PR-001 is a gene therapy commercialized by Eli Lilly and Co, with a leading Phase II program in Gaucher Disease Type I. According to Globaldata, it is involved in 3 clinical trials, which are ongoing. GlobalData uses proprietary data and analytics to provide a complete picture of PR-001’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

The revenue for PR-001 is expected to reach an annual total of $52 mn by 2038 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.

PR-001 Overview

PR-001 is under development for the treatment of Parkinson's disease with GBA1 mutation and neuronopathic Gaucher disease type II and Gaucher disease type I. The therapeutic candidate is administered intracisternally and intravenously. The drug candidate consists of AAV9 viral vector delivering the GBA1 gene which encodes glucocerebrosidase. It is developed based on NAV technology platform. NAV technology uses recombinant adeno-associated virus (rAAV) vectors for the delivery of new genetic materials to cells.

It was also under development for the treatment of Gaucher disease type III.

Eli Lilly and Co Overview

Eli Lilly and Co (Lilly) is a healthcare company that is engaged in the discovery, development, and marketing of human healthcare products. The company offers medicines for cardiovascular conditions, diabetes, endocrinology, cancer, neurological problems, immune disorders, men’s health and musculoskeletal problems. The company distributes its pharmaceutical health products through independent wholesale distributors. Lilly conducts research and development activities to discover and deliver innovative medicines. It also promotes products through sales representatives and marketing agreements with other pharmaceutical companies. The company also offers its products in North America, South America, Europe, South Asia, the Middle East, Africa and North Asia-Pacific. Lilly is headquartered in Indianapolis, Indiana, the US.

The company reported revenues of (US Dollars) US$28,541.4 million for the fiscal year ended December 2022 (FY2022), an increase of 0.8% over FY2021. The operating profit of the company was US$7,127.3 million in FY2022, compared to an operating profit of US$5,951.9 million in FY2021. The net profit of the company was US$6,244.8 million in FY2022, compared to a net profit of US$5,581.7 million in FY2021.

For a complete picture of PR-001’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.

GlobalData

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.