Company Description
Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) is a clinical-stage biotechnology company focused on developing advanced therapies for retinal disease. According to the company’s public disclosures, Kiora targets critical pathways underlying retinal diseases using small-molecule drug candidates designed to slow, stop, or restore vision loss. The company is associated with the pharmaceutical preparation manufacturing industry within the broader manufacturing sector.
Kiora describes itself as a developer of therapies for both inherited and inflammatory retinal conditions. Its pipeline centers on two main product candidates: KIO-301 and KIO-104. These programs are in clinical development and are being evaluated in multiple Phase 2 trials, as detailed in recent press releases and SEC filings.
KIO-301: Molecular photoswitch for inherited retinal diseases
KIO-301 is being developed for the treatment of retinitis pigmentosa, with plans described by the company to expand development into choroideremia and Stargardt disease. Kiora states that KIO-301 is a molecular photoswitch with the potential to restore vision in patients with inherited and/or age-related retinal degeneration. In company materials, KIO-301 is characterized as a new type of therapy for inherited retinal diseases that targets surviving cells in the retina.
For patients with conditions such as retinitis pigmentosa, rods and cones degenerate over time, leading to severe vision loss. Kiora reports that KIO-301 is designed to enter surviving retinal ganglion cells and, when activated by light, enable these cells to send signals to the brain. The company’s ABACUS-2 Phase 2 clinical trial is described as a multi-center, randomized, controlled study in patients with ultra-low vision or no light perception due to retinitis pigmentosa, using validated functional vision assessments as key endpoints.
KIO-104: DHODH inhibitor for retinal inflammation
KIO-104 is being developed for the treatment of retinal inflammation and a range of inflammatory retinal diseases. Kiora describes KIO-104 as a next-generation, non-steroidal, immuno-modulatory, small-molecule inhibitor of the mitochondrial enzyme dihydroorotate dehydrogenase (DHODH). The company states that KIO-104 is formulated for local delivery into the retina and is intended as an alternative to steroids or systemic anti-inflammatory drugs.
According to Kiora, KIO-104 works by inhibiting DHODH, which plays a crucial role in the synthesis of key building blocks of DNA and RNA. Without these building blocks, T-cell replication is significantly reduced, and their function is dampened, which may reduce inflammation. Kiora reports that KIO-104 is being studied in inflammatory retinal conditions such as posterior non-infectious uveitis and diabetic macular edema, and that a Phase 2 clinical trial, known as the KLARITY study, is actively screening and dosing patients with macular edema associated with several inflammatory retinal diseases.
Clinical development and trial programs
Company press releases describe two actively enrolling Phase 2 clinical trials for retinal diseases:
- ABACUS-2 (KIO-301): A Phase 2, randomized, controlled clinical trial in patients with retinitis pigmentosa, focused on vision restoration. Kiora notes that ABACUS-2 uses a validated efficacy endpoint to assess functional vision outcomes in patients with moderate to advanced vision loss due to retinal degeneration.
- KLARITY (KIO-104): An open-label, multicenter Phase 2 clinical trial evaluating KIO-104 in patients with retinal inflammation and macular edema across several inflammatory retinal diseases, including posterior non-infectious uveitis and diabetic macular edema. The study is described as a two-stage, multi-dose design that will investigate KIO-104 in up to 28 patients at different dose levels.
Kiora has reported continued recruitment, screening, and dosing of participants in both ABACUS-2 and KLARITY. The company also notes that ABACUS-2 incorporates a validated functional vision assessment that may serve as an approvable endpoint for potential future registration trials of KIO-301.
Intellectual property and patent protection
Kiora has announced multiple U.S. patents related to KIO-104 and its broader KIO-100 family of anti-inflammatory compounds. One patent (US Patent No. 12,364,680) covers KIO-104 for the treatment of a wide range of ocular diseases, including ocular inflammation, uveitis, age-related macular degeneration, and complications from refractive surgery. The company states that this patent also covers varying dosing schedules, necessary excipients, and methods for optimizing treatment of ocular inflammatory diseases, and that it is expected to extend market exclusivity for KIO-104 into 2043, absent patent term extensions.
Another patent (US-12,472,263) is described as covering additional and novel formulations of the KIO-100 family of compounds, including a novel formulation of the active pharmaceutical ingredient in KIO-104. According to Kiora, this patent allows for additional delivery options and expands the potential therapeutic utility of the active ingredient in KIO-104.
Strategic collaborations and partnerships
Kiora has disclosed several strategic relationships connected to its retinal disease pipeline. For KIO-301, the company reports an exclusive worldwide co-development and commercialization agreement (excluding Asia) with Théa Open Innovation (Laboratoires Théa or Théa) for the treatment of retinal diseases. In addition, Kiora has granted Senju Pharmaceutical Co., Ltd. an exclusive option to enter into a development and commercialization agreement for KIO-301 in key Asian markets, including Japan and China.
Under the option agreement with Senju, Kiora states that Senju has a defined period to exercise its option after topline data from the ABACUS-2 Phase 2 trial are reported. If the option is exercised, Senju would be responsible for development, regulatory, and commercial activities in the licensed territory, with potential up-front payments, regulatory and commercial milestones, and tiered royalties on net sales as described by Kiora. The company also notes that the combined potential value of its strategic partnerships related to KIO-301, including Théa and Senju, exceeds $400 million, plus royalties on sales, based on its public statements.
Engagement in rare ocular disease initiatives
Kiora has joined the RARE-X Vision Consortium, a program of Global Genes focused on rare ocular disorders. In public communications, the company describes its participation as part of a corporate–nonprofit partnership aimed at promoting collaboration among advocacy, research, and industry stakeholders to advance research leading to vision-improving therapies. The Vision Consortium is described as working to identify clinical trial–ready populations, define meaningful outcome measures, and accelerate the development of clinical trial programs for rare ocular diseases.
Through this involvement, Kiora aligns its development of therapies for inherited retinal diseases with broader efforts to address challenges in patient identification, recruitment, outcome measure development, and inclusive study design for rare ocular conditions.
Financial reporting and SEC filings
Kiora files periodic and current reports with the U.S. Securities and Exchange Commission (SEC). Recent Form 8-K filings dated August 8, 2025, and November 7, 2025, reference press releases announcing financial results for the quarters ended June 30, 2025, and September 30, 2025, respectively, along with updates on clinical development progress. These filings indicate that Kiora continues to operate as a reporting company and provide investors with information about its financial condition, research and development spending, and collaboration-related revenues and credits.
The company has publicly discussed its cash, cash equivalents, and short-term investments, collaboration receivables, and tax-related receivables, as well as research and development and general and administrative expenses. It has also described the impact of collaboration credits from partners such as Théa and the recognition of deferred collaboration revenue related to the Senju option fee.
Stock listing and sector classification
Kiora Pharmaceuticals, Inc. trades on the NASDAQ under the ticker symbol KPRX. Based on the provided classification, the company is associated with the pharmaceutical preparation manufacturing industry within the manufacturing sector. As a clinical-stage biotechnology company, its value proposition is centered on the development of small-molecule therapies for retinal diseases rather than on commercialized products.
Investor communications
In its press releases, Kiora notes that, in addition to news releases and SEC filings, it expects to post information that could be relevant to investors on its website and social media accounts, and it encourages investors to follow these channels or subscribe to email alerts. The company also participates in industry and investor conferences, where it showcases its pipeline of therapies targeting inherited and inflammatory retinal diseases.
FAQs about Kiora Pharmaceuticals (KPRX)
- What does Kiora Pharmaceuticals do?
Kiora Pharmaceuticals is a clinical-stage biotechnology company developing small-molecule therapies for retinal diseases. Its programs focus on conditions such as inherited retinal degenerations and inflammatory retinal diseases, with the goal of slowing, stopping, or restoring vision loss, as described in the company’s public materials.
- What are Kiora’s main drug candidates?
According to Kiora, its main drug candidates are KIO-301 and KIO-104. KIO-301 is a molecular photoswitch being developed for retinitis pigmentosa, with plans to expand into choroideremia and Stargardt disease. KIO-104 is a non-steroidal, immuno-modulatory small-molecule DHODH inhibitor being developed for retinal inflammation and inflammatory retinal diseases.
- Which retinal diseases is KIO-301 targeting?
Kiora states that KIO-301 is being developed for retinitis pigmentosa and is planned for expansion into choroideremia and Stargardt disease. These are inherited retinal diseases characterized by retinal degeneration and vision loss.
- How does KIO-301 work, according to Kiora?
The company describes KIO-301 as a molecular photoswitch that targets surviving retinal cells, specifically retinal ganglion cells. When activated by light, KIO-301 is intended to enable these cells to send signals to the brain, with the potential to restore visual function in patients with inherited and/or age-related retinal degeneration.
- What conditions is KIO-104 being developed to treat?
Kiora reports that KIO-104 is being developed for retinal inflammation and several inflammatory retinal diseases, including posterior non-infectious uveitis and diabetic macular edema. The company also notes that a patent covering KIO-104 references ocular inflammation, uveitis, age-related macular degeneration, and complications from refractive surgery.
- How does KIO-104 work?
According to Kiora, KIO-104 is a small-molecule inhibitor of the mitochondrial enzyme DHODH. By inhibiting DHODH, KIO-104 reduces the synthesis of key building blocks of DNA and RNA, which decreases T-cell replication and dampens T-cell–driven inflammation, potentially reducing retinal inflammation.
- What clinical trials is Kiora conducting?
The company has described two active Phase 2 trials: ABACUS-2, a randomized, controlled trial of KIO-301 in patients with retinitis pigmentosa, and KLARITY, an open-label, multicenter Phase 2 trial of KIO-104 in patients with retinal inflammation and macular edema across several inflammatory retinal diseases.
- Does Kiora have any strategic partnerships?
Yes. Kiora reports an exclusive worldwide co-development and commercialization agreement (excluding Asia) with Théa Open Innovation for KIO-301 in retinal diseases. It has also granted Senju Pharmaceutical an exclusive option to enter into a development and commercialization agreement for KIO-301 in certain Asian markets, including Japan and China.
- Is Kiora involved in rare disease initiatives?
In collaboration with Global Genes, Kiora has joined the RARE-X Vision Consortium. This consortium is described as a corporate–nonprofit partnership focused on advancing research and clinical trial readiness for rare ocular disorders, including inherited retinal diseases.
- On which exchange does Kiora trade and what is its ticker?
Kiora Pharmaceuticals, Inc. trades on the NASDAQ under the ticker symbol KPRX, as indicated in its press releases and regulatory filings.