Single-Dose Suprachoroidal AXT107 in Neovascular Age-Related Macular Degeneration: The Phase 1/2a DISCOVER Trial

Frequent injections remain a substantial burden in the treatment of neovascular age-related macular degeneration (nAMD). AXT107 (gersizangitide) is a long-acting, integrin-disrupting peptide designed for suprachoroidal administration. In the phase 1/2a, open-label DISCOVER trial, 15 participants with active nAMD received a single suprachoroidal injection of AXT107 microparticles at 0.125, 0.25, or 0.5 mg and were followed for 40 weeks. Safety and tolerability were the primary outcomes; visual acuity, retinal thickness, and time to protocol-specified rescue anti-vascular endothelial growth factor treatment were exploratory outcomes. No AXT107-related serious adverse events, clinically significant intraocular inflammation, or sustained intraocular pressure elevations were observed. Fourteen eyes met the protocol-specified baseline imaging criterion for efficacy analyses. Among these eyes, two treatment-experienced eyes completed 40 weeks without protocol-specified rescue, and a third first received protocol-specified rescue at week 36. Anatomical improvements were observed in at least one eye in each dose cohort, although responses varied. A single suprachoroidal injection of AXT107 was well tolerated in this small first-in-human study. The exploratory findings warrant evaluation in a controlled trial.

Suprachoroidal AXT107 was well tolerated, with some eyes remaining free of protocol-specified rescue for up to 40 weeks.