Adverum Initiates ARTEMIS Phase 3 Study to Evaluate Ixo-vec for Wet AMD

Adverum Biotechnologies announced the initiation of ARTEMIS phase 3 study to evaluate Ixo-vec gene therapy (ixoberogene soroparvovec) as a one-time intravitreal (IVT) injection for neovascular (wet) age-related macular degeneration (AMD).

Wet AMD is a leading cause of blindness in the elderly, affecting approximately 20 million individuals worldwide. The disease is bilateral, with the second eye being affected in up to 42% of patients in the first two to three years. The current standard of care requires frequent, life-long repeated injections of anti-VEGF in the eye.

The ARTEMIS study will compare a single administration of Ixo-vec to aflibercept, an anti-VEGF, every 8 weeks in approximately 284 patients with wet AMD. The study’s primary endpoint will be mean change in best corrected visual acuity (BCVA) at one year to assess for non-inferiority. Both treatment-naïve and previously treated patients will be included, which the study leads expect to quicken enrollment and best simulate real-world populations.

“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 landscape with a one-time IVT injection to help restore vision and prevent blindness,” Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies, said in a recent press release. 

Life-long repeated IVT injections can cause significant disease burden for many patients. Dr. Dante Pieramici, Medical Director of Research, California Retina Consultants and an investigator for ARTEMIS, cited recent data suggesting up to 42% of patients stop treatment for wet AMD after 2 years, which results in poor long-term visual outcomes. 

“Ixo-vec’s potential to dramatically reduce treatment burden, provide lifelong injection freedom and enhance long-term vision outcomes could deliver a compelling benefit for a large proportion of wet AMD patients,” Dr. Pieramici said. 

OPTIC and LUNA are Ixo-vec’s phase 1 and 2 clinical trials, respectively. In these trials, Ixo-vec reliably produced therapeutic levels of aflibercept with an acceptable long-term safety profile, suggesting an effective lifelong therapy that is well-tolerated. Many of the previous investigators continue to be part of the team piloting ARTEMIS.