AAV-AQP1 is a gene therapy commercialized by MeiraGTx, with a leading Phase II program in Sicca Syndrome (Sjogren). According to Globaldata, it is involved in 6 clinical trials, of which 2 were completed, and 4 are ongoing. GlobalData uses proprietary data and analytics to provide a complete picture of AAV-AQP1s 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 $16 mn by 2037 in the US based off GlobalDatas Expiry Model. The drugs 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 drugs 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. The companys product pipeline includes AAV-RPGR, AAV-RPE65, AAV-CNGB3, A006, AAV-GAD, AAV-UPF1 and AAV-AQP1. Its pipeline treats X-linked retinitis pigmentosa, gene deficiency, achromatopsia, macular degeneration, parkinsons disease, amyotrophic lateral sclerosis, radiation-induced xerostomia and ophthalmology diseases. MeiraGTx is developing riboswitch inducible expression programs targeting ophthalmology and oncology indications, diabetes and metabolic disorders among others. The company operates in the UK, the Netherlands and the US. MeiraGTx is headquartered in New York City, New York, the US.
The company reported revenues of (US Dollars) US$14 million for the fiscal year ended December 2023 (FY2023), a decrease of 12% over FY2022. The operating loss of the company was US$137.1 million in FY2023, compared to an operating loss of US$116.4 million in FY2022. The net loss of the company was US$84 million in FY2023, compared to a net loss of US$129.6 million in FY2022.
The company reported revenues of US$0.7 million for the first quarter ended March 2024, a decrease of 65.8% over the previous quarter.
For a complete picture of AAV-AQP1s valuation, buy the drugs risk-adjusted NPV model (rNPV) here.
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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.
