Adverum Biotechnologies Provides ADVM-022 Development Update and Anticipated Corporate Milestones for 2022

– Finalizing Two-Dose Study Design for Phase 2 Trial of ADVM-022 in Wet AMD with Intention to Dose First Patient in Q32022 – – Anticipates Near-term Hires to Further Strengthen Executive Team – – Board of Directors is Actively Pursuing the Addition of Two New Directors – – Expects Cash Position to Fund Development Plans and Operations into 2024 – REDWOOD CITY, Calif., Dec. 21, 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 the completion of a review of its ADVM-022 program, outlined plans for the next stage of development in wet age-related macular degeneration (wet AMD) and provided anticipated key milestones for 2022. Dr. Laurent Fischer, M.D., president and chief executive officer at Adverum Biotechnologies, stated: “We are pleased to conclude a thorough review of the totality of data generated in all 55 patients treated with ADVM-022, in wet AMD and diabetic macular edema, some of whom have been followed for over 2.5 years. The data analysis confirms our strategy to advance development of ADVM-022 in wet AMD. We intend to conduct a Phase 2 trial of ADVM-022 in wet AMD at 2 X 10^11 vg/eye dose and a new lower 6 X 10^10 vg/eye dose, with three new enhanced steroid prophylaxis regimens including topical, intravitreal and a combination of systemic and local steroids. Based on learnings from the OPTIC and INFINITY trials and extensive preclinical datasets, as well as the input and support of experts and members of our Scientific Advisory Board, we are confident in the plans we are sharing today to safely develop ADVM-022 in wet AMD, realizing the promise and full potential of our innovative gene therapy.” Dr. Fischer continued, “I believe we have the right plan in place to establish the benefits and safety of ADVM-022 and progress our development pipeline. We have focused our capital on our development strategy prudently, with adequate financial resources in hand to complete the planned Phase 2 study, and I very much look forward to providing an update on our progress in 2022.” Future Development of ADVM-022 for Wet AMD: Phase 2 Trial Design . Subject to regulatory review, Adverum is finalizing the design for a Phase 2 trial of ADVM-022 in wet AMD investigating the 2 X 10^11 (2E11) vg/eye dose and a lower 6 X 10^10 vg/eye (6E10) vg/eye dose, with three new enhanced steroid prophylaxis regimens, which the company anticipates will include a topical, intravitreal (IVT) and a combination of systemic and local steroids. The trial is expected to enroll approximately 72 patients and evaluate similar endpoints to the Phase 1 OPTIC trial in wet AMD. This study design is based on extensive analysis conducted by the company’s clinical development team and incorporates feedback from Adverum’s scientific advisory board and key retina, uveitis and gene therapy experts. IND Amendment . Adverum intends to file an investigational new drug (IND) amendment in the first half of 2022 and engage with the FDA with the aim of securing agreement on the Phase 2 trial design. The company anticipates completing this process by mid-2022. Phase 2 Initiation . Adverum is beginning preparations for the Phase 2 trial of ADVM-022 in wet AMD, with the intention of dosing the first patient in Q32022. Early feedback from potential Phase 2 investigators suggests completion of trial enrollment by year end 2022. Fully Resourced . Adverum reiterates its expectation that its cash position is sufficient to fund the company’s operations, including its development plans for ADVM-022, into 2024. “The long-term efficacy seen in the OPTIC trial after a single injection of ADVM-022 has been remarkable for my patients, who required frequent injections prior to entering the trial. The long-term safety data supports using the lower 2E11 vg/eye dose and a new lower 6E10 vg/eye dose with an enhanced steroid prophylaxis regimen. I look forward to evaluating ADVM-022’s potential to provide a safe and durable gene therapy from a single, in-office injection in patients with wet AMD in the upcoming Phase 2 trial,” said Arshad M. Khanani M.D., M.A., managing partner and director of clinical research, Sierra Eye Associates, member of Adverum’s Scientific Advisory Board, and the highest enrolling investigator in the wet AMD Phase 1 OPTIC trial. Review of ADVM-022 Program: During the second half of 2021, Adverum undertook a comprehensive review of the company’s ADVM-022 program, including analysis of its preclinical and clinical data. The review solidified the following rationale underpinning the development strategy and Phase 2 study design outlined today for ADVM-022 in wet AMD: Phase 1 Clinical Outcomes of ADVM-022 in Wet AMD: In October 2021, Adverum presented two-year follow-up data from its Phase 1 OPTIC study in wet AMD patients at the Retina Society’s 54 th Annual Scientific Meeting. The data presented demonstrated a greater than 80% reduction in annualized anti-VEGF injections following the 2E11 vg/eye ADVM-022 IVT injection that is expected to be validated in the Phase 2 study. ADVM-022 in wet AMD has been well tolerated and ocular inflammation was minimal and responsive to steroid eye drops. At latest visit, all 2E11 vg/eye patients were inflammation-free and none required any steroid eye drops to treat inflammation. Underlying Pathology and Patient Characteristics: The INFINITY data in diabetic macular edema (DME) indicate that poorly controlled diabetes with presence of microvascular and macrovascular diabetic complications are important criteria in the identification of an appropriate patient population for ADVM-022, and these criteria have been considered in the proposed design of the Phase 2 trial in wet AMD.

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