Adverum Biotechnologies (ADVM) FY Conference Transcript

Summary of Adverum Biotechnologies (ADVM) FY Conference - August 13, 2025 Company Overview - Company: Adverum Biotechnologies - Ticker: ADVM - Focus: Developing potential one-time gene therapies for debilitating ocular diseases, specifically targeting wet age-related macular degeneration (AMD) [1][5] Core Points and Arguments - Product Development: - Adverum is in Phase 3 of developing Ixavec, an intravitreal gene therapy delivering aflibercept, which is the gold standard for wet AMD treatment [3][6] - Ixavec aims to provide sustained aflibercept production in the retina, potentially for life [6] - Clinical Trial Results: - In the OPTIC and LUNA trials, Adverum achieved 50% injection freedom and an 80% reduction in treatment burden for hard-to-treat patients [7] - The ARTEMIS trial is currently enrolling rapidly, indicating strong interest from investigators and patients [7] - Market Need: - There are over 1.5 million wet AMD patients in the US and over 20 million globally, with a significant annual diagnosis rate [9] - Despite existing anti-VEGF therapies, many patients experience vision decline due to under-treatment and missed injections, highlighting a large unmet need for more durable treatments [9][10] - Competitive Landscape: - The gene therapy approach is seen as transformational, offering the potential for injection-free treatment, which is highly favored by retina specialists [11][12] - Adverum's Ixavec is positioned as best-in-class with the highest injection-free rates compared to competitors [28] - Patient Preference: - In the LUNA trial, 93% of patients preferred Ixavec over previous treatments, and 95% would choose it if diagnosed with wet AMD in both eyes [25] - Phase 3 Trial Details: - The ARTEMIS trial will randomize approximately 284 patients into two cohorts: one receiving Ixavec and the other receiving aflibercept [27] - The primary endpoint is non-inferiority in best-corrected visual acuity (BCVA) at 52-56 weeks [27] Additional Important Content - Safety Profile: - Ixavec has shown a favorable safety profile with no serious adverse events related to the treatment, and inflammation, when it occurred, was manageable with local steroids [23][24] - Long-term Efficacy: - Patients in the OPTIC trial maintained vision levels and showed significant reductions in central subfield thickness over four years, indicating a disease-modifying effect [14][22] - Market Potential: - Wet AMD represents a large market opportunity for gene therapy, with the potential for mass market pricing due to high prevalence and annual incidence [28] - Future Plans: - Adverum plans to initiate a second Phase 3 trial, Aquarius, in the second half of the year and release additional long-term follow-up data from the LUNA trial in Q4 [29]