Wet AMD treatment AVA-101 meets safety endpoint

The subretinal gene therapy injection treatment for wet age-related macular edema AVA-101 met its 12-month primary endpoint based on ophthalmic and systemic safety in a phase 2a trial, according to a press release from Avalanche Biotechnologies.

AVA-101 contains a gene encoding sFlt-1 anti-VEGF protein and AAV2 vector in a single subretinal injection intended to reduce the burden of repeated injection treatments, according to the release.

In the study, 21 subjects in the AVA-101 treatment group also received an injection of Lucentis (ranibizumab, Genentech) at day 0 and week 4. The 11 subjects in the control group received the ranibizumab injections as well.

No serious adverse events related to AVA-101 were observed in the trial.

Secondary endpoints included mean change from baseline in BCVA, the number of ranibizumab rescue injections and mean change from baseline in central retinal thickness as measured by SD-OCT, according to the release.

Of the 21 subjects in the treatment group, 23.8% had best corrected visual acuity improvement of at least 10 letters with two or fewer rescue injections in the 12-month follow-up, while none of the 11 subjects in the control group achieved those results.

From baseline, mean retinal thickness increased in the treatment group and decreased in the control group.

"The results of this study confirm the phase 1 safety results and suggest that AVA-101 could potentially benefit a significant portion of patients with wet AMD who require regular treatment with anti-VEGF therapy," Avalanche Chief Medical Officer Samuel B. Barone, MD, said in the release.

The company plans to initiate the phase 2b study later this year, the release said.