigned placebo (n = 2), an effect that was continued through the 26-week treatment period, according to the release.
Of the participants treated, 82% achieved triglyceride levels less than 500 mg/dL after 13 weeks of treatment compared with 14% of placebo participants (P < .0001), and the mean absolute reduction in triglyceride levels in those assigned volanesorsen was 869 mg/dL, the companies stated in the release.
“Current therapies are inadequate for patients with extremely high triglycerides, most specifically those with [familial chylomicronemia syndrome], leaving them at risk for significant morbidity and mortality, including pancreatitis,” Daniel Gaudet, MD, associate professor of medicine and director of the Community Genomic Medicine Center at the Université de Montréal and scientific director and strategic development officer of BioBank, Genome Quebec’s Technological Centers, said in the release. “People with [familial chylomicronemia syndrome] have particular challenges because in addition to have triglyceride levels that can be 10 to 20 times normal values, the currently available triglyceride-lowering drugs are usually ineffective. The data from studies in patients with [familial chylomicronemia syndrome] treated with volanesorsen, including those from the COMPASS study, show that for the first time these patients can achieve the triglyceride reductions needed to potentially improve their health.”
The most common adverse event was injection-site reactions, most of which were mild. According to the release, of the participants, 13% discontinued treatment due to injection site reactions, 7% discontinued for other non-serious adverse events and there were no deaths.
COMPASS is one of four phase 3 trials for volanesorsen and more data from it will be presented at an upcoming medical meeting, according to the release.
Disclosure: Gaudet reports serving on an advisory board for or participating in trials sponsored by Amgen, AstraZeneca, Cymabay, Gemphire, Ionis Pharmaceuticals, Novartis, Pfizer, Regeneron, Sanofi and Uniqure.