2f:I[703058,["/_next/static/immutable/chunks/3bhq_5eh1d760.js","/_next/static/immutable/chunks/1855-ogyi8eh4.js","/_next/static/immutable/chunks/2pfsjpn6k6zlg.js","/_next/static/immutable/chunks/26c7v3_3uwnwu.js","/_next/static/immutable/chunks/25a3ozcrvi1ff.js","/_next/static/immutable/chunks/2r4wx50nvjh_4.js","/_next/static/immutable/chunks/23fsw06hsi5b1.js","/_next/static/immutable/chunks/06_yt270h41rg.js","/_next/static/immutable/chunks/3ezsiz-7shkna.js","/_next/static/immutable/chunks/20x2y96ldu5wx.js","/_next/static/immutable/chunks/2lxntcm3pd1_h.js","/_next/static/immutable/chunks/00dgco8irq3jw.js","/_next/static/immutable/chunks/00nwe-kq0rboc.js","/_next/static/immutable/chunks/2051ffx3xzio9.js"],"PaperListPageClient"] 30:T425,Overall, 552 participants with wild-type ATTR-CM (mean [SD] age, 78 [6.3] years; 92.0% male and 8.0% female) and 59 participants with variant ATTR-CM (mean [SD] age, 73 [7.7] years; 77.3% male and 22.7% female) were randomized (mITT population), including 35 with p.Val142Ile. Consistent efficacy was observed in wild-type and variant subgroups for ACM/CVH through month 30 and ACM through month 42. At month 30, acoramidis reduced the risk of ACM/first CVH vs placebo by 31% in ATTRwt-CM (hazard ratio [HR], 0.69; 95% CI, 0.52-0.90; P = .007) and by 59% in ATTRv-CM (HR, 0.41; 95% CI, 0.21-0.81; P = .01). ACM was reduced through month 42 with HRs of 0.70 (95% CI, 0.50-0.98; P = .04) and 0.41 (95% CI, 0.19-0.93; P = .03) in the ATTRwt-CM and ATTRv-CM groups, respectively. Consistent treatment benefit was observed in participants with ATTRwt-CM and ATTRv-CM for secondary end points. Within variant subgroups (p.Val142Ile vs non-p.Val142Ile), consistent treatment benefits were observed for ACM/CVH through month 30 and ACM through month 42.31:Taf4,Transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive disease caused by misfolded transthyretin (TTR), occurs as wild-type (ATTRwt-CM) or variant (ATTRv-CM) forms. p.Val142Ile is the most common variant in the US, linked to rapid progression and increased mortality. Acoramidis achieves near-complete (≥90%) TTR stabilization and showed clinical benefit in the 30-month ATTRibute-CM trial and through month 42 in the ongoing open-label extension (OLE). To evaluate the efficacy of acoramidis in ATTRwt-CM, ATTRv-CM, and variant subgroups (p.Val142Ile and non-p.Val142Ile). This international, multicenter, phase 3, randomized placebo-controlled study took place from April 2019 to May 2023 with ongoing OLE (month 42). ATTRibute-CM enrolled 632 participants with ATTR-CM; 611 of 632 were included in the modified intention-to-treat (mITT) population. There were 380 participants who continued into the OLE. These data were analyzed from January 2025 to July 2025. Oral acoramidis, 712 mg, or placebo twice daily for 30 months, followed by 12 months of open-label treatment. All-cause mortality (ACM), cardiovascular-related hospitalizations (CVH), serum TTR, 6-minute walk distance, Kansas City Cardiomyopathy Questionnaire Overall Summary score, and N-terminal pro B-type natriuretic peptide in participants with ATTRwt-CM and ATTRv-CM. Post-hoc analyses were conducted in variant subgroups, including p.Val142Ile. Overall, 552 participants with wild-type ATTR-CM (mean [SD] age, 78 [6.3] years; 92.0% male and 8.0% female) and 59 participants with variant ATTR-CM (mean [SD] age, 73 [7.7] years; 77.3% male and 22.7% female) were randomized (mITT population), including 35 with p.Val142Ile. Consistent efficacy was observed in wild-type and variant subgroups for ACM/CVH through month 30 and ACM through month 42. At month 30, acoramidis reduced the risk of ACM/first CVH vs placebo by 31% in ATTRwt-CM (hazard ratio [HR], 0.69; 95% CI, 0.52-0.90; P = .007) and by 59% in ATTRv-CM (HR, 0.41; 95% CI, 0.21-0.81; P = .01). ACM was reduced through month 42 with HRs of 0.70 (95% CI, 0.50-0.98; P = .04) and 0.41 (95% CI, 0.19-0.93; P = .03) in the ATTRwt-CM and ATTRv-CM groups, respectively. Consistent treatment benefit was observed in participants with ATTRwt-CM and ATTRv-CM for secondary end points. Within variant subgroups (p.Val142Ile vs non-p.Val142Ile), consistent treatment benefits were observed for ACM/CVH through month 30 and ACM through month 42. The beneficial effect of acoramidis was observed consistently in ATTRwt-CM and ATTRv-CM groups. 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; NCT04988386.32:T1311, Importance Transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive disease caused by misfolded transthyretin (TTR), occurs as wild-type (ATTRwt-CM) or variant (ATTRv-CM) forms. p.Val142Ile is the most common variant in the US, linked to rapid progression and increased mortality. Acoramidis achieves near-complete (≥90%) TTR stabilization and showed clinical benefit in the 30-month ATTRibute-CM trial and through month 42 in the ongoing open-label extension (OLE). Objective To evaluate the efficacy of acoramidis in ATTRwt-CM, ATTRv-CM, and variant subgroups (p.Val142Ile and non-p.Val142Ile). Design, Setting, and Participants This international, multicenter, phase 3, randomized placebo-controlled study took place from April 2019 to May 2023 with ongoing OLE (month 42). ATTRibute-CM enrolled 632 participants with ATTR-CM; 611 of 632 were included in the modified intention-to-treat (mITT) population. There were 380 participants who continued into the OLE. These data were analyzed from January 2025 to July 2025. Interventions Oral acoramidis, 712 mg, or placebo twice daily for 30 months, followed by 12 months of open-label treatment. Main Outcomes and Measures All-cause mortality (ACM), cardiovascular-related hospitalizations (CVH), serum TTR, 6-minute walk distance, Kansas City Cardiomyopathy Questionnaire Overall Summary score, and N-terminal pro B-type natriuretic peptide in participants with ATTRwt-CM and ATTRv-CM. Post-hoc analyses were conducted in variant subgroups, including p.Val142Ile. Results Overall, 552 participants with wild-type ATTR-CM (mean [SD] age, 78 [6.3] years; 92.0% male and 8.0% female) and 59 participants with variant ATTR-CM (mean [SD] age, 73 [7.7] years; 77.3% male and 22.7% female) were randomized (mITT population), including 35 with p.Val142Ile. Consistent efficacy was observed in wild-type and variant subgroups for ACM/CVH through month 30 and ACM through month 42. At month 30, acoramidis reduced the risk of ACM/first CVH vs placebo by 31% in ATTRwt-CM (hazard ratio [HR], 0.69; 95% CI, 0.52-0.90; P = .007) and by 59% in ATTRv-CM (HR, 0.41; 95% CI, 0.21-0.81; P = .01). ACM was reduced through month 42 with HRs of 0.70 (95% CI, 0.50-0.98; P = .04) and 0.41 (95% CI, 0.19-0.93; P = .03) in the ATTRwt-CM and ATTRv-CM groups, respectively. Consistent treatment benefit was observed in participants with ATTRwt-CM and ATTRv-CM for secondary end points. Within variant subgroups (p.Val142Ile vs non-p.Val142Ile), consistent treatment benefits were observed for ACM/CVH through month 30 and ACM through month 42. Conclusions and Relevance The beneficial effect of acoramidis was observed consistently in ATTRwt-CM and ATTRv-CM groups. These hypothesis-generating results indicate that further studies are warranted to better characterize the therapeutic benefit of acoramidis in variant subgroups. Trial Registration ClinicalTrials.gov Identifiers: NCT03860935 ; NCT04988386 25:[[["$","script","0",{"type":"application/ld+json","dangerouslySetInnerHTML":{"__html":"{\"@context\":\"https://schema.org\",\"@type\":\"CollectionPage\",\"name\":\"Transthyretin Stabilizer: modality Research & Clinical Takeaways\",\"description\":\"Explore peer-reviewed papers on Transthyretin Stabilizer (modality). Clinician-written summaries and actionable practice insights. 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At 30 months, 47% of acoramidis recipients were \"alive and not worse\" vs 30% on placebo (NNT = 6), and 46% were \"alive and well\" vs 31% (NNT = 7).","limitations":"- This is a secondary/post hoc analysis of the trial, so some comparisons (\"alive and better\") were not pre-specified.\n- The population was predominantly older (mean age 77.2 years) and male (90.8%), limiting generalizability to women and younger patients.\n- KCCQ data were only available in surviving patients at each time point, which may introduce survivorship bias given the mortality difference between arms.","practice_takeaway":"In ATTR-CM, acoramidis not only reduces mortality and hospitalizations but also meaningfully preserves patient-reported quality of life — roughly 1 in 6 additional patients remain alive and not worse compared to placebo (NNT = 6). Clinicians can use these KCCQ data to counsel patients on expected functional benefits over a 2.5-year treatment course."}},{"doi":"10.1001/jamacardio.2026.0819","pmid":"41911489","title":"Long-Term Durability of Acoramidis Efficacy in Transthyretin Amyloid Cardiomyopathy","journal_title":"JAMA Cardiology","journal_issn":"2380-6583","published_at":"2026-03-30T00:00:00.000Z","url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC13036634/","source_score":114,"is_retracted":false,"is_open_access":true,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-05-18T13:25:18.543Z","updated_at":"2026-05-18T13:25:18.543Z","tags":[{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"age_group","value":"older_adult","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"brand","value":"Attruby","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"category","value":"Cardiac & Cardiovascular Systems","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"condition","value":"Transthyretin Amyloid Cardiomyopathy","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"generic","value":"acoramidis","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"impact_flag","value":"practice_changing","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"level_of_evidence","value":"2","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"modality","value":"Transthyretin Stabilizer","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"sample_size","value":"n=632","value_numeric":632},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"sex","value":"mixed","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"study_design","value":"Open-Label Extension of Randomized Controlled Trial","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"warning","value":"Level of evidence assigned as 2 (controlled trial with OLE extension) rather than 1, given the absence of a randomized comparator arm beyond month 30.","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"warning","value":"Only 389 of 632 original trial participants enrolled in the OLE; selective enrollment may introduce survivor bias and overestimate long-term benefit.","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"warning","value":"The full text PDF metadata appears to be from the FDA prescribing label for Attruby (acoramidis), not the full journal article — content beyond the abstract and results section is limited to label pages with no extractable body text. Summary is based solely on the abstract and results sections.","value_numeric":null},{"paper_doi":"10.1001/jamacardio.2026.0819","tag_type":"warning","value":"The OLE dose (800 mg BID) differs from the FDA-approved commercial dose (712 mg BID, as two 356 mg tablets); clinicians should use the approved labeling dose in practice.","value_numeric":null}],"summaries":{"what_studied":"This open-label extension (OLE) of the ATTRibute-CM RCT evaluated the long-term efficacy and safety of acoramidis 800 mg twice daily through month 54 in adults with ATTR-CM, comparing continuous acoramidis (n=263) vs. placebo-to-acoramidis switchers (n=126) who crossed over at month 30.","key_result":"Continuous acoramidis reduced all-cause mortality (HR 0.55; 95% CI 0.42–0.74; P<.001), cardiovascular mortality (HR 0.51; 95% CI 0.36–0.71; P<.001), and first cardiovascular hospitalization (HR 0.53; 95% CI 0.42–0.69; P<.001) through month 54, with stabilization of NT-proBNP, KCCQ-OS score, and 6MWD; late switchers showed stabilization of NT-proBNP and KCCQ-OS and improvement in sTTR and 6MWD, but did not fully recover the outcomes benefit seen with continuous treatment.","limitations":"- OLE design with no placebo arm beyond month 30 limits controlled comparison at later timepoints.\n- High dropout (389 of 632 enrolled in OLE), raising survivor bias concerns.\n- Population was predominantly older males (mean age 77.3 years, 90.2% male), limiting generalizability to women and younger patients.","practice_takeaway":"Start acoramidis early in ATTR-CM — 54-month data show sustained, incremental reductions in death and hospitalization with continuous therapy that are not fully recovered by switching later. Do not delay or interrupt treatment while awaiting disease progression."}},{"doi":"10.1001/jamacardio.2025.4477","pmid":"41205147","title":"Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy","journal_title":"JAMA Cardiology","journal_issn":"2380-6583","published_at":"2025-11-08T00:00:00.000Z","url":"https://doi.org/10.1001/jamacardio.2025.4477","source_score":103,"is_retracted":false,"is_open_access":true,"raw_metadata":{"ooir":{"day":"2026-01-16","doi":"10.1001/jamacardio.2025.4477","days":"14","issn":"2380-6583","rank":"5","field":"Clinical Medicine","score":"103","category":"Cardiac & Cardiovascular Systems","categories":["Cardiac & Cardiovascular Systems"],"_categories":["Cardiac & Cardiovascular Systems"]},"pubmed":{"doi":"10.1001/jamacardio.2025.4477","title":"Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: Results From ATTRibute-CM and Its Open-Label Extension.","journal":"JAMA cardiology","methods":"","results":"$30","abstract":"$31","keywords":[],"pubmed_id":"41205147","conclusions":"","publication_date":"2025-11-08T00:00:00"},"crossref":{"DOI":"10.1001/jamacardio.2025.4477","URL":"https://doi.org/10.1001/jamacardio.2025.4477","ISSN":["2380-6583"],"link":[{"URL":"https://jamanetwork.com/journals/jamacardiology/articlepdf/2841140/jamacardiology_alexander_2025_oi_250068_1767980815.15996.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"page":"57","type":"journal-article","issue":"1","score":1,"title":["Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy"],"author":[{"given":"Kevin M.","family":"Alexander","sequence":"first","affiliation":[{"name":"Stanford Amyloid Center, Division of Cardiovascular Medicine, Stanford University School of Medicine, Palo Alto, California"}]},{"given":"Margot K.","family":"Davis","sequence":"additional","affiliation":[{"name":"Division of Cardiology, University of British Columbia, Vancouver, British Columbia, Canada"}]},{"given":"Olakunle","family":"Akinboboye","sequence":"additional","affiliation":[{"name":"Laurelton Heart Specialists, Queens Heart Institute, Rosedale, New York, New York"}]},{"given":"John","family":"Berk","sequence":"additional","affiliation":[{"name":"Boston Medical Center, Boston University, Boston, Massachusetts"}]},{"given":"Kunal","family":"Bhatt","sequence":"additional","affiliation":[{"name":"Division of Cardiology of the Department of Medicine at Emory University, Emory Healthcare, Atlanta, Georgia"}]},{"given":"Francesco","family":"Cappelli","sequence":"additional","affiliation":[{"name":"Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence, Italy"}]},{"given":"Sarah A.M.","family":"Cuddy","sequence":"additional","affiliation":[{"name":"Heart and Vascular Center, Brigham and Women’s Hospital, Boston, Massachusetts"}]},{"given":"Marianna","family":"Fontana","sequence":"additional","affiliation":[{"name":"National Amyloidosis Centre, Division of Medicine, University College London, London, United Kingdom"}]},{"given":"Pablo","family":"Garcia-Pavia","sequence":"additional","affiliation":[{"name":"Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV, and Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid, Spain"}]},{"given":"Julian D.","family":"Gillmore","sequence":"additional","affiliation":[{"name":"Centre for Amyloidosis and Acute Phase Proteins, Division of Medicine, University College London, London, United Kingdom"}]},{"given":"Jan M.","family":"Griffin","sequence":"additional","affiliation":[{"name":"Division of Cardiology, Department of Medicine, Medical University of South Carolina, Charleston"}]},{"given":"Justin L.","family":"Grodin","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas"}]},{"given":"Daniel P.","family":"Judge","sequence":"additional","affiliation":[{"name":"Division of Cardiology, Department of Medicine, Medical University of South Carolina, Charleston"}]},{"given":"Michel G.","family":"Khouri","sequence":"additional","affiliation":[{"name":"Division of Cardiology, Department of Medicine, Duke University Health System, Durham, North Carolina"}]},{"given":"Kaitlyn","family":"Lam","sequence":"additional","affiliation":[{"name":"Western Australia Advanced Heart Failure and Cardiac Transplant Service, Fiona Stanley Hospital, Western Australia, Australia"}]},{"given":"Ahmad","family":"Masri","sequence":"additional","affiliation":[{"name":"Division of Cardiology, Oregon Health & Science University, Portland"}]},{"given":"Mathew S.","family":"Maurer","sequence":"additional","affiliation":[{"name":"Department of Medicine, Columbia University Irving Medical Center, New York, New York"}]},{"given":"Laura","family":"Obici","sequence":"additional","affiliation":[{"name":"Fondazione IRCCS Policlinico San Matteo, Pavia, Italy"}]},{"given":"Frederick L.","family":"Ruberg","sequence":"additional","affiliation":[{"name":"Boston University Chobanian & Avedisian School of Medicine, Boston Medical Center, Boston, Massachusetts"}]},{"given":"Nitasha","family":"Sarswat","sequence":"additional","affiliation":[{"name":"Division of Cardiovascular Medicine, University of Chicago Medicine, Chicago, Illinois"}]},{"given":"Keyur","family":"Shah","sequence":"additional","affiliation":[{"name":"Pauley Heart Center, Virginia Commonwealth University, Richmond"}]},{"given":"Prem","family":"Soman","sequence":"additional","affiliation":[{"name":"Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania"}]},{"given":"Lily","family":"Stern","sequence":"additional","affiliation":[{"name":"Department of Cardiology, Smidt Heart Institute, Cedars Sinai, Los Angeles, California"}]},{"given":"Richard","family":"Wright","sequence":"additional","affiliation":[{"name":"Pacific Heart Institute and Providence, St. John’s Health Center, Santa Monica, California"}]},{"given":"Kuangnan","family":"Xiong","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Xiaofan","family":"Cao","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Ted","family":"Lystig","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Jean-François","family":"Tamby","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Adam","family":"Castaño","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Leonid","family":"Katz","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Uma","family":"Sinha","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Jonathan C.","family":"Fox","sequence":"additional","affiliation":[{"name":"BridgeBio, San Francisco, California"}]},{"given":"Scott D.","family":"Solomon","sequence":"additional","affiliation":[{"name":"Heart and Vascular Center, Brigham and Women’s Hospital, Boston, Massachusetts"}]},{"given":"Martha","family":"Grogan","sequence":"additional","affiliation":[{"name":"Cardiovascular Medicine, Mayo Clinic Rochester, Minnesota"}]}],"issued":{"date-parts":[[2026,1,1]]},"member":"10","prefix":"10.1001","source":"Crossref","volume":"11","created":{"date-time":"2025-11-08T16:30:17Z","timestamp":1762619417000,"date-parts":[[2025,11,8]]},"indexed":{"date-time":"2026-01-15T02:49:23Z","timestamp":1768445363652,"date-parts":[[2026,1,15]]},"subject":[],"abstract":"$32","language":"en","relation":{},"resource":{"primary":{"URL":"https://jamanetwork.com/journals/jamacardiology/fullarticle/2841140"}},"subtitle":["Results From ATTRibute-CM and Its Open-Label Extension"],"deposited":{"date-time":"2026-01-14T16:00:54Z","timestamp":1768406454000,"date-parts":[[2026,1,14]]},"issn-type":[{"type":"print","value":"2380-6583"}],"published":{"date-parts":[[2026,1,1]]},"publisher":"American Medical Association (AMA)","reference":[{"DOI":"10.1093/eurheartj/ehab072","key":"hoi250068r1","year":"2021","issue":"16","author":"Garcia-Pavia","volume":"42","first-page":"1554","unstructured":null,"article-title":"Diagnosis and 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call to action.","journal-title":"Curr Cardiol Rep","doi-asserted-by":"crossref"},{"DOI":null,"key":"hoi250068r6","year":null,"issue":null,"author":null,"volume":null,"first-page":null,"unstructured":"Rowczenio? 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