Arshad Khanani, MD: ADVM-022 Insights from OPTIC Trial Cohorts 1-3

After presenting initial data from Cohorts 1 and 2 of the phase 1 OPTIC trial in 2019, Cohort 3 results announced by Adverum Biotechnologies are adding to the excitement around ADVM-022 and the prospect of gene therapy in ophthalmology.

The latest data from Adverum Biotechnologies on the intravitreal gene therapy for wet age-related macular degeneration (wAMD) indicates ADVM-022 was well-tolerated across all 3 cohorts and, in Cohort 3, use resulted in a mean gain of 6.8 letters in BCVA among with 20 weeks of follow-up.

“It’s impressive to see the long-term durability demonstrated at the higher dose of ADVM-022 in a patient population that previously required frequent injections to maintain their vision and are now beyond one year of follow-up with no rescue injections,” said Arshad Khanani, MD, director of clinical research, Sierra Eye Associates and principal investigator in the OPTIC trial, in a statement from Adverum. “Additionally, preliminary evidence in Cohort 3 shows vision and anatomical improvements and that the 6-week prophylactic steroid eye drop regimen is effective at minimizing early ocular inflammation.”

Originally scheduled to be presented at the 2020 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, Adverum announced the new data in a webcast on Monday, May 4. Among the highlights of the webcast was the long-term durability of the agent—demonstrated through the absence of rescue injections in Cohort 1 and no ADVM-022 related serious adverse events or non-ocular adverse events from all 3 cohorts.

Data from Cohort 3 of OPTIC also indicated a mean BCVA gain of 6.8 letters and a mean reduction of 137.8 mm from baseline among 5 patients with 20 weeks of follow-up. When examining the 4 rescue free patients from this group, patients experienced a mean gain of 8.8 letters and a mean reduction of 149.8 mm.

To learn more about ADVM-022 and the ongoing OPTIC trial, HCPLive® caught up with Khanani and that discussion is the subject of our latest ARVO 2020 House Call.