Surabgene Lomparvovec

Patients treated with RGX-202 demonstrate consistent, robust microdystrophin expression and functional improvement compared to natural history in Phase I/II portion of AFFINITY DUCHENNE® trial supporting potential approval via the accelerated approval pathway REGENXBIO continues to enroll patients in the confirmatory trial First batches intended for commercial supply manufactured at in-house Manufacturing Innovation Center Capacity to produce up to 2,500 RGX-202 doses per year Topline pivotal data now expected in early Q2 2026 and BLA submission in mid-2026 ROCKVILLE, Md., Oct. 30, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the completion of enrollment in the AFFINITY DUCHENNE® pivotal trial of RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy, as well as the successful production of the first batches intended for commercial supply. "The Duchenne community urgently needs new treatment options that provide durable, safe outcomes and can meaningfully change the course of this degenerative disease. Completing this pivotal trial milestone and manufacturing in-house our first doses intended for commercial use bring us even closer to delivering RGX-202 as a potential best-in-class gene therapy for Duchenne patients with limited options," said Curran Simpson, President and Chief Executive Officer, REGENXBIO. "The differentiated therapeutic approach behind RGX-202, including our industry-leading product purity levels and novel construct with the C-Terminal domain, has resulted in the positive safety and efficacy profile, with consistent functional benefit seen in Phase I/II. With these highly encouraging results, we are committed to expanding our commercial supply and sharing topline pivotal data in early Q2 of next year." REGENXBIO continues enrolling ambulatory participants aged 1 year and above in the confirmatory trial. AFFINITY DUCHENNE® TRIAL The pivotal portion of the multi-center, open-label Phase I/II/III AFFINITY DUCHENNE trial completed enrollment of 30 participants in October 2025. To support accelerated approval, the primary pivotal endpoint is the proportion of participants whose RGX-202 microdystrophin expression is ≥10% at Week 12. Secondary endpoints include change from baseline on timed function tests in participants aged 4 years and older. Participants aged 1 to < 4 years will be evaluated using the Peabody Developmental Motor Scale-Third Edition (PDMS-3) and SV95C. In the Phase I/II portion of the trial, microdystrophin levels ranged from 20% to 122% in participants who received the pivotal dose. As of May 7, 2025, RGX-202 was well tolerated, with no serious adverse events (SAEs) or adverse events of special interest (AESIs) reported in the Phase I/II trial. Pivotal dose participants exceeded baseline-matched external natural history controls on all functional measures. Commercial Readiness REGENXBIO has manufactured the first batches of RGX-202 intended for commercial supply, supporting the company's expected approval and commercial launch in 2027, when the vast majority of the prevalent market is expected to be available. The company has also manufactured full supply of RGX-202 for the confirmatory trial. RGX-202 is manufactured at the REGENXBIO Manufacturing Innovation Center at the company's headquarters in Rockville, Md., using its NAVXpress® suspension-based manufacturing process. This proprietary, high-yielding, commercial-ready process has consistently enabled industry-leading product purity levels of more than 80% full capsids, the highest in Duchenne gene therapy. REGENXBIO can produce 2,500 doses of RGX-202 per year. About RGX-202 RGX-202 is a potential best-in-class investigational gene therapy designed for improved function and outcomes in Duchenne. RGX-202 is the only gene therapy approved or in late-stage development for Duchenne with a differentiated microdystrophin construct that encodes key regions of naturally occurring dystrophin, including the C-Terminal (CT) domain. Additional design features such as codon optimization may potentially improve gene expression, increase protein translation efficiency and reduce immunogenicity. RGX-202 is designed to support the delivery and targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV® AAV8 vector and a well-characterized muscle-specific promoter (Spc5-12). RGX-202 is manufactured by REGENXBIO using its proprietary, high-yielding NAVXpress® suspension-based platform process. ABOUT REGENXBIO Inc. REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit . FORWARD-LOOKING STATEMENTS This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2024, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at . All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. Zolgensma® is a registered trademark of Novartis AG. All other trademarks referenced herein are registered trademarks of REGENXBIO. Contacts: Dana Cormack Corporate Communications [email protected] Investors: George E. MacDougall Investor Relations IR@regenxbio.com SOURCE REGENXBIO Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

编者按：2025年，药明康德迎来创立25周年的重要里程碑。值此契机，我们向所有与我们共同书写产业变革篇章的科学家、医药人和投资者致以衷心感谢与诚挚敬意，也特别推出“致敬时代”系列，回顾全球同仁如何借助科学与合作的力量，不断拓展治疗边界、改善患者命运。 四分之一个世纪的坚守，只为加速每一款新药的诞生。下一个25年，我们将继续心怀感恩与敬畏，依托独特的CRDMO模式，与全球伙伴携手同行，共赴健康未来。 导致视力“黑洞”的疾病 19世纪中叶，一种特殊的眼部疾病逐渐引起了医学界的注意。不少患者有着相似的困扰：他们的视力开始下降，眼中的轮廓也逐渐变形。医生们检查后发现，在患者视网膜中央名为黄斑的区域内，散布着异常的白色斑点或是出血点。研究者们意识到，视网膜中发生了某种病变，但确切的病因无人知晓。 荷兰眼科病理学家Franciscus Donders博士决心揭开谜团，他在显微镜下观察逝者的眼球样本时，捕捉到一条重要线索：在退化的视网膜上，一类被称作玻璃膜疣（drusen）的斑点会压迫视网膜，甚至影响视网膜色素上皮细胞的功能。这些特征在七八十岁的老人眼中尤为常见。尽管没有提出描述这一病症的具体名词，但Donders博士的发现为后续探索指明了方向。 随后上百年间，科学家们陆续将老年性弱视、黄斑的病变、视网膜血管渗出等现象联系到一起，并且提出了一系列描述这种症状的术语。直到上世纪80年代，年龄相关性黄斑变性（age-related macular degeneration，AMD）这一名称出现并得到广泛使用。 全球目前有接近2亿人受到AMD的困扰。到2040年，患者数量预计将进一步增加到2.88亿。AMD已经成为导致老年人群失明的首要原因。 图片来源：123RF AMD可分为两种类型：萎缩性（干性）AMD和渗出性（湿性）AMD。 其中，干性AMD占病例的85%，典型特征是视网膜色素上皮细胞和光感受器细胞逐渐萎缩，同时视网膜的炎症、活性氧水平增加。 湿性AMD虽然仅占15%，却是严重视力丧失的最主要原因之一。除了炎症水平升高，湿性AMD最显著的特点是，视网膜等区域会产生侵袭性的新生血管。这些脆弱的血管极易破裂，造成脂质、血液在视网膜中渗漏、聚集，压迫感光细胞和视网膜色素上皮细胞，导致它们退化并死亡。 作为光感受器最集中的区域，位于视网膜中央的黄斑遭受了最严重的不可逆损伤。这些患者的视野中央如同被一枚逐渐长大的“黑洞”吞噬，只能用余光辨认周围的一切。 20世纪80年代，激光光凝疗法成为治疗湿性AMD的首选方法，它能通过破坏异常新生血管来延缓病情进展。不过，这种疗法具有破坏性，易导致视野缺损并且复发率高。光动力疗法带来了更精准的治疗方式，可在不损伤周围组织的情况下封闭异常血管。但它仍无法从根源阻断血管生成，疗效有限且需反复治疗。 因此，AMD患者亟需一种能从源头阻止破坏信号的方法。 湿性AMD治疗里程碑 进入21世纪，科学界对血管内皮生长因子（VEGF）的全新认知为湿性AMD治疗带来了转机。 健康情况下，VEGF可以维护眼睛脉络膜的毛细血管发育，同时还能保证视网膜神经元的血液供给，防止缺血性凋亡。然而，VEGF一旦过度表达，就可以异常激活黄斑区域的血管生成活动，最终造成不可逆的中心视力损伤。 至此，一场靶向VEGF的AMD疗法革命蓄势待发。 2004年，Macugen（pegaptanib）成为首个获得美国FDA批准用于治疗湿性AMD的抗VEGF药物。它的作用机制类似于一把特制的钥匙，能够特异性地插入并堵住VEGF-A165这一主要致病亚型的“锁孔”，提升AMD患者的视力。 不过，在VEGF-A165之外，还有其他亚型的VEGF在AMD病理中同样扮演了重要角色，而Macugen的特异性意味着它的阻断范围有限。 尽管如此，Macugen的成功上市无疑是一次破冰之旅，验证了玻璃体内注射抗VEGF药物这一治疗途径的安全性与可行性。 不久之后，一款改变湿性AMD治疗格局的新药登场。 由基因泰克（Genentech，现为罗氏[Roche]旗下子公司）开发的Lucentis（ranibizumab，雷珠单抗）是一种人源化的抗VEGF单克隆抗体片段。如果说Macugen是“专属钥匙”，Lucentis就是一把“万能钥匙”，能以高亲和力结合并抑制所有VEGF-A亚型。 Lucentis的两项3期临床试验结果令人振奋。其中一项试验中，与对照组相比，每月接受Lucentis玻璃体注射的患者，超过94%避免了视力进一步丧失，这一比例在对照组中仅有约62%。此外，约四分之一到三分之一的患者视力得到了显著提升。在另一项试验中，Lucentis的治疗效果显著优于光动力疗法。Lucentis于2006年获批，迅速成为了全球治疗湿性AMD的标准疗法。 2011年，拜耳（Bayer）和再生元（Regeneron）公司推出的Eylea（aflibercept，阿柏西普）开启了又一项抗VEGF治疗策略。Eylea并非抗体，而是一种重组融合蛋白，其设计更为精巧：它融合了VEGF受体1和2的结合域，能够更紧密地“抓住”VEGF-A、VEGF-B以及胎盘生长因子。因此，Eylea的作用持续时间更长。 图片来源：123RF 患者仅需在初始三个月内每月完成注射，之后就能转为每两个月注射一次Eylea。这种治疗方式的效果与每月注射Lucentis完全相同。这意味着患者每年的注射次数可从12次减少到8次，减轻了治疗负担，以及反复注射带来的潜在风险。Eylea的上市为需要长期治疗的患者和医生提供了一个更具可持续性的管理方案。 “一石二鸟”新策略 随着更多临床数据的涌现，科学家也发现部分湿性AMD患者在使用抗VEGF药物后应答并不如预期，或很容易出现耐药。为了帮助这部分患者缓解症状，研究者开始寻找第二条血管生成相关信号通路。 血管生成素-2（Ang-2）是另一种参与血管生成和炎症过程的细胞因子。研究指出，Ang-2和VEGF-A协同作用，通过破坏血管稳定性形成新的渗漏血管，增加炎症的发生，从而驱动AMD等视网膜疾病。因此，同时靶向Ang-2和VEGF-A成为湿性AMD疗法开发的新方向。 2022年1月，FDA批准了基因泰克同时靶向Ang-2和VEGF-A的双特异性抗体Vabysmo（faricimab，法瑞西单抗）上市，这也是第一款用于治疗眼科疾病的双特异性抗体。 值得一提的是，Vabysmo首次获配的适应症不仅有湿性AMD，还有另一类眼科疾病：糖尿病性黄斑水肿（DME）。 DME影响了全球超2000万人，是工作年龄成年人视力丧失的主要原因。对于DME患者，高血糖水平导致视网膜血管受损、渗漏液体，引起黄斑肿胀，从而影响视力。 在分别针对湿性AMD患者和DME患者的临床试验中，相比于每2个月给药一次的标准治疗，Vabysmo每4个月给药一次就可以获得相当甚至更优异的视力改善，并且安全与耐受性良好。 在2024年公布的一项临床试验数据中，经过4年的Vabysmo治疗，超过90%的DME患者达到症状消失的标准！ 值得一提的是，FDA还在2023年批准Vabysmo用于治疗另一种常导致视力丧失的视网膜血管疾病——视网膜静脉阻塞（RVO）。 更多创新策略“点亮”视力 其他疗法类型同样蕴藏着治疗AMD的希望。 例如，REGENXBIO与艾伯维（AbbVie）联合开发的在研疗法ABBV-RGX-314，使用腺相关病毒8（AAV8）载体携带表达抗VEGF抗体片段的基因。它在体内表达的蛋白通过阻断VEGF信号传导，抑制血管生成和视网膜液体聚集。根据2期临床数据，湿性AMD患者接受ABBV-RGX-314治疗9个月后，治疗负担下降了97%。 另一款能够抑制4种血管生成因子（VEGF-A、VEGF-B、VEGF-C和PlGF）的在研基因疗法4D-150，也在治疗湿性AMD的2期临床试验中呈现出积极数据。高剂量4D-150可将抗VEGF注射治疗的年化注射率降低89%，63%的患者在接受治疗后的24周内无需补充注射抗VEGF药物。 值得一提的是，干性AMD也在近年迎来了关键疗法的获批。 干性AMD引起的地图样萎缩（GA）是干性AMD的终末期表现。这种由于过量补体活化引起的不可逆病变，导致黄斑区域的视网膜细胞大量死亡。因此，补体蛋白成为治疗地图样萎缩的重要靶点。 图片来源：123RF 2023年，靶向C3补体蛋白的疗法Syfovre（pegcetacoplan），以及抑制C5补体蛋白的RNA适配体（aptamer）疗法Izervay（avacincaptad pegol）相继上市，成为前两款获FDA批准用于治疗干性AMD引起的地图样萎缩的疗法。 一体化平台赋能眼科新药研发 作为全球医药创新的赋能者，药明康德很高兴在25年发展历程中助力合作伙伴，加速治疗AMD的创新疗法问世，造福病患。长期以来，药明康德都在支持全球合作伙伴从药物研究（R）、开发（D）到商业化生产（M）各个阶段的需求，通过独特的一体化、端到端CRDMO模式，助力更多药物加速从实验室来到患者身边。 例如，药明康德生物学业务平台体内药理学部眼科药效平台专注于眼部疾病的研究与治疗，验证了多种眼部疾病模型，同时可以提供系统的眼科药效评价、non-GLP安全性评价、PK分析等服务。此外，药明康德DMPK已建立临床前动物眼科药代评价平台，能够熟练操作临床前大小动物的各种眼局部给药、各种眼部细分组织的样品采集和药物浓度分析。药明康德DMPK的定量全身放射自显影技术（QWBA）平台可用于评价眼科药物在临床前动物眼部各精细组织的分布情况，为客户的新药研发提供助力。 随着各种创新药物的涌现，过去被认为不可逆的AMD已经能得到有效遏制，许多患者有了重新恢复清晰视力的希望。展望下一个25年，我们相信更多有效的AMD药物将继续走向前台，“点亮”患者丢失的世界。 正如药明康德的愿景一样：“让天下没有难做的药，难治的病。”在这条追光之路上，我们期待更多疗法早日到来，AMD患者也能拥抱美好的光明未来。 参考资料： [1] de Jong PT. A Historical Analysis of the Quest for the Origins of Aging Macula Disorder, the Tissues Involved, and Its Terminology. Ophthalmol Eye Dis. 2016 Nov 1;8(Suppl 1):5-14. doi: 10.4137/OED.S40523 [2] Fabre, M. et al., Recent Advances in Age-Related Macular Degeneration Therapies. Molecules 2022, 27, 5089. https://doi-org.libproxy1.nus.edu.sg/10.3390/molecules27165089 [3] Fleckenstein M, Schmitz-Valckenberg S, Chakravarthy U. Age-Related Macular Degeneration: A Review. JAMA. 2024;331(2):147–157. doi:10.1001/jama.2023.26074 [4] Ranibizumab for Neovascular Age-Related Macular Degeneration. N Engl J Med 2006;355:1419-1431. DOI: 10.1056/NEJMoa054481 [5] Ring Therapeutics Presents New Data on Anellogy™ Platform at the 27th Annual American Society of Gene & Cell Therapy Conference. Retrieved May 9, 2024, from https://ringtx.com/news/ring-therapeutics-presents-new-data-on-anellogy-platform-at-the-27th-annual-american-society-of-gene-cell-therapy-conference/ [6] U.S. FDA Approves Expanded Label for Astellas' IZERVAY™ (avacincaptad pegol intravitreal solution) for Geographic Atrophy. Retrieved February 13, 2025 from https://www.prnewswire.com/news-releases/us-fda-approves-expanded-label-for-astellas-izervay-avacincaptad-pegol-intravitreal-solution-for-geographic-atrophy-302375403.html [7] Roche’s Susvimo maintains vision over five years with two refills per year in people with neovascular age-related macular degeneration (nAMD). Retrieved August 1, 2025 from https://www.globenewswire.com/news-release/2025/08/01/3126063/0/en/Roche-s-Susvimo-maintains-vision-over-five-years-with-two-refills-per-year-in-people-with-neovascular-age-related-macular-degeneration-nAMD.html [8] Roche’s Vabysmo showed extended durability, continued efficacy and a consistent safety profile in long-term diabetic macular edema (DME) study. Retrieved July 17, 2024, from https://www.roche.com/media/releases/med-cor-2024-07-17b 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新