Adverum Biotechnologies Q2 2024 Financial Results and Program Highlights

Adverum Biotechnologies, Inc., a clinical-stage company dedicated to pioneering gene therapy for treating ocular diseases, has announced its financial results for the second quarter of 2024 and shared updates regarding its Ixo-vec program.

The LUNA trial, an essential investigation into whether lower doses of Ixo-vec with enhanced prophylactic regimens could present a product profile on par with or superior to that seen in the OPTIC study, has shown promising results. At the ASRS 2024 conference, the 26-week interim analysis demonstrated that the 6E10 dose of Ixo-vec has achieved this goal. According to Laurent Fischer, M.D., CEO of Adverum, the results indicate that this dose, combined with local prophylaxis, may offer a best-in-class product profile, supporting the choice of the 6E10 dose for upcoming Phase 3 pivotal trials.

Highlighting the effectiveness of Ixo-vec in the OPTIC study, Fischer noted that even with suboptimal prophylaxis, 93% of patients treated were free of inflammation after one year, and 100% were free after two years. The inflammation, when it did occur, was manageable with topical corticosteroids. The LUNA trial's interim results at 26 weeks further bolstered these findings, showing a higher proportion of patients free from injections compared to the OPTIC study, along with an improved safety profile.

Adverum is also looking forward to the 9-month data from the LUNA trial and updates on the design of their pivotal program, which are expected in the fourth quarter of 2024.

In another significant development, the FDA granted Ixo-vec the Regenerative Medicine Advanced Therapy (RMAT) designation for treating wet age-related macular degeneration (AMD), recognizing the transformative potential of this gene therapy. This designation provides benefits like intensive FDA guidance and potential priority review of the biologics license application.

The company also made notable appointments to its leadership team. Dr. Rabia Gurses Ozden has been appointed as Chief Medical Officer, and Dr. Szilárd Kiss has joined the Board of Directors, filling the vacancy left by Dr. Ozden. These changes aim to strengthen Adverum’s leadership as it advances its clinical and corporate goals.

Adverum's financial health appears robust, with $173.8 million in cash, cash equivalents, and short-term investments as of June 30, 2024. This financial reserve is expected to support operations into late 2025. The company reported research and development expenses of $17.1 million for the second quarter, a decrease from $20.6 million in the same period in 2023. This reduction was attributed to lower facility-related and compensation expenses. General and administrative expenses also saw a significant decrease, from $12.5 million in the second quarter of 2023 to $3.8 million in the second quarter of 2024, due to various cost-saving measures.

Regarding wet AMD, this condition is a leading cause of blindness in people over 65, affecting approximately 20 million individuals globally. Wet AMD results from the build-up of fluid in the macula and retina and is typically treated with frequent anti-VEGF injections. Adverum's Ixo-vec aims to revolutionize this treatment by providing a one-time gene therapy that could eliminate the need for regular injections.

Ixo-vec, using a proprietary vector capsid and an aflibercept coding sequence, is designed for a single intravitreal injection, potentially offering long-term efficacy and improved patient compliance. The ongoing LUNA Phase 2 trial and OPTIC Phase 1 extension study are evaluating its effectiveness. The FDA has granted Fast Track and RMAT designations for Ixo-vec, and it has also received PRIME designation from the European Medicines Agency and the Innovation Passport from the UK’s Medicines and Healthcare products Regulatory Agency.

Adverum Biotechnologies remains committed to establishing gene therapy as a new standard of care for ocular diseases, aiming to eliminate the recurrent need for treatments and to bring about significant improvements in vision outcomes for patients worldwide.