(HealthDay News) — An experimental drug, ISIS 304801, can lower triglyceride levels by as much as 71%, according to study results published in the New England Journal of Medicine.

Joseph Witztum, MD, a professor of medicine at the University of California, San Diego, and colleagues treated 57 patients with the drug (n=41) or an inactive placebo (n=16). The patients’ triglyceride levels ranged between 350 mg/dL and 2,000 mg/dL, and they received weekly doses of the drug over 13 weeks. 

The study also included 28 people who had triglyceride levels ranging from 225 mg/dL to 2,000 mg/dL, who had been receiving fibrate therapy. These patients also received the new drug or placebo.

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Overall, the researchers found that ISIS 304801 reduced triglyceride levels 31.3% to 70.9%. Results also linked ISIS 304801 to dose-dependent and prolonged decreases in plasma apolipoprotein C-III (APOC3) levels when administered as monotherapy and as an add-on to fibrates

No safety concerns were identified.

These results delineate the contribution of APOC3 to the mechanisms through which the body manages triglycerides, according to study researcher Daniel Gaudet, MD, PhD, of the University of Montreal.

“Decoding mechanisms opens the door to precise, individual interventions for the prevention of residual risk associated with the various causes of severe hypertriglyceridemia,” Gaudet said in a press release. 

“The results of these studies enable the acceleration of research targeting better understanding and control of the risk trajectory associated with various forms of severe hypertriglyceridemia.”

This trial was the second of three required for drug approval in the United States. Witztum told HealthDay that phase 3 trials are underway, but the results will not be available for 2 to 3 years.

The study was funded by Isis Pharmaceuticals, the manufacturer of ISIS 304801.


  1. Gaudet D et al. N Engl J Med. 2015;373:438-447.