MeiraGTx Shows 3-Year Saliva Flow Gains and Durable Xerostomia Relief

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MeiraGTx’s Phase 1 AQUAx study showed that AAV-hAQP1 treatment maintained clinically meaningful xerostomia questionnaire improvements and elevated unstimulated whole saliva flow rates at three years in both bilateral and unilateral cohorts. No dose-related safety issues emerged across all tested levels, supporting the therapy’s durable efficacy and tolerability.

1. Clinical Data Highlights

The Phase 1 AQUAx study delivered AAV-hAQP1 via Stensen’s duct and reported maintenance of patient-reported xerostomia questionnaire (XQ) improvements through 36 months in both unilateral and bilateral cohorts. Unstimulated whole saliva flow rates increased from baseline and remained elevated at the three-year mark, demonstrating sustained restoration of salivary function after a single administration.

2. Trial Design and Patient Cohorts

AQUAx is an open-label, non-randomized, dose-escalation trial enrolling patients with grade 2/3 late radiation-induced xerostomia who have been cancer-free for at least five years (two years if HPV positive). Participants received one-time AAV-hAQP1 injections to one or both parotid glands and will be followed for a total of five years to assess long-term outcomes.

3. Safety and Tolerability

AAV-hAQP1 was well tolerated across all dose levels with no serious adverse events linked to treatment. Safety endpoints remained favorable throughout the 36-month period, reinforcing the therapy’s acceptable risk profile for further development.

4. Implications and Next Steps

The three-year durability data underscores AAV-hAQP1’s potential as a disease-modifying, one-time in-office therapy for persistent xerostomia, a condition with no approved treatments. MeiraGTx plans to leverage these results to advance to later-stage trials and engage with regulatory authorities on pivotal study design.

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