Kiora Pharmaceuticals has obtained approval to commence the Phase Ib ABACUS clinical trial of the investigational therapy, KIO-301, to restore lost vision in retinitis pigmentosa patients.

A visible light-sensitive small molecule, KIO-301 works as a reversible ‘photoswitch’, specifically made for restoring the ability of the eyes to perceive and interpret light in patients who are visually impaired. 

It enters retinal ganglion cells that downstream degenerated rods and cones and ‘switches’ them into light-sensing cells, which alerts the brain on the presence or absence of visible light.

The open-label, first-in-human, single ascending dose trial will analyse KIO-301 in individuals with retinitis pigmentosa. 

This single-site trial will be carried out at The Royal Adelaide Hospital (RAH) in Adelaide, South Australia.

Kiora Pharmaceuticals chief development officer Eric Daniels said: “This is a pivotal development for Kiora as we pursue the goal of restoring functional vision in patients living with retinitis pigmentosa. 

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“We anticipate beginning enrolment of this study in the third quarter of this year and reporting interim data from this trial by the end of this year.”

An inherited genetic eye disease, retinitis pigmentosa causes severe loss of functional vision because of the retinal photoreceptor (rods and cones) degeneration.

In March this year, the US Food and Drug Administration granted Orphan Drug Designation for KIO-301 to treat retinitis pigmentosa. 

To encourage firms in developing therapies for rare conditions, the orphan drug status provides advantages such as facilitated development discussions with the regulatory agency, tax credits for trials, user fee waiver for a marketing application and longer market exclusivity of up to seven years.