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AAV2-hAQP1

Phase 2

Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands | Gene therapy | Oncology |MeiraGTx Holdings plc|Last Updated: Oct 16, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedNO_TREATMENT_CONTROLLEDDMC
Total Trials1
Total Enrollment276
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06544798Long-term Follow-up of Gene Therapy for Radiation-Induced XerostomiaPHASE2 ENROLLING BY_INVITATION 276Aug 2, 2024Feb 1, 2032Oct 16, 20258 United States, Canada
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Study Endpoints
Primary Endpoints
Number of participants with study drug-related adverse events and serious adverse events
From study start until Month 60 post-treatment
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Follow-up groupNO_INTERVENTIONParticipants who were randomized to AAV2-hAQP1 treatment in Study MGT-AQP1-201 will continue in this long-term follow-up schedule to complete an additional 48 months of follow-up after their end-of-study visit in Study MGT-AQP1-201. All study participants are to be followed for a period of 60 months after vector administration.
Active treatment groupEXPERIMENTALParticipants who were randomized to placebo treatment in Study MGT-AQP1-201, will be offered to transition from the long-term follow-up schedule to an active treatment schedule at the time of unblinding. Study drug administration should be completed after unblinding and after confirmation of the participant's continued eligibility for treatment. Upon completion of the 12-month primary treatment period, participants will continue in the study according to a follow-up schedule, to complete a total of 60 months of follow-up after AAV2-hAQP1 administration.
Interventions
NameTypeDescription
AAV2-hAQP1GENETICAdministration of a prespecified concentration of AAV2-hAQP1 via Stensen's duct to each parotid gland
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: * Received study drug in Study MGT-AQP1-201 Exclusion Criteria: * Withdrew consent to participate in Study MGT-AQP1-201.

Countries:United StatesCanada
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT06544798primaryCompletionDate: changed
LOWMay 24, 2026NCT06544798studyFirstPostDate: changed