AAV-AQP1 is a Gene Therapy owned by MeiraGTx, and is involved in 5 clinical trials, of which 4 are ongoing, and 1 is planned.

Gene therapy works by activating aquaporin-1. Aquaporin-1 is a water channel protein for plasma membranes of red cells. The therapy induce aquaporin-1 to stimulate water channel formation, thereby permitting water to move in the direction of an osmotic gradient and may checks the progression of disease.

The revenue for AAV-AQP1 is expected to reach a total of $92m through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the AAV-AQP1 NPV Report.

AAV-AQP1 is originated and owned by MeiraGTx.

AAV-AQP1 Overview

AAV-AQP1 is under development for the treatment of xerostomia resulting from the use of ionizing radiation in the treatment of head and neck cancer and xerostomia associated with Sjogren’s syndrome. The drug candidate is administered slowly into the single parotid salivary gland. It consists of adeno-associated virus vector serotype 2 containing the human aquaporin-1 gene (AAV2hAQP1). The therapeutic candidate is developed based on synthetic biology and gene regulation platform. 

MeiraGTx Overview

MeiraGTx is a gene therapy company that develops treatments for ocular, salivary gland and central nervous system diseases. It is investigating AAV-RPGR program against X-linked retinitis pigmentosa (XLRP); AAV-RPE65 gene therapy to treat RPE65 (retinal pigment epithelium-specific 65 kDa) gene deficiency; AAV-CNGB3 drug for achromatopsia (ACHM); and A006 program targeting wet AMD (age-related macular degeneration). The company is also evaluating AAV-GAD medicine to treat Parkinson’s disease; AAV-UPF1 against amyotrophic lateral sclerosis (ALS); AAV-AQP1 for radiation-induced xerostomia (RIX); and AAV-AQP1 program to treat xerostomia caused due to Sjogren’s syndrome. MeiraGTx is developing riboswitch inducible expression programs targeting ophthalmology and oncology indications, diabetes and metabolic disorders, among others. The company operates in the UK, and the Netherlands. MeiraGTx is headquartered in New York City, New York, the US.

The company reported revenues of (US Dollars) US$37.7 million for the fiscal year ended December 2021 (FY2021), compared to a revenue of US$15.6 million in FY2020. The operating loss of the company was US$73.2 million in FY2021, compared to an operating loss of US$62.6 million in FY2020. The net loss of the company was US$79.6 million in FY2021, compared to a net loss of US$58 million in FY2020. The company reported revenues of US$4.8 million for the third quarter ended September 2022, a decrease of 55.2% over the previous quarter.

Quick View – AAV-AQP1

Report Segments
  • Innovator
Drug Name
  • AAV-AQP1
Administration Pathway
  • Parenteral
Therapeutic Areas
  • Immunology
  • Mouth and Dental Disorders
Key Companies
Highest Development Stage
  • Phase II

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.