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MeiraGTx announces clinical data from the AQUAx Phase 1 study of AAV2-hAQP1
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MeiraGTx announces clinical data from the AQUAx Phase 1 study of AAV2-hAQP1

MeiraGTx announced positive clinical data from the ongoing Phase 1 AQUAx study of AAV2-hAQP1 for the treatment of grade 2/3 radiation-induced xerostomia. AQUAx is an open label, multi-center, dose escalation study of a single administration of AAV2-hAQP1 to one or both parotid glands in patients with radiation-induced salivary hypofunction and grade 2/3 xerostomia. As of the cutoff date of November 30, 2022, all 12 unilaterally treated participants have undergone their 12-month assessment, with 3 having completed their 24-month assessment and one having completed their 36-month assessment in the long-term follow-up study. Efficacy Data presented from the 24 participants treated in the AQUAx study: Clinically meaningful improvements in xerostomia symptoms reported consistently across two validated PROs assessing xerostomia symptom severity; Meaningful increases in whole saliva flow rates observed post-treatment, providing objective evidence of biological activity of AAV2-hAQP1 treatment; Early long-term follow-up data suggest durability of improvement 2+ years post-treatment. Global Rate of Change: Bilateral Cohorts to 6 Months: 10/12 participants reported symptoms of dry mouth as "better" at the 6 month timepoint; No participant reported any worsening of xerostomia symptoms. Unilateral Cohorts to 12 Months: 8/12 participants who reached the 12-month assessment reported symptoms of dry mouth as "better" following treatment; Improvement in xerostomia symptoms can be seen persisting through 2 years in all 3 patients who have reached that timepoint; Participant 1-1 reached the 3-year assessment and the maximum score of 7 was maintained; No participant reported any worsening of xerostomia symptoms. Combined Unilateral and Bilateral Cohorts: 18/24, or 75%, of all unilateral and bilateral treated participants reported symptoms of dry mouth as "better" following treatment; In the overall cohorts the average improvement in scores was greater in bilateral participants compared to unilateral participants; Unilateral cohort achieved overall improvement of greater than3 points at 12 months; Bilateral cohort achieved overall improvement of greater than3 points at 2 months and an overall improvement of 4 points by 6 months; overall improvement in scores was maintained and increased over time in both unilateral and bilateral cohorts

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