Leniolisib is a small molecule commercialized by Pharming Group, with a leading Pre-Registration program in PASLI Disease (Activated PI3K Delta Syndrome (APDS)). According to Globaldata, it is involved in 7 clinical trials, of which 3 were completed, 1 is ongoing, and 3 are planned. GlobalData uses proprietary data and analytics to provide a complete picture of Leniolisib’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

The revenue for Leniolisib is expected to reach an annual total of $87 mn by 2032 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.

Leniolisib Overview

Leniolisib (CDZ-173) is under development for the treatment of PASLI Disease (Activated PI3K Delta Syndrome (APDS), p110delta-activating mutation causing senescent T cells, lymphadenopathy. It is administered orally.  It acts on Phosphoinositide 3-kinase delta (PI3K Delta). It was under development for the treatment of primary Sjogren's syndrome.

Pharming Group Overview

Pharming Group (Pharming) develops precision medicines and innovative protein replacement therapies for the treatment of rare diseases and unmet medical needs. The company’s marketed product includes, Ruconest (conestat alfa), a recombinant human C1 inhibitor is used for the treatment of acute hereditary angioedema (HAE) and has been approved in the US, the UK, Europe, Israel, and South Korea. Its product pipeline consists of rhC1INH for the treatment of pre-eclampsia, COVID-19 and acute kidney injury; leniolisib for treatment of activated phosphoinositide 3-kinase delta syndrome (APDS); α-Glucosidase for the treatment of Pompe and Fabry’s disease. The company markets its products through own sales force and network of distributors and partners. Pharming is headquartered in Leiden, the Netherlands.

The company reported revenues of (US Dollars) US$198.9 million for the fiscal year ended December 2021 (FY2021), a decrease of 6.3% over FY2020. In FY2021, the company’s operating margin was 6.8%, compared to an operating margin of 35.9% in FY2020. In FY2021, the company recorded a net margin of 8%, compared to a net margin of 17.8% in FY2020. The company reported revenues of US$54.2 million for the third quarter ended September 2022, an increase of 8.2% over the previous quarter.

For a complete picture of Leniolisib’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.