Mozafancogene Autotemcel is a gene-modified cell therapy commercialized by Rocket Pharmaceuticals, with a leading Phase II program in Fanconi Anemia. According to Globaldata, it is involved in 8 clinical trials, of which 3 were completed, 4 are ongoing, and 1 is planned. GlobalData uses proprietary data and analytics to provide a complete picture of Mozafancogene Autotemcel’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

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The revenue for Mozafancogene Autotemcel is expected to reach an annual total of $25 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.

Mozafancogene Autotemcel Overview

RPL-102 is under development for the treatment of Fanconi anemia type A. The therapeutic candidate constitutes lentiviral vector carrying FANC-A gene as part of the PGK-FANCA-WPRE expression cassette which includes a phosphoglycerate kinase (PKG) promoter and an optimized woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). It is administered through intravenous route. The therapeutic candidate contains autologous CD34+ enriched cells, these CD34+ cells are mobilized by Granulocyte colony-stimulating factor (G-CSF) and plerixafor.

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$259.7 million in FY2023, compared to an operating loss of US$224.3 million in FY2022. The net loss of the company was US$245.6 million in FY2023, compared to a net loss of US$221.9 million in FY2022.

For a complete picture of Mozafancogene Autotemcel’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.

This content was updated on 22 April 2024

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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.