BridgeBio Pharma reports significant long-term efficacy of acoramidis in reducing mortality and improving quality of life for ATTR-CM patients.
Quiver AI Summary
BridgeBio Pharma, Inc. announced promising long-term results from the ATTRibute-CM open-label extension trial for acoramidis, a treatment for transthyretin amyloid cardiomyopathy (ATTR-CM), during the American College of Cardiology Annual Scientific Sessions. The findings demonstrated a significant 44.7% reduction in all-cause mortality and a 49.3% reduction in cardiovascular mortality after 54 months of treatment. Acoramidis also effectively mitigated NT-proBNP levels and maintained heart failure-related quality of life scores, indicating sustained clinical benefits for patients. The treatment was well-tolerated with no long-term safety issues. These results underscore the importance of early diagnosis and intervention in the management of ATTR-CM.
Potential Positives
- Significant risk reductions of 44.7% in all-cause mortality and 49.3% in cardiovascular mortality for acoramidis at the earliest timepoint in an open-label extension, indicating strong efficacy.
- Acoramidis demonstrated the ability to mitigate NT-proBNP rise through Month 54, an important marker in heart failure treatment.
- The treatment sustained improvements in quality of life scores for patients with ATTR-CM, highlighting its long-term benefit and effectiveness.
- Acoramidis maintains a favorable safety profile with no long-term safety concerns reported, which is crucial for patient compliance and trust in treatment.
Potential Negatives
- There are reported adverse reactions, such as diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%), associated with acoramidis, which could impact patient compliance and overall perception of the drug.
- The press release acknowledges that many patients with ATTR-CM still suffer from progressive heart failure despite therapeutic advances, indicating a potential limitation of acoramidis in addressing these patients' needs fully.
- There may be a disconnect in treatment satisfaction among physicians and patients, as a significant portion of physicians expressed dissatisfaction with current treatment options and a notable percentage of patients did not receive treatment at all.
FAQ
What are the key findings from the ATTRibute-CM trial?
The trial showed 44.7% reduction in all-cause mortality and 49.3% in cardiovascular mortality with acoramidis.
How does acoramidis impact heart failure-related quality of life?
Acoramidis treatment stabilized and maintained heart failure-related quality of life scores throughout 54 months.
What is the safety profile of acoramidis?
Acoramidis has shown a favorable long-term safety profile with few reported adverse reactions.
What is acoramidis approved for?
Acoramidis is approved for treating cardiomyopathy of transthyretin-mediated amyloidosis in adults.
Where were the trial results presented?
The results were presented at the American College of Cardiology Annual Scientific Sessions and published in JAMA Cardiology.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
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Full Release
- Earliest timepoint in an open-label extension with this magnitude of risk reduction at 44.7% in ACM (p<0.0001) and 49.3% in CVM (p<0.0001)
- Acoramidis mitigated the rise in NT-proBNP through Month 54 to an extent not seen in the era of disease modifying treatments
- Early and continuous acoramidis treatment stabilized and maintained all measures of heart failure-related QOL scores (KCCQ-OS), which demonstrates that the improvement in duration and quality of life in patients with ATTR-CM by acoramidis is sustained
PALO ALTO, Calif., March 30, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a biopharmaceutical company focused on developing medicines for genetic conditions, today presented long-term efficacy and safety data from the ATTRibute-CM open-label extension (OLE) trial, demonstrating sustained clinical benefit of acoramidis through Month 54 in patients with ATTR-CM. These data were presented at the American College of Cardiology (ACC) Annual Scientific Sessions & Expo in a late-breaking oral presentation by Prem Soman, M.D., Ph.D. of University of Pittsburgh School of Medicine and it was the only ATTR-CM presentation selected for the ACC late-breaker session. These data are now simultaneously published in JAMA Cardiology . Acoramidis is the only selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer.
“Despite dramatic therapeutic advances in the field, many patients with ATTR-CM still continue to suffer progressive heart failure, high mortality risk, and many have limited long-term treatment options,” said Dr. Soman. “The ATTRibute-CM long-term data show that early and continuous treatment with acoramidis can meaningfully change the trajectory of this disease, with sustained reductions in all-cause and cardiovascular mortality, cardiovascular hospitalization, continued mitigation of NT-proBNP progression, and a favorable long-term safety profile. These findings reinforce the importance of early diagnosis followed by prompt, durable treatment to deliver sustained clinical benefit for patients.”
The findings from the OLE trial included:
- At Month 54, continuous acoramidis treatment led to a statistically significant risk reduction of 44.7% in all-cause mortality (ACM) (p<0.0001) and 49.3% in c ardiovascular mortality (CVM) (p<0.0001) versus placebo-to-acoramidis, which is the earliest timepoint in an open-label extension with this magnitude of risk reduction in these events
- Through Month 54, acoramidis mitigated the rise in NT-proBNP versus placebo-to-acoramidis to an extent not seen in the era of disease modifying treatments
- Early and continuous acoramidis treatment stabilized and maintained all measures of heart failure-related QOL scores (KCCQ-OS), which demonstrates that the improvement in duration and quality of life in patients with ATTR-CM by acoramidis is sustained
- Acoramidis continued to be well tolerated with no long-term safety concerns
Three acoramidis posters were also shared at the ACC Annual Scientific Sessions & Expo, which included:
-
Long-Term Benefits With Acoramidis on Serum Transthyretin Concentrations and Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Score in Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM),
presented by Sarah Cuddy, M.D. of Brigham and Women's Hospital
- This analysis showed that early and continuous acoramidis treatment stabilized heart failure-related health status and maintained serum transthyretin (sTTR) levels through Month 54. These findings emphasize the need for early diagnosis followed by early treatment in patients with ATTR-CM
-
Association Between Early Increase in sTTR with Acoramidis and Long-Term Effects on Heart Failure – Related Health Status in ATTR-CM: Results from ATTRibute-CM,
presented by Jan Griffin, M.D. of Medical University of South Carolina
- This analysis showed that an early increase in sTTR at Day 28 with acoramidis was associated with clinically meaningful slower decline in KCCQ-OS score at Month 30 versus placebo in patients with ATTR-CM in ATTRibute-CM
-
T
reatment Patterns and Preferences in ATTR-CM in the United States: Results from a Real-World Survey,
presented by Jill Waldron, MSN, GNP, MS of University of Utah
- This analysis showed that in a real-world survey, physicians were not fully satisfied with treatment for more than half of those prescribed, and more than one-third of patients did not receive treatment at all. Patients were most comfortable with the prospect of oral therapy. These data highlight unmet treatment needs for ATTR-CM patients and importance of shared decision making for therapy selection
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, and the UK Medicines and Healthcare Products Regulatory Agency with all labels specifying near-complete stabilization of TTR.
More data on the benefit of Attruby for ATTR-CM patients is planned for future medical meetings.
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).
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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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 and commercial benefits of acoramidis; acoramidis’ observed impact on reductions in all-cause and cardiovascular mortality and cardiovascular hospitalization; acoramidis’ mitigation of NT-proBNP progression; the durability of improvements in heart failure-related QOL scores; the long-term safety and tolerability profile of acoramidis; and BridgeBio’s ongoing and future development programs.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, future regulatory filings, approvals and/or sales, the FDA or such other regulatory agencies not agreeing with the Company’s regulatory approval strategies, components of the Company’s filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, 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 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.
BridgeBio Media Contact:
Bubba Murarka, Executive Vice President, Corporate Development
[email protected]
(650)-789-8220
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Chinmay Shukla, Senior Vice President, Strategic Finance
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