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 ...

Core Viewpoint - Goldman Sachs downgraded Regenxbio Inc (RGNX) to Neutral from Buy, citing uncertainty in the appeal of gene therapy for wet AMD and diabetic retinopathy due to competition and cost-effectiveness of newer treatments [1][2]. Group 1: Market Competition and Treatment Efficacy - The analyst notes that AbbVie Inc's ABBV-RGX-314 faces increasing competition, particularly due to its invasive delivery method, which is less favorable compared to rival treatments [1][3]. - Additional competing treatments, such as EyePoint Pharmaceuticals Inc.'s Duravyu, are progressing in clinical trials, which may further diminish the appeal of gene therapies in retinal diseases [3]. - The overall appeal of gene therapies in retinal diseases is viewed as decreasing, leading to a reduction in the projected peak sales for ABBV-RGX-314 [3]. Group 2: Clinical Trials and Future Developments - Data from pivotal trials evaluating the safety and efficacy of ABBV-RGX-314 in patients with wet AMD are expected in 2026, with plans for a Phase 3 clinical program for diabetic retinopathy [2]. - Regenxbio and Nippon Shinyaku announced a strategic partnership to develop RGX-121 and RGX-111, with Regenxbio set to receive $110 million upfront and up to $700 million in potential milestone payments [6]. Group 3: Pricing and Market Dynamics - As more patients adopt treatments, prices are expected to decline, potentially setting a lower price benchmark for ABBV-RGX-314 as it approaches commercialization [4]. - The increasing use of Regeneron Pharmaceutical Inc's Eylea biosimilars is noted, which have captured market share from the branded version despite the availability of Eylea HD [4]. Group 4: Stock Performance - RGNX stock has experienced a decline of 7.29%, trading at $7.315 [7].

Shares of Regenxbio (RGNX) have gained 9.3% over the past four weeks to close the last trading session at $8.21, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $35.55 indicates a potential upside of 333%.The average comprises 11 short-term price targets ranging from a low of $22 to a high of $52, with a standard deviation of $10.42. While the lowest estimate indicates an increa ...

Core Insights - REGENXBIO Inc. announced that data from its RGX-121 program for treating mucopolysaccharidosis type II (MPS II) will be presented at the 21st Annual WORLDSymposium™ 2025 in San Diego, CA from February 3-7, 2025 [1] - The presentations will include topline results from the pivotal phase of the Phase I/II/III CAMPSIITE® trial of RGX-121 [1] Company Overview - REGENXBIO is a leading clinical-stage biotechnology company focused on gene therapy, founded in 2009 [3] - The company has developed AAV Therapeutics, a class of gene therapy medicines, and is advancing a pipeline for rare and retinal diseases, including RGX-202 for Duchenne and ABBV-RGX-314 for wet AMD and diabetic retinopathy in collaboration with AbbVie [3] - Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, which includes Novartis' ZOLGENSMA for spinal muscular atrophy [3] - AAV Therapeutics are designed as one-time treatments with the potential to transform healthcare delivery for millions [3]