Adverum Biotechnologies (ADVM) TD Cowen 45th Annual Healthcare Conference summary

Clinical program updates and trial data

• Ixo-vec, an aflibercept-encoding gene therapy, demonstrated stable, long-term efficacy in wet AMD, with up to five years of sustained protein expression and vision preservation in clinical trials.

• In the OPTIC and LUNA trials, over 54% of patients were injection-free at one year, with 75% injection freedom in less treatment-experienced patients, and robust reductions in disease activity biomarkers.

• Safety profile was favorable, with minimal, well-controlled inflammation managed by steroid eye drops; no new inflammation observed after week 30 through four years.

• Patient testimonials highlighted significant improvements in quality of life, vision, and reduced treatment burden after a single injection.

• Phase 3 ARTEMIS trial has begun enrolling both treatment-naive and experienced patients, aiming for non-inferiority to Eylea, with a global AQUARIUS trial to follow.

Patient experience and impact

• Patients reported dramatic improvements in daily activities, vision clarity, and independence, with many expressing preference for gene therapy over prior regimens.

• Burden of frequent injections and associated anxiety was alleviated, with some patients injection-free for over a year post-treatment.

• High satisfaction rates: 93% preferred gene therapy, 95% wanted treatment in the other eye, and 96% would recommend it.

• Plans are underway to study bilateral dosing, with regulators requesting data from at least 15 patients before BLA filing.

Competitive landscape and trial design

• Ixo-vec’s intravitreal delivery offers broader applicability and higher efficacy compared to subretinal approaches and other gene therapies.

• ARTEMIS and AQUARIUS phase 3 trials include both treatment-naive and experienced patients, aiming for a broad label and efficient trial design.

• No need for booster injections due to robust, sustained protein expression; this may differentiate the product in labeling and practice.

• Six-month steroid eye drop prophylaxis was selected for phase 3 based on ease of use and strong safety data.