Vanglusagene ensiparvovec is a gene therapy commercialized by Roche, with a leading Phase II program in Pompe Disease. 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 Vanglusagene ensiparvovec’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

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The revenue for Vanglusagene ensiparvovec is expected to reach an annual total of $41 mn by 2038 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.

Vanglusagene ensiparvovec Overview

SPK-3006 is under development for the treatment of Pompe diseases. It is administered through intravenous route. The drug candidate comprises of recombinant Adeno-associated virus (AAV) vector that contains a bio-engineered capsid (AAV-Spark100) and a codon-optimized expression cassette to drive expression of a secretable form of human acid a-glucosidase (GAA). The drug candidate acts by targeting GAA.

Roche Overview

Roche is a holding company that is involved in the business of offering oncology, immunology, infectious diseases, ophthalmology and neuroscience research services. The company is headquartered in Basel, Basel-Stadt, Switzerland.
The company reported revenues of (Swiss Francs) CHF58,716 million for the fiscal year ended December 2023 (FY2023), a decrease of 7.2% over FY2022. In FY2023, the company’s operating margin was 25.8%, compared to an operating margin of 27.5% in FY2022. In FY2023, the company recorded a net margin of 19.6%, compared to a net margin of 19.6% in FY2022.

For a complete picture of Vanglusagene ensiparvovec’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.