6月25日，港股创新药企云顶新耀（1952.HK）宣布，其伊曲莫德（VELSIPITY）用于治疗中重度活动 性溃疡性结肠炎（UC）患者的新药上市许可申请（NDA）正式获得韩国食品药品安全部（MFDS）受 理。这表明该潜力新药在亚洲市场准入方面取得关键进展，其亚洲商业化布局得以进一步完善。此前， 伊曲莫德已在中国澳门、新加坡和中国香港获批，且在中国大陆的上市申请已获受理，此次韩国上市申 请获受理，使得云顶新耀距离亚洲市场全面布局更近一步。 临床数据亮眼 填补百万患者市场空白 此外，云顶新耀在亚洲地区（包括中国大陆、中国台湾和韩国）开展的伊曲莫德多中心、随机、双盲、 安慰剂对照III期研究，更是为在亚洲UC患者中的应用提供了强有力的证据支持。 这项迄今为止完成的最大规模的亚洲中重度溃疡性结肠炎的III期注册临床研究，总计纳入340名中重度 溃疡性结肠炎患者随机接受伊曲莫德或安慰剂治疗。结果显示，每日一片2mg伊曲莫德治疗，所有主要 终点和次要终点均达到显著统计学意义改善，其中52周黏膜愈合率(ES≤1且Geboes <2.0)高达52%，黏膜 完全正常化（ES=0）高达46%。。 溃疡性结肠炎被世界卫生组织列为" ...

Core Viewpoint - The article highlights the rapid growth of China's innovative pharmaceutical companies, particularly in the context of supportive government policies and the increasing commercialization of their products, exemplified by the strategic shift of CloudTop New Horizon towards self-developed mRNA technology and its potential for global expansion [1][12]. Group 1: Policy Support and Market Dynamics - Recent government policies, including the State Council's approval of the "Full Chain Support for Innovative Drug Development Implementation Plan," have significantly boosted the innovative drug sector in China [1]. - In Q1 2025, the "License-out" transaction amount for Chinese innovative drugs reached $36.929 billion, indicating a surge in outbound transactions [1]. Group 2: CloudTop New Horizon's Strategic Shift - CloudTop New Horizon is transitioning from a "License-in" model to a self-developed innovative drug company, marking a significant strategic transformation [1][12]. - The company will hold its first mRNA technology platform R&D day on June 27, showcasing its self-research capabilities and signaling a potential value release [1]. Group 3: Commercialization and Revenue Projections - CloudTop New Horizon has successfully commercialized several key products, including Nefukang® and Yijia®, with projected sales reaching 10 billion yuan by 2030 [2][3]. - Nefukang® has seen a significant increase in sales following its inclusion in the medical insurance system, with expectations for revenue to double in the second half of the year [3][4]. Group 4: Product Pipeline and Market Potential - The company anticipates that Nefukang® could achieve peak sales of 5 billion yuan, while Yijia® is expected to reach similar sales figures due to its potential in the ulcerative colitis market [4][5][6]. - CloudTop New Horizon's product matrix, including Nefukang®, Yijia®, and the pipeline product Cefepime-Tazobactam, is projected to exceed 10 billion yuan in sales by 2030 [6]. Group 5: mRNA Platform Development - The mRNA platform is gaining attention from multinational pharmaceutical companies, with potential business development (BD) collaborations on the horizon [7][12]. - The company has developed a diverse pipeline within its mRNA platform, including personalized tumor therapeutic vaccines and CAR-T therapies, which are expected to attract further investment and partnerships [8][9]. Group 6: EVER001 Clinical Data and Global Expansion - EVER001, a new generation covalent reversible BTK inhibitor, has shown promising clinical data, with plans for global development and potential overseas licensing agreements [10][12]. - The positive clinical results for EVER001 in treating primary membranous nephropathy have garnered interest from multiple multinational pharmaceutical companies, indicating a strong market potential [11][12].

2025 年 06 月 15 日 生物医药Ⅱ 新药周观点：创新药 4 月进院数据更 新，多个新纳入医保创新药快速进院中 本周新药行情回顾： 2025 年 6 月 9 日-2025 年 6 月 13 日，新药板块涨幅前 5 企业：北海 康成-B（118.12%），欧康维视生物-B（28.59%），君圣泰医药-B （23.75%），药明巨诺-B（23.63%），歌礼制药-B（23.40%）；跌幅 前 5 企业：三生国健（-7.41%），诺思兰德（-7.14%），宜明昂科- B（-6.10%），华领医药-B（-4.38%），永泰生物-B（-3.48%）。 本周新药行业重点分析： 近日国家医保局更新了已纳入医保目录创新药截止 2025 年 4 月底的 进院数据，多个 2025 年新纳入医保国产创新药产品正在快速进院中， 部分药物进院速度较快，其中： 从增长速度来看，对比 25 年 3 月底，截止 25 年 4 月底入院数据增长 较快的药物主要有云顶新耀布地奈德肠溶胶囊、康方生物卡度尼利单 抗与依沃西单抗等。 从入院总体数量来看，截止 25 年 4 月底入院数据超 200 家的药物主 要有云顶新耀布地奈德肠溶胶囊、百济 ...

据介绍，经此次新增调整，广西单列门诊统筹支付药品总数达147种，其中有44种适用于门诊治疗 的罕见病药品，惠及多发性硬化、低磷性佝偻病等34种罕见病患者群体。截至2025年5月底，原72种国 家谈判药品纳入单列门诊统筹支付药品目录已有17.43万人次享受单列门诊统筹待遇，医保基金报销3.14 亿元，平均报销比例达65.30%。（罗琦 莫覃超） "该政策不仅在减轻患者就医负担方面发挥着重要作用，也明显减轻了患者因住院就医而产生的综 合成本。"自治区医保局副局长赖永东表示，部分国家谈判创新药纳入单列门诊统筹支付后，符合门诊 治疗条件的患者可凭临床医生处方直接在门诊就医取药或到药店购药，减轻了用药负担的同时也极大提 升了就医便捷度。 按照规定，参保人员在定点医疗机构门诊发生的单列门诊统筹支付的医保药品费用，不设起付线， 由医疗保险统筹基金和参保人员共同负担。其中，居民医保的统筹基金支付比例为50%；职工医保的统 筹基金支付比例为在职人员70%、退休人员75%。此外，设置有年度最高支付限额，其中居民医保为4 万元，职工医保为8万元，分别计入当地居民医保和职工医保年度最高支付限额。 以布地奈德肠溶胶囊的使用为例，广西壮 ...

在近日举行的第62届欧洲肾脏协会大会（ERA 2025）上，全球首个对因治疗药物耐赋康®（布地奈德 肠溶胶囊）陆续展示9项最新研究成果，其中最新两项研究证实，耐赋康®显著降低致病因子水平，对 于国际公认的IgA肾病发病机制"四重打击"学说中的第一、第三和第四重打击均有积极调节作用，并发 现Gd-IgA1或多聚IgA最初两个月内的变化与降蛋白尿效果具有相关性，为"对因治疗"策略提供了坚实 支持。其从源头干预发病机制，延缓疾病进展并保护肾功能的临床价值得到进一步验证，为国内外IgA 肾病治疗理念的变革和指南的更新奠定了证据基础。 作为全球首个也是目前唯一在中国、美国和欧洲获得完全批准，且不受蛋白尿水平限制的IgA肾病对因 治疗药物，耐赋康®先后获得了《2024版KDIGO IgA肾病和IgA血管炎临床管理实践指南（公开审查 版）》以及2025版《中国成人IgA肾病及IgA血管炎临床实践指南（预审版本）》国内外指南推荐。 指南预审版还强调了"对因治疗、尽早治疗、全部治疗"的新管理策略。耐赋康®此次在大会上公布的多 项研究结果，为这一新管理策略提供了坚实的科学依据和临床支持，助推IgA肾病治疗水平不断提升。 IgA肾 ...

Core Insights - The recent ERA 2025 conference showcased nine new studies on the drug Nefukang® (Budesonide enteric-coated capsules), confirming its renal protective value for patients with varying baseline eGFR levels and different pathological changes [1][2][3] - Nefukang® is the first and only approved drug for the treatment of IgA nephropathy in China, the US, and Europe, without restrictions based on proteinuria levels, establishing its leadership in first-line treatment [1][4] - The studies support a "total treatment" strategy, emphasizing the importance of early intervention with Nefukang® to protect renal function and improve overall prognosis for all diagnosed patients [1][3] Study Findings - A sub-analysis of the NefIgArd study indicated that treatment with Budesonide enteric-coated capsules for nine months led to reduced proteinuria and stable renal function (eGFR) across all baseline eGFR levels during a two-year study period [2] - Another multi-center study showed significant reductions in proteinuria and stable eGFR in patients with active lesions, particularly those with endothelial cell proliferation and/or crescent formation [2][3] - The findings provide strong evidence for the application of Budesonide enteric-coated capsules in treating IgA nephropathy, offering new treatment options for clinicians [2][3] Clinical Implications - Clinicians are encouraged to initiate cause-specific treatment immediately upon diagnosis to maximize renal function protection and improve patient outcomes [3] - The CEO of Yunding Xinyao highlighted that these studies further validate Nefukang®'s benefits across different renal function levels and pathological types, supporting a new disease management strategy for IgA nephropathy [3] - The prevalence of IgA nephropathy is significantly higher in Asia, with an estimated 5 million patients in China and over 100,000 new cases annually, indicating a substantial unmet clinical need [3] Additional Research - Besides the two key studies, seven other studies presented at the conference covered efficacy prediction markers, treatment sustainability, and safety mechanisms, broadening the applicability of Nefukang® in clinical settings [4] - Nefukang® was included in the national medical insurance drug list as of November 2024, with expectations to leverage its evidence-based advantages to enhance treatment initiation upon diagnosis [4]

Core Insights - The latest research confirms that Nefecon® significantly reduces kidney function deterioration by 50% through its positive effects on the first three hits in the "four-hit" model of IgA nephropathy [1][3][4]. Group 1: Efficacy and Mechanism - Nefecon® has been shown to significantly lower levels of Gd-IgA1, IgG anti-IgA antibodies, and IgA immune complexes (IgA-IC), indicating its effectiveness across multiple pathogenic pathways [1][3][4]. - Changes in Gd-IgA1 or polymeric IgA within the first two months of treatment are significantly correlated with reductions in proteinuria, suggesting these biomarkers could guide Nefecon® therapy [1][3][7]. - The NefIgArd trial involved 216 participants and demonstrated that Nefecon® effectively targets the gut to modulate immune responses, thereby reducing pathogenic IgA levels [4][5]. Group 2: Clinical Significance - Nefecon® is the first and only drug approved for the causal treatment of IgA nephropathy in China, the U.S., and Europe, and is recommended in multiple clinical guidelines, solidifying its position as a cornerstone therapy [2][4]. - The drug's ability to intervene at the source of IgA nephropathy's pathogenesis is expected to reshape treatment strategies and provide more options for a broader patient population [4]. Group 3: Patient Impact - In China, there are over 5 million patients with IgA nephropathy, with more than 100,000 new cases diagnosed annually, highlighting the significant healthcare burden and the need for effective treatments like Nefecon® [3].

第二项多中心研究聚焦分析伴不同病理表现的IgA肾病患者的治疗获益。结果显示，给予9个月的耐赋 康治疗后患者尿蛋白显著降低，肾功能（eGFR）保持稳定，且在伴有毛细血管内皮细胞增生（E1）和/ 或新月体形成（C1）的患者中，蛋白尿下降更显著。这一发现提示，这两种活动性病变可以积极使用 耐赋康治疗，且无论有无活动性病变，患者均可从治疗中维持eGFR稳定，保护肾功能。 在第62届欧洲肾脏协会（ERA 2025）大会上，耐赋康（布地奈德肠溶胶囊，NEFECON®）展示的两项 最新研究结果证实了耐赋康在不同基线肾功能水平患者中均取得卓越疗效，并揭示了其在全部不同病理 人群中的显著获益。作为全球首个且唯一在中国、美国和欧洲获得完全批准，且不受蛋白尿水平限制的 IgA肾病对因治疗药物，此次研究成果为"全部治疗"策略提供了坚实支持，证实耐赋康实现了IgA肾病全 人群肾脏保护。 耐赋康凭借创新的作用机制和临床优势，先后被纳入改善全球肾脏病预后组织（KDIGO）发布的 《2024版KDIGO IgA肾病和IgA血管炎临床管理实践指南（公开审查版）》，以及《中国成人IgA肾病 及IgA血管炎临床实践指南（预审版本）》。 两项研究证 ...

智通财经APP获悉，在第62届欧洲肾脏协会(ERA 2025)大会上，耐赋康(布地奈德肠溶胶囊，NEFECON)展示了9项最新研究成果，包括8项口头报告和1项电 子壁报。其中2项研究证实了耐赋康在"尽早治疗"IgA肾病方面可实现具有更显著临床获益，为推动IgA肾病诊疗向"对因治疗、尽早治疗、全部治疗"的新管 理策略提供有力证据支持。ERA 2025大会于6月4日至7日在奥地利维也纳召开。 此次在ERA 2025大会上公布的耐赋康最新研究结果，为IgA肾病的治疗时机与疗效关系提供了有力的循证依据。在一项布地奈德肠溶胶囊治疗IgA肾病患者 的肾脏获益与确诊时间的NefIgArd研究子分析中，该研究旨在评估确诊时间长短不同的IgA肾病患者接受布地奈德肠溶胶囊16 mg/d治疗的效果。研究纳入参 与NefIgArd研究的364例原发性IgA肾病患者，根据基线时IgA肾病确诊(通过肾活检确诊)后时间长短，将患者分为四个四分位数，观察估算的肾小球滤过率 (eGFR)、尿蛋白肌酐比(UPCR)随时间的变化。 结果显示，无论诊断时间长短，布地奈德肠溶胶囊相较于安慰剂均能显著保护肾功能(eGFR)并降低UPCR，且在近期确诊的 ...

Core Viewpoint - The release of the "Clinical Practice Guidelines for Adult IgA Nephropathy and IgA Vasculitis (Preliminary Version)" marks a significant advancement in the treatment of IgA nephropathy, with the inclusion of NEFECON as a recommended therapy for patients at risk of disease progression [1][2][3] Group 1: Guidelines and Recommendations - The guidelines emphasize the need for etiology-based treatment, early intervention, and comprehensive management strategies for IgA nephropathy patients in China [2] - For patients with proteinuria ≥0.5g/d, kidney biopsy and treatment initiation are recommended, aiming for proteinuria <0.5g/d and an estimated glomerular filtration rate (eGFR) decline of less than 1ml/min per year [2] - The guidelines introduce blood urine monitoring and suggest interventions targeting immune damage, including the use of NEFECON to reduce pathogenic IgA levels [2] Group 2: NEFECON's Clinical Significance - NEFECON is recognized as the only etiology-based treatment for IgA nephropathy, establishing its position as a first-line therapy in global kidney disease guidelines [3][4] - The NefIgArd study demonstrated that NEFECON significantly reduces the risk of kidney function decline, with a 50% reduction in eGFR decline compared to placebo, and up to 66% in the Chinese population [4] - NEFECON's approval by the National Medical Products Administration (NMPA) and its inclusion in the national medical insurance drug list further solidify its role in treating IgA nephropathy [4][5]