Regenxbio (RGNX) Investor update summary

Pipeline and milestones

• Top-line results from both pivotal wet AMD studies expected in Q4 2026, with a joint release anticipated with AbbVie; regulatory submissions targeted for 2027, with AbbVie holding the IND and leading the BLA filing.

• Duchenne program expects a pre-BLA meeting by year-end 2026 and BLA filing in Q1 2027.

• AbbVie partners on sura-vec across retinal disease areas; NS Pharma partners on rare disease programs, including Duchenne.

• Rockville manufacturing capacity: nearly 350,000 annual sura-vec doses and up to 2,500 annual Duchenne doses; facility inspection for RGX-121 had no observations.

Wet AMD clinical program

• Sura-vec uses an anti-VEGF fab gene, AAV8 vector and subretinal delivery, designed for targeted, durable protein expression in the eye.

• ATMOSPHERE and ASCENT completed enrollment in 2025; both compare low- and high-dose sura-vec with active controls, with BCVA change at week 54 and non-inferiority as the primary endpoint.

• ATMOSPHERE is U.S.-based with monthly ranibizumab control; global ASCENT uses on-label aflibercept Q8W control.

• Trials include patients across low to high disease activity; screening assesses response to ranibizumab at week 1, and the program includes fellow-eye treatment cohorts.

• Management identified meaningful treatment-burden reduction and a substantial share of patients needing no supplemental injections as key measures of pivotal success.

Early efficacy and safety signals

• Phase I/II and fellow-eye studies showed maintained or improved vision: gains of 3.9 and 3.3 letters in phase I high and second doses, 2.7 and 1.1 letters in phase II high and low doses, and 0.4 letters in the fellow eye study.

• Reported treatment-burden reductions ranged from 61% to 68% in phase I, over 75% in phase II, and 93% in the fellow-eye study; injection-free rates were 53% and 60% in phase II arms and 60% in fellow eyes.

• Five-year phase I follow-up showed cohort 3 gained nearly 9 letters and cohort 4 lost 1 letter; annual injections fell 65% and 64% in those cohorts.

• At pivotal doses, panelists reported no drug-related serious adverse events or central macular pigmentary changes; observed pigmentary changes were peripheral, stabilized over time and had no BCVA impact.

• Delivery optimization places the subretinal bleb in the far periphery; no prophylactic steroids are required, while routine post-vitrectomy steroid taper is used.