Diabetic retinopathy affects approximately 30% of adults with diabetes and stands as one of the leading causes of vision loss among working-age individuals worldwide. The condition damages the delicate blood vessels in the retina (the light-sensitive layer at the back of the eye) leading to fluid leakage, swelling, and potential blindness if left untreated.

Historically, treatment options for non-proliferative diabetic retinopathy (NPDR) have been limited primarily to invasive anti-VEGF injections delivered directly into the eyeball. Because of their invasive nature and the burden of repeated procedures, these injections are typically reserved for advanced stages after significant retinal damage has already occurred.

A new study published in Science Translational Medicine highlights a potential breakthrough: danegaptide, an oral small-molecule drug developed by Breye Therapeutics.

How Danegaptide Works

Unlike current injectable treatments, danegaptide is taken as a simple daily pill. It targets and reinforces the cell-to-cell connections in the retina, stabilizing the inner blood-retina barrier to prevent blood vessels from breaking down or leaking fluid.

Key Study Findings

• Preclinical Success: In laboratory models, oral danegaptide rapidly reduced blood vessel leakage, protected the blood-retina barrier, and reversed retinal swelling at levels comparable to standard eye injections.
• Early Clinical Trial Results: In an early-stage Phase Ib trial involving 24 patients with NPDR and macular edema across 11 sites in the U.K., Germany, and the U.S., the oral medication was safe and well-tolerated.
• Early Signs of Efficacy: Roughly 55% of study participants experienced early biological improvements, including reduced retinal leakage, decreased central retinal thickness, and resolution of fluid cysts.
• Broader Safety Data: Across various clinical trials, danegaptide has been evaluated in over 500 participants, maintaining a solid safety profile.

Looking Ahead

Researchers and leadership at Breye Therapeutics emphasize that an effective oral treatment could shift eye care toward proactive, early intervention. As senior author Matthew Campbell, PhD, noted, a pill could provide a non-invasive path to “protect vision in both eyes simultaneously, long before severe sight loss takes hold.”

Breye Therapeutics is now preparing to initiate a larger Phase II clinical trial, aiming to establish definitive clinical proof-of-concept by 2028.