Annexon(ANNX) - 2025 Q2 - Quarterly Results

Executive Summary & Highlights This section provides an overview of Annexon's Q2 2025 financial results, clinical portfolio progress, and CEO commentary Second Quarter 2025 Financial Results, Portfolio Progress and Key Anticipated Milestones Annexon reported its second quarter 2025 financial results, highlighting significant progress across its clinical portfolio, including Tanruprubart for GBS, Vonaprument for Dry AMD with GA, and ANX1502 for autoimmune conditions. The company's cash position is projected to support operations into the fourth quarter of 2026 - Annexon reported Q2 2025 financial results and highlighted portfolio progress for Tanruprubart (GBS), Vonaprument (Dry AMD with GA), and ANX1502 (oral C1s inhibitor), with cash supporting operations into Q4 2026[1] - Tanruprubart for GBS is advancing through regulatory interactions, with MAA submission in Europe anticipated in Q1 2026 and ongoing discussions with FDA for BLA support[1] - Enrollment for the global Phase 3 ARCHER II trial of Vonaprument for Dry AMD with GA was accelerated, with topline data expected in H2 2026[1] - ANX1502, a first-in-kind oral C1s inhibitor, exceeded target concentration in fasted patients, with an update on the proof-of-concept CAD study expected by year-end 2025[1] CEO Commentary CEO Douglas Love emphasized the consistent validation of Annexon's C1 platform across its therapeutic candidates, highlighting strong Phase 3 results for Tanruprubart, accelerated enrollment for Vonaprument, and promising initial exposure data for ANX1502 - CEO Douglas Love emphasized the consistent validation of Annexon's C1 platform across multiple potential best-in-class therapeutics for complement-mediated diseases[2] - In GBS, approximately 90% of tanruprubart-treated patients improved by week 1, and more than twice as many achieved normal health at week 26 vs. placebo in Phase 3[2] - Accelerated enrollment in the Phase 3 ARCHER II trial for vonaprument in dry AMD with GA, with topline data on pace for H2 2026, aims to provide a new vision-preserving treatment[2] - Annexon is capitalized into Q4 2026, through pivotal Phase 3 data in GA[2] Recent Corporate and Clinical Program Updates This section details recent advancements in Annexon's clinical programs, including regulatory progress, trial enrollment, and initial data Tanruprubart (ANX005) in GBS Tanruprubart, an immunotherapy for Guillain-Barré Syndrome (GBS), is progressing towards global registration, with MAA submission in Europe anticipated in Q1 2026 and ongoing discussions with the FDA regarding a BLA submission - Tanruprubart is a targeted immunotherapy for GBS, a rare neuromuscular emergency affecting ~150,000 people worldwide annually, with no FDA-approved therapies[3] - MAA submission for European registration is expected in Q1 2026[3][7] - Ongoing discussions with the FDA regarding the generalizability package to support a BLA submission, with an update expected upon further regulatory clarity[3][7] - Completed a generalizability package including a Real-World Evidence study showing favorable outcomes versus SOC, and a drug-drug interaction safety study with IVIg[7] Vonaprument (ANX007) in Dry AMD with GA Vonaprument, a neuroprotective inhibitor for dry Age-related Macular Degeneration (AMD) with Geographic Atrophy (GA), has completed accelerated enrollment for its global Phase 3 ARCHER II trial, with topline data anticipated in H2 2026 - Vonaprument is a neuroprotective inhibitor for dry AMD with GA, a leading cause of blindness affecting over eight million worldwide, with no approved vision-preserving therapies[4] - Accelerated enrollment of 659 patients completed for the global, pivotal, sham-controlled, double-masked Phase 3 ARCHER II trial[7] - Global registration path established with U.S. and European regulators, supporting potential for first approval in both regions for vision protection in GA[7] - Topline Phase 3 ARCHER II trial data is expected in the second half of 2026[7] ANX1502 for Autoimmune Conditions ANX1502, a first-in-kind oral C1s inhibitor for autoimmune conditions, has demonstrated exposure exceeding target concentrations in fasted patients during its ongoing proof-of-concept (POC) study for cold agglutinin disease (CAD), with an update expected by year-end 2025 - ANX1502 is a first-in-kind oral small molecule inhibiting activated C1s, offering potential for selective upstream classical complement inhibition with oral administration convenience[5] - Exposure exceeded target concentrations in fasted patients in the ongoing open-label POC study for cold agglutinin disease (CAD)[7] - Evaluation of PK/PD in relation to food intake, and reduction in complement and bilirubin markers as a measure of hemolysis, are ongoing[7] - An update on the POC trial in CAD is anticipated by year-end 2025[7] Second Quarter 2025 Financial Results This section presents Annexon's Q2 2025 financial performance, covering cash position, operating runway, operating expenses, and net loss Cash and Operating Runway As of June 30, 2025, Annexon reported $227.0 million in cash, cash equivalents, and short-term investments, which is expected to fund operations and late-stage milestones into the fourth quarter of 2026 | Metric | June 30, 2025 | | :----- | :------------ | | Cash and cash equivalents and short-term investments | $227.0 million | - Cash, cash equivalents, and short-term investments are expected to fund planned operating expenses and late-stage milestones into Q4 2026[6] Operating Expenses and Net Loss Annexon's R&D expenses significantly increased in Q2 2025 to $44.2 million, reflecting accelerated program advancement, while G&A expenses slightly decreased, resulting in a net loss of $49.2 million, or $0.34 per share | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | Change (YoY) | | :-------------------------------- | :--------------------- | :--------------------- | :----------- | | Research and development (R&D) expenses | $44,160 | $25,026 | +76.4% | | General and administrative (G&A) expenses | $7,566 | $8,554 | -11.6% | | Net loss | $(49,156) | $(29,610) | +66.0% | | Net loss per share, basic and diluted | $(0.34) | $(0.23) | +47.8% | - R&D expenses increased significantly due to the advancement of priority programs (GBS, GA, ANX1502)[10] Financial Statements (Unaudited) This section provides unaudited condensed consolidated financial statements, including statements of operations and balance sheets Condensed Consolidated Statements of Operations (Unaudited) The condensed consolidated statements of operations show a net loss of $49.2 million for the three months ended June 30, 2025, an increase from $29.6 million in the prior year, primarily driven by higher research and development expenses | Metric (in thousands) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------------------ | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development | $44,160 | $25,026 | $92,339 | $45,989 | | General and administrative | $7,566 | $8,554 | $16,792 | $16,163 | | Total operating expenses | $51,726 | $33,580 | $109,131 | $62,152 | | Loss from operations | $(51,726) | $(33,580) | $(109,131) | $(62,152) | | Interest and other income, net | $2,570 | $3,970 | $5,619 | $7,366 | | Net loss | $(49,156) | $(29,610) | $(103,512) | $(54,786) | | Net loss attributable to common stockholders | $(51,013) | $(29,610) | $(105,369) | $(54,786) | | Net loss per share, basic and diluted | $(0.34) | $(0.23) | $(0.71) | $(0.43) | | Weighted-average shares used in computing net loss per share, basic and diluted | 148,320,803 | 130,132,960 | 148,215,392 | 126,403,081 | | Stock-based compensation expense (R&D) | $2,688 | $2,311 | $5,517 | $4,593 | | Stock-based compensation expense (G&A) | $1,517 | $2,631 | $3,766 | $5,009 | Condensed Consolidated Balance Sheets (Unaudited) The condensed consolidated balance sheets show a decrease in total assets from $350.1 million at December 31, 2024, to $264.6 million at June 30, 2025, primarily due to a reduction in short-term investments, with total stockholders' equity also decreasing over the same period | Metric (in thousands) | June 30, 2025 | December 31, 2024 | | :------------------------------------ | :-------------- | :---------------- | | Assets | | | | Cash and cash equivalents | $132,288 | $49,498 | | Short-term investments | $94,729 | $262,519 | | Total current assets | $230,620 | $316,461 | | Total assets | $264,573 | $350,071 | | Liabilities and Stockholders' Equity | | | | Total current liabilities | $40,642 | $30,512 | | Total liabilities | $65,556 | $56,966 | | Total stockholders' equity | $199,017 | $293,105 | | Total liabilities and stockholders' equity | $264,573 | $350,071 | About Annexon Annexon Biosciences is a biopharmaceutical company focused on developing first-in-kind therapeutics to halt classical complement-driven neuroinflammation by targeting C1q, the initiating molecule of the classical complement pathway - Annexon is a biopharmaceutical company developing first-in-kind therapeutics targeting classical complement-driven neuroinflammation for serious neuroinflammatory diseases[8] - Their scientific approach focuses on C1q, the initiating molecule of the classical complement pathway, to stop neuroinflammatory cascades[8] - The pipeline spans autoimmunity, neurodegeneration, and ophthalmology, aiming to address unmet needs for nearly 10 million people worldwide[8] Forward-Looking Statements This section contains forward-looking statements regarding Annexon's future performance, therapeutic benefits, regulatory timelines, clinical trial outcomes, commercialization efforts, and financial outlook, which are subject to various risks and uncertainties - The press release contains forward-looking statements regarding potential therapeutic benefits, regulatory timings, clinical trial results, commercialization efforts, financial runway, and other future events[9] - These statements are subject to risks and uncertainties that could cause actual results to differ materially, as detailed in SEC filings[9] - Annexon undertakes no obligation to publicly update any forward-looking statements, except as required by law[9] Contacts This section provides contact information for investor relations and media inquiries Investor Contact For investor inquiries, please contact Joyce Allaire at LifeSci Advisors via email - Investor inquiries can be directed to Joyce Allaire at LifeSci Advisors via jallaire@lifesciadvisors.com[10] Media Contact For media inquiries, please contact Beth Keshishian via phone or email - Media inquiries can be directed to Beth Keshishian at 917-912-7195 or beth@bethkeshishian.com[11]