Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) jumped 22% on Tuesday after unveiling highly positive topline results from its pivotal Phase 3 CORE and CORE2 trials evaluating olezarsen in patients with severe hypertriglyceridemia (sHTG).
The therapy not only lowered fasting triglyceride levels by as much as 72% versus placebo, but also achieved an 85% reduction in acute pancreatitis events—marking the first time such an outcome has been demonstrated in treating sHTG.
In the CORE study, patients receiving monthly doses of 80mg and 50mg of olezarsen saw triglyceride reductions of 73% and 63% from baseline, compared with just 0.5% for placebo. Findings from CORE2 echoed these results, showing reductions of 68% and 63% at the same doses, while placebo patients experienced a 14% decline.
The treatment was generally well tolerated, with side effects balanced between groups. Mild injection site reactions were the most common adverse event. More than 90% of participants who completed the studies opted to move into the open-label extension.
“These data are groundbreaking, demonstrating that olezarsen is the first therapy for sHTG to significantly reduce acute pancreatitis events,” said Sam Tsimikas, senior vice president of global cardiovascular development at Ionis.
The company said it intends to file a supplemental new drug application with the FDA before year-end. If cleared, olezarsen would become Ionis’ third standalone product launch within two years.
Full study findings—including data from nearly 1,100 patients on standard lipid-lowering regimens—are set to be presented at an upcoming medical meeting.
Ionis Pharmaceuticals stock price
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