[PART I—FINANCIAL INFORMATION](index=5&type=section&id=PART%20I%E2%80%94FINANCIAL%20INFORMATION) [Item 1. Financial Statements (Unaudited)](index=5&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Presents REGENXBIO Inc.'s unaudited consolidated financial statements, including balance sheets, income statements, equity, and cash flows, with notes for Q1 2025 [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) Presents the company's financial position, including assets, liabilities, and equity, as of March 31, 2025, and December 31, 2024 | Metric | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | Change (in thousands) | | :--------------------------------- | :----------------------------- | :------------------------------- | :-------------------- | | Cash and cash equivalents | $130,104 | $57,526 | +$72,578 | | Marketable securities | $137,765 | $177,161 | -$39,396 | | Total current assets | $313,237 | $278,001 | +$35,236 | | Total assets | $490,929 | $465,989 | +$24,940 | | Deferred revenue (current) | $13,588 | $115 | +$13,473 | | Deferred revenue (non-current) | $26,297 | — | +$26,297 | | Total liabilities | $216,732 | $206,338 | +$10,394 | | Total stockholders' equity | $274,197 | $259,651 | +$14,546 | - Total assets increased by **$24.9 million**, primarily driven by a significant increase in cash and cash equivalents, partially offset by a decrease in marketable securities[17](index=17&type=chunk) - Deferred revenue saw a substantial increase from **$115 thousand to $13.6 million** (current) and from zero to **$26.3 million** (non-current), reflecting new collaboration agreements[17](index=17&type=chunk) [Consolidated Statements of Operations and Comprehensive Income (Loss)](index=6&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Income%20(Loss)) Details the company's revenues, expenses, and net income (loss) for the three months ended March 31, 2025 and 2024 | Metric | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | Change (in thousands) | | :------------------------------------- | :--------------------------------------------- | :--------------------------------------------- | :-------------------- | | License and royalty revenue | $87,049 | $15,344 | +$71,705 | | Service revenue | $1,963 | $278 | +$1,685 | | Total revenues | $89,012 | $15,622 | +$73,390 | | Total operating expenses | $76,885 | $79,485 | -$2,600 | | Income (loss) from operations | $12,127 | $(63,863) | +$75,990 | | Net income (loss) | $6,083 | $(63,330) | +$69,413 | | Basic net income (loss) per share | $0.12 | $(1.38) | +$1.50 | | Diluted net income (loss) per share | $0.12 | $(1.38) | +$1.50 | - Total revenues significantly increased by **$73.4 million**, primarily driven by a **$71.7 million** increase in license and royalty revenue and a **$1.7 million** increase in service revenue[18](index=18&type=chunk)[178](index=178&type=chunk)[179](index=179&type=chunk) - The company reported a net income of **$6.1 million** for Q1 2025, a substantial improvement from a net loss of **$63.3 million** in Q1 2024, resulting in positive basic and diluted EPS of **$0.12**[18](index=18&type=chunk) [Consolidated Statements of Stockholders' Equity](index=7&type=section&id=Consolidated%20Statements%20of%20Stockholders'%20Equity) Outlines changes in stockholders' equity, including net income and capital contributions, for the period ending March 31, 2025 | Metric | December 31, 2024 (in thousands) | March 31, 2025 (in thousands) | Change (in thousands) | | :--------------------------------- | :----------------------------- | :---------------------------- | :-------------------- | | Total Stockholders' Equity | $259,651 | $274,197 | +$14,546 | | Additional Paid-in Capital | $1,192,536 | $1,201,020 | +$8,484 | | Accumulated Deficit | $(932,149) | $(926,066) | +$6,083 | | Common Stock Shares Outstanding | 49,549 | 50,117 | +568 | - Total stockholders' equity increased by **$14.5 million**, primarily due to net income of **$6.1 million** and an increase in additional paid-in capital from stock-based compensation expense and employee stock purchase plan activities[21](index=21&type=chunk) [Consolidated Statements of Cash Flows](index=8&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) Reports cash inflows and outflows from operating, investing, and financing activities for the three months ended March 31, 2025 and 2024 | Cash Flow Activity | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | Change (in thousands) | | :--------------------------------- | :--------------------------------------------- | :--------------------------------------------- | :-------------------- | | Operating activities | $33,629 | $(55,455) | +$89,084 | | Investing activities | $44,743 | $13,183 | +$31,560 | | Financing activities | $(5,794) | $120,725 | -$126,519 | | Net increase in cash and cash equivalents and restricted cash | $72,578 | $78,453 | -$5,875 | | Cash and cash equivalents and restricted cash, End of period | $132,134 | $115,005 | +$17,129 | - Net cash provided by operating activities significantly improved to **$33.6 million** in Q1 2025 from a net cash used of **$55.5 million** in Q1 2024, largely due to the **$110.0 million** upfront fee from the Nippon Shinyaku collaboration[24](index=24&type=chunk)[187](index=187&type=chunk)[188](index=188&type=chunk) - Net cash provided by investing activities increased to **$44.7 million**, driven by maturities of marketable debt securities exceeding purchases[24](index=24&type=chunk)[191](index=191&type=chunk) - Net cash used in financing activities was **$5.8 million** in Q1 2025, a decrease from **$120.7 million** provided in Q1 2024, which included proceeds from a public offering[24](index=24&type=chunk)[193](index=193&type=chunk)[194](index=194&type=chunk) [Notes to Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) Provides detailed explanations of significant accounting policies, financial statement line items, and other disclosures [1. Nature of Business & Liquidity](index=9&type=section&id=Note%201.%20Nature%20of%20Business) Describes REGENXBIO's business, cumulative losses, and assessment of liquidity to fund operations for the next 12 months - 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[27](index=27&type=chunk) - The company has incurred cumulative losses of **$926.1 million** since inception and relies on successful product development, approvals, and additional capital raises to achieve profitability[28](index=28&type=chunk) - As of March 31, 2025, the company had **$272.7 million** in cash, cash equivalents, and marketable securities, which management believes is sufficient to fund operations for at least the next 12 months[28](index=28&type=chunk) [2. Summary of Significant Accounting Policies](index=9&type=section&id=Note%202.%20Summary%20of%20Significant%20Accounting%20Policies) Outlines the key accounting principles and estimates used in preparing the unaudited consolidated financial statements - The unaudited consolidated financial statements are prepared in conformity with GAAP, reflecting all normal and recurring adjustments, and should be read in conjunction with the annual audited statements[29](index=29&type=chunk)[30](index=30&type=chunk) - Estimates are used in areas such as revenue recognition, accrued R&D expenses, stock-based compensation, and fair value measurements, with actual results potentially differing materially[32](index=32&type=chunk)[33](index=33&type=chunk) - Revenue recognition follows ASC 606, involving a five-step model to identify contracts, performance obligations, transaction price, allocation, and recognition upon satisfaction of obligations[41](index=41&type=chunk)[42](index=42&type=chunk) [3. Marketable Securities](index=18&type=section&id=Note%203.%20Marketable%20Securities) Details the composition and fair value of the company's available-for-sale debt securities and their maturities | Type of Security | March 31, 2025 Fair Value (in thousands) | December 31, 2024 Fair Value (in thousands) | | :----------------------------- | :------------------------------------- | :---------------------------------------- | | U.S. government and agency securities | $34,722 | $44,215 | | Certificates of deposit | $733 | $1,462 | | Corporate bonds | $107,168 | $141,663 | | **Total Marketable Securities** | **$142,623** | **$187,340** | - The company's marketable securities, consisting solely of available-for-sale debt securities, decreased from **$187.3 million** at December 31, 2024, to **$142.6 million** at March 31, 2025[60](index=60&type=chunk) - As of March 31, 2025, no available-for-sale debt securities had remaining maturities greater than two years, and the company did not identify any credit losses[60](index=60&type=chunk)[62](index=62&type=chunk) [4. Fair Value Measurements](index=20&type=section&id=Note%204.%20Fair%20Value%20Measurements) Explains the fair value hierarchy and classification of cash equivalents and marketable securities | Financial Instrument | March 31, 2025 Fair Value (in thousands) | December 31, 2024 Fair Value (in thousands) | | :------------------- | :------------------------------------- | :---------------------------------------- | | Money market mutual funds | $103,496 | $43,895 | | U.S. government and agency securities (cash equivalents) | — | $2,498 | | U.S. government and agency securities (marketable) | $34,722 | $44,215 | | Certificates of deposit | $733 | $1,462 | | Corporate bonds | $107,168 | $141,663 | | **Total Cash Equivalents and Marketable Securities** | **$246,119** | **$233,733** | - All cash equivalents and marketable securities are classified as Level 2 in the fair value hierarchy, indicating valuations based on observable inputs other than quoted prices in active markets[63](index=63&type=chunk)[64](index=64&type=chunk) [5. Property and Equipment, Net](index=21&type=section&id=Note%205.%20Property%20and%20Equipment%2C%20Net) Presents the net book value of property and equipment, detailing categories and accumulated depreciation | Category | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | | :----------------------------- | :---------------------------- | :------------------------------- | | Laboratory and manufacturing equipment | $77,505 | $77,141 | | Computer equipment and software | $4,612 | $4,244 | | Furniture and fixtures | $7,032 | $7,031 | | Leasehold improvements | $101,517 | $101,465 | | Total property and equipment | $190,666 | $189,881 | | Accumulated depreciation and amortization | $(76,169) | $(72,292) | | **Property and equipment, net** | **$114,497** | **$117,589** | - Net property and equipment decreased from **$117.6 million** to **$114.5 million**, primarily due to accumulated depreciation and amortization[66](index=66&type=chunk) [6. Leases](index=21&type=section&id=Note%206.%20Leases) Discusses lease arrangements, including a sublease of office facilities and related impairment and income - In March 2024, the company subleased its New York office facilities, resulting in an impairment of **$2.1 million** on long-lived assets (right-of-use assets and property and equipment) in Q1 2024[66](index=66&type=chunk)[68](index=68&type=chunk)[70](index=70&type=chunk) - The sublease term commenced in April 2024 and expires in April 2027, with the company recognizing **$0.1 million** in sublease income during Q1 2025[68](index=68&type=chunk) - No impairment losses on long-lived assets were recorded during the three months ended March 31, 2025[70](index=70&type=chunk) [7. Liability Related to Sale of Future Royalties](index=22&type=section&id=Note%207.%20Liability%20Related%20to%20Sale%20of%20Future%20Royalties) Details the royalty purchase agreement for Zolgensma, including the Cap Amount, liability changes, and effective interest rate - The company has a royalty purchase agreement with HCR for Zolgensma royalties, with a Cap Amount of **$300.0 million** applicable from November 8, 2024, until the Novartis License termination[71](index=71&type=chunk)[72](index=72&type=chunk) - The liability related to the sale of future royalties decreased from **$59.7 million** at December 31, 2024, to **$53.7 million** at March 31, 2025, after Zolgensma royalties paid to HCR and recognized interest expense[76](index=76&type=chunk) - The estimated effective interest rate for the royalty purchase agreement was **66.9%** as of March 31, 2025, and **16.1%** over the life of the agreement, reflecting the amount and timing of royalty payments[75](index=75&type=chunk) [8. Commitments and Contingencies](index=24&type=section&id=Note%208.%20Commitments%20and%20Contingencies) Outlines license agreements, royalty obligations, and potential disputes, including the GSK License - The company has a license agreement with GlaxoSmithKline LLC (GSK) for its NAV Technology Platform, with royalty obligations now assigned to Penn[77](index=77&type=chunk)[78](index=78&type=chunk) - Expenses related to the GSK License were **$2.9 million** in Q1 2025, including **$2.6 million** in Zolgensma royalties[79](index=79&type=chunk) - A potential dispute with GSK regarding sublicense fees is ongoing, but the company does not believe a loss is probable and no reasonable range of loss is estimable[80](index=80&type=chunk) [9. Capitalization](index=25&type=section&id=Note%209.%20Capitalization) Reports on equity offerings, pre-funded warrants, and the At-the-Market Offering Program for common stock - In March 2024, the company completed a public offering, raising **$131.1 million** net proceeds from the sale of common stock and pre-funded warrants[81](index=81&type=chunk) - As of March 31, 2025, **1,324,740** pre-funded warrants remained outstanding, with no exercises during Q1 2025[83](index=83&type=chunk) - The company established an At-the-Market (ATM) Offering Program in December 2024, allowing for the sale of up to **$150.0 million** in common stock, though no shares were sold under this program as of March 31, 2025[84](index=84&type=chunk) [10. License and Collaboration Agreements](index=25&type=section&id=Note%2010.%20License%20and%20Collaboration%20Agreements) Summarizes key license and collaboration agreements, detailing revenue recognition and potential milestone payments [10.1 License and Collaboration Revenues](index=25&type=section&id=License%20and%20Collaboration%20Revenues) Details the breakdown of license and royalty revenue and service revenue from various collaboration agreements | Revenue Source | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | Change (in thousands) | | :----------------------------- | :--------------------------------------------- | :--------------------------------------------- | :-------------------- | | Zolgensma royalties | $16,993 | $15,214 | +$1,779 | | Nippon Shinyaku licenses | $69,979 | — | +$69,979 | | Other license and royalty revenue | $77 | $130 | -$53 | | **Total license and royalty revenue** | **$87,049** | **$15,344** | **+$71,705** | | Nippon Shinyaku services | $1,774 | — | +$1,774 | | Other service revenue | $189 | $278 | -$89 | | **Total service revenue** | **$1,963** | **$278** | **+$1,685** | | **Total revenues** | **$89,012** | **$15,622** | **+$73,390** | - Total revenues increased significantly by **$73.4 million**, primarily driven by **$70.0 million** in non-recurring license revenue and **$1.8 million** in development service revenue from the Nippon Shinyaku collaboration in Q1 2025[86](index=86&type=chunk)[178](index=178&type=chunk)[179](index=179&type=chunk) - Unachieved milestones from license and collaboration agreements could result in aggregate payments of up to **$2.20 billion**, including **$549.2 million** for clinical trials, **$121.3 million** for regulatory approvals, and **$1.53 billion** for sales targets[86](index=86&type=chunk) [10.2 Accounts Receivable, Contract Assets and Deferred Revenue](index=26&type=section&id=Accounts%20Receivable%2C%20Contract%20Assets%20and%20Deferred%20Revenue) Presents balances for accounts receivable, contract assets, and deferred revenue, primarily from collaboration agreements | Metric | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | | :----------------------------- | :---------------------------- | :------------------------------- | | Accounts receivable, net | $19,400 | $20,947 | | Contract assets | $44 | $239 | | Deferred revenue | $39,885 | $115 | - Deferred revenue increased substantially to **$39.9 million**, primarily due to consideration received from the Nippon Shinyaku collaboration for performance obligations not yet satisfied[87](index=87&type=chunk)[88](index=88&type=chunk) - The aggregate transaction price allocated to unsatisfied or partially satisfied performance obligations is **$47.6 million**, mostly from the Nippon Shinyaku agreement, expected to be satisfied over approximately five years[88](index=88&type=chunk) [10.3 Zolgensma License with Novartis Gene Therapies](index=27&type=section&id=Zolgensma%20License%20with%20Novartis%20Gene%20Therapies) Reports Zolgensma royalty revenue and related interest income from the Novartis license agreement | Revenue Type | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | | :------------------- | :--------------------------------------------- | :--------------------------------------------- | | Zolgensma royalties | $16,993 | $15,214 | | Interest income from licensing | $10 | $7 | - Zolgensma royalties increased by **$1.8 million** to **$17.0 million** in Q1 2025 compared to Q1 2024[91](index=91&type=chunk) - As of March 31, 2025, **$14.4 million** of Zolgensma royalties receivable was expected to be paid to HCR under the Royalty Purchase Agreement[91](index=91&type=chunk) [10.4 AbbVie Collaboration and License Agreement](index=27&type=section&id=AbbVie%20Collaboration%20and%20License%20Agreement) Outlines the collaboration with AbbVie for ABBV-RGX-314, including development responsibilities and cost reimbursements - The collaboration with AbbVie for ABBV-RGX-314 involves co-exclusive development and commercialization rights in the U.S. and exclusive rights outside the U.S.[92](index=92&type=chunk)[93](index=93&type=chunk) - AbbVie is responsible for the majority of ABBV-RGX-314 development expenses since January 1, 2023, and will lead global commercialization[93](index=93&type=chunk)[94](index=94&type=chunk) | Cost Reimbursement | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | | :--------------------------------- | :--------------------------------------------- | :--------------------------------------------- | | Research and development expense | $(14,681) | $(24,977) | | General and administrative expense | $597 | $402 | | **Total net cost reimbursement to (from) AbbVie** | **$(14,084)** | **$(24,575)** | [10.5 Nippon Shinyaku Collaboration and License Agreement](index=31&type=section&id=Nippon%20Shinyaku%20Collaboration%20and%20License%20Agreement) Describes the collaboration with Nippon Shinyaku for RGX-121 and RGX-111, including upfront fees and potential milestones - In January 2025, the company entered a collaboration with Nippon Shinyaku for RGX-121 and RGX-111, granting licenses for development and commercialization in the U.S. and certain Asian countries[99](index=99&type=chunk)[100](index=100&type=chunk) - The agreement included an upfront fee of **$110.0 million** and potential milestone payments of up to **$700.0 million**, plus double-digit royalties on net sales[101](index=101&type=chunk) | Revenue Type | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | | :------------------- | :--------------------------------------------- | :--------------------------------------------- | | License and royalty revenue | $69,979 | — | | Service revenue | $1,774 | — | | **Total revenues** | **$71,753** | **—** | [11. Stock-based Compensation](index=34&type=section&id=Note%2011.%20Stock-based%20Compensation) Details stock-based compensation expense by award type and unrecognized compensation for future periods | Award Type | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | | :-------------------------- | :--------------------------------------------- | :--------------------------------------------- | | Stock options | $4,707 | $5,978 | | Restricted stock units | $3,668 | $3,447 | | Employee stock purchase plan | $162 | $177 | | **Total stock-based compensation expense** | **$8,537** | **$9,602** | - Total stock-based compensation expense decreased by **$1.1 million** to **$8.5 million** in Q1 2025, primarily due to a decrease in stock option expense[109](index=109&type=chunk) - As of March 31, 2025, unrecognized stock-based compensation expense totaled **$65.8 million**, expected to be recognized over a weighted-average period of **2.5 years**[109](index=109&type=chunk) [12. Income Taxes](index=35&type=section&id=Note%2012.%20Income%20Taxes) Discusses the company's income tax position, including the valuation allowance for net deferred tax assets - The company has provided a full valuation allowance for its net deferred tax assets as of March 31, 2025, and December 31, 2024, due to a history of operating losses[115](index=115&type=chunk) [13. Net Income (Loss) Per Share](index=35&type=section&id=Note%2013.%20Net%20Income%20(Loss)%20Per%20Share) Presents basic and diluted net income (loss) per share calculations and weighted-average common shares outstanding | Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :------------------------------------- | :-------------------------------- | :-------------------------------- | | Net income (loss) | $6,083 (in thousands) | $(63,330) (in thousands) | | Basic net income (loss) per share | $0.12 | $(1.38) | | Diluted net income (loss) per share | $0.12 | $(1.38) | | Weighted-average common shares outstanding (basic) | 51,362 (in thousands) | 45,733 (in thousands) | | Weighted-average diluted common shares | 51,434 (in thousands) | 45,733 (in thousands) | - Basic and diluted net income per share were **$0.12** for Q1 2025, a significant improvement from a net loss per share of **$(1.38)** in Q1 2024[116](index=116&type=chunk) - Common stock equivalents were excluded from diluted net loss per share calculations in periods of net losses as their effect would be anti-dilutive[117](index=117&type=chunk) [14. Segment Information](index=36&type=section&id=Note%2014.%20Segment%20Information) Confirms the company operates as a single segment, with the CEO as CODM reviewing consolidated financial metrics - The company operates as a single operating segment, focusing on the development and commercialization of gene therapies, with its CEO serving as the chief operating decision maker (CODM)[118](index=118&type=chunk) - The CODM reviews consolidated net income (loss) and consolidated cash, cash equivalents, and marketable securities to assess performance and allocate resources[118](index=118&type=chunk) | Metric | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | | :--------------------------------- | :--------------------------------------------- | :--------------------------------------------- | | Revenues | $89,012 | $15,622 | | Cost of license and royalty revenues | $3,436 | $4,283 | | Total research and development expense | $53,087 | $54,844 | | Total general and administrative expense | $20,347 | $18,291 | | Net income (loss) | $6,083 | $(63,330) | [15. Supplemental Disclosures](index=37&type=section&id=Note%2015.%20Supplemental%20Disclosures) Provides additional details on other current assets, accrued expenses, and non-cash investing and financing activities | Other Current Assets Category | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | | :---------------------------- | :---------------------------- | :------------------------------- | | Net cost reimbursement due from AbbVie | $14,082 | $11,304 | | Accrued interest on investments | $965 | $1,094 | | Other | $1,035 | $1,376 | | **Total Other Current Assets** | **$16,082** | **$13,774** | | Accrued Expenses Category | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | | :-------------------------- | :---------------------------- | :------------------------------- | | Accrued personnel costs | $8,534 | $17,607 | | Accrued external R&D expenses | $6,332 | $8,998 | | Accrued sublicense fees and royalties | $6,183 | $8,658 | | **Total Accrued Expenses and Other Current Liabilities** | **$23,597** | **$38,070** | - Non-cash investing and financing activities included **$0.2 million** in purchases of property and equipment in accounts payable and accrued expenses as of March 31, 2025[125](index=125&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=38&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Analyzes REGENXBIO's Q1 2025 financial condition and operations, covering strategy, pipeline, revenues, expenses, and liquidity [2.1 Overview](index=38&type=section&id=Overview) Introduces REGENXBIO as a clinical-stage biotechnology company focused on gene therapy using its NAV Technology Platform - REGENXBIO is a clinical-stage biotechnology company focused on gene therapy, aiming to improve lives by delivering functional genes to address genetic defects[129](index=129&type=chunk) - The company's investigational gene therapies utilize its proprietary adeno-associated virus (AAV) gene therapy delivery platform (NAV Technology Platform) for one-time treatments[129](index=129&type=chunk)[130](index=130&type=chunk) [2.2 Overview of Product Candidates](index=38&type=section&id=Overview%20of%20Product%20Candidates) Summarizes the development status of key product candidates, including ABBV-RGX-314, RGX-202, RGX-121, and RGX-111 - ABBV-RGX-314 is being developed in collaboration with AbbVie for chronic retinal conditions (wet AMD, DR, DME), with pivotal trials (ATMOSPHERE, ASCENT) ongoing for subretinal delivery and Phase II trials (AAVIATE, ALTITUDE) for suprachoroidal delivery[130](index=130&type=chunk)[131](index=131&type=chunk)[132](index=132&type=chunk)[133](index=133&type=chunk)[134](index=134&type=chunk) - RGX-202 is an investigational AAV therapeutic for Duchenne muscular dystrophy, with the AFFINITY DUCHENNE trial beyond **50%** enrolled and topline data expected in H1 2026[135](index=135&type=chunk)[136](index=136&type=chunk) - RGX-121, for MPS II, achieved its primary endpoint in the CAMPSIITE trial, with a BLA submitted to the FDA in March 2025, and potential approval expected in H2 2025[137](index=137&type=chunk) - Development of RGX-111 for MPS I was halted in November 2023 due to strategic pipeline prioritization, but efforts to continue development with Nippon Shinyaku are ongoing[138](index=138&type=chunk) [2.3 Collaboration and License Agreement with AbbVie](index=41&type=section&id=Collaboration%20and%20License%20Agreement%20with%20AbbVie) Details the collaboration with AbbVie for ABBV-RGX-314, including development, commercialization, and financial terms - The AbbVie Collaboration Agreement, effective November 2021, involves joint development and commercialization of ABBV-RGX-314, with AbbVie leading global commercialization and sharing U.S. net profits/losses[139](index=139&type=chunk) - The company received an upfront fee of **$370.0 million** and is eligible for up to **$1.38 billion** in development and sales-based milestones, plus tiered royalties on ex-U.S. net sales[139](index=139&type=chunk) [2.4 Collaboration and License Agreement with Nippon Shinyaku](index=41&type=section&id=Collaboration%20and%20License%20Agreement%20with%20Nippon%20Shinyaku) Outlines the collaboration with Nippon Shinyaku for RGX-121 and RGX-111, including upfront payments and milestones - The Nippon Shinyaku Collaboration Agreement, effective March 2025, covers the development and commercialization of RGX-121 and RGX-111 in the U.S. and certain Asian countries[140](index=140&type=chunk) - The company received an upfront payment of **$110.0 million** and is eligible for up to **$700.0 million** in milestones, plus double-digit royalties on net sales[140](index=140&type=chunk) - REGENXBIO recognized **$71.8 million** in revenue under this agreement in Q1 2025 and retains rights to any priority review vouchers[141](index=141&type=chunk) [2.5 Overview of Our NAV Technology Platform](index=41&type=section&id=Overview%20of%20Our%20NAV%20Technology%20Platform) Describes the NAV Technology Platform, its licensing to other companies, and its application in commercial and clinical products - The NAV Technology Platform is selectively licensed to other biotechnology and pharmaceutical companies, expanding the gene therapy pipeline and generating additional revenue opportunities[142](index=142&type=chunk) - As of March 31, 2025, the NAV Technology Platform was applied in one commercial product, Zolgensma, and in the preclinical and clinical development of other licensed products[142](index=142&type=chunk) [2.6 Financial Overview](index=41&type=section&id=Financial%20Overview) Provides a general overview of revenue sources, future revenue dependency, and primary operating expense components - Revenues primarily stem from licensing the NAV Technology Platform and other intellectual property rights, with no revenues yet from commercial sales of the company's own products[143](index=143&type=chunk) - Future revenues are highly dependent on the successful development and commercialization of licensed products, which is uncertain and may fluctuate significantly[145](index=145&type=chunk) - Operating expenses consist mainly of cost of license and royalty revenues, research and development, and general and administrative expenses, with personnel costs being a significant component[147](index=147&type=chunk) [2.7 Critical Accounting Policies and Estimates](index=44&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) Highlights critical accounting policies, including revenue recognition under ASC 606 and treatment of variable consideration - Revenue recognition is a critical accounting policy, following ASC 606's five-step model, which involves identifying contracts, performance obligations, transaction price, allocation, and recognition[163](index=163&type=chunk)[164](index=164&type=chunk) - The company evaluates collaboration agreements under ASC 808 and ASC 606, determining if transactions are with customers or collaborative arrangements[167](index=167&type=chunk)[168](index=168&type=chunk) - Variable consideration, such as development and sales-based milestones and royalties, is included in the transaction price only when it is probable that a significant revenue reversal will not occur[170](index=170&type=chunk)[173](index=173&type=chunk)[174](index=174&type=chunk) [2.8 Results of Operations](index=48&type=section&id=Results%20of%20Operations) Analyzes the company's revenues, operating expenses, and net income (loss) for the three months ended March 31, 2025 and 2024 | Metric | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | Change (in thousands) | | :--------------------------------- | :--------------------------------------------- | :--------------------------------------------- | :-------------------- | | Total revenues | $89,012 | $15,622 | +$73,390 | | Cost of license and royalty revenues | $3,436 | $4,283 | -$847 | | Research and development | $53,087 | $54,844 | -$1,757 | | General and administrative | $20,347 | $18,291 | +$2,056 | | Income (loss) from operations | $12,127 | $(63,863) | +$75,990 | | Net income (loss) | $6,083 | $(63,330) | +$69,413 | - Total revenues increased by **$73.4 million**, primarily due to **$70.0 million** in non-recurring license revenue from the Nippon Shinyaku collaboration[178](index=178&type=chunk) - Research and development expenses decreased by **$1.8 million**, mainly due to a **$4.1 million** decrease in clinical trial activities and a **$1.4 million** decrease in preclinical activities, partially offset by a **$3.2 million** increase in manufacturing-related expenses[180](index=180&type=chunk) - General and administrative expenses increased by **$2.1 million**, driven by higher personnel-related costs, professional services, and corporate overhead[181](index=181&type=chunk) [2.9 Liquidity and Capital Resources](index=48&type=section&id=Liquidity%20and%20Capital%20Resources) Assesses the company's cash position, cash flow activities, and future capital requirements and funding strategies - As of March 31, 2025, the company had **$272.7 million** in cash, cash equivalents, and marketable securities, deemed sufficient to fund operations for at least the next 12 months[182](index=182&type=chunk) - Net cash provided by operating activities increased by **$89.1 million** to **$33.6 million** in Q1 2025, largely due to the **$110.0 million** upfront fee from the Nippon Shinyaku collaboration[187](index=187&type=chunk)[188](index=188&type=chunk) - Future capital requirements are significant and depend on factors such as clinical trial timing and results, regulatory approvals, commercialization costs, and the ability to secure additional financing through equity offerings, debt, or collaborations[196](index=196&type=chunk)[197](index=197&type=chunk)[199](index=199&type=chunk) [Item 3. Quantitative and Qualitative Disclosures about Market Risk](index=55&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) Reports no material changes to market risk exposure for Q1 2025 compared to the prior annual report - No material changes to market risk exposure occurred during the three months ended March 31, 2025[202](index=202&type=chunk) [Item 4. Controls and Procedures](index=55&type=section&id=Item%204.%20Controls%20and%20Procedures) Disclosure controls and procedures were effective as of March 31, 2025, with no material changes in internal control - Disclosure controls and procedures were evaluated and deemed effective at a reasonable assurance level as of March 31, 2025[203](index=203&type=chunk)[204](index=204&type=chunk) - No material changes in internal control over financial reporting occurred during the quarter ended March 31, 2025[205](index=205&type=chunk) [PART II—OTHER INFORMATION](index=56&type=section&id=PART%20II%E2%80%94OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=56&type=section&id=Item%201.%20Legal%20Proceedings) No pending legal actions are expected to materially adversely affect the company's business or financial condition - No pending legal actions are expected to have a material adverse effect on the company's business or financial condition[209](index=209&type=chunk) [Item 1A. Risk Factors](index=56&type=section&id=Item%201A.%20Risk%20Factors) No material changes to risk factors since the Annual Report on Form 10-K for December 31, 2024 - No material changes to risk factors since the Annual Report on Form 10-K for December 31, 2024[210](index=210&type=chunk) [Item 2. Unregistered Sales of Equity Securities, Use of Proceeds and Issuer Purchases of Equity Securities](index=56&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%2C%20Use%20of%20Proceeds%20and%20Issuer%20Purchases%20of%20Equity%20Securities) Reports no unregistered sales of equity securities, use of proceeds, or issuer purchases during the period - No unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities occurred[211](index=211&type=chunk) [Item 3. Defaults Upon Senior Securities](index=56&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon senior securities during the period - No defaults upon senior securities were reported[212](index=212&type=chunk) [Item 4. Mine Safety Disclosures](index=56&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) Mine Safety Disclosures are not applicable - Mine Safety Disclosures are not applicable[213](index=213&type=chunk) [Item 5. Other Information](index=56&type=section&id=Item%205.%20Other%20Information) Details Rule 10b5-1 trading plans adopted or terminated by Section 16 officers and directors during Q1 2025 | Name | Title | Action | Date Adopted | Expiration Date | Plan Provides for | Aggregate of Securities to be Purchased/Sold | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | Curran Simpson | President and Chief Executive Officer | Adoption | 3/28/2025 | 3/1/2026 | Sale | 36,169 | [Item 6. Exhibits](index=57&type=section&id=Item%206.%20Exhibits) Lists all exhibits filed with Form 10-Q, including collaboration agreements, CEO/CFO certifications, and XBRL financials - Key exhibits include the Collaboration and License Agreement with Nippon Shinyaku Co., Ltd. (Exhibit 10.1†)[215](index=215&type=chunk) - Certifications of the Chief Executive Officer and Chief Financial Officer (Exhibits 31.1, 31.2, 32.1) are included as required by the Sarbanes-Oxley Act[215](index=215&type=chunk) - The financial statements and cover page are formatted in Inline XBRL (Exhibit 101, 104)[215](index=215&type=chunk) [Signatures](index=58&type=section&id=Signatures) The Quarterly Report on Form 10-Q was signed by the President/CEO and CFO on May 12, 2025 - The report was signed by Curran Simpson, President and CEO, and Mitchell Chan, CFO, on May 12, 2025[220](index=220&type=chunk)

Financial Performance - Revenues for Q1 2025 were $89.0 million, a significant increase from $15.6 million in Q1 2024, primarily due to $71.8 million of license and service revenue from the collaboration with Nippon Shinyaku[13] - Net income for Q1 2025 was $6.1 million, or $0.12 per share, compared to a net loss of $63.3 million, or $1.38 per share, in Q1 2024[17] - Total revenues for Q1 2025 reached $89,012,000, a significant increase of 469% compared to $15,622,000 in Q1 2024[26] - License and royalty revenue surged to $87,049,000, up from $15,344,000 in the same period last year, marking a growth of 467%[26] - Basic net income per share improved to $0.12 in Q1 2025, compared to a loss of $1.38 per share in Q1 2024[26] - The company reported a comprehensive income of $6,062,000 for Q1 2025, compared to a comprehensive loss of $62,130,000 in Q1 2024[26] Cash and Assets - Cash, cash equivalents, and marketable securities totaled $272.7 million as of March 31, 2025, up from $244.9 million at the end of 2024, mainly due to a $110 million upfront payment from the Nippon Shinyaku partnership[12] - Total current assets increased to $313,237,000 as of March 31, 2025, compared to $278,001,000 at the end of 2024, reflecting a growth of 12.7%[25] - Total assets rose to $490,929,000, up from $465,989,000 at the end of 2024, indicating a growth of 5.3%[25] - Total liabilities increased to $216,732,000 from $206,338,000, representing a rise of 5.7%[25] Expenses - Research and development expenses for Q1 2025 were $53.1 million, a decrease from $54.8 million in Q1 2024, primarily due to lower clinical trial expenses[15] - General and administrative expenses increased to $20.3 million in Q1 2025 from $18.3 million in Q1 2024, mainly due to higher personnel-related costs[16] - Total operating expenses decreased to $76,885,000 in Q1 2025 from $79,485,000 in Q1 2024, a reduction of 3.9%[26] Clinical Development - The pivotal trial for RGX-202 in Duchenne muscular dystrophy is more than half enrolled, with completion expected in 2025 and a Biologics License Application (BLA) submission planned for mid-2026[5] - REGENXBIO expects to initiate commercial supply manufacturing for RGX-202 in Q3 2025, with all production sourced in the U.S.[6] - The FDA is expected to accept the BLA for clemidsogene lanparvovec (RGX-121) in May 2025, with potential approval in the second half of 2025[14] Partnerships - The partnership with Nippon Shinyaku includes an upfront payment of $110 million and potential additional payments of up to $700 million upon achieving certain milestones[11] - REGENXBIO's cash runway is expected to fund operations into the second half of 2026, excluding potential milestone payments and monetization of a Priority Review Voucher[18]

Core Insights - REGENXBIO Inc. reported significant advancements in its gene therapy pipeline, with multiple late-stage assets showing differentiation from standard treatments, positioning the company for potential first- or best-in-class therapies for rare and retinal diseases [2] Program Highlights and Milestones - RGX-202 is a novel microdystrophin gene therapy for Duchenne muscular dystrophy, utilizing the NAV® AAV8 vector, and is the only construct including the C-Terminal domain found in natural dystrophin [3] - Clemidsogene lanparvovec (RGX-121) is being developed as a first-in-class treatment for MPS II (Hunter syndrome) in partnership with Nippon Shinyaku [4] - Surabgene lomparvovec (sura-vec, ABBV-RGX-314) is on track to be the first-in-class treatment for wet age-related macular degeneration (wet AMD), with pivotal trial enrollment ongoing and completion expected in 2025 [5][8] Financial Results - As of March 31, 2025, REGENXBIO's cash, cash equivalents, and marketable securities totaled $272.7 million, an increase from $244.9 million at the end of 2024, primarily due to a $110 million upfront payment from the Nippon Shinyaku partnership [11] - Revenues for the first quarter of 2025 were $89.0 million, a significant increase from $15.6 million in the same period of 2024, largely driven by $71.8 million in license and service revenue from the collaboration with Nippon Shinyaku [12] - Research and development expenses decreased to $53.1 million in Q1 2025 from $54.8 million in Q1 2024, while general and administrative expenses rose to $20.3 million from $18.3 million [13][14] - The company reported a net income of $6.1 million, or $0.12 per share, compared to a net loss of $63.3 million, or $1.38 per share, in the prior year [15] Corporate Updates - REGENXBIO's partnership with Nippon Shinyaku, finalized in March 2025, includes an upfront payment of $110 million and potential additional payments of up to $700 million based on milestone achievements [10] - The company is preparing for a Biologics License Application (BLA) submission for clemidsogene lanparvovec (RGX-121) expected in May 2025, with potential approval in the second half of 2025 [6]

Core Insights - REGENXBIO Inc. is showcasing its advancements in gene therapy at the ASGCT 28th Annual Meeting, emphasizing its comprehensive capabilities in research, clinical development, and manufacturing [1][5] Presentations Overview - The company will present late-stage clinical trial data for RGX-121, aimed at treating MPS II, and RGX-202 for Duchenne muscular dystrophy, along with preclinical research on RGX-202's novel construct and manufacturing process [2][5] - Key oral presentations include: - Development of a commercial manufacturing process for RGX-202 [3] - Investigational gene therapy RGX-121 for neuronopathic MPS II [3] - Interim clinical data for RGX-202 [3] Poster Presentations - Various poster presentations will cover topics such as: - The impact of sample collection conditions on AAV endotoxin testing [4] - AAV-expressed microdystrophin's effects in a mouse model of Duchenne muscular dystrophy [4] - Development of in vitro methods for analyzing TLR9 stimulation by AAV vector genomes [4] - Characterization of oversized AAV vectors with high genome integrity [4] - Blood-brain barrier crossing AAV vectors targeting the transferrin receptor [4] Company Background - REGENXBIO, founded in 2009, focuses on gene therapy, particularly AAV gene therapy, with a late-stage pipeline targeting rare and retinal diseases [5] - The company has treated thousands of patients using its AAV platform, including those receiving Novartis' ZOLGENSMA® [5]

Core Viewpoint - Regenxbio (RGNX) is anticipated to report a significant year-over-year increase in earnings and revenues for the quarter ended March 2025, which could influence its stock price depending on the actual results compared to estimates [1][2]. Earnings Expectations - The upcoming earnings report is expected to be released on May 12, 2025, with a consensus estimate of $0.41 per share, reflecting a year-over-year increase of +129.7% [3]. - Revenues are projected to reach $109.98 million, representing a substantial increase of 604.1% from the same quarter last year [3]. Estimate Revisions - Over the last 30 days, the consensus EPS estimate has been revised down by 6.26%, indicating a reassessment by analysts [4]. - The Most Accurate Estimate for Regenxbio is higher than the Zacks Consensus Estimate, resulting in a positive Earnings ESP of +57.77% [10][11]. Earnings Surprise Prediction - The Zacks Earnings ESP model suggests that a positive Earnings ESP reading is a strong predictor of an earnings beat, especially when combined with a Zacks Rank of 1 (Strong Buy), 2 (Buy), or 3 (Hold) [8]. - Regenxbio currently holds a Zacks Rank of 3, indicating a likelihood of beating the consensus EPS estimate [11]. Historical Performance - In the last reported quarter, Regenxbio was expected to post a loss of $1.27 per share but actually reported a loss of $1.01, resulting in a surprise of +20.47% [12]. - Over the past four quarters, the company has surpassed consensus EPS estimates two times [13]. Industry Context - In the Zacks Medical - Biomedical and Genetics industry, Sangamo Therapeutics (SGMO) is expected to report a loss of $0.11 per share for the same quarter, with a year-over-year change of +59.3% [17]. - Sangamo's revenue is projected to be $9.17 million, up 1810.4% from the previous year [17]. - Despite a recent downward revision of 23.8% in consensus EPS estimates, Sangamo has an Earnings ESP of 27.27%, but it holds a Zacks Rank of 4 (Sell), making predictions of an earnings beat uncertain [18].

Company Overview - REGENXBIO Inc. is a biotechnology company focused on gene therapy with a mission to improve lives through its curative potential [3] - The company has been a pioneer in AAV gene therapy since its founding in 2009 [3] - REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for Duchenne, RGX-121 for MPS II, and RGX-111 for MPS I, in partnership with Nippon Shinyaku [3] - The company is also collaborating with AbbVie on surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy [3] - Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® [3] Upcoming Events - REGENXBIO will host a conference call on May 12, 2025, at 4:30 p.m. ET to discuss its financial results for Q1 2025 and recent operational highlights [1] - Listeners can register for the webcast and analysts can participate in the Q&A session through provided links [2] - A replay of the webcast will be available on the company's investor website approximately two hours after the call [2]

Group 1 - The Fortune Teller is a team of two analysts with over 30 years of combined market experience [1] - One of the principles has a background in asset management and is a formerly licensed investment advisor [1] - The team holds degrees in Accounting & Economics and Computer Sciences, as well as MBAs, which inform their stock selections [1] Group 2 - The team leads the investing group Wheel of Fortune, sharing actionable trading ideas across various asset classes, sectors, and industries [1] - The service aims to provide a comprehensive resource for investment and portfolio ideas while educating subscribers [1] - Features include the Funds Macro Portfolio for less active investors, the Single Macro Portfolio for more active investors, educational content, and a live chat room for discussions [1]

Core Insights - REGENXBIO Inc. reported positive interim data from the Phase I/II AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, presented at the 2025 MDA Clinical & Scientific Conference [1][2] Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, with RGX-202 being the only next-generation gene therapy in pivotal trials for Duchenne muscular dystrophy [2][9] - The company aims to improve lives through the curative potential of gene therapy and has a late-stage pipeline for various rare diseases [12] Clinical Trial Updates - The AFFINITY DUCHENNE trial is progressing towards completing enrollment in 2025, with a Biologics License Application (BLA) submission anticipated by mid-2026 [2][7] - New biomarker data from patients aged 1-3 showed microdystrophin expression levels of 122.3% compared to control in a 3-year-old patient, indicating robust expression across all ages [4][7] - RGX-202 demonstrated the highest reported vector genome copies (4.9-55.4) among approved or investigational gene therapies [6] Safety and Efficacy - As of February 21, 2025, RGX-202 was well tolerated with no serious adverse events (SAEs) reported, and common drug-related adverse events included nausea, vomiting, and fatigue [7][8] - The treatment has shown functional improvements exceeding external natural history controls, with patients demonstrating stable or improved function on the North Star Ambulatory Assessment (NSAA) [8] Future Prospects - The company plans to share additional Phase I/II functional data in the first half of 2025, reinforcing the potential of RGX-202 to alter the course of Duchenne muscular dystrophy [2][8]

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

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