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

Company and therapy overview

• Presented data on Ixo-vec, an aflibercept-encoding gene therapy for wet AMD, showing stable aflibercept expression up to five years in all tested patients.

• OPTIC and LUNA trials demonstrated maintained vision (BCVA) and significant reduction in disease activity (CST) over multiple years.

• LUNA trial showed 54% of patients were injection-free at one year, with 75% injection-free among those with fewer prior injections.

• Patient preference was high: 93% preferred Ixo-vec, 95% wanted it in the other eye, and 96% would recommend it.

• Favorable safety profile with minimal, well-controlled inflammation using a six-month steroid eye drop regimen.

Patient experience and impact

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

• Patients reported freedom from monthly injections, reduced anxiety, and improved daily functioning.

• No significant side effects or vision-threatening inflammation reported post-treatment.

• Long-term follow-up showed sustained vision gains and life transformation, including increased independence and activity.

• High willingness among patients to receive treatment in both eyes.

Clinical development and trial design

• Phase 3 ARTEMIS trial launched, enrolling both treatment-naive and experienced patients, aiming for non-inferiority to Eylea every eight weeks.

• AQUARIUS, a global phase 3 trial, will follow a similar design with minor adjustments for regulatory requirements.

• Six-month steroid eye drop prophylaxis adopted for phase 3 based on prior trial experience.

• No new inflammation observed after week 30, with all patients inflammation-free at years one through four.

• Plans to study bilateral dosing in at least 15 patients before BLA filing, with regulatory discussions ongoing.