Company Overview - Regenxbio Inc. is a biotech company focused on developing one-time gene therapies using its AAV (adeno-associated virus) platform aimed at treating rare genetic diseases and some more common conditions [1] Business Model - The company combines scientific expertise in drug development with financial and market analysis to identify promising biotechnology innovations, including novel mechanisms of action and first-in-class therapies [1] Research Focus - The research approach emphasizes evaluating the science behind drug candidates, the competitive landscape, clinical trial design, and potential market opportunities while balancing financial fundamentals and valuation [1] Industry Insights - The biotech sector is characterized by breakthrough science that can lead to significant returns, but it also requires careful scrutiny due to inherent risks [1]

Core Insights - REGENX is actively engaged in various initiatives within the gene therapy sector, focusing on multiple clinical areas [2] Company Overview - REGENX is introduced as a gene therapy platform, indicating its involvement in innovative treatments [2]

Summary of REGENXBIO Inc. Conference Call Company Overview - REGENXBIO Inc. has been operational for approximately 15 years, focusing on AAV (Adeno-Associated Virus) gene therapy, with significant advancements in late-stage development over the last decade [2] - The company has a workforce of about 350 employees and operates its own manufacturing facility in Rockville, Maryland [2] Key Programs and Developments RGX-202 for Duchenne Muscular Dystrophy (DMD) - RGX-202 is a gene therapy designed to deliver microdystrophin, which is engineered to resemble full-length natural dystrophin [3] - The program has achieved an 80% full capsid purity in manufacturing, which is a significant improvement [3] - A unique immune suppression regimen has been implemented, resulting in a positive safety profile and good functional outcomes for patients [4] - The program is seeing robust microdystrophin levels, particularly in patients aged eight and older, which is noteworthy [7] - Enrollment for the pivotal trial is expected to be completed by October [8] Regulatory and Commercial Insights - The FDA's stance on the accelerated approval pathway for DMD remains unchanged, with functional data included in the review package [11] - The company is optimistic about being second to market for DMD therapies, with a larger prevalent population than previously anticipated [14] - Commercial production is underway in Rockville, with plans to file for approval next year and a target launch in 2027 [14] Hunter Syndrome (RGX-121) - MS Pharma is the commercialization partner for RGX-121 in the U.S. and Asia, with double-digit royalties associated with the agreement [21] - The FDA requested additional 12-month data, which has been provided, leading to a three-month delay in the approval process [22] - Positive one-year data has been presented, reinforcing the efficacy of the treatment [23] Retinal Diseases (RGX-314) - The program for retinal diseases is partnered with AbbVie, with a focus on superchoroidal delivery for diabetic retinopathy (DR) [27] - A pivotal program has been restructured to include a 2B run due to encouraging data from earlier trials [27] - Enrollment for the DR study is expected to be rapid due to the in-office procedure nature of the treatment [28] Market Dynamics and Competitive Landscape - The company does not perceive immediate threats from rising biotech innovation in China, emphasizing their investments in manufacturing and clinical development [40] - AI is being leveraged for capsid discovery and optimizing clinical study sites [41] - Regulatory interactions with the FDA are described as normal, with no significant concerns regarding tariffs impacting the business [43] Financial Position - REGENXBIO has over $360 million in cash, providing a runway into early 2027, with potential for additional non-dilutive financing through monetization of PRV and other agreements [39] Conclusion - REGENXBIO is positioned for significant advancements in gene therapy, with promising data across multiple programs and a strong financial foundation to support ongoing development and commercialization efforts. The company is optimistic about its future in the competitive landscape of gene therapies.

Group 1 - The article discusses Regenxbio (NASDAQ: RGNX) and its ongoing developments related to Wet AMD ABBV-RGX-314, with results expected to remain on track for 2026 [2] - The author operates the Biotech Analysis Central service, which provides in-depth analysis of various pharmaceutical companies and includes a library of over 600 biotech investing articles [2] - The service offers a model portfolio of more than 10 small and mid-cap stocks, along with live chat and analysis to assist healthcare investors in making informed decisions [2]

Core Insights - REGENXBIO Inc. announced positive data from the Phase I/II/III CAMPSIITE trial for RGX-121, a treatment for Mucopolysaccharidosis Type II (MPS II), presented at the ICIEM 2025 [1][2] - The company submitted long-term pivotal results to the FDA as part of the ongoing Biologics License Application (BLA) review for RGX-121 [1] Data Summary - In the pivotal phase of the CAMPSIITE trial (n=13), participants showed an 82% median reduction in cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6, a key biomarker for MPS II, sustained over one year [3] - The primary endpoint of the trial was met with statistical significance (p < 0.0001) for the proportion of participants with CSF HS D2S6 below maximum attenuated levels at week 16 [3] - Positive neurodevelopmental outcomes were observed, with participants demonstrating skill acquisition or stability across all sub-scales of the Bayley Scales of Infant and Toddler Development at one year [4] Correlation and Biomarkers - Data from both the dose-finding and pivotal phases indicated a strong correlation between CSF HS D2S6 levels at week 16 and neurocognitive outcomes at one year, supporting its use as a surrogate endpoint for clinical benefit [5] - The buildup of glycosaminoglycans (GAGs) in MPS II correlates with clinical manifestations, including neurodevelopmental deficits [5][10] Regulatory Progress - The FDA completed a pre-license inspection for the RGX-121 BLA with no safety-related concerns raised, and the decision is expected by February 8, 2026 [6] - RGX-121 has received multiple designations from the FDA, including Orphan Drug Product and Fast Track [9] Product Overview - RGX-121 is a potential one-time AAV therapeutic designed to address the underlying genetic cause of Hunter syndrome, with over 80% reduction in CSF levels of HS D2S6 sustained through one year [7] - If approved, RGX-121 would be the first commercially available therapy targeting the genetic cause of Hunter syndrome [7] Company Background - REGENXBIO is a biotechnology company focused on gene therapy, with a late-stage pipeline including treatments for rare diseases [13] - The company has pioneered AAV gene therapy since its founding in 2009 and has treated thousands of patients with its AAV platform [13]

Core Viewpoint - The FDA has extended the review timeline for REGENXBIO Inc.'s biologics license application for RGX-121, a gene therapy for Mucopolysaccharidosis II (MPS II), from November 9, 2025, to February 8, 2026, following the submission of additional long-term clinical data [1][2]. Group 1: FDA Review and Data Submission - The extension of the PDUFA goal date is due to the company's submission of longer-term clinical data from the pivotal study involving 13 patients [2]. - The FDA completed a pre-license and bioresearch monitoring inspection for RGX-121 with no observations and has raised no safety-related concerns during the review [4]. Group 2: Clinical Data and Efficacy - Positive 12-month clinical data for RGX-121 are consistent with previously submitted biomarker and neurodevelopmental data and will be presented at the International Congress of Inborn Errors of Metabolism in September 2025 [3]. - MPS II patients treated with RGX-121 showed an 86% median reduction in cerebrospinal fluid levels of D2S6, a key biomarker of brain disease activity, approaching normal levels [7]. Group 3: Mechanism and Treatment Implications - RGX-121 is designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system, potentially providing a permanent source of the I2S protein beyond the blood-brain barrier [4][5]. - The treatment could lead to long-term cross-correction of cells throughout the CNS, addressing the underlying deficiency in MPS II patients [5][6]. Group 4: Market Reaction - Following the news, REGENXBIO's stock price decreased by 7.46%, trading at $8.06 [8].

Core Viewpoint - REGENXBIO Inc. announced an extension of the FDA review timeline for its Biologics License Application (BLA) for RGX-121, a treatment for Mucopolysaccharidosis II (MPS II), from November 9, 2025, to February 8, 2026 [1] Group 1: FDA Review and Clinical Data - The extension follows the submission of long-term clinical data for all patients in the pivotal study of RGX-121, which included 13 patients, in response to an FDA information request [2] - Positive 12-month clinical data are consistent with previously submitted biomarker and neurodevelopmental data and will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM) in September 2025 [2] - The FDA completed a pre-license inspection and bioresearch monitoring inspection for the RGX-121 BLA with no observations and no safety-related concerns raised during the review [3] Group 2: Company Statements and Designations - The President and CEO of REGENXBIO emphasized the urgent need for a therapeutic option for boys with Hunter syndrome and expressed confidence that commercial launch plans remain on track [4] - RGX-121 has received multiple designations from the FDA, including Orphan Drug Product, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) [4] Group 3: Product and Disease Overview - RGX-121 is a potential one-time AAV therapeutic designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system, potentially providing a permanent source of the I2S protein [5] - MPS II, or Hunter Syndrome, is a rare disease caused by a deficiency in the lysosomal enzyme I2S, leading to significant medical needs, particularly for neurological manifestations [6] Group 4: Future Plans and Market Potential - If approved, RGX-121 would be the first and only commercially available therapy designed to address the underlying genetic cause of Hunter syndrome [8] - REGENXBIO plans to present updated pivotal data during the ICIEM meeting in September 2025 [8]

Group 1 - Regenxbio reported a quarterly loss of $1.38 per share, which was worse than the Zacks Consensus Estimate of a loss of $1.13, and compared to a loss of $1.05 per share a year ago, indicating a significant earnings surprise of -22.12% [1] - The company posted revenues of $21.36 million for the quarter ended June 2025, missing the Zacks Consensus Estimate by 21.16%, and this represents a decline from year-ago revenues of $22.3 million [2] - Over the last four quarters, Regenxbio has surpassed consensus EPS estimates only once, indicating challenges in meeting market expectations [2] Group 2 - The stock has gained approximately 6.5% since the beginning of the year, underperforming compared to the S&P 500's gain of 7.9% [3] - The company's earnings outlook is crucial for investors, as it includes current consensus earnings expectations for upcoming quarters and any recent changes to these expectations [4] - The current consensus EPS estimate for the upcoming quarter is $0.29 on revenues of $102.44 million, while for the current fiscal year, it is -$0.56 on revenues of $293.35 million [7] Group 3 - The Zacks Industry Rank places the Medical - Biomedical and Genetics sector in the top 41% of over 250 Zacks industries, suggesting a favorable environment for stocks in this sector [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors [5] - The current estimate revisions trend for Regenxbio is mixed, resulting in a Zacks Rank 3 (Hold), suggesting the stock is expected to perform in line with the market in the near future [6]

Financial Data and Key Metrics Changes - REGENXBIO ended Q2 2025 with cash, cash equivalents, and marketable securities of $364 million, up from $245 million as of December 31, 2024, primarily due to a $110 million upfront payment from Nippon Shinyaku and $145 million in net proceeds from royalty monetization [26][28] - R&D expenses for Q2 2025 were $60 million, compared to $49 million in Q2 2024, attributed to manufacturing and clinical trial expenses for Cirovec and RGX-202 pivotal trials [26] Business Line Data and Key Metrics Changes - RGX-202 is positioned as a potential best-in-class gene therapy for Duchenne, with enrollment in the pivotal study expected to complete by October 2025, ahead of schedule [6][23] - The retinal disease franchise, particularly ADBV RGX-314 (Suravec), is advancing into pivotal trials for diabetic retinopathy, with a $100 million milestone payment upon the first patient dosed in the Phase 2b portion [10][27] Market Data and Key Metrics Changes - The diabetic retinopathy market impacts over 20 million people globally, with REGENXBIO's Suravec showing promising results in reducing vision-threatening events [14][18] - The FDA accepted the BLA for RGX-121 for Hunter syndrome, with a target PDUFA date of November 9, 2025, indicating strong market potential for this treatment [11][12] Company Strategy and Development Direction - The company is focused on executing its strategy to bring transformative gene therapies to market, with multiple late-stage programs and a strong financial position to support commercialization efforts [29][30] - REGENXBIO is initiating commercial manufacturing for RGX-202, with the capability to produce up to 2,500 doses per year, enhancing its market readiness [9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong interest from the Duchenne community and the proactive immune suppression regimen, which has garnered positive feedback [34] - The company anticipates significant progress across all late-stage programs, with a potential FDA approval on the horizon [24][30] Other Important Information - The company has a differentiated therapeutic approach for RGX-202, including a proactive immune suppression regimen aimed at improving safety outcomes [22] - The partnership with AbbVie for Suravec is expected to enhance the commercial opportunity in chronic eye care, with ongoing trials progressing well [10][19] Q&A Session Summary Question: What is the reaction from the DMD community regarding the prophylaxis regimen for RGX-202? - Management noted that interest in the program is at an all-time high, with the proactive immune suppression regimen being well-received by the patient community [34] Question: Can you elaborate on the improvements seen in diabetic retinopathy patients between year one and year two? - Management highlighted that the improvement in efficacy over time suggests sustained anti-VEGF activity, which is compelling for the indication [36] Question: What is the rationale for adding dose level four in the pivotal study for diabetic retinopathy? - The decision was driven by the desire to maximize efficacy and safety, ensuring no potential benefits are left unexamined [44] Question: How does the company view the market dynamics for RGX-202 at launch? - Management expects a significant portion of the prevalent population to remain available, with a strong product profile positioning RGX-202 as a potential blockbuster [78] Question: What is the expected path to approval for the diabetic retinopathy program? - The Phase 2b/3 study is intended to be one of the two required studies for regulatory approval, with robust data anticipated [71]

PART I—FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=5&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents REGENXBIO Inc.'s unaudited consolidated financial statements, including balance sheets, statements of operations and comprehensive loss, statements of stockholders' equity, and statements of cash flows, along with detailed notes explaining the company's accounting policies, financial instruments, and significant agreements for the periods ended June 30, 2025, and December 31, 2024 [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) Consolidated Balance Sheet Highlights (in thousands) | Metric | June 30, 2025 | December 31, 2024 | Change (vs. Dec 31, 2024) | | :-------------------------------- | :------------ | :---------------- | :------------------------ | | **Assets** | | | | | Cash and cash equivalents | $79,558 | $57,526 | +$22,032 | | Marketable securities | $243,740 | $177,161 | +$66,579 | | Total current assets | $373,752 | $278,001 | +$95,751 | | Total assets | $581,027 | $465,989 | +$115,038 | | **Liabilities & Equity** | | | | | Deferred revenue (current) | $13,977 | $115 | +$13,862 | | Royalty monetization liabilities (current) | $40,302 | $34,309 | +$5,993 | | Total current liabilities | $119,540 | $103,194 | +$16,346 | | Deferred revenue (non-current) | $23,804 | $— | +$23,804 | | Royalty monetization liabilities (non-current) | $153,693 | $25,378 | +$128,315 | | Total liabilities | $367,348 | $206,338 | +$161,010 | | Total stockholders' equity | $213,679 | $259,651 | -$45,972 | - Total assets increased by **$115.0 million**, primarily driven by increases in cash, cash equivalents, and marketable securities[17](index=17&type=chunk) - Total liabilities significantly increased by **$161.0 million**, largely due to a substantial rise in royalty monetization liabilities and deferred revenue[17](index=17&type=chunk) [Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Consolidated Statements of Operations and Comprehensive Loss Highlights (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change (YoY) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change (YoY) | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------- | :------------------------------- | :------------------------------- | :----------- | | License and royalty revenue | $18,465 | $21,846 | -$3,381 | $105,514 | $37,190 | +$68,324 | | Service revenue | $2,894 | $449 | +$2,445 | $4,857 | $727 | +$4,130 | | Total revenues | $21,359 | $22,295 | -$936 | $110,371 | $37,917 | +$72,454 | | Research and development expense | $59,500 | $48,869 | +$10,631 | $112,587 | $103,713 | +$8,874 | | General and administrative expense | $19,883 | $18,855 | +$1,028 | $40,230 | $37,146 | +$3,084 | | Loss from operations | $(63,278) | $(56,037) | -$7,241 | $(51,151) | $(119,900) | +$68,749 | | Interest expense | $(10,993) | $(449) | -$10,544 | $(19,563) | $(2,422) | -$17,141 | | Net loss | $(70,871) | $(52,989) | -$17,882 | $(64,788) | $(116,319) | +$51,531 | | Net loss per share, basic and diluted | $(1.38) | $(1.05) | -$0.33 | $(1.26) | $(2.41) | +$1.15 | - Total revenues for the three months ended June 30, 2025, decreased slightly by **$0.9 million** year-over-year, primarily due to a **$3.4 million** decrease in Zolgensma royalty revenue, partially offset by a **$2.4 million** increase in service revenue from the Nippon Shinyaku collaboration[18](index=18&type=chunk)[203](index=203&type=chunk)[204](index=204&type=chunk) - For the six months ended June 30, 2025, total revenues significantly increased by **$72.5 million**, driven by a **$70.0 million** upfront license revenue from the Nippon Shinyaku collaboration[18](index=18&type=chunk)[208](index=208&type=chunk)[209](index=209&type=chunk) - Net loss for the three months ended June 30, 2025, widened to **$(70.9) million** from **$(53.0) million** in the prior year, largely due to increased research and development expenses and a substantial increase in interest expense from royalty monetization liabilities[18](index=18&type=chunk)[205](index=205&type=chunk)[207](index=207&type=chunk) - Net loss for the six months ended June 30, 2025, improved to **$(64.8) million** from **$(116.3) million** in the prior year, primarily due to the significant increase in license and service revenues, despite higher operating and interest expenses[18](index=18&type=chunk)[208](index=208&type=chunk)[210](index=210&type=chunk)[212](index=212&type=chunk) [Consolidated Statements of Stockholders' Equity](index=7&type=section&id=Consolidated%20Statements%20of%20Stockholders'%20Equity) Changes in Stockholders' Equity (in thousands) | Metric | Three Months Ended June 30, 2025 | Six Months Ended June 30, 2025 | | :-------------------------------- | :------------------------------- | :------------------------------- | | Balances at March 31, 2025 / Dec 31, 2024 | $274,197 / $259,651 | $259,651 | | Issuance of warrants, net of transaction costs | $1,610 | $1,610 | | Stock-based compensation expense | $8,653 | $17,190 | | Net loss | $(70,871) | $(64,788) | | Balances at June 30, 2025 | $213,679 | $213,679 | - Total stockholders' equity decreased from **$259.7 million** at December 31, 2024, to **$213.7 million** at June 30, 2025, primarily due to the net loss incurred during the period, partially offset by stock-based compensation and warrant issuance[17](index=17&type=chunk)[24](index=24&type=chunk) [Consolidated Statements of Cash Flows](index=9&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) Consolidated Statements of Cash Flows Highlights (in thousands) | Metric | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change (YoY) | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------- | | Net cash used in operating activities | $(15,713) | $(100,952) | +$85,239 | | Net cash provided by (used in) investing activities | $(95,693) | $12,915 | -$108,608 | | Net cash provided by financing activities | $133,438 | $111,280 | +$22,158 | | Net increase in cash and cash equivalents and restricted cash | $22,032 | $23,243 | -$1,211 | - Net cash used in operating activities significantly decreased by **$85.2 million**, primarily due to the **$110.0 million** upfront fee from the Nippon Shinyaku collaboration[26](index=26&type=chunk)[220](index=220&type=chunk)[221](index=221&type=chunk) - Net cash used in investing activities increased by **$108.6 million**, mainly due to higher purchases of marketable debt securities in 2025 compared to 2024[26](index=26&type=chunk)[223](index=223&type=chunk)[224](index=224&type=chunk) - Net cash provided by financing activities increased by **$22.2 million**, driven by proceeds from the 2025 Royalty Bond and warrants, partially offset by royalty monetization repayments[26](index=26&type=chunk)[225](index=225&type=chunk)[226](index=226&type=chunk) [Notes to Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) [1. Nature of Business](index=10&type=section&id=1.%20Nature%20of%20Business) - REGENXBIO Inc. is a clinical-stage biotechnology company focused on gene therapy using its proprietary NAV Technology Platform, which includes exclusive rights to a large portfolio of AAV vectors[29](index=29&type=chunk) - The company has incurred cumulative losses of **$996.9 million** as of June 30, 2025, and relies on additional capital to fund operations, with current cash, cash equivalents, and marketable securities of **$363.6 million** expected to fund operations for at least the next **12 months**[30](index=30&type=chunk) [2. Summary of Significant Accounting Policies](index=10&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) - The company reclassified service revenue separately from license and royalty revenues retrospectively due to a collaboration and license agreement with Nippon Shinyaku Co., Ltd. in March 2025[36](index=36&type=chunk) - Restricted cash of **$2.03 million** as of June 30, 2025, collateralizes letters of credit for lease agreements[37](index=37&type=chunk) - Revenue recognition follows ASC 606, involving a five-step model to identify contracts, performance obligations, transaction price, allocation, and recognition, with specific considerations for variable consideration, milestones, and royalties[43](index=43&type=chunk)[44](index=44&type=chunk)[45](index=45&type=chunk)[52](index=52&type=chunk)[53](index=53&type=chunk)[54](index=54&type=chunk)[55](index=55&type=chunk) - New accounting pronouncements include ASU 2023-09 (Income Tax Disclosures, effective Jan 1, 2025) and ASU 2024-03 (Expense Disaggregation Disclosures, effective Jan 1, 2027), with the company currently evaluating their impact[60](index=60&type=chunk)[61](index=61&type=chunk) [3. Marketable Securities](index=19&type=section&id=3.%20Marketable%20Securities) Marketable Securities (Available-for-Sale Debt Securities) (in thousands) | Category | Amortized Cost (June 30, 2025) | Fair Value (June 30, 2025) | Amortized Cost (Dec 31, 2024) | Fair Value (Dec 31, 2024) | | :-------------------------------- | :----------------------------- | :------------------------- | :----------------------------- | :------------------------- | | U.S. government and agency securities | $132,032 | $132,019 | $44,281 | $44,215 | | Certificates of deposit | $245 | $245 | $1,466 | $1,462 | | Corporate bonds | $151,649 | $151,772 | $141,474 | $141,663 | | Total | $283,926 | $284,036 | $187,221 | $187,340 | - The company's marketable securities, consisting solely of available-for-sale debt securities, increased significantly from **$187.3 million** at December 31, 2024, to **$284.0 million** at June 30, 2025[62](index=62&type=chunk) - As of June 30, 2025, the company held **41** investment grade securities in an unrealized loss position, but did not identify any credit losses due to intent and ability to hold until recovery and low severity of losses[64](index=64&type=chunk) [4. Fair Value Measurements](index=21&type=section&id=4.%20Fair%20Value%20Measurements) Fair Value of Cash Equivalents and Marketable Securities (in thousands) | Category | Level 2 (June 30, 2025) | Level 2 (Dec 31, 2024) | | :-------------------------------- | :---------------------- | :--------------------- | | Money market mutual funds | $44,975 | $43,895 | | U.S. government and agency securities | $141,988 | $46,713 | | Certificates of deposit | $245 | $1,462 | | Corporate bonds | $151,772 | $141,663 | | Total cash equivalents and marketable securities | $338,980 | $233,733 | - The fair values of cash equivalents and marketable securities are primarily categorized as Level 2, based on quoted market prices or broker/dealer quotations for similar assets[65](index=65&type=chunk)[67](index=67&type=chunk) - Royalty monetization liabilities are estimated at fair value using Level 3 inputs, based on the company's estimate of future royalties, milestones, and repayment obligations[67](index=67&type=chunk) [5. Property and Equipment, Net](index=22&type=section&id=5.%20Property%20and%20Equipment,%20Net) Property and Equipment, Net (in thousands) | Category | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | Laboratory and manufacturing equipment | $77,765 | $77,141 | | Leasehold improvements | $101,533 | $101,465 | | Total property and equipment, net | $111,017 | $117,589 | - Property and equipment, net, decreased from **$117.6 million** at December 31, 2024, to **$111.0 million** at June 30, 2025[69](index=69&type=chunk) - An impairment of **$0.7 million** on furniture, fixtures, and leasehold improvements was recorded in Q1 2024 due to a sublease agreement for New York office facilities[69](index=69&type=chunk) [6. Leases](index=22&type=section&id=6.%20Leases) - The company subleased its New York office space in March 2024, with the sublease term commencing in April 2024 and expiring concurrently with the main lease in April 2027[70](index=70&type=chunk)[71](index=71&type=chunk) - Sublease income of **$0.1 million** and **$0.2 million** was recognized for the three and six months ended June 30, 2025, respectively[71](index=71&type=chunk) - An impairment loss of **$2.1 million** was recognized in Q1 2024 on the long-lived asset group associated with the New York Sublease, allocated to right-of-use assets (**$1.4 million**) and property and equipment (**$0.7 million**)[73](index=73&type=chunk) [7. Royalty Monetization Liabilities](index=23&type=section&id=7.%20Royalty%20Monetization%20Liabilities) Royalty Monetization Liabilities (in thousands) | Agreement | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | 2020 Royalty Purchase Agreement | $48,231 | $59,687 | | 2025 Royalty Bond | $145,764 | $— | | Total | $193,995 | $59,687 | | Current portion | $40,302 | $34,309 | | Non-current portion | $153,693 | $25,378 | - Total royalty monetization liabilities increased significantly from **$59.7 million** at December 31, 2024, to **$194.0 million** at June 30, 2025, primarily due to the new 2025 Royalty Bond[74](index=74&type=chunk) - The 2020 Royalty Purchase Agreement's effective interest rate was **71.4%** as of June 30, 2025, up from **65.5%** at December 31, 2024, based on estimated future Zolgensma royalty payments[80](index=80&type=chunk) - In May 2025, the company entered into a **$250.0 million** 2025 Royalty Bond with HCR, with an initial funding of **$144.5 million** (net of discounts/costs). This bond bears interest at **9.75%** plus 3-month SOFR (minimum **14.0%**) and is repaid from specified royalties and milestones[82](index=82&type=chunk)[83](index=83&type=chunk)[84](index=84&type=chunk) - In connection with the 2025 Royalty Bond, warrants to purchase **268,096** shares of common stock were issued to HCR, valued at **$1.7 million** and recorded as additional paid-in capital[86](index=86&type=chunk) [8. Commitments and Contingencies](index=27&type=section&id=8.%20Commitments%20and%20Contingencies) Expenses Related to GSK License (in thousands) | Category | Three Months Ended June 30, 2025 | Six Months Ended June 30, 2025 | | :-------------------------------- | :------------------------------- | :------------------------------- | | Royalties on net sales of Zolgensma | $5,204 | $7,786 | | Other cost of license and royalty revenues | $5 | $243 | | General and administrative | $53 | $107 | | Total | $5,262 | $8,136 | - The company has a potential dispute with GlaxoSmithKline (GSK) regarding sublicense fees, with GSK claiming underpayment based on a broader interpretation of sublicense revenue. The company disagrees and does not believe a loss is probable[92](index=92&type=chunk) [9. Capitalization](index=29&type=section&id=9.%20Capitalization) - In March 2024, the company completed a public offering, raising **$131.1 million** net, through the sale of common stock and pre-funded warrants[93](index=93&type=chunk) - As of June 30, 2025, **1,125,440** March 2024 Pre-funded Warrants remained outstanding, with **199,300** shares exercised during the six months ended June 30, 2025[95](index=95&type=chunk) - The May 2025 Warrants, issued in connection with the 2025 Royalty Bond, allow purchase of **268,096** common shares at **$14.92/share**, classified as equity, with none exercised as of June 30, 2025[96](index=96&type=chunk) - The company has an At-the-Market (ATM) Offering Program established in December 2024 to sell up to **$150.0 million** of common stock, with no shares sold as of June 30, 2025[97](index=97&type=chunk) [10. License and Collaboration Agreements](index=30&type=section&id=10.%20License%20and%20Collaboration%20Agreements) Revenues from License and Collaboration Agreements (in thousands) | Revenue Type | Three Months Ended June 30, 2025 | Six Months Ended June 30, 2025 | | :-------------------------------- | :------------------------------- | :------------------------------- | | Zolgensma royalties | $18,423 | $35,416 | | Nippon Shinyaku licenses | $— | $69,979 | | Other license and royalty revenue | $42 | $119 | | Total license and royalty revenue | $18,465 | $105,514 | | Nippon Shinyaku services | $2,720 | $4,494 | | Other service revenue | $174 | $363 | | Total service revenue | $2,894 | $4,857 | | Total revenues | $21,359 | $110,371 | - Unachieved milestones from license and collaboration agreements could result in aggregate payments of up to **$2.18 billion**, including **$548.2 million** for clinical trials, **$106.3 million** for regulatory approvals, and **$1.53 billion** for sales targets[99](index=99&type=chunk) - Deferred revenue as of June 30, 2025, was **$37.8 million**, primarily from the Nippon Shinyaku collaboration, expected to be satisfied over approximately **five years**[101](index=101&type=chunk) - The AbbVie Collaboration Agreement was amended in August 2025, modifying the development plan and milestone payment structure for the ABBV-RGX-314 DR program, splitting a **$200.0 million** milestone into two **$100.0 million** payments[112](index=112&type=chunk)[163](index=163&type=chunk) - The Nippon Shinyaku Collaboration Agreement, effective March 2025, granted licenses for RGX-121 and RGX-111, with an upfront fee of **$110.0 million** and potential milestones up to **$700.0 million**. The company recognized **$74.5 million** in revenue from this agreement in H1 2025[113](index=113&type=chunk)[115](index=115&type=chunk)[164](index=164&type=chunk)[165](index=165&type=chunk) [11. Stock-based Compensation](index=40&type=section&id=11.%20Stock-based%20Compensation) Stock-based Compensation Expense (in thousands) | Award Type | Three Months Ended June 30, 2025 | Six Months Ended June 30, 2025 | | :-------------------------------- | :------------------------------- | :------------------------------- | | Stock options | $4,778 | $9,485 | | Restricted stock units | $3,713 | $7,381 | | Employee stock purchase plan | $162 | $324 | | Total | $8,653 | $17,190 | - The 2015 Equity Incentive Plan expired in June 2025, replaced by the 2025 Equity Incentive Plan, which authorized **5,500,000** shares for issuance[123](index=123&type=chunk)[124](index=124&type=chunk) - As of June 30, 2025, **$65.6 million** of unrecognized stock-based compensation expense remains, to be recognized over a weighted-average period of **2.5 years**[127](index=127&type=chunk) [12. Income Taxes](index=42&type=section&id=12.%20Income%20Taxes) - The company maintains a full valuation allowance for its net deferred tax assets due to a history of operating losses, indicating that the benefit of these assets is not likely to be realized[133](index=133&type=chunk) - The company is evaluating the impact of the recently enacted One Big Beautiful Bill Act (OBBBA) on its consolidated financial statements, which amends U.S. tax laws related to bonus depreciation and R&D expense deductions[134](index=134&type=chunk) [13. Net Loss Per Share](index=43&type=section&id=13.%20Net%20Loss%20Per%20Share) - Due to net losses, common stock equivalents (stock options, restricted stock units, employee stock purchase plan, warrants) were excluded from diluted net loss per share calculations as their effect would be anti-dilutive[135](index=135&type=chunk) Potentially Dilutive Common Stock Equivalents Excluded (in thousands) | Category | June 30, 2025 | June 30, 2024 | | :-------------------------------- | :------------ | :------------ | | Stock options issued and outstanding | 11,810 | 9,638 | | Unvested restricted stock units outstanding | 2,982 | 1,668 | | Employee stock purchase plan | 111 | 78 | | Warrants outstanding | 268 | — | | Total | 15,171 | 11,384 | [14. Segment Information](index=43&type=section&id=14.%20Segment%20Information) - The company operates as a single operating segment, focusing on the development and commercialization of gene therapies, with the CEO reviewing consolidated net income (loss) and cash, cash equivalents, and marketable securities for performance assessment and resource allocation[136](index=136&type=chunk) - As of June 30, 2025, cash, cash equivalents, and marketable securities were **$363.6 million**, up from **$244.9 million** at December 31, 2024[136](index=136&type=chunk) [15. Supplemental Disclosures](index=45&type=section&id=15.%20Supplemental%20Disclosures) Other Current Assets (in thousands) | Category | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | Net cost reimbursement due from AbbVie | $16,989 | $11,304 | | Accrued interest on investments | $1,287 | $1,094 | | Other | $1,203 | $1,376 | | Total | $19,479 | $13,774 | Accrued Expenses and Other Current Liabilities (in thousands) | Category | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | Accrued personnel costs | $13,801 | $17,607 | | Accrued external research and development expenses | $11,418 | $8,998 | | Accrued sublicense fees and royalties | $7,548 | $8,658 | | Accrued external general and administrative expenses | $2,015 | $2,002 | | Accrued purchases of property and equipment | $39 | $156 | | Other | $736 | $649 | | Total | $35,557 | $38,070 | - Non-cash financing activities for the six months ended June 30, 2025, included **$2.7 million** of accrued interest converted to principal balance under the 2025 Royalty Bond[144](index=144&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=46&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides a detailed discussion of REGENXBIO Inc.'s financial condition and results of operations for the three and six months ended June 30, 2025, compared to the same periods in 2024, covering business overview, product pipeline, collaboration agreements, revenue streams, operating expenses, critical accounting policies, and liquidity and capital resources [Overview](index=46&type=section&id=Overview) - REGENXBIO is a clinical-stage biotechnology company focused on gene therapy using its proprietary NAV Technology Platform to deliver functional genes for one-time treatments of various diseases[147](index=147&type=chunk)[148](index=148&type=chunk) [Overview of Product Candidates](index=46&type=section&id=Overview%20of%20Product%20Candidates) - ABBV-RGX-314 (surabgene lomparvovec, sura-vec) is being developed in collaboration with AbbVie for chronic retinal conditions (wet AMD, DR, DME) using both subretinal and suprachoroidal delivery. Topline data from pivotal trials (ATMOSPHERE and ASCENT) for wet AMD subretinal delivery are expected in 2026[148](index=148&type=chunk)[149](index=149&type=chunk) - New ALTITUDE trial data for DR showed durable safety and efficacy through **two years** with a single, in-office suprachoroidal injection, leading to plans for a pivotal two-part Phase IIb/III trial[152](index=152&type=chunk) - RGX-202 for Duchenne muscular dystrophy (Duchenne) showed positive interim safety and efficacy data from the Phase I/II AFFINITY DUCHENNE trial, with pivotal study enrollment expected to complete by October 2025 and BLA submission planned for mid-2026[154](index=154&type=chunk)[155](index=155&type=chunk)[156](index=156&type=chunk) - RGX-121 for Mucopolysaccharidosis Type II (MPS II) achieved its primary endpoint in the CAMPSIITE trial, demonstrating a reduction in cerebrospinal fluid Heparan sulfate levels. The FDA granted priority review for the BLA submitted in March 2025, with a PDUFA target action date of November 9, 2025[158](index=158&type=chunk)[159](index=159&type=chunk) - RGX-111 for Mucopolysaccharidosis Type I (MPS I) development was halted in November 2023 due to strategic pipeline prioritization, but efforts to continue development are ongoing as part of the Nippon Shinyaku partnership[161](index=161&type=chunk) [AbbVie Collaboration for ABBV-RGX-314](index=49&type=section&id=AbbVie%20Collaboration%20for%20ABBV-RGX-314) - The collaboration with AbbVie for ABBV-RGX-314 involves joint development and commercialization, with AbbVie paying an upfront fee of **$370.0 million** and potential milestones up to **$1.38 billion**[162](index=162&type=chunk) - An August 2025 amendment to the AbbVie Collaboration Agreement modified the development plan and milestone structure for the DR program, splitting a **$200.0 million** milestone into two **$100.0 million** payments[163](index=163&type=chunk) [Nippon Shinyaku Collaboration for RGX-121 and RGX-111](index=49&type=section&id=Nippon%20Shinyaku%20Collaboration%20for%20RGX-121%20and%20RGX-111) - The January 2025 collaboration with Nippon Shinyaku for RGX-121 and RGX-111 included an upfront payment of **$110.0 million** and potential milestones up to **$700.0 million**, with **$74.5 million** revenue recognized in H1 2025[164](index=164&type=chunk)[165](index=165&type=chunk) - Future royalties and milestones from the Nippon Shinyaku agreement, along with other NAV Technology Platform licenses, will be used to repay principal and interest under the May 2025 Royalty Bond with HCR[166](index=166&type=chunk) [NAV Technology Licensing Platform](index=51&type=section&id=NAV%20Technology%20Licensing%20Platform) - The NAV Technology Platform is licensed to other biotechnology and pharmaceutical companies, with one commercial product (Zolgensma) and several others in preclinical and clinical development, providing additional revenue opportunities and technological validation[167](index=167&type=chunk) [Financial Overview](index=51&type=section&id=Financial%20Overview) - Revenues are primarily from licensing the NAV Technology Platform and other intellectual property rights, with Zolgensma royalties being a significant component[168](index=168&type=chunk)[171](index=171&type=chunk) - Operating expenses consist mainly of cost of license and royalty revenues, research and development (R&D), and general and administrative (G&A) expenses, with personnel costs being a significant component[172](index=172&type=chunk) - R&D expenses are expected to remain significant due to ongoing product candidate development and early-stage research[177](index=177&type=chunk) - Interest expense is primarily associated with royalty monetization liabilities, including the 2020 Royalty Purchase Agreement and the 2025 Royalty Bond with HCR[183](index=183&type=chunk) [Critical Accounting Policies and Estimates](index=56&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) - Revenue recognition under ASC 606 involves a five-step model, requiring significant judgment in identifying performance obligations, determining transaction price (including variable consideration and milestones), and allocating revenue[186](index=186&type=chunk)[187](index=187&type=chunk)[193](index=193&type=chunk)[194](index=194&type=chunk)[195](index=195&type=chunk)[196](index=196&type=chunk) - The company evaluates collaboration agreements under ASC 808 to determine if transactions are with customers (ASC 606) or collaborative arrangements, applying consistent recognition and presentation methods[191](index=191&type=chunk) [Results of Operations](index=61&type=section&id=Results%20of%20Operations) Key Financial Results (in thousands) | Metric | Three Months Ended June 30, 2025 | Change (YoY) | Six Months Ended June 30, 2025 | Change (YoY) | | :-------------------------------- | :------------------------------- | :----------- | :------------------------------- | :----------- | | Total revenues | $21,359 | -$936 | $110,371 | +$72,454 | | License and royalty revenue | $18,465 | -$3,381 | $105,514 | +$68,324 | | Service revenue | $2,894 | +$2,445 | $4,857 | +$4,130 | | Research and development expense | $59,500 | +$10,631 | $112,587 | +$8,874 | | General and administrative expense | $19,883 | +$1,028 | $40,230 | +$3,084 | | Interest expense | $(10,993) | -$10,544 | $(19,563) | -$17,141 | | Net loss | $(70,871) | -$17,882 | $(64,788) | +$51,531 | - Q2 2025 revenues decreased slightly due to lower Zolgensma royalties, while YTD 2025 revenues significantly increased due to the Nippon Shinyaku upfront payment[203](index=203&type=chunk)[204](index=204&type=chunk)[208](index=208&type=chunk)[209](index=209&type=chunk) - R&D expenses increased in both periods, driven by higher manufacturing-related expenses, clinical trial costs for ABBV-RGX-314 and RGX-202, and personnel costs[205](index=205&type=chunk)[206](index=206&type=chunk)[210](index=210&type=chunk) - Interest expense saw a substantial increase in both periods, primarily due to royalty monetization liabilities from the 2020 Royalty Purchase Agreement and the new 2025 Royalty Bond[207](index=207&type=chunk)[212](index=212&type=chunk) [Liquidity and Capital Resources](index=62&type=section&id=Liquidity%20and%20Capital%20Resources) - As of June 30, 2025, the company had **$363.6 million** in cash, cash equivalents, and marketable securities, which management believes is sufficient to fund operations for at least the next **12 months**[213](index=213&type=chunk) - Key liquidity sources include **$144.5 million** (net) from the May 2025 Royalty Bond and a **$110.0 million** upfront payment from the Nippon Shinyaku Collaboration Agreement in March 2025[213](index=213&type=chunk)[214](index=214&type=chunk)[216](index=216&type=chunk) - The company expects to incur significant R&D and G&A expenses, requiring additional capital through equity offerings, debt financings, or strategic alliances to fund operations and potential commercialization[217](index=217&type=chunk)[228](index=228&type=chunk) - Future capital requirements are dependent on clinical trial timing and results, regulatory approvals, manufacturing capacity build-out, and the success of licensing agreements[229](index=229&type=chunk)[236](index=236&type=chunk) [Cash Flows](index=64&type=section&id=Cash%20Flows) Cash Flow Summary (in thousands) | Activity | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | | Net cash used in operating activities | $(15,713) | $(100,952) | | Net cash provided by (used in) investing activities | $(95,693) | $12,915 | | Net cash provided by financing activities | $133,438 | $111,280 | | Net increase in cash and cash equivalents and restricted cash | $22,032 | $23,243 | - Net cash used in operating activities decreased by **$85.2 million** year-over-year, primarily due to the **$110.0 million** upfront fee from Nippon Shinyaku, which increased deferred revenue[220](index=220&type=chunk)[221](index=221&type=chunk) - Net cash used in investing activities was **$(95.7) million**, a significant change from **$12.9 million** provided in the prior year, mainly due to increased purchases of marketable debt securities[223](index=223&type=chunk)[224](index=224&type=chunk) - Net cash provided by financing activities increased to **$133.4 million**, driven by proceeds from the 2025 Royalty Bond and warrants, partially offset by royalty monetization repayments[225](index=225&type=chunk) [Off-Balance Sheet Arrangements](index=68&type=section&id=Off-Balance%20Sheet%20Arrangements) - The company did not have any off-balance sheet arrangements during the periods presented[232](index=232&type=chunk) [Item 3. Quantitative and Qualitative Disclosures about Market Risk](index=68&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) This section refers to the company's Annual Report on Form 10-K for detailed market risk disclosures and states that there have been no material changes to its exposure to market risk during the six months ended June 30, 2025 - No material changes to market risk exposure occurred during the six months ended June 30, 2025[233](index=233&type=chunk) [Item 4. Controls and Procedures](index=68&type=section&id=Item%204.%20Controls%20and%20Procedures) This section details the evaluation of the effectiveness of the company's disclosure controls and procedures, concluding they were effective as of June 30, 2025, and states there were no material changes in internal control over financial reporting during the quarter - Management, including the CEO and CFO, concluded that disclosure controls and procedures were effective at a reasonable assurance level as of June 30, 2025[234](index=234&type=chunk)[235](index=235&type=chunk) - There were no changes in internal control over financial reporting during the quarter ended June 30, 2025, that materially affected, or are reasonably likely to materially affect, internal control over financial reporting[237](index=237&type=chunk) PART II—OTHER INFORMATION [Item 1. Legal Proceedings](index=71&type=section&id=Item%201.%20Legal%20Proceedings) This section states that the company is not currently party to any pending legal actions that could reasonably be expected to have a material adverse effect on its business, financial condition, results of operations, or cash flows - No pending legal actions are expected to have a material adverse effect on the company's business or financial condition[241](index=241&type=chunk) [Item 1A. Risk Factors](index=71&type=section&id=Item%201A.%20Risk%20Factors) This section refers to the material risk factors disclosed in the company's Annual Report on Form 10-K for the year ended December 31, 2024, and confirms that there have been no material changes to these risk factors - No material changes to the risk factors previously disclosed in the Annual Report on Form 10-K for the year ended December 31, 2024[242](index=242&type=chunk) [Item 2. Unregistered Sales of Equity Securities, Use of Proceeds and Issuer Purchases of Equity Securities](index=71&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities,%20Use%20of%20Proceeds%20and%20Issuer%20Purchases%20of%20Equity%20Securities) This section indicates that there were no unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities during the reporting period - No unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities occurred[243](index=243&type=chunk) [Item 3. Defaults Upon Senior Securities](index=71&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This section states that there were no defaults upon senior securities during the reporting period - No defaults upon senior securities occurred[244](index=244&type=chunk) [Item 4. Mine Safety Disclosures](index=71&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This section indicates that mine safety disclosures are not applicable to the company - Mine safety disclosures are not applicable[245](index=245&type=chunk) [Item 5. Other Information](index=71&type=section&id=Item%205.%20Other%20Information) This section reports that none of the company's directors or Section 16 reporting officers adopted or terminated any Rule 10b5-1 trading arrangements or non-Rule 10b5-1 trading arrangements during the three months ended June 30, 2025 - No Rule 10b5-1 or non-Rule 10b5-1 trading arrangements were adopted or terminated by directors or Section 16 officers during Q2 2025[246](index=246&type=chunk) [Item 6. Exhibits](index=72&type=section&id=Item%206.%20Exhibits) This section provides a list of exhibits filed with the Quarterly Report on Form 10-Q, including corporate documents, equity incentive plans, the loan agreement for the 2025 Royalty Bond, and certifications - Exhibits include the 2025 Equity Incentive Plan, forms of stock option and restricted stock unit agreements, and the Loan Agreement for the 2025 Royalty Bond[247](index=247&type=chunk) - Certifications from the Chief Executive Officer and Chief Financial Officer are included as Exhibits 31.1, 31.2, and 32.1[247](index=247&type=chunk) [Signatures](index=73&type=section&id=Signatures) This section contains the signatures of the President and Chief Executive Officer, Curran Simpson, and the Chief Financial Officer, Mitchell Chan, certifying the report on behalf of REGENXBIO Inc - The report is signed by Curran Simpson, President and CEO, and Mitchell Chan, CFO, on August 7, 2025[251](index=251&type=chunk)