A majority of patients with wet AMD receiving a single injection of an AAV-delivered gene therapy delivering aflibercept did not require a second injection of aflibercept over 2 years, according to data reported at the Retina Society meeting in Chicago. Arshad M. Khanani, MD, of Sierra Eye Associates in Reno, Nevada, presented data from the phase 1 OPTIC study, in which Adverum Biotechnologies’ ADVM-022 was assessed for safety and reliability.

OPTIC enrolled 30 participants in 4 study arms receiving either 2x10¹¹ viral genomes (vg) or 6x10¹¹ vg of ADVM-022 and receiving steroid prophylaxis of injection-related inflammation either orally for 13 days or in eye drops for 6 weeks. Patients receiving the lower dose of ADVM-022 experienced an 83% reduction in the number of anti-VEGF injections they received, while those receiving the higher dose experienced a 97% reduction. Best-corrected visual acuity was maintained in all 4 study arms. Central subfield thickness (CST) was also maintained in 3 of the 4 groups, whereas in the group receiving the low dose and eye-drop protocol, CST improved by -142.3 µm as of data dating from July. ADVM-022 was related to only mild or moderate adverse events.

Adverum intends to launch a 5-year follow-up study of the 2x10¹¹-vg dose and will possibly also study even lower doses of ADVM-022 to determine the threshold of efficacy for the drug. If approved, ADVM-022 could significantly relieve the treatment burden in wet AMD patients.