Small-molecule eye drop safe, well-tolerated as treatment for retinal vascular diseases
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Key takeaways:
- EXN407 met primary endpoints of safety and tolerability and also demonstrated decreases in vascular leakage and macular thickness.
- Four mild, treatment-emergent adverse events were reported.
Exonate has announced successful safety and tolerability data from its phase 1b/2a trial of EXN407, a topical small-molecule therapeutic for retinal vascular diseases, such as diabetic retinopathy and diabetic macular edema.
“The results suggest that topical ocular EXN407 may provide clinical benefit and substantially reduce the injection burden for patients with diabetic eye disease,” CEO Catherine Beech said in a company press release.
According to the release, EXN407 is a twice-daily formulation comprising a small-molecule serine-arginine protein kinase-1 inhibitor, which allows EXN407 to selectively target proangiogenic isoforms of VEGF that can result in progression of vascular retinal disease.
In a double-masked, dose-ranging trial, 13 treatment-naïve participants with mild to moderate nonproliferative diabetic retinopathy (NPDR) or diabetic macular edema (DME) were randomized to receive three, twice-daily doses of EXN407 0.5 mg/mL, 1 mg/mL or 1.5 mg/mL or placebo for 8 days.
After dose escalation, 35 participants with mild DME were randomized to 1.5 mg/mL EXN407 or placebo for 85 days and monitored for 1 month after discontinuation of EXN407.
According to the release, EXN407 met safety and tolerability endpoints, with 100% of participants completing the study without anti-VEGF rescue. Four treatment-emergent adverse events were reported, two related to the placebo, and all were mild and resolved without treatment. None of the participants discontinued the eye drops throughout the study. In addition, comfort scores for EXN407 were similar to placebo and artificial tears.
The release also noted that vascular leakage was reduced in 60% of EXN407-treated participants compared with 20% on placebo and that EXN407 inhibited further increases in vascular leakage in 10% vs. 50% of participants, respectively. Participants treated with EXN407 also demonstrated sustained decreases in macular thickness compared with the placebo group.
“We look forward to engaging with strategic partners to support the CLEAR-DM phase 2b trial, which has been designed to fully demonstrate the clinical benefits of EXN407 in NPDR/DME,” Beech said in the release.
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