Kiora Pharmaceuticals Reports its Ion Channel Modulator, KIO-300, Suppressed Seizure Activity in Preclinical Epilepsy Model

Rhea-AI Summary

Kiora Pharmaceuticals (NASDAQ:KPRX) reported preclinical data showing its ion channel modulator KIO-300 significantly suppressed seizure-associated electrophysiological activity in an ex vivo temporal lobe epilepsy model.

KIO-300, the active ingredient in Phase 2 vision-restoration drug KIO-301, reduced epileptiform event frequency and cumulative burden without impairing normal brain electrical transmission.

News Market Reaction – KPRX

-1.56%

-1.56% Session close to close

In the Jun 18 session, KPRX declined 1.56%, reflecting a mild negative market reaction.

Data tracked by StockTitan Argus on the day of publication.

Market Context

This announcement highlights robust preclinical seizure suppression with p statistics and prior 50%...

Analysis

This announcement highlights robust preclinical seizure suppression with p < 0.0001 statistics and prior 50% retinal hyperactivity reduction, extending KIO-300’s potential beyond ophthalmology. Investors may watch for translational plans while considering low reported short positioning.

Key Figures

Onset of inhibition: 42 minutes Event frequency p-value: p < 0.0001 Cumulative burden p-value: p < 0.0001 +2 more

5 metrics

Onset of inhibition 42 minutes Time after KIO-300 treatment when epileptiform inhibition reached significance

Event frequency p-value p < 0.0001 Statistical significance for reduced epileptiform event frequency vs vehicle

Cumulative burden p-value p < 0.0001 Statistical significance for reduction in cumulative epileptiform burden

Retinal hyperactivity reduction 50% Approximate reduction in pathological retinal hyperactivity in earlier research

Conference dates June 18–19, 2026 Epilepsy Foundation Pipeline Conference presentation window

Historical Context

5 past events · Latest: May 08 (Neutral)

5 events

Date	Event	Sentiment	24h Move	Catalyst
May 08	Earnings update	Neutral	+0.0%	Quarterly financials and pipeline updates including ABACUS-2 and KLARITY.
Apr 14	Clinical results	Neutral	+0.0%	Phase 1 ABACUS-1 Nature Medicine publication and Phase 2 ABACUS-2 initiation.
Apr 07	Private placement	Neutral	+6.9%	Up to $24M financing with upfront proceeds and milestone-based warrants.
Apr 02	Management change	Neutral	+4.1%	Chief Development Officer departure alongside continued trial enrollment.
Mar 25	Earnings & pipeline	Neutral	-1.0%	Q4/FY 2025 results and advancement of two Phase 2 retinal trials.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

ion channel modulator, epileptiform activity, temporal lobe epilepsy, hippocampal ca1, +2 more

6 terms

ion channel modulator medical

"part of the company's novel ion channel modulator platform, significantly suppressed"

A molecule or therapy that changes how ion channels—protein 'gates' in cell membranes that control the flow of charged particles—open or close, altering electrical signals and cell behavior. Investors should care because these modulators are often drug candidates for conditions like heart rhythm problems, pain, or neurological disorders; their success or failure affects clinical risk, regulatory approval, market size, and safety profiles much like a new engine component determines a vehicle’s performance and reliability.

epileptiform activity medical

"Data Presented ... Demonstrated Significant Reduction in Epileptiform Activity"

Patterns of abnormal brain electrical activity seen on recordings, such as brief spikes or sharp waves, that indicate an increased risk of seizures or brain irritation. Investors watch changes in epileptiform activity during clinical trials or device testing because rising activity can signal safety problems or seizure risk, while reductions can suggest therapeutic benefit—similar to dashboard warning lights that reveal safety or performance issues affecting approval and commercial value.

temporal lobe epilepsy medical

"in an ex vivo temporal lobe epilepsy (TLE) model."

A form of epilepsy caused by abnormal electrical activity in the temporal lobe, the brain area involved in memory, language and emotion, that leads to recurrent seizures and changes in behavior or awareness. Investors care because shifts in diagnosis, treatment options or regulatory approvals—such as new drugs, devices or imaging tools—can affect healthcare costs, market demand and the value of companies working on therapies; think of it as a faulty control room circuit that creates unpredictable outages needing repair.

hippocampal ca1 medical

"in hippocampal CA1 slices from mice with induced temporal lobe epilepsy."

Hippocampal CA1 is a specific region inside the brain’s memory center that plays a key role in forming and retrieving memories, often likened to a critical processing lane on a data highway. It matters to investors because many neurological drugs, imaging tests, and biomarkers focus on protecting or measuring CA1 health; success or failure in affecting this area can drive clinical trial outcomes, regulatory decisions, and future revenue for companies in neuroscience.

electrophysiological activity medical

"significantly suppressed seizure-associated electrophysiological activity in an ex vivo"

Electrophysiological activity is the electrical signaling produced by cells and tissues—most commonly nerves, the heart, or the brain—that shows how well they are working. Investors care because changes in these signals are used to diagnose conditions, measure whether a drug or device is helping or harming, and support regulatory approval; think of it as the electrical readout doctors use like a car’s dashboard to judge performance and safety.

retinitis pigmentosa medical

"vision restoration in patients with gene mutation-agnostic Retinitis Pigmentosa"

Retinitis pigmentosa is a group of inherited disorders that slowly damage the light-sensing cells in the retina, causing progressive loss of peripheral and night vision and often leading to legal blindness. It matters to investors because the condition defines a clear patient population and long-term need for treatments, making clinical trial design, potential market size, and regulatory approval pathways central considerations when valuing companies developing diagnostics or therapies.

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Data Presented at Epilepsy Foundation Pipeline Conference Demonstrated Significant Reduction in Epileptiform Activity: KIO-300 is the active pharmaceutical ingredient of KIO-301, Kiora's ongoing Phase 2 clinical program in vision restoration in patients with gene mutation-agnostic Retinitis Pigmentosa

Encinitas, California--(Newsfile Corp. - June 18, 2026) - Kiora Pharmaceuticals (NASDAQ: KPRX) today announced preclinical data showing that KIO-300, part of the company's novel ion channel modulator platform, significantly suppressed seizure-associated electrophysiological activity in an ex vivo temporal lobe epilepsy (TLE) model. The findings were presented in a poster at the Epilepsy Foundation Pipeline Conference taking place June 18-19, 2026, in Leesburg, VA.

"These data reinforce the broad therapeutic potential of our ion channel modulator platform and suggest potential utility beyond ophthalmology," said Brian Strem, Ph.D., chief executive officer of Kiora Pharmaceuticals. "There is a compelling opportunity to explore additional therapeutic applications where we retain full development and commercialization rights outside the eye. Future translational R&D efforts will evaluate targeted molecular modifications and delivery strategies to enhance disease-specific benefits in epilepsy and other neurological disorders."

Key findings include:

• KIO-300 produced sustained suppression of epileptiform activity in hippocampal CA1 slices from mice with induced temporal lobe epilepsy.
• Treatment reduced spontaneous epileptiform event frequency compared with vehicle controls, with statistically significant inhibition beginning 42 minutes after treatment (p < 0.0001).
• Analysis of cumulative epileptiform burden also demonstrated a significant reduction following KIO-300 exposure (p < 0.0001).

The suppressive effect persisted throughout both treatment and washout periods, suggesting prolonged neural tissue retention. Importantly, KIO-300 did not impair broader electrical transmission in brain tissue, indicating its effects were specific to abnormal spontaneous activity rather than generalized neurological suppression.

The scientific rationale builds on earlier retinal degeneration research in which KIO-300 reduced pathological retinal hyperactivity by approximately 50%. Because neuronal hyperexcitability is also a hallmark of epilepsy, researchers selected temporal lobe epilepsy as an exploratory indication to evaluate whether KIO-300's membrane-calming effects extend to the central nervous system.

The poster, titled "KIO-300/BENAQ Modulates Epileptiform Activity in an Ex Vivo Hippocampal TLE Model," was authored by researchers from Kiora Pharmaceuticals.

About Kiora Pharmaceuticals

Kiora Pharmaceuticals is a clinical-stage biotechnology company developing advanced therapies for retinal disease. We target critical pathways underlying retinal diseases using innovative small molecules to slow, stop, or restore vision loss. KIO-301 is being developed initially for the treatment of retinitis pigmentosa, with potential to expand into 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 macular edema due to retinal inflammation. It is a next-generation, non-steroidal, immuno-modulatory, and small-molecule inhibitor of dihydroorotate dehydrogenase (DHODH).

In addition to news releases and SEC filings, we expect to post information on our website, www.kiorapharma.com, and social media accounts that could be relevant to investors. We encourage investors to follow us on X and LinkedIn as well as to visit our website and/or subscribe to email alerts.

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, Kiora's ability to execute on development and commercialization efforts and other regulatory or marketing approval efforts pertaining to Kiora's development-stage products, including KIO-104 and KIO-301, as well as the success thereof, which such approvals or success may not be obtained or achieved on a timely basis or at all, the sufficiency of existing cash and short-term investments on hand to fund operations for specific periods, the timeline of anticipated readouts, the potential for pipeline expansion, the potential to add trial centers, expand the geographic footprint of trials and/or accelerate enrollment, the potential for KIO-301 and KIO-104 to address multiple indications, and the possibility of future global registration studies and commercialization. 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, 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 25, 2026 or described in Kiora's other public filings, including on Form 10-Q filed with the SEC on May 8, 2026. 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.

Contacts:
Investors
Investors@kiorapharma.com

To view the source version of this press release, please visit https://www.newsfilecorp.com/release/301913

FAQ

What did Kiora (NASDAQ:KPRX) report about KIO-300 in the June 18, 2026 epilepsy study?

Kiora reported that KIO-300 significantly suppressed seizure-associated electrophysiological activity in an ex vivo temporal lobe epilepsy model. According to Kiora, treatment reduced spontaneous epileptiform event frequency and cumulative burden compared with vehicle controls, with effects persisting through treatment and washout.

How did KIO-300 affect epileptiform activity in the temporal lobe epilepsy model for KPRX?

KIO-300 produced sustained suppression of epileptiform activity in hippocampal CA1 slices from mice with induced temporal lobe epilepsy. According to Kiora, statistically significant inhibition began about 42 minutes after treatment, with p < 0.0001 for both event frequency and cumulative epileptiform burden reductions.

Did KIO-300 impact normal brain electrical transmission in Kiora’s preclinical epilepsy data?

KIO-300 did not impair broader electrical transmission in brain tissue in this model. According to Kiora, the modulatory effects appeared specific to abnormal spontaneous epileptiform activity, suggesting targeted action on pathological hyperexcitability rather than generalized neurological suppression in the ex vivo system studied.

What is the relationship between KIO-300 and KIO-301 in Kiora’s pipeline (KPRX)?

KIO-300 is the active pharmaceutical ingredient of KIO-301, which is in a Phase 2 vision restoration program for retinitis pigmentosa. According to Kiora, preclinical epilepsy findings may indicate broader ion channel modulator platform potential beyond ophthalmology, while rights outside the eye are retained.

How do Kiora’s epilepsy findings with KIO-300 relate to prior retinal research?

KIO-300 previously reduced pathological retinal hyperactivity by about 50% in retinal degeneration research. According to Kiora, because neuronal hyperexcitability also characterizes epilepsy, scientists tested temporal lobe epilepsy to see if these membrane-calming effects extend from the retina to central nervous system tissue.

Where and when were Kiora’s new KIO-300 epilepsy data presented?

The preclinical epilepsy data for KIO-300 were presented as a poster at the Epilepsy Foundation Pipeline Conference on June 18–19, 2026, in Leesburg, Virginia. According to Kiora, the poster was titled “KIO-300/BENAQ Modulates Epileptiform Activity in an Ex Vivo Hippocampal TLE Model.”