Vanglusagene ensiparvovec is a gene therapy commercialized by F. Hoffmann-La 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 2036 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.

F. Hoffmann-La Roche Overview

F. Hoffmann-La Roche (Roche) is a biotechnology company that develops drugs and diagnostics to treat major diseases. It provides medicines for the treatment of cancer, other auto-immune diseases, central nervous system disorders, ophthalmological disorders, infectious diseases, and respiratory diseases. The company also offers in vitro diagnostics, tissue-based cancer diagnostics, and diabetes management solutions. Roche conducts research to identify novel methods to prevent, diagnose, and treat diseases. The company offers its products and services to hospitals, healthcare professionals, commercial laboratories, researchers, and pharmacists. Together with its subsidiaries and partners, the company has operations in various countries. Roche is headquartered in Basel, Switzerland.

The company reported revenues of (Swiss Francs) CHF63,281 million for the fiscal year ended December 2022 (FY2022), an increase of 0.8% over FY2021. In FY2022, the company’s operating margin was 27.5%, compared to an operating margin of 28.9% in FY2021. In FY2022, the company recorded a net margin of 19.6%, compared to a net margin of 22.2% in FY2021.

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

This content was updated on 7 February 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.