"We believe RGX-202 has unique, differentiating features that support its potential to be a best-in-class product and we are pleased to expand its clinical development to reach a wider range of boys with Duchenne in need of treatment options," said Curran Simpson, Chief Operating Officer of REGENXBIO and President and CEOelect. "Supported by the strong safety profile and positive microdystrophin data demonstrated in the AFFINITY DUCHENNE trial, today's news marks significant steps in rapidly accelerating RG ...

Aligned with FDA on content of BLA and plans for submission: Submission of a rolling BLA using the accelerated approval pathway expected to start in Q3 2024 Confirmatory trial expected to begin in H2 2025 FDA confirmed RGX-121 commercial bulk drug is comparable to clinical material Positive biomarker, neurocognitive and systemic data will be part of BLA submission The FDA continues to be aligned with REGENXBIO's plan to use cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6, a key biomarker of br ...

Curran Simpson, Chief Operating Officer, Appointed as President and CEO Co-Founder Kenneth T. Mills to Step Down as President and CEO; Named Chairman of the Board Mr. Simpson is a seasoned biopharmaceuticals leader with over 35 years of industry experience. As the Chief Operating Officer (COO) at REGENXBIO since January 2023, he has been directly responsible for key business functions including Research & Clinical Development, Corporate Strategy, Manufacturing & Quality, Regulatory, and Commercial Operation ...

Company Overview - REGENXBIO Inc. is a clinical-stage biotechnology company focused on developing adeno-associated virus (AAV) gene therapies for both common and rare diseases, utilizing its proprietary NAV platform to create one-dose AAV treatments that target the root causes of diseases [9][24] - The company has a diverse pipeline that includes ABBV-RGX-314 for age-related macular degeneration (AMD) and diabetic retinopathy (DR), RGX-202 for Duchenne muscular dystrophy (DMD), and RGX-121 for Hunter syndrome [25][26][18] Market Potential - The global market potential for ABBV-RGX-314 is estimated to be approximately $17.0 billion over the next five years, with the US anti-VEGF market alone valued at $4.5 billion annually [6] - The US retinal disease market is projected to reach $11.5 billion, while the EU market is estimated at $4.5 billion and Japan at $1 billion [6] - The global market for DMD is estimated at $7.0 billion, and for Mucopolysaccharidosis Type II (MPS II), it is around $1.0 billion [6] Financial Position - RGNX's balance sheet shows $113.0 million in cash and equivalents, plus $225.7 million in marketable securities, totaling $338.7 million in liquidity after a recent $140.0 million equity raise [13] - The company's latest quarterly cash burn was $56.1 million, indicating a yearly cash burn rate of $224.4 million, which suggests a cash runway of about 1.5 years, sufficient to finance operations until late 2025 [13] Clinical Development - ABBV-RGX-314 is currently in phases 2 and 3 for wet AMD, with regulatory submissions planned for late 2025 and early 2026 [10] - RGX-202 is in phase 2 for DMD, utilizing an AAV8 vector to deliver a transgene that encodes a microdystrophin protein to restore muscle functionality [26] - RGX-121 is in a pivotal study for MPS II, with plans to file a Biologics License Application (BLA) in 2024 [18] Strategic Collaborations - RGNX has a strategic collaboration with AbbVie for the development of ABBV-RGX-314, which includes a $200.0 million milestone payment related to DR [8][15] - AbbVie has committed significant resources, including a $370 million upfront payment for the rights to ABBV-RGX-314, with potential additional development and milestone payments totaling up to $1.38 billion [27] Valuation and Investment Outlook - RGNX trades at a market cap of $693.5 million, with a forward price-to-sales (P/S) ratio of 2.8, which is considered cheap compared to the sector median of 3.7 [29] - The company is viewed as undervalued despite its promising pipeline and potential revenue streams from ongoing projects and collaborations [22][29]

Company Overview - REGENXBIO Inc. is a leading clinical-stage biotechnology company focused on gene therapy with a mission to improve lives through its curative potential [3] - The company has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines since its founding in 2009 [3] - REGENXBIO is advancing a pipeline of AAV Therapeutics targeting retinal and rare diseases, including collaborations with AbbVie for ABBV-RGX-314 [3] Upcoming Event - REGENXBIO will participate in the Goldman Sachs 45th Annual Global Healthcare Conference on June 12, 2024, at 8:00 a.m. EDT [2] - A live webcast of the event will be available on the Investors section of REGENXBIO's website, with an archived replay accessible for approximately 30 days post-presentation [2] Product Pipeline - The company is developing several AAV Therapeutics, including RGX-202 for Duchenne muscular dystrophy and RGX-121 for MPS II [3] - Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, which includes Novartis' ZOLGENSMA for spinal muscular atrophy [3] - AAV Therapeutics are designed as one-time treatments, potentially transforming healthcare delivery for millions [3]

REGENXBIO Inc. (NASDAQ:RGNX) Q1 2024 Earnings Conference Call May 8, 2024 4:30 PM ET Company Participants Patrick Christmas – Chief Legal Officer Ken Mills – Chief Executive Officer Curran Simpson – Chief Operating Officer Vit Vasista – Chief Financial Officer Conference Call Participants Gena Wang – Barclays Annabel Samimy – Stifel Brian Skorney – Baird Luca Issi – RBC Capital Daniil Gataulin – Chardan Operator Good day and thank you for standing by. Welcome to REGENXBIO's First Quarter 2024 Earnings Confe ...

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 ...