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

登录后查看更多信息

100 项与艾非凯泰相关的转化医学

登录后查看更多信息

100 项与艾非凯泰相关的专利（医药）

登录后查看更多信息

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.

2025-10-22·EUROPEAN HEART JOURNAL

Efficacy of aficamten in patients with obstructive hypertrophic cardiomyopathy and mild symptoms: results from the SEQUOIA-HCM trial

Article

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

Abstract:

Background and Aims:

Patients with obstructive hypertrophic cardiomyopathy (oHCM) treated with aficamten in SEQUOIA-HCM (NCT05186818) demonstrated marked improvement in symptoms and functional capacity. This analysis explores whether oHCM and mild symptoms patients experience similar clinical benefits with aficamten as patients with more advanced limitations.

Methods:

Patients in SEQUOIA-HCM (N = 282) were grouped at baseline according to symptom severity. Mild symptoms (n = 118) were defined as New York Heart Association (NYHA) class II and Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) ≥ 80, and moderate to severe symptoms (n = 150) as NYHA class II/III/IV and KCCQ-CSS <80. Primary endpoint was change in peak oxygen uptake (pVO2) from baseline to Week 24; secondary endpoints included change in NYHA class, KCCQ-CSS, outflow tract gradients, and N-terminal pro-B-type natriuretic peptide (NT-proBNP).

Results:

In aficamten-treated patients, change at Week 24 was not different between moderate to severe (1.8 mL/kg/min; n = 71) and mild (1.6 mL/kg/min; n = 62) symptom groups (P = .8). Likewise, the change in secondary endpoints (NYHA class, resting or Valsalva gradients, and NT-proBNP) did not differ significantly between the two symptom groups. Both groups experienced statistically significant improvements in KCCQ-CSS, but the extent of improvement was greater in the advanced symptom group (P = .02 for interaction). Treatment-emergent serious adverse events were infrequent in both groups.

Conclusions:

Patients with oHCM and mild symptoms treated with aficamten achieved significant improvement across a range of clinically relevant outcomes and generally similar to patients with more advanced symptoms. Less severely symptomatic patients could be considered for aficamten treatment.

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. ; Jacoby, Daniel L. ; McGinnis, Shaina ; 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.

351

项与艾非凯泰相关的新闻（医药）

2025-11-05

·新浪医药

赛诺菲阿夫凯泰、普乐司兰钠首秀；罗氏奥妥珠单抗、PDS等新品亮相；拜耳阿柏西普、Elinzanetant首展 | 进博看点

第八届中国国际进口博览会（以下简称“进博会”）今日启幕，新浪医药为读者盘点各大企业的首展、首秀以及重磅产品。赛诺菲：八赴进博，两款重磅新药全球首秀在本届进博会上，赛诺菲聚焦心血管、代谢、呼吸、肿瘤四大慢病领域，以及免疫炎症、移植、罕见病等全球患者亟需的免疫疾病领域，集中展示多款“同类首创”或“同类最佳”的创新药物和疫苗以及多款潜在重磅产品的全球研发管线。心血管领域两大重磅创新药，阿夫凯泰片和普乐司兰钠注射液迎来全球首秀。阿夫凯泰：针对导致猝死的“隐藏杀手”，突破现有治疗困局肥厚型心肌病（HCM）作为遗传性心脏疾病，可能引发猝死、心力衰竭、血栓栓塞等致命风险。阿夫凯泰片作为第二代心肌肌球蛋白抑制剂，获中国国家药品监督管理局突破性疗法认证，能够精准靶向心肌肌球蛋白，助力快速降低左心室流出道压差，是潜在“同类最佳”疗法。普乐司兰钠：填补相关治疗领域空白，降低高甘油三酯引发的系列风险普乐司兰钠注射液是家族性乳糜微粒血症综合征和严重高甘油三酯血症两大疾病的潜在治疗方案。目前中国尚无专门针对家族性乳糜微粒血症综合征的有效治疗药物。普乐司兰钠注射液是首个作用于创新靶点APOC3 mRNA的 siRNA类药物。全球3期临床数据显示，使用普乐司兰钠注射液患者10个月空腹甘油三酯水平较基线降低80%，12个月急性胰腺炎发生率较安慰剂降低83%，有望填补相关治疗领域空白。罗氏：八赴进博，十余款未来新药亮相今年进博会，罗氏首展首秀十余款即将在华上市及未来管线产品，覆盖乳腺癌、血液、神经科学、眼科、免疫、心血管及代谢等全疾病领域。其中，多个未来管线产品已进入大规模III期临床阶段奥妥珠单抗：首个抗CD20狼疮肾单抗罗氏重磅推出奥妥珠单抗在免疫治疗的新适应症。根据最新III期临床试验显示，奥妥珠单抗能显著提高患者的完全肾脏缓解率（CRR），是首个在狼疮性肾炎随机III期临床试验中显示出CRR益处的抗CD20单克隆抗体，目前已被美国FDA批准上市、获得欧盟优先审评审批，有望早日为中国广大狼疮性肾炎患者群体带来新的治疗选择。 PDS：全球首个、目前唯一的眼内植入方案罗氏眼科突破性解决方案PDS（Port Delivery System港式给药系统）首次亮相进博舞台。PDS是全球首个、目前唯一的眼内植入物, 仅有米粒大小，经手术置于睫状体平坦部，具有永久性，可再填充抗VEGF抗体雷珠单抗，在长达半年左右时间内持续释放药物，从而减少患者频繁接受眼底注射的需求，为眼底疾病患者的长期管理提供了全新解决方案。此外，下一代口服SERD药物Giredestrant、血友病创新药NXT007、阿尔茨海默在研新药Trontinemab、帕金森在研新药prasinezumab等多个未来管线产品也会集体亮相。吉利德科学携亚洲首发重磅展品亮相本次进博会，吉利德带来两款亚洲首发展品：长效HIV暴露前预防药物Lenacapavir（来那帕韦）和原发性胆汁性胆管炎（PBC）新药Seladelpar。 Lenacapavir：全球同类首创，预防HIV的有效性超过99.9% Lenacapavir（来那帕韦）是全球同类首创、一年仅需给药两次的长效HIV衣壳抑制剂。针对Lenacapavir用于HIV暴露前预防的全球III期临床研究证明，Lenacapavir（来那帕韦）预防HIV的有效性超过99.9%。凭借在HIV预防领域的重大突破，Lenacapavir（来那帕韦）荣获《科学》杂志“2024年度十大科学突破”榜首，入选美国《时代周刊》“2025最佳发明”榜单，并于近日摘得2025年盖伦奖“最佳医药产品奖”。2025年6月 Lenacapavir（来那帕韦）在海南博鳌乐城国际医疗旅游先行区获批用于HIV暴露前预防；10月22日，Lenacapavir（来那帕韦）在博鳌一龄生命养护中心完成中国首例应用。 Seladelpar：既能改善生化指标，又能缓解瘙痒症状 Seladelpar是针对原发性胆汁性胆管炎（PBC）患者的创新药物。PBC是一种慢性自身免疫性肝病，是易被忽视的肝脏“隐形杀手”，于2023年被纳入国家卫健委发布的《第二批罕见病》目录。除了疾病进展风险外，瘙痒是PBC患者最常见的症状之一，对生活质量产生显著影响。Seladelpar作为全球首个既能改善PBC患者生化指标、又可缓解瘙痒症状的创新疗法，于今年8月依托北京临床急需进口政策，在北京天竺罕见病药品保障先行区获批。拜耳：5个“全球首展”、8个“中国首展” 今年进博会，拜耳带来约26款亮点展品，包括5个“全球首展”、8个“中国首展”、13个“进博会首展”，还有4个“进博宝宝”。阿柏西普：获批后首展，延长患者治疗间隔阿柏西普8mg将迎来国内最新获批上市后的进博会首展。新生血管（湿性）年龄相关性黄斑变性（nAMD）是一种老年性的疾病，且随着人口老龄化的加剧，发病率逐年上升。阿柏西普在拥有更加强劲疗效的同时，可延长nAMD患者治疗间隔达4个月，显著降低患者的长期疾病负担。 Elinzanetant：首个治疗潮热的双重NK受体拮抗剂 Elinzanetant是首个获批用于治疗中度至重度潮热的双重NK受体拮抗剂，FDA的批准主要基于三项3期临床试验（OASIS-1、OASIS-2和OASIS-3）的数据，这些研究评估了Lynkuet用于治疗更年期中度至重度潮热的安全性和有效性。该疗法在三项研究中均达成所有主要终点，并展现出良好的安全性。此外，肺癌新型靶向治疗药物Sevabertinib、前列腺癌治疗药物达罗他胺的新适应症也将分别进行进博会首展和中国首展。雅培：带来十余款首秀产品本届进博会，雅培带来十余款首秀产品，涵盖全球领先的生物可穿戴传感技术、高效安全的微创诊疗、个性化的诊断解决方案、高质量的医学营养品和药品等。动态血糖监测系统雅培瞬感家族的多款新品，是中国市场目前唯一符合iCGM最高国际标准认证的动态血糖监测系统，包括：FreeStyle Libre Select 、FreeStyle Libre 2 Plus、FreeStyle Libre 2等。其中FreeStyle Libre Select传感器，每分钟自动传输血糖数值，适用于2岁及以上人群，连续监测长达15天，可兼容手机、胰岛素泵等多种数字化设备。电场消融导管一次性使用磁电定位压力监测球囊脉冲电场消融导管Volt™ PFA，采用独有的球囊篮式设计，配合脉冲电场，可高效锁定并精准消除异常心脏电信号，显著降低消融治疗风险。新浪医药综合加入读者交流群： —精彩回顾— 我国启动全球最大规模尿液HPV筛查研究针对红斑狼疮！阿斯利康首创生物制剂 III 期成功恒瑞乙肝「突破性疗法」来袭 ADC之后，ABC来了全球首例！中国医生体内CAR-T成功治疗狼疮商务合作：杨小雨 15210041717

临床3期优先审批siRNA上市批准信使RNA

2025-11-05

·香港济民药业

FDA预计10款新药将在2025年12月做出批准决定

处方药使用者费用法案(PDUFA)日期是指美国美国食品药品监督管理局(FDA)设定的审查新药申请(NDA)或生物制剂许可申请(BLA)并做出上市批准最终决定的最后期限。审查的典型周期是药品申请被机构接受后的10个月。对于具有优先审评权的药品，从申请受理之时起，审评期缩短至6个月。 Lisocabtagene Maraleucel治疗复发/难治性边缘区淋巴瘤 PDUFA日期：2025年12月5日美国FDA正在审查lisocabtagene maraleucel(liso-cel)的补充生物制品许可申请(sBLA)。liso-cel是一种靶向CD19的基因修饰自体T细胞免疫疗法，用于治疗既往接受过至少两线系统性治疗的复发或难治性(R/R)边缘区淋巴瘤(MZL)成人患者。该sBLA基于开放标签、单臂II期TRANSCEND FL试验(ClinicalTrials.gov注册号：NCT04245839)中MZL队列的结果。研究结果显示，在66例接受liso-cel治疗且疗效可评估的复发/难治性边缘区淋巴瘤(R/R MZL)患者中，总缓解率为95.5%（95% CI，87.3-99.1；P <.0001），其中62.1%的患者达到完全缓解（次要终点；95% CI，49.3-73.8；P <.0001）。结果还显示，24个月的缓解持续时间、无进展生存期和总生存期分别为88.6%（中位随访时间21.6个月）、85.7%（中位随访时间23.8个月）和90.4%（中位随访时间24.5个月）。安全性方面，liso-cel治疗后严重细胞因子释放综合征和神经系统事件的发生率较低。 Gepotidacin治疗单纯性泌尿生殖道淋病 PDUFA日期：2025年12月11日 Gepotidacin是一种正在接受审查的三氮杂苊类抗生素，用于治疗12岁及以上体重至少45kg的无并发症泌尿生殖道淋病患者。它通过阻断2种不同的II型拓扑异构酶来抑制细菌DNA复制。如果获得批准，gepotidacin将为淋病提供一种新的口服选择，淋病是一种性传播感染，已被美国疾病控制和预防中心视为紧急公共卫生威胁。该新药申请的数据来自EAGLE-1研究(ClinicalTrials.gov 注册号：NCT04010539)。该研究比较了口服gepotidacin与肌注头孢曲松钠联合口服阿奇霉素治疗由淋病奈瑟菌引起的单纯性泌尿生殖道感染的青少年和成人患者(N=628)的安全性和有效性。研究结果显示，该试验达到了主要终点，证明gepotidacin在治疗淋病方面不劣于标准疗法。接受gepotidacin治疗的患者中，微生物学治愈率为92.6%（95% CI，88.0-95.8），而接受头孢曲松联合阿奇霉素治疗的患者中，微生物学治愈率为91.2%（95% CI，86.4-94.7）。 Lerodalcibep用于降低低密度脂蛋白胆固醇患者 PDUFA日期：2025年12月12日 Lerodalcibep是一种基于附着蛋白的小蛋白结合型第三代前蛋白转化酶枯草杆菌蛋白酶/kexin 9型(PCSK9)抑制剂，其血浆半衰期延长(12至15天)。该药设计为每月一次皮下注射，无需冷藏，患者可自行给药。它旨在降低动脉粥样硬化性心血管疾病(ASCVD)或非常高或高风险ASCVD和原发性高脂血症(包括杂合子)患者以及10岁或以上纯合子家族性高胆固醇血症患者的低密度脂蛋白胆固醇(LDL-C)。该生物制品许可申请(BLA)的数据来自LIBerate项目，该项目纳入了2300多名已接受最大耐受剂量他汀类药物和其他口服药物治疗但仍需进一步降低LDL-C水平的患者。这5项试验的结果显示，使用Lerodalcibep治疗可显著且持续地降低低密度脂蛋白胆固醇(LDL-C)水平。 Zoliflodacin治疗单纯性淋病 PDUFA日期：2025年12月15日美国FDA正在审查Zoliflodacin（一种口服螺嘧啶三酮类抗生素）的新药申请(NDA)，用于治疗12岁及以上成人和儿童的单纯性淋病。体外研究表明，Zoliflodacin对包括对头孢曲松和阿奇霉素耐药的耐药菌株在内的多重耐药淋病奈瑟菌菌株具有活性，且与其他抗生素无交叉耐药性。该NDA申请包含全面的临床资料，其中包括一项关键的III期研究（ClinicalTrials.gov 注册号：NCT03959527），该研究比较了Zoliflodacin与标准治疗方案（肌注头孢曲松联合口服阿奇霉素）在930例单纯性淋病患者中的疗效。研究结果表明，与标准治疗相比，Zoliflodacin在泌尿生殖系统感染部位的微生物学治愈率方面不劣于标准治疗。生殖器外部位（直肠或咽部）的微生物学治愈率在各治疗组之间也相似。 Depemokimab治疗哮喘、慢性鼻窦炎伴鼻息肉 PDUFA日期：2025年12月16日葛兰素史克(GSK)正在寻求批准depemokimab用于治疗伴有2型炎症的哮喘和伴有鼻息肉的慢性鼻窦炎(CRSwNP)的附加维持治疗。Depemokimab是一种超长效生物制剂，对白细胞介素-5(IL-5)具有高亲和力。其延长的半衰期使其可以每6个月皮下注射一次，每年两次。 SWIFT-1(ClinicalTrials.gov注册号：NCT04719832)和SWIFT-2(ClinicalTrials.gov注册号：NCT04718103)试验结果显示，对于接受中高剂量吸入性糖皮质激素联合其他哮喘控制药物治疗的嗜酸性表型重度未控制哮喘患者，与安慰剂相比，depemokimab治疗可降低临床显著的哮喘急性发作率和住院率。在慢性鼻窦炎伴鼻息肉研究（ANCHOR-1[ClinicalTrials.gov注册号：NCT05274750]和 ANCHOR-2[ClinicalTrials.gov注册号：NCT05281523]）中，与安慰剂组相比，depemokimab治疗组患者的鼻息肉大小和鼻塞（共同主要终点）均有显著改善。 Aficamten治疗梗阻性肥厚型心肌病 PDUFA日期：2025年12月26日 Aficamten是一种口服小分子心肌肌球蛋白抑制剂，目前正在接受FDA审查，用于治疗梗阻性肥厚型心肌病患者。该疗法旨在减少每个心动周期中活性肌动蛋白-肌球蛋白横桥的数量，从而抑制与肥厚型心肌病相关的心肌过度收缩。此次新药申请(NDA)的提交基于III期SEQUOIA-HCM试验(ClinicalTrials.gov 注册号：NCT05186818)的数据，该试验纳入了282名患有症状性肥厚型心肌病和左心室流出道梗阻的成年患者。研究结果显示，与安慰剂组相比，Aficamten治疗组在第24周时，通过心肺运动试验测得的峰值摄氧量显著增加。接受aficamten治疗的患者也报告了心脏结构、功能和生物标志物的改善。在安全性方面，aficamten耐受性良好，不良事件发生率与安慰剂相当。未报告心力衰竭恶化或因左心室射血分数降低而导致治疗中断的情况。 Narsoplimab治疗造血干细胞移植相关血栓性微血管病 PDUFA日期：2025年12月26日 FDA正在审查narsoplimab重新提交的生物制品许可申请(BLA)，用于治疗造血干细胞移植(HSCT)相关血栓性微血管病(TA-TMA)。TA-TMA是干细胞移植的一种严重并发症。 Narsoplimab是一种人源单克隆抗体，特异性靶向甘露聚糖结合凝集素相关丝氨酸蛋白酶-2 (GBL-2)。其设计目的是预防TA-TMA中观察到的补体介导的炎症和内皮损伤。重新提交的BLA包含一项分析，比较了来自II期OMS721-TMA-001研究(ClinicalTrials.gov 注册号：NCT02222545)的28例TA-TMA患者的总生存期，以及来自外部对照HSCT注册库的100多例未接受Narsoplimab治疗的TA-TMA患者的总生存期。研究结果显示，与未接受治疗的外部对照组患者相比，接受Narsoplimab治疗的患者死亡风险显著降低（风险比，0.32[95% CI，0.23-0.44]；P <.00001）。该申请还纳入了来自TA-TMA的Narsoplimab扩大用药项目（ClinicalTrials.gov注册号：NCT04247906）的数据，该项目进一步证实了Narsoplimab的总体生存获益。 Tolebrutinib治疗非复发性继发进展型多发性硬化症 PDUFA日期：2025年12月28日 Tolebrutinib是一种口服的、可穿透血脑屏障的布鲁顿酪氨酸激酶抑制剂，目前正在接受FDA审查，用于治疗非复发性继发进展型多发性硬化症(nrSPMS)，并延缓成人患者的残疾进展，且该治疗与复发无关。该新药申请(NDA)的提交基于3期临床试验的数据，包括HERCULES（ClinicalTrials.gov 注册号：NCT04411641）、GEMINI 1（ClinicalTrials.gov 注册号：NCT04410978）和GEMINI 2（ClinicalTrials.gov 注册号：NCT04410991）。在HERCULES研究中，与安慰剂相比，Tolebrutinib治疗使非复发缓解型继发进展型多发性硬化症(nrSPMS)患者6个月确认的残疾进展发生时间延迟了31%(P = 0.003)。 GEMINI 1和GEMINI 2试验比较了Tolebrutinib与特立氟胺治疗复发型多发性硬化症(RRMS)患者的疗效。两项试验均未达到主要终点，即年复发率的统计学显著改善；然而，在汇总分析中，Tolebrutinib使6个月确认的残疾恶化发生时间延迟了29%(P = 0.023)。 Relacorilant治疗库欣综合征 PDUFA日期：2025年12月30日 Relacorilant是一种皮质醇调节剂，目前正在接受FDA的审查，有望成为治疗库欣综合征（高皮质醇血症）的潜在药物。此次新药申请(NDA)的提交基于关键性III期GRACE试验（ClinicalTrials.gov 注册号：NCT03697109）和确证性III期GRADIENT试验（ClinicalTrials.gov 注册号：NCT04308590）的数据。 GRACE试验的结果显示，Relacorilant治疗可显著改善高血压、高血糖以及库欣综合征的其他症状，例如体重、腰围和认知功能。GRADIENT试验的数据也支持了这些结果，并证实Relacorilant可改善多种高皮质醇血症的体征和症状。在安全性方面，这些试验中没有报告Relacorilant引起的低钾血症、子宫内膜肥大或相关阴道出血、肾上腺功能不全或QT间期延长的病例。 Tradipitant治疗晕动病相关呕吐 PDUFA日期：2025年12月30日 Tradipitant是一种神经激肽-1 受体拮抗剂，用于治疗晕动病相关呕吐的新药申请(NDA)正在接受FDA的审查。该药已在两项III期临床试验中进行研究：Motion Syros（ClinicalTrials.gov注册号：NCT04327661）和Motion Serifos（ClinicalTrials.gov注册号：NCT05903924）。这两项试验均在美国沿海水域的船只上进行，模拟真实世界环境。 Motion Syros试验纳入了365名有既往旅行晕动病史的患者。结果显示，与安慰剂组相比，Tradipitant治疗组患者的呕吐发生率显著降低。同样，在Motion Serifos研究中，316名在旅行中受晕动症困扰的参与者被随机分配接受Tradipitant或安慰剂治疗，以评估其对呕吐的影响。结果显示，与安慰剂组相比，Tradipitant显著预防了这些患者的呕吐。与安慰剂组相比，Tradipitant组的严重恶心和呕吐症状也明显减轻。注：以上资讯来源于网络，由香港济民药业整理编辑（如有错漏，请帮忙指正），只为提供全球最新上市药品的资讯，帮助中国患者了解国际新药动态，仅供医护人员内部讨论，不作任何用药依据，具体用药指引，请咨询主治医师。公众号：香港济民药业微信：pidrug_com 如需转载烦请注明出自香港济民药业感谢您的理解最后记得随手点「在看」

优先审批临床结果临床2期免疫疗法细胞疗法

2025-11-05

·Firstwordpharma

Heart disease startup Braveheart Bio makes debut, backed by $185M

Braveheart Bio is stepping into an increasingly competitive hypertrophic cardiomyopathy (HCM) space with $185 million in series A funding and a leadership team seasoned in cardiovascular drug development. Its backers include Andreessen Horowitz, Forbion, OrbiMed, Enavate Sciences and Frazier Life Sciences, with Biogen CEO Chris Viehbacher taking the chair of Braveheart's board."Braveheart is an exciting company with an excellent team and real potential to make an impact for patients," Viehbacher said.The San Francisco-based startup, which launched Wednesday, is working on an experimental cardiac myosin inhibitor, BHB-1893, that it hopes will combine efficacy with a safety profile that could support simpler dosing. The drug, also known as HRS-1893, was originally discovered by Jiangsu Hengrui Pharmaceuticals, which recently out-licensed ex-China rights to Braveheart in a deal worth up to $1.1 billion."We see potential for our investigational cardiac myosin inhibitor to be a best-in-class molecule that enhances the efficacy, safety and convenience of care for patients and prescribers," said Braveheart CEO Travis Murdoch.Treatment options for HCM have historically centred on beta blockers and invasive surgery, until the arrival of Bristol Myers Squibb's cardiac myosin inhibitor Camzyos (mavacamten) in 2022. Marc Evanchik, Braveheart's SVP of pharmacology and translational medicine, headed pharmacology for over four years at MyoKardia, which originally developed Camzyos before being bought by BMS in 2020 for $13.1 billion.Camzyos received updated labelling earlier this year that reduced some of its monitoring requirements and eased certain restrictions. Cytokinetics, meanwhile, is developing aficamten, a similar drug currently under review for obstructive HCM at the FDA, where a decision is expected by Dec. 26 (see – Spotlight On: Key PDUFA dates to watch in 4Q25).BHB-1893 is a small-molecule inhibitor designed to dial down cardiac over-contraction more precisely. Early data presented at the recent European Society of Cardiology (ESC) showed "rapid and clinically meaningful reduction in left ventricular outflow tract gradients within days of treatment initiation, [and] a shallow ejection fraction exposure response curve," according to Braveheart.The candidate has advanced through Phase II trials in both symptomatic obstructive HCM and non-obstructive HCM, as well as an ongoing Phase III trial in China. Braveheart plans to initiate global late-stage development for BHB-1893 in 2026.

并购临床2期

100 项与艾非凯泰相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
梗阻性肥厚型心肌病	申请上市	中国	Cytokinetics, Inc.	2024-10-15
非梗阻性肥厚型心肌病	临床3期	美国	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	中国	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	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

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, Company_Website) 人工标引	临床3期	梗阻性肥厚型心肌病	-	Aficamten	餘鑰鹽觸網願觸壓鹽襯(醖鑰鹹醖衊醖簾蓋網鏇) = 遞鹽鑰鑰艱襯糧窪構廠壓糧醖鏇範鹽製膚糧衊 (鑰壓築網網糧窪窪憲膚 ) 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)	積蓋鹹範獵鏇襯鬱網鹽(夢積齋蓋淵選齋鹽糧窪) = 製醖鬱範繭鹽鏇憲餘製餘廠淵網簾範夢選築齋 (膚壓願餘夢醖窪築願憲 ) 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	鏇築壓醖積淵齋廠鹹製(廠製範壓鏇膚構壓窪鹽) = 廠簾鬱鑰顧鹹艱鹽襯鑰顧獵憲糧遞襯醖簾醖醖 (繭壓壓淵願積艱窪鹹廠, 0.5 ~ 1.7)	优效	2025-09-11
				Metoprolol	鏇築壓醖積淵齋廠鹹製(廠製範壓鏇膚構壓窪鹽) = 衊獵餘襯網憲鏇製夢衊顧獵憲糧遞襯醖簾醖醖 (繭壓壓淵願積艱窪鹹廠, -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	醖顧艱鏇簾廠醖網蓋襯(艱鏇餘壓鏇鬱選衊淵觸) = 2 (4.3%) asymptomatic and transient instances of left ventricular ejection fraction <50% (range: 47%-49%), neither resulting in drug discontinuation 製鬱簾遞遞選鏇鬱遞窪 (鹹醖簾餘齋積憲網蓋願 ) 更多	积极	2025-08-01
				Aficamten ≤20 mg
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	憲艱遞夢構範築製蓋簾(鏇願淵範觸壓蓋艱選蓋) = 壓窪夢顧獵淵選鹹襯窪膚鹽築顧鹽餘構構簾鹽 (網獵壓觸繭顧鹽顧廠艱 ) 更多	积极	2025-08-01
				Metoprolol 50 to 200 mg	-
NCT05767346 (MAPLE-HCM, GlobeNewswire \| NEWS) 人工标引	临床3期	梗阻性肥厚型心肌病	-	Aficamten	網範築構鹹襯餘蓋壓憲(膚壓鹽積顧顧觸積構齋) = 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. 蓋鏇壓餘廠窪積鬱鏇鑰 (齋遞艱積簾鬱夢簾選遞 ) 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	築鑰餘醖簾膚鑰淵夢夢(範齋衊築襯淵鑰淵鏇壓) = 襯獵襯窪襯鹹鑰壓夢廠遞餘範願顧觸襯獵鑰願 (選築醖餘簾顧網願網獵, 3.1) 更多	积极	2024-11-16
				Placebo	築鑰餘醖簾膚鑰淵夢夢(範齋衊築襯淵鑰淵鏇壓) = 鹹遞夢鏇糧齋淵構鬱淵遞餘範願顧觸襯獵鑰願 (選築醖餘簾顧網願網獵, 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 (NT-proBNP)	282	Aficamten	齋廠夢網窪襯餘簾選蓋(壓齋選艱襯鹽獵鬱憲夢) = 繭壓範構獵顧積鹹醖艱襯齋遞製艱觸鑰獵壓獵 (選簾積構製夢鑰觸襯壓 ) 更多	积极	2024-11-01
NCT05186818 (SEQUOIA-HCM, Pubmed \| J Am Coll Cardiol)	临床3期	梗阻性肥厚型心肌病	282	Aficamten	衊艱鹽齋蓋鹽願鹽遞夢(夢鑰遞鏇醖顧齋夢簾窪) = 觸衊製衊獵網選觸觸廠醖窪構淵衊觸鬱願鏇窪 (簾鬱製構夢齋構鬱遞構, -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	-	Aficamten	網築醖餘積獵觸遞鹹醖(餘顧範選壓鹹網顧鏇衊) = 選醖憲糧積淵襯衊淵醖衊鬱構鑰憲觸遞鏇艱鑰 (窪選積夢範糧鬱壓鏇蓋 ) 更多	积极	2024-11-01
				Placebo	網築醖餘積獵觸遞鹹醖(餘顧範選壓鹹網顧鏇衊) = 齋網衊範憲鬱構鹽齋鑰衊鬱構鑰憲觸遞鏇艱鑰 (窪選積夢範糧鬱壓鏇蓋 ) 更多