RGX-202 (Regenxbio)

RGX-202 in Duchenne muscular dystrophy on track for BLA submission mid-2026 Pivotal trial more than half enrolled, with completion expected in 2025 Additional Phase I/II functional data expected 1H 2025 Initiating commercial supply manufacturing in Q3 2025 FDA acceptance of Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121, partnered with Nippon Shinyaku) expected in May 2025 Pivotal data evaluating the safety and efficacy of the subretinal delivery of surabgene lomparvovec (ABBV-RGX-314, partnered with AbbVie) in patients with wet age-related macular degeneration are expected in 2026 and planning of diabetic retinopathy pivotal study continues Conference call today at 4:30 p.m. ET ROCKVILLE, Md., May 12, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and recent operational highlights for the first quarter ended March 31, 2025. "We have made tremendous progress towards delivering multiple commercial gene therapies, starting this year," said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. "Each of our late-stage assets is demonstrating differentiation against standard of care or available treatments, giving us a pipeline of potential first- or best-in-class gene therapies for rare and retinal diseases. With advanced clinical programs, commercial-ready manufacturing in-house at our Rockville, MD headquarters, and a strong balance sheet, REGENXBIO is well-equipped to deliver potentially transformative gene therapies to patients in need." PROGRAM HIGHLIGHTS AND MILESTONES Neuromuscular Disease: RGX-202 is designed to use the NAV® AAV8 vector to deliver a differentiated, novel microdystrophin gene for improved muscle function and outcomes for patients living with Duchenne. It is the only investigational or approved microdystrophin gene therapy construct to include the C-Terminal (CT) domain found in naturally occurring dystrophin. The pivotal Phase I/II/III AFFINITY DUCHENNE® trial of RGX-202 is ongoing in ambulatory patients and is expected to enroll approximately 30 patients aged 1+ in the U.S. and Canada by 2025, with more than half already enrolled to support the pivotal dataset. REGENXBIO expects to share topline data in the first half of 2026 and submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026. Commercial readiness activities are underway to support an expected launch in 2027, when the majority of the prevalent market will still be available. REGENXBIO has manufactured RGX-202 supply for both clinical and confirmatory trials with the highest product purity levels in Duchenne gene therapies and expects to initiate commercial supply manufacturing in Q3 2025, with all production sourced in the U.S. Data continue to show consistent, robust expression and transduction of RGX-202 microdystrophin across all ages, supported by new biomarker data from two patients who received the pivotal dose of RGX-202 in the Phase I/II portion of the trial, which were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in March. In a patient aged 3 years at dosing, microdystrophin expression was measured to be 122.3% compared to control. In a patient aged 7 years old, RGX-202 microdystrophin expression was measured to be 31.5% compared to control. REGENXBIO expects to share additional functional, efficacy, and safety data in the first half of 2025. Neurodegenerative Disease: Clemidsogene lanparvovec (RGX-121) is a potential first-in-class treatment for MPS II, also known as Hunter syndrome, being developed and potentially commercialized in partnership with Nippon Shinyaku. This month, REGENXBIO expects the U.S. Food and Drug Administration (FDA) to accept the BLA submitted in March 2025, and potential approval of clemidsogene lanparvovec in the second half of 2025. FDA approval could result in receipt of a Priority Review Voucher (PRV), to which REGENXBIO retains full rights. Clemidsogene lanparvovec remains on track to be the potential first gene therapy and one-time treatment approved for MPS II. 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 DR (Suprachoroidal Delivery) AbbVie and REGENXBIO announced in January 2025 that they will plan a Phase III clinical program. The program is expected to support global regulatory submissions. The Phase II ALTITUDE® trial is enrolling a cohort of patients with center-involved diabetic macular edema (DME). Patients will receive a one-time, in-office injection of sura-vec at dose level 4 (1.5x10e12 GC/eye) with short-course prophylactic steroid eye drops. Sura-vec for the Treatment of Wet AMD (Subretinal Delivery) Enrollment is ongoing in the ATMOSPHERE® and ASCENT™ pivotal trials. REGENXBIO and AbbVie expect to share topline results in 2026. Sura-vec is on track to be the first approved gene therapy for wet AMD. Sura-vec for the Treatment of Wet AMD (Suprachoroidal Delivery) The Phase II AAVIATE® trial continues enrolling a new cohort to evaluate sura-vec at dose level 4 (1.5x10e12 GC/eye). Patients in this cohort will also receive short-course prophylactic steroid eye drops. CORPORATE UPDATES Partnership with Nippon Shinyaku In March 2025, REGENXBIO announced the successful closing of its strategic partnership with Nippon Shinyaku to develop and commercialize clemidsogene lanparvovec for the treatment of MPS II and RGX-111 for the treatment of MPS I in the United States and Asia. Per the agreement, REGENXBIO received $110 million up front and is eligible to receive up to an additional $700 million if certain milestones are achieved. FINANCIAL RESULTS Cash Position: Cash, cash equivalents and marketable securities were $272.7 million as of March 31, 2025, compared to $244.9 million as of December 31, 2024. The increase was primarily attributable to the $110.0 million upfront payment received under the Nippon Shinyaku partnership, and was partially offset by cash used to fund operating activities during the first quarter of 2025, which is generally in line with historical first quarter cash spend. Revenues: Revenues were $89.0 million for the three months ended March 31, 2025, compared to $15.6 million for the three months ended March 31, 2024. The increase was primarily attributable to $71.8 million of license and service revenue recognized under the collaboration with Nippon Shinyaku in the first quarter of 2025. Research and Development Expenses: Research and development expenses were $53.1 million for the three months ended March 31, 2025, compared to $54.8 million for the three months ended March 31, 2024. The decrease was primarily attributable to clinical trial expenses for sura-vec, clemidsogene lanparvovec, and RGX-202, preclinical activities and other early-stage research and development. The decrease was partially offset by an increase in manufacturing-related expenses and other clinical supply costs for the Company's lead product candidates. General and Administrative Expenses: General and administrative expenses were $20.3 million for the three months ended March 31, 2025, compared to $18.3 million for the three months ended March 31, 2024. The increase was primarily attributable to personnel-related costs, expenses for professional services and other corporate overhead costs. Net Income: Net income was $6.1 million, or $0.12 basic and diluted net income per share, for the three months ended March 31, 2025, compared to a net loss of $63.3 million, or $1.38 basic and diluted net loss per share, for the three months ended March 31, 2024. FINANCIAL GUIDANCE REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $272.7 million as of March 31, 2025 to fund its operations into the second half of 2026. 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 potential monetization of a PRV that would be received upon potential approval of clemidsogene lanparvovec. CONFERENCE CALL In connection with this announcement, REGENXBIO will host a conference call and webcast at 4:30 p.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, 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. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ROCKVILLE, Md., May 8, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, taking place in New Orleans, May 13 through 17, 2025. The presentations highlight REGENXBIO's leading end-to-end capabilities across research and early development, late-stage clinical development, and gene therapy manufacturing. Presentations include encore data from late-stage clinical trials of RGX-121 (clemidsogene lanparvovec) for the treatment of MPS II and RGX-202 for the treatment of Duchenne muscular dystrophy, as well as preclinical research supporting the novel construct of RGX-202 with the C-Terminal domain, capsid discovery research, and RGX-202 manufacturing process development enabling industry-leading purity levels in Duchenne gene therapy. Oral Presentations: Abstract Title: Development of a Commercial Manufacturing Process for RGX-202, a Systemically Delivered AAV for the Treatment of Duchenne Muscular Dystrophy Presenter: Donald Startt, Executive Director, Process Development, REGENXBIO Session: AAV Vector Manufacturing: Process Development Date/Time: May 13, 1:30 p.m. CT Abstract Title: RGX-121 (clemidsogene lanparvovec): an Investigational AAV Gene Therapy for the Treatment of Neuronopathic Mucopolysaccharidosis Type II Presenter: Olivier Danos, Ph.D., Executive Vice President, Chief Scientific Officer, REGENXBIO Session: CNS Gene Delivery for Metabolic Diseases: History and Challenges Date/Time: May 14, 8:00 a.m. CT Abstract Title: RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data Presenter: Olivier Danos, Ph.D., Executive Vice President, Chief Scientific Officer, REGENXBIO Session: Gene Therapy Trials: In-Vivo Gene Therapy Modification Date/Time: May 16, 3:45 p.m. CT Poster Presentations: Abstract Title: Impact of Sample Collection Container Material, Hold Time, and Storage Temperature on Adeno-Associated Virus Endotoxin Testing Presenter: Amanda Zhang, Associate Director Vectore Core, REGENXBIO Session: Tuesday Poster Session Date/Time: May 13, 6:00 p.m. CT Abstract Title: AAV-Expressed Microdystrophin Containing Extended C-Terminus Improves Muscle Function and Protects Against Injury in a Mouse Model of Duchenne Muscular Dystrophy Presenter: Benjamin Heithoff, Ph.D., Scientist II, REGENXBIO Session: Wednesday Poster Session Date/Time: May 14, 5:30 p.m. CT Abstract Title: Development of In Vitro Methods for the Analysis of TLR9 Stimulation by AAV Vector Genomes Presenter: Justin Glenn, Ph.D., Senior Scientist, REGENXBIO Session: Wednesday Poster Session Date/Time: May 14, 5:30 p.m. CT Abstract Title: In Vitro and In Vivo Characterization of Oversized AAV Vectors with High Genome Integrity that Encode Microdystrophins with Extended C-Terminal Sequences Presenter: Randolph Qian, Ph.D., Senior Scientist, REGENXBIO Session: Wednesday Poster Session Date/Time: May 14, 5:30 p.m. CT Abstract Title: Blood-Brain Barrier Crossing AAV Vectors Targeting the Transferrin Receptor Engineered Using Two Different Approaches Presenter: Elad Firnberg, Ph.D., Principal Scientist, REGENXBIO Session: Thursday Poster Session Date/Time: May 15, 5:30 p.m. CT 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 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. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED