BridgeBio Shares Data on Serum TTR Increase When Switching Participants from Placebo and Tafamidis to Acoramidis in ATTRibute-CM and its Open-Label Extension
BridgeBio Pharma (Nasdaq: BBIO) presented additional data from its Phase 3 ATTRibute-CM and open-label extension study of acoramidis in ATTR-CM at ESC 2024. Key findings include:
1. Participants switching from tafamidis and placebo to acoramidis showed a mean increase in serum TTR of 3.0mg/dL at Month 1 and 3.4mg/dL at Month 6 of the OLE.
2. Increased serum TTR at Day 28 correlated with reduced risk of all-cause mortality, cardiovascular mortality, and cardiovascular-related hospitalization.
3. Acoramidis demonstrated a 42% reduction in composite CVH and ACM events and a 50% reduction in cumulative CVH events at Month 30.
BridgeBio has submitted regulatory applications for acoramidis, with FDA PDUFA date set for November 29, 2024, and EMA decision expected in 2025.
BridgeBio Pharma (Nasdaq: BBIO) ha presentato ulteriori dati dal suo studio di Fase 3 ATTRibute-CM e dallo studio di estensione in aperto su acoramidis in ATTR-CM all'ESC 2024. I principali risultati includono:
1. I partecipanti che sono passati da tafamidis e placebo a acoramidis hanno mostrato un aumento medio di TTR sierico di 3.0mg/dL al Mese 1 e 3.4mg/dL al Mese 6 dello studio di estensione.
2. L'aumento del TTR sierico al Giorno 28 è stato correlato con una riduzione del rischio di mortalità per tutte le cause, mortalità cardiovascolare e ricoveri correlati a problemi cardiovascolari.
3. Acoramidis ha dimostrato una riduzione del 42% degli eventi compositi di CVH e ACM e una riduzione del 50% degli eventi cumulativi di CVH al Mese 30.
BridgeBio ha inviato domande regolatorie per acoramidis, con una scadenza FDA PDUFA fissata per il 29 novembre 2024 e una decisione EMA attesa nel 2025.
BridgeBio Pharma (Nasdaq: BBIO) presentó datos adicionales de su estudio de Fase 3 ATTRibute-CM y del estudio de extensión a etiqueta abierta de acoramidis en ATTR-CM en el ESC 2024. Los hallazgos clave incluyen:
1. Los participantes que cambiaron de tafamidis y placebo a acoramidis mostraron un aumento medio en el TTR sérico de 3.0mg/dL en el Mes 1 y 3.4mg/dL en el Mes 6 del estudio de extensión.
2. El aumento del TTR sérico en el Día 28 se correlacionó con una reducción del riesgo de mortalidad por todas las causas, mortalidad cardiovascular y hospitalización relacionada con problemas cardiovasculares.
3. Acoramidis demostró una reducción del 42% en eventos compuestos de CVH y ACM y una reducción del 50% en eventos cumulativos de CVH al Mes 30.
BridgeBio ha presentado solicitudes regulatorias para acoramidis, con una fecha PDUFA de la FDA fijada para el 29 de noviembre de 2024, y se espera una decisión de la EMA en 2025.
BridgeBio Pharma (Nasdaq: BBIO)는 ESC 2024에서 ATTR-CM에 대한 acoramidis의 3상 ATTRibute-CM 및 개방형 확장 연구의 추가 데이터를 발표했습니다. 주요 발견 내용은 다음과 같습니다:
1. tafamidis 및 위약에서 acoramidis로 전환한 참가자들은 OLE의 1개월 차에 평균 3.0mg/dL, 6개월 차에 3.4mg/dL의 혈청 TTR 증가를 보였습니다.
2. 28일 차에 혈청 TTR의 증가는 모든 원인 사망, 심혈관 사망 및 심혈관 관련 입원의 위험 감소와 관련이 있었습니다.
3. Acoramidis는 30개월 차에 CVH 및 ACM 사건의 복합체에서 42%의 감소와 누적 CVH 사건에서 50%의 감소를 나타냈습니다.
BridgeBio는 acoramidis에 대한 규제 신청을 제출하였으며, FDA PDUFA 날짜는 2024년 11월 29일로 설정되어 있으며, 2025년에 EMA의 결정을 기대하고 있습니다.
BridgeBio Pharma (Nasdaq: BBIO) a présenté des données supplémentaires de son étude de Phase 3 ATTRibute-CM et de l'étude d'extension ouverte sur acoramidis dans l'ATTR-CM lors de l'ESC 2024. Les principales conclusions incluent :
1. Les participants passant de tafamidis et de placebo à acoramidis ont montré une augmentation moyenne du TTR sérique de 3,0 mg/dL au mois 1 et de 3,4 mg/dL au mois 6 de l'étude d'extension.
2. L'augmentation du TTR sérique au Jour 28 a été corrélée à une réduction du risque de mortalité toutes causes confondues, de mortalité cardiovasculaire et d'hospitalisations liées aux problèmes cardiovasculaires.
3. Acoramidis a démontré une réduction de 42 % des événements composites de CVH et ACM et une réduction de 50 % des événements cumulés de CVH au mois 30.
BridgeBio a soumis des demandes réglementaires pour acoramidis, avec une date PDUFA de la FDA fixée au 29 novembre 2024, et une décision de l'EMA attendue en 2025.
BridgeBio Pharma (Nasdaq: BBIO) hat zusätzliche Daten aus seiner Phase-3-Studie ATTRibute-CM und der offenen Verlängerungsstudie zu acoramidis bei ATTR-CM auf dem ESC 2024 präsentiert. Zu den wichtigsten Ergebnissen gehören:
1. Teilnehmer, die von Tafamidis und Placebo auf Acoramidis umschwenkten, zeigten einen durchschnittlichen Anstieg des Serum-TTR um 3,0 mg/dL im Monat 1 und um 3,4 mg/dL im Monat 6 der offenen Verlängerung.
2. Der Anstieg des Serum-TTR am Tag 28 korrelierte mit einem verringerten Risiko für die Gesamtmortalität, die kardiovaskuläre Mortalität und kardiovaskuläre Krankenhausaufenthalte.
3. Acoramidis zeigte eine Reduktion von 42 % bei zusammengesetzten CVH- und ACM-Ereignissen und eine Reduktion von 50 % bei kumulierten CVH-Ereignissen im Monat 30.
BridgeBio hat regulatorische Anträge für Acoramidis eingereicht, wobei das FDA-PDUFA-Datum auf den 29. November 2024 festgelegt ist und die Entscheidung der EMA für 2025 erwartet wird.
- Acoramidis showed significant increase in serum TTR levels compared to tafamidis and placebo
- 42% reduction in composite CVH and ACM events at Month 30
- 50% reduction in cumulative CVH events at Month 30
- FDA and EMA applications submitted for acoramidis approval
- Partnership with Bayer for commercialization in Europe
- None.
Insights
The data presented on acoramidis in ATTR-CM patients is highly significant. The 3.0-3.4 mg/dL increase in serum TTR levels when switching from placebo/tafamidis to acoramidis is substantial. This increase correlates with reduced risks of mortality and hospitalizations, which are important outcomes in ATTR-CM management.
The study demonstrates that for every 5mg/dL increase in serum TTR, there's a
These results position acoramidis as a potential game-changer in ATTR-CM treatment, offering superior TTR stabilization and improved clinical outcomes compared to current standards of care.
The data on acoramidis is compelling for ATTR-CM treatment. The increase in serum TTR levels when switching to acoramidis suggests superior stabilization compared to tafamidis, the current standard. This is important as TTR stabilization is the primary mechanism for treating ATTR-CM.
The correlation between increased serum TTR and reduced mortality and hospitalization risks is a strong indicator of acoramidis's potential clinical benefit. The
If approved, acoramidis could become the new gold standard in ATTR-CM treatment, potentially offering better disease management and prognosis for patients with this challenging condition.
BridgeBio's acoramidis data presents a strong case for market potential. The superior efficacy compared to tafamidis, the current market leader, positions acoramidis for significant market share capture. With a PDUFA date of November 29, 2024 and potential EU approval in 2025, BridgeBio is poised for near-term revenue growth.
The partnership with Bayer for European commercialization is strategically sound, leveraging Bayer's established presence. The TRACE AI Network Study collaboration demonstrates forward-thinking in disease identification, potentially expanding the addressable market.
Investors should note the strong clinical data and approaching regulatory decisions as key catalysts. If approved, acoramidis could become a blockbuster drug, significantly impacting BridgeBio's financial outlook and stock performance.
- In participants who switched from tafamidis and placebo in the ATTRibute-CM study to acoramidis in its open-label extension (OLE), there was a mean of 3.0mg/dL increase in serum transthyretin (TTR) at Month 1 of the OLE (n=21) and mean of 3.4mg/dL increase in serum TTR at Month 6 of the OLE (n=18)
- Increased serum TTR at Day 28 of ATTRibute-CM was correlated with reduced risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular-related hospitalization (CVH) in transthyretin amyloid cardiomyopathy (ATTR-CM)
- Greater stabilization has been shown to improve clinical outcomes for patients, and in ATTRibute-CM, acoramidis, a near-complete stabilizer of TTR, demonstrated a significant impact on mortality, hospitalizations, and quality of life including:
-An early and sustained improvement relative to placebo in time to first event (CVH or ACM) starting at Month 3
-A
-A
PALO ALTO, Calif., Aug. 30, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases, presented additional data from an analysis of its Phase 3 ATTRibute-CM and open-label extension study of acoramidis in ATTR-CM at the European Society of Cardiology (ESC) 2024. ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of TTR.
The data on change from baseline in serum TTR levels in participants receiving acoramidis versus those receiving tafamidis in the placebo group in ATTRibute-CM at Month 30, as well as serum TTR levels in patients who transitioned from placebo and tafamidis to acoramidis in the OLE study, were presented by Mathew Maurer, M.D. of Columbia University Irving Medical Center. In participants who switched from tafamidis and placebo in the ATTRibute-CM study to acoramidis in its OLE, there was a mean of 3.0mg/dL increase in serum TTR at Month 1 of the OLE (n=21, p=0.01) and mean of 3.4mg/dL increase in serum TTR at Month 6 of the OLE (n=18, p=0.01).
TTR plays an important role in the body transporting thyroxine and vitamin A, and higher TTR levels are associated with less heart failure and better survival. BridgeBio previously shared results demonstrating that acoramidis treatment resulted in increased serum TTR levels by Day 28 that were sustained and were correlated with a reduced risk of ACM, CVM, and CVH in ATTR-CM participants through Month 30. This includes the following results:
- For every 5mg/dL increase in serum TTR level at Day 28 after treatment initiation, the risk of death through Month 30 was reduced by
30.9% (by the logistic model) and26.1% (by the Cox proportional hazards model), showing a statistically significant correlation between increasing serum TTR and decreasing risk of death - For each 1 mg/dL, increase in serum TTR on Day 28 after treatment initiation, there was a
5.5% risk reduction in cardiovascular death observed through Month 30 - For each 1 mg/dL, increase in serum TTR at Day 28 after treatment initiation was associated with a
4.7% lower risk of a first cardiovascular hospitalization over 30 months
“The data shared show that switching from tafamidis to acoramidis resulted in an increase in serum TTR, which has been associated with improved outcomes in patients with ATTR-CM,” said Dr. Maurer. “In a future arena of multiple disease modifying therapies for ATTR-CM, such data may provide a rationale for monitoring of patients with serum transthyretin levels and using these data in choosing a specific treatment.”
Additionally, as part of the ongoing partnership with the Cardiovascular Data Science (CarDS) Lab, Rohan Khera, M.D., M.S., cardiologist-data scientist at Yale School of Medicine will be presenting about the artificial intelligence tools being deployed in the TRACE AI Network Study. The BridgeBio-partnered initiative will provide a scalable screening toolkit for ATTR-CM across large, diverse health systems to quantify the potential prevalence of undiagnosed ATTR-CM among all patients undergoing routine cardiovascular evaluation, specifically among key socioeconomic and demographic subpopulations.
Based on the positive results from ATTRibute-CM, BridgeBio submitted a New Drug Application to the U.S. Food and Drug Administration, which has been accepted with a PDUFA action date of November 29, 2024, and a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025. BridgeBio has granted exclusive rights to Bayer to commercialize acoramidis for ATTR-CM in Europe.
About BridgeBio Pharma, Inc.
BridgeBio Pharma Inc. (BridgeBio) is a commercial-stage 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 and Bayer European License for Acoramidis – About the Collaboration
In March 2024, BridgeBio granted Bayer exclusive license to commercialize acoramidis as a treatment for patients with transthyretin amyloid cardiomyopathy (ATTR-CM) in Europe. Acoramidis is an investigational, highly potent and selective small molecule, under development as an orally administered transthyretin (TTR) stabilizer for the treatment of patients with ATTR-CM a progressive fatal disease presenting as an infiltrative, restrictive cardiomyopathy resulting in heart failure.
This partnership leverages Bayer’s long legacy of expertise in cardiovascular disease and its established European cardiovascular infrastructure paired with BridgeBio’s leadership in the emerging field of ATTR-CM.
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. 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 impact of acoramidis on clinical outcomes, including risk of death, cardiovascular death and hospitalization rates; potential benefits of acoramidis, including increases in serum TTR; the statements related to the planned actions and decisions of the U.S. Food and Drug Administration and the European Medicines Agency regarding our New Drug Application and Marketing Authorization Application submissions for acoramidis for the treatment of ATTR-CM; and the potential outcomes and expected timing of regulatory reviews by the U.S. Food and Drug Administration and the European Medicines Agency, and the corresponding statistically significant benefits on clinical event outcomes; and the clinical, therapeutic and market potential of our clinical development program and timeline for acoramidis 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, the U.S. Food and Drug Administration 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, potential volatility in our share price, uncertainty regarding any impacts due to global health emergencies, including delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, 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 rising interest rates, on our 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.
BridgeBio Media Contact:
Vikram Bali
contact@bridgebio.com
(650)-789-8220
FAQ
What were the key findings of BridgeBio's ATTRibute-CM study for acoramidis (BBIO)?
When is the FDA PDUFA date for BridgeBio's acoramidis (BBIO)?
What is the expected timeline for EMA decision on BridgeBio's acoramidis (BBIO)?
Who has BridgeBio partnered with for acoramidis commercialization in Europe (BBIO)?
