MeiraGTx Reports Durable Phase 1 AQUAx Gene Therapy Data

An update from Meiragtx Holdings ( (MGTX) ) is now available.

On April 16, 2026, MeiraGTx reported three-year follow-up data from its Phase 1 AQUAx trial of AAV-hAQP1 in patients with grade 2/3 late radiation-induced xerostomia, showing the one-time gene therapy remained safe and well tolerated with no dose-limiting toxicity or treatment-related serious adverse events. Clinically meaningful improvements in patient-reported Xerostomia Questionnaire scores and normalization of unstimulated whole saliva flow seen at 12 months were sustained through 36 months in both unilateral and bilateral treatment cohorts.

The company said these durable responses support viewing AAV-hAQP1 as a potential first-in-class, disease-modifying therapy for this severe, lifelong condition, which affects an estimated 165,000 people in the U.S. and about 435,000 globally. MeiraGTx highlighted strong physician enthusiasm and favorable payer perceptions from market research, reinforcing the therapy’s perceived commercial potential in a concentrated treatment landscape, although the AQUAx study remains an early-stage, open-label trial primarily designed to assess safety.

The AQUAx Phase 1 trial evaluates AAV-hAQP1 delivered via Stensen’s duct to one or both parotid glands in head and neck cancer survivors who have remained cancer-free for years after radiation. Participants are followed for five years after the single administration, with safety as the primary endpoint and efficacy assessed through symptom questionnaires and changes in salivary output over time.

The most recent analyst rating on (MGTX) stock is a Buy with a $18.00 price target. To see the full list of analyst forecasts on Meiragtx Holdings stock, see the MGTX Stock Forecast page.

Spark’s Take on MGTX Stock

According to Spark, TipRanks’ AI Analyst, MGTX is a Neutral.

The score is held back primarily by weak financial fundamentals—large ongoing losses, continued cash burn, and a deteriorated capital structure with negative equity—despite strong recent revenue/gross margin improvement. Technicals are constructive with the stock in an uptrend, and corporate events add support via regulatory and partnership/financing developments, but valuation remains challenging given negative earnings.

To see Spark’s full report on MGTX stock, click here.

More about Meiragtx Holdings

MeiraGTx Holdings plc is a vertically integrated, clinical-stage genetic medicines company focused on developing gene therapies for inherited and more common diseases in the eye, Parkinson’s disease, and radiation-induced xerostomia. The Nasdaq-listed group controls end-to-end in-house manufacturing, including GMP-licensed viral vector facilities and a proprietary platform for in vivo gene regulation using oral small molecules.

Average Trading Volume: 497,099

Technical Sentiment Signal: Buy

Current Market Cap: $916.3M

For an in-depth examination of MGTX stock, go to TipRanks’ Overview page.