Adverum Biotechnologies Initiates Phase 3 ARTEMIS Study for Gene Therapy in Wet AMD

REDWOOD CITY, Calif., March 3, 2025 – Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company focused on preserving vision, has announced the initiation of the ARTEMIS Phase 3 trial. This pivotal study will assess the efficacy and safety of Ixo-vec (ixoberogene soroparvovec), a one-time intravitreal (IVT) gene therapy, for patients with neovascular (wet) age-related macular degeneration (AMD).

ARTEMIS is the first registrational study of its kind in wet AMD, evaluating a single injection of Ixo-vec (6E10 vg/eye) against aflibercept (2 mg) administered every eight weeks. The trial will enroll approximately 284 patients, including both treatment-naïve and previously treated individuals. The primary endpoint is the mean change from baseline in best corrected visual acuity (BCVA) at one year, with a non-inferiority margin of -4.5 letters. Following FDA guidance, all patients will receive three loading doses of aflibercept before receiving Ixo-vec. Both treatment arms will allow for supplemental aflibercept injections, with patients also receiving prophylactic steroid eye drops.
The ARTEMIS trial is the first of two planned Phase 3 studies for Ixo-vec. The second study, AQUARIUS, will be conducted globally, with further details to be announced.

Transforming Wet AMD Treatment with Gene Therapy

“Gene therapy represents one of the most promising advancements for wet AMD, and the launch of ARTEMIS marks a significant step forward in our mission to reduce treatment burden and improve vision outcomes,” said Laurent Fischer, M.D., President and CEO of Adverum Biotechnologies. “With over four years of clinical experience, we believe Ixo-vec is positioned as a best-in-class, potentially lifelong treatment option for patients who rely on frequent injections to maintain their sight.”

Rabia Gurses Ozden, M.D., Chief Medical Officer at Adverum, highlighted the trial’s broad applicability: “By including both treatment-naïve and previously treated patients, ARTEMIS is designed to generate a dataset that reflects real-world patient demographics, accelerating enrollment and enhancing the likelihood of clinical, regulatory, and commercial success.”

Investigator Perspectives on ARTEMIS

Leading retina specialists emphasized the urgency of advancing long-lasting therapies for wet AMD, as up to 42% of patients discontinue treatment within two years, leading to poor long-term vision outcomes.

• Dr. Dante Pieramici, Medical Director of Research at California Retina Consultants and ARTEMIS investigator, stated: “Ixo-vec’s potential to provide lasting efficacy and injection freedom is highly appealing to patients. The strong preference for Ixo-vec seen in prior trials like LUNA underscores the demand for innovative, long-term solutions.”
• Dr. Sean Adrean, Partner at Retina Consultants of Orange County and ARTEMIS investigator, added: “By enrolling both treatment-naïve and experienced patients, ARTEMIS is well-positioned to answer critical questions about how gene therapy can benefit those with the highest treatment burden.”
• Dr. Carl Danzig, Rand Eye Institute and ARTEMIS investigator, noted: “In prior studies, Ixo-vec has demonstrated durable aflibercept production with a favorable safety profile, supporting its potential as a transformative, one-time treatment for wet AMD.”

Looking Ahead

With the ARTEMIS trial now underway, Adverum continues to advance its gene therapy pipeline with the goal of delivering sustained, long-term vision benefits to patients with wet AMD. The company plans to expand its Phase 3 program globally with the upcoming AQUARIUS trial and remains committed to establishing Ixo-vec as a groundbreaking treatment option.

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