Core Insights - REGENXBIO Inc. announced positive interim data from the Phase I/II AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, showing consistent functional benefits and safety [1][2][3] Functional Data - RGX-202 participants at dose level 2 (2x10^14 GC/kg) showed significant functional improvements at both 9 and 12 months, exceeding natural history controls on key measures such as the North Star Ambulatory Assessment (NSAA) [4][5] - At 9 months, participants improved an average of 4 points from baseline on NSAA, and at 12 months, the improvement was 4.5 points from baseline, with a 6.8-point increase compared to natural history [5][6] Biomarker Data - Biomarker data indicated robust microdystrophin expression, with one participant aged 2 showing an expression level of 118.6% compared to control [8][10] - The primary endpoint for the pivotal phase is the proportion of participants with microdystrophin expression ≥10% at Week 12 [8] Safety and Tolerability - RGX-202 demonstrated a favorable safety profile with no serious adverse events reported, and common drug-related adverse events included nausea, vomiting, and fatigue [11][12] - The proactive immune modulation regimen and high product purity levels contributed to the positive safety outcomes [11] Pivotal Trial and Future Plans - REGENXBIO is enrolling participants for the pivotal portion of the AFFINITY DUCHENNE trial, aiming to support a Biologics License Application (BLA) submission under accelerated approval by mid-2026 [14][15] - The company plans to share top-line data in the first half of 2026, including biomarker, functional, and safety data [15] About RGX-202 - RGX-202 is designed to improve function and outcomes in Duchenne muscular dystrophy, utilizing a differentiated microdystrophin construct that encodes key regions of dystrophin [17][18] - The therapy aims to support targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV® AAV8 vector [18] About Duchenne Muscular Dystrophy - Duchenne muscular dystrophy is a severe, progressive muscle disease affecting 1 in 3,500 to 5,000 boys born each year, caused by mutations in the dystrophin gene [19] About REGENXBIO Inc. - REGENXBIO is a biotechnology company focused on gene therapy, with a late-stage pipeline including RGX-202 for Duchenne and other investigational therapies for rare diseases [20]

Group 1 - REGENXBIO Inc. will host a webcast to discuss interim functional data from the Phase I/II AFFINITY DUCHENNE® trial of RGX-202, a gene therapy for Duchenne muscular dystrophy [1] - The webcast will feature principal investigator Aravindhan Veerapandiyan, M.D., from Arkansas Children's Hospital [1] - The event is scheduled for June 5, 2025, at 8:00 a.m. EDT, and will be accessible via REGENXBIO's website [2] Group 2 - REGENXBIO is a biotechnology company focused on gene therapy, founded in 2009, and has pioneered AAV gene therapy [3] - The company is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for Duchenne muscular dystrophy [3] - REGENXBIO's investigational therapies have the potential to significantly impact healthcare delivery for millions [3]

Summary of REGENXBIO Conference Call Company Overview - **Company**: REGENXBIO (RGNX) - **Industry**: Biotechnology, specifically focusing on gene therapy Key Points and Arguments Recent Financing Deal - REGENXBIO announced an opportunistic non-dilutive financing deal aimed at pulling forward midterm royalty streams to prepare for pre-commercial launches, including RGX-202 and RGX-314 [2][3] - The financing is characterized as "good debt" since it does not obligate the company to repay if product sales underperform, while retaining the royalty stream if the products become blockbusters [3][4] FDA Interactions and Regulatory Environment - The company is closely monitoring FDA developments, particularly with the nomination of Dr. Prasad, who has been critical of surrogate endpoints [6][7] - REGENXBIO is currently undergoing a review for its Hunter program, with ongoing interactions with the FDA that have been described as routine [7][8] - The company is optimistic about the potential for accelerated approval based on consistent microdystrophin levels associated with functional benefits [8][9] Market Reactions and Competitive Landscape - Following tragic events related to Sarepta's product, there is increased caution among doctors regarding prescribing to non-ambulatory patients, but REGENXBIO has not seen a change in patient enrollment [10][13] - The company views the lowered financial guidance from Sarepta as a potential opportunity, suggesting that a larger prevalence pool may be available by the time REGENXBIO launches its products [15][16] Product Development and Clinical Trials - REGENXBIO is preparing for pivotal studies and expects to report top-line data from these studies in the first half of next year [28][29] - The company is focused on expanding its dataset for the Hunter program and will provide updates on pivotal study enrollment later this year [27][28] Gene Therapy Strategy and Differentiation - REGENXBIO emphasizes its in-house manufacturing capabilities, which allow for the production of 2,500 doses per year at a competitive cost, as a key differentiator in the gene therapy space [49][50] - The company is advancing its subretinal program for wet AMD in collaboration with AbbVie, with pivotal studies enrolling 1,200 patients [37][38] Safety and Efficacy Considerations - The company has implemented a robust immune-modulating regimen to mitigate risks associated with liver-related adverse events, which has been well-received by investigators [12][13] - REGENXBIO is optimistic about the safety profile of its therapies, particularly in the context of the competitive landscape where safety is a critical concern [46][47] Underappreciated Aspects - The company believes that its cash runway has been improved through recent financing and that its in-house manufacturing capabilities are underappreciated aspects of its business model [49][50] Additional Important Content - The company is preparing for potential advisory committee meetings with the FDA but remains optimistic about the likelihood of not needing one based on precedents [29][30] - The potential for a Priority Review Voucher (PRV) upon approval is highlighted, with recent market data indicating values exceeding $150 million [31][32] This summary encapsulates the key discussions and insights from the REGENXBIO conference call, providing a comprehensive overview of the company's current status, strategic direction, and market positioning.

Core Viewpoint - REGENXBIO Inc. has secured a non-dilutive, limited recourse royalty bond agreement of up to $250 million with Healthcare Royalty, which will enhance its cash runway and support upcoming milestones in its gene therapy pipeline [1][2][3] Financing Details - The agreement allows REGENXBIO to receive $150 million at closing, with an extension of cash runway into early 2027 [1][8] - An additional $50 million will be funded upon achieving sales milestones for ZOLGENSMA, and another $50 million will be available upon mutual agreement between the parties [5][6] Strategic Implications - This financing is aimed at advancing late-stage activities, including potential FDA approvals and data readouts for key products such as RGX-121 and RGX-202 [2][3] - The capital infusion is expected to accelerate commercial preparations and maintain REGENXBIO's leadership in rare and retinal gene therapies [2][8] Agreement Terms - HCRx will receive rights to anticipated royalty payments from ZOLGENSMA and other products, with quarterly interest payments based on royalty and milestone revenue [3][4] - The agreement does not include other potential non-dilutive funding sources, such as the sale of a Priority Review Voucher for RGX-121 [6][8] Company Background - REGENXBIO is focused on gene therapy, with a late-stage pipeline targeting rare and retinal diseases, including partnerships with Nippon Shinyaku and AbbVie [8][9] - The company has pioneered AAV gene therapy and aims to improve healthcare delivery through its investigational therapies [9]

Core Insights - The U.S. FDA has accepted the Biologics License Application (BLA) for RGX-121, a gene therapy for Mucopolysaccharidosis II (MPS II), with a target action date of November 9, 2025 [1][5] - NS Pharma will exclusively commercialize RGX-121 in the U.S. upon potential approval, following a strategic partnership with REGENXBIO [2][3] Company Overview - NS Pharma, a subsidiary of Nippon Shinyaku Co., Ltd., focuses on developing and commercializing innovative therapies [8] - REGENXBIO is a clinical-stage biotechnology company specializing in gene therapy, particularly AAV Therapeutics [7] Product Details - RGX-121 is designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system, potentially providing a long-term solution for MPS II [4] - The therapy has received multiple FDA designations, including Orphan Drug Product and Rare Pediatric Disease [5] Disease Context - MPS II, also known as Hunter Syndrome, is a rare genetic disorder caused by a deficiency in the I2S enzyme, leading to significant health challenges, particularly neurological [6]

Core Viewpoint - REGENXBIO Inc. announced that the FDA has accepted the Biologics License Application (BLA) for RGX-121, a potential one-time gene therapy for Mucopolysaccharidosis II (MPS II), with a target action date of November 9, 2025 [1][7]. Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, founded in 2009, and has developed a late-stage pipeline for rare and retinal diseases [6]. - The company has pioneered AAV gene therapy and aims to improve lives through its curative potential [6]. Product Details - RGX-121 (clemidsogene lanparvovec) is designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system, potentially providing a permanent source of the I2S protein [4]. - The therapy aims to address both neurodevelopmental and systemic effects of Hunter syndrome, which currently relies on weekly enzyme replacement therapy [2]. Regulatory Designations - RGX-121 has received multiple designations from the FDA, including Orphan Drug Product, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) [2]. Commercialization Strategy - Following potential FDA approval, RGX-121 will be commercialized by NS Pharma, a subsidiary of Nippon Shinyaku, while REGENXBIO retains all rights and proceeds related to the potential sale of a Priority Review Voucher (PRV) [3].

Group 1 - Regenxbio reported quarterly earnings of $0.12 per share, missing the Zacks Consensus Estimate of $0.41 per share, compared to a loss of $1.38 per share a year ago, representing an earnings surprise of -70.73% [1] - The company posted revenues of $89.01 million for the quarter ended March 2025, missing the Zacks Consensus Estimate by 19.07%, compared to year-ago revenues of $15.62 million [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's immediate price movement will depend on management's commentary on the earnings call and the sustainability of earnings expectations [3][4] - Regenxbio shares have lost about 2.7% since the beginning of the year, while the S&P 500 has declined by 3.8% [3] - The current consensus EPS estimate for the coming quarter is -$1.21 on $21.98 million in revenues, and -$0.88 on $295.17 million in revenues for the current fiscal year [7] Group 3 - The Medical - Biomedical and Genetics industry, to which Regenxbio belongs, is currently in the top 34% of the Zacks industries, indicating a favorable outlook [8] - Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors [5][6]

Financial Data and Key Metrics Changes - REGENXBIO ended the quarter on March 31, 2025, with cash, cash equivalents, and marketable securities of $272 million, an increase from $245 million as of December 31, 2024, primarily driven by a $110 million upfront payment from the Nippon Shinyaku collaboration [24] - R&D expenses were $53 million for the quarter ended March 31, 2025, compared to $54.8 million for the same quarter in 2024, reflecting a decrease due to clinical trial expenses for RGX-314 and RGX-202 [25] Business Line Data and Key Metrics Changes - RGX-121, a potential first gene therapy for MPS II, is on track for potential FDA approval in the second half of 2025, with a BLA submitted under the accelerated approval pathway [8][9] - RGX-202, a next-generation candidate for Duchenne muscular dystrophy (DMD), has surpassed 50% enrollment in its pivotal study and is expected to submit a BLA in mid-2026 [10][11] - The retinal program, RGX-314, is advancing in two pivotal studies for wet AMD and is on track to be the first gene therapy on the market for this condition [12][19] Market Data and Key Metrics Changes - The DMD market is projected to have over half of the prevalent population untreated by 2027, highlighting a significant opportunity for RGX-202 [11] - The wet AMD and diabetic retinopathy markets represent large multibillion-dollar commercial opportunities, with RGX-314 positioned to serve as a meaningful alternative to current treatments [12][19] Company Strategy and Development Direction - The company is focused on transitioning to a commercial stage with in-house manufacturing capabilities and plans to secure non-dilutive funding [6][8] - A strategic partnership with Nippon Shinyaku aims to commercialize the neurodegenerative franchise, including RGX-121 and RGX-111 [9] - The company is preparing for commercial supply manufacturing of RGX-202 in anticipation of a 2027 launch [11][27] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the imminent acceptance of the BLA for RGX-121 and the positive trajectory of clinical programs [34][51] - The company is optimistic about the evolving regulatory landscape and the potential for accelerated approval pathways, particularly in light of recent developments in the industry [36][44] Other Important Information - The company has a robust cash runway expected to fund operations into the second half of 2026, with potential extensions through non-dilutive financing options [25][26] - The manufacturing innovation center in Rockville, Maryland, is capable of producing up to 2,500 doses of RGX-202 annually, ensuring readiness for market needs [11][12] Q&A Session Summary Question: Timing for the Hunter BLA - Management indicated that the BLA acceptance is imminent and they feel confident about the review process [34] Question: Competitor Insights and Regulatory Expectations - Management discussed the evolving expectations for accelerated approval in DMD, emphasizing the strength of their safety and functional data [36][44] Question: Changes in FDA Requirements - Management noted that there are no anticipated changes in the accelerated approval pathway and enrollment is on track [43][44] Question: Planning for Diabetic Retinopathy Phase III Trial - Management confirmed ongoing interactions with the FDA and that they are on track for first patient dosing this year [55] Question: Updates on DMD Functional Data - Management plans to release additional functional data in the first half of the year, focusing on dose level two patients [62][88] Question: Impact of Recent Pricing Announcements - Management stated it is too early to assess the impact of recent pricing discussions on their gene therapies [104]

Financial Data and Key Metrics Changes - REGENXBIO ended the quarter on March 31, 2025, with cash, cash equivalents, and marketable securities of $272 million, an increase from $245 million as of December 31, 2024, primarily driven by a $110 million upfront payment from the Nippon Shinyaku collaboration [19][20]. - R&D expenses for the quarter were $53 million, down from $54.8 million in the same quarter of 2024, mainly due to clinical trial expenses for RGX-314 and RGX-202 [20]. Business Line Data and Key Metrics Changes - RGX-121, a gene therapy for MPS II, is on track for potential FDA approval in the second half of 2025, with a BLA submitted under the accelerated approval pathway [5][6]. - RGX-202, a candidate for Duchenne muscular dystrophy (DMD), has surpassed 50% enrollment in its pivotal study and is expected to submit a BLA in mid-2026 [7][12]. - The retinal program, RGX-314, is advancing in two pivotal studies for wet AMD and is on track to be the first gene therapy for this condition [9][16]. Market Data and Key Metrics Changes - The DMD market is projected to have over half of the prevalent population untreated by 2027, highlighting a significant opportunity for RGX-202 [8]. - The wet AMD and diabetic retinopathy markets represent large multibillion-dollar commercial opportunities, with RGX-314 positioned to serve as a meaningful alternative to current treatments [9][16]. Company Strategy and Development Direction - The company is focused on transitioning to a commercial stage with in-house manufacturing capabilities and plans to secure non-dilutive funding [4][5]. - A strategic partnership with Nippon Shinyaku aims to commercialize the neurodegenerative franchise, including RGX-121 and RGX-111 [6]. - The company is preparing for commercial supply manufacturing of RGX-202 in anticipation of a 2027 launch [8][24]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the clinical progress and the potential for multiple first or best-in-class gene therapies to reach patients in the coming years [4][10]. - The company is optimistic about the FDA's review process for RGX-121 and believes the data supports a favorable benefit-risk profile for its gene therapies [30][46]. Other Important Information - The company has a robust cash runway expected to fund operations into the second half of 2026, with potential extensions through non-dilutive financing options [20][21]. - The manufacturing innovation center in Rockville, Maryland, is capable of producing up to 2,500 doses of RGX-202 annually, ensuring readiness for market needs upon approval [8][24]. Q&A Session Summary Question: Timing for the Hunter BLA - Management indicated that the BLA acceptance is imminent and they feel confident about the review process [30]. Question: Changes in Approvals and Biomarker Expectations - Management stated that they are prepared for an AdCom if required and are confident in their data supporting the accelerated approval pathway [32][82]. Question: Safety Profile Expectations - Management noted that they do not anticipate changes in the FDA's requirements regarding safety profiles and that enrollment for the pivotal study is on track [38][40]. Question: Updates on Diabetic Retinopathy Phase III Trial - Management confirmed that they are actively working with AbbVie on the final feedback for the trial and expect to begin site activation soon [51]. Question: Functional Data Updates for DMD - Management plans to release additional functional data for RGX-202 in the first half of the year, focusing on expanding the patient data set [57][84]. Question: Impact of Recent Pricing Announcements - Management believes it is too early to assess the impact of recent pricing discussions on their gene therapies [102].

[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)