LONDON and NEW YORK, Oct. 07, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the Company will exhibit two posters at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress, which is being held from October 7-10, 2025, in Seville, Spain. The posters are available on the Posters and Publications page of the Company’s website. The details of the poster presentations are as follows: Poster Numb ...

MeiraGTx Holdings plc (NASDAQ: MGTX ) is a genetic medicines biotech with a late-stage pipeline and capabilities for end-to-end GMP manufacturing. Its main drug candidates are AAV-AIPL1 for congenital blindness, which is working toward exceptional-circumstances filings and has a plannedMy name is Myriam Hernandez Alvarez. I received the Electronics and Telecommunication Engineering degree from the Escuela Politecnica Nacional, Quito, Ecuador, the M.Sc. degree in computer science from Ohio University, Athens ...

MeiraGTx Holdings plc (NASDAQ: MGTX ) is a genetic medicines biotech with a late-stage pipeline and capabilities for end-to-end GMP manufacturing. Its main drug candidates are AAV-AIPL1 for congenital blindness, which is working toward exceptional-circumstances filings and has a plannedMy name is Myriam Hernandez Alvarez. I received the Electronics and Telecommunication Engineering degree from the Escuela Politecnica Nacional, Quito, Ecuador, the M.Sc. degree in computer science from Ohio University, Athens ...

PART I. FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20%28Unaudited%29) This section presents the unaudited condensed consolidated financial statements for MeiraGTx Holdings plc and its subsidiaries, including the balance sheets, statements of operations and comprehensive loss, statements of shareholders' equity, and statements of cash flows, along with accompanying notes - The financial statements are **unaudited** and include normal recurring adjustments, prepared in conformity with GAAP[41](index=41&type=chunk)[42](index=42&type=chunk) [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) | Metric (in thousands) | June 30, 2025 | December 31, 2024 | Change (in thousands) | | :-------------------- | :------------ | :---------------- | :-------------------- | | Cash and cash equivalents | $32,166 | $103,659 | $(71,493) | | Total Current Assets | $47,084 | $123,518 | $(76,434) | | Total Assets | $198,716 | $269,751 | $(71,035) | | Total Current Liabilities | $53,436 | $60,783 | $(7,347) | | Total Liabilities | $195,756 | $201,924 | $(6,168) | | **Total Shareholders' Equity** | **$2,960** | **$67,827** | **$(64,867)** | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) | Metric (in thousands) | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | Change (YoY) (in thousands) | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | Change (YoY) (in thousands) | | :-------------------- | :----------------------------- | :----------------------------- | :-------------------------- | :----------------------------- | :----------------------------- | :-------------------------- | | Total revenue | $3,691 | $282 | +$3,409 | $5,617 | $979 | +$4,638 | | Total operating expenses | $48,484 | $46,191 | +$2,293 | $92,006 | $93,660 | $(1,654) | | Loss from operations | $(44,793) | $(45,909) | +$1,116 | $(86,389) | $(92,681) | +$6,292 | | **Net loss** | **$(38,795)** | **$(48,620)** | **+$9,825** | **$(78,776)** | **$(69,062)** | **$(9,714)** | | Basic and diluted net loss per ordinary share | $(0.48) | $(0.76) | +$0.28 | $(0.99) | $(1.08) | +$0.09 | [Condensed Consolidated Statement of Shareholders' Equity](index=9&type=section&id=Condensed%20Consolidated%20Statement%20of%20Shareholders%27%20Equity) | Metric (in thousands) | December 31, 2024 | June 30, 2025 | Change (in thousands) | | :-------------------- | :---------------- | :------------ | :-------------------- | | **Total Shareholders' Equity** | **$67,827** | **$2,960** | **$(64,867)** | | Capital in excess of par value | $773,565 | $791,280 | +$17,715 | | Accumulated deficit | $(702,022) | $(780,798) | $(78,776) | [Condensed Consolidated Statements of Cash Flows](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) | Metric (in thousands) | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | Change (YoY) (in thousands) | | :-------------------- | :----------------------------- | :----------------------------- | :-------------------------- | | Net cash used in operating activities | $(80,775) | $(56,197) | $(24,578) | | Net cash (used in) provided by investing activities | $(2,471) | $26,011 | $(28,482) | | Net cash provided by financing activities | $7,163 | $333 | +$6,830 | | **Net decrease in cash, cash equivalents and restricted cash** | **$(76,083)** | **$(29,853)** | **$(46,230)** | | Cash, cash equivalents and restricted cash at end of period | $34,424 | $101,025 | $(66,601) | [Notes to Condensed Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) [1. Organization and Basis of Presentation](index=12&type=section&id=1.%20Organization%20and%20Basis%20of%20Presentation) This note describes the company's business as a clinical-stage genetic medicines company, details the strategic collaboration with Hologen Limited, and outlines the asset purchase and related agreements 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, involving an **upfront cash payment of $200 million** (**$23 million** received by Q3 2025) and up to **$230 million in additional funding**[30](index=30&type=chunk)[147](index=147&type=chunk) - On December 20, 2023, MeiraGTx sold and assigned the UCLB RPGR License Agreement for botaretigene sparoparvovec (bota-vec) to Johnson & Johnson Innovative Medicine for a **$65.0 million upfront payment** and potential future contingent consideration of up to **$350.0 million**, with **$60.0 million in milestones** received in 2024[39](index=39&type=chunk)[40](index=40&type=chunk) - As of June 30, 2025, the company had an **accumulated deficit of $780.8 million** and **$34.4 million in cash, cash equivalents, and restricted cash**[43](index=43&type=chunk)[45](index=45&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 note outlines the company's significant accounting policies, including consolidation, use of estimates, restricted cash, fair value measurements, asset retirement obligations, IDA Ireland grant recognition, revenue recognition, research and development expense treatment, and net loss per ordinary share calculation - The company's financial statements **consolidate** Meira Holdings and its wholly-owned subsidiaries, eliminating all intercompany balances and transactions[49](index=49&type=chunk)[51](index=51&type=chunk) - Management uses **significant estimates** in areas such as service revenue, fair value of nonfinancial assets, share-based compensation, and leases, which may differ materially from actual results[52](index=52&type=chunk) - **Restricted cash totaled $2.3 million** as of June 30, 2025, primarily for a guarantee required by an IDA Ireland research and innovation grant[53](index=53&type=chunk) - A **$1.3 million gain on termination of lease liabilities** was recorded during Q1 2025 due to a change in estimate for an asset retirement obligation[60](index=60&type=chunk) - **Revenue recognition** follows a five-step model, allocating transaction price to performance obligations based on estimated standalone selling prices[66](index=66&type=chunk)[71](index=71&type=chunk) - **Research and development costs are expensed as incurred**, encompassing employee-related expenses, third-party vendor costs for clinical/preclinical studies, and manufacturing-related expenses[78](index=78&type=chunk)[79](index=79&type=chunk) [3. Equity Method and Other Investments](index=31&type=section&id=3.%20Equity%20Method%20and%20Other%20Investments) This note 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) | Cost Basis (in thousands) | | :------- | :-------------- | :------------------- | :---------------------------- | :------------------------ | | Visiogene LLC | Equity Method Investment | 25.0 % | $5,133 | $5,165 | | Other | Equity Investment | 0.9 % | $1,616 | $1,500 | | **Total** | | | **$6,749** | **$6,665** | [4. Accrued Expenses](index=32&type=section&id=4.%20Accrued%20Expenses) This note provides a breakdown of accrued expenses, showing a significant decrease from December 31, 2024, to June 30, 2025, primarily driven by a reduction in compensation and benefits | Accrued Expense (in thousands) | June 30, 2025 | December 31, 2024 | Change (in thousands) | | :----------------------------- | :------------ | :---------------- | :-------------------- | | Professional fees | $4,925 | $6,326 | $(1,401) | | Research and development | $3,254 | $2,234 | +$1,020 | | Manufacturing costs | $2,239 | $1,540 | +$699 | | Clinical trial costs | $1,879 | $3,864 | $(1,985) | | Compensation and benefits | $1,790 | $11,197 | $(9,407) | | Consulting | $1,055 | $1,530 | $(475) | | Fixed assets | $102 | $326 | $(224) | | Rent and facilities costs | $79 | $257 | $(178) | | Other | $160 | $140 | +$20 | | **Total** | **$15,483** | **$27,414** | **$(11,931)** | [5. Share-Based Compensation](index=32&type=section&id=5.%20Share-Based%20Compensation) This note details the company's equity incentive plans, including share options and restricted share units (RSUs), and the associated compensation expense recognized | Expense Category (in thousands) | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | | :------------------------------ | :----------------------------- | :----------------------------- | :----------------------------- | :----------------------------- | | Research and development | $2,091 | $2,832 | $4,139 | $5,707 | | General and administrative | $3,573 | $2,978 | $6,412 | $7,063 | | **Total** | **$5,664** | **$5,810** | **$10,551** | **$12,770** | - As of June 30, 2025, total **unrecognized compensation expense** for **unvested options was $6.2 million** (expected over **3.9 years**) and for **unvested RSUs was $33.7 million** (expected over **3.8 years**)[99](index=99&type=chunk)[103](index=103&type=chunk) [6. Ordinary Shares](index=37&type=section&id=6.%20Ordinary%20Shares) This note describes the company's "at-the-market" (ATM) equity offering program, through which it raised additional capital by selling ordinary shares - During the six months ended June 30, 2025, the company raised **gross proceeds of $9.9 million** through the sale of **1,510,300 ordinary shares** via its "at-the-market" equity offering program[106](index=106&type=chunk) - An **additional $81.8 million of ordinary shares** may be sold under the existing "at-the-market" equity program[106](index=106&type=chunk) [7. Income Taxes](index=37&type=section&id=7.%20Income%20Taxes) The company did not record an income tax provision due to sustained losses and maintains a full valuation allowance against its deferred tax assets - **No provision for income taxes** was recorded for the three and six months ended June 30, 2025 and 2024, as the company generated **losses** in all periods[107](index=107&type=chunk) - A **full valuation allowance** is maintained against deferred tax assets in the United States, United Kingdom, Ireland, and Netherlands due to uncertainty regarding their realizability[108](index=108&type=chunk) [8. Related-Party Transactions](index=38&type=section&id=8.%20Related-Party%20Transactions) This note details the Asset Purchase Agreement and related Supply Agreement with Johnson & Johnson Innovative Medicine for the RPGR Product, including milestone payments and revenue recognition - Under the Asset Purchase Agreement with Johnson & Johnson Innovative Medicine, MeiraGTx received a **$65.0 million upfront payment** (Dec 2023) and **$60.0 million in milestone payments** (2024), with eligibility for up to **$350.0 million in future contingent milestones**[109](index=109&type=chunk) - The **transaction price of $92.3 million** was allocated to four performance obligations: PPQ services, material rights for commercial supply, manufacturing technology transfer, and the sale of nonfinancial assets[110](index=110&type=chunk) - Recognized **$2.3 million** (H1 2025) and **$1.0 million** (H1 2024) of deferred revenue as **service revenue** from PPQ services under the Asset Purchase Agreement[117](index=117&type=chunk) - The company entered into a Notes Purchase Agreement with Perceptive (an affiliate of a >10% shareholder) for an initial **$75.0 million**, maturing August 2, 2026, with an **annual interest rate of 14.33%** at June 30, 2025[121](index=121&type=chunk)[128](index=128&type=chunk) [9. Leases](index=42&type=section&id=9.%20Leases) This note outlines the company's commitments under operating and finance leases for various facilities and equipment, detailing lease costs, balance sheet amounts, and future minimum lease payments | Lease Cost (in thousands) | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | | :------------------------ | :----------------------------- | :----------------------------- | :----------------------------- | :----------------------------- | | Finance lease cost | $305 | $281 | $588 | $560 | | Operating lease cost | $1,113 | $1,437 | $2,397 | $2,872 | | Short-term lease cost | $100 | $41 | $195 | $87 | | **Total Lease Cost** | **$1,518** | **$1,759** | **$3,180** | **$3,519** | - Future **minimum lease payments** under non-cancellable operating leases totaled **$7.8 million** as of June 30, 2025, with **total lease liabilities of $6.7 million**[126](index=126&type=chunk) - An **early termination option** on an operating lease for laboratory and office space was exercised in Q1 2025, leading to a remeasurement of the ROU asset and lease liability[124](index=124&type=chunk) [10. Debt Financing](index=46&type=section&id=10.%20Debt%20Financing) This note provides further details on the senior secured financing arrangement, the Notes Purchase Agreement with Perceptive, including the principal amount, interest rate, maturity, security, and covenants - The Notes Purchase Agreement provides for an initial **$75.0 million notes issuance** (Tranche 1 Notes) maturing on August 2, 2026[128](index=128&type=chunk) - Outstanding amounts bear interest at **10.00%** plus the one-month SOFR (subject to a **1.00% floor**), resulting in an **annual interest rate of 14.33%** at June 30, 2025[128](index=128&type=chunk) - The company's obligations are **secured** by its London and Shannon manufacturing facilities, **$3.0 million of cash**, bank accounts of Subsidiary Guarantors, and their equity interests[129](index=129&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[131](index=131&type=chunk) [11. Commitments and Contingencies](index=47&type=section&id=11.%20Commitments%20and%20Contingencies) This note states that there were no new material commitments or contingencies during the six-month period ended June 30, 2025 - **No new material commitments or contingencies** were entered into during the six-month period ended June 30, 2025[133](index=133&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=48&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides management's perspective on the company's financial condition and results of operations, including an overview of its business, recent strategic transactions, development highlights, and a detailed analysis of revenues, expenses, and liquidity - MeiraGTx is a **clinical-stage genetic medicines company** with a broad pipeline and internal end-to-end **manufacturing capabilities**[135](index=135&type=chunk) - The company reported **net losses of $38.8 million** for Q2 2025 and **$78.8 million** for H1 2025, with an **accumulated deficit of $780.8 million** as of June 30, 2025[139](index=139&type=chunk) - Current **cash, cash equivalents, and restricted cash of $34.4 million**, along with anticipated Hologen proceeds, are expected to fund operations into **2027** and repay the **$75.0 million debt** due August 2026[138](index=138&type=chunk)[142](index=142&type=chunk) - A **strategic collaboration** with Hologen Limited, involving a **$200 million upfront payment** and up to **$230 million in additional funding**, is expected to close in Q3 2025[147](index=147&type=chunk)[148](index=148&type=chunk) [Overview](index=48&type=section&id=Overview) - 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[135](index=135&type=chunk) - The company has internal end-to-end **manufacturing capabilities**, including two GMP viral vector production facilities and internal plasmid production[135](index=135&type=chunk) - Operations have been financed primarily through **equity sales ($632.2 million)**, **debt financing ($75.0 million)**, and **upfront/milestone payments from Johnson & Johnson Innovative Medicine ($130.0 million** from Collaboration Agreement, **$125.0 million** from Asset Purchase Agreement)[136](index=136&type=chunk)[138](index=138&type=chunk) - As of June 30, 2025, the company had **$34.4 million in cash, cash equivalents, and restricted cash**, and an **accumulated deficit of $780.8 million**[138](index=138&type=chunk)[139](index=139&type=chunk) [Hologen Transactions](index=52&type=section&id=Hologen%20Transactions) - MeiraGTx entered into a **strategic collaboration** with Hologen Limited on March 9, 2025, for multi-modal generative AI foundation models in clinical medicine and drug development[147](index=147&type=chunk) - The collaboration includes a **$200 million upfront cash payment** (with **$23 million** received by Q3 2025) and up to **$230 million in additional funding** from Hologen[147](index=147&type=chunk) - A joint venture, Hologen Neuro AI Ltd, is being formed to finance the development of AAV-GAD for Parkinson's disease and other CNS therapies, with MeiraGTx Neuro UK holding **30% ownership** and leading clinical development and manufacturing[152](index=152&type=chunk)[167](index=167&type=chunk) - Hologen will acquire a **minority interest** in MeiraGTx Manufacturing and contribute to its annual funding, with an option to increase its stake to **40% within 12 months**[155](index=155&type=chunk)[157](index=157&type=chunk) [Acquisition of Smart Immune Assets](index=56&type=section&id=Acquisition%20of%20Smart%20Immune%20Assets) - In July 2025, MeiraGTx Cell Therapies acquired certain assets and operations of Smart Immune, a French clinical-stage biotechnology company, for **€250,000** plus a **€100,000 transfer fee**[158](index=158&type=chunk) - The acquisition includes Smart Immune's ProTcell platform, a T-cell progenitor-based cell therapy, intended to advance off-the-shelf, allogenic ProTcell-derived CAR-T therapies incorporating MeiraGTx's **riboswitch technology**[158](index=158&type=chunk) [Recent Development Highlights and Anticipated Milestones](index=56&type=section&id=Recent%20Development%20Highlights%20and%20Anticipated%20Milestones) - AAV2-hAQP1 for Radiation-Induced Xerostomia received **RMAT designation** in December 2024, with Phase 2 AQUAx2 enrollment targeting completion in **Q4 2025** and pivotal data readout in **late 2026**[161](index=161&type=chunk) - AAV-GAD for Parkinson's disease received **RMAT designation** in May 2025, based on positive data from three clinical studies, and a **Phase 3 study** is planned for initiation in 2025[161](index=161&type=chunk)[162](index=162&type=chunk) - AAV-AIPL1 for LCA4 showed meaningful responses in **11/11 treated children**, leading to preparation for **MAA submission** in the UK and **BLA** in the US[168](index=168&type=chunk) - The **Riboswitch Gene Regulation Technology Platform** is progressing with compelling preclinical data in metabolic peptides and hormones, with the first **IND** using this platform (leptin) likely in 2025[172](index=172&type=chunk) - **Manufacturing facilities** in the UK and Ireland successfully renewed licenses and expanded capabilities, with the Shannon site now authorized for viral vector manufacturing for clinical trials[172](index=172&type=chunk)[176](index=176&type=chunk) [Components of Our Results of Operations](index=62&type=section&id=Components%20of%20Our%20Results%20of%20Operations) - **Service revenue** primarily consists of process performance qualification (PPQ) services under the Asset Purchase Agreement[177](index=177&type=chunk) - **Operating expenses** include general and administrative costs, research and development costs, and cost of service revenue, with R&D expenses expected to increase but partially offset by Hologen funding for AAV-GAD[178](index=178&type=chunk)[184](index=184&type=chunk) - Other non-operating income (expense) includes **foreign currency gains/losses**, **interest income**, **interest expense**, and **gain on sale of nonfinancial assets**[187](index=187&type=chunk)[189](index=189&type=chunk)[190](index=190&type=chunk)[191](index=191&type=chunk) [Results of Operations](index=66&type=section&id=Results%20of%20Operations) Comparison of Three Months Ended June 30, 2025 and 2024 (in thousands) | Metric | 2025 | 2024 | Change (in thousands) | | :---------------------------------- | :--- | :--- | :-------------------- | | Service revenue - related party | $3,691 | $282 | +$3,409 | | Cost of service revenue - related party | $2,676 | $0 | +$2,676 | | General and administrative | $12,313 | $11,257 | +$1,056 | | Research and development | $33,495 | $34,934 | $(1,439) | | Loss from operations | $(44,793) | $(45,909) | +$1,116 | | Foreign currency gain (loss) | $8,624 | $(284) | +$8,908 | | Interest income | $408 | $827 | $(419) | | Interest expense | $(3,034) | $(3,254) | +$220 | | **Net loss** | **$(38,795)** | **$(48,620)** | **+$9,825** | Comparison of Six Months Ended June 30, 2025 and 2024 (in thousands) | Metric | 2025 | 2024 | Change (in thousands) | | :---------------------------------- | :--- | :--- | :-------------------- | | Service revenue - related party | $5,617 | $979 | +$4,638 | | Cost of service revenue - related party | $4,054 | $0 | +$4,054 | | General and administrative | $21,677 | $24,404 | $(2,727) | | Research and development | $66,275 | $69,256 | $(2,981) | | Loss from operations | $(86,389) | $(92,681) | +$6,292 | | Foreign currency gain (loss) | $12,311 | $(819) | +$13,130 | | Interest income | $1,379 | $1,924 | $(545) | | Interest expense | $(6,077) | $(6,504) | +$427 | | Gain on sale of nonfinancial assets | $0 | $29,018 | $(29,018) | | **Net loss** | **$(78,776)** | **$(69,062)** | **$(9,714)** | - **Service revenue increased significantly** due to increased progress of PPQ services under the Asset Purchase Agreement[196](index=196&type=chunk)[210](index=210&type=chunk) - **Research and development expenses decreased** primarily due to reclassification of manufacturing costs and bota-vec funding shift to Johnson & Johnson Innovative Medicine, partially offset by increased AAV-GAD clinical trial expenses[203](index=203&type=chunk)[217](index=217&type=chunk) - A **substantial foreign currency gain** was recognized in both periods due to the weakening of the U.S. dollar against the pound sterling and euro[205](index=205&type=chunk)[218](index=218&type=chunk) [Liquidity and Capital Resources](index=71&type=section&id=Liquidity%20and%20Capital%20Resources) - The company used **$80.8 million in cash flows from operations** for the six months ended June 30, 2025, reflecting ongoing **operating losses**[221](index=221&type=chunk) - As of June 30, 2025, **cash, cash equivalents, and restricted cash totaled $34.4 million**[225](index=225&type=chunk) - Current funds, including a **$17.0 million deposit from Hologen** and remaining collaboration proceeds, are estimated to fund operating expenses and capital expenditures into **2027** and repay the **$75.0 million debt** due August 2026[224](index=224&type=chunk) Cash Flow Summary (in thousands) for the Six Months Ended June 30, | Activity | 2025 | 2024 | Change (in thousands) | | :-------------------------------------- | :--- | :--- | :-------------------- | | Net cash used in operating activities | $(80,775) | $(56,197) | $(24,578) | | Net cash (used in) provided by investing activities | $(2,471) | $26,011 | $(28,482) | | Net cash provided by financing activities | $7,163 | $333 | +$6,830 | | **Net decrease** | **$(76,083)** | **$(29,853)** | **$(46,230)** | [Off-Balance Sheet Arrangements](index=75&type=section&id=Off-Balance%20Sheet%20Arrangements) - The company has **not entered into any off-balance sheet arrangements** or holds any variable interest entities[234](index=234&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=75&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section discusses the company's exposure to market risks, specifically foreign currency exchange risk and interest rate risk, and provides quantitative estimates of their potential impact - The company is exposed to **foreign currency exchange risk**, primarily between the U.S. Dollar and the British pound sterling and euro, due to international operations[236](index=236&type=chunk) - A **10% unfavorable movement** in foreign currency exchange rates is estimated to result in an additional **foreign currency loss of approximately $2.2 million** for the six months ended June 30, 2025[236](index=236&type=chunk) - The company is exposed to **interest rate risk** from its Notes Purchase Agreement, which bears a **fluctuating interest rate** (**14.33%** at June 30, 2025, on **$75.0 million outstanding debt**)[237](index=237&type=chunk) - A hypothetical **1% increase in the SOFR** would increase annual **interest expense by approximately $0.8 million**[237](index=237&type=chunk) [Item 4. Controls and Procedures](index=77&type=section&id=Item%204.%20Controls%20and%20Procedures) This section addresses the effectiveness of the company's disclosure controls and procedures and reports no material changes in internal control over financial reporting during the quarter - Management, with CEO and CFO participation, concluded that **disclosure controls and procedures were effective** at a reasonable assurance level as of June 30, 2025[239](index=239&type=chunk) - **No changes in internal control over financial reporting** materially affected, or are reasonably likely to materially affect, internal control during the quarter ended June 30, 2025[240](index=240&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=78&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently subject to any material legal proceedings - The company is **not subject to any material legal proceedings**[242](index=242&type=chunk) [ITEM 1A. RISK FACTORS](index=78&type=section&id=ITEM%201A.%20RISK%20FACTORS) This section outlines various risks that could materially and adversely affect the company's business, financial condition, results of operations, and future growth prospects - Investing in the company's ordinary shares involves a **high degree of risk**, encompassing financial, operational, technological, regulatory, and other risks[46](index=46&type=chunk)[243](index=243&type=chunk) [Risks Related to Our Financial Position and Need for Additional Capital](index=78&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) This sub-section details risks associated with the company's financial health, including a history of significant losses, the need for additional capital, uncertainties regarding milestone payments from collaborations, and potential difficulties in managing debt obligations - The company has incurred **significant losses** since inception (**$78.8 million net loss** for H1 2025, **$780.8 million accumulated deficit**) and anticipates continued losses, potentially never achieving profitability[244](index=244&type=chunk) - There is **no guarantee of timely receipt of additional milestone payments** (up to **$285.0 million**) under the Asset Purchase Agreement with Johnson & Johnson Innovative Medicine or revenues from commercial supply of the RPGR Product[249](index=249&type=chunk)[250](index=250&type=chunk) - **Additional capital will be required** to fund operations, which may not be available on acceptable terms, potentially forcing delays or reductions in R&D programs[251](index=251&type=chunk) - The company may **not have sufficient cash flows** to satisfy debt obligations or comply with covenants under its financing arrangements, risking acceleration of debt and adverse business impacts[255](index=255&type=chunk)[256](index=256&type=chunk) [Risks Related to Discovery, Development, Clinical Testing, Manufacturing and Regulatory Approval](index=87&type=section&id=Risks%20Related%20to%20Discovery%2C%20Development%2C%20Clinical%20Testing%2C%20Manufacturing%20and%20Regulatory%20Approval) This sub-section highlights the extensive and unpredictable challenges in developing gene therapies, including the high failure rate of clinical trials, regulatory uncertainties, manufacturing complexities, and the potential for adverse events or smaller-than-projected patient populations - Future success is **substantially dependent** on the successful development, manufacturing, regulatory approval, and commercialization of product candidates, with no guarantee of marketability[259](index=259&type=chunk) - Gene therapy development is **difficult to predict** in terms of time and cost due to its novelty and limited approvals in the US or Europe, with potential for unexpected adverse events or toxicities[262](index=262&type=chunk)[264](index=264&type=chunk) - **Clinical trials are expensive, time-consuming, difficult to design**, and involve uncertain outcomes, with potential for substantial delays due to patient enrollment, regulatory consensus, or manufacturing issues[272](index=272&type=chunk)[276](index=276&type=chunk) - **Manufacturing facilities are subject to significant GMP regulation**, and issues such as contamination, raw material shortages, or failure to meet regulatory requirements could delay clinical development or commercialization[307](index=307&type=chunk)[308](index=308&type=chunk)[312](index=312&type=chunk) - Product candidates may cause **serious adverse events or undesirable side effects**, potentially leading to clinical trial interruptions, restrictive labels, or denial of regulatory approval[318](index=318&type=chunk)[322](index=322&type=chunk) [Risks Related to Healthcare Laws and Other Legal Compliance Matters](index=121&type=section&id=Risks%20Related%20to%20Healthcare%20Laws%20and%20Other%20Legal%20Compliance%20Matters) This sub-section addresses the complex and evolving regulatory environment for healthcare, including legislation impacting pricing and reimbursement, fraud and abuse laws, data privacy regulations, environmental compliance, and 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 could affect pricing[346](index=346&type=chunk)[350](index=350&type=chunk) - Business operations are **subject to various healthcare fraud and abuse laws** (e.g., Anti-Kickback Statute, False Claims Act, HIPAA), with potential for significant civil, criminal, and administrative penalties for non-compliance[362](index=362&type=chunk)[364](index=364&type=chunk) - The company is subject to **complex and evolving data privacy and protection laws** (e.g., HIPAA, CCPA, GDPR, UK GDPR), with compliance being costly and potential failures leading to fines, litigation, and reputational harm[365](index=365&type=chunk)[370](index=370&type=chunk)[372](index=372&type=chunk) - International operations are **subject to anti-corruption laws** (e.g., UK Bribery Act, FCPA) and Trade Control laws, with non-compliance risking criminal and civil penalties, disgorgement, and reputational damage[376](index=376&type=chunk)[379](index=379&type=chunk) [Risks Related to Commercialization](index=135&type=section&id=Risks%20Related%20to%20Commercialization) This sub-section details the challenges in commercializing product candidates, including intense competition, difficulties in securing adequate reimbursement, achieving market acceptance, and establishing sales and marketing capabilities - The gene therapy market is **highly competitive**, with major pharmaceutical and biotechnology companies developing similar or more effective therapies, potentially impacting market position and pricing[381](index=381&type=chunk)[385](index=385&type=chunk) - Successful commercialization depends on obtaining **adequate coverage and reimbursement** from governmental and private payors, which is uncertain and subject to increasing cost-containment pressures[386](index=386&type=chunk)[387](index=387&type=chunk) - Even if approved, product candidates may **fail to achieve market acceptance** by physicians, patients, and third-party payors due to factors like efficacy, cost, convenience, or competition[393](index=393&type=chunk)[395](index=395&type=chunk) - The company **lacks existing sales, marketing, and distribution infrastructure**, and establishing it is costly and risky; reliance on third-party collaborators may not be successful[396](index=396&type=chunk)[397](index=397&type=chunk) - Commercialization outside the US, UK, or EU involves **additional risks**, including differing regulatory requirements, intellectual property protection, economic instability, and currency fluctuations[403](index=403&type=chunk) [Risks Related to Our Dependence on Third Parties](index=145&type=section&id=Risks%20Related%20to%20Our%20Dependence%20on%20Third%20Parties) This sub-section outlines the risks associated with relying on third parties for manufacturing, clinical trials, and collaborations, including potential supply shortages, quality control issues, delays, and the challenges of managing external relationships - If internal GMP manufacturing facilities cannot meet demand, **reliance on third-party manufacturers** increases risks of insufficient supply, unacceptable costs, and delays, potentially impacting development or commercialization[410](index=410&type=chunk)[411](index=411&type=chunk) - **Failure of third-party manufacturers** to comply with GMP regulations or meet contractual requirements could result in sanctions, delays, or supply disruptions[413](index=413&type=chunk)[414](index=414&type=chunk) - **Challenges in establishing and maintaining collaborative relationships** (e.g., with Sanofi, Hologen) due to shifting priorities, unsatisfactory results, or disputes could significantly limit product development and commercialization[416](index=416&type=chunk)[418](index=418&type=chunk) - **Reliance on CROs** and other third parties for preclinical and clinical trials means limited direct control over their performance, risking extended trials, compromised data, and increased costs if they perform unsatisfactorily[419](index=419&type=chunk)[422](index=422&type=chunk) [Risks Related to Intellectual Property](index=151&type=section&id=Risks%20Related%20to%20Intellectual%20Property) This sub-section details the critical importance and inherent vulnerabilities of the company's intellectual property, including dependence on licensed technology, challenges in obtaining and maintaining broad patent protection, risks of infringement claims, and the potential for changes in patent laws - The company **depends on proprietary technology licensed from others**; loss of existing licenses or inability to acquire additional rights could prevent continued product development[424](index=424&type=chunk) - **Obtaining and maintaining broad patent protection is challenging**, expensive, and uncertain; patents may be challenged, narrowed, or invalidated, impacting competitive effectiveness[428](index=428&type=chunk)[429](index=429&type=chunk)[433](index=433&type=chunk) - **Risk of third parties asserting patent infringement claims**, leading to substantial costs, delays, or inability to commercialize products, and potentially requiring costly litigation or licensing[434](index=434&type=chunk)[438](index=438&type=chunk) - **Changes in patent laws** (e.g., AIA) or jurisprudence could diminish the value of patents, weakening the ability to obtain new patents or enforce existing ones[445](index=445&type=chunk)[448](index=448&type=chunk) - **Reliance on trade secrets** and confidentiality agreements for protection, but these are difficult to maintain as confidential and vulnerable to misappropriation or unauthorized disclosure[455](index=455&type=chunk)[459](index=459&type=chunk) [Risks Related to Employee Matters and Managing Growth](index=167&type=section&id=Risks%20Related%20to%20Employee%20Matters%20and%20Managing%20Growth) This sub-section addresses risks related to human capital, including challenges in managing organizational growth, retaining key personnel, potential product liability, and the impact of misconduct or system failures - **Managing organizational growth** (e.g., hiring, integration) can be difficult, potentially disrupting operations, diverting management attention, and increasing expenses[464](index=464&type=chunk) - Future success depends on **retaining key personnel** (CEO Alexandria Forbes, COO/CFO Rich Giroux, CDO Stuart Naylor) and attracting qualified talent in a competitive industry[465](index=465&type=chunk)[466](index=466&type=chunk) - The use of product candidates in clinical trials and commercial sales exposes the company to **product liability claims**, which could result in substantial liabilities, costs, and reputational damage[469](index=469&type=chunk) - The company's IT systems and those of its partners are **vulnerable to cybersecurity risks**, potentially leading to data loss, operational disruption, and liabilities[474](index=474&type=chunk)[477](index=477&type=chunk) - The use of new and evolving technologies, such as **AI**, may increase operational risks due to flawed algorithms, biased data, regulatory scrutiny, litigation, and potential release of confidential information[478](index=478&type=chunk)[479](index=479&type=chunk) [Risks Related to Our Ordinary Shares](index=175&type=section&id=Risks%20Related%20to%20Our%20Ordinary%20Shares) This sub-section discusses factors that could affect the market price and trading of the company's ordinary shares, including volatility, dilution from capital raises, influence of principal shareholders, reduced disclosure requirements, anti-takeover provisions, and challenges in enforcing foreign judgments - The **market price of ordinary shares is likely to be volatile** due to various factors, including clinical trial results, competitive products, regulatory developments, and general market conditions[482](index=482&type=chunk)[483](index=483&type=chunk) - **Future equity offerings**, including through the "at-the-market" program, could substantially **dilute existing shareholders' ownership interests**[484](index=484&type=chunk) - Executive officers, directors, and principal shareholders (holding **~55.2%** as of June 30, 2025) have **significant influence** over matters submitted to shareholders for approval[485](index=485&type=chunk)[488](index=488&type=chunk) - The company's status as a "**smaller reporting company**" allows for reduced disclosure, which may make its ordinary shares less attractive to some investors and increase price volatility[489](index=489&type=chunk)[490](index=490&type=chunk) - **Anti-takeover provisions** in organizational documents and Cayman Islands law may discourage or prevent a change of control, even if beneficial to shareholders[491](index=491&type=chunk) - The company may be classified as a **Passive Foreign Investment Company (PFIC)** for U.S. federal income tax purposes, which could result in adverse tax consequences for U.S. investors[498](index=498&type=chunk) [General Risk Factors](index=185&type=section&id=General%20Risk%20Factors) This sub-section covers broader operational and financial risks, including those related to acquisitions, exchange rate fluctuations, management's discretion over capital use, public company costs, analyst coverage, and environmental, social, and governance (ESG) expectations - **Engaging in acquisitions** (e.g., Smart Immune assets) could disrupt business, dilute shareholders, or reduce financial resources, with integration challenges and potential undiscovered liabilities[505](index=505&type=chunk) - **Exchange rate fluctuations**, particularly between the U.S. dollar, pound sterling, and euro, may adversely affect results of operations and financial condition due to international scope[506](index=506&type=chunk) - The company **incurs substantial costs as a public company**, requiring significant management time for compliance with new and existing initiatives and corporate governance practices[508](index=508&type=chunk) - Expectations related to **environmental, social, and governance (ESG) factors** may impose additional costs and expose the company to new risks, potentially affecting investor perception and reputation[511](index=511&type=chunk)[513](index=513&type=chunk) - The company does **not anticipate paying any cash dividends** on its ordinary shares in the foreseeable future, making capital appreciation the sole source of gain for investors[514](index=514&type=chunk) [Item 2. Unregistered Sales of Equity Securities, Use of Proceeds and Issuer Purchases of Equity Securities](index=189&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%2C%20Use%20of%20Proceeds%20and%20Issuer%20Purchases%20of%20Equity%20Securities) This section states that there were no unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities to report - **No unregistered sales of equity securities**, use of proceeds, or issuer purchases of equity securities were reported[515](index=515&type=chunk) [Item 3. Defaults Upon Senior Securities](index=189&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This section indicates that there were no defaults upon senior securities - **No defaults upon senior securities** were reported[516](index=516&type=chunk) [Item 4. Mine Safety Disclosures](index=189&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This section states that mine safety disclosures are not applicable to the company - Mine safety disclosures are **not applicable** to the company[517](index=517&type=chunk) [Item 5. Other Information](index=189&type=section&id=Item%205.%20Other%20Information) This section reports that no directors or officers adopted, modified, or terminated Rule 10b5-1 or non-Rule 10b5-1 trading arrangements during the three months ended June 30, 2025 - **No directors or officers adopted, modified, or terminated Rule 10b5-1 or non-Rule 10b5-1 trading arrangements** during the three months ended June 30, 2025[518](index=518&type=chunk) [Item 6. Exhibits](index=190&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including certifications, XBRL documents, and the cover page interactive data file - Exhibits include **certifications** (31.1, 31.2, 32.1, 32.2), **Inline XBRL Instance Document** (101.INS), and **Cover Page Interactive Data File** (104)[519](index=519&type=chunk) [Signatures](index=191&type=section&id=Signatures) This section contains the required signatures for the Form 10-Q, confirming its submission by authorized officers - The report was signed by **Alexandria Forbes** (Chief Executive Officer) and **Richard Giroux** (Chief Financial Officer and Chief Operating Officer) on August 14, 2025[523](index=523&type=chunk)

Group 1 - MeiraGTx Holdings PLC reported a quarterly loss of $0.48 per share, which was better than the Zacks Consensus Estimate of a loss of $0.52, and an improvement from a loss of $0.76 per share a year ago, resulting in an earnings surprise of +7.69% [1] - The company posted revenues of $3.69 million for the quarter ended June 2025, exceeding the Zacks Consensus Estimate by 59.78%, compared to revenues of $0.28 million in the same quarter last year [2] - MeiraGTx shares have increased approximately 37% since the beginning of the year, outperforming the S&P 500's gain of 10% [3] Group 2 - The earnings outlook for MeiraGTx is mixed, with the current consensus EPS estimate for the upcoming quarter at -$0.53 on revenues of $2.77 million, and -$0.71 on revenues of $59.52 million for the current fiscal year [7] - The Zacks Industry Rank for Medical - Biomedical and Genetics is currently in the bottom 41% of over 250 Zacks industries, indicating potential challenges for stock performance [8] - Clene Inc., another company in the same industry, is expected to report a quarterly loss of $0.49 per share, reflecting a year-over-year change of +53.8%, with revenues anticipated to be $0.1 million, up 11.1% from the previous year [9]

[Executive Summary & Corporate Update](index=1&type=section&id=Executive%20Summary%20%26%20Corporate%20Update) MeiraGTx achieved significant regulatory milestones in Q2 2025, including FDA alignment for AQUAx2, RMAT for AAV-GAD, and planned MAA/BLA filings for LCA4, while the CEO highlighted productive regulatory interactions and advancements in late-stage programs [Q2 2025 Highlights](index=1&type=section&id=Q2%202025%20Highlights) MeiraGTx reported significant regulatory and operational progress in Q2 2025, including FDA alignment for the pivotal Phase 2 AQUAx2 study for radiation-induced xerostomia (RIX), RMAT designation for AAV-GAD in Parkinson's disease, and plans to file for Marketing Authorization Approval for LCA4 in the UK and US - Gained alignment with U.S. Food and Drug Administration (FDA) on the ongoing Phase 2 AQUAx2 randomized double-blind, placebo-controlled pivotal study in Grade 2/3 radiation-induced xerostomia (RIX) to support a potential Biologics License Application (BLA) filing; on track for potential data readout late 2026[1](index=1&type=chunk) - FDA Granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD for the treatment of Parkinson's disease[1](index=1&type=chunk) - On track to file for Marketing Authorization Approval (MAA) under exceptional circumstances with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of LCA4, and BLA in the US with the FDA via a similar pathway to approval in the fourth quarter of 2025[1](index=1&type=chunk) [CEO Statement](index=1&type=section&id=CEO%20Statement) Dr. Alexandria Forbes highlighted productive regulatory interactions, successful FDA audit for AAV-GAD, and the advancement of late-stage clinical programs as planned - CEO emphasized productive regulatory interactions with MHRA and FDA around multiple later stage clinical programs as well as manufacturing[2](index=2&type=chunk) - Expressed excitement about the potential of AAV-GAD to show disease modifying changes to pathological circuitry and the substantia nigra, and the opportunity to discuss the use of AI analysis of imaging data to support labeling claims for the proposed Phase 3 study in Parkinson's disease[2](index=2&type=chunk)[3](index=3&type=chunk) - Engaged with regulators to be in a position to initiate first-in-human studies using the transformative riboswitch platform by the end of 2025[3](index=3&type=chunk) [Pipeline & Clinical Development Updates](index=2&type=section&id=Pipeline%20%26%20Clinical%20Development%20Updates) MeiraGTx advanced multiple late-stage clinical programs, including AAV2-hAQP1 for RIX, AAV-GAD for Parkinson's, AAV-AIPL1 for LCA4, and bota-vec for XLRP, achieving key regulatory designations and publishing compelling data [AAV2-hAQP1 for Radiation-Induced Xerostomia (RIX)](index=2&type=section&id=AAV2-hAQP1%20for%20Radiation-Induced%20Xerostomia%20(RIX)) The AAV2-hAQP1 program for RIX received RMAT designation and achieved FDA alignment on clinical and CMC requirements for its pivotal Phase 2 AQUAx2 study, on track for data readout in late 2026 - Granted RMAT designation by the FDA for AAV2-hAQP1 for the treatment of Grade 2/3 RIX in December 2024[5](index=5&type=chunk) - Aligned with the FDA on both the CMC and clinical requirements for the ongoing Phase 2 AQUAx2 study, including the use of a single Patient Reported Outcome (PRO) as primary endpoint and statistical analyses[5](index=5&type=chunk) - The Phase 2 AQUAx2 study is currently enrolling the final high dose cohorts, targeting completion of enrollment in the fourth quarter of 2025, with potential for pivotal data readout late 2026[5](index=5&type=chunk) [AAV-GAD for Parkinson's Disease](index=2&type=section&id=AAV-GAD%20for%20Parkinson's%20Disease) AAV-GAD received RMAT designation for Parkinson's disease, with a joint venture formed with Hologen AI to use AI-driven imaging analysis for a proposed Phase 3 study aiming for a disease modification claim - FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV-GAD for the treatment of Parkinson's disease on May 8th, 2025, following positive data from 3 clinical studies[5](index=5&type=chunk)[6](index=6&type=chunk) - MeiraGTx and partner Hologen are currently in discussion with the FDA around use of AI driven analysis of imaging data from the proposed double-blind, sham-surgery controlled Phase 3 study with the potential to support a disease modification claim on the label for AAV-GAD[8](index=8&type=chunk) - MeiraGTx and Hologen are forming a joint venture, Hologen Neuro AI Ltd, with additional committed funding from Hologen of up to **$230 million** to finance the development of the AAV-GAD program to commercialization. MeiraGTx will hold a **30% ownership** and lead clinical development and manufacturing[8](index=8&type=chunk) [AAV-AIPL1 for LCA4](index=4&type=section&id=AAV-AIPL1%20for%20LCA4) Published data for AAV-AIPL1 for LCA4 showed meaningful responses in all treated children, leading to preparations for MAA and BLA submissions supported by Orphan Drug and Rare Pediatric Disease Designations - Data demonstrating the efficacy of rAAV8.hRKp.AIPL1 for the treatment of LCA4 were published in The Lancet in February 2025, showing meaningful responses in **11 out of 11 treated children**, with all children blind at birth now able to see[13](index=13&type=chunk) - Preparing the submission of an MAA in the UK and a BLA in the US for AAV-AIPL1, a treatment for LCA4, based on clinical data and feedback from MHRA and FDA on a potentially expedited CMC PPQ package[13](index=13&type=chunk) - AAV-AIPL1 for the treatment of LCA4 has orphan drug designation in the US and EU, and Rare Pediatric Disease Designation (RPDD) from the FDA[13](index=13&type=chunk) [Botaretigene Sparoparvovec (bota-vec) for X-linked Retinitis Pigmentosa (XLRP)](index=4&type=section&id=Botaretigene%20Sparoparvovec%20(bota-vec)%20for%20X-linked%20Retinitis%20Pigmentosa%20(XLRP)) Compelling Phase 3 data for bota-vec for XLRP received strong support, with the program holding Fast Track and orphan drug designations and MeiraGTx eligible for significant milestone payments - Compelling Phase 3 data for botaretigene sparoparvovec (bota-vec) for the treatment of X-linked retinitis pigmentosa was presented, leading to strong public support for its filing and ultimate approval from the Foundation Fighting Blindness[13](index=13&type=chunk) - The FDA has granted Fast Track and orphan drug designations to bota-vec, and EU regulatory authorities have granted Priority Medicines (PRIME), advanced therapy medicinal product (ATMP), and orphan drug designations[13](index=13&type=chunk) - MeiraGTx is eligible to receive up to **$285 million** upon the 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[13](index=13&type=chunk)[14](index=14&type=chunk) [Technology Platforms](index=5&type=section&id=Technology%20Platforms) MeiraGTx is advancing its riboswitch gene regulation technology for in vivo delivery, focusing on metabolic diseases and CAR-T, and acquired ProTcell technology to enhance allogeneic RiboCAR-T therapies [Riboswitch Gene Regulation Technology](index=5&type=section&id=Riboswitch%20Gene%20Regulation%20Technology) The riboswitch platform is progressing for in vivo delivery, initially focusing on obesity, metabolic disease, neuropathic pain, and CAR-T, with first-in-human studies anticipated in 2025 - MeiraGTx continues to progress its riboswitch technology platform in multiple potential indications, with an initial focus on obesity and metabolic disease, neuropathic pain and CAR-T[18](index=18&type=chunk) - Generated compelling preclinical data with metabolic peptides and hormones, including incretins, myokines and leptin, indicating greater efficacy on weight loss and positive impact on fat to muscle ratio and post prandial glucose control[18](index=18&type=chunk) - Pre-clinical data from Riboswitch delivered leptin is particularly compelling and likely to be the first IND using the Company's riboswitch small molecule platform, with first-in-human studies intended to initiate in 2025[18](index=18&type=chunk) [ProTcell Technology & RiboCAR-T](index=5&type=section&id=ProTcell%20Technology%20%26%20RiboCAR-T) MeiraGTx acquired ProTcell technology to complement its RiboCAR platform, enabling the generation of T-cell progenitors outside the body for allogeneic high-performance RiboCAR-T therapies - Acquired ProTcell technology via the acquisition of certain assets and operations of Smart Immune, which allows T-cell progenitors to be generated outside the body, complementing the Company's RiboCAR platform[18](index=18&type=chunk) - This technology provides a unique potential for allogeneic high performance RiboCAR-T, with ProTcell technology having shown proof of concept in **20 patients** treated in **3 clinical studies**[18](index=18&type=chunk) [Manufacturing Capabilities](index=5&type=section&id=Manufacturing%20Capabilities) MeiraGTx maintains robust manufacturing capabilities with two licensed facilities in the UK and Ireland, ensuring GMP compliance and readiness for commercial production of gene therapies [United Kingdom Facility](index=5&type=section&id=United%20Kingdom%20Facility) MeiraGTx's UK manufacturing facility holds two MHRA authorizations and successfully renewed its licenses following a May 2024 inspection, confirming GMP compliance for IMPs and readiness for a commercial MIA license application - MeiraGTx's UK manufacturing facility holds two authorizations issued by the MHRA[16](index=16&type=chunk) - The UK facility was inspected in May 2024, and the licences were successfully renewed, confirming GMP compliance for Investigational Medicinal Products (IMPs) and readiness to support an application for a commercial MIA licence[16](index=16&type=chunk) [Ireland Facility](index=5&type=section&id=Ireland%20Facility) MeiraGTx's Shannon facility holds two HPRA authorizations, including an MIA Licence for QC testing and an MIA(IMP) Licence for manufacturing, with a recent inspection adding viral vector manufacturing capability - MeiraGTx's Shannon facility holds two authorizations issued by Ireland's Health Products Regulatory Authority (HPRA): MIA Licence (M1316) for QC testing of commercial products and MIA(IMP) Licence (MIA(IMP) 45522) for manufacturing, fill-finish, and QC testing of Investigational Medicinal Products (IMPs)[17](index=17&type=chunk)[18](index=18&type=chunk) - The latest HPRA inspection in February 2025 was highly successful, renewing both QC licenses and adding viral vector manufacturing to the MIA(IMP) license, a first-of-its-kind license for a gene therapy facility in Ireland[20](index=20&type=chunk) [Financial Review](index=6&type=section&id=Financial%20Review) MeiraGTx reported $32.2 million in cash as of Q2 2025, with a positive financial outlook into 2027 supported by the Hologen collaboration and potential bota-vec milestones, while Q2 2025 saw increased service revenue and an improved net loss [Financial Outlook & Liquidity](index=6&type=section&id=Financial%20Outlook%20%26%20Liquidity) As of June 30, 2025, MeiraGTx had $32.2 million in cash and cash equivalents, expecting to fund operations into 2027 and repay its $75.0 million debt with anticipated proceeds from the Hologen collaboration and bota-vec milestones Cash and Receivables (as of June 30, 2025) | Item | Amount (Millions) | | :-------------------------- | :---------------- | | Cash and cash equivalents | $32.2 | | Receivables (Johnson & Johnson) | $2.3 | | Tax incentive receivables | $4.5 | - The Company believes that with current funds, **$17.0 million** received in Q3 2025, and remaining proceeds from the Hologen collaboration, it will have sufficient capital to fund operating expenses and capital expenditure requirements into **2027** and to repay its debt obligation of **$75.0 million** (due August 2026)[21](index=21&type=chunk) - This estimate does not include the **$285.0 million** in milestones the Company is eligible to receive under the asset purchase agreement upon first commercial sale of bota-vec in the US and EU, manufacturing tech transfer, and regulatory approval of J&J's manufacturing facility[21](index=21&type=chunk) [Q2 2025 Financial Results](index=6&type=section&id=Q2%202025%20Financial%20Results) For Q2 2025, MeiraGTx reported a significant increase in service revenue to $3.7 million, with a net loss attributable to ordinary shareholders improving to $38.8 million, resulting in a lower basic and diluted net loss per share of $0.48 Q2 2025 vs Q2 2024 Key Financials (in thousands) | Metric | Q2 2025 | Q2 2024 | Change (YoY) | | :-------------------------------- | :------ | :------ | :----------- | | Service revenue | $3,691 | $282 | +$3,409 | | Cost of service revenue | $2,676 | $0 | +$2,676 | | General and administrative expenses | $12,313 | $11,257 | +$1,056 | | Research and development expenses | $33,495 | $34,934 | -$1,439 | | Foreign currency gain (loss) | $8,624 | $(284) | +$8,908 | | Interest income | $408 | $827 | -$419 | | Interest expense | $3,034 | $3,254 | -$220 | | Net loss | $(38,795) | $(48,620) | +$9,825 | | Basic and diluted net loss per share | $(0.48) | $(0.76) | +$0.28 | - Service revenue increased by **$3.4 million** for the three months ended June 30, 2025, compared to the prior year, due to increased progress of PPQ services under the asset purchase agreement with Johnson & Johnson Innovative Medicine[22](index=22&type=chunk) - Research and development expenses decreased by **$1.4 million**, primarily due to reclassification of batch costs and cost of service revenue, and reduced expenses for certain programs, partially offset by increased clinical trial expenses for the AAV-GAD program[25](index=25&type=chunk) - Foreign currency gain was **$8.6 million** for Q2 2025, a significant change from a **$0.3 million** loss in Q2 2024, primarily due to the weakening of the U.S. dollar against the pound sterling and euro[26](index=26&type=chunk) [Company Overview](index=7&type=section&id=About%20MeiraGTx) MeiraGTx is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of four late-stage clinical programs, leveraging innovative technology for capsid, promoter, and translational control optimization, supported by extensive in-house manufacturing capabilities and a transformative riboswitch gene regulation technology - MeiraGTx is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of four late-stage clinical programs, using local delivery of small doses for disease modifying effects in inherited and common diseases[30](index=30&type=chunk) - Utilizes innovative technology in optimization of capsids, promoters and novel translational control elements to develop best in class, potent, safe viral vectors[30](index=30&type=chunk) - Possesses comprehensive end-to-end in-house manufacturing capabilities with **5 facilities globally**, including two licensed for GMP viral vector production and a GMP QC facility with clinical and commercial licensure[30](index=30&type=chunk) - Developed a novel riboswitch gene regulation technology for in vivo delivery of any biologic therapeutic using oral small molecules, focusing on regulated in vivo delivery of metabolic peptides (e.g., GLP-1, Leptin) and cell therapy (CAR-T)[31](index=31&type=chunk) [Forward-Looking Statements](index=8&type=section&id=Forward%20Looking%20Statement) This section contains forward-looking statements regarding product development, clinical trial milestones, regulatory matters, potential milestone payments, and the Hologen collaboration, which are subject to risks and uncertainties - This press release contains forward-looking statements regarding product candidate development, anticipated milestones, clinical data, regulatory matters, potential milestone payments, and the collaboration with Hologen[32](index=32&type=chunk) - These statements are based on management's current expectations and involve known and unknown risks, uncertainties, and other important factors that may cause actual results, performance or achievements to be materially different[32](index=32&type=chunk) - The company disclaims any obligation to update such forward-looking statements at some point in the future, unless required by law[33](index=33&type=chunk) [Contacts](index=9&type=section&id=Contacts) This section provides contact information for investor relations and media inquiries [Financial Statements](index=10&type=section&id=Financial%20Statements) The financial statements present the unaudited condensed consolidated statements of operations and balance sheets, detailing the company's financial performance and position for Q2 2025 and year-to-date [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) The unaudited condensed consolidated statements of operations show a net loss of $38.8 million for Q2 2025, an improvement from $48.6 million in Q2 2024, driven by increased service revenue and slightly decreased R&D expenses Condensed Consolidated Statements of Operations and Comprehensive Loss (Q2 2025 vs Q2 2024, in thousands) | Metric | Q2 2025 | Q2 2024 | 6M 2025 | 6M 2024 | | :-------------------------------- | :------ | :------ | :------ | :------ | | Service revenue | $3,691 | $282 | $5,617 | $979 | | Cost of service revenue | $2,676 | — | $4,054 | — | | General and administrative | $12,313 | $11,257 | $21,677 | $24,404 | | Research and development | $33,495 | $34,934 | $66,275 | $69,256 | | Loss from operations | $(44,793) | $(45,909) | $(86,389) | $(92,681) | | Foreign currency gain (loss) | $8,624 | $(284) | $12,311 | $(819) | | Interest income | $408 | $827 | $1,379 | $1,924 | | Interest expense | $(3,034) | $(3,254) | $(6,077) | $(6,504) | | Net loss | $(38,795) | $(48,620) | $(78,776) | $(69,062) | | Basic and diluted net loss per share | $(0.48) | $(0.76) | $(0.99) | $(1.08) | | Weighted-average shares outstanding | 80,585,625 | 64,376,396 | 79,813,273 | 64,221,145 | [Condensed Consolidated Balance Sheets](index=11&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) The unaudited condensed consolidated balance sheets show total assets decreased to $198.7 million as of June 30, 2025, from $269.8 million at December 31, 2024, with a significant decrease in cash and cash equivalents Condensed Consolidated Balance Sheets (as of June 30, 2025 vs Dec 31, 2024, in thousands) | Asset/Liability/Equity | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | **ASSETS:** | | | | Cash and cash equivalents | $32,166 | $103,659 | | Total Current Assets | $47,084 | $123,518 | | Property, plant and equipment, net | $110,816 | $102,878 | | TOTAL ASSETS | $198,716 | $269,751 | | **LIABILITIES:** | | | | Total Current Liabilities | $53,436 | $60,783 | | Deferred revenue - related party | $63,044 | $57,576 | | Note payable, net | $73,773 | $73,221 | | TOTAL LIABILITIES | $195,756 | $201,924 | | **SHAREHOLDERS' EQUITY:** | | | | Total Shareholders' Equity | $2,960 | $67,827 |

Core Insights - MeiraGTx Holdings plc has made significant progress in its clinical programs, including alignment with the FDA on the Phase 2 study of AAV-hAQP1 for radiation-induced xerostomia, which is expected to support a Biologics License Application (BLA) filing by late 2026 [1][2] - The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD, a gene therapy for Parkinson's disease, indicating its potential to address serious conditions [1][5] - The company is preparing to file for Marketing Authorization Approval (MAA) in the UK and BLA in the US for AAV-AIPL1, targeting LCA4, with submissions expected in Q4 2025 [1][2] Regulatory Developments - The company has had productive interactions with the FDA and MHRA regarding multiple late-stage clinical programs and manufacturing processes [2] - AAV-GAD has shown significant disease-modifying effects in clinical trials, with plans to initiate a Phase 3 study later this year [1][6] - The FDA has completed a Good Clinical Practice inspection of the AAV-GAD bridging study, resulting in a clean inspection with no observations [6][8] Financial Performance - As of June 30, 2025, the company reported cash and cash equivalents of approximately $32.2 million, a decrease from $101.0 million a year earlier [18][19] - Service revenue increased to $3.7 million for Q2 2025, up from $0.3 million in Q2 2024, attributed to progress in process performance qualification services [19][22] - The net loss attributable to ordinary shareholders for Q2 2025 was $38.8 million, compared to a net loss of $48.6 million in Q2 2024, indicating an improvement in financial performance [28][32] Clinical Programs and Collaborations - The company is advancing its late-stage clinical programs, including AAV-hAQP1 for xerostomia and AAV-GAD for Parkinson's disease, with pivotal data readouts expected in late 2026 [1][2] - MeiraGTx is collaborating with Hologen to develop AAV-GAD, with a joint venture established to finance the program and utilize AI for data analysis [6][8] - The company is also preparing to initiate first-in-human studies using its riboswitch platform by the end of 2025 [2][20] Manufacturing and Technology - MeiraGTx has developed comprehensive in-house manufacturing capabilities, with facilities in the UK and Ireland holding necessary regulatory authorizations [15][17] - The company is focusing on optimizing its riboswitch gene regulation technology for various therapeutic applications, including metabolic diseases and chronic pain [29][30] - The manufacturing process for AAV2-hAQP1 will be in-house, ensuring consistency across different indications [5][8]

Clinical Trial Results - MeiraGTx's rAAV8.hRKp.AIPL1 gene therapy has shown efficacy in all 11 children (aged 1-4 years) treated for AIPL1 Retinal Dystrophy (LCA4) [3] - In the unilateral treatment group (4 patients), durable efficacy has been observed for up to 4 years [3] - In the unilateral treatment group, visual acuities of treated eyes improved to a mean of 0.9 LogMAR, while untreated eyes showed no improvement [6] - All 7 bilaterally-treated children showed meaningful improvements in functional vision, visual acuity, and neurophysiology [14] - Binocular visual acuities in bilaterally-treated children improved to a mean of 1.0 LogMAR after a mean follow-up of 5 months [14] Regulatory Status - MeiraGTx was granted an Innovation Passport designation in the UK for rAAV8.hRKp.AIPL1 [16] - MeiraGTx has been advised to file for 'Marketing Authorization Under Exceptional Circumstance' in the UK based on data from 11 children [16] - AAV-AIPL1 has orphan status in the US and EU and Rare Pediatric Disease Designation in the US, making it eligible for a PRV voucher upon approval [16] Overall Impact - Treatment with rAAV8.hRKp.AIPL1 has resulted in improvements in visual function, retinal function, and visual behavior in treated children [15] - These visual improvements have led to life-changing benefits in communication, behavior, schooling, mood, psychological well-being, and social integration [15]

Clinical Pipeline and Regulatory Milestones - The company has 3 late-stage clinical programs in pivotal/Phase 3 trials for prevalent non-inherited indications[4,7,30,36,42,90] - Potential global filings are anticipated in 2025, 2026, and 2027[7,30,36,42,90] - The company has a deep pre-IND pipeline targeting conditions like ALS, MC4R obesity, and metabolic disease[7,8,32,38,45,93] Manufacturing and Technology - The company operates 2 GMP facilities at commercial scale[5,9,33,39,46,94] - Proprietary vectorization technology increases potency by 2-10x from the same promoter[5,9,34,40,47,95] - AI-driven improvements are based on over 20 vectors and more than 50 GMP runs[5,9,33,39,46,94] Partnerships and Financials - MeiraGTx will receive up to $415 million from Janssen through an asset purchase agreement[11] - Sanofi made a $30 million strategic investment through the sale of 4 million ordinary shares at $7.50 per share[11] AAV-AQP1 for Radiation Induced Xerostomia - There are 170,000 Grade 2/3 RIX patients in the US[13] - There are 15,000 new cases of grade 2/3 RIX annually in the US[13] AAV-GAD for Parkinson's Disease - There are 10 million Parkinson's patients worldwide[18,28] - The estimated economic burden of Parkinson's Disease in the US is $52 billion[19]

Industry Overview - The field of genomics has rapidly evolved over the past decade, focusing on the complete set of genes and their interactions rather than individual genes [1] - Genomics is pivotal for developing targeted therapies, leading to a revolutionary era in genetic medicine, attracting significant attention from pharmaceutical and biotech companies [2] Technological Innovations - Synthetic biology, which applies engineering principles to biology, has emerged as a key concept within genomics, aiding in drug discovery, disease detection, and gene editing [3] - The cost, accuracy, and time required to map an individual's genome have drastically reduced, enhancing the spotlight on genomics companies [4] Market Potential - The genomics market is projected to reach $157.47 billion by 2033, while the global synthetic biology market was valued at $16.22 billion in 2024, with a projected CAGR of 17.30% from 2025 to 2030 [6] Company Highlights - MeiraGTx Holdings plc is focused on genetic medicine with a pipeline addressing conditions like Parkinson's disease and retinal dystrophy, showing promising efficacy data [8][9] - Beam Therapeutics is advancing base editing programs for genetic diseases, with FDA orphan drug designation for its BEAM-101 treatment for sickle cell disease [12][13] - Krystal Biotech received FDA approval for Vyjuvek, the first gene therapy for dystrophic epidermolysis bullosa, and is advancing a pipeline in various therapeutic areas [14][15]