A Phase 2/3 Multicenter, Randomized, Double-Blind, Placebo-Controlled and Open-Label Extension Trial to Evaluate the Efficacy and Safety of Aficamten in a Pediatric Population With Symptomatic Obstructive Hypertrophic Cardiomyopathy

The purpose of this study is to evaluate the efficacy, safety and PK of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

开始日期2024-05-29

申办/合作机构

Cytokinetics, Inc.

NCT06116968

/ Recruiting临床3期

An Open-Label Study of Aficamten for Chinese Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy

This is an open-label extension study of China cohort in the phase 3 study (CY 6031) of aficamten for the treatment of obstructive HCM (oHCM) to collect long-term safety and tolerability data, including assessments of cardiac function and steady-state Pharmacokinetics (PK) during chronic dosing with aficamten.

开始日期2023-11-14

申办/合作机构

箕星药业科技（上海）有限公司

NCT06081894

/ Recruiting临床3期

A Phase 3, Multi-Center, Randomized, Double-Blind Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy

This clinical trial will study the effects of aficamten (versus placebo) on the quality of life, exercise capacity, and clinical outcomes of patients with non-obstructive hypertrophic cardiomyopathy.

开始日期2023-08-30

申办/合作机构

Cytokinetics, Inc.

100 项与 Aficamten 相关的临床结果

登录后查看更多信息

100 项与 Aficamten 相关的转化医学

登录后查看更多信息

100 项与 Aficamten 相关的专利（医药）

登录后查看更多信息

项与 Aficamten 相关的文献（医药）

2025-05-01·American heart journal plus : cardiology research and practice

Efficacy of aficamten in obstructive hypertrophic cardiomyopathy: A systematic review and meta-analysis

Review

作者: Safiullah, Muhammad ; Irfan, Hamza ; Javaid, Hammad ; Fatima, Laveeza ; Haq, Muhammad Zain Ul ; Saddique, Muhammad Nabeel ; Iqbal, Javed ; Bhatti, Muhammad Imaz ; Ashraf, Saad ; Masood, Saad ; Qadri, Maria ; Jha, Anurag ; Mehmood, Qasim ; Shamoon, Sheena ; Iftikhar, Sana

Background:

Obstructive hypertrophic cardiomyopathy (oHCM), a morbid hereditary condition, is characterized by asymmetrical intraventricular septum enlargement, obstructing blood flow from the left ventricle outflow tract (LVOT) and lowering cardiac output. Aficamten, a novel selective, oral myosin inhibitor, has been suggested to reduce myocardial hypercontractility and decrease LVOT gradient in oHCM.

Methods:

A comprehensive search was conducted through PubMed, Embase, Scopus, and Cochrane databases for relevant literature from inception up to May 2024. Six studies focusing on efficacy and safety aficamten was included. Pooled outcome estimates were reported as mean difference (MD) and 95 % CI using random effect model. Statistical heterogeneity was assessed using I² and X² statistics.

Results:

We found a significant change of -143.23 (pg/ml) NT-proBNP [95 % CI -564.8 to 278.4, I² = 97.65 %, P < 0.001], -50.9 mmHg Valsalva LVOT gradient [95 % CI -55.2 to -46.6, I² = 0 %, P = 0.44], -38.5 mmHg resting LVOT gradient [95 % CI -49.9 to -27.6, I² = 0 %, P = 0.64], -5.98 % mean LVEF [95 % CI -9.4 to -2.6, I² = 64.18 %, P = 0.06] and -2.32 (ng/dl) Hs-Troponin I [95 % CI -7.55 to -2.91, I² = 0 %, P = 0.97] from the baseline. We found significant 64.9 % ≥ 1 NYHA class improvement [95 % CI 45.8 %-84.1 %, I² = 90.6 %, P < 0.001] in aficamten. There was 'low' overall risk of bias in included studies.

Conclusion:

We found that aficamten significantly reduced myocardial stress surrogates and functional disability parameters. The small sample sizes, diverse study designs and single-arm analysis limit our findings. More robust trials with larger sample sizes are required to establish conclusive evidence.

2025-05-01·American Journal of Cardiovascular Drugs

Cardiac Myosin Inhibitors for Obstructive Hypertrophic Cardiomyopathy: A Meta-analysis of Randomized Placebo-Controlled Trials

Review

作者: Alexandre, Felipe Kalil Beirão ; Kherallah, Riyad Yazan ; Felix, Nicole ; Alexandre Costa, Thomaz ; Nogueira, Alleh ; Serpa, Frans ; Andrade Duarte de Farias, Maria do Carmo ; Vianna Silva, Guilherme ; Godoi, Amanda ; Pirez, Jacqueline ; Teixeira, Larissa

BACKGROUND:

Cardiac myosin inhibitors (CMI) have emerged as the first disease-specific, noninvasive therapy with promising results in patients with hypertrophic cardiomyopathy. However, its role in obstructive hypertrophic cardiomyopathy (oHCM) remains uncertain, especially in secondary endpoints of randomized controlled trials (RCTs).

METHODS:

We systematically searched PubMed, Embase, Web of Science, and Clinicaltrials.gov from inception to June 2024 for RCTs comparing CMI versus placebo in patients with oHCM. We applied a random-effects model to evaluate efficacy and safety outcomes and primary or secondary outcomes of RCTs.

RESULTS:

We included five RCTs comprising 767 patients, of whom 402 (52.5%) were randomized to CMI. Relative to placebo, CMI were associated with a higher rate of improvement of at least one New York Heart Association (NYHA) functional class [risk ratio (RR) 2.33; 95% confidence interval (CI) 1.92-2.82]. In addition, CMI reduced resting left ventricular outflow tract (LVOT) [mean difference (MD) - 42.51 mmHg; 95% CI - 59.27 to - 25.75] and the provoked LVOT gradients (MD - 46.12 mmHg; 95% CI - 55.70 to - 36.54). However, CMI significantly increased the risk of reaching a left ventricular ejection fraction below 50% (RR 4.80; 95% CI 1.42-16.20), affecting 8% of patients during long-term follow-up of up to 120 weeks. There was no significant interaction across subgroups of class representatives, pointing to a class effect. The benefit-risk analysis indicated a larger benefit for NYHA class improvement than risk for systolic dysfunction.

CONCLUSION:

In patients with oHCM, mavacamten and aficamten as a class improve clinical and hemodynamic endpoints compared with placebo, albeit with a higher incidence of a reduction in left ventricular ejection fraction.

REGISTRATION:

PROSPERO CRD42023468079.

2025-05-01·JOURNAL OF CARDIOTHORACIC AND VASCULAR ANESTHESIA

Advances in Cardiovascular Pharmacotherapy. I. Cardiac Myosin Inhibitors

Review

作者: Crystal, George J ; Pagel, Paul S ; Hang, Dustin ; Freed, Julie K

Hypertrophic cardiomyopathy (HCM) is the most common inherited cardiomyopathy. The disease is characterized by asymmetric left ventricular (LV) remodeling with myocyte disarray and interstitial fibrosis, a hypercontractile state, dynamic subaortic obstruction of the LV outflow tract, impaired LV diastolic function, atrial and ventricular arrhythmias, and sudden cardiac death. HCM occurs as a result of pathological alterations in the cardiac myocyte's chemomechanical cycle, in which an enhanced rate of myosin-actin crossbridge formation and destabilization of the energy-conserving "super-relaxed off-actin state" of myosin play essential roles. For decades, management of HCM has been limited almost exclusively to medications (eg, beta-blockers, calcium channel blockers, disopyramide) and interventions (eg, septal reduction therapy, implanted cardioverter-defibrillator devices) that palliate symptoms, but do not address the disease's underlying causative mechanisms. A new class of cardiovascular medications, cardiac myosin inhibitors, has surged to the forefront of HCM treatment in recent years. These drugs, including mavacamten and aficamten, show great promise to profoundly affect the disease's clinical course. In this article, the authors review the molecular mechanisms of action of cardiac myosin inhibitors, discuss in detail the most recent data from mavacamten and aficamten clinical trials, describe future planned studies designed to address unanswered questions about their clinical utility in HCM phenotypes, and comment on their potential application to patients with other forms of heart failure with preserved ejection fraction. The possible anesthetic implications of mavacamten and aficamten are also discussed because it is highly likely that patients who are treated with these medications will begin to present for perioperative care with increasing regularity.

304

项与 Aficamten 相关的新闻（医药）

2025-05-15

·PMLiVE

Cytokinetics shares positive phase 3 results for aficamten in heart disease

Cytokinetics has shared positive top-line results from a late-stage study of its investigational oral cardiac myosin inhibitor aficamten in hypertrophic cardiomyopathy (HCM). The phase 3 MAPLE-HCM trial has been comparing the candidate against standard-of-care beta blocker metoprolol in more than 170 patients with symptomatic obstructive HCM. As many as one in 500 people in the UK and US are living with HCM, a common form of genetic heart disease. Some patients do not experience symptoms, causing a large percentage of cases to remain undiagnosed, while others may only feel symptoms such chest pain and shortness of breath during exercise. Approximately two-third of HCM patients have the obstructive form of the condition, in which the thickened part of the heart muscle blocks or reduces the blood flow from the heart, while non-obstructive HCM, when the heart muscle is thickened but does not block blood flow out of the heart, is less common. Aficamten is designed to reduce the hypercontractility associated with HCM by blocking myosin, a motor protein that plays a key role in muscle contraction, from pulling. MAPLE-HCM met its primary endpoint, with aficamten monotherapy significantly improving peak oxygen uptake, measured by cardiopulmonary exercise testing, compared to metoprolol monotherapy from baseline to week 24. The safety and tolerability profile of Cytokinetics’ drug was also shown to be favourable in comparison to metoprolol. The company’s executive vice president of research and development, Fady Malik, said the results “represent the first evidence that aficamten may be used as monotherapy to deliver clinically meaningful improvements in people living with obstructive HCM”. “Importantly, the results from MAPLE-HCM provide important context to the benefit of this potential new medicine compared to the current standard of care,” he added. Aficamten, which is also being evaluated in non-obstructive HCM, is currently under regulatory review by the US Food and Drug Administration, with a decision expected by 26 December. The drug is also being assessed by the European Medicines Agency.

临床3期临床结果申请上市

2025-05-14

·药明康德

突破性小分子疗法3期结果积极！有望年底获批上市；GSK囊获潜在“best-in-class”肝病疗法……

GSK囊获潜在“best-in-class”肝病疗法今日，GSK与Boston Pharmaceuticals宣布达成协议，GSK将收购Boston Pharmaceuticals的主打疗法efimosfermin alfa。该疗法目前处于3期准备阶段，用于治疗和预防脂肪变性肝病（SLD）的进展。根据协议，GSK将支付Boston Pharmaceuticals共12亿美元的预付款，并另有潜在总计8亿美元的里程碑款项。Efimosfermin是一种在研、潜在“best-in-class”、每月一次注射的成纤维细胞生长因子21（FGF21）类似物，旨在调节关键代谢途径以减少肝脏脂肪、改善肝脏炎症并逆转代谢功能障碍相关脂肪性肝炎（MASH）患者的肝纤维化。该药物在未来可能开发用于治疗酒精性肝病（ALD），MASH与ALD均属于SLD。鉴于efimosfermin的直接抗纤维化作用机制以及GSK基于人类遗传学和疾病表型研究的数据洞察，该药物有潜力针对SLD的更晚期阶段，并可能与GSK正在开发、用于其他SLD患者亚群的siRNA疗法GSK'990联用。最近一项2期试验的数据显示，efimosfermin能快速显著逆转肝纤维化并阻止其进展，且耐受性良好。该试验旨在评估每月一次皮下注射efimosfermin对经活检证实的中度至重度（F2或F3）MASH患者的疗效和安全性。这些数据表明，与其他治疗方法相比，efimosfermin可能带来更大的纤维化改善，且预期其益处独立于胰高血糖素样肽-1（GLP-1）疗法。此外，efimosfermin还可能降低甘油三酯并改善血糖控制，这对常伴有心脏代谢共病的MASH患者尤为重要。Efimosfermin还具备低免疫原性和延长的半衰期，提供了每月给药方案和改善患者便利性的潜力。SLD是一个存在显著未满足医疗需求的领域，全球约5%的人口受其影响，而患者的治疗选择有限。SLD的特征是肝脏脂肪堆积（脂肪变性），伴随炎症和纤维化。其中ALD影响全球约2600万患者，与MASH一起是美国肝移植的主要原因，给医疗资源带来重大负担。诺和诺德达成22亿美元小分子疗法合作Septerna公司与诺和诺德（Novo Nordisk）今日宣布达成一项全球独家合作与许可协议，旨在共同发现、开发并商业化用于治疗肥胖、2型糖尿病及其他心脏代谢疾病的口服小分子药物。双方将率先启动四项开发项目，针对一种或多种特定的G蛋白偶联受体（GPCR）靶点开展潜在小分子疗法的研发，这些靶点包括GLP-1、GIP和胰高血糖素受体。GPCR是细胞膜受体中最大且最具多样性的家族之一，有数百种不同的GPCR在人体几乎所有器官系统中调控着生理过程。Septerna公司通过其专有的Native Complex Platform平台，致力于充分释放GPCR疗法的潜力。该公司专注于在多个治疗领域中开发口服小分子药物管线，初期聚焦于内分泌、免疫与炎症、以及代谢性疾病领域。根据协议条款，Septerna有资格从诺和诺德获得总计约22亿美元的付款，包括首付款及研发、开发和商业化里程碑付款。其中包含超过2亿美元的首付款和近期里程碑付款。合作开发项目中的所有研发费用将由诺和诺德承担。双方将联合开展从发现阶段到开发候选物选择阶段的研究活动。自IND支持性研究阶段起，诺和诺德将全权负责全球范围内的所有开发与商业化工作。艾伯维达数十亿美元siRNA疗法开发合作艾伯维（AbbVie）与ADARx Pharmaceuticals今日宣布达成一项合作与许可选择权协议，旨在联合开发适用于多个疾病领域的小干扰RNA（siRNA）疗法，这些领域包括神经科学、免疫学和肿瘤学。此次战略合作将充分发挥ADARx在RNA发现方面的专业能力及其专有的siRNA技术，该技术有望实现持久且精确的mRNA沉默。艾伯维则将在抗体工程、抗体偶联药物以及组织递送策略等方面提供专业支持，以助力ADARx的药物发现工作。根据协议条款，ADARx将获得3.35亿美元的预付款，并有资格获得数十亿美元包含里程碑付款的款项。突破性小分子疗法3期结果积极！有望年底获批上市日前，Cytokinetics公布了MAPLE-HCM临床3期试验的积极顶线结果。分析显示，其在研小分子抑制剂aficamten单药用于治疗有症状的梗阻性肥厚型心肌病（HCM）患者时，疗效优于现有标准疗法。该试验的完整结果预计在即将召开的医学会议上公布。Aficamten目前正在接受美国FDA的审查，PDUFA日期为2025年12月26日。MAPLE-HCM是一项多中心、随机、双盲、活性对照的临床3期试验，在175例有症状的梗阻性HCM患者中比较aficamten单药与现有标准治疗β受体阻滞剂美托洛尔相较的疗效与安全性。试验主要终点是通过心肺运动试验（CPET）测量的从基线至第24周的峰值摄氧量（pVO2）变化。次要终点包括堪萨斯城心肌病问卷（KCCQ）评分从基线至第24周的变化、纽约心脏协会（NYHA）心功能分级改善≥1级的患者比例，以及左心室质量指数（LVMI）、左心房容积指数（LAVI）、Valsalva动作后左心室流出道梯度（LVOT-G）和心衰生物标志物NT-proBNP的变化。分析显示，MAPLE-HCM达到了主要终点，与活性对照药物相比，aficamten在基线至第24周的峰值摄氧量改善方面显示出统计学显著性优势。在MAPLE-HCM试验中，aficamten的安全性和耐受性特征亦优于对照组。Aficamten是一种在研、潜在“best-in-class”的选择性小分子心肌肌球蛋白抑制剂。Aficamten旨在减少每个心动周期中活跃的肌动蛋白-肌球蛋白交联桥数量，从而抑制与HCM相关的心肌过度收缩。在临床前模型中，aficamten通过直接结合心肌肌球蛋白上一个独特的选择性变构结合位点来降低心肌收缩力，从而阻止肌球蛋白进入力产生状态。Aficamten曾获得美国FDA和中国国家药品监督管理局（NMPA）授予的突破性疗法认定，用于治疗有症状的梗阻性HCM。参考资料：[1] GSK to acquire efimosfermin, a phase III-ready potential best-in-class specialty medicine to treat and prevent progression of steatotic liver disease (SLD). Retrieved May 14, 2025 from https://www.gsk.com/en-gb/media/press-releases/gsk-to-acquire-efimosfermin-a-phase-iii-ready-potential-best-in-class-specialty-medicine-to-treat-and-prevent-progression-of-steatotic-liver-disease-sld/[2] Novo Nordisk A/S: Septerna and Novo Nordisk to collaborate on oral small molecule medicines for obesity and other cardiometabolic diseases. Retrieved May 14, 2025 from https://ir.septerna.com/news-releases/news-release-details/novo-nordisk-septerna-and-novo-nordisk-collaborate-oral-small[3] AbbVie and ADARx Pharmaceuticals Announce Collaboration and License Option Agreement to Develop Next-Generation siRNA Therapies Across Multiple Therapeutic Areas. Retrieved May 14, 2025 from https://www.adarx.com/abbvie-and-adarx-pharmaceuticals-announce-collaboration-and-license-option-agreement-to-develop-next-generation-sirna-therapies-across-multiple-therapeutic-areas/[4] Cytokinetics Announces Positive Topline Results From MAPLE-HCM. Retrieved May 14, 2015 from https://ir.cytokinetics.com/news-releases/news-release-details/cytokinetics-announces-positive-topline-results-maple-hcm免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

siRNA上市批准临床3期引进/卖出临床2期

2025-05-14

·GlobeNewswire-Health Care

Cytokinetics Announces Positive Topline Results From MAPLE-HCM

Trial Demonstrates Superiority of Aficamten to Standard of Care Beta Blocker in Improving Peak Exercise Capacity in Patients with Obstructive Hypertrophic Cardiomyopathy May 13, 2025 -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced positive topline results from MAPLE-HCM (Metoprolol vs Aficamten in Patients with LVOT Obstruction on Exercise Capacity in HCM), a Phase 3 clinical trial comparing aficamten as monotherapy to the standard of care beta blocker metoprolol as monotherapy in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). MAPLE-HCM met its primary endpoint, demonstrating a statistically significant improvement in peak oxygen uptake (pVO2) from baseline to Week 24 for aficamten compared to metoprolol. The safety and tolerability profile of aficamten was favorable in comparison to metoprolol in MAPLE-HCM. The full results from MAPLE-HCM will be presented at an upcoming medical conference. “These results represent the first evidence that aficamten may be used as monotherapy to deliver clinically meaningful improvements in people living with obstructive hypertrophic cardiomyopathy,” said Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development. “Importantly, the results from MAPLE-HCM provide important context to the benefit of this potential new medicine compared to the current standard of care. We are grateful to the investigators, site personnel and patients who participated in MAPLE-HCM, and look forward to presenting the full results at an upcoming medical meeting.” MAPLE-HCM was a Phase 3, multi-center, randomized, double-blind active-comparator clinical trial of aficamten compared to metoprolol in patients with symptomatic obstructive HCM. The primary endpoint was the change in peak oxygen uptake (pVO2) from baseline to Week 24 measured by cardiopulmonary exercise testing (CPET). Secondary endpoints include the change from baseline to Week 24 in Kansas City Cardiomyopathy Questionnaire (KCCQ) score, the proportion of patients with ≥1 class improvement in New York Heart Association (NYHA) functional class, and changes in left ventricular mass index (LVMI), left atrial volume index (LAVI), post-Valsalva left ventricular outflow tract gradient (LVOT-G) and NT-proBNP. MAPLE-HCM enrolled 175 patients, randomized on a 1:1 basis to receive aficamten or metoprolol as monotherapy in a double-blind, double dummy fashion. Randomization was stratified by CPET exercise modality (treadmill or bicycle) and recently diagnosed versus chronic obstructive HCM. At screening, patients enrolled in MAPLE-HCM had a resting LVOT-G ≥30 mmHg and/or post-Valsalva LVOT-G ≥50 mmHg in addition to left ventricular ejection fraction (LVEF) ≥ 60%, respiratory exchange ratio (RER) ≥ 1.05 and pVO2 aficamten or metoprolol based on echocardiographic guidance as well as a matching placebo for the alternate therapy. Aficamten is an investigational selective, small molecule cardiac myosin inhibitor discovered following an extensive chemical optimization program that was conducted with careful attention to therapeutic index and pharmacokinetic properties and as may translate into next-in-class potential in clinical development. Aficamten was designed to reduce the number of active actin-myosin cross bridges during each cardiac cycle and consequently suppress the myocardial hypercontractility that is associated with HCM. In preclinical models, aficamten reduced myocardial contractility by binding directly to cardiac myosin at a distinct and selective allosteric binding site, thereby preventing myosin from entering a force producing state. The development program for aficamten is assessing its potential as a treatment that improves exercise capacity as measured by peak oxygen uptake (pVO2) and relieves symptoms in patients with HCM. Aficamten was compared to placebo in SEQUOIA-HCM, a positive pivotal Phase 3 clinical trial demonstrating improvements in functional capacity, LVOT gradients, and heart failure symptoms in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). Aficamten received Breakthrough Therapy Designation for the treatment of symptomatic obstructive HCM from the U.S. Food & Drug Administration (FDA) and from the National Medical Products Administration (NMPA) in China. Aficamten is currently under regulatory review in the U.S by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of December 26, 2025. Additionally, the European Medicines Agency (EMA) is reviewing a Marketing Authorization Application (MAA) for aficamten, and The Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) is reviewing an NDA for aficamten with Priority Review. Aficamten is also currently being evaluated in ACACIA-HCM, a Phase 3 clinical trial of aficamten in patients with non-obstructive HCM; CEDAR-HCM, a clinical trial of aficamten in a pediatric population with obstructive HCM; and FOREST-HCM, an open-label extension clinical study of aficamten in patients with HCM. Cytokinetics is a specialty cardiovascular biopharmaceutical company, building on its over 25 years of pioneering scientific innovations in muscle biology to advance a pipeline of potential new medicines for patients suffering from diseases of cardiac muscle dysfunction. Cytokinetics is readying for potential regulatory approvals and commercialization of aficamten, a cardiac myosin inhibitor following positive results from SEQUOIA-HCM, the pivotal Phase 3 clinical trial in patients with obstructive hypertrophic cardiomyopathy (HCM). Aficamten is also being evaluated in additional clinical trials enrolling patients with obstructive and non-obstructive HCM. Cytokinetics is also developing omecamtiv mecarbil, a cardiac myosin activator, in patients with heart failure with severely reduced ejection fraction (HFrEF), CK-586, a cardiac myosin inhibitor with a mechanism of action distinct from aficamten, for the potential treatment of heart failure with preserved ejection fraction (HFpEF) and CK-089, a fast skeletal muscle troponin activator with potential therapeutic application to a specific type of muscular dystrophy and other conditions of impaired skeletal muscle function. References: Xu, D., Lutz, J., & Divanji, P., et al. (2024, March). Drug–Drug Interaction Study to Evaluate the Effect of Strong CYP3A Inhibition and P450 Induction on the Pharmacokinetics of Aficamten and the Effect of Aficamten on P-Glycoprotein in Healthy Participants. Poster session presented at the American Society for Clinical Pharmacology & Therapeutics Meeting, Colorado Springs, CO. CVrg: Heart Failure 2020-2029, p 44; Maron et al. 2013 DOI: 10.1016/S0140-6736(12)60397-3; Maron et al 2018 10.1056/NEJMra1710575 Symphony Health 2016-2021 Patient Claims Data DoF; Maron MS, Hellawell JL, Lucove JC, Farzaneh-Far R, Olivotto I. Occurrence of Clinically Diagnosed Hypertrophic Cardiomyopathy in the United States. Am J Cardiol. 2016; 15;117(10):1651-1654. Gersh, B.J., Maron, B.J., Bonow, R.O., Dearani, J.A., Fifer, M.A., Link, M.S., et al. 2011 ACCF/AHA guidelines for the diagnosis and treatment of hypertrophic cardiomyopathy. A report of the American College of Cardiology Foundation/American Heart Association Task Force on practice guidelines. Journal of the American College of Cardiology and Circulation, 58, e212-260. Hong Y, Su WW, Li X. Risk factors of sudden cardiac death in hypertrophic cardiomyopathy. Current Opinion in Cardiology. 2022 Jan 1;37(1):15-21 The content above comes from the network. if any infringement, please contact us to modify.

临床结果申请上市突破性疗法优先审批

100 项与 Aficamten 相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
梗阻性肥厚型心肌病	申请上市	中国	Cytokinetics, Inc.	2024-10-15
非梗阻性肥厚型心肌病	临床3期	美国	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	中国	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	阿根廷	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	澳大利亚	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	巴西	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	加拿大	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	丹麦	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	法国	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	德国	Cytokinetics, Inc.	2023-08-30

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05186818 (SEQUOIA-HCM, GlobeNewswire) 人工标引	临床3期	肥大型心肌病	-	Aficamten	鏇願壓簾鹹選鬱積襯選(窪襯遞獵築顧醖鹹衊獵) = 廠遞鹽築餘糧築簾壓廠淵構壓淵鹽願構夢衊窪 (襯衊顧製蓋願築顧鹹簾, 3.1) 更多	积极	2024-11-16
				Placebo	鏇願壓簾鹹選鬱積襯選(窪襯遞獵築顧醖鹹衊獵) = 淵選膚襯築艱糧膚鏇製淵構壓淵鹽願構夢衊窪 (襯衊顧製蓋願築顧鹹簾, 2.7) 更多
NCT05186818 (SEQUOIA-HCM, Pubmed \| J Am Coll Cardiol)	临床3期	梗阻性肥厚型心肌病 N-terminal pro-B-type natriuretic peptide	-	Aficamten	構鹹衊簾獵壓鏇鏇觸顧(醖艱顧憲遞繭選淵齋鑰) = 構壓獵鏇網製網廠構獵構襯繭築餘鑰襯鏇簾淵 (鬱糧窪繭衊鏇糧醖鏇醖 ) 更多	积极	2024-11-01
				Placebo	構鹹衊簾獵壓鏇鏇觸顧(醖艱顧憲遞繭選淵齋鑰) = 壓鹽鬱繭鑰窪襯膚餘廠構襯繭築餘鑰襯鏇簾淵 (鬱糧窪繭衊鏇糧醖鏇醖 ) 更多
NCT05186818 (SEQUOIA-HCM, Pubmed \| J Am Coll Cardiol)	临床3期	梗阻性肥厚型心肌病	282	Aficamten	淵繭鏇構觸築艱襯壓襯(艱鬱夢憲構積蓋範觸憲) = 鏇窪簾淵積廠遞衊艱鹽網鹹繭壓構遞齋糧廠衊 (積鏇糧獵範顧廠網鏇膚, -25 ~ -6) 更多	积极	2024-11-01
NCT05186818 (SEQUOIA-HCM, Pubmed \| J Am Coll Cardiol)	临床3期	梗阻性肥厚型心肌病 N-terminal pro-B-type natriuretic peptide (NT-proBNP)	282	Aficamten	壓積鏇衊繭觸衊鏇製壓(糧餘壓鹹廠獵餘繭鹽壓) = 膚顧築艱夢鹽鏇積簾鏇膚淵築淵製繭淵齋選衊 (構願構範鏇願糧製壓夢 ) 更多	积极	2024-11-01
NCT05186818 (SEQUOIA-HCM, GlobeNewswire) 人工标引	临床3期	肥大型心肌病	282	Aficamten	鹹築簾膚觸衊窪鹹願糧(壓願製獵憲鹽蓋鹽夢顧): Difference = -12.2 (95% CI, -18.0 ~ -6.5), P-Value = <0.001 更多	积极	2024-09-01
				Placebo
NCT04219826 \| NCT04848506 \| NCT05186818 (FOREST-HCM, GlobeNewswire) 人工标引	临床2/3期	肥大型心肌病	-	Aficamtenaficamten (Cumulative aficamten-treated pool)	餘鑰鹽範製糧餘憲餘簾(網蓋憲壓蓋製積鹹觸遞) = 遞夢築餘膚窪糧襯窪蓋顧積顧淵衊顧鹽繭繭衊 (襯襯願糧積窪積餘襯願 ) 更多	积极	2024-09-01
				Aficamten (Placebo-controlled pool)	餘鑰鹽範製糧餘憲餘簾(網蓋憲壓蓋製積鹹觸遞) = 廠繭網範築憲獵壓醖憲顧積顧淵衊顧鹽繭繭衊 (襯襯願糧積窪積餘襯願 ) 更多
NCT04219826 \| NCT04848506 \| NCT05186818 (ESC2024)	N/A	梗阻性肥厚型心肌病	-	Aficamten	製鑰積鏇築鏇鹽顧憲淵(獵構醖願選衊糧築窪糧) = 遞憲鹽糧壓積願壓網鹽觸築艱醖衊獵襯糧遞鹹 (鏇鑰艱廠願憲夢糧壓構 ) 更多	-	2024-09-01
				Placebo	製鑰積鏇築鏇鹽顧憲淵(獵構醖願選衊糧築窪糧) = 襯觸繭糧醖構構糧觸顧觸築艱醖衊獵襯糧遞鹹 (鏇鑰艱廠願憲夢糧壓構 ) 更多
NCT05186818 (SEQUOIA-HCM, Pubmed) 人工标引	临床3期	梗阻性肥厚型心肌病	282	aficamten	蓋築製簾鑰醖網繭襯憲(觸顧遞積襯鬱餘醖積窪) = 積觸遞夢憲廠簾憲繭餘餘獵憲餘淵觸鑰淵築簾 (蓋淵願餘願醖衊網範願, 1.2 ~ 2.3) 达到更多	积极	2024-05-13
NCT04848506 (FOREST-HCM, Corporate Publications) 人工标引	临床2期	肥大型心肌病	46	Aficamten	鹽憲鏇衊衊築窪鑰襯窪(淵襯夢醖鏇顧蓋淵襯鹹) = none 衊築憲憲築糧簾繭觸製 (窪蓋積憲願積膚簾範齋 ) 更多	积极	2024-04-05
NCT04219826 (REDWOOD-HCM, Pubmed) 人工标引	临床2期	非梗阻性肥厚型心肌病	41	Aficamten 5-20 mg	鑰築鏇夢獵餘願夢夢壓(鑰廠簾蓋鏇鑰齋淵衊築) = One patient with prior history of aborted sudden cardiac death experienced a fatal arrhythmia during the study 網積艱餘艱獵願廠廠鏇 (醖網構蓋餘願壓顧醖憲 ) 更多	积极	2024-03-15