Adverum Launches ARTEMIS Phase 3 Trial for Wet AMD

Adverum Biotechnologies has announced the initiation of the ARTEMIS phase 3 study, designed to evaluate Ixo-vec (ixoberogene soroparvovec) gene therapy as a one-time intravitreal (IVT) injection for the treatment of neovascular (wet) age-related macular degeneration (AMD). The study aims to compare Ixo-vec with aflibercept, an anti-VEGF therapy, in terms of visual acuity outcomes and treatment burden reduction.

Burden of Wet AMD and Current Standard of Care

Wet AMD is a leading cause of blindness among the elderly, affecting approximately 20 million individuals worldwide. The disease is often bilateral, with the second eye developing AMD in up to 42 percent of patients within two to three years.

The current standard of care for wet AMD requires lifelong, frequent intravitreal injections of anti-VEGF therapies, which presents a significant burden for patients. According to recent data, up to 42 percent of patients discontinue treatment within two years, leading to poor long-term visual outcomes.

ARTEMIS Phase 3 Study Design

The ARTEMIS study will evaluate Ixo-vec as a single IVT injection compared to aflibercept administered every eight weeks. The trial will enroll approximately 284 patients with wet AMD, including both:

• Treatment-naïve patients

• Previously treated patients

The primary endpoint will be the mean change in best corrected visual acuity (BCVA) at one year, assessing non-inferiority between Ixo-vec and aflibercept. The inclusion of both treatment-naïve and previously treated patients is expected to accelerate enrollment and better reflect real-world populations.

Potential of Gene Therapy in Wet AMD

"Gene therapy is recognized as the most exciting modality on the horizon for the treatment of wet AMD. Initiation of the phase 3 ARTEMIS trial demonstrates our commitment to transforming the treatment landscape with a one-time IVT injection to help restore vision and prevent blindness," said Laurent Fischer, M.D., President and CEO of Adverum Biotechnologies.

According to Dr. Dante Pieramici, Medical Director of Research at California Retina Consultants and an ARTEMIS investigator, Ixo-vec has the potential to dramatically reduce treatment burden, provide lifelong injection freedom, and improve long-term vision outcomes for a large proportion of wet AMD patients.

Supporting Clinical Trials: OPTIC and LUNA

Ixo-vec has already been studied in:

• OPTIC (Phase 1 Trial)

• LUNA (Phase 2 Trial)

Findings from these trials indicate that Ixo-vec consistently produced therapeutic levels of aflibercept with an acceptable long-term safety profile, suggesting a well-tolerated and potentially lifelong therapy. Many investigators from these earlier studies remain involved in piloting ARTEMIS.

With the launch of ARTEMIS, Adverum Biotechnologies aims to bring a transformative, long-term solution to patients with wet AMD, reducing the need for repeated injections and improving long-term visual outcomes.