Exagamglogene Autotemcel is a gene-modified cell therapy commercialized by Vertex Pharmaceuticals, with a leading Pre-Registration program in Sickle Cell Disease. According to Globaldata, it is involved in 7 clinical trials, of which 6 are ongoing, and 1 is planned. GlobalData uses proprietary data and analytics to provide a complete picture of Exagamglogene Autotemcel’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

The revenue for Exagamglogene Autotemcel is expected to reach an annual total of $581 mn by 2035 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.

Exagamglogene Autotemcel Overview

Exagamglogene autotemcel (CTX-001) is under development for the treatment of sickle cell disease and beta-thalassemia. The therapeutic candidate constitutes of ex vivo corrected CD34 positive hematopoietic stem and progenitor cells (HSPCs). It is administered through the intravenous route and is developed based on CRISPR/Cas9 gene-editing technology.

Vertex Pharmaceuticals Overview

Vertex Pharmaceuticals (Vertex) is a biotechnology company that discovers, develops and commercializes transformative drugs for the treatment of serious and life-threatening diseases. The company’s product portfolio includes Trikafta, Symdeko/Symkevi, Orkambi (ivacaftor/lumacaftor), and Kalydeco (ivacaftor) for the treatment of cystic fibrosis. Through its research and development efforts, the company focuses on cystic fibrosis, sickle cell diseases, beta thalassemia, type-1 diabetes and pain. It uses state-of-the-art technology platforms to discover new disease targets, compounds, delivery mechanisms, and treatment modalities. Vertex has R&D centers and commercial offices in North America, South America, Europe, and Australia. The company offers its products in the US, Europe, Australia, and Canada. Vertex is headquartered in Boston, Massachusetts, the US.

The company reported revenues of (US Dollars) US$8,930.7 million for the fiscal year ended December 2022 (FY2022), an increase of 17.9% over FY2021. In FY2022, the company’s operating margin was 48.2%, compared to an operating margin of 36.7% in FY2021. In FY2022, the company recorded a net margin of 37.2%, compared to a net margin of 30.9% in FY2021. The company reported revenues of US$2,374.8 million for the first quarter ended March 2023, an increase of 3.1% over the previous quarter.

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

This content was updated on 2 September 2023

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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.