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

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

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.

This study assesses safety and efficacy of PP-001 after intravitreal injection in patients diagnosed with non-infectious chronic uveitis. PP-001 is a novel small molecule inhibitor of Dihydroorotate Dehydrogenase (DHODH) and has shown pre-clinical efficacy in treatment of non-infectious uveitis. PP-001 is to offer a local treatment alternative to corticosteroids with the objective to reduce or possibly replace steroid intake of non-infectious uveitis patients. In the current study, PP-001 will be injected in ascending doses into the vitreous body of the eye of study participants who suffer from non-infectious uveitis. Participants will be monitored for safety and efficacy evaluation of the study drug.

To assess safety and tolerability of ascending doses of PP-001 in participants with chronic, non-infectious uveitis when administered as single intravitreal injection

Evaluation of improvement of ocular inflammation between baseline and follow-up visits using slit-lamp examinations

To assess the improvement of inflammation using slit lamp examinations in participants with chronic, non-infectious uveitis after ascending doses of PP-001 when administered as single intravitreal injection

Evaluation of improvement of ocular inflammation between baseline and follow-up visits using Optical Coherence Tomography (OCT) examinations

To assess the improvement of inflammation using OCT examinations in participants with chronic, non-infectious uveitis after ascending doses of PP-001 when administered as single intravitreal injection

To evaluate the pharmacokinetics (Cmax) in participants with chronic, non-infectious uveitis after ascending doses of PP-001 when administered as single intravitreal injection

To evaluate the pharmacokinetics (AUC) in participants with chronic, non-infectious uveitis after ascending doses of PP-001 when administered as single intravitreal injection

Inclusion Criteria: Male or female patients at the age of 18 years or older who have diagnosis of chronic, posterior uveitis, intermediate uveitis or panuveitis Exclusion Criteria: Patients receiving specific medication/interventions as specified per protocol Pregnant or nursing patients

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.