Ionis eplontersen Phase 3 ATTR-CM trial misses primary endpoint; monotherapy subgroup shows nominal benefit

IONIS PHARMACEUTICALS INC

• CARDIO-TTRansform trial did not meet primary composite endpoint of CV mortality and recurrent CV events vs placebo at Week 140.
• In prespecified subgroup, eplontersen monotherapy showed nominally significant HR of 0.71 on the composite outcome.
• 57% of patients on stabilizer at baseline; no treatment effect observed in that subgroup.
• Secondary, imaging, and biomarker analyses favored eplontersen; safety profile consistent with prior results.
• Full results to be presented at ESC Congress in August 2026; Ionis and AstraZeneca continue analysis.