SUWON, South Korea--(BUSINESS WIRE)--OliX Pharmaceuticals, Inc. (KOSDAQ: 226950), a leading developer of RNAi therapeutics, today announced positive results from a Phase 1 study evaluating the safety and tolerability of OLX10212 for the treatment of Age-Related Macular Degeneration (AMD). AMD is the most common cause of blindness in the industrialized world, which affects more than 170 million people worldwide, and currently available treatments are often insufficient to improve existing AMD or mitigate disease progression.

韩国水原-（商业线）-OliX Pharmaceuticals，Inc。（KOSDAQ:226950），RNAi therapeutics的领先开发商，今天宣布了一项评估OLX10212安全性和耐受性的1期研究的积极成果。治疗年龄相关性黄斑变性（AMD）。AMD是工业化世界中最常见的失明原因，影响全球超过1.7亿人，目前可用的治疗方法通常不足以改善现有的AMD或减轻疾病进展。

The development of small interfering RNAs (siRNAs), like OLX10212 which showed efficacy in non-clinical models for both main forms of AMD, neovascular and dry AMD, allows targeting pathways that are believed to play a critical role in the development of AMD, and therefore present a novel approach for the treatment of this patient population..

小干扰RNA（siRNA）的开发，如OLX10212，其在非临床模型中显示对两种主要形式的AMD，新生血管和干性AMD的功效，允许靶向途径被认为在AMD的发展中起关键作用，因此，提出了一种治疗该患者群的新方法。。

This phase 1 study is a multi-center, single-dose, dose-escalating study to evaluate the safety and tolerability of OLX10212 in patients with neovascular AMD. The primary endpoints of this study were safety and tolerability assessments associated with each intravitreal OLX10212 injection. Safety and tolerability were assessed based on a combination of ophthalmologic and systemic evaluations for 2 weeks during the dose-limiting toxicity (DLT) evaluation period followed by an additional 22 weeks of clinical follow-up, for a total duration of 24 weeks.

这项1期研究是一项多中心，单剂量，剂量递增的研究，旨在评估OLX10212在新生血管性AMD患者中的安全性和耐受性。本研究的主要终点是与每次玻璃体内OLX10212注射相关的安全性和耐受性评估。在剂量限制性毒性（DLT）评估期间，基于眼科和全身评估的组合评估安全性和耐受性2周，然后再进行22周的临床随访，总共持续24周。

Preliminary efficacy evaluations included best corrected visual acuity (BCVA) changes from baseline during follow-up..

初步疗效评估包括随访期间最佳矫正视力（BCVA）从基线的变化。。

In this study, OLX10212 at dose levels between 100μg/eye/50μL and 950μg/eye/50μL was administered via a single intravitreal injection. Fifteen AMD patients received the treatment. Neither during the DLT period nor during the follow-up period up to 24 weeks post-injection, there were any observations of adverse effects related to OLX10212.

在该研究中，通过单次玻璃体内注射施用剂量水平在100μg/眼/50μL和950μg/眼/50μL之间的OLX10212。15名AMD患者接受了治疗。在DLT期间和注射后24周的随访期间，都没有观察到与OLX10212相关的不良反应。

Specifically, there were no signs of inflammation or changes of intraocular homeostasis noted in all patients. In addition, no systemic effects were observed. Sporadic ophthalmic adverse effects were transient, mild and related to the dose administration procedure as expected for intravitreal injections.

具体而言，在所有患者中均未发现炎症迹象或眼内稳态变化。另外，没有观察到全身作用。散发性眼科不良反应是短暂的，轻微的，并且与玻璃体内注射所预期的剂量给药程序有关。

Importantly, this study identified dose levels suitable for evaluations of efficacy testing in future clinical trials..

重要的是，这项研究确定了适合评估未来临床试验中疗效测试的剂量水平。。

Altogether, in this first-in-human study in AMD patients the study objectives were met. The safety and tolerability evaluations, together with preliminary BCVA improvement of OLX10212 encourage further development of OLX10212 for AMD.

总而言之，在这项针对AMD患者的首次人体研究中，达到了研究目标。安全性和耐受性评估，以及OLX10212的BCVA初步改进，鼓励了针对AMD的OLX10212的进一步开发。

Dong Ki Lee, Ph.D., CEO of OliX, said: “OliX and its Ophthalmology Division are strongly committed to use our proprietary siRNA platform technology to advance the development of novel, safe and effective treatments for our patients. We are thrilled about the outcome of this first-in-human trial in patients with wet AMD who were treated with OLX10212.

OliX首席执行官李东基博士说：“OliX及其眼科部门坚信致力于使用我们专有的siRNA平台技术，为我们的患者开发新颖，安全和有效的治疗方法。我们对这项首次人体试验在接受OLX10212治疗的湿性AMD患者中的结果感到兴奋。

The excellent safety and tolerability data, paired with encouraging preliminary BCVA improvement reassure the next steps in this program, and we are confident that OLX10212 and its novel mechanism of action may have potential to provide benefits in the treatment of AMD patients.”.

出色的安全性和耐受性数据，加上令人鼓舞的BCVA初步改善，使该计划的下一步放心，我们相信OLX10212及其新颖的作用机制可能有助于AMD患者的治疗。

Demetrios G. Vavvas, M.D., Ph.D., Director Retina Service, Harvard Medical School, and advisor to OliX said, “The safety data from this first in human eyes of asymmetric siRNA is a very important step in having this technology to our arsenal of therapeutics. I am looking forward to the next phase of these trials.”.

哈佛医学院视网膜服务主任Demetrios G.Vavvas博士，博士和OliX顾问说：“不对称siRNA首次在人眼中的安全性数据是将这项技术应用于我们的治疗药物库的一个非常重要的步骤。我期待着这些试验的下一阶段。”。

Veeral Sheth, M.D., University Retina and Macula Associates P.C who participated in the trial said: “Our commitment to addressing the challenge of blindness caused by macular degeneration has led to substantial advancements, though there remains a notable divergence between the efficacy observed in controlled clinical trials and the outcomes in everyday clinical practice.

参与试验的Veeral Sheth，M.D.，大学视网膜和Macula Associates P.C说：“我们致力于解决由黄斑变性引起的失明挑战已经取得了实质性进展，尽管在对照临床试验中观察到的疗效与日常临床实践中的结果之间仍存在显着差异。

This reality motivates our search for novel therapeutic options that promise safety, effectiveness, and an enhanced quality of life for our patients. The novel mechanism of OLX10212, coupled with the encouraging safety, tolerability, data emerging from the Phase 1 study, provides substantial optimism for the advancement of this treatment in the realm of wet macular degeneration therapy.”.

这一现实促使我们寻找能够为患者提供安全，有效和提高生活质量的新型治疗选择。OLX10212的新机制，加上令人鼓舞的安全性，耐受性，第一阶段研究中出现的数据，为湿性黄斑变性治疗领域的这种治疗进展提供了实质性的乐观。

In 2020, OliX inked a license and collaboration agreement with Théa Open Innovation (TOI) (a sister company of ophthalmic specialty pharmaceutical company Laboratoires Théa), where TOI has secured worldwide rights except Asia-Pacific to the OLX10212 program, while OliX retains the rights in Asia-Pacific..

2020年，OliX与ThéaOpen Innovation（TOI）（眼科专业制药公司LaboratoiresThéa的姐妹公司）签订了许可和合作协议，TOI已获得亚太地区OLX10212计划以外的全球权利，而OliX保留了权利在亚太地区。。