Kiora Pharmaceuticals (KPRX) Announces Publication of Phase 1 Trial Results for KIO-100
Enter Wall Street with StreetInsider Premium. Claim your one week free trial here.
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-invasive uveitis. infectious, inflammatory ophthalmic disease. The results were published today in an article titled “A new small molecule DHODH inhibitor [KIO-100 (PP-001)] targeting activated T cells for the intraocular treatment of uveitis – a phase I clinical trial“, in the review Frontiers in medicine.
This first-in-man, open-label, Phase 1 clinical trial investigated the use of KIO-100 for the treatment of uveitis, a T-cell mediated intraocular inflammatory disease. Results showed that a single intravitreal injection of KIO-100 decreased intraocular inflammation in a dose-dependent manner and significantly improved visual acuity over the duration of the study. The drug was well tolerated, with no serious intraocular tissue side effects or other adverse events observed.
KIO-100 is an inhibitor of dihydroorotate dehydrogenase (DHODH), a validated drug target in the treatment of systemic autoimmune diseases. As a third-generation DHODH inhibitor with an intravitreal formulation and high potency to suppress T and B cells, KIO-100 is a promising nonsteroidal anti-inflammatory agent.
“The publication of these data is clinical proof of concept for the use of KIO-100 as a safe and effective nonsteroidal immune modulator for a wide range of inflammatory eye diseases,” said Eric Daniels, MD, chief development officer of Kiora. . “We look forward to building on these promising results in future clinical studies.”
Study design and results
Twelve adult patients with noninfectious chronic bilateral intermediate, posterior, or panuterine uveitis were recruited and divided into three groups (n=4/group) to receive a single intravitreal injection in the most severely affected eye of 100 µl containing 0.3 µg, 0.6 µg or 1.2 µg KIO-100. All patients were tested at baseline and on days 2, 7, 14, 21 and 28 post injection. The drug was safe and well tolerated, with a transient increase in intraocular pressure reported in one eye. Consistent with intravitreal injections, mild conjunctival hemorrhages or conjunctival hyperemia were reported at the injection site in all treated eyes. All reported adverse events were considered unrelated to the drug.
Visual acuity was assessed using ETDRS (Early Treatment Diabetic Retinopathy Study) charts, and intraocular inflammation was assessed by measuring anterior chamber (AC) cells and vitreous veil. Reported results demonstrated a dose-dependent improvement in visual acuity, with the highest dose (1.2 µg) achieving three lines of improvement (equivalent to a doubling of resolution) at day 14 and remaining stable until at day 28. This improvement was statistically significant (p
According to Stephan Thurau, MD, Principal Investigator and Professor of Ophthalmology, University Hospital, LMU München, Germany, “Given the known and very real limitations of chronic intraocular steroid use, KIO-100 represents the future of inflammation in the eye. This data is both encouraging and exciting for patients with uveitis.”
Kiora previously tested the same inhibitor formulated as eye drops, KIO-101, in a Phase 1 clinical trial in healthy subjects and patients diagnosed with ocular surface inflammation. The results also demonstrated the favorable safety and tolerability of KIO-101 as well as statistically significant improvements in conjunctival hyperemia. Favorable KIO-100 results further validate the development of KIO-101 as the Company pursues a program for the treatment of patients with ocular presentations of rheumatoid arthritis (OPRA).
