Ionis eplontersen Phase 3 trial misses primary endpoint
Ionis Pharmaceuticals announced that its CARDIO-TTRansform Phase 3 trial for eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet its primary efficacy endpoint. The trial failed to show a statistically significant benefit in the composite outcome of cardiovascular mortality and recurrent cardiovascular clinical events compared to placebo. While a prespecified subgroup analysis of eplontersen monotherapy showed a nominally significant hazard ratio of 0.71, and multiple secondary analyses favored the drug, the overall primary goal was not achieved. Ionis and AstraZeneca will continue to analyze the data and present results at the European Society of Cardiology Congress in August 2026.
Key Highlights
- CARDIO-TTRansform Phase 3 trial for eplontersen in ATTR-CM missed primary efficacy endpoint.
- Primary endpoint was composite of CV mortality and recurrent CV clinical events up to Week 140.
- Adding eplontersen to standard of care did not provide statistically significant benefit.
- Prespecified subgroup analysis of monotherapy showed nominally significant hazard ratio of 0.71.
- Multiple secondary, imaging, and biomarker analyses favored eplontersen versus placebo.
- Eplontersen was well tolerated with a favorable safety profile.
- Full data will be shared at the European Society of Cardiology Congress in August 2026.
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