CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced topline results from EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study of ELEVIDYS (delandistrogene moxeparvovec-rokl) in patients with Duchenne muscular dystrophy between the ages of 4 through 7 years..

马萨诸塞州剑桥市-Sarepta Therapeutics，Inc.（纳斯达克股票代码：SRPT）是精确遗传医学治疗罕见疾病的领导者，今天宣布了全球EMBARK（研究SRP-9001-301）的顶级成果，随机，双盲，安慰剂对照，ELEVIDYS（delandistrogene moxeparvovec rokl）在4至7岁的Duchenne型肌营养不良症患者中的3期临床研究。。

“The results of EMBARK, our double-blind, placebo-controlled trial, support the conclusion that ELEVIDYS modifies the trajectory of Duchenne and benefits patients across age groups living with this ferociously degenerative disease. The results favored ELEVIDYS across all endpoints in the study, including achieving statistical significance on all pre-specified key secondary endpoints and in each age subgroup of the key secondary endpoints.

“我们的双盲安慰剂对照试验EMBARK的结果支持ELEVIDYS改变Duchenne轨迹并使患有这种恶性退行性疾病的年龄组患者受益的结论，结果有利于ELEVIDYS在研究的所有终点，包括在所有预先指定的关键次要终点和每个终点上达到统计学显着性关键次要终点的年龄子组。

Indeed, passing 5 seconds on time to rise is the strongest predictor of early loss of ambulation and in EMBARK, ELEVIDYS reduced those odds over 52 weeks by greater than 90 percent,” said Doug Ingram, president and chief executive officer, Sarepta. “Based on the EMBARK results, we intend to move swiftly to request an update to expand the labeled indication to treat all patients.

事实上，超过5秒的时间上升是早期失去行走能力的最强预测因素，在EMBARK中，ELEVIDYS在52周内将这些几率降低了90%以上，“Sarepta总裁兼首席执行官道格英格拉姆说。“根据EMBARK的结果，我们打算迅速要求更新，以扩大标记的适应症，以治疗所有患者。

Importantly, we have shared the EMBARK topline results with FDA leadership and they have confirmed that, based on the totality of the evidence, they are open to such label expansion if supported by review of the data, and that they intend to proceed rapidly with consideration of the submission.”.

重要的是，我们已经与FDA领导层分享了EMBARK topline的结果，他们已经证实，根据全部证据，如果得到数据审查的支持，他们可以接受这种标签扩展，并且他们打算快速进行考虑提交“。

In the study, ELEVIDYS-treated patients improved 2.6 points on their North Star Ambulatory Assessment (NSAA) total score 52 weeks after treatment compared to 1.9 points in placebo-treated patients. The difference of 0.65-points between treated and placebo groups did not reach statistical significance (n=125; p=0.24)..

在这项研究中，ELEVIDYS治疗的患者在治疗52周后的North Star门诊评估（NSAA）总分改善了2.6分，而安慰剂治疗的患者则为1.9分。治疗组和安慰剂组之间0.65分的差异未达到统计学显着性（n=125；p=0.24）。。

All key pre-specified functional secondary endpoints demonstrated robust evidence for a clinically meaningful treatment benefit that was consistent across age groups in ELEVIDYS-treated patients compared to placebo at 52 weeks. These include:

所有关键的预先指定的功能性次要终点都显示了强有力的证据，证明在52周时，与安慰剂相比，ELEVIDYS治疗患者的年龄组在临床上有意义的治疗益处是一致的。这些包括：

Time to rise (TTR)

上升时间（TTR）

Change vs Placebo LSM* Diff in Seconds

改变与安慰剂LSM*差异以秒为单位

Overall (n=124)

整体（n=124）

-0.64 (p=0.0025)

-0.64（p=0.0025）

Ages 4-5 (n=59)

年龄4-5岁（n 59）

-0.50 (p=0.0177)

-0.50（p=0.0177）

Ages 6-7 (n=65)

年龄6-7岁（n 65）

-0.78 (p=0.0291)

-0.78（p=0.0291）

10-meter walk test

10米步行测试

Change vs Placebo LSM Diff in Seconds

改变与安慰剂LSM差异以秒为单位

Overall (n=124)

整体（n=124）

-0.42 (p=0.0048)

-0.42（p=0.0048）

Ages 4-5 (n=59)

年龄4-5岁（n 59）

-0.33 (p=0.0319)

-0.33（p=0.0319）

Ages 6-7 (n=65)

年龄6-7岁（n 65）

-0.52 (p=0.0363)

-0.52（p=0.0363）

*least squared means

*最小二乘平均值

All other timed functional endpoints – including stride velocity 95th centile (SV95C) and time to ascend 4 steps – demonstrated consistent treatment benefit in favor of ELEVIDYS. Full results from EMBARK will be shared at future medical meetings and publication will be pursued in a medical journal.

所有其他定时功能终点-包括步速第95百分位（SV95C）和上升4步的时间-表现出一致的治疗益处，有利于ELEVIDYS。EMBARK的全部成果将在未来的医学会议上分享，并将在医学期刊上发表。

“The strong prognostic power of time to rise, and the particular importance of the 5 second milestone in predicting functional decline and future loss of ambulation, is clearly demonstrated in natural history.1 In EMBARK, the reduction in patients progressing past this milestone when treated with ELEVIDYS is highly clinically relevant,” said Craig McDonald, M.D., professor and chair of the UC Davis Health Department of Physical Medicine and Rehabilitation, and an investigator in the EMBARK study.

“自然史清楚地表明，时间上升的强大预后能力，以及预测功能衰退和未来行走能力丧失的第5个里程碑的特殊重要性.1在EMBARK中，使用ELEVIDYS治疗的患者进展超过这一里程碑的患者减少在临床上具有高度相关性，”Craig McDonald，M.D。说。，加州大学戴维斯分校物理医学与康复系教授兼主席，EMBARK研究研究员。

“The consistency of the positive effect across all timed function tests and age groups provides evidence of a meaningful treatment effect. In addition, it is important to note that this is the first clinical trial in the history of DMD trials to show a statistically significant and meaningful improvement on the novel measure of 95th centile stride velocity derived from an objective community wearable activity monitor.”.

“所有定时功能测试和年龄组的积极效果的一致性提供了有意义的治疗效果的证据。此外，重要的是要注意，这是DMD试验历史上第一个显示统计学显着性的临床试验，有意义地改进来自客观社区的第95百分位步速的新测量可耕地活动监视器“。

There were no new safety signals in the EMBARK study, reinforcing the favorable and manageable safety profile observed with ELEVIDYS to date. The most common treatment-related adverse events were gastrointestinal events (vomiting, nausea, and decreased appetite) and pyrexia. Seven participants (11.1%) experienced a treatment-related serious adverse event (SAE) and there were no clinically meaningful changes observed in SAEs associated with known risks of ELEVIDYS..

EMBARK研究中没有新的安全信号，加强了迄今为止ELEVIDYS观察到的有利和可管理的安全性。最常见的治疗相关不良事件是胃肠道事件（呕吐，恶心和食欲下降）和发热。7名参与者（11.1%）经历了与治疗相关的严重不良事件（SAE），并且在与已知的ELEVIDYS风险相关的SAE中没有观察到临床上有意义的变化。。

Conference call details

电话会议详情

On October 30, 2023, at 4:30 p.m. Eastern time, Sarepta will host a conference call and webcast to discuss these results.

2023年10月30日，东部时间下午4:30，Sarepta将主持电话会议和网络广播，讨论这些结果。

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下进行网络广播https://investorrelations.sarepta.com/events-presentations并且在事件发生后，重放将在那里存档一年。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..

在详细注册拨号后，所有电话参与者将收到一封自动生成的电子邮件，其中包含拨号号码的链接以及用于通过电话访问事件的个人PIN号码。。

As part of a collaboration agreement signed in 2019, Roche is working with Sarepta Therapeutics to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function, keeping them stronger for longer. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing.

作为2019年签署的合作协议的一部分，罗氏正在与Sarepta Therapeutics合作，为Duchenne社区改变未来，使患有该疾病的人能够维持和保护其肌肉功能，使他们更强壮更长时间。Sarepta负责美国ELEVIDYS的监管批准和商业化以及制造业。

Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world. Together, the companies are implementing a comprehensive joint clinical development plan to maximize the chances of broad approval and access so that ELEVIDYS can reach as many individuals with Duchenne as rapidly as possible..

罗氏负责监管部门的批准，并将ELEVIDYS带入世界其他地区的患者。这些公司正在共同实施一项全面的联合临床开发计划，以最大限度地获得广泛批准和访问的机会，以便ELEVIDYS能够尽快与Duchenne联系到尽可能多的个人。。

About EMBARK, Study 9001-301

关于EMBARK，研究9001-301

Study SRP-9001-301, also known as EMBARK, is a multinational, phase 3, randomized, two-part crossover, placebo-controlled study of ELEVIDYS in individuals with Duchenne muscular dystrophy between the ages of 4 to 7 years. The primary endpoint is change from baseline in NSAA Total Score at Week 52 following treatment.

研究SRP-9001-301，也称为EMBARK，是一项针对4至7岁的杜兴氏肌营养不良患者的ELEVIDYS的跨国，3期，随机，两部分交叉，安慰剂对照研究。主要终点是治疗后第52周NSAA总分与基线的变化。

Eligible participants received a single dose of ELEVIDYS during either Part 1 or Part 2 of the study..

符合条件的参与者在研究的第1部分或第2部分接受单剂量的ELEVIDYS。。

In Part 1, participants (n=125) were randomized according to age (≥4 to <8 years) or NSAA Total Score at screening (>16 to <29) and received either 1.33 x1014 vg/kg of ELEVIDYS or placebo with a follow-up period for 52 weeks. In Part 2, participants cross over - meaning, those who were previously treated with placebo in Part 1 receive ELEVIDYS and participants who were previously treated with ELEVIDYS receive placebo, with a follow-up period for 52 weeks.

在第1部分中，参与者（n=125）根据年龄（≥4至<8岁）或筛查时NSAA总分（>16至<29）随机分组，并接受1.33×1014 vg/kg的ELEVIDYS或安慰剂随访52周。在第2部分中，参与者交叉意味着，之前在第1部分接受安慰剂治疗的患者接受ELEVIDYS治疗，之前接受ELEVIDYS治疗的参与者接受安慰剂治疗，随访期为52周。

All patients remain blinded..

所有患者仍不知情。。

Secondary outcome measures in EMBARK include the quantity of shortened dystrophin produced by ELEVIDYS at week 12 as measured by western blot in a subset of participants, timed function tests, stride velocity and validated patient reported outcome measures for mobility and upper limb function.

EMBARK的次要结局指标包括ELEVIDYS在第12周产生的肌营养不良蛋白缩短量，通过western blot在一部分参与者中进行测量，定时功能测试，步幅速度以及经过验证的患者报告的活动性和上肢功能结局指标。

About ELEVIDYS (delandistrogene moxeparvovec-rokl)

关于ELEVIDYS（delandistrogene moxeparvovec rokl）

ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose gene transfer therapy for intravenous infusion designed to address the underlying cause of Duchenne muscular dystrophy through the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene and is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle observed in patients treated with ELEVIDYS.

ELEVIDYS（delandistrogene moxeparvovec-rokl）是一种用于静脉输注的单剂量基因转移疗法，旨在通过在骨骼肌中靶向产生ELEVIDYS微肌营养不良蛋白来解决Duchenne肌营养不良的根本原因。ELEVIDYS适用于治疗4至5岁患有Duchenne肌营养不良症（DMD）的门诊儿科患者，DMD基因已确认突变，并根据ELEVIDYS微肌营养不良蛋白在骨骼肌中的表达加速批准。用ELEVIDYS治疗的患者。

Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. ELEVIDYS has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental..

对该适应症的持续批准可能取决于验证性试验中临床益处的验证。ELEVIDYS已达到安全性和有效性的完整法定标准，因此不被视为研究性或实验性。。

In addition to EMBARK, which serves as the postmarketing confirmatory study, ELEVIDYS has been evaluated in three ongoing clinical studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. Accelerated approval was primarily based on data from SRP-9001-102 and SRP-9001-103.

除了作为上市后确认研究的EMBARK之外，ELEVIDYS还在三项正在进行的临床研究中进行了评估：SRP-9001-101，SRP-9001-102和SRP-9001-103。加速批准主要基于SRP-9001-102和SRP-9001-103的数据。

IMPORTANT SAFETY INFORMATION

重要的安全信息

CONTRAINDICATION:

禁忌：

ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

ELEVIDYS禁用于DMD基因第8外显子和/或第9外显子缺失的患者。

WARNINGS AND PRECAUTIONS:

警告和注意事项：

Acute Serious Liver Injury:

急性严重肝损伤：

Acute serious liver injury has been observed with ELEVIDYS. Administration of ELEVIDYS may result in elevations of liver enzymes (e.g., GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks.

ELEVIDYS观察到急性严重肝损伤。通常在8周内施用ELEVIDYS可导致肝酶（例如GGT，GLDH，ALT，AST）或总胆红素升高。

Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.

先前存在肝损伤，慢性肝病或急性肝病（例如急性肝病毒感染）的患者可能具有较高的急性严重肝损伤风险。推迟给急性肝病患者服用ELEVIDYS，直至解决或控制。

Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT and total bilirubin levels return to near baseline levels)..

在ELEVIDYS给药之前，在ELEVIDYS输注后的前3个月每周进行肝酶测试并监测肝功能（临床检查，GGT和总胆红素）。如果临床指示，继续监测，直到结果不明显（正常临床检查，GGT和总胆红素水平恢复到接近基线水平）。。

Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, a consultation with a specialist is recommended.

建议在输注ELEVIDYS之前和之后对患者进行全身皮质类固醇治疗。指示时调整皮质类固醇方案。如果怀疑有急性严重肝损伤，建议咨询专家。

Immune-mediated Myositis:

免疫介导的肌炎：

In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness including dysphagia, dyspnea and hypophonia were observed.

在临床试验中，在涉及DMD基因中外显子8和/或外显子9的缺失突变的患者中，在ELEVIDYS输注后约1个月观察到免疫介导的肌炎。观察到严重的肌肉无力症状，包括吞咽困难，呼吸困难和声音不足。

Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene between exons 1 to 17 and exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction.

对于外显子1至17和外显子59至71之间DMD基因突变的患者，可用于ELEVIDYS治疗的数据有限。这些区域缺失的患者可能有发生严重免疫介导的肌炎反应的风险。

Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea or hypophonia as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient’s clinical presentation and medical history if these symptoms occur..

如果患者出现任何不明原因的肌肉疼痛，压痛或虚弱，包括吞咽困难，呼吸困难或低音，建议患者立即联系医生，因为这些可能是肌炎的症状。如果出现这些症状，请根据患者的临床表现和病史考虑其他免疫调节治疗（除皮质类固醇外的免疫抑制剂[例如钙调神经磷酸酶抑制剂]）。。

Myocarditis:

心肌炎：

Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials.

在临床试验中ELEVIDYS输注后观察到急性严重心肌炎和肌钙蛋白-I升高。

Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath.

在输注ELEVIDYS之前监测肌钙蛋白-I，输注后第一个月每周监测肌钙蛋白-I，如果临床指示则继续监测。如果出现心脏症状，如胸痛或呼吸急促，可能需要更频繁的监测。

Advise patients to contact a physician immediately if they experience cardiac symptoms.

建议患者在出现心脏症状时立即联系医生。

Pre-existing Immunity against AAVrh74:

预先存在的针对AAVrh74的豁免：

In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all subjects developed anti-AAVrh74 antibodies.

在基于AAV载体的基因疗法中，预先存在的抗AAV抗体可能阻碍所需治疗水平的转基因表达。用ELEVIDYS治疗后，所有受试者均产生了抗AAVrh74抗体。

Perform baseline testing for the presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration.

在施用ELEVIDYS之前，对抗AAVrh74总结合抗体的存在进行基线测试。

ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400.

对于抗AAVrh74总结合抗体滴度大于或等于1:400的患者，不建议使用ELEVIDYS。

Adverse Reactions:

不良反应：

The most common adverse reactions (incidence ≥ 5%) reported in clinical studies were vomiting, nausea, liver function test increased, pyrexia, and thrombocytopenia.

临床研究中报告的最常见不良反应（发生率≥5%）是呕吐，恶心，肝功能检查增加，发热和血小板减少。

For further information, please see the full Prescribing Information.

有关详细信息，请参阅完整的处方信息。

About Sarepta Therapeutics

关于Sarepta疗法

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.

Sarepta的紧迫使命是：为破坏生命并缩短期货的罕见疾病设计精确的遗传医学。我们在杜氏肌营养不良症（DMD）和肢带肌营养不良症（LGMD）中担任领导职位，目前我们在各个发展阶段拥有40多个计划。

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 Twitter, LinkedIn, Instagram and Facebook..

我们庞大的管道由我们在基因治疗，RNA和基因编辑方面的多平台精确遗传医学引擎驱动。欲了解更多信息，请访问www.sarepta.com或在Twitter，LinkedIn，Instagram和Facebook上关注我们。。

Internet Posting of Information

互联网发布信息

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www.sarepta.com网站的“为投资者服务”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”。任何不是历史事实陈述的陈述都可能被视为前瞻性陈述。诸如“相信”，“预期”，“计划”，“期望”，“意愿”，“可能”，“打算”，“准备”，“看”，“潜力”，“可能”之类的词语旨在识别前瞻性陈述。

These forward-looking statements include, without limitation, statements relating to our future operations, business plans, priorities, research and development programs; our understanding that U.S. FDA has indicated openness to reviewing the data for label expansion based on the totality of evidence from EMBARK, if supported by review of the data, and that they intend to proceed rapidly with consideration of the submission; the potential benefits of ELEVIDYS, including the potential to modify the trajectory of Duchenne and benefit patients across age groups living with Duchenne; and expected plans and milestones, including moving swiftly to request an update to expand the labeled indication to treat all patients for ELEVIDYS, and sharing full results from EMBARK at future medical meetings and a publication pursued in a medical journal..

这些前瞻性声明包括但不限于与我们未来的运营，业务计划，优先事项，研究和发展计划有关的声明；我们的理解是，如果得到数据审查的支持，美国FDA已经表示开放根据EMBARK的全部证据审查标签扩展数据，并且他们打算在考虑提交内容时迅速进行；ELEVIDYS的潜在好处，包括修改Duchenne轨迹的潜力，并使Duchenne生活的各个年龄组的患者受益；以及预期的计划和里程碑，包括迅速要求更新以扩展标记的适应症以治疗所有患者的ELEVIDYS，并在EMBARK未来的医学会议和医学期刊上发表的出版物中分享全部结果。。

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: the FDA may not approve a supplement to expand the approved label for ELEVIDYS; 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; our data may not be sufficient for obtaining regulatory approval; we are subject to uncertainty related to reimbursement policies; 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; continued approval may be contingent upon verification of a clinical benefit in confirmatory trials; 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 collec.

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所陈述或暗示的结果大不相同。已知的风险因素包括：FDA可能不批准补充扩大ELEVIDYS的批准标签；我们可能无法及时或根本遵守所有FDA的要求；FDA和其他监管机构的法规和监管决策可能对我们的业务产生影响；我们的数据可能不足以获得监管部门的批准；我们受制于与报销政策有关的不确定性；临床前和临床试验的成功，特别是如果基于小患者样本，并不能确保后来的临床试验成功，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，或者可能无法满足候选产品安全性和有效性的监管审批要求；持续批准可能取决于验证性试验中临床获益的验证；由于多种原因，我们的临床试验开始和完成以及结果公布可能会被延迟或阻止，其中包括监管机构拒绝进行我们的临床试验的许可，或者暂停进行临床试验，识别，招聘，招募和留住患者参加临床试验以及进行临床试验所需的候选产品或其他材料的供应数量或质量不足；我们用来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，即使我们相信数据收集。

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本公司的业务，运营结果和Sarepta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说明，鼓励您查看Sarepta制作的SEC文件。我们提醒投资者不要严重依赖本新闻稿中包含的前瞻性声明。

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不承担任何义务根据本协议签署之日以后的事件或情况公开更新其前瞻性声明。。

Reference: 1) Zambon, AA, et al; The UK Northstar Clinical Network. Peak functional ability and age at loss of ambulation in Duchenne muscular dystrophy. Dev Med Child Neurol. 2022; 64: 979–988.

参考文献：1）Zambon，AA，et al；英国北星临床网络。杜兴氏肌营养不良症丧失活动能力和年龄的峰值。Dev Med Child Neurol。2022; 64: 979–988.