Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
KIO-301 · 2 trials · 3 indications
Change in ophthalmic and non-ophthalmic adverse events
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.
| Arm | Type | Description |
|---|---|---|
| 50 μg KIO-301 | EXPERIMENTAL | 50 μg KIO-301 or placebo administered by IVT injection bilaterally (OU) once every 6 weeks for 3 administrations per participant. |
| 100 μg KIO-301 | EXPERIMENTAL | 100 μg KIO-301 or placebo administered by IVT injection OU once every 6 weeks for 3 administrations per participant. |
| Patients with Retinitis Pigmentosa and Choroideremia | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Placebo (Sterile Saline or Balanced Salt Solution) | OTHER | A control 50 μl injection of clear sterile saline or balanced salt solution (BSS) liquid. |
| 100 μg KIO-301 | DRUG | KIO-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-301 | DRUG | KIO-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. |
| KIO-301 | DRUG | KIO-301 intravitreal injection at ascending doses |
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 ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Ocugen Inc | OCGN | 3 | PHASE3 | OCU400, OCU410ST |
| Johnson & Johnson | JNJ | 1 | PHASE3 | Botaretigene sparoparvovec |
| Belite Bio, Inc. ADR | BLTE | 1 | PHASE2 | Tinlarebant |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE2 | SAR422459 |
| Kiora Pharmaceuticals, Inc. | KPRX | 1 | PHASE2 | KIO-301 |
| ProQR Therapeutics N.V. | PRQR | 1 | PHASE1 | QR-1123 |
KIO-301 is an investigational small molecule being developed for the treatment of retinitis pigmentosa, a rare inherited retinal disease. It is also being studied in choroideremia and other non-syndromic rod-dominant inherited retinal diseases. KIO-301 is administered by intravitreal injection directly into the eye and is intended for patients with late-stage disease.
KIO-301 is being developed by Kiora Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker KPRX. Kiora is a clinical-stage biopharmaceutical company focused on rare ophthalmic diseases, and KIO-301 is its small molecule program for inherited retinal conditions including retinitis pigmentosa.
KIO-301 is in Phase 2 clinical development. A Phase 2 study in late-stage retinitis pigmentosa is currently recruiting, and an earlier Phase 1/Phase 2 dose-escalation study in retinitis pigmentosa and choroideremia has been completed. KIO-301 is investigational and has not been approved by the FDA.
KIO-301 has been evaluated in two clinical trials. NCT06628947 is a recruiting Phase 2 study of intravitreal KIO-301 in patients with late-stage retinitis pigmentosa, enrolling 36 participants in Australia. NCT05282953, known as the ABACUS study, was a completed Phase 1/Phase 2 dose-escalation trial in retinitis pigmentosa and choroideremia that enrolled 32 participants.
KIO-301 is given as an intravitreal injection, meaning it is delivered directly into the vitreous of the eye. Both the recruiting Phase 2 trial in late-stage retinitis pigmentosa and the completed Phase 1/Phase 2 ABACUS dose-escalation study used this intravitreal route of administration in participants aged 18 years and older.