**Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.**
Steen Hvitfeldt Poulsen
Aarhus University
Aarhus, Denmar

**Main Body of the Abstract**
Background
In the ATTRibute-CM trial, acoramidis demonstrated a 25% relative risk reduction (RRR) in all-cause mortality (ACM) in a modified intention-to-treat (mITT) population with eGFR ≥30 ml/min/1.73 m2. Those with eGFR >15 to <30 (stage 4 chronic kidney disease [CKD]) were recruited to explore safety in this high-risk subpopulation; they were excluded from the prespecified, primary mITT efficacy analysis.
Methods
The ITT population included the mITT plus stage 4 CKD population. Baseline characteristics, efficacy, and safety parameters were compared between acoramidis- and placebo-treated participants; prespecified sensitivity analyses were also performed.
Results
The Table shows the baseline characteristics of the mITT and the eGFR <30 populations. Safety was similar across groups, with no safety signals of potential clinical concern. For the ITT population there were 84 ACM events among 421 participants (20%) in the acoramidis group, and 57 events among 211 participants (27%) in the placebo group. The Cox model for ACM in the ITT population yielded a hazard ratio of 0.76 (95% CI 0.54 to 1.07; p=0.12). Two prespecified sensitivity analyses included a stratified log-rank test (p=0.05) and a Cochran-Mantel-Haenszel (CMH) test (p=0.04). The observed RRR with acoramidis in the ITT population was 26%.
Conclusions
ATTRibute-CM is the first ATTR-CM outcomes study to include patients with eGFR <25 ml/min/1.73 m2. Among patients with stage 4 CKD, acoramidis treatment was associated with proportionally fewer deaths versus placebo. In a prespecified sensitivity analysis for the ITT population, acoramidis significantly reduced ACM (CMH test, p=0.04).



**Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.**
Steen Hvitfeldt Poulsen
Aarhus University
Aarhus, Denmar

**Main Body of the Abstract**
**Background**
In the ATTRibute-CM trial, acoramidis demonstrated a 25% relative risk reduction (RRR) in all-cause mortality (ACM) in a modified intention-to-treat (mITT) population with eGFR ≥30 ml/min/1.73 m2. Those with eGFR >15 to <30 (stage 4 chronic kidney disease [CKD]) were recruited to explore safety in this high-risk subpopulation; they were excluded from the prespecified, primary mITT efficacy analysis.
**Methods**
The ITT population included the mITT plus stage 4 CKD population. Baseline characteristics, efficacy, and safety parameters were compared between acoramidis- and placebo-treated participants; prespecified sensitivity analyses were also performed.
**Results**
The Table shows the baseline characteristics of the mITT and the eGFR <30 populations. Safety was similar across groups, with no safety signals of potential clinical concern. For the ITT population there were 84 ACM events among 421 participants (20%) in the acoramidis group, and 57 events among 211 participants (27%) in the placebo group. The Cox model for ACM in the ITT population yielded a hazard ratio of 0.76 (95% CI 0.54 to 1.07; p=0.12). Two prespecified sensitivity analyses included a stratified log-rank test (p=0.05) and a Cochran-Mantel-Haenszel (CMH) test (p=0.04). The observed RRR with acoramidis in the ITT population was 26%.
**Conclusions**
ATTRibute-CM is the first ATTR-CM outcomes study to include patients with eGFR <25 ml/min/1.73 m2. Among patients with stage 4 CKD, acoramidis treatment was associated with proportionally fewer deaths versus placebo. In a prespecified sensitivity analysis for the ITT population, acoramidis significantly reduced ACM (CMH test, p=0.04).



ATTRibute-CM: ITT Sensitivity Analysis and Sub-Analysis Comparing Acoramidis and Placebo in Stage 4 CKD PC126 (#300)

We invite you to attend the XIX International Symposium on Amyloidosis to be held in Rochester, MN.

This symposium is organized as a hybrid meeting (with the focus on in-person) held from Sunday, May 26 through Thursday, May 30, 2024.

Together with the ISA Board and members of Mayo Clinic, a very attractive scientific hybrid program is being planned. The program will cover a variety of types of systemic amyloidosis: ATTR (hereditary and wild-type), AL (light-chain), AA and less frequent forms. There will be key lectures, specific symposia including those company sponsored, oral presentations and poster sessions of accepted abstracts.

COURSE DESCRIPTION
This program will provide a comprehensive review of state-of-the-art education in systemic amyloidosis: basic science, pathophysiology, diagnosis, current and emerging treatments. Scientific presentations will include competitive oral and poster abstracts. The rapid evolution of scientific discovery and expanding treatment options has created tremendous interest in the field and this meeting will fill and important gap in educating scientists and clinicians.