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]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2024 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) (State or other jurisdiction of incorporation or organiz ...

Financial Performance - Revenues for Q4 2024 were $21.2 million, a decrease of 4.5% from $22.2 million in Q4 2023; full-year revenues were $83.3 million, down 7.7% from $90.2 million in 2023[13]. - Net loss for Q4 2024 was $51.2 million, or $1.01 per share, compared to a net loss of $62.9 million, or $1.43 per share, in Q4 2023; full-year net loss was $227.1 million, or $4.59 per share, down from $263.5 million, or $6.02 per share, in 2023[16]. - Total revenues for the year ended December 31, 2024, were $83,328,000, a decrease of 7.5% compared to $90,242,000 for the year ended December 31, 2023[25]. - Net loss for the year ended December 31, 2024, was $227,102,000, compared to a net loss of $263,494,000 for the year ended December 31, 2023, representing a 13.8% improvement[25]. - The company reported a comprehensive loss of $223,414,000 for the year ended December 31, 2024, compared to a comprehensive loss of $252,522,000 for the year ended December 31, 2023, indicating a 11.5% improvement[25]. - The company reported a loss from operations of $233,346,000 for the year ended December 31, 2024, compared to a loss of $268,128,000 for the year ended December 31, 2023, reflecting a 13.0% improvement[25]. Cash and Assets - Cash, cash equivalents, and marketable securities totaled $244.9 million as of December 31, 2024, down from $314.1 million as of December 31, 2023, primarily due to cash used for operating activities[11]. - Cash and cash equivalents increased to $57,526,000 as of December 31, 2024, compared to $34,522,000 as of December 31, 2023, an increase of 66.6%[24]. - Total assets decreased to $465,989,000 as of December 31, 2024, from $573,970,000 as of December 31, 2023, a decline of 18.8%[24]. - Total liabilities decreased to $206,338,000 as of December 31, 2024, down from $262,228,000 as of December 31, 2023, a reduction of 21.4%[24]. Research and Development - Research and development expenses for Q4 2024 were $50.4 million, down from $55.7 million in Q4 2023; full-year expenses were $208.5 million, down from $232.3 million in 2023[14]. - Research and development expenses for the year ended December 31, 2024, were $208,522,000, down 10.2% from $232,266,000 in 2023[25]. - The pivotal trial for RGX-202 is nearly 50% enrolled, with completion expected in 2025 and a BLA filing anticipated in mid-2026[7]. - The pivotal data for the retinal franchise, including ABBV-RGX-314, is expected in 2026, with ongoing planning for diabetic retinopathy studies[6]. Strategic Partnerships - The strategic partnership with Nippon Shinyaku includes an upfront payment of $110 million and up to $700 million in milestone payments for RGX-121 and RGX-111[12]. - REGENXBIO submitted a BLA for clemidsogene lanparvovec (RGX-121) in March 2025, with potential FDA approval expected in the second half of 2025[6]. - REGENXBIO expects its cash runway to extend into the second half of 2026, supported by the $110 million upfront payment from Nippon Shinyaku[17]. Operating Expenses - General and administrative expenses for Q4 2024 were $20.1 million, an increase from $19.1 million in Q4 2023; full-year expenses were $76.6 million, down from $88.5 million in 2023[15]. - Operating expenses for the year ended December 31, 2024, totaled $316,674,000, a decrease of 11.6% from $358,370,000 in 2023[25]. - The weighted-average common shares outstanding increased to 49,509,000 for the year ended December 31, 2024, from 43,734,000 in 2023, an increase of 13.2%[25].

Biologics Licensing Application (BLA) for clemidsogene lanparvovec (RGX-121) submitted and on track for potential FDA approval 2H 2025; strategic partnership with Nippon Shinyaku aims to expand potential access and commercial opportunity in MPS II and MPS I Pivotal trial of RGX-202 for Duchenne Muscular Dystrophy progressing rapidly; enrollment completion expected in 2025 with BLA filing in mid-2026 AbbVie-partnered retinal franchise continues advancing; pivotal data evaluating the safety and efficacy of ...

Group 1 - REGENXBIO Inc. announced new interim biomarker data from the Phase I/II portion of the AFFINITY DUCHENNE® trial for RGX-202, aimed at treating Duchenne muscular dystrophy, to be presented at the 2025 MDA Clinical & Scientific Conference [1] - The presentation will include interim clinical data on RGX-202 and a discussion on the enhanced therapeutic potential of a microdystrophin with an extended C-terminal domain [2] - REGENXBIO is a leading clinical-stage biotechnology company focused on gene therapy, with a pipeline that includes treatments for rare and retinal diseases [3] Group 2 - The company has developed AAV Therapeutics, an innovative class of gene therapy medicines, and has treated thousands of patients with its platform [3] - Notable products in development include RGX-202 for Duchenne muscular dystrophy, ABBV-RGX-314 for wet AMD and diabetic retinopathy in collaboration with AbbVie, and RGX-121 for MPS II [3] - AAV Therapeutics are designed as one-time treatments, potentially transforming healthcare delivery for millions [3]

Company Overview - Regenxbio (RGNX) is expected to report a year-over-year increase in earnings driven by higher revenues for the quarter ended December 2024, with a consensus estimate of a quarterly loss of $1.27 per share, reflecting an 11.2% improvement from the previous year [1][3] - Revenues are projected to reach $22.9 million, which is a 3.1% increase compared to the same quarter last year [3] Earnings Estimates and Revisions - The consensus EPS estimate has been revised 20.37% higher in the last 30 days, indicating a reassessment by analysts regarding the company's earnings prospects [4] - The Most Accurate Estimate for Regenxbio is lower than the Zacks Consensus Estimate, resulting in an Earnings ESP of -1.58%, suggesting a bearish outlook from analysts [10][11] Earnings Surprise Prediction - The Zacks Earnings ESP model indicates that a positive or negative reading can predict the likelihood of actual earnings deviating from consensus estimates, with a positive ESP being a strong predictor of an earnings beat [6][8] - Regenxbio's combination of a negative Earnings ESP and a Zacks Rank of 2 (Buy) complicates the prediction of an earnings beat [11] Historical Performance - In the last reported quarter, Regenxbio was expected to post a loss of $1.16 per share but actually reported a loss of $1.17, resulting in a surprise of -0.86% [12] - Over the past four quarters, the company has only beaten consensus EPS estimates once [13] Industry Context - Another player in the biomedical sector, scPharmaceuticals, is expected to report an EPS of $0.40 for the same quarter, which represents a year-over-year decline of 14.3%, with revenues expected to be $12.08 million, up 98% from the previous year [17] - The consensus EPS estimate for scPharmaceuticals has been revised 26.3% lower in the last 30 days, but a higher Most Accurate Estimate has resulted in an Earnings ESP of 2.50%, despite a Zacks Rank of 4 (Sell) [18]

ROCKVILLE, Md., March 4, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the closing of its previously announced strategic partnership with Nippon Shinyaku. Under the terms of the agreement, REGENXBIO and Nippon Shinyaku will develop and commercialize RGX-121 (clemidsogene lanparvovec) for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome in the United States and Asia. "RGX-121 and RGX-111 repres ...

ROCKVILLE, Md., Feb. 26, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Thursday, March 13, at 4:30 p.m. ET to discuss its financial results for the fourth quarter and full year ended December 31, 2024, and recent operational highlights.Listeners can register for the webcast via this link. Analysts wishing to participate in the question and answer session should use this link. A replay of the webcast will be available via the company's investor webs ...