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 - Seite 3

• 8/12 participants at 12 months reported symptoms of dry mouth as “better” following treatment
• Each of these 8 participants rated changes in xerostomia scores that were “important” or “very important” with a score of 2 or more at 12 months
• 4 of these participants rated the change in xerostomia symptoms with the highest improvement scores of 6 or 7 denoting “a very important improvement”
• Improvement in xerostomia symptoms persisted through 2 years in 4 participants and 3 years in 3 participants who reached these timepoints
• No participant reported worsening of xerostomia symptoms
  

Combined Unilateral and Bilateral Cohorts

• Overall, the average improvement in GRCQ score was greater in bilaterally treated participants compared to those treated unilaterally
• Improvements were maintained and increased over time in both unilateral and bilateral cohorts 
• A 2-point change in GRCQ is considered important by patients
• Changes of 3 points or greater are considered a substantial improvement over standard of care and “transformative” by KOLs
• Unilaterally treated cohorts achieved overall improvement of >3 points at 12 months
• Bilaterally treated cohorts achieved overall improvement of >3 points at 2 months and an overall improvement of 4 points by 6 months, with this 4-point improvement maintained at 12 months

Xerostomia Questionnaire (XQ):

Xerostomia Questionnaire (XQ) PRO measure scoring scale

• An improvement (decrease) of 8 points or more is considered clinically meaningful
• A decrease in score of 10 or greater is considered a substantial improvement over standard of care and “transformative” by KOLs

Change from Baseline Unilateral and Bilateral Cohorts

• Unilateral: 7/12 had score improvements (decrease) ≥8 at 12 months
• Bilateral: 9/12 had score improvements ≥8 at 12 months
• Overall: 16/24 (66%) had an improvement following treatment of ≥8 points
• 50% of unilaterally treated participants and 75% of bilaterally treated participants at 12 months achieved at least a 10-point improvement
• There was good concordance between the responses on the GRCQ and XQ
  

Average Change from Baseline Unilateral and Bilateral Cohorts

• In unilaterally treated participants, an average 13-point improvement from baseline in XQ was seen at 12 months
• In bilaterally treated participants, an average 21-point improvement from baseline in XQ was seen at 12 months
• In both groups, XQ scores improved (declined) >8 points soon after treatment, and >10 points within 2 months after treatment
• This level of benefit is considered transformative by KOLs
• The degree of improvement in scores was greater in bilaterally treated participants compared to those treated unilaterally