CTX-203

▎药明康德内容团队编辑 2亿美元前期付款，恒瑞医药与默沙东就小分子心血管新药达成授权合作 恒瑞医药和默沙东（MSD）今日宣布，双方已签署一项针对HRS‑5346的独家许可协议。HRS‑5346是由恒瑞医药开发的一种口服小分子脂蛋白（a）——Lp（a）抑制剂，目前正在中国开展2期临床试验。 Lp（a）是一类独特的脂蛋白，含有低密度脂蛋白（LDL）样颗粒，具有促动脉粥样硬化、促炎、促钙化等作用。Lp（a）水平升高是最普遍的单基因脂质疾病之一，高Lp（a）是动脉粥样硬化性心血管疾病的独立危险因素，也是主动脉瓣狭窄的危险因素。因此靶向Lp（a）的降脂疗法成为心血管疾病防治的重要突破点之一。 根据协议，默沙东将获得HRS-5346在大中华区以外的全球范围内开发、生产和商业化的独家权利。恒瑞医药将获得2亿美元的首付款，并有资格获得不超过17.7亿美元的与特定的开发、监管和商业化相关的里程碑付款。 潜在首款！脊髓性肌萎缩症疗法获FDA优先审评资格 Scholar Rock公司今日宣布，美国FDA已受理其为在研疗法apitegromab递交的生物制品许可申请（BLA），用于治疗脊髓性肌萎缩症（SMA）患者。FDA同时授予这一申请优先审评资格，预计在今年9月22日之前完成审评。新闻稿表示，若apitegromab获批，将成为首款治疗SMA的肌肉靶向疗法。该公司同时宣布，欧洲药品管理局（EMA）也已经接受apitegromab的上市许可申请（MAA）。 Apitegromab的监管提交资料基于关键性3期SAPPHIRE试验的积极疗效与安全性数据，同时还有2期TOPAZ试验及长期延续ONYX试验的支持性数据。分析显示，SAPPHIRE试验达成了主要终点：与仅接受SMN靶向治疗（nusinersen或risdiplam）与安慰剂的患者相比，接受apitegromab联合SMN靶向治疗的SMA患者在汉默史密斯功能运动量表扩展版（HFMSE）评分中表现出统计学显著且临床意义明确的运动功能改善。 Apitegromab是一种正在研究中的全人源单克隆抗体，通过选择性结合骨骼肌中肌生成抑制蛋白（myostatin）的前体及潜在形式，抑制其激活。根据新闻稿，它是首个在3期关键性临床试验中取得临床成功的SMA肌肉靶向治疗候选药物。肌生成抑制蛋白属于TGFβ超家族生长因子，主要由骨骼肌细胞表达，其基因缺失可在包括人类在内的多种动物中导致肌肉质量与力量增加。 GSK启动带状疱疹疫苗与痴呆症风险评估研究 GSK今天宣布与英国痴呆症研究院（UK DRI）和英国健康数据研究院（HDR UK）达成一项重大新研究合作，以开展“first-of-its-kind”痴呆症研究，旨在探讨GSK重组带状疱疹疫苗（RZV）与降低痴呆症风险之间的潜在关联，从而进一步推动对神经退行性疾病的认识。若研究成果积极，该项目将为基于人口健康数据的研究模式树立典范。 多项回顾性观察性研究的数据表明，包括RZV在内的带状疱疹疫苗接种可能与痴呆症风险降低存在关联。然而，由于这些研究属于回顾性和观察性研究，结果易受未测量混杂因素影响，尚无法确定二者之间的因果关系。 本次合作将充分利用英国国家医疗服务体系（NHS）提供的高质量、去标识化的人口电子健康数据，评估RZV接种在降低痴呆症风险方面的实际效果。研究预计将纳入约140万名65至66岁人群的符合条件电子健康记录，构建一个全面且具代表性的数据集。在数据分析中，将综合考量RZV接种情况、年龄、性别及共存疾病等多重因素，以确保结果的科学性和可靠性。整个研究计划预计历时四年完成。 新锐完成近亿美元融资，开发潜在“first-in-class”眼科疗法 今日，Character Biosciences宣布完成超额认购的9300万美元B轮融资，以加速推进其针对退行性眼病的精准治疗药物的研发，首先针对年龄相关性黄斑变性（AMD）。此次融资由新投资者aMoon和Luma Group联合领投，同时获得博士伦（Bausch + Lomb）及Jefferson Life Sciences的参与，以及现有投资者Innovation Endeavors、Catalio Capital Management、S32与KdT Ventures的支持。 Character Biosciences通过数据驱动方法推动了其主打药物CTX203与CTX114的发现与研发，这两款药物分别针对视网膜细胞死亡和视力丧失的关键驱动因素。CTX114为一款潜在“best-in-class”补体抑制剂，旨在减缓晚期干性AMD中地图样萎缩的进展。CTX203则是一款潜在“first-in-class”的脂质调节剂，旨在预防AMD向晚期进展。Character Biosciences预计这两个项目将在未来一年内进入临床试验，该公司还同时积极拓展其药物研发管线至其他眼科疾病。 此次B轮融资所获得款项将主要用于支持CTX203与CTX114的1期及2期概念验证研究，并助力Character Biosciences拓展其他眼科疾病管线。今年一月，Character Biosciences宣布与博士伦达成合作，共同开发针对AMD的新型治疗药物。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] Merck Enters Exclusive License Agreement for HRS-5346, an Investigational Oral Lipoprotein(a) Inhibitor, for Cardiovascular Disease from Jiangsu Hengrui Pharmaceuticals Co., Ltd. Retrieved March 25, 2025, from https://www.merck.com/news/merck-enters-exclusive-license-agreement-for-hrs-5346-an-investigational-oral-lipoproteina-inhibitor-for-cardiovascular-disease-from-jiangsu-hengrui-pharmaceuticals-co-ltd/ [2] FDA Grants Priority Review for Biologics License Application (BLA) and EMA Accepts Marketing Authorisation Application (MAA) for Apitegromab as a Treatment for Spinal Muscular Atrophy. Retrieved March 25, 2025, from https://www.businesswire.com/news/home/20250325289782/en/FDA-Grants-Priority-Review-for-Biologics-License-Application-BLA-and-EMA-Accepts-Marketing-Authorisation-Application-MAA-for-Apitegromab-as-a-Treatment-for-Spinal-Muscular-Atrophy [3] GSK, UK Dementia Research Institute (UK DRI) and Health Data Research UK (HDR UK) to collaborate on first-of-its-kind dementia research initiative. Retrieved March 25, 2025, from https://www.gsk.com/en-gb/media/press-releases/gsk-uk-dementia-research-institute-uk-dri-and-health-data-research-uk-hdr-uk-to-collaborate-on-first-of-its-kind-dementia-research-initiative/ [4] Character Biosciences Raises $93 Million Series B to Advance Precision Medicine for Progressive Eye Diseases. Retrieved March 25, 2025, from https://www.businesswire.com/news/home/20250325445846/en/Character-Biosciences-Raises-%2493-Million-Series-B-to-Advance-Precision-Medicine-for-Progressive-Eye-Diseases 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

Character Biosciences developed its main assets using results from an ongoing observational study of patients with age-related macular degeneration. \n A New Jersey-based biotech is looking to become the main character in the age-related macular degeneration field. Character Biosciences has landed a $93 million series B to progress its precision medicine candidates through clinical development.The round was led by new investors aMoon and Luma Group, along with Bausch + Lomb, Jefferson Life ​​​​Sciences and existing investors.The proceeds will fund phase 1 and phase 2 trials for Character\'s lead AMD assets, CTX203 and CTX114, and expand its pipeline into other eye diseases, the company said in a March 25 release.“Millions of patients suffering from degenerative eye diseases lack effective treatments that delay disease progression,” Cheng Zhang, Character’s co-founder and CEO, said in the release. “This funding allows us to advance our lead programs into first-in-human trials​, with the goal of bringing​ new therapies to patients who urgently need them.”Character plans for its two protagonist assets to enter the clinic in 2026. The company also has another undisclosed early-stage program in AMD, as well as one pursuing primary open-angle glaucoma, according to its website. CTX203 is designed to be a first-in-class lipid regulator that prevents AMD from progressing to advanced disease, according to the release, while Character hopes for CTX114 to be a best-in-class complement inhibitor that slows the progression of geographic atrophy in advanced dry AMD.Both assets were developed using Character’s precision approach. Working with more than 150 ophthalmology treatment centers across the U.S., the biotech is conducting an observational AMD study that combines genetics, imaging and patient outcomes to identify subtypes of the notoriously complex disease and design targeted therapeutics for them. This approach led to the development of CTX203 and CTX114, Character said. That project hit a milestone of 5,000 patients enrolled in December 2024.“By identifying the genetic modifiers” of disease progression, Zhang said, “we can develop therapeutics to more precisely target the root causes of disease and improve clinical translation.”AMD is the most common cause of severe vision loss in patients 50 or older. Most patients have dry AMD, which occurs when a part of the retina called the macula breaks down over time. Dry AMD can then progress to the more serious wet AMD if blood vessels in the eye burst and leak fluid into the macula.Character was founded as Clover Therapeutics in 2019, announcing its rebrand to Character Biosciences (PDF) alongside an $18 million fundraising round in May 2022. The firm struck a deal in 2024 to develop AMD treatments with Bausch + Lomb, with the potential for work to expand into other eye disease, Character announced in a Jan. 10 release.Competition has been ramping up in the ophthalmology space as Regeneron’s dominant force Eylea (aflibercept) is set to lose exclusivity in the U.S. this year. The blockbuster injectable, which is approved for a suite of eye diseases including wet AMD, netted Regeneron $5.97 billion in 2024 together with its newer high-dose version Eylea HD, which doesn’t lose exclusivity until 2039.Eylea recently topped a competitor from Australian biotech Opthea in a phase 3 wet AMD trial, leaving that firm to weigh its future options.