An Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

开始日期2022-09-19

申办/合作机构

Eidos Therapeutics, Inc.

ISRCTN11628974 / Completed临床1期

A multiple dose study to evaluate the pharmacokinetics of an acoramidis (AG10) modified release tablet formulation in healthy subjects

开始日期2022-08-30

申办/合作机构

Eidos Therapeutics, Inc.

NCT04988386 / Active, not recruiting临床3期

An Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants With Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

开始日期2021-10-27

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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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）

2024-01-11·The New England journal of medicine

Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy.

Article

作者: Julian D Gillmore ; Daniel P Judge ; Francesco Cappelli ; Marianna Fontana ; Pablo Garcia-Pavia ; Simon Gibbs ; Martha Grogan ; Mazen Hanna ; James Hoffman ; Ahmad Masri ; Mathew S Maurer ; Jose Nativi-Nicolau ; Laura Obici ; Steen Hvitfeldt Poulsen ; Frank Rockhold ; Keyur B Shah ; Prem Soman ; Jyotsna Garg ; Karen Chiswell ; Haolin Xu ; Xiaofan Cao ; Ted Lystig ; Uma Sinha ; Jonathan C Fox ; ATTRibute-CM Investigators

BACKGROUND:

Transthyretin amyloid cardiomyopathy is characterized by the deposition of misfolded monomeric transthyretin (TTR) in the heart. Acoramidis is a high-affinity TTR stabilizer that acts to inhibit dissociation of tetrameric TTR and leads to more than 90% stabilization across the dosing interval as measured ex vivo.

METHODS:

In this phase 3, double-blind trial, we randomly assigned patients with transthyretin amyloid cardiomyopathy in a 2:1 ratio to receive acoramidis hydrochloride at a dose of 800 mg twice daily or matching placebo for 30 months. Efficacy was assessed in the patients who had an estimated glomerular filtration rate of at least 30 ml per minute per 1.73 m² of body-surface area. The four-step primary hierarchical analysis included death from any cause, cardiovascular-related hospitalization, the change from baseline in the N-terminal pro-B-type natriuretic peptide (NT-proBNP) level, and the change from baseline in the 6-minute walk distance. We used the Finkelstein-Schoenfeld method to compare all potential pairs of patients within strata to generate a P value. Key secondary outcomes were death from any cause, the 6-minute walk distance, the score on the Kansas City Cardiomyopathy Questionnaire-Overall Summary, and the serum TTR level.

RESULTS:

A total of 632 patients underwent randomization. The primary analysis favored acoramidis over placebo (P<0.001); the corresponding win ratio was 1.8 (95% confidence interval [CI], 1.4 to 2.2), with 63.7% of pairwise comparisons favoring acoramidis and 35.9% favoring placebo. Together, death from any cause and cardiovascular-related hospitalization contributed more than half the wins and losses to the win ratio (58% of all pairwise comparisons); NT-proBNP pairwise comparisons yielded the highest ratio of wins to losses (23.3% vs. 7.0%). The overall incidence of adverse events was similar in the acoramidis group and the placebo group (98.1% and 97.6%, respectively); serious adverse events were reported in 54.6% and 64.9% of the patients.

CONCLUSIONS:

In patients with transthyretin amyloid cardiomyopathy, the receipt of acoramidis resulted in a significantly better four-step primary hierarchical outcome containing components of mortality, morbidity, and function than placebo. Adverse events were similar in the two groups. (Funded by BridgeBio Pharma; ATTRibute-CM ClinicalTrials.gov number, NCT03860935.).

2023-11-06·CPT: pharmacometrics & systems pharmacology

Quantitative modeling of approved and emerging therapeutics for modifying TTR levels in patients with Transthyretin Amyloid Cardiomyopathy.

Article

作者: Kimiko McGirr ; Saheli Sarkar ; Kalyanasundaram Subramanian

Transthyretin amyloidosis is a rare yet lethal disease caused by an increase in the destabilization of transthyretin tetramer into monomers, leading to amyloid fibril aggregates in tissues. Multiple therapeutics have been developed to limit or halt disease progression by altering tetramer kinetics. Small molecules, like Tafamidis and AG10, stabilize the tetrameric structure while genetic therapies, like Patisiran and NTLA-002, limit the production of TTR protein by silencing genetic expression. Both of these interventions slow the accumulation of fibrils by intervening at different points in the tetramer-monomer-fibril pathway. We developed a mathematical model to compare the pharmacological efficacies of these modalities by comparing each drug's ability to reduce the rate of tetramer to monomer formation, or "tetrameric flux." The model was trained on in vitro tetramer data as well as clinical measurements of tetramer concentration in humans. Overall, genetic silencers reduced tetrameric flux more than small molecule stabilizers. Properties that led to an improvement in small molecule stabilizer function and potential benefit of gene therapy - small molecule combination were explored. This study exemplifies how modeling can be used to compare modalities with differing mechanisms of action.

2023-10-12·ESC heart failure

Biochemical and biophysical properties of a rare TTRA81V mutation causing mild transthyretin amyloid cardiomyopathy.

Article

作者: Qiuyan Sha ; Yanli Zhang ; Mengdie Wang ; Jialu Sun ; Yunlong Zhang ; Xinxin Zhang ; Ning Wang ; Yu Liu ; Ying Liu

AIMS:

We conducted a presentation on an 84-year-old male patient who has been diagnosed with TTRA81V (p. TTRA101V) hereditary transthyretin cardiac amyloidosis (hATTR-CM). In order to establish its pathogenicity, we extensively investigated the biochemical and biophysical properties of the condition.

METHODS AND RESULTS:

Transthyretin amyloid cardiomyopathy (ATTR-CM) is an increasingly acknowledged progressive infiltrative cardiomyopathy that leads to heart failure and potentially fatal arrhythmias. Gaining a comprehensive understanding of the biochemical and biophysical characteristics of genetically mutated TTR proteins serves as the fundamental cornerstone for delivering precise medical care to individuals affected by ATTR. Laboratory assessments indicated a brain natriuretic peptide of 200.12 ng/L (normal range: 0-100 ng/L) and high-sensitivity cardiac troponin I of 0.189 μg/L (normal range: 0-0.1 μg/L). Echocardiography identified left atrial enlargement, symmetrical left ventricular hypertrophy (16 mm septal and 16 mm posterior wall), and a left ventricular ejection fraction of 56%. Cardiac-enhanced magnetic resonance imaging revealed subendocardial late gadolinium enhancement. Tc-99m-PYP nuclear scintigraphy confirmed grade 3 myocardial uptake, showing an increased heart-to-contralateral ratio (H/CL = 2.33). Genetic testing revealed a heterozygous missense mutation in the TTR gene (c.302C>T), resulting in an alanine-to-valine residue change (p. Ala81Val, following the first 20 residues of signal sequence nomenclature). Biochemical analysis of this variant displayed compromised kinetic stability in both the TTRA81V:WT (wild-type) heterozygote protein (half-life, t_1/2 = 21 h) and the TTRA81V homozygote protein (t_1/2 = 17.5 h). The kinetic stability fell between that of the TTRWT (t_1/2 = 42 h) and the early-onset TTRL55P mutation (t_1/2 = 4.4 h), indicating the patient's late-onset condition. Kinetic stabilizers (Tafamidis, Diflunisal, and AG10) all exhibited the capacity to inhibit TTRA81V acid- and mechanical force-induced fibril formation, albeit less effectively than with TTRWT. Chromatographic assessment of the patient's serum TTR tetramers indicated a slightly lower concentration (3.0 μM) before oral administration of Tafamidis compared with the normal range (3.6-7.2 μM).

CONCLUSIONS:

We identified a patient with hATTR-CM who possesses a rare TTRA81V mutation solely associated with cardiac complications. The slightly reduced kinetic stability of this mutation indicates its late-onset nature and contributes to the gradual progression of the disease.

110

项与 Acoramidis 相关的新闻（医药）

2024-04-07

·GlobeNewswire-Health Care

BridgeBio Pharma Presents Cardiac Magnetic Resonance (CMR) Imaging Evidence Consistent with Clinical Improvement Observed in the ATTRibute-CM Phase 3 Study in Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

- In this exploratory substudy, treatment with acoramidis was associated with possible cardiac structural and functional improvement compared with placebo, with potential cardiac amyloid regression - The data demonstrate that targeting near-complete transthyretin (TTR) stabilization with acoramidis may enable cardiac remodeling and functional recovery in patients with ATTR-CM - These results are the first prospective, longitudinal evaluation of cardiac structure and function by CMR imaging in a double-blind, placebo-controlled, interventional study in ATTR-CM - The findings from this study build upon positive results from BridgeBio’s global ATTRibute-CM Phase 3 trial, wherein the primary endpoint was met (Win Ratio of 1.8) with a high statistical significance (p<0.0001); the substudy data are consistent with the cardiovascular clinical benefits observed with acoramidis - Acoramidis was well-tolerated, with no safety signals of potential clinical concern observed - BridgeBio’s New Drug Application (NDA) has been accepted by the U.S. Food and Drug Administration (FDA) with a PDUFA action date of November 29, 2024; a Marketing Authorization Application (MAA) for acoramidis has been accepted by the European Medicines Agency (EMA), with an expected decision in 2025 PALO ALTO, Calif., April 07, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today presented results from the exploratory CMR imaging substudy of ATTRibute-CM, its Phase 3 trial of acoramidis in ATTR-CM. These data were presented at the American College of Cardiology (ACC) Annual Scientific Sessions & Expo in a moderated poster session by Yousef Razvi, M.D. of University College London. ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of TTR. Based on the positive results from ATTRibute-CM, BridgeBio submitted an NDA to the U.S. FDA, which has been accepted with a PDUFA action date of November 29, 2024, and an MAA to the European Medicines Agency, with a decision expected in 2025. “CMR is the reference non-invasive imaging method to evaluate cardiac structure, function, and amyloid burden in patients with ATTR-CM. The results shown in the imaging substudy are indicative of potential improvement of cardiac structure and function in patients with ATTR-CM, consistent with the clinical outcomes with acoramidis treatment observed in the ATTRibute-CM Phase 3 clinical trial. The impact on cardiac amyloid load highlights the potential that acoramidis could lead to cardiac amyloid regression in a proportion of patients with ATTR-CM, which we have not observed in such a controlled clinical trial to date,” said Marianna Fontana, M.D., Ph.D., Professor of Cardiology and Honorary Consultant Cardiologist at the National Amyloidosis Centre, Division of Medicine, University College London, and Principal Investigator of the Substudy. CMR imaging was performed at baseline before the first dose in 35 participants or within three months after the first dose in 17 participants (range, 14-105 days); subsequent CMR imaging was performed at months 12, 24, and 30 in those individuals who were available to undergo imaging, which was influenced by survival. Images were read centrally at the National Amyloidosis Centre in a fashion blinded to other clinical data. Findings included: Key CMR imaging parameters measuring cardiac structure, including mean indexed left ventricular mass, were found to be stable or have a trend towards improvement on acoramidis vs. deteriorated on placebo over 30 monthsTreatment with acoramidis preserved or was associated with a trend towards improvement in measures of cardiac function including left ventricular ejection fraction and stroke volume compared to initial measures, and relative to placebo In July 2023, BridgeBio announced positive results from ATTRibute-CM, reporting a highly statistically significant result, demonstrated by a Win Ratio of 1.8 (p<0.0001) on the primary endpoint (a hierarchical analysis prioritizing in order: all-cause mortality, then frequency of cardiovascular hospitalization, then change from baseline in N-terminal prohormone of brain natriuretic peptide, then change from baseline in 6-minute walk distance). Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified. BridgeBio has also presented analyses from ATTRibute-CM at the European Society of Cardiology Congress 2023 and at the American Heart Association Scientific Sessions 2023. In February 2024, BridgeBio shared positive results of a single-arm Phase 3 study of acoramidis in Japanese patients with ATTR-CM, including no mortality observed in the trial at 30 months. About BridgeBio Pharma, Inc.BridgeBio Pharma Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter. BridgeBio Pharma, Inc. Forward-Looking StatementsThis press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “continue,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to the clinical and therapeutic potential of our programs and product candidates, including our clinical development program for acoramidis for patients with transthyretin amyloid cardiomyopathy, the timing and success of our clinical development programs, the progress of our ongoing and planned clinical trials of acoramidis for patients with transthyretin amyloid cardiomyopathy, including the expectations of receiving a PDUFA action from the FDA on November 29, 2024 and receiving a decision from the EMA on the MAA for acoramidis in 2025, the expected intellectual property protection of acoramidis, our planned interactions with regulatory authorities, the statements regarding the potential clinical benefits or of potential benefits for ATTR-CM patients in the quotes of Dr. Fontana, and the timing of these events, reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations, or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from our clinical trials not being indicative of final data, the design and success of ongoing and planned clinical trials, difficulties with enrollment in our clinical trials, adverse events that may be encountered in our clinical trials, the FDA or other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, the impacts of current macroeconomic and geopolitical events, including changing conditions from the COVID-19 pandemic, hostilities in the Middle East and Ukraine, increasing rates of inflation and rising interest rates, on our overall business operations and expectations, as well as those risks set forth in the Risk Factors section of our Annual Report on Form 10-K for the year ended December 31, 2023 and our other filings with the U.S. Securities and Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of our management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. BridgeBio Media Contact:Vikram Balicontact@bridgebio.com (650)-789-8220

临床3期临床结果申请上市AHA会议

2024-03-27

·Pharma Manufacturing

Bayer to let go of 90 workers at NJ site

Bayer will be letting go of 90 employees at its Whippany, New Jersey site, the company revealed this week in a Worker Adjustment and Retraining Notification (WARN). Bayer opened its U.S. headquarters on the 94-acre campus in Whippany back in 2013. The headquarters consolidated Bayer HealthCare’s operations that had previously been spread across Morristown, Montville, and Wayne, New Jersey, as well as Tarrytown, New York, into one campus. The layoff news comes just a few months after the drugmaker announced a significant workforce reduction at its German sites. At the time, the company also unveiled a new global operating model, "Dynamic Shared Ownership," which has the aim of “improving agility and operational efficiency by reducing hierarchies, eliminating bureaucracy, and expediting decision-making processes.” Earlier this month, Bayer secured exclusive rights to commercialize BridgeBio's promising heart disease drug, acoramidis, in Europe, as the drug awaits approval from regulators. Acoramidis, an experimental treatment for the rare and deadly heart condition ATTR-CM, could significantly impact heart failure outcomes. Bayer's deal with BridgeBio includes payments up to $310 million, covering initial, milestone payments, plus royalties.

上市批准引进/卖出

2024-03-26

·医药魔方

拜耳处方药全球总裁Stefan Oelrich：多维度赋能，加速医疗突破成为现实

近些年，随着抗体偶联、mRNA、基因编辑等新技术日臻成熟，ADC药物、CAR-T疗法、基因疗法、小核酸药物等新兴疗法不断涌现，它们或是填补了罕见病无药可用的临床治疗缺口，亦或是革新了疾病治疗格局，甚至是为患者创造了治愈机会......这一个又一个变革性技术突破正在让“不可能”成为“可能”。3月21日，拜耳以“未来已来：实现医疗创新突破”为主题，开启了2024年处方药全球媒体日，来自拜耳处方药事业部的高层管理人员、各领域专家以及患者代表齐聚德国柏林，他们重点探讨了医疗领域的最新趋势，拜耳在女性健康、肿瘤学、细胞和基因疗法（CGT）等领域的前沿创新、面临的挑战与机遇，以及如何将所取得的医疗突破成功转化为现实。本年度媒体日，拜耳积极传达了其实现下一个医疗突破的雄心壮志，同时也向外界宣告正在进入下一轮创新阶段，未来将借力前沿科技，持续专注创新疗法的开发，为目前尚无治疗手段的患者带来更加精准和友好的治疗方法。会议召开前夕，拜耳处方药全球总裁Stefan Oelrich接受了医药魔方的专访，就拜耳处方药业务的转型进展、长期发展战略等多个问题进行了讨论。拜耳处方药全球总裁Stefan Oelrich挖掘新的增长动力，夯实“100亿”规模业务底座3月5日，拜耳发布了2023年财报，集团总收入476.37亿欧元，其中处方药收入同比持平，销售额达180.81亿欧元。这份增涨有所“停滞”的成绩单，不免让外界对其处方药未来增长产生担忧。Stefan对此直言不讳：“即将失去拜瑞妥专利权确实会对拜耳处方药业务产生一定影响，不过这是可以克服和应对的。另外，我想补充，一款药物专利权到期能够对整体业务产生那么大的影响，也从侧面反映出这款产品非常成功。”为实现业务可持续性增长，拜耳已经前瞻性规划好发展路径，主要分为三个部分。首先是培育新的重磅药物，对诺倍戈、可申达和阿柏西普8mg这类已经上市的药物进行长生命周期管理，充分挖掘潜在价值以延长生命周期；而多款后期产品elinzanetant、asundexian、acoramidis等，则需要快速推进商业化落地，兑现临床价值。他强调，这些产品是拜耳100亿欧元规模体量的基础性业务，也是其处方药构成中稳定的业务底座。诺倍戈是继多菲戈后，拜耳推出的另一款前列腺癌相关的治疗药物。作为新型雄激素受体抑制剂（ARi），诺倍戈的AR亲和力更高，独特的结构赋予其更强的抗肿瘤活性和更高的安全性能。临床研究显示，经治患者的前列腺特异性抗原（PSA）达标率高达71%。凭借出色的疗效，诺倍戈上市以来销售业绩一路攀升，2023年几乎实现了翻番，达到了8.69亿欧元。至于心血管领域，拜耳倾力打造的可申达得益于其在慢性肾病伴2型糖尿病早期患者中的治疗优势，也展现出惊艳的市场表现力，2023年取得了2.7亿欧元销售成绩（+152.3%）。阿柏西普8mg的上市是拜耳成功探索成熟产品全生命周期管理的优秀范本，也使得阿柏西普重焕新生。数据显示，阿柏西普8mg疗效不亚于2mg版本，且实现了每5个月一次的更低用药频率。上市不到半年时间，阿柏西普 8mg便在美国地区创收1.66亿美元，预期成为下一代抗VEGF治疗的标准疗法。在成熟产品不断兑现商业价值的同时，拜耳也聚焦存在诸多未尽之需的医疗领域，为患者带来更多的治疗选择。例如，预计2025年上市的绝经相关中重度潮热治疗药物elinzanetant。绝经期是每位女性都会面临的阶段，据统计到2030年全球绝经期女性人口将增至12亿，每年约新增4700万人。诸多女性在绝经过渡期间会出现不同的症状，种类多达48种，其中潮热、睡眠障碍和情绪变化最为常见。尽管市面上有传统的激素疗法，但很多女性因为各种原因会本能抵触使用激素，最终错失治疗机会，导致生活质量严重受损。“女性健康已经融入拜耳DNA，”Stefan介绍道，“百年来，拜耳为女性带来了譬如长期、短期避孕方法，以及绝经管理和妇科疾病疗法。因此，我们有义务将创新带入女性健康领域。”针对心血管重点细分领域存在的疾病负担，拜耳也期待利用其丰富的专长焕新病人的治疗效果和体验。以转甲状腺素蛋白淀粉样变性心肌病（ATTR-CM）为例，这是一种罕见的心肌疾病，若不加以有效控制会演变成心衰，甚至有致死后果。BridgeBio的acoramidis是全球备受关注的一款口服强效ATTR-CM药物，已在III期临床研究中达到所有临床终点，其于EMA的上市申请已经递交。看中这款药物潜力的拜耳，在今年3月果断出手，引进了acoramidis欧洲独家商业化权益，收获期指日可待。“其次是进一步补充和推动研发管线，确保持续有足够分量的创新成果产出，”Stefan解释了战略第二部分：“拜耳将重点关注项目的质量和效率，目前管线推进速度非常快。去年，我们提交了8项新药临床试验申请。此外，4款具有first-in-class潜力的候选药物预计在2024年底前进入II期临床试验。”当然，能够从如此庞大的创新资源规模中快速挑选出优先级，并且同步推进多款产品临床开发，背后也体现了拜耳决策层面的高质量和高效率。组织架构调整是Stefan提到的战略第三部分，“为了进一步优化组织效率和生产效率，我们在2023年推出了新运营模式Dynamic Shared Ownership（DSO），希望资源真正放在应该被高度关注的领域。”全链条赋能，加速医疗突破成为现实让医疗突破成为现实，这不仅限于表态，拜耳正在用实际行动将这一设想落实到新药开发全链条。Stefan直言，研发战略调整后，拜耳拔高了科学技术应用的标准，科学与技术的融合中全新的创新之道顺势而生，目前研发管线涵盖了丰富的新治疗模式和方法。具体而言，为了加速医疗转化为现实应用，拜耳首先引进人工智能（AI）、蛋白质组学等技术手段加速药物发现与开发，攻克不可成药靶点难题；其次是融入细胞和基因疗法平台、放射性疗法、肿瘤免疫学，储备强大的创新力量；然后是改善肿瘤早期诊断，尽早为患者提供精准治疗。由AI驱动的创新药物开发趋势正以势不可挡的浪潮席卷整个医药行业，这是因为AI能够通过处理海量的数据集，挖掘出人类无法观测到的现象，进而改变靶点发现和药物设计的方式，极大提升创新药研发效率和成功率。今年3月中旬，拜耳与Aignostics开展战略合作，目标就是将AI机器学习与生物、化学、临床数据深度融合，创建新靶点识别平台，同时借力AI将基线病理数据与临床数据连接起来，以便合理设置临床试验患者分组，加快赋能更加精准的肿瘤治疗方案开发进程。拜耳对AI技术的认可已经直观体现在其频繁的投资动作上。除了Aignostics，拜耳此前已经先后与Cyclica、Exscientia、Genpact、Schrödinger、Sensyne、Recursion等知名AI药物发现公司达成了合作。此外，为了赋予不可成药靶点全新的生命力，拜耳在2021年以20亿美元总价收购了专注于高难度靶点开发的Vividion公司。“前期我们已经发现了一些靶点，但一直以来囿于技术难题，无法透视它们的价值。现在有了新技术的支持，我们可以将原有资产充分利用起来了。”Stefan对不可成药靶点的成功开发充满了期待。得益于Vividion化学蛋白质组学技术的助力，那些曾经“躺”在候选库的传统意义上“不可成药”靶点具备了更大的成功开发可能性。据Stefan介绍，过去半年中，Vividion管线中已经有两个项目推进到了临床开发阶段，分别是靶向Keap1的激活剂BAY 3605349和靶向STAT3的抑制剂BAY 3630914。创新存在多个层次，越前沿，越是会伴随极高的风险，但随之而来的是高额回报。如此密集的推进全新靶点以及不可成药靶点的研发，拜耳显然走上了更高层次的创新梯队，这也进一步撬动了拜耳精准肿瘤学更多的发展维度，“我们正努力扩充针对癌症真正弱点的可成药靶点库，建立一个高度差异化、竞争性和创新力的研发管线，以实现长期增长。”本次媒体日中，拜耳着重强调了其对于成为领先肿瘤公司的愿景，同时也为发展路径找准了方向，即推动有可能取得突破性进展的创新，重点关注结肠癌等消化道肿瘤和前列腺癌、肺癌等常见癌症。“为了成为拥有领先肿瘤业务的公司，拜耳实实在在做了很多工作，例如在波士顿成立了研究与创新中心，”Stefan分享道：“波士顿可以说是全球肿瘤学研发圣地，我们的精准分子肿瘤学研究团队就驻扎此地。”拜耳波士顿研究与创新中心所在地当然，除了关注更加有效耐受的肿瘤药物开发，正确及时的筛查也十分重要，拜耳深谙此道。今年3月，拜耳已经与赛默飞世尔科技达成合作，为其精准肿瘤学产品组合匹配下一代基于测序的伴随诊断检测方法。细胞和基因疗法（CGT）是拜耳实现医疗突破的另一重要版图。Stefan介绍了拜耳投资部门“飞跃计划”（Leaps by Bayer），该部门专注于寻找具有突破性的、有望治愈或逆转疾病进程的疗法和技术，BlueRock和AskBio就是其引以为豪的投资案例，被寄予了极高期许。两家子公司开发的在研药物，细胞疗法BRT-DA01和基因疗法AB-1002，分别在治疗帕金森病、充血性心衰的早期临床试验中展现了积极的疗效。除此以外，另有七个CGT项目处于不同临床阶段，它们都遍布医疗需求最高的领域。BlueRock实验室创新项目不断涌现的背后，是拜耳对于原创研发的持续性、高水准投入。据拜耳披露，为搭建出CGT平台，已累计投入约35亿欧元，全方位覆盖神经系统、心血管、眼科、罕见病等高需求疾病领域。不过，Stefan表示，CGT产品从临床研究到上市可及仍然需要冲破临床试验规模扩大、生产规模化、成本质量管控和商业化落地等重重难关。为此，拜耳已将生产、开发、商业化团队聚拢在一起，共同研究如何将CGT尽早推向市场，同时也在其他分销商、医疗服务机构等合作方思考怎样保证患者可及。在中国，拜耳正在与北京大学合作，助力CGT等多个重点领域内的基础研究成果向新药研发转化，并加速推动药物研发价值链上的前沿技术研究，同时与上海医药携手将拜耳Co.Lab共创平台引入中国，成为拜耳全球新药孵化网络的重要组成部分，促进CGT等领域的前沿创新。从早期研究，到拥有制造能力和平台，最终实现药物可及，拜耳为加快兑现CGT造福患者的承诺，正不计其数聚焦全链条投入，而这也将内生出拜耳处方药不竭的进化动力。总结最后，Stefan信心十足地表示，拜耳对新药研发重点有着清晰的认识，能够开发出更加精准和“友好”的新药，改善全球患者的生活质量。与此同时，已经进入中国超过140年的拜耳，其在中国的创新发展路径也已十分清晰。拜耳集团处方药事业部全球执行副总裁兼中国区总裁、拜耳集团大中华区总裁周晓兰在公共场合也多次提到，公司将围绕癌症、心脑血管疾病、糖尿病等慢性疾病这些重点领域加快产品组合的更新和迭代，向中国市场源源不断的提供更多，更好的药品。还将针对一些中国特有的疾病领域制订研发策略，以便最大程度的支持目前未被满足的临床需求，让更多的中国患者获益。可以看出，无论处方药业务如何演进，鼓励创新和惠及患者始终是拜耳的立身之道，即聚焦尚未得到满足的高需求领域，倾听患者心声，精准把握个体化需求和现实应用场景。Copyright © 2024 PHARMCUBE. All Rights Reserved.欢迎转发分享及合理引用，引用时请在显要位置标明文章来源；如需转载，请给微信公众号后台留言或发送消息，并注明公众号名称及ID。免责申明：本微信文章中的信息仅供一般参考之用，不可直接作为决策内容，医药魔方不对任何主体因使用本文内容而导致的任何损失承担责任。

财报细胞疗法基因疗法免疫疗法

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

适应症	最高研发状态	国家/地区	公司
甲状腺素运载蛋白淀粉样变心肌病	申请上市	美国更多	BridgeBio Pharma, Inc. 更多
家族性淀粉样神经病	临床3期	西班牙更多	BridgeBio Pharma, Inc. 更多
心肌疾病	临床3期	日本更多	AstraZeneca PLC 更多
淀粉样神经病	临床3期	-	Eidos Therapeutics, Inc. 更多
转甲状腺素运载蛋白淀粉样变性	临床3期	日本更多	Alexion Pharmaceuticals, Inc. 更多

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临床结果

适应症

分期

评价

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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


NCT03458130 (Pubmed \| J Am Coll Cardiol) 人工标引	临床2期	甲状腺素运载蛋白淀粉样变心肌病 TTR Mutation	49	AG10	遞窪憲夢壓齋夢壓願窪(夢繭製壓顧製鬱襯齋網) = 壓顧餘艱窪網獵艱選醖膚鹹淵獵鏇積觸觸憲廠 (鏇壓積製憲襯積鹹繭襯, 21)	积极	2019-07-23
				Placebo	-
NCT04622046 (Corporate Publications) 人工标引	临床3期	甲状腺素运载蛋白淀粉样变心肌病	-	Acoramidis	(糧鏇鏇壓選淵醖鏇憲觸) = The primary endpoint was met (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001) . 築構網窪壓蓋願餘壓鑰 (憲鑰鹹窪糧製繭繭積淵 ) 达到	积极	2024-02-02
NCT03458130 (CTgov)	临床2期	老年性心脏淀粉样变性 \| 甲状腺素运载蛋白淀粉样变心肌病	49	Placebo Oral Tablet	(鹽構膚獵鑰簾繭積鹽淵) = 淵製鑰衊網簾醖鏇鏇範憲網遞顧淵膚憲齋顧蓋 (淵齋鑰憲鏇選齋鏇艱鹽, 鹹窪鏇積齋淵夢遞襯壓 ~ 醖餘獵醖網範網築壓廠) 更多	-	2022-11-16
ESC2023	N/A	转甲状腺素运载蛋白淀粉样变性	-	Acoramidis	繭觸構網繭夢鹹襯製壓(夢積齋醖繭襯壓壓鹽簾) = 簾憲製獵願廠鑰鹹廠夢壓衊衊齋衊鬱遞餘憲製 (選淵觸膚餘觸鹽膚鹹壓 ) 更多	-	2023-08-26
				Tafamidis	繭觸構網繭夢鹹襯製壓(夢積齋醖繭襯壓壓鹽簾) = 鹹憲構襯衊壓醖鏇衊窪壓衊衊齋衊鬱遞餘憲製 (選淵觸膚餘觸鹽膚鹹壓 ) 更多
NCT03860935 (Literature) 人工标引	临床3期	甲状腺素运载蛋白淀粉样变心肌病	632	Acoramidis 800 mg	(鹽積鑰餘膚築鬱觸襯顧) = 襯網獵願艱壓簾窪淵網觸憲鬱顧遞鑰築鹹積廠 (餘壓壓鹹餘艱淵窪顧憲 ) 更多	积极	2024-01-11
				placebo	(鹽積鑰餘膚築鬱觸襯顧) = 襯鏇獵夢夢醖選構觸積觸憲鬱顧遞鑰築鹹積廠 (餘壓壓鹹餘艱淵窪顧憲 ) 更多
NCT03860935 (ATTRibute-CM, Corporate Publications) 人工标引	临床3期	甲状腺素运载蛋白淀粉样变心肌病	-	Acoramidis	(壓糧壓範獵鑰遞積憲觸) = 鏇遞鏇襯衊窪餘淵壓窪繭顧齋顧憲網膚構簾築 (網艱構衊觸壓鏇鏇鏇築, 62.7) 更多	不佳	2021-12-31
				Placebo	(壓糧壓範獵鑰遞積憲觸) = 鏇蓋遞顧願餘製壓餘蓋繭顧齋顧憲網膚構簾築 (網艱構衊觸壓鏇鏇鏇築, 59.7) 更多