Kiora Pharmaceuticals has entered into an agreement with Théa Open Innovation SAS concerning the KIO-301 molecular photoswitch product

Under the agreement, Kiora granted TOI exclusive worldwide development and commercialization rights, excluding Asia, to KIO 301 for the treatment of degenerative retinal diseases. In exchange, Kiora will receive an upfront, payment of $16 million; up to $285 million upon achievement of pre-specified clinical development, regulatory and commercial milestones; tiered royalties of up to low 20% on net sales; and reimbursement of KIO 301 research and development expenses.

"Our partnership with TOI provides us the strategic, financial and commercial resources that we believe will help to bring innovative treatments to market for patients living with inherited retinal disease," said Brian Strem, Ph.D., CEO of Kiora. "Based on the Phase I/II (ABACUS) data of KIO 301 in Retinitis Pigmentosa (RP), we have started to implement our plan to initiate our Phase II, multicenter, controlled clinical trial for retinitis pigmentosa, in early 2024 with the goal of reporting results in H1 2025 and explore other retinal disease where KIO-301 may be applicable."

Data from ABACUS, reported in November 2023, demonstrated meaningful vision improvements in patients with late-stage RP. Findings included significant improvement in visual field, concordant trended improvements in visual acuity and tests of functional vision relating to the use of sight in everyday activities. In addition, functional MRI demonstrated increased visual cortex activity (region of the brain responsible for processing vision) relative to baseline at two and 14 days after treatment.

The strategic partnership covers retinitis pigmentosa and potentially other indications in ophthalmology across all global geographies, excluding China, Japan, and certain other countries in Asia. Kiora is primarily responsible for the design and implementation of clinical development through phase II whereas Théa will assume primary responsibility for phase III clinical trials as well as for securing regional marketing authorizations. Upon approval in respective regions, Théa will be responsible for all commercial activities including sales, marketing and market access.

KIO 301 is a small molecule, referred to as a molecular photoswitch, designed to confer light-sensing capabilities to Retinal Ganglion Cells (RGCs), a special cell type of the retina. In healthy eyes, light detection is primarily performed by photoreceptors (rods and cones). In patients with numerous types of inherited retinal disease, mutations in one of more than hundreds of known genes can lead to the death of photoreceptors. This retinal degeneration results in lost vision for the patient. KIO 301 is able to selectively enter RGCs downstream of degenerated photoreceptors and once inside, KIO 301 interacts with voltage-gated ion channels. When light hits RGCs, KIO 301 alters its shape to change the flow of current, thereby activating the neurons, and resulting in signaling the brain. When light is removed, KIO 301 reverts to its lower energy shape, stopping the signaling to the brain. In this way, the molecule acts as a light switch within the eye.