Neurotrophic Keratopathy Eye Drops May Be First Line Therapy

Elderly man examined by an ophthalmologist
Doctor putting drops into a senior man’s eyes
An AAO presentation demonstrated RGN-259 represents a potential first-line treatment for neurotrophic keratopathy.

This article is part of Ophthalmology Advisor’s conference coverage from the 2021 meeting of the American Academy of Ophthalmology, held in New Orleans from November 12 to 15, 2021. The team at Ophthalmology Advisor will be reporting on a variety of the research presented by the ophthalmology experts at the AAO. Check back for more from the AAO 2021 Meeting.


RGN-259 is a safe and effective therapy for neurotrophic keratopathy (NK), according to research presented at the American Academy of Ophthalmology 2021 meeting in New Orleans, held November 12-15.

Researchers conducted a study to assess the efficacy of 0.1% RGN-259 in persistent epithelial defects (PEDs) among NK patients. Inclusion criteria included failure with at least 1 week of conventional treatment, stage 2 or 3 NK, and corneal sensitivity of 4.5 cm or less.

A total of 18 participants were randomly assigned to receive RGN-259 or a placebo. The participants received 5 daily doses for 28 days, with a 2 week follow-up. The primary endpoint was considered the proportion of patients with complete PED healing at day 29.

The primary endpoint was not met, the researchers noted. However, 60% in the RGN-259 group (n=6) experienced complete healing at day 29, compared with 12.5% (n=1) in the placebo group (P =.0656). At day 43, a notable difference (P <.05) was found: 50% (n = 5) with RGN-259 vs 0% (n = 0) with placebo. 

According to the research, 1 serious adverse event unrelated to RGN-259 was reported, but no treatment emergent adverse events led to study discontinuation.

“In a small study, the efficacy and safety of RGN-259 validate that RGN-259 represents a potential first-line therapy for NK,” the presenters reported.  

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Watson M, Ousler III GW, Yang J, Sosne G. SEER-1 demonstrates safety and efficacy of RGN-259 for neurotrophic keratopathy. Poster presented at: The American Academy of Ophthalmology 2021 Annual Meeting; November 12-15, 2021; New Orleans. Abstract PO176.