Kiora Pharmaceuticals Joins RARE-X Vision Consortium Promoting Collaboration and Development of New Treatments for Rare Ocular Disorders

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Kiora Pharmaceuticals (NASDAQ: KPRX) announced on October 30, 2025 that it joined Global Genes' RARE-X Vision Consortium, a corporate‑nonprofit collaboration to accelerate research and development of therapies for rare ocular disorders.

The consortium will build a de‑identified, individual‑level global data repository to support patient identification and recruitment, define meaningful outcome measures by aggregating functional, structural, and patient‑reported data, and promote inclusive study design with patient input. Kiora's CEO, Brian Strem, said the collaboration aims to increase the probability of advancing Kiora's experimental inherited retinal disease therapy toward patients.

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Clinical-trial strategy expert

Kiora joined the RARE-X Vision Consortium on Oct. 30, 2025, linking its inherited-retinal-disease program to a shared rare-vision data and stakeholder network.

The consortium creates a centralized, de-identified, individual-patient data repository and aims to ease two common bottlenecks: patient identification/recruitment and meaningful outcome-measure selection. By joining, Kiora gains access to aggregated functional, structural, and patient-reported datasets and to a community of advocacy, research, and industry partners listed in the release.

Progress depends on the consortium delivering high-quality, trial-ready cohorts and validated endpoints that correlate with real-world vision changes; these are explicit objectives of the Vision Consortium. Watch for concrete outputs such as expansion of the centralized dataset, defined outcome measures derived from the aggregated data, and new trial-ready patient cohorts as near-term milestones; these items will determine how quickly the membership translates into accelerated trial programs.

ENCINITAS, Calif. and WASHINGTON D.C., Oct. 30, 2025 /PRNewswire/ -- Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) and Global Genes today announced that Kiora has joined Global Genes' RARE-X Vision Consortium, a corporate-nonprofit partnership with a mission to promote and facilitate collaboration among advocacy, research, and industry stakeholders to advance research leading to vision-improving therapies. The Vision Consortium aims to transform the lives of people affected by rare ocular disorders by identifying clinical trial ready populations, ensuring meaningful outcome measures, and accelerating the development of clinical trial programs required to bring new treatments to patients.

The RARE-X Vision Consortium brings together multiple stakeholders with the goal to overcome shared challenges of successful drug development in a pre-competitive, open source environment. Data collected through the Vision Consortium programs will be available on a de-identified, individual patient level to researchers across academia, nonprofit organizations, and industry. Key solutions the Vision Consortium will provide include:

1. Patient Identification & Recruitment:  Rare disease clinical trials often face challenges to identify and enroll patients. Through collaboration and community-led participation, the Vision Consortium is building a global, centralized data repository for individuals affected by rare ocular diseases.
   
   
2. Outcome Measures for Clinical Trials:  Today's outcome measures may be inadequate for assessing meaningful changes in patients with rare ocular disorders, especially when loss of vision varies materially by condition. Through aggregating functional, structural, and patient reported outcome data, the Vision Consortium aims to identify endpoints that correlate with meaningful vision changes in patients' lives.
   
   
3. Inclusive Study Design & Planning:  Integrating the patient voice into the design and conduct of clinical trials requires early and consistent outreach to the patient community, including outreach to individuals who may not be initially inclined to participate in clinical research. The Vision Consortium brings the input of individuals and families impacted by rare disorders of vision loss and represents a global population with a wide diversity of ages, stages of vision loss, and perspectives.

"We view Global Genes' leadership and coordinated efforts as playing an invaluable role in aiding drug developers working to bring new treatment options to patients," said Brian Strem, Ph.D., President and CEO of Kiora. "As a developer of a novel treatment for inherited retinal diseases, we believe in the value of bringing people together with a common, shared goal; namely accelerating the pathway to new therapies for rare ocular disease. The Global Genes team employs concrete steps to increase the probability of advancing our experimental therapy to potentially help more patients."

"We welcome Kiora to the RARE-X Vision Consortium. The mission of the Vision Consortium is to promote collaboration across rare genetic ocular disorders among advocacy, research, and industry stakeholders to advance research leading to vision-enhancing therapies while improving the lives of those impacted by these disorders," said Karmen Trzupek, Senior Director of Scientific Programs at Global Genes. "We have assembled many of the leading rare disease advocacy groups in the ophthalmic space and look forward to expanding the Vision Consortium with foundational companies like Kiora in the private sector."

The Vision Consortium currently includes the following organizations:

• ADOA Association
• Curing Retinal Blindness Foundation
• Choroideremia Research Foundation
• Eyes on the Future
• Hope in Focus
• LHON Collective
• NPHP1 Family Foundation
• Save Sight Now Europe
• Snow Foundation
• Upstream Genes
• Usher Syndrome Coalition

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 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 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.

About Global Genes

Global Genes is a 501(c)(3) non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients, their families and disease communities globally. For nearly two decades, we've equipped rare disease patients and advocates with tools, training and support – to connect patients with needed resources, activate communities and advance research. Global Genes serves the more than 400 million people around the globe, and the nearly one in 10 Americans affected by rare diseases. With over 800 patient advocacy group members in our Global Advocacy Alliance, we work with patient advocates, industry partners and academia to build vital ecosystems to progress critical work in rare disease. Learn more at www.globalgenes.org.

About RARE-X

RARE-X is the research program of Global Genes. RARE-X provides a highly scalable approach for rare disease data collection, delivered in partnership with patient advocacy groups. RARE-X's global footprint includes more than 85 disorders from patients in over 90 countries. RARE-X's innovative, collaborative model is patient-driven with research grade data and is designed to accelerate research and advance urgently needed treatments. Learn more at www.globalgenes.org/about-us/about-rare-x/.

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, with 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 ability to timely complete planned initiatives for 2025, including Phase 2 clinical development of KIO-301 and KIO-104, the completion of enrollment and the timing of topline results from the ABACUS-2 Phase 2 trial, the potential for KIO-301 to be the first treatment options for patients with inherited degenerative diseases like RP, the potential for KIO-104 to reduce inflammation, the timing of topline results from the Phase 2 KLARITY trial of KIO-104, the potential for KIO-104 to apply to other retinal inflammatory diseases, expected trends for research and development and general and administrative spending in 2025, the expectations for market exclusivity of KIO-104, the potential proceeds that could be received from the Senju strategic partnership, and the expected endpoints for future KIO-301 trials. 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, 2025 or described in Kiora's other public filings, including on Form 10-Q filed with the SEC on August 8, 2025. 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 RARE-X

FAQ

What did Kiora (KPRX) announce on October 30, 2025 about rare ocular research?

Kiora announced it joined the RARE‑X Vision Consortium to collaborate on a global, de‑identified patient data repository and accelerate development of therapies for rare ocular disorders.

How will the RARE‑X Vision Consortium help Kiora's clinical trials for inherited retinal diseases?

The consortium aims to aid patient identification and recruitment and to identify meaningful outcome measures by aggregating functional, structural, and patient‑reported data.

What type of data will the RARE‑X Vision Consortium share and with whom?

The consortium will make de‑identified, individual‑level data available to researchers across academia, nonprofits, and industry.

Does Kiora expect the RARE‑X Vision Consortium to affect trial design timelines for KPRX programs?

Kiora said the consortium's collaborative resources are intended to accelerate the pathway to new therapies, potentially aiding trial readiness and design.

Which stakeholders are included in the RARE‑X Vision Consortium alongside Kiora (KPRX)?

The consortium includes rare disease advocacy groups, research organizations, and industry partners such as ADOA Association, Choroideremia Research Foundation, Usher Syndrome Coalition, and others.

Will patient privacy be protected in the RARE‑X Vision Consortium that Kiora joined?

Yes; the consortium will provide de‑identified individual patient‑level data to researchers to protect patient privacy while enabling research.