July 20, 2026 —
Adverum, a wholly owned subsidiary of Eli Lilly, presented five-year long-term extension data from the Phase 1 OPTIC trial evaluating investigational Ixo-vec in patients with wet age-related macular degeneration, or wet AMD.
The late-breaking data were presented by Szilárd Kiss, MD, at the American Society of Retina Specialists annual meeting in Montreal. The results highlight the long-term durability of Ixo-vec following a single intravitreal administration.
Ixo-vec is an investigational AAV-based intravitreal gene therapy designed to provide continuous intraocular expression of aflibercept, an anti-VEGF protein used to control wet AMD. The approach aims to reduce the need for frequent anti-VEGF injections while maintaining long-term anatomic and visual outcomes.
Patients treated with Ixo-vec experienced an 87% cumulative reduction in mean annualized anti-VEGF injections through five years compared with their pretreatment injection frequency. Before enrollment, patients required a mean of 9.9 annualized anti-VEGF injections, indicating a sustained reduction in treatment burden over the follow-up period.
The long-term extension data also showed durable anatomic control. Mean central subfield thickness improved by 131.6 µm below baseline through five years, supporting sustained retinal disease control after a single administration.
Visual outcomes were generally preserved in this previously treated, high-treatment-need population. Mean best-corrected visual acuity changed by -6.1 ETDRS letters through five years.
Investigators also reported sustained intraocular aflibercept expression throughout the five-year follow-up period. This finding supports the intended mechanism of Ixo-vec as a one-time gene therapy designed to enable long-term local anti-VEGF production within the eye.
From a safety perspective, no Ixo-vec-related serious adverse events or late-emerging ocular safety signals were reported through five years. Investigators reported no cases of hypotony, vasculitis, retinitis, choroiditis, or vascular occlusion attributed to the therapy during follow-up.
Ixo-vec is currently being evaluated in two Phase 3 trials, ARTEMIS and AQUARIUS. Enrollment in ARTEMIS has been completed, with topline data expected in the first quarter of 2027. Enrollment in AQUARIUS remains ongoing.
Eli Lilly entered into a definitive agreement to acquire Adverum Biotechnologies in October 2025, including rights to Ixo-vec. The five-year OPTIC data provide longer-term support for Ixo-vec’s potential as a durable AAV-based retinal gene therapy designed to reduce injection burden in wet AMD.
