业绩总结 - 针对非步态患者的ELEVIDYS，报告显示有一例因急性肝衰竭去世的肝损伤病例[6] - 公司承诺在非步态患者中持续监测肝酶升高情况，保持治疗协议不变[23] 用户数据 - Sirolimus被证明能够有效抑制T细胞，减轻可能导致急性肝损伤的炎症反应[8] - 预临床结果显示，Sirolimus有效降低肝酶升高，AST和ALT水平在不同时间点的变化被监测[17] 新产品和新技术研发 - 公司计划与多学科临床专家和监管机构协商，开发增强的免疫抑制方案[6] - 在临床设置中，继续在ELEVIDYS输注前后施用皮质类固醇[23] - 公司将对ENVISION研究的协议修订进行评估，以引入Sirolimus作为免疫抑制方案的一部分[23] 市场扩张和并购 - ENVISION研究暂停，等待协议修订以纳入额外的免疫抑制措施[6] - 在增强方案获得批准之前，暂停对非步态患者的ELEVIDYS商业发货[6] 负面信息 - 针对非步态患者的ELEVIDYS，报告显示有一例因急性肝衰竭去世的肝损伤病例[6]

公司事件调查 - 律师事务所Pomerantz LLP正在调查Sarepta Therapeutics公司及其部分高管是否涉及证券欺诈或其他非法商业行为 [1] - 调查涉及投资者权益保护 投资者可联系指定律师进行咨询 [1] 药品安全事件 - 2025年3月18日 Sarepta披露首例杜氏肌营养不良症患者在使用ELEVIDYS药物后因急性肝衰竭死亡 导致股价单日下跌27.81美元(跌幅27.44%)至73.54美元 [3] - 2025年6月16日 公司再次披露第二例ELEVIDYS用药后急性肝衰竭死亡病例 随即暂停该药物的临床试验并对丧失行走能力患者群体暂停分发 股价单日暴跌15.24美元(跌幅42.12%)至20.94美元 [4] 市场反应 - 两次药品安全事件导致Sarepta股价累计下跌81.41美元 较事件前价格累计跌幅达77.3% [3][4]

Sarepta Therapeutics TodaySRPTSarepta Therapeutics$21.56 +0.00 (+0.02%) 52-Week Range$18.30▼$173.25Price Target$70.76Add to WatchlistIn another tragic incident, Sarepta Therapeutics NASDAQ: SRPT saw a second death in connection with its Duchenne muscular dystrophy (DMD) treatment ELEVIDYS. The patient passed away due to acute liver failure (ALF) after doctors treated them with ELEVIDYS. Sadly, the same thing happened three months ago. As a result, Sarepta has suspended shipments of ELEVIDYS for non-ambulat ...

CANADA - 2025/06/08: In this photo illustration, the Sarepta Therapeutics logo is seen displayed on ... More a smartphone screen. (Photo Illustration by Thomas Fuller/SOPA Images/LightRocket via Getty Images)SOPA Images/LightRocket via Getty ImagesSarepta Therapeutics (NASDAQ: SRPT) suffered a devastating blow on Monday, June 16 when its stock plummeted 42% following news of a second patient death linked to its Duchenne muscular dystrophy gene therapy, Elevidys. The biotechnology company and its global part ...

Sarepta Therapeutics, Inc. (NASDAQ: SRPT ) stock was down 40% in pre-market trading on Monday, June 16th, as seen below. As soon as the market opened, Monday morning losses not only stuck but continued to deepen with the stock down over 47% soI’m a retired Wall Street PM specializing in TMT; since kickstarting my career, I’ve spent over two decades in the market navigating the technology landscape, focusing on risk mitigation through the dot com bubble, credit default of ‘08, and, more recently, with the AI ...

Key Takeaways SRPT reported a second fatality from acute liver failure after Elevidys gene therapy infusion. Sarepta suspends Elevidys dosing commercially and pauses the global ENVISION trial. Elevidys generated over half of the company's Q1 2025 revenue.Shares of Sarepta Therapeutics (SRPT) nosedived 42% in pre-market today after the company reported a second patient death following treatment with Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy (DMD).This latest fatality was caused by ...

About this content About Emily Jarvie Emily began her career as a political journalist for Australian Community Media in Hobart, Tasmania. After she relocated to Toronto, Canada, she reported on business, legal, and scientific developments in the emerging psychedelics sector before joining Proactive in 2022. She brings a strong journalism background with her work featured in newspapers, magazines, and digital publications across Australia, Europe, and North America, including The Examiner, The Advocate, ...

纪要涉及的公司和行业 - **公司**：Sarepta Therapeutics（SRPT） - **行业**：基因治疗行业 纪要提到的核心观点和论据 事件概述 - 一名接受Elevitus治疗的非卧床患者因急性肝衰竭去世，公司已为超900名患者使用Elevitus治疗，涵盖不同年龄、体重、活动状态，有超7年输注经验[3]。 采取措施 - 暂停非卧床患者研究（ENVISION或研究303），寻求协议修正案，在未来输注中预防性使用西罗莫司[4]。 - 暂时停止非卧床患者输注Elevitus的商业运输，直至完成召集专家小组分享数据并确定西罗莫司使用方案，以及获取FDA对在非卧床患者治疗方案中添加额外免疫抑制建议的反馈[5]。 西罗莫司作用机制和效果 - 安全事件可能与肝脏中的AV载体触发T细胞介导的免疫反应有关，西罗莫司是T细胞的有效抑制剂，可抑制T细胞活化，减轻免疫介导的肝脏炎症，降低肝损伤风险[8]。 - 非人类灵长类动物研究显示，西罗莫司可降低T细胞，抑制对AAV和转基因的免疫反应，有效调节或显著降低关键肝脏生物标志物（ALT、AST和胆红素）的升高，且不影响Elevitus在关键组织中的表达[9][10][11]。 对公司业绩的影响 - 暂停收入指引，计划在第二季度财报电话会议上提供指引更新，评估对2025年业绩和成本结构的影响，确保财务纪律[16]。 问答环节要点 - **患者情况**：去世患者为15岁非卧床患者，参与ENVISION研究，与首例病例有相似之处，剂量按体重计算，两例患者剂量不同但均按标准体重给药[21][44]。 - **风险范围**：急性肝衰竭信号仅在非卧床患者中出现，超900名患者接受治疗7年，此前无此严重信号，少数患者出现肝酶升高，对类固醇剂量适度增加反应迅速；活动状态可能是疾病进展的替代指标，制定非卧床患者方案后，医生可根据情况决定是否用于其他患者[39][40]。 - **西罗莫司使用**：有多个临床项目使用西罗莫司，公司希望与FDA积极讨论，确定非卧床研究和商业环境中的使用方案；西罗莫司有一定免疫抑制风险，需与专家小组和FDA确认具体方案和剂量[32][35][36]。 - **其他项目**：将与FDA讨论对4岁以下患者使用Elevitus的问题；LGMD项目疾病进展与DMD不同，未出现急性肝衰竭信号，但会在实施DMD免疫抑制方案时，以数据为依据考虑是否用于LGMD患者[96][98]。 其他重要但是可能被忽略的内容 - 公司有多个项目推进，除基因疗法外，还有肢带型肌营养不良（LGMD）项目、siRNA平台，今年有DM1和FSHD的概念验证、生物学验证读出结果，还有亨廷顿病等项目[75][76]。 - 2027年9月到期的票据有超两年管理时间，公司有多种途径解决，将积极管理费用，优先考虑成功率高、惠及患者多的项目，若EBITDA比率支持，可使用6亿美元循环信贷，财务状况良好[88][89]。 - 约30%的患者出现肝酶升高，非人类灵长类动物模型在评估急性肝衰竭的时间和持续时间方面相对较好[110][112]。

Analyst’s Disclosure:I/we have no stock, option or similar derivative position in any of the companies mentioned, and no plans to initiate any such positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article. This article is intended to provide informational content and should not be viewed as an exhaustive an ...

Thank you for reading my research on Seeking Alpha. If you want to learn even more about my method and how I discover these investment opportunities, please check out my subscription marketplace service, Compounding Healthcare , and sign up for a free trial.It has been nearly a year since my last Sarepta Therapeutics (NASDAQ: SRPT ) article , when the ticker was trading near $160 per share. At that time, Sarepta was riding high on the FDA’s landmark expanded approval ofHe is the leader of the investing grou ...