A Comprehensive Monograph on Anecortave: A Novel Angiostatic Cortisene from Clinical Promise to Discontinuation

Executive Summary

This report provides a comprehensive scientific and clinical monograph on Anecortave (DrugBank ID: DB12081), an investigational small molecule. Anecortave is the principal active metabolite of the prodrug Anecortave Acetate, which was developed by Alcon, Inc. under the proposed trade name Retaane. Anecortave Acetate was engineered as a first-in-class "angiostatic cortisene," a synthetic steroid derivative designed to potently inhibit angiogenesis—the formation of new blood vessels—while being devoid of the glucocorticoid activity and associated side effects typical of corticosteroids.

The primary therapeutic target for Anecortave Acetate was exudative (wet) age-related macular degeneration (AMD), a leading cause of severe vision loss. Its development was characterized by significant innovation, most notably a multi-faceted, downstream mechanism of action that targeted multiple steps in the angiogenic cascade, and a novel drug delivery system. This system, known as the posterior juxtascleral depot (PJD), utilized a specialized cannula to place a slow-release suspension of the drug behind the eye, offering the potential for sustained therapeutic effect with dosing intervals of up to six months.