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]

Core Insights - MeiraGTx announced a strategic collaboration with Hologen AI, involving a $200 million upfront payment and a joint venture, Hologen Neuro AI Ltd, with an additional $230 million committed to expedite the Phase 3 clinical development of AAV-GAD for Parkinson's disease [1][2][9] - The efficacy data for rAAV8.hRKp.AIPL1 for treating AIPL1-related retinal dystrophy was published, showing positive responses in all 11 children treated, leading to plans for Marketing Authorization Approval (MAA) in the UK and discussions with the FDA for a similar pathway in the US [1][2][10] Financial and Operational Highlights - As of March 31, 2025, MeiraGTx had cash and cash equivalents of approximately $66.5 million, with a net loss attributable to ordinary shareholders of $40.0 million for the quarter [21][31] - Service revenue increased to $1.9 million for the three months ended March 31, 2025, compared to $0.7 million for the same period in 2024, attributed to progress in process performance qualification services [23] - General and administrative expenses decreased to $9.4 million for the first quarter of 2025, down from $13.2 million in the same quarter of 2024, primarily due to reductions in share-based compensation and other costs [25] Clinical Development Updates - The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD for Parkinson's disease, allowing for expedited development and increased interaction with the FDA [4][9] - Positive discussions with the FDA have aligned on the requirements for ongoing Phase 2 studies for AAV-hAQP1 and AAV-GAD, supporting potential BLA filings [2][10] - The Phase 2 AQUAx2 study for AAV-hAQP1 continues to enroll participants, with a target for completion in Q4 2025 and potential BLA filing by the end of 2026 [13] Strategic Collaborations and Future Plans - The joint venture with Hologen Neuro AI Ltd aims to leverage AI technology to enhance the development of therapies targeting CNS disorders, with a focus on the AAV-GAD program [2][9] - MeiraGTx plans to initiate a Phase 3 study of AAV-GAD in the second half of 2025, continuing to work closely with regulatory agencies to expedite the development process [9][10]