OCU-400 is a gene therapy commercialized by Ocugen, with a leading Phase II program in Retinitis Pigmentosa (Retinitis) and Leber Congenital Amaurosis (LCA). According to Globaldata, it is involved in 2 clinical trials, of which 1 is ongoing, and 1 is planned. GlobalData uses proprietary data and analytics to provide a complete picture of OCU-400’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

The revenue for OCU-400 is expected to reach an annual total of $129 mn by 2038 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.

OCU-400 Overview

OCU-400 is under development for the treatment of inherited degenerative retinal diseases (IRDs) such as NR2E3, CEP290, PDE6B and RHO mutation-associated recessive retinal degeneration, Leber Congenital Amaurosis (LCA), Bardet-Biedl Syndrome (BBS), enhanced S-cone syndrome and rhodopsin mutations associated retinitis pigmentosa and other retinal degenerations. It is administered as sub-retinal injection. The therapeutic candidate is an adeno-associated virus serotype 5 capsid containing the gene for human nuclear hormone receptor NR2E3. It is developed based on modifier gene therapy platform.

Ocugen Overview

Ocugen, formerly Histogenics, is a biopharmaceutical company that discovers, develops, and commercializes drugs for the treatment of eye diseases. It is investigating OCU-400 against NR2E3, RHO, CEP290, and PDE6B mutation-associated retinal degeneration; and OCU-410 targeting dry age-related macular degeneration. The company is developing OCU200 drugs to treat diabetic macular edema, diabetic retinopathy, and wet-age-related macular degeneration. Ocugen is also evaluating COVAXIN in partnership with Bharat Biotech, for the prevention of COVID-19. It utilizes a modified gene therapy platform to develop its products. The company has an operational presence in Ireland and the US. Ocugen is headquartered in Malvern, Pennsylvania, the US.

The operating loss of the company was US$57.6 million in FY2021, compared to an operating loss of US$21.3 million in FY2020. The net loss of the company was US$58.4 million in FY2021, compared to a net loss of US$21.8 million in FY2020.

For a complete picture of OCU-400’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.


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.