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KIO-301

Phase 1

Retinitis Pigmentosa | Small molecule | Rare Disease |Kiora Pharmaceuticals, Inc.|Last Updated: May 4, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment32

FDA Designations

No designations recorded

Clinical trial landscape

KIO-301 · 1 trial · 3 indications

Phase 1 1
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)Retinitis Pigmentosa
COMPLETED32 Analytics
PHASE1COMPLETED
A 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)
Retinitis PigmentosaUnlock trial analytics

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 to 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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Frequently asked questions about KIO-301

What is KIO-301 used for?

KIO-301 is an investigational small molecule being developed for the treatment of retinitis pigmentosa, a rare inherited retinal disease. It is administered intravitreally, meaning it is injected into the eye. The drug is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who makes KIO-301?

KIO-301 is being developed by Kiora Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol KPRX. The company is conducting clinical research on this investigational drug for the treatment of retinitis pigmentosa.

What phase is KIO-301 in?

KIO-301 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The drug is being studied for safety, tolerability, and efficacy in patients with retinitis pigmentosa.

What clinical trials is KIO-301 in?

KIO-301 has one completed clinical trial, identified as NCT05282953, titled the ABACUS study. This was a Phase I/II dose-escalating study evaluating the safety, tolerability, and efficacy of KIO-301 administered intravitreally to patients with retinitis pigmentosa and choroideremia. The trial enrolled 32 participants in Australia.

How does KIO-301 work?

KIO-301 is a small molecule designed to treat retinitis pigmentosa. It is administered intravitreally, directly into the eye. The specific molecular target of KIO-301 has not been disclosed in available information.