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KIO-104

Phase 2

Macular Edema | Small molecule | Ophthalmology |Kiora Pharmaceuticals, Inc.|Last Updated: Jun 16, 2026

Target and mechanism

Molecular targetDHODH
Target classEnzyme
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment28

FDA Designations

No designations recorded

Clinical trial landscape

KIO-104 · 1 trial · 1 indication

Phase 2 1
NCT06825702Safety, Tolerability and Efficacy of Intravitreal KIO-104 in Patients With Macular EdemaMacular Edema
RECRUITING28 Analytics
PHASE2RECRUITING
Safety, Tolerability and Efficacy of Intravitreal KIO-104 in Patients With Macular Edema
Macular EdemaUnlock trial analytics

Study Endpoints

Primary Endpoints

Safety and Tolerability
23months

The primary objective is safety and tolerability. The incidence of AEs will be presented by System Organ Class (SOC) and Preferred Term (PT) by dose cohorts.

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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Low Dose KIO-104: 2-weeklyEXPERIMENTAL2-weekly dosing
High Dose KIO-104: 2-weeklyEXPERIMENTAL2-weekly dosing
Low or High Dose KIO-104: 2-weeklyEXPERIMENTAL2-weekly dosing
Low or High Dose KIO-104: 4-weeklyEXPERIMENTAL4-weekly dosing

Interventions

NameTypeDescription
KIO-104DRUGKIO-104 is an ophthalmic formulation for IVT injection of the active pharmaceutical ingredient (API) KIO-100, a novel, non-steroidal, small molecule inhibitor of dihydroorotate dehydrogenase (DHODH).
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Eligibility Criteria

Age Range18 Years to 85 Years
SexALL
Healthy VolunteersNo
Study Sites6

Inclusion Criteria: Participants must meet all the following criteria: 1. Be aged 18 to 85 years inclusive at the time of consent. 2. Provide informed consent prior to any study procedures, as stipulated by local laws, Ethics Committee (EC) and Regulatory Authority (RA) guidelines. 3. Be willing a...

Countries:Australia
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Recent Changes (Last 90 Days)

LOWJun 16, 2026NCT06825702lastUpdatePostDate: changed
LOWJun 16, 2026NCT06825702lastUpdatePostDate: changed
LOWJun 16, 2026NCT06825702lastUpdatePostDate: changed

Frequently asked questions about KIO-104

What is KIO-104 used for?

KIO-104 is an investigational small molecule being developed for the treatment of macular edema, a condition characterized by fluid accumulation in the macula of the eye. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.

What does KIO-104 target?

KIO-104 targets dihydroorotate dehydrogenase (DHODH), an enzyme involved in pyrimidine biosynthesis. By inhibiting DHODH, KIO-104 may modulate immune and inflammatory responses implicated in the pathogenesis of macular edema.

Who is developing KIO-104?

KIO-104 is being developed by Kiora Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol KPRX. The company is conducting clinical trials to evaluate the safety and efficacy of KIO-104 in patients with macular edema.

What phase is KIO-104 in?

KIO-104 is currently in Phase 2 clinical development. It is an investigational drug and has not yet received FDA approval. The ongoing Phase 2 trial is recruiting participants to assess the safety, tolerability, and efficacy of intravitreal KIO-104 in patients with macular edema.

What clinical trials is KIO-104 in?

KIO-104 is being evaluated in a Phase 2 clinical trial with the identifier NCT06825702. This study, titled 'Safety, Tolerability and Efficacy of Intravitreal KIO-104 in Patients With Macular Edema,' is recruiting 28 participants in Australia. The trial is controlled but not randomized or double-blinded.