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Investing.com -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) stock rose 22% after the company reported positive topline results from its pivotal Phase 3 CORE and CORE2 studies of olezarsen in patients with severe hypertriglyceridemia (sHTG).
The studies showed that olezarsen significantly reduced fasting triglycerides by up to 72% compared to placebo and demonstrated an 85% reduction in acute pancreatitis events. This marks the first and only time such a reduction in pancreatitis events has been achieved for sHTG treatment.
In the CORE study, olezarsen at 80mg and 50mg monthly doses reduced triglycerides by 73% and 63% from baseline respectively, compared to just 0.5% for placebo. The CORE2 study showed similar results with 68% and 63% reductions at the same dosage levels versus 14% for placebo.
The drug demonstrated a favorable safety profile, with adverse events generally balanced across treatment groups. Injection site reactions, mostly mild, were the most common side effect. More than 90% of patients who completed the studies chose to continue into the open-label extension study.
"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 plans to submit a supplemental new drug application to the FDA by the end of this year. If approved, olezarsen for sHTG would mark Ionis’ third independent product launch in under two years.
Detailed results from the studies, which included nearly 1,100 patients on standard of care lipid-lowering therapy, will be presented at an upcoming medical conference.
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