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2025-06-30

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

/ Completed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

100 项与阿夫凯泰相关的临床结果

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100 项与阿夫凯泰相关的转化医学

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100 项与阿夫凯泰相关的专利（医药）

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103

项与阿夫凯泰相关的文献（医药）

2026-04-01·AMERICAN JOURNAL OF CARDIOLOGY

Mavacamten and Aficamten in Hypertrophic Cardiomyopathy: A Systematic Review and Meta-Analysis of Randomized Trials

Article

作者: Salciccioli, Louis ; Khouri, Peter ; Fahim, Md ; Gorantla, Asher ; Wang, Roy ; Eldawud, Daoud ; Wahadneh, Omar ; Sharma, Nischal

Myosin inhibitors represent a novel therapeutic class for hypertrophic cardiomyopathy (HCM). While both mavacamten and aficamten have been evaluated in randomized trials, comparative evidence across the 2 agents remains limited. We performed a systematic review and meta-analysis of 8 randomized controlled trials of myosin inhibitors in HCM. Outcomes included improvement in the New York Heart Association (NYHA) functional class, Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS), postexercise and resting left ventricular outflow tract (LVOT) gradients, mean rest LVOT peak gradient, mean Valsalva LVOT peak gradient, change in left ventricular ejection fraction (LVEF), peak oxygen uptake, N-terminal pro-B-type natriuretic peptide (NT-proBNP), treatment-emergent adverse events, and serious adverse events. Pooled effects were estimated twice using random-effects models, once stratified by drug and once pooled altogether. Results showed that myosin inhibitors improved clinical and hemodynamic outcomes versus placebo. Both mavacamten and aficamten yielded improvements in New York Heart Association functional class and KCCQ-CSS, while reducing LVOT gradients and proBNP levels. Effect magnitudes were generally larger with mavacamten; aficamten demonstrated concordant but smaller effects and was the only agent to increase peak oxygen uptake. Safety was favorable for both agents, with pooled estimates indicating a marginally more favorable safety profile for aficamten. In conclusion, myosin inhibitors improve functional status, quality of life, and hemodynamics in HCM with an acceptable safety profile. Mavacamten generally demonstrates more robust benefits, while aficamten appears to have a more favorable safety profile. Additional trials directly comparing these agents are warranted to better clarify relative efficacy and safety.

2026-03-01·JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Aficamten in Obstructive Hypertrophic Cardiomyopathy

Article

作者: Costabel, Juan Pablo ; Claggett, Brian L ; Kulac, Ian J ; de Barros Correia, Edileide ; Bilen, Ozlem ; Schulze, P Christian ; Wohltman, Amy ; Solomon, Scott D ; Peña-Peña, Maria Luisa ; Reant, Patricia ; Lakdawala, Neal K ; Garcia-Pavia, Pablo ; Heitner, Stephen B ; Lewis, Gregory D ; Kupfer, Stuart ; Mann, Amy ; Malik, Fady I ; Poulsen, Steen H ; Masri, Ahmad ; Hegde, Sheila M ; Burroughs, Melissa ; Sohn, Regina ; Barriales-Villa, Roberto ; Berhane, Indrias ; Fifer, Michael A ; Maron, Martin S ; Nassif, Michael E ; Merkely, Bela ; Nair, Ajith ; Elliott, Perry ; Wang, Andrew ; Jacoby, Daniel L ; Dybro, Anne

BACKGROUND:

For >60 years, beta-blockers have been first-line therapy for symptomatic obstructive hypertrophic cardiomyopathy (oHCM), although limited data support this recommendation. The MAPLE-HCM trial showed greater improvement in exercise capacity and secondary endpoints for aficamten monotherapy vs metoprolol monotherapy.

OBJECTIVES:

The purpose of this study was to evaluate the effects of aficamten vs metoprolol across multiple clinically relevant and patient-centric measures of disease burden in oHCM.

METHODS:

Patients with oHCM and left ventricular outflow tract gradient (LVOT-G) ≥30 mm Hg at rest and/or ≥50 mm Hg with Valsalva were randomly assigned to aficamten (n = 88) or metoprolol (n = 87). Dose titration was based on vital signs and echocardiograms. Assessment of clinical response by treatment arm was based on improvement in multiple parameters reflecting oHCM disease burden: LVOT-G <30 mm Hg at rest and <50 mm Hg with Valsalva; ≥1 class improvement in NYHA functional class and/or ≥10-point improvement in Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score (KCCQ-CSS); ≥50% reduction in N-terminal pro-B-type natriuretic peptide; ≥1.0 mL/kg/min increase in peak oxygen consumption (pVO₂); and ≥10% reduction in left atrial volume index. Clinical response was classified by number of efficacy outcomes achieved: nonresponder (none), limited (1 or 2), positive (3 or 4), or complete (all 5).

RESULTS:

Baseline characteristics were similar for both treatment groups. For the overall cohort, mean age was 58 ± 13.2 years; 73 (42%) were women, 103 (70%) were in NYHA functional class II and 52 (30%) in class III, mean pVO₂ was 19.9 ± 5.1 mL/kg/min, and mean KCCQ-CSS score was 66 ± 16. At 24 weeks, aficamten treatment was associated with greater benefits in all efficacy outcome measures, with numbers needed to treat ranging from 1.5 to 5.0 relative to metoprolol treatment. Patients treated with aficamten were more likely to be either positive or complete responders compared with patients treated with metoprolol (78% vs 3%, respectively; P < 0.001).

CONCLUSIONS:

In patients with symptomatic oHCM, aficamten monotherapy led to superior and rapid treatment benefits across several clinically relevant outcome measures of disease burden compared with metoprolol. These observations support the emerging role of aficamten as monotherapy for oHCM. (Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Metoprolol Succinate in Adults With Symptomatic oHCM [MAPLE-HCM]; NCT05767346).

2026-03-01·JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Aficamten vs Metoprolol: A Turning Point in Obstructive Hypertrophic Cardiomyopathy Care?

作者: Heidecker, Bettina ; Olivotto, Iacopo

594

项与阿夫凯泰相关的新闻（医药）

2026-04-01

·药研苑

恒瑞新一代梗阻性肥厚型心肌病药物HRS-1893二期研究发布

药品营销避坑”试读：药品导入期管理视频课（试听）药品生命周期管理（案例）药品立项需要注意什么（案例） 2026年3月31日，恒瑞医药宣布在第75届美国心脏病学会年度科学会议（ACC.26）公布HRS-1893（BHB-1893）用于梗阻性肥厚型心肌病（oHCM）受试者2期临床试验的研究数据。该研究为多中心、随机、开放标签2期临床试验，共纳入42例受试者。入组患者按1:1:1比例随机分为组1（起始剂量为20mg，每日给药2次，可滴定至60mg）、组2（起始剂量为40mg，每日给药2次，可滴定至80mg）、组3（起始剂量为40mg，每日给药1次，可滴定至120mg）。根据受试者的LVEF（左心室射血分数）与Valsalva LVOT-G（瓦氏动作下左心室流出道压差）水平进行剂量调整。至第12周，各组Valsalva LVOT-G完全缓解率（<30 mmHg）为50%~86%，LVEF降幅仅1.8%~2.7%。第5天，各组平均Valsalva LVOT-G均可降至30 mmHg以下。所有42例受试者在随机阶段结束后，进入开放标签延长期（OLE）阶段，按组2的给药剂量持续用药。至第39周，Valsalva LVOT-G完全缓解率（<30mmHg）达到88%。在安全性方面，HRS-1893整体耐受性良好，不良事件均为轻至中度，无不良事件导致治疗中断或停药。所有受试者的LVEF均不低于55%。肥厚型心肌病（HCM）主要由于编码肌小节相关蛋白基因致病性变异导致的、或病因不明的以心肌肥厚为特征的心肌病，左心室壁受累常见，需排除其他的心血管疾病或全身性、代谢性疾病引起的心室壁增厚。根据血流动力学特点可分为梗阻性HCM和非梗阻性HCM。HCM的患病率据估计至少为1/200。β受体阻滞剂是梗阻性HCM的主要治疗药物。 HRS-1893为Myosin（肌球蛋白）选择性抑制剂。Myosin通过与肌动蛋白相互作用，水解ATP，将化学能转化为机械能。HRS-1893通过特异性抑制心肌肌球蛋白ATP酶活性，使心肌收缩性能正常化，减少左心室肥厚并改善舒张期顺应性。目前国内已批准Myosin选择性抑制剂阿夫凯泰（Aficamten）：用于治疗纽约心脏协会（NYHA）心功能分级 II-III 级的梗阻性肥厚型心肌病（HCM）成人患者，以改善运动能力和症状。推荐指数：★★★ 推荐原因：HRS-1893目前进展顺利，该领域的品种较少，竞争有限。该类药物有望替代β受体阻滞剂成为梗阻性HCM的一线治疗药物。相关阅读： 81.3%，迪哲发布舒沃替尼治疗EGFR PACC等突变2期试验数据阿斯利康1型神经纤维瘤病药物司美替尼获批用于成人翰森制药伊奈利珠单抗新增获批治疗成人全身型重症肌无力全国第二家，江正药业贝他斯汀口崩片获批上市风已满楼，比奇珠单抗在华获批用于银屑病及化脓性汗腺炎自带主角光环，德莫奇单抗用于重度嗜酸性粒细胞性哮喘在华获批终结者，特泽利尤单抗在华获批治疗重度哮喘和慢性鼻窦炎伴鼻息肉齐鲁抗慢性移植物抗宿主病药物甲磺酸贝舒地尔片首仿上市首款国产注射用重组A型肉毒毒素获批上市葛兰素史克重磅抗乙肝药物贝普若韦生申请上市第一三共德曲妥珠单抗序贯THP方案治疗早期乳腺癌在华获批 FDA批准诺和诺德周基础胰岛素依柯胰岛素用于2型糖尿病上海欣峰申请注射用氨曲南阿维巴坦首仿上市相关内容只作为药物研发的参信息。不构成任何与用药咨询有关的建议。如需发稿，您可通过在药研苑公众号下以发送信息的方式将官网或官方公众号的稿件链接和标题发送给我们。我们将择优确定是否发布。

2026-04-01

·久谦中台丨海外生科

Cytokinetics：肌球蛋白抑制剂验证后的战略纵深与边界

超过25年对肌肉收缩基本单元——肌节的研究，构成了Cytokinetics从研发型生物技术公司向商业化阶段转型的基础。该公司的核心价值在于其肌肉调节平台，通过小分子药物直接干预作为分子马达的肌球蛋白与肌钙蛋白，从而精准调节肌肉收缩力。其领先资产Aficamten在症状性梗阻性肥厚型心肌病领域的成功获批，标志着该平台在心血管领域的科学可行性已获确证。然而，公司在神经肌肉疾病领域的探索遭遇了明确挫折。本文旨在评估其技术平台的独特性、核心资产的差异化定位、管线扩展的商业逻辑，并剖析其面临的关键临床与商业化风险。核心平台：直接靶向肌节动力学的机制优势 Cytokinetics的肌节动力学调节策略提供了区别于传统间接路径的精准干预方式，其科学基础在于对肌肉超松弛状态或动力增强的调控。长期以来，针对心力衰竭或肥厚型心肌病的药物干预主要依赖肾上腺素能受体阻滞剂或钙通道阻滞剂等间接路径。此类方法能缓解症状，但常伴随副作用且无法直接纠正肌肉收缩的分子缺陷。Cytokinetics的平台则直接干预肌节内肌球蛋白与肌动蛋白的相互作用循环，其优势体现在降低或增强收缩力两个方向。在需要增强收缩力的疾病中，肌球蛋白/肌钙蛋白激活剂以不加钙方式增强动力。针对收缩力不足的情况，公司的激活剂策略旨在不增加细胞内钙离子浓度的前提下提升肌肉性能。心肌肌球蛋白激活剂Omecamtiv Mecarbil通过增加肌球蛋白向产生动力的结合状态转变的速率来增强收缩。快骨骼肌肌钙蛋白激活剂则通过减缓钙离子从肌钙蛋白复合物中的解离速率，使骨骼肌对神经输入更敏感。此类不加钙的强化方式，在理论上避免了传统强心药可能引发的室性心律失常风险。然而，这一理论优势尚未在关键III期临床试验中被完全验证。 Aficamten在oHCM领域的差异化优势基于SEQUOIA-HCM与MAPLE-HCM两项关键试验的数据，Aficamten确立了其在oHCM治疗中相对于首个获批同类药物Mavacamten的差异化临床定位。 Aficamten的获批主要基于III期SEQUOIA-HCM试验的数据，而活性对照研究MAPLE-HCM则进一步明确了其相对于标准治疗的优越性。与Mavacamten相比，Aficamten在滴定速度、药物相互作用风险和特定安全性事件管理上表现出潜在优势，这些差异源于其独特的分子设计和代谢途径。 MAPLE-HCM活性对照研究确立了Aficamten相对于β-受体阻滞剂的优效性。该研究直接对比了Aficamten与美托洛尔单药治疗的疗效。结果显示，在改善最大摄氧量和缓解症状方面，Aficamten显著优于美托洛尔。值得注意的亚组分析显示，无论患者是否伴有高血压病史，Aficamten均表现出一致的疗效。与Mavacamten的横向对比揭示了Aficamten在临床实践中的潜在便利性与安全性优势。尽管两者均属心肌肌球蛋白抑制剂，但分子层面的差异导致了不同的临床特征。 Aficamten半衰期更短，可在6-8周内完成剂量滴定达到稳态；Mavacamten通常需要12周。在代谢方面，Mavacamten严重依赖CYP2C19酶途径，与许多常用药物存在显著的相互作用风险；Aficamten的代谢途径更为多样，降低了联合用药的复杂性。在最核心的安全性指标——左心室射血分数下降的发生率与处理方式上，SEQUOIA-HCM试验中仅5.6%的Aficamten患者出现左心室射血分数低于50%，且均通过剂量调整解决；相比之下，Mavacamten的关键试验中该比例更高，且部分病例需中断治疗。管线扩张：同一平台机制下的风险平衡 Cytokinetics的管线扩张严格遵循其肌节动力学调节平台的逻辑，围绕心脏收缩力过强与不足两个方向进行适应症拓展，旨在最大化平台价值，但同时将风险分散于多个未经验证的疾病领域。公司正将Aficamten的适应症从梗阻性肥厚型心肌病扩展至非梗阻性肥厚型心肌病，并平行推进针对不同心衰类型的肌球蛋白激活剂与新型抑制剂。每一项新适应症的验证都伴随着独立的失败风险。在肥厚型心肌病领域，向非梗阻性肥厚型心肌病的扩展是Aficamten商业潜力的关键杠杆。目前，针对非梗阻性肥厚型心肌病尚无获批的药物疗法。Cytokinetics正在进行的III期ACACIA-HCM试验，旨在评估Aficamten在这一患者群体中的疗效。据估计，非梗阻性肥厚型心肌病患者数量与梗阻性肥厚型心肌病相当。若ACACIA-HCM试验在2026年第二季度获得积极顶线结果，Aficamten将成为该领域的首创疗法。然而，非梗阻性肥厚型心肌病的病理生理与治疗靶点与梗阻性肥厚型心肌病存在差异，其临床获益尚未被证实。在神经肌肉领域遭遇重大挫折后，公司仍保留了早期骨骼肌项目。在核心资产Reldesemtiv于肌萎缩侧索硬化症的III期COURAGE-ALS试验失败并终止后，公司并未完全放弃骨骼肌靶点。早期资产CK-089正处于I期临床阶段，旨在探索治疗一般性肌肉无力及相关虚弱症状的潜力。该项目的存在表明公司对骨骼肌激活机制仍抱有一定期待，但其前景远不明朗。风险聚焦：从临床失败到商业化的现实挑战 Reldesemtiv在肌萎缩侧索硬化症领域的III期失败，划定了Cytokinetics技术平台在复杂神经退行性疾病中的应用边界；同时，Aficamten的商业化成功面临来自市场竞争、财务压力与监管执行的多重挑战。技术平台并非万能，其在肌萎缩侧索硬化症适应症上的挫败揭示了机制转化中的困难。而在已获验证的梗阻性肥厚型心肌病市场，Aficamten需要在一个由制药巨头主导的竞争环境中争夺份额。 Reldesemtiv的失败揭示了快骨骼肌肌钙蛋白激活剂在肌萎缩侧索硬化症疾病背景下的局限性。尽管在II期FORTITUDE-ALS试验中观察到了在疾病进展较快患者亚组中的积极趋势，但随后的III期COURAGE-ALS试验在独立数据监测委员会的中期分析中因无效性而被提前终止。该药物未能证明其能延缓以ALSFRS-R评分衡量的功能衰退。该失败直接导致Cytokinetics终止了Reldesemtiv项目并将资源重新聚焦于心血管领域。 Aficamten的商业化面临与先行者Mavacamten的直接竞争。百时美施贵宝的Mavacamten拥有近三年的市场先发优势，积累了更长期的真实世界用药经验，且其背后是强大的商业化团队。尽管Aficamten在部分临床便利性指标上可能占优，但百时美施贵宝有能力通过积极的市场策略、更完善的长期安全性数据以及支付方关系来巩固其市场地位。Cytokinetics虽然设定了到2026年底在美国获得超过50%新患者首选份额的目标，但实现此目标需要持续且巨大的市场投入。公司严峻的财务状况构成了商业化道路上的根本性制约。 Cytokinetics仍处于深度亏损状态。2025年全年净亏损高达7.85亿美元。随着Aficamten在美国的商业化启动以及多项III期试验的同步推进，2026年的运营费用预计将进一步攀升。尽管公司拥有约18.3亿美元的现金及等价物，但高昂的现金消耗率意味着其可能在未来18-24个月内面临进一步的融资需求。全球合作架构在分散风险的同时也限制了利润空间。为缓解财务与运营压力，Cytokinetics将Aficamten在美国以外主要市场的权益授权给了合作方。此类模式降低了自建全球团队的成本与风险，但也意味着公司将大部分海外市场的销售利润与合作伙伴分享，长期收入潜力受到制约。后续关键临床试验的结果将直接影响公司估值与未来。 2026年第二季度预期的ACACIA-HCM顶线数据，以及未来的COMET-HF结果，是决定公司管线深度和长期增长故事的关键催化剂。若这些试验失败，将暴露公司对单一已上市资产Aficamten的过度依赖。以上是久谦的生物医药研究团队本次针对Cytokinetics的主要内容。之后我们还会继续推出一系列海外生科公司前沿生物医药研究文章。获取更多深度解析，敬请关注我们的公众号。我们将为您带来更多独家的海外创新药数据解读、管线与机制拆解、真实世界研究与交易案例分析与前沿趋势研判，助您保持领先认知，掌握全球生物医药信息差。我们自 2023 年起开始持续、系统性地追踪海外生物医药领域的创新药研发演进，重点覆盖创新药物作用机制（MOA）、平台型技术路径及其临床与商业化验证进展，涵盖上百家欧美及其他海外市场的主流 / 前沿生物科技公司。我们已逐步构建起一套以机制可重复性与商业可扩展性为核心的研究分析框架，完善了针对不同技术平台、适应症与研发阶段的多维度评估指标，并基于大量横向机制对比与临床、BD 及商业化场景的交叉验证，沉淀出对技术成熟度、应用边界与长期价值创造潜力的专业洞见。关于我们：我们是久谦旗下的生物医药研究团队，专注于创新药研发、生命科学工具、医疗技术平台及生物医药产业链等领域的研究，提供包含但不限于商业尽职调查、管线与机制评估、竞争格局分析、行业与赛道研究等服务类型。我们致力于对早期技术路径与行业演进过程进行系统性研究与持续跟踪，助力投资人与企业在关键技术周期中形成认知优势与高质量决策支持。我们相信，唯有基于一线研究与数据与事实驱动的真实洞察，才能为客户提供真正可落地、可验证的研究结论。若您有意向与我们进一步交流或合作，请扫描下方二维码联系我们：您最看好哪家生物技术公司？您对肌球蛋白抑制剂在心血管疾病的应用有何见解？欢迎在评论区与我们交流！

临床3期引进/卖出临床结果临床2期

2026-03-31

·allsci.com

Hengrui, Braveheart’s cardiac myosin inhibitor shows stronng early efficacy against cardiomyopathy

Hengrui Pharma and Braveheart Bio reported Phase II results for HRS-1893 (BHB-1893), a selective small-molecule cardiac myosin inhibitor, in patients with obstructive hypertrophic cardiomyopathy, the companies said on March 30. The data, from a 42-patient open-label dose-ranging study (NCT06516068), were presented as a late-breaking clinical research session at the American College of Cardiology Annual Scientific Session. Across three dose groups over 12 weeks, HRS-1893 produced complete Valsalva left ventricular outflow tract gradient (LVOT-G) responses — defined as a reduction below 30 mmHg — in 50% to 86% of patients, according to the companies. Mean LVEF declined between 1.8% and 2.7% across groups, and no patient recorded an ejection fraction below 55% during the core study period. The companies reported that average Valsalva LVOT-G fell below 30 mmHg as early as day 5. In Group 2, the 40 mg twice-daily cohort selected for an open-label extension, peak oxygen uptake increased by 1.0 mL/kg/min, the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score improved by 10.5 points, and NT-proBNP declined by 88%. At week 39 of the open-label extension, in which all 42 patients enrolled, the complete LVOT-G response rate was 88%. Adverse events were mild to moderate in severity, and none led to treatment interruption or discontinuation, the companies said. No new safety signals were identified during the 12-week study period. The trial randomized patients 1:1:1 to oral HRS-1893 at 20 mg twice daily (titrated up to 60 mg), 40 mg twice daily (up to 80 mg), or 40 mg once daily (up to 120 mg), with individual dose adjustments permitted based on LVEF and Valsalva LVOT-G assessments. The primary endpoint was change in Valsalva LVOT-G from baseline to week 12. The companies reported that 89% of patients were managed at 40 mg or 60 mg twice daily with minimal or no titration. The study was open-label, and no placebo arm was included, which limits interpretation of the magnitude of observed changes. Efficacy data from the once-daily group and full between-group comparative analyses were not disclosed in detail. The data are based on 42 patients, and the open-label extension findings remain preliminary. HRS-1893 is described by the companies as a selective cardiac myosin inhibitor intended to reduce hypercontractility in hypertrophic cardiomyopathy. The oHCM field already includes Bristol Myers Squibb’s mavacamten, approved by the US FDA in 2022, and aficamten. While cross-trial comparisons should be interpreted cautiously because endpoint definitions and trial designs differ, mavacamten was associated with a 48.1% rate of Valsalva LVOT gradient <30 mmHg at week 30 in EXPLORER-CN, while aficamten achieved a 49.3% rate of Valsalva LVOT gradient <30 mmHg at week 24 in SEQUOIA-HCM. Despite numerically higher response rates, the open-label design and small sample size mean HRS-1893’s apparent efficacy advantage over mavacamten and aficamten remains unproven. HRS-1893 was developed by Hengrui, with rights licensed outside of Greater China licensed to Braveheart in a September 2025 deal. Hengrui is leading a strong clinical development program, with a dose finding Phase II in symptomatic oHCM already completed, an ongoing Phase II in nHCM, and an ongoing Phase III study in oHCM in China (NCT07021976). Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

临床结果临床2期上市批准临床3期

100 项与阿夫凯泰相关的药物交易

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适应症	国家/地区	公司	日期
梗阻性肥厚型心肌病	中国	Cytokinetics, Inc.	2025-12-16

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适应症	最高研发状态	国家/地区	公司	日期
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	日本	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


NCT05186818 (SEQUOIA-HCM, CTgov)	临床3期	梗阻性肥厚型心肌病	282	Aficamten (Aficamten up to 20 mg)	鑰淵窪製鑰淵醖餘淵鬱(選齋窪製構夢選齋製觸) = 餘艱夢衊簾網繭觸繭願獵願夢醖簾鬱觸憲網餘 (鏇繭廠壓夢範膚製壓鹹, 0.25) 更多	-	2026-03-03
				Placebo to match aficamten (Placebo to Match Aficamten)	鑰淵窪製鑰淵醖餘淵鬱(選齋窪製構夢選齋製觸) = 蓋鏇願鏇鬱鹽壓繭醖願獵願夢醖簾鬱觸憲網餘 (鏇繭廠壓夢範膚製壓鹹, 0.25) 更多
NCT04219826 (REDWOOD-HCM Cohort 4 \| REDWOOD-HCM, CTgov)	临床2期	肥大型心肌病	96	CK-3773274 (Cohort 1 (oHCM) - Aficamten)	觸簾製網壓衊鏇衊糧糧 = 鹽襯網鬱觸醖蓋齋鏇夢鹹製顧餘構製遞壓襯齋 (選鑰觸鹽獵觸積鹽製糧, 衊蓋糧構醖蓋鹽壓鑰製 ~ 廠餘鑰齋鬱願夢鹹壓艱) 更多	-	2026-02-24
				CK-3773274 (Cohort 2 (oHCM) - Aficamten)	觸簾製網壓衊鏇衊糧糧 = 艱構夢糧築遞觸衊獵蓋鹹製顧餘構製遞壓襯齋 (選鑰觸鹽獵觸積鹽製糧, 糧齋築廠鹹製襯鏇淵遞 ~ 餘構簾衊構網壓顧襯積) 更多
NCT06412666 \| NCT05186818 (CEDAR-HCM, Pubmed \| Circ Heart Fail)	临床3期	梗阻性肥厚型心肌病	65	Aficamten	鬱艱鏇築構鬱憲網願範(願鹹衊蓋範鏇範鏇遞築) = 構鑰餘衊鑰網簾積繭夢蓋夢窪製願築鏇範窪窪 (夢鑰構簾鬱襯鬱鏇襯積 )	积极	2025-12-05
NCT05767346 (MAPLE-HCM, Pubmed \| J Am Coll Cardiol)	临床3期	梗阻性肥厚型心肌病	175	Aficamten 5 to 20 mg	鹽範糧構衊獵遞膚淵遞(衊夢淵淵繭獵鏇衊艱鏇) = 顧範壓觸鏇蓋淵構網餘淵製鏇壓鑰觸窪願夢衊 (廠膚網襯網壓製選範餘 ) 更多	积极	2025-12-01
				Metoprolol 50 to 200 mg	-
NCT05767346 (MAPLE-HCM, Pubmed \| J Am Coll Cardiol)	临床3期	梗阻性肥厚型心肌病	175	Aficamten	構齋顧衊鑰艱範艱窪鹹(蓋鏇膚醖願製餘窪鹽蓋): Difference = 7.8 (95.0% CI, 3.3 ~ 12.3), P-Value = < 0.001 更多	积极	2025-11-01
				Metoprolol
NCT05767346 (MAPLE-HCM, Pubmed \| J Am Coll Cardiol)	临床3期	梗阻性肥厚型心肌病	175	Aficamten	膚簾簾廠夢窪築製齋鏇(壓選選鬱選鹽糧醖築壓) = 製繭艱糧窪遞齋觸艱網淵築淵夢壓願願齋餘憲 (窪夢顧簾獵淵膚蓋憲顧 ) 更多	积极	2025-11-01
				Metoprolol	膚簾簾廠夢窪築製齋鏇(壓選選鬱選鹽糧醖築壓) = 廠積壓選鬱衊觸獵憲築淵築淵夢壓願願齋餘憲 (窪夢顧簾獵淵膚蓋憲顧 ) 更多
NCT05767346 (MAPLE-HCM, Company_Website) 人工标引	临床3期	梗阻性肥厚型心肌病	-	Aficamten	蓋壓淵夢構鹹夢觸顧齋(構願選顧襯齋顧餘廠襯) = 鹽餘窪構範齋網繭蓋憲醖糧鏇鏇鏇繭餘願襯繭 (繭獵鏇淵窪獵膚糧窪製 ) 更多	优效	2025-09-29
				Metoprolol	-
NCT04848506 (FOREST-HCM, Company_Website) 人工标引	临床2/3期	非梗阻性肥厚型心肌病	34	Aficamten (post-REDWOOD-HCM Cohort 4)	繭醖夢淵糧網願衊選衊(壓憲鹽醖鬱鹹齋艱鏇衊) = 襯膚膚壓壓願積壓構製選積壓觸築鹽廠齋鑰簾 (積積觸鑰積襯壓鏇製遞 ) 更多	积极	2025-09-29
NCT05767346 (MAPLE-HCM, Pubmed \| N Engl J Med) 人工标引	临床3期	梗阻性肥厚型心肌病	175	Aficamten	窪襯蓋獵觸網艱齋構構(廠製廠遞顧範觸鹹顧蓋) = 艱鏇鬱製獵餘鬱艱衊窪網網醖鹹齋窪鬱糧窪夢 (遞艱衊觸鏇窪構網窪糧, 0.5 ~ 1.7)	优效	2025-09-11
				Metoprolol	窪襯蓋獵觸網艱齋構構(廠製廠遞顧範觸鹹顧蓋) = 觸窪夢遞鹹鏇積齋繭廠網網醖鹹齋窪鬱糧窪夢 (遞艱衊觸鏇窪構網窪糧, -1.7 ~ -0.8)
NCT04848506 (FOREST-HCM, Pubmed \| JACC Heart Fail)	临床2/3期	梗阻性肥厚型心肌病	213	Aficamten 5 mg	壓顧繭艱願築衊積獵簾(構願齋壓選獵願築廠膚) = 2 (4.3%) asymptomatic and transient instances of left ventricular ejection fraction <50% (range: 47%-49%), neither resulting in drug discontinuation 餘鹹窪鏇構蓋憲襯醖膚 (糧觸繭鏇鏇鬱網範淵繭 ) 更多	积极	2025-08-01
				Aficamten ≤20 mg