Corporate Highlights

• Appointed Dr. Rabia Gurses Ozden as Chief Medical Officer and Dr. Szilárd Kiss as a Member of the Board of Directors:
  

In June 2024, we announced the appointment of Dr. Rabia Gurses Ozden to the Company’s executive leadership team as chief medical officer and of Dr. Szilárd Kiss to the Company’s Board of Directors. In connection with her appointment as chief medical officer, Dr. Ozden stepped down from the Company’s Board of Directors, with Dr. Kiss filling the resulting vacancy.

Upcoming Anticipated Milestones

• 2H 2024: Continued FDA and EMA formal and informal regulatory interactions

• 4Q 2024: Clinical data from the landmark LUNA 9-month analysis

• 4Q 2024: Phase 3 pivotal trial design update

• 1Q 2025: Clinical data from the landmark LUNA 52-week analysis

• H1 2025: Planned initiation of Phase 3 trial
  

Financial Results for the Three Months Ended June 30, 2024

• Cash, cash equivalents and short-term investments were $173.8 million as of June 30, 2024, compared to $96.5 million as of December 31, 2023. Adverum expects its cash, cash equivalents and short-term investments to fund operations into late 2025.

• Research and development expenses were $17.1 million for the three months ended June 30, 2024, compared to $20.6 million for the same period in 2023. Research and development expenses decreased due to lower facilities related expenses and lower compensation expenses. Stock-based compensation expense included in research and development expenses was $1.1 million for the second quarter of 2024.

• General and administrative expenses were $3.8 million for the three months ended June 30, 2024, compared to $12.5 million for the same period in 2023. General and administrative expenses decreased due to a cumulative catch-up adjustment of sublease income, lower facilities related expense, lower depreciation expense, and lower insurance expense driven by lower premiums. Decreases were partially offset by increased compensation expense and professional services expense. Stock-based compensation expense included in general and administrative expenses was $2.9 million for the second quarter of 2024.

• Net loss was $18.5 million, or $0.89 per basic and diluted share, for the three months ended June 30, 2024, compared to $31.5 million, or $3.13 per basic and diluted share for the same period in 2023.

About Wet Age-Related Macular Degeneration

Wet AMD, also known as neovascular AMD or nAMD, is a VEGF-driven advanced form of age-related macular degeneration (AMD) associated with the build-up of fluid in the macula and the retina. Wet AMD is a leading cause of blindness in people over 65 years of age, with approximately 20 million individuals worldwide living with this condition. New cases of wet AMD are expected to grow significantly worldwide as populations age. AMD is expected to impact 288 million people worldwide by 2040, with wet AMD accounting for approximately 10% of those cases. Additionally, wet AMD is a bilateral disease, and incidence of nAMD in the second eye is up to 42% in the first two to three years. The current standard of care requires frequent life-long bolus injections of anti-VEGF in the eye. Intravitreal (IVT) gene therapy has the promise to preserve vision and reduce most or all injections for the life of the patient by delivering stable therapeutic levels of anti-VEGF to control fluid associated with the disease.

About Ixo-vec in Wet AMD

Adverum is developing ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), its clinical-stage gene therapy product candidate, for the treatment of wet AMD. Ixo-vec utilizes 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 administer the gene therapy under the retina (sub-retinal approach), Ixo-vec is designed to be administered as a one-time IVT injection in the physician’s office, deliver long-term efficacy, reduce the burden of frequent anti-VEGF injections into the eye, optimize patient compliance and improve vision outcomes for patients with wet AMD. Ixo-vec is currently being evaluated for the treatment of neovascular or wet AMD in the ongoing LUNA Phase 2 clinical trial (NCT05536973) and the OPTIC Phase 1 extension study (NCT04645212). In recognition of the need for new treatment options for wet AMD, FDA has granted Fast Track and RMAT designations for Ixo-vec for the treatment of wet AMD. Ixo-vec has also received PRIME designation from the European Medicines Agency and the Innovation Passport from the United Kingdom’s Medicines and Healthcare products Regulatory Agency for the treatment of wet AMD.

About Adverum Biotechnologies
Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the aspiration of developing functional cures to restore vision and prevent blindness. Leveraging the capabilities of its proprietary intravitreal (IVT) platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians’ offices, to eliminate the need for frequent ocular injections to treat these diseases. Adverum is evaluating its novel gene therapy candidate, ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), as a one-time, IVT injection for patients with neovascular or wet age-related macular degeneration. Additionally, by overcoming the challenges associated with current treatment paradigms for debilitating ocular diseases, Adverum aspires to transform the standard of care, preserve vision, and create a profound societal impact around the globe. For more information, please visit www.adverum.com.

Forward-looking Statements
Statements contained 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 the favorable safety profile and potential best-in-class product profile of Ixo-vec, anticipated timing of interim data and trial design update for the Phase 2 LUNA trial and initiation of a Phase 3 trial, and the Company’s cash sufficiency and runway. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including risks inherent to, without limitation: Adverum’s novel technology, which makes it difficult to predict the timing of commencement and completion of clinical trials; regulatory uncertainties; enrollment uncertainties; the results of early clinical trials not always being predictive of future clinical trials and results; the potential for future complications or side effects in connection with use of Ixo-vec; and risks associated with market conditions. Additional risks and uncertainties facing Adverum are set forth under the caption “Risk Factors” and elsewhere in Adverum’s Securities and Exchange Commission (SEC) filings and reports, including Adverum’s most recent Annual Report on Form 10-K filed with the SEC, as updated by any subsequent reports on Form 10-Q. 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 such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Corporate, Investor and Media Inquiries
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^{(1) Derived from Adverum's annual audited consolidated financial statements.}