Adverum Biotechnologies(ADVM) - 2024 Q2 - Quarterly Results

markdown [Overview and Key Highlights](index=1&type=section&id=Overview%20and%20Key%20Highlights) Adverum announced Q2 2024 results, selecting Ixo-vec's 6E10 dose for Phase 3 trials and reporting **$173.8 million** in cash - The 6E10 dose of Ixo-vec has been selected for Phase 3 pivotal trials following promising interim results[1](index=1&type=chunk) - The LUNA 26-week interim analysis demonstrated a potential best-in-class product profile for Ixo-vec, with similar efficacy and a more favorable safety profile compared to the earlier OPTIC study[1](index=1&type=chunk)[2](index=2&type=chunk) - The company holds **$173.8 million** in cash, cash equivalents, and short-term investments, which is expected to fund operations into late 2025[1](index=1&type=chunk)[8](index=8&type=chunk) [Ixo-vec Program Highlights](index=1&type=section&id=Ixo-vec%20Program%20Highlights) Ixo-vec achieved key milestones, including positive LUNA Phase 2 data leading to 6E10 dose selection for Phase 3, and FDA RMAT designation LUNA 26-Week Interim Analysis Highlights (6E10 dose) | Metric | Result | | :--- | :--- | | Injection-Free Patients | **76%** | | Safety Profile | Improved inflammatory profile compared to OPTIC | | Patient Preference (difluprednate-alone regimen) | **100%** would prefer Ixo-vec over prior treatments | - The U.S. FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to Ixo-vec for treating wet AMD, recognizing its transformative potential and offering benefits like intensive FDA guidance and potential priority review[4](index=4&type=chunk) [Corporate Updates and Outlook](index=2&type=section&id=Corporate%20Updates%20and%20Outlook) Adverum strengthened leadership with new appointments and outlined a roadmap for upcoming milestones, including Phase 3 trial initiation in H1 2025 - In June 2024, Dr. Rabia Gurses Ozden was appointed Chief Medical Officer, and Dr. Szilárd Kiss joined the Board of Directors[5](index=5&type=chunk)[6](index=6&type=chunk) Upcoming Anticipated Milestones | Timeline | Milestone | | :--- | :--- | | 2H 2024 | Continued FDA and EMA regulatory interactions | | 4Q 2024 | LUNA 9-month analysis clinical data | | 4Q 2024 | Phase 3 pivotal trial design update | | 1Q 2025 | LUNA 52-week analysis clinical data | | H1 2025 | Planned initiation of Phase 3 trial | [Financial Results](index=3&type=section&id=Financial%20Results) Adverum reported a Q2 2024 net loss of **$18.5 million**, reduced from Q2 2023 due to decreased expenses, with cash increasing to **$173.8 million** [Key Financial Metrics (Q2 2024)](index=3&type=section&id=Key%20Financial%20Metrics%20(Q2%202024)) In Q2 2024, Adverum's cash and investments grew to **$173.8 million**, with R&D and G&A expenses decreasing, improving net loss per share - Cash, cash equivalents, and short-term investments stood at **$173.8 million** as of June 30, 2024, compared to **$96.5 million** at the end of 2023[8](index=8&type=chunk) Operating Expenses (Three months ended June 30) | Expense Category | 2024 ($ millions) | 2023 ($ millions) | | :--- | :--- | :--- | | Research & Development | $17.1 | $20.6 | | General & Administrative | $3.8 | $12.5 | Net Loss Summary (Three months ended June 30) | Metric | 2024 ($ millions) | 2023 ($ millions) | | :--- | :--- | :--- | | Net Loss | $18.5 | $31.5 | | Net Loss per Share | $0.89 | $3.13 | [Financial Statements](index=5&type=section&id=Financial%20Statements) The report includes selected data from the Consolidated Balance Sheet and Statements of Operations, providing a snapshot of financial position Selected Consolidated Balance Sheet Data | Metric | June 30, 2024 (Unaudited, $ thousands) | Dec 31, 2023 ($ thousands) | | :--- | :--- | :--- | | Cash, cash equivalents, and marketable securities | $173,827 | $96,526 | | Total assets | $253,803 | $173,010 | | Total stockholders' equity | $168,029 | $83,469 | Condensed Consolidated Statements of Operations (Three months ended June 30) | Metric | 2024 (Unaudited, $ thousands) | 2023 ($ thousands) | | :--- | :--- | :--- | | Total operating expenses | $20,882 | $33,065 | | Operating loss | $(20,882) | $(33,065) | | Net loss | $(18,476) | $(31,510) | [Company and Product Information](index=3&type=section&id=Company%20and%20Product%20Information) This section provides background on wet AMD, the target indication for Ixo-vec, detailing the disease, technology, and regulatory designations [About Wet Age-Related Macular Degeneration (AMD)](index=3&type=section&id=About%20Wet%20Age-Related%20Macular%20Degeneration%20(AMD)) Wet AMD is a leading cause of blindness globally, affecting **20 million** people, with current treatments posing a burden gene therapy aims to alleviate - Wet AMD is a leading cause of blindness in people over 65, with about **20 million** individuals affected worldwide[10](index=10&type=chunk) The standard treatment requires frequent, lifelong injections into the eye [About Ixo-vec in Wet AMD](index=3&type=section&id=About%20Ixo-vec%20in%20Wet%20AMD) Ixo-vec is Adverum's clinical-stage gene therapy for wet AMD, a one-time IVT injection for long-term efficacy, with multiple expedited regulatory designations - Ixo-vec is a one-time intravitreal (IVT) injection gene therapy designed to deliver long-term efficacy by carrying an aflibercept coding sequence via a proprietary AAV.7m8 vector capsid[11](index=11&type=chunk) - The therapy has received multiple designations to expedite its development, including Fast Track and RMAT from the FDA, PRIME from the EMA, and the Innovation Passport from the UK's MHRA[11](index=11&type=chunk)[12](index=12&type=chunk)