Adverum Biotechnologies Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted for Ixo-vec for the Treatment of Wet AMD

Rhea-AI Summary

Adverum Biotechnologies (Nasdaq: ADVM) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for Ixo-vec, its gene therapy candidate for treating wet AMD. This designation, along with the previously granted PRIME designation, recognizes Ixo-vec's potential to address unmet medical needs in nAMD treatment. The RMAT program, established under the 21st Century Cures Act, aims to expedite development and review of promising therapies for serious conditions.

RMAT designation offers benefits such as intensive FDA guidance on efficient drug development and potential priority review of the biologics license application (BLA). Adverum plans to share updates from regulatory discussions in Q4 2024.

Positive

• FDA granted RMAT designation for Ixo-vec, potentially expediting development and review
• Ixo-vec has both RMAT and PRIME designations, indicating strong regulatory support
• RMAT designation may lead to priority review of the biologics license application (BLA)

Negative

• None.

Insights

Biotechnology Analyst

The FDA's granting of Regenerative Medicine Advanced Therapy (RMAT) designation for Adverum's Ixo-vec is a significant milestone in the development of gene therapies for wet Age-related Macular Degeneration (AMD). This designation underscores the potential of Ixo-vec to address unmet medical needs in treating this serious eye condition.

Key implications of this news:

• Accelerated Development: RMAT designation provides Adverum with enhanced access to FDA guidance, potentially expediting the development and review process for Ixo-vec.
• Validation of Clinical Data: The designation is based on preliminary clinical evidence, suggesting that Ixo-vec has shown promising results in early trials.
• Market Potential: Wet AMD is a highly prevalent ocular disease, representing a substantial market opportunity if Ixo-vec proves successful in later-stage trials.
• Competitive Advantage: This designation, coupled with the previously obtained PRIME designation from the EMA, positions Adverum favorably in the competitive landscape of AMD treatments.

However, investors should note that while RMAT designation is encouraging, it does not guarantee ultimate approval or commercial success. The company still needs to successfully complete pivotal trials and navigate the regulatory approval process. The upcoming regulatory discussions in Q4 2024 will be important in determining the path forward for Ixo-vec.

Ophthalmology Specialist

The RMAT designation for Ixo-vec represents a potentially significant advancement in the treatment of wet AMD, a leading cause of vision loss in older adults. Current standard treatments for wet AMD typically involve frequent intravitreal injections, which can be burdensome for patients and healthcare systems.

Ixo-vec, as a gene therapy, aims to provide a longer-lasting treatment option. If successful, it could revolutionize wet AMD management by:

• Reducing Treatment Burden: Potentially decreasing the frequency of interventions needed to control the disease.
• Improving Compliance: Less frequent treatments could lead to better patient adherence and outcomes.
• Addressing Unmet Needs: Current treatments don't work for all patients and some experience diminishing efficacy over time. A gene therapy approach could potentially offer a more durable solution.

However, it's important to temper enthusiasm with caution. Gene therapies for ocular diseases are still in their infancy and long-term safety and efficacy data will be critical. The ophthalmology community will be closely watching the results of Adverum's pivotal trials to assess Ixo-vec's potential to become a new standard of care for wet AMD.

Regulatory Affairs Expert

The FDA's decision to grant RMAT designation to Ixo-vec is a significant regulatory milestone for Adverum Biotechnologies. This designation, along with the EMA's PRIME designation, offers several advantages in the regulatory pathway:

• Expedited Review: RMAT designation may lead to a priority review of the eventual Biologics License Application (BLA), potentially shortening the approval timeline.
• Enhanced Communication: Adverum will benefit from increased opportunities for consultation with the FDA, allowing for more efficient trial design and development strategies.
• Flexibility in Evidence Generation: The FDA may consider a more flexible approach to the evidence required for approval, potentially including the use of real-world evidence or other novel clinical trial designs.

However, it's important to note that RMAT designation does not guarantee approval. Adverum must still demonstrate safety and efficacy in well-designed clinical trials. The upcoming regulatory discussions in Q4 2024 will be important in determining the specific requirements for pivotal trials and potential approval pathways.

From a regulatory perspective, Adverum's dual RMAT and PRIME designations position Ixo-vec favorably for expedited development in both the U.S. and EU markets. This could potentially lead to a more streamlined global development strategy, which is particularly valuable in the competitive field of AMD treatments.

REDWOOD CITY, Calif., Aug. 01, 2024 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company pioneering the use of gene therapy as a new standard of care for highly prevalent ocular diseases, today announced that the U.S. Food and Drug Administration (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.

“The RMAT designation is based on the clinical data from Ixo-vec to date and recognizes the transformative potential of our gene therapy for patients with wet AMD,” stated Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies. “This validates that Ixo-vec has the potential to address the significant unmet medical needs in treating nAMD. 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.”

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies. A regenerative medicine therapy is eligible 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. The RMAT designation provides 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.

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 repeated 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 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. In recognition of the need for new treatment options for wet AMD, 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.

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.

Inquiries:

Adverum Investor Relations

Email: ir@adverum.com

FAQ

What is the RMAT designation granted to Adverum Biotechnologies' Ixo-vec?

The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Adverum Biotechnologies' Ixo-vec, a gene therapy candidate for treating wet AMD. This designation is designed to expedite drug development and review processes for promising pipeline products.

What potential benefits does the RMAT designation offer ADVM for Ixo-vec?

The RMAT designation offers Adverum Biotechnologies (ADVM) benefits such as intensive FDA guidance on efficient drug development, potential priority review of the biologics license application (BLA), and other opportunities to expedite the development and review process for Ixo-vec.

When will Adverum Biotechnologies (ADVM) share updates on regulatory discussions for Ixo-vec?

Adverum Biotechnologies (ADVM) plans to share updates from regulatory discussions regarding Ixo-vec in the fourth quarter of 2024.

What condition is Adverum Biotechnologies' Ixo-vec designed to treat?

Ixo-vec, Adverum Biotechnologies' gene therapy candidate, is designed to treat wet AMD (age-related macular degeneration), a serious eye condition that can lead to vision loss.