MeiraGTx to Present 3-Year AAV-hAQP1 Gene Therapy Data for Radiation-Induced Xerostomia Treatment

Key Takeaways

• MeiraGTx will present comprehensive 3-year follow-up data from Phase 1 AQUAx trial of AAV-hAQP1 gene therapy for Grade 2/3 radiation-induced xerostomia
• Data presentation will include both patient-reported outcomes and objective measures from bilateral and unilateral treatment cohorts
• Conference call scheduled for April 16, 2026, marking a significant milestone for this novel gene therapy approach to treating dry mouth after cancer radiation

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MeiraGTx Announces Major Data Presentation for Xerostomia Gene Therapy

MeiraGTx Holdings plc (NASDAQ: MGTX) announced it will host a conference call and webcast on April 16, 2026, to present comprehensive 3-year data from the Phase 1 AQUAx clinical study evaluating AAV-hAQP1 gene therapy for treating Grade 2/3 radiation-induced xerostomia.

Comprehensive Long-Term Data Release

The presentation will feature data from all cohorts in the Phase 1 trial, including detailed cohort-level and individual patient data from both bilateral and unilateral treatment groups. The company will present both patient-reported outcomes (PRO) and objective clinical measures, providing a complete picture of the therapy’s long-term efficacy and safety profile.

Addressing Critical Unmet Medical Need

Radiation-induced xerostomia, commonly known as dry mouth, affects thousands of cancer patients who receive radiation therapy for head and neck cancers. This condition significantly impacts quality of life, making eating, speaking, and swallowing difficult. Current treatment options are limited and often provide only temporary relief.

AAV-hAQP1 represents a novel gene therapy approach that aims to restore salivary gland function by delivering the human aquaporin-1 gene directly to damaged salivary tissue. The therapy uses an adeno-associated virus (AAV) vector to potentially provide long-lasting treatment benefits.

Market and Clinical Implications

The 3-year follow-up data represents crucial evidence for the therapy’s durability and long-term safety profile. Gene therapies typically require extensive long-term data to demonstrate sustained efficacy and acceptable safety profiles for regulatory approval.

MeiraGTx, a vertically integrated clinical-stage genetic medicines company, has been developing this treatment as part of its broader gene therapy portfolio. The company’s integrated approach allows for end-to-end development and manufacturing capabilities.

Looking Forward

The upcoming data presentation will be closely watched by investors, clinicians, and patient advocacy groups as it could inform future development decisions and potential regulatory submissions for this innovative treatment approach.

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Frequently Asked Questions

What is radiation-induced xerostomia and how does AAV-hAQP1 treat it?

Radiation-induced xerostomia is severe dry mouth caused by radiation therapy damaging salivary glands in cancer patients. AAV-hAQP1 is a gene therapy that delivers the aquaporin-1 gene to restore salivary gland function and saliva production.

When will AAV-hAQP1 be available to patients?

AAV-hAQP1 is currently in Phase 1 clinical trials. The therapy would need to complete additional clinical phases and receive regulatory approval before becoming commercially available, which typically takes several years.

How does this gene therapy compare to current xerostomia treatments?

Current treatments for radiation-induced xerostomia are limited to saliva substitutes, medications, and supportive care that provide temporary relief. AAV-hAQP1 aims to restore actual salivary gland function, potentially offering longer-lasting benefits than existing options.