OCU-400 is under clinical development by Ocugen and currently in Phase II for Leber Congenital Amaurosis (LCA). According to GlobalData, Phase II drugs for Leber Congenital Amaurosis (LCA) have a 60% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how OCU-400’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

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.

For a complete picture of OCU-400’s drug-specific PTSR and LoA scores, buy the report here.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.