中美瑞康宣布其渐冻症创新疗法RAG-17在IIT研究中取得突破性成果

这些积极的临床结果体现了中美瑞康此前发表的RAG-17的临床前数据的高度可转化性。中美瑞康的临床前研究表明，RAG-17在SOD1-G93A ALS小鼠和大鼠模型中表现出显著的治疗效果。研究显示，RAG-17显著延缓了疾病的进展并改善了生存率，这为其作为ALS-SOD1有效疗法的潜力提供了有力的临床前证据。

中美瑞康创始人兼首席执行官李龙承博士表示：“我们对这些初步的临床结果感到鼓舞，这使我们为 ALS 患者带来新希望的目标更接近了一步。这次试验的积极结果突显了 RAG-17 在治疗 ALS-SOD1 方面的潜力。我们将继续致力于推进其临床开发，最终为患者带来一种有可能改变生命的疗法。”

该研究的数据将于今年在三个重要会议上公布：9月的中国第27届全国神经病学大会，10月在美国芝加哥举行的神经科学2024大会，以及12月在加拿大蒙特利尔举行的第35届国际ALS/MND研讨会——这是全球最大的ALS和运动神经疾病研究年度会议之一。

RAG-17于2023年3月获得美国FDA的孤儿药（Orphan Drug Designation, ODD）认定，随后也获得了FDA临床试验许可，于2024 年 5 月获得中国国家药品监督管理局药品审评中心（CDE）的临床试验许可。

RAG-17是一款以SOD1为靶基因的双链小干扰RNA（siRNA），通过降低SOD1基因表达来治疗SOD1突变所致的ALS患者。RAG-17采用了中美瑞康自主开发的拥有独立知识产权的SCAD™（智能化学辅助递送）递送平台，以实现中枢神经系统的高效递送。临床前药效研究表明，RAG-17的治疗能够极其显著地延缓疾病发病时间、延长动物的生存时间和改善其运动功能。

ALS是一种以上、下运动神经元损害为突出表现的慢性进行性神经系统变性疾病，主要表现为肌无力、肌肉萎缩、延髓麻痹及锥体束征。ALS是一种无法治愈的疾病，患者发病后生存期通常为3-5年。ALS可分为散发性与家族性。家族性可由多种基因的突变引起，SOD1是常见的基因之一。在中国SOD1更是导致ALS的最常见的突变基因，约20%的家族性ALS和5%的散发性ALS为SOD1基因突变所致。

The trial was led by Dr. Yilong Wang and conducted at Beijing Tiantan Hospital, one of China’s leading centers for neurological diseases. The trial enrolled six ALS-SOD1 patients and primarily focused on assessing the safety of RAG-17. Results demonstrated that RAG-17, administered intrathecally, was well-tolerated across all dose levels. All adverse events were mild. Comprehensive safety evaluations, including laboratory assessments, vital signs, and electrocardiograms, further supported the favorable safety profile.

Encouragingly, early signs of clinical benefit were also evident. Notable changes in clinical outcomes and key biomarkers indicate the efficacy of RAG-17 within this patient population. These positive clinical findings align with Ractigen’s robust preclinical data, which demonstrated significant therapeutic effects of RAG-17 in SOD1-G93A ALS mouse and rat models, including delayed disease progression and improved survival.

“These initial clinical results are truly encouraging and bring us one step closer to our goal of offering new hope to ALS patients,” stated Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. “The positive outcomes from this trial underscore the potential of RAG-17 as a disease-modifying therapy for ALS-SOD1. We are fully committed to advancing its clinical development and ultimately delivering this much-needed treatment to patients.”

This promising data will be presented at three upcoming conferences: the 27th National Conference of Neurology in China this September, followed by Neuroscience 2024 in Chicago, USA, in October, and the 35th International Symposium on ALS/MND in Montreal, Canada, in December—one of the largest annual gatherings dedicated to ALS and motor neuron disease research.

RAG-17 received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in March 2023 and Investigational New Drug (IND) was cleared for clinical trials in the U.S.. Additionally, in May 2024, the IND application was approved by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) for clinical trials in China.