Kiora Pharmaceuticals Receives FDA Approval to Start Phase 2 Study KIO-101

Kiora Pharmaceuticals has announced that they have been granted the investigational new drug (IND) application approval for a phase 2 study of KIO-101. This study will evaluate the effectiveness of KIO-101 eye drops in treating the ocular presentation of rheumatoid arthritis and other autoimmune diseases (OPRA+).

The study will enroll about 120 patients and will be conducted at multiple centers in a controlled, randomized, double-masked trial format. The objective of the trial is to assess the safety and efficacy of KIO-101 for patients with autoimmune diseases who have signs and symptoms of ocular surface disease. The study is expected to begin enrolling patients in Australia in the first half of 2023.

"Ocular surface disease is the most common non-joint manifestation of patients living with Rheumatoid Arthritis (RA) as well as other common autoimmune diseases," Eric Daniels, MD, Chief Development Officer of Kiora, said in a company news release.

"While the systemic treatment of underlying autoimmune diseases has advanced dramatically in the past decade, these agents commonly do not address local ocular disease. This unfortunately leaves affected autoimmune patients with few available eye drug options that only offer limited relief in addressing the moderate to severe ocular dryness, pain and discomfort associated with these diseases. KIO-101 is a topical, patient-friendly eye drop with a proven mechanism of action in autoimmune diseases. Proof-of-concept has been established in previous ocular inflammation clinical studies for KIO-101. This drug has the potential to finally close the wide gap of untreated ocular disease in patients with common autoimmune diseases."

A phase 2 study of KIO-101 is planned to be conducted in up to 120 patients with autoimmune diseases and ocular symptoms, such as RA, psoriatic disease, systemic lupus erythematosus, or fibromyalgia. The study will be carried out at multiple centers and will follow a randomized, controlled, double-masked clinical trial design.

Two concentrations of KIO-101, 0.15% and 0.30%, will be evaluated in the trial. The efficacy of the drug will be evaluated by measuring established ocular signs and symptoms, including corneal staining and changes in the Schirmer's test score at 12 weeks. Safety and tolerability will also be monitored during the study.

KIO-101 is a non-steroidal drug belonging to the DHODH inhibitors class, which aims to reduce T-cell proliferation and the release of proinflammatory cytokines. The systemic drugs in this class, which target systemic diseases, generate around $2 billion in annual revenue. KIO-101 has the potential to impact the local immune response in the eye, which is responsible for ocular symptoms in autoimmune diseases. Approximately 3.43 million patients in the U.S. suffer from the ocular manifestations of the autoimmune diseases that KIO-101 is targeting.