Heart failure & cardiomyopathies
Differential Transthyretin Stabilization in Patients With Wild Type and Variant Transthyretin Amyloidosis
Authors' conclusion
Acoramidis is a near-complete stabilizer of wild type and variant TTR.
Study context
- Population
- Patients with wild type or variant ATTR-CM
- Participants
- 234 vs 34
- Comparison
- Acoramidis vs placebo plus tafamidis
- Acoramidisn = 234
- Placebo plus tafamidisn = 34reference
- Primary endpoint
- TTR stabilizing effect and magnitude of sTTR increase
- Follow-up
- Month 30
Methodological detailsin vitro analysis
- In vitro analysis
Primary result
As reported in the abstract
TTR stabilization
| Group | Western blot | Fluorescent probe exclusion |
|---|---|---|
| Acoramidis | 90.2% [n = 83] | 99.7% [n = 71] |
| Placebo plus tafamidis | 60.6% [n = 6] | 68.0% [n = 4] |
Western blot
Acoramidis90.2% [n = 83]
Placebo plus tafamidis60.6% [n = 6]
Fluorescent probe exclusion
Acoramidis99.7% [n = 71]
Placebo plus tafamidis68.0% [n = 4]
Further findings
In vitro TTR stabilization (acoramidis 10 μM vs tafamidis 16-26 μM)
- Acoramidis more effective TTR stabilizer than tafamidis across all 51 participant samples (17 unique variants)
Original publication
Differential Transthyretin Stabilization in Patients With Wild Type and Variant Transthyretin Amyloidosis. JACC CardioOncol. Published June 2026. doi:10.
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