AGN-151597 / AbbVie - LARVOL DELTA

Full Field Sensitivity Threshold Progression Over 24 Months in Patients with Advanced Retinitis Pigmentosa after treatment with AGN-151597 (ARVO 2025) - P1/2 | "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • Metastases • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa (clinicaltrials.gov) - P1/2 | N=14 | Completed | Sponsor: AbbVie | Active, not recruiting ➔ Completed

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

Visual acuity and full-field stimulus threshold test–retest reliability in patients with low vision due to advanced retinitis pigmentosa (ARVO 2023) - P1/2 | "This analysis evaluated test–retest reliability of visual acuity (VA) and FST in an ongoing Phase 1/2a study (NCT02556736) of RST-001, an investigational optogenetic gene therapy, in patients with advanced RP...Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • Metastases • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa (clinicaltrials.gov) - P1/2 | N=14 | Active, not recruiting | Sponsor: AbbVie | Trial completion date: Apr 2035 ➔ Sep 2024

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

Current and Future Treatment of Retinitis Pigmentosa. (PubMed, Clin Ophthalmol) - "Amongst the 15 cell and gene therapies in late-stage development, 5 leading candidates have been selected to profile based on the development stage, drug target and geography: gene therapies AGN-151597, GS-030 and VMCO-1 and human stem cell therapies jCell and ReN-003. Gene therapies may be attractive as their fundamental goal is to restore vision; however, cell therapies will likely have a broader application and do not rely on genetic testing, which can delay treatment. Ensuring effective therapeutic options for RP patients across disease stages requires the continued diversification and advancement of the development pipeline, and sustained efforts to promote early patient identification and timely diagnosis."

Journal • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa (clinicaltrials.gov) - P1/2; N=14; Active, not recruiting; Sponsor: Allergan; Trial primary completion date: Dec 2020 ➔ Jun 2020

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

Allergan: Retrosense Therapeutics Acquisition (Allergan) - Anticipated results from P1/2 open-label dose escalation trial (NCT02556736) in advanced retinitis pigmentosa in H2 2017; Anticipated approval for retinitis pigmentosa in 2020/2021

Anticipated P1/2 data • Anticipated regulatory • Inherited Retinal Dystrophy • Ophthalmology

Wayne state start-up, RetroSense, doses first patient in phase I/II RP clinical trial (Eurekalert) - P1/2, N=15; NCT02556736; "RetroSense Therapeutics...successfully dosed their first patient in the first clinical trial to evaluate the safety of RST-001."

Trial initiation date • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology

RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa (clinicaltrials.gov) - P1/2; N=15; Recruiting; Sponsor: Allergan; Trial primary completion date: Apr 2017 ➔ Aug 2019

Trial primary completion date • Biosimilar • Gene Therapies • Immunology • Inherited Retinal Dystrophy • Ocular Inflammation • Oncology

RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa (clinicaltrials.gov) - P1/2; N=14; Active, not recruiting; Sponsor: Allergan; Recruiting ➔ Active, not recruiting; N=21 ➔ 14

Enrollment change • Enrollment closed

RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa (clinicaltrials.gov) - P1/2; N=21; Recruiting; Sponsor: Allergan; Trial completion date: Oct 2033 ➔ Apr 2035; Trial primary completion date: Aug 2019 ➔ Dec 2020

Clinical • Trial completion date • Trial primary completion date