Regenxbio Completes Enrollment in Pivotal Phase 3 Trials of Sura-Vec Gene Therapy for Wet AMD

Regenxbio Inc. has announced the completion of enrollment in its two global, pivotal Phase 3 trials, ATMOSPHERE and ASCENT, evaluating surabgene lomparvovec (sura-vec; ABBV-RGX-314), an investigational, one-time gene therapy candidate 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 at 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.”

About the Phase 3 Program

ATMOSPHERE and ASCENT are multicenter, randomized, active-controlled studies designed to support regulatory submissions for sura-vec globally.

       • ATMOSPHERE is being conducted in the United States and compares sura-vec to ranibizumab.

       • ASCENT includes the US and 13 other countries, comparing sura-vec to aflibercept.

Together, the trials have enrolled more than 1,200 participants across over 200 clinical sites worldwide.

The primary endpoint for both studies is noninferiority in best corrected visual acuity (BCVA) from baseline at 54 weeks (ATMOSPHERE) and one year (ASCENT).
Secondary endpoints include:

       • Safety and tolerability

       • Changes in central retinal thickness (CRT)

       • Reduction in supplemental anti-VEGF injections

Topline data are expected in Q4 2026.

Supporting Data from Earlier Studies

In a long-term follow-up study, sura-vec demonstrated a durable treatment effect, with stable or improved vision for up to four years. Separately, a Phase 2 pharmacodynamic study using the same dose levels as the pivotal program showed that sura-vec was well tolerated in 60 treated participants, with no drug-related serious adverse events reported. Participants experienced:

       • Stable or improved vision and anatomical outcomes

       • A meaningful reduction in the frequency of anti-VEGF injections

These results support the therapy’s potential to reduce treatment burden for patients with chronic retinal conditions.