BridgeBio：acoramidis真实世界研究显示临床恶化风险较tafamidis降低34%

Acoramidis Demonstrates Real-World Benefit Versus Tafamidis in ATTR-CM, Reducing Risk of Clinical Worsening

- In the first peer-reviewed, real-world, contemporary, comparative effectiveness study of TTR stabilizers in ATTR-CM using U.S. claims data, acoramidis was associated with a statistically and clinically significant 34% reduction in a composite of clinical worsening events (diuretic intensification, heart failure hospitalization, and mortality) versus tafamidis (p=0.046), supporting improved clinical stability of acoramidis over tafamidis

- Acoramidis was also associated with a significant 43% reduction in risk of diuretic intensification versus tafamidis (p=0.021); diuretic intensification is an early marker of worsening heart failure and worse clinical outcomes

- Separation in clinical outcomes emerged within weeks of treatment initiation and increased over time, reinforcing a consistent early treatment effect and durable clinical stabilization

- Findings underscore acoramidis’s differentiated profile, with potential to inform frontline treatment decisions and support switching individuals to acoramidis

- An additional, independent real-world study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data is expected to be released at an upcoming 2026 medical meeting

PALO ALTO, Calif., Sept. 23, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage, multi-product biopharmaceutical company focused on developing medicines for genetic conditions, today announced results published in Cardiology and Therapy from the first peer-reviewed, contemporary real-world comparative effectiveness retrospective study of transthyretin (TTR) stabilizers in individuals with transthyretin amyloid cardiomyopathy (ATTR-CM), demonstrating that acoramidis was associated with significantly lower risk of early clinical worsening compared to tafamidis. This represents the first peer-reviewed, real-world evidence differentiating clinical outcomes between approved TTR stabilizers, reinforcing acoramidis’s differentiated clinical profile in present-day practice.

“These findings further position acoramidis as a differentiated TTR stabilizer in contemporary clinical practice,” said Richard Wright, M.D., M.A.C.C. of the Pacific Heart Institute, U.S. “The significant reduction in diuretic intensification, an early indicator of worsening heart failure, points to improved disease control and clinical stability. The progressive nature of ATTR-CM is evident in all Phase 3 trials and is seen again here. This demonstrates the need for clinicians to proactively mitigate disease progression, and these data have the potential to meaningfully inform treatment selection in newly diagnosed patients and in patients currently on other treatments.”

In this analysis of newly treated individuals living with ATTR-CM, acoramidis showed early, consistent, and clinically meaningful advantages compared to tafamidis across key measures of disease progression during a mean follow-up of 4.6 months.

The key findings from the study comparing acoramidis benefit versus tafamidis in individuals with ATTR-CM:

• 34% statistically and clinically significant reduction in risk of composite clinical worsening (HR 0.66; p=0.046), including diuretic intensification, heart failure hospitalization, and all-cause mortality
• 43% reduction in risk of diuretic intensification (HR 0.57; p=0.021), an established early marker of worsening heart failure and predictor of hospitalization and mortality; diuretic intensification was rigorously defined as initiation or dose-equivalent escalation of oral loop diuretics, parenteral loop diuretics use, or addition of a thiazide-type diuretic
• 48% reduction in initiation or dose-equivalent escalation of oral loop diuretics (HR=0.52; p=0.014)
• Rapid benefit, with separation of Kaplan-Meier curves observed within weeks of treatment initiation and increasing over time
• Fewer individuals with acoramidis initiated alternative ATTR-CM therapy as compared to those treated with tafamidis (4.6% vs 7.7%, respectively), consistent with findings of improved clinical stability with acoramidis
• Background heart failure therapies in this study were well balanced across arms and reflect contemporary practice: 43-44% SGLT2i, 36-39% MRA
• Multiple falsification analyses testing for residual bias were nonsignificant
  

The study leveraged a retrospective, longitudinal, new-user, active-comparator design using U.S. claims data and incorporated a unique dataset linking Komodo Healthcare Map U.S. claims with Claritas hub and specialty pharmacy data. After weighting, the analysis included 170 individuals with acoramidis and 448 individuals with tafamidis newly initiating treatment between Dec 2024-April 2025 and followed through July 2025, with a mean follow-up of approximately 4.6 months.

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 real-world benefit of acoramidis versus tafamidis in individuals living with ATTR-CM is planned for fall medical meetings and scientific publications, including an independent real-world study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data, expected to be released at an upcoming 2026 medical meeting.

As with all real-world evidence studies, these findings should be interpreted in the context of inherent limitations of administrative claims data, including limited capture of cardiac biomarkers and other clinical measures of disease severity. Follow-up was limited given the recent approval of acoramidis, and longer-term data are needed to further add to these findings.