Ixo-vec for Age-Related Macular Degeneration

(AQUARIUS Trial)

This trial explores a new treatment for individuals with wet age-related macular degeneration (nAMD), a condition affecting vision. Researchers aim to compare a single injection of Ixo-vec (a gene therapy) with the standard treatment, aflibercept, by measuring changes in vision clarity over time. The study includes both those who have never received treatment for nAMD and those with prior treatments. Individuals with active nAMD symptoms, who have responded to previous anti-VEGF treatments, and can manage daily eye drops might be suitable candidates for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking advancements in nAMD treatment.

The trial does not specify if you need to stop taking your current medications. However, you cannot use systemic immunosuppressive drugs within 90 days before the trial, but short courses of oral corticosteroids and certain other steroids are allowed.

Research has shown that Ixo-vec is generally safe for people with wet age-related macular degeneration (nAMD). In earlier studies, patients maintained or even improved their vision without needing many additional treatments. Over a year, more than half of the patients required no extra therapy, suggesting that the treatment reduces the usual need for frequent interventions. Over four years, it demonstrated "excellent safety signals," indicating no serious safety issues during that period.

Most treatments for age-related macular degeneration, like aflibercept, require regular injections to maintain vision. But Ixo-vec works differently by using a single gene therapy injection aimed at providing long-term production of a protein that combats the disease, potentially reducing the need for frequent treatments. Researchers are excited about Ixo-vec because it promises a more convenient option with sustained benefits, addressing a significant limitation of current therapies.

Research has shown that Ixo-vec, which participants in this trial may receive, holds promise for treating wet age-related macular degeneration (nAMD). In earlier studies, Ixo-vec was well-tolerated and helped maintain vision, with improvements in eye health. More than 50% of patients did not require additional treatments for a year. The treatment also significantly reduced the number of injections needed compared to standard care. These findings suggest that Ixo-vec could effectively manage nAMD by reducing the treatment burden while maintaining vision.¹²³⁴⁶