LONDON and NEW YORK, April 24, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical stage gene therapy company, today announced that Alexandria Forbes, Ph.D., President and Chief Executive Officer, will participate in the following investor conferences: Chardan 8th Annual Genetic Medicines and Cell Therapy Manufacturing Summit, Virtual Corporate presentation: Monday, April 29, 2024, at 11:00 a.m. ET  BofA Securities Healthcare Conference 2024, Las Vegas Corporate p ...

LONDON and NEW YORK, April 18, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical stage gene therapy company, today announced the Company gave an oral presentation at the American Academy of Oral Medicine Annual Conference, being held from April 16-20, 2024, at the Hyatt Regency Grand Cypress in Orlando, FL. The details of the oral presentation are below: Session: Oral Abstract Session IPresentation ID #196Title: Results of a Phase 1, Open-label, Dose-escalation ...

Financial Performance - The company has incurred significant losses since inception and anticipates continued losses for the foreseeable future, with no guarantee of achieving profitability[16]. - The net loss for 2023 was $84,027,000, an improvement from a net loss of $129,615,000 in 2022, reflecting a reduction of 35.2%[688]. - The company's pre-tax loss for 2023 was $84,027 million, an improvement from a loss of $129,615 million in 2022[812]. - The Company reported a gain on the sale of nonfinancial assets amounting to $54,208,000 in 2023[688]. - The Company recorded basic and diluted net loss per ordinary share as the same due to anti-dilutive effects of additional ordinary share equivalents[774]. Revenue and Income - The company recognized deferred revenue of $36.9 million and a gain on the sale of non-financial assets amounting to $54.2 million as of December 31, 2023, due to agreements with Janssen Pharmaceuticals[681]. - The company recognized $70.4 million and $73.3 million related to the reimbursement of research and development expenses under the collaboration agreement for the years ended December 31, 2023, and 2022, respectively[834]. - The collaboration agreement with Janssen includes a 20% royalty on net sales of products and additional milestones up to $340.0 million[822]. - The Company received a non-refundable upfront cash payment of $65.0 million from Janssen Pharmaceuticals in December 2023 as part of an Asset Purchase Agreement[696]. - The Company received a non-refundable upfront fee of $100.0 million from Janssen under a collaboration agreement for gene therapies[819]. Assets and Liabilities - Total current assets increased to $159,622,000 in 2023 from $154,339,000 in 2022, representing a growth of 3.3%[687]. - The company’s total liabilities decreased to $188,567,000 in 2023 from $200,499,000 in 2022, a reduction of 5.9%[687]. - Cash and cash equivalents at the end of 2023 were $129,566,000, up from $115,516,000 at the end of 2022, indicating an increase of 12.1%[687]. - The Company recorded unrecognized tax positions of $2.0 million, up from $0.9 million in 2022[772]. - The balance of asset retirement obligations increased from $2,179,000 in 2022 to $2,401,000 in 2023, reflecting amortization of interest and exchange rate changes[734]. Expenses - Total operating expenses rose to $151,078,000 in 2023, compared to $132,275,000 in 2022, marking an increase of 14.2%[688]. - The Company recorded depreciation and amortization expense of $12,300,000 in 2023, up from $7,300,000 in 2022, marking an increase of approximately 68.8%[790]. - The total compensation expense related to unvested options as of December 31, 2023, was $18.3 million, expected to be recognized over 3.7 years[800]. - Total share-based compensation expense for 2023 was $27.716 million, a decrease of 3.2% from $28.623 million in 2022[804]. - The total rent expense recorded under leases was $5.5 million for the year ended December 31, 2023, compared to $5.3 million for 2022[839]. Cash Flow - The company’s cash flows from operating activities showed a net cash used of $105,365,000 in 2023, compared to $73,098,000 in 2022, indicating an increase in cash outflow[693]. - For the year ended December 31, 2023, the Company used $105.4 million in cash flows from operations, with no assurances of generating positive cash flows in the future[699]. - As of December 31, 2023, the Company had cash, cash equivalents, and restricted cash totaling $130.6 million, which is expected to cover expenses for at least the next twelve months[700]. Capital and Financing - The annual interest rate on borrowings under the Notes Purchase Agreement was 15.32% as of December 31, 2023, with an outstanding balance of $75.0 million[669]. - A hypothetical 1% increase in the secured overnight financing rate (SOFR) would increase annual interest expense by approximately $0.8 million for the year ended December 31, 2023[669]. - The outstanding balance of the Tranche 1 Notes was $75.0 million plus accrued interest of $3.0 million as of December 31, 2023[848]. - The Company recorded interest expense of $11.3 million for the year ended December 31, 2023, compared to $4.0 million for 2022[848]. - The Notes Purchase Agreement matures on August 2, 2026, and is interest-only during the term[848]. 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[16]. - Clinical trials are expensive and time-consuming, with potential delays that could impact the company's operations[16]. - The Company recorded reductions to research and development expenses of $2.9 million and $6.8 million for the years ended December 31, 2023 and 2022, respectively, related to UK tax incentive programs[709]. - Research and development costs are charged to expense as incurred, including employee-related expenses and costs associated with clinical studies[767]. - The Company is focusing on developing a riboswitch platform for metabolic peptides and cell therapy for oncology and autoimmune diseases[694]. Regulatory and Market Risks - The company faces significant competition in a rapidly changing technological environment, which may affect its financial condition and ability to market products[23]. - The affected populations for the company's product candidates may be smaller than projected, impacting the addressable markets[16]. - The Company is subject to significant risks, including competition, regulatory challenges, and reliance on third parties for its operations[701]. - The UK corporation tax rate will increase from 19% to 25% effective April 1, 2023, impacting future accounting periods[815]. Shareholder and Equity Information - Shareholders' equity increased to $138,177,000 in 2023 from $117,738,000 in 2022, a rise of 17.3%[687]. - The total number of options outstanding increased from 6,858,409 in 2022 to 8,226,707 in 2023, a rise of about 20%[797]. - The weighted average grant date fair value of options granted in 2023 was $5.67, significantly lower than $12.81 in 2022[799]. - The Company issued 10,773,913 ordinary shares in a private placement in May 2023, raising approximately $62.0 million[807]. - In October 2023, the company issued 4,000,000 ordinary shares to Sanofi for gross proceeds of $30.0 million[808].

Dr. Forbes continued, "In the area of ophthalmology, in the third quarter of 2023, we completed enrollment in the large multi-center Phase 3 study of bota-vec for XLRP- RPGR in collaboration with Janssen. Data from this global pivotal study is expected after the third quarter of 2024. Additionally, in the retinal disease space, we recently received data from our ongoing compassionate use program under a Specials License for children with LCA4 due to mutations in the AIPL1 gene. MeiraGTx developed and optimi ...

[PART I. FINANCIAL INFORMATION](index=7&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) The unaudited condensed consolidated financial statements for the period ended September 30, 2023, reflect the company's financial position, results of operations, and cash flows Condensed Consolidated Balance Sheet Data (unaudited) | Account | September 30, 2023 ($ in thousands) | December 31, 2022 ($ in thousands) | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | 63,365 | 115,516 | | Total Current Assets | 103,531 | 154,339 | | TOTAL ASSETS | 267,194 | 318,237 | | **Liabilities & Equity** | | | | Total Current Liabilities | 63,514 | 82,072 | | TOTAL LIABILITIES | 175,308 | 200,499 | | Accumulated deficit | (574,446) | (470,204) | | Total Shareholders' Equity | 91,886 | 117,738 | | TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY | 267,194 | 318,237 | Condensed Consolidated Statements of Operations (unaudited) | Metric ($ in thousands, except per share) | Q3 2023 | Q3 2022 | 9 Months 2023 | 9 Months 2022 | | :--- | :--- | :--- | :--- | :--- | | License revenue - related party | 5,103 | 4,816 | 11,977 | 21,208 | | General and administrative | 10,009 | 10,762 | 35,169 | 32,548 | | Research and development | 27,856 | 16,862 | 70,115 | 63,960 | | Loss from operations | (32,762) | (22,808) | (93,307) | (75,300) | | Net loss | (44,297) | (37,284) | (104,242) | (102,302) | | Basic and diluted net loss per share | (0.74) | (0.83) | (1.91) | (2.29) | Condensed Consolidated Statements of Cash Flows (unaudited) | Cash Flow Activity ($ in thousands) | Nine-Month Period Ended Sep 30, 2023 | Nine-Month Period Ended Sep 30, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | (90,818) | (56,831) | | Net cash used in investing activities | (17,144) | (36,471) | | Net cash provided by financing activities | 56,312 | 70,367 | - On October 30, 2023, the company entered into an Investment Agreement with Sanofi, issuing 4,000,000 ordinary shares for gross proceeds of **$30.0 million**; the agreement also grants Sanofi a right of first negotiation for the company's Riboswitch gene regulation technology and other specified programs[149](index=149&type=chunk)[152](index=152&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=50&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management analyzes the company's financial condition and results, noting its clinical-stage status, history of losses, and liquidity outlook into mid-2025 - The company is a clinical-stage gene therapy company with significant operating losses since inception, reporting a net loss of **$104.2 million** for the nine months ended September 30, 2023, and an accumulated deficit of **$574.4 million**[155](index=155&type=chunk)[157](index=157&type=chunk) - Based on existing cash, expected research funding, and Sanofi investment proceeds, the company estimates sufficient funds to support operations into **mid-2025**[164](index=164&type=chunk)[217](index=217&type=chunk) - Recent strategic and clinical milestones include: - A **$30.0 million** strategic investment from Sanofi in October 2023 - Completion of enrollment in the pivotal Phase 3 LUMEOS trial for bota-vec for XLRP - Initiation of a Phase 2 study for AAV-hAQP1 for radiation-induced xerostomia - Anticipated completion of enrollment in the Phase 1 AAV-GAD trial for Parkinson's disease in Q4 2023[169](index=169&type=chunk)[171](index=171&type=chunk) [Results of Operations](index=60&type=section&id=Results%20of%20Operations) Net loss increased to **$44.3 million** in Q3 2023 and **$104.2 million** for the nine-month period, primarily due to higher R&D expenses driven by manufacturing costs Comparison of Results for the Three Months Ended September 30, | Metric ($ in thousands) | 2023 | 2022 | Change | | :--- | :--- | :--- | :--- | | License revenue - related party | 5,103 | 4,816 | 287 | | General and administrative | 10,009 | 10,762 | (753) | | Research and development | 27,856 | 16,862 | 10,994 | | Loss from operations | (32,762) | (22,808) | (9,954) | | Net loss | (44,297) | (37,284) | (7,013) | - The **$11.0 million** increase in R&D expenses for Q3 2023 was primarily driven by an **$18.0 million** increase in manufacturing costs, partially offset by decreases in preclinical program expenses and lower reimbursement from the Janssen collaboration[198](index=198&type=chunk) Comparison of Results for the Nine Months Ended September 30, | Metric ($ in thousands) | 2023 | 2022 | Change | | :--- | :--- | :--- | :--- | | License revenue - related party | 11,977 | 21,208 | (9,231) | | General and administrative | 35,169 | 32,548 | 2,621 | | Research and development | 70,115 | 63,960 | 6,155 | | Loss from operations | (93,307) | (75,300) | (18,007) | | Net loss | (104,242) | (102,302) | (1,940) | - For the nine-month period, R&D expenses increased by **$6.2 million**, mainly due to a **$39.2 million** increase in manufacturing costs, partially offset by decreases of **$12.2 million** in preclinical program expenses and **$9.5 million** in clinical trial expenses[210](index=210&type=chunk) [Liquidity and Capital Resources](index=67&type=section&id=Liquidity%20and%20Capital%20Resources) As of September 30, 2023, the company had **$64.4 million** in cash, with net cash used in operating activities at **$90.8 million**, and **$56.3 million** provided by financing activities - As of September 30, 2023, the company had cash, cash equivalents, and restricted cash of **$64.4 million**, along with **$22.4 million** in receivables due from Janssen[156](index=156&type=chunk)[219](index=219&type=chunk) - Net cash used in operating activities increased to **$90.8 million** for the nine months ended September 30, 2023, from **$56.8 million** in the prior-year period, primarily due to the net loss and changes in operating assets and liabilities[219](index=219&type=chunk)[220](index=220&type=chunk)[221](index=221&type=chunk) - Net cash provided by financing activities was **$56.3 million** for the nine months ended September 30, 2023, mainly from the **$57.8 million** net proceeds of the May 2023 private placement[224](index=224&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=71&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company faces market risks primarily from foreign currency exchange rate fluctuations and interest rate changes on its SOFR-linked borrowings - A hypothetical **10%** unfavorable movement in foreign currency exchange rates would result in an additional foreign currency loss of approximately **$29.0 million** for the nine months ended September 30, 2023[229](index=229&type=chunk) - The company's debt bears interest at **10.00%** plus SOFR (subject to a **1.00%** floor); a hypothetical **1%** increase in SOFR would increase annual interest expense by approximately **$0.8 million** based on the **$75.0 million** outstanding balance as of September 30, 2023[230](index=230&type=chunk) [Controls and Procedures](index=72&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of September 30, 2023, with no material changes to internal control over financial reporting during the quarter - The CEO and CFO concluded that the company's disclosure controls and procedures were effective at the reasonable assurance level as of the end of the period covered by the report[232](index=232&type=chunk) - There were no changes in internal control over financial reporting during the quarter ended September 30, 2023, that have materially affected, or are reasonably likely to materially affect, internal controls[233](index=233&type=chunk) [PART II. OTHER INFORMATION](index=73&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Legal Proceedings](index=73&type=section&id=Item%201.%20Legal%20Proceedings) The company reports that it is not currently subject to any material legal proceedings - The company is not subject to any material legal proceedings[235](index=235&type=chunk) [Risk Factors](index=73&type=section&id=Item%201A.%20Risk%20Factors) This section outlines substantial investment risks, including the company's history of losses, dependence on clinical-stage candidates, development and regulatory challenges, and competition - **Financial Risks**: The company has a history of significant losses, with an accumulated deficit of **$574.4 million** as of September 30, 2023, and anticipates continued losses, requiring additional capital that may not be available on acceptable terms[237](index=237&type=chunk) - **Development and Regulatory Risks**: The business heavily depends on the success of its advanced product candidates, operating in a novel gene therapy field with an uncertain and evolving regulatory landscape, making approval time and cost difficult to predict[255](index=255&type=chunk)[264](index=264&type=chunk) - **Manufacturing and Third-Party Risks**: The company is subject to significant manufacturing facility regulation, relies on third parties for components like plasmid, posing supply disruption risks, and depends on collaborators for key program development and commercialization[310](index=310&type=chunk)[417](index=417&type=chunk)[426](index=426&type=chunk) - **Commercialization Risks**: The company faces significant competition from larger pharmaceutical and biotech companies, with successful commercialization dependent on adequate payor reimbursement, market acceptance, and establishing sales and marketing capabilities[388](index=388&type=chunk)[392](index=392&type=chunk) [Unregistered Sales of Equity Securities, Use of Proceeds and Issuer Purchases of Equity Securities](index=184&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities,%20Use%20of%20Proceeds%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company reported no unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities during the period - None reported for the period[539](index=539&type=chunk) [Other Information](index=184&type=section&id=Item%205.%20Other%20Information) The company reported no other information required to be disclosed in this item for the period - None reported for the period[543](index=543&type=chunk) [Exhibits](index=185&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including amendments to financing agreements, the Investment Agreement with Sanofi, officer certifications, and XBRL data files - Key exhibits filed include the Consent and Amendment to the Note Purchase Agreement with Perceptive, the Investment Agreement with Sanofi, and the associated Registration Rights Agreement[545](index=545&type=chunk)

PART I. FINANCIAL INFORMATION [Financial Statements (Unaudited)](index=8&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents MeiraGTx Holdings plc's unaudited condensed consolidated financial statements for Q2 2023, including balance sheets, statements of operations, shareholders' equity, and cash flows Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2023 | December 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $92,773 | $115,516 | | Total Current Assets | $143,144 | $154,339 | | TOTAL ASSETS | $310,263 | $318,237 | | **Liabilities & Equity** | | | | Total Current Liabilities | $71,803 | $82,072 | | Note payable, net | $71,571 | $71,033 | | TOTAL LIABILITIES | $186,736 | $200,499 | | Total Shareholders' Equity | $123,527 | $117,738 | Condensed Consolidated Statements of Operations Highlights (in thousands, except per share data) | Metric | Three Months Ended June 30, 2023 | Three Months Ended June 30, 2022 | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | License revenue - related party | $3,540 | $10,759 | $6,874 | $16,392 | | Research and development | $19,937 | $23,999 | $42,259 | $47,098 | | Total operating expenses | $32,325 | $34,517 | $67,419 | $68,884 | | Net loss | $(29,581) | $(33,973) | $(59,945) | $(65,018) | | Basic and diluted net loss per share | $(0.53) | $(0.76) | $(1.15) | $(1.46) | Condensed Consolidated Statements of Cash Flows Highlights (in thousands) | Cash Flow Activity | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | $(66,404) | $(36,105) | | Net cash used in investing activities | $(13,580) | $(26,460) | | Net cash provided by (used in) financing activities | $56,716 | $(2,582) | | **Net decrease in cash and cash equivalents** | **$(23,268)** | **$(65,147)** | [Notes to Condensed Consolidated Financial Statements](index=13&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Detailed notes supplement the financial statements, covering company organization, liquidity, accounting policies, and specific financial line items - The company is a clinical-stage gene therapy company with six programs in clinical development, initially focusing on ocular diseases, neurodegenerative diseases, and severe forms of xerostomia[30](index=30&type=chunk) - Management estimates that cash and cash equivalents of **$92.8 million**, along with receivables from its collaboration with Janssen, will be sufficient to cover expenses for at least the next twelve months from the issuance date of the financial statements[36](index=36&type=chunk) - Revenue from the Janssen Collaboration Agreement is recognized using a cost-based input method, where progress towards completion is measured based on the ratio of actual costs incurred to total estimated costs[71](index=71&type=chunk)[122](index=122&type=chunk) Share-Based Compensation Expense (in thousands) | Period | Research and Development | General and Administrative | Total | | :--- | :--- | :--- | :--- | | **Three Months Ended June 30, 2023** | $3,293 | $3,978 | $7,271 | | **Three Months Ended June 30, 2022** | $3,666 | $3,637 | $7,303 | | **Six Months Ended June 30, 2023** | $6,319 | $7,384 | $13,703 | | **Six Months Ended June 30, 2022** | $6,319 | $7,583 | $14,881 | - The company entered into a senior secured financing agreement with Perceptive Credit Holdings III, LP, providing an initial **$75.0 million** in notes (Tranche 1 Notes), with an annual interest rate of **14.81%** at June 30, 2023[141](index=141&type=chunk)[142](index=142&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=49&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses financial condition and operational results, including business overview, clinical highlights, comparative analysis, and liquidity and capital resources - As of June 30, 2023, the company had cash and cash equivalents of **$92.8 million** and an accumulated deficit of **$530.1 million**, financing operations through equity sales, debt, and Janssen collaboration payments[154](index=154&type=chunk)[155](index=155&type=chunk) - The company estimates its current cash, cash equivalents, and expected research funding will be sufficient to fund operating expenses and capital requirements into the **second quarter of 2025**[161](index=161&type=chunk)[217](index=217&type=chunk) - Recent operational highlights include surpassing enrollment targets for the Phase 3 LUMEOS trial, initiating a Phase 2 study for AAV-hAQP1, receiving commercial MIA authorization for its Shannon QC facility, and dosing patients in the AAV-GAD clinical trial[168](index=168&type=chunk) Comparison of Results of Operations (in thousands) | Metric | Three Months Ended June 30, 2023 | Three Months Ended June 30, 2022 | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | License revenue - related party | $3,540 | $10,759 | $6,874 | $16,392 | | Research and development | $19,937 | $23,999 | $42,259 | $47,098 | | General and administrative | $12,388 | $10,518 | $25,160 | $21,786 | | Net loss | $(29,581) | $(33,973) | $(59,945) | $(65,018) | [Quantitative and Qualitative Disclosures About Market Risk](index=70&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section details the company's exposure to market risks, including foreign currency exchange risk from international operations and interest rate risk from variable-rate debt - The company is exposed to foreign currency risk between the U.S. Dollar, British pound sterling, and euro; a hypothetical **10% unfavorable movement** would result in an additional foreign currency loss of approximately **$28.6 million** for the six months ended June 30, 2023[229](index=229&type=chunk) - The company is exposed to interest rate risk from its Note Purchase Agreement, bearing interest at **10.00% plus SOFR**; a hypothetical **1% increase in SOFR** would increase annual interest expense by approximately **$0.8 million**[230](index=230&type=chunk) [Controls and Procedures](index=72&type=section&id=Item%204.%20Controls%20and%20Procedures) Management assessed disclosure controls and procedures as effective on June 30, 2023, with no material changes to internal control over financial reporting during the quarter - Management, including the CEO and CFO, evaluated disclosure controls and procedures, concluding they were **effective** at a reasonable assurance level as of the end of the reporting period[233](index=233&type=chunk) - No changes in the company's internal control over financial reporting during the quarter ended June 30, 2023, have materially affected, or are reasonably likely to materially affect, internal controls[234](index=234&type=chunk) PART II. OTHER INFORMATION [Legal Proceedings](index=73&type=section&id=Item%201.%20Legal%20Proceedings) This section confirms that the company is not currently involved in any material legal proceedings - The company is not subject to any material legal proceedings[236](index=236&type=chunk) [Risk Factors](index=73&type=section&id=Item%201A.%20Risk%20Factors) This section details significant risks and uncertainties that could adversely affect the company's business, financial condition, and future prospects, categorized by key operational and financial areas - The company has a history of significant losses, with an accumulated deficit of approximately **$530.1 million** as of June 30, 2023, and anticipates continued losses for the foreseeable future[238](index=238&type=chunk) - The company will require additional capital to fund operations, and its ability to raise funds may be affected by external factors; failure to secure financing could force delays or reductions in research and development programs[243](index=243&type=chunk) - The development of novel gene therapies is difficult, costly, and time-consuming, with an uncertain regulatory landscape and a high risk of failure in clinical trials[259](index=259&type=chunk)[264](index=264&type=chunk)[271](index=271&type=chunk) - The company faces significant competition from major pharmaceutical and biotechnology companies, and its success depends on obtaining regulatory approval and achieving market acceptance and adequate reimbursement for its products[386](index=386&type=chunk)[391](index=391&type=chunk) [Risks Related to Financial Position and Need for Additional Capital](index=73&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) [Risks Related to Discovery, Development, Clinical Testing, Manufacturing and Regulatory Approval](index=83&type=section&id=Risks%20Related%20to%20Discovery%2C%20Development%2C%20Clinical%20Testing%2C%20Manufacturing%20and%20Regulatory%20Approval) [Risks Related to Healthcare Laws and Other Legal Compliance Matters](index=117&type=section&id=Risks%20Related%20to%20Healthcare%20Laws%20and%20Other%20Legal%20Compliance%20Matters) [Risks Related to Commercialization](index=129&type=section&id=Risks%20Related%20to%20Commercialization) [Risks Related to Our Dependence on Third Parties](index=138&type=section&id=Risks%20Related%20to%20Our%20Dependence%20on%20Third%20Parties) [Risks Related to Intellectual Property](index=144&type=section&id=Risks%20Related%20to%20Intellectual%20Property) [Risks Related to Employee Matters and Managing Growth](index=159&type=section&id=Risks%20Related%20to%20Employee%20Matters%20and%20Managing%20Growth) [Risks Related to Our Ordinary Shares](index=167&type=section&id=Risks%20Related%20to%20Our%20Ordinary%20Shares) [Unregistered Sales of Equity Securities and Use of Proceeds](index=182&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This section discloses the company's unregistered equity sales during the reporting period, including a May 2023 private placement and an April 2023 issuance from a prior acquisition - On May 5, 2023, the company completed a private placement, selling **10,773,913 ordinary shares** at **$5.75 per share** for gross proceeds of **$62 million**[530](index=530&type=chunk) - On April 4, 2023, the company issued **40,138 ordinary shares** to former stockholders of Bullseye Therapeutics, Inc. as part of the 2021 merger agreement[531](index=531&type=chunk) [Defaults Upon Senior Securities](index=182&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This section indicates that there were no defaults upon senior securities during the reporting period - None[532](index=532&type=chunk) [Mine Safety Disclosures](index=182&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This section is not applicable to the company's operations - Not applicable[534](index=534&type=chunk) [Other Information](index=182&type=section&id=Item%205.%20Other%20Information) This section indicates that there is no other information to report for the period - None[535](index=535&type=chunk) [Exhibits](index=183&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including agreements, CEO and CFO certifications, and interactive data files (XBRL) - Exhibits filed include the Securities Purchase Agreement from May 2023, the Registration Rights Agreement, CEO and CFO certifications (Sections 302 and 906), and XBRL data files[537](index=537&type=chunk)

[PART I. FINANCIAL INFORMATION](index=7&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) This section presents unaudited financial statements and management's discussion of financial condition and operations [Item 1. Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Unaudited condensed consolidated financial statements for Q1 2023, covering balance sheets, operations, equity, cash flows, and notes - Financial statements are unaudited and prepared in conformity with GAAP for interim financial information[32](index=32&type=chunk) [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Summarizes the company's financial position, detailing assets, liabilities, and equity for Q1 2023 and year-end 2022 Balance Sheet Highlights (in thousands) | Metric | March 31, 2023 | December 31, 2022 | Change | | :-------------------------------- | :------------- | :---------------- | :----- | | Cash and cash equivalents | $68,784 | $115,516 | $(46,732) | | Accounts receivable - related party | $36,298 | $21,334 | $14,964 | | Total Current Assets | $121,481 | $154,339 | $(32,858) | | Total Assets | $288,140 | $318,237 | $(30,097) | | Total Current Liabilities | $81,104 | $82,072 | $(968) | | Total Liabilities | $197,918 | $200,499 | $(2,581) | | Total Shareholders' Equity | $90,222 | $117,738 | $(27,516) | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Details financial performance, including revenues, expenses, and net loss for Q1 2023 and 2022 Statements of Operations Highlights (in thousands) | Metric | 3 Months Ended March 31, 2023 | 3 Months Ended March 31, 2022 | Change | | :------------------------------------ | :----------------------------- | :----------------------------- | :----- | | License revenue - related party | $3,334 | $5,633 | $(2,299) | | General and administrative expenses | $12,772 | $11,268 | $1,504 | | Research and development expenses | $22,322 | $23,099 | $(777) | | Total operating expenses | $35,094 | $34,367 | $727 | | Loss from operations | $(31,760) | $(28,734) | $(3,026) | | Foreign currency gain (loss) | $3,857 | $(2,647) | $6,504 | | Net loss | $(30,364) | $(31,045) | $681 | | Basic and diluted net loss per ordinary share | $(0.62) | $(0.70) | $0.08 | - Weighted-average number of ordinary shares outstanding increased to **48,638,151** in Q1 2023 from **44,501,314** in Q1 2022[24](index=24&type=chunk) [Condensed Consolidated Statement of Shareholders' Equity (March 31, 2023)](index=9&type=section&id=Condensed%20Consolidated%20Statement%20of%20Shareholders'%20Equity%20(March%2031,%202023)) Outlines changes in shareholders' equity for Q1 2023, including capital, accumulated loss, and comprehensive income Shareholders' Equity Changes (in thousands) | Metric | Balance at Dec 31, 2022 | Share-based compensation activity | Other comprehensive loss | Net loss | Balance at Mar 31, 2023 | | :-------------------------- | :---------------------- | :------------------------------ | :--------------------- | :------- | :---------------------- | | Capital in Excess of Par Value | $581,893 | $5,201 | — | — | $587,094 | | Accumulated Other Comprehensive (Loss) Income | $6,047 | — | $(2,353) | — | $3,694 | | Accumulated Deficit | $(470,204) | — | — | $(30,364) | $(500,568) | | Total Shareholders' Equity | $117,738 | $5,201 | $(2,353) | $(30,364) | $90,222 | [Condensed Consolidated Statement of Shareholders' Equity (March 31, 2022)](index=10&type=section&id=Condensed%20Consolidated%20Statement%20of%20Shareholders'%20Equity%20(March%2031,%202022)) Outlines changes in shareholders' equity for Q1 2022, including capital, accumulated loss, and comprehensive income Shareholders' Equity Changes (in thousands) | Metric | Balance at Dec 31, 2021 | Share-based compensation activity | Other comprehensive income | Net loss | Balance at Mar 31, 2022 | | :-------------------------- | :---------------------- | :------------------------------ | :----------------------- | :------- | :---------------------- | | Capital in Excess of Par Value | $528,659 | $4,996 | — | — | $533,655 | | Accumulated Other Comprehensive (Loss) Income | $(2,671) | — | $1,932 | — | $(739) | | Accumulated Deficit | $(340,589) | — | — | $(31,045) | $(371,634) | | Total Shareholders' Equity | $185,401 | $4,996 | $1,932 | $(31,045) | $161,284 | [Condensed Consolidated Statements of Cash Flows](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Presents cash flow activities, detailing cash used in operating, investing, and financing for Q1 2023 and 2022 Cash Flow Summary (in thousands) | Cash Flow Activity | 3 Months Ended March 31, 2023 | 3 Months Ended March 31, 2022 | Change | | :-------------------------------- | :----------------------------- | :----------------------------- | :----- | | Net cash used in operating activities | $(37,392) | $(8,298) | $(29,094) | | Net cash used in investing activities | $(8,605) | $(12,460) | $3,855 | | Net cash used in financing activities | $(1,231) | $(2,582) | $1,351 | | Net decrease in cash and cash equivalents | $(47,228) | $(23,340) | $(23,888) | | Cash and cash equivalents at end of period | $68,784 | $113,781 | $(44,997) | [Notes to Condensed Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Detailed explanatory notes to the financial statements, covering accounting policies, related-party transactions, and debt [Note 1. Organization and Basis of Presentation](index=12&type=section&id=Note%201.%20Organization%20and%20Basis%20of%20Presentation) Describes MeiraGTx's business, structure, and financial statement basis, including its liquidity outlook - MeiraGTx is a vertically integrated, clinical-stage gene therapy company with **six clinical programs** and a broad preclinical pipeline, focusing on ocular, neurodegenerative, and severe xerostomia diseases[30](index=30&type=chunk) - The company operates a cGMP multi-product, multi-viral vector manufacturing facility in London, UK, and acquired a second cGMP viral vector manufacturing facility and a cGMP plasmid and DNA production facility in Shannon, Ireland in **January 2021**[30](index=30&type=chunk) - The company had an accumulated deficit of **$500.6 million** at March 31, 2023, and used **$37.4 million** in cash from operations for the three months ended March 31, 2023[34](index=34&type=chunk)[35](index=35&type=chunk) - Cash and cash equivalents were **$68.8 million** as of March 31, 2023. The company estimates that its cash, related-party receivables (**$36.3 million** from Janssen), and proceeds from a May 2023 Private Placement (**$62.0 million** gross) will be sufficient to fund operations into **Q2 2025**[36](index=36&type=chunk)[144](index=144&type=chunk) [Note 2. Summary of Significant Accounting Policies and Recent Accounting Pronouncements](index=14&type=section&id=Note%202.%20Summary%20of%20Significant%20Accounting%20Policies%20and%20Recent%20Accounting%20Pronouncements) Outlines key accounting policies and impact of recent accounting pronouncements on financial statements - The condensed consolidated financial statements include the accounts of Meira Holdings and its wholly-owned subsidiaries, with all intercompany balances and transactions eliminated[40](index=40&type=chunk)[42](index=42&type=chunk)[43](index=43&type=chunk) - Management applies significant judgment in areas such as collaboration revenue, research and development costs, share-based compensation, leases, asset retirement obligations, and tax incentive receivable[44](index=44&type=chunk) - The company adopted **ASU 2016-13** (Financial Instruments – Credit Losses) effective **January 1, 2023**, which did not have an impact on its consolidated financial statements[92](index=92&type=chunk) - Collaboration revenue from the Janssen agreement is recognized over time using a cost-to-cost input method, with variable consideration subject to constraints[71](index=71&type=chunk)[72](index=72&type=chunk)[119](index=119&type=chunk) [Note 3. Equity Method and Other Investments](index=28&type=section&id=Note%203.%20Equity%20Method%20and%20Other%20Investments) Details the company's equity method and other investments, including ownership percentages and carrying values Equity Investments (in thousands) | Investee | Investment Type | Ownership Percentage | Carrying Value | Cost Basis | | :------- | :-------------------- | :------------------- | :------------- | :--------- | | Visiogene LLC | Equity Method Investment | 25% | $5,156 | $5,165 | | Other | Equity Investment | 1.6% | $1,170 | $1,500 | | **Total** | | | **$6,326** | **$6,665** | [Note 4. Accrued Expenses](index=28&type=section&id=Note%204.%20Accrued%20Expenses) Provides a breakdown of accrued expenses, including clinical trial, R&D, compensation, and manufacturing costs Accrued Expenses (in thousands) | Category | March 31, 2023 | December 31, 2022 | Change | | :----------------------- | :------------- | :---------------- | :----- | | Clinical trial costs | $12,241 | $13,041 | $(800) | | Research and development | $6,644 | $7,400 | $(756) | | Compensation and benefits | $4,901 | $9,600 | $(4,699) | | Manufacturing costs | $2,712 | $4,326 | $(1,614) | | Professional fees | $1,716 | $732 | $984 | | Consulting | $1,365 | $694 | $671 | | Fixed assets | $1,167 | $3,093 | $(1,926) | | Other | $321 | $932 | $(611) | | **Total** | **$31,067** | **$39,818** | **$(8,751)** | [Note 5. Share-Based Compensation](index=30&type=section&id=Note%205.%20Share-Based%20Compensation) Details share-based compensation plans, including expense recognition, outstanding options, and restricted share units Total Share-Based Compensation Expense (in thousands) | Category | 3 Months Ended March 31, 2023 | 3 Months Ended March 31, 2022 | Change | | :-------------------------- | :----------------------------- | :----------------------------- | :----- | | Research and development | $3,026 | $3,632 | $(606) | | General and administrative | $3,406 | $3,946 | $(540) | | **Total** | **$6,432** | **$7,578** | **$(1,146)** | - Share options outstanding increased from **6,858,409** at December 31, 2022, to **8,233,380** at March 31, 2023, with the weighted-average exercise price decreasing from **$14.03** to **$13.09**[97](index=97&type=chunk) - Restricted Share Units (RSUs) outstanding increased from **2,182,500** at December 31, 2022, to **2,738,750** at March 31, 2023[101](index=101&type=chunk) - Unrecognized compensation expense for unvested options was **$29.1 million** and for RSUs was **$31.8 million** as of March 31, 2023, both expected to be realized over **3.9 years**[100](index=100&type=chunk)[103](index=103&type=chunk) [Note 6. Income Taxes](index=33&type=section&id=Note%206.%20Income%20Taxes) Explains income tax position, including no tax provision due to losses and valuation allowance on deferred tax assets - The company did not record a provision for income taxes for the three-month periods ended March 31, 2023 and 2022, due to generated losses[107](index=107&type=chunk) - A full valuation allowance is maintained against deferred tax assets in the United States, United Kingdom, Ireland, and Netherlands, indicating uncertainty about their future realization[108](index=108&type=chunk) [Note 7. Related-Party Transactions](index=33&type=section&id=Note%207.%20Related-Party%20Transactions) Describes significant transactions with related parties, primarily the Janssen collaboration and debt financing - The company entered into a Collaboration Agreement with Janssen Pharmaceuticals, Inc. on **January 30, 2019**, for the research, development, and commercialization of gene therapies for inherited retinal diseases (IRDs)[109](index=109&type=chunk) - Under the Janssen Collaboration Agreement, the company received a **$100.0 million** upfront payment in **March 2019** and a **$30.0 million** milestone payment in **December 2021**[36](index=36&type=chunk)[121](index=121&type=chunk) - Janssen pays **100%** of clinical and commercialization costs for Clinical IRD Product Candidates, with MeiraGTx eligible for **20% royalties** on net sales and up to **$340.0 million** in additional development and commercialization milestones[115](index=115&type=chunk) - The company recognized **$3.3 million** in license revenue from deferred revenue in Q1 2023 (vs. **$5.6 million** in Q1 2022) and **$22.6 million** in reimbursement of R&D expenses in Q1 2023 (vs. **$13.2 million** in Q1 2022)[122](index=122&type=chunk) - The company entered into a senior secured financing arrangement (Note Purchase Agreement) with Perceptive Credit Holdings III, LP, an affiliate of a greater than **10% shareholder**, for an initial **$75.0 million**[125](index=125&type=chunk)[126](index=126&type=chunk)[127](index=127&type=chunk) [Note 8. Leases](index=39&type=section&id=Note%208.%20Leases) Details lease arrangements, including finance and operating lease costs, right-of-use assets, and liabilities Total Lease Cost (in thousands) | Category | 3 Months Ended March 31, 2023 | 3 Months Ended March 31, 2022 | Change | | :-------------------------- | :----------------------------- | :----------------------------- | :----- | | Finance lease cost | $281 | $294 | $(13) | | Operating lease cost | $1,361 | $1,315 | $46 | | Short-term lease cost | $39 | $38 | $1 | | **Total lease cost** | **$1,681** | **$1,647** | **$34** | Lease Balances (in thousands) | Metric | March 31, 2023 | December 31, 2022 | | :-------------------------------- | :------------- | :---------------- | | Operating lease Right-of-Use asset | $19,427 | $20,109 | | Finance lease Right-of-Use asset | $24,851 | $24,718 | | Operating lease liabilities | $20,471 | $21,215 | - Weighted-average remaining lease term for operating leases was **5.4 years** (**5.6 years** at Dec 31, 2022) and for finance leases was **175.6 years** (**175.8 years** at Dec 31, 2022)[131](index=131&type=chunk) [Note 9. Debt Financing](index=41&type=section&id=Note%209.%20Debt%20Financing) Outlines terms of the senior secured financing arrangement, including interest rates, covenants, and warrants - The company's Note Purchase Agreement provides for an initial **$75.0 million** (Tranche 1 Notes) and an option for an additional **$25.0 million** (Tranche 2) at Perceptive's discretion before **August 2, 2024**[136](index=136&type=chunk) - The agreement matures on **August 2, 2026**, is interest-only during the term, and bears interest at **10.00%** plus the one-month secured overnight financing rate (SOFR), subject to a **1.00% floor** (**14.34%** at March 31, 2023)[136](index=136&type=chunk) - The company's obligations are secured by its London and Shannon manufacturing facilities, **$3.0 million** of cash, and the equity interests of Subsidiary Guarantors[137](index=137&type=chunk) - Covenants include enrolling in a Phase III trial for AAV-RPGR by **June 30, 2023**, and ensuring the Shannon manufacturing facility meets cGMP requirements by **December 31, 2023**[138](index=138&type=chunk) - Warrants were granted to Perceptive to purchase **400,000** ordinary shares at **$15.00** and **300,000** at **$20.00**, exercisable immediately and expiring **August 2, 2027**[141](index=141&type=chunk) [Note 10. Commitments and Contingencies](index=43&type=section&id=Note%2010.%20Commitments%20and%20Contingencies) Addresses material commitments or contingencies, confirming no new significant items for the reporting period - There were no new material commitments or contingencies entered into during the three-month period ended March 31, 2023[143](index=143&type=chunk) [Note 11. Subsequent Event](index=43&type=section&id=Note%2011.%20Subsequent%20Event) Discloses significant events after the balance sheet date, specifically a private placement financing in May 2023 - On **May 3, 2023**, the company entered into a private placement agreement to issue **10,773,913** ordinary shares at a purchase price of **$5.75** per share, raising gross proceeds of **$62.0 million**, which closed on **May 5, 2023**[144](index=144&type=chunk) - In connection with the Private Placement, the company is obligated to file a registration statement to register for resale the shares on or prior to **August 3, 2023**[145](index=145&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=44&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 and operating results, highlighting its clinical-stage gene therapy focus, recent financial performance, and future outlook, including liquidity and capital needs - The discussion and analysis should be read in conjunction with the financial statements and related notes in this Quarterly Report on Form 10-Q and the Annual Report on Form 10-K for the year ended December 31, 2022[147](index=147&type=chunk) - The section includes forward-looking statements that involve risks and uncertainties, as detailed in the 'Risk Factors' section[147](index=147&type=chunk) [Overview](index=44&type=section&id=Overview) This note describes MeiraGTx's business, organizational structure, and the basis for preparing its financial statements, including liquidity outlook - MeiraGTx is a vertically integrated, clinical-stage gene therapy company with **six clinical programs** and a broad preclinical pipeline, focusing on ocular, neurodegenerative, and severe xerostomia diseases[148](index=148&type=chunk) - The company has incurred significant operating losses since inception, with a net loss of **$30.4 million** in Q1 2023 (vs. **$31.0 million** in Q1 2022) and an accumulated deficit of **$500.6 million** as of March 31, 2023[150](index=150&type=chunk) - Operations have been financed primarily through equity offerings (**$471.0 million** gross), debt financing (**$75.0 million**), and the Janssen Collaboration Agreement (**$130.0 million** upfront/milestone payments, plus research funding)[149](index=149&type=chunk) - Cash and cash equivalents were **$68.8 million** as of March 31, 2023, with **$36.3 million** in receivables from Janssen. A subsequent Private Placement in May 2023 raised **$62.0 million** gross proceeds[149](index=149&type=chunk)[154](index=154&type=chunk) - The company estimates that current funds (cash, receivables, and PIPE proceeds) will be sufficient to fund operating expenses and capital expenditure requirements into **Q2 2025**[156](index=156&type=chunk) [Recent Development Highlights and Anticipated Milestones](index=48&type=section&id=Recent%20Development%20Highlights%20and%20Anticipated%20Milestones) This section highlights recent clinical development progress and key anticipated milestones for the company's gene therapy programs - Dosing in the pivotal Phase 3 LUMEOS clinical trial of bota-vec for X-linked retinitis pigmentosa (XLRP) continues, with a Biologics License Application (BLA) submission on track for **2024**[164](index=164&type=chunk) - Positive clinical data from the AQUAx Phase 1 clinical trial for AAV-hAQP1 in radiation-induced xerostomia was reported in **December 2022**, with a randomized, double-blind, placebo-controlled Phase 2 study expected to initiate in **Q2 2023**[164](index=164&type=chunk) - Patients are being dosed in the AAV-GAD clinical trial for Parkinson's disease under a new IND, with enrollment anticipated to complete in **Q3 2023**[164](index=164&type=chunk) - The next-generation riboswitch-based gene regulation platform demonstrates precise, dose-responsive control of gene expression using novel, synthetic, orally delivered small molecules, with first GMP material for IND currently being manufactured[164](index=164&type=chunk) - The company closed a private investment in public equity (PIPE) financing in **May 2023**, raising **$62 million** in aggregate gross proceeds[163](index=163&type=chunk) [Components of Our Results of Operations](index=50&type=section&id=Components%20of%20Our%20Results%20of%20Operations) This section explains the key components contributing to the company's financial results, including revenue and expense categories - License revenue consists of the amortization of upfront and milestone payments received from the Collaboration Agreement[166](index=166&type=chunk) - Operating expenses primarily comprise general and administrative (G&A) costs and research and development (R&D) costs, with R&D expenses charged as incurred[167](index=167&type=chunk)[170](index=170&type=chunk) - G&A expenses are expected to increase due to personnel growth and public company costs, while R&D expenses are expected to increase substantially for clinical trials, partially offset by collaboration funding[169](index=169&type=chunk)[171](index=171&type=chunk) - Other non-operating income (expense) includes foreign currency gains/losses from remeasurement of foreign subsidiary cash and intercompany balances, and other comprehensive (loss) income includes foreign currency translation adjustments[177](index=177&type=chunk)[179](index=179&type=chunk) [Critical Accounting Policies and Use of Estimates](index=53&type=section&id=Critical%20Accounting%20Policies%20and%20Use%20of%20Estimates) This section discusses the significant accounting policies and the use of estimates that are crucial to the preparation of the financial statements - The preparation of condensed consolidated financial statements requires significant estimates and judgments, particularly in areas such as collaboration revenue, share-based compensation, and accrued expenses[180](index=180&type=chunk) - Estimates are based on historical experience, known trends, and various other factors, but actual results may differ materially from these estimates[180](index=180&type=chunk) [Results of Operations](index=53&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, 2023 and 2022 Financial Performance Comparison (in thousands) | Metric | 3 Months Ended March 31, 2023 | 3 Months Ended March 31, 2022 | Change | | :------------------------------------ | :----------------------------- | :----------------------------- | :----- | | License revenue - related party | $3,334 | $5,633 | $(2,299) | | General and administrative expenses | $12,772 | $11,268 | $1,504 | | Research and development expenses | $22,322 | $23,099 | $(777) | | Loss from operations | $(31,760) | $(28,734) | $(3,026) | | Foreign currency gain (loss) | $3,857 | $(2,647) | $6,504 | | Interest income | $545 | $16 | $529 | | Interest expense | $(3,060) | $(77) | $(2,983) | | Net loss | $(30,364) | $(31,045) | $681 | | Comprehensive loss | $(32,717) | $(29,113) | $(3,604) | - License revenue decreased by **$2.3 million** due to reduced amortization of upfront and milestone payments from the Janssen Collaboration Agreement[183](index=183&type=chunk) - General and administrative expenses increased by **$1.5 million**, primarily due to higher legal and accounting fees, partially offset by lower share-based compensation and insurance costs[184](index=184&type=chunk) - Research and development expenses decreased by **$0.8 million**, driven by a **$9.3 million** increase in research funding reimbursement from Janssen, partially offset by an **$8.4 million** increase in clinical trial expenses (especially bota-vec Phase 3 and AAV-hAQP1 trials) and **$1.6 million** in manufacturing expenses[188](index=188&type=chunk)[190](index=190&type=chunk) - A foreign currency gain of **$3.9 million** was recorded in Q1 2023 (vs. **$2.6 million** loss in Q1 2022) due to an unrealized gain on intercompany payables/receivables from the weakening of the U.S. dollar[191](index=191&type=chunk) - Interest expense increased by **$3.0 million**, primarily due to interest accrued and amortization of the debt discount in connection with the debt financing[193](index=193&type=chunk) [Liquidity and Capital Resources](index=57&type=section&id=Liquidity%20and%20Capital%20Resources) This section discusses the company's financial liquidity, capital needs, and how it plans to fund its operations and future growth - The company has incurred significant operating losses since inception and expects to continue incurring substantial expenses, necessitating additional capital from equity, debt, or collaborations[196](index=196&type=chunk)[198](index=198&type=chunk) - As of March 31, 2023, cash and cash equivalents totaled **$68.8 million**. With expected research funding and proceeds from the May 2023 Private Placement, funds are estimated to be sufficient into **Q2 2025**[200](index=200&type=chunk)[201](index=201&type=chunk) - Net cash used in operating activities increased significantly to **$37.4 million** in Q1 2023 (from **$8.3 million** in Q1 2022), primarily due to the net loss and an increase in operating assets[202](index=202&type=chunk)[203](index=203&type=chunk) - Net cash used in investing activities was **$8.6 million** in Q1 2023 (vs. **$12.5 million** in Q1 2022) for property and equipment purchases[204](index=204&type=chunk)[205](index=205&type=chunk) - Net cash used in financing activities was **$1.2 million** in Q1 2023 (vs. **$2.6 million** in Q1 2022), primarily for tax withholding obligations on restricted share unit awards[206](index=206&type=chunk)[207](index=207&type=chunk) - The company is an 'emerging growth company' and will remain so until **December 31, 2023**, benefiting from reduced disclosure requirements and an extended transition period for accounting standards[209](index=209&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=62&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company is exposed to foreign currency exchange risk due to international operations and interest rate risk from its variable-rate debt. A 10% unfavorable movement in foreign currency could result in a $25.4 million loss, and a 1% increase in SOFR could increase annual interest expense by $0.8 million - The company is exposed to foreign currency exchange rate fluctuations, primarily between the U.S. Dollar and the British pound sterling and euro, due to operations in the United States, United Kingdom, Netherlands, Ireland, and Belgium[212](index=212&type=chunk) - A **10%** unfavorable movement in foreign currency exchange rates is estimated to create an additional foreign currency loss of approximately **$25.4 million** for the three months ended March 31, 2023[212](index=212&type=chunk) - The company is exposed to market risk from changes in interest rates applicable to borrowings under its Note Purchase Agreement, which bears interest at a fluctuating rate (**14.34%** at March 31, 2023)[213](index=213&type=chunk) - A hypothetical **1%** increase in the secured overnight financing rate (SOFR) would increase annual interest expense by approximately **$0.8 million**, assuming no change in outstanding borrowings[213](index=213&type=chunk) [Item 4. Controls and Procedures](index=62&type=section&id=Item%204.%20Controls%20and%20Procedures) 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, 2023. No material changes in internal control over financial reporting occurred during the quarter - Management, with the participation of the Chief Executive Officer and Chief Financial Officer, evaluated the effectiveness of disclosure controls and procedures as of **March 31, 2023**[215](index=215&type=chunk) - It was concluded that the disclosure controls and procedures were effective at the reasonable assurance level[215](index=215&type=chunk) - No changes in internal control over financial reporting occurred during the quarter ended March 31, 2023, that have materially affected, or are reasonably likely to materially affect, internal control over financial reporting[216](index=216&type=chunk) [PART II. OTHER INFORMATION](index=65&type=section&id=PART%20II.%20OTHER%20INFORMATION) This section provides additional information, including legal proceedings, risk factors, equity sales, defaults, and exhibits [Item 1. Legal Proceedings](index=65&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[217](index=217&type=chunk) [Item 1A. Risk Factors](index=65&type=section&id=Item%201A.%20Risk%20Factors) Details risks associated with investing, covering financial, development, regulatory, manufacturing, and market factors - Investing in the company's ordinary shares involves a high degree of risk[218](index=218&type=chunk) - If any of the described risks occur, the company's business, financial condition, results of operations, and future growth prospects could be materially and adversely affected, potentially leading to a decline in the market price of ordinary shares[218](index=218&type=chunk) [Risks Related to Our Financial Position and Need for Additional Capital](index=65&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) Highlights financial risks, including significant losses, need for capital, and covenants from debt financing - The company has incurred significant losses since inception, with net losses of approximately **$30.4 million** and **$31.0 million** for the three months ended March 31, 2023 and 2022, respectively, and an accumulated deficit of approximately **$500.6 million** as of March 31, 2023[219](index=219&type=chunk) - The company anticipates continued losses for the foreseeable future and will require additional capital to fund its operations, which may not be available on acceptable terms, if at all[219](index=219&type=chunk)[224](index=224&type=chunk) - The company's ability to fund operations into **Q2 2025** is based on estimates that may prove wrong, and it could utilize available capital resources sooner than expected[225](index=225&type=chunk) - The Note Purchase Agreement imposes covenants (e.g., Phase III trial for AAV-RPGR by **June 30, 2023**, Shannon facility cGMP by **Dec 31, 2023**) and restrictions on operations; non-compliance could lead to debt acceleration and material adverse effects[230](index=230&type=chunk)[231](index=231&type=chunk)[233](index=233&type=chunk) - Future success is substantially dependent on the successful development, regulatory approval, and commercialization of its Most Advanced Product Candidates (bota-vec, AAV-GAD, AAV-CNGB3, AAV-CNGA3, AAV-RPE65, and AAV-hAQP1)[235](index=235&type=chunk) [Risks Related to Discovery, Development, Clinical Testing, Manufacturing and Regulatory Approval](index=75&type=section&id=Risks%20Related%20to%20Discovery,%20Development,%20Clinical%20Testing,%20Manufacturing%20and%20Regulatory%20Approval) Covers risks in gene therapy development, clinical trials, manufacturing, and the evolving regulatory landscape - The company's novel gene therapy platform faces challenges in predicting development time and cost, with few gene therapies approved to date, and risks related to mechanism of action, safety, efficacy, and potential toxicities[241](index=241&type=chunk)[242](index=242&type=chunk) - The regulatory landscape for gene therapy is uncertain and evolving, with potential for frequent changes in requirements, uncoordinated oversight, and longer, more expensive approval processes[246](index=246&type=chunk)[247](index=247&type=chunk)[248](index=248&type=chunk) - Clinical trials are expensive, time-consuming, difficult to design, and have uncertain outcomes, with potential for substantial delays due to patient enrollment, regulatory agreements, third-party performance, or unforeseen adverse events[252](index=252&type=chunk)[253](index=253&type=chunk)[255](index=255&type=chunk)[258](index=258&type=chunk)[261](index=261&type=chunk) - The COVID-19 pandemic has caused and may continue to cause delays in clinical trials, supply chain disruptions, and adverse impacts on global economic conditions and capital raising[269](index=269&type=chunk)[272](index=272&type=chunk) - The company has received Fast Track, PRIME, and Orphan Drug designations for several product candidates, but these do not guarantee faster development, review, or approval, nor do they increase the likelihood of marketing approval or confer absolute market exclusivity[278](index=278&type=chunk)[279](index=279&type=chunk)[290](index=290&type=chunk)[283](index=283&type=chunk)[291](index=291&type=chunk) - Manufacturing processes and facilities (internal and third-party) are subject to significant cGMP regulation, with risks of non-compliance, contamination, raw material shortages, and difficulties in expanding capacity, which could delay development and commercialization[292](index=292&type=chunk)[293](index=293&type=chunk)[297](index=297&type=chunk)[298](index=298&type=chunk) - Product candidates may cause serious adverse events or undesirable side effects, which could interrupt, delay, or halt clinical trials, result in restrictive labels, or lead to denial of regulatory approval[303](index=303&type=chunk)[304](index=304&type=chunk) [Risks Related to Healthcare Laws and Other Legal Compliance Matters](index=109&type=section&id=Risks%20Related%20to%20Healthcare%20Laws%20and%20Other%20Legal%20Compliance%20Matters) Addresses risks from healthcare legislation, fraud and abuse laws, data protection, and international compliance - Enacted and future healthcare legislation (e.g., ACA, Medicare payment reductions, state pricing controls) may increase the difficulty and cost of obtaining marketing approval and affect product pricing[333](index=333&type=chunk)[337](index=337&type=chunk)[338](index=338&type=chunk) - The company's business operations are subject to broadly applicable fraud and abuse laws (e.g., U.S. federal Anti-Kickback Statute, False Claims Act, HIPAA) and other healthcare laws in the U.S., UK, and EU, with potential for significant penalties for non-compliance[345](index=345&type=chunk)[348](index=348&type=chunk) - The company is subject to evolving global data protection laws (e.g., HIPAA, CCPA/CPRA, GDPR, UK data protection regime); compliance is complex and costly, and failure could result in significant penalties and reputational harm[350](index=350&type=chunk)[352](index=352&type=chunk)[353](index=353&type=chunk)[357](index=357&type=chunk)[358](index=358&type=chunk) - Operations are subject to environmental, health, and safety laws regarding hazardous and biological materials, with risks of fines, sanctions, or production delays if regulations are not met or materials are restricted[359](index=359&type=chunk) - International operations expose the company to anti-corruption laws (e.g., UK Bribery Act, FCPA) and Trade Control laws, with potential for civil or criminal penalties, remedial measures, and legal expenses for non-compliance[361](index=361&type=chunk)[363](index=363&type=chunk)[364](index=364&type=chunk) [Risks Related to Commercialization](index=121&type=section&id=Risks%20Related%20to%20Commercialization) Details challenges in commercializing gene therapies, including competition, reimbursement, market acceptance, and third-party reliance - The development and commercialization of new gene therapy products is highly competitive, with major pharmaceutical and biotechnology companies possessing greater resources and expertise[366](index=366&type=chunk)[369](index=369&type=chunk) - Competitors may achieve regulatory approval sooner or develop safer, more effective, or less expensive therapies, which could harm the company's financial condition and ability to commercialize its product candidates[371](index=371&type=chunk) - Successful commercialization depends on obtaining adequate coverage and reimbursement levels from governmental authorities and health insurers; failure to do so could limit market access and revenue generation[372](index=372&type=chunk)[373](index=373&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[379](index=379&type=chunk) - The company lacks its own sales, marketing, and distribution infrastructure and relies on collaborators; failure to establish or maintain effective collaborative arrangements could delay commercialization[383](index=383&type=chunk)[384](index=384&type=chunk)[385](index=385&type=chunk)[387](index=387&type=chunk) - Commercialization outside the United States, UK, or EU involves additional risks, including differing regulatory requirements, intellectual property protection, reimbursement policies, and economic/political instability[390](index=390&type=chunk) - Product candidates approved as biologics may face competition from biosimilars sooner than anticipated, potentially reducing market exclusivity and revenue[392](index=392&type=chunk)[394](index=394&type=chunk) [Risks Related to Our Dependence on Third Parties](index=130&type=section&id=Risks%20Related%20to%20Our%20Dependence%20on%20Third%20Parties) Examines risks from reliance on third-party manufacturers, suppliers, collaborators, and clinical research organizations - The company relies on its own cGMP manufacturing facilities and third-party suppliers for plasmid and other components; issues with these facilities or suppliers could lead to insufficient quantities, delays, or increased costs[395](index=395&type=chunk)[396](index=396&type=chunk) - Reliance on third-party manufacturers entails risks such as breach of agreement, termination, regulatory non-compliance, quality issues, and supply chain disruptions (e.g., geopolitical actions, health emergencies)[397](index=397&type=chunk)[399](index=399&type=chunk) - The company collaborates with third parties for development, manufacture, and commercialization of product candidates; failure to establish or maintain these relationships could significantly limit its ability to succeed[402](index=402&type=chunk)[404](index=404&type=chunk)[407](index=407&type=chunk) - Reliance on CROs, clinical trial sites, and other vendors for preclinical studies and clinical trials means limited direct control over their performance, and unsatisfactory performance could harm business and delay development[408](index=408&type=chunk)[412](index=412&type=chunk)[413](index=413&type=chunk) [Risks Related to Intellectual Property](index=136&type=section&id=Risks%20Related%20to%20Intellectual%20Property) Covers risks related to patent protection, licensing, infringement claims, and the protection of trade secrets - The company depends on proprietary technology licensed from others; loss of existing licenses or inability to acquire additional proprietary rights could prevent continued product candidate development[414](index=414&type=chunk) - Failure to obtain and maintain sufficiently broad patent protection for technology and product candidates could impair competitive effectiveness, as the patent prosecution process is expensive, time-consuming, and uncertain[418](index=418&type=chunk)[419](index=419&type=chunk) - Third parties may assert claims of patent infringement, or the company may need to defend or enforce its patents, leading to substantial costs, delays, or loss of productivity[425](index=425&type=chunk)[426](index=426&type=chunk)[428](index=428&type=chunk)[430](index=430&type=chunk) - Changes in patent laws or jurisprudence (e.g., Leahy-Smith America Invents Act, U.S. Supreme Court rulings) could diminish the value of patents, impairing the ability to protect product candidates[435](index=435&type=chunk)[438](index=438&type=chunk) - Limited geographical patent protection and inadequate patent terms (due to lengthy development and regulatory review) may expose the company to competition from generic versions[439](index=439&type=chunk)[443](index=443&type=chunk)[444](index=444&type=chunk) - Reliance on trade secrets and confidential know-how carries risks of misappropriation or unauthorized disclosure, especially when shared with third parties, which could impair competitive position[447](index=447&type=chunk)[449](index=449&type=chunk)[450](index=450&type=chunk) [Risks Related to Employee Matters and Managing Growth](index=152&type=section&id=Risks%20Related%20to%20Employee%20Matters%20and%20Managing%20Growth) Addresses risks from organizational growth, key personnel, product liability, cybersecurity, and Brexit impacts - The company expects to expand its organization, which may lead to difficulties in managing growth, identifying, hiring, and integrating new personnel, potentially disrupting operations[454](index=454&type=chunk) - Future success depends on retaining key personnel (e.g., CEO, COO, CDO) and attracting qualified staff; loss of key individuals or inability to hire could seriously harm business strategy[455](index=455&type=chunk)[456](index=456&type=chunk) - The use and sale of product candidates expose the company to product liability claims, which could result in substantial liabilities, costs, and limits on commercialization[458](index=458&type=chunk) - Insurance policies are expensive and may not cover all business risks or be sufficient, leaving the company exposed to significant uninsured liabilities[459](index=459&type=chunk) - Misconduct by employees or independent contractors (e.g., non-compliance with regulations, fraudulent data) could lead to regulatory sanctions, reputational harm, and financial penalties[463](index=463&type=chunk) - The company's information technology systems are vulnerable to cybersecurity risks, including attacks, breaches, and data loss, which could disrupt operations, damage reputation, and delay product development[464](index=464&type=chunk)[468](index=468&type=chunk) - The UK's withdrawal from the EU (Brexit) has resulted in regulatory changes and economic uncertainties, potentially impacting the company's business, supply chains, and ability to attract/retain employees[469](index=469&type=chunk)[470](index=470&type=chunk)[474](index=474&type=chunk) [Risks Related to Our Ordinary Shares](index=160&type=section&id=Risks%20Related%20to%20Our%20Ordinary%20Shares) Details risks concerning share price volatility, shareholder influence, reduced disclosure, anti-takeover, and tax implications - The market price of the company's ordinary shares is likely to be volatile due to various factors, including clinical trial results, competitive products, regulatory developments, and general market conditions[476](index=476&type=chunk)[479](index=479&type=chunk) - Executive officers, directors, and principal shareholders (holding approximately **49.8%** of outstanding shares as of **May 8, 2023**) have the ability to significantly influence all matters submitted to shareholders for approval[480](index=480&type=chunk)[481](index=481&type=chunk) - As an 'emerging growth company' and 'smaller reporting company,' the company benefits from reduced disclosure requirements, which may make its ordinary shares less attractive to some investors and increase price volatility[484](index=484&type=chunk)[486](index=486&type=chunk)[487](index=487&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[490](index=490&type=chunk) - There may be difficulties in enforcing foreign judgments against the company's management or the company itself, as a significant portion of assets and personnel are located outside the United States[491](index=491&type=chunk)[492](index=492&type=chunk) - The rights of the company's shareholders differ from those typically offered to shareholders of a U.S. corporation, potentially making it more difficult to protect their interests[493](index=493&type=chunk)[494](index=494&type=chunk) - The company may be classified as a Passive Foreign Investment Company (PFIC) for U.S. federal income tax purposes, or U.S. persons owning at least **10%** of shares may be subject to Controlled Foreign Corporation (CFC) rules, leading to adverse U.S. federal income tax consequences[498](index=498&type=chunk)[499](index=499&type=chunk) - Changes in tax laws or challenges to the company's tax position could adversely affect results; significant U.S. federal/state Net Operating Losses (NOLs) and UK carryforward tax losses may be restricted or unrealized[500](index=500&type=chunk)[502](index=502&type=chunk)[503](index=503&type=chunk) [General Risk Factors](index=170&type=section&id=General%20Risk%20Factors) Covers general business risks including acquisitions, exchange rates, management discretion, public company costs, and ESG factors - The company may engage in acquisitions that could disrupt its business, cause dilution to shareholders, or reduce financial resources, and such acquisitions may not be on favorable terms or strengthen its competitive position[504](index=504&type=chunk) - Exchange rate fluctuations, particularly between the U.S. dollar, pound sterling, and euro, may adversely affect the company's results of operations and financial condition, and it currently has no hedging arrangements in place[505](index=505&type=chunk)[506](index=506&type=chunk) - Management has broad discretion over the use of net proceeds from financings, and these investments may not yield a favorable return or align with shareholder preferences[507](index=507&type=chunk) - Operating as a public company incurs substantial costs and requires significant management time for compliance initiatives and corporate governance practices, especially if the company no longer qualifies as an emerging growth company or smaller reporting company[508](index=508&type=chunk)[509](index=509&type=chunk) - If securities or industry analysts cease to publish research or issue adverse opinions about the company, its share price and trading volume could decline[510](index=510&type=chunk)[512](index=512&type=chunk) - Expectations relating to environmental, social, and governance (ESG) factors may impose additional costs, expose the company to new risks, and affect investor attractiveness if policies and disclosures are deemed inadequate[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 and Use of Proceeds](index=175&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) There were no unregistered sales of equity securities or use of proceeds to report for the period - None[515](index=515&type=chunk) [Item 3. Defaults Upon Senior Securities](index=175&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) There were no defaults upon senior securities to report for the period - None[516](index=516&type=chunk) [Item 4. Mine Safety Disclosures](index=175&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - Not applicable[517](index=517&type=chunk) [Item 5. Other Information](index=175&type=section&id=Item%205.%20Other%20Information) There is no other information to report for the period - None[518](index=518&type=chunk) [Item 6. Exhibits](index=176&type=section&id=Item%206.%20Exhibits) Lists exhibits filed with the Form 10-Q, including certifications from executive officers and XBRL documents - Exhibits include certifications from the Principal Executive Officer (**31.1, 32.1**) and Principal Financial Officer (**31.2, 32.2**), and various Inline XBRL Instance Documents (**101.INS, 101.SCH, 101.CAL, 101.DEF, 101.LAB, 101.PRE, 104**)[520](index=520&type=chunk) [SIGNATURES](index=177&type=section&id=SIGNATURES) Official signatures of the company's principal executive and financial officers, certifying the report - The report was duly signed on behalf of MeiraGTx Holdings plc by Alexandria Forbes (Chief Executive Officer) and Richard Giroux (Chief Financial Officer and Chief Operating Officer) on **May 11, 2023**[524](index=524&type=chunk)

Financial Performance - The company has incurred significant losses since inception and anticipates continued losses for the foreseeable future, with no assurance of achieving profitability[16]. - Net loss for 2022 was $129,615,000, compared to a net loss of $79,561,000 in 2021, indicating an increase in losses of about 62.9%[668]. - The Company's pre-tax loss for the year ended December 31, 2022, was $129,616,000, compared to a loss of $79,561,000 in 2021, representing a 62.8% increase[788]. - For the year ended December 31, 2022, the Company used $73.1 million in cash flows from operations, with no assurance of generating positive cash flows in the future[674]. - The Company has an accumulated deficit of $470.2 million as of December 31, 2022, and expects to incur substantial losses in future periods[674]. Revenue and Expenses - License revenue from related parties fell significantly from $37,701,000 in 2021 to $15,920,000 in 2022, a decrease of about 57.8%[668]. - Total operating expenses increased from $110,459,000 in 2021 to $132,275,000 in 2022, representing a rise of approximately 19.7%[668]. - Research and development expenses increased from $66,694,000 in 2021 to $85,725,000 in 2022, a rise of about 28.5%[668]. - The Company recognized collaboration revenue of $100.0 million from Janssen as a non-refundable upfront fee during the year ended December 31, 2019, and a milestone payment of $30.0 million in 2021[806]. - For the years ended December 31, 2022 and 2021, the Company recognized $15.9 million and $37.7 million, respectively, as license revenue from deferred revenue related to a related party[807]. Assets and Liabilities - Total assets decreased from $320,164,000 in 2021 to $318,237,000 in 2022, a decline of approximately 0.6%[666]. - Cash and cash equivalents decreased from $137,703,000 at the end of 2021 to $115,516,000 at the end of 2022, a reduction of approximately 16.1%[670]. - Total current liabilities rose from $68,128,000 in 2021 to $82,072,000 in 2022, an increase of about 20.5%[666]. - Shareholders' equity decreased from $185,401,000 in 2021 to $117,738,000 in 2022, a decline of approximately 36.5%[666]. - The company's long-lived assets totaled $163.9 million as of December 31, 2022, compared to $136.9 million in 2021, reflecting a growth of approximately 19.7%[754]. Capital and Financing - The annual interest rate on borrowings under the Note Purchase Agreement was 13.02% as of December 31, 2022, with an outstanding balance of $75.0 million[657]. - The outstanding balance of the Tranche 1 Notes was $75.0 million plus accrued interest of $4.0 million as of December 31, 2022, with an annual interest rate of 13.02%[828]. - The Note Purchase Agreement provides for an initial notes issuance of $75.0 million, with an option for an additional $25.0 million before August 2, 2024[828]. - The Company may seek additional capital through equity offerings, debt financings, and collaborations to support product development and commercialization[680]. - The Company raised approximately $25.0 million through a private placement of 3,742,514 ordinary shares at $6.68 per share on November 9, 2022[783]. Regulatory and Compliance Risks - The company is heavily dependent on the success of its Most Advanced Product Candidates, which are still in development, and failure to receive regulatory approval could harm the business[16]. - Compliance with privacy and data protection regulations is complex and costly, and failure to comply could materially harm the business[16]. - The Company operates in a highly competitive industry with significant risks, including uncertainty in product development and capital availability[678]. - The impact of the COVID-19 pandemic on the Company's financial position and operations remains uncertain, although it did not materially affect results in 2022[679]. Research and Development - The company has six programs in clinical development and a broad pipeline of preclinical and research programs, focusing on ocular diseases, neurodegenerative diseases, and severe forms of xerostomia[671]. - Research and development costs are charged to expense as incurred, including employee-related expenses and costs associated with clinical studies[740]. - The Company recorded reductions to research and development expenses of $6.8 million and $5.4 million for the years ended December 31, 2022, and 2021, respectively, related to tax incentive programs[689]. Tax and Deferred Tax - As of December 31, 2022, the Company had U.S. federal NOLs of $63,829,000 and UK carryforward tax losses of $187,939,000[11]. - The Company recorded deferred tax assets of $125,365,000 as of December 31, 2022, an increase from $100,340,000 in 2021[791]. - The Company has determined that it is more likely than not that its deferred tax assets will not be fully realized, resulting in a full valuation allowance[791]. - The UK corporation tax rate will increase from 19% to 25% effective April 1, 2023, impacting future accounting periods[792]. Share-Based Compensation - Share-based compensation expense for options was $16.1 million in 2022, compared to $14.8 million in 2021, representing an 8.8% increase[776]. - The total share-based compensation expense for RSUs was $12.5 million in 2022, significantly higher than $6.0 million in 2021, marking a 108.3% increase[780]. - The total compensation expense related to unvested options as of December 31, 2022, was $24.7 million, expected to be recognized over 4.0 years[778]. - The total compensation expense related to unvested RSUs as of December 31, 2022, was $26.9 million, expected to be recognized over 3.0 years[781].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION MeiraGTx Holdings plc WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-38520 (Exact Name of Registrant as Specified in its Charter) Cayman Islands 98-1448305 (State or othe ...

CONFIDENTIAL Corporate Presentation October 2022 Forward Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this presentation that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding our product development and anticipated milestones regarding our pre-clinical and clinical data and reporting ...