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KIO-301

Phase 1

Retinitis Pigmentosa | Small molecule | Rare Disease |Kiora Pharmaceuticals, Inc.|Last Updated: May 4, 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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment32
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05282953A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)PHASE1 COMPLETED 32Nov 10, 2022Mar 24, 2026May 4, 20263 Australia
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Study Endpoints
Primary Endpoints
Main Study Primary Outcome Measure
84 days.

Number of participants with treatment-related adverse events as assessed by overall changes in retinal thickness and appearance, clinical chemistry and hematology, and other eye assessments.

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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Patients with Retinitis Pigmentosa and ChoroideremiaEXPERIMENTAL -
Interventions
NameTypeDescription
KIO-301DRUGKIO-301 intravitreal injection at ascending doses
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Eligibility Criteria
Age Range18 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites3

Main Study: Inclusion Criteria 1. Be aged 18 to 80 years at Visit 1 of either sex and of any race. 2. Be willing and able to provide informed consent either written, or if the Participant is not able to read, provide consent as stipulated by local laws and Human Research Ethics Committee (HREC) gu...

Countries:Australia
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Recent Changes (Last 90 Days)
MEDIUMJun 4, 2026NCT05282953TRIAL_REMOVED: changed
MEDIUMJun 4, 2026NCT05282953TRIAL_REMOVED: changed
MEDIUMJun 4, 2026NCT05282953TRIAL_REMOVED: changed
MEDIUMJun 4, 2026NCT05282953TRIAL_REMOVED: changed
MEDIUMJun 4, 2026NCT05282953TRIAL_REMOVED: changed
HIGHMay 26, 2026NCT05282953Status: RECRUITING → COMPLETED
LOWMay 24, 2026NCT05282953studyFirstPostDate: changed