Kiora Pharmaceuticals
Kiora Pharmaceuticals is a clinical-stage biopharmaceutical company developing small molecule therapeutics for retinal diseases, including the molecular photoswitch KIO-301 for inherited retinal diseases (retinitis pigmentosa, choroideremia, Stargardt disease) and the DHODH inhibitor KIO-104 for retinal inflammation, serving patients and ophthalmology specialists through licensing partnerships.
- Company typePublic
- Founded1998
- HeadquartersSalt Lake City, United States
- Headcount11–50
- GTM typeB2B
- OfferingHardware or Manufacturing
What Kiora Pharmaceuticals does
Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) is a clinical-stage biopharmaceutical company headquartered in Encinitas, California and incorporated in Delaware, developing small molecule therapeutics for retinal diseases. The company targets critical pathways underlying vision-threatening conditions using two core technology platforms. KIO-301 is a molecular photoswitch that enters retinal ganglion cells and, upon activation by light, enables those surviving cells to transmit visual signals to the brain; it is being developed as a mutation-agnostic therapy for inherited retinal diseases including retinitis pigmentosa, choroideremia, and Stargardt disease. KIO-104 is a non-steroidal, locally delivered small molecule inhibitor of dihydroorotate dehydrogenase (DHODH) that suppresses T-cell-driven retinal inflammation; it is being developed for non-infectious uveitis, diabetic macular edema, and other inflammatory retinal indications.
The company has two Phase 2 trials actively enrolling — ABACUS-2 for KIO-301 in retinitis pigmentosa and KLARITY for KIO-104 in macular edema — with topline data anticipated in the first half of 2027. Kiora's business model is licensing- and milestone-driven: it has out-licensed global (excluding Asia) rights to KIO-301 to Laboratoires Théa in a deal worth up to $301 million (including a $16.0 million upfront payment recognized as 2024 revenue), and has an option agreement with Senju Pharmaceutical for Asian markets worth up to $110 million plus royalties. Both lead assets carry orphan drug designations — KIO-301 has US (March 2022) and EU (July 2024) orphan status, and KIO-104 has EU orphan status (May 2015) — and the KIO-100 family (active pharmaceutical ingredient in KIO-104) is protected by US Patent US-12,472,263 issued in 2025. Kiora is pre-revenue from product sales, ended 2025 with $17.1 million in cash, and closed a $24 million private placement in April 2026 (led by Perceptive Advisors and ADAR1 Capital Management), providing runway into late 2027 to bridge through anticipated clinical data readouts. The company has 11–50 employees and is led by Brian M. Strem, PhD (President & CEO), Melissa Tosca, CPA (CFO), and Stefan Sperl, PhD (EVP CMC and Operations).
Kiora Pharmaceuticals firmographics
Firmographics- Name
- Kiora Pharmaceuticals
- Legal name
- Kiora Pharmaceuticals, Inc.
- Website
- https://kiorapharma.com
- Company type
- Public
- Founded year
- 1998
- Operating status
- Operating
- Headcount range
- 11–50 employees
- Short description
- Kiora Pharmaceuticals is a clinical-stage biopharmaceutical company developing small molecule therapeutics for retinal diseases, including the molecular photoswitch KIO-301 for inherited retinal diseases (retinitis pigmentosa, choroideremia, Stargardt disease) and the DHODH inhibitor KIO-104 for retinal inflammation, serving patients and ophthalmology specialists through licensing partnerships.
- Ownership category
- akta.pro rank
Kiora Pharmaceuticals industry classification
Industry- Product category
- Ophthalmic Pharmaceuticals
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- Medicinal Chemicals & Botanical Products (2833)
- akta.pro primary industry
- Ophthalmology Specialty Pharmaceuticals (HLAIACAK)
- akta.pro secondary industries
- Ophthalmology Pharmaceuticals (HLAIAAAL), Rare Ophthalmology Disorder Therapies (HLAIAIAK)
Keywords
Where Kiora Pharmaceuticals is headquartered
LocationHeadquarters
- HQ city
- Salt Lake City
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Kiora Pharmaceuticals business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations
Revenue model
- Pharmaceutical Product Revenue: Pre-revenue clinical-stage company. Future revenue anticipated from commercialization of KIO-301 and KIO-104 upon regulatory approval. Commercial rights for KIO-301 outside Asia licensed to Laboratoires Théa.
- Upfront and Milestone Payments: The company received a $16.0 million upfront payment from Laboratoires Théa recognized as revenue in 2024. Option agreement with Senju Pharmaceutical for Asian markets worth up to $110 million plus royalties.
Go-to-market motion1 record
Distribution channels3 records
Marketing channels5 records
Kiora Pharmaceuticals product offering
Product offeringCore offering
Kiora Pharmaceuticals is a clinical-stage biopharmaceutical company developing small molecule therapeutics for retinal diseases. Its pipeline is anchored by KIO-301, a molecular photoswitch for inherited retinal diseases (retinitis pigmentosa, choroideremia, Stargardt disease) currently in Phase 2 ABACUS-2 trials, and KIO-104, a DHODH inhibitor for retinal inflammation (non-infectious uveitis, diabetic macular edema) in Phase 2 KLARITY trials. Both candidates are delivered via intravitreal injection.
Product overview
Kiora Pharmaceuticals is a clinical-stage biopharmaceutical company developing a focused pipeline of small molecule drugs for retinal diseases. The portfolio centers on two clinical-stage candidates: KIO-301, a molecular photoswitch that restores visual function in inherited retinal diseases by making retinal ganglion cells light-responsive, currently in Phase 2 ABACUS-2 trial for retinitis pigmentosa; and KIO-104, a DHODH inhibitor anti-inflammatory drug in Phase 2 KLARITY trial for macular edema and uveitis. Both products are delivered via intravitreal injection and target critical pathways underlying vision-threatening conditions with limited treatment options.
Differentiator
Problem solved
Functional benefit
Products and services
- KIO-301 A molecular photoswitch small molecule drug for inherited retinal diseases. When activated by light, it allows retinal ganglion cells to send signals to the brain, restoring visual function in patients with retinitis pigmentosa, choroideremia, and Stargardt disease. Delivered via intravitreal injection; mutation-agnostic approach covering 100+ genetic mutations.
- KIO-104 A non-steroidal anti-inflammatory small molecule inhibitor of dihydroorotate dehydrogenase (DHODH) for retinal inflammation. Suppresses T-cell replication and function to treat posterior non-infectious uveitis, diabetic macular edema, and proliferative vitreoretinopathy. Delivered via intravitreal injection; offers an alternative to chronic steroid use.
- KIO-100 Family A family of anti-inflammatory compounds including KIO-104, with the active pharmaceutical ingredient targeting retinal inflammation through DHODH inhibition. The KIO-100 family formulation patent (US-12,472,263) expands delivery options for KIO-104.
Quantifiable outcome
- Phase 1 ABACUS-1 trial met primary safety endpoint with no serious adverse events, dose-limiting toxicities, or structural retinal changes in 12 eyes of 6 participants
- +2 more outcomes
Companies that use Kiora Pharmaceuticals
Customer profileNamed customers2 records
Segments2 records
Ideal customer profiles2 records
Kiora Pharmaceuticals technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Kiora Pharmaceuticals partnerships and signals
Strategic signalPartnerships
Three partnerships are on record, tiered core, minor and flagship.
- Senju PharmaceuticalcoreOption agreement for development and commercialization of KIO-301 and KIO-104 in Asian markets. Deal worth up to $110 million plus royalties upon exercise of option.
- RARE-X Vision Consortium (Global Genes)minorCollaboration focused on advancing research for rare ocular disorders. Aims to improve clinical trial processes, patient identification, and outcome measures for rare retinal diseases.
- Laboratoires ThéaflagshipStrategic partnership through Théa Open Innovation program for KIO-301 development and commercialization. Théa acquired rights to KIO-301 for treatment of inherited retinal diseases in a deal potentially worth $301 million, including upfront payment and milestone payments. Théa handles commercialization in global markets less Asia.
Scale indicators8 records
Recent moves6 records
Expansion highlights6 records
Kiora Pharmaceuticals competitors and assessment
Company assessmentDirect peers
- ProQR Therapeutics: Clinical-stage biopharmaceutical company focused on inherited retinal diseases such as LCA10 (sepofarsen) and Usher syndrome. Directly comparable to Kiora as a small-cap, clinical-stage ophthalmology developer targeting rare IRDs with novel mechanisms.
- Aldeyra Therapeutics: Clinical-stage biopharmaceutical company developing novel small-molecule therapies for inflammatory ocular diseases (reproxalap for dry eye and uveitis). Comparable to Kiora's KIO-104 in anti-inflammatory ophthalmology positioning and clinical-stage status.
- EyePoint Pharmaceuticals: Clinical and commercial-stage ophthalmology company developing sustained-release intravitreal therapies for retinal diseases including DME, wet AMD, and uveitis (EYP-1901, DURYSTA). Directly comparable delivery mechanism (intravitreal) and retinal indication overlap with Kiora.
- Clearside Biomedical: Clinical-stage ophthalmology company developing suprachoroidal delivery of therapeutics for retinal diseases including macular edema and uveitis (CLS-AX, XIPERE). Comparable in retinal anti-inflammatory indication targeting and small-cap clinical-stage profile.
Emerging players
- REGENXBIO: Clinical and commercial-stage gene therapy company with RGX-314 for wet AMD and diabetic retinopathy, plus AAV programs for inherited retinal diseases. Competes indirectly with Kiora's KIO-301 by offering one-time disease-modifying treatment for retinal conditions.
- Adverum Biotechnologies: Clinical-stage gene therapy company developing ADVM-022 (intravitreal AAV) for wet AMD and diabetic macular edema. Comparable in retinal disease pipeline and intravitreal delivery approach, though using gene therapy rather than small molecules.
- Kodiak Sciences: Clinical-stage biopharmaceutical company developing KSI-301 (anti-VEGF antibody biopolymer conjugate) for wet AMD, DME, and retinal vein occlusion. Comparable as a small-cap clinical-stage retinal disease developer with a single lead asset targeting similar indications as KIO-104.
Broad incumbents
- Apellis Pharmaceuticals: Commercial-stage biopharmaceutical company with Syfovre (pegcetacoplan) for geographic atrophy and Empaveli for PNH. A larger, established retinal disease commercial player that competes in adjacent retinal indications and has deeper commercialization infrastructure than Kiora.
- Iveric Bio (Astellas): Acquired by Astellas in 2023, develops Izervay (avacincaptad pegol) for geographic atrophy. A larger commercial-stage retinal disease player with overlapping ophthalmology infrastructure; comparable to Théa as the type of strategic acquirer/licensee Kiora might face.
- Genentech / Roche: Global pharmaceutical leader with major retinal disease franchise (Lucentis, Vabysmo for wet AMD/DME). Represents the dominant incumbent in retinal anti-VEGF therapy that KIO-104 and partner-deployed KIO-301 must compete against for retina specialist mindshare.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat4 records
Key risks6 records
Key highlights6 records
Customer concentration
Kiora Pharmaceuticals social profiles
Digital presenceKiora Pharmaceuticals compliance and trust
Trust signalCompliance2 records
Kiora Pharmaceuticals financial estimates
Financial estimateRevenue estimate
Valuation estimate
Kiora Pharmaceuticals leadership team
Management profileNumber of profiles
Profiles5 records
Kiora Pharmaceuticals funding detail
Funding detailFunding overview
Funding rounds17 records
Investors13 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Kiora Pharmaceuticals M&A and investment
M&A and investmentM&A1 record
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Kiora Pharmaceuticals
What does Kiora Pharmaceuticals do?
Kiora Pharmaceuticals is a clinical-stage biopharmaceutical company developing small molecule therapeutics for retinal diseases. Its pipeline is anchored by KIO-301, a molecular photoswitch for inherited retinal diseases (retinitis pigmentosa, choroideremia, Stargardt disease) currently in Phase 2 ABACUS-2 trials, and KIO-104, a DHODH inhibitor for retinal inflammation (non-infectious uveitis, diabetic macular edema) in Phase 2 KLARITY trials. Both candidates are delivered via intravitreal injection.
Is Kiora Pharmaceuticals a public or private company?
Kiora Pharmaceuticals is a public company. It is classified as public and is currently operating.
When was Kiora Pharmaceuticals founded?
Kiora Pharmaceuticals was founded in 1998. It employs 11 to 50 people.
Where is Kiora Pharmaceuticals based?
Kiora Pharmaceuticals is headquartered in Salt Lake City, United States, in the North America region.
How does Kiora Pharmaceuticals make money?
Two revenue lines are on record. Pharmaceutical Product Revenue is the primary driver. The others are upfront and Milestone Payments.
Who are Kiora Pharmaceuticals's main competitors?
Direct peers on record are ProQR Therapeutics, Aldeyra Therapeutics, EyePoint Pharmaceuticals and Clearside Biomedical. Emerging players are REGENXBIO, Adverum Biotechnologies and Kodiak Sciences. Broad incumbents are Apellis Pharmaceuticals, Iveric Bio (Astellas) and Genentech / Roche.
Does Kiora Pharmaceuticals have an API?
No public API is recorded for Kiora Pharmaceuticals.
What industry is Kiora Pharmaceuticals in?
Kiora Pharmaceuticals's product category is Ophthalmic Pharmaceuticals. Its primary akta.pro industry code is HLAIACAK, Ophthalmology Specialty Pharmaceuticals, with a secondary code of HLAIAAAL, Ophthalmology Pharmaceuticals. Its NAICS code is 541714 and its SIC code is 2833.