Annexon (NasdaqGS:ANNX) FY Conference January 14, 2026 02:15 PM ET Company ParticipantsPriyanka Grover - Political Affairs and Public Diplomacy OfficerDouglas Love - CEOJoyce Zhou - Private Equity AssociateConference Call ParticipantsAnupam Rama - Senior Biotech AnalystAnupam RamaAll right, welcome everyone to the 44th Annual J.P.Morgan Healthcare Conference. My name is Anupam Rama. I am one of the Senior Biotech Analysts here at J.P.Morgan. I'm joined by my squad: Joyce Zhou, Priyanka Grover, and Ratih Pin ...

Core Insights - Annexon, Inc. has appointed Dr. Lloyd Clark as senior vice president of ophthalmology strategy and innovation, bringing over 25 years of experience in retina diseases and drug development [1][3] - The investigational therapy ANX007 is the only treatment shown to significantly preserve vision and central retinal photoreceptors in patients with dry age-related macular degeneration (AMD) with geographic atrophy (GA) [2][3] - The Phase 3 ARCHER II trial for ANX007 is expected to complete enrollment by Q3 2025, with topline data anticipated in the second half of 2026 [2][6] Company Overview - Annexon, Inc. focuses on developing novel therapies for neuroinflammatory diseases, targeting the classical complement pathway to prevent tissue damage [7] - The company aims to address significant unmet needs in the treatment of neurodegenerative and ophthalmic diseases, with a pipeline that includes investigational drug candidates for over 8 million patients globally [7] Product Details - ANX007 is designed to block C1q locally in the eye and is currently being evaluated in the pivotal Phase 3 ARCHER II trial, which will enroll approximately 630 patients [2][6] - The primary endpoint of the ARCHER II trial is to prevent a ≥15-letter loss in best corrected visual acuity (BCVA), a well-established functional endpoint for ophthalmology drug approvals [6] Market Context - Dry AMD with GA is a leading cause of blindness affecting over 8 million patients worldwide, with no approved therapies currently available to preserve vision [2][4] - The addition of Dr. Clark to the Annexon team is seen as a strategic move to enhance the company's position in the retina community and improve treatment options for patients with dry AMD [3]

Core Insights - Annexon, Inc. is advancing a late-stage clinical platform of novel therapies targeting classical complement-mediated neuroinflammatory diseases, with significant progress in its portfolio and financial results for Q1 2025 [1][2][9] Group 1: Clinical Development Updates - The FDA meeting for Tanruprubart (formerly ANX005), a potential treatment for Guillain-Barré Syndrome (GBS), is scheduled for Q2 2025 ahead of a planned Biologics License Application (BLA) submission [1][2] - The open-label Tanruprubart FORWARD study is set to initiate in Q2 2025, aimed at broadening patient and healthcare community experience in North America and Europe [1][8] - The Phase 3 ARCHER II trial for ANX007, targeting dry age-related macular degeneration (AMD) with geographic atrophy (GA), is on track for completion in Q3 2025, with pivotal topline data expected in the second half of 2026 [1][2][8] - Completion of the proof-of-concept trial for ANX1502, an oral C1s inhibitor for cold agglutinin disease, is anticipated by mid-2025 [1][2][8] Group 2: Financial Performance - As of March 31, 2025, the company reported $263.7 million in cash, cash equivalents, and short-term investments, providing a runway into the second half of 2026 [1][7] - Research and development expenses for Q1 2025 were $48.2 million, significantly higher than $21.0 million in Q1 2024, reflecting the advancement of priority programs [7][13] - General and administrative expenses increased to $9.2 million in Q1 2025 from $7.6 million in Q1 2024 [7][13] - The net loss for Q1 2025 was $54.4 million, or $0.37 per share, compared to a net loss of $25.2 million, or $0.21 per share, in Q1 2024 [7][13] Group 3: Market Potential and Strategic Positioning - Tanruprubart is positioned as the first potential therapy for GBS, addressing a significant unmet need with no FDA-approved treatments currently available [2][3] - ANX007 aims to be the first vision-preserving treatment for dry AMD with GA, potentially benefiting over eight million patients globally [2][5] - The company's innovative C1 platform is designed to halt harmful neuroinflammation, with a focus on addressing the unmet needs of nearly 10 million people worldwide [2][9]

Core Insights - Annexon, Inc. announced positive Phase 2 ARCHER trial results for ANX007, a novel therapy aimed at preserving vision in patients with dry age-related macular degeneration (AMD) and geographic atrophy (GA) [1][2][3] - The company is preparing for a Phase 3 trial, ARCHER II, which will enroll approximately 630 patients globally [1][10] Group 1: ANX007 and Its Mechanism - ANX007 is a first-in-kind, non-pegylated antigen-binding fragment designed to inhibit C1q locally in the eye, addressing neurodegeneration caused by classical complement pathway activation [3][4] - The therapy has shown significant vision preservation in clinical endpoints such as best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA) [3][6] - ANX007 has received Fast Track designation from the FDA and Priority Medicine (PRIME) designation in the EU, indicating its potential therapeutic advantage [5] Group 2: Clinical Trial Results - In the Phase 2 ARCHER trial, ANX007 demonstrated statistically significant protection against vision loss, with a time and dose-dependent effect observed [6][8] - The treatment effect was maintained during a six-month off-treatment period, suggesting a durable benefit [8] - ANX007 was well-tolerated, with no increase in choroidal neovascularization rates or retinal vasculitis events reported [8] Group 3: Future Developments - The Phase 3 ARCHER II trial aims to prevent ≥15-letter loss of BCVA, a well-established functional endpoint for ophthalmology drug approvals [10] - Topline data from the ARCHER II trial is expected in the second half of 2026 [10] - The trial will further assess safety and visual function outcomes, including low-luminance visual acuity and photoreceptor integrity [10]

Core Insights - Annexon, Inc. is advancing a late-stage clinical platform of novel therapies targeting classical complement-mediated neuroinflammatory diseases, with significant progress in its flagship programs [1][2][9] Group 1: Clinical Programs - ANX005 is positioned as a potential first targeted therapy for Guillain-Barré Syndrome (GBS), demonstrating early and durable functional improvements with a differentiated safety profile [2][3] - ANX007 aims to be the first vision-preserving treatment for dry age-related macular degeneration (AMD) with geographic atrophy (GA), with a groundbreaking global registration path established for approval in Europe and the U.S. [1][8] - ANX1502 is an oral small molecule currently in a proof-of-concept trial for cold agglutinin disease (CAD), with data from up to seven patients expected by mid-2025 [1][5][8] Group 2: Financial Performance - As of December 31, 2024, the company reported cash, cash equivalents, and short-term investments totaling approximately $312 million, providing a financial runway into the second half of 2026 [1][7] - Research and development (R&D) expenses for Q4 2024 were $43.4 million, up from $23.3 million in Q4 2023, reflecting the advancement of priority programs [11] - The net loss for the year ended December 31, 2024, was $138.2 million, compared to $134.2 million for the previous year, indicating ongoing investment in clinical development [11][13] Group 3: Regulatory and Market Context - GBS affects at least 150,000 people worldwide annually, with no FDA-approved therapies currently available, highlighting a significant unmet medical need [8] - The Phase 3 ARCHER II trial for ANX007 is expected to complete enrollment in the second half of 2025, with data anticipated in the second half of 2026 [8] - ANX007 is the only investigational therapy to have shown significant vision preservation in both normal and low light conditions, as demonstrated in the Phase 2 ARCHER trial [8]