Kiora Pharmaceuticals Granted Orphan Drug Designation for KIO-301, an Investigational Drug for the Treatment of Retinitis Pigmentosa

Kiora Pharmaceuticals Granted Orphan Drug Designation for KIO-301, an Investigational Drug for the Treatment of Retinitis Pigmentosa

March 21, 2022

Kiora Pharmaceuticals has received orphan drug designation from the FDA for its investigational treatment of retinitis pigmentosa, KIO-301. Kiora expects to initiate clinical trials of KIO-301 in Q3 2022.

Orphan drug designation provides for facilitated development discussions with the FDA, tax credits for qualified clinical trials, a waiver on the user fee for marketing application (PDUFA fee), and extended market exclusivity of up to 7 years as a way to encourage companies to develop treatments for rare conditions.

"This designation reflects the need for new treatment options for the estimated 80,000 patients living with retinitis pigmentosa in the U.S. alone," Brian M. Strem, PhD, President and CEO of Kiora, said in a company news release.

"While RP can be caused by a variety of gene mutations, our small molecule KIO-301 has the potential to be used as a standalone therapy regardless of the underlying mutation(s) or in combination with next-generation gene-specific therapies. We plan to begin a phase 1b clinical trial in Australia later this year before initiating larger studies in the U.S. and worldwide."

KIO-301 (benzyl ethyl aminoazobenzene quaternary ammonium) is a visible light-sensitive small molecule that acts as a reversible 'photoswitch', specifically designed to restore the eyes' ability to perceive and interpret light in visually impaired patients.

KIO-301 selectively enters specific retinal ganglion cells (those downstream of degenerated rods and cones) and converts them into light sensing cells, capable of signaling the brain as to the presence or absence of light.

Retinitis pigmentosa is a rare inherited disease that typically presents with loss of low light vision, followed by reduced visual field (loss of peripheral vision) and eventually loss of central vision. The condition affects approximately one out of every 3,000 to 5,000 people and is caused by more than 50 distinct genetic subtypes from more than 150 gene mutations.