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

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

开始日期2025-06-30

申办/合作机构

Bayer AG

NCT06412666

/ Recruiting临床2/3期

A Phase 2/3 Multicenter, Randomized, Double-Blind, Placebo-Controlled and Open-Label Extension Trial to Evaluate the Efficacy and Safety of Aficamten in a Pediatric Population With Symptomatic Obstructive Hypertrophic Cardiomyopathy

The purpose of this study is to evaluate the efficacy, safety and PK of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

开始日期2024-05-29

申办/合作机构

Cytokinetics, Inc.

NCT06116968

/ Completed临床3期

An Open-Label Study of Aficamten for Chinese Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy

This is an open-label extension study of China cohort in the phase 3 study (CY 6031) of aficamten for the treatment of obstructive HCM (oHCM) to collect long-term safety and tolerability data, including assessments of cardiac function and steady-state Pharmacokinetics (PK) during chronic dosing with aficamten.

开始日期2023-11-14

申办/合作机构

箕星药业科技（上海）有限公司

100 项与阿夫凯泰相关的临床结果

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100 项与阿夫凯泰相关的专利（医药）

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项与阿夫凯泰相关的文献（医药）

2026-02-01·CARDIOVASCULAR DRUGS AND THERAPY

Novel Selective Cardiac Myosin-Targeted Inhibitors Alleviate Myocardial Ischaemia–Reperfusion Injury

Article

作者: Davidson, Sean M ; Yellon, Derek M ; Yusof, Nur Liyana Mohammed

Abstract:

Purpose:

Reperfusion of the ischaemic heart is essential to limit myocardial infarction. However, reperfusion can cause cardiomyocyte hypercontracture. Recently, cardiac myosin-targeted inhibitors (CMIs), such as Mavacamten (MYK-461) and Aficamten (CK-274), have been developed to treat patients with cardiac hypercontractility. These CMIs are well tolerated and safe in clinical trials. We hypothesised that, by limiting hypercontraction, CMIs may reduce hypercontracture and protect hearts in the setting of ischaemia and reperfusion (IR).

Methods:

We investigated the ability of MYK-461 and CK-274 to inhibit hypercontracture of adult rat cardiomyocytes (ARVC) in vitro following ATP depletion. A suitable dose of CMIs for subsequent in vivo IR studies was identified using cardiac echocardiography of healthy male Sprague Dawley rats. Rats were anaesthetized and subject to coronary artery ligation for 30 min followed by 2 h of reperfusion. Prior to reperfusion, CMI or vehicle was administered intraperitoneally. Ischaemic preconditioning (IPC) was used as a positive control group. Infarct size was assessed by tetrazolium chloride staining and extent of hypercontracture was assessed by histological staining.

Results:

Treatment with CMIs inhibited ARVC hypercontracture in vitro . MYK-461 (2 mg/kg) and CK-274 (0.5 mg/kg to 2 mg/kg) significantly reduced infarct size vs. vehicle. IR caused extensive contraction band necrosis, which was reduced significantly by IPC but not by CMIs, likely due to assay limitations. GDC-0326, an inhibitor of PI3Kα, abrogated CK-274-mediated protection following IR injury. GDC-0326 reduced phosphorylation of AKT when administered together with CK-274.

Conclusion:

This study identifies CMIs as novel cardioprotective agents in the setting of IR injury.

2026-02-01·JACC-Heart Failure

Concomitant Aficamten and Disopyramide in Symptomatic Obstructive Hypertrophic Cardiomyopathy

Article

BACKGROUND:

Disopyramide, used in obstructive hypertrophic cardiomyopathy (oHCM) for its negative inotropic properties mediated by its reduction in cytosolic calcium, has been recommended for decades as an option to relieve resistant obstruction. Aficamten is a selective cardiac myosin inhibitor that reduces hypercontractility directly by reducing myosin-actin interaction.

OBJECTIVES:

This study aims to investigate the safety and efficacy of concomitant use and withdrawal of disopyramide in patients with symptomatic oHCM receiving aficamten.

METHODS:

Patients with oHCM enrolled in REDWOOD-HCM Cohort 3 (open-label), SEQUOIA-HCM (placebo-controlled), and FOREST-HCM (open-label) were analyzed. The authors identified 4 groups, each with patients symptomatic despite background therapy with disopyramide who received: 1) disopyramide plus aficamten and subsequent aficamten withdrawal per protocol (Diso-Afi Withdrawal); 2) disopyramide plus placebo (Diso-Pbo); 3) aficamten plus disopyramide with subsequent disopyramide withdrawal (Afi-Diso Withdrawal); and 4) continued both disopyramide and aficamten (Diso+Afi Continuous). Assessments were performed at baseline, after aficamten or placebo add-on therapy, and after washout (except at week 24 for Diso+Afi Continuous group).

RESULTS:

Overall, 50 unique patients from 3 trials enrolled, resulting in 93 subjects (segments) across 4 groups: Diso-Afi Withdrawal (n = 29), Diso-Pbo (n = 20), Afi-Diso Withdrawal (n = 17), and Diso+Afi Continuous (n = 27); mean disopyramide dose was 331 ± 146 mg/d. The addition of aficamten to disopyramide alleviated left ventricular outflow tract (LVOT) obstruction (resting: change [Δ] in least squares mean -27.0 ± 3.6, Valsalva: Δ least squares mean -39.2 ± 5.0, both P < 0.0001), symptoms (≥1 NYHA functional class improvement: 77.8% [95% CI: 61.0-94.5]; P < 0.0001; Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score: 12.3 ± 3.3 [P < 0.001]), and reduced N-terminal pro-B-type natriuretic peptide ratio: 0.35 [95% CI: 0.26-0.48]; P < 0.0001, and there was no significant change with placebo. Withdrawal of aficamten while on disopyramide resulted in return of LVOT obstruction, worsening of symptoms, and increase in NT-proBNP to baseline values. Conversely, withdrawal of disopyramide while on aficamten did not impact efficacy. There were no safety events associated with aficamten or disopyramide withdrawal, and no episodes of atrial fibrillation after disopyramide withdrawal.

CONCLUSIONS:

In this cohort of patients with symptomatic oHCM with persistent LVOT obstruction, combination therapy with aficamten and disopyramide was safe and well tolerated but did not enhance clinical efficacy vs aficamten alone. For such oHCM patients, aficamten treatment may be considered with an option to discontinue disopyramide. (Dose-finding Study to Evaluate the Safety, Tolerability, PK, and PD of CK-3773274 in Adults With HCM [REDWOOD-HCM]; NCT04219826) (Aficamten vs Placebo in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy [SEQUOIA-HCM]; NCT05186818) (Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Aficamten in Adults With HCM [FOREST-HCM]; NCT04848506).

2026-02-01·Circulation-Heart Failure

Efficacy and Safety of Aficamten in Children and Adolescents With Obstructive Hypertrophic Cardiomyopathy: Study Design and Rationale of CEDAR-HCM

Article

作者: Chiu, Michael ; Olivotto, Iacopo ; Melloni, Chiara ; Russell, Mark W. ; Kaski, Juan Pablo ; Maharao, Neha ; Nieto Morales, Paula F. ; Simkins, Tyrell ; Malik, Fady I. ; Lutz, Justin ; Ho, Carolyn Y. ; Kupfer, Stuart ; Nakano, Stephanie J. ; Jacoby, Daniel L. ; Godown, Justin ; German, Polina ; Wei, Jenny ; Kantor, Paul F. ; Heitner, Stephen B.

BACKGROUND::

Hypertrophic cardiomyopathy (HCM) is an important cause of morbidity and mortality in children, but treatment options are limited. Aficamten, a next-in-class cardiac myosin inhibitor, directly targets the hypercontractility underlying HCM. Aficamten improved exercise capacity, health status, and symptoms in adults with obstructive HCM in the pivotal, phase 3 SEQUOIA-HCM trial (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM; NCT05186818).

METHODS::

CEDAR-HCM (Clinical Evaluation of dosing With Aficamten to Reduce Obstruction in Pediatric Population With HCM) is an international, multicenter, randomized, double-blind, placebo-controlled trial followed by an open-label extension to evaluate the efficacy, safety, and pharmacokinetics of aficamten in pediatric participants with symptomatic obstructive HCM. The trial will enroll ≈55 adolescents (12 to <18 years) and subsequently expand to include at least 10 children (6 to <12 years) with nonsyndromic obstructive HCM, left ventricular ejection fraction ≥60%, Valsalva left ventricular outflow tract gradient ≥50 mm Hg, and New York Heart Association functional class ≥II. Participants will be randomized 2:1 to aficamten or placebo in addition to standard of care therapy or as monotherapy, with echocardiogram-guided dose adjustments targeting a Valsalva left ventricular outflow tract gradient <30 mm Hg while maintaining left ventricular ejection fraction ≥50%. The primary end point is the change in Valsalva left ventricular outflow tract gradient from baseline to week 12. Secondary end points include change in resting left ventricular outflow tract gradient, cardiac biomarkers, New York Heart Association functional class, and assessment of pharmacokinetics. After completing the 12-week randomized period, eligible participants will continue into a long-term open-label extension.

RESULTS::

The trial is currently enrolling.

CONCLUSIONS::

Results of CEDAR-HCM will provide insight into the safety and efficacy of aficamten in adolescents and in children as young as 6 years of age.

REGISTRATION::

URL: https://www.clinicaltrials.gov ; Unique identifier: NCT06412666.

555

项与阿夫凯泰相关的新闻（医药）

2026-02-20

·精准药物

2025年中国国家药品监督管理局（NMPA）批准的42款小分子药物及合成路线

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抑制剂。用于至少接受过一种系统性治疗的KRASG12C 突变型的晚期非小细胞肺癌成人患者。获批时间为2025年5月22日。 Retagliptin&Metformin Retagliptin&Metformin由山东盛迪医药有限公司研发，活性成分为瑞格列汀二甲双胍（I）/（II），商品名为瑞霖唐，是瑞格列汀和二甲双胍复合制剂。适用于适合接受磷酸瑞格列汀和盐酸二甲双胍治疗的2型糖尿病成人患者改善血糖控制。获批时间为2025年5月22日。本文介绍瑞格列汀的合成。 Fosrolapitant&Palonosetron Fosrolapitant&Palonosetron由福建盛迪医药有限公司研发，活性成分为磷罗拉匹坦及帕洛诺司琼，商品名为瑞坦宁，分别属于神经激肽1受体拮抗剂和5-HT3拮抗剂。适用于预防成人高度致吐性化疗（HEC）引起的急性和迟发性恶心和呕吐。获批时间为2025年5月29日。 Fosrolapitant Palonosetron Lerociclib Lerociclib由Genor Biopharma(USA), Inc.研发，活性成分为来罗西利（Lerociclib），商品名为汝佳宁，属于CDK4/6抑制剂。适用于HR+/HER2- 局部晚期或转移性乳腺癌成人患者：与芳香化酶抑制剂联合使用作为初始内分泌治疗；与氟维司群联合用于既往接受内分泌治疗后疾病进展的患者。获批时间为2025年5月29日。 Gecacitinib Gecacitinib由苏州泽璟生物制药股份有限公司研发，活性成分为吉卡昔替尼（Gecacitinib），商品名为泽普平，属于JAK和ACVR1双靶点抑制剂。用于中危或高危原发性骨髓纤维化（PMF）、真性红细胞增多症继发性骨髓纤维化（PPV-MF）和原发性血小板增多症继发性骨髓纤维化（PET-MF）的成人患者，治疗疾病相关脾肿大或疾病相关症状。获批时间为2025年5月29日。 Luvometinib Luvometinib由上海复星医药产业发展有限公司研发，活性成分为芦沃美替尼（Luvometinib），商品名为复迈宁，属于MEK1/2抑制剂。适用于朗格汉斯细胞组织细胞增生症（LCH）和组织细胞肿瘤成人患者；2岁及2岁以上伴有症状、无法手术的丛状神经纤维瘤（PN）的I型神经纤维瘤病（NF1）儿童及青少年患者。获批时间为2025年5月29日。 Fovinaciclib Fovinaciclib由锦州奥鸿药业有限责任公司研发，活性成分为伏维西利（Fovinaciclib），商品名为复妥宁，属于CDK4/6抑制剂。联合氟维司群，适用于既往接受内分泌治疗后出现疾病进展的HR+/HER2- 阴性的复发或转移性成年乳腺癌患者。获批时间为2025年5月29日。 Deutenzalutamide Deutenzalutamide由海创药业股份有限公司研发，活性成分为氘恩扎鲁胺（Deutenzalutamide），商品名为海纳安，属于雄激素受体拮抗剂。适用于接受醋酸阿比特龙及化疗后出现疾病进展，且既往未接受新型雄激素受体抑制剂的转移性去势抵抗性前列腺癌（mCRPC）成人患者。获批时间为2025年5月29日。 Famitinib Famitinib由江苏恒瑞医药股份有限公司研发，活性成分为法米替尼（Famitinib），商品名为艾比特，属于多靶点酪氨酸激酶受体抑制剂。联合注射用卡瑞利珠单抗用于既往接受含铂化疗治疗失败但未接受过贝伐珠单抗治疗的复发或转移性宫颈癌患者。获批时间为2025年5月29日。 Tibremciclib Tibremciclib由贝达药业股份有限公司研发，活性成分为泰瑞西利（Tibremciclib），商品名为康美纳，属于CDK4/6抑制剂。联合氟维司群，适用于既往接受内分泌治疗后进展的HR+/HER2- 的局部晚期或转移性成年乳腺癌患者。获批时间为2025年7月2日。 Ifupinostat Ifupinostat由广州必贝特医药股份有限公司研发，活性成分为伊吡诺司他（Ifupinostat），商品名为贝特琳，属于HDAC/PI3Kα双靶点抑制剂。适用于既往接受过至少两线系统性治疗的复发或难治性弥漫性大B细胞淋巴瘤成人患者。获批时间为2025年7月2日。 Smilaxextract Smilaxextract由苏州旺山旺水生物医药股份有限公司研发，活性成分为司美那非（Smilaxextract），商品名为昂伟达，属于磷酸二酯酶5抑制剂。适用于治疗勃起功能障碍。获批时间为2025年7月11日。 Lisaftoclax Lisaftoclax由苏州亚盛药业有限公司研发，活性成分为利沙托克拉（Lisaftoclax），商品名为利生妥，属于Bcl-2 和Bcl-xl 的双抑制剂。适用于既往经过至少包含布鲁顿酪氨酸激酶（BTK）抑制剂在内的一种系统治疗的成人慢性淋巴细胞白血病（CLL）/小淋巴细胞淋巴瘤（SLL）患者。获批时间为2025年7月11日。 Sebaloxavir Marboxil Sebaloxavir Marboxil由南京征祥医药有限公司研发，活性成分为玛硒洛沙韦（Sebaloxavir Marboxil），商品名为济可舒，属于RNA聚合酶PA抑制剂。适用于既往健康的成人单纯性甲型和乙型流感患者的治疗，不包括存在流感相关并发症高风险的患者。获批时间为2025年7月18日。 Tunodafil Tunodafil由扬子江药业集团有限公司研发，活性成分为妥诺达非（Tunodafil，曾用名：Yonkenafil），商品名为泰妥妥，属于磷酸二酯酶-5A 抑制剂。适用于治疗勃起功能障碍。获批时间为2025年7月25日。 Atrasentan Atrasentan由诺华研发，活性成分为阿曲生坦（Atrasentan），商品名为诺锐达，属于内皮素A受体拮抗剂。适用于降低具有疾病快速进展风险的原发性IgA肾病成人患者的蛋白尿。获批时间为2025年8月22日。 Dirozalkib Dirozalkib由轩竹生物科技股份有限公司研发，活性成分为地罗阿克（Dirozalkib），商品名为轩菲宁，属于ALK抑制剂。单药适用于未接受过ALK抑制剂治疗的ALK阳性局部晚期或转移性非小细胞肺癌患者。获批时间为2025年8月22日。 Zongertinib Zongertinib由勃林格殷格翰研发，活性成分为宗艾替尼（Zongertinib），商品名为圣赫途，属于HER2抑制剂。单药适用于存在HER2激活突变且既往接受过至少一种系统治疗的不可切除局部晚期或转移性NSCLC成人患者。获批时间为2025年8月29日。 Zeprumetostat Zeprumetostat由江苏恒瑞医药股份有限公司研发，活性成分为泽美妥司他（Zeprumetostat），商品名为艾瑞璟，属于EZH2抑制剂。适用于既往接受过至少1线系统治疗的复发或难治性外周T细胞淋巴瘤成人患者。获批时间为2025年8月29日。 Mufemilast Mufemilast由赣州和美药业股份有限公司研发，活性成分为莫米司特（Mufemilast），属于磷酸二酯酶4（PDE4）抑制剂。适用于符合光疗或系统治疗指征的中度至重度斑块状银屑病成人患者。获批时间为2025年10月13日。 Relmapirazin Relmapirazin由杭州中美华东制药有限公司研发，活性成分为瑞玛比嗪（Relmapirazin），属于荧光示踪剂，用于实时测量肾小球滤过率（GFR）。需与MediBeacon公司的TGFR设备配合使用。获批时间为2025年10月17日。 Nerandomilast Nerandomilast由勃林格殷格翰研发，活性成分为那米司特（Nerandomilast），商品名为博优维，属于PDE4B抑制剂。适用于治疗特发性肺纤维化（IPF）成人患者。获批时间为2025年10月28日。 Mifanertinib Mifanertinib由杭州中美华东制药有限公司研发，活性成分为美凡厄替尼（Mifanertinib），商品名为迈瑞东，属于EGFR抑制剂。适用于EGFR外显子21（L858R）突变的局部晚期或转移性NSCLC成人患者一线治疗。获批时间为2025年10月28日。 Olgotrelvir Sodium Olgotrelvir Sodium由浙江艾森药业有限公司研发，活性成分为奥格特韦钠（Olgotrelvir Sodium），商品名为奥维宁，属于CTSL抑制剂和SARS-CoV-2 3CLpro抑制剂。用于治疗轻中型新冠病毒感染的成年患者。获批时间为2025年11月5日。 Sitokiren Sitokiren由上海上药信谊药厂有限公司研发，活性成分为司妥吉仑（Sitokiren），商品名为信妥安，属于renin抑制剂。适用于原发性高血压患者。获批时间为2025年12月9日。 Culmerciclib Culmerciclib由正大天晴药业集团股份有限公司研发，活性成分为库莫西利（Culmerciclib），商品名为赛坦欣，属于CDK2/4/6抑制剂。联合氟维司群用于HR+/HER2- 的局部晚期或转移性乳腺癌成人患者。获批时间为2025年12月15日。 Pixavir marboxil Pixavir marboxil由健康元药业集团股份有限公司研发，活性成分为玛帕西沙韦（Pixavir marboxil），商品名为壹立康，属于帽依赖内切酶抑制剂。适用于既往健康的12岁及以上青少年和成人甲型和乙型流感患者。获批时间为2025年12月15日。 Zurletrectinib Zurletrectinib由北京诺诚健华医药科技有限公司研发，活性成分为佐来曲替尼（Zurletrectinib），商品名为宜诺欣，属于酪氨酸激酶抑制剂。用于携带NTRK融合基因的局部晚期或转移性实体瘤患者。获批时间为2025年12月15日。 Aficamten Aficamten由Cytokinetics, Incorporated研发，活性成分为阿夫凯泰（Aficamten），商品名为星舒平，属于心肌肌球蛋白抑制剂。用于NYHA II–III级梗阻性肥厚型心肌病成人患者。获批时间为2025年12月22日。 Pimicotinib Pimicotinib由默克雪兰诺（北京）医药有限公司研发，活性成分为匹米替尼（Pimicotinib），商品名为贝捷迈，属于CSF1R抑制剂。用于症状性腱鞘巨细胞瘤（TGCT）成年患者。获批时间为2025年12月22日。声明：发表/转载本文仅仅是出于传播信息的需要，并不意味着代表本公众号观点或证实其内容的真实性。据此内容作出的任何判断，后果自负。若有侵权，告知必删！长按关注本公众号粉丝群/投稿/授权/广告等请联系公众号助手觉得本文好看，请点这里↓

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2026-02-20

·GoodGood123

人类药物治疗开发进展⑥心血管内科药物下篇

一、从中药动植物中提取强心甙(见上篇) 二、强心甙结构改造与合成衍生物(见上篇) 三、合成化学药物治疗(见中篇) 四、精准治疗与长效制剂药物突破(见本篇) 21世纪初心血管内科治疗药物从长效治疗、基因治疗、精准治疗前沿方向推进。 ①NOAC（达比加群、利伐沙班）替代华法林药物，无需监测、出血风险更低。 ②PCSK9单抗如依洛尤单抗、阿利昔尤单抗等，有强效降脂作用，用于他汀不耐受/难治性高胆固醇血症，能改善心血管疾病患者的预后。 ③糖尿病与心血管保护药物SGLT-2抑制剂如达格列净、恩格列净等，不仅能降糖，还能降低心血管疾病患者的不良事件风险，改善心力衰竭患者的预后。GLP-1受体激动剂如利拉鲁肽、司美格鲁肽等，兼具降糖、减重和心血管保护作用，被推荐为2型糖尿病合并心血管疾病患者的一线治疗用药。 ④心力衰竭治疗药物血管紧张素受体-脑啡肽酶抑制剂，如沙库巴曲缬沙坦(ARNI)属于心衰治疗里程碑药物，具有双重调节作用，显著降低心血管死亡或心力衰竭再住院风险。 ⑤可溶性鸟苷酸环化酶激动剂如维立西呱，为恶化型射血分数减低的心力衰竭患者带来额外获益。 ⑥盐皮质激素受体拮抗剂如非奈利酮，可改善糖尿病肾病患者的预后，同时降低心力衰竭恶化风险。 ⑦肥厚型心肌病特效药物心肌肌球蛋白抑制剂：如玛伐凯泰、艾非凯泰，通过降低心肌肌球蛋白活性，改善肥厚型心肌病患者的心肌功能障碍，实现了该疾病特效药“从无到有”的突破。 ⑧2023年中国获批siRNA英克司兰钠上市，通过皮下注射每半年给药一次，可持续长效稳定降脂。 ⑧2025中国首批阿夫凯泰、玛伐凯泰，精准靶向心衰与心肌病，用于治疗梗阻性肥厚型心肌病，改善心功能。2025年中国获批司美格鲁肽药物治疗心血管适应症。 ⑩普乐司兰药物三个月打一针，用于难治性高甘油三酯血症。基因编辑靶向药物CTX310一次性永久降脂。治疗动脉粥样硬化药物ABP‑745有抗炎+斑块逆转作用。直接肾素抑制剂信妥安药物，从高血压源头上游阻断降压更平稳，靶器官保护潜力更大。五、心血管内科药物未来趋势。 ①长效化用药：siRNA/基因编辑治疗心血管病。 ②精准化用药：基因检测指导靶向罕见心血管病。 ③多靶点用药：抗炎+降脂+抗栓药联合使用。 ④AI赋能用药：靶点发现加速临床试验设计。 #基因治疗新时代 #精准医疗进展 #基因细胞治疗 #药物研发历程

2026-02-18

·BioPharmaDive

FDA accepts BMS protein degrader for review; Disc rare disease drug rejected

Listen to the article 3 min This audio is auto-generated. Please let us know if you have feedback. Today, a brief rundown of news from Bristol Myers Squibb and Cytokinetics as well as updates from Disc Medicine and Teva Pharmaceutical that you may have missed.The Food and Drug Administration has accepted Bristol Myers Squibbs approval application for its experimental multiple myeloma drug iberdomide, setting a decision deadline of Aug. 17, the company said Tuesday. The drug, from a new class of protein-degrading treatments, is intended for use in combination with Johnson & Johnsons Darzalex and the steroid dexamethasone in people whose disease has advanced or become resistant to early lines of treatment. In a Phase 3 trial, iberdomide plus Darzalex and dexamethasone helped significantly more people with multiple myeloma become minimal residual disease negative compared to older combination treatment. Iberdomides progress is one of the most important events in Bristol Myers year as it tries to shake off a 2025 that included a round of cost cuts and clinical setbacks. Jonathan GardnerMeanwhile, the FDA rejected Disc Medicines application for accelerated approval of bitopertin,the company said last Friday, though it indicated that it could evaluate data from a Phase 3 study of the drug for the rare disease erythropoietic protoporphyria in a traditional setting. In a complete response letter, the FDA wrote that two trials of bitopertin confirmed the drugs benefit in lowering levels of protoporphyrin IX, a substance that builds up in the red blood cells of people with EPP and causes extreme sensitivity to light. However, the agency wrote there are uncertainties regarding whether lower levels of protoporphyrin IX correlated to meeting sunlight exposure endpoints laid out in one of Discs studies. The company received a national priority voucher last fall to speed up its review, though a report published by Stat Newsin the months following revealed skepticism over the drugs efficacy from Center for Biologics Evaluation and Research Director Vinay Prasad. Gwendolyn WuThe European Commission has approved Cytokinetics pill Myqorzo to treat the heart condition obstructive hypertrophic cardiomyopathy, the company said Tuesday. The approval for the 27 member countries of the European Union follows its U.S. OK by about two months. In the EU, Myqorzo could have some advantages over Bristol Myerscompeting drug Camzyos, including more flexible dosing and no requirement for genotyping,Stifel analyst James Condulis wrote in a note to clients. Camzyos, from the same class of drugs called cardiac myosin inhibitors, recorded sales of $1.1 billion in 2025, with its international sales growth outpacing that in the U.S. Jonathan GardnerSanofi and Teva Pharmaceutical said their experimental antibody drug for ulcerative colitis and Crohns disease, duvakitug, maintained its efficacy over 44 weeks in the long-term extension stage of a Phase 2 trial. Remission and response rates for the high dose of 900 milligrams improved at the 44-week endpoint for both conditions compared with those seen at the main, 14-week endpoint, Jefferies analyst Dennis Ding wrote in a note to clients. That response rate compares favorably to competing drugs from the TL1A class being developed by Merck & Co. and Roche, Ding wrote. Merck and Roche have advanced their drugs into Phase 3 development. Jonathan Gardner '

临床3期临床结果上市批准临床2期加速审批

100 项与阿夫凯泰相关的药物交易

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

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适应症	最高研发状态	国家/地区	公司	日期
非梗阻性肥厚型心肌病	临床3期	美国	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	中国	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	日本	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	阿根廷	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	澳大利亚	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	巴西	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	加拿大	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	丹麦	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	法国	Cytokinetics, Inc.	2023-08-30
非梗阻性肥厚型心肌病	临床3期	德国	Cytokinetics, Inc.	2023-08-30

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT06412666 \| NCT05186818 (CEDAR-HCM, Pubmed \| Circ Heart Fail)	临床3期	梗阻性肥厚型心肌病	65	Aficamten	獵鏇蓋遞網鑰鬱鑰壓餘(構選糧繭壓範蓋積築夢) = 壓網鬱齋鹹膚製壓簾壓積淵繭窪醖築鑰齋襯鹽 (製餘憲鹽觸鹹醖獵廠夢 )	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	蓋齋顧觸築製顧鑰範簾(繭築網網糧構鏇蓋遞鏇) = 艱構鬱範範遞廠鏇蓋獵窪網餘願餘簾網齋顧蓋 (鑰鏇觸膚糧膚遞範壓鑰 ) 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	餘衊憲膚醖襯衊積願淵(糧餘壓艱夢積餘膚鏇顧) = 憲範網鹽範構鹹淵壓廠襯齋製築夢鬱憲製遞衊 (壓選鹽築鬱蓋醖顧鹹繭 ) 更多	积极	2025-11-01
				Metoprolol	餘衊憲膚醖襯衊積願淵(糧餘壓艱夢積餘膚鏇顧) = 鹽醖構鑰網製餘蓋淵淵襯齋製築夢鬱憲製遞衊 (壓選鹽築鬱蓋醖顧鹹繭 ) 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	衊願繭積鏇顧鏇蓋襯憲(構襯淵壓積醖廠窪醖遞): Difference = 7.8 (95.0% CI, 3.3 ~ 12.3), P-Value = < 0.001 更多	积极	2025-11-01
				Metoprolol
NCT05767346 (MAPLE-HCM, Company_Website) 人工标引	临床3期	梗阻性肥厚型心肌病	-	Aficamten	網網網積蓋鹹醖衊範鏇(簾構淵衊範窪衊鏇選淵) = 鑰廠簾襯鑰衊衊遞構夢鬱獵鹹夢糧鹹觸鑰觸窪 (淵齋憲鏇鬱醖獵選選窪 ) 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)	遞衊窪壓糧獵窪鹽簾膚(願顧鹽糧蓋構顧顧憲憲) = 夢繭鹹願襯醖憲築膚顧觸壓夢積範遞鏇範鑰淵 (齋選醖選艱繭艱餘鹽獵 ) 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	範窪構顧壓繭餘憲窪顧(鹽範夢繭繭製範廠積艱) = 鹹選積蓋衊糧簾蓋製蓋積遞夢鏇夢膚蓋醖顧衊 (繭膚選艱艱膚壓顧鏇構, 0.5 ~ 1.7)	优效	2025-09-11
				Metoprolol	範窪構顧壓繭餘憲窪顧(鹽範夢繭繭製範廠積艱) = 鏇顧壓淵醖壓鏇艱顧窪積遞夢鏇夢膚蓋醖顧衊 (繭膚選艱艱膚壓顧鏇構, -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	衊餘繭淵簾鏇淵衊壓鑰(顧窪醖齋夢遞襯壓獵鑰) = 2 (4.3%) asymptomatic and transient instances of left ventricular ejection fraction <50% (range: 47%-49%), neither resulting in drug discontinuation 襯構鹹選觸網齋壓網蓋 (積鬱廠餘構築網積膚積 ) 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	願獵遞構齋糧衊糧齋簾(鑰醖衊築衊餘鹽蓋網顧) = 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. 蓋艱觸餘蓋繭顧積鑰齋 (網構蓋廠範積蓋淵製願 ) 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	壓蓋廠膚遞襯蓋願壓窪(窪廠衊淵夢繭蓋齋鹽鏇) = 淵鹹淵齋廠憲範壓鹽廠積鏇構觸築艱築鹽餘餘 (餘獵願鹹鏇選膚衊簾積, 3.1) 更多	积极	2024-11-16
				Placebo	壓蓋廠膚遞襯蓋願壓窪(窪廠衊淵夢繭蓋齋鹽鏇) = 範壓廠願餘醖餘醖衊餘積鏇構觸築艱築鹽餘餘 (餘獵願鹹鏇選膚衊簾積, 2.7) 更多