Press release
MeiraGTx Reports Second Quarter 2023 Financial and Operational Results
Phase 3 LUMEOS clinical trial of botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP) enrollment
About this update from Meiragtx Holdings Plc
[{"type":"text","content":"Phase 3 LUMEOS clinical trial of botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP) enrollment target has now been surpassedAnnounced positive clinical data from the completed AQUAx Phase 1 study of AAV2-hAQP1 for the treatment of grade 2/3 radiation-induced xerostomia (RIX), showing bilaterally treated participants reaching normal levels of whole saliva flow rate by 2 months post-treatment and persisting through the final Month 12 assessment in the study Initiated Phase 2 randomized, double-blind, placebo-controlled study of AAV2-hAQP1 for the treatment of grade 2/3 RIXWholly-owned gene therapy manufacturing facility in Shannon, Ireland received Commercial MIA Authorization for QC Testing and is the first manufacturing site for gene therapy to receive a commercial license in IrelandPresented data on proprietary RiboCAR platform demonstrating improved CAR-T anti-cancer efficacy and durability at the 2023 American Society of Gene and Cell Therapy (ASGCT) Spotlight on Immuno-Oncology LONDON and NEW YORK, Aug. 10, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced financial and operational results for the second quarter ended June 30, 2023, and provided a corporate update. “Progress across our programs and infrastructure continues to be extremely impressive,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “This includes further advancement of our three lead clinical programs, our riboswitch gene regulation technology platform, and our manufacturing platform. This quarter, we announced positive data from the completed AQUAx Phase 1 study, demonstrating clinically meaningful improvements in long term xerostomia patients, and we are currently enrolling and treating patients in our Phase 2 trial. In the Phase 3 LUMEOS trial of bota-vec for the treatment of XLRP, we have now surpassed the enrollment target and expect a BLA submission next year. In addition, we presented unprecedented preclinical data from our novel gene regulation platform at the ASGCT Annual and ASGCT Spotlight on Immuno-oncology meetings. We are particularly excited by our groundbreaking RiboCAR technology. RiboCAR enables precise and reversible regulation of CAR expression in respo...