Six-Month Data Reported From Ongoing Phase 1/2 Clinical Trial in X-Linked Retinitis Pigmentosa
Gene therapy company Applied Genetic Technologies Corporation (also called AGTC) reported positive interim 6-month data from its ongoing phase 1/2 clinical program in X-linked retinitis pigmentosa (XLRP). Patients treated centrally with the product candidate demonstrated durable improvement in visual function 6 months after dosing. These data reinforce the promising efficacy and safety results reported in September 2019 and will help investigators to design an XLRP pivotal trial that the company plans to initiate by the end of 2020, according to a company news release.
Data from 17 of the 25 patients were reported in September and suggested a favorable safety profile. Of the 17 patients, eight peripherally treated patients had stable visual function through 6 months, and nine centrally treated patients experienced an improvement in visual function as measured by microperimetry and/or BCVA through 3 months.
At the 6-month time point for those nine centrally dosed patients, measurable improvements were observed in visual sensitivity for four of the evaluable eight patients. All nine patients demonstrated stable or improved visual acuity, with 78% experiencing an improvement of at least 5 letters.
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US FDA Approves First Treatment for Thyroid Eye Disease
The US FDA approved teprotumumab-trbw (Tepezza, Horizon Therapeutics) for the treatment of adults with thyroid eye disease, a rare condition in which the muscles and fatty tissues behind the eye become inflamed, pushing the eye forward and causing it to bulge outwards. This is the first drug approved for the treatment of thyroid eye disease.
Teprotumumab is a fully human monoclonal antibody and a targeted inhibitor of the insulin-like growth factor-1 receptor. It is administered to patients once every 3 weeks for a total of eight infusions.
Phase 3 Trial of the Port Delivery System for Diabetic Macular Edema Begins
Genentech and Roche have initiated a new phase 3 clinical trial of the Port Delivery System (PDS) with ranibizumab (Lucentis, Genentech) in patients with diabetic macular edema (DME). The PDS is an investigational, first-of-its-kind, refillable implant designed to continuously release a customized formulation of ranibizumab into the eye over a period of months.
The Pagoda trial is a phase 3, multicenter, randomized, active-comparator, noninferiority study that will evaluate the efficacy, safety, and pharmacokinetics of the PDS for the treatment of DME. Nearly 550 patients with DME will be randomly assigned to receive either the PDS 100 mg/mL refilled at fixed 6-month intervals or monthly intravitreal injections of ranibizumab 0.5 mg. The primary endpoint of the Pagoda trial is the change in BCVA from baseline averaged over weeks 48 and 50.
Oertli Launches the ViPer Illuminated Scleral Indentor for Retinal Surgery
Oertli Instrumente announced the launch of the ViPer illuminated scleral indentor for retinal surgery. The ViPer facilitates work in the periphery of the posterior segment thanks to its ability to indent and illuminate transsclerally at the same time, according to the company.
The tool is semitransparent and therefore an antiglare and backscatter-free instrument. The consistent illumination of the indented tissue improves the user’s view of the retinal periphery, while the rounded 360° tip ensures its easy application and excellent mobility, according to the company.