Ulefnersen Sodium is an antisense oligonucleotide commercialized by Ionis Pharmaceuticals, with a leading Phase III program in Amyotrophic Lateral Sclerosis. 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 Ulefnersen Sodium’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.
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The revenue for Ulefnersen Sodium is expected to reach an annual total of $16 mn by 2036 globally 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.
Ulefnersen Sodium Overview
ION363 is under development for the treatment of amyotrophic lateral sclerosis (ALS) in patients with mutations in the Fused in Sarcoma (FUS) gene. It is a generation 2 antisense drug that prevents the production of the FUS protein. It is administered through intrathecal bolus route.
Ionis Pharmaceuticals Overview
Ionis Pharmaceuticals (Ionis), formerly Isis Pharmaceuticals, focuses on the discovery and development of RNA-targeted drugs. The company develops medicines to treat various diseases including cancer, pulmonary, cardiovascular diseases, neurological and infectious diseases. Its major products include Spinraza for spinal muscular atrophy, Tegsedi for neuropathy, Waylivra for familial chylomicronemia syndrome, Qalsody for superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS), and Wainua for polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN). Ionis also has a pipeline of products in different phases of clinical trials. It develops products based on its proprietary antisense technology. The company partnered with other pharmaceutical companies to develop a range of products in different therapeutic areas. The company sells its products in North America and Europe. Ionis is headquartered in Carlsbad, California, the US.
The company reported revenues of (US Dollars) US$787.7 million for the fiscal year ended December 2023 (FY2023), an increase of 34.1% over FY2022. The operating loss of the company was US$340.3 million in FY2023, compared to an operating loss of US$417.9 million in FY2022. The net loss of the company was US$366.3 million in FY2023, compared to a net loss of US$269.7 million in FY2022.
The company reported revenues of US$225.3 million for the second quarter ended June 2024, an increase of 88.5% over the previous quarter.
For a complete picture of Ulefnersen Sodium’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.
