Adverum Biotechnologies Announces 3-Year Efficacy and Safety Results from the OPTIC Extension Study in Patients with Wet AMD at AAO 2023

- Patients in OPTIC continue to experience long-term benefit from Ixo-vec through 3 years of follow-up, including maintenance of vision, durability of anatomical improvements and sustained reduction in anti-VEGF treatment burden -

- 84% reduction in annualized anti-VEGF injections, with 53% of the participants at the 2E11 dose entirely free of injections at year 3 -

- Durable aflibercept protein levels up to 4.5 years after a single Ixo-vec IVT injection -

- Ixo-vec continues to be generally well tolerated -

- Previously announced aflibercept protein levels from the LUNA study were also presented -

REDWOOD CITY, Calif., Nov. 04, 2023 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today announced updated data from the OPTIC extension study of patients with wet AMD during the Retina Subspecialty Day at the American Academy of Ophthalmology (AAO) 2023 Annual Meeting in San Francisco, California. A summary of previously announced aflibercept protein levels from the LUNA study was also presented.

“We are pleased to be leading innovation for patients with wet AMD through the development of a treatment option that has the potential to be safe, efficacious and much less burdensome for patients,” stated Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies. “These efforts are now supported by the most mature dataset in wet AMD IVT gene therapy, with the OPTIC safety and efficacy data now sustained out to 3 years. Ixo-vec demonstrated a robust improvement in outcome measures while maintaining a favorable safety profile, particularly at the 2E11 dose, which we have advanced to our Phase 2 LUNA trial. We are particularly encouraged by the continuous and consistent aflibercept protein levels observed at up to 4.5 years post-treatment. These data highlight the potential for patients to experience life-long clinical benefit after a single IVT treatment with minimal or no need for additional injections and showcase the potentially transformative impact of gene therapy.”

OPTIC 3-year Extension Data Highlights

• An oral presentation titled “ADVM-022 Intravitreal Gene Therapy for Neovascular AMD: Preliminary Data from the Phase 2 LUNA Trial and 3-Year Results From the Phase 1 OPTIC-Extension Trial” was presented today by Carl D. Regillo, M.D. FACS at the AAO 2023 Annual Meeting.
• Patients in the OPTIC extension trial continue to experience long-term benefit from Ixo-vec through 3 years of follow-up, including maintenance of vision, durability of anatomical improvements and sustained reduction in anti-VEGF treatment burden.
• Patients at the 2E11 dose had an 84% reduction in annualized anti-VEGF injections, with 53% of the participants at the 2E11 dose receiving no supplemental injections through three years.
• Aflibercept protein levels have been sustained through follow-up, which is up to 4.5 years post-treatment.
• BCVA was maintained and CST was improved through 3 years.
• Ixo-vec was generally well tolerated with dose-dependent inflammation that was responsive to topical steroids.

LUNA Aflibercept Protein Data Highlights, Baseline Characteristics and Clinical Program Milestones

• Aflibercept protein data suggest Ixo-vec at the 2E11 and 6E10 doses deliver similar levels of aflibercept that are both within the therapeutically active range based on OPTIC and non-human primate data.
• The LUNA trial population has comparable injection frequency and other baseline characteristics, with slightly better visual acuity and fluid control, compared to the study population of OPTIC.
• In particular, the mean annualized anti-VEGF injection rate in the 12 months prior to Ixo-vec treatment in LUNA is 9.9 injections, indicating that these subjects require frequent injections, administered approximately every six weeks.
• Clinical Program Milestones:
  
  • Q4 2023 - LUNA preliminary efficacy and safety data.
  • Mid-2024 - Additional LUNA data, including the 26-week interim analysis.

“The long-term follow-up data from the OPTIC trial continue to affirm that Ixo-vec offers a potentially transformational treatment for wet AMD,” commented Carl Regillo, M.D., F.A.C.S., Chief of the Retina Service at Wills Eye Hospital and presenter of the data at the AAO Annual Meeting. “A favorable benefit-risk profile resulting in an 84% reduction in annualized anti-VEGF injections was demonstrated in participants receiving the 2E11 dose, with 53% of patients continuing to be free from supplemental injections at 3 years after a single IVT injection. Taken together, the efficacy and safety at the 2E11 dose in the OPTIC trial underscore Ixo-vec's potential as a promising treatment option for wet AMD patients addressing real-world unmet needs. I look forward to learning more from the ongoing Phase 2 LUNA trial.”

About Wet Age-Related Macular Degeneration

Wet AMD, also known as neovascular AMD or nAMD, is a VEGF driven advanced form of AMD affecting approximately 10% of patients living with 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 2 to 3 years. The current standard of care requires frequent life-long repeated bolus injections of anti-VEGF in the eye. 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 macular fluid.

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-vascular endothelial growth factor (VEGF) injections, optimize patient compliance and improve vision outcomes for patients with wet AMD. In recognition of the need for new treatment options for wet AMD, the U.S. Food and Drug Administration granted Fast Track designation 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 potential benefits of Ixo-vec in the treatment of wet AMD, the design of and enrollment in the LUNA trial, including the prophylactic corticosteroid regimens, and anticipated preliminary and interim data from the LUNA trial. 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; and the potential for future complications or side effects in connection with use of Ixo-vec. 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 Quarterly Report on Form 10-Q for the quarter ended June 30, 2023 filed with the SEC on August 10, 2023. 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.

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