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100 μg KIO-301

Phase 2

Retinitis Pigmentosa | Small molecule | Ophthalmology |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
RandomizedDouble-BlindCONTROLLEDBiomarker
Total Trials1
Total Enrollment36
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06628947A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis PigmentosaPHASE2 RECRUITING 36Aug 29, 2025Aug 1, 2027Apr 30, 20265 Australia
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Study Endpoints
Primary Endpoints
To assess the safety and tolerability of multiple doses of KIO-301 administered by IVT injection bilaterally of KIO-301 in patients with late-stage RP.
Baseline (Week 1/pre-dose) to Week 25 (12 weeks post 3rd IVT administration of KIO-301)

Change in ophthalmic and non-ophthalmic adverse events

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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
50 μg KIO-301EXPERIMENTAL50 μg KIO-301 or placebo administered by IVT injection bilaterally (OU) once every 6 weeks for 3 administrations per participant.
100 μg KIO-301EXPERIMENTAL100 μg KIO-301 or placebo administered by IVT injection OU once every 6 weeks for 3 administrations per participant.
Interventions
NameTypeDescription
Placebo (Sterile Saline or Balanced Salt Solution)OTHERA control 50 μl injection of clear sterile saline or balanced salt solution (BSS) liquid.
100 μg KIO-301DRUGKIO-301 drug product is an ophthalmic formulation of the drug substance KIO-300-Cl in sulfobutylether-β-cyclodextrin, sucrose, phosphate buffer salts and water suitable for IVT injection. The drug substance KIO-300-Cl is a quaternary ammonium chloride salt of the active compound KIO-300.
50 μg KIO-301DRUGKIO-301 drug product is an ophthalmic formulation of the drug substance KIO-300-Cl in sulfobutylether-β-cyclodextrin, sucrose, phosphate buffer salts and water suitable for IVT injection. The drug substance KIO-300-Cl is a quaternary ammonium chloride salt of the active compound KIO-300.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites5

Inclusion Criteria: 1. Be aged 18 years or older 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 and able to follow all study instructions, attend all study ...

Countries:Australia
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06628947primaryCompletionDate: changed
LOWMay 24, 2026NCT06628947studyFirstPostDate: changed