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Adverum Biotechnologies (ADVM) investor relations material

Adverum Biotechnologies H.C. Wainwright 5th Annual Ophthalmology Virtual Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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H.C. Wainwright 5th Annual Ophthalmology Virtual Conference summary13 Aug, 2025

Key clinical developments

  • Advancing a gene therapy for wet AMD, currently in phase three with Ixavec, which delivers sustained aflibercept to the retina.

  • Phase one OPTIC and phase two LUNA trials showed up to 50% injection freedom and 80% reduction in treatment burden for hard-to-treat patients.

  • Four-year OPTIC data demonstrated maintained vision and significant reduction in retinal fluid, with nearly half of patients remaining injection free.

  • LUNA trial confirmed high injection-free rates and dramatic treatment burden reduction, especially in patients with lower prior injection frequency.

  • Safety profile improved with lower doses and enhanced steroid prophylaxis, with minimal inflammation and high patient preference for the therapy.

Market opportunity and unmet need

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

  • Despite anti-VEGF therapies, under-treatment remains common, leading to vision decline; durability is the leading unmet need.

  • Gene therapy is viewed as the most promising modality for extended treatment duration by retina specialists.

  • Ixavec offers the highest injection-free rates among ocular gene therapies, with a significant advantage over competitors.

  • Wet AMD could become the first mass market gene therapy indication, with potential for broad adoption and mass market pricing.

Forward-looking statements and next steps

  • ARTEMIS phase three trial is enrolling ahead of schedule, targeting a broad patient population and aiming for non-inferiority in vision outcomes.

  • Second phase three trial, Aquarius, is planned to start in the second half of the year.

  • Additional two-year follow-up data from LUNA expected in Q4 this year.

  • Anticipates seamless integration into retina practices due to the established use of aflibercept.

  • Long-term vision is to expand gene therapy adoption from high-need patients to all wet AMD patients.

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Frequently asked questions

Adverum Biotechnologies Inc, originally known as Avalanche Biotechnologies Inc, is a clinical-stage gene therapy company based in Redwood City, California. Focused on addressing unmet medical needs, the company targets serious ocular and rare diseases, including wet age-related macular degeneration (wet AMD). It leverages its proprietary intravitreal (IVT) platform to develop durable, single-administration therapies intended to be delivered in physicians' offices. This approach aims to transform the standard of care for highly prevalent ocular diseases by eliminating the need for frequent ocular injections. The company is headquartered in Redwood City, California, and its shares are listed on the Nasdaq.

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