Sepiapterin is a small molecule commercialized by PTC Therapeutics, with a leading Pre-Registration program in Phenylketonuria (PKU). According to Globaldata, it is involved in 7 clinical trials, of which 4 were completed, and 3 are ongoing. GlobalData uses proprietary data and analytics to provide a complete picture of Sepiapterin’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.
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The revenue for Sepiapterin is expected to reach an annual total of $309 mn by 2034 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.
Sepiapterin Overview
Sepiapterin (PTC-923 (CNSA-001)) is under development for the treatment of moderate to severe diabetic gastroparesis, tetrahydrobiopterin (BH4) deficiency resulting in hyperphenylalaninemia and phenylketonuria, segawa disease, central nervous system disorders and unspecified indications. The drug candidate is a tetrahydrobiopterin derivative. It is a small molecule administered through oral route. The drug candidate acts by targeting tetrahydrobiopterin.
it was also under development for the treatment of Parkinson's disease.
PTC Therapeutics Overview
PTC Therapeutics discovers, develops, and markets medicines for the treatment of rare disorders. Its marketed products include Evrysdi (risdiplam) for spinal muscular atrophy, Translarna (ataluren), and Emflaza (deflazacort) for the treatment of Duchenne muscular dystrophy. The company’s pipeline candidates target the treatment of Huntington’s disease; children with inherited mitochondrial disease; leiomyosarcoma; acute myelogenous leukemia; and aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics works in collaboration with various biotechnology and pharmaceutical companies for advancing its pipeline candidates. It operates through its subsidiaries in the US and Europe. PTC Therapeutics is headquartered in South Plainfield, New Jersey, the US.
The company reported revenues of (US Dollars) US$937.8 million for the fiscal year ended December 2023 (FY2023), an increase of 34.2% over FY2022. The operating loss of the company was US$577.1 million in FY2023, compared to an operating loss of US$447.4 million in FY2022. The net loss of the company was US$626.6 million in FY2023, compared to a net loss of US$559 million in FY2022.
The company reported revenues of US$186.7 million for the second quarter ended June 2024, a decrease of 11.1% over the previous quarter.
For a complete picture of Sepiapterin’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.
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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.
