Sarepta Therapeutics宣布多个肢带型肌营养不良项目的研发进展-动脉网

Sarepta Therapeutics Announces Pipeline Progress for Multiple Limb-Girdle Muscular Dystrophy Programs

萨雷普塔等信源发布 2025-04-15 08:30



可切换为仅中文







–

U.S.

美国

FDA has confirmed that screening and dosing may proceed in Study SRP-9005-101 for LGMD2C/R5

FDA已确认，在LGMD2C/R5的SRP-9005-101研究中可以进行筛查和给药。

–

Enrollment and dosing completed in Study SRP-9004-102 for LGMD2D/R3

LGMD2D/R3的SRP-9004-102研究已完成入组和给药

–

Data expected for SRP-9003 for LGMD2E/R4 by mid-2025

预计到2025年中将获得LGMD2E/R4的SRP-9003数据。

CAMBRIDGE, Mass.

马萨诸塞州剑桥市

--(BUSINESS WIRE)--Apr. 15, 2025--

--（商业资讯）--2025年4月15日--

Sarepta Therapeutics, Inc.

(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared updates from its clinical programs focused on limb-girdle muscular dystrophy (LGMD) subtypes 2C/R5, 2D/R3, and 2E/R4.

(NASDAQ:SRPT)，罕见病精准基因药物领域的领导者，今天分享了其专注于肢带型肌营养不良（LGMD）亚型2C/R5、2D/R3和2E/R4的临床项目更新。

SRP-9005 for LGMD type 2C/R5:

SRP-9005 用于 LGMD 2C/R5 型：

Following input from the

以下是输入的

U.S. Food and Drug Administration

美国食品药品监督管理局

(FDA),

（FDA），

Office of Therapeutic Products

治疗产品办公室

(OTP), Sarepta is cleared to proceed with dosing in Study SRP-9005-101 (COMPASS) in the

(OTP)，Sarepta 获准在研究 SRP-9005-101（COMPASS）中进行给药

U.S.

美国

COMPASS is a first-in-human clinical study of SRP-9005, an investigational gene therapy for LGMD type 2C/R5, or gamma-sarcoglycanopathy.

COMPASS是一项针对LGMD 2C/R5型或γ-肌聚糖病的在研基因疗法SRP-9005的首次人体临床研究。

SRP-9004 for LGMD type 2D/R3:

SRP-9004 用于 LGMD 2D/R3 型：

Enrollment and dosing is complete in Study SRP-9004-102 (DISCOVERY).

研究 SRP-9004-102（DISCOVERY）的注册和给药已完成。

DISCOVERY is a phase 1, proof-of-concept study evaluating safety and expression of the alpha-sarcoglycan protein after treatment with SRP-9004, an investigational gene therapy for the treatment of LGMD type 2D/R3, or alpha-sarcoglycanopathy.

DISCOVERY 是一项一期的、概念验证研究，评估使用 SRP-9004（一种用于治疗 LGMD 2D/R3 型或 α-肌聚糖病的在研基因疗法）治疗后的安全性和 α-肌聚糖蛋白的表达。

SRP-9003 for LGMD type 2E/R4:

SRP-9003 用于 LGMD 2E/R4 型：

Enrollment and dosing is complete in Study SRP-9003-301 (EMERGENE). EMERGENE is a phase 3 clinical trial of SRP-9003 (bidridistrogene xeboparvovec) for the treatment of LGMD type 2E/R4, or beta-sarcoglycanopathy. EMERGENE is a global study and the primary endpoint is the biomarker expression of beta-sarcoglycan protein.

在研究SRP-9003-301（EMERGENE）中，受试者招募和给药已经完成。EMERGENE是一项关于SRP-9003（bidridistrogene xeboparvovec）的三期临床试验，用于治疗LGMD 2E/R4型或β-肌聚糖病。EMERGENE是一项全球性研究，主要终点是β-肌聚糖蛋白的生物标志物表达。

A pre-Biologics License Application (BLA) meeting has occurred and the OTP has confirmed eligibility for the accelerated approval pathway for the program. Sarepta remains on track to submit a BLA to the .

已召开生物制品许可申请（BLA）前会议，OTP已确认该计划符合加速审批通道的资格。Sarepta仍有望按计划向FDA提交BLA。

U.S.

美国

FDA in the second half of 2025.

2025年下半年，FDA。

“There are no disease-modifying treatments approved for patients with any subtype of limb-girdle muscular dystrophy, and the unmet medical need is significant. Following feedback from

“目前尚无获批用于治疗任何亚型的肢带型肌营养不良症患者的疾病修饰疗法，且未满足的医疗需求十分显著。根据反馈

U.S.

美国

FDA, we are pleased to announce that screening can proceed in Sarepta’s first clinical study for a gene therapy for individuals with LGMD type 2C – the fourth LGMD program that Sarepta has advanced into the clinic,” said

FDA，我们很高兴地宣布，Sarepta首个针对LGMD 2C型基因治疗的临床研究筛查可以继续进行——这是Sarepta推进到临床阶段的第四个LGMD项目，”

Louise Rodino-Klapac

路易丝·罗迪诺-克拉帕克

, Ph.D., executive vice president, chief scientific officer and head of research and development,

博士，执行副总裁，首席科学官兼研发部门负责人，

Sarepta Therapeutics

. “In addition to our progress with SRP-9004 and SRP-9005, we remain on track to share data in the first half of this year from the EMERGENE study with SRP-9003. Our confidence in the potential for gene therapy to bring meaningful treatments to patients with rare, genetic based diseases remains high and the rapid progress across our LGMD pipeline is encouraging.”.

“. 除了我们在SRP-9004和SRP-9005方面取得的进展外，我们仍有望在今年上半年分享来自SRP-9003的EMERGENE研究的数据。我们对基因治疗为罕见遗传病患者带来有意义治疗方法的潜力仍然充满信心，我们在LGMD管线上的快速进展令人鼓舞。”

About Limb-Girdle Muscular Dystrophy

关于肢带型肌营养不良症

Limb-girdle muscular dystrophies (LGMD) 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. There are more than 30 distinct subtypes of LGMD, each with a unique genetic mutation and distinct symptoms, progression, and treatment approaches..

肢带型肌营养不良症（LGMD）是一种遗传性疾病，会导致进行性、致残性的肌肉无力和萎缩，首先影响髋部和肩部周围的肌肉，随后逐渐扩展到手臂和腿部的肌肉。LGMD有30多种不同的亚型，每种亚型都有独特的基因突变以及不同的症状、病程和治疗方法。

Sarepta’s leading LGMD pipeline currently has gene therapy programs in different stages of development for LGMD 2B/R2, LGMD 2E/R4, LGMD 2D/R3, LGMD 2C/R5, and LGMD 2A/R1 which together represent more than 70 percent of known LGMD cases. Sarepta is also the sponsor of JOURNEY, a natural history study evaluating disease progression for four LGMD subtypes: 2C/R5, 2D/R3, 2E/R4 and 2A/R1.

Sarepta公司领先的LGMD管线目前在LGMD 2B/R2、LGMD 2E/R4、LGMD 2D/R3、LGMD 2C/R5和LGMD 2A/R1等不同开发阶段拥有基因治疗项目，这些项目合计占已知LGMD病例的70％以上。Sarepta还是JOURNEY的赞助者，这是一项自然史研究，评估四种LGMD亚型（2C/R5、2D/R3、2E/R4和2A/R1）的疾病进展。

JOURNEY is actively enrolling globally..

旅程正在全球范围内积极招募。

About SRP-9003 (bidridistrogene xeboparvovec)

关于SRP-9003（bidridistrogene xeboparvovec）

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 heart.

SRP-9003（bidridistrogene xeboparvovec）是一种研究性基因疗法，利用AAVrh74载体，该载体旨在系统且高效地递送到骨骼肌、膈肌和心肌，使其成为治疗神经肌肉疾病的理想候选药物。SRP-9003旨在递送全长的β-肌聚糖转基因，并使用MHCK7启动子，该启动子因其在心脏中强大的表达能力而被选中。

1,2,3

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.

这对肢带型肌营养不良症2E型（LGMD2E），也称为β-肌聚糖病和LGMDR4的患者来说至关重要，其中许多患者死于肺部或心脏并发症。

About SRP-9004 (patidistrogene bexoparvovec)

关于SRP-9004（patidistrogene bexoparvovec）

SRP-9004 (patidistrogene bexoparvovec) is an investigational gene therapy for limb-girdle muscular dystrophy Type 2D (LGMD2D/R3). LGMD2D/R3 causes muscle weakness and primarily affects the muscles around the hips, shoulders, and thighs. SRP-9004 is engineered using 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-9004（patidistrogene bexoparvovec）是一种用于治疗2D型肢带型肌营养不良（LGMD2D/R3）的在研基因疗法。LGMD2D/R3会导致肌肉无力，主要影响髋部、肩部和大腿周围的肌肉。SRP-9004利用AAVrh74载体设计，旨在系统性且高效地递送到骨骼肌、膈肌和心肌，使其成为治疗神经肌肉疾病的理想候选药物。

Intended to deliver a full-length alpha-sarcoglycan transgene, SRP-9004 uses the tMCK promoter, chosen for its ability to selectively express in skeletal muscle which is critically important for patients with LGMD2D/R3..

旨在递送全长的α-肌聚糖转基因，SRP-9004使用了tMCK启动子，选择该启动子是因为它能够在骨骼肌中选择性表达，这对LGMD2D/R3患者至关重要。

About SRP-9005

关于SRP-9005

SRP-9005 is an investigational gene therapy for patients with limb-girdle muscular dystrophy Type 2C (LGMD2C/R5), also known as gamma-sarcoglycanopathy. SRP-9005 is intended to deliver a full-length gamma-sarcoglycan transgene using the AAVrh74 vector. SRP-9005 uses the MHCK7 promoter, chosen for its ability to robustly express in the heart, which is critically important for those with LGMD2C, many of whom die from pulmonary or cardiac complications..

SRP-9005 是一种针对肢带型肌营养不良症 2C 型（LGMD2C/R5），也称为γ-肌聚糖病患者的在研基因疗法。SRP-9005 旨在使用 AAVrh74 载体递送全长的γ-肌聚糖转基因。SRP-9005 使用 MHCK7 启动子，选择该启动子是因为它能够在心脏中强烈表达，这对 LGMD2C 患者至关重要，其中许多人死于肺部或心脏并发症。

About

关于

Sarepta Therapeutics

萨雷普塔治疗公司

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 (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases.

萨雷普塔肩负着一项紧迫的使命：为那些摧毁生命、缩短未来的罕见疾病研发精准的基因药物。我们在杜氏肌营养不良症（Duchenne）和肢带型肌营养不良症（LGMDs）领域处于领先地位，并正在构建涵盖肌肉、中枢神经系统和心脏疾病的强大项目组合。

For more information, please visit .

欲了解更多信息，请访问。

www.sarepta.com

or follow us on

或关注我们

领英

，

Instagram

图享

and

和

Facebook

。

Internet Posting of Information

信息发布到互联网上

We routinely post information that may be important to investors in the 'For Investors' section of our website at

我们经常在我们网站的“对于投资者”栏目发布对投资者而言可能重要的信息。

www.sarepta.com

. We encourage investors and potential investors to consult our website regularly for important information about us.

我们鼓励投资者和潜在投资者定期查阅我们的网站，以获取关于我们的重要信息。

Forward-Looking Statements

前瞻性声明

In order to provide Sarepta’s investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements.

本新闻稿旨在让Sarepta的投资者了解其当前业绩和未来前景，因此包含前瞻性声明。本新闻稿中非历史事实的任何声明均可被视为前瞻性声明。

Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “may,” “intends,” “prepares,” “looks,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our future operations, business plans, market opportunities, priorities and research and development programs and technologies; the potential benefits of our technologies, scientific approaches and strategic partnerships; and expected milestones and plans, including beginning dosing in Study SRP-9005-101, sharing data from our EMERGENE study in the first half of the year, and our expectation to file a BLA for SRP-9003 in the second half of 2025..

诸如“相信”、“预期”、“计划”、“预计”、“将”、“可能”、“打算”、“准备”、“展望”、“潜力”、“可能性”等词语旨在标识前瞻性陈述。这些前瞻性陈述包括与我们未来的运营、业务计划、市场机会、优先事项以及研发项目和技术相关的陈述；我们技术、科学方法和战略合作伙伴关系的潜在优势；以及预期的里程碑和计划，包括在研究SRP-9005-101中开始给药、在今年上半年分享我们的EMERGENE研究数据，以及我们预计在2025年下半年为SRP-9003提交BLA的计划。

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. 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: 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 may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; the expected benefits and opportunities related to our agreements with strategic partners 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, challenges and uncertainties inherent in product research and development and manufacturing limitations; if the actual number of patients suffering from the diseases we aim to treat is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing our product candidates to market, for various reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our produc.

这些前瞻性陈述涉及风险和不确定性，其中许多是Sarepta无法控制的。由于这些风险和不确定性，实际结果可能与这些前瞻性陈述中所述或暗示的结果存在重大差异。已知的风险因素包括以下内容：在临床前和临床试验中的成功，特别是如果基于少量患者样本，不能确保后期临床试验会成功，未来研究的结果可能与过去的积极结果不一致或可能无法满足监管机构对候选产品安全性和有效性的审批要求；与战略合作伙伴协议相关的预期利益和机会可能无法实现，或可能比预期需要更长时间才能实现，原因包括多种因素，例如各方可能无法履行其承诺和义务、产品研究与开发以及制造限制所固有的挑战和不确定性；如果我们旨在治疗的疾病的实际患者数量小于估计，我们的收入和实现盈利的能力可能会受到不利影响；我们可能无法执行我们的商业计划，包括达到预期或计划的监管里程碑和时间表、研究和临床开发计划，以及将我们的候选产品推向市场，原因多种多样，其中一些可能超出我们的控制范围，包括公司财务和其他资源的可能限制、未能及时预见或解决的制造限制，以及监管机构、法院或其他机构的决定，例如美国专利商标局对我们产品相关专利的决定。

December 31, 2024

2024年12月31日

filed with the

提交给

Securities and Exchange Commission

证券交易委员会

(SEC) as well as other

（美国证券交易委员会）以及其他

SEC

证券交易委员会

filings made by the Company which you are encouraged to review.

公司提交的文件，建议您查阅。

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

Pozsgai ER 等。系统性AAV介导的β-肌聚糖递送靶向心脏和骨骼肌改善LGMD2E小鼠的组织学和功能缺陷。《分子治疗》2017年。

Apr 5

4月5日

;25(4):855-869.

；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.

门德尔 JR 等。评估rAAVrh74.MHCK7.micro-dystrophin系统递送在杜氏肌营养不良症儿童中的应用：一项非随机对照试验。《美国医学会神经病学杂志》。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。设计用于骨骼肌和心肌中高水平rAAV介导表达的组织特异性调控盒。《分子治疗》。2007；15（2）：320-329。

View source version on

查看源版本

businesswire.com

商业电讯网

：

https://www.businesswire.com/news/home/20250415170851/en/

https://www.businesswire.com/news/home/20250415170851/zh/

Investor Contact:

投资者联系方式：

Ian Estepan

伊恩·埃斯特潘