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

/ 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 项与艾非凯泰相关的临床结果

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100 项与艾非凯泰相关的转化医学

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100 项与艾非凯泰相关的专利（医药）

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项与艾非凯泰相关的文献（医药）

2025-12-01·Journal of cardiovascular imaging

Cardiac myosin inhibitors in hypertrophic cardiomyopathy

Review

作者: Lim, Jaehyun ; Kim, Hyung-Kwan

Abstract:

Mavacamten, the first selective and reversible cardiac myosin inhibitor (CMI), has been introduced to the clinical arena for the treatment of obstructive hypertrophic cardiomyopathy (HCM). By reducing excessive actin-myosin cross-bridging, this agent decreases myocardial contractility and alleviates the dynamic left ventricular outflow tract (LVOT) obstruction in obstructive HCM. In the EXPLORER-HCM trial, mavacamten significantly improved exercise capacity, symptoms, and LVOT pressure gradients, while the VALOR-HCM trial proved it can obviate the need for septal reduction therapy in patients who were deemed to be candidates for septal reduction therapy. Notably, long-term data (MAVA-LTE study) has demonstrated sustained benefits up to 180 weeks, with < 10% experiencing transient reductions in left ventricular ejection fraction < 50% and only 1.3% of permanent discontinuation rate. Aficamten, a next-generation CMI with a shorter half-life, has also demonstrated comparable efficacy. Reverse remodeling following treatment was noted in both agents. In nonobstructive HCM, preliminary studies (MAVERICK-HCM trial and cohort 4 of REDWOOD-HCM trial) have reported improvements in cardiac serum biomarkers and symptoms. However, the preliminary results from phase 3 trials (ODYSSEY-HCM trial) revealed that primary endpoints were not met in nonobstructive HCM. Regarding safety, both were generally well tolerated. Although an LVEF reduction occurred in some patients, it was reversible with a dose reduction or a short-term drug cessation. These results emphasize careful dosing strategy with regular echocardiographic monitoring. Real-world data have also demonstrated consistent efficacy and safety across varying ethnic groups without new safety signals. CMI is a major advance in HCM management. However, future studies must provide data on hard clinical outcomes, such as heart failure hospitalization or death. Ongoing trials comparing CMI to traditional first-line therapies, such as β-blockers, will clarify their potential role as an initial therapeutic option.

2025-10-07·CIRCULATION

Characterization and Application of Novel Exercise Recovery Patterns That Reflect Cardiac Performance: A Substudy of the SEQUOIA-HCM Trial

Article

作者: Kulac, Ian J. ; Owens, Anjali T. ; Malik, Fady I. ; Wohltman, Amy ; Claggett, Brian L. ; Campain, Joseph ; Griskowitz, Catharine ; Kupfer, Stuart ; Prasad, Cheshta ; Moreno, Fabely ; Coats, Caroline J. ; Heitner, Stephen B. ; Newlands, Chloe ; Lee, Matthew M.Y. ; Malhotra, Rajeev ; Landsteiner, Isabela ; Minasian, Alexandra ; Giverts, Ilya ; Maron, Martin S. ; McGinnis, Shaina ; Jacoby, Daniel L. ; Lewis, Gregory D. ; Iskenderian, Nick ; Rupert, Lillian

BACKGROUND::

Post-exercise oxygen uptake recovery (VO 2 Rec) is slow in advanced heart failure. We sought to establish easily derived VO 2 Rec measures and evaluate their cardiospecificity and prognostic relevance in patients with dyspnea on exertion. We further sought to determine VO 2 Rec modifiability proportional to changes in cardiac function with disease-specific treatment of obstructive hypertrophic cardiomyopathy.

METHODS::

VO 2 Rec patterns were evaluated in relation to cardiac performance and the primary outcome of heart failure hospitalization or death in a referral cohort with dyspnea on exertion undergoing cardiopulmonary exercise testing with hemodynamic monitoring (MGH-ExS [Massachusetts General Hospital Exercise Study]). We then investigated longitudinal measures of VO 2 Rec in the pivotal phase 3 randomized controlled trial SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in Hypertrophic Cardiomyopathy) of aficamten versus placebo for 24 weeks in participants with symptomatic obstructive hypertrophic cardiomyopathy. For both cohorts, VO 2 Rec was uniformly measured as time for VO 2 to decline by >0%, 12.5% (VO 2 T 12.5% ), 25%, and 50% of peak VO 2 .

RESULTS::

Among 814 MGH-ExS patients (58±16 years of age, 58% women), those with a longer VO 2 T 12.5% (≥35 versus <35 seconds) demonstrated elevated exercise pulmonary capillary wedge pressure to cardiac output slope ( P <0.0001) with no difference in peripheral oxygen extraction ( P =0.11). For each 15-second increase in VO 2 T 12.5% , the hazard ratio for heart failure hospitalization and all-cause death was 1.54 (95% CI, 1.35–1.76; P <0.001). In SEQUOIA-HCM participants with cardiopulmonary exercise testing at baseline and week 24 (n=263, 59.1±2.9 years of age, 41% women), baseline VO 2 T 12.5% was 45±20 seconds and improved 8 seconds (95% CI, −12 to −5 seconds; P <0.001) with aficamten treatment compared with placebo at 24 weeks. Participants treated with aficamten versus placebo were more likely to improve VO 2 T 12.5% by ≥15 seconds (odds ratio [OR], 3.7 [95% CI, 1.9–6.9]; number needed to treat=4.8). Shortening of VO 2 T 12.5% correlated with reduced NT-proBNP (N-terminal pro-B-type natriuretic peptide), high-sensitivity cardiac troponin I, and left ventricular outflow tract gradient (all P <0.005).

CONCLUSIONS::

This study established VO 2 T 12.5% as a new measure that reflects cardiac performance during exercise and predicted heart failure event-free survival. Furthermore, VO 2 T 12.5% improved proportional to improvements in left ventricular outflow tract gradient and cardiac biomarkers in response to aficamten treatment, a cardiospecific therapy for obstructive hypertrophic cardiomyopathy. The simplicity and physiological relevance of VO 2 T 12.5% support its regular inclusion in cardiopulmonary exercise testing protocols evaluating cardiac function during exercise.

REGISTRATION::

URL: https://www.clinicaltrials.gov ; Unique identifier: NCT05186818.

2025-10-01·PROTEIN SCIENCE

Understanding hypertrophic cardiomyopathy and its regulation by myosin drugs

Article

作者: Warshel, Arieh ; Halder, Ritaban

Abstract:

Hypertrophic cardiomyopathy (HCM) is an inherited form of heart disease, caused by specific mutations, many of which are encoded by the β‐cardiac myosin (MYH7) protein. This work provides molecular insight into the effect of an HCM‐causing mutation, R190T of β‐cardiac myosin. The Arginine190 (R190) resides near the active site of the cardiac myosin and its alteration by a threonine (T190) residue leads to cardiac abnormalities related to the fatal HCM. Since the mutations lead to change in the function of the myosin, we focused on our previous finding that the motion and its directionality are determined by the rate‐determining barrier, which in the current case is the phosphate release step. Our study of the change of the phosphate release barrier used several approaches, including all‐atom umbrella sampling simulations, renormalization simulations, binding energy and stability analysis as well as structural and multiple sequence analysis. Our free energy calculations of the barrier for the rate‐determining phosphate release step reproduced the observed effect. Furthermore, we show that three key myosin drugs, aficamten, mavacamten, and omecamtiv, modulate the phosphate release barrier of the faulty myosin, and by this means it could repair the defects of the HCM mutant associated with fast phosphate release. In exploring the reasons for the effects of the mutations and the drugs, we conclude that the R190T mutation leads to the destabilization of the prepowerstroke (PPS) state of cardiac myosin. Such destabilization triggers rapid phosphate release from cardiac myosin. Since phosphate release is the rate‐determining step of β‐cardiac myosin, such alteration of the phosphate release barrier of the R190T mutant is a crucial functional factor. Our study demonstrates the importance of using multiscale approaches for the revelation of key mechanisms of HCM disease. Furthermore, we provide further evidence of the crucial role of the rate‐determining barrier in establishing the overall function of the myosin cycle.

338

项与艾非凯泰相关的新闻（医药）

2025-10-14

·药时空

年终冲刺！18款新药待审批，2025年FDA最终成绩单将如何？

2025年，美国FDA的新药审批节奏明显放缓，全年批准数量或将创下近三年以来的新低。尽管批准数量有所放缓，但今年已获批的新药在肿瘤精准治疗、罕见病治疗以及药物作用机制多元化等方面，展现出显著的创新质量。本文将从批准趋势、重点领域突破、待批重磅药物等角度，系统梳理2025年FDA新药批准的总体图景。 01 审批数量“断崖式”下降，创近三年新低根据Endpoints News的统计，FDA今年迄今已批准42种新药和生物制剂，剩余约16项审批决定将在2026年前完成。不过，即便剩余申请全部获批，2025年全年总量也仅能达到58种左右，远低于2023年的80种和2024年的69种。将时间线继续向前推，2020年-2022年，FDA分别批准了61种、63种以及51种新药和生物制剂。由此看来，2025年或将成为近五年以来，FDA审批数量次低水平的一年。尽管审批数量下降，但数量并不完全等同于创新质量。真正的价值在于其是否解决了未被满足的临床需求。例如，2025年前三季度批准的32款新药中，包含多款First-in-class（首创机制）和孤儿药，显示出“少而精”的特点。值得注意的是，FDA发言人明确表示，药物审评与审批未受民主党主导的政府停摆影响，相关工作仍在持续推进。不过，目前还不清楚数千名FDA工作人员的流失，是否在今年减慢审批进程方面发挥了作用。如果申请继续以目前的速度被延迟，今年的批准数量不太可能达到历史高位。早在7月，FDA专员Marty Makary在接受CNBC采访时表示，该机构“可能在2025年创下批准数量的新纪录”。FDA发言人并未对这一预测是否仍然有效发表评论。 02 肿瘤与罕见病：创新药物的主战场据FDA新药图鉴统计显示，2025年1-9月FDA共批准上市32款新药。按季度看，Q1季度共批准上市7款新药，Q2季度共获批9款新药，Q3季度共获批16款新药。按分子类型看，前三季度共获批小分子药物22款，生物大分子药物10款。其中，10款生物大分子药物中，包括5款单克隆抗体药物、2款抗体药物偶联物（ADC）、2款小核酸药物，以及1款双特异性抗体药物。按获批适应症看，包括肿瘤12款、罕见病11款、感染性疾病2款、眼部疾病2款、皮肤病2款、肺部、免疫和神经性疾病各1款。涉及新药认定包括孤儿药（18/32）、First-in-class（17/32）、突破性疗法（10/32）、首轮批准、优先审批及快速通道。此外，4款药物带黑框警告上市。总体而言，2025年FDA现批准的新药呈现出肿瘤治疗继续向精准化、免疫化与联合化深入；罕见病药物开发蓬勃，许多既往无药可治的疾病迎来了首款疗法；药物作用机制日益多元，技术平台丰富化等研发趋势。这些进展共同体现了医药研发从传统机制向前沿技术平台的拓展，为治疗提供了更多可能性。 03 年底冲刺：18款重磅新药待批尽管2025年全年审批量遇冷，但在今年剩余的两个多月，仍有18款新药等待FDA的最终审批。这些待批药物涵盖了眼科、心血管、代谢疾病、抗感染等多个领域，其中不乏备受关注的重磅产品。而这些药物的审评结果，将直接影响2025年最终的新药批准总量和创新成色。以下为今年年底待批的18款新药产品： 10月20日：Glaukos的Epioxa，这是一种用于圆锥角膜患者的角膜疗法，无需去除角膜上皮； 10月23日：Sydnexis的SYD-101，一种用于儿童近视进展的低剂量阿托品眼药水； 10月26日：拜耳的elinzanetant，一种用于缓解中度至重度潮热（由更年期引起）的神经激肽1和神经激肽3受体拮抗剂； 11月18日：Arrowhead Pharmaceuticals的plozasiran，一种用于治疗高甘油三酯水平的RNAi药物； 11月28日：大冢制药的sibeprenlimab，用于IgA肾病； 11月30日：Kura Oncology的ziftomenib，用于治疗携带核磷蛋白1突变的成人复发或难治性急性髓系白血病； 11月30日：Ascendis Pharma的TransCon CNP，一种C型利钠肽的前药，用于治疗软骨发育不全患者； 12月5日：Axogen的Avance神经移植，一种用于桥接断裂周围神经的现成人类神经异体移植； 12月12日：BioCryst的Orladeyo口服颗粒，用于治疗遗传性血管性水肿儿童； 12月13日：Milestone Pharmaceuticals的etripamil鼻喷雾剂，用于治疗阵发性室上性心动过速患者； 12月15日：Innoviva的zoliflodacin，一种用于治疗12岁及以上成人和儿童单纯性淋病的口服抗生素； 12月16日：GSK的depemokimab，用于两种哮喘适应症； 12月16日：Aldeyra的reproxalap，用于缓解干眼症的体征和症状； 12月26日：Cytokinetics的aficamten，用于治疗梗阻性肥厚型心肌病； 12月26日：Omeros的narsoplimab，用于治疗造血干细胞移植相关血栓性微血管病； 12月30日：Vanda Pharmaceuticals的tradipitant，用于治疗胃轻瘫；第四季度：Biohaven的troriluzole，用于治疗脊髓小脑性共济失调；第四季度：三菱田边制药的帕金森病治疗药物。总的来说，2025年FDA新药审批的“降温”，更多是周期性波动而非创新衰退。在研发成本高企、监管趋严、靶点竞争激烈的背景下，药企正从“追求数量”转向“追求质量”。尽管全年批准总数可能下降，但First-in-class药物、罕见病突破和精准治疗的进展，依然为患者带来了新的希望。参考资料： [1] FDA on pace for lowest number of new drug approvals since 2022.Endpoints News.October 13, 2025 03:30 PM EDTUpdated 04:01 PM FDA+. [2] 2025年：FDA已批准33款新药. 药事纵横. 2025-10-10. [3] 新药图鉴 | 2025年1-9月FDA批准上市32款新药可视化盘点，一图胜千言！FDA新药图鉴. 2025-10-01.

孤儿药优先审批抗体药物偶联物快速通道突破性疗法

2025-10-08

·丁香园 Insight 数据库

2025 年 Q4，这 10 款重磅新药有望在国内获批上市

2025 年前三个季度，79 款新药首次获 NMPA 批准上市，包括全球首个获批的 GCG/GLP-1 双受体激动减重药物玛仕度肽、全球首个高选择性外周 κ 阿片受体激动剂镇痛药安瑞克芬、国产首个 EZH2 抑制剂泽美妥司他片... Q4，又有哪些重磅新药会在国内获批上市呢？本文将根据 Insight 数据库「中国上市策略& 时长预测」模块，选出其中 10 款重磅新药（排名不分先后）进行分享，仅供读者参阅。信达生物匹康奇拜单抗 2024 年 9 月 26 日，信达生物匹康奇拜单抗注射液（研发代号：IBI112）的新药上市申请获 CDE 受理，用于治疗中重度斑块状银屑病。此次报上市是基于一项在中国中重度斑块状银屑病受试者中开展的 III 期注册临床研究 CLEAR-1（NCT05645627）的积极结果。该研究于 2024 年 5 月顺利达成主要研究终点和全部关键次要研究终点，研究显示匹康奇拜单抗组达成皮损清除（PASI 90、PASI 75、PASI 100、sPGA 0/1 和 sPGA 0）和生活质量改善（DLQI 0/1）的受试者比例均显著优于安慰剂组。研究治疗期间，匹康奇拜单抗整体安全性良好，未发现新的安全性信号。匹康奇拜单抗是全球首个注册 III 期临床首要研究终点中第 16 周达到 PASI 90 的受试者比例突破 80% 的 IL-23p19 抗体药物，同时在同类生物药中具有最长的维持期给药间隔（每 12 周一次），有望为中国中重度斑块状银屑病患者带来皮损清除、生活质量改善和用药便利性提升等多方面的综合获益。除了 CLEAR-1 以外，匹康奇拜单抗当前还开展了多项临床研究，包括：在中重度银屑病患者中开展的随机撤药再治疗的 III 期临床；在中重度银屑病患者中开展生物制剂治疗转匹康奇拜单抗治疗的 II 期临床；在中重度活动性溃疡性结肠炎患者中开展的 II 期临床。恒瑞医药瑞拉芙普-α 2024 年 9 月 20 日，恒瑞 1 类新药瑞拉芙普-α 注射液（研发代号：SHR-1701）的上市申请获 CDE 受理，适应症为联合氟尿嘧啶类和铂类药物用于局部晚期不可切除、复发或转移性胃及胃食管结合部腺癌（G/GEJA）的一线治疗。 SHR-1701 是恒瑞医药自主研发的一种抗 PD-L1/TGF-βRII 双功能融合蛋白。在剂量递增、剂量扩展和临床扩展的 I 期研究中，SHR-1701 单药用于胃癌末线人群显示出良好的抗肿瘤活性。在此基础上，恒瑞开展了一项 SHR-1701 联合化疗用于晚期 G/GEJA 的 III 期临床研究。在 2024 年 ESMO 大会上，恒瑞首次以口头报告的形式公布了此项 III 期临床的结果。数据显示，SHR-1701 所取得的疗效数据显著，尤其是在降低疾病进展和死亡风险层面（HR 值）：在意向治疗人群（ITT）中的中位 OS 为 15.8 个月（vs 安慰剂组 11.2 个月），HR 为 0.66，降低 34% 的疾病进展或死亡风险；在 PD-L1 CPS≥5 人群中，中位 OS 为 16.8 个月（vs 安慰剂组 10.4 个月），HR 为 0.53，降低 47% 的疾病进展或死亡风险。截图来源：Insight 数据库诺和诺德帕西生长素 2024 年 9 月 5 日，诺和诺德帕西生长素在国内申报上市。根据公开信息和临床试验进展，Insight 数据库推断本次申报的适应症为用于治疗因生长激素分泌不足而导致生长缓慢的儿童患者。帕西生长素（Somapacitan）是一种每周一次长效人生长激素蛋白衍生物。与现有的每日一次生长激素相比，帕西生长素可以将每年的治疗次数从 365 次减少至 52 次，大大提升患者治疗的便利性和依从性。目前该药已在美国、欧盟、日本等海外国家和地区获批，用于治疗儿童和成人的生长激素缺乏症。在中国，诺和诺德开展了两项帕西生长素研究，其中治疗因生长激素分泌不足而导致生长缓慢的儿童患者的 III 期临床研究已经完成。截图来源：Insight 数据库值得一提的是，帕西生长素已经于今年 4 月率先落地海南博鳌乐城国际医疗旅游先行区，用于在生长激素缺乏症所致生长迟缓的 3 岁及以上儿童和青少年中替代内源性生长激素，并在 5 月 24 日开出中国首张处方。和美药业莫米司特-Hemay005 2024 年 4 月 24 日，和美药业 1 类新药莫米司特-Hemay005 上市申请获 CDE 受理，用于中至重度斑块状银屑病。在 2023 年美国风湿病学会年会（ACR）上，莫米司特治疗中至重度慢性斑块状银屑病的 III 期临床（NCT04839328）结果已公布。结果显示，莫米司特和安慰剂组 PASI75 比例分别为 53.6% vs 16.0%，sPGA 0/1 率分别为 31.3% vs 6.4%。 Insight 数据库显示，目前国内共有 2 款 PDE4 抑制剂获批上市，分别是阿普米司特（印度太阳制药/安进）和克立硼罗乳膏（辉瑞）。大冢制药的地法米司特、和美药业的莫米司特-Hemay005 均处于上市审评阶段，后者是首个国产 PDE4 抑制剂。截图来源：Insight 数据库此外，国内方面，恒瑞、中国生物制药、华东医药、济川药业、健康元等企业均在布局 PDE4 赛道，总共有 5 款 PDE4 抑制剂已进入 III 期临床。 GSK 玛贝兰妥单抗 2024 年 12 月 7 日，GSK 的 BCMA ADC 药物玛贝兰妥单抗在中国申报上市，玛贝兰妥单抗联合硼替佐米加地塞米松（BVd）治疗复发或难治性多发性骨髓瘤。该申请基于 III 期头对头 DREAMM-7 试验的中期结果。数据显示，该试验达到了其主要终点，显示与达雷妥尤单抗联合硼替佐米和地塞米松（DVd）治疗复发或难治性多发性骨髓瘤相比，BVd 方案在 PFS 方面具有统计学显著和临床意义的改善。在后续计划的中期分析中，DREAMM-7 试验达到了 OS 这一关键次要终点，表明与标准治疗 DVd 方案相比，BVd 方案显著降低了死亡风险。 CytokineticsAficamten 2024 年 10 月 18 日，箕星药业宣布 CDE 已正式受理艾非凯泰（Aficamten）用于治疗症状性梗阻性肥厚型心肌病（oHCM）的新药上市申请。 Aficamten 是一款在研的新一代选择性小分子心肌肌球蛋白抑制剂，其治疗 oHCM 获 FDA 和 NMPA 授予的突破性治疗药物认定。 2020 年 7 月，箕星与 Cytokinetics 就该药在大中华区的研发和商业化签订了独家许可协议。2024 年 12 月，箕星药业与赛诺菲共同宣布签订正式协议，协议约定赛诺菲将收购箕星在大中华区独家开发和商业化 Aficamten 的权益。该新药上市申请是基于关键性全球 III 期临床研究 SEQUOIA-HCM 所取得的积极结果。研究结果表明，与安慰剂相比，Aficamten 治疗能显著提高患者的运动能力，治疗效果在所有预设亚组中都是一致的，这些亚组反映了患者的基线特征和治疗策略，包括接受或未接受作为背景治疗的 β-受体阻滞剂的患者。在所有 10 个预先设定的次要终点中，也观察到具有统计学意义和临床意义的改善。辉瑞马塔西单抗 2024 年 8 月 13 日，马塔西单抗（Marstacimab、PF-6741086）在国内报上市，这是辉瑞研发的一种血友病新型疗法，是一种靶向组织因子途径抑制物（TFPI）K2 结构域的单克隆抗体，属于血友病再平衡疗法。目前马塔西单抗已在美国和欧盟获批用于治疗血友病 A 及血友病 B。马塔西单抗的获批是基于 III 期 BASIS 研究。该研究不伴抑制物队列的中期分析结果显示，在不伴抑制物的血友病 A 及血友病 B 受试者中，经过马塔西单抗 12 个月的积极治疗期后，与既往因子预防（RP）和按需治疗 (OD) 相比，其经治疗出血的年化出血率 (ABR) 分别降低了 35% 和 92%。 2025 年 2 月，马塔西单抗正式落地海南博鳌乐城先行区，通过预充式注射笔皮下注射固定剂量给药，只需每周一次，有望减少血友病患者因频繁输注带来的心理负担和用药负担。精准生物普基仑赛 2024 年 7 月 20 日，重庆精准生物自主研发的国家一类生物新药 pCAR-19B 细胞自体回输制剂（普基仑赛注射液）上市申请获 NMPA 正式受理，该产品是国内首款针对儿童白血病的 CAR-T 产品，用于治疗 3-21 岁患有 CD19 阳性复发/难治性急性淋巴细胞白血病（ALL）的患者。在 2024 年 ASH 大会上，该药用于 CD19 阳性 R/R B-ALL 的 II 期临床结果首次被披露。中位随访 211 天，最佳 ORR 为 90.63%，其中 78.13% 的患者达到 CR，12.5% 的患者达到 CRi，超过了当前商业化抗 CD19 CAR-T 疗法产品在 R/R B-ALL 中报道的疗效。此外，98.27% 的 ORR 患者获得了阴性微小残留病（MRD）阴性缓解。3 个月 ORR 为 76.56%。中位缓解持续时间（DOR）为 10.61 个月，中位总生存期（OS）为 23.92 个月。根据 Insight 数据库，目前国内已有 4 款靶向 CD19 的 CAR-T 产品获批上市，分别为恒润达生的雷尼基奥仑赛、复星凯瑞引进的的阿基仑赛、药明巨诺的瑞基奥仑赛、合源生物的纳基奥仑赛。普基仑赛（精准生物）、IM19（艺妙神州/先声）、布瑞基奥仑赛（吉利德/复星凯瑞）均处于上市审评阶段。截图来源：Insight 数据库 Alnylam/赛诺菲芬妥司兰钠 2024 年 5 月 7 日，siRNA 疗法芬妥司兰钠上市申请获 CDE 受理，拟用于作为常规预防治疗，用于有或无凝血因子 VIII 或 IX 抑制物的血友病 A 或 B 的成人患者和 ≥12 岁青少年患者，以预防或减少出血的发生频率。芬妥司兰钠来自于 Alnylam，2018 年 8 月，赛诺菲和 Alnylam 公司达成合作，获得 Fitusiran 的全球开发和商业化权利。该药采用 Alnylam Pharmaceutical 的 ESC-GalNAc 偶联技术，因此在皮下给药时具有更强的效力和持久性。 2025 年 3 月 28 日，该药在美国率先获批，商品名为 Qfitlia，成为美国首个获批的治疗 A 型或 B 型血友病（无论是否含有抑制剂）的疗法，每年只需注射六次即可提供持续保护，代表着血友病治疗的重要突破。在中国，该产品还被 CDE 纳入了《以患者为中心的罕见疾病药物研发试点工作计划（「关爱计划」）》试点项目，这是一项旨在罕见疾病药物研发领域落实以患者为中心的药物研发理念的试点工作。 Ascendis/维昇药业隆培促生长素 2024 年 3 月 8 日，维昇药业宣布 NMPA 已受理隆培促生长素的上市许可申请。这是首个在美国及欧洲获批上市的长效生长激素（每周注射一次），用于治疗儿童生长激素缺乏症（PGHD）。隆培促生长素由 Ascendis Pharma A/S 所有，并在全球范围内开发。2018 年 11 月，维昇药业从 Ascendis 引进了该产品在中国的开发和商业化权益。 2022 年 4 月，维昇药业在中国完成了隆培促生长素治疗 PGHD 的关键 III 期临床试验。结果显示，隆培促生长素 52 周年化生长速率（AHV）高于短效（每日注射）人生长激素，具有统计学显著差异，并可能使患有 PGHD 的儿童在有限的时间窗口内更有效达到治疗目标。第 52 周时，隆培促生长素 AHV 为 10.66 厘米/年，而短效（每日注射）人生长激素为 9.75 厘米/年（p=0.001）。如欲了解更多国内新药获批情况，可以扫描文末二维码，前往 Insight 数据库查询。封面来源：站酷海洛题图免责声明：本文仅作信息分享，不代表 Insight 立场和观点，也不作治疗方案推荐和介绍。如有需求，请咨询和联系正规医疗机构。编辑：ccai PR 稿对接：微信 insightxb 投稿：微信 insightxb；邮箱 insight@dxy.cn

临床3期上市批准申请上市

2025-09-29

·GlobeNewswire-Health Care

Cytokinetics Presents New Data Related to Aficamten at the HFSA Annual Scientific Meeting 2025

Additional Data from MAPLE-HCM Show Aficamten Significantly Improves Measures of Maximaland Submaximal Exercise Capacity and Recovery Compared to Metoprolol 96-Week Analyses from FOREST-HCM SupportLong-Term Efficacy and Tolerability of Aficamten in Patients with Non-Obstructive HCM SOUTH SAN FRANCISCO, Calif., Sept. 29, 2025 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that additional data related to aficamten were presented in a Late Breaking Clinical Research session at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2025 in Minneapolis, MN. The first presentation was a pre-specified analysis of the effect of aficamten compared to metoprolol on exercise performance in obstructive HCM (oHCM) in MAPLE-HCM (Metoprolol vs Aficamten in Patients with LVOT Obstruction on Exercise Capacity in HCM), and the second was an analysis from FOREST-HCM (Follow-up, Open-Label, Research Evaluation of Sustained Treatment with Aficamten in HCM) on the efficacy and tolerability of long-term treatment of aficamten in patients with non-obstructive hypertrophic cardiomyopathy (nHCM), simultaneously published in the Journal of Cardiac Failure.1 “The additional data from MAPLE-HCM expands our understanding of aficamten across several measures of exercise performance, with effects observed not only in peak exercise capacity, but also in submaximal exercise and recovery from exercise,” said Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development. “Additionally, the long-term data from FOREST-HCM in patients with nHCM show sustained improvements in both patient symptom burden and cardiac biomarkers associated with aficamten. Most patients achieved the highest doses available without any early treatment discontinuations, giving us confidence in the dosing strategy and the potential of aficamten to benefit patients with nHCM, as is being assessed in the ongoing Phase 3 clinical trial, ACACIA-HCM.” Pre-Specified Analysis of Supplemental Endpoints from MAPLE-HCM Shows Aficamten Improves Measures of Submaximal and Maximal Exercise Performance and Post-Exercise Recovery Compared to Metoprolol Gregory Lewis, M.D., Jeffrey and Mary Ellen Jay Chair and Section Head, Heart Failure Medical Director, Cardiopulmonary Exercise Testing (CPET) Laboratory, Professor of Medicine, Harvard Medical School presented pre-specified supplemental analyses of the impact of treatment with aficamten relative to metoprolol on CPET measures of exercise performance from onset through post-exercise recovery in MAPLE-HCM. The primary result of MAPLE-HCM demonstrated the superiority of aficamten to metoprolol on peak oxygen uptake (pVO2) (change from baseline to Week 24, least squares mean (LSM) treatment difference (SE), +2.3 (0.39) mL/kg/min, p<0.001). In these new analyses, aficamten compared to metoprolol was also shown to nominally significantly improve measures of submaximal exercise performance, including anaerobic threshold, aerobic efficiency (VO2/work), ventilatory efficiency (pre-anaerobic threshold and VE/VCO2 slope), and other key measures of maximal exercise performance, including peak workload, peak heart rate, exercise duration and heart rate reserve (Table 1). Additionally, the speed of VO2 recovery, a sensitive measure of cardiometabolic resilience, increased with aficamten and slowed with metoprolol. A responder analysis showed that any improvement in pVO2 was observed more commonly with aficamten compared to metoprolol, while any worsening was more frequent in the metoprolol group. Table 1Submaximal Exercise Response Variables CPET variableLSM (95% CI)*P-value**Anaerobic threshold, mL+76 (41, 111)<0.001Aerobic efficiency(VO2/work), mL/min/watt+0.8 (0.2, 1.3)0.004Ventilatory efficiency(pre-anaerobic threshold)−1.3 (−2.3, −0.3)0.013Ventilatory efficiency(VE/VCO2 slope)−2.8 (−4.0, −1.5)<0.001Maximal Exercise Response Variables Peak workload, watt+8 (3, 13)0.003Peak heart rate, bpm+28 (24, 32)<0.001Exercise duration, min+0.6 (0.2, 1.0)0.002Heart rate reserve, bpm+12 (8, 16)<0.001* LSM (95% CI) = change from baseline to Week 24 least squares mean treatment difference between aficamten and metoprolol, 95% confidence interval** All p-values are nominal and not controlled for multiple comparisons New Data from FOREST-HCM Support the Efficacy and Tolerability of Long-Term Treatment with Aficamten in Patients with Non-Obstructive HCM Ahmad Masri, M.D., MS, Director of the Hypertrophic Cardiomyopathy Center at Oregon Health & Science University, presented new data related to the safety and long-term use of aficamten in patients with nHCM in FOREST-HCM. These analyses included 34 patients with nHCM who enrolled in FOREST-HCM, the open-label extension study, after completion of Cohort 4 of the Phase 2 clinical trial REDWOOD-HCM. Patients were followed for at least 96 weeks of treatment. At the end of titration (Week 6), 82.4% of patients were on the highest available doses of 15 mg and 20 mg. Through 96 weeks of treatment, there were no early treatment discontinuations and aficamten demonstrated sustained reductions in symptom burden. At Week 96, 27 of the 34 patients (79%) improved by at least one NYHA Functional Class, including 20 (74%) who became asymptomatic (NYHA Functional Class 1). Patients also experienced a mean increase in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) of 11.2 points (±14.3 points). Aficamten also improved cardiac biomarkers, with both NT-proBNP and high-sensitivity cardiac troponin I rapidly declining by Week 12 and remaining low through Week 96. There was a modest reduction in left ventricular ejection fraction (LVEF) from hyperdynamic at baseline to within normal range at Week 12 (-6.2%; p<0.0001). Over the treatment period, four patients had LVEF <50% (5.4 per 100 patient-years) which were reversible after down titration or a short treatment interruption. Only one instance of LVEF <50% was corroborated by the core lab. The efficacy and safety of aficamten in non-obstructive HCM is being investigated in an ongoing Phase 3 clinical trial. 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.1 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 with 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 oHCM; and FOREST-HCM, an open-label extension clinical study of aficamten in patients with HCM. Disclaimer This communication contains a summary of new data related to the clinical development of aficamten presented at the HFSA Annual Scientific Meeting 2025. 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.2,3,4 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.5 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.6 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, and advancing 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. In addition, Cytokinetics is developing omecamtiv mecarbil, a cardiac myosin activator, in patients with heart failure with severely reduced ejection fraction (HFrEF), ulacamten, 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 relating to any of our clinical trials, statements relating to the potential benefits of aficamten or any of our other drug candidates, or our ability to obtain regulatory approval for aficamten in any jurisdiction by any particular date, if ever. Cytokinetics’ research and development activities; the design, timing, results, significance and utility of preclinical and clinical results; and the properties and potential benefits of Cytokinetics’ other drug candidates. 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, potential difficulties or delays in the development, testing, regulatory approvals for trial commencement, progression or product sale or manufacturing, or production of Cytokinetics’ drug candidates that could slow or prevent clinical development or product approval; Cytokinetics’ drug candidates may have adverse side effects or inadequate therapeutic efficacy; the FDA or foreign regulatory agencies may delay or limit Cytokinetics’ ability to conduct clinical trials; Cytokinetics may be unable to obtain or maintain patent or trade secret protection for its intellectual property; standards of care may change, rendering Cytokinetics’ drug candidates obsolete; and competitive products or alternative therapies may be developed by others for the treatment of indications Cytokinetics’ drug candidates and potential drug candidates may target. For further information regarding these and other risks related to Cytokinetics’ business, investors should consult Cytokinetics’ filings with the Securities and Exchange Commission. CYTOKINETICS® and the CYTOKINETICS and C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries. Contact:CytokineticsDiane WeiserSenior Vice President, Corporate Affairs(415) 290-7757 References: Masri A, et al. Safety and Efficacy of Aficamten in Patients with Nonobstructive Hypertrophic Cardiomyopathy: A 96-Week Analysis from FOREST-HCM. J Card Fail. 2025Chuang C, Collibee S, Ashcraft L, et al. Discovery of Aficamten (CK-274), a Next-Generation Cardiac Myosin Inhibitor for the Treatment of Hypertrophic Cardiomyopathy. J Med Chem. 2021;64(19):14142–14152. https://doi.org/10.1021/acs.jmedchem.1c01290CVrg: Heart Failure 2020-2029, p 44; Maron et al. 2013 DOI: 10.1016/S0140-6736(12)60397-3; Maron et al 2018 10.1056/NEJMra1710575Symphony 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

临床结果临床3期申请上市突破性疗法临床2期

100 项与艾非凯泰相关的药物交易

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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, Company_Website) 人工标引	临床3期	梗阻性肥厚型心肌病	-	Aficamten	鏇蓋夢選積艱淵繭網選(築觸廠構憲醖襯憲艱簾) = 夢獵壓鹽獵齋顧餘顧構鹹遞窪獵淵製壓餘繭範 (壓襯願積襯淵選鹹齋構 ) 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)	鏇鏇衊壓簾築願鹹願鑰(構鬱膚築繭艱顧廠鏇選) = 窪製襯衊鏇顧鹹築窪遞網壓衊獵鑰鏇廠憲糧夢 (醖觸鏇憲觸遞鹽膚製餘 ) 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	膚鬱襯鹽築構淵鹹願獵(顧襯鑰積網構鏇醖鬱蓋) = 遞膚齋鏇餘選窪選鹹憲範壓衊膚壓築衊衊顧憲 (鹽憲製觸鏇蓋鬱繭襯繭, 0.5 ~ 1.7)	优效	2025-09-11
				Metoprolol	膚鬱襯鹽築構淵鹹願獵(顧襯鑰積網構鏇醖鬱蓋) = 窪製鬱糧願糧鹹繭願憲範壓衊膚壓築衊衊顧憲 (鹽憲製觸鏇蓋鬱繭襯繭, -1.7 ~ -0.8)
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	夢積構夢願壓憲鑰鏇糧(遞淵憲鹽構餘鹽觸艱網) = 範淵壓獵窪壓壓夢顧憲觸觸膚壓憲鹹餘鑰壓壓 (餘餘醖觸顧鑰膚齋蓋廠 ) 更多	积极	2025-08-01
				Metoprolol 50 to 200 mg	-
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	鑰構鹽繭衊齋餘獵網鹽(鑰網選網鹹獵鹽獵衊衊) = 2 (4.3%) asymptomatic and transient instances of left ventricular ejection fraction <50% (range: 47%-49%), neither resulting in drug discontinuation 觸構壓繭襯憲簾憲廠鹹 (積顧襯艱夢壓觸鹹製構 ) 更多	积极	2025-08-01
				Aficamten ≤20 mg
NCT05767346 (MAPLE-HCM, GlobeNewswire \| NEWS) 人工标引	临床3期	梗阻性肥厚型心肌病	-	Aficamten	簾窪選範構糧淵醖壓蓋(鹽網網繭獵鑰觸壓衊遞) = 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. 構窪網憲壓願膚餘糧夢 (簾選構壓範壓淵網獵獵 ) 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	鏇糧觸鑰壓艱膚膚觸衊(齋鏇夢遞淵鹽鑰願遞範) = 鬱襯網憲廠鹹網艱鹹獵繭餘壓範構製窪糧繭糧 (構網觸鹹鬱製醖餘壓鏇, 3.1) 更多	积极	2024-11-16
				Placebo	鏇糧觸鑰壓艱膚膚觸衊(齋鏇夢遞淵鹽鑰願遞範) = 網鏇壓範選淵鏇衊繭鏇繭餘壓範構製窪糧繭糧 (構網觸鹹鬱製醖餘壓鏇, 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	繭獵襯顧壓艱憲壓觸廠(醖艱簾鹹獵窪襯醖膚範) = 餘壓餘淵築鏇壓醖廠鹽醖憲選遞窪衊壓選艱膚 (夢觸網蓋廠鏇夢廠簾膚 ) 更多	积极	2024-11-01
				Placebo	繭獵襯顧壓艱憲壓觸廠(醖艱簾鹹獵窪襯醖膚範) = 齋鑰顧壓艱醖衊鹹願鹹醖憲選遞窪衊壓選艱膚 (夢觸網蓋廠鏇夢廠簾膚 ) 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	觸鏇築膚簾醖築鹹構築(製願網糧憲襯製淵襯憲) = 齋蓋憲醖廠築獵遞積鑰糧壓廠選襯選範夢積鹹 (齋網選襯獵選鹽鹹鬱襯, -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	醖觸遞齋鑰鏇網壓構鹽(廠淵餘鹽顧範憲積觸淵) = 淵襯遞糧鹽遞築淵蓋憲觸憲憲壓範鏇襯製鏇壓 (鹽觸網鹹鏇簾壓範襯構 ) 更多	积极	2024-11-01