Regenxbio (RGNX) investor relations material

Pipeline and milestones

• ATMOSPHERE and ASCENT topline results expected in Q4 2026, with a joint release anticipated with AbbVie; global regulatory submissions are planned for 2027.

• The pivotal program enrolled more than 1,300 participants across 200+ sites in 10+ countries; both studies completed enrollment in 2025.

• RGX-202 for Duchenne is targeting a pre-BLA meeting by year-end 2026, a BLA filing in Q1 2027 and potential FDA approval in 2027.

• The diabetic retinopathy pivotal program began in 2026; RGX-121 for MPS II was placed on FDA clinical hold in August 2026.

Wet AMD pivotal program

• Sura-vec (surabgene lomparvovec; ABBV-RGX-314) is investigational and unapproved; an AAV8 vector delivers an anti-VEGF Fab by subretinal injection, aiming for durable VEGF inhibition after one administration.

• ATMOSPHERE and global ASCENT compare low- and high-dose sura-vec with active controls in treatment-experienced patients; BCVA change at week 54 and non-inferiority are primary measures.

• ATMOSPHERE uses monthly ranibizumab control in the U.S.; ASCENT uses on-label aflibercept Q8W control globally.

• The trials assess anatomy, injection-burden reduction and tolerability; stated success criteria include >50% lower injection burden and a meaningful injection-free proportion.

• The program includes patients across low to high disease activity, week-one ranibizumab response screening and fellow-eye treatment cohorts.

Clinical evidence and safety

• Earlier studies reported stable-to-improved vision; annualized injection reductions were 61.3%–92.8% across groups, with 53%–60% of pharmacodynamic-study participants injection-free at one year.

• Phase 1/2a follow-up showed vision maintained through five years; year-five annualized injection rates were 2.4 and 2.5 in the two cohorts.

• Five-year Phase I data showed cohort 3 gained nearly 9 letters and cohort 4 lost 1 letter; annual injections fell 65% and 64%, respectively.

• At pivotal doses, safety summaries across 71 eyes reported no drug-related serious adverse events or central macular pigmentary changes; observed pigmentary changes were peripheral, stabilized and had no BCVA impact.

• Subretinal delivery places the bleb in the far periphery; prophylactic steroids are not required beyond routine post-vitrectomy care.