This is an investigator-initiated two-center study. The goal of this study is to investigate the feasibility, safety and efficacy outcomes of a seamless transition from mavacamten to aficamten in patients with obstructive hypertrophic cardiomyopathy (oHCM).

开始日期2026-05-05

申办/合作机构

Oregon Health & Science University

[+1]

NCT07023341

/ Active, not recruiting临床3期

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

JPRN-jRCT2031250066

/ Active, not recruiting临床3期

A Phase 3, Multi-center, Randomized, Double-blind Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults with Symptomatic Non-obstructive Hypertrophic Cardiomyopathy. (A study of aficamten in adults with symptomatic non-obstructive hypertrophic cardiomyopathy (ACACIA-HCM))

开始日期2025-04-14

申办/合作机构

Cytokinetics, Inc.

100 项与阿夫凯泰相关的临床结果

登录后查看更多信息

100 项与阿夫凯泰相关的转化医学

登录后查看更多信息

100 项与阿夫凯泰相关的专利（医药）

登录后查看更多信息

116

项与阿夫凯泰相关的文献（医药）

2026-08-01·JAMA Cardiology

Exercise Performance With Aficamten vs Metoprolol in Obstructive Hypertrophic Cardiomyopathy

Article

Importance:

Patients with obstructive hypertrophic cardiomyopathy (oHCM) endure life-altering exercise limitations. Current treatment guidelines recommend β-blockers as first-line therapy primarily based on expert opinion. The Metoprolol vs Aficamten in Patients with Left Ventricular Outflow Tract Obstruction on Exercise Capacity in HCM (MAPLE-HCM) trial characterizes comprehensive exercise response to aficamten monotherapy vs β-blockade (metoprolol).

Objective:

To determine the effect of aficamten compared with metoprolol across all stages of exercise using 16 quantitative measures in individuals with oHCM.

Design, Setting, and Participants:

This was a prespecified secondary analysis of the MAPLE-HCM study, a phase 3, randomized, active-control trial conducted from June 2023 to March 2025 with data analyzed between May and July 2025 at 71 sites in North America, South America, Europe, Israel, and China. Patients with symptomatic oHCM with objective evidence of exercise intolerance (peak oxygen uptake [pVO 2 ] &lt;100% of predicted) were included. Those with a history of atrial fibrillation (paroxysmal or persistent), medical indication for β-blockers or calcium channel blockers prohibiting drug discontinuation, or intolerance or medical contraindication to β-blockers were excluded.

Intervention:

Randomized 1:1 to titrated aficamten (5-20 mg daily) or matching titrated metoprolol (50-200 mg daily) for 24 weeks.

Main Outcomes and Measures:

The main outcomes were submaximal exercise minute ventilation (VE)/carbon dioxide output (VCO 2 ) slope and anaerobic threshold; peak exercise duration, workload, heart rate and heart rate reserve; and postexercise oxygen recovery rates; and composite variables, such as circulatory power.

Results:

Of 175 randomized participants (mean [SD] age, 57.7 [13.2] years; 102 [58.3%] male), 165 (94%) had core laboratory–validated exercise tests at baseline and week 24. Compared with metoprolol, aficamten treatment improved multiple stages of exercise; including submaximal exercise VE/VCO 2 slope (−2.8; 95% CI, −4.0 to −1.5; P &lt; .001) and anaerobic threshold (76 mL/min; 95% CI, 41 to 111; P &lt; .001); peak workload (8 watts; 95% CI, 3 to 13; P = .003); the time required for VO 2 to recover by 12.5% postexercise (−11 seconds; 95% CI, −16 to −5; P &lt; .001); and circulatory power (819 mm Hg × mL/min per kg; 95% CI, 569 to 1070; P &lt; .001). Large improvements (≥3.0 mL/kg/min) in peak VO 2 were more common with aficamten compared with metoprolol (17 [20.5%] vs 3 [3.7%], respectively; odds ratio, 6.8; 95% CI, 2.0 to 22.5; P &lt; .001). In contrast, large reductions in peak VO 2 (≥3.0 mL/kg/min) were significantly more common with metoprolol than aficamten (17 [20.7%] vs 2 [2.4%], respectively; odds ratio, 10.6; 95% CI, 2.6 to ∞; P &lt; .001).

Conclusions and Relevance:

This prespecified analysis of MAPLE-HCM demonstrated monotherapy with aficamten was superior to metoprolol in promoting adaptation to multiple phases of exercise in patients with symptomatic oHCM and supports aficamten as first-line therapy for oHCM.

Trial Registration:

ClinicalTrials.gov Identifier: NCT05767346

2026-07-01·AMERICAN JOURNAL OF CARDIOLOGY

Efficacy of Aficamten in Hypertrophic Cardiomyopathy Patients With Very-High Left Ventricular Outflow Tract Gradients

Article

作者: Veselka, Josef ; Coats, Caroline J ; Nassif, Michael E ; Abraham, Theodore P ; Claggett, Brian L ; Wohltman, Amy ; Gimeno, Ramon ; Miao, Zi Michael ; Malik, Fady I ; Barriales-Villa, Roberto ; Olivotto, Iacopo ; Heitner, Stephen B ; Maron, Martin S ; Masri, Ahmad ; Kupfer, Stuart ; Januzzi, James L ; Jacoby, Daniel L ; Hegde, Sheila M

This sub-analysis of SEQUOIA-HCM (NCT05186818) evaluated the efficacy and safety of aficamten in obstructive hypertrophic cardiomyopathy (oHCM) and very-high Valsalva left ventricular outflow tract gradients (LVOT-G). Patients with oHCM and Valsalva LVOT-G ≥100 mm Hg (n = 73) were randomized to aficamten (n = 33) or placebo (n = 40) for 24 weeks. The primary endpoint was proportional change in Valsalva LVOT-G from baseline to Week 24. Secondary endpoints included the proportion of patients achieving Valsalva LVOT-G < 30 mm Hg, absolute change in Valsalva LVOT-G, Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CCS), New York Heart Association (NYHA) functional class, NT-proBNP, and exercise capacity (peak oxygen uptake [pVO₂], workload), and time eligible for septal reduction therapy (SRT). Over 24 weeks, aficamten significantly reduced Valsalva LVOT-G by 66% from 123 ± 28 mm Hg to 41 ± 30 mm Hg (p = 0.001), including to <30 mm Hg in 42% of aficamten patients (p <0.001). Aficamten was also associated with symptom relief: 16 patients (48%) had ≥1 improvement in NYHA class, including 9 with both class I and ≥5-point increase in KCCQ-CSS. Compared with placebo, aficamten substantially decreased NT-proBNP concentration (‒85%; p = 0.001) and increased pVO₂ (+1.8 ml/kg/min; p = 0.003). Aficamten also reduced time eligible for SRT (p = 0.002) without any clinically relevant decrease in systolic function (ejection fraction <50%). Aficamten effectively reduced gradients in patients with oHCM and very-high Valsalva LVOT-G. Hemodynamic changes were associated with enhanced exercise capacity and symptom relief, underscoring aficamten treatment benefit across the spectrum of LVOT-G.

2026-06-23·EUROPEAN HEART JOURNAL

Aficamten in symptomatic obstructive hypertrophic cardiomyopathy: the FOREST-HCM long-term study

Article

作者: Saberi, Sara ; Wei, Jenny ; Choudhury, Lubna ; Solomon, Scott D ; Barriales-Villa, Roberto ; Liu, Xueli ; Melloni, Chiara ; Simkins, Tyrell J ; Owens, Anjali Tiku ; Oreziak, Artur ; Malik, Fady I ; Abraham, Theodore P ; Cooper, Robert M ; Heitner, Stephen B ; Nassif, Michael E ; Kupfer, Stuart ; Olivotto, Iacopo ; Elliott, Perry M ; Tower-Rader, Albree ; Maron, Martin S ; Masri, Ahmad ; Jacoby, Daniel L

Abstract:

Background and Aims:

Aficamten is a next-in-class, oral selective cardiac myosin inhibitor that ameliorates hypercontractility in hypertrophic cardiomyopathy (HCM). This study assessed the safety and efficacy of extended aficamten treatment in symptomatic obstructive HCM (oHCM).

Methods:

Patients completing a parent aficamten study were eligible to enrol in FOREST-HCM (NCT04848506), an open-label study evaluating long-term aficamten treatment.

Results:

Patients with oHCM (N = 296; mean age ±SD 61 ± 12.3 years, 44.3% female) enrolled between May 2021 and August 2024. Cumulative exposure was 352 patient-years; median follow-up 51.6 (IQR 41.5, 70.8) weeks. At Weeks 12 and 96, aficamten reduced Valsalva left ventricular outflow tract gradient by 56 ± 43 and 62 ± 33 mmHg from baseline (both P < 0.0001), with minimal reduction in left ventricular ejection fraction (LVEF) (−3% ± 6% and −5% ± 5%); 69% and 93% of participants had at least one NYHA class improvement; Kansas City Cardiomyopathy Questionnaire–Clinical Summary Score improved by 15 ± 16 and 16 ± 16 points. Treatment-emergent serious adverse events (TESAEs) occurred in 36 (12.2%) patients; no deaths, heart failure, or events considered related to aficamten were reported. One (0.3%) patient terminated therapy due to a TESAE (ischemic colitis). LVEF<50% occurred in 10 (3.4%) patients [exposure-adjusted incidence rate (EAIR): 2.9 per 100 patient-years] with 2 having non-serious mild/moderate dyspnoea. No treatment interruptions for LVEF<50%, and no events of LVEF<40% occurred. New-onset atrial fibrillation occurred in seven (2.4%) patients (EAIR 2.0 per 100 patient-years).

Conclusions:

Extended aficamten treatment in patients with symptomatic oHCM yielded early and sustained hemodynamic and clinical responses with low incidences of new-onset atrial fibrillation and LVEF<50%.

726

项与阿夫凯泰相关的新闻（医药）

2026-09-01

·泓博医药人工智能药物设计平台

今日生物医药快讯-2026.09.01

本期快讯摘要临床方面，Jazz Pharmaceuticals 的 HER2 双特异性抗体 Ziihera 再次显著改善胃食管腺癌总生存期，Arrowhead 公布其 siRNA 药物 Redemplo 在严重高甘油三酯血症 III 期试验中安全性和疗效突出；公司动态方面，Takeda 与 Protagonist 推进 3 亿美元多肽药物 rusfertide 商业化合作，礼来以 28.75 亿美元收购 Merida 布局自身抗体降解疗法；FDA获批方面，PharmaEssentia 获 Besremi 治疗 ET 适应症扩展，Takeda 与 Protagonist 药物 Mimrylo 获批用于 PV 治疗；监管政策方面，Intelligencia AI 提出制药行业 AI 应用瓶颈在于信息组织而非模型能力。本文多维度解读全球生物医药产业新变化，助力读者高效洞察行业趋势。 01 专题板块临床试验 3 条 01Jazz Pharmaceuticals 披露 Ziihera 在 HER2 阳性胃食管腺癌中的第二项总生存期显著改善 Jazz Pharmaceuticals 披露其 HER2 双特异性抗体 Ziihera 在胃食管腺癌（GEA）中的第二项总生存期（OS）关键临床终点数据，进一步巩固其作为 HER2 靶向治疗新标准的潜力。在 Herizon-GEA-01 试验的第二次中期分析中，Ziihera 联合化疗相较于曲妥珠单抗（Herceptin）联合化疗显示出统计学显著且具有临床意义的 OS 改善。此前，Ziihera 联合 BeOne Medicines 的 PD-1 抑制剂 Tevimbra 及化疗在首次中期分析中也实现了 OS 显著提升，死亡风险降低 28%。Jazz 表示，无论是否联合 Tevimbra，Ziihera 的疗效均有所增强。公司研发负责人 Rob Iannone 表示，这些结果表明 Ziihera 有望成为一线 HER2 阳性晚期 GEA 的新标准治疗方案。 ZiiheraHER2胃食管腺癌双特异性抗体 02Arrowhead公布Redemplo（Plozasiran）治疗严重高甘油三酯血症的III期试验数据，安全性优势引发分析师分歧 Arrowhead Pharmaceuticals公布了其RNAi疗法plozasiran（商品名Redemplo）在严重高甘油三酯血症（severe hypertriglyceridemia）中的III期SHASTA-3和SHASTA-4试验详细数据。结果显示，plozasiran在治疗期间表现出类似安慰剂的安全性，治疗相关不良事件（TEAE）与安慰剂组相似，但严重不良事件发生率更低（8.3% vs 10%）。此外，未观察到过敏反应或肝酶显著升高，而目前获批药物Tryngolza（Ionis Pharmaceuticals）在这些方面存在约7%的不良事件率。在疗效方面，plozasiran在12个月内将甘油三酯水平中位数降低79%-81%，且在基线水平≥880 mg/dL的患者中效果更佳，中位数降幅达85%。同时，该药物将急性胰腺炎事件减少了78%。 RNAiRedemploplozasiran严重高甘油三酯血症 03Cytokinetics Myqorzo III期试验数据公布，疗效稳健但安全性引发争议 Cytokinetics 在 ESC 2026 年会上发布了其药物 Myqorzo 在非阻塞性肥厚型心肌病（HCM）患者中的 III 期临床试验详细数据。结果显示，Myqorzo 在 36 周内显著改善患者的心脏健康评分（平均提升 11.4 分），而安慰剂组仅提升 8.4 分。此外，Myqorzo 治疗组的峰值耗氧量有所提高，而安慰剂组则略有下降。尽管疗效数据被多数分析师认为“稳健”，但安全性问题引发市场担忧，试验中 Myqorzo 治疗组有 10 例心力衰竭事件，而安慰剂组仅 3 例。这一数据导致 Cytokinetics 股价下跌超 7%。不过，分析师指出，Myqorzo 治疗组无死亡病例，而安慰剂组有 3 例死亡，且当前非阻塞性 HCM 患者治疗选择极为有限，因此仍支持其获批并扩大使用。 CytokineticsMyqorzo肥厚型心肌病III期临床试验 02 专题板块公司动态 3 条 01Takeda获批首款靶向下丘脑肽的神经科学药物Orzeyful，开启多肽疗法新赛道 Takeda近日获得FDA批准，其首款靶向下丘脑肽orexin（又称hypocretin）的药物Orzeyful（oveporexton）正式上市，用于治疗罕见睡眠障碍发作性睡病1型（narcolepsy type 1）。该药物通过模拟orexin的作用，成为首个针对该疾病基础生物学机制的疗法。此前，Takeda在orexin激动剂研发中经历了多次失败，包括因肝脏毒性问题而终止的TAK-994，但最终成功开发出Orzeyful，并在短短五年内完成从首次人体试验到获批的进程。这一突破标志着orexin疗法在神经科学领域的潜力被广泛认可，被视为继GLP-1药物之后的又一革命性方向。Eli Lilly通过63亿美元收购睡眠专科公司Centessa，也表明其对orexin药物未来市场潜力的强烈信心。 Takedaorexin多肽疗法睡眠障碍 02礼来以28.75亿美元收购Merida Biosciences，布局自身免疫疾病靶向抗体降解疗法礼来近日宣布以28.75亿美元收购处于临床前阶段的生物技术公司Merida Biosciences，以获取其针对致病性自身抗体的降解疗法技术平台。Merida 的候选药物通过靶向特定自身抗体，而非广泛抑制免疫系统，从而有望更精准地治疗自身免疫和过敏性疾病。目前，Merida 正在推进其 Graves’ 病（GD）候选药物 MER511 的 I 期临床试验，初步数据显示其在降低促甲状腺激素受体（TSHR）刺激抗体方面具有显著效果。此外，Merida 还有针对 IgE 介导的过敏性疾病和原发性膜性肾病的候选药物进入临床前阶段。礼来表示，该收购符合其在免疫疾病领域“改变疾病进程”的战略方向，并进一步强化其在小分子和靶向疗法领域的布局。此次交易是礼来今年一系列重磅收购和授权交易中的最新一例，显示出其在创新疗法领域的持续扩张。礼来Merida Biosciences自身抗体降解Graves’病 03BMS 通过 Celgene 收购重塑神经科学管线，聚焦阿尔茨海默病与精神疾病治疗百时美施贵宝（BMS）近年来在神经科学领域加速布局，其核心战略依托于2019年以740亿美元收购Celgene所获得的资产。通过整合Celgene的神经科学管线，BMS重启了在神经免疫与神经炎症疾病上的研发，目前已拥有包括Zeposia（ozanimod）和Cobenfy在内的多个关键资产。Cobenfy于2024年获批用于治疗精神分裂症，现正推进用于阿尔茨海默病精神症状的扩展适应症。BMS还与BioArctic达成全球授权协议，利用其BrainTransporter技术开发可穿越血脑屏障的阿尔茨海默病抗体BAN2803。此外，公司正在探索多种脑递送平台，以支持小分子、寡核苷酸（如siRNA）等新型疗法在神经系统疾病中的应用。BMS强调“连续治疗”策略，旨在同时开发疾病修正疗法与症状缓解药物，以应对神经科学领域高失败率与复杂机制的挑战。这一系列动作表明，BMS正致力于打造世界级的神经科学研发团队，推动相关领域创新。 BMSCelgene阿尔茨海默病血脑屏障 03 专题板块FDA 获批 2 条 01Takeda 与 Protagonist 获 FDA 批准多肽疗法 Mimrylo 用于罕见血液癌症治疗 Takeda 与 Protagonist Therapeutics 联合开发的多肽药物 rusfertide（商品名 Mimrylo）近日获得 FDA 批准，用于治疗罕见血液癌症真性红细胞增多症（Polycythemia Vera, PV）。该药物是首个 hepcidin 模拟物，通过调节红细胞过度生成和铁分布来控制血细胞比容水平。其获批基于 Phase 3 VERIFY 临床试验数据，显示与标准治疗联合使用时，Mimrylo 显著提高患者反应率，减少放血治疗需求。此前，Takeda 已与 Protagonist 签署了 3 亿美元的预付款协议，共同开发和商业化该药物。Jefferies 预计 Mimrylo 峰值销售额可达 20 亿美元。此次批准标志着 Takeda 2026 年关键产品上市计划的重要进展，为其下一阶段增长奠定基础。 TakedaMimrylo多肽FDA 批准Polycythemia Vera 02PharmaEssentia 获 FDA 标签扩展，Besremi 新增治疗骨髓增殖性肿瘤 ET 适应症 PharmaEssentia 的长效干扰素药物 Besremi 获 FDA 批准新增适应症，用于治疗成人骨髓增殖性肿瘤（MPN）中的罕见病种——原发性血小板增多症（ET）。此次标签扩展基于两项临床试验数据，包括关键的 III 期 Surpass-ET 研究，结果显示 Besremi 在高危 ET 患者中的响应率显著优于已获批 29 年的 Agrylin（43% vs 6%），且在次要终点 JAK2 V617F 等位基因负担降低方面也表现良好，提示其可能更有效干预疾病病理。Besremi 作为同类首创药物，每 2-4 周注射一次，通过 IFNAR 激活 JAK-STAT 通路，发挥抗增殖、促凋亡和免疫调节作用。此次获批使 Besremi 在 MPN 领域覆盖 PV 和 ET 两个适应症，PharmaEssentia 表示正朝向成为全球首个覆盖全部四类 MPN 的制药公司迈进。 PharmaEssentiaBesremiETMPN 04 专题板块监管政策 1 条 01Intelligencia AI：制药行业AI应用的瓶颈并非模型能力，而是信息组织方式 Intelligencia AI 团队指出，尽管AI模型在制药研发中的能力不断提升，但行业在关键决策中仍面临挑战，其根本原因并非模型性能不足，而是制药知识的碎片化和缺乏结构化关系。当前的AI系统在处理需要跨多个独立来源整合证据的复杂问题时表现不佳，即使信息来源广泛，模型仍可能遗漏关键历史项目、支持证据或竞争背景。作者提出，应通过“Truth”和“Trust”两个维度评估AI性能：Truth衡量答案的准确性与完整性，而Trust衡量用户对答案的依赖程度，包括一致性与可追溯性。研究发现，直接访问权威数据库可提高Truth评分，但无法弥补信息源中未显式表达的关系和模糊数据。因此，构建决策级AI的关键在于将证据通过领域知识进行整合和结构化，而非单纯依赖模型迭代或信息扩展。该研究为制药企业在AI驱动研发中提供了新的思路和方法论。 AI drug discoveryTruth and Trust框架临床试验数据整合Intelligencia AI

临床3期siRNA临床2期

2026-08-31

·Radix药引pro

全球BD追踪｜8/31：19亿美元要约收官| Radix药引

本文约1782字　阅读约6分钟 8月最后一个交易日，全球生物医药 BD 市场以一笔近 19 亿美元的要约收购收官。过去 48 小时，交易、临床、融资、监管四条线并行：三星生物对瑞士肽类 CDMO 发起公开要约，海思科将临床前自免资产以超 15 亿美元潜在总额授权出海，ESC 2026 大会上心肌病、心衰、血脂赛道多项 III 期数据集中读数。【BD交易】三星生物要约收购PolyPeptide：瑞士肽类 CDMO 将易主（8月31日）三星生物通过瑞士子公司，对 PolyPeptide Group 全部公开持股发起现金要约，每股 CHF 44.31，隐含总权益价值约 CHF 14.6 亿（媒体口径约 19 亿美元）。要约价较传闻前最后交易日溢价 40%，要约期 9月15日至10月12日，接受门槛 66.67%。持股约 55.65% 的大股东 Draupnir 承诺全部参与，董事会一致推荐接受。配套融资同步启动约 22 亿美元配股增发，用于收购及产能扩张。PolyPeptide 为全球专业肽类 API CDMO，GLP-1 相关代谢管线暴露度高。海思科×Sentivera：临床前自免资产全球授权（8月25日）海思科授予 Sentivera 除中国大陆及港澳台外的全球开发、生产、商业化权利，资产为靶向 2 型炎症性疾病（哮喘、特应性皮炎、慢性鼻窦炎伴鼻息肉等）的临床前项目。首付款加股权对价合计约 7,590 万美元，后端里程碑加净销售分成最高 14.6 亿美元，潜在总价值超 15 亿美元。Sentivera 为 ARCH Venture Partners 与 Population Health Partners 新设公司，承接海思科核心免疫资产出海。 argenx 完成收购 Forte Biosciences（8月27日） argenx 经子公司完成全现金要约收购与合并，每股 77 美元，媒体口径总对价约 22 亿美元。核心资产 FB102 为靶向 CD122 的单抗，白癜风与乳糜泻已有临床概念验证，乳糜泻 II 期数据预计 2026 下半年读出。 SK Biopharmaceuticals×Biohaven：opakalim全球授权（8月27日） SK Bio 获得 Biohaven 的 Kv7 离子通道平台全球权益，核心资产 opakalim（BHV-7000）为选择性 Kv7.2/7.3 钾通道激活剂，处于局灶性癫痫II/III 期。首付款加里程碑合计最高 7.95 亿美元，另加分层销售分成。SK Bio 借此接棒 Xcopri 布局美国癫痫市场。 Genentech×Hanmi：HM17321 授权金额确认 23 亿美元（8月24日签署）此前披露的 UCN2 类似物（减脂保肌、非 GLP-1肥胖候选）授权，经多源确认总额最高 23 亿美元。【临床数据】 aficamten（MYQORZO）：非梗阻性 HCM 关键 III 期双主要终点均达（8月28日） ACACIA-HCM 研究纳入非梗阻性肥厚型心肌病（nHCM）患者 517 例，双主要终点均达：第 36 周 KCCQ-CSS 组间差 3.0（p=0.021），峰值摄氧量组间差 0.67 mL/kg/min（p=0.003）。关键次要终点中，NYHA 改善至少 1 级 41.9% vs 27.8%（p<0.001），NT-proBNP 降幅达 57%。此前没有 III 期研究在 nHCM 中同时显著改善患者报告与医生评估终点，数据同步发表于 NEJM，支持 MYQORZO 向 nHCM 适应症扩展。 finerenone（Kerendia）：FIND-CKD 高血压肾病预设亚组阳性（8月30日）非糖尿病 CKD 的 III 期研究 FIND-CKD 的高血压肾病亚组（459 例）显示：总 eGFR 斜率组间差 0.65（p=0.044），关键肾-心血管复合终点 12.0% vs 18.7%（HR 0.61，p=0.045）。高血压肾病目前缺乏指南推荐疗法，该数据支持 finerenone 向非糖尿病 CKD 全人群扩展。 124I-evuzamitide（AT-01）：心脏淀粉样变 PET 诊断 III 期达标（8月30日） REVEAL 研究（170 例）主要终点达成：诊断敏感度 94%、特异度 86%，ATTR 亚型敏感度 96%。该示踪剂源自 Bayer 年初收购的 Attralus 组合，已获 FDA突破性疗法认定，目前任何国家均未获批。 Alnylam ESC 2026 系列：vutrisiran 跨人群一致，zilebesiran 新亚组数据（8月30日） HELIOS-B 预设亚组显示，vutrisiran 对主要复合终点的获益与基线是否联用 tafamidis 无关；事后分析中患者"内在能力"下降风险降低 52%。与 Roche 共同开发的 zilebesiran 在 KARDIA-3 II 期背景利尿剂亚组中降压幅度更大，支持 ZENITH III 期推进。 eplontersen（Wainua）：CARDIO-TTRansform III 期主要终点未达（8月28日） Ionis 与阿斯利康联合开发的 eplontersen 在 ATTR-CM III 期研究中主要终点未达标，8月28日于 ESC 2026 公布。 TTR 沉默剂加稳定剂（tafamidis 背景）的联合策略受挫，成为 ESC 期间讨论焦点。 saroglitazar 镁盐：EVIDENCES-X IIb 期 MASH主要终点达成（8月31日） 189 例肝活检驱动 IIb 期研究（saroglitazar 2mg/4mg vs 安慰剂，52 周）主要终点达成，与安慰剂治疗差异 26.5%，关键次要及探索性终点亦见改善。完整数据将在此后肝病大会公布。saroglitazar 为 PPAR 双激动剂，印度已上市 Lipaglyn。 CTX310：体内 CRISPR 基因编辑ANGPTL3 一年随访（8月28日） 1a 期剂量爬坡 1 年数据：最高剂量组（0.8 mg/kg）ANGPTL3 平均降幅 78.6%、LDL-C 52.5%、甘油三酯 47.8%，无剂量限制性毒性。固定剂量 1b 期进行中。【融资与IPO】百利天恒：第四次冲刺港交所，重启 H 股上市（8月30—31日）百利天恒再次向港交所递交上市申请，为第四次冲刺（2024年以来三次递表后均未完成发行）。截至 8月28日市值 1,118 亿元，为科创板市值第二的创新药公司。 2023年12月与 BMS 的 BL-B01D1 授权协议潜在总额 84 亿美元，已收到 8 亿美元不可退还首付款；2025 年营收 25.18 亿元（较上年下降 56.7%）、净亏损 10.54 亿元。 2026年6月，宜泽康（双抗 ADC）获 NMPA 批准，7月起全国特药房上架。先博生物：完成近 4 亿元B 轮融资（8月31日）孚腾资本、千骥资本、浦东创投共同领投，张科禾润、元禾控股跟投。资金用于通用型 CAR-NK 管线 SNC103（CD19 靶点）确证性临床研究及体内 CAR-T 平台 SNC116注册临床开发。公司为先声药业孵化的细胞治疗平台。征祥医药：更新招股书再度冲刺港交所（8月26日）成立以来累计融资 9.4 亿元，最近一轮投后估值 25.52 亿元。核心产品玛硒洛沙韦片（流感 PA 核酸内切酶抑制剂）2025年7月获 NMPA 批准，正扩展青少年与暴露后预防适应症。【监管审批】 Ziihera±Tevimbra 联合化疗：FDA 批准一线 HER2+ 胃食管腺癌（8月25日）两个含 Ziihera（zanidatamab-hrii）方案获批用于一线治疗：与替雷利珠单抗（Tevimbra，百济神州）及氟尿嘧啶/铂类化疗三联方案覆盖 HER2 IHC 3+ 及 IHC 2+/ISH+ 全人群；双药加化疗方案用于 HER2 IHC 3+ 人群。依据 III 期 HERIZON-GEA-01，含 Tevimbra 三联方案中位总生存 26.4 vs 19.2 个月，死亡风险降低 28%。该方案为双特异性 HER2 靶向抗体、PD-1 抑制剂与化疗的组合。 Tivicay PD：FDA 批准用于 HIV 感染新生儿（8月26日） dolutegravir 儿科剂型获批用于体重至少 2 kg 的 HIV 感染婴幼儿（含出生后第一个月新生儿），填补婴幼儿 HIV 治疗空白。本期信号：ESC 2026 成为集中读数窗口，心肌病、心衰、血脂赛道多项 III 期数据同台发布，TTR 联合策略走向成为下一阶段关注点；中国药企在 BD 授权、融资、IPO 三线同步推进，license-out 金额结构（首付加里程碑加分成）持续演化。数据来源：Radix药引政策数据库（radixin.cn）全球BD追踪 Radix药引 — 医药政策智能知识库本文由 Radix药引编辑团队整理。

2026-08-31

·BioPharmaDive

ESC ‘26: Cytokinetics’ sell-off, Arrowhead’s edge and a heart drug autopsy

Today, BioPharma Dive is highlighting some notable clinical trial updates presented at the annual meeting of the European Society of Cardiology. Over the weekend, several large pharmaceutical and biotechnology companies released detailed study data on medicines for an array of different heart conditions.Below are three notable datasets and a look at how theyre being interpreted by investors and analysts.Cytokinetics stock sell-offCytokinetics claimed one of the bigger clinical trial victories in biotech this year when, in June, it disclosed that its drug Myqorzo succeeded in a Phase 3 trial in people with the non-obstructive form of the heart condition hypertrophic cardiomyopathy. That study made Myqorzo the first drug of its kind to prove beneficial against both forms of the condition, and separated it from Bristol Myers Squibbs market leading medicine Camzyos.On Friday, detailed data from that study were published in The New England Journal of Medicine and presented at ESC. Multiple Wall Street analysts claimed those findings reinforced Myqorzos potential despite the disclosure of a possible safety concern that sank Cytokinetics stock price.According to the NEJM paper, patients randomized to receive Myqorzo had an average 11.4-point improvement on a questionnaire assessing heart health after 36 weeks, compared to an 8.4-point change for placebo recipients. Peak oxygen consumption also improved for those treated with Myqorzo and slightly declined for those in the placebo group.Overall, Myqorzos effects were consistent across all study groups analyzed, reiterating Cytokinetics initial results, wrote Leerink Partners analyst Roanna Ruiz. And, importantly, the drugs impact on assessment scores appeared to grow over time, added Stifels James Condulis.The efficacy data are robust, Condulis wrote in his own note.Still, Cytokinetics shares fell more than 7% on Friday, as the paper also revealed 10 patients receiving Myqorzo experienced a specific kind of heart failure, versus three patients on placebo. That disclosure will likely add to an already lingering debate about the safety of Myqorzo and other drugs like it for people with non-obstructive disease, according to Condulis. (About one-third of patients have this form of HCM, which doesnt impede blood flow to the heart but can still put people at risk of poor health outcomes.)Yet Condulis and others argued the overall evidence should still support a new approval for Myqorzo and lead to broader uptake. Mizuho Securities analyst Salim Syed, for example, noted how there were no deaths among Myqorzo-treated patients, compared to three in the placebo arm. And patients with that form of the condition have essentially zero options currently.The stock sell-off is like missing [the] forest for trees, Syed added.Arrowheads edgeArrowhead Pharmaceuticals and Ionis Pharmaceuticals are already dueling for market supremacy in a rare disease that leads to extremely high triglycerides. That battle is soon to extend to a more common condition, with potentially billions of dollars at stake and where some analysts believe Arrowhead might have an advantage.Arrowhead in July said its drug plozasiran met the objectives of two late-stage studies in adults with severe hypertriglyceridemia, or sHTG. Those findings positioned Arrowhead to expand plozasirans use and directly challenge Ionis, which nabbed an approval in sHTG for its drug Tryngolza in June.At ESC, Arrowhead provided fuller details from those two studies and, to multiple analysts, described a drug with comparable efficacy to Tryngolza but potentially better safety. Prakhar Agrawal, of Cantor Fitzgerald, argued that plozasiran appears safer across several metrics, among them side effects leading to study discontinuations and liver enzyme or fat increases the latter of which has been a key focus for investors.The two drugs havent been tested against one another. But among patients who underwent a specialized imaging test in Arrowheads trials, plozasiran recipients had a roughly 1.5% placebo-adjusted increase in liver fat, compared to a 2% or 4% increase for Tryngolza recipients in Ionis studies, Agrawal noted.While specialists Agrawal spoke with suggested the liver fat increases Ionis reported may not be clinically meaningful, his team nonetheless expects it to be a factor in Arrowheads drug becoming the preferred option.Plozasirans results arguably support a best-in-class profile, wrote Condulis, from Stifel, in a separate note.Arrowhead intends to file for approval by the end of the year, setting up a potential launch in 2027. Agrawal expects plozasiran to claim 60% of a market opportunity that could reach $6 billion by 2035.A heart drug autopsyIn July, AstraZeneca and Ionis stunned industry watchers when they revealed eplontersen, a medicine theyve been developing, missed the central objective of a Phase 3 study in transthyretin amyloidosis-mediated cardiomyopathy, a progressive and deadly heart condition. At ESC, study investigators presented a detailed autopsy of those results, and Wall Street analysts began assessing the fallout for others with rival medicines.Alnylam already markets one drug called Amvuttra that, like eplontersen, interferes with the production of a protein implicated in the condition. It also has another, next-generation medication known as nucresiran thats in late-stage testing. Eplontersens failure raised doubts about Amvuttras impact on the disease, and heightened skepticism about nucresirans chances of success.William Blair analyst Myles Minter noted how AstraZeneca and Ionis findings call into question the benefits of combination therapy in TTR cardiomyopathy, where these silencer drugs might be used alongside the protein stabilizers patients typically receive. The results also fuel debate around the positioning of available therapies, which include Amvuttra and two branded stabilizers, Pfizers Vyndamax and BridgeBio Pharmas Attruby, according to Minter.In a separate note, Mizuhos Syed wrote that AstraZeneca and Ionis data increases [the] gap between stabilizers and silencers and effectively boosted the case for Attruby. In the placebo group in the failed eplontersen trial, a majority of the patients were on Vyndamax and the level of protein stabilization observed was well short of what was seen in testing of Attruby. Syed argued that the results show why a practitioner would choose Attruby over Vyndamax which will lose market exclusivity in 2031 and suggests investors may be modeling Attruby too conservatively once generics are available, he wrote.Analysts at Oppenheimer, meanwhile, rallied to Alnylams defense, noting that new analyses the company presented Sunday suggest Amvuttra may be more potent than eplontersen and that nucresiran might perform even better in Phase 3 testing. The reason, Oppenheimers Kostas Biliouris contended, is modality differences between Alnylam and Ionis drugs. Both medicines use different approaches RNA interference for Alnylams drugs and antisense oligonucleotide therapy for Ionis to achieve their intended effects. '

临床结果临床3期寡核苷酸

100 项与阿夫凯泰相关的药物交易

登录后查看更多信息

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

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

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
非梗阻性肥厚型心肌病	临床3期	美国	Cytokinetics, Inc.	2023-08-31
非梗阻性肥厚型心肌病	临床3期	中国	Cytokinetics, Inc.	2023-08-31
非梗阻性肥厚型心肌病	临床3期	日本	Cytokinetics, Inc.	2023-08-31
非梗阻性肥厚型心肌病	临床3期	阿根廷	Cytokinetics, Inc.	2023-08-31
非梗阻性肥厚型心肌病	临床3期	澳大利亚	Cytokinetics, Inc.	2023-08-31
非梗阻性肥厚型心肌病	临床3期	巴西	Cytokinetics, Inc.	2023-08-31
非梗阻性肥厚型心肌病	临床3期	加拿大	Cytokinetics, Inc.	2023-08-31
非梗阻性肥厚型心肌病	临床3期	丹麦	Cytokinetics, Inc.	2023-08-31
非梗阻性肥厚型心肌病	临床3期	法国	Cytokinetics, Inc.	2023-08-31
非梗阻性肥厚型心肌病	临床3期	德国	Cytokinetics, Inc.	2023-08-31

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05767346 (MAPLE-HCM, CTgov)	临床3期	梗阻性肥厚型心肌病	175	aficamten (Aficamten)	選憲齋餘鏇憲鹹襯願選(積鹽簾觸艱襯範觸鬱簾) = 窪衊鹹鏇遞蓋積願衊築積構鹽顧淵壓餘觸餘衊 (遞網築遞顧艱製衊鏇壓, 2.767) 更多	-	2026-06-09
				Metoprolol (Metoprolol)	選憲齋餘鏇憲鹹襯願選(積鹽簾觸艱襯範觸鬱簾) = 夢廠獵遞遞鬱遞憲壓構積構鹽顧淵壓餘觸餘衊 (遞網築遞顧艱製衊鏇壓, 2.186) 更多
NCT05186818 (SEQUOIA-HCM, CTgov)	临床3期	梗阻性肥厚型心肌病	282	Aficamten (Aficamten up to 20 mg)	衊衊繭構淵觸餘簾壓糧(憲積衊顧艱觸選簾顧襯) = 鑰遞顧遞獵蓋顧壓膚衊製觸醖夢簾夢構構構夢 (糧襯鑰壓鬱糧糧網願鏇, 0.25) 更多	-	2026-03-03
				Placebo to match aficamten (Placebo to Match Aficamten)	衊衊繭構淵觸餘簾壓糧(憲積衊顧艱觸選簾顧襯) = 廠選廠獵廠鏇範網願蓋製觸醖夢簾夢構構構夢 (糧襯鑰壓鬱糧糧網願鏇, 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)	鏇窪鏇醖餘鏇遞廠鏇願 = 廠齋鬱糧醖膚遞範顧廠鏇構顧觸廠鏇衊願築壓 (遞鏇醖製醖繭淵齋醖鏇, 願廠夢簾淵鬱願淵鹹鏇 ~ 襯選廠顧願襯獵選憲簾) 更多	-	2026-02-24
				CK-3773274 (Cohort 2 (oHCM) - Aficamten)	鏇窪鏇醖餘鏇遞廠鏇願 = 窪築鹽繭艱顧範顧獵廠鏇構顧觸廠鏇衊願築壓 (遞鏇醖製醖繭淵齋醖鏇, 選繭襯範齋簾襯獵鏇範 ~ 構鏇獵齋遞醖憲襯壓糧) 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	構築鑰鬱製壓蓋觸糧構(鑰壓壓積觸齋憲顧網遞) = 遞築顧顧糧鹽艱構選顧膚鑰廠範選憲範獵衊蓋 (積醖憲齋選顧築餘艱構 )	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	選鏇網窪選憲鑰遞壓顧(窪構願繭艱鹽夢壓獵鑰) = 觸鹹淵遞蓋繭築觸鏇艱積憲窪構壓鬱餘憲築夢 (網鑰網獵構廠膚獵膚壓 ) 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	窪選獵鏇鏇繭衊顧鑰憲(襯遞築夢淵構製鬱艱襯) = 積憲願範顧網構網淵鬱鑰窪襯獵觸選壓窪餘窪 (觸遞鬱繭鏇構壓糧簾範 ) 更多	积极	2025-11-01
				Metoprolol	窪選獵鏇鏇繭衊顧鑰憲(襯遞築夢淵構製鬱艱襯) = 鹹範膚觸繭壓鹹網構窪鑰窪襯獵觸選壓窪餘窪 (觸遞鬱繭鏇構壓糧簾範 ) 更多
NCT05767346 (MAPLE-HCM, Pubmed \| J Am Coll Cardiol)	临床3期	梗阻性肥厚型心肌病	175	Aficamten	膚壓夢餘選觸蓋遞鏇觸(艱壓獵網憲鹹糧壓夢簾): Difference = 7.8 (95.0% CI, 3.3 ~ 12.3), P-Value = < 0.001 更多	积极	2025-11-01
				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)	齋觸繭鬱齋選襯築蓋壓(衊範廠齋糧積齋積範遞) = 廠醖選製壓膚衊鏇網蓋選簾鹹顧鹹構衊繭鹽蓋 (繭積製夢顧築夢構憲範 ) 更多	积极	2025-09-29
NCT05767346 (MAPLE-HCM, Company_Website) 人工标引	临床3期	梗阻性肥厚型心肌病	-	Aficamten	夢壓衊壓繭繭餘鬱鑰壓(鬱築鏇構範淵窪鏇遞憲) = 艱襯網願壓構鏇餘遞範夢齋網窪壓築鏇構糧鬱 (艱網衊遞觸願憲鑰製顧 ) 更多	优效	2025-09-29
				Metoprolol	-
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	憲鏇夢淵壓憲衊簾獵膚(顧遞窪顧夢鬱製糧窪顧) = 鬱糧糧蓋獵築鹹範繭積範廠憲顧壓艱構衊鏇窪 (繭廠壓憲壓範鏇鑰顧鏇, 0.5 ~ 1.7)	优效	2025-09-11
				Metoprolol	憲鏇夢淵壓憲衊簾獵膚(顧遞窪顧夢鬱製糧窪顧) = 鏇膚積製製夢齋壓淵夢範廠憲顧壓艱構衊鏇窪 (繭廠壓憲壓範鏇鑰顧鏇, -1.7 ~ -0.8)