Adverum Biotechnologies (ADVM) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Program overview and clinical development

• Lead gene therapy program, Ixo-Vec, targets wet AMD and is advancing to phase III in H1 2025 using a proprietary 7m8 vector.

• Ixo-Vec aims for a one-time, functional cure by enabling the eye to produce its own anti-VEGF.

• Phase III trial design and next data release are expected in Q4 2024.

• LUNA phase II trial fully enrolled; interim data presented for 6 months, showing strong efficacy and safety.

• OPTIC phase I/II trial demonstrated long-term benefit and safety out to three years.

Efficacy and safety data

• At the 6E10 dose, 76% of patients were injection-free at six months, with a best-in-class profile.

• Over 50% of OPTIC patients remained injection-free at three years, with an 84% reduction in treatment burden.

• LUNA interim results showed 79% injection-free rate overall, and 90% in a subset with fewer prior injections.

• No Ixo-Vec-related serious adverse events in LUNA; all related AEs were mild or moderate and managed with local corticosteroids.

• Inflammation was dose-dependent but resolved with treatment and did not impact vision.

Market opportunity and patient impact

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

• Current standard of care requires frequent anti-VEGF injections, creating significant burden for patients and healthcare systems.

• Gene therapy offers potential for a functional cure and improved long-term vision outcomes.

• Patient preference survey showed 88% preferred Ixo-Vec over prior therapy, and 93% would opt for it in the second eye.

• 100% of lower-dose, drops-only arm patients would choose Ixo-Vec for their second eye.