New Trial Results: Kiora's KIO-301 Demonstrates Meaningful Vision Improvements in Retinitis Pigmentosa

Inflammatory Retinal Disease

Retinal inflammation can be the result of several underlying causes. This includes infection, autoimmune disease, and inherited conditions.

Kiora’s KIO-100 platform is based on a small molecule that can be formulated for a variety of delivery options to reach various tissues locally, such as the retina (back of the eye) or the anterior segment of the eye (front of the eye), as well as systemically. Given how effectively it can be formulated and delivered, it has the potential to treat several disease types. Kiora is focused on developing the family of compounds for retinal applications while leaving promising applications for the treatment of anterior eye segments as well as broader systemic diseases like Multiple Sclerosis or Rheumatoid Arthritis to potential strategic partners.

Posterior Non-Infectious Uveitis

Uveitis is an inflammation of the uvea, the eye’s middle layer, which can lead to serious complications like chronic inflammation, vision impairment, or even vision loss. The effects most commonly present themselves in individuals between the ages of 20 to 50 years old. Causes include autoimmune disorders, infections, or other unknown factors.

Kiora is developing KIO-104, a DHODH inhibitor, for the non-infectious form of Uveitis. Notably, 15% of all cases of legal blindness and visual handicap in the US and Europe are the result of this form of Uveitis. While steroids are often used in patients with this form of Uveitis, many patients are not eligible to use them due to the risk of cataracts or increased intra-ocular pressure.

Clinical proof-of-concept of the potential for the KIO-100 family of compounds has been demonstrated in multiple studies. This includes a first-in-human, open-label, phase-1 clinical trial, which investigated the use of KIO-104 for treating Uveitis, an intraocular inflammatory disease. Results, which were reported in October 2022, showed that a single intravitreal injection of KIO-104 decreased intraocular inflammation in a dose-dependent fashion, and improved visual acuity significantly during the duration of the study. The drug was well tolerated, with no serious side effects on intraocular tissues or other adverse events observed.

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