Ionis: CARDIO-TTRansform Phase 3 of eplontersen misses primary endpoint vs placebo

Ionis announced the CARDIO-TTRansform Phase 3 trial of eplontersen did not meet its primary composite endpoint of CV mortality and recurrent CV events through Week 140.

Key Highlights:

• Primary endpoint missed: eplontersen showed no statistically significant benefit vs placebo on composite CV mortality and recurrent CV events to Week 140.
• Subgroup signal: prespecified monotherapy subgroup showed a nominal HR 0.71 on the composite outcome vs placebo.
• No effect in stabilizer-treated patients: patients on TTR stabilizer at baseline showed no treatment benefit.
• Secondary and biomarker data favored eplontersen; large sustained TTR reductions observed consistent with silencer class.
• Safety: eplontersen was well tolerated with a favorable safety profile consistent with prior results; full dataset to be presented at ESC 2026.

Original SEC Filing:

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