Press release
MeiraGTx Reports First Quarter 2024 Financial and Operational Results
- Positive data from the Phase 1 AQUAx study in radiation-induced xerostomia (RIX) presented in an oral session at the American Academy of Oral Medicine 2024
About this update from Meiragtx Holdings Plc
[{"type":"text","content":"- Positive data from the Phase 1 AQUAx study in radiation-induced xerostomia (RIX) presented in an oral session at the American Academy of Oral Medicine 2024 annual meeting (AAOM) April 17-20, 2024 - Received $50 million milestone following initiation of the extension study for the Phase 3 LUMEOS clinical trial for botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP) - Clinical development milestones met and remain on track for the rest of the year for AAV-hAQP1 for RIX, bota-vec for XLRP, and AAV-GAD for Parkinson’s disease LONDON and NEW YORK, May 09, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced financial and operational results for the first quarter ended March 31, 2024, and provided a corporate update. “MeiraGTx’s first quarter was highlighted by significant progress in our wholly-owned late stage clinical programs, including our pivotal xerostomia program and Parkinson’s program as well as our riboswitch platform,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “At this year’s AAOM meeting in April, extremely encouraging data was presented from the Phase 1 AQUAx study, showing that treatment with AAV2-hAPQ1 resulted in significant improvements in 3 different patient reported outcomes (PROs) as well as unprecedented improvement in the production of saliva. We continue to hear from investigators that our therapy is ‘life changing’ for their patients fortunate enough to have had access to the treatment. Dosing in our pivotal Phase 2 AQUAx2 trial is ongoing, and we are pleased to have aligned with FDA on requirements for this Phase 2 study to be considered pivotal to support potential BLA filing for this significant unmet need.” Dr. Forbes continued: “Having completed dosing in our bridging study for AAV-GAD manufactured at MeiraGTx, we remain on track to discuss Phase 3 study design with global regulators for Parkinson’s disease in the second half of 2024. In addition, we continue to anticipate data from the large, multi-center Phase 3 study of bota-vec for XLRP-RPGR in collaboration with Janssen Pharmaceuticals, Inc. (Janssen) towards the end of this year, setting up the potential $285 million in milestones under the asset purchase agre...