Company will conduct Phase 3 trial of AMO-02 (tideglusib) in adults to support a future submission for marketing authorization in Type 1 myotonic dystrophy in both adult and pediatric indications.

该公司将在成人中进行AMO-02（tideglusib）的3期临床试验，以支持未来在成人和儿科适应症中提交1型强直性肌营养不良的上市授权。

LONDON, May 2, 2024 /PRNewswire/ -- AMO Pharma Limited ('AMO Pharma'), a privately held clinical-stage specialty biopharmaceutical company focusing on rare genetic disorders with limited or no treatment options, today provided an update on plans to advance the clinical development program for AMO-02 (tideglusib), the company's investigational therapy in development for the treatment of Type 1 myotonic dystrophy (DM1), following a recent meeting with the U.S.

伦敦，2024年5月2日/PRNewswire/-AMO Pharma Limited（“AMO Pharma”）是一家私营的临床阶段专业生物制药公司，专注于罕见的遗传性疾病，治疗选择有限或没有治疗选择，今天提供了一份最新计划，以推进AMO-02（tideglusib）的临床开发计划，该公司最近与美国会面后，正在开发用于治疗1型强直性营养不良（DM1）的研究疗法。

Food and Drug Administration (FDA). Based on the input from the meeting, AMO Pharma will conduct a Phase 3 clinical trial to assess the efficacy and safety of AMO-02 in adult patients with adult-onset DM1 to support a future submission for marketing authorization for the treatment of DM1 in children, adolescents and adults..

美国食品和药物管理局（FDA）。根据会议的意见，AMO Pharma将进行一项3期临床试验，以评估AMO-02在成人发病DM1的成年患者中的疗效和安全性，以支持未来提交用于儿童，青少年和成人DM1治疗的上市授权。。

Results from AMO Pharma's previous REACH-CDM Phase 2/3 clinical study (the 'REACH-CDM Study') involving children and adolescents with congenital-onset DM1 showed that AMO-02 was generally safe and well tolerated during the study, with no reported severe adverse events (SAEs) related to treatment. The primary and secondary outcome measures based on clinician administered subjective ratings showed an unexpected placebo effect that may have masked some beneficial effects of treatment.

AMO Pharma之前的REACH-CDM 2/3期临床研究（“REACH-CDM研究”）涉及先天性DM1患儿和青少年的结果显示，AMO-02在研究期间通常是安全且耐受性良好的，没有报告与治疗相关的严重不良事件（SAE）。基于临床医生主观评分的主要和次要结局指标显示出意想不到的安慰剂效应，这可能掩盖了治疗的一些有益效果。

Further post hoc analysis as reviewed with the FDA indicates that there is sufficient evidence to support the continued development of tideglusib for the treatment of DM1 with agreement that AMO Pharma will now advance a Phase 3 study in adults with the adult-onset version of DM1..

FDA审查的进一步事后分析表明，有足够的证据支持tideglusib治疗DM1的持续发展，并同意AMO Pharma现在将推进成人发病版本DM1的3期研究。。

During the meeting, FDA reviewed data from the REACH-CDM Study to assess the efficacy and safety of treatment with AMO-02 in children and adolescents with congenital DM1. The FDA also gave feedback on the design of the new Phase 3 clinical trial for adult patients with adult onset DM1. In this study in adult onset DM1, outcome measures will be similar to those that showed benefit in the REACH-CDM Study.

会议期间，FDA审查了REACH-CDM研究的数据，以评估AMO-02治疗先天性DM1儿童和青少年的疗效和安全性。FDA还对成人发病DM1的成年患者新的3期临床试验的设计提供了反馈。在这项针对成人发病DM1的研究中，结果指标将类似于REACH-CDM研究中显示出益处的指标。

Following discussion with the FDA, AMO Pharma will plan a future submission for approval in children, adolescents and adults with myotonic dystrophy based on a package of safety and efficacy data from the new Phase 3 trial in adults combined with efficacy, pharmacokinetic and safety data from AMO Pharma's REACH-CDM Study..

在与FDA讨论后，AMO Pharma将根据成人新的3期试验的安全性和有效性数据包，结合AMO Pharma REACH-CDM研究的有效性，药代动力学和安全性数据，计划未来提交儿童，青少年和成人强直性肌营养不良的批准。。

'We are pleased with the outcome of the productive discussions we had with the FDA regarding next steps for AMO-02 and continue to feel strongly that this innovative therapy can represent a significant advance in the treatment of patients with DM1, whether they are children, adolescents or adults,' said Alan Rubino, executive chair at AMO Pharma.

AMO Pharma执行主席艾伦·鲁比诺（AlanRubino）说：“我们对与FDA就AMO-02的下一步进行的富有成效的讨论的结果感到高兴，并继续强烈感到，这种创新疗法可以代表DM1患者治疗的重大进步，无论他们是儿童，青少年还是成年人。”。

'We are now focused on progressing a new clinical study in adults with adult-onset DM1. We are very grateful to the FDA and to the patients, families, clinicians and investors who have supported our research thus far and we look forward to working with the community to advance this program as rapidly as possible.

“我们现在专注于在成人发病DM1的成年人中进行一项新的临床研究。我们非常感谢FDA以及迄今为止支持我们研究的患者，家属，临床医生和投资者，我们期待着与社区合作，尽快推进这一计划。

We will make an announcement regarding the start date and site locations for the adult onset DM1 study shortly.'.

我们将很快宣布成人发病DM1研究的开始日期和地点。”。

'The results of the REACH-CDM Study showed measures of efficacy benefit across multiple symptom areas, and we are very excited that this clinical development program will continue to move forward,' said Hanns Lochmuller, investigator in the REACH-CDM Study at Children's Hospital of Eastern Ontario, Canada.

加拿大安大略省东部儿童医院REACH-CDM研究的研究员汉斯·洛赫穆勒（Hanns Lochmuller）说：“REACH-CDM研究的结果显示，多种症状领域的疗效获益指标，我们非常高兴这一临床开发计划将继续取得进展。”。

'We remain very hopeful that this investigational therapy can have a transformative impact on the health of patients living with this ultra-rare, severe muscular dystrophy in the years ahead.'.

“我们仍然非常希望这种研究性治疗能够在未来几年对患有这种极为罕见的严重肌营养不良症的患者的健康产生变革性影响。”。

About AMO-02

关于AMO-02

AMO-02 (tideglusib) is an investigational therapy in development for the treatment of DM1 with a dual mechanism disrupting the pathogenic RNA repeat in DM1and inhibiting excess levels of the kinase GSK3β.

AMO-02（tideglusib）是一种正在开发的用于治疗DM1的研究性疗法，具有双重机制，可破坏DM1中的致病性RNA重复序列并抑制激酶GSK3β的过量水平。

The REACH-CDM Study was a double-blind, placebo-controlled, randomized study in children and adolescents with congenital DM1 intended to support a future submission for marketing authorization in congenital DM1. The study included 56 participants at leading specialist sites in the US, Canada, New Zealand and Australia..

REACH-CDM研究是一项针对先天性DM1儿童和青少年的双盲，安慰剂对照，随机研究，旨在支持未来提交先天性DM1的上市授权。这项研究包括美国、加拿大、新西兰和澳大利亚领先专业网站的56名参与者。。

Data associated with functional and objective measures were collected during the study, including motor and muscle assessments (10 meter walk/run, myometry, lip strength and lean muscle mass as assessed by DXA scan), cognitive performance (Peabody Picture Vocabulary Test, NIH Toolbox Picture Sequence Memory Test, NIH Toolbox Dimensional Change Card Sort Test), adaptive (real world) function (Vineland Adaptive Behavior Communication Scale, Vineland Adaptive Behavior Daily Living Scale, Vineland Adaptive Behavior Socialization Scale), bone density (DXA scan) and creatine kinase measurements.

在研究期间收集了与功能和客观测量相关的数据，包括运动和肌肉评估（通过DXA扫描评估的10米步行/跑步，肌肉测量，嘴唇力量和瘦肌肉质量），认知表现（皮博迪图片词汇测试，NIH工具箱图片序列记忆测试，NIH工具箱尺寸变化卡分类测试），适应性（现实世界）功能（Vineland适应性行为沟通量表，Vineland适应性行为日常生活量表，Vineland适应性行为社会化量表），骨密度（DXA扫描）和肌酸激酶测量。

In post-hoc analyses more participants showed a positive response following AMO-02 treatment than placebo on 10 of the 12 of these measures. Treatment with AMO-02 was associated with clinically significant improvements in walking and cognition and significant effects on objective biomarkers of muscle and neuronal integrity.

在事后分析中，在12项措施中的10项中，更多的参与者在AMO-02治疗后比安慰剂显示出积极的反应。AMO-02治疗与步行和认知的临床显着改善以及对肌肉和神经元完整性的客观生物标志物的显着影响相关。

AMO-02 also showed significant improvement compared to placebo on a Multi-Domain Responder Index integrating clinically significant responses on measures of ambulation, muscle strength, cognitive performance, activities of daily living and an objective biomarker. Primary analyses of subjective rating assessments showed a placebo effect.

与安慰剂相比，AMO-02在多领域反应者指数方面也显示出显着改善，该指数综合了对步行，肌肉力量，认知表现，日常生活活动和客观生物标志物的临床显着反应。主观评分评估的初步分析显示安慰剂效应。

Patients from this study and treatment naïve patients with congenital or juvenile onset DM1 are currently being supported in the REACH CDM-X open label extension safety study..

这项研究的患者和未接受治疗的先天性或青少年发病DM1患者目前正在REACH CDM-X开放标签扩展安全性研究中得到支持。。

About AMO Pharma

关于AMO制药

AMO Pharma is a clinical-stage specialty biopharmaceutical company working to identify and advance promising therapies for the treatment of serious and debilitating diseases in patient populations with significant areas of unmet need, including rare and severe childhood onset neurogenetic disorders with limited or no treatment options.

AMO Pharma是一家临床阶段专业生物制药公司，致力于确定和推进有希望的治疗方法，用于治疗严重和衰弱的患者群体，这些患者群体的需求尚未得到满足，包括罕见和严重的儿童期神经遗传疾病，治疗选择有限或没有。

In addition to developing AMO-02 for DM1, the company is also progressing AMO-01 as a clinical stage treatment for Phelan-McDermid syndrome and AMO-04 as a clinic-ready potential medicine for Rett syndrome and related disorders. AMO-02, AMO-01 and AMO-04 are investigational medicines that have not yet been approved for the treatment of patients anywhere in the world.

除了为DM1开发AMO-02外，该公司还正在开发AMO-01作为Phelan-McDermid综合征的临床阶段治疗药物，以及AMO-04作为Rett综合征和相关疾病的临床就绪潜在药物。AMO-02，AMO-01和AMO-04是尚未被批准用于治疗世界各地患者的研究药物。

Advice provided to AMO Pharma by regulators is under the condition that any scientific advice given is not legally binding with regards to any future application for the product concerned. Furthermore, advice cannot be taken as indicative of any future agreed position..

监管机构向AMO Pharma提供的建议的条件是，所提供的任何科学建议对于相关产品的任何未来应用均不具有法律约束力。此外，建议不能被视为任何未来商定立场的指示。。

For more information, please visit the AMO Pharma website at http://www.amo-pharma.com/.

有关更多信息，请访问AMO Pharma网站http://www.amo-pharma.com/.

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SOURCE AMO Pharma Limited

SOURCE AMO Pharma Limited