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

Inherited Retinal Diseases

Inherited retinal diseases (IRDs), including Retinitis Pigmentosa, Choroideremia, and Stargardt Disease, are genetic disorders causing progressive and severe vision loss.

These conditions are characterized by the degeneration of photoreceptor cells in the retina, each with unique genetic underpinnings. The lack of effective treatments for monogenic IRDs presents a significant unmet medical need for the 2 million+ patients living with these conditions globally.

With advancements in gene sequencing and diagnosing inherited disease, we’re better able to identify patients who could benefit from potential new treatment options. Molecular Photoswitches offer a promising approach to restoring lost vision as they can target retinal ganglion cells, which are often preserved and retain an ability to relay visual information to the brain.

Retinitis Pigmentosa

KIO-301, Kiora’s Molecular Photoswitch, has been in clinical development for the treatment of Retinitis Pigmentosa since late 2022. Retinitis Pigmentosa is a hereditary degenerative disorder affecting the retina’s photoreceptor cells. Characterized by progressive loss of peripheral and night vision, it results from mutations in one of more than 150 genes. This disease affects ~1 in 4,000 individuals globally and more than 100,000 patients in the United States alone. The prevalence, combined with the fact that 50% of patients are not qualified to drive by age 37 and are legally blind by 55 underscores the need for treatment options. Kiora’s development of targeted therapies for RP could meet an urgent need. The condition’s complexity and heterogeneity make treatment challenging, underscoring the need for therapies that can address as many or all of the gene mutations implicated in the disease. In this regard, KIO-301 is ideally suited to be used as a standalone therapy against any and all gene variants as well as potentially in combination therapy should any promising gene therapies emerge.

Choroideremia

KIO-301, Kiora’s Molecular Photoswitch, is targeting Choroideremia, a rare inherited retinal disorder affecting ~1 in 50,000 individuals. Choroideremia is characterized by the progressive degeneration of the choroid, the retina’s photoreceptor cells, and retinal pigment epithelium. Initial symptoms include loss of night vision. The progressive nature of Choroideremia leads to severe vision impairment, often culminating in complete blindness, accentuating the critical demand for effective treatments. A Molecular Photoswitch such as KIO-301 offers a promising approach to treating this condition, with potential applicability across various stages of the disease.

Stargardt Disease

KIO-301, Kiora’s Molecular Photoswitch, is targeting Stargardt Disease, a rare inherited retinal disorder affecting ~1 in 10,000 individuals globally and  30,000 in the U.S. alone. Stargardt is a genetic eye disorder that results from mutations in the ABCA4 or the ELOVL4 gene, which causes damage to the retina’s central region. This ultimately leads to progressive central vision loss and significantly affects daily activities such as reading and driving. The progressive nature of Stargardt disease, often manifesting in youth and leading to legal blindness in many cases, highlights the urgency for effective treatments. Kiora’s development of Molecular Photoswitches like KIO-301, aims to address this critical need for this rare disease.

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