Adverum Biotechnologies (ADVM) Oppenheimer 35th Annual Healthcare Life Sciences Conference (Virtual) 2025 summary

Program overview and innovation

• Developed an intravitreal gene therapy using AAV.7m8 capsid, enabling aflibercept expression in retinal cells after a single injection, with efficacy shown for over five years.

• Approach eliminates need for surgery and fits current standard of care, addressing unmet needs in wet AMD by reducing injection frequency.

• Phase III Artemis trial will enroll 284 patients, comparing single-dose Ixo-vec to Eylea, including both treatment-experienced and naive patients for a broad label.

• Gene therapy targets hardest-to-treat patients, aiming for injection freedom and durable vision preservation.

• Patient preference data shows strong favorability for Ixo-vec over prior treatments.

Market landscape and economics

• Wet AMD market is valued at $9B, projected to reach $12–$15B, with Vabysmo nearing $4B in annual sales due to reduced injection frequency.

• 40% of patients discontinue treatment within two years, leading to vision loss and lost revenue for practices.

• Practice economics are significant, with buy-and-bill models making retina practices profitable and attractive to private equity.

• Gene therapy could be cost-effective, potentially saving Medicare money and fitting into current practice models.

• Even 3% market penetration could yield over $1B in annual revenue.

Clinical data and competitive positioning

• In the Luna trial, 54% of patients were injection-free at one year, outperforming competitors like 4DMT in similar populations.

• Efficacy remains high across both high-need and broader patient populations, with up to 75% of patients requiring less than one injection per year.

• Safety profile is strong, with no vision-threatening events and inflammation managed by topical steroids.

• Five years of clinical experience with Ixo-vec, including dose optimization and enhanced steroid prophylaxis, has led to a de-risked phase III program.

• Zero inflammation at 52 weeks with the 6E10 dose in Luna trial, supporting best-in-class benefit-risk profile.