Drugs / KIO-301
last change Aug 2026 re-read 3 minutes ago

KIO-301

also known as KIO-301, (E)- · KIO-301, TRANS-

Small molecule

Developed for
choroideremia · retinitis pigmentosa
Investigated by
Kiora Pharmaceuticals, Inc.

Trials 1

2023202420252026
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT05282953 Nov 2022 → Aug 2023 choroideremia, retinitis pigmentosa Kiora Pharmaceuticals, Inc. Completed Met primary

News releases announcing trial results or a regulatory action · 11

DateIssuerRelease
2026-04-14 Kiora Pharmaceuticals, Inc. Results Kiora's Phase 1 ABACUS Study of KIO-301 in Retinitis Pigmentosa Published in Nature Medicine; Phase 2 Trial Underway kiorapharma.com ↗
2024-11-08 Kiora Pharmaceuticals, Inc. Regulatory Kiora Pharmaceuticals Reports Third Quarter Results; Retinal Disease Pipeline Advancing Two Phase 2 Studies kiorapharma.com ↗
Regarding KIO-301, we recently received approval to initiate ABACUS-2, a 36-patient, multi-center, double-masked, randomized, controlled, multi-dose study in patients with ultra-low vision or no light perception due to retinitis pigmentosa.
2024-10-29 Kiora Pharmaceuticals, Inc. Regulatory Kiora Pharmaceuticals Receives Investigational New Drug Approval to Initiate ABACUS-2, a Phase 2 Clinical Trial of KIO-301 for the Treatment of Retinitis Pigmentosa kiorapharma.com ↗
2024-07-30 Kiora Pharmaceuticals, Inc. Regulatory Kiora Pharmaceuticals Receives European Orphan Medicinal Product Designation for KIO-301 for the Treatment of Inherited Retinal Dystrophies kiorapharma.com ↗
Kiora Pharmaceuticals (NASDAQ: KPRX) ("Kiora" or the "Company") today announced it has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for the treatment of a group of inherited retinal diseases (IRDs) that include retinitis pigmentosa (RP), choroideremia and more.
2024-05-06 Kiora Pharmaceuticals, Inc. Results Kiora Pharmaceuticals Reports Additional Clinical Data for KIO-301 Showing a Statistically Significant Increase in Brain Activity in the Visual Cortex in Patients with Retinitis Pigmentosa kiorapharma.com ↗
Additional analysis of functional MRI data demonstrated a statistically significant increase in neural activity over baseline within the brain's visual processing center.
2023-11-04 Kiora Pharmaceuticals, Inc. Results AAO Late-Breaking: Kiora's Small Molecule Photoswitch Demonstrates Meaningful Vision Improvements in Blind Patients with Retinitis Pigmentosa kiorapharma.com ↗
Kinetic visual field (Goldmann perimetry) increased significantly from baseline at days 7 and 14 post treatment (p < 0.05).
2023-10-03 Kiora Pharmaceuticals, Inc. Regulatory Kiora Pharmaceuticals Receives Investigational New Drug Application Approval to Expand Phase 1b Study of KIO-301 in Inherited Retinal Diseases; To Enroll Patients with Choroideremia and Additional Patients with Late-Stage Retinitis Pigmentosa kiorapharma.com ↗
2023-05-01 Kiora Pharmaceuticals, Inc. Results Kiora Pharmaceuticals Presents Encore Imaging Results of KIO-301 for Retinitis Pigmentosa at ASNR 2023 Meeting kiorapharma.com ↗
Early results of the ABACUS phase 1b trial show KIO-301 delivered intravitreally in patients with RP may restore functional vision.
2023-04-27 Kiora Pharmaceuticals, Inc. Results Kiora's Investigational Treatment for Retinitis Pigmentosa, KIO-301, Demonstrates Visual Function Restoration in Patients Who Are Blind kiorapharma.com ↗
Kiora Pharmaceuticals (NASDAQ: KPRX) presented preliminary results from an ongoing clinical trial showing its investigational drug, KIO-301, has the potential to restore light perception in patients who are blind or living with ultra-low vision due to retinitis pigmentosa.
2022-06-27 Kiora Pharmaceuticals, Inc. Regulatory Kiora Pharmaceuticals Receives Clinical Trial Approval for First-in-Human Evaluation of KIO-301 to Restore Vision in Patients with Retinitis Pigmentosa kiorapharma.com ↗
Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) ("Kiora" or the "Company") received approval to initiate a first-in-human clinical trial for KIO-301, which is intended to restore lost vision in patients with Retinitis Pigmentosa, a rare, inherited genetic eye disease that causes severe loss of functional vision due to degeneration of the retinal photoreceptors (rods and cones).
2022-03-18 Kiora Pharmaceuticals, Inc. Regulatory Kiora Pharmaceuticals Granted Orphan Drug Designation for KIO-301, an Investigational Drug for the Treatment of Retinitis Pigmentosa (RP) kiorapharma.com ↗
Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX), ("Kiora" or the "Company") has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its investigational treatment of Retinitis Pigmentosa, KIO-301.

All press releases naming this drug 21 releases

DateIssuerRelease

Evidence & citations 5 cited values

Every value below carries the sentence it was read from. 3 sources stand behind the page.

FieldValueCited text
Known as KIO-301 ClinicalTrials.gov intervention name — accepted as the source's own label NCT05282953 ↗
Known as KIO-301, (E)- ChEMBL registry synonym — accepted as the source's own label CHEMBL4796560 ↗
Known as KIO-301, TRANS- ChEMBL registry synonym — accepted as the source's own label CHEMBL4796560 ↗
Modality Small molecule “A small azobenzene photoswitch molecule (KIO-301)” PMID 41981307 ↗ Apr 2026
Route Intravitreal “KIO-301 was administered by intravitreal injection to 12 eyes of 6 participants” PMID 41981307 ↗ Apr 2026