Patidistrogene bexoparvovec is a gene therapy commercialized by Sarepta Therapeutics, with a leading Phase II program in Limb-Girdle Muscular Dystrophy. According to Globaldata, it is involved in 2 clinical trials, which were completed. GlobalData uses proprietary data and analytics to provide a complete picture of Patidistrogene bexoparvovec’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.
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The revenue for Patidistrogene bexoparvovec is expected to reach an annual total of $17 mn by 2037 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.
Patidistrogene bexoparvovec Overview
patidistrogene bexoparvovec (SRP-9004) is under development for the treatment of limb-girdle muscular dystrophy type 2D (LGMD2D). It is administered through injection. The therapeutic candidate comprises self-complementary recombinant adeno-associated virus serotype rhesus 74 (AAVrh.74) encoding alpha-sarcoglycan.
Sarepta Therapeutics Overview
Sarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. The company’s platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. Its commercial products include Exondys 51, Vyondys 53 and Amondys 45 indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene. Its pipeline product includes SRP-9001, SRP-5051, SRP-9003 and SRP-5045 indicated for the treatment of DMD, limb-girdle muscular dystrophies (LGDMs) and other neuromuscular and central nervous system disorders. Sarepta is headquartered in Cambridge, Massachusetts, the US.
The company reported revenues of (US Dollars) US$1,243.3 million for the fiscal year ended December 2023 (FY2023), an increase of 33.3% over FY2022. The operating loss of the company was US$583.5 million in FY2023, compared to an operating loss of US$664.2 million in FY2022. The net loss of the company was US$536 million in FY2023, compared to a net loss of US$703.5 million in FY2022.
For a complete picture of Patidistrogene bexoparvovec’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.
