Regenxbio Completes Enrollment in ATMOSPHERE and ASCENT Trials of Sura-Vec Gene Therapy for Wet AMD
Regenxbio announced the completion of enrollment in its two pivotal phase 3 studies—ATMOSPHERE and ASCENT—evaluating surabgene lomparvovec (sura-vec, ABBV-RGX-314), an investigational one-time gene therapy for wet age-related macular degeneration (AMD) delivered via subretinal injection.
“Completing enrollment in this large, global pivotal program is an exciting milestone in our efforts to deliver sura-vec to patients as the potential first gene therapy for wet AMD,” said Steve Pakola, MD, Chief Medical Officer of Regenxbio. “The millions of patients worldwide with wet AMD are in need of a treatment option that can preserve vision, prevent disease progression, and reduce the significant burden of frequent, life-long eye injections required with today’s standard of care. We are highly encouraged by the safety and long-term durability seen in multiple earlier-stage trials. We look forward to sharing topline data next year and advancing global registration of this potentially transformative treatment.”
The ATMOSPHERE and ASCENT trials are multicenter, randomized, active-controlled pivotal studies designed to support global regulatory submissions for sura-vec.
ATMOSPHERE, conducted in the United States, compares sura-vec to ranibizumab
ASCENT, conducted in the US and 13 other countries, compares sura-vec to aflibercept
Together, the studies have enrolled more than 1,200 participants across over 200 clinical sites worldwide.
The primary endpoint in both studies is noninferiority in best corrected visual acuity (BCVA) from baseline at 54 weeks and 1 year, respectively.
Secondary endpoints include safety and tolerability, change in central retinal thickness (CRT), and reduction in supplemental anti-VEGF injection requirements.
Topline data are anticipated in the fourth quarter of 2026.
In a long-term follow-up study, sura-vec demonstrated a durable treatment effect with stable or improved vision for up to 4 years. A separate phase 2 pharmacodynamic study using the same dose levels as the pivotal program reported that sura-vec was well tolerated in 60 treated participants, with no drug-related serious adverse events. Participants showed stable or improved vision and anatomy, and a meaningful reduction in anti-VEGF injection frequency, underscoring the therapy’s potential to reduce treatment burden.
About Surabgene Lomparvovec (Sura-Vec, ABBV-RGX-314)
Sura-vec is being developed as a one-time gene therapy for wet AMD, diabetic retinopathy, and other chronic retinal diseases. It uses Regenxbio's NAV AAV8 vector, which carries a gene encoding an antibody fragment that inhibits vascular endothelial growth factor (VEGF). By targeting this pathway, sura-vec aims to provide long-lasting suppression of VEGF, reducing the need for ongoing intravitreal injections.