Tafamidis Meglumine

- In the first peer-reviewed, real-world, contemporary, comparative effectiveness study of TTR stabilizers in ATTR-CM using U.S. claims data, acoramidis was associated with a statistically and clinically significant 34% reduction in a composite of clinical worsening events (diuretic intensification, heart failure hospitalization, and mortality) versus tafamidis (p=0.046), supporting improved clinical stability of acoramidis over tafamidis - Acoramidis was also associated with a significant 43% reduction in risk of diuretic intensification versus tafamidis (p=0.021); diuretic intensification is an early marker of worsening heart failure and worse clinical outcomes - Separation in clinical outcomes emerged within weeks of treatment initiation and increased over time, reinforcing a consistent early treatment effect and durable clinical stabilization - Findings underscore acoramidis’s differentiated profile, with potential to inform frontline treatment decisions and support switching individuals to acoramidis - An additional, independent real-world study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data is expected to be released at an upcoming 2026 medical meeting PALO ALTO, Calif., Sept. 23, 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, today announced results published in Cardiology and Therapy from the first peer-reviewed, contemporary real-world comparative effectiveness retrospective study of transthyretin (TTR) stabilizers in individuals with transthyretin amyloid cardiomyopathy (ATTR-CM), demonstrating that acoramidis was associated with significantly lower risk of early clinical worsening compared to tafamidis. This represents the first peer-reviewed, real-world evidence differentiating clinical outcomes between approved TTR stabilizers, reinforcing acoramidis’s differentiated clinical profile in present-day practice. “These findings further position acoramidis as a differentiated TTR stabilizer in contemporary clinical practice,” said Richard Wright, M.D., M.A.C.C. of the Pacific Heart Institute, U.S. “The significant reduction in diuretic intensification, an early indicator of worsening heart failure, points to improved disease control and clinical stability. The progressive nature of ATTR-CM is evident in all Phase 3 trials and is seen again here. This demonstrates the need for clinicians to proactively mitigate disease progression, and these data have the potential to meaningfully inform treatment selection in newly diagnosed patients and in patients currently on other treatments.” In this analysis of newly treated individuals living with ATTR-CM, acoramidis showed early, consistent, and clinically meaningful advantages compared to tafamidis across key measures of disease progression during a mean follow-up of 4.6 months. The key findings from the study comparing acoramidis benefit versus tafamidis in individuals with ATTR-CM: 34% statistically and clinically significant reduction in risk of composite clinical worsening (HR 0.66; p=0.046), including diuretic intensification, heart failure hospitalization, and all-cause mortality43% reduction in risk of diuretic intensification (HR 0.57; p=0.021), an established early marker of worsening heart failure and predictor of hospitalization and mortality; diuretic intensification was rigorously defined as initiation or dose-equivalent escalation of oral loop diuretics, parenteral loop diuretics use, or addition of a thiazide-type diuretic48% reduction in initiation or dose-equivalent escalation of oral loop diuretics (HR=0.52; p=0.014)Rapid benefit, with separation of Kaplan-Meier curves observed within weeks of treatment initiation and increasing over timeFewer individuals with acoramidis initiated alternative ATTR-CM therapy as compared to those treated with tafamidis (4.6% vs 7.7%, respectively), consistent with findings of improved clinical stability with acoramidisBackground heart failure therapies in this study were well balanced across arms and reflect contemporary practice: 43-44% SGLT2i, 36-39% MRAMultiple falsification analyses testing for residual bias were nonsignificant The study leveraged a retrospective, longitudinal, new-user, active-comparator design using U.S. claims data and incorporated a unique dataset linking Komodo Healthcare Map U.S. claims with Claritas hub and specialty pharmacy data. After weighting, the analysis included 170 individuals with acoramidis and 448 individuals with tafamidis newly initiating treatment between Dec 2024-April 2025 and followed through July 2025, with a mean follow-up of approximately 4.6 months. 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 real-world benefit of acoramidis versus tafamidis in individuals living with ATTR-CM is planned for fall medical meetings and scientific publications, including an independent real-world study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data, expected to be released at an upcoming 2026 medical meeting. As with all real-world evidence studies, these findings should be interpreted in the context of inherent limitations of administrative claims data, including limited capture of cardiac biomarkers and other clinical measures of disease severity. Follow-up was limited given the recent approval of acoramidis, and longer-term data are needed to further add to these findings. About Attruby® (acoramidis)INDICATIONAttruby 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 INFORMATIONAdverse ReactionsDiarrhea (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 BridgeBioBridgeBio 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 bridgebio.com and follow us on LinkedIn, X, Facebook, Instagram, YouTube, and TikTok. 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 real-world data regarding acoramidis, including the potential for the findings to inform frontline treatment decisions and treatment selection for newly diagnosed patients and patients currently receiving other treatments, and to support switching individuals to acoramidis; and BridgeBio’s plans and expectations regarding the presentation and publication of additional real-world data regarding acoramidis, including an independent real-world study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data expected to be released at an upcoming 2026 medical meeting. 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, the risk that results from retrospective, observational, real-world evidence studies, including studies based on administrative claims data, may be subject to confounding, selection bias, residual bias, incomplete or inaccurate data, limited follow-up or other limitations and may not be predictive of future clinical outcomes or treatment effects; that the observed associations and differences between acoramidis and tafamidis may not be replicated in additional analyses, independent studies or longer-term data or may not translate into improved long-term clinical outcomes; that the findings may not meaningfully inform treatment selection or support switching patients to acoramidis; that additional real-world studies, including the independent study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data, may yield results that differ from or do not confirm the findings described in this press release; that plans or timing for future medical meeting presentations or scientific publications 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. BridgeBio Media Contact:Kaitlyn Reilly, Director, Communicationscontact@bridgebio.com (650) 789-8220 BridgeBio Investor Contact:Kristen Kelleher, Director, Investor Relationsir@bridgebio.com