A Safety Study of Intravitreal PP-001 in Patients With Chronic, Non-Infectious Uveitis

Summary

In this study, PP-001 is assessed for safety and efficacy in patients diagnosed with non-infectious chronic uveitis. PP-001 is a novel small molecule that inhibits a specific enzyme (Dihydroorotate Dehydrogenase) and has shown pre-clinical efficacy in treatment of non-infectious uveitis. PP-001 will be administered to participants as a single intravitreal injection in ascending doses.

Timeline

Start

2017-01

Primary completion

2018-11

Completion

2019-04

Outcome

Results reported · no primary-endpoint met/missed verdict (typical of a dose-finding / safety study)

release “results from a Phase 1 study of KIO-100 (formerly PP-001) demonstrate its potential as a treatment for non-infectious uveitis, an ophthalmic inflammatory disease.” kiorapharma.com ↗

release “Intravitreal KIO-100 is safe and tolerable with the potential to treat patients with autoimmune posterior uveitis and Cystoid Macular Edema (CME).” kiorapharma.com ↗

Publications

• Thurau S, Deuter CME, Heiligenhaus A, Pleyer U, Van Calster J, Barisani-Asenbauer T, Obermayr F, Sperl S, Seda-Zehetner R, Wildner G. A new small molecule DHODH-inhibitor [KIO-100 (PP-001)] targeting activated T cells for intraocular treatment of uveitis - A phase I clinical trial. Front Med (Lausanne). 2022 Oct 17;9:1023224. doi: 10.3389/fmed.2022.1023224. eCollection 2022.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	KIO-100	Small molecule	—	Intravitreal

Indications