- Positive data from the Phase 1 AQUAx study in radiation-induced xerostomia (RIX) presented at the American Academy of Oral Medicine 2024 annual meeting (AAOM) showed meaningful improvements in patient-reported outcomes and saliva production with AAV2-hAQP1 treatment - Company awarded Innovation Passport Designation by the U.K. Innovative Licensing and Access Pathway Steering Group for AAV8-RK-AIPL1 for the treatment of AIPL1-Leber congenital amaurosis 4 (LCA4) LONDON and NEW YORK, Aug. 12, 2024 (GLOBE NEW ...

Core Viewpoint - MeiraGTx Holdings plc has announced an underwritten offering of 12,500,000 ordinary shares at a price of $4.00 per share, aiming to raise gross proceeds of $50 million before expenses [1][2]. Group 1: Offering Details - The offering is led by Sanofi, which invested $30 million in MeiraGTx through this offering [2]. - Other participants include Perceptive Advisors and various institutional healthcare funds [2]. - BofA Securities is acting as the sole underwriter for the offering, which is expected to close around August 13, 2024, pending customary closing conditions [2]. Group 2: Regulatory Information - The ordinary shares are being offered under an effective shelf registration statement on Form S-3, filed with the SEC on December 21, 2023, and declared effective on December 29, 2023 [3]. - The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC [3].

[PART I. FINANCIAL INFORMATION](index=7&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) The company presents its unaudited condensed consolidated financial statements for the period ending March 31, 2024 [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheet (in thousands) | Account | March 31, 2024 | December 31, 2023 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $119,206 | $129,566 | | Total Current Assets | $149,300 | $159,622 | | TOTAL ASSETS | $309,235 | $326,744 | | **Liabilities & Shareholders' Equity** | | | | Total Current Liabilities | $47,543 | $67,078 | | Deferred revenue - related party | $53,331 | $34,017 | | Note payable, net | $72,391 | $72,119 | | TOTAL LIABILITIES | $187,501 | $188,567 | | Total Shareholders' Equity | $121,734 | $138,177 | | TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY | $309,235 | $326,744 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Statement of Operations (in thousands, except per share data) | Account | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Total revenue | $697 | $3,334 | | General and administrative | $13,147 | $12,772 | | Research and development | $34,322 | $22,322 | | Total operating expenses | $47,469 | $35,094 | | Loss from operations | $(46,772) | $(31,760) | | Gain on sale of nonfinancial assets | $29,018 | $— | | Net loss | $(20,442) | $(30,364) | | Basic and diluted net loss per ordinary share | $(0.32) | $(0.62) | [Condensed Consolidated Statements of Cash Flows](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Statement of Cash Flows (in thousands) | Activity | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Net cash used in operating activities | $(36,741) | $(37,392) | | Net cash provided by (used in) investing activities | $27,340 | $(8,605) | | Net cash used in financing activities | $(1,270) | $(1,231) | | Net decrease in cash, cash equivalents and restricted cash | $(10,671) | $(47,228) | - Cash provided by investing activities in Q1 2024 was primarily driven by **$29.0 million in proceeds from the sale of nonfinancial assets** to Janssen, a significant change from the **$8.6 million used in Q1 2023** for property and equipment purchases[29](index=29&type=chunk)[233](index=233&type=chunk) [Notes to Condensed Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Key disclosures cover the Janssen asset sale, liquidity position, share-based compensation, and debt financing agreements - On December 20, 2023, the Company entered into an Asset Purchase Agreement with Janssen, selling assets related to its XLRP-RPGR product candidate (bota-vec) for **$65.0 million upfront** and up to **$350.0 million in future contingent payments**[32](index=32&type=chunk)[33](index=33&type=chunk) - A **$50.0 million milestone payment** from Janssen was achieved during the first quarter of 2024 related to the initiation of the extension study for the Phase 3 LUMEOS clinical trial[34](index=34&type=chunk)[130](index=130&type=chunk) - As of March 31, 2024, the Company had **$120.3 million in cash, cash equivalents, and restricted cash**, which management estimates is sufficient to cover expenses for at least the next twelve months[39](index=39&type=chunk) - The Company has a senior secured financing agreement with Perceptive for an initial **$75.0 million**, with an option for an additional **$25.0 million**, at an annual interest rate of **15.36%** as of March 31, 2024[153](index=153&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=52&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses financial performance, clinical developments, the Janssen asset sale, and the company's liquidity outlook [Recent Development Highlights and Anticipated Milestones](index=56&type=section&id=Recent%20Development%20Highlights%20and%20Anticipated%20Milestones) - **AAV-hAQP1 (Xerostomia):** The Phase 1 AQUAx study showed **statistically significant improvements** in patient-reported outcomes, and the pivotal Phase 2 study is ongoing with FDA alignment to support a potential BLA filing[175](index=175&type=chunk)[181](index=181&type=chunk) - **AAV-GAD (Parkinson's Disease):** Dosing is complete in the Phase 1 bridging study, with plans to initiate **Phase 3 study design discussions** with regulators in the second half of 2024[182](index=182&type=chunk) - **Riboswitch Platform:** The company is advancing its gene regulation platform for obesity and CAR-T therapies, with data to be presented at an **R&D Day in the second half of 2024**[182](index=182&type=chunk) - **Bota-vec (XLRP):** Received a **$50 million milestone payment** from Janssen in Q1 2024, with an additional **$15 million in near-term milestones** anticipated in 2024[180](index=180&type=chunk)[183](index=183&type=chunk)[189](index=189&type=chunk) [Results of Operations](index=66&type=section&id=Results%20of%20Operations) Comparison of Three Months Ended March 31, 2024 and 2023 (in thousands) | Account | 2024 | 2023 | Change | | :--- | :--- | :--- | :--- | | Total revenue | $697 | $3,334 | $(2,637) | | General and administrative | $13,147 | $12,772 | $375 | | Research and development | $34,322 | $22,322 | $12,000 | | Loss from operations | $(46,772) | $(31,760) | $(15,012) | | Gain on sale of nonfinancial assets | $29,018 | $— | $29,018 | | Net loss | $(20,442) | $(30,364) | $9,922 | - License revenue **decreased by $3.3 million** due to the termination of the Janssen Collaboration Agreement in December 2023[211](index=211&type=chunk) - Research and development expenses **increased by $12.0 million**, primarily because Janssen reimbursements decreased by **$21.9 million** following the collaboration's termination[217](index=217&type=chunk)[219](index=219&type=chunk) - A **gain on sale of nonfinancial assets of $29.0 million** was recognized in Q1 2024 from the allocation of the **$50.0 million** Janssen milestone payment[223](index=223&type=chunk) [Liquidity and Capital Resources](index=70&type=section&id=Liquidity%20and%20Capital%20Resources) - As of March 31, 2024, the company had **$120.3 million in cash, cash equivalents, and restricted cash**[228](index=228&type=chunk) - Management estimates current cash and expected milestones will fund operations and capital expenditures **into the first quarter of 2026**[227](index=227&type=chunk) - Net cash used in operating activities was **$36.7 million** for the three months ended March 31, 2024, compared to **$37.4 million** for the same period in 2023[228](index=228&type=chunk)[229](index=229&type=chunk)[230](index=230&type=chunk) - The company raised gross proceeds of **$1.6 million** in Q1 2024 through its **$100.0 million "at-the-market" (ATM) equity offering program**[122](index=122&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=74&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company details its primary market risk exposures from foreign currency fluctuations and variable-rate debt - **Foreign Currency Risk:** A hypothetical **10% unfavorable movement** in foreign currency exchange rates would have resulted in an additional loss of approximately **$10.0 million** for Q1 2024[239](index=239&type=chunk) - **Interest Rate Risk:** A hypothetical **1% increase in SOFR** would increase annual interest expense by approximately **$0.8 million** due to its variable-rate debt[240](index=240&type=chunk) [Item 4. Controls and Procedures](index=76&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective with no material changes in internal controls - The CEO and CFO concluded that the company's disclosure controls and procedures were **effective** as of March 31, 2024[242](index=242&type=chunk) - There were **no changes in internal control over financial reporting** during the quarter that have materially affected, or are reasonably likely to materially affect, internal controls[243](index=243&type=chunk) [PART II. OTHER INFORMATION](index=77&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=77&type=section&id=Item%201.%20Legal%20Proceedings) The company reports that it is not subject to any material legal proceedings - The company is **not subject to any material legal proceedings**[245](index=245&type=chunk) [Item 1A. Risk Factors](index=77&type=section&id=Item%201A.%20Risk%20Factors) The company outlines significant risks related to its financial position, development, commercialization, and intellectual property [Risks Related to Financial Position and Need for Additional Capital](index=77&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) - The company has a history of significant losses, with an **accumulated deficit of $574.7 million** as of March 31, 2024, and expects continued losses[247](index=247&type=chunk) - The company **will require additional capital** to fund operations, and failure to raise it could force delays or reductions in R&D programs[258](index=258&type=chunk) - There is **no guarantee of receiving future milestone payments** from the Janssen Asset Purchase Agreement, which could adversely affect the company's financial position[253](index=253&type=chunk) [Risks Related to Discovery, Development, Clinical Testing, Manufacturing and Regulatory Approval](index=89&type=section&id=Risks%20Related%20to%20Discovery%2C%20Development%2C%20Clinical%20Testing%2C%20Manufacturing%20and%20Regulatory%20Approval) - Gene therapy is a novel field with an **uncertain and evolving regulatory landscape**, making it difficult to predict the time and cost of obtaining approval[280](index=280&type=chunk) - Clinical trials are **expensive, time-consuming, and have uncertain outcomes**, with potential for substantial delays[286](index=286&type=chunk)[289](index=289&type=chunk) - The company is subject to **significant manufacturing regulations (GMP)**, and any failure to meet requirements could delay development and commercialization[323](index=323&type=chunk)[329](index=329&type=chunk) [Risks Related to Commercialization](index=136&type=section&id=Risks%20Related%20to%20Commercialization) - The company faces **significant competition** from large pharmaceutical and biotechnology companies[401](index=401&type=chunk)[403](index=403&type=chunk) - Successful commercialization depends on obtaining **adequate coverage and reimbursement** from government and private insurers, which is uncertain[404](index=404&type=chunk) - The company **lacks sales, marketing, and distribution infrastructure** and may not be successful in establishing these capabilities[416](index=416&type=chunk) [Risks Related to Intellectual Property](index=152&type=section&id=Risks%20Related%20to%20Intellectual%20Property) - The company **depends on technology licensed from others** and could lose its rights if it fails to comply with license obligations[450](index=450&type=chunk) - The ability to compete depends on **obtaining and maintaining patent protection**, but the patent process is uncertain and may be challenged[452](index=452&type=chunk) - Third parties may assert **patent infringement claims**, which could result in substantial costs and block commercialization[460](index=460&type=chunk) [Item 5. Other Information](index=189&type=section&id=Item%205.%20Other%20Information) The company discloses the adoption of a Rule 10b5-1 trading plan by its President and CEO - On March 21, 2024, President and CEO Alexandria Forbes, Ph.D., adopted a **Rule 10b5-1 trading plan** for the sale of up to **190,000 ordinary shares**[552](index=552&type=chunk) [Item 6. Exhibits](index=190&type=section&id=Item%206.%20Exhibits) The section lists all exhibits filed with the report, including officer certifications and XBRL data

Exhibit 99.1 MeiraGTx Reports First Quarter 2024 Financial and Operational Results - Positive data from the Phase 1 AQUAx study in radiation-induced xerostomia (RIX) presented in an oral session at the American Academy of Oral Medicine 2024 annual meeting (AAOM) April 17-20, 2024 - Received $50 million milestone following initiation of the extension study for the Phase 3 LUMEOS clinical trial for botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLR ...

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 ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION 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, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-38520 MeiraGTx Holdings plc (Exact Name of Registrant as Specified in its Charter) Cayman Islands 98-1448 ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION 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 June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-38520 MeiraGTx Holdings plc (Exact Name of Registrant as Specified in its Charter) Cayman Islands 98-1448305 ( ...