Overview
- Ionis and AstraZeneca announced on Thursday, July 9, 2026, that the CARDIO‑TTRansform Phase 3 trial of eplontersen (Wainua) in transthyretin‑mediated amyloid cardiomyopathy did not meet its primary composite endpoint of cardiovascular death plus recurrent cardiovascular events through Week 140.
- A prespecified subgroup of patients receiving eplontersen as monotherapy showed a nominally significant 29% relative risk reduction (hazard ratio 0.71) while the majority of participants already on TTR stabilizer therapy at baseline showed no measurable benefit, a finding sponsors link to the evolving standard of care.
- Secondary analyses, imaging measures and biomarkers strongly favored eplontersen and the drug produced large, sustained reductions in circulating transthyretin with a safety profile consistent with prior studies.
- Markets moved sharply after the announcement with AstraZeneca and Ionis shares plunging and rivals such as Alnylam and BridgeBio rallying as investors reassessed the competitive and commercial landscape for ATTR‑CM therapies.
- The companies will conduct deeper analyses of the full CARDIO‑TTRansform dataset and present detailed results at the European Society of Cardiology Congress in August, a step that will shape regulatory prospects, potential label strategies and access decisions that affect patients, clinicians and payers.