BridgeBio公布acoramidis在ATTR-CM长期随访和CMR亚组中的疾病逆转信号

- Acoramidis is the first therapy shown to potentially reverse cardiac structural disease progression and functional decline through 42 months based on CMR imaging,
with up to half of patients showing clinically meaningful improvement in cardiac function in the completer analysis
- Patients treated with acoramidis were observed to have an unprecedented 65 additional days alive and out of the hospital by Month 36 versus baseline placebo patients
- Acoramidis demonstrated long-term efficacy and safety through 54 months across variant ATTR-CM subgroups, including p.Val142Ile and non-p.Val142Ile. These findings were simultaneously published in the
European Journal of Heart Failure
PALO ALTO, Calif., Aug. 30, 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, presented new analyses from the Phase 3 ATTRibute-CM study of Attruby
®
(acoramidis) in transthyretin amyloid cardiomyopathy (ATTR-CM), including the cardiac magnetic resonance imaging (CMR) substudy and the open-label extension (OLE) at the European Society of Cardiology (ESC) Congress 2026. Acoramidis is the only selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer.
“The clinical community is excited about the potential to restore heart health found in these data. For a long time, patients living with ATTR-CM could only hope for a stop to the otherwise relentless progression of disease. These new CMR data from ATTRibute-CM shows evidence of reversal in a meaningful proportion of individuals treated with acoramidis, with roughly half showing improved left ventricular systolic function in the completer analysis, more than 2x the proportion observed in the natural history from a NAC cohort or in ATTRibute-CM participants treated with placebo. These findings support acoramidis as a therapy capable of altering the trajectory of this otherwise progressive disease,” said Marianna Fontana, M.D. of University College London, UK. “For patients and clinicians navigating ATTR-CM, this is an exciting signal that the treatment paradigm is shifting toward a therapy that could actively restore heart health rather than only manage decline.”
The CMR substudy of ATTRibute-CM and its open-label extension provide the first evidence from serial CMR that a therapy can potentially reverse disease progression through Month 42. The findings presented by Awais Sheikh, MBChB of the National Amyloidosis Centre, London, UK were evaluated using two complementary analytical approaches, which found:
In a completer analysis, clinically meaningful improvement from baseline in left ventricular (LV) systolic function was observed in 54% of acoramidis-treated patients versus 20% of placebo-treated patients at Month 30, and in 53% of continuous-acoramidis patients at Month 42
For context, only 26% of completers in an independent natural history cohort demonstrated improved LV systolic function by Month 24 – approximately half the rate observed with acoramidis, suggesting that this magnitude of improvement falls outside the expected natural course of disease
In a conservative analysis, long-term acoramidis treatment was associated with clinically meaningful improvement from baseline in LV systolic function in approximately one-third of patients over 30-42 months. Improvement was observed in 34% of acoramidis-treated patients versus 9% of placebo-treated patients at Month 30 and in 30% of continuous-acoramidis patients at Month 42
In addition, 46% of patients receiving continuous acoramidis demonstrated improvement from baseline in LV mass index at Month 42, providing evidence of favorable structural remodeling
These results provided sufficient evidence for BridgeBio to recently dose its first participant in ASCEND-ATTR, a Phase 3b/4 study designed to determine if acoramidis is associated with sustained improvement in myocardial structural disease progression, function and amyloid burden
In a post-hoc analysis of ATTRibute-CM presented by Richard Wright, M.D. of the Pacific Heart Institute, U.S., acoramidis preserved significantly more time alive outside the hospital for patients with ATTR-CM. The analysis evaluated days lost to death and/or cardiovascular-related hospitalization (DLDCVH), a patient-centered measure that integrates all-cause mortality, cardiovascular-related hospitalizations, and length of stay into a single assessment of disease burden. Key findings included:
In participants with ATTR-CM, acoramidis reduced the estimated mean percentage of DLDCVH to 7.5% versus 11.7% with placebo through Month 30
Acoramidis preserved more than one month of additional time alive and out of the hospital (38 days) over 30 months with the benefit nearly doubling to 65 days (observed) over three years, and nearly tripling to up to 94 days (modelled estimates) over three years, reflecting progressive divergence in outcomes over time
The p.Val142Ile genetic variant is the most common ATTR-CM genetic variant globally, disproportionately affecting individuals of Western African ancestry, with a carrier frequency of 3-4% in the U.S. Black population. Findings in the ATTRibute-CM OLE presented by Kevin Alexander, M.D. of Stanford University School of Medicine, U.S. showed continued benefit of acoramidis in 56 variant ATTR-CM (ATTRv-CM) patients, including 35 p.Val142Ile and 21 non-p.Val142Ile patients through Month 54, demonstrating:
All-cause mortality (ACM) and cardiovascular mortality (CVM) were markedly lower in the continuous acoramidis arm versus placebo-to-acoramidis across both p.Val142Ile and non-p.Val142Ile variant subgroups
Through Month 54, ACM was 30.4% with continuous acoramidis versus 66.7% with placebo-to-acoramidis in the p.Val142Ile subgroup, and 24.3% with continuous acoramidis versus 57.9% with placebo-to-acoramidis across the overall ATTRv-CM population, a consistent, more than two-fold difference in mortality favoring continuous treatment
The ACM and CVM rates at Month 54 were notably high (~65%) in the p.Val142Ile group who were randomized to placebo in ATTRibute-CM, underscoring the substantial unmet medical need in this high-risk subgroup
Continuous acoramidis achieved sustained increases in serum TTR (sTTR) and persistent attenuation of N-terminal pro-B-type natriuretic peptide (NT-proBNP) rise through Month 54 in both participants with p.Val142Ile or non-p.Val142Ile variants
These Month 54 findings extend the survival benefit and favorable biomarker trends previously reported at Month 30, demonstrating the long-term durability of efficacy and safety of acoramidis in ATTRv-CM, including in the p.Val142Ile subgroup
Acoramidis remained well tolerated through Month 54, with no new safety signals observed in the OLE
In addition to the one oral presentation and two moderated posters highlighted, two additional moderated posters on acoramidis were shared at the ESC Congress 2026, including:
Acoramidis Improves Health-Related Quality of Life in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: An EQ-5D-5L Subgroup Analysis from ATTRibute-CM
, presented by Emer Joyce, M.D., Ph.D. of The Mater Misericordiae University Hospital, IE
Treatment with acoramidis resulted in significant and clinically meaningful benefits in health-related quality of life (HRQoL) in both wild-type ATTR-CM (ATTRwt-CM) and ATTRv-CM. Greater impact on HRQoL versus placebo was observed in participants with ATTRv-CM
Improvement of Health Status with Acoramidis in Patients with Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: KCCQ Domains Analysis from the ATTRibute-CM
Study, presented by Nitasha Sarswat, M.D. of University of Chicago Medical Center, U.S.
In ATTRibute-CM, acoramidis attenuated the decline in heart failure-related health status versus placebo in participants with ATTRwt-CM and ATTRv-CM, with consistent benefits observed across Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) and individual domain scores. A numerical improvement was observed across almost all KCCQ domains in acoramidis-treated participants with ATTRwt-CM and ATTRv-CM relative to placebo
As part of BridgeBio's partnership with Yale's Cardiovascular Data Science (CarDS) Lab to advance AI networks for earlier detection of ATTR-CM, three posters were presented at the ESC Congress 2026. Findings from the partnership included:
A Novel AI-Derived Digital Biomarker for Monitoring Disease Progression in ATTR-CM: First-In-Trial Use of a Computer Vision AI-ECG Algorithm within a Phase 3 Pivotal Randomized Controlled Trial,
presented by Rohan Khera, M.D. of Yale School of Medicine, U.S.
This showed the first deployment of a computer vision AI-ECG algorithm, operating directly on ECG data, as a digital biomarker in a RCT (ATTRibute-CM). An image-based AI-ECG algorithm demonstrated discrimination across clinical subgroups at baseline and detected differential longitudinal changes between acoramidis and placebo over 30 months. These findings support the potential role of AI-ECG derived prediction scores as a scalable digital biomarker in clinical trials and potential routine cardiovascular care
A Fully Decentralized, Patient-Led Digital Registry for ATTR-CM Integrating Multisystem EHR and Wearable Data: The DISCOVER-ATTR Study
, presented by Aline Pedroso, Ph.D. of Yale School of Medicine
A fully decentralized, patient-led digital registry can successfully aggregate longitudinal multisystem electronic health records (EHR) data and wearable physiologic signals in ATTR-CM. Early results show substantial data yield and feasibility of longitudinal mapping of care trajectories and multimodal risk prediction, providing a blueprint for next-generation registries in rare cardiovascular diseases
Nationwide U.S. Federated Deployment of Artificial Intelligence for Multimodal Screening of ATTR Cardiomyopathy: First Multicenter Analysis from the TRACE-AI Network
, presented by Bruno Batinica, MBChB of Yale School of Medicine
In this largest-ever deployment of AI-electrocardiogram and AI-Echo models for opportunistic retrospective screening of individuals at risk of ATTR-CM, we demonstrate a large burden of probable undiagnosed ATTR-CM with prognostic implications. Leveraging this framework for screening holds promise for enabling broad, timely identification of patients to maximize the overall benefit of new therapies
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, including Heart Failure Society of America (HFSA) Annual Scientific Meeting 2026, taking place in Phoenix, Arizona on October 9-12, 2026.
About
Attruby
®
(acoramidis)
INDICATION
Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization.
IMPORTANT SAFETY INFORMATION
Adverse Reactions
Diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively).
BridgeBio Forward-Looking Statements
This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements include statements regarding the potential clinical significance and therapeutic implications of the data presented regarding acoramidis, including the potential clinical and therapeutic implications of observed changes in cardiac structure and function and the potential for acoramidis to alter the trajectory of ATTR-CM and restore heart health; the potential utility of AI-based tools and digital biomarkers for the detection, monitoring and screening of ATTR-CM in clinical trials and clinical practice; and BridgeBio’s plans to present additional data regarding Attruby at future medical meetings. Although the Company believes that its plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, the Company can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from the Company’s clinical trials not being indicative of final data, the design and success of ongoing and planned clinical trials, the risk that results from post hoc analyses, subgroup analyses or other analyses may not be predictive of future clinical outcomes or treatment effects, that observed improvements in cardiac structure, function or other measures may not be replicated in additional analyses or studies or translate into improved long-term clinical outcomes, that the potential utility of AI-based tools and digital biomarkers may not be demonstrated in further studies or translate into routine clinical use, that plans to present additional data may change, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Middle East, increasing rates of inflation and changing interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of the Company’s most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K and the Company’s other filings with the U.S. Securities and Exchange Commission. Moreover, the Company operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of the Company’s management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, BridgeBio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
About BridgeBio
BridgeBio exists to develop transformative medicines for genetic conditions. Millions of people worldwide living with genetic conditions lack treatment options, often because drug development for small patient populations can be commercially challenging. We aim to bridge the gap between advancements in genetic science and meaningful medicines for underserved patient populations. Our decentralized, hub-and-spoke model is designed for speed, precision, and scalability. Autonomous and empowered teams focus on individual conditions, while a central hub provides the clinical, regulatory, and commercial capabilities needed to bring innovation to market. For more information, visit
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