AstraZeneca PLCEplontersen missed primary endpoint in Phase 3 ATTR-CM trial.
Ionis Pharmaceuticals and AstraZeneca announced that the Phase 3 CARDIO-TTRansform trial of Eplontersen in adults with transthyretin-mediated amyloid cardiomyopathy did not meet its primary endpoint of cardiovascular mortality and recurrent cardiovascular events through Week 140 compared with placebo. In a prespecified subgroup analysis, patients on Eplontersen monotherapy showed a nominally significant hazard ratio of 0.71, while no treatment effect was seen in those already on stabilizer therapy. Secondary imaging and biomarker analyses favored Eplontersen, with large and sustained reductions in transthyretin levels. The trial enrolled 1,432 patients across 130 sites in 20 countries, making it the largest ATTR-CM trial to date. The companies plan to present full data at the European Society of Cardiology Congress in August 2026.
AstraZeneca PLCEplontersen missed primary endpoint in Phase 3 ATTR-CM trial.
Ionis Pharmaceuticals IncEplontersen missed primary endpoint in Phase 3 ATTR-CM trial.