Press release
MeiraGTx Reports Fourth Quarter and Full Year 2023 Financial and Operational Results and Recent Business Updates
- Company aligned with FDA on requirements for ongoing Phase 2 AQUAx2 clinical trial for Grade 2/3 radiation-induced xerostomia to be considered pivotal
About this update from Meiragtx Holdings Plc
[{"type":"text","content":"- Company aligned with FDA on requirements for ongoing Phase 2 AQUAx2 clinical trial for Grade 2/3 radiation-induced xerostomia to be considered pivotal supporting potential BLA filing - Company to present data from the Phase 1 AQUAx study in an oral presentation at the American Academy of Oral Medicine 2024 annual meeting (AAOM) April 17-20, 2024 - Completed dosing the three-arm randomized, sham-controlled Phase 1 clinical bridging study for AAV-GAD for the treatment of Parkinson’s disease - Company to present positive data on AAV-AIPL1 treatment of 8 LCA4 patients each with meaningful responses in second quarter 2024 - Entered into an asset purchase agreement with Janssen Pharmaceuticals, Inc. (Janssen) related to botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP) for up to $415 million, as well as a commercial supply agreement pursuant to which the Company will manufacture and supply bota-vec to Janssen - Received $30 million strategic investment from Sanofi and entered into a right of first negotiation (ROFN) agreement for the use of MeiraGTx’s riboswitch gene regulation technology for certain Central Nervous System (CNS) and Immunology and Inflammation (I&I) targets, as well as for GLP-1 and other gut peptides for metabolic disease, and for MeiraGTx’s Phase 2 xerostomia program LONDON and NEW YORK, March 14, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced financial and operational results for the fourth quarter and full-year ended December 31, 2023, and provided a corporate update. “We are very pleased with the progress made at MeiraGTx in the past year across every area of the company, as well as the two transactions that we executed in the fourth quarter of 2023, bolstering our balance sheet. We advanced each of our three lead clinical programs, and we now have three studies in late-stage clinical development. For our program for radiation-induced xerostomia, we presented unprecedented positive data from our Phase 1 AQUAx study in June 2023; we opened the Phase 2 AQUAx2 study mid-year 2023, a randomized, double-blind placebo-controlled study of AAV-hAQP1, and last month we aligned with the FDA on the requirements for this Phase 2 study to be pivotal supporting a poten...