Surabgene Lomparvovec

Late-stage gene therapy pipeline for rare and retinal diseases advancing toward key catalysts RGX-202 for Duchenne muscular dystrophy: New Phase I/II data at MDA, topline pivotal results expected early Q2 2026 Robust patient enrollment continues in confirmatory trial Surabgene lomparvovec (sura-vec, ABBV-RGX-314) subretinal wet AMD topline pivotal data expected in Q4 2026; first patient dosed in pivotal Phase IIb/III trial in diabetic retinopathy expected in Q2 2026 Company to engage FDA on potential path forward for RGX-121 Conference call today at 8:00 a.m. ET ROCKVILLE, Md., March 5, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and operational highlights for the fourth quarter and year ended December 31, 2025. "We are rapidly advancing our late-stage pipeline of gene therapies to treat rare and retinal diseases with significant unmet need, with multiple near-term catalysts in 2026," said Curran Simpson, President and Chief Executive Officer, REGENXBIO. "We will drive continued momentum across our programs – powered by our fully in-house, end-to-end capabilities, commercial-ready manufacturing, and global partners. We remain focused on executing on our mission to deliver meaningful new medicines to patients in need through the curative potential of gene therapy." PROGRAM HIGHLIGHTS AND MILESTONES Neuromuscular Disease: RGX-202 is a potential best-in-class gene therapy for Duchenne muscular dystrophy (Duchenne). RGX-202 is designed to address the underlying cause of Duchenne by enabling targeted expression of a novel microdystrophin that is closest to naturally occurring dystrophin. It is the only microdystrophin that includes the C-Terminal domain, which has been shown to protect and preserve muscle function. The differentiated therapeutic approach behind RGX-202 includes a novel construct, a proactive immune suppression regimen, and a suspension-based manufacturing process that delivers industry-leading product purity levels. RGX-202 is designed for improved muscle function, durability and safety outcomes for patients. Positive 18-month functional data from patients treated with the pivotal dose in the Phase I/II portion of the AFFINITY DUCHENNE® trial (n=4) were reported in January 2026. All patients exceeded expected disease trajectory on the North Star Ambulatory Assessment (NSAA) using the established cTAP disease progression model. RGX-202 recipients improved an average of 7.4 points compared to cTAP. These same patients improved an average of 6.6 points compared to cTAP at 12 months post-treatment. The Company plans to share additional Phase I/II safety, biomarker, and functional data at the MDA Clinical and Scientific Conference on March 11, 2026. REGENXBIO continues to manufacture RGX-202 intended for commercial supply at its in-house Manufacturing Innovation Center. REGENXBIO expects to share pivotal topline data in early Q2 2026. REGENXBIO plans to request a pre-BLA meeting in mid-2026 to align with FDA on the BLA submission. Additional regulatory interactions with the FDA and European Medical Association (EMA) are planned for 1H 2026. Following the completion of enrollment in the pivotal trial (n=30) in October 2025, the Company continues to enroll in the confirmatory trial (n=30) and expects to have the majority of this trial enrolled at the time of BLA submission. Retinal Disease: Surabgene lomparvovec (sura-vec, ABBV-RGX-314), developed in collaboration with AbbVie, is potentially the first-in-class treatment for wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR). Sura-vec for the Treatment of Wet AMD (Subretinal Delivery) REGENXBIO expects to share topline data with AbbVie from ATMOSPHERE® and ASCENT® pivotal trials of sura-vec using subretinal delivery in Q4 2026. Global regulatory submissions are expected in 2027. Sura-vec for the Treatment of DR (Suprachoroidal Delivery) REGENXBIO is activating U.S. clinical sites in the pivotal Phase IIb/III NAAVIGATE study. The Company will receive a $100 million milestone payment from AbbVie upon first patient dosed in the Phase IIb portion, expected Q2 2026. NAAVIGATE is a Phase IIb/III multicenter, randomized, masked, sham-controlled study to evaluate the safety and efficacy of sura-vec in subjects with non-proliferative DR (NPDR) without center-involved diabetic macular edema (CI-DME). The primary endpoint is ≥2-step improvement on the diabetic retinopathy severity scale (DRSS) at one year. Following an interim analysis, REGENXBIO and AbbVie will initiate a Phase III expansion, which will include two Phase III trials, including a U.S. trial and a parallel global trial, led by AbbVie. All subjects will receive sura-vec at 1.0x1012 genome copies (GC)/eye, which was evaluated as Dose Level 3 in the Phase II ALTITUDE® trial of sura-vec, and short-course topical prophylactic steroids. At two years post treatment with Dose Level 3 and short-course prophylactic topical steroids in ALTITUDE, no intraocular inflammation was observed (n = 15). The majority of subjects achieved DRSS improvement, with 50% achieving ≥2-step improvement without additional DR treatment. Sura-vec at Dose Level 3 also reduced the risk of disease progression, demonstrating a ≥70% risk reduction in vision-threatening complications compared to historical controls. Sura-vec for the Treatment of Wet AMD (Suprachoroidal Delivery) Enrollment is complete in the Phase II AAVIATE® trial. Neurodegenerative Disease: Clemidsogene lanparvovec (RGX-121) is a potential first-in-class treatment for Mucopolysaccharidosis (MPS) Type II, also known as Hunter syndrome. RGX-111 is an investigational one-time treatment for severe MPS I, also known as Hurler syndrome. These programs are partnered with Nippon Shinyaku. In January 2026, the FDA placed a clinical hold on RGX-111 following preliminary analysis of a single case of neoplasm (intraventricular CNS tumor) in a participant treated in its Phase I/II study. The FDA also placed a clinical hold on RGX-121 citing similarities to RGX-111, study populations, and shared risk between the clinical studies. In February 2026, the FDA issued a complete response letter (CRL) for the RGX-121 BLA. REGENXBIO is working to address concerns in the CRL with the goal of resubmitting the BLA. FINANCIAL RESULTS Cash Position: Cash, cash equivalents and marketable securities were $240.9 million as of December 31, 2025, compared to $244.9 million as of December 31, 2024. The decrease was primarily driven by cash used to fund operating activities during the year ended December 31, 2025, and was partially offset by the $110.0 million upfront payment received under the Nippon Shinyaku partnership in March 2025 and $144.5 million in net proceeds received from the royalty monetization with HCRx in May 2025. Revenues: Revenues were $30.3 million and $170.4 million for the three months and full year ended December 31, 2025, respectively, compared to $21.2 million and $83.3 million for the three months and full year ended December 31, 2024, respectively. The increases were primarily attributable to revenues recognized under our collaboration with Nippon Shinyaku, including $72.9 million of up-front license revenue and $11.8 million of service revenue recognized in 2025, as well as an increase in royalty revenues for Zolgensma and Itvisma. Research and Development Expenses: Research and development expenses were $59.6 million and $228.3 million for the three months and full year ended December 31, 2025, respectively, compared to $50.4 million and $208.5 million for the three months and full year ended December 31, 2024, respectively. The increases were primarily attributable to personnel-related costs and clinical trial expenses, largely driven by RGX-202 pivotal trials, for the fourth quarter and full year 2025, as well as an increase in manufacturing-related expenses Sura-vec, RGX-202 and RGX-121 for the full year 2025. General and Administrative Expenses: General and administrative expenses were $22.4 million and $82.9 million for the three months and full year ended December 31, 2025, respectively, compared to $20.1 million and $76.6 million for the three months and full year ended December 31, 2024, respectively. The increases were largely driven by professional services, consulting and other corporate advisory services. Net Loss: Net loss was $67.1 million, or $1.30 basic and diluted net loss per share, for the three months ended December 31, 2025, compared to a net loss of $51.2 million, or $1.01 basic and diluted net loss per share, for the three months ended December 31, 2024. Net loss was $193.9 million, or $3.76 basic and diluted net loss per share, for the year ended December 31, 2025, compared to a net loss of $227.1 million, or $4.59 basic and diluted net loss per share, for the year ended December 31, 2024. FINANCIAL GUIDANCE REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $240.9 million as of December 31, 2025 to fund its operations into early 2027. This cash runway guidance is based on the Company's current operational plans and excludes the impact of any material payments that may potentially be received from partners or licensees upon the achievement of development or regulatory milestones, or upon the approval or commercialization of product candidates, and excludes any additional potential non-dilutive funding opportunities. CONFERENCE CALL In connection with this announcement, REGENXBIO will host a conference call and webcast at 8:00 a.m. ET today. Listeners can register for the webcast via this link. Analysts wishing to participate in the question and answer session should use this link. A replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time. 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, clinical trials, regulatory interactions following the complete response letter, costs and cash flow. 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 timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timing or likelihood of payments from AbbVie or Nippon Shinyaku, the monetization of any priority review voucher, 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, 2025, which will be filed with the U.S. Securities and Exchange Commission (SEC) in the first quarter of 2026 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® and Itvisma® are registered trademarks of Novartis Gene Therapies, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO. CONTACTS: Dana Cormack Corporate Communications [email protected] George E. MacDougall Investor Relations [email protected] SOURCE REGENXBIO Inc. 21% more press release views with Request a Demo

本文搬运自《Ophthalmology Times》原作者：Lynda Charters 原标题： The status of real-world treatments for nAMD and DME and what’s coming next （译：nAMD和DME的实际治疗现状及未来展望） 主要要点 真实证据表明，更高的注射频率与更好的VA效果和减少CST波动有关，进一步强调耐久性是nAMD和DME管理中主要未被满足的需求。 实际的治疗不足驱动因素包括门诊容量、患者对灵活间隔的偏好、交通及多专科共病管理、就业需求以及共付额补助的变异性，这些都可能显著改变注射率。 法利单抗治疗及延长数据显示，注射次数较少，CST/VA控制较强，较每8周aflibercept 2毫克，~80%的患者在第1至2年内超过每12周。 Aflibercept 8毫克在96周内表现出类似的持久性信号，而ranibizumab的PDS显示维持期为5年，且每半年续药一次，救助率较低。 流程治疗包括可生物侵蚀的玻璃体内TKI植入物、一种设计用于穿越血液视网膜屏障的皮下TKI，以及产生抗VEGF蛋白的基因疗法，将眼睛转化为持续递送的“生物工厂”。 2月13日至16日在波多黎各举行的EnVision峰会上，讨论了新生血管性年龄相关性黄斑变性（nAMD）和糖尿病黄斑水肿（DME）治疗的疗效。科罗拉多视网膜协会临床研究主席Murtaza Adam医学博士和威斯康星眼科诊所研究医疗主任Deepak Sambhara医学博士回顾了该治疗的现实现状，以及眼科医生未来可以期待的成果。nAMD和DME治疗的现实影响 实际数据显示，更多nAMD和DME患者在更频繁的玻璃体内注射时获得更好的视力（VAs）。相比之下，慢性治疗不足的患者中枢亚野厚度（CST）波动较大，导致视力丧失。因此，眼科医生希望药物更耐用，亚当解释道。 Sambhara评论说，治疗不足源于医生排班冲突或患者希望治疗时间表灵活的因素。此外，对于通常较年长的nAMD患者群体来说，可靠的交通和多位医生专家的系统性疾病管理也是其他问题;对于年轻的DME群体，这些因素以及他们的个人工作时间表同样适用。 共付援助项目的资金对患者来说也是一个挑战，是另一个治疗障碍。亚当介绍了一项在其诊所进行的相关研究，发现患者在注册前后均获得覆盖治疗，入组前平均每年注射两到三次nAMD和DME，入组后每年注射八次。第二代药物 目前视网膜医生可使用三种药物：8毫克aflibercept（Eylea，Regeneron制药）、Susvimo Port Delivery System（PDS）含ranibizumab（Genentech/Roche）和faricimab-svoa（Vabysmo，Genentech/Roche）。 TENAYA和LUCERNE三期临床试验模拟了负荷剂量后真实世界的治疗加延（TAE）方案。研究比较了标准护理、三次负荷剂量后服用2毫克aflibercept和每8周一次治疗;接受法利单抗6毫克治疗的患者在四次负荷剂量后每8至16周接受一次治疗。亚当解释说，患者每月观察一次，然后根据需要延长。 他报告说：“接受法利西单抗治疗的患者在CST和VA上的效果非常显著，相比每8周一次的aflibercept 2毫克，且TAE阶段的注射次数是三次注射的一半，而TAE阶段的注射次数是一半。” 结果还显示，近80%的患者在第1和第2年被延长至超过每12周一次的用药。 Sambhara还指出，所有第二代药物通常比第一代贝伐单抗（Avastin、Genentech/Roche）、aflibercept和ranibizumab（Lucentis、Genentech/Roche）更具耐久性。 真实世界的TRUCKEE研究显示，从抗血管内皮生长因子（VEGF）药物或aflibercept换至法利西单抗的患者，液体显著减少。 法利西单抗的双重作用在整个研究中对维持干效应起到了关键作用。Sambhara表示，法里西单抗还能更快且减少注射次数，减少视网膜内硬质渗出物的数量和体积。 Aflibercept 8毫克也被证明具有持久性和效力，在96周内CST和视力均有类似改善;80%的患者每12周或更长时间服用一次，剂量与法利西单抗6毫克相似。 PDS的2期LADDER和3期ARCHWAY研究的5年数据表现良好，超过50%的患者保持20/40或以上，仅5%需要救援治疗。PDS每6个月补一次。 PHOTON研究中针对约2000名DME患者使用8毫克aflibercept的早期数据显示，CST和视力在注射间隔平均11周内有显著改善。治疗流程中有哪些酪氨酸激酶抑制剂（TKIs） Sambhara博士表示，这是一种不同类别的药物，正在研究用于治疗视网膜血管疾病，包括糖尿病视网膜病变、DME和nAMD，最终通过阻断泛VEGF受体和持续长期抑制来实现。 他指出，虽然市场上有药物可以阻断特定形式的VEGF，但TKIs可以通过其细胞内信号传递，全面阻断所有VEGF亚型。 目前正在研究两种产品：EYP-1901（Duravyu的试验性名称，EyePoint 制药公司）以及OTX-TKI（阿昔替尼植入剂，眼治疗）。 EYP-1901是一种玻璃体内植入物，可将TKI伏罗拉尼用于治疗nAMD和DME，并采用完全可生物腐蚀的Durasert平台。 针对nAMD的Davio和Davio2研究，针对EYP-1901患者，调查了研究前治疗负担较高的患者。结果显示，经过6个月和一次EYP-1901植入后，治疗负担显著减轻。第三阶段Lucia和Lugano试验已全部注册，预计2026年晚些时候公布主要数据。 Como和Capri试验正在开始招募，将评估EYP-1901用于DME。 OTX-TKI是一种生物侵蚀水凝胶，采用Elutyx平台，通过25号针头注射。澳大利亚和美国的两项1期和1b期研究纳入了曾接受阿弗利伯西普治疗的患者。结果与EYP-1901的Davio和Davio2研究相似，即缩短治疗间隔，并改善了视力和解剖结构。三期SOL和SOL-R研究预计很快将发布其主要数据。 亚当描述了皮下注射的TKI，称为migaldendranib  (Ashvattha Therapeutics)，这是一种新型羟基树枝聚体基小分子TKI，能够更轻松地通过血液视网膜屏障和血脑屏障，且系统性副作用极小。一项1/2期临床研究正在评估该药物以减轻nAMD的治疗负担。一项二期AMD/DME研究显示，CST和VA在减少VEGF补充的情况下得以维持。“这是一个令人振奋的方案，可以减轻治疗负担，”他强调道。基因治疗 这是治疗AMD和DME最令人兴奋的新兴治疗方法之一。目前有三种药物正在研究中。 ABBV-RGX-314（sura-vec，RegenxBio/AbbVie）利用AAV8载体在视网膜下递送一个基因，产生雷尼珠单抗，以减轻nAMD患者的治疗负担。一项二期临床试验实现了约70%的治疗负担降低。 Ixo-VEC（ixoberogene soroparvovec，前称ADVM-022，Adverum Biotechnologies）同样用于nAMD，使用AAV.7m8衣壳，促进aflibercept的产生并降低治疗负担。一项二期研究显示治疗负担减轻约75%。 4D-150（4D分子治疗）用于nAMD和DME。该试验药物同时产生aflibercept和VEGF抑制剂。 “基因治疗可以通过视网膜下切除术或玻璃体内传递，”亚当说。 他解释说，基因疗法的理念是将眼睛变成一个生物工厂。其他优点是飞蚊症被清除，治疗也较少引起炎症。 声明 本文相关生物医药及眼科领域产业新闻内容，由AI工具辅助完成网络信息搜集与整理工作。内容整理过程中已对信息来源及准确性进行核查，但AI生成内容可能存在疏漏，敬请谅解。本文仅传递产业资讯，不提供医疗、投资建议。鉴于互联网的开放性和文章创作的复杂性，我们无法保证所转载的所有文章均已获得原作者的明确授权。如果您是原作者或拥有相关权益，请与我们联系，我们将立即删除未经授权的文章。 本声明旨在保障信息透明度，AI工具仅为辅助手段，不享有相关内容的著作权及署名权。