April 14, 2026-
MeiraGTx Holdings plc has announced an upcoming webcast to present three-year follow-up data from its Phase 1 AQUAx clinical trial evaluating AAV-hAQP1, an investigational gene therapy for radiation-induced xerostomia (RIX). The presentation is scheduled for April 16, 2026, and will provide one of the longest follow-up datasets to date for a gene therapy targeting salivary gland dysfunction.
Radiation-induced xerostomia is a common and debilitating consequence of head and neck cancer treatment, often resulting in chronic dry mouth, impaired swallowing, increased dental complications, and reduced quality of life. Current treatment options are largely supportive, underscoring the need for disease-modifying approaches that restore salivary gland function rather than simply managing symptoms.
The AQUAx trial is an open-label, dose-escalation Phase 1 study designed primarily to evaluate safety, while also exploring efficacy through both patient-reported outcomes and objective biological measures. The therapy, AAV-hAQP1, is administered via Stensen’s duct directly into one or both parotid glands, enabling localized delivery of the aquaporin-1 gene to restore fluid secretion in damaged salivary tissue.
The upcoming presentation will include comprehensive three-year data across all treated cohorts, including both bilateral and unilateral dosing groups. Key endpoints include patient-reported outcomes measured באמצעות the Xerostomia Questionnaire (XQ), as well as objective assessments such as Unstimulated Whole Saliva Flow Rate (UWSFR). Importantly, both cohort-level and individual patient data will be disclosed, offering deeper insight into variability of response and durability of effect over time.
This long-term dataset builds on previously reported 12-month results and is particularly relevant in the context of gene therapy, where durability remains a central question for clinical validation and regulatory approval. Patients in the AQUAx study are followed for up to five years after a single administration, positioning this dataset as a critical milestone in understanding sustained efficacy and safety.
Beyond clinical outcomes, the webcast will also include investigator perspectives on disease burden, patient experience, and treatment administration, as well as a discussion of the commercial opportunity. These elements reflect growing interest in gene therapies targeting non-lethal but highly impactful chronic conditions, expanding the scope of the field beyond rare genetic diseases.
Notably, MeiraGTx’s xerostomia program has also received regulatory momentum, with its AAV2-hAQP1 therapy previously granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration. This designation highlights the potential of the therapy to address a significant unmet medical need and may support accelerated development pathways.
As the gene therapy field continues to mature, the release of long-term clinical data—particularly in indications like xerostomia—will play a critical role in demonstrating real-world durability, functional benefit, and broader applicability of AAV-based platforms.
