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

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) - "A total of 611 participants were analysed in the modified intention-to-treat population (acoramidis: 409; placebo: 202). Participant baseline characteristics were comparable between groups. A total of 106 (25.9%) participants in the acoramidis group showed improvement in at least one parameter compared with 19 (9.4%) in the placebo group (Table 1; OR 3.4, 95% CI 2.0–5.7, p<0.0001)."

Clinical • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Optimizing treatment strategies for transthyretin amyloid cardiomyopathy: a comprehensive Bayesian network meta-analysis of disease-specific therapies (ESC-WCC 2025) - "Emerging disease-specific therapies, including tafamidis, patisiran, acoramidis, and vutrisiran, have shown promise in improving clinical outcomes. This study highlights the differential benefits of disease-specific therapies in ATTR-CM. patisiran was most effective for mortality reduction, acoramidis for cardiovascular hospitalizations, patisiran for functional capacity, and tafamidis for quality of life. Treatment selection should be individualized based on mortality risk, functional capacity, and quality of life to optimize ATTR-CM management."

Retrospective data • 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) - "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-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) - "Acoramidis treatment resulted in improved or stable NT-proBNP at M30 in about 50% of study participants compared with fewer than 20% with placebo, indicating a clinically meaningful improvement in NT-proBNP and better stabilization of their disease. Further studies are warranted to assess the durability of these effects and implications for the long-term clinical benefits of acoramidis."

Clinical • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Acoramidis reduces cardiovascular mortality (CVM): results at month 42 from the ATTRibute-CM open-label extension (OLE) study (ESC-WCC 2025) - "Acoramidis treatment administered for 42 months led to a 44% hazard reduction in CVM compared with the placebo to acoramidis treatment group. These findings demonstrate the long-term clinical benefits of acoramidis, a near-complete TTR stabilizer, for reducing CVM in ATTR-CM, and the importance of early treatment."

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

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) - "Acoramidis treatment resulted in a greater proportion of participants whose NAC stage improved or remained stable at Month 30 compared with placebo, indicating better stabilization of their disease."

Clinical • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Acoramidis Demonstrates Statistically Significant Reduction in Cardiovascular Mortality (CVM) through Month 42 of the ATTRibute-CM Open Label Extension (The Manila Times) - "Acoramidis also demonstrated a significant 46% hazard reduction in the risk of the composite outcome of CVM or first CVH through 42 months; Acoramidis demonstrated higher rates of disease stabilization or improvement versus disease progression as compared to placebo as reflected in change from baseline in NT-proBNP and NAC Stage; A 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30"

P3 data • Amyloidosis • Cardiomyopathy

Transthyretin cardiac amyloidosis: advances and ambiguities. (PubMed, Heart Fail Rev) - "As of March 2025, Vutrisiran is the latest addition to the FDA-approved medications for ATTR-CM, alongside Tafamidis and Acoramidis. These AI-driven tools could be integrated into electronic medical record systems to flag at-risk patients and allow for more rapid diagnosis. This review provides an overview of the current landscape and future directions of the diagnosis, treatment, and screening of ATTR-CM."

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

BridgeBio to Present Additional Open-label Extension Data from ATTRibute-CM at ESC Congress 2025 (GlobeNewswire)

P3 data • Amyloidosis • Cardiomyopathy

To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE) (clinicaltrials.gov) - P3 | N=250 | Not yet recruiting | Sponsor: Eidos Therapeutics, a BridgeBio company

New P3 trial • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Heart Failure

Design of a Long-Acting Morphine Prodrug for Safe Opioid Delivery. (PubMed, ChemMedChem) - "Herein, the design and characterization of AG10-L-E2-morphine, a prodrug that forms a zwitterionic subcutaneous depot for sustained morphine release is reported. The prodrug is stable in human plasma and specifically activated by liver carboxylesterase enzyme 2 (CES2), supporting its safety and translational potential. This long-acting system could improve NAS care by enabling convenient dosing and early integration of nonpharmacologic strategies, ultimately reducing hospital stays and healthcare burden."

Journal • CNS Disorders • Depression • Psychiatry • Substance Abuse

Beyonttra® - neuer Ansatz bei ATTR-CM. (PubMed, MMW Fortschr Med) - No abstract available

Journal

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

Adverse events • 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

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

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

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