Core Insights - REGENXBIO Inc. announced positive interim data from the Phase I/II AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, showing consistent functional benefits and safety [1][2][3] Functional Data - RGX-202 participants at dose level 2 (2x10^14 GC/kg) showed significant functional improvements at both 9 and 12 months, exceeding natural history controls on key measures such as the North Star Ambulatory Assessment (NSAA) [4][5] - At 9 months, participants improved an average of 4 points from baseline on NSAA, and at 12 months, the improvement was 4.5 points from baseline, with a 6.8-point increase compared to natural history [5][6] Biomarker Data - Biomarker data indicated robust microdystrophin expression, with one participant aged 2 showing an expression level of 118.6% compared to control [8][10] - The primary endpoint for the pivotal phase is the proportion of participants with microdystrophin expression ≥10% at Week 12 [8] Safety and Tolerability - RGX-202 demonstrated a favorable safety profile with no serious adverse events reported, and common drug-related adverse events included nausea, vomiting, and fatigue [11][12] - The proactive immune modulation regimen and high product purity levels contributed to the positive safety outcomes [11] Pivotal Trial and Future Plans - REGENXBIO is enrolling participants for the pivotal portion of the AFFINITY DUCHENNE trial, aiming to support a Biologics License Application (BLA) submission under accelerated approval by mid-2026 [14][15] - The company plans to share top-line data in the first half of 2026, including biomarker, functional, and safety data [15] About RGX-202 - RGX-202 is designed to improve function and outcomes in Duchenne muscular dystrophy, utilizing a differentiated microdystrophin construct that encodes key regions of dystrophin [17][18] - The therapy aims to support targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV® AAV8 vector [18] About Duchenne Muscular Dystrophy - Duchenne muscular dystrophy is a severe, progressive muscle disease affecting 1 in 3,500 to 5,000 boys born each year, caused by mutations in the dystrophin gene [19] About REGENXBIO Inc. - REGENXBIO is a biotechnology company focused on gene therapy, with a late-stage pipeline including RGX-202 for Duchenne and other investigational therapies for rare diseases [20]

Group 1 - REGENXBIO Inc. will host a webcast to discuss interim functional data from the Phase I/II AFFINITY DUCHENNE® trial of RGX-202, a gene therapy for Duchenne muscular dystrophy [1] - The webcast will feature principal investigator Aravindhan Veerapandiyan, M.D., from Arkansas Children's Hospital [1] - The event is scheduled for June 5, 2025, at 8:00 a.m. EDT, and will be accessible via REGENXBIO's website [2] Group 2 - REGENXBIO is a biotechnology company focused on gene therapy, founded in 2009, and has pioneered AAV gene therapy [3] - The company is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for Duchenne muscular dystrophy [3] - REGENXBIO's investigational therapies have the potential to significantly impact healthcare delivery for millions [3]

Summary of REGENXBIO Conference Call Company Overview - **Company**: REGENXBIO (RGNX) - **Industry**: Biotechnology, specifically focusing on gene therapy Key Points and Arguments Recent Financing Deal - REGENXBIO announced an opportunistic non-dilutive financing deal aimed at pulling forward midterm royalty streams to prepare for pre-commercial launches, including RGX-202 and RGX-314 [2][3] - The financing is characterized as "good debt" since it does not obligate the company to repay if product sales underperform, while retaining the royalty stream if the products become blockbusters [3][4] FDA Interactions and Regulatory Environment - The company is closely monitoring FDA developments, particularly with the nomination of Dr. Prasad, who has been critical of surrogate endpoints [6][7] - REGENXBIO is currently undergoing a review for its Hunter program, with ongoing interactions with the FDA that have been described as routine [7][8] - The company is optimistic about the potential for accelerated approval based on consistent microdystrophin levels associated with functional benefits [8][9] Market Reactions and Competitive Landscape - Following tragic events related to Sarepta's product, there is increased caution among doctors regarding prescribing to non-ambulatory patients, but REGENXBIO has not seen a change in patient enrollment [10][13] - The company views the lowered financial guidance from Sarepta as a potential opportunity, suggesting that a larger prevalence pool may be available by the time REGENXBIO launches its products [15][16] Product Development and Clinical Trials - REGENXBIO is preparing for pivotal studies and expects to report top-line data from these studies in the first half of next year [28][29] - The company is focused on expanding its dataset for the Hunter program and will provide updates on pivotal study enrollment later this year [27][28] Gene Therapy Strategy and Differentiation - REGENXBIO emphasizes its in-house manufacturing capabilities, which allow for the production of 2,500 doses per year at a competitive cost, as a key differentiator in the gene therapy space [49][50] - The company is advancing its subretinal program for wet AMD in collaboration with AbbVie, with pivotal studies enrolling 1,200 patients [37][38] Safety and Efficacy Considerations - The company has implemented a robust immune-modulating regimen to mitigate risks associated with liver-related adverse events, which has been well-received by investigators [12][13] - REGENXBIO is optimistic about the safety profile of its therapies, particularly in the context of the competitive landscape where safety is a critical concern [46][47] Underappreciated Aspects - The company believes that its cash runway has been improved through recent financing and that its in-house manufacturing capabilities are underappreciated aspects of its business model [49][50] Additional Important Content - The company is preparing for potential advisory committee meetings with the FDA but remains optimistic about the likelihood of not needing one based on precedents [29][30] - The potential for a Priority Review Voucher (PRV) upon approval is highlighted, with recent market data indicating values exceeding $150 million [31][32] This summary encapsulates the key discussions and insights from the REGENXBIO conference call, providing a comprehensive overview of the company's current status, strategic direction, and market positioning.

Core Viewpoint - REGENXBIO Inc. has secured a non-dilutive, limited recourse royalty bond agreement of up to $250 million with Healthcare Royalty, which will enhance its cash runway and support upcoming milestones in its gene therapy pipeline [1][2][3] Financing Details - The agreement allows REGENXBIO to receive $150 million at closing, with an extension of cash runway into early 2027 [1][8] - An additional $50 million will be funded upon achieving sales milestones for ZOLGENSMA, and another $50 million will be available upon mutual agreement between the parties [5][6] Strategic Implications - This financing is aimed at advancing late-stage activities, including potential FDA approvals and data readouts for key products such as RGX-121 and RGX-202 [2][3] - The capital infusion is expected to accelerate commercial preparations and maintain REGENXBIO's leadership in rare and retinal gene therapies [2][8] Agreement Terms - HCRx will receive rights to anticipated royalty payments from ZOLGENSMA and other products, with quarterly interest payments based on royalty and milestone revenue [3][4] - The agreement does not include other potential non-dilutive funding sources, such as the sale of a Priority Review Voucher for RGX-121 [6][8] Company Background - REGENXBIO is focused on gene therapy, with a late-stage pipeline targeting rare and retinal diseases, including partnerships with Nippon Shinyaku and AbbVie [8][9] - The company has pioneered AAV gene therapy and aims to improve healthcare delivery through its investigational therapies [9]

Core Insights - The U.S. FDA has accepted the Biologics License Application (BLA) for RGX-121, a gene therapy for Mucopolysaccharidosis II (MPS II), with a target action date of November 9, 2025 [1][5] - NS Pharma will exclusively commercialize RGX-121 in the U.S. upon potential approval, following a strategic partnership with REGENXBIO [2][3] Company Overview - NS Pharma, a subsidiary of Nippon Shinyaku Co., Ltd., focuses on developing and commercializing innovative therapies [8] - REGENXBIO is a clinical-stage biotechnology company specializing in gene therapy, particularly AAV Therapeutics [7] Product Details - RGX-121 is designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system, potentially providing a long-term solution for MPS II [4] - The therapy has received multiple FDA designations, including Orphan Drug Product and Rare Pediatric Disease [5] Disease Context - MPS II, also known as Hunter Syndrome, is a rare genetic disorder caused by a deficiency in the I2S enzyme, leading to significant health challenges, particularly neurological [6]

Core Viewpoint - REGENXBIO Inc. announced that the FDA has accepted the Biologics License Application (BLA) for RGX-121, a potential one-time gene therapy for Mucopolysaccharidosis II (MPS II), with a target action date of November 9, 2025 [1][7]. Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, founded in 2009, and has developed a late-stage pipeline for rare and retinal diseases [6]. - The company has pioneered AAV gene therapy and aims to improve lives through its curative potential [6]. Product Details - RGX-121 (clemidsogene lanparvovec) is designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system, potentially providing a permanent source of the I2S protein [4]. - The therapy aims to address both neurodevelopmental and systemic effects of Hunter syndrome, which currently relies on weekly enzyme replacement therapy [2]. Regulatory Designations - RGX-121 has received multiple designations from the FDA, including Orphan Drug Product, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) [2]. Commercialization Strategy - Following potential FDA approval, RGX-121 will be commercialized by NS Pharma, a subsidiary of Nippon Shinyaku, while REGENXBIO retains all rights and proceeds related to the potential sale of a Priority Review Voucher (PRV) [3].

Group 1 - Regenxbio reported quarterly earnings of $0.12 per share, missing the Zacks Consensus Estimate of $0.41 per share, compared to a loss of $1.38 per share a year ago, representing an earnings surprise of -70.73% [1] - The company posted revenues of $89.01 million for the quarter ended March 2025, missing the Zacks Consensus Estimate by 19.07%, compared to year-ago revenues of $15.62 million [2] - Over the last four quarters, Regenxbio has surpassed consensus EPS estimates two times and topped consensus revenue estimates two times [2] Group 2 - The stock's immediate price movement will depend on management's commentary on the earnings call and the sustainability of earnings expectations [3][4] - Regenxbio shares have lost about 2.7% since the beginning of the year, while the S&P 500 has declined by 3.8% [3] - The current consensus EPS estimate for the coming quarter is -$1.21 on $21.98 million in revenues, and -$0.88 on $295.17 million in revenues for the current fiscal year [7] Group 3 - The Medical - Biomedical and Genetics industry, to which Regenxbio belongs, is currently in the top 34% of the Zacks industries, indicating a favorable outlook [8] - Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors [5][6]

Financial Data and Key Metrics Changes - REGENXBIO ended the quarter on March 31, 2025, with cash, cash equivalents, and marketable securities of $272 million, an increase from $245 million as of December 31, 2024, primarily driven by a $110 million upfront payment from the Nippon Shinyaku collaboration [24] - R&D expenses were $53 million for the quarter ended March 31, 2025, compared to $54.8 million for the same quarter in 2024, reflecting a decrease due to clinical trial expenses for RGX-314 and RGX-202 [25] Business Line Data and Key Metrics Changes - RGX-121, a potential first gene therapy for MPS II, is on track for potential FDA approval in the second half of 2025, with a BLA submitted under the accelerated approval pathway [8][9] - RGX-202, a next-generation candidate for Duchenne muscular dystrophy (DMD), has surpassed 50% enrollment in its pivotal study and is expected to submit a BLA in mid-2026 [10][11] - The retinal program, RGX-314, is advancing in two pivotal studies for wet AMD and is on track to be the first gene therapy on the market for this condition [12][19] Market Data and Key Metrics Changes - The DMD market is projected to have over half of the prevalent population untreated by 2027, highlighting a significant opportunity for RGX-202 [11] - The wet AMD and diabetic retinopathy markets represent large multibillion-dollar commercial opportunities, with RGX-314 positioned to serve as a meaningful alternative to current treatments [12][19] Company Strategy and Development Direction - The company is focused on transitioning to a commercial stage with in-house manufacturing capabilities and plans to secure non-dilutive funding [6][8] - A strategic partnership with Nippon Shinyaku aims to commercialize the neurodegenerative franchise, including RGX-121 and RGX-111 [9] - The company is preparing for commercial supply manufacturing of RGX-202 in anticipation of a 2027 launch [11][27] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the imminent acceptance of the BLA for RGX-121 and the positive trajectory of clinical programs [34][51] - The company is optimistic about the evolving regulatory landscape and the potential for accelerated approval pathways, particularly in light of recent developments in the industry [36][44] Other Important Information - The company has a robust cash runway expected to fund operations into the second half of 2026, with potential extensions through non-dilutive financing options [25][26] - The manufacturing innovation center in Rockville, Maryland, is capable of producing up to 2,500 doses of RGX-202 annually, ensuring readiness for market needs [11][12] Q&A Session Summary Question: Timing for the Hunter BLA - Management indicated that the BLA acceptance is imminent and they feel confident about the review process [34] Question: Competitor Insights and Regulatory Expectations - Management discussed the evolving expectations for accelerated approval in DMD, emphasizing the strength of their safety and functional data [36][44] Question: Changes in FDA Requirements - Management noted that there are no anticipated changes in the accelerated approval pathway and enrollment is on track [43][44] Question: Planning for Diabetic Retinopathy Phase III Trial - Management confirmed ongoing interactions with the FDA and that they are on track for first patient dosing this year [55] Question: Updates on DMD Functional Data - Management plans to release additional functional data in the first half of the year, focusing on dose level two patients [62][88] Question: Impact of Recent Pricing Announcements - Management stated it is too early to assess the impact of recent pricing discussions on their gene therapies [104]

Regenxbio (RGNX) Q1 2025 Earnings Call May 12, 2025 04:30 PM ET Speaker0 Welcome everyone to the First Quarter twenty twenty five REGENXBIO Earnings Conference Call. At this time, all participants are in a listen only mode. After the speakers' prepared remarks, we will conduct a question and answer session. As a reminder, this call may be recorded. At this time, I'd like to turn the conference over to Patrick Christmas, Chief Legal Officer of REGENXBIO. Please go ahead. Speaker1 Good afternoon, and thank yo ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___ to ___ Commission File Number 001-37553 REGENXBIO Inc. (Exact Name of Registrant as Specified in its Charter) Delaware 47-1851754 (State ...