Ionis Reports Substantial Drop In Acute Pancreatitis Events With Cholesterol Lowering Drug

Core Findings - Ionis Pharmaceuticals reported results from the Phase 3 CORE and CORE2 studies of olezarsen for severe hypertriglyceridemia (sHTG), achieving a statistically significant placebo-adjusted mean reduction in fasting triglyceride levels of up to 72% at six months, sustained through 12 months [2][5] - Olezarsen demonstrated an 85% reduction in acute pancreatitis events, with 86% of treated patients achieving triglyceride levels below 500 mg/dL, the risk threshold for acute pancreatitis [3][6] Study Details - Nearly 1,100 patients were enrolled in the CORE and CORE2 studies, marking the largest pivotal program ever conducted in sHTG, with participants required to be on standard-of-care lipid-lowering therapy [5] - The studies showed favorable safety and tolerability for olezarsen, with significant reductions in secondary endpoints including apoC-III, remnant cholesterol, and non-HDL-C [4][7] Future Plans - Ionis is set to submit a supplemental new drug application for both the 50 mg and 80 mg doses to the FDA by the end of the year, with an ongoing open-label extension study where over 90% of patients from CORE and CORE2 chose to continue [8] Efficacy Metrics - In terms of triglyceride levels, 89% and 88% of patients on olezarsen 50 mg and 80 mg, respectively, achieved levels below 880 mg/dL, while 34% and 54% achieved normal triglyceride levels below 150 mg/dL [9] - The number needed to treat (NNT) analysis indicates that treating 20 patients with olezarsen is estimated to prevent one acute pancreatitis event over one year, and treating four patients in the highest risk group is estimated to prevent one event over one year [9]