Heart failure & cardiomyopathies
Eplontersen with and without background transthyretin stabilizers in transthyretin amyloid cardiomyopathy: secondary analysis of a phase 3, randomized controlled trial
Authors' conclusion
Treatment with eplontersen versus placebo demonstrated a clinically meaningful benefit among patients not receiving background stabilizers but provided no additional clinical benefit in those on background stabilizer therapy.
Study context
- Population
- Patients with ATTR-CM57% on TTR stabilizers at baseline
- Participants
- 1,432 patients
- Comparison
- Eplontersen vs placebo, by baseline TTR stabilizer use
- Primary endpoint
- Composite of:
- CV mortality
- Recurrent CV events
- Follow-up
- Up to 140 weeks
Methodological detailsprespecified subgroup analysis, placebo-controlled
- Prespecified subgroup analysis
- Placebo-controlled
Primary result
As reported in the abstract
CV mortality and recurrent CV events, by baseline TTR stabilizer use
Not on stabilizers at baseline: RR, eplontersen vs placebo
0.71
95% CI 0.54 to 0.93, P = 0.012
On stabilizers at baseline: RR, eplontersen vs placebo
1.14
95% CI 0.85 to 1.53, P = 0.39
Further findings
Primary composite endpoint, overall study
- Eplontersen did not reduce the primary composite endpoint
Original publication
Eplontersen with and without background transthyretin stabilizers in transthyretin amyloid cardiomyopathy: secondary analysis of a phase 3, randomized controlled trial. Nat Med. Published 30 August 2026. doi:10.
Summarised from the published abstract; every number is checked against it