Limb-Girdle Muscular Dystrophy, Type 2E

▲8月15-16日 苏州2024生物医药创新者峰会 扫码报名  注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。 8月8日，小核酸龙头Sarepta Therapeutics发布了2024 H1业绩： 2024 H1净产品收入共计7.2亿美元（约51.54亿元），同比增长53%，总营收7.76亿美元，同比增长51%；研发费用3.8亿美元，同比下降22%；其中2024 Q2产品净收入共计3.61亿美元，同比增长51%；总营收3.63亿美元，同比增长39%。 Sarepta 提供了2025年产品净收入29亿至31亿美元的指导目标。 Sarepta已经开发并商业化了以下四款经批准用于治疗杜氏肌营养不良（DMD）的产品：Exondys 51（eteplirsen）、Vyondys 53（golodirsen）、Amondys 45（casimersen）和Elevidys，分别于2016年、2019年、2021年和2023年获FDA批准，分别用于外显子51、53和45跳跃的DMD患者和年龄在4-5岁之间患有DMD的非卧床儿童患者。 2024 H1，四款获批疗法的产品净收入总额为7.2亿美元；2024 Q2，产品净收入总额为3.605亿美元，同比增长51%。 PMO产品EXONDYS 51、VYONDYS 53和AMONDYS 45继续保持良好的业绩，上半年净产品收入4.64亿美元，同比下降1.3%；Q2 净产品收入2.39亿美元，去年同期基本持平。 Elevidys是一款基于腺相关病毒（AAV）载体的基因疗法，通过基因工程办法将编码微抗肌萎缩蛋白的靶基因递送到骨骼和心肌肌肉组织，以弥补抗肌萎缩蛋白缺失。自2023年6月推出以来销售额超2亿美元，Elevidys在2024 H1销售额2.56亿美元；2024 Q2销售额1.22亿美元，这为公司在2024 年下半年实现大幅增长奠定了良好的基础。 近期公司进展： 6月20日，美国FDA已批准扩大ELEVIDYS的适应症以包括4岁及以上的具有确认基因突变的杜氏肌营养不良症（DMD）患者。FDA为可行走的患者授予了常规批准，并为不可行走的患者授予了加速批准。此次适应症获批扩展，意味着该疗法将可应用于更广泛的DMD患者人群。 执行 ELEVIDYS 长期跟踪研究（ENDURE和EXPEDITION）：ENDURE是一项4期观察研究，将对接受ELEVIDYS治疗的患者进行长达10年的随访；EXPEDITION是一项3期研究，将招募约400名先前参加过ELEVIDYS临床试验的患者，对其进行长达5年的安全性和有效性一致性随访。 Sarepta 的合作伙伴罗氏公司宣布欧洲药品管理局（EMA）已开始审查ELEVIDYS 治疗杜兴氏症的上市许可申请（MAA），预计将于2025年获得批准。罗氏负责 ELEVIDYS在美国以外地区的商业化。 Sarepta 将在2024年10月8日至12日举办的第29届世界肌肉协会年会 (WMS 2024) 上展示其 SRP-9001项目的数据。 美国FDA批准SRP-9003（bidridistrogene xeboparvovec）快速通道资格认定：SRP-9003是一款在研基因疗法，用于治疗2E型肢带型肌营养不良症（LGMD2E，即β-肌聚糖病）。 研发管线 总结 国外布局杜氏肌营养不良症AAV基因疗法的公司除Sarepta、罗氏外，还有辉瑞、NS Pharma、RegenXBio等；国内金唯科、新芽基因也有布局，管线处于早期阶段。 参考资料：公司财报 版权声明：本文转自细胞基因治疗前沿，如不希望被转载的媒体或个人可与我们联系，我们将立即删除 8月15日  B馆B102  GLP-1及多肽论坛 会议日程 主持人：李湘，中肽生化创始人&董事长 09:00-09:25 多肽类药物在临床期间的CMC研究的多变性 苑字飞｜甘李药业，副总经理，首席技术官 09:25-09:50 GLP-RAs新药研发趋势-长效化、多靶点 陈小新｜众生睿创，CEO 09:50-10:15 创新型靶向GLP-1R/GIPR的双靶点激动剂的发现和临床转化 甘雨龙｜道尔生物，临床运营高级总监 10:15-10:40 iCVETide多肽新药发现平台技术及实用案例分享 孟广鹏｜成都诺和晟泰，创新药总监 10:40-11:05 多肽药物的质量控制策略 付玉清｜健元医药，高级研发总监 11:05-11:30 GLP类药物研发进展及商业化前景 章  华｜鸿运华宁，研发高级总监 11:30-11:55 GLP-1药物的差异化发展策略 谢  天｜甘李药业，医学事业部负责人 11:55-12:20 圆桌对话：多肽治疗领域扩展及出海机会 主持人：李湘 | 中肽生化创始人&董事长 陈小新｜众生睿创，CEO 孙汉栋｜九源基因，副总经理 徐洪岩 | 吉尔多肽，董事长 12:00-13:30 午餐 主持人：赖锦昌，美迪高，总经理 13:30-13:55 GLP-1，除了减重，还能卷什么？ 左亚军 | 仁会生物，总经理 13:55-14:20 司美格鲁肽与替尔泊肽杂质鉴定研究案例分享 刘国柱 | 长沙晨辰医药，创始人&董事长 14:20-14:45 新一代人源超长效GLP-1药物创新研发之路及关键临床数据解读 徐文洁｜银诺医药，高级副总裁&首席商务官 14:45-15:10 多肽实体文库高通量筛选体系及特色应用开发 林  定｜禾泰健宇生物，研发总监 15:10-15:35 伊匹乌肽-替代糖皮质甾体激素的广谱抗炎原创药 夏献民｜益承生物，董事长&总经理 15:35-16:00 EMPHASES多肽合成技术及在药物多肽合成中的应用 郑庆泉｜同隽医药，创始人&董事长 16:00-16:25 环肽创新药的发现：从长效棘白菌素到口服PCSK9抑制剂 原晨光｜祥根生物，联合创始人&CSO 8月16日  B馆B102  核酸药物开发前沿论坛 会议日程 主持人：冯冰，中肽生化，寡核苷酸研发资深总监 09:00-09:20 mRNA疫苗与mRNA药物 回爱民 | 惠正奇医药，创始人 09:20-09:40 靶向mRNA的碱基编辑技术开发与应用进展 忻   蓉 | 时夕生物，总经理 09:40-10:00 小核酸药物的临床前药代动力学研究 刘文玥 | 华西海圻，药代技术总监 10:00-10:20 小核酸干扰药物的递送和临床进展 张佩琢 | 吉玛基因，董事长 10:20-10:40 寡核苷酸药物中的杂质来源 冯  冰 | 中肽生化，寡核苷酸研发资深总监 10:40-11:00 小核酸药物的创新实践 苏晓晔 | 石药集团，核酸药物研究院负责人 11:00-11:20 Nucleosidic phosphoramidites: an optimized approach in their synthesis, quality evaluation and impurities characterization Beatrice Trucchi | Flamma S.p.A. - Italy R&D Project Manager  11:20-12:00 BH-RACETM新型核酸药物平台 胡  翰 | 滨会生物，副总裁 12:00-13:30 午餐 主持人：张力，北京瑞博奥医药科技有限公司，副总经理 13:30-13:50 小核酸干扰药物的递送和临床进展 袁旭东 | 艾康药业，CEO 13:50-14:10 治疗肝和肺部疾病的siRNA全新分子设计 黄  毅 | 国为医药，研究院副院长 14:10-14:30 全和诚核酸药物递送平台（拟定，以现场为准） 周经经 | 全和诚，研发副总经理 14:30-14:50 激发小核酸长效性潜力开发慢性病案例（拟定,以现场为准） 万金桥 | 先衍生物，总经理 14:50-15:10 AI驱动的高效mRNA疫苗开发 费才溢 | 澄实生物，vp&联合创始人 15:10-15:30 双靶向核酸适体技术 王雪强 | 中国科学院杭州医学研究所，研究员 15:30-15:50 寡核苷酸药物的DMPK的研究挑战和案例分享 陈桂英 | 宏韧医药，化学药物生物分析部门总监 扫码报名参会 【关于药融圈】 药融圈PRHub旨在帮助生物医药科技型企业进行品牌推广及商务拓展服务，针对客户的真实需求制定系统化解决方案，通过“翻译-降维-场景化”将客户的品牌信息以直白易懂的方式被公众知悉，同时在流量渠道覆盖100万+垂直用户基础上实现合作目的，帮助合作伙伴完成从品牌开始到商务为终的闭环营销服务。我们已经完成了数十场线下1000人规模的生物医药研发类会议，涵盖小分子新药，大分子新药，改良型新药，BD跨境交易等多个领域，服务了百余家上市/独角兽/生物技术/制药企业。

CAMBRIDGE, Mass.--( BUSINESS WIRE )--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the second quarter 2024. “ The second quarter of 2024 represents the most significant achievement in the advancement of medicine for Duchenne since researchers identified that lack of dystrophin was the underlying cause of Duchenne in 1986. Based on a wealth of compelling clinical evidence, the FDA broadened access to our gene therapy ELEVIDYS for all patients at least 4 years of age, with a traditional approval for ambulatory patients and accelerated approval for all non-ambulatory patients. We look forward to reviewing the comprehensive data supporting the safety and efficacy of ELEVIDYS at the 29 th Annual Congress of the World Muscle Society taking place in October, including muscle and cardiac MRI data and other biomarker results showing improvement in muscle health of treated patients,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. “ Sarepta is second to no other organization in the world in its ability to launch Duchenne therapies and support the community with education, access and reimbursement. We were well prepared for all aspects of this broadened launch and all signals thus far exceed even our optimistic expectations. To that point, second quarter total net product revenues across our four approved therapies were $360.5 million, a 51% increase over the same quarter last year. Our PMO products - EXONDYS 51, VYONDYS 53 and AMONDYS 45 - continue to perform, contributing $238.8 million in net product revenues. ELEVIDYS achieved $121.7 million in net product revenue, which positions us well for significant growth in the latter half of 2024 as families complete the 3-to-5-month process from enrollment form to infusion. Tracking our launch beyond 2024, based on our early launch signals and continuing performance of our PMOs, we anticipate that net product revenues in 2025 will be in the range of $2.9 to $3.1 billion.” Second Quarter 2024 and Recent Developments: U.S. FDA expands labeled indication for ELEVIDYS : On June 20, 2024, Sarepta announced U.S. Food and Drug Administration (FDA) approval of an expansion to the labeled indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age. Confirming the functional benefits, the FDA granted traditional approval for ambulatory patients. The FDA granted accelerated approval for non-ambulatory patients. Continued approval for non-ambulatory Duchenne patients may be contingent upon verification of clinical benefit in a confirmatory trial. Consistent with the accelerated approval pathway, Sarepta has committed to conduct and submit the results of a randomized, controlled trial to verify and confirm the clinical benefit of ELEVIDYS in patients with Duchenne who are non-ambulatory. ENVISION (Study SRP-9001-303), a global, randomized, double-blind, placebo-controlled Phase 3 study of ELEVIDYS in non-ambulatory and older ambulatory individuals with Duchenne, is underway and intended to serve as this postmarketing requirement. Sarepta’s partner Roche announced that the European Medicines Agency (EMA) has initiated review of the ELEVIDYS marketing authorization application (MAA) for the treatment of Duchenne: On June 24, 2024, Roche announced that the EMA has initiated the review of the MAA for ELEVIDYS for the treatment of ambulatory patients ages 3 to 7 years and is expecting approval in 2025. Roche is responsible for commercialization of ELEVIDYS outside of the United States. Sarepta to present data from its SRP-9001 program at the 29 th Annual Congress of the World Muscle Society (WMS 2024): In October, the Company will share data from its SRP-9001 program at WMS 2024, taking place Oct. 8-12, 2024, in Prague, Czechia. The full program is available at: https://www.wms2024.com/page/programme . This list does not include Sarepta's encore or pipeline presentations nor does it include late-breaker submissions. The following have been accepted as regular submissions for SRP-9001: Presentation # Title Date, Time 19O Muscle MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1 Oct. 12, 2024 1:45-2:00 AM EDT 7:45-8:00 AM CET 428P Cardiac MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1 Oct. 9, 2024 11:15 AM-12:15 PM EDT 5:15-6:15 PM CET 424P Micro-dystrophin expression and safety with delandistrogene moxeparvovec gene therapy for DMD in a broad population: Phase 1b trial (ENDEAVOR) 425P Five-year outcomes with delandistrogene moxeparvovec in patients with Duchenne Muscular Dystrophy (DMD): a phase 1/2a study Execution on the ELEVIDYS long-term follow-up studies: The long-term follow-up studies for ELEVIDYS include ENDURE, a Phase 4 observational study that will follow individuals treated with ELEVIDYS for up to 10 years, and EXPEDITION, a Phase 3 study enrolling approximately 400 patients who were previously enrolled in ELEVIDYS clinical trials and followed for consistent safety and efficacy measures for up to 5 years. U.S. FDA grants Fast Track designation to SRP-9003 (bidridistrogene xeboparvovec): The Company announced that they received Fast Track designation for SRP-9003 an investigational gene therapy being developed for the treatment of limb-girdle muscular dystrophy Type 2E (LGMD2E/R4), or beta sarcoglycanopathy. 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, which is critically important for patients with LGMD2E/R4, many of whom die from pulmonary or cardiac complications. The Fast Track designation is a process designed to facilitate the development and expedited review of drugs that treat serious conditions and fill unmet medical needs. Jerry R. Mendell, M.D., named to TIME100 Health inaugural list of 2024 most influential people in global health: TIME named renown neuromuscular researcher, physician and gene therapy pioneer Jerry R. Mendell, M.D., to the inaugural 2024 TIME100 Health, a list of 100 individuals who most influenced global health this year. Dr. Mendell was recognized for his lifetime commitment to neuromuscular disease and achievements in genetic medicine that propelled a new age of treatment for several genetic diseases, including Duchenne muscular dystrophy. The TIME100 Health list recognizes the impact, innovation, and achievement of the world’s most influential individuals in health. TIME selected a community of leaders across industries dedicated to creating tangible and credible change for a healthier population, including pioneering scientists who are steering the evolution of global health in 2024. Conference Call 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 . 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. Q2 2024 Financial Highlights 1 For the Three Months Ended June 30, 2024 2023 QTD Change (in millions, except for per share amounts) $ % Total Revenues $ 362.9 $ 261.2 101.7 39 % Operating (loss) income: GAAP $ (0.7 ) $ (133.5 ) 132.8 (99 %) Non-GAAP $ 57.9 $ (75.5 ) 133.4 NM* Net income (loss): GAAP $ 6.5 $ (23.9 ) 30.4 NM* Non-GAAP $ 46.7 $ (89.9 ) 136.6 NM* Diluted earnings (loss) per share: GAAP $ 0.07 $ (0.27 ) 0.34 NM* Non-GAAP $ 0.44 $ (1.01 ) 1.45 NM* For the Six Months Ended June 30, 2024 2023 YTD Change (in millions, except for per share amounts) $ % Total Revenues $ 776.4 $ 514.7 261.7 51 % Operating income (loss): GAAP $ 34.2 $ (271.6 ) 305.8 NM* Non-GAAP $ 141.6 $ (161.2 ) 302.8 NM* Net income (loss): GAAP $ 42.6 $ (540.7 ) 583.3 NM* Non-GAAP $ 124.9 $ (177.6 ) 302.5 NM* Diluted earnings (loss) per share: GAAP $ 0.44 $ (6.11 ) 6.55 NM* Non-GAAP $ 1.16 $ (2.01 ) 3.17 NM* *NM: not meaningful [1] For an explanation of our use of non-GAAP financial measures, please refer to the “Use of Non-GAAP Financial Measures” section later in this press release and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP measures, see the tables at the end of this press release. As of June 30, 2024 As of December 31, 2023 (in millions) Cash, cash equivalents, restricted cash and investments $ 1,476.1 $ 1,691.8 Revenues Total revenues increased by $101.7 million for the three months ended June 30, 2024, compared to the same period of 2023. The increase primarily reflects the product launch of ELEVIDYS in June 2023, partially offset by a $22.3 million decrease in the amortization of the single, combined performance obligation under our collaboration agreement with F. Hoffman-La Roche Ltd.'s (“Roche”), which was fully amortized as of December 31, 2023. Total revenues increased by $261.7 million for the six months ended June 30, 2024, compared to the same period of 2023. The increase primarily reflects the product launch of ELEVIDYS in June 2023, as well as the $48.0 million of collaboration revenue recognized related to an option declined by Roche to acquire the ex-US rights to a certain external, early stage Duchenne-specific program. Additionally, for the three and six months ended June 30, 2024, we recognized $2.4 million and $8.4 million, respectively, of contract manufacturing and other revenues associated with commercial ELEVIDYS supply delivered to Roche and royalty revenue received from Roche, with no similar activity for the same periods of 2023. Cost of sales (excluding amortization of in-licensed rights) Cost of sales (excluding amortization of in-license rights) increased by $10.4 million and $26.0 million for the three and six months ended June 30, 2024, compared with the same periods of 2023. The increase in both periods primarily reflects the product launch of ELEVIDYS in June 2023. Operating expenses and others Research and development expenses decreased by $62.2 million and $107.5 million for the three and six months ended June 30, 2024, compared with the same periods of 2023. The decreases in research and development expenses for both periods primarily reflect capitalization of commercial batches of ELEVIDYS manufactured after its approval in June 2023. Non-GAAP research and development expenses decreased by $58.3 million and $100.9 million for the three and six months ended June 30, 2024, compared with the same periods of 2023. Selling, general and administrative expenses increased by $20.2 million and $36.5 million for the three and six months ended June 30, 2024, compared with the same periods of 2023. The increase in selling, general and administrative expenses for both periods is primarily driven by professional services used for the launch of ELEVIDYS and ongoing litigation matters, the timing of charitable contributions, as well as the achievement of performance conditions related to certain Performance Stock Units. Non-GAAP selling, general and administrative expenses increased by $15.7 million and $32.9 million for the three and six months ended June 30, 2024, compared with the same periods of 2023. For the three months ended June 30, 2024, other income, net decreased by $104.6 million, compared with the same period of 2023, which primarily reflects a $102.0 million decrease in the gain on sale of a Priority Review Voucher (“PRV”) period over period. For the six months ended June 30, 2024, other income (loss), net increased by $276.5 million compared with the same period of 2023, which primarily reflects a $387.3 million loss on debt extinguishment that occurred during the six months ended June 30, 2023, offset by a $102.0 million gain on sale of a PRV, with no similar activities in 2024. Income tax expense for the three months ended June 30, 2024 and 2023, was approximately $7.1 million and $9.4 million, respectively. Income tax expense for the six months ended June 30, 2024 and 2023, was approximately $12.4 million and $13.4 million, respectively. Income tax expense for all periods presented primarily relates to state, federal and foreign income taxes. Use of Non-GAAP Measures In addition to the GAAP financial measures set forth in this press release, we have included certain non-GAAP measurements. The non-GAAP income (loss) is defined by us as GAAP net income (loss) excluding interest income, net, depreciation and amortization expense, stock-based compensation expense, the estimated income tax impact of each pre-tax non-GAAP adjustment and other items. The non-GAAP earnings (loss) per share is defined by us as non-GAAP income (loss), as defined previously, divided by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding. The non-GAAP earnings per share is calculated using diluted shares whereas the non-GAAP net loss per share is calculated using basic shares as all other instruments are anti-dilutive. The non-GAAP operating income (loss) is defined by us as GAAP operating income (loss) excluding depreciation and amortization expense, stock-based compensation expense and other items. Non-GAAP research and development expenses are defined by us as GAAP research and development expenses excluding depreciation and amortization expense, stock-based compensation expense and other items. Non-GAAP selling, general and administrative expenses are defined by us as GAAP selling, general and administrative expenses excluding depreciation expense, stock-based compensation expense and other items. 1. Interest, depreciation and amortization Interest income, net amounts can vary substantially from period to period due to changes in cash and debt balances and interest rates driven by market conditions outside of our operations. Depreciation expense can vary substantially from period to period as the purchases of property and equipment may vary significantly from period to period and without any direct correlation to our operating performance. Amortization expense primarily associated with patent costs are amortized over a period of several years after acquisition or patent application or renewal. 2. Stock-based compensation expenses Stock-based compensation expenses represent non-cash charges related to equity awards we have granted. Although these are recurring charges to operations, we believe the measurement of these amounts can vary substantially from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within our control. Therefore, we believe that excluding these charges facilitates comparisons of our operational performance in different periods. 3. Other items We evaluate other items of expense and income on an individual basis. We take into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relate to our ongoing business operations, and (c) whether we expect the items to continue or occur on a regular basis. These other items include impairment of strategic investments, change in fair value of contingent consideration, net, gain from sale of the PRV and loss on debt extinguishment and may include other items that fit the above characteristics in the future. We exclude from our non-GAAP results the impairment of any strategic investments as it is a non-cash item and is not considered to be a normal operating expense due to the variability of amount and lack of predictability as to the occurrence and/or timing of such impairments. We exclude from our non-GAAP results the loss on debt extinguishment, which is considered to be an infrequent and non-cash event as it is associated with a distinct financing decision and is not indicative of the performance of our core operations, which accordingly, would make it difficult to compare our results to peer companies that also provide non-GAAP disclosures. We exclude from our non-GAAP results the gain from sale of the PRV obtained as a result of the FDA accelerated approval of ELEVIDYS in June 2023 as it is a non-recurring event. We exclude from our non-GAAP results the change in fair value of contingent consideration, net related to regulatory-related contingent payments meeting the definition of a derivative to Myonexus selling shareholders as well as to academic institutions under separate license agreements as it is a non-cash item and is not considered to be normal operating expenses due to its variability of amounts and lack of predictability as to occurrence and/or timing. Beginning in the fourth quarter of 2023, amortization of in-licensed rights (formerly included within depreciation and amortization expense) and income tax (benefit) expense are no longer excluded from the non-GAAP results. We now include the income tax effect of adjustments, which represents the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable effective income tax rate. Non-GAAP financial results for the for the three and six months ended June 30, 2023 have been updated to reflect this change for comparability. We use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. We also believe these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating our performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of our financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP other income and loss adjustments, non-GAAP operating (loss) income, non-GAAP net income (loss), and non-GAAP diluted net earnings (loss) per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “ Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.” About EXONDYS 51 EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion, or “skipping”, of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. EXONDYS 51 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. Important Safety Information About EXONDYS 51 Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy. Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 mg or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended. The most common adverse reactions from observational clinical studies (N=163) seen in greater than 10% of patients were headache, cough, rash, and vomiting. For further information, please see the full Prescribing Information . About VYONDYS 53 VYONDYS 53 (golodirsen) uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 53 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. VYONDYS 53 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. Important Safety Information for VYONDYS 53 CONTRAINDICATIONS: VYONDYS 53 is contraindicated in patients with a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53. Anaphylaxis has occurred in patients receiving VYONDYS 53. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions : Hypersensitivity reactions, including anaphylaxis, rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in VYONDYS 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion, interrupting, or discontinuing the VYONDYS 53 therapy and monitor until the condition resolves. VYONDYS 53 is contraindicated in patients with a history of a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53. Kidney Toxicity: Kidney toxicity was observed in animals who received golodirsen. Although kidney toxicity was not observed in the clinical studies with VYONDYS 53, the clinical experience with VYONDYS 53 is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VYONDYS 53. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein- to-creatinine ratio should be measured before starting VYONDYS 53. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting VYONDYS 53. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to- creatinine ratio every three months. Only urine expected to be free of excreted VYONDYS 53 should be used for monitoring of urine protein. Urine obtained on the day of VYONDYS 53 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any VYONDYS 53 that is excreted in the urine and thus lead to a false positive result for urine protein. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation. ADVERSE REACTIONS: Adverse reactions observed in at least 20% of treated patients and greater than placebo were (VYONDYS 53, placebo): headache (41%, 10%), pyrexia (41%, 14%), fall (29%, 19%), abdominal pain (27%, 10%), nasopharyngitis (27%, 14%), cough (27%, 19%), vomiting (27%, 19%), and nausea (20%, 10%). Other adverse reactions that occurred at a frequency greater than 5% of VYONDYS 53-treated patients and at a greater frequency than placebo were: administration site pain, back pain, pain, diarrhea, dizziness, ligament sprain, contusion, influenza, oropharyngeal pain, rhinitis, skin abrasion, ear infection, seasonal allergy, tachycardia, catheter site related reaction, constipation, and fracture. Other adverse events may occur. To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . For further information, please see the full Prescribing Information . About AMONDYS 45 AMONDYS 45 (casimersen) uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 45 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. AMONDYS 45 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. AMONDYS 45 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. Important Safety Information for AMONDYS 45 CONTRAINDICATIONS : AMONDYS 45 is contraindicated in patients with a known serious hypersensitivity to casimersen or any of the inactive ingredients in AMONDYS 45. Instances of hypersensitivity including angioedema and anaphylaxis have occurred. WARNINGS AND PRECAUTIONS Hypersensitivity: Hypersensitivity reactions, including angioedema and anaphylaxis, have occurred in patients who were treated with AMONDYS 45. If a hypersensitivity reaction occurs, institute appropriate medical treatment, and consider slowing the infusion, interrupting, or discontinuing the AMONDYS 45 infusion and monitor until the condition resolves. AMONDYS 45 is contraindicated in patients with known serious hypersensitivity to casimersen or to any of the inactive ingredients in AMONDYS 45. Kidney Toxicity : Kidney toxicity was observed in animals who received casimersen. Although kidney toxicity was not observed in the clinical studies with AMONDYS 45, kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking AMONDYS 45. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting AMONDYS 45. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting AMONDYS 45. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio (UPCR) every three months. Only urine expected to be free of excreted AMONDYS 45 should be used for monitoring of urine protein. Urine obtained on the day of AMONDYS 45 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any AMONDYS 45 that is excreted in the urine and thus lead to a false positive result for urine protein. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation. Adverse Reactions: Adverse reactions occurring in at least 20% of patients treated with AMONDYS 45 and at least 5% more frequently than in the placebo group were (AMONDYS 45, placebo): upper respiratory infections (65%, 55%), cough (33%, 26%), pyrexia (33%, 23%), headache (32%, 19%), arthralgia (21%, 10%), and oropharyngeal pain (21%, 7%). Other adverse reactions that occurred in at least 10% of patients treated with AMONDYS 45 and at least 5% more frequently than in the placebo group were: ear pain, nausea, ear infection, post-traumatic pain, and dizziness and light-headedness. Other adverse events may occur. To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . For further information, please see the full Prescribing Information . About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin (noted hereafter as “micro-dystrophin”) in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. 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. 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). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended. Immune-mediated Myositis: 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. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. 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. Myocarditis : Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. 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. Advise patients to contact a physician immediately if they experience cardiac symptoms. Preexisting Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia. Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information . About Sarepta Therapeutics 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. 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 LinkedIn , X (formerly Twitter) , Instagram and Facebook . Forward-Looking Statements 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. 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, financial performance and projections, business plans, market opportunities, priorities and research and development programs, products and technologies; the potential benefits of our technologies and scientific approaches; our expectation that net product revenues in 2025 will be in the range of $2.9 to $3.1 billion; the potential for our partner Roche to receive approval from the EMA in 2025 for ELEVIDYS for the treatment of ambulatory patients ages 3 to 7 years; and expected milestones and plans, including reviewing the comprehensive data supporting the safety and efficacy of ELEVIDYS at the 29 th Annual Congress of the World Muscle Society taking place in October. 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: we may not be able to comply with all FDA post-approval commitments and requirements with respect to our products in a timely manner or at all; 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; certain programs may never advance in the clinic or may be discontinued for a number of reasons, including regulators imposing a clinical hold and us suspending or terminating clinical research or trials; 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, changes in coverage and reimbursement policies of health plans and health insurers, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2023, and our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review. Internet Posting of Information We routinely post information that may be important to investors in the '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. Sarepta Therapeutics, Inc. Condensed Consolidated Statements of Income (Loss) (unaudited, in thousands, except per share amounts) For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 Revenues: Products, net $ 360,548 $ 238,988 $ 720,032 $ 470,483 Collaboration and other 2,383 22,250 56,363 44,255 Total revenues 362,931 261,238 776,395 514,738 Cost and expenses: Cost of sales (excluding amortization of in-licensed rights) 44,545 34,124 95,104 69,141 Research and development 179,690 241,890 380,086 487,569 Selling, general and administrative 138,796 118,564 265,799 229,278 Amortization of in-licensed rights 601 179 1,202 357 Total cost and expenses 363,632 394,757 742,191 786,345 Operating (loss) income (701 ) (133,519 ) 34,204 (271,607 ) Other income (loss), net: Other income, net 14,278 16,934 20,821 29,641 Gain from sale of Priority Review Voucher — 102,000 — 102,000 Loss on debt extinguishment — — — (387,329 ) Total other income (loss), net 14,278 118,934 20,821 (255,688 ) Income (loss) before income tax expense 13,577 (14,585 ) 55,025 (527,295 ) Income tax expense 7,117 9,355 12,446 13,400 Net income (loss) $ 6,460 $ (23,940 ) $ 42,579 $ (540,695 ) Earnings (loss) per share: Basic $ 0.07 $ (0.27 ) $ 0.45 $ (6.11 ) Diluted $ 0.07 $ (0.27 ) $ 0.44 $ (6.11 ) Weighted average number of shares of common stock used in computing earnings (loss) per share: Basic 94,618 88,743 94,305 88,466 Diluted 99,144 88,743 99,129 88,466 Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands, except per share amounts) For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 GAAP net income (loss) $ 6,460 $ (23,940 ) $ 42,579 $ (540,695 ) Interest income, net (14,010 ) (15,980 ) (29,741 ) (28,972 ) Depreciation and amortization expense* 8,118 10,613 16,261 21,740 Stock-based compensation expense 50,482 47,377 91,174 88,627 Change in fair value on contingent consideration — (800 ) 10,100 (800 ) Gain from sale of Priority Review Voucher — (102,000 ) — (102,000 ) Loss on debt extinguishment — — — 387,329 Impairment of strategic investments — — — 321 Income tax effect of adjustments** (4,389 ) (5,172 ) (5,472 ) (3,189 ) Non-GAAP net income (loss)** $ 46,661 $ (89,902 ) $ 124,901 $ (177,639 ) GAAP net earnings (loss) per share - diluted: $ 0.07 $ (0.27 ) $ 0.44 $ (6.11 ) Add: impact of GAAP to Non-GAAP adjustments $ 0.37 $ (0.74 ) $ 0.73 $ 4.10 Non-GAAP net earnings (loss) per share - diluted*** $ 0.44 $ (1.01 ) $ 1.16 $ (2.01 ) Weighted average number of shares of common stock used in computing diluted earnings (loss) per share:**** GAAP 99,144 88,743 99,129 88,466 Non-GAAP 107,245 88,743 107,230 88,466 *Beginning in the fourth quarter of 2023, depreciation and amortization excludes amortization of in-licensed rights. Non-GAAP financial results for the three and six months ended June 30, 2023, have been updated to reflect this change for comparability. **Beginning in the fourth quarter of 2023, income tax (benefit) expense is no longer excluded from the non-GAAP results. We have replaced this metric with income tax effect of adjustments, which represents the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable statutory income tax rate. Refer below for a reconciliation of effective tax rates. Non-GAAP financial results for the three and six months ended June 30, 2023, have been updated to reflect this change for comparability. ***Non-GAAP earnings per share is calculated using diluted shares whereas non-GAAP net loss per share is calculated using basic shares as all other instruments are anti-dilutive. ****The difference between the weighted average number of shares of common stock used in computing diluted GAAP and non-GAAP earnings per share for the three and six months ended June 30, 2024, is a result of the exclusion of the potential share settlement of the 2027 Notes from the GAAP earnings per share as the inclusion of such shares was anti-dilutive. For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 Total effective tax rate, GAAP 8.4 % (1.7 )% 22.6 % (2.5 )% Less: impact of GAAP to Non-GAAP adjustments (4.6 ) (6.2 ) (10.0 ) (7.8 ) Total effective tax rate, Non-GAAP 3.8 % (7.9 )% 12.6 % (10.3 )% Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands) For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 GAAP research and development expenses $ 179,690 $ 241,890 $ 380,086 $ 487,569 Stock-based compensation expense (19,806 ) (21,577 ) (36,079 ) (37,990 ) Depreciation and amortization expense (5,982 ) (8,134 ) (12,028 ) (16,685 ) Non-GAAP research and development expenses $ 153,902 $ 212,179 $ 331,979 $ 432,894 For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 GAAP selling, general and administrative expenses $ 138,796 $ 118,564 $ 265,799 $ 229,278 Stock-based compensation expense (30,676 ) (25,800 ) (55,095 ) (50,637 ) Depreciation expense (2,136 ) (2,479 ) (4,233 ) (5,055 ) Non-GAAP selling, general and administrative expenses $ 105,984 $ 90,285 $ 206,471 $ 173,586 For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 GAAP operating (loss) income $ (701 ) $ (133,519 ) $ 34,204 $ (271,607 ) Stock-based compensation expense 50,482 47,377 91,174 88,627 Depreciation and amortization expense 8,118 10,613 16,261 21,740 Non-GAAP operating income (loss) $ 57,899 $ (75,529 ) $ 141,639 $ (161,240 ) Sarepta Therapeutics, Inc. Condensed Consolidated Balance Sheets (unaudited, in thousands, except share and per share data) As of June 30, 2024 As of December 31, 2023 Assets Current assets: Cash and cash equivalents $ 383,622 $ 428,430 Short-term investments 1,076,852 1,247,820 Accounts receivable, net 359,997 400,327 Inventory 485,795 322,859 Manufacturing-related deposits and prepaids 302,627 102,181 Other current assets 74,743 77,714 Total current assets 2,683,636 2,579,331 Property and equipment, net 276,200 227,154 Right of use assets 124,001 129,952 Non-current inventory 204,691 191,368 Other non-current assets 135,729 136,771 Total assets $ 3,424,257 $ 3,264,576 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 107,417 $ 164,918 Accrued expenses 350,404 314,997 Deferred revenue, current portion 122,036 50,416 Current portion of long-term debt 91,505 105,483 Other current liabilities 17,128 17,845 Total current liabilities 688,490 653,659 Long-term debt 1,134,810 1,132,515 Lease liabilities, net of current portion 143,601 140,965 Deferred revenue, net of current portion 325,000 437,000 Contingent consideration 48,200 38,100 Other non-current liabilities 7,087 3,000 Total liabilities 2,347,188 2,405,239 Stockholders’ equity: Preferred stock, $0.0001 par value, 3,333,333 shares authorized; none issued and outstanding — — Common stock, $0.0001 par value, 198,000,000 shares authorized; 95,282,600 and 93,731,831 issued and outstanding at June 30, 2024, and December 31, 2023, respectively 10 9 Additional paid-in capital 5,481,723 5,304,623 Accumulated other comprehensive (loss) income, net of tax (1,030 ) 918 Accumulated deficit (4,403,634 ) (4,446,213 ) Total stockholders’ equity 1,077,069 859,337 Total liabilities and stockholders’ equity $ 3,424,257 $ 3,264,576

– Net product revenues for the fourth quarter 2023 totaled $365.1 million, a 55% increase over the same quarter of the prior year; 2023 net product revenues for the full-year 2023 totaled $1.1 billion, an increase of approximately 36% over the prior year – ELEVIDYS revenues for the quarter totaled $131.2 million and for full-year totaled $200.4 million CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc., (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2023. “We are pleased to report another strong quarter and year of performance serving the patient community. With our three approved PMO therapies, EXONDYS 51, VYONDYS 53 and AMONDYS 45, and supported by the successful launch of our gene therapy, ELEVIDYS, Sarepta’s net product revenue grew 55% in the fourth quarter and 36% for the full year when compared to 2022, as we posted $1.14 billion in full-year net product revenue and we achieved GAAP profitability in the fourth quarter 2023 after achieving profitability on a non-GAAP basis in the third quarter 2023,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. “2023 was arguably Sarepta’s most significant year in service of our mission to improve the lives of Duchenne patients. With the acceptance for review of our BLA supplement to expand the ELEVIDYS label, continued performance of our four approved therapies, and as we continue to advance our pipeline, 2024 offers the potential of being the most important year yet for families living with Duchenne and those invested in the improvement of patients’ lives.” Fourth Quarter 2023 and Recent Developments: Announced U.S. FDA acceptance of an efficacy supplement to expand the ELEVIDYS indication: The U.S. Food and Drug Administration (FDA) has accepted and filed Sarepta’s efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl). The goals of the efficacy supplement are twofold: To expand the labeled indication for ELEVIDYS as follows: “[ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene.” To convert the ELEVIDYS accelerated approval to a traditional approval. The FDA has granted the efficacy supplement a Priority Review with a review goal date of June 21, 2024. The Agency has also confirmed they are not planning to hold an advisory committee meeting to discuss the supplement. Submitted efficacy supplement to expand the ELEVIDYS label to include Duchenne muscular dystrophy patients without restriction to age or ambulatory status: Submitted an efficacy supplement to the BLA for ELEVIDYS (delandistrogene moxeparvovec-rokl) to expand its labeled indication as follows "[ELEVIDYS is indicated for] the treatment of DMD patients with a confirmed mutation in the DMD gene.” The efficacy supplement is supported by results from EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study in patients with Duchenne between the ages of 4 through 7 years and data from ENDEAVOR (Study SRP-9001-103), an open label clinical study in patients with Duchenne, that is enrolling patients ages 2 years and older. The supplement was submitted to the U.S. Food and Drug Administration (FDA) with a request for Priority Review. Sarepta has also completed the EMBARK postmarketing requirement (PMR) and submitted the PMR to FDA requesting conversion from accelerated approval to traditional approval. Announced positive data from Part B of MOMENTUM, a phase 2 study of SRP-5051 in patients with Duchenne muscular dystrophy amenable to skipping exon 51: Announced positive data from Part B of the MOMENTUM study (Study SRP-5051-201), a global, Phase 2, multi-ascending dose clinical trial of SRP-5051 (vesleteplirsen) that enrolled patients aged 8 to 21 years. SRP-5051 is a next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping. Data from Part B of MOMENTUM found that at the higher, target dose, approximately 30 mg/kg dosed every four weeks, SRP-5051 resulted in mean dystrophin expression of 5.17%, and mean exon skipping of 11.11% at 28 weeks (n=20). Consistent dystrophin expression was seen in ambulatory (4.76%, n=11) and non-ambulatory (5.67%, n=9) participants at 28 weeks. Hypomagnesemia has previously been identified in patients taking SRP-5051 and was managed and monitored through prophylactic magnesium supplementation as part of the study protocol. Initiated screening in EMERGENE, a phase 3 clinical study of SRP-9003 for the treatment of limb-girdle muscular dystrophy type 2E/R4: Announced that screening is underway in Study SRP-9003-301, also known as EMERGENE. Study 9003-301 is a Phase 3, multi-national, open-label study of SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy Type 2E (LGMD2E/R4), or beta sarcoglycanopathy. EMERGENE will enroll 15 participants (ambulatory and non-ambulatory), aged 4 and older, and uses commercially representative process SRP-9003 material. The EMERGENE study design incorporates a six-month natural history lead-in. The primary endpoint is expression of beta-sarcoglycan 60 days after dosing. Other endpoints include functional measures through month 60 and safety. Conference Call The event will be webcast live under the investor relations section of Sarepta's website at 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. 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. Financial Results For the three months ended December 31, 2023, the Company reported a GAAP net income of $45.7 million, or $0.49 per basic and $0.47 per diluted share, compared to a GAAP net loss of $109.2 million reported for the same period of 2022, or $1.24 per basic and diluted share. The non-GAAP net income for the three months ended December 31, 2023 was $86.6 million, or $0.82 per diluted share, compared to a non-GAAP net loss of $53.6 million, or $0.61 per diluted share for the same period of 2022. For the twelve months ended December 31, 2023, the Company reported a GAAP net loss of $536.0 million, or $5.80 per basic and diluted share, compared to a GAAP net loss of $703.5 million reported for the same period of 2022, or $8.03 per basic and diluted share. The non-GAAP net loss for the twelve months ended December 31, 2023 was $59.5 million, or $0.64 per diluted share, compared to a non-GAAP net loss of $290.4 million, or $3.32 per diluted share for the same period of 2022. Revenues For the three months ended December 31, 2023, the Company recorded total revenues of $396.8 million, which consist of net product revenues and collaboration and other revenues, compared to total revenues of $258.4 million for the same period of 2022, an increase of $138.4 million. For the twelve months ended December 31, 2023, the Company recorded total revenues of $1,243.3 million, compared to total revenues of $933.0 million for the same period of 2022, an increase of $310.3 million. The increases primarily reflect increasing demand for EXONDYS 51, AMONDYS 45 and VYONDYS 53 (collectively, the “PMO Products”), as well as $131.2 million and $200.4 million of net product revenues associated with sales of ELEVIDYS during the three and twelve months ended December 31, 2023, respectively, after its approval in June 2023. For the three months ended December 31, 2023, the Company recorded net product revenues of $365.1 million, compared to net product revenues of $235.9 million for the same period of 2022, an increase of $129.2 million. For the twelve months ended December 31, 2023, the Company recorded net product revenues of $1,144.9 million, compared to net product revenues of $843.8 million for the same period of 2022, an increase of $301.1 million. The increases primarily reflect increasing demand for PMO Products, as well as $131.2 million and $200.4 million of net product revenues associated with sales of ELEVIDYS during the three and twelve months ended December 31, 2023, respectively. For the three and twelve months ended December 31, 2023, the Company recognized $31.7 million and $98.5 million of collaboration and other revenues, respectively. For the three and twelve months ended December 31, 2022, the Company recognized $22.5 million and $89.2 million of collaboration revenue, respectively. For all periods presented, collaboration and other revenues primarily relate to the F. Hoffman-La Roche Ltd. (Roche) collaboration arrangement. For the three and twelve months ended December 31, 2023, the Company recognized $9.2 million of contract manufacturing collaboration revenue associated with multiple batches of commercial ELEVIDYS supply delivered to Roche, with no similar activity for the three and twelve months ended December 31, 2022. Cost and Expenses Cost of sales (excluding amortization of in-licensed rights) For the three months ended December 31, 2023, cost of sales (excluding amortization of in-licensed rights) was $44.2 million, compared to $30.8 million for the same period of 2022, an increase of $13.4 million. The increase in the three months ended December 31, 2023 primarily reflects increasing demand for the Company's PMO Products, an increase in royalty payments due to ELEVIDYS sales in 2023 with no similar activity in 2022 and write-offs of certain batches of the Company's products not meeting the Company's quality specifications for the three months ended December 31, 2023, with no similar activity for the three months ended December 31, 2022. For the twelve months ended December 31, 2023, cost of sales (excluding amortization of in-licensed rights) was $150.3 million, compared to $140.0 million for the same period of 2022, an increase of $10.3 million. The change for the twelve months ended December 31, 2023 primarily reflects increasing demand for the Company's PMO Products, partially offset by a decrease in write-offs of certain batches of the Company's products not meeting the Company's quality specifications for the twelve months ended December 31, 2023, as compared to the same period of 2022, as well as a decrease in royalty payments due to changes in the BioMarin Pharmaceuticals, Inc. (BioMarin) royalty terms. Research and development Research and development expenses were $195.5 million for the three months ended December 31, 2023, compared to $213.8 million for the same period of 2022, a decrease of $18.3 million. The decrease in research and development expenses primarily reflects the following: $57.6 million decrease in manufacturing expenses primarily due to an increase in capitalization of commercial batches of ELEVIDYS manufactured after its approval in June 2023; $16.9 million decrease in up-front, milestone and other expenses primarily due to timing and costs related to the execution of certain research and license agreements and achievement of certain milestones year over year; $3.5 million decrease in research and other expenses primarily driven by a decrease in collaboration cost-sharing expenses related to Genethon's micro-dystrophin drug candidate; $1.6 million increase in pre-clinical expenses primarily due to an increase in toxicology study activity across multiple gene therapy and PPMO platforms; $1.9 million increase in professional service expenses primarily due to clinical consulting fees for various clinical trials sponsored by the Company; $3.2 million increase in stock-based compensation expense primarily due to the achievement of performance conditions related to certain shares with performance conditions (PSUs) during the twelve months ended December 31, 2023 with continuing vesting requirements related to a service condition, as well as changes in headcount and the value of stock awards; $4.8 million increase in facility- and technology-related expenses primarily due to the Company’s continuing expansion efforts; $7.9 million increase in compensation and other personnel expenses primarily due to changes in headcount; $12.0 million increase in clinical trial expenses primarily due to an increased patient enrollment and site activation for the Company's MOMENTUM and EXPEDITION programs, as well as additional PPMO clinical trials; and $28.2 million decrease in the offset to expense associated with a collaboration reimbursement from Roche primarily due to a decrease in reimbursed cost related to the minimum purchase requirements under the gene therapy manufacturing and supply agreement (the Thermo Agreement) with Thermo Fisher Scientific, Inc. (Thermo) for the three months ended December 31, 2022, with no similar activity in 2023, partially offset by the continuing development of the Company's SRP-9001 gene therapy programs. Research and development expenses were $877.4 million for the twelve months ended December 31, 2023, compared to $877.1 million for the same period of 2022, a slight increase of $0.3 million. The increase in research and development expenses primarily reflects the following: $51.5 million increase in clinical trial expenses primarily due to an increased patient enrollment and site activation for the Company's MIS51ON, MOMENTUM, ENVISION, EMERGENE and EXPEDITION programs, as well as additional PPMO clinical trials; $38.8 million increase in compensation and other personnel expenses primarily due to changes in headcount; $21.2 million increase in stock-based compensation expense primarily due to changes in headcount and the value of stock awards, as well as the achievement of performance conditions related to certain PSUs during the twelve months ended December 31, 2023 with continuing vesting requirements related to a service condition; $18.3 million increase in facility- and technology-related expenses primarily due to the Company’s continuing expansion efforts; $12.0 million increase in research and other expenses primarily driven by an increase in sponsored research with academic institutions during the twelve months ended December 31, 2023 and an increase in collaboration cost-sharing expenses related to Genethon's micro-dystrophin drug candidate; $9.7 million increase in professional service expenses primarily related to the launch of ELEVIDYS prior to its regulatory approval in June 2023; $3.1 million increase in pre-clinical expenses primarily due to an increase in toxicology study activity across multiple gene therapy and PPMO platforms; $21.9 million decrease in up-front, milestone and other expenses, primarily due to timing and costs related to the execution of certain research and license agreements and achievement of certain milestones year over year; $143.7 million decrease in manufacturing expenses primarily due to the capitalization of commercial batches of ELEVIDYS manufactured after its approval in June 2023 and a decrease of $54.0 million related to the minimum purchase requirements under the Thermo Agreement for the twelve months ended December 31, 2022, with no similar activity in the same period of 2023; and $11.3 million decrease in the offset to expense associated with a collaboration reimbursement from Roche primarily due to a decrease in reimbursed cost related to the minimum purchase requirements under the Thermo Agreement for the twelve months ended December 31, 2022, with no similar activity in the same period of 2023, partially offset by the continuing development of the Company's SRP-9001 gene therapy programs. Non-GAAP research and development expenses were $165.1 million and $186.8 million for the three months ended December 31, 2023 and 2022, respectively, a decrease of $21.7 million. Non-GAAP research and development expenses were $761.9 million and $784.1 million for the twelve months ended December 31, 2023 and 2022, respectively, a decrease of $22.2 million. Selling, general and administrative Selling, general and administrative expenses were $131.7 million for the three months ended December 31, 2023 compared to $120.5 million for the same period in 2022, an increase of $11.2 million. The increase in selling, general and administrative expenses primarily reflects the following: $18.4 million increase in professional service expenses primarily related to the launch of ELEVIDYS and ongoing litigation matters; $7.9 million increase in compensation and other personnel expenses primarily due to changes in headcount; $3.6 million increase in facility- and technology-related expenses primarily due to the Company’s continuing expansion efforts; $7.9 million decrease in stock-based compensation expense primarily related to the execution of the Chief Executive Officer (CEO) grant modification agreement in 2022, partially offset by the achievement of performance conditions related to certain PSUs during the twelve months ended December 31, 2023 with continuing vesting requirements related to a service condition, as well as changes in headcount and the value of stock awards; and $10.7 million decrease in other expenses primarily due to timing of charitable contributions. Selling, general and administrative expenses were $481.9 million for the twelve months ended December 31, 2023, compared to $451.4 million for the same period in 2022, an increase of $30.5 million. The increase in selling, general and administrative expenses primarily reflects the following: $60.9 million increase in professional service expenses primarily related to the launch of ELEVIDYS and ongoing litigation matters; $35.2 million increase in compensation and other personnel expenses primarily due to changes in headcount; $10.9 million increase in facility- and technology-related expenses primarily due to the Company’s continuing expansion efforts; $4.6 million decrease in other expenses primarily related to timing of charitable contributions; and $71.7 million decrease in stock-based compensation expense primarily related to the CEO grant modification agreement in 2022, partially offset by the achievement of performance conditions related to certain PSUs during the twelve months ended December 31, 2023 with continuing vesting requirements related to a service condition, as well as changes in headcount and the value of stock awards. Non-GAAP selling, general and administrative expenses were $105.7 million and $86.6 million for the three months ended December 31, 2023 and 2022, respectively, an increase of $19.1 million. Non-GAAP selling, general and administrative expenses were $372.0 million and $270.3 million for the twelve months ended December 31, 2023 and 2022, respectively, an increase of $101.7 million. Amortization of in-licensed rights For the three and twelve months ended December 31, 2023, the Company recorded amortization of in-licensed rights of approximately $0.8 million and $1.6 million, respectively. For the three and twelve months ended December 31, 2022, the Company recorded amortization of in-licensed rights of approximately $0.2 million and $0.7 million, respectively. This is related to the amortization of the in-licensed right assets recognized as a result of agreements the Company entered into with the University of Western Australia, Nationwide Children's Hospital, BioMarin and Parent Project Muscular Dystrophy in April 2013, December 2016, July 2017 and May 2018, respectively. Loss on debt extinguishment On November 14, 2017, the Company issued $570.0 million aggregate principal amount of senior convertible notes due on November 15, 2024 (2024 Notes). On March 2, 2023, the Company entered into separate, privately negotiated exchange agreements with certain holders of the outstanding 2024 Notes (Exchange Agreements). The Exchange Agreements resulted in a conversion of $313.5 million in aggregate principal value of the 2024 Notes held by the holders (2024 Notes Conversion). In connection with the 2024 Notes Conversion, the Company issued approximately 4.5 million shares of its common stock representing the agreed upon contractual conversion rate under the Exchange Agreements. The conversion was not pursuant to the conversion privileges included in the terms of the debt at issuance and therefore was accounted for as a debt extinguishment. The Company accounted for the debt extinguishment by recognizing the difference between the fair value of the shares of common stock transferred on the conversion date and the net carrying amount of the extinguished debt as a loss on debt extinguishment. The loss incurred on the extinguishment for the twelve months ended December 31, 2023 was $387.3 million, inclusive of $6.9 million in third-party debt conversion costs. Gain from Sale of Priority Review Voucher In June 2023, the Company entered into an agreement to sell the rare pediatric disease Priority Review Voucher (ELEVIDYS PRV) it received from the FDA in connection with the approval of ELEVIDYS for consideration of $102.0 million, with no commission costs. The transaction was not subject to the conditions set forth under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and closed during June 2023. The net proceeds were recorded as a gain from sale of the ELEVIDYS PRV during the twelve months ended December 31, 2023 as it did not have a carrying value at the time of the sale. Other income (expense), net For the three months ended December 31, 2023 and 2022, other income, net was $15.7 million and $5.5 million, respectively. The increase was primarily due to an increase in accretion of investment discount, net due to an increase in interest rates. For the twelve months ended December 31, 2023 and 2022, other income, net was $33.1 million and other expense, net was $28.3 million, respectively. The change is primarily due to an increase in accretion of investment discount, net and an increase in interest income due to the investment mix of the Company's investment portfolio and an increase in interest rates, as well as a reduction of interest expense incurred as a result of the repayment of the Company's December 2019 Term Loan in 2022, partially offset by the impairment of strategic investments and a decrease in gain (loss) on contingent consideration, net. Income tax (benefit) expense Income tax benefit for the three months ended December 31, 2023 was approximately $5.3 million. Income tax expense for the twelve months ended December 31, 2023 was $15.9 million. Income tax expense for the three and twelve months ended December 31, 2022 was $7.9 million and $13.5 million, respectively. Income tax (benefit) expense for the three and twelve months ended December 31, 2023 relates to state, foreign and federal income taxes, while income tax expense for the three and twelve months ended December 31, 2022 relates to state and foreign income taxes. Cash, Cash Equivalents, Restricted Cash and Investments The Company had approximately $1.7 billion in cash, cash equivalents, investments and long-term restricted cash as of December 31, 2023, compared to $2.0 billion as of December 31, 2022. This decrease is driven by cash used to fund the Company’s ongoing operations during 2023. Use of Non-GAAP Measures In addition to the GAAP financial measures set forth in this press release, the Company has included certain non-GAAP measurements. The non-GAAP income (loss) is defined by the Company as GAAP net income (loss) excluding interest (income) expense, net, depreciation and amortization expense, stock-based compensation expense, the estimated income tax impact of each pre-tax non-GAAP adjustment and other items. Non-GAAP research and development expenses are defined by the Company as GAAP research and development expenses excluding depreciation and amortization expense, stock-based compensation expense and other items. Non-GAAP selling, general and administrative expenses are defined by the Company as GAAP selling, general and administrative expenses excluding depreciation expense, stock-based compensation expense and other items. 1. Interest, depreciation and amortization Interest (income) expense, net amounts can vary substantially from period to period due to changes in cash and debt balances and interest rates driven by market conditions outside of the Company’s operations. Depreciation expense can vary substantially from period to period as the purchases of property and equipment may vary significantly from period to period and without any direct correlation to the Company’s operating performance. Amortization expense primarily associated with patent costs are amortized over a period of several years after acquisition or patent application or renewal. 2. Stock-based compensation expenses Stock-based compensation expenses represent non-cash charges related to equity awards granted by the Company. Although these are recurring charges to operations, the Company believes the measurement of these amounts can vary substantially from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within the Company’s control. Therefore, the Company believes that excluding these charges facilitates comparisons of the Company’s operational performance in different periods. 3. Other items The Company evaluates other items of expense and income on an individual basis. It takes into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relate to the Company’s ongoing business operations, and (c) whether the Company expects the items to continue or occur on a regular basis. These other items include impairment of strategic investments, loss (gain) on contingent consideration, net, gain from sale of the PRV and loss on debt extinguishment and may include other items that fit the above characteristics in the future. The Company excludes from its non-GAAP results the impairment of any strategic investments as it is a non-cash item and is not considered to be a normal operating expense due to the variability of amount and lack of predictability as to the occurrence and/or timing of such impairments. The Company excludes from its non-GAAP results the loss on debt extinguishment, which is considered to be an infrequent and non-cash event as it is associated with a distinct financing decision and is not indicative of the performance of the Company’s core operations, which accordingly, would make it difficult to compare the Company’s results to peer companies that also provide non-GAAP disclosures. The Company excludes from its non-GAAP results the loss (gain) on contingent consideration, net related to regulatory-related contingent payments meeting the definition of a derivative to Myonexus selling shareholders as well as to academic institutions under separate license agreements as it is a non-cash item and is not considered to be normal operating expenses due to its variability of amounts and lack of predictability as to occurrence and/or timing. The Company excludes from its non-GAAP results the gain from sale of the PRV obtained as a result of the FDA approval of ELEVIDYS in June 2023 as it is an infrequent event and is not indicative of the performance of the Company’s core operations, which accordingly would make it difficult to compare the Company’s results to peer companies that also provide non-GAAP disclosures. Beginning in the fourth quarter of 2023, amortization of in-licensed rights (formerly included within depreciation and amortization expense) and income tax (benefit) expense are no longer excluded from the non-GAAP results. The Company now includes the income tax effect of adjustments, which represents the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable effective income tax rate. Non-GAAP financial results for the fourth quarter and full-year 2022 have been updated to reflect this change for comparability. The Company uses these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. The Company also believes these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating the Company’s performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of the Company’s financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP other income and loss adjustments, non-GAAP net income (loss), and non-GAAP diluted net earnings (loss) per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.” About EXONDYS 51 EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. Important Safety Information About EXONDYS 51 Hypersensitivity reactions, bronchospasm, chest pain, cough, tachycardia, and urticaria have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy. Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 mg or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended. The most common adverse reactions from observational clinical studies (N=163) seen in greater than 10% of patients were headache, cough, rash, and vomiting. For further information, please see the full Prescribing Information. About VYONDYS 53 VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. Important Safety Information for VYONDYS 53 Hypersensitivity reactions, including rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in VYONDYS 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the VYONDYS 53 therapy. Kidney toxicity was observed in animals who received golodirsen. Although kidney toxicity was not observed in the clinical studies with VYONDYS 53, the clinical experience with VYONDYS 53 is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VYONDYS 53. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting VYONDYS 53. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months. Only urine expected to be free of excreted VYONDYS 53 should be used for monitoring of urine protein. Urine obtained on the day of VYONDYS 53 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any VYONDYS 53 that is excreted in the urine and thus lead to a false positive result for urine protein. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation. Adverse reactions observed in at least 20% of treated patients and greater than placebo were (VYONDYS 53, placebo): headache (41%, 10%), pyrexia (41%, 14%), fall (29%, 19%), abdominal pain (27%, 10%), nasopharyngitis (27%, 14%), cough (27%, 19%), vomiting (27%, 19%), and nausea (20%, 10%). Other adverse reactions that occurred at a frequency greater than 5% of VYONDYS 53-treated patients and at a greater frequency than placebo were: administration site pain, back pain, pain, diarrhea, dizziness, ligament sprain, contusion, influenza, oropharyngeal pain, rhinitis, skin abrasion, ear infection, seasonal allergy, tachycardia, catheter site related reaction, constipation, and fracture. For further information, please see the full Prescribing Information. About AMONDYS 45 AMONDYS 45 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. Important Safety Information for AMONDYS 45 CONTRAINDICATION: Known hypersensitivity to casimersen or any of the inactive ingredients. Instances of hypersensitivity including angioedema and anaphylaxis have occurred. WARNINGS AND PRECAUTIONS Hypersensitivity: Hypersensitivity reactions, including angioedema and anaphylaxis, have occurred in patients who were treated with AMONDYS 45. If a hypersensitivity reaction occurs, institute appropriate medical treatment, and consider slowing the infusion, interrupting, or discontinuing the AMONDYS 45 infusion and monitor until the condition resolves. Kidney Toxicity: Kidney toxicity was observed in animals who received casimersen. Although kidney toxicity was not observed in the clinical studies with AMONDYS 45, kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking AMONDYS 45. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting AMONDYS 45. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting AMONDYS 45. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio (UPCR) every three months. Only urine expected to be free of excreted AMONDYS 45 should be used for monitoring of urine protein. Urine obtained on the day of AMONDYS 45 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any AMONDYS 45 that is excreted in the urine and thus lead to a false positive result for urine protein. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation. Adverse Reactions: Adverse reactions occurring in at least 20% of patients treated with AMONDYS 45 and at least 5% more frequently than in the placebo group were (AMONDYS 45, placebo): upper respiratory infections (65%, 55%), cough (33%, 26%), pyrexia (33%, 23%), headache (32%, 19%), arthralgia (21%, 10%), and oropharyngeal pain (21%, 7%). Other adverse reactions that occurred in at least 10% of patients treated with AMONDYS 45 and at least 5% more frequently than in the placebo group were: ear pain, nausea, ear infection, post-traumatic pain, and dizziness and light-headedness. For further information, please see the full Prescribing Information. About ELEVIDYS (delandistrogene moxeparvovec-rokl) 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. This indication is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin observed in patients treated with ELEVIDYS. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Important Safety Information for ELEVIDYS CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Acute Serious Liver Injury: 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. 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. 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). 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. Immune-mediated Myositis: 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. 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. 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. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. 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. Advise patients to contact a physician immediately if they experience cardiac symptoms. Pre-existing Immunity against AAVrh74: 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. Perform baseline testing for the presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥ 5%) reported in clinical studies were vomiting, nausea, liver function test increased, pyrexia, and thrombocytopenia. For further information, please see the full Prescribing Information. About Sarepta Therapeutics 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 we currently have more than 40 programs in various stages of development. 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 or follow us on X (formerly Twitter), LinkedIn, Instagram and Facebook. Forward-Looking Statements 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. 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, financial performance and projections, business plans, market opportunities, priorities and research and development programs and technologies; the potential benefits of our technologies, product candidates and scientific approaches; the potential for the ELEVIDYS efficacy supplement to expand the approved label for ELEVIDYS and convert the ELEVIDYS accelerated approval to a traditional approval; the review goal date of June 21, 2024 for the ELEVIDYS efficacy supplement; our understanding that FDA does not plan to hold an advisory committee to discuss the ELEVIDYS supplement; the potential for 2024 being the most important year yet for families living with Duchenne and those invested in the improvement of patients’ lives; and expected plans and milestones. 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: we may not be able to comply with all FDA post-approval commitments and requirements with respect to our products in a timely manner or at all; because we are developing product candidates for the treatment of certain diseases in which there is little clinical experience and we are using new endpoints or methodologies, there is increased risk that the FDA, the EMA or other regulatory authorities may not consider the endpoints of our clinical trials to provide clinically meaningful results and that these results may be difficult to analyze; 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; certain programs may never advance in the clinic or may be discontinued for a number of reasons, including regulators imposing a clinical hold and us suspending or terminating clinical research or trials; 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 product candidates; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2023 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at . We encourage investors and potential investors to consult our website regularly for important information about us. Sarepta Therapeutics, Inc. Condensed Consolidated Statements of Operations (unaudited, in thousands, except per share amounts) For the Three Months Ended December 31, For the Twelve Months Ended December 31, 2023 2022 2023 2022 Revenues: Products, net $ 365,071 $ 235,933 $ 1,144,876 $ 843,769 Collaboration and other 31,710 22,494 98,460 89,244 Total revenues 396,781 258,427 1,243,336 933,013 Cost and expenses: Cost of sales (excluding amortization of in-licensed rights) 44,176 30,799 150,343 139,989 Research and development 195,517 213,804 877,387 877,090 Selling, general and administrative 131,700 120,478 481,871 451,421 Amortization of in-licensed rights 763 179 1,559 714 Total cost and expenses 372,156 365,260 1,511,160 1,469,214 Operating income (loss) 24,625 (106,833 ) (267,824 ) (536,201 ) Other income (loss), net: Other income (expense), net 15,746 5,527 33,055 (28,321 ) Loss on debt extinguishment — — (387,329 ) (125,441 ) Gain from sale of Priority Review Voucher — — 102,000 — Total other income (loss), net 15,746 5,527 (252,274 ) (153,762 ) Income (loss) before income tax (benefit) expense 40,371 (101,306 ) (520,098 ) (689,963 ) Income tax (benefit) expense (5,284 ) 7,938 15,879 13,525 Net income (loss) $ 45,655 $ (109,244 ) $ (535,977 ) $ (703,488 ) Net earnings (loss) per share: Basic $ 0.49 $ (1.24 ) $ (5.80 ) $ (8.03 ) Diluted $ 0.47 $ (1.24 ) $ (5.80 ) $ (8.03 ) Weighted-average number of shares of common stock used in computing basic net earnings (loss) per share 93,617 87,838 92,398 87,559 Weighted-average number of shares of common stock used in computing diluted net earnings (loss) per share 105,594 87,838 92,398 87,559 Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands, except per share amounts) For the Three Months Ended December 31, For the Twelve Months Ended December 31, 2023 2022 2023 2022 GAAP net income (loss) $ 45,655 $ (109,244 ) $ (535,977 ) $ (703,488 ) Interest (income) expense, net (17,469 ) (8,865 ) (64,034 ) 25,525 Depreciation and amortization expense* 10,609 10,374 42,838 41,150 Stock-based compensation expense 45,826 50,510 182,514 233,018 Impairment of strategic investments 2,500 2,575 30,321 2,575 Loss on debt extinguishment — — 387,329 125,441 Loss (gain) on contingent consideration, net — — 1,200 (6,700 ) Gain from sale of Priority Review Voucher — — (102,000 ) — Income tax effect of adjustments** (541 ) 1,075 (1,738 ) (7,915 ) Non-GAAP net income (loss)** $ 86,580 $ (53,575 ) $ (59,547 ) $ (290,394 ) Non-GAAP net earnings (loss) per share: Diluted*** $ 0.82 $ (0.61 ) $ (0.64 ) $ (3.32 ) Weighted-average number of shares of common stock used in computing earnings per share: Diluted 105,594 87,838 92,398 87,559 *Beginning in the fourth quarter of 2023, depreciation and amortization excludes amortization of in-licensed rights. Non-GAAP financial results for the fourth quarter and full-year 2022 have been updated to reflect this change for comparability. **Beginning in the fourth quarter of 2023, income tax (benefit) expense is no longer excluded from the non-GAAP results. The Company has replaced this metric with income tax effect of adjustments, which represents the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable effective income tax rate. Refer below for a reconciliation of effective tax rates. Non-GAAP financial results for the fourth quarter and full-year 2022 have been updated to reflect this change for comparability. ***Non-GAAP earnings per share is calculated using diluted shares whereas non-GAAP net loss per share is calculated using basic shares as all other instruments are anti-dilutive. For the Three Months Ended December 31, For the Twelve Months Ended December 31, 2023 2022 2023 2022 Total effective tax rate, GAAP (12.0 )% 5.5 % (3.1 )% (2.0 )% Less: impact of GAAP to Non-GAAP adjustments (0.7 ) (1.8 ) (39.0 ) (6.0 ) Total effective tax rate, Non-GAAP (12.7 )% 3.7 % (42.1 )% (8.0 )% Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands) For the Three Months Ended December 31, For the Twelve Months Ended December 31, 2023 2022 2023 2022 GAAP research and development expenses $ 195,517 $ 213,804 $ 877,387 $ 877,090 Stock-based compensation expense (22,174 ) (18,963 ) (82,489 ) (61,293 ) Depreciation and amortization expense (8,217 ) (8,013 ) (33,011 ) (31,713 ) Non-GAAP research and development expenses $ 165,126 $ 186,828 $ 761,887 $ 784,084 For the Three Months Ended December 31, For the Twelve Months Ended December 31, 2023 2022 2023 2022 GAAP selling, general and administrative expenses $ 131,700 $ 120,478 $ 481,871 $ 451,421 Stock-based compensation expense (23,652 ) (31,547 ) (100,025 ) (171,725 ) Depreciation expense (2,392 ) (2,361 ) (9,827 ) (9,437 ) Non-GAAP selling, general and administrative expenses $ 105,656 $ 86,570 $ 372,019 $ 270,259 Sarepta Therapeutics, Inc. Condensed Consolidated Balance Sheets (unaudited, in thousands, except share and per share data) As of December 31, 2023 2022 Assets Current assets: Cash and cash equivalents $ 428,430 $ 966,777 Short-term investments 1,247,820 1,022,597 Accounts receivable, net 400,327 214,628 Inventory 322,859 203,968 Other current assets 179,895 149,891 Total current assets 2,579,331 2,557,861 Property and equipment, net 227,154 180,037 Right of use assets 129,952 64,954 Non-current inventory 191,368 162,545 Other non-current assets 136,771 162,969 Total assets $ 3,264,576 $ 3,128,366 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 164,918 $ 95,875 Accrued expenses 314,997 418,996 Deferred revenue, current portion 50,416 89,244 Current portion of long-term debt 105,483 — Current portion of lease liabilities 17,845 15,489 Total current liabilities 653,659 619,604 Long-term debt 1,132,515 1,544,292 Lease liabilities, net of current portion 140,965 57,578 Deferred revenue, net of current portion 437,000 485,000 Contingent consideration 38,100 36,900 Other non-current liabilities 3,000 42 Total liabilities 2,405,239 2,743,416 Stockholders’ equity: Preferred stock, $0.0001 par value, 3,333,333 shares authorized; none issued and outstanding — — Common stock, $0.0001 par value, 198,000,000 shares authorized; 93,731,831 and 87,950,117 issued and outstanding at December 31, 2023 and 2022, respectively 9 9 Additional paid-in capital 5,304,623 4,296,841 Accumulated other comprehensive income (loss), net of tax 918 (1,664 ) Accumulated deficit (4,446,213 ) (3,910,236 ) Total stockholders’ equity 859,337 384,950 Total liabilities and stockholders’ equity $ 3,264,576 $ 3,128,366