LUNA Phase 2 Study in Wet AMD - Study Design
- LUNA trial will evaluate the 2x10^11 vg/eye (2E11) and a new lower 6x10^10 vg/eye (6E10) dose of Ixo-vec, with enhanced prophylactic steroid regimens in patients requiring frequent anti-VEGF injections
- Primary endpoints include the mean change in best corrected visual acuity (BCVA) from baseline to one year and the incidence and severity of adverse events
- Interim data anticipated throughout 2023
REDWOOD CITY, Calif., Sept. 14, 2022 (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 first subject was dosed in the LUNA Phase 2 trial evaluating ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022) for the treatment of wet age-related macular degeneration (wet AMD).
“Dosing the first subject in the Phase 2 LUNA study brings us a step closer to our mission of establishing gene therapy as a new standard of care for some of the leading causes of vision loss,” stated Richard Beckman, M.D., chief medical officer of Adverum Biotechnologies. “The trial is designed to assess the safety and efficacy of two dose levels of a single, in-office intravitreal injection of Ixo-vec for the treatment of wet AMD. We are excited that LUNA will allow us to explore a new, lower 6E10 dose that we hope will build on the robust, durable efficacy and safety profile already demonstrated with the 2E11 dose in the OPTIC trial. We will also be evaluating two enhanced local prophylactic steroid regimens with and without the use of systemic steroids to determine the most effective prophylactic regimen and dose pairing to optimize risk benefit. During 2023, we anticipate reporting initial data from the study, including aflibercept protein expression at Week 10, which we have found in the OPTIC trial to be predictive of long-term expression out to at least three years, and an interim analysis at Week 26.”
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 2E11 Ixo-vec dose and new lower 6E10 dose and across four prophylactic steroid regimens. Specific regimens being evaluated include topical difluprednate (Durezol®), IVT dexamethasone (Ozurdex®), or a combination of either topical Durezol® or IVT Ozurdex® with oral prednisone, with the aim of establishing a prophylactic regimen with minimal need for inflammation management post prophylaxis.
The LUNA trial primary endpoints are mean change in BCVA from baseline to one year and the incidence and severity of adverse events. Key secondary objectives include the mean change in central subfield thickness (CST) from baseline to one year and assessing the effectiveness of prophylactic steroid regimens on minimizing inflammation. Additionally, LUNA will assess aflibercept protein expression starting at Week 10 and include an interim analysis at Week 26. Study participants will also have the option to enroll in a long-term extension study.
“As a principal investigator in LUNA, I am excited at the potential of Ixo-vec to meaningfully reduce the treatment burden of frequent anti-VEGF injections for wet AMD patients,” commented Sean Adrean M.D., partner, Retina Consultants of Orange County. “I’m intrigued by the efficacy profile seen in the OPTIC trial, where a single intravitreal injection of Ixo-vec demonstrated three years of stable aflibercept expression to date, as well as maintenance of vision and improved anatomical outcomes. I look forward to studying the potential of Ixo-vec to offer a safe and efficacious, and potentially transformational, intravitreal treatment option for my patients with wet AMD.”
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 LUNA Trial of Ixo-Vec in Wet AMD
Ixoberogene soroparvovec (Ixo-vec) is Adverum’s clinical-stage gene therapy product candidate being developed 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 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. Ixo-vec received PRIME designation from the European Medicines Agency in June 2022.
The LUNA trial is a multicenter, double-masked, randomized, parallel-group Phase 2 study evaluating two doses of Ixo-vec, including the 2x10^11 vg/eye dose and a new, lower 6x10^10 vg/eye dose, in wet AMD. The trial will randomize up to 72 participants equally across two doses and four prophylactic steroid regimens in approximately 40 sites in the U.S. and Europe.
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 research 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 these 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.
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 and the timing of preliminary data from the LUNA trial evaluating the same. 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, 2022 filed with the SEC on August 11, 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.
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