<div id="readability-page-1">Sarepta Therapeutics Inc. ( SRPT ) on Monday released topline three-year functional results from Part 1-treated patients in the EMBARK (Study SRP-9001-301) Phase 3 study of Elevidys (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy. Three years after treatment, patients who received Elevidys in Part 1 of EMBARK demonstrated statistically significant, clinically meaningful, and durable efficacy across all key motor function measures, North Star Ambulatory Assessment (NSAA), Time to Rise (TTR), and 10-meter walk/run (10MWR), when compared to a pre-specified propensity-weighted untreated external control group. The mean NSAA score remained above baseline at Year 3 for the Elevidys-treated group (n=52) while the EC group (n=73) continued to show the expected age-related decline below their baseline score. The Elevidys group showed a 73% slowing of disease progression as measured by TTR and 70% slowing of disease progression as measured by 10MWR when compared to the control group. Patients treated with Elevidys in Part 1 maintained significantly higher levels of motor function three years after treatment compared to the control group. No new treatment-related safety signals were observed. Analysis of the three-year data is ongoing and includes functional results from crossover-treated patients two years after treatment. Elevidys is the only approved gene therapy for Duchenne, and to date, the drug has been administered to over 1,200 patients globally in clinical and real-world settings. Elevidys is available as appropriate to ambulatory individuals ages four and above, per an updated FDA label announced in November 2025. <h2>Roche Collaboration And Expanded Patient Studies</h2> As part of a collaboration agreement signed in 2019, Sarepta is working with Roche Holdings AG (OTC:RHHBY) to transform the future for the Duchenne community, with the goal of enabling those living with the disease to maintain and protect their muscle function. Sarepta handles U.S. regulatory approval, manufacturing, and commercialization of Elevidys, while Roche manages regulatory approvals and distribution globally outside the U.S., with Chugai Pharmaceuticals leading commercialization in Japan. In November 2025, the U.S. Food and Drug Administration (FDA) approved dosing in Sarepta’s Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort 8 is to evaluate the use of an enhanced immunosuppressive regimen as part of treatment with Elevidys for non-ambulant (patients who cannot walk) individuals with Duchenne muscular dystrophy. <h2>Recent Financial Performance</h2> Earlier in January, Sarepta reported a preliminary fourth-quarter product revenue of $369.6 million and $1.86 billion for full-year 2025. Fourth quarter and full-year 2025 net product revenue for Elevidys reached $110.4 million and $898.7 million, respectively. SRPT Price Action: Sarepta Therapeutics ( SRPT ) shares were up 5.06% at $22.20 during premarket trading on Monday, according to Benzinga Pro data. Photo via Shutterstock</div>

Sarepta Therapeutics Inc. 宣布其针对杜氏肌营养不良症的 Elevidys 第三阶段 EMBARK 研究的三年积极结果。接受 Elevidys 治疗的患者在运动功能上显示出显著改善，与对照组相比，疾病进展速度减缓了 73%。该药物是唯一获得批准的杜氏基因疗法，已被应用于超过 1,200 名患者。Sarepta 报告称，2025 年第四季度产品收入为 3.696 亿美元，全年收入为 18.6 亿美元。股价在盘前交易中上涨 5.06%，至 22.20 美元

Sarepta Says Elevidys Slows Duchenne Progression After Three Years

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Sarepta 表示，Elevidys 在三年后减缓了杜氏肌营养不良症的进展