Attruby (acoramidis) / BridgeBio, AstraZeneca, Bayer - LARVOL DELTA

Iron‑regulatory gene variants and their association with clinical phenotypes in Nigerian patients with sickle cell disease (ASH 2025) - "Results remained consistent afteradjusting for age.For lifetime transfusion burden, both UVA and MVA identified two significant SNPs: rs4667289, associatedwith higher transfusion burden (β = 2.6, 95% CI: 0.04–5.2, p = 0.047) and rs11568344, associated withlower transfusion burden (β = –1.8, 95% CI: –3.5 to –0.36, p = 0.02).Genotype distributions for the five significant SNPs were as follows: rs10177654 (SLC40A1, transcriptionfactor binding site), TT(WT) 12 (20.3%), TC 30 (50.8%), CC 17 (28.8%); rs10188230 (SLC40A1, intron variant),TT (WT) 32 (54.2%), TC 22 (37.3%), CC 5 (8.5%); rs10218795 (HJV, intron variant), CC (WT) 22 (37.3%),TC 29 (49.2%), TT 8 (13.6%); rs4667289 (SLC40A1 proximal intergenic), GG (WT) 46 (78.0%), AG 10 (16.9%),AA 3 (5.1%); rs11568344 (SLC40A1 splice region variant), GG (WT) 47 (79.7%), AG 9 (15.3%), AA 3 (5.1%).Compared with the homozygous WT, patients with rs10188230 had a lower median TS (21.65% vs. 43.3%,p = 0.04), while those with rs4667289 had..."

Clinical • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • SLC40A1

A plain language review of the ATTRibute-CM study: efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy. (PubMed, Future Cardiol) - No abstract available

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Stabilizers, Silencers, and Disease Stage: Lessons From Recent Trials of ATTR Cardiac Amyloidosis. (PubMed, J Am Coll Cardiol) - No abstract available

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Hydrophobicity-driven interfacial behavior in food-grade Lactobacillus: Cross-validation of natural surface variance and physicochemical determinants. (PubMed, Colloids Surf B Biointerfaces) - "Fourier Transform infrared spectroscopy (FTIR) and X-ray photoelectron spectroscopy (XPS) revealed that the most hydrophobic and hydrophilic strains, L. acidophilus ATCC4356 (1-1) and L. helveticus AG10-1 (8-8), exhibited the highest N/C (0.134) and O/C (0.530) ratios, indicating protein-rich and polysaccharide-dominant surfaces, respectively...Highly hydrophobic strains, lacking steric hindrance from hydrophilic polysaccharides, exhibited overall greater autoaggregation, though this behavior was also moderately influenced by zeta potential providing electrostatic repulsion between cells. These findings provide new insight into the molecular basis of Lactobacillus surface functionality and emphasize the importance of multi-method strategies for selecting and characterizing strains for probiotic development and biointerface applications."

Journal

Pyrazole-Based Transthyretin Kinetic Stabilizers Identified Using a Covalent Fluorescent Probe Assay for Selectivity Profiling in Human Serum. (PubMed, J Med Chem) - "Among these, 3,5-dichloropyrazole derivatives exhibited efficacy comparable to that of tafamidis and acoramidis. X-ray crystallography of the TTR-17 complex confirmed hydrogen bonding with Ser117/117' and electrostatic interactions with Lys15. Pharmacokinetic studies of compounds 16 and 17 demonstrated favorable exposure, bioavailability, and metabolic stability, supporting their preclinical development for hereditary- and wild-type TTR amyloidosis."

Journal • Amyloidosis

Relative efficacy of tafamidis, acoramidis, patisiran and vutrisiran in patients with transthyretin cardiac amyloidosis: a network meta-analysis. (PubMed, Eur Heart J Open) - "When the ATTR-ACT trial was excluded from the analysis, vutrisiran monotherapy consistently showed the highest probability of being ranked better than other treatments in terms of primary end-point. Although differences in trial design and study populations complicate direct efficacy comparisons, tafamidis demonstrated the highest efficacy in improving survival, reducing cardiovascular hospitalizations, and enhancing functional capacity and quality of life in patients with ATTR-CA, but also vutrisiran and acoramidis emerged as viable options."

Journal • Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Real-world effectiveness of targeted therapies in ATTR cardiomyopathy: A meta-analysis integrating population-based data. (PubMed, ESC Heart Fail) - "Targeted treatments for ATTR-CM significantly reduce mortality and cardiovascular hospitalizations. When extrapolated to population-level data, these treatments show clinical benefits, emphasizing the importance of early diagnosis and therapeutic intervention."

Journal • Real-world evidence • Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Beyonttra (acoramidis) has been approved for reimbursement by the HSE and is available in Ireland from December 1, 2025 for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). (Irish Medical Times)

Launch Europe • Reimbursement • Amyloidosis • Cardiomyopathy

ACO-REAL: A Study to Learn About the Use of Acoramidis in Patients With a Heart Condition Called Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in a Real-world Setting (clinicaltrials.gov) - P=N/A | N=2000 | Recruiting | Sponsor: Bayer

New trial • Real-world evidence • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure

Timing of Mortality Benefit in Outcomes Trials in Transthyretin Amyloidosis. (PubMed, J Am Coll Cardiol) - "TTR silencers and stabilizers in ATTR-CM confer a delayed but consistent mortality benefit, with no significant differences observed between the three major trials. This uniform pattern may reflect a shared mechanism of action-reducing new amyloid deposition rather than reversing established disease, and underscore the importance of early treatment initiation and adequate trial duration to capture delayed mortality effects."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Serum Transthyretin Levels at Day 28 are Associated with Cardiovascular Outcomes: Insights From the ATTRibute-CM Study (AHA 2025) - "Acoramidis, a near-complete (≥90%) TTR stabilizer, demonstrated a rapid and sustained increase from baseline in sTTR by Day 28 and reduced risk of cardiovascular outcomes in patients with ATTR-CM.Hypothesis: Acoramidis-mediated early increase in sTTR levels ≥20 mg/dL (lower limit of normal) at Day 28 reduces risks of CVM and cardiovascular-related hospitalizations (CVH) in patients with ATTR-CM. Analyses were conducted in the ATTRibute-CM modified intention-to-treat (mITT) population (acoramidis: 409; placebo: 202)... Across treatment groups, sTTR levels above normal range (≥20 mg/dL) at Day 28 were associated with a lower risk of cardiovascular outcomes at Month 30, when compared with sTTR levels below normal range (<20 mg/dL). Thus, these observations demonstrate that higher sTTR levels over time may have the potential for clinical benefits in both CVM and CVH."

Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Acoramidis Reduces the Risk of All-Cause Mortality and Cardiovascular-Related Hospitalization Compared With Placebo in Participants With Transthyretin Amyloid Cardiomyopathy and Early-Stage Heart Failure Regardless of Atrial Fibrillation History: Insights From ATTRibute-CM (AHA 2025) - P3 | "Lower risk of clinical outcomes (ACM and CVH) was observed with acoramidis in patients with early-stage HF, regardless of the presence or absence of an AF/AFL diagnosis at baseline."

Clinical • Amyloidosis • Atrial Fibrillation • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Acoramidis Reduces All-Cause Mortality and First Cardiovascular Hospitalization in Patients with Variant Transthyretin Amyloid Cardiomyopathy: Results From the ATTRibute-CM Study (AHA 2025) - "These previously unreported findings support the hypothesis that near complete TTR stabilization observed experimentally with acoramidis across multiple TTR mutations translates into improvement in long-term outcomes. Further validation by individual TTR variant is warranted."

Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Comparative Efficacy and Safety of Vutrisiran, Acoramidis, Tafamidis in Transthyretin Amyloid Cardiomyopathy: A Network Meta-Analysis (AHA 2025) - "Background: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, life-threatening disorder caused by transthyretin deposition in cardiac tissue. Vutrisiran demonstrated the most consistent survival and biomarker benefits, while Patisiran ranked highest in overall efficacy. Acoramidis showed strong improvement in functional capacity but varied across domains."

Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Transthyretin Cardiac Amyloidosis Evaluation and Management: 2025 ACC Concise Clinical Guidance. (PubMed, J Am Coll Cardiol) - "Finally, individuals with transthyretin amyloid cardiomyopathy have benefitted from advances in broadly effective heart failure therapies, namely mineralocorticoid receptor antagonists and sodium glucose-cotransporter 2 inhibitors, as well as specific disease-modifying therapies with transthyretin stabilizers, tafamidis and acoramidis, and the transthyretin silencer vutrisiran. The purpose of this Concise Clinical Guidance is to offer updated strategies to clinicians, reflecting the expanding therapeutic landscape, and reinforcing best practices for the diagnosis and management of transthyretin amyloid cardiomyopathy with a focus on choice of disease-modifying therapies, heart failure therapies, and future directions."

Clinical guideline • Journal • Amyloidosis • Atrial Fibrillation • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • CNS Disorders • Congestive Heart Failure • Heart Failure • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Pain • Transplantation

Adverse events profile of Transthyretin stabilizers in Transthyretin Amyloid Cardiomyopathy - A Meta-Analysis of randomised control trials (AHA 2025) - "Transthyretin (TTR) stabilizers such as Tafamidis and Acoramidis are reported to have increased clinical benefits; however, comparison of safety profiles remains limited. TTR stabilizers showed a favorable safety profile in ATTR-CM, with reduced rates of serious and cardiac failure-related adverse events and no increase in overall TEAEs. These findings support their clinical safety; however, limited study numbers and sample sizes warrant cautious interpretation and highlight the need for further research."

Adverse events • Retrospective data • Amyloidosis • Atrial Fibrillation • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Transthyretin Amyloid Cardiomyopathy: The Plot Thickens as Novel Therapies Emerge. (PubMed, US Cardiol) - "Finally, we explore key unresolved questions, including the need for head-to-head comparative trials, the potential role of combination therapy, and the optimal timing for initiating treatment as earlier recognition becomes more common. As novel therapies gain approval this review serves as a timely, focused resource to support clinicians managing ATTR-CM."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Density Functional Theory Study of Structural, Electronic and Optical Properties of 13-Atom Au-Ag-Cu Ternary Clusters. (PubMed, J Comput Chem) - "The results indicate that the structural configurations of Ag11-nCu2Aun (n = 2, 3, 4, 5, 6, 7, 9), Ag10-nCu3Aun (n = 3, 4, 6, 7, 8, 9), Ag8-nCu5Aun (n = 4, 5, 6, 7), Ag11-nCu1Aun (n = 8, 9), Ag7-nCu6Aun (n = 5, 6), and Ag6-nCu7Aun (n = 3, 5) clusters exhibit irregular structural configurations, while other ternary clusters exhibit icosahedral-like geometries...This study identified significant factors affecting the stability of Au-Ag-Cu ternary clusters, providing direction for future research on the stability of more complex ternary clusters. Investigation of optical properties offers a theoretical basis for the prospects of Au-Ag-Cu ternary clusters as optoelectronic materials."

Journal

Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: Results From ATTRibute-CM and Its Open-Label Extension. (PubMed, JAMA Cardiol) - P3 | "These hypothesis-generating results indicate that further studies are warranted to better characterize the therapeutic benefit of acoramidis in variant subgroups. ClinicalTrials.gov Identifiers: NCT03860935; NCT04988386."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Comparative Safety and Efficacy of Acoramidis Versus Tafamidis in Transthyretin Amyloid Cardiomyopathy: A Systematic Review (AHA 2025) - "Both Acoramidis and Tafamidis are quite effective in ATTR-CM. Acoramidis may generate more meaningful decreases in cardiovascular hospitalizations and biomarker improvements, with a positive trend in survival at extended terms. Comparative head-to-head trials are needed to determine comparative superiority.Keywords:Acoramidis, Tafamidis, ATTR-CM, Transthyretin Cardiomyopathy, All cause mortality, 6-minute walk distance, Cardiovascular Outcomes, Systematic review, Cardiology"

Clinical • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Fatigue • Infectious Disease • Nephrology

CA.APB.01: Electrophysiology, Amyloidosis & Precision Cardiology (AHA 2025) - "Several studies examine drug safety and efficacy (e.g., tafamidis, acoramidis, vutrisiran), while others investigate mechanisms of cardiac development, pacing strategies, and novel therapeutic approaches like gene transfer. This is not a CE accredited session. Poster Professor: Stavros Stavrakis (UNIVERSITY OF OKLAHOMA)"

Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Gene Therapies • Heart Failure • Infectious Disease • Novel Coronavirus Disease

Acoramidis Improved Clinical Outcomes, Function, Quality of Life and NT-proBNP in Patients With Transthyretin Amyloid Cardiomyopathy Regardless of Atrial Fibrillation Status at Baseline (AHA 2025) - P3 | "Acoramidis improved clinical outcomes (ACM, CVH), functional status, QoL and NT-proBNP levels relative to placebo in participants with ATTR-CM, regardless of AF/AFL diagnosis at baseline."

Clinical • Clinical data • HEOR • Amyloidosis • Atrial Fibrillation • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Acoramidis Effect on All-Cause Mortality in Patients with p.V142I (V122I) Variant ATTR-CM: Findings From the ATTRibute-CM Study (AHA 2025) - P3 | "These findings have biologic plausibility and may reflect the near-complete stabilization observed experimentally with acoramidis in p.V142I ATTR fibrils. These observations require confirmation in larger cohort studies."

Clinical • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Acoramidis Reduces All-Cause Mortality and Cardiovascular-Related Hospitalizations Through Month 42 in Transthyretin Amyloid Cardiomyopathy Across All Pre-specified Patient Subgroups (AHA 2025) - "The analyses of ACM/first CVH (Figure 1) and ACM (Figure 2) through M42 consistently favored patients initially randomized to acoramidis across all patient subgroups, including those based on sex, age, country, ATTR-CM genotype, baseline NT-proBNP, baseline eGFR, NYHA class, and NAC stage. The largest effect size for the reduction in ACM/first CVH (HR <0.50) was observed in the following subgroups: female, non-Caucasian patients, variant ATTR-CM, or NAC stage 2.ConclusionsThe long-term benefit of acoramidis was consistently shown in patients initially randomized to acoramidis treatment compared with placebo to acoramidis switch after 30 months across multiple clinically relevant subgroups, underscoring the importance of early initiation of acoramidis to reduce the long-term risk of ACM or CVH."

Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Phenotypic Evolution of Early to Late Cardiac Amyloidosis with Serial Cardiac Magnetic Resonance (AHA 2025) - "The patient was diagnosed with wild type transthyretin CA and started on acoramidis.DiscussionTimely diagnosis of CA is crucial, as therapies are more effective early in disease...Our patient had a Mayo score of 4 (screened out) and AMYLI score of 4.3 (not screened out), highlighting the clinical suspicion that can persist despite non-diagnostic testing.Conclusions This case illustrates that the "warranty period" of a CMR that does not show clear signs of CA despite reasonably high clinical suspicion may be under 5 years. Further investigations are necessary to define progression and screening intervals."

Amyloidosis • Atrial Fibrillation • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure