A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)

Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque- like substance that accumulates in different organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall apart. One way occurs as a person ages, where the normal TTR protein can fall apart and form amyloid that may no longer be sufficiently cleared by the body. This type of ATTR is known as wild-type ATTR (ATTRwt). The other way occurs when a person inherits a defective TTR gene that causes the TTR protein to spontaneously fall apart. This form of the disease is known as variant ATTR (ATTRv) and can be detected in adults by a genetic test of their TTR gene before they age.
Amyloid build-up in the heart causes the heart wall to become thick and stiff and can result in heart failure and even death. Accumulation of TTR amyloid in the heart is known as transthyretin amyloid cardiomyopathy or ATTR-CM. Amyloid can also deposit in the nerve tissues leading to nerve problems. Accumulation of TTR in the nerves is known as transthyretin amyloid polyneuropathy or ATTR-PN.
Acoramidis is an experimental drug designed to bind tightly to TTR in the blood and stabilize its structure, so it does not form the harmful amyloid plaques that can cause damage to organs.
This study is intended to determine if treatment with acoramidis in participants with ATTRv who have not yet developed any symptoms of disease can prevent or delay the development of ATTR-CM or ATTR-PN disease. If adults with an inherited defective TTR gene are treated early before any of the symptoms of disease have developed, it may be possible to delay the onset or prevent the disease entirely.

Start Date12 May 2025

Sponsor / Collaborator

Eidos Therapeutics, Inc.

ISRCTN11628974

/ CompletedPhase 1

A multiple dose study to evaluate the pharmacokinetics of an acoramidis (AG10) modified release tablet formulation in healthy subjects

Start Date30 Aug 2022

Sponsor / Collaborator

Eidos Therapeutics, Inc.

NCT04988386

/ Active, not recruitingPhase 3

An Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

Start Date27 Oct 2021

Sponsor / Collaborator

Eidos Therapeutics, Inc.

100 Clinical Results associated with Acoramidis

100 Translational Medicine associated with Acoramidis

100 Patents (Medical) associated with Acoramidis

Literatures (Medical) associated with Acoramidis

01 Dec 2025·Current heart failure reports

Etiological Treatment of Cardiac Amyloidosis: Standard of Care and Future Directions

Review

Author: Morfino, Paolo ; Emdin, Michele ; Vergaro, Giuseppe ; Chubuchna, Olena ; Aimo, Alberto ; Castiglione, Vincenzo ; Buda, Gabriele ; Ferrari Chen, Yu Fu ; Fabiani, Iacopo

Abstract:

Purpose of Review:

Cardiac amyloidosis (CA) is a condition caused by interstitial infiltration of misfolded proteins structured into amyloid fibrils. Transthyretin (ATTR) and immunoglobulin light chain (AL) amyloidosis represent the most common forms of CA. CA was traditionally perceived as a rare and incurable disease, but diagnostic and therapeutic advances have undermined the conventional paradigm.

Recent Findings:

The standard of care for ATTR-CA include agents capable of selectively stabilizing the precursor protein (e.g., tafamidis), whereas the plasma cell clone is the main target of chemotherapy for AL-CA. For long, tafamidis represented the only drug approved for patients with ATTR-CA. Recent data from ATTRibute-CM led to the approval of acoramidis, whereas patisiran received refusal based on the APOLLO-B trial. Novel CRISPR-Cas9-based drugs (i.e., NTLA-2001) hold great potential in the setting of ATTR-CA. Several hematological regimens are available to treat AL-CA. The main limit of current therapies is their inability to trigger removal of amyloid from tissues. However, the investigation of monoclonal antibodies targeting misfolded ATTR (e.g., PRX004, NI301A) or AL (e.g., birtamimab, anselamimab) has led to encouraging results.

Summary:

Various cutting-edge strategies are being tested for treatment of CA and may change the prognostic landscape of this condition in the next years.

01 Dec 2025·MOLECULAR BIOLOGY REPORTS

Transthyretin as a therapeutic target: the future of disease-modifying therapies for Alzheimer’s disease

Review

Author: Shaw, Swetaleena ; Aran, Khadga Raj ; Porel, Pratyush

BACKGROUND:

Alzheimer's disease (AD) is the most common neurodegenerative disease for causing memory deficits and primarily characterized by extracellular deposition of amyloid-β (Aβ) plaques, intracellular neurofibrillary tangles (NFTs), and hyperphosphorylation of tau protein, all are pathological hallmarks for AD. Transthyretin (TTR) is a highly conserved homo-tetrameric protein, primarily synthesized in liver and choroid plexus, and most importantly involved in transport of T₃-T₄ hormones and retinol.

OBJECTIVES:

This review explores the dual role of TTR, with a greater emphasis on its neuroprotective action, particularly in AD.

METHODS:

Based on the available literature, TTR's potential as a biomarker in the central nervous system (CNS), focusing its role in stabilizing Aβ aggregation and the senile plaque formation during neurodegeneration. Additionally, TTR's dual roles, in neurodegeneration and neuroprotection are studied, emphasizing its potential for improving AD diagnosis and treatment strategies.

RESULTS:

Recent research has revealed that TTR is gradually showcasing its promise in neuroprotection and neuronal viability in AD by binding with Aβ and mitigating its neurotoxic effects. Current preclinical and clinical studies also support that TTR is actively involved in maintaining the blood-brain barrier (BBB) integrity and maintain neurotransmitter balance, all of which offer significant therapeutic promise through TTR stabilizers, such as Tafamidis, Acoramidis, and Vutrisiran, highlighting their potential in AD treatment CONCLUSION: This review concludes that TTR plays bidirectional role and gaining interest as a potential biomarker, though several challenges must be addressed before it can be established a novel therapeutic target in AD management in the modern era of drug discovery.

16 Apr 2025·AMERICAN JOURNAL OF HEALTH-SYSTEM PHARMACY

Acoramidis Hydrochloride

Article

167

News (Medical) associated with Acoramidis

20 May 2025

·www.globenewswire.com

Acoramidis Reduced Incidence of Atrial Fibrillation Events in Patients with ATTR-CM

- 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

Clinical ResultDrug ApprovalPhase 3AHA

19 May 2025

·www.einpresswire.com

Early and Sustained Increase in Serum TTR Levels by Acoramidis Independently Predicted Improved Survival in the ATTRibute-CM Study

- For each 5-mg/dL increase in serum TTR level within 28 days of starting treatment, the relative risk reduction of mortality was up to 31.6% through Month 30, confirming the hypothesis that ever better levels of stabilization achieved by treatment with acoramidis, a near-complete (≥90%) TTR stabilizer, lead to ever better clinical outcomes - ATTRibute-CM is the only study to demonstrate a direct association between a prompt, sustained increase in serum TTR and survival in patients with ATTR-CM - The open-label extension data for all ATTRibute-CM participants at Month 42 showed that rapid, sustained TTR stabilization from acoramidis demonstrated statistically significant reductions in ACM and CVH (including urgent outpatient treatment for heart failure exacerbations) - 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, the Japanese Pharmaceuticals and Medical Devices Agency, and the UK Medicines and Healthcare Products Regulatory Agency PALO ALTO, Calif., May 19, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, published data showing that an early, sustained increase in serum transthyretin (TTR) levels predicted improved survival in ATTRibute-CM, its Phase 3 trial of acoramidis in transthyretin amyloid cardiomyopathy (ATTR-CM). These findings were published in Journal of the American College of Cardiology (JACC) in the Special Focus Issue: Amyloid . Acoramidis is a selective, small molecule, orally administered, near-complete (≥90%) TTR stabilizer. These findings further support the thesis that ever better increases in serum TTR lead to ever better clinical outcomes and that early elevations in serum TTR are an important prognostic marker to inform treatment selection. “Patients with ATTR-CM have progressive amyloid accumulation in the heart, which when untreated manifests as progressive heart failure, arrhythmias, and eventually can result in death. Increases in serum TTR seen with acoramidis therapy within 28 days of initiation and that were sustained with therapy, were associated with a decrease in all-cause mortality independent of baseline risk among subjects in the ATTRibute-CM trial. The increase in serum TTR is hypothesized to be due to a leftward shift in amyloidogenic TTR to a more stable tetrameric TTR. This is the first concrete evidence that there is a link between this rapid increase in serum TTR and survival. Such data may inform clinical practice, as early and sustained increases in serum TTR could represent a new potential ATTR disease-specific and prognostic biomarker that may further inform clinical decisions in optimizing care for ATTR-CM patients,” said Mathew Maurer, M.D. of Columbia University Irving Medical Center. In patients with ATTR-CM, aging or an inherited variant can cause tetrameric TTR to destabilize and misfold, causing buildup of amyloid fibrils in the organs, specifically in the heart. These data demonstrate that by using acoramidis, a selective near-complete TTR stabilizer to bind serum TTR, a rapid and sustained increase in tetrameric TTR was independently associated with an improvement in overall survival, even after adjustment for known predictors like TTR variant status, baseline New York Heart Association (NYHA) functional class, baseline National Amyloidosis Centre (NAC) stage, and baseline serum TTR levels. Findings from the analysis included: Treatment with acoramidis resulted in a sharp, significant early rise in serum TTR levels (mean 9.1 mg/dL) within 28 days which was sustained throughout the 30-month treatment period For every 5-mg/dL increase in serum TTR level, the Cox proportional hazards model predicted a relative risk reduction of mortality of 26.6% and the logistic model predicted a relative reduction of 31.6% in odds of death through Month 30 An early increase in serum TTR levels on Day 28 of dosing was associated with reduced all-cause mortality (ACM) in univariate analysis, an association which persisted in multivariate analysis independent of TTR variant status, baseline NYHA functional class, baseline NAC stage, and baseline serum TTR levels Logistic modeling demonstrated that among participants treated with acoramidis, the early increase in serum TTR was associated with reduced ACM, whereas there was no prompt and sustained increase in serum TTR observed in participants treated with placebo Causal mediation analysis showed evidence that the acoramidis treatment effect on ACM probability through month 30 was fully mediated by the observed prompt and sustained increase in serum TTR “This landmark analysis adds an incredibly important proof point to acoramidis’s repository of compelling data that higher serum TTR levels are directly correlated with and mediate a reduction in mortality risk.” said Jonathan Fox, M.D., Ph.D., Chief Medical Officer of BridgeBio Cardiorenal. “We believe that this will be an important measure for physicians treating ATTR-CM to consider and will be encouraging information for new and existing patients when reviewing options to treat their condition.” Data from 42 months sustained treatment in the open-label extension study from ATTRibute-CM showed that rapid and sustained TTR stabilization from acoramidis demonstrated statistically significant reductions in both ACM and cardiovascular-related hospitalizations (CVH, which included urgent outpatient treatment for heart failure exacerbations). The continued curve separation of the composite endpoint of ACM and CVH emphasizes the importance of early and continuous treatment resulting in early and sustained clinical benefits. These data further underscore the hypothesis that ever better levels of stabilization lead to ever better clinical outcomes and emphasizes the importance of a prognostic biomarker, serum TTR, to inform decision making for patient care. 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 with all labels specifying near-complete stabilization of TTR. 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 BridgeBio Pharma, Inc. BridgeBio Pharma, Inc. (BridgeBio) 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 for sustained increases in serum TTR to serve as a disease-specific and prognostic biomarker in ATTR-CM, the belief that early elevations in serum TTR are an important prognostic marker to inform treatment selection, the hypothesis that improved serum TTR stabilization is correlated with better clinical outcomes, and expectations about how these findings may guide physician decision-making and patient care, 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, Executive Vice President contact@bridgebio.com (650)-789-8220 BridgeBio Investor Contact: Chinmay Shukla, VP Strategic Finance ir@bridgebio.com Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Clinical ResultPhase 3Drug ApprovalAHA

14 May 2025

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Bluebird extends deal deadline; Affimed files for insolvency

Today, a brief rundown of news involving Bluebird bio and Affimed, as well as updates from Cytokinetics, BridgeBio Pharma and Acelyrin that you may have missed.Carlyle and SK Capital Partners, two investment firms attempting to buy Bluebird bio, have amended their offer to give Bluebird shareholders a choice of payouts. Holders of Bluebird stock can either receive $3 per share plus rights to $6.84 more if a certain sales milestone is achieved Carlyle and SK Capitals original bid or $5 per share. Shareholders previously had a deadline of May 12 to tender their stock to the original deal, but now have until May 29 to do so. Bluebirds board is in favor of accepting Carlyle and SK Capitals offer, warning investors in a statement Wednesday that, absent a deal, the company is at significant risk of default. Ned PagliaruloCancer drug developer Affimed has filed for insolvency, warning investors Tuesday that there is substantial doubt the company can sustain itself. As a result of the filing, Affimed shares will be suspended from trading on the Nasdaq stock exchange beginning May 20. The company has been talking with potential investors and partners about deals or other ways in which it might raise capital, but has so far been unsuccessful. Affimed, which has three drugs in clinical testing, last reported cash holdings of 24 million euros as of Sept. 30, 2024. Ned PagliaruloCytokinetics on Tuesday said its experimental drug aficamten outperformed beta blockers in a Phase 3 study of people with obstructive hypertrophic cardiomyopathy. U.S. regulators are already reviewing aficamtens use in the inherited heart condition, with an approval decision now expected by late December after a recent delay. The latest results, though generally expected by investors, could help boost its case with physicians and insurers, wrote RBC Capital Markets analyst Brian Abrahams. If approved, aficamten would compete for market share with Bristol Myers Squibbs Camzyos, which booked $602 million in sales last year. Ben FidlerBridgeBio Pharma has begun dosing patients in a first-of-its-kind Phase 3 trial testing whether its drug, Attruby, can prevent or delay the onset of transthyretin amyloidosis in people more likely to inherit it. The trial, ACT-EARLY, will evaluate Attruby as a prophylactic treatment in carriers of a disease-causing variant of the TTR gene thats implicated in the hereditary form of the condition. Attruby is one of three marketed drugs that can slow progression of a type of transthyretin amyloidosis that affects the heart. No medicines have been proven to prevent the disease, however. Ben FidlerShareholders in Acelyrin and Alumis voted Tuesday to combine the two biotechnology companies, ending a twisting merger saga that featured an unsolicited bid from an entity controlled by Tang Capital and a push from Trium Capital to force Acelyrin to liquidate its assets and return cash to its stockholders. Since announcing the planned combination, Alumis has tweaked the terms of the deal, saying its shareholders would own 52% of the new company, while Acelyrins would hold 48%. The merger is now set to close in the second quarter of 2025. Gwendolyn Wu '

Phase 3Acquisition

100 Deals associated with Acoramidis

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KEGG	Wiki	ATC	Drug Bank
D11972	-	-	-

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Amyloidosis, Hereditary, Transthyretin-Related	European Union	BridgeBio Pharma, Inc.	10 Feb 2025
Amyloidosis, Hereditary, Transthyretin-Related	Iceland	BridgeBio Pharma, Inc.	10 Feb 2025
Amyloidosis, Hereditary, Transthyretin-Related	Liechtenstein	BridgeBio Pharma, Inc.	10 Feb 2025
Amyloidosis, Hereditary, Transthyretin-Related	Norway	BridgeBio Pharma, Inc.	10 Feb 2025
Transthyretin Amyloid Cardiomyopathy	United States	BridgeBio Pharma, Inc.	22 Nov 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Polyneuropathies	Phase 3	United States	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	Bulgaria	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	Italy	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	Netherlands	Eidos Therapeutics, Inc.	12 May 2025
Polyneuropathies	Phase 3	Spain	Eidos Therapeutics, Inc.	12 May 2025
Transthyretin-related (ATTR) familial amyloid polyneuropathy	Phase 3	United States	Eidos Therapeutics, Inc.	21 Oct 2020
Senile cardiac amyloidosis	Phase 3	United States	Eidos Therapeutics, Inc.	19 Mar 2019
Senile cardiac amyloidosis	Phase 3	Australia	Eidos Therapeutics, Inc.	19 Mar 2019
Senile cardiac amyloidosis	Phase 3	Belgium	Eidos Therapeutics, Inc.	19 Mar 2019
Senile cardiac amyloidosis	Phase 3	Brazil	Eidos Therapeutics, Inc.	19 Mar 2019

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03860935 (ATTRibute-CM, Pubmed \| J Am Coll Cardiol)	Phase 3	Transthyretin Amyloid Cardiomyopathy \| Senile cardiac amyloidosis N-terminal pro-B-type natriuretic peptide level	632	Acoramidis hydrochloride 800 mg twice daily	gcgknklimh(ftefbparec): relative risk ratio = 50 (95% CI, 0.36 - 0.7), P-Value = < 0.0001 View more	Positive	01 Mar 2025
				Placebo
NCT04622046 (CTgov)	Phase 3	Transthyretin Amyloid Cardiomyopathy	25	ALXN2060	dqdgrjmrdy(awhsauamvd) = oitxfkqeka nwlujymijj (mdlmgqousv, 9.182) View more	-	16 Dec 2024
NCT03860935 (FDA_CDER) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	611	ATTRUBY 712 mg	lglnboclpy(rpjxmqqxna): P-Value = 0.018 View more	Positive	22 Nov 2024
				Placebo
NCT03860935 (ATTRibute-CM, Company_Website) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	389	Acoramidis	zomktxfghx(jbujxmimgl) = lgpixglegg lgrmmycrqs (rarhcxizro ) View more	Positive	18 Nov 2024
				Placebo	-
NCT03860935 (ATTRibute-CM, GlobeNewswire \| PipelineReview) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	-	Acoramidis	ekndczxjnw(avuwkvwxga) = camfygudeg mbltxqgucx (wdbcyvqbvo )	Positive	27 Sep 2024
				Placebo	-
NCT03860935 (ATTRibute-CM, ESC2024)	Phase 3	Transthyretin Amyloid Cardiomyopathy serum TTR levels	611	Acoramidis	fdaxaxnrkh(gyiftasqas) = ycfyilqdcg lrtdpgxaef (qcpdtbjafe )	Positive	30 Aug 2024
				Tafamidis	fdaxaxnrkh(gyiftasqas) = mpuagbtzcw lrtdpgxaef (qcpdtbjafe, 0.4 (4.92))
NCT03860935 (ATTRibute-CM, CTgov)	Phase 3	Transthyretin Amyloid Cardiomyopathy \| Senile cardiac amyloidosis	632	acoramidis (Acoramidis HCl 800 mg)	oofkdprvmp = meemkccmoq tvnhjpsvik (umzkxrlkar, vfrnzpeqzv - dppioteseg) View more	-	27 Jun 2024
				Placebo Oral Tablet (Placebo)	oofkdprvmp = brhnkksuqe tvnhjpsvik (umzkxrlkar, fefxnsmqsd - zizadspfjj) View more
NCT03860935 (ATTRibute-CM, Company_Website) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	-	Acoramidis	vhaodjxogy(nlwzcpmsad) = 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) icbzoujnux (vbcihhjose ) View more	Positive	29 May 2024
				Placebo
ESC2024	Not Applicable	Transthyretin Amyloid Cardiomyopathy	-	Acoramidis	wnalzxhcrb(kqllqjonsv) = A statistically significant (p<0.0001) and clinically meaningful treatment benefit on KCCQ-OS was observed with acoramidis versus placebo xchderacln (uoijjrjmjf ) View more	-	13 May 2024
				Placebo
ESC2024	Not Applicable	Transthyretin Amyloid Cardiomyopathy	-	Acoramidis	aotdarujcp(zcrnyolesz) = elxxuqawef cojnyllolj (kuufdbyuhc, 0.07 - 0.18)	-	13 May 2024

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