Press release
MeiraGTx Announces Positive Preliminary Data from the AQUAx Phase 1 Clinical Trial of AAV-hAQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia
Clinically meaningful improvements in xerostomia symptoms and disease burden reported in two validated Patient Reported Outcome (PRO) measures6 of the 7
About this update from Meiragtx Holdings Plc
[{"type":"text","content":"Clinically meaningful improvements in xerostomia symptoms and disease burden reported in two validated Patient Reported Outcome (PRO) measures6 of the 7 participants through 90-day assessments following treatment achieved clinically meaningful improvement in symptoms using both the McMaster Global Rate of Change PRO and the Xerostomia QuestionnaireOne participant with the maximum response evaluable at 12 months has now reached 24 months and the same level of response/xerostomia symptom improvement was maintainedAAV-hAQP1 appears safe and well-tolerated at each dose testedWebcast and conference call to be held today, December 7, 2021 at 8:00 a.m. ET LONDON and NEW YORK, Dec. 07, 2021 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced positive preliminary data from the ongoing Phase 1 AQUAx trial of AAV-hAQP1 for the treatment of grade 2/3 radiation-induced xerostomia (RIX). “We are very pleased to share this preliminary data from cohorts 1-3 of the AQUAx trial which provides encouraging evidence of the emerging clinical profile of AAV-hAQP1 for the treatment of radiation-induced xerostomia. While the primary endpoint of this Phase 1 trial is safety, we have seen both efficacy and durability in patients treated so far,” said Alexandria Forbes, Ph.D., President and Chief Executive Officer of MeiraGTx. “The size of the effects we are seeing at these early stages in the study are clinically meaningful and appear greater than those seen to date with approved drugs for RIX and xerostomia associated with Sjögren’s Syndrome. These results point to the potential for AAV-hAQP1 to be a disease-modifying one-time treatment for this large population of patients who currently have no effective treatment options for this devastating and intractable condition.” Study Design and Safety Update in the Phase 1 AQUAx Trial of AAV-hAPQ1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia AQUAx is an open label, multi-center, dose escalation study of a single administration of AAV-hAQP1 to one or both parotid glands in patients with radiation-induced salivary hypofunction and xerostomia. Four unilaterally treated escalating dose cohorts with a minimum of 3 subjects per cohort have completed treatment (n=12) and four bilaterally treated escalating dose cohorts h...