Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
AAV2hAQP1: 1 x 10^11 vg/gland · 1 trial · 3 indications
Safety will be assessed by number of adverse events occurring with treatment
| Arm | Type | Description |
|---|---|---|
| 1 x 10^11 vg/gland (single gland) | EXPERIMENTAL | - |
| 3 x 10^10 vg/gland (both glands) | EXPERIMENTAL | - |
| 3 x 10^11 vg/gland (single gland) | EXPERIMENTAL | - |
| 1 x 10^11 vg/gland (both glands) | EXPERIMENTAL | - |
| 1 x 10^12 vg/gland (single gland) | EXPERIMENTAL | - |
| 3 x 10^11 vg/gland (both glands) | EXPERIMENTAL | - |
| 3 x 10^12 vg/gland (single gland) | EXPERIMENTAL | - |
| 1 x 10^12 vg/gland (both glands) | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| AAV2hAQP1: 1 x 10^11 vg/gland (single gland) | DRUG | Intra-parotid administration of AAV2hAQP1 via Stensen's duct to a single parotid gland at a dose level of 1 x 10\^11 vg/gland |
| AAV2hAQP1: 3 x 10^10 vg/gland (both glands) | DRUG | Intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 3 x 10\^10 vg/gland |
| AAV2hAQP1: 3 x 10^11 vg/gland (single gland) | DRUG | Intra-parotid administration of AAV2hAQP1 of via Stensen's duct to a single parotid gland at a dose level of 3 x 10\^11 vg/gland |
| AAV2hAQP1: 1 x 10^11 vg/gland (both glands) | DRUG | intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 1 x 10\^11 vg/gland |
| AAV2hAQP1: 1 x 10^12 vg/gland (single gland) | DRUG | Intra-parotid administration of AAV2hAQP1 via Stensen's duct to a single parotid gland at a dose level of 1 x 10\^12 vg/gland |
| AAV2hAQP1: 3 x 10^11 vg/gland (both glands) | DRUG | Intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 3 x 10\^11 vg/gland |
| AAV2hAQP1: 3 x 10^12 vg/gland (single gland) | DRUG | Intra-parotid administration of AAV2hAQP1 via Stensen's duct to a single parotid gland at a dose level of 3 x 10\^12 vg/gland |
| AAV2hAQP1: 1 x 10^12 vg/gland (both glands) | DRUG | Intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 1 x 10\^12 vg/gland |
Inclusion Criteria: 1. Male or female subjects ≥18 years of age. 2. History of radiation therapy for head and neck cancer. 3. Abnormal parotid gland function as judged by both absence of unstimulated parotid salivary flow and a stimulated parotid salivary flow in the targeted parotid gland \>0 and ...
AAV2hAQP1 is an investigational gene therapy being developed for radiation-induced parotid gland hypofunction, a condition that causes dry mouth (xerostomia) in patients who have undergone radiotherapy for head and neck cancer. It is administered directly into the parotid gland at a dose of 1 x 10^11 vg/gland.
AAV2hAQP1 is being developed by MeiraGTx Holdings plc, a biopharmaceutical company traded on the NASDAQ under the ticker symbol MGTX. The company is conducting clinical research on this asset for the treatment of radiation-induced parotid gland hypofunction.
AAV2hAQP1 is in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA. The drug has received FDA designations including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Breakthrough Therapy designations.
AAV2hAQP1 has one completed Phase 1 clinical trial, identified as NCT04043104. This open-label, dose escalation study enrolled 24 participants to evaluate the optimal dose, safety, and activity of AAV2hAQP1 in subjects with radiation-induced parotid gland hypofunction and xerostomia. The trial was conducted in the United States and Canada.
AAV2hAQP1 is a gene therapy that uses an adeno-associated virus (AAV) vector to deliver the human aquaporin-1 (hAQP1) gene to the parotid gland. This is intended to restore water transport in salivary gland cells damaged by radiation, thereby improving saliva production and reducing symptoms of dry mouth.