BridgeBio's Acoramidis Shows Promise in ATTR-CM Treatment

Acoramidis shows promise in ATTR-CM patient outcomes By Investing.com

PALO ALTO, Calif. - BridgeBio Pharma, Inc. (NASDAQ:BBIO), a biopharmaceutical company focusing on genetic diseases, presented additional data from the Phase 3 ATTRibute-CM study and its open-label extension (OLE) at the European Society of Cardiology (ESC) 2024. The study evaluates the efficacy and safety of acoramidis, a small molecule stabilizer of transthyretin (TTR), in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). The results showed that participants who transitioned from tafamidis or placebo to acoramidis in the OLE experienced an increase in serum TTR levels, which is associated with reduced risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular-related hospitalization (CVH). Specifically, there was a mean increase of 3.0mg/dL in serum TTR at Month 1 (n=21, p=0.01) and 3.4mg/dL at Month 6 (n=18, p=0.01) after switching to acoramidis. The ATTRibute-CM study earlier demonstrated that acoramidis treatment resulted in increased serum TTR levels by Day 28, which correlated with a reduced risk of death and cardiovascular events over 30 months. For every 5mg/dL increase in serum TTR level at Day 28, the risk of death through Month 30 was reduced by 30.9%, and each 1 mg/dL increase in serum TTR was associated with a 5.5% reduction in cardiovascular death and a 4.7% lower risk of a first cardiovascular hospitalization. Dr. Mathew Maurer of Columbia University Irving Medical Center, who presented the data, noted that such findings could inform future treatment monitoring and decisions in ATTR-CM, particularly in the context of multiple disease-modifying therapies. BridgeBio has submitted a New Drug Application for acoramidis to the U.S. Food and Drug Administration, with a decision expected by November 29, 2024, and a Marketing Authorization Application to the European Medicines Agency, with a decision anticipated in 2025. Additionally, BridgeBio has partnered with Bayer (OTC:BAYRY), granting the latter exclusive rights to commercialize acoramidis for ATTR-CM in Europe. This news is based on a press release statement and highlights the potential benefits of acoramidis in improving clinical outcomes for patients with ATTR-CM, as well as the ongoing advances in the development and regulatory progress of the treatment. In other recent news, BridgeBio Pharma has made significant strides in the biopharmaceutical sector. The company has partnered with Yale School of Medicine's CarDS Lab on a study leveraging artificial intelligence for early detection of transthyretin amyloid cardiomyopathy, a frequently underdiagnosed heart condition. In a strategic move, BridgeBio has also formed a joint venture, GondolaBio, backed by a $300 million investment from a consortium of investors. The formation of GondolaBio aims to accelerate the development of new therapies. Analyst firms, including Citi, Wells Fargo (NYSE:WFC), and Goldman Sachs (NYSE:GS), have maintained positive ratings for BridgeBio. Citi, while adjusting its price target, maintained a Buy rating on BridgeBio. In addition, BridgeBio has launched a new online platform, MyAchonJourney, in collaboration with QED Therapeutics. This platform is designed to support individuals and families affected by achondroplasia. The company has also announced the appointment of Thomas Trimarchi, Ph.D., as its new President and Chief Operating Officer. Lastly, the Phase 3 FORTIFY study of BBP-418 by BridgeBio has exceeded its interim analysis enrollment target, with promising results shown in the Infigratinib trials. These are recent developments in the company's ongoing commitment to addressing genetic diseases. As BridgeBio Pharma, Inc. (NASDAQ:BBIO) garners attention with its promising clinical trial results for acoramidis, the financial health and market sentiment surrounding the company provide a broader context for potential investors. An InvestingPro analysis reveals several key financial metrics and insights that may influence investment decisions. According to recent data, BridgeBio Pharma's market capitalization stands at approximately $4.89 billion, reflecting the market's valuation of the company. Despite the lack of profitability over the last twelve months, analysts are optimistic about the company's sales growth in the current year. This is significant as it indicates a positive outlook on the company's ability to generate revenue, which is crucial for its long-term success and sustainability. InvestingPro Tips suggest that two analysts have revised their earnings estimates upwards for the upcoming period, signaling confidence in BridgeBio's future financial performance. However, they do not expect the company to be profitable this year, which aligns with the reported negative P/E ratio of -10.27. This metric indicates that the company is not generating net income currently, which is further supported by the adjusted P/E ratio for the last twelve months as of Q2 2024 at -8.97. When considering liquidity, BridgeBio appears to be in a strong position, with liquid assets surpassing short-term obligations. This is a reassuring sign for investors, as it suggests the company can meet its immediate financial liabilities. Furthermore, the company's gross profit margin for the last twelve months as of Q2 2024 is notably high at 98.91%, although this has not translated into operating income due to significant operating expenses. For those interested in exploring further, there are additional InvestingPro Tips available on BridgeBio Pharma, providing deeper insights into the company's financials and market performance. These tips can be accessed through the dedicated InvestingPro platform. BridgeBio's recent achievements in the clinical development of acoramidis may serve as a catalyst for future growth, and the InvestingPro metrics and tips offer a valuable perspective for stakeholders evaluating the company's potential in the biopharmaceutical landscape.

BridgeBio Pharma, Inc. Presents 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 2024

BridgeBio Pharma, Inc. 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) and 26.1% (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 and 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. 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.

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)

- 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 42% reduction in composite CVH and ACM events relative to placebo at Month 30 -A 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30 PALO ALTO, Calif., Aug. 30, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. 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) and 26.1% (by the Cox proportional hazards model), showing a statistically significant correlation between increasing serum TTR and decreasing risk of deathFor 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 30For 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 [email protected] (650)-789-8220 Market News and Data brought to you by Benzinga APIs