Ocustatin™ for Treatment of Intermediate AMD

The goal of this project is to develop a novel, high-dose reformulation of oral atorvastatin for early intervention in age-related macular degeneration (AMD).

This award provides critical funding for two initial development steps: (i) completion of drug formulation and characterization, and (ii) preparation of an Investigational New Drug (IND) application to enable a Phase 2 clinical trial. The objective of the Phase 2 trial is definitive confirmation of a very successful proof-of-concept trial conducted by our co-investigators at Harvard University.

We are—to our knowledge—uniquely targeting retinal pigment epithelium (RPE) phagocytic function, which is known to be severely impaired in AMD. In vitro and animal studies indicate that statins restore RPE phagocytosis and reverse the accumulation of the cholesterol-based lipoprotein deposits in the central retina called drusen which are the hallmark lesion of intermediate AMD. Our prior studies indicate that high-dose lipophilic statins are particularly suited for this.

Our product, Ocustatin, is aimed at patients who are still in the intermediate stages of the disease and currently have very limited options to prevent progression to late-stage blindness. There are no prescription drugs for this stage of the disease. Ocustatin offers the possibility of a simple, low-cost, oral medication to address this completely unmet need.