– Sarepta anticipates filing for accelerated approval of SRP-9003 in 2025

–Sarepta预计在2025年提交SRP-9003加速批准

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dec. 18, 2024--

马萨诸塞州剑桥市--（商业新闻短讯）--2024年12月18日--

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that enrollment and dosing is complete in EMERGENE (Study SRP-9003-301), a Phase 3 clinical trial of SRP-9003 (bidridistrogene xeboparvovec). SRP-9003 is an investigational gene therapy for the treatment of limb-girdle muscular dystrophy Type 2E/R4 (LGMD2E/R4), or beta-sarcoglycanopathy.

Sarepta Therapeutics，Inc.（纳斯达克：SRPT）是罕见疾病精密遗传医学的领导者，今天宣布，SRP-9003（bidridistrogene xeboparvovec）的3期临床试验（研究SRP-9003-301）的登记和给药已完成。SRP-9003是一种研究性基因疗法，用于治疗2E/R4型肢带型肌营养不良症（LGMD2E/R4）或β-糖皮质激素病。

EMERGENE is a global study, and the primary endpoint is the biomarker expression of beta-sarcoglycan protein, the absence of which is the sole cause of LGMD2E/R4..

。。

“The completion of enrollment in the EMERGENE study marks a significant milestone to bring a potentially disease-modifying treatment to individuals living with LGMD2E, an ultra-rare form of LGMD with no treatments beyond symptom management. We are committed to securing approval of SRP-9003 as quickly as possible and are now closer to reaching that goal for patients and their families,” said Louise Rodino-Klapac, Ph.D., executive vice president, chief scientific officer and head of research and development, Sarepta Therapeutics.

Sarepta Therapeutics执行副总裁、首席科学官兼研发负责人Louise Rodino Klapac博士说：“紧急研究登记的完成标志着一个重要的里程碑，可以为患有LGMD2E的个体带来潜在的疾病缓解治疗，LGMD2E是一种极为罕见的LGMD形式，除了症状管理之外没有其他治疗方法。我们致力于尽快获得SRP-9003的批准，现在更接近患者及其家人实现这一目标。”。

“The design of EMERGENE is an important precedent that informs development plans for Sarepta’s other LGMD pipeline programs, including our LGMD2D program which we just initiated and our LGMD2C program which we expect to initiate in the first quarter of 2025, and serves as a pathfinder for heterogenous neuromuscular gene therapies more broadly.”.

。

Data from EMERGENE are expected in the first half of 2025. Assuming a positive pre-Biologics License Application (BLA) meeting and supportive data from EMERGENE, Sarepta anticipates submitting a BLA to the U.S. Food and Drug Administration seeking accelerated approval for SRP-9003 in 2025.

EMERGENE的数据预计将在2025年上半年发布。假设生物制剂前许可证申请（BLA）会议取得积极进展，且EMERGENE提供了支持性数据，Sarepta预计将于2025年向美国食品和药物管理局提交BLA，以寻求SRP-9003的加速批准。

About Study SRP-9003-301 (EMERGENE)

关于研究SRP-9003-301（紧急）

EMERGENE, Study 9003-301 is a Phase 3, multinational, open-label study of SRP-9003 for the treatment of LGMD2E/R4 in ambulatory and non-ambulatory participants, ages 4 and older. EMERGENE’s primary endpoint is expression of beta-sarcoglycan 60 days after dosing. Secondary outcomes and endpoints include functional measures through month 60 and safety..

紧急情况下，研究9003-301是SRP-9003的第三阶段跨国开放标签研究，用于治疗4岁及以上的非卧床和非卧床参与者的LGMD2E/R4。EMERGENE的主要终点是给药后60天β-糖聚糖的表达。次要结果和终点包括第60个月的功能测量和安全性。。

About SRP-9003 (bidridistrogene xeboparvovec)

关于SRP-9003（双脱氧核糖核酸）

SRP-9003 (bidridistrogene xeboparvovec) is an investigational gene therapy that uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat neuromuscular diseases. SRP-9003 is intended to deliver a full-length beta-sarcoglycan transgene and uses the MHCK7 promoter, chosen for its ability to robustly express in the heart1,2,3 which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications..

SRP-9003（bidridistrogene xeboparvovec）是一种研究性基因疗法，使用AAVrh74载体，该载体被设计为全身和稳健地递送至骨骼肌，diaphragm肌和心肌，使其成为治疗神经肌肉疾病的理想候选者。。。

About Limb-girdle Muscular Dystrophy

关于肢带型肌营养不良症

Limb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

肢带型肌营养不良症是一种遗传性疾病，会导致进行性虚弱和消瘦，这种疾病始于臀部和肩膀周围的肌肉，然后发展为手臂和腿部的肌肉。

Patients with LGMD Type 2E/R4 (beta-sarcoglycanopathy) typically begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, frequently progresses to loss of ambulation in the teen years and often leads to early mortality..

LGMD 2E/R4型（β-糖聚糖病）患者通常在10岁之前开始出现神经肌肉症状，例如跑步，跳跃和爬楼梯困难。该疾病是LGMD的常染色体隐性亚型，在青少年时期经常发展为行走能力丧失，并常导致早期死亡。。

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 (formerly Twitter), 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（以前的Twitter）、Instagram和Facebook上关注我们。。

Internet Posting of Information

网上发布信息

We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

我们经常在我们网站www.sarepta.com的“为投资者”部分发布对投资者可能重要的信息。我们鼓励投资者和潜在投资者定期咨询我们的网站，以获取有关我们的重要信息。

Forward-Looking Statements

前瞻性声明

This press release contains “forward-looking statements.” Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements.

本新闻稿包含“前瞻性声明”。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”、“可能”、“打算”、“准备”、“展望”、“潜力”、“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, without limitation, statements relating to our future operations, technologies and scientific approaches, business plans, priorities, research and development programs, the potential benefits of SRP-9003, including SRP-9003 serving as a pathfinder for certain gene therapy more broadly, and expected plans and milestones, including data from EMERGENE in the first half of 2025 and potentially submitting a BLA in 2025, and initiating our LGMD2C program in the first quarter of 2025..

这些前瞻性声明包括但不限于与我们未来的运营、技术和科学方法、商业计划、优先事项、研究和发展计划有关的声明，SRP-9003（包括SRP-9003）作为某些基因治疗更广泛的探路者的潜在益处，以及预期的计划和里程碑，包括2025年上半年EMERGENE的数据，可能在2025年提交BLA，并在2025年第一季度启动我们的LGMD2C计划。。

Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: our data may not be sufficient for obtaining regulatory approval; success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; the commencement and completion of our clinical trials and announcement of results may be delayed or prevented for a number of reasons, including, among others, denial by the regulatory agencies of permission to proceed with our clinical trials, or placement of a clinical trial on hold, challenges in identifying, recruiting, enrolling and retaining patients to participate in clinical trials and inadequate quantity or quality of supplies of a product candidate or other materials necessary to conduct clinical trials; different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; we may not be able to execute on our business plans, including meeting our ex.

由于此类风险和不确定性，实际结果可能与这些前瞻性声明所述或暗示的结果存在重大差异。已知的风险因素包括：我们的数据可能不足以获得监管部门的批准；临床前和临床试验的成功，特别是如果基于少量患者样本，并不能确保以后的临床试验取得成功，未来研究的结果可能与过去的积极结果或咨询委员会的建议不一致，或者可能不符合监管机构对候选产品安全性和有效性的批准要求；我们可能无法及时或根本无法遵守FDA的所有要求；FDA和其他监管机构的法规和监管决定可能对我们的业务产生的影响；我们的临床试验的开始和完成以及结果的宣布可能会因多种原因而延迟或阻止，其中包括监管机构拒绝批准继续进行我们的临床试验，或暂停临床试验，识别，招募，招募和保留患者参加临床试验的挑战，以及候选产品或进行临床试验所需的其他材料的供应数量或质量不足；我们用于评估特定安全性或有效性参数的不同方法，假设和应用可能会产生不同的统计结果，即使我们认为从候选产品的临床试验中收集的数据是积极的，这些数据可能不足以支持FDA或其他全球监管机构的批准；我们可能无法执行我们的商业计划，包括满足我们的前任。

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

上述任何风险都可能对公司的业务、经营业绩和萨雷塔普通股的交易价格产生重大不利影响。有关Sarepta面临的风险和不确定性的详细描述，鼓励您查看Sarepta向SEC提交的文件。我们提醒投资者不要过分依赖本新闻稿中的前瞻性声明。

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..

除法律要求外，Sarepta不承担根据本协议日期后的事件或情况公开更新其前瞻性声明的任何义务。。

Source: Sarepta Therapeutics, Inc.

来源：Sarepta Therapeutics，Inc。

References:

参考文献：

Pozsgai ER, et al. Systemic AAV-Mediated b-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice. Mol. Ther. 2017 Apr 5;25(4):855-869.

针对心肌和骨骼肌的系统性AAV介导的b-糖聚糖递送改善了LGMD2E小鼠的组织学和功能缺陷。摩尔热。2017年4月5日；25（4）：855-869。

Mendell JR, et al. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Jun 15;77(9):1-10.

Mendell JR等人。Duchenne肌营养不良症患儿rAAVrh74.MHCK7.micro-dystrophin全身递送的评估：一项非随机对照试验。JAMA Neurol。2020年6月15日；77（9）：1-10。

Salva MZ, et al. Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle. Mol Ther. 2007;15(2):320-329.

Salva MZ等人。用于骨骼肌和心肌中高水平rAAV介导的表达的组织特异性调节盒的设计。摩尔热。2007年；15（2）：320-329。

View source version on businesswire.com: https://www.businesswire.com/news/home/20241218625378/en/

在businesswire.com上查看源代码版本：https://www.businesswire.com/news/home/20241218625378/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.

来源：Sarepta Therapeutics，Inc。