Interim 26-week results from the ongoing phase 2 LUNA study of ixoberogene soroparvovec (Ixo-vec, Adverum Biotechnologies) in neovascular age-related macular degeneration (nAMD) were presented at the 2024 American Society of Retina Specialists (ASRS) Annual Meeting. The multicenter, randomized, double-masked trial is evaluating the safety, tolerability, and efficacy of a single intravitreal injection of Ixo-vec in anti-VEGF treatment–experienced nAMD patients over 60 months.

Investigators reported data on 60 participants with a mean follow-up of 35.2 weeks. Patients were randomized to receive either 6x10¹⁰ or 2x10¹¹ vector genomes (vg)/eye of Ixo-vec, along with various prophylactic corticosteroid regimens. At baseline, participants had a mean age of 76.6 years, 3.0 years since nAMD diagnosis, and had received a mean of 10.1 annualized anti-VEGF injections prior to Ixo-vec.

The interim analysis revealed that all Ixo-vec–related adverse events were mild or moderate, with no cases of serious ocular inflammation or vascular events. Local corticosteroid prophylaxis effectively minimized inflammation in 85.3% of participants through week 26. Mean best-corrected visual acuity (BCVA) and central subfield thickness (CST) were maintained in both dose groups. Notably, Ixo-vec reduced the mean annualized anti-VEGF injection frequency by 90% (6x10¹⁰) and 95% (2x10¹¹), with 76% and 83% of participants requiring no supplemental injections, respectively.

The phase 2 LUNA study will continue to evaluate the long-term safety and efficacy of Ixo-vec in nAMD, with the aim of informing future phase 3 registrational trials. If successful, this gene therapy could provide an alternative to frequent anti-VEGF injections for patients with this sight-threatening condition.