Adverum Biotechnologies Granted Innovative Licensing and Access Pathway Designation in the United Kingdom for Ixo-vec for the Treatment of Wet AMD

Rhea-AI Summary

Adverum Biotechnologies (NASDAQ: ADVM) announced that its investigational gene therapy, Ixo-vec, received an Innovation Passport from the UK's Medicines and Healthcare Products Regulatory Agency under the Innovative Licensing and Access Pathway. This designation aims to expedite regulatory processes and enhance patient access. Ixo-vec has also secured Fast Track designation from the FDA and PRIME designation from the EMA. The therapy shows promise with over 80% reduction in anti-VEGF injections in the OPTIC trial, suggesting potential for a functional cure for wet AMD. The ongoing LUNA trial will assess further outcomes, with interim results expected in Q3 2023.

Positive

• Ixo-vec received Innovation Passport designation from the MHRA to accelerate regulatory review in the UK.
• Fast Track designation by the FDA and PRIME designation by the EMA both highlight Ixo-vec's potential.
• OPTIC trial data shows over 80% reduction in annualized anti-VEGF injections, indicating significant therapeutic benefits.

Negative

• None.

- Innovative Licensing and Access Pathway designation is intended to accelerate the regulatory review process in the United Kingdom -

- Ixo-vec has also been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) designation by the European Medicines Agency (EMA) -

REDWOOD CITY, Calif., April 18, 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 that the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted Ixo-vec (ixoberogene soroparvovec), an investigational gene therapy for the treatment of wet AMD, an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP). The Innovation Passport is the first step in the ILAP process, triggering the MHRA and its partner agencies, including the National Institute for Health and Care Excellence (NICE) and the National Health Service (NHS) England, to partner with Adverum to charter a roadmap for regulatory and development milestones with the goal of early patient access in the United Kingdom (U.K.)

“Our ILAP designation recognizes the innovative potential of Ixo-vec to deliver long-term, therapeutic levels of aflibercept after a single intravitreal injection based on the two-year data from our OPTIC trial. The OPTIC data indicates a greater than 80% reduction in annualized anti-VEGF injections with more than 50% of the trial subjects being supplemental injection-free at two years post-treatment with Ixo-vec, which data is suggestive of a functional cure,” commented Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies. “The current standard of care in wet AMD, requires frequent anti-VEGF injections in the eye that can be a lifelong burden for many patients and their caregivers. We believe Ixo-vec has the potential to provide an in-office treatment option that is durable and safe, provides cost-savings, and addresses the needs of wet AMD patients and their families, retina specialists and health systems worldwide. We look forward to leveraging the benefits of ILAP designation, as we continue our development of Ixo-vec.”

“The ILAP designation grants us enhanced regulatory and market access interactions and a potentially faster path towards approval in the U.K. Ixo-vec has already received Fast Track Designation by the FDA and PRIME designation by the EMA,” said Star Seyedkazemi, PharmD, chief development officer of Adverum Biotechnologies. “Our LUNA trial is ongoing, and we plan to provide interim datasets for a percentage of participants at 14 weeks, including aflibercept protein levels, in the third quarter of 2023, as well as preliminary efficacy and safety data in the fourth quarter.”

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 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.

About Ixo-Vec in Wet AMD
Adverum is developing ixoberogene soroparvovec (Ixo-vec), 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 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 LUNA Trial of Ixo-vec in Wet AMD
The LUNA trial is a double-masked, randomized, Phase 2 trial being conducted at approximately 40 sites in the U.S. and Europe. LUNA will evaluate Ixo-vec in subjects with wet AMD who are 50 years or older and have demonstrated a response to anti-VEGF treatment. Up to 72 subjects will be randomized equally between the previously evaluated 2E11 vg/eye dose and a new, lower 6E10 vg/eye dose. Four prophylactic steroid regimens will be studied with the aim of establishing a prophylactic corticosteroid regimen with minimal need for inflammation management post prophylaxis. Prophylactic regimens being evaluated include 22 weeks of a tapered regimen of topical difluprednate (Durezol^®), a single administration of IVT dexamethasone (Ozurdex^®), and a combination of either topical Durezol^® or IVT Ozurdex^® with up to 10 weeks of a tapered regimen of oral prednisone. All four prophylactic corticosteroid regimens in LUNA cover the period of peak immunogenicity observed in non-clinical studies and in the Phase 1 OPTIC study.

The LUNA trial primary endpoints are mean change in best corrected visual acuity (BCVA) from baseline to one year as well as the incidence and severity of adverse events. Important secondary endpoints in LUNA include the mean change in central subfield thickness (CST) from baseline to one year and assessing the effectiveness of prophylactic corticosteroid regimens on minimizing inflammation. Additionally, LUNA will assess aflibercept protein levels starting at Week 14 and include an interim analysis at Week 26. Study participants will have the option to enroll in a long-term extension study.

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. 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, the effectiveness of the prophylactic corticosteroid regimens, the LUNA study design, the timing of LUNA enrollment, the approval path in the United Kingdom, and the timing of preliminary 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 Annual Report on Form 10-K for the year ended December 31, 2022 filed with the SEC on March 30, 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.

Corporate, Investor and Media Inquiries
Anand Reddi
Vice President, Head of Corporate Strategy, External Affairs and Engagement
Adverum Biotechnologies, Inc.
T: 650-649-1358
E: areddi@adverum.com

FAQ

What is Ixo-vec and its significance for wet AMD treatment?

Ixo-vec, an investigational gene therapy by Adverum, is designed to provide long-term treatment for wet AMD, reducing the need for frequent injections.

What regulatory designations has Ixo-vec received?

Ixo-vec has received the Innovation Passport from the UK's MHRA, Fast Track designation from the FDA, and PRIME designation from the EMA.

When can we expect interim results from the LUNA trial?

Interim datasets from the LUNA trial are anticipated in the third quarter of 2023.

What were the results of the OPTIC trial for Ixo-vec?

The OPTIC trial indicated over 80% reduction in annualized anti-VEGF injections, with many subjects being injection-free after two years.

What is the current status of the LUNA trial?

The LUNA trial is ongoing and evaluates Ixo-vec in subjects with wet AMD; results are expected in upcoming quarters.