Tafamidis

- In a post-hoc analysis of ATTRibute-CM, acoramidis reduced the annual frequency of CVH due to AF/AFL by 43% compared to placebo and reduced the incidence of new-onset AF/AFL by 17% in the subgroup with no prior history of AF compared to placebo - 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 ATTRwt-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, Japanese Pharmaceuticals and Medical Devices Agency and UK Medicines and Healthcare Products Regulatory Agency PALO ALTO, Calif., May 20, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, presented data from ATTRibute-CM, highlighting the reduced incidence of atrial fibrillation (AF) events in the overall ATTR-CM population. These data were presented in a moderated ePoster at the Annual Congress of the Heart Failure Association of the ESC (Heart Failure 2025), taking place in Belgrade, Serbia from May 17 - 20, 2025. Acoramidis is a selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer. “The reduction in new-onset atrial fibrillation and AF-related hospitalizations represents an important finding for the ATTR-CM community. I am encouraged by the growing body of data from the ATTRibute-CM study, which adds to the understanding of acoramidis and its potential impact on clinical outcomes for patients. Furthermore, the observed reductions in hospitalizations and mortality, along with improvements in functional capacity and quality of life, suggest that acoramidis may offer benefit to both variant and wild-type ATTR-CM patients who have limited treatment options,” said Kevin Alexander, M.D. of Stanford University School of Medicine, USA. “These data support further consideration of acoramidis as a promising front-line therapy for ATTR-CM, particularly for patients with the hereditary form of the disease, who often face rapid and severe progression.” Details from the post-hoc analysis on incidence of AF in ATTRibute-CM included: Acoramidis Treatment Is Associated with a Lower Incidence of Atrial Fibrillation-related Events in Patients with ATTR-CM: A Post-hoc Analysis of the ATTRibute-CM Trial, presented by Dr. Alexander AF is a common complication of ATTR-CM, observed in up to 70% of patients, and the onset of AF is associated with an increased risk of cardiovascular-related hospitalizations (CVH)In ATTRibute-CM, a 43% relative risk reduction in the annual frequency of CVH due to AF/atrial flutter (AFL) was observed with acoramidis relative to placebo. In the subgroup who had no prior history of AF, a 17% lower incidence of new-onset AF/AFL was reported with acoramidis compared to placeboThese findings show the potential of acoramidis to reduce both disease progression, as indicated by a lower incidence of new-onset AF/AFL, and CVH morbidity caused by AF/AFL, in patients with ATTR-CM In addition to the moderated ePoster, three analyses were shared on the strong clinical outcomes in ATTRv-CM versus placebo. ATTRv-CM is associated with early age of disease onset with more advanced heart failure symptoms, which often leads to a poorer prognosis than those with wild-type ATTR-CM (ATTRwt-CM). These findings included: Acoramidis Improves Serum TTR Levels in Patients with Wild-type or Variant Transthyretin Amyloid Cardiomyopathy: Results from ATTRibute-CM, presented by Anique Ducharme, M.D. of Université de Montréal, CAN In both subgroups of ATTRv-CM and ATTRwt-CM, acoramidis treatment induced a rapid increase in serum TTR levels, a measure of TTR stability, by Day 28, with comparable serum TTR levels achieved in both subgroups from Day 28 through Month 30. 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 literature1 Effect of Acoramidis on Functional Capacity and Quality of Life in Patients with Variant ATTR-CM: Results from ATTRibute-CM, presented by Marianna Fontana, M.D. of University College London, UK Data from ATTRibute-CM showed that when acoramidis was administered for 30 months, participants with ATTRv-CM had a clinically significant slower decline in functional capacity and quality of life compared with placebo, consistent with the overall results in both ATTRv-CM and ATTRwt-CMAt Month 30, the mean difference between acoramidis and placebo treatment groups in the change from baseline in 6-minute walk distance was 86.7 meters (p = 0.0048) in favor of acoramidis and in the change from baseline in KCCQ-OS at Month 30, was 20.3 points (p = 0.0019) in favor of acoramidis, in patients with ATTRv-CM Effect of Acoramidis on All-cause Mortality, Cardiovascular Hospitalization and NT-proBNP in Variant ATTR-CM: Results from ATTRibute-CM, presented by Marianna Fontana, M.D. of University College London, UK In ATTRibute-CM, acoramidis treatment administered for 30 months led to a substantial reduction (>50%) in the composite of all-cause mortality (ACM)/CVH, ACM and CVH in participants with ATTRv-CM compared to placebo. This improvement was accompanied by favorable effects on N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels “Based on the findings from ATTRibute-CM, we believe that acoramidis has the potential to enable patients with both variant and wild-type ATTR-CM to live longer, healthier lives, especially for those with variant ATTR-CM who typically have a poorer prognosis. We observed both a striking reduction in the frequency of cardiovascular hospitalizations (which included clinic or emergency department visits for urgent heart failure management) and a clinically important and statistically significant reduction in all-cause mortality in the important subgroup. These clinical outcomes were further mirrored in robust improvements in functional capacity, quality of life, and biomarkers of heart failure severity,” said Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of BridgeBio Cardiorenal. “Given these compelling results, acoramidis should be considered as first-line treatment for newly diagnosed patients, and those currently on other therapies could be switched to acoramidis to maximize their potential to achieve such benefits.” Additional acoramidis moderated ePosters at Heart Failure 2025 included: Disease Progression Among Patients Receiving Tafamidis for ATTR-CM in a Real-world Setting, presented by Daniel P. Judge, M.D. of Medical University of South Carolina, USA This analysis suggests disease progression despite treatment with tafamidis in ATTR-CM. CVH was frequent, with approximately 1 in 5 tafamidis-treated patients hospitalized in the first six months of therapy. As more therapeutic options become available, measuring the clinical effectiveness of therapies in a real-world setting will be important to help inform physicians and patients when making treatment decisions Cause of Death in Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM): Findings from the ATTRibute-CM Study, presented by Laura Obici, M.D. of University of Pavia, ESP In the ATTRibute-CM study, total deaths were numerically lower with acoramidis compared with placebo. The relative risk reduction of 30% in cardiovascular-related mortality by Month 30 was driven predominantly by a reduction in heart failure-related deaths Time from First Recorded Clinical Manifestation to Diagnosis of Transthyretin Amyloid Cardiomyopathy: A Retrospective Cohort Study Using U.S. Claims Data, presented by Joshua Mitchell, M.D., Washington University School of Medicine in St. Louis, USA The median time from the first documented clinical manifestation to ATTR-CM diagnosis was almost 5 years, and over 2 years from the first heart failure diagnosis. This demonstrates that the patient journey to an ATTR-CM diagnosis can be prolonged and challenging, which potentially leads to more severe disease at diagnosis. Understanding the factors contributing to diagnostic delays is important to improving diagnostic pathways and patient outcomes Acoramidis is approved as Attruby by the U.S. FDA and is approved as BEYONTTRA by the European Commission, Japanese Pharmaceuticals and Medical Devices Agency, 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. 1Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102. 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 BridgeBioBridgeBio 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, Facebook, and YouTube. 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,” “could,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “potential,” “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, including statements regarding the potential impact of acoramidis on clinical outcomes for patients, the possibility that acoramidis may offer benefit to both variant and wild-type ATTR-CM patients, the consideration of acoramidis as a promising front-line therapy for ATTR-CM, the potential of acoramidis to reduce disease progression and cardiovascular hospitalization morbidity caused by AF/AFL, BridgeBio’s belief that acoramidis could help patients live longer, healthier lives—especially those with variant ATTR-CM—and the view that acoramidis should be considered as a first-line treatment or as a replacement for current therapies to maximize patient benefit, reflect BridgeBio’s current views about its plans, intentions, expectations, and strategies, which are based on the information currently available to BridgeBio and on assumptions it has made. Although BridgeBio believes that its plans, intentions, expectations, and strategies as reflected in or suggested by these forward-looking statements are reasonable, it can give no assurance that such 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 risks associated with BridgeBio’s dependence on third parties for development; regulatory authorities requiring additional studies or data to support the continued or expanded commercialization of acoramidis; whether data and results meet regulatory requirements or are sufficient for continued development, review, or approval; and whether other regulatory agencies agree with BridgeBio’s strategies or data interpretations. These risks also include impacts from global health emergencies, such as delays in regulatory reviews and other activities, manufacturing and supply chain interruptions, adverse effects on healthcare systems, and disruption of the global economy; and the impacts of macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing inflation rates, and fluctuating interest rates on BridgeBio’s operations and expectations. Additional risks are described in the Risk Factors section of BridgeBio’s most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings with the U.S. Securities and Exchange Commission. Moreover, BridgeBio 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 BridgeBio’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 these statements. Except as required by applicable law, BridgeBio assumes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events, or otherwise. BridgeBio Media Contact:Bubba Murarka, EVP Communicationscontact@bridgebio.com (650)-789-8220 BridgeBio Investor Contact:Chinmay Shukla, VP Strategic Financeir@bridgebio.com