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 1 clinical trial, which is 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 $6 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.
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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 for addressing 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$112.7 million for the fiscal year ended December 2022 (FY2022), a decrease of 76% over FY2021. The operating loss of the company was US$262.9 million in FY2022, compared to an operating profit of US$160 million in FY2021. The net loss of the company was US$280.3 million in FY2022, compared to a net profit of US$127.8 million in FY2021.
The company reported revenues of US$19.1 million for the first quarter ended March 2023, a decrease of 38.9% over the previous quarter.
For a complete picture of RGX-111’s valuation, buy the drug’s 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.
