Adverum Biotechnologies, Inc. announced initial aflibercept protein expression data from its ongoing Phase 2 LUNA trial evaluating ixoberogene soroparvovec (Ixo-vec) for the treatment of wet age-related macular degeneration (wet AMD). New data announced include aflibercept protein levels for both the 2E11 and 6E10 vg/eye doses, suggesting that both doses are within the therapeutically active range. Unlike the current standard of care that requires multiple bolus injections in the eye every year to maintain sufficient levels of aflibercept, gene therapy has the potential to dramatically reduce treatment burden for the life of the patient by delivering durable therapeutic levels of anti-VEGF.

The aflibercept levels across the 2E 11 and 6E10 doses are in the range observed in the OPTIC trial and in the range that was seen in previously presented non-human primate data. The efficacy of Ixo-vec at the 2E11 dose in OPTIC remain very promising. Ixo-vec utilizes the 7m8 capsid developed via directed evolution to enhance retinal transduction and to deliver therapy where it is needed most.

These results from the LUNA study could be clinically meaningful as the aflibercept levels measured in this trial are comparable to the sustained aflibercept levels seen in patients treated in the OPTIC trial. Ixo-vec i is an aflibercept-encoding AAV.7m8 vector designed via directed evolution to cross the inner limiting membrane, enabling in-clinic IVT delivery and enhanced retinal transduction. Unlike other ophthalmic gene therapies that require surgery to administer the gene therapy under the retina (sub-retinal approach), Ixo-vec is designed to be administered as a one-time IVT injection in the physician's office, deliver long-term efficacy, reduce the burden of frequent anti-vascular endothelial growth factor (VEGF) injections, optimize patient compliance and improve vision outcomes for patients with wet AMD.

In recognition of the need for new treatment options for wet AMD, the U.S. Food and Drug Administration granted Fast Track designation for Ixo-vec for the treatment of wet AMD. Ixo-vec has also received PRIME designation from the European Medicines Agency and the Innovation Passport from the United Kingdom'sMedicines and Healthcare Products Regulatory Agency for the treatment of wetAMD.