Ionis Pharmaceuticals(US:IONS) Businesswireยท2025-11-08 14:40Core Findings - Olezarsen demonstrated a placebo-adjusted mean reduction in fasting triglyceride levels of up to 72% at six months, with reductions sustained through 12 months [2][4][11] - 86% of patients treated with olezarsen achieved triglyceride levels below 500 mg/dL, which is below the risk threshold for acute pancreatitis [2][4][5] - Olezarsen is the first investigational treatment for severe hypertriglyceridemia (sHTG) to significantly reduce acute pancreatitis events, showing an 85% reduction in such events [2][5][7] Study Details - The CORE and CORE2 studies involved nearly 1,100 patients, making it the largest pivotal program for sHTG [4][11] - Participants were required to be on standard lipid-lowering therapy and had baseline triglyceride levels of at least 500 mg/dL [11][14] - The studies met their primary endpoint across doses, with significant reductions in triglyceride levels observed [4][11] Safety and Tolerability - Olezarsen exhibited a favorable safety profile, with adverse events balanced across treatment arms [7][8] - Serious adverse events were less frequent in the olezarsen group compared to placebo [7][8] - The most common treatment-emergent events were mild injection site reactions [7][8] Future Prospects - Ionis Pharmaceuticals plans to submit a supplemental new drug application for olezarsen to the FDA by the end of the year [9] - An open-label extension study of olezarsen for sHTG is ongoing, with over 90% of patients from the CORE and CORE2 studies opting to continue [9][10] - Olezarsen is expected to be one of two independent launches for Ionis in 2026, targeting a larger patient population at risk of acute pancreatitis [7][9]