Botaretigene Sparoparvovec is a gene therapy commercialized by MeiraGTx, with a leading Phase III program in Retinitis Pigmentosa (Retinitis). According to Globaldata, it is involved in 4 clinical trials, of which 1 was completed, and 3 are ongoing. GlobalData uses proprietary data and analytics to provide a complete picture of Botaretigene Sparoparvovec’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.
The revenue for Botaretigene Sparoparvovec is expected to reach an annual total of $59 mn by 2026 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.
Botaretigene Sparoparvovec Overview
AAV-RPGR is under development for the treatment of X-linked retinitis pigmentosa (XLRP) in adult and pediatric patients. It is administered through the subretinal route. The therapeutic candidate uses a recombinant adeno-associated virus vector (AAV2) expressing X-linked retinitis pigmentosa GTPase regulator (RPGR). It is a new molecular entity (NME).
MeiraGTx is a gene therapy company that develops treatments for ocular, salivary gland and central nervous system diseases. It is investigating AAV-RPGR program against X-linked retinitis pigmentosa (XLRP); AAV-RPE65 gene therapy to treat RPE65 (retinal pigment epithelium-specific 65 kDa) gene deficiency; AAV-CNGB3 drug for achromatopsia (ACHM); and A006 program targeting wet AMD (age-related macular degeneration). The company is also evaluating AAV-GAD medicine to treat Parkinson’s disease; AAV-UPF1 against amyotrophic lateral sclerosis (ALS); AAV-AQP1 for radiation-induced xerostomia (RIX); and AAV-AQP1 program to treat xerostomia caused due to Sjogren’s syndrome. MeiraGTx is developing riboswitch inducible expression programs targeting ophthalmology and oncology indications, diabetes and metabolic disorders, among others. The company operates in the UK, and the Netherlands. MeiraGTx is headquartered in New York City, New York, the US.
The company reported revenues of (US Dollars) US$37.7 million for the fiscal year ended December 2021 (FY2021), compared to a revenue of US$15.6 million in FY2020. The operating loss of the company was US$73.2 million in FY2021, compared to an operating loss of US$62.6 million in FY2020. The net loss of the company was US$79.6 million in FY2021, compared to a net loss of US$58 million in FY2020. The company reported revenues of US$4.8 million for the third quarter ended September 2022, a decrease of 55.2% over the previous quarter.
For a complete picture of Botaretigene Sparoparvovec’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.