Kiora Announces Publication of Phase 1 Study Demonstrating Safety, Tolerability and Anti-Inflammatory Activity of KIO-101 in the Treatment of Inflammation of the Eye

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Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) announced positive results from a Phase 1 study of KIO-101, a non-steroidal anti-inflammatory agent, showing significant reduction in conjunctival hyperemia in the eye. KIO-101 is well tolerated and aims to reduce T-cell-related inflammation locally in the eye without systemic side effects. The company plans a Phase 2 trial for KIO-104 to target posterior non-infectious uveitis, a rare inflammatory condition of the retina.
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The recent publication of the Phase 1 study results for Kiora Pharmaceuticals' KIO-101 is a pivotal development in the field of ophthalmic drug research. The significance of this study lies in its potential to offer a new therapeutic avenue for patients suffering from ocular inflammation, particularly those for whom current treatments may be inadequate or accompanied by undesirable side effects. The active molecule in KIO-101, a DHODH inhibitor, has been shown to effectively reduce T-cell proliferation, which is a promising alternative to steroidal treatments that carry a risk of systemic side effects.

From a research perspective, the successful tolerance of KIO-101 in healthy volunteers and patients with conjunctival hyperemia is noteworthy. The reduction in hyperemia and improvement in OSDI scores are indicative of the drug's efficacy. However, the presence of mild to moderate adverse events, such as epistaxis in the highest dose cohort, will require further scrutiny in subsequent trials. The Phase 2 trial of KIO-104 will be particularly important to monitor, as it targets posterior non-infectious uveitis, a condition with a high unmet medical need due to its potential to cause vision loss.

The announcement by Kiora Pharmaceuticals regarding the positive results of their Phase 1 study for KIO-101 could have a notable impact on the company's financial position and stock market performance. As investors often react to clinical trial outcomes, this development may lead to increased investor confidence and potentially an uptick in Kiora's stock price. The company's strategic focus on developing non-steroidal anti-inflammatory agents for ocular conditions addresses a niche yet significant market segment, which could translate into a competitive edge and revenue growth if the drug successfully progresses through the clinical trial pipeline.

Investors should monitor the upcoming Phase 2 trials, as positive results there could further validate the drug's efficacy and safety profile, potentially leading to increased market valuation. The fact that KIO-101 and KIO-104 are part of a family of compounds targeting different ocular conditions suggests a scalable drug development strategy that could diversify Kiora's product portfolio and revenue streams in the future.

Kiora Pharmaceuticals' progress in developing KIO-101 and KIO-104 positions the company within the growing ophthalmic anti-inflammatory market. The demand for non-steroidal options is driven by the need to manage ocular conditions with fewer side effects compared to traditional steroidal treatments. The differentiation of KIO-101 for topical delivery and KIO-104 for intravitreal injection allows Kiora to cater to a broader range of inflammatory ocular diseases, potentially expanding its market reach.

The company's strategic alignment with current healthcare trends, such as personalized medicine and targeted therapies, is commendable. The proprietary active molecule's ability to inhibit DHODH, a pathway involved in autoimmune diseases, aligns with the industry's push towards precision medicine. Assuming successful completion of clinical trials and eventual FDA approval, Kiora could seize a significant share of the market, particularly for conditions like non-infectious posterior uveitis, which lack adequate treatment options.

Encinitas, California--(Newsfile Corp. - March 8, 2024) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX), ("Kiora" or the "Company") announced the publication of results from a Phase 1 double-masked study of KIO-101, a potent, non-steroidal anti-inflammatory agent. KIO-101 contains the same active molecule as KIO-104, which belongs to a family of potent anti-inflammatory agents, The study showed that a 12-day treatment of KIO-101 topically at multiple doses was well tolerated in healthy volunteers and patients with inflammation of the eye. There was a significant decrease in conjunctival hyperemia in the treatment group compared to the placebo group. Conjunctival hyperemia, a sign of ocular inflammation, is due to a variety of non-infectious and infectious causes. The paper was published in the journal, Pharmaceutics (2024, 16(3), 367 |

Kiora's KIO-100 family of compounds contain the same proprietary active molecule but are uniquely formulated for a specific condition and region of the eye being targeted. KIO-101 is the designation for topical delivery and KIO-104 is the designation for intravitreal injection (directly into the eye). Kiora's active molecule belongs to a class of drugs known as DHODH inhibitors, including teriflunomide, an FDA-approved drug that has been prescribed to hundreds of thousands of patients with multiple sclerosis. DHODH inhibitors are believed to reduce the production of T-cells, a type of immune cell that, in certain conditions like autoimmune disease, can cause damaging inflammation. Delivering KIO-101 or KIO-104 locally in the eye aims to reduce T-cell-related inflammation without the associated potential side effects of systemic anti-inflammatory drugs.

"These results support the therapeutic potential of our molecule to address inflammation in the eye," explained Eric Daniels, MD, Chief Development Officer, Kiora. "A Phase 2 trial of KIO-104 is expected to start later this year to treat posterior non-infectious uveitis, a rare inflammatory condition of the retina. Because uveitis can lead to vision loss, there's an important need for new treatment options beyond chronic steroid injections or systemic autoimmune disease drugs currently being used today. Beyond uveitis, we see an opportunity to target additional retinal inflammatory diseases that could similarly benefit from a non-steroidal therapeutic option."

The published study involved a double-masked, placebo-controlled, randomized, parallel-group design that consisted of two parts. In Part I, 24 healthy volunteers received single or multiple administrations of KIO-101 eye drops in ascending doses (0.05%, 0.15% or 0.30%). Part II involved 21 patients with conjunctival hyperemia who received either 0.15% KIO-101 eye drops or vehicle (2:1) twice daily for 12 consecutive days. The findings include the following:

  • KIO-101 eye drops were well tolerated in all subjects.
  • There were no serious adverse events. All adverse events were transient and considered mild to moderate.
  • In the highest dose cohort (0.30%), epistaxis occurred in two subjects after multiple instillations.
  • In Part II, after 12 days of treatment with 0.15% KIO-101, conjunctival hyperemia decreased by 1.1 ± 0.27 points in the KIO-101 treated group versus 0.6 ± 0.79 points in the placebo group (p = 0.0385).
  • From baseline to day 12, the Ocular Surface Disease Index Questionnaire (OSDI) score decreased by 20.3 points in the treatment group, while in the placebo group decreased by 14.0 points (p=ns).

Kiora is planning a Phase 2 clinical trial of KIO-104 to be administered by intravitreal injection directly into the retina for the treatment of posterior non-infectious uveitis. The Company previously reported positive results from a Phase 1 study of KIO-104 for treatment of posterior non-infectious uveitis. Posterior non-infectious uveitis is a rare autoimmune disease characterized by inflammation of the retina, choroid, vitreous, or optic nerve, and can result in severe vision loss. It can result from many underlying causes, which may include the ocular manifestation of certain autoimmune diseases.

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 potential of the KIO-100 family to address inflammation in the eye, the expected timing of a Phase 2 trial of KIO-104, and the development and commercialization efforts and other regulatory or marketing approval efforts pertaining to Kiora's development-stage products, including KIO-104, as well as the success thereof, where such approvals or success may not be obtained or achieved on a timely basis or at all. 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 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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What is the active molecule in KIO-101 and KIO-104?

The active molecule in both KIO-101 and KIO-104 is a DHODH inhibitor belonging to a class of drugs known to reduce T-cell production.

What condition is KIO-101 formulated for?

KIO-101 is formulated for inflammation of the eye, specifically targeting conjunctival hyperemia.

What are the potential benefits of KIO-101 and KIO-104?

KIO-101 and KIO-104 aim to reduce T-cell-related inflammation in the eye without systemic side effects, potentially offering new treatment options for retinal inflammatory diseases.

What Phase 2 trial is Kiora planning?

Kiora is planning a Phase 2 trial for KIO-104 to target posterior non-infectious uveitis by intravitreal injection into the retina.

What is posterior non-infectious uveitis?

Posterior non-infectious uveitis is a rare autoimmune disease characterized by inflammation of the retina, choroid, vitreous, or optic nerve, leading to severe vision loss.

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