Adverum Biotechnologies (ADVM) investor relations material

Key clinical developments

• Advancing a gene therapy for wet AMD, currently in phase three with Ixavec, which delivers sustained aflibercept to the retina.

• Phase one OPTIC and phase two LUNA trials showed up to 50% injection freedom and 80% reduction in treatment burden for hard-to-treat patients.

• Four-year OPTIC data demonstrated maintained vision and significant reduction in retinal fluid, with nearly half of patients remaining injection free.

• LUNA trial confirmed high injection-free rates and dramatic treatment burden reduction, especially in patients with lower prior injection frequency.

• Safety profile improved with lower doses and enhanced steroid prophylaxis, with minimal inflammation and high patient preference for the therapy.

Market opportunity and unmet need

• Wet AMD affects over 1.5 million in the US and 20 million globally, with a growing patient pool due to aging populations.

• Despite anti-VEGF therapies, under-treatment remains common, leading to vision decline; durability is the leading unmet need.

• Gene therapy is viewed as the most promising modality for extended treatment duration by retina specialists.

• Ixavec offers the highest injection-free rates among ocular gene therapies, with a significant advantage over competitors.

• Wet AMD could become the first mass market gene therapy indication, with potential for broad adoption and mass market pricing.

Forward-looking statements and next steps

• ARTEMIS phase three trial is enrolling ahead of schedule, targeting a broad patient population and aiming for non-inferiority in vision outcomes.

• Second phase three trial, Aquarius, is planned to start in the second half of the year.

• Additional two-year follow-up data from LUNA expected in Q4 this year.

• Anticipates seamless integration into retina practices due to the established use of aflibercept.

• Long-term vision is to expand gene therapy adoption from high-need patients to all wet AMD patients.