Ionis Pharmaceuticals, Inc. announced that its CARDIO-TTRansform Phase 3 trial for eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) failed to meet its primary efficacy endpoint of composite cardiovascular mortality and recurrent cardiovascular clinical events up to Week 140 compared to placebo. While a subgroup analysis of monotherapy patients showed a hazard ratio of 0.71, no treatment effect was observed in patients receiving stabilizer therapy at baseline. The company and AstraZeneca will share full data set results at the European Society of Cardiology Congress in August 2026. Ionis Pharmaceuticals, Inc. is a biotechnology company that develops RNA-targeted therapeutics.
