AAV2hAQP1 / MeiraGTx - LARVOL DELTA

Long-term Follow-up of Gene Therapy for Radiation-Induced Xerostomia (clinicaltrials.gov) - P2 | N=120 | Enrolling by invitation | Sponsor: MeiraGTx, LLC

Gene therapy • New P2 trial • Dental Disorders • Gene Therapies • Oncology • Xerostomia

Results of a phase 1 open‐label, dose escalation study of gene therapy with AAV2‐hAQP1 as treatment for radiation‐induced xerostomia and parotid gland hypofunction (ESGCT 2023) - "On all efficacy measures, greater improvement was observed in bilaterally treated participants than in those treated unilaterally.Treatment with AAV2-hAQP1 was safe, resulted in important improvements in symptoms of radiation-induced xerostomia, and increased unstimulated whole saliva flow rate. Based on these promising results, a Phase 2 study has been initiated."

Clinical • Gene therapy • P1 data • Dental Disorders • Gene Therapies • Head and Neck Cancer • Immunology • Oncology • Solid Tumor • Xerostomia • AQP1

Long-Term Follow-Up Study of AAV2hAQP1 for Radiation Induced Xerostomia (clinicaltrials.gov) - P=N/A | N=22 | Active, not recruiting | Sponsor: MeiraGTx, LLC | Enrolling by invitation ➔ Active, not recruiting | Trial completion date: Nov 2027 ➔ Mar 2027 | Trial primary completion date: Nov 2026 ➔ Mar 2027

Enrollment closed • Trial completion date • Trial primary completion date • Dental Disorders • Xerostomia

AQUAX2: A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia (clinicaltrials.gov) - P2 | N=120 | Recruiting | Sponsor: MeiraGTx, LLC

Gene therapy • Trial completion date • Trial primary completion date • Dental Disorders • Gene Therapies • Head and Neck Cancer • Oncology • Xerostomia

Safety of a Single Administration of AAV2hAQP1, an Adeno-Associated Viral Vector Encoding Human Aquaporin-1 to One Parotid Salivary Gland in People With Irradiation-Induced Parotid Salivary Hypofunction (clinicaltrials.gov) - P1 | N=17 | Active, not recruiting | Sponsor: MeiraGTx, LLC | Recruiting ➔ Active, not recruiting

Enrollment closed • Viral vector • Dental Disorders • Head and Neck Cancer • Oncology • Oropharyngeal Cancer • Solid Tumor • Squamous Cell Carcinoma of Head and Neck • Xerostomia

A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia (clinicaltrials.gov) - P2 | N=120 | Recruiting | Sponsor: MeiraGTx, LLC

Gene therapy • New P2 trial • Dental Disorders • Gene Therapies • Head and Neck Cancer • Oncology • Oropharyngeal Cancer • Solid Tumor • Xerostomia

Safety of a Single Administration of AAV2hAQP1, an Adeno-Associated Viral Vector Encoding Human Aquaporin-1 to One Parotid Salivary Gland in People With Irradiation-Induced Parotid Salivary Hypofunction (clinicaltrials.gov) - P1 | N=21 | Recruiting | Sponsor: MeiraGTx, LLC | N=36 ➔ 21 | Trial completion date: Jul 2025 ➔ Jul 2026 | Trial primary completion date: Jul 2025 ➔ Jul 2026

Enrollment change • Trial completion date • Trial primary completion date • Viral vector • Dental Disorders • Head and Neck Cancer • Oncology • Oropharyngeal Cancer • Solid Tumor • Squamous Cell Carcinoma of Head and Neck • Xerostomia

A Phase 1 Open-Label, Dose Escalation Study to Determine the Optimal Dose, Safety, and Activity of AAV2hAQP1 in Subjects With Radiation-Induced Parotid Gland Hypofunction and Xerostomia (clinicaltrials.gov) - P1 | N=24 | Completed | Sponsor: MeiraGTx UK II Ltd | Active, not recruiting ➔ Completed

Trial completion • Dental Disorders • Head and Neck Cancer • Oncology • Solid Tumor • Xerostomia

A Phase 1 Open-Label, Dose Escalation Study to Determine the Optimal Dose, Safety, and Activity of AAV2hAQP1 in Subjects With Radiation-Induced Parotid Gland Hypofunction and Xerostomia (clinicaltrials.gov) - P1 | N=24 | Active, not recruiting | Sponsor: MeiraGTx UK II Ltd | Recruiting ➔ Active, not recruiting | Trial completion date: May 2022 ➔ Mar 2023 | Trial primary completion date: Dec 2021 ➔ Mar 2023

Enrollment closed • Trial completion date • Trial primary completion date • Dental Disorders • Head and Neck Cancer • Oncology • Solid Tumor • Xerostomia

Safety of a Single Administration of AAV2hAQP1, an Adeno-Associated Viral Vector Encoding Human Aquaporin-1 to One Parotid Salivary Gland in People With Irradiation-Induced Parotid Salivary Hypofunction (clinicaltrials.gov) - P1 | N=36 | Recruiting | Sponsor: MeiraGTx UK II Ltd | Trial completion date: Nov 2022 ➔ Jul 2025 | Trial primary completion date: Nov 2022 ➔ Jul 2025

Trial completion date • Trial primary completion date • Dental Disorders • Head and Neck Cancer • Oncology • Oropharyngeal Cancer • Solid Tumor • Squamous Cell Carcinoma of Head and Neck • Xerostomia

A Phase 1 Open-Label, Dose Escalation Study to Determine the Optimal Dose, Safety, and Activity of AAV2hAQP1 in Subjects With Radiation-Induced Parotid Gland Hypofunction and Xerostomia (clinicaltrials.gov) - P1; N=30; Recruiting; Sponsor: MeiraGTx UK II Ltd; Phase classification: P1/2 ➔ P1

Clinical • Phase classification • Dental Disorders • Head and Neck Cancer • Oncology • Solid Tumor • Xerostomia

A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Optimal Dose, Safety, and Activity of AAV2hAQP1 in Subjects With Radiation-Induced Parotid Gland Hypofunction and Xerostomia (clinicaltrials.gov) - P1/2; N=42; Recruiting; Sponsor: MeiraGTx UK II Ltd; Active, not recruiting ➔ Recruiting; N=15 ➔ 42

Clinical • Enrollment change • Enrollment open

Safety of a Single Administration of AAV2hAQP1, an Adeno-Associated Viral Vector Encoding Human Aquaporin-1 to One Parotid Salivary Gland in People With Irradiation-Induced Parotid Salivary Hypofunction (clinicaltrials.gov) - P1; N=50; Recruiting; Sponsor: MeiraGTx UK II Ltd; Phase classification: P1/2 ➔ P1

Clinical • Phase classification