Drugs / KIO-100
last change Dec 2025 re-read 3 minutes ago

KIO-100

also known as PP-001

Small molecule targets DHODH via inhibition

Developed for
macular retinal edema · uveitis · eye adnexa disorder
Investigated by
Panoptes Pharma GmbH · Kiora Pharmaceuticals, Inc.

Trials 3

20172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT03769454 Nov 2018 → Sep 2021 keratoconjunctivitis Panoptes Pharma GmbH Completed No outcome recorded
Phase 1/21 trial
Phase 1/2 NCT03634475 Jan 2017 → Nov 2018 uveitis Panoptes Pharma GmbH Completed No outcome recorded
Phase 21 trial
Phase 2 NCT06825702 May 2025 → Nov 2026 expected macular retinal edema Kiora Pharmaceuticals, Inc. Recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 2

DateIssuerRelease
2023-04-26 Kiora Pharmaceuticals, Inc. Results Kiora Pharmaceuticals' KIO-201 Heals Wounds in Phase 2 Trial of Patients with Persistent Corneal Epithelial Defects kiorapharma.com ↗
The results of this clinical trial demonstrate that an intravitreal KIO-100 formulation is safe and has the potential to be further developed as a new intraocular therapy for autoimmune uveitis and CME.
2022-10-17 Kiora Pharmaceuticals, Inc. Results Kiora Pharmaceuticals Announces Publication of Phase 1 Trial Results for KIO-100 as a Treatment for Ocular Inflammatory Disease kiorapharma.com ↗
Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX), ("Kiora" or the "Company") announced that 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.

Evidence & citations 6 cited values

Every value below carries the sentence it was read from. 4 sources stand behind the page.

FieldValueCited text
Known as KIO-100 “KIO-104 is an ophthalmic formulation for IVT injection of the active pharmaceutical ingredient (API) KIO-100, a novel, non-steroidal, small molecule inhibitor of dihydroorotate...” NCT06825702 ↗
1

“A new small molecule DHODH-inhibitor [KIO-100 (PP-001)]” PMID 36325389 ↗ Oct 2022

Known as PP-001 “A new small molecule DHODH-inhibitor [KIO-100 (PP-001)]” PMID 36325389 ↗ Oct 2022
1

NCT03769454 ↗

Action Inhibit “PP-001 is a novel small molecule inhibitor of Dihydroorotate Dehydrogenase (DHODH)” NCT03769454 ↗
Modality Small molecule “PP-001 is a novel, anti-inflammatory small molecule” NCT03769454 ↗
1

CHEMBL4580266 ↗

Route Topical “PP-001 eye drops” NCT03769454 ↗
Target DHODH “PP-001 is a novel small molecule inhibitor of Dihydroorotate Dehydrogenase (DHODH)” NCT03769454 ↗