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Kiora licenses KIO-401 for $3.5M upfront

Kiora's planned CRAO and NAION trials are conditional on funding, with initiation targeted for 2027 and 2028, respectively.

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Form Type
8-K

Rhea-AI Filing Summary

Kiora Pharmaceuticals (KPRX) entered an exclusive, sublicensable license with Senju Pharmaceutical for KIO-401, with development and commercialization rights outside Asia; the agreement is effective October 5, 2026. Kiora will pay $3.5 million upfront, a one-time payment in the low teen millions of dollars upon technology transfer expected in 2027, and up to $168.5 million upon specified regulatory and commercial milestones, plus single-digit royalties on net sales. A portion of the technology-transfer payment may be credited against future milestones. Kiora is responsible for and will fund development in its territory.

Senju completed Phase 2 and Phase 3 CRAO studies in Japan. The Phase 2a study reported statistically significant ETDRS visual-acuity improvement from baseline versus a prospective observational registry (p=0.0398); the Phase 3 study reported a significant improvement among patients legally blind at baseline (p=0.0195), while the overall primary endpoint was reported at p=0.0949. Kiora targets a multinational CRAO Phase 3 start in 2027, subject to funding and regulatory alignment, and a Phase 2b NAION start in 2028, subject to funding and regulatory considerations. Kiora is evaluating financing alternatives and has discontinued KIO-104 research and development; it plans to report enrolled KLARITY patient data and consider partnering or out-licensing.

Item 1.01 Entry into a Material Definitive Agreement Business
The company signed a significant contract such as a merger agreement, credit facility, or major partnership.
Item 7.01 Regulation FD Disclosure Disclosure
Material non-public information disclosed under Regulation Fair Disclosure, often investor presentations or guidance.
Item 9.01 Financial Statements and Exhibits Exhibits
Financial statements, pro forma financial information, or exhibit attachments filed with this report.
Upfront payment $3.5 million KIO-401 license agreement
Technology-transfer payment Low teen millions of dollars One-time payment upon technology transfer expected in 2027
Regulatory and commercial milestone payments Up to $168.5 million Subject to achievement of specified milestones
Target CRAO Phase 3 initiation 2027 Subject to funding and regulatory alignment
Target NAION Phase 2b initiation 2028 Subject to funding and regulatory considerations
Phase 2a study result p=0.0398 ETDRS visual acuity improvement versus a prospective observational registry
Phase 3 legally blind subgroup result p=0.0195 Improvement among patients legally blind at baseline
Phase 3 overall primary endpoint p=0.0949 Overall study result
sublicensable license financial
"exclusive, sublicensable license"
A sublicensable license is a grant of rights from a licensor to a licensee that explicitly allows the licensee to authorize third parties to use the licensed intellectual property or assets by issuing sublicenses. It means the licensee can transfer some or all of the licensed rights downstream, but those sublicenses remain subject to the original license’s scope, restrictions, duration and any conditions (for example, territorial limits or approval requirements), so the licensor’s original terms continue to control what sublicensees may do.
ETDRS medical
"Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity"
ETDRS (Early Treatment Diabetic Retinopathy Study) refers to a standardized eye chart and scoring system used in clinical trials to measure visual acuity. It converts how many letters a person can read on a chart into a numeric score, so doctors can track small but meaningful changes in sight. Investors care because ETDRS scores are common, objective endpoints in ophthalmology trials and can determine whether a treatment is judged effective.
BCVA medical
"best-corrected visual acuity (BCVA)"
Best corrected visual acuity (BCVA) is the sharpest level of vision a person can achieve when using the optimal prescription lenses during an eye exam, measured by reading standardized letters on a chart. Investors care because BCVA is a common, standardized clinical endpoint in eye‑disease trials and regulatory reviews; improvements in BCVA are used like a speedometer to gauge how well a treatment works and therefore influence a product’s market potential and valuation.
NAION medical
"non-arteritic anterior ischemic optic neuropathy (NAION)"
Nonarteritic anterior ischemic optic neuropathy (NAION) is a sudden loss of vision caused by reduced blood flow (ischemia) to the front portion of the optic nerve. It typically produces painless, often partial vision loss in one eye on awakening or over hours to days and is linked to risk factors that affect small blood vessels, such as high blood pressure, diabetes, sleep apnea, and an anatomically crowded optic nerve head; it is distinct from arteritic AION, which is caused by inflammatory artery disease.
single-digit royalties financial
"single-digit royalties on applicable net sales"

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What payments will KPRX make to Senju for KIO-401?
Kiora will pay Senju $3.5 million upfront, plus a one-time payment in the low teen millions of dollars upon technology transfer expected in 2027. Senju may also receive up to $168.5 million in specified milestone payments and single-digit royalties on net sales.

When does KPRX plan to start KIO-401 clinical trials?
Kiora targets a multinational Phase 3 trial for CRAO in 2027, subject to funding and regulatory alignment, and a Phase 2b trial for NAION in 2028, subject to funding and regulatory considerations.

How many patients are planned for KPRX's KIO-401 CRAO Phase 3 trial?
The planned multinational CRAO trial is estimated to enroll more than 100 patients. The final sample size is to be determined following discussions with regulatory authorities.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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0001372514false00013725142026-10-232026-10-23

UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, DC 20549

FORM 8-K
CURRENT REPORT PURSUANT
TO SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of report (Date of earliest event reported): October 23, 2026

Kiora Logo.jpg
KIORA PHARMACEUTICALS, INC.
(Exact name of registrant as specified in its charter)
Delaware
(State or other jurisdiction of incorporation)
001-3667298-0443284
(Commission File Number)(IRS Employer Identification No.)

169 Saxony Rd.
Suite 212
Encinitas, CA 92024
(858) 224-9600
(Registrant’s telephone number, including area code)
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
☐
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
☐Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
☐Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
☐Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Securities registered pursuant to Section 12(b) of the Act:
Title of each class:Trading Symbol(s)Name of each exchange on which registered:
Common Stock, $0.01 par valueKPRXNASDAQ
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Item 1.01.    Entry Into a Material Definitive Agreement.

On October 23, 2026, Kiora Pharmaceuticals, Inc. (the “Company”) entered into an Exclusive License Agreement (the “Agreement”) with Senju Pharmaceutical Co., Ltd. (“Senju”) to license a late-stage neuroprotective, oral small-molecule calpain inhibitor (“KIO-401”). Under the Agreement, which has an effective date of October 5, 2026, Senju granted the Company an exclusive, sublicensable license to develop, manufacture, commercialize, and otherwise exploit KIO-401 throughout the world except for certain specified countries, including countries in Asia (the “Territory”), to develop and commercialize a companion diagnostic tool for glaucoma in the Territory, and to file for regulatory approvals with respect to KIO-401 in the Territory for use in the field of ophthalmology (the “Field”).

Kiora will have sole responsibility, control, and decision-making authority, at its own cost and expense, for development of KIO-401 in the Field in the Territory, taking into account the availability, completeness and quality of Senju technology, regulatory feedback, clinical and commercial considerations, and other relevant factors.

Under the Agreement, the Company will pay Senju an up-front payment of $3.5 million. The Company will also pay Senju an amount in the low teen millions of dollars upon completion of the technology transfer, a portion of which may be credited against future milestone payments, and will make additional payments of up to $168.5 million subject to the achievement of certain specified regulatory and commercial milestones. In addition, the Company will pay single-digit royalties based on a specified percentage of net sales of KIO-401 in the Territory, subject to adjustment in certain circumstances. Either party may terminate the Agreement in its entirety upon certain customary events.

The foregoing description of the Agreement is qualified in its entirety by reference to the full text of the Agreement, a copy of which are attached hereto as Exhibit 10.1, and which is incorporated herein in its entirety by reference. The representations, warranties and covenants contained in the Agreement were made only for purposes of the Agreement and as of specific dates, were solely for the benefit of the parties to the Agreement and may be subject to limitations agreed upon by the contracting parties.

Item 7.01. Regulation FD Disclosure.
On October 29, 2026, the Company issued a press release announcing the signing of the Agreement with Senju. A copy of the press release is furnished as Exhibit 99.1, and is incorporated herein by reference.

Also on October 29, 2026, the Company posted an investor presentation, which may be accessed through the Company’s investor relations website. A copy of the presentation is furnished herewith as Exhibit 99.2 to this Current Report on Form 8-K and is incorporated by reference herein. The Company intends to use this presentation in meetings with analysts, investors and others from time to time.

The information furnished under this Item 7.01, including Exhibit 99.1 and Exhibit 99.2, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, and shall not be deemed incorporated by reference into any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as shall be expressly set forth by specific reference in such filing.



Item 9.01.    Financial Statements and Exhibits.
(d) Exhibits.
Exhibit 
Number		Title
10.1†*
Exclusive License Agreement by and between Kiora Pharmaceuticals, Inc. and Senju Pharmaceutical Co., Ltd., dated as of October 23, 2026
99.1
Press Release of Kiora Pharmaceuticals, Inc., dated as of October 29, 2026
99.2
Company Presentation, dated as of October 29, 2026
104Cover Page Interactive Data File (embedded within the Inline XBRL document).
†Certain confidential portions of this exhibit were omitted because the identified confidential portions (i) are not material and (ii) would be competitively harmful if publicly disclosed.
*Schedules and exhibits have been omitted from this exhibit pursuant to Item 601(a)(5) of Regulation S-K. The registrant hereby undertakes to furnish copies of any of the omitted schedules and exhibits upon request by the U.S. Securities and Exchange Commission.


SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
KIORA PHARMACEUTICALS, INC.
By:/s/ Melissa Tosca
Melissa Tosca
Chief Financial Officer
(Principal financial and accounting officer)
Date:October 29, 2026



Exhibit 99.1

Kiora Pharmaceuticals In-Licenses Late-Stage, Oral Neuroprotective Calpain Inhibitor for Central Retinal Artery Occlusion (CRAO) with Planned Expansion into NAION; Asset Completed Phase 3 Clinical Trial in CRAO in Japan

•The oral small-molecule calpain inhibitor demonstrated meaningful visual acuity improvement in Phase 2 and Phase 3 CRAO studies conducted in Japan compared to controls.
•No therapeutics exist today to treat patients that experience CRAO vision loss.
•Kiora plans to advance KIO-401 (mitucapistat) into a multinational Phase 3 registration trial in CRAO, with initiation targeted for 2027.
•Company also plans to evaluate KIO-401 in a Phase 2b clinical trial in non-arteritic anterior ischemic optic neuropathy (“NAION”).

SAN DIEGO, October 29, 2026 — Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) (“Kiora” or the “Company”) today announced that it has obtained exclusive development and commercialization rights, outside Asia, to KIO-401, a late-stage neuroprotective, oral small-molecule calpain inhibitor, from Senju Pharmaceutical Co., Ltd. (“Senju”). KIO-401 is being developed as a potential treatment for central retinal artery occlusion (“CRAO”), a rare, acute ischemic retinal disease described as “stroke of the eye” that can result in severe, permanent vision loss and for which there are currently no approved therapies.

A presentation reviewing KIO-401, its clinical data and Kiora’s planned development program is available in the Investors section of the Company’s website at ir.kiorapharma.com.

Senju has completed both Phase 2 and Phase 3 clinical studies of KIO-401 in patients with CRAO in Japan, providing an established clinical foundation for Kiora's planned multinational Phase 3 registration program. The Company is targeting initiation of the study in 2027 and also plans to expand development of KIO-401 into a Phase 2b clinical trial in patients with NAION, another rare, acute, ischemic, ophthalmic disease with no approved treatment.

“KIO-401 addresses a significant unmet need for patients with CRAO and NAION, two rare, acute ophthalmic diseases that result in permanent vision loss and have no approved therapies today,” said Brian M. Strem, Ph.D., President and Chief Executive Officer of Kiora. “We have an established relationship with Senju through our KIO-301 partnership, which facilitated our partnership on KIO-401. After reviewing results from Senju’s previous work, we identified KIO-401 as a compelling late-stage asset. We now plan to build on those findings with a larger, adequately powered multinational Phase 3 trial designed to more definitively evaluate its clinical benefit. We believe KIO-401 has the potential to meaningfully improve patient outcomes and achieve significant commercial value in the U.S., Europe and other regions.”

KIO-401: Late-Stage Program for Acute Ischemic Retinal Disease
KIO-401 (mitucapistat), designated by Senju as SJP-0008 in Japan and Asia, is a neuroprotective, oral small molecule designed to inhibit activated calpain, a calcium-dependent protease implicated in cellular injury following ischemia. CRAO is an acute retinal ischemic event caused by interruption of blood flow to the retina. NAION is caused by reduced blood flow to the optic nerve. Both conditions can trigger cellular injury through oxygen deprivation, increased intracellular calcium accumulation and excessive calpain activation. By inhibiting activated calpain, KIO-401 is intended to reduce downstream cellular damage and preserve visual function following ischemic injury.




Exhibit 99.1
graphic1a.jpg

graphic2a.jpg

In the randomized Phase 2a study in Japan conducted by Senju and its partners, KIO-401 demonstrated a statistically significant improvement from baseline in Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity compared with a prospective, observational registry (p=0.0398). Senju subsequently completed a randomized, double-masked Phase 3 study in Japan, which demonstrated improvement in best-corrected visual acuity (BCVA), including a statistically significant improvement among patients who were legally blind at baseline according to World Health Organization criteria (p=0.0195).

“CRAO remains an under-recognized medical emergency, and even when diagnosed, there is no approved therapy specifically shown to preserve vision,” said Brian Mac Grory, MB BCh BAO, MRCP, Associate Professor of Neurology and Ophthalmology at Duke University School of Medicine and Chair of the writing group for the American Heart Association scientific statement on CRAO. “An effective oral treatment could galvanize systems of care for acute CRAO recognition and management and expand treatment access to patients in regional and community hospitals. The reported clinical trial outcomes are the first encouraging results for a neuroprotective drug for CRAO and strongly support further evaluation of KIO-401.”

Planned Clinical Development in CRAO and NAION
Kiora intends to build upon the clinical experience generated by Senju in Japan through a US-focused, multinational Phase 3 registration trial in CRAO. The Company is targeting initiation in 2027, subject to funding and regulatory alignment. The planned randomized, double-masked, controlled trial will evaluate



Exhibit 99.1
the efficacy and safety of KIO-401 in patients with acute CRAO, with visual function outcomes expected to play a central role in the assessment of treatment benefit. The final study design, including enrollment criteria, primary endpoint and sample size, estimated to be more than 100 patients, will be determined following discussions with regulatory authorities.

“The clinical results generated in Japan support continued global development of this compound as a potential treatment for acute retinal ischemia,” said Shuhei Yoshida, President & CEO of Senju Pharmaceutical. “Kiora brings retinal drug development expertise and a focused strategy for advancing the program outside Asia. Combining Senju's expertise in Japan and broader Asia with Kiora's leadership in global development, we view Kiora as a strong partner to lead development in the United States, Europe and other ex-Asia markets, with a shared goal of bringing a new treatment option to patients suffering from these ischemic insults.”

In parallel, Kiora plans to advance KIO-401 into Phase 2b clinical development for NAION, an acute ischemic optic neuropathy that can cause substantial and permanent vision loss. The Company believes the shared involvement of ischemia-related cellular injury and calpain activation in CRAO and NAION provides a scientific rationale for evaluating KIO-401 in both indications. Initiation of the NAION study is targeted for 2028, subject to funding and regulatory considerations.

Kiora also intends to evaluate opportunities to expand the KIO-401 platform into additional ophthalmic indications in which calpain-mediated cellular injury may contribute to disease progression, including glaucoma. These potential indications would require further preclinical and clinical evaluation.

License Agreement with Senju Pharmaceutical
Under the terms of the agreement, Kiora has obtained exclusive rights to develop and commercialize KIO-401 outside Asia, while Senju retains rights within Asia. The agreement builds on an existing relationship between the companies involving KIO-301, Kiora's photoswitch therapy in development for advanced retinitis pigmentosa.

Under the license agreement, Kiora will make an upfront payment of $3.5 million, followed by a one-time milestone payment in the low teen millions upon technology transfer expected in 2027, a portion of which may be credited against future milestone obligations. Senju is also eligible to receive additional payments upon achievement of specified regulatory and commercial milestones, as well as single-digit royalties on applicable net sales outside Asia.

Financing and Clinical Development Strategy
Kiora is evaluating financing alternatives to fund advanced KIO-401 development in CRAO and NAION.
“Our priority is to secure capital to advance KIO-401 through late-stage development while leveraging our existing clinical infrastructure,” said Melissa Tosca, CPA, Chief Financial Officer of Kiora. “We will prioritize KIO-401 and partnered KIO-301 while pursuing out-licensing opportunities for KIO-104 following discontinuation of its development.”

As KIO-301's Phase 2 ABACUS-2 trial completes enrollment, Kiora expects to transition clinical resources to KIO-401's Phase 3 CRAO program. KIO-301 partner Théa Open Innovation reimburses R&D expenses and holds commercialization rights outside Asia. Kiora is eligible for up to $285 million in additional milestones and tiered royalties reaching the low 20% range. Senju Pharmaceutical holds an option for rights to KIO-301 in key Asian markets.

As Kiora prioritizes investment in KIO-401 and completes the ABACUS-2 trial, the Company made the decision to discontinue research and development of KIO-104. This decision is part of Kiora's portfolio optimization strategy and reflects the Company’s current priorities. Kiora will report data from enrolled patients in the KLARITY clinical trial and consider partnering and out-licensing opportunities for the asset.
KO Law PC served as legal counsel to Kiora in connection with the KIO-401 licensing transaction.

About Kiora Pharmaceuticals



Exhibit 99.1
Kiora Pharmaceuticals is a late-stage retinal therapeutics company developing small-molecule therapies targeting differentiated biological pathways implicated in vision-threatening diseases.

KIO-401 is an oral, neuroprotective calpain inhibitor in development for central retinal artery occlusion (CRAO) and non-arteritic anterior ischemic optic neuropathy (NAION), two rare, acute ischemic ophthalmic diseases with no approved pharmacologic treatments. Kiora is planning a multinational Phase 3 registration trial in CRAO and a Phase 2b clinical trial in NAION. The KIO-401 platform also has potential applications in additional ophthalmic indications, including glaucoma, where calpain-mediated injury may contribute to disease progression.

KIO-301 is a partnered Phase 2 photoswitch therapy for advanced retinitis pigmentosa. KIO-104, a DHODH inhibitor previously evaluated in the KLARITY clinical trial for retinal inflammation and fibrosis, is available for partnering or out-licensing.

For more information, visit www.kiorapharma.com.

Forward-Looking Statements
Some of the statements in this press release are "forward-looking" statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements include, without limitation, statements concerning Kiora's planned development and potential commercialization of KIO-401; the anticipated timing, design, initiation, conduct, enrollment, endpoints and results of clinical trials in CRAO and NAION; the potential efficacy, safety, therapeutic benefit and commercial opportunity of KIO-401; the interpretation and applicability of clinical results generated by Senju Pharmaceutical in Japan; the potential for calpain inhibition in additional ophthalmic indications; anticipated regulatory interactions and approvals; the ability to leverage existing clinical development infrastructure; the expected progression of the KIO-301 program and related clinical operations; the Company's plans for KIO-104; the expected capital requirements associated with its development programs, including the clinical trials of KIO-401 in CRAO and NAION; and Kiora's ability to secure additional financing to support its operations.

These forward-looking statements are based on management's current expectations and assumptions and involve risks and uncertainties that could cause actual results to differ materially from those expressed or implied. Such risks include, among others, uncertainties associated with clinical development and regulatory review; the possibility that results observed in previous clinical studies will not be reproduced in future trials; the cost, timing, enrollment, conduct and outcomes of clinical trials; the ability to obtain sufficient capital to fund planned development activities; the availability and terms of future financing; the potential dilution to existing stockholders; the Company's ability to execute its development strategy and licensing obligations; and other risks described under "Risk Factors" in Kiora's Annual Report on Form 10-K filed with the SEC on March 25, 2026, and in its subsequent filings with the SEC.

Kiora's results may also be affected by factors of which the Company is not currently aware. Forward-looking statements speak only as of the date of this release. Except as required by law, Kiora undertakes no obligation to publicly update or revise any forward-looking statement to reflect subsequent events, circumstances or changes in expectations.


Kiora Pharmaceuticals, Inc. Q4 2026 | Corporate Overview

 

2 This presentation is provided for informational purposes only. Some of the material and statements in this presentation are "forward-looking" statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements include, without limitation, statements concerning Kiora's planned development and potential commercialization of KIO-401; the anticipated timing, design, initiation, conduct, enrollment, endpoints and results of clinical trials in CRAO and NAION; the potential efficacy, safety, therapeutic benefit and commercial opportunity of KIO-401; the interpretation and applicability of clinical results generated by Senju Pharmaceutical in Japan; the potential for calpain inhibition in additional ophthalmic indications; anticipated regulatory interactions and approvals; the ability to leverage existing clinical development infrastructure; the expected progression of the KIO-301 program and related clinical operations; the Company's plans for KIO-104; the expected capital requirements associated with its development programs, including the clinical trials of KIO-401 in CRAO and NAION; and Kiora's ability to secure additional financing to support its operations. These forward-looking statements are based on management's current expectations and assumptions and involve risks and uncertainties that could cause actual results to differ materially from those expressed or implied. Such risks include, among others, uncertainties associated with clinical development and regulatory review; the possibility that results observed in previous clinical studies will not be reproduced in future trials; the cost, timing, enrollment, conduct and outcomes of clinical trials; the ability to obtain sufficient capital to fund planned development activities; the availability and terms of future financing; the potential dilution to existing stockholders; the Company's ability to execute its development strategy and licensing obligations; and other risks described under "Risk Factors" in Kiora's Annual Report on Form 10-K filed with the SEC on March 25, 2026, and in its subsequent filings with the SEC. Kiora's results may also be affected by factors of which the Company is not currently aware. Forward-looking statements speak only as of the date of this release. Except as required by law, Kiora undertakes no obligation to publicly update or revise any forward-looking statement to reflect subsequent events, circumstances or changes in expectations. Legal Disclaimers

 

3 Expanding Kiora’s Late-Stage Retinal Therapeutics Pipeline Added neuroprotective oral calpain inhibitor for CRAO with planned development in NAION KIO-401 New Phase 3-ready asset for Central Retinal Artery Occlusion (from Senju Pharmaceutical) § Global rights outside Asia § Small molecule calpain inhibitor § Positive Ph2 and Ph3 studies in Japan § Rare acute conditions with no currently approved treatments § Expansion opportunity beyond retina KIO-301 Existing Phase 2 asset for late-stage Retinitis Pigmentosa § Global partnerships with Laboratoires Théa, Senju Pharmaceutical & CKD Pharmaceuticals ⨠ $118M in potential development and regulatory milestones ⨠ $431M in potential commercial milestones ⨠ Royalties into the low 20% § Orphan Drug Designation § Phase 2 trial actively enrolling

 

4 Targeting the Retina to Slow or Stop Vision Loss or Restore Sight Development Pipeline of Proprietary Small Molecule Therapeutics Program Therapeutic Approach Indication Discovery Preclinical Phase 1 Phase 2 Phase 3 Commercial Rights Diseases of Retinal Ischemia KIO-401 Calpain Inhibitor CRAO Global Rights (outside Asia) NAION Inherited Retinal Disease KIO-301 Molecular Photoswitch Retinitis Pigmentosa (advanced) Partnered GloballyOther Inherited Retinal Diseases

 

5 KIO-401 Small Molecule Calpain Inhibitor

 

6 What are Calpains? Ischemia § Reduced blood flow § Decreased oxygen and glucose § Cellular stress Energy Failure § ↓ATP production § Failure of ion pumps § Loss of ionic homeostasis Calpain Activation § Excess Ca2+ causes dissociation of calpain from calpastatin, resulting in activated calpain § ↑Intracellular Ca2+ § Release from intracellular stores (ER, mitochondria) § Calpains are a family of calcium- activated cysteine proteases § Ubiquitously expressed in the cytoplasm of neurons and other cells § Calpains are endogenously inhibited by calpastatin Calcium Overload Proteolysis of Substrates and Cell Death § Cleavage of cytoskeletal proteins § Cleavage of ion channels, pumps and synaptic proteins § Mitochondrial dysfunction § Membrane destabilization § Neuronal cell death How Ischemia Activates Calpains Reduced perfusion Loss of ion homeostasis Increased Intracellular Ca2+ Calpain (active) Neuronal injury and cell death Ca2+ Calpastatin Calpastatin (inhibitor) Calpain (inactive) Calpastatin Calpain ↓ ATP X Ca2+ KIO-401 is a neuroprotective, oral small molecule calpain inhibitor, designed to protect retinal cells during ischemia Calpains: Calcium-Activated Proteases in Ischemia Injury

 

7 Central Retinal Artery Occlusion – An Acute Stroke of the Eye A Disease with No Available Treatments Unfortunately, ocular massage, thrombolytics (tPA) and other methods have limited-to-no efficacy Retinal ischemia activates calpains within 48 hours Patients typically recover ~2 lines after acute phase of disease Central retinal artery becomes occluded Blood flow stops Time is critical Early treatment may save vision Irreversible vision loss Limited oxygen is not enough Oxygen and nutrients are cut off Inner retina becomes ischemic Over 80% of patients have permanent, severe vision loss (worse than 20/200)

 

8 KIO-401: Phase 2a Study Design* Randomized, Dose Escalating, Multi-Center (Japan Only), Clinical Trial § Multicenter, two cohort, randomized, double-masked versus prospective registry § Baseline logMAR > 1.0 § Primary – Adverse Events (AEs), PK & labs § Secondary – Best Corrected Visual Acuity (ETDRS), Visual Fields § Imaging – Fundus, OCT, Fluorescein Angiography, Slit Lamp Study Design Endpoints Within 48 hrs of onset of symptoms Day 85 Visit 7 Cohort 1: KIO-401 (n=9) (100mg/day) Day 2 Visit 2 Day 57 Visit 6 Post Treatment Observation Cohort 2: KIO-401 (n=10) (200 mg/day) Day 8 Visit 3 Day 15 Visit 4 Day 29 Visit 5 Day 1 Visit 1 Prospective Observational Registry (n=9) * Study was conducted by Senju

 

9 KIO-401: Phase 2a Study Results* Mean Change from Baseline in ETDRS Letters p = 0.0398} p = 0.070 * Study was conducted by Senju

 

10 KIO-401: Phase 3 Study Design* Randomized, Controlled, 14 Sites (Japan Only) Clinical Trial § Multicenter, randomized, controlled, double-masked clinical trial § Baseline logMAR > 1.0 § Primary – Change from Baseline in Best Corrected Visual Acuity (BCVA) at 12 Weeks § Secondary – Change from Baseline in BCVA at Weeks 1-8, BCVA Responder Rates of 3 Lines at Week 12, Change from Baseline in Visual Field at Week 12, Treatment Emergent Adverse Events, ECG & labs § Imaging – Fundus, OCT, Fluorescein Angiography, Slit Lamp Study Design Endpoints Within 48 hrs of onset of symptoms Day 85 Visit 7 Day 2 Visit 2 Day 57 Visit 6 Post Treatment Observation KIO-401 (n=33) (200 mg/day) Day 8 Visit 3 Day 15 Visit 4 Day 29 Visit 5 Day 1 Visit 1 Placebo Control (n=30) Post Treatment Observation * Study was conducted by Senju

 

11 KIO-401: Phase 3 Study Results* Mean Change from Baseline in ETDRS Letters (Observed Data Only) p = 0.0949} p = 0.0195} B as el in e B C V A W o rs e T h an 2 0 /4 0 0 ** * Study was conducted by Senju ** 20/400 (logMAR = 1.3) is World Health Organization definition of legal blindness Responder analysis performed with Fisher’s Exact Test

 

12 KIO-401: Phase 3 Study Results* Categorical Visual Function Improvement * Study was conducted by Senju

 

13 Placebo KIO-401 (200 mg) (N=33) (N=33) n (%) events n (%) events Any TEAEs 10 (30.3) 17 13 (39.4) 21 Mild 7 (21.2) 13 9 (27.3) 17 Moderate 2 (6.1) 3 4 (12.1) 4 Severe 1 (3.0) 1 0 0 Serious TEAEs 3 (9.1) 3 1 (3.0) 1 TEAEs leading to death 0 0 0 0 Serious TEAEs except death 3 (9.1) 3 1 (3.0) 1 Significant TEAEs 0 0 0 0 Any study drug-related TEAEs 1 (3.0) 2 1 (3.0) 1 Mild 1 (3.0) 2 1 (3.0) 1 Moderate 0 0 0 0 Severe 0 0 0 0 Serious study drug-related TEAEs 0 0 0 0 Study drug-related TEAEs leading to death 0 0 0 0 Serious study drug-related TEAEs except death 0 0 0 0 Significant study drug-related TEAEs 0 0 0 0 * Study was conducted by Senju TEAE: Treatment Emergent Adverse Event KIO-401: Phase 3 Study Results* Safety – TEAE Summary (Treatment and Post-treatment Phases)

 

14 KIO-401: CRAO Planned Global Phase 3 Study Design Randomized, Controlled, up to 50 Sites (US/EU/Asia) Registration, Clinical Trial § Multicenter, randomized (1:1), controlled, double-masked clinical trial § Baseline BCVA worse than 20/200 (predefined subgroup for patients with BCVA worse than 20/400) § Primary – Best Corrected Visual Acuity (ETDRS) § Secondary – Visual Fields, AEs & labs § Imaging – Fundus, OCT, Fluorescein Angiography, Slit Lamp Study Design Endpoints Power Analysis Parameters: 80% power, p=0.05, 2-sided based on Senju P3 trial BCVA data (responder analysis will increase total patients by ~2x) Within 48 hrs of onset of symptoms Day 85 Visit 7 Day 2 Visit 2 Day 57 Visit 6 KIO-401 (n~72) (200 mg/day) Day 8 Visit 3 Day 15 Visit 4 Day 29 Visit 5 Day 1 Visit 1 Placebo Control (n~72) Post Treatment Observation Post Treatment Observation

 

15 Severe, Irreversible Vision Loss Sudden, painless vision loss in one eye – generally irreversible, with no effective treatment available1 Tx Initiated Within 2 Weeks Most patients present days to weeks after onset2 Roughly 10% keep worsening over the following four weeks3 No Current Treatment Today No medical or surgical treatment has been shown to improve the prognosis3 – steroids used off-label to help w edema 91% of specialists do not recommend routine treatment4 Limited Near-Term Options Many agents have been assessed but none has clearly shown benefit2,5 GLP-1 Link May Expand The Unmet Need. EU labels were updated in Sept ‘25 to add NAION for Ozempic, Rybelsus and Wegovy, and the UK followed in Feb ‘26.6,7 At roughly one extra case per 10,000 person-years against about 15 million US users, that is on the order of 1,000 additional US cases a year.8,9 1. WHO. Semaglutide medicines and risk of NAION. 27 Jun 2025. 2. Gibbons A, Henderson AD. Front Ophthalmol. 2022;2:848710. 3. Wu KY, Evoy F. NAION: Diagnosis and Management. AAO EyeNet. Aug 2022:29-31. 4. Bialer OY, Stiebel-Kalish H. Graefes Arch Clin Exp Ophthalmol. 2024;262:1399-1410. 5. Ischemic Optic Neuropathy: A Review of Current and Potential Future Pharmacotherapies. Pharmaceuticals. 2024;17(10):1281. 6. EMA PRAC, 6 Jun 2025; EU product information updated 30 Sep 2025. 7. MHRA Drug Safety Update, 5 Feb 2026. 8. KFF Health Tracking Poll, May 2024; 6% of US adults currently using a GLP-1. 9. Kiora calculation from references 6 and 8. NAION: Traditional Therapy Has No Current Options Sudden, Irreversible Vision Loss with No Approved Therapy

 

16 Established Treatment Oral neuroprotection aimed at ischemic injury, with proof of concept established in CRAO Proven Mechanism of Action Signal In CRAO patients legally blind at presentation, KIO-401 produced roughly 21 ETDRS letters, over four lines of vision, at 12 weeks versus about 9 on placebo (p=0.0195) Simple Route of Administration Oral administration – no surgery, no device, no IV procedure, no novel delivery system Rapid Path to Value Creation Target label expansion timing expected in 2033 The signal is strongest where the need is greatest. A known molecule with human proof of concept, given as a pill, in a disease with no approved therapy. The largest treatment effect was seen in patients who were already legally blind when they presented. KIO-401 Phase 3 study results, data on file. Figures shown are for the subgroup with baseline BCVA worse than 20/400 (WHO definition of legal blindness); the overall trended positive on its primary endpoint (p=0.0949). KIO-401 Is Poised to Offer a New Solution for NAION Pts An Oral Neuroprotectant with a Phase 3 Signal in Acute Ischemic Optic Injury

 

17 KIO-401: NAION Planned Phase 2b Study Design Randomized, Controlled, Registration-able, Clinical Trial § Multicenter, randomized (1:1), controlled, double-masked clinical trial § Baseline BCVA 20/50-20/500 § Visual Field Mean Deviation worse than or equal to -3.0 dB § Documented disc edema via fundus photography § Primary – Best Corrected Visual Acuity (ETDRS) § Secondary – Visual Fields, AEs & labs § Imaging – Fundus, OCT (RNFL), Slit Lamp Study Design Endpoints Power Analysis Parameters: 90% power, p=0.05, 2-sided based on 20-point difference in responder analysis Within 14 days of onset of symptoms Day 14 Visit 2 Day 168 Visit 7 KIO-401 (n~136) (200 mg/day) Day 28 Visit 3 Day 56 Visit 4 Day 112 Visit 6 Day 1 Visit 1 Placebo Control (n~136) Day 84 Visit 5

 

18 KIO-301

 

19 Neuron. 92, 100-113 (2016) trans cis § When photoreceptors die ⟶ downstream neurons (RGCs) are not capable of being activated § KIO-301 preferentially enters these RGCs and turns them “ON” in the presence of light* ‡ P2X7 is solely expressed on RGCs and amacrine cells in the retina * Visual light causes reversible isomeric shift, altering ion flux through Kv/HCN channels Intracellular KIO-301 P2X7‡ Extracellular KV/HCN Channels • KIO-301 lodges inside ion channels KIO-301 (MOA): Turns RGCs “ON” in the Presence of Light • In presence of light, alters ion flow, causing cell activation

 

20 In Patients with RP, Downstream Neurons Remain Viable § Many Inherited Retinal Diseases, including Retinitis Pigmentosa (RP), result in death of photoreceptors § Bipolar Cells and Retinal Ganglion Cells (RGCs) remain intact and retain ability to send signals to the brain Photoreceptors (rods and cones) sense light and pass the signal to special transmitting cells Bipolar cells and RGCs relay signal from rods and cones to the brain, where the image is “processed” RG C Bipolar Cell To the brain (connection intact) Cone Rod

 

21 KIO-301 Reanimates the Retina & Changes Behaviour Extensive Validation in Preclinical Models Neuron 2014: 81, 800-813. Light Dark Before KIO-301 After KIO-301

 

22 KIO-301: Phase 1b Study Design (ABACUS) Open Label, Single Ascending Dose Trial – 2 Sites (Australia) § Two Cohorts, non-randomized, open-label, single IVT injection per eye § Cohort 1 – NLP/BLP patients; Cohort 2 – HM/CF patients § Primary – AEs, PK & labs § Secondary – Assessment days (shown only for Cohort 1 above) is repeated for each cohort per eye; intensity & contrast assessment, kinetic perimetry, functional MRI, etc. § Safety review conducted by Investigators between after sentinel subject Study Design Endpoints Review N=6 3/cohort Binocular Day 90 Cohort 1: KIO-301 (7.5 µg) Day -30 to -2 Visit 1-Screening Day 29-30 Visit 5 Day 60 Day 120 Cohort 1: KIO-301 Contralateral (25 µg) Cohort 2: KIO-301 (25 µg) Cohort 2: KIO-301 Contralateral (50 µg) Day 1-3 Visit 2 Day 7-8 Visit 3 Day 14-15 Visit 4 Day -120 to -30 fMRI feasibility NLP – No Light Perception, BLP – Bare Light Perception, HM – Hand Motion, CF – Counting Fingers Nature Medicine v32, 1865–1870 (2026)

 

23 Changes in Vision & Patient QoL * *p<0.0 5 Kinetic Visual Field Mean Change from BL ± 80% CL Visual Acuity Mean ± SEM High Contrast Room Exit Test Mean ± SEM Quality of Life Survey Nature Medicine v32, 1865–1870 (2026)

 

24 Functional MRI Significant Increase of Cortical Activity NLP – No Light Perception, BLP – Bare Light Perception, CF – Counting Fingers Nature Medicine v32, 1865–1870 (2026)

 

25 KIO-301: Phase 2 Randomized, Controlled Clinical Trial (ABACUS-2) Multiple Dose, Bilateral Injection, Double-Masked, 36-Patient, Randomized, Controlled Trial § Clinical diagnosis of retinitis pigmentosa with visual acuity of logMAR1.6-3.0 OU (ULV) or logMAR ≥ 3.0 (NLP) § Recruitment in each Cohort begins with 6 patients for Safety Review Committee review § Primary (safety): AEs, PK, labs § Secondary (efficacy): functional vision (multi-luminance object identification), BCVA, peripheral field, FST, PROs Study Design * Also includes further test/retest validation to functional vision assessment OLE – Open Label Extension, BCVA – Best Corrected Visual Acuity, FST – Full-Field Stimulus Testing, PROs – Patient Reported Outcomes Day 42 Day 63 Ultra Low Vision (ULV, n=8) No Light Perception (NLP, n=4) Day 1 Day -30 to -1 Screening* Vehicle Controls (n=6) 50 µg KIO-301 Day 21 ULV (n=8) NLP (n=4) Vehicle Controls (n=6) 100 µg KIO-301 Day 84 Day 105 Day 165 Day 42 Day 63 Day 21 Day 84 Day 105 Day 165 // // Day 1 OLE for Controls Endpoints

 

26 KIO-301: Global Partnership Network Significant Upside Opportunity via Milestones and Royalties

 

27 Leadership and KOLs

 

28 Melissa Tosca, CPA Chief Financial Officer LinkedIn Keith Lane, MBA EVP – Clin Dev & Regulatory LinkedIn Brian M. Strem, PhD President & CEO LinkedIn Stefan Sperl, PhD EVP – CMC, NonClin, IP LinkedIn Leadership Team

 

29 Charlie Wykoff, MD, PhDTaiji Sakamoto, MD, PhD Allen Ho, MD, PhDRoger Goldberg, MD, PhD Brian Mac Grory, MD, PhD Christine Kay, MD, PhD Vitreo Retinal ASSOCIATES Mark Pennesi, MD, PhD Russell Van Gelder, MD, PhD KIO-301 KIO-401 Key Opinion Leaders

 

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