Adverum Biotechnologies, Inc.

– Equivalent human ADVM-022 dose of 6E10 vg/eye provided therapeutic levels of aflibercept in NHPs

– Oral booster presentation to highlight ADVM-022 reductions in mean annualized anti-VEGF injections ranging from 81% in all wet OPTIC subjects with AMD to 94% in those with neutralizing antibody titers

REDWOOD CITY, Calif., May 17, 2022 (GLOBE NEWSWIRE) — Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, will announce today new data from the ADVM-022 development program in wet age-related macular degeneration (wet AMD). Data presented at the 2022 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Washington, D.C. and virtually, show findings from non-human primate (NHP) studies that support the new 6 X 10^ 10 vg/eye (6E10) which Adverum intends to include in its next clinical trial.

“These new data suggest that the 6×10^10 vg/eye dose may provide therapeutic levels of aflibercept in the clinic and informs our dosing regimen in our upcoming Phase 2 trial,” said Laurent Fischer, MD, President and CEO of the management of Adverum Biotechnologies. “We plan to initiate the Phase 2 study in wet AMD in the third quarter of 2022 evaluating ADVM-022 at the dose of 2×10^11 vg/eye and the new lower dose of 6×10^10 vg/eye with new and improved steroid prophylaxis regimens. ”

Data Highlights

  • The human equivalent dose of 6E10 (3 X 10^10 vg/eye dose in NHP) demonstrated potential therapeutic levels in both aqueous and vitreous humor.

  • Administration of the human equivalent dose of 6E10 (3 x 10^10 vg/eye dose in NHP) was well tolerated. No adverse clinical signs were observed during the three-month study.

“We are very encouraged by the expression levels of aflibercept and the tolerability of the human equivalent dose of 6×10^10 vg/eye, supported by our data in non-human primates,” said Richard Beckman, MD, physician. -Head at Adverum Biotechnologies. “As we explore doses of 2×10^11 vg/eye and below, we are excited about the potential for improving the safety profile while building on the robust response that ADVM-022 has demonstrated to date in wet AMD.”

Earlier this month, Adverum reported data from the OPTIC trial at the 2022 Association for Research in Vision and Ophthalmology (ARVO) annual meeting, assessing the potential impact of baseline levels of neutralizing antibodies (NAbs) on efficacy and safety results with ADVM-022 (AAV .7m8-aflibercept). A reminder of these data will be presented in an oral presentation on May 19, 2022 at ASGCT by Szilárd Kiss, MD, Bob and Dolores Hope – Robert M. Ellsworth, MD Distinguished Associate Professor in Ophthalmology and member of the Scientific Advisory Board of Adverum . In all subjects, gene therapy by single intravitreal injection at a dose of 2E11 demonstrated a greater than 80% reduction in annualized injections of anti-vascular endothelial growth factor (VEGF). In subjects with baseline NAbs

The ADVM-022 poster and oral presentations will be available on the Publications page of the Pipeline section of the Adverum website.

About Wet Age-Related Macular Degeneration

Wet AMD, also known as neovascular AMD or nAMD, is an advanced form of AMD, affecting approximately 10% of AMD patients. Wet AMD is a leading cause of blindness in patients over the age of 65, with a prevalence of approximately 20 million people worldwide living with the disease. The incidence of new cases of wet AMD is expected to increase significantly worldwide as populations age. AMD is expected to affect 288 million people worldwide by 2040, with wet AMD accounting for around 10% of these cases.

About the OPTIC trial of ADVM-022 in wet AMD

ADVM-022 is Adverum’s clinical-stage gene therapy product candidate under development for the treatment of wet AMD. ADVM-022 uses a proprietary vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. Unlike other ophthalmic gene therapies that require surgery to deliver the gene therapy below the retina (subretinal approach), ADVM-022 has the advantage of being delivered as a single in-office IVT injection and is designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance and improve visual outcomes for patients with wet AMD.

The OPTIC trial is designed as a multicenter, open-label, dosing, safety, and efficacy trial of ADVM-022 in patients with wet AMD who demonstrated responsiveness to anti-VEGF therapy. OPTIC patients are experienced in treatment and previously required frequent injections of anti-VEGF to manage their wet AMD and maintain functional vision.

About Adverum Biotechnologies

Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious and rare eye diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a single intravitreal injection for the treatment of patients with neovascular or wet age-related macular degeneration. For more information, visit www.adverum.com.

Forward-looking statements

Statements in this press release regarding events or results that may occur in the future are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to , statements regarding Adverum’s plans for a Phase 2 study in wet AMD in Q3 2022 evaluating ADVM-022 at a dose of 2 X 10^11 vg/eye and a new lower dose of 6 X 10 ^ 10 yd/eye. Actual results could differ materially from those anticipated in these forward-looking statements due to various risks and uncertainties, including but not limited to: Adverum’s new technology, which makes it difficult to predict the timing of onset and the end of clinical trials; regulatory uncertainties; the results of early clinical trials are not always predictive of future clinical trials and outcomes; and the potential for future complications or side effects related to the use of ADVM-022. Additional risks and uncertainties facing Adverum are set forth under “Risk Factors” and elsewhere in Adverum’s filings and reports with the Securities and Exchange Commission (SEC), including Adverum’s Quarterly Report on Form 10-Q for the three months ended March 31, 2022 filed with the SEC on May 12, 2022. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update these statements to reflect events that occur or circumstances that exist after the date on which they were made.

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