Safety and tolerability of intravitreal umedaptanib pegol (anti-FGF2) for neovascular age-related macular degeneration (nAMD): a phase 1, open-label study

ObjectiveTo evaluate the efficacy and safety of a single-dose intravitreal umedaptanib pegol (anti-FGF2, investigational new drug) for the treatment of neovascular age-related macular degeneration (nAMD).MethodsNine participants who had a diagnosis of refractory nAMD were enrolled and received a single intravitreal injection of umedaptanib pegol at increasing doses of 0.2, 1.0 or 2.0 mg in the study eye.ResultsAll three doses of umedaptanib pegol evaluated in the study were safe and well tolerated. No severe adverse event (AE) was observed in the study. There was an improvement in retinal fluid measured by central subfield thickness (CST) in most subjects. Remarkably, all three subjects who received 2.0 mg/eye showed improvement of more than 150 mu m.ConclusionsIntravitreal umedaptanib pegol was safe, well tolerated, and demonstrated an indication of bioactivity in participants that have persistent subretinal fluid refractory to the treatment with anti-VEGFs.

Automated summary

This study evaluated the efficacy and safety of a single-dose intravitreal umedaptanib pegol for the treatment of refractory neovascular age-related macular degeneration (nAMD). The results showed that umedaptanib pegol was safe and well tolerated, with improvements in retinal fluid observed in most subjects. The study indicated that intravitreal umedaptanib pegol may be an effective and safe treatment for nAMD patients who do not respond to anti-VEGF therapy.