Regenxbio (RGNX) Q1 2025 Earnings Call May 12, 2025 04:30 PM ET Company Participants Patrick Christmas II - Chief Legal OfficerCurran Simpson - Board Member, President & CEOSteve Pakola - Chief Medical OfficerMitchell Chan - CFOMani Foroohar - Senior Managing DirectorGena Wang - MD - Biotech Equity ResearchTejas Wein - Associate Director - Biotechnology Equity ResearchAnnabel Samimy - Managing DirectorShelby Hill - Biotech Equity Research AssociateSean McCutcheon - Vice President - Biotechnology Equity Rese ...

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]

Group 1 - The Fortune Teller is a team of two analysts with over 30 years of combined market experience [1] - One of the principles has a background in asset management and is a formerly licensed investment advisor [1] - The team holds degrees in Accounting & Economics and Computer Sciences, as well as MBAs, which inform their stock selections [1] Group 2 - The team leads the investing group Wheel of Fortune, sharing actionable trading ideas across various asset classes, sectors, and industries [1] - The service aims to provide a comprehensive resource for investment and portfolio ideas while educating subscribers [1] - Features include the Funds Macro Portfolio for less active investors, the Single Macro Portfolio for more active investors, educational content, and a live chat room for discussions [1]

Core Insights - REGENXBIO Inc. reported positive interim data from the Phase I/II AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, presented at the 2025 MDA Clinical & Scientific Conference [1][2] Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, with RGX-202 being the only next-generation gene therapy in pivotal trials for Duchenne muscular dystrophy [2][9] - The company aims to improve lives through the curative potential of gene therapy and has a late-stage pipeline for various rare diseases [12] Clinical Trial Updates - The AFFINITY DUCHENNE trial is progressing towards completing enrollment in 2025, with a Biologics License Application (BLA) submission anticipated by mid-2026 [2][7] - New biomarker data from patients aged 1-3 showed microdystrophin expression levels of 122.3% compared to control in a 3-year-old patient, indicating robust expression across all ages [4][7] - RGX-202 demonstrated the highest reported vector genome copies (4.9-55.4) among approved or investigational gene therapies [6] Safety and Efficacy - As of February 21, 2025, RGX-202 was well tolerated with no serious adverse events (SAEs) reported, and common drug-related adverse events included nausea, vomiting, and fatigue [7][8] - The treatment has shown functional improvements exceeding external natural history controls, with patients demonstrating stable or improved function on the North Star Ambulatory Assessment (NSAA) [8] Future Prospects - The company plans to share additional Phase I/II functional data in the first half of 2025, reinforcing the potential of RGX-202 to alter the course of Duchenne muscular dystrophy [2][8]