Adverum Biotechnologies — FLORetina December 2025
19 slides from Adverum Biotechnologies's conference presentation, Dec 4, 2025
Slide 1: Ixo-vec Gene Therapy for nAMD: Clinical Progress from the LUNA Phase 2 Trial
Exhibit 99.1 FLORetina • December 2025 Ixo-vec Gene Therapy for nAMD: Clinical Progress from the LUNA Phase 2 Trial Charles C. Wykoff, M.D., Ph.D. Director of Research, Retina Consultants of Texas Professor of Clinical Ophthalmology, Blanton Eye Institute, Houston Methodist Hospital Disclosures: C=
Slide 2: Forward-Looking Statements
Forward-Looking Statements Statements contained in this document and any accompanying presentation regarding matters, events, statistics, or clinical or financial results that may occur in the future are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act
Slide 3: Ixo-vec: A Potentially Transformative Therapy for Wet AMD
Ixo-vec: A Potentially Transformative Therapy for Wet AMD Ixo-vec Gene therapy designed to preserve vision with a single intravitreal injection Single intravitreal Gold standard vector Transgene encoding Biofactory approach for injection in routine office for IVT gene therapy aflibercept, a clinical
Slide 4: Observations from OPTIC and LUNA Informed Phase 3 Trials
Observations from OPTIC and LUNA Informed Phase 3 Trials * ARTEMIS enrollment completed Population: Previously treated wet AMD patients with a high anti-VEGF injection burden 2-year Outcomes 5-year Outcomes 4-year Outcomes Ixo-vec OPTIC Phase 1 Study (N=30) Extension Study Corticosteroid Prophylaxis
Slide 5: OPTIC Trial 2E11 vg/eye: Marked and sustained reduction in treatment burden (4-year results)
Phase 1 OPTIC Trial 2E11 vg/eye: Marked and sustained reduction in treatment burden (4-year results) BCVA and CST Maintained Through 4 Years Sustained Reduction in Injection Burden OPTIC OPTIC EXT Cumulative Annualized Reduction 12 9.9 500 Mean (90% CI) CST 10 84% 81% 84% 86% 400 8 6 300 4 200 1.9 1
Slide 6: Ixo-vec Demonstrated Durable Therapeutic Levels After One Injection
Ixo-vec Demonstrated Durable Therapeutic Levels After One Injection Overlapping expression patterns across all 3 doses & 6E10 vg/eye consistent with higher doses Early aflibercept levels are predictive of long-term protein expression 100,000 1 NHP studies suggest aflibercept levels are ~ 7x higher i
Slide 7: Observations from OPTIC and LUNA Informed Phase 3 Trial Design
Observations from OPTIC and LUNA Informed Phase 3 Trial Design Population: Previously treated wet AMD patients with a high anti-VEGF injection burden 2-year Outcomes 5-year Outcomes 4-year Outcomes Ixo-vec OPTIC Phase 1 Study (N=30) Extension Study Corticosteroid Prophylaxis 6E11 vg/eye Key Safety a
Slide 8: Demographics and Baseline Characteristics
Demographics and Baseline Characteristics LUNA evaluated Ixo-vec in nAMD patients with a high injection burden 6E10 vg/eye 2E11 vg/eye Total N=30 N=30 N=60 Characteristics Mean (SD) age, years 75.4 (8.2) 77.7 (7.4) 76.6 (7.8) Female, n (%) 16 (53) 18 (60) 34 (57) Race, n (%) White 27 (90) 28 (93) 55
Slide 9: Sustained Anatomic Control and Visual Acuity Through 2 Years
Sustained Anatomic Control and Visual Acuity Through 2 Years Robust and durable clinical activity observed in both dose groups Least Squares Mean Change in Central Subfield Thickness (CST) 150 LS Mean (95% CI) 6E10 (n=30) 2E11 (n=30) 100 Change at 2 Years 50 9.8 (-10.7, 30.2) 6E10 vg/eye 0 -14.8 (-3
Slide 10: Robust and Consistent Reduction in Injection Burden
Robust and Consistent Reduction in Injection Burden 46%–61% of participants received ≤1 injection cumulatively through 2 years High Need Population: Total Injections (2 Years) Least Squares Mean Change in Central Subfield Thickness (CST) 150 LS Mean (95% CI) 100 Change at 2 Years 50 9.8 (-10.7, 30.2
Slide 11: Robust and Consistent Reduction in Injection Burden (Low Burden Population)
Robust and Consistent Reduction in Injection Burden * 75%−100% of participants with lower treatment burden received ≤1 injection cumulatively through 2 years Low Burden Population: Total Injections (2 Years) Least Squares Mean Change in Central Subfield Thickness (CST) 150 LS Mean (95% CI) 100 Chang
Slide 12: Robust and Consistent Reduction in Injection Burden Through Year 2
Robust and Consistent Reduction in Injection Burden Through Year 2 ~90% reduction in mean annualized anti-VEGF injections in a high need population Mean Prior Annualized Injection Rate Mean Post Ixo-vec Annualized Injection Rate 11 10.2 10.0 10 9 8 7 –89%–89% –92%–90% 6 5 4 3 2 1.2 1.1 0.9 0.8 1 0 1
Slide 13: Robust Reduction in Injection Burden in a High Need Population
Robust Reduction in Injection Burden in a High Need Population 65%–74% of participants received ≤1 annualized injection through 2 years Mean Prior Annualized Injection Rate Mean Post Ixo-vec Annualized Injection Rate 11 10.2 10.0 10 9 8 7 65% 74% 6 of participants received ≤1 of participants receive
Slide 14: Intraocular Pressure Remained Stable in Both Ixo-vec Dose Groups
Intraocular Pressure Remained Stable in Year 2 in Both Ixo-vec Dose Groups Mean Intraocular Pressure (IOP) 30 Study eye (n=30) Fellow eye (n=30) 25 20 6E10 15 vg/eye 10 5 0 0 4 8 12 16 20 24 28 32 36 40 44 48 52 56 60 64 68 72 76 80 84 88 92 96 100 104 Weeks 30 Study eye (n=30) Fellow eye (n=30) 25
Slide 15: LUNA 2-year Safety Results
LUNA 2-year Safety Results Ixo-vec Phase 3 dose well tolerated, no new inflammation observed in year 2 * 6E10 vg/eye Participants Receiving Local Prophylaxis LUNA Year 1 LUNA Year 2 M15 M18 M21 M24 0 0 t 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 None ANTERIOR 0 0 0 0 0 0 0 0 0 0 0 t 0 0 0 0 0 0 0 0 0 6E10
Slide 16: Participants Preferred Ixo-vec to Prior Treatments
100% of Participants Receiving Phase 3 Dose + Steroid Eyedrops Alone * Preferred Ixo-vec to Prior Treatments * Was the steroid treatment Phase 3 Dose + Steroid Eyedrops you received easy to Would you prefer Ixo-vec Would you want Ixo-vec in Would you recommend Ixo- manage? over your prior your other
Slide 17: Key Findings from the LUNA Extension Study
Key Findings from the LUNA Extension Study Intravitreal Ixo-vec in a high need nAMD population: 2-year Results Potential Disease Modifying Activity Sustained vision preservation Durable anatomic control Both Doses Well Tolerated No Ixo-vec–related SAEs; no episcleritis, vasculitis, retinitis, choroi
Slide 18: Ixo-vec Phase 3 Neovascular AMD Study Designs
Ixo-vec Phase 3 Neovascular AMD Study Designs Population: Treatment-naïve and experienced adults with nAMD Key Inclusion BCVA: 35–78 letters Criteria Anti-VEGF responsive: After 2 aflibercept loading doses Phase 3 Trial (N=284) Difluprednate * Prophylaxis (27 wks) Enrollment Completed ✓ Week -8 -4 D
Slide 19: Ixo-vec Gene Therapy for nAMD: Clinical Progress from the LUNA Phase 2 Trial
FLORetina • December 2025 Ixo-vec Gene Therapy for nAMD: Clinical Progress from the LUNA Phase 2 Trial Charles C. Wykoff, M.D., Ph.D. Director of Research, Retina Consultants of Texas Professor of Clinical Ophthalmology, Blanton Eye Institute, Houston Methodist Hospital Disclosures: C= Consultant |