Acoramidis Shows Statistically Significant Improvements in Cardiovascular Outcomes in Patients with Variant ATTR-CM
BridgeBio Pharma (BBIO) announced significant clinical outcomes for acoramidis in treating transthyretin amyloid cardiomyopathy (ATTR-CM). The drug showed a 59% risk reduction in variant ATTR-CM patients and 31.2% reduction in wild-type ATTR-CM patients for the composite of all-cause mortality (ACM) and cardiovascular-related hospitalization (CVH).
Key highlights from the ATTRibute-CM Phase 3 trial include:
- Hazard ratio of 0.41 for ATTRv-CM patients
- 42% reduction in composite ACM and recurrent CVH events at Month 30
- 50% reduction in cumulative CVH events at Month 30
- Benefits observed in as few as 3 months
Acoramidis, approved as Attruby™ in the US and BEYONTTRA™ in Europe and Japan, demonstrates near-complete (≥90%) TTR stabilization. The drug showed improved outcomes in both variant and wild-type ATTR-CM patients, with statistically significant results in both subgroups.
BridgeBio Pharma (BBIO) ha annunciato risultati clinici significativi per acoramidis nel trattamento della cardiomiopatia amiloide da transtiretina (ATTR-CM). Il farmaco ha mostrato una riduzione del rischio del 59% nei pazienti con ATTR-CM variante e una riduzione del 31,2% nei pazienti con ATTR-CM di tipo wild-type per il composito di mortalità per tutte le cause (ACM) e ospedalizzazione per cause cardiovascolari (CVH).
I punti salienti dello studio di fase 3 ATTRibute-CM includono:
- Rapporto di rischio di 0,41 per i pazienti con ATTRv-CM
- Riduzione del 42% nel composito di eventi ACM e CVH ricorrenti al mese 30
- Riduzione del 50% negli eventi CVH cumulativi al mese 30
- Benefici osservati in appena 3 mesi
Acoramidis, approvato come Attruby™ negli Stati Uniti e BEYONTTRA™ in Europa e Giappone, dimostra una stabilizzazione quasi completa (≥90%) della TTR. Il farmaco ha mostrato risultati migliorati sia nei pazienti con ATTR-CM variante che in quelli di tipo wild-type, con risultati statisticamente significativi in entrambi i sottogruppi.
BridgeBio Pharma (BBIO) anunció resultados clínicos significativos para acoramidis en el tratamiento de la cardiomiopatía amiloide por transtiretina (ATTR-CM). El fármaco mostró una reducción del riesgo del 59% en pacientes con ATTR-CM variante y una reducción del 31,2% en pacientes con ATTR-CM de tipo salvaje para el compuesto de mortalidad por todas las causas (ACM) y hospitalización relacionada con enfermedades cardiovasculares (CVH).
Los puntos destacados del ensayo de fase 3 ATTRibute-CM incluyen:
- Razón de riesgo de 0,41 para pacientes con ATTRv-CM
- Reducción del 42% en el compuesto de eventos ACM y CVH recurrentes al mes 30
- Reducción del 50% en eventos CVH acumulativos al mes 30
- Beneficios observados en tan solo 3 meses
Acoramidis, aprobado como Attruby™ en EE. UU. y BEYONTTRA™ en Europa y Japón, demuestra una estabilización casi completa (≥90%) de TTR. El fármaco mostró resultados mejorados tanto en pacientes con ATTR-CM variante como en aquellos de tipo salvaje, con resultados estadísticamente significativos en ambos subgrupos.
BridgeBio Pharma (BBIO)는 트랜스티레틴 아밀로이드 심근병증(ATTR-CM) 치료를 위한 아코라미디스의 중요한 임상 결과를 발표했습니다. 이 약물은 변이 ATTR-CM 환자에서 59%의 위험 감소를, 야생형 ATTR-CM 환자에서 31.2% 감소를 보여주었으며, 이는 모든 원인에 의한 사망률(ACM) 및 심혈관 관련 입원(CVH)의 복합 결과입니다.
ATTRibute-CM 3상 시험의 주요 하이라이트는 다음과 같습니다:
- ATTRv-CM 환자에 대한 위험비 0.41
- 30개월 차에 ACM과 재발 CVH 사건의 복합에서 42% 감소
- 30개월 차에 누적 CVH 사건에서 50% 감소
- 단 3개월 만에 관찰된 혜택
아코라미디스는 미국에서 Attruby™로, 유럽과 일본에서 BEYONTTRA™로 승인되었으며, TTR의 거의 완전한(≥90%) 안정화를 보여줍니다. 이 약물은 변이형 및 야생형 ATTR-CM 환자 모두에서 개선된 결과를 나타냈으며, 두 하위 그룹 모두에서 통계적으로 유의미한 결과를 보였습니다.
BridgeBio Pharma (BBIO) a annoncé des résultats cliniques significatifs pour l'acoramidis dans le traitement de la cardiomyopathie amyloïde à transthyretine (ATTR-CM). Le médicament a montré une réduction du risque de 59% chez les patients atteints d'ATTR-CM variante et une réduction de 31,2% chez les patients atteints d'ATTR-CM de type sauvage pour le composite de mortalité toutes causes confondues (ACM) et d'hospitalisations liées aux maladies cardiovasculaires (CVH).
Les points clés de l'essai de phase 3 ATTRibute-CM comprennent:
- Ratio de risque de 0,41 pour les patients atteints d'ATTRv-CM
- Réduction de 42% dans le composite d'événements ACM et CVH récurrents au mois 30
- Réduction de 50% des événements CVH cumulés au mois 30
- Avantages observés en aussi peu que 3 mois
L'acoramidis, approuvé sous le nom d'Attruby™ aux États-Unis et de BEYONTTRA™ en Europe et au Japon, montre une stabilisation presque complète (≥90%) de la TTR. Le médicament a montré des résultats améliorés tant chez les patients atteints d'ATTR-CM variante que chez ceux de type sauvage, avec des résultats statistiquement significatifs dans les deux sous-groupes.
BridgeBio Pharma (BBIO) hat bedeutende klinische Ergebnisse für Acoramidis bei der Behandlung der transthyretin-amyloiden Kardiomyopathie (ATTR-CM) bekannt gegeben. Das Medikament zeigte eine 59%ige Risikominderung bei Patienten mit ATTR-CM-Varianten und eine 31,2%ige Reduktion bei Patienten mit wildtypischer ATTR-CM für die Kombination aus Gesamtmortalität (ACM) und kardiovaskulärer Hospitalisierung (CVH).
Wichtige Highlights der ATTRibute-CM Phase-3-Studie umfassen:
- Hazard Ratio von 0,41 für ATTRv-CM-Patienten
- 42%ige Reduktion bei der Kombination von ACM und wiederkehrenden CVH-Ereignissen nach 30 Monaten
- 50%ige Reduktion der kumulierten CVH-Ereignisse nach 30 Monaten
- Vorteile bereits nach 3 Monaten beobachtet
Acoramidis, in den USA als Attruby™ und in Europa und Japan als BEYONTTRA™ zugelassen, zeigt eine nahezu vollständige (≥90%) TTR-Stabilisierung. Das Medikament zeigte verbesserte Ergebnisse sowohl bei Patienten mit ATTR-CM-Varianten als auch bei wildtypischen ATTR-CM-Patienten, mit statistisch signifikanten Ergebnissen in beiden Untergruppen.
- Statistically significant 59.1% risk reduction in variant ATTR-CM patients
- 42% reduction in composite ACM and recurrent CVH events at 30 months
- 50% reduction in cardiovascular hospitalization events at 30 months
- Rapid efficacy showing benefits within 3 months
- Regulatory approvals secured in major markets (US, EU, Japan)
- None.
Insights
BridgeBio's acoramidis data represents a significant clinical and commercial breakthrough in ATTR-CM treatment. The 59% hazard reduction (HR 0.41) in variant ATTR-CM patients for the composite primary endpoint is remarkably strong efficacy data that exceeds previous treatments in this indication. This positions acoramidis (marketed as Attruby/BEYONTTRA) as potentially best-in-class.
The rapid onset of efficacy (separation from placebo at just 3 months) is commercially advantageous, as physicians tend to favor treatments with earlier observable benefits. The 42% reduction in composite all-cause mortality/recurrent hospitalizations and 50% reduction in hospitalization frequency represent compelling real-world endpoints that payers value.
Already approved across major markets (US, EU, Japan), these new subgroup analyses showing statistically significant benefits in both variant and wild-type populations expand the addressable patient population. The mechanistic data demonstrating near-complete TTR stabilization (≥90%) provides a compelling scientific rationale for physicians to adopt the therapy.
Particularly noteworthy is the data showing no additional TTR stabilization when tafamidis (the current market leader) is added to acoramidis, suggesting potential for acoramidis to capture substantial market share from competitors. The robust results across multiple sensitivity analyses further strengthen the clinical package and should translate to strong market uptake.
The ATTRibute-CM trial results for acoramidis represent a potential paradigm shift in ATTR-CM treatment, particularly for variant ATTR-CM patients who face a more aggressive disease course and poorer prognosis. The HR of 0.41 for the primary endpoint translates to a 59% risk reduction - the largest efficacy signal observed to date in this challenging subpopulation.
From a clinical perspective, the improvement in NYHA class is especially meaningful. This functional classification directly correlates with patient symptoms and quality of life. Stabilization or improvement in NYHA class indicates that acoramidis is not merely affecting biomarkers but providing tangible benefits to patients' daily functioning.
The mechanism of near-complete TTR stabilization (≥90%) addresses the fundamental pathophysiology of ATTR-CM. By effectively stabilizing TTR tetramers, including the less stable variant forms, acoramidis prevents amyloid formation and deposition in cardiac tissue. The correlation between increased serum TTR levels and reduced cardiovascular events validates this approach.
The demonstrated benefits in reducing hospitalizations (50% reduction) are particularly valuable given the high burden of recurrent hospitalizations in ATTR-CM. The data suggesting early efficacy (within 3 months) is clinically significant as it allows for timely assessment of treatment response in a progressive disease where intervention windows are
- Acoramidis’s hazard ratio of .41 for time to ACM or first CVH versus placebo in the ATTRibute-CM study subgroup of ATTRv-CM patients achieved statistical significance in a pre-specified analysis
- This profound treatment effect, due to the near-complete stabilization (≥
90% ) and binding of TTR, represents the greatest observed benefit to date for ATTRv-CM patients, an ATTR-CM population with poor prognosis
- In the ATTRibute-CM study, acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date in both ATTRv-CM and ATTRw-CM patients:
- In as few as 3 months, the time to first event (ACM or CVH) durably separated relative to placebo
- A
42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30 - A
50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30
- Acoramidis is approved as Attruby™ by the U.S. FDA and is approved as BEYONTTRA™ by the European Commission and Japanese Pharmaceuticals and Medical Devices Agency
PALO ALTO, Calif., March 31, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today presented results showing statistically significant improvements in clinical outcomes as compared to placebo for time to all-cause mortality (ACM) or first cardiovascular-related hospitalization (CVH) in both variant (ATTRv) and wild-type (ATTRwt) transthyretin amyloid cardiomyopathy (ATTR-CM) patients from a pre-specified subgroup analysis of ATTRibute-CM, its Phase 3 trial of acoramidis in ATTR-CM. These data were presented at the American College of Cardiology (ACC) Annual Scientific Sessions & Expo in a poster presentation by Margot Davis, M.D. of Vancouver General Hospital, Canada. Acoramidis is a selective small molecule, orally administered, near-complete (≥
“Variant ATTR-CM patients’ condition often presents at an earlier age and progresses more rapidly than patients with wild-type disease, which translates into a worse prognosis in many such patients. We know that pathogenic TTR variant tetramers are less stable than the wild-type tetramer and this property is directly responsible for the more aggressive disease trajectory for these patients. The findings from the ATTRibute-CM trial clearly demonstrate that the rapid and sustained increases in serum TTR levels upon initiation of acoramidis treatment in variant ATTR-CM patients were similar if not greater than those observed in wild-type ATTR-CM patients,” said Jonathan Fox, M.D., Ph.D., president and chief medical officer of BridgeBio Cardiorenal. “This provides evidence that acoramidis is the only disease-modifying therapy that provides near-complete stabilization of TTR, improving clinical outcomes in both variant and wild-type ATTR-CM patients to an extent that is independently statistically significant in both subgroups.”
“Given the significant unmet need for patients with ATTRv-CM, we are highly encouraged by the magnitude of efficacy seen with acoramidis. A pre-specified analysis in the ATTRv-CM population has shown a statistically significant
Relative increases in serum TTR concentrations resulting from greater TTR stability have been associated with reduced risk of all-cause and cardiovascular mortality in the general population in recent literature.1 The serum TTR level increase with acoramidis was accompanied by a significant reduction in the risk of ACM or first CVH versus placebo in both the ATTRv-CM (
Additional acoramidis poster presentations and moderated posters at the ACC Annual Scientific Sessions & Expo included:
- Acoramidis Improves NYHA Class at Month 30 Versus Placebo in Patients with ATTR-CM: Results from the ATTRibute-CM Study shared by Kevin Alexander, M.D. of Stanford University School of Medicine
- In the ATTRibute-CM study, acoramidis treatment resulted in a greater proportion of patients whose New York Heart Association (NYHA) Class was stable or improved at Month 30 vs placebo, indicating better stabilization in their heart failure symptoms and functional status. The NYHA classification system categorizes heart failure patients into four classes based on a clinical assessment of their physical activity limitations and associated symptoms due to their condition
- In Participants Treated with Acoramidis, Addition of Concomitant Tafamidis Did Not Further Increase Serum TTR Levels shared by Mathew Maurer, M.D. of Columbia University Irving Medical Center
- The poster showed that in patients with ATTR-CM, treatment with acoramidis significantly increased serum TTR levels whether compared to placebo alone or in those who received placebo as well as tafamidis. Conversely, the addition of tafamidis to acoramidis did not demonstrate any further increase in serum TTR levels, reflecting the lack of any additional stabilization benefit of serum TTR from tafamidis in vivo, as previously demonstrated in vitro. The safety profile in this limited dataset of concomitant acoramidis and tafamidis was similar to the overall safety profile of acoramidis alone
- Primary Endpoint Efficacy Results in the ATTRibute-CM Study: Pre-specified Sensitivity Analyses Addressed Tafamidis Use shared by Daniel P. Judge, M.D. of Medical University of South Carolina
- Pre-specified analyses showed consistent results with the primary analysis, demonstrating that the concomitant use of tafamidis did not alter the statistical significance of the primary efficacy endpoint
- Acoramidis-mediated Early Increase in Serum Transthyretin Level Reduces Cardiovascular-related Hospitalizations and Mortality: Insights from the ATTRibute-CM Study shared by Nitasha Sarswat, M.D. of UChicago Medicine
- In this post-hoc analysis of ATTRibute-CM, incremental increases in serum TTR levels on Day 28, achieved with acoramidis, may independently predict greater reduction in risks of cardiovascular mortality and of first CVH in patients with ATTR-CM
- Robustness of Primary Endpoint Efficacy Results with Acoramidis in ATTR-CM in the ATTRibute-CM Study: Pre-specified NT-proBNP Sensitivity Analyses shared by Jan Griffin, M.D. of Medical University of South Carolina
- This poster shows that pre-specified sensitivity analyses using higher N-terminal pro-type natriuretic peptide (NT-proBNP) thresholds for declaring a difference in the changes in NT-proBNP levels between the treatment arms showed consistent efficacy favoring acoramidis in patients with ATTR-CM. This confirms the robustness of the acoramidis treatment effect regardless of NT-proBNP progression thresholds
- Geographic Healthcare Disparities and Diagnostic Trends Among Patients with Transthyretin Amyloid Cardiomyopathy shared by Joshua Mitchell, M.D. of Washington University School of Medicine in St. Louis
- Findings presented show that the diagnosed prevalence of amyloid has significantly increased since 2017 in the setting of available treatment, improved awareness and less invasive diagnostics. However, there remain geographic disparities and racial differences in ATTR-CM prevalence
Acoramidis is approved as Attruby by the U.S. FDA and is approved as BEYONTTRA by the European Commission and Japanese Pharmaceuticals and Medical Devices Agency with all labels specifying near-complete stabilization of TTR. More data on the benefit of Attruby for ATTRv-CM patients is planned for future medical meetings.
1Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102.
About Attruby™ (acoramidis)
Attruby is the first near-complete (≥
About BridgeBio
BridgeBio Pharma (BridgeBio; NASDAQ:BBIO) is a new type of biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn, Twitter and Facebook.
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,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “continue,” “will,” and variations of such words or similar expressions. We intend 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, including statements relating to the clinical and therapeutic potential of our programs and product candidates, including our clinical development program for acoramidis for patients with transthyretin amyloid cardiomyopathy and the statements regarding the potential clinical benefits or of potential benefits for ATTR-CM patients in the quotes of Dr. Alexander, reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, and strategies as reflected in or suggested by those forward-looking statements are reasonable, we 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 our preclinical studies and clinical trials not being indicative of final data, the potential size of the target patient populations our product candidates are designed to treat not being as large as anticipated, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, despite having ongoing and future interactions with the FDA and other regulatory agencies to discuss potential paths to registration for our product candidates, the FDA or such other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the continuing success of our collaborations, our ability to obtain additional funding, including through less dilutive sources of capital than equity financings, potential volatility in our share price, 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 our most recent Annual Report on Form 10-K and our other filings with the U.S. Securities and Exchange Commission. Moreover, we operate 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 our 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, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
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FAQ
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