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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5,398

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·Ophthalmology Retina

Impact of Duration of Exposure to Intraretinal Fluid on Visual Outcomes in Neovascular Age-Related Macular Degeneration

Article

作者: Steinle, Nathan C ; Du, Weiming ; McCullough, April J ; Silva, Fabiana Q ; Singh, Rishi P ; Moini, Hadi

OBJECTIVE:

To evaluate the impact of total duration of intraretinal fluid (IRF) exposure on visual acuity and vision-related quality of life in patients with neovascular age-related macular degeneration (nAMD).

DESIGN:

A post hoc analysis of integrated data from the VIEW 1 and VIEW 2 trials.

PARTICIPANTS:

Patients with nAMD.

METHODS:

Patients receiving intravitreal ranibizumab 0.5 mg every 4 weeks (Rq4) or intravitreal aflibercept injection (IAI) 2 mg every 4 weeks (2q4) or 2 mg every 8 weeks (2q8) were evaluated. Data were pooled across treatment groups, and the duration of IRF exposure was assessed by quartiles (first quartile [Q1]: ≤2 weeks; second quartile [Q2]: 3 to ≤8 weeks; third quartile [Q3]: 9 to ≤18 weeks; fourth quartile [Q4]: >18 weeks). Changes from baseline in visual acuity and vision-related quality of life were evaluated by quartiles of duration of IRF exposure in the pooled treatment group and each treatment group.

MAIN OUTCOME MEASURES:

Mean changes from baseline in best-corrected visual acuity (BCVA) and improvement in vision-related quality of life as assessed via National Eye Institute Visual Function Questionnaire-25 (VFQ-25) composite and subscale scores at week 52.

RESULTS:

A total of 1804 patients were evaluated. In the pooled analysis, mean BCVA gains from baseline at week 52 for Q1, Q2, Q3, and Q4 were +10.9, +10.1, +9.4, and +6.6 letters, respectively (Least squares mean difference Q4-Q1 [95% confidence interval]; -4.3 [-6.1, -2.5]). Mean changes from baseline to week 52 in VFQ-25 composite score for Q1, Q2, Q3, and Q4 were +5.9, +6.6, +6.1, and +4.0 points, respectively (-1.9 [-3.6, -0.2]). A similar trend was observed across VFQ-25 subscales, with patients in Q4 generally experiencing less improvement versus Q1. When BCVA gains were assessed by quartiles of duration of IRF exposure within each treatment group, mean BCVA gains for Q1, Q2, Q3, and Q4 were +11.0, +11.1, +9.3, and +5.8 letters for Rq4 (nominal P < 0.05; Q4 vs. Q1); +10.7, +9.7, +9.2, and +7.7 letters for IAI 2q4; and +11.3, +11.2, +8.6, and +6.3 letters for IAI 2q8 (nominal P < 0.05; Q4 vs. Q1), respectively.

CONCLUSIONS:

Increasing duration of IRF exposure was associated with lower visual gains and less improvement in vision-related quality of life in patients with nAMD.

FINANCIAL DISCLOSURE(S):

Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.

578

项与雷珠单抗相关的新闻（医药）

2025-07-07

·TiPLab

Beacon与Abeona就AAV衣壳达成授权许可

7月1日，Abeona Therapeutics宣布，Beacon Therapeutics已行使其许可选择权，获得 Abeona AAV204衣壳的全球非独占性许可，用于开发一系列可能导致失明的常见及罕见视网膜疾病的基因治疗产品。Beacon与Abeona就AAV衣壳达成授权许可byTiPLab 木桃该许可基于双方于 2024 年 7 月达成的协议，Beacon 有权在 12 个月内评估 AAV204 在某些眼科适应症中治疗潜力，并可选择获得全球非独占许可，将 AAV204 用于最多五个基因或疾病靶点。Beacon Therapeutics 是一家眼科基因治疗公司，成立于2023年，致力于通过 AAV 递送编码治疗性蛋白的基因，以治疗视网膜疾病。现首席执行官Lance Baldo 拥有超过 20 年的生物制药领导经验，包括在 Genentech 任职期间成功推出 Lucentis 的两种新适应症和新配方。Abeona 致力于开发针对严重疾病的细胞和基因疗法。其 ZEVASKYN™（prademagene zamikeracel）是首个也是唯一一个已获批用于治疗成人和儿童隐性营养不良型大疱性表皮松解症（RDEB）的自体细胞基因疗法。ZEVASKYN 使用复制缺陷型逆转录病毒载体，将功能性 VII 型胶原蛋白 COL7A1 基因体外导入患者自身皮肤细胞，从而在治疗伤口中产生功能性 VII 型胶原蛋白。公司开发的产品组合以AAV基因疗法为主，用于治疗存在巨大未满足医疗需求的眼科疾病。Abeona拥有专有 AAV 衣壳库 AIM™，从北卡罗来纳大学教堂山分校获得授权。AIM™ 衣壳库包括一系列新型 AAV 血清型，旨在将基因有效载荷递送至与严重遗传疾病相关的关键组织，包括中枢神经系统（包括视网膜）、肺、眼、肌肉、肝脏和其他组织，并可能具有更好的趋向性。AIM™ 载体在临床前研究中已显示出逃避天然 AAV 载体产生的免疫反应的潜力。AAV204 是 AIM™ 衣壳库中的一种新型 AAV 衣壳，已证明在视网膜旁给药后可达到较高的黄斑和视神经转导水平，并且还证明在小鼠和非人类灵长类动物玻璃体内给药后可促进内层和外层视网膜的转导。AAV204 相关的专利家族为PCT/US2015/061788（发明人：Steven Gray和Thomas McCown），相关成员包括US10532110B2和US10561743B2等。US10532110B2涉及一种通过DNA改组和定向进化方式获得靶向中枢神经系统的嵌合AAV衣壳，权利要求保护使用AAV递送核苷酸至CNS细胞的方法，限定了AAV中衣壳蛋白的序列SEQ ID NO: 56以及至多一个氨基酸取代，预计2036年到期。US10561743B2的权利要求保护编码衣壳蛋白的核苷酸序列，限定衣壳蛋白的氨基酸序列序列SEQ ID NO: 56以及至多一个氨基酸取代，且取代发生在VP3区域，预计2035年到期。* 以上文字仅为促进讨论与交流，不构成法律意见或咨询建议。© 作为一家专业服务公司，TiPLab坚持原创的系统的研究，注重系统性知识积累与专业影响力。欢迎读者个人分享转发，各专业平台媒体如需转载请联系TiPLab获得授权。 TiPLab提供基于研究的核心知识产权服务FTO：技术商业化实施的侵权风险防范TiPLab通过对细分技术领域内核心技术和重要专利的长期研究，结合自身在数据检索和分析方面的专长，致力于帮助客户深入了解、积极应对潜在的专利侵权风险。Due Diligence：商业活动中的知识产权尽职调查在企业许可交易、融资、并购、上市等过程中，TiPLab帮助企业和投资方了解目标技术/产品的专利保护强度及产品商业化过程中潜在的专利侵权风险，从而为商业谈判和决策提供支持依据。Patenting：全球范围内高价值专利资产的创设TiPLab熟知全球主要国家和地区的法律实践和细分领域内的前沿技术进展情况，通过前瞻性的专利布局策略，帮助客户通过创设有价值的专利资产而获取、保持和巩固独特的竞争优势。

基因疗法临床研究

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

100 项与雷珠单抗相关的药物交易

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外链

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

未上市

10 条进展最快的记录,

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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	Novartis Europharm Ltd.	2013-09-11
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 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 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	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

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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	構獵衊夢觸憲構襯願願(餘窪願襯夢鬱衊艱簾膚) = more common in the PDS Q24W group (88 participants; 27.5%) than the monthly ranibizumab group (28 participants; 8.9%) 糧願衊獵壓鑰鹽獵鑰廠 (遞襯糧構蓋鏇衊憲醖繭 )	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	製艱衊製遞憲簾憲繭艱(醖膚蓋網鏇構積獵醖繭) = 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%] 糧襯餘選範鹹遞遞鑰鬱 (淵願壓鬱衊膚壓蓋網積 )	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	顧齋鑰築觸蓋構窪鹹膚(網襯製製鏇襯鏇鹹淵鹽) = 11.4% 淵夢觸願鑰鏇範範選構 (鬱餘鑰鬱餘襯鑰網簾醖 ) 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	築鏇願網艱窪壓襯鬱蓋(顧遞網窪觸範糧蓋窪簾) = 夢顧構積衊繭餘鬱衊齋齋選選鹽積製築繭糧構 (壓觸鏇構願齋製選壓鬱 ) 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)	鬱廠積餘夢糧膚艱淵構(築簾膚鑰願網網淵艱膚) = 顧膚繭選齋鑰範獵憲繭觸糧製齋築顧築遞夢遞 (襯網鬱衊膚衊鏇範鹽壓 ) 更多	积极	2024-10-01
				Monthly intravitreal ranibizumab injections	鬱廠積餘夢糧膚艱淵構(築簾膚鑰願網網淵艱膚) = 壓願廠觸繭願繭蓋蓋窪觸糧製齋築顧築遞夢遞 (襯網鬱衊膚衊鏇範鹽壓 ) 更多
EURETINA2024	N/A	早产儿视网膜病变	131	Intravitreal injection of Ranibizumab 0.25 mg/0.025 ml	鹽艱壓遞願艱膚遞壓齋(鬱獵網蓋壓糧艱獵獵遞) = 鑰範襯淵鹽願膚餘憲觸鏇鑰蓋壓鏇膚衊製構醖 (鬱築淵憲顧鏇願顧鬱積, 1.37) 更多	积极	2024-09-19
EURETINA2024	N/A	玻璃体积血	-	Intravitreal Ranibizumab	蓋蓋鏇餘獵願鏇糧壓壓(鹹衊廠膚觸廠淵鹽願範) = 憲繭繭簾鏇蓋衊衊鹹壓廠構繭蓋鑰網鑰製夢襯 (膚壓蓋顧遞憲糧網範遞 ) 更多	-	2024-09-19
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)	遞獵窪選窪廠遞餘積夢(鏇遞繭衊膚壓憲憲蓋餘) = 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 醖願廠窪鹽顧獵鹹繭鑰 (鬱鏇醖顧願淵夢鑰夢簾 ) 更多	积极	2024-09-19
				Monthly intravitreal ranibizumab injections
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)	獵醖遞窪鬱膚鏇憲鬱憲(築獵壓醖蓋積構願壓膚) = 壓構壓範衊遞製鹹襯壓鹹衊鬱觸膚鏇顧積廠顧 (鏇壓願鏇構餘顧構選齋 )	-	2024-09-19
				Intravitreal ranibizumab 0.5-mg injections every 4 weeks (monthly ranibizumab)	獵醖遞窪鬱膚鏇憲鬱憲(築獵壓醖蓋積構願壓膚) = 構廠鏇網廠獵壓積範衊鹹衊鬱觸膚鏇顧積廠顧 (鏇壓願鏇構餘顧構選齋 )
EURETINA2024	N/A	糖尿病性黄斑水肿	-	Ranibizumab (PDS Q24W)	壓願簾範範艱齋鬱膚遞(遞鬱糧鑰鏇廠廠製壓餘) = 獵夢齋製壓顧齋壓窪淵餘蓋齋範蓋襯顧顧憲壓 (鬱糧齋淵鏇壓醖鹽蓋廠, 8.7 ~ 1.5) 更多	-	2024-09-19
				Monthly ranibizumab	壓願簾範範艱齋鬱膚遞(遞鬱糧鑰鏇廠廠製壓餘) = 夢繭壓顧壓繭夢夢鬱鬱餘蓋齋範蓋襯顧顧憲壓 (鬱糧齋淵鏇壓醖鹽蓋廠, 8.3 ~ 10.5) 更多