New data demonstrating disease modifying effects across clinical outcomes, biomarkers, and functional capacity. Acoramidis is the only selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer.
In a late-breaking oral presentation shared by Senthil Selvaraj, M.D. of Duke University School of Medicine, U.S., acoramidis showed an association between the treatment-related increase in serum transthyretin (sTTR) and a significant reduction of sTTR variability over time, which is independently associated with reduced all-cause mortality (ACM). Findings included:
- Lower intraindividual sTTR variability and higher achieved sTTR levels were each independently associated with reduced risk of all-cause mortality (HR: 0.56; p=0.014 and HR: 0.46; p=0.014, respectively)
- Participants with both higher achieved sTTR levels and less variable sTTR levels experienced the greatest survival benefit, while higher sTTR variability was associated with adverse clinical features of ATTR-CM
- Acoramidis increased sTTR levels early (Day 28) and sustained them through Month 30, while significantly reducing sTTR variability versus placebo (9.5% vs. 12.8%; p<0.001)
- Reduction in sTTR variability mediated 20% of acoramidis' treatment effect on mortality
"While increasing TTR levels on stabilizer therapy is important and strongly relates to risk of dying in ATTR-CM, these new results demonstrate that reducing TTR variability at the individual level over time also seems to be important for modifying disease outcomes. Acoramidis not only rapidly increases TTR levels, but significantly decreased this variability compared with placebo. By linking TTR variability independently to mortality, we're seeing a mechanistic signal that may help explain acoramidis' clinical benefit, providing complementary data to support its use in ATTR-CM," said Dr. Selvaraj
Additionally, Bayer, BridgeBio's exclusive European licensing partner of acoramidis, presented a late-breaking oral presentation by Marianna Fontana, M.D. of University College London, UK, showing that acoramidis drove early and sustained reductions in the risk of outpatient worsening heart failure within 30 days. These findings included:
- Acoramidis reduced the risk of outpatient worsening heart failure versus placebo by 41% (HR 0.59, 95% CI 0.46–0.75; p < 0.0001, with K-M curve separation seen within 30 days and sustained through Month 30)
- Outpatient worsening heart failure was a strong predictor of mortality and cardiovascular hospitalizations, reinforcing its role as an early and clinically meaningful marker of disease progression
- Even after adjusting for outpatient worsening heart failure, acoramidis reduced risk of mortality and recurrent cardiovascular hospitalizations by 41% (HR 0.59, 95% CI 0.45–0.77; p = 0.0001)
For the first time, BridgeBio also shared an anchored matching-adjusted indirect comparison presented by Emer Joyce, M.D., Ph.D. of the Mater Misericordiae University Hospital, IE, of acoramidis versus tafamidis using the pivotal study data with statistically significant, clinically meaningful benefit, which showed that:
- Acoramidis demonstrated a statistically significant, 34% reduction in cardiovascular hospitalizations versus tafamidis (RRR: 0.66; 95% CI: 0.46–0.95)
- Acoramidis showed a favorable mortality trend with 28% hazard reduction in all-cause mortality versus tafamidis, with consistent benefit across sensitivity analyses
- Comparable safety profile observed between acoramidis and tafamidis
In addition to the presentations highlighted, three additional acoramidis posters were also shared at Heart Failure 2026, which included:
- Acoramidis Treatment Attenuates the Rise in NT-proBNP from Baseline to Month 30 Compared to Placebo Across all Subgroups, presented by Marianna Fontana, M.D., of University College London, UK
- In ATTRibute-CM, acoramidis consistently blunted the 30-month increase in NT-proBNP by about 50% across all participant subgroups assessed, including advanced disease, compared with placebo, demonstrating its robust efficacy on a key biomarker of ATTR-CM disease progression
- Consistent Benefit on Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OS) with Acoramidis Treatment Compared with Placebo Across Participant Subgroups in ATTRibute-CM, presented by Dr. Fontana
- Acoramidis significantly attenuated the decline in heart failure-related health status compared with placebo in individuals with ATTR-CM. This effect was observed consistently across all pre-specified participant subgroups, including those with advanced heart failure symptoms
- Effect of Acoramidis on Improvement or Maintenance of Heart Failure-Related Health Status as Assessed by KCCQ-OS Score in ATTRibute-CM, presented by Charles Sherrod, M.D. of Saint Luke's Health System, Kansas City, U.S.
- In individuals with ATTR-CM, acoramidis treatment was associated with a significantly greater likelihood of maintenance or improvement in health status compared with placebo, suggesting a clinically relevant modification of disease trajectory. These data provide an integrated assessment of health status and survival to inform medical decision making
Acoramidis is approved as Attruby® by the U.S. FDA and is approved as BEYONTTRA® by the European Medicines Agency (EMA), Japanese Pharmaceuticals and Medical Devices Agency, Swissmedic, the Swiss Agency for Therapeutic Products, the UK Medicines and Healthcare Products Regulatory Agency, and the Brazilian Health Regulatory Agency (ANVISA) with all labels specifying near-complete stabilization of TTR.
Additional data on the benefit of Attruby for individuals with ATTR-CM is planned for future medical meetings.
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