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Update on CARDIO-TTRansform Phase 3 trial of eplontersen in adults with transthyretin-mediated amyloid cardiomyopathy

$IONSPress releaseJul 9, 2026, 2:00 AM ETRead the release

Ionis Pharmaceuticals and partner AstraZeneca announced that the Phase 3 CARDIO-TTRansform trial for eplontersen in ATTR-CM failed to meet its primary endpoint of reducing cardiovascular mortality and recurrent events. The failure is attributed to the high prevalence of stabilizer therapy in the study population, which masked the drug's efficacy in the overall cohort. However, a pre-specified subgroup analysis showed a nominally significant hazard ratio of 0.71 for patients on eplontersen monotherapy.

Key figures

Drug Name
eplontersen
Phase Of Trial
Phase 3
Trial enrollment
1432
Hazard ratio monotherapy
0.71
Stabilizer usage baseline
57%
Stabilizer usage during trial
24%

AI analysis

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    IONS: Update on CARDIO-TTRansform Phase 3 trial of eplontersen in adults with transthyretin-mediated amyloid cardiomyopathy — AI Analysis | Signal8