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KIO-104

Phase 2

Macular Edema | Small molecule | Other |Kiora Pharmaceuticals, Inc.|Last Updated: Apr 30, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDBiomarker
Total Trials1
Total Enrollment28
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06825702Safety, Tolerability and Efficacy of Intravitreal KIO-104 in Patients With Macular EdemaPHASE2 RECRUITING 28May 1, 2025Dec 1, 2026Apr 30, 20264 Australia
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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 — 85 Years
SexALL
Healthy VolunteersNo
Study Sites4

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)
LOWMay 26, 2026NCT06825702primaryCompletionDate: changed
LOWMay 24, 2026NCT06825702studyFirstPostDate: changed