Breye Therapeutics Reports Promising Phase 1b Results for Oral Diabetic Retinopathy Drug Danegaptide
Key Insights
Breye Therapeutics (search) presented encouraging Phase 1b clinical data for danegaptide, a first-in-class oral small molecule therapy for non-proliferative diabetic retinopathy (NPDR) with associated macular edema.
The multi-center study of 24 patients demonstrated that danegaptide was well tolerated and achieved statistically significant reduction in edema measures by study end.
More than half of participants showed retinal imaging data consistent with reductions in vascular leakage, supporting the drug's potential as an early intervention for diabetic retinopathy.
Breye Therapeutics (search) has reported promising Phase 1b clinical results for danegaptide, its lead oral therapy candidate for treating non-proliferative diabetic retinopathy (NPDR) with associated macular edema. The data, presented by Prof. Carl Regillo at the Angiogenesis, Exudation and Degeneration 2026 symposium, demonstrated encouraging safety and efficacy signals that support advancement to Phase 2 development.
Phase 1b Study Design and Results
The multi-center, open-label, dose-escalation study enrolled 24 patients with NPDR and associated diabetic macular edema across 11 clinical sites in the UK, Germany, and the US. The trial assessed safety, tolerability, pharmacokinetics, and early biological activity of danegaptide over a four-week treatment period.
According to Breye, danegaptide was well tolerated and achieved plasma levels within the targeted therapeutic range. More than half of participants showed retinal imaging data consistent with reductions in vascular leakage, which represents a key pathology in NPDR. Several patients demonstrated notable anatomical improvements after four weeks of treatment, and by study end, a statistically significant reduction in edema measures had been recorded.
Novel Mechanism of Action
Danegaptide is described as a first-in-class oral small molecule designed to stabilize the vasculature and protect against retinal capillary breakdown and vascular leakage caused by hyperglycemia. The drug's novel mode of action targets cell-cell uncoupling, addressing core pathological mechanisms underlying diabetic retinopathy progression.
Prof. Carl Regillo, Director of Retina Service of Wills Eye Hospital and Professor of Ophthalmology at Thomas Jefferson University, commented: "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."
Addressing Unmet Medical Need
Current treatment options for diabetic retinopathy are limited, particularly for patients with early or moderate-stage disease before loss of visual function. Intravitreal injections represent the mainstay treatment for severe retinal diseases but are typically prescribed for advanced stages including proliferative diabetic retinopathy, diabetic macular edema, and neovascular age-related macular degeneration.
The in-office intravitreal injection procedures are increasingly burdensome for patients and caregivers, and resource-demanding for clinical practices. Many patients find repeated injections burdensome, contributing to high discontinuation rates and less optimal outcomes. This represents the majority of patients who remain currently untreated despite diagnosed diabetic retinal disease.
Development Plans
Based on these encouraging results, Breye is preparing to initiate a Phase 2 randomized clinical trial in NPDR patients, using improvements in Diabetic Retinopathy Severity Scale (DRSS) scores as the regulatory endpoint. The company notes that reductions in vascular leakage are well-known to precede reductions in DRSS scores, providing scientific rationale for the planned Phase 2 design.
Ulrik Mouritzen, Chief Executive Officer of Breye Therapeutics (search), stated: "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."
Prof. Regillo emphasized the potential paradigm shift: "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."
The company is currently fundraising to support the next phase of development. Breye Therapeutics (search) is backed by Novo Holdings and Sound BioVentures, with additional support from the BioInnovation Institute, Danish Growth Foundation, and Danish Innovation Foundation.