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

NCT06563895

/ Recruiting临床3期

A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)

Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque- like substance that accumulates in different organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall apart. One way occurs as a person ages, where the normal TTR protein can fall apart and form amyloid that may no longer be sufficiently cleared by the body. This type of ATTR is known as wild-type ATTR (ATTRwt). The other way occurs when a person inherits a defective TTR gene that causes the TTR protein to spontaneously fall apart. This form of the disease is known as variant ATTR (ATTRv) and can be detected in adults by a genetic test of their TTR gene before they age.
Amyloid build-up in the heart causes the heart wall to become thick and stiff and can result in heart failure and even death. Accumulation of TTR amyloid in the heart is known as transthyretin amyloid cardiomyopathy or ATTR-CM. Amyloid can also deposit in the nerve tissues leading to nerve problems. Accumulation of TTR in the nerves is known as transthyretin amyloid polyneuropathy or ATTR-PN.
Acoramidis is an experimental drug designed to bind tightly to TTR in the blood and stabilize its structure, so it does not form the harmful amyloid plaques that can cause damage to organs.
This study is intended to determine if treatment with acoramidis in participants with ATTRv who have not yet developed any symptoms of disease can prevent or delay the development of ATTR-CM or ATTR-PN disease. If adults with an inherited defective TTR gene are treated early before any of the symptoms of disease have developed, it may be possible to delay the onset or prevent the disease entirely.

开始日期2025-05-12

申办/合作机构

Eidos Therapeutics, Inc.

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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项与 Acoramidis 相关的文献（医药）

2025-12-01·Current heart failure reports

Etiological Treatment of Cardiac Amyloidosis: Standard of Care and Future Directions

Review

作者: Morfino, Paolo ; Emdin, Michele ; Vergaro, Giuseppe ; Chubuchna, Olena ; Aimo, Alberto ; Castiglione, Vincenzo ; Buda, Gabriele ; Ferrari Chen, Yu Fu ; Fabiani, Iacopo

Abstract:

Purpose of Review:

Cardiac amyloidosis (CA) is a condition caused by interstitial infiltration of misfolded proteins structured into amyloid fibrils. Transthyretin (ATTR) and immunoglobulin light chain (AL) amyloidosis represent the most common forms of CA. CA was traditionally perceived as a rare and incurable disease, but diagnostic and therapeutic advances have undermined the conventional paradigm.

Recent Findings:

The standard of care for ATTR-CA include agents capable of selectively stabilizing the precursor protein (e.g., tafamidis), whereas the plasma cell clone is the main target of chemotherapy for AL-CA. For long, tafamidis represented the only drug approved for patients with ATTR-CA. Recent data from ATTRibute-CM led to the approval of acoramidis, whereas patisiran received refusal based on the APOLLO-B trial. Novel CRISPR-Cas9-based drugs (i.e., NTLA-2001) hold great potential in the setting of ATTR-CA. Several hematological regimens are available to treat AL-CA. The main limit of current therapies is their inability to trigger removal of amyloid from tissues. However, the investigation of monoclonal antibodies targeting misfolded ATTR (e.g., PRX004, NI301A) or AL (e.g., birtamimab, anselamimab) has led to encouraging results.

Summary:

Various cutting-edge strategies are being tested for treatment of CA and may change the prognostic landscape of this condition in the next years.

2025-12-01·MOLECULAR BIOLOGY REPORTS

Transthyretin as a therapeutic target: the future of disease-modifying therapies for Alzheimer’s disease

Review

作者: Shaw, Swetaleena ; Aran, Khadga Raj ; Porel, Pratyush

BACKGROUND:

Alzheimer's disease (AD) is the most common neurodegenerative disease for causing memory deficits and primarily characterized by extracellular deposition of amyloid-β (Aβ) plaques, intracellular neurofibrillary tangles (NFTs), and hyperphosphorylation of tau protein, all are pathological hallmarks for AD. Transthyretin (TTR) is a highly conserved homo-tetrameric protein, primarily synthesized in liver and choroid plexus, and most importantly involved in transport of T₃-T₄ hormones and retinol.

OBJECTIVES:

This review explores the dual role of TTR, with a greater emphasis on its neuroprotective action, particularly in AD.

METHODS:

Based on the available literature, TTR's potential as a biomarker in the central nervous system (CNS), focusing its role in stabilizing Aβ aggregation and the senile plaque formation during neurodegeneration. Additionally, TTR's dual roles, in neurodegeneration and neuroprotection are studied, emphasizing its potential for improving AD diagnosis and treatment strategies.

RESULTS:

Recent research has revealed that TTR is gradually showcasing its promise in neuroprotection and neuronal viability in AD by binding with Aβ and mitigating its neurotoxic effects. Current preclinical and clinical studies also support that TTR is actively involved in maintaining the blood-brain barrier (BBB) integrity and maintain neurotransmitter balance, all of which offer significant therapeutic promise through TTR stabilizers, such as Tafamidis, Acoramidis, and Vutrisiran, highlighting their potential in AD treatment CONCLUSION: This review concludes that TTR plays bidirectional role and gaining interest as a potential biomarker, though several challenges must be addressed before it can be established a novel therapeutic target in AD management in the modern era of drug discovery.

2025-11-01·American Journal of Cardiovascular Drugs

Contemporary Oral Medication Use and Frequency in Patients with Transthyretin Amyloid Cardiomyopathy

Article

作者: Grodin, Justin L ; Hennum, Liana ; Marwah, Rohit ; Shivanna, Pruthviraj ; Allison, Melissa ; Poulsen, Steen Hvitfeldt ; Falvey, Heather ; Dasgupta, Noel ; Siddhanti, Suresh ; Shah, Keyur B ; Tamby, Jean-François ; Emdin, Michele ; Ambardekar, Amrut V

INTRODUCTION:

Transthyretin amyloid cardiomyopathy (ATTR-CM) is an increasingly recognized cause of heart failure (HF), with a higher prevalence in older patients with comorbidities requiring concomitant medical therapy. Acoramidis is a next-generation transthyretin stabilizer with near-complete protein stabilization (≥ 90%) administered orally twice daily (BID) for treatment of ATTR-CM. We report on oral medication use in patients with ATTR-CM using two complementary sources: the ATTRibute-CM trial and real-world claims data.

METHODS:

In the ATTRibute-CM study, participants with ATTR-CM were randomly assigned 2:1 to receive 800 mg of acoramidis hydrochloride or matching placebo BID for 30 months. Participants from acoramidis and placebo groups were pooled for this analysis. Baseline oral medication use was collected upon enrollment in the study. Real-world data were obtained from patients with ATTR-CM in Optum's deidentified Clinformatics Data Mart Database (Optum CDM) who met the stability criteria. Patients meeting the stability criteria had: (1) ≥ 2 years of continuous enrollment with ≥ 3-months look-back and a 12-month look-forward from index diagnosis, during the study period of 2018-2021 and (2) ≥ 28 days of continuous treatment for a given dosing frequency within the 12-month look-forward period.

RESULTS:

The ATTRibute-CM study randomly assigned 632 participants with ATTR-CM (mean [± SD] age: 77.3 [6.6] years). At entry to the study, 407 (64.4%) participants were using a medication that was administered BID, three times daily (TID), or four times daily (QID), and 392 (62.0%) participants were using at least one medication administered BID. The most frequent BID medications were apixaban, furosemide, metformin, metoprolol, and carvedilol. In ATTRibute-CM, accountability to acoramidis was high (97.1%). From a pool of 2.46 million patients with HF and cardiomyopathy identified in the Optum CDM, 12,116 patients (mean [± SD] age: 76.3 [9.4] years) met the criteria for ATTR-CM, and 5601 patients met the stability criteria. Analysis from this real-world database demonstrated that 4351 (92.1%) patients were prescribed a medication that was administered BID, TID, or QID and 4166 (88.2%) patients were prescribed at least one BID medication. The most frequent medications regardless of dosing frequency included furosemide, atorvastatin, metoprolol, apixaban, and carvedilol. The most frequent BID medications were apixaban, carvedilol, furosemide, metoprolol, and potassium chloride.

CONCLUSIONS:

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.

CLINICAL TRIAL REGISTRATION:

NCT03860935.

281

项与 Acoramidis 相关的新闻（医药）

2025-11-08

·今日头条

辉瑞豪掷100亿美元赢竞购！Q3 财报藏答案，未来稳了？

在一场堪称《权力的游戏》的生物医药竞购战中，辉瑞笑到了最后。 Metsera最终被辉瑞以每股86.25美元、总估值约100亿美元的报价，成功将生物技术公司Metsera收入囊中，为这场历时数月、波澜起伏的竞购战画上句号。背后，是辉瑞重返增长赛道、押注“下一代爆款疗法” 的雄心。 1 峰回路转的竞购战这场收购故事的剧情反转堪比电视剧。 9月，辉瑞似乎已锁定交易，然而丹麦巨头诺和诺德突然出手，以更高报价插足，让Metsera一度摇摆。市场普遍认为，凭借GLP-1药物的绝对优势，诺和诺德更有胜算。但就在上周，美国联邦贸易委员会（FTC）致信Metsera，指出诺和诺德的提案存在反垄断风险。Metsera最终选择了“风险更低、执行更稳”的辉瑞方案。最终报价：每股86.25美元、总价近百亿美元。较上周五收盘价溢价3.7%，并包含最高20.65美元的额外或有价值权。 Metsera股价随即暴涨近60%，市值突破87亿美元；而诺和诺德宣布退出竞购。这场从华尔街延烧到监管层的对决，也宣告辉瑞在肥胖症治疗领域迎来了“第二次机会”。 2 辉瑞的“减重翻盘计划” 肥胖症治疗，正在成为制药业新的金矿。据分析机构预测，到2030年代初，全球GLP-1类药物市场规模将达到1500亿美元。辉瑞早年在减肥药领域屡战屡败，如今通过Metsera看到了重回赛场的机会。 Metsera的两款核心资产——GLP-1注射剂MET-097i和胰淀素模拟物MET-233i，被认为具备潜在的同类最佳（best-in-class）潜力。Leerink Partners分析师预计，若临床顺利，二者合计峰值销售额有望超过50亿美元。在赢下竞购之前，辉瑞CEO Albert Bourla在财报会上直言：“ 辉瑞认为诺和诺德的提议是虚幻的，根据辉瑞与Metsera的合并协议条款，不能构成更优提案，因为它违反了反垄断法，并且其最终完成的风险更高。 ”“对辉瑞与Metsera合并前景的信念是坚定和毫不动摇的。”“辉瑞已经证明能够通过Eliquis、Lipitor、Norvasc和Vyndaqel系列等关键心血管品牌推动领先的临床、商业和战略势头，辉瑞计划在与Metsera合作时以类似的方式执行，以重振辉瑞的心脏代谢领域影响力。” 3 财报背后的“增长驱动” 在竞购最为激烈的时刻，辉瑞发布了的最新财报显示，其仍处于新冠影响的出清过程中。 2025年前九个月，辉瑞营收 450亿美元，同比 -2% 。主要是美国市场有所下滑（-5%），国际市场继续增长（+4%），最新推出和收购的产品营收73亿美元，同比+9%。 2025年第三季度，辉瑞营收 167亿美元，同比 -7% 。主要是由COVID产品下降驱动。下降主要来自抗新冠口服药物Paxlovid，其次是新冠疫苗Comimaty。非COVID产品表现稳健，同比+4%，主要由Eliquis、Vyndaqel系列和Nurtec驱动。但与此同时，财报也显示出一些新趋，比如在肿瘤与心代谢的双线突破。业务层面，辉瑞分为生物制药（Biopharma）、Centreone、Ignite 。生物制药（Biopharma）占据总营收的98%，分为基础护理（占比42%）、专业护理（28%）、肿瘤（27%）三大板块。具体来看，在2025年前九个月：基础护理（Primary Care）营收188.82亿美元，同比-11%，主要是受新冠药物Paxlovid营收下滑57%的影响。抗凝药物Eliquis以及肺炎球菌疫苗Prevnar family（沛儿家族）是基础护理的板块的支柱产品，前者营收 59.41亿美元，同比+7% ；后者营收 47.86亿美元，同比-1% ，主要是受美国儿科市场上政府大宗订单延迟（约占美国Prevnar收入的60%）的影响。辉瑞的下一代肺炎球菌疫苗（25价）计划在2025年启动针对成人Ⅲ期研究（需经FDA同意）。辉瑞强调其25价候选疫苗有潜力改善血清型3的免疫原性，这类人群在肺炎球菌疾病占比较高，约占美国和欧盟65岁以上人群侵袭性疾病的近20%。新冠药物Paxlovid 营收虽然下滑过半，但仍贡献了21.44亿美元的销售额；新冠疫苗Comimaty 营收增长6%至20.97亿美元。新一代偏头痛药物Nurtec ODT 继续引领口服降钙素基因相关肽（CGRP）药物市场，营收增长17%，突破10亿美元大关。呼吸道合胞病毒（RSV）疫苗Abrysvo 在2025年第三季度由于流感季，在国际市场上取得了+81%的营收增长，但由于国际市场营收基数较小，被下滑34%的美国市场带动，整体营收下滑22%。在2025年前九个月，Abrysvo营收整体下滑1%。专业护理（Specialty Care）营收127.75亿美元，同比+5%，主要是受支柱产品——罕见病创新药Vyndaqel family（Tafamidis为主，氯苯唑酸软胶囊）增长所驱动。 Vyndaqel family 是口服转甲状腺素蛋白稳定剂，用于治疗转甲状腺素蛋白淀粉样心肌病（ATTR-CM，一种罕见的心血管疾病）。营收增长20%至46.92亿元。但该产品的营收环比有所下降，主要是由于市场上的竞争加剧。 2024年11月， BridgeBio公司的转甲状腺素蛋白稳定剂Attruby（辉瑞Tafamidis同类产品）获批用于治疗ATTR-CM；2025年3月， Alnylam公司的小干扰RNA产品Amvuttra 获得美国FDA批准，成为用于治疗ATTR-CM的首款RNAi抑制剂。不过，辉瑞称，Amvuttra迄今为止推动的转换微乎其微。同时辉瑞强调Vynda仍然是唯一一个在死亡率和心血管相关住院率两方面均实现统计学显著降低的ATTR-CM产品，并且是作为单药实现的；也是唯一一个提供每日一次胶囊、安慰剂般安全性以及近乎完全的TTR稳定性的产品。辉瑞在全美范围内为Vyndamax获得了90%的准入。展望Vynda的未来，辉瑞预计其销量将继续增长，Vynda的表现符合辉瑞的预期。肿瘤（Oncology）营收123.99亿美元，同比+7%，是辉瑞生物制药板块增速最高的细分领域。乳腺癌药物Ibrance是辉瑞肿瘤业务的支柱产品，营收30.83亿美元，同比-6%。尽管辉瑞努力将其关键专利延长到2027年，但乳腺癌领域层出不穷的新药物（如ADC）加剧了市场竞争。辉瑞与安斯泰来合作研发的Xtandi 营收16.02亿美元，同比+9%。该药物在激素敏感性前列腺癌中需求增长强劲，并且在约16,000名美国高风险生化复发的非转移性激素敏感性前列腺癌患者中迅速得到应用。全球首个Nectin-4 ADC药物Padcev（通过收购Seagen获得）营收14.32亿美元，同比+25%，Padcev主要用于治疗急性淋巴细胞白血病。Padcev与帕博利珠单抗（默沙东药物）联合使用继续扩大应用范围，并已成为局部晚期转移性尿路上皮癌患者的标准护理一线治疗方法。组合疗法Braftovi+Mektovi 在第三季度营收5.19亿美元，同比增长19%。该组合自2023年10月推出以来，新患者起始治疗数量增长了30%。近期，来自PHAROS单臂二期临床试验的随访结果，支持Braftovi+Mektovi作为携带BRAF V600E突变的转移性非小细胞肺癌患者的标准护理，进一步巩固了辉瑞肺癌产品组合的实力。辉瑞2025年前九个月业务数据 4 优化继续，BD活跃利润方面，辉瑞在2025年第三季度的调整后毛利率约为76%，报告稀释每股收益为0.62美元，调整后稀释每股收益为0.87美元。该利润表现包含了约0.20美元的来自三生制药（3SBio）交易收购的在研研发项目带来的不利影响。辉瑞表示，预计到27年底，制造优化计划的第一阶段将节省15亿美元，以支持辉瑞的长期营业利润率扩张目标。展望未来，制造网络中的成本管理仍然是其重中之重。辉瑞仍然有望在今年年底前通过正在进行的成本调整计划实现至少45亿美元的累计净成本节约目标，并预计到 2027年底总共节省约77亿美元。调整后销售、一般和管理费用（SI&A）下降3%，主要得益于生产力改进推动营销和促销支出的减少。调整后研发费用（R&D）也下降3%，是由于管线聚焦和优化（包括辉瑞数字能力的扩展）导致支出净减少。 2025年第三季度，辉瑞主要有三大亮点：拟议收购Metsera。完成了对三生制药SSGJ-707（PD-1×VEGF双抗）的授权引进。辉瑞表示，在此交易之后，辉瑞的BD能力约为130亿美元。辉瑞执行副总裁、首席战略与创新官Andrew Baum坦言： “辉瑞在所有地区（BD）都非常活跃，尤其是在中国。你看到了三生制药，它增加了一个基础性资产，将成为跨多个适应症的支柱——在中国及其他地区，跨越所有主要治疗领域。我们特别扩大了辉瑞在中国的团队规模，辉瑞有非常积极的努力。” 与美国政府达成协议，消除了两个关键政策方面的不确定性，成功响应了美国政府降低处方药成本并将价格与其他发达国家看齐的呼吁，并且辉瑞承诺进一步在美国投资制造业，从而获得美国某些关税的三年宽限期。辉瑞继续预计2025年全年收入将在 610亿-640亿美元之间；预计调整后研发将在100亿-110亿美元之间；提高并收窄2025年全年调整后稀释每股收益指引，中点约提高0.08美元，至每股3美元至3.15美元。 5 从“疫后焦虑”到“结构性重生” 过去两年，辉瑞经历了从巅峰到回调的剧烈转折。随着COVID产品收入下滑、内部结构性成本高企，辉瑞一度被视为“失速的巨舰”。而这场Metsera收购，无疑是其战略重启的标志性事件——辉瑞正从“防疫药厂”，回归“创新药巨头” 。与20多年前那场改变命运的华纳兰伯特收购相似，辉瑞再次以强势姿态下注未来。不同的是，这一次，它赌的不再是降脂药的时代，而是全球都在关注的“减重经济”。辉瑞赢下的不仅是一场竞购战，更是一场关于未来的选择。面对肥胖、心代谢、罕见病、肿瘤等高增长领域的叠加机会，辉瑞正在用资本与创新的双轮驱动，为自己构建新的增长曲线。

并购财报疫苗信使RNA

2025-11-06

·拜耳中国

VICTOR研究结果中国首发，夯实维立西呱心衰全程管理证据

上海，2025年11月6日——在第八届中国国际进口博览会上，“维立西呱VICTOR研究结果中国首发仪式”在拜耳展台隆重举行。该研究于2025年欧洲心脏病学会（ESC）大会上荣耀发布，为心衰患者的规范治疗提供了坚实的循证医学证据，开启了心衰治疗的新篇章。拜耳维立西呱VICTOR研究中国重磅发布心力衰竭是心血管疾病最后的战场。尽管以“新四联”为代表的标准治疗（GDMT）极大改善了患者预后，但临床实践中患者仍面临高死亡率、高住院率的严峻挑战，存在显著的残余风险。VICTOR研究的突破性在于，它首次在“病情相对稳定”、已接受充分标准治疗的HFrEF患者中，验证了维立西呱在降低心血管死亡和全因死亡方面的获益。中国科学院院士葛均波教授中国科学院院士葛均波教授担任VICTOR研究的中国Leading PI，他表示，“VICTOR研究的结果为临床医生提供了强有力的新证据，改变了我们对稳定期心衰患者的管理策略，即推动治疗转向更加主动的全程风险管理，这最终将为患者带来显著的生存获益和生活质量提升。研究的顺利开展与成果的获得，离不开全国48家研究中心研究团队的辛勤付出和医药企业的专业支持。期待未来能与拜耳这样的创新医药企业继续深入合作，将更多突破性疗法带给中国患者，共同携手推动中国心血管疾病防治水平的提升。” 复旦大学附属中山医院周京敏教授复旦大学附属中山医院周京敏教授作为作为中国VICTOR研究组长单位的执行PI对VICTOR研究结果进行了深入解读。研究结果显示，维立西呱能够降低心血管死亡相对风险17%，并降低全因死亡相对风险16%，尤其可降低心源性猝死风险达25%，且中位获益时间仅为5.4个月，提示其早期使用维立西呱即可发挥保护作用。周京敏教授介绍，“VICTOR研究传递出一个关键信息：对于看似“稳定”的HFrEF患者，其猝死及远期死亡风险依然严峻。稳定期不应被视为治疗的‘终点’，而应成为我们主动干预、进一步改善预后的‘关键窗口期’。这提示临床医生应转变观念，对稳定期患者进行更积极、主动的干预，从而进一步优化心衰的全程管理。” 拜耳集团处方药事业部全球执行副总裁兼中国区总裁，及拜耳中国区总裁周晓兰表示：“作为一家创新的生命科学企业，拜耳始终致力于将全球领先的突破性疗法引入中国，并深度支持本土临床研究，以切实满足中国患者的健康需求。面对超过千万的中国心衰患者，VICTOR研究这一里程碑结果的发布，既是维立西呱临床价值的重要探索，更是拜耳以创新践行‘共享健康’企业使命的核心体现。‘健康中国’战略目标的实现需要汇聚各方智慧，拜耳期待在研发、诊疗、疾病教育等各个环节，与社会各界深化合作，共同构建一个以患者为中心的创新生态系统，携手迎接中国心血管事件‘拐点’的早日到来。” 拜耳集团处方药事业部高级副总裁，核心产品业务总经理杨灏介绍，“从拥有百年历史的拜阿司匹灵®，到抗高血压药物拜新同®，口服抗凝药拜瑞妥®，慢性心衰治疗药物唯可同®,到今年中美同步递交心衰适应症的糖尿病相关慢性肾脏病治疗药物可申达®，以及在研的口服XIa因子抑制剂Asundexian和治疗ATTR-CM（蛋白淀粉样变性心肌病）的创新药物Acoramidis，拜耳以强大的产品线，构建起从抗血小板、降压、抗凝、心衰治疗到心肾共护的全程心血管疾病管理体系，应对中国慢病管理的巨大挑战。与此同时，我们积极携手学会等专业学术机构，支持房颤中心、心衰中心、胸痛中心等三大核心项目建设，持续推动中国心血管诊疗规范化，将循证医学证据和指南推荐转化为可落地的临床实践。” 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2024财年，拜耳的员工人数约为93,000名，销售额为466亿欧元。不计特殊项目的研究开发投入为62亿欧元。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http://www.bayer.com/上公开的拜耳各项报告。本公司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。

突破性疗法放射疗法

2025-10-31

·EndPoints

The BridgeBio and Alnylam rivalry in ATTR-CM may not be a race at all. What gives?

The much-ballyhooed competition between BridgeBio and Alnylam has been less of a tug-of-war and more of a rising-tide-lifts-all-boats scenario. That’s because both companies are hitting the milestones they each laid out for the respective launches of their transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) treatments, and that’s likely to continue, even as other companies attempt to enter the market, analysts tell Endpoints News . For BridgeBio, the commercial success of Attruby — combined with two late-stage rare disease wins this week — underscore the company’s increasingly diverse portfolio. Alnylam, meanwhile, is headed toward its long-stated goal of becoming profitable by the end of this year. “We’re definitely, I’d say, poised to achieve this goal that we set out for ourselves back in 2021,” Alnylam CEO Yvonne Greenstreet told Endpoints News . Attruby and Alnylam’s Amvuttra, which both received approval for ATTR-CM within the last year, continue to record strong sales and quarter-over-quarter growth. Amvuttra generated $685 million in sales in the third quarter, up from $492 million in the second quarter, and Attruby pulled in $108.1 million compared to $71.5 million in Q2. But the raw sales numbers don’t tell the whole story. Amvuttra costs more than twice as much as Attruby. Amvuttra is also covered by Medicare Part B, while Attruby is covered by Part D. Part B drugs are typically more expensive because they’re administered in doctors’ offices, and clinicians have a different set of incentives to prescribe them. Attruby is also a small molecule drug, which is typically cheaper to manufacture than other drugs. (Amvuttra is an RNA silencer.) What’s really driving the growth for both drugs is a significant increase in the number of patients being diagnosed, according to TD Cowen analyst Ritu Baral. Some investors theorized that ATTR-CM was underdiagnosed. Even after the first ATTR-CM drug was approved in 2019 — Pfizer’s Vyndamax/Vyndaqel franchise — it was primarily prescribed to patients with severe symptoms. That created an opening for both drugs to record strong early sales despite near-simultaneous launches, Baral said. But investor skepticism also drove the idea there would be stiff competition, she added. The market potential “just wasn’t recognized by the investors, where it was by the doctors,” Baral said. Most analysts say the key metric to watch is related to underdiagnoses. The goal is to grow the number of “first-line” individuals on a drug rather than simply count the patients who switch from Vyndamax/Vyndaqel. That’s happening, according to William Blair analyst Myles Minter, because there are so many undiagnosed patients. “The moment that I start hearing all these companies going, ‘Hey, the majority of our patients are switches,’ then I’ll start to get worried,” he said. “Because then you’re just fighting for a smaller piece of the pie.” In a third-quarter earnings call, BridgeBio CEO Neil Kumar said his “best guess” of Attruby’s first-line market share is “well in the 20s” in terms of percentage of market share. Kumar said he couldn’t give a precise figure, because Alnylam and Pfizer hadn’t yet reported their earnings. (Pfizer is set to report on Tuesday.) Alnylam did not break down the first-line and “switch” figures in Thursday’s earnings call, but Greenstreet told Endpoints that Amvuttra is “highly competitive” in that “first-line” segment. Despite the strong sales figures for each drug, BridgeBio and Alnylam’s stock prices both dropped on Thursday after Alnylam reported its quarterly results. BridgeBio $BBIO shares closed down 4.9%, while Alnylam $ALNY fell about 6.7%. It’s unclear why this happened. More than a handful of analysts, including Cantor Fitzgerald’s Olivia Brayer, put out notes to investors saying investor expectations have become inflated, particularly for Alnylam. Some are “starting to question” how much upside is left in Alnylam’s stock, given the company likely won’t launch another drug for several years, Brayer wrote. Leerink analyst Mani Foroohar agreed, saying investors seemed to want accelerating sales growth rather than the stable, linear growth Alnylam has shown so far. “As investor expectations got really, really high, driven by technical as well as fundamental reasons, it became tougher and tougher to hit that bar,” he told Endpoints. BridgeBio is more insulated from this dynamic, he added, because the company is closer to launching other drugs. The rare disease wins this week point to at least two new drugs coming to market sometime in 2027. Baral noted that the stock drops may have been influenced by other incentives. Attruby and Amvuttra sales are intensely scrutinized every quarter when doing so may not be warranted, given the “explosive” market that’s unfolded in the last 12 months. “Everybody’s pushing their narrative, and Alnylam has frequently been a stock that has been controversial,” Baral said. “Let’s put it that way.”

上市批准生物类似药

100 项与 Acoramidis 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11972	-	-	-

研发状态

批准上市

10 条最早获批的记录,

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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	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	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
多发性神经病	临床3期	爱尔兰	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	意大利	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	遞觸廠窪鏇鹽願構廠鹹(鹹築構範範鏇蓋簾蓋醖): 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	餘膚膚範願鹹鹹鏇鏇鹹(鏇遞艱觸壓繭網顧膚網) = 積積膚憲鹽鬱範醖願構窪繭蓋夢壓憲衊繭襯夢 (鏇觸廠鹹夢範顧簾製夢 ) 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	網願壓醖遞膚淵醖鹽鑰(夢構鑰餘鏇艱顧淵蓋範) = 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). 淵積醖鬱鏇簾糧膚選衊 (範襯簾積遞衊製廠觸艱 ) 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	憲窪築糧網膚膚構醖糧(積選鏇獵窪觸願淵積醖): 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	蓋襯憲蓋願鏇醖襯遞廠(餘積獵齋蓋繭鹹鏇糧襯) = 構獵構廠蓋獵積獵糧鹹鹹糧鏇願選選膚淵廠繭 (憲襯衊遞廠觸衊鑰獵壓, 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	憲蓋選醖鏇蓋遞糧構遞(蓋範觸鑰網鏇膚窪窪選): 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	獵觸積鏇醖廠餘範憲製(廠憲窪淵觸憲衊獵遞構) = 鏇積餘襯鏇衊鬱鏇鑰願淵膚遞艱艱醖鹽網淵襯 (遞積網鬱鏇鏇鏇蓋製簾 ) 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	構鏇遞網憲遞艱鏇築蓋(艱醖衊遞簾積鏇蓋選夢) = 築膚餘廠齋膚範壓壓網築築構製衊窪遞淵憲齋 (衊製觸衊醖製鬱顧觸築 )	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	願艱範齋鑰窪鬱壓糧製(襯鑰鹹糧構願衊選網鬱) = 選築鬱廠顧鹹憲構構獵鏇製選襯選廠築夢鹹選 (廠襯衊遞觸壓簾遞衊顧 )	积极	2024-08-30
				Tafamidis	願艱範齋鑰窪鬱壓糧製(襯鑰鹹糧構願衊選網鬱) = 蓋獵壓艱憲鬱製網淵廠鏇製選襯選廠築夢鹹選 (廠襯衊遞觸壓簾遞衊顧, 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)	憲鑰醖獵餘糧選積製鹹 = 願鬱糧餘糧觸醖膚淵鑰淵糧襯餘齋製製網構艱 (繭願繭衊蓋範廠醖醖壓, 顧憲餘製膚範衊網衊構 ~ 齋遞膚範壓遞鹽築鹹醖) 更多	-	2024-06-27
				Placebo Oral Tablet (Placebo)	憲鑰醖獵餘糧選積製鹹 = 顧憲選壓蓋衊鑰鏇製觸淵糧襯餘齋製製網構艱 (繭願繭衊蓋範廠醖醖壓, 窪衊夢壓窪構衊衊糧獵 ~ 積獵衊艱繭淵範選構製) 更多