Heart failure & cardiomyopathiesSecondary analysis
Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy
- Subgroup analysis of the ATTRibute-CM randomised controlled trial and its open-label extension
- international
- multicenter
- phase 3
- placebo-controlled
- post-hoc analyses in variant subgroups
- April 2019 to May 2023 with ongoing open-label extension (OLE) (month 42)
Authors' conclusion
The beneficial effect of acoramidis was observed consistently in ATTRwt-CM and ATTRv-CM groups.
Study context
- Population
- Participants with ATTR-CM, wild-type (ATTRwt-CM) or variant (ATTRv-CM), including p.Val142Ile611 modified intention-to-treat (mITT)
- Participants
- 632 participants
- Comparison
- Acoramidis vs placebo
- Primary endpoint
- ACM/first CVH
- Follow-up
- Month 30
Primary result
As reported in the abstract
ACM/first CVH by genotype subgroup
Acoramidis vs PlaceboHazard ratio (95% CI)P value
ATTRwt-CM (n = 552)
0.69(0.52–0.90)
P = 0.007ATTRv-CM (n = 59)
0.41(0.21–0.81)
P = 0.01Secondary outcomes
| Outcome | HR (95% CI) | P value | |
|---|---|---|---|
| ACM by genotype subgroup · month 42 — Acoramidis vs Placebo · ATTRwt-CM | 0.70 (0.50–0.98) | P = 0.04 | |
| ACM by genotype subgroup · month 42 — Acoramidis vs Placebo · ATTRv-CM | 0.41 (0.19–0.93) | P = 0.03 | |
| 0.20.5125Hazard ratio (log scale) |
Secondary end points in ATTRwt-CM and ATTRv-CM
- Consistent treatment benefit
All-cause mortality (ACM)/CVH (month 30) and ACM (month 42), p.Val142Ile vs non-p.Val142Ile
- Consistent treatment benefits within variant subgroups
- post-hoc
- 35 with p.Val142Ile
Original publication
Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy. JAMA Cardiol. Published 1 January 2026. doi:10.1001/jamacardio.2025.4477
Summarised from the published abstract; every number is checked against it