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

Transthyretin Kinetic Stabilizers for ATTR Amyloidosis: A Narrative Review of Mechanisms and Therapeutic Benefits. (PubMed, Cardiol Ther) - "Current availability of two kinetic stabilizers has increased interest in their pharmacological properties and clinical effects, including potential similarities and disparities. In this review, the mechanisms involved in TTR kinetic stabilization are summarized with preclinical and clinical study findings on the use of the kinetic stabilizers tafamidis and acoramidis."

Clinical • Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Pain

Evolving practices in ATTR-CM: from science to patient care (ESC-WCC 2025) - "Highlight the patient perspective and communicate the latest data on clinically meaningful improvements in markers of disease associated with acoramidis treatment...This event is sponsored by Bayer. Este medicamento está aprobado por la EMA pero aún no se encuentra comercializado en España.This drug is approved by EMA but still not marketed in Spain.July 2025 /// MA-M_ACR-ALL-0198-2 /// MA-M_ACR-ES-0030-3"

Clinical • Cardiovascular

Acoramidis leads to clinically meaningful improvements from baseline in NT-proBNP and 6-minute walk distance in patients with transthyretin amyloid cardiomyopathy: observations from ATTRibute-CM (ESC-WCC 2025) - No abstract available

Clinical • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Relative efficacy of tafamidis, acoramidis, patisiran and vutrisiran in patients with transthyretin cardiac amyloidosis: a network meta-analysis (ESC-WCC 2025) - No abstract available

Retrospective data • Cardiac Amyloidosis • Cardiovascular

Acoramidis-mediated improvement in NT-proBNP at month 30 compared with placebo in patients with ATTR-CM: results from the ATTRibute-CM study (ESC-WCC 2025) - No abstract available

Clinical • Cardiac Amyloidosis • Cardiovascular

Acoramidis reduces cardiovascular mortality (CVM): results at month 42 from the ATTRibute-CM open-label extension (OLE) study (ESC-WCC 2025) - No abstract available

Clinical • Cardiac Amyloidosis • Cardiovascular

Acoramidis has a beneficial effect compared with placebo on change from baseline in NAC ATTR stage at month 30 in patients with ATTR-CM: results from the ATTRibute-CM study (ESC-WCC 2025) - No abstract available

Clinical • Cardiovascular

Transthyretin Amyloid Cardiomyopathy-2025 Update: Current Diagnostic Approaches and Emerging Therapeutic Options. (PubMed, J Clin Med) - "First-generation therapies such as tafamidis have demonstrated survival benefits in ATTR-CM. More recently, second-generation agents-such as the TTR stabilizer acoramidis and RNA silencers including vutrisiran and eplontersen-have shown promising efficacy in clinical trials...This review outlines current diagnostic strategies and therapeutic options for ATTR amyloidosis, emphasizing the need for early detection and individualized treatment approaches. The expanding therapeutic landscape highlights the importance of accurate phenotyping and timely intervention to optimize clinical outcomes."

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

Case of steroid-induced DKA (ENDO 2025) - "Her outpatient diabetes medication included Januvia 25 mg and 10 units of insulin...Labs revealed hyperglycemia and anion gap metabolic acidosis from septic shock: Glucose: 284 mg/dL(70-90 mg/dL), bicarb15 mEq/L (22-28 mEq/L), BHB 0.1 mmol/L (0.1-0.3 mmol/L), AG 10.7 mmol/L (4-11 mmol/L), Cr 3.26mg/dL (0.50-1.10 mg/dL), lactic acid 3.0 mmol/L (0.5-2.0 mmol/L), pH 7.33 (7.35-7.45), pCO2 19 mmHg(33-45 mmHg) and pO2 115mmHg (75-105 mmHg)... The reported case details a patient with uncontrolled T2DM who developed steroid-induced DKA during treatment for septic shock. The two main mechanisms that could contribute to steroid-induced DKA include elevated peripheral insulin resistance at muscle and adipose levels through GLUT4 translocation inhibition and secondly increased glucose production through glycogenolysis and gluconeogenesis. Per the literature review, there are very few reported cases of steroid-induced DKA in T2DM patients."

Clinical • Diabetes • Metabolic Disorders • Nephrology • Obesity • Pain • Septic Shock • Type 1 Diabetes Mellitus • Type 2 Diabetes Mellitus • SLC2A4

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

BridgeBio Raises $300 Million Through Partial Capped Monetization of BEYONTTRA® European Royalty (BridgeBio) - "BridgeBio Pharma, Inc...announced it has sold a portion of royalties due to the Company from sales of BEYONTTRA in Europe to HealthCare Royalty ('HCRx') and funds managed by Blue Owl Capital ('Blue Owl') for $300 million. This royalty financing agreement monetizes select anticipated royalties and provides immediate less-dilutive capital to the Company...Under the terms of the agreement, BridgeBio has received $300 million from HCRx and Blue Owl managed funds in exchange for 60% of royalties on the first $500 million of annual BEYONTTRA net sales in Europe. The agreement includes an initial cap of 1.45x. Once the applicable cap is met, no further payments will be owed to the investors."

Commercial • Amyloidosis • Cardiomyopathy

Transthyretin Amyloid Cardiomyopathy: A Review of Approved Pharmacotherapies. (PubMed, Cardiol Rev) - "In 2019, the Food and Drug Administration approved tafamidis for the treatment of ATTR cardiomyopathy due to its ability to stabilize the TTR tetramer and prevent dissociation into monomers that subsequently cause amyloidosis. In 2024, acoramidis was approved for ATTR cardiomyopathy as a novel TTR stabilizer structurally designed to mimic the stabilizing effects of the T119M mutation by binding selectively and with high affinity to TTR, preventing the dissociation of TTR tetramer into monomers and aggregation into amyloid fibrils. In 2025, vutrisiran, a small interfering ribonucleic acid that cleaves TTR messenger RNA and decreases the production of TTR protein, was approved for use in ATTR cardiomyopathy. In their clinical trials, these approved therapies demonstrated significant mortality and morbidity benefits in patients with ATTR cardiomyopathy, including a decrease in cardiovascular events, hospitalizations, and functional status."

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

BridgeBio Raises $300 Million Through Partial Capped Monetization of BEYONTTRA European Royalty (GlobeNewswire) - "Under the terms of the agreement, BridgeBio has received $300 million from HCRx and Blue Owl managed funds in exchange for 60% of royalties on the first $500 million of annual BEYONTTRA net sales in Europe. The agreement includes an initial cap of 1.45x. Once the applicable cap is met, no further payments will be owed to the investors."

Financing • Amyloidosis • Cardiomyopathy

ACORAMIDIS UNDER THE MICROSCOPE: EXPOSING HIDDEN DANGERS AND ADVERSE EVENTS (CHEST 2025) - No abstract available

Adverse events • Cardiovascular

Hypertrophic Cardiomyopathy and Phenocopies: New Therapies for Old Diseases-Current Evidence and Future Perspectives. (PubMed, J Clin Med) - "In particular, many chemotherapy agents (alkylating agents, proteasome inhibitors, immunomodulatory drugs, and monoclonal antibodies targeting clonal cells) allowing one to treat AL amyloidosis, transthyretin stabilizers (tafamidis and acoramidis), and gene silencers (patisiran and vutrisiran) are available in transthyretin cardiac amyloidosis, and enzyme replacement therapies (agalsidase-alpha, agalsidase-beta, and pegunigalsidase-alpha) or oral chaperone therapy (migalastat) can be used in Anderson-Fabry disease. In addition, the introduction of cardiac myosin inhibitors (mavacamten and aficamten) has deeply modified the treatment of hypertrophic obstructive cardiomyopathy. The aim of this review is to describe the new disease-modifying treatments available in HCM and phenocopies in light of current scientific evidence."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Fabry Disease • Gene Therapies • Genetic Disorders • Hypertrophic Cardiomyopathy • Obstructive Hypertrophic Cardiomyopathy • Rare Diseases

Regulating Ag-Cu synergy effect via Cu doping numbers to boost CO2 electroreduction on Ag14 nanoclusters. (PubMed, Chem Commun (Camb)) - "A catalyst containing, on average, two Cu atoms per cluster (Ag11.192Cu2.808) delivers a CH4 faradaic efficiency of 17.1% at -1.6 V vs. RHE-dramatically higher than both the over-doped analogue Ag10.463Cu3.537 (11.16%) and the undoped Ag14 parent (∼0%). Density-functional-theory calculations reveal that introducing one to two Cu atoms optimally raises the Cu valence state, strengthening *CO adsorption and thereby accelerating the *CO → *CHO step that governs CH4 formation. These results demonstrate that 'less is more': beyond a critical Cu loading, the cooperative electronic advantages are diminished and activity declines."

Journal

Vutrisiran (Amvuttra) for transthyretin amyloid cardiomyopathy. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Differential Binding Affinities and Kinetics of Transthyretin Stabilizers. (PubMed, J Cardiovasc Pharmacol) - "The TTR stabilizers, tafamidis and acoramidis, are the only FDA approved treatments for patients with ATTR-CM. Relative to other stabilizers, acoramidis is more potent as independently assessed by TTR binding affinity, kinetic stability, and acid-mediated denaturation. These properties may contribute to the ability of acoramidis to achieve near-complete stabilization of TTR in plasma samples."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Pharmacological Management of Transthyretin Amyloid Cardiomyopathy: Where We Are and Where We Are Going. (PubMed, J Clin Med) - "TTR stabilizers, such as tafamidis and acoramidis, can reduce TTR instability and subsequent amyloid fibril formation...Gene-silencing therapies using small interfering RNAs (siRNAs), such as patisiran and vutrisiran, or antisense oligonucleotide inhibitors (ASOs), such as inotersen and eplontersen, serve as powerful therapeutic options by decreasing TTR production; trials on patients with ATTR-CM have been recently published or are ongoing. Novel, emerging therapies aim to enhance fibril clearance using monoclonal antibodies, such as NI006, that target amyloid deposits in the myocardium, promoting their depletion, plausibly with regression of the structural and functional impairments caused by the disease...Future directions will involve improving patients' screening to achieve earlier diagnoses, optimising patients' selection for disease-modifying therapy and identifying criteria for the treatment's response or lack thereof to possibly consider therapy..."

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

Current and emerging treatment options for transthyretin amyloid cardiomyopathy. (PubMed, Heart) - "Treatments available in clinical practice include TTR stabilisers (tafamidis and acoramidis), which prevent the dissociation of TTR tetramer into monomers and oligomers that subsequently form amyloid fibrils, and gene-silencing therapies (patisiran, inotersen and vutrisiran), which suppress the hepatic synthesis of TTR, which is the amyloid precursor protein. Novel treatment strategies that are at various stages of development include Clustered Regularly Interspaced Short Palindromic Repeats-Cas9 gene-editing technology (nexiguran ziclumeran), which, if successful, offers the prospect of a single-dose treatment, and monoclonal (cormitug and ALXN220) and pan-amyloid antibodies (AT-02) that seek to target and remove amyloid fibrils that have deposited in the myocardium...The success of ATTR-specific disease-modifying therapies has already altered the treatment landscape and changed the perception of ATTR amyloidosis from a progressive and fatal disease to one that is..."

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

Serum Transthyretin as a Biomarker of Treatment Response in ATTR Cardiomyopathy. (PubMed, J Am Coll Cardiol) - No abstract available

Biomarker • Journal • Cardiomyopathy • Cardiovascular

Early Increase in Serum Transthyretin by Acoramidis Independently Predicts Improved Survival in TTR Amyloid Cardiomyopathy. (PubMed, J Am Coll Cardiol) - P3 | "Acoramidis-mediated early ΔTTR is independently associated with improved survival after adjusting for known predictors. This provides strong evidence for a direct association between a prompt and sustained increase in sTTR upon initiation of treatment with acoramidis and survival. Early changes in sTTR could be used as a marker of the degree of TTR stabilization. (Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy [ATTRibute-CM]; NCT03860935)."

Clinical • Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

ATTRibutes of change: advances in treating and monitoring ATTR-CM (HEART FAILURE 2025) - "Beyonttra® is approved by FDA and EMA, but not yet registered by Serbian Health Authority. SmPC is available at the booth MA-M_ACR-ALL-0093-3 / MA-M_ACR-RS-0004-1 / March 2025"

Cardiovascular • Congestive Heart Failure • Heart Failure

Relative efficacy of tafamidis, acoramidis, patisiran and vutrisiran in patients with transthyretin cardiac amyloidosis: a network meta-analysis (HEART FAILURE 2025) - "Tafamidis demonstrated the highest efficacy in improving survival, reducing cardiovascular hospitalizations, and enhancing functional capacity and quality of life in patients with ATTR-CA, qualifying as the primary treatment choice. Vutrisiran and acoramidis emerged as viable alternatives."

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

Acoramidis improves serum TTR levels in patients with wild-type or variant transthyretin amyloid cardiomyopathy - results from ATTRibute-CM (HEART FAILURE 2025) - P3 | "In ATTRibute-CM, baseline sTTR levels, a measure of TTR stability, were lower in ATTRv-CM than in ATTRwt-CM. In both subgroups, acoramidis treatment induced a rapid increase in sTTR levels by Day 28, with comparable sTTR levels achieved in both subgroups from Day 28 through Month 30."

Clinical • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular