Avalotcagene Ontaparvovec is a gene therapy commercialized by Ultragenyx Pharmaceutical, with a leading Phase III program in Ornithine-Transcarbamylase Deficiency. According to Globaldata, it is involved in 4 clinical trials, of which 2 were completed, and 2 are ongoing. GlobalData uses proprietary data and analytics to provide a complete picture of Avalotcagene Ontaparvovec’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.
The revenue for Avalotcagene Ontaparvovec is expected to reach an annual total of $49 mn by 2037 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.
Avalotcagene Ontaparvovec Overview
DTX-301 is under development for the treatment of ornithine transcarbamylase deficiency (urea cycle disorder). It is administered through intravenous route as a concentrate for a solution. The therapy is an adeno-associated virus 8 (AAV8) gene therapy developed based on NAV vector technology.
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 Avalotcagene Ontaparvovec’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.