MeiraGTx reports sustained 36-month efficacy in Phase 1 Xerostomia gene therapy trial

MeiraGTx Holdings (NASDAQ:MGTX), a clinical-stage gene therapy company, announced positive long-term results today from its completed Phase 1 AQUAx clinical trial.

The study evaluated AAV-hAQP1, an investigational gene therapy designed to treat grade 2/3 late radiation-induced xerostomia (chronic dry mouth), a debilitating condition common in head and neck cancer survivors.

The full 36-month dataset, released on April 16, 2026, confirms that the clinical benefits of a single, one-time in-office administration are maintained through three years.

Patients across all dose cohorts showed sustained improvements in the Patient-Reported Outcomes (PRO) XQ symptom score.

Crucially, the data also revealed a durable increase in unstimulated whole saliva flow, providing objective evidence of the therapy's biological activity in restoring salivary gland function.

Throughout the three-year follow-up period, AAV-hAQP1 remained safe and well-tolerated.

No dose-limiting toxicities or significant treatment-related serious adverse events were reported, reinforcing the safety profile of the viral vector delivery system in the salivary glands.

The company highlighted that the in-office delivery method significantly reduces the burden on both patients and the healthcare system compared to current palliative options.