ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs, announced that the U.S.

ARTHEX Biotech s.L.，一家临床阶段的生物技术公司，专注于通过调节microRNA开发创新药物，宣布

Food and Drug Administration (FDA) cleared the Company to initiate the Phase I-IIa ArthemiR™ study of ATX-01 for the treatment of Myotonic Dystrophy Type 1 (DM1). .

美国食品和药物管理局（FDA）批准该公司启动ATX-01的I-IIa期ArthemiR™研究，用于治疗1型强直性营养不良（DM1）。

ATX-01, an antimiR designed to target microRNA 23b (miR-23b), is the first microRNA therapeutic to be investigated for DM1 and is the first therapeutic from ARTHEx's pipeline to enter the clinic. miR-23b is known to be associated with regulating the expression of MBNL proteins involved in the pathogenesis of DM1, a devastating, rare neuromuscular disorder that causes muscle weakness and other life-limiting complications.

ATX-01是一种旨在靶向microRNA 23b（miR-23b）的抗miR，是第一种针对DM1进行研究的microRNA治疗剂，也是ARTHEx管道中第一种进入临床的治疗剂。已知miR-23b与调节参与DM1发病机制的MBNL蛋白的表达有关，DM1是一种破坏性的罕见神经肌肉疾病，可导致肌肉无力和其他限制生命的并发症。

There are currently no disease-modifying treatments for DM1..

目前尚无针对DM1的疾病缓解疗法。

'Receiving FDA clearance to initiate our first-in-human study, ArthemiR™, for ATX-01 in DM1 is a major milestone for ARTHEX and for DM1 patients and their families who are in need of an approved therapeutic option,' said Dr. Judith Walker, Chief Medical Officer of Arthex. 'ATX-01 holds significant potential to deliver therapeutic benefit to DM1 patients, based on its dual mechanism of action that targets both the toxic DMPK mRNA and the reduced active MBNL levels.

ARTHEX首席医疗官朱迪思·沃克（JudithWalker）博士说：“获得FDA批准，启动我们针对DM1中ATX-01的首次人体研究ArthemiR™是ARTHEX以及需要批准治疗方案的DM1患者及其家属的一个重要里程碑。”基于其靶向毒性DMPK mRNA和降低的活性MBNL水平的双重作用机制，ATX-01具有为DM1患者提供治疗益处的巨大潜力。

We plan to launch the ArthemiR™ study first in the US, followed by Canada and Europe.'.

我们计划首先在美国启动ArthemiR™研究，然后在加拿大和欧洲启动。”

The ArthemiR™ trial is a Phase I-IIa double-blind, placebo-controlled, dose escalation study expected to enroll participants with classic Myotonic Dystrophy Type 1 (DM1). The primary objective is to determine the safety and tolerability of single and multiple ascending doses of ATX-01 in DM1 participants. ARTHEx will also investigate target engagement at the muscle level through biomarkers, including MBNL levels and splicing index.

ArthemiR™试验是一项I-IIa期双盲，安慰剂对照，剂量递增研究，预计将招募经典的1型强直性肌营养不良（DM1）患者。主要目的是确定DM1参与者单次和多次递增剂量ATX-01的安全性和耐受性。ARTHEx还将通过生物标志物（包括MBNL水平和剪接指数）研究肌肉水平的目标参与。

In addition, the clinical endpoints from the trial will include measures related to muscle function, patient-reported outcomes, and quality of life measures. Dr. Nicholas Johnson, Professor, Vice Chair of Research in the Department of Neurology at Virginia Commonwealth University, and the lead investigator for the ArthemiR™ trial, commented, 'It is exciting to see new agents coming to clinical trials, especially compounds with different mechanisms of action.

此外，该试验的临床终点将包括与肌肉功能，患者报告结果和生活质量测量相关的测量。博士。弗吉尼亚联邦大学神经病学系副主任、教授兼ArthemiR™试验首席研究员尼古拉斯·约翰逊（NicholasJohnson）评论道：“看到新药进入临床试验，特别是具有不同作用机制的化合物，令人兴奋。

This increases our hope of one day having effective and safe, disease modifying treatments for this multisystemic condition. Patients are eager for new trials, and we are delighted to start enrolment in the coming months.'About ATX-01ATX-01 is an antimiR oligonucleotide designed to target microRNA 23b (miR-23b), which is associated with regulating the expression of MBNL proteins involved in the pathogenesis of DM1.

这增加了我们有一天对这种多系统疾病进行有效，安全，改善疾病的治疗的希望。患者渴望进行新的试验，我们很高兴在未来几个月开始注册。”关于ATX-01ATX-01是一种抗miR寡核苷酸，旨在靶向microRNA 23b（miR-23b），它与调节参与DM1发病机制的MBNL蛋白的表达有关。

It has been demonstrated in human DM1 myoblast cell lines that ATX-01 has a unique, dual mechanism of action which reduces toxic DMPK mRNA and increases MBNL protein levels. Toxic DMPK and reduced levels of MBNL have been identified as the molecular underpinnings of DM1. ATX-01 will shortly be evaluated in the Phase I-IIa ArthemiR™ trial for the treatment of DM1.

已经在人DM1成肌细胞系中证明，ATX-01具有独特的双重作用机制，可降低毒性DMPK mRNA并增加MBNL蛋白水平。毒性DMPK和MBNL水平降低已被确定为DM1的分子基础。ATX-01不久将在I-IIa期ArthemiR™试验中用于治疗DM1。

ATX-01 has received Orphan Drug Designation for ATX-01 in DM1 from the US and European authorities. ATX-01 was disco.

ATX-01已从美国和欧洲当局获得DM1中ATX-01的孤儿药指定。ATX-01是迪斯科舞厅。