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– Company on track to initiate ELEVATE-44-201 in Q2 2025 –

–公司有望在2025年第二季度启动ELEVATE-44-201–

– ENTR-601-44 regulatory filings submitted in additional geographies including the U.S. and EU, with regulatory discussions ongoing –

–ENTR-601-44在包括美国和欧盟在内的其他地区提交的监管文件，监管讨论正在进行中–

BOSTON, Feb. 03, 2025 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA) today announced it had received authorization from the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee for its Clinical Trial of an Investigational Medicinal Product to initiate ELEVATE-44-201, a Phase 1/2 multiple ascending dose (MAD) clinical study of ENTR-601-44 for the potential treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the .

波士顿，2025年2月3日（环球通讯社）--Entrada Therapeutics，Inc.（纳斯达克：TRDA）今天宣布，它已获得英国药品和保健品管理局（MHRA）和研究伦理委员会的授权，用于临床试验一种研究性药品，以启动ELEVATE-44-201，这是一项ENTR-601-44的1/2期多重递增剂量（MAD）临床研究，用于治疗确诊突变患者的杜兴氏肌营养不良症（DMD）。

DMD

DMD公司

gene amenable to exon 44 skipping.

。

“The clearance by the MHRA marks a new phase in Entrada’s growth and, most importantly, moves us closer to realizing our commitment to families living with Duchenne muscular dystrophy,” said Dipal Doshi, Chief Executive Officer of Entrada Therapeutics. “As the first authorization for our global MAD clinical study of ENTR-601-44 in patients, we are pleased to be initiating the study at what we believe to be an effective therapeutic dose.

Entrada Therapeutics首席执行官迪帕尔·多西（DipalDoshi）表示：“MHRA的批准标志着Entrada发展进入了一个新阶段，最重要的是，它使我们更接近实现对患有杜兴氏肌营养不良症的家庭的承诺。”。“作为我们对患者进行ENTR-601-44全球MAD临床研究的首次授权，我们很高兴以我们认为有效的治疗剂量开始这项研究。

This is even more important since families living with Duchenne do not have time on their side as the progressive decline in function profoundly impacts the quality of life for patients and their care partners. It is this urgency that drives our work each day.”.

这一点更为重要，因为与杜兴生活在一起的家庭没有时间站在他们一边，因为功能的逐渐下降深刻地影响了患者及其护理伙伴的生活质量。正是这种紧迫感推动着我们每天的工作。”。

“The MHRA authorization of ELEVATE-44-201 is an exciting development in the clinical progress of ENTR-601-44, a new and very encouraging treatment option for boys and young men living with Duchenne muscular dystrophy who are exon 44 skipping amenable,” said Francesco Muntoni, MD, Professor of Paediatric Neurology.

儿科神经病学教授弗朗西斯科·蒙托尼（Francesco Muntoni）医学博士说：“MHRA对ELEVATE-44-201的授权是ENTR-601-44临床进展的一个令人兴奋的发展，ENTR-601-44是一种新的非常令人鼓舞的治疗选择，适用于患有杜兴氏肌营养不良症的男孩和年轻男性，他们可以跳过第44外显子。”。

“There is a significant unmet medical need in this population, with limited therapeutic options available. The unique properties of Entrada’s EEV-therapeutic candidates offer the potential to provide tangible benefits for people with this life-shortening disease.”.

“这一人群的医疗需求尚未得到满足，可用的治疗选择有限。恩特拉达EEV治疗候选药物的独特特性为这种缩短寿命的疾病患者提供了切实的益处。”。

ELEVATE-44-201 is a global, two-part, randomized, double-blind placebo-controlled Phase 1/2 study evaluating the safety, tolerability and effectiveness of ENTR-601-44 in ambulatory patients with DMD who are exon 44 skipping amenable. Part A is a multiple ascending dose study designed to evaluate the safety, pharmacokinetics, and pharmacodynamics, including exon skipping and dystrophin production in approximately 24 patients.

ELEVATE-44-201是一项全球性，两部分，随机，双盲安慰剂对照的1/2期研究，评估了ENTR-601-44在DMD门诊患者中的安全性，耐受性和有效性，这些患者的外显子44跳过是合适的。A部分是一项多剂量递增剂量研究，旨在评估大约24名患者的安全性，药代动力学和药效学，包括外显子跳跃和肌营养不良蛋白产生。

Dosing will be administered every six weeks, with the planned doses across three cohorts anticipated to range from 6 mg/kg up to 18 mg/kg. Part B of the study is designed to further evaluate the optimal dose established in Part A for safety and efficacy, including patient reported outcomes and quality of life measures.

剂量将每六周给药一次，预计三个队列的计划剂量范围为6 mg/kg至18 mg/kg。该研究的B部分旨在进一步评估A部分确定的最佳剂量的安全性和有效性，包括患者报告的结果和生活质量测量。

Study participants may be eligible to enter an open label extension study (OLE), in which the safety, efficacy and tolerability of ENTR-601-44 will be evaluated over a longer period of time. The Company is on track to initiate ELEVATE-44-201 in Q2 2025..

研究参与者可能有资格进入开放标签扩展研究（OLE），其中将在更长的时间内评估ENTR-601-44的安全性，有效性和耐受性。该公司有望在2025年第二季度启动ELEVATE-44-201。。

The MHRA authorization follows the completion of a Phase 1 clinical study to evaluate the safety and tolerability of a single dose of ENTR-601-44. This study demonstrated ENTR-601-44 was generally well-tolerated in healthy volunteers with no serious adverse events, no drug-related adverse events and no clinically significant changes or trends noted in vital signs, electrocardiograms, physical exams or laboratory assessments.

MHRA授权是在完成第一阶段临床研究以评估单剂量ENTR-601-44的安全性和耐受性之后进行的。这项研究表明，健康志愿者对ENTR-601-44的耐受性通常良好，没有严重的不良事件，没有药物相关的不良事件，在生命体征，心电图，体格检查或实验室评估中没有发现临床上显着的变化或趋势。

The study also demonstrated significant plasma concentration, muscle concentration and exon skipping..

该研究还表明血浆浓度，肌肉浓度和外显子跳跃显着。。

About ENTR-601-44

关于ENTR-601-44

ENTR-601-44, a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), is the lead product candidate within Entrada’s Duchenne muscular dystrophy franchise from its growing pipeline of EEV-therapeutics. Each EEV-PMO therapeutic candidate has an oligonucleotide sequence designed and optimized for the specific subpopulation of interest.

ENTR-601-44是一种专有的内体逃逸载体（EEV™）偶联的磷酸二酰胺吗啉代低聚物（PMO），是Entrada的Duchenne肌营养不良症特许经营权中的主要候选产品，来自其不断增长的EEV治疗渠道。每个EEV-PMO治疗候选物都有一个针对特定感兴趣的亚群设计和优化的寡核苷酸序列。

ENTR-601-44 is designed to address the underlying cause of Duchenne due to mutated or missing exons in the .

ENTR-601-44旨在解决由于突变或缺失外显子而导致Duchenne的根本原因。

DMD

DMD公司

gene. ENTR-601-44, an investigational therapy for the potential treatment of people living with Duchenne who are exon 44 skipping amenable, is being evaluated for its potential to restore the mRNA reading frame and allow for the translation of dystrophin protein that is slightly shortened but still functional..

基因。ENTR-601-441-441-441-441-441-6011-441-441-6011-441-6011-6011-441-6011-6011-6011-6011-6011-6011-6011-6011-6011-6014-6011-6011-6014-6011-6011-6011-444-6011-6011-6011-6011-6011-444-6011。。

About Duchenne Muscular Dystrophy (DMD)

关于杜兴氏肌营养不良症（DMD）

Duchenne muscular dystrophy (DMD) is a rare disease caused by mutations in the

DMD

DMD公司

gene, which encodes for the dystrophin protein. These mutations lead to inadequate dystrophin production. Dystrophin is essential to maintaining the structural integrity and function of muscle cells. Lack of functional dystrophin leads to progressive loss of muscle strength, impacting mobility and causing heart or respiratory complications that contribute to high mortality rates.

编码肌营养不良蛋白的基因。这些突变导致肌营养不良蛋白产生不足。肌营养不良蛋白对于维持肌肉细胞的结构完整性和功能至关重要。缺乏功能性肌营养不良蛋白会导致肌肉力量逐渐丧失，影响活动能力，并导致心脏或呼吸系统并发症，从而导致高死亡率。

An estimated 41,000 people in the U.S. and Europe have Duchenne..

据估计，美国和欧洲有41000人患有杜兴氏综合症。。

About Entrada Therapeutics

关于Entrada Therapeutics

Entrada Therapeutics is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines that engage intracellular targets that have long been considered inaccessible. The Company’s Endosomal Escape Vehicle (EEV™)-therapeutics are designed to enable the efficient intracellular delivery of a wide range of therapeutics into a variety of organs and tissues, resulting in an improved therapeutic index.

Entrada Therapeutics是一家临床阶段的生物制药公司，旨在通过建立一类新的药物来改变患者的生活，这些药物涉及长期以来被认为无法获得的细胞内靶标。该公司的内体逃逸载体（EEV™）治疗剂旨在将多种治疗剂有效地细胞内递送到各种器官和组织中，从而提高治疗指数。

Through this proprietary, versatile and modular approach, Entrada is advancing a robust development portfolio of RNA-, antibody- and enzyme-based programs for the potential treatment of neuromuscular, ocular, metabolic and immunological diseases, among others. The Company’s lead oligonucleotide programs are in development for the potential treatment of people living with Duchenne who are exon 44, 45 and 50 skipping amenable.

通过这种专有的，多功能的和模块化的方法，Entrada正在推进一个强大的基于RNA，抗体和酶的程序开发组合，用于潜在治疗神经肌肉，眼部，代谢和免疫疾病等。该公司的领先寡核苷酸计划正在开发中，用于治疗外显子44、45和50跳过的Duchenne患者。

Entrada has partnered to develop a clinical-stage program, VX-670, for myotonic dystrophy type 1..

Entrada合作开发了一个临床阶段计划VX-670，用于1型强直性营养不良。。

For more information about Entrada, please visit our website,

有关Entrada的更多信息，请访问我们的网站，

www.entradatx.com

网站：www.entradatx.com

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Forward-Looking Statements

前瞻性声明

This press release contains express and implied forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Entrada’s strategy, future operations, prospects and plans, objectives of management, the validation and differentiation of Entrada’s approach and EEV platform and its ability to provide a potential treatment for patients, expectations regarding Entrada’s planned Phase 1/2 multiple ascending dose clinical study ENTR-601-44, including its initiation in the United Kingdom in Q2 2025, the expectations about the planned dosing levels of the planned Phase 1/2 trial for ENTR-601-44 and their efficacy, the ability to recruit for and complete a global Phase 1/2 trial for ENTR-601-44 and the anticipated number of patients to be enrolled, the potential of Entrada’s EEV product candidates, including the potential for ENTR-601-44 to be a transformative treatment option, and the continued development and advancement of ENTR-601-44 for the potential treatment of DMD, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995.

本新闻稿包含涉及重大风险和不确定性的明示和暗示的前瞻性声明。除历史事实陈述外，本新闻稿中包含的所有陈述，包括关于Entrada的战略、未来运营、前景和计划、管理目标、Entrada方法和EEV平台的验证和区别以及为患者提供潜在治疗的能力的陈述，对Entrada计划的1/2期多剂量递增临床研究ENTR-601-44的期望，包括其于2025年第二季度在英国的启动，对ENTR-601-44计划的1/2期试验的计划剂量水平及其疗效的期望，招募和完成ENTR-601-44全球1/2期试验的能力以及预期登记的患者人数，Entrada EEV候选产品的潜力，包括ENTR-601-44成为变革性治疗选择的潜力，以及ENTR-601-44在DMD潜在治疗中的持续发展和进步，构成1995年《私人证券诉讼改革法案》所指的前瞻性声明。

The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” or “would,” or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

“预期”，“相信”，“继续”，“可能”，“估计”，“预期”，“打算”，“可能”，“可能”，“目标”，“正在进行”，“计划”，“预测”，“项目”，“潜在”，“应该”或“会”，或这些术语的否定词或其他类似术语旨在识别前瞻性陈述，尽管并非所有前瞻性陈述都包含这些识别词。

Entrada may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking st.

Entrada可能无法实际实现这些前瞻性声明中披露的计划、意图或期望，您不应过度依赖这些前瞻性st。

Investor and Media Contact

投资者和媒体联系

Caileigh Dougherty

失去 Dougherty

Head of Investor Relations & Corporate Communications

投资者关系与企业沟通主管

cdougherty@entradatx.com

cdougherty@entradatx.com