Preclinical and clinical evaluation of systemic danegaptide for the treatment of nonproliferative diabetic retinopathy.

Orally administered danegaptide significantly improves retinal vascular barrier function in NPDR, showing safety and early efficacy in a phase 1b trial with 24 patients.

• Why it matters: Nonproliferative diabetic retinopathy (NPDR) often leads to vision loss and currently relies on invasive VEGF-neutralizing treatments, which have limited patient acceptance and frequent injections.
• What they did: The study evaluated danegaptide’s effects in animal models and a phase 1b trial, demonstrating its ability to enhance blood-retina barrier integrity through connexin-43 modulation and VEGF antagonism, with RNA sequencing revealing its impact on VEGF-related pathways.
• The result: Findings suggest danegaptide is safe and effective at early stages, paving the way for larger trials to confirm its potential as a non-invasive treatment for NPDR.