The revenue for UX-701 is expected to reach an annual total of $606 mn by 2037 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.

UX-701 Overview

UX-701 (DTX-701) is under development for the treatment of Wilson disease. The therapeutic candidate constitutes adeno-associated virus (AAV) vector encoding for copper transporter P-type ATPase, ATP7B. It is developed based on next-generation adeno-associated virus (AAV) platform. It is administered through intravenous route.

Ultragenyx Pharmaceutical Overview

Ultragenyx Pharmaceutical (Ultragenyx) is a biotechnology company that develops novel medicines for rare and ultra-rare genetic diseases. It has four marketed products, Mepsevii (vestronidase alfa) for the treatment of Mucopolysaccharidosis (Sly syndrome); Dojolvi (triheptanoin), a highly purified, synthetic, 7-carbon fatty acid triglyceride for long-chain fatty acid oxidation disorders (LC-FAOD) and Crysvita (burosumab), a fully human monoclonal antibody for the treatment of X-linked hypophosphatemia (XLH); Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). Its pipeline includes DTX401 for the treatment of glycogen storage disease type Ia, or GSDIa; UX701 for Wilson disease; UX053 for glycogen storage disease type III; and GTX-102 for Angelman syndrome in partnership with GeneTx. It works in partnership with various academic institutes and biotechnology companies to advance its product candidates. Ultragenyx is headquartered in Novato, California, the US.

The company reported revenues of (US Dollars) US$363.3 million for the fiscal year ended December 2022 (FY2022), an increase of 3.4% over FY2021. The operating loss of the company was US$648.9 million in FY2022, compared to an operating loss of US$381.7 million in FY2021. The net loss of the company was US$707.4 million in FY2022, compared to a net loss of US$454 million in FY2021. The company reported revenues of US$100.5 million for the first quarter ended March 2023, a decrease of 2.8% over the previous quarter.

For a complete picture of UX-701’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.

This content was updated on 2 September 2023

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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.