Exonate

Exonate is a clinical-stage biopharmaceutical company focused on developing therapies for retinal vascular diseases that can lead to vision loss. The company was spun out of the University of Nottingham in 2013, founded by a team of academics including Professor David Bates and Professor Lucy Donaldson from the University of Nottingham, Professor Steven Harper from the University of Bristol, and Professor Jonathan Morris from the University of New South Wales. Dr. Catherine Beech, who has over 25 years of experience in the biotech and pharmaceutical industries, joined the scientific founders to establish the company and serves as its CEO. The company's formation was based on Professor Bates' research into the alternative splicing of Vascular Endothelial Growth Factor (VEGF), a protein that regulates blood vessel formation.

Exonate's business model centers on the discovery and development of small-molecule drugs, with the strategic aim of partnering with large pharmaceutical companies for late-stage development and commercialization. The company has successfully raised capital through multiple funding rounds, securing investments from angel investors, venture funds like Uniseed, and grants, including a significant £4.9 million award from the Wellcome Trust. A key milestone was a collaboration with Janssen Pharmaceuticals to advance its lead compound. The company's primary market is ophthalmology, specifically addressing conditions like diabetic macular edema (DME) and wet age-related macular degeneration (wet AMD), which currently rely on invasive monthly injections into the eye.

The lead product candidate, EXN407, is being developed as a first-in-class topical eye drop. This non-invasive approach represents a significant potential shift from the current standard of care. EXN407 works by selectively inhibiting the SRPK1 enzyme, which in turn modulates the alternative splicing of VEGF mRNA. This mechanism targets the production of pro-angiogenic VEGF isoforms that cause the abnormal and leaky blood vessel growth characteristic of retinal diseases. In a Phase Ib/IIa clinical trial involving patients with diabetic macular edema, EXN407 successfully met its primary safety and tolerability endpoints and showed encouraging signs of biological activity. Patients treated with the eye drops demonstrated a reduction in vascular leakage and macular thickness. Following these positive results, Exonate is planning to initiate a larger Phase IIb trial, named CLEAR-DE, in early 2026 to further assess efficacy and dosing.

Keywords: Exonate, retinal vascular disease, diabetic retinopathy, diabetic macular edema, EXN407, topical eye drops, SRPK1 inhibitor, VEGF, alternative splicing, ophthalmology drug development, Catherine Beech, David Bates, anti-angiogenic therapy, clinical trials, non-invasive treatment, vision loss, biopharmaceutical, wet AMD, small molecule therapeutics, macular degeneration treatment, Janssen collaboration, Wellcome Trust grantee, University of Nottingham spin-out, microvascular research