Regenxbio (RGNX) came out with a quarterly loss of $1.38 per share versus the Zacks Consensus Estimate of a loss of $1.13. This compares to a loss of $1.05 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -22.12%. A quarter ago, it was expected that this biotechnology company would post earnings of $0.41 per share when it actually produced earnings of $0.12, delivering a surprise of -70.73%.Over the last four quarters, the comp ...

Regenxbio (RGNX) Q2 2025 Earnings Call August 07, 2025 08:00 AM ET Speaker0Welcome everyone to the Second Quarter twenty twenty five BGENXBIO Earnings Conference Call. Please note, slide referenced during this call are available in the webcast and at the events page of Regenexx Bio's website. At this time, I'd like to turn the conference over to Patrick Christmas, Chief Legal Officer of Regenexx Bio. Please go ahead.Speaker1Good morning and thank you for joining us today. Earlier this morning, REGENXBIO rel ...

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 June 30, 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 ...

EXHIBIT 99.1 REGENXBIO Reports Second Quarter 2025 Financial Results and Operational Highlights ROCKVILLE, Md., August 7, 2025 (PR Newswire) -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and operational highlights for the second quarter ended June 30, 2025. "Our REGENXBIO team is accelerating multiple first or best-in-class gene therapies designed to improve the lives of patients and families facing serious diseases like Duchenne, Hunter syndrome, and retinal diseases. We continued our s ...

Core Insights - REGENXBIO Inc. reported strong operational momentum in advancing its gene therapy programs, particularly with RGX-121 and RGX-202, aimed at treating serious diseases such as Duchenne muscular dystrophy and Hunter syndrome [2][4][5] Financial Results - Cash, cash equivalents, and marketable securities increased to $363.6 million as of June 30, 2025, from $244.9 million at the end of 2024, primarily due to a $110 million upfront payment from Nippon Shinyaku and $144.5 million from royalty monetization [16][21] - Revenues for the second quarter of 2025 were $21.4 million, a slight decrease from $22.3 million in the same period of 2024, mainly due to a drop in Zolgensma royalties [17] - Research and development expenses rose to $59.5 million in Q2 2025 from $48.9 million in Q2 2024, driven by manufacturing and clinical trial costs [18] - General and administrative expenses increased to $19.9 million in Q2 2025 from $18.9 million in Q2 2024, attributed to personnel and consulting costs [19] - The net loss for Q2 2025 was $70.9 million, compared to a net loss of $53.0 million in Q2 2024, reflecting increased operational costs [20] Program Highlights - RGX-202 is positioned as a potential best-in-class gene therapy for Duchenne muscular dystrophy, with pivotal trial enrollment expected to complete by October 2025 [3][5] - Clemidsogene lanparvovec (RGX-121) is on track for potential FDA approval in November 2025, with successful completion of FDA inspections [4][13] - Surabgene lomparvovec (sura-vec) is advancing towards pivotal trials for diabetic retinopathy and wet AMD, with topline results expected in 2026 [7][9] Corporate Updates - In May 2025, REGENXBIO closed a $250 million royalty bond agreement, receiving $150 million upfront [11] - An amendment to the collaboration agreement with AbbVie was executed in August 2025, which includes milestone payments for the diabetic retinopathy program [14] Financial Guidance - The company expects its cash position to fund operations into early 2027, excluding potential payments from partners or monetization of a Priority Review Voucher [21]

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 August 7, 2025, at 8:00 a.m. ET to discuss its financial results for Q2 2025 and operational highlights [1] - Listeners can register for the webcast and analysts can participate in the Q&A session by dialing specific numbers [2] - A replay of the webcast will be available on the company's investor website approximately two hours after the call [2]

Core Insights - REGENXBIO Inc. announced preclinical results showing that a microdystrophin gene therapy construct with the C-terminal (CT) domain provides improved functional benefits for patients with Duchenne Muscular Dystrophy compared to a construct without the CT domain [1][4][5] - RGX-202 is the only investigational microdystrophin gene therapy candidate that includes the CT domain, making it closest to naturally occurring dystrophin [2][8] Group 1: Research Findings - The preclinical study published in Molecular Therapy Methods and Clinical Development demonstrated that the microdystrophin with the CT domain was maintained at higher levels in transduced muscles and effectively recruited the dystrophin-associated protein complex to the muscle membrane [4][5] - The incorporation of the CT domain enhances the microdystrophin design, allowing for higher accumulation levels in muscle and potentially improving functional benefits [5][7] Group 2: Clinical Trial Insights - Interim results from the Phase I/II AFFINITY DUCHENNE trial indicated that RGX-202 showed consistent evidence of positively changing the disease trajectory in patients with Duchenne and had a favorable safety profile [5][6] - REGENXBIO is currently enrolling participants in the pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial and plans to submit a Biologics License Application (BLA) via the accelerated approval pathway in mid-2026 [6][9] Group 3: Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, with a late-stage pipeline that includes RGX-202 for Duchenne, among other treatments for rare diseases [11] - The company has pioneered AAV gene therapy since its founding in 2009 and has treated thousands of patients with its AAV platform [11]

Company Overview - Regenxbio is a clinical-stage biopharmaceutical company that specializes in gene therapies, founded in 2008 [1] - The company's core technology involves the delivery of rescue genes using AAV (adeno-associated virus) payloads, which is increasingly being utilized in approved drugs [1] Industry Insights - The healthcare sector is complex, and financial professionals and investors require scientific and clinical expertise to navigate it effectively [1] - There is a growing emphasis on bridging the gap between advanced scientific research and financial strategy to uncover hidden value and assess risks accurately in life sciences [1]

Core Insights - RegenXBio Inc. has released new interim data from the Phase 1/2 AFFINITY DUCHENNE trial, showcasing positive functional, safety, and biomarker data for RGX-202, a gene therapy for Duchenne muscular dystrophy [1][2]. Group 1: Trial Data and Results - The trial involved five patients aged six to twelve years who received RGX-202, demonstrating consistent benefits in functional data among dose level 2 participants at 9 and 12 months post-treatment [1]. - At 9 months, RGX-202 participants showed improved function, exceeding external controls on all measures, with an average improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from baseline and 4.8 points compared to natural history [3]. - At 12 months, RGX-202 participants continued to demonstrate improved performance on timed function tests and NSAA, with an average improvement of 4.5 points from baseline and 6.8 points compared to natural history [4]. Group 2: Future Plans and Market Context - RegenXBio plans to share topline data for RGX-202 in the first half of 2026 and aims to submit a Biologics License Application (BLA) under the accelerated approval pathway by mid-2026, with commercial readiness activities underway for a potential launch in 2027 [5]. - The competitive landscape includes Sarepta Therapeutics, which recently reported a patient death following treatment with its approved gene therapy Elevidys, leading to a therapeutic clinical hold on several studies in the EU [6].

RGX-202 Therapeutic Approach - RGX-202 is designed to improve function and preserve muscle health, delivered by proprietary NAV® AAV8 vector[24] - The trial is designed to demonstrate meaningful change in disease trajectory in pursuit of broad label, with positive interim efficacy and safety outcomes reported in Phase I/II[25] - RGX-202 has product purity levels of more than 80% full capsids due to industry-leading manufacturing[21] - A proactive, short-course immune modulation regimen is designed to counter safety concerns common with high-dose AAV gene therapy and to improve safety outcomes[22] AFFINITY DUCHENNE Phase I/II Trial Results - RGX-202 was well-tolerated in 13 patients across both dose levels with no SAEs or AESIs[62] - Biomarker data support consistent robust expression, transduction, and sarcolemmal localization of RGX-202 microdystrophin[62] - At 9 months, Dose Level 2 participants (n=5) demonstrated a mean change in NSAA of 4.8 compared to natural history external controls (n=65)[44] - At 12 months, Dose Level 2 participants (n=4) demonstrated a mean change in NSAA of 6.8 compared to natural history external controls (n=26)[47] - Dose Level 2 timed task velocity changes exceeded MCID benchmarks at 12 months[50] - Caregivers reported improved function in the home and community as measured by key dimensions of the PODCI at 12 months compared to expected decline in natural history[54] Future Development - The company plans for a mid-2026 BLA submission using accelerated approval pathway and a potential FDA approval in 1H 2027[18]