Adverum Biotechnologies has initiated a phase 3 study, ARTEMIS, which will evaluate the efficacy and safety of its gene therapy Ixo-vec (ixoberogene soroparvovec) as a one-time intravitreal (IVT) injection for patients with neovascular age-related macular degeneration (nAMD). Ixo-vec uses an AAV.7m8 viral vector to deliver a modified aflibercept gene to retinal cells, enabling continuous production of anti-VEGF therapy and reducing the need for frequent injections.

ARTEMIS is a US-based study evaluating a single administration of Ixo-vec (6E10 vg/eye) compared to aflibercept (2 mg) every 8 weeks in approximately 284 patients with wet AMD, including both treatment-naïve and previously treated patients. The primary endpoint is mean change from baseline in best-corrected visual acuity (BCVA) at 1 year (average of weeks 52 and 56) with a noninferiority margin of -4.5 letters. Per FDA guidance, all patients will receive 3 loading doses of aflibercept prior to receiving Ixo-vec. Patients in both arms will be eligible for supplemental injections of aflibercept and will receive prophylactic steroid eye drops.

ARTEMIS is the first of 2 planned phase 3 registrational trials to evaluate Ixo-vec in patients with wet AMD, the company said in a press release. Information about the second study, named AQUARIUS and to be conducted globally, will be forthcoming, the company said. RP