Aficamten

跨国药企在中国重点资讯 文 | 苏丁 企业动态 碧迪 碧迪医疗科技(江苏)有限公司12月18日开业，标志着碧迪医疗(BD)在中国的第三座世界级本土生产基地正式投入运营。碧迪医疗宜兴工厂总投资额高达13.32亿元人民币，占地面积54亩，总建筑面积为35000平方米。全面建成后，预计年产能将达到瓶装注射剂250万升，预填充盐水注射器4亿支。目前，碧迪医疗已在中国构建起一个涵盖3座本土制造工厂、2个研发中心、6个培训中心、1个客户服务中心、1个客户体验中心以及1个创新中心于一体的强大基础设施网络。 洁定 瑞典洁定医疗集团（Getinge）医疗科技及生命科学创新体验中心（GEC）在上海揭幕。GEC作为一个集展示最新医疗科技成果、提供专业技能培训、以及促进科研合作于一体的综合性多功能平台，是洁定全球第5个也是最大的创新体验中心。其占地面积超过2000平方米，设置了多个实操功能区域，包括先进的复合手术室、标准化的心血管外科手术室和骨科手术室、设备完善的重症监护室、高效运作的消毒供应中心以及前沿的生命科学实验室。 勃林格殷格翰 位于深圳市罗湖区的“勃林格殷格翰-中欧中心联合创新孵化器”正式启用。这一孵化器以国际一流生物医药研发标准为指引，配备顶尖科研设备、先进实验仪器和高品质办公环境以及全方位企业辅导与商业化支持，是大湾区首个由跨国药企与创新中心深度合作的高规格创新平台。该联合创新孵化器的启用，标志着大湾区生命健康产业迈入新台阶，也为全国生物医药创新注入了强劲动能。 由中国红十字基金会发起，勃林格殷格翰中国公益支持的全国首个“精神卫生关爱小屋”12月19日正式落地上海市精神卫生中心，将聚焦精神分裂症等精神障碍疾病患者的预后及康复干预，旨在帮助患者增强疾病认知、消除病耻感、恢复社会功能、最终促进他们重新融入并回归社会。在这里将开展一系列针对患者的康复活动。 武田 第四届武田中国创新挑战赛路演顺利举办，经过专业评委团队的评估和遴选，最终5个极具临床应用潜力的数字医疗创新项目从报名参赛的47个创意想法中脱颖而出，进入概念性验证(PoC)阶段。获胜项目涵盖罕见病、消化及免疫、神经科学等领域，以医疗领域的“新质生产力”为武田中国旗下开放式创新孵化平台TakedaSpark注入了新活力。 默沙东 默沙东中国与中央广播电视总台携手，在中国人民大学举办“健康中国说·青春校园行”活动，围绕大学生最关心的“HPV感染”等健康困扰，通过丰富多样的创新形式科普相关知识。大学生正处于做好HPV预防的关键年龄。全国多地数百所高校陆续开展了一系列开学季HPV健康科普活动，将HPV创意科普带入校园。 鹏瑞利 鹏瑞利集团有限公司宣布，位于中国天津市的鹏瑞利综合医院（天津鹏瑞利医院）已成功通过该市卫健委的验收评审，并获得了《医疗机构执业许可证》，成为中国首家外商独资三级综合医院。该三级综合医院设置500张床位，总投资额约10亿元人民币，将很快开始运营。天津鹏瑞利医院位于西青区，紧邻天津南站，是天津南站鹏瑞利国际健康商旅城医疗板块的重要组成部分。 百汇医疗 百汇医疗入华二十周年暨品牌升级典礼12月11日在上海举行。百汇医疗隶属于全球最大私立医疗服务网络之一的IHH医疗集团，深耕中国市场二十年。百汇医疗围绕“精准关怀，精湛为你”的理念，以焕然一新的形象和全面升级的服务配置，满足患者持续变化的需求。预计在2025年，位于上海广场的全新大型旗舰医疗中心将开业。中心占地近7,000平方米，将提供多学科医疗服务。 产业动态 默沙东(MSD)表示，根据协议条款，该公司将获得中国制药商翰森制药的HS-10535的全球独家许可，HS-10535是一款针对GLP-1受体的实验性口服药物。默沙东将向翰森制药支付1.12亿美元的首付款，并可能额外支付19亿美元的里程碑付款。 赛诺菲与箕星药业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。该交易预计于2024年内完成，交易金额不对外公开。 丹纳赫集团旗下SCIEX本土化质谱产品交付量突破100台。2023年9月，SCIEX成功实现SCIEX 4500质谱系统及SCIEX 5500+质谱系统在中国的本土化生产落地。随后，SCIEX进一步加速本土化步伐，并于2024年10月宣布Triple Quad 6500+质谱系统和QTRAP 6500+质谱系统成功实现本土化生产。到2025年，SCIEX计划进一步扩大本地化生产规模，推出更多本土化高端产品。 波士顿科学公司宣布旗下新一代血管内超声（IVUS）诊断系统AVVIGO+正式获得国家药品监督管理局批准，用于冠心病的诊疗。该系统具备人工智能辅助血管评估功能，可优化医生在经皮冠状动脉介入（PCI）治疗中的评估流程，缩短操作时间。 住友制药与上药控股宣布，双方再次签订合同销售组织（CSO）合作协议，进一步深化双方在处方药联合营销领域的战略合作。2020年，双方首次签订CSO合作协议，启动了针对阿尔马尔（通用名：盐酸阿罗洛尔片）的合作。 复宏汉霖与Palleon Pharmaceuticals Inc.宣布签署合作与许可协议，以开发和商业化Palleon的同类首创（first-in-class）人唾液酸酶融合蛋白E-602与复宏汉霖自主开发的汉利康（利妥昔单抗）的联合疗法，用于治疗包括狼疮肾炎（LN）在内的自身免疫疾病。复宏汉霖获得E-602在中国的独家许可。Palleon有资格获得高达9530万美元的开发和商业化里程碑付款，以及E-602实现中国商业化后的销售特许权使用费。 药明生物和杭州多禧生物科技有限公司与Aadi Bioscience达成研究服务合作协议，支持Aadi公司研发三款处于临床前阶段的新一代抗体偶联药物（ADCs）。根据协议条款，Aadi公司将向药明生物和多禧生物支付4400万美元首付款，最高达2.65亿美元的开发里程碑付款和5.4亿美元的商业里程碑付款，以及个位数比例的销售提成。 北京大学首钢医院成立肿瘤治疗皮肤不良反应多学科管理门诊。这是理肤泉在中国推动和建立的第三家肿瘤治疗相关皮肤不良反应多学科管理门诊，也是北京地区首家肿瘤治疗相关皮肤不良反应的一站式管理门诊。 CDE官网公示，阿斯利康1类新药AZD6234注射液获批临床，拟开发用于在至少有一种肥胖相关合并症的超重或者肥胖成年人中长期体重管理。AZD6234是一款多肽类药物，为每周一次皮下注射的长效胰淀素受体激动剂。该产品正在国际范围内进行2b期临床研究，阿斯利康拟开发该产品作为肠促胰岛素不耐受肥胖患者的替代治疗方案。 中国国家药品监督管理局批准礼来公司的阿尔茨海默病疗法，记能达（多奈单抗注射液）用于治疗成人因阿尔茨海默病引起的轻度认知功能障碍和阿尔茨海默病轻度痴呆。多奈单抗是唯一一个有证据支持在清除淀粉样蛋白斑块后可停药的靶向淀粉样蛋白的疗法，这可以通过减少输液次数和治疗成本帮助患者降低治疗负担。 中国国家药监局药品审评中心公示，武田制药1类新药mezagitamab注射剂获得一项新的临床试验默示许可，拟开发治疗原发性免疫性血小板减少症(ITP)。Mezagitamab是一种全人源免疫球蛋白IgG1单克隆抗体(mAb)，Mezagitamab治疗旨在提供快速且持续的血小板应答改善，并恢复血小板计数至功能水平。 联系美通社 +86-10-5953 9500 info@prnasia.com

SOUTH SAN FRANCISCO, Calif., Dec. 23, 2024 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for aficamten, a next-in-class cardiac myosin inhibitor, for the treatment of obstructive hypertrophic cardiomyopathy (HCM). The MAA will now be reviewed by the Committee for Medicinal Products for Human Use (CHMP). “With regulatory filings for aficamten already under review in both the U.S. and China, the validation of the MAA marks an important milestone in bringing this potential medicine to even more patients with HCM worldwide,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “We look forward to working with EMA in connection with their review of our application.” The MAA is supported by the results from SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic obstructive HCM, which were published in the New England Journal of Medicine.1 The MAA validation follows the acceptance by the U.S. Food and Drug Administration (FDA) of the New Drug Application (NDA) for aficamten for the treatment of obstructive HCM. The FDA assigned the NDA a standard review with a Prescription Drug User Fee Act (PDUFA) target action date of September 26, 2025. About SEQUOIA-HCM SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM) was the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). The results from SEQUOIA-HCM showed that treatment with aficamten for 24 weeks significantly improved exercise capacity compared to placebo, increasing peak oxygen uptake (pVO2) measured by cardiopulmonary exercise testing (CPET) by 1.8 ml/kg/min compared to baseline in patients treated with aficamten versus 0.0 ml/kg/min in patients treated with placebo (least square mean (LSM) difference [95% CI] of 1.74 mL/kg/min [1.04 - 2.44]; p=0.000002). Statistically significant improvements were observed in all 10 prespecified secondary endpoints, including Valsalva left ventricular outflow tract (LVOT) gradient, New York Heart Association (NYHA) Functional Class, Kansas City Cardiomyopathy Clinical Summary Score (KCCQ-CSS), and proportion with LVOT gradient <30 mmHg, each at 12 and 24 weeks, as well as duration of guideline eligibility for septal reduction therapy (SRT), and total workload during CPET at 24 weeks. Treatment emergent serious adverse events occurred in 5.6% and 9.3% of patients on aficamten and placebo, respectively. Core echocardiographic left ventricular ejection fraction (LVEF) was observed to be <50% in 5 patients (3.5%) on aficamten compared to 1 patient (0.7%) on placebo. There were no instances of worsening heart failure or treatment interruptions due to low LVEF. Additional analyses from SEQUOIA-HCM have demonstrated that treatment with aficamten is associated with improvements in cardiac structure, function, and biomarkers without negatively impacting systolic function. 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 and as may translate into next-in-class potential in clinical development. 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 and relieves symptoms in patients with HCM as well as its potential long-term effects on cardiac structure and function. 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 obstructive HCM from the U.S. Food & Drug Administration (FDA) as well as the National Medical Products Administration (NMPA) in China where it is currently also under review for potential approval. Aficamten is also currently being evaluated in MAPLE-HCM, a Phase 3 clinical trial of aficamten as monotherapy compared to metoprolol as monotherapy in patients with obstructive HCM; 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 obstructive HCM; and FOREST-HCM, an open-label extension clinical study of aficamten in patients with HCM. 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 late-stage, specialty cardiovascular biopharmaceutical company focused on discovering, developing and commercializing muscle biology-directed drug candidates as potential treatments for debilitating diseases in which cardiac muscle performance is compromised. As a leader in muscle biology and the mechanics of muscle performance, the company is developing small molecule drug candidates specifically engineered to impact myocardial muscle function and contractility. Cytokinetics is readying for the potential commercialization of aficamten, a next-in-class 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. Cytokinetics is also developing omecamtiv mecarbil, a cardiac myosin activator, in patients with heart failure with severely reduced ejection fraction (HFrEF), CK-586, 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 express or implied relating to the properties or potential benefits of aficamten or any of our other drug candidates, our ability to obtain regulatory approval for aficamten for the treatment of obstructive hypertrophic cardiomyopathy or any other indication from FDA or any other regulatory body in the United States or abroad, and the labeling or post-marketing conditions that FDA or another regulatory body may require in connection with the approval of aficamten. 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 the risks related to Cytokinetics’ business outlines in Cytokinetics’ filings with the Securities and Exchange Commission. Forward-looking statements are not guarantees of future performance, and Cytokinetics’ actual results of operations, financial condition and liquidity, and the development of the industry in which it operates, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that Cytokinetics makes in this press release speak only as of the date of this press release. Cytokinetics assumes no obligation to update its forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release. CYTOKINETICS® and the CYTOKINETICS and C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries. Contact: Cytokinetics Diane Weiser Senior Vice President, Corporate Affairs (415) 290-7757 References: Source: Cytokinetics, Incorporated