Exonate’s EXN407 meets endpoints in Phase Ib/IIa trial

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The company’s lead ophthalmology candidate, EXN407, has the potential to be the first topical treatment for diabetic macular edema.

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Exonate’s lead ophthalmology candidate, EXN407 achieved its pre-specified endpoints in a phase Ib/IIa study. It also met all the safety and pharmacokinetic parameters. The trial was carried out in collaboration with Janssen. The company focuses on mRNA therapy treatments for diabetic complications, including diabetic retinopathy (DR) and diabetic macular edema (DME).¹

These results show promising biological activity and the company notes that EXN407 has the potential to be the first topical treatment for DR/DME, significantly reducing the treatment burden for patients and ophthalmologists.

EXN407 is described as a small molecule inhibitor of splice factor kinase SRPK1. Patients in the phase Ib/IIa trial were treated twice a day for 3 months, with either EXN407 or placebo.¹ The study met all endpoints 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).¹

In the company’s press release¹, 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 program.”

Following these results, Exonate will progress EXN407 to a phase II clinical study in 2024, to fully demonstrate efficacy in patients with severe DME.