First eye drop for diabetic macular oedema on horizon after successful trial

During the trial, EXN407 met all endpoints and displayed ‘encouraging’ biological activity in diabetic patients with retinal eye disease.

Dr Catherine Beech, CEO of Exonate, said: “The results from the EXN407 trial are very encouraging, with the data validating the hypothesis that modulating VEGF splicing can lead to clinical benefits."

Diabetic macular oedema is a condition affecting many diabetes patients worldwide, where leaking blood vessels in the eye cause a thickening of the macula region of the retina, leading to vision loss.

Current treatments for the condition involve monthly injections directly into the patient’s eye that prevent aberrant blood vessel growth. However, the invasive treatments place long-term burdens on patients

If successful, EXN407 will be the first topical eye drop available for the treatment of retinal vascular diseases, including diabetic retinopathy and diabetic macular oedema.

EXN407, is a small molecule inhibitor of splice factor kinase SRPK1. Patients in the trial, carried out in collaboration with Janssen Pharmaceuticals, were treated twice a day for three months, with either EXN407 or placebo.

In addition, the company has now regained full rights to its complete portfolio of ophthalmology assets from Janssen Pharmaceuticals.

Moving forward, Exonate will now progress EXN407 to a phase 2 clinical study in 2024.

"We are excited to progress to the phase II trial next year and welcome enquiries by potential partners for the programme," Beech added. 

Exonate’s small-molecule drugs are addressing the need for effective, non-invasive treatment of retinal diseases. The treatments offer high ocular permeability that allows targeted therapeutic delivery to the retina with topical eye drops, removing the need for unpleasant intravitreal injections, the company said.