– Sarepta obtains exclusive worldwide licenses to four clinical-stage and three preclinical-stage programs in muscle, central nervous system, and rare pulmonary disorders, including potential best-in-class siRNA-based treatments for DM1 and FSHD

–Sarepta获得了在肌肉、中枢神经系统和罕见肺部疾病方面的四个临床阶段和三个临床前阶段项目的全球独家许可证，包括针对DM1和FSHD的潜在最佳基于siRNA的治疗

– Additionally, Arrowhead and Sarepta have entered into a discovery partnership pursuant to which Sarepta will nominate, and Arrowhead will deliver, IND-ready constructs for six targets across skeletal muscle, cardiac, and CNS

–此外，Arrowhead和Sarepta已经建立了一个发现伙伴关系，根据该伙伴关系，Sarepta将提名，Arrowhead将为骨骼肌、心脏和中枢神经系统的六个目标提供IND-ready构建体

– Investigational treatments leverage Arrowhead’s leading Targeted RNAi Molecule (TRiMTM) platform, capable of deep and durable target-gene knockdown

–研究性治疗利用Arrowhead领先的靶向RNAi分子（TRiMTM）平台，能够深度持久地敲除靶基因

– Upon closing, Arrowhead to receive $500 million in an upfront payment and $325 million equity investment at a 35% premium, plus additional future milestone payments and royalties

–交割后，Arrowhead将收到5亿美元的预付款和3.25亿美元的股权投资，溢价为35%，再加上未来的里程碑付款和版税

– Separately, Sarepta’s Board of Directors has approved a share repurchase authorization of up to $500 million

–另外，萨雷塔董事会已批准最高5亿美元的股份回购授权

– Sarepta to host an investor conference call on Tuesday, Nov. 26, 2024, at 8:30 a.m. ET

–萨雷塔将于2024年11月26日星期二上午8:30主持投资者电话会议

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Nov. 26, 2024--

马萨诸塞州剑桥市--（商业新闻短讯）--2024年11月26日--

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced an exclusive global licensing and collaboration agreement with Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR). Sarepta will obtain exclusive global rights to multiple clinical, preclinical, and discovery-stage programs for rare, genetic diseases of the muscle, central nervous system (CNS), and the lungs..

Sarepta Therapeutics，Inc.（纳斯达克股票代码：SRPT）是罕见疾病精密遗传医学的领导者，今天宣布与Arrowhead Pharmaceuticals，Inc.（纳斯达克股票代码：ARWR）达成独家全球许可和合作协议。Sarepta将获得针对肌肉、中枢神经系统（CNS）和肺部罕见遗传疾病的多项临床、临床前和发现阶段计划的全球独家权利。。

The agreement will add meaningfully to Sarepta’s mid- and early-stage pipeline, complementing the Company’s existing leadership in Duchenne muscular dystrophy and limb-girdle muscular dystrophies and gene therapy, while adding new indications and expanding into adjacent therapeutic areas. In addition, Doug Ingram, president and chief executive officer, Sarepta, will be appointed to Arrowhead’s Board of Directors..

该协议将为Sarepta的中期和早期项目增加有意义的内容，补充该公司在杜兴氏肌营养不良症和肢带型肌营养不良症以及基因治疗方面的现有领导地位，同时增加新的适应症并扩展到邻近的治疗领域。此外，萨雷塔总裁兼首席执行官道格·英格拉姆（DougIngram）将被任命为箭头的董事会成员。。

The clinical-stage programs covered under the agreement include:

该协议涵盖的临床阶段计划包括：

ARO-DUX4​: designed to reduce the production of human double homeobox 4 (DUX4) protein in skeletal muscle; currently in a Phase 1/2 clinical study for the treatment of facioscapulohumeral muscular dystrophy (FSHD)

ARO-DUX4：旨在减少骨骼肌中人双同源框4（DUX4）蛋白的产生；目前正在进行面肩肱型肌营养不良症（FSHD）治疗的1/2期临床研究

ARO-DM1: designed to target and suppress myotonic dystrophy protein kinase (DMPK) in skeletal muscle; Phase 1/2 clinical study for myotonic dystrophy type 1 (DM1)​

ARO-DM1：旨在靶向和抑制骨骼肌中的强直性营养不良蛋白激酶（DMPK）；1型强直性营养不良（DM1）的1/2期临床研究

ARO-MMP7: ​​designed to reduce expression of matrix metalloproteinase 7 (MMP7) in pulmonary epithelial cells; Phase 1/2 clinical study for idiopathic pulmonary fibrosis​ (IPF)

ARO-MMP7：旨在降低肺上皮细胞中基质金属蛋白酶7（MMP7）的表达；特发性肺纤维化（IPF）的1/2期临床研究

ARO-ATXN2​: designed to target the ataxin-2 protein (ATXN2) in the CNS; expected to begin Phase 1/2 clinical study for spinocerebellar ataxia 2 (SCA2)​ by the end of 2024

ARO-ATXN2：旨在靶向中枢神经系统中的ataxin-2蛋白（ATXN2）；预计到2024年底开始脊髓小脑共济失调2（SCA2）的1/2期临床研究

The clinical programs use Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform, which is designed to deliver siRNA to multiple tissue and cell types throughout the body to initiate the RNA interference mechanism and induce rapid and durable knockdown of target genes.

临床程序使用Arrowhead专有的靶向RNAi分子（TRiMTM）平台，该平台旨在将siRNA传递至全身多种组织和细胞类型，以启动RNA干扰机制并诱导靶基因的快速持久敲低。

The preclinical programs covered under the agreement will leverage Arrowhead’s TRiM CNS delivery platform designed for subcutaneous administration and include:

该协议涵盖的临床前计划将利用Arrowhead为皮下给药设计的TRiM CNS递送平台，包括：

ARO-ATXN1: designed to target the ataxin-1 protein (ATXN1) for SCA1

ARO-ATXN1：旨在针对SCA1的ataxin-1蛋白（ATXN1）

ARO-ATXN3: designed to target the ataxin-3 protein (ATXN3) for SCA3

ARO-ATXN3：设计用于针对SCA3的ataxin-3蛋白（ATXN3）

ARO-HTT: designed to target huntingtin (HTT), a gene linked to Huntington's disease

ARO-HTT：旨在靶向亨廷顿蛋白（HTT），一种与亨廷顿舞蹈病相关的基因

Additionally, Sarepta and Arrowhead have entered into a discovery collaboration for up to six additional muscle, cardiac, and/or CNS targets, using Arrowhead’s novel delivery technologies. As part of the collaboration, Sarepta has an exclusive license to Arrowhead’s technology to develop therapeutics against a broad range of skeletal muscle gene targets..

此外，Sarepta和Arrowhead已经使用Arrowhead的新型递送技术为多达六个额外的肌肉，心脏和/或中枢神经系统靶标进行了发现合作。作为合作的一部分，Sarepta拥有Arrowhead技术的独家许可证，可以开发针对多种骨骼肌基因靶标的治疗方法。。

“With the launch of Elevidys going exceedingly well, this broad siRNA collaboration with Arrowhead provides a synergistic platform to complement Sarepta’s gene therapy and gene editing engine. Through a strategic deployment of capital, we are able to access Arrowhead’s leading RNAi platform and will work to rapidly advance new treatments for devastating genetic diseases where there is significant unmet need.

“随着Elevidys的推出进展非常顺利，与Arrowhead的这种广泛的siRNA合作提供了一个协同平台，可以补充Sarepta的基因治疗和基因编辑引擎。通过资本的战略性部署，我们能够访问Arrowhead领先的RNAi平台，并将致力于快速推进针对严重未满足需求的毁灭性遗传疾病的新治疗方法。

The agreement affords multiple potential blockbuster opportunities, serves our strategic priorities for the remainder of the decade and beyond, and diversifies our business model across one-time therapies and chronic treatments allowing for long-term growth and success. Given the strength of our performance and ability to generate substantial cash to invest in our business over the next several years, Sarepta’s Board of Directors has approved a $500 million share repurchase program as part of our overall capital allocation strategy,” said Mr.

该协议提供了多个潜在的重磅商机，为我们未来十年及以后的战略重点服务，并使我们的商业模式多样化，涵盖一次性治疗和慢性治疗，从而实现长期增长和成功。鉴于我们的业绩优势和在未来几年产生大量现金投资业务的能力，萨雷塔董事会批准了一项5亿美元的股份回购计划，作为我们整体资本配置战略的一部分。

Ingram. “We look forward to embarking on this partnership with Arrowhead, having been impressed with their scientific capabilities in developing a potentially best-in-class approach to siRNA and the quality of the team they have built. Over the course of the next 12-18 months, we expect to share multiple data readouts from across our pipeline.”.

英格拉姆。“我们期待着与Arrowhead建立这种合作关系，他们在开发潜在的最佳siRNA方法方面的科学能力以及他们建立的团队的质量给我们留下了深刻的印象。在接下来的12-18个月内，我们预计将分享我们管道中的多个数据读数。”。

“We welcome the Sarepta team as new Arrowhead collaboration partners who bring a wealth of clinical, regulatory, and commercial expertise in key areas outside of our cardiometabolic focus. We see our TRiM platform as a broad and elegant solution for delivery of siRNA to multiple cell types throughout the body.

“我们欢迎Sarepta团队成为新的箭头合作伙伴，他们在我们的心脏代谢重点之外的关键领域带来了丰富的临床，监管和商业专业知识。我们认为TRiM平台是一种广泛而优雅的解决方案，可将siRNA传递到全身多种细胞类型。

We also have a very efficient drug discovery engine that continues to generate many promising programs, and we have great confidence in Sarepta’s ability to take the next steps to advance and commercialize multiple Arrowhead-discovered drug candidates, which we believe have the potential to be best-in-class,” said Chris Anzalone, Ph.D., president and CEO at Arrowhead.

我们还有一个非常有效的药物发现引擎，它将继续产生许多有前途的项目，我们对Sarepta采取下一步行动推进多种箭头发现的候选药物并将其商业化的能力充满信心，我们相信这些候选药物有可能成为同类中最好的，”箭头总裁兼首席执行官克里斯·安佐隆博士说。

“At the close of this agreement, Doug Ingram will be appointed to the Arrowhead board of directors. He has led Sarepta as they advanced multiple investigational medicines through the clinical and regulatory process, built a commercial organization from the ground up, launched multiple drugs, and moved the company toward profitability.

“在本协议结束时，道格·英格拉姆（Doug Ingram）将被任命为箭头（Arrowhead）董事会成员。他领导萨雷普塔（Sarepta）通过临床和监管流程推进多种研究药物，从头开始建立商业组织，推出多种药物，并使公司走向盈利。

His experience and guidance will be valuable as Arrowhead seeks the same transition.”.

随着Arrowhead寻求同样的过渡，他的经验和指导将是宝贵的。”。

“Robust and compelling early data from Arrowhead’s differentiated siRNA approach platform suggests potentially best-in-class treatments that will profoundly improve the lives of those with rare, genetic diseases,” said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research and development, Sarepta.

。

“The targeted ligand approach, combined with Arrowhead’s clinically validated siRNA chemistry, suggests the potential for deep and durable knockdown of proteins that are over-expressed in these conditions. Arrowhead’s innovative approach to cross the blood brain barrier with subcutaneous dosing represents a potential paradigm shift for the CNS preclinical and discovery programs as part of this collaboration.”.

“靶向配体方法，结合Arrowhead临床验证的siRNA化学，表明在这些条件下过度表达的蛋白质有可能被深度和持久的敲除。Arrowhead通过皮下给药跨越血脑屏障的创新方法代表了中枢神经系统临床前和发现计划的潜在范式转变，作为这一合作的一部分。”。

Summary of Financial Terms

财务条款摘要

Under the terms of the agreement, Sarepta will pay Arrowhead an upfront payment of $500 million and an equity investment of $325 million in Arrowhead common stock, priced at a 35% premium to the 30-day volume weighted average price prior to the announcement of the agreement. Arrowhead will also receive $250 million to be paid in annual installments of $50 million over five years, in addition to being eligible for future milestone payments and royalties.

根据协议条款，Sarepta将向Arrowhead支付5亿美元的预付款和3.25亿美元的Arrowhead普通股股权投资，价格比协议宣布前的30天成交量加权平均价格溢价35%。箭头还将获得2.5亿美元，在五年内每年分期支付5000万美元，此外还有资格获得未来的里程碑付款和版税。

Arrowhead will be responsible for Phase 1/2 trials currently underway, and clinical stage programs will transition to Sarepta no later than the completion of current trials. Preclinical assets will transition to Sarepta upon completion of IND-enabling activities by Arrowhead..

Arrowhead将负责目前正在进行的1/2期试验，临床阶段计划将在不迟于当前试验完成后过渡到Sarepta。Arrowhead完成IND启用活动后，临床前资产将过渡到Sarepta。。

The deal will be funded with cash on hand and Sarepta does not plan to raise additional capital via the debt or equity capital markets. The transaction is expected to close early 2025, subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions..

这笔交易将由手头现金提供资金，萨雷塔不计划通过债务或股权资本市场筹集额外资金。根据1976年《哈特·斯科特·罗迪诺反托拉斯改进法案》和其他习惯条件，该交易预计将于2025年初结束，等待期到期或终止。。

Ropes & Gray LLP is serving as legal advisor to Sarepta.

Ropes&Gray LLP担任Sarepta的法律顾问。

Share Repurchase Program

股份回购计划

Sarepta’s Board of Directors has also approved a share repurchase program of up to $500 million of the Company’s outstanding common stock over the next 18 months.

萨雷塔董事会还批准了一项在未来18个月内回购公司5亿美元已发行普通股的计划。

The timing of any stock repurchases will be based upon the continuing analysis of market, financial, and other factors by the Company’s management. Repurchases under the authorized stock repurchase program may be made using a variety of methods, which may include, but are not limited to, open market purchases, privately negotiated transactions, accelerated share repurchase agreements or purchases pursuant to a Rule 10b5-1 plan under the Securities Exchange Act of 1934, as amended.

任何股票回购的时机将基于公司管理层对市场、财务和其他因素的持续分析。授权股票回购计划下的回购可以使用多种方法进行，包括但不限于公开市场购买、私下谈判交易、加速股票回购协议或根据经修订的《1934年证券交易法》第10b5-1条规定的计划进行购买。

The authorized stock repurchase program may be suspended, delayed or discontinued at any time..

授权股票回购计划可随时暂停、延迟或停止。。

Sarepta Investor Call Details

Sarepta投资者电话详情

At 8:30 a.m. ET on Nov. 26, 2024, Sarepta will host a conference call and webcast to discuss this agreement.

2024年11月26日美国东部时间上午8:30，萨雷塔将主持电话会议和网络广播，讨论这项协议。

The event will be webcast live under the investor relations section of Sarepta’s website at https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form.

该活动将在Sarepta网站的投资者关系部分进行在线直播https://investorrelations.sarepta.com/events-presentations活动结束后，重播将在那里存档一年。通过电话参与的相关方需要使用此在线表格进行注册。

After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone..

注册拨入详细信息后，所有电话参与者将收到一封自动生成的电子邮件，其中包含拨入号码的链接以及用于通过电话访问活动的个人PIN码。。

About Sarepta Therapeutics

关于Sarepta Therapeutics

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development.

萨雷塔正在执行一项紧急任务：为罕见疾病设计精确的基因医学，这些疾病会破坏生命并缩短未来。我们在杜兴氏肌营养不良症（DMD）和肢带型肌营养不良症（LGMD）方面担任领导职务，目前我们有40多个项目处于不同的发展阶段。

Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook..

我们在基因治疗，RNA和基因编辑方面的多平台精密遗传医学引擎推动了我们庞大的管道。有关更多信息，请访问www.sarepta.com或在LinkedIn、X、Instagram和Facebook上关注我们。。

Sarepta Forward-Looking Statements

Sarepta前瞻性声明

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements.

本新闻稿包含“前瞻性声明”。本新闻稿中任何不属于历史事实声明的声明都可能被视为前瞻性声明。诸如“相信”、“预期”、“计划”、“预期”、“将”、“打算”、“潜在”、“可能”等词语以及类似的表达方式旨在识别前瞻性陈述。

These forward-looking statements include but are not limited to statements regarding the closing of the transaction and the equity investment; Sarepta’s payment of regulatory and sales milestones, and royalty payments to Arrowhead pursuant to the agreement; the expected targets of the clinical and pre-clinical programs licensed pursuant to the agreement; ongoing development of therapeutics against a broad range of skeletal muscle gene targets by Sarepta and Arrowhead; Arrowhead’s potentially best-in-class approach to siRNA; the expected timing of future data readouts; and statements about the amount and timing of repurchases under Sarepta’s share purchase program and the methods to execute such repurchases..

；Sarepta根据协议支付监管和销售里程碑，以及向Arrowhead支付版税；根据协议许可的临床和临床前计划的预期目标；Sarepta和Arrowhead正在开发针对广泛骨骼肌基因靶标的治疗方法；箭头对siRNA的潜在最佳方法；未来数据读数的预期时间；以及关于Sarepta股份购买计划下回购金额和时间以及执行此类回购的方法的声明。。

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Known risk factors include, among others, market conditions, the expected benefits and opportunities related to the collaboration agreement may not be realized or may take longer to realize than expected due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement, challenges and uncertainties inherent in product research and development and manufacturing limitations; success in preclinical testing and early clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and early results from a clinical trial do not necessarily predict final results; Sarepta may not be able to execute on its business plans, including meeting its expected or planned regulatory milestones and timelines, research and clinical development plans, for various reasons, some of which may be outside of Sarepta’s control, including possible limitations of company financial and other resources; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as well as other SEC filings made by the Company which you are encouraged to review..

这些前瞻性陈述涉及风险和不确定性，其中许多超出了Sarepta的控制范围。已知的风险因素包括市场条件、与合作协议相关的预期利益和机会可能无法实现，或者由于各种原因可能需要比预期更长的时间才能实现，包括双方无法履行其在协议下的承诺和义务、产品研发和制造限制固有的挑战和不确定性；临床前测试和早期临床试验的成功，特别是如果基于少量患者样本，并不能确保后期临床试验的成功，临床试验的早期结果不一定能预测最终结果；由于各种原因，Sarepta可能无法执行其业务计划，包括满足其预期或计划的监管里程碑和时间表，研究和临床开发计划，其中一些可能超出Sarepta的控制范围，包括公司财务和其他资源的可能限制；以及Sarepta最新发布的截至2024年9月30日的10-Q表季度报告中“风险因素”标题下确定的风险，以及该公司提交的其他SEC文件中鼓励您审查的风险。。

Any of the foregoing risks could materially and adversely affect Sarepta’s business, results of operations and the trading price of Sarepta’s common stock. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law..

上述任何风险都可能对Sarepta的业务、经营业绩和Sarepta普通股的交易价格产生重大不利影响。我们提醒投资者不要过分依赖本新闻稿中的前瞻性声明。除法律要求外，Sarepta没有义务根据本协议日期后的事件或情况公开更新其前瞻性声明。。

Source: Sarepta Therapeutics, Inc.

资料来源：Sarepta Therapeutics，Inc。

View source version on businesswire.com: https://www.businesswire.com/news/home/20241126242387/en/

在businesswire.com上查看源代码版本：https://www.businesswire.com/news/home/20241126242387/en/

Investor Contact:

投资者联系人：

Ian Estepan, 617-274-4052

伊恩·埃斯特潘，617-274-4052

iestepan@sarepta.com

iestepan@sarepta.com

Media Contact:

媒体联系人：

Tracy Sorrentino, 617-301-8566

特蕾西·索伦蒂诺，617-301-8566

tsorrentino@sarepta.com

tsorrentino@sarepta.com

Source: Sarepta Therapeutics, Inc.

资料来源：Sarepta Therapeutics，Inc。