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Positive olezarsen Phase 3 data in familial chylomicronemia syndrome to be presented at 2024 American College of Cardiology (ACC) annual meeting
– First presentation of pivotal results showing significant reduction in triglycerides and substantial reduction in acute pancreatitis events in patients with
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"– First presentation of pivotal results showing significant reduction in triglycerides and substantial reduction in acute pancreatitis events in patients with rare, life-threatening disease, for which there are no approved treatment options in U.S. –\nCARLSBAD, Calif., March 25, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will present positive Phase 3 Balance results in patients with familial chylomicronemia syndrome (FCS) for the company's lead independent, investigational medicine, olezarsen, at the 2024 American College of Cardiology (ACC) Annual Meeting in Atlanta, Georgia.\n\n \n \n \n \n \n \n\n \nThe oral presentation will take place on April 7, 2024 at 10:08am ET during the session on Prevention and Health Promotion and the Year in Review. Ionis previously announced positive topline results. As previously shared, monthly dosing of olezarsen 80 mg met the primary endpoint with a statistically significant reduction in triglyceride levels at six months, demonstrated robust reductions in apolipoprotein C-III (apoC-III) and marked reductions in acute pancreatitis (AP) events versus placebo. Olezarsen also demonstrated a favorable safety and tolerability profile.\nIn addition, a late-breaking abstract entitled \"Efficacy and Safety of Olezarsen in Patients with Hypertriglyceridemia and High Cardiovascular Risk: Primary Results of the Bridge-TIMI 73a Trial\" will be presented on April 7, 2024 at 8:30am ET during the session on Late-Breaking Clinical Trials II.\nAbout the Balance Study\nThe global, multicenter, randomized, double-blind, placebo-controlled Phase 3 Balance study (NCT04568434) enrolled 66 patients aged 18 and older with confirmed FCS. Patients in the study received background therapies including statins, fibrates and omega-3 fatty acids. Patients were randomized in a 1:1:1 ratio to receive olezarsen 80 mg or 50 mg or placebo via subcutaneous injection once every four weeks for 53 weeks. The primary endpoint was the percent change from baseline in fasting triglyceride levels at six months compared to placebo. Secondary endpoints included percent changes in triglyceride levels at 12 months, percent changes in other lipid parameters, and adjudicated acute pancreatitis event rates over the treatment period.\nAbout Olezarsen\nOlezarsen is an RNA-targeted investigational LIgand...