MeiraGTx Announces Oral Presentation at the 2024 American Academy of Oral Medicine (AAOM) Annual Conference

April 18, 2024

LONDON and NEW YORK, April 18, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced the Company gave an oral presentation at the American Academy of Oral Medicine Annual Conference, being held from April 16-20, 2024, at the Hyatt Regency Grand Cypress in Orlando, FL.

The details of the oral presentation are below:

Session: Oral Abstract Session I
Presentation ID #196
Title: Results of a Phase 1, Open-label, Dose-escalation Study of Gene Therapy with AAV2-hAQP1 as Treatment for Grade 2 and 3 Radiation-induced Late Xerostomia and Parotid Gland Hypofunction – The AQUAx Study
Presenting Author: Dr. Michael Brennan
Time: 4:20pm ET

Results of a Phase 1, Open-label, Dose-escalation Study of Gene Therapy with AAV2-hAQP1 as Treatment for Grade 2 and 3 Radiation-induced Late Xerostomia and Parotid Gland Hypofunction

Michael Brennan5, Michael Passineau1, Deborah Saunders4, Herve Sroussi2, Dyani Gaudilliere3, Arthur Fernandez1, Jun Liu1, Nathalie Dubois1, Robert Zeldin1
1MeiraGTx, New York, NY; 2Brigham and Women's Hospital, Boston, MA; 3Stanford University, Palo Alto, CA; 4Health Sciences North, Sudbury, ON, Canada; 5Atrium Health, Charlotte, NC; 

Grade 2/3 late xerostomia is a chronic, debilitating complication of radiotherapy for head and neck cancers. We assessed the safety and efficacy of AAV2-hAQP1 gene therapy as a treatment for this condition.

Twenty-four participants with Grade 2/3 xerostomia at least five years after completing radiotherapy (2 years if HPV+) were enrolled in this multi-center, open-label, dose-escalation study. AAV2-hAQP1 was delivered to the parotid gland(s) via cannulation of Stensen’s duct. Twelve participants received AAV2-hAQP1 in one gland and 12 in both glands. Participants were followed for 12 months post-treatment.

Safety parameters included adverse events, physical examinations, laboratory tests, and electrocardiograms. Efficacy assessments included the Xerostomia-specific Questionnaire (XQ), MD Anderson Symptom Inventory-Head and Neck Module (MDASI-HN), Global Rate of Change Questionnaire (GRCQ), and measurement of unstimulated and stimulated whole saliva flow rates (UWSFR, SWSFR).

No treatment-related serious adverse events or dose-limiting toxicities were reported, and all participants completed the study.

Statistically significant improvements were seen in the patient-reported outcome (PRO) instruments by Day 30 and were maintained through Month 12, with greater improvement in the bilateral versus unilateral cohorts.

At Month 12, the mean percent change from baseline (%CFB) was -39.5% and -42.2% for the XQ Total Score and the Dry Mouth question of the MDASI-HN, respectively, and the mean GRCQ symptom score was 3.8. Overall, 16/24 participants reported an improvement of ≥8 points in the XQ Total Score, and 19/24 participants reported important improvements in xerostomia symptoms based on the GRCQ. The improvement reported across PRO instruments, measuring different aspects of xerostomia symptoms, provides compelling evidence of treatment effectiveness.

The mean %CFB in UWSFR at Month 12 was 112.5%, and a trend toward improved SWSFR was observed.

Treatment with AAV2-hAQP1 was safe and well-tolerated at all doses and resulted in meaningful improvements in xerostomia symptoms and unstimulated whole saliva flow rate.

The presentation will be available on the Posters and Publications page of the Company’s website after the respective presentation session has concluded.

About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage gene therapy company with a broad pipeline of late-stage clinical programs supported by end-to-end manufacturing capabilities. MeiraGTx has an internally developed manufacturing platform process, internal plasmid production for GMP, two GMP viral vector production facilities as well as an in-house Quality Control hub for stability and release, all fit for IND through commercial supply. MeiraGTx has core capabilities in viral vector design and optimization and a potentially transformative riboswitch gene regulation platform technology that allows for the precise, dose-responsive control of gene expression by oral small molecules. MeiraGTx is focusing the riboswitch platform on delivery of metabolic peptides including GLP-1, GIP, Glucagon and PYY using oral small molecules, as well as cell therapy for oncology and autoimmune diseases. Although initially focusing on the eye, central nervous system, and salivary gland, MeiraGTx has developed the technology to apply genetic medicine to more common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains great.

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Forward Looking Statement
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Jason Braco, Ph.D.
LifeSci Communications