Adverum Biotechnologies (ADVM) UBS Virtual Ophthalmology Day summary

Program overview and technology

• Ixo-vec is a phase III-ready gene therapy for wet AMD, using a proprietary 7m8 capsid for intravitreal delivery of a codon-optimized Eylea transgene.

• The therapy enables in-office administration, avoiding complex subretinal surgery, and achieves broad transduction of the retina and choroid.

• Converts the eye into a biofactory to produce Eylea at the disease site, supporting strong efficacy.

Market landscape and competitive positioning

• Gene therapy offers potential for long-term, injection-free treatment, addressing significant unmet needs in wet AMD.

• Current standard of care is shifting to VABYSMO and Eylea high dose, but real-world dosing intervals remain suboptimal.

• Competing gene therapies use different delivery methods or transgenes, with Ixo-vec offering advantages in administration and efficacy.

• Ixo-vec demonstrates higher injection-free rates and treatment burden reduction compared to competitors like REGENXBIO and 4DMT.

Clinical trial data and outcomes

• OPTIC trial shows over 50% of patients injection-free at years one, two, and three, with 80% annualized injection reduction and maintained vision.

• Three years of safety data indicate manageable, dose-dependent inflammation, resolved with steroids and no impact on vision.

• LUNA phase II trial at 6E10 dose shows 76% injection-free at six months, 90% reduction in treatment burden, and favorable safety profile.

• Patient preference data: 88% preferred Ixo-vec over prior injections, 93% would want it in their second eye.