PRESS RELEASE
29 July 2025
Exonate plans ‘CLEAR-DE’ Phase IIb clinical trial of lead candidate EXN407 for diabetic eye disease
· 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
· Phase IIb trial to investigate efficacy in NPDR patients to commence in Q1 2026
Cambridge, UK, 29 July 2025: Exonate Ltd., a biotechnology company developing novel, non-invasive, small-molecule therapeutics for patients with retinal vascular diseases, today announced plans to initiate a Phase IIb clinical trial for its lead candidate EXN407. The CLEAR-DE (Clinical Evaluation of a New Eye Drop for Alleviating Retinopathy in Diabetic Eye Disease) trial will further evaluate the clinical efficacy, optimal dosing and safety profile of EXN407 in patients with non-proliferative diabetic retinopathy (NPDR).
The progression to a Phase IIb clinical study follows encouraging results from Exonate’s Phase Ib/IIa study in March 20241. In the study, lead candidate EXN407 met its primary safety and tolerability endpoints, with no drug-related serious adverse events, and high patient compliance. Importantly, exploratory efficacy signals showed a reduction in vascular leakage, a key pathological driver in diabetic retinopathy. The Phase IIb trial to investigate the efficacy of EXN407 in NPDR patients is planned to begin in early 2026, with 140 patients currently enrolled across multiple sites in Australia, the Middle East and China.
Diabetic retinopathy (DR) is a leading cause of vision loss globally. An estimated one-third of the nearly 500 million people living with diabetes are affected by NPDR2, the early stage of diabetic eye disease, which can lead to more severe, vision-threatening complications. Current therapeutic approaches involve monthly intraocular injections, initiated only after symptoms appear. This limits opportunities for early therapeutic benefit, and due to the invasive nature and frequency, is often deferred until irreversible damage has occurred.
EXN407 is a first-in-class, twice-daily topical formulation of a selective SRPK1 inhibitor – a minimally-invasive approach with potential to become the first effective eye-drop therapy for NPDR and diabetic macular oedema (DME). Designed to modulate expression of vascular endothelial growth factor (VEGF) via alternative mRNA splicing, EXN407 selectively inhibits the pro-angiogenic isoforms that drive disease progression, causing abnormal and leaky blood vessel formation in the retina.
“The Phase Ib/IIa data demonstrated the clear potential of EXN407 as a non-invasive treatment for diabetic eye disease,” said Dr. Catherine Beech, Chief Executive Officer of Exonate. “This therapy could transform the treatment landscape for early-stage disease by providing clinical benefit while avoiding the burden of injections, representing a significant advancement for patients and physicians alike. We’re excited to progress to the CLEAR-DE Phase IIb trial and are actively seeking strategic partners to support its execution.”
For more information about Exonate’s pipeline and clinical development plans, please visit: https://www.exonate.com/
1. Press Release (5th March, 2024): Exonate first-in-class eye drop Phase Ib/IIa trial data demonstrate safety and biological activity in treatment of diabetic retinopathy and diabetic macular oedema
2. Wong TY, Tan TE. The diabetic retinopathy “Pandemic” and evolving global strategies: The 2023 Friedenwald Lecture. Invest Ophthalmol Vis Sci. 2023;64(15):47
ENDS
Notes to Editors
Dr Catherine Beech, CEO, Exonate
Imaging from Phase IIa study demonstrates significant reduction in vascular leakage, a critical marker for NDPR
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About Exonate
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).
The Company is led by a highly experienced management team, and has a strong foundation of scientific expertise, with cross-disciplinary experience in medicine and drug development.
For more information, visit the Exonate website, or find Exonate on X (@ExonateUK) and LinkedIn (@ Exonate Limited)