August 14, 2025 — MeiraGTx Holdings plc (Nasdaq: MGTX) announced its second-quarter financial and operational results, including alignment with the FDA on its Phase 2 study of AAV-hAQP1 in radiation-induced xerostomia (RIX). The company also received Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD in Parkinson’s disease.
The FDA granted RMAT designation for AAV-GAD to treat Parkinson’s. MeiraGTx and Hologen’s data shows significant disease-modifying effects. The company is on track to file for Marketing Authorization Approval (MAA) with the U.K. MHRA and a BLA in the U.S. for LCA4 treatment in Q4 2025.
MeiraGTx is collaborating with Hologen on AI analysis of imaging data to support labeling claims for AAV-GAD in Parkinson’s disease, with a Phase 3 study expected to begin later this year. The company also plans to initiate first-in-human studies using its riboswitch platform by the end of 2025.
Recent highlights include FDA alignment on CMC and clinical requirements for the Phase 2 AQUAx2 study of AAV2-hAQP1 for RIX. The company is also advancing AAV-GAD for Parkinson’s disease, with discussions ongoing with the FDA regarding AI-driven analysis of imaging data. Strategic collaboration with Hologen AI is progressing, with $23 million of the $200 million in cash consideration received.
MeiraGTx’s UK manufacturing facility holds MIA(IMP) and Specials Licences and the Irish facility holds MIA and MIA(IMP) Licences.
MeiraGTx had approximately $32.2 million in cash and cash equivalents as of June 30, 2025, believing that it has sufficient capital to fund operating expenses and capital expenditure requirements into 2027.
Investors:
MeiraGTx
Investors@meiragtx.com
Media:
Jason Braco, Ph.D.
LifeSci Communications
jbraco@lifescicomms.com
Source: MeiraGTx Holdings plc
