KOL Webinar on Genetic Blindness Points to Promise of Kiora's Pioneering Solution--Clinical Data in Q1

Retinitis pigmentosa (RP) is a cluster of genetic eye diseases that damage the retina, the light-sensitive layer of the eye. It typically affects people in their teens or 20s, although some variations initiate in children as young as 8.

There is currently no cure for the disease, and it inevitably leads to total blindness.

Stonegate Healthcare recently hosted an event focusing on the disease. “Battling Blindness in Retinitis Pigmentosa: A Discussion of Disease & the Pipeline of Hope”  featured three speakers: Ben Shaberman of the Foundation Fighting Blindness, who provided a patient perspective, retinitis pigmentosa specialist Dr. Christine Kay, an inherited retinal disease specialist and Kiora Pharmaceuticals Inc.’s Chief Development Officer, Dr. Eric Daniels. 

A Simple But Elegant Solution

RP has been a difficult disease for practitioners to tackle, and the U.S. Food and Drug Administration (FDA) has not approved any treatments to date specifically indicated for the condition. In many cases, the disease is diagnosed only after vision loss initiates.

According to Dr. Kay, mutations in more than 65 genes can cause RP and whilst gene therapy approaches offer promise to addressing RP, the FDA would have to essentially approve 65 unique gene therapy treatments to address all the patients with RP. Timing of diagnosis of RP may also present challenges, and for many patients, it may be too late to qualify for gene therapy treatment.  

Kiora Pharmaceuticals Inc. KPRX is a pharmaceutical company specializing in treating eye diseases with high unmet need. During the webinar, Dr. Daniels talked about the company’s novel treatment, KIO-301, which is in Phase 1b clinical trials. The study is expected to generate interim data before the end of this year, with top line results planned for the first quarter of 2023. 

Dr. Daniels describes KIO-301 as a “simple but elegant solution” to RP. KIO-301 does not focus on the photoreceptors in the eye, which die in patients with RP, but instead turns its attention to the retinal ganglion cell (RGC – the cells that comprise the optic nerve). RGCs are still viable and functional in RP patients — it’s the photoreceptors (rods and cones) that are failing. KIO-301 specifically enters the RGCs in the eye and makes these cells light sensitive. 

It is the hope that KIO-301 can restore significant visual functionality in patients with vision loss due to RP. The treatment does not interfere with native functional vision, which also makes it a potentially viable treatment for patients with less-advanced RP. 

RP specialist Dr. Kay is hopeful and excited about Kiora’s clinical trials. 

Patients Have Passion and Urgency For Treatment

Mr. Shaberman provided the patient’s perspective, indicating that people with RP are passionate about finding a treatment to address their blindness and are willing to participate in early stage clinical research of novel therapies to find one. Dr. Daniels said Kiora has a long list of patients from around the world who would like to be part of the company’s clinical trials.

What sets KIO-301 apart from other potential therapies, including gene therapy alternatives, is the ease of treatment. Dr. Kay said she is interested in KIO-301 because it is potentially safe, easy to administer and a reasonable approach to vision restoration. KIO-301 will be administered intravitreally, allowing most any ophthalmologists the capability to provide the treatment with no need for more invasive and expensive surgeries. The risk of infection is extremely low compared to other routes of administrations. 

“For patients with RP, even partial restoration of their vision can be life-changing”, Mr. Shaberman said. “RP is an emotionally burdensome disease for patients and their families. KIO-301 gives hope to patients who are eagerly awaiting a solution to their blindness.”

To watch a recording of the event, click here.

Click here for more information on Kiora Pharmaceuticals

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