A Randomized, Partially Masked, Controlled, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD

ABBV-RGX-314 (also known as RGX-314) is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-vascular endothelial growth factor (VEGF) therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. ABBV-RGX-314 is being developed as a potential one-time treatment for wet AMD.

Start Date28 Dec 2021

Sponsor / Collaborator

AbbVie, Inc.

[+1]

NCT05210803 / Enrolling by invitationNot Applicable

A Long-term Follow-Up Study to Evaluate the Safety and Efficacy of Suprachoroidal Administration of RGX-314 for Participants With Neovascular Age-Related Macular Degeneration

This is a prospective, observational study designed to evaluate the long-term safety and efficacy of RGX-314. Eligible participants are those who were previously enrolled in a clinical study of nAMD in which they received suprachoroidal space (SCS) administration of RGX-314. Enrollment of each participant in the current study should occur after the participant has completed either the end of study or early discontinuation visit in the previous (parent) clinical study. Participants will be followed for up to 5 years after RGX-314 administration (inclusive of the parent study). As such, the total study duration for each participant may vary depending on when they enroll in the current study following RGX-314 administration in the parent study.

Start Date20 Dec 2021

Sponsor / Collaborator

AbbVie, Inc.

NCT04832724 / CompletedPhase 2

A Phase 2, Open-label Study to Explore the Pharmacodynamics of Two Doses in Two Formulations of RGX-314 Gene Therapy Administered Via Subretinal Delivery in Participants With Neovascular Age-related Macular Degeneration

RGX-314 is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (nAMD) also referred to as Wet AMD. Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. The purpose of this phase 2, open label study is to evaluate whether different doses of RGX-314 from two different formulations (clinical versus eventual commercial formulation) perform the same in humans when delivered by subretinal administration

Start Date22 Feb 2021

Sponsor / Collaborator

AbbVie, Inc.

100 Clinical Results associated with RGX-314

100 Translational Medicine associated with RGX-314

100 Patents (Medical) associated with RGX-314

Literatures (Medical) associated with RGX-314

01 Apr 2024·Lancet (London, England)

Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study

Article

Author: Huddleston, Stephen M ; Brown, David M ; Ho, Allen C ; Heier, Jeffrey S ; Campochiaro, Peter A ; Khanani, Arshad M ; Jaffe, Glenn J ; Pakola, Stephen ; Avery, Robert ; Wykoff, Charles C ; Van Everen, Sherri ; Pieramici, Dante J

BACKGROUND:

Frequent anti-vascular endothelial growth factor A (VEGF-A) injections reduce the risk of rapid and severe vision loss in patients with neovascular age-related macular degeneration (nAMD); however, due to undertreatment, many patients lose vision over time. New treatments that provide sustained suppression of VEGF-A are needed. RGX-314 (currently known as ABBV-RGX-314) is an adeno-associated virus serotype 8 vector that expresses an anti-VEGF-A antigen-binding fragment, which provides potential for continuous VEGF-A suppression after a single subretinal injection. We report results on the safety and efficacy of subretinal injection of RGX-314 in patients with nAMD.

METHODS:

For this open-label, multiple-cohort, multicentre, phase 1/2a, dose-escalation study conducted at eight sites in the USA, we enrolled participants with nAMD aged 50-89 years who had previously been treated with anti-VEGF injections into five cohorts (with five different doses of RGX-314). To be eligible, participants had to have macular neovascularisation secondary to nAMD with subretinal or intraretinal fluid in the centre subfield, be pseudophakic (after cataract removal), and have a best-corrected visual acuity (BCVA) in the study eye between 20/63 and 20/400 for the first participant in each cohort and between 20/40 and 20/400 for others. Subretinal injection of RGX-314 was done without a pre-bleb by a wet-laboratory-trained vitreoretinal surgeon. Cohort 1 received 3 × 10⁹ genome copies per eye, cohort 2 received 1 × 10¹⁰, and cohort 3 received 6 × 10¹⁰. Two additional dose cohorts (cohort 4: 1·6 × 10¹¹; cohort 5: 2·5 × 10¹¹) were added. Participants were seen 1 day and 1 week after administration of RGX-314, and then monthly for 2 years (up to week 106). The primary outcome was safety of RGX-314 delivered by subretinal injection up to week 26. This analysis includes all 42 patients enrolled in the study. This study is registered with ClinicalTrials.gov, NCT03066258.

FINDINGS:

Between May 12, 2017, and May 21, 2019, we screened 110 patients for eligibility and enrolled 68. 42 participants demonstrated the required anatomic response to intravitreal ranibizumab and then received a single RGX-314 injection (dose range 3 × 10⁹ to 2·5 × 10¹¹ genome copies per eye) and were followed up for 2 years. There were 20 serious adverse events in 13 participants, of which one was possibly related to RGX-314: pigmentary changes in the macula with severe vision reduction 12 months after injection of RGX-314 at a dose of 2·5 × 10¹¹ genome copies per eye. Asymptomatic pigmentary changes were seen in the inferior retinal periphery several months after subretinal injection of RGX-314 most commonly at doses of 6 × 10¹⁰ genome copies per eye or higher. There were no clinically determined immune responses or inflammation beyond that expected following routine vitrectomy. Doses of 6 × 10¹⁰ genome copies or higher resulted in sustained concentrations of RGX-314 protein in aqueous humour and stable or improved BCVA and central retinal thickness with few or no supplemental anti-VEGF-A injections in most participants.

INTERPRETATION:

Subretinal delivery of RGX-314 was generally well tolerated with no clinically recognised immune responses. RGX-314 gene therapy provides a novel approach for sustained VEGF-A suppression in patients with nAMD that has potential to control exudation, maintain vision, and reduce treatment burden after a single administration. Results from this study informed the pivotal programme to evaluate RGX-314 in patients with nAMD.

FUNDING:

RegenxBio.

03 Oct 2022·Expert opinion on investigational drugs

Investigational drugs in clinical trials for macular degeneration

Review

Author: Tolentino, Michael J ; Tolentino, Andrew J

INTRODUCTION:

Intravitreal anti-vascular endothelial growth factor (VEGF) injections for exudative age-related macular degeneration (eAMD) are effective and safe but require frequent injections and have nonresponding patients. Geographic atrophy/dry AMD (gaAMD) remains an unmet medical need. New therapies are needed to address this leading cause of blindness in the increasing aged population.

AREAS COVERED:

This paper reviews the pathogenesis of macular degeneration, current and failed therapeutics, therapies undergoing clinical trials and a rationale for why certain AMD therapies may succeed or fail.

EXPERT OPINION:

VEGF-inhibitors reduce both vascular leakage and neovascularization. Experimental therapies that only address neovascularization or leakage will unlikely supplant anti-VEGF therapies. The most promising future therapies for eAMD, are those that target, more potently inhibit and have a more sustained effect on the VEGF pathway such as KSI-301, RGX-314, CLS-AX, EYEP-1901, OTX-TKI. GaAMD is a phenotype of phagocytic retinal cell loss. Inhibiting phagocytic activity of retinal microglial/macrophages at the border of geographic atrophy and reducing complement derived activators of microglial/macrophage is the most promising strategy. Complement inhibitors (Pegcetacoplan and Avacincaptad pegol) will likely obtain FDA approval but will serve to pave the way for combined complement and direct phagocytic inhibitors such as AVD-104.

01 Feb 2022·Eye (London, England)Q3 · MEDICINE

Review of gene therapies for age-related macular degeneration

Q3 · MEDICINE

Review

Author: Danzig, Carl J ; Yiu, Glenn ; Kaiser, Peter K ; Waheed, Nadia K ; Aziz, Aamir A ; Weng, Christina Y ; Khanani, Arshad M ; Thomas, Mathew J ; Kiss, Szilárd

Gene therapies aim to deliver a therapeutic payload to specified tissues with underlying protein deficiency. Since the 1990s, gene therapies have been explored as potential treatments for chronic conditions requiring lifetime care and medical management. Ocular gene therapies target a range of ocular disorders, but retinal diseases are of particular importance due to the prevalence of retinal disease and the current treatment burden of such diseases on affected patients, as well as the challenge of properly delivering these therapies to the target tissue. The purpose of this review is to provide an update on the most current data available for five different retinal gene therapies currently undergoing clinical trials for use against age-related macular degeneration (AMD) and the development of novel delivery routes for the administration of such therapies. Research has been performed and compiled from PubMed and the select authors of this manuscript on the treatment and effectiveness of five current retinal gene therapies: Luxturna, ADVM-022, RGX-314, GT-005, and HMR59. We present the available data of current clinical trials for the treatment of neovascular and dry age-related macular degeneration with different AAV-based gene therapies. We also present current research on the progress of developing novel routes of administration for ocular gene therapies. Retinal gene therapies offer the potential for life-changing treatment for chronic conditions like age-related macular degeneration with a single administration. In doing so, gene therapies change the landscape of treatment options for these chronic conditions for both patient and provider.

News (Medical) associated with RGX-314

12 Nov 2024

·www.globenewswire.com

Clearside Biomedical Announces Third Quarter 2024 Financial Results and Provides Corporate Update

- Recent ODYSSEY Phase 2b Trial of Suprachoroidal CLS-AX in Wet AMD Achieved All Primary and Secondary Outcomes - - Positive Topline Results Support Advancing CLS-AX to Phase 3 Targeting a Differentiated Flexible Dosing Approach Similar to a Biologic with the Potential Extended Duration of a Tyrosine Kinase Inhibitor (TKI) - - Recent Commercial Licensing Agreement for China by a Global Ophthalmic Pharmaceutical Company Provides Strategic Validation of Clearside’s Suprachoroidal Platform - - Compelling Data Presentations at AAO Demonstrated Potential Safety and Efficacy Benefits of Suprachoroidal Delivery Using Clearside’s Proprietary SCS Microinjector® in Multiple Clinical Programs - - Management to Host Webcast and Conference Call Today at 4:30 P.M. ET - ALPHARETTA, Ga., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), today reported financial results for the third quarter ended September 30, 2024, and provided a corporate update. “We are making outstanding progress advancing our differentiated suprachoroidal delivery pipeline,” said George Lasezkay, PharmD, JD, President and Chief Executive Officer. “The recent positive results from our ODYSSEY trial establish CLS-AX as a Phase 3 ready asset in the large and growing wet AMD market. We are positioning CLS-AX for real-world success by focusing on a Phase 3 program in wet AMD designed to evaluate extended efficacy duration compared to current standard of care intravitreal products and produce data supportive of a prescribing label that enables physicians to take advantage of flexible maintenance dosing between 3 and 6 months. We look forward to conducting an End-of-Phase 2 meeting with the FDA in early 2025 to align on the essential components of our Phase 3 program.” “As we work to expand the overall value of our suprachoroidal drug delivery platform, we are seeing significant interest among the retinal specialist community and from leading biopharmaceutical companies in applying our innovative approach to treating serious retinal diseases. The recent commercial collaboration announced by Santen Pharmaceutical Co. and our Asia-Pacific partner, Arctic Vision, is a compelling validation of our suprachoroidal platform from a well-respected leader in the global ophthalmic industry. The licensing of ARVN001, branded as XIPERE® in the U.S., is part of Santen’s commitment to bringing innovative eyecare solutions to patients in China,” concluded Dr. Lasezkay. Victor Chong, M.D., MBA, Chief Medical Officer and EVP, Head of Research & Development, added, “In addition to our partners’ promising programs, our research team is currently evaluating various small molecules through in vivo models for the potential treatment of geographic atrophy (GA), with a market size valued at over $20 billion in sales. We believe that GA is primarily a choroidal disease. Delivery of small molecules via suprachoroidal injection enables comprehensive drug coverage of both the retina and choroid, while potentially minimizing systemic and anterior segment side effects.” Key Highlights The ODYSSEY Phase 2b clinical trial of CLS-AX (axitinib injectable suspension) for the treatment of neovascular age-related macular degeneration (wet AMD) achieved its primary and secondary outcomes, demonstrated extended duration, stable vision and anatomic measures and a well-tolerated safety profile.Clearside’s Asia-Pacific partner, Arctic Vision, signed a new commercial collaboration with Santen Pharmaceutical Co., Ltd. for commercial rights in China to ARVN001, Arctic Vision’s triamcinolone acetonide injectable suspension for suprachoroidal use, in the treatment of uveitic macular edema (UME) and certain other ophthalmic indications under development. ARVN001 is branded as XIPERE in the U.S. and ARCATUS® in China.Arctic Vision reported positive topline results from its Phase 3 clinical trial of ARCATUS for the treatment of UME in China and announced that New Drug Applications for ARCATUS are under review by regulators in Australia and Singapore.Tony Gibney was appointed Chair of Clearside’s Board of Directors, effective November 1, 2024, succeeding Clay Thorp, who will continue serving as a member of the Board. Mr. Gibney joined Clearside’s Board as an independent director in April 2024 and is an experienced biotechnology executive and former investment banker, most recently serving as Executive Vice President, Chief Business & Strategy Officer, of Iveric Bio, Inc. until the company’s acquisition by Astellas Pharma Inc. in July 2023.Glenn Yiu, MD, PhD, Professor of Ophthalmology at the University of California, Davis, was appointed to Clearside’s Scientific Advisory Board in July 2024. Dr. Yiu, a board-certified vitreoretinal surgeon, leads the translational research program at UC Davis studying AMD and other retinal diseases, with a focus on ocular imaging technologies, gene editing and delivery, and animal models of retinal disease.An article was published titled “Early Adoption of Triamcinolone Acetonide Suprachoroidal Injection for UME: A Physician Survey” by Christopher R. Henry et al. that summarizes physicians’ “real-world” perspectives on early experiences with XIPERE for the treatment of patients with UME. Findings from this survey indicate that the suprachoroidal injection technique was easy to learn (92% found the injection procedure relatively easy post-training) and resulted in favorable patient outcomes consistent with clinical trial data. The full publication can be accessed here.Clearside’s gene therapy partner, REGENXBIO, reported on both of their programs administering ABBV-RGX-314 via Clearside’s SCS Microinjector. Based on positive interim results to date from the Phase 2 ALTITUDE® trial in diabetic retinopathy (DR), AbbVie and REGENXBIO announced that they have accelerated a planned End-of-Phase 2 meeting with the FDA expected this quarter. REGENXBIO expects to initiate the first global pivotal trial in DR in the first half of 2025. In addition, the ALTITUDE trial is enrolling a new cohort of patients with center-involved diabetic macular edema. In wet AMD, based on a favorable safety profile and to evaluate dose levels for a planned pivotal program, the Phase 2 AAVIATE® trial is enrolling a new cohort.In September 2024, Clearside’s ocular oncology partner, Aura Biosciences, presented positive Phase 2 end-of-study results evaluating bel-sar (AU-011) for the first-line treatment of early-stage choroidal melanoma at The Retina Society Annual Meeting. Bel-sar is being administered via Clearside’s SCS Microinjector.Multiple presentations were delivered at the 2024 Annual Meeting of the American Academy of Ophthalmology (AAO) and preceding events that highlighted encouraging safety and efficacy data from clinical trials of therapies utilizing Clearside’s SCS Microinjector to deliver drugs into the suprachoroidal space to treat a variety of retinal diseases. Third Quarter 2024 Financial Results License and other revenue for the third quarter of 2024 was $1.0 million, compared to $0.9 million for the third quarter of 2023. The revenue primarily related to payments pursuant to Clearside’s license agreements and revenue for services and the sales of SCS Microinjector kits to licensees.Research and development expenses for the third quarter of 2024 were $4.1 million, compared to $5.1 million for the third quarter of 2023. This decrease was primarily due to a $1.9 million decrease in costs related to the CLS-AX program, which was partially offset by increases in employee-related costs and device development and a research and development tax credit received in the prior year.General and administrative expenses were $2.8 million for the third quarter of 2024, compared to $2.6 million for the third quarter of 2023. This increase was primarily due to an increase in patent-related expenses and consulting fees.Net loss for the third quarter of 2024 was $7.7 million, or $0.10 per share of common stock, compared to net loss of $9.3 million, or $0.15 per share of common stock, for the third quarter of 2023.As of September 30, 2024, Clearside’s cash, cash equivalents and short-term investments totaled $23.6 million. The Company believes it will have sufficient resources to fund its planned operations into the third quarter of 2025. Conference Call & Webcast Details Clearside’s management will host a webcast and conference call today at 4:30 p.m. Eastern Time to discuss the financial results and provide a corporate update. The live and archived webcast may be accessed on the Clearside website under the Investors section: Events and Presentations. The live call can be accessed by dialing (877) 545-0523 (domestic) or (973) 528-0016 (international) and entering conference code: 756568. The Company suggests participants join 15 minutes in advance of the event. About Clearside Biomedical, Inc. Clearside Biomedical, Inc. is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®) to improve patient outcomes. Clearside’s SCS injection platform, utilizing the Company’s patented SCS Microinjector®, enables an in-office, repeatable, non-surgical procedure for the targeted and compartmentalized delivery of a wide variety of therapies to the macula, retina, or choroid to potentially preserve and improve vision in patients with sight-threatening eye diseases. Clearside is developing its own pipeline of small molecule product candidates for administration via its SCS Microinjector. The Company’s lead program, CLS-AX (axitinib injectable suspension), for the treatment of neovascular age-related macular degeneration (wet AMD), recently completed a Phase 2b clinical trial, and planning for a Phase 3 program is underway. Clearside developed and gained approval for its first product, XIPERE® (triamcinolone acetonide injectable suspension) for suprachoroidal use, which is available in the U.S. through a commercial partner. Clearside also strategically partners its SCS injection platform with companies utilizing other ophthalmic therapeutic innovations. For more information, please visit clearsidebio.com or follow us on LinkedIn and X. Cautionary Note Regarding Forward-Looking Statements Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “believe”, “expect”, “may”, “plan”, “potential”, “will”, and similar expressions, and are based on Clearside’s current beliefs and expectations. These forward-looking statements include statements regarding the clinical development of CLS-AX, the potential benefits of CLS-AX, Clearside’s suprachoroidal delivery technology and Clearside’s SCS Microinjector® and Clearside’s ability to fund its operations into the third quarter of 2025. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Clearside’s reliance on third parties over which it may not always have full control and other risks and uncertainties that are described in Clearside’s Annual Report on Form 10-K for the year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission (SEC) on March 12, 2024, Clearside’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2024 filed with the SEC on August 12, 2024 and Clearside’s other Periodic Reports filed with the SEC. Any forward-looking statements speak only as of the date of this press release and are based on information available to Clearside as of the date of this release, and Clearside assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise. *References XIPERE® (triamcinolone acetonide injectable suspension) for suprachoroidal use is being commercialized by Bausch + Lomb who has the exclusive license for the commercialization and development of XIPERE in the United States and Canada. Arctic Vision has the exclusive license for the commercialization and development of XIPERE, in Greater China, South Korea, Australia, New Zealand, India and the ASEAN Countries. XIPERE is approved by the U.S. Food and Drug Administration and is commercially available in the U.S. A link to the full prescribing information is available at https://www.xipere.com/hcp/#isi.Source: IMARC Geographic Atrophy Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034ALTITUDE® and AAVIATE® are registered trademarks of REGENXBIO, Inc. Investor and Media Contacts: Jenny Kobin Remy Bernarda ir@clearsidebio.com(678) 430-8206 -Financial Tables Follow- CLEARSIDE BIOMEDICAL, INC.Selected Financial Data (in thousands, except share and per share data)(unaudited) Statements of Operations Data Three Months EndedSeptember 30, Nine Months EndedSeptember 30, 2024 2023 2024 2023 License and other revenue $1,038 $859 $1,358 $1,881 Operating expenses: Cost of goods sold — 142 — 355 Research and development 4,128 5,134 14,346 14,533 General and administrative 2,844 2,637 8,745 8,922 Total operating expenses 6,972 7,913 23,091 23,810 Loss from operations (5,934) (7,054) (21,733) (21,929)Interest income 338 409 1,104 1,359 Other income, net 365 — 783 — Non-cash interest expense on liability related to the sales of future royalties (2,457) (2,622) (7,200) (7,083)Net loss $(7,688) $(9,267) $(27,046) $(27,653)Net loss per share of common stock — basic and diluted $(0.10) $(0.15) $(0.37) $(0.45)Weighted average shares outstanding — basic and diluted 74,745,415 61,983,987 73,115,896 61,605,648 Balance Sheet DataSeptember 30, December 31, 2024 2023 Cash and cash equivalents$13,888 $28,920 Short-term investments 9,703 — Total assets 29,161 34,018 Liabilities related to the sales of future royalties, net 49,188 41,988 Warrant liabilities 8,757 — Total liabilities 63,950 49,930 Total stockholders’ deficit (34,789) (15,912) Source: Clearside Biomedical, Inc.

Clinical ResultPhase 2Phase 3License out/inExecutive Change

06 Nov 2024

·www.prnewswire.com

REGENXBIO Reports Third Quarter 2024 Financial Results and Recent Operational Updates

Advancement in Phase I/II AFFINITY DUCHENNE® trial of RGX-202 for Duchenne Muscular Dystrophy; pivotal trial initiation and first functional data expected this month BLA submission for RGX-121 initiated and expected to complete in Q1 2025 Positive Phase II data support bilateral administration of subretinal ABBV-RGX-314; data consistent with that from multiple previous studies demonstrating favorable safety and efficacy profile End-of-Phase II meeting for ABBV-RGX-314 in diabetic retinopathy accelerated to Q4 2024 to support global pivotal program initiation in H1 2025 Conference call today at 4:30 p.m. ET ROCKVILLE, Md., Nov. 6, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and recent operational highlights for the third quarter ended September 30, 2024. "It has been a turning point year for REGENXBIO, as we are on the cusp of advancing AFFINITY DUCHENNE® to pivotal phase, completing our first BLA for MPS II and entering pivotal phase in a second indication within our global eyecare collaboration with AbbVie," said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. "The rapid progress we are making in RGX-202, which continues to demonstrate its potential to be a best-in-class gene therapy for Duchenne, is highly encouraging, and the near-term filing of our BLA for RGX-121 represents a major milestone for the patient community in need of a treatment to address both the neurocognitive and systemic effects of MPS II. The recent fellow eye data presented at the American Academy of Ophthalmology meeting demonstrate the potential of ABBV-RGX-314 to preserve vision long-term for patients with wet AMD as a one-time treatment for both eyes. Each of these programs represent one-time treatments with the potential to transform the trajectory and management of disease for patients in need of new and better options, and we look forward to continued momentum and important milestones in the last quarter of the year." PROGRAM HIGHLIGHTS AND MILESTONES Neuromuscular Disease: RGX-202 is a potential best-in-class gene therapy designed to deliver a differentiated, novel microdystrophin gene for improved function and outcomes for patients living with Duchenne. In the Phase I/II AFFINITY DUCHENNE trial of RGX-202, the last patient has been dosed in the dose level 2 (pivotal dose) expansion cohort for ages 4-11 and the first patient has been dosed in the cohort for ages 1-3. Patients with Duchenne under 4 years old have no access to gene therapy, and REGENXBIO is the only gene therapy sponsor recruiting patients in this age group in the U.S. A clinical trials application (CTA) for RGX-202 has been authorized by Health Canada. REGENXBIO expects to initiate sites in Canada in H1 2025. REGENXBIO plans to share a full program update this month, including pivotal trial design and plans for accelerated approval, as well as initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial. Retinal Disease: ABBV-RGX‑314, being developed in collaboration with AbbVie, is potentially the first-in-class treatment for wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR). ABBV-RGX-314 for Treatment of DR (Suprachoroidal Delivery) Based on positive interim results to date from the Phase II ALTITUDE® trial, AbbVie and REGENXBIO have accelerated a planned End-of-Phase II meeting with the U.S. Food and Drug Administration (FDA) expected this quarter. The Company expects to initiate the first global pivotal trial in H1 2025. The ALTITUDE trial is enrolling a new cohort of patients with center-involved diabetic macular edema (DME). DME is a vision-threatening complication of DR; an estimated 34 million people globally have DME. Patients will receive a one-time, in-office injection of ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye) with short course prophylactic steroid eye drops. ABBV-RGX-314 for the Treatment of Wet AMD (Subretinal Delivery) Enrollment is on track in the ATMOSPHERE® and ASCENT™ pivotal trials and these trials are expected to support global regulatory submissions with the FDA and European Medicines Agency in H1 2026. Positive data from the Phase II fellow eye sub-study evaluating the subretinal delivery of ABBV-RGX-314 in patients with bilateral wet AMD were recently presented at the American Academy of Ophthalmology annual meeting. As of September 11, 2024, ABBV-RGX-314 was well tolerated in the treated fellow eye, with no drug-related serious adverse events and no cases of intraocular inflammation observed. At nine months post-administration, patients saw a 97% reduction in anti-VEGF treatment burden and sustained vision and anatomy. These results support the potential of ABBV-RGX-314 to treat bilateral disease at an expected commercial launch. ABBV-RGX-314 for the Treatment of Wet AMD (Suprachoroidal Delivery) Based on a favorable safety profile and to evaluate dose levels for a planned pivotal program, the Phase II AAVIATE® trial is enrolling a new cohort to evaluate ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye). Patients in this cohort will also receive short course prophylactic steroid eye drops. At dose level 3, patients receiving ABBV-RGX-314 demonstrated an 80% reduction in annualized injection rate, with 50% of patients remaining injection-free at six months. Patients also demonstrated stable Best Corrected Visual Acuity and central retinal thickness. Neurodegenerative Disease: RGX-121 is a potential first-in-class treatment for MPS II. REGENXBIO initiated a rolling BLA submission for RGX-121 using the accelerated approval pathway in Q3 2024. The BLA submission is expected to be complete in Q1 2025. RGX-121 is on track to be the potential first gene therapy and one-time treatment approved for MPS II. Approval of RGX-121 could result in receipt of a Priority Review Voucher in 2025. FINANCIAL RESULTS Cash Position: Cash, cash equivalents and marketable securities were $278.6 million as of September 30, 2024, compared to $314.1 million as of December 31, 2023. The decrease was primarily driven by cash used to fund operating activities during the nine months ended September 30, 2024, and was partially offset by $131.1 million of aggregate net proceeds received from the follow-on public offering of the Company's common stock and pre-funded warrants completed in March 2024. Revenues: Revenues were $24.2 million for the three months ended September 30, 2024, compared to $28.9 million for the three months ended September 30, 2023. The decrease was primarily attributable to Zolgensma royalty revenues, which decreased from $28.4 million for the third quarter of 2023 to $23.9 million for the third quarter of 2024. Research and Development Expenses: Research and development expenses were $54.4 million for the three months ended September 30, 2024, compared to $58.2 million for the three months ended September 30, 2023. The decrease was largely driven by lower personnel-related costs and early-stage research and development activities, and was partially offset by increases in clinical trial expenses for ABBV-RGX-314 and RGX-202. General and Administrative Expenses: General and administrative expenses were $19.4 million for the three months ended September 30, 2024, compared to $23.1 million for the three months ended September 30, 2023. The decrease was primarily attributable to expenses for professional services and other corporate overhead costs. Net Loss: Net loss was $59.6 million, or $1.17 basic and diluted net loss per share, for the three months ended September 30, 2024, compared to a net loss of $61.9 million, or $1.41 basic and diluted net loss per share, for the three months ended September 30, 2023. FINANCIAL GUIDANCE REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $278.6 million as of September 30, 2024 to fund its operations into 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. CONFERENCE CALL In connection with this announcement, REGENXBIO will host a conference call and webcast at 4:30 p.m. ET. 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 leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for rare and retinal diseases, including RGX-202 for the treatment of Duchenne, ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, and RGX-121 for the treatment of MPS II. Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA® for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics 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, 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, 2023, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at . All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. Zolgensma® is a registered trademark of Novartis AG. All other trademarks referenced herein are registered trademarks of REGENXBIO. CONTACTS: Dana Cormack Corporate Communications [email protected] 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

Phase 2Clinical ResultGene TherapyFinancial Statement

30 Oct 2024

·www.prnewswire.com

REGENXBIO to Host Conference Call on November 6 to Discuss Third Quarter 2024 Financial Results and Recent Operational Highlights

ROCKVILLE, Md., Oct. 30, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Wednesday, November 6, at 4:30 p.m. ET to discuss its financial results for the third quarter ended September 30, 2024, and recent operational highlights. 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 leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for rare and retinal diseases, including RGX-202 for the treatment of Duchenne, ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, and RGX-121 for the treatment of MPS II. Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' Zolgensma® for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people. For more information, please visit . Contacts: Dana Cormack Corporate Communications [email protected] Investors: 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

Gene TherapyFinancial Statement

100 Deals associated with RGX-314

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Choroidal Neovascularization	Phase 3	US	AbbVie, Inc.	28 Dec 2021
Choroidal Neovascularization	Phase 3	JP	AbbVie, Inc.	28 Dec 2021
Choroidal Neovascularization	Phase 3	CA	AbbVie, Inc.	28 Dec 2021
Choroidal Neovascularization	Phase 3	FR	AbbVie, Inc.	28 Dec 2021
Choroidal Neovascularization	Phase 3	DE	AbbVie, Inc.	28 Dec 2021
Choroidal Neovascularization	Phase 3	HU	AbbVie, Inc.	28 Dec 2021
Choroidal Neovascularization	Phase 3	IT	AbbVie, Inc.	28 Dec 2021
Choroidal Neovascularization	Phase 3	PR	AbbVie, Inc.	28 Dec 2021
Choroidal Neovascularization	Phase 3	ES	AbbVie, Inc.	28 Dec 2021
Choroidal Neovascularization	Phase 3	GB	AbbVie, Inc.	28 Dec 2021

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04514653 (AAVIATE, Corporate Publications) Manual	Phase 2	Wet Macular Degeneration	106	ABBV-RGX-314	ofsukzjnzb(zurtbykxcb) = in the study eye were mild or moderate and included conjunctival hemorrhage, increased intraocular pressure, episcleritis, and conjunctival hyperemia. xzqmhzgkhp (vxukluwpqd ) View more	Positive	16 Jan 2024
EURETINA2023	Not Applicable	Diabetic macular oedema	-	RGX-314 at a dose level of 2.5 x E11 GC/eye	gujhwxexhx(dauyyazfzl) = three cases of mild intraocular inflammation through 6 months which resolved with topical corticosteroids hhanthhejr (ffgtjdmeyr ) View more	-	05 Oct 2023
EURETINA2023	Not Applicable	Diabetic macular oedema	-	RGX-314 at an increased dose level of 5 x E11 GC/eye		-	05 Oct 2023
EURETINA2023	Not Applicable	Age Related Macular Degeneration	-	RGX-314	tmlqgvyvbb(paraekyssz) = No new drug-related ocular adverse events (AEs) have been reported in Cohorts 1-4 blnchjgzng (xzyiugbbev ) View more	-	05 Oct 2023
Phase I/IIa (ARVO2023)	Phase 1/2	Wet age-related macular degeneration	42	RGX-314	zibmwemrky(rspisspnig) = no new drug-related ocular adverse events reported in Cohorts 1-4 and one drug-related adverse event of significantly decreased BCVA in Cohort 5 ogkzjhtzvo (rikocyqfly )	Positive	23 Apr 2023
NCT04567550 (ALTITUDE, Corporate Publications) Manual	Phase 2	Diabetic Retinopathy	60	RGX-314	szmqgcgctk(wyteralmpe) = csvvuzorvl zslcqjshlz (qkokgpgkje ) View more	Positive	02 Nov 2022
NCT04567550 (ALTITUDE, Corporate Publications) Manual	Phase 2	Diabetic Retinopathy	60	control	szmqgcgctk(wyteralmpe) = cpmswksxtw zslcqjshlz (qkokgpgkje ) View more	Positive	02 Nov 2022
NCT04567550 (ALTITUDE, Corporate Publications) Manual	Phase 2	Diabetic Retinopathy	20	RGX-314	xyegmisuod(ywhbgfsqxu) = mgwjdcbvfr gnixlalsmh (wowvdnvkvt ) View more	Positive	13 Sep 2022
NCT04567550 (ALTITUDE, Corporate Publications) Manual	Phase 2	Diabetic Retinopathy	20	Observational	xyegmisuod(ywhbgfsqxu) = zufgvkpowu gnixlalsmh (wowvdnvkvt ) View more	Positive	13 Sep 2022
NCT04514653 (AAVIATE, PRNewswire) Manual	Phase 2	Wet age-related macular degeneration	19	RGX-314	intwklpucn(qdsoulzarz) = tqpmqvexcz odkuiutmkk (naixlahydf ) View more	Positive	01 Oct 2021
NCT04514653 (AAVIATE, PRNewswire) Manual	Phase 2	Wet age-related macular degeneration	19	Ranibizumab	intwklpucn(qdsoulzarz) = slzxrrlsni odkuiutmkk (naixlahydf ) View more	Positive	01 Oct 2021
EURETINA2021	Not Applicable	Wet age-related macular degeneration	-	RGX-314	nlxzihajrs(otkhitwlha) = 20 serious adverse events (SAEs) reported in 13 patients, including one possibly drug-related SAE of significant decrease in vision in Cohort 5 ukevdctbar (fnkwmftghs ) View more	-	01 Sep 2021
RGX-314 (ASGCT2020)	Phase 1/2	Wet age-related macular degeneration	42	RGX-314 gene therapy	awdjrkwvxs(otnpooogtd) = improved central retinal thickness (-83 µm) glinyfbven (tbqaqkfcev ) View more	Positive	28 Apr 2020

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