Small molecule drug candidate and delivery system for corneal dystrophy

Background: Fuchs’ endothelial corneal dystrophy (FECD) is a progressive, blinding disease characterized by corneal endothelial cell apoptosis. FECD is currently treated only by surgical procedures such as corneal transplant and require extended periods of recovery. Oxidative stress has been demonstrated to play a major role in the pathogenesis of the FECD.

Inventions:

(i) Using HTS, we have identified several hit molecules with EC50 in the nM range. Our lead compound 424 protects corneal endothelial cells from oxidative stress-induced cell death in a dose-dependent manner (Figure A). Med chemistry of 424 is underway.

(ii) We are also developing a novel non-invasive small molecule ocular drug delivery/formulation system to optimize delivery and penetration of compound 424. Tolerability and pharmacokinetic studies for topical delivery of the lead compound and the formulation system are underway.

IP status: PRV application filed.

Innovators: Mark Fields, Ph.D., Lucian Del Priore, M.D., Ph.D.