MGTX - MeiraGTx Announces Oral Presentation at the 2024 American Academy of Oral Medicine (AAOM) Annual Conference | Benzinga
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
Abstract:
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;
Objectives
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.
Methods
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).
Results
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 ...