Press release
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
About this update from Meiragtx Holdings Plc
[{"type":"text","content":"- 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 AAV-GAD for the treatment of Parkinson’s disease - Gained alignment with 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 BLA filing - Efficacy data of rAAV8.hRKp.AIPL1 for the treatment of AIPL1-related retinal dystrophy, or LCA4, published in The Lancet, demonstrating meaningful responses in 11 out of 11 LCA4 children treated. Filing for Marketing Authorization Approval (MAA) under exceptional circumstances with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and in discussions with the FDA around a potentially similar pathway to approval in the US LONDON and NEW YORK, May 13, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced financial and operational results for the first quarter ended March 31, 2025, and provided a corporate update. “The first quarter of 2025 was a pivotal quarter for MeiraGTx, as we engaged in positive interactions with the FDA around each of our late-stage clinical programs and move forward with potentially BLA-supporting Phase 2 and Phase 3 clinical studies and BLA filings in each of our late-stage clinical programs,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “We are working with global regulators to file for expedited approval under exceptional circumstances of AAV-AIPL1 for children with LCA4 based on the unprecedented data from the treatment of the 11 young children under our Specials License in the U.K. We have now engaged in positive discussions with the FDA aligning on the requirements to support a similar pathway in the US. We have also gained alignment with the FDA on the requirements for the ongoing Phase 2 AQUAx2 study for AAV-hAQP1 for the treatment of RIX to support a potential BLA. In both cases, our alignm...