A Phase 3, Open Label, Single Arm Study to Evaluate Efficacy, Safety and Tolerability of Aficamten in Adult Japanese Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy

Researchers are looking for a better way to treat Japanese people who have symptomatic obstructive hypertrophic cardiomyopathy (symptomatic oHCM).
Obstructive hypertrophic cardiomyopathy (oHCM) is a type of heart disease where the heart muscles become thicker than normal due to over contraction. This thickening makes it harder than normal due to over contraction. This thickening makes it harder for the heart to pump blood out to the rest of the body. In symptomatic oHCM people with the condition experience symptoms like shortness of breath, chest pain, fainting, high blood pressure and irregular heartbeats.
The study treatment aficamten, also called BAY3723113, is under development to treat symptomatic oHCM. It aims to reduce the activity of cardiac myosin, a protein that helps heart muscles to contract, and thereby preventing over contraction and muscle thickening.
Although treatment options are available for symptomatic oHCM, there is still need for other treatment options that help target the root cause of the condition. In this study, researchers want to understand about the effects and long-term safety of aficamten in Japanese people with symptomatic oHCM.
The main purpose of the study is to learn how well aficamten works in Japanese with symptomatic oCHM.
For this, the researchers will check how participant's heart blood flow changes after 6 months of treatment. They do this by measuring the pressure needed for blood to leave the heart using a test called the left ventricular outflow tract (LVOT) gradient and a special breathing technique called Valsava maneuver.
Researchers will also look for:
* the number of participants who will have at least 1 level improvement on a scale doctors use to assess the effect of heart problems on daily activities after 3 and 6 months of treatment
* the change in the impact of heart problems on participant's daily lives based on their feedback on a questionnaire called Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score (KCCQ-CSS) after 3 and 6 months of treatment.
This study will have 2 treatment periods: main treatment period and long-term treatment period. During the main treatment period, participants will take aficamten tablets once daily by mouth for up to 6 months. After completing this period, the participants who can join the long-term treatment period will continue taking aficamten until the drug becomes commercially available in Japan or the study ends.
Each participant will be in the study as long as they benefit from the treatment.
Participants will visit the study site:
* once before the treatment starts
* 9 times with a gap of 2 to 4 weeks between the visits during treatment under the main treatment period, and in the long-term treatment period, participants will visit almost every 3 months until the treatment ends.
* then 2 more times with a gap of 1 month between the visits after the treatment ends.
During the study, the study doctors and their team will:
* check participant's health by performing tests such as blood and urine tests, and checking heart health using an electrocardiogram (ECG) and echocardiogram (ECHO)
* ask the participants questions about how they are feeling and what adverse events are they having
An adverse event is any medical problem that a participant has during a study. Study doctors keep track of all adverse events, irrespective if they think it is related or not to the study treatment. In addition, the participants will be asked to complete a questionnaire on quality of life at certain time points during the study.
If the participant benefits from the treatment, treatment with aficamten after the end of the study might be possible.

开始日期2025-06-20

申办/合作机构

Bayer AG

NCT06412666

/ Recruiting临床2/3期

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-

100 项与 Aficamten 相关的临床结果

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100 项与 Aficamten 相关的转化医学

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100 项与 Aficamten 相关的专利（医药）

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项与 Aficamten 相关的文献（医药）

2025-07-01·Cardiology in review

Aficamten—A Second in Class Cardiac Myosin Inhibitor for Hypertrophic Cardiomyopathy

Review

作者: Aronow, Wilbert S. ; Frishman, William H. ; Naidu, Srihari S. ; Wang, Andy ; Patel, Jay

Hypertrophic cardiomyopathy is an under-recognized disease with a genetic component that results in abnormal and often asymmetric thickening of the left ventricle in addition to decreased compliance and progressive fibrosis of the myocardium. It further poses significant complications related to dynamic left ventricular outflow obstruction over time in a significant majority. The medical management of obstructive hypertrophic cardiomyopathy has evolved over the decades as our understanding has grown. Traditionally, the mainstay in management has included the use of various negative inotropic agents. In contrast, the cardiac myosin inhibitors, aficamten and mavacamten, are novel therapies targeting cardiac contractility at the sarcomere level that have demonstrated improvement in clinical outcomes for patients, and mavacamten (Bristol Myers Squibb, Inc.) has now been approved by the Food and Drug Administration for the treatment of symptomatic obstructive HCM. Aficamten (Cytokinetics, Inc.) is the second in class cardiac myosin inhibitor that is currently being evaluated in ongoing phase III clinical trials, and is the subject of this review.

2025-06-01·INTERNATIONAL JOURNAL OF TOXICOLOGY

A Characterization of the Nonclinical Pharmacology and Toxicology of Aficamten, a Reversible Allosteric Inhibitor of Cardiac Myosin

Article

作者: Wang, Jingying ; Chin, Eva R. ; Hwee, Darren T. ; Winters, Brett R. ; Malik, Fady I. ; Morgan, Bradley P. ; Pugsley, Michael K.

Aficamten (CK-3773274) is a cardiac myosin inhibitor in development for the treatment of hypertrophic cardiomyopathy (HCM), a commonly inherited heart condition often characterized as a disease of the sarcomere. Aficamten reduces pathologic cardiac hypercontractility by selectively binding to an allosteric site on cardiac myosin. To characterize the pharmacology and toxicology of aficamten, a series of nonclinical repeated dose studies were conducted. In a 10-day repeated dose pharmacology study in Sprague Dawley rats, aficamten produced dose-dependent reductions in left ventricular fractional shortening (FS) which were fully reversible within 24 h. Aficamten did not change the ratio of heart weight to tibia length (HW/TL) or left ventricular posterior wall (LVPW) thickness at any dose tested. At a supratherapeutic dose of 6 mg/kg/day, there was a significant increase in interventricular septum (IVS) thickness. Aficamten did not affect mRNA expression of the cardiac injury biomarkers BNP, β-MHC, or ANP. In repeated dose Good Laboratory Practice (GLP) regulatory toxicology studies in Sprague Dawley rats for up to 6 months and beagle dogs for up to 9 months, the primary adverse findings at supratherapeutic doses were consistent across all studies and observed in the heart consisting of atrial/ventricular dilatation that correlated with increased heart weights. These findings were largely reversible and consistent with excessive on-target pharmacology associated with cardiac myosin inhibition. The reversible nature of aficamten-associated adverse effects is supportive of its clinical safety as this property suggests that these findings, should they occur in humans, may also be reversible, limiting long-term human cardiac risk.

2025-06-01·Annals of Medicine and Surgery

Cardiac myosin inhibitors for hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomized controlled trials

Review

作者: Khalid, Saif ; Ullah, Waheed ; Lwin, Nang Phyu Thant ; Ehsan, Muhammad ; Gupta, Akhil ; Alsubari, Asma’a Munasar Ali ; Umer, Mohammad ; Iqbal, Sohaba ; Korpal, Mayank ; Kumar, Rajanikant ; Ahmad, Adeel ; Haider, Faseeh ; Hassan, Ebaad ; Ahsan, Maria ; Hasana, Uswa

Background/objective::

HCM is a structural disorder of the myocardium that leads to sudden cardiac death in young adults. We synthesized an updated understanding of the role of Cardiac Myosin Inhibitors (CMIs) in HCM by pooling data from RCTs.

Methods::

We identified six published RCTs, involving 826 participants. Data were extracted pertaining to study characteristics; primary outcomes of interest–(1) change from baseline in resting left ventricular outflow tract (LVOT) peak gradient, (2) change from baseline in Valsalva LVOT peak gradient, and (3) improvement of ≥1 NYHA class–and secondary outcomes. These were pooled using Review Manager 5.4, employing a random-effects model, and reported as odds ratios (ORs) or mean differences (MDs).

Results::

We found statistically significant between-group difference favoring CMIs in change from baseline in LVOT peak gradient: at rest (MD −39.33; −53.01 to −25.64), post-Valsalva (MD −48.99; −53.96 to −44.03), and post-exercise (MD −37.11; −44.34 to −29.87); ≥1 NYHA class improvement (OR 4.10; 2.79–6.02), change from baseline in peak oxygen uptake (MD −37.11; −44.34 to −29.87), LVOT gradient ≤30 mm hg (RR 14.89; 7.47–29.67), participants eligible for septal reduction therapy (RR 0.26; 0.18–0.36), and change from baseline in KCCQ-CSS score (MD 8.54; 5.36–11.71). Subgrouping by intervention type (mavacamten vs. aficamten) revealed non-significant results for all primary outcomes.

Conclusions::

CMIs can contribute to improving key efficacy outcomes for patients with HCM while reducing incidence of SRT.

311

项与 Aficamten 相关的新闻（医药）

2025-05-28

·VIP说

新型肌球蛋白抑制剂是否足够安全？心肌病新药Aficamten审批遇阻

引言美国东部时间5月1日，Cytokinetics公司宣布，美国食品药品监督管理局（FDA）已将Aficamten用于治疗梗阻性肥厚型心肌病（oHCM）的新药申请（NDA）的《处方药用户付费法案》（PDUFA）目标行动日期延长至2025年12月26日。FDA通知Cytokinetics，需要更多时间对其提出的风险评估与缓解策略（Risk Evaluation and Mitigation Strategy，REMS）进行全面审查。（REMS是FDA针对一些具有严重安全问题的处方药和生物制品要求的药物风险管理计划，以帮助确保药物的获益大于其风险。）早在2024年12月，Cytokinetics就向FDA提交了Aficamten的新药申请（NDA），FDA原定于2025年9月26日完成审批。据悉，Cytokinetics与FDA在NDA前讨论中已涉及安全性和风险缓解措施，并提交了不附带REMS的Aficamten oHCM NDA且获受理。然而，近期NDA审查期间，FDA基于Aficamten的固有特性要求公司提交REMS。此次提交的REMS被认定为对NDA的重大修订，因此原PDUFA日期将标准延长三个月。患者安全考量或为关键因素此前，Aficamten在关键III期SEQUOIA-HCM试验（针对梗阻性HCM患者）中取得积极结果。但在近期召开的2025年美国心脏病学会（ACC）年会上，Cytokinetics发布的一项开放标签、固定序列的药物相互作用（DDI）研究显示，Aficamten的代谢涉及多种细胞色素P450（CYP）酶途径。具体而言：CYP2C9贡献50%代谢（代谢分数[fm]=50%），其次为CYP3A（26%）、CYP2D6（21%）和CYP2C19（3%）。这意味着，Aficamten主要通过CYP2C9代谢，与强效CYP抑制剂联用时需关注药效变化，与某些细胞色素P450抑制剂合用或停用某些细胞色素P450诱导剂可能会增加因收缩功能障碍导致心力衰竭的风险。基于此，业界推测，此类药物通过抑制心肌收缩力发挥作用，但存在过度放松心脏肌肉的风险（如引发心衰），因此需通过REMS严格监控使用条件（如定期超声心动图检查、特殊药房分发等）。FDA推迟Aficamten的PDUFA可能是基于对其药物相互作用风险与患者安全的考量，因此需要Cytokinetics充分考虑用药安全并提供全面的REMS。股价应声下跌受此消息影响，Cytokinetics公司股价当日下跌超11%。此后更是从5月1日最高点42.92美元，跌至近期低点29.31美元（5月15日），跌幅高达约32%。 Evercore ISI的分析师对此感到困惑，并在致客户的报告中写道：“我们完全没预料到这一点。最令我们惊讶的是，Aficamten的NDA最初竟未附带REMS计划。这是FDA对公司的指导失误，还是某种沟通不畅？鉴于申请已被受理，我们倾向于认为是前者。” Cytokinetics总裁兼首席执行官Robert Blum也在声明中表示。“我们期待继续与FDA就Aficamten的NDA进行建设性沟通。” 但近年来FDA在创新药的上市审批上日渐趋严，以高标准、高要求将多款创新药被拒之门外。未来，FDA 将如何应对这些问题，生物制药行业又将何去何从，都充满了不确定性，值得持续关注。

引进/卖出临床3期临床结果临床1期

2025-05-18

·药事纵横

再获突破性进展！HCM患者将迎来新希望

2025年5月13日，生物制药公司Cytokinetics宣布，其新药Aficamten的Ⅲ期临床试验MAPLE-HCM已达到主要终点，结果显示，与标准β受体阻滞剂美托洛尔相比，Aficamten显著改善了梗阻性肥厚型心肌病(HCM)患者的峰值摄氧量(pVO2)。该药被行业媒体Evaluate列为2025年的十款潜在重磅疗法之一。【关于Aficamten】Aficamten是一种心肌肌球蛋白抑制剂，是一种新型药物，旨在减少每个心动周期中活性肌动蛋白-肌球蛋白横桥的数量，从而抑制与肥厚型心肌病相关的心肌收缩过度。在临床前模型中，该药物通过在独特且选择性的变构结合位点直接与心肌肌球蛋白结合来降低心肌收缩力，从而阻止肌球蛋白进入产生力的状态。【临床研究效果显著】这项随机、双盲、活性对照MAPLE-HCM试验(ClinicalTrials.gov注册号：NCT05767346)纳入了175例有症状的肥厚型心肌病和左心室流出道阻塞患者（纽约心脏协会心功能分级II或III级；堪萨斯城心肌病问卷临床总结评分在35至90分之间）。筛选时，试验参与者的静息左心室流出道梯度(LVOT-G)为30 mmHg或更高和/或瓦尔萨尔瓦指压后LVOT-G为50 mmHg或更高，左心室射血分数至少为60%，呼吸交换率至少为1.05，且峰值摄氧量(pVO2)低于预测值的100%。患者按1:1的比例随机分配接受Aficamten或美托洛尔治疗。主要终点是通过心肺运动测试测量的从基线到第24周的pVO2变化。研究结果表明，该试验达到了其主要终点，证明了Aficamten在提高峰值运动能力方面优于美托洛尔。此外，安全性数据分析显示，与美托洛尔相比，Aficamten具有更佳的疗效。此前，上市申请基于3期SEQUOIA-HCM试验(ClinicalTrials.gov 标识符:NCT05186818)，该项研究纳入了282名患有症状性肥厚型心肌病和左心室流出道阻塞的成年人。结果显示，与安慰剂相比，Aficamten治疗24周显著提高了运动能力，与基线相比，Aficamten治疗的患者通过心肺运动试验(CPET)测得的最大摄氧量(pVO2)增加了1.8 ml/kg/min，而安慰剂治疗的患者为0.0mL/kg/min(1.74mL/kg/min[1.04-2.44]；p=0.000002)。在所有10个预先指定的次要终点，包括Valsalva左心室流出道(LVOT)梯度、纽约心脏协会(NYHA)功能分级、堪萨斯城心肌病临床综合评分(KCCQ-CSS)、LVOT梯度< 30 mmHg的比例(分别在第12周和第24周)、符合间隔缩小治疗指南的持续时间(SRT)和第24周CPET期间的总工作量，均观察到统计学显著改善。SEQUOIA-HCM试验的进一步分析表明，Aficamten治疗与心脏重塑以及心脏结构、功能和生物标志物的改善有关，且不会对收缩功能产生负面影响。这些结果首次证明Aficamten可作为单药疗法，为梗阻性肥厚型心肌病患者带来具有临床意义的改善，重要的是，MAPLE-HCM的阳性结果为这种潜在新药与现有标准治疗方案相比的益处提供了重要的背景信息。【展望】FDA目前正在审查Aficamten用于治疗梗阻性肥厚型心肌病(HCM)的新药申请，预计将于2025年12月26日做出监管决定。同时，该药物也正在接受欧洲和中国的监管审查。此外，Aficamten还有几项临床试验，分别是3期ACACIA-HCM临床试验，比较了Aficamten作为单药治疗阻塞性HCM患者的效果；针对患有阻塞性HCM的儿科人群进行的CEDAR-HCM临床试验；针对HCM患者进行的FOREST-HCM开放标签扩展临床研究。除了HCM，Cytokinetics还在开发心肌激活剂omecamtiv mecarbil，用于治疗射血分数严重降低(HFrEF)的心力衰竭患者；CK-586，一种作用机制不同于Aficamten的心脏肌球蛋白抑制剂，用于治疗射血分数保留的心力衰竭(HFpEF)；以及CK-089，一种快速骨骼肌肌钙蛋白激活剂(FSTA)，用于治疗特定类型的肌营养不良症。参考来源：[1] Cytokinetics官网[2] Cytokinetics announces positive topline results from MAPLE-HCM. News release. Cytokinetics. May 13, 2025.[3] Cytokinetics announces FDA acceptance of New Drug Application for aficamten for the treatment of obstructive hypertrophic cardiomyopathy. News release. Cytokinetics. December 2, 2024.药事纵横投稿须知：稿费已上调，欢迎投稿

引进/卖出临床3期临床结果临床1期突破性疗法

2025-05-18

·ir.cytokinetics.com

Cytokinetics Presents Additional Data Related to Aficamten at the European Society of Cardiology Heart Failure 2025 Congress

Two New Analyses from SEQUOIA-HCM on Effect of Aficamten Between Patients with Mild and Moderate-to-Severe Symptoms, and Across Geographic Regions HEOR Analyses of Real-World Data Reveal Disparities in Outcomes In Females and Older Patients with Non-Obstructive HCM SOUTH SAN FRANCISCO, Calif., May 18, 2025 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that additional data arising from two analyses from SEQUOIA-HCM, (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM), and results from a real-world analysis related to non-obstructive HCM were presented at the European Society of Cardiology Heart Failure 2025 Congress. The results from an analysis of the efficacy of aficamten in patients with obstructive HCM and mild symptoms in SEQUOIA-HCM were also simultaneously published in The European Heart Journal.1 “These analyses from SEQUOIA-HCM demonstrate that the effect of aficamten on exercise capacity, symptoms, hemodynamics and cardiac biomarkers is consistent in patients with obstructive HCM regardless of baseline symptom severity and geographic region,” said Stephen Heitner, M.D., Vice President, Head of Clinical Research. “These findings are informative as we continue to explore the potential application of aficamten across a range of patient phenotypes in obstructive HCM.” Effect of Aficamten in Patients with Mild Symptoms and Moderate-to-Severe Symptoms Data from an additional analysis from SEQUOIA-HCM related to the effect of aficamten in patients with mild symptoms were presented in a Late Breaking Clinical Trial session and simultaneously published in The European Heart Journal. Patients in SEQUOIA-HCM (n=282) were divided into two groups according to baseline symptom severity: mild symptoms, defined as New York Heart Association (NYHA) Functional Class II and Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) ≥80 (n=118; 62 randomized to aficamten), and moderate-to-severe symptoms defined as NYHA Functional Class II/III/IV and KCCQ-CSS <80 (n=150; 71 randomized to aficamten). The effect of aficamten on the primary endpoint of change from baseline to Week 24 in peak oxygen uptake (pVO2) was similar between symptom groups (1.6 and 1.8 mL/kg/min in mild and moderate-to-severe symptom groups respectively; interaction p=0.8). While both groups experienced improvements in KCCQ-CSS, the magnitude of improvement was greater in the moderate-to-severe symptom group compared to the mild symptom group (interaction p=0.02) as expected given the lower baseline KCCQ-CSS score. At the end of the treatment period, 54% of patients with mild symptoms and 36% of patients with moderate-to-severe symptoms were asymptomatic. Additionally, more than half of patients in both groups had an improvement of at least one NYHA Functional Class (interaction p=0.6). Improvements in resting and Valsalva left ventricular outflow tract (LVOT) gradients and NT-proBNP also did not differ significantly between the two groups (all interaction p≥0.3). The safety and tolerability profile of aficamten was similar to placebo in both subgroups. These data indicate that, in SEQUOIA-HCM, the effect of aficamten was observed independent of baseline symptom burden in patients with obstructive HCM. Effect of Aficamten in Patients with Obstructive HCM Consistent Across Geographic Regions Data from an additional analysis from SEQUOIA-HCM evaluating geographical differences in the effect of aficamten were presented in a Late Breaking Clinical Trial session. Participants from SEQUOIA-HCM (n=282) were classified into three geographic regions: Europe, including Israel (n=142; 50%), North America (n=94; 33%) and China (n=46; 16%). At baseline, patients in Europe and North America were on average older, with a higher BMI, lower LVOT gradients, lower KCCQ-CSS and were more likely to have comorbidities than those in China. A greater proportion of patients in North America were NYHA Functional Class III/IV compared to Europe and China. Peak VO2, Valsalva LVOT gradient, NT-proBNP and hs-cardiac troponin I were similar across geographical regions. The distribution of doses of aficamten was similar across regions. The effect of aficamten on the primary endpoint of change in pVO2 and all secondary endpoints was consistent, with no significant differences across regions (all interaction p>0.15). The incidence of serious adverse events was similar in the aficamten and placebo groups across regions, and occurrences of left ventricular ejection fraction (LVEF) <50% were infrequent. These results demonstrate that in SEQUOIA-HCM, while there were regional differences in patient baseline characteristics, the dosing, safety profile and effect of aficamten was consistent across the geographic regions studied. Analyses of Real-World Data Reveal Association Between Age, Sex and Cardiovascular Outcomes in Patients with Non-Obstructive HCM Data presented from a health economics and outcomes research (HEOR) analysis evaluated the association between age, sex and cardiovascular outcomes in patients with non-obstructive HCM. This retrospective cohort study included adult patients diagnosed with non-obstructive HCM from January 1, 2013 to December 31, 2021 using real-world data from Optum Market Clarity database. Of the 9,842 patients included, 46.2% were female, 53.8% were male and the age distribution was as follows: 24.2% were ages 55 to 64 years, 22.1% were ages 75 or older, 22.1% were ages 65 to 74, 19.9% were ages 40 to 54 and 11.7% were ages 18 to 39. Female patients had increased rates of stroke (risk ratio [RR] 1.32), heart failure (RR 1.22), cardiovascular hospitalization (RR 1.23) and cardiovascular rehospitalization (RR 1.15) compared to male patients (all p<0.01). However, female patients were less likely to have atrial fibrillation (RR 0.83) and ventricular tachycardia (RR 0.69) (p<0.001). Compared to patients aged 75 years or older, younger patients were less likely to have atrial fibrillation, stroke, heart failure, cardiovascular hospitalization and cardiovascular rehospitalization (all p<0.001). All-cause mortality was significantly greater in female patients compared to male patients (p=0.002). Patients aged 75 years or older had the highest all-cause mortality (16.6%; p<0.001), followed by patients aged 65 to 74 (8.3%), 55 to 64 (3.5%), 40 to 54 (3.1%) and 18 to 39 years (1.4%). These findings highlight disparities in morbidity and survival among females and older patients with non-obstructive HCM, emphasizing the potential role for novel treatments to help reduce the clinical burden. About Aficamten 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.2 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 evaluated in SEQUOIA-HCM, a positive pivotal Phase 3 clinical trial in patients symptomatic obstructive hypertrophic cardiomyopathy (HCM). Aficamten received Breakthrough Therapy Designation for the treatment of symptomatic HCM from the U.S. Food & Drug Administration (FDA) and for the treatment of symptomatic obstructive HCM from the National Medical Products Administration (NMPA) in China. 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. This communication contains a summary of new data related to the clinical development of aficamten presented at the European Society of Cardiology Heart Failure 2025 Congress. Aficamten is an investigational drug and is not approved by any regulatory agency. Its safety and efficacy have not been established. Aficamten is currently under regulatory review in the U.S, where the FDA is reviewing a New Drug Application (NDA) for aficamten 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. About Hypertrophic Cardiomyopathy Hypertrophic cardiomyopathy (HCM) is a disease in which the heart muscle (myocardium) becomes abnormally thick (hypertrophied). The thickening of cardiac muscle leads to the inside of the left ventricle becoming smaller and stiffer, and thus the ventricle becomes less able to relax and fill with blood. This ultimately limits the heart’s pumping function, resulting in reduced exercise capacity and symptoms including chest pain, dizziness, shortness of breath, or fainting during physical activity. HCM is the most common monogenic inherited cardiovascular disorder, with approximately 280,000 patients diagnosed, however, there are an estimated 400,000-800,000 additional patients who remain undiagnosed in the U.S.3,4,5 Two-thirds of patients with HCM have obstructive HCM (oHCM), where the thickening of the cardiac muscle leads to left ventricular outflow tract (LVOT) obstruction, while one-third have non-obstructive HCM (nHCM), where blood flow isn’t impacted, but the heart muscle is still thickened. People with HCM are at high risk of also developing cardiovascular complications including atrial fibrillation, stroke and mitral valve disease.6 People with HCM are at risk for potentially fatal ventricular arrhythmias and it is one of the leading causes of sudden cardiac death in younger people or athletes.7 A subset of patients with HCM are at high risk of progressive disease leading to dilated cardiomyopathy and heart failure necessitating cardiac transplantation. About Cytokinetics 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. For additional information about Cytokinetics, visit www.cytokinetics.com and follow us on X, LinkedIn, Facebook and YouTube. Forward-Looking Statements This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”). Cytokinetics disclaims any intent or obligation to update these forward-looking statements and claims the protection of the Act’s Safe Harbor for forward-looking statements. Examples of such statements include, but are not limited to, statements express or implied relating to the properties, treatment effect or potential benefits of aficamten or any of our other drug candidates or our ability to obtain regulatory approval for aficamten for the treatment of obstructive hypertrophic cardiomyopathy or any other indication from FDA or any other regulatory body in the United States or abroad by any particular date, if ever. Such statements are based on management’s current expectations, but actual results may differ materially due to various risks and uncertainties, including, but not limited to the risks related to Cytokinetics’ business outlines in Cytokinetics’ filings with the Securities and Exchange Commission. Forward-looking statements are not guarantees of future performance, and Cytokinetics’ actual results of operations, financial condition and liquidity, and the development of the industry in which it operates, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that Cytokinetics makes in this press release speak only as of the date of this press release. Cytokinetics assumes no obligation to update its forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release. CYTOKINETICS® and the CYTOKINETICS and C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries. Contact: Cytokinetics Diane Weiser Senior Vice President, Corporate Affairs (415) 290-7757 References: Source: Cytokinetics, Incorporated

临床3期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

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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

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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


NCT05767346 (MAPLE-HCM, GlobeNewswire \| NEWS) 人工标引	临床3期	梗阻性肥厚型心肌病	-	Aficamten	獵網窪窪齋廠糧鬱糧鏇(遞壓艱顧蓋憲憲觸醖廠) = 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. 壓餘齋顧鏇餘膚網醖觸 (壓選遞遞壓衊積鑰齋艱 ) 达到	积极	2025-05-13
				Metoprolol
NCT05186818 (SEQUOIA-HCM, GlobeNewswire) 人工标引	临床3期	肥大型心肌病	-	Aficamten	鹽醖襯積積窪壓艱壓衊(繭齋製蓋繭繭鹹壓願範) = 鏇憲憲獵鹽夢範網遞願鬱顧網顧積鑰繭選醖繭 (網糧襯製醖鏇淵餘簾憲, 3.1) 更多	积极	2024-11-16
				Placebo	鹽醖襯積積窪壓艱壓衊(繭齋製蓋繭繭鹹壓願範) = 鏇鑰鹽網顧醖鏇鬱願積鬱顧網顧積鑰繭選醖繭 (網糧襯製醖鏇淵餘簾憲, 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	顧齋鹽築夢糧衊齋鏇鬱(選醖範製膚構夢簾廠窪) = 鹽餘範糧襯製鏇鹽獵範鹽夢糧鑰網餘壓繭餘鏇 (願遞鑰糧膚顧壓繭醖憲 ) 更多	积极	2024-11-01
				Placebo	顧齋鹽築夢糧衊齋鏇鬱(選醖範製膚構夢簾廠窪) = 願醖築顧壓顧淵鏇蓋簾鹽夢糧鑰網餘壓繭餘鏇 (願遞鑰糧膚顧壓繭醖憲 ) 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 (NT-proBNP)	282	Aficamten	遞夢艱遞範醖憲艱醖選(糧願遞顧餘積積窪鏇鬱) = 鏇鬱顧醖鹹壓艱顧簾簾廠繭製築築窪襯觸製廠 (鹹蓋糧願襯顧壓膚壓築 ) 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	282	Aficamten	廠鹽淵簾醖製範壓顧憲(選範範壓築積夢繭鬱鏇) = 構壓衊網鏇製壓窪選鑰簾範網鏇壓鹽餘簾獵齋 (淵鹹網憲膚製繭簾餘鑰, -25 ~ -6) 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	蓋製觸壓壓繭觸窪醖獵(獵襯遞積憲製選淵廠選): 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)	觸製繭艱壓淵築獵膚觸(淵鬱夢選築壓製獵膚壓) = 築獵鹽壓餘積網蓋觸餘觸壓醖膚鏇膚網積製淵 (淵築糧積夢觸遞窪觸鹹 ) 更多	积极	2024-09-01
				Aficamten (Placebo-controlled pool)	觸製繭艱壓淵築獵膚觸(淵鬱夢選築壓製獵膚壓) = 襯觸鏇艱鏇鑰衊鹽醖遞觸壓醖膚鏇膚網積製淵 (淵築糧積夢觸遞窪觸鹹 ) 更多
NCT04219826 \| NCT04848506 \| NCT05186818 (ESC2024)	N/A	梗阻性肥厚型心肌病	-	Aficamten	繭鹹廠製築積選獵積積(餘艱獵鹹網鑰獵鹽觸窪) = 鑰齋鑰範醖顧襯遞淵選觸窪蓋襯壓艱鏇鏇製積 (窪壓鹽壓蓋窪齋獵窪齋 ) 更多	-	2024-09-01
				Placebo	繭鹹廠製築積選獵積積(餘艱獵鹹網鑰獵鹽觸窪) = 獵願鹹獵衊顧窪鬱構廠觸窪蓋襯壓艱鏇鏇製積 (窪壓鹽壓蓋窪齋獵窪齋 ) 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	遞衊選窪網願觸顧顧選(憲獵觸遞衊廠願觸遞簾) = 壓鹹鑰鹹窪艱顧衊鹹窪餘餘製顧膚齋餘憲範積 (觸餘廠鹽廠觸網積艱鏇, 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	構蓋觸夢鹹網壓廠憲襯(鏇廠壓鬱艱製窪範鬱願) = none 觸蓋獵製遞憲觸構齋構 (鏇膚艱鬱遞夢艱顧築夢 ) 更多	积极	2024-04-05