01 Dec 2023
Promising Trial Paves the Way for First-Ever Eye Drop for Diabetic Macular Edema
Exonate Ltd., a pioneering mRNA therapy company, has announced the successful completion of its phase Ib/IIa study for its lead ophthalmology asset, EXN407. The small molecule inhibitor of splice factor kinase SRPK1 exhibited promising results, meeting prespecified endpoints, and displaying encouraging signals of biological activity.
During the trial, EXN407 demonstrated impeccable safety and pharmacokinetic parameters, with no clinically meaningful changes or trends observed. The study, which treated patients with either EXN407 or a placebo twice a day for three months, reported only mild adverse events, and no issues affecting tolerability or treatment discontinuation. The positive outcomes, including macular thickness reduction and a decrease in retinal vascular leakage, position EXN407 as a frontrunner to become the first topical treatment for retinal vascular diseases, such as diabetic retinopathy and diabetic macular edema.
In a strategic move, Exonate has regained full rights to its complete portfolio of ophthalmology assets from Janssen Pharmaceuticals, Inc., a Johnson & Johnson company. This development underscores Exonate's commitment to advancing innovative therapies independently.
The current landscape of diabetic retinopathy and diabetic macular edema treatments involves invasive monthly injections directly into the patient's eye, posing long-term burdens. With a market demand for non-invasive solutions in this sector, Exonate aims to address this need with its topical therapy approach.
EXN407 is now slated to progress to a phase II clinical study in 2024, aiming to fully demonstrate its efficacy in patients with severe diabetic macular edema. Exonate, leveraging its expertise in alternative splicing, seeks to develop medicines that target end organ diseases resulting from poor diabetic control. The company's small-molecule drugs are designed to provide effective, non-invasive treatment for retinal diseases, offering high ocular permeability through topical eye drops and eliminating the need for intrusive intravitreal injections.
Dr. Catherine Beech, CEO of Exonate, expressed excitement about the trial results, stating, "The data validates the hypothesis that modulating VEGF splicing can lead to clinical benefits. We are eager to progress to the phase II trial next year and welcome inquiries from potential partners for the program."
This breakthrough represents a significant leap forward in the quest for advanced, patient-friendly treatments for diabetic complications.