Researchers at Trinity College Dublin, in collaboration with Breye Therapeutics, have found that an oral medication (“Danegaptide”) can protect against vision-threatening damage in Non-Proliferative Diabetic Retinopathy (NPDR), which is a leading cause of blindness in working-age adults.
The study, published today in leading international journal, Science Translational Medicine , highlights a new therapeutic approach that could replace or complement frequent, highly invasive eye injections for millions of patients with diabetes.
Diabetic retinopathy affects nearly 30% of people living with diabetes and the non-proliferative form (NPDR) represents the vast majority of cases.
Currently, standard treatments such as intravitreal anti-VEGF injections are reserved for advanced stages of the disease after significant, often irreversible, vision loss has already occurred. Because these injections require regular and direct administration directly into the eye, early intervention has remained impractical.
Key Findings
In pre-clinical models of the disease, administering Danegaptide rapidly reduced leakage of blood vessels in the retina, prevented inner blood-retina barrier (iBRB) breakdown, and reversed retinal edema (swelling) with efficacy comparable to injectable therapies.
Additionally, in an early stage clinical trial involving 24 patients with NPDR, oral Danegaptide was safe and well-tolerated. Notably, 55% of participants demonstrated early signs of biological activity, including marked reductions in retinal leakage, decreased central subfield thickness, and resolution of intra-retinal fluid cysts.
What are the potential impacts of this research?
Global estimates suggest tens of millions of people are living with NPDR, with roughly one-third of adults with diabetes likely to develop this or another form of diabetic retinopathy in their lifetime.
Given how debilitating the condition can be and the urgent need for better, less invasive treatments, this research offers significant hope that a simple oral medication could make a huge difference in effectively managing the condition and enabling affected people to live more normal lives.
The findings are extremely promising and the research team now underlines the need to now progress with larger Phase II clinical trials.
“Managing diabetic eye disease before irreversible damage occurs has long been constrained by the burden of invasive injections,” said Professor Matthew Campbell , Chair of Neurovascular Genetics at Trinity College Dublin and senior author on the research paper.
“ Our findings demonstrate that a simple oral pill can effectively target and repair the inner blood-retina barrier. This potentially offers a non-invasive, preventive path forward that could protect vision in both eyes simultaneously, long before severe sight loss takes hold.”
Dr Pete Adamson , CSO, Breye Therapeutics , added: "The data from this study validate a major paradigm shift in retinal care. By proving that an oral therapy like Danegaptide can directly protect the inner blood-retina barrier, we are opening the door to early, non-invasive intervention for NPDR. The successful Phase Ib results provide a very strong foundation for larger Phase II clinical trials to evaluate long-term efficacy.”
Science Translational Medicine