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Exonate diabetic macular oedema asset passes phase Ib/2a challenge

Exonate’s lead ophthalmology asset, EXN407, has achieved its prespecified endpoints in a phase Ib/IIa study.

The Duxford-based mRNA therapy company is focused on treatments for diabetic complications.

Older couple testing blood sugar. Picture: istock/Chris Ryan
Older couple testing blood sugar. Picture: istock/Chris Ryan

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

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

EXN407, a small molecule inhibitor of splice factor kinase SRPK1, is now well placed to be the first topical treatment for retinal vascular diseases, including diabetic retinopathy and diabetic macular oedema.

During the trial EXN407 met all safety and pharmacokinetic parameters and displayed encouraging signals of biological activity. Patients in the phase Ib/IIa trial were treated twice a day for three months, with either EXN407 or a placebo.

Exonate CEO Catherine Beech
Exonate CEO Catherine Beech

The study met all end points well and achieved encouraging results, including:

- No clinically meaningful changes or trends in any safety parameters

- No tolerability issues reported

- Only mild adverse events reported

- No adverse events leading to treatment or study discontinuation

- Highlighted signals of biological activity (macular thickness reduction and decrease in retinal vascular leakage).

Dr Catherine Beech, CEO, 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. We are excited to progress to the phase II trial next year and welcome enquiries by potential partners for the programme.”

Exonate, which was founded in 2013, will now progress EXN407 to a phase II clinical study in 2024, to fully demonstrate efficacy in patients with severe diabetic macular oedema.

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