RGX-111 is a gene therapy commercialized by RegenxBio, with a leading Phase II program in Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ). 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 RGX-111’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

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The revenue for RGX-111 is expected to reach an annual total of $10 mn by 2039 globally 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.

RGX-111 Overview

RGX-111 is under development for the treatment of children and adult subjects with Mucopolysaccharidosis Type I (MPS I). The drug candidate is administered through intracisternal route. The therapeutic candidate delivers  the  alpha-l-iduronidase (IDUA) gene using recombinant adeno-associated virus 9 (AAV9) vector. It is developed based on the NAV technology platform which is a recombinant adeno-associated virus (AAV) gene delivery platform.

RegenxBio Overview

RegenxBio, formerly ReGenX Biosciences LLC, is a clinical-stage biotechnology company involved in the discovery and development of gene therapy. It is focused on the treatment of various retinal, metabolic, and neurodegenerative diseases. The company develops, commercializes, and licenses recombinant adeno-associated virus (AAV) gene therapy. RegenxBio‘s products include AAV plasmids, AAV vector reporter systems, and custom AAV vectors. The company’s gene therapy product candidates deliver genes to cells to address genetic defects and enable cells in the body to produce therapeutic proteins or antibodies that act on the disease. RegenxBio employs its proprietary NAV technology platform, a gene delivery technology, to advance its product candidates. RegenxBio is headquartered in Rockville, Maryland, the US.
The company reported revenues of (US Dollars) US$90.2 million for the fiscal year ended December 2023 (FY2023), a decrease of 19.9% over FY2022. The operating loss of the company was US$268.1 million in FY2023, compared to an operating loss of US$262.9 million in FY2022. The net loss of the company was US$263.5 million in FY2023, compared to a net loss of US$280.3 million in FY2022.

For a complete picture of RGX-111’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.

This content was updated on 20 May 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.