MeiraGTx Announces Positive Clinical Data from the AQUAx Phase 1 Clinical Study of AAV2-hAQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia

• Clinically meaningful improvements in xerostomia symptoms measured by two different xerostomia PROs demonstrated across both unilaterally and bilaterally treated cohorts at 12 months
• Increases in whole saliva flow rates observed post-treatment, providing objective evidence of biological activity, reaching the normal range in bilaterally treated participants by 2 months and persisting through the Month 12 assessment
• Across assessments, greater improvements observed in bilaterally treated participants compared to those treated unilaterally

• Early long-term follow-up data suggest durability of improvement to at least 3 years post-treatment
• AAV2-hAQP1 appears safe and well tolerated at each dose tested
• The strong safety and encouraging, clinically meaningful activity data support further clinical development of AAV2-hAQP1
• Phase 2 randomized, double-blind, placebo-controlled study initiated with participants currently being dosed
• Webcast and conference call to be held today, June 27, 2023, at 8:00 a.m. ET

LONDON and NEW YORK, June 27, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical stage gene therapy company, today announced positive clinical data from the completed Phase 1 AQUAx study of AAV2-hAQP1 for the treatment of grade 2/3 radiation-induced xerostomia (RIX).

“Today we are releasing data from the completed Phase 1 AQUAx study which now includes the final 12 month data from all bilaterally treated participants. The data demonstrate clinically important improvements in two different patient reported outcome questionnaires for xerostomia, as well as meaningful increases in absolute saliva production in both bilaterally and unilaterally treated cohorts at 12 months,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “We are also presenting additional data from our long-term follow-up study demonstrating durability of improvement in participants who have reached the 2- and 3-year post-treatment assessment, as well as supportive data showing transduction of parotid glands treated with AAV2-hAQP1 up to 24 months post-treatment and AQP1 protein expression.”