Kiora Pharmaceuticals to Present Additional Data from Its ABACUS-1 Trial in Retinitis Pigmentosa at the ARVO 2024 Annual Meeting

Encinitas, California--(Newsfile Corp. - March 28, 2024) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX), ("Kiora" or the "Company") announced that additional data from the ABACUS-1 trial has been accepted for presentation at the Association for Research in Vision and Ophthalmology (ARVO) meeting in Seattle, WA, May 5-9, 2024. The additional data includes quantitative evaluation of the functional MRI measures. Qualitative assessment of fMRI demonstrated reactivation of the regions of the brain responsible for vision in a time-dependent manner, consistent with other improvements in visual function.

The presentation, titled, "Synthetic phototransduction with a light-responsive molecule (KIO-301) in advanced retinitis pigmentosa: the ABACUS-1 phase I/II trial," will be presented by Professor Robert James Casson, DPhil, Head of Ophthalmology and Visual Science at Adelaide University. The ABACUS-1 study was a Phase I/II first-in-human clinical trial of the Company's molecular photoswitch, KIO-301, in patients with late-stage retinitis pigmentosa (RP). Initial preliminary topline results from ABACUS-1 were announced at the American Academy of Ophthalmology (AAO) annual conference in November 2023.

Presentation details:

Presentation #: 407
Session Title: Retina miscellaneous: Translational
Date: May 5, 2024
2:00 PM Pacific Daylight Time

About KIO-301

KIO-301 is a small molecule photoswitch. It is designed to selectively confer light-sensing capabilities to retinal ganglion cells (RGCs). In healthy eyes, light is first converted to electrical signals via the rods and cones (photoreceptors) and transmitted through RGCs to the vision perceiving part of the brain (visual cortex). In many inherited retinal diseases (IRDs), genetic mutations cause photoreceptors to degenerate and die off, affecting an individual's ability to perceive light. However, while photoreceptors degenerate in IRDs, RGCs are preserved. They therefore represent a target cell to bypass degenerated photoreceptors, perceive light, and signal the brain. It has been shown KIO-301 selectively enters RGCs downstream of degenerated photoreceptors. In the presence of light, KIO-301 turns to an "on" position, triggering the RGC to signal the brain. In the absence of light, KIO-301 turns to an "off" position and signaling to the brain stops. In this way, the molecule acts as a light switch within the eye.

In January of 2024, Kiora granted Théa Open Innovation (TOI), a sister company of the global ophthalmic specialty company Laboratoires Théa (Théa), exclusive worldwide development and commercialization rights, excluding Asia, to KIO-301 for the treatment of degenerative retinal diseases. As part of this agreement, the companies are jointly planning a Phase 2 multicenter, controlled clinical trial for retinitis pigmentosa.

About Retinitis Pigmentosa

Retinitis pigmentosa (RP) is a hereditary degenerative disorder affecting the retina's photoreceptors with no approved therapies. Typically characterized by progressive loss of side (peripheral) vision and night vision, it results from mutations in one or more than 150 genes. This disease affects approximately 1 in 4,000 individuals globally and about 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 often considered legally blind by 55, underscores the need for treatment options.

The condition's complexity and genetic heterogeneity make developing treatments challenging, underscoring the need for therapies that address as many or all of the gene mutations implicated in the disease. Kiora's development of KIO-301 as a mutation agnostic treatment for RP could meet this need. This drug is being developed as a standalone therapy or in combination with a potential future gene therapy.

About Kiora Pharmaceuticals

Kiora Pharmaceuticals is a clinical-stage biotechnology company developing and commercializing products for the treatment of orphan retinal diseases. KIO-301 is being developed for the treatment of retinitis pigmentosa, choroideremia, and Stargardt disease. It is a molecular photoswitch that has the potential to restore vision in patients with inherited and/or age-related retinal degeneration. KIO-104 is being developed for the treatment of posterior non-infectious uveitis. It is a next-generation, non-steroidal, immuno-modulatory, and small-molecule inhibitor of dihydroorotate dehydrogenase.

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Forward-Looking Statements

Some of the statements in this press release are "forward-looking" and are made pursuant to the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These "forward-looking" statements include statements relating to, among other things, the development and commercialization efforts and other regulatory or marketing approval efforts pertaining to Kiora's development-stage products, including KIO-301 and KIO-104, as well as the success thereof, with such approvals or success may not be obtained or achieved on a timely basis or at all, the ability of KIO-301 to improve visual function, the potential to expand KIO-301 to other indications including choroideremia and Stargardt disease, and the planned design of the Phase 2 clinical trial for KIO-301. These statements involve risks and uncertainties that may cause results to differ materially from the statements set forth in this press release, including, among other things, the ability to conduct clinical trials on a timely basis, the ability to obtain any required regulatory approvals, whether future trials of KIO-301 will yield similar results for participants, market and other conditions, and certain risk factors described under the heading "Risk Factors" contained in Kiora's Annual Report on Form 10-K filed with the SEC on March 23, 2023, or described in Kiora's other public filings. Kiora's results may also be affected by factors of which Kiora is not currently aware. The forward-looking statements in this press release speak only as of the date of this press release. Kiora expressly disclaims any obligation or undertaking to release publicly any updates or revisions to such statements to reflect any change in its expectations with regard thereto or any changes in the events, conditions, or circumstances on which any such statement is based, except as required by law.



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SOURCE Kiora Pharmaceuticals, Inc.