Adverum Biotechnologies provided a new update to its OPTIC phase 1 trial of the gene-derived anti-VEGF ADVM-022 for wet AMD, this time reporting that all 6 patients in high-dose cohort 1 continued to be injection free for more than a year, with at least 1 patient going 92 weeks without retreatment. In high-dose cohort 4, 8 of 9 patients were injection free at 16 weeks.

In low-dose cohorts 2 (n=6) and 3 (n=9), 10 of 15 patients remained injection free at periods up to 64 weeks. Overall, patients maintained stable vision with a mean small reduction in retinal thickness. Ocular inflammation, when observed, has been responsive to steroid eye drops and overall is decreasing over time. The first pivotal trial in wet AMD is planned for mid-2021.

“With these impressive OPTIC data and the removal of the partial clinical hold on ADVM-022 by the FDA, our goal is to continue to advance into pivotal trials to demonstrate the transformative potential of our gene therapy,” said Laurent Fischer, MD, CEO of Adverum, in a news release. “We are excited that ADVM-022 has the potential to be a ‘one and done’ IVT injection that may dramatically reduce the treatment burden for the millions of patients with wet AMD and DME worldwide. Particularly during COVID-19, we are reminded of the benefits that ADVM-022, a novel gene therapy that has demonstrated long-term treatment benefit after one in-office IVT injection, could deliver to patients.”