RGX-314 cohort 3 shows +8 letters

REGENXBIO reported 24-week Phase 1 data on October 28, 2018 showing its gene therapy RGX-314 was generally well tolerated in patients with wet age-related macular degeneration and produced early signs of visual benefit. The update, presented at the American Academy of Ophthalmology meeting and reported by HCPLive, focused on cohort 3 of an open-label dose-escalation study in previously treated patients. Peter Campochiaro of Johns Hopkins Medicine said the mean change in best-corrected visual acuity in that cohort was +8 letters at 24 weeks. The same group averaged 1.3 anti-VEGF rescue injections over six months after a single administration, according to the report. ### Which patients were in the cohort that produced the +8-letter result? HCPLive reported that cohort 3 included six patients with a median age of 80 years who had been treated for wet AMD for years before entering the study. Those patients had a mean of 34.2 anti-VEGF injections since diagnosis and a median of 71.6 months since their first anti-VEGF injection, according to the report. (hcplive.com) ClinicalTrials.gov describes wet AMD as a disease typically managed with frequent anti-VEGF injections and identifies ABBV-RGX-314, also known as RGX-314, as a potential one-time gene therapy treatment. The study population in the suprachoroidal AAVIATE trial is different from the earlier subretinal Phase 1/2a program cited in the 2018 report, but both programs target patients who have already needed repeated anti-VEGF therapy. (hcplive.com) ### What exactly is RGX-314 designed to do inside the eye? The Lancet reported that RGX-314, now referred to as ABBV-RGX-314, uses an adeno-associated virus serotype 8 vector to deliver genetic instructions for an anti-VEGF-A antigen-binding fragment. That approach is intended to provide sustained VEGF suppression after a single administration, rather than requiring repeated injections of conventional anti-VEGF drugs. (clinicaltrials.gov) REGENXBIO says the therapy is being developed with AbbVie as a potential one-time treatment for wet AMD, diabetic retinopathy and other chronic retinal diseases treated with anti-VEGF agents. The companies now refer to the program as surabgene lomparvovec, or sura-vec, on REGENXBIO’s pipeline page. ### How much treatment burden changed in the early study? HCPLive reported that cohort 3 patients averaged 1.3 injections over the six months after treatment with RGX-314. (thelancet.com) The same article said those patients had required substantial anti-VEGF treatment before enrollment, which is why the post-treatment injection count drew attention in the conference presentation. (regenxbio.com) A REGENXBIO presentation posted in 2021 showed the company continued tracking rescue injection use across later dose groups in the Phase I/IIa program. That deck also described RGX-314 as generally well tolerated across 42 treated patients at the time of the presentation. ### How does this early cohort fit into the broader development program now? (hcplive.com) AbbVie and REGENXBIO said on January 13, 2025 that pivotal data for the subretinal delivery of ABBV-RGX-314 in wet AMD are expected in 2026. The companies named the two pivotal studies as ATMOSPHERE and ASCENT. ClinicalTrials.gov lists one pivotal wet AMD study, NCT04704921, as evaluating mean change from baseline in best-corrected visual acuity at Week 54 relative to ranibizumab. (regenxbio.com) The registry says about 630 participants are expected to enroll across three arms. ### Is the 2018 cohort update the latest evidence available on RGX-314? The 2018 HCPLive report is an early readout from the original Phase 1 program, not the latest program-wide evidence. (news.abbvie.com) The Lancet published longer-term results from the subretinal Phase I/IIa study in 2024, reporting that subretinal RGX-314 was generally well tolerated and that the findings informed the pivotal program. (clinicaltrials.gov) January 2025 is the most recent company update surfaced in this search on the wet AMD program’s next milestones. AbbVie and REGENXBIO said then that pivotal wet AMD data from ATMOSPHERE and ASCENT are expected in 2026, while ClinicalTrials.gov continues to list active studies for both suprachoroidal and pivotal subretinal development. (clinicaltrials.gov) (thelancet.com)