Breye Therapeutics ApS has presented clinical data showcasing the potential of its lead candidate, danegaptide at the Angiogenesis, Exudation, and Degeneration 2026 symposium.
Danegaptide is a first-in-class oral small molecule with a novel mode of action, that stabilises the vasculature and protects against cell-cell uncoupling, retinal capillary breakdown and vascular leakage caused by hyperglycemia. Conducted across 11 clinical sites in the UK, Germany and the US, the phase 1b study was a multi-centre, open-label, dose-escalation study assessing the safety, tolerability, pharmacokinetics (PK) and early signs of biological activity of danegaptide in patients with NPDR and associated diabetic macular oedema (DME), a complication of NPDR.
The data confirmed a favourable safety profile, with plasma levels within the targeted therapeutic range – and demonstrated early signs of clinical activity. Importantly, more than half of the patients showed retinal imaging data associated with reductions in vascular leakage and several patients achieved noteworthy anatomical improvements following the 4-week treatment period. By the end of the study, a statistically significant reduction in oedema measures was observed. Based on these encouraging data, a randomised phase 2 trial is planned to evaluate danegaptide in the targeted NPDR patient population, using the regulatory endpoint of improvements on the Diabetic Retinopathy Severity Scale (DRSS) score. Breye is actively fundraising to support this next phase of development.
Carl Regillo, director of Retina Service of Wills Eye Hospital and professor of Ophthalmology at Thomas Jefferson University in Philadelphia, Member of the Breye Therapeutics Scientific Advisory Board, said: “The clinical data are encouraging and support the pursuit of danegaptide as an oral, non-invasive treatment solution. Danegaptide has the potential to be a medicine capable of treating diabetic retinopathy from its earlier, NPDR stages. This would create new options for how diabetic retinopathy is treated – opening the door to halting or even reversing disease and treating it before the risk emerges of disease progression to advanced forms. As an oral therapy, it would enable treatment intervention earlier than what is possible today.”
Ulrik Mouritzen, CEO of Breye Therapeutics, said: “Breye is building the first oral, disease-modifying therapies that can stop and reverse vision-threatening damage, with a clear, de-risked path to phase 3 for the lead asset danegaptide. Diabetic retinopathy is a large and growing problem, but AI is revolutionizing our ability to identify the disease at its earlier stages. Finding more early-stage patients in need of therapy will drive the need for better and earlier treatment solutions.”
Currently, intravitreal (IVT) injections are the mainstay treatment for patients with severe retinal diseases caused by leaky blood vessels in the eye and with clinical symptoms of visual loss. Most injections are prescribed to treat advanced stages of disease, including proliferative diabetic retinopathy (PDR), DME, and neovascular age-related macular degeneration (nAMD). However, the in-office IVT injection procedures are increasingly burdensome for patients and their caregivers, and resource demanding for clinical practices. The healthcare systems are continuously needing to adapt to accommodate increasing demand. Many patients also find repeated IVT injections burdensome, contributing to high discontinuation rates and thus less optimal outcomes. Orally administered medications and IVT administered therapies would work in concert and help reaching a broader spectrum of patients in need.
Diabetic retinopathy is a leading cause of vision loss among working-age adults, affecting millions of people with diabetes. While IVT can help preserve vision in patients with late-stage disease, treatment options are currently very limited for patients with early or moderate-stage disease, before loss of visual function, representing the majority of patients who remain currently untreated, despite diagnosed diabetic retinal disease. Intravitreal treatments are often poorly tolerated and associated with low continued adherence, highlighting the critical need for effective non-invasive alternatives. An oral, non-invasive treatment solution would therefore make a real difference to patients and to healthcare providers – ultimately helping to preserve vision.