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Sarepta Therapeutics 2026 JPMorgan Conference.pdf

上传人: 明**** 编号:1069768 2026-01-17 24页 4.39MB

1、SAREPTA THERAPEUTICS,INC.2026.ALL RIGHTS RESERVED.1 1Sarepta Therapeutics,Inc.(NASDAQ:SRPT)44th Annual J.P.Morgan Healthcare ConferenceSan Francisco,CaliforniaJANUARY 12,2026DOUG INGRAMChief Executive OfficerMAX,age 10Dosed with ELEVIDYS at age 5SAREPTA THERAPEUTICS,INC.2026.ALL RIGHTS RESERVED.2For

2、ward-looking statements In order to provide Sareptas investors with an understanding of its current results and future prospects,forward-looking statements will be made during this presentation.Any statements that are not statements of historical fact may be deemed to be forward-looking statements.W

3、ords such as“believe,”“anticipate,”“plan,”“expect,”“will,”“may,”“intend,”“prepare,”“look,”“potential,”“possible”and similar expressions are intended to identify forward-looking statements.These forward-looking statements include,without limitation,statements relating to our preliminary earnings,fina

4、ncial projections and future operations;our pipeline,priorities and strategies;ELEVIDYS and Cohort 8 of ENDEAVOR;the potential benefits and differentiation of our siRNA programs and TRiM platform;our ongoing and planned clinical trials;and expected plans and milestones,including meeting with FDA to

5、discuss a path to traditional approval of casimersen and golodirsen,and our expected near-term milestones in 2026 for various programs.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 factor

6、s include the following:our products or product candidates may be perceived as insufficiently effective,unsafe or may result in unforeseen adverse events;our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing app

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1. **财务表现**:2025年ELEVIDYS净收入8.99亿美元,PMO净收入9.66亿美元,总收入18.6亿美元;现金及投资9.54亿美元。 2. **核心产品**:ELEVIDYS治疗杜氏肌营养不良症(DMD),约80%可治疗人群未使用;PMO疗法(如EXONDYS 51)已治疗超1800名患者,依从性超90%。 3. **研发进展**: - ELEVIDYS ENDEAVOR队列8数据预计2026年下半年完成; - siRNA管线覆盖SCA1/3、FSHD1、DM1等疾病,SRP-1005(亨廷顿病)2026年上半年启动给药; - TRiM平台优化递送与安全性。 4. **里程碑**:2026年计划发布SRP-1001/1003(FSHD/DM1)I/II期数据,与FDA讨论casimersen/golodirsen传统审批路径。
**ELEVIDYS疗效如何?** **siRNA管线有何突破?** **2026年里程碑有哪些?**
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