Core Insights - MeiraGTx Holdings plc is showcasing four poster presentations at the ASGCT 2025 Annual Meeting, highlighting advancements in gene and cell therapy technologies [1][19] Group 1: Poster Presentations - Poster 507 discusses an ultra-low dose localized CNS gene therapy targeting severe pediatric obesity, utilizing BDNF delivered via AAV to improve metabolic health and prevent severe obesity-related conditions [3][4][5] - Poster 893 presents novel CAG-based promoters that outperform traditional promoters in both mouse and human models, aiming to enhance gene therapy safety and efficacy by reducing required dosages [6][7][8][9][10] - Poster 897 explores the differential usage of transcription factor binding sites to enhance synthetic promoter activity, demonstrating significant improvements in gene expression through optimized designs [11][12][13][14] - Poster 1040 details the preclinical efficacy of an optimized AAV-hUPF1 gene therapy for ALS and Frontotemporal Dementia, showing promise in improving motor neuron survival and therapeutic efficacy [15][16][18] Group 2: Company Overview - MeiraGTx is a vertically integrated, clinical-stage genetic medicines company with a diverse pipeline, including four late-stage clinical programs targeting inherited and common diseases [19][20] - The company has developed a proprietary manufacturing platform and possesses comprehensive manufacturing capabilities, including GMP facilities for viral vector production [20] - MeiraGTx is focusing on innovative technologies for gene therapy, including riboswitch gene regulation technology for precise control of gene expression [20]

Group 1 - MeiraGTx Holdings PLC reported a quarterly loss of $0.51 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.38, and an improvement from a loss of $0.77 per share a year ago, indicating a surprise of -34.21% [1] - The company generated revenues of $1.93 million for the quarter ended March 2025, missing the Zacks Consensus Estimate by 76.85%, compared to revenues of $0.7 million in the same quarter last year [2] - MeiraGTx shares have declined approximately 7.7% since the beginning of the year, while the S&P 500 has seen a decline of -0.6% [3] Group 2 - The earnings outlook for MeiraGTx is uncertain, but current consensus EPS estimates for the upcoming quarter are -$0.39 on revenues of $8.32 million, and -$0.09 on revenues of $126.94 million for the current fiscal year [7] - The Medical - Biomedical and Genetics industry, to which MeiraGTx belongs, is currently ranked in the top 35% of over 250 Zacks industries, suggesting a favorable environment for stock performance [8]

PART I. FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents MeiraGTx's unaudited condensed consolidated financial statements, including balance sheets, statements of operations, equity, and cash flows, with explanatory notes [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) This section provides a snapshot of the company's financial position, detailing assets, liabilities, and shareholders' equity at specific dates | Metric | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | | :-------------------------------- | :----------------------------- | :----------------------------- | | Total Current Assets | $79,889 | $123,518 | | Total Assets | $224,014 | $269,751 | | Total Current Liabilities | $52,372 | $60,783 | | Total Liabilities | $190,915 | $201,924 | | Total Shareholders' Equity | $33,099 | $67,827 | - The company's total assets decreased from **$269.8 million** at December 31, 2024, to **$224.0 million** at March 31, 2025, primarily driven by a decrease in cash and cash equivalents[20](index=20&type=chunk) - Shareholders' equity significantly decreased from **$67.8 million** at December 31, 2024, to **$33.1 million** at March 31, 2025, largely due to the net loss incurred during the period[20](index=20&type=chunk) [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) This section details the company's financial performance, including revenue, operating expenses, and net loss, for the reported periods | Metric (in thousands) | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :-------------------- | :-------------------------------- | :-------------------------------- | | Service revenue - related party | $1,926 | $697 | | Total operating expenses | $43,522 | $47,469 | | Loss from operations | $(41,596) | $(46,772) | | Net loss | $(39,981) | $(20,442) | | Basic and diluted net loss per ordinary share | $(0.51) | $(0.32) | - The net loss for the three months ended March 31, 2025, significantly increased to **$39.98 million** from **$20.44 million** in the same period of 2024, primarily due to the absence of a large gain on sale of nonfinancial assets recognized in 2024[23](index=23&type=chunk)[197](index=197&type=chunk) - Service revenue from related parties increased from **$0.7 million** in Q1 2024 to **$1.9 million** in Q1 2025, driven by increased progress in PPQ services[23](index=23&type=chunk)[186](index=186&type=chunk) [Condensed Consolidated Statement of Shareholders' Equity](index=9&type=section&id=Condensed%20Consolidated%20Statement%20of%20Shareholders'%20Equity) This section outlines the changes in the company's shareholders' equity, reflecting net loss, share-based compensation, and share issuance | Metric (in thousands) | Balance at Dec 31, 2024 | Share-based Compensation | Issuance of Shares | Other Comprehensive Loss | Net Loss (Q1 2025) | Balance at Mar 31, 2025 | | :-------------------- | :---------------------- | :----------------------- | :----------------- | :----------------------- | :----------------- | :---------------------- | | Total Shareholders' Equity | $67,827 | $2,130 | $4,470 | $(1,347) | $(39,981) | $33,099 | - Shareholders' equity decreased by **$34.7 million** during Q1 2025, primarily due to the net loss of **$39.98 million**, partially offset by share-based compensation activity and issuance of ordinary shares[25](index=25&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) This section summarizes the company's cash inflows and outflows from operating, investing, and financing activities for the reported periods | Cash Flow Activity (in thousands) | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | Net cash used in operating activities | $(36,828) | $(36,741) | | Net cash (used in) provided by investing activities | $(1,710) | $27,340 | | Net cash provided by (used in) financing activities | $1,713 | $(1,270) | | Net decrease in cash, cash equivalents and restricted cash | $(36,825) | $(10,671) | - Net cash used in operating activities remained consistent at approximately **$36.8 million** for both periods, reflecting ongoing research and development expenses[28](index=28&type=chunk)[203](index=203&type=chunk)[204](index=204&type=chunk) - Investing activities shifted from providing **$27.3 million** in Q1 2024 (due to proceeds from sale of nonfinancial assets) to using **$1.7 million** in Q1 2025 for property and equipment purchases[28](index=28&type=chunk)[205](index=205&type=chunk) - Financing activities provided **$1.7 million** in Q1 2025, primarily from an at-the-market equity offering, contrasting with a **$1.3 million** use in Q1 2024[28](index=28&type=chunk)[207](index=207&type=chunk)[208](index=208&type=chunk) [Notes to Condensed Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) This section provides detailed explanations of the company's accounting policies, significant transactions, and financial statement items [1. Organization and Basis of Presentation](index=12&type=section&id=1.%20Organization%20and%20Basis%20of%20Presentation) This section details the company's business as a clinical-stage genetic medicines company, its strategic collaboration with Hologen Limited, and the asset purchase agreement with Johnson & Johnson Innovative Medicine - MeiraGTx is a vertically integrated, clinical-stage genetic medicines company with late-stage clinical programs in Parkinson's disease, radiation-induced xerostomia, and AIPL1-associated retinal dystrophy, supported by internal end-to-end manufacturing capabilities[29](index=29&type=chunk) - The company entered into a strategic collaboration with Hologen Limited on March 9, 2025, which includes an upfront cash payment of **$200 million** and up to an additional **$230 million** in funding for a joint venture (Hologen Neuro AI Ltd) focused on CNS genetic medicines[30](index=30&type=chunk)[33](index=33&type=chunk)[139](index=139&type=chunk) - Under the Hologen Neuro Framework Agreement, MeiraGTx Neuro US will grant exclusive, worldwide, royalty-free licenses for its Clinical Programs (AAV-GAD, AAV-BDNF) and Delivery Device to Hologen Neuro and Hologen Neuro UK[33](index=33&type=chunk) - The Manufacturing Framework Agreement involves Hologen acquiring a minority interest in MeiraGTx Manufacturing and contributing to annual funding, with an option for Hologen to increase its stake to **40%** within 12 months[36](index=36&type=chunk)[38](index=38&type=chunk) - The Asset Purchase Agreement with Johnson & Johnson Innovative Medicine, signed December 20, 2023, involved the sale of RPGR Product assets for an upfront payment of **$65.0 million** and potential future contingent consideration of up to **$350.0 million**, of which **$60.0 million** has been received[39](index=39&type=chunk)[40](index=40&type=chunk)[106](index=106&type=chunk) - The company has an accumulated deficit of **$742.0 million** as of March 31, 2025, and expects to incur substantial losses, but anticipates its current cash, receivables, and the Hologen collaboration proceeds will fund operations into 2027 and cover its **$75.0 million** debt obligation[43](index=43&type=chunk)[45](index=45&type=chunk)[135](index=135&type=chunk) [2. Summary of Significant Accounting Policies and Recent Accounting Pronouncements](index=17&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies%20and%20Recent%20Accounting%20Pronouncements) This section outlines the company's key accounting policies, including consolidation, use of estimates, restricted cash, fair value measurements, revenue recognition, and research and development expensing - The company's financial statements are prepared in conformity with GAAP and include the accounts of Meira Holdings and its wholly-owned subsidiaries, with all intercompany balances eliminated[41](index=41&type=chunk)[49](index=49&type=chunk)[51](index=51&type=chunk) - Restricted cash of **$2.087 million** at March 31, 2025, is held as a guarantee for a research and innovation grant from IDA Ireland, which offers financial assistance for operations in Shannon, Ireland[53](index=53&type=chunk)[61](index=61&type=chunk) - Revenue recognition follows a five-step model, allocating transaction price to performance obligations based on estimated standalone selling prices, with variable consideration estimated using the most likely amount method[66](index=66&type=chunk)[69](index=69&type=chunk)[71](index=71&type=chunk) - Research and development costs are expensed as incurred, including employee-related expenses, third-party vendor costs for clinical/preclinical studies, and manufacturing facility development[78](index=78&type=chunk)[172](index=172&type=chunk)[173](index=173&type=chunk) - The company has one reportable and operating segment: the development and manufacturing of genetic medicines[83](index=83&type=chunk) - Recent accounting pronouncements (ASU 2023-06, ASU 2023-09, ASU 2024-03, ASU 2025-01) are being evaluated, with ASU 2023-09 not expected to have a material impact[88](index=88&type=chunk)[89](index=89&type=chunk)[90](index=90&type=chunk) [3. Equity Method and Other Investments](index=30&type=section&id=3.%20Equity%20Method%20and%20Other%20Investments) This section details the company's equity method and other investments, primarily in Visiogene LLC and another equity investment | Investee | Investment Type | Ownership Percentage | Carrying Value (in thousands) | | :--------- | :--------------------- | :------------------- | :---------------------------- | | Visiogene LLC | Equity Method Investment | 25% | $5,133 | | Other | Equity Investment | 0.9% | $1,616 | | Total | | | $6,749 | [4. Accrued Expenses](index=30&type=section&id=4.%20Accrued%20Expenses) This section provides a breakdown of accrued expenses, highlighting changes in compensation, professional fees, and clinical trial costs between December 31, 2024, and March 31, 2025 | Accrued Expense (in thousands) | March 31, 2025 | December 31, 2024 | | :----------------------------- | :------------- | :---------------- | | Compensation and benefits | $8,129 | $11,197 | | Professional fees | $4,163 | $6,326 | | Research and development | $2,857 | $2,234 | | Interest on Tranche 1 Notes | $2,620 | — | | Clinical trial costs | $2,421 | $3,864 | | Manufacturing costs | $2,104 | $1,540 | | Consulting | $1,326 | $1,530 | | Total Accrued Expenses | $23,964 | $27,414 | - Accrued expenses decreased by **$3.45 million** from December 31, 2024, to March 31, 2025, primarily due to decreases in compensation and benefits, professional fees, and clinical trial costs[92](index=92&type=chunk)[93](index=93&type=chunk) [5. Share-Based Compensation](index=32&type=section&id=5.%20Share-Based%20Compensation) This section details the company's equity incentive plans, including share options and restricted share units (RSUs), and the associated share-based compensation expense recognized during the periods | Share-Based Compensation (in thousands) | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :-------------------------------------- | :-------------------------------- | :-------------------------------- | | Research and development | $2,046 | $2,875 | | General and administrative | $2,841 | $4,085 | | Total share-based compensation | $4,887 | $6,960 | - Total share-based compensation expense decreased by **$2.073 million**, from **$6.96 million** in Q1 2024 to **$4.887 million** in Q1 2025[102](index=102&type=chunk) - As of March 31, 2025, unrecognized compensation expense for unvested options was **$6.8 million** (expected over **2.9 years**) and for unvested RSUs was **$18.5 million** (expected over **3.1 years**)[98](index=98&type=chunk)[100](index=100&type=chunk) [6. Ordinary Shares](index=35&type=section&id=6.%20Ordinary%20Shares) This section describes the company's at-the-market (ATM) equity offering program, through which it raises capital by selling ordinary shares - During Q1 2025, the company raised gross proceeds of **$4.5 million** by selling **563,379** ordinary shares through its at-the-market offering program[103](index=103&type=chunk) - An additional **$87.1 million** of ordinary shares remain available for sale under the current at-the-market equity program[103](index=103&type=chunk) [7. Income Taxes](index=36&type=section&id=7.%20Income%20Taxes) This section explains that the company did not record income tax provisions due to sustained losses and maintains a full valuation allowance against its deferred tax assets - No provision for income taxes was recorded for Q1 2025 or Q1 2024, as the company has generated losses[104](index=104&type=chunk) - The company maintains a full valuation allowance against its deferred tax assets in the United States, United Kingdom, Ireland, and Netherlands due to uncertainty regarding their realizability[105](index=105&type=chunk) [8. Related-Party Transactions](index=36&type=section&id=8.%20Related-Party%20Transactions) This section details the financial aspects of the Asset Purchase Agreement and related Supply Agreement with Johnson & Johnson Innovative Medicine, including milestone payments, revenue recognition, and the debt financing arrangement with Perceptive Credit Holdings III, LP - Under the Asset Purchase Agreement with Johnson & Johnson Innovative Medicine, the company is eligible for up to **$350.0 million** in future contingent consideration, with **$60.0 million** already received[106](index=106&type=chunk) - The transaction price of **$92.3 million** from the Asset Purchase Agreement and related agreements was allocated to four performance obligations: PPQ services, material rights for commercial supply, manufacturing technology transfer, and sale of nonfinancial assets[107](index=107&type=chunk) - During Q1 2024, a gain of **$29.0 million** related to the sale of nonfinancial assets was recognized; no such gain was recognized in Q1 2025[110](index=110&type=chunk) - As of March 31, 2025, **$63.9 million** in aggregate transaction price was allocated to unsatisfied performance obligations, expected to be recognized over approximately **2.8 years**[111](index=111&type=chunk) - The company recognized **$0.7 million** of deferred revenue as service revenue in both Q1 2025 and Q1 2024 in connection with PPQ services[112](index=112&type=chunk) - The company has a senior secured financing arrangement (Notes Purchase Agreement) with Perceptive Credit Holdings III, LP, for an initial **$75.0 million**, maturing August 2, 2026, with an annual interest rate of **14.34%** at March 31, 2025[119](index=119&type=chunk)[120](index=120&type=chunk) [9. Leases](index=39&type=section&id=9.%20Leases) This section outlines the company's lease commitments for operating and finance leases, including lease costs, right-of-use assets, and future minimum lease payments | Lease Cost (in thousands) | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :------------------------ | :-------------------------------- | :-------------------------------- | | Finance lease cost | $138 | $281 | | Operating lease cost | $1,284 | $1,437 | | Short-term lease cost | $95 | $41 | | Total lease cost | $1,517 | $1,759 | - Total lease cost decreased from **$1.759 million** in Q1 2024 to **$1.517 million** in Q1 2025[118](index=118&type=chunk) - The company exercised an early termination option on an operating lease for laboratory and office space in Q1 2025, resulting in a **$1.3 million** gain on termination of lease liabilities[60](index=60&type=chunk)[117](index=117&type=chunk) | Lease Type | Right-of-Use Assets (Mar 31, 2025, in thousands) | Capitalized Lease Obligations (Mar 31, 2025, in thousands) | Weighted-Average Remaining Lease Term (years) | Weighted-Average Discount Rate (%) | | :--------- | :----------------------------------------------- | :--------------------------------------------------------- | :------------------------------------ | :--------------------------------- | | Operating | $6,348 | $7,383 | 4.0 | 11.7 | | Finance | $22,728 | N/A | 173.7 | 8.0 | [10. Debt Financing](index=41&type=section&id=10.%20Debt%20Financing) This section details the company's senior secured financing arrangement, including the Tranche 1 Notes, interest rates, collateral, covenants, and associated warrants and debt discounts - The company has a Notes Purchase Agreement for an initial **$75.0 million** (Tranche 1 Notes) with Perceptive, maturing on August 2, 2026, with interest-only payments during the term[119](index=119&type=chunk)[120](index=120&type=chunk) - The annual interest rate was **14.34%** at March 31, 2025, and the outstanding balance of Tranche 1 Notes was **$75.0 million** plus **$2.6 million** in accrued interest[120](index=120&type=chunk) - Obligations are secured by manufacturing facilities in London and Shannon, **$3.0 million** of cash, bank accounts of subsidiary guarantors, and equity interests of subsidiary guarantors[121](index=121&type=chunk) - The company granted warrants to Perceptive to purchase **700,000** ordinary shares at exercise prices of **$15.00** and **$20.00** per share, expiring August 2, 2027[123](index=123&type=chunk) - A debt discount of **$2.3 million** for warrants and **$2.1 million** for lender/legal costs (**$4.4 million** total) is being amortized to interest expense over the term of the Notes Purchase Agreement[123](index=123&type=chunk)[124](index=124&type=chunk) [11. Commitments and Contingencies](index=41&type=section&id=11.%20Commitments%20and%20Contingencies) This section states that there were no new material commitments or contingencies during the three-month period ended March 31, 2025 - No new material commitments or contingencies were entered into during the three months ended March 31, 2025[126](index=126&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations.](index=43&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations.) This section provides management's perspective on the company's financial condition, results of operations, and liquidity, including an overview of its business, recent strategic collaborations, development highlights, and a detailed comparison of financial performance for the three months ended March 31, 2025, and 2024 [Overview](index=43&type=section&id=Overview) MeiraGTx is a clinical-stage genetic medicines company focused on developing gene therapies for various conditions, supported by its integrated manufacturing capabilities, and continues to incur significant losses while requiring additional capital for future development and commercialization - MeiraGTx is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline including Parkinson's disease, radiation-induced xerostomia, and AIPL1-associated retinal dystrophy, leveraging proprietary manufacturing and gene regulation platforms[128](index=128&type=chunk) - The company has incurred significant operating losses since inception, with net losses of **$40.0 million** and **$20.4 million** for the three months ended March 31, 2025, and 2024, respectively, and an accumulated deficit of **$742.0 million** as of March 31, 2025[132](index=132&type=chunk) - Total operating expenses were **$43.5 million** in Q1 2025, down from **$47.5 million** in Q1 2024, but the company expects continued high costs for clinical activities, pipeline expansion, and manufacturing[133](index=133&type=chunk) - Current cash, receivables, and anticipated proceeds from the Hologen collaboration are estimated to fund operations into 2027 and cover the **$75.0 million** debt obligation, excluding potential **$285.0 million** in milestones from the Johnson & Johnson Innovative Medicine Asset Purchase Agreement[135](index=135&type=chunk)[200](index=200&type=chunk) [Hologen Strategic Collaboration](index=47&type=section&id=Hologen%20Strategic%20Collaboration) MeiraGTx entered into a strategic collaboration with Hologen Limited in March 2025, involving an upfront cash payment, additional funding for a joint venture (Hologen Neuro AI Ltd) focused on CNS genetic medicines, and Hologen acquiring a minority stake in MeiraGTx's manufacturing subsidiary - On March 9, 2025, MeiraGTx entered a strategic collaboration with Hologen Limited, a generative AI developer, expecting an upfront cash payment of **$200 million** and up to **$230 million** in additional funding for a joint venture[139](index=139&type=chunk) - The collaboration includes the formation of Hologen Neuro AI Ltd, a joint venture where MeiraGTx Neuro UK will hold **30%** ownership and lead clinical development and manufacturing for AAV-GAD and other CNS genetic medicines[143](index=143&type=chunk)[155](index=155&type=chunk) - Hologen will acquire a minority interest in MeiraGTx Manufacturing, contributing to annual funding and deploying AI capabilities to optimize manufacturing[146](index=146&type=chunk)[155](index=155&type=chunk) - The closing of these transactions is expected in Q2 2025, subject to customary conditions including UK foreign direct investment clearances[140](index=140&type=chunk) [Recent Development Highlights and Anticipated Milestones](index=51&type=section&id=Recent%20Development%20Highlights%20and%20Anticipated%20Milestones) This section highlights recent regulatory designations and clinical trial progress for key product candidates, including AAV-GAD for Parkinson's disease, AAV2-hAQP1 for xerostomia, AAV-AIPL1 for LCA4, and botaretigene sparoparvovec for XLRP, along with updates on the riboswitch platform and manufacturing capabilities [Strategic Collaboration with Hologen AI](index=51&type=section&id=Strategic%20Collaboration%20with%20Hologen%20AI) This section details the financial and operational aspects of the strategic collaboration with Hologen AI, including upfront payments, joint venture structure, and manufacturing involvement - MeiraGTx will receive **$200 million** upfront cash and Hologen will commit up to an additional **$230 million** to a joint venture (Hologen Neuro AI Ltd) for AAV-GAD and other CNS therapies[155](index=155&type=chunk) - MeiraGTx will hold **30%** ownership in the joint venture and lead clinical development and manufacturing, with Hologen licensing its multi-modal generative foundation models (LMMs)[155](index=155&type=chunk) - Hologen will acquire a minority stake in MeiraGTx's manufacturing subsidiary, contributing funding and AI capabilities for manufacturing optimization[155](index=155&type=chunk) [AAV-GAD for the Treatment of Parkinson's Disease](index=51&type=section&id=AAV-GAD%20for%20the%20Treatment%20of%20Parkinson's%20Disease) This section outlines the recent RMAT designation and positive clinical data for AAV-GAD in Parkinson's disease, along with plans for a Phase 3 study - On May 8, 2025, AAV-GAD received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for Parkinson's disease not adequately controlled by anti-Parkinsonian medications[155](index=155&type=chunk) - Positive data from three clinical studies (Phase 1, Phase 2, and a bridging study) demonstrated significant clinically meaningful benefit on UPDRS Part 3 and other measures[155](index=155&type=chunk) - AI analysis of data showed disease-modifying changes in brain circuitry and potentially protective changes in the substantia nigra and cognition/mood regions[155](index=155&type=chunk) - A Phase 3 study for AAV-GAD is planned for initiation in the second half of 2025[155](index=155&type=chunk) [RMAT Designation](index=51&type=section&id=RMAT%20Designation) This section explains the criteria and benefits of the Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA - RMAT designation requires the drug candidate to be an advanced regenerative medicine targeting a serious condition with clinical evidence of potential to address an unmet need[152](index=152&type=chunk) - Benefits of RMAT include Fast Track and Breakthrough Therapy designations, rolling review, potential Priority Review, and increased interaction with the FDA for development and manufacturing strategy[153](index=153&type=chunk) [AAV2-hAQP1 for the Treatment of Xerostomia](index=51&type=section&id=AAV2-hAQP1%20for%20the%20Treatment%20of%20Xerostomia) This section details the RMAT designation for AAV2-hAQP1 in radiation-induced xerostomia and the progress of the ongoing Phase 2 AQUAx2 study - In December 2024, AAV2-hAQP1 received RMAT designation from the FDA for the treatment of Grade 2/3 radiation-induced xerostomia[154](index=154&type=chunk)[156](index=156&type=chunk) - The company has aligned with the FDA on clinical and CMC requirements for the ongoing Phase 2 AQUAx2 study to support a potential BLA[157](index=157&type=chunk) - Enrollment for the Phase 2 AQUAx2 study's high dose cohorts is ongoing, with target completion in Q4 2025 and potential BLA filing by end of 2026[157](index=157&type=chunk) [AAV-AIPL1 for LCA4 Caused by Mutations in the AIPL1 Gene](index=53&type=section&id=AAV-AIPL1%20for%20LCA4%20Caused%20by%20Mutations%20in%20the%20AIPL1%20Gene) This section presents positive clinical data for AAV-AIPL1 in LCA4 and outlines the company's regulatory submission plans in the UK and US - Data published in The Lancet in February 2025 showed substantial benefit in **4 out of 4** young children with LCA4 treated unilaterally with rAAV8.hRKp.AIPL1, with improved visual acuity, functional vision, and protection against retinal degeneration[158](index=158&type=chunk) - All **11** LCA4 children treated to date (including **7** bilaterally) showed meaningful responses[158](index=158&type=chunk) - The company is preparing to submit a Marketing Authorization Application (MAA) under exceptional circumstances to the MHRA based on these results, with no further clinical data required[158](index=158&type=chunk) - Positive engagement with the FDA has led to alignment on clinical, non-clinical, and CMC requirements for potential expedited approval in the US[158](index=158&type=chunk) [Botaretigene Sparoparvovec for the Treatment of X-linked Retinitis Pigmentosa (XLRP)](index=53&type=section&id=Botaretigene%20Sparoparvovec%20for%20the%20Treatment%20of%20X-linked%20Retinitis%20Pigmentosa%20(XLRP)) This section summarizes the Phase 3 LUMEOS trial data for botaretigene sparoparvovec (bota-vec) in XLRP, highlighting its efficacy and safety profile, and potential future revenues - Phase 3 LUMEOS trial data for botaretigene sparoparvovec (bota-vec) in XLRP showed the primary endpoint (Visual Mobility Assessment) was not met but was directionally supportive[158](index=158&type=chunk) - All but one secondary endpoint, across functional vision, retinal function, and visual function domains, showed benefit with **95%** CI intervals not including 0[158](index=158&type=chunk) - **40%** of treated patients (**22/55**) showed improvement in ≥2 endpoints in different vision domains, compared to **0%** in the control group[158](index=158&type=chunk) - The safety profile was as expected, with >**90%** of treatment-related inflammation adverse events being mild[158](index=158&type=chunk) - MeiraGTx is eligible to receive up to **$285 million** upon first commercial sales of bota-vec in the US and EU and manufacturing tech transfer, and anticipates additional revenue from a commercial supply agreement with Johnson & Johnson Innovative Medicine[158](index=158&type=chunk) [Riboswitch Gene Regulation Technology Platform for in vivo Delivery](index=55&type=section&id=Riboswitch%20Gene%20Regulation%20Technology%20Platform%20for%20in%20vivo%20Delivery) This section describes the progress of the company's riboswitch technology platform, focusing on its application in obesity, metabolic disease, neuropathic pain, and CAR-T - The company is progressing its riboswitch technology platform with an initial focus on obesity and metabolic disease, neuropathic pain, and CAR-T[160](index=160&type=chunk) - Compelling preclinical data with metabolic peptides and hormones suggest greater efficacy on weight loss and positive impact on fat-to-muscle ratio with novel peptide combinations[160](index=160&type=chunk) - First-in-human studies using the riboswitch platform are intended to be initiated in 2025[160](index=160&type=chunk) [Manufacturing](index=55&type=section&id=Manufacturing) This section details the regulatory authorizations and capabilities of the company's manufacturing facilities in the UK and Ireland, highlighting their GMP compliance and readiness for clinical and commercial production - The UK manufacturing facility holds two MHRA authorizations (MIA(IMP) and Specials Licence) and successfully renewed its licenses in May 2024, confirming GMP compliance for IMPs and readiness for commercial MIA[161](index=161&type=chunk) - The Shannon, Ireland facility holds two HPRA authorizations (MIA Licence for QC testing of commercial products and MIA(IMP) Licence for QC testing of IMPs)[162](index=162&type=chunk)[165](index=165&type=chunk) - A February 2025 HPRA inspection renewed QC licenses and added viral vector manufacturing to the MIA(IMP) license, making Shannon the first gene therapy facility in Ireland authorized to manufacture clinical trial material[166](index=166&type=chunk) [Components of Our Results of Operations](index=57&type=section&id=Components%20of%20Our%20Results%20of%20Operations) This section defines the various components of the company's financial results, including service revenue, operating expenses (cost of service revenue, general and administrative, research and development), and other non-operating income/expense items (foreign currency, interest, gain on asset sale), as well as critical accounting policies - Service revenue is derived from process performance qualification (PPQ) services related to the Asset Purchase Agreement[167](index=167&type=chunk) - Operating expenses include cost of service revenue (for PPQ services), general and administrative expenses (salaries, legal, professional fees), and research and development expenses (employee costs, clinical/preclinical studies, manufacturing facilities)[168](index=168&type=chunk)[169](index=169&type=chunk)[170](index=170&type=chunk)[172](index=172&type=chunk) - Other non-operating income/expense includes foreign currency gains/losses, interest income/expense, and gains on sale of nonfinancial assets[177](index=177&type=chunk)[179](index=179&type=chunk)[180](index=180&type=chunk)[181](index=181&type=chunk) - The company's critical accounting policies involve significant estimates in areas such as service revenue, share-based compensation, and accrued expenses[183](index=183&type=chunk) [Results of Operations](index=61&type=section&id=Results%20of%20Operations) This section provides a detailed comparison of the company's financial performance for the three months ended March 31, 2025, and 2024, highlighting changes in revenue, operating expenses, and non-operating items that led to the net loss | Metric (in thousands) | 2025 | 2024 | Change | | :-------------------- | :---------- | :---------- | :---------- | | Service revenue - related party | $1,926 | $697 | $1,229 | | Total operating expenses | $43,522 | $47,469 | $(3,947) | | Loss from operations | $(41,596) | $(46,772) | $5,176 | | Foreign currency gain (loss) | $3,687 | $(535) | $4,222 | | Gain on sale of nonfinancial assets | $0 | $29,018 | $(29,018) | | Net loss | $(39,981) | $(20,442) | $(19,539) | - Service revenue increased by **$1.2 million** to **$1.9 million** in Q1 2025, driven by increased progress in PPQ services[186](index=186&type=chunk) - Cost of service revenue was **$1.4 million** in Q1 2025, with no comparable cost in Q1 2024, reflecting the initiation of PPQ services[187](index=187&type=chunk) - General and administrative expenses decreased by **$3.8 million** to **$9.4 million** in Q1 2025, primarily due to lower share-based compensation, legal/accounting fees, and a gain from lease termination[188](index=188&type=chunk) - Research and development expenses decreased by **$1.5 million** to **$32.8 million** in Q1 2025, mainly due to lower manufacturing costs and other R&D expenses, partially offset by reduced Johnson & Johnson Innovative Medicine reimbursements and increased AAV-hAQP1 program costs[193](index=193&type=chunk) - A foreign currency gain of **$3.7 million** was recorded in Q1 2025, a **$4.2 million** positive change from a **$0.5 million** loss in Q1 2024, due to the weakening U.S. dollar[194](index=194&type=chunk) - The absence of a **$29.0 million** gain on sale of nonfinancial assets (recognized in Q1 2024) was the primary driver of the increased net loss in Q1 2025[197](index=197&type=chunk) [Liquidity and Capital Resources](index=64&type=section&id=Liquidity%20and%20Capital%20Resources) This section discusses the company's cash position, historical funding sources, future capital requirements, and cash flow activities for the three months ended March 31, 2025, and 2024 - As of March 31, 2025, the company had **$68.6 million** in cash, cash equivalents, and restricted cash[202](index=202&type=chunk) - The company expects its current cash, receivables, and anticipated proceeds from the Hologen collaboration to fund operating expenses and capital expenditures into 2027 and cover its **$75.0 million** debt obligation due in August 2026[200](index=200&type=chunk) - Net cash used in operating activities was **$36.8 million** in Q1 2025, consistent with Q1 2024, primarily due to net losses and R&D expenses[203](index=203&type=chunk)[204](index=204&type=chunk) - Net cash used in investing activities was **$1.7 million** in Q1 2025 for property and equipment, a shift from **$27.3 million** provided in Q1 2024 due to proceeds from the sale of nonfinancial assets[205](index=205&type=chunk) - Net cash provided by financing activities was **$1.7 million** in Q1 2025, mainly from an at-the-market offering, compared to **$1.3 million** used in Q1 2024[207](index=207&type=chunk)[208](index=208&type=chunk) - The company has no off-balance sheet arrangements[209](index=209&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=68&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section updates the company's market risk disclosures, focusing on foreign currency exchange risk and interest rate risk - The company is exposed to foreign currency exchange rate fluctuations, primarily between the U.S. Dollar, British pound sterling, and euro[211](index=211&type=chunk) - A hypothetical **10%** unfavorable movement in foreign currency exchange rates would result in an additional foreign currency loss of approximately **$11.0 million** for Q1 2025[211](index=211&type=chunk) - The company is exposed to interest rate risk from its Notes Purchase Agreement, which bears a fluctuating interest rate (**14.34%** at March 31, 2025)[212](index=212&type=chunk) - A hypothetical **1%** increase in the Secured Overnight Financing Rate (SOFR) would increase annual interest expense by approximately **$0.8 million**[212](index=212&type=chunk) [Item 4. Controls and Procedures](index=69&type=section&id=Item%204.%20Controls%20and%20Procedures) This section addresses the effectiveness of the company's disclosure controls and procedures and reports on any changes in internal control over financial reporting - Management, including the CEO and CFO, concluded that the company's disclosure controls and procedures were effective at a reasonable assurance level as of March 31, 2025[214](index=214&type=chunk) - No changes in internal control over financial reporting occurred during Q1 2025 that materially affected, or are reasonably likely to materially affect, internal control over financial reporting[215](index=215&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=70&type=section&id=Item%201.%20Legal%20Proceedings) This section states that the company is not currently subject to any material legal proceedings - The company is not subject to any material legal proceedings[217](index=217&type=chunk) [ITEM 1A. RISK FACTORS](index=70&type=section&id=ITEM%201A.%20RISK%20FACTORS) This comprehensive section outlines various risks that could materially and adversely affect the company's business, financial condition, results of operations, and future growth prospects, categorized by financial position, development, regulatory approval, commercialization, third-party dependence, intellectual property, employee matters, and ordinary shares [Risks Related to Our Financial Position and Need for Additional Capital](index=70&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) This section details risks associated with the company's financial stability, including historical losses, reliance on future payments, and the need for additional capital - The company has incurred significant losses since inception (**$742.0 million** accumulated deficit as of March 31, 2025) and anticipates continued losses, with no guarantee of achieving or maintaining profitability[219](index=219&type=chunk) - There is no guarantee of timely receipt of additional milestone payments (up to **$285.0 million** remaining) under the Asset Purchase Agreement with Johnson & Johnson Innovative Medicine or revenues from the Supply Agreement, as these depend on Johnson & Johnson's development and commercialization success[223](index=223&type=chunk)[224](index=224&type=chunk)[225](index=225&type=chunk) - The company will require additional capital to fund operations, which may not be available on acceptable terms, leading to potential delays or termination of R&D programs and dilution for shareholders if raised through equity[226](index=226&type=chunk)[229](index=229&type=chunk) - Insufficient cash flows or non-compliance with debt covenants under the Notes Purchase Agreement could lead to acceleration of debt obligations, materially harming the business[231](index=231&type=chunk)[232](index=232&type=chunk)[233](index=233&type=chunk) - The review of potential strategic transactions, such as the Hologen collaboration, may not result in anticipated benefits or could be disruptive to business operations[234](index=234&type=chunk) [Risks Related to Discovery, Development, Clinical Testing, Manufacturing and Regulatory Approval](index=81&type=section&id=Risks%20Related%20to%20Discovery,%20Development,%20Clinical%20Testing,%20Manufacturing%20and%20Regulatory%20Approval) This section outlines risks inherent in the discovery, development, clinical testing, manufacturing, and regulatory approval processes for the company's genetic medicines - The company is heavily dependent on the success of its product candidates (AAV-hAQP1, AAV-GAD, AAV-AIPL1, riboswitch platform), which are still in development, and failure to receive regulatory approval or commercialize them would harm the business[235](index=235&type=chunk) - Predicting the time and cost of gene therapy product development is difficult due to the novelty of the platform and the limited number of approved gene therapies, with potential for unexpected adverse events or toxicities[238](index=238&type=chunk)[240](index=240&type=chunk) - The regulatory landscape for gene therapy is uncertain and evolving, making it difficult to predict the time and cost of obtaining regulatory approval, which may be lengthy and expensive[242](index=242&type=chunk)[246](index=246&type=chunk) - Clinical trials are expensive, time-consuming, and involve uncertain outcomes, with potential for substantial delays due to patient enrollment, regulatory disagreements, or manufacturing issues[248](index=248&type=chunk)[249](index=249&type=chunk)[252](index=252&type=chunk) - The affected patient populations for product candidates may be smaller than projected, impacting addressable markets and financial results[264](index=264&type=chunk)[265](index=265&type=chunk) - Negative public opinion of gene therapy or increased regulatory scrutiny could adversely impact public perception, clinical trial enrollment, and commercialization[266](index=266&type=chunk)[267](index=267&type=chunk) - Failure to maintain or obtain regulatory designations (e.g., RMAT, Fast Track, Orphan Drug, Rare Pediatric Disease Designation) or their associated benefits could delay development or commercialization, and such designations do not guarantee approval[271](index=271&type=chunk)[273](index=273&type=chunk)[281](index=281&type=chunk)[284](index=284&type=chunk) - Manufacturing facilities and contract manufacturers are subject to significant regulation (GMP), and failure to meet requirements or limited capacity could lead to delays, increased costs, or regulatory sanctions[286](index=286&type=chunk)[288](index=288&type=chunk)[291](index=291&type=chunk) - Product candidates may cause serious adverse events or undesirable side effects, leading to clinical trial delays/halts, restrictive labeling, or denial of regulatory approval[296](index=296&type=chunk)[300](index=300&type=chunk) - Success in preclinical studies or early clinical trials is not indicative of future results, and the regulatory approval processes are lengthy, unpredictable, and subject to change[302](index=302&type=chunk)[304](index=304&type=chunk)[306](index=306&type=chunk)[308](index=308&type=chunk) - Interim or preliminary clinical trial data may change upon comprehensive review, potentially leading to different conclusions or impacting regulatory approval[317](index=317&type=chunk)[320](index=320&type=chunk) [Risks Related to Healthcare Laws and Other Legal Compliance Matters](index=114&type=section&id=Risks%20Related%20to%20Healthcare%20Laws%20and%20Other%20Legal%20Compliance%20Matters) This section addresses risks related to compliance with complex and evolving healthcare laws, data privacy regulations, environmental laws, and international anti-corruption laws - Enacted and future healthcare legislation (e.g., ACA, IRA) may increase the difficulty and cost of obtaining marketing approval and commercializing product candidates, and may affect pricing[324](index=324&type=chunk)[328](index=328&type=chunk)[331](index=331&type=chunk) - Business operations are subject to various healthcare regulatory laws (e.g., Anti-Kickback Statute, False Claims Act, HIPAA, Physician Payments Sunshine Act), and non-compliance could lead to significant penalties[340](index=340&type=chunk)[342](index=342&type=chunk)[344](index=344&type=chunk) - The company is subject to complex and evolving data privacy and protection laws (e.g., GDPR, UK GDPR, CCPA), and failure to comply could result in substantial costs, fines, and reputational harm[343](index=343&type=chunk)[348](index=348&type=chunk)[350](index=350&type=chunk)[351](index=351&type=chunk) - Operations are subject to environmental, health, and safety laws, with potential for liability and substantial expenses in connection with compliance or remediation activities[352](index=352&type=chunk)[353](index=353&type=chunk) - International operations expose the company to anti-corruption laws (e.g., UK Bribery Act, FCPA) and Trade Control laws, with non-compliance potentially leading to civil or criminal penalties[354](index=354&type=chunk)[356](index=356&type=chunk)[357](index=357&type=chunk) [Risks Related to Commercialization](index=128&type=section&id=Risks%20Related%20to%20Commercialization) This section covers risks associated with commercializing product candidates, including competition, market acceptance, reimbursement, and international market challenges - The company faces significant competition from major pharmaceutical, biotechnology, and gene therapy companies, with competitors potentially achieving regulatory approval sooner or developing safer/more effective therapies[359](index=359&type=chunk)[361](index=361&type=chunk)[363](index=363&type=chunk) - Successful commercialization depends on governmental authorities and health insurers establishing coverage, adequate reimbursement levels, and favorable pricing policies; failure to obtain these could limit market access and revenue[364](index=364&type=chunk)[365](index=365&type=chunk)[369](index=369&type=chunk) - Even with regulatory approval, product candidates may fail to achieve market acceptance by physicians, patients, or third-party payors, impacting product revenues and profitability[371](index=371&type=chunk)[373](index=373&type=chunk) - Inability to establish sales, marketing, and distribution capabilities, either independently or through collaborations, could hinder commercialization efforts and delay product launch[374](index=374&type=chunk)[375](index=375&type=chunk)[378](index=378&type=chunk) - Commercialization outside the US, UK, or EU involves risks such as differing regulatory requirements, intellectual property protection, foreign currency fluctuations, and political instability[381](index=381&type=chunk) - Product candidates approved as biologics may face competition sooner than anticipated from biosimilars, potentially reducing market exclusivity and competitive advantage[386](index=386&type=chunk)[387](index=387&type=chunk) [Risks Related to Our Dependence on Third Parties](index=138&type=section&id=Risks%20Related%20to%20Our%20Dependence%20on%20Third%20Parties) This section addresses risks arising from the company's reliance on third-party manufacturers, collaborators, and contract research organizations for various aspects of its business - If internal GMP manufacturing facilities cannot meet supply needs, the company will rely on third-party manufacturers, increasing risks of insufficient quantities, unacceptable costs, and delays in development or commercialization[388](index=388&type=chunk)[389](index=389&type=chunk)[391](index=391&type=chunk) - Failure to establish and maintain collaborative relationships for product development, manufacturing, and commercialization could significantly limit the ability to successfully develop and commercialize product candidates[394](index=394&type=chunk)[397](index=397&type=chunk) - Reliance on third parties (CROs, clinical trial sites) to conduct preclinical studies and clinical trials poses risks if they perform unsatisfactorily, leading to delays, increased costs, or compromised data quality[398](index=398&type=chunk)[401](index=401&type=chunk) [Risks Related to Intellectual Property](index=144&type=section&id=Risks%20Related%20to%20Intellectual%20Property) This section details risks concerning the company's intellectual property, including dependence on licenses, patent protection, infringement claims, and trade secret safeguarding - Dependence on licensed proprietary technology means loss of existing licenses or inability to acquire new ones could prevent continued product candidate development[403](index=403&type=chunk) - Failure to obtain and maintain broad patent protection for technology and product candidates could impair competitive effectiveness, as the patent prosecution process is expensive, time-consuming, and uncertain[407](index=407&type=chunk)[408](index=408&type=chunk) - Third parties may assert infringement claims, or the company may need to defend/enforce its patents, leading to substantial costs, delays, or loss of proprietary rights[413](index=413&type=chunk)[417](index=417&type=chunk)[419](index=419&type=chunk) - Incorrect identification or interpretation of third-party patents could lead to infringement claims and negatively impact development and marketing[420](index=420&type=chunk)[422](index=422&type=chunk)[423](index=423&type=chunk) - Changes in patent laws or jurisprudence (e.g., AIA, Supreme Court rulings) could diminish patent value and protection for product candidates[424](index=424&type=chunk)[427](index=427&type=chunk) - Limited geographical patent protection and inadequate patent terms may expose the company to competition and limit its competitive position[428](index=428&type=chunk)[430](index=430&type=chunk) - Proprietary rights may not adequately protect technologies, and reliance on third parties increases the risk of trade secret misappropriation or disclosure[434](index=434&type=chunk)[437](index=437&type=chunk)[438](index=438&type=chunk) - Inadequate protection of trademarks and trade names could hinder brand recognition and adversely affect the business[439](index=439&type=chunk) - The need to license or acquire additional intellectual property from third parties, which may not be available or on commercially reasonable terms, could harm the business[440](index=440&type=chunk)[441](index=441&type=chunk) [Risks Related to Employee Matters and Managing Growth](index=160&type=section&id=Risks%20Related%20to%20Employee%20Matters%20and%20Managing%20Growth) This section addresses risks related to human capital, including managing organizational changes, retaining key personnel, potential product liability, and employee misconduct - The company may face difficulties in managing organizational changes, including hiring and integrating new personnel or reducing staff, which could disrupt operations and divert management attention[443](index=443&type=chunk) - Future success depends on retaining key personnel (e.g., CEO, COO, CDO) and attracting/motivating qualified new hires, which is challenging in a competitive industry[444](index=444&type=chunk)[445](index=445&type=chunk)[447](index=447&type=chunk) - Potential product liability lawsuits could result in substantial liabilities, costs, and limit commercialization, while insurance may not cover all risks or be sufficient[448](index=448&type=chunk)[449](index=449&type=chunk) - Employee or contractor misconduct (e.g., non-compliance with regulations, fraudulent data) could harm the business through regulatory sanctions, reputational damage, and legal actions[453](index=453&type=chunk) [Risks Related to Our Ordinary Shares](index=168&type=section&id=Risks%20Related%20to%20Our%20Ordinary%20Shares) This section outlines risks specific to the company's ordinary shares, including market price volatility, dilution, shareholder influence, and tax implications for investors - The market price of ordinary shares may be volatile due to various factors, including clinical trial results, regulatory developments, competition, and general market conditions, potentially leading to substantial losses for investors[461](index=461&type=chunk)[462](index=462&type=chunk) - Raising additional capital through equity offerings (including at-the-market programs) could substantially dilute existing shareholders' investment[463](index=463&type=chunk) - Executive officers, directors, and principal shareholders collectively hold approximately **56.0%** of outstanding ordinary shares, giving them significant influence over shareholder matters and potentially discouraging change of control[464](index=464&type=chunk)[467](index=467&type=chunk) - As a 'smaller reporting company,' reduced disclosure requirements may make ordinary shares less attractive to some investors, potentially leading to a less active trading market and more volatile share price[468](index=468&type=chunk)[469](index=469&type=chunk) - Difficulties may arise in enforcing foreign judgments against management or the company due to directors/assets being outside the US and differences in Cayman Islands law compared to US corporate law[471](index=471&type=chunk)[473](index=473&type=chunk) - The company expects to be treated as a UK tax resident but may be a dual resident, potentially restricting UK tax reliefs[475](index=475&type=chunk)[476](index=476&type=chunk) - Classification as a Passive Foreign Investment Company (PFIC) for US federal income tax purposes could result in adverse tax consequences for US investors[477](index=477&type=chunk) - US holders owning ≥**10%** of ordinary shares may be subject to adverse US federal income tax consequences related to Controlled Foreign Corporations (CFCs)[478](index=478&type=chunk) [General Risk Factors](index=178&type=section&id=General%20Risk%20Factors) This section covers broad risks that could impact the company, including those related to acquisitions, exchange rate fluctuations, management discretion over funds, public company costs, analyst coverage, ESG expectations, and dividend policy - Acquisitions could disrupt business, dilute shareholders, or reduce financial resources if not executed favorably or integrated successfully[484](index=484&type=chunk) - Exchange rate fluctuations, particularly between the U.S. dollar, pound sterling, and euro, may adversely affect results of operations and financial condition[485](index=485&type=chunk) - Management has broad discretion over the use of financing proceeds, and investments may not yield favorable returns or align with shareholder preferences[486](index=486&type=chunk) - Operating as a public company incurs substantial costs and requires significant management time for compliance initiatives (e.g., Sarbanes-Oxley Act, Nasdaq requirements)[487](index=487&type=chunk)[488](index=488&type=chunk) - Cessation of analyst coverage or adverse/misleading opinions could negatively impact share price and trading volume[489](index=489&type=chunk) - Expectations related to environmental, social, and governance (ESG) factors may impose additional costs, expose the company to new risks, and affect investor perception[490](index=490&type=chunk)[492](index=492&type=chunk) - The company does not anticipate paying cash dividends in the foreseeable future, making capital appreciation the sole source of gain for shareholders[493](index=493&type=chunk) [Item 2. Unregistered Sales of Equity Securities, Use of Proceeds and Issuer Purchases of Equity Securities.](index=182&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities,%20Use%20of%20Proceeds%20and%20Issuer%20Purchases%20of%20Equity%20Securities.) This section indicates that there were no unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities during the reporting period - No unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities occurred during the period[494](index=494&type=chunk) [Item 3. Defaults Upon Senior Securities.](index=182&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities.) This section states that there were no defaults upon senior securities during the reporting period - No defaults upon senior securities occurred during the period[495](index=495&type=chunk) [Item 4. Mine Safety Disclosures.](index=182&type=section&id=Item%204.%20Mine%20Safety%20Disclosures.) This section indicates that mine safety disclosures are not applicable to the company - Mine safety disclosures are not applicable[496](index=496&type=chunk) [Item 5. Other Information.](index=182&type=section&id=Item%205.%20Other%20Information.) This section confirms that no directors or officers adopted, modified, or terminated Rule 10b5-1 or non-Rule 10b5-1 trading arrangements during the three months ended March 31, 2025 - No directors or officers adopted, modified, or terminated Rule 10b5-1 or non-Rule 10b5-1 trading arrangements during Q1 2025[497](index=497&type=chunk) [Item 6. Exhibits.](index=183&type=section&id=Item%206.%20Exhibits.) This section lists the exhibits filed with the Form 10-Q, including a lease agreement, certifications of principal executive and financial officers, and XBRL-related documents - Exhibits include a lease agreement, certifications of principal executive and financial officers (pursuant to Sections 302 and 906 of Sarbanes-Oxley Act), and Inline XBRL documents[498](index=498&type=chunk) [SIGNATURES](index=184&type=section&id=SIGNATURES) This section contains the signatures of the registrant's authorized officers, Alexandria Forbes (CEO) and Richard Giroux (CFO and COO), certifying the report - The report is signed by Alexandria Forbes, Chief Executive Officer, and Richard Giroux, Chief Financial Officer and Chief Operating Officer, on May 13, 2025[502](index=502&type=chunk)

[Executive Summary / Q1 2025 Highlights](index=1&type=section&id=Executive%20Summary%20%2F%20Q1%202025%20Highlights) [Strategic Collaboration with Hologen AI](index=1&type=section&id=Strategic%20Collaboration%20with%20Hologen%20AI) MeiraGTx formed a strategic collaboration with Hologen AI, receiving a $200 million upfront payment and establishing Hologen Neuro AI Ltd, with Hologen committing an additional $230 million for AAV-GAD Parkinson's disease development - MeiraGTx received a **$200 million cash upfront payment** from Hologen AI[1](index=1&type=chunk)[3](index=3&type=chunk)[4](index=4&type=chunk) - A joint venture, Hologen Neuro AI Ltd, was established, with Hologen committing an **additional $230 million** to accelerate AAV-GAD development for Parkinson's disease[1](index=1&type=chunk)[3](index=3&type=chunk)[4](index=4&type=chunk) - MeiraGTx will hold a **30% equity stake** in the joint venture and lead all clinical development and manufacturing[4](index=4&type=chunk) [Regulatory and Clinical Milestones](index=1&type=section&id=Regulatory%20and%20Clinical%20Milestones) The company achieved significant Q1 2025 regulatory and clinical progress, including AAV-GAD RMAT designation, AAV2-hAQP1 Phase 2 AQUAx2 FDA alignment, and positive AAV-AIPL1 LCA4 data published in The Lancet with planned MAA submission - AAV-GAD received **FDA Regenerative Medicine Advanced Therapy (RMAT) designation** for Parkinson's disease treatment[1](index=1&type=chunk)[2](index=2&type=chunk)[10](index=10&type=chunk) - The Phase 2 AQUAx2 study for AAV2-hAQP1 in radiation-induced xerostomia (RIX) reached **FDA alignment** to support a potential BLA submission[1](index=1&type=chunk)[2](index=2&type=chunk)[11](index=11&type=chunk) - Efficacy data for rAAV8.hRKp.AIPL1 in AIPL1-related retinal dystrophy (LCA4) was published in The Lancet, showing **meaningful responses in all 11 treated children**[1](index=1&type=chunk)[15](index=15&type=chunk) - The company plans to submit a **conditional Marketing Authorization Application (MAA)** for AAV-AIPL1 to the UK MHRA and discuss similar US approval pathways with the FDA[1](index=1&type=chunk)[15](index=15&type=chunk) [Recent Development Highlights](index=2&type=section&id=Recent%20Development%20Highlights) [AAV-GAD for Parkinson's Disease](index=3&type=section&id=AAV-GAD%20for%20Parkinson's%20Disease) AAV-GAD received FDA RMAT designation based on three positive clinical studies, including two double-blind, sham-controlled Phase 2 trials showing significant motor benefits and AI-driven evidence of disease-modifying changes - AAV-GAD received **FDA RMAT designation on May 8, 2025**, for Parkinson's disease inadequately controlled by anti-Parkinsonian medications[10](index=10&type=chunk) - RMAT designation was based on **three positive clinical studies**, including one Phase 1 and two double-blind, sham-controlled Phase 2 trials[10](index=10&type=chunk) - Phase 2 study data demonstrated **significant clinical benefit** in the UPDRS Part 3 standard motor endpoint for AAV-GAD[10](index=10&type=chunk) - Hologen's AI analysis confirmed AAV-GAD treatment leads to **disease-modifying changes in Parkinson's brain circuits** and potential protective changes in substantia nigra and cognitive/emotional brain regions[3](index=3&type=chunk)[10](index=10&type=chunk) - The company plans to initiate a **Phase 3 study for AAV-GAD in H2 2025**[10](index=10&type=chunk) [AAV2-hAQP1 for the Treatment of Xerostomia](index=3&type=section&id=AAV2-hAQP1%20for%20the%20Treatment%20of%20Xerostomia) AAV2-hAQP1 received FDA RMAT designation in December 2024 for Grade 2/3 radiation-induced xerostomia; the company aligned with FDA on Phase 2 AQUAx2 study requirements, targeting Q4 2025 enrollment completion and potential BLA submission by late 2026 - AAV2-hAQP1 received **FDA RMAT designation in December 2024** for Grade 2/3 radiation-induced xerostomia (RIX)[11](index=11&type=chunk) - The company reached **FDA alignment** on clinical and CMC requirements for the ongoing Phase 2 AQUAx2 study to support a potential BLA submission[11](index=11&type=chunk) - The Phase 2 AQUAx2 study is actively enrolling and dosing across multiple centers in the US, Canada, and UK, targeting **enrollment completion in Q4 2025**[11](index=11&type=chunk)[12](index=12&type=chunk) - A **potential BLA submission is anticipated by late 2026**[12](index=12&type=chunk) [AAV-AIPL1 for LCA4 Caused by Mutations in the AIPL1 Gene](index=4&type=section&id=AAV-AIPL1%20for%20LCA4%20Caused%20by%20Mutations%20in%20the%20AIPL1%20Gene) Positive AAV-AIPL1 efficacy data for LCA4, showing significant improvement in all 11 treated children, was published in The Lancet; the company is preparing a conditional MAA for MHRA and discussing accelerated US approval with FDA, having secured Orphan Drug and Rare Pediatric Disease designations - In February 2025, The Lancet published efficacy data for rAAV8.hRKp.AIPL1 in LCA4, showing **meaningful responses in all 11 treated children**[15](index=15&type=chunk) - The company is preparing to submit a **conditional Marketing Authorization Application (MAA)** to the MHRA, requiring no further clinical data[15](index=15&type=chunk) - Alignment has been reached with the FDA on clinical, non-clinical, and CMC requirements to advance **potential accelerated approval in the US**[15](index=15&type=chunk) - AAV-AIPL1 has received **US Orphan Drug designation, EU Orphan designation, and FDA Rare Pediatric Disease designation**[15](index=15&type=chunk) [Botaretigene Sparoparvovec for the Treatment of X-linked Retinitis Pigmentosa (XLRP)](index=4&type=section&id=Botaretigene%20Sparoparvovec%20for%20the%20Treatment%20of%20X-linked%20Retinitis%20Pigmentosa%20(XLRP)) Botaretigene sparoparvovec (bota-vec) Phase 3 LUMEOS trial missed its primary endpoint but showed directional support and benefits across most secondary endpoints; the therapy is safe, has FDA Fast Track and Orphan Drug, and EU PRIME and Orphan Drug designations, with MeiraGTx eligible for up to $285 million in milestone payments - The Phase 3 LUMEOS trial for botaretigene sparoparvovec (bota-vec) **missed its primary endpoint** (visual mobility assessment) but showed directional support[15](index=15&type=chunk) - All secondary endpoints, except one, including **functional vision, retinal function, and visual function**, demonstrated benefit[15](index=15&type=chunk)[18](index=18&type=chunk) - Bota-vec's safety profile was **as expected and manageable**, with no new safety signals and over 90% of treatment-related inflammatory adverse events being mild[18](index=18&type=chunk) - Bota-vec has received **FDA Fast Track and Orphan Drug designations**, along with EU Priority Medicines (PRIME) and Orphan Drug designations[18](index=18&type=chunk) - MeiraGTx is eligible for **up to $285 million in milestone payments** upon bota-vec's first commercial sale and manufacturing technology transfer[18](index=18&type=chunk) [Riboswitch Gene Regulation Technology Platform for in vivo Delivery](index=5&type=section&id=Riboswitch%20Gene%20Regulation%20Technology%20Platform%20for%20in%20vivo%20Delivery) MeiraGTx is advancing its riboswitch technology platform, initially targeting obesity, metabolic diseases, neuropathic pain, and CAR-T therapies, with compelling preclinical data and plans to initiate first-in-human studies in 2025 - The company is advancing its riboswitch technology platform, initially focusing on **obesity and metabolic diseases, neuropathic pain, and CAR-T**[18](index=18&type=chunk) - Preclinical data for metabolic peptides and hormones (including incretins, amylin, and leptin) showed **greater efficacy in weight loss and fat-to-lean muscle ratio**[18](index=18&type=chunk) - The company plans to initiate **first-in-human studies using the riboswitch platform in 2025**[18](index=18&type=chunk) [Manufacturing Capabilities](index=6&type=section&id=Manufacturing%20Capabilities) [United Kingdom (MeiraGTx UK II Ltd.)](index=6&type=section&id=United%20Kingdom%20(MeiraGTx%20UK%20II%20Ltd.)) MeiraGTx's UK manufacturing facility holds two MHRA authorizations (MIA(IMP) and Special Licence), successfully renewed in May 2024, confirming GMP compliance and supporting commercial MIA license applications - The UK manufacturing facility holds **MIA(IMP) and Special Licence authorizations** from the MHRA[19](index=19&type=chunk)[23](index=23&type=chunk) - Following a May 2024 inspection, licenses were **successfully renewed**, confirming GMP compliance and supporting commercial MIA license applications[19](index=19&type=chunk) [Ireland (MeiraGTx Ireland DAC)](index=6&type=section&id=Ireland%20(MeiraGTx%20Ireland%20DAC)) MeiraGTx's Shannon facility holds two HPRA authorizations (MIA and MIA(IMP)), with its QC lab actively conducting PPQ batch release and stability testing; a successful February 2025 HPRA inspection renewed two QC licenses and added viral vector manufacturing to the MIA(IMP) license, a first for an Irish gene therapy facility - The Shannon facility in Ireland holds **MIA and MIA(IMP) licenses** from the HPRA[20](index=20&type=chunk)[24](index=24&type=chunk) - The QC laboratory actively conducts **release and stability testing for PPQ batches**[20](index=20&type=chunk) - A successful February 2025 HPRA inspection renewed two QC licenses and added **viral vector manufacturing to the MIA(IMP) license**, a first for an Irish gene therapy facility[21](index=21&type=chunk) [Financial Results](index=6&type=section&id=Financial%20Results) [Cash Position and Liquidity](index=6&type=section&id=Cash%20Position%20and%20Liquidity) As of March 31, 2025, MeiraGTx's cash and cash equivalents were approximately $66.5 million, down from $120.3 million on March 31, 2024; the company expects existing funds, combined with Hologen collaboration proceeds, to support operations and capital expenditures through 2027 and repay $75 million debt due August 2026 Cash, Cash Equivalents, and Restricted Cash | Metric | March 31, 2025 (Millions of USD) | March 31, 2024 (Millions of USD) | | :--- | :--- | :--- | | Cash, Cash Equivalents, and Restricted Cash | 68.6 | 120.3 | - As of March 31, 2025, cash and cash equivalents were approximately **$66.5 million**[22](index=22&type=chunk) - The company anticipates that existing capital, combined with proceeds from the Hologen collaboration, will be **sufficient to fund operations and capital expenditures through 2027** and repay **$75 million debt due August 2026**[22](index=22&type=chunk) [Statement of Operations (Income Statement)](index=7&type=section&id=Statement%20of%20Operations%20(Income%20Statement)) In Q1 2025, the company saw year-over-year service revenue growth but a decrease in total operating expenses; net loss increased from $20.4 million in Q1 2024 to $39.98 million, primarily due to the absence of non-financial asset sale gains from the prior year Key Financial Data for Q1 2025 (Compared to Q1 2024) | Metric | March 31, 2025 (Thousands of USD) | March 31, 2024 (Thousands of USD) | Change (Thousands of USD) | Change Rate | | :--- | :--- | :--- | :--- | :--- | | Service revenue - related party | 1,926 | 697 | 1,229 | +176.3% | | Cost - related party service revenue | 1,378 | — | 1,378 | N/A | | General and administrative expenses | 9,364 | 13,147 | (3,783) | -28.8% | | Research and development expenses | 32,780 | 34,322 | (1,542) | -4.5% | | Operating loss | (41,596) | (46,772) | 5,176 | -11.1% | | Foreign currency exchange gain (loss) | 3,687 | (535) | 4,222 | N/A | | Interest income | 971 | 1,097 | (126) | -11.5% | | Interest expense | (3,043) | (3,250) | 207 | -6.4% | | Gain on sale of non-financial assets | — | 29,018 | (29,018) | -100.0% | | Net loss | (39,981) | (20,442) | (19,539) | +95.6% | | Basic and diluted net loss per share | (0.51) | (0.32) | (0.19) | +59.4% | | Weighted average common shares outstanding | 79,032,341 | 64,065,895 | 14,966,446 | +23.4% | - Service revenue increased by **$1.2 million**, primarily due to accelerated progress on process performance qualification (PPQ) services under the asset purchase agreement with Johnson & Johnson Innovative Medicine[27](index=27&type=chunk) - General and administrative expenses decreased by **$3.8 million**, mainly due to lower share-based compensation, legal and accounting fees, consulting fees, and other office-related costs, along with a gain from early termination of a lease agreement, partially offset by increased salaries and related costs[29](index=29&type=chunk) - Research and development expenses decreased by **$1.5 million**, primarily due to reclassification of manufacturing costs and reduced R&D expenses, partially offset by lower reimbursements from Johnson & Johnson Innovative Medicine, increased clinical trial expenses for the AAV-hAQP1 program and other ophthalmology programs, and higher preclinical program expenses[30](index=30&type=chunk) - Foreign currency exchange shifted from a loss to a **gain**, primarily due to the weakening of the US dollar against the British Pound and Euro[31](index=31&type=chunk) - Gain on sale of non-financial assets decreased from **$29 million to $0**, leading to a significant increase in net loss[35](index=35&type=chunk)[36](index=36&type=chunk) [Balance Sheet Highlights](index=12&type=section&id=Balance%20Sheet%20Highlights) As of March 31, 2025, total assets were $224 million, a decrease from $269.75 million on December 31, 2024; total liabilities were $190.9 million, and total stockholders' equity was $33.1 million Key Balance Sheet Data (As of March 31, 2025 vs. December 31, 2024) | Metric | March 31, 2025 (Thousands of USD) | December 31, 2024 (Thousands of USD) | | :--- | :--- | :--- | | Cash and cash equivalents | 66,523 | 103,659 | | Total current assets | 79,889 | 123,518 | | Total assets | 224,014 | 269,751 | | Total current liabilities | 52,372 | 60,783 | | Total liabilities | 190,915 | 201,924 | | Total stockholders' equity | 33,099 | 67,827 | [About MeiraGTx](index=8&type=section&id=About%20MeiraGTx) MeiraGTx is a vertically integrated clinical-stage gene therapy company with a broad pipeline, including four late-stage clinical programs, focusing on localized, low-dose delivery for genetic and common diseases, supported by comprehensive in-house manufacturing across five global facilities, two with GMP viral vector licenses, and an innovative riboswitch gene regulation platform for precise, dose-responsive gene expression control via oral small molecules in metabolic peptides, cell therapies, and chronic pain - MeiraGTx is a **vertically integrated clinical-stage gene therapy company** with four late-stage clinical programs[37](index=37&type=chunk) - The company possesses **comprehensive in-house manufacturing capabilities** with five global facilities, including two with GMP viral vector manufacturing licenses and one GMP QC facility licensed for clinical and commercial use[37](index=37&type=chunk) - MeiraGTx has developed an **innovative riboswitch gene regulation technology** for precise, dose-responsive control of gene expression via oral small molecules[37](index=37&type=chunk) - The riboswitch platform focuses on **metabolic peptides** (e.g., GLP-1, GIP, glucagon, amylin, PYY, and leptin), **CAR-T cell therapies**, and PNS targets for intractable pain[37](index=37&type=chunk) [Forward Looking Statement](index=9&type=section&id=Forward%20Looking%20Statement) This press release contains forward-looking statements as defined by the Private Securities Litigation Reform Act of 1995, covering product candidate development, clinical data, regulatory matters, milestone payments, Hologen collaboration, AI technology efficacy, and manufacturing technology development, which are based on current management expectations and involve known and unknown risks, uncertainties, and other factors that may cause actual results to differ materially [Contacts](index=10&type=section&id=Contacts) Provides contact information for investors and media

Core Insights - MeiraGTx announced a strategic collaboration with Hologen AI, involving a $200 million upfront payment and a joint venture, Hologen Neuro AI Ltd, with an additional $230 million committed to expedite the Phase 3 clinical development of AAV-GAD for Parkinson's disease [1][2][9] - The efficacy data for rAAV8.hRKp.AIPL1 for treating AIPL1-related retinal dystrophy was published, showing positive responses in all 11 children treated, leading to plans for Marketing Authorization Approval (MAA) in the UK and discussions with the FDA for a similar pathway in the US [1][2][10] Financial and Operational Highlights - As of March 31, 2025, MeiraGTx had cash and cash equivalents of approximately $66.5 million, with a net loss attributable to ordinary shareholders of $40.0 million for the quarter [21][31] - Service revenue increased to $1.9 million for the three months ended March 31, 2025, compared to $0.7 million for the same period in 2024, attributed to progress in process performance qualification services [23] - General and administrative expenses decreased to $9.4 million for the first quarter of 2025, down from $13.2 million in the same quarter of 2024, primarily due to reductions in share-based compensation and other costs [25] Clinical Development Updates - The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD for Parkinson's disease, allowing for expedited development and increased interaction with the FDA [4][9] - Positive discussions with the FDA have aligned on the requirements for ongoing Phase 2 studies for AAV-hAQP1 and AAV-GAD, supporting potential BLA filings [2][10] - The Phase 2 AQUAx2 study for AAV-hAQP1 continues to enroll participants, with a target for completion in Q4 2025 and potential BLA filing by the end of 2026 [13] Strategic Collaborations and Future Plans - The joint venture with Hologen Neuro AI Ltd aims to leverage AI technology to enhance the development of therapies targeting CNS disorders, with a focus on the AAV-GAD program [2][9] - MeiraGTx plans to initiate a Phase 3 study of AAV-GAD in the second half of 2025, continuing to work closely with regulatory agencies to expedite the development process [9][10]

Core Viewpoint - The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to MeiraGTx's AAV-GAD for treating Parkinson's disease, indicating significant potential benefits based on clinical study data [1][2][3] Company Overview - MeiraGTx Holdings plc is a clinical-stage genetic medicines company with a focus on developing gene therapies, including AAV-GAD for Parkinson's disease [1][7] - The company has a broad pipeline with four late-stage clinical programs targeting various diseases, including inherited conditions and common diseases [7] Product Details - AAV-GAD is an investigational gene therapy designed to address the dysfunction of brain circuits in Parkinson's disease by promoting local production of GABA [6] - The therapy is administered via a one-time stereotactic infusion into the subthalamic nucleus, a critical area for movement regulation [6] Clinical Studies - The RMAT designation was awarded following positive results from three clinical studies: a Phase 1 study (n=12), a Phase 2 double-blind study (n=45), and a bridging study (n=14) [2][3] - The Phase 2 studies demonstrated significant benefits on the standard motor endpoint in Parkinson's disease, specifically in the UPDRS Part 3 [3] Regulatory Insights - RMAT designation allows for increased interaction with the FDA, facilitating expedited development and review processes for promising therapies [4] - The designation includes benefits similar to Fast Track and Breakthrough Therapy designations, such as rolling review and potential Priority Review for the biologics license application [4] Market Context - Parkinson's disease is the second most common neurodegenerative disease, affecting nearly one million people in the U.S. and over 10 million globally [5] - Many patients experience diminishing returns from traditional dopamine replacement therapies, highlighting the need for effective disease-modifying treatments [5]

Core Insights - The cone rod dystrophy market is projected to grow from US$131.29 million in 2024 to US$177.59 million by 2030, driven by increased awareness and demand for effective treatments [2][3] - The market is expected to experience a compound annual growth rate (CAGR) of 5.33% from 2025 to 2030 [3] Market Segmentation Analysis - X-Linked Cone Rod Dystrophy is the leading type in the market, with ongoing research into gene therapies and neuroprotective agents [4] - Gene therapy holds the highest market share in treatment options, particularly through adeno-associated virus vectors [4] - Hospitals dominate the end user segment, benefiting from advanced treatment options and expanding infrastructure, especially in emerging markets [5] Regional Insights - North America leads the market due to high prevalence of eye disorders and technological advancements [6] - Asia Pacific is the fastest-growing region, with significant contributions from China's healthcare investments and Japan's market share [6] Market Dynamics - Growth drivers include rising incidence of cone rod dystrophy, increased R&D activities, and advancements in gene therapy [7] - Challenges include a limited patient pool and inconsistency in insurance coverage [7] - Trends such as early detection and personalized medicine are improving the market environment for new therapies [7] Competitive Landscape - Key players include SparingVision, Beacon Therapeutics, and Nanoscope Therapeutics, with mergers and acquisitions shaping the market [8] - Notable product SPVN06 from SparingVision is undergoing trials for various genetic mutations associated with Retinitis Pigmentosa [8]

Company Overview - MeiraGTx (NASDAQ: MGTX) focuses on developing genetic therapies for ocular diseases, Parkinson's Disease, and xerostomia [1] - The lead asset of the company is AAV-AQP1, which targets xerostomia and is currently in a pivotal phase 2 trial [1] Industry Insights - The company operates within the genetic therapy sector, which is increasingly gaining attention for its potential to address various chronic and debilitating conditions [1]

Financial Performance - Total revenue for 2024 was $33,279,000, a significant increase from $14,017,000 in 2023, representing a growth of 137%[714] - Operating expenses increased to $197,491,000 in 2024 from $151,078,000 in 2023, marking a rise of 30.7%[714] - The net loss for 2024 was $147,791,000, compared to a net loss of $84,027,000 in 2023, indicating a 75.9% increase in losses[714] - Cash and cash equivalents decreased to $103,659,000 in 2024 from $129,566,000 in 2023, reflecting a decline of 20%[713] - Total current assets fell to $123,518,000 in 2024, down from $159,622,000 in 2023, a decrease of 22.6%[713] - Total liabilities increased to $201,924,000 in 2024 from $188,567,000 in 2023, an increase of 7.1%[713] - Shareholders' equity decreased significantly to $67,827,000 in 2024 from $138,177,000 in 2023, a decline of 50.9%[713] - The company reported a basic and diluted net loss per ordinary share of $2.12 for 2024, compared to $1.49 for 2023[714] - The segment net loss for 2024 was $147,791,000, compared to a net loss of $84,027,000 in 2023, indicating a worsening financial performance[814] Capital and Funding - The company requires additional capital to fund operations, which may not be available on acceptable terms[14] - The Company’s capital resources have been primarily funded through collaboration agreements and equity offerings[727] - The Company raised gross proceeds of $8.4 million through the sale of 1,508,517 ordinary shares under the "at-the-market" equity offering program during the year ended December 31, 2024[842] - The Company completed a public offering of 12,500,000 ordinary shares at a price of $4.00 per share, resulting in gross proceeds of $50.0 million on August 12, 2024[843] Research and Development - The company is heavily dependent on the success of its product candidates, which are still in development, and if none receive regulatory approval, the business may be harmed[14] - Clinical trials are expensive and time-consuming, with uncertain outcomes, and the company may encounter substantial delays[14] - The Company incurred research and development costs with Johnson & Johnson Innovative Medicine responsible for up to 100% of the costs, depending on the type of services performed[795] - Research and development costs are charged to expense as incurred, including employee-related expenses and costs associated with clinical studies[800] - The Company recorded reductions to research and development expenses of $5.5 million and $5.1 million for the years ended December 31, 2024 and 2023, respectively, related to tax incentive programs[736] Regulatory and Compliance - The company is subject to significant regulation regarding the manufacturing of its products, and its manufacturing facilities may not continue to meet regulatory requirements[14] - The Company has not recognized a provision for obsolete and excess inventory as of December 31, 2024[734] Competition and Market Environment - The company faces significant competition in a rapidly changing technological environment, which may affect its financial condition and ability to market its product candidates[21] - The company depends on proprietary technology licensed from others, and losing these licenses could hinder its ability to develop product candidates[21] Tax and Deferred Tax - The Company recorded unrecognized tax positions of $2.2 million and $2.0 million as of December 31, 2024 and 2023, respectively[805] - The Company has recorded a full valuation allowance against its deferred tax assets, indicating management's assessment that it is more likely than not that these assets will not be fully realized[852] - The Company recorded a deferred tax asset of $155.2 million as of December 31, 2024, compared to $130.3 million in 2023, primarily due to net operating loss carryforwards increasing from $70.7 million to $85.3 million[852] - The total deferred tax expense for the Company was $24.0 million in 2024, compared to $9.7 million in 2023, reflecting a significant increase in deferred tax liabilities[852] Collaboration Agreements - The Company received a non-refundable upfront cash payment of $65.0 million from Janssen in December 2023 under the Asset Purchase Agreement[720] - Janssen agreed to pay future contingent consideration of up to $350.0 million, including milestone payments of $50.0 million for initiating a Phase 3 clinical trial and $175.0 million upon the first commercial sale of the RPGR Product in the U.S.[720] - Under the Collaboration Agreement with Johnson & Johnson Innovative Medicine, the Company received a non-refundable upfront fee of $100.0 million and a milestone payment of $30.0 million in December 2021[856] Asset Management - Long-lived assets decreased from $157,356,000 in 2023 to $136,473,000 in 2024, with notable declines in assets located in the United States and the European Union[816] - The balance of asset retirement obligations increased from $2,401,000 in 2023 to $2,821,000 in 2024, reflecting a change in estimate of $345,000[763] Share-Based Compensation - The total share-based compensation expense for the year ended December 31, 2024, was $25.2 million, a decrease from $27.7 million in 2023[841] - The total compensation expense related to unvested options as of December 31, 2024, was $8.5 million, expected to be recognized over 3.2 years[836] - The total compensation expense related to unvested RSUs as of December 31, 2024, was $21.9 million, expected to be recognized over 3.3 years[839] - The Company recorded share-based compensation expense of $15.2 million for RSUs in 2024, compared to $14.1 million in 2023[838] Revenue Recognition - The Company recognizes revenue based on the control of promised goods or services, following ASC 606, and evaluates performance obligations to determine revenue recognition[782] - The Company recognizes revenues from nonrefundable, up-front fees allocated to licenses when the license is transferred and the licensee can benefit from it[792] - The Company’s collaboration revenue is recognized using the cost-to-cost input method under ASC 606, reflecting the progress towards completion of performance obligations[864] - The company recognized $33.3 million of service revenue during the year ended December 31, 2024, which included $14.0 million of deferred revenue recognized as service revenue[876]

Exhibit 99.2 MeiraGTx Reports Fourth Quarter and Full Year 2024 Financial and Operational Results and Recent Business Updates LONDON and NEW YORK, March 13, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced financial and operational results for the fourth quarter and full-year ended December 31, 2024, and provided a corporate update. "MeiraGTx demonstrated excellent execution in 2024, marked by significant advanc ...