Home Medizin Exonate ist der erste Augentropfen seiner Klasse, Daten aus der Phase Ib/IIa-Studie belegen Sicherheit und biologische Aktivität bei der Behandlung von diabetischer Retinopathie und diabetischem Makulaödem

Exonate ist der erste Augentropfen seiner Klasse, Daten aus der Phase Ib/IIa-Studie belegen Sicherheit und biologische Aktivität bei der Behandlung von diabetischer Retinopathie und diabetischem Makulaödem

von NFI Redaktion

Exonate Ltd., a biotechnology company developing novel, non-invasive, small molecule therapeutics for patients with retinal vascular diseases, announced today the data from a successful Phase Ib/IIa study for their lead candidate EXN407. These data demonstrate the safety and tolerability of EXN407, clear indications of biological activity, and position it well for further development as the first topical treatment for retinal vascular diseases such as diabetic retinopathy and diabetic macular edema. Exonate now plans to advance EXN407 into the Phase IIb clinical trial CLEAR-DM (Clinical Evaluation of a New Eyedrop for Alleviating Retinopathy in Diabetic Macular Oedema).

EXN407 is a twice-daily formulation consisting of a small molecule SRPK1 inhibitor. The eye drop formula targets SRPK1’s involvement in the alternative splicing of vascular endothelial growth factor (VEGF), a protein strongly involved in regulating blood vessel growth. By inhibiting SRPK1, EXN407 can selectively target pro-angiogenic isoforms of VEGF that lead to abnormal growth of leaky blood vessels in the eye, contributing to the progression of vascular retinal diseases.

The clinical study for mild NPDR/DME (NCT04565756) evaluated the safety, tolerability, and biological response signals to EXN407 monotherapy in a double-blind, placebo-controlled Phase Ib/IIa dose-finding study among treatment-naive patients with mild/moderate nonproliferative diabetic retinopathy (NPDR) and mild diabetic macular edema. The independent Dose Escalation Committee deemed EXN407 safe and well-tolerated, with 100% of patients completing the study without requiring anti-VEGF rescue and no major or severe adverse events related to EXN407 reported. Additionally, EXN407 exhibited a high level of tolerability, with drop comfort scores resembling those of placebo and artificial tears.

In addition to the primary safety and tolerability endpoints, the study reported promising signals of a biological response from EXN407, showing a sustained reduction in macular thickness compared to the placebo group and comparable to previously reported anti-VEGF injections. The study also found that treatment with EXN407 led to a significant reduction in vessel leakage (60% of patients treated with EXN407 compared to 20% under placebo) and inhibited further leakage progression (10% of patients treated with EXN407 compared to 50% under placebo).

The Phase Ib/IIa data demonstrate the clear potential of EXN407 as a non-invasive treatment for these devastating vision-threatening diseases, and the favorable safety profile and biological activity of EXN407 support its further clinical development in retinal vascular diseases.said Catherine Beech, CEO of Exonate. “The results suggest that topical application of EXN407 to the eye could bring clinical benefit and significantly reduce the injection burden for patients with diabetic eye disease. We look forward to partnering with strategic collaborators to support the CLEAR-DM Phase IIb trial designed to fully demonstrate the clinical benefits of EXN407 in NPDR/DME.

The full results of the Phase Ib/IIa study will be presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in May 2024: ARVO Annual Meeting

For more information on Exonate’s therapeutic pipeline for diabetic complications and other indications, as well as our clinical programs, visit: Exonate Website

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