Adverum Biotechnologies (ADVM) Status Update summary

Clinical trial results and efficacy

• Ixo-vec demonstrated over 80% reduction in injection burden and more than 50% of patients remained injection-free at one year in the LUNA study, with these results sustained through four years in the OPTIC study.

• 6E10 dose in LUNA showed 88% reduction in mean annualized anti-VEGF injections and 54% of patients were injection free at 52 weeks; 2E11 dose showed 92% reduction and 69% injection freedom.

• OPTIC 4-year data showed 86% reduction in annualized injections, with nearly 50% of patients injection free through 4 years.

• Both LUNA and OPTIC studies showed robust maintenance of visual acuity and central subfield thickness, with consistent injection-free rates through four years.

• Ixo-vec addresses key unmet needs in wet AMD, including reducing treatment burden and improving long-term vision outcomes.

Safety and tolerability

• No inflammation was observed at the 6E10 dose in LUNA at 52 weeks or in OPTIC at the 2E11 dose through four years, establishing a tenfold safety margin.

• Inflammation, when present, was mild, dose-dependent, manageable with local steroids, and resolved by week 52; no new inflammation occurred after week 30.

• Extended topical steroid prophylaxis was effective and considered easy to manage by patients, with oral steroids deemed less favorable.

• All Ixo-vec-related adverse events were mild or moderate, with no serious events reported.

• Long-term data establish a 10-fold safety margin from the highest dose tested in nAMD.

Patient experience and preference

• Patients expressed a strong preference for Ixo-vec over prior treatments, with 93% at 52 weeks and 100% at the pivotal dose indicating willingness to use it in both eyes and recommend it to others.

• No patients found the topical eyedrop prophylaxis difficult to manage, and compliance is expected to be high.

• Both KOLs and patients view the safety profile as acceptable and the benefit-risk balance as favorable.

• The product profile is seen as transformative, with potential for broad adoption and significant commercial opportunity.

• Over 90% of patients in LUNA preferred Ixo-vec to standard of care, and 100% of 6E10 + steroid drop patients found the regimen easy to manage.