Kiora publishes results from Phase 1 study demonstrating safety, tolerability and efficacy of KIO-101

KIO-101 is the designation for topical delivery and KIO-104 is the designation for intravitreal injection. Image credit: ©MYDAYcontent – stock.adobe.com

Kiora Pharmaceuticals Inc announced the publication of results from a Phase 1 double-masked study of KIO-101, a non-steroidal anti-inflammatory agent.

According to the company, KIO-101 contains the same active molecule as KIO-104, which belongs to a family of potent anti-inflammatory agents. The study showed that a 12-day treatment of KIO-101 topically at multiple doses was well tolerated in healthy volunteers and patients with inflammation of the eye.

The company noted in a news release¹ there was a significant decrease in conjunctival hyperemia in the treatment group compared to the placebo group. Conjunctival hyperemia, a sign of ocular inflammation, is due to a variety of non-infectious and infectious causes.

The paper was published in Pharmaceutics.²

Eric Daniels, MD, chief development officer of Kiora, pointed out in the news release the results support the therapeutic potential of our molecule to address inflammation in the eye.

“A Phase 2 trial of KIO-104 is expected to start later this year to treat posterior non-infectious uveitis, a rare inflammatory condition of the retina,” he said in the release. “Because uveitis can lead to vision loss, there’s an important need for new treatment options beyond chronic steroid injections or systemic autoimmune disease drugs currently being used today. Beyond uveitis, we see an opportunity to target additional retinal inflammatory diseases that could similarly benefit from a non-steroidal therapeutic option.”

Kiora’s KIO-100 family of compounds contain the same proprietary active molecule but are uniquely formulated for a specific condition and region of the eye being targeted. KIO-101 is the designation for topical delivery and KIO-104 is the designation for intravitreal injection (directly into the eye). Kiora’s active molecule belongs to a class of drugs known as DHODH inhibitors, including teriflunomide, an FDA-approved drug that has been prescribed to hundreds of thousands of patients with multiple sclerosis.¹

According to the news release, DHODH inhibitors are believed to reduce the production of T-cells, a type of immune cell that, in certain conditions like autoimmune disease, can cause damaging inflammation. Delivering KIO-101 or KIO-104 locally in the eye aims to reduce T-cell-related inflammation without the associated potential side effects of systemic anti-inflammatory drugs.

Moreover, the study involved a double-masked, placebo-controlled, randomised, parallel-group design consisting of two parts. In Part I, 24 healthy volunteers received single or multiple administrations of KIO-101 eye drops in ascending doses (0.05%, 0.15% or 0.30%). Part II involved 21 patients with conjunctival hyperemia who received either 0.15% KIO-101 eye drops or vehicle (2:1) twice daily for 12 consecutive days.

The findings include the following:

• KIO-101 eye drops were well tolerated in all subjects.
• There were no serious adverse events. All adverse events were transient and considered mild to moderate.
• In the highest dose cohort (0.30%), epistaxis occurred in two subjects after multiple instillations.
• In Part II, after 12 days of treatment with 0.15% KIO-101, conjunctival hyperemia decreased by 1.1 ± 0.27 points in the KIO-101 treated group versus 0.6 ± 0.79 points in the placebo group (p = 0.0385).
• From baseline to day 12, the Ocular Surface Disease Index Questionnaire (OSDI) score decreased by 20.3 points in the treatment group, while in the placebo group decreased by 14.0 points (p=ns).

According to the news release, Kiora is planning a Phase 2 clinical trial of KIO-104 to be administered by intravitreal injection directly into the retina for the treatment of posterior non-infectious uveitis.

In a separate study, the company reported positive results from a Phase 1 study of KIO-104 for treatment of posterior non-infectious uveitis.³

Posterior non-infectious uveitis is a rare autoimmune disease characterised by inflammation of the retina, choroid, vitreous or optic nerve, and can result in severe vision loss. It can result from many underlying causes, which may include the ocular manifestation of certain autoimmune diseases.

References:

1. Kiora Announces Publication of Phase 1 Study Demonstrating Safety, Tolerability and Anti-Inflammatory Activity of KIO-101 in the Treatment of Inflammation of the Eye. Kiora Pharmaceuticals, Inc. Published March 8, 2024. Accessed March 8, 2024. https://ir.kiorapharma.com/news-events/press-releases/detail/207/kiora-announces-publication-of-phase-1-study-demonstrating

2. Schmidl D, Hommer N, Kallab M, et al. Safety and Tolerability of KIO-101 Eye Drops in Healthy Volunteers and Patients with Ocular Surface Disease—A Phase I Study. Pharmaceutics. 2024;16(3):367-367. doi:https://doi.org/10.3390/pharmaceutics16030367

3. ‌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. PMID: 36325389; PMCID: PMC9621317.