SEOUL, South Korea--(BUSINESS WIRE)--ENCell Co., Ltd., a biotech in South Korea specializing in Cell and Gene therapy (CGT), presented promising results from their First-in-Human Phase 1 clinical trial of the novel mesenchymal stem cell therapy (EN001) for Charcot-Marie-Tooth disease type 1A (CMT1A) at the Peripheral Nerve Society (PNS) annual meeting in Montreal on June 23, 2024.

韩国首尔--（商业新闻短讯）--韩国专门从事细胞和基因治疗（CGT）的生物技术公司ENCell Co.，Ltd.在2024年6月23日于蒙特利尔举行的周围神经学会（PNS）年会上，首次在人体内进行新型间充质干细胞疗法（EN001）的1期临床试验，为1A型Charcot-Marie-Tooth病（CMT1A）提供了有希望的结果。

This annual conference is a key gathering for experts in the field, including neurologists, neurosurgeons, neuroscientists, and other healthcare professionals focused on peripheral nerve disorders..

这次年度会议是该领域专家的重要聚会，包括神经科医生、神经外科医生、神经学家和其他专注于周围神经疾病的医疗保健专业人员。。

EN001 is a next-generation allogeneic mesenchymal stem cell therapy developed using ENCell's proprietary ENCell Technology. ENCell reported EN001 can migrate to and regenerate damaged nerves and muscles through non-clinical studies. In June 2023, ENCell completed a Phase 1 clinical trial with nine CMT1A patients at Samsung Medical Center (SMC) with Professor Byung-Ok Choi from the Department of Neurology as the Principal Investigator.

EN001是使用ENCell专有的ENCell技术开发的下一代同种异体间充质干细胞疗法。ENCell报道EN001可以通过非临床研究迁移到受损的神经和肌肉并再生。2023年6月，ENCell在三星医疗中心（SMC）完成了一项针对9名CMT1A患者的1期临床试验，由神经内科的Byung Ok Choi教授担任主要研究者。

The trial utilized a dose-escalation design to ensure the safety and exploratory efficacy of EN001 over a 16-week period..

该试验采用剂量递增设计，以确保EN001在16周内的安全性和探索性疗效。。

The trial results, which were selected for oral presentation at the PNS meeting, showed no dose-limiting toxicity, serious adverse events, or infusion-related responses among participants, thereby confirming the safety of EN001. Additionally, exploratory efficacy evaluations revealed significant improvements.

在PNS会议上选择口服的试验结果显示，参与者之间没有剂量限制性毒性，严重不良事件或输注相关反应，从而证实了EN001的安全性。此外，探索性疗效评估显示显着改善。

The Charcot-Marie-Tooth Neuropathy Score version 2 decreased by an average of 2.89 points (p=0.0039) after 16 weeks, with the high-dose group showing a more pronounced reduction of 3.50 points (p=0.0313)..

16周后，Charcot-Marie-Tooth神经病评分第2版平均下降了2.89分（p=0.0039），高剂量组下降了3.50分（p=0.0313）。。

Further analysis indicated that patients receiving the high dose experienced clinical improvements in disease severity, with some moving from severe to moderate or from moderate to mild categories. Specific sensory and motor function tests also showed marked improvements, with the high-dose group. Enhancements in the 10 Meter Walking Test, Functional Disability Scale, Overall Neuropathy Limitation Score leg scale, and nerve conduction studies were also observed..

进一步的分析表明，接受高剂量治疗的患者在疾病严重程度方面取得了临床改善，其中一些患者从严重到中度或从中度到轻度。高剂量组的特定感觉和运动功能测试也显示出明显的改善。还观察到10米步行测试，功能障碍量表，整体神经病限制评分腿部量表和神经传导研究的增强。。

An ENCell spokesperson highlighted the significance of presenting these promising clinical results at a prestigious conference, emphasizing the company's dedication to developing new treatment options for CMT patients, who currently lack approved therapies. Professor Byung-Ok Choi, leading the clinical trial, expressed optimism, stating, 'These results provide hope to CMT1A patients, a group affected by a rare disease without existing treatments.' ENCell is planning to conduct repeat-administration clinical trial at SMC..

ENCell的一位发言人强调了在一次久负盛名的会议上展示这些有希望的临床结果的重要性，强调了该公司致力于为目前缺乏批准疗法的CMT患者开发新的治疗选择。领导这项临床试验的Byung Ok Choi教授表示乐观，他说：“这些结果为CMT1A患者带来了希望，CMT1A患者是一种罕见疾病的患者，没有现有的治疗方法。”恩塞尔计划在SMC进行重复给药临床试验。。