Regenxbio Inc. · NASDAQ:RGNX
Regenxbio — Q2 2025 Earnings Presentation
13 slides from Regenxbio's earnings presentation, Aug 7, 2025
Slide 1: Suprachoroidal Delivery of Sura-vec (ABBV-RGX-314): 2-Year Results in NPDR
Suprachoroidal Delivery of Sura-vec (ABBV-RGX-314): 2-Year Results in Non-Proliferative Diabetic Retinopathy The Phase II ALTITUDE® Study and Long-Term Follow-Up August 7, 2025 ABBV-RGX-314 is an investigational product that has not been approved by any regulatory authority. No conclusions regarding
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are gen
Slide 3: Diabetic Retinopathy is a Global Public Health Problem
Diabetic Retinopathy is a Global Public Health Problem 20M <1% 45-50 YRS ANNUAL DIABETIC Is the expected DR Of patients with Median age of Early treatment PDR RETINOPATHY NPDR patient population in early DR are disease onset with longer lasting 5M 13M PATIENTS US,EU,JP in the next treated due to hig
Slide 4: One time, in-office injection of gene therapy could potentially provide long-lasting improvement in DR
One time, in-office injection of gene therapy could potentially provide long-lasting improvement in DR severity and reduce risk of vision-threatening complications Potential Advantages of Suprachoroidal Space (SCS) Delivery SCS injection is a fast, simple and convenient in- office procedure Targeted
Slide 5: Phase II ALTITUDE Study Design
Phase II ALTITUDE® Study Design Moderately Severe NPDR, Severe NPDR, or Mild PDR Subjects without active CI-DME 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 2 2.5 3 4 5 Months Sura-vec SCS Admin
Slide 6: Baseline Characteristics
Baseline Characteristics NPDR (DRSS 47-53) Observational Dose Level 1 Dose Level 2 Dose Level 3 Total Control (N=6) (N=24) (N=14b) (N=56) VARIABLE (N=12) 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 7: 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 8: No Intraocular Inflammation in NPDR Subjects at 2 Years with Short-course Prophylactic Topical Steroids
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 9: Change in DRSS at 1 Year
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 10: ≥2-Step DRSS Improvement Without Additional DR Treatment at 2 Years
≥2-Step DRSS Improvement Without Additional DR Treatment at 2 Years Dose Level 3 sura-vec treated subjects outperformed all other groups 100.0% YEAR 1 YEAR 2 90.0% 80.0% ~6x increase % of Total Patients 70.0% 60.0% 50% 50.0% 40.0% 30.0% 22% 21% 20.0% 18% 10.0% 8% 8% 0.0% ALTITUDE DL2 DL3 Protocol Wb
Slide 11: DRSS Outcomes in Subjects over 2 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 12: Vision Threatening Events Over 2 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 (n=133) (n=164)
Slide 13: 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