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

Understanding Pharmacokinetic-Drug Interactions With Drugs Approved by the US Food and Drug Administration in 2024 to Better Manage the Risk of Drug Interactions With Concomitant Medications: A Review of Clinical Data From New Drug Applications. (PubMed, Curr Ther Res Clin Exp) - "Of these, 7 drugs were substrates of CYP3A, 3 of CYP2C9, one of CYP1A2, and one of CYP2C8, including the sensitive substrates vanzacaftor (CYP3A) and vorasidenib (CYP1A2). As precipitants, 6 drugs (acoramidis, cefepime/enmetazobactam, givinostat, lazertinib, mavorixafor, and resmetirom) were clinical inhibitors of CYP enzymes (2C8, 2C9, 2D6, 2E1, and 3A), with mavorixafor being a CYP2D6 strong inhibitor. Two drugs (elafibranor and tovorafenib) showed weak induction of CYP3A. Regarding transporter data, 3 drugs were substrates of transporters, including seladelpar (BCRP and OAT3), sulopenem (OAT3), and vadadustat (OAT1/3), and 8 drugs (arimoclomol, danicopan, givinostat, lazertinib, mavorixafor, resmetirom, vadadustat, and vazacaftor/tezacaftor/deutivacaftor) were inhibitors of transporters...Several DDIs with an AUC change <2 also had labeling recommendations, pertaining most often to the concomitant use of drugs with a narrow therapeutic index. Mechanistic DDI..."

Clinical data • FDA event • Journal • NDA • PK/PD data • Review • CYP1A2 • CYP2C9

2025 New Drug Update (Part 2): Recent Approvals and Clinical Implications. (PubMed, Sr Care Pharm) - "This review synthesizes the latest evidence on recently approved medications including Atzumi™ and Brekiya® (dihydroergotamine mesylate) for migraine headaches, Attruby™ (acoramidis) for transthyretin amyloid cardiomyopathy, and Blujepa® (gepotidacin) for uncomplicated urinary tract infections. Insights are offered into prescribing considerations for these agents in older populations, potential drug-drug interactions, and administration and handling tips. This work will be of significant interest to health care professionals as they evaluate the appropriateness of these new therapeutic options."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • CNS Disorders • Infectious Disease • Migraine • Nephrology • Pain

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

Effect of Acoramidis on Recurrent and Cumulative Cardiovascular Outcomes in ATTR-CM: Exploratory Analysis From ATTRibute-CM. (PubMed, J Am Coll Cardiol) - "Acoramidis significantly reduced the cumulative burden of CV outcomes in ATTR-CM over 30 months. Numerically fewer events were observed with acoramidis vs placebo by month 1, and the difference increased progressively over time, resulting in 53 events avoided per 100 treated patients at month 30. Almost one-fourth of the cumulative CV events occurred within the first 6 months. These exploratory findings suggest that cumulative event burden may occur early, highlighting the importance of timely evaluation, diagnosis and treatment in ATTR-CM."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

SAFETY AND EFFICACY OF ACORAMIDIS IN TRANSTHYRETIN AMYLOID CARDIOMYOPATHY: A SYSTEMATIC REVIEW AND META-ANALYSIS (ACC 2026) - "Abstract is embargoed at this time."

Retrospective data • Review • 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

ASSOCIATION BETWEEN EARLY INCREASE IN SERUM TRANSTHYRETIN (STTR) WITH ACORAMIDIS AND LONG-TERM EFFECTS ON HEART FAILURE-RELATED HEALTH STATUS IN TRANSTHYRETIN AMYLOID CARDIOMYOPATHY (ATTR-CM): RESULTS FROM ATTRIBUTE-CM (ACC 2026) - "Abstract is embargoed at this time."

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

Phenomenological model of transthyretin stabilization. (PubMed, Sci Rep) - "While kinetic stabilisers such as tafamidis or acoramidis are designed to prevent tetramer dissociation, clinical data show a puzzling increase in TTR levels after treatment-an effect that our study seeks to investigate by exploring possible underlying mechanisms. By integrating pharmacokinetic and pharmacodynamic data with experimental observations, our model provides fresh insights into TTR homeostasis and offers testable predictions for future research. This study highlights the power of simplified, hypothesis-driven models in uncovering biological mechanisms-or, at the very least, in identifying key questions that remain to be answered."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Pain

Current and Future Treatment Landscape of Transthyretin Amyloid Cardiomyopathy. (PubMed, Cardiol Ther) - "Therapeutic advances have significantly increased treatment possibilities, selection of appropriate therapy, switching between therapies, combination strategies, and how to monitor treatment response over time. This review summarizes available and investigational therapies for ATTR-CM and considers practical questions that guide clinical decision-making, with the goal of helping clinicians navigate the evolving therapeutic landscape."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Transplantation

Impact of Disease-Modifying Therapies on Imaging Parameters in Cardiac Amyloidosis: A Systematic Review and Meta-Analysis. (PubMed, Curr Probl Cardiol) - "NMA in ATTR cardiac amyloidosis showed that patisiran (MD -1.04%, 95% CI -1.56 to -0.52), tafamidis (-0.91%, -1.40 to -0.41), and vutrisiran (-0.90%, -1.75 to -0.05) improved GLS versus placebo, compared to acoramidis. Disease-directed therapy in ATTR and hematologic response in AL cardiac amyloidosis are associated with preservation or improvement of GLS and LV wall thickness, supporting their use as surrogate endpoints for treatment monitoring."

Journal • Retrospective data • Review • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Hematological Disorders

A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM (clinicaltrials.gov) - P3 | N=25 | Completed | Sponsor: Alexion Pharmaceuticals, Inc. | Active, not recruiting ➔ Completed

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

Long-Term Survival Benefits And Disease Stabilization With Acoramidis In Patients With Transthyretin Amyloid Cardiomyopathy (ATTR-CM) (ACC 2026) - "Abstract is embargoed at this time."

Clinical • Late-breaking abstract • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

LONG-TERM BENEFITS WITH ACORAMIDIS ON KANSAS CITY CARDIOMYOPATHY QUESTIONNAIRE-OVERALL SUMMARY (KCCQ-OS) SCORE AND SERUM TRANSTHYRETIN LEVELS IN PATIENTS WITH TRANSTHYRETIN AMYLOID CARDIOMYOPATHY (ATTR-CM) (ACC 2026) - "Abstract is embargoed at this time."

Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Contemporary Oral Medication Use and Frequency in Patients with Transthyretin Amyloid Cardiomyopathy. (PubMed, Am J Cardiovasc Drugs) - P3 | "Patients with ATTR-CM take oral medications administered multiple times a day for the treatment of HF and other comorbidities. As a BID medication, acoramidis does not appear to deviate from non-ATTR-CM pharmacotherapy strategies, and is therefore not expected to impose additional burden in a real-world setting. These data suggest that acoramidis may align with and could possibly be incorporated into patients' existing non-ATTR-CM pharmacotherapeutic regimens."

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

Investigation of Acoramidis-based ionic liquids as potential stabilizers of transthyretin using DFT calculations, molecular docking, and ADMET studies. (PubMed, Sci Rep) - "In addition, it formed a greater number of critical contacts with transthyretin (TTR) residues. Also, ADMET studies suggested favorable predicted oral bioavailability for these compounds."

Journal • Amyloidosis • Cardiac Amyloidosis

Exploring Dopamine Neurotransmitter and Silver Nanocluster (Agn; n = 4-20) Interactions: DFT Insights for Biomedical Applications. (PubMed, ACS Omega) - "Ab initio molecular dynamics simulations of select systems (DOP@Ag5 and DOP@Ag10) demonstrate their thermal and structural stability at 320 K, indicating their robustness under physiological conditions. Overall, this work offers insights into the adsorption mechanisms of dopamine on silver nanoclusters with potential implications for advancements in biosensing applications."

Journal

ACO-SWITCH: A Study to Learn More About the Change in the Blood Levels of Transthyretin When Participants With Transthyretin Amyloidosis With Cardiomyopathy Switch From Tafamidis to Acoramidis (clinicaltrials.gov) - P4 | N=50 | Not yet recruiting | Sponsor: Bayer

New P4 trial • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • CST3

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

Acoramidis (Beyonttra®) : a new era in the treatment of transthyretin amyloid cardiomyopathy (PubMed, Rev Med Liege) - "After tafamidis, acoramidis, a high-affinity tetrameric stabilizer, represents a major advance. Well tolerated, it has few drug interactions. Indicated in patients with NYHA functional class I-II, it represents a major therapeutic advance and reinforces the importance of early treatment."

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

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

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