Annexon(ANNX) - 2025 Q1 - Quarterly Results

Q1 2025 Overview and Corporate Update Annexon reported significant progress across its late-stage clinical portfolio in Q1 2025, ending the quarter with $263.7 million in cash and investments, providing an operational runway into the second half of 2026 - Annexon is advancing a late-stage clinical platform of novel therapies targeting classical complement-mediated neuroinflammatory diseases of the body, brain, and eye[1] - The company's CEO, Douglas Love, highlighted that the C1 platform has produced multiple late-stage programs showing positive patient outcomes by stopping harmful neuroinflammation[2] Key Anticipated Milestones | Program | Milestone | Anticipated Timing | | :--- | :--- | :--- | | Tanruprubart (GBS) | FDA Meeting for BLA Submission | Q2 2025 | | Tanruprubart (GBS) | Initiation of Open-Label FORWARD Study | Q2 2025 | | ANX007 (GA) | Completion of Phase 3 ARCHER II Enrollment | Q3 2025 | | ANX007 (GA) | Pivotal Topline Data | H2 2026 | | ANX1502 (CAD) | Completion of Proof-of-Concept Trial | Mid-2025 | - As of March 31, 2025, the company has $263.7 million in cash, cash equivalents, and short-term investments, which is expected to fund operations into the second half of 2026[1][2] Clinical Program Updates Annexon is advancing its three flagship programs: Tanruprubart for Guillain-Barré Syndrome (GBS), ANX007 for Geographic Atrophy (GA), and the oral inhibitor ANX1502 for autoimmune diseases Tanruprubart (ANX005) in Guillain-Barré Syndrome (GBS) Tanruprubart, a first-in-kind monoclonal antibody for GBS, is advancing towards a Biologics License Application (BLA) submission, with a key FDA meeting scheduled for Q2 2025 - Tanruprubart is a first-in-kind monoclonal antibody designed to block C1q with a single infusion to halt neuroinflammation in GBS, a condition affecting approximately 150,000 people worldwide annually[3] - A meeting with the FDA's Center for Drug Evaluation and Research (CDER) is scheduled for Q2 2025, preceding the planned BLA submission[1][9] - The open-label FORWARD study will initiate in Q2 2025 to provide experience with tanruprubart to patients and physicians in the United States, Canada, and Europe[1][9] ANX007 in Geographic Atrophy (GA) ANX007 is being developed as a potential first-in-kind, vision-preserving treatment for dry AMD with Geographic Atrophy (GA), with enrollment in the pivotal Phase 3 ARCHER II trial expected to complete in Q3 2025 - ANX007 is a non-pegylated antigen-binding fragment (Fab) designed to block C1q locally in the eye, targeting a leading cause of blindness affecting over eight million people globally[5] - Enrollment in the global, pivotal Phase 3 ARCHER II trial is expected to complete in Q3 2025[1][9] - Pivotal topline data from the ARCHER II trial is expected in the second half of 2026[1][9] ANX1502 for Autoimmune Conditions ANX1502 is a first-in-kind oral small molecule C1s inhibitor being developed for autoimmune conditions, with its proof-of-concept study in cold agglutinin disease (CAD) expected to complete in mid-2025 - ANX1502 is an oral small molecule designed to inhibit C1s, a key enzyme in the classical complement cascade, offering a potential convenient treatment for antibody-mediated autoimmune diseases[6] - Completion of the proof-of-concept (POC) trial in up to seven patients with cold agglutinin disease (CAD) is anticipated in mid-2025[1][9] First Quarter 2025 Financial Results For the first quarter of 2025, Annexon reported a net loss of $54.4 million, an increase from the $25.2 million loss in Q1 2024, primarily due to a significant rise in R&D expenses to $48.2 million Financial Summary In Q1 2025, Annexon's R&D expenses more than doubled to $48.2 million from $21.0 million in Q1 2024, resulting in a net loss of $54.4 million, or $0.37 per share - Cash, cash equivalents, and short-term investments totaled $263.7 million as of March 31, 2025, which is expected to fund planned operating expenses into the second half of 2026[1][9] Q1 2025 vs. Q1 2024 Financial Highlights | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | R&D Expenses | $48.2 million | $21.0 million | | G&A Expenses | $9.2 million | $7.6 million | | Net Loss | $54.4 million | $25.2 million | | Net Loss Per Share | $0.37 | $0.21 | Condensed Consolidated Statements of Operations The unaudited statement of operations for the three months ended March 31, 2025, details a total operating expense of $57.4 million, leading to a net loss of $54.4 million, or $0.37 per share Statement of Operations (in thousands, except per share data) | | Three Months Ended March 31, | | :--- | :--- | :--- | | | 2025 | 2024 | | Operating expenses: | | | | Research and development | $48,179 | $20,963 | | General and administrative | $9,226 | $7,609 | | Total operating expenses | $57,405 | $28,572 | | Loss from operations | ($57,405) | ($28,572) | | Interest and other income, net | $3,049 | $3,396 | | Net loss | ($54,356) | ($25,176) | | Net loss per share, basic and diluted | ($0.37) | ($0.21) | Condensed Consolidated Balance Sheets As of March 31, 2025, Annexon's balance sheet showed total assets of $303.0 million, a decrease from $350.1 million at year-end 2024, with total stockholders' equity at $243.8 million Balance Sheet Highlights (in thousands) | | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | $97,122 | $49,498 | | Short-term investments | $166,574 | $262,519 | | Total current assets | $268,162 | $316,461 | | Total assets | $303,027 | $350,071 | | Liabilities and Equity | | | | Total current liabilities | $33,547 | $30,512 | | Total liabilities | $59,239 | $56,966 | | Total stockholders' equity | $243,788 | $293,105 | About Annexon and Forward Looking Statements Annexon is a biopharmaceutical company focused on developing first-in-kind treatments for serious neuroinflammatory diseases by targeting C1q, with its pipeline addressing unmet needs in autoimmunity, neurodegeneration, and ophthalmology - Annexon's therapeutic approach is centered on targeting C1q, the initiating molecule of the classical complement pathway, to stop neuroinflammation before it causes tissue damage[8][10] - The company's pipeline addresses unmet needs in autoimmunity, neurodegeneration, and ophthalmology, aiming to help nearly 10 million people worldwide[10] - This press release contains forward-looking statements regarding trial timelines, regulatory submissions, potential therapeutic benefits, and financial runway. These statements are not guarantees of future performance and are subject to risks described in the company's SEC filings[11]