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, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to 450 East 29 th Street, 14 th Floor New York, NY 10016 (Address of principal executive offices) (Zip Code) Commission File Number: 001-3852 ...

Exhibit 99.1 MeiraGTx Reports Second Quarter 2025 Financial and Operational Results - Gained alignment with U.S. Food and Drug Administration (FDA) on the ongoing Phase 2 AQUAx2 randomized double-blind, placebo-controlled pivotal study in Grade 2/3 radiation-induced xerostomia (RIX) to support a potential Biologics License Application (BLA) filing; on track for potential data readout late 2026 - FDA Granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD for the treatment of Parkinson' ...

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

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

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

Multiple Poster Presentations Highlight the Depth and Novelty of MeiraGTx’s Technology Platforms for Gene and Cell TherapyLONDON and NEW YORK, May 13, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced the Company will exhibit four poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting, which is being held from May 13-17, 2025, in New Orleans, LA. The posters will be avai ...

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

Table of Contents ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 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 UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) (Exact Name of Registrant as Specified in its Charter) Cayman Islands 98-1448305 ...

Exhibit 99.1 MeiraGTx Reports First Quarter 2025 Financial and Operational Results - Announced strategic collaboration with Hologen AI, including a $200 million cash upfront payment to MeiraGTx and the formation of a joint venture, Hologen Neuro AI Ltd, with a further $230 million in capital committed to initially focus on expediting Phase 3 clinical development of AAV-GAD for Parkinson's disease - U.S. Food and Drug Administration (FDA) Granted Regenerative Medicine Advanced Therapy (RMAT) designation for ...

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