Adverum Biotechnologies (ADVM) RBC Inaugural Virtual Ophthalmology Conference summary

Key clinical and technological updates

• Four-year data from OPTIC and one-year data from LUNA show long-term efficacy and safety for Ixo-vec, with a majority of patients remaining injection-free and significant reduction in treatment burden.

• Phase III ARTEMIS trial has launched, enrolling both treatment-experienced and treatment-naive patients, with strong interest from over 150 sites in the US.

• Ixo-vec uses the AAV.7m8 capsid, enabling efficient intravitreal delivery and robust, durable aflibercept expression, with a tenfold dose reduction maintaining efficacy.

• Enhanced steroid eye drop regimen in LUNA trial led to improved safety and tolerability, with no serious adverse events and lower inflammation rates.

• Over 90% of patients in a preference survey favored Ixo-vec over standard care, indicating strong patient demand.

Market landscape and commercial outlook

• Wet AMD market exceeds $12 billion, with current standard of care offering only minor improvements in treatment burden.

• Eylea remains the benchmark anti-VEGF therapy; gene therapy is positioned as the only modality with one-and-done potential.

• Clinics may benefit economically from gene therapy by capturing multi-year revenue in a single injection and freeing up clinic time.

• Patient demand and cost-effectiveness for Medicare Part B are expected to drive adoption.

• Biosimilars and new modalities are entering, but gene therapy’s durability and patient preference may support its uptake.

Regulatory and operational developments

• FDA leadership changes are being monitored, but optimism remains for continued support of innovative therapies.

• ARTEMIS phase III trial is actively enrolling, with screening underway at multiple sites and strong investigator enthusiasm.

• Co-pay assistance challenges are motivating patient enrollment in clinical trials.

• Plans are in place to dose the second eye in up to 20 patients by BLA filing, with preclinical data supporting safety.