Regenxbio Inc. · NASDAQ:RGNX
Regenxbio — AAO 2025
23 slides from Regenxbio's conference presentation, Oct 17, 2025
Slide 1: Suprachoroidal Sura-vec: First Time 2-Year Results in NPDR
Suprachoroidal Surabgene Lomparvovec (Sura-vec, ABBV-RGX-314*) First Time 2-Year Results in NPDR Charles C. Wykoff MD PhD *Sura-vec is an investigational product that has not been approved by any regulatory authority. No conclusions regarding the safety and efficacy of the product can be made. © 202
Slide 2: Disclosures
Disclosures 4DMT (C, R), AbbVie (C), Adverum (C,R), Aerie (C), AffaMed (R), Alcon (C), Alexion (R), Alimera (C,R), Allgenesis (C, R), Annexon (C,R), Apellis (C, R), Ascidian (C), Aviceda (C), Bausch + Lomb (C), Bayer (C,R), Beacon (C), Bionic Vision (C), Boehringer Ingelheim (C,R), Clearside (C,R),
Slide 3: Ranibizumab = 2017, Aflibercept = 2019
Ranibizumab = 2017 Aflibercept = 2019
Slide 4: ≈80% do not consider anti-VEGF for NPDR w/o DME
≈80% do not 2025 consider anti-VEGF for NPDR w/o DME N=985
Slide 5: Key Barriers to Clinical Adoption of Intravitreal injections for NPDR w/o DME
Key Barriers to Clinical Adoption of Intravitreal injections for NPDR w/o DME Repeated Injections &
Slide 6: Leading Cause of Vision Loss
Leading Cause of Vision Loss
Slide 7: More advanced DR is associated with greater probability of blindness
More advanced DR is associated with greater probability of blindness Sustained Blindness (SB), % 1.000 Index DR Severity Probability of Not 0.975 Mild NPDR Survival Curve Unspecified Developing Moderate NPDR 0.950 Severe NPDR 2 years after DR Dx, eyes with baseline 0.925 severe NPDR & PDR were 3.6 &
Slide 8: Unmet need: better, more durable, safer therapies
Unmet need: better, more durable, safer therapies
Slide 9: Challenges & Failures
Challenges & Failures Systemic or Non-Intravitreal Bolus Therapies to Slow DR Progression • Oral Pills – APX-3330 Oral Delivery – CU06-1004 – ASP8232 – OPL-0401 – Vicasinabin (RG7774) – Runcaciguat – Ruboxistaurin – Vatalanib – Ripasudil – Pazopanib • Eye Drops Eye – Nesvategrast (OTT166 5%) Drops
Slide 10: Sura-vec: AAV8-based Investigational Gene Therapy Designed to Produce an Anti-VEGF Fab
Sura-vec: AAV8-based Investigational Gene Therapy Designed to Produce an Anti-VEGF Fab Artistic View of Suprachoroidal Delivery Thermal Imaging of in-office SCS delivery of sura-vec in ALTITUDE subject 10
Slide 11: Goal of one-time in-office SCS injection of gene therapy
The goal of a one-time, in-office SCS-injection of gene therapy is to provide long-lasting improvement in DR severity and reduce risk of vision- threatening complications IMPROVE GOAL of ANTI-VEGF Disease Severity GENE THERAPY Stable WORSE Vision Threatening Complications Including DME, PDR & NVG VI
Slide 12: ALTITUDE: Phase II Randomized Dose-escalation Study Design
ALTITUDE®: A Phase II, Randomized, Dose-escalation, Observation-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Investigational Sura-vec via Suprachoroidal Injection in DR Primary Objective Route of Administration ▪ Evaluate proportion of patients with ≥2 step improvement in ▪
Slide 13: Phase II ALTITUDE Study Design
Phase II ALTITUDE® Study Design Sura-vec for the treatment of diabetic retinopathy ALTITUDE LTFU Baseline Treatment Evaluation Long Term Follow Up Assessment DR Treatment at Investigator Discretion Screening Day 1 M3 M6 M9 Year 1 1.5 Year 2 2.5 3 4 Year 5 Sura-vec SCS Administration Primary Endpoint
Slide 14: Baseline Characteristics
Baseline Characteristics NPDR (DRSS 47-53) Observational Control Dose Level 1 Dose Level 2 Dose Level 3 Total VARIABLE (N=12) (N=6) (N=24) (N=14b) (N=56) Mean Age (Years) 54.9 57.2 60.1 54.4 57.2 Gender – Female 3(25.0%) 4 (66.7%) 10 (41.7%) 4 (28.6%) 21 (37.5%) Hemoglobin A1c- Mean 7.7 7.9 7.8 7.7
Slide 15: Change in DRSS at Year 1
Change in DRSS at 1 Year Dose Level 2 and 3 sura-vec treated subjects outperform control with similar outcomes at 1 yr Control Dose Level 1 Dose Level 2 Dose Level 3 C1, C2, + C4 (n=12) 1 Year Sura-vec (n=6) 1 Year Sura-vec (n=24) 1 Year Sura-vec (n=14**) 1 Year 90 90 90 90 80 80 80 80 70 70 70 70 ≥
Slide 16: ≥2-Step DRSS Improvement Without Additional DR Treatment Through Year 2
≥2-Step DRSS Improvement Without Additional DR Treatment Through Year 2 Dose Level 3 sura-vec treated subjects outperformed all other groups 100.0% YEAR 1 YEAR 2 90.0% 80.0% ~6x increase % of Patients with ≥ 2 Step DRSS 70.0% 60.0% 50% Improvement 50.0% 40.0% 30.0% 22% 21% 20.0% 18% 10.0% 8% 8% 0.0%
Slide 17: DRSS Outcomes in Dose Level 3 Subjects over Years
DRSS Outcomes in Dose Level 3 Subjects over 2 Years Majority of subjects improved ≥ 2 steps with a single injection of sura-vec Dose Level 3 Baseline DRSS Sura-vec Year 1 Year 2 53 2 Steps Better 3 Steps Better 47 2 Steps Better 2 Steps Better 47 1 Step Better 2 Steps Better NO ADDITIONAL DR TREATME
Slide 18: Vision Threatening Events Over Years
Vision Threatening Events Over 2 Years ≥ 70% risk reduction in VTEs observed in DL3 subjects treated with a single dose ≥70% Reduction in VTEs 100 90 HISTORICAL CONTROLS 80 OVER 2 YEARS 70 Subjects, % 60 50.4% 48.0% 50 40 33.0% 30 25.0% 20 14.3% 10 0 PANORAMAa PROTOCOL Wb DL1 DL2 DL3 Sham Sham Sura-
Slide 19: Cumulative Safety Summary
Cumulative Safety Summary Dose Levels 1–3 Through 2 Years: NPDR (DRSS 47-53) • 16 SAEs: none considered drug-related Sura-vec has been well–tolerated in Dose Levels 1, 2 and 3 NPDR subjects (n=45) • No cases of vasculitis, occlusion, hypotony, or chorioretinitis • Zero IOI with short course of proph
Slide 20: No Intraocular Inflammation in Dose Level 3 NPDR Subjects at 2 Years
No Intraocular Inflammation in Dose Level 3 NPDR Subjects at 2 Years with Short-course Prophylactic Topical Steroids ANTERIOR CHAMBER CELLS, FLARE, OR PIGMENTED CELLS Topical Steroid None Drops 7 weeks Trace 1+ 2+ 3+ Dose Level 3 4+ (n=15) Time D2 W1 M1 M3 M6 M9 1Y 1.5Y 2Y VITREOUS CELLS, FLARE, OR
Slide 21: Case #1: Dose Level 3
Case #1: Dose Level 3 Courtesy of Amr Dessouki, MD 2-Step Improvement Baseline Year 1 Year 2 DRSS 53 DRSS 53 DRSS 43 Vol: 9.0 mm3 Vol: 8.8 mm3 Vol: 8.6 mm3 Data cut: June 09, 2025. This slide presents results from individual participants and is not indicative of outcomes experienced by all participa
Slide 22: Case #2: Dose Level 3
Case #2: Dose Level 3 Courtesy of Charles Wykoff, MD, PhD 1-Step Improvement 2-Step Improvement Baseline Year 1 Year 2 DRSS 47 DRSS 43 DRSS 35 Vol: 9.3 mm3 Vol: 9.2 mm3 Vol: 8.8 mm3 Data cut: June 09, 2025. This slide presents results from individual participants and is not indicative of outcomes ex
Slide 23: Sura-vec 2 Year Results in NPDR Subjects from the Phase II ALTITUDE Study
Sura-vec 2 Year Results in NPDR Subjects from the Phase II ALTITUDE® Study Single in-office suprachoroidal injection of sura-vec Well-tolerated at Dose Levels 1, 2, and 3 No IOI in Dose Level 3 NPDR subjects with short-course topical steroids No drug-related serious adverse events. No cases of vascu