PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-DM1, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy..

加利福尼亚州帕萨迪纳-（商业线）-Arrowhead Pharmaceuticals Inc.（纳斯达克股票代码：ARWR）今天宣布已申请批准启动ARO-DM1的1/2a期临床试验，该公司的研究RNA干扰（RNAi）治疗药物正在开发中，作为最常见的成人型肌营养不良症1型肌强直性营养不良症（DM1）的潜在治疗方法。。

ARO-DM1 is designed to reduce expression of the dystrophia myotonica protein kinase (DMPK) gene. There is currently no approved disease-modifying therapy for DM1. Treatments have focused on symptomatic management, including physical therapy, exercise, ankle-foot orthoses, wheelchairs, and other assistive devices..

ARO-DM1旨在减少肌营养不良症肌强直蛋白激酶（DMPK）基因的表达。目前尚无批准的DM1疾病缓解疗法。治疗的重点是对症治疗，包括物理治疗，运动，踝足矫形器，轮椅和其他辅助设备。。

James Hamilton, M.D., MBA, Chief of Discovery and Translational Medicine at Arrowhead, said: “ARO-DM1 is Arrowhead’s second clinical candidate utilizing our TRiM™ platform to deliver RNAi therapeutics to skeletal muscle. Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities and may become physically disabled and have a shortened life expectancy.

Arrowhead发现与转化医学总监James Hamilton博士说：“ARO-DM1是Arrowhead利用我们的修剪技术的第二位临床候选人™ 向骨骼肌提供RNAi疗法的平台。DM1患者有肌肉无力和消瘦，肌强直，白内障，并经常发展为心脏传导异常，可能会身体残疾，预期寿命缩短。

ARO-DM1 represents a novel approach to treat DM1 by silencing aberrantly transcribed DMPK mRNA, which could lead to improvements in multiple symptoms, including muscle strength and function.”.

ARO-DM1代表了一种通过沉默异常转录的DMPK mRNA来治疗DM1的新方法，其可以导致多种症状的改善，包括肌肉力量和功能。

An application for approval of the clinical trial was submitted to a local Ethics Committee and to the New Zealand Medicines and Medical Devices Safety Authority for review by the Standing Committee on Therapeutic Trials. Pending clearance, Arrowhead intends to proceed with ARODM1-1001, a Phase 1/2a dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-DM1 in up to 48 subjects with DM1..

批准临床试验的申请已提交给当地伦理委员会和新西兰药品和医疗器械安全局，由治疗试验常设委员会审查。在清除之前，Arrowhead打算继续进行ARODM1-1001，这是一项1/2a期剂量递增研究，旨在评估ARO-DM1在多达48名DM1受试者中的安全性，耐受性，药代动力学和药效学。。

About Arrowhead Pharmaceuticals

关于箭头制药

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes.

箭头药物通过沉默导致它们的基因来开发治疗难治性疾病的药物。箭头疗法利用广泛的RNA化学和有效的递送模式，触发RNA干扰机制，诱导靶基因的快速，深度和持久的敲低。

RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing..

RNA干扰或RNAi是存在于活细胞中的机制，其抑制特定基因的表达，从而影响特定蛋白质的产生。Arrowhead基于RNAi的疗法利用这种基因沉默的自然途径。。

For more information, please visit www.arrowheadpharma.com, or follow us on Twitter @ArrowheadPharma. To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.

欲了解更多信息，请访问www.arrowheadpharma.com，或在Twitter@arrowheadpharma上关注我们。要添加到公司的电子邮件列表中并直接接收新闻，请访问http://ir.arrowheadpharma.com/email-alerts.

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此外，任何涉及我们未来财务绩效预测，业务趋势，对我们产品线或产品候选人的期望的声明，包括预期的监管提交和临床计划结果，我们与其他公司合作的前景或收益，或对未来事件或情况的其他描述是前瞻性陈述。

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这些前瞻性陈述包括但不限于关于我们的临床前研究和临床试验以及我们的研究和开发计划的启动，时间，进展和结果的陈述；我们对合伙关系，许可和/或合作安排以及我们已签订或可能签订的其他战略安排和交易的潜在利益的期望；我们对现有协议下可能由第三方或第三方支付的里程碑，版税或其他付款的信念和期望；以及我们对未来收入，研发费用，资本要求和向第三方支付的估计。

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这些陈述是以u为基础的。

Source: Arrowhead Pharmaceuticals, Inc.

来源：Arrowhead Pharmaceuticals，Inc。