Screening for Adverum’s first Phase 3 trial of its intravitreal gene therapy for wet AMD will close by September 30, seven months after trial initiation. Full enrollment of at least 284 patients is now projected for 4Q 2025, pulled forward from 1Q 2026, with a topline readout targeted for 1Q 2027.
The core move is operational: ARTEMIS, the first of two planned registrational studies of ixoberogene soroparvovec (Ixo-vec), is tightening its intake window based on faster-than-expected screening velocity. The study evaluates a single intravitreal administration of Ixo-vec (6E10 vg/eye) against aflibercept 2 mg dosed every eight weeks in both treatment-naïve and previously treated wet AMD patients. ARTEMIS positions Adverum’s office-based gene therapy approach—using the AAV.7m8 capsid delivering an aflibercept transgene—against a widely used anti-VEGF regimen in an active-comparator design. The program carries FDA Fast Track and RMAT status, alongside EMA PRIME and the UK Innovation Passport.
Strategically, the accelerated closure of screening looks like a pacing decision to control conversion from the screen log to randomization while locking in timelines. It also insulates the program from a shifting comparator landscape as higher-dose aflibercept and extended-interval regimens gain traction. By capturing a broad mix of treatment-naïve and previously treated patients, Adverum is maximizing the enrollment funnel but will need to manage heterogeneity at analysis. The choice of an office-based intravitreal delivery, rather than a surgical subretinal approach, is a clear bet on operational adoption by retina clinics—if the safety and durability profile holds. It also signals urgency: competing long-acting anti-VEGF strategies, including alternative gene therapies and device-enabled durability, are converging on the same value proposition of fewer injections and sustained disease control.
For sites, early closure of screening will concentrate efforts on converting the existing pipeline and managing early post-dosing care, including corticosteroid prophylaxis and monitoring for intraocular inflammation and pressure elevations. Sites will need robust SOPs for gene therapy handling, immune management, and vector-related testing without the support structures typical of surgical gene therapy settings. CROs and vendors should anticipate heavier-than-standard ophthalmic central imaging, immune monitoring, and safety adjudication workloads, as well as CMC-driven documentation demands common to AAV programs. Regulators have already signaled openness via expedited designations, but the bar remains high: non-inferiority or superiority on visual and anatomic endpoints, clinically meaningful reduction in rescue injections, and a clean inflammation profile will be essential to offset the permanence and front-loaded risk of a one-time therapy. Payers will focus on durability, retreatment rates, and comparator relevance to contemporary practice.
Next, watch for activation and cadence of the second Phase 3 trial, clarity on primary and key secondary endpoints, and details of steroid regimens and rescue criteria, which will shape both safety outcomes and interpretability. Manufacturing scale and lot-to-lot consistency for AAV.7m8 will be a gating factor before any BLA discussions. The main risks are familiar: immune-mediated ocular events, durability that falls short of cost and risk expectations, and a comparator framework that lags evolving real-world dosing patterns. If ARTEMIS converts its operational momentum into a clean one-year efficacy and safety dataset, the program could force a practical question for retina practices: are they ready to integrate office-based gene therapy into routine care pathways, and under what reimbursement terms?
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
