Clinical trial of KH631 evaluates single administration gene-therapy treatment for wet AMD

KH631的临床试验评估了湿性AMD的单药基因治疗

CHENGDU, China and ROCKAWAY, N.J., Nov. 20, 2023 /PRNewswire/ -- Chengdu Origen Biotechnology Co., Ltd. ('Chengdu Origen') and Vanotech Ltd. ('Vanotech') announced today the first patient dosed in the VAN-2201 multi-center Phase 1 clinical trial evaluating treatment with KH631 for patients with wet Age-related Macular Degeneration (wet AMD).

中国成都和新泽西州ROCKAWAY，2023年11月20日/PRNewswire/-成都奥利根生物技术有限公司（“成都奥利根”）和Vanotech有限公司（“Vanotech”）今天宣布第一名患者服用VAN-2201多中心1期临床试验评估KH631治疗湿性年龄相关性黄斑变性（湿性AMD）患者的疗效。

This multi-center, open-label, dose-escalation clinical trial in the U.S. will assess the safety, tolerability and efficacy of KH631 as a single administration gene therapy for patients with previously treated wet AMD..

这项在美国进行的多中心，开放标签，剂量递增的临床试验将评估KH631作为先前治疗过的湿性AMD患者的单药基因疗法的安全性，耐受性和有效性。。

KH631 is a recombinant adeno-associated virus vector that encodes a human VEGF receptor fusion protein. In preclinical studies of wet AMD disease models, administration of KH631 resulted in retention of the transgene product in the retina for prolonged periods and prevented the disease progression. These findings may indicate the potential of KH631 to offer a single administration treatment for wet AMD patients..

KH631是编码人VEGF受体融合蛋白的重组腺相关病毒载体。在湿性AMD疾病模型的临床前研究中，KH631的施用导致转基因产物长时间保留在视网膜中并阻止疾病进展。这些发现可能表明KH631可能为湿性AMD患者提供单次给药治疗。。

'Dosing our first patient in the VAN-2201 Phase 1 trial is an important milestone in the advancement of KH631 by exploring the potential of single administration gene therapy as treatment for wet AMD. KH631 is designed to deliver anti-VEGF continuously to the retina, with the potential to provide sustained levels that can control the disease,' said Avner Ingerman, M.D., Chief Medical Officer of Vanotech..

'通过探索单药基因治疗作为湿性AMD治疗的潜力，在VAN-2201 1期试验中给我们的第一位患者服用药物是KH631进展的重要里程碑。Vanotech首席医疗官Avner Ingerman博士说，KH631旨在将抗VEGF持续递送至视网膜，并有可能提供可控制疾病的持续水平。。

'I am excited to participate as a principal investigator in this important study. Dosing the first patient is an important milestone in the development of the single-administration gene therapy approach, which may hold promise to our patients in need of frequent treatment for their retinal diseases,' said Jeffrey S. Heier, M.D., a principal investigator in the VAN-2201 Phase 1 trial, director of the Vitreoretinal Service and Director of Retina Research at Ophthalmic Consultants of Boston.

“我很高兴参加这项重要研究的首席研究员。VAN-2201第一阶段试验的学术带头人Jeffrey S.Heier说，给第一个病人服用药物是开发单一给药基因治疗方法的一个重要里程碑，这种方法可能为我们需要频繁治疗视网膜疾病的病人带来希望，波士顿眼科顾问玻璃体视网膜服务总监和视网膜研究总监。

About the VAN-2201 Phase 1 Clinical Trial VAN-2201 is a Phase 1, multi-center, open-label, dose‑escalation study in subjects with wet AMD to evaluate safety, tolerability and efficacy of a single administration of KH631. The study is expected to include approximately twenty-five previously treated wet AMD subjects that are responsive to anti-vascular endothelial growth factor (anti-VEGF) therapy. For more information, please visit https://www.clinicaltrials.gov/study/NCT05657301..

关于VAN-2201 1期临床试验VAN-2201是一项针对湿性AMD患者的1期多中心开放标签剂量递增研究，旨在评估单次给予KH631的安全性，耐受性和有效性。该研究预计将包括大约25名先前治疗过的对抗血管内皮生长因子（抗VEGF）治疗有反应的湿性AMD受试者。欲了解更多信息，请访问https://www.clinicaltrials.gov/study/NCT05657301..

About Wet AMDWet AMD is the rarer form of AMD, characterized by loss of vision due to abnormal blood vessel formation in and under the retina. These blood vessels tend to leak, and the fluid interferes with the normal retinal structure and function, leading to vision loss. Wet AMD is a leading cause of irreversible vision loss or blindness in the United States and Europe, especially if left untreated.

关于湿性AMDWet AMD是AMD的罕见形式，其特征在于由于视网膜内和视网膜下的异常血管形成而导致视力丧失。这些血管倾向于泄漏，并且流体干扰正常的视网膜结构和功能，导致视力丧失。湿性AMD是美国和欧洲不可逆转的视力丧失或失明的主要原因，尤其是如果不加以治疗的话。

It is estimated that ~1.5 million people living with wet AMD in the United States..

据估计，美国约有150万人患有湿性AMD。。

Anti-VEGF therapies have become the standard of care for wet AMD. Anti-VEGF treatments reduce leakage and fluid accumulation, thereby restoring normal retinal architecture and function, in many patients. Anti-VEGF agents require frequent, repeat injections to maintain efficacy and control the disease..

抗VEGF疗法已成为湿性AMD的护理标准。在许多患者中，抗VEGF治疗减少渗漏和液体积聚，从而恢复正常的视网膜结构和功能。抗VEGF药物需要频繁重复注射以维持疗效并控制疾病。。

About Chengdu Origen and VanotechChengdu Origen is a clinical-stage gene therapy company focused on developing gene therapy for unmet medical needs and providing meaningful clinical benefits for patients suffering from genetic and chronic diseases and has comprehensive viral vector manufacturing capabilities and infrastructure..

关于成都Origen和VanotechChengdu Origen是一家临床阶段基因治疗公司，致力于为未满足的医疗需求开发基因治疗，并为患有遗传和慢性疾病的患者提供有意义的临床益处，并拥有全面的病毒载体制造能力和基础设施。。

Vanotech is operating as the sponsor-representative, and is responsible for the clinical development program of KH631 in the united States.

Vanotech是赞助商代表，负责KH631在美国的临床开发计划。

Forward Looking StatementsThis press release contains 'forward-looking statements,' within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, Chengdu Origen's and Vanotech's research and development plans in connection with its clinical development program and the gene therapy treatments.

前瞻性声明本新闻稿根据1995年“私人证券诉讼改革法”的含义，包含“前瞻性声明”，其中包括成都Origen和Vanotech的临床开发计划和临床开发计划基因治疗。

Such forward-looking statements include inherent risks and uncertainties, including factors that could cause actual results to differ materially from those projected by such forward-looking statements. All of Chengdu Origen/Vanotech development timelines could be subject to adjustment depending on recruitment rate, regulatory agency review and other factors that could delay the initiation and completion of clinical trials.

此类前瞻性声明包括固有风险和不确定性，包括可能导致实际结果与此类前瞻性声明所预测的结果大不相同的因素。所有成都Origen/Vanotech开发时间表均可根据招聘率，监管机构审查和其他可能延迟临床试验启动和完成的因素进行调整。

Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the potential impact of pandemics and other health emergencies, including their impact on the global supply chain; the uncertainty that our product candidates will receive regulatory approval necessary to initiate human clinical studies; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical tri.

每个前瞻性声明都存在重大风险和不确定性，可能导致实际结果与本声明中表达或暗示的结果有重大差异，包括但不限于与以下相关的风险和不确定性：我们开发，获得监管批准的能力，并将我们的产品候选人商业化，这可能需要比计划更长或更高的成本；我们筹集可能无法获得的额外资金的能力；我们为产品候选人获得，维护和执行专利和其他知识产权保护的能力；流行病和其他突发卫生事件的潜在影响，包括其对全球供应链的影响；我们的产品候选人将获得启动人体临床研究所需的监管批准的不确定性；我们的候选产品的临床前测试以及来自临床前研究和临床试验的初步或中期数据可能无法预测正在进行或以后的临床tri的结果或成功。

All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. Investors are cautioned not to place undue reliance on these forward-looking statements. These forward-looking statements speak only as of the date of this press release.

本新闻稿中包含的所有前瞻性声明均受本文所包含或引用的警示性声明的明确限制。提醒投资者不要过分依赖这些前瞻性陈述。这些前瞻性声明仅在本新闻稿发布之日起才发表。

Chengdu Origen and Vanotech undertake no obligation to update any forward-looking statements, whether as a result of new information, future developments or otherwise..

无论是由于新信息，未来发展还是其他原因，成都Origen和Vanotech均无义务更新任何前瞻性声明。。

SOURCE Vanotech Ltd.

源Vanotech有限公司。