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100 μg KIO-301

Phase 2

Retinitis Pigmentosa | Small molecule | Ophthalmology |Kiora Pharmaceuticals, Inc.|Last Updated: Jun 18, 2026

Success Probability

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials1
Total Enrollment36

FDA Designations

No designations recorded

Clinical trial landscape

100 μg KIO-301 · 1 trial · 1 indication

Phase 2 1
NCT06628947A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis PigmentosaRetinitis Pigmentosa
RECRUITING36 Analytics
PHASE2RECRUITING
A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
Retinitis PigmentosaUnlock trial analytics

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 to 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)

LOWJun 18, 2026NCT06628947lastUpdatePostDate: changed
LOWJun 18, 2026NCT06628947lastUpdatePostDate: changed
LOWJun 18, 2026NCT06628947lastUpdatePostDate: changed

Frequently asked questions about 100 μg KIO-301

What is KIO-301 used for?

KIO-301 is an investigational small molecule being developed for the treatment of retinitis pigmentosa, a group of inherited eye disorders that cause progressive vision loss. It is administered via intravitreal injection and is currently being studied in patients with late-stage disease.

How does KIO-301 work?

KIO-301 is a small molecule designed to restore light sensitivity in the retina of patients with retinitis pigmentosa. It acts as a photoswitch, conferring light responsiveness to remaining retinal neurons that have lost their natural photoreceptors, thereby potentially restoring some visual function.

Who is developing 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 advancing the drug through clinical trials for the treatment of retinitis pigmentosa.

What phase is KIO-301 in?

KIO-301 is currently in Phase 2 clinical development. It is an investigational drug and has not yet been approved by regulatory authorities. The ongoing Phase 2 study is actively recruiting participants to evaluate its safety and efficacy in patients with late-stage retinitis pigmentosa.

What clinical trials is KIO-301 in?

KIO-301 is being evaluated in a Phase 2 clinical trial registered as NCT06628947. This randomized, double-blind, controlled study is recruiting 36 participants with late-stage retinitis pigmentosa at sites in Australia. The trial is currently in the recruiting stage and is not yet completed.

Is KIO-301 the same as 100 μg KIO-301?

Yes, 100 μg KIO-301 refers to a specific dose of the drug KIO-301. The Phase 2 clinical trial NCT06628947 is investigating this particular dose administered via intravitreal injection in patients with late-stage retinitis pigmentosa.