RPA-501 is a gene therapy commercialized by Rocket Pharmaceuticals, with a leading Phase II program in Glycogen Storage Disorders (GSD). According to Globaldata, it is involved in 3 clinical trials, of which 2 are ongoing, and 1 is planned. GlobalData uses proprietary data and analytics to provide a complete picture of RPA-501’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.
The revenue for RPA-501 is expected to reach an annual total of $42 mn by 2037 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.
RPA-501 is under development for the treatment of Danon disease (a glycogen storage disease Type IIb). It is developed based on AAV technology. It comprises of recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene. The drug candidate is administered through intravenous route.
Rocket Pharmaceuticals Overview
Rocket Pharmaceuticals (Rocket Pharma), formerly Inotek Pharmaceuticals Corp is a clinical-stage biopharmaceutical company. It focuses on gene therapies for rare and devastating diseases. The company has three clinical-stage ex vivo lentiviral vector (“LVV”) programs which include programs for Fanconi Anemia (“FA”), a genetic defect in the bone marrow that reduces the production of blood cells or promotes the production of faulty blood cells, Leukocyte Adhesion Deficiency-I (“LAD-I”), a genetic disorder that causes the immune system to malfunction, and Pyruvate Kinase Deficiency (“PKD”), a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. It also has a clinical-stage in vivo adeno-associated virus (“AAV”) program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure. Rocket Pharma is headquartered in Cranbury, New Jersy, the US.
The operating loss of the company was US$224.3 million in FY2022, compared to an operating loss of US$167.3 million in FY2021. The net loss of the company was US$221.9 million in FY2022, compared to a net loss of US$169.1 million in FY2021.
For a complete picture of RPA-501’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.