KIO-101 is under clinical development by Kiora Pharmaceuticals and currently in Phase I for Retinal Vein Occlusion. According to GlobalData, Phase I drugs for Retinal Vein Occlusion have a 90% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how KIO-101’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.

KIO-101 overview

KIO-101 (PP-001) is under development for the treatment of dry eye in ocular presentation of rheumatoid arthritis (OPRA), conjunctival hyperemia, adenoviral conjunctivitis, retinal vein occlusion, diabetic macular edema and coronavirus disease 2019 (COVID-19). The drug is a biphenyl 4-yl-carbamoylthiophene- 2-carboxylic acid derivative and can be administered through oral, topical and intravitreal route. It is a specific immunomodulating small molecule. It acts by targeting dihydroorotate dehydrogenase (DHODH). It was also under development for the treatment of diabetic retinopathy, intermediate and posterior uveitis.

Kiora Pharmaceuticals overview

Kiora Pharmaceuticals (Kiora), formerly EyeGate Pharmaceuticals, is a clinical-stage specialty pharmaceutical and drug delivery company that develops and commercializes products for treating diseases and disorders of the eye. The company’s pipeline consists of three product candidates, including KIO-101, KIO-301, and KIO-201. Its lead product, KIO-301, is a potential vision-restoring small molecule that is designed to restore vision in patients with inherited and age-related degenerative retinal diseases. KIO-101 is a non-steroidal, immuno-modulatory, small-molecule inhibitor of Dihydroorotate Dehydrogenase (DHODH) that focuses on patients with Ocular Presentation of Rheumatoid Arthritis (OPRA). Its KIO-201 is a synthetic modified hyaluronic acid (HA) capable of coating the ocular surface and designed to resist degradation under conditions present in the eye. Kiora is headquartered in Salt lake city, Utah, the US.

For a complete picture of KIO-101’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.