AbbVie/Regenxbio recently presented data from a Phase II study of its lead diabetic retinopathy (DR) and neovascular age-related macular degeneration (nAMD) gene therapy candidate, RGX-314, at the American Society of Retinal Specialists (ASRS) annual meeting, which took place on July 13-16. RGX-314 has the potential to address a key unmet need for drugs that reduce patient burden and compliance-related issues among diabetic macular edema (DME) patients, says GlobalData, a leading data and analytics company.

Currently, patients receiving anti-vascular endothelial growth factor (VEGF) therapy for DME are obliged to visit the hospital six to eight times a year on average. However, despite being an anti-VEGF therapy, similar to many other treatments for DME including the gold standard Bayer’s Eylea (aflibercept), RGX-314 is unique among anti-VEGF treatments as it is a gene therapy, and therefore offered as a one-time treatment.

Sara Reci, Pharma Analyst at GlobalData, comments: “RGX-314’s potential has many vital implications for DME patients. Firstly, it helps to address the heavy treatment burden by reducing in-hospital treatment days. Many patients receiving treatment for DME are of working age, and in most cases have other comorbidities, which they must also tend to. As a gene therapy, RGX-314 addresses this issue by being a one-time treatment, thereby significantly reducing in-hospital treatment days.”

Key opinion leaders (KOLs) interviewed by GlobalData have emphasized their enthusiasm regarding RGX-314. Specifically, they shared their high hopes that RGX-314’s indications will be extended to include DME, and that results from the current trials, namely those for AMD, will be replicated in DME patients. However, some KOLs are sceptical of the use of a gene therapy to inhibit VEGF expression, noting the importance of constitutive VEGF expression and recommending against the chronic suppression of VEGF in DME patients.

Reci concludes: “Despite the fact that RGX-314 is currently indicated towards use in DR and nAMD trials, the ASRS annual meeting and Regenxbio’s website note the therapy’s potential use in DME too. As of now, physicians are mostly keen on the idea of a gene therapy finally reaching the DME space; however, further trials of RGX-314 involving DME patients are warranted to reassure physicians on the effects of the gene therapy in this disease population. Provided the clinical trials yield positive results for safety and efficacy, this new therapy may be set on a trajectory to compete directly with the standard-of-care anti-VEGF drugs across the ophthalmological space.”