Pegaptanib, an anti-vascular endothelial growth factor (VEGF) therapy, was evaluated in two concurrent, prospective, randomized, double-blind, multicenter, dose-ranging, controlled clinical trials for the treatment of neovascular age-related macular degeneration (AMD). The primary endpoint was the proportion of patients who lost fewer than 15 letters of visual acuity at 54 weeks. All three doses of pegaptanib (0.3 mg, 1.0 mg, and 3.0 mg) demonstrated efficacy without a dose-response relationship, with a significant reduction in visual acuity loss compared to sham injections. The risk of severe visual acuity loss (30 letters or more) was significantly reduced, and more patients maintained or gained visual acuity. Adverse events were generally mild to moderate and included eye pain, vitreous floaters, and cataracts. The rate of injection-related adverse events was low, and the long-term safety of pegaptanib remains to be determined. The study concluded that pegaptanib is an effective therapy for neovascular AMD, regardless of lesion size or baseline visual acuity.Pegaptanib, an anti-vascular endothelial growth factor (VEGF) therapy, was evaluated in two concurrent, prospective, randomized, double-blind, multicenter, dose-ranging, controlled clinical trials for the treatment of neovascular age-related macular degeneration (AMD). The primary endpoint was the proportion of patients who lost fewer than 15 letters of visual acuity at 54 weeks. All three doses of pegaptanib (0.3 mg, 1.0 mg, and 3.0 mg) demonstrated efficacy without a dose-response relationship, with a significant reduction in visual acuity loss compared to sham injections. The risk of severe visual acuity loss (30 letters or more) was significantly reduced, and more patients maintained or gained visual acuity. Adverse events were generally mild to moderate and included eye pain, vitreous floaters, and cataracts. The rate of injection-related adverse events was low, and the long-term safety of pegaptanib remains to be determined. The study concluded that pegaptanib is an effective therapy for neovascular AMD, regardless of lesion size or baseline visual acuity.