Adverum Presents Data at ASGCT Further Supporting Phase 2 Development Plans for ADVM-022

Rhea-AI Summary

Adverum Biotechnologies announced promising results from its ADVM-022 program for treating wet AMD. Data show that the human equivalent dose of 6E10 vg/eye achieved therapeutic levels of aflibercept in non-human primates (NHPs), supporting plans for a Phase 2 trial in Q3 2022 with doses of 2E11 vg/eye and 6E10 vg/eye. The OPTIC trial indicated an 81% to 94% reduction in annual anti-VEGF injections, depending on neutralizing antibody levels, with no adverse clinical signs noted. These findings pave the way for potential long-term efficacy against wet AMD.

Positive

• Therapeutic levels of aflibercept achieved with 6E10 vg/eye dose in NHP studies.
• Phase 2 clinical trial planned in Q3 2022 for further evaluation.
• Significant reductions in annualized anti-VEGF injections (81% to 94%) observed in the OPTIC trial.

Negative

• None.

- Human equivalent ADVM-022 dose of 6E10 vg/eye provided therapeutic levels of aflibercept in NHPs

- Oral encore presentation to highlight ADVM-022 reductions in mean annualized anti-VEGF injections ranged from 81% in all OPTIC wet AMD subjects to 94% in those with neutralizing antibody titers <1:125 at 2 X 10^11 vg/eye

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, today will announce new data from the ADVM-022 development program in wet age-related macular degeneration (wet AMD). The data being presented during the American Society of Gene and Cell Therapy (ASGCT) 2022 Annual Meeting in Washington, D.C. and virtually, shows results of the non-human primate (NHP) studies that provide support for the new 6 X 10^10 vg/eye (6E10) dose that Adverum intends to include in its next clinical trial.

“These new data suggest the 6x10^10 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, M.D., president and chief executive officer at Adverum Biotechnologies. “We plan to initiate the Phase 2 study in wet AMD in the third quarter of 2022 evaluating ADVM-022 at the 2x10^11 vg/eye dose and the new, lower 6x10^10 vg/eye dose with new enhanced prophylactic steroid 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 humor 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 highly encouraged by the aflibercept expression levels and tolerability of the human equivalent dose of 6x10^10 vg/eye supported by our data in non-human primates,” said Richard Beckman, M.D., chief medical officer at Adverum Biotechnologies. “As we explore doses of 2x10^11 vg/eye and lower, we are excited by the potential of enhancing 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 Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting, assessing the potential impact of baseline levels of neutralizing antibodies (NAbs) on efficacy and safety outcomes with ADVM-022 (AAV.7m8-aflibercept). An encore of these data will be featured in an oral presentation on May 19, 2022 at ASGCT by Szilárd Kiss, M.D., Bob and Dolores Hope – Robert M. Ellsworth, M.D. Distinguished Associate Professor in Ophthalmology and a member of Adverum’s Scientific Advisory Board. In all subjects, a single intravitreal injection gene therapy at the 2E11 dose demonstrated a greater than 80% reduction in annualized anti-vascular endothelial growth factor (VEGF) injections. In subjects with baseline NAbs <1:125, the reductions in mean annualized injection frequency increased to 94%. Over 80% of patients screened for the OPTIC trial had baseline NAbs titers of <1:125. There was no correlation between baseline NAbs and safety events.

The ADVM-022 poster and oral presentations will be made available on the Publications page in the Pipeline section of Adverum’s 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 patients living with AMD. Wet AMD is a leading cause of blindness in patients over 65 years of age, with a prevalence of approximately 20 million individuals worldwide living with this condition. The incidence of new cases of wet AMD is 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.

About OPTIC Trial of ADVM-022 in Wet AMD

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

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

About Adverum Biotechnologies

Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of patients with neovascular or wet age-related macular degeneration. 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 Adverum’s plans to initiate a Phase 2 study in wet AMD in the third quarter of 2022 evaluating ADVM-022 at the 2 X 10^11 vg/eye dose and a new, lower 6 X 10^10 vg/eye dose. 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; 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 ADVM-022. 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 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 such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Inquiries

Anand Reddi
Vice President, Head of Corporate Strategy and External Affairs & Engagement
Adverum Biotechnologies, Inc.
T: 650-649-1358

Investors

Laurence Watts
Gilmartin Group
T: 619-916-7620
E: laurence@gilmartinir.com

Media

Megan Talon
Associate Director, Corporate Communications
Adverum Biotechnologies, Inc.
T: 650-649-1006
E: mtalon@adverum.com

FAQ

What are the key results from Adverum's ADVM-022 program for wet AMD?

Adverum's ADVM-022 program demonstrated therapeutic levels of aflibercept with a 6E10 vg/eye dose in non-human primates, leading to significant reductions in annualized anti-VEGF injections in the OPTIC trial.

When is the Phase 2 trial for ADVM-022 expected to start?

The Phase 2 trial for ADVM-022 in wet AMD is planned to initiate in the third quarter of 2022.

What reductions in anti-VEGF injections were observed in the OPTIC trial?

The OPTIC trial observed a reduction of 81% in anti-VEGF injections for all subjects, increasing to 94% for those with neutralizing antibody titers <1:125.

What is the significance of the 6E10 vg/eye dose in the study?

The 6E10 vg/eye dose is significant as it provided therapeutic levels of aflibercept and is being considered for future clinical applications.