OT-551

OT-551 is a novel small molecule drug developed as a topical eye drop for the treatment of age-related macular degeneration (AMD), particularly the geographic atrophy (GA) form. It is a lipophilic disubstituted hydroxylamine with antioxidant and anti-inflammatory properties. After ocular administration, it penetrates cell membranes and is converted by intraocular esterases to its active metabolite Tempol-H. Both OT-551 and Tempol-H contain active hydroxylamine groups that interact with free radicals in the eye to terminate free radical reactions and limit oxidative damage implicated in AMD pathogenesis. Additionally, OT-551 downregulates overexpression of nuclear factor kappa B (NF-kappa B), which is associated with oxidative stress, inflammation, and angiogenesis[1][2][4]. Clinical trials have investigated its safety and efficacy for GA in AMD as well as other indications such as cataracts and dry eyes; however, studies outside AMD were discontinued due to limited benefit[4].

Other names

OT-551OT551OT 551Tempol-H (active metabolite)DZ02S3A31NDZ-02S3A31NDZ 02S3A31NUNII-DZ02S3A31NUNII-DZ-02S3A31NUNII-DZ 02S3A31N