PART I—FINANCIAL INFORMATION [Item 1. Financial Statements](index=6&type=section&id=Item%201.%20Financial%20Statements) The unaudited consolidated financial statements for Q1 2024 show a reduced net loss, strengthened balance sheet from a public offering, and decreased cash burn from operations [Consolidated Balance Sheets](index=6&type=section&id=Consolidated%20Balance%20Sheets) Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2024 | December 31, 2023 | | :--- | :--- | :--- | | Cash and cash equivalents | $112,975 | $34,522 | | Total current assets | $395,418 | $334,971 | | Total assets | $629,215 | $573,970 | | Total current liabilities | $110,179 | $130,272 | | Total liabilities | $238,549 | $262,228 | | Total stockholders' equity | $390,666 | $311,742 | - The increase in cash and stockholders' equity was primarily driven by a public offering of common stock and pre-funded warrants in March 2024[22](index=22&type=chunk)[69](index=69&type=chunk) [Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Consolidated Statements of Operations Highlights (in thousands, except per share data) | Metric | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Total revenues | $15,622 | $19,138 | | Research and development | $54,844 | $58,516 | | General and administrative | $18,291 | $22,634 | | Loss from operations | ($63,863) | ($66,157) | | Net loss | ($63,330) | ($66,676) | | Net loss per share, basic and diluted | ($1.38) | ($1.53) | - Revenues decreased primarily due to non-recurring development milestone revenue recognized in Q1 2023 and a **$0.9 million decrease** in Zolgensma royalty revenues[137](index=137&type=chunk) [Consolidated Statements of Stockholders' Equity](index=8&type=section&id=Consolidated%20Statements%20of%20Stockholders%27%20Equity) - Total stockholders' equity increased from **$311.7 million** at year-end 2023 to **$390.7 million** at March 31, 2024, primarily driven by **$131.1 million** in net proceeds from a public offering, offset by a net loss of **$63.3 million** for the quarter[22](index=22&type=chunk)[69](index=69&type=chunk) [Consolidated Statements of Cash Flows](index=9&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) Consolidated Cash Flow Summary (in thousands) | Cash Flow Activity | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Net cash used in operating activities | ($55,455) | ($80,918) | | Net cash provided by investing activities | $13,183 | $63,094 | | Net cash provided by (used in) financing activities | $120,725 | ($9,037) | | Net increase (decrease) in cash | $78,453 | ($26,861) | - The significant cash provided by financing activities in Q1 2024 was due to **$131.6 million** in net proceeds from a public offering of common stock and pre-funded warrants[25](index=25&type=chunk)[149](index=149&type=chunk) [Notes to Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) - In March 2024, the company entered into an agreement to sublease its New York office, resulting in an impairment charge of **$2.1 million** on long-lived assets, including the right-of-use asset and property and equipment[57](index=57&type=chunk)[59](index=59&type=chunk)[61](index=61&type=chunk) - The liability related to the sale of future Zolgensma royalties to HCR decreased to **$83.3 million** as of March 31, 2024, from **$94.1 million** at year-end 2023[68](index=68&type=chunk) - In March 2024, the company completed a public offering of common stock and pre-funded warrants, receiving aggregate net proceeds of **$131.1 million**[69](index=69&type=chunk) - Zolgensma royalties for Q1 2024 were **$15.2 million**, a decrease from **$16.1 million** in Q1 2023[74](index=74&type=chunk)[81](index=81&type=chunk) - Under the AbbVie collaboration, the company recorded a net cost reimbursement of **$24.6 million** as a reduction to operating expenses in Q1 2024, compared to **$18.3 million** in Q1 2023[91](index=91&type=chunk) - The company implemented a corporate restructuring in November 2023, which included a **15% workforce reduction**, with the remaining restructuring liability at **$0.3 million** as of March 31, 2024[101](index=101&type=chunk)[102](index=102&type=chunk)[103](index=103&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=39&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses clinical program progress, analyzes decreased revenues and operating expenses, and highlights improved liquidity from a recent public offering [Overview of Business and Product Candidates](index=39&type=section&id=Overview%20of%20Business%20and%20Product%20Candidates) - **ABBV-RGX-314 (for wet AMD & DR):** - Subretinal delivery pivotal trials (ATMOSPHERE® and ASCENT™) are on track to support global regulatory submissions in the first half of 2026 - Suprachoroidal delivery (AAVIATE® trial) data updates are expected in Q3 2024 - The first pivotal trial for DR (ALTITUDE®) is expected to initiate in the first half of 2025[111](index=111&type=chunk)[112](index=112&type=chunk)[113](index=113&type=chunk) - **RGX-202 (for Duchenne):** - Dose level 2 has been selected as the pivotal dose - Initiation of the pivotal trial is expected in late Q3 to early Q4 2024, with plans to use microdystrophin expression as a surrogate endpoint for accelerated approval[114](index=114&type=chunk)[115](index=115&type=chunk) - **RGX-121 (for MPS II):** - The pivotal phase of the CAMPSIITE® trial achieved its primary endpoint - The company plans to submit a Biologics License Application (BLA) in 2024, using a surrogate endpoint for accelerated approval[115](index=115&type=chunk) [Results of Operations](index=46&type=section&id=Results%20of%20Operations) Comparison of Operating Results (in thousands) | Line Item | Q1 2024 | Q1 2023 | Change | | :--- | :--- | :--- | :--- | | License and royalty revenue | $15,622 | $19,138 | ($3,516) | | Research and development | $54,844 | $58,516 | ($3,672) | | General and administrative | $18,291 | $22,634 | ($4,343) | | Net loss | ($63,330) | ($66,676) | $3,346 | - The decrease in R&D expenses was primarily due to a **$7.1 million reduction** in manufacturing costs, a **$3.3 million decrease** in personnel costs (related to the 2023 restructuring), and a **$2.0 million drop** in lab facility costs, partially offset by a **$9.2 million increase** in clinical trial activity costs[138](index=138&type=chunk) - General and administrative expenses decreased mainly due to a **$2.8 million reduction** in professional services and a **$1.3 million decrease** in personnel-related costs, also driven by the 2023 restructuring[139](index=139&type=chunk) [Liquidity and Capital Resources](index=48&type=section&id=Liquidity%20and%20Capital%20Resources) - As of March 31, 2024, the company had **$380.5 million** in cash, cash equivalents, and marketable securities[139](index=139&type=chunk) - Management believes current capital is sufficient to fund operating expenses and capital expenditure requirements for at least the next 12 months[139](index=139&type=chunk) - In March 2024, a public offering of common stock and pre-funded warrants generated aggregate net proceeds of **$131.1 million**[140](index=140&type=chunk) - Net cash used in operating activities decreased to **$55.5 million** in Q1 2024 from **$80.9 million** in Q1 2023, largely due to lower operating expenses and increased cost reimbursement from AbbVie[143](index=143&type=chunk)[144](index=144&type=chunk) [Item 3. Quantitative and Qualitative Disclosures about Market Risk](index=53&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) No material changes to the company's market risk exposure were reported for Q1 2024 compared to the 2023 Annual Report - There have been no material changes to the company's exposure to market risk during the first quarter of 2024[158](index=158&type=chunk) [Item 4. Controls and Procedures](index=53&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls and procedures were effective as of March 31, 2024, with no material changes to internal control over financial reporting - The CEO and CFO concluded that as of March 31, 2024, the company's disclosure controls and procedures were effective at a reasonable assurance level[160](index=160&type=chunk) - There were no changes in internal control over financial reporting during the quarter that materially affected, or are reasonably likely to materially affect, the company's internal control over financial reporting[161](index=161&type=chunk) PART II—OTHER INFORMATION [Item 1. Legal Proceedings](index=55&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any legal proceedings expected to materially adversely affect its operations or financial condition - The company is not currently party to any legal proceedings expected to have a material adverse effect on its business[165](index=165&type=chunk) [Item 1A. Risk Factors](index=55&type=section&id=Item%201A.%20Risk%20Factors) No material changes to the company's risk factors were reported compared to the 2023 Annual Report on Form 10-K - No material changes have occurred to the risk factors disclosed in the company's 2023 Annual Report on Form 10-K[166](index=166&type=chunk) [Item 2. Unregistered Sales of Equity Securities, Use of Proceeds and Issuer Purchases of Equity Securities](index=55&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%2C%20Use%20of%20Proceeds%20and%20Issuer%20Purchases%20of%20Equity%20Securities) No unregistered sales of equity securities, use of proceeds, or issuer purchases were reported for the period - None reported for the quarter[167](index=167&type=chunk) [Item 5. Other Information](index=55&type=section&id=Item%205.%20Other%20Information) Several Section 16 officers and directors adopted Rule 10b5-1 trading plans during the first quarter of 2024 Adoption of Rule 10b5-1 Trading Plans in Q1 2024 | Name | Title | Action | Date Adopted | | :--- | :--- | :--- | :--- | | Olivier Danos | Executive Vice President, Chief Scientific Officer | Adoption | 3/15/2024 | | Curran Simpson | Executive Vice President, Chief Operating Officer | Adoption | 3/15/2024 | | A.N. "Jerry" Karabelas | Director | Adoption | 3/25/2024 | | Vittal Vasista | Executive Vice President, Chief Financial Officer | Adoption | 3/27/2024 | [Item 6. Exhibits](index=56&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with Form 10-Q, including CEO/CFO certifications and Inline XBRL financial statements - Exhibits filed include CEO/CFO certifications (31.1, 31.2, 32.1) and Inline XBRL data (101)[172](index=172&type=chunk)

REGENXBIO Reports First Quarter 2024 Financial Results and Recent Operational Highlights ROCKVILLE, Md., May 8, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the first quarter ending March 31, 2024. Recent operational highlights, including acceleration of the prioritized pipeline supports meaningful value generation from the Company's strong portfolio of AAV Therapeutics. "Exciting program and data updates continue in 2024 and demonstrate remarkable progress on how ...

Financial Data and Key Metrics Changes - REGENXBIO ended the year with cash, cash equivalents, and marketable securities totaling $314 million, a decrease from $565 million as of December 31, 2022, primarily due to cash used for operating activities in 2023 [136][45] - R&D expenses were $232 million for the year ended December 31, 2023, compared to $242 million in 2022, mainly due to clinical trial and manufacturing expenses for ABBV-RGX-314 [9] Business Line Data and Key Metrics Changes - RGX-314 is in pivotal trials for the treatment of wet AMD and diabetic retinopathy, with expectations for global regulatory submission by the end of 2025 through the first half of 2026 [121][10] - RGX-202 demonstrated strong microdystrophin expression, with the third patient at dose level 1 showing 83.4% expression compared to normal control at three months [124] Market Data and Key Metrics Changes - The anti-VEGF market opportunity is expected to grow significantly as the population ages, with RGX-314 being evaluated in two ongoing pivotal trials expected to enroll a total of 1,200 patients across the U.S., Europe, and Japan [6] Company Strategy and Development Direction - The company is focusing on a pipeline prioritization plan to concentrate resources on large commercial opportunities, particularly in gene therapies for wet AMD, diabetic retinopathy, Duchenne, and MPS II [4][5] - The operational goals with AbbVie include completing pivotal trials for RGX-314 and supporting the initiation of new trials for RGX-202 [10][46] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's positioning for success in 2024 and beyond, citing recent milestones and upcoming data updates as key drivers [21][32] - The company is optimistic about the potential of RGX-202 to address a significant unmet need in Duchenne, with plans to initiate a pivotal trial this year [46][127] Other Important Information - The company plans to share new data updates for RGX-202 at the Muscular Dystrophy Association Conference and for RGX-314 in Q2 and mid-2024 [5][48] - Management highlighted the importance of long-term vision health and the differentiation of RGX-314 in the competitive landscape [56][54] Q&A Session Summary Question: What competitive moat does RGX-314 have if approved? - Management emphasized the durability of treatment outcomes and the partnership with AbbVie as key competitive advantages, along with a large database of patient data [27][29] Question: What are the expectations for initial strength and functional data from RGX-202? - Management indicated that they expect to share strength and motor function data for both dose levels throughout 2024, with a focus on microdystrophin expression as a key indicator [33][34] Question: Will longer-term data from the suprachoroidal RGX-314 program be required before starting Phase 3? - Management stated that while longer-term data is valuable, they are on track to initiate Phase 3 based on current data and endpoints [63][64] Question: How does the company view the regulatory landscape for RGX-121? - Management expressed confidence in the use of biomarkers for accelerated approval and highlighted ongoing discussions with the FDA regarding the use of well-characterized biochemical markers [104][106]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-37553 Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 1 ...

Financial Performance - REGENXBIO reported revenues of $22.2 million for Q4 2023 and $90.2 million for the full year, down from $31.3 million and $112.7 million in the same periods of 2022, respectively[10]. - The company had a net loss of $62.9 million, or $1.43 per share, for Q4 2023, compared to a net loss of $59.9 million, or $1.38 per share, in Q4 2022; the full year net loss was $263.5 million, or $6.02 per share, compared to $280.3 million, or $6.50 per share, in 2022[15]. - Total revenues for the year ended December 31, 2023, were $90,242 thousand, down 20% from $112,724 thousand in 2022[24]. - License and royalty revenue decreased from $31,345 thousand in Q4 2022 to $22,213 thousand in Q4 2023, representing a decline of 29%[24]. - Net loss for the year ended December 31, 2023, was $263,494 thousand, compared to a net loss of $280,321 thousand in 2022, showing an improvement of 6%[24]. - Comprehensive loss for the year ended December 31, 2023, was $252,522 thousand, compared to $293,153 thousand in 2022, indicating a reduction of 13.8%[24]. - Basic and diluted net loss per share improved from $(6.50) in 2022 to $(6.02) in 2023[24]. - The company reported a total operating expense of $358,370 thousand for the year ended December 31, 2023, down from $375,600 thousand in 2022, a decrease of 4.6%[24]. Cash and Assets - Cash, cash equivalents, and marketable securities totaled $314.1 million as of December 31, 2023, down from $565.2 million a year earlier, with the current balance expected to fund operations into the second half of 2025[10][16]. - Total assets decreased from $833,268 thousand in December 2022 to $573,970 thousand in December 2023, a decline of approximately 31.2%[23]. - Total liabilities decreased from $317,073 thousand in December 2022 to $262,228 thousand in December 2023, a reduction of approximately 17.3%[23]. - Stockholders' equity fell from $516,195 thousand in December 2022 to $311,742 thousand in December 2023, a decline of about 39.5%[23]. Research and Development - Research and development expenses for Q4 2023 were $55.7 million, down from $62.5 million in Q4 2022; full year R&D expenses were $232.3 million, compared to $242.5 million in 2022[13]. - Research and development expenses for the year ended December 31, 2023, were $232,266 thousand, a decrease of 4.2% compared to $242,453 thousand in 2022[24]. Clinical Trials and Future Plans - ABBV-RGX-314 is being evaluated in nine ongoing clinical trials for wet AMD and diabetic retinopathy, with pivotal trials expected to support regulatory submissions in late 2025 to early 2026[4][6]. - In the AAVIATE trial, patients treated with ABBV-RGX-314 showed an 80% reduction in annualized injection rate, with 50% remaining injection-free at six months[6]. - REGENXBIO plans to file a Biologics License Application (BLA) for RGX-202 in 2024, using microdystrophin expression as a surrogate endpoint[11]. - The company expects to share updates on Duchenne and retinal disease programs starting in March 2024, with pivotal dose determination for RGX-202 anticipated in mid-2024[5][11]. - The company anticipates significant data readouts and milestones across all programs in 2024, marking a potential turning point in its journey[3]. General and Administrative Expenses - General and administrative expenses for Q4 2023 were $19.1 million, compared to $21.2 million in Q4 2022; full year G&A expenses were $88.5 million, up from $85.3 million in 2022[14].

REGENXBIO Inc. (NASDAQ:RGNX) Q3 2023 Earnings Conference Call November 8, 2023 4:30 PM ET Company Participants Patrick Christmas - Chief Legal Officer Ken Mills - Chief Executive Officer Steve Pakola - Chief Medical Officer Vit Vasista - Chief Financial Officer Conference Call Participants Gena Wang - Barclays Dane Leone - Raymond James Alec Stranahan - Bank of America Lisa Walter - RBC Capital Markets Brian Skorney - Baird Mani Foroohar - Leerink Partners Operator Good day, and thank you for standing by. W ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___ to ___ Commission File Number 001-37553 REGENXBIO Inc. (Exact Name of Registrant as Specified in its Charter) Delaware 47-1851754 (S ...

REGENXBIO Inc. (NASDAQ:RGNX) Q2 2023 Earnings Conference Call August 2, 2023 4:30 PM ET Company Participants Patrick Christmas - Chief Legal Officer Ken Mills - Chief Executive Officer Steve Pakola - Chief Medical Officer Vit Vasista - Chief Financial Officer Conference Call Participants Dane Leone - RJF Alec Stranahan - Bank of America Ellie Merle - UBS Lisa Walter - RBC Andreas Argyrides - Wedbush Securities Caroline Palomeque - Berenberg Capital Markets Operator Good day, and thank you for standing by. W ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___ to ___ Commission File Number 001-37553 REGENXBIO Inc. (Exact Name of Registrant as Specified in its Charter) Washington, D.C. 20549 FORM 10-Q (Mark One) (State or other jurisdictio ...

Financial Data and Key Metrics Changes - REGENXBIO ended Q1 2023 with cash, cash equivalents, and marketable securities totaling $474 million, down from $565 million as of December 31, 2022, primarily due to cash used for operating activities [73] - R&D expenses for Q1 2023 were $59 million, an increase from $56 million in Q1 2022, attributed to higher personnel costs and laboratory expenses related to the manufacturing innovation center [74] Business Line Data and Key Metrics Changes - The company is advancing its 5x'25 strategy, aiming to move five AAV therapeutics into pivotal or commercial stages by 2025 [22][25] - The RGX-314 program has dosed over 600 patients across seven trials, demonstrating good tolerability and potential as a one-time treatment for major retinal vascular diseases [29][30] Market Data and Key Metrics Changes - The global market for diabetic retinopathy (DR) is estimated to reach $15 billion in the next decade, with RGX-314 positioned as a potential one-time treatment option [47] - The anti-VEGF market is calculated to be over $14 billion globally, with RGX-314 targeting over 30 million patients with high unmet needs [66] Company Strategy and Development Direction - REGENXBIO is focused on expanding its global reach through collaboration with AbbVie, particularly in the eye care sector, to bring RGX-314 to patients worldwide [44][62] - The company is leveraging its NAVXpress platform for manufacturing, which is operational and designed to meet global regulatory standards [64] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory pathway for RGX-314, anticipating submissions to the FDA and EMA in late 2025 to early 2026 [62] - The company is optimistic about the potential of RGX-314 to address the high unmet need in retinal diseases, particularly given the limitations of current anti-VEGF treatments [63] Other Important Information - The company announced the transfer of INDs for ongoing clinical trials to AbbVie, marking a significant step in their collaboration [23][26] - REGENXBIO's manufacturing innovation center is set to produce commercial-scale GMP material for the RGX-314 clinical program [45] Q&A Session Summary Question: What is the impact of the increased enrollment in ATMOSPHERE and ASCENT on regulatory submissions? - Management indicated that the increased sample size enhances power for both primary and secondary endpoints, which is crucial for regulatory submissions [80][81] Question: Was there an alteration in the agreement with AbbVie regarding regulatory responsibilities? - Management clarified that the transition of regulatory responsibilities was part of the original collaboration agreement and is now being implemented as planned [84][109] Question: How does the decision to expand enrollment impact the registration pathway for suprachoroidal delivery? - Management believes that the expansion will support important milestones for regulatory approvals in both the U.S. and internationally [91][92] Question: What is the expected safety profile for RGX-202 in the Duchenne program? - Management stated that the trial is excluding patients with preexisting AAV8 antibodies to ensure safety and efficacy [95][97] Question: How will the fellow eye study be conducted in relation to the pivotal trials? - The fellow eye study will treat patients at a later time to avoid confounding results, aligning with FDA discussions [120][135]