A Prospective, Single-arm, Phase 4 Study to Evaluate the Course of Serum Transthyretin (TTR) Level With Acoramidis in Adult Patients With Variant or Wild-type Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM) Previously Treated With Tafamidis

Transthyretin (TTR) is a protein made by the liver that helps transport thyroid hormone and vitamin A in the blood. In some people, this protein breaks down and forms harmful clumps called amyloid. TTR amyloid gets deposited in the heart wall and stops it from pumping blood properly, which may lead to heart failure. The breakage in TTR protein can be age-related (wild-type ATTR-CM), or genetic (variant ATTR-CM).
The study drug, acoramidis, works by attaching itself to the TTR protein, making TTR more stable and less likely to break down and form amyloid (clumps). This helps to slow down the progression of the disease, improve heart function, and increase the TTR levels in the blood. Acoramidis is an approved treatment for wild-type or variant ATTR-CM in Europe and the United States.
Tafamidis is another drug that stabilizes TTR and prevents it from breaking down.
In this study, acoramidis will be studied in participants with ATTR--CM who were previously treated with tafamidis. The main purpose of this study is to assess the change in blood TTR levels after participants are switched from tafamidis to acoramidis. This will be studied to understand if acoramidis causes an increase in blood TTR levels beyond the levels achieved with tafamidis.
For this, the researchers will measure the change in the levels of TTR protein in participants' blood after 6 months of the treatment with acoramidis, or earlier if a participant stops the treatment before reaching that six-month mark.
All participants will continue taking tafamidis during the screening period. In the treatment period of the study, participants will take acoramidis as two tablets twice daily by mouth, for up to 6 months.
At the start of this study, the study doctors will review each participant's medical history and check their overall health. The study doctors will perform electrocardiograms (ECG), and measure blood pressure and heart rate. Researchers will also take blood and urine samples from the participants to measure levels of TTr, NT-proBNP, hs-TnT, hs-CRP, RBP4, eGFR, creatinine, cystatin-C, UACR, and TSH at the start of the study, and at various time points thereafter (during the study) to assess heart, kidney and thyroid function.
There will be a total of 9 study check-ins. Participants will visit the study site twice: at screening and at the end of treatment period. A study nurse will visit the participant's home 6 times, at the start of treatment, Weeks 1, 2, 3 and 4, then again at 3 months. The final check-in will be done by phone.
The study doctors will monitor the health of the participants regularly for any medical problems during follow-up visits. Participants will know the treatment they will receive during the study. Each participant could be in the study for about 8 months.

开始日期2026-02-13

申办/合作机构

Bayer AG

NCT07116473

/ Not yet recruiting临床3期

A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients With Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)

The AG10-504 study is an open-label extension study of acoramidis in participants with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM) or both ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN).

开始日期2025-11-01

申办/合作机构

Eidos Therapeutics, Inc.

NCT07235462

/ RecruitingN/A

ACO-REAL - A Non-interventional Study Providing Insights Into the Use of Acoramidis in Patients With ATTR Amyloidosis With Cardiomyopathy (ATTR-CM) in Routine Clinical Practice

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is a serious and life-threatening condition where a protein called transthyretin (TTR) misfolds and builds up as amyloid fibrils in the heart muscle. This buildup causes the heart to become stiff, leading to restrictive cardiomyopathy and progressive heart failure. There are two forms of ATTR-CM: a hereditary or 'variant' form (vATTR-CM) caused by a gene mutation, and a 'wild-type' form (wtATTR-CM) which is associated with aging. Because its symptoms can be similar to other heart conditions, ATTR-CM is often diagnosed late. However, recent advances in medical imaging are helping doctors to identify the disease earlier. Acoramidis is a new medication designed to treat ATTR-CM. It works by stabilizing the TTR protein, preventing it from misfolding and forming the harmful amyloid deposits. Acoramidis has been shown to be effective and safe in a major clinical trial (the ATTRibute-CM study), which led to its approval for use in both the United States and Europe. While clinical trials provide valuable information, data on how a new medicine performs in everyday clinical practice is also very important. This type of information is called real-world evidence. Currently, there is limited real-world information about the use of acoramidis. This study, called ACO-REAL, is an observational study, which means researchers will observe patients who are receiving acoramidis as part of their normal clinical care, without introducing any experimental interventions. The study will take place in approximately 20 European countries and aims to enroll up to 2,000 adults who have been diagnosed with either wild-type or variant ATTR-CM and are starting treatment with acoramidis. This includes patients who have not been treated for ATTR-CM before, as well as those who have been treated with other therapies. The main goals of the study are to understand the characteristics of patients being treated with acoramidis and to document how the treatment is used in routine medical practice. The study will also collect information on the safety of acoramidis. Furthermore, researchers will assess how the treatment affects patients' heart function, their functional capacity (such as their ability to walk), their overall health status, and their quality of life. The study will also track how often patients need to use healthcare resources like hospitals or emergency rooms. This information will help to improve the understanding and management of ATTR-CM in a real-world setting, ultimately aiming to optimize care for patients with this progressive disease.

开始日期2025-10-29

申办/合作机构

Bayer AG

100 项与 Acoramidis 相关的临床结果

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100 项与 Acoramidis 相关的转化医学

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100 项与 Acoramidis 相关的专利（医药）

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项与 Acoramidis 相关的文献（医药）

2026-06-01·CURRENT THERAPEUTIC RESEARCH-CLINICAL AND EXPERIMENTAL

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

Review

作者: Owens, Katie ; Argon, Sophie ; Yu, Jingjing ; Wang, Yan ; Ragueneau-Majlessi, Isabelle

Objective:

This analysis aimed to provide a mechanistic understanding and an evaluation of the clinical relevance of pharmacokinetic drug-drug interactions (DDIs) associated with drugs approved by the Food and Drug Administration in 2024.

Methods:

Drug metabolism, transport, and absorption-based DDI data available in New Drug Applications (NDAs) of small molecular drugs approved (n = 34) was analyzed. The mechanism and clinical outcome of these interactions were characterized based on in vitro, in silico, and clinical data.

Results:

As objects, 11 drugs were identified as clinical substrates. 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. All clinical DDIs with AUC changes ≥ 2-fold triggered labeling recommendations. 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.

Conclusions:

Mechanistic DDI insights from newly approved therapies can be extrapolated to inform the management of commonly co-administered drugs, supporting a safer and more effective use of new drug products in the context of polypharmacy.

2026-05-01·INTERNATIONAL JOURNAL OF CARDIOLOGY

Hidden overlap between heart failure with preserved ejection fraction and transthyretin amyloid cardiomyopathy: Why it matters and how to avoid missing it

Review

作者: Tanaka, Hidekazu

Transthyretin cardiac amyloidosis (ATTR-CM) is increasingly recognized as a treatable cause of heart failure, while heart failure with preserved ejection fraction (HFpEF) has become one of the most common and heterogeneous heart failure syndromes worldwide. Growing evidence shows that a substantial proportion of patients diagnosed with HFpEF actually harbor unrecognized ATTR-CM. Missing this diagnosis may limit the effectiveness of HFpEF therapies and delay initiation of disease-modifying treatment. Screening studies consistently demonstrate that ATTR-CM represents an important subset of HFpEF, particularly in individuals with increased left ventricular wall thickness, elevated natriuretic peptides, or unexplained conduction abnormalities. Although HFpEF and ATTR-CM share significant clinical overlap, several reproducible red-flags such as discordance between electrocardiographic voltages and wall thickness, pseudo infarct patterns, carpal tunnel syndrome, progressive troponin elevation, and apical sparing on strain imaging provide important early diagnostic clues. These features, together with emerging scoring tools, help identify patients at increased risk. In addition, bone scintigraphy has transformed the diagnostic pathway by enabling noninvasive confirmation of ATTR-CM in the absence of monoclonal protein. As disease-modifying agents such as tafamidis, acoramidis, and vutrisiran become widely available, early recognition is increasingly essential. This review synthesizes epidemiologic data on the prevalence of ATTR-CM among HFpEF cohorts, highlights key diagnostic pitfalls, and outlines practical strategies to facilitate timely detection. The aim is to support clinicians managing HFpEF by proposing a pragmatic, red-flag based screening framework that reduces missed diagnoses and enables appropriate initiation of disease-modifying therapy.

2026-04-01·CURRENT PROBLEMS IN CARDIOLOGY

Impact of disease-modifying therapies on imaging parameters in cardiac amyloidosis: A systematic review and meta-analysis

Review

作者: Zervas, Georgios ; Briasoulis, Alexandros ; Androulakis, Emmanuil ; Patras, Raphael ; Georgiopoulos, Georgios ; Tepetes, Nikolaos ; Kastritis, Efstathios ; Lama, Niki ; Kourek, Christos ; Stamatelopoulos, Kimon ; Theodorakakou, Foteini

Transthyretin-mediated (ATTR) and immunoglobulin light-chain (AL) cardiac amyloidosis causes progressive myocardial dysfunction and poor prognosis. Disease-specific therapies in ATTR and anti-clonal therapies in AL cardiac amyloidosis may modify disease, but treatment-induced changes in prognostic cardiac imaging markers remain incompletely defined. We systematically searched PubMed/MEDLINE, Scopus, CINAHL, Web of Science, and EMBASE for studies reporting treatment-associated changes in global longitudinal strain (GLS), left ventricular (LV) wall thickness, and extracellular volume (ECV) assessed by echocardiography or cardiac magnetic resonance. Random-effects meta-analyses pooled mean changes. Seventeen studies met inclusion criteria. In ATTR cardiac amyloidosis, therapy with silencers or RNA-depleting agents was associated with improved GLS (pooled mean difference [MD] -0.97 %, 95 % CI -1.27 to -0.68) and reduced LV wall thickness (MD -0.72 mm, 95 % CI -1.26 to -0.17) versus no therapy, while ECV showed no significant change (MD -1.93 %, 95 % CI -15.27 to 11.41). In AL amyloidosis, complete hematologic response was linked to greater improvement than non-response in GLS (MD -1.19 %, 95 % CI -2.20 to -0.17) and LV wall thickness (MD -0.87 mm, 95 % CI -1.31 to -0.43). 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.

329

项与 Acoramidis 相关的新闻（医药）

2026-04-01

·拜耳中国

加速增长：拜耳处方药史上最强的产品组合和前景广阔的研发管线

五大增长催化剂助力拜耳处方药打造史上最强大的产品组合； 2025年创下纪录，获得五项首次获批（三项新产品获批、两项新适应症获批），以及六项积极III期临床试验结果；预计2026年将在重点治疗领域取得多项关键研发管线里程碑；拜耳处方药确认双重目标：从2027年起恢复中个位数增长，并从2028年起实现营业利润率提升，到2030年达到30%。柏林，2026年4月1日——在拜耳处方药2026年全球媒体日活动上，拜耳集团管理委员会成员、拜耳处方药事业部全球总裁Stefan Oelrich阐述了公司如何通过持续专注于科学和业务优先，推动实现2030年业务增长目标。 Stefan Oelrich 拜耳集团管理委员会成员、拜耳处方药事业部全球总裁 “凭借坚定的业务准则和严谨的科学态度，我们正在见证变革性战略带来的回报，它正推动着当下和未来的增长。得益于史上最强大的处方药产品组合、多元化的研发管线以及日益人工智能化的运营模式，我们有望从2027年起恢复中个位数增长，并从2028年开始扩大利润率，到2030年达到30%。” 关键治疗领域的五大增长催化剂推动拜耳处方药未来十年的业绩增长 Christine Roth 拜耳处方药执行副总裁、全球产品战略与商业化负责人兼领导团队成员 “我们的目标是提供first-in-class或best-in-class产品，满足患者需求。过去几年，我们做出的大胆选择正在赋能团队，加速创新，最终将为患者和公司带来实实在在的影响。” ASUNDEXIAN（在研）向上滑动阅览最新进展：拜耳正在对一款活化凝血因子XI（XIa）抑制剂进行临床开发，即asundexian。在非心源性缺血性卒中或高风险短暂性脑缺血发作（TIA）患者中，在接受抗血小板联合治疗基础上， asundexian在减少缺血性卒中复发方面优于安慰剂。 Asundexian已被美国食品药品监督管理局（FDA）授予快速通道资格。凝血因子XIa是凝血途径中的一种蛋白质，抑制凝血因子XIa的目的，是将止血与血栓形成进行解耦，从而在保留正常止血功能的同时，防止病理性血栓的形成。1，2，3 迄今，asundexian尚未被任何国家的监管机构批准用于任何适应症治疗。重要性：全球每年约有1200万人发生卒中4，其中80%为缺血性卒中5。尽管当前已有不少卒中二级预防的治疗措施，仍有五分之一的卒中幸存者将在五年内再次发生卒中。6 达罗他胺/诺倍戈Ⓡ 向上滑动阅览最新进展：达罗他胺是首个与雄激素受体抑制剂（ADT）联合使用用于治疗转移性激素敏感性前列腺癌（mHSPC）的雄激素受体抑制剂（ARi），无论患者是否接受化疗。拜耳的目标是成为第二代雄激素受体抑制剂的全球领导者。一项III期临床试验正在评估在激素敏感性前列腺癌（HSPC）治疗中，达罗他胺联合ADT对比单独使用ADT对激素敏感性高风险生化复发（BCR）前列腺癌患者的疗效。这些患者经传统影像学检查未证实疾病转移，而基线PSMA PET/CT结果为阳性。达罗他胺具有独特的分子结构，能够通过抑制雄激素受体功能，抑制前列腺癌细胞的生长。重要性：前列腺癌是男性第二常见癌症7。2022年，全球约有40万男性死于该疾病。8 非奈利酮/可申达Ⓡ 向上滑动阅览最新进展：非奈利酮是一种选择性非甾体类盐皮质激素受体拮抗剂（nsMRA），也是首个在五项关键性的III期研究中显示出心血管和/或肾脏获益的靶向盐皮质激素受体（MR）的药物，这些研究针对的是：左心室射血分数（LVEF）≥40%的心力衰竭患者 2型糖尿病相关的慢性肾脏病（CKD）患者 1型糖尿病相关的慢性肾脏病患者以及非糖尿病慢性肾脏病患者基于其心血管和肾脏获益，我们预期非奈利酮将成为综合治疗方案中的重要支柱。拜耳非奈利酮在全球100多个国家获批用于治疗2型糖尿病相关的慢性肾脏病成人患者，在中国的商品名为可申达Ⓡ。在美国和、日本和欧盟，非奈利酮也已获批用于治疗LVEF≥40%的心力衰竭（HF），该适应症的上市申请目前在中国等多个市场接受审评。非奈利酮的临床项目包括十项III期研究，分别针对心力衰竭和慢性肾脏病，其中五项已完成。非奈利酮是一种选择性非甾体类盐皮质激素受体拮抗剂，具有独特的作用机制，可靶向心血管和肾脏病中的炎症和纤维化病理过程。重要性：全球有超过8.75亿人患有慢性肾脏病和/或心力衰竭9，10，由于全球生活方式趋势、人口老龄化和糖尿病发病率上升，同时罹患这两种疾病的风险正不断攀升9，11，12，13，14。无论是慢性肾脏病还是心力衰竭患者，均面临较低的生存率。当这两种疾病同时存在，住院、疾病进展和死亡的风险会进一步增加。15 ACORAMIDIS / BEYONTTRA™ 向上滑动阅览最新进展： Beyonttra旨在通过模拟转甲状腺素蛋白（TTR）基因中一种天然存在的“保护性突变”（T119M），有效稳定TTR四聚体，从而用于治疗转甲状腺素蛋白淀粉样变性心肌病（ATTR-CM）的根本原因。拜耳致力于在欧洲快速且持续地推广Beyonttra，用于治疗转甲状腺素蛋白淀粉样变性心肌病。该药已在欧盟获批用于治疗野生型或变异型转甲状腺素蛋白淀粉样变性心肌病的成年患者。16 重要性：转甲状腺素蛋白淀粉样变性心肌病是一种常被漏诊的进行性、潜在致命的心脏病，由转甲状腺素蛋白不稳定引起。这种不稳定会导致淀粉样蛋白沉积在心脏中，从而严重损害心脏功能17，18。据估计，转甲状腺素淀粉样变性心肌病正在影响全球40万人19，其中欧洲约有19万人。20 ELINZANENTANT / LYNKUET™ 向上滑动阅览最新进展： Elinzanetant是全球首个双重神经激肽靶向疗法（NK-1和NK-3受体拮抗剂）。 Elinzanetant在美国和其他市场以LynkuetTM的商品名获批用于治疗绝经相关中重度血管舒缩症状（VMS），在欧盟获批用于治疗与绝经相关的或乳腺癌辅助内分泌治疗（AET）引起的中重度血管舒缩症状，使其成为欧盟该适应症中唯一获批的治疗选择。不断增加的证据表明，雌激素活性下降会导致表达NK-1和NK-3受体的kisspeptin、神经激肽B和强啡肽（KNDy）过度活跃，从而导致调节体温和睡眠的中枢功能紊乱，最终引发VMS。 Elinzanetant通过拮抗NK-1和NK-3受体发挥作用，不仅能够降低血管舒缩症状的发生频率和严重程度，还能在改善睡眠障碍和提升生活质量方面带来额外获益。重要性：预计到2030年，全球绝经期女性人数将增至12亿，而这一生理阶段正值女性社交和职业活跃期21。2020年，全球新增乳腺癌病例230万例，其中近70%为激素受体阳性肿瘤，患者需要接受抗雌激素治疗，而这可能会引发绝经期症状。22，23，24，25 重塑研发管线，助力未来增长 Christian Rommel 博士拜耳处方药执行副总裁、全球研发负责人兼领导团队成员 “我们通过创新和差异化产品重塑研发管线，这一战略正在释放潜力：2025年，三个新产品获批，重磅产品的两项新适应症获批，六项III期临床试验取得积极结果，创下业绩纪录。2026年，我们预计将在心血管和肿瘤等核心治疗领域的精准治疗、再生细胞和基因疗法，以及分子影像等多个领域取得关键里程碑，进一步验证以创新驱动影响的战略。” 精准药物研发进展 225Ac-PSMA-Trillium (BAY 3563254) 向上滑动阅览最新进展： 225Ac-PSMA-Trillium（BAY 3563254）是一种在研靶向α疗法（TAT），用于治疗晚期转移性去势抵抗性前列腺癌（mCRPC）患者。正在进行的全球I期首次人体剂量递增PAnTHa研究取得了令人振奋的结果，支持将225Ac-PSMA-Trillium推进到下一阶段的临床开发。靶向α疗法是拜耳精准肿瘤学战略支柱，有望加速为转移性去势抵抗性前列腺癌患者的新型治疗方案的开发进程。重要性：前列腺癌是男性第二常见癌症7，其中转移性去势抵抗性前列腺癌患者中位生存期小于三年。26，27 Vividion Therapeutics总裁兼首席执行官Aleksandra Rizo博士表示： “ 小分子药物凭借其可及性、可预测性和全球可扩展性，仍然是提供具有深远影响疗法的最有效途径之一。我们正利用化学蛋白质组学筛选平台，针对以往被认为无法成药的驱动基因，推进癌症和免疫疾病口服疗法的研发。随着我们自主研发的三种分子进入临床开发阶段，我们正致力于将研究成果转化为有望改变患者命运的新药。 ” WRN抑制剂（VVD-214）向上滑动阅览最新进展： WRN抑制剂（VVD-214）是一种新型的共价、不可逆的Werner解旋酶（WRN）抑制剂，旨在利用微卫星不稳定性高表达（MSI-High）的癌细胞对WRN介导的DNA修复机制的特异性依赖。这种癌症特异性机制有望在降低毒性和副作用的情况下控制疾病进展并提高患者生存率。它是全球首个进入临床试验的WRN共价抑制剂。在一项I期临床试验中，VVD-214耐受性良好，并在微卫星不稳定性高表达的实体瘤患者中显示出令人鼓舞的活性。拜耳计划继续开展Ib期临床试验，并启动II期临床试验，以评估VVD-214在晚期微卫星不稳定性高表达（MSI-High）或错配修复缺陷型（dMMR）结直肠癌患者中的有效性。重要性：结直肠癌（CRC）是全球第三大常见癌症，每年新增病例近200万28。约15%的结直肠癌为MSI-High，这构成了规模最大的MSI-High患者人群29，也为靶向治疗提供了巨大的机会。VVD-214在其他MSI-High癌症中也具有潜在的治疗价值，包括子宫内膜癌、卵巢癌和胃癌。 GIRK4抑制剂（BAY 3670549）最新进展： GIRK4抑制剂（BAY 3670549）是一种高选择性G蛋白偶联内向整流钾通道4（GIRK4）抑制剂，有望帮助控制房颤（AFib）患者心脏细胞的电活动。该GIRK4抑制剂源于与美国麻省理工学院和哈佛大学博德研究所的战略研究联盟。 I期临床试验已启动。重要性：房颤是最常见的心律失常（心律不齐）类型，正在影响全球超过6000万人，也是卒中和心力衰竭的重要危险因素。30，31，32 塞伐艾替尼向上滑动阅览最新进展：塞伐艾替尼是一种新型口服、可逆性的小分子酪氨酸激酶抑制剂（TKI），可阻断参与癌细胞生长的特定酪氨酸激酶活性。塞伐艾替尼已在美国和中国获得突破性疗法认定，用于携带人表皮生长因子受体2（HER2）激活突变的非小细胞肺癌（NSCLC）成人患者的一线治疗。在美国和加拿大，塞伐艾替尼以商品名HyrnuoTM获批用于既往接受过治疗的晚期HER2突变非小细胞肺癌患者。重要性：非小细胞肺癌是最常见的肺癌类型，占所有肺癌病例的85%以上33，其中2-4%为HER2突变非小细胞肺癌。34，35 基因疗法和细胞疗法产品组合有望为患者带来变革性影响基因疗法 AskBio首席执行官Gustavo Pesquin表示： “ 通过将 AskBio 的腺相关病毒一体化平台（从衣壳创新到生产）与拜耳的全球开发和运营能力相结合，我们正在努力开发有望改变罕见病和常见病患者生活的药物。 ” AB-1002 最新进展： AB-1002 是一种单次给药的在研基因疗法，目前正被开发用于治疗射血分数降低的心力衰竭（HFrEF）患者。 II期临床研究的最后一名受试者已完成随机分组。重要性：据估计，全球约有6400万人患有心力衰竭。尽管疗法不断优化，但死亡率和发病率仍然很高。36，37 Ametefgene parvec (AB-1005) 最新进展： Ametefgene parvec是一种单次给药的在研基因疗法，旨在针对帕金森病（PD）的潜在生物学机制，从而恢复神经元功能，并有可能延缓疾病进展。进行中的II期临床试验正在美国、德国、波兰和英国的多个临床中心招募受试者。重要性：过去25年间，帕金森病的患病率翻了一番。目前全球约有近1200万人罹患帕金森病。现有治疗方案的疗效通常会在数年后逐渐减退，患者随后会出现运动波动及治疗相关性运动障碍。38，39 细胞疗法 BlueRock Therapeutics公司总裁兼首席执行官Seth Ettenberg表示： “ 对于以不可逆细胞损失为特征的疾病，再生医学提供了一种根本性的全新治疗方法。我们正在推进新型细胞疗法进入临床开发阶段，同时实现可规模化、可重复的制造。 ” Bemdaneprocel 最新进展： Bemdaneprocel是一种单次给药的在研细胞疗法，旨在替代帕金森病中丢失的多巴胺能神经元。 Bemdaneprocel目前正在美国、加拿大和澳大利亚的多个临床中心进行III期临床试验。重要性：过去25年间，帕金森病的患病率翻了一番。目前全球约有近1200万人罹患帕金森病。现有治疗方案的疗效通常会在数年后逐渐减退，患者随后会出现运动波动及治疗相关性运动障碍。38，39 OpCT-001 最新进展： OpCT-001是一种研究性诱导多能干细胞（iPSC）衍生的感光细胞疗法，用于治疗原发性感光细胞疾病。 OpCT-001已获得美国FDA快速通道资格认定，目前正在进行I/IIa期临床试验。美国FDA已授予OpCT-001治疗视网膜色素变性的孤儿药资格认定。重要性：原发性感光细胞疾病是遗传性视网膜疾病的一个亚群，会导致儿童和成人不可逆的视力丧失。目前针对原发性感光细胞疾病的治疗选择非常有限。仅在美国，受该疾病影响的人数预估为11万人。40，41，42 推动医学影像领域的创新拜耳影像诊断业务全球研发负责人Konstanze Diefenbach博士表示： “ 医学影像正在迅速发展，变得更加个性化，并越来越深入地融入患者的就医流程。在拜耳，我们正在引领推动这一领域的创新——从低剂量对比剂、更智能的互联工作流，到新的分子方法。通过实现更早、更准确的诊断，我们帮助临床医生选择精确的治疗方案，改善患者护理。 ” Gadoquatrane 向上滑动阅览最新进展： Gadoquatrane是一种新一代高弛豫率、低剂量的大环状钆基MRI对比剂，用于检测和显示成人及儿童（包括新生儿）全身各部位及中枢神经系统的病变。 Gadoquatrane在所有大环状MRI对比剂中钆剂量最低，与目前市售其他制剂相比，每次检查可减少高达 60%的钆剂量，同时仍能保持图像质量。拜耳于2026年3月宣布，该产品在日本以 Ambelvist™ 为商品名获全球首次批准上市。目前拜耳已向包括中国等在内的多个国家和地区的监管机构递交了gadoquatrane的上市申请。重要性： MRI对比剂有助于疾病的诊断、治疗决策以及多种疾病的随访监测。低剂量MRI对比剂之所以重要，是因为可以降低患者一生中的钆暴露量。各国监管机构和医学学会一贯建议，在满足诊断目的的前提下使用尽可能低的钆剂量。这一原则适用于所有患者，尤其是那些需要多次进行MRI增强检查的患者（如癌症等慢性疾病患者）、肾功能受损患者以及儿童。据估计，2024年全球使用的钆基对比剂剂量超过6000万剂次。43 分子示踪剂AT-01和 AT-05 向上滑动阅览最新进展： AT-01 和 AT-05 是在研的非侵入性泛淀粉样分子影像示踪剂，专门用于以高敏感性和高特异性检测淀粉样蛋白沉积，从而支持对心脏淀粉样变性及其他类型系统性淀粉样变性的准确诊断。通过进军诊断示踪剂领域，拜耳进一步彰显其在分子影像领域持续拓展的雄心，同时巩固其在心血管精准医疗领域的领先地位。 AT-01（124-Iodine-evuzamitide）是一种 PET（正电子发射断层显像）示踪剂，是首个用于心脏淀粉样变性的淀粉样蛋白沉积影像剂，已获得美国FDA授予的“突破性疗法”认定，并在美国和欧盟均获得“孤儿药”资格，目前其Ⅲ期临床研究已完成给药。 AT-05（99mTc-p5+14）是一种SPECT（单光子发射计算机断层显像扫描）示踪剂，目前处于I期临床试验阶段，有望拓宽诊断选择。重要性：随着针对既往治疗手段不足的疾病不断出现新疗法，在分子水平上对疾病进行精准检测和监测变得愈发重要。AT-01和AT-05的研发有望满足对系统性淀粉样变性，尤其是心脏淀粉样变性，进行早期且准确诊断的迫切临床需求。心脏淀粉样变性是一种目前明显诊断不足且常常致命的心脏疾病，据估计在全球范围内影响超过40万名患者。44，45 通过将内部人工智能与战略合作伙伴关系相结合，提升研发生产力拜耳处方药研发数据科学与人工智能高级副总裁兼负责人Sai Jasti表示： “ 我们的目标是到2030年利用人工智能将研发生产力提高40%。通过将人工智能平台架构与匿名的以患者为中心的数据相结合，数据科学家能够最大化内部生物制剂产品组合，验证人工智能模型，拓展解决方案，最终加快药物发现进程。 ” 拜耳已与美国范德比尔特大学医学中心、芬兰FinnGen以及新加坡PRECISE等组织建立战略合作伙伴关系，利用结合了匿名化数据和人工智能驱动分析的全球生态系统，加快心血管和肾脏病等关键领域的药物研发进程。拜耳公司近期也与Cradle公司签署了合作协议。Cradle公司的平台旨在压缩研发周期，并以更优的速度和精度，推动更高质量的分子进入临床开发阶段。在整个价值链中，人工智能正日益成为拜耳运营中不可或缺的一部分。从全球产品和市场的端到端规划，到新一代智能体工具套件，人工智能正在重塑拜耳员工的工作方式。向上滑动阅览参考文献 Fredenburgh JC, Weitz JI. 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Open Heart. 2021:8(2):e001755. doi: 10.1136/openhrt-2021-001755. Jain A, Zahra F. Transthyretin amyloid cardiomyopathy (ATTR-CM). ClinicalTrials.gov. Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy (ATTRibute-CM). 2024. Available at: https://clinicaltrials.gov/study/NCT03860935. Last accessed: March 2026. https://www.ema.europa.eu/en/documents/orphan-maintenance-report/beyonttra-orphan-designation-withdrawal-assessment-report-initial-authorisation_en.pdf Hill K. The demography of menopause. Maturitas. 1996;23(2):113-27. World Health Organization. Breast cancer. 2025. Available at: https://www.who.int/news-room/fact-sheets/detail/breast-cancer. Last accessed: March 2026. Selli C, et al. Accurate prediction of response to endocrine therapy in breast cancer patients: current and future biomarkers. Breast Cancer Research. 2016;18:118. doi: 10.1186/s13058-016-0779-0. Burstein HJ, et al. Adjuvant Endocrine Therapy for Women With Hormone Receptor-Positive Breast Cancer: ASCO Clinical Practice Guideline Focused Update. Journal of Clinical Oncology. 2019;37(5):423-38. Cucciniello L, et al. Estrogen deprivation effects of endocrine therapy in breast cancer patients: Incidence, management and outcome. Cancer Treatment Reviews. 2023;120:102624. Freedland SJ, et al. Real-world treatment patterns and overall survival among men with Metastatic Castration-Resistant Prostate Cancer (mCRPC) in the US Medicare population. Prostate Cancer Prostatic Dis. 2024;27(2):327-33. Verry C, et al. Pattern of Clinical Progression Until Metastatic Castration-Resistant Prostate Cancer: An Epidemiological Study from the European Prostate Cancer Registry. Target Oncol. 2022;17(4):441-51. Sung H, Ferlay J, Siegel RL, Laversanne M, Soerjomataram I, Jemal A, Bray F. Global Cancer Statistics 2020: GLOBOCAN Estimates of Incidence and Mortality Worldwide for 36 Cancers in 185 Countries. CA Cancer J Clin. 2021;71(3):209-249. doi: 10.3322/caac.21660. Vilar E, Gruber SB. Microsatellite instability in colorectal cancer-the stable evidence. Nat Rev Clin Oncol. 2010;7(3):153-62. Linz D, et al. Atrial fibrillation: epidemiology, screening and digital health. The Lancet Regional Health – Europe. 2024;37(100786). Volume 37, 100786. American Heart Association. Why Atrial Fibrillation Matters. 2025. Available at: https://www.heart.org/en/health-topics/atrial-fibrillation/why-atrial-fibrillation-af-or-afib-matters. Last accessed: March 2026. Kornej J, et al. Atrial fibrillation: global burdens and global opportunities. Heart. 2021;107:516-18. Molina JR, et al. Non–Small Cell Lung Cancer: Epidemiology, Risk Factors, Treatment, and Survivorship. Mayo Clin Proc. 2008;83(5):584-94. Yu X, et al. HER2-Altered Non-Small Cell Lung Cancer: Biology, Clinicopathologic Features, and Emerging Therapies. Front Oncol. 2022;12-860313. doi: 10.3389/fonc.2022.860313. Stephens P, et al. Lung cancer: intragenic ERBB2 kinase mutations in tumours. Nature. 2004;431(7008):525-26. Savarese G, et al. Global burden of heart failure: a comprehensive and updated review of epidemiology. Cardiovasc Res. 2023;118(17):3272-87. Myhre PL, et al. Digital tools in heart failure: addressing unmet needs. Lancet Digit Health. 2024;6(10):e755-e766. Mayo Clinic. Parkinson’s disease. 2024. Available at: https://www.mayoclinic.org/diseases-conditions/parkinsons-disease/diagnosis-treatment/drc-20376062. Last accessed: March 2026. Luo Y, et al. Global, regional, national epidemiology and trends of Parkinson's disease from 1990 to 2021: findings from the Global Burden of Disease Study 2021. Front Aging Neurosci. 2025;16:1498756. doi: 10.3389/fnagi.2024.1498756. National Institute on Deafness and Other Communications Disorders. Usher Syndrome. 2017. Available at: https://www.nidcd.nih.gov/health/usher-syndrome#b. Last accessed: March 2026. Hamel CP. Cone rod dystrophies. Available at: https://pmc.ncbi.nlm.nih.gov/articles/PMC1808442/. Last accessed: March 2026. O’Neal TB, et al. Retinitis Pigmentosa. StatPearls. 2024. Available at: https://www.ncbi.nlm.nih.gov/books/NBK519518/. Last accessed: March 2026. Endrikat, J et al. Rationales for Non-standard GBCA Dosing—Low?—High?—When? and Why?: A Literature-based Study. Investigative Radiology. 2025. doi: 10.1097/RLI.0000000000001259. Kim M, et al. Comparative Outcomes of a Transthyretin Amyloid Cardiomyopathy Cohort Versus Patients With Heart Failure With Preserved Ejection Fraction Enrolled in the TOPCAT Trial. J Am Heart Assoc. 2023;12(15):e029705. https://doi.org/10.1161/JAHA.123.029705. ClinicalTrials.gov. Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy (ATTRibute-CM). Available at: https://clinicaltrials.gov/study/NCT03860935. Last accessed: March 2026. 关于拜耳拜耳作为一家跨国企业，在生命科学领域的医疗健康与农业方面具有核心竞争力。秉承“共享健康，消除饥饿”的使命，公司致力于通过产品和服务，帮助人们克服全球人口不断增长和老龄化带来的重大挑战，造福人类和地球繁荣发展。拜耳致力于推动可持续发展并对业务产生积极影响。同时，集团还通过科技创新和业务增长来提升盈利能力并创造价值。在全球，拜耳品牌代表着可信、可靠及优质。在2025财年，拜耳的员工人数约为88,000名，销售额为456亿欧元。不计特殊项目的研究开发投入为58亿欧元。更多信息请见www.bayer.com。前瞻性声明本新闻稿包括拜耳集团管理层基于当前设想和预测所作的前瞻性声明。各种已知和未知的风险、不确定性和其它因素均可能导致公司未来的实际运营结果、财务状况、发展或业绩与上述前瞻性表述中所作出的估计产生重大差异。这些因素包括在拜耳官方网站www.bayer.com上公开的拜耳各项报告中。拜耳没有责任更新这些前瞻性声明或使其符合未来发生的事件或发展。拜耳是一家在全球范围内运营子公司的控股公司。本文中所提及的“拜耳”或“公司”可能根据具体语境指一个或多个子公司。

2026-03-31

·allsci.com

BridgeBio’s acoramidis shows sustained mortality reduction in 54-month ATTR-CM extension study

BridgeBio Pharma (Nasdaq: BBIO) reported 54-month open-label extension data for acoramidis (Attruby) in ATTR-CM associated with a 44.7% reduction in all-cause mortality and 49.3% reduction in cardiovascular mortality versus patients who had received placebo before crossing over to active treatment, with both results reaching p<0.0001. The ATTRibute-CM open-label extension is an active, unblinded follow-on study enrolling adults with transthyretin amyloid cardiomyopathy who completed the parent randomized controlled trial, comparing outcomes in patients who received continuous acoramidis against those who transitioned from placebo. At Month 54, the mortality separation between the continuous-treatment and placebo-crossover groups was statistically robust on both endpoints. Acoramidis also attenuated the rise in NT-proBNP — a marker of cardiac wall stress and a surrogate for disease progression — to a degree the company characterized as exceeding what has been observed with other disease-modifying treatments in ATTR-CM. Heart failure-related quality of life, measured by the Kansas City Cardiomyopathy Questionnaire Overall Summary score, was stabilized and maintained through Month 54 in patients who received early and continuous treatment. A secondary analysis showed that an early increase in serum transthyretin at Day 28 was associated with slower KCCQ-OS decline at Month 30, suggesting the pharmacodynamic response predicts longer-term functional benefit. While continuous acoramidis treatment was associated with statistically significant reductions in all-cause and cardiovascular mortality versus patients who crossed over from placebo, because the extension was open-label and non-randomized, the findings should be interpreted as supportive long-term durability data rather than evidence of superiority versus other approved ATTR-CM therapies. The safety profile remained consistent with the parent trial. Diarrhea occurred in 11.6% of acoramidis-treated patients versus 7.6% on placebo, and upper abdominal pain in 5.5% versus 1.4%; both were predominantly mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between groups (9.3% versus 8.5%). Developed by BridgeBio Pharma and licensed from Stanford University, acoramidis is a second-generation oral transthyretin (TTR) stabilizer approved in late 2024 (USA) and early 2025 (EU, Japan) for transthyretin amyloid cardiomyopathy (ATTR-CM). Designed to mimic a protective genetic variant, it offers near-complete stabilization (around 90%) of the TTR protein, a figure that distinguishes it mechanistically from tafamidis (Vyndaqel/Vyndamax), the incumbent standard of care, which achieves approximately 60% stabilization in vitro. Whether that pharmacological difference translates into a clinically meaningful outcome advantage over tafamidis has not been established in a direct head-to-head trial; cross-trial comparisons are limited by differences in patient populations, trial design, and follow-up duration. A comparative imaging study of tafamidis versus acoramidis is currently recruiting, and a Phase IV switch study is listed as not yet recruiting. The competitive landscape in ATTR-CM has shifted since acoramidis received FDA approval in November 2024. Alnylam’s vutrisiran (Amvuttra), an RNA interference therapy that silences hepatic TTR production rather than stabilizing the protein, received FDA approval for ATTR-CM in March 2025, introducing a mechanistically distinct option with quarterly subcutaneous dosing, following a Phase III trial in ATTR-CM. The entry of an RNAi agent into the same patient population creates a two-axis competitive dynamic: acoramidis competes with tafamidis on potency within the oral stabilizer class, and with vutrisiran on route of administration and mechanism. A real-world survey presented alongside the OLE data at the American College of Cardiology meeting found that physicians were unsatisfied with treatment in more than half of patients currently prescribed therapy, and that more than one-third of patients received no treatment at all — with oral therapy the most preferred format among patients. The OLE data were presented as a late-breaking oral at the ACC Annual Scientific Sessions and simultaneously published in JAMA Cardiology, lending the readout additional visibility in the cardiology community. The open-label, non-randomized design of the extension study introduces limitations that are standard for this type of analysis: the comparator group is patients who started treatment later rather than a concurrent control arm, and unblinding may influence both patient behavior and clinical management decisions. The patient population size for the OLE was not disclosed in the press release. BridgeBio stated that additional Attruby data are planned for future medical meetings. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

临床结果临床3期上市批准

2026-03-24

·EndPoints

UK biotech co-founded by Nobel laureate secures $86M for antibody program

A British biotech based in Cambridge, England, raised $86 million to advance an antibody drug designed to deplete protein deposits in people with amyloidosis. The company, called Immutrin, was co-founded by Gregory Winter. He won the 2018 Nobel Prize in Chemistry along with two other scientists for pioneering a lab technique known as “phage display” to develop new proteins, including therapeutic antibodies. Academics Mark Pepys and Daniel Christ were also involved in founding Immutrin. The biotech’s approach is largely based on Pepys’ work, but Winter was “instrumental in terms of precisely engineering the antibody to the specifications that we needed,” Immutrin CEO Mihriban Tuna told Endpoints News. That unnamed antibody is now in preclinical development for a form of amyloidosis called ATTR cardiomyopathy, which affects the heart. It targets the disease in a “very unique” way compared to available treatments like Pfizer’s Vyndaqel and BridgeBio’s Attruby, Tuna said. Vyndaqel and Attruby stop the disease getting worse by preventing the production of further amyloid fibrils. By contrast, Immutrin’s candidate is designed to selectively bind to fibrils that have already formed in the heart, removing them by inducing a targeted immune response. “It actually offers the possibility for reversing disease, rather than simply slowing down progression,” Tuna said. The two approaches could even be used in combination, she said. Immutrin is working to start its proof-of-concept trial “as soon as possible.” While the biotech is currently focused on ATTR cardiomyopathy, it could also address other forms of amyloidosis in the future, including rarer types for which there are no approved treatments, Tuna said. Frazier Life Sciences led the Series A funding round, and F-Prime Capital, Qiming Venture Partners and SR One also invested. The company’s founding investors, Cambridge Innovation Capital and Cambridge Enterprise Ventures, chipped in as well.

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适应症	国家/地区	公司	日期
遗传性转甲状腺素蛋白淀粉样变性	欧盟	Bayer AG	2025-02-10
遗传性转甲状腺素蛋白淀粉样变性	冰岛	Bayer AG	2025-02-10
遗传性转甲状腺素蛋白淀粉样变性	列支敦士登	Bayer AG	2025-02-10
遗传性转甲状腺素蛋白淀粉样变性	挪威	Bayer AG	2025-02-10
转甲状腺素蛋白淀粉样变性心肌病	美国	BridgeBio Pharma, Inc.	2024-11-22

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适应症	最高研发状态	国家/地区	公司	日期
多发性神经病	临床3期	美国	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	日本	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	阿根廷	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	澳大利亚	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	比利时	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	巴西	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	加拿大	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	丹麦	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	法国	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	德国	Eidos Therapeutics, Inc.	2025-05-12

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03860935 (ATTRibute-CM OLE, GlobeNewswire) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	286	acoramidis	選鹹壓淵廠壓窪顧壓膚(鹽遞願鹹齋膚膚壓簾繭): Difference (%) = -44 更多	积极	2025-08-30
				Placebo
NCT03860935 (ATTRibute-CM, Pubmed \| Am J Cardiovasc Drugs)	临床3期	转甲状腺素蛋白淀粉样变性心肌病	632	Acoramidis hydrochloride	廠鏇觸糧範醖糧艱糧遞(顧簾蓋鏇衊夢廠壓廠獵) = 齋壓鏇夢構衊鏇壓簾淵淵鹽鏇窪鹹夢遞醖顧窪 (襯鑰鹹衊顧衊廠衊齋簾 ) 更多	积极	2025-08-01
NCT03860935 (ATTRibute-CM, Pubmed \| J Am Coll Cardiol)	临床3期	转甲状腺素蛋白淀粉样变性心肌病 sTTR levels	557	Acoramidis	糧獵齋顧顧衊選積築積(網簾蓋鑰製艱築鑰廠窪) = was associated with reduced ACM in univariate analysis (HR: 0.96 per 1 mg/dL increase in early ΔTTR; 95% CI: 0.93-0.98; P = 0.002). In the multivariate analysis, after adjusting for TTR variant status, baseline New York Heart Association functional class, baseline National Amyloidosis Centre stage, and baseline sTTR level, early ΔTTR remained independently associated with reduced ACM (P < 0.001). 餘醖構蓋壓夢齋觸蓋鬱 (襯廠夢夢夢鹽觸夢淵積 ) 更多	积极	2025-05-01
NCT03860935 (ATTRibute-CM, Pubmed \| J Am Coll Cardiol)	临床3期	转甲状腺素蛋白淀粉样变性心肌病 \| 老年性心脏淀粉样变性 N-terminal pro-B-type natriuretic peptide level	632	Acoramidis hydrochloride 800 mg twice daily	觸網遞鹽餘壓衊憲範鏇(襯壓觸願鬱憲簾鏇壓構): relative risk ratio = 50 (95% CI, 0.36 ~ 0.7), P-Value = < 0.0001 更多	积极	2025-03-01
				Placebo
NCT04622046 (CTgov)	临床3期	转甲状腺素蛋白淀粉样变性心肌病	25	ALXN2060	淵餘製糧齋糧餘淵築鬱(衊壓繭鬱蓋醖獵醖顧繭) = 廠鑰憲餘衊淵餘選鹽膚醖蓋築積淵網夢衊糧願 (鑰醖觸夢餘襯廠憲齋顧, 9.182) 更多	-	2024-12-16
NCT03860935 (FDA_CDER) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	611	ATTRUBY 712 mgATTRUBY 712 mg	廠顧遞鏇膚齋衊糧範糧(衊艱廠艱淵鹽艱艱願鹹): P-Value = 0.018 更多	积极	2024-11-22
				Placebo
NCT03860935 (ATTRibute-CM, Company_Website) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	389	Acoramidis	繭夢選餘衊蓋艱鹽齋醖(鏇蓋糧遞膚獵鹽顧範醖) = 製齋鑰夢願鏇齋襯憲憲繭顧廠築餘艱餘醖蓋網 (窪製願襯廠夢選廠鹽鹹 ) 更多	积极	2024-11-18
				Placebo	-
NCT03860935 (ATTRibute-CM, GlobeNewswire \| PipelineReview) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	-	Acoramidis	繭醖廠膚願製夢觸衊網(願衊鹽範遞製醖遞膚襯) = 憲壓餘憲膚餘製構構憲築醖醖鏇醖壓廠淵構餘 (鏇網壓醖獵醖鑰糧憲積 )	积极	2024-09-27
				Placebo	-
NCT03860935 (ATTRibute-CM, ESC2024)	临床3期	转甲状腺素蛋白淀粉样变性心肌病 serum TTR levels	611	Acoramidis	願積築廠餘窪遞鹽襯積(選憲餘憲淵鏇築壓範觸) = 鹽獵衊範膚廠醖構衊襯獵齋築築鬱糧餘範鬱範 (窪繭醖選構窪鑰壓鏇衊 )	积极	2024-08-30
				Tafamidis	願積築廠餘窪遞鹽襯積(選憲餘憲淵鏇築壓範觸) = 選膚遞醖構憲衊願獵簾獵齋築築鬱糧餘範鬱範 (窪繭醖選構窪鑰壓鏇衊, 0.4 (4.92))
NCT03860935 (ATTRibute-CM OLE \| ATTRibute-CM, CTgov)	临床3期	转甲状腺素蛋白淀粉样变性心肌病 \| 老年性心脏淀粉样变性	632	acoramidis (Acoramidis HCl 800 mg)	觸積齋遞膚襯願醖製鏇 = 製構選製範鏇窪製夢願淵構製構膚鬱鹹遞鹹遞 (鑰遞選艱窪範鏇膚艱鏇, 繭獵艱顧鬱鹽蓋淵範鹽 ~ 醖繭繭製蓋觸蓋築艱選) 更多	-	2024-06-27
				Placebo Oral Tablet (Placebo)	觸積齋遞膚襯願醖製鏇 = 獵獵願遞願繭積夢鬱醖淵構製構膚鬱鹹遞鹹遞 (鑰遞選艱窪範鏇膚艱鏇, 膚鹽構艱糧選鬱構簾艱 ~ 選鹽積鑰獵餘餘憲築鹹) 更多