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)

[Executive Summary & Company Overview](index=1&type=section&id=Executive%20Summary%20%26%20Company%20Overview) [CEO Statement & Q2 2025 Highlights](index=1&type=section&id=CEO%20Statement%20%26%20Q2%202025%20Highlights) REGENXBIO's CEO, Curran M. Simpson, highlighted the acceleration of multiple first or best-in-class gene therapy programs in Q2 2025, including the potential FDA approval of clemidsogene lanparvovec (RGX-121) in November, rapid progress of RGX-202, and the advancement of a late-stage program for diabetic retinopathy - REGENXBIO is accelerating multiple first or best-in-class gene therapies for serious diseases like Duchenne, Hunter syndrome, and retinal diseases[3](index=3&type=chunk) - Potential FDA approval of clemidsogene lanparvovec (RGX-121) is on track for **November 2025**[3](index=3&type=chunk) - RGX-202 is progressing rapidly through pivotal study, and a new late-stage program for diabetic retinopathy is advancing[3](index=3&type=chunk) [About REGENXBIO Inc.](index=4&type=section&id=About%20REGENXBIO%20Inc.) REGENXBIO is a biotechnology company focused on AAV gene therapy, advancing a late-stage pipeline for rare and retinal diseases, including RGX-202 for Duchenne, RGX-121 for MPS II, RGX-111 for MPS I (with Nippon Shinyaku), and surabgene lomparvovec (sura-vec) for wet AMD and diabetic retinopathy (with AbbVie), with their AAV platform utilized in treatments for thousands of patients, including Novartis' ZOLGENSMA® - REGENXBIO is a biotechnology company pioneering AAV gene therapy since its founding in **2009**[23](index=23&type=chunk) - The company is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202, RGX-121, RGX-111, and surabgene lomparvovec[23](index=23&type=chunk) - Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®[23](index=23&type=chunk) [Program Highlights and Milestones](index=1&type=section&id=PROGRAM%20HIGHLIGHTS%20AND%20MILESTONES) [Neuromuscular Disease Programs](index=1&type=section&id=Neuromuscular%20Disease%20Programs) REGENXBIO is making significant progress in its neuromuscular disease pipeline, with RGX-202 for Duchenne muscular dystrophy on track for accelerated pivotal trial enrollment and BLA submission, and clemidsogene lanparvovec (RGX-121) for MPS II nearing potential FDA approval following successful inspections [RGX-202 for Duchenne Muscular Dystrophy](index=1&type=section&id=RGX-202%20in%20Duchenne%20muscular%20dystrophy) RGX-202, a potential best-in-class gene therapy for Duchenne, is on track for topline results in 1H 2026 and BLA submission mid-2026, with pivotal trial enrollment accelerating and expected to complete in October 2025, ahead of previous guidance, and positive Phase I/II data demonstrating a favorable safety profile and robust microdystrophin expression - RGX-202 pivotal trial enrollment is expected to complete in **October 2025**, ahead of previous guidance[5](index=5&type=chunk)[6](index=6&type=chunk) - Topline results for RGX-202 are expected in the **first half of 2026**, with a Biologics License Application (BLA) submission planned for **mid-2026** under the accelerated approval pathway[5](index=5&type=chunk)[10](index=10&type=chunk) - Phase I/II data showed a favorable safety profile with no serious adverse events or signs of liver injury, and all dose level 2 participants exceeded external natural history controls on functional measures[10](index=10&type=chunk) [Clemidsogene lanparvovec (RGX-121) for MPS II (Hunter Syndrome)](index=1&type=section&id=Clemidsogene%20lanparvovec%20(RGX-121)%20for%20MPS%20II%20(Hunter%20Syndrome)) Clemidsogene lanparvovec (RGX-121) is on track to be the first gene therapy and one-time treatment for MPS II, with an FDA Prescription Drug User Fee Act (PDUFA) target action date of November 9, 2025, following successful FDA inspections of manufacturing, quality systems, and clinical data practices with no observations, and potential FDA approval could result in a Priority Review Voucher (PRV) - RGX-121 is on track for potential FDA approval as the first gene therapy for MPS II, with a PDUFA target action date of **November 9, 2025**[5](index=5&type=chunk)[10](index=10&type=chunk) - FDA Pre-license inspection (PLI) of the in-house manufacturing facility and Bioresearch monitoring information (BIMO) inspection of laboratory and clinical data practices were successfully completed with no observations[5](index=5&type=chunk)[10](index=10&type=chunk) - FDA approval could result in the receipt of a Priority Review Voucher (PRV), to which REGENXBIO has full rights[10](index=10&type=chunk) [Retinal Disease Programs](index=1&type=section&id=Retinal%20Disease%20Programs) REGENXBIO's surabgene lomparvovec (sura-vec) program for retinal diseases is advancing, with positive Phase II data supporting a pivotal program for diabetic retinopathy (DR) via suprachoroidal delivery, and ongoing pivotal trials for wet age-related macular degeneration (wet AMD) via subretinal delivery, with topline results expected in 2026 [Surabgene lomparvovec (sura-vec) for Diabetic Retinopathy (Suprachoroidal Delivery)](index=1&type=section&id=Sura-vec%20for%20the%20Treatment%20of%20DR%20(Suprachoroidal%20Delivery)) Sura-vec for diabetic retinopathy (DR) is advancing to a global pivotal program, supported by positive Phase II ALTITUDE® trial data reported in June 2025, demonstrating a durable safety and efficacy profile through two years with a single, in-office injection and no drug-related serious adverse events, with a two-part placebo-controlled Phase IIb/III trial to be initiated with 2-step DRSS improvement as the primary endpoint - Sura-vec using suprachoroidal delivery for diabetic retinopathy is advancing to a global pivotal program, supported by positive Phase II trial data[5](index=5&type=chunk)[10](index=10&type=chunk) - Phase II ALTITUDE® trial data demonstrated durable safety and efficacy in patients with non-proliferative DR through **two years** with a single, in-office injection, with no drug-related serious adverse events[10](index=10&type=chunk) - A two-part placebo-controlled Phase IIb/III trial will be initiated, with the primary endpoint being **2-step DRSS improvement**[10](index=10&type=chunk) [Surabgene lomparvovec (sura-vec) for Wet AMD (Subretinal Delivery)](index=1&type=section&id=Sura-vec%20for%20the%20Treatment%20of%20Wet%20AMD%20(Subretinal%20Delivery)) Enrollment is ongoing in the ATMOSPHERE® and ASCENT™ pivotal trials for the subretinal delivery of sura-vec in patients with wet AMD, with topline results expected in 2026, positioning sura-vec to be the first approved gene therapy for wet AMD - Enrollment is ongoing in the ATMOSPHERE® and ASCENT™ pivotal trials for subretinal delivery of sura-vec in patients with wet AMD[11](index=11&type=chunk) - Topline results for subretinal sura-vec in wet AMD are expected in **2026**[5](index=5&type=chunk)[11](index=11&type=chunk) - Sura-vec is on track to be the first approved gene therapy for wet AMD[5](index=5&type=chunk)[11](index=11&type=chunk) [Surabgene lomparvovec (sura-vec) for Wet AMD (Suprachoroidal Delivery)](index=3&type=section&id=Sura-vec%20for%20the%20Treatment%20of%20Wet%20AMD%20(Suprachoroidal%20Delivery)) The Phase II AAVIATE® trial continues enrolling a new cohort to evaluate sura-vec at dose level 4 (1.5x10e12 GC/eye) for wet AMD using suprachoroidal delivery, with patients in this cohort also receiving short-course prophylactic steroid eye drops - The Phase II AAVIATE® trial is enrolling a new cohort to evaluate suprachoroidal sura-vec at **dose level 4 (1.5x10e12 GC/eye)** for wet AMD[12](index=12&type=chunk) - Patients in this cohort will receive short-course prophylactic steroid eye drops[12](index=12&type=chunk) [Corporate Updates](index=3&type=section&id=CORPORATE%20UPDATES) [Royalty Monetization](index=3&type=section&id=Royalty%20Monetization) In May 2025, REGENXBIO closed a non-dilutive, limited recourse royalty bond agreement of up to $250 million with Healthcare Royalty (HCRx), receiving $150 million at closing and eligible for an additional $100 million in two separate $50 million tranches - Closed a non-dilutive, limited recourse royalty bond agreement of up to **$250 million** with Healthcare Royalty (HCRx) in **May 2025**[13](index=13&type=chunk) - Received **$150 million** at closing and is eligible for an additional **$100 million** in two **$50 million** tranches[13](index=13&type=chunk) [AbbVie Eye Care Collaboration Amendment](index=3&type=section&id=AbbVie%20Eye%20Care%20Collaboration) In August 2025, REGENXBIO and AbbVie amended their collaboration and license agreement, updating the milestone structure for the diabetic retinopathy (DR) program, with AbbVie paying REGENXBIO $100 million upon the first subject dosed in the Phase IIb/III trial and an additional $100 million for a second Phase III clinical trial, and AbbVie independently advancing and funding a new Phase III ACHIEVE trial for wet AMD - Amendment to the collaboration and license agreement with AbbVie includes an updated milestone structure for the DR program[17](index=17&type=chunk) - AbbVie will pay REGENXBIO **$100 million** upon first subject dosed in the Phase IIb/III trial and an additional **$100 million** upon first subject dosed in a second Phase III clinical trial[17](index=17&type=chunk) - AbbVie will independently advance and pay all costs for a new Phase III ACHIEVE trial in wet AMD[17](index=17&type=chunk) [Financial Results (Q2 2025)](index=3&type=section&id=FINANCIAL%20RESULTS) [Cash Position](index=3&type=section&id=Cash%20Position) REGENXBIO's cash, cash equivalents, and marketable securities increased to $363.6 million as of June 30, 2025, from $244.9 million at December 31, 2024, primarily driven by a $110.0 million upfront payment from the Nippon Shinyaku partnership and $144.5 million in net proceeds from the HCRx royalty monetization, partially offset by cash used in operating activities Cash, Cash Equivalents and Marketable Securities | Metric | June 30, 2025 (in millions) | December 31, 2024 (in millions) | Change | | :-------------------------------- | :-------------------------- | :-------------------------- | :----- | | Cash, cash equivalents & marketable securities | $363.6 | $244.9 | +$118.7 | - The increase was primarily attributable to a **$110.0 million** upfront payment from the Nippon Shinyaku partnership (March 2025) and **$144.5 million** in net proceeds from the royalty monetization with HCRx (May 2025)[15](index=15&type=chunk) [Revenues](index=3&type=section&id=Revenues) Total revenues for the three months ended June 30, 2025, decreased slightly to $21.4 million from $22.3 million in the prior year period, primarily due to lower Zolgensma royalties, partially offset by an increase in service revenues, mainly from the Nippon Shinyaku partnership Revenues (Three Months Ended June 30) | Revenue Type | Q2 2025 (in thousands) | Q2 2024 (in thousands) | YoY Change (in thousands) | YoY Change (%) | | :------------------------ | :--------------------- | :--------------------- | :------------------------ | :------------- | | License and royalty revenue | $18,465 | $21,846 | $(3,381) | -15.5% | | Service revenue | $2,894 | $449 | $2,445 | +544.5% | | **Total revenues** | **$21,359** | **$22,295** | **$(936)** | **-4.2%** | - The decrease in total revenues was primarily attributable to Zolgensma royalties, which decreased from **$21.8 million** in Q2 2024 to **$18.4 million** in Q2 2025[16](index=16&type=chunk) - The decrease was partially offset by an increase in service revenues, driven primarily by **$2.7 million** of development service revenue under the Nippon Shinyaku partnership in Q2 2025[16](index=16&type=chunk) [Operating Expenses](index=3&type=section&id=Operating%20Expenses) Operating expenses increased to $84.6 million in Q2 2025 from $78.3 million in Q2 2024, primarily driven by higher research and development expenses related to pivotal trials and increased general and administrative costs [Research and Development Expenses](index=3&type=section&id=Research%20and%20Development%20Expenses) Research and development expenses increased to $59.5 million for Q2 2025, up from $48.9 million for Q2 2024, primarily due to higher manufacturing-related expenses, other clinical supply costs, and clinical trial expenses for the sura-vec and RGX-202 pivotal trials Research and Development Expenses (Three Months Ended June 30) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | YoY Change (in thousands) | YoY Change (%) | | :------------------------ | :--------------------- | :--------------------- | :------------------------ | :------------- | | Research and development | $59,500 | $48,869 | $10,631 | +21.8% | - The increase was primarily attributable to manufacturing-related expenses and other clinical supply costs and clinical trial expenses for sura-vec and RGX-202 pivotal trials[18](index=18&type=chunk) [General and Administrative Expenses](index=4&type=section&id=General%20and%20Administrative%20Expenses) General and administrative expenses increased slightly to $19.9 million for Q2 2025, compared to $18.9 million for Q2 2024, mainly due to increased personnel-related costs and expenses for consulting and professional services General and Administrative Expenses (Three Months Ended June 30) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | YoY Change (in thousands) | YoY Change (%) | | :-------------------------- | :--------------------- | :--------------------- | :------------------------ | :------------- | | General and administrative | $19,883 | $18,855 | $1,028 | +5.5% | - The increase was primarily attributable to personnel-related costs and expenses for consulting and professional services[19](index=19&type=chunk) [Net Loss](index=4&type=section&id=Net%20Loss) REGENXBIO reported a net loss of $70.9 million, or $1.38 basic and diluted net loss per share, for Q2 2025, widening from a net loss of $53.0 million, or $1.05 per share, in Q2 2024, primarily driven by higher operating expenses and a significant increase in interest expense Net Loss (Three Months Ended June 30) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | YoY Change (in thousands) | YoY Change (%) | | :------------------------ | :--------------------- | :--------------------- | :------------------------ | :------------- | | Net loss | $(70,871) | $(52,989) | $(17,882) | +33.7% | | Net loss per share (basic & diluted) | $(1.38) | $(1.05) | $(0.33) | +31.4% | - The increase in net loss was influenced by higher research and development expenses and a significant increase in interest expense[18](index=18&type=chunk)[20](index=20&type=chunk)[30](index=30&type=chunk) [Financial Guidance](index=4&type=section&id=FINANCIAL%20GUIDANCE) [Cash Runway Guidance](index=4&type=section&id=Cash%20Runway%20Guidance) REGENXBIO expects its balance of cash, cash equivalents, and marketable securities, totaling $363.6 million as of June 30, 2025, to fund its operations into early 2027, with this guidance based on current operational plans and excluding the impact of potential material payments from partners or licensees, as well as the potential monetization of a Priority Review Voucher (PRV) - Cash, cash equivalents, and marketable securities of **$363.6 million** as of **June 30, 2025**, are expected to fund operations into **early 2027**[21](index=21&type=chunk) - This cash runway guidance excludes the impact of any material payments from partners or licensees and potential monetization of a PRV[21](index=21&type=chunk) [Financial Statements](index=6&type=section&id=Financial%20Statements) [Consolidated Balance Sheets](index=6&type=section&id=Consolidated%20Balance%20Sheets) As of June 30, 2025, REGENXBIO's total assets increased to $581.0 million from $466.0 million at December 31, 2024, while total liabilities significantly increased to $367.3 million from $206.3 million, primarily driven by a substantial rise in royalty monetization liabilities and deferred revenue, consequently decreasing total stockholders' equity to $213.7 million from $259.7 million Consolidated Balance Sheet Highlights (in thousands) | Metric | June 30, 2025 | December 31, 2024 | Change | | :------------------------- | :-------------- | :---------------- | :----- | | Total assets | $581,027 | $465,989 | +$115,038 | | Total liabilities | $367,348 | $206,338 | +$161,010 | | Total stockholders' equity | $213,679 | $259,651 | $(45,972) | - Royalty monetization liabilities increased significantly from **$25,378 thousand** at December 31, 2024, to **$153,693 thousand** at June 30, 2025[29](index=29&type=chunk) - Deferred revenue (non-current) increased from **$0** to **$23,804 thousand**[29](index=29&type=chunk) [Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) For the six months ended June 30, 2025, total revenues significantly increased to $110.4 million from $37.9 million in the prior year period, primarily due to a substantial rise in license and royalty revenue, and despite an increase in total operating expenses, the loss from operations improved, leading to a net loss of $64.8 million, a reduction from the $116.3 million net loss in the same period of 2024, though interest expense also saw a notable increase Consolidated Statements of Operations Highlights (Six Months Ended June 30, in thousands) | Metric | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | YoY Change (in thousands) | YoY Change (%) | | :-------------------------------- | :--------------------------- | :--------------------------- | :------------------------ | :------------- | | Total revenues | $110,371 | $37,917 | $72,454 | +191.1% | | Total operating expenses | $161,522 | $157,817 | $3,705 | +2.3% | | Loss from operations | $(51,151) | $(119,900) | $68,749 | -57.3% | | Total other income (expense) | $(13,637) | $3,581 | $(17,218) | -480.8% | | Net loss | $(64,788) | $(116,319) | $51,531 | -44.3% | | Net loss per share (basic & diluted) | $(1.26) | $(2.41) | $(1.15) | -47.7% | - The substantial increase in total revenues for the six-month period was primarily driven by license and royalty revenue, which rose from **$37,190 thousand** to **$105,514 thousand**[30](index=30&type=chunk) - Interest expense significantly increased from **$2,422 thousand** in the first six months of 2024 to **$19,563 thousand** in the same period of 2025[30](index=30&type=chunk) [Additional Information](index=4&type=section&id=Additional%20Information) [Conference Call Details](index=4&type=section&id=CONFERENCE%20CALL) REGENXBIO hosted a conference call and webcast on August 7, 2025, at 8:00 a.m. ET to discuss the second quarter 2025 financial results and operational highlights, with details for webcast registration and dial-in for the Q&A session provided - REGENXBIO hosted a conference call and webcast at **8:00 a.m. ET** on **August 7, 2025**[5](index=5&type=chunk)[22](index=22&type=chunk) - Listeners could register for the webcast via a provided link, and analysts could access the live call by dialing specific numbers and entering a passcode[22](index=22&type=chunk) [Forward-Looking Statements](index=4&type=section&id=FORWARD-LOOKING%20STATEMENTS) This press release contains forward-looking statements regarding REGENXBIO's future operations, clinical trials, costs, and cash flow, based on current expectations and assumptions, which are subject to various risks and uncertainties, including the timing and success of clinical trials, regulatory approvals, and market acceptance, as detailed in the company's Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the SEC - The press release includes forward-looking statements concerning REGENXBIO's future operations, clinical trials, costs, and cash flow[24](index=24&type=chunk)[25](index=25&type=chunk) - These statements are subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials, regulatory approval, and market acceptance[25](index=25&type=chunk) - Readers are cautioned not to rely too heavily on these statements, and REGENXBIO does not undertake any obligation to update or revise them, except as required by law[25](index=25&type=chunk) [Contacts](index=5&type=section&id=CONTACTS) Contact information for REGENXBIO's Corporate Communications (Dana Cormack) and Investor Relations (George E. MacDougall) is provided for inquiries - Contact information for Corporate Communications (Dana Cormack) and Investor Relations (George E. MacDougall) is provided[27](index=27&type=chunk)

Core Insights - REGENXBIO Inc. reported strong operational momentum in advancing its gene therapy programs, particularly with RGX-121 and RGX-202, aimed at treating serious diseases such as Duchenne muscular dystrophy and Hunter syndrome [2][4][5] Financial Results - Cash, cash equivalents, and marketable securities increased to $363.6 million as of June 30, 2025, from $244.9 million at the end of 2024, primarily due to a $110 million upfront payment from Nippon Shinyaku and $144.5 million from royalty monetization [16][21] - Revenues for the second quarter of 2025 were $21.4 million, a slight decrease from $22.3 million in the same period of 2024, mainly due to a drop in Zolgensma royalties [17] - Research and development expenses rose to $59.5 million in Q2 2025 from $48.9 million in Q2 2024, driven by manufacturing and clinical trial costs [18] - General and administrative expenses increased to $19.9 million in Q2 2025 from $18.9 million in Q2 2024, attributed to personnel and consulting costs [19] - The net loss for Q2 2025 was $70.9 million, compared to a net loss of $53.0 million in Q2 2024, reflecting increased operational costs [20] Program Highlights - RGX-202 is positioned as a potential best-in-class gene therapy for Duchenne muscular dystrophy, with pivotal trial enrollment expected to complete by October 2025 [3][5] - Clemidsogene lanparvovec (RGX-121) is on track for potential FDA approval in November 2025, with successful completion of FDA inspections [4][13] - Surabgene lomparvovec (sura-vec) is advancing towards pivotal trials for diabetic retinopathy and wet AMD, with topline results expected in 2026 [7][9] Corporate Updates - In May 2025, REGENXBIO closed a $250 million royalty bond agreement, receiving $150 million upfront [11] - An amendment to the collaboration agreement with AbbVie was executed in August 2025, which includes milestone payments for the diabetic retinopathy program [14] Financial Guidance - The company expects its cash position to fund operations into early 2027, excluding potential payments from partners or monetization of a Priority Review Voucher [21]

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 August 7, 2025, at 8:00 a.m. ET to discuss its financial results for Q2 2025 and operational highlights [1] - Listeners can register for the webcast and analysts can participate in the Q&A session by dialing specific numbers [2] - A replay of the webcast will be available on the company's investor website approximately two hours after the call [2]

Core Insights - REGENXBIO Inc. announced preclinical results showing that a microdystrophin gene therapy construct with the C-terminal (CT) domain provides improved functional benefits for patients with Duchenne Muscular Dystrophy compared to a construct without the CT domain [1][4][5] - RGX-202 is the only investigational microdystrophin gene therapy candidate that includes the CT domain, making it closest to naturally occurring dystrophin [2][8] Group 1: Research Findings - The preclinical study published in Molecular Therapy Methods and Clinical Development demonstrated that the microdystrophin with the CT domain was maintained at higher levels in transduced muscles and effectively recruited the dystrophin-associated protein complex to the muscle membrane [4][5] - The incorporation of the CT domain enhances the microdystrophin design, allowing for higher accumulation levels in muscle and potentially improving functional benefits [5][7] Group 2: Clinical Trial Insights - Interim results from the Phase I/II AFFINITY DUCHENNE trial indicated that RGX-202 showed consistent evidence of positively changing the disease trajectory in patients with Duchenne and had a favorable safety profile [5][6] - REGENXBIO is currently enrolling participants in the pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial and plans to submit a Biologics License Application (BLA) via the accelerated approval pathway in mid-2026 [6][9] Group 3: Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, with a late-stage pipeline that includes RGX-202 for Duchenne, among other treatments for rare diseases [11] - The company has pioneered AAV gene therapy since its founding in 2009 and has treated thousands of patients with its AAV platform [11]

Company Overview - Regenxbio is a clinical-stage biopharmaceutical company that specializes in gene therapies, founded in 2008 [1] - The company's core technology involves the delivery of rescue genes using AAV (adeno-associated virus) payloads, which is increasingly being utilized in approved drugs [1] Industry Insights - The healthcare sector is complex, and financial professionals and investors require scientific and clinical expertise to navigate it effectively [1] - There is a growing emphasis on bridging the gap between advanced scientific research and financial strategy to uncover hidden value and assess risks accurately in life sciences [1]

Core Insights - RegenXBio Inc. has released new interim data from the Phase 1/2 AFFINITY DUCHENNE trial, showcasing positive functional, safety, and biomarker data for RGX-202, a gene therapy for Duchenne muscular dystrophy [1][2]. Group 1: Trial Data and Results - The trial involved five patients aged six to twelve years who received RGX-202, demonstrating consistent benefits in functional data among dose level 2 participants at 9 and 12 months post-treatment [1]. - At 9 months, RGX-202 participants showed improved function, exceeding external controls on all measures, with an average improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from baseline and 4.8 points compared to natural history [3]. - At 12 months, RGX-202 participants continued to demonstrate improved performance on timed function tests and NSAA, with an average improvement of 4.5 points from baseline and 6.8 points compared to natural history [4]. Group 2: Future Plans and Market Context - RegenXBio plans to share topline data for RGX-202 in the first half of 2026 and aims to submit a Biologics License Application (BLA) under the accelerated approval pathway by mid-2026, with commercial readiness activities underway for a potential launch in 2027 [5]. - The competitive landscape includes Sarepta Therapeutics, which recently reported a patient death following treatment with its approved gene therapy Elevidys, leading to a therapeutic clinical hold on several studies in the EU [6].

RGX-202 Therapeutic Approach - RGX-202 is designed to improve function and preserve muscle health, delivered by proprietary NAV® AAV8 vector[24] - The trial is designed to demonstrate meaningful change in disease trajectory in pursuit of broad label, with positive interim efficacy and safety outcomes reported in Phase I/II[25] - RGX-202 has product purity levels of more than 80% full capsids due to industry-leading manufacturing[21] - A proactive, short-course immune modulation regimen is designed to counter safety concerns common with high-dose AAV gene therapy and to improve safety outcomes[22] AFFINITY DUCHENNE Phase I/II Trial Results - RGX-202 was well-tolerated in 13 patients across both dose levels with no SAEs or AESIs[62] - Biomarker data support consistent robust expression, transduction, and sarcolemmal localization of RGX-202 microdystrophin[62] - At 9 months, Dose Level 2 participants (n=5) demonstrated a mean change in NSAA of 4.8 compared to natural history external controls (n=65)[44] - At 12 months, Dose Level 2 participants (n=4) demonstrated a mean change in NSAA of 6.8 compared to natural history external controls (n=26)[47] - Dose Level 2 timed task velocity changes exceeded MCID benchmarks at 12 months[50] - Caregivers reported improved function in the home and community as measured by key dimensions of the PODCI at 12 months compared to expected decline in natural history[54] Future Development - The company plans for a mid-2026 BLA submission using accelerated approval pathway and a potential FDA approval in 1H 2027[18]