AAV-AQP1 is a gene therapy commercialized by MeiraGTx, with a leading Phase II program in Xerostomia. According to Globaldata, it is involved in 5 clinical trials, of which 2 were completed, and 3 are ongoing. GlobalData uses proprietary data and analytics to provide a complete picture of AAV-AQP1’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

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The revenue for AAV-AQP1 is expected to reach an annual total of $6 mn by 2039 in the US based off GlobalData’s Expiry Model. The drug’s revenue forecasts along with estimated costs are used to measure the value of an investment opportunity in that drug, otherwise known as net present value (NPV). Applying the drug’s phase transition success rate to remaining R&D costs and likelihood of approval (LoA) to sales related costs provides a risk-adjusted NPV model (rNPV). The rNPV model is a more conservative valuation measure that accounts for the risk of a drug in clinical development failing to progress.

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 through intraductal route. 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$15.9 million for the fiscal year ended December 2022 (FY2022), a decrease of 57.8% over FY2021. The operating loss of the company was US$116.4 million in FY2022, compared to an operating loss of US$72.8 million in FY2021. The net loss of the company was US$129.6 million in FY2022, compared to a net loss of US$79.6 million in FY2021.

For a complete picture of AAV-AQP1’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.

This content was updated on 18 March 2024

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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, drug margins and company expenses. 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 valuation, 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.