MeiraGTx Reports Fourth Quarter and Full Year 2024 Financial and Operational Results and Recent Business Updates

Core Viewpoint - MeiraGTx announced a strategic collaboration with Hologen AI, involving a $200 million upfront payment and the formation of a joint venture, Hologen Neuro AI Ltd, aimed at advancing the development of therapies for neurodegenerative diseases, particularly Parkinson's disease [1][2] Recent Developments - The joint venture will focus on the Phase 3 clinical development of AAV-GAD for Parkinson's disease, with an additional$230 million committed by Hologen [1][2] - MeiraGTx reported positive results from a clinical study of AAV-GAD, showing significant improvements in the Unified Parkinson's Disease Rating Scale (UPDRS) and the Parkinson's Disease Questionnaire (PDQ-39) [1][8] - The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV2-hAQP1 for treating Grade 2/3 radiation-induced xerostomia (RIX) [1][8] Clinical and Regulatory Milestones - MeiraGTx achieved FDA Rare Pediatric Disease Designations for four therapies targeting rare inherited retinal diseases, including rAAV8.hRKp.AIPL1 for LCA4 [3] - The company plans to submit a Marketing Authorization Application (MAA) for rAAV8.hRKp.AIPL1 based on exceptional clinical results observed in children [4][14] Financial Overview - As of December 31, 2024, MeiraGTx reported cash and cash equivalents of approximately $103.7 million, with expectations to fund operations into 2027 [19] - The company experienced a net loss of$147.8 million for the year ended December 31, 2024, compared to a net loss of $84.0 million in 2023 [32][39] - Service revenue for 2024 was$33.3 million, a significant increase from zero in 2023, attributed to process performance qualification services [24][26] Manufacturing and Regulatory Compliance - MeiraGTx's UK manufacturing facility received successful GMP compliance renewal, supporting its application for a commercial MIA license [16] - The Shannon facility in Ireland obtained additional licenses for GMP viral vector manufacturing, marking a significant milestone for gene therapy in the region [18] Strategic Partnerships - The collaboration with Hologen AI is expected to enhance the efficiency and success probability of MeiraGTx's clinical programs through the application of advanced AI technologies [2][8] - Sanofi's strategic investment of $30 million in MeiraGTx provides additional financial backing and a right of first negotiation for certain therapeutic targets [23]