KIO-301 / Kiora Pharma - LARVOL DELTA

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) (clinicaltrials.gov) - P1/2 | N=48 | Recruiting | Sponsor: Kiora Pharmaceuticals, Inc. | N=12 ➔ 48

Enrollment change • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

ABACUS-2: A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa (clinicaltrials.gov) - P2 | N=36 | Recruiting | Sponsor: Kiora Pharmaceuticals, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

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) (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Kiora Pharmaceuticals, Inc. | Trial completion date: Sep 2025 ➔ Jul 2026

Trial completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

ABACUS-2: A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa (clinicaltrials.gov) - P2 | N=36 | Not yet recruiting | Sponsor: Kiora Pharmaceuticals, Inc. | Trial completion date: May 2026 ➔ Aug 2027 | Initiation date: Mar 2025 ➔ Aug 2025 | Trial primary completion date: Mar 2026 ➔ Aug 2027

Trial completion date • Trial initiation date • Trial primary completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

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) (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Kiora Pharmaceuticals, Inc. | Trial completion date: Jan 2025 ➔ Sep 2025 | Trial primary completion date: Dec 2024 ➔ Jun 2025

Trial completion date • Trial primary completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

ABACUS-2: A Phase II Study of Intravitreal KIO-301 in Patients with Late-stage Retinitis Pigmentosa (clinicaltrials.gov) - P2 | N=36 | Not yet recruiting | Sponsor: Kiora Pharmaceuticals, Inc.

New P2 trial • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Synthetic phototransduction with a light-responsive molecule (KIO-301) in advanced retinitis pigmentosa: the ABACUS-1 phase 1/2 trial (ARVO 2024) - P1/2 | "KIO-301 displayed an excellent safety profile in this phase 1/2 clinical trial. The concordance of subjective and objective efficacy signals and positive participant-reported experiences demonstrate a proof-of-concept for vision restoration with KIO-301 and motivate a larger, randomized, controlled trial of this novel, gene-agnostic small molecule therapy for advanced RP and potentially other inherited retinal diseases. Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand."

Metastases • P1/2 data • Glaucoma • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

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) (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Kiora Pharmaceuticals, Inc. | Trial completion date: Mar 2024 ➔ Jan 2025 | Trial primary completion date: Feb 2024 ➔ Dec 2024

Trial completion date • Trial primary completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Intravitreal Injection of “Photoswitch” Molecule (KIO 301) Improves Visual Function in Late-Stage Retinitis Pigmentosa Patients (AAO 2023) - No abstract available

Clinical • Late-breaking abstract • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

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) (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Kiora Pharmaceuticals, Inc. | Active, not recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

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) (clinicaltrials.gov) - P1/2 | N=6 | Active, not recruiting | Sponsor: Kiora Pharmaceuticals, Inc. | Recruiting ➔ Active, not recruiting | N=12 ➔ 6

Enrollment change • Enrollment closed • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of Small Molecule KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa (ABACUS) (clinicaltrials.gov) - P1/2 | N=6 | Recruiting | Sponsor: Kiora Pharmaceuticals, Inc. | Trial completion date: May 2023 ➔ Feb 2024 | Trial primary completion date: May 2023 ➔ Jan 2024

Trial completion date • Trial primary completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

An Intravitreal 'Photoswitch' Molecule (KIO-301) for Reanimation in Retinitis Pigmentosa: a first-in-human trial (ARVO 2023) - P1/2 | "KIO-301 is a first-in-class small molecule being developed to restore vision in this patient population. This work describes early safety data and patient reported outcomes from the first blind human to receive a single low-dose of KIO-301."

P1 data • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of Small Molecule KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa (ABACUS) (clinicaltrials.gov) - P1/2 | N=6 | Recruiting | Sponsor: Kiora Pharmaceuticals, Inc. | Not yet recruiting ➔ Recruiting | Initiation date: Aug 2022 ➔ Nov 2022

Enrollment open • Trial initiation date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa