Exonate is a biotechnology company developing novel, non-invasive, small-molecule therapeutics for patients with retinal vascular diseases which may lead to vision loss. The Company’s pipeline is built around inhibition of pro-angiogenic VEGF by influencing splice patterns, for the treatment of conditions that have the formation of new blood vessels as a significant contributor, such as wet Age-Related Macular Degeneration (wet AMD) and Diabetic Eye Disease. Developed to penetrate to the back of the eye, reach the retina and prevent growth of blood vessels, Exonate’s treatments for retinal diseases are being developed as eye drops, eliminating the need for monthly injections into the eye, laser surgery or use of steroids, without sacrificing potency or permeability. There is a large unmet market opportunity for a topical therapy for diabetic retinopathy and diabetic macular oedema that can treat the conditions earlier in the disease process. Exonate’s lead asset, EXN407, has the potential to be the first topical treatment for retinal vascular diseases, including diabetic retinopathy and diabetic macular oedema. EXN407 has shown encouraging signs of efficacy, alongside meeting all endpoints, in a Phase Ib/IIa trial in diabetic patients with retinal eye disease (diabetic macular oedema).
EXN407 is the first topical SRPK1 inhibitor to demonstrate safety and efficacy as a monotherapy for non-proliferative diabetic retinopathy (NPDR) and diabetic macular oedema.
Explore how emerging in vitro systems — built from primary cells, cocultures, and vascularized tissues — are improving translational research outcomes.