Adverum Biotechnologies Presents Long-term Data through March 10, 2021 from the OPTIC Trial of ADVM-022 Intravitreal Gene Therapy in Treatment-experienced Wet AMD Patients at ARVO 2021

— Long-term durability and maintained efficacy; sustained robust aflibercept protein expression observed — — 60% of patients injection free beyond 1 year following 2 x 10^11 single dose — REDWOOD CITY, Calif., May 01, 2021 (GLOBE NEWSWIRE) — Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced new long-term data from the OPTIC clinical trial of ADVM-022 single intravitreal (IVT) injection gene therapy in patients requiring frequent anti-VEGF injections for their neovascular or wet age-related macular degeneration (wet AMD). Safety and efficacy data from patients followed for a median of 88 and 68 weeks at the 2 x 10^11 vg/eye dose (for Cohorts 2 & 3, respectively) and 104 and 36 weeks at the 6 x 10^11 vg/eye dose (for Cohorts 1 & 4, respectively) are being presented at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Virtual Meeting with a pre-recorded presentation uploaded on April 16, 2021. “The long-term OPTIC data show the potential for ADVM-022 to offer disease modifying treatment for patients with wet AMD,” said Laurent Fischer, M.D., chief executive officer of Adverum Biotechnologies . “Patient safety is our absolute priority and following the unexpected adverse event we reported this past week in a patient treated with the 6e11 high dose in the INFINITY study in diabetic patients with macular edema, we are unmasking the INFINITY study in order to analyze all data available and monitor every patient who has received our gene therapy. We are also working closely with our data monitoring committee and scientific advisors and conducting a thorough review of all the data from our ADVM-022 program. We will report our findings as the analysis progresses to inform next steps for development.” Dr. Fischer continued, “In OPTIC, ADVM-022 with wet AMD has demonstrated durability out to two years with the ease of a single, in-office intravitreal injection. We believe that we are well within the therapeutic window with the 2e11 dose with 60% of patients supplemental injection free beyond one year. Additionally, the aflibercept protein levels at the 2e11 dose were within the modeled therapeutic range and sustained out to at least one year, consistent with levels observed 4-6 weeks after an aflibercept injection.” The data reported in this press release and the related statements relate only to the OPTIC clinical trial evaluating ADVM-022 gene therapy for the treatment of wet AMD. Adverum reported new interim data from the OPTIC trial (March 10, 2021 cutoff date, n=30) that continue to demonstrate the potential of ADVM-022 to greatly reduce the anti-VEGF injection burden for patients with wet AMD: All ADVM-022-related ocular adverse events (AE) were mild (80%) to moderate (20%) in OPTIC patients with wet AMD. No clinical or fluorescein evidence of posterior inflammation No vasculitis, retinitis, choroiditis, vascular occlusions, or endophthalmitis Inflammation when observed was mild and responsive to steroid eye drops At 2 x 10^11 vg/eye dose, ocular inflammation was minimal and responsive to steroid eye drops; 87% of patients (13/15 1 ) have discontinued steroid eye drops Cohort 3 Safety Data for 2 x 10^11 vg/eye Dose: A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/b6f22004-1612-4316-a481-9ac63157b54f A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/cbe15798-cd87-444f-b81a-f989dd73fc4a A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/7e3ef263-ebd4-4a27-a783-b41515ed8bdd Durable expression of aflibercept following a single, in-office IVT injection of ADVM-022, for both doses (2 x 10^11 vg/eye and 6 x 10^11 vg/eye) Maintained or gained vision (mean BCVA 2 ) Maintained to improved retinal anatomy (mean CRT 3 ) Majority of patients are supplemental anti-VEGF injection free: 60% of patients (9/15 1 ) injection free following 2 x 10^11 vg/eye dose beyond one year 73% of patients (11/15 1 ) required zero or one injection following 2 x 10^11 vg/eye at one-year 87% of patients (13/15 4 ) injection free following 6 x 10^11 vg/eye dose A photo accompanying this announcement is available at: https://www.globenewswire.com/NewsRoom/AttachmentNg/f20337ce-b5a4-45de-8895-404bcd79b843 Substantial reduction in annualized anti-VEGF injection frequency 5 following ADVM-022 in patients who previously required frequent injections: 85% reduction for 2 x 10^11 vg/eye 96% reduction for 6 x 10^11 vg/eye Robust sustained aflibercept expression levels within therapeutic range was observed for both doses and reaching the top of the dose response curve. A photo accompanying this announcement is available at: https://www.globenewswire.com/NewsRoom/AttachmentNg/e83dc5f6-25da-4922-96f3-49e30b1c7811 OPTIC Clinical Trial Data: Results Following a Single ADVM-022 Dose: Cohort 1 Cohort 2 Cohort 3 Cohort 4 Patients n=6 n=6 n=9 n=9 Median (Range) Follow-up Visit (Weeks) 104 (All) 88 (64 6 to 92) 68 (48 to 72) 36 (32 to 44) ADVM-022 Dose High dose 6 x 10^11 vg/eye Low dose 2 x 10^11 vg/eye Low dose 2 x 10^11 vg/eye High dose 6 x 10^11 vg/eye Prophylactic Steroid Regimen 13-day oral 13-day oral 6-week eye drops 6-week eye drops

The release as fetched from its publisher. adverum.com ↗