Pipeline therapies within the diabetic macular oedema (DME) space have recently gathered interest following the American Society of Retina Specialists (ASRS) Annual Meeting, which took place on 13–16 July. The spotlight was placed on many up-and-coming pharmacotherapies for retinal diseases, one of which was AbbVie’s/RegenxBio’s gene therapy RGX-314.

RGX-314 is currently in Phase II development for diabetic retinopathy (DR) in the ALTITUDE trial and Phase III development for neovascular age-related macular degeneration (nAMD) in the ASCENT trial. The drug is of paramount interest due to addressing vital unmet needs for anti-vascular endothelial growth factor (VEGF) treated retinal diseases, including DME. Clinical trial results in DR patients have shown that 47% of eyes treated with RGX-314 have experienced diabetic retinopathy severity score (DRSS) improvements of two or more steps. While the current trials are centred around DR and nAMD, the ASRS annual meeting and Regenxbio’s website note the therapy’s potential use in DME, a disease for which DR is a prerequisite.

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RGX-314 is an anti-VEGF therapy that is similar to many other treatments for DME on the market, including the gold standard, Bayer’s Eylea (aflibercept). RGX-314 stands out, however, by offering a unique feature unlike any other anti-VEGF treatment, as it is a gene therapy, thereby offered as a one-time treatment. This has many implications for patients, including the fact that it helps to address the heavy treatment burden experienced by DME patients, consequently diminishing issues related to patient compliance.

At present, patients are obliged to visit the hospital an average of six to eight times a year to receive anti-VEGF injections, which is the first-line treatment for DME. In addition, many patients receiving treatment for DME are of working age, and in most cases have other comorbidities as well. RGX-314’s status as a one-time therapy is, therefore, of utmost importance within the DME space.

Many key opinion leaders (KOLs) interviewed by GlobalData have emphasised 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. Some KOLs, however, are skeptical of the use of gene therapy to inhibit VEGF expression, with one KOL noting: “These patients [will] be exposed to chronic suppression of VEGF, and I’m not sure that that is a good approach for diabetic patients. They do need some VEGF. Constitutive expression of VEGF is important.”

There is no doubt that RGX-314’s practicality gives it overwhelming potential to be favoured among many patients and clinicians alike if it reaches the DME market. If the Phase II and subsequent Phase III trials trial yields good results upon completion, this new therapy may be set on the trajectory to compete directly with the standard-of-care anti-VEGF drugs across the ophthalmological space.