US Market News
4週前
REGENXBIO Announces Presentations at the American Society of Gene & Cell Therapy 2026 Annual MeetingMay 7, 2026 7:05 AM
PR Newswire (US) ROCKVILLE, Md., May 7, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the 2026 American Society of Gene & Cell Therapy Annual Meeting ("ASGCT 2026") taking place May 11-15, 2026, in Boston, Massachusetts. Oral Presentations:
The oral presentations include an overview of recent advancements in production titer and product quality achieved through REGENXBIO's NAVXpress® manufacturing platform and an encore presentation of topline pivotal data from RGX-202, the company's next generation gene therapy program for Duchenne muscular dystrophy.Title: Advancing AAV manufacturability toolbox for early programs and beyond
Presenter: Metewo Selase Kosi Enuameh, Ph.D., Associate Director, Vector Core at REGENXBIO
Presentation: 266
Session: AAV Critical Quality Attributes
Date/Time: Thursday, May 14, 8:45 – 9:00am ET
Location: MCEC Room 204AB (Level 2)Title: RGX-202: Investigational gene therapy for Duchenne Muscular Dystrophy
Presenter: Aravindhan Veerapandiyan, M.D., Director of the Comprehensive Neuromuscular Program, PPMD Certified Duchenne Care Center, and Co-Director of the Muscular Dystrophy Association Care Center at Arkansas Children's Hospital
Presentation: 506
Session: In vivo clinical trials in eye and muscle disorders
Date/Time: Friday, May 15, 4:30 – 4:45pm ET
Location: MCEC Room 210ABC (Level 2)Poster Presentations:
These presentations demonstrate REGENXBIO's continued leadership in the discovery and advancement of next-generation gene therapies for rare and retinal diseases, and highlight the company's unique capsid engineering and translational capabilities.Title: NVG82, a capsid engineered for enhanced outer retinal gene transfer when administered in the suprachoroidal space, demonstrates superior on-target activity compared to AAV8
Poster: 1031
Presenter: Brendan Lilley, Ph.D., Director of Ophthalmology Research, REGENXBIO
Location: MCEC Exhibit and Poster Hall (Halls B2-C, Exhibit level)
Date/Time: Tuesday, May 12, 5:00 – 6:30pm ETTitle: Examination of Route-Dependent Ocular Transduction in Rodent Models by the Novel AAV Capsid NVG82
Poster: 3031
Presenter: Huzzatul Mursalin, Ph.D., Scientist II, Gene Therapy Research, REGENXBIO
Location: MCEC Exhibit and Poster Hall (Hall B2-C, Exhibit level)
Date/Time: Thursday, May 14, 5:00 – 6:30pm ETTitle: Development of an engineered hybrid AAV with reduced liver and DRG transduction and high productivity
Poster: 3030
Presenter: Samantha Yost, Ph.D., Senior Scientist, Gene Therapy Research, REGENXBIO
Location: MCEC Exhibit and Poster Hall (Hall B2-C, Exhibit level)
Date/Time: Thursday, May 14, 5:00 – 6:30pm ETTitle: Single-nuclei transcriptomic analysis of skeletal muscle mRNA in mdx mice treated with RGX-202, an AAV vector encoding micro-dystrophin
Poster: 3499
Presenter: Justin Glenn, Ph.D., Principal Scientist, Gene Therapy Research, REGENXBIO
Location: MCEC Exhibit and Poster Hall (Hall B2-C, Exhibit level)
Date/Time: Thursday, May 14, 5:00 – 6:30pm ETAll presentations will be available on the Publications page of REGENXBIO's website, www.regenxbio.com.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 WWW.REGENXBIO.COM.Contacts:Dana Cormack
Corporate Communications
dcormack@regenxbio.comInvestors:
George E. MacDougall
Investor Relations
IR@regenxbio.com View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-announces-presentations-at-the-american-society-of-gene--cell-therapy-2026-annual-meeting-302765129.htmlSOURCE REGENXBIO Inc. Original: REGENXBIO Announces Presentations at the American Society of Gene & Cell Therapy 2026 Annual Meeting
US Market News
3月前
REGENXBIO Reports Fourth Quarter and Full Year 2025 Financial Results and Operational HighlightsMarch 5, 2026 7:05 AM
PR Newswire (US)
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 2026Robust patient enrollment continues in confirmatory trialSurabgene 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 2026Company to engage FDA on potential path forward for RGX-121Conference call today at 8:00 a.m. ETROCKVILLE, 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 MILESTONESNeuromuscular 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 www.REGENXBIO.com.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 WWW.SEC.GOV. 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
Dcormack@regenxbio.com George E. MacDougall
Investor Relations
IR@regenxbio.com REGENXBIO INC.CONSOLIDATED BALANCE SHEETS(unaudited)(in thousands)
As of December 31,
2025
2024
Assets
Current assets
Cash and cash equivalents
$34,466
$57,526
Marketable securities
195,604
177,161
Accounts receivable
26,379
20,473
Prepaid expenses
11,927
9,067
Other current assets
12,905
13,774
Total current assets
281,281
278,001
Marketable securities
10,785
10,179
Accounts receivable
2,312
474
Property and equipment, net
104,855
117,589
Operating lease right-of-use assets
47,156
53,716
Restricted cash
2,030
2,030
Other assets
4,613
4,000
Total assets
$453,032
$465,989
Liabilities and Stockholders' Equity
Current liabilities
Accounts payable
$21,358
$22,798
Accrued expenses and other current liabilities
38,390
38,070
Deferred revenue
10,452
115
Operating lease liabilities
8,286
7,902
Royalty monetization liabilities
39,609
34,309
Total current liabilities
118,095
103,194
Deferred revenue
18,943
—
Operating lease liabilities
65,215
74,131
Royalty monetization liabilities
147,408
25,378
Other liabilities
638
3,635
Total liabilities
350,299
206,338
Stockholders' equity
Preferred stock; no shares issued and outstanding
at December 31, 2025 and 2024
—
—
Common stock; 50,892 and 49,549 shares issued
and outstanding at December 31, 2025 and
2024, respectively
5
5
Additional paid-in capital
1,229,442
1,192,536
Accumulated other comprehensive loss
(687)
(741)
Accumulated deficit
(1,126,027)
(932,149)
Total stockholders' equity
102,733
259,651
Total liabilities and stockholders' equity
$453,032
$465,989
REGENXBIO INC.CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(unaudited)(in thousands, except per share data)
Three Months
Years
Ended December 31,
Ended December 31,
2025
2024
2025
2024
Revenues
License and royalty revenue
$27,148
$20,788
$156,267
$81,960
Service revenue
3,189
426
14,174
1,368
Total revenues
30,337
21,214
170,441
83,328
Operating Expenses
Cost of license and royalty revenues
5,928
6,318
20,298
33,567
Research and development
59,611
50,380
228,299
208,522
General and administrative
22,380
20,051
82,863
76,619
Credit losses (recoveries)
—
(5,000)
—
(5,000)
Impairment of long-lived assets
—
—
—
2,101
Other operating expenses
54
833
179
865
Total operating expenses
87,973
72,582
331,639
316,674
Loss from operations
(57,636)
(51,368)
(161,198)
(233,346)
Other Income (Expense)
Interest income from licensing
18
83
83
174
Investment income
2,745
9,516
12,245
18,729
Interest expense
(12,276)
(9,417)
(45,008)
(12,659)
Total other income (expense)
(9,513)
182
(32,680)
6,244
Net loss
$(67,149)
$(51,186)
$(193,878)
$(227,102)
Other Comprehensive Income (Loss)
Unrealized gain (loss) on available-for-sale securities, net
(35)
(159)
54
3,688
Total other comprehensive income (loss)
(35)
(159)
54
3,688
Comprehensive loss
$(67,184)
$(51,345)
$(193,824)
$(223,414)
Net loss per share, basic and diluted
$(1.30)
$(1.01)
$(3.76)
$(4.59)
Weighted-average common shares outstanding, basic and diluted
51,752
50,871
51,573
49,509
View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-reports-fourth-quarter-and-full-year-2025-financial-results-and-operational-highlights-302704786.htmlSOURCE REGENXBIO Inc.
Original: REGENXBIO Reports Fourth Quarter and Full Year 2025 Financial Results and Operational Highlights
US Market News
4月前
REGENXBIO Announces Regulatory Update on RGX-121 BLA for MPS IIFebruary 9, 2026 4:05 PM
PR Newswire (US)
FDA issues Complete Response Letter for RGX-121 (clemidsogene lanparvovec) for treatment of Mucopolysaccharidosis II (MPS II) REGENXBIO plans to work with FDA on a path forward with the goal of resubmitting the BLAROCKVILLE, Md., Feb. 9, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding its Biologics License Application (BLA) for RGX-121 (clemidsogene lanparvovec) for the treatment of Mucopolysaccharidosis II (MPS II), an ultra-rare neurodegenerative disease also known as Hunter syndrome.In May 2025, the FDA accepted the RGX-121 BLA under the accelerated approval pathway. In the February 7, 2026 CRL, the FDA stated that it had agreed to the study protocol in principle and outlined several reasons for not approving the gene therapy, including uncertainty regarding the study eligibility criteria to adequately define a population with neuronopathic disease (vs. attenuated disease), the comparability of the natural history external control to the study population, and the appropriateness of CSF HS D2S6 as a surrogate endpoint reasonably likely to predict clinical benefit. The CRL lists several potential paths forward, including a new study, treating additional patients and conducting longer-term follow up, and using an untreated control arm, all of which would be challenging in an ultra-rare disease population, like MPS II."This decision is devastating for the families of boys living with this progressive, life-threatening disease," said Curran Simpson, President and CEO of REGENXBIO. "We are concerned about FDA's feedback regarding the overall development path and evaluation of the data in the context of the urgent need for this irreversible ultra-rare disease. We remain confident in the quality and volume of evidence demonstrating the long-term potential of RGX-121 to positively change the trajectory of Hunter syndrome. This program has been in development for over 10 years. We are incredibly grateful to all the patients, their families, investigators, and site staff who have supported this program and our continued efforts to bring a much-needed new treatment option to the Hunter syndrome community. We will continue those efforts."Throughout active discussions during the BLA process, REGENXBIO believed it had addressed the points raised in the CRL through the submission of additional data and responses to numerous information requests. Independent, leading global MPS and biomarker experts conducted analyses and reviews with the FDA, as well. Ultimately, the FDA did not agree the data set provided substantial evidence of effectiveness to support approval of RGX-121 for the treatment of MPS II.REGENXBIO plans to request a Type A meeting to discuss the CRL, as well as the planned BLA resubmission to provide additional evidence from global MPS II experts to further clarify the neuronopathic patient population and additional longer-term clinical data to support evidence of effectiveness. REGENXBIO intends to find a path forward as quickly as possible with the goal of resubmitting the BLA."MPS II is a very complex disease, but its impact is well established, resulting in irreversible brain damage for the majority of patients; without appropriate treatments stopping this neurocognitive decline, the neuronopathic MPS II child will die prematurely, usually in their mid-teens," said Joseph Muenzer, M.D., Ph.D., Director, Muenzer MPS Research and Treatment Center, Bryson Distinguished Professor in the Division of Genetics and Metabolism, Department of Pediatrics Genetics, University of North Carolina at Chapel Hill. "I remain encouraged by the clinical data behind RGX-121. New innovations like gene therapy could make a significant impact for these patients, and time is precious for these families.""I've seen the severe impact of MPS II on patients and their families firsthand and am extremely disheartened by today's news," said Terri Klein, President and CEO, National MPS Society. "Families know the devastating trajectory of this disease all too well and have waited 20 years for new treatment options. They cannot wait any longer. Drug development for ultra-rare disease must be streamlined to allow new medicines to reach patients. We urge the FDA to find a swift path forward so that boys living with MPS II and their families have the chance for a better life."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 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.The BLA for RGX-121 for MPS II was supported by positive biomarker, functional and safety data from the CAMPSIITE I/II/III trial, including out to 12 months. RGX-121 has been well tolerated in all patients dosed across all phases of the CAMPSIITE trial.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 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 www.REGENXBIO.com.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 WWW.SEC.GOV. 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
Dcormack@regenxbio.com George E. MacDougall
Investor Relations
IR@regenxbio.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-announces-regulatory-update-on-rgx-121-bla-for-mps-ii-302682941.htmlSOURCE REGENXBIO Inc.
Original: REGENXBIO Announces Regulatory Update on RGX-121 BLA for MPS II
US Market News
4月前
REGENXBIO Announces Regulatory Update on Ultra Rare MPS ProgramsJanuary 28, 2026 12:30 PM
PR Newswire (US)
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 a-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 www.REGENXBIO.com.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 WWW.SEC.GOV. 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
Dcormack@regenxbio.comGeorge E. MacDougall
Investor Relations
IR@regenxbio.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-announces-regulatory-update-on-ultra-rare-mps-programs-302672061.htmlSOURCE REGENXBIO Inc.
Original: REGENXBIO Announces Regulatory Update on Ultra Rare MPS Programs
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