Fenretinide (Compound ST-602) - Dry AMD Oral

Fenretinide, or (N-[4-hydroxyphenylretinamide), is an oral compound that decreases serum retinol by binding to retinol-binding protein, and promotes renal clearance of retinol.

This in turn decreases the bioavailability of retinol for the retinal pigment epithelium (RPE) and photoreceptors.

A2E (N-retinylidene-N-retinylethanolamine), a retinoid byproduct, is a major fluorophore in lipofuscin and a significant source of RPE cytotoxicity⁽⁶⁶⁾.

It is hypothesized that by reducing toxic retinoid byproducts of visual cycling, there will be a slowing of GA progression.

Sirion Therapeutics, Inc. (Tampa, FL), is sponsoring a phase II trial to assess the benefit of fenretinide in the treatment of GA⁽⁶⁷⁾.

The study group is ongoing.

Patients have been randomized to 1 of 2 doses (100 mg or 300 mg) or placebo, and they are being followed for 2 years.