AMT-130 is a rnai gene therapy commercialized by UniQure, with a leading Phase II program in Huntington Disease. According to Globaldata, it is involved in 2 clinical trials, which are ongoing. GlobalData uses proprietary data and analytics to provide a complete picture of AMT-130’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

The revenue for AMT-130 is expected to reach an annual total of $85 mn by 2037 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.

AMT-130 Overview

Ifezuntirgene inilparvovec (AMT-130) is under development for the treatment of Huntington’s disease. The therapeutic candidate consists of Adeno-associated virus type 5 (AAV5) vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene. It is developed based on ddRNAi (miQURE) technology. DNA-directed RNAi (ddRNAi) involves the use of adeno-associated virus and thereby introduces DNA sequence directly to the nucleus of the cells. It is administered through intravenous route.

UniQure Overview

UniQure develops and markets gene therapy products. The company develops adeno-associated virus (AAV) based gene therapies using its gene technology platform and offers disease-modifying treatments to patients with severe genetic diseases and other devastating diseases. UniQure’s lead product, AMT-060, is a gene therapy for the treatment of moderately-severe hemophilia B. It preclinical products include AMT-130, an AAV5 vector for the treatment of Huntington’s disease and AMT-150 for Spinocerebellar Ataxia type 3. The company develops a gene platform to bring new disease modifying therapies to patients with genetic diseases. The company conducts drug discovery and preclinical research in collaboration with academic research institutions. UniQure is headquartered in Amsterdam, the Netherlands.

The company reported revenues of (US Dollars) US$524 million for the fiscal year ended December 2021 (FY2021), compared to a revenue of US$37.5 million in FY2020. The operating profit of the company was US$310.6 million in FY2021, compared to an operating loss of US$125.4 million in FY2020. The net profit of the company was US$329.6 million in FY2021, compared to a net loss of US$125 million in FY2020. The company reported revenues of US$1.5 million for the third quarter ended September 2022, compared to a revenue of US$0.5 million the previous quarter.

For a complete picture of AMT-130’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.


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.