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]

An updated edition of the May 21, 2025, article.The promising field of genomics has undergone rapid evolution over the past decade. Genomics includes the study of a complete set of genes, their work process and way of interacting with each other and the environment. The primary focus is to evaluate all the genes of an organism rather than individual genes.This genetic information is being used to evaluate individual responses to drugs, leading to the development of targeted therapies. This, in turn, has pav ...

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 ...

- This RMAT designation is based on data from 3 clinical studies demonstrating the potential benefit of AAV-GAD as a one-time treatment for Parkinson’s disease - RMAT designation includes the benefits of the Fast Track and Breakthrough Therapy designations, allows frequent regulatory interactions with the FDA, and potential routes to accelerated approval and Priority Review LONDON and NEW YORK, May 09, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage gen ...

Dublin, April 29, 2025 (GLOBE NEWSWIRE) -- The "Cone Rod Dystrophy Market: Analysis By Type, By Treatment Type, By End User, By Region Size and Trends - Forecast up to 2030" has been added to ResearchAndMarkets.com's offering. Cone rod dystrophy represents a group of genetic eye disorders impacting the retina's cone and rod cells. The market for treating this condition is witnessing substantial growth, projected to rise from a value of US$131.29 million in 2024 to US$177.59 million by 2030. This growth is ...

I used to cover MeiraGTx (NASDAQ: MGTX ) a few years ago, but haven’t looked at it since 2022. MGTX develops genetic therapies for ocular diseases, Parkinson’s Disease and xerostomia. Lead asset is AAV-AQP1 targeting Xerostomia in a pivotal phase 2About the TPT ServiceThanks for reading. At the Total Pharma Tracker, we offer the following:-Our Android app and website features a set of tools for DIY investors, including a work-in-progress software where you can enter any ticker and get extensive curated rese ...