Adverum Biotechnologies (ADVM) Status Update summary

Clinical results and product profile

• 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 and 54% injection freedom at 52 weeks, while 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.

• No inflammation was observed at the 6E10 dose in LUNA at 52 weeks or any subsequent visit, and inflammation, when present, was mild, manageable, dose-dependent, and resolved by year one without impacting vision.

• The product profile is supported by a tenfold safety margin and robust, durable efficacy, with aflibercept levels sustained for up to five years.

• Ixo-vec is positioned as a one-time gene therapy with the potential for lifelong benefit, addressing major unmet needs in wet AMD, and is designed for office administration.

Patient experience and preference

• 93% of LUNA patients at 52 weeks preferred Ixo-vec and its steroid regimen over prior treatments, increasing from 88% at 26 weeks; 100% of patients on the 6E10 pivotal dose and topical eyedrop steroid regimen preferred Ixo-vec and would recommend it to others.

• No patients found the topical eyedrop prophylaxis difficult to manage, and 95% would elect to receive Ixo-vec in the other eye if both eyes had wet AMD.

• Patient preference data show overwhelming support for Ixo-vec, with ease of use for steroid eyedrops and strong willingness to recommend and use the therapy in both eyes.

• Both injection freedom and burden reduction are important to patients, with vision preservation as the primary goal.

Safety and tolerability

• No LUNA patients on local steroid prophylaxis had inflammation at week 52 or later; all OPTIC 2E11 patients were free of inflammation through year 4.

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

• Extended topical steroid prophylaxis is effective and manageable, with minimal long-term steroid use required.

• Compliance with eyedrop regimens is expected to be high, as patients are motivated to avoid injections; lapses can be managed without loss of efficacy.

• The 6E10 dose is considered optimal for both efficacy and safety, and further dose reduction is not planned.