GTX-102 is an antisense oligonucleotide commercialized by Ultragenyx Pharmaceutical, with a leading Phase II program in Angelman 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 GTX-102’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

The revenue for GTX-102 is expected to reach an annual total of $63 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.

GTX-102 Overview

GTX-102 is under development for the treatment of Angelman syndrome. The therapeutic candidate is a chimeric locked nucleic acid and ribonucleic-deoxyribonucleic antisense oligonucleotide specific for the human UBE3A-antisense transcript. It is administered through intrathecal route. It is an antisense oligonucleotide which acts by targeting ubiquitin-protein ligase E3A (UBE3A).

Ultragenyx Pharmaceutical Overview

Ultragenyx Pharmaceutical (Ultragenyx) develops novel medicines for rare and ultra-rare genetic diseases. It has three 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). 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.

For a complete picture of GTX-102’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.


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.