A biotech firm, Exonate, has made strides in developing a novel treatment for retinal vascular diseases. Their flagship product, EXN407, is a topical SRPK1 inhibitor that has shown promising results in both safety and effectiveness as a monotherapy for conditions like diabetic retinopathy and diabetic macular oedema. The company is gearing up for Phase IIb clinical trials for EXN407 in 2024, following the positive outcomes of a Phase Ib/IIa study.
The Phase Ib/IIa clinical trial, a double-masked, placebo-controlled investigation, evaluated the safety, tolerability, and biological activity of EXN407 in patients with mild to moderate non-proliferative diabetic retinopathy and mild diabetic macular oedema. The study found that EXN407 was well-tolerated, with no serious adverse events reported, and demonstrated a high level of comfort for patients. Additionally, the drug showed significant biological activity, including a notable reduction in macular thickness and vascular leakage, when compared to the placebo group.
The EXN407 eye drop targets the SRPK1 enzyme, which plays a role in the alternative splicing of vascular endothelial growth factor (VEGF), a protein pivotal in blood vessel development. By inhibiting SRPK1, EXN407 aims to halt the progression of vascular retinal diseases by targeting the specific isoforms of VEGF that contribute to abnormal blood vessel growth within the eye.
The full data from the Phase Ib/IIa trial is set to be presented at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting in May 2024. Exonate's CEO, Catherine Beech, highlighted the potential of EXN407 as a non-invasive treatment option that could offer substantial benefits to patients with diabetic eye diseases, potentially reducing the need for injections.
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