PBGM-01 is a gene therapy commercialized by Passage Bio, with a leading Phase II program in GM1 Gangliosidosis (Beta Galactosidase 1 Deficiency). 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 PBGM-01’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.
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The revenue for PBGM-01 is expected to reach an annual total of $2 mn by 2040 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.
PBGM-01 Overview
PBGM-01 is under development for the treatment of rare monogenic central nervous system disorder infantile GM1 gangliosidosis. It comprises the adeno-associated virus vector (AAVhu68) to deliver modified DNA encoding the b-gal enzyme. It acts by targeting GLB1 and administered through intra-cisterna magna injection.
Passage Bio Overview
Passage Bio is a genetic medicines company that develops therapies for the treatment of monogenic central nervous system disorders. The company’s pipeline products include PBGM01 injection to treat GM1 gangliosidosis, PBFT02 targets frontotemporal dementia (FTD), amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease; and PBAL05 for the treatment of neurodegenerative disease. Passage Bio works in collaboration with the University of Pennsylvania and its gene therapy program (GTP) for preclinical works. Its manufacturing and global clinical distribution center is located at Hopewell, New Jersey. Passage Bio is headquartered in Philadelphia, Pennsylvania, the US.
The operating loss of the company was US$108.4 million in FY2023, compared to an operating loss of US$138.4 million in FY2022. The net loss of the company was US$102.1 million in FY2023, compared to a net loss of US$136.1 million in FY2022.
For a complete picture of PBGM-01’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.
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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.
