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].

Regenxbio (RGNX) came out with quarterly earnings of $0.12 per share, missing the Zacks Consensus Estimate of $0.41 per share. This compares to loss of $1.38 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -70.73%. A quarter ago, it was expected that this biotechnology company would post a loss of $1.27 per share when it actually produced a loss of $1.01, delivering a surprise of 20.47%.Over the last four quarters, the company ...

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 ...

Financial Performance - Revenues for Q1 2025 were $89.0 million, a significant increase from $15.6 million in Q1 2024, primarily due to $71.8 million of license and service revenue from the collaboration with Nippon Shinyaku[13] - Net income for Q1 2025 was $6.1 million, or $0.12 per share, compared to a net loss of $63.3 million, or $1.38 per share, in Q1 2024[17] - Total revenues for Q1 2025 reached $89,012,000, a significant increase of 469% compared to $15,622,000 in Q1 2024[26] - License and royalty revenue surged to $87,049,000, up from $15,344,000 in the same period last year, marking a growth of 467%[26] - Basic net income per share improved to $0.12 in Q1 2025, compared to a loss of $1.38 per share in Q1 2024[26] - The company reported a comprehensive income of $6,062,000 for Q1 2025, compared to a comprehensive loss of $62,130,000 in Q1 2024[26] Cash and Assets - Cash, cash equivalents, and marketable securities totaled $272.7 million as of March 31, 2025, up from $244.9 million at the end of 2024, mainly due to a $110 million upfront payment from the Nippon Shinyaku partnership[12] - Total current assets increased to $313,237,000 as of March 31, 2025, compared to $278,001,000 at the end of 2024, reflecting a growth of 12.7%[25] - Total assets rose to $490,929,000, up from $465,989,000 at the end of 2024, indicating a growth of 5.3%[25] - Total liabilities increased to $216,732,000 from $206,338,000, representing a rise of 5.7%[25] Expenses - Research and development expenses for Q1 2025 were $53.1 million, a decrease from $54.8 million in Q1 2024, primarily due to lower clinical trial expenses[15] - General and administrative expenses increased to $20.3 million in Q1 2025 from $18.3 million in Q1 2024, mainly due to higher personnel-related costs[16] - Total operating expenses decreased to $76,885,000 in Q1 2025 from $79,485,000 in Q1 2024, a reduction of 3.9%[26] Clinical Development - The pivotal trial for RGX-202 in Duchenne muscular dystrophy is more than half enrolled, with completion expected in 2025 and a Biologics License Application (BLA) submission planned for mid-2026[5] - REGENXBIO expects to initiate commercial supply manufacturing for RGX-202 in Q3 2025, with all production sourced in the U.S.[6] - The FDA is expected to accept the BLA for clemidsogene lanparvovec (RGX-121) in May 2025, with potential approval in the second half of 2025[14] Partnerships - The partnership with Nippon Shinyaku includes an upfront payment of $110 million and potential additional payments of up to $700 million upon achieving certain milestones[11] - REGENXBIO's cash runway is expected to fund operations into the second half of 2026, excluding potential milestone payments and monetization of a Priority Review Voucher[18]

Core Insights - REGENXBIO Inc. reported significant advancements in its gene therapy pipeline, with multiple late-stage assets showing differentiation from standard treatments, positioning the company for potential first- or best-in-class therapies for rare and retinal diseases [2] Program Highlights and Milestones - RGX-202 is a novel microdystrophin gene therapy for Duchenne muscular dystrophy, utilizing the NAV® AAV8 vector, and is the only construct including the C-Terminal domain found in natural dystrophin [3] - Clemidsogene lanparvovec (RGX-121) is being developed as a first-in-class treatment for MPS II (Hunter syndrome) in partnership with Nippon Shinyaku [4] - Surabgene lomparvovec (sura-vec, ABBV-RGX-314) is on track to be the first-in-class treatment for wet age-related macular degeneration (wet AMD), with pivotal trial enrollment ongoing and completion expected in 2025 [5][8] Financial Results - As of March 31, 2025, REGENXBIO's cash, cash equivalents, and marketable securities totaled $272.7 million, an increase from $244.9 million at the end of 2024, primarily due to a $110 million upfront payment from the Nippon Shinyaku partnership [11] - Revenues for the first quarter of 2025 were $89.0 million, a significant increase from $15.6 million in the same period of 2024, largely driven by $71.8 million in license and service revenue from the collaboration with Nippon Shinyaku [12] - Research and development expenses decreased to $53.1 million in Q1 2025 from $54.8 million in Q1 2024, while general and administrative expenses rose to $20.3 million from $18.3 million [13][14] - The company reported a net income of $6.1 million, or $0.12 per share, compared to a net loss of $63.3 million, or $1.38 per share, in the prior year [15] Corporate Updates - REGENXBIO's partnership with Nippon Shinyaku, finalized in March 2025, includes an upfront payment of $110 million and potential additional payments of up to $700 million based on milestone achievements [10] - The company is preparing for a Biologics License Application (BLA) submission for clemidsogene lanparvovec (RGX-121) expected in May 2025, with potential approval in the second half of 2025 [6]

Core Insights - REGENXBIO Inc. is showcasing its advancements in gene therapy at the ASGCT 28th Annual Meeting, emphasizing its comprehensive capabilities in research, clinical development, and manufacturing [1][5] Presentations Overview - The company will present late-stage clinical trial data for RGX-121, aimed at treating MPS II, and RGX-202 for Duchenne muscular dystrophy, along with preclinical research on RGX-202's novel construct and manufacturing process [2][5] - Key oral presentations include: - Development of a commercial manufacturing process for RGX-202 [3] - Investigational gene therapy RGX-121 for neuronopathic MPS II [3] - Interim clinical data for RGX-202 [3] Poster Presentations - Various poster presentations will cover topics such as: - The impact of sample collection conditions on AAV endotoxin testing [4] - AAV-expressed microdystrophin's effects in a mouse model of Duchenne muscular dystrophy [4] - Development of in vitro methods for analyzing TLR9 stimulation by AAV vector genomes [4] - Characterization of oversized AAV vectors with high genome integrity [4] - Blood-brain barrier crossing AAV vectors targeting the transferrin receptor [4] Company Background - REGENXBIO, founded in 2009, focuses on gene therapy, particularly AAV gene therapy, with a late-stage pipeline targeting rare and retinal diseases [5] - The company has treated thousands of patients using its AAV platform, including those receiving Novartis' ZOLGENSMA® [5]

Core Viewpoint - Regenxbio (RGNX) is anticipated to report a significant year-over-year increase in earnings and revenues for the quarter ended March 2025, which could influence its stock price depending on the actual results compared to estimates [1][2]. Earnings Expectations - The upcoming earnings report is expected to be released on May 12, 2025, with a consensus estimate of $0.41 per share, reflecting a year-over-year increase of +129.7% [3]. - Revenues are projected to reach $109.98 million, representing a substantial increase of 604.1% from the same quarter last year [3]. Estimate Revisions - Over the last 30 days, the consensus EPS estimate has been revised down by 6.26%, indicating a reassessment by analysts [4]. - The Most Accurate Estimate for Regenxbio is higher than the Zacks Consensus Estimate, resulting in a positive Earnings ESP of +57.77% [10][11]. Earnings Surprise Prediction - The Zacks Earnings ESP model suggests that a positive Earnings ESP reading is a strong predictor of an earnings beat, especially when combined with a Zacks Rank of 1 (Strong Buy), 2 (Buy), or 3 (Hold) [8]. - Regenxbio currently holds a Zacks Rank of 3, indicating a likelihood of beating the consensus EPS estimate [11]. Historical Performance - In the last reported quarter, Regenxbio was expected to post a loss of $1.27 per share but actually reported a loss of $1.01, resulting in a surprise of +20.47% [12]. - Over the past four quarters, the company has surpassed consensus EPS estimates two times [13]. Industry Context - In the Zacks Medical - Biomedical and Genetics industry, Sangamo Therapeutics (SGMO) is expected to report a loss of $0.11 per share for the same quarter, with a year-over-year change of +59.3% [17]. - Sangamo's revenue is projected to be $9.17 million, up 1810.4% from the previous year [17]. - Despite a recent downward revision of 23.8% in consensus EPS estimates, Sangamo has an Earnings ESP of 27.27%, but it holds a Zacks Rank of 4 (Sell), making predictions of an earnings beat uncertain [18].

Company Overview - REGENXBIO Inc. is a biotechnology company focused on gene therapy with a mission to improve lives through its curative potential [3] - The company has been a pioneer in AAV gene therapy since its founding in 2009 [3] - REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for Duchenne, RGX-121 for MPS II, and RGX-111 for MPS I, in partnership with Nippon Shinyaku [3] - The company is also collaborating with AbbVie on surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy [3] - Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® [3] Upcoming Events - REGENXBIO will host a conference call on May 12, 2025, at 4:30 p.m. ET to discuss its financial results for Q1 2025 and recent operational highlights [1] - Listeners can register for the webcast and analysts can participate in the Q&A session through provided links [2] - A replay of the webcast will be available on the company's investor website approximately two hours after the call [2]