A Phase IIIb, Multicenter, Single-arm Study Assessing the Effectiveness, Safety and Patient Reported Outcomes of a 36-week Refill Exchange Regimen for the Port Delivery System With Ranibizumab in Patients With Neovascular Age-related Macular Degeneration

The purpose of this study is to evaluate the effectiveness, safety, and PROs of the port delivery system with ranibizumab 100 milligrams/milliliters (mg/mL) refilled every 36 weeks (Q36W) in participants with nAMD.

开始日期2025-11-18

申办/合作机构

Hoffmann-La Roche Ltd.

[+1]

NCT07007065

/ Not yet recruiting临床3期

A Randomized, Controlled, Partially Masked, Phase 3b Study to Assess the Injection Burden, Efficacy, Safety, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec (ABBV-RGX-314) in a Real-World Context in Subjects With Neovascular Age-Related Macular Degeneration (nAMD)

Neovascular age-related macular degeneration (nAMD), also known as "wet" AMD, is the abnormal growth of new blood vessels in the light-sensitive tissue at the back of the eye called the retina. The purpose of this study is to assess how safe and effective Surabgene Lomparvovec is in treating participants with Neovascular age-related macular degeneration (nAMD).
Surabgene Lomparvovec (ABBV-RGX-314) is an investigational gene therapy being developed for the treatment of neovascular age-related macular degeneration (nAMD). Participants will be placed into 1 of 3 groups, called treatment arms. Each group receives different treatment. Adult participants aged 50 and older years with a diagnosis of previously treated nAMD will be enrolled. Around 561 participants will be enrolled in the study at approximately 150 sites worldwide.
Participants in groups 1 and 2 will receive a single subretinal dose of ABBV-RGX-314. Participants in group 3 will receive Ranibizumab as needed throughout the study. Ranibizumab will be given as an intravitreal injection (injection into the jelly-like tissue that fills the eyeball injection), and ABBV-RGX-314 will be given as a subretinal (between the retina and the back of the eye) injection. The Assessment Period begins after randomization (1:1:1) to one of the ABBV-RGX-314 treatment groups or control at Week -2 and lasts up to 5 years.
There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular monthly visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

开始日期2025-09-17

申办/合作机构

AbbVie, Inc.

ChiCTR2500095798

/ Suspended早期临床1期IIT

Clinical study of regulatory T cell exosomes combined with VEGF antibody (Ranibizumab) in the treatment of refractory exudative age-related macular degeneration

开始日期2025-01-16

申办/合作机构

河北省人民医院

100 项与雷珠单抗相关的临床结果

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100 项与雷珠单抗相关的转化医学

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100 项与雷珠单抗相关的专利（医药）

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5,391

项与雷珠单抗相关的文献（医药）

2025-12-31·JOURNAL OF MEDICAL ECONOMICS

Cost-effectiveness analysis of bispecific antibody faricimab for treatment of neovascular age-related macular degeneration and diabetic macular edema in Japan

Article

作者: Igarashi, Ataru ; Sakashita, Naotaka ; Shoji, Ayako ; Higashi, Kentaro ; Tsujimura, Jun ; Ohno, Shinya ; Yanagi, Yasuo

OBJECTIVE:

To assess the cost-effectiveness of faricimab vs. other anti-vascular endothelial growth factor (anti-VEGF) drugs for treatment of neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME) in Japan, while considering societal burden associated with treatment.

METHODS:

A Markov model for cost-effectiveness analysis of anti-VEGF treatment in patients with nAMD and DME was applied based on cost and utility value data from Japan. Faricimab administered through a treat-and-extend (T&E) regimen was compared with ranibizumab administered pro re nata (PRN) and T&E, aflibercept T&E, brolucizumab T&E, and best supportive care (BSC). Further to treatment costs (public payer perspective), the societal burden (societal perspective), including costs of travel, informal care, and productivity, was assessed.

RESULTS:

In treatment of nAMD, lifetime quality-adjusted life years (QALYs) gained were highest with faricimab (faricimab T&E: 6.92, ranibizumab PRN: 6.88, ranibizumab T&E: 6.91, aflibercept T&E: 6.89, brolucizumab T&E: 6.89, BSC: 5.99). From the public payer perspective, the lifetime total cost for faricimab T&E was lower than those for ranibizumab (PRN, T&E) and brolucizumab (T&E), comparable to aflibercept T&E, and higher than BSC (incremental costs: 158,385 and 6,475,511 JPY, respectively). As a result, faricimab was cost-effective or dominant in the treatment of nAMD, excluding BSC. From the societal perspective, faricimab was dominant against all comparators in nAMD. In treatment of DME, QALYs gained were highest with faricimab (faricimab T&E: 8.51, ranibizumab PRN: 8.17, aflibercept PRN: 8.36, ranibizumab T&E: 8.13, BSC: 5.16). From both the public payer and societal perspectives, faricimab was dominant against all comparators in DME.

CONCLUSIONS:

When societal burdens were considered, faricimab was dominant in both nAMD and DME against all comparators, suggesting that the extended dosing interval associated with faricimab treatment may alleviate societal burdens and consequently improve patient outcomes.

2025-09-01·JOURNAL FRANCAIS D OPHTALMOLOGIE

Disease control, visual and anatomic outcomes at week 48 of brolucizumab treatment in patients with previously suboptimal anatomically controlled neovascular age-related macular degeneration – The SWIFT study

Article

作者: Delyfer, M-N ; Cohen, S Y ; Devin, F ; Baillif, S ; Ponthieux, A ; Kodjikian, L ; Souied, E H ; Aubry-Quenet, I ; Creuzot-Garcher, C ; Couturier, A ; Weber, M

PURPOSE:

Neovascular age-related macular degeneration (nAMD) patients refractory to one anti-vascular endothelial growth factor (VEGF) agent often switch to another anti-VEGF for better disease control. Our objective is to assess nAMD control with brolucizumab 6mg treatment in previously treated patients with suboptimal anatomical control.

METHODS:

SWIFT was a 48-week, prospective, single-arm, open-label clinical trial at 52 sites in France (NCT04264819). Participants were adults (≥ 50 years) with active CNV lesions secondary to nAMD, diagnosed<18 months, previously treated with either ranibizumab or aflibercept with a 4- or 8-week interval treatment, with baseline BCVA 38-83 letters. Eyes were treated with intravitreal brolucizumab 6mg at weeks 0, 4 and 8 (loading phase), followed by treat-to-control dosing with intervals up to 16 weeks, depending on investigator-assessed disease activity. The main outcome is the proportion of eyes without investigator-assessed disease activity at week 16.

RESULTS:

Two hundred and eighty-nine patients were included and analyzed (mean age 76.3 years, 61.9% female). Eyes were previously treated for a mean of 9.1 months. Baseline mean BCVA was 68.9 letters, and central subfield foveal thickness (CSFT) was 417μm. Over the course of the study, patients received a median of 6 brolucizumab injections (range: 1-8) with a mean (SD) treatment duration of 32.1 (14.1) weeks. The mean last brolucizumab treatment interval (SD) was 8.7 (3.5) weeks, notably longer than the interval of 6.2 (2.8) weeks with the previous anti-VEGF agent before inclusion in SWIFT. The proportion of eyes with no disease activity was 41.0% (95% CI: 34.4-47.9) at week 16 and 52.0% at week 48 (47.0 and 43.4%, respectively, using last observation carried forward sensitivity analysis). Improvements were observed from baseline to week 48 in BCVA (mean +3.2 [9.2] letters; P<0.0001), CFST (-66 [101] μm; P<0.0001). At week 48, 22% of eyes were on a regimen of every 12 weeks or more, and 40.4% of eyes had no retinal fluid (36.0 and 42.4% had intraretinal and subretinal fluid respectively). Intraocular inflammation (IOI) was confirmed in 29/295 (9.8%) eyes. Four of these patients had BCVA losses of≥15 letters at or after IOI resolution.

CONCLUSIONS:

In patients with refractory nAMD, brolucizumab allowed control of disease activity for up to 41.0% of patients, with visuals gains, anatomical improvements and extended treatment interval, despite poor response and discontinuation for some patients in addition to a 9.8% rate of IOI.

2025-07-01·RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES

CAN CHANGES ON OPTICAL COHERENCE TOMOGRAPHY ANGIOGRAPHY PREDICT EXUDATIVE ACTIVITY ON OPTICAL COHERENCE TOMOGRAPHY IN EYES WITH NEOVASCULAR AGE-RELATED MACULAR DEGENERATION?

Article

作者: Baillif, Stéphanie ; Weber, Michel ; Devin, François ; Blanco-Garavito, Rocio ; Miere, Alexandra ; Souied, Eric H. ; Colantuono, Donato ; Amoroso, Francesca

Purpose::

To explore the predictability of changes in neovascularization morphology on optical coherence tomography angiography (OCTA) in relation to exudative activity on structural OCT in eyes with neovascular age-related macular degeneration treated with ranibizumab 0.5 mg.

Methods::

Eighty-five eyes of 85 patients with neovascular age-related macular degeneration, diagnosed less than 1 year before study entry and treated with ranibizumab 0.5 mg, were included in this longitudinal, prospective study. Included eyes had no exudative activity feature at study inclusion and were treated with ranibizumab 0.5 mg in a PRN regimen for the following 9 months. At each study visit, structural OCT and OCTA were performed. Qualitative and quantitative vascular characteristics of macular neovascularization were extracted at each visit and correlated with the presence of exudative activity on OCT.

Results::

No significant correlation was found between OCTA (neo)vascular characteristics in the absence of exudation with the presence of exudative activity on structural OCT the following month. Optical coherence tomography angiography's positive predictive value for the overall population was 38.87% (confidence interval [33.8%–44.2%]). The negative predictive value of OCTA on the overall population was 60.92%. Interobserver reproducibility of OCTA assessments Cohen's kappa = 0.82.

Conclusion::

Although OCTA may not be predictive of imminent exudative activity in neovascular age-related macular degeneration, it brings additional information on macular neovascularization flow and holds potential as a tool for understanding vascular remodeling. Neovascularization remodeling and exudation may be two separate findings in the disease process in neovascular age-related macular degeneration without necessarily having a causal relationship.

577

项与雷珠单抗相关的新闻（医药）

2025-07-05

·动脉网

错过7.55亿美元BD大单，这家肾科Biotech逆势IPO

2024年10月，Maze Therapeutics（以下简称“Maze”）在美国肾脏病学会（ASN）年会上公布了其核心产品MZE829的I期临床结果，实验数据显示血浆暴露量（AUC）与剂量线性相关。这款针对APOL1介导的口服小分子抑制剂支持每日一次给药，目前已启动Ⅱ期临床试验。2024年底，Maze官宣完成1.15亿美元的D轮融资，随后又在2025年1月完成IPO，获得1.4亿美元。Maze凭借Maze Compass平台将人类遗传学发现的“天然保护机制”转化为具有高度靶向性的口服小分子药物管线。这种“向人体自身寻求解药”的策略，显著提高了靶点验证效率和开发成功率，直击肾病领域巨大的未满足需求。01精准靶向遗传机制，针对“无药可治”的APOL1双突变肾病Maze聚焦APOL1介导肾病与慢性肾病（CKD）的药物开发。CDC（Centers for Disease Control and Prevention：疾病控制与预防中心）官网显示，仅美国就有七分之一的人患有CKD。据Maze官网，在美国大约600万非裔美国人携带高风险APOL1基因变异的双等位基因突变，并面临患AKD（APOL1介导的肾病）的风险。目前，针对肾脏疾病的治疗较为棘手。传统治疗以“延缓进展”为主，缺乏根治手段，且存在疗法单一、作用有限等问题。首先是疗法单一。对原发性肾小球疾病、遗传性肾病等缺乏根治性药物。现有疗法（如激素、免疫抑制剂）仅能控制症状，无法逆转肾纤维化进程，糖尿病肾病等继发性肾病需长期管理血糖、血压，但多数患者仍会进展至肾功能不全。其次是疗效局限。近年虽有SGLT2抑制剂、GLP-1受体激动剂等被证实可延缓肾病进展，但仅适用于特定人群（如合并糖尿病者），且无法阻止所有患者进入透析。针对APOL1双突变患者，当前无获批疗法，标准治疗仅延缓进展（如RAS抑制剂），疗效有限。Maze的发展实现了传统药物开发依赖“试错”的范式创新。传统药物研发的核心痛点是靶点验证不足，大部分失败正源于此。Maze的创新在于用人类遗传学数据前置验证靶点——相当于利用自然界完成的“临床试验”。目前，Maze的核心产品是针对急性肾脏病（AKD）的MZE829与针对慢性肾病（CKD）及苯丙酮尿症（PKU）的MZE782。● MZE829：模拟保护性变体解决APOL1基因突变带来的AKD风险APOL1变异最初因抵抗非洲锥虫病（睡眠病）被自然选择保留。然而，携带APOL1基因的群体存在高危变异（G1/G2）风险。一般说来，携带两个APOL1等位基因，且这两个基因均为G1/G1、G2/G2或G1/G2的人群被界定为高危变异群体。这一高危变异最终会导致肾小球损伤，肾脏过滤功能受阻等问题。这一情况在西非血统人群中富集（非裔美国人中约13%携带双拷贝突变），约20%携带者（超100万人）最终会发展为AKD。MZE829的优势在于可直接抑制APOL1毒性蛋白活性，精准靶向遗传机制，安全性更优。具体来看，APOL1高危突变（G1/G2）表达的蛋白会在肾细胞膜上形成孔道，导致细胞裂解。MZE829可以通过模拟保护性变体（G3）的功能，将APOL1锁定在闭合状态，阻止孔道形成。Ⅰ期数据显示，480 mg单剂量给药后，血浆暴露量（AUC）与剂量线性相关，无严重不良反应。目前，Maze已在2025年第一季度完成Ⅱ期首例患者给药，预计在明年第一季度公布顶线数据。● MZE782：慢性肾病及苯丙酮尿症多适应症疗法通过Maze Compass平台的遗传学分析，Maze发现保护性基因溶质转运蛋白SLC6A19（中性氨基酸转运体，SLC6A19基因突变导致中性氨基酸尿症）的功能缺失变异与肾功能改善显著相关。MZE782 是一款口服SLC6A19抑制剂，具有双重适应症疗效。针对慢性肾病（CKD），它通过抑制肾小管中的SLC6A19蛋白减少氨基酸的重吸收，从而减轻肾脏的代谢负担，达到改善肾功能的目的。对于苯丙酮尿症（PKU），该药物则发挥阻断肠道对苯丙氨酸吸收的作用，有效降低患者血液中苯丙氨酸水平。MZE782的优势在于能覆盖更广泛的CKD人群及PKU患者，市场潜力可观，尤其体现在口服优势和潜在联合用药能力上。2024年9月，MZE782已启动Ⅰ期试验，将对其安全性、药代动力学及生物标志物（如PBMC糖原）进行全面评估，预计2025年下半年公布初步数据。02Maze Compass平台：从基因变异中寻找天然抗体Maze目前的几款核心产品都是通过自研专有平台Maze Compass实现突破性发现的。Maze Compass是一套将人类遗传学数据转化为精准药物的系统性工具，围绕“保护性基因变异”展开，通过破解疾病机制并设计药物。遗传学家Mark Daly曾发现一个矛盾现象：某些非裔美国人携带APOL1基因的高危突变（G1/G2），却未发展为肾病。进一步分析显示，这群人携带一种罕见的基因变异，能抑制APOL1蛋白的毒性功能。这一发现，成为Maze Compass技术平台的起点。Maze Compass是覆盖药物开发每个阶段的技术平台。该平台可理解和整合变体功能化的关键步骤，其核心是“解码-模拟-验证”的三步循环：首先是从海量数据中“淘金”。Maze Compass整合了芬兰FINNIGEN、英国生物样本库等超大规模人群数据库，筛选与疾病风险降低相关的基因变异。例如，某些SLC6A19基因的功能缺失突变携带者，肾脏功能异常风险显著降低。在寻找到合适的基因变异后，在实验室重现这些变异的保护机制。MZE829的设计正源于此。它通过稳定APOL1蛋白的闭合构象，阻止其形成破坏细胞膜的孔道。最后是进行“基因拟态”：通过开发化合物模拟变体的功能，最终设计小分子药物以模拟保护性变体的生物学效应。图源：公司官网Maze Compass的核心优势在于靶点验证率高、精准患者分层并实现了口服给药突破。在靶点验证方面，由于平台起点是人体内天然存在的有效“实验证据”，大幅降低了传统药物开发中因靶点错误导致的失败风险。然后是患者分层，平台选择天然携带相关基因变异或处于类似病理状态的患者，更可能对药物产生响应，提高临床试验的针对性和成功率。最后，MZE829和MZE001均为口服制剂，患者依从性显著优于需频繁住院的酶替代疗法（如庞贝病现有疗法）。031.4亿美元IPO，曾是赛诺菲的BD标的2025年1月31日，Maze 宣布完成IPO融资1.4亿美元，至此公司的总融资额达6.36亿美元。这次融资体现了资本对其创新技术与研发模式的认可，也凸显了公司战略合作的一波三折。2023年5月，赛诺菲曾以预付1.5亿美元+最高6亿里程碑付款的协议锁定其庞贝病药物MZE001，意图巩固自身在庞贝病市场的垄断地位。然而美国联邦贸易委员会（FTC）以反垄断为由强势介入，指控该交易恶意垄断，维持救命药高价现状，最终迫使赛诺菲于同年12月终止合作。戏剧性的是，仅五个月后，日本盐野义制药以1.5亿美元预付款接手MZE001项目，其CEO吉田达守直言该项目“与盐野义战略高度契合”，并强调其有望成为全球首款庞贝病口服药。罕见病领域虽靶点明确、临床开发周期短，但患者基数有限、支付环境苛刻，且易被巨头垄断。正如赛诺菲对庞贝病市场的绝对控制，由此带来的反垄断交易终止，Maze与盐野义战略合作不失为一个好选择。资本的纷至沓来见证了Maze的成长潜力。尽管公司尚未盈利，2024年Q4每股亏损18.32美元，但凭借其与盐野义的战略合作以及APOL1肾病领域的庞大市场空间，Maze的商业价值依然不可小觑。若MZE829在2025年推进的Ⅱ期临床试验中数据持续亮眼，公司有望实现传统试错式研发的范式创新。Maze历史投融资情况（来源：Crunchbase）多轮大额融资和BD源于Maze兼备学术洞察与产业转化能力的团队。 Jason Coloma博士是Maze的首席执行官，在被任命为首席执行官之前，他是Maze的首席运营官和Third Rock Ventures的风险合作伙伴。Bernstein博士是Maze的首席医疗官，他在基础科学研究、转化医学和临床开发方面拥有超过三十年的经验，主要负责基因新药物的战略性开发。Atul Dandekar现任Maze首席战略和业务官，全面负责公司战略规划、合作拓展、企业发展和投资组合管理。在加入Maze之前，他曾担任罗氏眼科全球特许经营主管，领导全球眼科业务战略制定，并主导开发了faricimab、雷珠单抗PDS以及行业领先的视网膜疾病管线。04临床数据有待检验，APOL1治疗竞争激烈目前，对于Maze来说还存在诸多尚待跨越的难题：在临床表现上，MZE829的Ⅱ期试验将首次在真实患者中验证疗效。APOL1肾病的异质性极高——约80%携带高危突变者终身未发病，如何筛选出20%的进展性患者成为关键。市场竞争方面，诺华、Vertex等巨头均在布局APOL1突变适应症药物治疗，若抢先获批可能会挤压Maze的市场空间。总的来说，巨头在APOL1领域的竞争已进入临床验证白热化阶段：Vertex凭借先发机制和突破性认定暂时领先，Maze则以精准分层和广谱适应症布局寻求差异化优势。后续核心关注2025年VertexⅢ期数据与2026年MazeⅡ期结果的直接对比，这将影响百亿美元AMKD市场的分配格局。如果您想对接文章中提到的项目，或您的项目想被动脉网报道，或者发布融资新闻，请与我们联系；也可加入动脉网行业社群，结交更多志同道合的好友。近期推荐声明：动脉网所刊载内容之知识产权为动脉网及相关权利人专属所有或持有。未经许可，禁止进行转载、摘编、复制及建立镜像等任何使用。文中如果涉及企业信息和数据，均由受访者向分析师提供并确认。动脉网，未来医疗服务平台

IPO临床2期临床1期免疫疗法

2025-07-01

·GlobeNewswire-Health Care

Bio Usawa Biotechnology and Bioeq AG Partner to Create Widespread Access to Vision-Saving Medicine Across Sub-Saharan Africa

Landmark partnership will democratize access to critical eye treatment for millions facing preventable blindness from diabetes complicationsKIGALI, Rwanda and SAN FRANCISCO and ZUG, Switzerland , July 01, 2025 (GLOBE NEWSWIRE) -- Bio Usawa Biotechnology Ltd. (Bio Usawa) and Bioeq AG today announced a groundbreaking agreement that will bring a life-changing biosimilar medicine—ranibizumab, used to treat serious retinal diseases—within reach for millions across Sub-Saharan Africa. This partnership grants Bio Usawa exclusive rights to register and commercialize Bioeq’s ranibizumab biosimilar under the brand name BioUcenta™, marking a critical step forward in democratizing access to essential biologic therapies across the region. Ranibizumab, a biosimilar to Lucentis®, is a monoclonal antibody used to treat diabetic macular edema (DME), wet age-related macular degeneration (AMD), and other blinding retinal diseases. While this breakthrough therapy has been transforming lives in higher-income countries for nearly 20 years, patients across Africa have been denied access due to prohibitive costs and limited availability. This partnership aims to end that disparity, bringing hope to millions who face preventable vision loss. “By making ranibizumab accessible in Sub-Saharan Africa, we are empowering doctors to save the vision of millions, particularly those suffering complications from diabetes—a disease that is quietly but rapidly becoming one of Africa’s greatest health threats,” said Dr. Menghis Bairu, Co-founder, President and CEO of Bio Usawa. "Our partnership with Bio Usawa is core to our commitment to expanding global access to essential medicines," said Martin Huber, Managing Director of Bioeq AG. "Together, we are breaking down the barriers that have denied effective treatment for serious retinal diseases to patients across Sub-Saharan Africa for too long. Bringing a high-quality, affordable ranibizumab biosimilar to this region is a significant step towards health justice." Africa is at the front line of a growing diabetes crisis. According to the International Diabetes Federation, more than 24 million Africans were living with diabetes in 2024, with that number projected to double by 2050. Diabetic retinopathy, a leading complication, threatens the sight of up to one-third of these patients. Without timely intervention, many will face blindness—an outcome that is both preventable and tragic. BioUcenta™, administered via intravitreal injection, works by blocking abnormal blood vessel growth in the retina and preventing fluid leakage—two key causes of vision loss. The biosimilar has already been approved in both Europe and the United States and will now be registered and distributed across Sub-Saharan Africa by Bio Usawa. “This collaboration is about equity,” added Dr. Bairu. “Access to advanced biologic therapies should not be a privilege of geography or income. We are proud to work with Bioeq to make this a reality for Africa—ensuring that patients, regardless of where they live, can receive the care they deserve.” The agreement marks the initial fulfillment of Bio Usawa's mission to democratize access to high-quality, affordable, and proven biotherapies in low-middle income countries. By establishing local capabilities and partnerships, the initiative creates a foundation for broader access to essential medicines across the continent. The partnership also represents a new model for global health equity, demonstrating how innovative collaborations can bridge the gap between life-saving medical advances and the patients who need them most. Rather than accepting that cutting-edge treatments remain exclusive to wealthy nations, this agreement shows how strategic partnerships can extend medical breakthroughs to underserved populations. About Bio Usawa Bio Usawa is an African biotechnology company with a mission to expand access to affordable, high-quality biosimilars across the continent. By combining deep industry experience with regional partnerships, Bio Usawa is building a future where life-saving biomedicines are made in Africa, by Africans, for Africans—and beyond. For more information, visit www.biousawa.com. About Bioeq AG Bioeq AG is a Swiss biopharmaceutical joint venture between Polpharma Biologics Group and Formycon AG. Bioeq develops and licenses biosimilars for global markets, combining innovation and quality to improve patient access to vital therapies. Learn more at www.bioeq.ch. Media Contact:Daniel LevineLevine Media Group+1 510-280-5405danny@levinemediagroup.com

上市批准

2025-06-28

·CPHI制药在线

荣昌生物泰它西普授权出海，总交易额超40亿美元；信达生物玛仕度肽国内获批上市 | 制药在线一周药闻复盘

点击蓝字关注我们本周，热点不少。首先是审评审批方面，非常值得关注的有两个事情，一个是信达生物玛仕度肽国内获批上市，成为全球首个且唯一获批的GCG/GLP-1双受体激动减重药物。另一个是，科济药业舒瑞基奥仑赛注射液申报上市，成为球首款且唯一一款提交NDA的用于治疗实体瘤的CAR-T细胞产品；其次是研发方面，值得一说的就是，百奥泰VEGF单抗Ⅲ期临床成功；再次是交易及投融资方面，荣昌生物泰它西普授权出海，总交易额超40亿美元；最后是上市方面，药捷安康成功登陆港交所。本周盘点包括审评审批、研发、交易及投融资以及上市四大板块，统计时间为2025.6.23-6.27，包含28条信息。审评审批NMPA上市批准1、6月27日，NMPA官网显示，信达生物申报的胰高血糖素（GCG）/胰高血糖素样肽-1（GLP-1）双受体激动剂玛仕度肽注射液获批上市，用于在控制饮食和增加体力活动基础上对成人患者的长期体重控制，初始体重指数（BMI）为：BMI≥28kg/㎡（肥胖）；或BMI≥24kg/㎡（超重），并伴有至少一种体重相关的合并症（例如高血糖、高血压、血脂异常、脂肪肝、阻塞性睡眠呼吸暂停综合征等）。申请2、6月25日，CDE官网显示，科济药业的舒瑞基奥仑赛注射液（CT041）申报上市，用于治疗Claudin18.2表达阳性、至少二线治疗失败的晚期胃/食管胃结合部腺癌（G/GEJA）患者。舒瑞基奥仑赛注射液是全球首款且唯一一款提交NDA的用于治疗实体瘤的CAR-T细胞产品。临床批准3、6月23日，CDE官网显示，汇宇制药全资子公司汇宇海玥1类新药注射用HY0001a获批临床，拟用于晚期实体瘤患者。本次是该药首次获批临床。HY0001a是汇宇海玥自主研发的一种CDCP1 ADC，目前国内外尚无同靶点产品获批上市。该药为全球首个披露针对该靶点推向临床的药物。4、6月24日，CDE官网显示，丽珠医药1类新药YJH-012注射液获批临床，拟开发治疗痛风。这是一款基于小干扰RNA（siRNA）技术开发的创新药，有望为患者提供疗效更好、安全性更高的长效痛风治疗方案。5、6月25日，CDE官网显示，阿斯利康两款1类新药AZD6234注射液以及AZD9550注射液获批临床，拟定适应症为：AZD6234注射液联合AZD9550注射液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的长期体重管理。AZD6234为每周一次皮下注射的长效胰淀素受体激动剂，AZD9550为每周一次的合成GLP-1/胰高血糖素（GCG）双受体激动剂。6、6月25日，CDE官网显示，和剂药业的Soquelitinib（CPI-818）获批Ⅰb/Ⅱ期临床，用于治疗特应性皮炎（AD）患者。Soquelitinib是处于临床阶段的具有高选择性的口服小分子ITK（白细胞介素-2诱导的T细胞激酶）抑制剂。7、6月25日，CDE官网显示，华东医药全资子公司中美华东申报的注射用HDM2020获批临床，适应症为晚期实体瘤。注射用HDM2020是由中美华东研发的1类生物新药，是一款靶向成纤维细胞生长因子受体2b（FGFR2b）的新型抗体偶联药物（ADC）。突破性疗法8、6月23日，CDE官网显示，智康弘义的高选择性内皮素受体A（ETA）小分子拮抗剂SC0062胶囊被纳入突破性疗法，用于治疗伴有白蛋白尿的糖尿病肾脏病（DKD）。此前，SC0062已被CDE纳入突破性疗法，用于治疗伴有蛋白尿的IgA肾病，目前正在进行Ⅲ期临床中。FDA上市批准 9、6月23日，FDA官网显示，第一三共和阿斯利康的德达博妥单抗（Dato-DXd，商品名：Datroway）获批新适应症，用于治疗既往接受过EGFR靶向药物和铂类化疗治疗的局部晚期或转移性EGFR突变非小细胞肺癌（NSCLC）成人患者。这是FDA批准的首个可用于治疗肺癌的TROP2ADC药物。突破性疗法10、6月23日，FDA官网显示，宜联生物的B7-H3靶向ADC药物YL201被授予突破性疗法认定，用于治疗小细胞肺癌（SCLC）。YL201是一款基于宜联生物医药自主创新TMALIN®技术平台开发的靶向B7-H3的ADC药物。2024年10月，YL201已被CDE纳入突破性治疗，用于治疗经初始含铂治疗失败的复发性小细胞肺癌。研发临床状态11、6月25日，舶望制药宣布，其在研小干扰RNA（siRNA）药物BW-40202完成临床I期首例健康受试者给药，该里程碑在澳大利亚临床研究中心达成。此外，BW-40202已获得CDE的新药临床试验（IND）批准，将在中国临床研究中心同步推进。12、6月25日，药物临床试验与信息公示平台显示，挚盟医药登记了一项ZM-H1505R（科诺卡帕韦）用于慢性乙型肝炎的Ⅲ期临床试验。这是该药启动的首个Ⅲ期临床。ZM-H1505R是挚盟医药自主研发的一种强效的HBV核衣壳抑制剂，可以有效抑制HBV核衣壳形成的小分子候选化合物。13、6月27日，药物临床试验登记与信息公示平台显示，信达生物启动了一项CLDN18.2抗体偶联药物（ADC）IBI343用于治疗晚期胰腺癌的多中心、随机、双盲、对照Ⅲ期临床研究，旨在评估IBI343单药联合最佳支持治疗对比安慰剂联合最佳支持治疗用于既往接受过至少两线系统性治疗的CLDN18.2阳性、局部晚期不可切除或转移性胰腺癌受试者的疗效和安全性。该研究是IBI343在胰腺癌中开展的第一项Ⅲ期研究。临床数据14、6月23日，驯鹿生物公布了靶向BCMACAR-T细胞疗法伊基奥仑赛注射液治疗多发性硬化症（MS）的临床研究（ⅡT）。数据显示，伊基奥仑赛注射液在治疗多发性硬化症中表现出良好的耐受性和显著疗效，具体表现为患者躯体功能改善及脑脊液中寡克隆带（OCBs）消失。15、6月24日，博瑞医药公布了胰高血糖素样肽-1受体（GLP-1R）/葡萄糖依赖性促胰岛素多肽受体（GIPR）双靶点激动剂BGM0504的两项Ⅱ期临床数据。数据显示，该药物在2型糖尿病患者中表现出良好的降糖疗效，并初步显示出优于一款已获批的GLP-1受体激动剂的临床疗效。同时在超重/肥胖非2型糖尿病成人群体中，展现出显著的体重管理潜力和代谢风险指标综合获益的潜质。16、6月24日，博瑞医药公布了新型长效胰淀素（Amylin）类似物BGM1812的临床前结果。BGM1812临床前数据显示，其受体激活能力显著增强，可实现强效减重，并与GLP-1/GIP双靶点激动剂产生协同作用，支持其作为新一代肥胖治疗药物开发。17、6月24日，信达生物公布了胰高血糖素（GCG）/胰高血糖素样肽-1（GLP-1）双受体激动剂玛仕度肽（IBI362）在中国2型糖尿病患者中的Ⅲ期临床（DREAMS-1）。数据显示：基于疗效估计目标，第24周时，玛仕度肽4mg和6mg组体重较基线变化值分别为-5.61%和-7.81%，均显著优于安慰剂组（-1.26%）；玛仕度肽4mg和6mg组分别有50.9%和69.0%的受试者体重较基线降幅≥5%，达标率均显著高于安慰剂组（7.3%）。此外，第24周时，玛仕度肽4mg和6mg组分别有40.6%和64.9%的受试者HbA1c<7.0%且体重较基线降幅≥5%，安慰剂组为0。18、6月24日，百奥泰公布了抗VEGF人源化单抗BAT5906针对新生血管性年龄相关性黄斑变性（w-AMD）的关键注册Ⅲ期临床。数据显示，在黄斑中心凹视网膜厚度（CRT）自基线至第52周的变化值方面，BAT5906组显著优于雷珠单抗组，即BAT5906抑制视网膜新生血管渗漏或降低炎症活跃程度明显优于雷珠单抗，均达到预设终点。交易及投融资19、6月23日，百奥泰宣布，与SteinCares就BAT2406签署授权许可及商业化协议，SteinCares将拥有该药在巴西以及其余拉丁美洲地区的独家分销权及销售权。百奥泰将获得首付款及里程碑款总金额最高至1000万美元，其中包括100万美元首付款、累计不超过900万美元里程碑付款，以及净销售额的两位数百分比作为收入分成。BAT2406是度普利尤单抗注射液生物类似药。20、6月23日，和铂医药宣布，与大塚制药达成合作。根据协议，大塚制药将获得在全球范围2内（不包括大中华区）开发、生产和商业化一款用于治疗自身免疫性疾病的BCMAxCD3双特异性T细胞衔接器HBM7020的独家许可。和铂医药将获得总计4700万美元的首付款和近期里程碑付款。此外，和铂医药还有权获得高达6.23亿美元的额外付款等。21、6月24日，Revolution宣布，与Royalty达成合作，后者将为其提供20亿美元的灵活资金，以支持Revolution独立的全球开发和商业化战略和运营该融资协议将提供20亿美元的承诺资本，其中包括高达12.5亿美元的综合特许权使用费货币化，用于销售Revolution的RAS（ON）多选择性抑制剂daraxonrasib（RMC-6236），以及高达7.5亿美元的债务贷款。22、6月25日，荣昌生物宣布，与VorBio达成合作，VorBio将获得泰它西普在除大中华区以外的全球开发、生产和商业化的独家权利；荣昌生物将取得值1.25亿美元现金及认股权证，最高可达41.05亿美元的里程碑付款，总金额42.3亿美元等。泰它西普是注射用重组B淋巴细胞刺激因子（BLyS）/增殖诱导配体（APRIL）双靶点的新型融合蛋白产品。23、6月25日，华润三九宣布，与南京艾尔普再生医学签署HiCM-188（iPSC来源心肌细胞）在中国大陆的合作协议。HiCM-188是国内首个自研iPSC来源心肌细胞药物，该药物已在我国进入Ⅱ期临床，并已经在美国、新加坡、泰国等地区启动Ⅰ/Ⅱ期临床试验。全球已有多款用于心力衰竭iPSC产品在研，但基本都处于临床前研究阶段。24、6月26日，ProFound Therapeutics宣布，与诺华达成一项为期四年的合作协议，双方将基于ProFound专有的ProFoundry平台，共同开展心血管疾病新疗法的发现与开发。根据协议ProFound将获得2500万美元的预付款及近期里程碑付款，并有望在每个候选靶点后续开发中实现高达7.5亿美元的里程碑款项。25、6月26日，迈威生物宣布，与Calico就包括靶向人白介素-11（IL-11）的人源化单抗9MW3811在内的疗法达成独家许可协议。根据协议，迈威生物独家许可Calico在除大中华区以外所有区域内独家开发、生产和商业化9MW3811的权利。Calico将向迈威生物支付一次性不可退还的首付款2,500万美元，此外，迈威生物还可额外获得合计最高达5.71亿美元的近端、开发、注册及商业化里程碑付款等。26、6月26日，迈威生物宣布，与齐鲁制药就注射用阿格司亭α（8MW0511）签署协议。根据协议，迈威生物独占许可齐鲁制药在大中华区（包括中国大陆、中国香港、中国澳门和中国台湾）内开发、生产、改进、利用和商业化许可产品的权利。迈威生物将获得首付款和销售里程碑合计最高达5亿元人民币，其中包括一次性不可退还的首付款3.8亿元人民币，以及许可产品净销售额最高两位数百分比的特许权使用费。上市27、6月23日，药捷安康在港交所挂牌上市，当天股价大涨70%，此后几个交易日股价持续稳定上涨，目前市值达到100亿港元。药捷安康成立于2014年，专注于发现和开发用于治疗肿瘤、炎症和心脏代谢疾病领域的小分子创新药，成立10年来建立起丰富且具差异化特色优势的小分子创新药管线。28、6月24日，上交所上市审核委员会发布公告，将于7月1日召开2025年第21次审议会议，审议发行人为禾元生物。这意味着时隔两年，上交所再次迎来采用科创板第五套标准上市的生物制药企业。END来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com▼更多制药资讯，请关注CPHI制药在线▼点击阅读原文，进入智药研习社~

细胞疗法免疫疗法抗体药物偶联物临床3期上市批准

100 项与雷珠单抗相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
D05697	雷珠单抗	S01LA04	DB01270

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
糖尿病性视网膜病变	美国	Genentech, Inc.	2015-02-06
视网膜静脉阻塞	日本	Novartis Pharma AG	2013-08-20
黄斑水肿	美国	Genentech, Inc.	2010-06-22
年龄相关性黄斑变性	日本	Novartis Pharma AG	2009-01-21
伴有糖尿病的增殖性视网膜病变	澳大利亚	Novartis Pharmaceuticals Australia Pty Ltd.	2008-11-10
早产儿视网膜病变	澳大利亚	Novartis Pharmaceuticals Australia Pty Ltd.	2008-11-10
视力低下	澳大利亚	Novartis Pharmaceuticals Australia Pty Ltd.	2008-11-10
脉络膜新生血管	欧盟	Novartis Europharm Ltd.	2007-01-22
脉络膜新生血管	冰岛	Novartis Europharm Ltd.	2007-01-22
脉络膜新生血管	列支敦士登	Novartis Europharm Ltd.	2007-01-22
脉络膜新生血管	挪威	Novartis Europharm Ltd.	2007-01-22
糖尿病性黄斑水肿	欧盟	Novartis Europharm Ltd.	2007-01-22
糖尿病性黄斑水肿	冰岛	Novartis Europharm Ltd.	2007-01-22
糖尿病性黄斑水肿	列支敦士登	Novartis Europharm Ltd.	2007-01-22
糖尿病性黄斑水肿	挪威	Novartis Europharm Ltd.	2007-01-22
视网膜静脉阻塞相关黄斑水肿	欧盟	Novartis Europharm Ltd.	2007-01-22
视网膜静脉阻塞相关黄斑水肿	冰岛	Novartis Europharm Ltd.	2007-01-22
视网膜静脉阻塞相关黄斑水肿	列支敦士登	Novartis Europharm Ltd.	2007-01-22
视网膜静脉阻塞相关黄斑水肿	挪威	Novartis Europharm Ltd.	2007-01-22
湿性年龄相关性黄斑变性	美国	Genentech, Inc.	2006-06-30

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适应症	最高研发状态	国家/地区	公司	日期
近视脉络膜新生血管	临床3期	中国	北京诺华制药有限公司	2013-09-11
近视脉络膜新生血管	临床3期	中国	Novartis Europharm Ltd.	2013-09-11
近视脉络膜新生血管	临床3期	中国	Novartis Pharma Stein AG	2013-09-11
近视脉络膜新生血管	临床3期	印度	Novartis Europharm Ltd.	2013-09-11
近视脉络膜新生血管	临床3期	印度	北京诺华制药有限公司	2013-09-11
近视脉络膜新生血管	临床3期	印度	Novartis Pharma Stein AG	2013-09-11
近视脉络膜新生血管	临床3期	韩国	Novartis Europharm Ltd.	2013-09-11
近视脉络膜新生血管	临床3期	韩国	Novartis Pharma Stein AG	2013-09-11
近视脉络膜新生血管	临床3期	韩国	北京诺华制药有限公司	2013-09-11
近视脉络膜新生血管	临床3期	泰国	Novartis Pharma Stein AG	2013-09-11

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临床结果

适应症

分期

评价

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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


NCT04108156 (Pubmed \| JAMA Ophthalmol)	临床3期	糖尿病性黄斑水肿	634	Ranibizumab 100 mg/mL via PDS Q24W	獵醖窪鏇壓襯選選獵獵(醖鬱餘糧願築餘齋積齋) = more common in the PDS Q24W group (88 participants; 27.5%) than the monthly ranibizumab group (28 participants; 8.9%) 顧衊醖襯顧憲鬱夢蓋鹹 (獵鏇憲遞窪齋壓積鏇顧 )	积极	2025-04-01
				Monthly Ranibizumab 0.5 mg
NCT04503551 (Pubmed \| JAMA Ophthalmol)	临床3期	非增殖性糖尿病视网膜病变	174	Port Delivery System with Ranibizumab	蓋鏇淵窪醖製繭餘窪餘(顧繭憲築顧遞觸鹹製獵) = cataract, 7 participants [6.7%]; vitreous hemorrhage, 6 participants [5.7%]; conjunctival bleb, conjunctival retraction, and hyphema, each 2 participants [1.9%]; conjunctival erosion and retinal detachment, each 1 participant [1.0%] 鏇鹹鑰簾鑰淵網鏇衊製 (顧繭繭獵築願壓鹽觸廠 )	积极	2025-04-01
				Ranibizumab (Control (No PDS))
NCT03683251 (Portal Extension Trial, Pubmed \| Ophthalmol Retina)	临床3期	湿性年龄相关性黄斑变性	555	PDS 100 mg/ml	夢鏇衊願網廠窪選廠廠(構簾顧廠鹹淵鏇憲廠鏇) = 11.4% 廠積鹽鹹鹽壓衊獵製鹽 (膚餘夢鬱鬱製選顧築醖 ) 更多	积极	2025-02-01
				Monthly intravitreal ranibizumab 0.5-mg injections
JPRN-jRCTs011200010 (HGCSG 1903, ASCO_GI2025)	临床2期	晚期胃癌二线	36	Ramucirumab plus Docetaxel	繭繭選積襯網繭簾製蓋(鬱顧觸糧獵顧壓範襯鑰) = 築淵繭廠積觸膚餘願顧範壓簾願淵簾蓋鑰鹹艱 (鹽選鏇簾夢齋餘鹽夢範 ) 更多	积极	2025-01-23
NCT03677934 (Archway, Pubmed \| Ophthalmol Retina)	临床3期	anti-vascular endothelial growth factor therapy	418	Port Delivery System with ranibizumab (PDS)	鬱膚構艱選構願鏇構廠(鏇製鹽構範壓淵鹽窪鹹) = 齋醖簾膚鹹繭簾構積鏇獵窪願獵鹽鹽鬱遞壓願 (壓網網衊壓餘網蓋衊廠 ) 更多	积极	2024-10-01
				Monthly intravitreal ranibizumab injections	鬱膚構艱選構願鏇構廠(鏇製鹽構範壓淵鹽窪鹹) = 鹽齋顧觸積範觸淵鬱襯獵窪願獵鹽鹽鬱遞壓願 (壓網網衊壓餘網蓋衊廠 ) 更多
EURETINA2024	N/A	怀孕	-	Aflibercept	願遞顧鏇蓋餘積餘憲簾(夢齋鬱壓艱觸觸廠選積) = 簾壓構餘窪齋積範觸憲淵憲觸襯願壓網憲艱蓋 (襯網廠淵艱鑰鑰網願糧 )	-	2024-09-19
				Bevacizumab	願遞顧鏇蓋餘積餘憲簾(夢齋鬱壓艱觸觸廠選積) = 範築夢廠鏇鑰簾築膚淵淵憲觸襯願壓網憲艱蓋 (襯網廠淵艱鑰鑰網願糧 )
NCT02510794 \| NCT03683251 \| NCT03677934 (Archway \| Portal \| Ladder, EURETINA2024)	临床2/3期	湿性年龄相关性黄斑变性 vascular endothelial growth factor	-	Port Delivery System with ranibizumab (PDS)	觸窪顧憲鹽選齋襯蓋築(艱範選鏇廠繭觸築築範) = BCVA remained stable for 5 years, with an observed BCVA Snellen equivalent of 20/40 and mean (95% CI) change from baseline of −1.8 ETDRS letters (−8.1, 4.4; n=17) at month 60 窪餘簾餘鹹網鏇鹽鹽繭 (簾網餘鬱鹽願遞壓襯鑰 ) 更多	积极	2024-09-19
				Monthly intravitreal ranibizumab injections
EURETINA2024	N/A	玻璃体积血	-	Intravitreal Ranibizumab	夢憲醖齋獵壓鹹築艱積(積鏇壓積蓋壓製築獵選) = 獵鹹獵獵夢範艱窪鬱顧蓋蓋膚遞糧襯築簾簾窪 (範鬱醖淵艱築壓鹽蓋廠 ) 更多	-	2024-09-19
EURETINA2024	N/A	湿性年龄相关性黄斑变性	-	PDS with ranibizumab 100 mg/mL with fixed 24-week refill-exchanges (Q24W)	夢網齋鹹製醖製廠壓夢(觸醖衊餘廠願糧鏇鑰簾) = 鹽遞遞製繭鑰繭糧鏇遞顧築構窪鏇願積網壓壓 (積製窪製夢築廠選夢繭 )	-	2024-09-19
				Intravitreal ranibizumab 0.5-mg injections every 4 weeks (monthly ranibizumab)	夢網齋鹹製醖製廠壓夢(觸醖衊餘廠願糧鏇鑰簾) = 獵簾遞窪築遞鑰窪衊壓顧築構窪鏇願積網壓壓 (積製窪製夢築廠選夢繭 )
EURETINA2024	N/A	湿性年龄相关性黄斑变性	-	Intravitreal Ranibizumab (IVR)	襯獵觸淵網繭鹹憲襯獵(衊選壓壓夢窪繭膚鹽醖) = 窪襯膚觸淵淵衊鏇襯獵顧窪膚觸網鑰醖衊艱憲 (鏇醖衊夢簾遞鏇網鑰繭 ) 更多	-	2024-09-19
				Intravitreal Aflibercept (IVA)	襯獵觸淵網繭鹹憲襯獵(衊選壓壓夢窪繭膚鹽醖) = 糧繭襯醖選衊鏇積憲築顧窪膚觸網鑰醖衊艱憲 (鏇醖衊夢簾遞鏇網鑰繭 ) 更多