FDA Clears New Drug Application for RGX-314 for Diabetic Retinopathy


A phase 2 trial will begin to evaluate the safety and efficacy of RGX-314.

Steve Pakola, MD

Steve Pakola, MD

REGENXBIO yesterday announced the clearance of the Investigational New Drug application by the US Food and Drug Administration (FDA) to evaluate suprachoroidal delivery of RGX-314 to patients with diabetic retinopathy.

The biotechnology company plans to begin dosing patients in a phase 2 trial, ALTITUDE, in the second half of 2020.

“We believe one-time treatment with RGX-314 can provide sustainable, long-term anti-VEGF delivery to the eye, potentially reducing the severity of (diabetic retinopathy) and preventing vision-threatening complications,” Steve Pakola, MD, chief medical officer at REGENXBIO, said in a statement.

ALTITUDE is a multi-center, open-label, randomized, controlled dose-escalation study that aims to evaluate the safety, efficacy, and tolerability of suprachoroidal delivery of RGX-314. Investigators of the study will randomize patients to receive RGX-314 or an observational control at a 3:1 ratio. They will evaluate 2 dose levels, 2.5x1011 GC/eye and 5x1011 GC/eye.

The study’s primary endpoint is the proportion of patients that improve in diabetic retinopathy severity at 48 weeks based on the Early Treatment Diabetic Retinopathy Study-Diabetic Retinopathy Severity Scale.

The announcement of the clearance of the Investigational New Drug application comes on the heels of the company announcing positive initial data for RGX-314 for wet age-related macular degeneration (wet AMD).

REGENXBIO reported positive one-year results in the phase 1 and 2a trial.

“The data demonstrated stable to improved visual acuity and retinal thickness, as well as a meaningful reduction in anti-VEGF injection burden, in these higher dose levels at one year,” Pakola said.

In the trial, investigators evaluated 42 patients with long-standing severe wet AMD requiring anti-vascular endothelial growth factor (anti-VEGF) injections treated across 5 dose cohorts. Investigators assessed many efficacy measures including the number of anti-VEGF intravitreal injections, change in vision measured by Best Corrected Visual Acuity, change in central retinal thickness, and RGX-314 protein expression levels.

“We are pleased to advance this program and look forward to initiating our second clinical trial utilizing suprachoroidal delivery, building upon clinical and preclinical data from the RGX-314 program," Pakola said yesterday.

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