Adverum Biotechnologies announces FDA Regenerative Medicine Advanced Therapy designation granted for Ixo-vec for treatment of wet AMD

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Adverum Biotechnologies Inc announced the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for Ixo-vec, its clinical-stage gene therapy product candidate, for the treatment of wet AMD.

Laurent Fischer, MD, president and CEO of Adverum Biotechnologies, noted in a news release the designation is based on the clinical data from Ixo-vec to date and recognizes the potential of the company’s gene therapy for patients with wet AMD.

“This validates that Ixo-vec has the potential to address the significant unmet medical needs in treating nAMD,” Fischer said in the release. “With both RMAT and PRIME designations, we look forward to continuing our close collaborations with both the FDA and EMA on our pivotal program and to sharing updates from regulatory discussions in the fourth quarter of this year.”

According to the news release, the RMAT designation was established under the 21st Century Cures Act, and is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies.

A regenerative medicine therapy can qualify for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition.

Moreover, Adverum noted in its news release the RMAT designation gives companies the benefits of intensive FDA guidance on efficient drug development, including potential priority review of the biologics license application (BLA), and other opportunities to expedite development and review.

Adverum noted in the news release it 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.¹

Unlike other ophthalmic gene therapies that require surgery to administer the gene therapy under the retina, 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.

According to the release, Ixo-vec is currently being evaluated in the ongoing Phase 2 clinical trial LUNA (NCT05536973) and the Phase 1 OPTIC extension study (NCT04645212) for the treatment of neovascular or wet AMD.

Recognizing the need for new treatment options for wet AMD, the FDA granted Fast Track and RMAT designations for Ixo-vec for the treatment of wet AMD. Ixo-vec has also received PRIME designation from the EMA and the Innovation Passport from the United Kingdom’s Medicines and Healthcare products Regulatory Agency for the treatment of wet AMD.¹

At the recent ASRS annual meeting in Stockholm, Adverum announced results from the landmark 26-week interim analysis of the ongoing LUNA Phase 2 trial of Ixo-vec.

During the meeting, Fischer noted in a separate news release the company’s pre-specified Patient Preference Survey, presented at ASRS for the first time, supports that patients want to preserve long-term vision and be free of injections and are not deterred by corticosteroid prophylaxis.²

“Over the next couple quarters, we plan to present data at important 9-month and 1-year timepoints. In OPTIC, nearly all patients who were injection free at 1 year continued to be injection free beyond 3 years,” Fischer said.

According to the company, the LUNA trial builds on its experience with the OPTIC study, for which landmark 2-year data was published in The Lancet’s eclinicalmedicine and landmark 3-year data was presented at the American Academy of Ophthalmology 2023 Annual Meeting.

Upcoming milestones for the LUNA trial include a continued FDA and EMA formal and informal regulatory interactions in the second half of 2024; presentation of landmark LUNA 9-month analysis during Q4 of 2024; Phase 3 pivotal trial design update during Q4; presentation of landmark LUNA 52-week analysis in Q1 of 2025; and a planned initiation of the Phase 3 trial during the first half of 2025.

References:

1. Inc AB. Adverum Biotechnologies Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted for Ixo-vec for the Treatment of Wet AMD. GlobeNewswire News Room. Published August 1, 2024. Accessed August 2, 2024. https://www.globenewswire.com/news-release/2024/08/01/2922728/32452/en/Adverum-Biotechnologies-Announces-FDA-Regenerative-Medicine-Advanced-Therapy-RMAT-Designation-Granted-for-Ixo-vec-for-the-Treatment-of-Wet-AMD.html

2. Inc AB. Adverum Biotechnologies Presents Positive Ixo-vec Clinical Data from the 26-Week Interim Analysis of the LUNA Phase 2 Trial at the 2024 ASRS Annual Meeting. GlobeNewswire News Room. Published July 17, 2024. Accessed July 17, 2024. https://www.globenewswire.com/news-release/2024/07/17/2914283/0/en/Adverum-Biotechnologies-Presents-Positive-Ixo-vec-Clinical-Data-from-the-26-Week-Interim-Analysis-of-the-LUNA-Phase-2-Trial-at-the-2024-ASRS-Annual-Meeting.html