Surabgene Lomparvovec

> 2026年1月28日，美国食品药品监督管理局（FDA）对生物技术公司REGENXBIO的一项基因疗法试验实施临床搁置，消息传出后，该公司股价在盘前交易中暴跌32%。这一监管行动立即引发市场对该公司估值及关键研发管线前景的深度担忧，凸显基因疗法领域的高风险与严监管常态。 1. 事件冲击：股价暴跌与市值蒸发 根据财联社、格隆汇等多家媒体报道，FDA的临床搁置决定直接触发了投资者抛售。REGENXBIO股价在盘前交易中大幅下滑，跌幅达**32%**。截至1月27日收盘，该公司股价为**13.41美元**，总市值约**6.79亿美元**。以32%的跌幅计算，此次事件使公司市值蒸发约**2.17亿美元**。 临床搁置意味着FDA要求暂停试验的招募或给药，通常基于对安全性或试验设计的疑问。 2. 核心管线：多个项目处于关键阶段 REGENXBIO的研发管线中包括几个重点项目，正处于审批或数据公布的关键时期，这些进展直接关联公司估值： - **RGX-111**（针对亨特综合征）：正处于生物制剂许可申请（BLA）审查中，原定的处方药用户费用法案（PDUFA）日期为**2026年2月8日**。该疗法旨在通过一次性治疗，使患者体内持续产生所需酶。 - **RGX-202**（针对杜氏肌营养不良症）：已完成关键试验的30名患者入组，计划在**2026年年中提交BLA**。 - **RGX-314**（针对湿性年龄相关性黄斑变性，与艾伯维合作）：顶线数据预计在**2026年晚些时候公布**，可能成为首个针对非罕见病的基因治疗。 3. 历史伏笔：FDA曾因数据要求延期审查 此次临床搁置并非毫无征兆。2025年8月，FDA因要求获取所有**13名II型黏多糖贮积症（MPS II，即亨特综合征）患者的更长期临床数据**，将RGX-121（与RGX-111相关）的审批决定日期从2025年11月9日推迟至**2026年2月8日**。公司首席执行官Curran Simpson当时表示： > “我们迅速向FDA提供了所需信息……并预计商业推出计划仍按轨道进行。” 4. 影响分析：PDUFA日期与管线推进 尽管参考文章未明确说明此次搁置针对的具体试验，但事件发生在**RGX-111的PDUFA日期（2月8日）临近之际**，加剧了市场不确定性。临床搁置可能影响该产品的审批时间表，并对其他管线如RGX-202和RGX-314的推进产生连锁反应。 依赖早期数据或替代终点寻求加速批准的策略，正面临FDA更严格的审查。 5. 行业警示：基因疗法的监管挑战 REGENXBIO的遭遇反映了基因治疗领域普遍面临的监管挑战。FDA越来越强调**长期安全性数据和确凿的临床获益证据**，这要求生物技术公司在创新速度与数据质量之间找到平衡。此次事件可能促使行业重新评估快速上市策略，尤其是在涉及一次性修改DNA的疗法中，长期随访数据成为监管硬通货。 6. 未来展望：公司应对与市场观察 截至发稿，REGENXBIO尚未公布针对此次临床搁置的具体应对措施。投资者和行业观察者正密切关注公司是否会像去年一样，迅速提交FDA要求的信息以解决搁置问题。股价的后续表现将取决于公司能否提供清晰解释和解决方案，以及搁置原因是否涉及严重安全性问题。 此次FDA的临床搁置行动，不仅对REGENXBIO的估值构成直接冲击，也凸显了基因疗法研发中监管风险的核心地位。在追求医学突破的同时，严格的监管框架正成为影响生物技术公司生存与发展的关键变量。

ROCKVILLE, Md., Jan. 28, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug Administration (FDA) placed a clinical hold on its investigational gene therapy, RGX-111, for the treatment of MPS I, also known as Hurler syndrome, 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, for the treatment of MPS II, also known as Hunter Syndrome, citing the similarities in products, study populations, and shared risk between the clinical studies. The case was identified during a routine brain MRI of an asymptomatic five-year-old participant who received intracisternal RGX-111 four years prior. Preliminary genetic analysis of the resected tumor detected an AAV vector genome integration event associated with overexpression of a proto-oncogene (PLAG1), which is known to be susceptible to chromosomal rearrangements. The investigation to determine if this SAE is drug related is ongoing. The causality has not been established. The participant continues to be asymptomatic, with positive developmental advancements noted by the treating physician. No evidence of neoplasm has been reported in the nine other participants treated with RGX-111 nor in the 32 participants treated with RGX-121. "We are surprised by FDA's decision to place our RGX-121 program on hold while the investigation of this single, inconclusive incident in RGX-111 continues," said Curran Simpson, President and CEO of REGENXBIO. "These are separate therapies, and the positive safety profile of RGX-121 in more than 30 patients treated, including those dosed nearly seven years ago, remains unchanged. Patient safety is our top priority, and we, our investigators, and the patient community remain confident in the benefit-risk ratio of RGX-121 and are highly encouraged by the meaningful efficacy profile demonstrated in the pivotal trial. RGX-121 presents an opportunity to address the urgent, significant unmet medical need in this ultra-rare disease community, and continued delay means continued neurodevelopmental decline in boys with MPS II." REGENXBIO has not yet received the full clinical hold letter and awaits additional details from the FDA. About RGX-121 (clemidsogene lanparvovec) RGX-121 is a potential one-time gene therapy for the treatment of boys with MPS II, designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS). Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted iduronate-2-sulfatase (I2S) protein beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. RGX-121 expressed protein is structurally identical to normal I2S. RGX-121 has received Orphan Drug Product, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA and advanced therapy medicinal products (ATMP) classification from the European Medicines Agency. About Mucopolysaccharidosis Type II (MPS II) MPS II, or Hunter Syndrome, is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme I2S leading to an accumulation of glycosaminoglycans (GAGs), including heparan sulfate (HS) in tissues which ultimately results in cell, tissue, and organ dysfunction, including in the central nervous system (CNS). In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by 18 to 24 months. Specific treatment to address the neurological manifestations of MPS II remains a significant unmet medical need. Key biomarkers of I2S enzymatic activity in MPS II patients include its substrate CSF HS D2S6, which has been shown to correlate with neurocognitive manifestations of the disorder. About RGX-111 RGX-111 is designed to use the AAV9 vector to deliver the α-l-iduronidase (IDUA) gene to the central nervous system (CNS). Delivery of the IDUA gene within the cells in the central nervous system (CNS) could provide a permanent source of secreted IDUA beyond the blood-brain barrier, allowing for long-term cross-correction of cells throughout the CNS. By providing rapid IDUA delivery to the brain, RGX-111 could potentially help prevent the progression of cognitive deficits that otherwise occurs in MPS I patients. RGX-111 has received orphan drug product, rare pediatric disease and Fast Track designations from the FDA. About Mucopolysaccharidosis Type I (MPS I) MPS I is a rare autosomal recessive genetic disease caused by a deficiency in the lysosomal enzyme alpha-L-iduronidase (IDUA), leading to an accumulation of glycosaminoglycans (GAGs) including heparan sulfate (HS) in tissues which ultimately results in cell, tissue, and organ dysfunction, including in the central nervous system (CNS). This can include excessive accumulation of fluid in the brain, spinal cord compression, and cognitive impairment. MPS I is estimated to occur in 1 in 100,000 births. Current disease modifying therapies for MPS I include hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy with a recombinant form of human IDUA administered intravenously. However, intravenous enzyme therapy does not treat the CNS manifestations of MPS I, and HSCT can be associated with clinically significant morbidity and mortality. Key biomarkers of IDUA enzymatic activity in MPS I patients include its substrate heparan sulfate (HS), which has been shown to correlate with neurocognitive manifestations of the disorder. 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; NAVSUNLI 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, 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, 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 Gene Therapies. 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