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]

Financial Data and Key Metrics Changes - REGENXBIO ended Q4 2024 with cash, cash equivalents, and marketable securities of $245 million, down from $314 million as of December 31, 2023, primarily due to cash used for operating activities [38] - R&D expenses were $209 million for the year ended December 31, 2024, compared to $232 million in 2023, reflecting a decrease attributed to reduced headcount and preclinical activities [39] Business Line Data and Key Metrics Changes - The company submitted its first Biologics License Application (BLA) for RGX-121, a treatment for Hunter syndrome, under the accelerated approval pathway, with potential FDA approval expected in Q4 2025 [8][36] - The pivotal study for RGX-202, targeting Duchenne muscular dystrophy, is advancing rapidly, with nearly half of the enrollment completed and expected to submit a BLA by mid-2026 [15][17] - The diabetic retinopathy program is preparing for a pivotal study following a successful end of Phase 2 meeting with the FDA [20] Market Data and Key Metrics Changes - The company anticipates that more than half of the prevalent population for Duchenne muscular dystrophy will remain untreated in the coming years, indicating a significant market opportunity [16] - The partnership with AbbVie for RGX-314 is expected to yield additional milestone payments, enhancing the company's financial position [21] Company Strategy and Development Direction - REGENXBIO aims to launch multiple first or best-in-class gene therapies, focusing on sustainable profitability and leveraging over fifteen years of gene therapy leadership [9] - The company is strategically positioned to deliver on multiple late-stage opportunities, with a focus on differentiation against standard care treatments [45] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential approval of RGX-121 and the progress of RGX-202, highlighting strong momentum across the pipeline [42] - The company is optimistic about its partnerships and the potential for non-dilutive financing options to extend its cash runway beyond 2026 [40] Other Important Information - The company has a robust manufacturing capability, producing 2,500 doses of RGX-202 per year with industry-leading purity levels [17] - The BLA submission for RGX-121 is based on significant clinical data, including a high percentage of patients discontinuing enzyme replacement therapy [36] Q&A Session Summary Question: Can you delve deeper into the components of non-dilutive financing? - Management outlined three components: expected milestone payments from AbbVie, potential monetization of a priority review voucher upon regulatory approval, and the reversion of Zolgensma royalty income once a cap is reached [50][53] Question: What are the expectations around potentially going to an advisory committee for RGX-202? - Management indicated that while they do not foresee a significant issue that would necessitate an advisory committee, they are prepared for such a scenario if it arises [60] Question: What is the current pace of enrollment for the DMD pivotal trial? - Management reported encouraging enrollment and expects acceleration as new sites are activated, aiming to complete enrollment this year [76][78] Question: Are cardiac endpoints being measured in the DMD study? - Management confirmed that cardiac function will be monitored, including ejection fraction and troponin levels, but noted that significant changes may not be observable in younger patients [90][91] Question: Are there any key differences between US and OUS regulatory pathways for diabetic retinopathy? - Management highlighted that while the US pathway is clear, there are ongoing discussions with EMA and Japanese regulators to establish a solid case for the diabetic retinopathy severity scale [112][115]

Group 1 - Regenxbio reported a quarterly loss of $1.01 per share, better than the Zacks Consensus Estimate of a loss of $1.27, and an improvement from a loss of $1.43 per share a year ago, resulting in an earnings surprise of 20.47% [1] - The company posted revenues of $21.21 million for the quarter ended December 2024, missing the Zacks Consensus Estimate by 7.36%, and down from $22.21 million in the same quarter last year [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 has underperformed the market, losing about 13.7% since the beginning of the year compared to the S&P 500's decline of 4.8% [3] - The company's earnings outlook, including current consensus earnings expectations for upcoming quarters, will be crucial for investors [4] - The trend for estimate revisions for Regenxbio is currently favorable, leading to a Zacks Rank 2 (Buy) for the stock, indicating expected outperformance in the near future [6] Group 3 - The current consensus EPS estimate for the upcoming quarter is -$1.08 on revenues of $20.36 million, and for the current fiscal year, it is -$1.52 on revenues of $274.95 million [7] - The Medical - Biomedical and Genetics industry, to which Regenxbio belongs, is currently in the top 26% of Zacks industries, suggesting a positive outlook for the sector [8]

Financial Data and Key Metrics Changes - REGENXBIO ended the quarter on December 31, 2024, with cash, cash equivalents, and marketable securities of $245 million, down from $314 million as of December 31, 2023, primarily due to cash used for operating activities [26] - R&D expenses were $209 million for the year ended December 31, 2024, compared to $232 million in 2023, attributed to decreases in headcount and preclinical activities [26][27] - The company expects the cash runway to fund operations into the second half of 2026, with potential extensions through non-dilutive financing options [27] Business Line Data and Key Metrics Changes - The pivotal study for RGX-202 is advancing rapidly, with nearly half of the enrollment completed, and the company expects to submit a BLA under the accelerated approval pathway by mid-2026 [12][29] - The BLA for RGX-121 has been submitted, with potential FDA approval anticipated in the second half of 2025 [7][24] - The diabetic retinopathy program is moving towards a pivotal program with AbbVie, with successful end-of-Phase II meetings held with the FDA [14][20] Market Data and Key Metrics Changes - The partnership with Nippon Shinyaku for MPS programs is strategically significant, combining REGENXBIO's development expertise with Nippon's commercialization experience [9][10] - The company is preparing for potential approval of RGX-121 for MPS II in Q4 2025, which could enhance its market position [10][24] Company Strategy and Development Direction - REGENXBIO aims to launch multiple first or best-in-class gene therapies, focusing on sustainable profitability and leveraging over 15 years of gene therapy leadership [8][29] - The company is positioned to deliver on multiple late-stage opportunities, with a focus on differentiation against standard care and available treatments [29][31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong momentum across the pipeline, with key milestones approaching [29] - The company believes it is well-positioned to deliver transformative treatments for patients in need of new options, particularly in the Duchenne muscular dystrophy and retinal disease markets [29][31] Other Important Information - The company has a robust commercial capability and global partnerships, which are expected to support the launch of its gene therapies [7][10] - REGENXBIO is actively working on non-dilutive financing options, including milestone payments and potential monetization of a priority review voucher [27] Q&A Session Summary Question: Can you delve deeper into the components of non-dilutive financing? - Management outlined three components: expected milestone payments from the diabetic retinopathy program, potential monetization of a priority review voucher upon regulatory approval, and the reversion of royalty income from Zolgensma once a cap is reached [36][39][40] Question: What are the expectations around potentially going to an advisory committee for RGX-202? - Management indicated that while they do not foresee a significant issue that would necessitate an advisory committee, they are prepared for it if required [46][47] Question: Can you provide an update on the NDA and patient data for the one to three-year-old cohort? - Management plans to update on one patient from the one to four cohort at the upcoming MDA meeting, with expectations of higher microdystrophin levels in younger patients [51][53] Question: What is the current pace of enrollment for the DMD pivotal trial? - Management reported encouraging enrollment and expects acceleration as more sites are activated, with a significant number of patients showing interest [58][59] Question: Are cardiac endpoints being measured in the DMD study? - Management confirmed that they are measuring cardiac endpoints, including ejection fraction and troponin levels, but noted that significant changes may not be observable in younger patients [67][72] Question: Will enrollment for the wet AMD study be cut short to accelerate timelines? - Management stated that they will not cut enrollment short, as they aim to fully characterize safety and efficacy in collaboration with AbbVie [75][76]